Senior Consultant, Pediatric Hemato-Oncology and Bone Marrow Transplant, Artemis Hospitals, Gurugram, India
Part 5 of 11 in Childhood Cancer: The Journey from Despair to Durable Survival
Brain Tumors in Children: Survival, DIPG and the Rise of Liquid Biopsy
August 27, 2026
A relatively favorable tumor, with one major exception
Brain tumors are the second most common pediatric cancer after leukemia. The World Health Organization recognizes roughly 25 distinct types, though in practice gliomas, medulloblastoma, ependymoma and embryonal or germ cell tumors of the brain account for most cases. Overall survival runs 70 to 80%, a figure that would be considered excellent in most oncology. The exception is diffuse intrinsic pontine glioma, or DIPG, where survival sits at around 5%, because the tumor forms in a part of the brainstem where surgery is not possible.
The pediatric oncologist as the coordinating role
High-risk medulloblastoma illustrates why treatment sequencing matters as much as the individual therapies. Adding carboplatin chemotherapy to radiotherapy raises the chance of success by 20 to 25 percentage points, a decision that sits with the pediatric oncologist coordinating the surgeon, the radiation oncologist and the chemotherapy protocol together. A study published in the New England Journal of Medicine found roughly an 11% response rate with chemotherapy alone in a specific medulloblastoma molecular subgroup, compared with 40 to 50% when targeted therapy was matched to that subgroup's genetics.
Diagnosing tumors that surgery cannot reach
For DIPG and similarly located tumors, liquid biopsy, testing cerebrospinal fluid for circulating tumor DNA, can diagnose the tumor and identify a druggable target without surgery. Gliomas carrying a BRAF or MEK mutation that recur or resist chemotherapy can often be controlled with oral targeted drugs, dabrafenib or trametinib. Dr. Danewa noted these remain expensive for now, but expects costs to fall as generic versions reach the market.
He described one patient whose tumor occupied a large part of the frontal and temporal lobe. Genetic testing identified an NTRK mutation, and targeted therapy against it was started. Diagnosed in 2022, the tumor had almost disappeared by 2026, monitored with periodic blood tests and MRI scans every six months, with no meaningful side effects from the treatment itself.
This guide is based on a live Jivo Masterclass — Dr. Arun Singh Danewa taught doctors across Africa on March 8, 2026.
FROM THE LIVE Q&A
Dr. Ivan Ipavu, Uganda
Can you briefly touch on the role of stem cell transplant in pediatric care?
Dr. Arun Singh Danewa
Dr. Danewa explained that upfront bone marrow transplant has no role in most pediatric leukemia. It is reserved for cases without morphological remission (blasts above 10% at the end of induction), for hypodiploidy (fewer than 44 chromosomes), or for relapse. For benign conditions, the leading indication, especially in Africa, is sickle cell disease, where earlier transplant, ideally before age 12 and before pain crises, stroke or chest syndrome accumulate, gives a better outcome. He described three transplant types: matched sibling, matched unrelated donor via registry, and haploidentical transplant from a parent, with haploidentical success running around 70 to 80% depending on the underlying disease.
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Frequently Asked Questions
Other than cancer, do you offer bone marrow transplant for disorders like sickle cell disease, and is there a cure?▼
Yes. Dr. Danewa confirmed bone marrow transplant is a cure for sickle cell disease, and that outcomes are best the earlier it is done, since delay allows organ damage and comorbidities to accumulate and lowers the chance of success.
How effective is CAR T-cell therapy?▼
In relapsed or refractory leukemia, where prior treatment options offered only a 5 to 10% chance of success, both Western data and four to five years of follow-up on India's own indigenous CAR T-cell programs are now showing 50 to 60% success.
For laymen, where does CAR T-cell therapy sit compared to chemotherapy, immunotherapy and targeted therapy? Is it tailored to the tumor's genetic makeup?▼
Dr. Danewa explained that CAR T-cell therapy targets surface antigens on the cancer cell itself, such as CD19 or CD22 in ALL: a patient's own T cells are removed, engineered to recognize that antigen, and returned to attack the cancer directly. Because cancer cells can develop antigen escape by losing one target, dual CAR T-cell therapy now targets CD19 and CD22 together, and similar antigen-targeted approaches (anti-GD2) are being developed for neuroblastoma and, increasingly, for brain tumors.
What are the risk factors for pediatric cancer, the way smoking and alcohol are known risk factors in adults?▼
Only about 1% of pediatric tumors are familial or have an identifiable genetic cause. Most arise from spontaneous mutations that the body's own immune checkpoints fail to catch. There is no equivalent of smoking or alcohol as a modifiable risk factor in children, and no established viral cause, so there is currently no basis for a preventive vaccine.
Can you recommend a textbook on pediatric cancers that doctors can follow?▼
Dr. Danewa recommended Nathan and Oski's Hematology and Oncology of Infancy and Childhood as the primary reference, alongside Lanzkowsky's Manual of Pediatric Hematology and Oncology, and offered to share his own treatment protocols and presentations directly with any doctor who requests them.
Why is DIPG survival so much lower than for other pediatric brain tumors?▼
Overall pediatric brain tumor survival runs 70 to 80%, but diffuse intrinsic pontine glioma, or DIPG, survival sits at around 5%, because the tumor forms in a part of the brainstem where surgery is not possible.
How can a tumor like DIPG be diagnosed without surgery?▼
Liquid biopsy, testing cerebrospinal fluid for circulating tumor DNA, can diagnose the tumor and identify a druggable target without surgery.
What does adding chemotherapy to radiotherapy do for high-risk medulloblastoma?▼
Adding carboplatin chemotherapy to radiotherapy raises the chance of success by 20 to 25 percentage points in high-risk medulloblastoma.
Can gliomas with a BRAF or MEK mutation be controlled without further surgery?▼
Gliomas carrying a BRAF or MEK mutation that recur or resist chemotherapy can often be controlled with oral targeted drugs, dabrafenib or trametinib, though these remain expensive for now.
How many distinct types of pediatric brain tumor are recognized?▼
The World Health Organization recognizes roughly 25 distinct types, though gliomas, medulloblastoma, ependymoma and embryonal or germ cell tumors of the brain account for most cases in practice.
In This Series: Childhood Cancer: The Journey from Despair to Durable Survival
- 1.Childhood Cancer
- 2.When to Suspect Cancer in a Child: Warning Signs by Organ System
- 3.Acute Lymphoblastic Leukemia: The 70-Year Climb from 20% to 90% Survival
- 4.Acute Myeloid and Chronic Myeloid Leukemia in Children
- 5.Brain Tumors in Children: Survival, DIPG and the Rise of Liquid Biopsy
- 6.Neuroblastoma and Wilms Tumor: Treating the Two Most Common Abdominal Cancers in Children
- 7.Osteosarcoma and Ewing Sarcoma: Choosing Limb Salvage Over Amputation
- 8.CAR T-Cell Therapy in Childhood Leukemia: How It Works and Where It Is Headed
- 9.Bone Marrow Transplant in Children: Relapsed Leukemia and Sickle Cell Disease
- 10.The Real Cost of Childhood Cancer Treatment: Targeted Therapy Prices Explained
- 11.How India and Africa Can Collaborate on Childhood Cancer Care