Advanced pre-clinical evaluation of sphingosine kinase 1-targeting agents as a therapy for acute myeloid leukaemia
Background
Leukaemia is the second most frequently diagnosed cancer and is among the leading cause of cancer death.
Acute myeloid leukaemia (AML) in particular, is one of the most common forms of leukaemia in adults and outcomes are frequently poor, with an overall survival of only 1-2 years.
Conventional chemotherapy has remained the main AML treatment regimen for over 30 years, and despite being often highly effective at achieving initial disease remission, this is often short-lived resulting in patient relapse and death.
Indeed, in some AML subtypes, 5-year survival rates are as low as 10%, meaning new therapies are desperately needed.
About the Project:
Venetoclax is a promising AML therapy, but its response is short-lived and acquired resistance has become a hurdle in achieving long term disease remission.
Professor Pitson and his team will build on the concept that targeting the pro-survival protein, sphingosine kinase 1 (SK1) is a viable therapeutic approach for AML.
Specifically, the development of advanced, drug-like small molecules that can potently target SK1 has the potential for SK1-targeted clinical therapies.
With these clinically ready compounds already in place, this project has been designed to provide pre-clinical data to facilitate clinical trials for AML therapy.
Impact
If successful, this project will generate clinically translatable approaches to enhance AML therapy ready for progression to clinical trials to improve outcomes for AML patients.
In the short term, this project will provide the evidence needed to facilitate translation into clinical trials for AML therapy.
This project then aims to enter early clinical trials, with the purpose of providing clinical evidence of safety and higher doses, in combination with other therapies, like chemotherapeutics or Venetoclax-based therapies.