Developing New Drug Treatments
The most crucial prerequisite for successful drug development to treat any disease is to have identified specific targets against which therapies can be directed. In the last two decades, scientists have identified a number of therapeutic targets specific to Alzheimer's disease. The discovery of the three genes that invariably cause early-onset AD and of ApoE-ε4, the first and most significant susceptibility gene for late-onset AD, have lent a great deal of strength to the amyloid hypothesis. The importance of these genetic findings resides in the fact that these four genes all act on the same cascade of events. Through various mechanisms, they all lead to an increase in the buildup of beta-amyloid and neurofibrillary tangles in the brain, which eventually causes neurons to die. This convergence of evidence pointed toward beta-amyloid as a major therapeutic target.
Hundreds of laboratories around the world are contributing knowledge about the role of beta-amyloid in AD. Each, in a multitude of increasingly understood steps in the beta-amyloid cascade, might become a site to target with promising therapeutic compounds.
Dr. Paul Aisen, director of the Alzheimer's Disease Cooperative Study—a long-standing, National Institute on Aging-backed initiative to coordinate clinical trials of promising drugs that may be outside the purview of pharmaceutical companies—described the encouraging tenor of this era in AD drug development: "Everybody's coming together now. It's a huge endeavor, but we're working together to make progress, so that now we have candidate drugs that target the specific process that's initiating Alzheimer's disease, we have brought them into human trials, and we are perhaps close to success."
Like many others in the field, Dr. Aisen believes that researchers are well on track toward reaching their goals. "We need to optimize the treatment of the symptoms, but even more importantly, we need to slow the disease process. We need to halt it. We need to be able to prevent the development of Alzheimer's disease. We need to promote healthy aging—aging without memory impairment, without Alzheimer's disease."
These are undoubtedly ambitious objectives. Many drug targets are being investigated. Clinical trials of ninety-one drugs were underway as of 2008. More drug candidates are awaiting Food and Drug Administration approval to enter human testing. Promising drugs may help people with AD maintain their mental functioning. Others may slow, delay, or even prevent AD. Inside the world of AD drug research today, the optimism is palpable, but scientists also realize that significant challenges remain to be overcome.
Previous: Diet and the Brain
Next: Drugs in Development
Excerpted from THE ALZHEIMER'S PROJECT: MOMENTUM IN SCIENCE, published by Public Affairs, www.publicaffairsbooks.com.
In This Section
- Building Cognitive Reserve
- Brain Efficiency
- The Role of Education
- Brain Building
- Personality Factors and Social Networks
- Assessing the Potential Benefits of Exercise and Diet
- Exercise and the Brain
- Diet and the Brain
- Developing New Drug Treatments
- Drugs in Development
- Practical Challenges
Momentum in Science: The Supplementary Series
- Understanding and Attacking Alzheimer's 12 min
- How Far We Have Come in Alzheimer's Research 15 min
- Identifying Mild Cognitive Impairment 20 min
- The Role of Genetics in Alzheimer's 12 min
- Advances in Brain Imaging 11 min
- Looking Into the Future of Alzheimer's 6 min
- The Connection Between Insulin and Alzheimer's 21 min
- Inflammation, the Immune System, and Alzheimer's 29 min
- The Benefit of Diet and Exercise in Alzheimer's 16 min
- Cognitive Reserve: What the Religious Orders Study is Revealing about Alzheimer's 20 min
- Searching for an Alzheimer's Cure: The Story of Flurizan 30 min
- The Pulse of Drug Development 15 min
- The DeMoe Family: Early-Onset Alzheimer's Genetics 25 min
- The Nanney/Felts Family: Late-Onset Alzheimer's Genetics 20 min
- The Quest for Biomarkers 17 min
Video: Inside the Brain: Unraveling the Mystery of Alzheimer's Disease
This 4-minute captioned video shows the progression of Alzheimer's disease in the brain.
Inside the Brain: An Interactive Tour
The Brain Tour explains how the brain works and how Alzheimer's affects it.
Alzheimer's Disease: Unraveling the Mystery
This book explains what AD is, describes the main areas in which researchers are working, and highlights new approaches for helping families and friends care for people with AD.
- About The Scientists
Connect with Alzheimer's Research
Find out how you can participate in clinical trials or studies, find a research center, or get up-to-date information at 1-800-438-4380.
The Alzheimer's Association 24/7 Helpline provides reliable information and support to all those who need assistance. Call us toll-free anytime day or night at 1-800-272-3900.
Create A Tribute
Honor someone you care about and share your stories by contributing to The Tribute Wall on Facebook.
The Alzheimer's Association message boards and chat rooms are your online communication forum. Share your thoughts and experiences, query your colleagues, and make new friends.
- Rapid advances in our knowledge about AD have led to the development of promising new drugs and treatment strategies. However, before these new strategies can be used in clinical practice, they must be shown to work in people. Advances in prevention and treatment are only possible thanks to volunteers who participate in clinical trials.
- Among those touched by Alzheimer's (excluding self), nearly one-third provide support as a friend or relative, another 3% provide support as a healthcare professional, and the remaining two-thirds provide no support to the person suffering from Alzheimer's. When support is provided, it most often entails emotional support, followed by care-giving support. While small in comparison, more than one person in ten is providing financial support. Read more.