On Friday, November 2nd, several PatientsLikeMe team members donned their finest to attend the 2nd Annual White Coat Affair, a charity gala benefiting the ALS Therapy Development Institute (ALS TDI).  Founded by PatientsLikeMe Co-Founder and Chairman Jamie Heywood in 1999, ALS TDI is the world’s most advanced research laboratory dedicated to ALS, also known as Lou Gehrig’s disease.

This gala fundraiser was held in conjunction with ALS TDI’s 8th Annual Leadership Summit, which included in-depth scientific presentations by top ALS researchers, a discussion panel with industry leaders and an awards ceremony honoring individuals who have made extraordinary contributions to ALS research and advocacy.  (View the summit webcast here.)  PatientsLikeMe was a proud sponsor of this annual gathering of the most influential minds in the ALS community.  In particular, we were honored to witness the posthumous awarding of the Stephen Heywood Patients Today Award – given annually to an individual who exemplifies what it means to be an educator, role model and advocate – to beloved PatientsLikeMe member Persevering (Rob Tison).

Held at the Fairmont Copley Plaza in Boston the next night, A White Coat Affair was an opportunity for summit attendees to unwind and socialize via cocktails, a dinner program, live music and dancing.  In honor of the theme, servers wore white lab coats, name cards were labeled like the periodic table and vases were filled with brightly colored liquid to resemble lab tubes.  A special highlight of the dinner program this year was the Young Perspectives on ALS segment, which featured the stories of two young people living with ALS (Corey Reich and Pete Frates) as well as four young people who are the children of ALS patients (Katie Shambo, Sam Ketchum, Jenn Sutherland and Alex Heywood).

As you might have guessed, Alex Heywood is the son of Stephen Heywood and nephew of PatientsLikeMe Co-Founders Jamie Heywood and Ben Heywood.  Both ALS TDI and PatientsLikeMe were inspired by Stephen’s seven-year battle with ALS, a rapidly progressive neurodegenerative disease with an average life expectancy of two to five years following diagnosis.

Approximately 350 guests attended A White Coat Affair, helping to raise $450,000 towards ALS TDI’s efforts to discover and develop effective treatments for ALS. After a decade of progress, the institute is entering a time of great promise, with several therapies in clinical trials and their own work on Gilenya (a drug currently approved for treating multiple sclerosis) and other potential therapeutics moving into the clinical realm.  Learn more about ALS TDI’s current research projects here.

To see more photos from the 2nd Annual White Coat Affair, visit the event’s Facebook page.

The term “hat trick” originated in 1858 after English cricketer HH Stephenson successfully bowled out three batsmen with consecutive bowls and he was presented with a hat to commemorate his feat. In June, PatientsLikeMe’s research team scored a hat trick of our own by publishing three new studies in scientific journals in just four days, bringing our total number of published studies to 27.

As employees at a start-up company, we all wear many hats – literally, as you can see from the photos below, and figuratively, in terms of our responsibilities as scientists, product developers and business people across different disease areas. For instance, in this trio of papers, we address research issues in multiple sclerosis (MS), ALS and mood disorders. Click on the study titles below to read the full papers and a big thank you to all of our patients for sharing your voices and making this research possible.

Monday, June 18th – First PatientsLikeMe study published.

The multiple sclerosis rating scale, revised (MSRS-R): Development, refinement, and psychometric validation using an online community

Members of our MS community will be familiar with the MS Rating Scale (MSRS), which is their primary outcome measure. We developed it a few years ago to address an unmet need for a brief, easy-to-use rating tool that covered more areas than simply walking. In collaboration with a neurologist, we sketched out our first version of the MSRS, which has now been used over 90,000 times by our 28,000+ MS members to share your progress, track your relapses and disability, and gain insight into how your treatments are working.

Published in the Journal of Medical Internet Research, this new study describes work to improve the MSRS to a revised version (the MSRS-R) and establish that the instrument measures what it is supposed to measure (reliability), that it correlates well with other instruments (concurrent validity) and that it allows differences between groups of patients (sensitivity to change). This process of “validating” a patient-reported outcome (PRO) is an important step in increasing the value of the data that we produce for researchers. Studies are underway right now to continue improving the instrument and compare it to doctors’ ratings of patient disability.

As with all outcome measures we develop at PatientsLikeMe, we have licensed the MSRS-R for anyone to use freely in their own research studies. This work also serves as a foundation for our resident predictive modeler and particle physicist Dr. Tim Vaughan to begin work on predicting the course of an individual patient’s disease using your MSRS scores!

Tuesday, June 19th – Second PatientsLikeMe study published.

E-mental health: A medium reaches maturity

The Internet has transformed many aspects of healthcare in the past decade, and to open a special issue on “E-Mental Health” in the Journal of Mental Health, our R&D Director Dr. Paul Wicks was commissioned to write a special editorial.

