At My Parkinson's Info we are always cautious when we are talk about clinical trials, because most of them do not work out. So, we do not want to get peoples' hopes up, but we want to pass along the progression of therapies that appear to have a bit more promise. Right now we suspect that CERE-120 has about a 50/50 chance of proving to be effective as well as safe. It may be five years or more away from being approved by the FDA and available to people with Parkinson's disease.
The Phase I clinical trial involved 12 patients with Parkinson's disease. The growth factor gene was delivered as part of a modified virus, or "viral vector", called adeno-associated virus (AAV). This viral vector delivers the gene to the correct brain cells, but has been modified so that it cannot reproduce or damage brain cells. The growth factor gene neurturin was utilized, which is a protein closely related to GDNF. Neurturin has been shown in laboratory studies to help prolong survival of dopamine-producing cells. AAV-neurturin was delivered directly to the brain via 16 needle injections into the striatum, the part of the brain most deficient in dopamine. This was performed through small openings in the skull.
The patients were studied using standard rating scales of movement in PD, the Unified Parkinson's Disease Rating Scale (UPDRS) prior to surgery and on a continual basis after the surgery for 12 months. At the end of 12 months the research team concluded that:
- There were no major adverse effects from this treatment at the low or high doses.
- In nine of the 12 patients for which one-year outcome data was available, the improvement in the UPDRS was 38 percent.
"Patients with PD urgently need therapeutic approaches that not only improve their symptoms and daily functions, but positively modify the underlying components of the disease," Dr. Philip A. Starr stated. "Existing therapies for PD treat only the symptoms, and are effective for a limited period of time, so any trial that is safe and results in promising efficacy data is worth pursuing. The safety data and preliminary efficacy data that resulted from this Phase 1 study are encouraging, and clearly warrant the need for a larger, Phase II study," concluded Dr. Starr.
Dr. Starr presented the results of this clinical trial during the 75th Annual Meeting of the American Association of Neurological Surgeons in Washington, D.C.
Novel gene therapy hints at improvement from the Associated Press and FortWayne.com
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