$6M grant aids work into calcium channel blocker to protect neurons
Inhibiting the CaV2.3 channel could be disease modifying for Parkinson's
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The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has awarded a $6 million grant to Lario Therapeutics to advance early studies into CaV2.3 calcium channel inhibitors as a potential disease-modifying therapy for Parkinson’s disease.
Lario’s preclinical program is being developed in collaboration with the Oxford Parkinson’s Disease Centre, which will test various inhibitor compounds in stem cell models derived from Parkinson’s patients. Richard Wade-Martins, a professor of molecular neuroscience at the University of Oxford and head of the Parkinson’s center, will lead the testing effort.
“We are grateful to MJFF for their funding support for Lario’s efforts to test and develop a potential new therapy designed to prevent the loss of neurons that causes Parkinson’s disease. If our research is successful, this will represent an important new treatment option for patients,” Tom Otis, PhD, chief scientific officer at Lario, said in a company press release.
Blocking the CaV2.3 calcium channel aided neurons in a disease mouse model
“Our research centre works to understand the development of Parkinson’s, with the ultimate aim of targeting the molecular mechanisms of disease, to prevent disease onset or to delay progression,” said Wade-Martins. “We see great potential in the CaV2.3 program and are very happy to collaborate to study patient stem cell-derived neuronal models as part of the research.”
Parkinson’s is caused by the progressive dysfunction and death of dopaminergic neurons, the nerve cells responsible for producing the signaling molecule dopamine, in a part of the brain called the substantia nigra. This loss leads to a variety of disease motor symptoms — tremors, muscle stiffness, and slowed movement — as well as such Parkinson’s nonmotor symptoms as depression, anxiety, or cognitive issues.
Although exactly what prompts the loss of dopaminergic neurons is not known, studies point to a potential CaV2.3 calcium channel role in neuronal death.
Calcium channels are specialized proteins that regulate the movement of calcium ions in and out of a cell, and are important for the function and survival of neurons.
In studies in a Parkinson’s mouse model, the absence of CaV2.3 was seen to protect neurons from degeneration. This suggests that the selective inhibition of these channels may be a disease-modifying approach for Parkinson’s. By definition, a disease-modifying therapy targets a condition’s underlying cause, and works to prevent, slow, or reverse its progression.
Lario aims to develop selective CaV2.3 channel blockers that could target with high specificity and acceptable safety calcium channels in dopaminergic neurons.
“This grant will help drive our ambition to progress our CaV2.3 program swiftly towards the clinic, to provide a new, effective treatment option for people with Parkinson’s disease,” said Henning Steinhagen, PhD, Lario’s co-founder and CEO.
“We look forward to seeing the results of Lario’s research on CaV2.3 as a novel disease-modifying approach for Parkinson’s,” added Gaia Skibinski, PhD, director of research programs for the MJFF.