Entries in research (14)


The Emergence of Patient-centered outcomes in guiding clinical care

Last week, I had the opportunity to attend an educational forum offered by MassBio entitled “Communicating Clinical Benefit: The Role of Patient-Centered Measures” that opened my eyes to the importance of patient-reported outcomes (PRO) in improving clinical care. For those who are unfamiliar, PRO research aims to develop assessment tools that can be used to capture a patient’s self-reporting of health and quality of life. The assessment tools are often in the form of a questionnaire or interview asking patients directly about their symptoms, functioning, and overall mental state as they pertain to disease and/or treatment. These data are different from physiological measurements and investigator-reported measurements, which objectively measure and observe the patient’s biology. By creating standardized, reproducible methods to record reports from patients, these tools permit a more objective way to characterize the patients’ experience. Patient-centered tests obviously require thorough validation before FDA approval for use, as potential primary or secondary endpoints in clinical trials.

The compiled outcome data from these studies provides relevant guidance for patients in evaluating their care options and understanding the treatment-associated risks. In addition, these data offer insight for payers as to which clinical benefits are most meaningful to the patient, which in turn can help assign a monetary value to a particular therapy. The information generated in such studies may also be used to support labeling on the drug package insert and marketing indications for a product. Additionally, drug companies can use these data to confirm their product is fulfilling their patients’ needs early on in clinical development.

Growing interest in measuring patient-centered outcomes reflects a shift away from medicine’s former disease-centric approach. As the field of genomics uncovers an array of genetic and epigenetic influencers, diseases have become increasingly complex, easing the path for personalized medicine to emerge as a more direct way to help individuals solve health problems. While the need for research to identify biological mechanisms and markers of disease and therapeutic efficacy remains critical, it is exciting to learn about the research that helps to connect the biology to clinical outcomes important to the patient.



The Importance of the Presentation in Science

Looking back to the start of my scientific career, I always assumed that once I had great data, my talks would be as flashy and convincing as the seminars I attended when top school professors sell their research. I still think that you need reasonably good data to give a convincing talk, but over the years I have seen many talks with amazing data that overall left a stale taste and didn’t excite any listener, even though the findings were quite remarkable if you were familiar with the field. On the contrary, I have seen talks about researchers saving the world with their work, of course only if you didn’t know that the work was comparably less exciting and fundamental. And I have realized for myself that once I had my 5-minute elevator speech down about my research, people were actually interested in my work and didn’t just ask me whether as a chemist I was able to replicate Breaking Bad or tell me how horribly they did in high school science. It was one of the more important lessons I have learned so far in my PhD: A good presentation is just as important as good results. Luckily it’s less time consuming and serendipity based than science to come up with a couple of intriguing slides. Some scientists have published articles about writing good papers[i], giving good presentations[ii] and so forth. Everybody has their own strengths and individual weaknesses, but there are some generally very useful guidelines to rapidly improve one’s performance in a talk.

For one, it is worth the effort to film one’s presentations. A good portion of what you learn with a personal “presentation trainer” becomes fairly obvious after watching yourself for just a couple of minutes. Did you have your hands in your pockets, was 80% of the content “as in, like, you know, that thing!” or were you reading off your own slides? You know when you hear a good talk and you know what’s bad about a talk most of the time, so watch yourself at least once on video.

In Germany we say “no master has fallen out of the sky”, which basically just reminds you to practice. A talk is at least twice as good if you’ve actually given it before, maybe not in front of an audience but definitely out loud. I didn’t take this advice early on in grad school and spent these last two hours before perfecting my slides. I am not saying that you shouldn’t have perfect slides, but practicing for an hour instead would have had a much more convincing outcome that having that one reference italicized. Which brings me to my next point…

Go over your slides with someone else. You have been staring at them for days, weeks, or however long, and they’ll look much better to you than they actually are. Also, choose someone with similar background as your audience. Always keep in mind that the most exciting statistics are just going to bore an organic chemist if the lingo is unintelligible.

There are a lot of things to keep in mind about not having too much text on your slide, how the title should be a summary, the right size of text, consistency throughout the talk, good color schemes (consider that a relatively large part of the population is color blind, so be considerate and use appropriate colors- there’s good literature about it[iii])… Lots of work, but negligible to the work you put into the science!  

