Zeitschrift für Regenerative Medizin

CD133 Clinical Trials: Safety and Efficacy

Matti Ullah, Marc Pocard, Massoud Mirshah

In human body there are cells that can proliferate and differentiate into various kinds of adult cells known as “Stem cells”. These cells express a membranous protein known as “Prominin-1” or “CD133”. CD133 exhibits 7 isoforms, distributed in different tissues of the body. The isolation and understanding of these cells helped develop an innovative and new kind of therapeutic approach called “Stem Cell Therapy”. Mutations in Prominin-1 gene are known to cause genetic disorders like Stargardt disease and retinal macular dystrophy. On the other hand, in clinical trials, CD-133+ cells are being used to treat certain diseases. These clinical trials are targeted for variety of diseases such as Duchenne muscle dystrophy, severe combined immunodeficiency syndrome, degenerative diseases like Asherman’s syndrome, cerebral palsy, liver cirrhosis and cardiovascular diseases like ischemia and myocardial infarction. CD133+ cells, owing to their stem cell properties, not only help in regenerating the damaged tissues, but also enhance the healing process via decreased inflammatory reactions and slowing down of apoptotic processes. CD133+ has a significant role in cancer studies. In cancer cells, a subpopulation that express CD133 is termed as cancer stem cells (CSCs). These CSCs are rare, proliferative and resistant to chemotherapy and can survive drug treatments, resulting often in relapse of the disease. Many studies have reported the presence of CD133+ cells with the decreased survival rate in cancer patients. Hence, CD133+ cells play an important role in prognosis and outcome of cancer treatment.