Fewer than half of people with a subtype of Myeloid/Lymphoid Neoplasms (MLN) are alive one year after diagnosis. This statistic underscores the critical need for new and effective treatments such as personalized medicine. Researchers at the Stanford University School of Medicine have taken a key step, marking the beginning of a new era. As a result of their Phase 2 international clinical trial, FIGHT-203, the FDA has approved pemigatinib, a drug for rare types of blood cancer caused by a change in the FGFR1 gene.
For centuries, blood cancer treatment was primarily based on chemotherapy. Medications in most cases destroyed cells indiscriminately, which often had a severe impact on the patient’s health. In recent decades, targeted therapies have emerged, but the real medical revolution has proven to be the genetically personalized approach.
Study Methodology and Patients
A total of 45 patients participated in the study, each with a rare blood cancer caused by a change in the FGFR1 gene. The patients were divided into two groups: 24 of them had the disease in a chronic phase, which indicates a relatively slow progression. The other 18 patients were in the alkaline, or blast phase, which represents a more aggressive form of the disease.
The treatment regimen was strictly defined. Patients received the drug pemigatinib in one of two possible schemes: once daily with a two-week course followed by a one-week break, or daily without a break.
During the trial, researchers closely monitored side effects, which are expected with this type of treatment. The most frequently reported side effects were an increase in blood phosphate levels and problems with the oral mucosa. It is noteworthy that these side effects were completely controllable with dose adjustments, which further highlights the drug’s safety and good tolerability.
Encouraging Study Results
The study showed that the disease disappeared in almost all patients in the chronic phase (23/24) after an average of 6 weeks of treatment. In patients with the blast phase, the complete response rate was approximately 44%, which is also impressive, although the response lasted for a shorter duration.
In assessing the patients’ condition, the study showed significant results. After 12 months, about 78% of patients in both the chronic and blast phases were able to survive without disease progression, meaning no symptom exacerbation or new tumor formations were observed during this time. The absence of progression was confirmed not only by clinical signs but also by blood, bone marrow, and imaging (CT/MRI/PET) analyses when necessary. At the 24-month mark, this figure was 70%, which again confirms the drug’s long-term effectiveness. Additionally, the overall survival rate was quite high, with 12-month survival at 79% and 24-month survival at 72%. Some patients were successfully transitioned to stem cell transplantation after receiving the drug, which may be the only way to cure this disease.
Broader Perspective and Future
Myeloid/lymphoid neoplasms (MLN) caused by FGFR1 gene changes are difficult-to-treat diseases, whose chronic phase often transitions to the blast phase within a few months. The average survival period is less than two years. Until now, effective treatments were limited, especially in the blast phase.
Pemigatinib acts on the activity of the FGFR1 gene and helps reduce the disease burden in patients who are not yet able to undergo stem cell transplantation. Furthermore, the drug reduces the likelihood of recurrence after transplantation. The drug clearly showed the effectiveness of personalized therapy: it does not destroy cells indiscriminately but precisely targets the genetic cause, which significantly improves survival chances.
Conclusion
This achievement underscores that personalized medicine is no longer a matter of imagination. Pemigatinib is already giving hope to patients who previously had no effective treatment options. This new approach could become the standard for treating rare types of blood cancer and an example of how targeted therapies can improve life expectancy and quality of life.
Stanford Research :
Conger, K. (2025, August 27). Stanford Medicine-led trial leads to FDA approval of drug for rare, deadly blood cancer. Stanford Medicine.
