Promising new ALS treatment funded by Ice Bucket Challenge donations shows significant slowing in phase 2 trial


An experimental medication may slow the progression of amyotrophic lateral sclerosis, or ALS, researchers reported Wednesday. The research was supported in part by donations from the Ice Bucket Challenge, the social media sensation that raised more than $200 million worldwide.

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.”

About 20,000 people in the U.S. have ALS at any given time, according to the ALS Association. It usually strikes between the ages of 40 and 70. Once symptoms set in, life expectancy is two to six years, on average.

The treatment studied by Paganoni and her colleagues targets two cellular structures damaged by the disease: the mitochondria, which are the cells’ power plants, and the endoplasmic reticulum, the cellular dump trucks that cart away waste that can gunk up the cells’ machinery.

The multicenter, randomized, double-blind study is the second step — a phase 2 trial — in a three-step process required by the Food and Drug Administration for drug approval. 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.

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. “Each question addresses a specific domain of function and is scored on a scale from zero to four.”

For example, for walking:

4 = Normal

3 = Early ambulation difficulties

2 = Walks with assistance

1 = Nonambulatory functional movement only

0 = No purposeful leg movement

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. US SPREAD GOOD NEWS!

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By Justgivemepositivenews Team