Available by clicking the link above, the paper describes the progress of online systems for people with mental health issues, from government-provided resources (such as this UK NHS Choices site about self-harm) to commercial, computerized cognitive-behavioral therapy programs like “Beating the Blues” and collaborations between different sectors of the health system. For instance, in the UK if you Google “suicide” there is a special message from the Samaritans mental health support service right at the top of the page.

At PatientsLikeMe, our vibrant mood community has been active since 2008, and our published research has shown that it provides improved outcomes to many of our members.

Thursday, June 21st – Third PatientsLikeMe study published.  Hat trick completed.

Mining Online Social Network Data for Biomedical Research: A Comparison of Clinicians’ and Patients’ Perceptions About Amyotrophic Lateral Sclerosis Treatments

As you probably know, the story of PatientsLikeMe starts with ALS and trying to find treatments that can improve the symptoms, including anxiety, stiffness, or constipation. Back in 2003, ALS nurse Dallas Forshew and Dr. Mark Bromberg published a small study describing massive variation in the way ALS doctors from 39 specialist centers treat the symptoms of ALS. This data was also described in our 2010 TEDx Berkshires talk about the value of crowd-sourced data.

In this new study published in the Journal of Medical Internet Research, we collaborated with researchers at the University of Utah to compare the passively collected ALS patient data from our system with what the clinicians said. Although they agreed in most areas, there were split opinions too, particularly when it came to the perceived level of efficacy that these treatments had. Click the link above to read more.

This paper is also notable for being our own Shivani Bhargava’s very first scientific publication! Shivani started with us as an intern, then became a research assistant, and has recently made a career change to start studying as a software engineer. A true renaissance woman!

Yesterday, our interview with ALS blogger and three-star member Rachael gave you a glimpse into what it’s like to live with ALS (Lou Gehrig’s disease).  Today we take a closer look using the data and experiences shared by our 4,844 ALS members, who comprise the world’s largest online ALS population.

ALS, which stands for amyotrophic lateral sclerosis, is a degenerative disorder affecting upper motor neurons in the brain and lower motor neurons in the brain stem and spinal cord. Some of the most debilitating symptoms include progressive weakness, atrophy, fasciculations (muscle twitches), dysphagia (swallowing difficulty), and eventual paralysis of all respiratory function.  Other commonly reported symptoms are shown in the chart above.

Given the severity of ALS symptoms, the life expectancy of an ALS patient averages two to five years from diagnosis, according the ALS Association (ALSA).  However, ALSA states, “The disease is variable, and many people live with quality for five years and more.”  Rachael, who is six years post-diagnosis and living a busy, active life thanks to assistive technology, is a perfect example.

What does assistive technology entail?  For many ALS patients like Rachael, this involves the use of medical equipment to aid basic functions such as:

• Walking (a power wheelchair)
• Breathing (a non-invasive ventilator or “bi-pap” machine)
• Eating (a feeding tube)
• Going to the bathroom (a catheter)
• Communication (an eye gaze system).

How well do these interventions work?  Click on each treatment name above to read evaluations from hundreds of patients about the effectiveness, side effects, cost and more.  In addition to these various types of equipment, one of the most commonly reported treatments for ALS is Rilutek, the first prescription drug to be approved specifically for ALS.  While it does not cure ALS or improve symptoms, it may extend survival or the time to tracheostomy (the creation of an artificial airway in the throat), which occurs when a patient is no longer able to breathe on his or her own.  Currently, we have 1,124 patients taking Rilutek, with 293 treatment evaluations submitted.

What do patients say about this drug?  We leave you with a sampling of comments that patients have shared on their treatment evaluations.

• “One day I was having tremors in my left arm. I took the Riluzole [generic name for Rilutek] and one hour later the tremors stopped. I know it is helping.”

• “I made a decision that 10% increased lifespan from onset was not worth being very sleepy all the time. I would rather require far less sleep each day than live slightly longer.”

• “It is a slight pain because you’re not supposed to eat for two hours before or an hour after, and I’m trying to keep weight on.”

• “I think this extended my time by at least six months. I started taking it about two months after my diagnosis. I’ve been told it’s more effective when you start taking it early like I did.”

• “Quit taking due to elevated enzymes in my liver. Drug caused increased hunger, protein cravings, and very sluggish feeling.”

• “Currently purchasing under Medicare as a tier 4 drug. When in the doughnut hole, the cost is approximately $985 per month.”

• “We can never know if Rilutek does any of us any good. If it doesn’t seem to be doing any harm, I believe it is better to take it than not to.”

This is just a sample of the wealth of experience and data to be found at PatientsLikeMe.  Dive in today to learn more about ALS.