How Cell Therapy Emerges as the Beacon of Hope for Immunocompromised Patients

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Healthcare (Commonwealth Union) – A groundbreaking immunotherapy designed to combat severe viral infections has saved the lives of numerous critically ill, immunocompromised Australians treated under compassionate access programs.

The therapy, created by Professor Rajiv Khanna and his team at QIMR Berghofer, is made available through the Therapeutic Goods Administration’s (TGA) Special Access Scheme. This program permits the use of unapproved medical treatments as a last resort for patients with no other options.

The Queensland-based researchers work closely with top clinicians across Australia to provide the innovative cellular immunotherapy. Their extensive 15-year study appeared recently in Nature Communications, highlighting the therapy’s impact.

Professor Khanna explained that the study documents clinical improvements in 46 out of 71 patients who received the treatment, demonstrating its potential to effectively manage complex diseases in critically ill individuals.

Professor Khanna indicated that this clinical experience highlights the effectiveness of our breakthrough cellular therapy in targeting viruses with minimal side effects, saving the lives of numerous children and adults who had exhausted all other options.

He further indicated that many patients come to them at an advanced stage of their illness after multiple treatments, resulting in a success rate of around 65%. They believe earlier access to this therapy could significantly increase survival rates.

Dr. Michelle Neller, Clinical Trials Manager with the Tumour Immunology Group at QIMR Berghofer, explained that the therapy targets uncontrolled viral infections caused by common viruses such as Epstein–Barr virus (EBV), cytomegalovirus (CMV), BK polyomavirus (BKV), John Cunningham virus (JCV), and adenovirus (AdV).

Dr. Neller further indicated that these viruses typically cause mild or no symptoms in healthy individuals but can become life-threatening in immunocompromised patients, where standard treatments may fail, cause severe side effects, or are unavailable.

Researchers of the study pointed out that viral infections are a leading cause of mortality among vulnerable groups, including children undergoing stem cell transplants for leukemia, organ transplant recipients, and individuals with genetic conditions that impair their immune systems.

One notable success story is Koby, a young boy from Perth. At just eight years old, his mother, Jodi, was told he had less than a 10% chance of surviving a severe viral infection. Koby is one of the recipients who benefited from the QIMR Berghofer therapy.

“Koby breezed through his bone marrow transplant, but then he caught adenovirus. It just got worse and worse. He was riddled with it, and we couldn’t get on top of it,” said Jodi.

“He had the first infusion of the therapy and in a week, he was a different child. In two weeks, they were talking about discharging him from hospital. The virus was just gone.”

Perth Children’s Hospital Consultant Paediatric Oncologist Dr. Shanti Ramachandran Stunned by Koby’s Remarkable Recovery.

“Koby had no more pain and he was able to eat and mobilise. It was unbelievable. In the era where we didn’t have this therapy, we would lose these children. And the cost is so relatively cheap compared to the cost of hospitalisation,” explained Dr Ramachandran.

Cellular therapy, often referred to as adoptive immunotherapy, has proven to be a powerful approach for treating cancer, autoimmune disorders, and infectious diseases. Despite its potential, it has not yet received approval for use in managing severe viral infections.

In Australia, QIMR Berghofer is the exclusive provider of this therapy for compassionate use. The treatment is produced at the Institute’s Brisbane-based cell therapy manufacturing facility, Q-Gen Cell Therapeutics.

Professor Khanna emphasized the need for dedicated funding to sustain the growing demand for this immunotherapy program. He also expressed hope that such funding could facilitate further investment in QIMR Berghofer’s cellular therapy development facilities, enabling them to meet the increasing needs of clinicians and patients across the nation for this and other groundbreaking treatments.

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