Anti-alpha-synuclein Parkinson therapy develops in clinical trials
A disease-modifying therapy to treat Parkinson’s disease has moved through a phase I clinical trial and is set to enter a phase II study, co-sponsor the Michael J. Fox Foundation for Parkinson’s Research (MJFF) announced Thursday.
The drug, PD01A, a vaccine produced by Vienna-based AFFiRiS AG, made its way through the trial, known as AFF008, and data from the study is imminent.
"A treatment that could slow or stop Parkinson's progression would be a game changer for the five million worldwide living with this disease and the many more who will become at risk as our population ages," said MJFF CEO Todd Sherer, PhD, in an official statement. "The AFF008 trial is one of the most promising efforts toward that goal, and we're proud to support this work of AFFiRiS AG."
The phase I trial involved the administration of two different doses of the vaccine. Preliminary results suggest that the therapy is feasible and free of serious health risk. PD01A’s main mechanism of treatment is in creating an immune response by tripping off alpha-synuclein specific antibodies. A-synuclein is of particular interest due to its presence in Parkinson’s pathology. Lewy bodies, an aggregation of a-synuclein and other materials, take up residence in brain cells causing eventual neurodegeneration and destruction of neurons. Researchers are hoping that an anti-synuclein therapy could diminish some of these effects and protect neurons from new damage.
Researchers from the first trial noted that PD01A was provided to 12 patients per group in doses of 15 micrograms and 75 micrograms. Subjects received four vaccinations in four-week courses. Another group of eight patients served as healthy controls. All participants were followed for a year. Results of the study showed that 50 percent of vaccinated subjects produced the desired antibodies. Preliminary analysis suggests stabilization of disease, but further research is needed to validate these findings.
The Michael J. Fox Foundation has announced that it will be financially backing a phase II trial, which is slated to take place in Vienna in an effort to evaluate the effects of the booster drug. Enrollment is scheduled to begin in September.