From: Emlyn O'regan (oregan.emlyn@healthsolve.com.au)
Date: Thu Oct 03 2002 - 21:38:40 MDT
>From the article:
<quote>
The suspended trials sought to cure severe combined immune deficiency, a
disorder that leaves infants without working immune systems. Abbreviated as
SCID, but commonly called "bubble boy disease," it is extremely rare and is
fatal in the first year of life if left untreated.
In the most severe form, the disease affects boys who have faulty
X-chromosomes. The only treatment is bone marrow transplant. But the
transplants fail in as many as 40 percent of all children who lack a perfect
donor match, so scientists looked to gene therapy as an alternative.
In April 2000, Dr. Alain Fischer and his colleagues at the Necker children's
hospital in Paris announced that they had used gene therapy to successfully
insert corrective genes into the bone marrow stem cells of three babies with
X-linked SCID. Coming on the heels of Mr. Gelsinger's death, Dr. Fischer's
study was hailed as long-sought proof that gene therapy could work.
Dr. Fischer went on to treat six more babies and a teenager, who survived
because he had a partial immune deficiency. "Up until now, all these
patients, more than three and a half years after treatment, are doing well,"
he said. All had "close to normal immune functions," he said.
But last spring, Dr. Fischer said, one of the boys showed elevated levels of
a particular type of white blood cell, known as a T-lymphocyte, though he
had no symptoms. Subsequently, though, the boy developed chickenpox. By
August, Dr. Fischer said, he had a "significant increase" in the white cell
counts, as well as an enlarged spleen, anemia and a drop in platelets.
</quote>
So this treatment has (maybe) caused problems (not death or disablement) in
one of nine patients, for a disease that otherwise would have cut through
them like a knife through hot butter?? Hmm, that's a failure in my books.
Emlyn
> -----Original Message-----
> From: Robert J. Bradbury [mailto:bradbury@aeiveos.com]
> Sent: Friday, 4 October 2002 12:34
> To: Extropy List
> Subject: MEDICINE: gene therapy setback
>
>
>
> The NY Times has an interesting article about a possible case
> of gene therapies using retroviruses causing a leukemia like
> condition.
>
> Trials Halted on a Gene Therapy
> Sheryl Gay Stolberg
> http://www.nytimes.com/2002/10/04/health/04GENE.html?pagewanted=print
>
> Of course as the article points out retroviruses are a really blunt
> edged tool to do this kind of work with. Much better will be
> Epstein-Barr
> based vectors that do not need to integrate into chromosomes
> or vectors
> that can integrate at precise locations.
>
> Robert
>
>
***************************************************************************
Confidentiality: The contents of this email are confidential and are
intended only for the named recipient. If the reader of this e-mail is not
the intended recipient you are hereby notified that any use, reproduction,
disclosure or distribution of the information contained in the e-mail is
prohibited. If you have received this e-mail in error, please reply to us
immediately and delete the document.
Viruses: Any loss/damage incurred by using this material is not the sender's
responsibility. Our entire liability will be limited to resupplying the
material. No warranty is made that this material is free from computer virus
or other defect.
This archive was generated by hypermail 2.1.5 : Sat Nov 02 2002 - 09:17:24 MST