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How one family’s generosity helped advance research on the deadliest childhood brain tumor

How one family’s generosity helped advance research on the deadliest childhood brain tumor

Back in February 2014, Libby and Tony Kranz found themselves at the center of every parent’s worst nightmare. Their six-year-old daughter Jennifer died just four months after being diagnosed with diffused intrinsic pontine glioma (DIPG), an incurable and fatal brain tumor. At the time, the Kranzes decided to generously donate their daughter’s brain to research in hopes that scientists could hopefully develop more effective treatments for DIPG, which affects 200-400 school-aged children in the United States annually and has a five-year survival rate of less than 1 percent.

As reported in the above Bay Area Proud segment, Michelle Monje, MD, PhD, an assistant professor of neurology and neurological sciences who sees patients at Lucile Packard Children’s Hospital Stanford, and colleagues harvested Jennifer’s tumor and successfully created a line of DIPG stem cells, one of only 16 in existence in the world. More from the story:

Using Jennifer’s stem cell lines and others, Monje and her team tested dozens of existing chemotherapy drugs to see if any were effective against DIPG. One appears to be working.

The drug was able to slow the growth of a DIPG tumor in a laboratory setting. Monje’s hope is that this treatment one day could extend the life of children diagnosed with DIPG by as many as six months.

That would have more than doubled Jennifer’s life expectancy.

“It’s a step in the right direction if we can effectively prolong life and prolong quality of life,” Monje said.

Libby Kranz says that for their family, donating their daughter’s tumor to researchers “just felt right.” She and Tony hope that by aiding the research efforts, parents and families will have more, and better quality time with their sick children.

“It’s incredible and it’s humbling,” she said, “to know my daughter is part of it, and that we’re part of it too.”

Previously: Existing drug shows early promise against deadly childhood brain tumor, Stanford brain tumor research featured on “Bay Area Proud,Emmy nod for film about Stanford brain tumor research – and the little boy who made it possible and Finding hope for rare pediatric brain tumor

Medical Education, Research, Videos

Students draw inspiration from Jimmy Kimmel Live! to up the cool factor of research careers

Students draw inspiration from Jimmy Kimmel Live! to up the cool factor of research careers

To better understand how teens feel about scientific research and to make a career in health or medicine a more desirable occupation among adolescents, University of Chicago researchers and a group of high-school students from Chicago Public Schools took a page out of the Jimmy Kimmel Live! playbook.

Using the model of Kimmel’s “Lie Witness News” segment, the predominantly minority teens asked their peers what they think about research. The project was part of the NIH-funded TEACH STRIVES program, which aims to prepare and inspire Chicago public school students to pursue careers in health-related research. Samantha Ngooi, a project manager with TEACH STRIVES, and Vineet Arora, MD, a principal investigator with the program, discuss the students’ project in a recent KevinMD post:

What did these students find when they asked their peers about research? Well, not surprisingly [the] term “research” had a largely negative connotation — “lots of paperwork,” “lab rats.” However, our teens went one step further. They found studies that would be of interest to them — about things they cared about, such as teen health with cell phone use. When presented with research that linked cell phone use at night with depression, teens on the street were inspired to learn more. Unfortunately, this idea that research is esoteric and irrelevant is common amongst teenagers. Ask your average teenager what they aspire to be and more often than not a “researcher” will not be a contender. In fact, data suggests that few high-achieving high school students are considering a career in research, let alone healthcare research.

Why is this important? To make breakthroughs in science and medicine for the future, we need a healthy pipeline of diverse, talented teens to consider entering research careers in STEM fields…

Watch above to see the full video.

Previously: High schoolers share thoughts from Stanford’s Med School 101, At Med School 101, teens learn that it’s “so cool to be a doctor” and Stanford’s RISE program gives high-schoolers a scientific boost

NIH, Obesity, Public Health, Research

Capturing the metabolic signature of obesity

Capturing the metabolic signature of obesity

scale_weightWorldwide obesity rates have more than doubled since 1980, and today the majority of the global population live in areas where being overweight kills more people than being underweight, according to data from the World Health Organization. But new research that provides a comprehensive view of the metabolic signature that may correlate with obesity could help scientists develop more effective ways to manage and prevent obesity, and it offer insights into how variability in genes, environment, metabolism and lifestyle affect our health individually.

