Does advocacy make a difference for those suffering from multiple system atrophy? You bet it does and the more people that participate the better.
When you suffer from a rare, little known disease like multiple system atrophy it is easy to feel isolated and alone. Often it can take years for a proper diagnosis and to get a name for the disease that caused so many problems. Under such circumstances it is easy to think only you, family, and friends are the only people in the world who know or care. How can so few people make a difference, you might wonder.
Well, first of all, its important to know you are not alone and there are resources available to connect with other affected by multiple system atrophy. Secondly, its important to know that even a small group of people can make a difference.
Just last month, The MSA Coalition®, a few other advocacy group leaders, two caring physicians, and about a dozen patients suffering from neurogenic orthostatic hypotension (neurogenic OH) made a big impact on an important decision by the FDA. On January 14 the FDA Cardiovascular and Renal Advisory Committee held an open public hearing to make a recommendation for or against approval of droxidopa, a new treatment for neurogenic OH. Neurogenic OH affects about 80% of all multiple system atrophy patients; but, there was only one FDA approved drug to treat it and many patients don't benefit from it for various reasons. At the end of the hearing the committee voted and recommended approval, 16-1. During the committee comments after the vote, members made it very clear that the patient testimony played an important role in their vote. Thanks to advocacy by a small group of patients the 80% of MSA patients currently suffering from neurogenic OH will soon have a new treatment choice.
As we approach the beginning of Multiple System Atrophy Awareness Month this March there will be a number of opportunities to get involved. Whether it is as simple as "liking" or "sharing" a post on Facebook, writing a letter to Congress, or hosting your own MSA Awareness event, do your best to get involved. A very small group of 18 people made a big difference on January 14. Imagine what several thousand MSA advocates could accomplish if we unite as one loud voice!
Over the next 5 weeks, from August 21 through September 24, 2013 The Multiple System Atrophy Coalition is competing in a fundraising challenge called #STARTaRYOT on CrowdRise. The top nonprofit fundraiser over the 5 weeks wins $75,000. Second place gets $50,000. Third place gets $25,000. Throughout the 5 weeks there are bonus drawings were nonprofits can get up to $10,000.
This is a tremendous opportunity for The MSA Coalition and for the MSA community. We are having our best fundraising year ever! This year alone we have already placed nearly $110,000 into our MSA Research Fund. Because of our early fundraising success this year, we are funding our first ever MSA Research Grants later this year.
Help us to keep the ball rolling! Please donate what you can during this 5 week challenge. Even better, start your own fundraiser and ask your friends, family and coworkers to support our cause. We can't lose. Even if we don't win a big prize, whatever we raise during this event goes directly into the MSA Research Fund. The money will be put to use this year to fund meaningful MSA research.
A guy dies and finds himself in Heaven standing in a line of people waiting to pass through the Pearly Gates. As he’s waiting in line, he looks around and notices off in the distance an old man with a white beard, a white lab coat, and a stethoscope around his neck pacing back and forth and looks to be in deep thought. Curious, the guy asks an angel walking past the line, “Excuse me. Who’s that old man over there in the white lab coat.” The angel looks over at the old man, smiles, and replies, “Oh, that’s God…he thinks he’s a doctor!”
Now the mission:
When I was diagnosed with multiple system atrophy (MSA), one of the most difficult pieces of information to hear was that modern medicine had nothing to offer: no cure, no treatment, no hope. How could that be? As I communicated with experts and learned about the disease, I learned the cause is unknown and complex and like other neurodegenerative diseases, protein misfolding seems to be the culprit. The question is what are the triggers and root cause of protein misfolding and currently the answer lies beyond today’s scientific knowledge.
I dream of the day when multiple system atrophy will be a disease of the past. My mission is to do what I can to help in that effort.
