Real World Health Care Blog

Prevention/Patient-Centered Care

NSCLC: The Emerging Role of Liquid Biopsies

Real World Health Care concludes its series on non-small cell lung cancer by speaking with Erica Carpenter, MBA, PhD, Research Assistant Professor in the Department of Medicine at the Perelman School of Medicine at the University of Pennsylvania. Dr. Carpenter also serves as Director of the Circulating Tumor Material Laboratory in the Division of Hematology/Oncology at the Abramson Cancer Center.

Dr. Carpenter served as senior author on a recent study that suggests for patients with advanced lung cancer, a non-invasive liquid biopsy may be a more effective and suitable alternative to the gold standard tissue biopsy to detect clinically relevant mutations and help guide the course of treatment.

Real World Health Care: Explain your role at the Perelman School of Medicine.

carpenter_erica_businessErica Carpenter: I am Director of the Circulating Tumor Material Laboratory and an Assistant Professor in the Division of Hematology-Oncology at the Perelman School of Medicine. My lab focuses on the identification, capture, and analysis of Circulating Tumor Cells (CTCs) and cell-free DNA (cfDNA), exosomes and other material shed from cancer patient tumors.  Blood, bone marrow, pleural effusions, and other non-invasively captured patient samples are used to detect biomarkers which will allow: early detection of disease as well as post-therapy monitoring of minimal residual disease, an efficient means of determining clinical and biological response to therapy and, thus, clinical decision making, and cancer genetic phenotyping to drive personalized medicine that obviates the need for serial biopsies in a population of patients for whom these procedures can be difficult, risky, and insufficient.

RWHC: Can you give us an overview of your recent research as it relates to non-small cell lung cancer and liquid biopsies?

EC: While the work described in the Clinical Cancer Research paper is the only NSCLC liquid biopsy study we have completed, we have several studies currently enrolling, including studies on the use of liquid biopsies to monitor and predict response of patients receiving a form of immunotherapy known as checkpoint inhibitors, and characterization of the tumor cells and circulating tumor DNA found in the pleural effusions (excess fluid that sometimes builds up in patients’ lungs) of lung cancer patients as another method for monitoring without an invasive biopsy.

RWHC: Just a few months ago, the FDA approved the first liquid biopsy test for patients with metastatic NSCLC. Why is this an important development?

EC: This is important for a number of reasons. First, the cobas test detects mutations in a gene called EGFR that can be targeted with specific drugs that have been shown to be effective in NSCLC patients with these specific mutations. In addition, liquid biopsies in general can greatly improve sensitivity of mutation detection in metastatic patients. Metastatic disease means that a patient has tumors in multiple sites in the body, and this can make surgical biopsy or fine needle aspirate very difficult to perform without great discomfort to the patient. Metastases can sometimes occur in places like the bone or brain where it is impossible to invasively obtain tumor tissue. Moreover, the genetic profile of a tumor tends to evolve over the course of a patient’s therapy, and it is not unusual for a metastatic patient to exhibit mutational heterogeneity where not all mutations are expressed in all tumor locations. In these cases, it is thought that a liquid biopsy will detect all or most mutations as all tumor sites, whether primary or metastatic, are thought to shed DNA into the blood. Finally, when a next-generation sequencing panel, such as the one used in our study, is applied to a liquid biopsy, comprehensive and clinically actionable data can be obtained with a single blood test, including mutations in multiple genes associated with therapy resistance.

RWHC: Do you think that liquid biopsies will become a standard of care for NSCLC patients? Will they, or should they replace tissue biopsy? Why or why not?

EC: Liquid biopsies are already becoming a routine part of care for NSCLC and other cancer patients here at the Abramson Cancer Center at Penn Medicine. These tests can be incorporated into the blood draw procedure for other standard of care monitoring blood tests and performed at already scheduled outpatient visits, thus causing no additional pain or inconvenience to the patient. We are working to better understand the different ways in which liquid biopsies can be used to better manage the care of these patients. However, while I expect liquid biopsy use for NSCLC and other cancer patients to continue to expand, I do not expect that they will replace tissue biopsy. Next-generation sequencing of circulating tumor DNA cannot provide crucial phenotypic information, such as information about the physical characteristics of the tumor, that are essential for initial diagnosis. Tissue biopsies can also play a unique role in monitoring of patients for the development of neuroendocrine disease, which can be associated with therapy resistance but is not detectable using ctDNA.

RWHC: What challenges need to be overcome to make them a standard of care?

EC: More studies need to be done, and this is an area of active focus for us, to measure whether the use of liquid biopsies has a positive effect on patient outcomes. In addition, clinical ctDNA tests tend to be ordered for detection of mutations that can be therapeutically targeted, or the emergence of mutations that signal the development of therapy resistance. To become a larger part of the standard of care for NSCLC and other cancer patients, we must better understand whether liquid biopsies can be used to effectively monitor patients receiving other important forms of therapy, including recently approved types of immunotherapy.

RWHC: This approved liquid biopsy test detects a specific type of gene mutation that is present in about 10-20 percent of NSCLC patients. Do you think other liquid biopsy tests will eventually be able to do the same for the remaining 80-90 percent of NSCLC patients? If yes, where does the biggest opportunity exist? If no, why not?

EC: In our study, we detected 275 mutations in 45 different genes, with at least one alteration found in the liquid biopsy for 84 percent of patients. Further underscoring the clinical utility of such a comprehensive liquid biopsy approach, 70 percent of patients were deemed to have a relevant clinical trial available on the basis of their ctDNA result, 55 percent of patients had an off-label targeted therapy that could possibly be used, and 31 percent of patients had an FDA-approved therapy available. So, I would say that liquid biopsy tests are already providing meaningful results for the majority of NSCLC patients.

RWHC: Beyond detecting the gene mutation responsible for NSCLC, how can liquid biopsies be used to monitor the progression of the disease?

EC: As I mentioned earlier, circulating tumor DNA tests for NSCLC patients are typically used to detect driver mutations and/or mutations that indicate the development of therapy resistance, some of which can also be therapeutically targeted. However, sometimes the physical characteristics of the tumor, including the number of circulating tumor cells in blood and expression of certain protein markers, can be measured by liquid biopsy and used to monitor disease progression. For instance, an increase or decrease in the number of circulating tumor cells (CTCs) detected in a tube of blood can be an indication of disease progression or response to therapy, respectively. Moreover, these CTCs can be isolated and gene expression measured to detect signatures associated with sensitivity or resistance to certain forms of chemotherapy in other cancers. CTCs can also be used to non-invasively assess expression of a protein known as PDL-1 which has been shown to be associated with a response to a form of immune therapy known as checkpoint inhibitors.

