Thursday, November 17, 2011

Baby You're A Rich Man


How does it feel to be
 
One of the beautiful people?
 
Now that you know who you are
 
What do you want to be?
 
And have you travelled very far?
 
Far as the eye can see. 

Baby you're a rich man, 
Baby you're a rich man,
 
Baby you're a rich man too.

This time of the year offers an odd mix of Christmas joy and New Year's anticipation. You'll find the rare station still playing classic Christmas carols, trying to retain the warmth of the season, while at the same time racks covered with paparazzi magazines listing the best things of 2011.  You'll find lists with the best news stories, best movies, best celebrities, best looking, the best scandals, and the best scoundrels. One list that never misses attention is the richest people.  

We list those who have the most family wealth at the end of 2011, those who were paid the most money in 2011, and even those that lost the most money in 2011. Some of these stories are meant to make us feel good and others are meant to make us upset.  In these times of economic struggle we must find it satisfying to stand on the side of the good rich (aka Steve Jobs) and against the bad rich (aka anyone who works on Wall Street).  I'm not sure this makes anyone feel any better on January 2nd, but such is life.

One thing missing from the list is a definition of the word "rich". Merriam-Webster defines it as "having abundant possessions and especially material wealth".  This is easy to understand.  Dollars and cents are simple to count and compare.  When we think rich we think Bill Gates, Warren Buffett, or some movie star relaxing on a beach in the French Riviera.  I propose this is only a partial definition of the word.  Perhaps there is more than meets the eye.

A few weeks ago we were invited to Amicus Therapeutics to speak about our experiences with Pompe disease.  Amicus (http://www.amicustherapeutics.com) is a biopharmaceutical company, lead by John Crowley, which is working on pharmacological chaperones for the treatment of genetic diseases.  The science is much more sophisticated than I can detail, but the idea is chaperones will bind to a protein to increase its stability and increase its activity.  For Pompe patients, these investigational drugs may one day be used in collaboration with Enzyme Replacement Therapy to heighten ERT's effectiveness in the body.  In other words, if the product is successful, our girls will receive the same ERT via IV therapy, but hopefully each drop will work a bit better than today.        

We've done these types of events for the MDA, CHOP, and others before, so we quickly agreed.  Any opportunity to tell our story and meet others living our life or working to improve it is time well spent.  So, we loaded up the car and drove to Amicus's offices in Cranbury, New Jersey.  We received a warm welcome by members of the staff and were set up in a big conference room just in time of lunch.  Before we knew it, the food and the employees starting rolling in.  You never know how many people might attend these sessions, but we were lucky enough to have seventy employees. Working for corporate America myself, I know how tough it can be to get a few people away from their emails and meetings, so we were honored.

I started off the meeting with a few slides highlighting our story, our learnings, and our hopes.  As soon as I began to talk I couldn't help but move around the room, test out a few one-liners, and make sure my message was as clear as possible.  I was in "work mode", trying to engage the group and make sure their time was well spent. However, as I continued I didn't see people checking watches and Blackberries as is so common in today's fast paced business environment.  Rather, I began to see emotion.  In the midst of a never-ending focus on quarterly financials and clinical trial schedules, they were remembering the reason why they chose this profession.  They are heroes to people who look to science to bring solutions to their loved ones facing disease.

After I finished my presentation, I invited Emma, Maddie, and Carter to take questions. To ease any concern about asking prying health questions to teenagers, Emma stepped up, spoke about her love of public speaking, and her interest in answering anything and everything about life with Pompe.  The crowd seemed to relax and the questions began.  

"Where do you receive your treatment?" 
"Do you find yourself getting weaker or stronger?"
"How do you cope at school?"
"What do you tell your friends about Pompe and what do they think?"
"What is it like to be a brother with two sisters affected by Pompe?"
"How do you plan to transition to college?"

Donna and I chimed in here and there when necessary or when the questions were directed to us, but we tried to let the kids do their thing.  For most of the time, I just sat back and grinned with pride.  