One thing that is definitely known is that medical discovery requires money. Moreover, since MSA is a rare disease and since rare disease does not provide the profit opportunity for the pharmaceutical industry, finding a cure for MSA is dependent on federal funding of National Institute of Health (NIH) research grants and private donations. NIH funding has historically been the major financial resource for rare disease medical research and discovery. This funding has been cut significantly in the current financial sequester; for example, NIH has had to cut funding for 700 research grants (http://www.nih.gov/news/health/jun2013/nih-03.htm).
There’s not much power in one MSA patient, but there is power in many MSA patients. Too few people, especially our elected officials, know about MSA and its affect and impact on the patient and their family.
Cutting NIH funding is not acceptable and costs lives. No one cares more about a finding cure than we do.
Below is a guest blog post from a caregiver dealing with mesothelioma – Cameron Von St. James. While the disease it very different from multiple system atrophy, caregivers of people with different diseases can learn from each other.
The Day I Became Her Caregiver
November 21, 2005. It's a day that is forever etched into my mind. It was that day in the doctor’s office that my wife Heather and I received life-altering news: Heather was diagnosed with malignant pleural mesothelioma. It was in that moment that I went from being Heather's faithful husband to taking on the additional role of being her caregiver. I wasn't prepared for my new responsibilities to say the least.
Just three month's prior to Heather's mesothelioma diagnosis, we celebrated the birth of our only child, our daughter Lily. We were eagerly looking forward to celebrations such as Lily's first Christmas and Thanksgiving. But the diagnosis turned our lives upside down.
The consequences of being thrown abruptly into the role of caregiver for a love one with a life-threatening illness began before we left the doctor’s office when Heather was diagnosed. The doctor told us about what mesothelioma is and that we needed a specialist to treat the condition. He gave us a few options. First, a local university hospital; Second, a regional hospital that lacked a developed mesothelioma program but had a great reputation; third, an expert in mesothelioma locatec in Boston. I looked to my wife, expecting her to choose one of the options laid before us. But her face was stricken with grief and horror. I knew she needed help desperately. In that moment I said to the doctor, “Get us to Boston!”
After Heather’s diagnosis, our daily routines devolved into complete chaos. Heather could no longer work her full time job. I was only able to work part time. My days were filled with making travel arrangements and traveling to and from Boston with Heather for her doctor’s appointments. I was also taking care of our daughter Lily. The never-ending list of responsibilities that was given to me began to overwhelm me. I recall being inundated with the fear that my wife would die of cancer and I would be a widow with a young daughter to take care of.
I also stressed over our financial struggles, wondering if all our money would be spent fighting a disease that would take my wife away from me. How could I care for my infant daughter if I was alone, broke and homeless? I found myself balling on the kitchen floor. The fear didn’t subside but my resolve returned when I would focus on what needed to be done. I also made sure that Heather would never see me at my weakest and darkest moments. She needed me. And I needed to be her strength.
Throughout our struggle, we had friends, family and complete strangers to carry us through. Comforting words came when we needed them the most. And so many people offered financial assistance that we were overwhelmed by gratitude. If you are ever in the position where you must fight a difficult diagnosis or be the caregiver of a loved one with a serious illness, don’t turn away the help that is offered to you. Every little bit helps. Let the generosity of others lift you out of those dark moments. Know that you are cared for and can call upon others to help carry the load.
Being a caregiver of a loved one with a serious and life-threatening condition will drain you. There is no getting around it. You will feel like your life has devolved into chaos and constant stress. Uncertainty can easily take over your mind and spirit. It will be the hardest thing you will ever go though. You can’t escape the reality of your situation, but you can’t give up. Know that the good days will come with the bad and don’t let fear rule your life. Don’t lose hope. Lean onto others for help. Use the people and resources that you have to keep your sanity.
After many years and treatments that included surgery, radiation and chemotherapy, our lives began to return to a sense of normalcy. Heather was able to defy the enormous odds against her and ignored the poor prognosis for mesothelioma. Now, over seven years later, she remains healthy and cancer free.
If you ever find yourself in the role of caregiver, you will learn many life lessons. You will learn that pride is something that you must let go of and accept the help of others. Because asking for and receiving help is not a sign of weakness. It is a sign of strength. You will learn how to manage stress that you thought would be unmanageable. My caregiver days also taught me to never stop fighting for the one you love and to know that you are capable of achieving more than you ever dreamed possible.
About the Author: Cameron Von St. James:
Cameron is husband to Heather Von St. James, survivor advocate for the Mesothelioma Cancer Alliance, and father to Lily Rose. He, along with Heather and young Lily, had their world's turned upside down when Heather was diagnosed with malignant pleural mesothelioma, just 3 1/2 months after the birth of his only child. When faced with the very real possibility of raising Lily on his own, he fought alongside Heather in her battle with mesothelioma. It was his determination and refusal to compromise on doctors or treatments that led them to Boston to receive radical surgery from esteemed mesothelioma surgeon, David Sugarbaker. Heather continues to thrive 7 years later.
Through out the next few years, while being a caregiver to Heather and dad to Lily, Cameron returned to school full time to finish his degree. He worked full time, went to school full time and graduated with high honors at the top of his class in June of 2010 with a Bachelors of Science in Information Technology. He was soon offered a position at US Bank as Network Analyst and continues to pursue his career.
Like Heather, Cameron is passionate about bringing awareness to mesothelioma and the dangers of asbestos exposure. It is his hope that sharing his story will help others those battling cancer and their caregivers who provide them care and guidance in their journey.
What it Means for North American and European Patients
There has been a great deal of chatter in the multiple system atrophy (MSA) community over the last few days regarding a recent genetic study published in the New England Journal of Medicine. The study findings are largely attributed to a group of MSA patients from Japan. In the study it was found that the COQ2 genes were mutated in MSA-C (cerebellar) patients, with the highest correlation found in inherited MSA-C patients. With the heavy emphasis on inherited MSA-C in Japanese patients a few important questions need to be answered for patients here in North America and Europe. Therefore, below we provide a question and answer session about the study with answers provided by Dr. Phillip Low of the Mayo Clinic in Rochester, MN and Prof. Gregor Wenning of the University of Innsbruck in Austria, both of whom provided DNA from groups of patients in North America and Europe, respectively to the study.
Q: How does the recent multiple system atrophy genetic research completed primarily in Japan, translate to North American and European MSA patients?
A: Asian multiple system atrophy is different to European and North American MSA in both phenotype and genetics. We don’t see inherited MSA in North America, however in Asia inherited MSA is not uncommon. In direct contrast to North America and Europe, MSA-C (Cerebellar) is more prevalent in Japanese patients than is MSA-P (Parkinsonian). There were no meaningful mutations in the North American or European groups of DNA samples. All the causative mutations are seen in the Asian study population and all the mutations are in the CoQ10 biosynthetic pathway.
Q: Is this only relevant to MSA-C patients?
A: The findings are relevant to MSA-C and likely mainly to inherited MSA-C.
Q: Which MSA patients should be evaluated for COQ2 abnormalities?
A: There is no case, based on present evidence for genetic testing for non-inherited MSA.
Q: Should the children of multiple system atrophy patients get genetic testing to look for COQ2 mutations?
A: MSA is not inherited in the great majority, especially in North America and Europe. Therefore, there is no evidence to suggest that genetic testing would be useful for the children of MSA patients.
Q: Are any studies planned to evaluate COQ10 in MSA?
A: It is uncertain whether these findings can be translated into a meaningful clinical trial with Q10, especially in North America and Europe where inherited multiple system atrophy is virtually nonexistent.
Q: Should patients be taking COQ10?
A: There is no evidence to suggest that multiple system atrophy patients will benefit from taking CoQ10.
