Medical Oncologist
Targeted therapy
Targeted Therapy for — Brain Tumours and Glioma
Targeted therapy can help a proportion of people with brain tumours, but only where a specific mutation has been found in the tumour tissue. The first step is knowing whether your tumour carries one.
Medically reviewed by Dr. C. Raghavendra Reddy, Medical Oncologist, MBBS (Gold Medal) · DNB · DM (Medical Oncology, Gold Medal) · Last reviewed August 2026
- Mutation testing decides eligibility — IDH, BRAF, and NTRK testing on your biopsy tissue determines whether targeted drugs apply to you.
- Not every grade responds equally — Lower-grade gliomas with IDH mutations have the most established targeted options. Glioblastoma currently has very limited targeted therapy.
- The blood-brain barrier matters — Not every drug approved for a mutation in another organ works in the brain. Drugs used here must cross the barrier in meaningful amounts.
- Given as oral tablets at home — Targeted therapy for brain tumours is usually taken as a daily tablet, with regular clinic visits for monitoring.
17+
Cancer Specialists
on Panel
on Panel
96.9%
Breast Cancer
Survival Rate*
Survival Rate*
15,000+
Patients
Treated
Treated
4.8★
Google Rating
(800+ reviews)
(800+ reviews)
Targeted therapy for brain tumours only works where a specific mutation has been found in your tumour tissue. The most commonly tested markers include IDH1, IDH2, and BRAF V600E. Your oncologist orders molecular testing on your biopsy to find out which, if any, apply to you.
What do these terms mean?
- IDH mutation
- A change in the IDH1 or IDH2 gene, found in a large proportion of lower-grade gliomas. It helps classify the tumour and determines whether IDH-inhibiting drugs may be suitable for you.
- BRAF V600E
- A specific mutation in the BRAF gene. Found in some adult gliomas and a higher proportion of pediatric brain tumours. Drugs that block this mutation are approved for use where it is present, regardless of tumour type.
- NTRK fusion
- A gene rearrangement involving the NTRK gene. Rare in brain tumours, but when it is present, drugs approved for any tumour type with this change are available.
- MGMT methylation
- A change in how the MGMT gene is switched on or off. It predicts response to a specific type of chemotherapy, not to targeted therapy. Your team may mention it alongside mutation results.
- Molecular profiling
- The laboratory process that analyses your biopsy tissue for mutations, gene fusions, and other markers. It usually takes one to two weeks and is done on the tissue already collected at surgery.
- Blood-brain barrier
- The protective filter between your bloodstream and your brain. It blocks many medicines, which is why not every drug approved for a mutation in another organ automatically works for a brain tumour.
Which mutations does your team test for in a brain tumour?
The standard first step is molecular profiling of your biopsy tissue. For gliomas, this typically covers IDH1 and IDH2 mutations, BRAF V600E, NTRK gene fusions, and MGMT promoter methylation. NCCN and ASCO both recommend this testing as part of routine workup for newly diagnosed glioma.
IDH mutations are found in a large proportion of lower-grade gliomas — particularly grade 2 and grade 3 tumours — and are much less common in glioblastoma. If your tumour carries one, your oncologist will assess whether an IDH inhibitor is appropriate for your grade and situation.
BRAF V600E is less common in adult gliomas but is found in a meaningful proportion of pediatric brain tumours and some adult low-grade gliomas. When it is present, approved drugs exist that work regardless of the organ the tumour is in.
NTRK fusions are rare in brain tumours but are important to test for, because tumour-agnostic approvals make effective drugs available even for uncommon cancer types.
Which targeted drugs are used for brain tumours?
For IDH-mutant grade 2 glioma, vorasidenib is an IDH inhibitor developed specifically for brain tumours. It received regulatory approval in some countries for this indication and is taken as a daily oral tablet. Your oncologist will confirm whether it is approved and accessible through Indian regulatory channels at this time.
For BRAF V600E-positive brain tumours, the combination of dabrafenib and trametinib is used. Blocking BRAF alone can lead to resistance appearing more quickly, which is why the two are given together. Both are oral tablets and carry tumour-agnostic approval in some settings.
For NTRK-fusion-positive tumours, larotrectinib or entrectinib are available through tumour-agnostic approvals — they are permitted for any cancer type carrying an NTRK fusion. Your oncologist will confirm what is currently accessible through CDSCO-approved channels.
