Gene Therapy for Parkinson's Disease

This trial tests a new gene therapy, CAP-003, to determine its safety and effectiveness for individuals with Parkinson's disease linked to a GBA1 mutation. Participants will receive a one-time infusion of the treatment and undergo regular checkups over two years. The study seeks adults diagnosed with Parkinson's using specific criteria who can generally walk without a wheelchair. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.

The trial information does not specify if you need to stop taking your current medications. However, it mentions that other illnesses or medications that may affect the study results could be a concern, so it's best to discuss your specific medications with the trial team.

Research has shown that CAP-003, a gene therapy, is safe and well-tolerated in early animal studies. One detailed study demonstrated that a single dose of CAP-003 administered through an IV increased brain activity related to the target enzyme (GCase) without causing safety concerns, indicating the treatment's tolerability so far.

Unlike the standard treatments for Parkinson's Disease, which typically include medications like levodopa and dopamine agonists, CAP-003 is a gene therapy offering a fresh approach. Researchers are excited about CAP-003 because it targets the disease at the genetic level, potentially addressing its root cause rather than just alleviating symptoms. This therapy involves a single intravenous dose, which could simplify treatment compared to the ongoing medication regimens currently necessary for managing Parkinson's symptoms. By focusing on modifying gene expression, CAP-003 has the potential to bring longer-lasting benefits for patients.

Research has shown that CAP-003 gene therapy offers promising results for treating Parkinson's Disease related to GBA1 mutations. One study found that a single intravenous dose of CAP-003 increased brain enzyme activity by up to 206%. This enzyme is crucial because low levels are linked to Parkinson's symptoms. In another study, CAP-003 improved enzyme activity by more than 30%, potentially enhancing brain function significantly. This trial will evaluate different dose levels of CAP-003 to determine its effectiveness in managing symptoms or slowing the disease's progression.¹²³⁴⁵