A drug being developed to treat chronic kidney disease in Alport syndrome is showing promising results in patients in an on-going phase 2 clinical trial. Patients have shown an increase in kidney function after twelve weeks of taking the drug, and this has continued through to the thirty-sixth week of treatment.
The original news release by Reata Pharmacuticals, the company developing the drug, can be viewed here.
Alport syndrome is a genetic condition that causes kidney damage, eye problems, and hearing loss. It affects approximately one in 50,000 people. The disease is caused by a change to the genes that code for a specific protein involved in making IV collagen. When the genes change, the protein they produce doesn’t function and the IV collagen can’t function properly. One important role of IV collagen is to be part of kidney structures that filter waste products and water from the urine. In people with Alport syndrome the changes to the IV collagen prevent this from effectively happening, and often blood and protein pass into urine. This protein causes inflammation and progressive scarring, which further interferes with kidney functioning. Unfortunately, there are not currently any therapies approved to treat Alport syndrome.
However, Reata Pharmaceuticals is developing a drug called bardoxolone designed to treat chronic kidney disease (CKD) caused by Alport syndrome. The drug works by activating the molecule Nrf2. Nrf2 is involved in helping mitochondria to work to reduce oxidative stress, which is an imbalance of certain chemicals. This should stop the body from creating the inflammation that contributes to CKD. Due to the potential the drug has for helping people with Alport syndrome, bardoxolone has been granted Orphan Drug Designation. This means that the government will assist Reata Pharmaceuticals with drug development in various ways, including through advice, tax breaks, and extended marketing exclusivity.