15 April 2024

A new ‘functional precision medicine’ approach shows signs of improving outcomes for children with relapsed or refractory cancer, doctors in Florida, USA, have reported.

The technique involves genomic profiling and testing drugs on cancer samples in the laboratory, to identify the best drug combination for treatment of the relapsed cancer.

Researcher Dr Diana Azzam, of Florida International University, has developed techniques to enrich and process cancer cells in the laboratory in ways that closely resemble their growth in the body. The matured cells are then exposed to more than 120 FDA-approved drugs, which can be tested in various combinations in a process that lasts about a week.

Reporting in Nature Medicine, Dr Azzam and her fellow doctors report that five of the six patients (83%) who received a treatment guided by the functional precision medicine (FPM) process experienced a response to the treatment.

Patients who received a FPM-guided treatment also had significantly better outcomes compared with patients who received a treatment of the physician’s choice.

Trials have so far involved 25 patients with cancer, of which six had blood cancers.

Dr Azzam and her colleagues have given details of a patient, aged eight, successfully treated for relapsing acute myeloid leukaemia two years ago using drugs guided by the FPM process. The analysis also showed that the patient could safely avoid treatment with idarubicin, which causes a risk of cardiac toxicity. The patient experienced remission 33 days after treatment, and two years later, the patient remains cancer-free.

Dr Azzam said: “Seeing improvement in 83% of patients is incredibly promising. This could be the way we turn cancer into a manageable disease.”

Fellow researcher Professor Tomás Guilarte, dean of the university's Robert Stempel College, said: “The Azzam lab approach gets rid of the guesswork and delivers a list of the most effective drugs that the oncologist can work with. It’s accelerating our understanding of which cancer treatments work best for patients and their specific needs.”


Acanda De La Rocha AM, Berlow NE, Fader M, Coats ER, Saghira C, Espinal PS, Galano J, Khatib Z, Abdella H, Maher OM, Vorontsova Y, Andrade-Feraud CM, Daccache A, Jacome A, Reis V, Holcomb B, Ghurani Y, Rimblas L, Guilarte TR, Hu N, Salyakina D, Azzam DJ. (2024) “Feasibility of Functional Precision Medicine for Guiding Treatment of Relapsed or Refractory Pediatric Cancers.” Nature Medicine, 11 April 2024, doi: 10.1038/s41591-024-02848-4.

Link: https://www.nature.com/articles/s41591-024-02848-4   

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