NHS Breaks New Ground as First to Use Trojan Horse Therapy for Blood Cancer

The Trojan Horse Arrives: A New Weapon for Blood Cancer in the NHS

Picture this: a drug that sneaks inside a cancer cell, delivers its lethal payload, and destroys the cell from within—leaving healthy neighbors untouched. The NHS in England has just become the first health system in the world to begin using such a therapy for a form of blood cancer. This breakthrough marks a turning point in how we treat difficult tumors that resist standard therapies.



What Is This Therapy, and Why “Trojan Horse”?
At its core, this is a type of antibody-drug conjugate (ADC). Imagine an antibody—a guided missile—that binds specifically to cancer cells. But rather than just marking them for destruction, it carries a toxic payload inside. Once the antibody is taken into the cancer cell, the poison is released, killing the cell from the inside. That’s the “Trojan horse” metaphor.

The specific drug now being rolled out in the NHS is belantamab mafodotin (also known by its brand name Blenrep). It targets BCMA (B-cell maturation antigen), a protein often found on malignant plasma cells in multiple myeloma. Once bound, the drug enters the cell and delivers its cytotoxic cargo—destroying that cancer cell while sparing many normal ones.

This targeted internal attack is a departure from classic chemotherapy, which can affect both healthy and cancerous cells broadly. The idea is more precision, more punch, and less collateral damage.

Why Multiple Myeloma? And Which Patients Are Eligible
Multiple myeloma is a cancer of plasma cells in the bone marrow. Over time, it often relapses, becomes refractory, and resists multiple lines of therapy. Although progress has been made, it remains essentially incurable in most patients, with treatment focused on extending remission, preserving quality of life, and delaying progression.

The new therapy is intended for patients whose disease has progressed or failed to respond to first-line treatments—specifically, those exposed to lenalidomide. Around 1,500 patients per year in England are expected to qualify. The regimen will combine the new therapy with bortezomib (an injected proteasome inhibitor) and dexamethasone (oral corticosteroid), given as infusions every three weeks.

The National Institute for Health and Care Excellence (NICE) endorsed its use in these settings, and the NHS is fast-tracking access through its Cancer Drugs Fund.

What the Clinical Trials Showed: Promise, but With Caveats
When considering a new therapy, it’s critical not only to look at the hopeful stories, but at the data. Here’s what is known so far.

Efficacy and Progression Delay
In broader patient populations with relapsed or refractory myeloma, combining belantamab mafodotin with bortezomib and dexamethasone (the regimen now approved) delayed the time to disease progression for an average of three years, compared with about one year using the more traditional combination involving daratumumab. That represents a near tripling of progression-free survival in those trials.

Importantly, the subset of patients for whom NICE approved it (post-lenalidomide) was more narrowly defined, so the exact benefit in that group is less publicly detailed. But the data from the larger trials strongly influenced its approval.

Real Patient Impact
One early-access patient, after failing standard therapies, experienced remission within weeks of starting the new drug. That kind of anecdote gives human context to the numbers—but caution is always needed, as not every patient will respond so dramatically.

Safety Profile and Adverse Events
No therapy is risk-free. Some important side effects have emerged:

• Eye toxicity: Changes in the corneal epithelium are known risks. Patients may experience blurred vision, dry eyes, and other ocular symptoms.
  
  
• Infusion reactions, fatigue, nausea, and other standard systemic adverse effects have been reported.
  
  
• Dose adjustments and careful monitoring must be part of any clinical rollout.
  

Some key precautions: patient selection is essential, dose timing must be managed, and ocular screening protocols may become standard in real-world use.

Also, note that in the U.S., this drug was previously withdrawn after a phase III trial (DREAMM-3) failed to confirm efficacy in certain settings. That raises the caution flag: results may not always replicate in broader practice. (Belantamab mafodotin’s Wikipedia entry mentions this history.)

Thus, while the excitement is justified, we remain in the phase of guarded optimism.

What Makes This Rollout Historic
A few features make this moment especially noteworthy:

• First in the world: The NHS is the first health system to offer this therapy broadly.
  
  
• UK innovation: The drug was researched and developed domestically, and many trial sites were UK-based.
  
