6 posts tagged “study”

The record on research: A chat with Duke’s Dr. Rick Bedlack

Posted March 7th, 2017 by

“This is the fastest enrolling trial in ALS history.”

 

A brightly-colored blazer and the determination to make a difference for ALS patients are two of Dr. Rick Bedlack’s defining characteristics. Dr. Bedlack is a tenured associate professor of Medicine/Neurology at Duke University. He’s also the director of the Duke ALS Clinic that’s partnering with PatientsLikeMe in the current Lunasin study. We recently spoke with him about his background with ALS and the ins and outs of the study.

He saw his first patient with ALS in the late 1990s during his residency at Duke.  He says, “I remember being amazed by the person’s history and neurological exam findings, intrigued by the mysteries of why this was happening, and horrified when I heard my attending physician say ‘you have 2-3 years. There is nothing we can do. Go and get your affairs in order.’” Driving home that day, he decided to build a program for people with ALS that would give them options for living the best possible life with the disease and for participating in research that would stimulate some hope.

Fast forward to March of 2016 when the Lunasin study started. What’s Lunasin and why does it matter to the ALS community? Lunasin is a peptide first extracted from soybeans, which has several potential mechanisms by which it could help a person with ALS. “I first heard about it in a video that my ALSUntangled team was asked to review. In this, a man named Mike McDuff reported that he had ALS, started taking a Lunasin-containing supplement regimen, and unexpectedly experienced dramatic improvements in his speech and swallowing,” says Rick.

He found Mike McDuff and validated his ALS reversal. “One possible explanation for his ALS reversal is that the Lunasin regimen really works,” he says. “Other possible explanations are that Mr. McDuff has an undiagnosed ALS-mimic syndrome, or that his body is somehow naturally ‘resistant’ to this disease. I am testing all these hypotheses in my ALS Reversals program.”

The Lunasin study is a clinical trial of the exact same Lunasin-containing regimen that Mike McDuff took when he experienced his ALS reversal. Because they’re looking for the largest signal ever in an ALS trial, they’ve been able to incorporate some unusual design features into this trial:

  • The inclusion criteria are very broad. There are no cutoffs related to disease duration or breathing function.
  • There are no placebos. All 50 people in the trial will get the real treatments.
  • There are very few in-person visits. Most of the visits are virtual, with participants logging into PatientsLikeMe to enter measurements we teach them to make.
  • The results of the study are available in real time. Anyone can go onto PatientsLikeMe and type in “Lunasin Duke Virtual Trial” and see what participants are saying is happening to them.

“I appreciate the frustration many people with ALS have expressed about the way most of our trials are designed and I wanted to do something different to help them,” says Dr. Bedlack. “It took longer than I expected to get the study open. Constipation is much more common on the Lunasin regimen than I expected, and drop outs have been higher than I hoped thus far.”

The IRB-approved protocol is published so that anyone who wants to try the Lunasin regimen outside the trial can do so using the exact same products and doses, and even record their same outcome measures on PatientsLikeMe.

So, what’s the end game of this study? Dr. Bedlack comments, “I hope to find a way to reverse ALS or at least slow it down. If that does not happen, then I hope I can at least show that this unusual design enrolls more quickly and retains study participants better than a more traditional ALS trial. This is the fastest enrolling trial in ALS history.”

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Study results: What patients like you said about sleep medications

Posted March 28th, 2016 by

Over a year ago, we partnered up with Merck Pharmaceuticals to learn more about insomnia and sleep medications. More than 1,200 PatientsLikeMe members responded to questions about how long they’ve had sleep problems, what treatments they’re using, what interferes with sleep most and what their overall quality of sleep is like.

Here’s what we uncovered (tap each graphic for a larger view).

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PatientsLikeMe usability study for mobile app

Posted June 3rd, 2015 by

Designing a new app is like designing a car. Your engineer and designer may have done a flawless job, but nothing matters until the person actually steps into the driver’s seat and test-drives the product. So when it came time for us to launch the first pass at our new mobile app, called PLM Connect, that’s exactly what we did. We invited a handful of members to come into the PatientsLikeMe office and test-drive the app.