As reported today on the NIH Director’s Blog:

The new analysis uncovered changes to 29 molecular metabolites, or biomarkers, that correlated with obesity in 1,880 people from the United States. Most of those biomarkers—25 to be exact—also turned up in the urine of obese people from the other side of the Atlantic, offering confirmation that the findings represent a shared metabolic signature of obesity.

Several of the biomarkers are byproducts of what a person eats, which may reflect differences in the diets of obese and non-obese people. For example, urine from obese people was more likely to contain a metabolite that comes from eating red meat, while thinner folks were more likely to have a metabolite indicative of citrus fruit consumption.

However, not all of the biomarkers were directly related to food. Some appeared to stem from widespread changes in kidney function, skeletal muscle, and metabolism that may occur as a person packs on extra pounds. And, intriguingly, nine of the biomarkers significantly associated with obesity weren’t even produced by the human body, but rather by the trillions of microbes that live inside our guts. Those microbial partners play important roles in the breakdown of essential vitamins, amino acids, and protein. In fact, recent research findings suggest that a significant portion of obesity risk may be explained by the activity of gut microbes. This discovery adds to mounting evidence, spurred in recent years by the NIH-funded Human Microbiome Project, for the intricate and essential role of microbes—collectively known as the microbiome—in many aspects of our health.

The piece goes on to say that the findings also “raise the intriguing possibility that people might one day be able to visit their health-care providers, receive a blood or urine test, and leave with precise, individualized information regarding their risk” for obesity and other health issues.

Previously: Childx speaker Matthew Gillman discusses obesity prevention, Discussing how obesity and addiction share common neurochemistry, Stanford team awarded NIH Human Microbiome Project grant and Obesity is a disease – so now what?
Photo by Matthew

Big data, In the News, Technology

Vinod Khosla shares thoughts on disrupting health care with data science

Vinod Khosla shares thoughts on disrupting health care with data science

14252833785_63316bba75_zProminent Silicon Valley venture capitalist Vinod Khosla is a strong believer that data science will reinvent health care as we know it – and it’s position he has reiterated on a number of occasions, including at the 2014 Big Data in Biomedicine conference at Stanford. In a recently published Washington Post Q&A, Khosla expands on his comment that over the next ten years “data science and software will do more for medicine than all of the biological sciences together.”

On the topic of books and papers that have influenced his views, Khosla said:

A lot of what I’ve been thinking about started with articles by Dr. John Ioannidis at Stanford School of Medicine. What he found through decades of meta-research is that half of what’s in medical studies is just plain wrong… His research is focused on why they are wrong and why all sorts of biases are introduced in medical studies and medical practice.

He also explains one of the reasons he believes innovation in data science and software is outpacing the biological sciences:

The pace of innovation in software, across all industries, has consistently been much faster than anything else. Within traditional health-care innovation (which intersects with “biological sciences”) such as the pharma industry, there are a lot of good reasons those cycles of innovation are slow.

It takes 10 to 15 years to develop a drug and actually be in the marketplace, with an incredibly high failure rate. Safety is one big issue, so I don’t blame the process. I think it’s warranted and the [Food and Drug Administration] is appropriately cautious. But because digital health often has fewer safety effects, and iterations can happen in 2- to 3-year cycles, the rate of innovation goes up substantially.

Previously: Countdown to Big Data in Biomedicine: Leveraging big data technology to advance genomicsCountdown to Big Data in Biomedicine: Mining medical records to identify patterns in public health, Collecting buried biomedical treasure – using big data, Big data used to help identify patients at risk of deadly high-cholesterol disorder and Examining the potential of big data to transform health care
Photo of Khosla at the 2014 Big Data in Biomedicine conference by Saul Bromberger

Big data, Events, Videos

Countdown to Big Data in Biomedicine: Leveraging big data technology to advance genomics

Countdown to Big Data in Biomedicine: Leveraging big data technology to advance genomics

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During last year’s Big Data in Biomedicine conference, David Glazer, director of engineering at Google, described how the search giant is developing technological tools to help those working in life sciences to store, process, explore, and share genomic data.

In this 2014 Big Data in Biomedicine video, Glazer explains how he and colleagues fed a computer network 10 million random YouTube videos and asked the system to look for patterns. The computer determined that most frequently occurring sequence of 1s and 0s in the sample was that of a human face. Not surprisingly, the face of a cat was the second most-frequent pattern the computer found.

