Every year millions of Americans suffer damage to their cartilage, either in their knee or other joints, that can eventually lead to osteoarthritis, pain and immobility. Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved two projects targeting repair of damaged cartilage.
The projects were among 17 approved by CIRM as part of the DISC2 Quest Discovery Program. The program promotes the discovery of promising new stem cell-based and gene therapy technologies that could be translated to enable broad use and ultimately, improve patient care.
Dr. Darryl D’Lima and his team at Scripps Health were awarded $1,620,645 to find a way to repair a torn meniscus. Every year around 750,000 Americans experience a tear in their meniscus, the cartilage cushion that prevents the bones in the knee grinding against each other. These injuries accelerate the early development of osteoarthritis, for which there is no effective treatment other than total joint replacement, which is a major operation. There are significant socioeconomic benefits to preventing disabling osteoarthritis. The reductions in healthcare costs are also likely to be significant.
The team will use stem cells to produce meniscal cells in the lab. Those are then seeded onto a scaffold made from collagen fibers to create tissue that resembles the knee meniscus. The goal is to show that, when placed in the knee joint, this can help regenerate and repair the damaged tissue.
This research is based on an earlier project that CIRM funded. It highlights our commitment to helping good science progress, hopefully from the bench to the bedside where it can help patients.
Dr. Kevin Stone and his team at The Stone Research Foundation for Sports Medicine and Arthritis were awarded $1,316,215 to develop an approach to treat and repair damaged cartilage using a patient’s own stem cells.
They are using a paste combining the patient’s own articular tissue as well as Mesenchymal Stem Cells (MSC) from their bone marrow. This mixture is combined with an adhesive hydrogel to form a graft that is designed to support cartilage growth and can also stick to surfaces without the need for glue. This paste will be used to augment the use of a microfracture technique, where micro-drilling of the bone underneath the cartilage tear brings MSCs and other cells to the fracture site. The hope is this two-pronged approach will produce an effective and functional stem cell-based cartilage repair procedure.
If effective this could produce a minimally invasive, low cost, one-step solution to help people with cartilage injuries and arthritis.
The full list of DISC2 grantees is:
|Application||Title||Principal Investigator and Institution||Amount|
|DISC2-13212||Preclinical development of an exhaustion-resistant CAR-T stem cell for cancer immunotherapy||Ansuman Satpathy – Stanford University||$ 1,420,200|
|DISC2-13051||Generating deeper and more durable BCMA CAR T cell responses in Multiple Myeloma through non-viral knockin/knockout multiplexed genome engineering||Julia Carnevale – UC San Francisco||$ 1,463,368|
|DISC2-13020||Injectable, autologous iPSC-based therapy for spinal cord injury||Sarah Heilshorn – Stanford University||$789,000|
|DISC2-13009||New noncoding RNA chemical entity for heart failure with preserved ejection fraction.||Eduardo Marban – Cedars-Sinai Medical Center||$1,397,412|
|DISC2-13232||Modulation of oral epithelium stem cells by RSpo1 for the prevention and treatment of oral mucositis||Jeffrey Linhardt – Intact Therapeutics Inc.||$942,050|
|DISC2-13077||Transplantation of genetically corrected iPSC-microglia for the treatment of Sanfilippo Syndrome (MPSIIIA)||Mathew Blurton-Jones – UC Irvine||$1,199,922|
|DISC2-13201||Matrix Assisted Cell Transplantation of Promyogenic Fibroadipogenic Progenitor (FAP) Stem Cells||Brian Feeley – UC San Francisco||$1,179,478|
|DISC2-13063||Improving the efficacy and tolerability of clinically validated remyelination-inducing molecules using developable combinations of approved drugs||Luke Lairson – Scripps Research Inst.||$1,554,126|
|DISC2-13213||Extending Immune-Evasive Human Islet-Like Organoids (HILOs) Survival and Function as a Cure for T1D||Ronald Evans – The Salk Institute for Biological Studies||$1,523,285|
|DISC2-13136||Meniscal Repair and Regeneration||Darryl D’Lima – Scripps Health||$1,620,645|
|DISC2-13072||Providing a cure for sphingosine phosphate lyase insufficiency syndrome (SPLIS) through adeno-associated viral mediated SGPL1 gene therapy||Julie Saba – UC San Francisco||$1,463,400|
|DISC2-13205||iPSC-derived smooth muscle cell progenitor conditioned medium for treatment of pelvic organ prolapse||Bertha Chen – Stanford University||$1,420,200|
|DISC2-13102||RNA-directed therapy for Huntington’s disease||Gene Wei-Ming Yeo – UC San Diego||$1,408,923|
|DISC2-13131||A Novel Therapy for Articular Cartilage Autologous Cellular Repair by Paste Grafting||Kevin Stone – The Stone Research Foundation for Sports Medicine and Arthritis||$1,316,215|
|DISC2-13013||Optimization of a gene therapy for inherited erythromelalgia in iPSC-derived neurons||Ana Moreno – Navega Therapeutics||$1,157,313|
|DISC2-13221||Development of a novel stem-cell based carrier for intravenous delivery of oncolytic viruses||Edward Filardo – Cytonus Therapeutics, Inc.||$899,342|
|DISC2-13163||iPSC Extracellular Vesicles for Diabetes Therapy||Song Li – UC Los Angeles||$1,354,928|