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Cancer, Chronic Disease, Clinical Trials, Science Policy

A look at crowdfunding clinical trials

A look at crowdfunding clinical trials

1024px-Assorted_United_States_coins I’ve been able to watch the crowdfunding phenomenon up close: My husband is a Kickstarter addict, and he, like millions of others, funds projects that speak to his passions and social priorities. In recent years, some non-profits have applied the crowdfunding model to clinical trials (something he hasn’t funded yet), and others may follow suit as federal-funding dollars dries up. Last week, Nature Medicine published an article that describes the first few years of those efforts and the questions they bring up.

As outlined in the piece, critics argue that the system unfairly penalizes those that may not have a large online social network to use to publicize their funding efforts, while proponents say it makes it possible for donors to connect more directly with the research and it increases transparency of research funding. As one source explains:

“One key thing is tangibility,” says Catherine Ferguson, Innovation Project Lead at Cancer Research UK, “It’s an inherent part of crowdfunding that isn’t inherent in regular funding.” Whether it’s a particular type of cancer or a particular therapy, crowdfunding allows for a “more direct relationship with both the researcher and the research,” she adds, emphasizing that this directed approach is good for maintaining relationships with donors.

Cancer Research UK, which we’ve written about before, was one of the early advocates of clinical trial crowdfunding. It recently concluded it first effort to crowdfund a clinical trial to study a vaccine for Epstein-Barr virus in cancer patients. The group fell far short of their goal, raising only six percent of the £40,000 ($61,000) goal on their Indiegogo campaign, so it returned the funds to donors. Again, from the article:

The organization chose a so-called fixed-funding model, in which they chose a goal amount but kept none of the funds that were raised if the goal wasn’t met. “It felt disingenuous to keep some of the money but not make the research happen,” said Ferguson. “We really wanted to emphasize that the money was for a specific project and if the project couldn’t be fully funded, then why keep the money?” Because the campaign wasn’t successful, the funds raised were returned to those who pledged the money, but Ferguson said that many of the donors reached out to make contributions to the organizations anyway.

Other organizations are using slightly different models, and the coming months, or maybe years, will reveal whether any are able to successfully fund clinical trials through this new avenue.

Previously: New crowdfunding sites apply Kickstarter model to health and medicineCan crowdfunding boost public support and financing for scientific research? and Crowdsourcing the identification of cancer cells
Photo by Elembis

Autoimmune Disease, Chronic Disease, Immunology, Stanford News, Videos

Chronic fatigue syndrome gets more respect (and a new name)

Chronic fatigue syndrome gets more respect (and a new name)

As has been widely reported, an Institute of Medicine (IOM) report released yesterday acknowledged that chronic fatigue syndrome is a real and serious disease and renamed the disorder “systemic exertion intolerance disease” to better reflect its key symptoms.

Stanford professor José Montoya, MD, who served as a reviewer on the IOM report, is featured in the video above, which accompanied Washington Post coverage of the development. The Post article goes on to say:

“We just needed to put to rest, once and for all, the idea that this is just psychosomatic or that people were making this up, or that they were just lazy,” said Ellen Wright Clayton, a professor of pediatrics and law at Vanderbilt University, who chaired the committee of the Institute of Medicine, the health arm of the National Academy of Sciences.

Although the cause of the disorder is still unknown, the panel established three critical symptoms for the condition (also known as myalgic encephalomyelitis):

  • A sharp reduction in the ability to engage in pre-illness activity levels that lasts for more than six months and is accompanied by deep fatigue that only recently developed.
  • Worsening of symptoms after any type of exertion, including “physical, cognitive or emotional stress.”
  • Sleep that doesn’t refresh the sufferer.

The panel also requires that a patient have one of two other disease manifestations, either cognitive impairment or orthostatic intolerance. Orthostatic intolerance is an autonomic nervous system disorder that is caused by an abnormal increase in heart rate and low blood pressure, believed to be triggered by the disease.

