Mara here, saying hello to our tribe and others just joining the amazing ALS community we’ve come to love.
Today for the first time since the diagnosis, I have hope that we may actually put the brakes on Pat’s progression (not just beat it back a little only to have it return the next day). This optimism is thanks to I AM ALS, an organization intent on breaking down the same barriers to treatment that Pat and I have wanted to break down.
These obstacles, all man-made, sit between my husband, Pat, and several promising treatments in late trial phases. The obstacles can be overcome by 1) the FDA taking actions related to the recent federal Right to Try (RTT) law and 2) putting funds in key places to speed up the research.
While I want to laser focus on #1 and #2 above, what I’m asking you to do in this blog post relates only to #1. (As an aside, I’m also passionate about making trials more humane, which is another I AM ALS initiative. So I’ve included that ask in this blog post as well.)
So here’s what you can do (and please do this as soon as you can, as every ALS patient has a ticking clock on their head):
Write to the contact below and include the three bullets (three things we want the FDA to do) from the letter from Mike Coffman (CO, 6th), Ask FDA to Finalize Guidance for ALS and RTT (PDF) (or just attach the whole letter). The three things are: finalize the guideline for research, publish a guideline that better opens up access to ALS drugs/treatments in trials, and appoint an ALS point person in the FDA.
Scott Gottlieb, M.D.
Commissioner, Food and Drug Administration (FDA)
U.S. Department of Health and Human Services
10903 New Hampshire Avenue
Silver Spring, MD 20993
Example letter (feel free to use in part or in whole or use your own words):
Dear Dr. Gottlieb,
My [friend/family], Pat Dolan, along with 1 in 400 battling ALS, is in a fight for his life, and you can save him by connecting him with the promising treatments (in trial) today. I beg you to listen closely to PALS (patients with ALS) when you/the FDA meet with them Feb. 11. I’m sure you/the FDA, together with Congress, could come up with creative solutions to remove the barriers (man-made) to these treatments TODAY. I beg you to put time and resources to fast track us, but short of that, I ask that you support the three “asks” outlined in the attached letter from Mike Coffman (CO, 6th), Ask FDA to Finalize Guidance for ALS and RTT (PDF).
The three asks are: 1) quickly finalize the guideline for research 2) publish a guideline that better opens up access to ALS drugs/treatments in trials and 3) appoint an ALS point person in the FDA.
Please contact Pat’s wife, Mara (firstname.lastname@example.org) for details on how the FDA can help or how this disease drastically negatively impacts families, communities, insurance companies, and health care (why ALS deserves all FDA gun barrels pointed at it until it is stopped). But in summary, ALS (Lou Gehrig’s) kills 50% of those diagnosed within 15 months, basically by eating them alive. ALS has been studied for 149 years and we still are far from knowing what causes it; we don’t have any REAL treatments for it. All we have are a handful of promising trials, but even those are out of PALS’ reach, in large part due to FDA guidelines not accounting for the ferocity and complexity of ALS, as explained in the attached.
Pat’s case is typical. Like all PALS, every day Pat fights to preserve function, only to lose ground the next day. Seeing promising treatments in trials, Pat last summer applied for three of the most promising trials (NurOwn, FORTITUDE, and a steroid) near where he lives. He met the published eligibility criteria for all three (strict as they were). After fighting to get screened for those trials before the 2-years-from-diagnosis date arrived (a date which dooms all ALS patients to ineligibility), then going through the screening process for each, Pat was told he was ineligible for all three. Why? Well each reason given was flimsy: 1) His progression is “just shy” of being fast enough (even though he was running daily in February 2016 and now no longer has use of his arms, hands, legs, or torso) 2) He has a port (he didn’t want to remove his port for a short-term trial, a decision that disqualified him, even though he would not use the port during the trial) 3) He had been taking Radicava and would have to go off of it for several months, which would’ve put him past the 2-year-since-diagnosis mark (Radicava is the only drug believed to do anything remotely worthwhile for ALS, which is to slow progression by a third in some patients).
The kicker is that even if Pat would’ve gotten into one of these trials, he would spend what little time and energy left in his life dealing with invasive procedures, endless trips to the clinic for status reports and all for nothing if he was in the placebo group (50% chance!)
