Breaking News
May 3, 2019 - Vaping and Smoking May Signal Greater Motivation to Quit
May 3, 2019 - Dementia looks different in brains of Hispanics
May 3, 2019 - Short-Staffed Nursing Homes See Drop In Medicare Ratings
May 3, 2019 - Study of teens with eating disorders explores how substance users differ from non-substance users
May 3, 2019 - Scientists develop new video game that may help in the study of Alzheimer’s
May 3, 2019 - Arc Bio introduces Galileo Pathogen Solution product line at ASM Clinical Virology Symposium
May 3, 2019 - Cornell University study uncovers relationship between starch digestion gene and gut bacteria
May 3, 2019 - How to Safely Use Glucose Meters and Test Strips for Diabetes
May 3, 2019 - Anti-inflammatory drugs ineffective for prevention of Alzheimer’s disease
May 3, 2019 - Study tracks Pennsylvania’s oil and gas waste-disposal practices
May 3, 2019 - Creating a better radiation diagnostic test for astronauts
May 3, 2019 - Vegans are often deficient in these four nutrients
May 3, 2019 - PPDC announces seed grants to develop medical devices for children
May 3, 2019 - Study maps out the frequency and impact of water polo head injuries
May 3, 2019 - Research on Reddit identifies risks associated with unproven treatments for opioid addiction
May 3, 2019 - Good smells may help ease tobacco cravings
May 3, 2019 - Medical financial hardship found to be very common among people in the United States
May 3, 2019 - Researchers develop multimodal system for personalized post-stroke rehabilitation
May 3, 2019 - Study shows significant mortality benefit with CABG over percutaneous coronary intervention
May 3, 2019 - Will gene-editing of human embryos ever be justifiable?
May 3, 2019 - FDA Approves Dengvaxia (dengue vaccine) for the Prevention of Dengue Disease in Endemic Regions
May 3, 2019 - Why Tonsillitis Keeps Coming Back
May 3, 2019 - Fighting the opioid epidemic with data
May 3, 2019 - Maggot sausages may soon be a reality
May 3, 2019 - Deletion of ATDC gene prevents development of pancreatic cancer in mice
May 2, 2019 - Targeted Therapy Promising for Rare Hematologic Cancer
May 2, 2019 - Alzheimer’s disease is a ‘double-prion disorder,’ study shows
May 2, 2019 - Reservoir bugs: How one bacterial menace makes its home in the human stomach
May 2, 2019 - Clinical, Admin Staff From Cardiology Get Sneak Peek at Epic
May 2, 2019 - Depression increases hospital use and mortality in children
May 2, 2019 - Vicon and NOC support CURE International to create first gait lab in Ethiopia
May 2, 2019 - Researchers use 3D printer to make paper organs
May 2, 2019 - Viral infection in utero associated with behavioral abnormalities in offspring
May 2, 2019 - U.S. Teen Opioid Deaths Soaring
May 2, 2019 - Opioid distribution data should be public
May 2, 2019 - In the Spotlight: “I’m learning every single day”
May 2, 2019 - 2019 Schaefer Scholars Announced
May 2, 2019 - Podcast: KHN’s ‘What The Health?’ Bye-Bye, ACA, And Hello ‘Medicare-For-All’?
May 2, 2019 - Study describes new viral molecular evasion mechanism used by cytomegalovirus
May 2, 2019 - SLU study suggests a more equitable way for Medicare reimbursement
May 2, 2019 - Scientists discover first gene involved in lower urinary tract obstruction
May 2, 2019 - Researchers identify 34 genes associated with increased risk of ovarian cancer
May 2, 2019 - Many low-income infants receive formula in the first few days of life, finds study
May 2, 2019 - Global study finds high success rate for hip and knee replacements
May 2, 2019 - Taking depression seriously: What is it?
May 2, 2019 - With Head Injuries Mounting, Will Cities Put Their Feet Down On E-Scooters?
May 2, 2019 - Scientists develop small fluorophores for tracking metabolites in living cells
May 2, 2019 - Study casts new light into how mothers’ and babies’ genes influence birth weight
May 2, 2019 - Researchers uncover new brain mechanisms regulating body weight
May 2, 2019 - Organ-on-chip systems offered to Asia-Pacific regions by Sydney’s AXT
May 2, 2019 - Adoption of new rules drops readmission penalties against safety net hospitals
May 2, 2019 - Kids and teens who consume zero-calorie sweetened beverages do not save calories
May 2, 2019 - Improved procedure for cancer-related erectile dysfunction
May 2, 2019 - Hormone may improve social behavior in autism
May 2, 2019 - Alzheimer’s disease may be caused by infectious proteins called prions
May 2, 2019 - Even Doctors Can’t Navigate Our ‘Broken Health Care System’
May 2, 2019 - Study looks at the impact on criminal persistence of head injuries
May 2, 2019 - Honey ‘as high in sugars as table sugar’
May 2, 2019 - Innovations to U.S. food system could help consumers in choosing healthy foods
May 2, 2019 - FDA Approves Mavyret (glecaprevir and pibrentasvir) as First Treatment for All Genotypes of Hepatitis C in Pediatric Patients
May 2, 2019 - Women underreport prevalence and intensity of their own snoring
May 2, 2019 - Concussion summit focuses on science behind brain injury
May 2, 2019 - Booker’s Argument For Environmental Justice Stays Within The Lines
May 2, 2019 - Cornell research explains increased metastatic cancer risk in diabetics
May 2, 2019 - Mount Sinai study provides fresh insights into cellular pathways that cause cancer
May 2, 2019 - Researchers to study link between prenatal pesticide exposures and childhood ADHD
May 2, 2019 - CoGEN Congress 2019: Speakers’ overviews
May 2, 2019 - A new strategy for managing diabetic macular edema in people with good vision
May 2, 2019 - Sagent Pharmaceuticals Issues Voluntary Nationwide Recall of Ketorolac Tromethamine Injection, USP, 60mg/2mL (30mg per mL) Due to Lack of Sterility Assurance
May 2, 2019 - Screen time associated with behavioral problems in preschoolers
May 2, 2019 - Hormone reduces social impairment in kids with autism | News Center
May 2, 2019 - Researchers synthesize peroxidase-mimicking nanozyme with low cost and superior catalytic activity
May 2, 2019 - Study results of a potential drug to treat Type 2 diabetes in children announced
May 2, 2019 - Multigene test helps doctors to make effective treatment decisions for breast cancer patients
May 2, 2019 - UNC School of Medicine initiative providing unique care to dementia patients
May 2, 2019 - Nestlé Health Science and VHP join forces to launch innovative COPES program for cancer patients
May 2, 2019 - Study examines how our brain generates consciousness and loses it during anesthesia
May 2, 2019 - Transition Support Program May Aid Young Adults With Type 1 Diabetes
May 2, 2019 - Study shows how neutrophils exacerbate atherosclerosis by inducing smooth muscle-cell death
May 2, 2019 - Research reveals complexity of how we make decisions
Precision medicine offers a glimmer of hope for Alzheimer’s disease

