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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

Cancer, Stanford News

For this doctor couple, the Super Bowl was about way more than football

For this doctor couple, the Super Bowl was about way more than football

Paul and Lucy at Super Bowl - smallEarlier this month, football fans across the world watched as the New England Patriots shocked the Seattle Seahawks with a very dramatic last-minute win. While the game itself was a thrill, equally as exciting for two people in the seats at University of Phoenix Stadium was what had gotten them there. Neurosurgeon Paul Kalanithi, MD, and his wife, Lucy, had won a trip to the big game by raising money for lung-cancer research and winning the Lung Cancer Survivors Super Bowl Challenge, sponsored by the Chris Draft Family Foundation.

Kalanithi had attended Stanford as an undergrad in the 90s, the same time as did Draft, a former professional football player who later started his foundation and whose wife, Keasha, died of lung cancer in late 2011. Kalanithi received a diagnosis of lung cancer in 2013 and re-connected with Draft not long after.

“The foundation is putting a new face on the disease,” Lucy Kalanithi, MD, a clinical instructor in general medical disciplines at Stanford, told me during a recent conversation. Team Draft, an initiative of the foundation, puts the spotlight on, and brings together, young lung-cancer patients such as Paul Kalanithi, with the aim of getting out the message that anyone can get lung cancer. It’s also working to stop the smoking stigma from negatively impacting research funding for lung cancer.

Paul at Super Bowl - small“Even though Paul and I are both physicians, prior to his diagnosis, neither of us was fully aware of the global toll of lung cancer and the major gap in federal and private funding due to the anti-smoking stigma,” Lucy Kalanithi said. “More people die from lung cancer than from breast, colon and prostate cancers combined: It’s the top cancer killer.”

I asked if her husband had ever experienced the sense of judgment or blame that can come with a lung-cancer diagnosis. “Paul’s never had the experience – common among lung-cancer patients – of being asked, ‘Did you smoke?’ Kalanithi said, noting that her husband was never a smoker. “But everyone with lung cancer is affected by the anti-smoking stigma, because it means that much, much less money goes to lung cancer research compared with other cancers. And survival rates for all cancers are directly related to research funding. When people think of breast cancer, they think of a sympathetic character like a young mom. But when people think of lung cancer, they don’t think of a vibrant young dad like Paul.”

Through the foundation, the Kalanithis connected with other young families affected by lung cancer (“There’s a lot of camaraderie and optimism,” Kalanithi told me), and when they learned of the Super Bowl Challenge, a friendly fundraising competition among lung-cancer survivors, they jumped at the chance to compete. There was an “overwhelming response from Paul’s friends, family and colleagues – including many from Stanford,” Kalanithi said, which led to a call from Draft on New Year’s Day. They had won the challenge, Draft told the couple, and they would be attending not only the Super Bowl but also Taste of the NFL, a fundraiser attended by former NFL players and renowned chefs from around the country, and an exclusive pre-game stadium tour. As icing on the cake: Their (too-cute-for-words) seven-month-old daughter, Cady, would be making the trip with them.

Kalanithis at Super Bowl - smallWhen I asked Kalanithi for a sampling of the moments etched in her mind from the weekend, she offered two: lying on the Super Bowl field and getting a photo taken with her husband and baby daughter forty-eight hours before the game (“It was surreal”) and watching Paul, a huge football fan, “jump up and down” in their incredible seats on the Seahawks’ 50-yard line. (For the record, they were rooting for the Seahawks. And next year, “we hope to see [Stanford alum] Andrew Luck out there.”)

Despite the excitement of this once-in-a-lifetime experience, the Kalanithis’ relationship with Team Draft seemingly extends far beyond the football field. Kalanithi has noted that the foundation has “helped boost our family’s spirits during this challenging time,” and she sounds eager to partner with Draft on other initiatives. “Helping raise awareness and research funds impacts families everywhere, and it gives me hope,” she said.

