Author: Rachael Hansford

Magstim Horizon 3.0 Transcranial Magnetic Stimulation now in UK

Approval by UKCA Empowers UK physicians to advance care of Treatment Resistant Depression, Obsessive Compulsive Disorder, and Anxious Depression with non-invasive brain stimulation

The advanced and FDA-cleared Magstim Horizon 3.0 transcranial magnetic stimulation (TMS) system is now UKCA registered and available for sale in the UK, providing physicians with a proven precision therapy to improve the lives of patients with depression, obsessive compulsive disorder (OCD), and anxious depression1.

The first company to manufacture and commercialise TMS as a treatment for brain disorders, Magstim technology has been cited in more than 16,000 published studies in the neuroscientific and clinical research.

“Magstim was founded in Wales, where our TMS line is manufactured and continues to be advanced,” said Ronnie Stolec-Campo, CEO, Magstim. “The Horizon 3.0 technology is already in use in the US, and we are thrilled and honoured to make this technology available to the dedicated UK physicians working hard to advance the field and alleviate the pain and suffering caused by mental illness.”

From July 10-13, Professor Alex O’Neill Kerr, MBChB, Medical Director of Transforming Mind Solutions, and the Magstim team will be at the Royal College of Psychiatrists Congress in Liverpool to educate clinicians on utilising Horizon 3.0 in their practices. Studies show that patients respond positively to Magstim TMS Therapy2. Horizon 3.0 offers an intuitive system that optimises treatment and helps improve patient care.

“Our focus is developing innovative treatments to aid the cure of depression and anxiety,” states Professor Alex O’Neill Kerr. “Our patients have achieved significant improvements in depression and mental health using TMS therapy. We use Magstim technology and look forward to this Horizon 3.0 launch in the UK.”

To learn more about Magstim research and clinical neurotechnology innovations, visit Magstim.com or call 01994 241111.

About Welcony

Globally, Welcony technologies have supported thousands of research labs, clinics, hospitals and universities that focus on mental health, brain disorders, cognitive neuroscience and neuromonitoring. Key brands include Magstim Magnetic Stimulation, MagstimEGI high-density EEG, Technomed Clinical Neurophysiology and Neurosign Interoperative Nerve Monitoring.

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1USFDA. Horizon 3.0 is indicated for the treatment of Major Depressive Disorder, and decreasing co-morbid anxiety symptoms where present, in adult patients who have failed to achieve satisfactory improvement from prior antidepressant medication in the current episode, as well as an adjunct for the treatment of adult patients suffering from Obsessive-Compulsive Disorder (OCD)

2Carpenter, L et al. “Transcranial Magnetic Stimulation (TMS) for Major Depression: A multi-site, naturalistic, observational study of acute treatment outcomes in clinical practice (2012)” Depress Anxiety 2012: 29, 587-596.

FDA approves LEQEMBI™ (lecanemab-irmb)

Traditional approval for the treatment of Alzheimer’s disease in the US

July 6, 2023: Eisai Co Ltd and Biogen Inc have announced that the US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) supporting the traditional approval of LEQEMBI® (lecanemab-irmb) 100 mg/mL injection for intravenous use, making LEQEMBI the first and only approved treatment shown to reduce the rate of disease progression and to slow cognitive and functional decline in adults with Alzheimer’s disease (AD). LEQEMBI demonstrated clinically meaningful slowing of cognitive and functional decline in a patient group generalisable to US Medicare beneficiaries, which included a mix of racial and ethnic groups, patients with common comorbid conditions, concomitant medications and patients with mild cognitive impairment (MCI) due to AD or mild AD. Treatment with LEQEMBI should be initiated in patients with MCI or mild dementia stage of disease, (collectively referred to as early AD) the population in which treatment was initiated in clinical trials.

