Immunologist Matthias von Herrath to Work for Novo Nordisk

For those of you who missed the news, it was announced today that immunology researcher Matthias von Herrath will be sharing his duties with La Jolla Institute for Allergy and Immunology (the announcement actually says he'll stick around La Jolla part-time, probably to continue with his existing teaching obligations) to take a position leading Danish insulin giant Novo Nordisk's type 1 diabetes research and development center in Seattle which will focus on autoimmunity treatments (see the press release at http://prn.to/zQ3xJZ).  For those of you in the Seattle area, the type 1 diabetes research and development center will be will actually be housed in South Lake Union neighborhood alongside Novo's team of autoimmune/inflammatory disease researchers.  The Novo Nordisk Type 1 Diabetes R&D Center is expected to open this summer (2012), and will be staffed by approximately 20 researchers.

As I reported in August 2008, Novo Nordisk actually opened a research facility in Seattle back in 2008 (see my blog posts at http://bit.ly/w0I2Y1 AND http://bit.ly/wh50SH for details), but in my assessment, not much progress has been reported since that happened, with nothing even mentioned in the company's investor presentations or SEC filings.

Readers may recall that not one but TWO autoimmunity treatments designed for type 1 diabetes both failed to meet their efficacy endpoints in 2011: specifically, teplizumab which was being developed by a Maryland-based biotechnology company known as Macrogenics and big pharma partner Eli Lilly and Company, as well as otelixizumab which was being developed by Massachusetts-based biotech company Tolerx and big pharma partner GlaxoSmithKline. It should be noted that there is still additional research being done into these products to determine if these drugs might work on certain groups of patients, but it seems clear that neither is likely to become a product administered to all newly-diagnosed patients to arrest the autoimmune response that causes type 1 diabetes.

Perhaps Teva's [Andromeda's] recent progress in the field is prompting them putting some genuine resources into it now? (see http://buswk.co/viRH12 for more, also catch the post at DiabetesMine.com in which I was asked for input at http://goo.gl/DkI4u).

Aharon Schwartz, vice president of innovative ventures at Teva told Bloomberg BusinessWeek in an interview on the company's Diapep 277 in late 2011 (see my 2010 coverage on that treatment HERE):

"When we started we thought, even if the results are positive, we would have competition. Now we are the only game in town."

My guess is that statement didn't sit well with Novo Nordisk's top brass in Denmark.  I can only hope that Novo Nordisk, especially after it's poorly chosen Paula Deen endorsement for Victoza (check out the Forbes story HERE) disaster (which rightfully deserves some of the criticisms it has received from others within the pharmaceutical industry) will make good on the type 1/autoimmunity research.

Novo Nordisk reportedly sought Dr. von Herrath to lead the Center due to his stellar record in type 1 diabetes research.

"Dr. von Herrath is generally regarded as one of the top researchers in the world in type 1 diabetes," said Dr. Jacob Sten Petersen, Novo Nordisk corporate vice president. "He has made several key advances and is the kind of dedicated, talented researcher that can fuel true innovation in type 1 diabetes treatment." Dr. von Herrath is the recipient of the Juvenile Diabetes Research Foundation's (JDRF's) prestigious Scholar Award in 2008 for Live Imaging of Islet Destruction (the announcement for that can be viewed HERE, and the stunning videos can be accessed HERE, be sure to look towards the end for "supplemental data" for the actual videos) and he also received the Outstanding Scientific Achievement Award from the American Diabetes Association.

The company (Novo Nordisk) has done very little with the Seattle autoimmunity and inflammation research facility it opened since it was announced back in 2008 (see HERE), so the recent news that the company had hired a world-class immunologist could be a positive sign.  However, I would dare say that it will likely be a few years before this move is likely to yield much of anything, but it's nevertheless positive.

End-of-Year Reconciliations; New Year, New Insurance

Happy New Year Everyone! What would a new year's post be withough including the awesome ABBA song "Happy New Year" (it's Flash audio, sorry for the Apple loyalists who really can't enjoy it the way the web was truly meant to be enjoyed) in honor of the occasion? See HERE (it's a Vietnamese site) to download the MP3 to the song (it was working when I wrote this post).



But beyond New Years tunes from 1970's-era Pop Stars from Sweden, the "Happy" New Year had a particular meaning for me in 2012, at least when it comes to managing diabetes. I ended 2011 much like I've ended prior years. I spent part of the last few weeks of 2011 (I began shortly before Thanksgiving) trying to make sure all the t's were crossed and the i's were dotted when it comes to the byzantine healthcare coverage system. I can be thankful for having coverage, but am less-than-thankful at how freakin' complicated all of it is.

Part of my year-end process also included making my last 90-day supply orders from Medco for the year, especially since I had a very high-deductible plan and because insurance was finally paying 100% of these orders at the end of the year I wanted to get what I was entitled to IMHO. Of course, that starts all over on January 1, so at least if I was able to stock-up, I should have sufficient supplies for a few months into the new year.

Personally, in spite of the big cost-savings for insurance companies, I find absolutely nothing convenient about mail-order pharmacies. Notably, I can't have them shipped to my apartment because stuff literally tends to disappear from my doorstep (I live in the city) or worse, dogs treat packages left at my front door like a fire hydrant. Either way, "home" delivery simply isn't a viable option. Of course, I am seeing both CVS and Walgreens are now promoting the ability to fill 90-days of scripts at the retail pharmacy, provided of course, your plan ALLOWS retail pharmacy fulfillment (my plan did not).

As a result, I am obliged to ship all this crap to my office. I have boxes of testing supplies, needles and the rest shipped to work. When those stupid pillow-like containers they put temperature-sensitive items like insulin in arrive, I have to figure out how lug all that crap home on public transit (the subway). I usually toss all the unnecessary packaging (including like my 300th ice-pack, I have more of those than anyone needs, and don't have room in my tiny freezer for any more, so they go directly into the trash -- my, what an efficient "system" mail-order is!). When all is said and done, they take up far less space and make it easier for me to carry home on the subway ride home.

As I already hinted, I pretty much had to order ALL of my scripts by mail-order with this plan (I had a limit of just $250/year in retail pharmacies, mainly for emergency prescriptions for things like antibiotics which patients simply can't wait a week to receive), I knew the drill very well. I also had many other accounts to manage as part of my year-end ritual: HRA, FSA, transit, and I needed to reconcile those. The reason for my doing this is that I've learned over the years that it saves much aggrivation and money. The last thing I need to be dealing with is an unexpected bill for services and NOT being able to expense it because the bill arrived too late to be submitted for reimbursement.
Beyond Medco stuff, I also looked at balances in all of my various healthcare (and transit) accounts. I also had an HRA (health reimbursement account paid for by my emplployer) since my employer subsidized part of my huge deductible (that was a great benefit, but it meant having yet another account that I had to keep track of). And, I had an FSA (again, great benefit, but yet another account for me to keep track of). To top things off, I also had a transit account (again, nice because it enables me to pay for these things using pre-tax dollars reducing my taxible income, but that too was yet another account for me to reconcile and keep track of. Fortunately, my administrator allowed online access to these accounts, so I could login and check out the details without much difficulty. Guess what -- I DID find a discrepancy!

U.S. Leads the World in healthcare (The Third-World, that is)


As it turns out, there was an issue related to services from June 15th, when I saw my endo this summer. I was back to see him in September, so this was a really old claim issue that no one bothered to ask about until I raised the issue with a question. Man, this kind of crap really pisses me off. Anyone who claims the U.S. has a "world-class" healthcare system is in my opinion, an absolute fucking idiot. The U.S. may lead the world in healthcare -- the third world, maybe! I doubt anyone in Europe (Western or Eastern) has to deal with all this payment "he-said, she-said" bullshit, and when interviewed, few would ever want the U.S. system. It just sucks.

In the process, I discovered that my FSA/HRA administrator believed that I owed them $204.40, but when I questioned them on their math, they actually acknowledged that the amount should really have been $172.90 (and they're the ones entrusted to "manage" tens of thousands of healthcare dollars from both my employer and me on MY behalf?! No wonder U.S. healthcare costs are so out-of-control!!). It appears that my insurance company had covered some services from my June 15 endo appointment that the FSA administrator wasn't paid for (since I pay upfront using my HRA/FSA account, and then wait for reimbursement since my endo was out-of-network, although I can expense my co-pays using my FSA), so I called them to figure out whether they were trying to charge it against the wrong account or something (stranger things have happened).

As it turned out, my insurance company supposedly paid for that particular claim, but neither my doctor nor I was the recipient of their payment! So I called my insurance company and after dealing with VRU-hell to finally reach a real, live person (and, they weren't in India, but New York of all places!), I learned that they actually did send 2 checks totalling $172.90, but neither of those checks were ever cashed. I said to the rep "Gee, that just might be a sign the check got lost." I mean, how many recipients, whether it was a doctor, a lab, or a patient would be holding onto a reimbursement check for nearly $200 for 6 months, uncashed? Hello?! Fortunately, this had (has?) a seemingly easy solution.

