In a new find, scientists have experimentally developed a new drug that might solve the problem of fatal neurodegenerative disease among new born babies. Although the spinal muscular atrophy or SMA is a rare disease but it is the most common genetic cause of death in childhood which affects the brainstem and the motor neurons of the spinal cord.
Over 11,00 babies die of SMA every year. The disease affects nerve cells present in the spinal cord which results in progressive muscle weakness and problems related to eating and breathing.
Now, scientists have developed a drug named nusinersen which can cure the SMA in child aged between two years to 12 years. Researchers have conducted the clinical trials of the drugs and believe that nusinersen can cure affected cells in the brain and spinal cord. It is to be noticed that the clinical trials in August and on November 7 were stopped on the grounds that it was unethical to deny the drug to children in the control arm. However, scientists are seeking approval from Food and Drug Administration (FDA) as no drug is present in the market to treat the disease.
“These [infant-onset] SMA kids are going to die. And not only are they now not dying, you are essentially on the path to a true cure of a degenerative [neurological] disease, which is unheard of,” says Jeffrey Rothstein, a neurologist at the Johns Hopkins School of Medicine in Baltimore, Maryland.
Scientists explained that the survival motor neuron (SMN) is produced by a defect in the gene called SMN2 which also leads to one of the most severe form of genetic disease called infantile-onset. Past records suggest that the disease affect babies under the age of six months and they live up to two years without much breathing and feeding support.
Clinical trials suggest that the use of drug nusineren can increase production of SMN protein and improve motor functioning of cells. Study authors said that the drug is safe to use for a baby as small as five months. The drug enables children to sit, roll over, stand, and live without using a ventilator.
Richard S. Finkel, lead author from Nemours Children’s Hospital in Florida, US said “With nusinersen, these infants are not only living longer, but living better”.
Finkel added, “SMA is no longer a death sentence for infants. This treatment is by no means a cure, but it is more than we’ve ever been able to offer these families before”.
The study appeared in the Lancet.