Steve Heronemus

The FDA, Genervon and Kangaroo Trials

By the time another kangaroo trial is over, 15,000 pALS will be dead. Conditional approval of GM6, giving supervised access to thousands of pALS is the only way the FDA and Genervon will know if GM6 is effective.

People with ALS may not have voices but we deserve to be heard. In October 2014, biomedical firm Genervon announced results of their Phase 2 clinical trial of a novel drug code-named GM6 in people with ALS. In this trial, 92% of patients demonstrated either slowing or stopped disease progression and, as in another trial of GM6 in Parkinson’s Disease, not one person had adverse effects.

Based on these facts Genervon will petition the FDA to grant conditional approval for GM6. Over 100,000 people have signed a petition supporting this request. Conditional approval is a defined program of the FDA that allows wide distribution of a new drug when the patient need is critical and there are no other reasonable treatment options. ALS fits this description perfectly as it is always fatal and riluzole, the only drug approved to treat the disease, lengthens survival by a scant 2-3 months. Genervon would be required to continue monitoring patients for safety and efficacy during this period.

By all accounts Genervon’s petition is dead on arrival at the FDA. The reasons are all too familiar to pALS. The trial was small – 8 pALS received GM6 and 4 got a placebo. The trial was short, *12 weeks. Neither the trial size or length are unusual for Phase 2 trials in the ALS world. Clinic neurologists, researchers and Institutional Review Boards, the gatekeepers of approval for doctors to participate in new therapies and procedures, all want to see results from a larger, longer trial before considering the widespread use of GM6. Such a trial would likely involve around 100 patients and could easily take 3 years before the FDA gives a go/no go decision. Nonprofits like Les Turner Foundation and ALSA won’t throw their weight behind Genervon’s petition without approval from the above-mentioned medical professionals.

And so GM6 will likely undergo another trial, but here’s the dirty secret the medical professionals and scientists don’t talk about. Every trial in the history of ALS is mathematically invalid. ALS is not one disease, it is a category of diseases with many causes and many pathways of disease progression that happen to have similar symptoms. Worse, we don’t know how many different diseases there are or how to tell most of them apart. Clinical trials depend on grouping like patients together to form a statistically significant sample group that will be a good indicator of success with the entire population. At present it is impossible to create such a group of patients. There is no way to know if a trial needs to include 100 people or 1000 to be scientifically valid. But we will have to endure the ruse.

The one group left out of this decision process is the patients and their loved ones who live with this vile, damnable disease. Every day we lose our ability to move, speak, swallow and breathe. Every day the dreams of a long future with our families disappear. Every day we long for a past when we could hold, hug and kiss our children and spouses. Every day we die.

By the time another kangaroo trial is over, 15,000 pALS will be dead. Conditional approval of GM6, giving supervised access to thousands of pALS is the only way the FDA and Genervon will truly know if GM6 is effective. We pALS know there is a risk GM6 will not work and we know other promising drugs have accelerated disease progression in larger trials. But we also know our voices should be heard and we say conditional approval is the only way to do real science.