The Value of Openness: The PatientsLikeMe Blog

This month marks the two-year anniversary of our flagship ALS community. It’s amazing to reflect on what we’ve achieved in just two short years. Not only did we build the world’s largest treatment and outcome sharing communities for ALS (1,800+ patients), MS (4,500+ patients) and Parkinson’s (1,300+ patients), but this year we began evolving into the powerful research platform we always envisioned we could be.

At the heart of this research initiative are our new community reports, which analyze the makeup of our patient communities. In January, we released our first MS Community Report covering factors such as first symptoms, age of diagnosis, disease type, etc. Recently we released our ALS Community Report, with the Parkinson’s Community Report to follow shortly. You can find the much-anticipated results on the blog as well as the ALS forum.

But that’s just the beginning. Your commitment to collaborative learning has now driven our evolution to the next level. On March 7th, we launched the ALS Lithium Study. As co-founder Jamie Heywood wrote in a recent blog entry, “Today, we allow patients to begin to answer how to treat ALS.” What could be more central to our mission than that? We are delighted to collaborate with Humberto Macedo, a patient, and Karen Felzer, PhD, whose father has ALS, on the study. “Together…we will run the first real-time, real-world, open and non-blinded, patient-driven trial,” wrote Heywood.

The question at hand is: Does lithium slow ALS? With almost 150 patients participating to date, we already have more than eight times the number of participants as the most recent published study about lithium and ALS. Stay tuned as this exciting and unprecedented study continues. As always, thanks to everyone - whether involved in the study or not - for sharing your treatment data. You made this study possible, and we intend for it to be the first of many across our communities. The age of patient-led research is here!

Finally, we are thrilled to announce the March 23rd launch of our beta community for Mood Conditions, which coincided with the New York Times Magazine article, Practicing Patients. If you know anyone affected by Mood Conditions such as depression, bipolar disorder, obsessive compulsive disorder (OCD) or anxiety, please invite him or her to join PatientsLikeMe today.

PatientsLikeMe was born of a passion to provide the best tools for patients to participate in their own care, share experiences and change the way medical research is done.Thanks to our members and the dedication of our growing team, our first community, ALS, has now been open to the public for two years! The community includes over 1650 patients, the U.S. members represent over 4% of all the ALS patients in the States.

Over three quarters of our members have entered substantive information about their treatment history and status. Each time a member adds information, that information benefits how other people care for themselves and heightens how we as a community contribute to medical knowledge and drug discovery.

Already we have published exciting findings from our community. For example, hundreds of ALS patients completed Paul Wick’s survey on Excessive Yawning and the results were published in a psychiatry journal (Acta Psychiatica Scandinavica). Another exciting development in ALS is first real time drug study - on the use of Lithium in ALS. More published and presented research will soon be featured on our blog and in a new section on the site. Each project demonstrates how we, as a community, can conduct research quickly and easily to accelerate the pace of gathering and disseminating new knowledge. SO THANK YOU.

To show you how the research process works and to celebrate our second anniversary, we have put together a report on our ALS community. In it we observe that the community is a good one to study in that it looks a lot like the ALS patient population at large. As is true for ALS generally, there are 3 men for each 2 women in the community and mostly sporadic cases (8% hereditary, 92% sporadic). And, PatientsLikeMe members experience all types of onset with the most common being leg and arm onset (39% and 37% respectively). The only real difference is that site members are a little younger than the average (48.4 at onset on the site compared to 55 generally). In the report, we also looked at how different ways to better understand ALS and connect patients. In the figure below, we look at the relationship between age at onset and onset type. Separating each age group, we see whether different types of ALS present themselves at different times of life. This can contribute to scientific understanding of the disease. Arm onset appears to affect slightly younger people than bulbar and leg onset. Also, when each number in the chart is a link on the site, it will help you locate others like you and of interest to you. See the next figure. We also report early stage research of our own. Specifically, we look at how patients are utilizing health services and how our members with different types of onset are doing over time. One surprising finding is how long some people report between experiencing their first ALS symptom and receiving a diagnosis. In the figure below, the most common length of time between onset and diagnosis was 12 – 18 months. There are also a number of patients who did not receive a diagnosis for several years. We are going to do some more research into these results.

Lastly, we begin to look at how ALS differs by onset. We see from our user reported Functional Rating Scale that bulbar onset patients experience a faster decline than users with arm or leg onset. People with bulbar onset experience the same level of decline in 8 months as leg and arm onset patients experience in 16 months.

Through member participation, we are gathering the information we need to better understand the course and characteristics of ALS. At the same time, we are creating methods to use patient supplied data to discover and evaluate the effects of new possible treatments. If you are an ALS patient or caregiver, please take a look at the full report (Note: you must be a registered user).

by James Heywood

Update (March 7, 2008):  PatientsLikeMe ALS Lithium Research released.

