ALS Association Partnership to Fund RNS60 Treatment Trial with Biomarkers to Measure Neuroinflammati
The ALS Association, in partnership with ALS Finding a Cure and the Northeast ALS Consortium (NEALS), is pleased to announce $1 million in funding to support a new ALS clinical trial to test the efficacy of the experimental drug RNS60 in reducing inflammation that may hasten the disease process. The trial will be led by Ettore Beghi, M.D., neurologist at the IRCCS Mario Negri Institute for Pharmacological Research in Milan; and Letizia Mazzini, M.D., neurologist at the University Hospital of Novara in Novara, Italy; and Sabrina Paganoni, M.D., Ph.D., neurologist at Massachusetts General Hospital in Boston.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which leads to total paralysis and death, usually within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.
While ALS is not primarily an inflammatory disease, inflammation within the central nervous system is believed to accelerate the disease once it has begun. Thus, treatments to reduce this inflammation may help slow disease progression. In preclinical studies, RNS60 has been shown to have strong immunomodulatory effects, reducing inflammation and protecting motor neurons. This phase II trial is being funded through ALS ACT, a partnership among the three funding entities to promote the use of biomarkers in ALS clinical trials. In the trial, 142 people with ALS will receive either the active drug or a placebo for 24 weeks. Markers of neuroinflammation will be measured in the blood to determine if the treatment is having the intended effect on the immune system. Secondary outcome measures will include clinical measures of disease progression.
“The use of biomarkers in this ALS clinical trial offers us the ability to determine if the drug is doing what we hope it will do in people with ALS, namely reducing inflammation,” commented ALS Association Chief Scientist Lucie Bruijn, Ph.D., MBA. “With that, we can then ask whether reducing inflammation in this way can be beneficial in the disease. Most previous studies in ALS have not had biomarkers available to determine whether the drug was engaging its molecular target. Without that knowledge, it has remained difficult to say whether a drug failed because it didn't hit its target, or whether the target wasn't significant for altering the disease. We are optimistic that the use of biomarkers in this trial will be able to answer that important question.”
“We are very excited to support this well designed phase II trial of RNS60 in people with ALS,” said Merit Cudkowicz, M.D., Chief Medical Officer for ALS Finding a Cure and Chief of Neurology at Massachusetts General Hospital. “There is a great need to support and accelerate bringing new targeted therapies forward for testing in people with ALS. The ALS ACT phase II program is instrumental to funding early phase trials in ALS.”