Once a week, I fill my pillbox. The box keeps me on track, especially since I swallow a rainbow of pills and capsules three times a day. You might think it impossible to forget whether you have swallowed 13 or 14 pills. Trust me, when it becomes a day-to-day routine, it’s easy to forget without a pillbox that stands empty. Crazy, right?
Before Mr. ALS arrived on the scene, I did not own a pillbox, and I did not fret about forgetting to take my vitamin D.
As for today’s collection, you might enjoy knowing that I have a giant capsule filled with golden oil, and a tiny rectangular tablet the color of a robin’s egg. Theracumin is a standout for two reasons. It is a uniquely thin capsule, easy to swallow, and it is a strikingly beautiful butterscotch-mustard-yellow.
A wide array of white capsules are distinguished only by size. The six giant sodium phenylbutyrates (which I take twice a day) must be taken 2 at a time, and even then, they often stick to the back of my throat, unless, of course, I remember to tip my head forward to widen the epiglottic valleculla.
Randomly, I’ll mention here that as a boy I collected marbles. I had cat’s eyes, and boulders, steelies, and solids. In all humility, my collection was the envy of Riverside Drive. Somehow this seems full-circle.
Every pill and capsule I ingest has a clearly described scientific rationale. Some of them come with a prescription from my neurologist. Others I purchase on Amazon.
A major player in my arsenal is the tag team of Tudca and Sodium phenylbutyrate. This combination is also known as AMX0035, which is making its way through the regulatory approval process at the FDA. AMX0035 is only available through a randomized controlled trial, which means some of the people are receiving the actual medication and some are receiving a placebo.
AMX0035 has shown promising evidence in the treatment of ALS: it appears to slow the progression to respiratory failure by 10 months.
Until recently I have been using a small compounding pharmacy in New Jersey for my monthly supply of sodium phenylbutyrate, all 360 capsules. We purchase it off label, which also means it is not covered by insurance.
Much to my dismay, the sole manufacturer of sodium phenylbutyrate halted production in May. I talked to them, of course, and they offered no clear explanation, nor definite time frame for when production might resume.
I ask you: what’s a person to do with Mr. ALS breathing down their back? Take the regular dose and accept a pause in a proven therapy? Take a half-dose to make it last longer and accept the possibility of it being sub-therapeutic? Try to find another pharmacy that compounds sodium phenylbutyrate and ships it out of state?
After consulting with three neurologists who are experts in the field of ALS, I decided on a half-dose.
In the meantime, if I were to sense a subtle, new symptom of ALS, should I worry the new symptom is the result of the decreased dose, or just the natural progression of ALS?
Another conundrum: as new therapeutic opportunities arise, what should be the calculus for the order of operation, because certain options preclude others.
The happy ending here is that we have found another compounding pharmacy and I am back to a full dose of sodium phenylbutyrate. The injustice and inequity of my privilege is on full display as I pay thousands of dollars each month for this opportunity.
Yesterday, I spent the entire day on zoom, in virtual meetings lobbying members of congress with a group from the ALS Association. As people living with ALS, we did our very best to elucidate the realities of our lives. The legislative aides listened carefully, mostly. They took notes and expressed dismay for our misfortune. We asked for their support regarding appropriations for the Act For ALS, and in general they assured us they would keep it under consideration.
ALS is unlike any other adult infirmity in its swift path to disability. The glacial speed of the federal government is not well suited to respond to this kind of urgency in a rare disease. Still we must persist.
I take hope in the power of the people. The ALS community is a feisty lot. We will find a way.