Researchers led by Omar Mabrouk reported that the first human trials of the experimental drug BIIB094 (ION859) have shown promising results, GxP News writes. The drug significantly reduces levels of the LRRK2 protein — a key factor in the development of Parkinson’s disease. The findings were published in the journal Nature Medicine. The drug is a joint development of Biogen and Ionis Pharmaceuticals.
The clinical trial, named REASON, enrolled 82 patients, GxP News reports. In the randomised, placebo-controlled study, participants received the drug directly into the cerebrospinal fluid. Treatment was generally safe: side effects were mostly mild or moderate and did not require discontinuation of therapy.
Analysis showed that LRRK2 protein levels in patients receiving the drug fell by up to 59%, GxP News reports. The effect was observed regardless of the presence of a genetic mutation, suggesting potentially broader applicability of the therapy.
The researchers stressed that this is only an early-stage trial evaluating safety and biological effect, not clinical improvement, GxP News notes. Nevertheless, the results confirm the possibility of targeting the disease’s mechanisms rather than just its symptoms, and pave the way for the development of new targeted treatments.
Biogen, which funded the study, said it is prepared to provide de-identified participant data to qualified scientific groups for independent verification and follow-up research, GxP News reports.
Last month, Japanese drugmaker Sumitomo Pharma received approval to manufacture and sell the experimental Parkinson’s drug Amshepri, GxP News notes. The company claims it is the world’s first regenerative cell-based therapy using iPS cells to receive official approval.
Parkinson’s disease is the second most common chronic neurodegenerative disorder after Alzheimer’s, in which dopamine-producing neurons die, GxP News explains. This leads to serious motor impairments: tremor, muscle rigidity, slowness of movement (bradykinesia) and balance disorders.