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Developing a new drug treatment for children with a rare inherited kidney condition that does not respond well to existing medications

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Nephrotic syndrome is a serious and debilitating kidney condition, which mainly affects children. Most children will respond well to steroid medication, but those with steroid-resistant nephrotic syndrome (SRNS) do not, leading to kidney failure. At this point, they will need dialysis or a kidney transplant – which has a huge impact on their quality of life and will also reduce their life expectancy. Professor Moin Saleem of the University of Bristol is searching for potential drugs that could help slow kidney damage in children with SRNS. This laboratory research could ultimately lead to an effective new treatment that can help improve the lives of children with SRNS.

This project is jointly funded by Action Medical Research and LifeArc.

How are children’s lives affected now?

A major function of the kidneys is to filter the blood to remove waste products and excess fluid, which is necessary to maintain the right balance of chemicals in the body. But in children with nephrotic syndrome, the kidneys don’t work as well as they should – leading to a range of problems.

“A child will usually have swelling in various parts of their body – and they may also feel tired and generally unwell,” says Professor Saleem. “In most children, these symptoms are usually controlled with steroid medication – but unfortunately, this treatment doesn’t work for everyone.”

Sadly, few treatment options are available for children with steroid-resistant nephrotic syndrome (SRNS) – and they will usually experience total kidney failure within a few years of diagnosis.

“A child will then need dialysis and ultimately, a kidney transplant – which, although life-saving, can have a huge impact on their quality of life and may have long-term complications,” says Professor Saleem. “There is an urgent need for effective new treatment options for children with SRNS.”

How could this research help?

“Our long-term goal is to develop an effective new drug treatment that can help slow kidney damage in children with SRNS,” says Professor Saleem.

The researchers will use cutting-edge technologies to create 3D cell models from children with SRNS – which mimic the affected part of the kidney.

“We will use these cell models to screen thousands of potential drug compounds to identify any that can help improve kidney function,” says Professor Saleem. “As many of these are drugs already used to treat other conditions, this could help to speed up their progression into clinical trials.”

The team will then carry out further laboratory tests on some of the most promising compounds – to narrow down suitable drug candidates for the next stage of development.

We hope this project will ultimately lead to new treatments that will transform the outlook for many of the children with SRNS who don’t respond well to existing medications.

Professor Saleem

Research table

Project details

Project Leader Professor Moin A Saleem, MBBS FRCP PhD
Location Bristol Medical School, University of Bristol
Project Team Professor Gavin Welsh, BSc PhD
Grant Awarded
Grant Amount £241,916
Start Date
End Date
Duration 36 months
Grant Code (GN number) GN3013


  1. Genetic Steroid-Resistant Nephrotic Syndrome Overview, Gene Reviews:


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