1. Research networks in movement disorders Joaquim Ferreira, MD, PhD Instituto de Medicina Molecular Faculdade de Medicina de Lisboa

2. Clinical Research (experimental medicine) NIH Definition 1. patient-oriented research: research conducted with human subjects (or on material of human origin such as tissues, specimens and cognitive phenomena) for which an investigator directly interacts with human subjects. Includes: (a) mechanisms of human disease (b) therapeutic interventions (c) clinical trials (d) development of new technologies 2. epidemiologic and behavioral studies 3. outcomes and health services research conducted physician-scientists vs. basic/fundamental research ≠ (but includes) pharmacological research or clinical trials

3. “the whole animal” cellsgenes manageability productivity Evolution in biomedical research 1950 David G. Nathan, JAMA 2008 Claude Lenfant, NEJM 2003 1960197019801990 Clinical research “crisis” 19301940 NIHNINDS Clinical Center for Medical Research

4. David G. Nathan, JAMA 1998 Report of the Clinical Research Subcommittee of the Scientific Issues Committee of the AAN.Neurology, Apr 1995 NIH applications by degree Clinical research “crisis” 1995

5. Transnational research cooperation Coordination of public and charitable funding for non-commercial clinical trials Research focussing on rare diseases, orphan drugs and European-health needs Pan-European clinical trials (large samples, easy recruitment, reliable estimates of effect, pragmatic) European Society of Cardiology, Lancet 2002 Clinical research in Europe: needs European Society of Cardiology

6. Parkinson´s Disease

8. Cochrane Collaboration

9. Symptomatic treatment

10. PD progression Unilateral Dementia Motor complications & dyskinesias Bilateral Psychosis Instability & falls Disability Time PreclinicalSigns and symptomsProdromal -4–6 yrs Ferreira JJ

11. Disease modifying

13. Late stage

14. Huntington’s Disease

15. rare degenerative brain disorder genetic cause identified in 1993 30,000 people effected in Europe 150,000 at risk for inheriting the illness Huntington's disease Total functional capacity How many patients are needed for “neuroprotective trials”? Marder K, Neurology 2000

17. 18 Countries > 130 study sites ru The European HD Network (EHDN)

18. REGISTRY

19. Enrollment in REGISTRY 6133 participants 14873 visits

20. PARTICIPANTES de CENTROS PORTUGUESES (2006-2009) 116 85 50 25 Portugal. 2009 REGISTRY-HD REGISTRY-HD

21. Research networks in movement disorders Joaquim Ferreira, MD, PhD Instituto de Medicina Molecular Faculdade de Medicina de Lisboa