King’s College Hospital has enrolled the first patient in the world for a major clinical trial testing a potential new treatment for Myelodysplastic Syndromes (MDS), a rare blood disorder that often leads to severe anaemia and an increased risk of acute leukaemia. The trial, led by Takeda Pharmaceuticals, is evaluating erlitercept, a novel therapy designed to address anaemia in adults with lower-risk MDS. The study will compare its effectiveness against erythropoietin, the current standard treatment.
The Phase 3 trial (TAK-226-3001) aims to recruit 300 patients globally, with participants potentially staying in the study for up to five years to assess long-term outcomes. King’s is one of only two UK sites involved, leveraging its status as a National Reference Centre for MDS. The condition affects around 4 in every 100,000 people in the UK, predominantly older adults, and often requires blood transfusions if supportive treatments like erythropoietin fail.
Chief Investigator Highlights Patient Benefits
Dr. Austin Kulasekararaj, a consultant haematologist at King’s and the study’s chief investigator, said the hospital’s role as a specialist centre allows patients access to cutting-edge trials. “The only approved treatment in the UK for ‘lower risk’ MDS is supportive treatment with erythropoietin (natural hormone) injections and if this fails, patients start needing blood transfusions. This trial could provide a fantastic opportunity for our patients to benefit from this therapy.”
The recruitment of the first patient was supported by the National Institute for Health and Care Research (NIHR) Clinical Research Facility, which helped meet new government targets introduced in April 2026 to speed up clinical research setups across the UK. These changes aim to give patients faster access to experimental therapies while reinforcing the country’s position in global medical research.
King’s Hospital Leads Rare Blood Disorder Research
King’s College Hospital consistently ranks among the UK’s top NHS trusts for research recruitment, managing over 1,200 active studies annually. Its research portfolio spans discovery science, clinical trials, and data-enabled studies, with centres like the Clinical Age Research Unit and HIV Research Centre driving innovation in multiple specialties. The hospital’s focus on rare blood disorders aligns with its broader mission to improve patient outcomes through advanced therapies.
The trial’s success hinges on its ability to demonstrate long-term benefits for patients who currently face limited treatment options. Unlike many experimental therapies, erlitercept targets anaemia directly, a symptom that significantly impacts quality of life. If proven effective, it could shift the standard of care for a condition that predominantly affects older adults, many of whom already struggle with frailty and other comorbidities.
Targeted Therapy Marks New Direction
While earlier trials for MDS have often focused on chemotherapy or bone marrow transplants—approaches with severe side effects—this study represents a shift toward targeted, less invasive treatments. The five-year follow-up period will be critical in determining whether erlitercept not only improves blood counts but also reduces progression to acute leukaemia, a common concern in MDS management.
King’s College Hospital’s involvement reflects its long-standing commitment to rare disease research. The hospital’s infrastructure, including dedicated research units and partnerships with pharmaceutical companies, positions it as a key player in advancing therapies for conditions with few existing options. The first patient’s enrollment marks a step toward potentially transforming care for thousands of adults living with MDS.
