36 Hours in DC

What an incredible trip to DC for a meeting that brought together most of the top Amyloidosis researchers from across the world, patient advocates, caregivers, FDA representatives, and, most importantly, representatives from the Amyloidosis Research Consortium.  The ARC is an organization “committed to building collaborative relationships between patients, academia, industry, foundations, federal funders, and regulators to facilitate and speed new therapies to market.”  Highlights of the meeting included—

• Meeting or seeing again “my people” especially my Amyloidosis sister Kim Tank, who I met in person for the first time
• Confessing my research fan crush on Dr. Ray Comenzo with him…and several of my Amyloiodosis friends (hilarity ensued, FYI)
• Hearing the top researchers in the world share with the FDA why the current drug development methodology in Amyloidosis is killing patients (my words, there)
• Learning of the strength and courage of others who are struggling with this disease and hearing their heartwarming…and gut wrenching…stories.
• Having people share with me how my blog has given them motivation and hope

Basically, the overall theme was HOPE!

Right now, there are no FDA approved therapies for AL Amyloidosis.  Every treatment is an “off-label” use of a drug designed for Multiple Myeloma.  Problem is, we don’t have MM.  As Dr. Morie Gertz noted at the session, “The massive gains in myeloma survival are not translating sufficiently to AL patients.  Unique interventions are needed for amyloid population.  The amyloid population is uniquely fragile.  Typical myeloma regimens have much more profound impact on AL.”  I’m assuming that impact is negative. 

Presenters, including Dr. Giampaolo Merlini from Italy and Dr. Vaishali Sanchorawala from Boston talked about the physical, mental and emotional costs of the disease.  30% of patients diagnosed with AL are dead at 6 months and for those with cardiac involvement, median survival is 18 months.  Not having approved drugs and the slow pace of drug development inherent in diseases with small populations is causing deaths that could be prevented.  The pharma companies have potential drugs but using overall survival as the success measure in clinical trials for small populations makes the approval process too long and not economically sound.  For AL Amyloidosis, it makes sense to use biomarkers that are proven to be predictors of overall survival as success measures in trials.  If the FDA would allow that, it would reduce the time to complete a trial from 6 years to 3….cut in half!

Dr. Sanchorawala shared results showing the impact the disease has on quality of life, another measure the FDA can use in determining whether a drug is shown to be effective.  Her study showed that Amyloidosis patients have a similar reduction in quality of life reported by people with Rheumatoid Arthritis.  And for some groups of patients, it’s at a similar level as chronic lung disease.  The impact to quality of life by those two diseases is pretty well understood.  For many with Amyloidosis, it is hard to measure with a clinical test the impact of the disease.  How do you quantitatively measure the impact that infiltration into the nerves of the mouth and salivary gland has?  I know a woman who’s dealt with this for 13 years.  It’s painful for her to talk, or to smile at her grandchildren, so she feels isolated.   If she were willing, why not let her try a drug that might help improve her quality of life?  But since that’s not an outcome that gets measured, she’s ineligible to participate in any trials, regardless of how willing she is because her outcome doesn't help the pharma company prove the efficacy of the drug to the FDA.

I’m HOPING that the ARC will be able to have the FDA accept biomarkers and quality of life measures as study endpoints for Amyloidosis drugs very soon….like starting next year.  It’s interesting that I was asking on Twitter about this quality of life question over the summer, thinking about the woman with the mouth involvement.  So happy to see it might happen.

It was so wonderful meeting so many of the physicians whose work I’ve read over the past two years.  First and foremost was being able to chat with Dr. Ray Comenzo, who I met at the Amyloidosis Gala in Nashville in October.  I cornered him there for about 20 minutes and told him how much I appreciated his work and how much hope he’s given me.  He was very gracious as I gushed on and talked his ear off. I read this article (“How I treat Amyloidosis”) when I was first diagnosed and the first case was so close to my disease course.  Reading about her recovery gave me so much hope.  When Dr. Comenzo came into the breakfast room, I told my friends at the table he’s my  researcher crush and that while I’ve only met him once, I know him through his writing and I feel a deep connection to him. I said his writing speaks to me more intimately than the most graphic romance novel and, of course, lots of laughter ensued. Right after that, a woman went up to him and they hugged.  I said, quietly so only my table could hear, “Don’t you go hitting on my man.  You ladies see what I’m up against with him.”

Later in the day, I shared this discussion with Dr. Comenzo and he got quite the laugh.  I thanked him for his research. We talked about the need for more treatments for Amyloidosis and I shared with him my perspective on maintaining a positive mental state.  I said that I pictured a sine wave going up and down across a horizontal axis.  Right now, I’m above the line and wasn’t going to waste a perfectly good remission worrying about relapse.  As long as new treatments are being developed, I can count on one of them being available if I relapse to put me back above the line.  My hope is to keep the wave going as long as possible.  He said, “You may have a crush on me but you inspire me.”  Swoon!

