Medical Oncologist
Targeted therapy in children
Targeted Therapy for — Childhood Cancers
Hearing that your child may need targeted therapy is often overwhelming. Targeted therapy means matching a drug to a specific fault in the cancer's DNA — and whether your child is eligible depends on what molecular testing finds in the tumour.
Medically reviewed by Dr. C. Raghavendra Reddy, Medical Oncologist, MBBS (Gold Medal) · DNB · DM (Medical Oncology, Gold Medal) · Last reviewed August 2026
- Testing decides eligibility — Molecular profiling of the tumour tells you which children have a targetable gene change and which do not.
- Not all childhood cancers have a target — A targeted drug only works when a specific mutation is present. A negative result is not a bad sign — it means a different treatment fits the tumour better.
- Testing usually uses existing tissue — The biopsy your child has already had is the starting point. A new procedure is rarely needed just for molecular testing.
- Treatment is usually given as day care — Most targeted therapies for children are taken as a daily tablet at home or given as a short infusion without an overnight hospital stay.
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Targeted therapy for childhood cancer matches a specific drug to a specific fault in the cancer's DNA. The tumour is tested for mutations — changes in genes such as NTRK, ALK, BRAF or BCR-ABL1 — and if one is found, a targeted drug may be recommended alongside or instead of standard chemotherapy.
How does targeted therapy testing work for a child with cancer?
Biopsy or bone marrow sample is collected
At or shortly after diagnosis, tissue from the tumour — or a bone marrow sample in blood cancers — is taken and sent to a pathology laboratory. This is usually the same sample collected for your child's initial diagnosis.
The sample is sent for molecular testing
A specialist laboratory examines the cancer's DNA for specific gene changes, including fusions and mutations that may have a matching targeted drug.
Your oncologist reviews the results
The molecular report is considered alongside your child's cancer type, stage, and overall fitness. Not every mutation found will have a matched drug available, and your oncologist will explain what each result means.
A treatment plan is built around the findings
If a targetable mutation is found and a drug is supported by guidance from the Children's Oncology Group, NCCN or ASCO, it may be added to — or in some subtypes replace — standard chemotherapy.
Treatment is given as a tablet or day-care infusion
Most targeted therapies for children are taken as a daily tablet or given as a short infusion. An overnight hospital stay is rarely needed for the treatment itself.
What do the mutation names in my child's report mean?
- NTRK fusion
- A change where two genes join abnormally, producing a protein that drives cancer growth. NTRK fusions are found across many different childhood tumour types. Drugs called TRK inhibitors are approved for use in children when this fusion is present, and this is one of the most established targets in paediatric oncology.
- ALK fusion
- A gene rearrangement seen in some childhood lymphomas and certain solid tumours. ALK inhibitors have established evidence in ALK-positive disease and appear in COG and NCCN guidance for relevant tumour types.
- BRAF V600E
- A specific point mutation found in some childhood brain tumours and papillary thyroid cancers. BRAF-targeting drugs are an active area in paediatric neuro-oncology, and this mutation is now routinely tested in those tumour types.
- BCR-ABL1 (Philadelphia chromosome)
- A gene fusion that produces an abnormally active signalling protein, found in Philadelphia chromosome-positive acute leukaemia. Adding a tyrosine kinase inhibitor to chemotherapy is standard practice for this subtype according to COG and ASCO guidance.
- FLT3 mutation
- A mutation found in a proportion of children with acute myeloid leukaemia. FLT3 inhibitors are being studied alongside chemotherapy in this group, with guidance evolving through international cooperative trials.
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Dr. Bharati Devi Gorantla
MBBS, MD(General Medicine), DM(Medical Oncology)(Adyar,Chennai), ECMO, MRCP SCE(UK)
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Dr. Owais Mohammed
MBBS, MD (General Medicine), DrNB (Medical Oncology), ECMO, MRCP SCE (Medical Oncology) (UK)
Medical Oncologist
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Dr. Muralidhar Muddusetty
MBBS (AIIMS), MS (Surgery) (AIIMS), DNB (Surgical Oncology), MRCS (Edinburgh)
Surgical Oncologist
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Dr. Vinay Mamidala
MBBS, MS(General Surgery), M.Ch(Surgical Oncology), FMAS, FARIS(Ongoing)
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Dr. Mohammed Imran
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Dr. Vajja Sandeep Kumar
MBBS, MS (General Surgery), DrNB (Surgical Oncology), FALS Oncology
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What happens when a matching mutation is found?
