Lymphyblastic leukemia is a destructive, painful blood cancer that progresses quickly and can be fatal within months if left untreated. And while treatments such as chemotherapy are currently commonly used, many patients suffer relapse, lowering the disease’s overall cure rate to between 30 and 40 percent.
But the genetic modification of T-lymphocytes is now emerging as a powerful approach to investigate T cell function. Alliance for Cancer Gene Therapy (ACGT) funded research grants for two investigators work in the area: that of Dr. Michel Sadelain, director of the Center for Cell Engineering & Gene Transfer and Gene Expression Laboratory at Memorial Sloan-Kettering Cancer Center, and Dr. Carl June, professor in Immunotherapy, Pathology and Laboratory Medicine at the University of Pennsylvania’s Abramson Cancer Center.
Dr. Sadelain’s research on T cell engineering and artificial antigen was bolstered by a 2004-2007 ACGT research grant for a program that investigated the therapeutic potential of genetically-targeted T cells in leukemia and lymphoma treatments. This Phase 1 of clinical trials research followed Sadelain’s demonstration that blood T cells can be genetically redirected to vanquish B-cell tumors – a discovery that demonstrated for the first time that genetically modified human T cells can induce enduring remission.
Following his ACGT-funded research, Sadelain’s treatment went through the subsequent phases of clinical trials and the JCAR015 drug – which came out of that research and was developed by Sadelain and his Sloan-Kettering colleague Renier Brentjens – was granted Breakthrough drug status by the FDA earlier this year. This “breakthrough” status, reserved for treatments for serious or life-threatening conditions, expedites the four phases of clinical trials and final FDA approval.
Sadelain is also a scientific founder of Juno Therapeutics, a biotech company focused on cell-based cancer immunotherapies. The company recently released its IPO at the very end of 2014 and closed up more than 45% on its first day of trading, the Wall Street Journal reported.
Sadelain’s fellow ACGT-funded investigator Dr. Carl June has made similarly groundbreaking strides in cell therapy treatment for lymphyblastic leukemia.
ACGT funded Dr. June’s early work in 2004, which studied whether the immune system’s T cells can be altered genetically so that they become “guided missiles” that kill tumor cells. The clinical trial used genetically modified versions of the patient’s own T cells, and showed remission for up to a year in a small group of patients – several others of whom are now in complete remission. Since then, Dr. June and his team at the University of Pennsylvania Abramson Cancer Center have made great strides for the treatment of advanced chronic lymphocytic leukemia, culminating most recently with the FDA’s breakthrough designation for a new therapy .
The designation was awarded after 27 of the 30 patients in the studies achieved a complete remission a month after receiving an infusion of these engineered cells, and 78 percent of patients were still alive following the trial in 2012.
ACGT continues to fund research, which is carefully selected by its esteemed Scientific Advisory Council. By funding this breakthrough research, ACGT strives to reduce the painful side effects of cancer treatments, and allow patients to live longer, better quality lives .
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