RWHC: How can NSCLC liquid biopsy tests be used to help increase the length of NSCLC patient survival?

EC: This is an area of active study for us. We will seek to measure whether repeat, non-invasive liquid biopsies can be used to detect therapeutic targets when a tissue biopsy isn’t possible, thus possibly enhancing the chance of a clinical response to therapy. Others have shown that a liquid biopsy can detect therapy resistance weeks or even months before standard of care imaging. It will be important to determine whether such early detection, especially when used to guide re-stratification of the patient onto a different therapy, has a positive effect on outcomes. Additionally, one of the most significant predictors of prognosis in NSCLC is the stage of disease at presentation, and our lab is actively focused on determining whether liquid biopsies can be utilized to identify patients with early stage disease.

How to Help Sick Kids Get Better When Insurance Isn’t Enough

During the month of August, Real World Health Care will take a short break from focusing on medical breakthroughs and the researchers who are shaping the future of medicine. We will instead bring you a special series from our sponsor, the HealthWell Foundation, about what happens when families cannot afford the medical treatments their children desperately need. The families we will profile have turned to the Foundation for help, through the HealthWell Pediatric Assistance Fund®, the only fund of its kind.

Since its launch in 2013, HealthWell’s Pediatric Assistance Fund has awarded more than $850,000 in grants to help more than 400 children start or continue critical treatments covering more than 90 disease areas and conditions, including ADHD, autism, cerebral palsy, Type 1 Diabetes, epilepsy, scoliosis, seizure disorder and many more. The Fund covers family cost-shares for surgical procedures, medical devices, counseling services and prescription drug copays. This week, we’d like you to meet Karis, whose family can’t afford her type 1 diabetes testing supplies.

Imagine this: You take your child to the doctor thinking she has an infection. You discover that she has a life-altering condition and your health insurance doesn’t cover all the costs.

That’s what happened to Alicia Bell when her daughter Karis was diagnosed with type 1 diabetes. Says Bell: “There are no vacations from being a parent, and there are no vacations from being a parent of a T1D child.”

Bell was not only surprised at her daughter’s diagnosis, she was further amazed to find her insurance wouldn’t pay for all the diabetic testing supplies Karis would need.

Did You Know?

60% of bankruptcies in the U.S. are related to medical expenses.

The Bell family is far from unique. Each year, more and more Americans are forced to choose between paying for lifesaving treatments and for food, housing and utilities. People may cut pills in half, skip meals or housing payments or declare bankruptcy. An estimated 29 million Americans are underinsured and more than 60 percent of all bankruptcies in the U.S. are related to medical expenses. In the Bells’ case, Alicia would need to go into serious credit card debt just to pay for her daughter’s diabetes monitoring and testing supplies.

“I would never want money to factor into my daughter’s health care,” Bell says. “I’d sell my

Karis and her new insulin monitor

Karis and her new insulin monitor

house and everything I own if I had to.”

Fortunately, when health insurance is not enough, there is a group that helps close the gap, putting life-changing medications within reach for thousands of people in need and helping to pay for prescription drug copayments, deductibles and health insurance premiums for critical treatments.

A HealthWell Foundation Pediatric Assistance Fund grant not only pays for Karis’ supplies—including an insulin monitor, so Karis doesn’t have to have her finger stuck several times a day—it pays the co-insurance for her hospital and clinic visits and will help pay for an insulin pump if and when she needs one.

Your generous gift to the HealthWell Foundation can help kids like Karis and others afford the medical treatments they desperately need. Consider a monthly gift, a tribute or memorial donation, or an employer-sponsored fundraiser or end-of-year giving campaign to make an extraordinary and lasting difference in the lives of kids in need. Donate today.

Attacking All Angles of Alzheimer’s

Editor’s Note: This article originally appeared in Biotech Primer Weekly. For more information on the science behind the headlines, please subscribe.

Emily Burke,

Emily Burke,

Alzheimer’s disease ranks as one of the toughest nuts to crack within drug discovery and development. Current treatments merely manage symptoms, so finding a better solution becomes more and more urgent as the aging population grows.

The pathology most commonly associated with Alzheimer’s disease (AD) is the buildup of amyloid-beta (Aβ) plaques in the brain. Recent research from Stanford University suggests the plaques bind to a receptor on nerve cells, disrupting their function. However, there is no absolute consensus that these clumps of protein are the origins of AD or a symptom of the underlying cause.

Most experimental drugs have focused on “mopping up” or inhibiting the production of Aβ plaques. Failures in early clinical trials dominate the treatment landscape, with a few potentials in the pipeline (aducanumab in Phase III). In the race to find a cure, every possibility offers a glimmer of hope, so let’s shine a light on the developmental drugs stepping away from Aβ plaques.

Loss of Neurons

A key clinical feature of AD patients is the loss of neurons. What if there was a therapy that could jump start the development of new neurons? Two companies are leading the charge in developing small molecule activators of neurogenesis. By screening large chemical libraries, they have identified various compounds that show promise in activating neurogenesis from adult neural stem cells, both in tissue culture and in mouse models.

In a mouse model of Alzheimer’s, compound NNI-362 promoted the growth of new hippocampal neurons that not only migrated to the correct functional location but also differentiated and survived long enough to reduce the previously observed cognitive declines. The hippocampus is thought to play a role in memory formation and spatial navigation and is one of the first regions of the brain to show damage in AD. Phase I trials for NNI-362 are currently in preparation.

Another neurogenesis candidate, NSI-189, increased the hippocampal region of mouse brains by as much as 20 percent. Phase I trials for NSI-189 were recently completed for major depressive order, with an aim to branch out to Alzheimer’s disease in the future.

Engineering Yeast Cells

Rather than directly targeting Aβ plaques, one group of researchers is working to identify their roots. By engineering yeast cells to produce the Aβ protein, this research is monitoring the detrimental downstream effects of over-expression — like the disruption in the folding of other essential cellular proteins. Compounds that show promise in the yeast cells are then tested in AD patient-derived cells to screen for potential drugs. The sponsor is currently preparing to begin clinical trials with its lead compound.


Another company is bypassing Aβ plaques altogether and going after neuroinflammation. This pathway grew out of research conducted at Stanford, suggesting that a protein known as c1q is present in higher levels in the brains of AD sufferers. C1q accumulates at neuronal synapses, the key points of communication between brain cells. C1q also acts as a flag for other immune cells like macrophages — these “big eaters” chomp up cellular debris. The correlation of c1q could account for the observed reduction in synapse numbers and the accompanying loss of cognitive function seen in AD. The sponsor’s lead candidate, now in preclinical development, is a monoclonal antibody which “mops up” excess c1q.