I told the insurance company "I need you to stop payment on those checks and reissue them both, and mail them to ME" (evidently, they were made payable to me). She did not give me any excuses about how she might not be able to do it for me (which I was expecting), but she cautioned me that it may take 6 weeks for those checks to arrive, which should be in February sometime. I can live with that. I guess my HRA/FSA is going to have to wait for 6 weeks before they get paid, but its no skin off my back, even though all of this is actually MY money, but I had it taken using pre-tax dollars long ago and if I don't use it, it disappears permanently. This is the kind of stuff that makes me wonder precisely what planet all those people who believe and actually claim the U.S. healthcare system is world-class actually live on? The way I see it, the U.S. healthcare system has far too many people with their hands collecting (and skimming something off the top) all this money, but not enough people auditing or actually ensuring that patients really receive quality healthcare. Don't blame patients or doctors, blame all the other layers of "covered entities" (as defined by HIPPA) for that.

Anyway, it looks like when all this billing B.S. is finally resolved, I still had a few extra dollars in my FSA to buy about 6 months of contact lenses and some Dex4 LiquidBlast shots, and because I already had my endo write me "prescription" for those items when I went in September, so I can indeed expense them, but I had to wait until my last two prescriptions of the year were filled via Medco, so the week before Christmas for me involved a lot of shopping, but not for holiday gifts, but FSA-expensible items!

Experience has proven that a few phone calls at the end of the year makes sure I'm not stuck the next year.

As I've lamented a number of times over the years, I work boutique consulting firm, and although I really love the work environment at this company, as a smaller-sized firm with offices in New York and London, the company has been challenged with skyrocketing healthcare costs in the U.S. (it's a non-issue in our U.K. office, which has national healthcare). U.S. insurance companies, as I've discovered, play games that ought to be illegal with low rates to lure new clients in, only to try and double the rates (or more) they charge the following year. As a result, I've literally had about 5 different healthcare (insurance) providers over the last 7 years. I've seen the ugly, the $#!tty and the reason why U.S. healthcare reform was a real necessity for U.S. business: because without it, the U.S. economy simply cannot create quality jobs -- we're likely to produce dozens of jobs that require a paper hat (McJobs, anyone?) or in places like Walmart, but decent jobs, not so much. We're not talking about trying to compete with China (who the hell would want the working conditions those poor workers, many of them children, tolerate?!), we're talking multinational companies choosing to do things like manufacture stuff in places like Canada, Europe or Japan rather than in the U.S. because the liability for healthcare in the U.S. is simply uncompetitive with these places.

Last year, I thought I was certain to join the growing ranks of Americans (Mercer Consulting estimated the percentage around 40%) who have very high-deductible plans without any subsidies from thier employers. In fact, I had been saving in anticipation of this unfortunate reality for much of 2011 knowing what my employer had said was going to be the case in 2012. However, a surprising thing occurred in 2011, namely that my healthcare insurer (one that overwhelmingly serves the City of New York's employees, people like the FDNY, NYPD, teachers in the public school system, City Hall employees, etc.) had decided to stop offering the plan my employer was offering in 2012. The company, which is preparing to de-mutualize itself to become yet another for-profit insurance company (I wasn't exactly in love with this company, but had learned to work within their confines over the past 2 years), stopped offering a plan with out-of-network coverage, so my employer decided to remarket it's healthcare business, extending our coverage with the existing carrier until the end of 2011.

As a result, although our plan year ended officially in August 2011, we remained with the same provider until the end of 2011 while my employer remarketed it's healthcare business. Although so-called insurance "exchanges" mandated by the new healthcare law to be done at the state level should address this starting in 2014, that's still 2 years from now. Even though New York is not among the states that have opted out of the healthcare law (none can opt out of creating the exchanges), small employers are still challenged today to buy healthcare insurance at costs comparable to what large employers can, and the rates of cost increases tend to be in the range of 40% or more PER YEAR.

Anyway, because my employer relies upon professionals with high education levels (our entire staff has a degree of some sort, with more than a few staff members with master's degrees, and even a few doctorates among us), they are acutely aware that they can't get away with Walmart-like benefits and survive. So they actually have a vested business interest seeing healthcare reforms, like insurance exchanges, be launched because they are the very businesses which are being squeezed out of the healthcare market today. Unfortunately, 2014 is a long way off and they have needs TODAY.

The result of the remarketing the company's healthcare business resulted in my employer contracting with what is referred to as a PEO (professional employer organization) to manage various items like benefits, payroll, etc. The biggest benefit is that as a "co-employer", I will be part of a healthcare plan that has over 10,000 "employees" rather than a small company with only 50 or so employees (we also have a few more in our London office, but those employees aren't included as part of the PEO arrangement since they have national healthcare coverage in the U.K. -- in spite of local complaints, it still blows the U.S. system away in terms of long-term patient outcomes AND actual cost according to many studies). Starting January 1, 2012, my healthcare provider officially became United Healthcare. I had United a number of years ago, and although I would never use the term "love" when it comes to any, for-profit healthcare insurance company, my past experience with United Healthcare was pretty positive, especially when compared to WellPoint/Anthem, which I cannot say enough bad things about.

All of this brings me back to the whole Happy New Year theme I began with. In 2012, much of the crap I dealt with with high deductibles, separate HRA, as well as FSA and Transit accounts has been made easier. Now, I can continue to see my own endo, but he'll actually be in-network! This new arrangement also means that the costs of healthcare coverage are unlikely to increase on average 40%-50% every year, as the PEO they've contracted has seen increases in the range of 7% for the last 5 years from what we've been told.

The new arrangement also means that an entire layer of extra bullshit will be eliminated from last year's equation. Another thing I've done when I got my new insurance card was notifying my new pharmacy benefits manager (PBM) United Healthcare uses that I want them to transfer my prescriptions from Medco to (which I believe is also Medco, but it still needs to be transferred) so I don't need to get brand new scripts from my doctor. This way, I was able to start in January when my new insurance plan year begins, I could theoretically fill them on day 1 if I wanted to (I'll wait until the FSA debit card arrives on that). I discovered I could have Rx's transferred from one pharmacy to another last year, when I found Medco's prices for generics to be significantly higher than using Walgreens.com, even if I did have to pay the full cost out-of-pocket and the cost did not apply towards my deductible amount, it was still a much a better deal. To facilitate this, the mail-order pharmacy of your new insurance company must contact the old pharmacy and ask them to transfer the scripts to them.

As it turns out, my endo is part of United Healthcare's network, and even the brands of insulin I prefer to use are on their formulary. I may ultimately have to give up my preferred meter (which was my Bayer Contour USB, but I can live with that) for another brand. On the bright side, a Dexcom CGMS may finally be a realistic option for me with the new plan, so apparently the Great One taketh away while simultaneously giving. I'm still figuring out what labs they use. I think they switched to LabCorp, which may be the nation's second largest laboratory (after Qwest), but that company still has relatively few locations where I live, and I've seen the company build new locations all around for the past few years, a process that's likely to continue.

When it comes to test strips, I am of the opinion that these are all pretty undifferentiated commodities. Most testing supplies (except for the Agamatrix/Sanofi system which I'll get to in a second) of the big brands of regular finger-stick meters all use the same basic electrochemical technology.

Hence, few products that have any true points of differentiation, rather they have come to rely on lame marketing gimmicks like coloring the strips blue and using truly unbelievable ploys such as "Double-Sure" technology, which I see as negated and made completely irrelevant by rules of proper testing, namely washing your hands before testing, but hey, it sounds catchy, doesn't it? (Whoever their ad agency is stinks IMHO!), but as noted, that doesn't do much to encourage me to stick with J&J. My endo gave me samples of both the J&J OneTouch Utra meter (I tossed my old one out when I switched to Bayer Countour USB) and Roche Accu-Chek meters since both of them are "preferred" brands on United Healthcare's formulary. I'll sample them both over the next month, and let my endo which one I want to continue with for the next few months.

Who knows, maybe Sanofi's i-Thing (also known as the iBGStar [http://www.ibgstar.us/]), which right now only works on Apple's iPhones and iPodTouch devices [I have the latter at least], but hopefully someday Sanofi will come the the realization that Apple's much-beloved iOS only has around 16% of the global SmartPhone market compared to Google's Android which now has over 52% of the market, see HERE) and is growing much more rapidly than Apple's iOS is) might actually be covered by then. Since it has only had FDA approval for a few weeks, that may be coming before too long. I could always go back to J&J's OneTouch Ultra piece-of-crap for a few months until that happens, after all, I lived with their product before switching meter brands (see HERE), even though there was absolutely nothing I liked about their product which would make me a "loyal" user. I suspect with the Sanofi moves, as well as some smart technology Roche has to help track insulin on board without the use of an insulin pump (see HERE) which is already being sold in the U.K. but is pending FDA approval may someday render the present oligopoly J&J has on the U.S. home diagnostics history in the not-too-distant future unless J&J cleans up their act. Besides, J&J has had an unprecedented number of recalls across the company, raising questions in my mind as to whether it deserves my business. Accu-Chek has a genuine shot this time, although depending on what Sanofi does, I make no guarantees on staying with the brand for the entire year.

Anyway, with that, I'm hopeful 2012 will bring fewer healthcare coverage hassles! Wishing my readers similar hassle-free coverage in 2012.

