Author: Rachael Hansford

WFNR Franz Gerstenbrand Award 2022

Posted on September 5, 2022 by - Awards & Appointments, Rehabilitation

This year’s WFNR Franz Gerstenbrand Award closes for entries on 31st October, with £3,000 for the winner.   The Award is open to  clinicians, researchers or allied health professionals worldwide currently working in neurorehabilitation.  

Entries are open to WFNR members and non-members and are particularly encouraged from those individuals under the age of 35 years. 

The WFNR Award is named after Professor Franz Gerstenbrand in recognition of his contribution to neurorehabilitation, and recognises and rewards a neurorehabilitation project that has benefited patients. 

The entries for the WFNR Award must demonstrate a difference to patient outcomes and can involve any aspect of neurorehabilitation, such as a patient or clinic management initiative, research project, best practice development or the use of a new technological development.  The work described must be completed and have already produced results or been published in the last 12 months.

The Award is for a travel bursary to a clinical/scientific conference, professional development course or research project. For further information and an application form please visit: https://www.wfnr.co.uk/awards/awards

The winner will be announced at the 12th World Congress for Neurorehabilitation which takes place from 14-17 December 2022 at the Messe Wien Exhibition and Congress Centre, Vienna, Austria.   

New method of examining the brain’s electrical signals

Posted on August 26, 2022 by - Epilepsy

Method could hold the key to better treatment of epilepsy and schizophrenia

  • Researchers are exploring new ways to ‘listen’ to and record electrical signals emitted from brain cells
  • Findings could be used to help treat conditions like epilepsy and schizophrenia
  • Project will use newly developed nanomaterials to keep removed samples of brain healthy for longer to allow more understanding of what generates epileptic seizures

Researchers at Aston University are exploring new ways to ‘listen’ to and record electrical signals emitted from brain cells, which could be used to help treat the conditions.

Dr Petro Lutsyk, lecturer in electronic engineering and systems in the College of Engineering and Physical Sciences and member of Aston Institute of Photonic Technologies (AIPT), together with Dr Stuart Greenhill, senior lecturer in neuroscience in the College of Health and Life Sciences and member of Aston Institute of Health and Neurodevelopment (IHN), have been awarded £100,000 by the Royal Society to conduct the project Nanomaterial Webs for Revolutionary Brain Recording.

Currently, epilepsy patients who can’t be helped by drugs may undergo brain surgery in order to prevent seizures, removing the part of the brain that is the ‘focus’ of the seizures.

The research project will use newly developed nanomaterials to keep samples of brain healthy and active for far longer than current technology allows, whilst recording the activity of the tissue. This allows more understanding of what generates epileptic seizures and opens up new avenues for drug development, meaning fewer surgeries may be needed in the future. Eventually, the technology may lead to new and better ways of recording from patients’ brains before surgery.

Dr Stuart Greenhill

The two-year project will see materials and electronic engineering applied to translational neuroscience research.

The grant is from the Royal Society APEX Awards scheme (Academies Partnership in Supporting Excellence in Cross-disciplinary research award) which offers researchers with a strong track record in their area an opportunity to pursue interdisciplinary research to benefit wider society.

Imaging yields new insights into stroke

Posted on August 4, 2022 by - Stroke

Synchrotron’s “superhuman vision” made it easy to detect markers of brain damage

Haemorrhagic stroke, where a weakened vessel in the brain ruptures, can lead to permanent disability or death. Across the globe, over 15 million people are coping with its effects.

A study by researchers from the University of Saskatchewan (USask) and Curtin University in Australia has moved one step closer to identifying when the bleeding associated with a haemorrhagic stroke starts – critical information for improving patient outcomes.

Time is of the essence when it comes to stroke; the sooner doctors can start treatment, the better the odds they can limit damage.

Using the Mid-IR beamline at the Canadian Light Source at USask, the team examined brain tissue samples with a special technique called Fourier-transform infrared imaging. The researchers were led by Dr. Lissa Peeling, a neurosurgeon at the Royal University Hospital and an Associate Professor in the Department of Surgery at USask.

The novel approach enabled the researchers to identify changes in the brain specific to haemorrhagic stroke.

