Polycystic Kidney Disease May Now Be Treatable
The development of cysts, which are fluid-filled sacs, in the kidneys, hindering with their ability to filter waste products from the blood, is termed as polycystic kidney disease. These cysts may grow bigger and enlarge the kidneys, eventually resulting in their failure. Cyst formation may also be observed in other organs, such as the liver. The two major forms of this disease are autosomal dominant (AD) PKD and autosomal recessive (AR) PKD.
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Even though cysts are present in kidneys of people with ADPKD since birth or childhood, the disease starts displaying symptoms in adulthood. It can be further divided into type 1 and 2 depending on the affected genes. The signs and symptoms associated with ARPKD appear at birth or early infancy, and even though this form of PKD is rare, it often proves to be lethal early in life.
Current Pipeline Scenario
The PKD therapeutics pipeline comprised about 16 candidates in November 2017. Tolvaptan, a drug in the pipeline being developed by Otsuka Pharmaceutical Co. Ltd., was in the filed stage of the development process in 2017 in the U.S. Obtained from a synthetic source, it is a small molecule being developed to be administered orally. This drug is a selective vasopressin V2 receptor antagonist that works by blocking vasopressin at the V2 receptor. It has been effective in decreasing cyst cell proliferation and fluid secretion, thereby reducing cyst development.
Another drug in the PKD therapeutics pipeline is Tesevatinib, which is currently under the Phase II stage of the development process. It is being developed by Exelixis Inc. as a potential treatment for ADPKD and ARPKD. Similar to Tolvaptan, it is also a small molecule derived from a synthetic source. It is to be administered orally, after which it would act as an epidermal growth factor receptor antagonist and EphB4 inhibitor.
This shows that the pipeline is constantly growing, which is positive news for people suffering from PKD.