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.

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.

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.

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.)

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.

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. 

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!

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, Facebook, Twitter, 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.

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!

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.

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.

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!

“Before me, there were hundreds and thousands of other people with Parkinson’s who participated in clinical trials that gave me the ability to have the medications that I take today. If people today do not participate in clinical trials, there will be no cure. There will be no new medications.” – Parkinson’s patient Jean

Last year, PatientsLikeMe unveiled our Clinical Trials search tool, which helps you find upcoming studies that might be right for you.  We also introduced a matching feature that notifies you on your profile when you meet the criteria for a new study.  That means when your condition, gender, age and location are all a match.

But if you’re unfamiliar with the clinical trial process, the idea of participating in a study may be a bit daunting.  That’s why the National Institutes of Health (NIH) recently launched a new microsite called NIH Clinical Research Trials and You.  Here you’ll find a clear overview of how clinical trials work as well as a number of personal stories from both study volunteers as well as researchers.

Below are two volunteers’ stories to give you a sense of the different reasons people have for participating in research.

Jenny (Type 1 Diabetes)

Jean (Parkinson’s Disease)

Have you participated in a clinical trial?  Share your experiences in the comments section.

What’s it like to work at PatientsLikeMe?  We are continuing to reveal just that with our monthly blog series “A Day in the Life,” which features employees from different departments.  So far, we’ve interviewed User Experience Engineer Cris Necochea, Research Assistant Shivani Bhargava, Office Manager Alison Dutton and Research Scientist Timothy Vaughan. Today we hear from Arianne Graham, a Harvard MBA graduate who joined the business development team in 2010.  What current project is she most excited about?  And how is working at PatientsLikeMe different than her previous healthcare jobs?  Find out that and more in our interview.

1.  What’s it like to be part of the business development team?

When spending time with friends and family or even meeting new people, I’m always excited to answer that small talk question: “What do you do?” I happen to think I have the best job ever.

As part of the business development team here at PatientsLikeMe, my colleagues and I work with everyone who cares about patients (including providers, researchers, pharmaceutical companies, insurance companies, pharmacies, and many more!). We look at how the real-world data shared by our members can transform their businesses for the benefit of patients everywhere.

Sounds like fun, right? Most of the time, it really is. This is also hard work, though. Sometimes I feel as though the rest of the world is not yet ready to hear the patient’s voice. The truth of each patient does not always fit perfectly inside the constraints of a randomized clinical trial or a research protocol. But it’s here to stay, and we are actively finding ways to layer this emerging data source on top of the world in which we live. That’s reason enough for me to get up every day and come to work with my very talented team.

2.  What kind of projects are you working on at the moment?

I am often the first contact for individuals and organizations that contact us via partners@patientslikeme.com or support@patientslikeme.com. We receive multiple messages a day with ideas regarding how PatientsLikeMe might work with another company or integrate a new technology. It’s up to me to prioritize which opportunities to pursue now given the market landscape, and which to save for later.

One of our most basic and popular services is clinical trial recruiting. On any given day, I have to do something related to one of these campaigns. The members that use PatientsLikeMe are probably more likely than most to be motivated to try investigative therapies, to access better healthcare, or to simply participate in research. Finding the right patients for a study can be so difficult, but thanks to the demographic and health data patients report to us, we can easily let patients know about clinical trials for which they may be eligible.

The project I am most excited about, however, is a clinical trial of PatientsLikeMe as an intervention. We have partnered jointly with a pharmaceutical company and an integrated payor/health system to empirically test the effects of the website on patient activation and health literacy. Additionally, this is one of the first projects to really integrate PatientsLikeMe into the clinical encounter between doctor and patient. We haven’t publicly announced the details yet, so stay tuned. I can’t wait to tell you more!

3.  How have your parents – who are both doctors – influenced your career?

As the child of two pediatricians, I know I was exposed to the healthcare industry and certainly aware of it more early in life than most people. I noticed and appreciated that they treat their patients as people, not diseases. They remain committed to serving their patients, to helping them get better so they can lead productive lives. For them, the medical field is not so much a job as a calling. I know I felt led to serve, too. Even when I abandoned my pre-med plans in college, I knew that healthcare is where my heart is. I just found another way to pursue it.

