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Redefining management of uveal melanoma liver metastases: what oncologists need to know

17th July 2025

Metastatic uveal melanoma may be a rare disease, but it poses a major clinical challenge, with limited systemic treatment options and a strong predilection for liver metastases. This expert commentary by consultant oncologist Dr Matthew Wheater examines the potential of liver-directed therapies, specifically melphalan-based percutaneous hepatic perfusion, and highlights key findings from the pivotal FOCUS trial that may alter the treatment approach for patients with liver-dominant disease.

Although rare, uveal melanoma is the most common primary intraocular malignancy in adults, with an incidence of approximately five cases per million per year.1 Despite effective local control of the primary tumour, nearly 50% of patients develop metastatic disease, often years after initial diagnosis.2

Metastatic uveal melanoma (mUM) differs significantly from cutaneous melanoma in terms of its molecular profile, behaviour and treatment response. A striking feature of mUM is its propensity for hepatic metastasis, with up to 90% of metastatic cases involving the liver, either as the sole site or in combination with other sites.3

Despite advances in immunotherapy and targeted agents for cutaneous melanoma, these therapies have shown limited efficacy in mUM due to a lack of shared driver mutations such as BRAF and a relatively immunologically ‘cold’ tumour microenvironment.1 The distinct biology of mUM requires equally distinct treatment strategies, particularly those that address the dominant hepatic involvement.

The unmet clinical need in mUM

Patients with mUM face a poor prognosis, particularly those with liver-dominant disease. The median overall survival (OS) after development of metastases is approximately 12 months, and this figure has remained largely unchanged over decades.4

In the absence of approved systemic therapies specifically for mUM, treatment has often relied on the use of drugs approved for cutaneous melanoma, such as immune checkpoint inhibitors. However, multiple studies have demonstrated limited clinical benefit from anti-PD-1 or anti-CTLA-4 therapy in this population, with response rates typically less than 5%.5

Chemotherapy, either as monotherapy or in combination, also lacks meaningful efficacy. Hepatic metastases are typically resistant to systemic therapy due to the liver’s unique immunosuppressive environment and the typically low mutational burden of uveal melanoma.6

Tebentafusp – a recently approved T-cell receptor-directed therapy for HLA-A\*02:01-positive patients with mUM – has shown benefit but has limited applicability due to the human leukocyte antigen restriction, which limits availability to around 40% of the population.

This agent is the first to show a significant impact on survival in mUM, but it has modest activity in patients with liver-dominant disease.7 This persistent unmet need highlights the importance of liver-directed approaches in mUM, particularly those that could achieve higher intrahepatic drug concentrations while minimising systemic exposure.

The case for liver-directed therapy in mUM

Given that over 90% of patients with mUM develop liver metastases, and often with the liver as the dominant or only site of disease, local hepatic therapies offer a rational and potentially impactful treatment approach.8

Percutaneous hepatic perfusion (PHP) with melphalan is one such approach that delivers high-dose chemotherapy directly to the liver via the hepatic artery, followed by extracorporeal haemofiltration to reduce systemic exposure. This method allows for significantly higher hepatic drug concentrations than can be achieved with systemic chemotherapy alone, offering the potential for enhanced anti-tumour activity with reduced systemic toxicity.9

The goal is to saturate the hepatic circulation with melphalan while protecting other organs, especially the bone marrow, from its toxic effects. By isolating the liver’s blood supply, PHP creates a pharmacologic advantage for liver-dominant disease.

Other liver-directed strategies – including transarterial chemoembolisation, radioembolisation and hepatic resection – have been explored, but PHP benefits from its repeatability and systemic-sparing pharmacokinetics.10 This makes it particularly well-suited for a disease like mUM where systemic therapy options are sparse and liver failure is the predominant cause of death.

Focus on the FOCUS trial

The FOCUS trial (NCT02678572) is a pivotal phase 3 study evaluating the efficacy and safety of melphalan/hepatic delivery system (melphalan/HDS) PHP in patients with liver-dominant mUM.

This randomised, controlled, multicentre trial enrolled patients who had progressed on, or were not candidates for, immune checkpoint inhibitors or other systemic therapies. Patients with liver-only or liver-dominant metastatic disease were included. Those with liver-dominant disease were defined as bone, subcutaneous, lung or lymph nodes amenable to resection or radiation with a defined treatment plan.

Patients were randomised to receive melphalan/HDS or the investigator’s choice of best alternative care, which included options such as transarterial chemoembolisation, pembrolizumab or dacarbazine.11

The rationale behind FOCUS stemmed from prior phase 1/2 studies suggesting that melphalan/HDS PHP can achieve meaningful responses and disease control in mUM.

The FOCUS trial was initiated as a randomised study, however, relatively early into recruitment, it was apparent that significant numbers of patients were dropping out of the control arm at the point of randomisation. Following discussions with the US Food and Drug Administration, the trial protocol was amended to a single-arm study, utilising benchmark data from a systematic review of prior studies to inform the power analysis for the primary outcome.

Data have been published on both the primary analysis of the single-arm study and the results from the randomised section of the study.11,12

Despite these hurdles, the FOCUS trial provides key evidence supporting the use of PHP in the subset of mUM patients with liver-dominant disease and limited extrahepatic spread.

Demonstrating promising efficacy and clinical activity

Results from the FOCUS study and earlier trials have demonstrated encouraging clinical activity for melphalan/HDS in mUM. In the FOCUS trial, melphalan/HDS was associated with an improved overall response rate of approximately 36% compared with 2% in the best alternative care arm.12

Median progression-free survival was also significantly extended to 9.0 months versus 3.1 months, respectively. Importantly, the disease control rate, which includes patients achieving partial response or stable disease, was over 70% in the treatment arm.

Although the study was not powered for OS, trends favoured PHP with a median survival of 20.5 months. Earlier phase studies have reported a median OS duration of 19 months in treated patients, notably higher than historical controls.13 These outcomes are particularly significant given the limited effectiveness of other systemic therapies.

The FOCUS trial results support melphalan/HDS as a meaningful treatment modality for patients with mUM, especially those with unresectable, liver-only or liver-dominant disease. Further research into predictive biomarkers and combination strategies may enhance patient selection and efficacy.

An acceptable safety profile

The safety profile of melphalan/HDS therapy reflects both the chemotherapeutic and procedural aspects of the treatment. The most commonly observed adverse events include haematologic toxicities such as thrombocytopaenia, neutropaenia and anaemia, which are consistent with melphalan’s known myelosuppressive effects.14 These are typically transient and manageable with supportive care, including primary growth factors and transfusions.

Non-haematologic toxicities include hypotension, nausea and transient elevations in liver enzymes. The procedure itself requires coordination of vascular access, general anaesthesia and extracorporeal filtration, all of which introduce procedural risks. Despite these complexities, melphalan/HDS has demonstrated an acceptable safety profile when performed at experienced centres with appropriate multidisciplinary support.

Serious adverse events occur in a minority of cases, with improved safety outcomes seen over time due to procedural refinements and greater operator experience.15 Importantly, the systemic toxicity profile is far milder than that observed with comparable doses of systemic chemotherapy. This balance between high intrahepatic drug exposure and reduced systemic toxicity is central to the rationale for melphalan/HDS and is a key advantage in the treatment of mUM.

Implications for clinical practice

Melphalan/HDS PHP offers a significant addition to the limited therapeutic armamentarium for mUM. Given the liver-centric nature of metastatic spread in this disease, PHP provides a rational and targeted approach, particularly in patients with limited or no extrahepatic involvement. With demonstrated clinical activity and a manageable safety profile, PHP has the potential to serve as a bridge to extended survival and improved quality of life in a historically hard-to-treat population.

In clinical practice, PHP should be considered for patients with unresectable, liver-dominant mUM, especially those who have failed or are ineligible for systemic options. Its integration into treatment pathways requires careful multidisciplinary planning and access to centres equipped to perform the procedure safely. Patient selection remains critical, as those with extensive extrahepatic disease or poor hepatic function may derive less benefit.

Furthermore, awareness and education about PHP among oncologists – particularly outside major referral centres – must improve to ensure equitable access to this promising therapy.

Shaping the future of liver-directed therapy for mUM

Looking ahead, several key research areas are likely to shape the future of liver-directed therapy in mUM. Combination strategies involving PHP and systemic agents – particularly immune checkpoint inhibitors, targeted therapies or investigational T-cell engagers – may enhance overall outcomes.16 Additionally, translational research aimed at identifying biomarkers of response to PHP could refine patient selection and maximise benefit.

Longitudinal studies and real-world data will be essential to understanding the durability of response, optimal sequencing with other therapies and quality-of-life outcomes. Further exploration of repeat treatment strategies and alternative dosing schedules may also enhance the clinical utility of PHP.

In conclusion, melphalan/HDS PHP represents a targeted and effective therapeutic option for patients with liver-dominant mUM. Its development addresses a significant unmet need in a rare and challenging malignancy. Continued clinical integration, coupled with robust research, will be key to fully realising its potential in improving outcomes for patients with mUM.

Author

Matthew Wheater BM FRCP PhD
Consultant oncologist, University Hospital Southampton NHS Foundation Trust

References

  1. Carvajal R et al Advances in the clinical management of uveal melanoma. Nat Rev Clin Oncol 2023;20(2):99–115.
  2. Collaborative Ocular Melanoma Study Group. Assessment of metastatic disease status at death in 435 patients with large choroidal melanoma in the collaborative ocular melanoma study (COMS): COMS report no 15. Arch Ophthalmol 2001;119(5):670–6.
  3. Kujala E, Mäkitie T, Kivelä T. Very long-term prognosis of patients with malignant uveal melanoma. Invest Ophthalmol Vis Sci 2003;44(11):4651–9.
  4. Rantala E et al. Overall survival after treatment for metastatic uveal melanoma: a systematic review and meta-analysis. Melanoma Res 29(6):561–8.
  5. Algazi AP et al. Clinical outcomes in metastatic uveal melanoma treated with PD-1 and PD-L1 antibodies. Cancer 2016 Nov 15;122(21):3344–53.
  6. Carvajal RD et al. Selumetinib in Combination With Dacarbazine in Patients With Metastatic Uveal Melanoma: A Phase III, Multicenter, Randomized Trial (SUMIT). J Clin Oncol. 2018 Apr 20;36(12):1232–9.
  7. Nathan P et al; IMCgp100-202 Investigators. Overall Survival Benefit with Tebentafusp in Metastatic Uveal Melanoma. N Engl J Med 2021 Sep 23;385(13):1196–206.
  8. Kolb M et al. Selective Internal Radiotherapy (SIRT) and Chemosaturation Percutaneous Hepatic Perfusion (CS-PHP) for Metastasized Uveal Melanoma: A Retrospective Comparative Study. Cancers (Basel) 2023 Oct 11;15(20):4942.
  9. Hughes MS et al. Results of a Randomized Controlled Multicenter Phase III Trial of Percutaneous Hepatic Perfusion Compared with Best Available Care for Patients with Melanoma Liver Metastases. Ann Surg Oncol 2016 Apr;23(4):1309–19.
  10. Rowcroft A et al. Systematic review of liver directed therapy for uveal melanoma hepatic metastases. HPB (Oxford) 2020 Apr;22(4):497–505.
  11. Zager JS et al. Efficacy and Safety of the Melphalan/Hepatic Delivery System in Patients with Unresectable Metastatic Uveal Melanoma: Results from an Open-Label, Single-Arm, Multicenter Phase 3 Study. Ann Surg Oncol 2024;31:5340–51.
  12. Zager JS et al. An Open-label, Randomized Study of Melphalan/Hepatic Delivery System Versus Best Alternative Care in Patients with Unresectable Metastatic Uveal Melanoma. Ann Surg Oncol 2025;32:4976–88.
  13. Meijer TS et al. Percutaneous Hepatic Perfusion with Melphalan in Patients with Unresectable Ocular Melanoma Metastases Confined to the Liver: A Prospective Phase II Study. Ann Surg Oncol 2021 Feb;28(2):1130–41.
  14. Karydis I et al. Percutaneous hepatic perfusion with melphalan in uveal melanoma: A safe and effective treatment modality in an orphan disease. J Surg Oncol 2018 May;117(6):1170–8.
  15. de Leede EM et al. Percutaneous Hepatic Perfusion (PHP) with Melphalan as a Treatment for Unresectable Metastases Confined to the Liver. J Vis Exp 2016 Jul 31;(113):53795.
  16. Sorrentino FS et al. Uveal melanoma: Recent advances in immunotherapy. World J Clin Oncol 2024 Jan 24;15(1):23–31.

P2Y12 inhibitor monotherapy has lower MACCE risk than aspirin after PCI, study finds

Monotherapy using clopidogrel or ticagrelor is associated with lower rates of future major adverse cardiac and cerebrovascular events compared with traditional aspirin following percutaneous coronary intervention (PCI), researchers have found.

An analysis of data from five randomised clinical trials involving 16,117 patients with stents showed that after a median 3.7 year follow-up, P2Y12 inhibitor use was associated with a 23% lower risk of cardiovascular death, heart attack or stroke, compared with aspirin.

In addition, there was no significant difference in major bleeding with clopidogrel or ticagrelor compared with aspirin, the team of international researchers reported in the BMJ.

They calculated that one cardiovascular death, heart attack, or stroke would be prevented for every 46 patients taking a P2Y12 inhibitor instead of aspirin after dual therapy.

Taking into account individual outcomes, P2Y12 inhibitor therapy reduced heart attacks and stroke compared with aspirin, but all-cause death, cardiovascular death and stent thrombosis were similar between the treatments, they noted.

Despite some limitations on how the trials were designed, the results were consistent after further analyses accounting for age, sex, geographical region, smoking, previous heart attack or stroke, underlying conditions and medication history, they added.

Challenging the standard of care after PCI

Dual therapy (DAPT) is the standard of care after PCI for one to several months after the stent is inserted. Then, long-term monotherapy with aspirin is recommended irrespective of the number, type, and location of implanted coronary stents.

‘In patients who underwent PCI and had completed DAPT, monotherapy with a P2Y12 inhibitor, consisting of clopidogrel or ticagrelor, compared with aspirin was associated with lower rates of cardiovascular death, myocardial infarction or stroke, owing to lower risk of non­fatal cardiac or cerebrovascular events without an increase in major bleeding,’ the researchers concluded.

A linked editorial from UK experts said the updated study ‘supports preferential P2Y12 inhibitor monotherapy prescription over aspirin’ due to reductions in major adverse cardiac and cerebrovascular events without increasing major bleeding ‘in the medium term’.

But they added: ‘Medium term efficacy does not necessarily extend lifelong, which is the duration we advise patients to continue these medications.’

As such, they recommend the commencement of a large-scale globally representative trial directly comparing different monotherapy strategies, including discontinuation, with extended follow-up.

This ‘would benefit our understanding of the long-term impact of P2Y12 inhibitor monotherapy across the treatment class for secondary prevention following PCI’, they concluded.

A version of this article was originally published by our sister publication Pulse.

Single national formulary for prescribing in England within two years

15th July 2025

A single national formulary (SNF) for prescribing in England will be in place within the next two years under proposals, the Government has announced.

Outlined in the new NHS 10-year plan and backed by the National Institute for Health and Care Excellence (NICE), the SNF will reduce duplication and the ‘postcode lottery’ that has arisen out of locally derived formularies, the Government said.

A new formulary oversight board will be set up and given responsibility for sequencing products included in the formulary on the basis of clinical and cost effectiveness.

Prescribers such as clinicians and pharmacists will be ‘encouraged’ to use products ranked highly in the SNF. But they will ‘retain clinical autonomy’ as long as they prescribe in line with NICE guidance, the plan states.

‘The system for getting new medications to patients is needlessly complicated,’ the plan continues.

‘The process by which each local area decides which drugs are available is bureaucratic and creates a postcode lottery.

‘These local formularies do not make sense in a universal service that should provide a core standard of high-quality care to everyone.’

It is hoped that implementing a SNF will drive ‘rapid and equitable adoption of the most clinically and cost-effective innovations’.

‘We will work with industry throughout the implementation of these policies to make sure we realise these objectives together.’

An expanded medicines role for NICE

Under the new Government plan for the NHS, NICE will also be tasked with deciding what treatments are no longer providing value for money.

One analysis had suggested 30% of the costs of treatment were no longer good value for money and in some areas such as heart failure lives could be saved by treating people earlier in the pathway, the plan said.

‘To create more space for the myriad of innovations of the future, we will need to improve the outcomes and value we are delivering from innovations already in use.’

NICE will be asked to re-evaluate priority clinical pathways ‘on a rolling basis’, identifying where existing innovation should be retired, as well as instances when one technology should be sequenced after another to improve value.

‘We will link NHS compliance with these decisions to core clinical standards, best practice tariffs and incentives,’ the plan noted.

NICE’s technology appraisal process will be expanded to cover some devices, diagnostics and digital products. It will focus on those that meet the most urgent needs in the NHS and support financial sustainability, such as digital behavioural therapy for adolescents on mental health waiting lists, the Government said.

Getting medicines to patients faster

Dr Sam Roberts, chief executive of NICE, said: ‘The plan gives NICE the power to get medicines to patients faster, reduces the postcode lottery for high impact health technology, and maximises the value for money of existing innovations used in the NHS.’

And Dr Roberts outlined the ways in which NICE will work to achieve this, including new recommendations to extend access to effective treatments beyond their initial indications, providing advice and clearer decision pathways for clinicians on which treatments to try first when there are many choices for the same condition, and helping the NHS to identify and phase out innovations that no longer represent good value or best care.

A version of this article was originally published by our sister publication Pulse.

Forgo radioiodine ablation after thyroidectomy in low-risk cancer patients, say researchers

14th July 2025

Postoperative radioiodine is unnecessary for patients with low-risk differentiated thyroid cancer who have undergone thyroidectomy, a large UK randomised trial shows, potentially saving thousands of patients from undergoing the treatment.

Thyroid cancer is the seventh most common type of cancer globally, with 821,000 new cases diagnosed each year and approximately 48,000 annual deaths, as of 2022.

It has been standard practice internationally to perform a total thyroidectomy on patients with well differentiated thyroid cancer followed by postoperative radioiodine, also known as radioiodine ablation.

However, radioiodine ablation involves hospital admission and isolation, together with the possibility of treatment-related side effects, as well as the need to avoid close contact with children for several days once discharged.

Writing in The Lancet, the researchers noted controversy over this practice, pointing to previous observational studies and one randomised trial called ESTIMABL2 indicating that selected patients with low-risk differentiated thyroid cancer could avoid ablation.

As such, their recent Iodine or Not (IoN) study was a multicentre, Cancer Research UK-funded, phase three trial designed to assess whether recurrence-free survival was non-inferior after no ablation in selected low-risk thyroid cancer patients.

Recurrence-free rates without radioiodine ablation

The researchers recruited 504 patients (77% female) aged 17-80 years who had complete (R0) resection following total thyroidectomy; stage pT1, pT2, pT3 or pT3a disease; and N0, Nx or N1a disease.

All patients who had a thyroidectomy were randomly assigned 1:1 to have either no ablation or 1.1 GBq ablation, with a median 6.6 years follow-up in the ablation group and a median 6.8 years follow-up in the no ablation group.

The researchers observed patients with annual neck ultrasound and six-monthly serum thyroglobulin measurements.

They found five-year recurrence-free rates were 97.9% in the no ablation group compared with 96.3% in the ablation group in an intention-to-treat analysis.

The five-year absolute risk difference was 0.5 percentage points, they reported, showing non-inferiority was reached.

There were no treatment-related deaths and adverse events were similar between groups, with the most common being fatigue, lethargy and dry mouth.

‘The IoN trial shows that ablation (or postoperative radioiodine) can be avoided for patients with pT1, pT2, and N0 or Nx tumours with no adverse features,’ the researchers concluded.

There were only a small number of patients with pT3, pT3a, or N1a tumours in the IoN trial, meaning subgroup analyses were underpowered for these patients and care was need when applying the results of the trial to all pT3 or N1a tumours, they cautioned.

‘A clinical decision was made to not provide a definitive statement on the basis of numerically higher recurrence rates in these patients, even though the rates were not higher among patients who did not have postoperative radioiodine than those who did have ablation,’ the researchers wrote.

Change in UK policy required

The results from IoN, taken with the findings from ESTIMABL2, suggested that at least 400,000 patients with low-risk differentiated thyroid cancer worldwide could avoid radioiodine ablation after a total thyroidectomy.

‘A change in UK policy to not offer ablation to all patients with suitably low-risk disease could mean that approximately 2,500 patients could safely avoid ablation each year (if 70% of patients newly diagnosed with differentiated thyroid cancer would typically be given ablation),’ the researchers said.

Professor Allan Hackshaw, a trial investigator from UCL Cancer Institute in London, said the results showed radioactive iodine represented over-treatment in these low-risk patients.

‘The only impact of not receiving this radiotherapy was a positive one on people’s quality of life,’ he said.

This was particularly important because around a third of patients in the study were aged 40 years or younger, and many would have children to care for, Professor Hackshaw noted.

‘Being spared radioactive iodine therapy means they will be able to get back to a near normal life much more quickly, as well as receiving the emotional and physical support of their families in the weeks after their operation,’ he added.

In 2020,

Be alert to Guillain-Barré risk after RSV vaccination in older adults, says MHRA

Healthcare professionals have been urged to be alert for a small increased risk of Guillain-Barré syndrome following respiratory syncytial virus (RSV) vaccination in adults aged 60 years and older.

In updated advice, the Medicines and Healthcare products Regulatory Agency (MHRA) said this applies to all recipients of Abrysvo (Pfizer) and Arexvy (GSK). They advised being attentive to signs and symptoms of Guillain-Barré syndrome to ‘ensure early and correct diagnosis’ as well as initiating appropriate care and treatment.

Symptoms include tingling, numbness or pins and needles in the feet and hands, which is usually followed by muscle weakness and difficulty moving joints. Other symptoms can include nerve pain in the legs or back, breathing difficulties, drooping face muscles or trouble swallowing or speaking, and double vision. Paralysis can occur in some severe cases.

Early hospital treatment for Guillain-Barré syndrome can reduce severity and improve outcomes, the medicines regulator said, adding that the benefits of vaccination against RSV for older adults still outweigh the small risk of developing the condition.

The MHRA update stressed that currently there is no evidence of an increased risk of Guillain-Barré syndrome in pregnant women following RSV vaccination.

The NHS currently offers Abrysvo to adults aged 75-79 years old and to pregnant women, with the latter being effective against severe RSV infection in infants.

Arexvy is not currently available on the NHS but may be available privately for use in individuals aged 60 years and older, or those aged 50-59 years who are at increased risk of RSV.

Yellow Card reports for RSV vaccine

Up to 2 June 2025, the MHRA said it had received 21 Yellow Card reports of suspected Guillain-Barré syndrome in older adults following a vaccination with Abrysvo.

The regulator is reminding healthcare professionals and patients to continue to report side effects associated with the RSV vaccine to the Yellow Card scheme.

So far, around 1.9 million doses of Abrysvo have been given as part of the older adult RSV vaccination programme.

A post-marketing observational study in the United States in older adults estimated that Abrysvo and Arexvy were associated with nine and seven excess Guillain-Barré syndrome cases per million vaccine doses administered, respectively.

Preliminary unpublished post-marketing study data from the UK Health Security Agency (UKHSA) and Public Health Scotland studies in adults aged 75-79 years estimate a combined excess of 15 to 25 Guillain-Barré syndrome cases per million vaccine doses of Abrysvo administered.

A review of the latest UK data by the Commission on Human Medicines has advised that the ‘benefits of the vaccine outweigh the risk of developing Guillain-Barré syndrome in older adults’.

An analysis by the UKHSA published in The Lancet in March found a 30% drop in hospital admissions in 75- to 79-year-olds – who are eligible for the RSV vaccine – after the vaccination programme was introduced.

A version of this article was originally published by our sister publication Pulse.

Diagnostic AI tool ‘more accurate’ than physicians aims to ‘reshape’ healthcare

A generative artificial intelligence (AI) diagnostic tool has been launched by Microsoft, which it claims is four times more accurate than experienced physicians and has the potential to ‘reshape healthcare’.

The Microsoft AI Diagnostic Orchestrator (MAI-DxO) was tested on 304 complex diagnostic cases published in the New England Journal of Medicine and achieved an 85% diagnostic accuracy rate, according to a recent study.

The accuracy of diagnoses by 21 physicians working in the US or UK was also tested in comparison, with a median of 12 years’ of experience. Of these, 17 were primary care physicians and four were in-hospital generalists. Their average diagnostic accuracy was 19.9%.

The research also looked at the cost of the tool, claiming it saved 20% in diagnostic costs compared to physicians.

The study, called Sequential Diagnosis with Language Models, which was not peer reviewed, was published on arXiv – a research sharing platform maintained by Cornell University.

Challenging the traditional structure of healthcare with AI

‘When doctors begin their careers, they face a key decision: should they become generalists, with broad knowledge across many medical areas, or specialists, with deep expertise in a narrow field? This division is necessary because medicine is too vast for any one person to master in full,’ the study said.

‘To manage this complexity, healthcare systems rely on collaboration: generalists and specialists work together in clinics and hospitals, combining their diverse and complementary knowledge and decision-making skills to provide patients with the comprehensive and effective care that they need.

‘Today, frontier AI language models are challenging this traditional structure. These advanced systems show remarkable versatility, demonstrating both broad and deep medical understanding, and the polymathic ability to reason across specialties. In effect, they combine the generalist’s range with specialists’ depth.

‘As a result, they significantly outperform individual physicians on complex diagnostic problems, such as those featured in the NEJM clinicopathological conference cases. Our findings highlight this impressive capability. Expecting any single doctor to master the full range of such cases is unrealistic.’

Eliminating  or complementing doctors?

This study comes after Microsoft co-founder Bill Gates, suggested earlier this year on The Tonight Show that AI could mean doctors would not be needed ‘for most things’ within a decade.

However, Microsoft added that its MAI-DxO system is meant to ‘complement’ doctors and other healthcare professionals.

It said: ‘While AI is becoming a powerful tool in healthcare, our team of practising clinicians believes AI represents a complement to doctors and other health professionals.

‘While this technology is advancing rapidly, their clinical roles are much broader than simply making a diagnosis. They need to navigate ambiguity and build trust with patients and their families in a way that AI isn’t set up to do.

‘Clinical roles will, we believe, evolve with AI giving clinicians the ability to automate routine tasks, identify diseases earlier, personalise treatment plans, and potentially prevent some diseases altogether. For consumers, they will provide better tools for self-management and shared decision making.’

AI has a large role in the new NHS 10-year plan, which was published this week. This includes an AI scanning tool for NHS systems in order to flag safety issues in real time.

Last year, a white paper from the Microsoft company Nuance found that over a third of patients are in favour of clinicians using AI in consultations to improve documentation processes such as clinical letters.

Research by the Health Foundation also found that three quarters (75%) of the public support sharing some of their personal health data to aid the development of AI systems in the NHS.

A version of this article was originally published by our sister publication Healthcare Leader.

Calls for AI roadmap as teledermatology set to become standard practice

11th July 2025

Dermatologists working in the NHS are calling for an artificial intelligence (AI) roadmap to help them navigate the use of assistive technology as its use becomes increasingly mainstream.

A new report from the British Association of Dermatologists (BAD) highlights the need for urgent action and urges the Government and regulators to implement changes that ensure patient safety while promoting the adoption of suitable AI technology.

Without stronger regulation and targeted workforce training, there is a concern that the promise of diagnostic AI in dermatology outlined in the new NHS 10-year plan will not be effectively harnessed. Underpinned by both the need to innovate while improving patient outcomes, the report identifies five urgent priorities for policymakers: a clear regulatory framework, investment in digital infrastructure, better data quality and diversity, clearer market signals for developers, and enhanced training for healthcare professionals.

Dermatology waiting lists on the rise

AI technology is already being used in many NHS settings. As demand for dermatology services increases, dermatologists are urging national policymakers to take decisive action to help alleviate this burden. One in four people in England and Wales visit their GP for a dermatologically related condition every year. Since January 2025, the waiting list for dermatology services has been 118% above its July 2020 level – one of the highest increases among all clinical specialities.

One major driver of this pressure is the sharp rise in urgent skin cancer referrals. Over the last decade, referrals have jumped by 170%, but according to NHS data, only 6% of referrals result in a confirmed skin cancer diagnosis. The remaining cases are either benign or non-urgent but place a significant burden on already stretched services, as well as contributing to patient anxiety.

Dr Rubeta Matin, consultant dermatologist at Oxford University Hospitals NHS Foundation Trust and chair of the BAD’s AI Working Party Group, said: ‘Diagnostic AI has huge promise across many disease areas in dermatology, but we are keen to see if it can safely triage and reduce the huge number of patients referred with a suspected skin cancer who don’t need to be seen. If it can, this would significantly ease capacity pressures and help ensure that those who do need care are seen more quickly.’

Clinical input for diagnostic AI development and adoption

The report authors said that ‘it is not inevitable that diagnostic AI in dermatology will improve current services’ and warned that innovation and the implementation of AI in dermatology must be done without exacerbating existing pressures or inequalities.

Dr Tamara Griffiths, president of the BAD, commented: ‘We are fortunate to have an innovative and dynamic AI industry in the UK, but it needs support and unambiguous signals from the NHS about the type of tools which are required.’

She continued: ‘In return, the NHS needs a clear regulatory framework and appropriate clinical input to ensure it is adopting tools which are relevant and safe. Most importantly, we need to ensure that when AI is adopted, it improves the overall patient experience and outcomes in terms of safety, convenience, and waiting times.’

Commenting after the launch of the NHS 10-year plan, she noted that while ‘dermatology is seen as especially well-positioned to capitalise on technologies like teledermatology and AI diagnostics’, its potential ‘will only be realised with the right regulation, digital infrastructure and clinical safeguards in place’.

‘Without these, such tools risk falling short of expectations,’ she concluded.

A version of this article was originally published by our sister publication Nursing in Practice.

Can plastic waste really be turned into sustainable paracetamol?

10th July 2025

E. coli is capable of turning plastic waste into paracetamol and could offer a new and sustainable production method, according to a new study by the University of Edinburgh.

Published in the journal Nature Chemistry, the researchers looked to combine traditional chemistry with engineering biology to create sustainable chemicals using the plastic polyethylene terephthalate (PET).

They used a biocompatible Lossen rearrangement to transform PET-derived terephthalic acid from industrial waste into paracetamol using a harmless and genetically reprogrammed E. coli catalysed by phosphate.

Carried out at room temperature and in less than 24 hours, 92% of the final yield was paracetamol.

The researchers said this paves the way ‘for a general strategy to bioremediate and upcycle plastic waste in native and engineered biological systems’.

Sustainable paracetamol production

The process created virtually no carbon emissions, unlike traditional paracetamol production methods reliant on fossil fuels such as crude oil.

What’s more, over 350 million tons of PET waste is produced annually, often ending up in landfill or polluting oceans. While PET recycling is possible, the resulting products continue to contribute to plastic pollution throughout their lifetime.

This innovative paracetamol production process generates a circular economy by producing sustainable chemicals, reducing waste and greenhouse gas emissions and disrupting the reliance on fossil fuels.

Plastic with the ‘potential for treating disease’

Professor Stephen Wallace, UK Research and Innovation Future Leaders Fellow and chair of Chemical Biotechnology at the University of Edinburgh, said: ‘This work demonstrates that PET plastic isn’t just waste, or a material destined to become more plastic – it can be transformed by microorganisms into valuable new products, including those with potential for treating disease.’

Further development is needed before paracetamol can be produced using this method at commercial levels, the researchers said.

‘Future work will also focus on applying the biocompatible Lossen rearrangement to other chemo-enzymatic cascades and fully integrating this new-to-nature reaction within metabolically evolved microorganisms,’ they added.

Earlier this year, a study examined public and prescriber perceptions of pharmaceutical pollution in Scotland’s water environment, with a focus on eco-directed sustainable prescribing as a strategy to mitigate this pollution.

New DNA repair deficiency syndrome highlights need to tailor chemo regimens

9th July 2025

A new inherited DNA repair deficiency syndrome, which increases a patient’s risk of developing blood cancer but also leaves them susceptible to DNA damage from chemotherapy, has been identified by a UK research team, highlighting the need to tailor cancer treatment regimens to minimise toxicity in affected patients.

Writing in the journal Nature Communications, the research team, led by the University of Birmingham, reported the discovery of DIAPH1 Loss-of-function (DIAL) syndrome, which is characterised by biallelic loss-of-function mutations in the actin nucleating factor diaphanous-related formin 1 (DIAPH1 or mDia1) and causes clinical symptoms early in life.

The DIAPH1 gene mutations were identified in a single index patient, who had symptoms including microcephaly, behavioural abnormalities, intellectual disabilities, cortical blindness and seizures.

Working with neurogeneticists at University College London, researchers later identified more than 30 patients with these mutations, all of whom exhibited similar symptoms such as microcephaly, intellectual disability, impaired vision and seizures.

Among the cohort, one patient developed Hodgkin lymphoma, and another developed a large B cell lymphoma.

In addition, the clinical symptoms in this cohort were strikingly similar to the DNA repair disorders Nijmegen breakage syndrome (NBS) and Warsaw breakage syndrome, both of which are risk factors of blood cancer.

This finding prompted the team to investigate whether DIAPH1 functioned to regulate the actin-dependent DNA double strand break repair (DSBR) process, which was fundamental to maintain genome stability and prevent the onset of disease.

Through a series of cell analyses, they showed DIAPH1 was essential for facilitating homologous recombination (HR)-dependent repair of DNA double strand breaks induced by ionising radiation and the chemotherapeutic agents, camptothecin and etoposide.

‘Taken together, we identify DIAL syndrome as a previously undiscovered DSBR deficiency syndrome associated with neurodevelopmental abnormalities and tumour predisposition akin to those observed in patients with NBS,’ the study authors concluded.

‘Furthermore, we demonstrate that loss of DIAPH1 results in defective HR-dependent DNA repair, which is consistent with many of the clinical deficits exhibited by affected patients as well as the suggestion that in some cases DIAPH1 may act as a tumour suppressor.’

DNA repair deficiency syndromes and anti-cancer treatment

Study corresponding author Professor Grant Stewart, professor of cancer genetics in the Department of Cancer and Genomic Sciences at the University of Birmingham, said patients with inherited DNA repair deficiency syndromes were ‘usually children who are very sick’.

