One Step Closer to a Regenerative Therapy for HLHS

Dr. Timothy Nelson, director of the Todd and Karen Wanek Program for Hypoplastic Left Heart Syndrome, has brought us one step closer to making a regenerative therapy for HLHS using Induced Pluripotent Stem (iPS) Cells a reality. “Pluripotent stem cells show great promise in the field of regenerative medicine; however, the risk of uncontrolled cell growth will continue to prevent their use as a therapeutic treatment,” says Dr. Nelson, lead author on the study, which appeared in the October issue of STEM CELLS Translational Medicine.

"This scientific discovery directly impacts the utilization of pluripotent stem cells as regenerative therapeutics for many different diseases, including HLHS, by increasing their safety; thereby reducing one hurdle of getting this treatment into the clinic," says co-author Dr. Alyson Smith. She explains that while no therapy is totally safe, minimizing any harmful side effects of a potential treatment is crucial. "This study demonstrates that we can eliminate potential tumor formation by a therapeutic stem cell population (a current, unacceptable side effect) while maintaining their ability to form the reparative heart cells.”

Other members of the Mayo research team included Clifford Folmes, Ph.D., Katherine Hartjes, Natalie Nelson and Saji Oommen, Ph.D. The research was supported by the Todd and Karen Wanek Family Program for Hypoplastic Left Heart Syndrome, National Institutes of Health New Innovator Award OD007015-01, and a Mayo Clinic Center for Regenerative Medicine accelerated research grant.