Old arthritis drug takes on tough cancers in early trial

NCT ID NCT04997993

Recruiting now Disease control Sponsor: Deborah Doroshow Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

PTEN-ALTERED ADVANCED SOLID TUMORS AND HER2 NEGATIVE BREAST CANCER

Contacts and locations

Locations

Icahn School of Medicine at Mount Sinai
RECRUITING

New York, New York, 10029, United States

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