Top 10 ALS stories of 2023
Cholesterol levels, gene editing, and an herbal patch as therapy among coverage
Here are the year’s top 10 most-read articles, each with a brief description. We’re excited to remain a dependable resource for the ALS community in 2024.
No. 10 – Omega-3 fatty acid slows ALS progression, extends survival
A diet rich in omega-3 fatty acids has been tied to a lower risk of developing ALS, but whether it also might affect the rate of disease progression is not known. Researchers at Harvard found that higher blood levels of alpha-linolenic acid (ALA) — an omega-3 fatty acid found in flaxseed, walnuts, chia, canola, and soybean oils — linked with a slower disease progression and a 50% lower risk of death.
Two other fatty acids also were tied to a lower mortality risk. This discovery suggests that adding to the diet more foods containing these fatty acids could be beneficial for ALS patients. Researchers hope to confirm these findings in a clinical trial.
No. 9 – Further questions set back European approval of AMX0035
Amylyx Pharmaceuticals’ application seeking the approval of AMX0035 in treating ALS patients across Europe faced a second round of questions from the Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency. The committee still had major objections to AMX0035 that needed to be addressed, after a scientific advisory group meeting of experts provided recommendations based on current data.
The therapy is approved in the U.S. and available under the brand name Relyvrio (sodium phenylbutyrate and taurursodiol), and approved in Canada as Albrioza. CHMP, however, later confirmed that it remained opposed to AMX0035’s approval in Europe.
No. 8 – Masitinib as add-on therapy seen to benefit mild to moderate ALS
Masitinib is an experimental ALS therapy that intends to slow disease progression and ease ALS symptoms by blocking certain enzymes needed by immune cells thought to drive inflammation and neurodegeneration in ALS. An analysis of AB Science’s completed AB10015 Phase 2/3 clinical trial (NCT02588677) showed that treatment with masitinib as an add-on to Rilutek (riluzole) benefited most of its ALS patients with mild to moderate disease severity.
Although the 48-week study failed to show better survival rates overall with masitinib compared with a placebo, treated patients with moderate ALS experienced a 25-month extension in survival. The combination treatment also significantly slowed disease progression, improved lung function, and enhanced life quality for patients who still retained some degree of physical function at the study’s start.
No. 7 – Stem cell transplants seen to extend survival by 4 years
A stem cell transplant using mesenchymal stromal cells (MSCs) taken from bone marrow significantly extended survival for ALS patients beyond their anticipated lifespan, a new analysis of data from two Phase 1 trials, both conducted in the early 2000s, showed.
Treated patients lived about 118 months, or nearly 10 years, without needing permanent breathing support, surpassing a predicted survival time of around 70 months by about four years. At the time of the analysis, four of the trials’ 19 total patients were alive, including one still living without any need for breathing or feeding assistance at 303 months — more than 20 years later.
No. 6 – Neurologists frustrated with current ALS treatments
Neurologists, while quick to prescribe their patients new treatments showing a potential to delay ALS progression in clinical trials, often remain frustrated with current available therapies. Fewer than 20% of these doctors responding to a survey expressed full satisfaction with responses to treatments now on the market.
This so-called first market landscape study on ALS, conducted by Spherix Global Insights, involved 103 neurologists. It found that many already were prescribing Relyvrio and Qalsody (tofersen), two ALS medications recently approved in the U.S. Spherix data also showed that neurologists lacked awareness of experimental ALS treatments, possibly because many such therapies fail to win approval.
No. 5 – COYA 302 seen to slow ALS progression in proof-of-concept trial
COYA 302, an experimental immune-modulating therapy under development by Coya Therapeutics, markedly slowed disease progression among four ALS patients treated in a small, 48-week proof-of-concept clinical trial. Data also showed a treatment-related increase in the activity of regulatory T-cells, an anti-inflammatory immune cell, demonstrating that the therapy worked as designed.
Coya intends to open larger ALS trials of the therapy, administered via a subcutaneous (under-the-skin) injection. The company announced plans to file early this year a request for clearance to launch a Phase 2 clinical study.
No. 4 – Tofersen, now Qalsody, approved by the FDA for SOD1-ALS
In April, the U.S. Food and Drug Administration conditionally approved Biogen’s Qalsody (tofersen) for treating ALS linked to SOD1 gene mutations. The decision marked the country’s first conditional approval for an ALS therapy.
Qalsody’s approval relied on biomarker data from the VALOR Phase 1/2/3 clinical trial (NCT02623699) and its extension study (NCT03070119). Data demonstrated significant, treatment-related reductions in blood neurofilament light chain (NfL) levels, indicating a decline in nerve cell damage. Exploratory analyses also suggested the treatment’s use could slow ALS progression, particularly in people with rapidly progressing disease.
No. 3 – Chinese herbal skin patch, as add-on therapy, slows progression
A skin patch containing a mixture of Chinese herbs called Ji Wu Li (JWL), combined with standard treatments, significantly slowed ALS progression in a placebo-controlled clinical trial involving 120 adult patients. Benefits were seen after eight weeks of treatment and sustained for up to 20 weeks, or about five months.
Administered via a self-adhesive patch placed on the patient’s upper back, the transdermal, or on-the-skin, therapy may provide a way to deliver a concentrated formulation of JWL without swallowing, which can be challenging for some with ALS. Use of the patch, worn for six consecutive days each week and for six hours each day, also was reported to slow quality of life declines and ease weight loss.
No. 2 – Gene editing ‘cuts out’ ALS-linked mutation in early study
In mouse models and human cells, the gene-editing tool CRISPR/Cas9 removed the repeat expansion defect in the C9ORF72 gene, the most common genetic cause of ALS, scientists in a proof-of concept study reported.
While not interfering with the production of the C9ORF72 protein, such gene editing notably lowered levels of defective RNA molecules and small proteins associated with nerve cell death in ALS. RNA acts as the intermediary molecule derived from a DNA sequence, functioning as a template for protein production.
No. 1 – Poorer ALS survival linked with higher ‘good’ cholesterol levels
A Netherlands-based population review study found that elevated levels of high-density lipoprotein, also known as “good” cholesterol, significantly correlated with a reduced survival rate in people with ALS. Individuals in more advanced disease stages also were seen to have lower levels of total cholesterol and low-density lipoprotein, commonly referred to as “bad” cholesterol.
Its researchers acknowledged their study contrasts with previous research suggesting that a lower ALS risk was tied to higher levels of good cholesterol. Still, its findings contribute to insights into fat-like lipid metabolism and ALS severity, as their levels appear to be related to disease progression.
At ALS News Today, we hope our stories and reporting throughout 2023 positively impacted the lives of those affected by ALS. We look forward to serving the community further in 2024, seeking to provide ongoing support and information.
A very happy new year to all our readers!