Tufts MC researchers advance new therapy to increase longevity, improve quality of life for ALS patients.
For more than 20 years, Tufts Medical Center Division of Hematology Oncology Investigator Guo-fu Hu, PhD has dedicated his research to understanding the mechanisms of angiogenin. A protein found in the blood stream, angiogenin helps control bone, muscle and nerve function and is essential for motor neuron survival and growth, especially when the body experiences stress. Dr. Hu’s latest angiogenin research efforts have focused on using the protein as a supplemental therapy for amyotrophic lateral sclerosis (ALS); a recent breakthrough has the potential to result in the first-ever disease-modifying drug to treat the rare but deadly neurodegenerative disorder.
According to the ALS Association, 5,600 people in the United States are diagnosed with the disease every year. Rapidly progressive and almost always fatal, ALS destroys motor neurons in the brain and spinal cord, resulting in a loss of speech and motor function, difficulty swallowing, weight loss and respiratory failure. ALS patients typically live three-to-five years after diagnosis.
Dr. Hu and his research team found that ALS patients have a genetic mutation that inactivates and slows the production of angiogenin. By using a simple angiogenin injection to replace that critical lost protein in a mouse model, Dr. Hu saw a dramatic improvement in neuromuscular function and an increased life expectancy, more than double that of the only ALS drug on the market. While angiogenin therapy did not cure the mice, they experienced better quality of life, in addition to regaining muscle strength and increased longevity.
“The lone FDA-approved drug currently available to ALS patients is only moderately effective,” said Dr. Hu. “It has shown to increase survival by two-to-three months for people who take it for 18 months—and it doesn’t alleviate symptoms or have any effect on quality of life. Our hope is that angiogenin therapy will double that longevity, improve symptoms and enhance quality of life.”
Dr. Hu’s group is now actively pursuing industrial partners to develop an angiogenin drug for human use.
“Once an appropriate partner is found, clinical trials could begin within a year,” said Dr. Hu. “Angiogenin has the potential to make a real difference for ALS patients, who are suffering from this terrible disease.”