Glioblastoma (grade 4) currently has very limited targeted therapy options. Most agents tested in GBM clinical trials have not improved outcomes in published studies. Standard treatment remains surgery, radiation, and temozolomide. Your oncologist will explain what evidence supports for your grade.
Meet the Specialists
17+ senior cancer specialists. One panel for your case.
Trained at AIIMS, Tata Memorial, and leading international centres. Combined 150+ years of experience. Every complex case is reviewed by 3+ of them — together.
Medical Oncologist
Dr. C. Raghavendra Reddy
MBBS(Gold Medal), DNB(General Medicine), DM(Medical Oncology)(Gold Medal)
Medical Oncologist
Dr. Bharati Devi Gorantla
MBBS, MD(General Medicine), DM(Medical Oncology)(Adyar,Chennai), ECMO, MRCP SCE(UK)
Medical Oncologist
Dr. Owais Mohammed
MBBS, MD (General Medicine), DrNB (Medical Oncology), ECMO, MRCP SCE (Medical Oncology) (UK)
Medical Oncologist
Medical Oncologist
Surgical Oncologist
Dr. Muralidhar Muddusetty
MBBS (AIIMS), MS (Surgery) (AIIMS), DNB (Surgical Oncology), MRCS (Edinburgh)
Surgical Oncologist
Surgical Oncologist
Surgical Oncologist
Dr. Vinay Mamidala
MBBS, MS(General Surgery), M.Ch(Surgical Oncology), FMAS, FARIS(Ongoing)
Surgical Oncologist
Radiation Oncologist
Radiation Oncologist
Radiation Oncologist
Hematologist
Interventional Radiologist
Dr. Mohammed Imran
Surgical Oncologist
Dr. Vajja Sandeep Kumar
MBBS, MS (General Surgery), DrNB (Surgical Oncology), FALS Oncology
Surgical Oncologist
Want a specific doctor for your case? Mention them when booking.
Book Free ConsultationBook an appointment with our specialist
Share your name and number — we'll call you back within 30 minutes to schedule your consultation.
Get a straight answer from a specialist
45 minutes, your reports reviewed, your questions answered in plain language.
Why is targeted therapy harder for brain tumours than for other cancers?
The blood-brain barrier is a protective filter between your bloodstream and your brain. It blocks many medicines that work well in other parts of the body from reaching the tumour in sufficient amounts.
This is why not every drug approved for a mutation elsewhere automatically works in the brain. Vorasidenib was developed specifically because earlier IDH inhibitors used in blood cancers do not cross the barrier effectively enough for brain tumour use.
Targeted therapy for brain tumours is given as oral tablets taken at home rather than by infusion in hospital. You come in at regular intervals for blood tests and monitoring scans. Your oncologist will set the specific schedule for your drug.
Did you know?
Molecular testing on glioma tissue now routinely identifies mutations that were not tested for a decade ago. NCCN and ASCO name IDH, BRAF, and NTRK testing as part of the standard workup for newly diagnosed glioma — not an optional extra.
If your team has not yet discussed molecular profiling results with you, that is a reasonable question to ask at your next appointment.
Source: NCCN Guidelines: Central Nervous System Cancers
What else do families want to know?
Can targeted therapy replace surgery for a brain tumour?
No. Surgery remains the first step for most brain tumours — it both confirms the diagnosis and provides the tissue that molecular testing is done on. Targeted therapy is given after surgery, usually alongside or after radiation and chemotherapy depending on grade and mutation status. No targeted drug currently replaces the need to know what the tumour is, and establishing that requires tissue from surgery or biopsy.
How will we know if the targeted drug is working?
The main tool is MRI scanning done at regular intervals your oncologist will set. Because targeted therapy can sometimes cause a temporary appearance of swelling on MRI before the tumour responds — a phenomenon called pseudoprogression — scans are interpreted alongside your clinical state and symptoms, not in isolation. Your team will explain what each scan result means in your specific case rather than reading the image alone.
What are the side effects of IDH inhibitors like vorasidenib?
