Dear Stephen

Dear Stephen,

I’ve been meaning to write to you for awhile. We’ve only met a few times, but as shared adversity tends to do, I feel like you and your family are a part of my own. While we probably wouldn’t have met if not for ALS, I’ve often found myself wishing we had been diagnosed at the same time. Given our similar situations with a young family and your sense of humor, I imagine sharing the journey into paralysis with you would have been a hell of a lot more fun.

But with all that you and your family have done for the ALS world, I find myself incredibly grateful to have known you at all. I know guys don’t normally say this kind of thing to other guys, but you’re a hero to me. Forget Superman, Michael Jordan, or the latest over-hyped celebrity. You have been defying a killer disease with courage and tenacity for more than 8 years. You’ve done more than fight to stay alive, you’ve essentially made ALS irrelevant to how you approach living.

You inspire me to do the same. Whenever I question my ability to keep going, I think of you and your son Alex, and how you interact. You are a wonderful husband and father. I recall goofing around with Alex at a party in your parent’s backyard. We chatted and played with pop cans for about 10 minutes. In that little time it became clear to me: he is a wonderful young man and loves his father infinitely. I aspire to the same relationship with my daughter.

I’d also like to thank you for all of your practical advice. I share your willingness to embrace technology and have learned a great deal from your progression to wheelchair, feeding tube, and ventilator. I now have the same wheelchair as you, a damn nice one I must say. Perhaps it’s because I’m an only child, but I’ve come to think of you as a kind of older brother, setting an example for me to follow. Minus the beatings.

Most importantly, I want to thank you for allowing the world into your life. As unwanted a role as it might be, you and your family have proven to be outstanding leaders in the ALS community. Honest, open, resilient, and above all, human. My family and I are and always will be committed to supporting you and the ALS Therapy Development Foundation.

Your brother in the fight,
Scott

Epilogue: Stephen Heywood passed away last Sunday morning following the accidental disconnection of his ventilator. I join the entire ALS community in sending our love and sympathy to his family and friends. I meant to send him an email like this some time ago and never got around to it. I hope he receives it somehow. While deeply saddened, I celebrate his extraordinary life. He is still my hero.

Place Your Bets: Of Mice & Men

So currently we have one rather pathetic drug to treat ALS. But surely researchers are working on something better? Optimistically speaking, the answer is yes. Scientists have been conducting clinical trials for years, with rather meager resources, against an enormously perplexing foe. Yet the more I explore the world of clinical research, the more I am overwhelmed and frustrated. Pessimistically speaking, it is a cavernous maze of restrictions, guesswork, and secrecy. And it produces results slower than an ALS patient tying their shoes with chopsticks in a snowstorm.

It’s impossible to paint a clear picture. Generally speaking, we still don’t know squat about why ALS occurs, and little more about how it progresses. Thus far it seems to be the Mount Everest of neuromuscular disease. Scaling it has proven exceedingly difficult, and will require a virtual mountain of money to reach the summit.

Currently, researchers are very much focused on the mechanics of the disease, trying to get a sense of what is actually happening. Technically speaking, they talk about things like excitotoxicity and apoptosis. They write papers with titles like “Pharmacological activation of mGlu4 metabotropic glutamate receptors reduces nigrostriatal degeneration in mice treated with 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine.” They use their incremental findings to make educated guesses about what to try next. But the pace is glacial, and quite often they find themselves shooting in the dark.

However, advances in the last 10 years have enabled doctors to genetically engineer mice with ALS. Theoretically speaking, we can test a potential treatment on mice more quickly and cost-effectively. But it still takes about 6 months to run a mouse trial, and thus far, drugs that have shown a small improvement in mice have yet to work in human beings. Even more frustrating, most of the drugs tried thus far are not even targeted at ALS. Researchers are largely confined to drugs already approved for other conditions.

Some people question whether the mouse model is an appropriate predictor of success. Yet a thorough analysis of the data suggests otherwise. There is no standard by which to test new drugs in mice, making the results nearly impossible to compare. One study on a drug may show a 10% improvement using 4 mice, and another using 20 shows no improvement at all. The folks at ALS TDF believe it may take as many as 48 mice to eliminate random effects in a given sample (24 getting the drug, 24 in a control group).

Financially speaking, this makes the climb much steeper. Each engineered mouse costs $300, and budgets already limit the number of drugs tested each year. Convincing the research community to spend more money on fewer drugs will not be easy. But until a standard is in place, we’ll continue to spend millions of dollars each year for human trials based on minuscule and unduplicated improvements in a handful of mice.

Frankly speaking, all of this creates a shitload of confusion for patients. How are we to weed through all this information? How are we to determine who to trust is breeding the best horse to win the race for a cure? How are we to decide which clinical trial is worth betting on?

Unfortunately, my options are pretty limited at the moment. Mathematically speaking, I have one. There are currently 31 clinical trials for ALS in the world, 21 of them in the U.S., and only 1 accepting patients at a clinic in Minnesota. And guess what? The one trial I could feasibly join is testing a combination of 2 drugs that individually showed no effect in mice or men. Sounds like a pretty lame horse to me.

But the fun doesn’t end in picking a horse. Pragmatically speaking, the rules which govern a clinical trial are hugely constraining. Typically you must have a breathing capacity of over 60% of normal, been diagnosed within 3 to 5 years, and you can’t take other drugs. Furthermore, depending on the number of dosages being tested, only half the patients receive the actual drug. The rest get a placebo. Even if you’re lucky enough to get the drug, you won’t likely have access to it after your part in the trial ends, unless the drug company is feeling generous.

So, given that a trial usually takes 12-24 months to recruit, test, and report, the average ALS patient can only participate in 1 or 2 before they die. But even if you have an atypically long progression, you’re prevented from joining anyway, lest you skew the results. Although I’d gladly take the tradeoff.

I hope I don’t seem overly critical. Fundamentally speaking, the system is rigorous because it has to be. People’s lives are at stake. Neurology doctors and scientists dedicate their entire lives to chip away at a mountain of work, and I have an enormous respect for their efforts. But at the end of the day, clinical research is a business. Careers and funding are on the line. It’s all about promise, potential, and hope. And let’s be honest, patients are desperate to hear that a cure is just around the corner.

Too often, however, those promises never materialize. Too often the data isn’t shared with everyone or the funding comes up short. As a patient, it’s easy to feel like nobody really has a grasp on the big picture, or that people aren’t telling you the whole truth. I’m not suggesting the system is broken or corrupt, but it seems to lack a focus on patients, and on saving people who are alive today. At minimum, it needs some streamlining. More likely it needs a maximum strength enema. But I’m running out of time to place my bets. I need to find a horse soon. Quite literally speaking, I’d prefer to find one while I’m still speaking at all.