Over 5 million Americans suffer from congestive heart failure. Heart failure is characterized by the loss of functional heart cells and thereby its inability to pump enough blood to maintain physiological functions. The last resort for the patients with end stage heart failure is heart transplantation. Often, this is limited by the availability of a perfectly matched organ. The demand for organ supply is increasing steadily, necessitating development of new therapeutic options.
The recent technology termed “Cellular Reprogramming” enables creation of a versatile type of stem cells called “induced Pluripotent Stem Cells (iPSC)” from any somatic cell, such as skin, blood, fat, and hair follicle. These stem cells have 2 important characteristics:
- the potential to grow indefinitely in a lab
- the potential to give rise to any cell type including:
- insulin secreting beta cells
These cells hold promise for cell-based regenerative therapy of degenerative diseases; including heart failure, Parkinson’s disease, Alzheimer’s disease, blindness and diabetes. These cells hold the potential to be grown and transplanted into patients to repair failing organs, the immune system, or a number of novel treatments.
Scientists in our Stem Cell Center are working to generate billions of clinical grade heart cells from skin cells. The cells grown in our lab, derived from the skin biopsy, hold the potential to patient needing a transplant to receive their own cells, thus removing the need transplant rejection therapy. These cells have the potential to not only repair the organs but with less scaring and faster recovery.
These induced pluripotent stem cells also permit the development of human models of disease using cells isolated from patients with various diseases. These cells, for example, can be directed to grow into heart cells, thus providing individual cells that have the same genetic defect as the patient. By utilization of these cells as cellular models, a more focused treatment can potentially be developed based on the patient’s own genetic makeup, paving the way for the development of personalized treatments.