- In a phase II clinical trial, only 47% of patients treated with Neuralstem's surgical stem-cell therapy responded to therapy, meaning they experienced stabilization of muscle function in the short term.
- Neuralstem has not been entirely forthcoming about the results of the trial. Instead, they chose to release results from the 15 ALS patients enrolled, which simply compared responders versus nonresponders, The Street reports.
- One of the secondary endpoints was response to therapy as measured by ALSFRS score, which measures muscle function. (A previous version of this post referred to this as the primary endpoint—that was actually safety). Subsequent analysis shows that therapy, known as NSI-566 did not significantly improve muscle function in ALS patients (CLARIFICATION: it did not significantly improve for patients in the entire aggregate of the trial, not just the ones who actually responded to the treatment). The stock was down 14% on the news.
Failure to disclose full results of a clinical trial is generally a bad sign, and efforts to hide clinical trial data are becoming increasingly difficult—especially given the focus on clinical trial transparency.
Neuralstem's comparison of responders versus nonresponders is considered wishy washy and led the market to discount the stock partly on lack of transparency. Nonetheless, Neuralstem plans to continue investigating the therapeutic potential of NSI-566, which is a cocktail of neural stem cells, in patients with ALS.