Paediatric specialists in Europe have launched a consortium to work on the development of new drugs and additional therapies for children.

The Collaborative Network for European Clinical Trials for Children, otherwise known as conect4children (c4c), will be comprised of researchers from 33 academic institutions and ten industry partners from 20 European nations, as well as more than 50 third party groups and approximately 500 affiliated collaborators.

The consortium aims to leverage existing expertise and patient access in order to improve Europe’s competitiveness in making paediatric drugs.

In addition, the partners intend to create processes that can be applied to disease natural history and new treatment studies, registries, and comparisons of existing therapies.

Over the next six years, the c4c network will devise a pan-European framework to design and implement multinational clinical trials for children across Europe, while building connections with regulators.

Their efforts will also include new trial designs and quantitative methods to develop medicines for rare paediatric conditions and high medical need areas.

The University of Liverpool Institute of Translational Medicine clinical neonatologist Dr Mark Turner said: “This network will have a significant impact on how we develop much-needed innovative and improved medicines for babies, children and young people.

“A number of collaborations built up over the past decade will contribute to this pan-European research network.”

Turner will lead the development and implementation of the network, while the c4c hub in the UK will be headed by the Liverpool University’s Child Health professor Michael Beresford.

The c4c project is being funded by Europe’s largest public private partnership, Innovative Medicines Initiative 2 Joint Undertaking, which is financially supported by the European Union’s Horizon 2020 research and innovation programme and the region’s pharmaceutical industry.

Johnson & Johnson-owned Janssen Pharmaceutical has secured marketing authorisation from the European Commission for the use of its Juluca drug to treat human immunodeficiency virus type-1 (HIV-1) infection.

Juluca is a once-daily, two-drug regimen composed of ViiV Healthcare’s 50mg dolutegravir and 25mg of Janssen’s rilpivirine.

The combination of two antiretrovirals in a single pill is intended to minimise the cumulative drug exposure, as well as maintain the efficacy of standard three-drug regimens at 48 weeks.

The indication covers daily use in virologically suppressed adults who were on a stable antiretroviral regimen for a minimum of six months and without history of virological failure and known or suspected resistance to non-nucleoside reverse transcriptase inhibitor (NNRTI) or integrase inhibitor (INI).

Since ViiV Healthcare is the marketing authorisation holder, it will be responsible for the commercialisation of the drug in all countries across the European Union and European Economic Area.

Janssen Research & Development Infectious Diseases, Late Development vice president and global head Brian Woodfall said: “The European Commission decision for dolutegravir/rilpivirine marks a significant milestone in our 25-year commitment to make HIV history.

“At Janssen, the driving force behind our R&D efforts is to advance science and to discover and develop transformational medicines that advance health for humanity.”

The approval is supported by results from two Phase III clinical trials during which Juluca is reported to have demonstrated non-inferiority to three and four-drug regimens in maintaining virologic suppression for 48-weeks.

Janssen Sciences Ireland partnered with ViiV Healthcare in June 2014 to investigate whether the combination of dolutegravir and rilpivirine in a single pill could be a treatment option for HIV-1.

At the 30th Commonwealth Health Minister Meeting (CHMM), government representatives committed their countries to a series of proposals designed to protect Commonwealth citizens from non-communicable diseases (NCDs).

The plans include introducing completely smoke-free public spaces, a 20% sugar tax on drinks, creating strategies for behaviour modification through public awareness campaigns and education, increasing the tax on tobacco products to 75% of the purchase price, as well as implementing graphic warnings or plain packaging.

NCDs, also known as chronic diseases, are often long in duration and are caused by a combination of genetic, physiological, environmental and behavioural factors. Examples of NCDs are cardiovascular diseases, cancers, chronic respiratory diseases and diabetes.

During her speech at the conference, Commonwealth secretary-general Patricia Scotland said: “This year’s meeting enables us to focus on fully playing our part as the Commonwealth in mobilising to tackle the growing crisis of NCDs, which is causing devastation in all our member countries, and affecting with particular severity small island developing states and low and middle-income countries.”

The CHMM also agreed on collaboration and collective action in a variety of areas, including compulsory immunisations against cervical cancer for girls aged between nine and 13 by 2025; supporting negotiating a fair price for HPV vaccines; establishing a Commonwealth evidence base for cancer control; creating a database to share pricing info; and assessing and reviewing procurement legislation.

