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Interview with Donald Small, M.D., Ph.D., Director of the Division of Pediatric Oncology

Donald Small, M.D., Ph.D., director of the Division of Pediatric Oncology
Donald Small, M.D., Ph.D., director of the Division of Pediatric Oncology
Donald Small, M.D., Ph.D., director of the Division of Pediatric Oncology

How would you describe our Division of Pediatric Oncology, its makeup and mission?
Our division in the Kimmel Cancer Center is composed of clinicians and physician scientists, all of whom are actively doing clinical research or basic science research in the lab. Our overarching aim is to forward the state of the art of cancer care for children through research that makes life better, not only for our patients but also for children throughout the United States and around the world who are afflicted with cancer.

And how would you distinguish the role of nurses and other staff in the division?
All of our nurses are strictly pediatric oncology nurses, who have become experts on the side effects that therapy can entail. They make sure chemotherapy is done in as safe a manner as possible. We also offer a team approach around a patient-centric model in which every child has a primary faculty member, fellow, social worker and primary nurse on both the inpatient and outpatient side. Our team also includes child life specialists, nutritionists, pharmacists, psychologists and social workers. Taking care of children with cancer is very labor intensive, so we try to provide as much support as possible. Our families tell us they feel supported.

In what ways?
They appreciate that we do family rounds after we do our separate clinical rounds with the entire team. Also, having a child with cancer is a huge stress on the family structure; children, adolescents and young adults worry about whether or not they will be cured. Anna George, a psychologist, has dramatically improved our services for patients and families in helping them address issues like anxiety and depression. Our child life specialists and social workers also help families facing these issues.

In the cancer research arena what are our strengths and goals?
We are already very strong in leukemia research, so right now our focus is on building up our brain tumor program, with brainstem tumor researcher Eric Raabe, and our sarcoma research program headed by Christine Pratilas, who has been working on inhibiting some of the pathways in sarcoma and other solid tumors. We are also putting more effort into harnessing the immune system to directly target malignant tumor cells in treating diseases like acute lymphocytic leukemia, thanks to investigators like Brian Ladle and Nicolas Llosa. We are trying to get away from the era of chemotherapy and replace it with directly targeted mutations or mutated pathways in liquid or solid tumors.

Why the focus on immunotherapy?
Because it means less in terms of short- and long-term side effects than chemotherapies, which are basically poisons that kill rapidly growing cells. We have been fairly successful with chemotherapies, but we’ve gone about as far as we can with them. Immunotherapy tends to be more tolerable.

Other research highlights?
Heather Symons has pioneered haploidentical, or half-matched, bone marrow transplant here to combat severe graft versus host disease in the adult population. She has very rapidly brought that approach into the pediatric setting, as well. This is important because family size is smaller now, and we are increasingly a nation of minorities, who are underrepresented in bone marrow registries. Haploidentical transplants have enabled us to stop turning away kids for bone marrow transplants. As long as someone has a parent or sibling, he or she now has a match. We are also using haploidentical transplant as a platform for solid tumors and even non-cancerous diseases.

Non-cancerous conditions?
Yes, with haploidentical transplants we are able to do bone marrow transplants for aplastic anemia, sickle cell and thalassemia, which we tend to see in our patients from the Middle East. We are also hoping to use this haploidentical platform as a way to do organ transplants. If a parent donates a kidney or part of their liver to their child, that child normally would have to take lifelong anti-rejection drugs, which eventually results in the organ being destroyed by its own immune system. However, if you can transplant the parent’s immune system at the same time as the organ, the parent’s immune system does not see the organ as foreign and does not try to reject it. The idea is to get patients off immune suppressants much more quickly and have the organ potentially last them their lifetime.

To what do you attribute the success of our research enterprise?
Key to all of our research efforts is having a critical mass of investigators to collaborate and build on each other’s work. That is why one of my goals when I took over as division director in 2006 was to increase the number of physician scientists. At that time, we had around 12 faculty members—now we have 21. Moreover, their work has brought us success in getting outside funding from both the National Cancer Institute and private and corporate foundations supporting cancer research, such as The St. Baldrick’s Foundation, Alex’s Lemonade Stand and Giant Foods.

Any closing thoughts?
Just that we all feel like it is a real privilege to take care of these kids. We see them so many times, from diagnosis to the end of treatment, and they keep us going. In addition, the children who relapse and die keep us re-energized in the lab to keep working hard to improve the cure rate for cancers we do not have much success with, and to do away with the long term side effects by developing more targeted therapies. We really have a great team, and we all show the families we do not forget them.

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