Accelerated federal project, combining expertise from CU Anschutz, CU Boulder and Colorado State University scientists, aims for human clinical trials within 18 months
A groundbreaking and fast-paced effort - combining biologic, clinical and regulatory expertise at the University of Colorado Anschutz - has helped propel an ambitious nationwide attempt to reverse osteoarthritis (OA), a painful degenerative disease that affects 32 million Americans.
Over just two years, a team of multidisciplinary researchers, including at CU Boulder and Colorado State University, developed a suite of therapies that show promise restoring cartilage and bone in animals with OA. The incremental progress made for OA treatments over decades - which has been limited to anti-inflammatory joint injections or expensive joint replacement surgery - is now accelerating toward a possible eradication of the disease - and at an affordable cost to patients.
The federal agency behind the collaborative effort, the Advanced Research Projects Agency for Health (ARPA-H), announced this week that the team will advance to the next phase of the up to $33.5 million project. The phase-two goal of Novel Innovations for Tissue Regeneration in Osteoarthritis (NITRO), the inaugural ARPA-H program, is to move the technologies toward investigational new drug-enabling studies that are needed to begin human clinical trials within 18 months. NITRO is led by ARPA-H Program Manager Ross Uhrich, DMD, MBA.
Point of the program: 'It's a moonshot'
"Two years ago, some of these therapies were just ideas, reflecting the moonshot nature of this program from the very start," said Karin Payne, PhD, associate professor of orthopedics at the CU Anschutz School of Medicine and co-principal investigator on the award. "Across the three institutions we had anywhere from 40 to 50 people working collaboratively on all these different technologies. Here at CU Anschutz, we isolated cells from patients that were undergoing total joint replacement and consented to have their tissues donated so we could isolate cartilage and bone cells."
Key points:
- In two years, a multidisciplinary team of researchers developed a suite of regenerative therapies that restore cartilage and bone in animal models.
- The next phase of the inaugural program of ARPA-H is to begin safety and toxicology studies before human trials planned for 2028.
- The project's goal is to cure osteoarthritis, a degenerative disease that affects 32 million Americans and currently has no cure.
The cells from these tissues, which would have otherwise been discarded, were shipped to CU Boulder and tested with biomaterials developed in the lab of project leader Stephanie Bryant, PhD, professor in the Department of Chemical and Biological Engineering, Materials Science and Engineering, and the BioFrontiers Institute at CU Boulder. The biomaterial-based therapies developed at CU Boulder were then sent back to CU Anschutz, or CSU, to be tested in animal models of osteoarthritis.
Bryant and Payne are joined by co-Principal Investigator Michael Zuscik, PhD, professor and vice chair of research in the Department of Orthopedics at CU Anschutz; co-Principal Investigator Laurie Goodrich, DVM, PhD, a veterinary clinician scientist at CSU; as well as Senior Investigator Kelly Santangelo, DVM, PhD, a veterinary clinician scientist at CSU. This multidisciplinary Colorado-based team is now on the forefront of developing a minimally invasive therapy that regenerates cartilage and bone cells - essentially allowing a joint to heal itself.
The Colorado team's main goals in the next phase of the award are to continue developing:
- An intra-articular joint shot that would be given to a patient once per year. The injection involves a controlled delivery system that releases a medication that regenerates bone and cartilage in a specific joint space, such as the knee.
- A cocktail of engineered proteins and novel biomaterials that can be injected arthroscopically and solidified in specific locations to repair defects. Because it recruits the body's own progenitor cells to patch holes or gaps in the cartilage, the treatment is for patients with significant lesions in the cartilage or bone.
Working together toward one goal
While the program operates on a much faster schedule than a typical federally funded research grant, the researchers appreciated the regular oversight and communications from ARPA-H, which closely monitors the reaching of key milestones.
"Being part of this program has been a great experience," Payne said. "We all share a common goal of developing therapies that will someday reverse osteoarthritis. The collaborative approach has allowed us to advance science at a fast pace."
Zuscik highlights that the scope of financial and programmatic support provided by NITRO has created a clear pathway for developing and testing innovative approaches to treat OA, representing a major acceleration of progress in a field that has yet to deliver new effective treatments.
"It has been very exciting for me to be part of this effort. NITRO underpins a set of cross‑institutional collaborations that now has the potential to move the needle both for researchers seeking new solutions and for patients who need effective approaches to address their OA." he said. "It's really the only way you can make a difference, to have visionary-level people - Karin, Stephanie, Laurie and Kelly - bringing thought process to a problem from multiple perspectives."
New resources through community outreach
As part of a two-year community outreach campaign, the CU Anschutz team also identified barriers to accessing care for OA patients statewide. Through open discussion, listening sessions and community engagement efforts, the team's engagement officer Charlene Shelton, PhD, assistant professor in the Department of Orthopedics at CU Anschutz, created new resources to both educate and engage the community, including a quarterly newsletter informing a network of OA patients about OA disease and the research team's progress toward new treatment options.
Learn more about osteoarthritis
To learn more about participating in this osteoarthritis research, or just staying connected with the CU Anschutz study team:
- Clinical Trial Interest List: If you'd like to put your name on a list of interested participants and be contacted about future clinical trials, please fill out this short survey: Osteoarthritis Clinical Trial Interest
- Newsletter:
