In a bold new approach ultimately aimed at trying to cure AIDS, scientists used genetic engineering in six patients to develop blood cells that are resistant to HIV, the virus that causes the disease.
Three months later, five men had three times the number of modified cells expected. As much as 6 percent of their total T-cells appear to be the new type — resistant to HIV, Lalezari said.The sixth man also had modified cells, but fewer than expected. In all six patients, the anti-HIV cells were thriving nearly a year after infusion, even in tissues that can hide HIV when it can`t be detected in blood."The cells are engrafting — they`re staying in the bloodstream, they`re expanding over time," said Lalezari, who has no personal financial ties to Sangamo, the study`s sponsor.The only side effect was two days of flulike symptoms. It will take longer to determine safety, but several AIDS experts said they were encouraged so far."It is a huge step" and a first for the field of genetics, said John Rossi, a researcher at City of Hope in Duarte, Calif., where he and Zaia plan another study to test Sangamo`s approach. "The idea is if you take away cells the virus can infect, you can cure the disease."On Wednesday, Dr. Carl June, a gene therapy expert at the University of Pennsylvania, will report partial results from a second, federally funded study of 10 people testing Sangamo`s product. He treated his first patient with it in July 2009.Many questions remain:• People born without the CCR5 gene are generally healthy, but will deleting it have unforeseen consequences?• Will HIV find another way into cells? Certain types of the virus can use a second protein receptor, though this is less common and usually when AIDS is advanced. Sangamo is testing a similar approach aimed at that protein, too.• How long will the modified cells last? Will more be needed every few years?• Could doctors just infuse Sangamo`s product rather than removing cells and modifying them in the lab?• What might this cost?Sangamo spokeswoman Liz Wolffe said it`s too early in testing to guess, but it would be "a premier-priced" therapy — in the neighborhood of Dendreon Corp.`s new prostate cancer immune therapy, Provenge — $93,000.Yet AIDS drugs can cost $25,000 a year, so this could still be cost-effective, especially if it`s a cure.Jay Johnson, 50, who works for Action AIDS, an advocacy and service organization in Philadelphia, had the treatment there in September."My results are excellent," he said. "The overall goal is to not have to take medication, and then hopefully lead maybe to a cure."Matt Sharp, 54, of suburban San Francisco, also had the treatment in September."I would trade anything to not have to take a handful of medications every day for the rest of my life and suffer all the consequences and side effects," he said."I may not live long enough to see the cure, but I always hoped for a chance."Bureau Report
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