OUR COMMITMENT:
We aspire for a brighter tomorrow for people living with devastating neurodegenerative diseases
Patients and their caregivers are at the center of our work
We are engaging and collaborating with patient communities, caregivers, and advocacy groups to understand their needs to inform our research and to raise awareness.
We strive to alleviate the burden and to improve the lives for those struggling with devastating neurodegenerative diseases. We recognize the urgency and seek to deliver solutions quickly and safely to patients and caregivers impacted by rare microgliopathies for which there are no treatment options available. When these diseases are genetic, they can affect generations of families, many of whom become caregivers and advocates.
If you would like to learn more about how Vigil is working with the patient community or you would like to collaborate with us, please contact us at patients@vigilneuro.com.
About Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP)
ALSP is a fatal, progressive, rare neurological disease characterized by issues with judgment, personality and psychological changes, cognitive decline, and problems with movement that eventually lead to loss of overall function. It is caused by a mutation in the CSF1R (colony-stimulating factor 1 receptor) gene and affects approximately 10,000 people in the United States with similar prevalence in Europe and Asia.
Initial diagnosis for ALSP is mainly clinical and later is confirmed through magnetic resonance imaging (MRI) and genetic testing for CSF1R mutation. Since ALSP symptoms are similar to other diseases, especially other neurodegenerative disorders, it can often be misdiagnosed. Genetic testing to identify a CSF1R mutation can be an important step in confirming an ALSP diagnosis. There are currently no FDA-approved treatments for ALSP.
Vigil is dedicated to supporting the ALSP community. We have led efforts to create a network of global disease experts, supported genetic testing initiatives, worked to improve our understanding of ALSP through a disease registry and a natural history study (called Illuminate), and we actively collaborate with patient organizations with a shared interest in improving the lives of those affected by ALSP.
Genetic Testing
ALSPAware is a program providing no-cost genetic testing and counseling services for the diagnosis of ALSP. By increasing access to genetic testing for both individuals and healthcare providers, ALSPAware has the potential to reduce initial misdiagnosis of this disease while also providing appropriate disease management services for those living with ALSP.
A single gene confirmatory test is available to individuals with a family history of ALSP and a custom gene panel is available for participating physicians to order on behalf of their patients to improve the diagnostic process. ALSPAware is coordinated by InformedDNA®, the largest independent provider of genetic counseling services, and PreventionGenetics, an Exact Sciences Company running an accredited laboratory that delivers high-quality genetic testing.
To learn more about ALSPAware or to find out how to access genetic testing services through the program, visit:
Community Perspectives
This video features Heidi Edwards, President and founder of Sisters’ Hope Foundation, a non-profit organization supporting families struggling with ALSP. The link to Sisters’ Hope Foundation website can be found in the Community Resources section below.
If you would like to learn more about Vigil Neuroscience or are interested in sharing your story, we would very much like to hear from you. Please reach out to us at patients@vigilneuro.com.
Heidi’s Story: A Family with ALSP
ALSP Community Webinar, December 2023
This is a recording from our December 2023 ALSP Community Webinar which includes updates from Vigil Neuro as well as a presentation from guest speaker, Dr. Florian Eichler, Director of the Leukodystrophy service at Massachusetts General Hospital. Dr. David Gray (Chief Science Officer) joins Cynthia Cassandro (Vice President, Patient Advocacy and Engagement) in a Q&A session on the recent VGL101 IGNITE Phase 2 clinical trial and ILLUMINATE Natural History Study interim analysis, and Dr. Eichler presents on the importance of the patient voice within the drug development process. The webinar also provides an overview of the impressive efforts and progress made by the ALSP community this year. If you have any questions or to request more information, please reach out to patients@vigilneuro.com.
This is a recording from our September 2023 Community Webinar providing an update for the ALSP community including Vigil Neuroscience leadership as well as Dr. David Lynch of the National Hospital for Neurology and Neurosurgery, University College London and Dr. Ashley Cannon of InformedDNA®. The webinar discusses stages of the drug development process, provides updates on Vigil's ALSP research and clinical trials and shares information on genetic testing - including ALSPAware, a Vigil sponsored, no cost, confidential genetic testing program in the United States. The webinar also offers resources and support for the ALSP community and shares details on Vigil’s community engagement efforts. If you have any questions or to request more information, please reach out to patients@vigilneuro.com.
ALSP Community Webinar, September 2023
ALSP Community Webinar, January 2023
This is a recording from our January 2023 Community Webinar providing an update for the ALSP community including Vigil Neuroscience leadership as well as Dr. Jennifer Orthmann-Murphy of Penn Medicine and patient advocacy organizations; Sisters’ Hope Foundation, United Leukodystrophy Foundation, ALEX The Leukodystrophy Charity and Hunter’s Hope Foundation. The webinar provides updates in clinical studies and trials, current thinking in ALSP research and resources and support for the community. If you have any questions or to request more information, please reach out to patients@vigilneuro.com.
Clinical Trials
As a leader in ALSP research, Vigil has multiple efforts ongoing to increase our understanding of ALSP while also working to bring a potential new therapy to the community. In addition to a Phase 1 clinical trial for iluzanebart (VGL101) involving healthy volunteers, our current trials for the ALSP community include:
An observational non-interventional natural history study, ILLUMINATE, with ALSP patients to better understand the disease and help us inform the clinical development of iluzanebart (VGL101) (NCT Number: NCT05020743)
A Phase 2 clinical trial, IGNITE, to evaluate the safety and effects of iluzanebart (VGL101) on imaging and biomarkers of disease progression in subjects with ALSP (NCT Number: NCT05677659)
Additional information about ALSP clinical trials can be found at clinicaltrials.gov.
If you or a loved one would like to learn more about our studies, please visit www.alspstudy.com or email us at trials@vigilneuro.com.
Community Resources
Increasing awareness and knowledge about ALSP is essential to patient empowerment. The following organizations offer support and education for those living with ALSP and the rare disease community as a whole:
* By clicking these links, you will leave Vigil Neuroscience website. These links are provided for informational purposes only. Vigil Neuroscience is not affiliated with these organizations and assumes no responsibility for the information, statements, or content.
Contact Us
If you would like to learn more about Vigil Neuroscience or are interested in sharing your story, we would very much like to hear from you. Please reach out to us at patients@vigilneuro.com.
If you would like to learn more about our clinical trials, please contact trials@vigilneuro.com.