Throughout 2017, ALS News Today brought you daily coverage of amyotrophic lateral sclerosis (ALS)-related advocacy events, clinical studies, and the latest research updates. We have compiled a top 10 list of the most-read ALS news stories of that year, with a brief description of what made them interesting and relevant to patients, family members, and caregivers.
Worldwide Clinical Trials was selected in July as the clinical research organization (CRO) to run a Phase 3 study of NurOwn (NCT03280056), an investigational stem-cell therapy to possibly treat ALS. NurOwn is based on the collection and expansion of mesenchymal stem cells (MSCs) harvested from patients. In December, BrainStorm Cell Therapeutics, the maker of NurOwn, announced that the first patient had enrolled in the Phase 3 trial.
In April, Genervon Biopharmaceuticals published the results of a Phase 2a clinical trial of GM604 in ALS patients, showing that treatment with GM6 improved clinical and functional measures in ALSFRS-R (a scale that measures disease status) and FVC (a measure of respiratory capacity). The drug also improved several ALS biomarkers. GM604 is a synthetic drug containing six amino acids that acts on multiple pathways involved in the development of neurons and the human nervous system during the embryonic stage.
Before selecting Worldwide Clinical Trials to run its Phase 3 study of NurOwn, BrainStorm Cell Therapeutics announced positive top-line results of its Phase 2 studies in April. The Phase 2 data mainly showed that NurOwn was safe and well-tolerated. Treatment achieved multiple efficacy endpoints in 48 ALS patients, including the ability to halt or reverse disease progression over the course of a six-month follow-up period. These results were the grounds of the U.S. Food and Drug Administration (FDA) giving permission to start the pivotal Phase 3 trial.
While limb-onset ALS patients show brain damage mainly in regions linked to movement, patients with bulbar-onset ALS have more widespread brain tissue loss, researchers found in a study published in January. The findings may help explain why bulbar-onset patients fare worse, even if fewer time has passed since diagnosis. Researchers suggest that the method used to assess brain damage may be a valuable tool for predicting the disease’s progression and prognosis.
In a review published in February, researchers detailed their conclusions regarding the use of cannabis-derived compounds to treat ALS. At the time, the only available therapy for ALS patients was Rilutek (riluzole) – Radicava (edaravone) was approved in May – and researchers noted its limited therapeutic value and moderate benefits. To help patients explore alternatives, they analyzed the use of cannabinoids, providing a short but concise overview of their efficacy and mechanism of action.
“Radicava (edaravone) is a minor miracle for some ALS patients,” said Stephen Byer, co-director of ALS Worldwide in a telephone interview with ALS News Today. An interview with Byer, conducted in June, was one of our most viewed articles of the year. Radicava, an infusion treatment, became the first ALS therapy in 22 years to receive U.S. Food and Drug Administration (FDA) approval one month earlier. Byer and his organization were helping several patients at the time of the interview to access the treatment, and offered his opinion as to its benefits.
In May, AB Science presented the results of a Phase 3 clinical study of masitinib, administered at 4.5 mg/kg/day in ALS patients, that showed it to be effective at improving physical function. The trial compared masitinib with riluzole (the active ingredient in Rilutek) against placebo plus riluzole. Results, presented at the 2017 European Network for the Cure of ALS annual meeting, in Ljubljana, Slovenia, showed that masitinib slowed patients’ loss of function, improved progression-free survival, and reduced the decline in quality of life.
AB Science also presented findings on masitinib at the 27th International Symposium on ALS/MND in Dublin, reported in a January article, with data demonstrating that masitinib protects muscles and nerves against damage from ALS. The findings relate to the treatment’s ability to reduce inflammation in immune-system cells (such as mast cells and microglia) that are thought to promote the development of ALS. These data served as the grounds for further studies of masitinib.
AB Science continued with the good news in March, announcing that masitinib met its primary Phase 2/3 clinical trial goals of improving the ability of ALS patients to function, especially of note in the Phase 3 study that involved almost 400 patients and extended treatment for 48 weeks. Olivier Hermine, president of AB Science’s scientific committee, said in a comment of these findings that the most impressive achievement could well be the fact that masitinib treatment showed a significant difference in progression free survival with respect to patients given placebo, suggesting a clear benefit in favor of masitinib.
Finally, the most widely read news of this past year was the approval of Radicava (edaravone). After 22 years without any newly approved therapies, the May 5 FDA decision, hailed by physicians and disease advocates, brought renewed hope to the ALS community. The approval was based on finding in a Phase 3 clinical trial in Japan that demonstrated the treatment’s ability to slow decline in daily function in ALS patients. According to the ALS Association, the treatment’s list price in the United States is $1,000 per infusion, or about $146,000 annually.
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We wish all our readers a happy and inspiring 2018.