New targeted therapy shows promise for Tough-to-Treat leukemia

NCT ID NCT02204085

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a drug called GO-203-2C, which targets a protein (MUC1) found on cancer cells while sparing healthy cells. It was given alone or with another drug (decitabine) to 33 adults with acute myeloid leukemia that had returned or not responded to treatment. The main goals were to find the safest dose and check for early signs of effectiveness.

What this could mean

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

Active substance

GO-203-2C (a targeted drug that blocks MUC1 protein in cancer cells), given alone or with decitabine

What this could lead to

If successful, this could point toward a new treatment option for patients with hard-to-treat acute myeloid leukemia who have not responded to standard therapy.

What could go wrong

This is an early-phase trial with only 33 participants, focused on safety and dosing. The drug may not prove effective enough, and side effects or lack of response are possible.

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.

acute myeloid leukemia

As listed by the trial registrant

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

Contacts and locations

Locations

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02115, United States