12th July 2021 saw the launch of the British Society of Pharmacy Sleep Services (BSPSS) bspss.org The Society will support through education, community pharmacists’ knowledge and understanding of various sleep disorders, to ensure that patients receive timely and appropriate advice and are signposted to other healthcare professionals where necessary.
The BSPSS is undertaking research bspss.org examining community pharmacists’ current knowledge and awareness of various sleep-related problems; to identify critical knowledge gaps and guide subsequent development of future training materials. The Society invites all community pharmacists to complete the Quiz.
The Community Pharmacists’ role in sleep problems
President of the BSPSS, Community Pharmacist Gareth Evans, says:
The BSPSS will promote the role of community pharmacy as a trusted source of advice and information for patients with sleep problems, in order to create a patient-centric network of pharmacy-based sleep services. Community pharmacists are the healthcare professionals on the high street and a highly accessible source of advice on a wide range of health issues.”
Nevertheless, he believes that community pharmacists are an under-utilised resource for patients with sleep problems. He continues “with the appropriate training, community pharmacists can triage patients seeking advice on a wide range of sleep-related problems including insomnia, snoring, sleep apnoea and even narcolepsy.”
The BSPSS will establish a network of sleep-trained pharmacists who liaise with local sleep services and perhaps in the future, refer patients directly into these services.
About the British Society of Pharmacy Sleep Services
The Board of the BSPSS are pharmacy and sleep medicine professionals, who come together to enhance sleep education for community pharmacists. The BSPSS will provide up to date sleep and circadian research, and information to pharmacists around the world, to improve patient access to valid, evidence-based sleep information at their first healthcare interaction.
The Scottish Medicines Consortium recommends Novartis’ Kesimpta®▼ (ofatumumab) for patients living with relapsing remitting multiple sclerosis (RRMS)
Novartis’ Kesimpta®(ofatumumab) is the first at-home self-administered B-cell therapy recommended by the Scottish Medicines Consortium (SMC) for adult patients with relapsing remitting multiple sclerosis (RRMS) with active disease defined by clinical or imaging features 1
Scotland has the highest average prevalence rate of MS in the UK. Amongst the MS Scottish population this rate is higher in women than men, and is more prevalent to those living in the north of the country 2,3
SMC approval is based on pivotal trial data that showed ofatumumab reduced relapses by more than half in patients with RMS (relapsing forms of MS) – the most common form of the disease – when compared with another disease modifying therapy, teriflunomide4
A high efficacy treatment that offers self-administration at home, with initial guidance from a healthcare professional, gives people with RRMS more flexibility versus other treatments and reduces the burden on patients, healthcare professionals and the NHS which is facing capacity challenges in light of COVID-195
On July 12th 2021, Novartis announced that eligible patients in Scotland will soon have access to Kesimpta® (ofatumumab), the first self-administered, targeted B-cell therapy for patients with relapsing remitting multiple sclerosis (RRMS) with active disease defined by clinical or imaging features. The news comes as the Scottish Medicines Consortium (SMC) published their final advice recommending ofatumumab for the treatment of RRMS with active disease defined by clinical or imaging features1.
Scotland has the highest average prevalence rate of MS in the UK, with about 15,750 people estimated to be living with the disease2,3. Rates are significantly higher amongst women than men, and further to this MS is more prevalent to those living in the north of the country2.
Patients with RRMS in Scotland have previously been unable to access a high efficacy, B-cell therapy through the NHS without regular visits to a clinic or hospital for treatment. For patients in the far north of the country where MS is most prevalent, that could have meant a long journey to the nearest specialist centre in Inverness6.
Ofatumumab, designed to be self-injected once a month using the Sensoready® autoinjector pen, uses specially engineered antibodies that target a subset of the body’s B-cells, a type of immune cell that abnormally damages nerves in the brain and spinal cord in patients with MS7,8. In clinical trials, ofatumumab delivered on the primary endpoint significantly reducing relapses by more than 50% when compared to teriflunomide (a commonly prescribed oral disease modifying therapy for RRMS)4.
The SMC concluded that:
Ofatumumab has the potential to reduce relapse rates, slow disease progression and improve quality of life….while some people with MS may need help from a carer or family member for administration, self-injection once a month is likely to be straightforward and will minimise the treatment burden for many people. Taking medication at home also minimises any delay in starting treatment and reduces the need for regular hospital visits1.
