New hope in fight against 'Lorenzo's Oil' disease

A dramatic breakthrough in the possible treatment of childhood diseases of the nervous system – including the disorder featured in the film Lorenzo's Oil – was announced by scientists in America yesterday.

For the first time, researchers appear to have cured a similar disease in mice by the injection of human stem cells designed to re-build the sheath of fatty tissue surrounding nerve cells that is lost in inherited diseases such as adrenoleukodystrophy, which last week claimed the life of Lorenzo Odone, whose battle against the disease was dramatised in the film.

The myelin sheath performs the vital role of insulating nerve impulses; the possibility of re-building it offers hope to thousands of children with a host of gene disorders such as Tay-Sachs and Krabbe disease, as well as adults suffering from multiple sclerosis.

Although the research is still at the earliest stages, the scientists believe its results are a further demonstration of the power of stem-cell treatments.

The laboratory mice in the study all suffered from an inherited condition that would normally kill them within about 20 weeks of birth. However, some of the mice that received the treatment not only lived for several months after that, but actually improved to the point of seeming to be cured.

"It's extremely exciting to think about not only treating but actually curing a disease," said Professor Steven Goldman, of the University of Rochester Medical Centre in New York, who led the research team. "Unfortunately, right now, we can do little more besides tell parents to prepare for their kids to die," said Professor Goldman, whose study is published in the journal Cell Stem Cell.

Start your day with The Independent, sign up for daily news emails
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

Recruitment Genius: Multi Trade Operative

£22000 - £24000 per annum: Recruitment Genius: An established, family owned de...

Recruitment Genius: Customer Services Assistant

Negotiable: Recruitment Genius: An exciting position has risen for a Customer ...

Recruitment Genius: Tour Drivers - UK & European

Negotiable: Recruitment Genius: This is a fantastic opportunity to join a is a...

Recruitment Genius: Fundraising Manager / Income Generation Coach

£21000 - £25000 per annum: Recruitment Genius: A smart software company locate...

Day In a Page

Syria crisis: Celebrities call on David Cameron to take more refugees as one young mother tells of torture by Assad regime

Celebrities call on David Cameron to take more Syrian refugees

One young mother tells of torture by Assad regime
The enemy within: People who hear voices in their heads are being encouraged to talk back – with promising results

The enemy within

People who hear voices in their heads are being encouraged to talk back
'In Auschwitz you got used to anything'

'In Auschwitz you got used to anything'

Survivors of the Nazi concentration camp remember its horror, 70 years on
Autumn/winter menswear 2015: The uniforms that make up modern life come to the fore

Autumn/winter menswear 2015

The uniforms that make up modern life come to the fore
'I'm gay, and plan to fight military homophobia'

'I'm gay, and plan to fight military homophobia'

Army general planning to come out
Iraq invasion 2003: The bloody warnings six wise men gave to Tony Blair as he prepared to launch poorly planned campaign

What the six wise men told Tony Blair

Months before the invasion of Iraq in 2003, experts sought to warn the PM about his plans. Here, four of them recall that day
25 years of The Independent on Sunday: The stories, the writers and the changes over the last quarter of a century

25 years of The Independent on Sunday

The stories, the writers and the changes over the last quarter of a century
Homeless Veterans appeal: 'Really caring is a dangerous emotion in this kind of work'

Homeless Veterans appeal

As head of The Soldiers' Charity, Martin Rutledge has to temper compassion with realism. He tells Chris Green how his Army career prepared him
Wu-Tang Clan and The Sexual Objects offer fans a chance to own the only copies of their latest albums

Smash hit go under the hammer

It's nice to pick up a new record once in a while, but the purchasers of two latest releases can go a step further - by buying the only copy
Geeks who rocked the world: Documentary looks back at origins of the computer-games industry

The geeks who rocked the world

A new documentary looks back at origins of the computer-games industry
Belle & Sebastian interview: Stuart Murdoch reveals how the band is taking a new direction

Belle & Sebastian is taking a new direction

Twenty years ago, Belle & Sebastian was a fey indie band from Glasgow. It still is – except today, as prime mover Stuart Murdoch admits, it has a global cult following, from Hollywood to South Korea
America: Land of the free, home of the political dynasty

America: Land of the free, home of the political dynasty

These days in the US things are pretty much stuck where they are, both in politics and society at large, says Rupert Cornwell
A graphic history of US civil rights – in comic book form

A graphic history of US civil rights – in comic book form

A veteran of the Fifties campaigns is inspiring a new generation of activists
Winston Churchill: the enigma of a British hero

Winston Churchill: the enigma of a British hero

A C Benson called him 'a horrid little fellow', George Orwell would have shot him, but what a giant he seems now, says DJ Taylor
Growing mussels: Precious freshwater shellfish are thriving in a unique green project

Growing mussels

Precious freshwater shellfish are thriving in a unique green project