The 14-patient study, which began in the summer of 2010, enrolled
patients who had failed to benefit from standard treatments for
chronic lymphocytic leukemia (CLL), a cancer of white blood cells
that most commonly affects adults.
They were given one treatment with CTL019, a therapy developed by
the university and licensed to Swiss drugmaker Novartis AG. Patients
have since been monitored to assess durability of the therapy's
benefits.
The treatment is made by taking immune system soldiers called T
cells from the body, genetically modifying them to have
cancer-spotting abilities of antibodies, and then infusing the
altered cells back into the patient.
The first patient to receive the therapy is cancer-free after five
years and another of the first three enrolled patients also remains
in remission, the university said on Wednesday.
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All signs of cancer disappeared in four patients, or 29 percent, but
one of them died almost two years after therapy due to an unrelated
infection.
"The durability of the remissions we have observed in this study are
remarkable and have given us great hope that personalized cell
therapies are going to be important options for patients," said Dr.
David Porter, director of blood and marrow transplantation at the
university's Abramson Cancer Center.
Another four patients achieved some reduction in tumors, with
responses lasting an average of about seven months.
Six patients, or 43 percent, failed to respond to therapy, and their
leukemia progressed within one to nine months.
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Novartis plans next year to seek marketing approval of CTL019 to
treat pediatric patients with another cancer of the white blood
cells, acute lymphoblastic leukemia. Although it can occur in
adults, it is the most common type of cancer in children.
Novartis is considered in the lead among a handful of companies
racing to develop and launch CAR T cells to treat blood cancers,
including Kite Pharma Inc, Juno Therapeutics Inc and Bluebird Bio
Inc.
But the therapies can cause a life-threatening inflammation called
cytokine release syndrome, which was seen in all patients who
responded to CTL019 in the University of Pennsylvania study.
All patients recovered from the inflammation, including some treated
with steroids.
(Reporting by Ransdell Pierson in New York; Editing by Matthew
Lewis)
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