A new molecule capable of emancipating patients suffering from myelodysplastic syndromes and beta-thalassemia from the constant need for blood transfusions.
The new molecule against anemia.
Luspatercept has been shown to reduce the number of transfusions required for patients with myelodysplastic syndromes and beta-thalassemia with a significant impact on the clinical course and quality of life.
The emerging therapeutic revolution coincides with luspatercept, produced by Celgene now part of Bristol Myers Squibb, and already approved by the European Medicines Agency (EMA) in Europe based on two studies published in the New England Journal of Medicine. The drug, available quickly, has been shown to reduce the number of transfusions required, with a significant impact on the clinical course and quality of life.
Also because severe anemia, often present both in myelodysplastic syndromes and in beta-thalassemia, forces patients on the one hand to frequent sessions in hospital due to the need for red blood cells, and on the other to take daily iron chelation therapy to avoid that excess iron can damage organs such as the heart, liver and pancreas.
Myelodysplastic syndromes and beta-thalassemia
To better understand the effects of luspatercept it is necessary to first explain what is meant by myelodysplastic syndromes and beta-thalassemia. The myelodysplastic syndromes are a heterogeneous group of blood cancers, in which the bone marrow cells fail to become fully functional and healthy cells but they stop at an immature stage.
They are defined as clonal diseases because their development is due to a single cell that, escaping the control mechanisms, multiplies, giving rise to cells altered in shape and function. Symptoms and course vary significantly depending on the type of affected blood cells.
In severe cases, these syndromes can develop into acute myeloid leukemia. What is certain is that their lowest common denominator is to cause bone marrow failure in affected patients, which in turn prevents the cells in the bone from reaching full maturation. Myelodysplastic syndromes mostly affect elderly people, with an average age of 74. It is estimated that 4-5 people are affected per 100,000 inhabitants.
Even the beta-thalassemia forces patients to undergo blood transfusions every 2-3 weeks for a lifetime and take a daily iron chelation therapy to prevent the damage caused by the accumulation of iron in vital organs.
The role of the luspatercept molecule
This is where the role of luspatercept comes into play, a molecule that has the potential to change the history of the two aforementioned diseases, reducing the need for continuous transfusions. It consists of a subcutaneous injectable drug every 21 days, which the patient will be able to perform at home in the future.
Luspatercept reduces ineffective erythropoiesis – the main cause of the clinical manifestations of thalassemia – and enables the production of mature red blood cells. It can also be potentially administered to all patients affected by beta-thalassemia, unlike other options available such as bone marrow transplant, the only therapy that can lead to recovery but with the limit of availability of a compatible donor, or gene therapy. still to be consolidated.
Even in myelodysplastic syndromes, the only definitive therapy is stem cell transplantation, which can only be practiced in a limited number of cases and in younger patients, under 70 years old. In the lower-risk forms, the cell line most affected is red blood cells, with severe anemia causing debilitating symptoms.
Therapy in these cases consists of red blood cell growth factors such as erythropoietin, blood transfusions and iron chelation therapy. Immunosuppressive therapy is indicated to lower the immune response only in some young patients with particular characteristics of the disease and in good general conditions. Luspatercept instead promises to open new perspectives, with the intention of improving the quality of life of patients.