New drug combo shows promise for rare nerve tumors

NCT ID NCT03433183

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tested two drugs, selumetinib and sirolimus, in 21 people with advanced malignant peripheral nerve sheath tumors (MPNST), a rare cancer often linked to neurofibromatosis type 1. The goal was to see if the combination could shrink or stabilize tumors. The study measured how many patients had their disease controlled for at least four treatment cycles.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Selumetinib and sirolimus

What this could lead to

If successful, this combination could offer a new treatment option to slow or stop tumor growth in patients with advanced malignant peripheral nerve sheath tumors.

What could go wrong

This is a small, early-phase trial with only 21 participants. The results may not apply to all patients, and the combination may cause side effects without improving survival.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

malignant peripheral nerve sheath tumor neurofibromatosis type 1 neurofibrosarcoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Johns Hopkins University

    Baltimore, Maryland, 21287, United States

  • National Cancer Institute

    Bethesda, Maryland, 20892, United States

  • Washington University

    St Louis, Missouri, 63110, United States