26 posts tagged “clinical trials”

The record on research: Catching up with TOA member Cris

Posted March 15th, 2017 by

Back in January we introduced Cris, a member of the 2016-2017 PatientsLikeMe Team of Advisors living with ALS. She’s also a fierce patient advocate who’s participated in several clinical trials since her diagnosis. We recently caught up with her to learn about her experience with clinical trials and why access and awareness of clinical trials is so important.

Despite her shy personality, Cris got involved with patient advocacy with the encouragement of her ALS specialist, Dr. Richard Bedlack. Determined to overcome her shyness, Cris found motivation and purpose in advocating for future patients, family and friends and the belief that access to all trials should be easily available for a patient and not complicated to locate. In May, she attended the ALS Advocacy Day in Washington D.C., where she represented pALS from North Carolina and shared her own ALS journey with her state’s senators.

Cris believes that clinical trials are critical for survival, present and future, but physicians don’t know about, or take the time to discuss current trials with patients unless their facility is active in research. There are very few active ALS clinical trials and because of stringent trial protocols, many patients are not accepted due to progression of the disease. She believes that since ALS is fatal, drug companies are worried patients might succumb before end of trial.

Since her diagnosis in 2014, Cris has participated in several clinical trials:

  • Started a new trial but ultimately failed in the qualification process.
  • Started a “blind” (placebo vs drug dosage) – but couldn’t tolerate the side effects of the drug dosage and was advised not to continue.
  • Participated in a monitored daily physical activity study.
  • Currently enrolled in the year-long Lunasin Virtual Trial (completing at the end of March 2017). This trial is in partnership with Duke and PatientsLikeMe and all monthly input and results can be viewed by PatientsLikeMe patients.
  • Currently participating in a 6-month study through the Precision Medicine Program (ALSTDI), to compare movement data collected using accelerometers and overlay those data with an individual’s self-reported ALSFRS-R scores. Involves keeping track of progression with activity monitors for six days.

What is the goal of the Duke Lunasin Virtual Trial?

  • The primary hypothesis is that a supplement regimen containing Lunasin can decrease the rate of ALSFRS-S progression by 50% relative to matched historic controls.
  • While it might not be the most delicious drink, Cris makes it work by mixing the Lunasin with juices or smoothies. Cris, like many of her fellow pALS, is willing to go to any length for a treatment or cure.
  • Want to know more about the Duke trial? Check out what Dr. Bedlack had to say.

New to the ALS community? Cris has some words of advice:

Know that it’s okay and expected, when first diagnosed with this disease, to have the wide spectrum of emotions from sadness, madness, confusion, “why me” and fear (to name a few). I highly recommend putting aside all the negativity that goes with this journey as quickly as possible. Regroup, focus and be creative on establishing a new way of life to cope with various stages of disability. Contact your local ALS chapter, national or worldwide or other pALS – they’re great resources.”

On PatientsLikeMe:

Cris shared about Lunasin and being a part of the Duke trial. With 151 PatientsLikeMe members reporting that they’ve used Lunasin to treat their ALS, 140 treatment evaluations and almost 45,000 forum posts, Lunasin is a popular topic in the ALS community.

Want to learn more? Head to the forum to check what people are saying about Lunasin.

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The power of your data: Improving clinical trials

Posted December 6th, 2016 by

For these 24 Days of Giving, we’re rethinking what it means to give and how the power of patient data can change healthcare for the better. Another example of the impact your data can have? Making the clinical trial experience better for patients like you.

Now let’s be real, that’s no short order. So where do we even start? With you, and every new member who joins the community. Every day, members are sharing their thoughts on trials, what would make them most likely to take part, and what would turn them off. It’s an ongoing research experience, but let’s take a look at snippet of results so far:

4,718 members have taken part so far…

How do you learn about clinical trials?

  • 59% learned about a trial from your health team, 24% via the web.
  • 24% of you said you first thought of participating in a trial without suggestion from anyone else. For those who participated in past trials, the first person to suggest that you participate in a trial was a doctor (43%) or another healthcare provider (19%).
  • Those of you who took part in clinical trials decided to because of your own desire (80%).

Key takeaway: Most people are still finding out about trials through their care teams or providers, but when it comes to actually deciding to take part, it’s your own desire that motivates you.  

What are your impressions of clinical trials?

Side note: These results are just from those that have taken part in a trial.

  • 55% of you were very or extremely satisfied, and 51% would tell other patients about the trial. Not bad, but not awesome.
  • 9% considered dropping out — side effects and worsening of overall health after trial were the main reasons.
  • You said the best parts of the trial were the opportunity to contribute to science and medicine (78%), and learning more about your condition (46%).
  • You said that the worst parts of the trial were the chance of being given an inactive drug or placebo, experiencing negative effects and discomfort with study procedures.
  • Only 38% of you recall being told about the results. Yikes!!! As you know at PatientsLikeMe, we have a give data, get data philosophy. And you can better believe we work with our partners to get on board with that philosophy, too.

Bottom line: Trial results need to get back to those that take part. Period.

Thanks to all who’ve participated and put the patient perspective into improving clinical trials!

 

 

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PatientsLikeMe Launches Virtual Trial for ALS Patients

Posted October 25th, 2016 by

Study With Major Academic Medical Center to Evaluate the Potential of the Soy Peptide Lunasin to Reverse ALS Symptoms


DURHAM, N.C., October 25, 2016—PatientsLikeMe and The Duke ALS Clinic have just completed enrollment in a virtual trial designed to test whether Lunasin, a peptide found in soy and some cereal grains, is helpful or harmful to patients living with amyotrophic lateral sclerosis (ALS). The Lunasin Virtual Trial is the first study of the supplement in ALS patients and follows a review analyzing its potential to reverse ALS in ALSUntangled, a website for clinicians, patients and researchers to explore alternative treatments.

Duke ALS Clinic Director and ALSUntangled Founder Richard Bedlack, MD, said he first heard about Lunasin’s potential from Mike McDuff, an ALS patient who took the supplement and experienced dramatic improvements in speech, swallowing and limb strength.