It further endorsed the acceleration of universal health coverage (UHC) across the Commonwealth and discussed possible financing solutions, including engaging political actors at the highest levels, fiscal space analysis, better coordinating external financing at both national and international levels, and developing national health financing plans.

Global human rights and peace NGO The Elders representative Graça Machel said: “The Commonwealth represents a huge range of experiences and lessons in moving towards UHC. At all income levels there are shining examples of Commonwealth countries outperforming their peers in increasing coverage of essential health services and protecting their people from the costs of these services.”

A new clinical trial called POINT and funded by the US National Institute of Health (NIH) has demonstrated positive outcomes in some stroke patients treated with a combination of aspirin and clopidogrel.

After a small stroke or minor stroke symptoms, patients administered with the combination therapy were found to have experienced reduced risk of a secondary stroke, heart attack or other ischemic event within 90 days.

However, it was observed that the therapy led to an increase in major bleeding episodes, most of which were non-fatal and did not occur in the brain.

NIH National Institute of Neurological Disorders and Stroke director Walter Koroshetz said: “These findings are likely to have a global effect on clinical practice, as these drugs are easily available in many hospitals and clinics.

“As the benefit of the combination was concentrated in the first two weeks while risk of bleeding was constant over 90 days, it may be especially valuable in acute management of a minor ischemic stroke or transient ischemic attack (TIA).”

The trial recruited more than 4,880 patients who had a minor stroke or a TIA and compared the combination with aspirin alone.

While aspirin blocks molecules activating the clotting process, clopidogrel prevents a specific chemical from attaching to a receptor.

The study revealed that the combination of aspirin with clopidogrel prevented more ischemic events.

Of the total participants, 5% treated with the combination experienced an ischemic event within three months, compared to 6.5% treated with only aspirin.

Moreover, 0.9% subjects administered with aspirin and clopidogrel experienced severe bleeding versus 0.4% of patients in aspirin-only arm who had a major haemorrhage.

Lead author of the study and professor of neurology at Dell Medical School, University of Texas, Austin, S. Claiborne Johnston said: “We saw a real benefit with the combination therapy, but that treatment does come with a risk. Overall, the risk of severe bleeding was very small, but it was not zero.”

Based on the findings, the researchers concluded that for 1,000 patients, the combination would prevent 15 ischemic attacks but may result in five instances of major haemorrhage.

A report by the UK Business, Energy and Industrial Strategy Committee (BEIS) has stressed the need for a post-Brexit deal for the pharmaceutical industry to avoid harming patients and maintain current footprint in the sector.

Titled ‘The impact of Brexit on the pharmaceutical sector’, the report revealed that leaving the European Union (EU) without an agreement would diminish access to markets, including £11.9bn of exports, and to more than 446 million potential patients and consumers in the EU.

It predicts that patients may find it harder to access to medicines, as about 75% of UK pharmaceutical imports come from the EU.

The regulatory divergence from the European Medicines Agency (EMA) would mean that companies would need to have facilities and operations in both the UK and EU.

BEIS noted that the separation, barriers to trade and duplicate regulations could lead to increased drug prices, making the UK unappealing for new and innovative medicines.

The committee said that pursuing a deal with the EMA is required for continued access to markets. It recommends that the government seeks to retain a presence for the EMA in the UK after the agency relocates to Amsterdam.

BEIS chair Rachel Reeves said: “The government’s own analysis identifies pharmaceuticals as the sector for which UK/EU market access is the most important given the industry is reliant on friction-free border movement for their products.

“Any delays at the border faced by short-life pharmaceuticals for emergency treatments would have a hugely detrimental impact on patients.

“The government must do all it can to reach an agreement that not only protects the UK’s status as a world leader for pharmaceuticals but also allows patients across the continent to continue to be provided with the medicines they need.”

Novo Nordisk has announced an increased commitment to stem cell therapies and an expansion into other serious chronic diseases beyond type 1 diabetes.

This comes after Novo Nordisk achieved a milestone in developing human embryonic stem cell (hESC) lines under its partnership with the University of California San Francisco (UCSF).

Under the deal, the company has licensed technology designed to generate hESC lines and has the right to advance them into regenerative medicine therapies.

Earlier this month, Novo Nordisk and UCSF achieved a milestone upon opening a good manufacturing practice laboratory at the university.

The university and the company will jointly work at the facility to derive quality cell lines to be used in stem cell therapies.

