Stem Cell Stories that Caught our Eye: Gene Rx, New and Rejuvenated Blood Stem Cells and 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.

Tinkering with stem cell genes safe. Research at the Salk Institute provides some reassurance that using gene-editing techniques to correct disease-causing mutations in stem cells is safe. This type of intervention aims to give people a corrected version of a gene that can produce a functional protein to replace the bad one they were born with, such as the hemoglobin gene in sickle cell disease. The CIRM-funded Salk team made gene corrections with both of the two most common gene-editing techniques: using a virus to carry the correct gene into the cell, and using an enzyme to cut and splice the genes. The fear, the lead researcher said in Space Daily, has been that this gene manipulation would cause unwanted mutations. Instead the team found that the very small number of mutations in the edited cells did not exceed the number in normal cells growing in the lab for the same length of time.

Blood cells

This is great news for CIRM, since eight of our Disease Teams—all of which have the goal of moving therapies into the clinic—use gene modification techniques. These include efforts to correct the genetic mutation that causes sickle cell disease and beta thalassemia.

Interview with Nobelist on stem cell potential. The Raw Story ran an interview with Nobel Laureate Martin Evans about the field he helped to create when he first isolated mouse embryonic stem cells in 1981. He won the Nobel in 2007 for later work in which he used embryonic stem cells to create specific gene modifications in mice. He said we are “just scraping the surface” in unlocking the potential of stem cells to change medicine. He also addresses various aspects of reprogramming cells to become different types of tissue and provides a bit of advice to young scientists: “You should not believe in all that you read.”

Keep your blood stem cells acting young. Blood stem cells, like most of the adult stem cells in our various tissues, become less adept at doing their job of replenishing our tissues as we age. A team at New York’s Mount Sinai has fingered the decrease of a specific protein in older stem cells as the culprit. That protein, SIRT1, was not a surprise as it has been implicated in other aging research. When laboratory animals eat a severely calorie restricted diet and live longer, SIRT1 is active at a higher than normal level. So, it makes some sense that low levels of SIRT1 would be associated with conditions of aging. The team now wants to see if increasing SIRT1 levels can put the kick of youth back into older blood stem cells. The web portal Hospital Newspaper ran a story on the research that the team published in Stem Cell Reports.

Or use a new way to create blood cells. If you can’t get your own blood stem cells to behave like vigorous youthful cells another option is to get some new one. The problem is many folks cannot find a matching donor and previous attempts to grow them from earlier stage stem cells have not worked. Using either embryonic or reprogrammed iPS type stem cells to try to grow large quantities of blood-forming stem cells has always resulted in immature cells that cannot make all the blood cells and don’t readily take up residence—engraft—in the patient. Researchers at Cornell Medical College may have solved this problem by growing the stem cells in a more natural environment. They grew them in a bed of cells like those that would have surrounded them in blood vessels in a developing fetus. The resulting cells engrafted in mice and produced nearly all the components of blood. They had a few lingering problems with creating the immune system’s T cells, but got much closer than previous work. Device Space picked up the medical school’s press release.

This goal of creating fully functional blood stem cells is sufficiently important but vexing to the research community that CIRM organized an international workshop on the topic. You can read the resulting whitepaper “Breaking the Bottleneck.”

Donors needed to power discovery. With federal support for research shrinking many institutions are relying more and more on donors to fund the research that leads to discoveries and eventually therapies. The New York based web publication Capital Playbook painted a picture of the deficit citing a 22 percent reduction in the inflation-adjusted budget for the National Institutes of Health since 2003. It goes on to quote senior scientists fearing the loss of a generation of scientists. A great comment came from my friend and former colleague David Scadden, co-director of the Harvard Stem Cell Institute. “They are seeing their senior mentors spending more and more time writing grants and going hat in hand. That’s not a good way to inspire the best and brightest.”

Don Gibbons

Argentina Soccer Star Pins his World Cup Final Hopes on Stem Cells

I suppose we should have expected it. Every time there is a big sporting event stem cells seem to come into the conversation. So it’s not surprising that the World Cup in Brazil, the biggest sporting event on the planet, was bound to somehow, in some way, involve stem cells. And it has.

Argentina’s speedy attacker, Angel Di Maria, suffered a torn hamstring in the game against Belgium. He was initially ruled out for the rest of the tournament but then came news that he was hoping to be able to play in the final – if his team made it, which they have – by getting a stem cell therapy.

Angel Di Maria: Photo courtesy Fanny Schertzer

Angel Di Maria: Photo courtesy Fanny Schertzer

Now, as often happens in instances like this, the reports have been light on specifics although there are some hints in the media that it might involve the use of stem cells taken from Di Maria’s own fat tissue or from his blood.

The web site Inside Spanish Football mentioned that another player, Atletico Madrid’s Diego Costa, underwent a similar procedure to try and recover from an injury before a recent championship game. The web site described it this way:

“The medical procedure is used to regenerate damaged cells using the patient’s own healthy cells, with the primary object being to reduce inflammation and repair the torn muscle tissue.”

Not surprisingly, because famous athletes are involved, the therapy is getting a lot of exposure in the media. The same thing occurred when Peyton Manning, the quarterback for another kind of football team, the Denver Broncos, got a stem cell treatment for a neck injury; and when Yankee’s baseball pitcher C. C. Sabathia underwent a stem cell treatment for a knee injury. We blogged about both of these instances.

The problem with the coverage is that the media typically does a good job of explaining what the therapy is designed to do, but then fails to mention that none of these therapies have been tested or proven to work in a clinical trial. It gives the impression that this is a routine therapy for an injury. It’s not. It is, in every sense, experimental. And therein lies the problem. While the treatment may be safe there’s also a chance it is not. While it may be effective to some extent, we really have no way of knowing.

Another confounding factor in all this is that alongside the stem cell therapy, Di Maria is also getting intensive traditional therapy – ice, kinesiology, electro pulse stimulation and some rehabilitation exercises in the swimming pool. So even if Di Maria does beat the odds and return in time for the World Cup Final, we really won’t know if it was the stem cells, the traditional therapy, or both that worked.

And that’s the real problem here. It’s not that a professional athlete is doing everything he can to be ready for the biggest game of his life – that’s to be expected – but that the media doesn’t dig a little deeper to see if there’s any evidence this approach could work. By failing to do that they leave the playing field open to other “clinics” to offer this same kind of therapy to anyone; clinics who will promote their treatment “as used by” and give the impression that if it helped Argentina win the World Cup, or at least come in second, then it can certainly help you bounce back from your injury.

So next time you read about a superstar athlete turning to stem cells for a miracle cure don’t assume that it will help them. The odds are it won’t. Sports are fun. But your health is nothing to play around with.

Before considering any stem cell treatment, we highly suggest looking at educational information for patients provided by the International Society for Stem Cell Research, the world’s leading stem cell research organization. Their printable, take-it-along Patient Handbook identifies questions any patient should ask. It would be a good idea to review answers with a physician you trust.

kevin mccormack