Tuesday, April 27, 2010

Wednesday Three: News Worthy of Sharing

A few interesting news items, along with my unbiased opinions, as my reader's have come to expect.

LCT Gets A $500,000 Grant From the Juvenile Diabetes Research Foundation (JDRF)

First, of particular note is the fact that Living Cell Technologies (better known by it's acronym LCT) announced today that it has received a grant of US $500,000 for its ongoing Phase II clinical trial of DIABECELL® in New Zealand from the Juvenile Diabetes Research Foundation International. From my perspective, I would dare say that the actual investment from JDRF may be less critical for the company from a financial perspective (but the grant is certainly welcome), because as a publicly-held company listed on the Australian Securities Exchange as well as in being listed in the U.S. (ticker symbol: "OTCQX"), the company already has access to worldwide capital markets making it easier for them to obtain financing than many privately-held firms or nonprofit organizations. More important, I think this grant effectively gives the technology the imprimatur of this important diabetes organization, which in my humble opinion may be worth far more than the actual financial investment from JDRF itself.

For those of you who aren't familiar with the company, LCT is an Australia-based company that has developed a method of encapsulating (using a material derived from seaweed) transplanted islets and enables them to function while simultaneously protecting the islets from the body's immune system which caused type 1 diabetes to occur in the first place. Islets could then be implanted without requiring ongoing immunosuppression. The company is also pursuing treatments for diseases other than type 1 diabetes using similar or related technology, including degenerative diseases of the nervous system including Parkinson's disease, Alzheimer's disease, Huntington's disease and stroke, as well as a treatment for hemophilia. The idea is that healthy islet cells would be encapsulated and transplanted into the body to produce insulin and regulate blood glucose levels in a completely physiological mannner, quite unlike any other treatment for type 1 diabetes. The capsules ensure that the cells are not recognised as foreign by the patient, so no immunosuppressant drugs will be needed.

That sounds nice, but there's a massive shortage of islets derived from human cadavor pancreata, so the company is proposing a form of xenotransplantation, using islets derived from pigs (which are guaranteed virus-free due to the source from an isolated island in New Zealand) and the undisputed similarity of porcine insulin to human insulin, which actually is MORE similar to human insulin than such widely-used "modern" insulins as insulin aspart rDNA origin (brand-name Novolog/Novorapid), insulin glulisine rDNA origin (brand-name Apidra), insulin glargine rDNA origin (brand-name Lantus), and insulin detemir rDNA origin (brand-name Levemir), and insulin secreted directly into the bloodsteam would not require any manipulation because the time-activity profile would be measured in minutes rather than hours for any subcutaneously dosed insulin.

However, until recently, xenotransplantation of any form has been all but banned, prohibiting trials from conducting human clinical trials in any developed nations, which has raised some skepticism among the Western medical establishment and regulators alike. Russia, on the other hand, allowed the trials, so the company completed it's Phase I human clinical trials in that country, and those trials were regarded as meeting the research goals, this enabling the company to proceed to Phase II human clinical trials. The Russian Phase I trials, however, followed well-established Western medicine scientific protocols, and were closely monitored by a Boston-based contract research organization. Because of this, the early phase trial was therefore instrumental in convincing Parliament in New Zealand to enable Phase II human clinical trials to take place in that country. The New Zealand Ministry of Health has authorized clinical trials in NZ and patient enrolment has already commenced, with the first implant taking place last October (October 2009).

According to Professor Bob Elliott, Medical Director and one of LCT's founders, early results from the clinical trial, which began in October 2009, showed that the cell implants eliminated hypoglycemia unawareness in one of the trial participants -- a complication that occurs in as many as 2 in 10 people with long-term type 1 diabetes, and can be fatal.

As the company addresses the Phase II trials, and heads towards the final phase III human clinical trials, many eyes are on LCT as a possible curative treatment (in spite of the American Diabetes Association's move to stop the use of the word "cure" altogether, catch their 2009 "consensus" statement on that here) as fancier regenerative and autoimmune treatments proceed as well.

Afrezza Claims to have Met Clinical Endpoints

As I wrote in January, at least two new insulin products are now pending approvals, with more likely in coming years. One of those is Mannkind's Afrezza, the rapid-acting inhalable insulin.

According to a two-year study presented at the American Association of Clinical Endocrinologists 19th Annual Meeting in April 2010 (see here), Mannkind showed that Afrezza provided glucose control similar to standard insulin therapy (along with weight loss and reduced incidence of hypoglycemia) in patients with Type 1 diabetes and poorly controlled blood sugar levels. That much was required to submit Afrezza for FDA approval, but nagging questions on lung-function persist.

