New research shows that gene therapy may be a viable treatment option for those with leukemia or other blood cancers. In these studies, researchers filtered patients’ blood and removed all the white blood cells also known as ‘T-cells’, that are part of the body’s immune system. They then added a gene to the T-cells in order to turn them into “cancer fighter” cells. These altered T-cells were infused back into the patients’ body over the course of three days.
The concept of coaxing a patient’s own blood cells into attack cells that target and eliminate any cancer cells is quite astonishing. Currently, more than 120 patients with different types of blood and bone marrow cancer have received the treatment and many have went into remission and stayed in remission up to three years later. In one study, five adults and 19 of 22 children with acute lymphocytic leukemia (ALL) were cleared of cancer. However, a few did relapse after the study was completed. In another trial, 15 of 32 patients with chronic lymphocytic leukemia (CLL) responded to the treatment well with seven experiencing complete remission from their cancer. All patients that underwent this treatment had few options left and some were ineligible for bone marrow transplants or refused to have that procedure done.
This treatment is a beacon of hope for patients with any type of blood cancer. The immune system and these modified attack cells are working together to attack tumors in an effective way. Penn’s Abramson Cancer Center has treated 59 patients with gene therapy, along with researchers at U.S. National Cancer Institute, Memorial Sloan-Kettering Cancer Center, University of Texas MD Anderson Cancer Center and Baylor University, who have treated smaller groups of patients.
Many companies are developing these types of cancer therapies and researchers are hopeful for a clinical trial next year that could lead to federal approval of the treatment by 2016. The gene therapy is catered towards each patient and lab costs are around $25,000. The treatment has been seen to cause flu-like symptoms and other side effects, however doctors assure that these symptoms have been temporary and reversible.
Genetic Alliance and its partners have created a system in which participants can make their own choices, with the help of relevant guides, and can change preferences as needed on a contextual basis. It is called the Platform for Engaging Everyone in Research (PEER Network). It can be seen in three different manifestations: TrialsFinder, Reg4All and Free the Data. It is time to put participants at the center and drastically change the forces that shape clinical trials and the resulting data.