Clinical Development of a Small Molecule Drug Targeting Endogenous Stem Cells to Restore Cartilage and Relieve Pain in Osteoarthritis Patients

Funding Type: 
Strategic Partnership I
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

We propose to develop the first drug therapy to reverse the damage of osteoarthritis (OA) in joints. OA is a serious disease that involves the degeneration of joint cartilage and the underlying bone resulting in joint pain, stiffness and immobility. OA is the leading cause of disability and chronic pain in the world and its impact on patients and its economic burden are rapidly increasing with our aging demographics. The statistics for OA are staggering: 27 million Americans and 3.25 million Californians have moderate to severe OA, 25% of OA patients cannot perform their major daily activities of life and 80% have limitations in movement. Ultimately, the pain and disability caused by OA is so severe that 1 million patients annually opt for painful, and for certain patients life-threatening, joint replacement surgery.

Our proposed therapy is a small molecule drug that will be delivered as a single injection into the affected knee. Once injected, the drug activates the patient’s own stem cells that are already in the knee, causing them to change into cells that produce the cartilage tissue that is lacking in OA joints. It’s this lack of joint cartilage that leads to most of the debilitating symptoms associated with OA, including joint swelling, pain and immobility. By recruiting the patient’s own stem cells and natural repair machinery to fix the cause of OA, our drug can repair the diseased joint and allow the patient the opportunity to return to a normal productive, active lifestyle. The drug itself is only present at appreciable levels in the joint and nowhere else in the body, making it extraordinarily safe.

This drug has proven safe and active in regenerating cartilage tissue in all preclinical models to date and we expect it to begin the clinical trial required by the US Food and Drug Administration in the first quarter of 2013. Depending on the results of those trials, the drug could be broadly available as early as 2017.

Statement of Benefit to California: 

The proposed research comprises the clinical development to demonstrate safety and efficacy of an interventional drug to treat Osteoarthritis (“OA”) and restore patients to a normal active lifestyle. OA is a serious degenerative disease involving joint cartilage and the underlying bone. It affects 3 million Californians and prevents an estimated 750 thousand Californians from performing their major daily activities of life, and its impact is growing fast with our aging population.

Our drug candidate offers the potential to regenerate cartilage tissue, repair the affected joint and relieve the patient of debilitating pain, thereby restoring many of these patients back to a state of well being, permitting them to return to their normal productive daily activities. Additionally, the economic cost of OA to the state of California is substantial, approximately $7-11 billion per year including direct medical, drug and work loss costs.

Due to the medical impact of OA on patients and its economic burden on our health care system, an approved drug for this disease would be valued highly across the nation and globally and could bring countless new jobs and tax revenue to the citizens and State of California. Furthermore, an early success in regenerative medicine in California would likely generate additional excitement and investment in the stem cell field, generating more jobs and helping to ensure California’s leadership in this important new industry.