Stem Cell Therapy Could Offer Hope for Children Suffering from Hurler Syndrome

Stem cells are being tested in hundreds of clinical trials across the country right now. Stem cells have shown promise at treating several types of conditions and could one day be used to treat and cure ‘incurable’ diseases and syndromes. The Keck School of Medicine at the University of Southern California (USC) recently may have unlocked a stem cell therapy that could treat a rare genetic condition in children known as Hurler syndrome.

Hurler syndrome takes away a vital enzyme from the body that helps control the breaking down of molecular building blocks that naturally occur in our tissue and bone. Without policing this molecule, blocks build up and can interfere with the body’s natural processes. This buildup causes several problems in the sufferer including loss of coordination, breathing difficulties, abnormal growth, joint stiffness, and other abnormalities. Sufferers of Hurler syndrome do not typically make it past the age of 10.

Researchers at USC, led by Toshio Miki, found embryonic stem cell injections in mice with the disorder helped restore enzymatic function that is found lacking in Hurler syndrome. The mice that received the injections had better coordination and reduced issues in bone and growth development. Miki believes the same treatments can be used in humans to erase the problem genes and allow children with Hurler to live normal lives.

Stem cell clinical trials across the world are focused on several different goals and breakthroughs. The team at USC is using stem cells to treat a rare disorder while other doctors are focusing on issues that may not be as debilitating as Hurler syndrome but affect millions of Americans.

Millions of Americans suffer from chronic musculoskeletal diseases and conditions like arthritis, the breakdown of cartilage, and degenerative disc disease. Kenneth Pettine MD (retired) and his team based in Colorado are putting stem cells to work in clinical trials to reduce the symptoms and issues associated with these diseases and to help patients avoid invasive and expensive surgery. Pettine’s results looked promising with the majority of the patients in his trials showing improvement and increased the quality of life against these disorders.

Stem cells may one day be used for cancer, paralysis, genetic disorders like Hurler syndrome, and even a bad neck or knee. The research is happening right now across the world, and it will help bring stem cells to a doctor’s office near you.

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