National Research Development Corporation's associate institute the National Physical Laboratory, New Delhi has developed a novel drug, iron chelator formulation known as 'Beta -diskette type complex' for Thalassemia patients.
Chelators are small molecules. Iron chelator is used to remove excess iron that accumulates with chronic blood transfusions. The novel formulation 'Beta -diskette type complex' acts as a catalyst to help control fever and the body ache problem associated with Thalassemia patients.
The technology to develop the new formulation is now transferred to NRDC by NPL and is now open for commercialization. The novel formulation has bagged several patents including from India, the US and Germany, according to G Lakshminarayan, manager, National Research Development Corporation.
The formulation is soluble in body fluids and can be excreted from the body of patients through urine, thus reducing pathological iron overload in the organs, he added.
Current treatment modality for Thalassemia is regular blood transfusion with the administration of the drug 'Desferal' injection. The drug is expensive and medical scientists have been looking for an alternative option which is an affordable oral drug administered without side effect. NPL has developed this and now we are looking for upscale of the research, stated Lakshminarayan.
There are no side effects reported with the new drug. The cost is also negligible and can be easily consumed. The existing drugs are injected daily under the skin of the patients in the controlled manner to avoid side effect causing painful allergic conditions.
Thalassemia is a dreaded disease among children because of genetic disorders. It is caused by defective haemoglobin synthesis, characterised by hypochromia, microcytosis, haemolysis and a variable degree of anaemia. The disease involves a heterogeneous group of molecular defects and is present with a wide spectrum of clinical expressions.
Patients suffering from Thalassemia suffer anaemia with decreased or absence of synthesis of a globulin chain of a normal haemoglobin. Two types of Thalassemia affect the globulin chain. The patients suffering from Alpha Thalassemia are associated with decreased or absence of Alpha-chain synthesis. The patients suffering from Beta Thalassemia or Thalassemia major are associated with decreased or absence of Beta -chain synthesis.
The Beta -Thalassemia disorder usually is characterised by absence of beta or decreased beta plus globulin synthesis. This type of Thalassemia is also classified according to the severity of the anaemia. These clinical classification serves to differentiate homozygous Thalassemia intermedia or Thalassemia major from heterozygous state: Thalassemia minima or Thalassemia minor. Though it does not reflect genetic mutations, Thalassemia minor is a reduced rate of Beta-globulin synthesis, with an increased Alpha-Beta globulin chains, but is not threat to a normal life, he said.
In the case of Thalassemia major cases, the iron in the haemoglobin breaks down and gets deposited in the vital organs of the body of the patients like liver, kidney and spleen and make them rigid. This is also known as iron overloading in the body. The life span of the child suffering from Thalassemia major becomes unpredictable.
Globally, Thalassemia occurs with a particular high incidence in the Mediterranean basin and in the South East Asia.