Thanks to scientific innovation, researchers can now take adult body cells, such as a skin or blood cell and turn back the clock to create what are called induced pluripotent stem cells (iPSCs). Pluripotent cells are unspecialized cells that can form virtually any cell of our body.
The promise of this new technology is that researchers can now either create healthy iPSCs that can be used as a treatment for a variety of diseases or scientists can create “diseased” iPS cells to determine the impact of different therapies on these diseased cells. Thus, diseased iPS cells can become part of the drug development pipeline.
Dr. Marcel Daadi has more than 20 years of experience conducting research in stem cell biology and preclinical development of therapies that use stem cells to treat debilitating diseases. His primary focus is on developing a stem cell treatment for Parkinson’s disease, which is a consequence of the death of the brain cells that produce dopamine.
Daadi’s strategy is to take a skin biopsy from a person, use those skin cells to create iPS cells, and then to treat the iPS cells with growth factors that cause them to differentiate into healthy brain cells that produce dopamine. These dopamine-producing neurons can then be implanted into the brain of a Parkinson’s patient, replacing the dead and dying cells and restoring brain function. While Daadi’s primary interest lies in Parkinson’s disease, the approach with stem cell therapy holds similar promise for treatment of post-traumatic stress disorder, traumatic brain injury and stroke, all of which Daadi is studying.
Dr. Tiziano Barberi has more than 20 years of experience in stem cell biology. His primary areas of focus are the directed differentiation of PSCs and iPSCs, with the goal of using these cells to treat muscular dystrophy, and eye disorders.
Barberi explains that muscular dystrophy is currently an incurable disease. His long-term hope is to use PSC-derived muscle precursor cells that he makes in the lab and replace the diseased muscle upon transplantation of these healthy muscle cells. For the short term, Dr. Barberi is optimizing procedures for functional tests in animal models.