
NIH Seeks 'Self' Cure in Patients With Sickle Cell Disease
The new initiative will not focus on donor-related bone marrow transplants in sickle cell disease, but instead will home in on genetic modifications of a patient’s own stem cells.
Earlier this month, the National Institutes of Health (NIH) announced the beginning of a new initiative to find a cure for sickle cell disease (SCD). This development will not focus on donor-related bone marrow transplants and instead home in on genetic modifications of a patient’s own stem cells.
An inherited blood disease, SCD affects
The only cure for SCD remains a bone marrow transplant, a process where a patient receives bone marrow from a healthy, genetically compatible donor. However, transplants are dangerous because it can lead to serious complications such as graft-versus-host disease (GVHD), veno-occlusive disease, infections, and graft failure. When a patient with SCD receives the bone marrow of another person, there is the risk that the immune system of the patient with sickle cell rejects and attacks the donor cells. The chances of either instance even in matched-related transplants can be up to 10%. Veno-occlusive disease can also occur if the blood vessels near the liver become damaged.
Each year, the NIH spends about $100 million on SCD research. For this year, the NIH already committed an extra $7 million to start off the initiative. Current research focuses on using a SCD patient’s own hematopoietic cells and genetically modifying them before being returned to the patient through a bone marrow transplant, thus eliminating the need to find a matching donor, and removing the risk of GVHD.
“Our scientific investments have brought us to a point where we have many tools available to correct or compensate for the defective gene that causes SCD. We are now ready to use these tools to speed up our quest for a cure,”
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