Juvenile Diabetes Adult Stem Cell Cure?

The Journal of the American Medical Association has published a study - free online here - that describes successful treatment of 13 patients with their own stem cells. Some of the patients have been able to go without insulin or any other medications to control their diabetes.

15 patients with new onset "Juvenile Onset," "Insulin Dependent," or Type I diabetes received shots to stimulate production of their own bone marrow stem cells. Those cells were collected by "leukapheresis," a process where the blood is filtered to remove specific cells.

The bone marrow was killed and the patients received anti-thymocite antibodies to wipe out more of the white blood cells (from the thymus that might not be in the bone marrow.

Then, the patients received their own bone marrow stem cells.

During a 7- to 36-month follow-up (mean 18.8), 14 patients became insulin free
(1 for 35 months, 4 for at least 21 months, 7 for at least 6 months; and 2 with late response were insulin-free for 1 and 5 months, respectively).

Just as Dr. John Willerson of the University of Texas Health Science Center at Houston did a few years ago to explore the use of adult stem cells in the treatment of heart disease, Dr. Richard Burt of Northwestern University in Chicago went down to Brazil in order to perform the research. It was easier to receive permission from the local ethics board to use stem cell transplants - actually, an autologous bone marrow transplant.

The news reporters and some bloggers are criticizing the research for a lack of controls as well as the use of teen subjects.

Tell me - if your son or daughter were diagnosed tomorrow with insulin dependent diabetes, would you look into a plane trip to Brazil?

Texas Politics, Bias and Bioethics

"All politics is local," is a quote attributed to - and the title of a book co-authored by - the late, former Speaker of the House, Tip O'Neill.

The lesson seems to be one that Texas State Representative Juan Garcia, D-Corpus Christi, learned well. It doesn't hurt to stack the deck in your favor, either.

Evidently, the Representative held a meeting at a church in Corpus Christi, Texas and only invited the people that agreed with him to present arguments on stem cell research to a local group of clergy.

Read "stem cell research" to include embryonic stem cells from human embryos.

I'm certain that the Representative knows the names of groups who could have directed him to people like me who could make the case for the basic science and human rights issues inherent in "the stem cell debate." (Okay, I did say, "people like me.")

Instead, the clergy evidently found themselves faced with advocates who do not believe that research in stem cells and regenerative, cellular medicine can proceed without embryonic stem cells. Advocates who include representatives from State Universities and from the "Texans for the Advancement of Medical Research," a group dedicated to the advancement of destructive embryonic stem cell research and cloning.

A similar one-sided, and self-serving argument was made this week by Tom Okarma, the president of Geron, one of the biotech companies that holds the patents on human embryonic stem cells.

This, in spite of proof such as that given to the House State Affairs Committee last Monday, of children who are alive because of stem cell transplants from cord blood. And the hope of so much more from readily available umbilical cord cells: including functional liver tissue, lung cells, nerve cells and pancreatic islet cells.

Texas, Adult Stem Cells, Multiple Sclerosis

Opexa is a division of Pharmafronteirs (or it's the other way around, I'm not sure) which is based at the Woodlands, near Houston, Texas.

The company specializes in cell therapies, based on adult stem cells and the controlled manipulation and replication of adult cells.

Multiple sclerosis (MS)is a disease that causes the loss of the myelin around nerves. Think of myelin as insulation that speeds the transmission of nerve signals. When myelin is lost, nerve signals can't go where they're needed, as fast as they are needed. People end up weak, with tremors, and the lack of balance, loss of coordination and the loss of the ability for the muscles that enable us to breathe and cough to function.

We know that MS is a sort of autoimmune disease in most cases. The cells that are supposed to fight infection and keep abnormal or injured cells that can cause cancer actually decide that the myelin needs to be destroyed.

For over 4 years, Pharmafrontiers or Opexa has been running a series of experiments using T cells - the specialized white blood cells that mature in the Thymus and which are supposed to kill foreign cells, like bacteria or cancer cells.

The company has a technique for isolating the patient's specific T cells that attack their myelin, growing them in the lab until they have millions, and then treating them so they can't multiply. The treated cells are then injected under the skin of the patient, and the body really notices the cells, and uses all the immune system to attack them - and all or most of the T cells in the body that act like them. So the myelin is not destroyed anymore - or at least not as fast.


Opexa are now in Phase IIb - meaning that they know it's safe to use in people (Phase I tests) and are finding out more about how much is needed and who can be helped.

There's a great first-person story about someone who is being treated as part of the experiment at "I Have MS."

For a couple of very pretty videos that explain all this much better than I ever could - and the press release by the company about the Phase IIb trial -- take a look at the Opexa site, at this page.

Added:

Opexa is selling the treatment as "Tovaxin™" - a vaccine.

That's how vaccinations work, by the way. Our bodies are convinced to make antibodies and specialized white blood cells to kill or destroy the foreign bacteria, virus -- and someday, cancer and all sorts of cells that inappropriately make those antibodies and attacks against our own normal cells, treating them as though they are damaged or foreign. As long as our bone marrow is healthy, we seem to be able to make a nearly unlimited number of those white blood cells (there's also some "depots" or reserves out in the lymph nodes and in the liver, spleen and the gut lining where the cells lurk and wait for their chance to multiply and fight disease, evidently). And I think this is how "allergy shots" work.

Review of Umbilical Cord Pancreas Cells

I've read the unproofed draft (their English is much better than my Korean) of "Induction of human umbilical cord blood-derived stem cells with embryonic stem cell phenotypes into insulin producing islet-like structure" by B. Sun, et. al. (Biochem. Biophys.Res. Commun. (2007), doi:10.1016/j.bbrc.2007.01.069)

The authors do not tell us how much of the insulin-secreting cells or colonies of cells they were able to obtain. However, they do report that at one stage, the cells exhibit Oct-4 and "stage-specific antigen 4"(which are characteristic of embryonic stem cells). After inducing umbilical cord cells to revert to these embryonic-like stem cells, they then followed a previously reported protocol for inducing embryonic stem cells into beta islet cells. Colonies of cells grew in islet-like structures and were positive for both insulin and C-peptide. The C-petide is indication that the insulin came from the cells, and not from an artificial source in the nutrients in which the cells were grown.

Time will tell whether the photos are real, whether the results can be replicated in other labs, whether there are enough babies born and cord blood to be obtained to match all the diabetic patients in the world, whether it's possible to transplant immune-matched islet cell structures into humans, and whether those islet cells will survive and function in the recipients. I wonder whether the manipulated cells will be genetically stable or whether they will be prone to die or mutate.

On the other hand, if as in hematopoietic stem cell umbilical cord blood transplants, the cells do not have to be as closely matched in order to be accepted by the immune system, and if these cells persist as long as maternal stem cells have been reported to do, then this report brings us the most promising news of a possible treatment if not cure for diabetes that I've seen in all these years of stem cell reports.