The University of California and Regeneron Pharmaceuticals announced yesterday that they have received a US patent covering the use of a growth factor treatment for such retinal diseases as retinitis pigmentosa and macular degeneration.

The patent is based on the work of UCSF researchers Matthew M. LaVail, PhD, professor of anatomy and ophthalmology, and the late Roy H. Steinberg, PhD, professor of physiology and ophthalmology, and Regeneron scientists.

The company is developing its second-generation growth factor -- a ciliary neurotrophic factor (CNTF) molecule known as AXOKINE -- for retinitis pigmentosa, a degenerative disease involving photoreceptors. The disease affects approximately 125,000 people in the US. It is an inherited disorder in which photoreceptors -- the light sensitive elements in the back of the eye -- cease functioning, degenerate and die. Because the photoreceptors responsible for vision in dim light are the first to disappear, retinitis pigmentosa is commonly first recognized as “night blindness.” Eventually, its victims suffer from progressive narrowing of the field of vision and become totally blind. Mutations of a number of genes have been found to cause the disease.

Studies by LaVail and Steinberg have shown that CNTF can retard the functional loss of photoreceptors in rats bearing abnormal human retinitis pigmentosa genes. Regeneron is planning to conduct clinical trials to determine whether AXOKINE is safe and can have the same effect in human retinitis pigmentosa sufferers.

1st appeared 9/18/97