Whit staff and expert patient help to advocate for life-saving gene therapy

16 Aug 2024
The first therapy that uses gene-editing was approved last Thursday 8 August to be offered to patients who live with transfusion dependent beta thalassaemia (TDT).
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The first therapy that uses gene-editing was approved last Thursday 8 August to be offered to patients who live with transfusion dependent beta thalassaemia (TDT).
Whittington Health NHS Trust’s Dr Emma Drasar (Consultant Haematologist and North Central London’s Haemoglobinopathy Network Lead), Dr Clare Samuelson (Consultant Haematologist and Clinical Lead, North East & Yorkshire Haemoglobinopathy Coordinating Centre), Roanna Maharaj, (Whittington Health patient living with transfusion dependent beta thalassaemia), and Executive Director of the UK Thalassaemia Society, Romaine Maharaj were instrumental in advocating and providing submissions for this therapy on behalf of the TDT community.
After 5 years of lobbying, Dr, Drasar, Dr Samuelson and Roanna Maharaj presented evidence at an approval panel, explaining the impact the treatment could have on those who live with the disorder.
During the 5 hour hearing, they spoke on topics like disparities in health outcomes between the various ethnic communities affected by TDT, how debilitating and challenging living with TDT is for individuals and their families, and the reduced quality of life and life expectancy people with TDT had when compared to the general public.
People with TDT do not produce enough haemoglobin, which is needed to carry oxygen around the body. The disorder requires lifelong treatment of blood transfusions and the symptoms – such as anaemia and chronic pain – can greatly impact on quality of life.
In international clinical trials, 93% of patients who underwent the therapy did not need a blood transfusion for at least a year. This included several Whittington Health patients, who took part in trials and are now showing signs of being cured of the condition.
One patient at the Whittington, Abdul Qadeer Akhtar, described the gene-therapy treatment as ‘challenging’ but ‘worth it’ noting that he no longer had to plan activities around his health.
Whittington Health NHS Trust has a long history of providing care for people with thalassaemia, and treats the largest number of people with thalassaemia in the country.
In line with the Trust’s vision to help local people live longer healthier lives, the team supports patients through a multidisciplinary approach, in both hospital and the community. They work in collaboration with many other local hospitals, as well as the new North Central London Red Cell Service, which is a service that aims to address the physical, emotional, and social needs of patients and families affected by red cell disorders like thalassemia.
Dr Ryan Mullally, Consultant Haematologist at our organisation said, “This groundbreaking new treatment will transform the lives of people who live with TDT. We’re so pleased that some of our patients at the Whit are already seeing results, and can look forward to being able to offer this as a treatment option to more patients.”
Romaine Maharaj, UK Thalassaemia Society Executive Director said, "This is yet another victory in our ongoing battle to secure treatment options for all individuals living with thalassaemia, enabling them to live fuller, healthier, and happier lives."
Abdul-Qadeer Akhtar, 28, a patient at Whittington Health NHS Trust, received the new gene therapy as part of a clinical trial in 2020. He said: “My overall health and quality of life have significantly improved. I’m gaining more from my workouts and have even taken up boxing. I can travel more freely now, which is fantastic – I’m eager to embrace life to the fullest.
“Since the transfusion, things have been excellent. The biggest change is being healthier and more active. My only daily requirement is taking penicillin, but I am now independent and no longer need blood transfusions or have to plan my activities around my health.
“The treatment itself was challenging – the preparatory chemotherapy was tough, and I had a reaction after receiving the new cells, leading to a stay in the ICU. The recovery process included struggles like climbing stairs, but it was all worth it, because with hardship comes ease.”