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 Of 18 patients who received a therapeutic dose of bb2121, all but
one responded to the treatment, a 94 percent response rate, while 56
percent remained in remission with a median follow-up of 40 weeks
after treatment.
Researchers, who reported the data at the American Society of
Hematology meeting in Atlanta, said the initial response to the
treatment was very quick and that many of the patients continued to
improve over time.
Patients in the Phase I dose-escalation study had received seven
prior treatment regimens, including regimens with the newest
multiple myeloma drugs, such as Johnson & Johnson's Darzalex, and
had undergone at least one stem cell transplant before receiving
bb2121, which is being co-developed with Celgene Corp.
"Some of these patients were going to hospice until they got this,"
said Dr. Jesus Berdeja, the study's lead investigator.
"This is unheard of, something that we haven't seen with any drugs
approved for myeloma in this type of population. The excitement
among all the myeloma providers is crazy," said Berdeja, director of
myeloma research at the Sarah Cannon Center for Blood Cancer in
Nashville.
bb2121 belongs to a potentially revolutionary new type of one-time
treatment called CAR-T therapy that involves genetic manipulation of
a patient's immune system. A patient's own disease-fighting T-cells
are harvested and genetically reengineered to target specific
proteins on cancer cells before being replaced so they can circulate
seeking out and attacking the cancer, possibly for years.
The first two CAR-T therapies from Novartis and Gilead Sciences,
through its acquisition of Kite Pharma, were approved earlier this
year for other blood cancers.
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Three patients who received what proved to be sub-optimal doses of
cells early in the bb2121 study died. Of the 18 who received higher
doses, four patients have now experienced disease progression, while
14 continue to respond, researchers reported.
The treatment was very well tolerated for a CAR-T product, Berdeja
said.
Only two patients experienced serious cytokine release syndrome with
no reports of serious brain toxicity, both common side effects of
CAR-T treatment. The most serious side effect was neutropenia, or
very low white blood cell count, researchers said.
While this was a small Phase I study, the results were considered so
impressive that Celgene plans to begin enrolling patients this month
for a larger, potentially pivotal trial that could position bb2121
to become the third approved CAR-T.
"They're hitting it so far out of the park that they're not wasting
any time," Berdeja said.
(Reporting by Bill Berkrot; Editing by Nick Zieminski)
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