Stem Cell Can Help
Osteoporosis, Bone and Cartilage Disease
CIRM funds many projects seeking to better understand bone related diseases including osteoporosis, osteoarthritis and osteonecrosis 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 $19 billion a year.
Osteoarthritis is a disease that affects the cartilage in joints. It is one of the most common forms of disability, effecting more than 27 million people in the U.S. CIRM funds several projects looking to replace or repair the cartilage lost in the degenerative disease. Projects include creating new cartilage from donor stem cells as well as developing a drug to drive a person’s own stem cells to do a better job of repair.
Osteonecrosis is a disease that decreases blood circulation to bones causing them to weaken and eventually die. If left untreated, patients with osteonecrosis can develop end-stage hip arthritis and require surgical joint replacement. Despite the low prevalence compared to primary osteoarthritis or degenerative arthritis, femoral head (hip) osteonecrosis has a significant economic impact because it largely affects individuals in the prime of life (peak age 35 years).
CIRM also funds project to help the thousands who live with fractures too big to heal or fractures in older individuals that their aging bodies can no longer heal. 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).
Clinical Stage Programs
California Institute for Biomedical Research
Researchers at the California Institute for Biomedical Research (CALIBR) have been awarded $8.447 million to test KA34, a drug that, in preclinical tests, recruits stem cells to create new cartilage in areas damaged by osteoarthritis. CIRM funded the research that developed this technology and now this Phase 1 trial will test this stem cell directed treatment in people with osteoarthritis of the knee, hopefully slowing down or even halting the progression of the disease.
A team at UC Davis has developed a new stem cell-based treatment for osteonecrosis. They are using a small molecule drug that targets the mesenchymal stem cells in a patient's bone marrow and directs the stem cells to the surface of the bone where they then develop new bone tissue. The team is testing the safety and efficacy of this drug treatment in a Phase 1 clinical trial.