Toward the end of the session, a man raised his hand so he could ask a question. Before he did, he stood up and told us that he too had three kids.  One was a teenager, one was a bit younger, and one was soon to be born.  His voice began to quiver as he said his wish was that one day his three children would be as strong and as close as those he saw before him.  The power of his statement hit me as I looked at our three kids.  Two years ago their world was rocked and now they are standing confidently together fielding questions and displaying their commitment to each other. "Wow!”  

We finished off the meeting with a bit of dessert, a chat with some of the attendees, and a promise to stay in touch.  Just like other events before, I hope we left behind just as much value as we gained.  As I look back, I realize this day offered much more than I expected.  It's been a crazy couple months as I've scurried around trying to finish this task and that task at work.  The busy time has kept me away from some of the things I love such as this blog and made me focus too much on the short term issues of finance and quarter end results.  Looking back, I remember that while all those things are important, they are fleeting.  Just like the definition of "rich", the definition of "life" is so much deeper and so much more fulfilling.  

So, as this year comes to a close you can spend some time searching if you wish, but I'll save you the time.  You won't find me on any of the Best of 2011 lists.  I haven't starred in a top 10 film, been rated as the most beautiful, or been named the richest guy in America.  However, as midnight approaches and 2012 is rung in, I won't be worried by any of it.  Instead, I will be spending the time with my loved ones, counting my blessings, and thinking of the day we visited Amicus and heard the kind words from another father, who saw our children proudly standing together, and remind myself once again that...

Baby you're a rich man,
Baby you're a rich man,
Baby you're a rich man too.

Happy New Year!
Matt

Credit to: The Beatles, "Baby You're A Rich Man"

Wednesday, November 9, 2011

And Your Bird Can Sing

You tell me that you've got everything you want
And your bird can sing
But you don't get me, you don't get me


When I was a teenager I thought my future was pre-determined. I didn't have the athletic ability to become a professional athlete nor the interest in becoming a lawyer. I was going to be a musician, writing songs, playing guitar, and singing in a band. I took the typical first step toward my goal, buying the first guitar in a pawn shop and taking lessons. Once a week I would head down to La Habra Music for lessons and each night I would slide on the turntable and play over the loud music coming out of my cheap speakers. 

With the help of my teacher I was able to learn how to play chords, a couple simple riffs, and eventually my first song called "And Your Bird Can Sing" by some band named The Beatles. I must have played that song a hundred times, each time imagining the excitement of being on stage. Thankfully my parents were gracious enough to let me go at it over and over until I had it down. I'm sure it didn't sound anything like George Harrison, but I didn't care. I knew a song! 

Time went by and that guitar was slowly replaced by college books and a girl I would someday call my wife. As the years rolled by we moved place to place from crappy apartments, to starter homes, and eventually to a nice place we are proud of. Each time we moved that guitar came along even if it found itself stashed away in a garage or basement. Here and there I would search it out and begin to play again just to see if I remembered that old riff and maybe that old song. 

As our kids aged I noticed that we did have a musician in the house, but it wasn't me. It was Maddie. It started with clarinet in elementary school and then transitioned to violin. When she was in third grade she was playing with the fifth and sixth graders. By fifth grade she was first chair, tuning the orchestra, and leading in solos. Over time Maddie added a bit of piano and eventually decided to open her heart to singing.

The first time I really heard her sing a solo in public was at last year's school musical when she played Alice Applebee. I sat in the audience and heard a voice shooting out from a little girl with such joy and strength.  She did a great job and her confidence boomed.  After the play ran its course, she was hooked. She soon started singing lessons once a week and began to prepare for the next musical. 

A wonderful thing she did not consider was singing's importance to her health.  Just like the benefits she'd seen from those nights in the physical therapy pool, singing was helping her. You see, in addition to muscle weakness in arms and legs, adult onset Pompe carries the risk of respiratory complications caused by weakening of the diaphragm and other respiratory muscles. If allowed to progress without treatment by ERT, diet, and exercise, Pompe can lead to breathing issues including the use of bipaps at night and potentially respiratory failure.

People say a muscle at rest usually stays at rest, while a muscle in motion usually stays in motion.  I don't know if Maddie will ever face these challenges with Pompe, but if the chance is there, why not keep those muscles in motion?  After all, what's wrong with the sound of music throughout the house especially if it brings joy to a little girl's heart?

