Categories: Meeting Report

Momentum there for ALS Treatments According to Panel at TDI Summit

clock November 13, 2011

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.

Henri TermeerHenri Termeer

George ScangosGeorge Scangos

Douglass OnsiDouglass Onsi

“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

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Categories: Meeting Report

Highlights from the 3rd Annual Partnering for Cures, a FasterCures Meeting

clock November 13, 2011

Priming the Pump: Filling the Therapeutic Pipeline

Earlier this month, the 3rd annual Partnering for Cures conference was held in New York City. This event, which began in 2009 is organized by FasterCures, a “action tank” that works to improve the medical research system in order to speed up the time it takes to get important new medicines from discovery to patients. ALS TDI is a member of the TRAIN program at FasterCures, which is a subsidiary of the Milken Institute.

To being the conversation, an opening plenary focusing on the convergence of stakeholders in the funding of research. According to conference organizer and FasterCures president and CEO, Margaret Anderson, the FDA approved 35 new therapeutic in 2010; up from the 22 it approved a year earlier. She summed up this growth due to their being strong science, industry leadership and regulatory flexibility. However, Anderson summarized the current pipeline outlook saying, “There is a pipeline issue - they can only review and approve what comes before them. The reason for high approval rate was that these products were in the pipeline years ago. Today, the pipeline isn't as robust. The challenge may be in having too many opportunities but no way to choose or implement or prioritize them.”  An excited group of panelists, which included FDA Commissioner Margaret Hamburg, spent the next 90 minutes tackling this topic.

John Mendlien, who is at the helm of several small biotechnology companies, suggested that the focus needs to be on saving time - not necessary about money. To this point, Susan Desmond Hellmann, chancellor of the University of California at San Diego, added that the field needs biomarkers of disease and drug response desperately and the creation of animal models are areas where academics can lead the way. She also reflected that the cost of doing science has lessened as innovation has increased; using the example of the cost of sequencing a person’s genome, dropping to about $2,000 per person from the $95 million it was not too long ago. “It will soon cost less than an MRI, likely within the next year,” said Desmond-Hellmann. This comment led to a lively exchange between several of the panelists, including Mendlein who asked rhetorically and excitedly what the potential opportunities would exist for therapeutic development if every person in the US had their genome sequenced. Responding to this, at the behest of Anderson, Garry Neil, of Johnson & Johnson, interjected that while having data is great it needs to be collected and used in a meaningful way and the collection of such, if done by a private corporation, would provide them a competitive advantage.

Representing the patient advocacy perspective on the panel was Jeff Brewer, the president and CEO of the Juvenile Diabetes Research Foundation (JDRF). He offered these comments, “Incentives are needed for companies to invest in a specific area of research. Partnerships with companies are key for a non-profit foundation.” Brewer then outlined how JDRF has dozens of direct financial relationships and hundreds of other relationships with biotechnology and pharmaceutical companies. “You need to erode the competition between groups working on the same disease especially, when they fund the same researchers and work,” said Brewer. You can view the entire panel discussion online at FasterCures being clicking here or hitting play on the video above. 

Back to Basics: Patient Activism Thirty Years After HIV/AIDS

Another highlight of the first day was the “Back to Basics” panel organized by Susan Love, which can be viewed in its entirely be viewed at the FasterCures website. Warren Lammert, chairman and co-founder of the Epilepsy Therapy Project, and others were invited to provide advice to emerging non-profits on how best to organize and advocate. Lammert’s advice was that each organization needs to have clear ways to measure their success, otherwise you won’t have an impact. His organization for example has decided to focus on ensuring that the pipeline of potential treatments for epilepsy stays robust and therefore they fund projects that were unable to get funding elsewhere from government or other private sources.

ALS Therapy Development Institute, TDP-43 Mouse Model Partnership

Steve Perrin, president, CEO and CSO of the ALS Therapy Development Institute was also invited by the steering committee of this event to present an overview on the $1 million + collaboration between his organization and three others to charectorize an emerging model of neurodegeneration: the TDP43 mouse. The collaboration included both funding and scientific input from the Alzheimer's Drug Discovery Foundation, the Association for Frontotemporal Degeneration and the Muscular Dystrophy Association. Perrin suggested that such partnerships allow for the speeding of research and in turn eventually treatments by working across diseases at the same time rather than focusing each group to do the exact same experiment which would take longer and cost patients and donors more money.

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