Fall 2013 Newsletter
Making the Most of Your Support - Robert McBurney, CEO
How will we get to a cure for multiple sclerosis? At ACP, we strongly believe that a cure will come from a deep understanding about the characteristics that distinguish people with MS from people with other diseases or disorders and from people who have no apparent illness.
Right now, we don’t even know whether MS is a single disorder with a single cause or a collection of disorders with similar symptoms and pathologies. One thing we do know is that people with MS often don’t benefit from one drug treatment but do benefit from another that has a different mechanism of action. That’s already a clue that MS might not be a single disorder. On the path to a cure (or cures) as our understanding of MS grows, more effective, personalized treatment strategies using the already-approved MS medicines will undoubtedly be developed.
Clinical research is the pursuit of the deep knowledge we need. Information about family and medical history, nutrition, lifestyle and exposure to many factors, including toxins, infectious agents and medications, is essential data for clinical research. When this data is combined with the results of modern molecular analyses of biological materials, great insights can be gained into the causes and biochemical mechanisms that contribute to the disorder and to the factors that determine which person with MS will benefit most from which treatment.
The founders of Accelerated Cure Project for MS recognized that a lack of high-quality biological materials and extensive information from people with MS and other individuals represented a major barrier to the best and brightest scientists undertaking the research that would drive new knowledge towards a cure.
So, in 2005, about the same time as the word “crowdsourcing” appeared ( Wikipedia: Crowdsourcing), they decided to create a repository, or bank, of extensive information and high-quality biological materials, such as DNA, RNA, plasma, serum and white blood cells, that could be provided to scientists, anywhere in the world, who had great ideas for clinical research investigations that couldn’t be done without the materials or information. The primary condition for access to the materials was that the scientists agree to return to the ACP Repository all new data and information gained from studying the Repository’s materials – for the benefit of future researchers.
Fast forward to today, thanks to the participation of over 3200 people at 10 MS clinics around the US, materials and data from the Repository have made possible almost 80 clinical research studies undertaken by groups in academia and industry, all over the world. New knowledge about MS and related disorders has definitely been gained - and published in leading scientific journals - and a number of diagnostic tests are being developed based on research using Repository materials. AND, to date, over 25 separate datasets, containing about 300 million datapoints, have been deposited in the Repository database from completed research – with over 50 more datasets to come in the next 18-24 months, and then more and more. (You get the idea.) The ACP Repository database currently contains more information about many people with MS than has ever been collected in one place. The founders’ vision has become a reality, the ACP Repository is crowdsourcing critical deep knowledge about people with MS.
How can ACP give its supporters a real sense of the value that their support generates?
Ultimately, that value will be new diagnostics, new treatments and cures for MS. While these are coming from research enabled by the Repository, is there another way to estimate the value of your support?
Just as I was thinking about this question, I was alerted to an article about one of “our” scientists, Thomas Aune from Vanderbilt University, in a Scientific American blog (see Scientific American Blog). In the article, Thomas says, “Working with them (ACP) allowed us to obtain critical samples and confirm our results for only $20,000. If I had to obtain these samples from scratch, it would have cost $1 million and added 5 years to the project.”
If Thomas’s estimate of the value of ACP’s contribution to his research efforts is also true for all the research studies that have been enabled by the Repository, then, to date, your support – as donors or Repository participants – has likely generated almost $80 million of research value and saved almost 400 years of research time!!!
Considering that about $14 million has been spent on the Repository since its inception, in financial terms alone the value created has been over 5 times the money invested. Put another way, every $10 spent on the Repository creates more than $50 of research value and speeds the pace of research.
You ain’t seen nothin', yet!
We have ambitious plans for the Repository over the next few years. As the returned data accumulates and demands analysis, we are working with an emerging local company, Exaptive, Inc., to create a way of visualizing for all scientists the richness of the Repository database. You can see a recent presentation of this visualization at Exaptive Presentation. In addition, we are working to link our MS research news site, the Multiple Sclerosis Discovery Forum ( www.msdiscovery.org), with outreach efforts for the Repository. Finally, the OPT-UP program will bring to the Repository biological materials and information about an additional 3500 participants with a focus on optimizing MS treatment strategies and understanding the nature of progressive MS disability. Read about the importance of OPT-UP to one person with MS, Joe Laferrera, later in this newsletter.
