The Food and Drug Administration (FDA) recently granted Oncophage® orphan drug status for metastatic melanoma.

Melanoma is a cancer of the skin that usually begins in the form of a mole. The cancer can grow deep into the skin and spread to different parts of the body through blood or lymph vessels. It usually spreads first to lymph nodes that are near the site of cancer origin and when advanced, can spread to organs and other lymph nodes throughout the body. Treatment for advanced or metastatic melanoma may consist of surgery, radiation, chemotherapy and/or biologic therapy. However, the prognosis for patients diagnosed with this disease is poor, as melanoma typically does not respond well to standard therapies. Biologic therapy, or therapy used to stimulate the immune system to fight cancer, has demonstrated anti-cancer activity in the treatment of melanoma. Researchers are trying to expand the role of biologic therapy for melanoma, with many clinical trials underway evaluating this therapeutic approach.

Oncophage® (HSPPC-96) is a vaccine that is composed of components of the patient’s own cancer cells, which stimulates the patient’s immune system to attack the cancer. Every cell in the body displays specific antigens (small carbohydrates and/or proteins) on their surface. The immune system relies on antigens to recognize “foreign” material, such as bacteria, viruses or cancer cells. Dendritic cells are specific immune cells that “present” foreign antigens to other cells in the immune system. The immune system then mounts an attack against foreign cells that display the antigen(s) that were presented by the dendritic cells.

Heat shock proteins (HSPs) are an important component of all cells and their many roles are becoming more apparent to researchers. One role of HSPs is to form a complex with antigens and “escort” the antigens to dendritic cells. HSPs of a cancer cell form a complex with antigens specific to the cancer cell. Clinical studies have revealed that when HSPs from a cancer cell loaded with antigens bind to a dendritic cell, the HSP/antigen complex is internalized and biochemical processes occur within the dendritic cell that further stimulate the immune system. First, this process allows the dendritic cell to “display” the antigens specific to the cancer cells to other immune cells to initiate an immune response against the cancer. In addition, this process has been shown to activate lymphocytes, which are very important cells of the immune system; induce dendritic cells to secrete cytokines, substances released into the lymph that stimulate immune cells; and induce dendritic cell maturation.

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Oncophage® is prepared by isolating HSPs and antigens from a patient’s cancer cells. The HSP/antigen complexes are made into a vaccination which the patient receives once a week on an outpatient basis. The injected HSP/antigen complex stimulates the immune system to fight cancer cells elsewhere in the body.

Orphan drug status is granted to agents that target an illness affecting 200,000 or fewer people in the U.S. Financial incentives, such as tax breaks, specific regulatory fee waivers, available funding for clinical trials and marketing exclusivity for 7 years if the product is approved are granted in order to aid in the development of the product. Oncophage® has entered phase III clinical trials for both metastatic melanoma and renal cell carcinoma. Researchers are hopeful that data analysis from the trials can begin by mid-year next year. Oncophage® has already been issued “fast-track” status by the FDA for metastatic melanoma and renal cell carcinoma.

Patients with metastatic melanoma may wish to speak with their physician about the risks and benefits of participation in a clinical trial evaluating Oncophage® or other promising therapies. Two sources of information regarding ongoing clinical trials include the National Cancer Institute ( and also provides personalized clinical trial searches on behalf of patients.

Reference: Antigenics. Antigenics’ Oncophage – HSPPC -96 – receives orphan drug status in melanoma; second orphan drug designation for Oncophage. Available at: Accessed July 31, 2002.

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