“I reviewed Mike’s records and reports, and both his diagnosis and his improvements appeared real,” Bedlack said. “Of course, Mike might have an ALS mimic we don’t know how to test for, or his body may have found a way to beat ALS independent of treatment. But there is one more possibility: his Lunasin regimen might have actually worked. I was compelled to design a study to treat other ALS patients with the same regimen to test if anyone else improves.”

As part of the trial, 50 participants will make three in-person visits to the Duke ALS clinic to measure the supplement’s impact over the course of the year-long study. They will also complete virtual check-ins as members of PatientsLikeMe every 30 days to update their weight, complete an evaluation of their Lunasin regimen, and complete the ALS Functional Rating Scale (Revised, ALSFRS-R), a widely-used patient-reported outcome developed by PatientsLikeMe that allows patients to see their disease progression visually and in context.

Bedlack said the study’s design is patient-centric and eliminates many of the frustrations patients say are inherent in traditional clinical trials. The design also likely contributed to what Bedlack calls the fastest enrollment of any trial in ALS history: the virtual trial took just over five months to fill.

“There are no placebos, and we made the inclusion criteria very broad so that even ALS patients who can’t qualify for other studies due to their long disease duration or use of a ventilator can qualify,” Bedlack said. “We’re also limiting the burden on the participants by helping them check in online from the comfort of home. And we’ve already published the protocol on our ALS Reversals website. We’re taking a completely open approach so that anyone, anywhere, can see the details of what we’re testing and learn more about whether this is helpful, harmful, or does nothing at all. I think all of these factors made the trial very attractive to ALS patients.”

PatientsLikeMe began as a website for people living with ALS to openly share their experiences with each other and study alternative and off-label treatments. Vice President of Innovation Paul Wicks, PhD, said the company’s research focus gave ALS patients a science-based platform to monitor themselves, get ideas from other patients, and play a more active role in their own healthcare. That focus has also helped the company produce more than 30 published research studies on the condition, including a 2011 observational study in Nature Biotechnology that refuted the results of a 2008 clinical trial on the efficacy of lithium carbonate on ALS. More recently the company has worked with Bedlack to study the frequency of ALS plateaus and reversals. Findings from that study were recently published online and in the March 2016 print edition of the journal Neurology.

Anyone taking Lunasin outside of the Lunasin Virtual Trial is invited to separately track their experiences on PatientsLikeMe at www.patientslikeme.com.

ALS is a degenerative disorder affecting upper motor neurons in the brain and lower motor neurons in the brain stem and spinal cord. Symptoms include progressive weakness, atrophy, fasciculation, dysphagia, and eventual paralysis of respiratory function.

Study Design Methodology

The Lunasin Virtual Trial is a 12-month, widely inclusive, largely virtual, single-center, open-label pilot trial utilizing a historical control group. Participants will be asked to sign in or register for an account on www.patientslikeme.com with the help of the study coordinator and will be given a welcome packet that includes a one sheet description of the study, a checkup checklist, and the first month of a total 12-month supply of Lunasin. User IDs associated with participants’ PatientsLikeMe account will be recorded by the study coordinator and shared with study staff at PatientsLikeMe. After the initial in-clinic visit, participants will be asked to enter the following data online: weight, Lunasin study drug regimen evaluation, and their ALSFRS-R score.

About PatientsLikeMe

PatientsLikeMe 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 400,000 members, PatientsLikeMe is a trusted source for real-world disease information and a clinically robust resource that has published more than 80 research studies. Visit us at www.patientslikeme.com or follow us via our blog, Twitter or Facebook.

Contact                                                                                                Margot Carlson Delogne
PatientsLikeMe
mcdelogne@patientslikeme.com
+1.781.492.1039


Bringing the patient voice to clinical trials

Posted October 3rd, 2016 by

PatientsLikeMe has a long tradition of bringing the patient voice into drug discovery and development, but it’s only been in the last year or two that industry has been tuning in to hear what that voice is saying about clinical trials. For the last 18 months, we’ve been asking many of our members about their attitudes toward and experiences with clinical trials, and working to ensure pharmaceutical companies understand what patients want most from trials, now and in the future. We talked with Jeremy Gilbert, Vice President of PLM Health, and Paul Wicks, Vice President of Innovation, about what we’ve found, and how that’s already helped to change the design and execution of trials and their future direction. Jeremy will be presenting more about this topic and details of a key project with AstraZeneca and Takeda that leverages the patient voice in new ways at the Patient-Centered Clinical Trials Conference in Philadelphia on October 11. More info about discounted tickets below.

 

Jeremy Gilbert, Vice President, PLM Health

You’ve surveyed more than 1,400 people about their experiences and opinions with clinical trials. What have they shared, and what has PatientsLikeMe done with that information?

Jeremy: A lot of people in our community are living with life-altering, degenerative conditions, and they’re acutely aware of the importance of new drugs coming to market to address their problems. In conditions like MS, where there are perhaps a dozen treatments on the market, people are keenly aware that the drug they’re taking may stop working, so there is great interest in helping new drugs come to market. Our members are vocal, too, about how involved they want to be. About 92 percent of our survey participants said they would be willing to help researchers design clinical trials to make them better for patients. At the same time, our industry partners are looking for ways to improve trial participation, especially since trials will grow more complex. So we’ve found these two groups want to come together to improve the trial experience for everyone. And there’s no better place to start than by involving patients in the trial design process.

Paul: The state of art in designing trials has been to use the advice of physician experts and to review literature and published papers, but patients have been left out. Researchers have told us this is because of legal and regulatory concerns about asking patients about experimental treatments; they don’t want to be seen as promoting a treatment that hasn’t yet undergone testing. They also cite their lack of experience and fears about the extra time it might take them as reasons why they’ve carried on with the status quo. We’ve long believed that online communities like PatientsLikeMe provide a great vehicle to attack those barriers. We can act as an honest broker of information between researchers and patients who want to help, we’ve developed a repeatable methodology to gain patient input, and we’re delivering results in a matter of weeks to researchers to improve their protocols. Increasingly now researchers are coming to us before they design their trial to incorporate the patient voice, which is a great step.

