A bucket of ice cold water rains on your head while friends challenge you and film this cold maneuver to post on social media. The shock … the cold … the soaking.
Who doesn’t remember the 2014 Ice Bucket Challenge that raised $ 115 million in donations for ALS? The unique challenge of fundraising has helped bring researchers closer to breakthroughs in the diagnosis, treatment, and eventual cure of amyotrophic lateral sclerosis or Lou Gehrig’s disease, a degenerative disease with no known cure.
There are currently only two drugs approved to treat ALS, a progressive nervous system disorder that affects nerve cells in the brain and spinal cord, causing loss of muscle control and affecting a person’s ability to speak, move, eat and eat to breathe. One drug, riluzole, has been available for 25 years and extends its lifespan by an average of a few months. The second drug approved in 2017 is edaravone, which has been shown to help patients stay in their condition for longer.
“[Edaravone’s] The effect is relatively small, but is believed to slow the progression of the disease for those who are in the early stages of the disease, “social worker Leigh Stephens, a psychosocial advisor for the ALS Society of Quebec, told Medical Daily.
Research is currently underway on the third drug, AMX0035, a neuroprotective research therapy designed to reduce motor neuron death and dysfunction.
AMX0035 is in phase 2 clinical trials in people with the disease. The study is being conducted by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Amylyx Pharmaceuticals, Inc., the company that makes the drugs. The results are encouraging when it comes to slowing the progression of ALS and extending patient survival.
“Research in ALS has been booming since the Ice Bucket Challenge 2014,” said Stephens. “Families are sometimes frustrated with the time it takes to see the fruits of this labor.”
What is AMX0035?
The researchers looked at 137 participants, ages 18 to 80, who had been symptomatic of the disease for less than 18 months. They were randomly given either AMX0035 or a placebo over a six month period.
Patients who completed the study were then eligible for an open-label extension, with all patients receiving AMX0035 to further test long-term effectiveness. The results of the Phase 2 efficacy study of AMX0035 were published in the journal Muscle and Nerve.
The study aimed to measure a change in ALSFRS-R (the revised ALS functional rating scale) compared to the placebo group. The researchers also measured muscle strength, vital lung capacity, tracheostomy needs, hospital stays, and survival. About 5% of participants had diarrhea and abdominal pain, the most common side effects, which subsided over time. Ninety-eight of the 137 participants completed the study.
AMX0035 is not brand new. It’s a combination of two drugs already in use, sodium phenylbutyrate and taurursodiol, that target oxidative stress in the mitochondria of nerve cells to prevent neurodegeneration.
Results offer hope
The researchers found that AMX0035 slowed the progression of ALS disease over six months and had positive effects on daily life such as: B. Walking, talking, using utensils and swallowing food. Participants randomized to receive the drug lived an average of 6.5 months longer than those who received the placebo.
Study researchers view the results as an important step in the treatment and eventual cure of ALS, as AMX0035 has been linked to both longer survival and improved functionality.
“[This research] brings hope to many, if only to gain time, to increase the chances of being still alive for others to make advances in disease treatment, and hopefully in healing, ”said Ms. Stephens. “It can encourage others to refer to a follow-up study.”
Usually, drugs will become available after phase 3 trials. There are currently 57 phase 3 studies for ALS in progress. Some are currently recruiting participants and some are not. More information can be found here.
Jennifer Nelson is a Florida-based health writer who also writes on health and wellness for AARP, PBS ‘Next Avenue, Shondaland, and others.