Multiple Scleroris Lyle Wiemerslage Ph D Typical Clinical

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Multiple Scleroris Lyle Wiemerslage, Ph. D

Multiple Scleroris Lyle Wiemerslage, Ph. D

Typical Clinical Vignette • Female in her late 20’s – About twice as common

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

Molecular Description https: //vimeo. com/68315620

Molecular Description https: //vimeo. com/68315620

Clinically Isolated Syndrome (CIS) versus MS

Clinically Isolated Syndrome (CIS) versus MS

Treatments for MS • Interferon α and β – NAbs • Glatiramer acetate –

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

Oral Medications • Fingolimod • Teriflunomide • Dimethyl fumarate

Oral Medications • Fingolimod • Teriflunomide • Dimethyl fumarate

Making an animal model for MS + Freund's adjuvant

Making an animal model for MS + Freund's adjuvant

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

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

Lifespan of a drug

Lifespan of a drug

Preclinical • Basic science – Academic work – Drug discovery – High throughput screening

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

Finding Effective Treatments • IFN-γ – Thought it may work as antiviral, MS patients

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

Measuing Symptoms • Number of relapses – Placebo effect • MRI • Expanded Disability

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

Contract Research Organiztion • ”A multicenter, double-blind, randomized, placebo-controlled, parallel group, efficacy and safety

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

Economics of Clinical Research • US patent lasts 17 years – Development to approval

Economics of Clinical Research • US patent lasts 17 years – Development to approval takes ≈10 years • Research is expensive! – 80. 000 to 400. 000 kr per patient • Orphan drugs • Price of medication can be 400. 000 kr/year/pt

More Information • Curing MS: How Science is Solving the Mysteries of Multiple Sclerosis

More Information • Curing MS: How Science is Solving the Mysteries of Multiple Sclerosis – Howard Weiner • Blog for clinical research: http: //pipeline. corante. com/