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Clinical Research

When you think of the life you can give by successfully treating a small child or young person who has cancer or a blood disorder, it's easy to understand the importance of pediatric cancer and hematology research.

When we say we really care for kids, we're not just talking about medicines and treatments. When our patients and their families come to us, medical treatment can last months and, in many cases, years. That means they become a part of our team ... and part of our family here at the medical center. They touch our hearts and our lives, and that pushes us even harder to continue to advance the research and technologies available to them.

Active studies

Hematology research studies

Oncology research studies

Stem cell transplant research studies

ASPHO 2020 poster presentations

Were you looking forward to ASPHO 2020? So were we. Unfortunately, ASPHO 2020 had to be cancelled, but the research goes on. As we look forward to ASPHO 2021, we continue to look ahead toward the development of research that advances and enhances care in pediatric hematology and oncology.

In the meantime, we’re pleased to share our ASPHO 2020 accepted posters.

View here

Listen to our researchers discuss their programs

Dr. Johnson with patient

Advanced Treatments and Research for Sickle Cell Disease

Dr. Clarissa Johnson, director of the Sickle Cell Program at Cook Childrens, talks about advanced treatments and therapies as well as promising new clinical research on the horizon for sickle cell disease. Her extensive knowledge and passion for treating patients with sickle cell disease is the driving force behind bringing relief to hundreds of patients at Cook Children’s.

Dr. Vallance with patient

Targeted Therapy for Pediatric Cancer

Dr. Kelly Vallance discusses how research through Cook Children's Targeted Therapeutics program is opening the door to the most advanced and promising clinical trials available to pediatric cancer patients—leading to higher response rates, less toxicities and more cures.

Targeted Therapy for Cancer

Dr. McCavit with hemophilia patient

Advanced Research and Treatments for Hemophilia

Timothy McCavit, M.D., from Cook Children's Bleeding Disorder Program and Hemophilia Treatment Center, discusses our participation in revolutionary clinical research that holds great promise for children and young adults with hemophilia. This new clinical research is currently the best available and in patients on preliminary drug trials, the treatments have been transformative and their quality of life has greatly improved. Listen to learn more about new breakthrough treatments for hemophilia patients.

New Era in Hemophilia

Dr. Heym with patient

Total Therapy Study XVII

Dr. Kenneth Heym Medical Director, Oncology Program, Cook Children's Medical Center, talks about our participation in a new Total Therapy XVII clinical research trial. This important investigational research is currently the best available targeted therapy for ALL and LLy cancers.

The Incredible Promise of Total Therapy XVII

Dr. Torres and Acosta reviewing brain images

BE FAST to recognize the signs of Pediatric Stroke

Cook Children's Stroke and Thrombosis Program co-directors Marcela Torres, M.D., Medical Director, Hematology Program, and Fernando Acosta Jr., M.D., Neurology, talk about the causes of pediatric stroke and the importance of the two specialties collaborating to improve awareness, diagnosis, treatment and risk of recurrence.

Unique Approach to Stroke Care

Dr. Zhao at microphone

Innovations in Pediatric Neuro-Oncology Research and Treatments

Sibo Zhao, M.D. Medical Director, Cook Children's Neur-Oncology program, discusses the challenges of pediatric brain and spinal tumors, and the research and clinical trials that are transforming outcomes for pediatric patients, including developmental therapeutics and anti-cancer care.

Dr. Pacenta

CAR T-cell therapy

Dr. Holly Pacenta Cook Children’s hematologist/oncologist, Cellular Immunotherapy Program, introduces CAR T-cell therapy, a targeted treatment now available for patients with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).

Stem cell transplant research studies

For many years cancer research and treatment focused on adults, but children have very special needs because their bodies are still growing and changing. What works for adults doesn't always work for kids, sometimes resulting in side effects and secondary conditions later in life. As an active research facility, we are at the forefront of treatments for kids from prenatal through early adulthood. This lets us work closely with each patient and the families to look at the options and advanced care–from genetics to stem cell transplants to targeted therapies–that otherwise might not be available to them.

Working together

Not only do we build relationships with a global network of leading researchers, we also partner with our patients and parents starting on day one of the diagnosis; and it is only in the context of that relationship that we introduce the subject of research. Parents do not want to expose their children to risks, but most of them come to understand the concept of clinical trials. We explain how our clinical research has advanced the field of medicine and how it contributes to our cure rates here, as well as advances in oncology and hematology.

Our approach

Children's Oncology Group (COG)

The goal of Children's Oncology Group (COG) members is to cure all children and teens with cancer, reduce the short- and long-term complications of cancer treatments, and determine the causes and find ways to prevent childhood cancer. Learn more about the COG and what it means to be a member here.

Although Cook Children's is not a traditional academic center affiliated with a university, we are academic in how we approach research and practice day in and day out. As a key collaborator with the nation's leading pediatric research organizations, our team offers the best available treatments and contributes to the search for a cure.

Cook Children's is a member institution or has received accreditation from several nationally-known cancer and blood disorder organizations for conducting research trials including:

  • Children's Oncology Group (COG).
  • New Approaches to Neuroblastoma Therapy (NANT).
  • Therapeutic Advances for Childhood Leukemia (Phase I TACL).
  • Center for International Bone Marrow Transplant Research (CIBMTR).
  • Clinical Transplant Network (CTN).
  • International Pediatric Stroke Society (IPSS).
  • Regional Hemophilia Treatment Center.
  • National Marrow Donor Program (NMDP).
  • Many national and international patient registries.

