Donations from the Ice Bucket Challenge helped fund some of the study

According to a new study, a new therapy or ALS treatment options might help halt the course of ALS.

According to the ALS Association, around 20,000 people in the United States have ALS at any given moment. Between the ages of 40 and 70, it is most common. On average, life expectancy is two to six years once symptoms appear. The therapy Paganoni and her colleagues investigated targets two cellular components that are harmed by the disease: the mitochondria, which are the cells' power generators, and the endoplasmic reticulum, which serves as a biological dump truck for trash that may clog up the cells' machinery.

The multicenter, randomized, double-blind study is the second of three steps required by the Food and Medication Administration for drug approval, known as a phase 2 trial. Neither the patients nor the researchers know who is getting the medication in a double-blind trial. The FDA generally needs a larger and longer phase 3 trial if a phase 2 research yields good findings.

The researchers gathered 137 ALS patients who had been symptomatic during the preceding 18 months to assess the efficiency of the two-drug combination. The medication was given to almost two-thirds of the patients (89), while the other third got a placebo. The disability caused by the illness was measured on a scale of 0 to 48 for each participant.

"Patients had dropped an average of 12 points by the time they entered the study. On average, their baseline score was around 36 "Paganoni said. "Each question is assessed on a scale of zero to four and addresses a distinct domain of function." In terms of time of death, tracheostomy, or permanent intubation, or hospitalization, the experiment found no difference between the drug and the placebo. But that might be due to the fact that it only lasted six months.

With the positive findings of the study, the ALS Association aims to persuade the FDA to grant other patients' access to the medication even before the results of the phase 3 trial are released. The ALS Association's chief mission officer, Neil Thakur, stated, "It's quite uncommon for an ALS clinical trial to meet its primary goal, so we're really thrilled about it." "It's the difference between being able to feed oneself vs. being fed or having to use a wheelchair against not having to use one." Experts in ALS advised against moving forward without further information.