New Pedi BMT and Immunotherapy Program

July 19, 2016 by

Please briefly summarize your program.
Doctors have been using the immune system to fight cancer for decades in the form of bone marrow or hematopoietic stem cell transplants (HSCT).  Many children with leukemia or lymphoma and even solid tumors such as neuroblastoma require a HSCT for a chance at a cure.  More recently, HSCT has been used for children with life-threatening immunodeficiencies and even severe sickle cell disease.  I have been tasked with establishing a HSCT program here at UVA for children with the goal of never having to send a child to another state for their transplant.

But, the immune system can now be harnessed in a different, and perhaps more effective way. T cells are part of the immune system that normally help us fight off infections. Through genetic engineering, we can now re-educate patients’ T cells to recognize and kill their tumor. Chimeric Antigen Receptor (CAR) T cell therapy, as it is called, has been shown to be very effective in early studies at the NIH, which I led. In a few months, children and young adults with the most common type of childhood leukemia will be able to enroll on a ground-breaking clinical trial of CAR T cells here at UVA.  Also, through a collaboration with Dr. Larry Lum we will be opening another trial for children with relapsed neuroblastoma where antibodies are used to redirect the patient’s T cells to their tumors. These types of modern immunotherapies for cancer is an exciting and exploding field and will complement our new HSCT program.

How do you define innovation?
Innovation can mean many things to different people. In the context of my job as a pediatric oncologist, innovation is taking what we know about a system and redesigning or sometimes even redefining it in such a way that a new, more powerful tool is created. The best innovations are those that make the greatest impact in the shortest amount of time.

How do you apply that each day?
CAR T cells are a great example. A CAR is the marriage between the tumor specificity of an antibody with the inherent and incredible power of the T cell. These were two completely separate parts of the immune system that were known for decades. But, in combination they are more powerful than either alone.

There is no reason, though, that a CAR has to be based on an antibody. If you can find a system where one molecule specifically interacts with a second, and at least one of those is on a tumor then you can make a CAR with that. Every day I try to think of new ways of improving the targeting of CARs or managing their side effects and how I can test that in my research laboratory quickly so that we can take those discoveries back to the clinic as rapidly as possible.

What makes your program stand out?
UVA currently does not have a HSCT program for children. We will be establishing this standard-of-care therapy here so that no child will have to leave the Commonwealth for this life-saving procedure. But, we will also be providing a paradigm-changing treatment for children with relapsed leukemia in the next month or two in the form of CAR T cells. Only a few hundred children throughout the world have been able to receive this cutting-edge therapy to date. UVA will be the only center to offer this therapy in the areas between Ohio, Missouri, North Carolina and Pennsylvania. My laboratory work will serve as a pipeline for more CAR therapies for other tumors.

What do you feel is the most exciting part of your program?
Through new efforts like the HSCT and Immunotherapy Programs at UVA, we will soon be poised to provide the most advanced care to children with cancer and other life-threatening diseases.  I am so excited to be a part of building these programs so that we can bring new treatments to children as rapidly as possible.


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