Dr. Sanchorwala came to me during one of the breaks after I’d shared that I was treated at Ohio State.  She asked me if I was treated by Dr. Efebera and when I told her I had, she shared with me how much she enjoyed Dr. E when she trained in Boston. I told Dr Sanchorwala how much I love Dr. E and how my friends and family believe she’s the perfect personality for me.  She mentioned that she recognized me from Twitter from an online Amyloidosis Journal Club that another physician has organized.  It was really cool to talk with her.

Meeting so many patients I’ve interacted with online was a blast.  First and foremost, I got to FINALLY meet my sister in this Amyloidosis sorority, Kim Tank.  Kim and I met through a Facebook group as I was recovering from my stem cell transplant and she was preparing for hers.  She’s a badass runner who completed a 100K run while finishing up her chemo and qualified for the Boston Marathon 9 months after her transplant.  She’s got great faith and great humor and is my sister from another mister.  I’ve felt close to her for over a year, even though she lives in California, and it was great to meet her.  We shared a room and had our Kappa Lambda Delta Amyloidosis Sorority slumber party and then did a whirlwind drive through DC on our way to the airport after the meeting was over.  Lots of love and fun squished into 29 hours.   

I got to reunite with Joanne Campbell who I met when I was at Mayo and visited while she was having her stem cells harvested.  I finally met Carole Harber and her husband David.  He’s a cyclist who had his bike set up on a trainer in their hotel room during his stem cell transplant about a year before mine.  Last year, he was the number one ranked road racer in his age group in the state of Oklahoma.  I saw the actor Michael York again, first met him at the gala in Nashville.  He, his wife and I had a lovely chat.  Sharing a room with more than 100 folks who are “my people” was a blast. The love, energy and support was just phenomenal.  Hearing stories from folks who have had such a tougher road with this disease than I have inspired me and filled me with gratitude. 

I also finally met in person my fellow Amyloidosis Kat that I met on Twitter.  Kat Timpf is a reporter for the National Review and a Fox News Correspondent. Her mom died from cardiac Amyloidosis last year only three weeks after being diagnosed.  We’ve shared some messages on Twitter and tend to re-tweet one another’s amyloidosis tweets.  She got up and shared her mom’s story at the meeting. You can hear an interview she gave about it here.  https://soundcloud.com/ben-kissel/katherine-timpf  It was heart-wrenching to hear her talk about how the family celebrated when the doctors determined that her mom didn’t have cancer.  They didn’t know it at the time, but she had something worse.  Let that sink in.

Josh Lacy shared his story of Amyloidosis.  He was diagnosed with AL Amyloidosis with cardiac involvement in 2012 when he was in his mid-30s.  He was married with a 4 year old daughter and 1 year old son.  You can imagine how devastating this diagnosis would be at that time.  During his talk, he made an impassioned plea so he would be alive to see all the milestones ahead of his children—high school, college, marriage and adult life.  Boy do I know that feeling.  He’s a former college baseball player and talked about how hard it is to know his son will never know his dad as the strong athlete Josh once was.  That really choked me up.   He talked about being too weak to go into school for his daughter’s first day at kindergarten but he was able to go to school in his wheelchair. He also gave me a good laugh when he described how he didn’t have the strength to lift his son when he was sick so if his son didn’t want to do something, he’d lie on the floor and look at Josh like he was saying, “What are you going to do now?”

Later in the day, Josh came up to me and introduced himself.  I told him my name and he said he knew who I was through my blog.  That his wife had read it and used it to motivate him during some of his tough times basically saying, “Look at what that lady is doing.  You can get back, too.”  (My words, not theirs.)  That was my whole reason for starting the blog…to give hope to people that it is possible to have a full and satisfying life with Amyloidosis.  I felt so honored to have served as a tiny bit of motivation for Josh and his wife Adrienne.  I had a great chat with them.  Such a great couple that joined this crappy club.  I am so inspired by their faith and strength.

I thought about my girls as Kat was telling her story.  If I had cardiac involvement, I’m firmly convinced that I wouldn’t be alive today after my delay in diagnosis.  I would have been like Kat’s mom or my friend Cara or any of those other cardiac patients who have a median survival of 18 months after diagnosis.  But, because of some variable portion of the germ line of some gene, my free light chains didn’t effect my heart.  And so I’m here today trying to use the time God has given me as effectively as possible.  

My doctor introduced me to Cara who was diagnosed about 6 months after me.  I told her I'd be her big sister in this crappy sorority we both pledged in to.  She had cardiac involvement and, even though her hematology was starting to respond from chemo, her heart was too damaged and she died about 6 months after being diagnosed.  She left behind a loving husband, three children between the ages of 9 and 14, and scores of loving family members and friends.  I regularly think about the comment one of Cara’s sisters in law said to me at her viewing, “We wanted your story to be her story.”  I vowed then to try to change the story for a Cara in the future. So that someone with cardiac Amyloidosis could have my story.  Yesterday was a HUGE step in that direction.