Asthma is the leading cause of school absenteeism and affects an estimated 6.2 million American children under the age of 18, according to the American Lung Association. One medicine in development for allergic asthma is a recombinant humanized, monoclonal antibody that stops an antibody known as IgE from attaching to airway cells and triggering inflammation. In clinical trials involving children, those treated with the medicine had fewer asthma attacks, and many were able to discontinue use of inhaled steroids.
Malignant brain and spinal cord tumors, the second most common cancers in children (after leukemia), account for about 17% of malignant tumors, according to the American Cancer Society. Around 3,200 central nervous system tumors are diagnosed each year in children under the age of 20. One medicine in development for malignant glioma is a recombinant protein composed of two parts: a tumor-targeting molecule (IL13) and an anticancer agent. IL13 receptors are found on malignant glioma cells, but not on healthy cells. The medicine is designed to bind to the IL13 receptors on the tumor cells causing the cells to absorb the IL13 and the cancer fighting agent, resulting in cancer cell death.
Cystic Fibrosis is the second most common life-shortening, childhood onset genetic disorder in the United States, affecting approximately 30,000 children and adults, according to the Cystic Fibrosis Foundation. Cystic fibrosis is caused by mutations in the CFTR gene, a chloride channel that regulates the amount of salt and water in pulmonary secretions. Gene function failure causes production of thick lung mucus and despite the use of antibiotics, cystic fibrosis patients suffer from recurrent lung infections and ultimately, respiratory failure. One gene therapy in development directly delivers a functional CFTR gene to the airway cells of the lung to correct the regulation of salt excretion.
Diabetes affects about 176,000 Americans under the age of 20, and about 2 million adolescents between the ages of 12 and 19 have pre-diabetes, according to the American Diabetes Association. One medicine, already approved for use in adults, is being tested in children with type 1 diabetes. The medicine has a rapid onset of action and a shorter duration of action than regular human insulin, offering greater mealtime dosing flexibility because it can be taken before or after a meal.
Epilepsy affects 45,000 children under the age 15 annually in the United States, according to the Epilepsy Foundation. The highest incidence of epilepsy is under age 2 and over age 65. One medicine is in development for both partial-onset seizures in patients 12 years and over, and for Lennox-Gastaut syndrome (LGS) in patients four years and over. LGS is a severe form of epilepsy, where seizures usually begin before four years of age, and often involve several types of seizures. In clinical trials, the medicine has shown to have clinical benefit in the treatment of these types of epilepsy.
Juvenile Rheumatoid Arthritis is the most common form of arthritis in children, affecting 70,000-100,000 children under age 16, according to the American Academy of Orthopedic Surgeons. One medicine in development is a humanized interleukin-6 monoclonal antibody that reduces the activity of a key cytokine (IL6) involved in the inflammatory process. Studies suggest that by reducing the activity of IL6 it may reduce inflammation of the joints, prevent long-term damage and relieve certain systemic effects of rheumatoid arthritis such as anemia, fatigue and osteoporosis.
