Conference in New York provides perfect venue for CEO/CSO of ALS TDI to describe new way to think and enact research programs for orphan diseases such as ALS (Lou Gehrig's disease)
There is no shortage of non-profit organizations in the United States. Many of them vying for the limited resources available from the traditional funding sources which exist at the local, state and federal government level. All of them tailoring their mission statement to communicate their specific goals, in order to fit into the categories of funding from major medical research foundations. This traditional approach has achieved many great things for patients living with diseases all over the world and is one that has worked for years, particularly in providing patient care and services. However, as demonstrated by the 50 rare and orphan disease groups that presented at the Partnering for Cures meeting, which took place in New York December 2-4, the current system isn't designed to meet the needs of all organizations. Partnering for Cures, organized by the non-profit FasterCures, highlighted the issues most important to both established and emerging non-profits working to meet the dire needs of their constituents: therapeutics that slow, arrest or even reverse disease progression.
On December 3rd, FasterCures' Executive Director Margaret Anderson laid out the challenges faced by non-profit research and advocacy foundations, such as ALS TDI, during the morning panel discussion. Billy Tauzin, of the trade organization PhRMA, joined the panel to discuss the issues that organizations working on rare and orphan disease face as they bring drugs to the FDA for potential clinical trial, and the public policy challenges that may need to be overcome in order to meet the needs of so many different disease populations. Joining him in the panel discussion, both Robert Beall (CF Foundation) and Kathy Giusti (MMRF) commented directly on the potential for policy and regulation to be amended for today's dramatically changed research landscape. The nearly 700 attendees, representing dozens of disease foundations, pharmaceutical companies, philanthropic advisory and government agencies, shared an interest in learning from each other how to best accelerate research and, ultimately, cures for rare and orphan diseases. A video of the session is available via this link: http://www.partneringforcures.org/descriptions/philanthropy.html
The assembly of CEOs, Executive Directors and other top management was then divided for the first of the breakout sessions and partnering meetings. Dr. Steven Perrin, CEO and CSO of ALS TDI, was a panelist during this first series of open discussions titled ''Cure Entrepreneurs''. During this panel, Perrin discussed the challenges and opportunities which are faced by a ''brick and mortar'' non-profit research Institute. His advice to the room was twofold: keep your mission statement clear and concise and speak directly to your patient population. Led by Wall Street Journal staff reporter, Amy Dockser Marcus, Steve was joined on the panel by Rusty Bromley (COO of the Myelin Repair Foundation – an ALS TDI collaborator), Susan Kelley, (Chief Medical Officer of the Multiple Myeloma Research Consortium) and Jana Armstrong (Executive Director of the Drugs for Neglected Diseases iNitiative).
''Every Tuesday before we start our New Leads meeting, I repeat our mission statement: to bring forth effective therapeutics for today's ALS patients. I then tell everyone in the room that if they aren't there in the room to accomplish that goal - ''There's the door''. Our professional team can walk into any one of the biotechs in Kendall Square and get a job doing science. And if they are not here to get this done for patients today than they shouldn't be here,'' said Perrin during his opening remarks.
The lunch-time discussion featured the founder of FasterCures and the Milken Institute, Michael Milken and the Chairman and CEO of Pfizer, Jeffrey Kindler. Pfizer was one of the dozen sponsors of the event, many of which were many major pharmaceutical and biotech companies. One of the discussion segments between Milken and Kindler was centered on identifying potential barriers for some diseases in recruiting for clinical trial. The mutual feeling was that there may potentially be actionable modifications to the clinical trial process, specifically within recruitment, surrounding the importance of privacy. A video of this discussion is available online via this link: http://www.partneringforcures.org/descriptions/lunch120209.html. A panel on the second day of the event titled ''Patients 2.0'' directly addressed privacy concerns and expanded the discussion on clinical trial design. ALS TDI and PatientsLikeme co-founder, James Heywood, was a featured speaker as part of that session.
Throughout the two day event, ALS TDI staff spoke and held private partnering sessions with nearly two dozen potential collaborators from both large and small biotechs, philanthropic organizations and others interested in accelerating cures for debilitating diseases such as ALS. In addition, the Institute's leadership met privately with several emerging non-profit research organizations interested in learning more about how ALS TDI built the infrastructure it has today which allows it to prioritize and robustly test several dozen therapeutics each year.
''Before I came to this meeting I looked at all of the 990s available for the other organizations attending. I plotted their spending on a graph depicting overall and research spending. ALS TDI is just below the fourth quartile. While we have accomplished a lot, it became clear, when doing this comparison, that in addition to a willingness to take a risk to build a new system for drug development, there is an monetary inflection point that a non-profit needs to reach on an annual basis in order to do good research and take several well qualified ''shot on net.'' That point is somewhere in the $15 million range,'' said Perrin during his afternoon corporate presentation.
The second day featured several additional panels and discussions, including one led by Jim Greenwood of the Biotechnology Industry Organization entitled ''Breakthrough Science''. A video of that plenary session is available online here: http://www.partneringforcures.org/descriptions/breakthrough.html The midday plenary session on December 4th featured a conversation between the Josh Sharfstein, the Principal Deputy Commissioner of the FDA, and Gail Cassell, the VP of Scientific Affairs of the Eli-Lily company. The discussion and debate was facilitated by Mike Milken and focused on encouraging a serious and open discussion of the clinical and the approval process. The panelists all agreed on several items that are often of direct interest to ALS patients and families. For instance, the speakers spoke directly about the need for additional resources at the FDA, not just in funding but specifically in expanded knowledge of research and production. Furthermore, the speakers advised all of the diseases represented by leaders in attendance to work early and closely with the FDA. For a video of the full conversation, click on this link: http://www.partneringforcures.org/descriptions/lunch120309.html
ALS TDI is a proud member of the TRAIN Network at FasterCures. For pictures from Steve's presentations and more information about the meetings and the organization behind it, please visit http://www.partneringforcures.org