It has been a busy few weeks for The Children’s Hospital of Philadelphia’s Stephan Grupp, MD, PhD. Dr. Grupp, the director of Translational Research at the Center for Childhood Cancer Research, recently received three awards for his groundbreaking immune therapy work using genetically engineered, cancer-fighting T cells. Dr. Grupp received awards from the Clinical Research Forum, the European Society for Blood and Marrow Transplantation, and was honored alongside the University of Pennsylvania by the bioscience organization Pennsylvania Bio.
Dr. Grupp has received a great deal of attention for his investigation of using cell therapy (known as CART19 or CTL019 therapy) to treat an aggressive form of childhood leukemia, acute lymphoblastic leukemia (ALL). Last year his work — conducted in partnership with the University of Pennsylvania’s Carl June, MD — led to dramatic, extraordinary results published in The New England Journal of Medicine: two children with untreatable ALL achieved a complete response after being treated with immune therapy. Since receiving the treatment one of those patients remains healthy and cancer-free two years later.
The Clinical Research Forum (CRF) award, the 2014 Herbert Pardes Clinical Research Excellence Award, is one of ten recent awards handed out by the CRF to projects “that benefit the health and wellbeing of the general public.” Named for New York-Presbyterian Hospital’s Herb Pardes, MD, the 2014 Herbert Pardes Award is the CRF’s top prize, and was awarded at an April 10 reception in Washington, DC.
The European Society for Blood and Marrow Transplantation (EBMT), meanwhile, handed a team of researchers led by Dr. Grupp the 2014 van Bekkum Award, named for transplantation and bone marrow researcher Dirk van Bekkum, MD, PhD. The EBMT’s van Bekkum award is given to the best abstract presented at the Society’s annual meeting, held March 30 to April 2 in Milan, Italy.
In addition to Dr. Grupp, Penn’s Dr. June and David Porter, MD, as well as CHOP’s David Teachey, MD and Shannon Maude MD, PhD, among others, were co-authors on the abstract, “T cells engineered with a CAR targeting CD19 (CTL019 cells) produce significant in vivo proliferation, complete responses and long-term persistence without GVHD in children and adults with relapsed/refractory ALL.”
The data presented at the Clinical Research Forum and EBMT updated the data published in the New England Journal of Medicine. “Our group has now treated 25 kids and 5 adults with relapsed/refractory ALL,” said Dr. Grupp. “We have seen unexpectedly high rates of complete remission: 90 percent in this group of patients, many of who had no other treatment options. These results are leading to a phase 2 trial at six pediatric hospitals, with CHOP as the lead site.”
Rounding out the list of plaudits, at the recent 2014 Pennsylvania Bio Annual Dinner & Awards Celebration, CHOP and Penn — led by Drs. Grupp and June, respectively — were jointly honored for their cell therapy work with PA Bio’s Patient Impact Award.
“It’s an honor to have our cell therapy research recognized by such prestigious organizations,” said Dr. Grupp. “This is potentially revolutionary work, but its success to date — and going forward — would not have been possible without multidisciplinary, truly collaborative input from investigators across CHOP and Penn. I cannot thank my colleagues enough.”
Recent papers which Dr. Grupp has either led or contributed to include a study of managing the cytokine release syndrome associated with T cell therapy, a study of chimeric antigen receptors, and managing toxicity for patients receiving T cell therapies.