Author: Rachael Hansford

Can trichloroethylene (TCE) cause Parkinson’s disease (PD)?

In a paper published online on March 14th 2023 in the Journal of Parkinson’s Disease, researchers suggest that a common chemical, TCE, may be a key reason for the dramatic increase in Parkinson’s disease (PD).

Lead investigator Ray Dorsey, MD, professor of neurology, University of Rochester, US has called PD “the world’s fastest-growing brain disease,” and said it “may be largely preventable”.[1] TCE has been used in industry, commerce, military, and medicine, including producing refrigerants, cleaning electronics, and degreasing engine parts.

It has also been used in dry cleaning, although a similar chemical (perchloroethylene [PCE]) is more commonly used. However, the authors note that in anaerobic conditions, perchloroethylene often transforms into TCE “and their toxicity may be similar.” TCE is also found in consumer products such as paint removers and aerosol cleaning products and it was used to decaffeinate coffee until the 1970s.

TCE affects those who work with it, but it also pollutes outdoor air, taints groundwater, and contaminates indoor air. It is present in a substantial amount of US groundwater and it “evaporates from underlying soil and groundwater and enters homes, workplaces, or schools, often undetected,” the researchers note.


[1] What’s Driving the ‘World’s Fastest-Growing’ Brain Disease’? (

Phase 2 Trial of NLY01 in Parkinson’s Disease: Topline results

 – This randomised, double-blind, placebo-controlled trial was designed to assess the safety, tolerability, and efficacy of NLY01 in subjects with early, untreated PD.

– The primary endpoint, change from baseline in the sum of MDS-UPDRS Parts II and III after 36 weeks of NLY01 treatment, compared with placebo, did not reach statistical significance.

– Age was identified as a significant factor in response. Statistically significant and dose-related improvements in UPDRS Parts II + III were observed in patients under the age of 60.

– NLY01 was safe and well tolerated.

Neuraly, a clinical-stage biotechnology company developing disease-modifying agents for neurodegenerative disorders, announced on March 27th topline results from the Phase 2 trial with NLY01 in patients with early, untreated Parkinson’s disease (PD). The primary endpoint, change from baseline to Week 36 in the sum of MDS-UPDRS Parts II (Motor Experiences of Daily Living) and III (Motor Examination) after 36 weeks of NLY01 treatment, compared to placebo, did not reach statistical significance. NLY01 was safe and well tolerated.

Post hoc analysis of efficacy measures by age groups suggests a treatment effect in younger patients (<60 years). This group accounted for 37 percent of the study population (N=95). In patients <60 years of age, a clinically significant reduction in the sum of UPDRS Parts II and III was observed over the 36 weeks of treatment in subjects treated with NLY01 (an approximate 5-point reduction at 36 weeks, P<0.01 vs. placebo). The effect was statistically significant, dose-related, and appeared persistent over eight weeks after treatment was discontinued. These results suggest slower disease progression in NLY01-treated subjects compared to placebo-treated subjects in this age group. Detailed data will be presented at future medical meetings.

The Phase 2 study enrolled 255 patients across 55 clinical sites in the United States and Canada. The randomized, double-blind, placebo-controlled trial is designed to assess the safety, tolerability, and efficacy of NLY01 in subjects with early PD. Enrolled patients were randomized to receive either weekly injections of NLY01 (2.5 mg or 5.0 mg) or placebo for 36 weeks. Clinical assessments were made periodically during treatment and again eight weeks after treatment was discontinued. More information about the ongoing Phase 2 study of NLY01 in PD is available at under the identifier NCT04154072.

Although the study didn’t meet its primary endpoint, it is intriguing that NLY01 demonstrated a beneficial effect in patients under 60. This potentially may represent interest for further clinical evaluation of NLY01 in younger Parkinson’s patients.

Viktor Roschke, Head of R&D at Neuraly

Seulki Lee, CEO of Neuraly, also mentioned that the company will thoroughly analyse the data from the study to better understand the outcomes and determine the next steps for NLY01.

About NLY01

NLY01 is a proprietary long-acting analogue of exendin-4, a glucagon-like peptide-1 receptor (GLP-1R) agonist that slows progression in animal models of Parkinson’s and Alzheimer’s disease. In clinically relevant animal models, NLY01 prevented neuronal cell death by inhibiting microglial activation and forming neurotoxic reactive astroglial cells. Treatment with NLY01 slowed disease progression, improved motor and cognitive functions, and extended the lifespan in mice with Parkinson’s disease. NLY01 penetrates the blood-brain barrier (BBB) in animal models, and its receptor (GLP-1R) is highly expressed on glial cells. NLY01 is being developed as a potentially disease-modifying agent for the treatment of neurodegenerative disorders, including Parkinson’s and Alzheimer’s.