Mid-IR Beamline

Dr Jake Pushie, a member of Dr Kelly’s and Dr Peeling’s research team at USask’s College of Medicine, said the combination of the beamline and infrared imaging made it easy to detect markers of brain damage caused by haemorrhagic stroke.

“In a sense, this is giving us ‘superhuman vision’ to look at these brains and map out what’s happening metabolically,” said Pushie.

With synchrotron technology, the team could see where a bleed originated and the extent of oxidative damage it caused – something impossible to do with a microscope or traditional approaches to imaging. Their findings were published in Metallomics.

Armed with this new approach, and a better understanding of what they are looking for, Pushie and colleagues will now go back through their extensive “library” of stroke tissue samples to gain a clearer picture of the speed at which oxidative damage begins to ramp up.

The team’s findings could eventually enable doctors to use clinical imaging – such as MRI or CT scans – to pinpoint where, and how long ago, a haemorrhagic stroke occurred in the brain.

Miranda Messmer is another member of the team who was a USask undergraduate research student with the College of Arts and Science at the time of the study and will be a graduate student with the USask College of Medicine in the fall.

Messmer said knowing when bleeding has started can provide clinicians with a clearer picture of the time window they have to act. “Being able to understand what is going on biologically, when we see any kinds of changes in the clinical images, could help doctors provide better care when it comes to minimising the tissue damage associated with stroke,” said Messmer.

Related video: This story is about the team’s most recent published findings on stroke. This video from 2021 features the team members describing how they were examining risk factors for bleeding in the brain after stroke. 

International Encephalitis Research Seed Funding 2022

Posted on August 1, 2022 by - Uncategorised

Deadline: 9th September 2022

The Encephalitis Society are inviting researchers to apply for up to £10,000 in seed funding for a project specific on encephalitis with a duration of up to 18 months.

The fund is open to applicants from low-to-middle income countries worldwide and to all levels from medical students, junior doctors and early researchers, those returning to research to post-doctoral research or equivalent professional experience.

For more information on eligibility and conditions of the grant and to apply please visit www.encephalitis.info/seedfund or email research@encephalitis.info

seed

First ever Marketing Authorisation Application (MAA) for biosimilar natalizumab

Posted on July 20, 2022 by - Uncategorised

Polpharma Biologics announced on July 15th 2022 that the European Medicines Agency (EMA) has accepted the first-ever Marketing Authorisation Application (MAA) for biosimilar natalizumab, a proposed biosimilar to Tysabri®.

In the European Union, Tysabri is approved to treat adults with relapsing-remitting MS (RRMS) who have highly active disease. It is approved for adults with relapsing forms of MS in the US.

The application is for an intravenous (IV) route of administration, with the same dosing regimen, presentation and indication as the reference medicine Tysabri® – a single disease-modifying therapy (DMT) in adults with highly active relapsing-remitting multiple sclerosis (RRMS).

The MAA submission was supported by a robust analytical, preclinical and clinical data package, including evidence from the Phase III Antelope study in RRMS patients (NCT04115488). 

Antelope trial results, presented at this year’s American Academy of Neurology (AAN) annual meeting, broadly showed that the biosimilar was comparable to Tysabri in terms of its efficacy at reducing inflammatory activity on MRI scans. The safety and tolerability profile of the biosimilar also was comparable to that of the name-brand medication.

Biosimilar natalizumab was developed by scientists at Polpharma Biologics and the company retains responsibility for the manufacturing and supply of the medicine. Sandoz, a division of Novartis, has the rights to commercialise and distribute the proposed biosimilar.

The acceptance of the filing of proposed biosimilar natalizumab by the European regulatory authorities means we are a critical step closer to getting this important medicine to the patients who need it the most. We are deeply proud of the dedication of our scientific teams to take biosimilar natalizumab from cell line and technical development, through clinical trials to registration – today’s milestone is testament to their achievements.

Michael Soldan, CEO

Upstaza™ – First disease-modifying treatment for AADC deficiency

Posted on July 20, 2022 by - Industry News

PTC Therapeutics to launch first gene therapy directly administered into the brain

PTC Therapeutics, Inc. announced on July 20th, 2022 that Upstaza™ (eladocagene exuparvovec) was granted marketing authorisation by the European Commission. Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older.