Wayback Wednesday: Holiday-Themed Posts at Scott's Web Log

One of the things I pride myself on here at Scott's Web Log is that a visitor can visit virtually any post from years ago, and virtually all of the links contained in my old posts still work. I say most because keeping the links "alive" is no small task, especially since I've been blogging since 2005. Although I don't blog as often as I once did (I'm still on Twitter pretty much daily), over time, with the internet being one of the most ephemeral of media, news items move, YouTube videos are taken down, or a news organization that publishes items reorganizes their own website and all their link addresses change. Hence, maintaining the links in my posts is an ongoing effort!

To a large extent, I have come to rely on the Internet Archve "Wayback Machine" which is nicknamed for the time-travel device featured in "Rocky and Bullwinkle" cartoons (which ran on TV from 1959 to about 1973, but were replayed in syndication throughout the 1970s). I elaborated more on that when I told visitors how they could retrospectively visit my recently-departed (see HERE if you missed the news) friend Deb Butterfield's site, and while my original page on that disappeared with free webhosting by Geocities, it remains permanently available, see HERE. Incidentally, while Geocities' days of free website hosting may be history, some of us have discovered that Google presently offers some limited free websites at Google Sites which happens to be where I host my disclosure policy. So for those of you with a desire to build your own website without paying for it, thanks to the Google advertising machine, you can still do so!

Back to the Wayback Machine.

These days, I don't think Rocky and Bullwinkle cartoons can even be found on Cartoon Network's Boomerang cable channel, as kids today seem to prefer computer animation or at least top-rated classics like Warner Brothers' Looney Tunes over the old-school cartoons, but Rocky and Bullwinkle are preserved forever on DVD, and I would imagine are therefore available for streaming via Netflix (although I'm still new to streaming videos having just acquired a Sony Streaming Media Player, so those may or may not be there yet, I'm not certain). Anyway, keeping my all of my links in old posts active is a full-time job, and while I try to stay on top of it,every once and a while, a link stops working. Just know that those are relatively rare when you visit the archives of Scott's Web Log.

Anyway, over the years, I have posted a few "holiday" themed blog posts, and in honor of the "Wayback Machine" noted, I'm referring to today's post as a "Wayback Wednesday" post featuring some holiday-themed posts from years past. Have a look at a few of my former holiday posts (along with some comments I'm adding) here:

Season's Greetings 2008
(Includes some parodies of classic holiday videos)

On a somewhat (marginally?) related note, check out this post entitled "10 Things You Probably Didn't Know About 'A Charlie Brown Christmas' HERE for some interesting things you might not know about the Charles Schultz classic holiday special.

Then, last year, I did this post which I'm still kind of proud of. Re-writing the lyrics to the holiday song "Santa Baby" really stretched my creative imagination!

Santa D-Baby (the D stands for diabetes)

For some more retro stuff, check out the site entitled BetamaXmas.com (Get it? Betamax, the now defunct rival VCR format to VHS from Sony ... a few years from now, readers may even ask what a VCR is/was!) which I learned about from another blog known as GenXTinct.com. More details can be found at their site, which is at http://www.genxtinct.com/2011/12/betamaxmas.html.

I hope to have time to write again before the New Year, but to everyone in the diabetes online community, please enjoy this holiday retro-moment and have very happy holidays!

Dollars for Docs Discussion

First, I want to acknowledge Ellen Ullman (@CureT1Diabetes) who tipped me off to this event on Google+. Although I might have enjoyed the opportunity to attend this in person and ask questions of the presenters, alas, I had other plans that evening.

My readers may recall that earlier this year (on Valentines Day, no less!), I wrote a post (see HERE) about a new database that was assembled by ProPublica, which is a non-profit corporation based in NYC. ProPublica describes itself as "an independent non-profit newsroom that produces investigative journalism in the public interest". That database is known as Dollars for Docs.

ProPublica senior reporters Charles Ornstein and Tracy Weber, both past winners of the Pulitzer Prize, are the lead reporters on the Dollars for Docs project that examined how pharmaceutical company payments to doctors for consulting, speaking, research and other expenses can have undue influence on the drugs they prescribe to patients. This was a discussion with someone else whose opinion I respect, specifically former editor of the New England Journal of Medicine and author of "The Truth About the Drug Companies", Dr. Patricia Angell. My readers may recall that I've quoted her from a 2007 editorial she wrote for the Boston Globe (see http://bo.st/t9B90w for that) entitled "Taking back the FDA" about how much of the FDA and much of its staff are now indirectly on the payroll for the very industries it is supposed to be regulating. In this video, Dr. Angell speaks with ProPublica senior reporters Ornstein and Weber.

In my opininion, the biggest and most important component of this research was the development of a searchable database that shows over $760 million worth of payments from pharma companies to doctors. Dr. Angell, who teaches in Harvard's Division of Medical Ethics, also wrote a book that explains how "Big Pharma" uses much of their revenue for big marketing campaigns instead of research and development.

In the following video, the trio talk about why pharma makes payments to doctors, whether the new healthcare law might help make these payments more transparent, what patients should ask their doctors about their prescriptions, and who benefits most in the end. I would just paraphrase a relevant paragraph from my original post covering the Dollars for Docs database post that's worth repeating:

Under the new U.S. healthcare law, starting in 2013 [that's next year!], ALL drug/biotech/medical device companies selling products in the U.S. will be required to disclose this data in a public database to be operated by the U.S. Department of Health and Human Services. Let's just call the Propublica "Dollars for Docs" database a sneak peek at what we're likely to see much more of in the next few years.

However, there are legal challenges to portions of this landmark legislation (the healthcare law), and it's popular in some circles to talk about dismantling it, but it's clear that it's not going away anytime soon, even while 26 states try to challenge the law and the federal government. In fact, earlier this week, The New York Times published an infomative article (see http://nyti.ms/rVL7cE) which indicated that although the U.S. Supreme Court agreed to hear a challenge to the 2010 healthcare law, it only agreed to hear appeals from just one decision, which was from the U.S. Court of Appeals for the 11th Circuit in Atlanta, which was the only one so far that struck down the so-called "individual mandate" which obliges everyone to purchase healthcare insurance. The appeals court went no further, severing the individual mandate from the rest of the law, at least for the moment.

Sidenote (you can skip the next 2 paragraphs if you're pressed for time):

This ProPublica discussion with Dr. Patricia Angell took place earlier this week, on Tuesday, November 8, 2011 at 6:30 pm EST in a former tenament that was restored as a museum and public space on Manhattan's (NYC) Lower East Side. The Lower East Side of Manhattan has, in recent years. become a pricey neighborhood (in fact, most of Manhattan is pricey these days, with former areas that were as recently as the 1990's considered, shall we say, dicey becoming playgrounds for the wealthy, complete with the neighborhood's own Whole Foods Market and everything). In other words, the area has gone from dicey to pricey, but was once a storied area of how immigrants once lived in cramped tenements – today's immigrants are more likely to live in places like the Bronx, Queens, parts of Brooklyn as well as parts of Northern New Jersey, having been completely priced out of Manhattan. We may like to think that things are much better for immigrants today, but things aren't really much different today, rather, they have changed form.

Today's immigrants to New York City often live in what appears to be regular housing (such as the rows and rows of two-family homes that characterize the Borough of Queens), but a growing amount of housing stock in these areas has been carved up illegally, which is a big concern in the area I live. I see many old homes around me with an unbelievable number of tenants living there, often with children. In effect, the buyers of these properties have created one or more additional dwelling units within a home without first receiving the approval of, and permits from, the NYC Department of Buildings. Some were formerly 2 family homes have been illegally converted into spaces that now are home to three or more families (see HERE for more). I think of this and compare it to a "whack-a-mole" where you hit one that pops up, and as soon as you do that, another one pops up in another location. But the discussion tenements are digression, other than the tenement museum was the location where the Dollars for Docs Discussion took place.

Back to the ProPublica Dollars for Docs Video

I should forwarn you that you can actually skip ahead about 6 1/2 minutes into the video through all the obligatory introductions in order to get directly to Dr. Angell's speech and the rest of the conversation. In this video, Dr. Angell and the ProPublica producers of Dollars for Docs discussed what they found. The short link to this video can be found at http://ustre.am/:1fbSD:



I will close by once again including access to the Dollars for Docs database HERE:

In all, I think this videocast was an informative discussion. Please, share your thoughts!

The Business of Diabetes: Geron No Mo!

I hope you'll pardon my title (its supposed to be a pun meant to sound like "Geronimo!", an exclamation used by anyone about to jump from great heights, which I thought was fitting this case). The company conducting the first-ever FDA-sanctioned human clinical trial in the U.S. of a therapy using human embryonic stem cells said that it was stopping that trial and exiting the stem cell business (see http://nyti.ms/rTNktH for The New York Times article on this) altogether. Like many biotech startups, Geron Corp., a Menlo Park, California-based company, isn't yet profitable. While this company helped pay for the initial derivation of human embryonic stem cells at the University of Wisconsin in the late 1990s, which gives it access to some patent rights in the field, like all startups, venture capitalists and shareholders want to see concrete progress and be assured that their investments might actually yield something in the foreseeable future.

