Destined to Die for Not Fitting In?

I'm angry because I'm being punished for living too long...

During the last eight years, ever since I was first diagnosed with Amyotrophic Lateral Sclerosis (ALS), I have been doing everything possible to fight this horrible disease. In 2005, I was given two to five years to live, but deep-down I didn't believe it. However, two years later the harsh reality of this fatal diagnosis changed my thinking and decision-making process.

When my partner and I were living in New York City, I started falling for no apparent reason other than ALS. When I fell on the sidewalk in Manhattan and shattered my collarbone, I knew I could no longer ignore my deadly condition.

Leaving our New York life behind, we came to Boston in 2008 and immediately sought out the ALS team at Massachusetts General Hospital. They were welcoming, compassionate and attentive and, most importantly, they gave us hope in our fight against ALS. One of the first things they talked about was a promising new ALS clinical trial for a drug called Ceftriaxone. The only catch: I was required to be within two years of my ALS diagnosis and my breathing capacity needed to be above 60 percent. Since I checked both of these boxes and was young and relatively healthy, I was quickly accepted into the trial. A port was placed into my chest and I was off to the races!

My partner was trained to give me the drug and we went through the daily regiment for nearly nine months. Did it help me? I don't really know. My guess is that it probably didn't; just recently that trial was stopped since they found no evidence that the drug worked on patients. But even though it may not have stopped my ALS progression, it was a hopeful attempt at trying to save my life and I appreciated the hell out of that!

Fast-forward five years later. I am well past the "within two years of ALS diagnosis" mark and my breathing capacity is now at about 30 percent. But those things aside, I am still fairly stable. Hell, some would say I'm thriving! I can walk with assistance, talk, eat without a food-tube and I don't need to use a BiPAP machine for respiratory assistance.

What's so painful for me and people like me? It's simple. We do not qualify for the few meaningful clinical trials tied to treatments that could potentially save our lives. Truth is, like many others I have either lived too long or have some other physical limitation that would throw off the "scientific model" of a trial. In other words, we are the patients who are not like the others and we are being penalized for that. Right now, there are promising stem cell trials that many people like me, with long-term ALS, would give anything to be part of -- but I've been told I will not qualify. It feels so unfair.

So, what is supposed to happen with people with ALS (pALS) who are living longer lives? According to the ALS Association, approximately 20 percent of pALS live five years or more and up to 10 percent will survive more than 10 years and 5 percent will live 20 years. There are people in whom ALS has stopped progressing and a small number of people in whom the symptoms of ALS reversed.

I recently visited my neurologist, Dr. Amiram Katz, and he emphatically stated: "I believe open label trials should be available for whoever wants to participate. The medical community needs to deviate from standard protocols in ALS cases. Right now we are punishing people for living too long by excluding them (from participating in trials)."

Sadly, this issue goes beyond ALS patients. I was recently made aware of the powerful story of Nick Auden. In 2011, Nick was diagnosed with Stage 4 melanoma. He's tried several times to gain access to a clinical trial for PD-1 -- a drug that is showing positive early results combating melanoma. Although he's been very close to being accepted, last-minute complications have disqualified Nick and he has not been able to get the therapy he so desperately needs. Interestingly, Nick's neurosurgeon also believes that the trial protocols are outdated and that, in his case, the existence of brain tumors should not disqualify him from participating in trials.

Just like my loved ones and I are rallying to beat ALS, Nick and his family do not want to lose hope and stop fighting. He must gain access to PD-1 -- either on a compassionate or single-trial basis. The drug is currently being manufactured for trial by both Bristol-Myers Squibb and Merck. Nick's family and friends started this website to help him get the drug.

So what do people like Nick and I do? Sit around and wait to die, or find some way to obtain these promising therapies through a compassionate use or single-trial basis? Many will say there's too much risk involved. My answer: it's my risk, my life and my live. Let me have it!

Randy Pipkin is a consummate ALS Fighter and 2013 national campaign chair of Breakthrough ALS for the ALS Therapy Alliance.

He has also formed "Team Randy" for the ALS Association's Boston "Walk to Defeat ALS"