Myeloma UK Launches New Clinical Trial to Develop Therapies for High-Risk Myeloma

Myeloma UK has launched a new clinical trial to identify the best treatment options for myeloma patients classified as high-risk. The study is the first of its kind in the U.K. and one of the few that focus on high-risk myeloma patients, who represent a sub-group with urgent, unmet medical needs.

The MUK nine trial is divided into two parts. In MUK nine a, up to 700 newly diagnosed patients will undergo genetic profiling using state-of-the-art genetic techniques developed at the Institute of Cancer Research (ICR) in London, with Myeloma UK funding.

Researchers will analyze bone marrow samples from patients to identify genetic abnormalities usually linked to a more active and difficult to treat form of the disease, known as “high-risk” myeloma. These patients often fail to respond to available therapies and constantly relapse.

The genetic screening will allow researchers to stratify patients according to their genetic risk, which creates the possibility for more tailored treatments to this specific sub-group.

Patients identified as high-risk will have the opportunity to enroll in the trial’s second phase, called MUK nine b. This Phase 2 study (NCT03188172) will determine the effectiveness of a new combo of four therapies – Velcade (bortezomib), Revlimid (lenalidomide), Darzalex (daratumumab) and dexamethasone – combined with low-dose cyclophosphamide and stem cell transplantation.

“It’s vitally important that people with myeloma get the most effective treatment for them, particularly people with the highest risk forms of the disease. But we urgently need the evidence to show which therapeutic approaches are right for different groups of patients,” MUK nine trial lead investigator Martin Kaiser said in a press release.

“The MUK nine trial will give patients access to innovative new treatments and state-of-the-art molecular testing. We hope it will transform myeloma treatment from a one-size-fits-all approach to a stratified approach driven by disease characteristics,” added Kaiser, who also is a senior researcher at ICR.

The trial results will contribute to a deeper understanding of the genetic causes of the disease by identifying disease markers for sub-groups of myeloma patients.

A total of 30 Clinical Trial Network (CTN) centers across the U.K. will participate in the MUK nine trial. The study is part of the Myeloma UK CTN, which comprises early-stage trials coordinated and sponsored by the Clinical Trials Research Unit at the University of Leeds.

“Around 20% of myeloma patients are characterized as having high-risk myeloma, yet there is relatively little research looking at high-risk disease and treatment. This trial is looking to the future – we are trying to gain more insight into which treatment combinations might work best in different groups of patients,” said Simon Ridley, Myeloma UK director of research.

“It also offers patient access to novel combination treatments that they cannot currently get access to through the NHS. The data this trial will generate can be used in the UK and beyond to support patient access to the most innovative and effective combination treatments,” he said.

MUK nine is funded by Myeloma UK, together with the support of Celgene UK and Ireland and Janssen Oncology, which will provide Revlimid and Darzalex, respectively, along with financial support.