Pharmaceutical company MSD and the Francis Crick Institute are partnering to try to learn more about what causes motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS), so that treatments may be developed.
Funded by MSD and the Medical Research Council, the project is the first under a five-year agreement between MSD and the London-based biomedical discovery institute to find out more about particularly complex disorders such as ALS.
Current ALS treatments cannot cure or undo damage the disease causes, but can slow disease progression and increase patient comfort and independence.
“In the clinic, it’s devastating when I have to tell a patient they have motor neurone disease,” Rickie Patani, MD, project leader and a Crick and University College London (UCL) research group leader, said in a press release. “Often, the patient first walks in with mild weakness in a limb, and then I see the disease progressing relentlessly with every visit. Within a year or two, they might be in a wheelchair and require breathing support. This disease destroys families, and I feel profoundly guilty that we still have no effective treatments to offer.”
The near-term goal is to understand the disorder’s fundamental biology. That alone could offer patients solace, Patani said, even when no promising therapies loom.
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“I am excited to start our new collaboration with MSD, which we hope will give us a comprehensive understanding of the earliest events that cause motor neurone disease. This is a unique science-led partnership, driven by our shared commitment to helping patients in the long term,” he said.
The effort is intended to capitalize on earlier work by Patani’s team, which pinpointed essential mechanisms able to destroy motor neurons in patients. By examining patients’ skin cell-derived neurons, the scientists hope to gain knowledge that could lead to identification of prospective treatment targets.
“I am hopeful that this collaboration could produce groundbreaking research which will ultimately change how we treat a broad spectrum of diseases in the future,” said Fiona Marshall, vice president, head of discovery science at MSD United Kingdom. “This collaboration is a great example of the opportunities that can arise from a thriving life science community when doors and minds are open.”
The project is unconventional in that it takes a multidisciplinary approach, involving academic, clinical, and industry research and calling for neurologist Patani to work with Crick, UCL, and MSD researchers.
“When outstanding scientists in industry and academia work together, it creates a great opportunity to accelerate discoveries that can improve human health,” said Fiona Watt, executive chair of the Medical Research Council.
One of the Crick’s founding partners, the Medical Research Council works to improve the health of U.K. residents through education and research support. Established in 2015, Crick’s goal is to understand the fundamental underlying biology of diseases.
“There is so much we don’t know about neurodegeneration, and working together from such an early stage will help us to build knowledge and understanding from the ground up,” said Veronique Birault, Crick’s director of translation. “By combining our expertise, we hope to truly advance the field and offer hope for future generations.”