Development and Implementation of Gene Therapy Treatment

Many of you may be aware that earlier this week, news broke that the FDA approved the second ever gene therapy treatment for adult aged individuals with non-Hodgkin lymphoma. This cancer accounts for about 4% of all cancers in the USA, and the B-cell category of non-Hodgkin lymphoma is a particularly difficult type of cancer to treat.Yescarta

genetically modifies the patient’s own white blood cells, which are injected back into them, as a means of targeting and killing cancer cells. The treatment can be given at certified hospitals and the treatment is still undergoing observational study to ensure the safety and effectiveness of the therapy. This is particularly due to the potentially serious side effects that can result from using the therapy. The FDA’s approval of this gene therapy treatment shows its continued support and enthusiasm towards turning gene therapy from a theory into an effective, accessible treatment for so many people. In fact, the FDA Commissioner Scott Gottlieb was quoted on the FDA website saying, “This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products…We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”

This comes shortly after the FDA approved the first every gene therapy treatment plan for use in the USA in August of this year. The first treatment type approved was Kymriah, a treatment approved for patients under 25 years of age with B cell acute lynphoblastic leukemia (ALL). Understanding the need to follow patients closely, especially since gene therapy is such a new treatment method, Novartis, the producer of Kymriah is planing on a long term follow up study to evaluate lasting risks and health outcomes.

Of note, these are not the only gene therapy treatments available globally. In fact, in 2003, China approved a therapy called Gendicine for head and neck squamous cell carcinoma. This treatment causes tumor cells to upregulate their expression of the p53 oncogene. This gene is implicated in tumor and cancer development, and the increased expression of the gene causes the cell to marked at damaged. These cells then undergo apoptosis (cell death) or are attacked by the body’s natural tumor fighting immune responses. Due to the diverse nature of cell types and cancer types, many of these treatments available on the market or under development are only available to a specific subset of the population.

Although gene therapy research and development has evolved enormously over the last 15 years (at least), this article, ‘Gene Therapy for Cancer Treatment: Past, Present and Future’ posted in Clinical Medicine and Research is a great resource that explains the 3 main categories of gene therapy development and treatment methods. The article is a little dated, since it was published in 2006, but is never the less a great resource! As these sources point out, most of these treatments plans are recommended for patients who have failed to respond to the other available treatment options and have no other choices left, and many are recommended to be used in conjunction with existing treatment methods. Looking into the price of these new gene therapy options shows one limiting factor- cost. A single Kymriah treatment costs $475,000! Ideally, as the development and use of gene therapy methods increases, the price will decrease, and we will replace older, less refined treatments with a new standard of care. The battle with cancer has been a long and winding road, but it looks that a major breakthrough in treatment and patient management is in our future!


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