Ground-Breaking Gene Therapy Helps Children Suffering from Tay-Sachs Disease

SARASOTA, FL, June 03, 2011 (Press-News.org) StemCellRegenMed continues its commitment to providing treatment for Tay-Sachs disease by administering ground breaking gene therapy to three children who suffer from the disease. The gene therapy treatment provided by StemCellRegenMed may offer hundreds of families a chance to help their children who suffer from this devastating genetic disease.

A child suffering from Tay-Sachs disease often follows what appears to be a normal developmental path after birth. But symptoms of the disease begin to appear around 6-8 months of age, when the child will no longer be able to sit, crawl, turn himself over or even raise his head. Some will lose vision or hearing and suffer from seizures. By age four, many Tay-Sachs sufferers will die from the disease. Until the treatments provided by StemCellRegenMed, there have been no curative therapies or treatments for these children.

Tay-Sachs is caused by a genetic mutation of the HEXA gene. That mutation impacts the production of the beta-hexosaminidase A enzyme, an enzyme that helps break down deposits of fat in the cells of the brain. Without an adequate amount of beta-hexosaminidase A, deposits of fat can accumulate in the spinal cord and brain. Burton Feinerman, M.D. studied the pathology of Tay-Sachs disease and developed methods to improve the beta-hexosaminidase A activity, correct misfolded proteins that did not function and empty the substrates that form in cells. Dr. Feinerman, who trained at the Mayo Clinic and currently practices in Sarasota, FL, partnered with a colleague, Javier Paino, M.D., Ph.D., to take his research further. Dr. Paino is a scientist and neurosurgeon who trained at George Washington University School of Medicine and NY's Mt. Sinai Hospital Medical School. In 2010, Dr. Feinerman and Dr. Paino provided treatment for the first time to a child from Alabama who suffered from Tay-Sachs. 14-month-old Aspen Brown exhibited signs of improvement within days of the treatment. She was able to grasp, move her neck and head, follow the movement of objects and had increased muscle tone.

Dr. Feinerman and Dr. Paino continued their research and took it a step further in April 2011. Without using a viral vector, they inserted the HEXA gene into neuron stem cells. Those stem cells were then administered into the brain and central nervous system. At the Concebir Clinic in Lima, Peru, Dr. Feinerman and Dr. Paino administered the gene treatment to three children. Two days later, the doctors were met at the hospital with greetings from the overjoyed parents of those children. The children were able to move their legs and arms and make movements with their heads and necks. They could show facial expressions, make sounds and follow the movement of objects. They were able to swallow more and had a decrease in drooling. Shortly after treatment, two of the children who had previously suffered from many seizure per day no longer had seizures. Within the next few months, Dr. Feinerman and Dr. Paino are planning to provide the gene treatment again and are very hopeful that this therapy may help the many children who suffer from Tay-Sachs.

To learn more about StemCellRegenMed or this ground-breaking stem cell gene therapy for Tay-Sachs disease, contact Burton Feinerman, M.D., at 1(877) 760-8784 or bfeinerman@hotmail.com and visit http://www.stemcellregenmed.com

About StemCellRegenMed:
StemCellRegenMed 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 disease.