Susan Kruetzer, an SEID patient interviewed by Erin Allday in this San Francisico Chronicle article, expressed guarded optimism about the report’s ability to generate more research funding and patient support, saying “What I want to see is someone in Congress get pretty riled up by this report — have them see how many people are affected, how these people are really ill, how they’ve been mistreated,” Kreutzer said. “I’d just like to light a fire. I don’t know if this report will do that, but I suppose it gives us some ammunition.”

Previously: Some headway on chronic fatigue syndrome: Brain abnormalities pinpointed, Unbroken: A chronic fatigue syndrome patient’s long road to recovery and Deciphering the puzzle of chronic fatigue syndrome

Cardiovascular Medicine, Chronic Disease, Research, Stanford News, Surgery, Transplants

Growing number of donor hearts rejected for transplantation, Stanford study finds

Growing number of donor hearts rejected for transplantation, Stanford study finds

KhushAs a health writer, I’ve interviewed and written about numerous heart patients whose lives were saved when someone else died and donated their hearts for transplantation.

Those patients expressed both the anguish of hoping and praying for a new heart — when that means someone else has to die — and the overwhelming gratefulness for those donor hearts that saved their lives.

So when I wrote a story about a new Stanford study that shows an increasing number of donor hearts being rejected for transplantation, it struck a chord.

The study, published today online in the American Journal of Transplantation, found that the number of hearts rejected for transplant by surgeons and transplant centers is on the rise despite the growing need for such organs. As cardiologist Kiran Khush, MD, the lead author of the study, said in my story on the work, “We’ve become more conservative over the past 15-20 years in terms of acceptance, which is particularly troubling because of the national shortage of donor hearts and the growing number of critically ill patients awaiting heart transplantation.”

Khush and her colleagues sought to study national trends in donor-heart use by examining data from the federal government’s Organ Procurement and Transplantation Network on all donated hearts from 1995-2010. Of 82,053 potential donor hearts, 34 percent were accepted and 48 percent were declined. The remainder were used for other purposes such as research.

The researchers found a significant decrease in donor heart acceptance, from 44 percent in 1995 to 29 percent in 2006, which rebounded slightly to 32 percent in 2010. They also found, as I wrote in the story:

Among a portion of donor hearts that are referred to as “marginal” — those with undesirable qualities, such as being small or coming from an older donor — their use in transplantation varied significantly across geographical regions depending on choices made by the surgeons and the transplant centers.

The study explored possible reasons for so few organs being accepted. Increasing scrutiny by regulatory agencies of the 140 or so transplant centers across the country may have had the unintended result of making surgeons and centers more risk averse and as a result reject more hearts. Also, an increasing us of mechanical circulatory support devices that help keep patients alive while waiting for donor hears, may cause surgeons to wait longer for “better hearts.”

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Big data, Cardiovascular Medicine, Chronic Disease, Research, Stanford News

Big data used to help identify patients at risk of deadly high-cholesterol disorder

Big data used to help identify patients at risk of deadly high-cholesterol disorder

Familial hypercholesterolemia is not exactly a catchy name. But Stanford cardiologist Josh Knowles, MD, is determined to make it easier to remember. This little known, high-cholesterol disease is a silent killer. If you don’t know you have it, it can strike suddenly – and years before most people ever start worrying about heart attacks.

Knowles and fellow researchers at Stanford have launched a new research project aimed at identifying people at-risk of having FH. Using “big data” research methods and software that “teaches” a computer how to recognize patterns, researchers plan to comb through electronic medical records at Stanford hospitals and, if successful, pinpoint those who might have the disease and not know it.

In a story I wrote on the new project, Knowles described how this innovative technology could potentially be used to transform health care:

Machine learning, in which computer algorithms learn to recognize patterns within data, is widely used by Internet businesses such as Amazon and Netflix to improve customer experience, get information about trends, identify likes and dislikes and target advertisements. These techniques have not been widely applied in medicine, but we believe that they offer the potential to transform health care, particularly with the increased reliance on electronic health records.