A partnership between two sponsors is targeting AD-associated protein aggregates by activating a cellular component known as the proteasome. Proteasomes get rid of damaged proteins and dysfunctional protein aggregates by dismantling the peptide bonds holding them together. USP14 is one of the proteins that inhibits the proteasome, so this work is focused on the preclinical development of a USP14 inhibitor to allow proteasomes to be fully activated in AD patients.

Attacking Alzheimer’s from all angles is the surefire way to get closer to better treatments and a real cure.

Dr. R. Scott Turner on Resveratrol and the Amyloid Hypothesis

This week, we sit down with R. Scott Turner, MD, PhD, Professor of Neurology at Georgetown University and Director of Georgetown’s Memory Disorders Program. Dr. Turner specializes in cognitive behavioral neurology, memory disorders, Alzheimer’s disease and Neurodegenerative dementias. Here, he discusses his latest research as well as the roles of resveratrol and anti-amyloid treatments in AD treatments.

Real World Health Care: Why is it so important in Alzheimer’s disease treatment to find treatments that penetrate the blood-brain barrier?

Dr. R. Scott Turner, Georgetown University

Dr. R. Scott Turner, Georgetown University

Raymond Turner: Alzheimer’s disease affects the brain, and only the brain. In order for a drug treatment to be effective, the drug must penetrate the blood-brain barrier to get into the brain. For example, a drug given orally will be absorbed into the blood, and a fraction of that drug will then penetrate the brain.

RWHC: Can you provide a brief overview of your current and near future research aimed at improving AD treatment options? What research program(s) are you most excited about?

RT: The leading treatment approaches in progress are focused on blocking amyloid production from amyloid precursor protein (APP). These are BACE-1 inhibitors given by mouth daily. Or, promoting amyloid clearance (anti-amyloid monoclonal antibodies given intravenously once a month). A small fraction of antibody gets into the brain to promote clearance.

Another approach includes development of more potent sirtuin activators (building on our resveratrol trial results). Finally, a class of drugs called tyrosine kinase inhibitors (repositioned cancer drugs) promote amyloid degradation in neurons in the brain by a process called autophagy.

A major new development in AD research has been the discovery of biomarkers, including amyloid PET and tau PET scans of the brain. With these new scans, we can see and quantitate AD pathologies before death instead of the classical approach of examining brain tissue under the microscope after death. This had led to treatment studies of earlier stages of AD called mild cognitive impairment (MCI) and prodromal AD. Treatment earlier in the disease process maybe more successful, and thus we are now moving towards AD prevention studies as well.

RWHC: Why study resveratrol in relation to AD?

RT: Major risk factors for AD include diabetes, midlife obesity, and metabolic syndrome. In contrast, caloric restriction (consuming two-thirds of normal daily calories) prevents all disease of aging in laboratory animals, including AD. It is not clear if this also applies to humans, but diabetes is known to accelerate aging in man.

How does calorie restriction prevent AD? We propose a link between energy metabolism and amyloid precursor protein (APP) metabolism, which is the normal turnover and breakdown of APP to amyloid. This link may be mediated by a group of proteins/enzymes/genes called sirtuins that link energy balance to gene expression. Resveratrol and other polyphenols directly activate sirtuins to mimic the effects of calorie restriction. While resveratrol may not be the optimal molecule due to its rapid metabolism and poor brain penetration, the effects of resveratrol in our trial point to a new pathway that may be exploited to develop more potent and patentable molecules (drugs) designed to prevent and treat AD.

RWHC: What, if anything, can be done to accelerate AD research?

RT: Given the financial cost of AD to society, AD is the most underfunded disease in the NIH portfolio. We desperately need more research funding to support parallel development of more preclinical and clinical research strategies. We also need more research volunteers. Currently, less than one percent of AD patients participates in research, partly because many are never diagnosed (dementia is not a diagnosis), and because many aren’t aware of research opportunities. We also need more diverse participation in research. While our current study population is mostly Caucasian, AD affects the African-Americans and Hispanic population even more than Caucasians.

With greater funding and more research participation, we will develop safe and more effective strategies to prevent and treat AD. It’s just a question of when.

RWHC: What is your position on treatments designed to target beta amyloid protein? There seems to be some debate within the research community.

RT: So far, we have not developed a new treatment for AD based on the amyloid hypothesis: that progressive amyloid deposition in the brain causes AD. While amyloid basically defines AD, many other important pathological processes are triggered by amyloid — inflammation, tau/tangle formation, neuronal loss, synapse loss and neurotransmitter loss. We think that anti-amyloid strategies now in the pipeline will be proven safe and effective for patients with AD. However, we will likely end up with a cocktail approach, similar to the management of diabetes, hypertension and HIV. This cocktail will likely include an anti-amyloid and an anti-tangle strategy.

RWHC: Why did you get into the field of AD research?

RT: As I was completing my neurology residency, transgenic mouse models of AD were just developed and published. This presented a great new opportunity to discover new treatments in mice, and then translate these new treatments to the clinic with clinical trials. It turns out that the mice are easy to treat; we have over a thousand ways to cure AD in mice. But translation has not been so easy. We have not had a new FDA-approved drug for AD since 2003, but remain hopeful that strategies in the pipeline will be successful. All our research studies are add-on to the already available drug treatments for individuals with AD.

Patient Agitation in Alzheimer’s Disease: Implications for Patients and Caregivers

Real World Health Care recently sat down with Anton Porsteinsson, MD, Department of Psychiatry, University of Rochester School of Medicine. Dr. Porsteinsson is the Director of the University of Rochester Alzheimer’s Disease Care, Research and Education Program and has devoted his career to the care and study of individuals with memory disorders. Following up on his recent randomized clinical trial of citalopram, we discussed why it’s important to focus on treatments for AD-related agitation — both for AD patients and their caregivers.

Real World Health Care: Why is it important to study agitation in patients with Alzheimer’s disease?

Anton Porsteinsson, MD, University of Rochester School of Medicine

Anton Porsteinsson, MD, University of Rochester School of Medicine

Anton Porsteinsson: Agitation is quite common in people with AD. It has a huge impact on their quality of life, as well as their family members’ and caregivers’ quality of life.

RWHC: Prior to your CitAD Randomized Clinical Trial, why had previous pharmacological treatment options been deemed unsatisfactory?

AP: A number of medications have been studied over time for this condition, including atypical and conventional anti-psychotics and mood stabilizers. But the efficacy they showed, if any, was modest at best. At the same time, these medications have serious potential complications such as increases in cerebrovascular events, sedation, falls, Parkinsonism and even increased mortality.

