Senior Vice President, Drug Development Services
Vice President, Global Therapeutic Lead
Parkinson’s disease is the second most common neurodegenerative disorder after Alzheimer’s disease.
The anti-Parkinson disease medications currently available primarily promote dopamine levels in the brain to compensate for the loss of dopamine due to neuro-degeneration. Apart from the need to better manage symptoms in advanced stages of the disease, there is an even greater need to develop neuro-protective or neuro-regenerative therapies to slow, stop or reverse disease progression.
Taking into consideration the lessons learned from Alzheimer’s disease studies could therefore benefit the design of such neuro-protective studies. These are summarised as follows:
- Target early disease stages: In most neurodegenerative diseases, symptoms only occur when more than 70% of the relevant nerve cells are already compromised. Neuro-regenerative therapies that may still work at such a late stage are not yet on the horizon.
- Make sure the study population is sufficiently homogeneous: With more precise diagnostic tools, it becomes evident that a clinical diagnosis of Parkinson’s disease may be wrong in about 15% of cases.
- Don’t rush into costly, large, phase III studies: Take the time to explore preliminary evidence of efficacy, suitable therapeutic dosage ranges and proper patient population in phase II clinical development.
- Explain to study subjects and caregivers that they should not expect improvements of Parkinson’s disease symptoms, although they may benefit from a slower disease progression over time.
- Take special measures to ensure adherence to the investigational product for non-injectable therapies: Patients who received education were 96% adherent at six months, whereas with no education, adherence was 58.5% at six months.
- Do not rely on biomarkers, but put clinical outcomes as an endpoint in phase II trials: If the validity of a biomarker is not fully proven, it should not be used to take milestone-related decisions in the development plan.
Taking all these lessons learned into consideration could markedly lower the risk of a false negative result in clinical trials for disease-modifying therapies against Parkinson’s disease.
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This blog is an edited version of “Key Considerations for Disease-Modifying Parkinson’s Disease Trials” which appeared in Pharmaceutical Market Europe in November 2018. To view the full article, please visit http://www.pmlive.com/