- what is Typ-1-Diabetes at all?
- What were the researchers able to do? find out?
- development steps of the medication
- What does this mean for the future?
One type 1 diabetes disease usually begins at an early age. So far it has not been possible to postpone the outbreak of the disease; but now it was in the US for the first time one antibody therapy approved, which is intended to achieve just that. What this for you future therapy could mean and what exactly the researchers found out have:
Type 1 diabetes: This is what is behind the disease
Type 1 diabetes is not uncommon. In Germany alone, around 373,000 people live with the disease. These include around 32,000 children and young people. How the health information of the Institute for Quality and Efficiency in Health Care (IQWiG) reveals, fall ill every year about 2 in 10,000 children new. In most cases, the disease begins in childhood, adolescence or young adulthood. Type 1 diabetes is therefore also common juvenilesi.e. juvenile, called diabetes.
Diabetes is fundamentally one umbrella term for different diseases of the metabolism. The different Diabetes Forms have in common that patients through a lack of insulin and/or reduced insulin action have high blood sugar levels.
Type 1 diabetes is characterized by an absolute Mangel des Hormons Insulin caused. This form of diabetes is caused by a failure of the cells in the pancreas caused. These normally produce the hormone insulin. Insulin is one of the 6 important hormones that mainly control our body. Type 1 diabetes has not yet been cured, which is why patients all her life need to inject insulin. He has to do it several times a day blood sugar measured become. It is important for those affected that they Nourishment on the Adjust insulin dosageto prevent blood sugar fluctuations.
Drug against the onset of type 1 diabetes
In the US, one was now antibody therapy approved, which should make it possible to delay the onset of type 1 diabetes. Like the magazine spectrum reported, 21-year-old Mikayla from Idaho is currently living diabetes-free as a result of the treatment. With her, a strong increased risk of disease found out, after which she underwent treatment with the drug teplizumab. The active ingredient is aimed at those T cells in our immune system to hold backwhich the insulin producing cells in the pancreas destroy.
Mikayla was one of a total of 76 subjects who between 2011 and 2018 respectively two-week treatments with teplizumab as part of a clinical study. Some of the study participants actually received the active ingredient, while others only received a placebo. The result: On average, those subjects who were treated with teplizumab only developed after about five years Diabetes Symptoms. These include, for example, extreme thirst, severe unwanted weight loss, a noticeable drop in performance, frequent infections, blurred vision, difficulty concentrating and nausea as well as abdominal pain. Symptoms developed in those who received a placebo already after 2 years on.
Some study participants did not have to start the insulin therapy otherwise required for the disease for a decade or more. For Mikayla it is now six and a half years. The drug was in November 2022 by the drug authorities approved in the US. Teplizumab is approved for people aged eight and older. The drug has not yet been approved in Germany.
Development of teplizumab: first steps
The active ingredient teplizumab goes back to the pharmaceutical company Ortho Pharmaceutical in New Jersey. Here became one first version of the antibody developed by scientists. This active ingredient version still bore the name OKT3. The molecule originally comes from mice and has the ability to attach itself to the surface of T cells. This will do that cell-killing potential of these T-cells is reduced. Already 1986 OKT3 was approved as the first therapeutic antibody to prevent organ rejection after kidney transplantation.
However, OKT3 worked not optimal, but triggered life-threatening complications in high doses. The scientists suspected that the cause was that the human immune system recognizes the antibodies originally from mice as foreign and fights them. Outside the company, the search for the antibody’s formula began. Soon could developed teplizumab become.
1999 the antibody was used as a treatment for people in tested in clinical studies. For this purpose, subjects were selected in whom type 1Diabetes was newly diagnosed. The Study was led by Kevan Herold, an immunologist. His result: Only a single treatment could help those affected to maintain or even increase their insulin activity for years.
So the search for the active substance continued
Kevan Herold’s study was very small, which is why one larger study followed. This one, on the other hand, ended with one disappointment: Teplizumab proved no more effective than placebo. In addition, a similar drug called otelixizumab. This drug was also unable to show any effect in the study, so both therapies were dropped. This study was a knockout for all diabetes research; however, was not given up.
In different Experiments using the drug, the team found that teplizumab depletes the T cells that target the cells in the pancreas. This can protect the pancreas from further attacks. In particular, patients in whom the disease only slightly advanced is appeared to be able to benefit from the drug.
Kevan Herold’s conclusion was that teplizumab can only really unfold its effect if it is administered before the onset of diabetes symptoms. 2011 was then the Teplizumab Prevention Study started, in which Mikayla also took part as a high-risk patient. The study results were convincing, so teplizumab 2022 for those who no T1D diagnosis yet but have two or more autoantibodies against islet cells and show signs of altered sugar metabolism.
The active ingredient reveals these opportunities
Aaron Kowalski, the president and chief executive officer of the New York-based nonprofit research organization JDRF, which specializes in type 1 diabetes, stressed Spectrum, that this one very big progress be in the area. This is the first disease-modifying therapy available for type 1 diabetes.
In addition, the active ingredient is the first to be proven to delaying the onset of an autoimmune disease can. In this sense, the drug could groundbreaking for other drugs against other autoimmune diseases. These include, for example, rheumatoid arthritis or multiple sclerosis.
Teplizumab can prevent type 1 diabetes only delay and not completely prevent it. The researchers are therefore not yet at the end of their research, but are continuing to search for a remedy that could possibly prevent the disease entirely. For those who get the drug, however, it can mean a few more years of carefree childhood or adolescence. If the disease occurs later, this means for those affected less stress. One reason for this is that blood sugar levels do not have to be measured constantly. In addition, very high blood sugar levels also mean stress for blood vessels, nerves and other organ systems, which can therefore be avoided at least temporarily.
Conclusion – therapy has its price
For children and adolescents, delaying type 1 diabetes for a few years can Big relief be. So far, the drug is only approved in the US and only for people who don’t yet have a type 1 diabetes diagnosis, however two or more autoantibodies against islet cells and Signs of altered sugar metabolism exhibit. It is unclear whether the drug will also be approved in Germany and depends largely on what happens to teplizumab in the USA. However, there is still an incentive for diabetes research to find a drug that treats type 1 diabetes can completely prevent. Other research groups are also investigating several other drugs that are also suitable for people with stage 2 disease, for example.
However, it should also be considered that the treatment is anything but cheap and affordable for everyone. A one-time treatment costs about $194,000; there are also costs for the infusions and the initial screening. For the drug manufacturer, the high pricing is noisy spectrum appropriate, since it is, after all, a groundbreaking drug.
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