But the biggest hurdle yet may be the cost of the therapies.
The
genetic engineering involved means CAR therapies are very complex to
manufacture, and each is a unique personalized treatment using a
patient’s own blood cells. The inability to mass-produce them has likely
implications for how much companies will charge for them.
“What
we’re talking about here is a single, very expensive therapy that’s
used once for a specific patient and is not generalizable,” says Dr.
Malcolm Brenner,
director of the Center for Cell and Gene Therapy at the Texas
Children’s Hospital in Houston.
Dr. Brenner signed a deal in March to commercialize his own CAR research with Celgene.
Novartis
and Juno say it is too early to speculate on price, although Dr. Usman
agrees the challenge is getting the manufacturing process to “a viable
level where it’s both affordable and attractive.”
While
most analysts think it is too early to estimate potential revenue or
price, Citigroup believes CAR therapies could cost in excess of $500,000
per patient, which it notes is roughly in line with the cost of a stem
cell transplant.
“This technology needs
to be widely developed and accessible to patients,” says Dr. DeAngelo.
“If the cost is going to be a hindrance, it’s going to be a really sad
day.”
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