In January, neuroscientists from the University of Edinburgh announced that they had identified, and more importantly, repaired a defect in cultured motoneurons from an inheritable form of amyotrophic lateral sclerosis (ALS). This condition is commonly known as Lou Gehrig’s disease here in the States, and is a sub-class of a larger family of conditions known as motoneuron (or motor neurone to the Brits) disease. What’s the Harry Potter connection?
The study was funded by the Medical Research Council, Motor Neurone Disease Association, Euan MacDonald Centre for MND Research, My Name’5 Doddie Foundation, UK Dementia Research Institute and Anne Rowling Regenerative Neurology Clinic.
J.K. Rowling founded the clinic in 2013, in honor of her mother, Anne, who died of multiple sclerosis. She made another major gift in 2019.
The neurons turned out to have defective mitochondria, the “powerhouse” of the cells, which shorted the axon, the all-important long fiber that carries electrical signals from the spinal cord to the muscle. But, when a virus was used to boost mitochondria, the axons were restored to their normal length. This opens the possibility of using drugs already available in clinical trials to treat this condition.
As a neuroscientist whose own dissertation work focused on a motor nucleus of particular interest to both motoneuron disease and sexual differentiation, I am delighted to see this research. Although JK Rowling has received criticism for her position on transgender issues, some of which is, in my opinion, well-deserved, she must be given credit for the great use she has made of her wealth. Research like this doesn’t happen without funding. ALS is currently a death sentence, with most patients dying within two years, and there are few treatments available. The benefits of this work cannot be understated.
Recent Conversation