Research at King’s Leads to New Treatment for Sickle Cell Disease
A ground breaking treatment for patients with beta haemoglobinopathies, including sickle cell disease, has recently been made available through the NHS, marking a significant leap forward in the management of these blood disorders. Utilising the cutting-edge CRISPR/Cas9 gene-editing technology, this therapy reprograms patients’ own bone marrow to activate a form of haemoglobin known as fetal haemoglobin, which is unaffected by the sickle mutation. By doing so, it protects red blood cells, significantly reducing the frequency and severity of painful episodes experienced by patients with sickle cell disease.
The Science Behind the Breakthrough
This revolutionary treatment is built upon the discovery of a molecular “off switch” for fetal haemoglobin—a transcription factor known as BCL11. This critical finding was first published by researchers at King’s College London in 2007, under the leadership of Professor Swee Lay Thein. The research continues to advance at King’s, with Dr. Stephan Menzel at the helm, alongside an expert team of academics including John Strouboulis, David Rees, Ghulam Mufti, and others. Their collaborative efforts are driving the development of innovative treatments to bring hope to those living with blood disorders like sickle cell disease.
The High Costs of a Life-Saving Treatment
While the therapy offers life-changing potential, the costs are considerable – each treatment currently exceeds £1.6 million, limiting its availability to a select group of patients. Despite this, the team at King’s and their international partners are undeterred in their mission to expand therapeutic options. The search for additional, more accessible treatments continues through collaborations with researchers in Nigeria, Tanzania, Brazil, and the United States, reflecting the global effort to combat these devastating diseases.
Future Research and Global Collaboration
Dr. Stephan Menzel’s research is generously funded by the Medical Research Council, and in partnership with John Strouboulis, they have recently secured a grant from the Fetal Medicine Foundation. This funding will support ongoing work with MiNA Therapeutics, a London-based biotech start-up, to explore new ways of activating fetal haemoglobin in patients. Additionally, King’s College Hospital Charity and LIBRA have provided critical support, underscoring the essential role of philanthropy in advancing life-saving research.
Help LIBRA Fund Vital Research
The first gene-editing treatment for beta thalassaemia has been hailed as a “revolutionary breakthrough,” offering the potential to cure patients and free them from the burden of lifelong blood transfusions. However, the journey is far from over. With the high cost of treatment and the need for ongoing research, we urgently need your support to continue this vital work. By raising funds for LIBRA, you can directly contribute to the development of new therapies for patients with sickle cell disease and other blood disorders.
Whether it’s participating in a fundraising event, organising a community bake sale, or donating through our website, every contribution brings us one step closer to making these life-changing treatments available to more people. Help us provide hope and life-saving care to those who need it most. Join us in the fight today!
Back to all posts