Novartis has asked the FDA and EMEA to approve Gleevec for four rare forms of cancer. These include Dermatofibrosarcoma protuberans, or DFSP, a type of tumor that begins as a hard lump in the skin, and three kinds of blood cancer. Due to the rarity of these diseases the company was unable to carry out the large-scale Phase III trials that are usually a prerequisite for requesting additional indications for a drug. Gleevec has already been approved for myeloid leukemia and a type of stomach cancer.
The diversity of these indications suggests future treatments might focus more on the underlying genetic mutations that cause cancer rather than on the part of the body the cancer affects. "One day, cancer may no longer be classified by site, or even by single genes or proteins, but instead by the way in which the cancer is expressed. This could potentially give rise to more targeted treatment options such as Gleevec."
- read this article from The Wall Street Journal for more (sub. req.)