gets NICE's approval as a treatment option for ADA- deficient Severe
combined immunodeficiency (ADA-SCID).
treatment which costs 594,000 Euros is offered only in one hospital in
- The ex-vivo gene
therapy involves genetically correcting the defective stem cells of the
SCID patient and reinserting them back into the patient.
It has been one year since GlaxoSmithKline's gene therapy Strimvelis for
treating ADA- deficient Severe combined immunodeficiency (ADA-SCID) has been
approved for sale in Europe. However, the cost of the treatment which amounts
to 594,000 Euros has limited its sale. The National Institute for Health and
Care Excellence (NICE) has in an unprecedented manner approved GSK's pricey
gene therapy, Strimvelis, as an option to treat the rare immune deficiency
condition. In its judgement, NICE stated that the treatment is cost effective,
as it helps more children survive the disease than the current standard stem cell
How does Strimvelis
Strimvelis is a gene therapy for ADA deficient SCID that was developed by
GlaxoSmithKline. After Glybera, Strimvelis is only the second gene therapy for
an inherited disease ever to be approved for sale and the first corrective
gene therapy for children to be approved anywhere in the world.
is an advanced form of treatment for genetic conditions where
the cause of the disease is due to genetic mutations or variations. Gene
therapy involves correcting the root cause of the disease at its DNA level.
Strimvelis is an ex-vivo gene therapy method where the patient's bone marrow
cells are taken outside of the body and reprogramed to correct the defective
copy of the gene in the cells. The reprogramed cells are infused back into the
patient. The modified cells now containing the corrected copy of the gene are
now able to produce the ADA enzyme and produce immune cells that were
previously not being produced as required.
Current treatment for
The current treatment for SCID is a bone marrow stem cell transplant
which can restore the immune system. However, in order to be successful, there
needs to be an exact match between the donor and the recipient. The procedure
has a high risk of mortality due to graft and host infection.
Severe combined immunodeficiency or SCID is an inherited genetic
condition where the body is not able to produce functional immune cells. This
leaves patients at a high risk of developing life threatening and recurrent
infections with the slightest of exposures. If left untreated, children usually
die before school age. Children with SCID are extremely vulnerable to
infections and live in complete isolation to minimize the risk; and hence the
nickname "baby in a bubble".
When to opt for
According to theNational Institute for Health and Care Excellence
(NICE), Strimvelis is an option to treat SCID when there are no matched stem
cell donors for a bone marrow transplant
. Despite the sky
rocketing cost of 594,000 Euros, NICE believes that the cost is a good value for
money given that the treatment offers a lifelong cure for the devastating
disease. Moreover, it has to be administered only once and the effects last a
‘Costing 594,000 Euros, GlaxoSmithKline’s gene therapy Strimvelis is given only once and the effects are thought to be life-long.’
"Strimvelis represents an important development in the treatment of
ADA-SCID, offering the potential to cure the immune aspects of the condition
and avoid some of the disadvantages of current treatments," said Professor
Carole Longson, director of the Centre for health technology assessment at
The limitations of
Other than the cost, a major disadvantage with the
treatment is that its offered only in one particular hospital in Italy. The
Ospedale San Raffaele in Milan is the only place that provides the specialized
environment to administer Strimvelis. This means that people will travel to
Italy to have the treatment. But NICE has suggested that this should be funded
by the NHS.
Despite its limitations, Bobby
Gaspar, who is carrying out separate research into gene therapy treatment for
ADA-SCID at Great Ormond Street, said that the NICE decision on Strimvelis was an important moment.
"It's an extremely exciting time
for gene therapy," Professor Gaspar said. "Over the next five years we will see
therapies against different forms of cancer and genetic forms of blindness."
He compared the science to where
organ transplants were in the 1960s. "When organ transplants started people
thought it was science fiction medicine. It's now carried out in so many
different hospitals and we transplant hearts, kidneys, livers, lungs— it's
standard medicine," he said.
- NICE approves gene therapy for rare 'bubble baby syndrome' - (https://www.nice.org.uk/news/article/nice-approves-gene-therapy-for-rare-bubble-baby-syndrome)