Monash rare disease drug trial gets $1.5 million boost

A/Prof David Curtis, left, in clinic.
Still from Monash University at AMREP video

by Anne Crawford

Monash University researchers will start a national clinical trial into a new treatment for a life-threatening complication that follows bone marrow transplant, thanks to a $1.5
million federal government grant.

Associate Professor David Curtis was among four medical researchers from Monash University and The Alfred Hospital awarded more than $5.5million under the Medical Research Future Fund’s (MRFF) Rare Cancers, Rare Diseases and Unmet Needs Clinical Trials Program.
A colleague in the Australian Centre for Blood Diseases (ACBD), Adjunct Associate Professor Andrew Wei, also received funding (see Andrew Wei story and summary story of MRFF AMREP recipients). 

Bone marrow transplant can be life-saving for people with leukaemia but 40 per cent of these patients develop a major complication called graft versus host disease (GVHD). GVHD is a rare, debilitating disease causing symptoms including damage to organs and skin conditions that can lead to disfigurement. Often those with it are unable to work.

“It’s very hard to get funded to do this sort of work into rare diseases – this new medical research grant is really going to make a difference,” Associate Professor Curtis said. “It’s very exciting.”

The funds will allow Associate Professor Curtis to begin a randomised clinical trial comparing the new drug with the standard strategy used to prevent GVHD. “These treatments trying to prevent it happening haven’t changed in 30 years,” he said.

The drug, cyclophosphamide, is inexpensive and has already been used in a different transplantation setting.

“It seems to work very well in that setting,” Associate Professor Curtis said. “We’ve been testing it for the last two years at The Alfred and seeing very good preliminary results with no obvious problems,” he said. “But the only way to see if it is better than the existing treatment is a randomised trial.”

Associate Professor Curtis said the researchers were the first in the world to conduct such a trial. Ten transplant centres throughout Australia and New Zealand have agreed to participate in it.

“If we can reduce the risk of getting this horrible disease it would quickly change the way we do transplants in Australia and probably internationally because it’s cheap and easy,” he said.