December 2013

CHOP Research Featured Prominently at ASH Annual Meeting

Hematology

The Children’s Hospital of Philadelphia made a strong showing at this year’s American Society of Hematology (ASH) Annual Meeting, with a number of CHOP researchers presenting data, chairing sessions, and delivering lectures. The “premier hematology event of the year,” the 55th ASH Annual Meeting featured special lectures, symposia on cancer, hematology, and genomic sequencing, and data from more than 5,300 abstracts.

Held December 7-10 in New Orleans, the conference brought together approximately 20,000 researchers, clinicians, and industry representatives. In just a few examples of CHOP’s strong showing at the meeting, Rodney M. Camire, PhD, co-chaired a session on targets in hemostasis and thrombosis, while sickle cell researcher Stella T. Chou, MD, gave a talk on transfusion therapy to treat sickle cell disease. Monica Bessler, MD, PhD, director of the Pediatric Comprehensive Bone Marrow Failure Center, chaired a discussion of the congenital disorder Shwachman-Diamond Syndrome, and Mortimer Poncz, MD, chief of the Division of Hematology, co-chaired a session on platelets.

There were also a number of special talks given by internationally prominent researchers, including a lecture by Children’s Hospital hematologist Katherine A. High, MD, who discussed her decades of experience developing gene therapy.

Hematologist and Molecular Researcher Honored

For her pioneering hemophilia research Dr. High, director of the Center for Cellular and Molecular Therapeutics (CCMT), was honored with the 2013 E. Donnall Thomas Lecture and Prize. The lecture and prize is named for the late E. Donnall Thomas, MD, who was awarded the Nobel Prize in Medicine in 1990 for his work developing bone marrow transplantation as a leukemia treatment. The award recognizes “pioneering research achievements in hematology that have represented a paradigm shift or significant discovery in the field,” according to the ASH.

“Dr. High is unquestionably a leader in gene therapy and has made remarkable, groundbreaking contributions to the field, demonstrating creativity, determination, and the ability to overcome numerous obstacles,” said Janis L. Abkowitz, MD, ASH president.

An internationally recognized hematologist and molecular researcher, in addition to her role at the CCMT Dr. High is also a professor of Pediatrics at the University of Pennsylvania and a Howard Hughes Medical Institute investigator. She has led translational studies in hemophilia, delineating mutations that cause the disease in patients and in a naturally occurring dog model of the disease, and reported on the first cure of hemophilia B in the canine model using gene therapy.

Recently, Dr. High’s work has expanded to include clinical studies of gene therapy for other inherited diseases, such as congenital blindness. For example, Dr. High contributed to a study of RPE65-related inherited retinal dystrophy, a rare retinal disease that progresses to total blindness by adulthood. The study team reported on improvements in vision in three adult patients previously treated with an innovative gene therapy in one eye who then received the same therapy in their second eye. And in July, a research team led by Dr. High published a study in Science Translational Medicine on their work with a bioengineered decoy designed to improve gene therapy by fooling the immune system.

In addition, Spark Therapeutics, a new “fully integrated gene therapy company” based in part on Dr. High’s work, was launched in October with a $50 million capital commitment from CHOP. Along with Children’s Hospital’s J. Fraser Wright, PhD, Dr. High is scientific co-founder of Spark. Dr. High is also a member of a number of prestigious organizations, including the American Academy of Arts and Sciences.

In her talk at the ASH Annual Meeting, “Sailing to Ithaca: Gene Therapy’s Odyssey from Investigational Agent to Therapeutic Product,” Dr. High discussed the evolution of gene therapy.

“I am honored to receive this award from the ASH, particularly because Professor Thomas was a pioneer in the development of a novel type of therapy, bone marrow transplantation, about which many people were skeptical in its early days, but which is now a well-accepted therapy for hematologic, oncologic, and genetic diseases,” Dr. High said.

Focus on Leukemia Research

Several leukemia-related research projects led by Children’s Hospital investigators were also featured at the meeting. Oncologist David Teachey, MD, presented his investigation of autoimmune cytopenias, including Automimmune Lymphoproliferative Syndrome, an inherited disorder that can lead to autoimmune issues and enlarged organs due to an accumulation of white blood cells. And Sarah Tasian, MD, an instructor in the Division of Oncology, presented her work on Philadelphia chromosome-like acute lymphoblastic leukemia (ALL). The most common form of leukemia found in children, ALL is largely curable, with a roughly 85 percent cure rate. However, the remaining 15 percent of ALL cases resist standard therapy.

Children’s Hospital’s Stephan A. Grupp, MD, PhD, co-chaired a special session on immunotherapy and gave a talk on his investigation of treating ALL with engineered T cells. Dr. Grupp, director of Translational Research for the Center for Childhood Cancer Research, in April published a study in The New England Journal of Medicine showing two leukemia patients achieved complete responses after receiving T cells engineered to selectively kill cancerous cells.

On December 7 at the ASH Annual Meeting, Dr. Grupp and colleagues presented follow-up results of their T cell clinical trial. Of the 24 pediatric and adult patients who were treated for ALL, 18 had ongoing complete responses at a median of 2.6 months after treatment.

One of Dr. Grupp’s patients, then 7-year-old Emily Whitehead, was the first pediatric patient to receive the engineered T cells in April of 2012. Though the treatment led to a life-threatening illness — known as cytokine release syndrome — Emily recovered after Dr. Grupp and his team were able to treat her symptoms. Since receiving the T cells, Emily remains healthy and cancer-free.

“Our results serve as another important milestone in demonstrating the potential of this cell therapy for patients who have no other therapeutic options,” said Dr. Grupp at ASH.

To read more about the 2013 ASH Annual Meeting, see the ASH website. To learn more about The Children’s Hospital of Philadelphia’s hematology and pediatric leukemia research, see the CHOP site.

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