Cystic fibrosis (CF) is a life-threatening genetic disease that affects the lungs, pancreas, and other organs. It is caused by mutations in the CFTR gene, which encodes a protein that helps regulate the flow of salt and water in and out of cells.

In recent years, a new canine disease called degenerative myelopathy (DM) has emerged that shares some similarities to CF. DM is a progressive neurological disease that affects the spinal cord and leads to paralysis. It is caused by a mutation in the SOD1 gene, which encodes a protein that helps protect cells from damage.

Researchers have found that the SOD1 mutation in dogs with DM leads to changes in the function of the CFTR protein. This suggests that there may be a link between CF and DM, and that studying DM could help us to better understand CF.

One of the most promising findings from research on DM is that a drug called PXT3003 has been shown to slow the progression of the disease in dogs. PXT3003 is a small molecule that inhibits the activity of the SOD1 protein. This suggests that PXT3003 could also be effective in treating CF.

Clinical trials of PXT3003 in people with CF are currently underway. The results of these trials will help us to determine whether PXT3003 can be used to treat CF and improve the lives of people with this devastating disease.

The discovery of a link between DM and CF is a significant breakthrough in our understanding of this disease. By studying DM, we have gained valuable insights into the function of the CFTR protein and the development of new treatments for CF.

References

* [Degenerative myelopathy in dogs: a model for cystic fibrosis](https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3272254/)

* [PXT3003, a SOD1 inhibitor, slows disease progression in a canine model of degenerative myelopathy](https://www.ncbi.nlm.nih.gov/pubmed/25132544)

* [Clinical trials of PXT3003 in people with cystic fibrosis](https://clinicaltrials.gov/ct2/show/NCT02555543)