Mission

Saving the lives and dignity of young people with life-threatening disease

Neuroblastoma

Neuroblastoma is a pediatric cancer that affects the nervous system. It is one of the most aggressive and treatment-resistant childhood cancers, which manifests in ways defying easy diagnosis. After brain tumors, neuroblastoma is the most common pediatric solid tumor, with 500 - 600 new diagnoses in the United States every year. Treatment normally involves several rounds of high-dose chemotherapy, surgery, local and total-body irradiation, and one or more stem-cell or bone-marrow transplants.

Frequently, one or more experimental treatments may be administered, including targeted therapies which, unlike chemotherapy, seek and destroy only cancerous cells. Areas of research have included screening to determine which newborns may have the disease; new kinds and combinations of chemotherapy and targeted therapies; immune system enhancers; and, most recently, bioinformatics to understand possible mechanisms of genetic factors.

Following an initiative of the Foundation’s president, Michael DeLaurentis, working with Representative Lawrence Curry, the Pennsylvania House of Representatives has since 2006 designated April Neuroblastoma Month in Pennsylvania to honor the memory of the thousands of children who have lost their lives to the disease and to foster greater awareness of the disease.

For more about neuroblastoma, visit here.

Cystic Fibrosis

Cystic fibrosis (CF) is the most common fatal genetic disease in North America and among Caucasians worldwide. CF is primarily a disease of the lungs, although most CF patients also experience digestive malabsorption. Individuals with CF require two or more chest percussive therapy treatments a day, which may be performed manually or by a mechanical inflated vest. Patients also inhale medicated mists, similar to those taken by asthmatics. The daily regimen includes vitamin supplements and pancreatic enzymes to enhance digestion. About one in 20 newborns carries the defective CF gene. Today, more than half of newborns with CF can expect to live to age 37 or beyond.

Thirty thousand Americans have CF. One in 3,000 newborns is diagnosed with CF, resulting in approximately 1,000 new diagnoses in the United States each year. CF is genetically uncomplicated: if both parents carry a defective CF gene, a child who inherits the defective gene from both parents will have the disease. On average, one in four children born of parents who carry the defective gene will inherit the disease; one in four will not be a carrier; and two in four will be carriers but not have the disease.

The cellular defect is an incapacity to transfer sodium and chloride properly into and out of the cell, resulting in thick, sticky mucus which results in persistent coughing and failure to gain weight. The inability to clear the mucus naturally invites colonization by bacteria, which ultimately destroys lung tissue. Diagnosis is made by a “sweat test,” which measures the cell’s salt regulation.

The United States Senate has designated May National Cystic Fibrosis Awareness Month.

For more about CF, visit here