Stem Cell Gene Therapy For Tay Sachs disease!
A couple of scientists have succeeded in treating three children suffering from Tay Sachs disease using stem cell gene therapy provided by a company named StemCellRegenMed.
Tay Sachs disease affected children though appear normal at birth, develop a set of symptoms that include loss of muscle strength, decreased eye contact, deafness, seizures, loss of muscle function or paralysis, inability to turn over, raise the head, crawl, sit and swallow. Most of the affected children do not live to celebrate their 5th birthday. No treatment is available for this terrible condition till date.
Tay Sachs disease is the result of a mutation on HEXA gene. This mutation affects the activity of an enzyme named beta-hexosaminidase A, which is required to break down the accumulating fat deposits in the brain. Due to this enzyme deficiency, fat piles up in the brain and spinal cord of affected individuals.
Dr. Burton Feinerman was the first person to come up with techniques that help in increasing the activity of the enzyme and correct the misfolded, non-functioning proteins.
A 14 month old girl named Aspen Brown was the first patient to receive the treatment from Dr. Feinerman and his colleague Dr. Javier Paino in 2010 November. Within a few days after the treatment, the girl showed improvement in neck and head movement and she was able to grasp objects.
Recently, a few days back, these two doctors inserted the HEXA gene into neural stem cells without a viral vector. These stem cells were then injected into the brain and nervous system of three children suffering from Tay Sachs disease.
Miraculously, just within a week’s time, all three of them showed drastic improvement in performing activities that were not possible before the treatment. They could move their legs, arms, neck and head. They could also show facial expressions, swallow efficiently and make sounds.
Two children among three were facing multiple seizures in a day. These completely stopped soon after the treatment.
The doctors are planning to conduct this treatment trial again in the next three months. If they succeed in it, children enduring the affects of Tay Sachs disease will find a solution.