1. Multiple Scleroris
Lyle Wiemerslage, PhD

2. Typical Clinical Vignette
Female in her late 20’s
About twice as common in women
Genetic susceptibility
Geography, more common far from equator
First symptom optic neuritis
Loss of vision OD OS

3. Molecular Description

4. Clinically Isolated Syndrome (CIS) versus MS

5. Treatments for MS Interferon α and β Glatiramer acetate
NAbs
Glatiramer acetate
Molecular decoy?
Monoclonal antibodies
Natilizumab
Alemtuzumab

6. Oral Medications
Fingolimod
Teriflunomide
Dimethyl fumarate

7. Making an animal model for MS
+ Freund's adjuvant

8. Clinical Trials for MS
clinicaltrials.gov
Outside of academia

9. Lifespan of a drug

10. Preclinical Basic science No Observable Adverse Effect Levels (NOAEL)
Academic work
Drug discovery
High throughput screening
No Observable Adverse Effect Levels (NOAEL)
Investigator's brochure (IB)
Animal studies
Adverse effects

11. Finding Effective Treatments
IFN-γ
Thought it may work as antiviral, MS patients also had decreased production of it (false)

12. Measuing Symptoms Number of relapses MRI
Placebo effect
MRI
Expanded Disability Status Scale (EDSS)
multiple sclerosis functional composite (MSFC)

13. Contract Research Organiztion
”A multicenter, double-blind, randomized, placebo-controlled, parallel group, efficacy and safety trial of *drug* in patients with...”

14. Economics of Clinical Research
US patent lasts 17 years
Development to approval takes ≈10 years
Research is expensive!
to kr per patient
Orphan drugs
Price of medication can be kr/year/pt

15. More Information
Curing MS: How Science is Solving the Mysteries of Multiple Sclerosis
Howard Weiner
Blog for clinical research: