Old arthritis drug takes on tough cancers in early trial

NCT ID NCT04997993

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial is testing leflunomide, a drug currently used for rheumatoid arthritis, in people with advanced solid tumors that have a PTEN gene change or HER2-negative breast cancer. The study has two parts: first, finding a safe dose and checking for side effects; second, looking for any signs that the drug shrinks tumors. Only 23 participants will be enrolled, so results will be very preliminary.

What this could mean

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

Active substance

Leflunomide (a drug typically used for rheumatoid arthritis, taken as a daily pill)

What this could lead to

If it works, this could point toward a new treatment option for certain advanced cancers that have a PTEN gene change or HER2-negative breast cancer.

What could go wrong

This is a very early, small Phase 1 trial with only 23 participants. The main goal is safety and dosing, not yet proving the drug works against cancer. Side effects may be serious.

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.

Her2-receptor negative breast cancer

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

    Contact