Pathology and Clinical Trials
The following post is written by Mrs. Paula Takacs who previously posted a note about The Value of Pathology from the perspective of a cancer patient. For more information on Paula, her story and a race to support her foundation in April, please visit The Paula Takacs Foundation for Sarcoma Research and The Sarcoma Stomp. My personal thanks to Paula for her perspective on an important issue pertinent to many of us in the pathology and laboratory communities.
It is amazing how far researchers have come over the last decade in addressing cancer treatment. One of the more stunning themes has been the shift from curing cancer to the more realistic goal of making cancer a chronic disease. One of the drivers of this has been the genetic study of tumors and the identification of their pathways.
One recent study that I have found interesting focuses on leiomyosarcoma and is being conducted at Brigham and Women’s Hospital and Harvard Medical School. Not only are leiomyosarcomas rare and genetically complex, but like so many other sarcomas, are pretty resistant to traditional therapies. The study attempts to identify diagnostic markers, which will allow doctors to recognize tumors with recurrent or metastatic potential, ultimately improving diagnostic accuracy and better determine targeted treatments.
Dr. Bradley Quade, a pathologist, and one of the principal investigators on the trial stated, “When one studies the chromosomes of leiomyosarcoma, where the DNA is packaged, one finds both numerical and structural abnormalities, many of which vary from cell to cell, suggesting a great deal of ‘genomic instability.’ Thus, finding a ‘leiomyosarcoma gene’ is like looking for a genetic needle in a chromosomal haystack. By studying earlier stages, we hope that the size of the haystack is much, much smaller!”
Molecular studies are at the core of another exciting type of targeted treatment. However, this time, by going after the protein MDM2, researchers at many of the large pharmas are hoping to not only cut off the pathway for liposarcoma, but are hoping this pathway may be effective across a number of different cancers, including leukemia.
Treatment approaches for cancer always appear to be evolving. First, it seemed as though chemotherapy was the answer, eradicating all fast growing cells in attempt to kill the cancer cells. Then, the focus shifted to targeting cancer specific mutations. However, this newest concept attempts to find genetic mutations common across a number of cancers, not just one.
The impact for rare cancers is significant. One of the greatest setbacks historically is that pharmas had little interest in producing a drug with such a limited distribution, due to high R&D costs. Often, separate orphan drug funds are established, but in order to preserve profits, companies go after products, which can bring in the greatest return. After all, a bankrupt company does no one any good. However, if rare cancers could be investigated along side of common cancers, they should finally receive some attention.
Overall, I feel very encouraged by the developments in clinical research and look forward to what the next wave of ideas will bring. It is amazing to think that a molecular target may one day be more critical to treatment than the actual organ from which the tumor arises. Now, we just need to find a way to fund the researchers, so they have the tools needed to do their jobs.
