To many fighting cancer, palliative care is considered the ending point – just dealing with pain management until life ends.
Recent developments outside of normal cancer treatment have emerged with both promise and staggering treatment pricing. While chemotherapy is still considered a mainstay in fighting cancer, it has its drawbacks in the form of adverse side-effects. As more cancer cells grow in the patient’s body, they take over useful cell space. Chemotherapy drugs are designed to interfere with the cancer cell’s ability to divide and reproduce. Unfortunately, chemotherapy can also interfere with healthy cells, further weakening the patient’s immune system. To many, the treatments mean dealing with the pain and hoping things improve.
Recent approvals from the Food and Drug Administration (FDA) have created controlled chaos with pharmaceutical development companies being purchased at sky-high multiples and drug treatment costs reaching stratospheric heights. Kite Pharma, the developer of an FDA-approved treatment which genetically alters a patient’s cells to actually attack cancer, fits both scenarios. The innovative treatment for aggressive non-Hodgkin’s lymphoma has seen strong positive results in all trials, and the company was recently purchased by Gilead Sciences for $11.9 billion. In addition, the treatment known as CAR T-cell therapy and branded as Yescarta has an eye-opening treatment price of $373,000.
Innovative cancer treatment is quickly developing. Immuno-oncology holds the promise of treating certain forms of cancer, such as prostate cancer and melanoma, by leveraging the power of the human immune system. The human immune system is our built-in natural defense mechanism which eliminates foreign substances, cells and tissues that attack our bodies. The practical application of this practice is just beginning to emerge. Immuno-oncology molecules are designed to help identify cancer cells and destroy them.
Gene replacement therapy sounds invasive and significantly complex to most patients. In actuality, gene replacement is a simple concept: insert a copy of a specific gene into targeted cells. The process introduces genetic material into targeted cells to either produce a protein or to overcompensate for abnormal genes. The process is intended to place a normal gene to replace an abnormal gene, or to provide restoration of a malfunctioning protein.
Researchers have found that a gene cannot just be inserted into a cell and cause a desired reaction. The gene has to be delivered by a carrier called a vector, which also is genetically engineered to perform the transport. In addition, modified viruses become the vector since they can infect the cells. In this case, the viruses have been modified so that they cannot cause a disease in humans. The process starts with a patient’s cells being removed and joined with a vector in the laboratory. The modified vector-containing cells are then either injected or given intravenously into targeted tissue in the body. The goal is to deliver the new gene into the cells that will fight the cancer.
A gene therapy developed by Novartis called Kymriah uses the patient’s own T cells to fight cancer. The one-time treatment is the first therapy based on gene transfer approved by the FDA. The FDA approval is only for patients up to 25 years old who have acute lymphoblastic leukemia either relapsing or refractory. According to The Washington Post, “the therapy’s approval signals a new chapter in treating cancer by mobilizing the body’s own immune system and by using modified genes to fight disease.”
Unique selling proposition
Novartis, the drug manufacturer of the gene therapy formulation for leukemia treatment called Kymriah, has also developed a unique marketing program to move the focus off the therapy’s high price tag. The treatment price, reported to be $475,000 for a one-time treatment, is at the top of the oncology list for most expensive therapy formulations. Novartis’ unique selling proposition being developed is to only charge insurance companies and CMS for reimbursement of the sky-high price tag if the patient goes into remission within one month of receiving the treatment. Novartis calls the plan “outcomes-based pricing” and is waiting for CMS approval.