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Barriers to Treatment Chip Wilmot, MD PhD Emory University.

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Presentation on theme: "Barriers to Treatment Chip Wilmot, MD PhD Emory University."— Presentation transcript:

1 Barriers to Treatment Chip Wilmot, MD PhD Emory University

2 Disclosure Member, Data Safety Monitoring Board for studies involving Idebenone (Santhera Pharmaceuticals)

3 “Why Don’t We Have A Treatment for Ataxia Yet?” -anonymous at NAF AMM 2012

4 “Why Don’t We Have A Treatment for Ataxia Yet?” -anonymous at NAF AMM 2012 and FAPG and GAASG and my clinic and …

5 “Why Don’t We Have A Treatment for Ataxia Yet?” -Chip Wilmot, MD, PhD Emory University

6 Treatment the care and management of a patient to combat, ameliorate, or prevent a disease, disorder, or injury.

7 Cure A method or course of medical treatment used to restore health.

8 Cure A method or course of medical treatment used to restore health. Don’t forget about prevention (at least for dominant SCA’s)

9 Treatment -Symptomatic -pain, cramps, depression, mobility -Disease-modifying -slowing the rate of progression

10 “Why Don’t We Have A Treatment for Ataxia Yet?” -Developing a treatment is HARD

11 “Why Don’t We Have A Treatment for Ataxia Yet?” -Developing a treatment is HARD -Expectations for treatments can be unrealistic

12 “Why Don’t We Have A Treatment for Ataxia Yet?” -Developing a treatment is HARD -Expectations for treatments can be unrealistic -We are doing a good job, but …

13 “Why Don’t We Have A Treatment for Ataxia Yet?” -Developing a treatment is HARD -Expectations for treatments can be unrealistic -We are doing a good job, but … - … there are certainly ways to facilitate treating ataxia

14 Clinical Disease Treat Human Disease

15 Clinical Disease Treat Human Disease Etiology (Cause) Model Disease Treat Model

16 Clinical Disease Treat Human Disease Etiology (Cause) Model Disease Treat Model

17 Clinical Disease Treat Human Disease Etiology (Cause) Model Disease Treat Model

18 Clinical Disease Treat Human Disease Etiology (Cause) Model Disease Treat Model

19 Clinical Disease Etiology (Cause) Model Disease Treat Model Treat Human Disease Treat Symptoms Treat Disease

20 What is needed to develop effective treatments? 1.Knowledge of the disease pathophysiology 2.A way to measure the disease 3.An understanding of the natural history of the disease 4.Research Infrastructure

21 What is needed to develop effective treatments? 1.Knowledge of the disease pathophysiology -the cause – -ataxia genes galore -downstream consequences, e.g mitochondrial dysfunction in FRDA -insights into cerebellar (dys)function

22 What is needed to develop effective treatments? 1.Knowledge of the disease pathophysiology -the cause -relevant models -mice, fruit flies, worms, etc.

23 What is needed to develop effective treatments? 1.Knowledge of the disease pathophysiology -the cause -relevant models -candidate treatments -EPI-743, pioglitazone, lithium, riluzole, VEGF

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25 FRDA Timeline First clinical description Gene Discovered Outcome Measures Validated Natural History Studies Begun Treatment Trials

26 What is needed to develop effective treatments? 1.Knowledge of the disease pathophysiology -the cause -relevant models -candidate treatments 2.A way to measure the disease -clinical scales, instrumented measures, biomarkers

27 What is needed to develop effective treatments? 1.All the preliminary info: -the cause -relevant models -candidate treatments 2.A way to measure the disease 3.An understanding of the natural history of the disease -rate of progression -variability

28 What is needed to develop effective treatments? 1.All the preliminary info: -the cause -relevant models -candidate treatments 2.A way to measure the disease 3.An understanding of the natural history of the disease -rate of progression -variability 4.Research Infrastructure -ataxia centers -$$$ -research subjects

29 IS THIS TREATMENT EFFECTIVE?

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32 Copyright restrictions may apply. Lynch, D. R. et al. Arch Neurol 2010;67:

33 How can the likelihood of a positive trial be improved?

34 1. Reduce variability -large numbers -homogeneous study population -precise measures

35 How can the likelihood of a positive trial be improved? 1. Reduce variability -large numbers -homogeneous study population -precise measures 2. Increase trial timeline

36 How can the likelihood of a positive trial be improved? 1. Reduce variability -large numbers -homogeneous study population -precise measures 2. Increase trial timeline 3. Use a more effective treatment

37 How can the likelihood of a positive trial be improved? 1. Reduce variability -large numbers -homogeneous study population -precise measures 2. Increase trial timeline 3. Use a more effective treatment Note: These are not always possible

38 What can be done to facilitate treatment development? 1. Learn from other diseases

39 ALS

40 ALS vs. ataxia -ALS is 90% sporadic; pathophysiology not well understood -Quicker progression, more definite clinical measures

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42 What can be done to facilitate treatment development? 1. Learn from other diseases 2. Don’t disregard low lying fruit!!! -non-sexy treatments ARE available -(e.g. ALS PEG tubes and ventilatory support)

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45 What can be done to facilitate treatment development? 1. Learn from other diseases 2. Don’t disregard low lying fruit!!! -non-sexy treatments ARE available

46 What can be done to facilitate treatment development? 1. Learn from other diseases 2. Don’t disregard low lying fruit!!! -non-sexy treatments ARE available 3. Never underestimate the power of dedicated action

47 What can be done to facilitate treatment development? 1. Learn from other diseases 2. Don’t disregard low lying fruit!!! -non-sexy treatments ARE available 3. Never underestimate the power of dedicated action

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51 Thanks - Patients, families - Coordinators: Bettye Robinson RN, Sue Gronka RN -Colleagues -NAF, FARA, MDA, NIH


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