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Wednesday
A new gene therapy approach was safe and well tolerated for Parkinson's
In this small study, a new gene therapy approach was safe and well tolerated. The idea was to make striatal cells “dopamine factories.” Notably, this therapy involved a lentivirus, unlike the traditionally used adenoviruses. Whether this change in vector will prove beneficial remains for future studies to show. Although this was not a transplant trial, one cannot help but be concerned for the emergence of dyskinesia in the off-medication state (runaway dyskinesia). The 12-month follow-up and the sample size were inadequate to conclude that this effect will not occur. The choice of surgical site minimized the possibility of runaway dyskinesia. The positive change in motor outcomes is good news, but this was an open-label study. The motor outcomes were, disappointingly, much lower than the preoperative levodopa response. For this therapy to be viable, a follow-up study must demonstrate outcomes similar to deep brain stimulation. The data suggest that the highest dose may have the best chance to optimize outcomes in future studies. Even if successful, ProSavin will not treat the many nonmotor and levodopa-resistant symptoms, which are critical to address.
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In this small study, a new gene therapy approach was safe and well tolerated. The idea was to make striatal cells “dopamine factories.” Notably, this therapy involved a lentivirus, unlike the traditionally used adenoviruses. Whether this change in vector will prove beneficial remains for future studies to show. Although this was not a transplant trial, one cannot help but be concerned for the emergence of dyskinesia in the off-medication state (runaway dyskinesia). The 12-month follow-up and the sample size were inadequate to conclude that this effect will not occur. The choice of surgical site minimized the possibility of runaway dyskinesia. The positive change in motor outcomes is good news, but this was an open-label study. The motor outcomes were, disappointingly, much lower than the preoperative levodopa response. For this therapy to be viable, a follow-up study must demonstrate outcomes similar to deep brain stimulation. The data suggest that the highest dose may have the best chance to optimize outcomes in future studies. Even if successful, ProSavin will not treat the many nonmotor and levodopa-resistant symptoms, which are critical to address.
READ MORE