The mission of Jacob’s Cure is to support the advancement of research in gene-therapy and neural stem cell transplantation that has exhibited great hope in curing Canavan. Stem cells have the unique ability to provide what the brain is lacking, while also repairing any damage that has already occurred.

In addition to raising awareness and funds for research, Jacob’s Cure launched a campaign to obtain the support of federal funding for Canavan Disease. Categorized as an ‘orphan’ disease, federal funding for Canavan has been in short supply and federal grant applications by researchers are often denied. It has been the sole result of parental fund-raising since 1994 that has funded an estimated 7 million dollars towards therapeutic research.

Jordana Holovach founded Jacob’s Cure in the fall of 2000 because of her passionate fight to save her son, Jacob, who is afflicted with Canavan Disease. Because of an enzyme deficiency, Canavan demyelinates the brain. Without myelin or white matter, Jacob never reached his first milestone...lifting his head. He is essentially trapped in his body. Without intervention, Jacob would lose his sight, his ability to swallow, may be stricken with seizures and become too weak to fight off illness.

In 1998, when Jacob was 2 years old, Jordana’s efforts resulted in Jacob’s enrollment in a Phase 1 Gene-Therapy Clinical Safety Trial. Jacob, along with 14 other Canavan children exhibited positive results and miraculously, Jacob developed new myelin.

For one year, Jacob and 24 other children suffering from Canavan waited helplessly for the approval of an advanced gene-therapy procedure that had fallen victim to a lengthy FDA review process that had begun in May 2000. In April of 2001, the approval was finally received and Jacob underwent this critical procedure on July 10, 2001. The results have been dramatic. Miraculously, Jacob again developed more myelin. He has gained significant weight, his eyesight has improved, he is stronger and uses his vocalizations to communicate. Other families of treated Canavan children report the same findings.

On March 15, 2001, Jordana was invited to testify before the House Sub-Committee on Labor, Health, Human Services and Education. As a result of her passionate pleas and additional lobbying with House and Senate delegation, the NIH approved a 2.5 million dollar grant that supported our gene-therapy research project and the clinical trial that was held at Thomas Jefferson University and then at Robert Wood Johnson/Cooper Hospital in Camden, New Jersey.

With great achievements, not only have the lives of Canavan children been lengthened, but also simultaneously, quality of life has been enhanced. Equally important is the application of research findings to more common brain disorders like Parkinson’s, MS and ALS. The momentum of research is quickly moving ahead towards a cure, but costs have vastly increased because of staffing requirements, materials, equipment and clinical costs. Estimated budgetary cost for the next three years is a staggering 7 million dollars, a task too large for the parents of dying children who must also care for and manage the day-to-day therapeutic, educational and medical needs of typical Canavan children.

Without this much needed support, research may cease or most definitely move at a snail pace if only to rely on the efforts of parental fund-raising. Children afflicted with Canavan will continue to die and any scientific gains that have already been achieved will have been wasted, throwing away any knowledge already achieved towards a cure for this devastating disorder and other diseases that look to Canavan as a model.