{"id":162351,"date":"2026-06-15T13:42:00","date_gmt":"2026-06-15T12:42:00","guid":{"rendered":"https:\/\/www.kidneyresearchuk.org\/?p=162351"},"modified":"2026-06-15T13:42:00","modified_gmt":"2026-06-15T12:42:00","slug":"testing-new-therapies-for-children-with-autosomal-recessive-polycystic-kidney-disease","status":"publish","type":"post","link":"https:\/\/www.kidneyresearchuk.org\/2026\/06\/15\/testing-new-therapies-for-children-with-autosomal-recessive-polycystic-kidney-disease\/","title":{"rendered":"Testing new therapies for children with autosomal recessive polycystic kidney disease"},"content":{"rendered":"\n\n\t\t\n\t

Professor David Long from University College London\u00a0Great Ormond Street Institute of Child Health\u00a0has received a Kidney Research UK paediatric\u00a0and rare diseases research project\u00a0grant of \u00a3200,000\u00a0to\u00a0test new therapies for autosomal recessive polycystic kidney disease<\/a> (ARPKD) that focuses on how cysts use energy.\u00a0This project forms\u00a0part\u00a0of\u00a0the\u00a0PKD Partnership<\/a>,\u00a0led by Kidney Research UK and the PKD Charity.\u00a0<\/p>\n\t

New treatments are needed for children with ARPKD<\/b>\u00a0<\/h2>\n

Polycystic kidney disease<\/a> (PKD)\u00a0is an inherited condition\u00a0that causes fluid-filled cysts to grow in the kidneys, gradually damaging them and often leading to kidney failure<\/a>.\u00a0The condition is\u00a0passed from parents to children through faulty genes.\u00a0\u00a0<\/p>\n

One of the most severe forms is autosomal recessive polycystic kidney disease (ARPKD), a rare condition that places a heavy burden on children and their families from an early age. There is currently no cure for ARPKD, and there are no approved drug treatments to slow or stop the disease.\u00a0<\/p>\n\t

Focusing on how cysts use energy\u00a0\u00a0<\/h2>\n

In this project, Professor David Long\u00a0and the team\u00a0are looking more closely at a\u00a0protein called AMP-activated protein kinase (AMPK). AMPK controls how cells use energy in a process called metabolism.\u00a0This protein has already been studied in\u00a0other\u00a0diseases such as cancer,\u00a0obesity\u00a0and diabetes, where drugs can alter its activity. Now,\u00a0the team\u00a0want to know whether a similar approach could work in ARPKD.\u00a0<\/p>\n

As cysts grow, the kidney’s structure becomes increasingly abnormal. The team believes these cysts may depend on energy processes to keep expanding. \u00a0<\/p>\n

To test this,\u00a0David and the team\u00a0are\u00a0looking at lab models of\u00a0kidneys with a faulty PKD gene to see how changing AMPK activity affects cyst growth and kidney function. They\u00a0will also\u00a0compare\u00a0how metabolism differs between healthy and affected\u00a0kidneys\u00a0to see whether altering AMPK with drugs changes their ability to form cysts. Together, this work will show whether targeting cell energy could form the basis of a new treatment for ARPKD.<\/p>\n\t\t\t\t

ARPKD carries a very high burden, with many children going on to develop kidney failure. We want to understand how cysts use energy to keep growing, and whether changing those energy pathways can reverse the severity of the disease. By testing drugs that alter metabolism, we can begin to see if this approach could make a real difference to ARPKD patients.<\/p>\n\t\t\t\t

Professor David Long<\/h4>\n
\n\t\t\t\t\"Professor