Using these methods to help identify patients with FH is a good place to start, Knowles said, since there are currently few systematic approaches to finding people with FH, and many doctors are unfamiliar with the disease. As he told me:

This disorder certainly leads to premature death in thousands of Americans each year … Less than 10 percent of cases are diagnosed, leaving an estimated 600,000 to 1 million people undiagnosed. If found early enough and treated aggressively with statin-based regimens, people can live longer, healthier lives.

The project is part of a larger initiative called FIND FH (Flag, Identify, Network, Deliver), a collaborative effort involving Stanford Medicine, Amgen Inc., and the nonprofit Familial Hypercholesterolemia Foundation to use innovative technologies to identify individuals with the disorder who are undiagnosed, untreated, or undertreated.

Previously: Registration for Big Data in Biomedicine conference now open, Hope for patients with familial hypercholesterolemia and Born with high cholesterol
Photo by Dwight Eschliman

Chronic Disease, Medicine and Society, Mental Health

Boldly Me: Bay Area non-profit helps build self esteem in those who feel different

Alanna headshot

Alanna Powell

Boldly Me is the dream-come-true of founder Alanna Powell, who was diagnosed with Alopecia Universalis as a toddler and consequently spent the majority of her life completely bald. Powell has built upon her own experience with shaky self-confidence and self-doubt to support and inspire those who “feel different.”

I recently had the chance to talk with Powell, who founded the Fremont, CA-based organization in 2009. Boldly Me programming, which aims to improve the psychological health and self-esteem of both kids and adults, takes place around the Bay Area: The Fremont Unified School District has widely adopted it, and Powell reports that Stanford physician Joyce Teng, PhD, MD, is advocating for it to reach Palo Alto schools.

Boldly Me offers a variety of classes and assembly presentations teaching self-compassion and communication, starting with one called “Me and my thoughts.” Programming also includes activities like hula dancing, baton twirling, and arts and crafts, and community events showcase the kids’ talents and celebrate individuality. The company bases its approach on cognitive and dialectical behavioral therapy, and it has volunteer psychologists and doctors on its advisory board.

image

Kids dance hula at a Boldly Me event

While the core of the program is creating personal compassion and self esteem to enable internal resistance to external reactions, it also approaches the problem from the outside by educating peers and parents. However, Powell says that programming directed at parents is often met with defensiveness, so Boldly Me starts with the kids by teaching them how to communicate problems to parents. Once the parents receive this information, they are receptive to suggestions.

Powell’s personal story was initially a big part of the programming; participants found it inspiring. When I spoke with her, she shared how her parents, with the best of intentions, were overprotective and encouraged her to always wear a wig in company. Her self-consciousness limited her in school, and as a young woman she was afraid to get married and have kids because she worried that a bald woman was unlovable. Her now-husband inspired her by saying that if she was afraid of her wig falling off, she should just “go bald!” She began to do things she never thought she would, such as river rafting and roller coasters. But the process of self-confidence is gradual – she wore her wig during labor, nervous to meet her new baby. Now her kids – ages 13, 11, and 9 – talk about their “cool bald mom!”

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CDC, Chronic Disease, Health Policy, In the News, Infectious Disease, Public Health

To screen or not to screen for hepatitis C

Hep CIn the past few years, newer, more effective treatments have been introduced for hepatitis C – a disease that can lead to chronic liver problems and in the worst cases, liver cancer. In 2012, the Centers for Disease Control and Prevention recommended screening for the disease in anyone born between 1945-1965, since about three-quarters of cases occur in this age group, the Baby Boomers. Last year, the World Health Organization also called for more screening for the disease.

But in a recent analysis piece in The BMJ (formerly the British Medical Journal), several scientists, including Stanford epidemiologist John Ioannidis, MD, DSc, lay out the case that universal screening in this age group may not be warranted. A story in the San Francisco Chronicle today quotes Ioannidis:

“The question is whether these aggressive screening policies are justified and whether they would result in more benefit than harm,” said Dr. John Ioannidis...“We know very little about the potential harms of these drugs, especially in the long-term. And we don’t know how they will translate into long-term benefits.”

