Nevada
Cancer Institute Researcher Yupo Ma, M.D., Ph.D., has identified a type
of non-embryonic stem cell therapy that may hold the key for a new
treatment of Hemophilia A, a genetic disorder in which blood fails to
clot properly.
Nevada Cancer Institute (NVCI) is the official cancer institute for the State of Nevada. A nonprofit organization, NVCI is committed to reducing the burden of cancer by pursuing the development of a comprehensive cancer research institute, as defined by the National Cancer Institute. Through the knowledge and expertise of the finest scientists, clinicians, educators and caregivers, the Institute provides hope to communities in Nevada, the southwest and beyond through research, education, early detection, prevention and high quality patient care. NVCI is striving for a future without cancer that is achieved through initiated and collaborative research in basic, clinical and population science. For more information, please visit www.nevadacancerinstitute.org or call (702) 822-LIFE.
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Dr. Yupo Ma reports a specific stem cell therapy may hold the key to many diseases in the latest issue of Proceedings of the National Academy of Sciences (PNAS)
Dr. Ma’s study takes adult somatic cells (for example normal skin cells) and turns back their developmental clock so the cells act and “think” like embryonic cells. This process is termed retro-differentiation and it produces cells called induced pluripotent stem (iPs) cells. The paper, titled “Phenotypic correction of murine hemophilia A using an iPS cell based therapy” was published in the latest issue of the Proceedings of the National Academy of Sciences (PNAS) by Dan Xu (a), Zaida Alipio (a), Louis M. Fink (a), Dorothy M. Adcock (b), Jianchang Yang (a), David C. Ward (a), and Yupo Ma (a) With those iPS cells and appropriate growth factors, Dr. Ma then created re-differentiated cells of the endothelial cell type that produce the Factor VIII protein (the clotting factor) that is defective in those afflicted with Hemophilia A. Studies show that Dr. Ma was able to produce enough Factor VIII to reverse the excessive bleeding characteristic of Hemophilia A. Previous attempts at gene therapy had failed for a variety of reasons, including rejection by the immune system, something avoided in this treatment because the subject’s own cells were used. This type of stem cell therapy, Dr. Ma suggests in the article, may also prove useful in treating other types of genetic diseases. Cancer applications with this treatment would make treatments more efficacious, such as replacement of bone marrow by using a person’s own skin cells to create new bone marrow. “This is just the first step in a long list of diseases that could be affected by this application,” Dr. Ma said. “Induced pluripotent stem cells have tremendous clinical potential since they can be directed to make every kind of cell types within the body; this provides endless therapeutic possibilities.” No one has been able to cure Factor VIII hemophilia before. There has been a successful study at MIT using this same method to cure sickle cell. The only way that this could be done until a couple years ago was with embryonic stem cells. Using somatic cells, and utilizing three different factors to revert them to iPS cells, eliminates the ethical issues surrounding embryonic stem cells. It also eliminates rejection issues from someone else’s adult stem cells – the individual’s own cells are used to treat their own specific disease – and is an example of “personalized medicine.” Published in one of the preeminent scientific journals, Ma’s study is an important milestone for the Institute and represents the first of many groundbreaking research endeavors that will soon come to fruition. “This is why we are here – this is the idea that Nevada Cancer Institute was created on; to bring groundbreaking transformational research to Nevada for Nevadans,” said Heather Murren, chairman of the Board. “We expect this to be one of many scientific discoveries that will change the face of medical research going forward.”
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