Grant Award Details
- The objective of this project is to treat six patients in the Phase 1 portion of a Phase 1/2 clinical trial testing the use of placental mesenchymal stem cell-seeded dural matrix to surgically repair the defect in myelomeningocele patients in utero. The patients will be monitored for 30 months postnatally, assessing both safety and efficacy outcomes.
Grant Application Details
- The CuRe Trial: Cellular Therapy for In Utero Myelomeningocele Repair
Therapeutic Candidate or Device
Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM)
Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenatally
Placenta-derived mesenchymal stem cells (PMSCs) act by a paracrine mechanism, secreting a variety of growth factors, cytokines, and extracellular vesicles. This secretory profile is unique to PMSCs and is responsible for protecting motor neurons from apoptosis, which occurs due to chemical and mechanical trauma when motor neurons are exposed to the intrauterine environment. PMSC treatment increases the density of motor neurons in the spinal cord, leading to improved motor function.
Unmet Medical Need
The current standard of care in utero surgery, while promising, still leaves 58% of patients unable to walk independently. There is an extraordinary need for a therapy that prevents or lessens the severity of the devastating and costly lifelong disabilities associated with the disease.
Phase 1 trial completed
Major Proposed Activities
- Enrollment of 6 patients to demonstrate safety and preliminary efficacy of PMSC-ECM product
- FDA-compliant manufacturing and testing studies of the PMSC-ECM product
There is a high incidence of MMC in CA with 39.1% of the population being of Hispanic or Latino descent, a demographic that is affected by MMC at a disproportionately high rate. The cost to CA is approximately $532,000 per child, but for many, the cost may be several million dollars due to ongoing treatment. Indirect costs include pain and suffering, specialized childcare, and lost time of unpaid caregivers. A therapy for MMC would relieve the tremendous emotional and economic cost burden to CA.
- Bioact Mater (2023) A bioactive material with dual integrin-targeting ligands regulates specific endogenous cell adhesion and promotes vascularized bone regeneration in adult and fetal bone defects. (PubMed: 35663336)
- J Pediatr Surg (2022) Early investigations into improving bowel and bladder function in fetal ovine myelomeningocele repair. (PubMed: 35093254)
- J Surg Res (2022) Impact of Gestational Age on Neuroprotective Function of Placenta-Derived Mesenchymal Stromal Cells. (PubMed: 35093836)
- Bioengineering (Basel) (2022) Engineering Extracellular Microenvironment for Tissue Regeneration. (PubMed: 35621480)
- J Dev Biol (2022) Spina Bifida: A Review of the Genetics, Pathophysiology and Emerging Cellular Therapies. (PubMed: 35735913)
- Theranostics (2022) Engineered extracellular vesicles with high collagen-binding affinity present superior in situ retention and therapeutic efficacy in tissue repair. (PubMed: 35966577)
- Cells (2021) The Unique Properties of Placental Mesenchymal Stromal Cells: A Novel Source of Therapy for Congenital and Acquired Spinal Cord Injury. (PubMed: 34831060)