Friday, May 13, 2011

Epic fail?

"The search for a non-insulin approach to treating type 1 diabetes goes on. Diamyd Medical’s (DMYDF.PK) eponymous vaccine for the autoimmune form of the metabolic illness has failed in a European phase III trial, wiping out four-fifths of the Swedish group’s market capitalization, more than $450m, in one morning.
While the company is still awaiting results of a US trial due in 2012 this setback, following recent disappointments with antibody approaches, dashes most late-stage hopes for novel ways to treat early onset diabetes (Otelixizumab another disappointment in type 1 diabetes, March 14, 2011). For Diamyd, it casts doubt on receiving any milestones or royalties from partner Johnson & Johnson (JNJ), leaving the Swedish group shouldering half of all development costs."

Monday's news that the Diamyd vaccine had failed in its European Phase III trial was disheartening, to say the least. My mother, who is employed by Diamyd and is working on the company's trials in the U.S., was understandably more upset than I was, because she believed so strongly in this treatment and had been so excited to hear the results of the study. When it came out that the results were not good - well, it was a big letdown for her and her colleagues, and I was sorry to see such all-around disappointment. 
The news has been somewhat demoralizing for me, too, although I hadn't expected it to be. I thought once, "Since when can we vaccinate against autoimmune diseases?" And I said to my mom, "Have any chronic illnesses been cured in your lifetime? In the past 100 years? Why is everyone SO SURE that diabetes will be cured?" 
Don't get me wrong - I fully support diabetes research (I especially enjoy being a research subject - participating in clinical trials has given me a greater understanding of my condition and a sense of empowerment and I highly recommend signing up to be a guinea pig for one of these studies if you ever have the opportunity!), but I am not waiting for any big breakthroughs. I feel like I've been informed a million different times that they cured Type 1 diabetes in mice. That's nice. Keep working - I'll advocate for federal funding for your research and if you ever get to human trials I'll volunteer as a subject - but I'm not getting my hopes up anytime soon. 
Although I honestly don't expect a cure for this disease, I would be shocked if one day the scientific community announced that they were going to stop looking for one. And I guess that's kind of how I feel about the Diamyd study and the reaction to it - everyone is just giving up and it's unusual, and sad.
I was surprised to see the word "failure" in so many headlines and hear the simple phrase "it doesn't work" in conversations. It almost seems as if this whole avenue of research, which has been pursued with great hope and enthusiasm by so many for so long, is just being given up on. As if, with the results of this trial, Diamyd and the wider diabetes research community have put up a big sign that says "Dead End." 
I never assumed or expected this treatment to work - but I didn't expect a conclusive NO GO from it, either.
               I've been hearing so much over the past couple years about Diamyd and its GAD treatment - so much that I should be able to sum up the concept of GAD in a few words on my own, but instead let me give you the description from the Seeking Alpha article:
Diamyd’s treatment strategy focused on the presence of the protein glutamic acid decarboxylase (GAD) in insulin-producing pancreatic beta cells. Its hypothesis was the immune system in type 1 diabetics was overly sensitised to the protein, and the Diamyd vaccine, an isoform of recombinant glutamic acid decarboxylase, aimed to induce tolerance. The hope was to preserve beta function for longer and delay eventual insulin dependence, as had been demonstrated in phase II trials.”
There was good reason to believe that Diamyd was on the cusp of developing something that could prevent and stop Type 1 diabetes. My mom wanted to have Diamyd t-shirts that said "In GAD we trust" on the backs. 
And now, as of Monday, everything has changed and there can be no more hope placed in this - at least that is what the news reports are saying. Let me note that Diamyd's U.S. trials are continuing, and maybe there are valuable scientific insights to be gained from this "failed study" that could still somehow help people with diabetes. 
From the article I quoted earlier:
"With enrollment already complete in the remaining diabetes trial it is likely to run to completion; if positive trends were detected in Europe, investigators may still retain some hope that the resources already committed will yield statistically significant results in US diabetics. However, investors are probably right to write off the Diamyd vaccine – positive US trial results would now be a major surprise. With its next-most advanced products in the tricky Parkinson’s disease and pain spaces, Diamyd Medical is reverting to a higher-risk, mid-stage development company, and one with significantly less value."
(The full article is here.)
My mom, in her role coordinating things for the U.S. studies, had these wonderful postcards and stickers made that say "Don't let type 1 diabetes kick you around! Get involved in diabetes research."
I hope the decision-makers at Diamyd don't entirely abandon their efforts to treat diabetes... this enterprise with the GAD vaccine was not just a branch of a business that can be shut down in a "let's cut our losses and move on" kind of way; people with diabetes around the world invested their emotions and energies into believing in this, and it's just so tough for them to suffer a conclusive letdown like this. I'm not sure if I'm advocating for companies like Diamyd to slowly but ambiguously reveal that their treatments aren't actually effective rather than being honest and upfront and saying "hey, this is the end," but that sure would have been easier to deal with. I think. I don't know.

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