Sickle cell disease has been a challenge for decade now especially recently that medical expert advised couple to know their blood group before getting married. The reason given was to control the increase of sickle cell anaemia population. Another reason given is the suffering that some of the sickle cell anaemia patient always pass through and even the challenges that the family members do pass through in taking care of them.
The term sickle cell disease (SCD) is describes as a group of inherited red blood cell disorders. People with SCD have abnormal haemoglobin, called haemoglobin S or sickle haemoglobin, in their red blood cells.
Haemoglobin is a protein in red blood cells that carries oxygen throughout the body.
“Inherited” means that the disease is passed by genes from parents to their children. SCD is not contagious. A person cannot catch it, like a cold or infection, from someone else.
People who have SCD inherit two abnormal haemoglobin genes, one from each parent. In all forms of SCD, at least one of the two abnormal genes causes a person’s body to make haemoglobin S. When a person has two haemoglobin S genes, Haemoglobin SS, the disease is called sickle cell anaemia. This is the most common and often most severe kind of SCD.
Haemoglobin SC disease and haemoglobin Sβ thalassemia (thal-uh-SEE-me-uh) are two other common forms of SCD.
The deformed red blood cell that is haemoglobin cells can lock together as a result block the flow of blood around the body. This can cause serious pain, organ damage and in some occasion it can be fatal.
Scientists have tried all they could do in the past to find a cure to this disease but to no avail. There is good news from Doctors at the Necker Children’s Hospital in Paris that they now have cure for the disease.
The world first operation was carried out by a group of scientists at Necker Children’s Hospital in Paris offers to people suffering from sickle cell disease.
The operation was carried out on a 15 months child by altering the genetic instructions in his bone morrow so it can make healthy red blood cells and the child is no longer on medication.
The teenager who received the treatment had serious internal damage he needed to have his spleen removed and his hips replaced.
He has to have his blood replaced every month to dilute the defective blood but when he was 13, doctors at the Necker Children’s Hospital in Paris did something different.
They had his bone marrow removed, the part that produces blood. It was genetically altered by the doctors in a lab to compensate for the defect in his DNA that caused the disease.
A virus was introduced into his bone marrow to infect it with new and correct instructions. The corrected bone marrow was then put back into the patient.
The result showed that the teenager has been making normal blood since the procedure was carried out on him since 15 month ago as shown in the New England Journal of medicine.
Prof Leboulch said thus; she is nervous about using the word “cure” as this just the first patient to come through clinical trials.
She further said that study shows the potential power of gene therapy to transform the lives of people with sickle cell disease.
Her word from the University of Oxford thus; “i think it’s very significant, essential they’ve given him his life back”
According to what she told BBC “I’ve worked in gene therapy for a long time and we make small steps and know there’s years of more work”.
“But here you have someone who has received gene therapy and has complete clinical remission- that is huge step forward”.
“However, the expensive procedure can only be carried out in cutting-edge hospitals and laboratories, while most sickle cell patients are in Africa.”
How to transform this pioneering science into something that really can help millions of people is the challenge.
The people that are most affected by sickle cell disease are Africa, Caribbean, Middle Eastern, Eastern Mediterranean and Asian origin.