ALS Drug Reverses Symptoms in Patients for First Time

Amanda Sifford could barely walk 10 steps without gasping for air. Her lung function had crashed from 86 percent to just 48 percent in five months, and the ALS that killed 14 family members was stealing her ability to breathe.

The 58-year-old school psychologist from Cape Coral, Florida, had her affairs in order. Her doctors at the University of Miami worried she had months left to live.

Then in May 2023, the FDA approved tofersen, the first therapy for genetic ALS. The drug targets a rare form of the disease caused by a mutation in the SOD1 gene, affecting about 2 percent of ALS patients.

Sifford began receiving monthly infusions of the drug into her spinal canal. Progress came slowly, and she faced setbacks including spinal inflammation that forced a temporary pause in treatment.

But something remarkable happened. Her breathing, muscle strength, and mobility stopped declining and then improved. By January 2026, her lung capacity test reached 63 percent, the highest since starting treatment.

Why This Inspires

For a disease that always causes continual decline and death, any improvement feels like a miracle. Rickey Malloy, 44, from Hillsboro, Illinois, started tofersen weeks after his diagnosis in July 2023 when he was limping and needed help climbing stairs. Most of his symptoms have now stabilized or improved, and doctors cleared him for knee replacement surgery and physical therapy trials.

A three-year study following 46 patients found nearly 20 percent showed improvement in breathing, strength, and function. The remaining 75 percent declined more slowly than expected, especially those who started treatment earlier.

Dr. Timothy Miller, the trial's principal investigator at WashU Medicine in St. Louis, says patients who received tofersen immediately lived about three years longer than those who started on placebo. For fast-progressing ALS patients who typically die within a year, this represents transformative change.

The drug works by reducing levels of toxic SOD1 protein that damages neurons. While it only helps a small fraction of ALS patients, it proves something experts once thought impossible: this devastating disease is treatable.

Researchers are now exploring whether similar approaches could work for other genetic forms of ALS and eventually for the 90 percent of cases with no known genetic cause.

For Sifford, each breathing test brings tears of joy and gratitude from both her and her medical team. The woman who once prepared for death is now planning her future.