About Neuraly

Neuraly is a clinical-stage company whose mission is to translate scientific discoveries in neurology into revolutionary new drugs that can radically improve and prolong the lives of people suffering from the devastating consequences of diseases such as Parkinson’s disease (PD), Alzheimer’s disease (AD) and other neurodegenerative disorders. The company is leveraging a deep understanding of the role of glia biology in neuroinflammation and neuroprotection in advancing a risk-diversified product portfolio for PD and AD. Neuraly is a subsidiary of D&D Pharmatech, a global biotech company that funds the development of revolutionary medicines through disease-specific subsidiary companies founded and guided by top-tier medical research faculty and biotechnology veterans, please visit D&D Pharmatech (

Vestibular Rehabilitation in Concussion

Clinical Education in concussion at Southampton Football Club

Emma Jewer, Concussion Service Lead at Hobbs Rehabilitation, recently delivered 2 workshops with practical and theory sessions to the medical teams from the youth, adult and women’s teams. 

This has led to requests for more practical training on baseline testing and embedding this into their existing rehab and exercise protocols.

Hobbs Rehabilitation are currently working on further clinical education in response to the demand for specialist services for those with persistent concussion symptoms or balance and vestibular issues across the wider British elite sports sector. 


Effects of median nerve stimulation on Tourette syndrome

Results of UK-wide double-blind sham-controlled clinical trial of the Neupulse device for suppressing tics in Tourette syndrome

The trial was run by the University of Nottingham, conducted between 18th March 2022 and 5th of March 2023, sponsored by Nottingham University Hospitals NHS Trust. The aim was to evaluate the effectiveness of the Neupulse device in reducing the severity and frequency of tics in individuals with Tourette Syndrome. A total of 121 participants took part in the study and went on to receive either active or sham stimulation for 10 minutes a day for 4 weeks.

tourette syndrome device neupulse

The results of the study revealed that people who received active stimulation experienced a significant reduction in the severity and frequency of their tics. On average, they saw a reduction in tic frequency of more than 25% while they received stimulation.

After using the device for 4 weeks, people who received active stimulation experienced a reduction in their tic severity of more than 35%. In total, 59% of the people who received active stimulation experienced a reduction in tic severity of at least 25% compared to baseline.

These positive results will help start the development of a commercial medical device, that will run for the next 18 months. Neupulse hope to obtain regulatory approval for a commercial device by 2025 and have a device available by 2026.

Primary Progressive MS confirmed to be distinct from other MS subtypes

Long suspected by the research community, new study from Tisch MS Research Center of New York, published in Brain, finally establishes that PPMS is pathophysiologically separate from other forms of MS

Findings have groundbreaking implications for targeted treatment of PPMS, a disease characterised by worsening neurological function and progressive disability in patients

The Tisch MS Research Center of New York has published new research classifying primary progressive multiple sclerosis (PPMS) as pathophysiologically distinct from other forms of MS. Published in the journal Brainthe study finally confirms a conclusion long suspected by the scientific community: PPMS is an antibody-mediated disease with unique clinical and pathological features.

Tisch MSRCNY researchers developed the first ever animal model of PPMS via intrathecal injection of cerebrospinal fluid (CSF) from PPMS patients into the cervical spinal cord. Unlike the CSF from patients with other forms of MS (relapsing-remitting MS (RRMS) and secondary progressive MS), only PPMS patient CSF induced motor disability and hallmark MS-like pathology in the spinal cord including: demyelination, impaired remyelination, reactive astrogliosis, and axonal damage.

Furthermore, removal of immunoglobulin G (antibodies) from PPMS CSF via filtration or immunodepletion weakened its pathogenic capacity, while injection of recombinant antibodies derived from PPMS CSF mimics this pathology – suggesting that the disease is not only pathophysiologically distinct, but also antibody-mediated. These findings could lead to the development of more effective treatments for PPMS – a disease that has traditionally lagged behind RRMS, the more common disease subtype, in terms of research and treatments developed.