“Today’s approval from the European Commission for Upstaza for the treatment of AADC deficiency is momentous for patients, for PTC, as well as for the larger gene therapy community,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “We are proud to bring this innovative therapy to the marketplace so that patients may benefit. Upstaza is the first and only approved disease-modifying treatment for patients living with AADC deficiency. We are ready to deliver this long-awaited treatment to patients as soon as possible.”

During Upstaza clinical studies, patients went from not achieving any developmental motor milestones to demonstrating clinically meaningful motor skills from as early as three months following treatment, with transformational improvements shown to continue up to ten years after treatment. In addition, cognitive skills improved in all treated patients.1,2 Upstaza also reduced symptoms that cause potentially life-threatening and morbid complications.

Before treatment, our daughter had not met any development milestones. She suffered from oculogyric crises that evolved into hours of pain, and we were told she would be bedridden for life. After receiving Upstaza, she is now speaking, walking, running, and even riding horses. We’re thrilled with the EMA approval and the hope that this milestone brings to other children and families impacted by AADC deficiency.

Richard Poulin, Patient organisation Teach RARE founder, whose daughter was treated as part of a clinical trial.

The marketing authorisation is applicable to all 27 European Union member states, as well as Iceland, Norway and Liechtenstein. 

References

1 Tai CH, et al. Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency. Mol Ther. 2022;30(2):509-518.

2 Chien et al. AGIL-AADC gene therapy results in sustained improvements in motor and developmental milestones through 5 years in children with AADC deficiency. Poster presented at the 48th Annual Meeting of the Child Neurology Society, Charlotte, NC, USA, Oct 23-26, 2019.

3 Chien YH, et al. Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial. Lancet Child Adolesc Health. 2017;1(4):265-273.

About Upstaza™ (eladocagene exuparvovec)

Upstaza is a one-time gene replacement therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L‑amino acid decarboxylase (AADC) deficiency with a severe phenotype. It is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, containing the human DDC gene.1 It is designed to correct the underlying genetic defect, by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.2,3

The efficacy and safety profile of Upstaza has been demonstrated across clinical trials and compassionate use programmes.1 The first patient was dosed in 2010. In clinical trials, Upstaza demonstrated transformational neurological improvements. The most common side effects were initial insomnia, irritability and dyskinesia.

Administration of Upstaza occurs through a stereotactic surgical procedure, a minimally invasive neurosurgical procedure used for the treatment of a number of paediatric and adult neurological disorders. The Upstaza administration procedure is performed by a qualified neurosurgeon in centres specialised in stereotactic neurosurgery.

About aromatic L-amino acid decarboxylase (AADC) deficiency

AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life – physical, mental and behavioral. The suffering of children with AADC deficiency may be exacerbated by: episodes of distressing seizure-like oculogyric crises causing the eyes to roll up in the head, frequent vomiting, behavioral problems, and difficulty sleeping.

The lives of affected children are severely impacted and shortened. Ongoing physical, occupational and speech therapy, and interventions, including surgery, also are often required to manage potentially life-threatening complications such as infections, severe feeding and breathing problems.

About PTC Therapeutics, Inc.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialisation of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to innovate to identify new therapies and to globally commercialise products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients.

www.ptcbio.com

National Acquired Brain Injury Strategy

Posted on July 15, 2022 by - Acquired Brain Injury

Fact finding tour to help shape Acquired Brain Injury strategy

Chris Bryant MP has visited three very different facilities offering neurorehabilitation services across the country to gather information for the new Government wide Acquired Brain Injury (ABI) Strategy.

Chris Bryant is the Chair of the All Party Parliamentary Group for ABI and the Co-Chair of the ABI Strategy Programme Board and secured the Government’s commitment to the ABI Strategy after proposing a Private Members Bill last year.

He visited Walkergate Park in Newcastle with UKABIF’s Chief Executive, Chloe Hayward, who is also the Co-Chair of the Strategy’s Patient and Public Voice Reference Group. The Tyneside Centre provides neurorehabilitation and neuropsychiatry services for adults with a disability caused by injury or disease affecting the brain or spinal cord.