I cannot imagine working outside of this industry because it appeals to me on so many levels: I can serve others, and I’ll never get bored. Healthcare is what we used to call in consulting a “big, hairy, ugly.” It is messy and complicated, it affects us all, and there are no clear answers. All of that makes this sector intellectually stimulating and extremely rewarding.

4.  You’ve worked for pharmaceutical companies, a hospital and a consulting firm.  How is working at PatientsLikeMe different?

I think all of my previous work experience has prepared me well because I lean heavily on what I know to be true in healthcare in my current role. While I knew exactly what to expect in pharma, hospitals, and in consulting, PatientsLikeMe has proven to be a bit like the Wild Wild West. We are creating a world that never existed before: where patients take charge, and their real experiences are being integrated into the healthcare system around them. There are absolutely no rules yet. It’s exciting and uncomfortable; inspiring and frustrating. And you know what? I wouldn’t have it any other way.

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Interested in making a difference in patients’ lives?  Check out our Careers page to see our current job openings.  Headquartered in Cambridge, Massachusetts, PatientsLikeMe is looking for a Marketing Coordinator, Community Moderator, Research Client Manager and more at the moment.

Earlier this month, we told you about a new study showing that ALS patients reporting their health status over the Internet (using a rating scale known as the ALSFRS-R) is just as reliable as a trained nurse rating the patient’s score.   Now, we’d like to share our interview with lead study author Dr. Thomas Meyer, a neurologist at Charité University Hospital in Berlin, Germany.  What role did PatientsLikeMe play in this research?  And what are the study’s implications for the future of clinical trials?  Find out that and more in our interview with Dr. Meyer below.

1.  When did you become interested in patient-reported outcomes?

In 2005, we first used tablet PCs in our outpatient department to capture patient-reported outcomes (PROs). To us neurologists, PROs play a crucial role. Many neurological conditions can be captured by means of PROs only – I am thinking of pain in neuropathies, spasticity in multiple sclerosis (MS), the subjective perception of movement ability in Parkinson’s syndrome and dyspnoea (breathing difficulty) in neuromuscular conditions, including ALS. Given this fact, we neurologists have always listened to our patients a little more carefully so that we can do a good job. Therefore, the systematic capturing of PROs is a natural process to us, and we are most happy to be able to support any advancement and positive development thereof.

2.  Your study showed remarkable agreement between the two ALSFRS-R reporting methods.  Were you surprised that they were so similar?

You are absolutely correct. Correlation here looks like a textbook example of medical statistics. We were indeed very surprised to find that the data were so unequivocal.  Notwithstanding, our previous experience with the offline electronic capturing of ALSFRS-R had taught us that the data captured in a personal interview are very close to those captured in computer-based self-assessments.

Then we took the next step and progressed from offline to online assessment. Other work groups had already shown good correlation between the face-to-face capturing of the score on the one hand, and data capturing over the phone on the other. That was very useful upfront information for us. Insofar, the success of our study didn’t come as a complete surprise to us.

3.  How did Dr. Paul Wicks, PatientsLikeMe’s Director of Research & Development, contribute to this research project?

Our great interest in the issue of PROs in ALS goes back a long time. Nevertheless, we were very much aware of the fact that PatientsLikeMe is by far the most experienced organization with regard to PROs worldwide and also the one that identifies with this topic most. So, a small group of four of us flew from Berlin to Boston to introduce our clinical trial to PatientsLikeMe and to learn from their experience. Once we had concluded the study, we flew to Boston once again to see Paul and to prepare the paper. We profited immensely from Paul’s input, and he gave the manuscript a superordinate perspective.

So it is for a good reason that he is listed as co-author of this publication. Overall, this scientific research project was a collaboration between the Charité University Hospital and PatientsLikeMe. I believe it is also an important, gratifying and affirming experience for PatientsLikeMe to see that in terms of methodology the online capturing of PROs is at least equal to an interview conducted face-to-face. I can even imagine situations where the online mode of capturing PROs is actually better than a personal interview, especially where rather complicated and very private issues are addressed. This is just one of the many points we discussed vividly with Paul.

4.  Do you believe online patient reporting will become an acceptable practice for clinical trials?  What are the ramifications if it does?

