Tuesday, June 22, 2010

Diabetes type 1 and the Cure - What I feel Are the Most Promising

I first wanted to hear if from a child's perspective. Here it goes:

Mom: What do you feel is the cure for diabetes?        

Gabriel: What I had done.

Mom: What did you have done?

Gabriel: Stem Cells from my body.

Mom: How do you feel?

Gabriel: Good. I thank God. I just hope that my anteebuddies don't attack my new cells.

Mom: How about if one day your Endo gives you a sheet of paper and allowed you to choose one to cure you. Let's assume you were given these options. Which one would you choose?

1. Adult stem cell transplant (what you had done)

2. Artificial pancreas

3. Islet Cell Transplantation from a donor pancreas

4. Islet Sheet

5. Pig Islet Cell Transplant

6. Stem Cell Therapy Drug(from donor) via injections

I explained the above in child's terminology.

Gabriel: I like #1. What I had done. I know what to expect and it didn't hurt and it was safe. I am doing well.

Mom: Why not the others?

Gabriel: I don't know about pigs in my body. I may become one. I don't get it. I don't want # 2 because I already wear an insulin pump and I don't want more things on my body. I am full of holes and this is really not the cure. It's just something better than the pump. I don't know about the others.

Mom: How about #6.

Gabriel: It's something like #1, but it's from other people's body not my own. I want my own body to go back into my body. Why can't they do #6 with the person's own body? Oh, on #4.....I don't know if that sheet will melt or pop inside my body. Do I need to go back and get a new one? how do they get the sheet inside my body? Mommy, remember when we travel and we go through those bars at the airport and I take off my pump? Would the sheet inside my body make the security bar ring? and then I cannot take it off. How about when I go to the doctor and get X-rays will it explode the sheet? I don't think I like #4.

Mom: Good questions and I don't know the answers. I'm assuming it will be fine.

Mom: So, what are you going to do when you are cured?

Gabriel: I will close my eyes and look at the sky and raise my hands up really really high and thank God for no more insulin, no more counting carbs, no more backpack wherever I go! And then, and then, ummmm... I will have a bubble gum party. Yes! I will have a bubble gum party!

Mom: Who are you going to invite to the bubble gum party?

Gabriel: Everyone that wants to come.

Mom: Who will pay this big party?

Gabriel: hmmmmm..You and Dad. Maybe JDRF.




Research towards the cure for type 1 diabetes continues to advance.

I feel one of the most realistic promising one is the field of regenerative medicine, using adult stem cells.

This new field is rapidly evolving and is the next evolution of medical treatments. Regenerative medicine can: (1) regenerate damaged tissues and organs in vivo (in the living body) through reparative techniques that stimulate previously irreparable organs into healing themselves. (2) empower scientists to grow tissues and organs in vitro (in the laboratory) and safely implant them when the body is unable to be prompted into healing itself.

This revolutionary technology has developed therapies for previously untreatable diseases and conditions. Stem Cell therapy is one of which has recently evolved. There are several sources of stem cells that are being studied, such as adult stem cells, embryonic stem cells and induced pluripotent stem cells.

1. Adult stem cells are obtained from living bone marrow, blood, brain tissue, skin, and body fat. Other sources rich in adult stem cells are umbilical-cord blood and the placenta.

2. Embryonic stem cells are derived from human embryos. In order to harvest embryonic stem cells, an embryo must be destroyed. The following are remarks from President Bush and President Obama towards federal funding on embryonic research.

Remarks from President Bush opposing Embryonic Stem Cell Research in 2001 and 2006.

Remarks from President Obama lifting the Embryonic Stem Cell research band in 2009.

Research on embryonic stem cells requires the destruction of a living human being. In contrast, research on adult stem cells does not require the loss of life. I believe that research on adult stem cells is a legitimate field of study, well within the will of God.

Just recently, "The National Institute of Health's (NIH) Advisory Committee recommended withholding federal research funding for embryonic stem cell lines on Wednesday that were thought to be eligible with Obama's policy."

3. Induced pluripotent stem cells

Adult Stem Cell Therapy (Transplant)

Studies have shown that stem cell transfusions using adult stem cells helped type 1 diabetes by reducing their disease severity, possibly re-setting the immune system and slowing the destruction of their insulin-producing cells. Below are various procedures on these studies.

1. Adult Stem Cell Therapy - Autologous Stem Cell Therapy Transplant- no immunosuppression

I’m most familiar with the Adult Stem Cell Therapy, also known as Autologous Stem Cell Therapy, which means originating from your own body. This therapy is used to restore the function of organs and tissues and has proven to be safe and has treated many patients. Stem Cells can be harvested from many areas of the body including the bone marrow, fat, and peripheral blood. Once the cells have been harvested, they are sent to the lab where they are purified and assessed for quality before reintroduced back in the patient. Since the stem cells come from the patient, there is no possibility for rejection. Studies have shown that stem cells isolated from the patient have the ability to become different cell types and are capable of repairing damaged tissues.