Ioannidis and his colleagues suggest that instead of rolling out widespread screening programs, researchers, as soon as possible, start a randomized trial to test the usefulness of screening and who may benefit from it.

On top of the medical uncertainties of the new treatments, they’re expensive, costing about $84,000 for the 12-week treatment. But they’ve been shown to cure patients of their hepatitis C infections at the end of that 12 week stint. Not all people who contract the disease will develop chronic infections, but a majority – two-thirds -will. Twenty percent of those cases will go on to develop severe liver disease.

Advocates of universal screening say that the new screening strategy could identify many people who don’t know they’re sick – symptoms from hepatitis C chronic infections can take years to manifest. But Ioannidis and his colleagues note that many people will get unnecessary treatment and that the long-term uncertainties of the treatment should be taken into consideration.

Previously: Despite steep price tag, use of hepatitis C drug among prisoners could save money overallA primer on hepatitis CFor patients with advanced hepatitis C, benefits of new drugs outweigh costsDrugs offer new hope for hepatitis C and Program examines hepatitis C, the “silent epidemic”
Photo of hepatitis C virus by AJ Cann

Chronic Disease, Mental Health

The art of healing

The art of healing

painting a heart

We’ve partnered with Inspire, a company that builds and manages online support communities for patients and caregivers, to launch a patient-focused series here on Scope. Once a month, patients affected by serious and often rare diseases share their unique stories; this month’s column comes from a patient advocate from Syracuse, New York.

“There’s no cure or additional treatment as all viable options are exhausted. This is your new normal.”

My gastroenterologist was stating the obvious, yet I stared at him with incredulity. I’d had my second pancreatectomy a year prior, and it failed to control my critical and idiopathic hypoglycemia and pain. I was now receiving my nutrition via IV since repeated surgeries had reduced my ability to digest and absorb food, creating erratic swings in blood sugar necessitating constant monitoring. I was stunned that this was how things would be from now on.

I cried after that appointment as images of my life prior to getting sick flashed before my eyes. Until three years earlier, I had been working as a registered dietitian and diabetic educator, I cared for two busy teens, and I lived a life rife with joyful spontaneity. After a few week of impenetrable sadness I raged at God for condemning me to this life of constant pain and uncertainty. Why me? How could I be so sick when I lived such a healthy and active lifestyle? I knew I needed help to cope with this transition. Chronic illness was a dark and unfamiliar place. At 50, my life as I knew it was over.

I had always been a bibliophile, and I used audio books when I was too ill to read. Music was inspiring and while I had countless songs on my iPod it didn’t fill my days. My counselor gently encouraged me to try something new, so I decided to visit my local art store and see if there was anything of interest. I’d taken classes over the years but didn’t consider myself an artist, so I walked through the door with trepidation. But I was immediately welcomed by a knowledgeable artist who took me in hand. We walked down the aisles and she spoke with ardent enthusiasm about the different mediums available to me. When I saw the rainbow of acrylic paints I made my decision: I was going to paint.

Immediately following my purchase, I had buyer’s remorse. What did I know about painting? I had been encouraged to pursue excellence, and this was akin to jumping off the proverbial cliff. It took two weeks before I prepared my first canvas and put brush to paper. And what transpired next surprised me.

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Aging, Chronic Disease, Ethics, Health Policy

Exploring the value of longevity with bioethicist Ezekiel Emanuel

baby hand in old hand - big

In a popular article in the Atlantic published this past fall, Ezekiel Emanuel, MD, bioethicist and prominent federal policy adviser, made a controversial case against longevity. Just before the recent holidays, the USC Annenberg School for Communication & Journalism sponsored a webinar during which Emanuel explained and elaborated on his message. It’s not that he “hopes to die at 75,” as the title chosen by the Atlantic suggested, but he wants a life at 75 focused on living, not on living longer.

Emanuel essentially argues for quality of life over quantity of years, and he claims that American society is becoming obsessed by the latter. I listened in on his December talk, which began with an ad from the AARP that glorifies “going on forever” – a value he says is reflected in both the medical system and our contemporary culture.

