Drug Information Q&A Q48. What is a "molecular targeted drug"?
Answer
Molecularly targeted drugs are drugs developed to treat diseases more safely and effectively by targeting specific molecules in the body and suppressing their functions.
Explanation
Molecularly targeted drugs are attracting attention as drugs that efficiently target proteins and genes on the surface of diseased cells (e.g., cancer cells). Some "antibody drugs" (see Q49) are classified as molecular-targeted drugs. Today, there are more than 10 types of molecular-targeted drugs in use, including low-molecular-weight compounds.
Taking cancer drugs as an example, many anticancer drugs attack not only cancer cells but also normal cells, often causing serious side effects. In the past, the effectiveness of treatment was obtained by killing cancer cells, but recent advances in cancer research have revealed that the reason cancer cells proliferate and metastasize is because substances produced by abnormal genes are causing problems. In other words, if we can suppress the function of substances that cause cancer, the proliferation and metastasis of cancer cells should be reduced.
Based on this concept, "molecular targeted drugs" were born.
Molecularly targeted drugs recognize the characteristics of cancer cells at the genomic and molecular levels and target only the specific molecules that cause cancer cell proliferation and metastasis, thereby minimizing damage to normal cells. Although they are not completely free of side effects, they are less burdensome for patients than conventional cancer drugs.
Imatinib, one of the molecular targeted drugs, is a treatment for leukemia (chronic myeloid leukemia), a cancer of the blood. It was developed as a drug with a mechanism that detects abnormal proteins that cause white blood cells to proliferate and suppresses their function. Subsequently, imatinib was found to inhibit the growth of certain types of cancer (gastrointestinal stromal tumors), such as stomach cancer, and the range of indications for imatinib is expanding.
As the use of these drugs advances, it is expected that patients will be able to continue treatment while maintaining a high quality of life, as they will be able to receive treatment on an outpatient basis or at home, even in cases that would have required hospitalization in the past.
Chart/Column
48｜Mechanism of action of "molecular targeted drugs
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