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Daily News Update: Tuesday, 18 June

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Welcome to your daily round-up of everything happening in the world of neurorehabilitation.

Research news

Rapid test of cerebrospinal fluid decreases time to diagnosis for brain tumours

A test that looks for genetic hallmarks of brain cancers in samples of cerebrospinal fluid can decrease the time to diagnosis and eliminate the need for invasive brain biopsies for some patients. Mass General Brigham experts in neurosurgery, cancer and pathology worked together to develop a rapid, genotyping test that can detect key mutations associated with brain cancers from samples taken during a lumbar puncture. The team evaluated the technique known as TetRS (Targeted Rapid Sequencing) among 70 patients admitted to Massachusetts General Hospital with new central nervous system (CNS) lesions of unknown origin. The test detected mutations in samples from 42 percent of patients with tumors, including cases of primary and secondary CNS lymphoma, glioblastoma, and gliomas. Results are published in Blood.

A new approach to neuroimaging analysis

A group of neuroscientists based at University of California San Diego School of Medicine has applied an approach to neuroimaging that they believe will reinvigorate the work of many of their fellow brain researchers. Their work, published in the journal Cerebral Cortex, outlines a method for the use of neuroimaging data to predict elements of cognition and behavioural variables. The technique involves examining widely used magnetic resonance images (MRIs) of brains to find associations to various behaviours, and then applying predictions from those associations to an independent unseen sample.

Getting images of a fast-acting brain protein

New groundbreaking images – from Columbia University Vagelos College of Physicians and Surgeons and published in Nature – of one of the brain’s fastest-acting proteins are providing critical clues that may lead to the development of targeted therapies to treat epilepsy and other brain disorders. The lightning-fast moves of the brain’s kainate receptor are indispensable for neuron-to-neuron communication but create a quandary for structural biologists trying to capture images of the receptor in action. When called into action, a kainate receptor embedded in the surface of a neuron opens its ion channel, then slams the channel shut within milliseconds.

Company and financial neuro-rehab news

Topline results from Phase 3 RAISE Trial

Marinus Pharmaceuticals, a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced topline results from the Phase 3 double-blind, randomized, placebo-controlled RAISE trial evaluating the safety and efficacy of intravenous (IV) ganaxolone for the treatment of refractory status epilepticus (RSE).

Topline data demonstrated that: The trial met the first co-primary endpoint: A statistically significant proportion of patients had status epilepticus cessation within 30 minutes of initiating IV ganaxolone compared to placebo: 80% vs. 13%, respectively.

The trial did not meet the second co-primary endpoint: RAISE failed to achieve statistical significance in the proportion of patients not progressing to IV anesthesia for 36 hours following initiation of IV ganaxolone compared to placebo: 63% vs. 51%, respectively.

The results also demonstrated that the incidence of serious adverse events was similar between the treatment and placebo arms, with hypotension being more commonly seen in the IV ganaxolone arm.

Afamelanotide in fair-skinned Parkinson’s patients

CLINUVEL has announced a novel clinical programme evaluating afamelanotide as a treatment in early-stage Parkinson’s Disease in fair-skinned patients. The programme objectives are to determine whether afamelanotide – through melanocortin-1 receptor (MC1R) activation – is able to lower α-synuclein (a toxin) in blood levels in PD patients, and positively affect neurons of the midbrain. MC1R is known to be a key receptor in brain and skin cells.

In large studies, it was found that fair-skinned patients have a higher risk of Parkinson’s associated with a malfunctioning MC1R.a, 1-2 Since afamelanotide is known to optimise the function of the MC1R, it is hypothesised that the drug treatment would have a positive effect in PD by lowering α-synuclein, as recently demonstrated in preclinical studies.²ˉ³ Afamelanotide is marketed in Europe and the USA as SCENESSE for patients diagnosed with erythropoietic protoporphyria.

Neurotechnology news

New technology allows researchers to precisely, flexibly modulate brain

Human brain diseases, such as Parkinson’s disease, involve damage in more than one region of the brain, requiring technology that could precisely and flexibly address all affected regions simultaneously. Researchers at Washington University in St. Louis have now developed a non-invasive technology combining a holographic acoustic device with genetic engineering that allows them to precisely target affected neurons in the brain, creating the potential to precisely modulate selected cell types in multiple diseased brain regions.

Hong Chen, associate professor of biomedical engineering in the McKelvey School of Engineering and of neurosurgery in the School of Medicine, and her team created AhSonogenetics, or Airy-beam holographic sonogenetics, a technique that uses a noninvasive wearable ultrasound device to alter genetically selected neurons in the brains of mice. Results of the proof-of-concept study were published in Proceedings of the National Academy of Sciences June 17.

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