Scientists have had little success in growing skeletal muscle for patients with muscular dystrophy and other disorders that degrade and weaken muscle. Undertaking experiments in zebrafish, mouse and human cells, researchers have identified a way to do that, creating cells that Leonard Zon, MD, hopes to see tested in patients in the next several years.
But what really excites Zon, director of the Stem Cell research program at Boston Children’s Hospital, is the power of the chemical screening platform he and his colleagues used. Described last week in the journal Cell, it found a cocktail of three compounds that induced human muscle cells to grow—in just a matter of weeks. Zon believes it could fast-track drug discovery for multiple disorders. …
There are many HSCs in the bone marrow, but getting them out in sufficient numbers is laborious—and for the donor, can be a painful process. Small numbers of HSCs circulate within the blood stream, but not nearly enough. And while umbilical cord blood from newborn babies may present a relatively rare but promising source for HSCs, a single cord generally contains fewer cells than are necessary.
And here’s the rub: The demand for HSCs is only going to increase. Once a last resort treatment for aggressive blood cancers, HSCTs are being used for a growing list of conditions, including some solid tumor cancers, non-malignant blood disorders and even a number of metabolic disorders.
As a hematologist, I see all too many children battling blood disorders that are essentially untreatable. Babies with immune deficiencies living life in a virtual bubble, hospitalized again and again for infections their bodies can’t fight. Children disabled by strokes caused by sickle cell disease, or suffering through sickle cell crises that drug treatments can’t completely prevent. Children whose only recourse is to risk a bone marrow transplant—if a suitably matched donor can even be found.
Over the past 20 years, my lab and that of George Daley, MD, PhD, at Boston Children’s Hospital have worked hard to give these children a one-time, potentially curative option—a treatment that begins with patients’ own cells and doesn’t require finding a match. …
If you look at the range of research models available to scientists today (from fungi to flies to mice and larger), one little guy stands out – a tropical freshwater fish from the rivers of Bangladesh called the zebrafish. While it may be small, this fish is having a big impact on medical science, especially in genetics, stem cell biology, and drug screening, as covered in today’s Wall Street Journal.
As we’ve mentioned previously on Vector, the zebrafish is swimming its way into many research programs, both here at Children’s Hospital Boston and across the country. As a model, they are quite attractive to researchers, in part due to their small size, their fecundity, and their surprising similarities to us (from a genetic standpoint, that is).
One of the characteristics that make zebrafish a fantastic model for research is that they spawn…a lot. A healthy female zebrafish can lay upwards of 1,000 eggs each week. By comparison, the mouse, another species widely used in research, may have a single 12-pup litter each month.
Sometimes, though, that isn’t enough. A researcher screening a library of chemicals for potential drugs, for instance, might need tens of thousands of zebrafish embryos, all at the same developmental stage, to have statistically meaningful results.