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ALS advocate Archives | PatientsLikeMe
“Social media is the MOST important means of socialization, communication and education for patients and their caregivers today, particularly with a disease like ALS where our minds are still sharp and active inside our failing bodies. Sites like PatientsLikeMe, Facebook and other ALS sites allow me to discuss issues, share experiences, research new treatment options and keep up to date on the latest research.”
36 essays were submitted, more than 11,000 votes were tallied, and ultimately three scholarship recipients were chosen. While PatientsLikeMe member Susan Speranza (Susan702) was not among the finalists, we wanted to spotlight her inspiring essay about how social media has become an invaluable part of her life since her diagnosis with ALS (Lou Gehrig’s disease) in July 2011.
Here’s an excerpt describing the aftermath of her diagnosis:
“Last year I was given this diagnosis at the age of 47. I was living a busy, wonderful life with my husband and two children, working as a Physician Assistant (PA) and had just started a PhD program in Public Health. Of course when I went to the neurologist’s office that morning, I already knew it could be nothing else: The Dreaded Disease! After spending the last 20 years practicing medicine and several weeks feverishly searching every website I could find looking for a benign cause, I knew it could be no other. My life came to a screeching halt on that day and I spent the next few weeks isolated, depressed, hopeless, wondering ‘Why Me?” and worrying about the tremendous burden I had just imposed on my family. I desperately needed to adjust my ‘new life’ to this ‘new reality’. Fortunately, social media helped with that adjustment.”
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.”