A new experimental drug known as NU-9 may be more effective in treating ALS than existing FDA-approved drugs. The corresponding study was published in Nature.
ALS is characterized by fatal paralysis caused by the degeneration of the upper section of motor neurons known as axons, which connect the brain to the spinal cord. For a drug to be effective against ALS, it must thus improve axon outgrowth and health.
In research published last year, scientists found that NU-9 improved two critical factors that lead to ALS in upper motor neurons: protein misfolding and protein aggregation in cells. They further found that neurons in mouse models resembled healthy controls after 60 days of treatment with NU-9.
In the present study, the researchers tested NU-9 on mouse models of ALS again, both in combination with FDA-approved drugs for ALS - riluzole or edaravone, and by itself. They also compared these results to mice treated exclusively with these pre-existing drugs.
In doing so, the researchers found that NU-9 was able to lengthen axons in diseased upper motor neurons in mice. They also found that its therapeutic effects were stronger than those of existing FDA-approved drugs when taken alone. However, they noted that a combination treatment of NU-9 and either pre-existing drug produced the most significant results.
NU-9 is now undergoing animal safety studies to determine dose levels and toxic effects in preparation for clinical trials. Should these prove successful, the researchers hope to start Phase 1 clinical trials in early 2023.
"It is a long process - possibly 10 to 12 years - to discover and bring a new drug to the market," said Richard B. Silverman, the Patrick G. Ryan/Aon Professor at Northwestern University, and one of the study’s authors, "But this drug has us very excited and hopeful about its possibilities to improve the lives of ALS patients, who have been without hope for so long."