Anti-rejection drug for bone marrow transplants to hit human trials by 2016

Armed with a new $6.8 million grant from the U.K.'s Wellcome Trust, Australian scientists are fine-tuning a drug designed to stop cancer patients from rejecting bone marrow transplants. Assuming they proceed on schedule, they hope to start human clinical trials by 2016, Sky News reports.

Specifically, the drug is designed to inhibit production of the protein perforin. In doing so, it would help blood cancer patients recover their immunity more quickly after bone marrow stem cell transplants, without the issue of rejection. Left untouched, perforin's activity makes rejection more likely. Researchers at the Peter MacCallum Cancer Center in Melbourne are pursuing the project, according to the story, after generating promising success in preclinical mouse studies. Monash University and Queensland Institute of Medical Research scientists are also working on the project, among others.

"In the mouse models we use, we know the inhibitors are effective," Joe Trapani, the center's executive director of cancer research, told the Australian Associated Press, as quoted by Sky News.

Of course, mouse model test results can show a drug works well, but that data isn't always repeated in subsequent human trials. The advance of such a drug is promising, however. If it works in people, the researchers said they see the drug as helping to boost the number of patients who can have bone marrow stem cell transplants. After all, a drug that prevents rejection could help boost donor matches, the story notes.

The research team's work builds on unraveling the molecular structure of perforin, the Sky News story notes, a protein that has drawn a decade of research attention as scientists have pursued how to unlock its function.

- here's the Sky News story