Previously: Tackling the stigma of lung cancer – and showing the real faces of the disease, A neurosurgeon’s journey from doctor to cancer patient“Stop skipping dessert:” A Stanford neurosurgeon and cancer patient discusses facing terminal illness and A Stanford physician’s take on cancer prognoses – including his own
Photos courtesy of Lucy Kalanithi

Cancer, Research, Stanford News

The Big Bang model of human colon cancer

The Big Bang model of human colon cancer

big bangLike the Big Bang model of the formation of the universe, the Big Bang model of human colon cancer deduces tumor conditions in the past based on current data. Instead of cosmic radiation, the cancer model uses genomic data from a mature tumor to infer how it grew, starting from when it was composed of a small number of mutated cells.

Christina Curtis, PhD, a recent addition to Stanford’s School of Medicine, and her collaborators at the University of Southern California developed the model, which was published online this week in Nature Genetics.

Testing the Big Bang model confirmed that most detectable differences in tumors come from early disordered growth patterns of cells.

As I wrote in a story about the Big Bang model:

Using an array of genomic techniques, Curtis and her team profiled colorectal tumors at multiple spatial scales, ranging from single cells to tumor glands consisting of fewer than 10,000 cells, as well as “bulk” tumor fragments that were taken from opposite sides of a full-grown tumor. These methods provided a panoramic and high-resolution view of how cells within a tumor were different and how tumors from the same patient differed from one another. From the genomic data, the researchers reconstructed a tumor growth history. A tumor growth history can be thought of like a slide-show at a graduation party, which starts off with baby pictures and ends with images of the young adult. The Big Bang model describes how a tumor evolves from a few thousand cells to a full-grown tumor composed of billions of cells.

Carcinomas had disordered growth histories — identifiable even when the tumors were just a few thousand cells — and benign adenomas had ordered growth histories. Curtis told me that these findings suggest that some tumors are “born to be bad” and the malignant potential of a tumor is determined early. She added that disordered growth patterns identified in emerging tumors could potentially serve as a biomarker, enabling early detection of cancerous growths.

Kimberlee D’Ardenne is a writing intern in the medical school’s Office of Communication and Public Affairs.

Previously: Stanford researchers explore new ways of identifying colon cancer, Bacterial balance in gut tied to colon cancer risk, Study shows evidence-based card eliminates racial disparity in colon-cancer survival rates and Researchers explore colonoscopy’s effect on the incidence of colorectal cancer
Image by Atilla Szűcs

Cancer, Public Health, Science

Research institute or detective agency? Investigating the “perp” known as cancer

Sherlock Holmes2After graduating from college, I accepted a job as a white-collar crime analyst for the Iowa Bureau of Criminal Investigation. It was an exciting first job for a 21-year old. I worked closely with a team of highly trained and dedicated public safety officers to help detect and prosecute white-collar criminals. My specific role was to identify patterns in criminal activity and assist in building a narrative so others could understand how and why the crime was committed.

Little did I know how closely that job would parallel my current one.

Forty years later, I find myself leading a different type of “detective agency,” the Cancer Prevention Institute of California (CPIC). CPIC employs highly skilled researchers who function a lot like private investigators. They are epidemiologists – scientist-sleuths who examine trends and patterns in the population to identify risk factors and causes and effects of disease, and their work is anything but “elementary.”

As curious and persistent as any detective, epidemiologists are driven to solve challenging public-health cases. At CPIC we pursue understanding the traits and tendencies of a particular perpetrator: cancer. For example, our researchers look at how cancers occur geographically, examining incidence and mortality rates by specific regions across California and the U.S.

“Data! Data! Data!” Sherlock Holmes cried impatiently in The Adventure of the Copper Beeches. “I can’t make bricks without clay.” Well, like Holmes, our scientists also need data; lots of it. CPIC maintains the population-based registry of all Greater Bay Area cancer cases, as mandated by California state law. The registry is a deep source of information on the approximately 30,000 new cancer cases diagnosed each year across our nine-county area. To date, more than 850,000 Greater Bay Area cancers have been registered. Through this and other data bases, our researchers are able to “investigate” a wide range of cancers, from the most common to the rarest forms, and examine important evidence linking cancer risk with such factors as race/ethnicity, genes, environment, migration status and lifestyle.

