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Promising results from world-first brain cancer trials

Scientists at the QIMR Berghofer Medical Research Institute have used immunotherapy to make a major breakthrough in the treatment of the aggressive brain cancer Glioblastoma Multiforme (GBM).

Study leader, Professor Rajiv Khanna, said most of the study participants lived much longer than the six-month prognosis normally given to a patient with recurrent GBM, and some patients showed no signs of disease progression.

“It is early days, but this is exciting,” Professor Khanna said.

“Survival rates for this aggressive cancer have barely changed in decades. There is an urgent clinical need for new treatments.

“If this treatment can buy patients more time, then that is a big step forward.”

GBM is the most common malignant brain cancer, diagnosed in about 800 Australians every year. Despite surgery, radiotherapy and chemotherapy, less than 10% of patients survive beyond five years.

This study built on previous research which found that many brain tumours carry cytomegalovirus (CMV). About half of all Australians have the virus, but usually show no symptoms.

Professor Khanna developed a technique to modify the patients’ T-cells in the laboratory, effectively “train” them to attack the virus, and then return them to the patient’s body. When the killer T-cells destroyed the virus, they also destroyed the cancer.

“It’s becoming increasingly clear that immunotherapy – manipulating a person’s own immune system – is a rich new frontier for cancer treatment,” Professor Khanna said.

The Phase I trials were conducted at Brisbane’s Wesley Hospital, under the leadership of neurosurgeon Professor David Walker.

“Working with patients with malignant brain tumours can be distressing, because we know so many will succumb,” Professor Walker said.

“But this new branch of therapy lets us offer some hope that the future is going to be brighter, that new and innovative treatments mean things will hopefully improve in the future.

“We have a long way to go, and there is hard work to be done, but we seem to be on the right track, and it is a pleasure to work with scientists at QIMR Berghofer to try to make a real difference.”

The research team is now keen to begin the next phase of trials, involving patients at an earlier stage of the cancer’s development.

“These would be patients who have just been diagnosed and are about to start the standard treatments – surgery, then radiotherapy or chemotherapy.  We would generate the T cell therapy before their standard treatments an then administer T cells in conjuction with the standard therapy,” Professor Khanna said.

“We hope that the treatment can be even more effective if given at an earlier stage of the disease.”

This study is published online today in the prestigious US journal Cancer Research and can be viewed at

The research was funded by Flagship Funding from the Rio Tinto Ride to Conquer Cancer, the NHMRC and private donors.