This pharmaceutical company is irresponsible beyond belief. Based on results of a tiny Phase 2 clinical trial, Genervon has petitioned the FDA to approve their drug for ALS under the Accelerated Approval program, which bypasses the normal requirement for a Phase 3 trial. In concert with this petition they have issued press releases indicating that the drugs effects are “very robust” and “dramatic.” This media blitz has caused ALS patients, faced with near certain death, to put huge pressure on the FDA to approve the drug.
The thing is, the results of their Phase 2 trial are neither robust nor dramatic. Their data (Table 3 in the following link) shows that the results just barely reach statistical significance, or fail to reach statistical significance, depending on which statistical test is applied. The company argues that this is remarkable because of the small number of patients enrolled in the trial. But the company has it exactly backwards - the smaller the number of individuals in a trial, the less the statistical "p" values should be trusted. http://www.genervon.com/genervon/about_pressreleases.php