Weekly Spotlight - 10.10.24

In the News

Loqus23 secures funding to advance Huntington's treatment development

Loqus23 Therapeutics has successfully secured £35 million in funding to advance its promising small molecule candidate for Huntington's disease. This significant financial boost, led by Forbion and supported by existing investors like SV Health Investors Dementia Discovery Fund and the Novartis Venture Fund, will propel the candidate through preclinical development and early clinical trials anticipated in 2026. The focus is on developing a treatment that targets the somatic expansion of CAG repeats in the HTT gene, a key factor in Huntington's progression. By suppressing the MutS-beta protein, Loqus23 aims to slow or halt disease progression, offering hope for a transformative treatment.

The company's approach is grounded in the understanding that somatic expansion, driven by aberrant DNA mismatch repair, is a primary cause of Huntington's. By targeting this mechanism, Loqus23's small molecules could potentially delay the onset or progression of symptoms. This development is a beacon of hope for those affected by Huntington's, as it represents a step towards a disease-modifying treatment. The collaboration with Forbion and other investors underscores the potential impact of this innovative approach on patients' lives.

Researchers discover huntingtin protein mutation disrupts DNA repair in Huntington's patients

Researchers at McMaster University have uncovered a significant finding regarding Huntington's disease, offering hope for future treatments. The study reveals that the mutated huntingtin protein in patients fails to repair DNA as it should, impacting brain cell recovery. This discovery highlights the protein's role in creating molecules called Poly [ADP-ribose] (PAR), essential for DNA repair. Surprisingly, lower PAR levels were found in Huntington's patients, contrasting with other neurodegenerative disorders. This insight opens new avenues for research, particularly in cancer treatment, as PARP inhibitors, which halt PAR production, are used in oncology.

The implications of this research extend beyond Huntington's disease. The study suggests that drugs lowering huntingtin levels, currently in clinical trials, might also benefit cancer patients. Collaborations with other research centres are underway to explore this potential further. This discovery not only sheds light on the genetic disorder but also offers a glimmer of hope for broader medical applications. The research underscores the importance of continued investigation into the complex interactions of proteins and DNA repair, promising advancements in both neurodegenerative and cancer therapies.

FDA taps URMC to develop new digital measures for Huntington's disease

The University of Rochester Medical Center (URMC) is embarking on a promising new study, funded by the FDA, to develop digital measures for Huntington's disease. This initiative aims to detect early signs of the disease using wearable sensors, focusing on mobility and chorea, which are key symptoms. Led by neurologist Jamie Adams, the research seeks to provide objective measures that could accelerate the development of new therapies. The study will involve continuous data collection from individuals with early-stage Huntington's, using innovative symptom mapping to ensure the measures' relevance to patients.

This research holds the potential to transform clinical trials for Huntington's by providing crucial endpoints for assessing motor impairments. As genetic interventions for neurological disorders advance, early and accurate assessment becomes vital. The study, involving experts from Columbia, Tel Aviv, and Massachusetts universities, is set to begin participant enrolment later this year. By focusing on early-stage symptoms, the research not only aims to benefit Huntington's patients but also offers hope for those with other neurological diseases, such as Parkinson's, highlighting a future of improved care and support.

Huntington's Disease: Brain and Blood Vessel Impact Explored

Huntington's disease, a genetic condition leading to dementia, affects not only nerve cells but also the brain's microscopic blood vessels. This discovery, made by researchers from Lancaster University and other institutions, highlights the potential for early detection and monitoring of the disease. By examining the coordination between neuronal activity and brain oxygenation, the study reveals how the brain's energy needs are met through a complex interplay of rhythms. These findings could pave the way for new treatments targeting both the vasculature and brain metabolism, offering hope for those affected by Huntington's and similar neurodegenerative diseases.

The research utilised non-invasive techniques, such as infrared light and electrodes, to measure brain blood oxygenation and electrical activity. This approach allowed the team to assess the efficiency of the neurovascular unit, which is crucial for brain function. Professor Aneta Stefanovska expressed optimism that these methods could monitor disease progression and evaluate potential treatments. The study's insights into the brain's vasculature and metabolism may inspire new therapeutic strategies, providing reassurance and hope to those impacted by Huntington's disease.

HDBuzz seeks community support to sustain unbiased HD reporting

HDBuzz, a vital resource for the Huntington's disease (HD) community, is seeking support to ensure its continued operation. Since 2010, HDBuzz has provided unbiased, accessible scientific information, funded by non-profit organisations. However, a recent loss of a major donor has highlighted the need for a more sustainable funding model. HDBuzz remains committed to delivering free, plain-language reports on HD research and clinical trials, helping families make informed decisions about their care and treatment. The organisation is now reaching out to its readers for donations to maintain independence and expand its services.

Donations will support website maintenance, content translation, and community engagement, ensuring HDBuzz remains a trusted source of information. The team, deeply embedded in the HD community, is dedicated to advancing understanding and treatment of HD. By contributing, supporters can help HDBuzz continue its mission of providing hope and education to HD families worldwide. Every contribution, no matter the size, is appreciated and will aid in sustaining and expanding HDBuzz's invaluable work.

Sage Therapeutics faces setback in Alzheimer's research, awaits Huntington's data

Sage Therapeutics Inc. recently faced a setback in their Alzheimer's research, as their phase II Lightwave study of dalzanemdor did not meet its primary outcome measure. This has led the company to halt the development of this NMDA receptor positive allosteric modulator for Alzheimer's. While this news is disappointing, it is important to remember that the journey of medical research is often fraught with challenges. Each step, even those that don't yield the desired results, contributes to the broader understanding of these complex diseases.

Despite this hurdle, Sage remains hopeful as they await data from their Huntington's disease research. The scientific community continues to work tirelessly, driven by the shared goal of finding effective treatments for neurodegenerative diseases. It's crucial to support these efforts, recognising the dedication and resilience of researchers who strive to improve the lives of those affected by such conditions. As we look forward to future developments, the commitment to advancing medical knowledge and patient care remains unwavering.