Update: An ALS Caregiver’s View on Clinical Trials 12

6/7/2013:  Earlier this year, we were honored to have Jenny Dwyer author the following post focusing on how clinical trials must be changed to be more patient focused. Jenny’s role as caregiver and advocate in the fight against amyotrophic lateral sclerosis (ALS) provides unique insight into a family’s need for better clinical research options.

We are saddened to hear earlier this week of the passing of Pat Dwyer after a long, powerful struggle with ALS.  Jenny and Pat remind us of the need to change the status quo around clinical trials, because people’s lives are at stake.  Our thoughts and prayers are with Jenny and her family.

The following blog post is by Jenny Dwyer. 

Jenny and her husband Pat live in Seattle, Washington. Jenny is an amazing caregiver to her husband, who has been afflicted by ALS, and has become very well known in the ALS patient advocacy community. After meeting Jenny at the ALS Therapy Development Institute Leadership Summit, we invited her to share her caregiver perspective on clinical trials and drug development.

The opinions expressed by Jenny are her own, and do not necessarily reflect those of the Lilly COI Team.

My husband, Pat Dwyer, was diagnosed with Amyotrophic Lateral Sclerosis, more commonly known as ALS (0r Lou Gehrig’s disease) 7 years ago. Pat was involved in the commercial fishing and marine industry in Alaska and when he first started to lose feeling in his left hand, he thought years of working in the bitter cold were finally getting to him.

However, the strength in his hand began to decrease soon and eventually he started to experience twitches in his upper arm. At a regular annual checkup – our doctor referred Pat to a neurologist. After a few rounds of testing, Pat was diagnosed with ALS.

Pat and Jenny Dwyer

As the following months turned into years, Pat’s disease progressed (albeit slowly) and he continued to lose motor function through his left shoulder as the disease spread to his right arm, his diaphragm, his legs, and more recently, his speech and swallowing.

As far as medical care goes, we were lucky – it only took four months for Pat to get properly diagnosed. You see, many doctors haven’t seen ALS in their practice and typically ALS is diagnosed long after other medical conditions have been ruled out. Many stricken with ALS go mis-diagnosed as ALS is a condition that is classified as somewhat rare.

One factor that makes it very hard for ALS patients to find clinical trials is that unless your medical professional is involved in a trial, doctors don’t typically inform you about the other trials in progress.

Also, because patients can’t travel very easily as the most of the time they’re wheelchair bound, traveling to other geographies where trials might be occurring isn’t realistic in most cases.

Information is too hard to find

In my role as a patient advocate for my husband who’s fighting ALS, I’m lucky that I’m computer savvy. I can find information easily online and use the Internet to connect with others. However, if you don’t know where to look online, the lack of information on clinical trials is disheartening and frustrating.  There are tools such as ClinicalTrials.gov, NEALS, and ALS TDI which lists trials and if they do pop up in a search, that’s great.

Being able to access information about clinical trials is great, but I’m the type who really wants to learn as much as possible about how the drugs being explored might help my husband.  Given that drugs that have made it to clinical trials have gone through testing in the lab and in animals, there’s a wealth of information about them that I’d like to get my hands on. Even if the data was negative, I’d still like to know what the process and results were.

You spend many of your days with ALS frustrated.  Frustrated about the disease, and frustrated about the lack of information out there about what’s happening, and what didn’t work.  It’s not so easy to find that information, so I use a combination of Dr. Google and on-line networks and forums to try to keep up as best I can.  It seems it should be easier for patients to get information about clinical trials and potentially emerging new treatments to the people who might be affected most by it.

Endpoints, measures and patient reported outcomes

I understand the value of clinical research.  Pat participated in a NeuRx Diaphram Pacing System study in 2007, and we know the benefits of clinical trials first hand.  The DPS delayed Pat’s using a wheelchair, and helped us to run the business for longer than we would have been able to otherwise.  I’m grateful for all everyone involved in driving forward clinical research.

Still, there are ways that clinical trials could be improved, at least from the patient perspective and, I believe, to help find better treatments more quickly.  One of the things that seems limiting is the way trial endpoints are measured.  In many cases, trials measure only one endpoint.  For example, with ALS, there’s a Functional Rating Scale that measures ALS patients to assess progress.  However, this rating scale alone isn’t a sufficient measure to show the efficacy of a drug in terms that matter to a patient.

For Pat, his ability to swallow is almost gone altogether. So for him, if he was able to use a straw to drink a glass of water in 10 swallows instead of 30, that’s a huge difference. However, this measure isn’t taken with the Functional Rating Scale. I wonder if the real quality of life measurements for ALS patients aren’t missed in the clinical trials.