Finding a targetable mutation does not change the diagnosis — it adds information that may change the treatment. Your child's oncologist will explain what the mutation means for their specific cancer and whether a matched drug is recommended.
In some subtypes, such as Philadelphia chromosome-positive leukaemia, adding a targeted drug is standard practice. In others, the evidence is newer and the drug may be offered as part of a clinical trial.
Being told no targetable mutation was found does not mean options have run out. It means targeted therapy is not the right route for this tumour, and the plan will use other evidence-based treatments.
What should you expect during your child's targeted therapy?
Many targeted drugs for childhood cancers are taken as daily tablets at home. Others are given as a short infusion in day care, without an overnight stay.
Side effects differ from chemotherapy. They can include fatigue, skin changes, liver enzyme changes, and with some drugs effects on bone growth. Your child's team will monitor closely throughout treatment and adjust the plan if a side effect becomes significant.
Response is reassessed at regular intervals with imaging and blood tests. The treatment plan may be refined based on how the cancer is responding.
Explore 119 more Targeted Therapy Basics, Testing & Your Cancer Type topics
Cancer-Type Specific Targeted Therapy
- CML Treatment with TKIs: Living a Normal Life Span
- Cancer of Unknown Primary: Can Molecular Testing Help?
- GIST Treatment: Why This Cancer Changed Targeted Therapy Forever
- Targeted Therapy for Bile Duct Cancer (Cholangiocarcinoma)
- Targeted Therapy for Bladder and Urothelial Cancer
- Targeted Therapy for Brain Tumours and Glioma
- Targeted Therapy for Breast Cancer: HER2, HR+ and Triple Negative
- Targeted Therapy for CLL and Lymphoma
- Targeted Therapy for Cervical and Endometrial Cancer
- Targeted Therapy for Childhood Cancers
- Targeted Therapy for Colorectal Cancer: RAS, BRAF and HER2
- Targeted Therapy for Head and Neck Cancer
- Targeted Therapy for Kidney Cancer (RCC)
- Targeted Therapy for Liver Cancer (HCC)
- Targeted Therapy for Lung Cancer: Every Mutation and Drug Explained
- Targeted Therapy for Melanoma: BRAF and MEK Inhibitors
- Targeted Therapy for Multiple Myeloma
- Targeted Therapy for Neuroendocrine Tumours
- Targeted Therapy for Ovarian Cancer: PARP Inhibitors and Beyond
- Targeted Therapy for Pancreatic Cancer: Limited but Real Options
- Targeted Therapy for Prostate Cancer
- Targeted Therapy for Sarcoma
- Targeted Therapy for Stomach and Gastroesophageal Cancer
- Targeted Therapy for Thyroid Cancer
- Targeted Therapy in AML: Beyond Standard Chemotherapy
- Tumour-Agnostic Drugs: When the Mutation Matters More Than the Organ
Before You Start - Preparation & Baseline
- 25 Questions to Ask Your Oncologist Before Starting Targeted Therapy
- Before You Start Targeted Therapy: The Complete Preparation Checklist
- Dental Check-Up Before Cancer Treatment: Why It Matters
- Financial Planning Before Starting Long-Term Targeted Therapy
- How Long After Surgery Can You Start Targeted Therapy?
- How to Store Your Targeted Therapy Tablets Correctly
- Setting Up a Dosing Routine You Won't Forget
- Should Targeted Therapy Be Taken With Food or on an Empty Stomach?
- Vaccinations Before and During Cancer Treatment
- What to Expect in Your First Month on Targeted Therapy
- Which Baseline Tests Are Done Before Starting a TKI?