RWHC: Your trial focused on patients receiving psychosocial intervention. Why is psychosocial intervention important for AD patients?

AP: Medications should rarely be the first line of treatment for AD patients. Not everyone needs to be treated with medications. That’s why it’s important to evaluate the root cause of the agitation problem. Is the patient agitated because of something going on in his or her environment? I remember a situation from a few years ago when I was seeing a summertime spike in agitation-related consultations from patients in a particular nursing home. It turns out the nursing home didn’t have air conditioning. So it’s not surprising patients were bothered and agitated. In AD cases, agitation may have as much to do with your roommate as your receptors.

Psychosocial intervention helps to channel nervous energy and restlessness by involving patients in something purposeful, or even giving them some sort of outlet, like an area to pace around where they are safe and not in anyone’s way.

RWHC: The trial also focused on the effects of citalopram on caregiver distress. Why is this an important area of study?

AP: Caregivers, who are often family members with little or no medical training, may not understand what is going on with their loved one. They may take the patient’s behavior personally, which can cause a great deal of stress. Even if you’re a saint, it can build up and take a lot out of you.

Providing care for someone with AD is very hard under the best of circumstances. It’s even more difficult when the patient is verbally or physically aggressive, uncooperative, or agitated. Caregivers need advice, support and tools to help them handle the situation. They need to learn to give themselves breaks, that it’s OK not to be perfect, and that help is available for them. I find that a lot of caregiver stress is alleviated when, as health care providers, we listen to them and take their concerns seriously.

Caregivers need to know that agitation in AD patients is common and that there are ways to deal with it. Providers must connect them with resources like the Alzheimer’s Association and community agencies. We need to help alleviate their concerns about finances. And we need to help them set up a working plan on how to deal with their situation — to bring order to the chaos.

RWHC: How will the results of the CitAD Randomized Clinical Trial inform your future AD research?

AP: This trial was extremely educational for the research community. It was one of the first studies to show that a medication was effective in multiple ways — both on a clinical scale in reducing agitation among patients and in reducing caregiver distress. We also found efficacy for other AD patient behaviors like anxiety, irritability and delusions or hallucinations.

On the flip side, we discovered some complications. Citalopram has been used widely for decades with vulnerable populations. But in the last five or six years, it’s been found to not be as safe as once thought. It has the traditional SSRI side-effects of mild gastrointestinal distress and mild sleep pattern disturbances. But it also has been found to have an impact on cardiac conduction, especially in higher doses. In fact, when we were about three-quarters of the way through the study, the FDA suggested that, for people older than 60, there should be a dose limit of 20mg per day.

We confirmed this finding in our study. We also saw a drug placebo difference on a cognitive measure, the MMSE (Mini Mental State Examination). It isn’t clear if this was due to baseline differences between the two groups and drift toward the mean, as the placebo group improved on the MMSE and the drug group saw a modest decline, or if it was a true modest cognitive toxicity. Until proven otherwise, we have opted to assume this is a potential side effect and we warn against it.

For our next study, we considered testing a lower dose of citalopram (20mg daily), but then we found that the active isomer of citalopram (S-citalopram) seemed to be better correlated to benefits seen in the study, while the inactive isomer (R-citalopram) more correlated with the adverse effects. S-citalopram is available as a generic drug, approved for depression and anxiety. We intend to study that drug further.

RWHC: What are some other areas of AD research you’re currently involved in within the URMC Memory Care Program? What do you see as your most promising area of research?

AP: We have a broad portfolio of research programs at URMC. We’re one of the more active academic-based clinical research programs in the country. Currently, we’re conducting two behavior-focused studies. One is ongoing and is based on positive findings on dextromethorphan hydrobromide and quinidine sulfate, with a new formulation that uses less quinidine. We’re also looking at methylphenidate for treatment of apathy in patients with AD.

We’re also investigating new imaging techniques and various biomarkers to improve our ability to identify those at risk. And, we’re working to find better ways of monitoring the progression of the disease and response to treatment through the ADNI study, which just received a fourth wave of funding.

Other areas we’re investigating include prevention studies with people who are cognitively normal, but who have elevated beta-amyloid or genetic biosignatures that indicate future pre-disposition. We’re looking at a passive and active vaccine against amyloid production. And we have a number of different studies on the prodromal stage of AD, working with beta secretase inhibitors that block the production of beta-amyloid.

It’s actually a very exciting time in Alzheimer’s disease research. We’re seeing improved funding from federal sources and a rejuvenation of interest from the pharmaceutical industry. I’m quite optimistic that in the next five to ten years, we will make substantive progress in terms of our ability to limit AD. I think it’s overly optimistic to expect a cure in that timeframe, but we can certainly make a dent, particularly from an early intervention standpoint. Treating this disease early is the critical factor.

Alzheimer’s Association Outlines Research Priorities & Treatment Horizons

This week, Real World Health Care brings you a conversation with Maria Carrillo, PhD, Alzheimer’s Association Chief Scientific Officer. Formed in 1980, the Alzheimer’s Association advances research to end Alzheimer’s and dementia while enhancing care for those living with the disease.

Real World Health Care: According to your literature, more than 5 million Americans suffer from Alzheimer’s disease. What do the majority of these patients most need in terms of treatments: treatments to address the symptoms of the disease, or treatments to stop or slow the progression of the disease? Why?

Maria Carrillo, Alzheimer's Association

Maria Carrillo, Alzheimer’s Association

Maria Carillo: What people need most is hope – hope that comes from significant progress in Alzheimer’s research that they can understand and that benefits their day to day life. Thanks to the amazing work of thousands of Alzheimer’s Association advocates, and many dedicated legislators, we have in place a U.S. National Plan to Address Alzheimer’s Disease. The Plan calls for real progress in treating and preventing Alzheimer’s disease by 2025. An expert workgroup convened by the Alzheimer’s Association recommended a federal investment of $2 billion per year for 10 years to make this achievement a real possibility. We are making strides toward that level of commitment – we’re over half way there – but we still have a way to go.

To more directly answer your question, both symptomatic and preventive therapies have a lot of value. People with Alzheimer’s, and those at high risk of getting it in the near-term, need something to help them now. Greatly reducing or eliminating dementia symptoms and/or pushing back the onset of dementia by ten, or even five, years, will act as something like prevention for the great majority of people – they will live most if not all of their lives free from debilitating symptoms.

Finally, we all look forward to the development of proven ways to reduce risk and prevent Alzheimer’s disease and other dementias – for the millions of lives saved, the billions of dollars saved, and the enormous suffering eliminated.

RWHC: What are some of the most promising areas of treatment research that the Alzheimer’s Association is funding?