When assessing a clinical trial for my husband, I want to know the patient reported outcomes – I want a gauge of how a treatment might affect my husband.  I know Pat won’t be able to eat a steak independently, but if he can eat 5 bites of something without choking instead of just one bite, this makes a huge difference.

Communication on how trials fit into research and treatment plans

Another very important factor for any successful trial (for pharma and the patient) is to have an open and honest dialogue around the particular trial to consider how it fits into overall research and treatment plans.  Clear communication more regularly with patients and caregivers as they prepare to join a trial, in terms that are understandable to patients and with a treatment perspective in mind can help avoid some real frustration.

Take for example the case where a drug shows real promise in phase II, can’t there be some way to stay on the drug as they progress to phase III?  There was a recent trial of a drug called NP-001 that showed huge benefit to some patients who were receiving the high dose and low dose of the drug.  Some of the symptoms actually started to regress.  But then the trial was over, and because the trial was also trying to find a marker for the disease, it was almost a year before results came out.  During that time a couple of the patients, who had experienced huge benefit from the drug, died.  Had they been able to stay on the drug while the research was going on, maybe they would still be here.


NeurRx Diaphragm Pacing System, Synapse Biomedical

Another example, Dexpramipexole and the diaphragm pacer.  About mid way through the IIa phase, the Diaphragm pacer was approved for humanitarian use by the FDA. Patients in the Dex trial, who’s breathing functionality was failing, had to choose between continuing on with the trial, or receiving the diaphragm pacer.  The company that is running the clinical trial said that patients who chose to get the Daipahragm pacer could no longer participate in the clinical trial, and if the drug went to open label for clinical participants, they would not have access to the drug.  Even if they were a month or two away from finishing up their time in the trial.  People were forced to make life changing choices.

Clearly what these two examples show is that ALS is a disease that can progress rapidly and life conditions change while in the course of the trial. With ALS, because there aren’t any treatments, it seems to me that clinical trials should somehow benefit all parties involved, mainly the patients. Clear and cooperative dialogue between ALS patients and trial developers could go a long way in helping everyone involved survive.

ALS could be a huge win

ALS is a very expensive disease to fight.  One facet to ALS is that the veterans show a high likelihood of acquiring the disease – more than twice as likely than the general population. In fact, the DOD has classified ALS as a ‘presumptive disease.’  There is an ALS research program within the DoD budget.  It funds basic research and theraputic research,

If we are able to invest in more therapy development/research, and make some headway against ALS, consider how much money would be saved in VA, Medicare, Medicaid  and private insurance.  The payoff would be enormous.

The bottom line

ALS is 100% fatal.  There are no survivors of this disease, like there is with other diseases.  Technology is making it so people can live longer with their disease and that’s a good thing if you chose to go that route.  As a caregiver for an ALS patient, I want to feel I’m getting the whole picture of what’s available to my husband. I seek more knowledge about what potential treatments may be available and what impact they might have on my husband.

One thing that has stood out to me in the ALS community is how committed we are to fighting this disease. Also, we love sharing information. If we can help others afflicted by this condition, we’re all about finding a treatment  not only for ourselves – but for anyone who may benefit.  We love the notion of sharing and feel it’s the only way to make a difference against this horrific disease.


  1. wow! It is important for us to hear this message. . .stop, and really think about it. We talk about patient centered design of trials. . .so how do we let patients and caregivers get intimately involved in the design process. .and not 1 or 2 inputs, rather the global crowd!

    • Jeff K,
      The FDA will be holding an ALS specific meeting/hearing Feb. 25th. I think you should go! While you are not specifically in the ALS business, you are connected to it. (Via the author of the above message!)

      You are connected because you want to learn and make things more efficient for clinical trials. People with ALS (PALS) are some of the brightest minds around. They know what works, what doesn’t work, what they want, and can offer valuable advice on clinical trials that will translate over to all diseases.

      Trust me, the PALS and their caregivers who attend this meeting and speak, will be the best teachers you will ever have the pleasure of meeting.

      Think about it!

  2. Thanks Lilly for the chance to share my story with you, AND for all you are doing to include patients in the CT process. Figuring out how to involve the global crowd shouldn’t be that hard…ALS is hard, this is not! I say build a data base, ask patients what they want, make it a point to include patients at every meeting, and it will come together!

    Here’s another glimpse into the Dwyer ALS world. This was filmed in September. https://www.facebook.com/photo.php?v=3523644459080

    Again, I am honored to have been invited to be a guest blogger. Thank you so much.

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  7. Do you think creating a law that focuses on how clinical trial should be would be useful? Just like in some other countries I think in India, wherein people demanded a compensation for clinical trials in order to protect their rights.

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