Genervon's small sample clinical trial

Amyotrophic lateral sclerosis (ALS) is a deadly motor neuron disease which causes death of neurons and ultimately wasting of muscles. The average survival after onset is less than four years, although 10% of those diagnosed live for more than 10 years. The famous Physicist Stephen Hawking is perhaps the best known long term survivor of the disease. More recently ALS was in news for the ice bucket challenge.

Genervon, a privately held drug development company, has conducted Phase 2 clinical trials on 12 ALS patients over a period of 12 weeks with its drug GM 604. They have performed small sample statistical tests to argue in favour of the drug’s effectiveness – see box below (source: http://www.genervon.com/genervon/about_pressreleases.php). (Forced Vital Capacity or FVC is a measurement that assesses respiratory compromise in ALS patient).

Critics have panned this evidence because of the small sample size. Genervon on the contrary argues that small sample tests have limited power and the fact that significant differences have appeared with such a small sample is all the more remarkable. The ubiquitous t-distribution is quite at the centre stage in this debate.

There is a change.org petition with over 536,000 signatures requesting the US-FDA to rapidly approve GM604. It will be interesting to follow the course FDA takes given the evidence in favour of the drug comes from such a small sample. More importantly though, an effective drug that halts ALS will be a really welcome breakthrough.