ST. LOUIS – Research taking place at Washington University’s School of Medicine is giving hope to those affected by amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
The experimental drug tofersen shows signs of slowing down the disease by lowering the levels of a disease-causing protein in people with an inherited form of ALS.
News of the breakthrough was released Wednesday afternoon.
Two clinical trial phases have been completed and a third phase is now underway.
“Hopefully, that will continue to show that we’re slowing down the disease,” said Dr. Timothy Miller, Washington University School of Medicine.
Miller said patients with an inherited form of ALS caused by mutations of a certain gene could someday have a treatment.
“Breaking news like this gives people with ALS the hope that they deserve,” said Maureen Barber Hill, president and CEO of the ALS Association St. Louis Regional Chapter. “We have such heroes that struggle and battle with this disease and it’s not just the person with ALS, it’s the whole family, their whole support system.”
The ongoing research involves a rare form of ALS but could lead to breakthroughs in the fight against other forms of ALS and other debilitating diseases.
“The concept of targeting these gene products is exactly what can be done in a whole host of other neuro degenerative diseases and neurologic diseases,” said Miller.
There are approximately 20,000 people in the US living with ALS, according to Washington University’s School of Medicine. Patients lose the ability to use their muscles and few survive more than 5 years after the diagnosis. Researchers have yet to find a cure.
More information: ALSCenter.wustl.edu/