AARP Eye Center
If there’s one thing neuroscientist Kasper Roet is feeling these days, it’s optimism.
QurAlis, the Cambridge, Massachusetts-based biotechnology company he cofounded in 2016, recently ushered two drug candidates for the treatment of amyotrophic lateral sclerosis (ALS) into early-stage clinical trials.
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Though they work in different ways — one increases levels of an important protein, the other activates a potassium channel — both therapies have the same goal: to protect against the deterioration and death of motor nerve cells in the brain and spinal cord. This hallmark of ALS leads to many of its symptoms, including muscle twitches, muscle weakness and difficulty chewing, swallowing and eventually breathing.
If all goes according to plan, the two experimental drugs could help to grow a short list of treatments available for the rare but devastating disease that affects roughly 5,000 Americans each year. There is no cure for ALS, also known as Lou Gehrig’s disease, and most people with it live only three to five years after symptoms appear, according to the National Institute of Neurological Disorders and Stroke.
Researchers hope that will soon change.
“I think we were in a different situation than we’ve been in the past,” says Roet, CEO of QurAlis, which he launched alongside two Harvard stem cell researchers and professors. “We actually understand the disease much better now.”
One thing that’s led to better understanding is the ability to study the disease in a lab dish using adult stem cell models from patients with ALS. This allowed the founders and researchers at QurAlis to pinpoint different drivers of the disease, which in most people occurs at random, and design precision therapies that target the faulty mechanisms and “bring diseased cells to a healthy state,” Roet explains. “This is really what is core for QurAlis,” he says about the company’s approach.
The potential benefits of QurAlis’ medicines could extend beyond the motor symptoms of ALS. About half the genetic mutations that can cause ALS can also cause frontotemporal dementia (FTD) and their biology is linked, Roet says. ALS and FTD are considered a spectrum disorder, and some people on that spectrum can have ALS combined with FTD to varying degrees, according to the ALS Association.
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