Spinal-bulbar muscular atrophy (also known as Kennedy's disease) was identified 56 years ago and affects about one in 40,000 Americans. Its relative newness and rarity have made it difficult to find a cure or treatment. But ongoing clinical research is testing medications and interventions that may one day be approved by the U.S. Food and Drug Administration. Phase 2 trials test whether a drug or treatment is effective, while phase 3 trials confirm safety and efficacy in larger populations.

Exercise

A pilot study in 2020 demonstrated the benefits of exercises for aligning posture, activating core muscles, building endurance, improving balance, and addressing rhythmic walking in one person with Kennedy's disease, says Kenneth Fischbeck, MD, FAAN, scientist emeritus at the National Institute of Neurological Disorders and Stroke in Bethesda, MD. Following that result, phase 2 clinical trials are now assessing the impact of structured exercise programs on muscle strength and function.

Clenbuterol

This asthma drug increased walking distance in people with Kennedy's disease in a pilot trial. Now it's being tested in a phase 2 trial to see if the drug can improve muscle stamina and quality of life for people with the condition, says Alejandro Tobon, MD, FAAN, chief of neurology at South Texas Veterans Health Care System in San Antonio.

AJ201

This new drug based on curcumin, a natural compound found in turmeric known for its anti-inflammatory and antioxidant properties, is being tested by AnnJi Pharmaceutical in phase 1/2a trials for safety, tolerability, and biochemical properties in people with Kennedy's disease, says David M. Simpson, MD, FAAN, professor of neurology and director of the clinical neurophysiology laboratories and neuromuscular diseases division at the Icahn School of Medicine at Mount Sinai in New York City. AJ201 may help by breaking down the faulty androgen receptor protein that causes Kennedy's disease.

Leuprorelin Acetate

Originally used for treating prostate cancer because of its ability to lower testosterone levels, leuprorelin acetate is now being explored for its potential to slow the progression of Kennedy's disease by reducing the harmful effects of a mutant protein linked to the condition, says Dr. Tobon, who is also associate professor of neurology at the University of Texas Health Science Center in San Antonio. A study published in Internal Medicine in 2019 showed some promise, and phase 3 trials are testing the drug's long-term efficacy and safety.

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