The FDA has approved a novel treatment for Lou Gehrig’s disease, a rare form of Amyotrophic Lateral Sclerosis (ALS).
The injectable medicine is now available for individuals with a rare mutation that accounts for less than 500 cases of ALS in the US.
How Qalsody Works?
Qalsody (tofersen) is only intended to benefit the 2% of ALS patients who have a highly particular mutation called SOD1.
The medicine may decrease muscle degeneration in people who qualify by inhibiting the production of SOD1 mRNA, the genetic information that informs the body how to build proteins.
According to the Food and Medication Administration, the medication decreased plasma neurofilament light (NfL), a biomarker of axonal (nerve) injury and neurodegeneration.
Dr. James Berry, chief of the division of ALS and motor neuron diseases at Massachusetts General Hospital, says that the SOD1-mutant form of ALS can be one of the most aggressive types of the disease.
This medicine is currently being tested on ALS patients at MGH, where the gene was first found in the 1990s.
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Adverse reactions to Qalsody
Soreness, weariness, joint and muscular soreness, and increased white blood cells in cerebrospinal fluid were the most often reported adverse effects when studying the safety of Qalsody.
The medication was granted fast-track status by the FDA and awarded conditional clearance. Manufacturer Biogen still has to conduct larger-scale tests to confirm the drug’s efficacy.
Dr. Colin Quinn, a neurologist at Penn Medicine, and others are hopeful that this medicine can lead to treatments for individuals with sporadic ALS, which develops unexpectedly rather than as a result of a genetic mutation, even though it is only for a subset of ALS patients.
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