Gene Therapy AB-1005 Shows Promise in Treating Parkinson’s Disease

AB-1005, a gene therapy being developed by AskBio, appears to be safe and may help improve motor symptoms in people with Parkinson’s disease, regardless of how long they’ve lived with the diagnosis. This is based on the full results of a small early-stage (Phase 1) clinical trial conducted in the United States. How AB-1005 Works Parkinson’s disease affects brain cells that produce dopamine, a chemical involved in movement and coordination. Over time, these cells become damaged or die. AB-1005 is designed to deliver a protective molecule called glial cell line-derived neurotrophic factor (GDNF) directly into a brain region called the putamen, which is deeply affected in Parkinson’s. GDNF helps support and protect dopamine-producing nerve cells. The therapy is given through a surgical procedure that places the treatment precisely where it’s needed. Key Findings from the Phase 1 Trial The Phase 1 trial included 11 adults with mild or moderate Parkinson’s disease. Participants continued their usual treatments like levodopa while receiving the gene therapy. They were followed for 18 months, and longer-term monitoring is ongoing. Safety Results No serious safety concerns were directly linked to the gene therapy itself. One person experienced a complication (reduced blood flow in the brain) possibly related to the surgical procedure. Common temporary side effects included headaches and fatigue, which resolved within a month. Researchers concluded that AB-1005 was safe and well tolerated over the 18-month period. Encouraging Motor Symptom Outcomes Brain imaging showed that the gene therapy successfully delivered GDNF to about two-thirds of the targeted brain cells. In People with Mild Parkinson’s: Motor symptoms did not get worse, which is notable since Parkinson’s symptoms typically progress over time. In People with Moderate Parkinson’s: Motor symptoms improved over 18 months. Improvements were observed even without the effects of other medications. Improved Daily Function Among participants with moderate disease: “Off” time (when symptoms are not well-controlled) decreased by 1.7 hours per day. “Good on” time (periods of better symptom control) increased by 2.2 hours per day. The need for Parkinson’s medications was reduced. Non-Motor Symptoms and Next Steps While motor symptoms showed encouraging changes, non-motor symptoms (such as mood or sleep difficulties) did not change significantly. Because this was a small study without a placebo group, researchers emphasized the need for larger, more rigorous trials to better understand the potential of AB-1005. Phase 2 Trial Now Underway Based on these promising results, AskBio has launched a Phase 2 clinical trial called REGENERATE-PD. This ongoing trial aims to enroll around 87 adults with moderate Parkinson’s across the U.S., Poland, U.K., and soon Germany. It will compare AB-1005 to a sham (placebo-like) procedure to test its true effectiveness. In Summary AB-1005 gene therapy shows early signs of being safe and potentially helpful in stabilizing or improving motor symptoms in people with Parkinson’s disease. While more research is needed, these results offer hope for a treatment that could modify the course of the disease — not just manage symptoms.