Experimental Parkinson’s Treatment Elicits Antibodies Against Toxic Protein in Phase I
20 Jun 2024
Clinical ResultPhase 1Phase 2Immunotherapy
Pictured: Illustration of a brain surrounded by a vaccine and a needle/Nicole Bean for BioSpace
During the past few years, disease-modifying treatments for Alzheimer’s disease have broken through, but another prevalent neurodegenerative disease, Parkinson’s, has lagged behind. Vaxxinity is one company looking to change that. It published data Thursday showing that its experimental immunotherapy could improve daily movement in Parkinson’s patients and elicit antibodies against a toxic protein involved in the disease process.
The treatment “could potentially be disease-modifying as shown by its high immunogenicity, and its ability to reduce aggregated [αSyn] in the CSF [cerebral spinal fluid] of [Parkinson’s] patients,” Vinata Vedam-Mai, an assistant professor in the department of neurosurgery and the Fixel Institute at the University of Florida, told BioSpace in an email.
The article, published in Nature Medicine, describes the results of a Phase I study of UB-312, a synthetic peptide designed to provoke an immune response against toxic clumps of the alpha-synuclein (αSyn) protein. Duplications, point mutations or single nucleotide polymorphisms in the SNCA gene encoding αSyn contribute to Parkinson’s disease, and αSyn aggregates in the form of Lewy bodies are common hallmarks of the condition.
The data—which were presented earlier this year at the AD/PD 2024 International Conference on Alzheimer’s and Parkinson’s Disease—showed that treatment with UB-312 elicited a 20% reduction in clumped αSyn levels for patients given UB-312, compared with a 3% increase in patients given a placebo. Additionally, 12 out of 13 patients (92%) developed antibodies against these harmful clusters, though UB-312 did not lead to the generation of antibodies against full-length αSyn compared with placebo. Patients in the treatment group also reported improved daily movement.
“We target the root cause of Parkinson’s, not just the symptoms,” Mei Mei Hu, CEO and co-founder of Vaxxinity, told BioSpace in an email. “It offers the first real hope for patients and their families in the fight against Parkinson’s.”
In terms of safety, most treatment-emergent adverse effects were mild or moderate, according to the paper, with three severe adverse events—one possibly treatment-related—reported.
The fact that the treatment “appears to be well-tolerated” also makes it promising as a disease-modifying therapy, Vedam-Mai said. As a future step, she noted that “it would be desirable to see effects of the therapy on the brain using recent MRI techniques.”
Vedam-Mai also said that the study of UB-312 “needs to be scaled up to see better the effects of this treatment.” She additionally recommended that the patient population be more diverse in order to better show efficacy. “The patients should also be chosen at different and later stages of the disease to show that the therapy can ameliorate more advanced patients.”
Indeed, Vaxxinity hopes to “reproduce our promising results” in more Parkinson’s patients by moving into a larger Phase II study, Hu said. That study has not yet been scheduled.
Heather McKenzie is a senior editor at BioSpace. You can reach her at heather.mckenzie@biospace.com. Also follow her on LinkedIn.
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