Wednesday, January 27, 2010

Notes from the International ALS/MND Symposium in Berlin

In December 2009, Prize4Life’s Scientific Program Officer, Dr. A. Sheila Menzies, was among the 900 ALS researchers, neurologists, clinicians, and caretakers who convened in Berlin for the 20th Annual Symposium on MND/ALS. The Symposium spanned three days and featured simultaneous sessions on ALS biomedical and clinical research.

The Symposium’s abstract book is available here. In addition to being a symposium guide, the booklet is a map of current ALS research, detailing the research landscape at the end of 2009.

Sessions covered a wide variety of relevant topics, ranging from discussions of proteinopathies to biomarkers to axonal transport. The proteinopathies session emphasized how the aggregation of mutated proteins in ALS patients is characteristic of other neurodegenerative diseases as (such as Alzheimer’s), suggesting a common disease mechanism (this topic was also recently covered in depth in a webinar organized by the Alzheimer Research Forum; find it here). A session on functional genomics explored the range of symptoms that may occur in ALS patients due to mistakes in the genetic code. A session on trial design showcased results of recent clinical trials, in the context of methodology, and encouraging trends in Knopp Neurosciences’ KNS-760704 Phase 2 clinical trial for ALS (read more on Knopp here and on ALS clinical trials here). The Biomarkers session predominantly featured discussions of protein-based ALS biomarkers.

A poster session saw several hundred people informally discussing their work with colleagues. Posters covered therapeutic strategies, human cell biology, epidemiology, and diagnostics, among other topics.

The clinical end of the spectrum hosted sessions on spiritual care, translating research discoveries into relevant care practices, information and conversations on exercise, metabolism, and nutrition, and a session on disease progression worked to increase audience understanding of current research focused on how to accurately portray expectations in terms of disease progression and life expectancy. A session on cognitive change explored the relationship between frontotemporal dementia and ALS, as well as covering cognitive assessments. A session on emerging disease models discussed options for identifying causes and developing therapies (see a related piece on the growing number of TDP43-based animal models here). The session on motor neuron biology provided in-depth conversation about the growth, development, and functioning of motor neurons themselves.

For more information on the sessions, see notes from the MND Alliance and ALS-TDI’s brief overview. Tune in to for more in-depth scientific coverage of this and other meetings in the future

Professor Michael Strong, from the University of Western Ontario, is quoted on the MND Alliance’s site saying “The science has been very good this year. We are starting to see a real coming together of research work. No longer as research teams are we working in isolation but instead we are looking at ‘families’ of research ideas.”

Prize4Life was pleased to be present for these sessions, and for the opportunities to network and converse. We were also inducted into the International Alliance of MND/ALS Associations, at the annual Alliance meeting hosted by the Deutsche Gesellschaft fur Muskelkranke, held the week before the Symposium. Prize4Life joined the Alliance as an associate member, and is excited to be able to connect with such a respected body of organizations, exchanging information and participating in support fora with other ALS organizations worldwide.

Thursday, January 14, 2010

Networks & collaborations: the great R&D facelift

As 2009, an economically brutal year, drew to a close, there was a new buzz about the future of pharmaceutical R&D. The Wall St. Journal, the New York Times, and the Boston Globe have all been commenting on it. Several weeks ago, Prize4Life attended the Partnering for Cures conference in New York City, which also emphasized this trend: increasingly, drug development is experiencing a move towards a more networked, collaborative approach to R&D.

Jonathan Rockoff reports in his piece for the Wall St. Journal that Eli Lilly & Co, like most other pharmaceutical companies, is trying to make its drug-development processes more productive while coping with both a recession and leaner pipelines. Lilly’s new approach mirrors that of other companies right now: they are hiring outside contractors to test drug candidates.

Pharma and biotech outfits have traditionally kept drugs under development close to their chests and conducted most R&D in-house, fearing intellectual infringement or losing a blockbuster to competitors. However, a large batch of patents on blockbuster drugs are expiring or will soon expire. With no obvious successors lined up, the biggest drug makers are "externalizing" much of their R&D, said Peter Tollman of Boston Consulting Group, a frequent adviser to drug companies.

By way of an example, Lilly is considering moving a molecule into Phase 3 testing that was developed by scientists outside its own research team. The potential market for the molecule under development is $16 billion annually. In the past, a major pharmaceutical company like Eli Lilly would have been far less likely to entrust any aspect of the development of such a potentially valuable compound to an outside team, and relying entirely upon outside scientists for the development of such a compound represents a significant change in strategy.