“It is welcome news that we will soon have access to a convenient, new medicine for the treatment of relapsing remitting multiple sclerosis in Scotland”, said Dr Javier Carod Artal, Consultant Neurologist at Raigmore hospital, NHS Highlands, Inverness.
As the first targeted B-cell therapy that can be taken at home, ofatumumab provides a much-needed high-efficacy treatment option that clinicians can prescribe from diagnosis onwards, even to patients who can’t travel for regular hospital visits for treatment.
Research published by the MS Society in June 2020 found that during the first wave of the pandemic, one in four MS patients surveyed in the UK said they had appointments delayed or cancelled, and a further 6% said they cancelled their appointments for fear of infection9.
“People living with MS need to have access to a range of different treatment options so they can work with their healthcare team to find the one that’s best for them. But, the ability to administer yourself once a month will be a game changer”, said Iain Morrison, Chief Executive, Revive MS Support. “Many people living with MS in Scotland live in very rural areas a long way from specialist MS care, and despite widespread vaccination some still tell us they are worried about catching COVID-19 if they travel to visit them.”
The SMC’s decision follows a similar recommendation from NICE earlier this year. The Final Appraisal Document (FAD) from NICE was published in April unusually quickly, just two weeks after the Medicines and Healthcare products Regulatory Agency (MHRA) granted Marketing Authorisation, recognising the value ofatumumab brings to patients and the NHS10,11. The SMC’s decision today means that eligible patients in Scotland can soon access ofatumumab.
“Innovating to solve complex challenges is at the heart of Novartis. In Scotland, where many people with MS live a long way from specialist care, ofatumumab provides an opportunity for them to access a highly effective treatment that can be self-administered at home. This is especially important whilst COVID-19 is still front of mind for many patients as well as for the NHS”, said Chinmay Bhatt, Managing Director UK, Ireland & Nordics for Novartis Pharmaceuticals. “We are proud to help improve access to medicines for patients no matter where they live in the UK, and continue to build on our heritage to reimagine medicines for all.”
4 Hauser S, Bar-Or A, Cohen J, et al. Ofatumumab versus teriflunomide in relapsing multiple sclerosis. N Engl J Med. 2020;383(6):546–557.
5 MS Society and MS Trust. Findings of a survey of MS healthcare professionals on the impact of Covid-19 on MS services in the UK. [Accessed: July 2021]
12 Smith P, Kakarieka A, Wallstroem E. Ofatumumab is a fully human anti-CD20 antibody achieving potent B-cell depletion through binding a distinct epitope. Poster presented at ECTRIMS; 14–17 September 2016; London, UK.
13 Savelieva M, Kahn J, Bagger M, et al. Comparison of the B-Cell Recovery Time Following Discontinuation of Anti-CD20 Therapies. ePoster presented at ECTRIMS; October 25–28, 2017; Paris, France.
Ofatumumab is a fully human anti-CD20 monoclonal antibody (mAb) licensed for adults with relapsing forms of multiple sclerosis (RMS) with active disease defined by clinical or imaging features, which is intended to be self-administered by a once monthly injection, delivered subcutaneously4,11. As shown in preclinical studies, ofatumumab is thought to work by binding to a distinct epitope on the CD20 molecule, inducing potent B-cell lysis and depletion11,12. Ofatumumab allows faster repletion of B-cells upon discontinuation versus other anti-CD20 mAbs, and therefore may offer flexibility in the management of RRMS13.
About ASCLEPIOS I and II studies
The ASCLEPIOS I and II studies are twin, identical design, flexible duration (up to 30 months), double-blind, randomised, multicentre, Phase III trials evaluating the safety and efficacy of ofatumumab versus teriflunomide in adults with relapsing forms of multiple sclerosis (RMS)4. The studies were conducted in 37 countries and enrolled 1,882 patients between the ages of 18 and 55 years, with an Expanded Disability Status Scale (EDSS) score between 0 and 5.54. The primary endpoint from the ASCLEPIOS I and II studies was the annualised relapse rate (ARR), defined as the number of confirmed MS relapses per year, up to the end of the trial4.
