Research roundup: drug-testing platform for ALS, NHS dementia savings, sex hormones and neurological health, and more

Neuro Rehab Times explores the latest neurology research and developments, from cost-savings for the NHS to mapping the complex interactions between sex hormones and neurological health and more.

Remotely-delivered support programme could save NHS £9000 per year for each person with dementia

The NIDUS-Family package of care uses goal setting to help people with dementia live well at home for longer.

Now, new research from Queen Mary University of London in collaboration with UCL shows that, in addition to these known benefits, NIDUS-Family also reduces the costs associated with providing support to people with dementia.

People with dementia who took part cost the NHS and social care £8934 (37 per cent) less on average over one year than people who did not receive the additional help.

These cost savings came about thanks to the person receiving the intervention spending less time in hospital and using less state-funded social care, compared with controls, costs of which far exceeded the modest £346 annual cost of this preventive intervention.

The new therapy has the potential to be rolled out to support consistent, evidence-based personalised dementia care across the NHS. The findings coincide with a call from the All-Party Parliamentary Group (APPG) inquiry on dementia for a levelling up of diagnosis rates and the care people receive after a diagnosis, recommending that high-quality post-diagnostic support services for dementia must be available more equitably across England.

Introduction of the National Plan for Epilepsy Act in Congress

A new Epilepsy Action plan has been reintroduced into Congress which will support the development of a comprehensive strategy to prevent, diagnose, treat, and cure epilepsy and improve the wellbeing of people with epilepsy and their families.

Over 120 epilepsy-related organisations joined together to celebrate the efforts and urge members of Congress to co-sponsor and pass the bill. The legislation would bring more attention, support, and resources to the nearly 3.4 million Americans living with epilepsy and other seizure disorders.

If passed by Congress and signed into law by the President, this bill will direct the federal government to develop a National Plan for Epilepsy. This plan will enable the federal coordination necessary to ensure better outcomes for people with epilepsy and prioritise the development of more effective epilepsy treatments.

Lipid nanoparticle platform delivers mRNA to brain through blood-brain barrier

Scientists have developed a lipid nanoparticle system capable of delivering messenger RNA (mRNA) to the brain via intravenous injection, a challenge that has long been limited by the protective nature of the blood-brain barrier.

The findings, in mouse models and isolated human brain tissue, demonstrate the potential of this technology to pave the way for future treatments for a wide range of conditions such as Alzheimer’s disease, amyotrophic lateral sclerosis, brain cancer, and drug addiction.

For the study, the research team designed and tested a library of lipids to optimize their ability to cross the blood-brain barrier. Through a series of structural and functional analyses, they identified a lead formulation, termed MK16 BLNP, that exhibited significantly higher mRNA delivery efficiency than existing lipid nanoparticles approved by the Food and Drug Administration (FDA).

The system takes advantage of natural transport mechanisms within the blood-brain barrier, including caveolae- and γ-secretase-mediated transcytosis, to move nanoparticles across the barrier, say the investigators.

In studies using mouse models of disease, the BLNP platform successfully delivered therapeutic mRNAs to the brain, demonstrating its potential for clinical application.

Accelerated drug-testing platform for ALS paves way for therapeutic innovation

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease with few treatment options.

Since 2018, the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH), a founding member of the Mass General Brigham healthcare system, has been working with national and international stakeholders to accelerate ALS research by launching the first platform trial in ALS to simultaneously test multiple drugs using shared trial infrastructure and placebo data.

Findings from the first four drugs evaluated through the trial have now been published.

By pooling placebo data across trials, the ALS platform reduces the proportion of patients assigned to placebos to 25 per cent, allowing more patients to receive investigational therapies.

Each of the first four trials included approximately 160 patients, with roughly 120 receiving an investigational drug and 40 receiving a placebo. As Phase 2 trials, the goal of these studies was to rapidly assess drug candidates and determine whether to proceed with larger trials. Of the four regimens, two drugs (CNM-Au8 and pridopidine) are moving to phase 3 testing.

Though neither met criteria demonstrating statistically significant benefit over 24 weeks, the Phase 2 trials showed promising trends in other outcome measures and biomarkers and helped clarify dosage information and target populations, informing the Phase 3 design.

The two other drugs tested, zilucoplan and verdiperstat, will not move to phase 3 trials, though deidentified placebo data and samples will be shared in public databases for future research thus supporting additional research in ALS and other neurodegenerative diseases. Three new trials from the platform have completed enrolment, and additional trials will launch in the future.

How the brain recognises what is familiar and important

A new study has identified part of a brain circuit mixes sensory information, memories, and emotions to tell whether things are familiar or new, and important or just “background noise”.

Led by researchers from NYU Langone Health, the work found that a circuit known to carry messages from a brain region that processes sensory information, the entorhinal cortex (EC), to the memory processing center in the hippocampus (HC) has a previously unrecognized pathway that carries messages directly back to the EC.

A better understanding of the interplay between the two brain regions may yield new solutions to problems within related circuits, the researchers say, such as those seen in patients with post-traumatic stress disorder who struggle to tell apart past trauma from current loud noises, or in the sensory overload experienced by some children with autism as they try to tell apart objects or interact with people.

Mapping complex interactions between sex hormones and neurological health

A new review details how sex hormones influence neurological function through multiple mechanisms, including direct effects on nerve cells, epigenetic modifications, and the newly discovered brain glymphatic system.

The analysis covers an extensive range of conditions including vascular disorders such as migraine and stroke, movement disorders such as Parkinson’s disease, Wilson’s disease, and various forms of chorea, as well as epilepsy, multiple sclerosis, Alzheimer’s disease, sleep disorders, brain tumors, neuromuscular conditions, and other conditions such as intracranial hypertension and the porphyrias.

Some of the key findings from the review include that neurosteroids, which are hormone-derived molecules produced in the brain, have significant therapeutic potential; sex-specific approaches may be necessary for treating many neurological conditions; and that hormonal fluctuations during menstruation, pregnancy, and menopause can substantially impact disease manifestation and treatment responses, among other findings.

Singapore launches Mental Health Innovation Asia Hub at international symposium

In a significant advancement for mental health care, the SingHealth Duke-NUS Global Health Institute, in collaboration with the Mental Health Innovation Network (MHIN), has unveiled the MHIN Asia Hub.

The milestone event, a cornerstone of the inaugural Global Mental Health in Asia Symposium, marks a major step in addressing mental health challenges across the continent.

With ongoing initiatives and planned activities, the MHIN Asia Hub is poised to be a pivotal force in reshaping mental health landscapes across Asia, ensuring that innovations and solutions reach those in need through sustained and meaningful collaboration.