Am80 (tamibarotene) and ATRA induce highly similar molecular responses and myeloid differentiation in non-APL AML, enhanced by LSD1/GCN5 inhibition and increased RARA expression - Scorecard - MDSpire

Am80 (tamibarotene) and ATRA induce highly similar molecular responses and myeloid differentiation in non-APL AML, enhanced by LSD1/GCN5 inhibition and increased RARA expression

  • By

  • Faezeh Ghazvini Zadegan

  • Clara Stanko

  • Franziska Fiedler

  • Laura Ölsner

  • Setenay Gupse Özcan

  • Jacqueline Schütt

  • Tina M. Schnöder

  • Yordan Sbirkov

  • Lukasz Szymanski

  • Sven Stengel

  • Peter Dittrich

  • Jörg P. Müller

  • Florian H. Heidel

  • Andreas Hochhaus

  • Sebastian Scholl

  • Ulf Schnetzke

  • Annamaria Brioli

  • Tino Schenk

  • June 27, 2026

  • 0 min

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Clinical Scorecard: Tamibarotene (Am80) and ATRA Trigger Comparable Molecular Changes and Myeloid Differentiation in Non-APL AML, Enhanced by Inhibition of LSD1/GCN5 and Elevated RARA Levels

At a Glance

CategoryDetail
ConditionAcute Myeloid Leukemia (AML)
Key MechanismsRegulated by retinoic acid receptor (RAR) transcription factors, particularly RARA and RARG, influencing myeloid differentiation.
Target PopulationPatients with non-APL AML, particularly those with high RARA expression.
Care SettingClinical trials and laboratory research.

Key Highlights

  • Am80 is more potent than ATRA in inducing differentiation in RARA-high AML.
  • Inhibition of LSD1 and GCN5 enhances ATRA and Am80 responses.
  • High RARA expression correlates with better treatment outcomes in AML patients.

Guideline-Based Recommendations

Diagnosis

  • Assess RARA expression levels in AML patients.

Management

  • Consider Am80 for patients with high RARA expression.

Monitoring & Follow-up

  • Monitor differentiation markers and response to treatment.

Risks

  • Evaluate potential resistance mechanisms involving LSD1 and GCN5.

Patient & Prescribing Data

Patients with non-APL AML, especially those with elevated RARA levels.

Combination of Am80 with azacytidine shows improved responses in clinical trials.

Clinical Best Practices

  • Utilize RARA-selective agonists like Am80 in RARA-overexpressing AML.
  • Incorporate epigenetic inhibitors to enhance retinoid therapy.

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