While these examples of machine pattern-recognition capabilities may not be earth-shattering to those who spent an inordinate amount of time watching YouTube videos, the findings demonstrate the potential of computers to rapidly identify significant patterns in large volumes of biomedical information. Imagine researchers performing the same experiment, but instead of YouTube videos they used genomic data. “We don’t have 10 million genomes available for this type of analysis, yet,” he said. “But as we move in that direction the tools are ready.”

Watch the full presentation to learn how Google is working to remove computing restraints to advance genomic research. And check out Glazer at the 2015 Big Data in Biomedicine conference, which will be held May 20-22 at the Li Ka Shing Center for Learning and Knowledge at Stanford.

Previously: Countdown to Big Data in Biomedicine: Mining medical records to identify patterns in public healthStanford bioengineer discusses mining social media and smartphone data for biomedical research, Using genetics to answer fundamental questions in biology, medicine and anthropologyExamining the potential of big data to transform health care and Registration for Big Data in Biomedicine conference now open

Big data, Neuroscience, Videos

Countdown to Big Data in Biomedicine: Mining medical records to identify patterns in public health

Countdown to Big Data in Biomedicine: Mining medical records to identify patterns in public health

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The routine information contained in medical records holds the potential to unlock important public-health discoveries. That was the message conveyed at the 2014 Big Data in Biomedicine conference at Stanford by Martin Landray, PhD, a professor of medicine and epidemiology at Oxford University and deputy director of the Big Data Institute within the Li Ka Shing Centre for Health Information and Discovery. In the above video from last year’s event, Landray explains how he and colleagues are working to better understand the determinants of common life-threatening and disabling diseases through the design, conduct and analysis of large-scale epidemiological studies and the widespread dissemination of both the findings and methods used to generate them.

This month, Landray will return to the Big Data in Biomedicine conference and moderate a discussion on neuroimaging. Among the panelists are Michael Greicius, MD, associate professor in the Department of Neurology and Neurological Sciences at Stanford, and Brian Wandell, PhD, founding director of Stanford’s Center for Cognitive and Neurobiological Imaging and deputy director of the Stanford Neurosciences Institute.

Registration for the conference, which will be held May 20-22 at Stanford, is currently open. More details about the program can be found on its website.

Previously: Stanford bioengineer discusses mining social media and smartphone data for biomedical research, Using genetics to answer fundamental questions in biology, medicine and anthropologyBig data used to help identify patients at risk of deadly high-cholesterol disorder, Examining the potential of big data to transform health care and Registration for Big Data in Biomedicine conference now open

Events, Global Health, Health Policy, Pediatrics, Stanford News, Videos

Rajiv Shah discusses efforts to end preventable child deaths worldwide at Childx

Rajiv Shah discusses efforts to end preventable child deaths worldwide at Childx

The inaugural Childx conference was held here this month, and video interviews featuring keynote speakers, panelists and moderators are now on the Stanford YouTube channel. To continue the discussion of driving innovation in maternal and child health, we’ll be featuring a selection of the videos this month on Scope.

More than six million children under the age of five die from preventable diseases each year. During this year’s Childx conference, Rajiv Shah, MD, the former administrator of USAID, told the crowd, “I do think it’s possible to end preventable child death.”

In the video above, he explains how innovations in drug development, diagnostics and vaccines are among the solutions that are effectively reducing child mortality rates around the world. But there is still more that can be done. Using global health data to see in real-time where children are dying because of a lack of vaccines and places children are suffering as a result of poor health care, Shah said, could assist in more efficiently directing resources to these areas and other pockets of need. Watch the full interview with Shah to hear more about why he thinks ending preventable child death is achievable in the next 20 years.

Previously: Childx speaker Matthew Gillman discusses obesity prevention, Pediatric health expert Alan Guttmacher outlines key issues facing children’s health today, “It’s not just science fiction anymore”: Childx speakers talk stem cell and gene therapy and Global health and precision medicine: Highlights from day two of Stanford’s Childx conference

Cancer, Events, Pediatrics, Stanford News, Videos

Pediatric nephrologist Mary Leonard discusses bone health in children with chronic diseases at Childx

Pediatric nephrologist Mary Leonard discusses bone health in children with chronic diseases at Childx

The inaugural Childx conference was held here last month video interviews featuring keynote speakers, panelists and moderators are now on the Stanford YouTube channel. To continue the discussion of driving innovation in maternal and child health, we’ll be featuring a selection of the videos this month on Scope.

Stanford pediatric nephrologist Mary Leonard, MD, initially began her career as a physician-scientist by investigating the bone complications of pediatric kidney disease. One of her earlier findings was that a number of the risk factors for poor bone development were also associated with many other childhood diseases, such as inflammatory bowel disease and cancer.

In the above video, Leonard explains how advances in treating pediatric kidney failure, cancer and other diseases is creating a growing population of survivors who are entering adulthood facing other health risks, including poor bone health, insulin resistance and cardiovascular disease. Watch the full interview to understand the magnitude of the problem and learn about efforts to develop prevention methods.

Previously: Childx speaker Matthew Gillman discusses obesity prevention, Pediatric health expert Alan Guttmacher outlines key issues facing children’s health today, “It’s not just science fiction anymore”: Childx speakers talk stem cell and gene therapy and Global health and precision medicine: Highlights from day two of Stanford’s Childx conference

Big data, Events, Stanford News, Videos

Stanford bioengineer discusses mining social media and smartphone data for biomedical research

Stanford bioengineer discusses mining social media and smartphone data for biomedical research

During the 2014 Big Data in Biomedicine conference, Stanford bioengineer and geneticist Russ Altman, MD, PhD, spoke about the possibility of collecting data directly from patients, via social media or smartphones, and using it to compliment traditional methods of gathering medical information to give clinicians an unprecedented capability to assess individuals’ state of health.

“One of the most exciting things is the ability to combine data at multiple levels,” he says in the video above. “We have an amazing ability to collect molecular data, cellular data, organism data from electronic medical records and population data about what’s happening at the population and global scale. The beauty of informatics is we don’t have to be tied to one of those levels.”

At the upcoming Big Data in Biomedicine conference, Altman will moderate a discussion with Kathy Hudson, PhD, deputy director for Science, Outreach, and Policy at the National Institutes of Health. Hudson leads the science policy, legislation, and communications efforts of the NIH and serves as a senior advisor to the NIH Director Francis Collins, MD, PhD. She is responsible for creating major new strategic and scientific initiatives and was a key architect of the National Center for Advancing Translational Sciences and the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative.

Registration for the conference, which will be held May 20-22 at Stanford, is currently open. More details about the program can be found on its website.

Previously: Big data used to help identify patients at risk of deadly high-cholesterol disorder, Examining the potential of big data to transform health care and Registration for Big Data in Biomedicine conference now open

Research, Sports, Stanford News

Stanford bioengineers and clinicians team up to shed light on how concussions affect the brain

Stanford bioengineers and clinicians team up to shed light on how concussions affect the brain

9764280602_4d132cd012_zIn an effort to better understand and prevent concussions, bioengineers and clinicians at Stanford have turned athletic fields into laboratories to tackle fundamental questions about brain injuries. A story recently published in Stanford Magazine offers a detailed look at the ongoing research involving high-tech, data-gathering mouth guards worn by players during games to record the impact of hits and advanced imaging studies to measure subtle changes on athletes’ brain scans.

Kristin Sainani writes:

Unfortunately, after years of inattention, the science of concussions remains in its infancy. “We don’t even know what a concussion is at a basic, biological level,” says Mona Hicks, who oversaw traumatic brain injury research at the National Institutes of Health for nine years and is now chief scientific officer at One Mind, a nonprofit focused on brain disease. This scientific void creates uncertainty when it comes to addressing such controversies as how long to hold concussed athletes out of play, whether to ban heading in youth soccer and how much to change the game of football.

David Camarillo, assistant professor of bioengineering and a former football player at Princeton University, is studying the physics of such hits. His lab has outfitted most of Shaw’s team with high-tech, data-gathering mouth guards that the players wear during games. Seattle-based X2Biosystems had developed prototypes for a commercial product; Camarillo’s group customized the design for research use. The devices measure how violently a player’s head gets tossed around during collisions, falls and other impacts.

“My long-term goal is to prevent concussions,” Camarillo says. “The first step is to understand what causes them.”

Previously: Forces at work in concussions more complicated than previously thought, new Stanford study reveals, Stanford undergrad studies cellular effects of concussions, Developing a computer model to better diagnose brain damage, concussions and Stanford researchers working to combat concussions in football
Photo by West Point – The U.S. Military Academy

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