MC: The Alzheimer’s Association is the largest private, nonprofit funder of Alzheimer’s research, awarding more than $350 million to more than 2,300 projects. Our goals are to (1) advance our understanding of Alzheimer’s, (2) help identify new treatments, (3) improve care, and (4) further our knowledge of brain health and Alzheimer’s prevention.

The Alzheimer’s Association has provided essential startup funding to, and/or funding to expand the scope of, three major Alzheimer’s disease prevention trials currently underway – the A4 Trial, DIAN TU, and API APOE4.

The Alzheimer’s Association grants program has funded – and continues to fund – some of the most important research threads in Alzheimer’s science. These topics and ideas move the field forward by contributing to knowledge about Alzheimer’s, refining research questions, and yielding clues to causes and treatments. Examples include:

  • Sex, gender and vulnerability to Alzheimer’s and other dementias
  • Commonalities between cancer and Alzheimer’s
  • Effects of oxidative stress and inflammation in Alzheimer’s
  • Vascular contributions to Alzheimer’s
  • Protein misfolding and Alzheimer’s
  • Tau toxicity in Alzheimer’s
  • Biomarkers for Alzheimer’s
  • Development of new scales for Alzheimer’s, including pain and clinical meaningfulness
  • Vaccines/Immunotherapies for Alzheimer’s
  • Insulin and insulin-degrading enzyme in Alzheimer’s
  • Down syndrome and Alzheimer’s
  • Patient ability to consent in Alzheimer’s
  • Differences in Alzheimer’s in minority communities
  • Blood pressure control and Alzheimer’s

RWHC: What are some of the biggest challenges or obstacles that Alzheimer’s researchers are facing in terms of developing new treatments? What can or should be done to overcome those challenges or obstacles, and how is the Alzheimer’s Association helping to overcome them?

MC: The two biggest issues/obstacles are insufficient funding for Alzheimer’s research and the need for more participants for clinical studies.

The Alzheimer’s Association and its nationwide constituency is the leading advocate for increased research funding at the federal level, and also the leading nonprofit funder of Alzheimer’s research. That said, many more voices need to be heard – every day and through every vehicle – to ensure that the federal government understands exactly how high a priority this issue is, and allocates the needed funds.

Recruiting and retaining clinical trial participants is now the greatest obstacle, other than funding, to developing new and better treatments for Alzheimer’s disease. Before any drug or therapy can be used in medical practice, it must be rigorously tested to certify that it is safe and effective.

To address the growing need for clinical trial participants, the Alzheimer’s Association launched Alzheimer’s Association TrialMatch – a free clinical studies matching service. More than 100,000 individuals have registered to search for Alzheimer’s clinical trials using TrialMatch.

Alzheimer’s Association TrialMatch provides users with comprehensive clinical trial information and a customized list of studies they may seek to enroll in. TrialMatch is open to everyone, including people with Alzheimer’s or other dementias, their caregivers, family members, and anyone who wants to get involved in the fight against Alzheimer’s.

There are several other registries also working to identify and marshal individuals for Alzheimer’s disease research. The primary need is for increased awareness that these services exist, and that participating in Alzheimer’s research is one of the greatest gifts that one can give to current and future generations.

RWHC: Is a preventative or curative treatment on the horizon? Do you think this is a disease that can ultimately be prevented before it starts or cured after it starts?

MC: Similar to the way we address heart disease, the preferred strategy for Alzheimer’s would include:

  • Early detection methods that identify those most at risk. Preferably these would be simple, inexpensive and non-invasive, such as a blood test.
  • Effective methods to track the biological course of the disease, and the impact of drugs and other interventions.
  • Preventive and risk reduction strategies – lifestyle and pharmacological – that can be engaged in by everyone throughout life.
  • Multiple effective treatments – that address the disease in multiple ways – for those who, despite the points above, suffer from dementia symptoms.

The Alzheimer’s Association believes that all these things are possible in the relative near-term with the appropriate investment in research.

We have made great strides in treating and preventing many diseases – even major killers such as cancer, heart disease, and HIV/AIDS – when we have made the issue a high priority and made the resources available for research. Now is the time to do the same for Alzheimer’s disease.

RWHC: What sort of initiatives or activities can we expect to see from the Alzheimer’s Association over the next few years?

MC: We still do not completely understand the causes of Alzheimer’s disease, and many questions remain about exactly how it progresses. The Alzheimer’s Association plans to redouble its efforts to support and spotlight in the basic science of the disease, for example at the Alzheimer’s Association International Conference. Increasing our knowledge in these areas should uncover multiple opportunities for therapeutic targets.

At the same time, the Association is deeply involved in clinical trials aiming to prevent Alzheimer’s disease. In addition to the funding initiatives mentioned earlier, we collaborate with Fidelity Biosciences Research Initiative (FBRI) and the principal investigators of all the major prevention studies in the Collaboration for Alzheimer’s Prevention (CAP) seeking to facilitate the sharing of information, resources and expertise that may speed the discovery of new preventive treatments. CAP was first convened in 2011 to help researchers learn from and support each other’s work; share data; harmonize data gathering and trial outcomes to allow for comparability across studies; and hold open, informal dialogue with regulators.

Inflammation is a factor that contributes to the cause and progression of many diseases and conditions. This is emerging as a significant area now in Alzheimer’s research. The Alzheimer’s Association’s Part the Cloud initiative, for example, is making the development of new therapies in this area a target of multi-million dollars in research funding.

Newly launched is Alzheimer’s Combination Therapy Opportunities (ACTO), which aims to provide pilot funding to explore combination therapy opportunities in Alzheimer’s disease. This program will support a biomarker-based clinical trial testing of repurposed drug combinations through Phase II proof of concept.

The Alzheimer’s Association also continues to prioritize issues such as the impact of sex/gender, race/ethnicity, education, and socioeconomic status on the development and progression of Alzheimer’s and other dementias.


BrightFocus Foundation Leaving No Stone Unturned in Fight against Alzheimer’s

This week, Real World Health Care talks with Diane Bovenkamp, Ph.D., Chief Scientific Officer for BrightFocus Foundation about the basic science and therapeutic research the Foundation is funding. Dr. Bovenkamp administers and ensures a high level of scientific accountability in the foundation’s research grant award program and scientific review process, and serves as a scientific liaison, fostering strong relationships with grantees, the academic research community, and other like-minded non-profits. 

Real World Health Care: It’s estimated that there may be more than 14 million Americans with Alzheimer’s disease by 2050. Why is the prevalence of this disease increasing?

