NEW ORLEANS (By Team Gleason) – June 26, 2014 - When Team Gleason hosted an international ALS Summit 1 year ago, Steve challenged the audience of leading researchers, patients, caregivers and advocates to “think differently”. The primary focus of our unique meeting was collaboration and the results are not only a reflection of that during the Summit, but also as an ongoing effort long after.
Given the successful collaboration among those impacted by ALS and researchers at the Summit, Team Gleason is launching Answer ALS to activate the findings from the Summit. Answer ALS is not another organization, but rather an initiative, led by a Council of representatives to be chosen from ALS stakeholders, pharma, research and individuals. The Council will oversee the implementation of the Summit findings.
Two key elements were identified in the Summit outcomes:
*A united effort for people with ALS to be part of the process in addressing ALS now and for the future.
*The Individualized ALS Treatment Initiative
To address the need for a united effort, a collective group of people with ALS, also known as pALS, caregivers, and advocates were asked to draft a document outlining their desired outcomes based on what was discussed at the Summit. A 17 page document was created covering details ranging from immediate public policy actions to data collection and from technology to care and more. One focus of the pALS document is to create a new and open method of collecting, integrating and linking ALS patient information. Having this data source as a tool not only empowers those impacted by ALS, but also assists the research community in many areas, including our second and most aggressive focus, the Individualized ALS Treatment Initiative.
The Summit resulted in a transformational approach to ALS Research and patient interventions by bridging basic to clinical neuroscience. With our focus on collaboration, we will utilize the proven model of the Robert Packard Center for ALS Research to create an entirely new and expansive ALS therapeutics program through induced pluripotent stem cells or iPS cells. Research teams will be combined with pharmaceutical companies in a carefully coordinated fashion. This unique human cellular approach could allow rapid and individualized ALS therapy discovery. This Individualized ALS Treatment Initiative will bring together experts in iPS cells, clinicians, pharmaceutical companies, experienced drug discovery teams, all operating under one umbrella to coordinate and facilitate rapid individualized discovery, while completely sharing all data.
“While all that sounds very complex and if you know me, you know I don’t typically speak on those terms.” Said Paul Varisco, Executive Director of Team Gleason. “But, what all that means is actually quite simple. We, and by we, I mean scientists have the ability to take human SKIN cells and turn them into STEM cells,” he added. “Those stem cells can then be turned into neurons and treated as the individual patient in a dish, allowing testing to be done on the largest scale in the history of the disease and much more quickly and widespread.”
Now that the Summit results are complete, Team Gleason’s next steps are to connect with ALS stakeholders, individuals and organizations to create the Council to begin the first phases of this effort. Additionally, the goal is to raise the funds needed through private donors rather than creating a campaign. Groups and organizations fighting this disease are already competing for sustainable funds. Our hope is to not negatively impact their ongoing efforts, but rather to compliment them with our collective actions.
Answer ALS is a tremendous undertaking and our goal is to secure a minimum of $125 million annually for 5 years. We believe that through a massive assault on this disease, we can have solutions in the foreseeable future. “Tens of thousands of people die every year from ALS. That is not ok.” Said Steve Gleason. “If we are to change the course of this disease, we must be bigger than it is. The price tag for this effort will be over $500million dollars,” Steve continued. “But, we believe that if we commit to this effort, on this scale, we will have meaningful results in our lifetime.”
“We couldn’t be more proud to join in this effort,” said Jeff Rothstein, Co-Chair of the Team Gleason Summit and Director of the Robert Packard Center for ALS Research at Johns Hopkins. “Answer ALS is exactly the type of collaborative effort we encourage and demand from our research partners.” Rothstein added. “Through iPS therapeutics and on a large enough scale, we have the ability to move the needle on this disease faster and farther.”