Six advances in drug discovery for MND / ALS

Motor neuron disease awareness day

Ahead of Motor Neurone Disease (MND) Awareness Day on 21 June, we take a look back over the latest developments in the treatment of MND and amyotrophic lateral sclerosis (ALS).

  1. A new UK research partnership

In June, UK charity LifeArc pledged £1.5 million to fund a new two-year collaborative partnership with King’s College London (KCL) to validate a portfolio of human genetic driven therapeutic targets for MND.

The new partnership, which will involve scientists from both organisations working closely together, is part of LifeArc’s MND Translational Challenge, aimed at discovering innovative and potentially disease-modifying treatments for MND, a key component of the new UK MND Research Institute.

Read More: UK research partners team up to accelerate MND drug discovery

  1. Drug trial targets the blood brain barrier

In April, Axoltis Pharma received authorisation from ANSM, the French agency for the safety of health products, to launch the SEALS study for ALS, the most common form of MND.

This Phase II clinical trial of drug candidate NX210c in patients with ALS is the first to target the integrity of the blood brain barrier (BBB).

Read more: Drug candidate for ALS patients authorised for Phase II trial

  1. An oligonucleotide therapy collaboration

Also in April, PrecisionLife and the Nucleic Acid Therapy Accelerator (NATA) announced a collaboration to co-develop novel oligonucleotide therapies for sporadic motor neurone disease/ALS.

The collaboration combines NATA’s expertise in NAT design and optimisation with PrecisionLife’s understanding of complex disease biology and the mechanisms, drug targets and treatments that are relevant to specific ALS patient subgroups.

Read more: R&D partnership to jointly develop new ALS drugs

  1. Drug improves quality of life in MND

In March, NeuroSense Therapeutics reported additional positive data from its six-month double-blind Phase IIb PARADIGM trial of its lead drug candidate PrimeC for the treatment of ALS.

The PARADIGM trial’s secondary clinical efficacy outcome measure endpoints included Quality of Life and Survival. Consistent with the previously reported results, PrimeC displayed a clinically meaningful effect on various aspects of patients’ quality of life, including mental and physical health.

Read more: NeuroSense reports positive results from its ALS trial

  1. New genetic insights into neurodegenerative diseases

In January, new understanding of a gene that is linked to some forms of dementia and other age-related diseases gave scientists fresh hope that action can be taken against these diseases long before the onset of symptoms.

The gene – called Angiogenin or ANG – is associated with a number of neurodegenerative diseases commonly associated with old age, including frontotemporal dementia (FTD), MND and Parkinson’s disease.

Read more: Organoid research sheds light on gene mutation linked to dementia

  1. Guidelines to de-risk clinical trials

New guidance for the MND drug discovery community was published at the end of 2023 to ensure that future drug discovery and development efforts have the best chance of successfully translating from the lab to the clinic.

The whitepaper ‘Guiding principles for drug discovery and development in amyotrophic lateral sclerosis’ outlines a consensus-based approach to de-risk clinical trials of novel therapeutics by an enhanced evidenced-based approach to pre-clinical studies.

Read more: New guiding principles to overcome barriers to MND drug discovery

Diana Spencer, Senior Digital Content Editor, DDW

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