A 15-year-old boy is the first to be cured through Sickle Cell gene therapy, according to a BBC report. Blood is produced in the bone marrow. The procedure involves removing the bone marrow of the patient and then genetically altering it in a laboratory to atone for the defect in the DNA that is the cause of the disease. The report says: "A virus was used to infect the bone marrow with new, correct instructions." The patient has now been free from sickle cell disease (SCD) for 15 months.
SCD is a hereditary blood disease. Medical professionals often do not have much knowledge about the extent to which a sickle cell crisis can affect a patient.
According to the Sickle Cell Society, there are approximately 15,000 people in the UK affected by SCD. 10,000 are directly living in London and yet there are only four sickle cell specialist centres in all of London.
Teach them young
During a crisis, sufferers are often at a crossroads; despite being in serious pain, the lack of knowledge on the disease within the general medical world is a deterrent. The general care- or lack there of- administered is poor. Many patients are left unseen to for hours by nurses who do not fully understand just how excruciating the pain is and patients are sometimes seen as hypochondriacs. When a crisis ensues sufferers often do not want to go to the hospital, as they would rather stay at home and hope for the best.
Jenica Leah, 27, is a self-published author of children's book, 'My Friend Jen'- the first book in a series of a beautifully illustrated book that teaches children about sickle cell. Ms. Leah, who has sickle cell anaemia herself, had this to say:
"I wanted to give children growing up with sickle cell the peace of mind that I didn't have as a child.
I think it is important hat they learn and understand their health condition from a young age. There is nothing that they cannot do, they're just a little different."
"I used to play down my condition like it wasn't a big deal. I have had almost every complication that is related to the disorder; stroke; acute chest syndrome; crisis affecting your liver; gall stones; and a hip replacement at 25.
Since sickle cell is something I live with and I am very passionate about it."
'My daughter knew from day one'
SCD can kill, and despite it being one of the most commonly inherited diseases in Britain, there is very little known about it. Unfortunately, a lot of sufferers generally feel failed by hospitals and so many take it upon themselves to not only educated themselves but also to educated their children on just how serious the disease is and how to cope with it.
Kehinde Salami, 35, father of two, only found out he had the disease in his mid 20's. He had multiple bad experiences in hospitals since finding out. As a sickle cell advocate himself, Mr. Salami took it upon himself to educate his children on the condition that their daddy has.
"My daughter, Angel, knew from day one as she has sickle cell too. From when she could speak I told her about the disease. My children know why I get tired and about my sickle cell. I had to explain to my son, Amari, why his sister gets tired as well."
He went on to say, "I explained her medication to both of them as my son had to know too, so that he knows what to do just in case she goes into a crisis. I also credit most of the advice to their mother, she was very active in getting them to understand the condition."
Conclusion
Maybe if it were a different disease then there would be no need to talk about it with children at such an early age, but when considering the fact that despite it affects thousands of people in the UK, is there possibly foul play?
SCD has been a global health risk for over 100 years and yet investment into treatment and care has been painfully slow compared to other chronic conditions. It seems, when it comes to sickle cell, you have no choice but to teach them young. So, while the therapy is a development, many with the disease are still being failed.