IDH inhibitors tend to have a different side-effect profile from intensive chemotherapy. Fatigue, nausea, and sensitivity to sunlight are among the effects reported in clinical trials. Liver enzyme changes are monitored through regular blood tests. They do not typically cause the hair loss or severe infection risk associated with intensive chemotherapy regimens. Your oncologist will go through the specific side effects for the drug being considered for you before you start.
What if no actionable mutation is found — does that mean no treatment?
Being negative for IDH, BRAF, and NTRK mutations does not mean there is no treatment available. It means targeted drugs are not the right tool for your tumour. Standard treatment for glioma — surgery, radiation, and temozolomide chemotherapy — does not require a specific mutation and remains the backbone of care for most glioma patients. Your oncologist will explain the recommended pathway for your tumour type and grade.
Is targeted therapy covered by government insurance schemes in India?
Coverage varies by scheme, state, and specific drug. Some targeted therapies are included on approved drug lists under schemes such as Ayushman Bharat, while others are not listed or require prior approval. Your oncologist's team or a patient navigator at your treating centre can check what your specific scheme covers. Do not assume either way — the coverage position for newer agents like vorasidenib may not yet be established in India.
Can children with brain tumours have targeted therapy?
Yes, and BRAF V600E-targeted therapy is more commonly relevant in children than in adults, because the mutation occurs at higher rates in pediatric brain tumours, including certain low-grade gliomas and gangliogliomas. NCCN pediatric oncology guidance recommends BRAF testing as routine for children with brain tumours. The decision to use a targeted drug in a child is made with a pediatric oncologist and takes into account the specific tumour type, grade, and the child's overall clinical situation.
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
Next step
Still not sure what applies to you?
Send your reports across and a senior medical oncologist will go through what they mean, what is known, and what the options actually are.
Common questions
Frequently asked questions
What does IDH-mutant glioma mean for my treatment options?
An IDH mutation classifies your tumour as a specific subtype of glioma that behaves differently from IDH-wild-type tumours. It means your oncologist will consider whether an IDH inhibitor is appropriate alongside or after standard treatment. IDH-mutant gliomas also tend to grow more slowly than IDH-wild-type tumours of the same grade, which affects how treatment is sequenced. Ask your oncologist which mutation you have — IDH1 and IDH2 are distinct, and the drugs targeting them differ.
How long does molecular profiling take, and will it delay starting treatment?
Molecular testing typically takes one to two weeks from when the laboratory receives the sample. For most gliomas, this does not delay treatment because surgery — which provides the tissue — is usually followed by a recovery period before radiation and chemotherapy begin. If you are already waiting for results before the next treatment decision, ask your team when the sample was sent and when results are expected so you have a clear timeline.
Is vorasidenib available in India?
Vorasidenib received FDA approval in the United States for grade 2 IDH-mutant glioma, and regulatory approval processes in other countries, including India through CDSCO, take additional time. Whether it is accessible at the time of your appointment is something your oncologist will need to confirm, as the regulatory and access position can change. If it is not yet locally approved, your oncologist may discuss whether a clinical trial or compassionate access pathway applies to your situation.
Will targeted therapy work for glioblastoma?
Glioblastoma currently has very limited targeted therapy options. Most agents that showed promise in laboratory studies have not improved outcomes in clinical trials for GBM. Standard treatment remains surgery, radiation, and temozolomide, with bevacizumab used in some settings. NCCN and ASCO guidance does recommend molecular testing even in GBM — partly to identify the small proportion carrying actionable mutations, and partly for clinical trial eligibility. Ask your oncologist what testing has been done and what trials may be open.
How is targeted therapy given for brain tumours at CION?
The targeted drugs currently used for brain tumours — including vorasidenib, dabrafenib, trametinib, larotrectinib, and entrectinib — are all oral tablets taken at home, not infusions requiring a hospital visit for each dose. You come in at regular intervals for blood tests and monitoring. At CION, day-care appointments cover this monitoring across 35-plus centres. Your team will explain the schedule and what to watch for before you start.
What happens if the tumour comes back after targeted therapy?
Recurrence is assessed with a repeat scan, and in some cases a repeat biopsy to understand whether the tumour has changed its molecular profile. Tumours can develop resistance to targeted drugs over time, and the mutation present initially may still be there or the tumour may have found another route to grow. Recurrence does not necessarily mean there are no further options — it means the treatment plan needs to be reviewed. Your oncologist will discuss what the evidence supports at that point.