Diane Bovenkamp, PhD, BrightFocus Foundation

Diane Bovenkamp, PhD, BrightFocus Foundation

Diane Bovenkamp: Part of it is demographics.  The large, but aging Baby Boom generation is now entering the risk period for this disease.  Each day, an estimated 10,000 Americans turn 65. And with today’s longer life span, this population may be living with the disease for longer periods than in years past.  Alzheimer’s will take a growing toll on families, taxpayers, and the economy.

Another factor may be improved diagnosis of the disease. We can expect that as diagnostic tools get better—and there is less of a stigma surrounding the reporting of Alzheimer’s as a cause of death—we will identify more people with the disease.  These numbers may fluctuate, however. We may discover that some conditions that are currently being called Alzheimer’s disease are actually other types of dementias that are new to us.  In short, the way we look at or diagnose Alzheimer’s may change.

RWHC: Where is most of the Alzheimer’s disease treatment research being focused in this country: treatments to mitigate symptoms, treatments to cure the disease, to stop or slow the progression of the disease, or treatments to prevent the disease?

DB: Right now there are no drugs to stop Alzheimer’s, only ones that mask symptoms, at best.  Research is showing that there is no magic bullet or single pathway to attacking Alzheimer’s.  Our understanding of the disease is likely to come from multiple lines of inquiry. And we just don’t have the time to limit the scope of inquiry.

There are a few trends, however, in the direction of new research.  In recent years, BrightFocus has seen a dramatic shift towards prevention studies, or the examination of any activities, foods, or therapies that might reduce one’s risk of the disease. For example, researchers are investigating the ties between reducing Alzheimer’s risk by reducing one’s risk of heart disease or diabetes.  One could say that “what’s good for the heart may be good for the brain.”

Diagnosis or early detection is another area of study.  Because disease symptoms may not appear until decades after the disease has begun, medical scientists have to find ways to detect and intervene early in the course of the disease—which at a minimum may slow or stop disease progression and its symptoms.

Advances in biomarkers, the indicators of a condition, have been huge in the last decade.  Our design of clinical trials is evolving too.  Cheaper, faster, and statistically rigorous designs are being used, and the use of repurposed drugs is also speeding up the discovery process.

RWHC: Where does BrightFocus Foundation concentrate its research funding?

DB: Our research support is investigator initiated, meaning that we don’t dictate what topics to pursue, but offer our support—through a rigorous, peer-reviewed selection process—for any promising research idea from any investigator around the world.  Our philosophy is that we will leave no stone unturned in the effort to end Alzheimer’s disease.

BrightFocus is currently managing a portfolio of 74 Alzheimer’s research projects around the globe and, since inception, we have funded more than $100 million in Alzheimer’s research.

Our funded projects cover the usual range of topics including the roles of Amyloid Beta (ABeta), tau, and ApoE, as well as subjects like vascular factors, inflammation, cell death, bioenergetics, and neuroplasticity.

We encourage researchers to take risks that might not be funded elsewhere.  We “bridge the gap,” by supporting many scientists early in their career, who then go on to receive an average of ten times more funding from larger organizations such as the National Institutes of Health.

BrightFocus is known for its support of basic science, which is critical when we still have so much to learn about this disease. However, we have seen an increase in the study of potential therapies, with more translational (patient care) and clinical research projects each year. Drug discovery and diagnosis were two of the popular topics in our fiscal year 2016 grant awards, which will be announced later this summer.

You can read more about currently active research projects on our website.

RWHC: What are some of the most important Alzheimer’s research breakthroughs that the BrightFocus Foundation has funded?

DB: We funded two researchers who went on to become Nobel Laureates.  One is Stanley Prusiner, M.D., of the University of California at San Francisco (1997 Laureate) for research on how improperly folded protein can lead to disease, which has profoundly affected our knowledge of Alzheimer’s. The other is Paul Greengard, Ph.D. (2000 Laureate), of the Rockefeller University. His research clarified the various parts of the signaling machinery in the brain and thus paved the way for the development of future therapies.

Other milestone achievements by scientists we funded include the initial identification and characterization of mutations in the familial (early-onset) type of Alzheimer’s disease [amyloid precursor protein, presenilin 1 and presenilin 2 genes], and descriptions of the connections between the immune system and amyloid.

RWHC: What are some of the more promising Alzheimer’s research programs you are currently funding?

DB: As I previously mentioned, BrightFocus has awarded more than $100 million to support promising research in fields ranging from molecular biology, genetics and drug discovery to clinical studies and epidemiology. We are currently supporting 74 outstanding Alzheimer’s projects led by innovative researchers, so it’s hard to highlight only a few of them.

Randall Bateman, M.D., at Washington University in St. Louis, is one of the original scientists to have developed a method called stable isotope labeling kinetics (SILK) to study the kinetics of proteins found in the human central nervous system. His SILK detection method for ABeta in the fluid surrounding the brain is currently the gold-standard for clinical studies. We are currently funding Dr. Bateman to develop a tau protein SILK detection method. ABeta and tau are two proteins involved in the progression of Alzheimer’s. By measuring labeled tau, they will calculate how fast the brain produces tau and clears it away.

Recent news coverage has spotlighted the research of Donald Redelmeier, M.D., of the Sunnybrook Research Institute in Toronto.  Leveraging Canadian health care records to examine a population of 300,000, he has been investigating whether use of statins reduces the long-term risk of dementia following a concussion. He is also looking at the connection between suicide risk and concussions, finding that a single mild concussion may triple the long-term risk of suicide.

And two of our funded researchers are looking at the linkage between Alzheimer’s, body weight, and metabolism.  Frank LaFerla, Ph.D., of UC-Irvine, is seeking to understand if the cognitive symptoms of Alzheimer’s and Type 2 diabetes are linked.  Makoto Ishii, MD, PhD, of Weill-Cornell Medical College in New York, is looking at why many Alzheimer’s patients show early weight loss, and trying to understand how ABeta may interact with fat hormones. Success in these two studies could help scientists find early disease biomarkers.

RWHC: What do you see as the major obstacles or challenges facing Alzheimer’s research today, and how is the BrightFocus Foundation helping to overcome these challenges?

DB: The biggest need at the moment is more funding for Alzheimer’s research. We and others estimate that a minimum of $2 billion a year in research funding is needed if the U.S. is to reach the national goal of preventing and effectively treating Alzheimer’s disease by the year 2025. Yet the US government spends far less.  Not only are we losing the potential of new discoveries, but we are becoming increasingly ill-equipped to handle the needs of patients and families touched by Alzheimer’s, along with other major health care costs.

