StemCellRegenMed Continues Commitment to Gene Therapy Treatment for Children with Tay-Sachs Disease

June 30, 2011 - PRLog -- StemCellRegenMed has long been committed to finding therapy for those suffering from Tay-Sachs disease.  It recently strengthened that commitment by providing ground-breaking treatment for three children who have been living with the disease.  With this gene therapy treatment, StemCellRegenMed hopes to offer help to the many children and families who suffer from the devastating effects of Tay-Sachs disease.

A child with Tay-Sachs will begin exhibiting symptoms of the disease at around six to eight months of age, sometimes after following what seemed to be normal development from birth.  Children with the disease may lose the ability to crawl, sit on their own, raise their heads or turn over.  Some may have seizures or lose the ability to see and hear.  Many children with Tay-Sachs will die from the disease before they are four years of age.  A curative treatment or therapy had not been available for these children, until StemCellRegenMed’s ground-breaking treatment.  

Tay-Sachs disease results from a HEXA gene mutation that inhibits production of the enzyme beta-hexosaminidase A.  The beta-hexosaminidase A enzyme aids the breakdown of fat deposits in cells in the brain.  These fat deposit can accumulate to abnormal amounts in the spinal cord and brain, if beta-hexosaminidase A is deficient.  
StemCellRegenMed’s Burton Feinerman, M.D., a Mayo Clinic trained physician who currently practices in Florida, began studying the pathology of Tay-Sachs disease.  He developed approaches to encourage beta-hexosaminidase A enzyme activity, empty substrates that did form in cells and correct proteins that were misfolded and not functioning.  Dr. Feinerman took his research further by partnering with neurosurgeon and scientist Javier Paino, M.D., Ph.D., a long time colleague.  Dr. Paino studied and trained at George Washington University School of Medicine and Mt. Sinai Hospital Medical School in NY.  Together in November 2010, Dr. Feinerman and Dr. Paino provided a new treatment to a child from Alabama who was sufferering from the symptoms of Tay-Sachs.  14-month old Aspen Brown was the first to receive the ground breaking therapy and showed signs of improvement after only a few days.  She exhibited increased muscle tone and could follow moving objects, move her neck and head and grasp objects.  

In April 2011, Dr. Feinerman and Dr. Paino continued their commitment to providing treatment for Tay-Sachs disease by taking their research even further.  They were able to insert the HEXA gene into neuron stem cells without utilizing a viral vector and then administer those stem cells to the central nervous system and brain.  Three children with Tay-Sachs received this ground-breaking gene treatment in Lima, Peru, at the Concebir Clinic.  Much like the improvement of Aspen Brown, the three children showed signs of improvement within days of having received the treatment.  As you can imagine, the parents of the children were overjoyed.  The children were drooling less and could swallow more.  They could follow moving objects and move their heads, necks, arms and legs.  Two of the children who were experiencing frequent seizures prior to treatment were no longer suffering from them.  Dr. Feinerman and Dr. Paino hope that this gene therapy treatment may eventually help many more children who are suffering from the devastating effects of Tay-Sachs disease.  They plan to provide the treatment again in the coming months.  

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 www.stemcellregenmed.com

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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.