DDW’s Diana Spencer reviews some of the advances in drug discovery for diseases of the brain in the last month.
Brain Awareness Week is an annual global campaign that takes place from March 13-19, aimed at raising awareness and support for brain science and issues related to the brain and nervous system.
It is described on the website as “the global campaign to foster public enthusiasm and support for brain science.”
One of the conditions that the campaign seeks to highlight is stroke. According to GlobalData, the incidence of ischaemic stroke in the US is 0.2% as of 2022, and that number is expected to slowly increase to 0.3% by 2030. As for haemorrhagic stroke, GlobalData forecasts suggest that the incidence rates in the US were 0.02% in 2022 and are predicted to rise to 0.03% by 2030.
Alexandra Murdoch, Medical Analyst at GlobalData, comments: “Ischaemic strokes are far more common, accounting for roughly 80% of stroke cases, but haemorrhagic strokes are usually more damaging. Time is a really important factor when discussing stroke treatment. It is crucial to seek medical attention when stroke symptoms occur to treat it as quickly as possible.”
Even in just the last month, the drug discovery sector has made some incredible advances in the treatment of diseases of the brain and central nervous system, including stroke. Here are a few of them.
Stroke
Biopharmaceutical company Basking Biosciences revealed positive Phase I study results for its reversible thrombolytic therapeutic for ischaemic stroke.
The company’s novel von Willebrand Factor (vWF)-targeting thrombolytic agent, BB-031 demonstrated safety and tolerability following a single intravenous dose.
Read more: RNA aptamer reopens blocked arteries
Parkinson’s disease
Researchers identified that a gene associated with an increased risk of Parkinson’s Disease also contributes to a build-up of cell debris in the brain.
Dr Adekunle Bademosi from The Queensland Brain Institute said the discovery could change the focus of Parkinson’s Disease treatment.
Read more: Focus on autophagy could lead to more effective Parkinson’s drugs
Dementia
An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study.
The study found that four out of five patients diagnosed with Sanfilippo have continued to gain cognitive skills in line with development in healthy children after being given the investigational gene therapy, OTL-201.
Read more: Gene therapy for ‘childhood dementia’ shows promise
Adrenoleukodystrophy
Minoryx Therapeutics revealed the findings from the first and largest international study to enroll adult male X-linked adrenoleukodystrophy patients.
The Phase II/III ADVANCE clinical trial of lead candidate, leriglitazone, published in The Lancet Neurology, did not meet the primary outcome for the six-minute walk test.
However, clinical measures of body sway (assessing balance) demonstrated clinically relevant differences and favourable trends were observed for Expanded Disability Status Scale (EDSS), Severity Score System for Progressive Myelopathy (SSPROM) and quality of life.
Read more: New treatment slows progression of neurodegenerative disease
TUBB4a leukodystrophy
The US Food and Drug Administration (FDA) has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.
RPDs are designed to encourage the development of new drugs with high unmet medical needs, including rare diseases in children.
Read more: Treatment for leukodystrophy could be “revolutionary”
Find out more about Brain Awareness Week and how to get involved: https://www.brainawareness.org