Overall across both studies, 946 patients were assigned to receive ofatumumab and 936 teriflunomide, resulting in ofatumumab demonstrating a significant reduction in annualised relapse rate (ARR) by 50.5% (0.11 vs 0.22) and 58.5% (0.10 vs 0.25) compared with teriflunomide in ASCLEPIOS I and II, respectively (p<0.001 in both studies)4,14. Ofatumumab also showed a relative reduction in three- and six- month confirmed disability worsening (CDW) compared to teriflunomide: 34.4% (p=0.002) in three-month CDW (10.9% for ofatumumab and 15.0% for teriflunomide) and 32.5% (p=0.01) in six-month CDW (8.1% for ofatumumab and 12% for teriflunomide)4. In addition, results demonstrated a significant relative reduction in the mean number of both Gd+ T1 lesions (97.5% (0.01 vs 0.45) and 93.8% (0.03 vs 0.51), respectively, both p<0.001) and new or enlarging T2 lesions (82% (0.72 vs 4.00) and 84.5% (0.64 vs 4.15) versus teriflunomide, respectively, both p<0.001)4. The most important and frequently reported adverse events for ofatumumab are upper respiratory tract infections, systemic injection-related reactions, injection-site reactions, and urinary tract infections4.
About multiple sclerosis (MS)
There are approximately 130,000 people with MS in the UK, and each year around 7,000 people are newly diagnosed with the condition15. MS is a chronic disorder of the central nervous system (CNS) that disrupts the normal functioning of the brain, optic nerves and spinal cord through inflammation and tissue loss16. The evolution of MS results in an increasing loss of both physical and cognitive functions (e.g. mobility problems, numbness, bladder and bowel problems, and problems with thinking, learning, and planning)17. There are three types of MS: relapsing-remitting MS (RRMS), secondary progressive MS (SPMS) and primary progressive MS (PPMS)18. Patients with relapsing forms of MS (RMS) – including RRMS and SPMS with active disease – experience distinct attacks of symptoms, known as relapses19,20. Around 85% of people are considered to have RRMS at their point of diagnosis20. SPMS, which typically follows from an initial RRMS course, is characterised by a gradual worsening of neurological function over time and can be described as active (with relapses and/or evidence of new magnetic resonance imaging [MRI] activity) or not active (no evidence of current activity)19,21.
About Novartis in multiple sclerosis (MS)
Novartis has a strong ongoing commitment to neuroscience and to bringing innovative treatments to patients suffering from neurological conditions where there is a high unmet need. The Novartis MS portfolio includes Gilenya®▼ (fingolimod, an S1P modulator), which is licensed in the UK and Europe for the treatment of adults and children aged 10 years and older with highly active relapsing-remitting MS (RRMS). Mayzent®▼ (siponimod) is licensed in the UK and Europe for the treatment of adult patients with secondary progressive MS (SPMS) with active disease evidenced by relapses or imaging features of inflammatory activity. Extavia® (interferon beta-1b for subcutaneous injection) is licensed in the UK and Europe to treat people with RRMS (≥2 relapses in the last 24 months), people with SPMS with active disease (evidenced by relapses) and people who have had a single clinical event suggestive of MS with an active inflammatory process.
Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for pridopidine for the treatment of ALS.
Prilenia recently received a positive opinion from the Committee of Orphan Medicinal Products (COMP) in the EU for Orphan Drug Designation for treatment of ALS and is expecting to receive the European Commission decision soon.
Pridopidine acts as a highly selective and potent Sigma-1 receptor (S1R) agonist. Extensive safety data demonstrate pridopidine has a favorable safety and tolerability profile. Pridopidine is currently being assessed in the HEALEY ALS Platform Trial in the US, the first for ALS. The pridopidine regimen enrolled its first participant in January 2021 and is on track to generate results in H2 2022. Pridopidine is also being studied for the treatment of Huntington’s Disease (HD) in a global Phase 3 clinical trial that just met the 50% enrollment milestone ahead of schedule.
Pridopidine is an exciting compound that offers potential disease-modifying treatment for patients with ALS. Orphan Drug Designation will speed development and simplify the approval pathway should efficacy be demonstrated in ongoing trials, which will be good news for patients with ALS.
Dr Jeremy Shefner, MD, PhD, Kemper and Ethel Marley Professor and Chair of Neurology of Barrow Neurological Institute and Regimen Lead Investigator for the Pridopidine Regimen
“We are pleased to have been granted Orphan Drug Designation by the FDA for pridopidine for the treatment of ALS”, said Dr. Michael R. Hayden, CEO and Founder of Prilenia. “This marks significant progress towards our goal of advancing pridopidine to address the unmet medical need for ALS.”
The Orphan Drug Designation qualifies Prilenia for various development and commercial incentives in the U.S., including a waiver of the new drug application fee and seven years of market exclusivity following approval.
