This clinical trial is testing a new cure for a deadly rare blood cancer

In 2018 Scott Griffiths developed a deadly rare blood cancer in his brain. He believed he was going to die. But a new precision medicine developed by his doctor Professor Maher Gandhi cured Scott. Maher is conducting a clinical trial to test if his new treatment works for other patients.

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Finding a treatment for rare lymphomas

The common Epstein Barr virus caused Scott Griffiths’ rare blood cancer. An organ transplant had weakened his immune system, and this made him vulnerable to the virus. This blood cancer is called a lymphoma because it begins in the lymph glands.

Brain lymphomas are hard to treat because drugs must pass through the protective blood brain barrier. Scott’s doctor, Professor Maher Gandhi from Mater Research Institute, University of Queensland, explains:

‘The conventional approach to treating these cancers is to prescribe a high dose of drugs so that some of the drugs pass into the brain. The problem with that is most of the drugs stay in the body, and they are toxic and horrible.

‘Because Scott was already so unwell, I could not treat him using this conventional therapy.’

Special access to a ground-breaking new therapy

Maher and his team were developing a more effective way to get medicine through the blood brain barrier using biological precision medicine.

‘There are two ways we can do this,’ Maher explains. ‘We can use small molecules and we can use cellular therapy because small molecules and cells can pass through the blood brain barrier. We decided to combine both.’

The first step is a tablet that contains a small molecule drug called Ibrutinib. Doctors use this drug to prevent other kinds of blood cancer cells from growing.

‘Ibrutinib isn’t available to treat Epstein Barr virus lymphoma but we felt there was enough data to suggest it would work,’ Maher says. ‘The company that produces the drug gave it to us on trial so we can test if it works for this lymphoma.’

The second step of the treatment is cellular therapy. Maher obtains healthy white blood cells from donors who are tissue matched with the patient. The white blood cells are part of our immune system. Some of them can recognise and attack the virus. Maher and his team select and grow these cells in the lab and give them to the patient in a large dose.

‘The white blood cells attack the patient’s cells that contain the virus. Since these cells are the lymphoma cells, it's another way of attacking the cancer,’ Maher explains.

Low toxicity

‘The two treatments combined are not very toxic,’ Maher adds. Even though the patients are extremely sick and can’t tolerate difficult therapies, they can tolerate this new treatment as outpatient therapy.’

Maher’s new treatment worked for Scott and another patient. ‘They did extremely well and are still alive,’ Maher says.

A paradigm shift for treating rare cancers

Maher is now completing a clinical trial of this new lymphoma treatment, supported by a $1.64 million grant from the MRFF. The trial gives 20 other patients the chance to receive the therapy. Maher says this opportunity is improving health equity.

‘Up until now patients with such rare cancers have not had access to new treatments in clinical trials,’ Maher says. ‘Financially the drug companies do not see a return because it's a rare cancer.

‘Without the MRFF grant the trial would not have taken place.’ Maher’s next step will be to analyse the data from the trial. He plans to publish the results in 2024.

A lifelong quest for a cure

Maher has been trying to improve cures for blood cancer since he first began treating leukaemia as a young research fellow. ‘We treat leukaemia with a bone marrow transplant,’ he says. ‘This process takes all of the immune system from the donor and puts it into the patient.

‘The cells are not in any way selected and some of those cells cause problematic reactions. Although it works well in some patients, I realised there had to be a cleverer way. I thought, wouldn't it be better if we just took the part of the immune system we needed?

‘That’s what we do in this trial. We only give the patient the cells that kill the Epstein-Barr virus.’

Persevering against opposition and scepticism

In earlier days, Maher faced opposition and scepticism about his ideas. But that changed when Maher’s biological precision medicine began curing patients. In 2021 Maher’s colleagues nominated him for a Research Australia Frontiers Award to recognise this achievement.

‘Developing better cures for rare cancers has been my life's work,’ Maher says. ‘I am proud that I persevered with something I believe in.’

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