Ice Bucket Challenge Helps Fund Potential ALS Medication – GNSOTD Wed, Sept 16th 2020

The internet has featured many challenges over various platforms, several of which have been for charitable causes, one of the most well known is the Ice Bucket Challenge that raised more than $200 million worldwide for ALS research. Recently it appears that that challenge may pay off.

An experimental medication has begun testing and shows that it may slow the progression of amyotrophic lateral sclerosis, or ALS. The research was supported in part by donations from the Ice Bucket Challenge.

The drug is not a cure, but it may help slow the inexorable disability caused by ALS, which rapidly destroys the nerve cells that control the muscles that allow us to move, speak, eat and even breathe.

“Patients keep telling me their No. 1 goal is to be able to retain physical function for as long as possible,” said the study’s lead author, Dr. Sabrina Paganoni, a neuromuscular specialist at Massachusetts General Hospital’s Sean M. Healey & AMG Center for ALS. “They want to be able to continue to walk and to use their hands.”

More than 200,000 people around the world, about 20,000 people in the U.S., and around 3,000 Canadians have ALS at any given time, according to the ALS Association and the ALS Society of Canada. It usually strikes between the ages of 40 and 70. Once symptoms set in, life expectancy is two to six years, on average.

To test the effectiveness of the two-drug combination, the researchers recruited 137 ALS patients who had become symptomatic within the previous 18 months. About two-thirds of the patients (89) received the drug, while the remaining third were given a placebo.

Participants were evaluated on a scale of 0 to 48, measuring the disabilities caused by the disease.

“By the time they entered the trial, on average, patients had already lost 12 points. Their baseline score was about 36, on average,” Paganoni said.

During the six months of the study, patients taking the medication lost an average of 2.32 points less than those receiving placebos, a 25 percent better functional outcome.

“A 2- to 3-point change can mean the difference between being able to do an activity independently or with an assistance device,” Paganoni said. And that is very much worth being your Good News Story of the Day. Read the story in full and watch the video here.

The next step is a phase-2 double blind study. In a double-blind study, neither the patients nor the researchers know who is receiving the drug. If a phase 2 study generates positive results, the FDA typically requires a larger and longer phase 3 trial.

Story and Image from NBC News.

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