The first treatment for sickle cell disease in over 20 years will soon be available to thousands of patients like Toks Odesanmi.
People with the condition endure severe pain during a ‘sickle cell crisis’ that can occur multiple times a year.
This often requires hospital treatment so the pain can be controlled by morphine and to prevent organ failure.
The new treatment is a drug called Crizanlizumab and has been trialled at Cambridge University Hospitals NHS Foundation Trust (CUH).
Sickle cell disease has defined me, defined my body and made a big dent to my dreams.
Toks Odesanmi, a sickle cell patient at CUH
Toks Odesanmi has been treated for sickle cell disease at CUH for a number of years and has needed extensive surgery including a liver transplant, hip replacements, ankle fusion and laser eye treatment. She keeps her symptoms under control by having blood transfusions every eight weeks. She said:
“No matter how hard I fight it continues to defeat me. A new treatment brings hope and might make dreaming possible again."
Patients with sickle cell suffer from monthly episodes, making it difficult for people to continue their jobs or other everyday activities.
The hereditary condition is much more prevalent among people from African or African-Caribbean origin.
It's hoped the new treatment will reduce the number of times a sickle cell patient needs to go to A&E by two fifths.
Crizanlizumab will transform sickle cell treatment.
Dr Martin Besser, CUH consultant in haematology
Dr Martin Besser has been leading the CUH trial into Crizanlizumab. He said:
"Despite only having one per cent of the UK sickle cell population under our care , CUH has become the UK lead recruiter for this trial.
"Through its rapid action within 2-4 weeks of infusion, this new drug will allow us to target the worst affected patients, giving them additional education and preventative care when they attend in the day unit."
The disease is characterised by unusually shaped red blood cells that are produced which can cause serious health issues across the body, sending organs into crisis and causing extreme levels of pain.
The new treatment will be delivered by a intravenous drip and works by binding to a protein on white blood cells, platelets and blood vessels. This prevents the adhesion of sickle cells which causes the blockage of vessels and shutdowns oxygen supply, leading to a sickle cell crisis.
This is a historic moment for people with sickle cell disease who will be given their first new treatment in over two decades.
NHS chief executive, Amanda Pritchard
Announcing the new treatment, Amanda Pritchard, NHS chief executive, said the drug deal, struck by the NHS, would help as many as 5,000 people over the next three years to have a much better quality of life. She said:
“This revolutionary treatment will help to save lives, allow patients to have a better quality of life and reduce trips to A&E by almost half.
“The NHS has agreed a deal for this drug, so we are able to provide the latest and best possible treatments for patients at a price that is affordable for taxpayers”.
The announcement paves the way for the National Institute for Health and Care Excellence (NICE) to publish final guidance on Crizanlizumab which will take into consideration the data that will be collected as part of the agreement.
People aged 16 and over who suffer from multiple sickle cell crises, or vaso-occlusive crises as they are scientifically known, per year will be eligible for the treatment.