psychiatry, and chronic pain. Various participants mentioned the many biotech and medium-sized companies working on neurodegenerative diseases. Don Cleveland of the University of California, San Diego, described therapeutic approaches that target gene products known to be truly causative of disease, such as the treatments based on antisense oligonucleotides presented by Frank Rigo of ISIS Pharmaceuticals, as “at least one ray of real optimism” in therapies for neurodegenerative disease. Participants went on to discuss various ways to encourage therapeutics development for neurodegenerative diseases. For example, they noted that small and medium biotech companies may do innovative science and then partner with or be absorbed by larger companies. Several participants discussed how National Institutes of Health funding could help support Phase II clinical trials in small companies, as well as models of collaboration between academic investigators and companies.
In thinking about promising strategies going forward, several participants discussed the balance between going after a single receptor or single transcription factor versus embracing complexity. One participant noted that although the intrinsic complexity may be concerning to large pharmaceutical companies and a single focus may provide the appealing promise of clarity, the biological phenomena themselves may require a systems approach. Similarly, a number of participants highlighted the difficult balance between pursuing a diversified strategy and the reality of finite budgets. Lennart Mucke of the Gladstone Institutes and the University of California, San Francisco, noted that with personalized medicine, the days of pursuing one blockbuster drug are coming to an end. He mentioned the example of Herceptin and said, “As we understand patient population heterogeneity better, we will see that kind of personalized medicine approach expand. The pockets of investment will be smaller.” He went on to say, “We need to diversify our strategy in drug development. We need to be prepared for the possibility that most sporadic neurodegenerative disorders have a multifactorial etiology and will require a multipronged therapeutic approach.”
Some participants highlighted large collaborative efforts, such as the Alzheimer’s Disease Neuroimaging Initiative (ADNI) and Parkinson’s Progression Markers Initiative (PPMI). John Trojanowski of the University of Pennsylvania called ADNI a great success story, encompassing public– private partnership, data made publicly available, and excellent use of funding in a strategic way during difficult financial times. He asked, “Why can’t we do this again and again” for different diseases and biomarkers? Lucie Bruijn of the ALS Association encouraged this suggestion, and the group discussed differences in the levels of knowledge among the different diseases and how this might impact the appropriate timing for launching large collaborations.