FDA Panel Approves What Could Be the First Gene Therapy in the United States

First there was Sipuleucel-T (Provenge) that involved the modification of a person’s own white blood cells (T cells) to sensitive these cells to the presence of and to treat prostate cancer, now we might be on the verge of an even bigger change, personalized gene therapy, or the modification a person’s own genetics to tweak the immune system to become sensitized to a cancer.

On Wednesday a U.S. Food and Drug Administration advisory panel gave unanimous approval to what could soon be the first gene therapy to be marketed in the world and it is for a rare cancer.

CTL019, a potential treatment genetically tweaks a person’s own immune system cells into what scientists call "a living drug" to battle a form of acute lymphoblastic leukemia (ALL).  

Although not guaranteed, usually the FDA accepts and approves this type of strong recommendation from their Advisory Panels.  If it does, this would be a landmark, game-changing treatment of ALL.   Not only would this approval reverberate in the ALL Community, its implications will open a new era of gene-based medicines, opening up the future and changing the way we treat many diseases including cancers.

According to Dr. John Maris, a pediatric oncologist at The Children's Hospital of Philadelphia. "This is truly a turning point in the management of this disease."  He also said that "When fully commercialized, this [CTL019] therapy will no doubt save the lives of many children and young adults who have had no other effective therapy for relapsed and refractory B-cell acute lymphoblastic leukemia [ALL]," 

ALL is a rare pediatric cancer affects about 5,000 Americans each year, about 60 percent of whom are children or young adults. In 15 percent of ALL cancers, our current treatments fails and patients do not respond or they relapse.  CTL019 might offer these 15% a new option for treatment.

Like Provenge, an individual’s cells are removed from them and then shipped to a laboratory at the drug maker Novartis (the developer of the investigational treatment) (cells for Provenge are shipped to Dendreon, not Novartis) where the cells are then genetically modified.  After being modified these sensitized cells are shipped back to the patient and re-infused. 

The treatment is not without its own risks as side effects in some of the trial patients were almost lethal.  However, the end results seem stunning, creating long-term, seemingly cancer-free remissions.

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Joel T. Nowak, MA, MSW wrote this Post.  Joel is the CEO/Executive Director of Cancer ABCs.  He is a Cancer Thriver diagnosed with 5 primary cancers - Thyroid, Metastatic Prostate, Renal, Melanoma and a rare cancer, Appendiceal Cancer.