Panobinostat is an oncology medication initially developed by Novartis Pharmaceuticals. This medication attacks several enzymes involved in the growth and development of cancer cells and appears to be effective as a single chemotherapy agent for some types of cancer. It can also be used in combination therapy with other medications. Initial clinical trials showed promise in the treatment of multiple myeloma, certain solid tumors, and other blood cancers. As of 2012, panobinostat was still considered experimental and was not widely available.
Chemotherapy drugs formulated to attack specific enzymes can hit tumor cells at weak points, interfering with their ability to reproduce, grow, and maintain cell wall integrity. In addition to harming cancer cells, panobinostat also interferes with angiogenesis, the growth of new blood vessels, which cuts off the supply of blood to a tumor and limits the amount of oxygen and nutrients it can access. This combination of assaults can force a tumor to shrink because it cannot survive under these conditions.
Depending on the nature of a cancer, surgery may be appropriate before panobinostat therapy to de-bulk the tumor or remove it entirely, and the medication targets remaining cells in the body. In other cases, the medication may be used to shrink a tumor and its blood supply before surgery, as this can improve patient outcomes. Other medications and therapies like radiation can also be added to the treatment regimen if there are concerns about how well the patient will respond to the cancer treatment.
Patients can take panobinostat orally and initial clinical trials indicated it was well tolerated. Like other chemotherapy medications, it can cause symptoms like nausea, vomiting, and fatigue, but serious complications were not noted. As with other drugs in development, this information was subject to change; larger clinical trials as well as reports from patients after drug approval might reveal patterns of side effects that weren’t apparent in smaller, more controlled trials.
During clinical trial phases, members of the general public do not have access to medications. Patients interested in panobinostat prior to approval would need to enroll in clinical trials, which are usually open on a limited basis to people who meet strict criteria. If this is an option for a patient, a physician may bring it up in counseling. It may also be possible to obtain it on the grounds of compassionate use if a patient is terminally ill, in which case the manufacturer does not accept liability for complications associated with use of the medication.