Hormone combination therapy delivers gains for men with FSHD
Testosterone-growth hormone combo boosts strength in trial
A combined regimen of testosterone and growth hormone led to significant improvements in muscle mass, strength, and mobility in men with facioscapulohumeral muscular dystrophy (FSHD) in a clinical trial.
The Phase 1/2 STARFISH trial (NCT03123913) evaluated the combination therapy’s safety, tolerability, and potential efficacy in men with FSHD.
“We’ve never seen a therapy in FSHD deliver both real gains in strength and lasting benefit after treatment stops,” Chad Heatwole, MD, director of the University of Rochester Center for Health + Technology and lead author of the study, said in a university news story. “This hormone combination could mark the first treatment to not only slow this disease, but help patients regain function.”
The results were described in the study, “Study of Testosterone and Recombinant Human Growth Hormone in Facioscapulohumeral Muscular Dystrophy,” published in Neurology Genetics.
FSHD is a form of muscular dystrophy characterized by progressive weakness of muscles of the face, shoulders, upper arms, and hips. Over time, patients may have difficulty performing everyday tasks and walking. Current treatment options intend to ease symptoms.
Testing combo in MD
Previous studies suggested that a combination therapy of testosterone and lab-made human growth hormone (HGH) may increase muscle mass and strength in healthy adult men. The STARFISH trial was the first to test the combination in muscular dystrophy patients.
The trial enrolled 20 men with FSHD who had a mean age of 41 and had been diagnosed for a mean of 10.9 years. The majority were white (95%), had a college education (65%), were employed full time (70%), and were married (55%).
They were treated daily for six months with somatropin, a lab-made form of growth hormone, given subcutaneously (under the skin) at a dose of 5 micrograms (mcg)/kg. Every two weeks, they were treated with testosterone enanthate, a form of testosterone, at 140 mg by intramuscular injection to support muscle building. During the study, the researchers tracked changes in body composition, walking ability, and overall strength.
After the six months, treatment was stopped for three months so the investigators could assess how long any benefits would last.
Of the 19 patients who completed the study, none experienced a serious adverse effect or discontinued treatment. The most common adverse event was injection site reaction (35%), followed by pain, soreness, flu-like symptoms, cough, limb swelling, increased appetite, and muscle cramps, each reported in two participants.
After six months, lean body mass had increased by 2.2 kg (about 4.8 lbs) while fat mass decreased by 1.3 kg (2.9 lbs). The distance walked by patients in six minutes increased by 37.3 meters (122.4 feet), and overall muscle strength increased by 3% over the predicted normal score for the participants’ age and size.
The FSHD Composite Outcome Measure (FSHD-COM) score, which assesses the functional impact of the disease, improved by 2.4 points. Lower extremity strength also increased significantly. Participants also reported an overall reduction in disease burden, as measured by the FSHD-Health Index.
During the three months after stopping treatment, changes in walking ability, FSHD-COM, FSHD-HI, and lower extremity strength remained significantly different compared with the study’s start.
“The outcomes of this open-label 36-week study of testosterone and rHGH in FSHD indicate that this treatment approach is safe and well tolerated over 24 weeks and is promising as a therapy to combat and reverse impaired ambulation, weakness, muscle loss, and symptomatic burden in FSHD,” the researchers wrote.
The team plans multicenter, placebo-controlled trials to confirm these benefits, adjust dosing, and include women with FSHD. Heatwole said the therapy may also be applicable to other types of muscular dystrophy.
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