Name:
Institution:
Type:
PI
Grant Award Details
Placental Mesenchymal Stem Cell Augmentation of Fetal Myelomeningocele Repair
Grant Type:
Grant Number:
CLIN1-11404
Project Objective:
Conduct activities to successfully filed an IND and begin clinical start-up activities.
Investigator:
Disease Focus:
Neurological Disorders
Spina Bifida
Human Stem Cell Use:
Adult Stem Cell
Award Value:
$5,666,077
Status:
Active
Grant Application Details
Application Title:
- Placental Mesenchymal Stem Cell Augmentation of Fetal Myelomeningocele Repair
Public Abstract:
Therapeutic Candidate or Device
Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM)
Indication
Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenataly
Therapeutic Mechanism
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.
Project Objective
IND filing, Phase 1/2 trial start-up activities
Major Proposed Activities
- Manufacture product to supply the proposed studies and clinical trial
- Assess safety of the therapeutic PMSC product
- Assess efficacy using clinical-grade product
Statement of Benefit to California:
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.
