Osteoporosis, Bone and Cartilage Disease Fact Sheet

CIRM funds many projects seeking to better understand bone related diseases including osteoporosis, osteoarthritis and osteonecrosis to translate those discoveries into new therapies.

Description

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. 

UC Davis

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.

CIRM Grants Targeting Bone & Cartilage Disease

Researcher NameInstitutionGrant TitleGrant TypeAward Amount
Dmitriy SheynCedars-Sinai Medical CenterThe role of WNT and BMP signaling pathways in iPSC to iTenocyte step-wise differentiation for tendon repairFoundation – Discovery Stage Research Projects$1,516,563
Dr. Darryl D. D’LimaScripps HealthPluripotent Stem Cells for Tendon Tissue EngineeringQuest – Discovery Stage Research Projects$2,734,163
Dmitriy SheynCedars-Sinai Medical CenterMicrogel encapsulated iPSC-derived notochordal cells to treat intervertebral disc degeneration and low back painQuest – Discovery Stage Research Projects$2,020,166
Dr. Darryl D. D’LimaScripps HealthMeniscal Repair and RegenerationQuest – Discovery Stage Research Projects$1,361,775
Dr. Kevin StoneThe Stone Research Foundation for Sports Medicine and ArthritisA Novel Therapy for Articular Cartilage Autologous Cellular Repair by Paste GraftingQuest – Discovery Stage Research Projects$1,180,309
Alireza MoshaveriniaUniversity of California, Los AngelesGingival mesenchymal stem cells as a novel treatment modality for periodontal tissue regenerationInception – Discovery Stage Research Projects$194,483
Dmitriy SheynCedars-Sinai Medical CenterIVD rejuvenation using iPSC-derived notochordal cellsInception – Discovery Stage Research Projects$241,992
Dr. Ahmed Gamal IbrahimCedars-Sinai Medical CenterNoncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosisTherapeutic Translational Research Projects$2,590,224
Dr. Farhad Parhami Phd, MBAMAX BioPharma, Inc.Therapeutic development of Oxy200, an oxysterol with bone anabolic and anti-resorptive properties for intervention in osteoporosisTherapeutic Translational Research Projects$1,400,000
Dr. Denis A Evseenko Dr.University of Southern CaliforniaPluripotent stem cell-derived chondrocytes for articular cartilage repairTherapeutic Translational Research Projects$2,503,104
Ying ZhuAnkasa Regenerative Therapeutics, Inc.An autologous somatic stem cell therapy for the treatment of osteonecrosisTherapeutic Translational Research Projects$2,088,780
Professor Jeffrey Charles LotzUniversity of California, San Francisco2023 Center for Dental, Oral, & Craniofacial Tissue & Organ Regeneration (C-DOCTOR) Annual Spring ConferenceConference II$30,250
Dr Robert HayesImmusoft CorporationA Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1Clinical Trial Stage Projects$8,000,000
Dr. Thomas W Chalberg Jr.Genascence CorporationA Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OAClinical Trial Stage Projects$11,637,194
Dr. Gayatri RaoRocket Pharmaceuticals, Inc.A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)Clinical Trial Stage Projects$1,010,000
Dr. Kristen A JohnsonCalibrEvaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee OsteoarthritisClinical Trial Stage Projects$8,447,523
Kyriacos A AthanasiouUniversity of California, IrvineTreatment of the TMJ disc complexLate Stage Preclinical Projects$6,000,000
Frank PetriglianoUniversity of Southern CaliforniaPlurocart: a novel stem cell-based implant for articular cartilage restorationLate Stage Preclinical Projects$5,999,782
Ying ZhuAnkasa Regenerative Therapeutics, Inc.IND-enabling development of ART352-L, an endogenous stem cell reactivation therapy to enhance bone healing in the elderlyLate Stage Preclinical Projects$3,987,693
Jack J. WangCellular Biomedicine Group, Inc.Allogenic human adipose-derived mesenchymal stem cells for the treatment of knee osteoarthritisLate Stage Preclinical Projects$1,200,000
