A recent clinical trial conducted at Great Ormond Street Hospital in London, England, has successfully treated seven patients suffering from the genetic disorder Wiskott-Aldrich Syndrome by utilizing autologous [the patient’s own] stem cells. The therapy was an example of translational genomics, in which doctors extract autologous stem cells, correct the faulty gene that causes Wiskott-Aldrich Syndrome, and then implant the stem cells back into the patients to produce new, healthy cells.
Today, stem cells are rightfully perceived as the future of regenerative medicine, set to bring the marvels of science fiction into reality. But in looking ahead at all of the promise that stem cells hold for the future, it becomes easy to miss the scientific advances made to date for the millions of people around the world suffering from disease, trauma, and injury. Thus, today marks Stem Cell Awareness Day: a global celebration of stem cell research coordinated to highlight the treatments and therapies currently in development to create personalized regenerative therapies for patients.
Researchers at the Cedars-Mt. Sinai Regenerative Medicine Institute, led by Dr. Robert H. Baloh, have devised a method to study and develop potential treatments for Amyotrophic Lateral Sclerosis [ALS] by utilizing patients’ stem cell-derived neurons. In an example of what is referred to as translational genomics, researchers extract autologous (the patient’s own) stem cells, correct the defective gene causing the disease, and then, in a potential treatment protocol, transplant the cells back into the patient to reverse neural degeneration.
A team of Gladstone research scientists has utilized stem cells to develop a new gene-editing technique for inducing and studying rare genetic mutations linked to disease. The technique, called TALENs, presents an opportunity for patients with genetic disorders to use their own stem cells to identify their specific genetic mutations.
Researchers at UMass Medical School use human stem cells to ‘shut down’ the chromosomes causing Down syndrome. The lead researcher, Jeanne B. Lawrence, a professor of cell and developmental biology at UMass Medical School, explained, “Our hope is that for individuals living with Down syndrome, this proof-of-principal opens up multiple exciting new avenues for studying the disorder now, and brings into the realm of consideration research on the concept of ‘chromosome therapy’ in the future”. The treatment seeks to address the root cause of the disease as opposed to merely mitigating the symptoms of the disease.
Gene therapies utilizing stem cells are being developed that may lead to a cure for several genetic diseases. Currently, two clinical trials were announced to treat Cerebral Adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease.
Recently, researchers utilized stem cells to successfully treat six children with rare genetic diseases. Three of the children were born with Metachromatic Leukodystrophy (MLD), a hereditary neurodegenerative disease. The other three children were born with Wiskott-Aldrich Syndrome (WAS), a hereditary immune system disorder.