Stem Cells Being Studied as a Treatment for Batten Disease

Stem Cells Being Studied as a Treatment for Batten Disease

Batten disease is a devastating condition for patients and their loved ones because it is generally a death sentence. The symptoms are also extremely hard to handle because they leave a person unable to do most things they love. It is a disease that children are born with that progressively gets worse as they age. Stem cell therapy is being studied as a treatment and cure for Batten disease so patients can live a long and happy life with their families. Stem cells may be able to treat the symptoms of Batten disease and stop the progression of the condition.


What is Batten Disease?


Batten Disease is a fatal, inherited disorder of the nervous system that begins in childhood. The first sign of the disease is often loss of vision, and may first be suspected during an eye exam when a patient is young. Other early signs are subtle but may include personality and behavior changes, slower learning and development, clumsiness, and/or stumbling. Over time, affected children suffer mental impairment, worsening seizures, and a progressive loss of sight and motor skills. Eventually, children with Juvenile Batten Disease become blind, bedridden, and completely unable to communicate with their loved ones and caregivers. Juvenile Batten Disease is always fatal by the late teens or twenties.


What is Stem Cell Therapy?


Stem cells have recently been used in regenerative medicine because of their ability to repair damaged cells and create new, healthy ones. Stems cells are the source for all our tissues, muscles, bones, and brain cells. There are many different methods of harvesting stem cells from our own tissues, with some methods being much easier than others. Stem cells are found in embryonic cells, adult stem cells like bone marrow cells and fat tissue cells, adult stem cells that have been changed into embryonic cells, and perinatal cells. Several of these stem cells have the ability to become mesenchymal stem cells, which can become bone, fat, and cartilage. The easiest and most pain-free method of harvesting stem cells in adults is from a patient’s own fat tissue. These fat-derived stem cells never decline in abundance as you age, as adult bone marrow stem cells do. Bone marrow stem cells can also be extremely painful to harvest, unlike fat cells that are quick and painless to sample. Embryonic stem cells have been considered unethical to use, as they use unborn embryos to harvest the stem cells they use to create organs and tissues for a fetus. Researchers have attempted to overcome this ethical dilemma by using embryos that are fertilized outside of the womb who are not destined to become a living human.


How Can Stem Cell Therapy Treat Batten Disease?


There is no cure for Batten disease, but stem cells may offer a solution for the symptoms and progression, allowing patients to live longer than their average estimated life span. Clinical trials of stem cell therapies have been carried out in some types of Batten disease, and preclinical studies in animals are ongoing to improve the approach. The treatment has been proven as safe, but the results were not as promising. The patients studied had advanced stages of the disease, so more research needs to be done in patients who are not as severely affected. It may be a treatment to prevent the disease from progressing further in the earlier stages of Batten disease in children.

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