Adelaide researchers have discovered a possible new method to treat myelofibrosis (MF), that could have the potential to greatly extend lifespan without the side effects caused by current treatments.

While MF symptoms can often be controlled with the drug Ruxolitinib, side effects may be problematic and currently, the only cure for MF is an allogeneic stem cell transplant.

Dr Daniel Thomas, leader of the Myeloid Metabolism Lab at the South Australian Health and Medical Research Institute, (SAHMRI) and Associate Professor of Medicine at the University of Adelaide, led the study in collaboration with Professor Angel Lopez at SA Pathology.

Dr Thomas says it was a stroke of luck that led to the extraordinary find.
“We were actually trying to make a tool to study myelofibrosis. We didn’t realise the antibody we made would have therapeutic properties,” Dr Thomas said.
“Our drug blocked the growth of cancer cells in a very aggressive live model of the disease, significantly increasing survival rate without noticeable negative side effects,” Dr Thomas said.

Co-lead author and biochemist Dr Denis Tvorogov generated antibodies using a peptide fragment called ‘neoepitope’ that’s only present within the cancer and not on any normal tissues. What he didn’t expect was for the antibody to kill cancer cells when he tested it on patient samples, working with an early career scientist Dr Chloe Thompson-Peach.
“What is really exciting is that many other cancers have similar peptide fragments that we could also target by harnessing the immune system,” Dr Tvorogov said.
“These fragments are created by the insertion or deletion mutations within the cancer. We’ve found they not only drive cancer growth but also vulnerable for targeting without side effects.”

The new antibody is currently being prepared for early phase clinical trials set to run in South Australia later this year, supported by local biotech company, AusHealth.  Pre-clinical models have shown the drug is effective at shrinking tumours and Dr Thomas is confident the antibodies will prove to be safe and effective in humans.
“We estimate there are at least 12,000 Australians living with cancer or having had cancer that express a recurrent neoepitope similar to what is found in myelofibrosis, that could be curable with
an immunotherapeutic approach,” Dr Thomas said.

“This discovery brings us a fresh perspective. We need to build cell therapies and antibody therapies against these fragments as fast as possible.”

The full media release is available:  SAHMRI media release – Lucky find could hold key to beating rare blood cancer —