As reported in Alzforum, could a dietary supplement marketed directly to consumers moonlight as an effective treatment for ALS? It is within the realm of possibility, according to the results of a small Phase 1 pilot study testing EH301 in people with the disease. Essentially the same product as EH301 is sold under the name Basis by New York-based Elysium Health as a supplement to support cellular health.
- In four-month trial, EH301 apparently slowed ALS.
- In open-label extension, the treatment reportedly held off decline at one year.
- EH301 is for sale on the internet as Basis, an unregulated dietary supplement.
In the January issue of the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, researchers led by José Estrela at the University of Valencia, Spain, reported that ALS patients taking EH301 for four months not only stopped getting worse, but even improved, on several clinical endpoints. What’s more, the treatment appeared to stave off decline for up to one year in participants enrolled in an open-label extension. The authors, as well as outside commentators, called for a larger study to evaluate EH301 in ALS.
“It is remarkable and encouraging to see that the investigators report a clinical improvement in the ALS patients, and not just a slowing of progression of this devastating disease,” commented Patrick Weydt of University Clinic Bonn, Germany. “Findings like these urgently require independent confirmation,” Weydt wrote, (full comment below).
Elysium Health co-founder and chief scientist Leonard Guarantee, Massachusetts Institute of Technology, is a co-author on the paper. Guarantee forwarded Alzforum’s request for an interview to the company’s public relations office, which declined it. Estrela originally scheduled an interview but backed out several days later upon company direction.
Weydt and other commenters pointed out several caveats with the trial, including its small size, short duration, and 38 percent dropout rate. Merit Cudkowicz of Massachusetts General Hospital in Boston noted that the data were encouraging, but not definitive. “Because ALS is such a heterogenous disorder, results from small studies can sometimes not bear out when a larger confirmatory study is done,” Cudkowicz wrote, “It would be important to move forward expeditiously, though, for a confirmatory trial in larger groups of participants and for longer duration.” (Full comment below).
EH301 is a combination of pterostilbene (PT)—an analog of resveratrol found in blueberries—and nicotinamide riboside (NR), a precursor to the coenzyme nicotinamide adenine dinucleotide (NAD+). In addition to its pivotal role in the mitochondrial oxidative metabolism that fuels cells, NAD+ drives the activity of sirtuins. The human genome encodes seven sirtuins. They carry out multiple functions, including deacetylation of transcription factors, histones, and other targets, and are involved in processes known to go awry during aging and in neurodegenerative disease, including energy metabolism, inflammation, and DNA repair.