Alex (ITalkToTheWind), a PatientsLikeMe community member, took some extra time to share about her experiences testing the app in an interview. One of the features, InstantMe, is a tool on the PatientsLikeMe website that’s a simple way to track and share how you’re doing. In this first pass of the app, and since she’s become a member in 2010, Alex has posted more than 1500 (!) InstantMe updates. Not only was she extremely helpful with her feedback, but she also brought in the artwork she’s created based on her InstantMe entries. Everyone on the PatientsLikeMe team was honored that Alex brought her art into the office for us to see, and we wanted to share it with the rest of the community, too. Here’s her story: 

Alex, what was it like doing the usability testing?
It was great to come down to the PatientsLikeMe headquarters to give my personal feedback on the first pass at PLM Connect.

I update my InstantMe every day, so I’ve been very eager for an application I can use while I am away from my computer so my charts can be as accurate as possible. Throughout testing the beta application, I got to dictate my thought process step by step with the engineering team. I communicated different ways to improve the application’s features, programming and design to make it more user-friendly and to have it operate more smoothly.

I also got to tell the team what my personal wants would be for a PatientsLikeMe application. Since I am an artist, it was great to be able to talk about how the design could compliment the different functions of the application. I mentioned how important it is to carry over the community and support aspect that is available at the main site over to the application. Overall, I was trying to think about the entire diversity of conditions in the community and how they could benefit using the application, and how to make it as easy to use as possible.

How do you use InstantMe on PatientsLikeMe?
I’ve been using InstantMe religiously to keep track of my moods for five years now, since I was diagnosed with my primary condition and began my treatment process. I basically see everything I’ve accumulated through my InstantMe as a diary of my life for the past 5 years.

It is very important to me to see how my treatments, symptoms, and life events all correlate with my moods to understand what is or isn’t currently working, and what has or hasn’t worked in the past, especially in a visual way. I’ve been able to keep track of absolutely everything I’ve tried to treat my conditions including medication, herbal supplements, vitamins, diets, different therapists and therapeutic approaches, exercise, lifestyle changes and meditation.

InstantMe also gives you the option to explain “Why” you are experiencing your mood, which has been crucial to me in reflecting on the moments that seemed so difficult at the time.

Another interesting part of that feature is reviewing how my automatic thoughts have changed through my treatment process, which is also really important for someone who suffers from mood swings like I do. I react differently now to difficult life events and can see how I’ve been better able cope with stress over the five years. It’s really nice to see how far I’ve come. 

Will you share a little about your InstantMe artwork?
My work is about the disconnect that occurs with one’s experiences and personal identity over time. I make paintings and drawings using my InstantMe mood logs, and other PatientsLikeMe charts by transferring them onto a physical material such as a wooden panel or paper.

I correlate the text from the InstantMe’s to remember why I was feeling good, bad or neutral and paint symbols to represent that time period, or draw the mood logs themselves into a painted environment or memory in which I was experiencing that mood.

It’s a lot about trying to gain a connection with these fleeting moments and my shifting identity over time and learning to accept them … the process of creating the artwork itself helps me cope with my condition.

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Update and results – the Patient Voice Analysis study

Posted April 27th, 2015 by

About a year ago, the PatientsLikeMe Parkinson’s disease (PD) community started something totally different: a study to compare the sound of their voices to their self-reported PD Rating Scale (PDRS) on PatientsLikeMe. It’s called the Patient Voice Analysis (PVA), and we teamed up with you, Max Little, Ph.D. and Sage Bionetworks to get it done.

A little bit of background
Dr. Little had done some earlier work and compared the voice signals of people who were living with PD to those of people who were not, but we wanted to take that to the next level. With their PDRS, they shared how Parkinson’s was affecting them, and we were able to match their self-reported scores to the sound of their voice.

By matching a PDRS to voice samples, we might develop the ability to predict PDRS scores (which takes a few minutes to complete) by using the voice test (which only takes a few seconds). We might also be able to detect more subtle changes in people’s Parkinson’s through their voice than we can through the PDRS. This is what Dr. Little is working toward, and all the voice samples you donated will help make it happen.

Community results, starting with the basics
Who took part, from where, and how many PDRS scores could we match to voice recordings?

  • Most of the recordings came from the U.S. (81%), with others coming from the U.K. (12%), Canada (5%) and some from Australia and New Zealand, too.
  • 677 of you recorded 851 voice samples
    • Since our original goal was 500 samples, you blew that out of the water!
  • 114 of you took the test two or more times, and one community member even contributed 10 recordings!
    • For those that took part more than once, we can start to examine how your symptoms changed over time.

Why voice recording quality matters
For the PVA study, you were able to use your landlines, cell phones, even Skype or Voice-Over-Internet-Protocol (VoIP) to submit your voice samples. The recording quality varies depending on which type of call you used, occasionally creating technical issues in analyzing the voice samples.

