I don’t have any children of my own currently but I get to be Uncle Jason to all my friends’ kids so I feel like I have a ton of nieces and nephews. That is obviously how I feel about the Seckler’s kids including of course Charley. I especially look forward to the summers when they come out to the Hamptons and stay with Randy. One of my favorite things to do is throw a baseball with Charley. He is an amazing athlete and for me that’s a special way to bond. It also serves as a huge motivator to find a cure for this disease.

Knowing that Matty started Matt’s Promise in the midst of his own battle exemplifies all that one can strive to be. Watching how hard Benjy and Tracey fight for Charley and all the other kids with DMD and seeing how hard Randy works to make Matt’s promise a huge part of that is unbelievably humbling and inspirational. So yes, for me, this is personal. It’s a no-brainer. I want to do everything I can to help. I want to keep Matt’s promise!

Vis et liber inciderint scribentur. Ius erat aperiri an, ut mandamus conclusionemque mea, prima latine insolens an mea. At mei expetenda consulatu contentiones, eum id solet prompta. Epicuri electram expetenda mel cu, epicuri delicatissim

Caroline attended University of Delaware and worked in Advertising and Public Relations prior to joining Randy Reiff 9 years ago.

As the Executive Director of Matt’s Promise I want to personally thank her for her dedication, support and contribution to Matt’s Promise. There are so many pieces of the puzzle that she handles for us on a daily basis and her volunteer efforts are essential to the success we have had over the years.

Thank you Caroline for everything you do!

The cornerstone project of Matt’s Promise is research for the treatment and cure of Duchenne muscular dystrophy (DMD). While receiving treatment for his own terminal disease, Matt learned that the son of his childhood best friend was diagnosed with this dreadful and devastating disease. The news hit Matt hard, and he was determined to do all that he could to help Charley and other young people receive the best medical care and emotional support available and to fund research to find a cure.

The disease, which affects approximately 100,000 boys, is traced to a genetic mutation that results in the degeneration of the muscles. Typically, by the time a child with DMD enters his teens, he uses a wheelchair, and in most cases, by the time he is twenty, he dies from respiratory or heart failure. Matt’s Promise, in partnership with Charley’s Fund, has directed funds into the hands of researchers who have the best shot at developing a treatment or cure for Duchenne muscular dystrophy.

There is an Urgency to Act Now!

For Charley’s Fund, the urgency is real. Charley Seckler turned 11 in November 2011. He cannot wait for the medical establishment to lumber through the typical 20+ year process to bring a new drug to market. By the time Charley is in his late teens or early 20s, he will have lost the ability to walk, then to breathe. And then his heart will stop pumping. But it doesn’t have to play out that way for Charley and the thousands of other children like him. Why now? Over the past two years, scientists have made significant advances in molecular medicine and gene therapy. Major biotech and pharmaceutical companies have discovered that bringing drugs to market for orphan diseases like Duchenne can be profitable.  This is the right moment for a big, final push to extinguish this cruel disease. We can’t let it slip away or wait for a more robust economy. We must seize the opportunity now.

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is the most common fatal genetic disorder to affect children around the world.  Children with DMD cannot produce dystrophin, a protein necessary for muscle strength and function.  As a result, every skeletal muscle in the body deteriorates.  Although Duchenne is the most common fatal genetic disorder to affect children, there is no cure.

Read More >

News From Charley’s Fund

For the past seven years, with your help we have been funding medical research in the hope of finding a treatment that could save Charley’s life and the lives of thousands of children like him who have been struck by Duchenne muscular dystrophy. Now, we have arrived at a crucial turning point. Human clinical trials are finally underway. Additional trials will startin 2012 and 2013 as long as we continue to fund the development of these promising medications.

Read More >

Those are the words Dr. Brian Tseng must utter when families come to his clinic at Massachusetts General hospital seeking a treatment for thier children with Duchenne Muscular Dystrophy (DMD). This disease is the number one genetic killer of children worldwide. For the first time ever in the history of this notorious disease, groundbreaking new therapies are being developed.