Hence, on Monday, November 14, 2011, Geron Corp. announced that, effective immediately, the company would focus on its oncology programs, and pull the plug on any further development of its stem cell programs. However, the company is now seeking partners for its novel stem cell assets. Of particular interest to the type 1 diabetes community is what Geron referred to as pancreatic islet cells (known as "GRNIC1") for diabetes. Geron was granted U.S. Patent No. 7,033,831 in April 2006 covering the production of insulin-secreting cells from hESCs as well as two U.K. patents covering similar production methods. Geron also has a worldwide exclusive commercial license covering hESC-derived islets from the Wisconsin Alumni Research Foundation.

Geron once boasted that not only had it differentiated human embryonic stem cells into islet-like clusters ("ILCs"), but unlike many other previous efforts to culture islets in vitro, Geron also claimed that its (along with its collaborators at at the University of Alberta) ILCs actually secreted insulin in response to elevated glucose levels (meaning they were appropriately glucose-responsive). Many other similar efforts to differentiate stem cells into insulin (and amylin) producing pancreatic beta cells had failed to respond properly to blood glucose levels. The protocol Geron used to to produce the ILCs supposedly involved a series of different cell culture steps that were supposed to mimic the progressive differentiation stages during development of the pancreas in humans (described in the journal Stem Cells in May 2007, see HERE for the journal citation). Other pancreatic cell types resembling those of the exocrine pancreas were also observed during the differentiation process. Also of note is the fact that the protocol supposedly does not utilize serum or feeder cells of any kind. Many others have used feeder cells from mice or other animals, raising questions as to whether cells derived from those processes would cross the line into xenotransplantation, and how regulators might view them as a result.

Geron did NOT receive U.S. taxpayer dollars for its work (nor did it rely upon funding from nonprofit organizations like the JDRF), but the company did receive funding from the State of California for its stem-cell efforts. In May, the California Institute for Regenerative Medicine announced a $25 million loan to Geron to support the spinal-cord injury trial. However, on Monday, Geron reported that the company had repaid the $6.4 million it received for the loan, along with with accrued interest. My readers may recall that the California Institute for Regenerative Medicine was started, in large part, by families with type 1 diabetes. This was discussed in a chat I hosted with the former Vice Chair of the U.S. NIH Stem Cell Task Force Dr. James F. Battey, MD. You may catch the transcript HERE.

Getting back to the topic du jour, many Californians were fed up with the promising field of regenerative medicine research being held hostage by national politics, hence they helped push for the State of California to fund it since the research was being stymied at that time by the Federal government and President George W. Bush's decision to limit Federal funding for the research to a handful of stem cell lines developed by the arbitrary date of his announcement. Although President Obama has since lifted those restrictions, it remains controversial, but California can do what it wishes regardless of who's in the White House (and this was funded specifically by a voted-sponsored proposition hence it's not subject to state lawmakers' whims ... yet). Because of the controversy, it's likely to be funded inconsistently subject to the personal views of lawmakers, which does little to advance the field.

There remains some skepticism among many researchers on the credibility of Geron's lofty claims, in part, because the methods used by the company have not (to my knowledge) been widely replicated by other scientists. The reason for focusing on oncology was that the company concluded that its cancer treatments were the most likely to generate cash soon, and management also expressed concern that capital for its stem cell research would be harder to attain today, so the company is laying off 38% of its relatively small staff to conserve the capital it already has. The Wall Street Journal reported (see http://on.wsj.com/tCmef9) that Geron's decision to exit embryonic stem cell research was due more to financial constraints rather than any scientific setbacks in the emerging, yet still controversial research field.

Roth Capital Partners analysts wrote that it's difficult to predict whether Geron will be successful in finding a partner (or buyer) for the stem cell assets. However, the Wall Street Journal speculated that one potential corporate buyer might be drug giant Pfizer Inc., which in 2008 created a Regenerative Medicine research unit in Cambridge, UK, and earlier this year started a clinical study of a stem-cell therapy for ulcerative colitis (another autoimmune disease). A Pfizer spokeswoman was not available to comment for the Wall Street Journal.

In the Phase I clinical trial which Geron struggled to gain FDA approval for (but was ultimately successful in attaining last summer, see HERE for more), which was for nervous system cells derived from embryonic cells, the cells were being injected into people with severe spinal cord injuries. So far, just four patients have been treated with these stem cells. However, Geron's CEO Dr. John A. Scarlett said there were "no signs" that the treatment was yet helping patients, but he added that was not expected in the early-phase initial trial, which mainly focuses on safety. So far, he said there had been no sign of any safety problems.

Dr. Scarlett also said in the interview with the aforementioned New York Times reporter: "I deeply believe in the promise of stem cells. I don't think that promise [of stem cell therapies] is in any way, shape or form changed by what we're doing" adding that Geron was not divesting itself of the stem cell business because of trial results. Dr. Scarlett, who took over as chief executive less than 2 months ago, said with money scarce, the company decided to focus on its experimental cancer therapies, which are further along in development, adding that Geron needed to conserve resources at a time when it was difficult for small, unprofitable life science firms to raise capital. Geron currently has no products on the market, and would have spent $25 million per year to continue its stem cell program.

There are still a few companies pursuing this research. Another one I've mentioned before is also a California-based company in San Diego known as ViaCyte (formerly known as Novocell). But the soonest ViaCyte's technology would begin human trials would be 2013, which is optimistic.

Some respected diabetes researchers helpedput the status of this field of regenerative medical research into perspective:

Dr. Camillo Ricordi, an islet cell transplantation expert at the University of Miami Diabetes Research Institute told the Los Angeles Times "There was too much hype for this type of technology. There are no shortcuts in this kind of research." But he added "Next century, when you look back at it, two decades won't seem like much. But for those affected right now, every month is too long."

Dr. Gordon C. Weir, a diabetes researcher at the Joslin Diabetes Center in Boston seems to share both the researcher and patient perspective, saying "It's maddeningly simple as a concept. It's been incredibly frustrating that we can't bring this to the clinic more quickly."

Even though skepticism about Geron's technology remains, there also appears to be an opportunity for nonprofit organizations such as the Juvenile Diabetes Research Foundation, the Diabetes Research Institute Foundation where Dr. Ricordi works, and even the American Diabetes Association to license Geron's proprietary stem cell regeneration methods, test them and possibly bring them to trials, which might be preferable to a company like Pfizer buying the assets and keeping them sealed in a vault. I reached out to each of these nonprofit organizations, but none had responded to my inquiry at the time I prepared this posting. Stay tuned for more!

Meet Snowball

My readers may recall in my last blog post, I touched on the subject of Invisible Illness Week about 2 months after that blog-themed event actually took place. I also shared my rationale for that, by writing: "limiting ourselves to just a week to acknowledge invisible illnesses does a bit of a disservice to the concept, so I theorized 'why not address invisible illnesses some other time?!' (I have the same feeling about No D-Day, which I hope to cover at another time). With that, I decided I would do it when time enabled me to do it, not according to someone else's calendar."

Hence, today's posting: No-D Blog Day 2011 (see HERE for some additional information).

This is one of the few themed blog days I actually look forward to. In fact, a few years back, George Simmons didn't do one and I was one of the individuals who asked him why he didn't do it, and to the best of my knowledge, that themed day has since been continued annually ever since. But I was actually really busy with my aunt and uncle in from Seattle (and I don't get to see them very often), so I didn't get to writing about it on October 7, 2011 -- the "official" No D-Day. It doesn't matter -- in fact, you might actually remember MY posting for No D-Day vs. the something like 40 other No D-Day posts from October 7, 2011! (See, there's a method to my madness!)

Back in January, I wrote about the loss of 17 year-old cat Phyllis (see my post HERE). I really agonized over the decision to have her euthenized, but in hindisght, I know that I chose to put my longtime pet's well-being first over of my own selfish desires to keep her around for just a little while longer while she would have suffered in agony. I don't regret that decision, but it still makes me very sad.

I wasn't in big a rush to get a new cat (or kitten). I had a fairly busy travel schedule ahead of me, and I was still mourning the loss of a longtime pet, so I decided to take a break and maybe look at adopting new pet at a later point in time. I actually waited over 6 months before I even considered getting a new pet.

I really wanted to go the route of adopting a kitten, in part, because they can be trained more easily than adult pets who tend to be much more stubborn (to the extent that any cat can really be trained). I visited a few animal rescue centers including the well-known North Shore Animal League which is based in the town I lived in when I first moved to New York (so I KNEW where it was located) and is billed as the world's largest no-kill animal rescue and adoption organization. I also visited a few other animal rescue and adoption centers in the NYC area. When I visited those, it became painfully evident to me that kittens (and puppies) have little if any problem getting adopted, but far too many adult animals are often overlooked, and sit in cages for months at a time waiting for a new owners. There were a few that had no problem, but there were also some mother cats whose kittens had been adopted while the poor mothers remained in their cages, sometimes for many months.

While that's all very touching and emotional, I wasn't going to take just any fleabagfeline with a sob story home with me, especially if I have to live with this animal for many years to come. The animal had to be alluring, yet appear to have some kind of colorful personality behind a cute face. Shortly before Labor Day, I visited a local adoption center for another local animal rescue organization and saw a little girl cat known as Snowball. She has very stunning blue eyes (definitely a Siamese mix) with a beautiful, tan, domestic shorthair coat. Snowball was definitely an adult at around 4 years old, but seemed to have a compelling personality.