- Which of Your Existing Medicines Must Stop Before Targeted Therapy
- Why You Need an ECHO and ECG Before Certain Cancer Drugs
Biomarker & Molecular Testing
- Can Mutation Testing Be Done on an Old Biopsy Block or Slides?
- Cost of NGS and Mutation Testing in India: 2026 Price Guide
- Do I Need Genetic Testing Before Starting Targeted Therapy?
- Germline vs Somatic Testing: The Difference Nobody Explains Properly
- Getting a Second Opinion on Your Genomic Report
- How Long Do Mutation Test Results Take in India? Realistic Timelines
- How to Read Your Molecular Pathology Report, Line by Line
- IHC, FISH, PCR or NGS: Which Mutation Test Is Right for Your Cancer?
- Is Mutation Testing Worth It If I Can't Afford the Drug?
- Liquid Biopsy vs Tissue Biopsy: Which Test Do You Need?
- My Report Says a Mutation Is 'Actionable': What Does That Mean?
- No Mutation Found in My Report: What Are My Options Now?
- Should I Start Chemotherapy While Waiting for Mutation Results?
- Should You Repeat Molecular Testing After Your Cancer Progresses?
- Single-Gene Test or Full Panel? How to Choose Without Wasting Money
- What Are PD-L1, TMB and MSI, and Do They Affect Targeted Therapy?
- What Does 'Variant of Uncertain Significance' (VUS) Mean?
- What Happens If There Isn't Enough Tissue for Mutation Testing?
- What Is NGS (Next-Generation Sequencing) Testing in Cancer?
- What Is a Molecular Tumour Board and Why Should You Want One?
- Which Cancers Should Always Be Tested for Mutations? A Checklist
- ctDNA and MRD Testing: Tracking Cancer Through a Blood Test
Foundations & How Targeted Therapy Works
- Adjuvant and Neoadjuvant Targeted Therapy: Treatment Before or After Surgery
- Antibody-Drug Conjugates (ADCs): The Smart Bombs of Cancer Treatment
- Biosimilars and Generic Targeted Therapy Drugs: What They Are
- Can Targeted Therapy Be Combined with Chemotherapy or Radiation?
- Can Targeted Therapy Cure Cancer? An Honest Answer
- First-Line, Second-Line and Beyond: How Treatment Lines Work
- How Doctors Decide Between Chemo, Immunotherapy and Targeted Therapy
- How Does Targeted Therapy Actually Work? Explained Without Jargon
- How Long Does Targeted Therapy Take to Start Working?
- Is Targeted Therapy Painful? What the Treatment Actually Feels Like
- Is Targeted Therapy a Type of Chemotherapy? Clearing Up the Confusion
- Oral Tablets vs IV Infusion Targeted Therapy: What's the Difference?
- Precision Oncology vs Personalised Medicine: Are They the Same Thing?
- Targeted Therapy Success Rates: What the Numbers Really Mean
- Targeted Therapy for Stage 4 Cancer: What You Can Realistically Expect
- Targeted Therapy vs Chemotherapy: 12 Differences That Actually Matter
- Targeted Therapy vs Immunotherapy: Which One Is Right for You?
- The 8 Main Types of Targeted Therapy Drugs (With Examples)
- What Do Cancer Drug Endings Like -nib, -mab and -tinib Mean?
- What Is Targeted Therapy for Cancer? A Complete Patient Guide
- Which Cancers Can Be Treated with Targeted Therapy? Full List
- Why Didn't My Doctor Recommend Targeted Therapy for Me?
Mutation & Target-Specific Pages
- ALK-Positive Lung Cancer: Drugs, Response and What Comes Next
- Antiangiogenic Drugs (VEGF Inhibitors): How Starving a Tumour Works
- BCR-ABL and the Philadelphia Chromosome in CML
- BRAF V600E Mutation: Targeted Therapy Across Multiple Cancers
- BRCA vs HRD Testing: Which One Determines PARP Inhibitor Eligibility?