Does what patients want from trials depend on the condition they have?

Paul Wicks, PhD., Vice President, Innovation

Paul: Some elements, such as their desire to altruistically help other patients like them, stay remarkably consistent. But depending on their demographics and the impact of disease, we do see a lot of variation. Working age adults might want extended hours so they can participate outside of normal business hours, whereas older people might prioritize having transport organized for them. It really depends on the aim of the study as much as the condition; some trials are for drugs that slow the progression of a disease, while others are to treat specific symptoms or even to take an existing drug and make it more convenient to take, such as moving from an injection to a pill. Against that you’ve also got the overall commitment required to the trial protocol such as the number of study visits, their duration, any special testing that people have to undergo and, of course, any special measures put in place to mitigate those burdens. We think patients are interested in participating in research in general because of altruism, that they choose to enroll in a particular trial because of its objectives, and that they stay enrolled because of their relationship with trial staff and the level of burden the study incurs on their daily lives.

The design of a trial is just the start. What happens as trials begin, or end?

Jeremy: We’re starting to see another gap now, which is that companies have no way of soliciting feedback from patients as they participate in a trial, to find out what patients think of real trials. This is a surprise, because given most of us are consumers, we’re used to being able to give feedback about a product or our experience at any time. That inspires us to think about how to gather patient input not just about a trial’s design, but also during the trial itself, and even after it ends. And if we can figure out how to share the knowledge that’s gathered, that would help industry design and run even better trials in the future.

Where do you think trials are headed in the future, and how is the idea of patients as partners instrumental in guiding that direction?

Paul: Today, taking part in a clinical trial is like having a part-time job. In the future, we want it to be much more convenient and to harness the digital technologies that have become a normal part of daily life. We’re also looking at novel technologies to see if we can do away with some of the aspects of trials that patients dislike, such as placebo arms. Using online digital platforms, it might be possible to produce enough carefully-matched historical controls to more rapidly identify when a treatment is having an effect. That could reduce costs, speed up trials and ultimately increase the number of “shots on goal” that researchers can take to fight disease.

 
Note: Jeremy will be presenting more about this topic at the Patient-Centered Clinical Trials Conference in Philadelphia on Tuesday, October 11 at 2:00 PM. He’ll be joined by Arnold Degboe, Director of Patient Reported Outcomes, Health Economics and Outcomes Research at AstraZeneca. They will talk for the first time about their work to change clinical trials with patient feedback. Discounted tickets for patients and industry participants are available from the conference organizer. Please contact Katie Gadsby for further information at kgadsby@eyeforpharma.com.

 

 

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14 questions to ask before you enroll in a clinical trial

Posted July 18th, 2016 by

Have you ever participated in a clinical trial? How much did you know going into it? Our partners over at the Center for Information and Study on Clinical Research Participation (CISCRP) came up with a whole list of questions that will help you decide if a clinical trial is right for you before you commit.

Here are a few of them below, but you can check out the full list and a printable version here.

 

Got any questions you’d add to this list? Head over to the forum and share them with the community!

 

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Patients as Partners: John and David share their clinical trial experiences

Posted June 1st, 2016 by

We’ve been hearing from members of the Team of Advisors about how they’ve used the Partnership Principles in their health journeys. Recently, we sat down with John (Dockstoy), living with ALS, and David (Davidgewirtz), who’s living with lung cancer. John and David are both interested in research and have been involved in a few clinical trials. Below, they share their firsthand trial experiences, offer up some advice for others, and talk about the importance of collaboration and mutual respect with their care teams along the way.

Can you tell us a little about the clinical trials you’ve participated in?

John: I took part in trials at:

  1. Massachusetts General Hospital (September 2014) – phase 2, randomized, double-blind placebo-controlled Multicenter Study of Autologous MSC-NTF Cells in Patients With ALS
  2. Bronx VA – Brain and nerve stimulation for hand muscles in spinal cord injury and ALS
  3. Weill Medical College of Cornell University – Safety of Capryclic Triglycerides in ALS: A Pilot Study

Team of Advisors member David, living with lung cancer

David: I was diagnosed with stage four metastatic lung cancer just about five years ago. June 2011. Since that time my only form of treatment has been targeted therapies which were available to me through clinical trials. The first clinical trial I received the drug Erlotinib known as Tarceva for three and a half years. The second clinical trial I am now on for about fifteen months targets a mutation that is responsible for resistance to Tarceva. The Clovis pharmaceutical company makes the drug with the code name C01686 which does not have FDA approval and Clovis recently disclosed that the drug has been withdrawn from future development. Both drugs have enabled me to live a very high quality of life and unless told nobody would know I had a terminal disease.

Clinical trials require the patient to be an active participant in the treatment modality. For me both clinical trials required that I have monthly visits to the clinic for blood work and scans every two months. The routine of my CT scans include my chest, abdomen, and pelvis plus a whole body bone scan. Normally I spend six hours at the hospital to compete these tests. That I am monitored very closely is a double edged sword. The downside is the frequency of scans exposes me to significant radiation which overtime is also associated with cancer. There is also generalized anxiety that comes with this routine. When you’re done with one cycle which includes a discussion with the oncologist about the radiology report, you automatically start to think about the next cycle. The silver lining in this cloud is that detection of new disease is caught very early and more often treatable than not – a really good benefit, which takes some of the sting out of the routine.

Have any of the partnership principles helped you get involved in these clinical trials?

John: I believe all principles apply. Respect is key; and alignment and teamwork from both parties involved.