The weekend before Labor Day, the animal adoption folks interviewed me (they interview adoptive families to make sure the new owners aren't living in squalor), and brought this new cat home to live with me. I learned that Snowball's owner had passed away, leaving her homeless. Fortunately, she was rescued (or taken to a rescue facility) that does not destroy animals, but learned she had been in the adoption center for a while. As I said, kittens and puppies have little problem finding new homes, but adult animals struggle.

Since bringing Snowball home, she has taken some time to get assimilated, and although she's not a lap-cat, she does like hanging out with me and can sometimes get underfoot. She also has some really nice personality traits. Most notably, I bought a special cat bed for her (my former cat couldn't be bothered with those; she slept on my bed, and I was fortunate that she permitted me to do the same!), and she actually uses it regularly. I put it on a chair that's even with the mattress at the foot of the bed, and she seems to like the fact that she can sleep there without me moving my feet and waking her up.

She has also proven to be a very good verminator. Since moving in, she has caught two mice which I didn't even know had moved in with me! Both were baby mice and were really tiny, but Snowball's rodenticidal skills are proving to be a great asset! She is also pretty quiet, although she has definite Siamese vocal skills when she needs them. Each evening, when I get home from work, I hear Snowball at the door excited and greeting me with her voice, apparently excited that I'm home. I don't know how she knows it's me, and not someone else in the building, but apparently, she does! This cat seems to have hidden talents! Meet my new cat Snowball!

A Parallel Autoimmune Online Community

Back in early September (September 12-19, 2011), it was national "Invisible Illness Week". The theme for 2011 was "Deep Breath, Start Fresh" and the basic idea behind the theme was that sometimes, it's very easy to get caught up in the day-to-day roller coaster of emotions, doctor's appointments and the like, so the organizers hoped to use the week to help remind everyone with a chronic, invisible illness to take care of themselves, and possibly share some healthy ways of actually doing that … but also to try and add some more joy (and even sunshine, hence the logo) into life! Truth be told, I joined Invisible Illness Week late anyway, so I did not really do anything with it at the time, but my feeling is that limiting ourselves to just a week to acknowledge invisible illnesses does a bit of a disservice to the concept, so I theorized "why not address invisible illnesses some other time?!" (I have the same feeling about No D-Day, which I hope to cover at another time). With that, I decided I would do it when time enabled me to do it, not according to someone else's calendar.

So I did not officially "participate" this year as some of my fellow diabetes blogging counterparts did, but shortly before Invisible Illness Week, I did something else. I began following a few individuals with various OTHER autoimmune diseases besides type 1 diabetes. This has been an area of interest for me for a while, and that was how I decided to acknowledge Invisible Illness Week in 2011.

New research suggests that there may be a kind of 'master key' that may help unlock new treatments for many different autoimmune disorders (see HERE for details). Plus, most (except, perhaps psoriasis) autoimmune diseases are actually invisible illnesses, not unlike type 1 diabetes. Hence, my reason for "reaching across the autoimmunity aisle" was because sometimes those of us in the diabetes community tend to view our diseases with a bit of tunnel-vision, almost oblivious to the fact that there are some 80 other autoimmune diseases out there, and I think it's safe to say having those diseases, sucks too! However, I discovered that many of them have a lot of the same issues, complaints and concerns!

A Parallel Universe or Another Earth?

Some of this kind of reminds me of the film that won the Alfred P. Sloan Feature Film Prize (see HERE) at this year's [2011] Sundance Film Festival, specifically a film entitled "Another Earth". Another Earth was a science fantasy/drama film directed by Mike Cahill (and much of it filmed not too far from where I grew up in Connecticut) and starred William Mapother and up-and-coming star Brit Marling. Long story short: the film was about a bright young woman who is accepted into MIT's astrophysics program and aspires to explore the cosmos. Brit Marling's character goes on to discover a duplicate of the planet Earth. I'm including the trailer at http://youtu.be/N8hEwMMDtFY because it's not often that SciFi films are so interesting IMHO.



In the process of following some of the other bloggers with a host of other autoimmune diseases, I realized that there are actually a surprising number of similarities not just in what causes our diseases, but also in how we deal with the day-to-day of trying to live life with a chronic, presently incurable disease. Some of these discoveries I probably should have realized, but just didn't (to be truthful, I sometimes have difficulty understanding what each autoimmune disease is best known for because there are over 80 of them). But the fact is that symptoms of various autoimmune diseases often cross many medical specialties, and therefore affect all body organs, just as in the case of type 1 diabetes. Patients don't just have to deal with specific types of symptoms, but a host of other long-term health issues that are caused by their autoimmune diseases.

For example, were you aware that people with Sjögren's Syndrome are much more likely to suffer from peripheral neuropathy? Or that people with psoriasis are significantly more likely to suffer from cardiovascular diseases (including strokes)? It's true, although unlike diabetes, I usually don't hear people making rude comments at funerals of people with these other autoimmune diseases for their failure to manage their diseases as so many people who don't have a clue what's involved with managing a chronic condition sometimes feel justified in doing when discussing people who lived with diabetes. For that, we can blame the mass media for turning our disease into a short sound-bite. But as you might imagine, regardless of our diseases, we all bitch about insurance companies!

At the same time, most medical education provides would-be doctors with pretty basic learning about autoimmune diseases, and most physicians (including many specialists) are generally unaware of interrelationships among the different autoimmune diseases or advances in treatments outside their own specialty areas unless they have taken Continuing Medical Education (CME) credits on specific topics related to these other autoimmune diseases. In the case of type 1 diabetes, far too many specialists presume they know more than they actually do. Still, most research being conducted tends to be disease-specific and rather limited in scope. More information-sharing and crossover among research projects on all different autoimmune diseases is clearly needed.

Lurking In Other Autoimmune Disease Communities in the Social Media Space

The result of my blog (and Twitter) "lurking" on some of these autoimmune disease bloggers and communities was the realization that there's actually a number of groups that are a lot like the Diabetes Online community (or "D-OC") for people who suffer from, for example, Rheumatoid Arthritis (a.k.a. "RA", or on Twitter typically designated by the hashtag #rheum).

Beyond having created similar social circles of fellow patients who blog about life with RA, many also carry around similar baggage from know-it-all ignoramuses in society (much like those who assume that life with type 1 diabetes is exactly the same as life with type 2 diabetes) who assume that the autoimmune form of arthritis known as RA is exactly the same as osteoarthritis which is commonly associated with aging (for the record, RA is NOT the same as osteoarthritis, just as type 1 diabetes is, in at least a few notable ways, a rather different disease from type 2 diabetes).

Aside from dealing with public misperceptions of their disease, the RA community also has a similar recurring theme we've seen in the diabetes community, specifically the idea of portraying RA patients as "Superheroes" (which we ARE, after all!!).

In the Diabetes Community, catch Chris Bishop's [from the D-OC] more recent post on that topic HERE, some of his earlier iterations of the Diabetes Superheroes can still be found on the Internet Archive (my personal superhero avatar can be viewed HERE), and of course, there's the Diabetes Duo (Captain Glucose and Meter Boy):

One RA blogger has assumed a role of highlighting the RA superheroes (see HERE), although there are not the cool graphics the D-OC has seen in some iterations, but the point behind the listing is similar. What's more, the unacknowledged baggage many people with these other autoimmune diseases are forced to carry around is, in many cases, surprising similar to life with type 1 diabetes. I also discovered there are even a few Podcasts, BlogTalkRadio programs and many people with these various autoimmune diseases who also participated in Invisible Illness Week.

Imagine that ... a parallel universe of people living with other autoimmune-mediated diseases in the blogosphere, on Twitter, BlogTalkRadio, Google Plus, Facebook, podcasting, etc.!

A Need for United Efforts Across the Autoimmunity Community (gee, that rhymes!)

Although there are some umbrella organizations such as the American Autoimmune Related Diseases Association which is dedicated to the eradication of autoimmune diseases and the alleviation of suffering and the socioeconomic impact of autoimmunity through fostering and facilitating collaboration in the areas of education, public awareness, research, and patient services in an effective, ethical and efficient manner. However, IMHO, there's much work remains to be done as far as research coordination (especially with the U.S. National Institutes of Health). One of ARDA's objectives is also too address some common themes impacting ALL autoimmune diseases.

