According to a story from pm360online.com, the speciality pharmaceutical company Chiesi USA and the biotech company Holostem Terapie Avanzate S.r.l. of Italy recently announced that the U.S. FDA recently granted orphan drug designation for its experimental treatment GPLSCD01. This product is intended for the treatment of limbal stem cell deficiency. In effect, GPLSCD01 is intended as a supplement to help replace the deficiency of stem cells.
Limbal stem cell deficiency (LSCD) describes a condition of an insufficient number of corneal epithelial stem cells, or limbal stem cells. These cells form a border between the white part of the eye and the cornea. Limbal stem cells are essential for maintaining the structure and health of the cornea; they can replace the entirety of the corneal epithelium, and replace cells lost in tears. Limbal stem cell deficiency can be caused by congenital conditions or environmental factors, such as infections, certain drugs, burns, chemicals, surgery, and contact lenses. Symptoms include edema, eye pain, conjunctivalisation, vascularisation of the cornea, visual impairment, and blindness. When caused by injury, treatment usually starts with artificial tears, eye drops, and “bandage” contact lenses, followed by surgery. A transplant of limbal tissue may be necessary. To learn more about limbal stem cell deficiency, click here.
GPLSCD01 offers the potential to provide supplemental limbal stem cells to patients in order to repair limbal stem cell deficiency without the need for a transplant procedure. This would represent a major improvement in treatment for these patients, who would no longer have to expect an invasive and expensive transplant operation and would at least have the option for an alternative. Whether the therapy can fully replace transplant options has yet to be determined.
