Efforts Initiated to Conduct First FDA Phase I Trial for ALS

No orphan has ever had this much family.

Although designated an "orphan disease" because it affects less than 200,000 Americans, Amyotrophic Lateral Sclerosis (ALS) saw millions of benefactors stand tall last year to douse themselves with ice water in support of a cure.

The numbers were staggering. According to the ALS Association, of the over 17 million people who took the Ice Bucket Challenge, 2.5 million donated $115 million to fight the degenerative neurological condition known as Lou Gehrig's disease. It was single largest episode of giving, says the association, outside of a disaster or emergency.

So, whatever happened to all that money? One summer later, the ALS Association says that about $77 million has been earmarked for research aimed at finding a treatment and ultimately a cure.

One research initiative for ALS stands out from them all. Hospital for Special Surgery (HSS) in New York City and Tikun Olam, an Israeli medical marijuana company founded in 2005, have initiated efforts to collaborate on a much-needed clinical trial that would test the effectiveness of certain high cannabidiol strains of the drug on ALS.

The initiative is being lead by Dr. Dale J. Lange, M.D., Chairman of Neurology & Neurologist-in-Chief and Professor of Neurology at Weill Medical College of Cornell University, who would write the protocols for the trial. "I am very interested in looking into the effects of high CBD/low TCH in patients with ALS and UMN predominant motor system disease," said Dr. Lange. "We have more than 20 years of experience conducting clinical therapeutic trials for patients with neuromuscular disorders at all phases of drug development. This would be a phase I trial assessing safety and data gathering for endpoints."

Tikun Olam, which is Hebrew for "Healing the World," is already licensed and regulated in Israel and Canada, and has submitted a proposal under the name Compassionate Care Center of New York (CCCNY) to the NYS Department of Health to be one of five companies awarded a medical marijuana license in coming days. Working on this initiative for CCCNY is Chief Medical Officer, Dr. Suzanne Sisley, M.D., who brings with her a unique perspective as the only medical cannabis researcher in the U.S. to receive FDA approval for a Phase II Clinical Trial for Post Traumatic Stress Disorder.

"In preparation for this IND Application and others, as U.S. restrictions on medical cannabis research continue to ease, we are in the process of getting GMP Certification, so our production protocols are compliant with FDA requirements," said a spokesperson for Tikun Olam. "We will be ready by the time this clinical trial commences."

For now, it looks like it will be a while before all that ice water from the ALS Ice Bucket Challenge comes to a boil for those afflicted with the disease, but this is why I was so intrigued by this one initiative that is sure to turn up the "flame".