Targeted therapy for brain and spinal cord tumours

Targeted therapy uses drugs to target specific molecules (such as proteins) on cancer cells or inside them. These molecules help send signals that tell cells to grow or divide. By targeting these molecules, the drugs stop the growth and spread of cancer cells and limit harm to normal cells.

Targeted therapy may also be called molecular targeted therapy.

Some people with brain and spinal cord tumours have targeted therapy. If you have targeted therapy, your healthcare team will use what they know about the cancer and about your health to plan the drugs, doses and schedules.

You may have targeted therapy:

• to destroy cancer cells left behind after surgery and reduce the risk that the cancer will come back (recur) (called adjuvant therapy)
• after trying other treatments that haven't worked
• to treat some brain and spinal cord tumours that have recurred
• in combination with chemotherapy

The following targeted therapy drugs are used for brain and spinal cord tumours.

Vorasidenib (Voranigo) targets mutations in the isocitrate dehydrogenase (IDH) genes, IDH1 and IDH2. It is used for oligodendroglioma tumours and astrocytoma grade 2 tumours that have the IDH mutations.

Larotrectinib (Vitrakvi) targets the neurotrophic tyrosine receptor kinase (NTRK) gene fusion. It may be used for brain and spinal cord tumours that have this gene fusion and cannot be removed with surgery or for tumours that have no other treatment options.

Both vorasidenib and larotrectinib are given as a pill.

Side effects of targeted therapy will depend mainly on the type of drug or combination of drugs, the dose, how it’s given and your overall health. Tell your healthcare team if you have side effects that you think are from targeted therapy. The sooner you tell them of any problems, the sooner they can suggest ways to help you deal with them.

Targeted therapy drugs may cause these side effects:

• fatigue
• headache
• sore mouth
• loss of appetite
• nausea and vomiting
• diarrhea
• constipation
• low white blood cell count
• liver problems
• increased blood pressure
• skin problems, such as a rash

All drugs, including those used to treat cancer, are approved for use in Canada by Health Canada. If a drug is mentioned on cancer.ca, it means that Health Canada has approved the drug and pharmaceutical companies sell it in Canada. It does not mean that the drug is offered everywhere in Canada.

Paying for drugs is the responsibility of provincial and territorial health departments. Each health department makes its own decisions about which drugs to cover (pay for) in that province or territory. This means that a drug may be covered in one province, but not in others.

Your oncologist will talk to you about different drug treatments and what is available through your provincial or territorial drug program.

Find out more about targeted therapy. To make the decisions that are right for you, ask your healthcare team questions about targeted therapy.

Details on specific drugs change quite regularly. Find out more about sources of drug information and where to get details on specific drugs.