Weekly Spotlight - 03.10.24

In the News

Webinar: Advanced Respiratory Care in ALS

Join us for the final chapter in the Advanced Respiratory Care in ALS series, where we unravel the intricate tapestry of respiratory failure in ALS. This session promises to be a treasure trove of insights, focusing on end-of-life and quality of life considerations, with an eye towards timely advanced directives. Our expert presenters will navigate the labyrinth of respiratory care, shedding light on the medical management of breathlessness and the pivotal decision-making process between non-invasive and tracheostomy-invasive ventilation.

Designed for healthcare professionals, caregivers, and those living with ALS, this webinar aims to elevate your understanding and enhance patient care through informed decision-making. With a stellar line-up of presenters from esteemed institutions like VCU Health, UT Southwestern, and Mayo Clinic, you're in for an enlightening experience. So, mark your calendars and prepare to dive deep into the world of ALS respiratory care, where science meets compassion, and knowledge paves the way for better patient outcomes.

Charlotte Hawkins to present MND Association's BBC Lifeline Appeal

Charlotte Hawkins, a well-known radio and television presenter, will lead the Motor Neurone Disease Association's BBC Lifeline Appeal next month. As a dedicated patron, Charlotte's connection to MND is deeply personal; her father, Frank, was diagnosed with the disease in 2011 and passed away four years later. The appeal, set to air on BBC1 and BBC2, aims to raise awareness and highlight the crucial support the Association provides across England, Wales, and Northern Ireland. Viewers will hear poignant stories from individuals like James, Eoin, and Maggie, who have been directly affected by MND.

Charlotte expressed her pride in presenting the appeal, emphasising the importance of continuing the Association's work in funding vital research and supporting families. The broadcast will be available on BBC1 on 13 October, with the exact time to be confirmed. This initiative offers a unique opportunity to shed light on the devastating impact of MND and the essential services provided by the Association.

Epigenomic Insights into ALS Risk Factors and Progression

Researchers at MIT have made significant strides in understanding amyotrophic lateral sclerosis (ALS) by analysing epigenetic modifications in motor neurons derived from induced pluripotent stem cells of nearly 400 ALS patients. This study, part of the Answer ALS consortium, identified about 30 genomic regions with chemical modifications linked to disease progression. These findings could pave the way for developing targeted treatments for different ALS subtypes, offering hope for more personalised and effective therapies.

The study highlights the importance of large-scale patient data in uncovering the genetic and epigenetic factors driving ALS. By focusing on specific genomic regions, researchers aim to develop drugs tailored to individual genetic profiles, potentially transforming ALS treatment. This approach, supported by extensive funding and collaboration, underscores the potential for significant advancements in ALS therapy, bringing hope to patients and their families.

Discovering New Biomarkers for MND: A Researcher's Journey

Eleni Christoforidou, a Research Fellow at the University of Sussex, is dedicated to identifying biomarkers for Amyotrophic Lateral Sclerosis (ALS), a form of Motor Neurone Disease (MND). Her research focuses on non-coding RNAs (ncRNAs) found in blood samples from clinical trials. By analysing these ncRNAs with machine learning, she aims to track disease progression and predict its severity, potentially leading to earlier diagnoses and personalised treatments.

Attending MND EnCouRage UK was transformative for Eleni, providing her with invaluable feedback from both scientists and those affected by MND. The event underscored the importance of translational research and effective communication, reinforcing her commitment to making a tangible difference in the lives of those living with MND.

Japan Approves New ALS Drug, Offering Hope to Patients

Japan's health ministry has approved mecobalamin, a new drug for amyotrophic lateral sclerosis (ALS), sparking hope among patients. This vitamin B12 derivative, initially proposed in 2015, showed significant efficacy in extending patients' survival by over 500 days in recent clinical trials, surpassing existing treatments.

Conducted by Tokushima University and other facilities, the trial revealed that mecobalamin, when administered early, significantly outperformed riluzole, the current standard treatment. Researchers are optimistic about combining mecobalamin with other drugs to potentially halt ALS progression. ALS patient Koichiro Miho expressed strong hopes for a cure, reflecting the broader patient community's optimism.

Blood Test for ALS Diagnosis Validated by Scientists

Scientists have validated a blood test that can distinguish ALS patients from healthy individuals and those with other neurodegenerative diseases with up to 98% accuracy. This test measures eight specific microRNAs, creating an "ALS fingerprint" that could speed up diagnosis and improve patient outcomes. The test is expected to be available within two years, pending patent approval.

The study, published in Brain Communications, involved 119 ALS patients and various control groups. The results showed consistent differences in microRNA levels, providing confidence in the test's reliability. Researchers hope this biomarker will complement clinical assessments, reducing diagnostic uncertainty and potentially aiding in monitoring disease progression and treatment responses.

MediciNova Supports NIH-Funded ALS Clinical Trial with MN-166

MediciNova, in collaboration with NIH, will support a $22 million Expanded Access Protocol to evaluate MN-166 (ibudilast) for ALS. This trial, funded by the ACT for ALS Act, aims to provide the investigational drug to 200 ALS patients, enhancing understanding of its potential therapeutic benefits.