“Primary progressive MS is arguably one of the most severe and debilitating disease forms, but for many years, we have not had an effective way to research and ultimately treat it,” said Dr. Saud A. Sadiq, Director and Chief Research Scientist at Tisch MSRCNY. “By developing an animal model specifically designed for PPMS, our team has been able to at long last confirm a widely-suspected hypothesis: that PPMS is in fact its own disease. This finding not only gives us the ability to conduct more precise research on PPMS and develop more targeted treatments – but it also represents another important step toward our center’s overarching goal to find the cause of and the cure for MS.”

For thousands of PPMS patients and their families, who have long been without effective treatment options, this research is potentially game-changing. We look forward to using our novel model to learn more about how pathogenic antibodies trigger pathology in PPMS and to develop potential therapeutic strategies specific for PPMS.

Dr. Jamie K. Wong, principal investigator at Tisch MSRCNY and lead author of the study.

About the Tisch MS Research Center of New York

The mission of the Tisch Multiple Sclerosis Research Center of New York is to conduct groundbreaking medical research to ensure unparalleled care and positive outcomes for MS patients. Its integrated relationship with the International MS Management Practice (IMSMP) accelerates the pace at which research discoveries translate from lab bench to bedside. The Center aims to identify the cause of MS, understand disease mechanisms, optimise therapies, and repair the damage caused by MS while offering patients access to the best and most advanced treatments possible.

‘Microbot’ development could help treat seizures

University of Glasgow researchers are part of a new project which is setting out to develop tiny injectable robots capable of predicting and mitigating epileptic seizures.

The project, called CROSSBRAIN, is led by Tor Vergata University of Rome in Italy and is funded by the European Innovation Council. 

Over the course of the next four years, the CROSSBRAIN collaborators will develop implantable ‘microbots’, about a tenth of a millimetre in size, made from advanced nanomaterials with specially-tailored physical properties. 

Once implanted in the brain, they will be controlled by a small, wearable central control unit capable of monitoring electrical activity to detect the onset of a seizure and modulate its effect through targeted neurostimulation. 

The microbots will be able to deliver genetic material on command, enabling cell- and microcircuit-level neuromodulation in rodent brains during the later stages of the project’s development.

Professor Hadi Heidari, of the University of Glasgow’s James Watt School of Engineering, is leading the UK contribution to CROSSBRAIN. Professor Heidari’s Microelectronics Lab conducts pioneering research on integrated micro and nanoelectronics design for medical and industrial applications. In this project, the Microelectronics Lab will help to design and develop the microbots’ wireless power and data management and delivery systems. 

The CROSSBRAIN team will develop a cutting-edge FBAR magnetoelectric antenna at the world-class cleanroom facilities of the University’s James Watt Nanofabrication Centre. 

Professor Heidari said: “We’re pleased to be part of this ambitious project, which has the potential to pave the way for transformative treatments for pathological brain conditions like epilepsy.

CROSSBRAIN brings together leading researchers from across Europe, with a wide range of expertise in bioengineering, artificial intelligence, nanomaterial design and fabrication, and medical physics. I’m looking forward to collaborating with my colleagues to develop this exciting technology in the years to come.

Professor Heidari

The CROSSBRAIN team are Dr Rupam Das, of the University of Exeter, Dr Finlay Walton, Mahdieh Shojaei Baghini, Jungang Zhang and Laura Mazon Maldonado. Professor Muhammad Imran, director of the University of Glasgow’s Communications, Sensing and Imaging Hub, is also lending his support on wireless power and data transmission. 

Professor Nicola Toschi, of the Department of Biomedicine and Prevention at the Tor Vergata University of Rome, is the project’s principal investigator. 

He added: “Within brain tissue, neurons communicate through a complex interplay of signaling mechanisms, including chemical, thermal, and electrical (depolarisation/repolarisation) changes. It is widely known that many pathological brain conditions directly involve aberrant electrical activity of the brain, such as, epileptic seizures or panic disorders.

“In such conditions, timely recognition and prompt intervention are essential to begin effective periodic and adaptive treatment. However, the technologies available to guide and modulate brain activity in a precise and selective way for therapeutic purposes are severely limited to date, considerably reducing the therapeutic options.

“However, recent advances in nanotechnology could facilitate access to new modalities and innovative paradigms in the field of neuromodulation. Innovation in the field of nanomaterials provides the opportunity to modulate neuronal activity with greater precision and sensitivity. The CROSSBRAIN project aims to create radically new neurostimulation strategies and devices in the field of precision medicine with a key role in the predictive management of brain diseases.”

