Crohn's Disease

remestemcel-L is being developed for the treatment of Crohn’s disease refractory to steroids and immune suppressants.

Disease Indication and Patient Population

Crohn’s Disease is a chronic inflammatory disorder of the gastrointestinal tract, characterized by periods of remission and symptomatic relapse.

The United States has the highest prevalence of the disease, with more than 600,000 people afflicted and approximately 20,000 new cases diagnosed each year1. Of the 600,000 American patients, studies have shown that approximately 8-20% are unresponsive, resistant or intolerant to existing treatments, which include corticosteroids, immunosuppressants and biologics2. The global Crohn’s disease therapeutics market was estimated to be worth $4.4 billion in 20123.

A treatment to induce rapid remission is highly needed, particularly in high-risk patients such as those with biologic-resistant disease and those with fistulas, a complication of Crohn’s disease which occurs in 20-40% of patients and often requires invasive surgical procedures4.


remestemcel-L is a tier 2 product candidate which consists of 100 million mesenchymal stem cells (MSCs). All doses of remestemcel-L for the treatment of Crohn’s disease consist of 100 to 200 million MSCs delivered intravenously in a multiple dose regime.

Mechanism of Action

remestemcel-L has demonstrated immunomodulatory properties in preclinical studies to regulate T-cell mediated inflammatory responses by inhibiting T-cell proliferation and down-regulating the production of the pro-inflammatory cytokines, including tumor necrosis factor-alpha, or TNF-alpha, and interferon gamma. More critically, MLCs have been shown to be capable of effective down-regulation of Th17 cells, reduction in IL-17 levels, and induction of FOXP3 regulatory T cells. These inflammatory pathways are acknowledged to be central to the pathogenesis of Crohn’s disease and other inflammatory conditions.

Clinical Trials

A randomized controlled trial for remestemcel-L for the treatment of Crohn’s disease is ongoing.


1Decision Resources Crohn’s Disease December 2013

2Abreu, Maria T. Anti-TNF Failures in Crohn's Disease. Gastroenterol Hepatol (N Y). 2011 Jan; 7(1): 37–39

3Decision Resources Crohn’s Disease December 2013

4Swartz, David et al. The Natural History of Fistulizing Crohn’s Disease. Gastroenterology 2002;122:875–880