Subject: Early Tests Show Protein May Fight Anemia in AIDS Date: Published: 5/4/87 120 lines Source: WALL STREET JOURNAL. Copyright Dow Jones & Co. Inc. Early Tests Show Protein May Fight Anemia in AIDS --- Research Suggests GM-CSF Blocks Reproduction Of HIV Virus as Well --- By Marilyn Chase Staff Reporter of The Wall Street Journal SAN DIEGO -- Scientists reported early tests show a new human protein can successfully treat the problem of anemia in patients with acquired immune deficiency syndrome. Jerome Groopman, a researcher at the New England Deaconess Hospital in Boston, told scientists at a meeting here he treated 16 AIDS patients with the protein, a blood-cell growth factor called GM-CSF. He said all patients' white blood-cell counts resumed normal levels. Side effects included mild aches, chills, and fever. Phlebitis, an inflammation of the veins, occurred in four patients. The protein, naturally produced by the body in small amounts, was synthesized by gene-splicing techniques at Genetics Institute Inc. of Cambridge, Mass. It is licensed to Sandoz Inc., the Hanover, N. J., pharmaceutical concern that funded the clinical trial. Dr. Groopman's findings, if borne out in further tests, are important because a low white-blood-cell count invites infections that can be fatal to those whose immune systems are weakened by AIDS, cancer chemotherapy or other disorders. As previously reported, tests of GM-CSF against cancer began at M. D. Anderson Hospital in Houston, using a version of the protein made by Immunex Corp. of Seattle. Anemia is a double-barreled problem for AIDS patients, who often suffer a lowered white count from the syndrome itself. In addition, a lowered white count is a complication of therapy with Burroughs-Wellcome Co. 's AZT -- the only drug so far approved to treat the fatal disorder. Burroughs-Wellcome is a unit of Wellcome PLC, Britain. Dr. Groopman said the drug may also block reproduction of HIV, the virus that causes AIDS. He said five out of nine patients in the test experienced a drop in viral proteins in their blood after treatment with GM-CSF. Three patients had no change, and one showed an increase. However, GM-CSF needs more study before it can be considered an anti-viral drug. Dr. Groopman stressed his early results warrant further study in an expanded group of patients with both AIDS and a variety of malignancies, including lung cancer, breast cancer and lymphoma. "The question is whether GM-CSF will improve host defenses," Dr. Groopman said. Yet to be proven is whether the protein will live up to its performance in the test tube, where it has shown it may prevent infection-fighting cells from migrating from an infection site, and may boost the tumor-fighting capability of killer cells. Test results so far, however, were good news for both the company and AIDS patients. Despite Dr. Groopman's refusal to preview his results to non-scientists until Saturday's meeting of the American Society of Clinical Investigation, apparent leaks to Wall Street from other sources sparked near-doubling of Genetics Institute's stock price, from the low 20's in January to the low 40's in recent weeks. In national over-the-counter trading Friday, Genetics Institute closed at $41.25, down 12.5 cents. Also at the conference, scientists from the National Institutes of Health presented other AIDS drug developments. H. Cliff Lane of the National Institute for Allergy and Infectious Diseases said continuing tests of alpha interferon induced tumor shrinkage in some patients with Kaposi's Sarcoma, an AIDS-linked malignancy. But he noted the drug had no effect when patient's T4 cells, a type of immune sentry ravaged by the disease, had fallen below 100 per cubic millimeter. Though manufactured by a number of companies, Alpha interferon for the test was supplied by Schering-Plough. In addition, two new members of the AZT family of drugs are advancing through development at the National Cancer Institute. Dideoxyadenosine, or DDA, and Cyanothymidine, or CNT, like AZT, are false building blocks of DNA that insert themselves into the virus and interrupt its replication. Samuel Broder, NCI's chief of clinical oncology, said he hopes to launch human trials of DDA this summer because test-tube studies suggest it is less toxic to the bone marrow than is AZT. Saturday's meeting marked the first public mention of CNT, which appears as active as AZT in tissue culture, and thus "should be another candidate for pre-clinical development," Dr. Broder said. A presentation about ICN Pharmaceuticals Inc.'s drug ribaviran prompted a renewal of scientific skepticism about the drug, however. Several scientists suggested the patients receiving a placebo or sugar pill as a control, who should have been carefully selected to match patients in the drug group, were perhaps sicker at the outset. Thus they developed AIDS much faster, making the drug recipients look good. "I wonder how carefully patients were evaluated. How many placebo patients may have had brewing cases of (pneumonia)?" asked Dr. Groopman. Peter N. R. Heseltine, a researcher at the University of Southern California, denied there was any statistical "manipulation," but conceded that differences in patients' T4 cells, a kind of white blood cell that fights infection, did affect the disease's progression. He maintained the drug "has promise," but needs further study. (See: "Corrections & Amplifications: Genetics Institute Inc." -- WSJ May 5, 1987) [This article is made available here by Dow Jones Co. for the personal and non-commercial use of callers to this bbs, in the hope that it will be of some help to those who are suffering from the disease and others who are seeking to help them.]