Little Princess Trust News
Developing a new treatment for ALT-positive cancers

Working together to try and make an enzyme-blocking medicine
Cancer cells run on DNA – the instructions inside all your cells that control how they behave and what they make and do. While some cancers are very different from each other, some share sections of DNA that help the cancer cells survive.
ALT-cancers are a group of different types of cancer which share a section of DNA. This DNA allows cancer cells to grow using a different route, called the Alternative Lengthening of Telomeres (ALT) pathway.
Many types of brain and bone tumour use the ALT pathway, and these cancers almost always affect children and young people.
ALT-cancers can be extremely difficult to treat – they are very aggressive and current chemotherapy and radiotherapy approaches often don’t work.
With their Little Princess Trust New Ideas project, Dr Jon Elkins and Dr Anna Rose want to change this. They are researching brand new treatments which specifically target the ALT pathway.

Dr Anna Rose’s research team have discovered an enzyme (a type of protein) which helps the cancer cells use the ALT pathway to grow.
This enzyme is not particularly active in normal cells, which makes it an exciting target for a new treatment that could kill ALT-cancer cells without harming healthy cells. In the lab, blocking the enzyme also increased how well several chemotherapy medicines worked.
After having promising early results with chemicals in the lab, Anna teamed up with Jon, an expert in developing safe new treatments. Together, they hope to make a brand new, ALT-cancer fighting and enzyme-blocking medicine.

Jon’s team of drug chemistry experts will look at thousands of potential medicines to identify those which are best at blocking the ALT-cancer enzyme. The researchers will then modify potential treatments further to increase their ability to block the enzyme.
Dr Anna’s team will test the top candidates inside ALT-cancer cells. Her team will look at how effective the candidates are at killing brain and bone cancer cells, and study what happens to their DNA.
This will help scientists understand more about how ALT-cancer cells work at the DNA level. The two teams will continue to work closely together to produce the best new medicine possible.
Jon said: “We are very excited to start this work. It should allow us to generate important preliminary data – showing that targeting this enzyme with a drug is an effective new way to tackle ALT-cancers.
"This will allow us to progress the research to the next stage.”


