Grant Award Details
Conduct activities to successfully filed an IND and begin clinical start-up activities.
Grant Application Details
- Placental Mesenchymal Stem Cell Augmentation of Fetal 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 prenataly
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.
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
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.
- J Pediatr Surg (2019) In Utero Treatment of Myelomeningocele with Placental Mesenchymal Stromal Cells - Selection of an Optimal Cell Line in Preparation for Clinical Trials. (PubMed: 31672407)
- Fetal Diagn Ther (2019) Spinal Angulation: A Limitation of the Fetal Lamb Model of Myelomeningocele. (PubMed: 30970373)
- FASEB J (2019) Neuroprotective effect of placenta-derived mesenchymal stromal cells: role of exosomes. (PubMed: 30753093)