Inclusive ALS Grant Expands Access to CNM-Au8 Therapy for ALS: Karen S. Ho, PhD

WATCH TIME: 7 minutes

“We're very excited about this new therapy because it is a very novel type of therapy. It's not a small molecule, it's not biologic. It's a suspension of gold nano crystals that have catalytic activity. This catalytic activity helps neurons survive many disease-related stressors, and in the context of ALS, helps those motor neurons survive that are dying in us. The grant allows us to grant access to give access to patients living with ALS, who would normally not qualify for a clinical trial, who are perhaps more advanced in their disease state.”

An Expanded Access Protocol (EAP), also known as Compassionate Use, is an FDA-regulated pathway that allows patients with a serious and life-threatening disease to access an investigational drug not yet approved by the FDA. In recent news, the National Institute of Neurological Disorders and Stroke awarded a 4-year grant to Clene Nanomedicine in collaboration with Columbia University and Synapticure, totaling $45.1 million, in support of an EAP for Clene’s CNM-Au8 for amyotrophic lateral sclerosis (ALS).¹ The EAP grant is part of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS) which is supported by Congress and was signed into law by President Biden on Dec. 23, 2021.

According to a recent statement, the grant is a call for increased support of public-private partnerships that will innovate development and increase access to potential new treatments for ALS.¹ Additionally, the EAP study assessing CNM-Au8 will continue to observe safety, survival, and clinical worsening as well as levels of key biomarkers related to disease progression with the treatment.

In a recent interview with NeurologyLive®, Karon S. Ho, PhD, Vice President of Translational Medicine at Cene Nanomedicine, sat down to discuss how the ACT for ALS grant opens opportunities for patients with the disease who don't usually qualify for traditional clinical trials. She talked about the unique aspect of CNM-Au8, and its potential to benefit patients with ALS. In addition, Ho spoke about how the expanded access protocol aims to include a wide range of patients with ALS and the type of data that it will collect.

REFERENCES
1. National Institutes of Health Awards $45.1 Million NINDS Grant for CNM-Au8® Study in ALS. News Release. Clene Nanomedicine. Published October 5, 2023. Accessed November 6, 2023. https://invest.clene.com/news-releases/news-release-details/national-institutes-health-awards-451-million-ninds-grant-cnm