Transcript Document

Jesse Gelsinger
had a scary brush with
death. The teenager
had stopped taking his
medications, was having
bouts with nausea and
vomiting and eventually
found himself curled up
listlessly on the couch,
15 pounds underweight
and scared.
http://www.genelet
ter.org/05-0100/features/gelsing
er1.html
By the time his parents
got him to the hospital, his
metabolic processes had
careened out of control. He fell
into a coma and a few days
later, he stopped breathing in
his father's arms.
Jesse suffered from a
disorder called partial ornithine
transcarbamylase (OTC)
deficiency, which kept him
perpetually in danger of hoarding
toxic levels of ammonia in his
blood. Half of all infants born
with the condition die within a
month.
As soon as he turned 18,
Jesse Gelsinger volunteered for
a gene therapy trial to for OTC
and died as a result of the
procedure.
In Jesse’s case, a
replacement gene for OTC was
packaged into an engineered
adenovirus, which was injected in
high dose, directly into his liver.
The gene carrier, or vector,
was the prime suspect in
Gelsinger's death, since the
adenovirus can cause liver and
lung damage. (The virus can
trigger widespread bloodclotting and inflame surrounding
tissue.)
Why did they choose
adenovirus?
It can be grown easily in great
amounts.
They have engineered versions
with deleted genes E1 and E4.
It is good at infecting human
cells.
In response to his death,
the NIH asked researchers all
over the country and found more
than 650 dangerous adverse
reactions they also had
previously kept secret, including
several deaths.
A Canadian Story ……… this is
a case of “Suicide Gene Therapy”
cbc.ca/national/ magazine/gene/
James Dent had a malignant
brain tumor. With little hope of
conventional treatment, Dent
signed a consent form for gene
therapy, which did present a risk
of death. There was no mention
of patients who suffered serious
adverse events relevant to his
experiment.
James Dent would be
treated in two stages. The
first consisted of tumor surgery
and gene therapy. He was
injected with an adenovirus
vector, carrying the Herpes
Simplex thymine kinase gene.
This was injected directly into
his brain tumor.
In the meantime, he
returned home. ……Two weeks
later, he began the second stage
of the experiment, the injection
of the antivirus drug ganciclovir…
Within two days, he he went
into septic shock and died.
Only days before James
Dent began the second stage of
his gene therapy treatment, a
patient died in Indiana during
the same experiment. That
death was reported to U.S.
regulators as unexpected and
possibly related to the gene
therapy.
Gene therapy has about a 10
year history. To date, over
4,000 people have participated in
over 350 gene therapy clinical
protocols (25% with adenovirus),
with at best limited success.
By far, most of the clinical
trials involve gene therapy for
cancer. About 100 such trials
are currently ongoing.
The major limitation of gene
therapy in general, seems to be a
limitation in being able to deliver
enough of the “engineered gene.”