Precision medicine offers a glimmer of hope for Alzheimer’s disease

PET scan of a human brain with Alzheimer’s disease. Credit: public domain

The decadeslong search for effective ways to treat or prevent Alzheimer’s disease is littered with failures, leaving 5.7 million Americans already stricken with this form of dementia without a lifeline. The rest of us are left to hope we won’t be among the 1 in 10 over 65 who gets the devastating diagnosis.

But precision medicine—an approach that is changing the treatment of cancer and spawning targeted therapies for a wide range of diseases—may open new avenues for the treatment of Alzheimer’s disease. And new ways to test experimental treatments promise to more quickly identify treatments that work, and perhaps the patients in whom they will work best.

This week, dementia specialists gathered at the Alzheimer’s Association’s International Conference in Chicago to assess the state of the field. And researchers gleaned some glimmers of real hope in both precision medicine and innovative trial designs to deliver new treatments for Alzheimer’s disease.

These approaches raised expectations that at least two experimental drugs—one called BAN2401 and the other Anavex 2-73—might successfully treat some with Alzheimer’s.

In the case of BAN2401, the successful preliminary finding of a trial that enrolled 856 patients with early Alzheimer’s disease has breathed new life into a hypothesis that persists despite withering failures: that reducing clumps of proteins called amyloid plaques that accumulate in the brains of those with Alzheimer’s might slow or reverse their symptoms of memory loss and cognitive confusion.

