disease. A modified per-protocol analysis was devised that showed that complete fistula healing was observed in 27 of 33 patients (82%) by eight weeks after ASC injection. Of 27 patients with fistula healing, 26 patients completed additional observation study for one year and 23 patients (88%) sustained complete closure. There were no adverse events related to ASC administration. Furthermore, complete closure was well sustained. These results strongly suggest that autologous ASCs could be a novel treatment option for the Crohn’s fistula with high risk of recurrence. However, stem cell–based therapies receive criticism, as autologous transplantation reintroduces the genetically defective immune cells.
Osiris Therapeutics developed a product called Prochymal from adult bone marrow for graft-versus-host disease (GVHD) and Crohn’s disease. It is the only stem cell therapeutic currently designated by the FDA as both an Orphan Drug and Fast Track product. Phase 3 clinical trials are under way to establish the safety and diversity of Prochymal in patients with moderate to severe refractory disease.
As of December 2013, researchers at Brigham and Women’s Hospital and Massachusetts Institute of Technology were able to produce large numbers of intestinal stem cells and direct these cells to different types of mature intestinal cells. This advancement is not only a milestone in stem cell therapy, but it will also be useful for pharmaceutical companies to screen and identify new drugs.
A recent study published by Duijvestein et al. showed that the presence of mesenchymal stem cells does not hamper the immunosuppressive effects of conventional therapy. Vos et al. found that mesenchymal stem cells can turn the macrophages into the regulatory phenotype. This combined therapy is expected to promote rapid recovery and tapering of immunosuppressive therapy.
Stem cell therapy will be an important adjunct to conventional management of Crohn’s disease, but much work remains to be done.
Isma Nusrat Javed
Rawalpindi Medical College
See Also: Gut: Current Research on Isolation or Production of Therapeutic Cells; Gut: Development and Regeneration Potential; Gut: Existing or Potential Regenerative Medicine Strategies; Gut: Major Pathologies; Gut: Stem and Progenitor Cells in Adults.
Further Readings
Dalal, Jignesh, Kimberly Gandy, and Joe Domen. “Role of Mesenchymal Stem Cell Therapy in Crohn’s Disease.” Pediatric Research, v.71 (2012).
Lee, W. Y., K. J. Park, Y. B. Cho, et al. “Autologous Adipose Tissue-Derived Stem Cells Treatment Demonstrated Favorable and Sustainable Therapeutic Effect for Crohn’s Fistula.” Stem Cells, v.31/11 (2013).
Lincoff, N. “Stem Cells Used to Repair IBD Gut Damage.” HealthlineNews (October 24, 2013). http://www.healthline.com/health-news/crohns-gut-stem-cells-could-repair-ibd-damage-102413.
Medline Plus. “Clinical Trials.” http://www.nlm.nih.gov/medlineplus/clinicaltrials.html (Accessed October 2014).
Clinical Trials, U.S.: Diabetes
Clinical Trials, U.S.: Diabetes
239
243
Clinical Trials, U.S.: Diabetes
About 8.3 percent of the global population—a total of 366 million people in the world—have diabetes mellitus. In the United States, about 7.8 percent suffer from diabetes, a group of chronic metabolic disorders in which a person has high blood sugar and which affect the body’s metabolism. Type 2 diabetes is recognized by clinicians as the most common form of diabetes, affecting roughly 90–95 percent of those diagnosed with the disease. Type 2 diabetes is often linked to obesity, older age, genetics, physical inactivity, and certain ethnic groups. The body’s inability to process insulin coupled with insulin resistance allow the diagnosis of this condition.
In contrast to type 1 diabetes, previously referred to as insulin-dependent diabetes mellitus (IDDM) or juvenile diabetes, the symptoms of type 2 diabetes develop over time, and at a slow pace. Researchers and scientists are currently focusing their efforts on disease prevention and on the ways to delay the occurrence of type 2 diabetes in at-risk patients. They have come to the conclusion that the individual’s lifestyle is key in keeping these conditions at bay, and agreed on the fact that some medications or interventions can reduce the progress of the disease.
The public health toll of diabetes is constantly growing, with the current statistics and the projections of its prevalence estimated today at more than 552 million people worldwide by 2030. The costs related to diabetes and its associated complications in North America alone are assessed to be about US$223 billion. In light of this, it is essential to keep conducting clinical trials to define the potential interventions needed to prevent and treat diabetes. One can wonder if the numerous current active clinical trials and future studies will be able to enlighten researchers and clinicians on better ways to address the existing deficiencies that confront them.
How Type 1 and Type 2 Diabetes Are Different
The onset of type 1 diabetes, also known as insulin-dependent diabetes or juvenile diabetes, is typically sudden: an individual visits his or her health care provider and discovers the existence of symptoms of high blood sugar. In type 1 diabetes, the body’s immune system kills the cells that produce insulin, ultimately eliminating completely insulin production by the person’s body. Without insulin, cells cannot absorb sugar. The most frequent symptoms of this condition include increased urination, sensation of dry mouth, thirst, hunger, blurred vision, weight loss, pain in the hands or feet, and frequent infections.
Insulin, produced in the pancreas regulates metabolism and removes excess glucose from the blood. Type pdb 9INS, shown here in a hexamer, highlights the threefold symmetry, the zinc ion that holds it together as well as the histidine residue involved in binding. (Wikimedia Commons)
Type 2 diabetes, also called non-insulin-dependent or adult-onset diabetes, can affect people at any age, although it usually occurs in middle-aged and older people. Obesity and physical inactivity are causes more likely to develop type 2 diabetes. Associated with type 2 diabetes, is insulin resistance—a condition that occurs when fat, muscle, and liver cells do not use insulin to carry glucose into the body’s cells.
Current Treatments for Type 1 Diabetes and Type 2 Diabetes
As stated above, there is no cure for diabetes. Contrary to type 2 diabetes that can most of the time be controlled by a healthy diet and regular exercise, type 1 diabetes cannot. Individuals suffering from type 1 diabetes must test their blood sugar levels several times a day and get insulin injections whenever needed. If not treated, high blood sugar levels can seriously damage the eyes, heart, kidneys, blood vessels, and nerves. On the other hand, injecting too much of insulin can reduce the blood sugar level to a dangerous level that could result in hypoglycemia and be fatal.
Current treatment of type 1 diabetes takes place through transplantation of islet cells or even pancreas, into the patient. Transplants serve to incite the body to regain control of blood sugar levels so that insulin injections