Hello my name is Don Crouse and I serve on the Board of Directors for
The Multiple System Atrophy Coalition, which was formerly the SDS/MSA Support Group, a charity that was near and very dear to Don's heart. Multiple System Atrophy is a rare and fatal neurodegenerative disease with about 13,000 Americans diagnosed at any given time; 13,000 too many having to fight and struggle with this horrible disease. Multiple system atrophy brought Don and me together.
Don Summers was a kind, compassionate and caring man. He had a profound influence on my life and on the lives of so many others.
Don was a mentor to me. He taught me about compassion for a cause greater than oneself. He showed me that it is okay for a strong man to cry. He wore his heart and his emotions on his sleeve. Hopefully, I won't demonstrate that skill set over the next few minutes as I speak to you, but if I do, I know it is okay because of Don.
We met in 1995. Don and his first wife Gwen attended the SDS/MSA Support Group annual meeting that year as Gwen had multiple system atrophy. After Gwen passed away in 1997 Don went from being an attendee at the meeting to becoming an active leader in the MSA community. During this same time, I was a young pharmaceutical executive marketing a new medication that happened to have benefits for MSA patients. When we met, we connected right away. As our relationship grew Don encouraged me to be more than just a guy doing his job, but to truly care for and help the patient communities that I worked with. He lit a fire in my soul to give back and to do everything in my power to help the multiple system atrophy community. The power of giving back is a gift that I will always cherish and remember Don for giving to me.
In 1998, Don became President of the SDS/MSA Support Group and served in that role for more then a decade. He was a selfless giver spending untold hours every week on the phone with others trying to cope with the devastation of multiple system atrophy. Anyone who has spent time in Don’s house knows it was not uncommon for a special phone to ring. Don would answer the phone and slip into another room for an hour or more to listen to and counsel an MSA patient, caregiver or family member. It should be noted too, all of the work, effort and emotion that Sylvia contributed to the MSA community. Don and Sylvia were a powerhouse team fighting for an important cause.
When Don passed last month he was, know doubt, greeted in heaven by thousands of MSA angels, including Gwen! On the MSA Coalition Facebook page, so many have expressed their sorrow at hearing of the loss of Don, but the comments are filled with gratitude and thanks because he truly helped so many people. When you know the effort Don put into the MSA community, this outpouring is not surprising. For so many he was the empathetic, caring voice on the support line or the emotional leader at an MSA conference. He spent so many hours just listening to the troubles of others, a service that was so desperately needed by those affected with MSA. Perhaps that's why God gave him such a great set of ears. He also offered sage wisdom learned from years of caring for and loving Gwen during her battle with MSA. His ability to grow and share from what he learned during his own trials and tribulations is one of the things that made Don such an extraordinary human being.
On a personal level, Don was a great friend. We considered each other family. It was such a great honor to have Don and Sylvia drive their RV all the way from Coupland up to NJ for my wedding. Because of distance, we didn’t get together in person as often as we would have liked, but we talked on the phone often. Don ended every call with me the exact same way. Don would say “Love ya, son” and then he would always asked me to give a big hug and lots of love to my wife and two little girls. But most important he helped me to grow from a young pharmaceutical marketer into a compassionate adult with a primary mission of helping others, no matter my course in life.
In fact, Don has influenced each Director of The MSA Coalition in a positive way. With Don in our hearts we will carry on in his memory. One day, hopefully in the not too distant future, Don, Gwen and their gang of multiple system atrophy angels will be celebrating when we announce a cure has been discovered.
Don, rest in peace! You've earned it. Very few people have ever given so much of themselves to help others. I am proud to have known you, to call you a friend and a member of the family. I will miss you dearly, as will the entire multiple system atrophy community.
