Sean M. Healey & AMG Center for Integrated ALS Care and Discovery awarded NIH grant to support expanded access with NUZ-001 in conjunction with Neurizon Therapeutics
Key takeaways
- NIH awards grant to Healey & AMG Center for a new Expanded Access Protocol (EAP) (FAIN U01NS153995)
- The grant will allow people living with ALS who are otherwise not eligible for clinical trials to access NUZ-001, an investigational drug by Neurizon Therapeutics
- This EAP will provide real world safety, biomarker, and clinical data in 200 people living with ALS
The Sean M. Healey & AMG Center for ALS at Mass General Brigham is pleased to announce that they have been awarded a grant from the National Institutes of Health (NIH) to conduct an Expanded Access Protocol (EAP) in amyotrophic lateral sclerosis (ALS) with NUZ-001 in conjunction with Neurizon Therapeutics.
Expanded Access, also referred to as Compassionate Use, is a pathway for individuals with a serious and life-threatening disease to access an investigational product that is not yet approved by the Food and Drug Administration (FDA). The EAP program provides the option for people living with ALS who are not otherwise eligible for clinical trials to be part of research by coordinating access to experimental drugs and enabling the collection of safety, disease progression and biomarker data. This data can help inform additional clinical trials or potentially help support market approval for people living with ALS .
This EAP will provide access to the investigational product NUZ-001 under development by Neurizon Therapeutics. NUZ-001 is an oral drug that targets key pathological mechanisms in ALS, including TDP-43 protein aggregation and impaired autophagy, which are central features across multiple neurodegenerative diseases. The safety and efficacy of NUZ-001 are currently being tested as Regimen I in the HEALEY ALS Platform Trial, a multicenter, double-blind, placebo-controlled trial for ALS created in partnership with the Network of Excellence for ALS (NEALS).
The EAP will be led by:
- Sabrina Paganoni, MD, PhD, Co-Principal Investigator of the HEALEY ALS Platform Trial and Co-Director of the Neurological Clinical Research Institute (NCRI) at Mass General Brigham
- Lori Chibnik, PhD, MPH, Associate Professor of Neurology at Harvard Medical School and Biostatistician at the NCRI
- Elisa Giacomelli, PhD, Research Associate in Neurology at Harvard Medical School and Research Scientist at the NCRI
- Doreen Ho, MD, Assistant Professor of Neurology at Harvard Medical School and Director of the ALS Clinic at the Healey & AMG Center
“We are committed to facilitating access to experimental products for all people living with ALS,” says Dr. Ho. “Many people living with ALS are not eligible for clinical trials because of their disease progression. EAPs provide an important opportunity for them to participate in research, and we are grateful to the NIH for awarding this grant to provide access.”
“In addition to providing access to an investigational product, EAPs provide safety, biomarker, and clinical data that helps researchers better understand ALS, leading to advancements in the field” says Dr. Chibnik.
“We would like to sincerely thank the researchers, study staff, Neurizon, and the ALS community for making this EAP possible,” says Dr. Giacomelli. “We look forward to keeping everyone updated through our community webinars as things progress.”
Next steps
As the program is currently in its early stages, specific enrollment dates and site details will be shared via upcoming community updates as they become available.
To learn more about the EAP program at the Healey & AMG Center, please email HealeyAMGCenterforALS@mgh.harvard.edu and visit our website.
Media contact
Brandon Chase
Senior Specialist, External Communications
bchase7@mgb.org
About the Sean M. Healey & AMG Center for Integrated ALS Care and Discovery at Mass General Brigham
At the Sean M. Healey & AMG Center for ALS at Mass General Brigham, we are committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development. Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it.
With many clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care. Together, we will find the cures.
About the Neurological Clinical Research Institute
The Neurological Clinical Research Institute (NCRI) at Mass General Brigham is an academic research organization composed of innovative researchers experienced and passionate about designing, developing, facilitating, and conducting multicenter clinical trials in neurological diseases. Our mission is to accelerate translational research in neurological disorders by initiating clinical development of novel therapies and leading trials of these compounds. We strive to be at the cutting edge of innovative trial design to accelerate therapy development by creating new trial methodology, discovering novel biomarkers and refining outcome measures.
About Mass General Brigham
Mass General Brigham is an integrated academic health care system, uniting great minds to solve the hardest problems in medicine for our communities and the world. Mass General Brigham connects a full continuum of care across a system of academic medical centers, community and specialty hospitals, a health insurance plan, physician networks, community health centers, home care, and long-term care services. Mass General Brigham is a nonprofit organization committed to patient care, research, teaching, and service to the community. In addition, Mass General Brigham is one of the nation’s leading biomedical research organizations with several Harvard Medical School teaching hospitals. For more information, please visit massgeneralbrigham.org.