For those of you who don’t know what an orphan disease is, it is a disease that effects so few people that it is essentially “orphaned” in medical research. My daughter, who was 10 years old at the time of my diagnosis understood my cancer was very rare. She came up to me one day as I was searching for treatment and said “Mom, no one is going to try real hard to cure appendix cancer, are they? It would cost too much money and help too few people”. She understood exactly what was meant by an orphan disease, it is a disease that is not cost-effective to attempt to cure. Remember the movie “Lorenzo’s Oil”? My daughter later said when she was older and I started this non-profit that I had “adopted” the orphan disease.
Appendiceal cancer most often kills by spread into the abdomen (peritoneal surface malignancy) and interference with digestion and the functioning of organs in the abdomen. Because of this, it can be grouped in research with diseases that can kill in the same way but are more common…ovarian, colon, gastric etc. that can also cause “peritoneal surface malignancies”.
Most of the specialist listed on my website treat these other cancers along with appendiceal cancer. Many of the current clinical trials using this therapy include appendiceal cancer with other cancers that have metastasized into the abdomen, such as colon and ovarian cancer.
Since appendiceal is an orphan disease, we benefit from this inclusion in research studies.
Heated intraperitoneal chemotherapy is a benefit for these cancers, as there exists a “plasma-peritoneal barrier”. Much like the “blood brain barrier” that protects harmful things in the blood from reaching the brain, the plasma-peritoneal barrier can prevent what is harmful in the blood from traveling to the abdomen. For this reason, IV chemotherapy is not as effective for these cancers, but chemotherapy placed directly in the abdomen is effective. The chemotherapy in the abdomen can also be concentrated more, as just like chemo from the blood does not enter the abdomen as well, chemo in the abdomen does not enter the blood stream and circulation as well, hence few side effects from the peritoneal (abdominal) chemo. A good thing!