Still other organizations, such as the FAIR Foundation aren't specific to autoimmune diseases, but work for attaining fair and equitable distribution of bio-medical research funds allocated by the U.S. government for ALL diseases, a fair number of which are autoimmune diseases. For example, although the U.S. Federal government will allocate $1 billion to diabetes research in in 2012 according to Government statistics, when one examines how much is being spent for each person with diabetes, it equates to just $42 per patient, compared to an unfathomable $46,890 for each person with West Nile Virus, $4,901 for each person with some form of cancer (including $3,721 for each person with breast cancer specifically compared to $177 for each person with prostate cancer), and $3,047 for each person with HIV/AIDS. Their objective is to change the current budgeting system into one that actually takes into consideration the incidence, mortality, suffering, morbidity, communicability and preventability of each disease, and secondarily, to help eliminate America's organ-donor crisis. (see HERE for references on these stats)

Although a few diabetes non-profit organizations, such as the Juvenile Diabetes Research Foundation (JDRF), have in recent years begun collaborating with research organizations associated with other autoimmune diseases on mutual areas of interest on the autoimmunity front, including the JDRF's partnership with Fast Forward, LLC (which is a wholly-owned subsidiary of the National Multiple Sclerosis Society) in a collaborative partnership with an Italian company known as Axxam SpA to develop new treatments for both autoimmune diseases (multiple sclerosis and type 1 diabetes, see the press release HERE), most of these collaborations are still pretty recent. Because most of these collaborative efforts are still quite new, few have yielded much in the way of progress so far, although these could yield some very important results in the future. Such partnerships are, IMHO, long overdue. Ideally, one might presume that the U.S. National Institutes of Health (NIH) would be coordinating research efforts across diseases with very similar etiologies, but as I hope to demonstrate in the next section, that doesn't seem to be the case.

NIH Autoimmune Disease Coordinating Committee's Absent Participant: NIDDK

To facilitate collaboration among institutes of the NIH, other Federal agencies, and private organizations with an interest in autoimmune diseases, both the U.S. House and Senate Appropriations Committee reports in June 1998 encouraged the establishment of an NIH Autoimmune Disease Coordinating Committee under the direction of the National Institute of Allergy and Infectious Diseases (NIAID).

Ironically, you might be surprised to learn that the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) responsible for diabetes research has committed few dedicated resources to the autoimmune disease coordinating committee except when it's been required to do so by law. There is NOT a dedicated NIDDK representative on this committee. We can probably thank the Special Statutory Funding Program for Type 1 Diabetes Research which dedicates substantial taxpayer dollars to research on the topic of autoimmunity for almost all of the NIDDK's involvement to date.

The Special Statutory Funding Program is a very unique appropriation that the NIDDK administers on behalf of the Secretary of the Department of Health and Human Services, in collaboration with multiple NIH Institutes and Centers and the CDC for research on the prevention and cure of type 1 diabetes in the amount of $1.89 billion for Fiscal Year (FY) 1998 through FY 2013. However, beyond that appropriation, there is now some evidence to suggest that type 2 diabetes may actually have an autoimmune basis (see HERE).

New Data Also Suggests Autoimmunity in Type 2, Albeit With Different Immune Response Targets

The immune response in type 2 diabetes does not specifically target insulin or the islet cells as is the case with type 1 diabetes. In type 1 diabetes, autoreactive T cells targets insulin and other normal, "self" beta cell proteins as if they belonged to dangerous microbes, whereas in type 2 diabetes, the immune response involves two different types of immune system cells, specifically T cells and B cells in addition to macrophages. In other words, individuals with insulin resistance would NOT test positively for ICA's (or islet cell antibodies) — one of several different lab tests that may be done to confirm an appropriate diagnosis of autoimmune-mediated type 1 diabetes, but the research does suggest that people with insulin resistance actually DO make antibodies to a select group of their own proteins which sets off a cascade of metabolic abnormalities.

This logically suggests that type 2 diabetes may also have an autoimmune basis as well (see the April 17, 2011 scientific/medical journal Nature Medicine for more background on that). Hence, although interventions and/or treatments to induce self-tolerance might be different for type 1 and type 2 diabetes, because both diseases have an inherent interest in autoimmunity, it's logical to presume that the NIDDK would have a permanent member with some leadership role on the Autoimmune Diseases Coordinating Committee.

That presumption would be mistaken.

The closest representation we have was revealed in the National Institute of Allergy and Infectious Diseases' (NIAID) Autoimmune Disease Coordinating Committee's 2005 report to Congress (see HERE), yet in the staff bio (see HERE) for Dr. Stephen P. James, MD who is cited as the NIDDK's committee participant, there is no mention of the Autoimmune Disease Coordinating Committee as part of his "Current Portfolio/Responsibilities" or under "Committees/Working Groups" he works in, or in "Other Activities" listed among his duties. This suggests he is NOT a permanent NIDDK participant on this important committee. Yet he was rightly chosen because he has the most experience within the NIDDK in issues pertaining to autoimmunity.

My response was: WTF is going on here?

In other words, the NIDDK does NOT seem to have permanent membership on this seemingly important committee, especially since the NIDDK has interest in autoimmunity for BOTH type 1 AND type 2 diabetes, but NIDDK management has literally had to be forced (by law) into participating.

NIDDK & Autoimmunity Coordination: Only When the Law Requires Our Participation

Federal records indicate that although the NIDDK is obliged to have some representative on the Cooperative Study Group for Autoimmune Disease Prevention (CSGADP) efforts and a handful of other programs and committees, each meeting's attendance from the NIDDK has been a completely different individual. Even more WTF? from my perspective!

In fact, it appears that because the NIDDK is required by the Special Statutory Funding Program for Type 1 Diabetes to have representation on the Cooperative Study Group for CSGADP within the NIH that may very well be the ONLY reason the NIDDK even bothered to send someone to these meetings. The NIH autoimmune disease prevention effort was established in 2001 as a collaborative network of investigators with a focus on prevention of autoimmune disease, defined as halting the development of autoimmune disease prior to clinical onset by means other than global immunosuppression, and a particular emphasis on type 1 diabetes.

Indeed, I think it's safe to say that it’s only because of the Special Statutory Funding Program, which enables the creation of unique and collaborative research consortia and clinical trials networks focused on the prevention, treatment, and cure of type 1 diabetes specifically, that NIDDK even participates.

Why?

The bigger question we should be asking is whether the NIDDK the leadership considers autoimmune research something that is even core to the institute's mission?! I blame the current leadership for this lack of involvement. Here again, we see that the person running the NIDDK is a long-term bureaucrat (albeit a person who is a medical doctor), Dr. Griffin P. Rodgers. One might presume that the man running the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) to be, oh I don't know, maybe a person who's an endocrinologist, a gastroenterologist or perhaps a nephrologist?

No!

Dr. Rodgers is, by training and Board Certification, a hematologist (in other words, he's a blood doctor). The previous Director of the NIDDK, Dr. Allen M. Spiegel, was logically a Board-certified endocrinologist. While all medical doctors have a basic understanding of the various body functions and disease, is it really that difficult to find someone with training in diabetes, digestion or the kidneys to run the organization known as the National Institute of Diabetes and Digestive and Kidney Diseases?

Apparently so.

Although the layers of government bureaucracy are deep, Dr. Rodgers is in my opinion, not the most appropriate selection to run one of the world's largest funders of diabetes and kidney research since his knowledge is really not much deeper than that of many family doctors. This is an appropriate question to be asking your lawmakers in Washington the next time you reach out to them for some other advocacy issues pertaining to diabetes.

While I would welcome comments on this topic, getting back to my parallel universe theme, I plan to reach out to some of the other autoimmune disease bloggers I've connected with since Invisible Illness Week because their stories are actually very interesting, and there may be opportunities for the Diabetes Online Community to collaborate with them on different areas of mutual interest. I hope you will return for my next posting I do on this topic!

Puncture (The Movie) Starring Chris Evans

Over the years, there have been a handful of groundbreaking movies that address a variety of topics of interest to people within the diabetes community. For example, there was "Supersize Me" (2004) and "Fast Food Nation" (2006) which addressed how obesity is frequently driven by the prevalence of fast food. However, many people never patronize fast food joints, although these films did help pave the way for more critical exposes of the nation's industrial food supply system and its adverse effects on the nation's health. King Corn (2007) addressed issues with America's most overly-subsidized food crop and the adverse effects it has on such unrelated areas like beef production today, or Food, Inc. (2009) which shed light onto our less-than-pretty industrial food supply system, and of course, Michael Moore's SiCKO addressed the fact that not only does the U.S. have the world's most costly healthcare system, but we also have the highest percentage of individuals left completely out of this system thanks to runaway costs and non-universal insurance coverage (although the Patient Protection and Affordable Care Act should help to address that major hole starting in 2014).

On September 23, 2011, another movie called "Puncture" opened in select cities, and is expected to open on a national basis this weekend. As Metro NYC described it: "At first blush, the words 'health care industry' and 'thriller' seem like two terms that should never be in the same sentence together." In essence, the movie is loosely based on the true story of two struggling young lawyers, Michael Weiss and Paul Danziger, who were hired by an eccentric inventor/manufacturer to find out why he couldn't sell his remarkable, lifesaving safety syringe to U.S. hospitals. The real case was actually settled before trial for $150 million dollars.

It began when a nurse who, while dying of AIDS due to an accidental needle stick went to two lawyers proposing a lawsuit. The accident could have been avoided if the hospital carried safer, single-use needles with protective sheaths. But hospitals refused to carry the device because of their long-standing relationships with big medical device suppliers brokered by so-called group purchasing organizations (GPOs).

In the process, the film reveals that in order to sell to many big hospitals, startups have to effectively bribe GPOs with financial kickbacks to hospital bigwigs which enables the existing suppliers to basically keep other suppliers out of the hospital supply chain altogether. In fact, the inventor of the new device was barred from even showing it to hospital purchasing agents. The lawyers discovered a very corrupt arrangement between monolithic hospital purchasing cartels and a big needle maker, in which the industry giant was able to pay millions in kickbacks to the cartels to make sure its less safe products — and only its products — were used in hospitals. They also found that these cartels, known as hospital GPOs, effectively blocked the introduction of all kinds of innovative medical products, not just syringes, costing the U.S. healthcare system over $37 billion each year. For the record, Metro NYC also reports that GPOs are now a $100 billion/year business in the U.S. today.

