Posted on 30 April 2025

A new study involving researchers from the University of York is set to transform treatments for one of the most aggressive and hard-to-treat blood cancers - acute myeloid leukaemia (AML).

Dr William Grey, Research Fellow in the Centre for Blood Research at the University of York. Credit: Jeff Moore

If successful, the research could significantly improve the lives of patients by offering gentler, more effective treatments that reduce suffering and increase survival chances.

The TARGET study is a collaboration between Leeds Teaching Hospitals NHS Trust and the Centre for Blood Research at the University of York.

Targeted therapies

The research will focus on developing targeted therapies that are easier for patients to tolerate.

AML is an aggressive type of blood cancer that is very difficult to treat. Standard chemotherapy is highly toxic and often does not work long-term, as most patients experience relapse. The main reason for this is chemotherapy-resistant leukaemic stem cells.

For patients, this means going through challenging treatments with a high risk that the cancer will return. The TARGET study aims to change this by developing safer, more effective therapies that directly target these resistant cells, preventing relapse and giving patients hope for a longer, healthier life.

The study will build on previous proof-of-concept work and focus on creating treatments targeting the most important types of cancer cells that are easier for patients to handle. The goal is to develop oral treatments that patients can take at home, reducing hospital visits, and for the elderly to improve their overall fitness and get them home earlier. If successful, these treatments would improve quality of life by reducing side effects such as nausea, hair loss, the need for transfusions, or the risk of infections.

In addition, these safer treatments could help older patients and those with other medical conditions, who currently have very few treatment options because of the risks linked to intensive chemotherapy.

Scientific expertise

Dr William Grey, Research Fellow in the Centre for Blood Research at the University of York, said: “We are really excited to develop true bench-to-bedside research in Yorkshire to combine our scientific expertise with that of an outstanding hospital.

“We are leveraging multiple new technological advances to get to the core issue of how to eradicate a deadly disease without the need to put patients through the harshest chemotherapy, effectively aiming to change the way we treat the disease”.

Dr Anjum Khan, Consultant Haematologist at Leeds Teaching Hospitals NHS Trust, said: “Our standard treatment for acute myeloid leukaemia is still based upon aggressive chemotherapy drugs developed many years ago.

“This study is a very exciting approach, building upon recent technological advances. We’re developing safer kinder drugs without the toxic side effects – to prevent relapse whilst protecting healthy blood cells.”

Dr Suzanne Ri,x said: "Acute myeloid leukaemia is one of the most aggressive and hardest-to-treat types of blood cancer, and for too long, treatment options have remained limited and highly toxic.

“Research like the TARGET study is critical in changing this, offering hope of kinder, more effective therapies that could transform outcomes for people with AML. At Blood Cancer UK, we are committed to funding and supporting research that leads to better, less harmful treatments for all blood cancers, and we welcome this vital work taking place in Yorkshire.

“Every step forward in understanding and tackling chemotherapy resistance brings us closer to a future where more people survive blood cancer with a better quality of life.”

Pioneering research

Dr Simon Ridley, Director of Research and Advocacy at Leukaemia UK, said: “Leukaemia UK is thrilled to see the TARGET study advancing in the search for better, kinder treatments for acute myeloid leukaemia (AML), and taking pioneering research directly to patients. We are delighted that Dr William Grey, is co-leading this important work at the University of York.

“At Leukaemia UK, we are dedicated to supporting researchers at key stages in their careers, enabling discoveries that drive life-saving change. Dr Grey’s work is a prime example of how early-stage funding can lead to significant progress for all those diagnosed with this devastating disease. We look forward to the outcomes of the TARGET study and hope it leads to more effective, less toxic treatments for AML patients.”

Despite decades of research, there is still limited knowledge about how changes at the molecular level allow leukaemic stem cells to resist chemotherapy. This study will take a new approach by studying protein changes (proteomics) along with genetic factors. By looking at all the protein changes in AML, a related condition called myelodysplastic syndrome (MDS) and comparing this to healthy blood cells, researchers hope to find new ways to treat the disease.

Transplant procedures

Experts in protein research and blood stem cell biology will carry out the work using advanced research facilities at the Centre for Blood Research and Bioscience Technology Facility at the University of York.

The study will use surplus clinical samples from adult patients with AML/MDS who choose to take part—these samples are collected during routine procedures, so no extra tests are needed.

The study also partners with the Anthony Nolan Research Institute to compare these findings to healthy blood cells which are used as part of transplant procedures.

If successful, this research could change the way AML is treated, replacing chemotherapy with a more targeted, gentler approach. Patients could have better outcomes, with a focus on treatments that cure the disease rather than just delaying its return.