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Drug Target Review·3 min read·hard

NTX-301 targets treatment-resistant AML via Hippo pathway in preclinical study

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Drug Target Review
NTX-301 targets treatment-resistant AML via Hippo pathway in preclinical study
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Researchers at MD Anderson Cancer Center have identified that the experimental drug NTX-301 shows promise in treating therapy-resistant acute myeloid leukaemia (AML). The drug works by activating the Hippo pathway, which helps suppress tumor growth and overcome drug resistance.

Why it matters

This discovery offers a potential new therapeutic pathway for patients with high-risk, relapsed AML who have exhausted standard treatment options.

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An investigational epigenetic therapy has shown encouraging results against treatment-resistant acute myeloid leukaemia (AML) in preclinical studies from The University of Texas MD Anderson Cancer Center .

The research discovered that an experimental drug, called NTX-301, remained effective in therapy-resistant AML by activating the Hippo pathway, a tumour-suppressing signalling pathway involved in regulating cell growth and drug resistance.

The findings suggest the treatment could provide a new approach for patients with relapsed AML, including those with TP53 mutations, one of the highest-risk forms of the disease.

AML is an aggressive blood cancer in which many patients initially respond to frontline treatment combining hypomethylating agents with venetoclax. However, resistance frequently develops, allowing the disease to return.

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