DNA: a revolution in the service of pharmacology
World DNA (Deoxyribonucleic Acid) Day is celebrated annually on April 25, which coincides with the date when the article “Molecular Structure of Nucleic Acids” was published in the prestigious journal Nature in 1953; structure of deoxyribonucleic acid.”
The authors, James Watson and Francis Crick, discovered the double helix structure of DNA, paving the way for the study of the human genome and the development of new molecular biology techniques that revolutionized the therapeutic field.
One of the first steps was made by using restriction enzymes to isolate and clone specific genes, allowing the identification of specific regions of the genome associated with the presence of a particular disease.
These advances formed the conceptual framework that led, several decades later, to the development of drugs that can transfer new genetic material to an individual for therapeutic purposes—a type of breakthrough therapy known as gene therapy.
The General Council of Colleges of Pharmacy wishes to join in celebrating World DNA Day in recognition of the major advances that have occurred in both the research and application of gene therapy products.
Pharmacist fame
Typically, the dispensing and administration of gene therapy drugs is limited to the hospital, so the participation of hospital pharmacists in multidisciplinary teams evaluating these drugs before their use gives these professionals a critical role.
The community pharmacist also plays an important role in healthcare, as many patients receiving this type of advanced therapy medication will be discharged to go about their daily lives in an outpatient setting and are likely to make frequent visits to the community pharmacy to get their medications off their medication. for other concomitant diseases or other health problems. For this reason, care coordination continues to be an important factor in ensuring the quality of care offered to patients undergoing gene therapy treatment, and knowledge of the benefit-risk profile of these relatively “new” treatments must be continually updated. “.
In this sense, it should be taken into account that some gene therapies, especially CAR-T therapies (such as tisagen, lecleucel, axicabtagene ciloleucel or brexucabtagene autoleucel, which are three of the five gene therapies available in Spain), are associated with a pronounced toxicity profile. emphasizing the risk of cytokine release syndrome, which occurs due to activation of the immune system with an intense release of interleukins, such as IL-1, IL-2 or IL-6 and interferon gamma, and which is characterized by the appearance of fever, tachycardia, arterial hypotension, shortness of breath, in a number cases of renal and liver failure, which can lead to the death of the patient. For this reason, patients receiving these treatments must remain in the hospital for at least 10 days after taking the drug, remaining under the supervision of a clinical team that includes hematologists, intensivists, neurologists, and hospital pharmacists, among others. It is up to professionals to recognize and address these toxicities early.
These patients may also benefit from collaboration between different levels of care, including the community pharmacy. As part of the risk management program, the patient receives a card that identifies him or her as a recipient of the CAR-T drug. This will allow any other healthcare professional to better identify a sudden side effect and also facilitate immediate contact with their specialists.
Thus, it has been proposed, for example, to include alerts in the prescribing and dispensing systems associated with these patients throughout the health system (primary and specialist care, community pharmacy) to allow for double pre-screening. the use of certain drugs, such as corticosteroids, which are contraindicated in these patients.
In addition to healthcare functions, it is worth highlighting the role of pharmacists in research into this type of advanced treatment. As healthcare professionals and medical experts, pharmacists can participate at any level in clinical trials of new pharmacotherapy options from a particularly integrative perspective.
At the basic research level, pharmacists working in teaching and research at the Academy are trained and familiar with the identification and isolation of active ingredients from various sources, chemical synthesis, analytical chemistry, biotechnology and pharmacological research at various scales. In the field of industrial and herbal pharmacy, a specialization characteristic of this profession, pharmacists contribute to the development of new forms of management, the improvement and development of pharmaceutical forms and the optimization of purchasing processes.
In the field of clinical research, specialty pharmacists are often involved in experimental developments from the hospital pharmacy, as the principal investigator typically delegates tasks related to the management of study medications, and pharmacists are responsible for administration, storage, and preparation. the same thing.
Their tasks include recording all movements of the product under study, its receipt, issue, return or waste management, including its possible destruction; The pharmacist also usually keeps a record of the medications.
On the other hand, in the pharmaceutical industry, pharmacists practice in clinical research departments, which they usually enter after specialized postgraduate training. The department, commonly called clinical operations, is responsible for initiating and designing the study, and there you can find pharmacists in coordinating positions and mostly as observers.
Pharmacists also work in the data management department, primarily performing coordination tasks, responsible for ensuring the completeness and accuracy of study data(1); They also participate in the development of the data collection log and database, submit requests for permission, and are responsible for harmonizing medical and scientific terminology so that all entries in the database are the same for the same concept (eg, headache). , migraine, cephalalgia, etc.). Another field of activity of a pharmacist in the industry occurs in medical departments, where they are involved in the development of protocols, preparation of reports, amendments and any other documents related to clinical studies of a technical nature.
In short, the professional profile of a pharmacist is one of specialization in different areas, although in all of them, knowledge of medicines is the essence of the profession. Therefore, the participation of pharmacists in the processes associated with the development, distribution and dispensing of medicines, as well as in monitoring the results of pharmacotherapy, helps to ensure quality care for patients, which is especially important in this case. gene therapy drugs because of the many aspects of innovation they involve.