Mannkind discussed pulmonary function test results (PFTs) in patients treated with Afrezza at the AACE meeting, where the company claimed that follow-up measurements were similar to PFT results observed in patients receiving standard insulin therapy (see here).

Adults with diabetes who participated in any of the four controlled clinical trials of Afrezza were invited to participate in this follow-up trial. Patients were followed for a total of up to three months with PFTs assessed at the end of the parent trial and one and three months after completion of the parent trial. Of the 649 patients in the study, 315 subjects received Afrezza and 334 subjects received usual teatment regimen without Afrezza during the parent trials.

While these results were still greeted with legitimate skepticism considering patients will be expected to use the product over a lifetime, the company is trying to present as much data as it can at this point in time, but until the company has FDA approval, skepticism among healthcare professionals remains high, and long-term safety will remain a key question because long-term usage results cannot be measured in a clinical trial, unfortunately for the company.

One thing that kind of irritates me is the fact that Mannkind refers throughout it's press releases to "antidiabetic therapy" rather than diabetes treatments. Ummmm, remember when Pfizer conveniently ignored the type 1 audience in it's marketing and just look what happened to Exubera. Well, note to Mannkind and Alfred Mann: stop calling treatments for this disease "antidiabetic" -- there's no such thing, they are treatments FOR the symptoms of diabetes, but they aren't supposed to be anti-patient, and that terminology is more than a bit offensive. Watch your language, or it may come back to bit you in the @$$ -- just ask Pfizer, whose marketing folks once told me before Exubera launched that by changing the dosage amounts in "blisters" they were trying to change the diabetes treatment paradigm to be more "intuitive" and less rigid. Yeah, we all know where that ended up ...

Bayer Launches Didget: A Combination Handheld Videogame & Glucose Meter Aimed at Kids With Diabetes

Yesterday, to much fanfare, Bayer Diabetes Care announced the introduction of the DIDGET(TM) blood glucose monitoring system in the U.S. It was billed as being the only blood glucose meter that connects directly to Nintendo DS(TM) and DS Lite gaming systems to help kids manage their diabetes by rewarding them for building consistent testing habits. One feature of this thing I absolutely did NOT like was the fact that the device was also designed to reward kids for "meeting personalized blood glucose target ranges". Bayer claims the DIDGET meter is now available for purchase in the U.S. through CVS.com, Drugstore.com and Walgreens.com. There's a video here you can have a look at:

Personally, I have mixed emotions about this thing. In some ways, I really like the idea, and anything that rewards kids for establishing good testing habits is probably a good thing. But as Amy Tenderich noted, toys generally do have a more transient nature. While video games have longer lives due to the ability to continuously add new games to the mixture, kids do eventually outgrow those, too, otherwise I'd still be playing games on my Atari 2600!

My only real concern is the notion that we should somehow reward kids for "being in target" rather than simply rewarding the notion of testing regardless of the result. That's a big mistake that may cause kids to cheat or lie about results, rather than rewarding the act itself. There are no "good" or "bad" results, it's merely a tool to help guide your next actions. Instead, I think this should reward the idea of testing regardless of the result, because the information from the test can be used for valuable purposes regardless of the result.

My other gripe is the hefty sticker on this device. Bayer gains by selling more devices and test strips, so why do they need to price it at a premium? They ideally should be giving this thing away given the long-term financial benefit they stand to gain if the kids test more. But if parents of kids with diabetes like the idea, there's no reason not to offer a device like this. They might outgrow it, but be ready to replace it with Bayer's Contour USB meter (catch my post on that here) when the fascination with video games start to fade!


Jenny said...

As a mom, I find this a bit troubling. The addictive nature of video games and their negative impact on young children who spend the hours they should be giving to creative play with other children in thrall to these solipsistic devices makes me think that "rewarding" children for anything with gaming is a very bad strategy. To say nothing about the fact that gaming is a mostly male activity so the game reward gives a strange message to girls.


Nici said...

I'm glad to see that LCT is moving forward with Phase II trials.

I don't feel like their "cure" is a real one because it does not fix the root cause (the over-active immune system,) but at least it's a start.

While their solution would not solve all of my diabetes-related problems (my thyroid would still be a mess, for example,) it would definitely improve my quality of life.

Thank you for sharing this information.