For example, if you’re using a cell phone in a busy restaurant, your microphone will automatically get louder so that the person you’re talking to can hear your voice better and without distortion. But that also changes the loudness of the background noise. In this study, that automatic change could actually affect the quality of the voice recording, so we have to identify where this has been an issue and find ways to overcome it.

Partly because of this, we’re still analyzing the voice samples in detail. We’re looking for subtle markers of Parkinson’s, such as fluctuations in volume, pitch and breathiness. We’re also training intelligent algorithms to identify when the quality of the voice recording is strong enough so we can develop a consistent and repeatable process.

You be the researcher
To give you some idea of what we are looking for in these voice recordings, we wanted to share a couple with you. The first is someone living with severe Parkinson’s, who scores 55 on the PDRS. You can probably hear the noticeable tremor in pitch, and the occasional short breaks in voicing.

The second is a recording of someone with mild Parkinson’s, who has a very low PDRS score of 1.

Can you hear the subtle drift in pitch? This is, most likely, indistinguishable from normal pitch drift. Subtle pitch variations such as this are one of the kinds of symptoms that algorithms attempt to identify from these voice recordings, and they contribute towards making the PDRS prediction.

So, what’s next?
At this stage, the PVA project is still just a research tool and isn’t quite ready for clinical or diagnostic use. We’re still working on analyzing the data to compare the severity of voice patterns to the reported severity of Parkinson’s disease. But in the meantime, if you’re looking to share more info with your doctor, the most useful tool is your PDRS score on your PatientsLikeMe profile. It contains items that make sense to a neurologist. If your clinic has access to speech and language pathologists, they would also have the ability to map any vocal issues you may be experiencing as part of your Parkinson’s.

As we continue to evolve the tools, we hope to provide individual level feedback and information for clinicians. But before that can happen, we want to make sure that the data quality is high enough to support drawing clinical conclusions.

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PatientsLikeMe study monitors walking activity in people with MS

Posted April 15th, 2015 by

          

Cambridge, MA, April 15, 2015—PatientsLikeMe today announced results of a novel study conducted with Biogen that showed how people living with multiple sclerosis (MS) can use wearable activity tracking devices to collect and share their mobility data, which could potentially provide relevant information to their clinicians and to other MS patients. These data are being presented at the 67th American Academy of Neurology’s (AAN) Annual Meeting in Washington, DC April 18-25.

“MS impairs the ability to walk for many people with MS, yet we only assess walking ability in the limited time a patient is in the doctor’s office,” said Richard Rudick, MD, vice president, Value Based Medicine, Biogen. “Consumer devices can measure number of steps, distance walked, and sleep quality on a continuous basis in a person’s home environment. These data could provide potentially important information to supplement office visit exams.”

The study was designed to assess the feasibility of using a consumer wearable device to monitor activity among people with MS in a real-world setting. In it, 248 PatientsLikeMe members were provided with Fitbit One™ activity trackers. Of those who received them, 213 (82%) activated the device with the Fitbit website and authorized PatientsLikeMe to access their data. Two-hundred and three of those who authorized sharing of the data synchronized the device with the service and produced tracking data. Participants synced an average 18.21 days of data over the 21-day study (87% adherence).

Paul Wicks, PhD, Vice President of Innovation at PatientsLikeMe, said that advances in wearable health technology have the potential to shed light on disease characteristics. “PatientsLikeMe is in a unique position to combine self-reported data with objective measurement and help patients and researchers learn more to impact self discovery and research.”

The three-week study had a lasting impact on its participants, who together took a total of 15 million steps and walked 6,820 miles, the distance from Boston to Beijing. “I got positive reinforcement to do more each day, and that really encouraged me,” said Annette Smiling, a PatientsLikeMe member and study participant who had never used a wearable activity tracker before. “The Fitbit also allowed me to track what I was eating and how I was sleeping. I made more positive choices as a result.”

After the study period, participants were surveyed to learn more about their study experiences and about their attitudes toward technology and physical activity tracking. Of the 191 participants who responded to the post-study survey, 88 percent reported the device was easy to use and incorporate into their daily routine; 83 percent agreed that they would continue to use the device after the study; and 68 percent believed that the device would be useful to them in managing their MS. Additional survey data is available at http://news.patientslikeme.com.

