New drug mix tested in Cambridge could lead to safer treatment for childhood leukaemia
Scientists in Cambridge are planning patient trials after lab tests showed two existing drugs could treat B-cell acute lymphoblastic leukaemia without the harsh side effects of chemotherapy
Scientists in Cambridge say a new mix of two drugs could help treat B-cell acute lymphoblastic leukaemia (B-ALL) and avoid the harsh side effects of chemotherapy. They hope to begin testing it in patients soon.
So far, the treatment has only been tested in the lab and on mice. But early results show that using two drugs together – venetoclax and inobrodib – can kill the cancer cells that cause B-ALL. Both drugs can be taken by mouth.
“These are very promising findings,” said Professor Brian Huntly from the Cambridge Stem Cell Institute. “We now want to trial our approach in adults and teenagers with B-ALL.”
B-ALL is the most common cancer in children and affects over 500 people in the UK each year. Most children can be cured, but the treatment takes more than two years and includes strong chemotherapy. This can lead to serious side effects like infections, sickness, tiredness, and long-term problems with the heart and nerves. Adults and older children often have worse outcomes.
“Every week, I see adult patients who are going through tough treatment for this very aggressive type of leukaemia,” said Dr Simon Richardson from the same research team. “We need better and kinder treatments.”
Venetoclax is already used for a related blood cancer. It works by switching off a protein in cancer cells, making them die. But in B-ALL, venetoclax on its own isn’t always enough.
The team found that adding inobrodib, a drug made by a Cambridge spinout company called CellCentric, helped venetoclax work better. Together, they made the cancer cells die in a different way – by damaging the fat in the cells’ outer layer, causing them to break down.
“The good news is that venetoclax should become cheaper in the next few years when generic versions are available,” added Dr Richardson.
B-ALL is caused by too many early-stage B-cells, a type of immune cell, building up in the bone marrow. These cells stop healthy blood cells from growing and can spread around the body. The new drug combination targets these cells while leaving the body able to make healthy B-cells again after treatment ends.
One person who knows what current treatment is like is Gill Murphy. She was diagnosed with B-ALL in 2013 at the age of 42 after feeling very tired. A blood test led to her diagnosis and she began chemotherapy straight away.
“I thought I’d be in hospital for one night,” said Gill. “It turned into five-and-a-half weeks.”
The treatment was very hard. She had sickness, hair loss, infections, and at times couldn’t see her young daughter due to the risk of catching bugs.
“I was quite calm at first – I treated it like a project,” she said. “But the longer it went on, the more scared I got. There were a lot of sleepless nights.”
Gill had a stem cell transplant to try to cure the cancer. It worked, and she is now in remission. But she still lives with long-term side effects, including tiredness, joint pain, and a higher risk of infection.
“I was very lucky,” she said. “I had a really good donor match. There’s no doubt that they saved my life.”
Gill now works with the charity Blood Cancer UK, sharing her story and helping to improve support for others.
“The doctors I meet all want to find better treatments,” she said. “If we can help more people survive and make the treatment easier to go through, that’s a win for everyone.”
The Cambridge study was funded by Cancer Research UK and Leukaemia UK. The University of Cambridge and Addenbrooke’s Charitable Trust are also raising money to build a new cancer hospital. It will help patients across the East of England and support research to find better treatments for the future.