Lung health–what to do when

Mara here. When we started running into choking issues with ALS, there were many days and nights where I didn’t think I could get the love of my life’s airway clear. Get him to the other side of it.

Then a friend introduced me to an RT who made a table of “what to do when” for me. It changed everything. I’m no longer terrified when we have a choking incident. Even better, because we use the daily regimen described below the table, we have far fewer choking incidents and zero hyperventilation incidents.

I hope this table brings you as much peace AND peace of mind as it did me: Lung table–when to do what

 

Mara’s list of NurOwn responders

I’ve been making little “notes to self” every time I come across someone who describes themselves as a responder to NurOwn. By my count there are at least 14, but probably many more as these are just ones I stumbled across (it’s only been tried on something like 150 people w/ 50% placebo!)

Please contact your reps by Jan. 30

Dear friends and family,

I have an update on my last ask AND another ask (greedy! but I truly believe this could save Pat’s life).

You may recall or even helped with my last ask of contacting your congressman asking them to sign a letter from Mike Coffman, which had 3 asks.

Well 11 or so congressman signed the letter and 40+ voiced their support for it (effectively signing it). Then Mike Coffman was voted out of office. So while there’s no longer “a Mike Coffman letter” to sign, the asks remain nearly the same and besides, it’s not about the letter. It’s about educating the congressman about how behind ALS research and funding is and about finding out if the congressmen is with us or not in getting our modest requests fulfilled.

Although my ask today is similar to my last one, there are some differences. So here’s what I’d like you to do now, and I wouldn’t be asking if I didn’t think this could save Pat:

  • If you’ve already contacted your 3 congressman (House Rep and 2 Senators) or their health aides per my last ask and they’ve gotten back to you, please let me know what each said. If they didn’t get back to you or said no, please call them before Jan. 30 and:
    • Remind them of the 3 asks (below) and ask if they will support them and help you/I AM ALS make them happen. If they say yes, let me know and you’re done! If not:
      • Ask why, record their answer (to send to me).
      • Ask if the congressmen will please meet with Sandy Morris or Cathy Collet on Feb. 13 in DC to discuss these asks. Tell the congressman that Sandy and Cathy are representing you, their constituent. The congressman can set up a time to meet Feb. 13 by contacting them directly at Sandy Morris, sandymorris333@gmail.com, 530-448-1622, or Cathy Collet collet.mc@gmail.com
      • Ask them to drop by the More Than Our Stories event in DC Feb. 12.
      • If they cannot commit to Feb. 12 or 13, ask them to contact Sandy or Cathy directly to discuss (so they can better see our side and we can better see theirs).
    • If you haven’t contacted your 3 congressman, please call them before Jan. 30 and ask if they’re familiar with the three asks (below). If they don’t want to support the asks, go through the above four bullets. If they’re unfamiliar with the asks, tell them what they are, then ask if they could please meet with Sandy and Cathy per above (or if not, could they call them to discuss).
    • In any case, let me know who you’ve contacted so I can give YOUR name as constituent names to Sandy and Cathy. They will reach out and try to set up a meeting with your congressmen.

The 3 asks

  • ALS patients want access to experimental drugs until a treatment or cure is found. We don’t care if it’s through RTT, Extended Access, clinical trials, or what mechanism.
  • We want the same/similar FDA guidance document that other diseases have. We want wording like “RTT and EAP does not affect drug companies’ trials or put them in a liable position.” We want more humane clinical trials like oncology has (limited placebo usage, if on placebo then you can get the real drug right after trial, and you can get access to the treatment after the trial if it’s working for you).
  • We want a single point of contact at the FDA who can work with I AM ALS (and other ALS groups) moving forward.

How to answer “Can you do an extended stay with us?”

One of the few silver linings of having ALS is that you are often asked by friends and family if you can come for a stay so they can spoil you rotten. We highly recommend saying yes to this question, because you will indeed get spoiled rotten.

However, the answer to “Can you come stay with us?” isn’t a simple yes-or-no if you’re in a powerchair and have to be “hoyered” to bed and bath. We recently had to capture these requirements so we could stay with a friend, so we wanted to share them in case they can help other PALS (person with ALS). We hope that PALS, as we do, can fire off this list to friends or family and be that much closer to getting spoiled. Note that our powerchair is a Permobil and our hoyer is a Molift 150, but we suspect the measurements will work for nearly all powerchairs and wheelchairs and most hoyers (but as always, best to check it out for yourself!)

Powerchair and hoyer lift requirements (assuming you have a portable ramp)

  • First, measure the bed and “bathroom area,” because if those don’t work, there’s no need to measure the rest.
    • Bed—Measure the clearance under the bed the PALS will sleep on (for the hoyer lift). The height between the floor and the bottom of the bed must be 4.8” or greater.
    • Bed—Under the bed must be clear to 35” of the side of the bed that has the most floor space (for hoyer lift maneuvering). The hoyer’s two 35” “feet” must both be able to roll all the way under the bed.
    • Bedroom—You need enough room to be able to “park” the powerchair and roll the hoyer lift around it so that the hoyer’s two long feet surround the powerchair. Then you need to be able to back the hoyer lift away from the powerchair (with the PALS in it) and turn its long feet under the bed (to lower PALS into the bed).
    • Bathroom (or a place to park the shower chair)—You need quite a bit of space when the PALS needs to use the bathroom. We need a place for the shower chair to sit (preferably in a bathroom or on a non-carpeted floor but not required). The shower chair must have a decent amount of clearance around it so the hoyer lift can roll around it so the hoyer’s two feet can surround the showerchair.
    • Basically you need room to maneuver three rather large pieces of equipment.
  • Showers—If there’s no roll-in shower with enough clearance to fit an entire shower chair, you’ll need to find a nearby hotel with this if you’re staying longer than the PALS wants to sponge-bath-only showers.
  • Measure any doorways needed to get into the house/cabin, to get into an accessible bedroom, to get into a bathroom area, and to get into the gathering area (where people will hang out)or of any tight turns along the path. You need a 30” wide clearance.
  • Stairs–You’ll need the measurements of any stairs needed to get into the areas listed above (house, bedroom, etc.) as follows:
    • The height of the steps from the bottom of the bottom-most step to the top of the topmost step.
    • The horizontal measurement of the stairs, from the bottom of the first step to the top of the last step.
    • (Basically you need the ratio of full stair height to full stair width for each staircase or set of steps.)
  • Stair landings—For the most part, they won’t work, but if they’re really big, they could work. You’ll need measurements to make sure there’s enough room to move the ramp past the PALS (unless you have two ramps) and enough room for the PALS to get off the ramp so it can be placed again (unless you have two ramps).
  • Stair wall clearance–Walls around the stairs must be 30” apart the whole way (powerchair is 30” wide).
  • If all of the above measurements look good, measure any tight “turns” in the pathway (halls, doorways) to get to any of the areas listed above (house, bedroom, etc.)

First shimmer of hope—there’s a way you can help!

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):

1
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 (mara.dolan4@gmail.com) 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

2
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!