My son had this procedure and I am fortunate to have had the opportunity to be able to share my experience and his results in this blog. It was as easy as 1,2,3! (1.) Bone marrow extracted from patient’s iliac crest (hip bone) (2.) Using a centrifuge, stem cells are separated from the bone marrow (3.) Implanted into pancreatic artery

I have seen great improvement in his blood glucose control and continue to see excellent results as far as his control and his insulin dosages. This procedure is a natural self-repairing process.

2. Autologous Nonmyeloablative Stem Cell Therapy (Transplantation) using high-dose immunosuppression

This procedure requires high-dose immunosuppression followed by autologous nonmyeloablative hematopoietic stem cell transplantation.

Some severe adverse effect has been documented.

3. Transfusion of Stored, Autologous Umbilical Cord Blood

This procedure showed potential benefits in a small pilot study.

4. Stem Cell Therapy using Drug

Adult stem cells have been studied for the past 10 to 15 years and large pharmaceutical companies are now wanting to get into this therapeutic area. I believe they are willing to put money in because they have realized there is enough safety and efficacy in this.

FDA has recently approved an orphan drug designation to Osiris therapeutics for a stem cell therapy. The product, Prochymal, is an intravenous formulation of adult mesenchymal stem cells (MSCs) that are isolated from the bone marrow of healthy young adult donors, thereby avoiding the controversy associated with embryonic and fetal cell sources. Cell culture allows the large-scale production of thousands of doses from a single donation.

This study is ongoing.

How about the autoimmune disorder that attacks the beta cells?

Although the Adult Stem Cell Therapies for type1 diabetes have shown promising results in studies, these treatments do not eliminate the original autoimmune disorder that triggers type-1 diabetes, so eventually the body may attack the new beta cells. However, because the cells come from your own stem cells, you could receive periodic treatments.

This autoimmunity must be overcome if researchers hope to use transplanted cells to replace the damaged ones.

Currently, there are no approved treatments for altering the rate of destruction of these critical islet cells, called beta cells. Researchers are working on protecting transplanted beta cells from the immune system attacks.

I have listed some studies focused on the immune system attacks.

1. The BHT-3021

This is designed to turn off the immune attack via BHT-3021 injections by Bayhill Therapeutics.

2. Encapsulation of islet cells

Encapsulation technology involves wrapping healthy cells in a capsule allowing them to be safely implanted into patients without the need for immunosuppressive drugs to prevent rejection.

Below are some encapsulation studies:

a. Encapsulation of islet cells using pig cells implant using DiabeCell by Living Cells Technologies.
           Company Announcement on DiabeCell Trial
           JDRF Funding

b. Encapsulation of Islet Cells using PTFE

Researchers have found a way to protect transplanted beta cells from immune system attacks by wrapping the cells in polytetrafluorethylene (PTFE). PTFE allows the cells to produce and release insulin while protecting them from a hostile immune system.

3. The Islet Sheet

This removable bio-invisible sheet, a bio-artificial pancreas, is a development led by Scott King, founding President of Cerco Medical. It has the size and appearance of a transparent business card. This thin alginate sheet protects transplanted islets without immune suppression drugs. The sheet can use islets from stem cells or animals. The Islet Sheet responds to changing blood sugar levels with the release of insulin in real time, mimicking the normal function of pancreatic islets. The Islet Sheet consists of live, functional islets contained within an immunologically protective coating made from a hydrogel polymer called alginate derived from kelp. This has the potential to replace the artificial pancreas, a system that integrates two current technologies, the continuous glucose monitor and the insulin pump.

4. Diamyd Vaccine

In early clinical trials, the Diamyd® vaccine has been shown to slow the immune attack in recent-onset type 1 diabetes.

The Cure?

I feel the adult stem cell therapy is one of the most promising and safest. With advances in stem cell research, the cure will be a gradual process building over the years. The techniques will improve incrementally and will become more effective with fewer adverse effects.

I look forward to a day in the near future when the two procedures, adult stem cell therapy and an immune system protection technique are combined to create a lasting, effective solution that will relieve patients of the daily burden of type-1.

Besides adult stem cell therapies, there are other studies towards a cure for type1 diabetes that sound also promising and my friend Kathy, has done an excellent job in summarizing which ones she feels are the most promising. She has lots of information and has documented her own personal experience of having an islet cell transplant. She has quite an amazing story to share.