Research for a new treatment for retinitis pigmentosa

Research for a new treatment for retinitis pigmentosa (RP), a genetic disease affecting an average of one person every 3,500-5,000 is currently being carried out in Italy.

This line of investigation originates from the research of Rita Levi Montalcini, winner of the 1986 Nobel Prize for Medicine, into the identification of nerve growth factors. The work relating to the studies of this eminent scientist is being continued by Dompé, Italian biopharmaceutical company engaged in the research of new therapeutic solutions for the treatment of rare and orphan diseases, which is developing rhNGF (recombinant human Nerve Growth Factor) for ophthalmic use.

The molecule – currently in clinical development for the treatment of neurotrophic keratitis (a rare disease of the cornea) in the Reparo study, involving 158 patients in 39 centres in nine European countries – is now being evaluated for its safety profile and to obtain preliminary efficacy data, as part of the Lumos study, involving five centres of excellence in Italy. The first patient was enrolled at the Careggi University Hospital in Florence.

Lumos is a Phase Ib/II, multicentre, randomised, double-masked, placebo-controlled study that aims to assess the safety and potential efficacy of rhNGF eye drops in two doses (60 and 180 µg/ml), compared to a placebo. The research will involve 50 patients, who will be enrolled at five centres in Florence, Milan, Rome (two centres) and Naples, internationally recognised for their expertise in this therapeutic area. Patients with typical retinitis pigmentosa will be divided into three groups. The first will be given eye drops at the lower dose, the second an increased dose and the third placebo eye drops. The monitoring schedule for each patient is 24 weeks.

“Retinitis pigmentosa is a great challenge for ophthalmology, because it is a disease which still has no cure”, explains Francesca Simonelli, director of clinical ophthalmology at the Second University of Naples. “In patients who suffer from this chronic disease, the photoreceptors (retinal cells called cones and rods whose task it is to capture visual signals and send them the brain) are damaged and undergo progressive degeneration, there being no way of stopping the pathological process. We are, therefore, very interested in potential treatments such as that offered by rhNGF, which could lead to new perspectives in the treatment of retinitis pigmentosa”.

rhNGF has been developed by Dompé and is produced at their L’Aquila facilities using recombinant DNA technology, that is, through the transfer to bacteria of human genetic material. Bacteria become the drug “factory,” producing material equivalent to the growth factor produced by the human body.

“We are proud to announce the start of the Lumos study and the news of the first enrolment”, explains Eugenio Aringhieri, CEO of the Dompé Group. “This is further evidence of our commitment to protecting the patient, in researching therapeutic solutions in areas of high demand for healthcare, such as the treatment of rare and often orphan diseases. Commitment to research and development is at the heart of our Group objectives and our mission, which is directly related to the unsatisfied therapeutic needs of patients throughout the world. We will continue to focus on our ability to generate innovation, making use of the fundamental contributions of a network of 70 international centres of excellence, with the aim of making significant advances in respect of currently available therapeutic solutions, in areas such as ophthalmology, organ transplant and oncology”.

Click here for more information about rhNGF and Retinitis Pigmentosa.

Click here for more information about Dompé.