Category: ALS advocacy

My FDA Advisory Committee NurOwn Statement

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Dear Drs. Califf and Marks,

I am a young woman living with ALS, and I am writing in strong support of the approval of the ALS treatment debamestrocel, which has proven to be both safe and effective in clinical trials.

ALS is a brutal enemy. This equal opportunity killer destroys the nerves that allow us to move, speak, eat, swallow, and eventually breathe. It is 100% fatal and has no cure. It can strike anyone at any time.

When I was diagnosed with ALS at age 28 in 2015, I lost everything. My body, without warning or reason, turned on me, and that meant the end of so much. My career as a 5th grade English teacher and dreams of motherhood and growing old with my husband were all swept away like sand when I believed they were stone.

Let me paint you a picture of my life with ALS. My day starts with 40 minutes of breathing treatments to prevent infection in my lungs that leave me feeling like I’m suffocating. We stop when my oxygen drops to 92.

I’m on a ventilator which is supposed to provide me with full, even breaths, but if the settings are even slightly off, it either doesn’t provide me with enough air or pushes too much too fast, forcing me to hyperventilate and pass out.

I survive on a feeding tube and haven’t enjoyed real food in 7 years. We start my “food,” a beige nutrition solution that comes in cartons, after my breathing treatment. It’s delivered to me through a pump over the course of 20 hours, often leaving me nauseous.

Because I am on a ventilator, I can’t produce sound, not even a whimper when I’m in pain. I communicate with a device called a Tobii that uses eye gaze technology. I am actually typing this letter with my eyes.

Why not write to you with my hands like a healthy person? I forgot to mention that because of ALS, I’m a quadriplegic, meaning I can only move my face. I miss being able to touch. I haven’t hugged my husband Evan in 6 years. I can’t even hold his hand. I’m totally paralyzed and totally dependent on others to meet my every need.

Imagine you can’t scratch yourself when you’re itchy, can’t adjust the thermostat or even cover yourself when you’re cold, can’t clean yourself after using the toilet. That’s my life. ALS has stripped me of my independence. I’m utterly helpless without others, and I am nothing without my machines.

The ALSFRS-R is a tool to attempt to quantify how ALS is affecting a person. A 1 score increase could be the difference between choking and eating normally or requiring a walker and walking with a minor foot drop.

The ALSFRS-R is imperfect at best. One person can have a score of 37 and be walking and breathing normally but have their hands, swallow, and voice impaired, while another person with a score of 37 could have significant shortness of breath and require a power wheelchair but have full function of their voice, swallow, and hands. It is not an accurate representation of every individual’s unique case or the changes that are occurring.

My 2015 score was 34, and my 2021 score was 1. Does a score of 1 accurately reflect me? I still communicate, live, love, smile. I have rich friendships and a healthy, passionate marriage. I’m a talented writer who raises awareness of the reality of ALS by blogging (howilivewithals.com). A score of 1 is completely unfair attempt to display ME.

Debamestrocel has achieved remarkable feats in clinical trials, including maintaining and even increasing ALSFRS-R points. Every point matters in preserving an individual’s independence and dignity. Debamestrocel also improved CSF biomarkers in ALS. No other drug has accomplished this. The benefit to those in the early phases of ALS is clear in P3 trials.

Debamestrocel is a beacon of hope to members of the ALS community, and with rapid disease progression ending in death within an average of 2 – 5 years, we don’t have the luxury of time to wait for treatments.

Give those in the early stages of ALS a chance to have more years on earth and make precious memories with their loved ones. Approve debamestrocel.

Sincerely,
Rachel Doboga

You’re Invited!

Consider this your invitation to write your own statement urging the FDA to approve NurOwn / debamestrocel! The deadline is September 20th, so get moving! The organization I AM ALS has created a wonderful guide to writing a powerful statement. Check it out! And remember, you MUST include the following:

Docket No. FDA-2023-N-2608 for Cellular, Tissue, and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments

Can AI Voice Cloning Be Used For Good?

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Photo by Polina Kovaleva on Pexels.com

I’m pleased to present the link to the story for which I was recently interviewed, “Can AI Voice Cloning Be Used For Good?” by Kaleef Starks, Mark Armendariz-Gonzales, and Clera Rodrigues. The interview focused on the changes ALS has caused in my communication, as well as my experience with AI voice cloning and preservation. To read my full interview, click the book icon on the left. It’s under the heading, “Rachel’s Story.” Enjoy reading!

Rejoice, for Yesterday a Miracle Happened!

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Photo by Pixabay on Pexels.com

When I read the news, I immediately started sobbing. We were about to start trach care and Evan was holding all the materials, but as soon as he saw the first tear fall, he put it all down.

“What happened? What’s going on, honey?”

I could hear the mounting panic in his voice, but I was crying too hard to type my answer – that these were tears of joy because the FDA had made the right decision and approved Tofersen, a miracle drug for people with SOD1 ALS and familial ALS. Thank God, Goddess, and Jesus Christ I don’t have either of those. I’m very, very lucky. Still I am overjoyed! I know one family who lost 33 people to ALS. They cut through generations and you’re almost guaranteed to get it.

