Paediatric
Dr. Charles Mullighan (St. Jude Children’s Research Hospital) Acute lymphoblastic leukemia (ALL) remains a leading cause of childhood cancer death, and one type of this disease, Philadelphia-like ALL (Ph-like ALL), has an extremely poor outcome. A common feature of Ph-like ALL is the aberrant function of a particular gene named CRLF2. Dr Mullighan will develop two novel therapeutic approaches to counteract the CRLF2-related defects in cancer cells.

Myeloma
Dr Stephen Nutt (Walter & Eliza Hall Institute) Multiple myeloma (MM) is a cancer characterised by overgrowth of a white blood cell called plasma cells that form in the bone marrow. This aberrant growth depends on a regulatory protein called IRF4. Dr. Nutt will employ a novel approach to identify and test drugs that can inhibit IRF4 and block the growth of MM cells.

Myelodysplastic syndrome
Dr. Ashwin Unnikrishnan (University of New South Wales) The drug azacitidine is currently the best available treatment for myelodysplastic syndrome (MDS), yet the majority of patients do not respond to the drug, and others eventually suffer relapse. Dr. Unnikrishnan will investigate mechanisms by which azacitidine inhibits MDS cells and identify cellular pathways and features that are modulated by azacitidine treatment. This study will provide insight into why some patients do not respond and lay the groundwork for development of alternative therapeutic strategies.

Lymphoma
Dr. Ricky Johnstone (Univ. of Melbourne) Dr Johnstone aims to advance novel precision medicine therapies for diffuse large B-cell lymphoma (DLBCL), the most common form of non-Hodgkin lymphoma. He is testing therapies that inhibit a gene called TET2 that is often mutated in blood cancers, including DLBCL.TET2 plays a role in a mechanism called epigenetics, which switches genes on and off. When these epigenetic changes activate or inactivate genes improperly it allows cancer cells to proliferate.

Total Project cost: $656,516
Funding received to date: $437,678
Seeking: $218,838 for Year 3 funding
Leverage: $4 for every $1 donated to Snowdome Foundation