Personalized liposome-based small inhibitory RNA therapy for acute myeloid leukemia (OBS/AML)

ACELLMED Sp. z o.o. and the Silesian Medical Technology Park Kardio-Med Silesia have established cooperation for the implementation of a project co-financed by the European Regional Development Fund under the Regional Operational Programme of the Silesian Voivodeship for 2014-2020 from Priority Axis I. Modern Economy Measure 1.2 Research, Development and Innovation in Enterprises.
 
The project is named: “Personalized liposome-based small inhibitory RNA therapy for acute myeloid leukemia (OBS/AML).”
 
PROJECT OBJECTIVE – to develop R&D activity and competitiveness through the implementation of R&D work that will result in a drug that inhibits leukemic cell proliferation used during oncology therapy. The goal will be achieved by June 2023.
 
The main scientific objective is to develop a targeted, liposome form of miRNA inhibiting the proliferation of cancer cells characterized by selectivity of action, low non-specific, cytotoxicity and high efficiency of nucleic acid delivery to cancer cells, which alone or in combination with standard used cytostatics will significantly improve the effectiveness of treatment. Acellmed and the Silesian Medical Technology Park Kardio-Med Silesia aim to develop a drug that can be used in oncological treatment.
 
Project objectives:
  • Studies on the verification of selected miRNAs used as an inhibitory factor for OBS-specific blast proliferation and on the synthesis of oligonucleotide aptamers.
  • The therapeutic activity and specificity of the prepared preparations will be verified on several cell lines (leukemic and control), the specificity of the activity will be determined in flow cytometer by fluorescence labeling and survival analysis.
  • Verification of therapeutic activity and specificity of action of the prepared formulations on leukemic cells obtained from (6-8) patients with different variants of OBS. Cells will be cultured in special medium along with liposome preparations at different concentrations.
  • Preparation of at least 100 ml of each preparation targeting the respective surface markers (CD33, CD34, CD117) for animal testing.
  • Obtaining a formulation with confirmed lack of non-specific toxicity – tests on healthy animals. Confirmation of the therapeutic activity and specificity of the drug preparations – in vivo studies on experimental animals with implanted leukemia derived cell lines. Therapeutic activity and specificity of action of drug preparations will be confirmed on one selected line obtained from one of the above mentioned patients.
Delivery time: 18 months
 
Project value:
  • Eligible expenditure: PLN 4,076,744.70
  • Requested funding: PLN 3,465,232.99