Does it work? 
On February 12th of this year, Proceedings of the National Academy of Sciences (one of the leading science journals) published a paper entitled — Lithium Delays Progression of Amyotrophic Lateral Sclerosis. After 10 years researching ALS, I believe it is fair to say this paper includes the most promising suggestive set of data from a clinical trial ever published. I say “suggestive” because there are many flaws with both the information presented and with the publication process itself. These flaws make it so that patients and their doctors are left trying to draw conclusions about the use of Lithium to treat ALS, without actually having any realistic confidence in the data or its meaning.

For a patient, there is genuine risk either way. Lithium is not a harmless drug, and, although it is widely used, it can have significant side effects if it is not monitored properly. In addition, the reality is that in several of the last clinical trials in ALS, including minocycline and topiramate, the patients in the treatment group did worse than those in the control group. So, fears about the risk of an unproven drug are well founded. However, there is also the risk of doing nothing. If the paper turns out to be even half true, the effect on the progression of the disease could be dramatic.

We also must consider the consequences of waiting for more information. For someone with a life expectancy of several years, the consequence is obvious. Unfortunately, the harsh reality is that the traditional medical research system will not provide any better data to patients for at least 2 years – that is, 6 months to start a trial, 15 months of evaluation, and 3 months to share the data. In fact, 2 years is being optimistic, if truth be told. History teaches us that it will most likely be much longer.

History also teaches us that patients sharing stories with each other will not answer the question alone. Chinese stem cells, herbal supplements, nutraceuticals — all have been discussed extensively on the internet with some claiming cures and some describing great harm; yet we have no definitive answer. Despite the thousands of postings, very little knowledge has advanced the treatment of ALS, and patients are still left unable to make effective treatment decisions.

We can and will do better
PatientsLikeMe was built to solve this problem and accelerate the transfer of knowledge about what works and what does not. Today, PatientsLikeMe has data on the progression and history of more than 1600 ALS patients - twice the number in the largest ALS trial in history. Even before the trial results were published, 50 patients worldwide who had elected to start taking lithium, in collaboration with their doctors, have been tracking their progression and blood levels on PatientslikeMe. This is more than twice the number of patients participating in the trial itself! We have data on historical forced vital capacity, the ALS Functional Rating scale, and a full symptom battery for most of the patients who have started, as well as for all the other non-lithium users in our system.

PatientsLikeMe is committed to solving this problem. We are collaborating with Humberto Macedo, a patient, and Karen Felzer, who’s father has ALS, to recruit all patients taking lithium. Together, with all the patients involved, we will run the first real-time, real-world, open and non-blinded, patient-driven trial. We believe we will have the power, within months, to begin answering the question of how much lithium modifies the progression of ALS. Unlike a blind placebo control trial, we are watching the use of this drug in the real world, and because of the number of patients and our system’s sophisticated data modeling, we can determine the significance of each reported change in each patient as he/she deviates from his/her predicted course. There are many risks to our approach, patient optimism, the placebo effect, uncertain quality, and many other variables will compromise our data. Despite these, and many other challenges, we remain committed to solving this problem.

Our Pledge to ALS Patients
We will use all our shared patients’ data to determine, to the highest predictive power possible, the effect of lithium on ALS patients in the real world. We will share that information in real time with all patients. We commit to displaying that information in a realistic manner that communicates the true confidence and uncertainties it contains. We will build a platform that allows patients, doctors and researchers the ability to drill down into all of the data in the system, to each and every data point, so that they can trust that our analysis is based on what really happened. We commit to engaging in an open and productive dialogue about our methods, so we can all learn to do this better – today and tomorrow.

What you need to do
Regardless of whether you take lithium or not, we need your data. The more patients that share their information, the more power we have to detect the effect of lithium, or any of the other 800 treatments in our system. We encourage all patients, including those who have chosen on their own in effective consultation with their doctor to take lithium, to join PatientsLikeMe and share your data with the world. We do not encourage any patient to start taking lithium. As noted above, all drugs have risks and, in general, ALS patients have experienced more harm than good trying experimental treatments. It is important to note that, either way, you help if you participate, because the more data we have, the more ability we have to answer the question of what’s working.

Realistic Hope
In the 9 years since my brother, Stephen, was diagnosed with ALS, we have been through so many cycles of hope and disappointment. We have tried treatments that turned out not to work, and we have tried treatments that were and remain unproven. Each time, we approach the data with a little more skepticism, as each time before it has been proven to be wrong. Someday a treatment will work. I hope and pray that lithium is the one, but I am realistic given the failures of the past. The realistic hope of PatientsLikeMe is that together we can accelerate the day when we know. We know most patients use PatientsLikeMe because they want to talk to someone like them and support their friends, they use PatientsLikeMe to share their insights; they use PatientsLikeMe, because, without question, we improve patients’ quality of life through the sharing of information. We value that greatly, but we also have higher goals, Today, we start achieving them. Today, we allow patients to begin to answer how to treat ALS, and that will help us answer it for all diseases.