That guitar now hangs in my home office waiting for the opportunity to be used once again.  Maddie has been getting ready for her next audition by practicing lines and singing songs.  Maybe it's time to pick it up, get it back in tune, and play it beside my little bird, who clearly has more musical talent than I ever will.  As I listen to her I wonder if each time she opens those lungs and fills the house with her song, she is looking Pompe disease right in the eye and singing...

But you don't get me, you don't get me!

Encore!
Matt


Credit to: The Beatles, "And Your Bird Can Sing"

Sunday, November 6, 2011

Hello Goodbye

You say yes, I say no

You say stop and I say go, go, go
Oh, no
You say goodbye and I say hello
Hello, hello

I don't know why you say goodbye
I say hello
Hello, hello

Last July, I wrote the post Two of Us where I first shared the story of Mike Stanzione. As a reminder, Mike is a Pompe patient who has faced many of the same struggles other adult Pompe patients do, but with a twist.  While Mike had the same challenges with muscle weakness, respiratory deficiencies, etc., he wasn't able to manage them in the comfort of his home.  Rather, due to his 24 hour dependency on a ventilator and his health insurance, he was required to stay in a hospital.  The twist is Mike's hospital stay lasted over four years with no end in sight.

When I met Mike last summer I was inspired.  I was inspired because despite the steadfast position from his insurance company who said he could not go home, he continued to believe that his stay was temporary. I was inspired by his commitment to wife and son and his family's commitment to him, never giving up, visiting and supplying posters of his young son's achievements.  Most of all, I was inspired by the man's spirit.  After four years in the hospital, he had every right to pack it in and give up, but he refused to say goodbye. 

The day I visited him he had just finished a call from a member of Dr. Barry Byrne's staff at the University of Florida. He was in conversations about a revolutionary procedure where he would be the first person with Pompe to get a diaphragmatic pacemaker. If the procedure worked, it would allow him to breathe without his ventilator for part of the day.  This was the key to returning to the only place he wanted to be...home.

Mike's residence was the long-term care area of Bergen Regional Medical Center in New Jersey.  This was not a hospital wing filled with the joyful sound of newborn babies. This was a serious place where people with long-term health challenges came and stayed.  I don't know what it is like to live month after month and year after year in a long term care center, but I'm sure it is not easy.  During my visit I saw people who seemed full of life and others who seemed resolved to live out their days in quiet. I'm not sure what makes the two of them different, but my belief is hope. 

Mike had that hope - the hope that one day he would be reunited with his family. Rather than listen to the officials telling him to say goodbye, he chose instead to reach out over the internet for a solution.  Through Facebook he found a worldwide network of Pompe patients waiting to help him.  He found new people with new ideas and new solutions.  This eventually led him to Dr. Byrne and the amazing people at University of Florida's Shands Hospital. 

Through the efforts of many, Mike made it down to Florida for that revolutionary procedure.  He recovered well and headed back to New Jersey where a great thing happened.  Over time, it seemed to work.  One day he was able to be off the ventilator for an hour.  A few days later it was two and a half hours.  Then it started to roll.  Four hours became six, six hours became eight, and eight became twelve. His 24-hour dependency was broken and he was finally coming home!

You can read the rest of the story in the web link below: 
If the link does not work, please copy and paste the following into your web browser: (http://newyork.cbslocal.com/2011/10/18/nj-man-suffering-from-pompe-disease-returns-home-in-medical-miracle/)

I have a feeling that when all the hoopla resides, the welcome home signs are put away, and day to day life returnes, Mike will look back.  He will remember those days in the hospital when others told him he would never go home.  He will remember the battles he won and the support from his family and friends.  Best of all, he will be remember the reason behind it all each time his son walks in, throws his arms around his dad, and says... 

I don't know why they said goodbye
I say hello!

Congrats Mike!

Matt

PS: To all my friends at University of Florida, Great job!  It's a good day to be a Gator! 

Credit to: The Beatles, "Hello Goodbye"