I can very well imagine that the online capturing of PROs will one day become an integral part of clinical trials. It is quite an obvious thing; however, owing to regulatory requirements, it will be quite some time before it will actually be possible to implement this. The bottom line is that clinical trials will have to be conducted for each score demonstrating equivalence between paper-based and web-based capturing. Not all of the scores have actually been evaluated for online capturing. Another critical point surely is Internet access.

Having said that, it certainly also depends on the patient group and the actual medical condition under examination. I suppose that from a medical-ethical point of view it is problematic to exclude patients from a trial simply because they are unable to realize an online completion of the score. In this regard, I believe the first step to be taken must be to demonstrate equivalence between online and offline capturing of the score. Then one could give patients the option of using online assessment in the context of participating in a clinical trial and see what they would prefer to do.

The benefits would be immense. This method could highly enhance the quality of the data, the efficiency of data capturing and, not least of all, it would help reduce the costs of a clinical trial. I believe that online assessment will be a matter of course in the future, but not immediately.

You may remember our interview with ALS advocate Debra Quinn from last fall.  Today, we’d like to introduce you to another ALS patient activist in our midst:  Tom Murphy, a PatientsLikeMe member since January 2011.  As part of the ALS Treat Us Now nonprofit group, Tom and several other ALS patients around the country have launched a petition on Change.org that’s gaining steam.  Their goal?  20,000 signatures.

Addressed to “corporate citizens, FDA executives and neurologists,” the petition asks that “compassionate use” drugs be made available to ALS patients now.  What this means is that Tom and the Treat Us Now group want promising Phase II drugs showing safety and efficacy to be made available to ALS patients prior to FDA approval.  The reason is simple. ALS patients, who face an average life expectancy of two to five years following diagnosis, don’t have time to wait.

Check out our interview with Tom to learn how he became part of ALS Treat Us Now and what two experimental drugs his group is focused on in particular.

1.  How did you get involved in the “Treat Us Now” movement?

The ALS community seems to be a very close-knit group of people who readily share information and collaborate openly.  This is often related to the objectives of spreading ALS awareness, accelerating new treatments for people with ALS, and most importantly, doing whatever we can to assist in the identification and development of a cure for this rare and terminal disease that has been with us for way too long without an effective treatment or hope for a cure.

Since being diagnosed with ALS in December 2010, I (and many others like myself) have developed a very extensive “network” within this community utilizing blogs, email, Facebook, Tumblr, Twitter, LinkedIn, PatientsLikeMe, ALS Therapy Development Institute (ALS TDI), the Muscular Dystrophy Association (MDA) and the ALS Association (ALSA) and other ALS-related internet sites.

This network included Ms. Sherron Greene from Kaplan, LA (whose brother Kendall was officially diagnosed in October 2010), who was working with the Treat Us Now group and reached out for my assistance with some research and ALS points of contact at various medical facilities involved in ALS clinical trials. I wanted to be part of this Treat Us Now team and the rest is history.

2.  What kind of response have you received to the petition so far?

As of 12:00 p.m. EST today, we’ve gotten 10,520 petition signatures.  I’d ask folks to please take the time to watch the complete video below about my friend Kendall Saltzman, and you will begin to understand much better the urgency of our fight regarding compassionate use drugs for people with ALS.

3.  Tell us about the two drugs – dexpramipexole and NP001 – mentioned in the petition.

Highly related to the Parkinson’s disease drug pramipexole (Mirapex), the neuro-protectant dexpramipexole (Empower) may slow ALS progression by keeping the power on in deteriorating motor nerves. Dexpramipexole is thought to maintain mitochondrial function in people with ALS by detoxifying reactive oxygen and nitrogen species.

In the Phase II study, ALS progressed 31% slower in patients taking 300mg of dexpramipexole daily compared to placebo.  It also appeared to be safe and well-tolerated.  The drug, now licensed to Biogen, is currently being tested at the Phase III stage in about 800 patients at 81 sites worldwide.  ALS TDI calls it “extremely promising.”

In May 2011, I was one of the first 10 ALS patients to be enrolled in the Phase III trial at the University of Virginia.  The FDA fast-tracked dexpramipexole in 2009 due to the need for a more effective treatment for ALS.  Initial Phase III results are expected in 2013.  (Learn more about dexpramipexole here.)

