Earlier this month, doctors announced the results of an experimental gene therapy that is offering hope to those who suffer from the disease.
A French teenager with the genetic condition achieved complete clinical remission after researchers in Paris injected his stem cells with an anti-sickling gene from his bone marrow.
So far, it seems to be working for the teen. Fifteen months since his last treatment, the patient is no longer dependent on medication, and his blood cells show no sign of the disease.
Clinical studies have begun in other parts of the world to address the disease. However the therapy is described as an incredible curative potential but not a cure yet.
The Lagos state president of Sickle Cell Aid Foundation (SCAF), Bukky Bolarinwa in a discussion with foreign doctors on Al Jazeera’s ‘The Stream,’ talks about a recent ‘breakthrough cure’ found for Sickle Cell Anemia.
What we do in gene therapy is that we actually use the patient with SC diseases own bone marrow and we put in a new gene into their marrow that makes healthy hemoglobin. Hemoglobin is the protein in the red cell that carries oxygen.
And so if we can put a new healthy hemoglobin that’s not sickle, the blood cells won’t be sickle, then we have to give the bone marrow that we’ve now doctored back to the patient and to do that, we have to do an Autologous Bone Marrow Transplant.
In English that means we have to do chemotherapy and then let the new bone marrow get infused so it swims back to where it’s supposed to be into the blood stream, into the bone marrow, to make a new healthy cells.
It’s a long process and it has great potentials and we might be there but so far we only have this one individual who has really got curative potential with this and we’re really excited, we are doing more work but I wouldn’t quite say it’s there yet.
However we’ve come so far from the old gene therapy studies and we’ve made incredible headway and this is very applicable to other diseases as well.