ACP continues to need your support. Our ambitious vision for the Repository can only be realized with your support. Our goal is to generate even more value from your generous support while we drive research toward improved diagnoses, optimized treatments and cures for MS. Please visit our website to donate to ACP.
Thank you so much for making our efforts possible.
In his own words...
On September 26th the OPT-UP (OPtimizing Treatment, Understanding Progression) Community Advisory Panel (CAP) was honored with the ACP Director's Award for Collaboration at our All-Star Event. Joe Laferrera accepted the award on behalf of the CAP and gave a moving speech about how OPT-UP will change the life of a person with MS. Read his heartfelt words below:
My name is Joe, and I was diagnosed with multiple sclerosis about three and a half years ago. Over the course of a few weeks, what began as mild numbness in my shin quickly progressed to the point where I was unable to walk down a flight of stairs or even step off a curb without stumbling. I fortunately have the mildest form of the disease, called "Relapsing/Remitting MS," which means the symptoms come and go. I now walk normally again, and unless you saw an MRI of my brain (which is littered with rather disconcerting white spots) you'd never guess that anything was wrong.
That will change. It will get worse. MS is chronic, progressive and incurable. My body is attacking the coating that protects the nerves in my brain and spinal cord, causing them to short out like wires without insulation. But there's no way to know how bad it will get, or how fast. MS injects a whole new level of uncertainty into life. You get used to it.
The good news is that this is a golden age for MS. There are a number of effective drugs on the market and more are in the pipeline, giving me better prospects than people who got the disease just a decade or two ago. But those drugs are not cheap. Some cost the equivalent of a new car, every year. Some cost the equivalent of a new house. Those are not exaggerations; I've been on drugs that fit both descriptions. In a little over three years, my treatments cost more than a third of a million dollars.
That figure is staggering. But unfortunately, you don't always get what you pay for. The first three drugs I took turned out to be largely ineffective. I am currently on my fourth MS drug, and there is no way to know if that one is working either. Until I get worse, that is. Then we'll try yet another drug.
Oddly enough, that is a common story with MS patients. Pharmaceutical companies spend hundreds of millions of dollars developing very sophisticated MS drugs, but there is really no way to know which treatments will work with which patients. To a large extent, it is "trial and error" medicine. That approach is not only costly from a financial perspective, it costs MS patients months or years in terms of disease progression. Choosing the wrong drugs three times in a row has given my MS a three and a half year head start. Whatever this disease has in store for me will happen much sooner because of it.
So why can't doctors do a better job matching MS drugs to MS patients? I'd love to say that it's not for lack of trying, but that's just not true. Sadly and surprisingly, although billions of dollars are spent each year to treat people with MS, there have been no broad-based long-term studies designed to determine what factors can predict whether a particular drug will work with a specific patient. Neurologists are fighting MS armed with sniper rifles, but wearing blindfolds.
Fortunately, MS doctors and other experts from across the country have taken the obvious and necessary step of designing a study to address this fundamental problem. It is called OPT-UP, and it will meticulously follow thousands of MS patents for a number of years, to obtain insight into how best to match patients with treatments. If it lives up to expectations, OPT-UP will revolutionize the treatment of multiple sclerosis.
Pharmaceutical advances have given us the tools we need to battle MS. OPT-UP will teach us how best to use them.
Volunteer Spotlight - Marion Leeds Carroll
The 2013 Music to Cure MS concert was held on Sunday October 27th in Arlington, MA. This phenomenal annual event, now in its 11th year, is produced and organized by Marion Leeds Carroll, a former opera singer and stage director. Music to Cure MS, ACP's longest running volunteer event, features vocal and instrumental performances spanning a broad range of genres including opera, classical, and musical theater.
ACP would like to recognize Marion Leeds Carroll for her over 10 years of dedication in supporting ACP.
When did you first get involved with ACP, and why?
