With momentum today in the lab and in the clinic, now may be the time for real outcomes in the effort to alter the
progression of ALS. That was the message from the panel of experts during the afternoon Leadership Panel at the 7th
annual Leadership Summit organized by the ALS Therapy Development Institute. The first year of such a panel included
sparkling debate, quality analogies, and clear advice for the nonprofit organization focused on developing effective
treatments for patients today. Guest speakers and panelists included: Henri Termeer, former President & CEO of Genzyme
Corporation; Dr. George Scangos, CEO of Biogen Idec; Dr. Christopher Austin, Science Director of the NIH National
Center for Translational Therapeutics; Douglas Onsi, Managing Director of Healthcare Ventures; Robert Blum, President
& CEO of Cytokinetics; Dr. Steve Perrin, CEO & CSO of ALS TDI. The panel was moderated by Dr. Myles Axton, Editor of
Nature Genetics magazine, who's
editorial review of the day appeared in the November issue of that publication. The day's events concluded with
the presentation of the ALS TDI Leadership Awards.
Click Here to View the 2011 Research Symposium
During the 6th Annual Leadership Summit, Fernando Vieira, M.D. kicked off the event covering a myriad of topics, including
recent advancements in turning knowledge about TDP43 (a gene associated with about 1% of all cases of ALS) into a tool that
can be used in the effort to identify and validate potential treatments for the progressive neurodegenerative disease. Following
Dr. Vieira to the microphone was Merit Cudkowicz, M.D., who is the director of the ALS clinic at MGH and the co-founder and
co-director of the Northeast ALS Consortium (NEALS). Dr. Cudkowicz provided a lengthy update on nearly a dozen completed,
ongoing and pending clinical trials for ALS as well as an in-depth description of the clinical trial process.
After a lunch break, Gilmore O'Neill, M.D. of Biogen Idec, provided the group with an intriguing scientific rationale for
the need of researchers to focus as early as possible on integrating biomarker development into their therapeutic research. Up
next was Clive Svendsen, Ph.D. the director of the regenerative medicine institute at Cedars Sinai Medical Center in Los Angles.
Dr. Svendsen walked through all the different approaches to using stem cells as both research tools to model disease as well as
potential therapeutic agents. Finally, Steve Perrin, Ph.D., ALS TDI's CEO and CSO, gave the final talk of the day by showing
data from about a dozen completed or on-going preclinical screens at ALS TDI, including early signs of success with a new drug
terms ALS TDI 00903. A panel discussion followed during which the guest speakers, joined by ALS TDI's Dr. John Lincecum, talked
questions ranging from what causes ALS to what the future or research looks like.
The final portion of the event was the Leadership Award Ceremony, during which four annual awards were presented.
Click Here to View the 2010 Research Symposium
ALS TDI's 5th Annual Leadership Summit was a huge success, with more than 60 patients and supporters touring our lab on Sunday, October 4th during our
Open House, and twice that many for the Monday, October 5th Research Symposium.
Monday's morning session began with a dedication to ALS TDI's late President, Sean Forrester Scott, who was instrumental in making ALS TDI who it
is today - a leader in ALS research. ALS TDI's CEO and CSO, Steve Perrin, welcomed everyone in the room and on the webcast before introducing Dr. John
McCarty to discuss the Biology and History of ALS. The next few hours we opened the session up to guest researchers, Jude Sumulski ("Developing Viral
Vectors for ALS Gene Therapy"), Zuoshang Xu ("RNAi Therapy for ALS"), and Erika Holzbaur ("Understanding the unique biology of the motor neuron: why
we need to know more"). Our guest speakers also took part in a panel discussion along with ALS TDI researchers to answer any questions from guests
in the room and via webcast.
The day ended on a powerful note with the Leadership Summit awards being presented by past winners to the 2009 winners.
The 2008 Leadership Summit featured Lab Tours on Sunday, October 19 and a
Research Symposium on Monday, October 20. The morning session included talks from both internal researchers
and guest researchers including: Sharon Hesterlee, Ph.D. (VP Translational Research Muscular Dystrophy Association),
Michael J. Strong, M.D. (Co-Chair, Department of Clinical Neurological Sciences, Arthur J. Hudson Chair in ALS Research,
London Health Sciences Centre ), Jean-Pierre Julien, Ph.D. (Professor Laval University), G. Thomas Caltagirone, Ph.D.
(President & CEO, Aptagen, LLC), and Stanley H. Appel, M.D. (Co-Founder & Co-Director The Methodist Neurological
Institute, The Methodist Hospital).
The afternoon session included a lively panel discussion among researchers and members of the
ALS Community including patients and caregivers. Troy Jurgenson, Co-Founder of Speak Up ALS, presented on a eye-controlled
communication system for ALS patients followed by Steve Perrin, Ph.D. Chief Executive Officer & CSO of ALS TDI who gave an update
on the genomic and proteomic approach to ALS research. The event concluded with the annual Leadership Awards given to ALS
Research advocates and a demonstration session from DynaVox showcasing the EyeMax communication device. For patients and
family members who were unable to attend, the afternoon session was available as a webcast.
Last year's Summit created some of the most lively discussion yet and one of the most unique opportunities for patients
and researchers to have an interactive discussion about approaches taken to discover a treatment or cure for ALS.
The two-day event featured several labs tours given to nearly 100 people followed by a major research symposium held at the
Museum of Science (MOS) in Boston. At the MOS, Dr. Steve Perrin of ALS TDI provided an overview of the power of mapping the
genetic and protein interactions in the disease for creating a list of potential drug targets.
Dr. Raymond Onders (The Cleveland Clinic) provided information on his diaphragm pacing surgery. Dr. Dale Lange (Mt. Sinai
Hospital) provided a presentation on a clinical trial he conducted on a potential therapeutic and Dr. Hans Kierstead
(California Stem Cell, Inc.) gave a lecture on the potential for stem cell usage in the effort to bring about an
effective treatment for ALS.
After a successful start, the Leadership Summit grew out its space at the Massachusetts Biotechnology Council and moved to the
Hotel @ MIT. In June 2006, ALS patients, families and friends gathered in Cambridge, Mass., for ALS TDI's Annual Leadership
Summit. The two-day conference was packed with lab tours and research presentations, plus plenty of socializing.
The second summit featured presentations from John Lincecum, Ph.D. (ALS TDI), Robert Bowser, Ph.D. (University of Pittsburgh)
and Dan Benjamin, Ph.D. (ALS GEN, Inc.).
The Summit helped scientists put a face on the disease and helped patients and families get to know the people behind the
research studies. A special screening of "So Much So Fast" as well as an exciting Awards Dinner followed the
Summit which created memorable highlights for all in attendance. Read More in the ALS TDI Newsletter here.
The inaugural Leadership Summit was held in the fall of
2005. The summit included an Open House and tours of ALS TDI followed by seminars held in the executive offices of the Mass
Biotechnology Council (MBC) in Cambridge overlooking the Charles River. Several ALS patients participated in this first-time
event which included an early look at the Apollo Projects, which has since been both expanded and accelerated due to a new
partnership with MDA Augie's Quest. A round-table discussion of how best to integrate the newest ALS TDI fundraising
programs, Driving 4 Life and the Tri-State Trek, provided new ideas and excitement. A traditional New England lobster dinner
was hosted by John & Peggy Heywood in Newton where the first
Leadership Awards were presented.