May is amyotrophic lateral sclerosis (ALS) Awareness Month. As many out there know, PatientsLikeMe was inspired by the life experiences of Stephen Heywood, who was diagnosed with this serious neurological disease back in 1998. Stephen’s brothers (Ben and Jamie) made many attempts to slow the progression of his condition, but their trial-and-error approach just wasn’t working. They knew there had to be a better way, and in 2004 PatientsLikeMe was created.
Every year, about 5,600 people are diagnosed with ALS in the U.S. It can affect any race or ethnicity and there is currently no treatment or cure that will reverse or even stop its progression. By getting involved, you can help change that.
The ALS Association has put together a terrific calendar of events called “31 Ways in 31 Days.” For each day in the month they’ve created a simple way to get involved and help raise awareness.
Looking for more info on non-profits during ALS awareness month? There are a bunch of organizations dedicated to the cause. A fellow PatientsLikeMe community member put together a great list in his forum thread Comparison of ALS/MND Organizations.
If you’re living with ALS, find others just like you in our growing community of almost 6,000 ALS patients on PatientsLikeMe. Learn what they’re doing to manage their condition with symptom and treatment reports, and share your own experience with a personal health profile or in the community forums.
Can you really understand concepts such as health, mobility or well-being without measuring or comparing them? See why Jamie argues that you can’t – and also why one’s “health span” may be more important than one’s “lifespan.” Click the image below to tune in.
*After clicking the image above, select the “08:45” link to your left to start the presentation.
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.
Recently, our ALS community mourned the loss of Persevering, a highly proactive three-star member who was known for his unfailing efforts to support fellow patients, record and share data, report website problems and recruit ALS clinical trial participants. Offline, he was also a tireless advocate for ALS awareness and research, attending conferences and lobbying on Capitol Hill. Persevering passed away on September 10, 2012, at the age of 42, and is deeply missed by both our members and our staff.
While we are unable to recognize every member who passes away on our blog, we wanted to take this opportunity to highlight how our community responds to loss as well share what happens to a deceased patient’s profile data. When our community managers are notified of a member’s death – typically by a family member, caregiver or another member who was close to the person – they add the date of death to the member’s profile. This automatically updates their icon nugget with a black band to show that the member has passed away. (See image below.)
Also, our members often create a forum thread about the member, to which the tag “In Memory” is added by other members or the community manager so that it is searchable and “followable” using this tag. In these emotional threads, members acknowledge the deceased member’s contributions, reflect on the loss to the community and pay their respects. Essentially, it’s a place for remembering a friend, telling stories, supporting one another, sharing funny memories and sending condolences to the family.
Each month, our community managers update our “In Memoriam” thread in the PatientsLikeMe forum with a list of members who have passed away during the previous month, and they include links to each profile. That way, members who haven’t logged on for a while or may have missed the news of someone’s passing can stay up-to-date. Members can also choose to “follow” that thread if they wish to be notified whenever there is a new monthly update.
As for the profiles of members who have passed, they effectively create a legacy of data on our site, as their profile pages remain accessible to our members in perpetuity. As a result, present and future members may continue to access these profiles to compare and learn from similar experiences. Persevering’s detailed treatment, symptom and disease progression data, for example, will live on as a rich source of information and insight for other ALS patients. What was his experience in the Phase II Study of NP001? Read his comprehensive treatment history here. What side effect led him to stop taking Riluzole? Find out here.
So as you can see, Persevering is still helping others today, and we thank him for that. We also want to recognize his contributions to our recent publication about NP001. We have dedicated this new work to him as it was inspired by his keen desire as a “citizen scientist” to analyze and understand the impact of NP001 on his ALS progression.
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.)
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.”
Held the night before the institute’s 7th Annual Leadership Summit, the gala event included the presentation of the first-ever “ALS TDI Lou Gehrig Award” to US Congressman Michael Capuano as well as special recognition for ALS TDI Chairman of the Board Augie Nieto, who has raised more than $30 million for the institute since 2007.
Congratulations to ALS TDI on 12 years of cutting-edge research and leadership.
As we mentioned in our blog post last week, our PatientsLikeMeInMotion program is off to a running start this year with more than 30 sponsored teams thus far.What does one of these run/walk/bike teams look like?Below is Team Sylvan, a 40-person team led by ALS patient Panda155.
Decked out in bright blue PatientsLikeMe t-shirts, Team Sylvan took to the streets for the ALSTDI Cure Is Coming Walk, held on May 7, 2011, in Lexington, Massachusetts.This is the third year that they’ve been sponsored by PatientsLikeMeInMotion, which makes them one of our veteran superstar teams.
Congrats to Team Sylvan and all of our sponsored teams for your efforts to raise funds and awareness for your condition.We’re honored to support you every step of the way. For more inspiring PatientsLikeMeInMotion team photos, check out our new Flickr slideshow.
Organizing a team for this fall? Sign up for PatientsLikeMeInMotion today.
At PatientsLikeMe, people with every type of condition are coming together to share their health experiences, find patients like them and learn how to take control of their health. The result is improved care for patients as well as an acceleration of real-world medical research.
Stay tuned to our blog for the latest happenings with our company, our patients and our mission of opening up the healthcare system.