Backgrounder: Selected Biologics in Development

Backgrounder: Selected Biologics in Development

Alzheimer’s disease affects more than 5 million Americans, according to the Alzheimer’s Association.  It also costs the U.S. health care system $200 billion a year. These sobering statistics are projected to get much worse as the 76 million American baby boomers age.A gene therapy for the treatment of Alzheimer's disease in clinical trials is designed to deliver nerve growth factor (NGF) to the brain.  NGF is a naturally occurring protein important for neuron survival.  The gene treatment is injected into the brain region where neuron degeneration occurs in Alzheimer’s disease. It is thought that the resulting sustained expression of NGF in the neurons can restore their lost functions, leading to memory and cognition improvement. 


Asthma is a debilitating condition for more than 25 million Americans, including 7.1 million children under the age of 18, according to the American Lung Association.  A monoclonal antibody (mAb) 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.  MAb’s are highly specific, purified antibodies that are derived from only one clone of cells and recognize only one antigen. 


Lymphoma is a blood cancer that develops in the lymphatic system. About 80,000 new cases are diagnosed each year and more than 20,000 Americans die from lymphoma, according to the American Cancer 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.  A third-generation antisense medicine in development for the treatment of lymphoma inhibits production of a specific protein which regulates many key genes important in cancer growth -- angiogenesis, cell metabolism, cell proliferation, cell death and cell invasion.  An overexpression of the protein in tumors results in resistance to treatment.  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 monoclonal antibody in development targets receptors thought to regulate B-cell function.  B-cells can cause the immune system to turn against itself by producing antibodies against the body’s own cells and tissue, resulting in inflammation and tissue damage. 


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 – about 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 (mAb) 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 236,000 to 327,000 people alive today in the United States and an estimated 12,000 new cases of spinal cord injury each year, according to the Spinal Cord Injury Information Network. One monoclonal antibody (mAb) is in development for potential use in the regeneration of corticospinal tract fibers resulting from an acute spinal injury. By neutralizing a protein that inhibits growth of spinal fibers, the mAb may promote regeneration of spinal fibers and functional recovery in spinal cord injury patients.


* From Biologic Medicines in Development, 2013

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