The Food and Drug Administration has fast-tracked a drug that could provide treatment for those who have Parkinson's Disease.
The single-dose drug could help in the treatment of 7-10 percent of Parkinson's patients with a GBA1 mutation, according to Prevail Therapeutics.
The first part of a two-part trial will begin later this year.
“PR001 is an experimental therapy that does not yet have an established safety or efficacy record in people, so the purpose of the trial is to test both aspects,” said James Beck, chief scientific officer at the Parkinson's Foundation.
Around 90,000 patients with Parkinson's Disease, which affects nearly 7 million worldwide, have the genetic mutation that PR001 targets, however, it can be difficult to narrow down those patients.
“With any experimental therapy, there can be rare adverse events,” Beck said. “If PR001 is successful, it could potentially stop the progression of Parkinson’s disease and transform the lives of people who receive the therapy. That remains a high hurdle which none have succeeded in overcoming.”
The most likely effect of PR001 is that it will fail to be productive in treating Parkinson's, Beck said, as many potential treatments for this disease have been. The neurodegenerative disorder currently has no treatment course to slow down its progressive nature, only treatments that target specific symptoms.
“These symptomatic medications control many of the obvious symptoms of the disease but there is tremendous pill burden and increased medication side effects as the disease progresses and the therapies become more complex,” Dr. Beck said.
Despite the difficulty in treatment for the disease, the Parkinson's community remains hopeful that a cure will be found and treatments like PR001, which target specific areas of the illness show potential to treat the 10 percent of patients that have the genetic form of the disease. Identifying those who could benefit from this treatment can be difficult and then there is the chance the treatment will not work.
“This is why the Parkinson’s Foundation has just launched an effort to speed testing,” Beck said. “Hopefully, approval of these new precision medicine therapies by providing widespread genetic testing and counseling to those living with Parkinson’s.”
