The largest clinical study of patients with advanced leukemia found that 88 per cent went into complete remission after being treated with genetically modified versions of their own immune cells.
“These extraordinary results demonstrate that cell therapy is a powerful treatment for patients who have exhausted all conventional therapies,” said Michel Sadelain, Director of the Center for Cell Engineering at Memorial Sloan Kettering in the US.
The results of study of 16 patients were published today in Science Translational Medicine.
Adult B cell acute lymphoblastic leukemia (B-ALL), a type of blood cancer, is difficult to treat because the majority of patients relapse.
The patients in the study had few treatment options. Only 30 per cent with their type of cancer respond to savage chemotherapy. Without a successful bone marrow transplant, few have any hope of long-term survival.
The 16 patients with relapsed B-ALL were given an infusion of their own genetically modified immune cells called T cells.
The cells were re-educated to recognise and destroy cancer cells.
Dennis J. Billy Pennsylvania was one of the first patients to receive this treatment more than two years ago.
He was able to successfully undergo a bone marrow transplant and has been cancer-free and back at work teaching theology since 2011.
For more than a decade, researchers at Memorial Sloan Kettering have been exploring ways to re-engineer the body’s own T cells to attack cancer.
Renier Brentjens, Director of Cellular Therapeutics at Memorial Sloan Kettering and one of the study’s senior authors, said:
“It’s extremely gratifying to witness the astonishing results firsthand in my patients, having worked for more than a decade developing this technology from the ground up.”
In March 2013, the same team of researchers first reported the results of five patients with advanced B-ALL who were treated with cell therapy. All five patients achieved complete remissions.
In the latest study, seven of the 16 patients (44 per cent) were able to successfully undergo bone marrow transplantation — the standard of care and the only curative option for B-ALL patients — following treatment.
Three patients were ineligible due to failure to achieve a complete remission, three were ineligible due to preexisting medical conditions, two declined, and one is still being evaluated for a potential bone marrow transplant.
Historically, only 5 per cent of patients with relapsed B-ALL type cancer have been able to transition to bone marrow transplantation.
The study also provides guidelines for managing side effects of cell therapy, which can include severe flu-like symptoms such as fever, muscle pain, low blood pressure, and difficulty breathing.
The researchers developed diagnostic criteria and a laboratory test that can identify which patients are at greater risk for developing this syndrome.
Additional studies to determine whether cell therapy can be applied to other types of cancer are underway.
Dr Sadelain and Dr Brentjens are co-holders of a patent on the technology used to create the modified T cells in this study. The technology was licensed to Juno Therapeutics in December 2013.
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