Shocking it may sound, but the patients including kids suffering from sickle cell anaemia (SCA), a genetic blood disorder in the tribal areas of the country are being treated with the drug—Hydroxy urea—which is not even approved by the Drug Controller General of India (DCGI).
The Union Health Ministry has, for decades, been allowing use of the drug for pain management among the SCA patients maintaining that there are not much side effects. However, Health Ministry's views have not gone down well with the Council of Scientific and Industrial Research (CSIR), a research body under the Union Science and Technology.
Putting a question mark on the efficacy of the drug, the CSIR scientists have alleged that "Hydroxy Urea treatment in itself has several side effects including increased risk for developing infections and tumor formation. Further this drug does not show any efficacy in one third of the patients. Therefore, new therapies need to be developed for effective management of SCA with fewer side effects," they said.
The SCA is a debilitating disease transmitted from parents to children. It is characterised by abnormal red blood cells that stick together in patients' blood vessels, blocking the blood flow to organs, which can lead to severe pain, organ failure, stroke, and even death. At present there is no effective and safe treatment for the ailment that hits mostly tribals and people belonging to poor section of society.
Every year approximately 5, 00,000 children are born with SCA worldwide with India accounting for nearly 50 per cent of the cases.
Sources in the CSIR argued that the Hydroxy Urea is only USFDA approved drug for SCA and not adequate for Indian settings.
Now, to facilitate the process of getting the DCGI approval for the Hydroxy Urea for treatment of the SCA as also for its diagnosis, prevention and management, the CSIR has launched a systematic, first of its kind efforts in the form of Sickle Cell Anemia Mission.
The Mission envisages conducting phase I/II clinical trials of a herbal formulation to be developed based on the plants (or their substitute plants in India) used in the preparation of Niprisan, a herbal drug extracted from indigenous herbs that has been developed by the Nigerian National Institute for Pharmaceutical Research and Development for the prophylactic management of the SCA patients.
The mission also envisages, screening of population and develop counselling protocol for effective management of the disease to ensure quality life of the patients.
Though the exact reason is not known, the sickle cell gene is mainly concentrated in scheduled tribal, scheduled caste and other backward caste populations of Madhya Pradesh, Orissa, Chhattisgarh, Jharkhand, Gujarat, Andhra Pradesh, where carrier frequencies range between 5-40 per cent or more.
Individuals suffering from sickle cell disease show symptoms such as body pain, clotting, dyspnea, anaemia, jaundice, pneumonia, repeated infection etc. Their lifespan is usually reduced to 42-48 years with 50 per cent of kids with SCA dying before the age of 5.
The CSIR scientists also plan discovery and development of new lead molecules for management of SCA and thus, improve the quality of life with better life expectancy. This will involve screening pure natural/ synthetic/ semisynthetic compounds and botanicals against various validated sickle cell anaemia targets.
The project will also focus on social, clinical, pre-pregnancy, prenatal-test counselling and creation of genetic database for SCA population ie patients, carriers and their extended families.