Old drug may be key to bone-marrow transplant treatment
MELBOURNE researchers have found a promising way to prevent serious and lifelong complications from bone-marrow transplants.
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MELBOURNE researchers have found a promising way to prevent serious and lifelong complications from bone-marrow transplants.
These affect almost half of blood cancer patients who undergo the lifesaving procedure.
A team at The Alfred hospital has successfully used a 30-year-old drug as a simple and cheap treatment in about 20 patients over the past two years.
It is now preparing to fully test its effectiveness in a $1.5 million clinical trial across Australia and New Zealand.
Bone marrow, or stem cell, transplants are last-ditch treatments for leukaemia patients who need urgent replacement of immune-fighting cells when other treatments have failed to control the cancer.
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But in 40 per cent of patients a major complication called graft versus host disease occurs, where the donor’s immune cells mistakenly attack the patient’s normal cells.
Clinical haematologist Associate Professor David Curtis, also from Monash University’s Australian Centre for Blood Diseases, said this immune reaction could happen in the first three months after transplant, causing widespread rashes and hepatitis, or as late as two years afterwards, when severe scarring of the skin and joint immobility occurred.
“It’s similar to organ rejection, but the reverse. The new immune system is trying to reject the rest of the body,” Prof Curtis said.
“It’s an awful thing. The treatment is done to cure the patient, but many are left with problems requiring ongoing medication, frequent hospital visits, and it takes away their ability to work. They are reminded every day of what they’ve had to go through.”
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For the past 30 years, the drug methotrexate has been given to patients for three months after their transplant, with the aim of suppressing this immune response.
Cortisone steroids are also used to treat the condition, with limited success. And they can have serious side effects such as osteoporosis, which leads to fractures.
But Prof Curtis said an older drug called cyclophosphamide, used to treat other auto-immune conditions and administered as part of newer types of transplant where a so-called “half-matched” donation from a sibling isn’t available, was emerging as a better option.
He and his team have seen promising results using the drug instead of standard treatment, and through a grant from the Medical Research Future Fund they will run a trial to conclusively compare its effectiveness in 134 patients.
“One of the nice things is this drug has been around for 30-40 years, we know it’s safe, cheap and not a fancy drug,” Prof Curtis said.
“If this works, it will make a difference to the patients’ quality and length of life.”