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hans keirstead on embryonic stem cell research

Hans Keirstead is the scientists behind the first embryonic stem cell clinical trial in the US. He explains the hurdles that research faces to becoming a viable medical therapy.

Hans Keirstead used embryonic stem cells to help paralyzed rats walk again. His research is the basis for the first FDA approved clinical trial for the use of embryonic stem cells (ESC) - currently underway by Geron and aimed at treating spinal cord injuries. After years of controversy in the first part of the decade, ESC trials have finally started on the path that may let them deliver on the vast promises of stem cell enabled medicine. Yet we have already seen how autologous stem cell therapies (those which use a patient’s own cells) are becoming available in the U.S and all over the world. Why the hold up on ESC treatments? Autologous therapies are part of the medical practice of individual doctors, given to their individual patients. Geron’s clinical trials hope to usher in a new wave of globally used drugs and procedures. The rigorous science needed to obtain FDA approval for such widespread treatments is not easily achieved, but many still lament the slow process. To all of us wondering why ESCs are not yet available in every hospital across the world, Hans Keirstead has an explanation. He doesn’t make an impassioned plea, or take a rhetorically defensive stance. In just 5 minutes Keirstead walks us through the fundamental hurdles that scientists face as they try to bring ESC therapies to fruition. Everyone who wants an intellectual and scientific explanation of stem cell research should watch the video below.

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advanced cell technology FDA embryonic stem cells

ACT gained FDA orphan drug status for its embryonic stem cell derived product MA09-hRPE.

Massachusetts based biotech company Advanced Cell Technology recently announced that the FDA has granted orphan drug status to MA09-hRPE – an embryonic stem cell derived treatment for a specific form of blindness (Stargardt’s Macular Dystrophy). Orphan drug status is targeted to those therapies which are designed to treat fewer than 200,000 Americans and gives ACT access to tax credits, grants for clinical trials, and a seven year exclusivity to market MA09-hRPE. This is the first such FDA approval for an embryonic stem cell derived therapy and ACT plans on using the orphan drug status to accelerate clinical testing. While Advanced Cell Technology has something of a checkered past, this recent FDA status could signal not only an approaching success for the MA09-hRPE treatment, but also a promising advancement in the company’s goal to pioneer new forms of regenerative medicine.

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by Aaron Saenz on November 3rd, 2009
By color coding stem cells, reserachers were able to isolate those that would form different parts of the heart.

By color-coding stem cells, reserachers isolated those that would form different parts of the heart.

It’s Alive! Researchers at Harvard University and Massachusetts General Hospital have succeeded in taking embryonic stem cells from mice and growing cardiovascular tissue. The research team, led by Dr. Kenneth Chien, believes that a similar process may one day serve to repair cardiac damage in humans. The work was recently published in the journal Science. You can see the mouse heart cells beating at different speeds in the video from Boston.com after the break.

Cardiac injury is some of the most difficult damage to heal in the body. When the heart undergoes massive damage from a coronary, you have few options – replace broken parts, add a pacemaker, or get a whole new heart. The work done by Chien and his team focuses on creating a new way to repair tissue damage. Instead of adding in mechanical parts, or finding a donor organ, stem cells may be used to replace and heal the damaged cardiac tissue. Eventually, those patients that develop a myocardial injury could have pluripotent stem cells harvested from their skin, marrow, or fat which would then be introduced into the heart via injection. No open heart surgery, no pacemakers, just stem cells and a needle.

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Artificially created blood? That's for real. Vampires? Yeah, not so much.

Artificially created blood? That's for real. Vampires? Yeah, not so much.

Ask any vampire and she’ll tell you, when it comes to blood there’s nothing like the real thing. But as everyone who donates knows, hospitals and blood banks always seem to have dangerously low supplies. So scientists have gotten around to finding ways to make new blood, or something like blood, that doesn’t have to come from humans but that can still supply human demand. The new blood comes in two flavors: blood substitutes (without red blood cells) and synthetically produced blood (derived from stem cells). While it looked like blood substitutes could be the dominant force in the market, that is quickly changing. Why? Well, it turns out that synthetic blood is less likely to kill you.

A recent study published in the Journal of the American Medical Association (JAMA) and led by Dr. Charles Natanson has revealed that five major brands of blood substitute were increasing the likelihood of heart attacks and death. This has devastated interest in blood substitutes and bankrupted several companies that hoped to produce it. Synthetic blood is now the future vampire’s soup du jour.

