At this time, there is no known cure for Tay-Sachs disease. There are medications only available to help make the patient more comfortable and able to function a bit better. However, scientists, doctors, and researchers are working together to find a cure for this illness and they have been coming up with some cutting-edge techniques and research theories. Some of these studies include stem cell therapy to help slow or reverse the effects of Tay-Sachs disease.
There is research being done with adult stem cells, umbilical stem cells, and Hematopoietic stem cells which are cells that have not differentiated yet. It is hoped that stem cells could help replace genes that are defective or mutated such as the Tay-Sachs mutation. There has even been research on using stem cells to regrow tissues and neurons. Scientists are hopeful, but they have said on many occasions that any cures resulting from stem cells are still many years of research and experiments away.
There are many controversies with the use of stem cells in research to cure diseases and illnesses. Many are ethical and moral issues involving the type of stem cells used and in what way they are being utilized. A large barrier to using stem cells for treatment is that we still don’t know enough about these cells and what they can and cannot do. It’s difficult to just put something into someone’s body when we do not fully understand the pros and cons of that treatment.
When it comes to Tay-Sachs, scientists are running into challenges with how to best insert the stem cells into the brain of an affected person and having the cells aid in transport of genes and enzymes into the correct neurons. Also, it’s difficult to find a cure or therapy that can be inserted into the bloodstream and then cross the blood brain barrier. This is a very, very selective barrier the brain has so as to protect this vital organ from bacteria and diseases that may be in the blood itself, but this is what makes treatments difficult to work properly. Tay-Sachs results from problems in the brain itself, and if the treatment cannot simply be inserted into the bloodstream and pass this barrier, it becomes so much more difficult to administer therapy effectively and safely. This barrier is there for a reason and to just manually insert the stem cells into the brain may increase the likelihood of infection and bacteria getting into the brain which is potentially fatal. Also, the brain is less likely to accept the new stem cells when they did not “naturally” come into the brain via normal passageways.
Stem cell research is looking more and more like a possible treatment for not only Tay-Sachs, but other diseases and illnesses, as well. There is still a long road ahead of research studies and experiments and treatment trials with stem cell therapy and transplantation.