  
• Public health scale: It’s not a small pilot; it’s a national rollout, bringing advanced therapy to many patients.
  
  
• Potential shift in standard care: Because of the strong data, this may alter treatment guidelines for relapsed myeloma globally.
  

In that sense, we may look back at this as a turning point—when targeted, internal delivery of cytotoxic agents steps into mainstream oncology.

What to Monitor: Risks, Challenges & Uncertainties
To make the most of this therapy—and avoid pitfalls—clinicians must keep an eye on several areas.

1. Long-term outcomes and resistance
We don’t yet know how long responses will last in a real-world setting or how frequently resistance will develop. Genetic subclones may evade the drug or downregulate BCMA, requiring combination strategies.

2. Safety in broader populations
Clinical trials often select healthier patients without severe comorbidities. When extended to older, frailer patients with kidney disease, cardiac disease, or other comorbidities, side effects may be more frequent or worse.

3. Ocular toxicity and quality of life
Eye toxicity is not trivial. If patients develop vision issues, dose reduction or discontinuation may be needed, potentially compromising benefit. Real-world ophthalmologic follow-up will be essential.

4. Cost, access, and equity
High-cost therapies often face rationing or access disparity. Ensuring that patients, regardless of location or socioeconomic status, can benefit will demand careful planning.

5. Regulation and global acceptance
Other regulatory agencies (e.g. FDA, EMA) may review the same data with different thresholds. Also, some countries may wait for longer-term safety before adoption.

6. Managing expectations
Patients hearing peak-case successes may expect cures. It's important to calibrate expectations: this therapy delays progression, not (yet) cures.

Clinical and Research Implications for Hematology/Oncology
For clinicians and researchers, this rollout opens new frontiers and responsibilities.

• Strategic sequencing: Where does this therapy fit in the line-up? Should it be earlier, or reserved?
  
  
• Biomarker monitoring: BCMA levels, ocular toxicity markers, minimal residual disease (MRD) status—all become crucial.
  
  
• Combination strategies: Syndrome with immune therapy, cellular therapy, or small molecules.
  
  
• Resistance profiling: Study escape mechanisms and plan next steps.
  
  
• Real-world registries: Capturing outcomes, adverse events, and patient-reported quality of life is as vital as the clinical rollout.
  
  
• Education and training: Oncologists, hematologists, ophthalmologists, nurses—all must learn to manage and monitor this therapy safely.
  
  
• Ethical oversight: Informed consent is more complex when risks and long-term data are still evolving.
  

A Narrative Glimpse: Mrs. Thompson’s Journey
Consider Mrs. Thompson, a 67-year-old woman with relapsed multiple myeloma. She’s already had lenalidomide-based therapy and is now refractory. Traditional options exist, but benefit is limited.

She becomes eligible for the new therapy. After getting ophthalmology baseline screening, she receives her first infusion with bortezomib and dexamethasone. Within weeks, her bone pain eases, lab markers fall, and she feels stronger. Over months, imaging confirms shrinking plasma cell burden.

But halfway through, she notices blurry vision. Her ophthalmologist adjusts dosing and closely monitors, and fortunately the ocular effects stabilize without major vision loss.

She is among those whose lives this therapy extends—months or years of better quality of life, delayed progression, and more time with loved ones.

Her story is not guaranteed—but it is now plausible.

Where We Go from Here: Futures, Questions, and Vision

• Expanded indications: Could this therapy move into earlier lines of treatment or to patients who can’t tolerate lenalidomide? NICE will likely reassess.
  
  
• Worldwide uptake: Countries beyond the UK may adopt it, depending on cost, infrastructure, and regulatory review.
  
  
• Next-generation ADCs: Scientists will build newer “trojan horses” with better safety, more potency, or dual targeting.
  
  
• Resistance countermeasures: Combination regimens, sequential treatments, or rescue therapies may emerge.
  
  
• Patient stratification: We may learn who benefits most (by age, renal function, BCMA levels, prior therapy history) and personalize accordingly.
  
  
• Long-term survival data: As time passes, we’ll see whether delaying progression translates into longer overall survival.
  
  
• Integration with cell therapy: Could this ADC be a bridge to CAR-T or bispecific therapies? Or could it combine for synergy?
  

This moment is not an end, but a beginning.