13 posts from March, 2012

MS Awareness Week: Real-World Descriptions (and a Poem!) from Our MS Community

Posted March 12th, 2012 by

We kicked off MS Awareness Month by sharing some key facts we’ve learned from our MS community (26,000+ members and counting).  Now, we are digging a bit deeper in honor of MS Awareness Week.  You’ve already heard about common MS symptoms as well as frequent topics in our MS forum.  But what it is really like to live with MS – and how does MS impact not just those diagnosed with the disease, but all of us?

The National Multiple Sclerosis Society (NMSS) has put together a video showing all of the ways MS impacts individuals, families and society.  For example, did you know that MS costs the US economy $28 billion each year?   Or the average MS patient $69,000 per year?  Another startling fact is that the average person with MS leaves the workforce 10 years after diagnosis.  (Most are diagnosed in their 20s and 30s).  Check out the video above to discover more about how MS impacts you, even if you don’t know anyone with the condition.

As for an accurate picture of life with MS, the best descriptions come from those who know best – MS patients.  In a recent discussion in our MS forum, patients asked each other what one word they think best sums up life with this chronic, often disabling disease of the central nervous system.  What were their responses?  Here is a word cloud illustrating 30+ answers submitted by our community.

A Word Cloud of Members' Answers to "Describe MS in One Word"

Putting words together in a full portrait, below is a poem by friendinflare, a three-star MS member who has lived with MS for 10 years.  Not only does she depict some of the issues MS patients face, but she also speaks to the relief that comes from sharing your experiences with others like you.  (At PatientsLikeMe, you can share treatment and symptom data, status updates, private messages, forum discussions and much more.)

In early November, I discovered a website.
A place where MS’rs can talk about our plight.
Our symptoms are as varied as our usernames.
There are threads for information, advice and even games.
We have legs of jello, vision that’s blurred,
And limbs that tremble from damaged nerves.
We still remain hopeful – but no one’s been cured.
“Daily injections will help” our doctors have reassured.
Our fatigue gets so bad we are glued to our beds,
We have cognitive issues from lesions in our heads.
Most don’t understand us, we’re a breed of our own.
We talk everyday without picking up a phone.
Our computers are a lifeline to hope,
Letting us talk with MS’rs from all over the globe.
Even though fights can break out at lightning speed,
Members always support those in need.
Here’s to everyone at PatientsLikeMe.

Getting a clearer picture of MS?  Good – but there’s even more to come.  Stay tuned for our two-part interview with an MS patient who authored a book called “The Dumbest Things Smart People Say to Folks with MS.”


Meet ALS “Treat Us Now” Steering Committee Member Tom Murphy

Posted March 9th, 2012 by

ALS Activist and Petition Co-Author Tom Murphy

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?

ALS Treat Us Now Is a Nonprofit Organization Dedicated to Accelerating Access to ALS Treatments

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.”

US and Canadian Locations for the Dexpramipexole Phase III Clinical Trial

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.”