Three-million Americans have type I diabetes, a disease where the immune system stops the pancreas from making insulin. Patients rely on daily blood sugar checks and insulin injections to survive. But now, there's hope on the horizon. Researchers are conducting the first study to look at embryonic stem cells for type-one diabetes.
Jeremy Pettus, MD, Associate Professor at the University of California, San Diego has checked his blood sugar every day, several times a day, for the past 20 years.
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Dr. Pettus told Ivanhoe, "I figured out the other day, I think I've checked my blood sugar somewhere in-between 40-50,000 times in my life."
Jeremy has type I diabetes. His pancreas doesn't make insulin, so he has to inject his own.
"It's tough you know. It's not any easy disease," he explained.
But Jeremy isn't just a patient with the disease; he's also a doctor who wants to cure it.
Dr. Pettus said, "It would be absolutely life-changing."
Jeremy and Robert Henry, MD, Endocrinologist at the University of California, San Diego, are conducting the first study in humans to see if embryonic stem cells can help patients with type I diabetes make insulin.
Dr. Henry told Ivanhoe, "I think this is the most exciting thing that's happened!"
The stem cells are grown in the lab and programmed to develop into islet cells — the special cells in the pancreas that normally produce insulin. Then they are placed in a special capsule to prevent immune rejection.
"Then that's placed underneath the skin in patients with type I diabetes" Dr. Henry explained.
In animal studies, the stem cells secreted insulin and regulated blood sugar levels. Researchers are hoping for the same in humans. They are studying 40 patients in a phase one safety trial. If it works, it could change millions of lives, including Jeremy's.
Dr. Pettus said, "It would be the greatest thing ever."
Type I diabetes typically affects children and young adults, but older adults can also be diagnosed with the disease. For more information on this clinical trial, log onto ivanhoe.com.
Contributors to this news report include: Cyndy McGrath, Supervising Producer; Marsha Hitchcock, Field Producer; Cortni Spearman, Assistant Producer; Jamison Koczan, Editor and Rusty Reed, Videographer.
BACKGROUND: Type 1 diabetes occurs when the body does not produce insulin. Doctors may also refer to type 1 diabetes as insulin-dependent diabetes, juvenile diabetes or early onset diabetes. Type 1 diabetes accounts for about five to ten percent of diabetes cases and is usually diagnosed in teens and children. It is caused by a lack of insulin as a result of the beta cells of the pancreas being attacked by the immune system. Type II diabetes is far more common and occurs in about 90 to 95 percent of diabetes cases and is caused when the body becomes resistant to insulin or simply does not produce enough insulin. In some cases, those with type II diabetes have plenty of insulin but their bodies are unable to use it properly, often as a result of an unhealthy lifestyle. There is no cure for type 1 diabetes but it can be managed with proper treatment. (Source: http://www.mayoclinic.org/diseases-conditions/type-1-diabetes/basics/definition/con-20019573
SIGNS AND SYMPTOMS: These may be the first signs someone with type 1 diabetes may experience:
· Extreme thirst
· Hunger
· Feeling tired all of the time
· Blurry eyesight
· Numbness or tingling in your feet
· Weight loss
· Frequent urination
Symptoms differ depending on if your blood sugar is high or low.
(Source: http://www.nlm.nih.gov/medlineplus/ency/article/000305.htm)
NEW TECHNOLOGY: The UC San Diego Sanford Stem Cell Clinical Center has launched the first ever human Phase I/II clinical trial of a stem cell derived therapy for patients with type 1 diabetes. Jeremy Pettus, MD, Assistant Professor of Medicine in the Department of Endocrinology said, "The stem cells are perpetually grown in a laboratory. The original cells are taken one time from an embryo and are programmed to develop into islet cells." These islet cells are the cells that produce insulin. Although promising, this research is in a safety trial which is the first step of many for approval.
FOR MORE INFORMATION ON THIS REPORT, PLEASE CONTACT:
Jacqueline Carr
619-543-6427
If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Marjorie Bekaert Thomas at mthomas@ivanhoe.com
Robert Henry, M.D., Endocrinologist at UCSD is working on a stem cell transplant to enhance the care of Type I diabetes.
Interview conducted by Ivanhoe Broadcast News in November 2014.
