Wasting Away

One of the biggest challenges when treating life-threatening diseases is a syndrome called cachexia, the wasting away process. This extreme form of malnutrition leads to profound fatigue and muscle loss and is oftentimes seen in patients with diseases like cancer and AIDS.

For years physicians have tried treatment after treatment to improve a patient’s nutrition in hopes of increasing muscle mass. GTx, Inc., founded by Mitch S. Steiner, M.D., recently completed a Phase II trial of a drug called Ostarine, a selective androgen receptor modulator or SARM, designed to increase muscle mass.

Results show that patients treated with Ostarine built muscle mass and climbed stairs faster with more power than patients taking the placebo.

“If we can create a drug that builds muscle and can impact the quality of life or a person’s ability to respond to chemotherapy, for example, that would be wonderful,” said Steiner, a Vanderbilt University graduate and former fellow. “When a patient is suffering from severe cachexia and does not have enough protein stores that increase in muscle mass may improve quality of life and survival.

“We’re not certain where we would fit into the treatment process to make the most impact. It could be anywhere in the entire spectrum. If a patient feels better and comes to the dinner table because he has an appetite, then from a social and well-being standpoint he is part of the family. But when a patient has no appetite, lies in bed most of the time and begins to waste away, it completely affects the family dynamics.”

Steiner’s foray into the world of drug discovery came after successfully re-tooling a breast cancer drug, toremifene citrate, to reduce the estrogen deficiency side effects of androgen deprivation therapy for prostate cancer.

Steiner earned his BS in molecular biology from Vanderbilt in 1982 and his MD from the University of Tennessee. He completed his surgery and urologic training at Johns Hopkins Hospital. He later returned to Vanderbilt University Medical Center as an assistant professor of Urology, Cell Biology and Pathology during a fellowship under Hal Moses, M.D., and Jay Smith, M.D., in 1993. He then joined the University of Tennessee at Memphis as the endowed chair of Urologic Oncology in 1995 and served as the chair and professor of Urology.

GTx, formed in 1997, is a biopharmaceutical company dedicated to the discovery, development and commercialization of small molecules that selectively target hormone pathways to treat cancer, osteoporosis and bone loss, muscle wasting and other serious medical conditions.

Aimed at treating cachexia in cancer patients, the Ostarine study enrolled 159 patients with non-small cell lung, colorectal, non-Hodgkin’s lymphoma, chronic lymphocytic lymphoma and breast cancer. Thirty-five sites in the United States and Argentina participated in the randomized, double-blind, placebo, controlled study.

“Literature has suggested that lean body mass correlates with survival,” Steiner said. “This is not a curative therapy. If we can minimize many of the side effects of cancer treatments — fatigue, nausea (loss of appetite) — then quality of life is impacted.

“When you think of terminal diseases, it doesn’t mean you don’t want to live. It just means you need to think about how you want to live.”
- JESSICA PASLEY