Maltese woman in groundbreaking cancer treatment

A Maltese woman was one of the early scientists whose experimental work which started almost 2 decades ago,   is now leading to ground breaking cancer treatment, as witnessed by the results of recent trials, carried out by professionals in the field of immunotherapy.

Dr Joanna Black (née Galea-Lauri) led a group of researchers between March 1995 and October 2004 on an idea about the possibility of tackling cancer using patients’ own immune system. Dr Black said ‘when i first had the idea with my seniors they didn’t understand how immunotherapy would work given the fact the cancer dampens down the immune system to the point we stop  making the right immue molecules for our defences’.  She persevered with the idea and obtained successful funding from the UK charity ‘Leuakemia Research Fund’ and describes her work as ‘great fun working with the most exciting immune cells ever: dendritic cells’. Dr Black built on the work of Dr Ralph Steinman who won a Nobel Prize for the recognition of dendritic cells and they met in the USA at a keynote conference where they exchanged ideas.

In 2018, the first medicines of their kind, where the patient’s cells are reprogrammed and re-injected to fight off specific cancer cells, were approved in the US, introduced to and approved by the EU, and are being introduced by the UK’s National Health Service.

The Chief of the NHS, Simon Stevens described this treatment known as CAR-T cell therapy as “one of the most innovative treatments that has ever been offered on the NHS”. Dr Tomas Salmonson, the chair of the EU’s Committee for Medicinal Products for Human Use also said that “innovative treatments such as CAR-T cells have potential to change the outlook for patients with cancer”.

What is CAR-T therapy?

The process works by first extracting T cells from the patient which are ordinarily there to fight pathogens, but fail to recognise some advanced cancers. These cells are then genetically modified to include surface receptors called Cars which allow the T-cells to recognise and attach to specific proteins on tumour cells. While the CAR-T cells are multiplying in the lab, the patient undergoes chemotherapy to remove the remaining T-cells. The blood is then transfused back into the patient’s body, thus allowing the newly modified cells to kill the cancerous ones.

As Dr Martina Schüssler-Lenz, the Chair of the Committee for Advanced Therapies concisely put it, “Kymriah and Yescarta (the two medicines approved in the EU) offer an innovative approach where patients’ cells are reprogrammed and re-injected to attack the cancer.”

Although pricing in the US is exorbitant with Yescarta priced at $373,000 (€316,000) and Kymriah up to $475,000 (€400,000), EU pricing will be regulated on a Member State basis. In the UK, the NHS said that they “need the help of the manufacturers to ensure we can get these treatments to patients as quickly and cost-effectively as possible” as the price range far exceeds the £50,000 per year ceiling.

How did it all start?

Dr Black’s team of researchers including but not limited to eight clinicians and six PhD students obtained funding from Cancer Research UK and the Leukemia Research fund, despite little faith in her immunotherapy idea. In comments to, she said that they “made great progress and published significantly and in the meantime other immunologists unravelled more how the immune system works.”

By 2002, they had enough data to apply to government and ethics agencies to start human trials, and after mountains of bureaucracy, they managed to get permission. The downside, however, was the price of this treatment which at the time cost £275,000 per patient and funding “became almost next to impossible”.

Dr Black’s journey into immunotherapy ended in 2004 after the birth of her son and a new career path and is currently the Head of Regulatory Affairs and Quality Assurance in Clinical Research. That being said, 14 years after she started, her work spread to others laboratories and now these medicines are finally being regulated and slowly introduced.

“Other scientists like Emma Morris (Professor of Clinical Cell and Gene Therapy at University College London Hospital) were starting to pick up our ideas from lectures we gave, papers we published, etc,” Dr Black said. “I feel very proud of this milestone because I recognised the importance of the immune system and braved my way through getting funding way back then.”

What next in immunotherapy?

While the trials has overall spectacular results, there are also risks and even deaths associated to the treatment. Prof Charles Swanton, Cancer Research UK’s Chief Clinician said about the treatment that “CAR-T cell therapies have shown great promise in some patients with specific types of blood cancer, but the treatment is complicated, highly personalised and can cause severe side effects in some patients.”

A Press Release by the European Medicine’s Agency explains that “the main safety concerns related to the administration of CAR-T cells are cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and neurological toxicities.” While both of these can be life-threatening, there are monitoring and mitigation strategies described in the product information, and part of the medicines’ authorisation was a risk management plan.