Grant Award Details
Conduct a phase 1 trial to assess the safety and potential efficacy of limbal stem cell therapy to regenerate a normal corneal surface (autologous product manufacturing, trial conduct and diagnostic assay development)
Grant Application Details
- Regeneration of a Normal Corneal Surface by Limbal Stem Cell Therapy
Therapeutic Candidate or Device
cultivated patient-specific corneal epithelial stem cells (limblal stem cells, LSC)
Corneal blindness from inability to heal due to corneal epithelial stem cell deficiency as a results of injury
Limbal stem cell deficiency (LSCD) leads to inability to heal. The most desired treatment is to replace the necessary amount of the stem cell to maintain a normal, transparent corneal surface. Any remaining LSCs will be identified and biopsy from the patient to be expanded in culture. Once sufficient amount of LSCs are produced, these LSCs will then transplanted back to patient's eye to restore a normal corneal surface.
Unmet Medical Need
Cultivated LSCs has been shown to be effective and a safer treatment than direct transplantation for LSCD since 1997 in Europe. This stem therapy is not available in the United States. Our therapy will be the first patient-specific stem cell therapy to treat both unilateral and bilateral LSCD.
IND filling and ready for phase 1 trial
Major Proposed Activities
- LSC manufacture development and certification
- Establishment of manufacture process in GMP facility
- biomarker development
California is the most populated state in the USA. The number of residents with LSCD may disproportionately increase as a result of multiple environmental risk factors. A safe treatment to restore vision would be an important benefit to the people of California.
Our project will further benefit California through the training of new stem-cell researchers, create more jobs, and attract funding from the federal government and investment from the private sector.
- Cornea (2019) Classification of Limbal Stem Cell Deficiency Using Clinical and Confocal Grading. (PubMed: 30371569)
- Ocul Surf (2019) The application of human amniotic membrane in the surgical management of limbal stem cell deficiency. (PubMed: 30633967)
- Surv Ophthalmol (2019) Diagnostic criteria for limbal stem cell deficiency before surgical intervention-A systematic literature review and analysis. (PubMed: 31276736)
- Stem Cells Transl Med (2019) Mesenchymal Stem Cells Reduce Corneal Fibrosis and Inflammation via Extracellular Vesicle-Mediated Delivery of miRNA. (PubMed: 31290598)
- Sci Rep (2019) Notch Inhibition Prevents Differentiation of Human Limbal Stem/Progenitor Cells in vitro. (PubMed: 31316119)
- Transl Vis Sci Technol (2018) In Vivo Evaluation of the Limbus Using Anterior Segment Optical Coherence Tomography. (PubMed: 30112250)
- Ocul Surf (2018) The diagnosis of limbal stem cell deficiency. (PubMed: 29113917)
- Cornea (2018) A Case of Corneal Neovascularization Misdiagnosed as Total Limbal Stem Cell Deficiency. (PubMed: 29781927)
- Cornea (2017) Characterization of the Corneal Subbasal Nerve Plexus in Limbal Stem Cell Deficiency. (PubMed: 27941384)
- Clin Exp Ophthalmol (2017) Correlation between the existence of the palisades of Vogt and limbal epithelial thickness in limbal stem cell deficiency. (PubMed: 27591548)
- Cornea (2016) Existence of Normal Limbal Epithelium in Eyes With Clinical Signs of Total Limbal Stem Cell Deficiency. (PubMed: 27362882)
- Stem Cell Res (2016) A 3D culture system enhances the ability of human bone marrow stromal cells to support the growth of limbal stem/progenitor cells. (PubMed: 26896856)
- Am J Ophthalmol (2015) Epithelial Thinning in Limbal Stem Cell Deficiency. (PubMed: 26163009)
- Am J Ophthalmol (2015) Limbal Basal Cell Density Decreases in Limbal Stem Cell Deficiency. (PubMed: 26149968)