About Pridopidine
Prilenia’s lead asset is pridopidine, a first-in-class, highly selective S1R agonist investigational drug. Pridopidine has an established safety profile and therapeutic potential in several neurodegenerative diseases affecting adults and children. Prilenia acquired pridopidine from Teva in 2018. Pridopidine is currently in late-stage clinical development for ALS and HD.
Pridopidine for ALS
Compelling preclinical data supports the potential use of pridopidine as a therapeutic for ALS. In ALS SOD1G93A motor neurons (MNs), pridopidine exerts neuroprotective effects via activation of the S1R. Specifically, pridopidine improves BDNF (brain-derived neurotrophic factor) and GDNF (glial cell line-derived neurotrophic factor) axonal transport, restores synaptic activity and neuro-muscular junction (NMJ) function, and increases neuronal survival. In vivo, pridopidine reduces toxic protein aggregates and ameliorates muscle fiber wasting. Previous clinical data also suggests that S1R is a promising target for ALS therapy, indicating that S1R activation may enhance bulbar and speech function in ALS patients. The sigma 1 receptor has been genetically validated for ALS, as patients with mutations in this gene develop ALS.
The Organization for Human Brain Mapping (OHBM) is an international society dedicated to fostering our understanding of brain organisation and function using neuroimaging techniques. Within the Society, various Special Interest Groups (SIGs) are dedicated to the promotion of shared interests, such as open science or trainees education.
The annual OHBM competition highlights an ongoing aim of our BrainArt SIG, namely to foster the dialogue between artists and scientists and blurring the line between Art and Science. We believe that the exchange of ideas and tools between the two disciplines encourages the development of novel approaches to scientific data visualisation, and promotes the exploration of different perspectives on human brain structures and functions. Researchers, scientists, and everyone in between are encouraged to submit their original work. In 2021, we accepted submission for 5 awards categories (for a teaser, check out here):
Big Data & Me – a new category in celebration of this year’s BrainArt Exhibition
Beautiful Mistake – anything resulting from a artifact, bug, or failed attempt
Pictures to Prose – BrainArt poem or short story
Still Images – any BrainArt image
Videos and Animations – any BrainArt audio-visual or animated contribution
We received a total of 79 submissions and the announcement of the winners can be found here.
The OHBM BrainArt SIG consists of:
AmanPreet Badhwar, MSc, PhD, Centre de recherche de l’Institut universitaire de gériatrie de Montréal (CRIUGM), Université de Montréal
Valentina Borghesani, MSc,PhD, Centre de recherche de l’Institut universitaire de gériatrie de Montréal (CRIUGM), Université de Montréal
Sridar Narayanan, PhD, McConnell Brain Imaging Centre, McGill University
Ting Xu, Child Mind Institute
Zoltan Nagy, MSc, PhD, University of Zurich
Roselyne Chauvin, PhD, Donders Research Institute for Brain, Cognition and Behaviour
Peter Kochunov, University of Maryland School of Medicine
Désirée Lussier, MSc,, PhD, Centre de recherche de l’Institut universitaire de gériatrie de Montréal (CRIUGM), Université de Montréal
Anastasia Brovkin, University Medical Centre Hamburg Eppendorf, Hamburg University
Calvert Reconnections has today opened its doors – transforming the one-time Lake District home of poet William Wordsworth into a neurorehabilitation centre. It is located at Old Windebrowe, a Grade II listed Tithe barn on the outskirts of the bustling market town of Keswick in the Lake District. The centre is the UK’s first residential brain injury rehabilitation programme combining traditional clinical therapies with physical activity in the outdoors.
Giles Mounsey-Heysham, Chairman of the Lake District Calvert Trust (LDCT) Trustees said:
The opening of our new centre is the culmination of years of meticulous planning by the Lake District Calvert Trust. It is a landmark moment for the brain injury rehabilitation market and for us. Working with leading clinicians and academics, our new rehabilitation centre will provide a ground-breaking, world-class rehabilitation programme tailored to support individuals in their recovery.
Calvert Reconnections will actively seek to incorporate a wide range of outdoor activities into each participant’s rehabilitation programme. This may range from reflective activities such as fishing, bird watching or a nature walk to higher adventure activities such as horse riding, canoeing, rock-climbing and abseiling – all closely assessed and graded to a person’s interests and functional abilities.
