Cutting edge treatment
In 2022, at 13 years old, Alyssa became the first patient in the world to be treated with a new therapy using cutting edge genetic technology to treat her blood cancer, using cells from Anthony Nolan donors. Three years on, she is disease-free and interested in pursuing her own career as a cancer researcher.
Now a further eight children and two adults have undergone the treatment at Great Ormond Street Hospital (GOSH) and King’s College Hospital (KCH). The clinical trial, published this month, shows promising results in helping patients achieve remission.
The research shows the potential that new cell therapies can have for patients – especially those who have limited treatment options – and how Anthony Nolan donations to medical research are helping to make new breakthroughs in treatment possible.
The treatment explained
The world-first treatment uses a cutting-edge type of gene editing called ‘base editing’ to modify a type of immune cell called T cells so that they can treat T-cell acute lymphoblastic leukaemia (T-ALL).
The majority of current CAR-T therapies treat B-cell cancers. It’s been harder to develop a CAR-T therapy that targets T-cell cancers because the CAR T-cells could end up targeting themselves.
Using base editing, a GOSH and University College London (UCL) team were able to design unique CAR T-cells that could hide from being accidentally destroyed by other CAR T-cells, while also being able to target cancerous T cells in patients.
After the treatment has killed the patient’s cancer cells, they can then have a stem cell transplant to help rebuild their immune system and maintain their remission.
Of the 11 patients treated with this world-first CAR-T therapy, 82% reached remission and 63% are now classified as disease-free.
The treatment offers a new hope to patients who have limited treatment options while suffering from T-ALL.
How our donors helped
While CAR-T is often developed from a patient’s own cells, this particular treatment was developed using cells from donors. Part of the base editing process allowed cells to be modified so that close genetic matching between patients and donors was not necessary.
Anthony Nolan donors provided the blood cells required to manufacture the treatment, showing how far our cells can go beyond transplants. Stem cells can do more than making transplants possible – they can also be used to investigate and develop new cellular therapies like the one that has helped the patients in today’s trial.
Saving lives through stem cells
One of our key aims is to support the development of new cell therapies that can help more patients survive and thrive in future. This work is only possible thanks to our generous donors who volunteer to donate their cells for medical research and treatments. Each donation has the chance to be used to create a therapy that could end up treating thousands of patients.
We also conduct our own research into new cell therapies that have the potential to support stem cell transplants. Overall this helps us have the biggest impact we can in making sure more patients survive and thrive thanks to cell therapies.
Follow Alyssa through our labs
Earlier this year, Alyssa – the first patient who received this groundbreaking treatment – joined us at the Anthony Nolan Research Institute to learn more about our work. Since she’s interested in becoming a cancer researcher herself, she had lots of great questions to ask our researchers, and even had a chance to try out some lab work! You can watch her visit below: