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 advise for the nonprofit organization focused on developing effective treatments for patients today. The entire two-hour "Leadership Panel" discussion was recorded and is available to view online at www.ALS.net/summit.
“Do what you are doing, enhance it and make it sustainable from a funding point of view. I think you will get a true effect on this space at this point because I think that at this point this space is particularly conducive for real progress,” said Henri Termeer, former chairman, president and chief executive of Genzyme Corporation. Genzyme has developed several therapeutics for rare and orphan diseases. Termeer, pictured to right at top, stepped down recently after Sanofi purchased the 30 year old biotechnology company.
During a period of questions from the audience, a caregiver for a PALS named Bob who can no longer speak due to the progression of the disease, asked CEO of Biogen Idec, Dr. George Scangos, why his company limited enrollment into their Phase III Clinical Trial "Empower" to people diagnosed with ALS relatively recently. The caregiver relayed that Bob felt that he was being "punished" for surviving longer than others diagnosed with the disease. Scangos, pictured to the right in the middle, empathized, saying that it was a very difficult decision for Biogen when it came to determining the trial design. He reported that after a review of the process and timeline, that it would be faster to determine efficacy if they continued with the design that had been used by Knopp Neurosciences and the NEALS Consortium during the Phase II trial of the experimental treatment. Biogen licensed the molecule from Knopp earlier this year and enrolled more than 800 PALS in less than 4 months into the trial. PALS within 24 months of overt symptom onset were targeted for enrollment, which is the same criteria used in the Phase I and Phase II clinical trials by the University of Virginia and Knopp prior to Biogen licensing the compound from that company. He commented that Biogen is committed to the ALS community and walked through their disease focused pipeline, which includes a soon to begin enrolling FDA-required second Phase III trial of dexpramipexole (Endeavor) as well as several other promising leads in the biotech-leader's preclinical pipeline.
Representing the venture capital and venture philanthropy worlds, Douglas Onsi, Managing Director of Healthcare Ventures provided clear advice to the more than 200 advocates, patients and caregivers in the room at the Hotel Marlowe: you have the power to influence investment. He encouraged audience members to advocate to groups and projects they supported and to never back down. Onsi, picture to the right at the bottom, commented that venture firms often rely on experts at TDI and similar organizations for advice and information about fields they are interested in investing it. In addition to helping to create new companies through his venture work, Onsi is a member of the MS Society's Fast Forward board of directors, where he helps to raise capital and make strategy investments in promising research for new treatments for multiple sclerosis. "You all are the real venture capitals," said Onsi to the audience of mostly PALS and CALS.
During the closing remarks, Dr. Christopher Austin, Science Director of the NIH National Center for Translational Therapeutics, suggested that in these financially challenging times, where there is momentum everywhere but little cash, all the players need to come together. "It's like this, when a slime mold runs out of food it groups together with other slime molds around it to create a totally new entity in order to survive and grow. We need to all start doing the slime mold."
Other participants included Dr. Steve Perrin, CEO/CSO of ALS TDI, as well as Robert Blum, President & CEO of Cytokinetics, which recently announced the start of Phase IIb enrollment for its own clinical trial in ALS with a drug, CK-506 (link to clinical trial page), targeting skeletal muscle.
"ALS TDI is a highly efficacious effort. Why tweak this? I am extraordinary impressed," said Termeer reflecting on what advice to give ALS TDI. He closed in reiterating that he believes that the time may be right and momentum is there. "I have learned so much today that I did not know before."
To facilitate the continuation of that momentum, the panel's moderator, Dr. Myles Axton, is planning on publishing an editorial review of the advice received and outcomes from the panel in Nature Genetics later this month. To view a recording of the panel discussion, click here.