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Researchers at IDIBAPS (August Pi i Sunyer Biomedical Research Institute) and the University of Calgary, led by Dr. Pere Santamaria, have discovered new cell mechanisms and drugs that protect the body from autoimmune diseases without attacking the immune system and, therefore, leaving patients exposed to other diseases, like current drugs do.

Type-1 diabetes, multiple sclerosis and rheumatoid arthritis are the first three autoimmune diseases the treatment has been applied to in a study with mice that has proven the drug to be effective, safe and without any significant side effects. This has prompted the beginning of manufacturing the drugs for clinical trials.

The drugs are designed with nanoparticles that adhere to the T lymphocytes that act inappropriately due to an autoimmune disease (meaning they attack the body’s own cells instead of protecting them). When the two agents interact, the T lymphocytes become regulatory cells, suppressing inflammation and curbing the progression of the disease. So, the new drugs identify defective lymphocytes and only attack them. “They wouldn’t cure the diseases or regenerate the part of the organs that have been destroyed, but they would bring balance back to the organism’s immune system,” explains Dr. Pere Santamaria.

The research, published in the journal Nature, identified the action mechanism that explains the efficacy of these nanoparticles, which is a chain mechanism. “The regulated lymphocytes start to give instructions to other white cells in the immune system to make them into ‘good cells’ and this creates a domino effect,” explains Santamaria. So, they expand in vivo efficiently and in a way that can be reproduced.


Drugs based on unique design

One of the most important aspects of the nanoparticles is that they could be effective in treating many of the 80 known autoimmune diseases. In fact, the drugs developed for type-1 diabetes, multiple sclerosis and arthritis are nearly identical. “Just by changing 14 amino acids, we can redirect the drug towards one disease or another. It has everything a drug should: specificity and efficacy,” says the professor at the University of Calgary.

The research group has tested the efficacy of these drugs in treating five different autoimmune diseases and all five have yielded positive results. “If it works in humans like it does in mice –which we aren’t sure of yet- we’ll simplify the complex therapeutic approach to autoimmune diseases and you can imagine the impact that would have on society,” says Pere Santamaria. In fact, the studies were conducted on humanized mice that had been transplanted with leucocytes from human diabetes patients.

After the study was published, the research was transferred to pharmaceutical company Parvus Therapeutics, founded in 2009 by Pere Santamaria, to start manufacturing the drugs and doing clinical trials. The first will be that for type-1 diabetes, expected within the next three years, and will cost approximately €30 millions. “We have private capital and are negotiating a partnership with large pharmaceutical companies to help us fund clinical trials. If all goes well, after diabetes we’ll start with multiple sclerosis,” explains Santamaria.

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