The other partners in the CROSSBRAIN project are SISSA International School of Advanced Studies of Trieste, Italy; PERC PERCUROS BV, Netherlands; NLB NAMLAB GMBH, Dresden, Germany; FAU Friedrich Alexander Universitaet, Erlangen-Nuernberg, Germany; CIC Associacion Centro de Investigacion Cooperativa en Biomateriales, San Sebastian, Spain; IIT Italian Institute of Technology Foundation, Genoa, Italy and the CSIC Agencia estatal consejo superior de investigaciones cientificas Madrid, Spain.

CROSSBRAIN is funded by the EIC Pathfinder Challenge Grant, funded by the European Innovation Council (European Commission) under the Horizon Europe program. EIC is Europe’s flagship innovation program that identifies, develops, and scales up breakthrough technologies and innovations throughout their lifecycle, from the early research stage, proof of concept, technology transfer and funding and development of start-ups and SMEs.

NICE publishes reimbursement recommendation for Translarna™

On 22nd February 2023, PTC Therapeutics, Ltd. announced the National Institute for Health and Care Excellence (NICE) has published its Final Guidance recommending Translarna™ (ataluren) for reimbursement and use across the National Health Service (NHS) in England and Wales. Translarna is the only approved treatment for patients with nonsense mutation Duchenne muscular dystrophy aged 2 years and older who can walk.

The final Guidance allows patients to continue treatment on ataluren and does not include a formal stopping rule, which is a criterion used to identify when someone should stop receiving a treatment. NICE concluded that the decision to stop treatment should be taken after individual discussions between patients, caregivers, and clinicians.

“The published final NICE Evaluation and agreement with the NHS provide critical access to Translarna for newly diagnosed and existing patients with nonsense mutation Duchenne in England and Wales,” said Jesse Sibarium, Senior Vice President and General Manager – EMEA, PTC Therapeutics. “The decision to not include a formal stopping rule in the Evaluation is positive. It recognises points raised by Action Duchenne, MDUK and other patient and clinical experts, in evidence to NICE, that clinicians would want the option to continue using Translarna after their patients lose the ability to walk because of the potential ongoing therapeutic benefits seen.”

Duchenne is a severe progressive disease that leads to rapidly worsening muscle function with children often using a wheelchair by early adolescence and eventually requiring artificial ventilation to breathe.1,2 The NICE recommendation was based on data from clinical trials and real-world evidence demonstrating Translarna’s potential to slow disease progression and improve patient outcomes.1-3

The decision to not include a formal stopping rule in the Evaluation is positive. It recognises points raised by Action Duchenne, MDUK and other patient and clinical experts, in evidence to NICE, that clinicians would want the option to continue using Translarna after their patients lose the ability to walk because of the potential ongoing therapeutic benefits seen.

Jesse Sibarium, Senior Vice President and General Manager – EMEA, PTC Therapeutics


1 Birnkrant DJ et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018; 17:251–67.

2 Duchenne Muscular Dystrophy. Muscular Dystrophy Association. Available at: Last Accessed October 2021.

3 National Institute for Health and Care Excellence Highly Specialised Technologies Guidance HST22 accessed 22nd February 2023   

About Translarna™ (ataluren)

Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the trade name of ataluren, is licensed in multiple countries including Great Britain, Northern Ireland and the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States.

About Duchenne Muscular Dystrophy (Duchenne)
Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20’s due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s.

FINTEPLA®▼ (fenfluramine) oral solution approved in the EU for adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS)

UCB’s FINTEPLA®▼ (fenfluramine) oral solution has been approved in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) as an add-on therapy to other anti-epileptic medicines for patients two years of age and older.1

The approval by the European Commission (EC) was based on safety and efficacy data from a global, randomised, placebo-controlled Phase 3 clinical trial, in 263 patients with LGS (aged 2-35 years), that demonstrated adjunctive fenfluramine at a dose of 0.7/mg/kg/day provided a significantly greater reduction in the frequency of drop seizures (p=0.001) compared to placebo. The most common treatment-emergent adverse events were decreased appetite, somnolence, fatigue, and pyrexia (fever). No cases of valvular heart disease or pulmonary arterial hypertension were observed.2

Professor Rima Nabbout, MD, PhD, Professor of Paediatric Neurology at University Paris cité, APHP, Necker Enfants Malades, Institut Imagine, Paris, France, said: “LGS is a developmental and epileptic encephalopathy where seizures are frequent, inducing high level of trauma injuries and negatively impacting development and quality of life. Seizures are often resistant to currently available medications, making this approval especially important for the individuals affected and their families.”

