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Treatment

Batten disease is a terminal illness, with no cure. Palliative treatment is symptomatic and supportive, however, there are possible treatments available to help alleviate symptoms. Drugs to control seizures may benefit some patients, and physical and occupational therapy can help with motor problems that develop. Ongoing research is studying anti-inflammatory drugs to treat the effects on vision that often develop with the disease. Ongoing Research

There are many current studies being done to develop treatments for patients with Batten disease.

Gene Therapy-Researchers have studied the effectiveness of gene therapy in slowing the progression of symptoms of the disease. They used an adeno-associated virus that included the gene that encodes tripeptidyl peptidase 1 into the brains of canines that have the disease. They saw that symptoms of the canines progressed much slower and that they always lived longer when given the gene therapy treatment.

Enzyme Replacement Therapy-There are many studies involving replacing the deficient enzyme with human product. One study treated mice by giving them tripeptidyl peptidase 1 via the ventricular system of the brain. Side effects of the disease were blessed when treated with the enzyme, specifically the tremors that are characteristic of the disease. Enzyme replacement therapy has also been studied is other animal models including the monkey and canine.

Stem Cell Therapy- Stem cell transplantation is used to treat patients with other diseases similar to Batten disease. Researchers are currently studying the most effective use of stem cells for in patients with the disease, as well as what kind of stem cells, hematopoietic stem cells, neural stem cells, and induced pluripotent stem cells, would provide the most success in treating the symptoms of the disease. One study transplanted human stem cells into the brains of mice with no PPT1 enzyme and found that the mice showed improvements in the disease course which could potentially lead to treatment in human patients.