Potential for First Gene Therapy Approval in the U.S. Gets a Green Light from FDA Panel
Key Takeaways:
- The gene therapy CTL019 has received unanimous consent from the FDA advisory board in helping to treat acute lymphoblastic leukemia (ALL).
- Functioning as a living drug, CTL019 works by altering a patient’s existing immune cells to combat ALL, potentially revolutionizing gene-based treatments.
- Developed with support from Stand Up to Cancer and St. Baldricks’s Foundation, CTL019 is particularly beneficial for B-cell acute ALL patients who have relapsed or found no relief in other treatments.
- Despite testimonies from witnesses about the transformation brought by CTL019, the treatment comes with a high price and the potential for serious complications.
- Doubts about potential side effects and a hypothetical risk of secondary cancers remain.
Activating a new epoch of gene-centered healthcare, the soon-to-be approved gene therapy CTL019 has gained unanimous consent from an FDA advisory board. This pioneering treatment might shine a ray of hope for thousands suffering from acute lymphoblastic leukemia (ALL).
CTL019: A Gamechanger in Gene Therapy
An ingenious breakthrough in medical science, CTL019 functions as a living drug, honing a patient’s existing immune cells to combat ALL. This innovative approach might revolutionize the realm of gene-based treatments, which hold substantial potential against a spectrum of diseases.
According to a leading pediatric oncologist, Dr. John Maris, “the fully commercialized CTL019 therapy might save numerous lives, primarily the children and young adults who had no other effective treatment for advanced B-cell ALL”. Dr. Maris believes that the therapy indicates a significant shift in how the disease is managed.
Funded Research and the Manufacturing Giant Behind CTL019
Two noteworthy cancer charities, the Stand Up to Cancer and the St. Baldricks’s Foundation supported the development of CTL019. The treatment is a boon for B-cell acute ALL sufferers who have either relapsed or found no relief in other treatments. The recommended age for the therapy is between 3 and 25 years, as stated by a prime news source.
ALL, impacting nearly 5,000 American citizens annually, is prevalent in about 60% of children or young adults. For 85% of the affected population, standard therapeutic procedures can cure the cancer, but in the remaining 15% of cases, the cancer persists despite treatment, or the patient faces a relapse.
The Life-Changing Impact of CTL019
Witnesses at the FDA panel advocated for the approval of CTL019, sharing their transformative experiences with the treatment. Among them were patients who survived extreme adverse conditions, sailed through the therapy, and have managed to remain cancer-free since then.
The Science Behind CTL019
This therapy begins by extracting millions of T-cells of a patient’s immune system and freezing them. These cells are then manipulated genetically in lab settings, conditioning them to target and terminate the ALL cells.
The scientist’s toolkit includes a harmless, non-functional form of the HIV virus, tasked with carrying the new genetic material into the T-cells of the patient. Post genetic modification, these enhanced T-cells target the malignant B-cells responsible for ALL.
The modified T-cells, aptly dubbed “serial killers” by Dr. Carl June, a renowned researcher, are incredibly efficient, with one cell capable of eliminating 100,000 cancer cells. These re-engineered cells then get reinserted back into the patient through an IV drip.
As per a leading publication, numerous patients have reported significant remissions from ALL, some alleging potential cures, following a single dose of treatment.
Challenges and Concerns
Despite its promising future, the therapy comes at a hefty price, estimated by analysts to cross $300,000. The complex nature of the treatment also poses a risk of serious complications, thus initial applications of CTL019 will be limited to only 30 or 35 approved centers across the U.S.
Nevertheless, potential side effects and a hypothetical risk of secondary cancers, which can only be assessed with time, remain a cause of concern.
For More Information
Learn more about Acute Lymphoblastic Leukemia by visiting the Lymphoma and Leukemia Society website.
