Earlier studies found that growth hormone secretion is reduced in men with lipodystrophy. Since directly injecting growth hormone can have significant side effects, researchers at the MGH previously investigated the use of growth-hormone-releasing hormone (GHRH) to increase levels in a way that mimics natural control of hormone levels. In a 2004 study, they showed that patients receiving GHRH injections appeared to have more normal growth hormone levels and improvements in fat distribution.

The current study followed up an earlier Phase 2 study of tesamorelin, a growth-hormone-releasing factor that can be dosed only once a day instead of twice. Study participants – HIV lipodystrophy patients recruited from 43 sites around the U.S. and Canada – were randomly assigned to receive either tesamorelin or a placebo, self administered daily for six months. At the end of the study period, researchers measured participants’ visceral fat – deposits around organs deep in the abdomen – and subcutaneous fat in arms and legs. They also recorded key lipid measurements and levels of the hormone IGF-1, which reflects the release of growth hormone. In addition, participants were surveyed at the beginning and end of the study on their perceptions of their bodies and any distress they felt.

Among the more than 325 participants who completed the study, those receiving tesamorelin had significant reductions in abdominal fat, measured by CT scan, resulting in a 20 percent difference from those in the placebo group. Lipid measurements – including triglycerides, total cholesterol, HDL, and the ratio of total cholesterol to HDL – also improved significantly; IGF-1 levels reflected increased release of growth hormone in the tesamorelin group. Participants receiving the drug also reported significant improvements in their body image and reduced levels of distress.

“Longer-term studies are necessary to confirm our results, and another confirmatory Phase 3 trial needs to be completed to comply with FDA requirements,” says Grinspoon. “But this study shows clearly that the novel strategy of inducing the release of endogenous growth hormone can improve symptoms of lipodystrophy, relieving patient distress – which may improve their compliance with therapy – and reducing several cardiovascular risk factors.” Grinspoon is an associate professor of Medicine at Harvard Medical School.

The study was supported by Theratechnologies, a biopharmaceutical company based in Montréal that is developing tesamorelin, and the data were analyzed by Quintiles, Canada. Julian Falutz, MD, of McGill University Health Center, is the first author. Additional co-authors are Soraya Allas, MD, PhD, Koenraad Blot, MD, and Diane Potvin, MSc, Theratechnologies; Donald Kotler, MD, Columbia College of Physicians and Surgeons; Michael Somero, MD, Palm Springs, Calif.; Daniel Berger, MD, Northstar Health Care, Chicago; Stephen Brown, MD, AIDS Research Alliance, Los Angeles; Gary Richmond, MD, Ft. Lauderdale, Fla.; Jeffrey Fessel, Kaiser Foundation Research Institute, San Francisco; and Ralph Turner, MD, PhD, Phase V Techologies, Wellesley, Mass.

Massachusetts General Hospital, established in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH conducts the largest hospital-based research program in the United States, with an annual research budget of more than $500 million and major research centers in AIDS, cardiovascular research, cancer, computational and integrative biology, cutaneous biology, human genetics, medical imaging, neurodegenerative disorders, regenerative medicine, systems biology, transplantation biology and photomedicine.