Using an innovative clinical trial design, the U.S. and Japanese companies developing BAN2401 found that over 18 months of treatment, patients who got the highest doses of the medication had dramatic reductions in amyloid plaque deposits in their brains. And compared with subjects who got a placebo, those who got the highest dose showed a 26 percent slowing of clinical decline after 18 months.

As the trial of BAN2401 progressed, its “adaptive design” ensured that when new subjects were recruited, they were more likely to be assigned to arms of the trial that showed the greatest promise. While considered controversial by some researchers, clinical trial designs that flex to seize upon promising preliminary findings have a high-level supporter in the Trump administration’s Food and Drug Administration: Commissioner Scott Gottlieb.

“Alzheimer’s disease trials need to evolve,” said James A. Hendrix, director of global science initiatives at the Alzheimer’s Association. “We need to try new things and learn from other diseases,” he added, including fields such as cancer, where for select subsets of patients, new targeted therapies are delivering cures.

Distinguishing between likely responders and those not likely to be helped by a medication first, said Hendrix, “is an exciting new way to think about” new treatments for Alzheimer’s. “Maybe one-size-fits-all is not the best approach,” he added.

In a first-ever bid to apply the principles of precision medicine to Alzheimer’s disease, researchers also reported this week on a small study of Alzheimer’s patients who bear a few “actionable genetic variants.” In these patients, they found, Anavex 2-73 appeared to slow and perhaps even reverse early cognitive decline.

The new findings emerged from a clinical trial designed to test the safety of Anavex 2-73, and it involved just 32 patients with mild to moderate Alzheimer’s. So it’s far too early to tout the success of this experimental drug.

But researchers built an extra step into their safety trial: sequencing the subjects’ genomes. In doing so, they hoped to find genomic signatures in some patients that would make them more likely to respond positively to the drug.

While the researchers combed through more than 33,000 genes, they had a pretty good picture of what molecular processes (or pathways) Anavex 2-73 worked on. So they knew what they were looking for: gene variations that were likely to interfere with the actions of Anavex 2-73 and make treatment unsuccessful. They found two such genetic variants, present in about one-fifth of humans.

When researchers from Anavex narrowed the subject pool to just those patients whose genetic makeup was compatible with its mode of action, they found that subjects who got the medication for 57 weeks experienced “clinically meaningful” improvements in their ability to reason, remember and carry on daily activities.

A larger new trial on subjects with Alzheimer’s is set to begin in Australia and North America. The experimental medication is also being tested as a possible treatment for dementia associated with Parkinson’s disease, for Rett syndrome and for Fragile X syndrome.

The idea that a medication to treat Alzheimer’s disease might work well in some dementia patients and not as well in others is consistent with a growing sense, first, that Alzheimer’s may be many different diseases. But it also fits with the notion that genetic factors—acting alone or collectively—predispose some to Alzheimer’s while protecting others.

In precision medicine, sometimes called personalized medicine, researchers work to identify the genetic factors that drive or contribute to a disease and build medicine that targets the downstream effects of those miscreant genes. Then, they use genomic sequencing technologies to identify just those patients who bear the distinctive genetic signatures their drug works on. More often than not, these drugs are costly, and they don’t work on everyone. But when the right patients get the right medicine at the right time, treatments will be more effective and have fewer side effects.

“If you include a biomarker, which can be detected in a matter of hours or days by a swab test, then you can enrich a study—enroll just the subjects most likely to respond—and of course, improve the chances of success,” said Christopher Missling, a founding director of Anavex Life Sciences Corp.

Such new approaches to treating dementia could turn the tide in the discouraging search for Alzheimer’s treatments, said Dr. Deepak Bhatt, an expert on adaptive trial designs and precision medicine.

“Identifying which patients might benefit from a novel therapy using biomarkers or genetics will likely be a big part of how medicine is individualized in the future—so-called precision medicine,” said Bhatt, who directs interventional cardiovascular programs at Brigham and Women’s Hospital in Boston.

“This approach may be especially well suited for therapies that might have side effects or are expensive. While further confirmatory studies are needed for this particular Alzheimer’s drug before clinical use, the potential of personalizing care for such a devastating disease is exciting. ”


Explore further:
Alzheimer’s drug trial offers new hope, but uncertainty, too

Tagged with:

About author

Related Articles