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Scientists use gene therapy to cure dogs of type 1 diabetes

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February 9, 2013

Led by Fàtima Bosch (fifth from left), a University of Barcelona research team has cured d...

Led by Fàtima Bosch (fifth from left), a University of Barcelona research team has cured dogs of type 1 diabetes using gene therapy (Photo: UAB/Pierre Caufapé)

Researchers from the Universitat Autònoma de Barcelona (UAB) have claimed a first by successfully using a single session of gene therapy to cure dogs of type 1 diabetes. The work has shown that it is possible to cure the disease in large animals with a minimally-invasive procedure – potentially leading the way to further developments in studies for human treatment of the disease.

The researchers, led by Fàtima Bosch, showed that after only a single gene therapy session the dogs no longer displayed symptoms of type 1 diabetes. In some of the cases, monitoring continued over a four-year period with no recurrence of the disease. The same team has previously tested the therapy on mice, but these recent and highly positive results are, as Fàtima Bosch says, “the first to demonstrate a long-term cure for diabetes in a large animal model using gene therapy.”

Using simple needles common in cosmetics treatments, the single session consisted of various injections in the animal’s rear legs in what is said to be a safe and stress-free procedure. The injections introduce gene therapy vectors with two objectives – firstly, to express the insulin gene and secondly, to introduce the enzyme glucokinase.

Glucokinase is an enzyme which regulates the uptake of glucose from the blood. When both genes function in unison they work as a kind of “glucose sensor” that reduces diabetic hyperglycemia (the excess of blood sugar associated with the disease) by automatically regulating the glucose uptake.

The study highlights the safety of gene therapy mediated by adeno-associated vectors (AAV) in diabetic canines. These vectors, derived from non-pathogenic viruses, are commonly used in other gene therapies and have claimed success in the treatment of several other diseases.

Over the long term, the dogs that were treated displayed good glucose control when fasting and after eating, and also after exercising – which is an improvement on dogs that receive daily injections of insulin. No occurrences of hypoglycemia were recorded. Adding to this, the dogs treated with adeno-associated vectors maintained good body weight and did not develop secondary complications.

As there have been numerous clinical trials where AAV vectors have been introduced into skeletal muscle, the strategy applied in this research is certainly valid for clinical application in a wider sense. Further studies and development of the treatment should lead to veterinary trials, which may in turn supply key information for trials with human diabetes sufferers into the future.

Source: UAB

8 Comments

Interesting but I want to monitor the offspring for several generations before trying it on humans.

Slowburn
9th February, 2013 @ 09:52 pm PST

Yay for bioengineering!

www.youtube.com/watch?v=6cBf-EGahvs

Geo Max Lemay
10th February, 2013 @ 04:32 am PST

previous human trials of gene therapy suffered setbacks because of gene therapy induced cancer in some patients caused by the use of AAV. So, we are still at square one as far as therapy in humans is concerned

ugosugo
10th February, 2013 @ 04:54 am PST

My 7 year old daughter has type 1, to see her inject herself 5 times a day and prick for her blood test upto 10 times makes me weep. We have to monitor everything she eats and deal with mood swings when her blood sugar is out of wack. A safe cure can't come fast enough !

John Findlay
10th February, 2013 @ 03:25 pm PST

John Findlay,

Help from another diabetic I saw on YouTube: http://www.youtube.com/user/DiabeticDanica?feature=watch

There are pros and cons to resolve the emotional impact. Using Insulin Pen's can help with smaller needles and easier portability: http://www.novolog.com/insulindiabetes/novologflexpen.aspx . Using Levemir or similar can provide a "base" 24-hour insulin level that allows for other shots for meals and high sugars only (you still need a regular shot for meals though). http://www.levemir.com/Levemir/Levemir.aspx . (Levemir is not for meals nor high sugars, remember). The next is expensive, and I understand the high cost being a barrier, but having to take so many shots gave reason enough to turn into taking one insertion every 2 to 3 days by turning to an insulin pump. The pump method has its own benefits and drawbacks from needles, like the need for better discipline, but I sure thought it took the emotional impact down to every three days rather than every 3 hours. It gets easier over time and becomes a part of life--less emotional issue later. Of course you still monitor your blood as usual or provide a continuous glucose monitor that has to be replaced every 4-6 days with a few finger pricks for calibration. OK, so here are some links for you:

Animas Pump: http://www.animas.com/

tslim Pump: http://www.tandemdiabetes.com/products/t-slim/

dexcom CGM: http://www.dexcom.com/dexcom-g4-platinum

Good perspectives!!!!:

m1st03
11th February, 2013 @ 09:50 am PST

For the nay-sayers another 200 years of "clinical trials" should just about give us enough data on safety and efficacy. In the mean time, lets allow well informed patients to determine whether they would prefer their current disease or risk the occurrence of another, hopefully lesser, condition; for which, we can presume, another gene therapy will be along shortly.

Mirmillion
11th February, 2013 @ 10:08 am PST

Hi All, Does anyone out there know if the study tracked specific immune system responses to the therapy, or if it was entirely inferential? That is, the dogs are symptom-free, ergo their immune systems did not destroy any resultant insulin-producing bodies. Any endocrinologists out there who'd like to comment? Thanks, Mac (lifelong type 1)

Mac McDougal
11th February, 2013 @ 10:29 am PST

As a Type 1 diabetic myself, I've been trying to keep up on some of the promising research going on out there. To me, the most promising of all is the research being done by Dr. Denise Faustman at Massachussets General Hospital.

You can check out the details of her research at http://faustmanlab.org

The research is currently in Phase 2, well on its way to FDA approval (hopefully!), and they need all the help they can get.

SlowburnAZ
11th February, 2013 @ 02:06 pm PST
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