“By outsourcing human tests of such a potentially important drug, Lilly is among a crowd of pharmaceutical giants adopting out-of-the-box strategies to revive fallow research-and-development organizations,” Rockoff writes.

Sanofi, in another R&D ‘makeover’, announced that it intends to strengthen 'exploratory structures' that work in close collaboration with outside entities, reports a piece in FierceBiotech. It will pursue partnerships with public and private research entities, academic institutions and biotechnology companies.

"We are living through radical times of change for Research," said Dr. Marc Cluzel, Senior Vice President of R&D at Sanofi in the FierceBiotech release. “Tomorrow's research will be carried out through networks. We will be open to knowledge from outside sources, becoming a key partner. We need to reinvent R&D."

"Trying to do everything yourself won't work. The odds of owning everything and being at the right place at the right time are not high," echoed Peter Johnson, Lilly's vice president of corporate strategic planning.

A major driver of this shift is, presumably, cost. Lilly wants its contract research organizations (CROs) to reduce development costs and risks by quickly identifying things that won't work. Other companies such as Sanofi are leaving early-stage R&D almost entirely to its biotech partners, reports the WSJ piece. This approach enables companies to manage a much larger portfolio than they would if they were doing R&D in-house.

The Association of Clinical Research Organizations (ACRO) estimates that there are more than 1,000 CROs with greater than $20 billion in yearly revenue, staffed primarily by former drug-industry researchers. At its best, such a partnership enables both the contracted party and the contractor to work within their areas of strength.

In extreme cases, we’ll see rival companies such as Pfizer and GlaxoSmithKline combining HIV-drug businesses into a single company, figuring, figuratively, that two heads may be better than one.

Even the NIH is on board with the new emphasis on collaboration—it recently put up $27 million to see if social networking technology can virally link a generation of biomedical researchers, reports another article. The University of Florida and Harvard University Medical School received the grant money to establish sites to share ideas and data. The NIH-funded efforts will come in addition to the growing scientific social network site ResearchGate, which is rapidly becoming a well-established professional exchange platform for researchers around the world.

"National networking provides opportunities for scientists to collaborate in new, exciting ways to improve abilities to uncover underlying pathways and mechanisms of biology and to develop new diagnostics, treatments and prevention strategies," said NCRR Director Barbara Alving, M.D. "The infrastructure created and implemented through these awards has the potential to greatly facilitate the pace of biomedical research nationwide."

Prize4Life encountered the same trends—networking, outsourcing, collaborating—at the Partnering for Cures conference, a meeting of philanthropic organizations, medical research foundations, and the biotechnology and pharmaceutical industries intended to foster collaborations, held in the first week of December, 2009.

Speakers at the conference emphasized that non-profit organizations and disease foundations are well-positioned to assume risk. Indeed, these collaborations will be critical for reducing drug development costs, and finding a cure for orphan and infectious diseases. Panelists argued that companies will be more likely to invest in newer technologies if not-for-profit counterparts ‘de-risked’ them.

Further, non-profit foundations are in a unique position to facilitate relationships among stakeholders. Non-profits and disease foundations have the time and the mandate to identify relevant actors across sectors and to facilitate meetings and collaborations that are technically outside the purview of company operations.

In a conversation with Michael Milken, Jeff Kindler (CEO, Pfizer) spoke extensively about how partnerships between pharmaceutical companies and smaller biotechs, foundations, and disease organizations will be necessary in order to both survive and thrive in a changing global economy.

Competitive medical research has been internationalized in the last ten years, with important developments coming out of Asia and other regions. Emerging foreign research institutions are enticing skilled researchers to leave the U.S., and U.S. pharmaceutical companies are faced with new competition. Milken emphasized just how much ground the U.S. has lost in the global economy of late, noting that the U.S. approach to drug discovery and innovation needs to reflect the changes that are occurring worldwide.

Kindler also noted that the primary barrier to getting new treatments on the market is lack of clinical trials participants, and suggested that this is where disease organizations can be especially helpful. This is certainly the case in ALS, where a paper published in 2008 reported that the percent enrollment in ALS clinical trials is 25%, highly variable, and has not been improving over time; Gina Kolata, in a piece in the New York Times, wrote extensively about this issue in the context of the fight against cancer. Organizations such as the ALS Association have sites that offer information on clinical trials, as does the government site,

Due to a combination of economic and political factors, it seems that all aspects of the research establishment are indeed beginning to look differently upon collaborations. Let us hope that this exciting trend bears fruit.

by Meghan Kallman