The Multiple System Atrophy Coalition has exciting news! Over the next few weeks the Coalition will be rolling out it's new MSA Research Grant Request program. Over the past two year's The MSA Coalition's fundraising has increased and we are now ready to begin funding meaningful research toward finding a cause and hopefully a cure for multiple system atrophy. Over the years, the former SDS/MSA Support Group encouraged research by providing travel grants each year to the American Autonomic Society (AAS) meeting for the best MSA research submitted to the AAS for presentation. The travel program will continue, however, our new grant request program is different and very exciting as it is intended to directly fund MSA research.
The MSA Research Grant Program will:
Provide an online tool to prospective MSA researchers for submitting their research proposals. The grant program will be advertised to prospective researchers to create awareness for the program.
A scientific advisory board is being formed by the MSA Coalition to review all submitted proposals to identify the most promising research
Once per year the MSA Coalition will announce grant winners
Our goal is to announce our first research grant awards via press release on October 3, 2013 as part of World MSA Day. As we finalize details of the program we will determine rules for how much money will be awarded, etc. In the next few weeks, we will issue a formal press release announcing the grant program.
Thank you to everybody who has donated to the SDS/MSA Support Group and now The MSA Coalition. Our grass roots fundraising has enabled us to reach this important milestone and continued donations will be the key to its long-term success.
We want to close out Multiple System Atrophy Awareness Month by making the unified voice of the MSA community heard in Washington, DC and elsewhere! Writing letters to your elected officials about multiple system atrophy is an important way to spread awareness and to help ensure our lawmakers keep rare disease research in mind as they pass legislation.
Your letter doesn't need to be long, fancy or perfect. In fact, we have already provided an introduction, an explanation of MSA and a closing request. All you need to do is fill in your story. Write with your heart about how MSA has impacted your life as a patient, caregiver, family member, friend or healthcare provider. Here are a few tips and ideas for your letter:
Tell them where you live so the elected officials know you are their constituent (very important)
Tell them about the difficulties you faced in the process of being diagnosed with multiple system atrophy
Tell them how multiple system atrophy has impacted your life (physically, mentally, financially, etc.)
On March 1, 2013 a Flag of the United States of America was flown over the U.S. Capital in honor of Multiple System Atrophy Awareness Month. The request to fly the flag was made by the Honorable Richard Burr, United States Senator from North Carolina.
It was a very easy process and it was a great way to kick off MSA Awareness Month. In fact, anybody can contact their US Senator via their websites and request a flag to be flown over the U.S. Capital.
Today is March 21st – 10 Days Left to Contribute Your Voice
In the United States, there are only about 13,000 diagnosed patients with multiple system atrophy. That is 13,000 too many, but it points out the need for as many MSA patients, caregivers, family and friends, healthcare providers and rare disease advocates as possible to contribute their voices to make noise about the desperate need to find a cause and a cure for MSA. Here are some ideas on how to contribute your voice to MSA Awareness Month:
Like the MSA Coalition and other MSA groups on Facebook and “like” our posts so your connections can see posts about MSA. Even better, “share” our posts directly on your own Facebook pages.
Join Twitter and create a user name with MSA in it. For example @Bob-MSA. Use the search and discussion tools to find tweets about #MultipleSystemAtrophy and retweet them to your followers.
Most important – Write letters to your members of Congress (U.S. Representatives and Senators) to let them know you are their constituent and that you suffer from multiple system atrophy. Be sure to include the following in your letter:
Your full addressso they know they represent you
Explain what multiple system atrophy is (a rare and fatal neurodegenerative disease) and provide detail on how it is impacting your life
Explain the challenges in qualifying for disability insurance
Emphasis that there is no known cause or cure and that the NIH needs more money dedicated to rare disease research
Ask them to push the FDA to use existing protocols to approve orphan drugs quickly so the pharmaceutical industry and investors will become better motivated to research medications for rare diseases.
Buy and wear MSA bracelets
Post comments on blogs like this one to help add content and to show there is an active MSA community
Start a fundraiser on First Giving to support the Multiple System Atrophy Coalition
As individuals we can make a difference, but together we can be much strong!