David: “Know your needs” was the principal driver of why I entered a clinical trial. This principle requires that the expectations of the patient (me) are aligned in a partnership with the goals of the oncologist. In my situation there was a shared expectation of the value of clinical trial. In collaboration with my oncologist we discussed the likely benefits, side effects and the efficacy of the trial as a treatment plan for my advanced stage lung cancer. We shared the belief that the benefits of the trial far exceed the risks associated with taking Tarceva. We shared the value that (1) I would receive state-of-the-art treatment with the expectations that the drug Tarceva would (2) not not only control my cancer, but would extend my progression free survival of my disease.

How do you decide if a clinical trial is right for you?

Team of Advisors member John, living with ALS

John: Timing is a critical decision factor. What I mean by that is many clinical trials have two-year and three-year exclusion criteria. Also, location — most trials do not reimburse for travel or nominal reimbursement. You must believe that the trial will have a benefit, not only to you but for those to follow.  

David: In collaboration with caregivers the patient must know his/her needs in terms of the benefits and risks of entering a clinical trial. This requires that the care team fully explain the benefits of the trial versus the risk of participating. A phase one trial poses the most risk to the patient with an uncertain outcome. In contrast, a phase three trial poses the least. For example, my first clinical trial was a phase three trial where I was given the drug Tarceva with expectation that my medium progression free survival would be 12 months. In my case I did not progress until 3.5 years. Truly a great outcome despite some manageable side effects or risks.

A patient also has the opportunity to enhance the understanding of their disease. All types of clinical data captured in the study helps organizations like PatientsLikeMe market data to pharmaceutical and insurance companies who are stakeholders needed to fast-track new drugs from the bench to the clinic. In the end, your participation along with others may save or prolong the quality of your life. This possibility was the primary driver for me to enroll in another clinical trial that has kept my cancer in check, now five years, with minimal side effects.

What advice do you have for other patients who want to learn more about participating in clinical trials?

John: Research, research, research! Scour the Internet for details, speak with friends and family, ask your doctor what they think and what they see on the horizon for new trials. Be prepared to look at what’s next in clinical trials so as one completes you can start up another.

David: The decision to participate in clinical trials encompasses many of our PatientsLikeMe partnership principles. For example, where you are in your treatments phase may dictate what principles are more important than others. If you are at the start of your treatment, then you must have a very good understanding of what a clinical trial can do for you — how much more quality time the treatment offers. You can only arrive at this decision point if you and your care team have a shared understanding of the benefits of participating in a clinical trial versus pursuing a different line of treatment. Through my experience with clinical trials you are well served if you operate under the assumption that participation is a shared responsibility. Alternatively, if you reach a milestone in your treatment where you must go to another line of treatment then it is critical to reflect, evaluate and reprioritize if your current care team is the right team to meet your needs. Finally, regardless of the phase you enter a clinical trial, you must self-educate yourself

That means you have done some basic research by:

  1. Reading about relevant clinical trials on sites like the National Cancer Institute, which has a listing and description of all clinical trials in the U.S. and worldwide.
  1. Preparing yourself for entering a clinical trial by visiting the government website that explains what patients and caregivers should know about participating in clinical trials.
  1. Participating in organizations like PatientsLikeMe that have communities where patients share their treatment experiences, side effects and their success and failures. Personally, these organizations have empowered me through emotional support and knowledge of other clinical trials that may become options for me while I travel on my journey.

 

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A patient’s perspective: member Gary reports back from the FDA public meeting on Parkinson’s disease

Posted October 27th, 2015 by

Back in September, Sally Okun, Vice President of Advocacy, Policy and Patient Safety, and community moderator Molly Cotter, attended the FDA public meeting on Parkinson’s disease (PD) along with PatientsLikeMe member Gary (tupelo).

We posted some interesting developments from the meeting in the forum, but we also caught up with Gary for his perspective on the event. Here’s what he had to say:

Why did you want to be part of the FDA public meeting?

On the day I was diagnosed, back in 2012, I spent a great deal of time reflecting on my life. I thought a lot about how Parkinson’s disease will impact me and my family in the future. On that day I made a commitment to myself that I was going to take control, to the best of my ability, on the course of the disease progression, and would do anything possible to find a cure. I was determined to educate myself as much as possible about the disease, put together the best possible health care team, learn all I could about treatments currently in research, and participate in clinical trials. Parkinson’s was a new challenge in my life and I intended to tackle it like I would any other challenge that I had faced in the past. Along the way in this new journey, I became more publicly involved as a PD advocate, both for advancing research as well as supporting newly diagnosed patients. So, when I noticed the FDA announcement regarding the meeting, I decided to send in my comments and thoughts in hope that they might be helpful, to whatever degree, in moving research along for better treatments.

What did it mean to be accepted?

When I submitted my comments I never expected to be asked to participate on one of the panels, nor was I intending to attend the meeting, which was on the eve of a religious holiday. I was quite surprised when I was contacted by the FDA, but was caught in a dilemma. Participation meant missing an important time with my family. However, when we discussed it, we all agreed that this was such a great opportunity, and honor, to be able to represent the needs of the millions of Parkinson’s disease patients worldwide to the FDA. There was no way I could turn down that kind of invitation.

What was it like being there as a patient representative at the FDA event? Did you feel like your voice was heard?

I’ve participated in a number of seminars and conferences in the past, so there wasn’t much difference with the arrangement for this meeting. If anything, it was much more low key. Nevertheless, I definitely had a sense of awe when I arrived at the FDA complex. There were multiple buildings all over the sprawling campus. It immediately reminded me of the importance of this event compared to others I’ve attended. We were going to be presenting to the Director of the FDA’s Division of Neurology Products and nine other senior staff members. This is about as high up as we could go in our advocacy to advance Parkinson’s disease research.

During the meeting, it was difficult for me to tell how our presentations were going and whether they would have an impact on the FDA’s staff, although it appeared to me that they were listening intently. I was certainly concerned about the limited amount of time I had to speak. Several days later, though, I watched the webinar presentation of our event and I felt that we hit on the large majority of the most important areas of concern that our community has regarding current and new treatments. Only time will tell if the FDA will act accordingly with our hopes and suggestions. One small item that I noticed which suggests that they were listing involves a question on the Patient Questionnaire at the meeting. We were asked to choose from a list of Parkinson’s symptoms the ones that were most impactful on our lives. In my presentation, along with several of the other panel members, we noted that they didn’t include pain or orthostatic hypotension, two very common symptoms, on their list. I did, however, see that on the new follow-up survey the FDA is conducting, they have added both symptoms to the bottom of the list. So, at a minimum, they were listening as we spoke about these debilitating symptoms.