I have not seen the film personally, but with a cast including "Captain America" hottie Chris Evans who plays character Michael Weiss (who's character is actually a drug addict) and Mark Kassen who plays Paul Danziger, this film might be worth seeing, and on a broader level, revealing how much business is really transacted in the U.S. today, painting a slightly different picture than politicians would have us believe, and raising new questions about just why healthcare in the U.S. remains so incredibly expensive.

Check out the movie trailer at http://youtu.be/9wLQCOqzLv4:

The Business of Diabetes: Biodel Linjeta Update

It's been some time since I posted a "Business of Diabetes" update, so today's post is probably overdue. Today's subject is particularly timely since Dr. Errol De Souza, President and CEO of Biodel, Inc., presented this morning (see the presentation deck HERE) at 8:30 AM EST at the 6th Annual JMP Securities Healthcare Conference at the St. Regis Hotel in New York. My experience has proven that a fair number of presentations made at similar investor "events" like these can quite repetitive (for example, if a company presented a month earlier for another investment bank, whether its Morgan Stanley, Goldman Sachs, JPMorgan, Sanford Bernstein, Credit Suisse, Jeffries or any of the others), many presenters are already very well-rehearsed and prepared for questions. This means that often, they may simply re-present the same content, making the Q&A that follows their actual presentations the main point of differentiation (sometimes you can listen to these online, but not always). Still, I always try to review the content from these events when time permits because there may be occasional nuggets of interesting information that are shared. However, we can expect more detail from rivals including Halozyme Therapeutics made tomorrow at the same event.

Truth be told, I already reviewed this morning's JMP Securities Healthcare Conference presentation, and there really wasn't much in the way of new news that wasn't already in the public domain, but my readers may not be aware of where things stand, so if you're interested, you can view the webcast for about the next month by visiting Biodel's investor relations site HERE. I will address the most salient points that people with diabetes may want to know about in this post.

Taking a quick step back, my readers may recall my POST from early 2010 in which I addressed a number of startups eager to stake a claim in the multi-billion dollar insulin market (notably, rapid-acting "prandial" insulins is now approaching $5 billion per year, so there's VERY big money involved). A fair number of the startups I wrote about experienced delays (all for different reasons), one of which was Biodel, Inc.'s Linjeta™ [formerly known as VIAject] which is the subject of today's post and was the first to seek regulatory approval. The other was MannKind's Afrezza® which I will not really address in today's post. Both experienced some delays with the agency many (most?) of us love to hate (although a few of us dislike them for entirely different reasons!): the U.S. FatalFood and Drug Administration.


However, in both of the aforementioned cases, I think it's safe to say that the delays were pretty much the fault of the companies that were seeking product approvals (see HERE for my update on Linjeta). When former FDA Chief Andrew von Eschenbach ran the agency, I don't believe either would have been delayed, but his leadership (thankfully) is history now. Under Andrew von Eschenbach's leadership, the FDA's staff was instructed that the FDA should not be viewed as an agency to protect the public health per se, but an agency to serve the needs of the industries the agency regulated (especially since most of the funding for the FDA now comes from user fees paid for by the drug, biotech and medical device industries except in the case of generics, which are incredibly backlogged) and that the FDA should be working to address the needs of these "clients". While that was good for pharma, and user fees did fund a lot of FDA staffing, the biggest beneficiaries of those fees were pharmaceutical/biotech/med device companies, although few lawmakers seem to agree this means the FDA is on pharma's payroll even though they pretty much are. Fortunately, under the leadership of Margaret Hamburg, the agency is returning to its mission of actually trying to protect the public health. While many complain the FDA has moved too far on the side of caution, especially in comparison to other regulatory agencies around the world such as the European Medicines Agency, I'll reserve judgement on that when I have the benefit of hindsight! Let's just be glad we don't have evidence of recent approvals for truly dangerous drugs like Vioxx or Avandia (so far, anyway) that were approved while Dr. von Eshenbach was still on the FDA's payroll.

Still, more than a year has passed since I wrote my original post on startups in the insulin field, and I think its time for an update on at least one of them since details are finally in the public domain. So today, I'll cover the product that applied for FDA approval first: Biodel's rapid-acting Linjeta insulin formulation.

Where Things Stand Today

On Tuesday, August 16, 2011, the insulin startup Biodel, Inc. presented (see HERE for the presentation) at the Wedbush Securities' 2011 Life Sciences Conference in New York. Although I did not attend, the presentations themselves usually appear on each of the presenters' investor relations department (at least for those companies that are publicly-held) shortly after these events take place, so I try to read through these for clues on where things stand. Typically, more is revealed there than will EVER come from the FDA itself (another practice which I'd like to see change to look a bit more like Europe, but I digress ...).

My experience has proven that a fair number of presentations made at similar investor "events" like these can quite repetitive (for example, if a company presented a month earlier for another investment bank, whether its Morgan Stanley, Goldman Sachs, JPMorgan, Sanford Bernstein, Credit Suisse, Jeffries or any of the others), many presenters are already very well-rehearsed and prepared for questions. This means that often, they may simply re-present the same exact content, making the Q&A that follows their actual presentations the main point of differentiation (sometimes you can listen to these online, but not always). Still, I always try to review the content from these events when time permits because there may be occasional nuggets of interesting information that are shared. I actually found quite a bit of information about Biodel, Inc., so today, I'm sharing a few of those take-aways as an update.

For Biodel, since the FDA move to delay its decision on Linjeta™ earlier this year, the news has been quiet as the company addresses the FDA concerns and prepares for worldwide commercialization. I should note that Biodel's rapid-acting insulin formulation, unlike ALL other competitors on the market today, is NOT a genetically-modifed, man-invented insulin-like molecule that is commonly referred to as an insulin analogue, but regular biosynthetic human insulin which has been made faster than today's analogues thanks to some proprietary technologies (in theory, the same technology could also be applied to old-fashioned highly-purified insulin formulations sourced from abbatoir animals rather than using agars derived from abbatoir animals used in making biosynthetic human insulin and its analogues, thereby making them faster than today's analogues if the company ever chose to offer such a thing -- unlikely, but the point is that the technology makes even old-fashioned insulin varieties work a LOT faster).

Of course, Linjeta is reportedly only slightly faster (we're talking a few minutes faster here: Linjeta starts to work in about 8-18 minutes (8-13 minutes for the formulation used in the company's new drug application with the FDA) compared to about 25 minutes for most of today's rapid-acting analogues, and about 45 minutes to an hour for regular insulin. Whether that's enough to convince insurance companies to pay for it remains to be seen, but Linjeta differs because the way it accomplishes this is not be altering the insulin molecule itself via genetically modification, although as Close Concerns once said, "in this business, time is money". I have long suspected the company viewed Linjeta as an interim product that could be marketed very soon, but sees the real opportunity to get insurance coverage for their product by making today's analogues slightly faster using their technology, which is patent-protected making Biodel itself potential acquisition bait for a big pharma company like Lilly, Novo or Sanofi. At least one analyst has already gone on record as saying Sanofi is a likely buyer because even though its Apidra rapid-acting analogue still enjoys several more years of patent protection relative to Humalog and Novolog, what insurance company will pay a premium for it when generic versions of the other product are expected to become widely available? This means aquiring Biodel may protect its products (and give them some technology for Lantus as well ... see HERE. The other firm he discusses is Halozyme, but that company is slightly behind Biodel from a development standpoint. He has a valid point.

About Biodel and Linjeta™: How It Works Differently From Today's Analogues

Biodel makes regular insulin faster than today's analogues by removing zinc ions via EDTA, which destabilizes the hexamer of insulin; then by adding citric acid (you know, the stuff found in many powdered lemonade formulations which makes them taste "tart") which also happens to masks surface charges on insulin monomers, which in term prevents re-aggregation and facilitates more rapid absorption into the bloodstream (wow, that's a run-on sentence, but I make no apology for it!). EDTA stands for ethylenediaminetetraacetic acid (which is a real mouthful, hence the acronym is more widely used), and its a compound of amino carboxylic acids that's water-soluble. It is used in various drug and biotech medicine manufacturing to bind to metal ions in order to remove these metal ions from various drug formulations which contain them -- I guess one could liken this to a magnetic substance that removes the traces of zinc from the the insulin protein (note: its not really magnetic, but I thought the analogy made sense in this case).

Note that zinc has long been associated with insulin. In fact, before the days of geneticially-modified insulin analogue formulations such as Lantus and Levemir, zinc was widely used as an additive to delay regular insulin's action in Lente (meaning Lente and Ultralente) formulations, hence Lente is known generically as "insulin zinc suspsension", but even in non-Lente formulations (particularly in regular insulin, as well as in virtually all analogues), tiny zinc ions remain. These ions are one reason it takes regular insulin so long to be absorbed into the bloodstream.

