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I had a remarkable experience recently at an all-day meeting in Boston with Monica and the scientists of RSRT’s Gene Therapy Consortium that I wanted to share.
The Consortium is a collaboration of four labs that are developing a way to use gene therapy to treat or maybe even reverse Rett symptoms. I certainly wasn’t expecting to add anything to the conversation at this meeting, and truth be told I was a little nervous about being there. I’m a parent, not a scientist, and here I was going to a meeting with some of the world’s leading gene therapy experts. This was going to be a far cry from tenth-grade biology class, which was a long time ago. I went to the meeting to be a fly on the wall, learn what I could, and try to get a big-picture sense of progress. It turned out I got all this, but I also got much more.
It was amazing and even moving to see these scientists talking so enthusiastically about gene therapy as a potential way to treat or cure our daughters. It’s one thing to read about these projects; it’s quite another to be there and see ten scientists (the four principal scientists brought lab members with them) discussing and sharing their progress. I was struck by how the Consortium is a true collaboration. These scientists were sharing ideas and resources freely, and I know they returned to their labs with critical new information. Something else that surprised me was their compassion. Maybe I was expecting a sort of detached scientific approach from them. But that’s not at all what I saw. The Consortium members care deeply about their work and the impact it will have on those with Rett. They are constantly thinking about the details of gene therapy of course—the over-and under-expression of genes, DNA packaging, and vector optimization (a vector is the vehicle or “Trojan Horse” that carries a healthy gene to a mutated cell)—but it’s all driven by a desire to change lives. This was wonderful to see. We have Monica to thank for propelling these and other scientists to care about outcomes for our daughters as much as she and all of us parents do.
It was also clear at this meeting that meaningful progress was being made. I’ve learned enough about gene therapy to understand that the vectors that Consortium members are developing are critical. An effective vector will need to deliver just the right amount and parts of a gene, which is much easier said than done. At the meeting one of the Consortium scientists presented data on a vector tested in mouse models that looks promising. While this is very good progress, a lot more research lies ahead. Using gene therapy to treat Rett remains theoretical until the Gene Therapy Consortium members prove otherwise.
Science is complex and I know sometimes it’s hard to envision exactly how funding for it is used. At this meeting I had an acute sense of how every dollar contributed to RSRT matters—what I watched unfold that day simply would not have happened without the generosity of many people. It was another reminder of how grateful I am to everyone who supports RSRT. I feel lucky to have been there and to have had the chance to literally watch progress being made. The meeting renewed my excitement about the future for my daughter and all the other girls and women I’ve met with Rett Syndrome.
I am sitting in a plane on the tarmac at Chicago O’Hare, returning home from an amazing event that was spearheaded by a Rett family. The captain just announced that we are last in line for takeoff—could be an hour wait. Bummer! It’s a beautiful Friday afternoon and I was hoping to get home to my wife and daughter early to start the weekend.
But the delay gives me a chance to reflect a little on the last few months, which have been a whirlwind. Since the beginning of April, three new events have been held to raise funds for RSRT’s research—one outside of Boston, one in central Massachusetts, and one in Chicago. Two established events also were held near Baltimore and Phoenix. There were other successful efforts too—a friend of a Rett family did an online campaign for a 10K race she ran in Philadelphia; a Connecticut fourth-grader, inspired when she met a young girl with Rett Syndrome last year, raised over $1,500 for research.
It always feels like such an understatement to say how grateful I am to people who hold events or raise funds in other ways for RSRT. They are making possible research that will change so many lives, including my own daughter’s. No words can adequately express how I feel about that.
Each of these events was different. Some were hosted by parents of daughters with Rett, others were spearheaded by their friends. One was organized by Rett grandparents, another was led by a young man whose sister has Rett. They were in cities, suburbs and rural areas. They were cocktail galas or sit-down dinners. I wore a suit to one, jeans and a sports jacket to another, and shorts and a t-shirt to the race. No two events were the same. This, I’ve learned, is the beauty of events—they take on the personalities of the people who run them. They are whatever their hosts want them to be.
But there were similarities too in these events. At each one, people came together enthusiastically for the cause. Committees made up of family and friends had key roles in planning and running them. There was a shared ethos behind all of them of hope and optimism, and with good reason given the progress in research. The remarks made by parents and others were poignant, filled with the love they feel for their daughter, granddaughter, sister, or friend’s daughter; filled with a sense of empowerment and accomplishment that they are making a difference. They were tinged with sadness too of course—for what our girls have missed out on; for what their lives and our own might be like if it weren’t for a random and rare mutation on one gene.
