US disquiet over death of teenager in gene therapy

A FURORE has broken out in the upper reaches of the American scientific and medical establishment following the death of a young man who was being treated in an experimental gene therapy programme. The circumstances surrounding the death of 18-year-old Jesse Gelsinger were the subject of a tumultuous three-day conference near Washington, which ended yesterday with calls for far tougher regulation of this pioneering sector.

Jesse, who suffered from a chronic liver condition, died in September, four days after being injected with a virus designed to carry the gene into his body. Researchers hoped that this would cure him permanently. Instead, he developed a high temperature, breathing difficulties and other complications that proved fatal.

Jesse was a volunteer in the programme, which was based at one of the most respected medical research centres in the US, at the University of Pennsylvania in Philadelphia. Taking part in experimental programmes at teaching hospitals is one way that people who are chronically ill, and so uninsurable in the US system of private health-care, can obtain state of the art treatment.

At the time, Jesse Gelsinger's death was not directly attributed to the gene treatment. It was only late last month that a report established that it probably killed him. It transpired that as many as three people may have died as a result of experimental gene therapy in the US, but Jesse Gelsinger's is the first case to be documented.

Gene therapy, which is still at a very early experimental stage, holds out the prospect of curing genetically transmitted diseases if the faulty gene can be corrected or replaced. Experiments on rats had produced hopeful results, and the University of Pennsylvania obtained funding for human treatment.

With the stakes so high, this week's conference at the National Institutes of Health uncovered depths of passion that were exceptional in the normally cool and collected world of medical science.

Several of the researchers, including the head of the programme, James Wilson, apologised for breaches of the regulations, while parents of sick children begged for the experiments to be allowed to continue. However, participants also heard revelations about the University of Pennsylvania programme that cast serious doubt both on its effectivenessand reliability.

Officials from the US Food and Drug Administration, which oversees the introduction of new drug treatments, said that they had not been told of deaths among monkeys more than a year before Jesse Gelsinger died. The university researchers for their part admitted that they had accepted Jesse even though he did not strictly qualify under FDA regulations.

The FDA is expected to respond by announcing a new study to consider how the regulations can be tightened to reduce the risks to patient.

Start your day with The Independent, sign up for daily news emails
Have you tried new the Independent Digital Edition apps?
ebooksNow available in paperback
Latest stories from i100
Have you tried new the Independent Digital Edition apps?
Independent Dating

By clicking 'Search' you
are agreeing to our
Terms of Use.

iJobs Job Widget
iJobs General

Opilio Recruitment: Product Development Manager

£40k - 45k per year + Benefits: Opilio Recruitment: We are currently recruit...

Ashdown Group: IT Support Analyst - Chessington

£25000 per annum: Ashdown Group: IT Service Desk Analyst - Chessington, Surrey...

Recruitment Genius: Domestic Gas Breakdown Engineers

£28000 - £35000 per annum: Recruitment Genius: Domestic Gas Breakdown Engineer...

Sheridan Maine: Regulatory Reporting Accountant

Up to £65,000 per annum + benefits: Sheridan Maine: Are you a qualified accoun...

Day In a Page

Homeless Veterans appeal: 'You look for someone who's an inspiration and try to be like them'

Homeless Veterans appeal

In 2010, Sgt Gary Jamieson stepped on an IED in Afghanistan and lost his legs and an arm. He reveals what, and who, helped him to make a remarkable recovery
Could cannabis oil reverse the effects of cancer?

Could cannabis oil reverse effects of cancer?

As a film following six patients receiving the controversial treatment is released, Kate Hilpern uncovers a very slippery issue
The Interview movie review: You can't see Seth Rogen and James Franco's Kim Jong Un assassination film, but you can read about it here

The Interview movie review

You can't see Seth Rogen and James Franco's Kim Jong Un assassination film, but you can read about it here
Serial mania has propelled podcasts into the cultural mainstream

How podcasts became mainstream

People have consumed gripping armchair investigation Serial with a relish typically reserved for box-set binges
Jesus Christ has become an unlikely pin-up for hipster marketing companies

Jesus Christ has become an unlikely pin-up

Kevin Lee Light, aka "Jesus", is the newest client of creative agency Mother while rival agency Anomaly has launched Sexy Jesus, depicting the Messiah in a series of Athena-style poses
Rosetta space mission voted most important scientific breakthrough of 2014

A memorable year for science – if not for mice

The most important scientific breakthroughs of 2014
Christmas cocktails to make you merry: From eggnog to Brown Betty and Rum Bumpo

Christmas cocktails to make you merry

Mulled wine is an essential seasonal treat. But now drinkers are rediscovering other traditional festive tipples. Angela Clutton raises a glass to Christmas cocktails
5 best activity trackers

Fitness technology: 5 best activity trackers

Up the ante in your regimen and change the habits of a lifetime with this wearable tech
Paul Scholes column: It's a little-known fact, but I have played one of the seven dwarves

Paul Scholes column

It's a little-known fact, but I have played one of the seven dwarves
Fifa's travelling circus once again steals limelight from real stars

Fifa's travelling circus once again steals limelight from real stars

Club World Cup kicked into the long grass by the continued farce surrounding Blatter, Garcia, Russia and Qatar
Frank Warren column: 2014 – boxing is back and winning new fans

Frank Warren: Boxing is back and winning new fans

2014 proves it's now one of sport's biggest hitters again
Jeb Bush vs Hillary Clinton: The power dynamics of the two first families

Jeb Bush vs Hillary Clinton

Karen Tumulty explores the power dynamics of the two first families
Stockholm is rivalling Silicon Valley with a hotbed of technology start-ups

Stockholm is rivalling Silicon Valley

The Swedish capital is home to two of the most popular video games in the world, as well as thousands of technology start-ups worth hundreds of millions of pounds – and it's all happened since 2009
Did Japanese workers really get their symbols mixed up and display Santa on a crucifix?

Crucified Santa: Urban myth refuses to die

The story goes that Japanese store workers created a life-size effigy of a smiling "Father Kurisumasu" attached to a facsimile of Our Lord's final instrument of torture
Jennifer Saunders and Kate Moss join David Walliams on set for TV adaptation of The Boy in the Dress

The Boy in the Dress: On set with the stars

Walliams' story about a boy who goes to school in a dress will be shown this Christmas