Testing a new treatment for childhood polycystic kidney disease
Dr Jennifer Chandler from University College London Great Ormond Street Institute of Child Health has been awarded a £98,000 PKD research project to test a new treatment for children with autosomal recessive polycystic kidney disease (ARPKD) in human kidney cells. This project forms part of the PKD Partnership, led by Kidney Research UK and the PKD Charity.
Understanding childhood polycystic kidney disease
Polycystic kidney disease (PKD) is an inherited condition that causes fluid-filled cysts to grow in the kidneys, gradually damaging them and often leading to kidney failure.
There are two main types of PKD;
- Autosomal dominant polycystic kidney disease (ADPKD)
- Autosomal recessive polycystic kidney disease (ARPKD)
Autosomal recessive polycystic kidney disease (ARPKD) affects around 1 in 20,000 babies, and unfortunately, it can progress very quickly. Many children develop kidney failure early in life, and there are currently no treatments that can stop or reverse the disease.
A new hope for children with ARPKD
In ARPKD, there is a genetic change that disrupts a key protein needed for healthy kidney function. When this protein doesn’t work properly, cysts begin to form and grow.
In this project, Dr Jennifer Chandler aims to design a therapy that restores the functional part of this protein, helping kidney cells behave more normally again.
"Children with rare kidney diseases have waited far too long for treatments designed specifically for them. Our ambition is to help change that by developing therapies tailored to childhood conditions, rather than relying on treatments that were originally developed for adults.
"Thanks to funding from Kidney Research UK, we're testing a new approach for childhood polycystic kidney disease in human kidney cells. We hope this research will lay the foundations for more effective treatments and, ultimately, improve the lives of children living with this devastating condition and their families." Dr Jennifer Chandler.
A targeted delivery system
Jennie and the team will use a non-harmful virus as a delivery system. This type of virus has been carefully adapted so it cannot cause disease, but it is very efficient at entering human kidney cells.
Inside this virus, the medicine instructions are packaged so they can be delivered directly to the cells affected by ARPKD. This works like a delivery service: the harmless virus carries the treatment directly to the kidney cells where it is needed most. The cells can then produce the medicine using their normal cell machinery
They will then test this therapy in human kidney cells carrying the most common genetic change found in ARPKD to find out whether it can reduce or slow the formation of cysts associated with the disease.
What could this mean for children and families affected by rare kidney diseases?
By testing a targeted therapy in human kidney cells affected by ARPKD, researchers will learn whether this approach has real potential to slow or stop cyst growth in patients.
"This research is an important first step in understanding whether this new therapeutic approach could slow the progression of ARPKD. More broadly, we hope this project helps address the gap in research for rare childhood kidney diseases and informs the development of new therapies for a range of kidney conditions in the future." Dr Jennifer Chandler.
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