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Sci-Fi Leukemia Treatment Gets Amazing Results
This is one of those great stories that you want to be able to tell to people who are facing a really scary cancer diagnosis. Three patients with advanced cases of a deadly blood cancer, chronic lymphocytic leukemia (CLL), were treated with an experimental therapy that had far better results than anyone would have dared hope. As Carl June, MD, a professor of pathology and laboratory medicine at the University of Pennsylvania Abramson Cancer Center in Philadelphia, wrote in his August 10, 2011 New England Journal of Medicine report, "Within three weeks, the tumors had been blown away -- in a way that was much more violent than we ever expected."
Thatís violent in a good way... more than a year after the treatment, all three patients are still alive, with two in full remission (meaning that thereís no evidence of cancer) and one in partial remission.
This is a dramatically better outcome than what might have been expected if these patients had been given the standard treatment for their disease, which is a bone marrow transplant that offers only a 50% chance of survival five years after receiving the treatment. And before patients can even get the bone marrow transplant, itís often a challenge to find a bone marrow donor who is a match, so many patients die while waiting for one. Plus, the bone marrow procedure itself is so grueling that one in five patients dies from it.
BLAST! ZAM! POW!
Now that Iíve told you the punch line, here are the details of the treatment. Blood was drawn from the patients and T cells (types of white blood cells that defend the body against diseases or foreign materials) were extracted. Then the T cells were taken to a laboratory where doctors genetically modified them. The researchers essentially reprogrammed the T cells to multiply and selectively seek tumor cells instead of more generalized infection or inflammation -- sending these T cells on a search-and-destroy mission with the goal of eradicating the cancerous cells in each patientís body. In addition, the researchers programmed these altered T cells to "reawaken" other healthy T cells that had been suppressed by the leukemia and stimulate the production of "memory" T cells to protect against cancer recurrence. Dr. June and his colleagues reintroduced the modified cells back into the patientsí bloodstreams using an intravenous infusion.
When they examined their patients three weeks later, they found that...
Serial killer T cells had replicated at least 1,000-fold, building an army of cells to attack the cancer.
The replicated T cells killed at least two pounds of tumor in each patient.
WHAT WAS IT LIKE FOR THE PATIENTS?
Here is Dr. Juneís description of one patientís encounter with the new treatment: A 64-year-old man with extensive tumors in his blood and bone marrow experienced no significant side effects during the first two weeks of the new treatment when the altered T-cells were put into his bloodstream -- but on day 14, he developed nausea, chills, fever and other flulike symptoms.
Paradoxically, this was good news. When doctors tested his blood on day 14, they discovered that a huge increase in T cells had caused widespread death of cancer cells, which must have caused the uncomfortable (but temporary) symptoms. On day 28, his blood and marrow showed no signs of leukemia.
IS IT A CURE?
The doctors are hesitant to call this new treatment a "cure" at this stage -- especially since the treatment was tested on just three patients -- but for individuals with CLL who would otherwise face a bone marrow transplant and the many risks that entails, these patient results are extremely promising, Dr. June told me. Moving forward, the investigators plan to test genetic T cell modification in more CLL patients and also in people who have similar types of cancers, such as non-Hodgkinís lymphoma and acute lymphatic leukemia. Unfortunately, this breakthrough CLL treatment isnít available to the public yet, but the hope is that it will provide a road map to treat many other types of cancer.
Carl H. June, MD, professor of pathology and laboratory medicine, department of pathology and laboratory medicine, and director of translational research, Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia.
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