Bone Marrow Mesenchymal Stem Cell (BMSC)-Derived Exosomes for the Treatment of Glaucoma

Glaucoma is one of the world’s leading causes of irreversible blindness. There is no cure and vision lost from glaucoma cannot be restored. Glaucoma is associated with fluid build-up in the eye resulting in an increased intraocular pressure (IOP). The pressure may cause damage to the optic nerve and lead to progressive degeneration of retinal ganglion cells (RGC) and vision loss. Currently, available treatments for glaucoma delay progression by reducing IOP, but no therapies exist to directly protect RGC from degradation and loss. 

Scientists at the National Eye Institute (NEI) have developed a method to treat glaucoma using exosomes derived from bone marrow-derived mesenchymal stem cells (BMSC).  BMSC‐derived exosome administration for glaucoma may confer a significant neuroprotective effect for RGC and prevent vision loss. Isolated exosomes have several immediate advantages for clinical translation compared to potential whole-cell (stem cell) therapies for glaucoma. Isolation and purification of BMSC‐derived exosomes, or their therapeutically active components, is relatively simple via centrifugation. BMSC‐derived exosomes are stable, and once isolated, can be stored at 4C for months to years. Prior clinical trials of BMSC‐derived exosomes administered systemically have shown a positive safety profile. These exosomes are immunologically inert and, due to their small size and stability, are also easy to dose & deliver and will also readily diffuse from the vitreous into the retinal cell layers.  Significant therapeutic- neuroprotective effects for isolated BMSC‐derived exosomes have been shown in in vitro and in vivo glaucoma models. BMSC‐derived exosomes are a promising potential cell‐free therapy for glaucoma and degenerative ocular diseases associated with loss of RGC.

Competitive Advantages:

• More inexpensive than bone marrow-derived mesenchymal stem cells (BMSC)
• Safer than BMSC
• BMSC and BMSC exomes represent the only known treatments for protecting RGC

Commercial Applications:

• Therapeutic to treat glaucoma
• Therapeutic to treat degenerative ocular diseases associated with loss of retinal ganglion cells (RGC)