CIRM funds many projects seeking to better understand osteoperosis and to translate those discoveries into new therapies.
Estimates of the number of people in the U.S. with osteoporosis range from 10 million to 25 million, with 75 percent being women. For many of those individuals it can be a disease with minimal immediate impact but incredible lingering risk. Between 1.5 million and 2 million of those with the condition develop osteoporosis-related fractures each year. About 70 percent of fractures are in the vertebra of the spine, and they can range from minor to completely debilitating. The next most common fracture is in the hip, which increases the risk of premature death and frequently lands otherwise healthy functional elderly in nursing homes for the remainder of their lives. Osteoporosis costs the nation an estimated $17 billion a year.
Osteoporosis is a disease of aging. When we are young we have plenty of bone-forming stem cells that can replace any bone loss and repair most damage. Between the ages of 30 and 80 we have a 10-fold decrease in the number of these stem cells and the ones we have left are less effective at replacing and repairing bone. California’s stem cell agency has funded several projects that propose various ways to increase the number of these stem cells, or improve their effectiveness to help keep bones healthier longer (the full list of CIRM awards is below).
One team is testing bone-forming stem cells in animal models and revving up those cells with a hormone known to accelerate bone repair. Another group has isolated from fat tissue what they are calling a master mesenchymal stem cell, the class of stem cells capable of forming bone. They too, are giving the stem cells a boost, in this case with a special growth factor they have identified, and are testing it in animal models.
In July 2012, CIRM awarded one of its Disease Team awards to a group of researchers who are trying to use a new drug to improve the ability of patients’ own stem cells to make new bone. These awards require assembling the team necessary to do the experiments that are required before the Food and Drug Administration (FDA) will allow the potential therapy to be tested in people. In some cases, these awards also fund the early phase clinical trial once it is approved.
Disease team awards
University of California, Davis
This team proposes to increase the effectiveness of a patient’s own bone-forming mesenchymal stem cells. They have developed a drug that directs the stem cells to go to the bone surface and form new bone. During the first phase of the grant they will be working to ensure this drug meets the highest quality standards required by the FDA, and testing it in the lab to make sure it is both safe and shows some signs of being effective. During the second part of the project they plan to conduct a phase 1 trial to gauge safety, first in postmenopausal women and then in both women and men.