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New grants support blood cancer and liver disease research

Posted: 09th December 2021

SVI projects tackling inherited liver disease and the blood cancer multiple myeloma have been awarded grants from the St Vincent’s Hospital Melbourne Research Endowment Fund (REF).

The REF is an annual competitive grant round designed to support research activity of high quality on the St Vincent’s campus, and in particular, projects with clinical application or potential clinical significance and research collaborations.

Associate Professor Elaine Sanij, in collaboration with clinical haematologist Professor Hang Quach, will seek a new treatment combination to overcome drug resistance in the blood cancer multiple myeloma.

“The second most common blood cancer, multiple myeloma can be treated, but patients commonly relapse and the cancer becomes more and more resistant to treatment over time. While new drugs have emerged to treat this disease, treatment resistance remains an urgent challenge,” says Elaine.

“An estimated 2,400 Australians will have been diagnosed with multiple myeloma this year – more commonly men aged 60 or older. Around half of those diagnosed do not survive five years following diagnosis of this cancer.”

Dr Kiryu Yap, in collaboration with gastroenterologist Professor Alex Thompson, surgeon Mr Adrian Fox and metabolic physician Dr Heidi Peters, will investigate the genetic liver disease hereditary tyrosinemia type 1 (HT1), which affects about one in 100,000 people worldwide.

“If left untreated, children born with HT1 usually do not survive past age 10,” Kiryu explains. “The disease is caused by an inability to break down tyrosine – a building block of most proteins. Tyrosine builds up in the body and this can result in kidney and liver failure.”

Current treatments for HT1 include dietary protein restriction, costly drugs and liver transplant.

Kiryu’s project aims to develop new and more cost-effective treatments, by growing “mini-liver” organoids in the laboratory from cells donated by patients with HT1.

“Using just 10ml of a patient’s blood, we can grow stem cells in unlimited quantities and mature them into the cell types normally found in the liver,” says Kiryu. “We can use these organoids to better understand the drivers of disease in each patient, and in future to develop gene therapy approaches to treating this and potentially other genetic liver diseases.”

Collaborating on these projects are experts from St Vincent’s Hospital Melbourne, the Peter MacCallum Cancer Centre, The Royal Children’s Hospital and the Murdoch Children’s Research Institute.