A new service for brain injury survivors in the Scottish Borders has been launched following a £165,000 funding boost from the National Lottery Community Fund. Dynamic Community Fusion (DCF), a Galashiels-based social enterprise, has been awarded two years of funding to deliver the new Reconnect with Confidence programme for people living with brain injuries or neurological conditions. The initiative builds on the success of DCF’s previous Reconnect programme, which helped people overcome isolation and loneliness. This will continue to be a key element of the new Reconnect with Confidence programme, with additional financial and benefits guidance. The programme will support more than 100 people in the Scottish Borders over the next two years. Online delivery will move to community based support across the region as restrictions allow.
Service Manager at DCF, Peter Cockburn, says:
Feelings of loss, isolation and loneliness are a common experience for people with neurological conditions, particularly following brain injury. These feelings can occur because of personality change, loss of self-identity, breakdown of relationships, difficulties socialising, or altered financial situations. “These issues have been exacerbated by the Covid-19 pandemic with additional fears around financial management and benefit support.
People will be offered benefits advice, financial guidance, and support to engage in meaningful activities to improve their physical and mental wellbeing. Help will be given to try different activities, access social groups or voluntary opportunities. Participants can also become DCF volunteers or befrienders.
David Horder, aged 61 and from Roxburghshire, benefited from previous DCF programmes after a stroke left him with cognitive difficulties. A regular at support groups and social gatherings, David struggled with lockdown restrictions, but with DCF support, mastered online shopping, avoided phone scammers, completed online forms and regularly posted music and photos of his craft projects on social media. “I wanted to cheer people up and inspire then to keep going, even after a life-altering event. I’m currently completing a giant jigsaw puzzle – about 10 pieces a day. I also walk daily, keep up with my music lessons, and volunteer. The service has helped me a lot. Being alone during lockdown was hard and it was great to receive regular calls from the DCF team. That wee bit of contact helped me not feel so alone. They’re a great group of people who help in ways that I didn’t know were possible. A big ‘thank you’ to them!”
The design for a new phase III, double-blind, randomised trial for opicapone has been presented for the first time at the European Academy of Neurology (EAN) congress. Around 50% of patients with PD experience some degree of motor complications within 2–5 years of starting L-dopa/DDCI therapy and almost all develop such symptoms after 10 years of L-dopa/DDCI therapy.9,10 Opicapone is a once-daily catechol-O-methyltransferase (COMT) inhibitor, approved in Europe for the treatment of end-of-dose motor fluctuations in adult PD patients on L-dopa/DDCI therapy.11 The EPSILON (Early Parkinson with L-dopa/DDCI and OpicapoNe) study will investigate the use of opicapone earlier in the treatment pathway, prior to the appearance of motor complications, over the course of 6 months, to explore its potential to enhance the benefit of L-dopa/DDCI in early-stage PD.1
Design of phase III study to investigate the efficacy of opicapone in early-stage Parkinson’s disease (PD) as an add-on to stable levodopa/dopa decarboxylase inhibitor (L-dopa/DDCI) therapy has been presented for the first time.1
A design for an additional new study to evaluate the potential for opicapone as a first-line option to address ‘wearing off’ for patients on L-dopa/DDCI was also presented.2
Designs for three further opicapone studies were also shared at EAN 2021, alongside new data from the real-world OPTIPARK study on the influence of age, disease duration and onset of motor fluctuations (MF) on opicapone treatment outcomes3-8
The design for a new randomised, prospective, open-label exploratory trial, ADOPTION (eArly levodopa with Opicapone in Parkinson’s paTients with motOr fluctuatioNs), was also presented at the congress.2 As with EPSILON, the study will evaluate the potential for earlier opicapone use, this time as a first-line add-on treatment for patients on L-dopa/DDCI experiencing ‘wearing off’.2
Lead author for both study designs, Professor Joaquim Ferreira, commented:
We are pleased to share the designs for these new studies exploring the potential for opicapone as a first-line treatment for both early motor complications and early-stage Parkinson’s disease before motor complications have occurred. Previous clinical studies have demonstrated the efficacy of opicapone in the treatment of end-of-dose motor fluctuations so it will be interesting to see if this is reflected at different points in the treatment pathway. We look forward to sharing data from these studies as they become available.
