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
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
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Dana-Farber Cancer Institute
Boston, Massachusetts, 02115, United States