http://www.sciencedaily.com/releases/2013/02/130207114422.htm

Type 1 Diabetes Cured in Dogs, Study Suggests

Feb. 7, 2013 — Researchers from the Universitat Autònoma de Barcelona (UAB), led by Fàtima Bosch, have shown for the first time that it is possible to cure diabetes in large animals with a single session of gene therapy. As published this week in Diabetes, the principal journal for research on the disease, after a single gene therapy session, the dogs recover their health and no longer show symptoms of the disease. In some cases, monitoring continued for over four years, with no recurrence of symptoms.

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Health & Medicine
•Diabetes
•Gene Therapy
•Personalized Medicine
•Genes
•Diseases and Conditions
•Hormone Disorders

Reference
•Blood sugar
•Vector (biology)
•Diabetes mellitus type 2
•Hyperglycemia

The therapy is minimally invasive. It consists of a single session of various injections in the animal's rear legs using simple needles that are commonly used in cosmetic treatments. These injections introduce gene therapy vectors, with a dual objective: to express the insulin gene, on the one hand, and that of glucokinase, on the other. Glucokinase is an enzyme that regulates the uptake of glucose from the blood. When both genes act simultaneously they function as a "glucose sensor," which automatically regulates the uptake of glucose from the blood, thus reducing diabetic hyperglycemia (the excess of blood sugar associated with the disease).

As Fàtima Bosch, the head researcher, points out, "this study is the first to demonstrate a long-term cure for diabetes in a large animal model using gene therapy."

This same research group had already tested this type of therapy on mice, but the excellent results obtained for the first time with large animals lays the foundations for the clinical translation of this gene therapy approach to veterinary medicine and eventually to diabetic patients.

The study was led by the head of the UAB's Centre for Animal Biotechnology and Gene Therapy (CBATEG) Fàtima Bosch, and involved the Department of Biochemistry and Molecular Biology of the UAB, the Department of Medicine and Animal Surgery of the UAB, the Faculty of Veterinary Science of the UAB, the Department of Animal Health and Anatomy of the UAB, the Spanish Biomedical Research Centre in Diabetes and Associated Metabolic Disorders (CIBERDEM), the Children's Hospital of Philadelphia (USA) and the Howard Hughes Medical Institute of Philadelphia (USA).

A safe and efficacious gene therapy

The study provides ample data showing the safety of gene therapy mediated by adeno-associated vectors (AAV) in diabetic dogs. The therapy has proved to be safe and efficacious: it is based on the transfer of two genes to the muscle of adult animals using a new generation of very safe vectors known as adeno-associated vectors. These vectors, derived from non-pathogenic viruses, are widely used in gene therapy and have been successful in treating several diseases.

In fact, the first gene therapy medicine ever approved by the European Medicines Agency, named Glybera®, makes use of adeno-associated vectors to treat a metabolic disease caused by a deficiency of lipoprotein lipase and the resulting accumulation of triglycerides in the blood.

Long-term control of the disease

Dogs treated with a single administration of gene therapy showed good glucose control at all times, both when fasting and when fed, improving on that of dogs given daily insulin injections, and with no episodes of hypoglycemia, even after exercise. Furthermore, the dogs treated with adeno-associated vectors improved their body weight and had not developed secondary complications four years after the treatment.

The study is the first to report optimal long-term control of diabetes in large animals. This had never before been achieved with any other innovative therapies for diabetes. The study is also the first to report that a single administration of genes to diabetic dogs is able to maintain normoglycemia over the long term (more than 4 years). As well as achieving normoglycemia, the dogs had normal levels of glycosylated proteins and developed no secondary complications of diabetes after more than 4 years with the disease.

Application in diabetic patients

There have been multiple clinical trials in which AAV vectors have been introduced into skeletal muscle, so the strategy reported in this study is feasible for clinical translation. Future safety and efficacy studies will provide the bases for initiating a clinical veterinary trial of diabetes treatment for companion animals, which will supply key information for eventual trials with humans. In conclusion, this study paves the way for the clinical translation of this approach to gene therapy to veterinary medicine, and eventually to diabetic patients.

Story Source:

The above story is reprinted from materials provided by Universitat Autònoma de Barcelona, via AlphaGalileo.

Note: Materials may be edited for content and length. For further information, please contact the source cited above.

Tags: 1, dogs, gene, therapy, type

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I wish I was a dog so I can have my diabetes cured. If I can't be a dog then I want to be a mouse and then I can surely have my D cured.

I struggle to keep my healthy skepticism from turning into cynicism. Sometimes I wish I were a dog, too.

The worst aspect of so-called cures is that it could keep a gullible person from doing what they need to do to take care of their D. Although I guess people would have to be majorly gullible to take a dog cure.

bantam and best tested the first insulins on dogs!

I don't know how to take these type of articles, with a little bit of hopeful skepticism might be the best way. I'm hoping there is something to it but I'm not holding my breath.

I also want to be a dog and have my D cured, but not just any dog, a cute lap dog maybe. One that has nothing to do but be cute and accept an occasional belly rub.

My Aussie Shepherd Mix dog was 5 years old when she got very sick.

She was vomiting, wouldn't eat, so we were at PetSmart getting her some new flavors of dog food, natural, healthy ones. She fell over at the checkout, literally!

A vet was in the store and we went to them. They found that she was bleeding from her bowels and kidneys! They sent me straight to UT Vet School with her. The school told me that she would probably not make it through the night, so we needed to say goodbye. I was heartbroken. I told her that we loved her dearly and that I knew she would do what she needed to do. They gave her blood transfusions and medicine, a powerful antibiotic.

She made it. Slowly, she regained her health. They never did figure out what had happened to her...they just let her come home with me about a week later, and she ate homemade chicken soup for a good long while until she returned to her strong beautiful self. She's eight now, and just pulled us around the block. Such a good girl.

Almost a year to the day after our dog was so sick, I was the next patient...at a human hospital, and they gave my family no hope at that time...I came out with a new diagnosis of type 1 diabetes.

This story makes me hope that there's a mystery to unfold here, and someone is tugging at the clues, unraveling the mystery strand by strand!

Hi Joni, what a lovely story! Thank you so much for posting it. I do think that medical research will indeed unravel a lot of mysteries.

I keep getting articles on promising sounding research from Australia, one day someone somewhere in the World will go Bingo, I have the cure! Or even if not a cure something that will alleviate the symptoms to some degree.

The wording of the article is interesting:
-minimally invasive
-simple needles
-injections introduce gene therapy vectors
-safe and efficacious
-widely used in gene therapy

We are still talking about this: the use of virus strategies on muscles cells to alter their DNA irreversibly. This will activate two sections of their DNA that are normally only active in beta cells. One section - the glucose sensor - will measure the blood glucose and will control the other section. This other section - the insulin blueprint - will produce RNA that will be translated to the hormone insulin. The approach of the cure is elegant and in its result comparable to the natural production - although there is no direct secretion into the portal vein like the beta cells do (risk of spikes). On the other hand the treatment is a one way ticket. We get much older than dogs and it will take long to really proof its safety or risk/benefit ratio. Despite of my remarks I really wish it will work!

They don't mention how the fact that we apparently have antibodies continuously killing off our beta cells might affect this type of treatment?

but from what Holger just said, the beta cells aren't needed. So it wouldn't matter if betas get killed. I guess we can wait and see

In more detail the autoimmune reaction is focused on the beta cells. The signature of the muscle cells for the immune system will not change after their DNA has been altered. Thus they will not be attacked.

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