That’s one reason why BrightFocus and other members of advocacy coalitions are working together to educate policy makers that they can’t afford to fund Alzheimer’s research at inadequate levels.  More and more families are becoming advocates for their loved ones with this disease.  We provide advocacy suggestions and publications and other resources to spread awareness of the cost of Alzheimer’s.

RWHC: Whether you are an Alzheimer’s patient or a caregiver, living with this disease is difficult. Why is it so important for patients and their caregivers to have a support community? How is the BrightFocus Foundation helping to provide such a community?

DB: Alzheimer’s disease takes a toll not only on the patient, but also on the health of loved ones and caregivers.  Both patients and caregivers benefit from meeting with others who are going through the same experiences they are. They have lots of questions and issues to address.  We provide a range of resources for families and individuals with the disease on questions to ask their doctor, financial and legal planning, housing concerns, and much more.

BrightFocus also joins forces with other advocates to increase public awareness and understanding of people with dementia.  The new Dementia Friendly America initiative, which started in Minnesota, has as its mission to educate and mobilize communities across the US about the needs of people impacted by dementia.

We have also convened a panel of experts from Johns Hopkins, the University of Michigan, Indiana University, Purdue University Indianapolis, and the Veterans Administration to examine what it means to age at home.  The objective of this Home-Based Dementia Care Panel is to raise awareness of home-based care in the context of a broader dementia care continuum, and to accelerate the development, testing, and dissemination of home-based dementia care interventions.

The panel is sparking discussion on closing the gap between patients and families in the health care system. The goal is to find out what allows people to “age in place with good quality of life and lower caregiver burden.” Something most of us want in our own life.

New Series to Launch: Alzheimer’s Disease and the Central Nervous System

According to a report from the Alzheimer’s Foundation of America, as many as 5.1 million Americans may have Alzheimer’s Disease (AD), a figure that is expected to triple to nearly 13.8 million by 2050. Alzheimer’s is the sixth leading cause of death in America, and individuals with AD use a disproportionate amount of health care resources compared to people with other diseases.

While there currently is no cure for AD, researchers are optimistic that effective treatments are on the horizon. We look forward to highlighting some of this research with you as we launch our series on Alzheimer’s disease and the Central Nervous System. We’ll also be sharing perspectives and programs from leading Alzheimer’s patient advocacy groups.

We encourage you to check back here over the coming months, and don’t forget to join the discussion on Twitter via @RWHCblog.


Big Data Declares a War on Cancer

In 1970, cancer was the second-leading cause of death in the United States. President Nixon made fighting this disease a priority in his 1971 State of the Union address: “I will also ask for an appropriation of an extra $100 million to launch an intensive campaign to find a cure for cancer, and I will ask later for whatever additional funds can effectively be used. The time has come in America when the same kind of concentrated effort that split the atom and took man to the moon should be turned toward conquering this dread disease. Let us make a total national commitment to achieve this goal.”

DataSocietyLots of great progress has been made over the past 45 years. Many challenges remain, but the technological capabilities have vastly improved.

In his last State of the Union address, President Obama re-iterated Vice President Joe Biden’s plea for a concerted effort to use the brightest minds in the U.S. to cure cancer, and announced the creation of a national cancer moonshot. President Obama asked Vice President Biden to be “in charge of Mission Control.” “For the loved ones we’ve all lost, for the family we can still save, let’s make America the country that cures cancer once and for all,” Obama said.

The good news is that today there is a massive amount available for cancer researchers to use in their mission. The challenge is that due to the lack of reporting standards and the disparate databases, much of the data is left un-analyzed, which can lead to lots of missed opportunities for breakthroughs.

Since President Obama’s declaration, Vice President Biden has met with leaders of the MD Anderson Cancer Center at the University of Texas, which in 2012, launched the Moon Shots Program aimed at reducing cancer mortality. There are many types of cancers. While they are all driven by gene mutations in various cells, every type of cancer requires a targeted approach. The Moon Shots Program has many mini-projects, or Moon Shots, aimed at treating specific cancers.

The program’s innovation is driven by the multitude of specialists involved in the project, from clinicians to biostatisticians and programmers. The Moon Shots include research into B-cell lymphoma, glioblastoma (brain cancer), cancers caused by the human papillomavirus (HPV), high-risk multiple myeloma, colorectal and pancreatic cancers, breast and ovarian cancers, chronic lymphocytic leukemia, lung cancer, melanoma, myelodysplastic syndrome/acute myeloid leukemia and prostate cancer. It covers an unprecedented number of diseases by one effort.

Cancer is a complicated ailment with complex treatments. A single tumor can have more than 100 billion cells and each cell can have different genetic mutations. The mutations are not constant over time, which requires an evolving treatment. To understand each cancer, clinicians need to understand the kinds of mutations that are driving it. There are 3 billion code letters, or amino acids, in each cell so understanding the mutations expressed in each tumor is no small task. There are as many as 300 billion opportunities for mutation in just one tumor.

With so much complexity, there are many ways to approach cancer research. For example, scientists at the NIH have used network analysis methods to map out protein interactions to discover new biomarkers and significant players in the cell’s architecture. These discoveries help guide clinical studies and other research on gene expression.

Researchers across Moon Shots programs are using machine-learning models to predict whether a patient has various types of cancer based on the expression levels of specific genes. Implementation for thyroid cancer has been especially fruitful. Thyroid cancer usually causes a lump at the base of the neck, and around 5 to 15 percent of these lumps are malignant. By measuring gene expression at the lump the machine-learning model is able to predict with greater than 90 percent accuracy whether it is malignant or benign. The work was published in Clinical Cancer Research in 2012.

Protein data is not the only kind of information used by researchers. Scientists at Case Western University have used machine-learning techniques on Magnetic Resonance Images (MRIs) of breast cancer patients to predict if a patient is suffering from aggressive triple-negative breast cancer, slower-moving cancers or non-cancerous lesions with 95 percent accuracy. Today’s capabilities of image analytics can significantly augment the insights gleaned from lab tests. The challenge with cancer is getting a full picture.

Text stored in medical records is another powerful source of relatively untapped data. Modern natural language processing capabilities can analyze massive amounts of unstructured data and combine the results with structured research and clinical information. Combining doctors’ notes versus numerical lab tests, for example, can give context to the condition and symptoms of the patient at various stages of different cancers.

Medical records include a treasure trove of data. Factors such as family histories, clinical test results and genomic data are stored in repositories across the world. The challenge is combining all that data in one database.

“Big data is not just big. The term also implies three additional qualities: multiple varieties of data types, the velocity at which the data is generated, and the volume seen within MD Anderson,” says Keith Perry, associate vice president and deputy chief information officer.

