Children

Asthma is the most common chronic disorder in childhood and affects an estimated 6.7 million American children under the age of 18, according to the American Lung Association. In a recent study, the National Institutes of Health found that more than 50 percent of current asthma cases in the United States can be attributed to specific allergies. 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 on average, 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 21 percent of malignant tumors in children, according to the American Cancer Society. Around 3,800 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 both the IL13 and the cancer fighting agent, resulting in cancer cell death.

Cystic Fibrosis is a life-shortening, childhood onset genetic disorder, affecting approximately 30,000 children and adults in the United States, 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.

Crohn’s disease, a chronic disorder that causes inflammation of the digestive or gastrointestinal tract, affects about 100,000 American children under the age of 18, according to the Crohn’s and Colitis Foundation of America. One medicine already approved to reduce the signs and symptoms of moderate to severe Crohn’s disease in adults, and now in pediatric trials, is a human-derived antibody that binds to human tumor necrosis factor alpha (TNF alpha). TNF alpha is a protein responsible for the inflammatory process in the intestines that is caused by Crohn's disease.

Diabetes affects about 186,300 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 2 diabetes. The medicine was the first DPP-4 (dipeptidyl peptidase-4) inhibitor approved in the United States for the treatment of type 2 diabetes. The medicine enhances a natural body system called the incretin system, which helps to regulate glucose by affecting the beta cells and alpha cells in the pancreas. Through DPP-4 inhibition, it works only when blood sugar is elevated to address diminished insulin due to beta-cell dysfunction and uncontrolled production of glucose by the liver due to alpha-cell and betacell dysfunction.

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 partial onset seizures and already approved for adults. Seizures begin in the brain, which is made up of millions of nerve cells which communicate with each other by releasing electrical signals. In people with epilepsy, overexcited nerve cells release too many electrical signals that can then cause seizures. The medicine works by reducing the number of "extra" electrical signals that are sent out from damaged or over-excited nerves.

Juvenile Arthritis is one of the most common childhood diseases in the United States, affecting some 294,000 children under age 18, according to the Arthritis Foundation. 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 arthritis such as anemia, fatigue and osteoporosis.