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Amyotrophic Lateral Sclerosis (ALS) is a devastating neurological disorder. Despite investments and dozens of experimental trials, few ALS interventions currently exist. Of the ALS treatment options available, two are currently FDA approved. And these medications have little benefit, extending life only a few months in most cases. However, given that the average time from diagnosis to death is 2.5 years, months matter. And because ALS affects many people in middle age, significant efforts are being made to find better ALS therapies.

With this in mind, the pharmaceutical company Amylyx announced some positive news recently regarding its new AL treatment drug. The drug, AMX0035, has been shown in Phase II/III trials to increase longevity in ALS patients by 6.5 months on average. While this does not sound impressive, it is notable that AMX0035 was administered in combination with existing FDA approved ALS therapies. Therefore, Amylyx’s medication could be an additional add-on drug that could benefit patients soon.

“In addition to the significant functional benefit previously reported, people taking AMX0035 appear to live several months longer. That is a very meaningful benefit for people affected by this devastating, fatal disease.” – Neil Thakur, Chief Mission Officer of the A.L.S. Association

Amylyx’s New ALS Treatment

The founders of Amylyx are two former students of Brown University. Together, they came up with the idea that a two-drug combination ALS treatment might offer some advantages. The two drugs include sodium phenylbutyrate and taururosodiol, which are used in other existing conditions. The former is used in pediatrics for children with urea cycle disorders. The latter is used to dissolve gallstones. But their effect is ALS is completely different.

A syringe, some pills and the letters ALS
ALS treatments are few and far between, but innovation is breeding hope.

Sodium phenylbutyrate works by reducing the stress on a cell’s endoplasmic reticulum. This organelle is responsible for making cell proteins, and it is routinely damaged in ALS. Taururosodiol, on the other hand, is used to improve cellular mitochondrial dysfunction. This is also commonly present in neurons affected by ALS. In theory, Amylyx’s cofounders believed this combination might delay cell death and offer a new strategy for ALS therapies. And preliminary evidence seems to support this.

The recent trial involved a total of 137 patients who had been diagnosed with ALS within the last 18 months. Half of the patients received AMX0035 while the others received placebo. The initial administration lasted 24 weeks with some patients taking up to 30 weeks. The researchers then followed all the patients for up to 3 years. At the end of the study, it was evident that the ones taking the ALS treatment lived over half a year longer. Based on these results, the researchers are seeking expedited FDA approval or at least compassionate use approval.

“The commitment of so many people with ALS to this research over the course of nearly three years, especially when weighing just how valuable time is for this patient community, has led us to this critical development milestone where we can now reimagine outcomes for ALS.” -Justin Klee, Co-CEO and Co-founder, Amylyx

Amylyx’s Drug Potentially First of Several New ALS Therapies

While Amylyx offers a potential new options of ALS treatment for patients, it appears others are soon coming. Several ongoing clinical trials of new agents offer some hope that better ALS therapies on the horizon. And what’s notable is that several work by completely different mechanisms. It may very well be likely that ALS therapies in the future will consist of a cocktail of medicines. The following are current research studies that should provide results within the coming year.

  • Biogen – The current clinical trial being conducted by Biogen is testing a drug called Toferson. This hopeful ALS treatment works by inhibiting Superoxide Dismutase (SOD), which is involved in free radical inflammation. The current trial involves 183 participants, and results are expected in the second half of 2021.
  • Brainstorm Cell Therapeutics – One of the more intriguing possible ALS therapies may soon involve cellular therapies. Brainstem Cell Therapeutics is currently conducting a trial involving 261 ALS patients of its drug NurOwn Program One. And perhaps the best news is that top line data from the study is expected next month.
  • Alexion – Given that targeted cellular inflammation plays a major role in the disease, some companies are exploring anti-inflammatory agents. Alexion has a current trial underway of Ultomiris, its anti-complement monoclonal antibody factor. This rather large trial involves 354 patients with results expected in 2022.
  • Clene Nanomedicine – In many health areas, nanomedicine is being considered in the development of novel therapies. The same is true for ALS. Clene Nanomedicine is conducting a research trial of its potential ALS treatment CNM-Au8. The drug consists of gold nanocrystals, and 180 patients are currently under study. The results of this trial should be available in spring of 2021.
  • Orphazyme – Other pharmaceutical companies are exploring SOD compounds as ALS therapies as well. Orphazyme’s current trial is testing its drug Arimoclomol Citrate among 231 ALS patients. Its compound is described as a SOD chaperone molecule that may also reduce cellular inflammatory damage. The study’s results should be published in the first half of 2021.

“We’re thrilled to see the pipeline as full as it is now.” – Joshua Cohen, Co-founder and Co-CEO, Amylyx

Advanced Technology Offering New Hope

For many years, the availability of new ALS therapies has been scant. Riluzole and Edaravone have been the only FDA approved treatments for some time. But fortunately, it appears that a number of new medications may soon be approved for ALS treatment. The reason for the increase in clinical trials relates largely in part to newer technologies. Nanotechnology and cellular medicine offer new avenues to develop ALS treatment compounds. The same is true for many evolving advances in immune-technologies. While Amylyx’s new ALS treatment is exciting, its effects are still modest. But the progress made signals that important discoveries are around the corner. And this holds great promise for the future of ALS management.

 

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