What did you learn while you were there?

Well, I can’t say that I learned much about the disease while I was there, but, of course, that wasn’t the purpose of the meeting. Our presentations were intended to educate the senior staff and decision makers of the FDA. That being said, what I did learn was the amazing dedication and effort that members of our Parkinson’s community will undertake in order to be heard at a forum. Members with severe difficulties traveled from all parts of our country in order to be able to speak for a few minutes. I was truly honored, and most definitely humbled, to be part of a panel with these dedicated individuals. If you haven’t yet seen the archived presentation of the meeting online, I suggest that you at least watch the members of the first panel speak to the FDA about their symptoms, it was amazing.

What are your hopes for future research in Parkinson’s disease?

We were asked by the FDA to limit our answers to their questions in the context outside of finding an actual cure. In addition, we were mainly directing our responses to the FDA’s role of controlling the approval and distribution of prescription drugs, more so than studying the potential impact of alternative therapies such as exercise, vitamins or other supplements, or the development of new medical devices to assist with symptomatic control (all of which are needed). So I’ll stay within that premise.

I see the need for a three-direction approach for future research. First, we need to continue and expand research into the biological science of Parkinson’s disease, as well as other neurological diseases with similar developmental processes, such as Alzheimer’s and ALS. With a better understanding of the disease cause, pathways, and progression, we will have a much greater chance of finding better treatments and potential cures.

Second, I would like to see research into the development of better symptomatic treatments. These treatments should be effective for multiple symptoms, have fewer disabling side effects, and have extended release and longer term activity. Although today’s treatments are somewhat effective treating motor impairment, they are greatly lacking in effectiveness for many other non-motor symptoms. Many of the drugs have side effects that can be more disabling than the symptoms they treat. In addition, it would be desirable for new treatments have ease of administration, such as oral, sublingual or inhalable as opposed to invasive surgery or device implantation.

Third, and most importantly, we need research to discover interventional treatments that can slow or halt the disease progression, if not totally cure it. This research should target the development of new drugs and vaccines that can intercede along all of the numerous biological pathways as the disease progresses. For example, we would find means to either stop the misfolding of proteins, prevent proteins already misfolded from accumulating in brain cells, or eliminate misfolded proteins which have already accumulated and are leading to cell death. Finally, I would like to see more research in alternative treatments, such as stem cells and human growth factors, in addition to traditional drug development. It’s imperative that the FDA allow our scientists to be as innovative as possible as they attempt to find treatments and cures for the most complex illnesses we have in brain diseases. With new interventional disease modifying drugs, along with improved symptomatic treatments and a proper exercise regimen, most, if not all of us dealing with Parkinson’s disease would be able to live active and productive lives for many years beyond our diagnosis.

For more information, check out the full video of Gary’s panel. And don’t forget to visit the site to connect with Gary and the more than 11,000 other PatientsLikeMe members living with Parkinson’s disease.

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Subjects no more: what happens when trial participants realize they hold the power in clinical trials?

Posted February 18th, 2014 by

When I first became involved with online communities back in 2002, I moderated a small forum for patients with ALS/MND in the UK at King’s College Hospital to connect with one another, share tips and support, and to help our care center to serve them better. One area that remains controversial even to this day is clinical trials. ALS is a rapidly fatal and incurable illness, and as a young researcher I was often trying to field questions that patients had about the trials process:

“Why aren’t there more trials taking place?”

“Why do we have to have a placebo?”

“If researchers think commonly available treatments like dietary supplements or antibiotics might slow my disease, why can’t I just take them anyway rather than being enrolled in a trial?”

Given the severity of their situation and the slow pace of clinical trials (it’s estimated that it takes over 10 years for a new discovery to go from the lab to the clinic), I certainly sympathized with their frustrations and did my best to get the answers they needed, asking my medical colleagues where I could and translating the technical jargon. A few patients decided to go a step further than challenging the status quo, however, and took actions into their own hands. One patient taking part in a trial sent her medication off to a private lab to test whether or not she was on placebo, and several others took off-label experimental ALS treatments like creatine or minocycline. Some of them even went abroad to China for highly controversial stem cell treatments. In the end, none of these interventions were shown to slow the disease, but it certainly showed what was possible when a smart, dedicated, and adventurous group of patients could organize online.

Fast-forward more than ten years and we see a very different world, where patients have a growing voice in treatment approval, the design of patient reported outcome measures, and increasingly share their trial experiences through social media. That’s what inspired us to write our latest article just published in the British Medical Journal which details our experiences with how members of our community have replicated a clinical trial, have started sharing their data with leading researchers to debunk alternative “cures” for their disease, and have even started taking trials into their own hands. We feel these are symptoms of a greater underlying problem that has been with the design of clinical trials from the start – trials are all take and no give.

Patients take all the risk with their health, their time, their bodies, and in return they are supposed to temporarily suspend their instinctual curiosity to know more about their health status through self-monitoring or to interact with other patients who might be in a trial, all for the good of science and other patients. Modern technology up-ends that power dynamic though, and now it’s almost as easy for a patient to measure their health status with patient-reported outcomes, wearable devices, or even lab tests as it is for their doctor. In that case, can we really say patients are truly blinded anymore? The double-blind placebo-controlled randomized controlled trial is a “gold standard” in medicine, but unless it adapts to the changing realities of the patient empowerment landscape, it will be inherently unsustainable.

That’s why our team is embarking on research this year to gain a new understanding of what patients want from trials and a new understanding of the social contract as patients want it written. Our hope is that we can bring them together with the scientific needs of researchers to conduct robust science. It won’t be easy, and it might not be popular with everyone, but it’s the best path we can see towards faster cures that respects the rights of patients to be partners, not just subjects.

