Friday, May 10, 2013

Stem cell Stories that caught our eye: getting therapies to market, creating new bone, healing skin ulcers

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.

Getting a therapy to market. Fierce Biotech published an insightful interview with Henry Termeer, former CEO of Genzyme, which was the first company to market a stem cell-derived product, one for cartilage repair. In the years he lead Genzyme the company gained a reputation for developing drugs for rare diseases, but also developing the reimbursement structures and markets that allowed them to become products for patients, not just research finding. Since he left the firm Henry has become a consultant to many small firms trying to travel that difficult path, including Verastem, which is developing drugs to rein in errant cancer stem cells. Henry sat on our advisory board during my years at Harvard Med School and I came to greatly respect his insights. This interview shares much of that learned wisdom from years in the field.

Bone as a living organ. While many teams have created bone cells from various types of stem cells, our friends at the New York Stem Cell Foundation have taken it one step further. They created bone tissue in three dimensions that contained the compartments for the blood vessels and nerves needed to make the bone a living organ. They started with stem cells created by reprogramming skin cells, so called iPS cells, which could come from a specific patient in the future making them compatible with the patient’s immune system. The work requires many more steps to become a therapy, but these structures with all the parts of functional bone move it significantly closer. Bio-Medicine wrote about the work, as did we.

Smart route to drug discovery. We have often written about the ability to make iPS cells from a patient with an inherited disease and model that disease in a dish. You can not only find out more about what is really causing the symptoms, but also test potential therapies. A CIRM-funded team at University of California, Los Angeles has use iPS cells to screen for potential drugs to treat a rare brain disease that is a form of ataxia. We blogged about that work here.

Budget cuts impact research. Many folks have written print and online commentaries on how the mandatory federal budget cuts known as the sequester will impact the pace of scientific research. This analysis starts with a formal report developed by the Federation of American Societies for Experimental Biology. The stories from some of the group’s members do make you wonder how much slower progress to therapies will become because of these cuts.

Healing wounds today. So much of stem cell science is so new, we most often write about potential therapies that are several years from reality, or just beginning clinical trials in people. It is easy to forget there are a few stem cell-based products already on the market. Organogenesis has one of those products. It speeds healing of difficult to heal skin ulcers. The firm’s product contains two types of cells on a scaffold to hold them in place. One is a type of skin cell that seems to secrete growth factors that accelerate healing according to data from nearly 1,500 patients presented at a recent meeting and described in this article. Geoff MacKay, the firm’s president is quoted discussing the fact that many of these patients had struggled for long periods with wounds that just would not heal. Geoff is also president of the Alliance for Regenerative Medicine. CIRM is a founding member of the Alliance and I had the privilege of chatting with Geoff at the group’s annual meeting in D.C. earlier this week. He is passionate about the field and its potential as a whole, while obviously proud that his company is able to make a difference in people’s lives today.

Silly and instructive. This video of an animated stem cell as an arrogant actor is quite fun. Predictably, the actor is proud of his ability to play any character. He boosts, “Working with me, it’s not easy. But frankly my dear I don’t give a damn.” Stem cells are not easy to work with, but their potential is so great, it is worth the effort.

D.G.

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