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Immunotherapy Study Funded by The Leukemia & Lymphoma Society Demonstrates Remarkable Results in Patients with Acute Lymphoblastic Leukemia

Posted: 16th October 2014 10:15
Funding from The Leukemia & Lymphoma Society (LLS) over the past 22 years helped advance a study published today in the New England Journal of Medicine showing that 90 percent of children and adults with acute lymphoblastic leukemia (ALL) achieved complete remission after receiving an investigational personalized cellular therapy.
LLS-funded researchers at the Perelman School of Medicine at the University of Pennsylvania, led by Carl June, M.D., and at Children's Hospital of Philadelphia (CHOP), led by Stephan Grupp, M.D., Ph.D, reported that of the first 25 children and young adults (ages 5-22) treated at CHOP, and the first five adults (26-30) treated at Hospital of the University of Pennsylvania, 27 of 30 achieved complete remission after being treated with genetically engineered T cells. All of these ALL patients had previously relapsed multiple times, making these results even more remarkable. Similar results have been reported employing a related personalized cellular therapy in ALL patients by groups at National Cancer Institute and Memorial Sloan Kettering Cancer Center, further validating this important advance.
This investigational immunotherapy, called CTL019, also known as chimeric antigen receptor (CAR) therapy, is an approach that genetically engineers patients' immune T cells and reintroduces them into the body to kill cancer cells. The FDA recently granted Breakthrough Therapy designation for clinical trials of this approach to treat pediatric and adult ALL patients who have not responded to or who have relapsed after treatment with conventional therapies.
"This study comes almost exactly 70 years after Robert Roesler de Villiers, the son of the founders of LLS, died from ALL," said Louis J. DeGennaro, Ph.D., LLS's president and chief executive officer. "At that time, the chance of surviving ALL was less than five percent of patients. Today nearly 90 percent of pediatric patients with ALL survive more than five years, and the findings announced today suggest that we may see even more patients surviving this blood cancer.  LLS's long-term funding of this team underscores our commitment to saving blood cancer patients' lives by funding research to find cures and better treatments. And it is also thanks to the generosity of our donors whose support over the past two decades helped make possible this promising therapeutic advance."

LLS has invested in the work of June and colleagues since 1998 and has committed to investing a total of $21 million through 2017 to get this first treatment to more patients. LLS first funded Grupp in 1992 through its career development program. LLS has also been funding another member of the team, David Porter, M.D. of University of Pennsylvania since 1994.

"Dr. Grupp's pivotal role in this new therapy, nearly 22 years after his initial involvement with LLS, defines the success of our career development program," said Lee Greenberger, Ph.D., chief scientific officer of LLS. "We continue to support this personalized cellular therapy approach as it may apply to other blood cancers, such as acute myeloid leukemia and chronic lymphocytic leukemia, as well as explore other methods to activate the immune system."

DeGennaro noted that seeing such dramatic responses from patients for whom there appeared to be no hope is truly cause for optimism.

"The first pediatric patient treated with this innovative therapy is Emily Whitehead, whose story has been widely chronicled in the media. Now 9, Emily is still thriving two years after first being treated." DeGennaro said. "Emily and all of the patients in this trial are inspiring all of us at LLS to find cures not someday but today."

About The Leukemia & Lymphoma Society

The Leukemia & Lymphoma Society ® (LLS) is the world's largest voluntary health agency dedicated to blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin's disease and myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care.
Founded in 1949 and headquartered in White Plains, NY, LLS has chapters throughout the United States and Canada. To learn more, visit Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.

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