Objectives
Acute myeloid leukemia is a rare disease of adulthood that, unfortunately, remains difficult to treat in Italy, affecting blood and bone marrow cells. Administering therapy to patients with this disease is made very difficult and complicated by several factors: the first is age, since the disease occurs in elderly individuals, which can in turn prevent treatment options or increase the risk of treatment failure; the second factor is the complexity of the disease itself, which can be effectively addressed in fewer than 50% of cases and which can recur even more aggressively as a relapse, even after therapy and bone marrow transplant.
The research project funded by IFAB fits into this exact context, pursuing a twofold goal: improving the effectiveness of treatments and, as a result, expanding the percentage of treatable patients.
Initial Challenge
At the heart of the project is the need to move beyond the current histopathological, binary approach, which applies a similar treatment to all patients who have developed the disease but which, by its very nature, cannot be universally effective: in statistical terms, we know that for every patient for whom conventional treatments are actually effective, there is another for whom they prove impossible to administer or unsuccessful. Cancer, moreover, is not a standard physical phenomenon, identical in every manifestation, but arises and develops within a context of significant differences depending on the patient involved. These elements of diversity form the starting point of the research, coordinated by Professor Giovanni Roti (University of Parma).
The project proposes a genuine paradigm shift: instead of treating the disease as something universally given, it treats the disease as it manifests at a specific moment in a specific person. Looking ahead, this also implies a major change for the world of pharmacology.
Solution
The pharmaceutical industry only manufactures certain drugs — those most broadly useful for people affected by a known disease (e.g., acute myeloid leukemia). However, there is a range of pharmacological molecules, not yet commercialized, that are potentially more effective for treating specific manifestations of myeloid leukemia that do not respond to standard therapies. This project aims to investigate precisely the therapeutic potential of alternative pharmacological molecules in relation to the individual patient.
How? Through the use of special algorithms that make it possible to test the effects of hundreds of drugs on the cancer cells of a single patient. To grasp the scale and significance of this method, it’s worth noting that treatments for acute myeloid leukemia are currently studied starting from a pool of 15 drugs, while the “Adult Leukemia” project uses hundreds of drugs, exponentially increasing testing capacity, systematizing it, and making it customizable for each individual patient. In other words, drug libraries are used as probes capable of generating far more information about their effects in treating the specific disease.
Benefits
creation of an integrated testing and computing system
Once the method is fully implemented, a key step will be moving from individual variability to intra-individual variability — that is, moving from processing data related to a single person’s therapy to processing data related to the therapy of groups of people who show similar disease characteristics. In essence, the ultimate aim of the “Adult Leukemia” project is not to identify a single therapy profile tailored to the manifestation of a specific disease, but rather to generate, through algorithms, therapy profiles shared by similar disease manifestations.
The main effect of all this will be the identification of treatment pathways capable of being even more effective for an even larger number of people: in numerical terms — quantifiable based on experience gained over the years in other countries in this area — this means being able to treat between 50% and 70% of people who, today, would be considered untreatable.
The role of IFAB
IFAB took part in the project as a funding body, selecting it as part of its investment lines in research, innovation, and technology transfer. IFAB’s contribution consisted of recognizing the project’s scientific and practical value and providing financial support, making it possible to carry out the research activities conducted by the technical partners. This involvement confirms IFAB’s mission as an accelerator of high-impact initiatives for the local area and productive system.
Partners
For more information, contact: projects@ifabfoundation.org
