Life-changing treatment offered to children with blood disorders
Meet JoelSamuel and Vian – one is at the start of pioneering curative treatment for a blood disorder, whilst the other is pursuing her dream of becoming a nurse after completing the therapy.
Vian, aged 16, was born with thalassemia, leaving her tired, short of breath and with no appetite, needing monthly blood transfusions.
The teenager, from Coventry, would regularly miss school due to the illness which meant she lacked haemoglobin, leading to a reduction in the flow of oxygen around her body.
The transfusions caused severe side effects. Many patients wait years for a bone marrow transplant to help cure symptoms of condition.
However, the groundbreaking gene therapy received at Birmingham Children’s Hospital edited her DNA and reactivated a gene that produces haemoglobin in her body – meaning she no longer has the symptoms or needs blood transfusions.
Following the treatment early this year, Vian was able to study for her GCSEs and qualify for college where she is studying health and social care so she can become a nurse specialising in thalassemia.
“I have grown up going to hospital, surrounded by nurses and they have inspired me to join them,” beamed Vian.
“Now thanks to this therapy I am much better and have the energy to study and go onto college so I can be a nurse.”
Birmingham Children’s Hospital is one of three centres in the country providing this life-changing treatment.
Vian’s mum, Ahlam, added: “I am so pleased with this outcome. Vian has no symptoms and she can now continue with her life without suffering.
“We would take her to hospital on a regular basis and this would mean missing school. Whenever she had a childhood vaccination, she would be ill for weeks on end suffering from fever. But now this has gone.
“She has gone through so much over the years.”
Meanwhile sickle cell disease sufferer JoelSamuel is embarking on the specialist treatment which he and his family hope will rid him of excruciating pain that affects his body.
The disease is an inherited condition that affects red blood cells, causing them to become hard and curved instead of soft and round. These cells can get stuck in blood vessels, causing severe pain, and break down more quickly than normal, leaving people feeling tired and short of breath
JoelSamuel undergoes regular blood transfusions which leave him so tired he's unable to go to school for long periods of time.
His mum Juliette, from Oldbury, said: "This is why we believe gene therapy could be life-changing for him. Our hope and prayer is that the treatment will free him from these constant crises and give him the quality of life every child deserves. We want to see Joel regain his strength, attend school regularly, play football, participate in PE, walk without severe pain and enjoy his childhood without always living in fear of the next crisis."
Explaining the symptoms he suffers, Juliette added: "He will experience puffiness around his eyes, skin rashes, severe pain and swelling in his fingers and feet, swollen thighs and intense pain in his legs that can leave him unable to walk. “He has also experienced acute chest syndrome, sometimes referred to as a chest crisis, which affects his breathing and can become very serious.
“He suffers severe headaches and many other complications.”
Dr Sarah Lawson, Consultant Paediatric Haematologist and Clinical Lead for Blood and Stem Cell Transplant at Birmingham Children’s Hospital, said: “We are delighted we have been selected as an Authorised Treatment Centre for the gene editing to be able to give patients like Vian a curative treatment that is more accessible than a transplant - the current option.
“Vian is an extremely bright, intelligent young lady and with this treatment available to her she will be able to do so much more, having a life free of blood transfusions and fewer hospital visits.
“It’s lovely to see her thriving and going onto college to study to become a nurse. And we are very happy to offer JoelSamuel this treatment.”
Currently, the treatment has a strict eligibility criterion and is only available to young people aged 12 and over.
Vian, who is the second paediatric patient nationally to ever have this therapy spent eight days with Ahlam, by her side as she received the first stage of the treatment as an inpatient.
It took six months for the collected cells to be processed and Vian also returned to hospital during this time to have chemotherapy to prepare her body for the edited genes being put back in her body.
As part of the final stage of her treatment, the edited cells were injected back into her body.
Days later, Vian was able to ring the end of treatment bell (pictured above) to mark the significant moment.
Ahlam added: “For years, this has been my dream to see my daughter get this treatment. I am looking forward to the life she can have now and in the future.
“I am very proud of my daughter. She is so strong. I hope sharing her experience will help many others, including JoelSamuel.”