Development of a Targeted Therapy to Repair Osteoporosis-Related Hip Fractures

Funding Type: 
Disease Team Therapy Development - Research
Grant Number: 
DR2A-05368
Investigator: 
ICOC Funds Committed: 
$0
Public Abstract: 
We propose the development of a stem cell-targeted drug therapy to improve and accelerate bone healing in patients with osteoporotic hip fractures. Hip fractures are a devastating consequence of osteoporosis striking 1.5 million Americans and 180,000 Californians annually. Among these patients, approximately 375,000 patients in the US and 45,000 patients in California die within one year of their injury. This debilitating and potentially deadly disease-related injury currently has no effective treatment. Current treatment is typically orthopedic surgery to attempt to join the bones, 1-2 weeks of hospitalization followed by extensive rehabilitation and commonly, admission to a nursing home. Current treatment is painful, does nothing to stimulate bone regeneration, often fails, and incurs significant risks of mortality and morbidity. Standard of care leaves large numbers of hip fracture patients in intractable pain, unable to return to normal activities and at significant risk for recurrent fractures. If fracture union is not achieved, the patient may suffer long-term disability. The unmet clinical need for the population of osteoporosis patients with debilitating fracture is enormous and growing with our aging demographics. Bone is often thought of as a permanent substance that does not change after childhood. However, bone is alive, dynamic and constantly being renewed though a process of the breakdown of old bone and the formation of new bone. As we age, more bone is lost than generated which can lead to osteoporosis, a disease of the bone that greatly increases the bone's fragility. Upon successful development of this project, the drug will drive the patient’s own stem cells at the site of the injury to change into bone forming cells that will build and strengthen the affected bone to speed the healing process and reduce the risk of future fractures. This project will take an already identified compound that is currently under manufacture, through non-clinical and clinical safety studies as well as preliminary efficacy studies as required by the Food and Drug Administration (FDA). This includes supporting data for filing an investigational new drug (IND) application to enter clinical studies and Phase I/II safety and efficacy clinical trials in patients within this grant’s time frame of four years. The goal of this project is to significantly advance development of a medication that will be administered when a patient has incurred a fracture and is undergoing the current standard of care, orthopedic surgery. Utilizing the body’s own cellular machinery, by inducing stem cells to build localized bone at the point of fracture, is a therapeutic strategy with a high likelihood of success with a low probability of side effects. This proposal offers a therapy that will significantly improve hip fracture patient’s post-surgical prognosis, revolutionizing the treatment of hip fractures.
Statement of Benefit to California: 
The aim of this project is to advance the clinical development of a stem cell targeted therapy to improve and accelerate bone healing in osteoporotic hip fracture patients through Phase I/II clinical trials. Osteoporosis is a disease of the bone that increases bone fragility and the possibility of fracture. Hip fractures are a devastating consequence of osteoporosis striking approximately 180,000 Californians annually at an estimated cost of approximately $2 billion. Of these patients, an estimated 45,000 Californians die within one year of their injury. There is no approved drug to treat this scourge of California's aging population. Current hip fracture treatment is typically orthopedic surgery to attempt to join the bones, 1-2 weeks of hospitalization followed by extensive rehabilitation and commonly, admission to a nursing home. Current treatment is painful, does nothing to stimulate bone regeneration, often fails, and incurs significant risks of mortality and morbidity. Standard of care leaves large numbers of hip fracture patients in intractable pain, unable to return to normal activities and at significant risk for recurrent fractures. If fracture union is not achieved, the patient may suffer long-term disability. The clinical unmet need for the population of California’s osteoporosis patients with debilitating fracture is enormous and growing with the aging demographics of our society. This project will advance development of a compound that is already identified and currently under manufacture, through non-clinical and clinical safety studies as well as preliminary efficacy studies as required by the Food and Drug Administration (FDA). The drug will be administered at the time of fracture when a patient is undergoing the current standard of care, orthopedic surgery. Successful development of this therapy will revolutionize the treatment of osteoporotic hip fractures; not only saving lives, but also significantly improving thousands of Californian’s lives every year after sustaining a hip fracture. This therapy will also contribute to a substantial cost savings for the State of California’s healthcare budget.

© 2013 California Institute for Regenerative Medicine