Today's common cancer treatments are notoriously intense and physically taxing, with varying effectiveness. Fortunately, advancements in research and technology have made possible a new treatment method that is highly targeted providing enhanced efficacy: CAR T-cell therapy.
One of medicine's youngest and most rapidly advancing fields, CAR T-cell therapy has grown from a single biopharmaceutical introduced in 2017 to now approximately ten approved therapies on the market, numerous in the FDA approval pipeline, and untold numbers in development. Upon closer inspection, the reason for this buzz is apparent— unlike conventional cancer treatments, which kill cells indiscriminately, CAR T-cell therapy can explicitly target cancerous cells while leaving healthy tissues untouched. Because of this, it contains none of the costs inherent in other methods. Instead, it often outperforms its counterparts in efficacy while requiring far less from the patient, both physically and mentally.
Yet, because this therapy is so new, several obstacles prevent it from reaching those who need it most. The production is complex, time-consuming, and extremely expensive— from $350k to over $3M for a single treatment. Meanwhile, because this production occurs in a remote, centralized lab location, production slots are vastly limited, with a backlog of patients waiting on therapies. Per surveys of US CAR T-cell treatment centers, at least 20 patients are on a waiting list at any given time, vying for slots that only become available at a rate of one per month. To truly usher in the next generation of cancer treatment, CAR T-cell therapy must be democratized, made more accessible and affordable to reach a larger population and begin to influence real change. This includes improving production capabilities, increasing turn-around time, and lowering the cost per therapy. This is the first of a 5-part blog series that will road-map this democratized approach.
To truly grasp CAR T-cell therapy's value and potential impact, we must first review the general treatment methods most commonly employed today. Here, it is important to note that these treatment methods are not mutually exclusive but are often combined to achieve a more significant effect.
When cancer growth is localized (i.e., a tumor) in an accessible area of the body and may be removed without significant impact on general well-being, surgery is the preferred treatment method.
Allowing for targeted, complete removal of malignant tissues, surgical treatment methods yield immediate results and often are successful enough that only a single operation is required. Surgery carries an inherent risk of complications due to its invasive nature. It must be performed with great precision to have maximum effect, as leaving any cancerous cells in the surgical site can result in later relapse.
Radiation is a non-invasive treatment method when cancer is localized but cannot be accessed via surgical means.
Though less given to complications than surgical options, radiation is both physically and mentally taxing; while requiring numerous treatment sessions, it also utilizes a non-targeted approach that, in the process of destroying malignant cells, causes significant damage to healthy tissues.
Used to treat systemic cancer types, chemotherapy is currently the only widely available method of addressing cancer that has spread throughout the body, infecting blood, lymph nodes, and other nonspecific areas.
This type of treatment is punishing on patients as well as their caregivers. Like radiation, chemotherapy involves a non-targeted approach that kills healthy cells alongside cancerous ones; yet, because it is used to treat non-localized cancers, chemotherapy is responsible for several serious side effects. As white blood cells are destroyed, patients experience compromised immune systems and are at a much higher risk of infection. They also typically suffer anemia, nausea, fatigue, weight loss, hair loss, and a reduction in platelets, making it difficult for blood to clot. Chemotherapy can even cause fertility issues.
Also known as bone marrow transplants, stem cell transplants are another method of treating systemic cancers, often paired with chemotherapy, with the latter applied prior to transplant.
Administered intravenously, these transplants may be derived from one of two sources: the patient or a donor. The former is known as an autologous transplant, in which stem cells are harvested from the patient's blood via an apheresis machine before chemotherapy. The latter, which relies on donor stem cells (typically harvested from a family member), is known as an allogeneic transplant. In either case, harvested cells are injected into the bloodstream and carried into the bone marrow, where they are then able to kickstart the production of healthy blood cells.
Because stem cell transplants restore the body's natural ability to produce white blood cells and other key cell types, they are invaluable tools for repairing patients' immune systems fresh from chemotherapy. In some cases, allogeneic transplants can even bolster the immune response past its original capability and provide an additional layer of protection against relapse, as donor cells are often more capable to recognize and attack cancerous cells than the patient's own.
Although stem cell transplants can be difficult to acquire and even more so to undergo. In the case of allogeneic transplants, availability is severely limited by a lack of suitable donors, especially for patients of specific ethnic backgrounds or with rare tissue types. Meanwhile, those fortunate enough to be matched with appropriate donors must be carefully monitored for signs of Graft-vs-Host Disease (GVHD), a syndrome in which donor immune cells attack the recipient's healthy tissues, causing various complications ranging from mild to life-threatening. Additionally, patients can expect a long and difficult recovery period, no matter the source of transplanted cells. Recovering from a transplant of either sort can take months, if not years, and typically includes extended bouts of weakness, fatigue, and other disconcerting side effects significantly impairing quality of life.
In subsequent blogs within this series, we will look at the fundamentals of CAR T-cell therapy, discover precisely how it outperforms conventional methods, and discuss the strategies that must be employed to turn this currently exclusive, bespoke treatment into a solution readily available for the majority of patients in need.
To hear more about the democratization of CAR T-cell therapy and how it is operationalized, tune in to the next installment of the MIDI Innovation Vault!