LEQEMBI’s traditional approval is based on Phase 3 data from Eisai’s large, global Clarity AD clinical trial, in which LEQEMBI met its primary endpoint and all key secondary endpoints with statistically significant results and confirmed the clinical benefit of LEQEMBI. The primary endpoint was the global cognitive and functional scale, Clinical Dementia Rating Sum of Boxes (CDR-SB). LEQEMBI treatment reduced clinical decline on CDR-SB by 27% at 18 months compared to placebo. Additionally, the secondary endpoint of AD Cooperative Study-Activities of Daily Living Scale for Mild Cognitive Impairment (ADCS MCI-ADL), as measured by people caring for patients with AD, noted a statistically significant benefit of 37%. This measures the ability of patients to function independently, including being able to dress, feed themselves and participate in community activities. Full results of the Clarity AD study were presented at the Clinical Trials on Alzheimer’s Disease (CTAD) 2022 conference and simultaneously published in the peer-reviewed medical journal The New England Journal of Medicine on November 29, 2022.

Importantly, following FDA’s traditional approval of LEQEMBI, CMS confirmed that broader coverage of LEQEMBI is now available and released more details on the registry, including the easy-to-use data submission process. The CMS-facilitated registry is now available for healthcare professionals to submit required patient data to CMS.

“Today, the FDA approved LEQEMBI under the traditional approval pathway, making LEQEMBI the first and only approved anti-amyloid Alzheimer’s disease treatment shown to reduce the rate of disease progression and to slow cognitive impairment in the early and mild dementia stages of the disease. As a research and development-focused company based on our hhc (human health care) concept, we are proud that the results of Eisai’s AD research over the past 40 years have been recognised and delivered to people living with this disease in the United States.

Haruo Naito, Chief Executive Officer at Eisai

“Alzheimer’s disease is a progressive, fatal disease that greatly impacts not only the people living with it, but also their loved ones, care partners and society. We continue to work to create broad and simple access to LEQEMBI for patients and to support diagnosis and treatment at the early stage of the disease. Eisai will diligently work to educate physicians on the safe and appropriate use of LEQEMBI to maximise its benefit to people living with early AD and their families.”

“Today marks a breakthrough in the treatment of Alzheimer’s disease, and we are proud to be at the forefront of ushering in a new era of advances for a disease that was previously considered untreatable. We would like to express our sincere appreciation to those who have worked tirelessly to find a treatment for this unrelenting disease, without whom this progress would not be possible,” said Christopher A. Viehbacher, President and Chief Executive Officer of Biogen. “Our focus is now on the path forward, working alongside Eisai with the goal of making LEQEMBI accessible to eligible patients as soon as possible.”

LEQEMBI is a humanised immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (protofibril*) and insoluble forms of amyloid beta (Aβ). Critically, LEQEMBI targets and clears the most neurotoxic form of Aβ that continuously accumulates as well as removes the existing plaques to treat this progressive, chronic disease. In June 2023, the FDA’s Peripheral and Central Nervous System Drugs (PCNS) advisory committee voted unanimously that the data from Eisai’s Clarity AD clinical trial confirmed the clinical benefit of LEQEMBI for the treatment of AD. Committee members also confirmed the overall risk-benefit of LEQEMBI. On January 6, 2023, LEQEMBI was approved by the FDA under the accelerated approval pathway.

Eisai has developed and deployed Understanding ARIA™, a multi-faceted educational initiative to further advance understanding in the AD healthcare community of the real-world management and monitoring of amyloid-related imaging abnormalities (ARIA). In collaboration with experts in the field of medical imaging as well as major professional societies, Understanding ARIA™ offers resources and programmes that include peer-to-peer education, individual and group educational sessions and subject-matter-expert evaluation of historical case studies.