Now is a crucial time to identify existing drugs that can keep DMD kids “in the game” until thse new treatments become available. Several medecines that have been FDA-approved for usin other illnesses have demonstrated exciting resuls in DMD animal testing. Pilot Trials Now is an innovative program that supports small clinical trials for FDA-approved drugs that show promise as treatments for DMD.

Trial #1
Who: Dr. Kathryn Wagner and Dr. Daniel Judge with 30 DMD boys ages 16+
What: sildenafil (Revatio)
When: July 2010 – August 2012
Where: Johns Hopkins University/Kennedy Krieger Institute
Why: Animal studies show that sildenafil can delay and possibly even prevent heart failure in DMD.
Drug and placebo provided by Pfizer.

Trial # 2
Who: Dr. Meilan Rutter with 40 DMD boys ages 5+
What: IGF-1 (Increlex)
When: Oct 2010 – Nov 2011
Where: Cincinnati Children’s Hospital Medical Center
Why: IGF-1 offers potential as a therapeutic agent for DMD, as it may improve or preserve motor function and reduce the side effects of chronic steroid use.
Drug provided by Ipsen.

Pilot Trials Now website >

Matt’s Promise has been proud to support the research of Dr. Melinda Merchant, which explores the development of new targeted therapies against sarcomas such as Ewing’s sarcoma, synovial sarcoma and desmoplastic small-round-cell tumor. Funds raised during the Matt’s Promise Memory Run went to furthering Dr. Merchant’s search for an answer and a cure.

“Despite intensive chemotherapy and surgical regimens, the prognosis can be unacceptably poor for children and young adults who are diagnosed with metastatic cancers,” Dr. Merchant said. “New therapies are being developed to target cancer cells with hopefully less toxicity to normal cells. I want to be on the cutting edge of these advances.”

Identification of specific targets and new drugs for rare tumors such as synovial sarcoma often lags behind similar efforts in common tumors. Often, big pharmaceutical companies won’t devote the funds necessary to conduct the research and clinical trials for an illness that is so uncommon.

Dr. Merchant, the pediatric oncologist who treated Mike LaViola, is interested in translational research that brings new therapies to the clinical treatment of synovial sarcoma and other high-grade sarcomas. “The ultimate goal is to get new effective therapies to the clinics so that we can better fight these aggressive cancers,” Dr. Merchant added enthusiastically. “Thank you, Matt’s Promise, for partnering with us to hopefully make this a reality.”

Dr. Merchant currently conducts research and treats patients at the National Cancer Institute, National Institutes of Health, in Bethesda, Maryland. She was a member of the pediatric sarcoma team at Memorial Sloan-Kettering, involved in clinical care, clinical research, and laboratory research, with the goal of improving the therapy for children and young adults diagnosed with rare tumors. Dr. Merchant received her M.D. and Ph.D. from the University of Miami School of Medicine. She did her residency at Children’s National Medical Center and a fellowship at the National Cancer Institute, National Institutes of Health,Bethesda,Maryland.

For the past seven years, with the help of Matt’s Promise we have been funding medical research in the hope of finding a treatment that could save Charley’s life and the lives of thousands of children like him who have been struck by Duchenne muscular dystrophy. Now, we have arrived at a crucial turning point. Human clinical trials are finally underway. Additional trials will be starting in 2012 and 2013 as long as we continue to fund the development of these promising medications.

During the past year, we initiated critical work to ensure the success of these trials. We formed a clinical trial network with a cadre of Duchenne doctors who are eager to test new medicines for their patients. We began preparing for a clinical trial to test halofuginone (HT-100), a potent antifibrotic that could prevent and possibly even reverse muscle fibrosis. We committed to support the development of exon-skipping drugs so more children can get access to these promising medications on a faster timeline than would be possible without our help. We teamed up with a group of foundations to support the reformulation of a promising new molecule that needs to achieve better bioavailability.

