FDA approves ALS treatment for patients with genetic diseases

Ice Bucket Challenge Funded New ALS Treatment Gets FDA Approval

In a recent announcement, the Food and Drug Administration (FDA) approved a new treatment for ALS patients called Topherson, which is a spinal injection that kills a gene causing the disease to progress. This approval comes nearly a decade after the viral online Ice Bucket Challenge brought awareness and funding to ALS research. This new treatment is a significant breakthrough in the fight against ALS, offering hope to those suffering from this incurable disease.

ALS patient, Sarah Wheeler, worked as a full-time nurse in 2018, loved to travel and lead an active lifestyle, but around the same time, strange symptoms began. She began experiencing leg cramps and pain eventually reaching the point where she couldn’t stand on her toes. It took her three years to receive an official diagnosis of ALS. Around 10% of ALS cases are genetic, and of that group, 1-2% have the SOD-1 gene, which is known to cause ALS.

Dr. Jill Dalager Brunner, executive director of the ALS Association Southwest Territory, calls the new treatment a game-changer, giving hope to not only SOD-1 patients. Topherson’s trial with the FDA already began a year ago under the FDA’s charitable use program. While undergoing treatment, Wheeler says that the process is intense, and she has to travel for three hours every month. However, she expressed that the treatment is working, and she can see significant improvements, such as the dimple in her calf disappearing.

The new treatment could be a massive win for ALS patients, potentially slowing down the progression of the disease and increasing life expectancy, which is typically between three to five years. Anyone diagnosed with ALS should have a genetic test to see if they are eligible for this treatment.

To protect themselves, patients should seek information and support from the ALS Association as both Dalager Brunner and Wheeler emphasize that patients should avoid searching Google for treatment information. Patients should connect to resources, find a multidisciplinary ALS clinic, and take advantage of physical therapy, occupational therapy, nutritionist care, respiratory therapy, and speech therapy.

In conclusion, the FDA’s approval of Topherson is a significant milestone for the ALS community and provides hope to ALS patients worldwide. The new treatment is expected to slow down the progression of ALS, and it is also hoped that a cure for the disease would eventually be found.

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