PatientsLikeMe member PaulWicks


PatientsLikeMe and CISCRP Team Up with Sanofi to Spotlight Medical Heros and Clinical Trial Participation

Posted May 13th, 2013 by

CAMBRIDGE, Mass.— May 13, 2013—PatientsLikeMe and the nonprofit Center for Information and Study on Clinical Research Participation (CISCRP) announce a new partnership with global healthcare leader Sanofi (NYSE: SNY) to raise awareness about the benefits of participating in clinical trials. The partnership will focus on: spotlighting “Medical Heroes” (those who “give the gift of their participation in clinical trials” to advance medical knowledge); increasing patient awareness about clinical trial opportunities; and advancing PatientsLikeMe’s mission to accelerate research by analyzing patients’ real-world disease experiences.

The partnership will utilize the PatientsLikeMe’s Clinical Trial Awareness tool to match patients with relevant Sanofi clinical trials and CISCRP’s educational materials to inform patients and their support network about the clinical research process. Through the tool, patients can search for and be notified about trials based on age, sex, disease or condition, and location criteria.

As part of this collaboration, the clinical trial tool on PatientsLikeMe will become part of Sanofi’s clinical trial recruitment process for a range of conditions, including asthma, cardiovascular disease, diabetes and oncology. Additionally, the partners will work together to raise awareness among doctors of the benefits of clinical trials for their patients. This collaboration adds to CISCRP’s expansive outreach and education channel, including clinical research and health professionals, disease advocacy groups and patient communities.

“Tens of thousands of people are actively engaged in monitoring their health and sharing their experiences on PatientsLikeMe,” says PatientsLikeMe Co-founder and Chairman Jamie Heywood. “They’re not just helping each other, but contributing directly to research. They are heroes in the truest sense, ideal partners for medical discovery, and the best source for real-world, real-time evidence.”

“An integrative approach that combines patient education with recruitment communication is essential to engaging informed study volunteers,” says Ken Getz, CISCRP’s founder and chairman. “We’re very excited about our involvement in this groundbreaking collaboration.”

About PatientsLikeMe
PatientsLikeMe 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. PatientsLikeMe is a trusted source for real-world disease information and a clinically robust resource that has published more than 30 peer-reviewed research studies. Visit us at www.patientslikeme.com or follow us on our blog, on Twitter or via Facebook.

About CISCRP
The Center for Information and Study on Clinical Research Participation (CISCRP), now in its ninth year is a nonprofit organization dedicated to educating and informing the public, patients, medical/research communities, the media, and policy makers about clinical research and the role each party plays in the process. CISCRP provides a broad portfolio of products and services to educate and engage patients and enhance the participation experience. For more information about CISCRP’s educational initiatives visit www.ciscrp.org.


PatientsLikeMe Unveils New Tool to Match Patients with Clinical Trials Worldwide

Posted March 13th, 2013 by

Launch at European NHS Healthcare Innovation Expo Comes as PatientsLikeMe Chairman Calls for Revolution in Disease Measurement

CAMBRIDGE, Mass. —  — The U.S.-based patient network and real-time research platform PatientsLikeMe unveils its global clinical trials tool today at Europe’s Healthcare Innovation Expo 2013, hosted by the National Health Service (NHS). The free tool, unveiled today by Research Director Paul Wicks Ph.D., draws on open data to match patients from around the globe with clinical trials based on their condition and location. The U.S. prototype was launched last year and has already helped thousands of patients find suitable clinical trials.  The tool is available at http://www.patientslikeme.com/clinical_trials.

Last week, PatientsLikeMe Co-founder and Chairman Jamie Heywood spoke about innovative solutions to healthcare at the 2013 Nuffield Trust Health Policy Summit in London. Nuffield Trust is an independent source of evidence-based research and policy analysis for improving health care in the UK. Heywood returns to London tomorrow to speak on the Expo’s Masterclass Stage about the importance of measurement in building a learning health system.

In his Nuffield speech, Heywood called for a “revolution in measurement,” or what he calls “measurement-based medicine.” He adds, “We should measure the severity of each condition and its impact on the patient. The measurement should support the patient in life choices, clinicians in care choices and researchers in learning what’s effective. And every patient should be measured as part of the care process to the degree appropriate for the severity of their condition, so that their experience can be used to guide the next patient.”


Taking Psoriasis Treatment to the Next Level: An Interview with Dermatologist Dr. Jerry Bagel

Posted February 11th, 2013 by

Board-Certified Dermatologist Dr. Jerry Bagel, MD

A nationally recognized psoriasis expert, Dr. Jerry Bagel, MD, has been an investigator on more than 45 clinical trials seeking new treatments for psoriasis and other skin conditions.  In addition to treating patients at his private dermatology practice in East Windsor, New Jersey, Dr. Bagel serves as Senior Attending Physician at the Princeton University Medical Center and Director of the Psoriasis Treatment Center of Central New Jersey.   He is also a three-term member of the National Psoriasis Foundation’s Medical Advisory Board.  What investigational treatment for psoriasis is he currently studying?  And what does he wish more psoriasis patients knew?  Check out our insightful interview below.

1. What led you to focus your research on psoriasis?

I started studying psoriasis when I was resident at Columbia-Presbyterian in 1982-1985. At that time I would spend six months on in-patient service with 30 hospitalized psoriatic patients who would stay in for treatment for a month.   And I realized the burden of disease – the psychological, emotional and physical impact of this disease on people.

So when I opened up my private practice in 1985, I decided that psoriasis treatment and care of psoriasis patients would be a major part of my professional career and so I started seeing a lot people with psoriasis.  I started doing research in 1992 and then just kept working with pharmaceutical companies and developing our own protocols for research into the treatment of psoriasis, trying to find better treatments, safer treatments, more effective treatments.

