On October 3rd and 4th, the ALS Therapy Development Institute, our colleagues in Kendall Square, opened their doors for their sixth annual conference and open house, and Prize4Life was in attendance.
One of the great things about ALS-TDI’s Summit is the lengths they go to ensure the event is open and comprehensible to patient and families, not just researchers and scientists. As open and comprehensible as an incredibly complex and evolving scientific field can be, of course…
The research symposium portion of the Summit featured speakers such as Dr. Steve Perrin, CEO and Chief Scientific Officer of ALS-TDI, Dr. Fernando Vieira, Director of In Vivo Validation at ALS-TDI, Dr. Merit Cudkowicz, ALS Clinic Director at Mass General Hospital and Co-Founder of the Northeastern ALS Consortium, and several others. Topics ranged from drugs currently in the development pipeline and new mouse models, to the ongoing controversy over stem cells and which clinical trials show the most promise for participants.
This article in the MDA/ALS Newsmagazine covers many of the presentations and discussions in great detail. And ALS-TDI has also posted video of the conference on its website, though you do have to register with an email address to view it.
Two portions of the day were of particular interest to us at Prize4Life: Dr. Cudkowicz’s and Dr. O’Neill’s (of Biogen Idec) discussions on the need for a biomarker to advance research.
Dr. Cudkowicz acknowledged some of the frustration she has witnessed in the ALS community at the fact that the disease was discovered in the late 1800’s and yet there remains no effective treatment. But she pointed to the relatively rapid progress following the discovery of a gene associated with ALS in 1993, which brought new scientists, new minds, and new ideas into the ALS field. More recently, she discussed how the 2006 correlation of TDP-43 with sporadic ALS caused scientists studying TDP-43 in other fields, such as dementia, to begin to think about ALS and how a therapy might potentially be developed.
In order to spark a leap forward in research progress, Dr. Cudkowicz says a biomarker is needed. The vast majority of ALS treatments fail in Phase II trials. This makes the development of drugs both incredibly time-consuming and incredibly costly. A biomarker dramatically shortens the time required for drug development, thus driving down the overall cost of that development. If such a biomarker were discovered, “an explosion of [ALS] therapies would result,” according to Cudkowicz. When asked by patients what they can contribute, it is towards the discovery of a biomarker that Cudkowicz points them—encouraging them to donate blood and tissue samples which are critical tools for researchers on the biomarker quest.
Dr. Gilmore O’Neill, Vice President of Experimental Neurology at Biogen Idec, also emphasized the importance of a biomarker. He explained that any trial has three possible outcomes. A positive result is, of course, the most desirable—when a drug slows or arrests the progress of a disease. But the least desirable result is not a negative study, but rather a failed one—a study where not only does the drug not have the desired effect, but researchers cannot even be certain if the drug hit its intended target.
In essence, failed trials tell us nothing, and they do not advance research in the way that negative results can. An effective biomarker means, in Dr. O’Neill’s words, that researchers “…can throw out the garbage very quickly.” Without a biomarker, trials are forced to include more patients, must last longer, and are more likely to yield uninterpretable (failed) results.
Dr. O’Neill discussed biomarkers in the context of drug development for several other diseases—his underlying theme being the need for an ALS biomarker. At Prize4Life, we wholeheartedly agree. We believe the discovery of a biomarker for ALS is so important that we’ve put up a $1 million prize to spur on research.
And there has never been an organization more anxious for a reason to give away its money.