Gene therapy shows promise in repairing damaged brain tissue from strokes.

From the NIH Director’s Blog by Dr. Francis Collins.

It’s a race against time when someone suffers a stroke caused by a blockage of a blood vessel supplying the brain. Unless clot-busting treatment is given within a few hours after symptoms appear, vast numbers of the brain’s neurons die, often leading to paralysis or other disabilities. It would be great to have a way to replace those lost neurons. Thanks to gene therapy, some encouraging strides are now being made.

In a recent study in Molecular Therapy, researchers reported that, in their mouse and rat models of ischemic stroke, gene therapy could actually convert the brain’s support cells into new, fully functional neurons.¹ Even better, after gaining the new neurons, the animals had improved motor and memory skills.

Researchers have developed a method to recreate the formation of Lewy bodies in human neurons, shedding light on the essential roles of alpha-synuclein and immune responses in their development. This breakthrough offers new insights into Parkinson’s disease, showing that Lewy bodies form only under specific conditions and highlighting the potential…

A widely available and inexpensive antidepressant drug may soon save lives from an altogether different kind of disease.

The growth of the most aggressive and deadly brain cancer, glioblastoma, was effectively suppressed in both ex vivo human tissue samples and in living mice by an FDA approved serotonin modulator currently used to treat major depression.

It’s not a cure, but it may offer some relief and constitute an effective part of a treatment regime for glioblastoma patients. Human clinical trials are the next step; patients are cautioned against self-medicating at this stage.