Gene Therapy

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.

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.

At 16 months of age, curly-headed Max can reach out and grab a toy. With his eyes he can follow his mother, Ilyce Randell, as she walks around their home in Palatine, Ill.For a child with Canavan's disease, a rare neurological disorder that starts unwiring the brain after birth, Max wasn't supposed to be playing with toys and smiling at the sight of his mother. The defective gene he inherited wipes out thinking, feeling, emotions, vision and muscles.But Max has millions of copies of a healthy gene implanted in his brain to do the job that the defective one has failed to do."

STRASBURG, Pa. -- This was not the typical audience for a medical conference, but then again, this was no typical conference. At an inn set in the lush farmland of Lancaster County, doctors attending scientific lectures were joined by young Mennonite and Amish couples, the women in bonnets and the men in suspenders, babies fussing in their laps.The Amish and Mennonites may travel by horse and buggy and forgo most modern conveniences, but there is one bit of progress that they are eager to embrace: gene therapy.

It's been almost a year since Dr. Ronald G. Crystal and Dr. Todd Rosengart of New York Hospital-Cornell Medical Center opened the chest of a 60-year-old man and did what no surgeon had done before: They injected a gene-loaded solution into the man's heart in the hope of stimulating blood vessel growth.The goal of the pioneering treatment is to alleviate a recurrence of the pain and heart damage that can result from blocked coronary arteries.Aside from making medical history, the doctors touched off a crowded and intense race to develop gene therapies for heart and vascular diseases.

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.

A researcher who runs one of the nation's most closely watched cancer labs said yesterday that two new drugs that shrink tumors by starving them of their blood supply will likely be used to augment older therapies before they will be used alone.Dr. Judah Folkman, whose experiments touched off a media frenzy and a surge of interest on Wall Street earlier this year, said he was encouraged by studies at the University of Chicago that showed one of the drugs dramatically improved the effectiveness of radiation on cancerous mice.

ORLANDO, Fla. -- Scientists might have one of the most effective weapons yet in the fight against deadly ovarian cancer if a treatment presented yesterday in Orlando succeeds in clinical tests."

M. James Barrett and his lean team of scientists and engineers are hot on the trail of overcoming a challenge that has stumped medical device makers for years: developing a fast, accurate blood-sugar monitor small enough to be implanted in diabetics.Barrett puts the task before him succinctly: "The field is littered with the dead bodies of those who have tried and failed at this."Nevertheless, the 56-year-old executive is no stranger to the high-wire risk involved in launching a new company to develop and commercialize breakthrough technologies.

More than 60 Maryland scientists and technicians will lose their jobs under a plan by Novartis AG, the world's largest drug company, to merge its two gene therapy subsidiaries, Gaithersburg-based Genetic Therapy Inc. and Palo Alto, Calif.-based Systemix Inc.Under the Swiss company's consolidation plan, Systemix will become the headquarters for development of gene therapy treatments, while GTI will take a back seat, focusing on core research and overseeing studies to assess the safety of experimental gene therapies.

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.

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.

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+.

These events are scheduled at the Baltimore Convention Center, Howard and Pratt streets: May 30-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+.

These events are scheduled at the Baltimore Convention Center, Howard and Pratt streets: May 21-26 American Geophysical Union -- convention. Estimated attendance: 3,000+. Contact number: 202-777-7333, ext. 325 May 22-24 Towards Electronic Patient Records -- convention. Estimated attendance: 4,000+. Contact number: 617-964-3923, ext. 204 May 24-27 Azusa 100 -- convention. Estimated attendance: 5,000+. Contact number: 410-750-1735 May 27-28 BACNA -- meeting. Estimated attendance: 1,500+.

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.

ROCKVILLE - A Food and Drug Administration advisory panel recommended yesterday that two gene therapy experiments be allowed to resume only if the patients have exhausted other "reasonable alternatives," including bone marrow transplants. Otherwise, the panel suggested the trials - at the National Institutes of Health and the University of Southern California - remain suspended. Along with another trial at USC, the experiments had been curtailed after a third child developed leukemia in a similar French study.