I’ll admit something else—I’ve had fun at these events, and I think everyone involved with them has too. It has certainly been hectic and stressful sometimes, but mostly it has been fun. It feels slightly out of place to say that; after all we are raising funds to cure a disease that causes such severe disability and physical and mental pain. But it’s the truth. I’ve had a good time over the last two months. We families that have Rett in our lives need some silver lining, and this may be it. Events give us a chance to be together with family and friends, to meet other families that have the same challenges that we do. Rett Syndrome has given us a common enemy that together we can fight and ultimately beat.
This has been a rather long-winded way of trying to say one thing—Thank You. Thank you to everyone who puts so much energy into events and raising funds for RSRT and for your commitment to our daughters’ futures. Because of you, that future that we all want so badly is getting closer. We are very fortunate to count you as friends. And I am now being told to put away my laptop for the flight home.
Brenna and Mike Johnson of Tustin CA were devastated when their daughter Gisele was diagnosed with Rett last fall. But the Johnsons quickly got to work to make a difference to the research that they know will change Gisele’s life. In early December they started an online campaign for RSRT using FirstGiving.com. They called it A Cure for Christmas, and their friends, colleagues, and family came out in full force to contribute to RSRT’s research program. So far they have raised almost $60,000, a record for an online campaign for RSRT. Mike added further interest by swearing off shaving during the campaign, although their friends and family were so quick to support the cause that his beard didn’t even get very long. As Brenna says, “we were overwhelmed by everyone’s generosity and eagerness to be a part of this; and we’re proud to be helping RSRT move the science forward.” Hearty congratulations and thank you from RSRT to Brenna and Mike and all their network of supporters. Starting a fundraising campaign on FirstGiving.com is easy and effective. Contact Tim Freeman at RSRT if you have questions or need help, firstname.lastname@example.org or 609.309.5676.
Guest Blogger Beth Jones, whose daughter Jocelyn has Rett Syndrome, urges more families to take action
Yesterday, we sat in 5 hours of traffic taking Jocelyn to Los Angeles for her orthopedic appointment. Her scoliosis is turning her into a question mark, her back brace is so uncomfortably tight it disturbs her g-tube and makes feedings difficult. We strive to do the best for our daughter but we are constantly juggling details like this. After the long day, the-oh-too-familiar feelings of “defeat” and “alone” swarmed over me. A feeling I am certain every Rett Syndrome parent feels from time to time.
But today is a new day. Today I am preparing for our first committee meeting of the year for Jocelyn’s Journey. Today, I get to fight back and drink up the hope that once Jocelyn is cured, days like yesterday will be a memory, instead of my day to day reality.
This is our 5th year hosting Jocelyn’s Journey and we’ve proudly supported the Rett Syndrome Research Trust with 100% of the revenue from each event. As I reflect on our first year hosting, I was a mess, honestly. It was hard for me to admit that I needed help. I was so afraid no one would buy tickets to come to our event. I was worried what people would think of me, asking for money. I was worried my friends and family would be too busy with their own lives to join forces with me and make a committee. I have learned, in the past five years, that I couldn’t have been more wrong.
The outpouring of people who support us each year has humbled us. I’ve learned that people want to help us, they just don’t know how. Having an annual event that supports the research that will one day CURE Jocelyn inspires and thrills everyone to help. Our committee has grown from 5 people to 20 in just a few short years! We sell out of tickets each and every year for the event, donations roll in and we are becoming well known around our community. RSRT is very helpful each and every year and has guided us on what to do, as I had NEVER done anything like this before. Year one, left me hooked—I figured out how to get over the sadness, defeat, feeling alone and helpless: I fight back! Jocelyn’s Journey’s moto: “No donation is too small or too large” and we stand on that! We are grateful for every dollar and I know RSRT is too!
A few months ago, I was speaking to Tim Freeman, Program Director at RSRT, and he expressed to me “if just 10 more families would do what you are doing with fundraising, it would change the research”. I was sad that money was standing in the way of Jocelyn and all Rett girls getting treatment. But then I was excited—this was in our control! We, all Rett families, can fix this problem and expedite treatment!
