Find Information About:

Drugs & Supplements

Get information and reviews on prescription drugs, over-the-counter medications, vitamins, and supplements. Search by name or medical condition.

Pill Identifier

Pill Identifier

Having trouble identifying your pills?

Enter the shape, color, or imprint of your prescription or OTC drug. Our pill identification tool will display pictures that you can compare to your pill.

Get Started

My Medicine

Save your medicine, check interactions, sign up for FDA alerts, create family profiles and more.

Get Started

WebMD Health Experts and Community

Talk to health experts and other people like you in WebMD's Communities. It's a safe forum where you can create or participate in support groups and discussions about health topics that interest you.

  • Second Opinion

    Second Opinion

    Read expert perspectives on popular health topics.

  • Community


    Connect with people like you, and get expert guidance on living a healthy life.

Got a health question? Get answers provided by leading organizations, doctors, and experts.

Get Answers

Sign up to receive WebMD's award-winning content delivered to your inbox.

Sign Up

Font Size

NIH Committee Suggests Changes Following Fatal Gene Therapy Experiment

WebMD Health News

Dec. 9, 1999 (Washington) -- A federal committee charged with overseeing the safety of gene therapy is recommending a series of changes in the wake of a patient death that has attracted national attention.

After two days of hearings focused on gene therapy, which uses a disabled common cold virus to deliver a gene that corrects a liver enzyme problem, the National Institutes of Health's Recombinant DNA Advisory Committee suggested the need for better standards as well as more communication between the researchers, the FDA, and NIH.

Specifically, the panel, comprised of members from a number of government agencies, wants to see greater uniformity in the use of these viruses since the potency and the dosages of these powerful biologic agents vary widely. In addition, the committee, known as the RAC, wants to develop a database so that gene therapy researchers as well as regulators can compare their results.

"We have no interest in stopping human gene therapy, none whatsoever. We are very interested in ensuring that the quality of the trials and the quality of the science are worth the investment of the patient," Claudia Mickelson, PhD, chairwoman of the NIH panel, tells WebMD.

The hearings revolved around the fatal result of an experimental gene therapy given to 18-year-old Jesse Gelsinger on Sept. 13. It was apparently the first time a patient has died directly as a result of gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency, an inherited liver disorder that allows ammonia to build up to dangerous levels. The condition is often fatal.

The FDA was critical of the researchers, led by James Wilson, MD, director of the Institute for Human Gene Therapy at the University of Pennsylvania, saying that they shouldn't have given Gelsinger the therapy because his ammonia levels were too high immediately prior to the treatment. The agency says that some of the other 17 patients in the trial also had liver "toxicities" and also should have been disqualified.

FDA officials say they should have been notified about these issues, as well as the results of animal studies using a similar therapy that preceded Jesse's treatment. The FDA's Anne Pilaro says 11 monkeys developed liver toxicity and that five of them died. That information came to the agency after Jesse died, but a spokeswoman for the University of Pennsylvania insists that no primates got the same treatment as Jesse. A formal investigation is underway.

Today on WebMD

man holding his stomach
Get the facts on common problems.
blueberries in a palm
Best and worst foods.
woman shopping
Learn what foods to avoid.
fresh and dried plums
Will it help constipation?
top foods for probiotics
couple eating at cafe
sick child
Woman blowing bubble gum

Send yourself a link to download the app.

Loading ...

Please wait...

This feature is temporarily unavailable. Please try again later.


Now check your email account on your mobile phone to download your new app.

Woman with crohns in pain
Woman with stomach pain
diet for diverticulitis
what causes diarrhea