Peter G SchultzCalibrDevelopment of a Chondrogenic Drug Candidate Targeting Resident Mesenchymal Stem Cells for the Treatment of OsteoarthritisLate Stage Preclinical Projects$1,667,832
Peter G SchultzCalibrDevelopment of a Chondrogenic Drug Candidate Targeting Cartilage-residing Mesenchymal Stem Cells for the Treatment of OsteoarthritisPreclinical Development Awards$2,306,703
Dr. Darryl D. D’LimaScripps HealthEmbryonic Stem Cell-Derived Chondroprogenitor Cells to Repair Osteochondral DefectsPreclinical Development Awards$7,660,211
Dr. J. Kent LeachUniversity of California, DavisMulti-modal technology for non-destructive characterization of bioengineered tissuesTools and Technologies III$1,574,151
Dr. Fan Yang PhDStanford UniversityInjectable Macroporous Matrices to Enhance Stem Cell Engraftment and SurvivalTools and Technologies III$1,434,235
Dr. Dan GazitCedars-Sinai Medical CenterGene Targeting to Endogenous Stem Cells for Segmental Bone Fracture HealingEarly Translational IV$5,121,514
Nancy E. LaneUniversity of California, DavisTreatment of non-traumatic osteonecrosis with endogenous Mesenchymal stem cellsDisease Team Therapy Development – Research$18,227,898
Prof. Kyriacos A. Athanasiou Ph.DUniversity of California, DavisTissue engineered cartilage from autologous, dermis-isolated, adult, stem (DIAS) cellsEarly Translational III$1,735,703
Dr. Dan GazitCedars-Sinai Medical CenterGenetically Engineered Mesenchymal Stem Cells for the Treatment of Vertebral Compression Fractures.Disease Team Therapy Planning I$107,622
Nancy E. LaneUniversity of California, DavisIncreasing the endogenous mesenchymal stem cells to the bone surface to treat osteoporosisDisease Team Therapy Planning I$107,750
John HoodSamumed, LLCClinical Development of an osteoinductive therapy to prevent osteoporosis-related fracturesDisease Team Therapy Planning I$99,110
Dr. Dan GazitCedars-Sinai Medical CenterSystemic Adult Stem Cell Therapy for Osteoporosis-Related Vertebral Compression FracturesEarly Translational II$1,927,698
Prof. Bruno PeaultUniversity of California, Los AngelesHarnessing native fat-residing stem cells for bone regenerationEarly Translational II$5,359,076
Peter G. SchultzScripps Research InstituteCartilage Regeneration by the Chondrogenic Small Molecule PRO1 during OsteoarthritisEarly Translational II$6,047,249
Dr. Songtao ShiUniversity of Southern CaliforniaOral and Craniofacial Reconstruction Using Mesenchymal Stem CellsNew Faculty I$3,242,651
Dr. Jill HelmsStanford UniversityEnhancing healing via Wnt-protein mediated activation of endogenous stem cellsEarly Translational I$6,464,126
Dr. Darryl D. D’LimaScripps HealthStem Cell-Based Therapy for Cartilage Regeneration and OsteoarthritisEarly Translational I$3,118,431
Dr. Francesca MARIANIUniversity of Southern CaliforniaSkeletogenic Neural Crest Cells in Embryonic Development and Adult Regeneration of the JawNew Faculty II$2,247,403
Professor A. Hari ReddiUniversity of California, DavishESCs for Articular Cartilage RegenerationSEED Grant$301,703
Total:
$132,895,067.31

CIRM Bone & Cartilage Disease Videos

Jill Helms, Stanford – CIRM Stem Cell #SciencePitch

Edward Hsiao, UCSF – CIRM Stem Cell #SciencePitch

Songtao Shi, USC – CIRM Stem Cell #SciencePitch

Healing Bones with Stem Cells at UC Davis – Intro: CIRM Spotlight on Disease

Healing Bones with Stem Cells at UC Davis – Kent Leach: CIRM Spotlight on Disease

Healing Bones with Stem Cells at UC Davis – Mark Lee: CIRM Spotlight on Disease

Stem Cell Clinical Trial for Broken Bones – A Patient’s Story: CIRM Spotlight on Disease

Healing Bones with Stem Cells at UC Davis – Q&A: CIRM Spotlight on Disease

Fat-Derived Stem Cells for Tissue Repair: Michael Longaker – CIRM Science Writer’s Seminar

Resources

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