There they met with both staff and patients as they were shown around the 64 bed Centre, learning about the different rehabilitation services provided and the equipment and technology available to help people affected by an ABI to live as independently as possible.

In Sheffield they visited Steps Rehabilitation Centre which provides 23 rooms for residential rehabilitation for those aged over 16 years. Everything from hydrotherapy to music therapy is used along with the latest technology to offer a range of rehabilitation to patients. There Chris and Chloe met with staff and patients to hear their thoughts on what resources are currently available and what is needed.

Finally a trip to Birmingham Women and Children’s Hospital to see what rehabilitation services are available for children. Chris met with staff, patients and families on a tour of the city centre hospital and also spoke with The Child Brain Injury Trust on the visit.

At each location staff and patients were given the opportunity to say what they thought was needed to improve the lives of those living with acquired brain injuries.

Being able to see and speak to both staff and patients means we have real life experiences from people. As Co-Chair of the Strategy’s Patient and Public Voice Reference Group I want to make sure patients’ views are taken into consideration when the Strategy is being established.

Chloe Hayward, Executive Director of UK Acquired Brain Injury Forum (UKABIF)

Chris Bryant MP said: “These visits have been hugely helpful as we look to gather information and first hand views from people working in ABI neurorehabilitation. If the ABI Strategy is to be fit for purpose we need to know what is currently working but perhaps more importantly what isn’t working in order to ensure the needs of people with an ABI are properly addressed.

“I want to say a huge thank you to everyone we met for sharing their experiences for us to consider as we take the Strategy forward.”

Birmingham Children’s Hospital
Steps Rehabilitation
Walkergate Park

Man feeds himself with robotic arms

Posted on July 14, 2022 by - Rehabilitation

Robotic arms connected directly to brain of partially paralysed man allows him to feed himself.

Recent advances in neural science, robotics, and software have enabled scientists to develop a robotic system that responds to muscle movement signals from a partially paralysed person relayed through a brain-machine interface.

Two robotic arms – a fork in one hand, a knife in the other – flank a seated man, who sits in front of a table, with a piece of cake on a plate. A computerised voice announces each action: “moving fork to food” and “retracting knife.” Partially paralysed, the man makes subtle motions with his right and left fists at certain prompts, such as “select cut location”, so that the machine slices off a bite-sized piece. Now: “moving food to mouth” and another subtle gesture to align the fork with his mouth.

In less than 90 seconds, a person with very limited upper body mobility who hasn’t been able to use his fingers in about 30 years, just fed himself dessert using his mind and some smart robotic hands.

A team led by researchers at the Johns Hopkins Applied Physics Laboratory (APL), in Laurel, Maryland, and the Department of Physical Medicine and Rehabilitation (PMR) in the Johns Hopkins School of Medicine, published a paper in the journal Frontiers in Neurorobotics that described this latest feat using a brain-machine interface (BMI) and a pair of modular prosthetic limbs.

Also sometimes referred to as a brain-computer interface, BMI systems provide a direct communication link between the brain and a computer, which decodes neural signals and ‘translates’ them to perform various external functions, from moving a cursor on a screen to now enjoying a bite of cake. In this particular experiment, muscle movement signals from the brain helped control the robotic prosthetics.

The study built on more than 15 years of research in neural science, robotics, and software, led by APL in collaboration with the Department of PMR, as part of the Revolutionizing Prosthetics program, which was originally sponsored by the US Defense Advanced Research Project Agency (DARPA). The new paper outlines an innovative model for shared control that enables a human to manoeuvre a pair of robotic prostheses with minimal mental input.

“This shared control approach is intended to leverage the intrinsic capabilities of the brain machine interface and the robotic system, creating a ‘best of both worlds’ environment where the user can personalise the behaviour of a smart prosthesis,” said Dr Francesco Tenore, a senior project manager in APL’s Research and Exploratory Development Department. The paper’s senior author, Tenore focuses on neural interface and applied neuroscience research.