The experimental drug NP001, administered by intravenous injection, is still in the Phase II clinical trial stage.  NP001 may lower the levels of activated cytotoxic macrophages in people with ALS, thus reducing inflammation and further injury to the motor nerves.  The Phase II double-blind, randomized, placebo-controlled trial is ongoing at several US locations.  Unless an early usage allowance is granted, it may require an additional 5-7 years of testing.

Led by top ALS researcher Dr. Robert G. Miller of Forbes Norris MDA/ALS Research Center in San Francisco, this trial is notable as the scientific foundation of NP001 is extremely sound.  Andrew Gengos, CEO of Neuraltus, the manufacturer of NP001, says, “Our hope is that if we can reduce or eliminate the neuroinflammation, it will have a beneficial effect on the underlying progression of ALS.”  (Learn more about NP001 here.)

4.  Beyond signing the petition, what else can people do to become ALS activists?

Take part in the activities and initiatives of the ALS Association (ALSA), the Muscular Dystrophy Association (MDA), the ALS Therapy Development Institute (ALS TDI, founded by PatientsLikeMe’s Jamie Heywood) and Treat Us Now.

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What does PatientsLikeMe think about compassionate use?  “Our society needs to rethink the balance of risk and reward to allow patients the right to pursue the treatments they believe can help them,” says Co-Founder and Chairman Jamie Heywood, whose brother Stephen died from ALS. “This is a complex problem with many balancing issues, but I believe that there needs to be a mechanism for those that consent and who understand the risks to pursue options they chose regardless of regulatory status. Those facing illness should not be denied the right to self-determine their own path to life, liberty and the pursuit of happiness.”

(This post has been repurposed from an article written by PatientsLikeMe’s Paul Wicks for the scientific journal Clinical Investigation.)

But when it comes to clinical trials, can we afford to let it be?

“Information (or data) wants to be free” – so goes the mantra of hackers and data activists. In the past this meant computer geeks hacking into secure private networks to fulfill their own curiosity or liberate secret knowledge. Today, voluntary “data liberation,” as practiced by governments and corporations, is relatively commonplace and semiorganized groups with data freedom agendas, such as Wikileaks and Anonymous, have entered the mainstream consciousness.

For me, it feels increasingly challenging to delineate the margins where free data is good or bad. A highly networked, mobile-enabled popular uprising is considered a “revolution” when it’s against an oppressive regime, but considered a “menace” when it is a disenfranchised mob rioting in a western democracy. Bravely recorded videos of civilians attacked by autocratic regime military forces are essential in prosecuting crimes against humanity, but videos leaked from within a democratic military are a “threat to national security.” So while data itself may want to be free, we don’t always want it to be. And so, to clinical trials.

From a societal perspective, the requirement for any trials conducted in the USA to register on ClinicalTrials.gov can only be a good thing in preventing past sins, such as suppressing negative trials or changing end points. As the US government makes this data open, it also allows repurposing. For instance, PatientsLikeMe imports the complete dataset from ClinicalTrials.gov every night to let our membership know (free of charge) about the 30,000+ active trials for which they may be eligible. So far, so good. But what if even more clinical trial data were free?

Read the rest of R&D Director Paul Wicks’ editorial in Clinical Investigation here.

Last June, we shared how our award-winning Clinical Trials tool was a product of “Wow Week,” which takes place every sixth week here at PatientsLikeMe.  It’s essentially our version of Google’s “20-percent time,” or a period of unstructured time where our engineers have the freedom to work on their own ideas.  We believe it fosters passion, experimentation and risk-taking.  In fact, we know it has given how many other site improvements were conceived during “Wow Week” in 2011.

The list is quite long (kudos, engineering team!), so we singled out three “Wow Week” projects that seemed to really resonate with our 128,000+ members.

MyCycle

Engineering Team Credits: Amy Newell, Doug Martila, Ben Zack and Scott Listfield

For women, your menstrual cycle can be intricately tied to your health condition(s), as well as how you feel. Now, with MyCycle, you can monitor your cycle length, view it next to your other health information and predict the next start date.  (If you’re a female who has not indicated your gender on your basic information, you’ll need to do so in order to receive access to this feature.)