He directed his scientific criticism against the idea of the “compression of morbidity” – the belief that with enough medical advances, disabilities will go away and people will live in good health until, more or less suddenly, they die. He calls this the “rectangularization” of life: falling off a cliff instead of rolling down a gradual decline. The idea is immensely popular and money-making, but while some research claims to prove it, far more proves the opposite. There is, in fact, an expansion of morbidity as people live longer, an elongated and more gradual decline with more disabilities and less and less creativity.

Given this, Emanuel’s hope and recommendation is two-fold: that people will consider what makes their life meaningful and spend their energy cultivating that, and that medical resources will be redirected from prolonging life to improving its quality at the outset – by reducing the country’s exceptionally high rate of premature births, for example.

American life expectancy is at an all-time high at nearly 80 years, but are octogenarians living meaningfully? Last month in the New York Times, David Brooks argued against Emanuel’s earlier piece by saying that the “happiest people” are ages 82-85; in the webinar, Emanuel says this misses the point. It’s much easier to measure happiness than meaningfulness, yet the latter is the real aim of life (and, furthermore, Brooks didn’t account for the perspectives of those in nursing homes, assisted living, or suffering from dementia). Emanuel talked of patients who say cancer was “the best thing that happened to me” because it made them focus on what was meaningful in their life. The end of life is important to think about, as it helps us see what we want from life now.

“Contorting life around living as long as possible seems to me to be counter-productive,” he said. “What we should be focusing our life on is what’s meaningful, how we enrich other people and contribute to our families and society.”

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Chronic Disease, Nutrition, Obesity, Videos

The role of nutrition in diabetes prevention and management

The role of nutrition in diabetes prevention and management

Can certain diets help patients prevent or manage their diabetes? Which foods are best for diabetics and which ones should they avoid? If you increase your coffee consumption, will it reduce your risk of diabetes? Kathleen Kenny, MD, a clinical associate professor at Stanford, and Jessica Shipley, a clinical dietitian at Stanford Hospital & Clinics, answered these questions and others about diabetes and nutrition in a recent Stanford Health Library talk.

In the above video, Kenny and Shipley also discuss the glycemic index and how it should be used to tailor dietary choices; examine how Mediterranean, low-carb and low-calorie diets affect diabetes; and explain how eating healthy can prevent or reverse the disease. The lecture is a must-watch for anyone wanting to make healthier food choices to benefit their health.

This video is the final lecture in a three-part series addressing important questions related to diabetes and lifestyle choices.

Previously: Diabetes and nutrition: Healthy holiday eating tips, red meat and disease risk, and going vegetarian, Diabetes and nutrition: Why healthy eating is a key component of prevention and management, Diabetes self-management program helps at-risk teens and their families make healthier choices and New evidence for a direct sugar-to-diabetes link, Examining how diet soft drinks impact your health

Chronic Disease, Health and Fitness, Videos

The importance of regular exercise in delaying and treating diabetes

The importance of regular exercise in delaying and treating diabetes

Looking for motivation to exercise regularly? Consider this statistic: People who engage in physical activity for seven hours a week have a 40 percent lower risk of dying early than those who are active for 30 minutes or less a week. Among the many health benefits bestowed is helping prevent and manage diabetes.

In this Stanford Health Care video, Baldeep Singh, MD, a clinical professor at Stanford who focuses on chronic disease management, explains how exercise can lower blood sugar during your workout and afterwards and help insulin work better. He says in the talk:

[Exercise] is one of the most important things that you can do as you get older because it really has all plus and no minuses … One of the keys is consistency. You want to be consistent in your regiment – even the same time of day … It’s much better to make small incremental changes and be consistent with them, than to make a huge change and than do nothing the rest of the year.

This video is the second lecture in a three-part series addressing important questions related to diabetes and lifestyle choices.

Previously: Examining the role of exercise in managing and preventing diabetesWithout exercise, Americans are growing more obese, according to Stanford researchers, Preventing pre-diabetes from turning into diabetes and Fighting a fatalistic attitude toward diabetes 

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