Revolutionary New Treatment Signals Hope for Sickle Cell Sufferers

•England approves first drug in 20 years

A major breakthrough has been made in healthcare, giving hope to millions of Sickle Cell Disease (SCD) sufferers across the globe, especially in Nigeria which currently ranks highest in rate of SCD in the world, as the first treatment for the disease in over 20 years has been approved in England by the National Health Service (NHS).

The drug, Crizanlizumab, made by Novartis, is injected into a vein and can be taken on its own or alongside standard treatment and regular blood transfusions.

And in a trial, patients taking the crizanlizumab had a sickle-cell crisis 1.6 times a year on average, compared with nearly three times a year normally.

The NHS which disclosed this in a statement posted on its website yesterday, described the development as revolutionary.

People with SCD endure severe pain during the ‘sickle cell crisis’ that can occur multiple times per year, often requiring hospital admission so they can be given morphine to control the pain and prevent organ failure which can be fatal.

Announcing the new treatment, NHS’ chief executive, Amanda Pritchard, said the drug deal would help as many as 5,000 people over the next three years to have a much better quality of life.

Nigeria has an estimated 25 per cent of its adult population being carriers of defective S-gene. SCD is a genetic (hereditary) disorder that occurs when an individual has inherited two mutant (abnormal) haemoglobin (Hb) genes from both parents, at least one of which is HbS and the resulting symptoms and signs are due to abnormality in the shape of red blood cells.

The Minister of Health, Dr. Osagie Ehanire recently disclosed that available record showed that SCD affects nearly 100 million people in the world and was also responsible for over 50 per cent of deaths in those with the most severe form of the disease (Hb SS).

He had said sickle cell was the most prevalent genetic disease in Africa, adding that in many countries in the country, including Nigeria, 10 to 40 per cent of the population carry the sickle-cell gene resulting in estimated SCD prevalence of at least two per cent.

He had estimated that 150,000 affected children are born every year in Nigeria.

“Nigeria currently has the highest burden of Sickle Cell Disease in the whole World ahead of Democratic Republic of Congo and India, with an estimated 25 per cent of her adult population being carriers of defective S-gene.

“WHO in 2015 estimated that two per cent of new-borns in Nigeria are affected by sickle cell anaemia, giving a total of about 150,000 affected children born every year. About 50 per cent–80 per cent of the estimated 150,000 infants born yearly with SCD in Nigeria die before the age of five years and those that manage to survive suffer end-organs damage which shortens their lifespan including stroke,” Ehanire had said.

Continuing, Pritchard said patients with sickle cell suffer from monthly episodes, making it difficult for people to continue in their jobs or other everyday activities.

“The hereditary condition is much more prevalent among people from African or African-Caribbean origin.

The drug will also reduce the number of times a sickle cell patient needs to go to A&E by two fifths,” the NHS boss explained.

Last year, NHS England set up ten new dedicated centres to treat sickle cell disease across the country and patients would be able to access the new treatment through their consultant at one of these clinics.

The NHS agreed a deal to make Crizanlizumab available for patients earlier than would have otherwise been possible at a price that is fair for taxpayers.

It was the latest in a line of deals secured by the NHS, including a new cholesterol-busting jab and lung cancer drug last month as well as the world’s most expensive drug which treats spinal muscular atrophy in infants, earlier in the year.

Pritchard added: “This is a historic moment for people with sickle cell disease who will be given their first new treatment in over two decades.

“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.”

This announcement paves the way for the National Institute for Health and Care Excellence (NICE) to publish final guidance on Crizanlizumab which would take into consideration the data that would 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 would be eligible for the treatment.

Toks Odesanmi, a sickle cell patient at Cambridge University Hospitals Trust, was quoted to have said: “Sickle Cell Disease has defined me, defined my body and made a big dent to my dreams.

“No matter how hard I fight it continues to defeat me. A new treatment brings hope and might make dreaming possible again”.

The Chair of the Sickle Cell Society, Kye Gbangbola MBA, said: “A new treatment brings new hope for people living with Sickle Cell Disorder, the world’s most common genetic blood condition.

“SCD is a ‘medical emergency’; it causes excruciating pain, this new treatment will reduce the number of agonising pain episodes we have to endure. The hope is improved quality of life for many living with the condition and their families”.

Health and Social Care Secretary Sajid Javid said: “It is fantastic news that this ground-breaking new treatment for sickle cell disease will soon be available in England where it will make a difference to thousands of people’s lives.

“The UK has proven itself to be a world leader in rolling out innovative, life-saving treatments to ensure every NHS patient gets access to the medicines they deserve.

“This is a historic day for those living with this condition and I want to extend my thanks to the National Institute for Health and Care Excellence, NHS England and Novartis for their work to provide this vital treatment to the patients who need it.”

Novartis Oncology President, Susanne Schaffert, said: “Today’s decision is reflective of the new approach to partnerships with healthcare systems that Novartis is taking around the world, to drive equity of access for people living with potentially debilitating conditions such as sickle cell.

“We are pleased that this close partnership with NICE, N