Although our results are preliminary, we are excited about giving users with limited capability a true sense of control over increasingly intelligent assistive machines

Dr Francesco Tenore, a senior project manager in APL’s Research and Exploratory Development Department

Trial shows benefits in patients with Duchenne Muscular Dystrophy

Posted on July 13, 2022 by - Uncategorised

Topline Data from Phase 3 Trial shows beneficial effect of Givinostat 

22nd June, 2022: Italfarmaco Group announced positive topline data from its completed Phase 3 EPIDYS trial with Givinostat, the company’s proprietary histone deacetylase (HDAC) inhibitor, in boys with Duchenne Muscular Dystrophy (DMD). The primary objective of the study was to evaluate the effects of Givinostat on slowing disease progression in ambulant DMD boys aged 6 years and above on chronic steroids. The study compared Givinostat to placebo and met the primary endpoint (change from baseline in the time to climb 4 stairs) following 18 months of treatment in the target population1 with key secondary endpoints consistent with the functional primary endpoint. Givinostat continued to demonstrate a tolerability profile in line with previous studies. The topline data were presented by Italfarmaco Group’s Chief Medical Officer, Dr. Paolo Bettica on June 25, 2022, at the hybrid Parent Project Muscular Dystrophy (PPMD) Annual Conference.

In October 2020, the US Food and Drug Administration (FDA) granted a Rare Pediatric Disease designation to Givinostat for the treatment of DMD. Earlier, the company also received an Orphan Drug Designation from the FDA and EMA, and a Fast Track designation from the FDA. Based on the study results, which show that the addition of Givinostat to steroid treatment leads to clinical benefits for the individuals in the study, the company plans to meet with US and EU regulators to discuss the potential for marketing application submission and seek input on a future submission of the application for regulatory approval. The company intends to submit the complete results of the EPIDYS study for publication in a peer-reviewed journal in due course.

The EPIDYS Phase 3 clinical trial is a randomised, double-blind, placebo-controlled, multicentre study evaluating the efficacy and safety of Givinostat in individuals with DMD (Cinicaltrials.gov: NCT02851797). The trial enrolled 179 male ambulant individuals with a mean age of 9 years and on stable steroids for at least 6 months. Boys were randomised 2:1 and treated chronically with an oral suspension of Givinostat or placebo for a period of 18 months. Of these, 120 boys formed the target population1.

Overview of Clinical Results

Primary Endpoint: 

The mean change from baseline to climb 4 stairs was selected as the primary endpoint to assess the efficacy of Givinostat compared to placebo. The results demonstrated a slower decline to perform this functional task in the Givinostat-treated group (difference vs Placebo of 1.78 seconds, p=0.0345).

Key Secondary Endpoints: 

A variety of secondary endpoints were analysed that showed results consistent with the functional primary endpoint. These included functional tests such as the North Star Ambulatory Assessment (NSAA) and the time to rise (TTR) test along with muscle strength analyses. Fat infiltration in the vastus lateralis (VL) muscle of the thigh is a characteristic of disease progression in DMD patients and was measured using a non-invasive objective imaging method called Magnetic Resonance Spectroscopy (MRS) to assess the efficacy of Givinostat. The data indicated that treatment with Givinostat delayed fat infiltration by approximately 30% (difference vs Placebo, -2.9%, nominal p=0.035). Additional exploratory endpoints were also analysed in the clinical study. These results further support the potential of Givinostat in providing a clinical benefit for boys with DMD.

Safety and Tolerability: 

The majority of Adverse Events (AE) were mild to moderate in severity (95%). Three (2.5%) boys treated with Givinostat withdrew from the trial because of an AE. Similar to what had been previously observed, the AE occurring in at least 1/10 subjects were diarrhea, abdominal pain, thrombocytopenia, hypertriglyceridemia, platelet decrease and triglyceride increase. Givinostat tolerability was managed with appropriate monitoring and dose adjustments. No other safety concerns were observed.

These results show Givinostat’s beneficial effect in DMD boys providing evidence of its ability to slow down disease progression.

Prof. Eugenio Mercuri, Professor of Paediatric Neurology at the Catholic University, Rome, Italy