LIFELAB: Clinical Trial

Clinical trial gives hope to patients with brain cancer

In a Phase 1 clinical trial, Queensland researchers have found that patients who received a cellular immunotherapy for the deadly brain cancer glioblastoma multiforme (GBM) on average survived longer than would have been expected without the treatment.

GBM is the most common malignant brain tumour in adults and sadly is almost always fatal; patients survive an average of 14-17 months after diagnosis. It is a particularly aggressive cancer that usually recurs quickly after treatment.

In Australia, brain cancer kills more children than any other disease, and kills more people under 40 in Australia than any other cancer. While other cancers have seen higher survival rates in recent years, brain cancer has not, and the chance of surviving five years after diagnosis is only 20% (AIHW).

Brain cancer treatment is very challenging, due to many reasons, including the brain’s natural defenses, accessibility of the tumors and their ability to spread rapidly.

The cellular immunotherapy used in the recent trial was developed by the head of QIMR Berghofer’s Centre for Immunotherapy and Vaccine Development, Professor Rajiv Khanna AO, and his team.

Immunotherapy is a type of treatment that uses our own immune systems to fight cancer and other diseases. There are many different types of immunotherapy, which can either enhance the immune response or remove the barriers that prevent the immune system from fighting the cancer or disease.

Immunotherapy is not yet as widely used as surgery, chemotherapy and radiotherapy for the treatment of cancer, but there are many ongoing clinical trials like this one, testing new immunotherapies. These therapies are sometimes used alone, or they are combined with existing treatments for the disease.

This GBM treatment was tested in a phase I (safety) clinical trial of 25 patients at Briz Brain and Spine in collaboration with neurosurgeon Professor David Walker.

The cellular immunotherapies were produced by taking blood samples from patients and ‘turbocharging’ their immune cells, known as T cells, in the laboratory to recognise and destroy the Immunology cytomegalovirus (CMV), which is present in the tumour cells.

The treatment each trial patient received was produced with their own immune cells and a genomic analysis of each patient’s immunotherapy and found that patients with certain gene signatures inside their immune cells had better results.

Professor Khanna said the patients were given the immunotherapy as a supplementary treatment after receiving surgery, chemotherapy and/or radiation.

‘At the end of this clinical trial, 10 patients were still alive and five of those still had no signs of their cancer recurring,’ Professor Khanna said.

The 25 patients who received the treatment survived for an average of 21 months. This is an improvement on the current survival rates.

The results were even better in the 20 patients who received the immunotherapy before their tumours had recurred. Those patients survived for an average of 23 months, which is six to nine months better than we would have expected without this treatment.

Considering GBM is a highly deadly cancer, these are very promising results.

According to Professor Walker, brain cancer survival rates have hardly changed in decades, and this trial gives hope that we might be on the cusp of changing that.

‘None of the patients experienced any adverse effects from the immunotherapy, which gives us confidence in progressing further clinical trials,’ Professor Walker said.

Queensland’s Deputy Premier and Minister for Health, Steven Miles, has also welcomed the promising results.

‘Brain cancers are usually aggressive and have poor survival outcomes, so any new treatment that prolongs patients’ lives is very welcome,’ Mr Miles said.

‘It is great to see Queensland’s medical research institute working at the global forefront of research into immunotherapy.’

The team now hopes to conduct another clinical trial combining the cellular immunotherapies with other forms of immunotherapy.

The cellular immunotherapies were produced at QIMR Berghofer’s specialist cell manufacturing facility, Q-Gen Cell Therapeutics and was funded by the National Health and Medical Research Council (NHMRC) and philanthropic donors.

Professor Rajiv Khanna
Group Leader
Tumour Immunology



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