Hope for hemophiliacs
16th June 2008, 18:00 WST
New Australian research which aims to trick the body into accepting
blood clotting drugs could offer new hope to hemophiliacs.
A group of Sydney scientists are working on a new way to overcome a
major problem that has hamstrung treatment advances for the genetic
disease, hemophilia.
Australian genetics experts led research showing that hemophiliacs,
who lack certain proteins needed for blood to clot, could be given
the much-needed clotting gene by inserting it into a genetically-
engineered virus.
The virus was introduced into the livers of seven male patients
through a catheter with celebrated results, heralding a new gene
therapy that could dramatically reduce disease.
"We showed it to be safe, and we also showed, to our amazement, that
we were able to increase the production of this deficient factor by
at least tenfold, which would convert someone from severe hemophilia
to a much milder form of the disease," lead researcher Professor John
Rasko, who heads the gene and stem cell therapy program at the
Centenary Institute, said.
However, the "cure" was only short-lived, with one of the patients
developing minor liver damage when his own immune cells attacked the
therapeutic virus which had carried the gene into the liver cells.
Their immune systems were "primed" against the virus and were ready
to attack it once it entered the body, posing a major problem for
scientists who had been trialling the therapy since 2001.
But now Prof Rasko and his team aim to create a form of immune
suppression that will trick the body into ignoring the virus coat for
long enough to allow the gene to become permanently established
within the liver.
"If we can do that, the relevance will not just apply to hemophilia,
but to a range of illnesses including cancer, heart disease and
diabetes," he said.
"Once we learn how to develop gene transfer efficiently, we will
really overcome the biggest hurdle presented to gene therapy since
the very first clinical trial that was conducted in 1990," he
said.
The new trial involving nine patients in Sydney and Philadelphia will
begin by the end of the year, with all patients followed for up to 15
years to ensure the treatment or possible cure is long-lasting.
The announcement was made by NSW Minister for Science and Medical
Research Verity Firth at a biotechnology conference in San Diego
today.
SYDNEY
AAP
http://www.thewest.
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StemCells subscribers may also be interested in these sites:
Children's Neurobiological Solutions
http://www.CNSfoundation.org/
Cord Blood Registry
http://www.CordBlood.com/at.cgi?a=150123
The CNS Healing Group
http://groups.yahoo.com/group/CNS_Healing
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