Bloodwise research highlights 2018

The breakthrough that everyone’s most enthusiastic about is CAR-T therapy, a new way to treat some blood cancers. It is now available on the NHS for some people with diffuse large B cell lymphoma and some children and young adults with acute lymphoblastic leukaemia (ALL), and the first children were treated at Great Ormond Street Hospital at the end of November. CAR-T works by taking immune cells from the person with cancer, ‘editing’ them to make them more effective cancer killers, and putting them back in to do their job. Clinical trials have shown that CAR-T can cure blood cancers, so there is a lot of expectations with this treatment. However, since the treatment is personalised it can be very expensive. Professor Anastasios Karadimitris at Imperial College London may have found a way to make this treatment less expensive by mass-producing it. The team used a slightly different type of immune cell that can attack cancer cells more powerfully than CAR-T, and doesn’t have to be tailored to the individual.

CAR-T isn’t the only promising new treatment on the horizon. At the University of Birmingham, Professor Tatjana Stankovic has been exploring how drugs called PARP inhibitors can stop cancer from growing. Researchers already knew that PARP inhibitors could stop some kinds of cancer from growing, but Professor Stankovic’s work also revealed that they might be able to work for a bigger group of cancers, including myeloma and chronic lymphocytic leukaemia (CLL).

Meanwhile, Professor Terry Rabbitts at the University of Oxford has been working on developing a new method for making drugs small enough to get inside cancer cells, where they can more effectively target cancer cells, while leaving healthy cells relatively untouched.

Excitingly, these breakthroughs might also hold promise for treating other kinds of disease. Professor Karadimitris’s CAR-T cells have potential as a treatment for brain cancer, and Professor Stankovic’s PARP inhibitors could be used as a treatment for bladder and prostate cancers, as well as sarcomas. It’s not just cancers that might be affected, either – the new drug-making technique developed by Professor Rabbitts could be used to target proteins that are involved in infections or inflammatory diseases like asthma and coeliac disease.

It’s also been a very good year for the fight against childhood blood cancers. In April, Dr Caroline Furness and other scientists at the Institute of Cancer Research in London published their findings on ‘founder mutations’ that can lead to ALL in children. These founder mutations are genetic errors that are present in all the cancer cells of someone with this type of leukaemia and can drive the development of the disease from its very early stages. The team identified founder mutations that occur in about a quarter of children and young adults with ALL. Targeting these mutations could offer more effective ways to treat this disease.

In May, Professor Mel Greaves, one of the researchers working on the founder mutations project, published the conclusion of 40 years of research on ALL. While we know that random genetic errors that happen while the baby is still in the womb can lead to ALL, only some children with these genetic errors will go on to develop ALL later in childhood. Professor Greaves’ research has now revealed the reason for this: the second trigger for the development of ALL is due to children having an abnormal immune reaction to common infections. This is fantastic news as it suggests that it may in the future be possible to prevent childhood ALL by helping children develop a strong immune system early in life. But it’s really important for parents to know that there is nothing they could have done to prevent their child from developing leukaemia. This is all down to the genetic changes that happen by chance before their child is even born.

Finally, our research on acute myeloid leukaemia (AML) is looking very promising, with researchers at the University of Glasgow and the University of Birmingham both having breakthroughs in understanding this complicated disease.

AML is one of the most aggressive forms of blood cancer and can often resist treatment. In a project supported by Bloodwise, Children with Cancer UK and other funders, Dr Karen Keeshan has now identified activity from a gene called Trib2 as one of the reasons for treatment resistance. This gene can make AML cells resistant to standard chemotherapy, but they do still respond to chemotherapy in combination with a targeted drug called venetoclax (already used to treat CLL). The researchers are now in the early stages of investigating whether these findings are relevant for AML in children as well as in adults.

Finally, Professors Constanze Bonifer and Peter Cockerill have published the culmination of 10 years of research on the genetic errors that lead to AML. There are many different genetic errors driving AML, with each error affecting AML cells in a different way. This is crucial in the treatment of AML, as drugs that work on one genetic error are unlikely to work on another. Their work has revealed which genetic errors are most important in driving the development of these different types of AML. This is a huge step forward in the drive to make sure that each person with AML receives the treatment that is most effective for them.

We’ve also asked some of our researchers about their personal highlights of the year. You can read about them here.

Thanks to all our researchers for their hard work this year, and to all our supporters for making it possible for these exciting advances in blood cancer research to happen. We hope that 2019 will bring even more good news!