Evan wiped my tears so I could type. “I’m so happy! Tofersen just got accelerated approval from the FDA. It’s almost like a cure for familial ALS! Everyone has been been campaigning so hard for this on Twitter. It’s a dream come true!”

I feel bad for not including him in my passion over the past few weeks, but we have been consumed by our efforts to put together a fundraiser for our dog Pickle who desperately needs veterinary care.

I go on and on. “One man was on life support and now he’s ice skating with his daughter! And another woman’s ALS clinic score hasn’t changed in 2.5 years! Mine went down every 3 months until they stopped measuring. She cooks, does laundry, does everything she shouldn’t be able to do!”

Evan raises his eyebrows, speechless.

“The FDA approving Tofersen is a huge deal for the whole ALS community because it’s the FDA recognizing a biomarker for ALS. Who knows where that could lead?! This is the 3rd ALS treatment to be approved in 6 years. Things are really ramping up! I’m not eligible for any of them because I have sporadic ALS and I’m on a ventilator, but I’m still absolutely thrilled for the rest of my community!

“I’m proud of you, honey. You contributed to this.”

“Barely. Just a few tweets and reading a lot.”

“That’s still something. I love you.”

I’m suddenly very tired. All the crying and the roller-coaster of emotion wore me out. I have a feeling this is just the first wave of joy and tears today. Evan closes the curtains and peaceful sleep takes over my Tofersen dream come true.

The Surprising Reason I Need to See You Dump Ice Water on Your Head

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For me, the Ice Bucket Challenge is not just a way to raise money for ALS research. It’s not just about hope. The effect on me goes much deeper when I watch the videos. I know I have a vast support system that includes you, my dear readers. However, I can’t see you read my blog and articles. I get statistics on how many people read my words, and occasionally there are kind comments, but there’s a certain distance between us.

When I watch your Ice Bucket Challenge videos and you say my name, I feel seen. I feel less alone. The gift you give me when you make and post those videos is long-lasting and powerful. You kindle my heart, and I hold that light in my chest until next year when it’s time to repeat the challenge.

This year, I have only seen one video, and that weighs heavily on me. I feel forgotten, like the challenge was just a short-lived trend, not the promise of support and camaraderie that I originally believed it to be. I am holding on to hope that the last few days of August will surprise me, though.

Won’t you let your heart kindle mine?

If you need a reminder of how the Ice Bucket Challenge, follow the instructions below. Don’t forget to challenge three people in your video and tag them when you share the video on Facebook. If you are able to make a donation, you can do it at alsa.org.

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Are you wondering what the donations have accomplished so far? Check it out!

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On the Delivery of my CDC National ALS Conference Speech

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This morning, my mom and dad attended the CDC National ALS Conference to deliver a speech I wrote. I had hoped to attend the conference myself and have my husband speak for me since my voice is almost completely lost. However, my ALS threw a few wrenches, a hammer, and a screwdriver in that plan. At first, the trip seemed so manageable. However, as I learn more about the process of transporting the breathing machines and my wheelchair, and the damage that could be done to them, I have become uncomfortable with the idea of travel. It just seems a bit too risky to me. If my wheelchair is disassembled, I would be up a creek without a paddle. And there would be piranhas in the creek. And a hole in the boat. Plus, my intense spasms could make for a rough and painful flight. This is the nature of my beast.

Watching my mom give the speech, I felt no bitterness or envy. Her practice with my dad showed in her fluency. She delivered my words perfectly all of the jokes landed, and she addressed the room with conversational warmth that engaged the audience and fit my message. I was thrilled, smiling like a lunatic as I watched her do what I couldn’t in exactly the way I hoped. My dad handed out my business cards at the break while my mom texted that everyone raved about my speech and promised to email me. I have already heard from the VP of Public Policy at the ALS Association! The gratitude I feel for my parents is bigger than words.

I hope to get a recording of the speech soon to share with you. Stay tuned!

Watch My Speech at the CDC National ALS Conference!

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My speech at the National ALS Conference on outreach is tomorrow ( Wednesday ) at 9:30 AM EST! Due to travel complications, I am not able to attend, so my mom will be giving the speech for me! You can watch the event live at this link: http://www.alsregistrymtg.com

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My mom and dad register for the CDC National ALS Conference.

Save Medicaid from Being Slashed!

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The health care bill draft currently being reviewed by the Senate slashes Medicaid to the bone. If the bill is passed, 14 million of the most expensive beneficiaries – particularly, the elderly and disabled, including those suffering from ALS – will be kicked off Medicaid! Use the following text to write to or call your representatives and let them know that you expect them to oppose this health care bill (contact information here138). We must act quickly since senator Mitch McConnell intends to have the Senate vote by JUNE 30th!

The Honorable [NAME]
United States House of Representatives (or: United States Senate)
United States Capitol
Washington, DC

Dear Representative (or: Senator) [NAME]:

I am writing to ask for your help to ensure that people with Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease have the coverage and health care they need and deserve. If passed, the Better Care Reconciliation Act will seriously impact the lives of people suffering from ALS and their families. In fact, it may even be a death sentence since many will be kicked off Medicaid.

