The beginning of a new era of personalized medicine.
The National Health Service (NHS) is the publicly-funded national healthcare system for England. Together with the biopharmaceutical company, Gilead Sciences, the two organizations have reached an agreement to provide CAR T-cell therapy to patients with a type of cancer that has, sadly, left them running out of options. CAR T-cell therapy uses a patient’s own immune system to re-engineer their T-cells to target the CD19 protein expressed by cancerous B cells, thereby tackling the cancer. Its dosage is specifically tailored to each patient’s own immune system in relation to its ability to ward off cancerous cells.
Axicabtagene ciloleucel, also known as Yescarta, will be made available to patients whose large cell lymphoma has recurred or stopped responding to previously used treatments, leaving them with only a few months to live. It is a part of the NHS’s long-term plan to improve cancer outcomes by increasing investment in such services. “The NHS has world-leading clinicians, researchers and scientists, and today’s announcement is proof positive that we are open to constructive and flexible partnerships with industry that rapidly bring life sciences innovation to NHS patients in a way that is also fair to British taxpayers” said Simon Stevens, chief executive at NHS England.
The beginning of a new era of personalized medicine
Yescarta has been approved for the treatment of adult patients with primary mediastinal B-cell lymphoma (PMBCL) and diffuse large B-cell lymphoma (DLBCL) who have failed to respond to two previous chemotherapy sessions. Its approval came from the Food and Drug Administration (FDA), who made their decision based on data gathered from the ZUMA-1 clinical trial. The trial involved the participation of 101 adults with relapsed/refractory large B-cell lymphoma and found that 72% of the patients treated with a single infusion responded to the therapy. Moreover, 51% of the patients remained in complete remission six months following the therapy.
Yescarta, which was previously accessible only to patients in Europe through clinical trials, is now making headway among hospitals in several areas around England. The hospitals that are preparing to offer the therapy to nearly 200 patients with a form of lymphoma on a yearly basis are in:
At its regular price, Yescarta would cost around 300,000 pounds per patient. However, the new agreement by the NHS and Gilead Sciences has allowed for the funds for the therapy to come from The Cancer Drugs Fund (CDF) in England. While there is still a need for a permanent funding agreement, the current arrangement is a good step forward for England.
“It’s admirable that the NHS has worked to make this pioneering treatment available so quickly, giving hope to hundreds of patients and their families,” said Dr. Alasdair Rankin, director of research and patient experience at Bloodwise.
The hurdles of a medical revolution
In treatment with Yescarta, a few T-cells are collected from the patient through leukapheresis, which is a procedure that involves separating the T-cells from other blood cells in a blood sample. The T-cells are then sent to a laboratory, where they are re-engineered by being mixed in with a chimeric antigen receptor gene (CAR) to toughen their ability to identify and kill off cancerous cells. Afterward, they are multiplied into millions and millions of T-cells.
In the meantime, the patient needs to undergo three days of chemotherapy sessions after which he or she will be able to receive the newly re-engineered T-cells via intravenous administration. Following the infusion, the patient will need to be monitored for at least one week so that the cancer care team is able to recognize any unwanted side effects and to take action if they should arise. One month following the therapy, it is recommended that the patient stay near the cancer center where they received treatment for further monitoring.
In regards to its safety profile, Yescarta may trigger unwanted side effects. It carries a warning label for neurotoxicities and cytokine release syndrome, which is a systematic inflammatory response that can cause high fever and flu-like symptoms. Both neurotoxicities and cytokine release syndrome can be life-threatening or fatal. Other possible side effects include severe infections, a weakened immune system, and low levels of blood cell count. They usually appear within the first week, although some side effects may appear later on.
In order to guarantee its long-term safety, the FDA requires hospitals to be certified in the specific process involving treatment with Yescarta. That being said, any staff members involved prescribing or supplying the therapy are required to be trained in order to be able to diagnose and treat neurotoxicities and cytokine release syndrome – should they arise. Furthermore, patients must be informed about the potentially severe side effects, as well as about the significance of returning to the treatment site if any unwanted side effects should develop.
A new scientific paradigm
Seeing as how cancer survival in England is reaching high levels, the NHS continues to play a large role in the development and implementation of cutting-edge technology for cancer treatment. So far, this is just the beginning of the benefits that CAR T therapy has to offer. “CAR-T cell therapy is one of the most promising new treatments in a generation for lymphoma and leukemia, and NHS patients will now be among the first in the world to benefit,” said Stevens.
In just 10-20 years, gene therapy has transformed from a promising approach to a therapeutic solution for ‘uncurable’ forms of cancer. And the approval of Yescarta validates the continuous support in the development of new and hopefully, routine therapeutic solutions.
Nevertheless, even following its approval, the FDA recommends more clinical studies be performed in order to speed up the safe implementation of this therapy in other health-related areas. As treatments continue to improve in terms of safety and tolerability, as well as to become more effective, this will be of great benefit to patients with other types of cancers in the years to come. “We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms,” said Scott Gottlieb, Commissioner of the FDA.