Newly Diagnosed

Treating Brain Tumors With Targeted Therapies

Sometimes your child’s treatment team will choose a type of medication which targets specific molecules (usually proteins, genes or pathways) involved in the growth, spread, or survival of cancer cells. Other medications are designed to affect cancer cells with specific mutations or markers. These medications target tiny changes in the cells of your child’s tumor that make them different from the other cells you want the therapy to avoid.  That differentiates them from other cells you want the therapy to avoid

These therapies are sometimes called “targeted therapies,” “molecularly targeted drugs,” “molecularly targeted therapies,” or “precision medicine.”

Doctors use these medicines to block the growth, progression, and spread of cancer by disrupting specific tumor molecules.  They also cause less damage to healthy cells than other kinds of chemotherapy.

Molecular Testing and Clinical Trials

When your child is diagnosed with a brain tumor, the doctor may recommend molecular testing to look at the DNA and genetic material inside the tumor’s cells. This can make your child’s diagnosis more precise and help the doctor choose a targeted treatment for them. Molecular testing can also help doctors find out if your child is a match for a clinical trial of a new targeted treatment.

When doctors find a new drug or therapy they think may help treat a type of childhood brain tumor, they organize a clinical trial to learn what happens when a group of patients receives the treatment.

Examples include:

Monoclonal Antibodies (mABS) – These are laboratory made antibodies that bind to specific proteins on the surface of cancer cells. They can:

• Mark cancer cells for immune destruction

• Block growth signals

• Deliver chemotherapy or radioactive particles directly to tumor cells.

Molecular Pathway Inhibitors alter molecular pathways that control cell growth, especially when genes are mutated or overactive. Examples include:

• BRAF Inhibitors:  Target the RAS–RAF–MEK–ERK s pathway Used when tumors have a BRAF V600E mutation, commonly seen in some low-grade gliomas. BRAF protein sends signals inside the cell that signal for cell division and growth
  • Drugs: Dabrafenib, Vemurafenib
  • Often combined with MEK inhibitors like Trametinib for better effect.
• MEK Inhibitors: Target the MAPK/ERK , which is part of the RAS-RAF-MEK-ERK pathway,pathway, often altered in gliomas. The MEK protein is important for cell growth and inhibiting it can cause cell death.
  • Drugs: Trametinib, Selumetinib
  • Selumetinib is under investigation or used in *neurofibromatosis type 1 (NF1)-associated tumors.
• mTOR Inhibitors: Target the PI3K/AKT/mTOR pathway, which is involved in cell growth and survival.
  • Drugs: Everolimus, Sirolimus
  • Used in subependymal giant cell astrocytoma (SEGA) in patients with tuberous sclerosis complex (TSC).

Choosing to take part in a clinical trial is an important decision. The Pediatric Brain Tumor Foundation works closely with other organizations to support families wanting to learn more. Fill out this short form to talk with our team about clinical trials for children with brain tumors.