New Parkinson's drug could slow or reverse progression of disease, researchers say: 'Big step forward'
A new therapeutic that could slow down or halt the progression of Parkinson's disease has completed Phase 1 trials with positive results. Researchers and doctors spoke about the findings.
A new drug is being tested to relieve Parkinson’s disease (PD) symptoms – and it’s reportedly showing promise.
The drug is designed to slow or halt the progression of the disease in patients by targeting toxic proteins that build up in the brain, according to the study published in the journal Nature Medicine on June 20.
Researchers conducted a phase 1 placebo-controlled trial of an investigational immunotherapy drug called UB-312, testing for safety, tolerability and immunogenicity (strength of immune response).
The trial results showed that the drug was generally safe and well-tolerated by patients as a disease-modifying treatment for Parkinson's.
The researchers stated that to their knowledge, this is the first report showing a positive effect of an investigational therapy of this kind.
There is currently no cure for Parkinson's, only drugs that treat the symptoms.
"UB-312 is designed to modify the course of Parkinson’s disease by targeting the underlying cause," Lou Reese, co-founder of Vaxxinity, the Texas-based pharmaceutical company that worked on the study, told Fox News Digital.
Leiden University Medical Centre in the Netherlands, University of Texas McGovern Medical School, and Mayo Clinic in Rochester, Minnesota, also helped conduct the study.
"In our phase 1 trial, we showed that UB-312 may be able to stop or even reverse the course of disease by successfully targeting aggregated alpha-synuclein."
(Alpha-synuclein is an acidic protein that builds up in the brains of Parkinson's patients.)
UB-312 is given as an injection, typically via multiple doses over several months, Reese noted.
During the trial, PD patients reported improved daily movement after receiving the new drug.
The medication was found to be safe and well-tolerated in healthy people and Parkinson's patients alike, with only minimal side effects that included headaches and fatigue, according to Reese.
UB-312 works by targeting the "harmful Parkinson’s protein" alpha-synuclein and producing antibodies against it, the researcher said.
In the trial, 12 out of 13 patients developed antibodies, which Reese described as a "big step forward in PD treatment."
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"These antibodies reached the brain and interacted with the target protein," he said.
Based on the "promising" results of Phase 1, UB-312 will now progress into phase 2 trials, focusing on a larger patient population while optimizing the dose, according to Reese.
"The ultimate goal is to develop effective, disease-modifying treatments that can improve outcomes and provide hope for individuals living with Parkinson’s disease," he added.
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"This is exciting because we are targeting the root cause of Parkinson’s and not the symptoms. It’s the first drug ever to take a patient from positive to negative."
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Some doctors, however, cautioned that Parkinson's patients shouldn't get their hopes up just yet.
"People with Parkinson’s should be aware that although the findings were interesting, this was only a safety, tolerability and immunogenicity study, and thus there is a long way to go for development of this treatment," Michael S. Okun, M.D., Parkinson’s Foundation medical adviser and director at the Norman Fixel Institute for Neurological Diseases at the University of Florida Health, told Fox News Digital.
Okun was not involved in the study.
The injections did seem to "rev up the immune system," Okun acknowledged, as the researchers observed the appearance of antibodies in the blood samples of most study participants.
"The worry that many scientists have about this approach is that it may neither improve clinical outcomes nor slow disease progression," he added.
"Two similar antibody trials of prasinezumab and cinpanemab were published in 2022 in The New England Journal of Medicine, but both of those trials failed to meet their primary outcomes."
Okun concluded that "time and science will help us to determine if this newer approach will fare better."