On July 14, 2013, the New York Times published Do Clinical Trials Work?, an article focused on clinical trials for cancer, but which just as easily could be about ALS/MND. This article, reprinted with the authorization of The New York Times, speaks to the present failure of Phase III Clinical Trials due to the heterogeneous, not homogenous, nature of patient groups--a theme we have been pursuing for several years, most recently in our address to the FDA in February 2013.
In 2007, researchers mapped the complete genome of a human being, Craig Venter, laying the foundation for personalized medicine through genotyping. That phenotyping, of course, followed the even more massive effort by National Institutes of Health (NIH) and Celera Genomics, beginning in the 1990's, to identify and map all 25,000 genes in the human genome system.
Applied to ALS/MND, this article suggests the need for genetic phenotyping as developed at Johns Hopkins and elsewhere, rather than the common practice of "big numbers" Phase III clinical trials (trials with massive patient populations) that continue to fail--as happened in the Phase III trial for Dexpramipexole. This drug, also known by its chemical name R+Pramipoexole, was considered to be the greatest hope for the ALS/MND patient community, having succeeded in its nearly 10 years of Phase I and Phase II testing by Knopp Biosciences and its Investigational New Drug testing by Dr. James Bennett of Virginia Commonwealth University and colleagues. However, on January 3, 2013, Biogen Idec stated that Dexpramipexole was "no more effective than the placebo" in its 943-patient Phase III trial. Over 95% of Phase III trials do not succeed, which is why this phase is often referred to as the "valley of death."
For over 150 years, scientists have tried and failed to develop systematic ALS/MND drug treatments through the classical methods. Now, it is time to change course and use genetic profiling to optimize the trial procedure.