Drugs for Retinal Degeneration
The DRUGSFORD project aims at finding new ways to counteract hereditary photoreceptor degeneration. DRUGSFORD is supported by the EU 7th framework programme and has started on 1st September, 2012.
The retina sits in the back of our eyes and is a tissue that we depend on for our ability to see. Within the retina, the photoreceptor cells are responsible for capturing light and for transforming it into messages that can be sent to and interpreted by the brain. Photoreceptors are nerve cells, and will not be replaced when lost, which means that if they die, the retina loses its ability to capture light forever, with dramatic consequences for vision.
Hereditary loss of photoreceptors, or hereditary photoreceptor degeneration, is a collective term, which describes a group of diseases leading to severe visual impairment and blindness. Among these diseases there are several important subgroups such as Retinitis Pigmentosa (RP), Lebers Congenital Amaurosis (LCA), or Achromatopsia. Altogether, these diseases are considered the most common cause for visual loss in the working population, and it is estimated that they today affect 250.000 people in Europe alone. Hereditary photoreceptor degenerations are caused by mutations in the genetic material (DNA), and are passed on from parent to child. While the mutations behind many of the disease types are known, there are yet no treatments available for these feared disabilities.
A major difficulty for therapy development is the high number of disease causing mutations. While a particular therapeutic approach may be beneficial for one patient, it may be useless for most other patients and could even be detrimental in some cases. Another difficulty lies in the fact that the retina is shielded from most drugs by the so called blood-brain-barrier. It is these two important problems that the DRUGSFORD project addresses:
In many different types of hereditary photoreceptor degeneration the regulation of an intracellular signaling molecule named cGMP is disturbed. Such a common degenerative process may constitute an ideal target for therapeutic intervention. Indeed, there are several scientific reports showing that the degeneration may be halted or slowed down if the effects of cGMP are rebalanced. DRUGSFORD will have access to novel cGMP regulating substances and these will be combined with drug delivery techniques that will help the compounds to cross the blood-brain-barrier and to reach the photoreceptors, so that these can be protected.
The DRUGSFORD consortium consists of two biotech companies and three academic research groups. The German company BIOLOG (Bremen) is a world leader in developing and producing compounds that interact with various cGMP-governed cellular reactions. The Dutch company to-BBB (Leiden) has developed specialized drug delivery systems that help pharmaceutical substances pass across the blood-brain-barrier. The combined expertise of these two companies allows the project to come up with novel drugs and ways to deliver them across the blood-retina-barrier, so that they can reach the photoreceptors. The academic research groups are connected to Valeria Marigo (University of Modena and Reggio Emilia, in Modena, Italy), Per Ekström (Lund University, in Lund, Sweden) and François Paquet-Durand (Eberhard Karls University, in Tübingen, Germany), and have already previously collaborated regarding photoreceptor degeneration mechanisms and the protective effect of treatments with various substances. The academic groups will test the drug and delivery systems for their protective effects on diseased photoreceptors and retinas.
Together, the DRUGSFORD consortium members aim at having a photoreceptor protective drug and delivery system ready for initial clinical trials by the finalization of the three-year project period.