Funding for new study in to paediatric AML

Leukaemia & Lymphoma NI are delighted to announce that they have provided funding of around £140,000 for The Cured Project which is focused on Childhood Leukaemia.

The project will be led by research fellow Lydia Roets under the supervision of Dr Katrina Lappin and Dr Graeme Greenfield.

Dr Lappin explains the aims of the project:

“Acute myeloid leukaemia (AML) is an aggressive blood cancer that can be diagnosed in children. This cancer has a poor outcome, especially when patients do not respond to the chemotherapy treatment currently used (termed treatment failure). Following treatment failure, there are limited medicines available for these children. This highlights a need for a personalised medicine treatment approach in this patient group to reduce the risk of treatment failure.

Drugs that target a specific type of AML, known as MLLr-AML, are currently being tested in patients in clinical trials. This type of AML has a poor outcome and accounts for 20% of childhood AML. The results from the clinical trials have been promising for some patients however, some patients in the study quickly developed resistance to one of the drugs being tested. This demonstrates the quick ability of cancer to prevent a drug from working and enable continued growth of cancer cells, which is a major issue in the treatment of cancer.

In this study, we will identify and explore additional unique methods of drug resistance used by MLLr-AML cells, to the drugs in clinical trial. To do this we will knockout genes using technology called CRISPR/cas9, which is like molecular scissors that can cut genes out, and assess how the cancer cells respond to the clinical trial drugs with loss of certain genes. For this we will use a CRISPR/Cas9 screen that contains DNA probes to guide the scissors to specific genes. We need to ensure all the probes in the screen are presented at equal levels so we do not generate false data. To do this we will use sequencing technology performed by a company. Using the date generated we will also identify ways to re-sensitise the MLLr-AML cells to the clinical trial drugs, providing new additional treatment options for this patient group.”

We look forward to sharing updates on this exciting and vital new study.

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