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To: wlheatmoon who wrote (941)	4/28/2000 2:10:00 PM
From: wlheatmoon	Respond to of 2850

Genomic news. go2net.com Gene Therapy May Have Scored First Cure By Maggie Fox, Health and Science Correspondent Apr 27 5:38pm ET WASHINGTON (Reuters) - In what some experts are calling the first true gene therapy success, French researchers said on Thursday they had treated two infant boys for a rare, inherited immune system disorder. The boys have human severe combined immunodeficiency (SCID) X1, which leaves them without any working immune system. Patients usually live out their short lives in sterile ''bubbles'' because any infection would overwhelm them. Dr. Alain Fischer of the Necker Hospital in Paris and colleagues said they had managed to restore their immune systems using gene therapy. ``Both enjoy normal growth and psychomotor development. No side effects have been noted,'' they wrote in their report, published in the journal Science. Although they do not know how long the new genes will last and continue to provide an immune system for the boys, the researchers said their results ``pave the way'' for the same approach to be used in other genetic diseases. ``I think this is the first evidence ever that gene therapy does anything,'' said Dr. David Nelson, an expert in inherited immune diseases at the National Cancer Institute in Bethesda, Maryland, said in a telephone interview. ``This is really good stuff.'' MISSING MASTER CONTROL GENE REPLACED SCID X1 patients are missing a master control for the immune system, a cell receptor that turns on the different immune system cells, including T-cells that flag and destroy invaders and natural killer cells. Attempts to introduce an improved version of the gene into patients' bodies have not worked, so Fischer's team tried a new approach, using stem cells. Stem cells are nursery cells, found in this case in the bone marrow. They give rise to all the different kinds of blood cells, including the immune system cells. Fischer's team took bone marrow cells from the two boys, aged 8 and 11 months at the time, and purified out the stem cells. They nursed these cells along in a special cocktail of compounds meant to help the stem cells thrive and multiply, and also to make them more amenable to genetic engineering. Added into the mix was a virus carrying a healthy version of the gene the boys needed. After three days, the mixture was purified and the infected cells put back into the boys. RESULTS WITHIN 15 DAYS Within 15 days the researchers saw results. The boys started producing immune cells and chemicals. One boy who had suffered from diarrhea and skin lesions had his symptoms clear up and both went home after three months. The researchers think the stem cells carrying the new and improved gene had a survival advantage over the defective immune cells. They worked better, so they thrived. Each boy has been home for nearly a year without treatment. They have normal levels of T, B, and natural killer immune cells and have been successfully vaccinated against tetanus, diphtheria, and polio -- vaccinations that would not have worked for the boys before. Fischer said a third patient had also gone through the treatment and looked healthy after four months. Dr. William French Anderson of the University of California Los Angeles, who performed the first-ever gene therapy experiment in 1990, said Fischer's work is pivotal. ``If it hadn't worked, it would have been extremely depressing,'' he said in a telephone interview. ``SCID is unique ... if you can't successfully treat SCID you are not going to be able to treat anything else.'' PLANS TO TRY SIMILAR METHOD IN U.S. Most gene therapy has not worked because so few cells take up the new genes and produce the needed protein. But with SCID, the faulty gene is one that causes many different cells to proliferate, so a strong effect can be seen quickly. ``If you get a few corrected cells in the body, the body will amplify those and basically replace the blood system with the corrected cells,'' Anderson said. Many researchers are now looking at using stem cells to help deliver gene therapy to the body. Anderson said he intends to try it with the very first gene therapy patient, Ashanthi DeSilva, who is now 13. Anderson's gene therapy treatment of DeSilva partly corrected her immune disorder, known as ADA deficiency. ''Although she is holding up beautifully, that is not a full correction,'' Anderson said. ``We plan to go in and give her gene-corrected stem cells this summer. Once you have got stem cells in a patient, if it's a high enough level, then the patient should be corrected for life.''

To: wlheatmoon who wrote (941)	4/28/2000 2:49:00 PM
From: Chip McVickar	Read Replies (1) \| Respond to of 2850

An ancient old Geezer, whose bony tail is getting sore sitting on an old wooden bench in front of this computer. At night I use candle light and a fireplace for warmth and warring a large woolen cowling. I come to you live from mythical times, long gone. Reaching out through the eons. Fred Barter was an old family friend and mentor who is nolonger kicking about. I have no special knowledge of the medical world, just a curiosity and friends in the business. Family also knew Lewis Thomas.