Top 10 ALS news stories of 2024
Of most interest to readers was news on clinical trials for potential treatments
Throughout 2024, ALS News Today brought our readers daily coverage of the latest in scientific breakthroughs, treatment advances, and clinical trial updates related to amyotrophic lateral sclerosis (ALS).
Here is a list of the top 10 most-read articles published in 2024, along with a brief description. We look forward to continuing to be a trusted resource for the ALS community in 2025.
No. 10 – TPN-101 benefits ALS/FTD patients with C9orf72 gene mutations
Data from a Phase 2a clinical trial showed that Transposon Therapeutics‘ oral therapy candidate TPN-101 reduced disease-related biomarkers and slowed respiratory decline in people with ALS or frontotemporal dementia (FTD) associated with C9orf72 gene mutations. TPN-101 is designed to block an enzyme involved in activating pieces of ancient viral DNA that are believed to contribute to neurodegeneration in ALS and FTD. Based on the interim results after six months, and other findings from the trial, the developer indicated its plans to advance the therapy to a registration Phase 3 study for C9orf72-related ALS.
No. 9 – IGFBP7 gene mutation linked to rare cases of ALS reversal
U.S. researchers identified a mutation in the IGFBP7 gene that’s associated with very rare reversals in ALS disease progression. Typically, as ALS is a progressive disease, its symptoms continuously get worse. However, in a minority of patients, the signs and symptoms of ALS ease or disappear over time. To learn more, the researchers analyzed genetic data from 22 people who had experienced ALS reversal and compared it with data from patients with a typical disease trajectory. A particular mutation in the IGFBP7 gene was associated with up to a 12 times greater likelihood of ALS reversal. The mutation results in lesser production of the gene’s protein product, so researchers believe — though more research still is needed — that inhibiting the protein or the pathway it participates in may be of therapeutic benefit in ALS.
No. 8 – COYA 302 immune-modulating therapy shows promise in small early trial
Coya Therapeutics’ investigational therapy COYA 302 was found to slow ALS disease progression and lower levels of disease-related biomarkers in a small Phase 1 clinical trial. COYA 302 is a combination of an immune signaling molecule called interleukin-2 and a fusion protein called CTLA4-Ig that together are intended to ease inflammation that’s believed to contribute to neurodegeneration in ALS. The clinical trial involved four ALS patients who received injections of COYA 302 over nearly a year. The results showed the treatment was well tolerated and generally slowed patients’ functional declines relative to the trial’s start. Improvements in relevant biomarkers also were observed. Based on the study’s findings, Coya indicated plans to conduct a placebo-controlled trial.
No. 7 – FDA clears Phase 1/2 trial of once-daily SPG302 for ALS
The U.S. Food and Drug Administration gave Spinogenix the green light to launch a Phase 1/2 clinical trial to test its oral treatment candidate SPG302 in ALS patients. The planned trial aims to evaluate the once-daily treatment’s safety, tolerability, and pharmacological properties. SPG302 is designed to trigger the formation of synapses, which are the junctions through which nerve cells communicate — the loss of which is believed to contribute to ALS symptoms. The experimental therapy has demonstrated an ability to regenerate synapses and improve motor and cognitive function in animal models of ALS. It’s now being tested in another Phase 1/2 trial in Australia.
No. 6 – High-dose vitamin B12 approved in Japan for treating ALS
Japanese regulators approved an ultra-high dose of mecobalamin — a naturally occurring version of vitamin B12 — for treating ALS. It’s being sold by Eisai under the brand name Rozebalamin. Vitamin B12 is found in foods such as meats, fish, and dairy, and plays a role in supporting nerve cell health. The approval was based largely on data from a Phase 3 trial involving people with early-stage ALS and moderate disease progression. Data showed that Rozebalamin significantly slowed disease progression, by 43% compared with a placebo, when given alone or combined with standard ALS therapies. The treatment also was well tolerated.
