Gene cure trials for blood-clot disorder

Doctors are preparing to run the first British clinical trial of a radical treatment for haemophilia, the blood- clotting disorder inherited by one in every 5,000 babies.

Doctors are preparing to run the first British clinical trial of a radical treatment for haemophilia, the blood- clotting disorder inherited by one in every 5,000 babies.

The trial will involve injecting volunteers with a genetically engineered version of the common cold virus containing a human blood-clotting gene. If the experiment works, patients will probably need only one injection every year rather than the three injections of blood-clotting agents they now receive each week to prevent uncontrolled bleeding.

Haemophilia, one of the first diseases to be shown to have a genetic cause, is seen as an important test of whether gene therapy can cure inherited diseases. Gene therapy has had only limited success, such as in treating rare disorders of the immune system.

Scientists from the Royal Free Hospital in London have had talks with the Government's gene therapy advisory committee, which regulates genetic experiments on patients. The trial could begin soon after they receive regulatory approval

The experiment will be based on a trial in the United States in which eight men with haemophilia are being injected with a virus that carries the corrected gene into the cells of the body, where it begins orchestrating the production of a protein factor that triggers the formation of blood clots.

Katherine High, a haematologist at the Children's Hospital of Philadelphia who is running the trial, had to suspend it last year after she detected viral genes in the semen of one of the volunteers. This raised fears that the gene therapy might result in the alteration of genes within sperm, risking the passing on of a genetic alteration of children. This would constitute "germline gene therapy", which is banned in both Britain and the United States.

Further research showed that the viral genes had not penetrated the sperm cells and that the virus was eventually cleared from the semen. The American Food and Drug Administration has therefore allowed the trial to continue, raising the prospect of a similar experiment in Britain.

Christine Lee, a professor of haemophilia and the director of the haemophilia centre at the Royal Free, said trials were expected, but when they would start was not clear. The formal proposal to the government committee was not finished, she said. "My guess is that certainly in the next five years we'll be participating in trials but I feel a little conservative about the time it will take for it to become mainstream treatment," Professor Lee said. "If someone has mild haemophilia, I don't think you're going to use gene therapy because the only time they're going to need treatment is when they have an operation."

Professor Lee said there were ethical difficulties with introducing a new treatment when existing therapies worked so well. People had to be told they were being used in an experiment rather than treated. Gene therapy, however, would have many advantages if it worked, she said. "It's a fantastic model for other inherited conditions."

Start your day with The Independent, sign up for daily news emails
Arts and Entertainment
Brendan O'Carroll as Agnes Brown in the 2014 Mrs Brown's Boys Christmas special
tvCould Mrs Brown's Boys have taken lead for second year?
Arts and Entertainment
Madonna is not in Twitter's good books after describing her album leak as 'artistic rape and terrorism'
music14 more 'Rebel Heart' tracks leaked including Pharrell Williams collaboration
Arts and Entertainment
Jack O'Connell stars as Louis Zamperini in Angelina Jolie's Unbroken
film review... even if Jack O'Connell is excellent
News
peopleIt seems you can't silence Katie Hopkins, even on Christmas Day...
PROMOTED VIDEO
ebooks
ebooksA year of political gossip, levity and intrigue from the sharpest pen in Westminster
Latest stories from i100
Have you tried new the Independent Digital Edition apps?
Independent Dating
and  

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

iJobs Job Widget
iJobs General

Ashdown Group: Senior Marketing Executive- City of London, Old Street

£40000 - £43000 per annum + benefits: Ashdown Group: Senior Marketing Executiv...

Ashdown Group: Marketing Manager

£40000 - £43000 per annum + benefits: Ashdown Group: An international organisa...

Ashdown Group: Internal Recruiter -Rugby, Warwickshire

£25000 - £30000 per annum: Ashdown Group: Internal Recruiter -Rugby, Warwicksh...

Ashdown Group: Marketing Manager/Marketing Controller (Financial Services)

£70000 - £75000 per annum + benefits: Ashdown Group: Marketing Manager/Marketi...

Day In a Page

A Christmas without hope: Fears grow in Gaza that the conflict with Israel will soon reignite

Christmas without hope

Gaza fears grow that conflict with Israel will soon reignite
After 150 years, you can finally visit the grisliest museum in the country

The 'Black Museum'

After 150 years, you can finally visit Britain's grisliest museum
No ho-ho-hos with Nick Frost's badass Santa

No ho-ho-hos with Nick Frost's badass Santa

Doctor Who Christmas Special TV review
Chilly Christmas: Swimmers take festive dip for charity

Chilly Christmas

Swimmers dive into freezing British waters for charity
Veterans' hostel 'overwhelmed by kindness' for festive dinner

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
Isis in Iraq: Yazidi girls killing themselves to escape rape and imprisonment by militants

'Jilan killed herself in the bathroom. She cut her wrists and hanged herself'

Yazidi girls killing themselves to escape rape and imprisonment
Ed Balls interview: 'If I think about the deficit when I'm playing the piano, it all goes wrong'

Ed Balls interview

'If I think about the deficit when I'm playing the piano, it all goes wrong'
He's behind you, dude!

US stars in UK panto

From David Hasselhoff to Jerry Hall
Grace Dent's Christmas Quiz: What are you – a festive curmudgeon or top of the tree?

Grace Dent's Christmas Quiz

What are you – a festive curmudgeon or top of the tree?
Nasa planning to build cloud cities in airships above Venus

Nasa planning to build cloud cities in airships above Venus

Planet’s surface is inhospitable to humans but 30 miles above it is almost perfect
Surrounded by high-rise flats is a little house filled with Lebanon’s history - clocks, rifles, frogmen’s uniforms and colonial helmets

Clocks, rifles, swords, frogmen’s uniforms

Surrounded by high-rise flats is a little house filled with Lebanon’s history
Return to Gaza: Four months on, the wounds left by Israel's bombardment have not yet healed

Four months after the bombardment, Gaza’s wounds are yet to heal

Kim Sengupta is reunited with a man whose plight mirrors the suffering of the Palestinian people
Gastric surgery: Is it really the answer to the UK's obesity epidemic?

Is gastric surgery really the answer to the UK's obesity epidemic?

Critics argue that it’s crazy to operate on healthy people just to stop them eating
Homeless Veterans appeal: Christmas charity auction Part 2 - now LIVE

Homeless Veterans appeal: Christmas charity auction

Bid on original art, or trips of a lifetime to Africa or the 'Corrie' set, and help Homeless Veterans
Pantomime rings the changes to welcome autistic theatre-goers

Autism-friendly theatre

Pantomime leads the pack in quest to welcome all