Clinical Trials Continue the Advancement of Pediatric Oncology Treatment

Children, adolescents and young adults with cancers that do not respond to traditional treatments continue to find new treatment options because of CHOC’s extensive efforts and active engagement in clinical trial research.

The Hyundai Cancer Institute at CHOC Children’s is a member of the Children’s Oncology Group (COG) and one of only 21 elite facilities in North America and three in California that has received a prestigious Phase 1 clinical trial designation to offer COG’s investigational, potentially promising and innovative clinical trials. COG is the most experienced organization in the world when it comes to the research and development of new therapeutics for children and adolescents with cancer.

“I’ve witnessed the dramatic progress made in the survival of our pediatric patients because of clinical trials,” says Dr. Ivan Kirov, medical director of oncology and the Hyundai Cancer Institute at CHOC Children’s. “Clinical trials are the mortar behind our successes here at CHOC.”

Dr. Ivan Kirov, medical director of oncology and the Hyundai Cancer Institute at CHOC Children’s
Dr. Ivan Kirov, medical director of oncology and the Hyundai Cancer Institute at CHOC Children’s

CHOC currently offers more than 140 clinical trials in varying phases, including multiple pharmaceutical industry-sponsored clinical trials. Besides membership in COG, CHOC is also a member of the Therapeutic Advances in Childhood Leukemia & Lymphoma consortium (TACL), which offers novel treatments in Phase 1 studies for childhood leukemia and lymphoma; the Lymphoma consortium; and the UC Children, Adolescent and Young Adults Cancer Consortium, which includes all of the University of California pediatric oncology programs.

Among the research at CHOC is an upcoming clinical trial for the treatment of diffuse intrinsic pontine glioma (DIPG), a highly aggressive and one of the most difficult-to-treat childhood tumors.

“We are in the process of opening and initiating this clinical trial which, in my opinion, will be extremely important for patients in the future,” Dr. Kirov says. “DIPG is a brainstem tumor which is universally deadly, and very few patients survive more than a year, or even six months. CHOC, the Dana-Farber Cancer Institute in Boston and Lurie Children’s Hospital in Chicago are the only three sites where this new study will be offered. This study will explore a new vaccine for the treatment of DIPG in combination with checkpoint inhibitors. We’re hoping this study will be open in the next several months to offer hope to patients with this disease.”

While clinical research is fundamental to advancing pediatric oncology treatments, Dr. Kirov said the trials themselves are only part of CHOC’s comprehensive approach to helping children and young adults survive cancer.

“These cutting-edge medications and products we are testing, including new targeting agents, monoclonal antibodies and various types of small molecules and vaccines, for example, require an extremely strong supportive and clinical research infrastructure, which CHOC can offer,” says Dr. Kirov. “Our highly educated clinical research coordinators, physicians-scientists, nurses, educators, pharmacists, and other professionals, along with our unparalleled supportive services for both patients and their families, such as social workers, psychologists, child life specialists, palliative care experts and spiritual services, make our patients’ experiences at CHOC unique, and I think this is why CHOC truly stands out. In fact, many patients who come to CHOC for Phase 1 studies express their desire to stay here even after the study is completed, which really speaks very highly of CHOC and the continuum of care and support we provide to young patients and their families.”

Our Care and Commitment to Children Has Been Recognized

CHOC Children’s Hospital was named one of the nation’s best children’s hospitals by U.S. News & World Report in its 2020-21 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.

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.

Avoiding burnout: A physician shares self-compassion strategies for provider wellness

By Dr. Rishikesh S. Chavan, pediatric oncologist and medical director of the blood and bone marrow transplant program at the Hyundai Cancer Institute at CHOC Children’s

Navigating the COVID-19 pandemic and a rapidly evolving medical landscape has put added pressure and stress on the healthcare workers braving the front lines. This is why it’s more important than ever to recognize signs of burnout in yourself and your colleagues, and essential that we as physicians practice self-compassion.

As physicians juggle their position as a healthcare provider, possibly the head of their household, and many other roles, it can be almost unnatural for physicians to think of themselves and their own needs.

But as the saying goes, you cannot pour from an empty cup. Physicians can’t do their best to care for patients and their own children at home unless they’re supporting their own comfort. I liken it to the safety instructions on an airplane – put your oxygen mask on before assisting others.

Self-compassion tactics

To avoid self-sabotage or self-destructive tendencies, one needs to feel a deep sense of love and acceptance of themselves. That’s why practicing self-compassion is so important. Here are some ways to get started:    

  • Make your own checklist Similar to a checklist one uses at the end of a shift to transition cases, make a personal one to end or begin your day. Everyone’s checklist will look different. We each classify different habits or rituals as essential. Do you need your morning coffee to function best? If you have exercised are you then your best self? For your checklist, consider elements such as:
    1. Acknowledge something that was difficult during your shift. After the feelings come up, let them go.
    2. Name three things that went well.
    3. Did you notice anyone else have a particularly hard shift? Check on them.
    4. Check in with yourself. Are you OK?
    5. Rest and recharge.
  • Relax – Identify your strategy for relaxation to help you take your mind away from the daily grind. Be aware of self-compassion versus self-indulgence. For example, watching an episode of your favorite TV show is one thing but binge watching an entire season is another. For several people, activities like bike riding, working in the garden, reading a book, practicing a musical instrument, or taking a yoga class may help establish a state of flow and provide an opportunity to go deeper.
  • Meditate – By definition, meditation means to focus on something. As you gently have a subtle focus on your heart, you can be a silent observer of your thoughts without reacting to them. An assumption that you are not your thoughts allows you to ignore intrusive thoughts and achieve a sense of peace. Sitting quietly with a guided meditation via apps such as Heartfulness, Headspace or Calm may help you get started. Studies have shown that peace and tranquility rank among the most common feelings people report after meditating, in whatever modality suits them.
  • Check in with your colleagues –Not only should we check in with ourselves, but we should check on our colleagues as well. If you see signs of burnout in a colleague, gently bring them into a conversation, or bring them a cup of coffee, and ask, “Is everything OK? Is there something that you want to talk about? Can I help you with anything?”

