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New Combination Treatment Effective Against Chemo Resistance Acute Leukaemias
Phase1 study produces 83% remission from combination approach.

Much respected US based cancer research pioneer, Professor Joseph Scandura, Assistant Professor of Medicine at Weill Cornell Medical College in New York, is in Cork today to share the results of his clinical study to improve treatment of acute leukaemias and bone marrow cancers. Physician-scientist Prof. Scandura is presenting the results of his Phase 1 study, which showed that adding the drug Decitabine (an epigenetic modifier) in conjunction with standard chemotherapy achieved remission in the vast majority of patients (83%).
Currently, the most effective treatments for acute myelogenous leukemia (AML) involve a combination of cytotoxic chemotherapeutics but some subtypes of AML are resistant to this standard approach. One way that the leukemia cells escape is by turning off the genes that make them sensitive to chemotherapy. They essentially repackage their genes such that they cannot be turned on. This repackaging doesn’t change the genetic code like a mutation but simply makes the gene inaccessible. This is called epigenetic regulation. This is an exciting new development in our understanding of gene regulation as it is potentially more easily reversible than a mutation. Prof Scandura’s study has shown the potential of drugs which target this change; epigenetic modifiers.
Prof Scandura’s group are now conducting a much larger multicenter trial to compare this treatment to standard chemotherapy. In addition, they are assessing early markers or ‘signatures’ of response in patient leukaemia cells. This will help to optimize and perhaps individualize the dose and schedule used for decitabine so that patients are given just enough to achieve remission but not more. It is hoped that this personalized approach will increase the effectiveness of drugs like decitabine while at the same time minimizing damage to normal cells and reducing the side effects associated with chemotherapy.
Prof. Joseph Scandura, is sharing the recent developments in his research at both University College Cork and Cork University Hospital this week. This is timed to coincide with the imminent transfer of a Cork-Cornell Graduate student Dr Nina Orfali. Dr Orfali will shortly complete the first year of her Ph.D. in the Cork Cancer Research Centre and then travel to New York for one year’s research training in the Dept Pharmacology in Weill Cornell Medical College. This new program was set up by the Cork Cancer Research Centre to encourage the training of clinicians in cancer research and incorporate best practice from both sides of the Atlantic and is funded in part by Breakthrough Cancer Research.
Commenting on Dr. Orfali’s current research programme, Dr Scandura said, “We are looking forward to welcoming Dr Nina Orfali to Weill Cornell Medical College, where we hope this collaboration from both sides of the Atlantic will lead to new breakthroughs in cancer research. We are very excited about her research into drug resistance in leukemia. Caring for patients and providing support for young scientist-physicians like Dr. Orfali are so important for research institutions like ourselves and we look forward to teaching and learning from each other.”
"We are delighted to have Prof. Scandura here at Cork University Hospital and CCRC. His work on new treatments for acute Leukemia is a tremendous example of how the integration of scientific research can deliver significant improvements in patient care. The model of the physician scientist is something we very much want to promote here in Cork and throughout Ireland" said Dr. Mary Cahill (Dept. of Haematology, Cork University Hospital) who coordinated his visit to Cork and jointly supervises the haematology research carried out by Dr Nina Orfali between CCRC and Weill Cornell Medical College
Also commenting, Dr. Sharon McKenna of Cork Cancer Research Centre said, “We are delighted to invite researchers from Weill Cornell Medical College to Cork and to partner with such an innovative and prestigious institute. The opportunity to learn from each other will help to accelerate cancer research projects on both sides of the Atlantic. We would also like to wish Dr Nina Orfali the very best in her research programme.”


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