-- HealthDay staff
SUNDAY, Dec. 8, 2013 (HealthDay News) -- Preliminary research
shows that gene therapy might one day be a powerful weapon against
leukemia and other blood cancers.
The experimental treatment coaxed certain blood cells into
targeting and destroying cancer cells, according to research
presented this weekend at the American Society of Hematology's
annual meeting in New Orleans.
"It's really exciting," Dr. Janis Abkowitz, blood diseases chief at the University of Washington in Seattle and president of the American Society of Hematology, told the Associated Press. "You can take a cell that belongs to a patient and engineer it to be an attack cell."
At this point, more than 120 patients with different types of
blood and bone marrow cancers have been given the treatment,
according to the wire service, and many have gone into remission
and stayed in remission up to three years later.
In one study, all five adults and 19 of 22 children with acute
lymphocytic leukemia (ALL) were cleared of the cancer. A few have
relapsed since the study was done.
In another trial, 15 of 32 patients with chronic lymphocytic
leukemia (CLL) initially responded to the therapy and seven have
experienced a complete remission of their disease, according to a
news release from the trial researchers, who are from the
University of Pennsylvania.
All the patients in the studies had few options left, the
researchers noted in the news release. Many were ineligible for
bone marrow transplantation or did not want that treatment because
of the dangers associated with the procedure, which carries at
least a 20 percent mortality risk.
The gene therapy could become a much needed alternative for
those with blood cancers.
"Our findings show that the human immune system and these modified 'hunter' cells are working together to attack tumors in an entirely new way," research leader Dr. Carl June, professor in immunotherapy in the department of pathology and laboratory medicine and director of translational research at Penn's Abramson Cancer Center, said in the news release.
Penn researchers have treated the most patients, 59, with this
gene therapy. Scientists at the U.S. National Cancer Institute,
Memorial Sloan-Kettering Cancer Center in New York City, and the
University of Texas MD Anderson Cancer Center and Baylor University
in Houston have treated smaller groups of patients, according to
In the studies, researchers filtered the patients' blood,
removing white blood cells known as T-cells that are part of the
body's immune system. They then added a gene to the T-cells that
would target cancer cells. The altered T-cells were returned to the
patients' body in infusions that were given over the course of
Several companies are developing these types of cancer
therapies, and a clinical trial next year could lead to federal
approval of the treatment by 2016, the
"From our vantage point, this looks like a major advance," Lee Greenberger, chief scientific officer of the Leukemia and Lymphoma Society, told the AP. "We are seeing powerful responses... and time will tell how enduring these remissions turn out to be."
The gene therapy must be made individually for each patient, and
lab costs now are about $25,000, without a profit margin, the
The treatment can cause severe flu-like symptoms and other side
effects, but these have been reversible and temporary, doctors
For more on leukemia and lymphoma, visit the
Leukemia and Lymphoma
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