Fuzzy Dates

Engineering Team Credits: Jeffrey Chupp (no longer with the company) and Michael Berkowitz

Are you often fuzzy about your dates?  So are most people.  In recognition of the fact that photographic memory is a rare trait, you can now specify dates with varying levels of accuracy or “fuzziness” – the year (2008), the year and month (May 2008) or the full date (May 22, 2008).  Whatever you can remember is better than no data at all!

Mobile InstantMe

Engineering Team Credits: Steve Hammond, Thomas Mayfield, Joe Rodriguez, Keenan Brock, James Kebinger and Doug Martila

For InstantMe (our daily log of how you’re feeling) to be more instant, we needed an app, right?  This mobile website/application uses HTML5 to support the tracking of your InstantMe scores on your iPhone, iPad or Android smartphone.  Learn more about what it can do here.  (Note that this feature is now open to all members, regardless of their star level.)

Are we right?  Did these new tools and features add more “Wow” to your PatientsLikeMe experience?  We’ve love to hear your thoughts in the comments section.  And as always, stay tuned to our Release Notes page for the latest technology updates and innovations.

What kinds of things do we cover in our monthly newsletters for members? Take a look at the excerpt below from our July edition. Also, in case you didn’t know, anyone – whether a PatientsLikeMe member or not – can view our current and past newsletters in our Newsletter Archive.  See what we’ve been up to recently, and if you are member who’s not opted in to the newsletter, sign up today.

MONTHLY MUSINGS

How can you stay connected with PatientsLikeMe – and other patients?

Let’s say you’re a new member. You’ve completed your profile, but you haven’t connected with that many other patients yet. Some weeks, you forget to check in and update things at PatientsLikeMe because life gets in the way. What can do you to stay on top of your profile as well as what patients like you are learning? Here are four easy ways to get more involved and stay better connected:

Saved Search Alerts
On the Patients tab, you can search for patients just like you using filters such as condition, gender, age, treatment and more. Now, you can also save your searches and get an email notification anytime someone who meets your search criteria joins. Simply click the yellow bell icon to turn on these alerts.

Profile Update Reminders
Things happen with your health all the time – not just on the day you’ve got a doctor’s appointment. Why not sign up to get regular email prompts to update your profile? It’ll help you capture subtle changes, spot emerging patterns and have a complete record for your Doctor Visit Sheet.

Mobile InstantMe Update
Has something with your health put you in a good or bad mood today? Update your InstantMe survey on the go using your iPhone/iPad. (See “This Month’s Site Improvements” below for more details on this new application (or “app”), currently available to three-star members.

Thread/Profile Subscriptions
Have you noticed that certain PatientsLikeMe members have a similar health status as you…but are trying a different treatment? Follow their experiences with other treatments by “subscribing” to their profiles and/or forum threads. Their updates will then automatically feed to your homepage.

THIS MONTH’S SITE IMPROVEMENTS

Thanks for your awesome feedback as we continue working to improve our site. In this section, we’ll keep you up-to-date on new tools and features designed to make your PatientsLikeMe experience even better.

Here are our top functionality enhancements for July:

Are you an iPhone or iPad user? We have taken the first steps towards an app that will allow you to update your InstantMe survey on the go! No matter where you are, you can now share how you’re feeling and why.

Before rolling it out to everyone, we’re first giving our three-star members access to this app to help us test it out. (If you earn three stars, you’ll automatically see the button to download the app on your profile.)  Stay tuned as we work to expand this new and exciting functionality to all members as well as other types of mobile devices.

Last month, we introduced our clinical trial search feature, and since then, you’ve given us a lot of helpful feedback. Thank you! Here are some of our recent upgrades based on your input.

• Now you can select “Dismiss All” to clear out a large number of clinical trial matches – instead of having to delete them individually.
• Want to search “closed trials” that are no longer actively enrolling? Now you can do that too. This is helpful for past trial participants as well as those taking a drug that may be under investigation.

Recently, PatientsLikeMe Chief Marketing Officer David S. Williams III had the opportunity to participate in a podcast interview with the industry magazine PharmaVOICE. Along with Bonnie Brescia, a Founding Principal of BBK Worldwide, he fielded questions about how the new alliance between our two companies can both enhance and expedite the clinical trial recruitment process. Listen in to hear what each had to say about what this partnership means and what we hope to achieve together.