With more than 38,000 members, PatientsLikeMe’s MS community is the largest and most active MS research community online.

Study Design Methodology
A total of 248 PatientsLikeMe members living with MS were recruited to participate in a study deploying Fitbit One™ activity trackers. Information on patient demographics and level of self-reported functional disability were captured from the participants’ PatientsLikeMe profiles. Devices were mailed to participants with instructions on activation and authorization of data sharing between the manufacturer and PatientsLikeMe. As part of PatientsLikeMe’s member engagement framework, a live concierge service was available to participants to provide answers to technical and other questions. The study also took full advantage of the PatientsLikeMe platform and health tracking tools to engage participants with their data, and with each other. Study participants were able to track their physical activity levels on the PatientsLikeMe website and connect with each other in the MS discussion forum to talk about changing symptoms, benefits and issues. Data were collected for a period of three weeks, and patients were asked to complete a survey to provide feedback on their experiences with the device.

About PatientsLikeMe
PatientsLikeMe® (www.patientslikeme.com) is a patient network that improves lives and a real-time research platform that advances medicine. Through the network, patients connect with others who have the same disease or condition and track and share their own experiences. In the process, they generate data about the real-world nature of disease that help researchers, pharmaceutical companies, regulators, providers, and nonprofits develop more effective products, services and care. With more than 325,000 members, PatientsLikeMe is a trusted source for real-world disease information and a clinically robust resource that has published more than 60 peer-reviewed research studies. Visit us at www.patientslikeme.com or follow us via our blog, Twitter or Facebook.

Contact
Margot Carlson Delogne
(781) 492-1039
mcdelogne@patientslikeme.com


More Than Meets the Eye: Non-motor Symptoms in Young-onset Parkinson’s Disease

Posted August 20th, 2008 by

Most people think of Parkinson’s disease (PD) as a condition of aging, and most of the time they’re right. Looking at the population as a whole, about 1-2% of people over the age of 65 have PD, and it’s usually a condition that is most severe in patients in their 70’s and 80’s. However, about 10% of patients buck this trend and experience their first symptoms before the age of 40; these patients are known as Young-Onset PD or YOPD for short. The R&D team here at PatientsLikeMe recently carried out a research study examining an aspect of PD that has only recently gained much attention, “non-motor symptoms.” When a clinician diagnoses PD, they are usually looking for a triad of cardinal features, specifically tremor, slowness, and stiffness. However, in the past 5-10 years it has been increasingly clear that patients with PD also experience a number of non-motor symptoms, ranging from dizziness to constipation, from a loss of sense of smell to hallucinations, and from apathy to urinary problems.

Several of our members had mentioned in forum postings that they were finding non-motor symptoms, such as cognitive confusion and fatigue, to be a particular burden, so we decided to carry out a research study using the well-validated Non-Motor Symptoms Questionnaire (NMS-Quest) developed by Professor Ray Chaudhuri and colleagues. We sent the survey out to all of our PD patients earlier this year. In total, we got responses from 307 patients, including 260 “classic” PD patients and 46 with YOPD. The results show a higher number of non motor symptoms among YOPD patients (14/30) than PD patients (11/30). That’s an average of three more non-motor symptoms for YOPD patients than their older counterparts. More specifically, YOPD patients (at the individual level) report apathy, concentration problems, falls, pains, and sadness more often than older onset PD patients.

You can listen to a short presentation, or read our full report for a more detailed analysis of our methodology and findings, but there were several points I found particularly interesting.

YOPD NMS Study

  • It’s unusual enough to have YOPD, which can be an isolating and confusing experience. For YOPD patients to experience even more non-motor symptoms than patients with more typical PD seems alarming, and requires urgent attention from clinicians, researchers, and patient advocacy groups to ensure their needs are being met.
  • This sentiment is echoed in some of the patient interviews we conducted with members earlier this year. Click here to view the first in a series of interviews to be posted on our YouTube page, and hear for yourself what YOPD patients had to say when we asked what people should know about PD. (More videos to come on our YouTube and Facebook pages, so stay tuned!)
  • YOPD patients are more likely to still be working, to be supporting families, and to be looking after young children or parents. Therefore, even minor changes in cognitive function or mood could have a substantial impact on their ability to function.
  • Because YOPD patients are relatively rare and spread out, researchers aren’t often able to gather sufficiently large samples to study them in detail. A research platform like PatientsLikeMe provides an ideal opportunity to collect high quality data in an efficient way.

PatientsLikeMe member pwicks