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New Study of MPNST Combination Therapy Based on Children’s Tumor Foundation Research

By April 2, 2014December 18th, 2023NF1, Science & Research

A malignant peripheral nerve sheath tumor (MPNST) is a rare cancer that can develop in a small subset of patients with NF1. If found and treated early, surgery can improve survival. However, patients with more widespread or inoperable MPNSTs have fewer options.

A growing trend in tackling MPNST and other soft-tissue tumors is using combination therapies. The idea is to give two drugs, each one working against cancer cells in a different way. These one-two punches hit the cancer again before it has time to recover from the first hit.

CTF-funded researcher Karen Cichowski identified a particular combination of two different types of cancer drugs that shrank MPNSTs in animals. Following the success of that work, Dr. Brigitte Widemann at The National Cancer Institute and Dr. AeRang Kim at Children’s National Health System in Washington, D.C. will test the same combination treatment against MPNSTs in patients.

A new clinical trial, A Trial of Ganetespib Plus Sirolimus: Phase 1 Includes Multiple Sarcoma Subtypes and Phase 2 MPNST, will be opening for enrollment soon.

The drugs used are Sirolimus, a type of drug called an mTOR inhibitor, and Ganetespib, a type of drug called an Hsp90 inhibitor.

Sirolimus is also known as rapamycin. This drug can shrink tumors initially, but eventually tumors can stop responding.

Ganetespib is being tested in human patients for other types of tumors and appears promising for safety and effectiveness. By inhibiting Hsp90, Ganetespib is thought to weaken cancer cells’ ability to survive.

You can find out more about this study by Googling its identifier, NCT02008877.