Gene Therapy

WASHINGTON -- Pipe down. You are overselling a new and unproven method of treating serious diseases and are misleading the public into believing in miracle cures that do not exist.That sounds like mainstream medical science assailing the quacks. But there's something different about this one. It's the establishment talking to the establishment. And the admonition, a disturbing indictment of mainstream medical research, arises from the new era of super-hyped science, personal glory-seeking and overnight millionaires from biotech speculation.

It looks like there might be a genetic mutation in men that makes it easier for them to cheat, and if it is true, the nature of marriage, not to mention country music, could be changed forever. The hormone vasopressin, known in rarefied scientific circles as "the cuddle chemical," is released in men under the direction of a particular gene. Swedish researchers found that men who have extra copies of that gene actually produce less of the hormone, and those men are less likely to marry.

When two infants received gene therapy in California last weekend, doctors were making a dramatic attempt to create working immune systems that nature was obligated to provide but didn't.The newborns inherited a rare fatal immune disorder. They carried two copies of a defective gene for the enzyme adenosine deaminase (ADA) that is crucial for the immune system to fight infections. To correct their condition, doctors extracted from the umbilical cord blood stem cells that give rise to all blood cells, then placed them in a nurturing culture with genetically-altered viruses containing normal ADA genes.

Report highlights baby-bottle risk Chemicals Parents who heat plastic baby bottles risk feeding their children a synthetic hormone linked with medical, reproductive and developmental problems, according to a University of Missouri study released last week. The chemical - bisphenol A - is used in making hard, polycarbonate plastic and leaches out of the bottles when heated to 80 degrees or filled with hot liquids, researchers said. Bisphenol A is a synthetic estrogen that can cause feminization in boys, an onset of early puberty in girls, prostate and breast cancer, and some forms of diabetes.

WASHINGTON -- A scientific panel gave its approva yesterday for experimental gene therapy for cystic fibrosis patients, marking the first time the revolutionary but still-developing technique will be tried against so prevalent and lethal a disease.The unanimous approval by the federal Recombinant DNA Advisory Committee underscores the accelerating pace of gene therapy experiments.Until now, they had been tried only against a handful of extremely rare genetic diseases and a few types of cancers for which there are no cures.

BETHESDA -- The Johns Hopkins School of Medicine received preliminary approval yesterday to conduct its first gene therapy experiment on 26 kidney cancer patients who have little chance of survival.The National Institutes of Health advisory committee gave the Hopkins researchers the approval to do an experiment to determine whether the therapy was safe.The committee also approved other gene therapy experiments for brain cancer and leukemia in children, which will be done in Des Moines, Iowa, and Memphis, Tenn.

BOSTON -- Sometimes it's hard to tell the hype from the hope. For a decade now, gene therapy has been described as the Promised Land of modern medicine. When we get there, we'll be able to cure disease by changing our genetic makeup. In his State of the Union address, the president talks about "miraculous improvements" and "precision therapies." In quarterly reports, biotech firms talk about their race for the cure. At conferences, scientists talk as if success were a tantalizing breakthrough away.

By designing a special gene that can be turned on and off as needed, and then making it work in live animals, researchers in Pennsylvania said this week that they have taken a vital step toward gene therapy.Scientists at the University of Pennsylvania School of Medicine have tailored a genetic system so that it can be controlled from outside the animal by giving doses of a drug, rapamycin, according to Dr. James Wilson and his colleagues.The three-part system has been shown to work in mice and monkeys, and has continued working for months.

Oncormed Inc. of Gaithersburg said yesterday that it will collaborate with Introgen Inc., of Austin, Texas, on an early-stage clinical trial of an experimental gene therapy for cancer.Under the deal, Oncormed will provide analytical services for tissue samples collected from healthy human volunteers administered doses of a gene therapy that Introgen developed to correct mutations in what's known as the p53 gene.Mutations of that gene have been found in more than 50 percent of all cancer cases.

Two rigorous studies of gene therapy have found that it failed to help patients with cystic fibrosis or Duchenne muscular dystrophy.The diseases are caused by mutated genes, and in each case the hope was that if a good gene could be added to the cells that lack it, it might correct the defect in those cells.Scientists say that the discouraging results of the two studies show that it is time to rethink the method they are using for gene therapy, which attempts to cure diseases by adding genes to cells.

