Sickle Cell Disease
Sickle cell disease (SCD) comprises a group of genetic disorders characterised by the presence of haemoglobin S (Hb S).
SCD is characterised by a modification in the shape of the red blood cell from a smooth, donut-shape into a crescent or half moon shape. The misshapen cells lack plasticity and can block small blood vessels, impairing blood flow. This condition leads to shortened red blood cell survival, and subsequent anaemia. Poor blood oxygen levels and blood vessel blockages in people with sickle-cell disease can lead to chronic acute pain syndromes, severe bacterial infections, and tissue death.
Life expectancy is shortened, but most people with SCD can now live into their 30s and 40s; with good medical treatment, many live much longer; however the longer the people live the greater the risk of organ damage.
It is estimated that SCD affects 90,000 Americans. In Europe, a high prevalence of the disease has been observed in France. This is a result of population growth in overseas French departements, and immigration essentially from North and sub-Saharan Africa to mainland France. In the United Kingdom one baby in every 2,000 is born with SCD.
TriStem’s clinical study
TriStem is currently planning a clinical study in Gulf Cooperation Council (GCC) countries which have a high prevalence of the disease. Dr Lanetta Bronté is advising on the design of the study.
In light of the experience obtained in the Beta Thalassemia trial (also a genetic disease), this study will be conducted with allogeneic-derived stem cells.