A fun fact about drug development is that despite the best effort of everyone involved, it's hard to predict what the drug is going to do. Rogaine and Viagra were both originally developed as blood pressure medications. And then there's thalidomide.
Originally infamous for causing birth defects, Thalomid (thalidomide) was given to pregnant women in the 1960's to ease the nausea of early pregnancy.
Serious, lethal birth defects in those patients led to a long-standing ban on drugs in the thalidomide family.
Years later, it was again given to cancer patients to treat nausea. Doctors noticed that in some patients, it helped fight the cancer, so it became a key part of leukemia therapy. But nobody knew how it worked.
In a study presented to the American Society of Hematology, Keith Stewart M.D., MBA found a link between resistance to the cancer drugs in the thalidomide family, and the level of a cellular protein called cereblon, which helps cells develop normally.
Dr. Stewart's research shows that when drugs in the Thalomid family, including Revlimid (lenalidomide) and pomalidomide (Actimid), are given to cancer patients, the drug will usually not work if the patient has a high level of cereblon.
Furthermore, Dr. Stewart found that lowering the level of cereblon in the cell allowed Thalomid to have an effect in fighting the cancer.
Further research in this area could give new targets for multiple myeloma treatment. Or, as Dr. Stewart states, "These findings help us understand which patients may be more or less likely to respond to [Thalomid] therapy."
Research was funded by the Mayo Clinic, a non-profit institution. No conflicts of interest were disclosed by any party.
According to the Leukemia and Lymphoma Society, there are over 245,000 people in the United States living with some form of leukemia, and over 44,000 new cases will be diagnosed each year. Among children with cancer, one out of every three will have some form of leukemia. However, over 90% of all cases of leukemia occur in adults.
Leukemia broadly defines a number of different cancers that originate in the bone marrow and blood cell lines, and typically produces an abnormal increase in the number of white blood cells in the body. Leukemias are subdivided into whether they are acute (occur rapidly) or chronic (develop over many years) and then further subdivided into what type of blood cell they start from, lymphocytic (usually from white blood cells that fight infection, like B-cells) or myelogenous (arising from cells in the bone marrow that eventually produce red blood cells). These categories make up the four most common types of leukemia:
- Acute Lymphoblastic (ALL): Affects adults over age 65 (50% survival); also the most common form of leukemia in children (85% survival)
- Chronic Lymphocytic (CLL): Affects mostly men over age 55 (75% survival)
- Acute Myelogenous (AML): Affects mostly adult men (40% survival)
- Chronic Myelogenous (CML): Affects mainly adults (90% survival)


