Parkinson’s drug trial has researchers optimistic
BY MONIFA THOMAS Health Reporter July 31, 2014 10:10AM
File lab photo. AP Photo/Keystone, Fabrice Coffrini
Updated: July 31, 2014 4:11PM
Results of a Phase I clinical trial of what could be the first vaccine for Parkinson’s disease has experts cautiously optimistic.
Those results were released Thursday by the maker of the drug and the Michael J. Fox Foundation for Parkinson’s Disease.
The disease, a progressive disorder of the nervous system that affects movement, is believed to be caused by abnormal deposits in the brain of a protein that helps nerve cells talk to each other.
Many drugs can make movement easier or alleviate other symptoms of the disease. But no drug has been shown to effectively slow or stop the progression.
The drug being tested is known as PD01A, made by AFFiRiS AG, an Austria-based biotech company. It is supposed to stimulate the body’s immune system to produce antibodies that bind to the protein alpha-synuclein, clearing it from the brain and slowing disease progression.
Phase 1 looked only at whether PD01A was safe and tolerable to the relatively small sample of 32 people with Parkinson’s disease. The drug passed that test.
It was also found to generate alpha-synuclein-specific antibodies in 50 percent of the patients. If that is duplicated on a larger population, it could be a breakthrough for Parkinson’s disease.
“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,” Todd Sherer, CEO of the Michael J. Fox Foundation, said in a statement. The most recent drug trial “is one of the most promising efforts toward that goal.”
Dr. Christopher Goetz, director of the Parkinson’s Disease and Movement Disorders Center at Rush University Medical Center, agreed Phase I of PD01A is “very promising.”
But Goetz, who was not involved in the study, also cautioned that because testing of this drug is in the early stages, it remains to be seen whether increasing antibodies to fight alpha-synuclein actually does anything to effectively treat Parkinson’s disease.
Indeed, not everyone is convinced of that premise. And the findings are considered preliminary because they haven’t been published in a peer-reviewed medical journal.
Dr. Jose Biller, chair of the neurology department at Loyola Medicine, said he was cautiously optimistic at what could wind up being a “significant advance” in how Parkinson’s disease is treated.
“The basic science that is behind this trial is quite robust. And therefore, while nobody can talk about guarantee ….I think that it is a very cogent approach,” he said.
That said, though, Biller, who was not involved in the study, said, “We need to wait for the [follow-up] results.”
The next study will take place in Vienna and focus on assessing the immunological and clinical effects of a boost vaccination. Recruitment is expected to begin September.