Novel treatment for retinitis pigmentosa entering clinical trial (21 February 2019)

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On 21 February, KyotoU Hospital's associate professor Hanako Ikeda announced that her team will begin an investigator-initiated clinical trial 1 March to evaluate the potential efficacy of branched-chain amino acids (BCAAs) against retinitis pigmentosa. Members include the Graduate School of Medicine's Dr Akitaka Tsujikawa, a professor of ophthalmology, and Tomoko Hasegawa, a researcher.

Retinitis pigmentosa, or RP, is a group of eye disorders involving the degeneration of photoreceptor cells in the retina, leading to loss of peripheral vision and poor eyesight. There is currently no effective treatment to halt the progression of these conditions, which in Japan represent the third leading cause of blindness and are estimated to afflict 30,000 individuals.

In a world-first discovery, Professor Ikeda and her colleagues found that BCAAs can prevent the death of retinal neurons in mouse models of RP. The compounds have long been used in Japan for medical treatment, but not for eye diseases.

The planned clinical trial will involve orally administering BCAAs to RP patients, and monitoring the progression of visual field loss in the subjects.

This plan was developed with support from the University Hospital's Institute for Advancement of Clinical and Translational Science (IACT), and evaluated by the Hospital's institutional review board, before being submitted to the Pharmaceuticals and Medical Devices Agency (PMDA) for final approval.

P articipants will be selected from those who took part in the recently concluded naturalistic-observational studies, in which subjects were observed without treatment.

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