The development of stem cell lines with UCSF has allowed the company to foray into areas of other serious chronic diseases.

Novo Nordisk executive vice president and chief science officer Mads Krogsgaard Thomsen said: “Finding a cure for diabetes is part of Novo Nordisk’s vision and recent progress in our stem cell research and the access to robust and high-quality cell lines raises hopes for people with type 1 diabetes.

“Our collaboration with UCSF is also expected to accelerate current and future partnerships to develop stem cell-based therapies for treatment of other serious chronic diseases.”

Research by Novo Nordisk on the differentiation of pluripotent stem cells into insulin-producing beta cells has achieved preclinical proof-of-concept.

The company is also working with Cornell University to develop an encapsulation device to protect beta cells transplanted into patients against attack from the immune system.

Novo Nordisk has already launched operations under its alliances with Biolamina and Lund University to develop stem cell-based therapies for Parkinson’s disease.

A separate collaboration with Biolamina and the DUKE National University Singapore Medical School is focusing on chronic heart failure and age-related macular degeneration.

The company intends to form additional partnerships to develop other disease treatments.

Kite Therapeutics, a subsidiary of Gilead Sciences, has announced it has leased a facility in the Netherlands to engineer cell therapies in Europe.

The 117,000ft² facility will engineer and produce cell therapies, including axicabtagene ciloleucel, a chimeric antigen receptor T cell (CAR T) therapy that is currently under review by the European Medicines Agency and approved in the US as of October 2017 under the name Yescarta. Yescarta is one of just two CAR T drugs approved in the US.

In a statement, Kite said that it hoped the new facility would make it easier to deliver cell therapies to cancer patients living in Europe.

Gilead president and CEO Dr John Milligan said: “We are pleased to be leading a new frontier of cancer innovation that is bringing hope for people living with cancer. This new European manufacturing facility will enable personalised cell therapies to be manufactured in closer geographic proximity to the patients who will receive them, potentially shortening the turnaround time for people who urgently need care.”

According to Kite, the new facility will provide 300 new jobs when fully operational in 2020.

As well as this site, Kite also recently purchased two buildings in Santa Monica and Maryland respectively, which will both be used for cell therapy research.

The Maryland site will support the work of a new Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute to develop adoptive cell therapies targeting patient-specific tumour neoantigens, mutations found on the surface of cancer cells.

Gilead senior vice president, haematology and oncology therapeutic area head Alessandro Riva said: “We are proud to be at the forefront of advancing cell therapy, which we believe has the potential to transform cancer treatment. The addition of these three new facilities and the expanded CRADA with our research collaborators at the NCI will help us bring cell therapies to more people with cancer around the world.”

Kite was acquired by Gilead for $11.9bn in October 2017 as part of the company’s increased focus on developing CAR T therapies. Gilead also agreed to acquire Cell Design Labs in a $567m deal in December last year.

US start-up Beam Therapeutics has secured a worldwide licence from Harvard University to develop and commercialise a DNA base editing platform for precision genetic medicines.

The platform was developed by the university’s chemistry and chemical biology professor David Liu, who is a co-founder of Beam.

Based on CRISPR technology, the platform includes access to base editing and associated technologies that enhance the targeting scope of base editing.

The base editing uses an engineered multi-component protein that carries an altered Cas9 to ‘unzip’ a targeted DNA helix site. This opens a small bubble where surgery can be performed on a single base without any double-stranded breaks in DNA.

The base editors directly convert the mutated form of the target base to the corrected form, and insert an additional protein component where needed to prevent the reversal of the correction.

At the same time, the engineered Cas9 cuts the unedited DNA strand, triggering the cell to repair the second strand using a base that compliments the corrected one.

Liu said: “We developed programmable molecular machines that go to a target site of our choosing in the genomic DNA of a cell and directly convert one base to another base without making a double-stranded break in the DNA.”

The platform could potentially be used to tackle a broad range of difficult-to-treat genetic disorders.

Under the agreement, Beam will make an undisclosed upfront licensing payment to Harvard, which retains the right to continue using the intellectual property for education and non-profit research.

Harvard University Office of Technology Development business development director Vivian Berlin said: “Our goal is to see this innovation develop into transformative treatments for the widest possible range of human diseases.

“Licensing the commercial rights to a new start-up ensures a rapid mobilisation of resources to fully develop and exploit the technology in this field.”