The cost of living with ALS is staggering. Depending on the level of care a person needs, expenses can reach $250,000 per year. If patients are left with the burden of paying all the costs that Medicaid currently covers, these people will go without the feeding tube surgery and supplies that prevent them from starving to death. They will lack the medicine that relieves their tremendous pain. They will not be able to afford the caregivers who help them with toileting, medication, tube feedings, bathing, and monitoring vital machines such as ventilators. Most cruel of all, thousands will face the decision to go on a ventilator, or forgo the life-saving procedure in order to avoid bankrupting their families. In short, thousands of Americans with ALS will lose their dignity and their lives an agonizing death if they are deprived of Medicaid. (Optional: insert personal story of the impact of ALS on your life).

This is a matter of life and death, not politics. This health care bill is inhumane. I know there are a number of initiatives and programs under review. However, I think, and I hope you do as well, that Medicaid, which saves the lives of countless citizens, must be preserved and well-funded.

As a resident of (STATE), I hope that I can count on your support and look forward to watching closely as the health care bill moves through the legislative process.

(Optional) If you need more information on the impact of ALS on the people in our state, please don’t hesitate to contact the ALS Association of (STATE ORGANIZATION) at: (insert chapter website).

Sincerely,

Your Name

Help Launch Phase 2 of the “Save the National ALS Registry Campaign”!

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WANTED! I am looking for people with experience canvassing for petition signatures. Phase 2 of my “Save the National ALS Registry Campaign” is to create and publish a guide that allows citizens to reach out to their neighbors and get signatures to send to representatives along with a letter explaining why we need them to oppose trump’s budget as long as it includes completely defunding the National ALS Registry.

Even if you don’t have experience, message me if you are willing to help do some research on how to support and mobilize advocates who are ready to pound the pavement!

Remember, this is NOT a partisan issue. You can love Trump and still advocate for the ALS community! #ALSaware #DefeatALS #NationalALSRegistry

Pacing Myself

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I have always struggled to acknowledge and accept my limitations to the point that I often push myself far past them. From middle school through college, I participated in at least three extracurricular activities each year. OK, that’s a lie. It was more like four or five. I was in a leadership position as often as possible, too. Of course, that all came on top of a driving need to get straight A’s (and yes, I know that is a misplaced apostrophe, but I stand by it as a legitimate way to make a letter grade plural).

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In high school, I was president of Latin Club. In this photo my dad and I are on a Latin Club field trip.

My first year teaching, I started the middle school book club, served on the information technology curriculum committee, proposed and planned an interdisciplinary curriculum fellowship and a fellowship to rework the English curriculum to include multicultural literature.

I love being busy, operating at full speed, running out of room in my planner, making multiple to-do lists and slashing through each item before falling into bed exhausted.

Now, though, at least half the days of the week, just functioning leaves me too tired to do anything productive. ALS puts such a strain on the body that simply existing is like running a marathon every day. Three years into this nightmare, I’m finally coming to terms with the fact that I can’t set the same amount of goals for myself as I used to. Doing that sets me up for depression, frustration, and failure. If I want to preserve my mental health and use my time well, I have to learn to prioritize and decide where to scale back. I’ve got to stop spreading myself too thin. Thus, without further ado, my freshly pruned list of goals:

1. Enact Phase 2 of my “Save the Registry” campaign (you didn’t really think I would stop at an article and blog post, did you?)

2. Fundraise for The Walk to Defeat ALS

3. Write one essay or section per week for my book on living with ALS

What I expect to sacrifice to accomplish all of this is the frequency of my blog posts and social media updates. Rather than posting every few days, I think it will only be manageable to post once every 1.5 weeks. I consider this a loss since connecting with readers is such a source of joy for me. However, I am hopeful that after I torpedo Trump’s attempt to defund the National ALS Registry and complete my fundraising efforts at the end of September, I will be able to write for my blog weekly. Getting my muscle spasms under control would also be a big help since I wouldn’t spend half the week sedated by Baclofen and Vicodin. The plan right now is to increase the amount quinine sulfate I take and undergo a test to see if installing a pump to push Baclofen directly into my spinal fluid would eliminate the spasms. The pump should increase the effectiveness of the Baclofen while diminishing the sedating side effects. I will definitely keep you posted on that.

For now, I will conclude by thanking you for your support of my writing, my health, and my dream of a world without ALS. I’ll write again soon(ish)!

“Extraordinary” Collaboration Brings Together Project MinE, Answer ALS and the New York Genome Center

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Here’s something to smile about! A big thank you to #TheALSAssociation for facilitating and helping fund this collaboration and countless other invaluable partnerships. In the shadow of Trump’s attempt to defund the National ALS Registry, witnessing The ALS Association’s commitment to streamlining research efforts to push us ever closer to a cure is truly heartening!

PS I know you are wondering how The ALS Association is able to fund so much research. It is possible because of the Ice Bucket Challenge, which is coming up in August. Get ready to get wet!