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Hope for transplant patients after world-first Queensland immunotherapy trial

Organ transplant patients who face a lifetime of medication to ward off viral infection – or the potentially deadly threat of drug-resistant infection – may have new hope following a world-first clinical trial that uses a patient’s own immune cells as the treatment.

The immunotherapy treatment involved taking blood from patients, effectively training their killer T (immune) cells to destroy the cytomegalovirus (CMV) and reinfusing the cells into sick patients.

QIMR Berghofer Medical Research Institute’s world-leading immunologist, Professor Rajiv Khanna, led the clinical trial, which treated 13 organ transplant patients with a potentially life-threatening cytomegalovirus infection using their own immune cells.

The Phase 1 clinical trial started in 2014 in conjunction with Associate Professor Scott Campbell at Brisbane’s Princess Alexandra Hospital and Professor Dan Chambers at Brisbane’s Prince Charles Hospital, as well as doctors at the Royal Adelaide Hospital.

Professor Khanna, who is also the coordinator of QIMR Berghofer’s Centre for Immunotherapy and Vaccine Development, developed the immunotherapy treatment by using killer T (immune) cells from patients and training them to recognise and destroy the virus.

He said the phase 1 clinical trial showed the T cell therapy for existing or drug-resistant cytomegalovirus was safe and had provided a clinical benefit to some patients.

Eleven patients showed improved symptoms following the immunotherapy, and some of those patients were able to reduce or stop taking anti-viral medication altogether.

“The patients we treated were quite sick from the complications caused by cytomegalovirus, which is an infection experienced by some organ transplant recipients,” Professor Khanna said.

Associate Professor Campbell said that typically, a patient who has had an organ transplant received drugs to protect them from cytomegalovirus.

“But taking those drugs long-term means that some patients can develop a drug-resistant strain of the virus, and develop cytomegalovirus-associated complications due to their weakened immune system,” Associate Professor Campbell said.

“The anti-viral drugs designed to prevent and treat cytomegalovirus also have many other unpleasant or dangerous side-effects that can lead to severe illness, and can even make it impossible to continue using the drug.”

Professor Chambers said the killer T cell immunotherapy may in future provide doctors with another option to treat very unwell patients.

“We believe the treatment has potentially saved the lives of some patients,” Professor Chambers said.

Professor Khanna said the group had now developed a new, expanded and improved version of the therapy, and planned to commence a clinical trial next year, in collaboration with several clinical centres around Australia.

He said the new version of the immunotherapy also aimed to treat other viral infections that are common in solid organ transplant patients.

QIMR Berghofer’s Director and CEO, Professor Frank Gannon, said the immunotherapy was produced in-house at the Institute’s cell manufacturing facility, Q-Gen Cell Therapeutics.

“These early results are extremely promising and we hope in future it will provide a new and routine way to treat transplant patients who become sick as a result of cytomegalovirus infection,” he said.

The QIMR Berghofer-led research has been published today in the journal Clinical Infectious Diseases and was made possible with funding from the National Health and Medical Research Council.

The journal article is available online here: