New drug therapy for young children with severe eczema

Tue, 20 Sep 2022 03:39:00 +0100

A biologic therapy for very young children with a moderate to severe form of a common skin condition has been shown to be safe and effective in an international trial which involved University of Ұ�� clinical scientists working within the Clinical Trials Facility at Royal Ұ�� Children’s Hospital.

The study of the drug, , in inadequately controlled eczema is the first large-scale randomised double-blind trial of a monoclonal antibody (a lab-made protein that binds to certain targets in the body) for any skin disease, in patients aged six months to six years.

The international clinical study involved University of Ұ�� and clinical scientists, and was sponsored by the biotech company, .

Eczema is a chronic inflammatory skin condition with prevalence of 20 per cent or higher in children younger than six years.

It is characterised by an itchy red rash particularly on the face and the bends of the elbows and knees, as well as an increased risk of skin infection.

The study, published in The Lancet [SA(MUNF1] showed that the drug greatly improved the severity of the condition, reducing skin itching and pain within two weeks.

It also significantly improved patients’ sleep, and the quality of life of patients and their parents.

Doctors expect that the international study of 162 patients will ensure this treatment is approved for British children in the near future, following its adoption in the United States in June this year.

Lead Investigator for Ұ��, Dr Peter Arkwright, Senior Lecturer at The University of Ұ�� and Consultant in Paediatric Allergy, Immunology and Infectious Diseases at , has been investigating the use of dupilumab in children with severe eczema – also known as atopic dermatitis – at the at RMCH since August 2015.

Although the therapy is already licensed in the UK for adults and children aged six to 18, researchers see the positive results for young children as ‘the icing on the cake’.

The patients in the trial were enrolled from 31 hospitals, clinics, and academic institutions in Europe and North America from June 2020, to February 2021.

A total of 83 patients were given an injection of dupilumab under the skin, and 79 a placebo every four weeks as well as continuing on standard therapy with low-potency steroid cream for 16 weeks. Independent assessors scored the level of inflammation.

They found:

28 per cent of patients receiving dupilumab achieved a global skin score of 0 or one indicating clear or almost clear skin at week 16.
53 per cent of the patients experienced a 75 per cent improvement from the baseline in Eczema Area and Severity Index (EASI) – standardized evaluation tool – at week 16.
, an eye condition also known as ‘red’ or ‘pink’ eye, was slightly more frequent with dupilumab than placebo (five per cent vs 0 per cent), however no dupilumab-related adverse events were serious or led to treatment discontinuation.

Dr Arkwright said: “Young children and infants who have moderate-to-severe eczema have a substantially reduced quality of life. It is also incredibly stressful for their families, particularly as children’s sleep is so disturbed.

“The fact that infants and young children with moderate-to-severe eczema are inadequately controlled with creams means they have a high unmet medical need.

“We are delighted that dupilumab has provided clinically meaningful improvement, with minimal side effects.

“These pivotal trial results strongly support the global approval of dupilumab in infants and children with eczema. It will revolutionise clinical practice worldwide.”

The paper will appear here

Blood sample to help select the right early phase clinical trials for cancer patients

Mon, 22 Apr 2019 16:04:00 +0100

Scientists could help match cancer patients with no other treatment options to clinical trials with experimental medicines, by analysing the genetic faults in a sample of their blood, according to research published in Nature Medicine.

The researchers, funded by Cancer Research UK, The Christie Charity, AstraZeneca and the NIHR Ұ�� Biomedical Research Centre (BRC), demonstrated in their feasibility study that a blood test can be carried out and analysed in a timeframe that can help clinicians select a matched, targeted treatment.

Currently, enrolment to trials depends on a patient’s type of cancer or genetic data obtained from an invasive tumour biopsy, which is often months or years old and may not represent a patient’s current disease due to their tumours’ evolutionary changes over time.

Scientists from the CRUK Ұ�� Institute at The University of Ұ�� showed that a small volume of blood can contain up-to-date genetic information about a patient’s cancer to inform treatment choices. In this feasibility study of the first 100 patients, 11 were enrolled onto an available and molecularly matched clinical trial.

Dr Matthew Krebs, the lead clinician of the study from The University of Ұ�� and The Christie Hospital NHS Foundation Trust, said: “This study is bringing clinicians and scientists together to develop a new approach to treating patients with advanced cancers.

“Historically, patients who have exhausted other options but are still reasonably well might access a clinical trial based on their cancer type, but without that new therapy being targeted to their tumour's particular genetic profile. Now, that paradigm is shifting toward personalised medicine. By understanding the genetic faults underpinning a patient’s cancer from a blood test, as demonstrated in this study, this raises the hope of matching more patients to a specific targeted clinical trial treatment with better chance of benefit.”

In the first of the two-part trial, called TARGET**, the researchers were able to collect, process and analyse blood samples from 100 patients in the Ұ�� area.

Professor Caroline Dive, the laboratory lead author of the study from the Cancer Research UK Ұ�� Institute, said: “Now that we have demonstrated the feasibility of matching clinical trials for patients who have not responded to previous treatments by analysing the tumour DNA in their blood, we are working to improve our blood testing approach. We are making the test more sensitive and adding new elements to it in order to understand more about a patient’s disease. We are also taking several blood samples over time to see if a faulty gene(s) is disappearing with treatment, or if there is emergence of a new genetic fault that could lead to treatment resistance. This would allow us to stop a failing treatment and consider new options to stay a step ahead of the disease.”

The authors caution that while this study is promising, not every patient will have identifiable and ‘druggable’ faulty genes in their blood, nor will every patient have the opportunity to receive a treatment tailored to their cancer.

The researchers now hope the second part of TARGET, which is already underway, will show how often the blood test is successful at matching patients to early phase clinical trials and the impact this has on their overall survival. There is also an option of referring patients to other clinical trial sites, if suitable matched trials are available in other parts of the country.

Rothwell et al. Utility of ctDNA to support patient selection for early phase clinical trials: The TARGET Study. Nature Medicine

*** This trial was run within the Experimental Cancer Medicine Centre (ECMC) network Ұ��, part of a wider network made of 18 adult centres and 11 paediatric locations around the UK. The ECMC network, which is funded by Cancer Research UK, the National Institute for Health Research in England, and the Health Departments for Scotland, Wales, and Northern Ireland, could allow patients based in Ұ�� access to clinical trials beyond the immediate area. The ECMC network is piloting a ‘trial finder’ technology to help identify the best trial for patients, which could offer more treatment options for people entering clinical trials

Newsroom University of Ұ������

New drug therapy for young children with severe eczema

Blood sample to help select the right early phase clinical trials for cancer patients

Newsroom University of Ұ��