The Jocelyn’s Journey committee and I have set a goal this year to be one of those 10 families by doubling our average fundraising earnings. So that leaves 9 families! There has to be someone out there who has thought of hosting an event but has been worried about how to get started. I relate to the hesitation, but be assured, you will have more support and guidance than you would expect. Call Monica or Tim, please! An event is absolutely worth it and brings in the most funding. An event can be anything you want it to be—a barbecue, a poker party, a pancake breakfast, a 5K walk or run. And there are other things you can do too. I did an “informational” booth at church to talk about Jocelyn and without asking, people handed over donations! Be creative, be fun! If all 15,000 Rett families hosted something small that raised just a few hundred to a thousand dollars, the impact would be huge. No event is too large, or too small! Please learn from my fear in the beginning: your friends and family want to help you—they just need you to give them the opportunity to help.
As Rett parents we know, the first step is the hardest one. Some of us, like myself, are still waiting to see their girl take her first step. The same holds true for fundraising—getting started is the first step and the hardest part. Once you get going though, you don’t want to stop. I won’t stop, I will not give up. I don’t expect Jocelyn to give up and I owe her the same strength in return.
I’ve been thinking a lot lately about the phenomenon of the Ice Bucket Challenge that swept the nation this past summer. This was a major coup for research on ALS, also known as Lou Gehrig’s disease. By most accounts it resulted in more than $100 million going to several organizations that support ALS research. It also created a whole new level of awareness of the disease. I was very happy to see this. ALS is an awful disease and research to find treatments or a cure is highly worthy of support. It was also great to see the country so caught up in a movement for a good cause.A number of people have asked me if I wished the whole phenomenon had focused on Rett Syndrome instead of ALS. My answer—of course I do. How could I not? I have a daughter who struggles every moment of every day with Rett. What would I not do to hear her say “daddy” or “mommy” or to watch her walk across a room? It’s not just my daughter who moves me to say this; it’s also the more than 150 other girls and women with Rett Syndrome I’ve now met. It’s their faces and their expressive and intelligent eyes that keep me awake at night. So, yes, I wish the Ice Bucket Challenge had raised $100 million for Rett research to change their lives and my daughter’s life. That kind of funding would create a sea change for our research. It would greatly speed up progress. It would buy more time from the scientists who are already working so hard on Rett Syndrome; it would allow us to expand the number of researchers focusing on Rett; it would enable more projects and more scientific collaboration. In short, it would very likely have a tremendous impact on accelerating us towards what we all want so badly—effective treatments and a cure.
I could go on wishing, but that wouldn’t be very productive. So instead I started thinking about what I learned from the Ice Bucket Challenge and how I might apply it. I asked myself why people gave to this phenomenon so readily and generously. It’s clear that they weren’t giving because of information they got on ALS or the research. Most of the Ice Bucket videos I saw contained little or no information on ALS. So why did people give? I think they gave because of who asked them. They gave because they were “challenged” by someone they knew, trusted, and respected—a friend, a colleague, their daughter or son, their parents, or an old college or high school pal. Even celebrities gave because they were “challenged” by some other celebrity they know or work with.The Ice Bucket Challenge showed me that what’s all important is who is doing the asking. It showed me something that sounds basic, but is so important—that each of us has the power to ask and that we all can act on it with our own friends and family and make a difference to the research and to our daughters’, granddaughters’, sisters’, and nieces’ lives. And now, around the holiday season as the year is ending and people are thinking about their giving, is the best time to do it. I know I’m making it sound easier than it is. Asking people for money, even for a compelling and promising cause like ours, is hard. But what I find is that the results are surprising and rewarding—not just the fact that people give, but that they give so happily and that they are so pleased to be making a difference.
You might say to me—well that’s nice; but how do I ask? What do I say? Do I do it in person or in a letter or email? Those are all good questions. But there’s no one-size-fits-all answer. My advice is to tell your story about why you care about this, and then ask if people will give to this cause that matters so much to you. You know best what would work for your friends and family—maybe it’s a heartfelt letter or email; maybe it’s knocking on a few doors; maybe it’s taking a group of friends out for coffee or a drink. There’s no right or wrong way to do it. Keep it simple, be honest, respectful, and do it from the heart.
So this holiday season, if you’re not already involved with events or asking family and friends for support of the research, try it. Try asking a few people and see what kind of response you get. I think you’ll be pleasantly surprised. I am here to help if you want to think it through or run a letter by me. Please do not hesitate to call or email me any time. Thank you so much.
A year ago today I started as program director for RSRT. I thought I would share a few reflections about the people I’ve met and what I’ve experienced and learned over that time.