Has our scientists’ sleuthing paid off? Definitely. To illustrate, CPIC’s researchers and their colleagues at the Stanford Cancer Institute (SCI) recently found that a double mastectomy does not improve survival over the less invasive option of lumpectomy plus radiation for the average breast cancer patient, contributing important information for breast cancer patients and their physicians worldwide as they evaluate their treatment options. Research conducted by CPIC and SCI scientists also detected alarming rates of deadly melanomas in Californians, as cited in 2011 legislation that made California the first state to ban the use of tanning beds by minors. CPIC researchers also discovered that California nail salons had higher than expected levels of carcinogens, identifying a need for better health standards.

And, yes, just like Adrian Monk or Colombo, we have some quirky scientists who help make coming to work both mighty interesting and very fulfilling. If you come to visit us at CPIC, you just might spy a rumpled raincoat or two.

Donna Randall, PhD, is Chief Executive Officer of the Cancer Prevention Institute of California.

Previously: Breast cancer patients are getting more bilateral mastectomies – but not any survival benefit, Gel polish: What risks lie beneath painted beauty? and New law: No more tanning beds for California teens
Photo by dynamosquito

Cancer, Health Disparities, Patient Care, Research, Stanford News

Study shows evidence-based care eliminates racial disparity in colon-cancer survival rates

Study shows evidence-based care eliminates racial disparity in colon-cancer survival rates

For the past two decades, the National Cancer Institute has documented that African-American patients have consistently had lower survival rates in colon cancer when compared with white patients. In a study published today in the Journal of Clinical Oncology, lead author Kim Rhoads, MD, PhD, and colleagues from Stanford show that receiving high quality, evidence-based treatment can eliminate this racial disparity. As Rhoads explains in our press release:

Historically, we’ve taken less than a critical eye on our own health-care system in terms of how we can take the lead in addressing disparities. The big take away in this paper is that it’s treatment, not necessarily patient factors, but following evidence-based guidelines that gives all patients the best chance for survival. Our work also suggests a real opportunity to equalize these racial differences.

The evidence-based guidelines were created by the National Comprehension Cancer Network, which used clinical trials and medical research to create step-by-step, evidence-based treatments for most cancers. However, adherence to those guidelines depends on the facility and research shows that minority patients tend to receive care from hospitals that have low adherence rates.

The study found that integrated health-care organizations, which provide all of a patient’s health-care services, hospital care and insurance, delivered evidence-based care for colon cancer at a higher rate than non-integrated health-care organizations. In these facilities, all patients had higher survival rates and racial disparity for colon cancer survival disappeared.

“In integrated systems, there’s already a big push to thinking about following evidence-based guidelines, so everyone within that system is in the same mindset,” said co-author Manali Patel, MD, MPH. “It’s easier to do the right thing when you have the system-level support to do so.”

Integrated health-care systems are well suited for coordinating care among several specialists, which is another advantage for colon-cancer patients, because the treatment of colon cancer requires different types of treatments and different types of specialists, the study pointed out.

The results support the development of integrated health care models as envisioned by Affordable Care Act.

“With health-care reform, millions more patients are coming into the system, and we’re going to need to become more integrated in order to meet the demand. We’re going to need to work more closely together, decrease variations in care and standardize what we do,” Rhoads told me. “In this paper, we have a model that shows that when you do this, you get better colon cancer outcomes for everyone.”

Previously: Stanford researchers examine disparities in use of quality cancer centers, Uncommon hero: A young oncologist fights for more humane cancer care and Report shows continuing health disparities for racial and ethnic minorities

Applied Biotechnology, Bioengineering, Biomed Bites, Cancer, Imaging, Technology, Videos

Beam me up! Detecting disease with non-invasive technology

Beam me up! Detecting disease with non-invasive technology

Here’s this week’s Biomed Bites, a feature appearing each Thursday that introduces readers to Stanford’s most innovative biomedical researchers.

Star Trek fans rejoice! Stanford radiologist Sam Gambhir, MD, PhD, hopes that someday he’ll be able to scan patients using a handheld device — similar to the one used by Bones in the popular sci-fi series — to check their health.