No. 5 – Prilenia plans for Phase 3 trial of pridopidine for ALS
Prilenia Therapeutics indicated its plans to launch a Phase 3 clinical trial of pridopidine in ALS patients after promising Phase 2 results in the HEALEY ALS platform trial. Pridopidine intends to slow ALS progression by activating the sigma-1 receptor, a protein involved in processes important for nerve cell function and survival. Data from the pridopidine arm of HEALEY — a pioneering trial seeking ALS treatments over a faster timeframe — showed that while pridopidine failed to slow disease progression across the entire study group, benefits were observed in a subgroup of patients who were still early in the disease course. These benefits included slower declines in functional ability, respiratory function, and quality of life as compared with a placebo, as well as a possible survival benefit. Pridopidine also was associated with speech improvements across all treated patients.
No. 4 – Some recreational activities linked to greater ALS risk
Participating in certain recreational activities, including golfing, was associated with an increased risk of developing ALS in a study by U.S. researchers. Over the years, scientists have identified environmental risk factors for ALS, including workplace exposures to things like toxic chemicals and pesticides. In this study, researchers surveyed 400 ALS patients and nearly 300 people without the disease to learn more about how recreational activities might influence ALS risk. Several activities, including gardening and woodworking, as well as golfing, were significantly associated with ALS, especially among men. The researchers indicated that more work is needed to validate their findings and to better understand how these activities raise ALS risk.
No. 3 – Amylyx considers discontinuing Relyvrio after trial failure
After Relyvrio (sodium phenylbutyrate and taurursodiol) failed to slow disease progression for ALS patients in a Phase 3 clinical trial, developer Amylyx Pharmaceuticals paused its promotion of the therapy and eventually discontinued it altogether. The therapy had initially been approved in the U.S. and Canada based on Phase 2 trial data showing it could slow functional declines in people with recently diagnosed and rapidly progressing ALS. That finding, however, could not be replicated in the Phase 3 study. The treatment also failed to meet other secondary trial goals related to quality of life, lung function, and survival. Amylyx continues to develop Relyvrio for other neurodegenerative conditions.
No. 2 – Early trial data show promise of vet medication monepantel for ALS
Monepantel, a repurposed veterinary medication that’s now named NUZ-001, was found to slow disease progression for people with ALS or motor neuron disease (MND) in a small Phase 1 clinical trial. Used in veterinary practices as a deworming agent, the therapy is also able to block a signaling pathway that scientists believe could help clear the toxic protein clumps that contribute to ALS progression. Findings from the trial, which involved 12 adults with ALS or MND, showed that daily oral treatment was well tolerated and slowed the rate of disease progression by up to 58% relative to an external group of untreated ALS patients. These results and other long-term findings prompted developer PharmAust — now Neurizon Therapeutics — to plan for the launch of a Phase 2/3 study, which will run as part of the HEALEY ALS trial.
No. 1 – PrimeC prolongs patient survival without complications in trial
The most-read story on ALS News Today in 2024 reported that the ALS therapy candidate PrimeC extended complication-free survival and improved quality of life relative to a placebo among patients in a Phase 2b trial. PrimeC contains a fixed-dose combination of two agents that together are expected to block processes believed to drive ALS progression. Top-line trial data showed that the treatment slowed disease progression in patients who adhered to the trial’s established rules. This analysis demonstrated that PrimeC extended complication-free survival — survival without respiratory failure, ALS-related hospitalizations, or advances in disease stage — by 53% compared with a placebo. Treatment also was associated with positive effects on physical and mental aspects of quality of life. Developer NeuroSense has since met with U.S. regulators to discuss plans for a Phase 3 clinical trial, and is in the process of seeking early approval for PrimeC in Canada.
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At ALS News Today, we hope our reporting throughout 2024 had a meaningful impact on those affected by ALS. We look forward to serving the ALS community in 2025, and wish all of our readers a very happy new year!
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