Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.
Building blood vessels where you need them. One of the treats of my professional career was the opportunity to work with Judah Folkman, the brilliant yet gentle scientist at Children’s Hospital, Boston who developed the theory that we might be able to treat cancer more effectively if we could cut off its food supply by stopping tumors from growing new blood vessels. In my current position everyone here has a keen interest in the flip side of that coin. If we can’t get new tissue built with stem cells to also connect to blood vessels, that tissue—no matter whether it’s a patch on a broken heart or new cartilage for a sore knee—will not thrive and survive. Also for some diseases, the only new tissue you need is those new blood vessels. Now a team at the University of Michigan reports that with the right mix of cells they can get new blood vessels to grow and remain functional over time. Judah’s theories have resulted in a top selling cancer drug; let’s hope this yin of that yang works as well.
Stem cells as carpenters. Three faculty members from CIRM-funded UC San Francisco have written a thoughtful review of where cell-based therapies will fit into medicine in the future. They describe it as the third pillar of therapy, with the first two being common drugs and the second the newer so-called biologics, the antibodies and other proteins first created by the biotechnology industry. One of the authors states, “if small molecules and biologics are tools, then cells are carpenters — and architects and engineers as well.” The full review was published in the journal Science Translational Medicine, but a shorter summary made it into the popular media.
Lessens from clever little viruses. Some viruses have an innate ability to evade our immune system, which is supposed to detect them and eliminate them. This is an ability that would be nice to embed in replacement tissue grown from stem cells that came from a donor. Many in the field think that cells from donors are more likely to be commercially viable than using a person’s own cells, because they would more closely model an off-the-shelf medication. But immune rejection is a huge problem and even many clinical trials reporting positive results show that the impact of the cells occurs early because the cells generally don’t survive very long. Now a team at Wake Forest University has looked at a virus that most of us carry, but our body never notices. They found a protein that seems to be acting as a science fiction-like shield of invisibility. They have genetically engineered that protein into adult stem cells, and indeed found longer survival in recipients—just mice for now, but it is promising.
15 stem cells in late-stage clinical trials—Part 2. A blogger I follow is writing about the 15 stem cell trials in late stages of testing known as Phase 3. When stem cell research pushes a therapy into the clinic the cells need to pass through three phases, the first checks for safety, the second looks for early signs that the cells make a difference, and the third is pivotal. That Phase 3 trial has to show definitively that the cells benefit the patient and do so better than any standard therapy. Here is his second installment.
Research funding cuts will slow progress. I have spent much of my career arguing for the need for sustained, predictable funding for research. Science is not an enterprise that you can turn on and off quickly like an oil pipeline. Building a lab with talented people and the sophisticated equipment they need takes time. And splitting up that team when funding dries up inevitably slows progress toward new therapies. I had to make this case on Beacon Hill in Boston, and Capitol Hill in Washington, but I have not had to fight quite so hard at CIRM. The wise folks that drafted the ballot initiative that created the agency locked in our funding for at least ten years or a bit more. However, we have always said that CIRM can make the greatest contribution in collaboration with other funding, in particular federal dollars from the National Institutes of Health. The Sacramento Bee published a thoughtful opinion piece on the impact the budget cuts known as the “sequester” will have on NIH and the work it funds.