The Search for Survival-Driven Therapy in t-MDS

Uma Borate, MBBS, evaluates the current therapeutic landscape for therapy-related myelodysplastic syndrome (t-MDS), a particularly challenging clinical subset of myeloid malignancies. She highlights a persistent and fundamental dilemma within the field: the identification of a truly "best" treatment approach that moves beyond temporary remission to provide lasting survival.

The Limitation of Current Standards

Borate notes that while the medical community has successfully developed strategies to improve response rates, long-term outcomes remains stagnant. She points out that although combining various novel agents with hypomethylating agents (HMAs) has been shown to increase initial remission rates, these clinical wins have not yet translated into the metric that matters most to patients.

"We know that adding things to hypomethylating agents can increase the remission rates, but I don't think we've ever seen data... that leads to overall survival benefit."

She specifically references the VERONA study, a pivotal phase 3 trial that many hoped would redefine the standard of care. With that study being "the last phase 3 study standing" in its category, its failure to demonstrate a significant overall survival (OS) benefit has left a void in the treatment algorithm.

The Goal of Future Research

For Borate, the ultimate benchmark for any new therapy—whether a patient is eligible for a stem cell transplant or not—must be the extension of life. She emphasizes that the field is still waiting for a breakthrough combination that can reliably improve OS.

Borate remains optimistic but cautious, expressing a hope for the emergence of novel agents that can be integrated with HMAs to achieve these deeper clinical goals. She underscores the human element of this research, noting that for the patient, the priority is simple but profound: they want to live longer while maintaining a high quality of life. Until a treatment can prove it delivers on both longevity and lifestyle, the search for the optimal t-MDS regimen continues.

Read the full interview with Dr Borate here.

REFERENCE

Borate U et al. Phase II study of clinical efficacy of venetoclax in combination with azacitidine in patients with therapy related myelodysplastic syndrome (t-MDS). Presented at: 67th Annual ASH Meeting; December 6–9, 2025; Orlando, Florida. Abstract 238.