Stem cell stories that caught our eye: replacement organs, gene therapy for HIV and NIH budget cuts

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. 

Dr. Anthony Atala shows a kidney created using a 3D printer – Photo by Steve Jurveston  

Progress in creating replacement organs. Malcolm Ritter from the Associated Press wrote a good lay-level review of our progress toward one of the grand long-term goals of stem cell science—generating replacement organs. It got picked up the last few days all around the country. Here is one version. He duly notes that we have had considerable success, but only on very simple organs, sheets of skin and simple tubes like blood vessels and wind pipes. He interviews the leaders in the field who describe the hopes for building more complex organs, and the many hurdles that remain in achieving that goal.

Growing whole organs from scratch. While most work toward creating replacement organs involves seeding stem cells onto scaffolds like the work Malcolm discusses above, a Japanese researcher has proposed an entirely different approach. He wants to get pigs to grow new organs that contain human cells. For diabetes, for example, he starts by genetically altering pig embryos so that they can’t produce a pancreas, the organ needed to produce insulin. He proposes to then insert human stem cells, into those embryos. That should succeed in directing the pig embryo to make a human pancreas. After the piglet was born, the pancreas tissue could be isolated for transplant in patients. If the stem cells were made by reprogramming the patient’s skin into iPS type stem cells, they would be a genetic match and be less likely to be rejected by the patient’s immune system, making them better than current transplants. He has shown this works in other species, but Japanese law does not allow him to grow human tissue in a piglet that is born. This article in the journal Science discusses the research and potential changes in the regulations being proposed in Japan. The work would be legal in the U.S.

Gene therapy in stem cells for HIV. CIRM funds two multi-institution teams that are trying to recreate “the Berlin” patient, the HIV-positive person who needed a bone marrow transplant to treat his leukemia, and was given marrow stem cells from a person who was naturally immune to the virus. It worked for his leukemia and to give him an immune system that seems completely resistant to the virus. Our two teams are trying to use gene modifying techniques to alter a patients’ own stem cells so they have the same gene mutation as the Berlin patient’s donor. David Baltimore, one of the co-founders of Calimmune, the company leading one of our projects, discussed the effort at the recent World Science Festival in New York. This biotech web site offers a nice comparison of the two projects CIRM is funding and notes that the Calimmune project is taking the extra step of altering two different genes involved in infection by the virus. You can read more about both projects here.

Stem cells shed light on cancer. A new paper from the Harvard Stem Cell Institute (HSCI) used the understanding of how embryonic stem cells function to find a potential cancer therapy. They successfully tested it in liver cancer, but speculated that it might be effective in many cancers. Having worked with the founders of HSCI in the early years of the organization I know they were looking forward to both stem cell-based treatments, and medical advances made by understanding normal human function that could only be found using embryonic stem cells. In the current paper, published this week in the New England Journal of Medicine, and discussed in a university press release posted by Science Codex , they found that aggressive forms of cancer have a gene turned on that is normally only turned on in stem cells in the early stages of embryonic development. An inhibitor of that gene is the potential therapy they found.

Patient hopes versus federal cuts. CIRM works frequently with many patient advocates, many of whom are patients who struggle through major adversity to work toward the long-term goal of better therapies. The St Louis newspaper did a nice write-up on one of the more remarkable tails of patient determination. It details the struggles of Patrick Rummerfield who was a paralyzed in a car accident, and after intense physical therapy was able to participate in a triathlon. He finished last, but he did it. The article concludes with Rummerfield talking about the hope he has for stem cell research, and his fears that those hopes will take much longer to materialize now because of the federal budget cuts known as the “sequestration.”

Senate leader laments NIH budget cuts. I came across this video clip from the Senate floor yesterday thanks to a Tweet from the National Institutes of Health director Francis Collins. In it, Senate majority leader Harry Reid talks about the many lives saved through research funded at NIH, noting that there has been a 60 percent drop in deaths due to heart disease in the past 50 years and that AIDS is no longer a death sentence. However, he called the cuts from the sequester “short sighted cuts that will cost us cures tomorrow.” They slash $1.5 billion from NIH this year and $4 billion over the next two years. He lamented that while we are cutting our investment, our competitors are increasing theirs, siting a 25 percent increase in China, 20 Percent in India and 10 percent in Korea, Brazil and Germany. But one of the biggest losses he noted, may be the promising young scientists who will abandon the field of research completely. When we loose them, we loose a generation of scientific creativity



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