why the bubble trial was a landmark ....
from mor2danc who posts both on Yahoo and on another
thread here on SI ...
Why Trial is a Landmark
April 27 — The first gene therapy was performed on a
child with severe combined immunodeficiency disease in
1990 by Dr. W. French Anderson and Dr. Michael
Blaese, when they were both at the National Institutes of
Health.
The girl, Ashanti De Silva, now 13 years old, received
a gene to correct a form of SCID in which the enzyme
adenosine deaminase is missing from every cell in the
body. But in the study the child also received the enzyme
by injection, and continues to do so, to correct her
disease because no one knew whether the gene therapy
would work.
While Anderson says the gene therapy helped cure
the girl, others question whether it is the added enzyme
that is really keeping the girl healthy.
The holy grail in gene therapy for this type of disease
has been to try to transform the earliest stage in the cell
lineage so future generations of cells would have the new
gene. The French trial for X-linked SCID is such a
success, Anderson says, because it might have done
just that: corrected the disease at the level of the bone
marrow stem cells in the two patients.
While over 340 trials are currently ongoing in the
United States only a handful have been promising, and
they had either local or transient effects, according to
Anderson. A clotting factor gene was recently put into
muscle cells of hemophiliac, but it is unknown how long
and at what level the cells will produce the protein.
Another gene also allowed for the growth of a new blood
vessel. In this case, the gene only had to be present for a
short time for positive results.
Follow-up findings may determine how the French
technique might be used to treat other diseases besides
SCID. “Gene therapy will not be a cookie cutter type of
treatment,” says Dr. Jennifer Puck of the National
Institutes of Health.“Each disease will require its own
unique approach,” although aspects of the French
method could be applied to other diseases such as
cancer and AIDS.
— Robin Eisner, ABCNEWS.com
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regards,
John McCarthy |
