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Role of Autophagy in Differentiation Therapy in AML

Posted on: 15 Jun 2018

Role of Autophagy in Differentiation Therapy in AML



Principal Investigator: Prof. Mary Cahill and Dr. Sharon McKenna

Lead Researcher: Dr. Dalyia Benjamin

Grant Type: Joint Funding Grant with HERO Foundation


Dr Benjamin has identified a novel treatment regime, which can lead to the induction of both autophagy and differentiation in cells derived from Acute Myeloid Leukaemia (AML). AML is the most common acute leukaemia in adults overall.

The 5-year survival rate of patients under 60 years old is 47% and this drops to 20% for those over 60 years. These figures highlight the urgent need for improved and tolerable therapeutic strategies. In the last two years we have been collecting primary AML cells so that this novel treatment can be tested on patient cells. This would be a better indication of the likelihood of treatment success in patients. It is hoped that this project will lay the foundations for a new clinical trial of differentiation therapy in AML.

Pharmacologic override of the cellular differentiation block in AML, is one attractive strategy with a favourable toxicity profile. Success in ‘differentiation therapy’ has been achieved with the use of all-trans-retinoic acid (ATRA) for the treatment of acute promyelocytic leukaemia (APL). This proposed research project is intended to advance our understanding of differentiation and discover new avenues for therapeutic intervention in AML.


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