Cartistem is manufactured from mesenchymal stem cells derived from allogeneic (donor) umbilical cord blood. Umbilical cord blood is a readily accessible source of high-quality stem cells, is associated with minimal health risks and carries relatively few ethical concerns.
The stem cells are mixed with hyaluronan, a natural polymer that plays a major role in wound healing and is a building block of joint cartilage. Cartistem is surgically administered into the area of cartilage damage following an arthroscopic surgery as an adjunct to microfracture, a commonly used technique used to repair cartilage damage.
The principal investigator on the study is Brian Cole, MD, a professor in the Department of Orthopedic Surgery and anatomy and cell biology at Rush University Medical Center. Cole is the head of Rush’s Cartilage Restoration Center and is also the head team physician for the Chicago Bulls. Cole and his co-researchers will assess the drug’s safety as well as its ability to regenerate cartilage repair tissue and reduce pain in patients with localized cartilage loss in the knee.
Treating cartilage damage can be problematic because the tissue does not contain blood vessels or nerves and therefore has a limited ability to regrow. Various treatments for cartilage degeneration, such as drug therapy, arthroscopy and joint replacement, yield mixed results and are unable to regenerate damaged tissue.
The two-year, phase I/IIa study will enroll a total of 12 participants age 18 years and older, with a body mass index of less than 35. Initially, six individuals with lesions of 2 to 5 centimeters will be recruited into the study; an additional six volunteers with lesions larger than 5 centimeters will be enrolled sequentially. Each participant will undergo eligibility screening followed by a 12-month observation period to determine the safety and efficacy of the drug, with an additional long-term follow-up evaluation at 24 months.
"With a burgeoning aging, yet active population, our patients are looking for effective non-joint replacement solutions to treat their damaged knee cartilage,” Cole said. “This research is significant in that it utilizes a commonly performed operation (microfracture) in an effort to improve upon variable outcomes.”