Three further study designs will be presented at the congress; these studies have been developed to investigate the impact of opicapone on L-dopa pharmacokinetics at different L-dopa/carbidopa-optimised treatment regimens, PD-associated sleep disorders and motor fluctuation-associated pain, all in patients with end-of-dose motor fluctuations.3-5 In addition to this, a wealth of data will be shared from the OPTIPARK real-world study, revealing the impact of various factors, including age, disease duration and onset of MF, on the effectiveness of opicapone.6-8
Professor Soares da Silva, Director of Research & Development of Bial, shared his thoughts: “More than 10 million people worldwide are living with Parkinson’s disease,12 a condition that can have an enormous impact on quality of life. At Bial we are committed to improving the lives of patients with Parkinson’s through our medical research and ongoing support and resources. A key part of this is our dedication to new avenues of research for existing treatments. We are proud to be presenting designs for so many new opicapone studies at EAN 2021, as well as being able to share such a range of data from our previous trials.”
BIAL is presenting opicapone data from 22 abstracts at the EAN 2021 congress.
Key abstracts include:
Abstract no EPO-443: Ferreira J et al. The EPSILON (Early Parkinson with L-dopa/DDCi and OpicapoNe) study in early Parkinson’s disease: design and rationale of a phase III, double-blind, randomized, placebo-controlled study.1
Abstract no EPO-444: Ferreira J et al. The ADOPTION (eArly levodopa with Opicapone in Parkinson’s paTients with motOr fluctuatioNs) study in Parkinson’s disease: design and rationale of a randomized prospective, open-label exploratory trial.2
Abstract no EPO-442: Ferreira J et al. Study design to assess the effect of once-daily opicapone on levodopa pharmacokinetics at different levodopa/carbidopa-optimised treatment regimens.3
Abstract no EPO-744: Chaudhuri KR et al. The OCEAN (OpiCapone Effect on motor fluctuations and associated pAiN) study in Parkinson’s disease: design and rationale or a randomized double-blind placebo-controlled trial.5
Abstract no #EPO-300: Costa R et al. The OASIS (OpicApone in Sleep dISorder) study in Parkinson’s disease: design and rationale of an open-label, single-arm, pilot trial.4
Abstract no #EPO-745: Mohamed B et al. Influence of demographic characteristics in the effectiveness of opicapone in Parkinson’s disease patients with motor fluctuations: findings from the real-world OPTIPARK study.6
Abstract no #EPO-746: Warnecke T et al. Influence of disease duration in the effectiveness of opicapone in Parkinson’s disease patients with motor fluctuations: findings from the real-world OPTIPARK study.7
Abstract no #EPO-260: Jost W et al. Influence of onset of motor fluctuations in the effectiveness of opicapone in Parkinson’s disease patients with motor fluctuations: findings from the real-world OPTIPARK study.8
References
Ferreira J et al. The EPSILON (Early Parkinson with L-dopa/DDCi and OpicapoNe) study in early Parkinson’s disease: design and rationale of a phase III, double-blind, randomized, placebo-controlled study. EAN June 2021. Abstract No. EPO-443
Ferreira J et al. The ADOPTION (eArly levodopa with Opicapone in Parkinson’s paTients with motOr fluctuatioNs) study in Parkinson’s disease: design and rationale of a randomized prospective, open-label exploratory trial. EAN June 2021. Abstract No. EPO-444
Ferreira J et al. Study design to assess the effect of once-daily opicapone on levodopa pharmacokinetics at different levodopa/carbidopa-optimised treatment regimens. EAN June 2021. Abstract No. EPO-442
Costa R et al. The OASIS (OpicApone in Sleep dISorder) study in Parkinson’s disease: design and rationale of an open-label, single-arm, pilot trial. EAN June 2021. Abstract No. EPO-300
Chaudhuri KR et al. The OCEAN (OpiCapone Effect on motor fluctuations and associated pAiN) study in Parkinson’s disease: design and rationale or a randomized double-blind placebo-controlled trial. EAN June 2021. Abstract No. EPO-744
Mohamed B et al. Influence of demographic characteristics in the effectiveness of opicapone in Parkinson’s disease patients with motor fluctuations: findings from the real-world OPTIPARK study. EAN June 2021. Abstract No. EPO-745
Warnecke T et al. Influence of disease duration in the effectiveness of opicapone in Parkinson’s disease patients with motor fluctuations: findings from the real-world OPTIPARK study. EAN June 2021. Abstract No. EPO-746
Jost W et al. Influence of onset of motor fluctuations in the effectiveness of opicapone in Parkinson’s disease patients with motor fluctuations: findings from the real-world OPTIPARK study. EAN June 2021. Abstract No. EPO-260
Lopiano L et al. Motor and non-motor outcomes in patients with advanced Parkinson’s disease treated with levodopa/carbidopa intestinal gel: final results of the GREENFIELD observational study. Journal Neurol. 2019;266(9):2164-2176
Salamon A et al. Opicapone for the treatment of Parkinson’s disease: an update. Expert Opinion on Pharmacotherapy. 2019;20(18):2201-2207
Opicapone is a once-daily, peripherally-acting, third-generation, highly-selective COMT inhibitor.11 Opicapone works by decreasing peripheral levodopa’s conversion rate into 3-O-methyldopa, thereby prolonging the duration of levodopa’s effect in reducing the OFF-time period of PD and extending the ON-time period.11
In June 2016, the European Commission authorised ONGENTYS® (opicapone) as an adjunct therapy to preparations of levodopa/DOPA decarboxylase inhibitors (DDCIs) in adult patients with PD and end-of-dose motor fluctuations who cannot be stabilised on those combinations.11 In Europe, opicapone is currently marketed in Germany, United Kingdom, Spain, Portugal, Italy and Switzerland.