Scientists at the University of Pennsylvania have designed a gene therapy strategy to treat HIV, the precursor to AIDS, and a pilot study on five patients resistant to current therapies suggests that it may work. "It's very heartening," said one of the patients in the study, published this week in the Proceedings of the National Academy of Sciences. "The goal of the trial was safety. I didn't expect to see a benefit." The 44-year-old Pennsylvania man signed on to the experimental study in 2003, when his treatment options were limited and the medicines he was taking were no longer as effective.

The first studies of human gene therapy for Parkinson's disease have shown that the technique is safe and can reduce symptoms for patients, two groups of researchers have reported. Each of the 24 patients who received therapy in the two separate trials received some benefit and none had any significant side effects, researchers reported at neuroscience meetings Tuesday and last week. Gene therapy has a tarnished reputation because of problems encountered in trials against other diseases, said Katie Hood, deputy chief executive of the Michael J. Fox Foundation for Parkinson's Research.

In a small study of patients with terminal skin cancer, Maryland scientists have demonstrated for the first time that genetically engineered immune cells can kill off large tumors. The research, conducted by a team at the National Cancer Institute in Bethesda, remains highly experimental, and it worked on just two of the 17 patients in the study. But excited government scientists said it is the first time gene therapy has vanquished an advanced cancer. More important, the technique could serve as a potent new assault weapon against breast, lung and other, more common cancers, researchers said.

These events are scheduled at the Baltimore Convention Center, Howard and Pratt streets: June 4 American Society of Gene Therapy -- meeting. Estimated attendance: 2,500+. Contact number: 414-278-1341 June 2-8 American Society of Echocardiography -- convention. Estimated attendance: 2,500+. Contact number: 919-861-4537 June 8-9 Reality Check Plus -- meeting. Estimated attendance: 800+. Contact number: 410-837-2727 June 11-14 Special Library Association -- convention. Estimated attendance: 7,800+.

NEW YORK -- A novel gene therapy technique is safe and effective at staving off worsening symptoms of Parkinson's disease, according to the first scientific review of a dozen patients who have received the treatment over the last two years. The patients are in advanced stages of the illness and were no longer responding to medicines when they signed on for the experimental therapy. On Monday, one of the study investigators, Dr. Andrew Feigin of North Shore University Hospital in Manhasset, N.Y., told colleagues at a meeting on movement disorders in San Diego that there have been no problems with the technique, and that patients had a 27 percent improvement in symptoms.

NATIONAL Bush taps scientist for EPA post President Bush turned to a career scientist yesterday to lead the Environmental Protection Agency and push changes Bush wants in air-pollution and clean water programs. Bush nominated Stephen L. Johnson, a biologist and pathologist by training, to become the first person in the agency's 35-year history to rise from within its ranks to the top job of administrator. [Page 3a] Gene therapy recommendations Two gene therapy trials should be allowed to resume as a last recourse for the patients, an FDA advisory panel recommended yesterday.

NEW YORK -- A novel gene therapy technique is safe and effective at staving off worsening symptoms of Parkinson's disease, according to the first scientific review of a dozen patients who have received the treatment over the last two years. The patients are in advanced stages of the illness and were no longer responding to medicines when they signed on for the experimental therapy. On Monday, one of the study investigators, Dr. Andrew Feigin of North Shore University Hospital in Manhasset, N.Y., told colleagues at a meeting on movement disorders in San Diego that there have been no problems with the technique, and that patients had a 27 percent improvement in symptoms.

The brain of a terminally ill cancer patient was injected with genetically engineered mouse cells Monday in the first experiment in treating brain tumors with gene therapy.Neurosurgeons at the National Institutes of Health performed the treatment, which involves injecting foreign genes into tumor cells and then killing those cells with a drug, on a brain cancer patient who had not been helped by conventional treatments.The operation was an important step for researchers who are seeking a gateway to cure a host of fatal diseases, from cystic fibrosis to cancer and AIDS, by tapping the basic building blocks of the human body.