Interventions for physician burnout

A 2017 JAMA study found that the strongest evidence for effectiveness in combating physician burnout was organization-directed interventions, but the study noted such programs were rare. Most interventions for physician burnout put the onus back on the physician, with a focus on incentivizing physicians to participate. More effective intervention models are engrained across an entire hospital or healthcare system.

In January 2018, CHOC convened a Physician Wellness Subcommittee, composed of a group of physicians dedicated to help CHOC continue to be proactive and supportive of physicians. Its mission is “To promote physician wellness to benefit ourselves and others.”

Additionally, at CHOC, the spiritual care team offers regular “Tea for the Soul” sessions where chaplains are available to clinicians and provide a compassionate, non-anxious, non-judgmental presence to help them cope with added stressors.

CHOC leadership has taken other steps to provide additional support for its physicians and staff, recognizing the additional stressors placed upon CHOC staff during COVID-19. CHOC’s on-site daycare was set up within 72 hours, giving clinicians peace of mind that their children are safe and happy while they work. Recognizing that shopping for groceries and sundries might be challenging for staff, CHOC has set up in-house shopping resources, as well as a grab-and-go meal program and farmers market.

Fertility future with testicular cryopreservation

Fertility preservation is now a reality for male pediatric and adolescent/young adult (AYA) patients with cancer or blood disorders.

The Hyundai Cancer Institute at CHOC Children’s is a center of excellence and the only program on the West Coast offering testicular preservation for prepubertal patients unable to bank sperm. Testicular tissue is biopsied and preserved in liquid nitrogen.

“Recent research indicates these biopsies contain stem cells, leading to the possibility of generating sperm in the future,” said Dr. Carol Lin, a pediatric oncologist at the CHOC Hyundai Cancer Institute.

Evidence-based research

CHOC participated in an eight-year study alongside U.S. and Israeli institutions to establish a standardized protocol and centralized process to freeze and collect biopsies.

A CHOC Children's pediatric urologist speaks with an adolescent male patient in an exam room

“In total, 189 patients provided samples. A quarter of each sample was used for research and the rest stored for future use. These patients ranged from ages 5 months to 34, with an average age of about 8,” said Lin.

The study discovered that a centralized process and testicular biopsies from multiple sites was a viable option and could accelerate recruitment. Click here to learn more about the study.

Sharing expertise

CHOC is a member of the Oncofertility Consortium, a group of scientists and providers who are committed to fertility preservation in cancer patients. The consortium studies the science of cryopreservation methods, storage of tissue, in vitro follicle growth and communication between patients and doctors, as well as ethical and legal concerns.

Our care and commitment to children has been recognized

CHOC Children’s Hospital was named one of the nation’s best children’s hospitals by U.S. News & World Report in its 2020-21 Best Children’s Hospitals rankings and ranked in the cancer specialty.

Best Children's Hospitals, U.S. News & World Report, Cancer, 2020-21

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

Testicular Cryopreservation bringing hope to CHOC cancer patients

Young male patients undergoing treatment for some cancers or blood disorders at CHOC Children’s have another hope for fertility preservation.

The Hyundai Cancer Institute at CHOC Children’s is the only program on the West Coast offering testicular cryopreservation as an option for young male patients unable to bank sperm but who would like to take steps to preserve future fertility.

Under the program, testicular tissue is biopsied and stored in liquid nitrogen at temperatures close to absolute zero for future use. Recent research indicates these biopsies contain stem cells, leading to the possibility of generating sperm in the future, says Dr. Carol Lin, an oncologist at the Cancer Institute, and Lisa Klimpel, an oncology nurse practitioner.

CHOC was among a group of U.S. and Israeli institutions to participate in an eight-year study wherein a network of centers used standardized protocol and centralized processing and freezing to collect and store biopsies.

In total, 189 patients provided samples. A quarter of each sample was used for research and the rest stored for future use. These patients ranged from ages 5 months to 34, with an average age of about 8.

Recently published in the journal Human Reproduction, the study, of which Klimpel is a co-author, found that centralized processing and freezing of testicular tissue from multiple sites is feasible and could accelerate recruitment.

A generation ago, cancer survivors had few options to have biological children. Today, many solutions exist, however many oncologists, despite these advancements, still don’t discuss fertility preservation with their patients, particularly young patients. 

At CHOC, however, discussions about fertility preservation with cancer patients early into diagnosis and treatment are a matter of course, as the program works to ensure a meaningful survivorship, says Dr. Lin and Klimpel.

Learn more about referring to the Hyundai Cancer Institute.