Eisai is committed to ensuring that appropriate patients have access to LEQEMBI and has established a Patient Assistance Programme to provide LEQEMBI at no cost, for eligible uninsured and underinsured patients, including Medicare beneficiaries, who meet financial need and other programme criteria. Additionally, Eisai offers patient support for improving access through LEQEMBI Patient Navigators, who will provide information about accessing LEQEMBI, help patients and their families understand their insurance coverage and options, and identify financial support programmes for eligible patients. People in the US can learn more about these services by visiting LEQEMBI.com, calling 1-833-4-LEQEMBI (1-833-453-7362), Monday-Friday, 8 a.m. to 8 p.m. Eastern Time or faxing an enrollment form to 1-833-770-7017.

Eisai serves as the lead of LEQEMBI development and regulatory submissions globally with both Eisai and Biogen co-commercialising and co-promoting the product and Eisai having final decision-making authority.

*Protofibrils are large Aβ aggregated soluble species of 75-5000 Kd.2,3.4

Real-world study on AJOVY® reveals sustained long-term effectiveness in migraine

Data presented at the European Academy of Neurology Congress 2023: Teva’s 3rd interim analysis of PEARL real-world study on AJOVY® (fremanezumab) reveals sustained long-term effectiveness in reducing frequency, duration and severity of attacks in patients with chronic and episodic migraine

JULY 2023, TEL AVIV, Israel:  Teva Pharmaceutical Industries Ltd has announced further positive data from the pan-European PEARL study investigating the impact of AJOVY® (fremanezumab) on the prevention of migraine in a real-world setting,[i] due to be completed in 2024.

The data from the 3rd interim analysis[ii] reveals that almost 60% of patients achieved a ≥50% reduction in monthly migraine days from baseline for migraine prevention, with sustained improvement in disability scores and acute medication use observed over 12 months. Treatment persistence rates were high, with 82.3% of patients remaining on treatment by month 12.

Not only was fremanezumab effective in preventing migraine attacks in patients with chronic and episodic migraine, but it has also shown to be effective in reducing the severity and duration of remaining migraine attacks.

Four abstracts from the third interim analysis of the PEARL study will be presented at the 9th European Academy of Neurology (EAN) Congress in Budapest, Hungary. The primary analysis was presented as an oral presentation by Professor Cristina Tassorelli, Department of Brain and Behavioural Sciences, University of Pavia, Pavia, Italy on July 1, 2023.

Commenting on the findings, Professor Tassorelli said: “Preventive treatments are of the utmost importance for reducing the burden of severe migraine, but levels of use of preventive drugs across Europe are still low, resulting in sub-optimal patient care.

These interim findings add to our growing evidence with fremanezumab in the real-world, showing how the burden of migraines can be reduced when an eligible patient has access to monoclonal antibodies like fremanezumab.

Professor Tassorelli

Pinar Kokturk, M.D. Vice President & Head of Medical Affairs Europe at Teva, said: “The 3rd interim analysis of the PEARL study provides valuable insights for clinicians and patients into the effectiveness of fremanezumab (AJOVY®) in a real-world setting. Real-world data allows us to bridge the gap between scientific evidence and the complexities of real-life scenarios, offering a comprehensive understanding of how treatments truly impact patients’ lives. The PEARL study is particularly relevant to clinicians due to its large patient cohort, coming from 11 countries across Europe”.1


[i] Ashina, M. et al, PEARL study protocol. Pain management, 11(6), 647–654. (v0.1) – The two year Pan-  European Real World (PEARL) prospective, observational study of AJOVY® (fremanezumab).

[ii] Ashina, M., et al. Real-world effectiveness & safety of fremanezumab in migraine: 3rd interim analysis of the pan-European PEARL study. Presented at European Academy of Neurology (EAN); 1-4 July 2023; Budapest

EAN-EPR-045

AI in stroke: tool outperforms human emergency call handlers in identifying stroke

AI in stroke: A team of researchers from Denmark have developed a new artificial intelligence (AI) framework to address the number of strokes that go unrecognised by human emergency call handlers.[i] The framework outperformed emergency call handlers in recognising stroke for both sexes and across all age groups studied, indicating its potential as a supplementary tool for early and precise stroke identification in the future.