Seven years ago, we didn’t dare dream that within such a short time frame we would have multiple clinical trials underway for kids with Duchenne. But, thanks to you, we are here. Now here’s the catch: This is the expensive part.
Human clinical testing costs millions of dollars. That’s why we are leveraging your donations by teaming up with other foundations, pharmaceutical and biotechnology companies, the federal government, even venture capital groups to make sure our contributions have the highest possible impact.

With each passing year, as we “de-risk” potential treatments for Duchenne, the odds that we will save Charley’s life get better. We have seen the landscape change before our eyes from a hazy vision in the far-off distance to a clear path right here in front of us. We will not waver from the path until we reach the end, which—for the first time in history—is clearly in sight. What we are accomplishing together is tremendous. It’s amazing. It’s historic. It’s a miracle in the making. And you are a huge part of it.

With special thanks to the supporters of Matt’s Promise,

Tracy Kramer Seckler and Benjamin Seckler, MD

For the moment, Charley is an active, athletic child who adores baseball, soccer, and snowboarding in the backyard.  While the disease has yet to show its devastation, the stark facts about DMD are hard to believe:

DMD has a 100% fatality rate. Most DMD kids die in their late teens or early twenties. Most boys with DMD are in a wheelchair, most between 10-12 years old. DMD is associated with respiratory failure, heart failure, and debilitating orthopedic complications. Families all over the world are suffering from the effects of Duchenne, the most severe form of muscular dystrophy. Approximately one in 3,500 boys is born with DMD. An estimated 15,000-20,000 children in America are afflicted. DMD affects boys of every ethnicity and geographic location. One third of all DMD cases are the result of random in utero mutation, with no warning before the affected baby is born. Although much is known about the cause and effects of DMD, the current treatment options are extremely limited. Doctors recommend steroids and daily stretching, but these approaches are inadequate. Steroids have been shown to delay the progression of the disease. Steroids are often associated with serious side effects. Much is still unknown about how steroids work. Despite daily stretching, many DMD boys need orthopedic braces and corrective surgery to combat debilitating muscle contractures. Because there is no acceptable universal treatment for Duchenne, many families turn to alternative therapies (nutritional supplements, vitamins, acupuncture).

Parent of a child in the DMD trial

“The IGF-1 trial proved very positive for my son Timothy. He grew 1 1/3” in the six months he was on it and his stamina seemed to pick up a bit while on the drug…We are convinced the drug made a difference…Thanks for sponsoring the trial, it was a great experience and it provided something we now feel very strongly about.”

It isn’t hard to imagine that dinner conversation at the Wiederkehr-Rothberg house often turns to issues surrounding Matt’s Promise. For a long time, Justin Rothberg has been hearing about the projects funded by Matt’s Promise and its cornerstone project — fighting Duchenne muscular dystrophy.

One dinner conversation revolved around Charley Seckler, the young son of Matthew Wiederkehr’s childhood best friend. Justin learned over the last few years that Charley’s condition is worsening. He learned that there is no cure. He learned the nasty truth about Duchenne muscular dystrophy — that most, if not all, boys end up in a wheelchair by the age of twelve, and that year by year, it robs those afflicted with this dreadful disease of the ability to breathe. Needless to say, the facts jolted Justin into action.

Together with his friend Michael Budlow, Justin created Hoops for Hope, which was born out of necessity. Both boys felt that not only was it important to raise funds to delve into research for a treatment but that it was critical to raise the hopes of the sixty thousand boys who suffer from this deadly illness. On April 18, 2010, an action-packed, fun-filled three-on-three basketball and free-throw contest was held at Millburn High School to support Matt’s Promise’s core mission of helping Charley and other boys with DMD.

“What spawned the ideas was that all of our friends love sports and we have been athletic our whole lives,” Justin Rothberg said. “What better way to raise money and have a good time than to play Charley’s favorite sport — in honor of him!”

This will become an annual event that will have the potential to inform young people and propel them to act in a compassionate and earnest way. Justin and Michael have shown others that people can have an impact, no matter their age or level of experience. Everyone can make a difference. “It was definitely a success, and we will make it a tradition at our school,” Justin added.