Selected Medicines in Development for Children

Selected Medicines in Development for Children

Asthma is one of the most common chronic disorders in childhood and affects an estimated 7.1 million American children under the age of 18, according to the American Lung Association. Eosinophils (a type of white blood cell) are important for killing parasites in the body, but in asthma they can accumulate in lung tissue and cause damage to the lining of the air passages. Interleukin-5 (IL-5) is essential for the production, activation and maturation of eosinophils.  A monoclonal antibody in development is an anti-IL-5 that inhibits IL-5 resulting in a sustained reduction in the numbers of eosinophils accumulating in the lungs and stopping those already there from causing damage.

Malignant brain and other nervous system tumors are the second most common cancers in children (after leukemia), accounting for about 27 percent of malignant tumors in children, according to the American Cancer Society. One medicine in development for malignant glioma is a recombinant protein comprised of two parts: a tumor-targeting molecule (IL-13) and an anticancer agent.  IL-13 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.

Crohn’s disease, a chronic disorder that causes inflammation of the digestive or gastrointestinal tract, affects about 140,000 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 215,000 Americans under the age of 20, 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 326,000 children under the age 15 in the United States, according to the Epilepsy Foundation.  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.

Fragile X Syndrome is the most common cause of inherited intellectual disability (mental retardation). It is caused by a change in a gene called FMR1 where a small part of the gene code is repeated on a fragile area of the X chromosome. The FMR1 gene is responsible for making the FMRP protein needed for the brain to grow properly. A defect in the gene makes the body produce too little of the protein, or none at all. Boys and girls can both be affected, but because boys have only one X chromosome, a single fragile X is likely to affect them more severely. A medicine in development for Fragile X is potentially the first to treat the underlying disorder instead of just its symptoms. The medicine is an antagonist of mGluR5 (metabotropic glutamate receptor 5), a receptor protein on brain cells that is involved in many aspects of normal brain function. The FMRP protein normally acts as a blocker for the brain cell pathways activated by mGluR5.  When the FMRP protein is missing, mGluR5 pathways are overactive resulting in behavioral and cognitive impairments associated with Fragile X. The medicine is designed to block the activity of mGluR5.

Inherited Hypercholesterolemia is an inherited metabolic disorder resulting in an abnormal amount of cholesterol in the blood. It leaves patients at a high risk for adverse cardiovascular events, such as accelerated atherosclerosis and early heart attack. Dietary treatment seldom helps in these cases. A first-in-class medicine in development is an inhibitor of the protein, apolipoprotein, which plays a pivotal role in the production of low-density lipoprotein (LDL) or “bad” cholesterol.  LDL is a type of protein that transports cholesterol and triglycerides from the liver to tissue in the body.

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