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Hematology


Graft-Versus-Host Disease

Over 60,000 stem cell transplants are performed annually worldwide. In the U.S. alone, some 18,000 allogeneic bone marrow transplant procedures were performed in 2004, estimated to increase to 30,000 in 2007. Depending on whether the source is autologous (60%) or allogeneic (40%), cost per procedure ranges from $150,000 to $300,000. Not accounting for the current 10%-35% mortality rate, the overall healthcare burden was a staggering $4B in 2004. With an expected 25% annual growth rate (ITI Life Sciences), this burden is expected to reach $25-30B by 2010 (TechConnect Summit). These numbers do not even account for the much larger number of patients who endured long transplantation waiting lists, only to succumb to their disease prior to locating an appropriate HLA-matched donor (National Marrow Donor Program).

Region Potential Donors1 Annual Demand2 Annual Allogeneic Transplants Unmet Medical Need
USA
3,100,000
46,100
18,200
61%
Europe
2,900,000
37,500
9,720
76%
Other
500,000
2,400
925
62%
Total
6,500,000
86,000
28,845
67%
168% of total donors are typed for HLA-A, B, DR
2Match searches initiated each year


Furthermore, imperfectly matched recipients may suffer from a potentially fatal side effect known as graft-versus-host disease. Recently, MSCs have been shown to possess immunosuppressive effects. Human clinical trials showed these cells could be used to suppress graft-versus-host disease in patients undergoing bone marrow transplantation. Unfortunately, results from clinical trials are revealing loss of benefit within two weeks of transplantation and disappointingly low rates of long-term engraftment. A need for biweekly infusions severely limits the future clinical utility of current MSC products.

2010 Escape Therapeutics, Inc. All rights reserved.

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