The project aims to improve the treatment of Myelodysplastic syndrome that currently lacks effective treatments.
Myelodysplastic syndrome (MDS) is a group of blood disorders in which the blood cells in the bone marrow do not develop normally, leading to various types of anemias such as low red red blood cell count, neutropenia (low white blood cell count) or thrombocytopenia (low platelet count). The disease is caused by genetic mutations in the blood stem cells.
Currently, there is only one curative treatment for this syndrome, Stem cell transplantation. The other treatments such as frequent blood transfusions treat the symptoms such as low red blood cell counts and aim to slow the disease progression.
Despite advances in treatment, the challenges of MDS are great, with the average survival of MDS patients only 2 years. New drugs and genetic tests are being developed to better tailor treatment to the patient's specific genetic changes, but the complex nature of the disease and the many mutations make this difficult. Clonal hematopoiesis, where cancer cells form new mutations and evade treatment can make it difficult to find effective treatments for MDS.
Further research and better diagnostic methods are needed to improve patients' treatment outcomes and quality of life.