Gene therapy has been applied in Bangladesh for the first time to treat the chronic neurological disease Spinal Muscular Atrophy (SMA).
The National Institute of Neurosciences & Hospital (NINS) has applied the therapy to a child to treat this congenital disease.
This gene therapy, curable and USFDA (United States Food and Drug Administration) approved, costs around Tk22 crore for a single dose and has been provided free of charge by the multinational pharmaceutical company Novartis Bangladesh, said a statement on Tuesday.
Spinal Muscular Atrophy (SMA) is a rare and complex congenital disease of the nervous system caused by genetic disorder.
The muscles of children suffering from this disease continue to weaken. As a result, the children cannot sit or stand. But they possess normal intelligence. Later, the affected children die due to respiratory complications.
Every year, many children die in the world because of the lack of treatment for this disease. This disease usually destroys the nerve cells of the spinal cord of children.
As the nerve cells that control the muscles are damaged, the muscles become weak. Most people in Bangladesh are not aware of this disease. The newborns suffering from this disease become very weak and gradually suffer from congenital heart disease.
A Bangladeshi child has been selected for this treatment under the GMAP programme which is the Global Managed Access Program. This programme is designed to provide a feasible pathway for patients. Under this, patients who fulfill the required eligibility are selected. Treatment is then completed in accordance with applicable local laws and regulations.
As the drug is prepared by taking into account each patient's genetic characteristics and various other physical factors, which is a very complicated process, the treatment is very expensive.
So far, this medicine has been administered to 2300 patients worldwide, according to Novartis.
“Novartis has been ensuring not only the treatment for the patients, but also making sure that the doctors of this country can use this gene therapy for their patients through proper training. Novartis has played an instrumental role in taking the healthcare sector to a new height by creating an opportunity for the doctors of the country to get a direct experience of applying the latest gene therapy treatment,” read the statement.