An evening of research: Rewriting the future of child health
11 Apr 2024, 9:20 a.m.
In February, GOSH joined organisations from all over the world in celebrating Rare Disease Day, raising awareness for patients, families and carers around the world that are affected by rare diseases.
The evening began with an opportunity to tour the world class laboratories in GOSH’s research facility, The Zayed Centre for Research into Rare Disease in Children.
This was followed by a panel discussion of world-class GOSH researchers, Professor Waseem Qasim and Professor Claire Booth, hosted by BBC Medical Editor, Fergus Walsh. The panel shared their insights in the field of gene and cell therapies and the pioneering breakthroughs these have enabled for children with rare diseases.
A global centre of excellence for gene therapy
Since the first child in the UK was treated with gene therapy at GOSH in 2001, the hospital has become a world leader for gene therapy and has treated more patients with the technology than anywhere else in Europe.
Among those leading gene and cell therapy clinical trials for children with cancer is Professor Waseem Qasim. He is a Consultant Immunologist at GOSH and a Professor of Cell and Gene Therapy at UCL Great Ormond Street Institute of Child Health (UCL GOS ICH).
Speaking on the panel, Professor Waseem Qasim shared the story of the first leukaemia patient he treated with pioneering CAR T-cell therapy.
“It was called a breakthrough because it was the first example of cells being taken out [of a patient] and edited”
“Some time ago a one-year-old girl who'd come by our haematology colleagues and then had a very rough time with a type of blood cancer called B-cell leukaemia. She had been through chemotherapy, had been through a bone marrow transplant, and the disease had come back.
"And at the time we were just getting going with treatments where you can use those disabled viruses to make CAR-T cells from the patient's blood.
"However, this wasn’t possible for the patient as her blood counts were so low. So, we put together a treatment that was based on taking donor cells from a healthy donor and using new gene editing techniques at the time called Talons to make the cells in such a way that they could be used without causing harm.
"We disarm them so they can only do one job, fight leukaemia. And we make them invisible to some of the drugs that are being used at the same time.
"It turned out to be incredibly powerful and cleared the disease within a matter of four weeks.
"The patient is now at school, living a normal, unrestricted lifestyle and is doing well.
"That was a world first and it was an extraordinary breakthrough.”
The Genomics lab in the Zayed Centre for Research into Rare Disease in Children
Professor Claire Booth is a leader in gene and cell therapies for children with immune and metabolic conditions. She is a Consultant Paediatric Immunologist at GOSH and a Professor of Gene Therapy and Paediatric Immunology at UCL GOS ICH.
Speaking on the panel, Professor Claire Booth shared recent successful results of a stem cell gene therapy clinical trial for a rare form of immunodeficiency called ADA-SCID.
“What we're doing is taking the patient's own blood stem cells, taking them outside of the body into the labs that you've seen upstairs, and using the virus or techniques to correct the cells outside of the body and then giving them back to the patient afterwards.
"By correcting the gene in the blood stem cells, you could give those patients a working immune system for the rest of their life”.
Professor Claire Booth and her team have treated over 100 patients through their pioneering research, enabling children who were not able to go to school or be socialised, to do exactly that, with no medicines.
“Whatever they want to do, they can do it.”
"One of the amazing things about working in a place like this is that that information that you get from treating patients, you can feed back into the research and then develop safer and more effective treatments for patients in the future."
Every child deserves a breakthrough.
Many of the children who pass through the doors of GOSH have rare or complex conditions that are life-limiting or life-threatening. It is often a place of last hope and with that comes a huge responsibility to make life-changing differences for the children and their families.
Here at GOSH Charity, we are the UK’s largest dedicated funder of child health research. We believe it is our role to accelerate child health research and provide hope of a better future for seriously ill children through research-led care.
We are committing £70 million over five years towards research into rare or complex childhood diseases and improving the everyday experience for children and their families. This will enable us to build on our existing strengths and successes with the aim of making further, significant advances in the diagnosis, treatment and care of children with rare or complex conditions at GOSH, nationally and around the world, so that many more can survive and thrive into adulthood.