From the January/February 2018 issue of HealthCare Business News magazine

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.

Clinical trial success
Marie Miceli, an active real estate administrative professional in St. Louis, received the gift of a lifetime by being offered a chance to participate in an early clinical trial as a last-resort effort in her fight against non-Hodgkin’s lymphoma. Previous chemo and bone-marrow transplant treatments were ineffective. In 2015, after the failed treatments, doctors told Miceli to get her life in order. Nothing else could be done. Participating in the trial was considered a last grab at hope, but innovative treatment focused on re-programming the patient’s own cells worked when conventional cancer treatment failed.

January/February 2018 Magazine

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