Jan 24

Can UW reprogram skin cells to become heart cells? Yes.

In January John Foy,MD and I (shown in photo below)  had an opportunity to visit the Institute for  Stem Cell and Regenerative Medicine of the University of Washington School of Medicine in the South Lake Union neighborhood of Seattle. What we saw there has given me a new perspective on the convergence of genomics, robotics, and the future of medicine.

ISCRM2-ps-cropped

John and I were hosted by Charles Murry, MD, PhD,  Director of the Center for Cardiovascular Biology and a Professor at the School of Medicine. This research is at the forefront of medical advances in the use of stem cells to produce new heart muscle cells.  Dr. Murry explained that people with damaged heart tissue currently have few therapeutic options.  He observed that there have been no new drugs in 15 years for heart failure. Heart transplants are an option for some patients but the supply of donors is constrained and the costs are very high, and so there are only about 2,000 transplants in the US per year. Because there are between 5-6 million heart failure patients, transplantation is not a viable option for most. A second option is the Left Ventricular Assist Device, or LVAD, which can extend the life of a patient  but carries significant risks of blood clots, stroke, and other complications.  Regenerative medicine – using artificially grown cells –  to repair damaged heart tissue is a third option.  Like a transplant, it would mean that the patient would face a lifetime of suppressed immune response to prevent rejection of the new heart cells, but it would presumably have cost savings and better outcomes than either of the current options. They are striving towards human clinical trials and then for use in mainstream practice.

We saw early mouse embryos (blastocysts, the stage where embryonic cells can be derived), which are about the same size as Roosevelt’s eye on the dime. We saw embryonic stem cells, derived from human blastocysts “left over” from fertility clinics, which Dr. Murry says can divide forever and turn into any cell type in the body. In addition to the embryonic cells, Dr. Murry extracted some of his own cells from in the skin of his arm to be regressed – through genetic manipulation –  from mature skin cells back to create a new stem cell line. From that point these former skin cells have been genetically programmed to become real human heart muscles. I saw heart muscle cells beating in a dish during this tour. The Institute has engineered 3D pieces of beating human heart muscles from these cells. It was truly an amazing sight.

ISCRM1-psThe purpose of our trip was to explore collaborative opportunities across the health innovation community in Seattle.  The hour we spent at the Institute for Stem Cell and Regenerative Medicine (ISCRM- pronounced as “ice cream”) provided strong confirmation that this potential is very real. Like most of the denizens of the Seattle startup community, the people at ISCRM are increasingly thinking and acting like entrepreneurs. The Heart Regeneration Program is becoming the equivalent of a biotech startup with about 8 employees. They recruited new talent, including Scott Thies, PhD, from Fate Therapeutics in San Diego to provide business leadership for this venture. Their goal is to be ready for a Phase 1 clinical trial in 4 years.  This is a strategic shift beyond their historical reliance on state and federal grant funding. The transformation of the Heart Regeneration Program from academic research to a biotech startup is evidence that the entrepreneurial climate in this area is, like their cells, alive and growing.

Another aspect of  convergence that readers of this blog may appreciate is the use of advanced robotics to discover the right recipes for achieving the reprogramming of stem cells. Tim Martins, PhD demonstrated the Quellos High Throughput Screening Core, a shared facility that serves many of the programs at ISCRM and other organizations. Because of this technology, a brute force approach can be taken to test every possible combination of certain reagents and concentrations.

The process of programming human stem cells –  which Dr. Murry called “stem cell wrangling” – is on the frontier of medical science. It might not be too great a stretch to see it as engineering of the human platform.

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