Weekly Spotlight - 10.10.24

In the News

Cancer Therapy Triggers Myasthenia Gravis and Heart Inflammation in Patient

The case of a 72-year-old man developing myasthenia gravis (MG) and myocarditis after receiving chemoimmunotherapy for gastric cancer highlights the need for further research into immune checkpoint inhibitors like sintilimab. These inhibitors, designed to help the immune system target cancer cells, have been linked to immune-related adverse events, including heart inflammation and muscle weakness. The patient's symptoms, such as eyelid droopiness and double vision, emerged shortly after treatment, necessitating a change in therapy and the introduction of immunosuppressants. His condition improved over time, with biomarkers of heart damage returning to normal levels.

This case underscores the complexity of managing immune-related side effects in cancer treatment, particularly with the use of immune checkpoint inhibitors. The researchers emphasise the importance of understanding the mechanisms behind these adverse effects to improve patient outcomes. As the first reported case of its kind, it calls for the development of effective management strategies and long-term care plans for patients experiencing similar complications. This highlights the ongoing need for scientific exploration to enhance therapeutic approaches and patient safety in cancer treatment.

AI Enhances Neurologists' Personalised Treatment for Myasthenia Gravis

The MG-PerMed project, coordinated by Paola Cavalcante, aims to personalise treatment for Myasthenia Gravis (MG) patients by integrating clinical and biomarker data from Italy, Israel, and France. This EU-financed initiative seeks to develop an AI model that assists neurologists in selecting the most effective therapies based on patient-specific factors, such as genetic variants and circulating biomarkers. The project underscores the importance of patient involvement, ensuring that treatments are tailored to individual needs while maintaining ethical standards, such as data protection and equal access to AI tools.

Despite the advancements in AI, the project emphasises that these tools are designed to support, not replace, neurologists, who retain the ultimate responsibility for treatment decisions. Ethical considerations, including the risk of discrimination and the need for patient-centred care, are central to the project's approach. By fostering patient-clinician interactions and utilising telemedicine, the initiative aims to enhance the quality of care and ensure that patients are active participants in their treatment journey, reflecting a broader trend towards personalised medicine in healthcare.

Myasthenia Alliance Webinar on PBAC Submission Outcome

The Myasthenia Alliance Australia (MAA) Board invites the Australian Myasthenia community to participate in an informative webinar on 11 October 2024. This session, facilitated by Alexion, aims to elucidate the processes involved in securing new treatment options, focusing on the recent outcome of the Pharmaceutical Benefits Advisory Committee (PBAC) submission. Attendees will gain insights into ongoing advocacy efforts, with the opportunity to engage through a Q&A chat post-presentation. The session prioritises privacy, ensuring no personal data is collected.

Scheduled for 12pm daylight saving time in Canberra, Melbourne, and Sydney, and 11am in Queensland, the webinar promises a comprehensive exploration of the PBAC submission process. Participants are encouraged to provide feedback via an anonymous survey at the session's conclusion. The MAA Board emphasises the importance of this event in fostering understanding and engagement within the Myasthenia community, highlighting the collaborative efforts to advance treatment options.

Predictors of Poor Outcomes in Myasthenia Gravis with COVID-19 Infection

The study conducted at Tongji Hospital in Wuhan, China, explored the impact of COVID-19 on patients with myasthenia gravis (MG). It revealed that while COVID-19 did not significantly alter the MG course for most, certain factors predicted poor outcomes. Patients with generalised MG, chronic kidney disease (CKD), or malignancies were more likely to experience exacerbations, hospitalisation, or myasthenic crises. Immunosuppressive therapy, while reducing risks of exacerbation and hospitalisation, posed long-term challenges, necessitating careful management.

Age at MG onset and the presence of anti-acetylcholine receptor antibodies were also significant predictors of poor outcomes. The study highlighted that older age at MG onset increased the risk of exacerbation and hospitalisation. Additionally, patients with positive anti-AChR antibodies had a higher likelihood of adverse outcomes. The research underscores the importance of monitoring immune markers and managing immunosuppressive therapy to mitigate risks, providing valuable insights into the complex interplay between MG and COVID-19.