There has been a lot of work on treating diabetes, but nothing to stop diabetes. Is there anything out there to really cure it?
Dr. Henry: It's probably good for me to make it clear there are many forms of diabetes. By far, the more common form is Type II diabetes. There are many medications that treat it, but there is no evidence or signs of impending cure or prevention. What we're talking about today is Type I diabetes. Type I is quite different in its underlying cause than Type II. Type I is due to an absolute or near absolute loss of beta cell function, which are cells in the pancreas that produce insulin. They are destroyed by an autoimmune process which destroys the cells in the pancreas. These specific cells in the pancreas lead to almost complete, if not absolute complete, absence of insulin secretion.
Does that usually happen in childhood?
Dr. Henry: I believe the mean age is about twelve. Sometimes, you can get new onset in babies or young children, and similarly you can get it in somebody seventy five or eighty, but it is uncommon. The vast majority occurs between the ages of about six and eighteen years of age and then less as you take the years out.
You are now working with stem cells. Is this the first time the stem cells have been used?
Dr. Henry: Yes. This is the first time that they've been used in Phase III studies. There have been one or two previous efforts at stem cells in Europe, but have not been part of a registration program or a preregistration program. In other words, a systematic approach to addressing whether or not stem cells would work. Most of them have been in one or two patients to see whether it was feasible at all.
Why do you think stem cells would work?
Dr. Henry: We all have stem cells and we all come from stem cells. We start from two cells, the mom and dad, and those cells do what we call, differentiate. They develop into different forms of cells. Some are for the skin or for other tissues. In the case of trying to produce cells that are functional, you take cells that are early stem cells and encourage their differentiation, or their development into specific cell types. In this case, they took early stem cells and used them to push towards developing islet cells. Islet cells are beta cells which produce insulin as well as two other hormones known as glucagon and somatostatin.
Are you using skin stem cells?
Dr. Henry: Yes. Epidermal stem cells and embryonic stem cells that have been encouraged to differentiate into islet cell types. Again, the islet cells are the parts of the pancreas where insulin and other hormones are produced.
For this clinical trial, are you using the person's own stem cells?
Dr. Henry: No. We are using those that are derived from embryos.
How does that work?
Dr. Henry: Those cells are taken and harvested. I'm not privy to the fine details. Those are done by the companies, Viacyte, but they're in differentiation which is the development into specific cell types. In this case, insulin producing cells is initiated but the cells are not fully developed at that point.
Is this done through an injection or an infusion?
Dr. Henry: Good point. One of the big problems with Type I diabetes is it's an autoimmune disease which means the body recognizes the beta cells that are insulin producing cells of the pancreas as foreign. It sends out cells to destroy those cells. If you tried to just input cells into people, any insulin producing cells, they're immediately destroyed unless you give the patient very powerful drugs to prevent rejection.
Do they do the same if you had a kidney transplant?
Dr. Henry: Yes, with any tissues whether it's stem cell or non-stem cell. In the case of Type I diabetes, the disease is characterized by self-recognition of their own tissues and the body destroys them for unknown reasons. This has been the work of many, many people for decades to try and figure out exactly why. We're getting closer to that answer, but we're still not there. We're taking insulin producing cells that are not fully developed and putting them into Type I diabetic patients and they're rejected right away. That rejection can be minimized or perhaps prevented by immune rejection therapy or therapy that prevents rejection or reduces rejection.
Would the anti-rejection drug be an infusion?
Dr. Henry: No. This company has developed the process of taking the so-called pancreatic embryonic epidermal cells and differentiating them. They developed a device known as the Encapta, which houses the cells and then that's placed underneath the skin in patients with Type I diabetes. The concept is that this Encapta device allows hormones out and other substances in and out, but not the cells that destroy a pancreatic islet cell. It's sort of immunity from destroying those cells.
How many patients do you have in this trial?
Dr. Henry: The initial proposal is for a total of about forty patients broken into two different groups. The first group will be between three and six subjects with Type I diabetes and will all be conducted at UC San Diego. Once those first three to six are completed, the data will be evaluated and submitted to the FDA who will then hopefully approve the remaining thirty. Those would occur at four or five other sites throughout the United States.
This is a Phase III trial, right?