No matter which substance ultimately triumphs (and both may still succeed), natural blood donations may eventually become things of the past. Why worry about diseases, spoiling, and matching blood types when an endless supply of near perfect blood could be available. Hemophiliacs to wounded soldiers, everyone could end up enjoying a new kind of blood.

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Traditional media outlets have been tripping over each other this week with sensational titles such as “Chinese Scientists Reprogram Cells to Create Mice” to describe the work of Chinese stem cell researchers.  Indeed, the study by hot shot Chinese stem cell researcher Qi Zhou of the Chinese Academy of Sciences in Beijing is notable, but not for the reasons most headlines would make you believe.

mouse-stem-cellsFirst, lets set the record straight.  Chinese scientists did not create an entire functioning mouse simply by reprogramming some mouse skin cells.  What they did do was reprogram mouse skin cells back into their more versatile, pluripotent embryonic state and inject them into an already healthy early stage mouse embryo.  The embryo, now partly its original self, and partly augmented with the foreign cells that were injected, was able to continue to grow normally into a fully mature, reproductively viable mouse.  What is the point, and why do we care?

The first point of interest here is that the study joins a  growing mountain of studies that show that mouse and even human skin cells can be reprogrammed into pretty much anything.  In this particular study, skin cells from a mouse were essentially reprogrammed into embryonic stem cells.  These reprogrammed skin cells were injected into an early stage embryo and then multiplied and mutated into all of the many different types of cells and tissues required to make a mature mouse.  In the coming decades this type of research could unlock the ability to take your own skin cells and create any cell, tissue, or organ you need for your body without any fear of rejection by your immune system.  Immortality anyone?

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If you like controversy there’s nothing that will bring you more joy than yelling “stem cell research” in a mixed crowd and then sadistically sneaking out of the room. The debate over the use of the extremely potent little cells has led to massive regulation in all realms of stem cell transplants in the United States. That’s why stem cell research for diabetes has been centered in Brazil, and why your dog or horse can receive the newest stem cell treatments now, while you’re likely to have to wait years or decades.

C'mon girl, help me get a stem cell treatment!

C'mon girl, help me get a stem cell treatment!

What are these treatments promising? According to anecdotes, the results are amazing. Old dogs with bad hips frolic like puppies. Race horses with injuries come back to become world class winners. One such racehorse, Be A Bono, won 16 out of 24 starts, earned more than 1.3 million in prize money, and was the 2004 World Champion Quarterhorse. All after a stem cell treatment. The success stories with dogs are equally remarkable, if a little tinted by emotion; check out the video from Vet-Stem after the break.

Most animals that have been treated with stem cells suffer from joint ailments. Damage to cartilage, tendons, ligaments, or arthritic inflammation top the list. Stem cells are seen as a way to provide almost magical regenerative healing to combat these ailments. The process is actually pretty simple. Rather than embryonic stem cells, adult stem cells are used. These adult stem cells are harvested in a veterinary office from fat cells in the animal, and then sent to a lab. Processing separates out the stem cells from other cells, and a concentrated dose is sent back to the vet (The turn around time for processing is only a day). The adult cells are then injected into the animal in the area that needs regeneration.

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The Short:
News outlets are reporting that researchers from Advanced Cell Technology, Inc have been able to differentiate human embryonic stem cells into red blood cells. This could be a major breakthrough in mankind’s quest to find a safe, unlimited source of blood that can be used for blood transfusions and other medical needs.

red blood cells

red blood cells

Currently the world’s blood supply is obtained from human donors in a system that is short on supply and fraught with complications related to sterilization, contamination, disease, storage life (typically less than 42 days), and collection logistics.

The Long:
First off, one can’t help but be a little skeptical here because of the financial condition of Advanced Cell Technology, which reportedly is on the brink of bankruptcy. Their website appears not to have been updated in several months, perhaps symbolizing the malaise that is happening over there.

Let us hope that this research truly is legit, however, because it would be a fantastic breakthrough for mankind. Other researchers have been able to derive red blood cells in the past from sources such as cord blood and bone marrow, but these sources are still donor limited. They are also rarely of type O(-) which is the universal blood type that virtually every person can accept. Embryonic stem cells can be multiplied infinitely, allowing for the generation of unlimited blood supply and theoretically can be developed into type O(-).

This breakthrough is still nowhere near to producing a clinical trial and has many technical hurdles to overcome, so don’t expect anything to come of it for many years. Still, it is exciting to see a breakthrough that might be taking us that much closer to an improved blood supply. Now, if someone could just make us a respirocyte then we would really be in business!

The research paper was published in the journal Blood.