2. Tell us about some of your most recent clinical trial findings.

The most recent clinical trial I’m doing as of today is IL-17 receptor antibodies with Amgen that is remarkable, using subcutaneous injections once every other week.   People are getting clear within 2-4 weeks, so it’s really a quite effective treatment.  I mean, obviously we don’t know [everything yet], but so far it’s been good at our facility, and the data I’ve read about it seems to be pretty good.  So I think it could take the treatment of psoriasis to another level in the sense that over half the people will probably clear and probably 85% will have a very effective level of clearing.  So it’s going to take it to another level of clearing given what existed before.

3. Speaking of what existed before, what current treatments are most effective in your experience?

Well, we still use phototherapy, but people have to come in three times a week for about 12 weeks. It works about 75% of the time, but people only stay clear for about six months maybe 75% of the time.  So in the end it only works well for about 50% of the people.  But it’s totally safe, so therefore the benefit ratio is high.  We also do all the biologic agents, like Enbrel and Humira and Stelara, and they’re quite effective treatments.

In fact, Enbrel and Humira are FDA-approved for the treatment of psoriatic arthritis as well – and up to 25% of people with psoriasis have psoriatic arthritis.  So it helps the arthritic component of psoriasis as well.   So we do use the biologics, although sometimes we have to tweak them with some systemic therapies.   Sometimes we use cyclosporine, sometimes we use methotrexate.  But the treatment of psoriasis today is certainly a lot better than it was say 10 years ago.

4. Do you have any dietary recommendations for psoriasis patients?

I think diet helps, but I don’t think diet necessarily clears psoriasis for very many people, if any.  But we know that psoriasis is associated with the metabolic syndrome, and many patients are obese and have diabetes, which are inflammatory triggers.  I think patients that eat a low-inflammatory diet – with more fish like mackerel, salmon or anchovies, and less red meat – tend to be better.  Staying away from fatty foods like animal fats, and eating more vegetable fats like nuts, tends to be helpful.  Also, I do think that weight loss tends to help people with psoriasis.  The other culprit is alcohol.  Alcohol is not good for people with psoriasis.

5. What is something you wish more psoriasis patients knew?

I wish more psoriasis patients knew that there are physicians providing effective care for psoriasis.  For instance, today I had a young woman who was 23 years old.  She came into my office with psoriasis covering about 20% of her body.  She’d been to three dermatologists before me, and two of them told her there was nothing they could do about it, and she should learn to live with it.  So I wish there was more education for people with psoriasis to know there are dermatologists or psoriasis caregivers who are interested in the treatment of psoriasis – and a better way of connecting people with psoriasis with the physicians interested in treating psoriasis.


Recognizing the Rare Disease Community’s Champions of Hope

Posted October 9th, 2012 by

PatientsLikeMe Is Proud to Be Partnered with the Global Genes / RARE Disease Project

Did you know that 1 in 10 people worldwide have rare and genetic conditions?

PatientsLikeMe was a proud sponsor of the 1st Annual Tribute to Champions of Hope, organized by our partner the Global Genes / RARE Project. Held on September 27th in Newport Beach, CA, the gala recognized outstanding individuals who are working to affect change in the rare and genetic disease community.  All proceeds from the event – which attracted celebrities, medical researchers, pharmaceutical executives and even Olympic champions – go to benefit programs for patients and advocates.

PatientsLikeMe

In attendance from PatientsLikeMe were Ben Heywood, Arianne Graham and Deborah Volpe, who got to hear inspirational stories of the work these champions of hope are doing.  Highlights included the Biotechnology Award given to Charles Dunlop of Ambry Genetics and the Mauli Ola Foundation, which organizes therapeutic surf experiences for kids with cystic fibrosis, and a preview of the documentary Here. Us. Now., which chronicles a family with twin daughters diagnosed with Niemann-Pick Type C, a rare, incurable and fatal genetic disease.  Lifetime Achievement Award recipient Henri Termeer, the former President and CEO of Genyzme, aptly summed up all those who received awards as “people that have a sense that if they focus, they can make a difference.”  (Click here to read about all eight Champion of Hope honorees.)

Even the Dessert Made You Think About Your DNA and the Impact of Genetics at the 1st Annual Tribute to Champions of Hope Gala

After uplifting musical performances and talks by celebrity presenters Jason George (“Grey’s Anatomy”) and Nestor Serrano (Act of Valor), the night ended with a heart-stopping, impromptu rendition of “Somewhere Over the Rainbow” performed by several rising singer-songwriters, including Chris Mann and Katrina Parker from the “The Voice,”  Elliott Yamin from the fifth season of “American Idol” and Gracie Van Brunt, a young girl battling a rare genetic disease called Shwachman-Diamond Syndrome who performed her own original song earlier in the evening.

Chris Mann, Gracie Van Brunt, Katrina Parker and Elliot Yamin (Left to Right) Performing at the Tribute to Champions of Hope

The gala was followed the next day by the 2012 Patient Advocacy Summit, a forum where patient advocates could discuss issues that directly affect them, from resources to policy. PatientsLikeMe Co-Founder and President Ben Heywood was featured on the Innovative Technologies and Platforms panel, sharing the stage with Dr. David Eckstein of the NIH’s National Center for Advancing Translational Sciences. Discussing the topic of “Accessing the Science,” they each gave an overview of how patient registries and clinical trials are promoting change and advancing medical research.

As a prime example, PatientsLikeMe joined forces with the Global Genes / RARE Project last year to create the RARE Open Registry Project, a resource for patients and families fighting rare and genetic diseases.  What makes it different from other registries is every time you enter data into the system, you receive information back showing how your data compares to others fighting similar diseases or taking similar therapies.  If your family is impacted by a rare or genetic disease, we invite you to join the registry today


How Social Media Is Changing Research (Part II): A Guest Post by MS Clinical Trial Participant and Blogger Jeri Burtchell

Posted September 13th, 2012 by

Today’s guest post is written by PatientsLikeMe member Jeri Burtchell (TickledPink), who has been living with multiple sclerosis (MS) for 13 years.  A tie dye apparel store owner and mother of two, she writes a blog entitled “Gilenya and Me:  My Story of Being an MS Patient, a Hypochondriac and a Guinea Pig.”  Her patient advocacy and social media presence led to her being invited to speak the Disruptive Innovations conference taking place in Boston this week.

