As we look towards the development of the next generation of drugs and therapies, it starts to beg the question of whether we are creating the next “cure” or the next “treatment.” A cure would require actively restoring the patient to full health with no disease left whereas a treatment shows an improvement in health but the underlying disease still remains. Both cures and treatments are obviously important but the goal of actively curing a disease remains the ultimate outcome we strive to achieve. However to develop a cure is highly difficult thereby making treatments just as important to develop. A treatment could be a “bandage” on a problem while a cure is developed or implemented.
For example, in the case of a heart attack or a myocardial infarct, the blood supply is blocked creating tissue death in the heart. This tissue death is irreversible and results in lowered pumping function in the heart. To treat a heart attack, surgeons look to re-establish the blocked blood flow in the damaged area of the heart to stop the tissue death from being catastrophic. This blood flow is re-established through a bypass surgery. This “treatment” helps to improve the condition of the patient but does not “cure” the permanently damaged tissue. The only cure for this pathology currently being used in the clinic is a total heart transplant which is not a suitable option for many who suffer a heart attack. Bypass surgery, as a “treatment,” does amazing things and provides patients with a better outlook on life going forward but a “cure” would actively fix the underlying problem at hand.
Instead of a discrete distinction between treatment and cure, could there be a treatment that turns into a cure over time? This is the expectation of some of the newer generation therapeutics that are being engineered against specific cellular targets. Furthermore, this type of approach could look to leverage the body’s natural ability to cure itself to treat and cure pathologies that are currently affecting large numbers of patients, such as cancer. One such approach that is looking to create a treatment that then turns into a long-term cure can be found in CAR T cell therapy. Briefly, CAR T cells use either the patient’s own T cells or healthy donor t cells and engineers them to specifically target the tumor cells. The CAR T cells are generated by inserting a gene that is responsible for specifically targeting the tumor cell so that the CAR T cell can kill the tumor. CAR T cell therapy has been successful in its ability to treat certain cancers but its long term efficacy, a key step towards becoming a cure, is yet to be determined. There have been some instances where patients who received the treatment were effectively cured from their cancer a decade after treatment. In fact, two of the patients who were recipients of one of the original CAR T cell therapies responded well to the therapy were found to have still had CAR T cells lingering in their bloodstreams, effectively making the treatment into a cure for their blood cancers. Because these patients were in the earliest cohort undergoing the treatment, there is a lot of promise in these results. However, only long term monitoring of the treated patients and an increase in the number of recipient patients will allow us to be better able to call it a cure. These are still early results but showcases where drug development could be headed over time.
With advances in immunotherapy, regenerative medicine, gene therapy, cell therapy and other therapeutic interventions that look to harness the body to treat itself and become a living drug, there are a lot of exciting avenues where these “treatments” could develop into long term “cures” for many debilitating diseases. As these new drugs are developed and tested, we are closer to living in a reality where more treatments may become cures.
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