Amgen's Uplizna Shows Promise in Myasthenia Gravis Treatment Trial

Amgen's Uplizna has demonstrated promising results in a phase 3 trial for myasthenia gravis (MG), a rare autoimmune disorder. The study revealed a median improvement of 4.2 points on the MG-ADL scale for patients treated with Uplizna, compared to a 2.3-point improvement for those on placebo. This significant finding underscores Uplizna's potential as a meaningful treatment option, particularly due to its unique dosing schedule and ability to maintain efficacy while reducing steroid use, which is crucial given the harmful effects of prolonged high-dose steroid therapy.

The success of Uplizna in this trial positions it for regulatory approval to treat MG, adding to its existing indication for neuromyelitis optica spectrum disorder. Amgen's strategic acquisition of Horizon Therapeutics has bolstered its portfolio, with Uplizna generating $92 million in sales in the second quarter. This expansion into new indications not only enhances Amgen's market presence but also offers hope for patients with limited treatment options, highlighting the company's commitment to advancing therapeutic solutions for rare diseases.

Marathon Participation Supports Neuromuscular Disease Research and Patient Independence

Participating in the 2024 TCS New York City Marathon with MDA's Team Momentum offers a unique opportunity to support individuals living with muscular dystrophy, ALS, and related neuromuscular diseases. Registration opens on March 28 for previous participants and on March 29 for new entrants. As a Team Momentum athlete, you will receive guaranteed marathon entry, fundraising support, a personalised training schedule, and more. The commitment involves raising £4,000 by October 20, with a £50 registration fee contributing towards this goal.

The experience promises not only the thrill of the marathon but also the satisfaction of contributing to a meaningful cause. Participants are encouraged to share their journey with friends and family through social media and other channels. For further details or inquiries, Angelica Peck can be contacted via email or phone. This initiative not only highlights the spirit of athleticism but also underscores the importance of community support in advancing the lives of those affected by neuromuscular conditions.

Algorithm Enhances Myasthenia Gravis Diagnosis via Telehealth

Researchers at George Washington University have developed a promising algorithm to standardise myasthenia gravis assessments via telehealth. This innovation aims to address the challenges faced by patients unable to attend clinics, offering a digitised version of the Myasthenia Gravis Core-Exam (MG-CE). By employing deep learning and image processing, the algorithm provides objective, reproducible, and quantitative reports, enhancing clinicians' decision-making capabilities. The study involved capturing Zoom videos of 52 patients and 15 healthy controls, demonstrating the algorithm's potential in accurately measuring symptoms such as eye tracking and muscle weakness.

Despite its promise, the algorithm requires further validation to overcome telehealth's inherent challenges, such as motion capture and patient adherence to video protocols. Dr Gulsen Oztosun highlighted the need for additional testing to refine the system, acknowledging limitations like network difficulties and patient compliance. The research, presented at the American Neurological Association annual meeting, underscores the potential of digital tools in revolutionising myasthenia gravis diagnosis, though significant work remains to achieve full automation and reliability in telehealth settings.

Danish Musician's Journey with Myasthenia Gravis: A Story of Resilience

Mika Filborne, a Danish musician, faced the onset of myasthenia gravis in 2016, initially experiencing symptoms like double vision and a droopy eyelid. Despite the challenges, including a temporary halt in his music career, Mika adapted to life with this invisible disability. His journey underscores the importance of acceptance and adaptation, as he resumed composing and performing with renewed vigour. His story highlights the resilience required to live with myasthenia gravis, emphasising the need to listen to one's body and embrace the condition rather than resist it.

Three years after the initial symptoms, Mika received an official diagnosis, which he accepted with a positive outlook. His experience illustrates the transformative power of perspective, as he learned to manage his symptoms by reducing stress and focusing on his music. Mika's advice to others with myasthenia gravis is to respect the condition and find peace within oneself. His narrative serves as a testament to the human spirit's ability to adapt and thrive, even in the face of chronic illness, offering valuable insights into living with myasthenia gravis.