Seeking new horizons
Dompé is currently committed to researching and developing innovative treatment solutions in ophthalmology.
This process starts from the study and development of a biotechnological drug based on NGF – the product of the research conducted by Nobel Prize winner Rita Levi Montalcini – as a potential therapeutic response to rare eye diseases, like neurotrophic keratitis and retinitis pigmentosa, and goes on to focus on solutions for the treatment of more common conditions like dry eye.
The journey of a “Nobel prize winning” molecule
In many cases, rare eye diseases constitute a healthcare need that currently available treatments are unable to meet. This is why Dompé is particularly committed to developing innovative therapeutic approach through studies on the Nerve Growth Factor (NGF).
NGF is a soluble protein that stimulates neuron growth, maintenance and survival in the central nervous system. Discovered by Nobel prize winner Rita Levi Montalcini, it was the first neurotrophic factor to be identified and purified; over the last fifty years, its role in neuron development and its biological activity on neuronal tissues have been of great interest to science.
Dompé's R&D centre in L'Aquila has been set up for the biotechnological production of a drug for clinical use based on recombinant human NGF (rhNGF).
rhNGF: clinical trials and new scenarios
Following the encouraging results of the first clinical observations on more than 100 patients with neurotrophic keratitis treated with murine NGF1, Dompé has started clinical trials concerning various ophthalmological disorders.
In the treatment of neurotrophic keratitis, a rare degenerative eye disease for which a treatment is not available, a study named REPARO is currently in progress. The randomized, double blind, placebo controlled study involves 37 centres in 9 European countries.
The disease, which in its more severe forms affects less than one person out of 5,000 worldwide2, causes a progressive damage of the cornea, which may even lead to its puncturing and consequent loss of vision. As the cornea is the most innervated organ in the human body, the study aims to investigate the potential recovery of an adequate nerve response, the positive outcome of which would result in the healing and preservation of the cornea's integrity.
The dry eye syndrome, which affects the ocular surface, is another condition for which Dompé is evaluating the use of rhNGF through a Phase II study.
The rhNGF development process is not confined to its possible use in the anterior segment of the eye, but also includes disorders that affect the posterior segment. In particular, after rhNGF was designated as an orphan drug in the treatment of retinitis pigmentosa by the FDA and the EMA, a Phase Ib/II multicentre clinical study named LUMOS was started.
Retinis pigmentosa affects on average one person out of 3,500-5,0003, particularly among young adults. In patients suffering from this chronic disease, the photoreceptors (i.e. the retinal cells known as cones and rods, responsible for capturing visual signals) are damaged and subject to progressive degeneration. So far, there are no satisfactory treatments able to slow down its evolution and restore patients' vision.
The possible action of the molecule on the retina opens up new potential for optical nerve diseases, such as glaucoma.
1 Fonti: Bonini S, Lambiase A, Rama P, Caprioglio G, Aloe L., Topical treatment with nerve growth factor for neurotrophic keratitis, Ophthalmology 2000; 107:1347-51
Lambiase A, Rama P, Bonini S, Caprioglio G, Aloe L., Topical treatment with nerve growth factor for neurotrophic corneal ulcers, New Engl J Med 1998; 338:1174-80
2 Fonte: Sacchetti M., Lambiase A., Diagnosis and management of neurotrophic keratitis. Clinical Ophthalmology 2014; 8: 571–579.
3 Fonte: Anasagasti A, Irigoyen C, Barandika O et al., Current mutation discovery approaches in retinitis pigmentosa, Vision Res. 2012; 75: 117-129.