Another kicker… if Pat were lucky enough to get into a trial, not get the placebo, and the trial treatment worked, there is no way, even with his own money, that he could continue on the treatment that could save his life. Not through Right to Try, not through the Early Adopter Program, not through the trial.
Even though the promising NurOwn treatment is being administered to trial participants (well, 50% of them!) next door to Pat, he’s unable to get it. To get the treatment, he must do what several PALS are doing: move to Korea. Can you imagine a worse decision–stay in America to be eaten alive while your loved ones helplessly watch… or pick up and move to Korea. During the most desperate, chaotic, vulnerable time of your life, a time when you have a mountain of day-to-day requirements, move to a foreign country where you have no support system and you don’t speak the same language as your doctors, or for that matter, your neighbors. Good luck surviving the long trip to Korea in the first place with most stages of ALS. Also good luck getting the money for this fun relocation, as most ALS patients require two caregivers and lots of equipment at a minimum, almost none of which is covered by insurance.
Getting NurOwn and other promising treatments into PALS as soon as possible is the only humane thing for the FDA, trial clinics, pharmacies, and Americans to work together to GET DONE NOW. With collaboration and creativity, it is within our power to stop ALS in its tracks, possibly before its ridiculous milestone of year 150 in 2019, and possibly in time to save Pat.
[Be sure to include a bit about the disease impact from your perspective–adding the human element helps… bonus if you can include a picture of Pat. There are some at https://www.flickr.com/gp/shotspot/whsb72%5D
Send an email and/or snail mail to your congressman/legislative aide and attach the Ask FDA to Finalize Guidance for ALS and RTT letter. Then the most important part: follow up two weeks later via phone call or even a visit. You may get access to the actual house rep but most likely it will be one of their legislative aides. Stay in contact with the aide, especially if the rep isn’t signing—find out why they’re not signing and let me know their answer (or let I AM ALS know).
Here’s suggested wording for the email/letter, but of course use as you see fit. Include a picture of Pat and his story, in your words.
Note! Nine House members have already joined our cause, so if you contact them, thank yous only to them! The nine are: Mike Coffman (CO, 6th), Andy Biggs (AZ, 5th) Ken Buck from Colorado (4th), Doug Landborn from CO (5th), Brian Fitzpatrick (PA, 1st), Mike Quigly (IL 5th), Barbara Comstock (VA, 10th) Tom McClintock (CA 4th), Walter Jones (NC, 3rd).
Dear [name of your congressman preceded with “Honorable” and/or name of their legislative aide].
My [friend/family], Pat Dolan, along with 1 in 400 battling ALS, is in a fight for his life, and you have the power to save him by connecting him with the promising treatments (in trial) today.
Currently there are several drugs and treatments in late-phase trials that have shown great promise in helping people with ALS (Lou Gehrig’s disease) battle this terrible disease. If you’re not familiar with the disease, imaging watching someone you love being eaten alive over the course of 6 to 36 months and there’s nothing you can do about it—no one knows what causes the disease, even though it will be 150 years old next year.
Terminal patients would be able to get these treatments if it weren’t for a few man-made obstacles. Please join the initiative to remove these obstacles by asking the FDA to:
- Finalize its guideline for research
- Publish a guideline that better opens up access to drugs/treatments in trials
- Appoint an ALS point person in the FDA
Details for these three requests are provided in the attached letter, Ask FDA to Finalize Guidance for ALS and RTT (PDF), which you may have seen come across your desk from Mike Coffman as well.
These three asks, while significant, would be even better served if you (Congress) could brainstorm with I AM ALS and other groups intent on fast-tracking research to figure out how to get to a real treatment TODAY. It’s time to get creative while pointing all gun barrels at ALS so it doesn’t reach year 150 with no treatment and next to nothing known about it.
[Insert Pat’s story above here, or parts of it. Attach a picture with Patrick Dolan.]
[At the end of the letter, give a little bit about how the disease has impacted you and others you’ve observed—make it clear how devastating and terrible this disease is. Include a picture of Pat (some are at https://www.flickr.com/gp/shotspot/whsb72). Then don’t forget to sign it and include your contact info. In two weeks call your rep (get rep’s aide’s name that you’re working with) to make sure it was received. Ask for a status (if not signed, ask why). Keep in touch with your contact–don’tlet up!