|Renee Reijo-Pera and Bertha Chen|
ALS nerves in a dish yield therapy candidate. We often write about our ability to make iPS type stem cells from the skin of a patient, turn those cells into the tissue impacted by a disease, use those cells to understand the defect and test potential therapies. However, we are often detailing projects that have accomplished a couple of those steps.
This week a CIRM funded team at Cedars-Sinai in Los Angeles reported completing all those tasks with cells from a patient with the most common form of inherited ALS, Lou Gehrig’s disease. They replicated the disease, analyzed it, verified the defect and treated it. They found the defect causes a buildup of the intermediate form of genetic code knows as RNA, and verified that this buildup becomes toxic to the motor neurons. They then used a genetic trick called an antisense molecule to stop the excess buildup and get rid of what was already there. The work was published in Science Translational Medicine and a detailed press release from Cedars was posted to EuekAlert.
The release notes that CIRM funds a team hoping to begin a clinical trial with another approach to treating ALS. You can read about that project and others we fund on our ALS information page.
Another reason to love iPS—study evolution. With evolution denial creeping into the high school curriculum in a few states, I have to applaud any discovery that helps us study the underpinnings of evolution. Human genes seem to be more stable than the genes from lesser primates, but no one has known why.
Since iPS technology allows us to reprogram adult tissue into stem cells that can be grown long-term in the lab, researchers can now compare living cells from humans and non-human primates. In the current study, a team at the Salk Institute in La Jolla is comparing our genes to those in chimpanzees and bonobos. The team looked at the activity of “jumping genes,” which earn their name because they literally shift around in the chromosomes and in doing that can cause changes in neighboring genes. They found human cells had a much lower level of jumping gene activity. That could explain the relative stability of our genetic material. They published the paper in Nature and it was described on the web site HEALTHCANAL.
First windpipe holding up over time. Five years ago, a team in Barcelona for the first time implanted into a patient an artificial windpipe engineered with stem cells. They used the patient’s own stem cells seeded on the windpipe of a human donor that had been stripped of its soft tissues so that it was just a scaffold.
It was a pleasure to read in several outlets this week stories telling that the patient is doing well and leading a normal work life. The researchers published the update in Lancet and US News & World Report was one of many sources reporting the good news.
Stem cells and infant eye disease. Underdevelopment of the optic nerve is the leading cause of blindness in children and there are no effective therapies. That unmet need has driven many families to go to clinics in China that are offering treatments with umbilical cord blood. The claims of benefit from those clinics have been difficult to verify because children with the condition do sometime have spontaneous, but often transitory, minor improvement in vision.
One of the most well known of those clinics, Beike Biotech, agreed to let researchers at Children’s Hospital Los Angeles monitor 10 of its patients in China and compare them to untreated control patients back in Los Angeles. The Chinese clinic was only able to recruit two patients during the study period, but those patients showed no differences in a number of subjective and physical measurements compared to the controls. This is the type of comparative trial that needs to occur for most therapies being offered by unregulated clinics. The team published the results in the Journal of the American Association for Pediatric Opthalmology and Strabismus and it was covered by ScienceDaly.