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Remmy Kamya, 23, is not just free from the crippling bouts of pain that have affected him for years - he is cured.
Remmy has become the first British adult to undergo a pioneering stem cell treatment for sickle cell disease.
The success of the procedure gives hope to thousands of adults who may otherwise face a future filled with pain and a premature death.
Sickle cell disease is a genetic blood condition affecting 13,500 in the UK. Red blood cells are produced by stem cells within the marrow found inside the bones.
Healthy red blood cells are biconcaved discs which can bend and flex easily. In those with sickle cell disease, faulty stem cells produce red blood cells that are crescent shaped.
These are rigid, unable to squeeze through smaller blood vessels, and prone to causing blockages that deprive parts of the body of oxygen, leading to periods of intense pain.
Symptoms may be mild or severe, depending on how many sickle cells the body produces, and can include exhaustion and susceptibility to infection.
Crises can last for days and internal organs may gradually become damaged from restricted blood flow. It is the most common cause of stroke in children. Sufferers are not expected to live beyond 60.
Progress: The success of the procedure gives hope to thousands of adults who may otherwise face a future filled with pain and a premature death (picture posed by model)
Treatment focuses on alleviating symptoms by using painkillers, administering oxygen, drinking lots of water and prescribing antibiotics.
The only cure until now has been a bone marrow transplant. During this, extremely high doses of chemotherapy are given to kill the unhealthy stem cells within the existing bone marrow, before it is replaced with donor marrow.
The procedure carries a 20 per cent risk of fatality and could be offered to sufferers only under the age of 18 as the internal organs of adult patients are typically too damaged by the disease to withstand chemotherapy.
Life-saver: Stem cells are separated from a blood sample
'Stem cell transplants have been offered only to paediatric patients whose organs are still healthy,' says Dr Mickey Koh, consultant haematologist and director of the bone marrow transplant unit at St George’s Hospital, London.
'When considering transplants we had to weigh up the severity of symptoms and the risk to life. Chemotherapy kills the faulty stem cells but harms organs.'
The new procedure uses low dosage radiotherapy that targets only the bone marrow, so it avoids further damage to the liver, kidney, lungs or heart. Special drugs that suppress specific parts of the immune system are used to make sure the donor tissue is not rejected.
Remmy was diagnosed with sickle cell disease at the age of two. ‘I used to have attacks twice a month, lasting for days,’ he says.
The technique was pioneered in America two years ago with ten adult patients, all of whom survived and were cured. There is still a risk of fatality, but Dr Koh says it is less than eight per cent.
Having mainly suffered in his legs, feet and arms, seven years ago he began to get pains in his chest, considered more serious as they indicate lung damage.
‘I also had a brain scan which revealed I had suffered a stroke in the past,’ he says.
‘Doctors were worried this could happen again and the next time it might cause more damage.’
Remmy was referred to Dr Koh who tested his family for donor suitability and brother Ronnie, 33, was a good match.
Remmy was admitted into hospital in February and given daily injections of antibodies for five days followed by anti-rejection drugs to prepare for the transplant. Ronnie was given an injection to stimulate the production of more stem cells that would enrich his blood.
Stem cells are transplanted by blood transfusion – they travel through the arteries and end up in bone marrow.
Within days Remmy’s blood was being populated with healthy new red cells and after a week he was allowed home. Next February he can stop taking anti-rejection drugs.
‘Thousands of adults can now be treated, saving more lives,’ says Dr Koh.
www.aclt.org
Read more:
http://www.dailymail.co.uk/health/article-2046817/Pioneering-stem-cell-treatment-saves-life-sickle-cell-victim.html#ixzz1aENjoT28