A great day for stem cell research

Stem cell researcher Willy Lensch
Stem cell researcher Willy Lensch

By M. William Lensch, PhD, from the Stem Cell Program at Children’s Hospital Boston

At just a little after 12:30 p.m. EST today we reached the end of a very long road. That was when the NIH announced that the first human embryonic stem cell lines (hESC) had been approved for federal funding eligibility under the rules put forth in President Obama’s executive order from earlier this year. A small group of us here in George Daley’s lab were listening in to the NIH press conference over the speaker phone. I couldn’t help but clap my hands and cheer! A lot of us have worked toward this moment for a long time.

I joined George’s lab toward the end of 2001, not long after President Bush permitted funding for a few hESC lines but restricted federal money for any others. Since that time and despite the restrictions, our lab has continued to work in the field using hESC because of the incredible things these cells could teach us. Quite honestly, an important aspect of what made that work possible was simply being here at Children’s Hospital Boston. Children’s knew the work was important, too important to set aside, and now these years later, we’ve got a tremendous Stem Cell Program because of this forward-looking vision and support. Of the 13 hESC lines approved today, 11 of them were derived here, at this hospital.

The first cell line listed on the official NIH Registry is NIHhESC-09-001. This cell line and several more were published in 2008 from work conducted by Paul Lerou and others in George Daley’s laboratory. The lab-based portion that has brought us to today has been enormous. Each cell line listed in the NIH Registry represents hundreds if not thousands of hours of work to establish, culture and validate before the first actual experiments could even begin. Now that these cell lines are eligible for funding through the NIH, we are able to apply for long awaited federal dollars to bolster the private contributions that have nurtured our work over the years. I’ve always thought of our research as taking up “lost causes” of medicine; conditions where we simply don’t know enough to be able to offer a substantial therapy to patients and their families. We are talking about diseases so rare that the only time I ever meet anyone who has heard of one of them is because a family member or friend has it currently or, unfortunately, died from it. Having hESC lines allows us to ask questions about how tissues form during the earliest stages of both normal and abnormal development alike. It’s a powerful methodology and is forging a veritable renaissance in our thinking about human development and disease.

Human ES cells have also driven the science towards new areas like cellular reprogramming; work that shows that mature cells of the body may be pushed “developmentally backwards” to a state like an embryonic stem cell. These so-called induced pluripotent stem cells or iPSC are an amazing technological breakthrough that, like hESC, are helping us blaze news trails into understanding the very foundations of disease. Our lab has worked in this area as well, publishing papers on not only the basics of cellular reprogramming, but also providing a more detailed analysis of how the process works, how reprogramming may be used to generate cell lines that contain disease-causing mutations, and in turn, how these mutations directly impact complex disease processes. It’s been exciting research (to say the least) and has only been possible because of what we’ve learned using ES cells.

What also came to mind in listening to the press conference today was all of the churches, town halls, libraries, retirement homes, pubs, school classrooms, politicians’ offices, board rooms, and other venues that I and other folks have been to in order to explain this work more clearly and to encourage a more informed approach to national stem cell policy. All the interviews, debates, panel discussions, public lectures, radio call-in shows, and the like have also worked steadily towards this day. Looking back, it’s a bit mind boggling to reflect on it all. For example, in the “early days,” both George and I once lectured in rooms posted with armed police officers just in case things got out of hand. It’s amazing to sit here and remember those events. What’s more important though, is to consider what today’s news means looking forward.

What it means is that we are going to be in a much better position to capitalize on what we’ve accomplished thus far. The “lost causes” still remain and the most important days of research remain ahead of us. I look forward to them, very much in fact. Today’s news also reminds me that hard work pays off and that having the patience to continue making your case despite other, often louder voices, can lead to better things. It’s been a group effort with a cast of thousands. To all of them, I say, congratulations. As I mentioned at the beginning, we’ve reached the end of a very long road, though several others continue to stretch out before us. It comes with no small sense of relief to see that we are now better prepared for those journeys than we once were. Here’s to the future.