Why is progress in rare cancers such a challenge?


Rare cancers are often called “orphan” diseases—and with good reason. They have nobody to look after them unless they are fortunate enough to be adopted.

While there is clearly a need for more research and improved treatments for common cancers, much progress has been made in recent years. This progress has been due to interest and funding from several sources. Common cancers affect many people; thankfully government funding from the National Cancer Institute is available to research these cancers. In addition, because there are many patients to study, academic centers have an easier time performing research. Common cancers are also an area that attracts research and development funding from pharmaceutical companies, who need to be sure that there is potential for a marketable product before they commit their resources. Private funding, from patients, families, and friends also makes a difference.

Rare cancers have none of these advantages. They are not a public health hazard, and government funding is not readily available. It is more challenging for academic institutions to perform research on small numbers of patients. Pharmaceutical companies are wary of developing products that might be used by only a handful of people. And there are not as many patients and families to raise awareness.

Ironically, rare cancers may also be among the most treatable. We already know that some of these cancers have a very specific cause that can be effectively targeted with the right treatment. Examples include gastrointestinal stromal tumors (GIST), or acute promyelocytic leukemia, both which are caused by specific molecular alterations that can be successfully treated with  molecularly targeted therapies.

One in four people with cancer have a rare cancer. With more attention and funding, we will have a tremendous opportunity to make a difference for the many people who suffer from these potentially treatable, and even curable, diseases.