“Our long-term goals are to be able to figure out what’s going on in each and every one of you cells anywhere in your body by essentially scanning you,” Gambhir said in the video above. “We’ve been working on this area for well over three decades.”

This is useful because it will help doctors diagnose diseases such as cancer months or even years before the symptoms become apparent, Gambhir said.

And these advances aren’t light-years away. “Many of the things we’re doing have already started to move into the hospital setting and are being tested in patients. Many others will come in the years to follow,” he said.

Gambhir is chair of the Department of Radiology. He also directs the Molecular Imaging Program and the Canary Center for Cancer Early Detection.

Learn more about Stanford Medicine’s Biomedical Innovation Initiative and about other faculty leaders who are driving biomedical innovation here.

Previously: Stanford partnering with Google [x] and Duke to better understand the human body, Nano-hitchhikers ride stem cells into heart, let researchers watch in real time and weeks later and Developing a new molecular imaging system and technique for early disease detection

Cancer, Complementary Medicine, Events, Patient Care

Knitting needles cancer while helping patients

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It may sound unusual, but knitting is one way to cope with difficult experiences, such as undergoing cancer treatment. Rhythmic and relaxing, knitting can sooth the mind and soak up the downtime that’s a big part of cancer treatment, according to Holly Gautier, RN, a nurse and director of the Cancer Supportive Care Program at Stanford.

“It’s the repetitive motion that you have with knitting… You’re focused on the stitching and your mind becomes somewhat blank – it really feels good to be making something new,” Gautier explained to me recently.

Although she administers a slew of programs – from yoga to art – Gautier said she’s particularly excited about a new knitting class, which meets weekly at the Stanford Cancer Center.  It’s free and open to all cancer patients and their families — not just those being treated at Stanford.

The class is led by a volunteer knitters, who provide supplies and teach the basic stitches. They can even accompany patients to treatment rooms to answer questions or undo an error, Gautier said. And they’re happy to put together “knitting-to-go” care packages for those who can’t stay.

While participants are welcome to work on other projects, such as scarves and hats, the class is currently making squares to create a quilt to raffle off at an upcoming benefit for the Cancer Survivorship Program. Gautier said the quilt project provides patients with an opportunity to give back – something that nearly all patients yearn to do.

Although the first session last Tuesday drew eight female patient-knitters, Gautier said she hopes other patients and caregivers, particularly men, stop by in coming weeks. More details on the Knitting with Friends program can be found here.

Previously: Knitting as ritual — with potential health benefits?, Image of the Week: Personalized brain activity scarves and A look at how helping others can be healing
Photo by meknits

Aging, Cancer, Research, Stanford News

Stanford researchers deliver double punch to blood cancer

Stanford researchers deliver double punch to blood cancer

Acute myeloid leukemia is an aggressive and deadly cancer affecting cells that turn into our blood. Now, a study published in Nature Medicine shows that a drug known to cause cell death might be effective for a particular subtype of this lethal disease.

To get an idea of just how aggressive and deadly acute myeloid leukemia is, consider the survival rates for the 13-14,000 American adults who are sickened each year. The overall survival rate is 30-40 percent, according to Stanford cancer researcher Ravindra Majeti, MD, PhD, but if patients are over 65 years old, the survival rate dips to just 5 percent. The majority of acute myeloid leukemia patients are elderly.

Apoptosis, or cell death, of tumors is the goal in cancer treatments. There are multiple pathways leading to cell death, and identifying ways to nudge cancer cells towards dying is the focus of much cancer research.

But sometimes the path to cell death is nonlinear and hard to find.

In this work, a team of Stanford cancer researchers led by Majeti and Steven Chan, MD, identified a two-pronged attack for acute myeloid leukemia cancer cells. The researchers first focused on mutated proteins called isocitrate dehydrogenase 1 and 2 (known as IDH1/2 for short). Cancer cells containing mutated IDH1/2 proteins often survive traditional chemotherapy treatments, contributing to relapse, and they exist in 15 percent of acute myeloid leukemia patients. The second focus of the Stanford researchers was the BCL-2 gene, which is known to enable cancerous growth by putting the brakes on cell death in acute myeloid leukemia and other cancer cells. Simply stopping BCL-2 activity in acute myeloid leukemia patients is not very effective, as indicated by low survival rates for the disease.