Dr. Henry: In terms of development, it doesn't get to Phase III yet, but a Phase I-II. Our hope is that it's safe and tolerable, but ultimately that it starts to show some evidence of secretion of insulin. Our major goal is to see if, at least in the relative short-term, the implantation of this Encapta device starts to produce some insulin and it's not being destroyed by the natural autoimmune process that occurs in Type I diabetes.
Do you have any specific numbers for success?
Dr. Henry: No. This has been published, but I don't have the exact number. There has been evidence that they do produce insulin in animals. But, remember these animals don't have Type I diabetes. You can make models that re-create Type I diabetes, but only humans get the classic Type I diabetes. So, although we know what happens in animal models and in culture, in cell cultures humans are completely different.
Does the immune system stop attacking the insulin making cells?
Dr. Henry: My guess would be that inate system, inate meaning being whatever that natural system is that leads to the destruction of islet cells or beta cells in Type I diabetes, is still intact and will be intact but won't be able to attack those cells in the Encapta device. That's our hope and I think there's reasonably good experimental evidence that they'll be successful.
Could a person live with just one stem cell transplant and not have to take insulin anymore with this safe house?
Dr. Henry: I think that's unlikely. It's more likely that the initial implants will last maybe nine to twelve months and then have to be replaced. But, that's based on animal data. This is the first time in humans, so we'll know that information.
Where on the body is it transplanted?
Dr. Henry: It's transplanted in the back in the flank region. That's based upon factors that the company Viacyte has determined would be the safest and least likely to get traumatized.
How big is it?
Dr. Henry: These devices are small. They are about an inch by two inches or a little more. Patients will have several of these Encapta devices placed in them. However, it depends on how the study progresses to know how many they'll get, but that's just going to be determined over time.
Is it almost like an implanted insulin pump that you don't have to worry about?
Dr. Henry: No. An implanted insulin pump doesn't regulate itself. One of the things that is currently being developed is an insulin pump that senses what the glucose is and then responds appropriately. There are still some obstacles to overcome. They're trying to work it so that the pump can sense some of the glucose and then send the insulin out in response to that signal, but they're not there yet. The difference with this implant will be that these cells should sense the prevailing glucose and produce at least some insulin in response to that. We don't know how much insulin will be produced. That's something that we will learn over the next year or two.
Is this one of the more exciting things you've seen come to diabetes patients?
Dr. Henry: I think that this is the most exciting thing that has happened. We were overwhelmed when the press release came out with people calling and volunteering to participate. I mean overwhelmed.
Is this a hard disease to live with?
Dr. Henry: Things have gotten so much better. I've had thirty years dealing with people with Type I and Type II and other forms of diabetes. But, Type I is with you every minute. You can imagine if you're a mom or dad and your child has diabetes, you have trouble sleeping at night because you're worried about your child. It's with you every step of the way and the disease can be very difficult to control. Obviously, all kinds of developments have occurred in the last two to three decades that have been phenomenal and have really made life better for people with Type I diabetes. I still think if you were ten years of age when you got Type I diabetes and are now thirty, you've had twenty years of this disease and are looking for a break. You're looking for something that's going to make your life more easier and less stressful and maybe take a step back a little bit. Not that I'm encouraging people to step back from their disease, but I can see if you have to keep an eye on everything so frequently and so intensively, that the potential for some kind of therapy that helps with a cure is going to be very important.
Is there a lot less risk involved with the stem cell transplant over use of medications?
Dr. Henry: I can't speak for all stem cells, but these stem cells are at least encased. They're in a safe house and that makes it less likely, not impossible, that they will get outside of that device. These stem cells have the capability of developing in to all types of tissues under the wrong condition. But, this is a special device that hopefully will not allow the cells out and hopefully will not allow any of the detrimental cells in. That's how it's been designed.
Is there anything else you would like to add?
Dr. Henry: Any improvement in insulin secretion is a major benefit to people with Type I diabetes. The closer to normal secretion, regulation, response to changes and fluctuations, the better it will be. The concept is, why would you want to make a cure for a disease when a lot of people benefit from therapies of that disorder. That's against the grain in medicine. There are many, many diseases that most care providers and physicians are dedicated to making sure we can rid people of. In the case of Type I diabetes, I honestly hope that we make some kind of dent. In terms of therapy, I think the advances have been marvelous. But, in terms of a cure, I hope this disease ultimately is cured.