Question

6. If you need to design a drug for (a) treating sickle cell anemia disease, what would your strategy be? Why? (b) What is the cause of sickle cell anemia?

Answer 1

New therapeutic strategies might be divided into pathophysiology related novel therapies and innovation in curative therapeutic options such as haemopoietic stem cell transplantation and gene therapy. Agents which reduce sickling or prevent sickle red cell dehydration. Agents targeting SCD vasculopathy and sickle cell endothelial adhesive agent and anti oxidant agents. Hydroxyurea or hydroxycarbamide (HU) is a key theraputic tool for SCD that can increase HBF production resulting in delayed HBS polymerization, increase no availability targeting cGMP production,modulates endothelial activation and reduces neutrophil counts, reduction in chronic inflammation.

Sickle cell anemia results from a mutation in a gene called HBB which contains the blueprint for cells to make part of a protein called haemoglobin. The mutation in HBB genes in sickle cell anemia changes one of the amino acids, the building blocks of protein, in the beta chain of haemoglobin. This defect causes haemoglobin protein to stick together and form stiff fibres. These fibres distort shape of the red blood cells and make them more fragile.