CAMERON AND HAYDEN LORD FOUNDATION

2008 Annual Update

Part I of our Mission: To decrease the incidence of chronic and complex diseases in children

Last year, we announced that the Lord Foundation now pools the annual research grant we’ve dedicated to our medical mission with the National Tay-Sachs and Allied Diseases Research Initiative. With the support of the CHLF and a handful of other similar foundations, NTSAD and its partners have tripled the research monies available and are now funding and following the progress of more projects than any other time in the history of the NTSAD research grants in such areas as gene therapy, stem cell therapy, and small molecule treatments. We are hopeful that each of these projects will lead to future advancements in testing and treatments.

Of particular note this year has been the progress of the Gene Therapy Consortium which has come along the furthest and fastest. This model of collaboration of researchers and doctors from four institutions in two countries - Auburn University, Boston College, Cambridge University (U.K.), and the Massachusetts General Hospital (Harvard University) - has had recent breakthroughs in the delivery of critical genes to the brain (Tay-Sachs is a neurological disease). Specifically, the collaboration has found a way to splice two gene therapy vectors into one gene—genetic material which is used to introduce specific genes into the genome of an organism. This one gene is then injected into the brain of the animals used in the research experiment. This accomplishment is significant because the therapeutic effect of the injection appears to reach all parts of the brain, even the sensory organs such as the optic nerve. Already, the researchers have begun treating the larger mammal cats with the particular gene therapy, and their goal is to begin human trials in three-to-four years. This research has far-reaching implications for many neurological and degenerative diseases, including Alzheimer’s, so that even as Tay-Sachs and the other allied diseases are considered ‘orphan diseases,’ the research to treat them holds promise for millions.

Part II of our Mission: To increase the quality of emotional, spiritual and medical care for families facing these diseases

We have funded two innovative programs for our coping mission this year, very different from each other, both of which are receiving two-year grants.

(1) As a veritable definition of this second part of our mission, we are proud to tell you that The Cameron and Hayden Lord Foundation, Boston Childrens’ Hospital and the Dana Farber Cancer Institute have joined together to fund a Pediatric Palliative Care Nursing Fellowship within the hospitals’ model Pediatric Advanced Care Team (PACT). This fellowship will help educate the next generation of nursing leaders in the highly skilled and critically important field of pediatric palliative care. Providing children who have life-limiting illnesses with the best, most compassionate care requires the specialized skills of a multi-disciplinary team and the addition of a

nurse practitioner fellowship to PACT’s training initiative creates a truly integrated educational opportunity that benefits caregivers, patients and their families. A critical piece is that the nursing fellows will then go on to serve as clinicians, educators and mentors at other institutions, thereby elevating the standard of care for countless young patients around the country. The first fellow is Ashley Atkins, who is currently a graduate student at the Yale School of Nursing and received her BA from Harvard.

(2) We have also funded a research initiative at the city hospital Boston Medical Center led by Cameron’s pediatrician Dr. Richard Goldstein. Sadly, statistics show that poor children bear the greatest burden of child death in the US, and yet the populations served by pediatric palliative care programs do not reflect this reality—poor populations are typically underserved in this area. This research grant will interview the parents of poor children who have died in an attempt to understand what their experiences were like—how they were treated, talked to and supported. Insights gained from these interviews will then be used to design a palliative care program at Boston Medical Center assuring high quality end-of-life care to poor children in an urban setting. Results of the study will be published in relevant journals, with an eye towards disseminating the findings and improving palliative care services to poor children around the country.

Finally, we are delighted to share the remarkably good news that Cameron’s Arc, the film we produced with Dr. Goldstein and the American Academy of Pediatrics (AAP), was awarded a Freddie Award from the International Health and Medical Media Awards, beating out HBO’s Alive Day Memories: Home from Iraq and CNN’s VA Tech: One Last Shot. Needless to say, we are thrilled, gratified and hopeful that this additional recognition will help promote the film as a compelling and effective tool to train residents and medical professionals in how to provide effective care to families and children with life-limiting illness. Blyth Lord and Dr. Goldstein accepted the award at the Freddie Awards in Philadelphia on November 14^th.