Replace insulin with betatrophin?
A new hormone promises a breakthrough in the treatment of type 2 diabetes
NanoNewsNet based on HSCI materials: Potential Diabetes BreakthroughScientists at the Harvard Stem Cell Institute (HSCI) have discovered a hormone that gives hope for a significant increase in the effectiveness of the treatment of type 2 diabetes, a metabolic disease that affects hundreds of millions of people worldwide.
Researchers believe that this hormone may play a role in the treatment of type 1 diabetes, also known as juvenile.
In mice, a hormone called betatrophin accelerates the proliferation of insulin-secreting beta cells of the pancreas by more than 30 times. New beta cells produce insulin only when the body needs it, thereby making possible the natural regulation of its synthesis, which significantly reduces the likelihood of diabetes–related complications - the leading causes of limb amputation and non-genetic vision loss.
The researchers who discovered betatrophin, HSCI co-director Doug Melton, PhD, and postdoctoral fellow Yi Peng, warn that much more needs to be done before this hormone is used as a therapeutic agent. But the results of their work have already attracted the attention of pharmaceutical companies.
"If it can be used in the clinic," Dr. Malton predicts, "it will ultimately mean that daily triple injections of insulin can be replaced with one injection of our hormone once a week or once a month, and perhaps even once a year."
Type 2 diabetes, a disease associated by scientists with the obesity epidemic that has engulfed highly developed countries, is usually caused by a combination of excess weight and lack of exercise. During the development of the disease, patients gradually lose beta cells of the pancreas, and with them the ability to produce an adequate amount of insulin.
"Our idea is pretty simple," Dr. Malton continues. "We provide this hormone to a patient with type 2 diabetes, more of his own insulin-producing cells are formed in his body, and this slows down, if not stops, the progression of diabetes. I have not yet seen a treatment method that would cause such a huge jump in beta cell replication. Experiments were conducted on mice, but, of course, we are not interested in the treatment of diabetes mellitus in mice, and now we know that this gene exists in humans. We have cloned this gene and, moreover, we know that this hormone is present in human blood plasma. A person definitely has betatrophin!".
The discovery of betatrophin would not have been possible without federal funding for basic science. Dr. Malton's application, called "The search for genes and compounds that cause beta cell replication," impressed the officials of the National Institutes of Health (National Institutes of Health) involved in the provision of grants, and received financial support for 80 percent of the work.
For more than 15 years, the main focus of Dr. Malton's work was not type 2 diabetes, but the less common juvenile diabetes, on which he focused his attention when such a diagnosis was made to his young son (and later to his daughter). In addition, most of Malton's work was related to the use of stem cells, the fundamental building blocks of all organs, as treatment methods and targets for drug development. But stem cells did not play a direct role in the discovery of betatrophin. Rather, this discovery is a classic example of how scientists, with sufficient resources, ask questions and get answers to them that go beyond the usual topics of their laboratories and institutes.
"I could tell you that this discovery is the result of deep reflection, and we knew that we would make it, but rather it was a happy accident," Dr. Malton, who is also a researcher at the Howard Hughes Medical Institute, shares his thoughts. "We were just wondering what happens if an animal lacks insulin. We were lucky enough to find this new gene, which had previously gone largely unnoticed. Another clue came in the process of studying what people know about, but do not attach due importance to, namely: what happens during pregnancy? During pregnancy, the carbohydrate load and the need for insulin increase significantly due to nutrition and fetal growth. Now the body needs more beta cells, and it turns out that during pregnancy the level of this hormone increases. By studying pregnant mice, we found that when pregnancy occurs, this hormone is turned on to create more beta cells."
Although Dr. Malton sees betatrophin primarily as a treatment for type 2 diabetes, he does not rule out that the hormone may play an important role in the treatment of juvenile diabetes, possibly increasing the number of beta cells and thereby slowing the progression of this autoimmune disease at the earliest stages of its development.
Emphasizing the need for additional research, Malton, however, believes that clinical trials of a betatrophin-based drug can begin in the next three to five years – the shortest possible time for the development of a drug.
Working with the Technology Development Bureau of Harvard University, Malton and I have already signed a cooperation agreement with the German biotech firm Evotec, where 15 scientists are currently working on betatrophin. The compound is licensed to the Johnson & Johnson-affiliated company Janssen Pharmaceuticals, which has already begun work on promoting betatrophin to the clinic.
Portal "Eternal youth" http://vechnayamolodost.ru29.04.2013