We have also collaborated with several recognized research institutions including St. Jude Children's Research Hospital, Texas Children's Hospital as well as other high-performing regional academic institutions.

Not every cancer or blood disorder requires treatment through a current clinical trial. In fact, there are many cases that we can now treat and even cure with broad scope solutions, but those available treatments exist because of ongoing research. However, there are still many cancers and blood disorders that are rare or challenging for which there are no cures. For the children who are diagnosed with them, research often provides the best chance of recovery. It is for that reason that we are involved in research and our continued pursuit of a cure for each and every child. And we won't stop until we find a way to make childhood carefree and cancer free for all kids, now and in the future.

Clinical trials

Many successful treatments have been made possible by research. The survival rate for childhood cancers has improved, in great part, due to clinical trials. In fact, many young cancer patients receive treatment by participating in clinical trials. In fact, 60 percent of cancer patients under the age of 29 are enrolled in trials.

What is a clinical trial?
Clinical trials are the final step in a long process of research. For many patients with cancer, especially children, they are the first step toward beating their disease. Clinical trials test new ways to find and diagnose cancer, treat cancer, manage symptoms of cancer and side effects from treatment and, ultimately, to prevent cancer.

Who should participate in a clinical trial?
If your child is diagnosed with cancer, clinical trials are an option you should discuss with your doctor. Many people believe that clinical trials are only for patients who are in the final stages of the disease or who aren't responding to treatments. But the truth is, there are clinical trials available for every stage of cancer.

What clinical trials are available?
Our team is currently active in clinical trials through many venues. Cook Children's must meet the guidelines of each group we partner with as well as successfully pass routine audits and site visits to be accepted and maintain privileges. The groups we currently partner with include:

New approaches to neuroblastoma therapy (NANT)

In 2008, Cook Children's was selected to join the New Approaches to Neuroblastoma Therapy (NANT) consortium, an organization comprised of just 15 hospitals throughout North America specializing in neuroblastoma research. NANT attempts to advance new therapies by conducting select early-phase clinical trials.

As a NANT site, Cook Children's actively participates in research on neuroblastomas in children. We are part of a very tightly knit group of expert physicians, researchers and investigators from 15 select university and children's hospitals that make up the consortium. Together, we are working continuously to test new therapies for high-risk and relapsed neuroblastoma and to advance neuroblastoma treatment for kids.

Presently, Cook Children's offers clinical trials targeting neuroblastoma cells that are resistant to established chemotherapy and radiation. This research is focused on finding treatments for otherwise hopelessly advanced neuroblastoma.

Participating in a clinical trial

Clinical trials offer patients new approaches to treatment or drugs, as well as the advantage of a therapeutic approach that has been mapped out and reviewed by a team of investigators who specialize in the disease being studied – in this case, neuroblastoma.

Generosity. It helps to cure what ails you

Thanks to the generous support of our donors, and our NANT participation, children like Hayden can now experience their own miracles closer to home, as Cook Children's new cancer center includes an MIBG suite.

I-131 MIBG therapy is still considered a clinical trial. But for children with high-risk recurrent neuroblastoma, it may be the preferred course of treatment. This relatively new treatment enables the medical team to deliver radioactive iodine (RAI) therapy directly to the tumor cells without disturbing any of the body's healthy cells. Currently offered only through research trials, Cook Children's is one of only a dozen or so centers in the country to offer the this therapy for children.

NANT is supported by the National Cancer Institute. If you would like to learn about NANT, and the research that is being done for neuroblastoma patients, visit for more information.

For information on sponsored clinical research trials, collaborative group efforts, quality improvement and investigator-initiated research, please email us at or call 682-885-2103.

The Children's Oncology Group (COG)

The Children's Oncology Group joins together some of very brightest minds from around the world, all intent on understanding the causes of cancer so that we can cure cancer. We are proud to be a recognized COG cancer center ... not only for the research that we do, but for the treatments and cures we bring to the patients and families we treat here at Cook Children's.

Formed in 2000, Children's Oncology Group (COG) is the nation's largest and most established cooperative children's cancer research network for pediatric cancer research. It brings together treatment centers, physicians, laboratory scientists, nurses, psychologists and others working to beat cancer in children, adolescents and young adults. And Cook Children's is proud to be an active member, fighting cancer in kids every day.

Global collaboration
The COG unites more than 8,000 experts in childhood cancer at more than 200 leading children's hospitals, universities, and cancer centers across North America, Australia, New Zealand, and Europe in the fight against childhood cancer. Through this collaborative research, the Children's Oncology Group has improved rates for children's cancer at a pace much faster than any one individual or single institution could accomplish alone.
The Children's Oncology Group research has turned children's cancer from a virtually incurable disease 40 years ago to one with an overall cure rate of 78 percent today.

Comprised of 210 treatment centers in North America, Europe, Australia, New Zealand and South Africa, COG includes more than 6,500 individual members.

Cook Children's is proud to be an active participant in these many accomplishments because they have a positive effect right here at home. And they help us to fulfill our promise to improve the health and lives of every child in our region – and beyond.

To learn more about COG, visit

For information on sponsored clinical research trials, collaborative group efforts, quality improvement and investigator-initiated research, please email us at or call 682-885-2103.

We are here to help.

If your child has been diagnosed, you probably have lots of questions. We can help. If you would like to schedule an appointment, refer a patient or speak to our staff, please call our offices at 682-885-4007.