BIAL entered into an exclusive licensing agreement with Neurocrine Biosciences, Inc. in February 2017 for the development and commercialisation of opicapone in the U.S. and Canada.
In November 2019, ONGENTYS® (opicapone) was approved by the regulatory authorities of South Korea and is commercialised by BIAL’s partner SK Chemicals Co., Ltd.
In April 2020, the U.S Food and Drug Administration (FDA) approved ONGENTYS® (opicapone) as an add-on treatment to levodopa/carbidopa in patients with Parkinson’s disease experiencing “off” episodes. ONGENTYS® (opicapone) is marketed in Japan by BIAL’s partner Ono Pharmaceutical Co., Ltd., after approval of the Japanese authority in June 2020.
ONGENTYS® (opicapone) is also marketed in Finland since May 2021 by BIAL’s partner NordicInfu Care AB
UKABIF (UK Acquired Brain Injury Forum) is calling for social workers to be trained and informed around acquired brain injuries following a review into the death of a Brighton man.
A review into the death of 42 year old ‘James’, who had an acquired brain injury (ABI), has found the current safeguarding system to be insufficient and a lack of expert knowledge in brain injury among the agencies working to protect him.
Now, UKABIF has written to Social Work England and the Secretary of State for Health, gathering more than 100 signatures from experts across health, social work and law in support of their call for improved training for social workers.
James died in July 2019 and his death led to a Safeguarding Adult Review (SAR) looking at the involvement of Brighton & Hove Health and Adult Social Care, Brighton & Hove CCG/Sussex NHS Commissioners, Sussex Police, Brighton and Sussex University Hospitals Trust, Brighton & Hove Housing Department, Money Advice Plus and Brighton and Hove Safer Communities Team.
The Review looked at the period from December 2016, around the time the local authority began a formal safeguarding enquiry, until James’ death. During this period, there was multi agency involvement with concerns around James’ substance misuse, vulnerability and ability to live independently.
His acquired brain injury was not taken into account by the professionals involved in James’ care and the review concluded ‘the assessments undertaken of James’ needs did not take sufficient account of his ABI and in particular the absence of capacity assessments informed by expertise in brain injury meant the care provided to him was ineffective.”
The review stated: “…the safeguarding system within Brighton & Hove is insufficiently developed to enable people with acquired brain injury to be safe.”
Chloe Hayward, UKABIF Executive Director, said: “Sadly, the issues raised by this SAR are not unique to James or to Brighton & Hove. It identified significant failings and a complete failure to assess mental capacity. Underpinning all of this is an acknowledged absence of ABI knowledge by social workers. We believe social workers play a vital role in supporting individuals and families affected by ABI but cannot do so effectively without the knowledge they need to practice to the best of their abilities.
We want to work with Social Work England and look at how social worker training can take into account the needs of people with brain injury.
The Coroner issued a Regulation 28 report following the case which gives all the authorities involved 56 days to produce a future plan.
Dr Mark Holloway, a social worker and brain injury case manager, was an expert witness for the Coroner leading the case into James’s death. He added: “Acknowledged and serious failings in James’ support and treatment are a personal tragedy for him and his family. However, these failings are representative of a systemic issue based in an absence of training and of knowledge by social workers of the impact of ABI upon an individual’s functioning and behaviour.
