Esperite’s R&D Division The Cell Factory is working with the Women’s and Children’s Health Department of the University of Padua and the Padua University Hospital in Italy on a innovative project to develop therapies using extracellular vesicles (EVs) to treat perianal fistulas, a serious complication of Crohn’s disease (CD). The first study in adult patients is scheduled to begin in 2017.
Inflammatory bowel disease (IBD) is a group of inflammatory conditions of the colon and small intestine, affecting approximately 0.5 percent of western countries’ populations; the number is rising. Crohn’s disease (CD) and ulcerative colitis (UC) are the principal types of inflammatory bowel disease.
According to Crohn’s and Colitis UK, perianal fistulas are the most common fistulas in IBD patients and surgery often is considered. Perianal fistulas increase the risk of cancer and life-threatening systemic inflammation.
Treatment for perianal fistulas has relied in the past years on surgical procedures, either surgical seton (thin silicon stitch) placement for chronic drainage of the fistula, or closure of the internal fistula opening by creating an advancement plasty. Introducing anti-TNF agents (infliximab and adalimumab) shifted patients’ treatment from surgical procedures to almost exclusive anti-TNF -based therapies.
However, some patients with perianal fistulas often do not respond to these therapies. Consequently, clinical trials are currently testing the use of allogenic mesenchymal stem cells (MSCs) to target perianal fistulas. So far, outcomes are encouraging.
MSCs are ideal for cell-based therapy in various inflammatory diseases because of their immunosuppressive and tissue-repair properties.
“Our approach is focused on using extracellular vesicles (including exosomes) derived from mesenchymal stem cells (MSCs) for the first time in the treatment of inflammation responsible for Crohn’s disease perianal fistulas,” Esperite wrote in a press release.
Extracellular vesicles (including exosomes) are small, natural biological particles secreted by different types of cells in vivo and in vitro that contain proteins, growth factors, messenger RNA, as well as other particles responsible for the therapeutic effect of MSCs.
Compared to allogenic MSCs, EVs have the advantage of being much cheaper to produce, they present no risk of uncontrolled proliferation and differentiation, they lower the risk of immune response, and they are easily and safely delivered into different tissues and organs in vivo.
Multiple preclinical studies have shown encouraging data on the anti-inflammatory effects of EVs. Using animals models, Professor Maurizio Muraca at the Bambino Gesù Children’s Hospital in Rome, and now at the University of Padua in Italy, and his team have shown that MSC-derived EVs are effective in treating IBD.
In collaboration with the Cell Factory, the research team at the University of Padua is conducting tests in animals using clinical grade EVs to confirm their efficiency and safety, as well as its bio-distribution. They also are aiming to better explain the mechanism of action of clinical grade EVs in IBD before they enter into clinical trials, scheduled for 2017.
Professor Muraca said in a news release “We expect (EVs) therapeutic effect to be more reproducible and less prone to the influence of the recipient’s environment.”
Esperite now owns a broad portfolio enabling the use of EVs derived from MSCs in treatment of autoimmune, chronic and acute inflammatory diseases.