Research and Clinical Trials

As one of the leading Cancer Centers in the United States, we are at the forefront of developing new treatments and designing clinical trials. Each year, more than 12,000 children and teens are diagnosed with cancer ranging from leukemia to sarcoma. Most of them will be cured and go on to lead healthy, productive lives because of innovative research and cooperative clinical trials. As a leader in pediatric cancer research, we are dedicated to making new discoveries to improve the lives of children with cancer in Maryland, the nation, and around the world.

Childhood Cancer Clinical Trials

To search for childhood cancer clinical trials, please search our clinical trials database. Choose from the drop down menu called Pediatric Cancers.

Personalized Approaches in Pediatric Oncology

"The potential to save decades of life" ...Donald Small, Director of Pediatric Oncology and his team are working on behalf of the youngest victims of cancer.

Research Milestones in Pediatric Oncology Discovery and Innovation

Genetic Culprits

Pediatric Oncology director Donald Small was the first to identify and clone the FLT3 gene.  Mutations to this gene are linked to treatment-resistant types of childhood leukemia, including infant leukemia.  Our pediatric oncology team has identified drugs that block or inhibit FLT3 function and are now directing multi-institutional clinical trials.

Brain Cancer Discoveries

Brain cancers are among the most common cancers in children. Johns Hopkins scientists in the Vogelstein/Kinzler/Velculescu laboratory of the Kimmel Cancer Center linked mutations two genes, IDH1 and IDH2, to nearly three-quarters of the most common types of brain cancers known as gliomas.  Patients with certain tumors that carry these genetic alterations appear to survive at least twice as long as those without them. They believe further research on the genes could also lead to more precise diagnosis and treatments.

Kenneth Cohen, clinical director of Johns Hopkins Pediatric Oncology,  and colleagues developed an “up-front” radiation therapy for pediatric brain tumors, now widely adopted by other cancer treatment facilities . Dr. Cohen also is studying the first use of arsenic given in combination with radiation therapy in children with high risk brain tumors.  This treatment is part of an overall program to develop novel therapies for children with poor prognosis brain tumors.

Pilomyxoid Astrocytoma (PMAs) is a type of brain cancer largely seen in children and was first identified by Hopkins pathologist Peter Burger, M.D., and colleagues.  To further advance research and treatment of PMA, we invite adults or children who have been diagnosed with PMA to join the Johns Hopkins PMA Registry, a resource for patients and physicians alike supervised by Dr Cohen.

New Anticancer Drug

The Kimmel Cancer Center led a multi-institutional study of an experimental drug that blocks the cancer-associated hedgehog gene pathway. The drug showed impressive activity in medulloblastoma brain cancer and led to clinical trials of the drug in children with medulloblastoma.

Bone Marrow Transplant

Our Center was a pioneer in bone marrow transplant therapy.  In 1995, we established one of the first pediatric bone marrow transplant programs. Today, we are again leading the field with development of haploidentical, or half identical, bone marrow transplants.  This form of transplant allows patients who do not have an identical immune system match to undergo potentially curative bone marrow transplants with a half-matching parent or sibling has a marrow donor.  Our investigators have learned how to manage immune complications—even making them work to their benefit against the cancer. Recent finding reveal half-identical transplants may actually produce better results than standard, perfectly-matched transplants. The Kimmel Cancer Center is the only cancer center performing half-identical bone marrow transplants.

Pediatric Oncology Fellowship Program 

The fellowship program is a joint endeavor of Johns Hopkins and the National Institutes of Health. It is designed to provide clinical and research exposure to develop and train the next generation of specialists in pediatric cancer laboratory and clinical research and patient care.

Pilomyxoid Astrocytoma (PMA) Registry 

PMA, studied and defined by Johns Hopkins researchers as a distinct type of glioma, is a form of brain cancer most commonly seen in children. Despite its growing recognition, little is known about the tumor. Researchers will use data gathered in the registry to help refine current therapies and to develop new ones.

Isolating Blood Stem Cells

Our researchers developed the technology to isolate the rare blood stem cell from within a sea of hundreds of billions of blood cells.  The blood, or hematopoietic stem cell controls the development of all other blood and immune cells. The discovery has led to better diagnostics in blood-born cancers such as leukemia and blood stem-cell based therapies.

Inducing Cancer Cell Death

Pediatric oncology researchers identified a gene, called PASG, that when altered causes cells to age prematurely and die.  In order to grow and stay alive, cells depend on PASG to reduce the activity of other genes.  Researchers are studying agents that block the function of the gene as a way to force cancer cells into an early death.

Preventing and Managing Late Effects

As cure rates improved for children with cancer, experts began to recognize treatment-related side effects. The Kimmel Cancer Center established one of the first childhood cancer survivors programs in the nation to study, monitor, treat, and develop methods to prevent long-term complications of cancer therapy.  These late effects include infertility, cardiac toxicities, hyperthyroidism, and developmental and cognitive delays.

Acute Lymphocytic Leukemia (ALL)

ALL is one of the most common types of pediatric cancers. By defining specific subtypes of the cancer and altering the administration of chemotherapy, Kimmel Cancer Center researchers improved cure rates to nearly 90 percent.

Immune Therapy for Childhood Cancers

With their low toxicities, immune therapies seem tailor-made for pediatric cancer patients, where long-term effects caused by conventional therapies are of great concern. 

New research shows Hodgkin’s lymphoma may be one of the best responders so far to anti-PD-1 immune checkpoint blockade therapy, with response rates approaching 90 percent. Pediatric oncology experts say there is emerging evidence that immune therapies may be effective against chemotherapy-resistant cancer.

Pediatric oncology scientists are discovering crucial links between T cell behavior, the main cells activated in an immune response, and the epigenetic or chemical environment of T cell DNA. Although the DNA code of a T cell that has never been activated is identical to that of T cells engaged in an immune attack, significant changes occur in the chemicals that surround the DNA that help signal it to remain dormant or go into action. As a result, the scientists are deciphering the normal epigenetic activity of immune T cells and exploring whether existing epigenetic-targeted treatments might be able to improve immune responses to cancer. 

The scientists believe epigenetic drugs may augment the effectiveness of other immune treatments, including cancer vaccines and immune checkpoint blockers such as anti-PD-1 drugs. They are also looking for other proteins expressed by tumor cells that work like PD-1 to shut down an immune response to cancer.  In addition, they are working on ways to use PD-1 blockade and other similar immune agents to fight pediatric cancers.


Promise & Progress: Immune Therapy for Childhood Cancers