The retrospective study, presented on 18th May at the European Stroke Organisation Conference (ESOC) 2023, drew from the Danish Stroke Registry and a dataset of over 1.5 million calls made to the Copenhagen Emergency Medical Services between 2015 and 2020, which included over 7000 stroke-related calls. Researchers utilised this data to train an AI framework to firstly transcribe the call audio and then predict the risk of stroke based on the transcribed text.

The results, which were evaluated on calls from 2021, revealed that the AI framework performed more effectively than emergency call handlers in identifying stroke cases. The AI framework achieved a recall (sensitivity) of 63.0% and a precision (positive predictive value) of 24.9%, which resulted in an F1 score of 35.7.* In contrast, emergency call handlers had a recall of 52.7% and precision of 17.1%, resulting in an F1 score of 25.8.

Dr Jonathan Wenstrup, one of the lead authors of the study from Copenhagen University Hospital, commented, “As one of the first points of contact for patients seeking medical assistance, emergency call handlers play a critical role in facilitating early and accurate stroke recognition. Many stroke cases can go undetected at this stage, leading to delays in treatment that can have potentially life-threatening consequences for patients.” 

Across Europe, stroke is the second leading cause of death and a major cause of adult disability, affecting over 1 million people each year.[ii],[iii] As populations continue to grow and age, the number of people living with stroke is projected to increase by 27% between 2017 and 2047 in the European Union (EU).iii Despite this, many strokes can be prevented, and if treated early, the likelihood of a positive outcome can be greatly improved.[iv],[v]

“As with any new tool, further research and development are necessary to improve the framework’s accuracy and expand its capabilities. In the future, it may be possible to train the framework directly from the call audio, bypassing the transcription step, as well as incorporating non-word audio – such as a slurred voice – into the training data. However, given the promising results of this study, it is already clear that technologies like this have the capability to completely transform stroke diagnosis and care.”

With the implementation of this new, cost-effective supporting tool, we can enhance stroke identification by call handlers and ensure more patients receive appropriate and timely care, ultimately improving patient outcomes

Dr Jonathan Wenstrup

*Key terms defined

  1. Recall (sensitivity): A measure of the proportion of actual positive cases that are correctly identified
  2. Precision (positive predictive value): A measure of the proportion of predicted positive cases that are actually positive
  3. F1: A harmonic mean of precision and recall, which combines both metrics into a single score

References

[i] Havtorn, J.D., Wenstrup J., Borgholt L., et al. A retrospective study on deep learning-enabled stroke recognition for a medical help line. Presented at the European Stroke Organisation Conference; 24 May 2023; Munich, Germany.

[ii] OECDiLibrary. Mortality following stroke. Available at: https://www.oecd-ilibrary.org/sites/dbabdd9d-en/index.html?itemId=/content/component/dbabdd9d-en#:~:text=Stroke%20is%20the%20second%20leading,diseases%E2%80%9D%20in%20Chapter%203) (Accessed: May 2023).

[iii] Wafa HA, et al. Burden of stroke in Europe: Thirty-year projections of incidence, prevalence, deaths, and disability-adjusted life years. Stroke. 2020;51(8):2418–2427.

[iv] American Stroke Association. Preventing Another Stroke. Available at: https://www.stroke.org/en/life-after-stroke/preventing-another-stroke (Accessed: May 2023).

[v] American Stroke Association. Why Getting Quick Stroke Treatment is Important. Available at: https://www.stroke.org/en/about-stroke/types-of-stroke/is-getting-quick-stroke-treatment-important (Accessed: May 2023).

Could new materials lead to implantable treatments for epilepsy?

Breakthrough research on materials may help new types of probes be safely implanted in the brain. 

Bioengineering researchers from the University of Glasgow have investigated new dissolvable coatings which could help safely guide flexible implants into brains to help regulate temporal lobe epilepsy.

The development of the material, outlined in an early-view paper in the journal Advanced Nanobiomed Research, is part of a European-funded collaboration which aims to tackle epilepsy by treating and regenerating damaged brain tissue.

