Home' Trinidad and Tobago Guardian : July 16th 2013 Contents A28
body & soul
Guardian www.guardian.co.tt Tuesday, July 16, 2013
A disease which robs children of the ability to
walk and talk has been cured by pioneering gene
therapy to correct errors in their DNA, say doctors.
The study, in the journal Science, showed the three
patients were now going to school.
A second study published at the same time has
shown a similar therapy reversing a severe genetic
disease affecting the immune system.
Gene therapy researchers said it was a "really excit-
Both diseases are caused by errors in the patient's
genetic code - the manual for building and running
Babies born with metachromatic leukodystrophy
appear healthy, but their development starts to reverse
between the ages of one and two as part of their brain
Wiskott-Aldrich syndrome leads to a defective
immune system. It makes patients more susceptible
to infections, cancers and the immune system can
also attack other parts of the body.
The technique, developed by a team of researchers
at the San Raffaele Scientific Institute in Milan, Italy,
used a genetically modified virus to correct the dam-
aging mutations in a patient's genes.
Bone marrow stem cells are taken from the patient
then the virus is used to 'infect' the cells with tiny
snippets of DNA which contain the correct instructions.
These are then put back into the patient.
Three children were picked for treatment from fam-
ilies with a history of metachromatic leukodystrophy,
but before their brain function started to decline.
Dr Alessandra Biffi told the BBC: "The outcome
has been very positive, they're all in very good con-
dition, with a normal life and going to kindergarten
at an age when their siblings were unable to talk.
"It is something which is very pleasing to us."
She said that all treatments had side effects and
these patients needed to be followed for longer, but
the evidence so far suggested the treatment was safe.
Gene therapy is a field that has promised far more
than it has delivered and has been hampered by serious
concerns about safety.
Dr Biffi said lessons had been learnt from previous
failings: "Experience showed that gene therapy could
be improved and we could be at the starting point
for a new era to achieve more than we did in the
In the other study, published simultaneously in the
journal Science, symptoms such as repeat infections
and eczema had lessened in the three patients treat-
ed.Prof Bobby Gaspar, from Great Ormond Street Hos-
pital in London is working on a Medical Research
Council trial using gene therapy as a treatment for
adenosine deaminase deficiency - which also leads
to immune problems.
He told the BBC News website: "This is really excit-
ing. Metachromatic leukodystrophy is a very significant
neural degeneration which cannot be cured in any
other way and now the study shows they can live rel-
atively normal lives.
"It raises the prospect that other diseases can be
treated in the same way."
Prof Luigi Naldini, who leads the San Raffaele
Telethon Institute for Gene Therapy, said: "Three
years after the start of the clinical trial the results
obtained from the first six patients are very encour-
"The therapy is not only safe, but also effective and
able to change the clinical history of these severe dis-
"After 15 years of effort and our successes in the
laboratory, but frustration as well, it's really exciting
to be able to give a concrete solution to the first
Gene therapy trial 'cures children'
The hype around gene therapy was huge -
nipping into the genome and tweaking a bit of
DNA was supposed to change medicine.
However, it failed to meet expectations - there
was a death in one trial and other patients
It turned out that introducing the new genes
could activate cancer genes.
The potential was there - but safety was a
There has been years of research to come up
with safer options.
It took until 2012 for the first gene therapy to
be approved in Europe.
But there is now growing optimism that other
therapies will follow.
Gene therapy is a field that has
promised far more than it has
delivered and has been hampered by
serious concerns about safety.
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