CNS Trials Almost 15% of ALL FDA-Regulated Clinical Trials in 2007--PAREXEL Report
Clinical studies for neurology drugs represented 8.9% percent of all FDA-regulated clinical trials in 2007, according to a new study released in PAREXEL's Bio/Pharmaceutical R&D Statistical Sourcebook. The study also found that trials for psychiatric therapies comprised another 5.5% of studies regulated by the FDA.
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Imaging technologies open new horizons for CNS research
The aging of the population in North America, Western Europe, and Japan is bringing increased research focus on Alzheimer’s disease (AD), Parkinson’s disease (PD), and other neurodegenerative disorders that affect significant numbers of older people. In the US alone, more than 4.5 million people are estimated to be afflicted by AD – about 10 percent of the population over 60. Some studies predict that the number of AD patients will reach 13 million by 2050 unless an effective AD treatment is discovered.
Efforts to develop treatments for devastating central nervous system (CNS) disorders such as AD have been bolstered in recent years by significant advances in neuroimaging that allow researchers to view the changes in the brain that characterize these disorders. Enhancements to technologies such as magnetic resonance imaging (MRI) and positron emission tomography (PET) have opened new horizons for researchers working to identify key biomarkers associated with the onset and progression AD and other CNS diseases. Those biomarkers could point the way to promising therapeutic targets and speed up the development of effective therapies. CNS biomarkers could also serve as surrogate endpoints in future clinical trials, allowing potential new treatments to be tested more quickly and efficiently.
Understanding CNS disorders
“The current use of advanced imaging technologies to search for CNS disease biomarkers is the culmination of decades of scientific progress in imaging,” said James Paskavitz, M.D., Medical Director, Perceptive Informatics, who joined the company in 2006 following a distinguished career in neuroimaging and neurology, including leading one of largest dementia practices in the Northeast. “The technology is now being applied to help researchers better understand the biological mechanisms and progression of these disorders inside the brain, which we had previously been unable to view directly in patients. We are beginning to see studies indicating that imaging biomarkers correlate to cognitive test results in AD, and that they may be more accurate indicators of disease progression than cognitive tests. Validation of imaging biomarkers as a way to accurately predict the progression of neurodegenerative disorders would be a major step forward in developing efficacious treatments.”
While neuroimaging is proving invaluable in expanding scientific knowledge about serious CNS disorders – and pointing the way toward new therapeutic targets – the industry still faces a major challenge in fully validating imaging biomarkers. “Because there are no current treatments that effectively delay or halt the progress of these diseases, there is no way to directly establish a cause-and-effect correlation between the changes we see in the brain and the disease,” Dr. Paskavitz noted. “We cannot be sure whether the physiological changes are the cause or the symptom of a neurodegenerative disease until we can observe a significant change in the disease’s progression as a result of a therapeutic agent. We have made substantial progress in understanding AD, PD, and other CNS diseases as a result of neuroimaging, but there is still more work to be done to turn that knowledge into effective therapies.”
Using imaging to improve product development
Although effective therapies for AD and PD may still be years in the future, advanced imaging technology offers many benefits to current CNS drug development efforts, particularly in the pre-clinical stages, according to James Vornov, M.D., Ph.D., Senior Medical Director of PAREXEL’s Clinical Research Services group, who is a neurologist with extensive experience in the use of biomarkers in clinical trials (see a profile of Dr. Vornov). “Neuroimaging gives researchers tremendous insight into the physical changes taking place within the brain that are associated with disorders such as AD, PD, and stroke,” Dr. Vornov said. “Even though the biomarkers of these conditions have not been fully validated, there is sufficient correlation to incorporate imaging and other biomarkers into the development process for CNS therapies.”
“These biomarkers can be used to guide early development projects along pathways that potentially offer the greatest chance for success,” said Dr. Vornov. “Imaging technologies can play a particularly important role during the translational stage of early product development to select promising therapeutic approaches. Neuroimaging can also provide unbiased results to help company executives evaluate research direction. Those within the research community who are waiting for neuroimaging biomarkers to be fully validated are missing out on a powerful tool that could greatly enhance their research efforts.”
The future of neuroimaging
The future of imaging technology and CNS research should be even more exciting. “As our knowledge of CNS biomarkers increases, I would expect to see neuroimaging technologies provide surrogate endpoints for Phase II and Phase III clinical trials that could substantially reduce the time and cost of these trials,” Dr. Paskavitz said. “Fewer patients would be needed for the trials, and the results would be more definitive because of imaging technology. The ability to speed up the drug development process will be especially important as serious conditions like AD affect a larger segment of the population.”
Imaging biomarkers are also expected to play an increasingly important role in diagnosing CNS disorders – especially in their early stages – and may also help provide more precise identification of diseases such as depression, bipolar disorder, and schizophrenia that are currently diagnosed by clinical observation. Further in the future, neuroimaging may be able to help identify patients who might respond better to specific CNS therapies based on the presence or absence of particular biomarkers. This type of “personalized” medicine could be especially useful for treating psychiatric disorders, where empirical evidence shows that patients with similar symptoms have widely different responses to a given therapy. The ability to target therapies to the patients most likely to benefit from them would be a major step forward in the treatment of CNS disorders.
Leveraging imaging technologies
PAREXEL is uniquely positioned to help sponsors leverage advanced imaging technologies to improve their research into CNS diseases and other therapeutic areas. Experts such as Dr. Vornov in the company’s Clinical Research Services group offer a vast depth of experience in incorporating neuroimaging into CNS research and clinical trials, as well as the use of biomarkers as surrogate endpoints to speed up early-stage drug development. Dr. Paskavitz, a pioneer in neuroimaging, leads a team of therapeutic and medical imaging experts in the Medical Imaging Division of Perceptive Informatics – PAREXEL’s technology subsidiary – that provides sponsors with a wide range of capabilities in the application of imaging techniques from early development through peri-approval studies in multiple therapeutic areas. The Medical Imaging Division also has significant experience in the use of the latest imaging modalities such as fluorodeoxyglucose (FDG) PET scanning and dynamic contrast-enhanced (DCE) MRI.