Before starting at RSRT I had met two girls with Rett Syndrome—my own daughter and Monica’s daughter. Now, in my travels to events and meetings with families, I have met 47 girls and young women with Rett. Each of them, often despite terrible symptoms, has tried to engage me in some way, sometimes just through a flash of the eye or a smile. I met a teenage girl at an event who had been seizure-free for six months. But midway through the event she had a seizure. I watched as she trembled and her muscles seized; a single tear rolled down her cheek. Was it pain, frustration, fear? It was so clearly all of those. Her father and mother cried too. So did I. Of all I have learned and experienced over the last year, nothing sticks in my memory or keeps me awake at night like the faces of these girls and women and the strength I have seen in them and in their families. Our daughters have reminded me again and again without saying any words that it’s imperative that we change their lives.
I have also learned that it’s we—the families of girls and women with Rett—who are going to make this happen. We are the leaders in this cause. I’m not at all suggesting that we families have to throw money at this. I am well aware that we all have plenty on our plates; we have to live our lives, and the daily challenges of Rett Syndrome add emotional and financial stresses that most people don’t even have to think about. So we can’t be expected to shoulder the full burden of supporting Rett research on top of everything else. But, we all can make a critical difference by getting friends and contacts to support RSRT. Thanks to the efforts and outreach of some Rett families, many people who are not directly affected by Rett have generously and happily supported the research and have made it a priority for their giving. We, all of us families, have to continue and expand this outreach to our friends and contacts. Our daughters’ futures depend on it, and we all must get involved if we are to turn the possibility of a cure into a reality.
My respect for Monica and for RSRT as an organization, already great when I started, has only grown. I wanted to work for RSRT because I knew it did one thing and one thing only—supported research to find a cure. This is what I want. This is what we all want. I’ve worked in the non-profit world long enough to know that a 4% administrative cost rate is remarkable. The fact that RSRT spends 96 cents of every dollar directly on research is a reflection of its dedication, efficiency, and integrity. This is not an organization that is flashy or that spends a lot of time or money promoting its accomplishments; instead it focuses on finding a cure for Rett Syndrome. The result is, well, results. It is RSRT-supported scientists who are accumulating the knowledge needed to take the next steps in gene therapy and other promising approaches. I can talk about RSRT this way because I’m still new and in a way I’m looking from the outside in, as a parent. I have had nothing to do with this organizational culture myself; it is driven by Monica, by the RSRT trustees, and by the families that support us.
I have learned a lot about the science behind Rett Syndrome. I have much more to learn, but I know enough to say definitively that a cure is much more than a dream. It is a very realistic possibility. But it’s not going to happen unless we grow RSRT’s financial resources above and beyond the funds we raise from existing events. Money matters in scientific research. The more money RSRT has, the more resources it can put into projects like the Gene Therapy Consortium, and the faster and more efficiently these projects are likely to lead to a cure.
I know this has turned into a pitch—not for your dollars, but for your involvement. I am somewhat of a shy person by nature, so I guess I’ve also learned over the last year not to be shy about asking for help. There is so much to gain by it. We need more of you—as many Rett families as we can get—involved and supporting RSRT. Whether you are a parent, grandparent, aunt or uncle, cousin, or sibling—start an event of your own; support an existing event and get friends to join you; do a letter writing campaign. I know all of our lives are busy and full of the challenges of Rett Syndrome. If you can’t do a lot, do a little. But do something. It all makes a difference.
To all of you reading this who are involved already, this is a big thank you. None of what RSRT does would be possible without you.
I have one further thought. I know it’s hard to consider ourselves fortunate. My wife and I often find ourselves asking why us—why did our beautiful, bright-eyed daughter get such a bad roll of the dice? But when I take a step back and look at the bigger picture, I see that we are very fortunate. We’re fortunate that scientists have pinpointed the cause of Rett Syndrome; we’re fortunate that Adrian Bird demonstrated that Rett is a reversible condition; we’re fortunate that the best geneticists and neurobiologists in the world are now attracted to Rett research and are taking the next steps on Prof. Bird’s discovery; we’re fortunate that we have RSRT to lead, support, and push forward the science; and we’re fortunate that we have a cause that speaks to people so compellingly and with so much promise. Most of all, we are fortunate to have our daughters; to love them and to be loved by them in such a profound and special way; and to see brightness in their futures.
I look forward to hearing from you. Thank you.