“Experience from across the country demonstrates that a lack of basic knowledge (and lack of interdisciplinary working) leads to recognisable and often preventable harm. Presently social workers are not being given the tools they require to carry out their vital role. Until they are, more harm will be caused and statutory duties will remain unmet. Individuals and families affected by ABI need the best support possible, knowledgeable social workers, equipped to carry out their roles, play a significant part in that process.”
Tracy, one of James’ sisters, said: “As the SAR into our brother’s life and death has shown, the services that were supposed to help and protect him could not do so because they did not understand his brain injury and how this affected him.
“He needed guidance and support in every aspect of his life and most importantly he needed safeguarding, to protect him from others who took advantage of his vulnerability and exploited him in every way possible. He was left unprotected and unsafe – he could have been helped instead. As a family we hope that the lessons learnt from his death can help others, so other families do not have to go through what we have, and that people with a brain injury can be supported and given hope.”
UKABIF raises awareness of acquired brain injury and works to ensure that people with ABI have early access to local, specialist neurorehabilitation and follow up services in the community.
On June 8th, the US Food and Drug Administration approved Aduhelm (aducanumab) for the treatment of Alzheimer’s. Aduhelm was approved using the accelerated approval pathway, which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatments. Accelerated approval can be based on the drug’s effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients, with a required post-approval trial to verify that the drug provides the expected clinical benefit.
Alzheimer’s disease is a devastating illness that can have a profound impact on the lives of people diagnosed with the disease as well as their loved ones. Currently available therapies only treat symptoms of the disease; this treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s. As we have learned from the fight against cancer, the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation.
Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research.
While the specific causes of Alzheimer’s disease are not fully known, it is characterised by changes in the brain—including amyloid plaques and neurofibrillary, or tau, tangles—that result in loss of neurons and their connections. These changes affect a person’s ability to remember and think.
Aduhelm represents a first-of-its-kind treatment approved for Alzheimer’s disease. It is the first new treatment approved for Alzheimer’s since 2003 and is the first therapy that targets the fundamental pathophysiology of the disease.
Researchers evaluated Aduhelm’s efficacy in three separate studies representing a total of 3,482 patients. The studies consisted of double-blind, randomised, placebo-controlled dose-ranging studies in patients with Alzheimer’s disease. Patients receiving the treatment had significant dose-and time-dependent reduction of amyloid beta plaque, while patients in the control arm of the studies had no reduction of amyloid beta plaque.
These results support the accelerated approval of Aduhelm, which is based on the surrogate endpoint of reduction of amyloid beta plaque in the brain—a hallmark of Alzheimer’s disease. Amyloid beta plaque was quantified using positron emission tomography (PET) imaging to estimate the brain levels of amyloid beta plaque in a composite of brain regions expected to be widely affected by Alzheimer’s disease pathology compared to a brain region expected to be spared of such pathology.
The prescribing information for Aduhelm includes a warning for amyloid-related imaging abnormalities (ARIA), which most commonly presents as temporary swelling in areas of the brain that usually resolves over time and does not cause symptoms, though some people may have symptoms such as headache, confusion, dizziness, vision changes, or nausea. Another warning for Aduhelm is for a risk of hypersensitivity reactions, including angioedema and urticaria. The most common side effects of Aduhelm were ARIA, headache, fall, diarrhea, and confusion/delirium/altered mental status/disorientation.
Under the accelerated approval provisions, which provide patients suffering from the disease earlier access to the treatment, the FDA is requiring the company, Biogen, to conduct a new randomised, controlled clinical trial to verify the drug’s clinical benefit. If the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug.
Aduhelm was granted Fast Track designation, which seeks to expedite the development and review of drugs that are intended to treat serious conditions where initial evidence showed the potential to address an unmet medical need.
We await the opinion of the European Medicines Agency and the outcome of any application made to the UK regulatory authorities, to give clarity to people with early Alzheimer’s disease in the UK. Whatever the outcome of their decision, this is just the beginning of the road to new treatments for Alzheimer’s disease. As this drug will only benefit a proportion of people in the early stages of Alzheimer’s disease, there are hundreds of thousands more who may not be eligible, so we must keep searching for drugs for all stages of Alzheimer’s disease and for other types of dementia. We are proud to say that Alzheimer’s Society was part of a pivotal genetic discovery for the first Alzheimer’s gene in the 1990s, which paved the way for anti-amyloid drugs like the one announced by Biogen.
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