One of the ambitious objectives of the MD Anderson Cancer Center is to collect and combine patient information including a profile of their genetic makeup, clinical histories, test results, treatment courses and treatment responses. This data will be interpreted by the massive data analytics, which provide real-time decision support to rapidly improve clinical outcomes. This is a much more challenging task than meets the eye.

When the startup Flatiron Health launched with an ambitious goal to improve cancer treatment, one of the largest obstacles they faced is the inconsistency of records from various Electronic Health Record systems (EHRs).

With over $100 million in backing from Google Ventures, Flatiron is facing this basic problem: when measuring the level of a single protein commonly tested in cancer patients, a single EMR from a single cancer clinic showed results in more than 30 different formats. There are over 100 different kinds of protein and genetic tests, biopsies, and other diagnostic methods used in cancer care. And all the various EMR systems out there report these metrics in different ways. This is an incredibly complex data integration problem. So much so that Flatiron purchased Altos Solutions, which makes an EMR service for oncology practices. This allows the company to control the data collection process.

Finding cures and treatments for various types of cancer is truly a Big Data problem. And the ability to collect, store, share and analyze the data cohesively is still in relevant infancy. This isn’t a problem you can solve with just one approach. Whether using network analysis, text mining or other machine learning techniques, the task is a true inter-disciplinary challenge that requires numerous types of expertise and really Big Data.

Big Data and machine-learning don’t hold all the keys, human analysis and contextualization is key. Yet these technologies are starting to shine the light on how humanity will fight one of the most potent killers on the planet. President Nixon’s initiative gave us the Frederick Cancer Research and Development Center, an internationally recognized center for cancer research, and has achieved many breakthroughs. President Obama’s initiative has the potential to revolutionize the state of cancer treatment. We’ll make a comparison in 45 years!

Big Data in Health Care: Speaking with Nursing Leader Dr. Bonnie Westra

As part of our ongoing series on big data in health care, we spoke with Bonnie Westra, Ph.D., RN, FAAN, FACMI, Associate Professor and Director, Center for Nursing Informatics, at the University of Minnesota School of Nursing. Dr. Westra is also a member of the Institute for Health Informatics and works to improve the exchange and use of electronic health data. Here, she discusses the importance of including nursing data in big data science.

Dr. Bonnie Westra

Dr. Bonnie Westra

Real World Health Care: Why is the University of Minnesota’s School of Nursing spearheading a national action plan to include nursing data in big data science? What are your goals?

Bonnie Westra: Here at the Center for Nursing Informatics, our goal is to lead the discovery, application, and cutting edge thinking for nursing and health informatics scholarship to improve the health of individuals and communities. Nurses and the field of nursing make major contributions to health care. However, as a profession, we need to do a better job of making nursing data more useful for research purposes. We have a good foundation for how to think about nursing data, but we need to move from idea to action and develop standards for capturing, documenting and integrating nursing data with other health care information systems.

Through the Nursing Knowledge Big Data Science initiative, we seek opportunities to standardize and integrate the information nurses gather in electronic health records (EHRs) and other health information technologies. This data is the source of insights and evidence used to prevent, diagnose, treat and evaluate health conditions. The addition of rich contextual data about patients and nursing care will help us develop actionable predictive models that can increase the confidence of nursing leaders’ decisions to improve patient outcomes and safety and control costs.

The key element is having nurses involved in health information policy so that nursing data is included in clinical data warehouses for analytics and research.

RWHC: How do you differentiate between the concepts of “big data” and “big data science?”

BW: When thinking about big data, most people consider the volume, or amount of data. But you also need to think about its velocity, or the rate at which data accumulates. Variety is another hallmark of big data. In health care, that can include structured or semi-structured nursing documentation, data from monitoring devices and imaging studies, scheduling and human resource data, and patient-generated data. You also need to consider the veracity, or certainty of the data, in terms of how appropriate it is for either its original purpose — perhaps at the point of use — or for a secondary use in research and analytics.

Big data science has been defined by the National Consortium for Data Science as the systematic study of the organization and use of digital data to accelerate discovery, improve critical decision-making processes and enable a data-driven economy. It encompasses the principled acquisition, curation, exploration, manipulation and interpretation of big data sets.

RWHC: Why is it so important for nursing leaders to understand the value of big data science?

BW: The ability of nurses to make optimal clinical decisions depends on having access to accurate, real-time information, regardless of the care setting. Not only does big data have the potential for improving enterprise operating and financial performance by providing greater visibility to operational issues that support or detract from cost-effective value-based care and services. It also has the potential to improve nursing practice and patient outcomes. It can support improved decision making by offering a comprehensive and synthesized understanding of patients, nurses and organizations. The results of big data analysis enhance confidence in conclusions and can be fed back into systems as clinical or managerial decision support. Plus, it can produce a more robust, timely and valid research agenda.

Unfortunately, many nursing leaders don’t come out of school with a background in information science and informatics. They are then put in a spot where they don’t necessarily understand how to make sure data is usable.

RWHC: How can big data science help to improve positive patient outcomes?

BW: Big data science can help practitioners comply with evidence-based practice and tailoring treatment to subgroups based on patients’ unique characteristics. It also gives us the ability to understand how system characteristics such as staffing models can impact patient outcomes.

As an example, I’m currently working on a study in which we’re using EHR data to understand how compliance with the Surviving Sepsis Campaign (SSC) recommendations affects mortality and complications such as kidney or cardiovascular problems. The challenge here is to make sure data is collected and organized in a consistent way so that eventually, we can determine which SSC recommendations work best for which patients.

RWHC: What are some of the main challenges nursing leaders face in terms of accessing and utilizing big data to improve positive patient outcomes?

BW: One of the biggest issues is the lack of nurse informaticians and researchers who know how to create and harness the use of the data. Couple that with competition from other health care priorities such as meaningful use, and the fact that health systems don’t receive direct reimbursement for nursing care, and you can see how nursing-related big data becomes a lower priority. We need the health care industry to look beyond financial reimbursement to the overall value of nursing in terms of preventing adverse events and readmissions and improving patient satisfaction.

Nurses are the largest group of health care providers, and it’s critical for nurse leaders to have data to demonstrate the impact of their decisions, the value of their practice and how data can facilitate decision-making. We need to prepare nurses for the future with educational programs in informatics and involve nurses in the development of health care informatics technology.

RWHC: What type of support do you think is needed for your efforts from industry?

BW: First and foremost, software vendors in this space need to start collaborating on how to standardize data across disparate systems. A common core is needed. We’ll be exploring the topic of standardizing data and processes at the upcoming Big Data Science Conference, June 1-3 in Minneapolis. I invite all Real World Health Care audience members to attend and learn how they can contribute to the future of a national big data science initiative.