Now there is much more to the art of presentation than that, but the point I’m trying to make is that there are a lot of scientists with amazing data, but often it is the presentation skills that set you apart from the masses and open doors for collaborations or jobs. Needless to say, I am over sitting in on boring talks about great science that would really shine with just a little extra time spent to perfect the presentation.

[i] Adv. Materi. 2014, 16, 1375-1378

[ii] Angew. Chem. Int. Ed. 2013, 52, 3780 – 3781

[iii] http://www.somersault1824.com/tips-for-designing-scientific-figures-for-color-blind-readers/


An open mind for improving human health

‘Astounding’ is how I would describe the results presented by Dr. Roland Griffiths (a 40+ year veteran researcher at Johns Hopkins University School of Medicine) at the closing sessions of the 54th annual meeting of the American College of Neuropsycho-pharmacology.  Dr. Griffith and his colleagues shared study results that after a single treatment with the study drug, the severely depressed mood of terminally-ill cancer patients had been dramatically improved (and I would wager this as an understatement). Their perspective on life had been powerfully changed for the better and was evidenced not only on the way the patients felt about themselves but also from the feedback of members of the patients’ individual communities – the patients seemed much happier and more at peace to family, loved-ones, co-workers and community.  Even more incredible was that these positive changes were not only profound in magnitude, but remained very strong, even 6 MONTHS after treatment.  The effects seemed a bit like magic; the test drug was psilocybin – ‘magic mushrooms’.

Dr. Griffiths' landmark paper in 2006 remains a watershed in modern psilocybin research (http://www.ncbi.nlm.nih.gov/pubmed/16826400) and caused a resurgence of interest in the compound as a pharmacological tool that could be safely investigated in humans after a decades-long lag in research. The 2006 report, through a careful scientific approach, provided some of the best-controlled evidence for the positive and lasting effects of psilocybin in healthy volunteers. Highlights can be seen in his 2009 TEDxMidAtlantic talk, currently posted on YouTube.

Where had psilocybin gone? After widespread, and arguably fallible research (poor study design) in the 1950s and 1960s on then-legal psilocybin, concern of substance abuse as a street drug led to classification as a Schedule I drug in the US (high abuse potential with no accepted medical use). Psilocybin is a naturally occurring psychoactive compound produced by more than 200 types of mushrooms. Considered an ‘entheogen,’ it has been used for centuries in religious ceremonies to “generate the divine within” however its illegal status relegated it as an underground psychoactive drug, known also as ‘mushrooms’ or ‘shrooms’. 

Where has the anxiety gone? Whereas subjects in Dr. Griffiths studies emerged from treatment with a deeply positive recalibration of life’s meaning, a lead question during last week’s ACNP session was in the apparent absence of experiences occasioned by the lay user which are highly variable and dominated by feelings of intense panic and fear.  Here a key feature of Dr. Griffiths’ studies - ‘supportive conditions’  - are highly important and being with several visits between test subjects and study staff prior to psilocybin administration to develop trust and rapport. During the 8-hour psilocybin treatment session, study staff were present as ‘guides’ to reassure subjects and navigate darker experiences with greater confidence and a philosophy of discovery. 

Modern neuroscience has a close eye on this ancient drug, and beyond subjective mood testing, research led by Dr. Robin Carhart-Harris (Imperial College London) is using functional magnetic resonance imaging to better understand how brain activation patterns are modulated by psilocybin (http://www.ncbi.nlm.nih.gov/pubmed/22308440). In addition to the growing evidence from studies by Dr. Griffiths and similar trials at New York University (see a great article in the New Yorker from Feb. 2015; http://www.newyorker.com/magazine/2015/02/09/trip-treatment), the in vivo imaging results provide compelling evidence that under controlled conditions, psilocybin is safe and highly effective in improving the well-being patients in need. This is a fascinating example of science to me and I am excited to see how psilocybin’s status as an illegal drug with ‘no accepted medical use’ will change when the benefit to patients seems so clear.



Photo credit: RollingStone.com

Media links:

1.  Griffiths, et al, 2006 Psychopharmacology (Berlin) http://www.ncbi.nlm.nih.gov/pubmed/16826400.

2.  Griffiths, 2009 TED talk: https://www.youtube.com/watch?v=LKm_mnbN9JY.

3.  Carhart-Harris, et al, 2012 PNAS (http://www.ncbi.nlm.nih.gov/pubmed/22308440)

4. Feb. 2015 New Yorker article: (http://www.newyorker.com/magazine/2015/02/09/trip-treatment


Your brain on music-what does the research tell us?