I first heard about ACP in 2002, and was intrigued: its goals and plans made so much sense! This was the most positive, active MS-related project I had run across, and I wanted to do whatever I could to help. My MS diagnosis in 1988 made it clear to me that all the little problems and limitations which had been holding me back for decades were not going away - so it was time to find a new goal. Luckily I had studied music on a professional level, and was a stage director for opera and Gilbert & Sullivan. As I've repeatedly found since starting the annual Music to Cure MS concert, mentioning MS to performers, audience members, sponsors, or others typically results in some people telling of friends or relatives with MS, and others admitting that they have it themselves. I find myself listening to stories of MS told by business owners who are eager to support us... while performers (including Barbara Quintiliani, the internationally-renowned soprano who performs in spite of her own MS) tell me their own stories. This is a key cause! The first Music to Cure MS concert was held in October of 2003 in Arlington, MA. Our performance roster included over 20 solo and/or ensemble singers, three pianists, a 70+ voice children's choir, and a conductor. Our roster has shrunk to a more manageable size, but the concert keeps going. Every year I question whether it's worth doing again... and every year it's clear that everyone involved is looking forward to the next one.
MS is different for everyone, and it changes for each MSer as the years go by. Enjoy what you have, don't mourn for what you've lost, and keep looking for the next good thing in your life.What do you do to stay positive and motivated?
I've fought depression all my life, since long before my diagnosis. I often remind myself what I said when I was 19, "I have all eternity to find out what it's like to be dead, and only a few decades to find out what it's like to be alive." That's one thought that usually gets me over the worst spots. The second: Making sure I have positive, happy people near me, doing positive activities, to keep me upbeat. Also essential: Admitting my need for rest and solitude.Why would you recommend ACP as an organization to be involved with?
ACP doesn't claim to help individuals with MS. It sets out to support researchers as they ask hard questions, find good answers, and share their new knowledge, growing the database that ACP set up and continues to utilize. ACP is focused on solving problems that will help us all in the long run.Thank you, Marion, for the beautiful music you have brought to our lives in support of ACP.
Falmouth Road Race
10 intrepid runners took strides to cure MS by participating in the Falmouth Road Race on August 11th. Thank you to Nathan Butze, Joscelyn Chang, Kelly Holway-Chubb, Eliza Duddy, Ed Harrington, Deborah Heines, Patrick Halloway, Chen Sapir and ACP staff members Karen Crumback and Robert McBurney for raising over $11,000 for ACP.
MS Global 2013-Montana
Over the course of 7 days, 25 cyclists took to their bicycles to raise funds for ACP. These ACP supporters braved the course winding through Montana and Wyoming. During the ride the riders climbed 6,400 feet to the top of the Hyalite Canyon, enjoyed the views on Chief Joseph Scenic Highway, and appreciated and endured the challenge of Beartooth Pass. Thank you to EMD Serono for the continued support as Presenting Sponsor for the last 5 years. This phenomenal event wouldn't be possible without the hard work and dedication of event producer Bill Ha
It was an evening fit for champions when over 150 people gathered at Fenway Park's EMC Club on September 26th at ACP's inaugural All-Star Event. We gathered together to celebrate the individuals who have donated their time and efforts to support ACP's mission to accelerate research efforts, to improve diagnosis, to optimize treatments and to cure MS. ACP recognized our OPT-UP Leadership Team with the 2013 Water Cove Award for Innovation, with Dr. Rip Kinkel accepting the award. The OPT-UP Community Advisory Panel was honored with the 2013 Director's Award for Collaboration (read Joe Laferrera's acceptance speech on behalf of the CAP in this newsletter) and ACP's Young Professionals Accelerating the Cure (YPAC) received the 2013 President's Award for Impact. Attendees enjoyed a behind-the-scenes tour of Fenway, admired the two World Series trophies (there will be 3 next year!), viewed silent auction items, and learned more about ACP. Thank you to our sponsors Biogen Idec, EMD Serono, Genentech, Genzyme, Questcor, and Sam Adams.
Don your most festive gear to support raising money for MS:
Set Boston aglow! Help ACP light the Prudential Tower orange for MS: 31 Nights of Lights • Friday, December 6th, 5pm • Prudential Center, Boston
The ceremonial flipping of the switch will be followed by a celebration afterwards at a nearby location! Visit our website for more information.
For more information on event sponsorship and volunteering opportunities or to purchase tickets for upcoming events, please call 781-487-0008 or email firstname.lastname@example.org.
The Accelerated Cure Project for MS is a non-profit, 501(c)(3) tax-exempt organization whose mission is to accelerate efforts toward a cure for multiple sclerosis by rapidly advancing research that determines its causes and mechanisms.