Patients and charities react to NICE decision to reject gene therapy treatment for sickle cell disorder

Anthony Nolan responds to NICE's draft decision to block CRISPR gene treatment Exa-cel for sickle cell disorder.
March 14, 2024

Stem cell charity Anthony Nolan and the Sickle Cell Society are calling on NICE (the National Institute for Health and Care Excellence) to reconsider its draft decision to reject the CRISPR gene therapy, exa-cel, as a treatment for sickle cell disorder.

Exa-cel, also known as brand name Casgevy, involves modifying a patient’s own stem cells to treat sickle cell. The treatment was approved by the medical regulator MHRA in November for use in the UK as a treatment for sickle cell, followed by FDA approval in the US in December.

Sickle cell can be completely debilitating as patients can suffer from acute pain episodes known as ‘crises’. Crises are a physical, emotional, and mental burden to patients. Sickle cell also has a chronic effect on almost every major organ system, impacting life expectancy and day-to-day quality of life. 

Currently the only curative treatment available to people with sickle cell in the UK is a donor stem cell transplant – however not all patients have a suitable donor available. Exa-cel would provide a ‘functional cure’ for anyone unable to have a stem cell transplant.  

Anthony Nolan and the Sickle Cell Society are urging NICE and pharmaceutical company Vertex to find a solution so this provisional decision can be reversed without delay.

Caitlin Farrow, Director of Strategy and Influencing at Anthony Nolan, said: “We are deeply disappointed with this decision. Sickle cell is one of the most common genetic conditions affecting people in England from predominantly African and African-Caribbean backgrounds, and this community has been waiting years for a new, effective treatment.  

“There is a longstanding history of the experiences of people with sickle cell not being taken seriously. It’s not fair that the community is being asked yet again to explain the severely negative impact this disease has on their lives. We urge NICE and Vertex to find a solution to deliver this much-needed treatment to patients as soon as possible.”

People who live with sickle cell have expressed their extreme disappointment.

Funmi Dasaolu, 31 from Oxfordshire, said: “Sickle cell disease has a profound impact on all aspects of my life. I can't remember a day when I haven't experienced pain. 

“I’ve tried multiple treatments to manage my symptoms but nothing has worked long term. Having access to gene therapy would be life-changing for me and many others, so I am beyond frustrated.”

Toby Bakare said: “This decision will upset and disappoint many who live with this disease and have very few options. There are so many people I know who live with acute life-long pain, chronic fatigue and regular crisis events who are constantly in and out of hospital… without access to newer treatments they’ll live spend their whole lives without hope, managing pain and living with a cruel disease.”

Following NICE’s provisional recommendation, there is now a period of consultation before a second NICE committee meeting to consider the evidence and comments from stakeholders. Charities Anthony Nolan and the Sickle Cell Society are calling on people with lived experience and healthcare professionals to contribute to the consultation and inform NICE’s final recommendation. The deadline to respond is 11 April 2024.

Sickle cell is a genetic disorder that affects the shape of red blood cells, which take on an abnormal sickle shape. These abnormal cells can get stuck in small blood vessels, leading to blockages that can cause pain, organ damage and other complications. Exa-cel (Casgevy) treatment involves extracting and modifying a patient’s own blood stem cells, before returning them to their body – these modified cells will then produce healthy red blood cells.

People with sickle cell are more susceptible to life-threatening events such as strokes, blood clots, congestive heart failure and liver failure. There are approximately 17,500 people living with sickle cell in the UK.

To contribute to the ongoing NICE consultation of exa-cel for sickle cell, visit:

Click here for FAQs on NICE's appraisal of exa-cel for sickle cell disorder.