Adjust Font Increase Font Decrease Font
  • Email
  • Print

Backgrounder: Selected Biotechnology Medicines in Development

Age-Related Macular Degeneration (AMD) is the leading cause of blindness in the United States for those ages 55 and over, and it affects 10 million Americans, according to the American Macular Degeneration Foundation.  AMD is diagnosed as either wet (neovascular) or dry (non-neovascular). In wet AMD, new blood vessels grow beneath the retina and leak blood and fluid, causing disruption and dysfunction of the retina and creating blind spots. A new medicine in development for the wet form of AMD is a recombinant fusion protein that binds to the growth factor protein that plays a critical role in blood-vessel formation in the eye. The medicine works by blocking new blood-vessel growth and leakiness.
 
Asthma is a debilitating condition for more than 23 million Americans, including 7 million children under the age of 18.  A monoclonal antibody in development is designed to block the IL-13 cytokine, a protein messenger between cells that triggers inflammation.   When IL-13 is overexpressed, it results in airway inflammation which is a key feature of asthma. Blockage of IL-13 will reduce the risk of asthma and other respiratory diseases.  Monoclonal antibodies are highly specific, purified antibodies that are derived from only one clone of cells and recognize only one antigen.
 
Chronic lymphocytic leukemia (CLL) is the most common type of leukemia in adults and does not occur in children.  In the United States, an estimated 100,760 people are living with or in remission from CLL, according to the Leukemia & Lymphoma Society. When a gene is known to contribute to a particular disease, antisense technology can synthesize a strand of nucleic acid (DNA, RNA or chemical) that binds to the messenger RNA produced by the gene and inactivate it.  An antisense medicine in development for the treatment of CLL inhibits production of a specific protein made by cancer cells that is believed to block chemotherapy-induced cell death.  By reducing the amount of the protein in cancer cells, the antisense drug may be able to enhance the effectiveness of current anticancer treatment.
 
Lupus affects at least 1.5 million Americans, 90 percent of whom are women, according to the Lupus Foundation of America.  In patients affected with lupus, the body develops antibodies (immune system proteins) that react against normal tissue, leading to inflammation, pain, tissue injury and major organ damage.  A new monoclonal antibody in development targets B-cells that cause the immune system to turn against itself and produce antibodies against the body’s own cells and tissue.  
 
Melanoma accounts for less than 5 percent of skin cancer cases but causes more than 75 percent of skin cancer deaths, according to the Skin Cancer Foundation.  In the United States, one person dies from melanoma every 62 minutes.  Two therapeutic vaccines in development stimulate the immune system to fight cancer but in different ways.  A virus-based therapeutic vaccine in development for the treatment of melanoma is genetically-modified to replicate selectively in tumor cells and express a gene for an immune-stimulating protein.  It is injected directly into the tumor where it replicates and spreads within the tumor, causing the death of cancer cells and stimulating the immune system to destroy cancer cells.  It is also being tested in head/neck cancer.  Another medicine in development to treat melanoma is an immunotherapeutic designed to train the immune system to recognize and eliminate cancer cells in a highly specific way.  The medicine is a combination of tumor antigens, delivered as recombinant proteins, and a proprietary adjuvant (an agent that modifies another substance in its action) to stimulate the immune response to cancer cells. It is intended to only affect cancer tissue and not harm normal tissue.
 
Psoriasis is the most prevalent autoimmune disease in the United States.  As many as 7.5 million Americans – approximately 2.2 percent of the population – have psoriasis, according to the National Psoriasis Foundation.  A potential medicine in development for the treatment of psoriasis is an engineered human antibody to interleukin-17 (IL-17).  IL-17 is a key cytokine involved in inducing and mediating inflammation associated with psoriasis. 
 
Rheumatoid Arthritis (RA) affects 1.3 million Americans, most of them women, according the Arthritis Foundation.  RA is a chronic autoimmune disorder where the joints become painful, swollen, stiff, and in severe cases, deformed. One medicine in development is a fully human monoclonal antibody directed against interleukin-6 (IL-6) alpha, a signaling protein involved in the regulation of immune and inflammatory responses associated with RA.  The mAb interrupts the inflammatory signaling cascade of IL-6 by blocking its binding to a certain receptor necessary for inflammatory cascade.
 
Spinal Cord Injuries affect about 225,000 to 288,000 in the United States and there are an estimated 11,000 new cases of spinal cord injury each year, according to the Spinal Cord Injury Information Network. One monoclonal antibody is in development for potential use in the regeneration of corticospinal tract fibers resulting from an acute spinal injury. The antibody neutralizes a protein that inhibits growth of spinal fibers.