‘In addition to developmental abnormalities that affect many different organs, these patients are often prone to developing cancer. Unfortunately, the DNA repair defect that is present in every cell in their body makes them extremely sensitive to the therapy that would normally be used to treat their cancer,’ he said.

‘While inherited DNA repair deficiency syndromes like DIAL syndrome are rare, it is critical to identify children with these conditions early in life – especially before starting anti-cancer treatment to avoid life-threatening consequences.’

Professor Stewart said the research was crucial for affected patients and their families, as it not only provided a diagnosis for a previously unrecognised genetic condition but also informed parents and medical professionals about disease progression, potential complications, and cancer risks.

He added: ‘Additionally, this research will help guide oncologists in adjusting cancer treatment protocols to minimise toxicity in affected patients, potentially improving both their quality of life and treatment outcomes.’

The researchers want to ensure DIAL syndrome is included in sequencing panels to allow newborn diagnosis and give the opportunity for more personalised therapeutic regimes to be used if individuals with DIAL syndrome develop cancer.

Dr Laura Danielson, children’s and young people’s research lead at Cancer Research UK – the charity that funded the research – welcomed the study’s findings and the potential for more personalised care.

‘It’s a powerful example of how research can make a real difference, even in the rarest of cases,’ she said.

AI-led early warning system to flag NHS safety issues in real time

8th July 2025

The Care Quality Commission (CQC) will carry out ‘rapid response inspections’ of healthcare providers based on prompts from a new artificial intelligence (AI)-led early warning system, the Government has announced.

Forming part of its NHS 10-year plan, this system will see the CQC moving to a ‘more data-led regulatory model’.

The new national AI-powered early warning system will build on the capabilities in the Federated Data Platform (FDP) to monitor real-time data to flag safety issues. Where problems are detected, inspection teams will be deployed quickly to assess service quality.

The changes will mean that in future the CQC will use two different models of inspection:

  • Rapid response inspections, where concerns are identified and inspectors are deployed to understand the nature and significance of problems
  • Routine planned inspections, where services are independently inspected by experts – generally on a three- to five-year cycle, depending on the level of risk.

The Government also pledged to take firmer action where standards are not met by healthcare providers.

The CQC will ‘make sure persistent poor-quality care results in the decommissioning or contract termination of services or providers, no matter the setting, no matter whether the provider is in the NHS or independent sector, and no matter whether they are a GP practice or an individual NHS Trust’, the plan said.

In its first stage, a maternity outcomes signal system will launch across NHS trusts from November, to flag higher than expected rates of stillbirth, neonatal death and brain injury. But when fully implemented it could be used to identify patterns of abuse, serious injuries, deaths, or other incidents.

Health secretary Wes Streeting said: ‘While most treatments in the NHS are safe, even a single lapse that puts a patient at risk is one too many. Behind every safety breach is a person – a life altered, a family devastated, sometimes by heart-breaking loss.

‘Patient safety and power are at the heart of our 10 Year Health Plan. By embracing AI and introducing world-first early warning systems, we’ll spot dangerous signs sooner and launch rapid inspections before harm occurs.

‘This technology will save lives – catching unsafe care before it becomes a tragedy. It’s a vital part of our commitment to move the NHS from analogue to digital, delivering better, safer care for everyone.’

Extending legal powers and inspection remit

Other planned changes include extending the regulator’s legal powers and expanding its inspection remit. The current three-year time limit for the CQC to bring legal action against healthcare providers will be extended.

The CQC will also assume the function of checking ‘whether every provider (and in time, ICB) has effective freedom to speak up functions’, according to the plan.

As part of plans to reduce the number of NHS quangos, the CQC will also absorb organisations such as the Health Services Safety Investigations Body to counter and simplify the ‘regulatory tsunami that is overwhelming the system but has not led to sustained improvements in safety’, the plan said.

It follows last year’s Dash review into the CQC’s effectiveness which found it had ‘lost credibility’, including a marked increase in the time it took to re-inspect healthcare services.

Average re-inspection times increased from 87 days in 2015 to 136 days in 2024 for ‘inadequate’ ratings, and from 142 days to 360 days during the same period for ‘requires improvement’ ratings.

In addition, the CQC was plagued by IT system failures which led to the temporary ‘loss’ of hundreds of inspection reports.

CQC chief executive Sir Julian Hartley said: ‘We welcome this ambitious, future-focused plan for a world-class NHS that truly delivers for patients and the public.

‘We will develop a stronger focus on all dimensions of quality and on tackling inequalities in access, experience, and outcomes. Our ambition to become intelligence-led will be further boosted by the development of tools that help us capture the voice and experiences of people using health and social care, which we will use alongside our own data and that held by partners to spot and act on risk earlier.

‘We are already developing our new clearer, simpler, assessment approach, and in the future our experienced teams of inspectors, led by our newly appointed Chief Inspectors, will be able to conduct more inspections and share feedback on the findings more quickly – so that providers can make faster improvements, and the public have timely information about care.’

Sir Julian added that a focus on tackling longstanding inequalities and the CQC’s unique perspective across local systems will enable the regulator ‘to understand where gaps between services impact people – and how to close those gaps’.

A version of this article was originally published by our sister publication Pulse.

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