Amyotrophic lateral sclerosis or A.L.S. is a neurodegenerative disease that causes the loss of muscle control. The illness affects the nerve cells in the brain and the spinal cord, determining the progressive loss of most motor functions and resulting in difficulties walking, chewing, swallowing and breathing.
Scientists from a small company in Massachusetts, who were studying this disease, have come up with a cure in the form of a treatment called Albrioza. The drug has been approved for being used in Canada, on the condition that further evidence of the drug’s effectiveness will be provided in the future. Furthermore, The New York Times wrote that the experimental treatment is also under review in by the F.D.A. in the United States, giving hope to patients suffering from A.L.S. The F.D.A. is supposed to issue its decision on September 29th.
Even if the perspective is promising, patients must be aware of the fact that Albrioza has been approved by Health Canada under the program called Notice of Compliance with Conditions. This program streamlines the approval of certain drugs meant for the treatment of severe diseases, but without sufficient data to prove their efficiency. While companies are required to file some evidence that their treatment works, the program allows them to make the therapies available to patients under the obligation of continuing their studies and providing regular safety reports.
In the United States, there are currently only two FDA-authorized drugs, reason for which the pressure to approve the newly found therapy is increasing. According to The New York Times, “last month, 38 doctors who treat A.L.S. patients sent a letter to the F.D.A. urging approval. The A.L.S. Association said its campaign for approval had in recent weeks generated more than 6,000 emails asking the agency to greenlight the drug.”
At the moment, a Phase 3 clinical trial is taking place, which is set to end in 2024. According to Healthline, “the purpose of phase III is to evaluate how the new medication works in comparison to existing medications for the same condition”. This is meant to test the safety and efficiency of the treatment with Albrioza on patients with A.L.S. The clinical trials usually involve dividing the participants into two groups, one of which will receive the standard treatment, while the other will be given the experimental treatment.