Sickle cell anemia (SCA) is a common genetic disorder affecting red blood cells in the Indian population. The disease is passed on to children with the faulty beta globin gene from their parents, although the parents themselves do not get the disease. About 0.4% of the population suffers from the disease, while 10% are carriers of the disease, giving rise to new SCA patients.
Like most genetic disorders, there is no cure for SCA. But symptomatic treatments exist for problems such as pain, anemia, and severe vaso-occlusive disease. One of the relatively inexpensive drugs, hydroxyurea, which is used extensively as an anticancer agent, is also used in SCA treatment without any formal approval. However, now the use of hydroxyurea to treat sickle cell anemia has received the approval of the Controller General of Drugs.
This approval opens the way for legalizing the use of standard doses of the drug for the treatment of SCA. This would pave the way for designing different formulations of smaller doses, which could lead to better drug efficacy and adaptation rates in children with SCA. Researchers say that in the future, it may also develop into a syrup-based formulation.
Dr. Giriraj Ratan Chandak, Chief Scientist, CSIR-CCMB and Mission Director, CSIR-SCA Mission, says, “The Drugs Controller General’s approval for the use of hydroxyurea for the treatment of sickle cell anemia is significant. This is through a targeted screening programme. This will help in better identification of patients with the disease.While a major objective of the screening program is to prevent the birth of children with such disease through genetic and social counseling, it provides comprehensive treatment to the identified patients. Now there is a need to reach out to doctors across the country so that they can regularly use Hydroxyurea for their patients
The disease has been observed prominently in the tribal population as well as in the general population of states like Maharashtra, Madhya Pradesh, Chhattisgarh and Orissa. SCA begins early in childhood. In children affected by this, conditions such as deficiency of hemoglobin (anemia), lack of strength in the body, stunted growth and other abnormalities and persistent pain are seen, which are known as vaso-occlusive disease.
Commercially available hydroxyurea formulations are designed with anti-cancer role in mind. Therefore, they are usually larger in size (minimum 500 mg). At the same time, children with SCA are usually underweight, and keeping this in mind, their dosage should be relatively low.
Currently, due to the large size of commercially available hydroxyurea capsules, it is difficult to give the correct dosage to SCA patients. Although the results of hydroxyurea therapy are highly effective, favorable results are relatively rare due to the lack of dosage and the complications involved. Sometimes unexpected results are also seen.
Under the CSIR-Sickle Cell Anemia (CSIR-SCA) mission, scientists and physicians with six CSIR laboratories and three government hospitals in Chhattisgarh, Madhya Pradesh and Maharashtra are working together to address various gaps in SCA diagnosis and disease management. Huh. The mission focuses on identifying patients through population-based screening in high-prevalence states and helping families find appropriate treatment and prevent the disease in the next generation.
The CSIR-SCA mission is being coordinated by the CSIR-Centre for Cellular and Molecular Biology (CSIR-CCMB), in collaboration with Cipla, one of the manufacturers of Hydroxyurea and with the active support of CSIR-IIIM. The Drugs Controller General of India was urged to approve the use of Hydroxyurea in SCA treatment. The approval has been given after a thorough evaluation of the proposal and approved marketing of hydroxyurea for the treatment of SCA by a committee of experts constituted by the Central Drugs Standard Control Organization (CDSCO).
