Novel Method of Treating Polycystic Kidney Disease

New method of use for mitochondrial targeted peptide, Elamipretide.. Promising treatment to slow or halt the progression of PKD in both adults and infants.
  • New method of use for mitochondrial targeted peptide, Elamipretide..
  • Promising treatment to slow or halt the progression of PKD in both adults and infants.

Abstract:

Technology Description

Polycystic Kidney Disease (PKD) is a genetic disorder that causes cysts to form on the kidneys and other organs such as the liver. Over time, this causes complications such as high blood pressure, heart valve issues, increased risk for aneurysms, and eventually kidney failure. There is no cure for PKD, and often patients will need to undergo a kidney transplant. Currently, the only FDA therapeutic approved to slow the progression of cyst formation is the Vasopressin receptor 2 (V2R) antagonist, Tolvaptan. Unfortunately, Tolvaptan is only recommended for certain patients, can cause serious side effects, and is expensive. Therefore, there is a great need for cost-effective therapeutics which slow or halt the progression of PKD in order to extend patient life expectancy and improve quality of life.

Inventors at the University of Iowa have developed a new method of treating Polycystic Kidney Disease (PKD) using the mitochondrial targeted peptide, Elamipretide (also called ELAM and SS-31). The inventors have discovered that administration of Elamipretide reduces mitochondria reactive oxygen species (ROS) and oxidative damage and attenuates the progression of cyst formation in a PKD mouse model. When given to pregnant mice, pups born with severe PKD experience a significantly longer lifespan than non-treated littermates, demonstrating that Elamipretide may also serve as a treatment for infantile PKD. Importantly, this peptide is inexpensive to produce, making it an ideal therapeutic for long term treatment. Therefore, Elamipretide is a promising treatment to slow or halt the progression of PKD in both adults and infants, while reducing the cost of treatment for patients worldwide.

UIRF Case No. 2022-008

Stage of Development

Elamipretide has been tested in a PKD mouse model in both adults and pups born with severe PKD.

Website:

https://uiowa.flintbox.com/technologies/DE0BF58C0ADC4BBBB5CAD0E11FA79F0C

Advantages:

  • Slows cyst formation in adults and infants.
  • Reduces ROS and kidney fibrosis.
  • Inexpensive to produce.
  • Already in clinical trials for other indications.
  • May be used for intrauterine treatment.

Contact Information:

Name: Kellen Sensor

Title :

Department :

Email: kellen-sensor@uiowa.edu

Phone: 319.335.4546

Address :