Charley's Fund - Since our inception, we have been incredibly fortunate to receive tremendous gifts of financial support from those who knew and loved Matt, but also those who believed in our mission and goals. This generosity has allowed us to explore some of the most innovative discoveries that are unfolding at this time. Furthermore, it moves us along our journey to fulfill Matt’s Promise of potentially saving thousands of lives through early detection, prevention or possibly finding a cure for unspeakable diseases that rob young people of their lives. These cutting-edge projects WILL help young people who face incredible pain and suffering caused by various terminal illnesses.

Charley Seckler, the son of one of Matt’s closest childhood friends, has Duchene Muscular Dystrophy (DMD). Three months prior to Matt’s death, he was devastated with the news that Charley has this incurable disease and he set out to do something about it. What has unfolded in the years since brings incredible hopes – not only to Charley’s parents, but the 20,000 other boys who have DMD. This disease is traced to a genetic mutation that results in the degeneration of the muscles. The awful prognosis for children afflicted with this disease typically includes being confined to a wheelchair by early adolescence, and in most cases, by the time he is 20, he dies from respiratory or heart failure. These children are robbed of their childhood, their treatment is painful and until now, less than promising. They may never play high school sports or have a first kiss. From the start, this became Matt’s Promise’s cornerstone project because of the devastation of the disease and the great potential for a cure. In the past 18 months, three human clinical trials have begun for promising treatments. For the first time in the history of the disease, biotechnology and pharmaceutical companies are joining us and investing in research that has the potential of bringing new therapies to market at an accelerated pace. Our foundation has joined with Charley’s Fund in supporting cutting-edge research, allowing us to help scientists purchase laboratory equipment and hire personnel needed to accelerate research in several areas, including gene modification therapy and high throughput screening – the two most promising therapies that exist today. Matt’s Promise has invested in a varied portfolio of therapeutic approaches, ranging from a brand new gene modification technique called exon skipping to searching for drugs that are already on the market for other indications. This is a groundbreaking moment in the history of DMD, and we intend to build on that momentum until a treatment or cure is brought to market. Exon skipping has now been proven safe and effective when injected into the single muscle of a boy with this disease. A systemic trial will begin at the end of 2007, in which DMD boys will have the compound injected intravenously so the therapy can travel throughout the body. Our fight for a cure is gathering momentum. Recently, we funded the work of Dr. Justin Fallon of Brown University. Dr. Fallon has discovered a protein called biglycan that can increase the amount of utrophin the body produces. Utrophin is an important protein that can act as a substitute for dystrophin, the missing protein in DMD boys. With our support, Dr. Fallon treated mice with biglycan, and the animals’ muscle function improved. He is now planning to test biglycan in boys with DMD. We are hopeful that this therapy will help Charley and other boys by compensating for their lack of dystrophin, allowing them to walk and live much longer.

Through the overwhelming support of Matt's Promise's donors and through the hugely successful gala last spring, we are proud to announce the funding of a new colorectal cancer registry led by Dr. Thomas K. Weber, Professor of Surgery & Molecular Genetics at the Albert Einstein College of Medicine and Chief of Surgery at Einstein Hospital, Montefiore Medical Center in New York City. Dr. Weber works with thousands of patients, attempting to uncover the causes of early-onset colon cancer. He leads a reputable research fellowship mentoring program and his New York Colon Cancer Challenge Run raises significant funds to promote colon cancer awareness and prevention. Patients with early-onset colon cancer where there is no family history are often perplexed and shocked to learn that they've been struck with this disease. Accordingly, Dr. Weber is determined to uncover the similarities between these young patients so he recently created the New York-Metropolitan Familial Colorectal Cancer Registry. The Registry provides the public with information on screening, surveillance, treatment and locating a health-care provider to obtain a screening test. It also provides information on current colorectal cancer research opportunities. Today there are screening guidelines in place for individuals over fifty, but if you are a person without a family history or some specific risk factor, you aren't on anyone's surveillance screen, according to Dr. Weber. His goal is to harness genomic science to help identify people at increased risk and raise the level of awareness for physicians and patients under their care. The irony is that through early-detection procedures, there is a high rate of a cure. Dr. Weber's research has the ability to cull out those who are predisposed to colon cancer. This can then lead to awareness, early screening and if the cancer does develop, the high possibility of a cure. To find out more information about the registry and Dr. Weber's work, please see page 3 of our Winter 2008 newsletter.

Matt’s Promise funded the work of Dr. Chris Sander at Memorial Sloan-Kettering Cancer Center who is on the cusp of acquiring incredible knowledge using computation biology. Dr. Sander and his research team aim to use mathematical algorithms and information systems to simulate the behavior of molecules, cells, and organisms and to make useful diagnostic and therapeutic predictions. Using the powerful knowledge of the human genome, along with creative experiments and computational methods, today’s cancer research can be compared to “zooming in on a building with high resolution clarity rather than seeing it from a 10,000 foot view flying in a plane,” according to Dr. Sander. Today’s technologies have the potential to help humankind in a profound way. Computational biology helps to predict what’s going to happen next within a cell. Somewhat similar to weather forecasting, an enormous amount of data is collected – unintelligible when observed on its own. What’s required is the application of the appropriate mathematical equations embodied in a software system. Computational biologists combine findings in biology with computer algorithms and databases to conduct biological research on powerful computers that use sophisticated software to strengthen the work coming out of traditional laboratory and clinical research. Dr. Sander’s research team is helping to interpret detailed molecular profiles of cancerous and non-cancerous cells, how cells respond to various drug therapies, and a person’s genetic profile to assist in the development of better diagnostics and prognostics, as well as improved therapies. One day, the outcome can include reduced trial and error of drug development and lead to shorter, more accurate clinical trials. When this technology is applied to cancer biology, Dr. Sander and his group want to have the ability to predict how a cancer will move from a non-aggressive to an aggressive form and, more importantly, to accurately anticipate the consequences of possible treatments. The goal is to identify the best choice of therapy that is tailored to the individual – possibly using more than one drug in combination and following carefully designed protocols.

Our Biomarker Project at the Mission Bay campus at the University of California, San Francisco (UCSF) focuses on proteins found in the body that mark the progression of cancer – from the development of a healthy cell into a precancerous and, at times, a malignant one. The scientists and physicians in the Weiderkehr Laboratory at UCSF have reached many new milestones. The team is submitting proposals to the National Institutes of Health using the preliminary data that was generated through the Matt’s Promise Foundation funding. The team has identified an enzyme on the surface of cells that are involved in certain cancers such as colon, breast, prostate and ovarian. This enzyme is known as a protease and it cuts out other proteins. A scientist on the research team found that one of the proteins that it cuts may be involved in activating other receptors on these cells that are important for their growth. The protease may be an important target to inhibit as well as being a possible biomarker. The team is preparing this work for publications after further research is completed. While this research is in an early stage, it is exciting and promising. The emerging ability to control and pattern matter and to probe and measure properties on the smallest scale is creating an incredible opportunity for advances in cancer research. The gift from Matt’s Promise to the Biomarker Project provides the vehicle to initiate efforts that have the potential to develop into a center of excellence to foster clinical applications of nanotechnology in cancer.