Hematology
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.