Personalized medicine is beginning to reshape this approach by taking into account both the biological characteristics of a disease and the individual patient’s profile.
In this article, László Pálinkás, country medical affairs head at Novartis Hungary, presents four therapeutic approaches that seek to answer the same question: How can a disease be treated more precisely and effectively while placing the least possible burden on the patient?
Personalized medicine recognizes that patients with the same diagnosis may respond differently to treatment. If patient-specific data reveal the exact genetic or molecular mechanism underlying a disease, physicians can select the therapy best suited to that particular condition.
The continued development of personalized medicine depends on accurate diagnostics capable of identifying a patient’s genetic and molecular characteristics, together with innovative therapies that can make effective use of this information. Significant progress has been made in both areas in recent years. Like all medical treatments, these innovative therapies may be associated with side effects and require appropriate monitoring by specialized healthcare teams to support their safe use. Novartis has contributed to this transformation globally, while Novartis Hungary supports the local implementation of these innovations.
“It aims to address the underlying cause, not only the symptoms.” This is how Pálinkás describes the essence of gene therapy. Unlike conventional medicines, which act on abnormal biological processes, gene therapy can deliver a functional copy of a gene into cells where its absence or malfunction has caused disease.
Many gene therapies target rare inherited diseases caused by a defect in a single gene. One example is spinal muscular atrophy (SMA), in which the SMN1 (Survival Motor Neuron 1) gene is unable to produce the protein essential for the survival of motor neurons.
“Novartis’ SMA gene therapy delivers a functional copy of the gene into motor neurons with the help of an adeno-associated virus (AAV),” explains Pálinkás. “The virus acts as a delivery vehicle, entering the target cell and transferring the new genetic information.”
Available long-term data suggest sustained benefit, although ongoing follow-up remains important. However, Pálinkás stresses that gene therapy is not a universal solution. It can only be applied to diseases in which the underlying genetic abnormality is clearly identified.
While gene therapy aims to correct or replace a defective gene, cell therapy follows a different approach by harnessing the patient’s own immune system to fight disease.
Cell therapy begins by collecting the patient’s T cells, which are genetically modified to recognize and selectively attack cancer cells before being returned to the patient by infusion, explains Pálinkás.
Unlike conventional medicines based on small molecules or antibodies, CAR-T therapy uses living, modified cells. The Novartis-developed CAR-T therapy combines genetic engineering, personalized manufacturing and the use of living cells as the therapeutic product.
The treatment is primarily used for certain hematological malignancies, including some forms of leukemia and lymphoma, offering a new option for patients whose previous therapies have not produced the desired results. Novartis is also investigating CAR-T therapy in severe immunological diseases through ongoing clinical studies.
The previous examples show that gene and cell therapies are currently used primarily for specific diseases. By contrast, targeted therapies have become an established part of oncology and illustrate how cancer treatment has evolved over recent decades.
Rather than broadly destroying rapidly dividing cells, targeted therapies identify and block the molecular abnormalities that drive tumor growth. This approach depends on accurate diagnostics to determine which therapy is most likely to benefit an individual patient. The concept of targeted therapy continues to evolve. In chronic myeloid leukemia (CML), for example, most traditional therapies target one region of the BCR::ABL1 protein. Newer therapies were designed to target a different region of the same protein and this approach has shown the potential to improve treatment outcomes while maintaining a favorable safety profile.
As Pálinkás points out, targeted therapies in oncology may also be used after surgery (adjuvant treatment) to reduce the risk of recurrence by acting against microscopic residual disease that cannot be detected by imaging. In certain types of breast cancer, for example, adjuvant targeted therapies have become an important part of treatment strategies designed to lower the likelihood of the disease returning.
According to Pálinkás, one of the most important achievements of innovative therapies is that certain cancers can now be controlled over the long term. In melanoma, for example, modern therapies have substantially improved long-term outcomes for some patients. At the same time, he emphasizes that the goals of modern oncology vary from complete recovery to long-term disease control or preserving quality of life based on the type and stage of the disease. This reflects the central principle of personalized medicine: selecting the treatment strategy that offers each patient the greatest chance of success based on the biological characteristics of their disease.
Alongside gene, cell and targeted therapies, radioligand therapy represents another important pillar of personalized medicine, combining diagnostic imaging and treatment within a single therapeutic process known as theranostics.
The therapy uses a molecule, known as a ligand, that recognizes a target protein on tumor cells. First linked to a diagnostic radioactive isotope, it identifies patients suitable for treatment. The ligand is then paired with a therapeutic isotope that delivers radiation directly to tumor cells while reducing exposure to healthy tissue.
Because the targeting molecule can recognize the relevant protein wherever it is present, the therapy may reach small metastatic lesions that are difficult to detect using conventional imaging.
Radioligand therapy is currently available in Hungary primarily for advanced prostate cancer and certain neuroendocrine tumors, while research is exploring its use in other indications. Pálinkás emphasizes that successful implementation depends not only on scientific innovation but also on multidisciplinary collaboration and the specialized infrastructure required to deliver these therapies safely.
FA-11764119. Dated 25.08.2026.
This article was first published in the Budapest Business Journal print issue of September 4, 2026.
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