Tay-Sachs Children Respond to Ground-Breaking Stem Cell Gene Therapy
As a result of Dr. Feinerman’s continued ground-breaking efforts to treat Tay-Sachs disease through stem cell gene therapy, the treatment of three children suffering from this devastating genetic disorder have recovered. Children with Tay-Sachs appear to develop normally after birth, but symptoms begin to affect them around 6 months of age. They lose the ability to turn over, raise their heads, sit or crawl. They make little or no movement and may have trouble even swallowing. Many have frequent seizures and lose the ability to hear or see. The disease usually results in death by age four.
Until now there has been no curative treatment for children suffering from Tay-Sachs. The gene therapy treatment provided by StemCellGeneticMed, however, will offer thousands of families a chance to help their children who suffer from this catastrophic disease.
Burton Feinerman, M.D., who trained at the Mayo Clinic and now practices in Sarasota, Florida, studied the natural pathology of Tay-Sachs, which is caused by a genetic mutation on the HEXA gene and affects production of the enzyme beta-hexosaminidase A. This enzyme assists in breaking down fat deposits in brain cells. When deficient, an abnormal accumulation of these fat deposits will occur in the brain and spinal cord.
Dr. Feinerman first developed methods to increase enzyme activity, empty substrates that formed in cells, and correct non-functioning misfolded proteins.
Dr. Feinerman teamed up with longtime colleague, Javier Paino, M.D., Ph.D., a neurosurgeon and scientist who trained at Mt. Sinai Hospital Medical School in NY and George Washington University School of Medicine. Dr. Feinerman and Dr. Paino administered treatment to Aspen Brown, a 14-month-old child from Alabama suffering from Tay-Sachs. Aspen was the first child to receive the treatment. Within days of the treatment she exhibited improvement in muscle tone, the ability to move her head and neck, follow movement, and grasp objects.
Dr. Feinerman and Dr. Paino took their research one step further by inserting the HEXA gene into neuron stem cells without using a viral vector. The neuron stem cells with the HEXA gene were administered into the central nervous system and brain. The gene treatment was provided to three children at the Concebir Clinic in Lima, Peru. Although Dr. Feinerman and Dr. Paino were not expecting any miracles from the treatment, they were greeted by the overjoyed parents of the children at the hospital just two days later. The children were making sounds, showing active facial expressions, moving their arms and legs, following moving objects, making head and neck movements, showing increased muscle tone, swallowing more and exhibiting a decrease in drooling. Two of the children had been suffering from multiple seizures a day, but those seizures stopped shortly after the treatment.
Tay Sachs Recovery
StemCellGeneticMed focuses on stem cell and gene treatments for serious debilitating diseases such as Tay-Sachs and other genetic diseases,
Huntington’s, ALS, Alzheimer’s, multiple sclerosis, Parkinson’s, brain damage, autism, heart disease, COPD, diabetes and chronic kidney
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