'I feel lucky to get remarkable Casgevy gene-editing therapy'
The NHS is one of the first health systems in the world to use the therapy for the condition, which mainly affects people of Asian, Mediterranean, and Middle Eastern descent. Mr Chronis was diagnosed with the inherited condition when he was just five years old and is severely anaemic. Since he was a child he has had blood transfusions every three weeks, but this causes its own problems as the body builds up an excess of iron.
'Live without worry'
Patients are often on a strong drug regime to deal with the side effects. They can also suffer damaged organs.Mr Chronis, a student, has already had to have his gall bladder removed."It would be fantastic if I could just live the rest of my life without having to worry about having to be near a hospital or having access to a medical team," he said. Previously the only effective cure for thalassaemia was for patients to have a stem cell transplant. But there is a national shortage of donors, and the procedure can also cause complications where the recipient's body rejects the donated cells.Casgevy uses the patient's own stem cells which are removed, edited over a six-month period so they produce haemoglobin, then reintroduced into the body via infusion. In international clinical trials the technique removed the need for blood transfusions for at least a year in 93% of patients.
Last August, NHS England negotiated a deal with the manufacturer Vertex which gives patients access to the treatment for the next five years while further evidence on its benefits to patients is gathered.Manchester Royal Infirmary is one of just seven sites in the UK to offer the treatment – the Royal Manchester Children's Hospital is another.Dr Nandini Sadasivam, a consultant haematologist who has been treating Mr Chronis for the past five-and-a-half years, said the treatment was not just life-changing, but potentially life-saving."The majority of people with beta thalassaemia are young. Survival is shortened and the mean age of death is about 55," she said."It's an amazing opportunity having this treatment finally arrive. It is something he has been waiting for, and we all have been waiting for, for a long time".
The stem cells arrive at the hospital in phials stored in liquid nitrogen at nearly -200 C (-328 F).Once the team is ready to perform the infusions, one by one the phials are gradually warmed in a saline solution before the contents are mixed and drawn up into a syringe to be taken into the treatment room. Time is of the essence – once the product has thawed it has to be given within 20 minutes.Mr Chronis received seven phials in a procedure taking three-and-a-half-hours. He had already spent a week in hospital for chemotherapy treatment to prepare his body.
Patients stay in hospital for several days after and are then monitored. It takes between four and six weeks to establish if it has been a success.Two months on from his treatment, Mr Chronis sounded upbeat."My check-ups so far have been very encouraging. I've seen my blood counts increasing on their own for the first time ever…it's remarkable," he said.The hope is that he will no longer need the regular transfusions which have restricted his life for decades, giving him the freedom to travel, live and work where he chooses."It's quite a privilege. I feel very lucky," he said."I'm very grateful that the team here in Manchester made the push to make this happen and I hope it's open to many more people".
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