Hopes for thousands of people battling two deadly blood disorders as UK approves world’s first ‘cure’: 1m drug allows sufferers to feel ‘born again’

Thousands of Britons suffering from two blood disorders could receive the first treatment that could potentially cure them.

Casgevi was today approved by the UK medicines watchdog for everyone over the age of 12 after ‘rigorous’ safety, quality and effectiveness checks.

The drug, which is expected to cost about one million per patient, treats sickle cell disease and the transfusion-dependent disease thalassemia, lifelong conditions that, in severe cases, can be fatal.

Experts say the gene-editing treatment acts as a “functional cure” for both disorders, removing the faulty gene andrelief of symptoms.

However, it will only be introduced to the NHS if it receives further approval from the National Institute for Health and Care Excellence (Nice).

Patients with sickle cell disease, of which there are around 15,000 in the UK, do not properly make hemoglobin, the substance in red blood cells that carries oxygen around the body. As a result, their red blood cells become rigid and crescent-shaped instead of disc-like (pictured), which can cause them to die and get stuck in blood vessels

Jimmy Olager, 36, who lives in the US, suffered from sickle cell disease since childhood and was hospitalized almost every month

Jimmy Olager, 36, who lives in the US, suffered from sickle cell disease since childhood and was hospitalized almost every month

Both genetic conditions are caused by errors in genes for hemoglobin, which red blood cells use to carry oxygen around the body.

Patients with sickle cell disease, of which there are around 15,000 in the UK, do not properly make hemoglobin, the substance in red blood cells that carries oxygen around the body.

As a result, their red blood cells become rigid and crescent-shaped instead of disc-shaped, which can cause them to die and become stuck in blood vessels.

Patients experience bouts of severe pain that can last for days or weeks, serious and life-threatening infections, and anemia, which can cause fatigue and weakness.

About 1,000 Britons have transfusion-dependent thalassemia. This group has a lack of healthy red blood cells, leading to severe anemia.

How does Casgevi work?

Casgevi, made by Vertek Pharmaceuticals and Crispr Therapeutics of Switzerland, works by editing the faulty HBB gene behind both conditions in the patient’s bone marrow stem cells so that the body produces functional hemoglobin.

To do this, stem cells are taken from the patient’s bone marrow and edited in the lab using molecular “scissors” that precisely disable the faulty gene.

The stem cells are then reinfused into the patient, who may have to spend a month or more in the hospital while the treated cells start making healthy red blood cells.

The results have the potential to be lifelong.

An ongoing trial of the drug so far shows that 97 percent of sickle cell patients were free of severe pain for at least a year after treatment.

In a separate study for -thalassemia, 93 percent of participants did not need a blood transfusion for at least a year. Among those who did, their need for transfusions dropped by 70 percent.

Side effects included nausea, fatigue, fever and an increased risk of infection.

They often require monthly blood transfusions to survive and require lifelong injections and medication. If left untreated, the condition can cause organ damage and be fatal.

Casgevi, made by Vertek Pharmaceuticals and Crispr Therapeutics of Switzerland, works by editing the faulty HBB gene behind both conditions in the patient’s bone marrow stem cells so that the body produces functional hemoglobin.

To do this, stem cells are taken from the patient’s bone marrow and edited in the lab using molecular “scissors” that precisely disable the faulty gene.

The stem cells are then reinfused into the patient, who may have to spend a month or more in the hospital while the treated cells start making healthy red blood cells.

The results are thought to have the potential to last a lifetime.

An ongoing trial of the drug so far shows that 97 percent of sickle cell patients were free of severe pain for at least a year after treatment.

In a separate study for -thalassemia, 93 percent of participants did not need a blood transfusion for at least a year. Among those who did, their need for transfusions dropped by 70 percent.

Side effects included nausea, fatigue, fever and an increased risk of infection.

No significant safety concerns have been identified, but the Medicines and Healthcare products Regulatory Agency (MHRA) and drug manufacturers will continue to monitor patients.

