Genetic therapy treatments have become a significant presence in many medical branches. For instance, nearly two-thirds of all gene therapies are now focusing specifically on cancer – typically those in or entering advanced stages.
However, these revolutionary treatment options have grown to transcend any singular concentration, and they are now an inevitable part of medical science in general. These notions in mind, there are a variety of treatments that have risen to the cusp of commercial success – many of which strive to expand long standing medical boundaries. In this regard, the future is now.
The following are a few of the most successful and commercially promising gene therapy treatments currently in development.
As the first FDA-approved CAR-T cell therapy treatment, Novartis’s Kymriah is already regarded as a historic name in gene therapy implementation. The “CD19-directed genetically modified autologous T cell immunotherapy” is aimed at the treatment of patients up to 25 years in age. Specifically, the treatment strives to combat B-cell precursor acute lymphoblastic leukemia (in all forms) that is either refractory or in second or later relapse.
- Availability by country: Kymriah is currently available at various treatment centers in the United States.
- Clinical prevalence/general success: There are currently 36 clinics offering Kymriah, with other centers preparing to administer the treatment in the foreseeable future.
- Patient remission stats: Kymriah was found to reflect an 83% overall remission rate.
- Cost (USD): $475,000
Another big name in recent genetic therapy advancement, Gendicine is the first treatment of its kind to be successfully approved for human use. The recombinant adenovirus strives to eliminate tumors exhibiting mutated p53 genes, which act as tumor suppressors and regulate cell division. This treatment works by entering tumor cells and emphasizing genetic coding for p53 protein needed to fight these cells.
Gendicine was first approved in 2003 by the China State Food and Drug Administration (SFDA), seeing use for the treatment of head and neck squamous cell carcinoma (HNSCC). The therapy remains one of the only gene therapies to be approved to date.
- Availability by country: Gendicine is only available in China.
- Clinical prevalence/general success: The treatment has reflected an exemplary safety record across nearly 30,000 patients and has shown efficacy for cancers other than HNSCC.
- Patient remission stats: 93% response rate across 135 patients receiving it in addition to radiotherapy, with complete remission observed in 64% of the test group.
- Cost (USD): at $360/dose, a full treatment of Gendicine can cost anywhere between $3,000 to $100,000.
Gilead’s Yescarta is a gene therapy primarily intended to treat non-Hodgkin lymphoma. The personalized CAR-T treatment was granted regular approval by the FDA in 2017 – the first such treatment to focus on adult B-cell lymphoma after two or more lines of systemic therapy; it essentially modifies a patient’s white blood cells, adding a chimeric antigen receptor gene known as CAR. This modification allows the cells to recognize and attack lymphoma cells.
Recently, the treatment has encountered a few hurdles, as the Centers for Medicare and Medicaid Services (CMS) were reported to have not yet finalized a payment system for hospitals administering the therapy. Still, despite a currently cloudy price and distribution outlook, Gilead continues to push for Yescarta’s success across the widest possible range of patients.
- Availability by country: Yescarta is only available in the United States.
- Clinical prevalence/general success: Clinics offering Yescarta are limited as the treatment was just approved in October 18, 2017. Gilead has expressed desire in ramping up the amount of clinics available to administer the therapy, proving that the treatment has become stable enough to handle a bigger patient load.
- Patient remission stats: A multicenter clinical trial of Yescarta yielded a remission rate of around 51%.
- Cost (USD): $373,000