Each year on September 21st, people across the globe unite to raise awareness about dementia and Alzheimer’s Disease by recognizing World Alzheimer’s Day. With an estimated 50 million people worldwide living with Dementia, and the number expected to triple by 2050, it is more critical than ever that we unite to advance clinical research and the development of new medicines to address this disease.
On World Alzheimer’s Day 2018, Kemi Olugemo, M.D., Senior Medical Director, PAREXEL, reflects on the impact of Alzheimer’s Disease, progress toward new treatments, and the need for patient and caregiver involvement to accelerate delivery of impactful future therapies.
Alzheimer’s disease (AD) is a glaring global health burden and one of our most complex health crises. Individuals with AD experience progressive deterioration in their memory, cognitive abilities, and daily functioning. A lengthy prodromal phase preceding cognitive impairment is now recognized, and symptoms of AD typically emerge a decade or longer after the microscopic disease process begins.
Although increasing age is a known risk for developing this fatal disease, AD is thought to result from a combination of genetic and environmental factors. Geographical variations in AD incidence, prevalence, risk factors, co-morbidities, and prognosis make understanding this disease and developing treatments for it an immensely complex challenge.
Dementia’s cost to society was estimated at $818 billion in 2015 and is forecast to cost $2 trillion by 2030. To add to the burden, current approved treatments for AD are relatively ineffective, exerting only a limited effect on symptoms such as confusion and memory loss. These therapies do not alter the insidious progression of AD or modify its course. The success rate for developing new therapies in AD has been hampered by various factors; including insufficient understanding of disease biology, suboptimal diagnostic criteria, and variation in the endpoints used to assess cognition and functioning. Drug development in AD has been plagued by a daunting 99 percent failure rate. In comparison, the failure rate for cancer drugs is 81 percent.
Despite past failures, the need to persist in AD drug discovery is widely recognized. There are currently over 300 active trials for AD therapeutics, with 92 candidate drugs targeting markers such as amyloid, tau, cell death/survival, and modifiers of inflammation.
Although treatment is needed across all stages of disease progression, a therapy that can modify or even halt AD prior to symptom onset could impact millions of patients. Regulators and governing bodies are demonstrating their commitments to this end. Recent regulatory guidance provided by the United States Food and Drug Administration describes innovative approaches to studying very early disease before patients experience any symptoms of dementia. Regulators in Europe have also revised their guidelines.
The International Working Group and the US National Institute on Aging and Alzheimer's Association are seeking to unify the staging and definitions of AD to standardize reporting of research findings. Several registries exist to collect epidemiological data on AD, facilitate research, and to support best clinical practice.
At this critical time for AD drug development, patients and their advocates are equipped to shape the future of and outlook for AD. The voice of the patient and those closest to this disease must be heard and actioned for research to make an impact. Engaging patients and their caregivers can enhance the process by:
As we mark another World Alzheimer’s Day and look to change the future of drug development in AD, the importance of international collaboration in research, inclusive of the patient voice, cannot be overstated.
Central Nervous System
Global Drug Development