Music, one element of the performing arts, has been part of our lives for centuries. It is now easier than ever to access our music no matter where we are. We listen whilst we cook, drive, clean and even when going for a run. My daily routine includes habitually navigating YouTube in search of music to play before starting work. I wonder if this contributes towards my productivity throughout the day; or maybe it has become something of a distraction, a means to escape the pressure to perform at work by becoming an audience for the musicians and singers I select.

The relationship between music and fundamental function has been heavily explored. N. Perham and J. Vizard preformed a small scale trial (25 participants) to understand the effect of music on remembering items in a specific order. Tests were carried out to explore the difference in results whilst music was prevalent and also when it was absent. Participants achieved the highest scores in the absence of music and lower scores when background music was played, whether it was liked or disliked by the participants.

An investigation by W. Brodsky and Z. Slor also demonstrated that music can be disruptive to young newly qualified drivers when taking to the wheel. The driving of 85 young-novice drivers was tested while playing preferred music, in-car music and no music. Driving while playing preferred music was reported to boost positive mood and enjoyment; however, increased driver miscalculation deficiency and traffic violations were also observed. On the other hand, listening to music that generated moderate levels of perceptual complexity improved the drivers’ performance leading to increased driver safety.
  Both experiments required individuals to be highly attentive and focused. The results indicate that preferred music has some negative effect on an individual’s performance; Nevertheless, this does not mean that music is bad for you. Another study carried out by A. Cabanac et. al. showed that students (560 participant aged 14 – 17) that chose a music course in their curriculum obtain higher grades on average compared to students that did not choose music as an optional course. This is the case in all subjects including Sport, Math, English and Science. The authors conclude that music can help relieve stress and as a result students taking musical courses perform better.
Personally, I think that the effect of music differs from person to person and depends on the task at hand. I would ideally avoid listening to music when high concentration is required; however, I believe music could help when performing repetitive tasks, as it can lead to positivity and enjoyment and, thus, increased productivity.
N. Perham and J. Vizard (2010) “Can preference for background music mediate the irrelevant sound effect?” Appl Cogn Psychol. 25:625-631.
W. Brodsky and Z. Slor (2013) “Background music as a risk factor for distraction among young-novice drivers” Accid Anal Prev. 59:382-93.
A. Cabanac et. al. (2013) “Music and academic performance” Behav Brain Res. 256:257-60.




Objective data using wearable tech...the future

I have worn my fitbit for 2 weeks now and I am obsessed. Each time I have to take it off to charge it, I am torn; I don’t want to lose that data. I cannot wait to see how my sleep patterns change with the seasons and how my heart rate adapts to different types of exercise; not to mention how all of those things change with age! I believe wearable technology is a great unobtrusive way to collect objective data about patients and research subjects for medical care and clinical studies, respectively. If nothing else, measurements about sleep, exercise, and heart rate can help report lifestyle data that may otherwise be skewed when self-reported. One of the first things I noticed when looking at my fitbit data is how much I had previously overestimated the amount of sleep I was getting and the time I was keeping my heart rate up at the gym. It’s definitely easy to round those numbers up or down to make yourself feel better, but the hard data forces you to acknowledge your daily habits and is a more tangible data set for research.

A recent feature in Nature brought my attention to other types of technological updates for collecting lifestyle data. The Centre for Time Use research have a collection of diaries recording people’s daily activities in 30 minutes increments. These studies were initiated in 1961 by the BBC to help design their television programming schedule with a better understanding of their audience’s daily availability. Similar daily diaries have been collected since documenting the evolving lives of the population over the past 50 years. These data are part of an enormous archive that is being used by social scientists to understand the way people are spending their days and how that has changed with time. Epidemiologists are also using these data to see how the changes correlate with chronic disease prevalence. 

The modernization of the daily diary includes an accelerometer to track activity and a wearable camera that snaps photos a few times a minute, all day, capturing images of how you are actually spending your time. Researchers are hoping this data will offer additional details that people may forget to record in the self-reported diary. Helen Pearson, author of the Nature feature, tests out this new technology and reports on her experience in a great article that I highly recommend. Although this data seems like it has the potential for subjective interpretation, it is an interesting approach to acquiring objective data describing a person’s life. I hope all the big data crunchers are ready for what they have coming to them!


Pearson, H. The lab that knows where your time really goes. Nature. 526; 492-496 (2015)