The National Tay-Sachs & Allied Diseases Association, a non-profit organization dedicated to supporting research in search of a cure, reports that gene therapy results in animals has been promising, and human clinical trials may be underway soon. The therapies are exploring either restoring the missing enzyme or fighting the waste that accumulates as a result of a lack of the enzyme. As of now, scientific researchers believe that no single cure will be found, but a combination of various techniques may stop the accumulated damage and perhaps reverse them.

There are clinical trials underway at the University of Minnesota, investigations by the Metachromatic Leukodystrophy Natural History Study and researchers participating in the Efficacy Study of an Online Educational Module before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent in Montreal, Canada. Admission to these studies is competitive, but some reimbursement for travel, lodging and living expenses may be available. Of course, because these are trials, there is no guarantee that the research will result in any reversal, halting or cure for the disease. Those remain hopes for the future.

Until such time as a cure or other arresting development is discovered, genetic counseling and testing is the best hope for avoiding the heartbreak associated with this terrible disease.