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Tumor therapy by blocking the heme synthesis-export axis

5-Aminolevulinic AcidcancerHemeRNA interferenceTumorTumore


Heme is crucial in processes that provide energy to cells, including cancer cells. The invention consists of a strategy for the treatment of tumors, based on blocking the “heme synthesis-export” system, in order to interfere with the energetic remodelling of cancer cells necessary for their survival/proliferation/motility and interaction with the microenvironment.

Technical features

The invention is based on a blocker of the “heme synthesis-export” system to counteract the development of the tumor mass and potentially of vessels/nerves that feed it. The blocker is represented by an inhibitor, already exploited in clinic with other use, of the heme biosynthetic enzyme ALAS1, or by a negative regulator of ALAS1 or of the heme exporter FLVCR1a. The approach does not imply the use of surgery and can, therefore, be useful also in cases diagnosed late, in elderly patients or with other comorbidities. In addition, unlike radiation therapy, cryotherapy and chemotherapy, it is potentially selective for the tumor, since the targeted process is increased in cancer cells, making them more dependent on it than normal cells. Finally, the approach does not depend on the presence/absence of specific mutations, as often occurs in case of targeted therapies; therefore, it is potentially applicable on multiple categories of patients and on different types of cancer.

Possible Applications

  • Cancer therapy in various types of cancer;
  • Cancer therapy in cancer patients who cannot benefit from surgery (elderly, patients with comorbidities, late diagnosed patients);
  • Anti-angiogenic therapy in cancer patients;
  • Tumor-associated pain therapy.


  • Unified approach against tumor and tumor-associated vessels / nerves;
  • More options for the blocker: ALA or shRNA, but potentially also siRNA, ASO, molecules, peptides;
  • Selective for the tumor;
  • Administrable without the use of specific devices; Applicable to different categories of cancer patients.