ALS Emergency Treatment Fund thanks you for your perseverance and support in 2015.  We are honored to lead the work for wide access to exploratory medicine in ALS and other life-threatening conditions.

Your year-end contribution will help us achieve a better treatment landscape for patients and doctors everywhere.

Thank You! 

Meaningful expanded access programs are multi-site, data-generating clinical trials of unapproved drugs that are in development for the particular disease, and they are conducted for the purpose of including patients and clinics who cannot take part in the drug's traditional research trials. Though expanded access is primarily for "treatment use" as opposed to research use, expanded access trials can be designed to yield valuable endpoint data and patient profiles that can support the drug's ongoing clinical research and may increase the likelihood of a swift market approval.

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ALS-ETF was launched in 2012 to serve as a third-party sponsor of expanded access programs.   We do it ourselves, because (1) as specialists, we know the current regulations and historical best practices better than any drug company, (2) We have a growing network of renowned ALS clinics who know their patients and who want to provide the option of exploratory treatment, and (3) these programs serve a humanitarian goal that is not the responsibility of shareholder-owned drug companies, especially the small companies bringing new medicines through Phase 2 trials in neurodegenerative diseases.

http://alsetf.org/access/

ALS-ETF spent much of 2015 preparing to launch a multi-site Expanded Access trial for the investigational drug NP-001, a proprietary immunomodulation drug owned by Neuraltus Pharmaceuticals.   As a potential ALS treatment, the product is generally well regarded by the key opinion leaders in the disease community.  By 2012, clinical studies of this drug had generated enough data to make it eligible for Expanded Access in the U.S.

 

Our charitable partners have committed the financial support necessary for ALS-ETF to sponsor and manage an NP-001 Expanded Access program for at least two years, allowing our collaborative network to cover all tasks, including regulatory filings, site selection and monitoring, pharmacovigilance, data collection, as well as funding for product delivery and treatment.  It will begin with up to 100 patients across the country, and may grow to much larger numbers depending on additional resources. However, we cannot launch without the permission of the drug company, and we hope Neuraltus will give the green light soon, in a partnership that allows doctors and patients to receive their potentially effective product through our platform.

2015 was a huge year for ALS-ETF! Founder and Executive Director Jess Rabourn delivered presentations on Expanded Access at six public conferences this year and contributed to several collaborations for better drug access in ALS and other disease areas. In 2016, we will be at the annual JP Morgan / Biotech Showcase events in January, then speaking in the Spring at conferences held by CBI, Kauffman Foundation, and Marcus Evans.

As a result of this added visibility and leadership, we have a growing pipeline of drug companies who now want to integrate Expanded Access into their clinical development strategies, and we'll be there to help.