Read Part I of Jeri’s guest post first!

MS Patient, Blogger and Activist Jeri Burtchell (TickledPink at PatientsLikeMe)

Because blogging a clinical trial from start to finish was unheard of, I attracted the interest of not only patients, but those in charge of clinical trials. They are interested in the impact of social media on clinical trials, and how they can utilize it to their benefit. Sites such as personal blogs, FacebookTwitter, and PatientsLikeMe are here to stay and people naturally want to share information.

I got a direct message on Twitter from Craig Lipset, who is Head of Clinical Innovation, Worldwide Research & Development for Pfizer. Social media brought us together to have a conversation about research that never could have taken place before the Internet. Everyone is more connected and approachable now. Naturally, I blogged about it.

But that was just beginning.  Tomorrow, September 14th, at 9:45 a.m. , I will be speaking along with Craig at the Disruptive Innovations conference, where the leaders in pharmaceutical research will be gathering to share ideas and come up with innovative ways of conducting clinical trials that take the “ePatient” into consideration. The 30-minute segment is entitled “Patient Leaders as Key Stakeholders in Clinical Trials,” and I will be there to represent – and put a human face to – clinical trial patients everywhere.

Knowing this is a chance of a lifetime for a trial patient to have the researchers as their audience, I wanted to reach out to those who have participated in past or current trials. My question to them is: “If you could ask or tell researchers just one thing about your own experience as a trial patient, what would that be?”

I plan to attend this conference and speak on behalf of all patients and put a face to the humans behind the data. I want to show them that we are connected now more than ever by social media. Researchers need to harness that power to their benefit. Soon they may use it to recruit and retain trial participants. I would like to see them provide a monitored gathering place for these trial patients to reduce the spread of misinformation as patients share data.

How Many of the 35,000+ Clinical Trials Currently Recruiting Do You Qualify for?  Use PatientsLikeMe's Clinical Trials Search Tool to Find Out!

To people who are considering a trial I recommend using tools like PatientsLikeMe and ClinicalTrials.gov to stay informed about ongoing research and find a doctor willing to support your interest in participating. Remember that not every trial will culminate in a drug that wins FDA approval. By joining a clinical trial you will be taking risks, but you may also be reaping benefits long before the general public will have access to the drug. Never forget that you are a pioneer and by entering a trial you are giving the greatest gift possible. Without volunteers we would have no medical advancements.

I hope that researchers never forget the impact they are having on the lives of people everywhere. They aren’t just going to work every day; they are the makers of miracles. Often patients are joining these trials as a last resort. The work of researchers gives us all promise for a brighter future.

I hope that patients everywhere will take one clear message away from this: NEVER GIVE UP! It would have been so easy that day to end it all. I was depressed and certain my life could get nothing but worse. But, by choosing to fight, I have changed my life forever and doors continue to open for me. By reaching out through social media I know I am not alone. You never know what tomorrow may bring, so don’t give up on today!

Editor’s Note:  Jeri isn’t the only PatientsLikeMe member blogging about her experience in a clinical trial.  See our interview with PGen study participant PF Anderson for another patient’s chronicle!


How Social Media Is Changing Research (Part I): A Guest Post by MS Clinical Trial Participant and Blogger Jeri Burtchell

Posted September 12th, 2012 by

Today’s guest post is written by PatientsLikeMe member Jeri Burtchell (TickledPink), who has been living with multiple sclerosis (MS) for 13 years.  A tie dye apparel store owner and mother of two, she writes a blog entitled “Gilenya and Me:  My Story of Being an MS Patient, a Hypochondriac and a Guinea Pig.”  Her patient advocacy and social media presence led to her being invited to speak the Disruptive Innovations conference taking place in Boston this week.

MS Patient, Blogger and Activist Jeri Burtchell (TickledPink at PatientsLikeMe)

One rainy day in April 2007, I was lying in bed, staring at the ceiling, talking myself out of suicide. I was having another MS relapse. This time it was attacking the part of my brain responsible for controlling emotion. As a result I was having panic attacks almost daily. Along with the emotional issues, I was also having trouble walking and horrible spasticity.

I had been diagnosed with MS for eight years at that point and, although I was on one of the FDA approved treatments, I was continuing to relapse three to four times a year. It felt like standing in the ocean; every time I would stand up and catch my breath, another “wave” of MS knocked me back down.

Deciding against suicide, I made some proactive choices that led to my meeting with the lead investigator of the Fingolimod (now marketed as Gilenya) clinical trials in Jacksonville, Florida. At my first appointment we discussed the Fingolimod trial called TRANSFORMS. I took the informed consent document home and went over all the risks and benefits with my family. After extensive baseline testing, I officially started the trial on August 20, 2007, a.k.a. “Randomization Day”, when I received my first dose of medication. I would return for regular testing many times over the next several years.

Joining the trial changed my life. I was very fortunate that I did not suffer any major side effects, and I am happy to say that my last MS attack to date was the very one which led me to contemplate suicide that day in April 2007.

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I was fascinated by the research during the trial. They did a lot of testing, and I have never felt more assured that my overall health was being tracked, observed and cared for as I did in the clinical trial. Since I did not have medical insurance, this was a plus on top of benefits I might be getting if on the real drug.

When the trial began, I wanted to know what to expect. I tried searching the Internet for a clinical trial from a patient’s perspective and could find nothing. I decided to share my experience with the world so others considering a trial might have their own fears put at ease. Thus my blog, www.gilenyaandme.com, was born.

I blogged all of my checkups and along the way something unexpected happened. Many people wrote to thank me for being the reason they felt able to overcome their own fears and join a clinical trial. We began connecting and sharing our personal experiences in a way only the Internet could enable.

Read Part II of Jeri’s guest post!