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Being Alive
Wasting Syndrome and Human Growth Hormone
Brian Stott
September 5, 1996
Being Alive 1996 Sep 5: 3

On August 23, the Food and Drug Administration granted accelerated approval for the commercial distribution of Serono Laboratories' Serostim (somatotropin), a human growth hormone for the treatment of aids-related wasting syndrome. Wasting in people with aids is a strong predictor of disease progression and death.

In various studies, human growth hormone (HGH) has shown significant promise in treating this condition. Under an accelerated approval program, Serono will be required to conduct additional trials before Serostim can receive full market approval. Testing will seek to confirm the positive results found in previous studies and to further evaluate whether Serostim improves quality of life and physical performance.

Serano hopes to be able to release the drug "before Thanksgiving," and is working to produce adequate supplies, draft prescribing information, and finalize details regarding distribution. In the meantime, the drug is available from qualified doctors under an FDA-approved treatment IND (Investigational New Drug) program. This type of program has specific restrictions as to who can receive the drug. The treatment IND program allows Serono to charge for Serostim, which can cost $150 per day. Fortunately for some, a number of insurance companies and state Medicaid programs (including Medi-Cal) are paying for the drug on a case-by-case basis. Serono also operates an indigent program. For further information regarding drug availability, patients and doctors can call the Serostim Access Line at 800.714.2437.

Weight Loss In People With AIDS Unintentional weight loss in people with aids can be caused by many factors, including opportunistic infections, the use of medications, or simply poverty. PWAs should be individually evaluated to determine the causes of their weight loss and to evaluate appropriate treatments.

Wasting syndrome is one of the primary causes of weight loss in PWAs and refers to a condition in which an individual loses lean body mass, especially from muscle and other vital tissues which are high in protein. Usually, the body uses sugars to provide immediate energy, and converts fats to sugars when needed. In PWAs, proteins instead of fats are converted to sugars to provide needed energy. In the process, muscles and other structures begin to deteriorate, further damaging the person's ability to fight disease. Although this alteration of body metabolism is not fully understood, over-production by the damaged immune system of inflamatory chemicals called cytokines (especially tumor necrosis factor) is thought to be among the principal culprits.

Treating Wasting Syndrome Many drugs are being studied to correct the metabolic process found in wasting syndrome. Thalidomide has received the most attention in this regard but has not yet been approved by the FDA for this purpose. Some of the other compounds which may correct the metabolic process are available through health food stores and buyers clubs. A good reference for further information on this subject is the May 1995 of the newsletter GMHC Treatment Issues, which is available from GMHC (Gay Men's Health Crisis), 129 West 20th Street, New York, NY 10011, Fax: 212.337.3656.

Testosterone and other anabolic steroids, while not correcting the underlying metabolic defect, appear to be effective in compensating for weight loss due to wasting syndrome. There is significant evidence that steroids, when combined with exercise, increase muscle mass and break down fat. Various steroids are already approved by the FDA and are usually much cheaper than many other treatments used to treat aids-related conditions. Steroids are also reported to increase energy and improve overall quality of life. Adverse side effects may include acne, high blood pressure, liver damage, personality changes, and increased masculinization. Synthetic anabolic steroids are less masculinizing than testosterone, and therefore may be more useful in treating women with wasting syndrome.

Human Growth Hormone As is the case with steroids, human growth hormone has not been shown to correct the metabolic process of wasting, but it appears to be able to overcome the defect through other hormonal mechanisms, even in cases where steroids are not particularly effective. Gordon Sanford, a physician assistant in the office of Marcus Conant, MD in San Francisco, has been quoted as explaining who is a good candidate for HGH as opposed to steroids: "If we had ten patients on steroids and a vigorous exercise program, nine of ten would gain weight. But people who are truly wasting-taking in 100% of caloric intake and exercising but not gaining muscle mass-those are people who will benefit from HGH." While HGH has been used for years in the U.S. to treat hormone deficiency in children, its use has been highly restricted to prevent abuse by athletes. There has also been concern that the drug is being used (i.e., abused) in children of short stature who are not hormone-deficient. Therefore, very few PWAs have been able to obtain the drug from the two companies that market it in this country.

One indication of wasting syndrome is the amount of nitrogen-a critical component of proteins-which patients lose in their urine. A 1994 study found that patients stopped losing nitrogen about a day after their first dose of HGH! A 1995 Phase III study of Sterostim resulted in participants gaining an average of 6.6 pounds of lean body mass over a 12-week period. Increases in lean body mass were greater than total increases in weight because the subjects typically lost fat during the studies. This is what we would expect to see when wasting syndrome is reversed.

In various studies the side effects of the use of HGH included changes in glucose (sugar) tolerance and joint swelling. In most cases side effects were mild and went away either on their own or when the drug was stopped or the dose was reduced. Some test tube studies have shown that human growth hormone may cause hiv to replicate more quickly. Therefore, hiv+ people should probably combine an antiretroviral treatment with the use of HGH.

The Role of aids Activists AIDS treatment activists have played a crucial role in encouraging the development of and access to Serostim. According to a spokesperson for Serono, gaining accelerated approval "would have been difficult" without the support of activists. In a news release, the company gave "special thanks" to Act Up and other organizations for their support. The FDA confirmed the importance of Act Up's role.

Activists were especially concerned about the $75,000 maximum per patient per year price which Serono wanted to charge for treatments of Serostim. Activist Jeff Getty, of baboon marrow transplant fame, was able, in direct negotiations with the president of Serono, to obtain a written agreement capping the price at $36,000 per year in exchange for Act Up's support. Getty, who has received the drug as part of a long term study, credits HGH with saving his life and that of several friends.

It is very likely, however, that Serostim will prove effective in treating wasting syndrome at a significantly lower price than the $36,000 per year cap. It is probable that Serostim will show good results when used at a lower dose than in previous studies, especially when used in combination with other treatments and an exercise program.

Personal Observations Since I was first diagnosed with aids several years ago, I have experienced significant weight loss and have been treated with IV infusions (particularly TPN), steroids, and other medications in order to slow that process. In addition, I have maintained an exercise program to the best of my ability under the circumstances. Although I believe these efforts were crucial in prolonging my life, I feel they were stopgap measures.

I became interested in HGH early in 1995 when my primary care physician, Michael J. Scolaro, MD, and his staff were working to qualify to provide the drug under treatment IND status. The first of Dr. Scolaro's patients received HGH in May 1995, and several other patients received the drug in the following months. However, my insurance company declined to pay for HGH, and it was with growing frustration that I heard glowing reports of the drug's effectiveness from fellow patients.

My insurance expired on July 1, 1996 and I began receiving HGH through Medi-Cal. Administering the drug to myself by injection has been no problem; it's amazing what one can get used to when working to stay alive. Although I have experienced some transient side effects, I also have been very pleased with the visible benefits of increased muscle mass and improved muscle definition which I believe are the result of HGH, and which I was unable to obtain through other treatments and exercise, even before I was diagnosed with AIDS.