- BRCA1 and BRCA2 Mutations in Cancer Treatment: PARP Inhibitors
- BTK Inhibitors in CLL and Lymphoma
- CD20, CD38 and Other Antibody Targets in Blood Cancers
- CDK4/6 Inhibitors in Hormone-Positive Breast Cancer
- Co-Mutations: When You Have Two Mutations at Once
- EGFR Exon 20 Insertion: Why It's Different and Harder to Treat
- EGFR Mutation in Lung Cancer: Complete Patient Guide
- ESR1 Mutation: Why Your Hormone Therapy Stopped Working
- FGFR Alterations in Bladder and Bile Duct Cancer
- FLT3 Mutation in AML: What It Means for Your Prognosis
- HER2 Mutation in Lung and Gastric Cancer
- HER2-Low Breast Cancer: A New Category That Changes Treatment
- HER2-Positive Breast Cancer: The Full Targeted Therapy Roadmap
- IDH1 and IDH2 Mutations in Glioma, AML and Bile Duct Cancer
- KIT and PDGFRA Mutations in GIST
- KRAS G12C Mutation: The Target That Was Undruggable for 40 Years
- KRAS Mutation but Not G12C: What Are My Options?
- MET Exon 14 Skipping and MET Amplification Explained
- MSI-High and Mismatch Repair Deficiency: Testing and Treatment
- NRG1, MET, RET and Other Rare Fusions: Where to Get Tested
- NTRK Fusion: One Drug for Many Different Cancers
- Osimertinib: What to Expect, Side Effects and How Long It Works
- PALB2, ATM and Other BRCA-Like Genes: Do They Get the Same Drugs?
- PIK3CA Mutation in Breast Cancer: Alpelisib and What to Expect
- PSMA and AR-Targeted Therapy in Prostate Cancer
- RET Fusion Cancer: Lung, Thyroid and Beyond
- ROS1-Positive Cancer: A Rare Mutation with Excellent Options
- TP53 Mutation: Why There's Still No Targeted Drug for It
- Understanding Variant Allele Frequency (VAF) in Your Report
- What If My Mutation Has No Approved Drug Yet?
- mTOR Inhibitors in Cancer: Everolimus and Temsirolimus
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Common questions
Frequently asked questions
Does my child need a new biopsy for molecular testing?
Usually not. Molecular testing is done on tissue already collected at diagnosis — the same biopsy or bone marrow sample your child has already had. A new procedure is only needed if the original sample was too small, or if a repeat assessment is requested at a later stage. Your team will tell you clearly if more tissue is needed and why.
How long does mutation testing take?
Comprehensive molecular profiling — which looks across a wide range of gene changes — typically takes one to two weeks from when the sample reaches the laboratory. Standard pathology results may be faster. If results are needed urgently, some tests can be prioritised. Ask your team what has been ordered and when the result is expected so you are not waiting without a timeline.
What if no targetable mutation is found?
A negative result means chemotherapy, radiation, surgery, or a combination of these remains the recommended approach for your child's cancer type and stage. It is not a sign that the cancer is more serious or that options have run out — it is information about which treatment route fits the biology of the tumour. Ask your oncologist to explain the alternative plan and what it is intended to achieve.
Is targeted therapy easier to tolerate than chemotherapy?
Not always, and the comparison differs by drug. Targeted therapies have their own side effects — some children experience fatigue, skin changes, liver enzyme changes, or effects on growth. These are different from chemotherapy side effects rather than necessarily milder. Your child's team monitors for both throughout treatment and adjusts the plan if something significant arises.
Are targeted therapy drugs for children available in India?
Some are approved by CDSCO and available through oncology centres. Others may be accessible through clinical trials, expanded access programmes, or compassionate use. Availability changes as approvals are updated, so your oncologist is the right person to ask about any specific drug. Do not assume a drug is unavailable without checking directly with your treating team.
Will my child need to stay in hospital for targeted therapy?
Most targeted therapies for children are given either as daily tablets taken at home or as short infusions in day care — a session that does not require an overnight stay. There are exceptions: certain treatments, or certain side effects, may need admission. Your team will explain the schedule before treatment begins so you can plan around it.