Microvillus inclusion disease (MVID) is a rare, gastrointestinal disease which can cause impaired development in children. It is characterized by severe diarrhea which leads to dehydration and malnutrition. MVID can be lethal when left untreated. Unfortunately, the current treatment options are far from ideal. The standard therapy is parenteral nutrition up to 24 hours every day. This consistent intravenous therapy can cause vein damage, infections, and even liver failure.
Some patients end up receiving a liver transplant or small bowel transplant, but of course these procedures come with their own risks and complications.
However, recent research conducted by Dmitry Kravtsov, the Vice President of Research and Development at Vanessa Research, has indicated that the development of a new therapeutic option for MVID patients may be possible. He was able to identify what role intestinal cell immaturity plays in the onset of diarrhea. His research suggests that the gut’s immature cells could be restored. This would allow the gut to properly absorb nutrients, effectively halting the progression of MVID.
The Turkish Ministry of Health has just recently granted Vanessa Research regulatory approval to conduct a clinical trial for Shylicine. This is an oral therapy which researchers believe could allow MVID patients to live, function, and thrive without parenteral nutrition.
This is a Phase 2 trial which aims to evaluate the safety and efficacy of the therapy. It will take place at the Ankara University School of Medicine in Turkey. Vanessa, a United States based company, has already received a drug import permit, allowing them to deliver the investigative therapy to the school.
The primary endpoints of this investigation are reduction of diarrhea volume/frequency of stool and decrease in parenteral nutrition.
Hopefully this trial will indicate continued promise of this oral therapy, and will eventually lead to a new option for MVID patients.
In addition to MVID, Vanessa is already in preclinical investigations of Hunazine, which could serve as a treatment for those affected by cholera.
You can read more about the Shylicine clinical trial and Vanessa’s work here.