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Know About Ulcers Blog

New Deal – New nanotherapy research for inflammatory bowel diseases


In Europe over 2 million people are
affected by Ulcerative Colitis or Crohn’s Disease, conditions that are
characterized by an inflammation of the bowel. They occur when cells from our
immune system become activated and react against contents of the intestine,
leaving the patients with fatigue, abdominal pain and severe diarrhea, blood in stools or even with life-threatening complications such as bowel perforation,
bleeding, and in long-standing disease, an increased risk of cancer. Alarmingly, the incidence of IBDs is increasing. It is now one of the most common
immune-mediated diseases in young adults. The main therapeutic options are
conventional drugs or biological therapy. Both inhibit immune cells but increase,
as a side effect, the risk of infections. Moreover, they are only effective for
limited subsets of patients. Recent clinical studies testing a new class of drugs have pointed out two potential targets inside immune cells: JAK1 and JAK3.
These proteins are needed for immune cell activation thus, without them
the inflammation will be hampered. Unfortunately, these drugs have a
systemic distribution, are not specific, and present adverse effects. That’s why scientists and companies all over Europe have joined forces to fix this unmet clinical need in a research project called New Deal. They will target JAK1and JAK3 in a more specific way, using small pieces of genetic material called siRNAs. These molecules will interfere with the cellular production of JAK1 and
JAK3 by binding exclusively the molecules that contain their genetic
instructions and impeding the synthesis of these two proteins. Without them the
activation of the cells is hindered and the inflammation decreases. But to make it into these cells is a challenge, to reach the bowel the siRNAs will be
incorporated into micro capsules and inside them lipid nanoparticles will
guarantee their stability and allow them to enter the cells. This formulation will allow a local distribution diminishing possible side effects. Scientists will test this strategy in mouse models of the disease to be able to provide the
regulatory agencies a solid preclinical proof-of-concept that would
open the door to start human clinical trials and test the real capabilities of
this therapy in the future.

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