The Stanford scientists found that giving a drug that inhibits BCL-2 successfully lifted the blockade on cell death, but only in cells with mutated IDH1/2 proteins. Majeti said the drug that promotes cancer cell death by inhibiting BCL-2 is now in clinical trials.

Kimberlee D’Ardenne is a writing intern in the medical school’s Office of Communication and Public Affairs.

Previously: The latest on stem-cell therapies for leukemia, Blood cancers shown to arise from mutations that accumulate in stem cells and Leukemia prognosis and cancer stem cells

Biomed Bites, Cancer, Obesity, Research, Stanford News

Stanford researcher tackles tricky problem: How does a cell become a fat cell?

Stanford researcher tackles tricky problem: How does a cell become a fat cell?

Here’s this week’s Biomed Bites. Check each Thursday to meet more of Stanford’s most innovative biomedical researchers.

Mary Teruel had no intention of becoming a biology professor — after all, she was in a PhD program for aeronautical engineering. But the more she learned about cells, the more fascinated she became.

“I became very interested in the challenging problem of trying to understand the complex network in cells and trying to see if you could apply some of the principles from engineering to understand theses processes and make an insight into human disease,” Teruel says in the video above.

Teruel’s drive to investigate cells led her into her current role as an assistant professor of chemical and systems biology, where she’s striving to unravel a puzzle that underlies the obesity crisis in America: How do cells called pre-adipocytes (or pre-fat cells) become adipocytes (or adipocytes)?

By learning more about cell differentiation, Teruel’s research can also shed light on processes — and potential treatments — involved in cancer.

Learn more about Stanford Medicine’s Biomedical Innovation Initiative and about other faculty leaders who are driving biomedical innovation here.

Previously: Secrets of fat cells discovered, Fed Up: A documentary looks for answers about childhood obesity and How physicians address obesity may affect patients’ success in losing weight

Cancer, Medical Apps, Stanford News, Technology

Using a smartphone and the Folding@home app to advance disease research

Using a smartphone and the Folding@home app to advance disease research

protein

Smartphones now have the power that personal computers had a few years ago, and more and more people have them. So researchers are developing ways to harness that computing power to solve pressing biomedical problems.

As described in a Stanford News piece, Stanford’s Vijay Pande, PhD, in partnership with Sony, recently developed a smartphone app that “folds” proteins while the phone’s owner sleeps. “There are a ton of people with really powerful phones, and if we can use them efficiently, it sets the stage for something really great,” said Pande, a Stanford chemistry professor.

This particular mobile app, called Folding@home, investigates the biology of diseases, including cancer, Alzheimer’s, and Parkinson’s disease. It’s an extension of the Folding@home distributed computing project started in 2007, and it’s now available on GooglePlay.

Disease biology is dependent on proteins, which are complex linear chains of molecules that become “folded up”, like snarled balls of yarn. The chain needs to be absolutely correct; any mutation that shifts a few molecules out of place will cause the protein to not work optimally, not work at all, or, worse, work in a way that does damage to the organism.

Understanding protein configurations is key to developing cures for disease. While real proteins take milliseconds to curl up, simulating this process with computers takes thousands of hours. But if 10,000 people download and use the Folding@home app, and it runs 8 hours a day while the phone is not otherwise in use, the team’s first research question could be solved in three months.

The app’s first focus is a kinase protein found in breast cancer. It seems that different people’s tumors respond differently to the several drugs available; currently, doctors use a guess-and-check method to choose a drug, but information derived from the proteins could enable doctors to choose correctly on the first try. In something as time-sensitive as cancer, this could save lives.

Next up for the app is a project related to Alzheimer’s disease. Eventually, if enough people enroll, the researchers could launch several projects simultaneously, allowing people to choose to take part in one that is personally meaningful.

Image of a protein Argonne National Laboratory

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