MACPAD Reaches $1 Million Raised for PKU May 15, 2011 By Virginia Harrison, MACPAD Board member Lancaster, Pa.â€”This spring, the Mid-Atlantic Connection for PKU and Allied Disorders (MACPAD) will reach its $1 million milestone in funds raised for phenylketonuria (PKU) research and awareness activities. Since the creation of MACPADâ€™s research fund in 2000, the all-volunteer organization has funded 13 research projects that have led to the significant advancement of treatment for PKU. Its funds have helped develop Kuvan, the first prescription medicine to lower phenylalanine levels in one-third of PKU patients, and PEG-PAL, which is currently in clinical trials to treat patients who do not respond to Kuvan. â€œThis milestone is the ultimate achievement for MACPAD because it reflects the organizationâ€™s dedication to making a difference in the lives of individuals and families with PKU,â€ says Judy Griffith, a current and founding board member of MACPAD. â€œReaching $1 million in just 13 years of our existence is a reflection of the tenacity and dedication of the individuals who have made it happen.â€ In addition to research dollars raised, MACPAD has been instrumental in connecting members of the PKU community by collaborating to form the National PKU Alliance (NPKUA) and creating National PKU Awareness Month in May. Its five chapters in states across the mid-Atlantic region organize numerous community and fundraising events each year, and it publishes a free tri-yearly newsletter for more than 650 members. MACPAD has also sponsored four national conferences and provides support to families through initiatives like its Newborn Basket program. â€œWe formed MACPAD to encourage the exchange of ideas and enrich the lives of people with PKU,â€ says former President Sharon Johnstone. â€œWe have grown to accomplish so much more.â€ Today, MACPAD is the largest local PKU nonprofit organization in terms of donations received, community events held, and research funds raised. â€œI view MACPAD as the backbone of the PKU community,â€ says Dr. Ray Stevens, who discovered Kuvan and currently works on PEG-PAL. â€œOne of the biggest struggles with PKU communities is continued growth and leadership, and MACPAD has navigated this better than anyone. MACPAD is unique in their continuity, growth, impact, and national leadership." â€œThe energy and drive of the people of MACPAD gives me the strength, desire, and courage to continue PKU research. These people care, and they push me to a higher level to want to care.â€ In addition to advancing Kuvan and PEG-PAL research, MACPAD funds have helped advance research on GMP (a whey protein with very little phenylalanine that may revolutionize the foods and formulas available to patients) and methods to control blood phe levels in pregnant women with PKU whose offspring are at risk for birth defects if maternal phe levels are too high. Currently, MACPAD supports research through the National PKU Alliance for liver cell transplantation to cure PKU, GMP to control bone density problems in some PKU patients, and long-term health of PKU moms and their offspring. â€œThe funding I received from MACPAD was absolutely critical in helping me receive a $1.2 million grant from the National Institute of Health,â€ Dr. Stevens says of his work on Kuvan. â€œWe could not have done the initial experiments without the funding.â€ MACPAD was founded after the birth of Judyâ€™s son, Charley, in 1997. Looking for a support network of other PKU families, Judy, her sister Laura Assayag, and father Bob Johnstone, attended a conference held by the New England Connection for PKU and Allied Disorders (NECPAD), which called for more local organizations to be founded across the nation. What began as a casual conversation on their car ride back to Pennsylvania became a passion to create an organization supporting the mid-Atlantic PKU community. On May 8, 1998, MACPAD was officially formed with Laura the first president; Sharon the first vice president; Judy the first secretary; and Bob the first treasurer. â€œMACPAD was never about money,â€ says treasurer Bob Johnstone. â€œOur mission is and always was to provide information and support to PKU families. An important part of that support became support of PKU research.â€ In 2007, MACPAD collaborated with organizations across the country to form the NPKUA, the first national PKU nonprofit organization to even further connect the community and advance research. MACPAD volunteers provided free legal advice to form NPKUAâ€™s bylaws and membership agreements; created the organizationâ€™s first budget; and raised more than $21,500 to pay start-up expenses and nonprofit registration fees. â€œMACPAD has been extremely lucky in attracting the many people who volunteer to help us,â€ Bob says. â€œTheir commitment to serving the PKU community is unmatched anywhere else.â€ Even with all of the accomplishments, the Million Dollar Milestone is only the beginning. â€œReaching this milestone lays the foundation of our future,â€ says president Jill Ambrogio, whose daughter has PKU. â€œOur organization is only getting stronger thanks to the dedicated volunteers who bring new ideas and while incorporating othersâ€™ experience and knowledge we have from the past.â€ For more information, please e-mail email@example.com.