The €8m Hybrid Enhanced Regenerative Medicine Systems project – HERMES – was launched in 2019. It brings together 12 partners from seven EU countries to find new ways to heal brain disorders using transplants which combine biological and artificial components.

Neural probes capable of deep brain stimulation have been used to help treat people living with Parkinson’s disease and other conditions like obsessive-compulsive disorder. They are a promising future treatment for temporal lobe epilepsy, which can be resistant to drugs.

Currently, deep brain stimulation probes, which are made from Silicon, often cause scarring around their implantation site because of a mismatch between the stiffness of the artificial materials and the soft tissue of the brain. 

One solution could be a new generation of flexible probes made from new bendable materials which offer a better match with the softness of brain tissue. Flexible implants could also widen the possibilities of where the implants could be placed in the brain, opening up treatments for more conditions.

However, the increased flexibility of the materials can increase the risk of the probes bending or breaking when introduced into brain tissue – a key problem that needs to be solved before the HERMES team and others can use them effectively as implants.

In the paper, the Glasgow team and colleagues in Italy outline how they explored the potential of four different biological materials as coatings for future HERMES implants. The materials act as temporary stiffeners, which could allow flexible probes to reach their target in the brain without bending, before dissolving once the surgery is complete. 

Maria Cerezo-Sanchez, from the James Watt School of Engineering, is the lead author of the article. She said: “The tests we conducted show some really promising results for creating coatings for future flexible neural probes that could help safely guide them to their targets in the brain.

It’s an exciting step forward, and we’re continuing to explore the potential of these materials for use in neural implant procedures.

Maria Cerezo-Sanchez, James Watt School of Engineering

The Glasgow team’s research was conducted in partnership with Prof Giulia Curia’s group at Università degli Studi di Modena e Reggio Emilia, and Dr Gemma Palazzolo at IIT – Istituto Italiano di Tecnologia in Italy.

‘Bioresorbable Insertion Aids for Brain Implantable Flexible Probes: A Comparative Study on Silk Fibroin, Alginate, and Disaccharides’, is published in Advanced Nanobiomed Research and is available at https://onlinelibrary.wiley.com/doi/epdf/10.1002/anbr.202200117

The research was supported by funding from the EU’s Horizon 2020 programme, the University of Glasgow, the Engineering and Physical Sciences Research Council, and the European Commission.

The HERMES consortium consists of: IIT – Istituto Italiano di Tecnologia (Italy), Università degli Studi di Modena e Reggio Emilia (Italy), Università degli Studi di Verona (Italy), Agencia Estatal Consejo Superior de Investigaciones Cientificas (Spain), Politecnico di Milano (Italy), Aarhus Universitet (Denmark), University of Glasgow (United Kingdom), Tampere University (Finland), Fundacion Instituto de Estudios de Ciencias de la Salud de Castilla y Leon e Universidad de Salamanca (Spain), Eurokleis S.r.l. (Italy), Radbound Universiteit (The Netherlands), Den Institute (Belgium).

Opicapone 50mg in adult levodopa treated PD patients without motor complications

BIAL announces positive results from phase 3 study

June 5th, 2023: BIAL announces positive topline results of its phase 3 EPSILON (Early ParkinSon wIth L-DOPA/DDCI and OpicapoNe) study evaluating opicapone 50mg in adult levodopa treated Parkinson’s Disease (PD) patients without motor complications.

In the EPSILON study, opicapone 50 mg was found to be statistically significantly superior versus placebo in its primary endpoint (adjusted mean change from baseline to week 24 in the MDS-Unified Parkinson’s Disease Rating Scale – MDS-UPDRS – Part III score). Opicapone 50 mg did not increase motor complications score in PD patients compared to placebo (as assessed by the MDS-UPDRS Part IV, secondary endpoint).

