The forefront of CAR T-Cell immunotherapy research for high-risk pediatric leukemia patients

Innovative research conducted at the Hyundai Cancer Institute at CHOC Children’s includes investigations into immunotherapy, which has emerged as an effective therapeutic modality for patients with hematologic malignancies. Chimeric Antigen Receptor (CAR) T-cell therapy is capturing the attention of the medical community as a prominent example of cellular immunotherapy and is often referred to as a “living drug.”

“CAR T-cell therapy has revolutionized the treatment landscape of relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) — a disease that was historically considered to have a very poor prognosis,” says pediatric oncologist Dr. Van Huynh, who leads the CAR T-cell program at CHOC “With this remarkable approach, we are able to harness the patient’s own immune system to potentially cure their leukemia.”

Dr. Van Huynh, pediatric oncologist at the Hyundai Cancer Institute at CHOC Children’s

CHOC was one of the first pediatric sites for CAR T-cell therapy clinical trials in 2015. Along with providing access to innovative clinical trials for CAR T-cell therapy and other immunotherapy, CHOC is now a certified treatment center for providing the recent U.S. Food and Drug Administration-approved CAR T-cell therapy called KYMRIAH. It is available to pediatric and adolescent/young adult (AYA) patients who are up to 25 years of age with second or later relapse or refractory (R/R) B-cell ALL or patients with R/R large B-cell lymphoma.

CAR T-cell therapy involves the collection of a patient’s own T-cells and genetic modification of these cells to express a CAR that can recognize a tumor antigen such as CD19 on leukemia cells. The CAR T-cells are then expanded in the lab and later re-infused back into the patient, where they continue to expand and seek out the target antigens to eradicate the leukemia.

CAR T-cell therapy has given hope to many patients who otherwise had no other treatment options. Furthermore, dramatic initial clinical responses and high rates of complete remission have been observed. Relapses can occur due to antigen loss (leukemia cells no longer express the target antigen) or CAR T-cell exhaustion (the CAR T-cells no longer provide protection).

“Currently, researchers are looking into ways to overcome these two barriers to make this therapy even more effective,” Dr. Huynh says. “I’m also excited for the potential of CAR-Ts to target other antigens besides CD19 and the possibility that it may be able to treat other types of cancer.”

Our Care and Commitment to Children Has Been Recognized

CHOC Children’s was named one of the nation’s best children’s hospitals by U.S. News & World Report in its 2020-2021 Best Children’s Hospitals rankings and ranked in the cancer specialty.

Learn how CHOC’s pediatric oncology treatments, expertise and support programs preserve childhood for children in Orange County, Calif., and beyond.

In the Spotlight: Rishikesh Chavan, M.D.

CHOC welcomed Dr. Rishikesh Chavan to the oncology department in January 2019. As the Medical Director of the Stem Cell Transplant and Cellular Therapy program, he works with young patients going through leukemia, lymphoma or other conditions requiring bone marrow or stem cell transplants. He attended medical school at Lokmanya Tilak Municipal Medical College in India, followed by a pediatrics residency at Tulane University School of Medicine and a Hematology/Oncology fellowship at Baylor College of Medicine. Subsequently, he served as the Medical Director of Stem Cell Transplant at Tulane University and Loma Linda University Children’s Hospital.

Dr. Rishikesh Chavan, Oncology

What are your special clinical interests?

Stem cell transplant and immunotherapy for high risk leukemias/lymphomas. I am also interested in reduced intensity stem cell transplants and gene therapy for immunodeficiencies, aplastic anemia and sickle cell disease patients.

What are some new programs or developments within your specialty?

Comprehensive whole patient care model involving psychology, nutritionists, social workers, child life specialists, pharmacists and care coordinators as an integrated team approach to improve the ease of transition between inpatient and outpatient care, to ensure patient satisfaction and compliance, and to achieve best possible outcomes—all while lowering the cost of care.

What would you most like community/referring providers to know about you or your division at CHOC?

We believe every patient coming to transplant deserves personal attention, and we strive to over-communicate with the referring providers to share updates about their patients’ health as well as
facilitate with transitioning the patients back to the referring providers as soon as the patients are ready to be followed by them.

What inspires you most about the care being delivered here at CHOC?

Healthcare delivery is going through a transformation, and CHOC is at the forefront of this healthcare transformation by bringing the latest evidence-based treatments as well as clinical trials to benefit our kids while simplifying the care they need.

If you weren’t a physician, what would you be and why?

I would be a farmer. I feel it is a skill that requires patience, selflessness and long-term thinking, and that the efforts put in by one generation are likely to benefit the next generation.

What are your hobbies/interests outside of work?

I volunteer at the local library to facilitate a Heartfulness Meditation group (a network of volunteer meditation coaches and a meditation app). I also like to spend time with my kids and dog at the park. When time permits, I try to go to the gym and play tennis. I also read a lot—nowadays audiobooks.

What have you learned from your patients?

Resilience. Given the patients we have, I can never really have a bad day. If I do feel I’m having a bad day, my patients’ situations give me perspective.