Casgevi is the first licensed drug to use the innovative gene-editing tool CRISPR, known as “genetic scissors” that allow scientists to make precise changes to DNA. Its inventors won the 2020 Nobel Prize.

Until now, a bone marrow transplant from a close donor has been the only long-term treatment for these two blood disorders.

However, they are not often performed because of the risks, which include the transplanted cells attacking other cells in the body, which can be life-threatening.

Julian Beach, interim chief executive for healthcare quality and access at the MHRA, said: “I am delighted to announce that we have approved an innovative and first-of-its-kind gene editing treatment called Casgevi.”

He added: “The MHRA will continue to closely monitor the safety and efficacy of Casgevi, through real-world safety data and post-approval safety studies conducted by the manufacturer.”

“I would like to thank the lived experience patients who engaged with us as part of the assessment process and gave us valuable insight into their lives and the challenges of managing their condition.”

John James OBE, chief executive of the Sickle Cell Society, said: “Sickle cell disorder is an incredibly debilitating condition that causes significant pain for people living with it and potentially leads to early death.

“There are a limited number of medicines currently available to patients, so I welcome today’s news that a new treatment has been assessed as safe and effective, which has the potential to significantly improve the quality of life for so many.”

The price of Kesgevy has not yet been disclosed by drugmakers, but it would cost more than a million, based on the price of similar drugs.

This could make it too expensive to be approved in the UK.

Reality TV star Rosie Williams, from Glamorgan, Wales, previously shared her battle with thalassemia minor, a less severe version of transfusion-dependent thalassemia

Reality TV star Rosie Williams, from Glamorgan, Wales, previously shared her battle with thalassemia minor, a less severe version of transfusion-dependent thalassemia

Casgevi, made by Boston-based Vertek Pharmaceuticals (pictured) and Crispr Therapeutics in Switzerland, works by editing the faulty HBB gene behind both conditions in the patient's bone marrow stem cells so that the body produces hemoglobin that works.

Casgevi, made by Boston-based Vertek Pharmaceuticals (pictured) and Switzerland’s Crispr Therapeutics, works by editing the faulty HBB gene behind both conditions in the patient’s bone marrow stem cells so that the body produces hemoglobin that works.

In 2021, Nice rejected the agent therapy Zinteglo, made by US-based Bluebird Bio, for the treatment of transfusion-dependent thalassemia.

It concluded that the trials were too small and that the cost of 1.45 million per patient was too high. However, the drug was approved in the US last year.

Jimmy Olager, 36, who lives in the US, suffered from sickle cell disease since childhood and was hospitalized almost every month.

The tech entrepreneur told the BBC the condition feels like “shards of glass running through your veins or someone hitting your wrists with a hammer.”

“You wake up in the morning with pain and go to bed with pain,” he said.

However, Mr Olager became one of the first patients to have the revolutionary new gene-editing treatment as part of Vertek Pharmaceuticals and Crispr Therapeutics’ clinical trials in the US in 2020.

He said he woke up without any pain and “like he was reborn.”

‘When I look back, it’s like, “Wow, I can’t believe I lived with that.”

Reality TV star Rosie Williams, from Glamorgan, Wales, previously shared her battle with thalassemia minor, a less severe version of transfusion-dependent thalassemia.

She said: “It’s the kind of fatigue where you can’t function, you’re too tired to talk or eat and it makes me very sensitive to pain.

‘If I catch a finger in a door or hit my knee, the pain makes me feel unconscious.’

Dr Sarah Trompeter, consultant haematologist at UCLH and NHS Blood and Transplant, said: “Sickle cell disorder is a very complex, life-limiting and life-threatening disorder with very limited treatment options.

“While curative treatments may not be suitable for everyone, gene therapy offers a real chance of a cure for those who do not qualify for a bone marrow transplant and we are therefore delighted that it has been approved by the MHRA.”

“We look forward to NICE approval so this can be delivered, free of charge to patients, across the NHS.”

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