The data from this phase 3 study support our view that opicapone 50 mg once-daily used early could also provide clinical benefits to levodopa treated PD patients without motor complications. These new data demonstrate the potential for using opicapone in a new range of patients and are valuable additional information for clinicians considering opicapone use in routine clinical practice.

Joaquim Ferreira, Professor of Neurology and Clinical Pharmacology at Faculty of Medicine, University of Lisbon

António Portela, BIAL’s Chief Executive Officer, said: “We sincerely thank the people living with PD and all the investigators for their participation in this study. We are very pleased with the successful outcome of our Phase 3 EPSILON study. These results represent a step forward towards our ambition to bring additional treatment options to patients with PD, moving the adjunctive treatment with opicapone from motor fluctuations to early levodopa treated patients.”

Opicapone, a once-daily Catechol-O-methyltransferase (COMT) inhibitor launched in 2016 and present in more than 20 countries, demonstrated efficacy in reducing OFF-time in adult patients with Parkinson’s disease and end-of-dose motor fluctuations.1,2

BIAL plans to report EPSILON’s results in scientific conferences and publications after the summer. The company will be discussing the data with relevant health authorities to determine the next steps so that people living with PD can benefit from this innovation.

References

  1. Ferreira J. et al., Lancet Neurol. 2016;15(2):154–65
  2. Lees AJ. et al., JAMA Neurol. 2017;74(2):197–206
  3. Ferreira J. et al., Neurol Ther. 2022 Sep;11(3):1409–1425


About EPSILON study:

EPSILON is a 24-week, phase III, double-blind, randomised, placebo-controlled and parallel-group study, designed to evaluate the efficacy and safety of once-daily opicapone 50 mg as add-on to levodopa/DDC inhibitor therapy in 350 patients at an early stage of PD when they do not exhibit signs of motor complications (ClinicalTrials.gov Identifier: NCT04978597).

Patients (aged 30−80 years) with idiopathic PD, treated with 3 to 4 daily oral doses of up to 500 mg levodopa, with signs of treatable motor disability but no motor complications were randomised in a 1:1 ratio to receive opicapone 50mg once-daily or placebo during a 24-week double-blind evaluation period.3

About Ongentys (opicapone):

Opicapone is a once-daily COMT inhibitor. It was evaluated in two pivotal trials in patients with PD and end-of-dose motor fluctuations (BIPARK-I and II), and the results demonstrated an acceptable tolerability profile, combined with efficacy in reducing OFF-time.1,2 These studies led to the approval of opicapone in the European Union, USA, Japan, Australia, and other countries.3

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.*

ONGENTYS® (opicapone) is not authorised for use in patients without motor fluctuations.

About BIAL:

BIAL is an innovation-driven pharmaceutical company aiming to improve people’s lives worldwide. With 99 years of experience, BIAL is strongly committed to therapeutic innovation, consistently investing over 20% of its annual turnover in R&D, being neurosciences its major area of research.

Based on its own innovative medicines and with a consistent partnering program, BIAL has extended its presence worldwide. The company has affiliates in three different continents – Europe, America and Africa – and its products are present in fifty countries, including the US, Japan, Germany, Canada, Korea and Australia, fulfilling its purpose of making a real difference in the lives of people living with severe diseases across the world.

For more information about BIAL, please visit http://www.bial.com

Janssen is a proud sponsor of the ABN-INA joint meeting

Janssen is proud to have sponsored the recent ABN-INA joint meeting in Belfast. We recognise the educational impact of events like these, and are grateful to the delegates who attended our lunchtime symposium on Wednesday 10th on the topic of ‘How can brain volume measures inform clinical practice? What have we learned from multiple sclerosis?’

Brain volume loss (BVL) has been shown to correlate well with disease progression, and is increasingly used as an efficacy outcome measure on the group level in clinical trials of disease modifying treatments (DMTs). Despite this, BVL is rarely used to monitor individual patients in the clinical setting due to methodological, statistical and resource challenges. In addition, there remains to be a widespread understanding among the clinical and scientific community of what pathological processes brain volume loss actually shows.

