Nov 30 Most patients who previously were not
helped by repeated treatments for multiple myeloma showed strong
benefits from a new type of therapy in a small study, and with
no worrisome side effects, drugmaker Bluebird Bio Inc
said on Wednesday.
The Phase 1 study enrolled patients who had basically run
out of other options for the blood cancer, after failing on
average six previous rounds of treatment, including with prior
stem cell transplants. Data from nine patients was evaluated for
safety and efficacy and slated for presentation on Thursday at a
medical meeting in Munich.
Among three patients given the lowest dose of the
experimental treatment, one showed at least a 50 percent
reduction in signs of the disease, including of a protein
considered a hallmark of the cancer.
But all three patients receiving a medium dose of the
treatment, and all three receiving a high dose showed at least a
50 percent reduction in signs of cancer. Among those six
patients, two had no minimum residual disease (MRD), meaning
they had no remaining detectable trace of the cancer as
evaluated by the most highly sensitive available diagnostic.
"The consistency of the response and the depth of the
response is surprising, especially getting patients that are MRD
negative, which is something you typically do not see in this
population," Bluebird Chief Executive Nick Leschly said in an
The treatment, called bb2121 and being developed in
partnership with larger U.S. biotech Celgene Corp,
targets a protein called BCMA that is found on cancerous blood
plasma cells. Normal plasma cells are found in the bone marrow
and are an important part of the immune system.
Bluebird's infused treatment is a member of an emerging
potent new type of cancer therapy called CAR T cells. The
treatments are T-cells, white blood cells that act as soldiers
against foreign invaders, that have been genetically altered to
make them better able to spot and attack cancer.
Leschly said other researchers, including ones sponsored by
Novartis AG, are also attempting to develop CAR T cells
that target the BCMA protein. But he said side effects of those
treatments have included a potentially life-threatening
inflammatory condition called cytokine release syndrome (CRS)
seen with CAR T cells, although it typically can be controlled
Bluebird said no CRS incidents, or side effects that could
preempt higher doses of bb2121 in future studies, have been seen
with its treatment.
(Reporting by Ransdell Pierson; editing by Diane Craft)