We need your help! With promising new research projects underway such as the Gene Therapy Consortium, now more than ever RSRT needs families affected by Rett Syndrome to get involved and raise funds. Here’s what you can do:
- Start a new event. Anyone can start an event—parents, grandparents, brothers and sisters, aunts and uncles. Events can be whatever you want them to be—a gathering in a home, a picnic in a park, or a big gala. We can help you with ideas and planning.
- If you live in an area that already has an RSRT event, please get involved. Come to the events and get friends to join you, ask for sponsorships, and donate auction items.
- Do a letter-writing campaign to your friends and contacts. This is easy to do, and we can help. Most people are thrilled to support our cause. But they need to learn about it and be asked. A thoughtful letter from you can do this. We can help you draft it and even mail it out for you.
- Raise funds online. This is easy and fun to do. Go to FirstGiving and click on “Start Fundraising.” You can do this for an occasion like a birthday or anniversary, for a run or a walk, or in honor or memory of someone.
We need all hands on deck. Rett research is poised for breakthroughs, but we need help from the entire Rett community. To get started, contact Tim at 609.309.5676 or email@example.com. Thank you!
A year and a half ago my wife, Rachel, and I received the worst phone call of our lives—it was the Children’s Hospital of Pennsylvania informing us that our beautiful, bright-eyed, giggling two-and-a-half year old daughter, Eleanor, had tested positive for the MECP2 mutation that causes Rett Syndrome. We were simply devastated and didn’t know what to do or where to turn. The ensuing months were the hardest of our lives. Our dreams and hopes for our only child had been crushed. We watched helplessly as Eleanor stopped scooting on her rear end, as she had been doing, and developed a repetitive hand motion and other symptoms.
Several months later, Rachel found the RSRT website. We made a call and RSRT’s co-founder and executive director, Monica Coenraads, answered the phone. We soon learned that we were not alone in our sadness and that there was a community of wonderful, supportive parents, grandparents, family members, and friends of families who have been impacted by Rett Syndrome. And even more exciting, we learned that there was hope for a cure for Rett. In fact, it was clear that there was a good deal more than hope—Rett symptoms had been reversed in an animal model, and very promising scientific progress was being made, much of it encouraged and supported by RSRT.
Monica and I continued to correspond. I was seeking her advice and thoughts about Eleanor’s diagnosis, and I was trying to understand the research and science. Monica began seeking my advice about fundraising and public relations when she learned that I headed the development office of the Woodrow Wilson Foundation in Princeton, New Jersey, and before that had served as director of development at Columbia University’s Teachers College. It may have dawned on Monica and me at the same time that there was a fit here—that I cared deeply and personally about the work that RSRT was doing and that I had nearly 20 years of experience that could help grow RSRT and its impact on the lives of girls and women with Rett.
The rest, to use the cliché, is history. My first official day as a Program Director at RSRT was June 17. Under Monica’s direction, I’m responsible for fundraising, public relations, and strategic thinking about the organization. I couldn’t be more excited. So many people, Monica foremost among them, have worked so hard and contributed so much to making RSRT the respected force that it is and to building the cumulative scientific knowledge that will lead to a cure. I’m honored and humbled to join this team. Frankly, I never imagined that I would be able to put my knowledge and skills to use for something so important to me.
I’ve gone on longer than I intended, but I have one further thought. I’ve been thinking lately about President Kennedy’s 1961 speech to Congress in which he announce the dramatic and ambitious goal of sending an American to the Moon before the end of the decade. It took a huge team effort, conviction and confidence in a very clear mission, ample resources, and leadership, for the goal to be met. I think RSRT’s goal is not unlike President Kennedy’s in its ambition, its clarity, its importance, and its attainability. I also believe that all of us working together are the team that will get us to the moon. And, like building a rocket, a cure depends on cumulative knowledge that is the sum of its parts. The rocket needs its nose cone, its fuel tank, its electronics, its landing pads, and other components to meet the goal. Research is like this too. All the parts have to work together. It is cumulative knowledge that will get us there.
I am tremendously grateful to Monica, to the RSRT Board, and to all of you who contribute your time, energy, and resources to RSRT for your confidence in me. I promise Eleanor and all of our daughters that I will do my best in everything I do for RSRT. I will need your help, advice, and counsel—most of you know RSRT and all of its accomplishments and the Rett community at large far better than I do—so I hope I can call on you. Please don’t hesitate to contact me any time. My office line is 609-309-5676; my cell is 609-815-5102; and my email is firstname.lastname@example.org. I look forward to meeting you.