Rare diseases

Hope for patients with Fuchs’ Endothelial Corneal Dystrophy: Innovations in treatment

31
May

Today we talk in our blog about another of the rare diseases that the OLIGOFASTX consortium is working on: Fuchs’ Corneal Endothelial Endothelial Dystrophy (FECD). This pathology affects approximately 4% of the population over 40 years of age, and much more women than men.

Results from miRNA expression profiling in FECD endothelial cells demonstrates widespread miRNA downregulation in FECD that may be associated with increased subendothelial extracellular matrix accumulation. Since there is no effective treatment that restores vision or prevents blindness without resorting to corneal transplantation, OLIGOFASTX and its partners, in particular Arthex Biotech, are investigating over- or under-regulated microRNAs in FECD models to develop and validate an anti-miR treatment for this disease.

What is Fuchs Endothelial Corneal Dystrophy and what is its impact on vision?

Fuchs’ Corneal Endothelial Dystrophy is a chronic eye disease that affects the endothelial cell layer of the cornea. This disease can cause a gradual decrease in vision and, in some cases, even blindness.

Fuchs’ Endothelial Corneal Dystrophy is more common in women and may be hereditary. Symptoms include blurred vision, sensitivity to light and halos around lights. As the disease progresses, corneal edema may develop, which can further compromise vision. Fortunately, there are innovations in treatment that are offering hope to patients with Fuchs’ Endothelial Corneal Dystrophy.

Treatments include endothelial cell transplantation, gene and cell therapies and oligonucleotide-based therapy. These treatments could help restore vision and improve patients’ quality of life.

It is important that patients with Fuchs’ Endothelial Corneal Dystrophy undergo regular eye examinations and work closely with their ophthalmologist to determine the best treatment plan for their individual situation. With the help of medical innovation and proper medical care, patients can live full and active lives despite their eye disease.

Conventional treatment: Corneal transplantation and its limitations

The conventional treatment for Fuchs’ Endothelial Corneal Dystrophy is corneal transplantation, but this procedure has limitations. First, the availability of corneal donors is limited and there may be a long waiting list to receive a suitable cornea.

In addition, corneal transplantation is an invasive procedure that requires surgery and a long recovery time. There is also the risk of transplant rejection and the need to take immunosuppressive drugs for life.

Fortunately, there are innovations in treatment that offer hope for patients with Fuchs’ Endothelial Corneal Dystrophy. Cell therapy and tissue engineering are in development and may provide an alternative to corneal transplants. In addition, gene therapy is also being investigated as a possible cure for this disease. We cannot forget oligonucleotide-based therapies, one of which is being developed at OLIGOFASTX. These treatment innovations offer hope to patients and can significantly improve their quality of life. It is important to continue researching and developing new treatment options to improve the lives of people affected by Fuchs’ Endothelial Corneal Dystrophy.

Innovations in treatment

Fuchs’ Endothelial Corneal Dystrophy is an eye disease that affects the innermost layer of the cornea, the endothelium, and can lead to vision loss. Fortunately, thanks to new innovations in treatment, such as the development of specific oligonucleotides or cell therapy with stem cells and growth factors, new hope has opened up for patients suffering from this disease.

The OLIGOFASTX consortium has on its radar the development of oligonucleotide-based therapies to improve the quality of life of patients affected by this rare disease.

As we have already told you in this blog, oligonucleotides are RNA or DNA molecules naturally present in the organism or synthetic molecules capable of performing multiple functions. Applied to the therapeutic field, RNA oligonucleotides are paired with disease-related target messenger RNAs and elicit a physiological effect.

In the case of rare diseases, oligonucleotides are a very interesting option because many of these pathologies are caused by specific genetic mutations. In the case applied to the treatment proposed in OLIGOFASTX, the RNA oligonucleotide to be developed must be able to block miRNAs (small molecules that regulate a messenger RNA) related to the development of Fuchs Corneal Dystrophy disease.

Cell therapy consists of introducing stem cells into the cornea to convert them into healthy endothelial cells and repair the existing damage; On the other hand, growth factors are substances that stimulate tissue growth and regeneration and are applied in the form of eye drops. Both techniques have been shown to be effective in improving vision and reducing symptoms of Fuchs’ Endothelial Corneal Dystrophy. Although they are still in the research phase, it is hoped that in the near future they can be used more widely for the treatment of this disease and improve the quality of life of affected patients.

In conclusion, Fuchs’ Endothelial Corneal Dystrophy is an eye disease that affects many people worldwide. Fortunately, treatment innovations are now being developed that offer an alternative to conventional corneal transplantation and are a step towards personalized and more effective medicine.

In addition, personalized medicine will make it possible to tailor treatment to the specific needs of each patient, which will further increase its effectiveness. However, it is important to continue researching and developing new techniques to improve the quality of life of patients with Fuchs’ Endothelial Corneal Dystrophy. The hope of a definitive cure for this disease is still alive, and thanks to technological and medical advances, it is getting closer and closer to becoming a reality.

At OLIGOFASTX we continue working and researching in this direction and with this objective in mind.

Sources:

  1. Oligofastx: https://oligofastx.com/es/arthex/
  2. Arthex Biotech: https://www.arthexbiotech.com/
  3. Sociedad Española de Oftalmología: https://www.oftalmo.com/seccion/distrofia-corneal-de-fuchs
  4. Asociación Argentina de Oftalmología: https://www.oftalmologos.org.ar/paciente/distrofia-corneal-de-fuchs-n-414
  5. Clínica Mayo: https://www.mayoclinic.org/es-es/diseases-conditions/fuchs-dystrophy/symptoms-causes/syc-20353850
  6. MedlinePlus: https://medlineplus.gov/spanish/ency/article/001656.htm
  7. National Eye Institute: https://www.nei.nih.gov/es/health/corneal-dystrophies