Precision Medicine Offers Hope for Acute Myeloid Leukemia Patients unsuited for Intensive Chemotherapy
[CITY, STATE] – for over half of individuals diagnosed wiht acute myeloid leukemia (AML), intensive chemotherapy is not a viable option, severely restricting treatment possibilities and diminishing their chances of survival. Though, breakthroughs in precision medicine, particularly the detection of mutations like IDH1, are paving the way for more effective, targeted therapies and improved prognoses.
This progress was underscored during a recent roundtable discussion featuring leading experts in the field. The panel included Pilar Rodríguez Romero, Market Access and Regulatory Affairs Director at Servier España; Adolfo de la Fuente Burguera, head of the Hematology and Hematotherapy Service at MD Anderson Cancer Center-Hospiten in Madrid; and José Manuel Martínez Sesmero, Deputy Medical Director of the Hospital Lozano Blesa in Zaragoza.Rodríguez noted that “the IDH1 mutation is present, according to estimates by Spanish experts, in approximately 10 percent of newly diagnosed patients who are not candidates for intensive chemotherapy.” This presents a significant challenge, as it constitutes what she termed “a rare disease” with “limited” treatment alternatives.
Targeted Therapies: A More Precise Approach
De la Fuente emphasized the importance of moving beyond a one-size-fits-all approach to AML treatment. “What we call AML actually encompasses many subtypes that are differentiated by the underlying mutations,” he explained. Developing treatments specifically targeting each of these mutations “allows us to act more precisely and effectively.” He asserted that “if there is no targeted therapy, unfortunately, we will continue to have to use conventional treatments with less satisfactory results.”
This shift toward precision medicine is reflected in the increasing number of FDA-approved targeted therapies for AML in the U.S. As a notable example, drugs like enasidenib and ivosidenib specifically target IDH1 and IDH2 mutations, respectively, offering patients with these genetic abnormalities a more tailored treatment option.
Streamlining Access to Innovative Therapies
One of the major hurdles in AML treatment is ensuring timely and equitable access to these targeted therapies. International guidelines recommend genetic testing within three to five days to identify appropriate treatments. However, logistical and bureaucratic delays often impede this process.
Martínez Sesmero stressed the need for better coordination among healthcare teams to ensure that patients receive the most effective treatments as quickly as possible. “Offering adequate and sustained access to therapeutic innovations as soon as possible” is the ultimate goal, he said.
The Role of Pharmaceutical Companies
Pharmaceutical companies like Servier are playing a crucial role in advancing precision medicine for AML. Servier’s acquisition of Agios’s oncology division in 2021 brought with it a promising drug for treating AML with the IDH1 mutation. Sence then, the company has been working to develop new alternatives “that provide a longer life expectancy and a lower rate of adverse effects and complications associated with treatment,” according to Rodríguez.
She also highlighted Spain’s significant contribution to the clinical development of this new drug, stating that “Spain is one of the countries that has contributed the most to the clinical development” of the treatment. Servier is committed to providing access to the drug “as soon as possible” and is currently conducting a Phase 3b clinical trial to collect real-world data.
Addressing Access Disparities
Despite advancements in treatment, disparities in access to care remain a significant issue. De la Fuente pointed out that “territorial heterogeneity affects both ‘diagnostic possibilities’ and ‘therapeutic options.'” Eliminating these barriers requires improved coordination between regions, possibly aided by the implementation of new healthcare technology assessment regulations.
Bureaucratic delays also hinder access to new therapies. According to de la Fuente, “there is a considerable period of time” between the finding of a therapy’s benefits, its approval in Europe, and its eventual approval in individual countries.Scientific and professional societies are working to address this issue by advocating for early access programs.
The Importance of Patient-Centric Care
Beyond the clinical aspects of treatment, the experts emphasized the importance of considering the patient’s overall well-being. “We are all aware of the emotional impact of the word leukemia,” said de la Fuente, who also noted that targeted therapies are not only more effective but also safer and more beneficial in terms of quality of life.
Martínez Sesmero stressed the need to “ask people and develop methods of capturing data about what the patient is experiencing in their social, personal, and work context.” This information can then be incorporated into treatment decisions.
Looking to the Future
The future of AML treatment lies in precision medicine, with an increasing number of “aberrations or mutations” in cancer cells that “can be targeted with a molecule or drug,” according to de la Fuente. He envisions a future where “combinations of these types of drugs” are used to achieve even better outcomes.
Martínez Sesmero believes that the “main challenge” moving forward will be “to manage the avalanche of innovations” that are becoming available. For pharmaceutical companies, Rodríguez said, the key challenge is “to maintain the commitment to continue collaborating with Spanish cooperative groups” to support “any initiative that aims to improve the diagnosis and knowledge of the disease.”
New Developments in 2024-25
As the roundtable discussion, there have been several notable developments in AML research and treatment. In the U.S., the FDA has approved several new targeted therapies for AML, including quizartinib, which targets the FLT3 mutation. Additionally,researchers are exploring novel approaches such as CAR-T cell therapy and bispecific antibodies for treating AML.
However, challenges remain in ensuring that all patients have access to these cutting-edge treatments. The cost of targeted therapies can be prohibitive for some individuals, and disparities in access to care based on race, ethnicity, and socioeconomic status persist.
Practical Implications for Patients and Healthcare Providers
Genetic Testing: Patients with AML should undergo comprehensive genetic testing to identify potential targets for therapy,ideally within 3-5 days of diagnosis.
Access to Clinical Trials: Patients should consider participating in clinical trials to gain access to promising new therapies.
Patient Advocacy: Patients and their families should advocate for policies that promote equitable access to AML treatments.
Collaboration: Healthcare providers should collaborate to ensure that patients receive the most appropriate and timely care.
By embracing precision medicine and addressing the challenges of access and affordability,the medical community can continue to make progress in the fight against AML and improve the lives of patients with this devastating disease.