In our symposium we explored this measure further and considered how we can bridge the gap between clinical trial results and management of patients in the clinical setting. In addition, we presented recent Janssen data on not only BVL, but also other markers of disease progression, including neurofilament light chain and myelin preservation.

CP-393915               June 2023

Seer partners with NHS hospitals

Seer partners with NHS hospitals to deliver cloud-based video-EEG-ECG monitoring services that enhance the efficiency of neurology and neurophysiology teams. Seer supports the integration of home-based diagnostics into current clinical workflows through the Seer Home service, a range of home video telemetry offerings. Hospitals can access customised services — including patient set-up, home-based monitoring, data review, and reporting — to meet their individual needs. The Seer Home service is designed to reduce time to diagnosis and treatment for people experiencing seizures and seizure disorders by helping the NHS to keep wait lists down, overcome staffing challenges, and free up hospital beds.

Seer UK | Home Video EEG Monitoring with ECG (seermedical.com)

Grant to explore new epilepsy treatment for Tuberous Sclerosis Complex (TSC)

  • Dr Felix Chan awarded £174k grant to study Tuberous Sclerosis Complex (TSC) and epilepsy by Epilepsy Research UK
  • The new study will investigate lysine metabolism as a therapeutic target for epilepsy in TSC
  • Research designed with TSC and epilepsy patient community through Tuberous Sclerosis Association and Epilepsy Research UK

Dr Felix Chan awarded £174k grant to study Tuberous Sclerosis Complex (TSC) and epilepsy by Epilepsy Research UK. The new study will investigate lysine metabolism as a therapeutic target for epilepsy in TSC Research designed with TSC and epilepsy patient community through Tuberous Sclerosis Association and Epilepsy Research UK.

A researcher based in Aston Institute for Health and Neurodevelopment has been awarded an Endeavour Project Grant of £174,495 by Epilepsy Research UK.  The grant will fund a 36-month study entitled ‘Lysine Metabolism as a therapeutic target for epilepsy in tuberous sclerosis complex (TSC)’. The study will focus on Tuberous Sclerosis Complex (TSC), a rare genetic disease that causes non-cancerous tumours to grow in the brain and other areas of the body. TSC is often accompanied by epilepsy, which affects around 80% of people with the condition. Importantly, epilepsy in TSC is the highest cause of death and remains an unmet clinical need for TSC patients as it is hard to treat with currently available medications.

Dr Felix Chan’s research will investigate the potential of lysine metabolism as a therapeutic target for epilepsy in TSC. This involves understanding how the body processes the amino acid lysine and how it can be manipulated to reduce seizures in people with TSC.  A therapeutic target is a biological molecule, biological pathway or physiological response that is associated with a particular disease process and may be inhibited or activated by a therapy in a way that will change the course of the disease in a positive way.

The study will also involve a PhD studentship to work with Dr Chan, Professor Gavin Woodhall and collaborator Professor Wyatt Yue from Newcastle University, with recruitment for intake expected in October 2023.

Dr Chan’s research project is notable for its collaborative approach. The TSC and epilepsy patient community has been involved in the study design process through collaboration with the Tuberous Sclerosis Association TSC Research Volunteer Network and the Epilepsy Research UK Shape Epilepsy Research Network.

I am delighted to have been awarded this Endeavour Project Grant from Epilepsy Research UK. TSC is a rare genetic disease that affects around one in 6,000 people and can have a profound impact on individuals and their families. My hope is that this research will contribute to the development of new treatments for epilepsy in TSC, improving the lives of those affected by this condition.” Dr Pooja Takhar, joint chief executive of the Tuberous Sclerosis Association, added: “We are pleased to see that the most important voice in TSC – individuals and families living with the condition itself – has been included in the design process of this vital research project. This ensures that the research is focused on the areas that are of most importance to those living with TSC, boosting the potential impact on the lives of people with TSC.”

Dr Felix Chan, lecturer in neuroscience, at Aston University