Mike Davis, Head of Global Epilepsy & Rare Syndromes, UCB, said: “With this approval, fenfluramine is now an important additional treatment option for those impacted by this difficult to treat condition in Europe. This approval underscores our commitment to improving treatment outcomes, while addressing the high unmet need for new treatments for people living with LGS and rare epilepsies.”

LGS is a severe childhood-onset developmental and epileptic encephalopathy (DEE) characterised by multiple types of drug-resistant seizures with high morbidity, as well as serious impairment of neurodevelopmental, cognitive, and motor functions,3,4 affecting an estimated 2 in 10,000 people in European Union (EU).5 Seizures leading to falls (“drop attacks/seizures”) are common in LGS and tonic seizures are a hallmark feature of this syndrome.3,4 In addition, convulsive seizures (e.g., generalised tonic-clonic [GTC] seizures) are also commonly observed and usually occur in later stages of LGS, but sometimes may precede core seizure types. In addition to being associated with bodily injury and hospitalizations, GTC seizures are a primary risk factor of sudden unexpected death in epilepsy (SUDEP). Patients with GTC seizures have an approximately 10-fold greater risk for SUDEP than patients with other seizure types.2

Additionally, the EC has also adopted the EMA Committee for Orphan Medicinal Products (COMP) recommendation that the orphan designation for fenfluramine be maintained.6

LGS is a developmental and epileptic encephalopathy where seizures are frequent, inducing high level of trauma injuries and negatively impacting development and quality of life. Seizures are often resistant to currently available medications, making this approval especially important for the individuals affected and their families.

Professor Rima Nabbout, MD, PhD, Professor of Paediatric Neurology at University Paris cité, APHP, Necker Enfants Malades, Institut Imagine, Paris, France

About fenfluramine C-IV in EU1

Fintepla is indicated for the treatment of seizures associated with Lennox-Gastaut and Dravet syndrome as an add-on therapy to other anti-epileptic medicines for patients 2 years of age and older. Fenfluramine is a serotonin releasing agent, and thereby stimulates multiple 5-HT receptor sub-types through the release of serotonin. Fenfluramine may reduce seizures by acting as an agonist at specific serotonin receptors in the brain, including the 5-HT1D, 5-HT2A, and 5-HT2C receptors, and also by acting on the sigma-1 receptor. The precise mode of action of fenfluramine in Dravet syndrome and Lennox-Gastaut syndrome is not known.

Fenfluramine oral solution is available under a controlled access programme to ensure regular cardiac monitoring and to mitigate potential off-label use.

Please refer to Fintepla, INN-fenfluramine ( (SmPC) before prescribing. 

This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.


  1. Fintepla EMA PI. Accessed February 2023.
  2. Knupp K, Scheffer I, Ceulemans B, et al. Efficacy and safety of fenfluramine for the treatment of seizures associated with Lennox-Gastaut syndrome. A Randomized Clinical Trial. JAMA Neurol. 2022;79(6):554-564.
  3. Strzelczyk A, Schubert-Bast S. Expanding the Treatment Landscape for Lennox-Gastaut Syndrome: Current and Future Strategies. CNS Drugs. 2021;35(1):61-83.
  4. Specchio, N, Wirrell, EC, Scheffer, IE, Nabbout, R, Riney, K, Samia, P, et al. International League Against Epilepsy classification and definition of epilepsy syndromes with onset in childhood: Position paper by the ILAE Task Force on Nosology and Definitions. Epilepsia. 2022;63:1398-1442.
  5. EU/3/17/1836: Orphan designation for the treatment of Lennox-Gastaut syndrome Accessed February 2023.
  6. EMA/COMP/946245/2022: Committee for Orphan Medicinal Products (COMP). Minutes for the meeting on 06-08 December 2022. Accessed February 2023.

NICE approves eptinezumab (Vyepti) to treat episodic and chronic migraine

On 25th January 2023, the National Institute for Health and Care Excellence (NICE) announced that eptinezumab (Vyepti) has been approved for use within the NHS in England for the treatment of episodic migraine.

Eptinezumab is given by infusion once every 12 weeks. It is one of a number of calcitonin gene-related peptide (CGRP) antibody drugs, which are the first medication specifically designed to prevent migraine.

The drug should be available for patients who experience migraine on four or more days a month and who have already failed to respond to at least three other migraine preventive drugs.

Integrated Care Systems (ICSs) in England now have three months to comply with this recommendation.