Technology issues aside, the company (Biodel, Inc.) is now completing a small, additional study in order to resubmit Linjeta for FDA approval. I would describe as follows: to compensate for their botched human clinical trial in India which was supposed to save the company lots of money, but the actual cost savings were nothing more than a mirage, and they would probably would have been better off just doing the entire clinical trials in the U.S., Canada and Europe, but I digress. Of course, this company (as well as the established players) bitch and moan that using regular insulin means it's tough to recruit participants in their rapid-acting insulin trials and are trying to convince the FDA that future trials should be against rapid-acting insulin analogues like Humalog/Novolog/Apidra. I don't agree about that is appropriate, but they are lobbying hard to make that the case, and in Biodel's case, for the newer, fix-up version they're now trialling, they've already proved Linjeta is comparable to regular with the first trial, hence the fix-up trial is a quickie that can be done against Humalog instead of regular because they've already established it's comparable to regular, hence fix-up trial was able to recruit faster against Humalog. Slippery slope? You be the judge on that.

While that's gone on, the company also hired a new CEO named Errol De Souza who I noted above was giving this morning's JMP Securities Healthcare Conference presenation (Solomon "Sol" Steiner who was the CEO since the company's inception in 2003 was Chief Scientific Officer for a period, but he retied late last year and is still on their scientific advisory board ... FYI, Dr. Steiner previously worked for another startup company that was once known as MiniMed, Inc. if that tells you anything, and Dr. Steiner also remains on Biodel's Board of Directors). In addition to Biodel's lead product candidate Linjeta, they also have not one but TWO long-acting insulin formulations, one being an extended version of Lantus, and the other is perhaps more compelling (in my opinion) since it enables adjustment of just how long the basal insulin product will actually work, because not all patients need or want 24 hours of basal coverage, nor do they want a pump device attached to them at all times (or they simply cannot afford such a device). Although Novo Nordisk's Levemir is a competent 8-10 hour insulin formulation, most doctors view it as less appropriate for many patients with diabetes than Sanofi's blockbuster Lantus unless the patient needs less than the typical 18 hours of basal coverage offered by Lantus (few believe either lasts 24 hours as both are advertised to) even though the basal needs vary widely from patient-to-patient.

The primary patent for Lantus expired in 2010, although various other patents will remain in place until 2014 (which is right around the corner in pharma-speak), plus Biodel also has a "stablilized" glucagon analogue now in development (one which would not need to be reconstituted prior to use). While it might not be available soon enough to compete with Enject's GlucaPen [http://www.enject.com/] for hypoglycemic emergencies (that's not yet approved, either, but since it is a device for delivery of an already-approved drug, the FDA review and approval process is likely to be faster ... knock on wood!), and if it gains approval, it could be used for that as well as in a bi-hormonal pump which have been studied in a few clinical trials and have provided patients with superior glycemic management and added security).

So where do things stand with Linjeta Today?

1) Tweak the Formula of Linjeta to Ensure Injection Site Discomfort Matches Competitors

First, Biodel is making some slight changes to Linjeta which company management believes will help it's finished product sell better. To quote this morning's presentation: "It is prudent to incur the marginal cost and time delay to move forward with a new formulation(s) to optimize the product's label." Specifically, a fair number of patients in the clinical trials for Linjeta complained of an annoying "stinging" or "burning" sensation following an injection of Linjeta, and that incidence was higher than with existing insulin analogues. While the stinging was not sufficient to deter continued use of Linjeta in the trial, the company believes it is nevertheless prudent to incur the cost and time delay the ultimate introduction slightly in order to move forward with a slightly modified new formulation(s).

Specifically, the pH of Linjeta used in clinical trials was around 4; most other insulin analogues are closer to zero, so the new formulations will have a "neutral" pH, which the company has data showing will reduce injection site discomfort. What's behind the injection site discomfort? The company believes it is caused by the EDTA used to remove the zinc ions, so the company is now undergoing trials with either calcium disodium EDTA or disodium EDTA + calcium, which it believes reduces injection site discomfort, as both had significantly reduced injection site discomfort in other studies.

In other words, the company wants the launch version of Linjeta they plan to market to be fully comparable to the alternatives as far as injection site discomfort. These changes have delayed the finished product from going to market slightly, but are likely in the best interests of becoming a true competitor in the field. Also, the delay is marginal, because it can be class 2 resubmission, which the company expects will result in a 6-month review time, and is already being reviewed by the FDA now.

2) Trials To Position Linjeta Against Humalog and In Insulin Pumps

Beyond that, Biodel has also undergone some additional clinical trials against Humalog (insulin lispro rDNA origin) and also in insulin pumps, so the product will be ready to market in all scenarios current competiton can. In other words, if trials against Humalog work as anticipated, then Linjeta can advertise that it works better than a key analogue competitor, and the pump trials will enable Biodel salespeople to promote its use in pumps (rather than an off-label use as many analogues were promoted initially; in recent years the FDA has cracked down on off-label promotions for drugs). Neither is a big deal, since doctors can prescribe most drugs for uses that are not specifically approved, but having these studies will enable Biodel salespeople to promote this insulin in a manner that is consistent with this insulin's label. Personally, I don't see this one as really necessary because every endocrinologist I've ever seen prescribed insulin analogues "off-label" (meaning before they had approval for use in insulin pumps), and I would not expect anything different with Linjeta, but they're doing the trials anyway, which will enable the products to be marketed -- legally -- for use in insulin pumps.

Both of these items should position Linjeta to be promoted from almost the minute it is approved head-to-head with its key competitors. However, a word of caution for anyone who is thinking of investing in Biodel: note that while these moves are smart business moves, the party may not last long, because the new healthcare law mandates that the FDA outline a way for "follow-on" versions of biotech medicines including insulin analogues. As Medco's CFO (although an offer to acquire Medco was recently made by rival Express Scrips) believes we will see generic versions of Humalog, Novolog, and Lantus by 2015, see my post on that HERE for details), and it's been written into a law that's been on the books for over a year now and the FDA is due to release guidelines for follow-ons anytime according to the press. This means we may FINALLY see follow-on versions of various insulin formulations in the next few years, which I see as a big threat to the oligopoly Lilly-Novo-Sanofi have enjoyed for the last 30 years. Although I still have concerns about follow-ons (or "biosimilars"), my belief is that the world will ultimately be a better place when the choices for patients are expanded ... provided the choices are made by patients and their doctors, not penny-pinching insurance companies.

Challenges Ahead

Biodel is on record (in one of the company's SEC filings, though I don't recall which one) in noting to investors that "since Biodel started the Linjeta program in 2005, Sanofi, Novo Nordisk and Halozyme have all announced programs or active in-licensing efforts in the ultra-rapid-acting insulin space" (although truth be told, Halozyme has been planning to enter this space via partnerships for quite a while). Nevertheless, it may be a challenge to gain traction in this market with insurance companies growing increasingly penny-pinching, but company management appears cognizant of what they're up against. Some analysts believe that many biotech startups in this space would likely be acquisition bait for companies like Sanofi Aventis, which faces patient expirations on its key insulin products Lantus and Apidra but lacks much to protect this franchise thanks to their own lack of investment in it (I already noted this, but again, you may refer to that article HERE which positions these both Biodel and Halozyme favorably to be acquired by the company. That remains to be seen, but these companies are eager to have a slice of the growing worldwide insulin market.

However, in today's presentation, just as I suspected, the very same technology used in Linjeta is also planned to expedite today's rapid-acting analogues to make them slightly faster -- most likely when the patents expire beginning next year. However, we shouldn't expect this to deliver significant speed increases (like those we see with regular) given that these molecules are already genetically-modified to prevent the insulin molecules from forming hexamers which delay absorption because the hexamer must equilibrate back into monomer form in order to bind to the insulin receptors. Most likely, this will enable the company to gain insurance coverage which could be a more difficult sell for today's version of Linjeta, although there is no doubt the company will work to make that happen, too. After all, it will be a few more years before they can market more rapid-acting analogues, and the company will need to start generating revenues if investors have anything to say about it.

Bottom Line: (sorry, this one isn't a link) The steps needed to gain regulatory approval are already well underway, although the commercial launch of Linjeta may be delayed slightly to make the finished product more marketable. Biodel expects be able to sell a slightly less "painful" version of Linjeta upon approval, and also expects to have approval to market this insulin for use in insulin pumps from the get-go. New management is very experienced in working with the FDA and has revealed that the FDA does not expect further delays once the fix-up trial results are ready. Biodel management also seems quite confident in their strategy, and has secured additional funding to keep things moving. What's more, because the company has a considerable scientific staff in Germany, we're likely to see it launch pretty much concurrently in both the U.S. and Europe barring any unforseen (and nondisclosed) issues. If I had to guess, I would say we could see an FDA New Drug Application (NDA) decision by early 2012, and we can anticipate they will be ready to start marketing Linjeta very shortly thereafter (remember, the company outsources the actual manufacture of rDNA insulin to Merck's Organon unit, and a few other subcontractors including Albany Molecular Research/Hyaluron who manufactures the vials they will use for Linjeta and and Wockhardt Ltd. who makes the insulin pen they plan to sell [after a few unnamed modifications Biodel made to the product design which I am guessing is to enable 1/2 unit dosages) and these suppliers are already being reviewed for approval by the FDA as I write this.