MiRacle aims to develop a new treatment for head and neck cancers using miRNA with proven anti-tumour activity. The end result will be a drug with a safe drug profile for Investigational New Drug (IND) filing ready for clinical testing.


Despite substantial progress in understanding the cancer-signaling network, effective therapies remain scarce due to insufficient disruption of the pathways of cancer development, drug resistance and drug-induced toxicity. In addition, a growing cancer patient population makes it necessary to bring new therapeutic cancer drugs to the market.

The discovery of microRNAs (miRNAs) has provided new hope for accomplishing this task. Evidence shows that miRNAs play a critical role in cancer and their unique mechanism of action could make them a new class of targeted therapeutics.

In contrast to existing cancer therapies, which mostly target isolated disease pathways and genes, the MiRacle project takes into account the multiple gene involvement of cancer. Therefore, MiRacle will use promising tumour-selective miRNAs, which have shown they can target and kill head and neck cancer tumours but not normal tissues. Moreover their mechanism of action may reduce the incidence of therapeutic resistance by acting on multiple genes. MiRacle will also make sure the treatment reaches its specific goal by developing a new and accurate drug delivery system. Targeted delivery works to reduce any impact a drug may have on other crucial and unaffected organs of the body.

This miRNA-based approach will sit alongside traditional therapeutics both because miRNAs are naturally present in the body and because miRNAs rely on a unique and potentially broader biology.

Head and neck cancer
Head and neck cancers develop in the mucosal linings of the nose, throat, voice box, windpipe, lungs and swallowing tube and contribute to approximately 5% of all cancers in the Western world (1). Most head and neck cancers begin in the squamous cells, which are cells that line the moist surfaces inside the head and neck. Tobacco use, alcohol use, and human papillomavirus infection are important risk factors for head and neck cancers. Typical symptoms include a lump or sore (for example, in the mouth) that does not heal, a sore throat that does not go away, difficulty swallowing, and a change or hoarseness in the voice (2).

The treatment plan for an individual patient depends on a number of factors, including the exact location of the tumour, the stage of the cancer, and the person’s age and general health. Treatment can include surgery, radiation therapy, chemotherapy, targeted therapy, or a combination of treatments. Rehabilitation and regular follow-up care are important parts of treatment for patients as recurrences and secondary cancer development is a real risk which result in 5-years survival rates of less than 60% of patients (2). The development of new anti-cancer agents to improve outcome is therefore urgently needed.

MiRacle will be the first project to take a miRNA-based drug, focusing on head and neck cancers, which will be delivered directly to the tumour.


The overall objective of this project is to develop a new treatment for head and neck cancers using miRNA with proven anti-tumour activity to create a safe drug profile to prepare for Investigational New Drug (IND) filing ready for clinical testing.

More specifically, MiRacle will attempt to advance a tumor-killing miRNA formulation towards the clinic by combining two innovative technologies

  1. therapeutic miRNA and
  2. head and neck tumour specific targeted drug delivery.

To achieve this aim, the project has put together a consortium with
  • expertise in the therapeutic application of miRNA and knowledge on the synthesis of biochemical active miRNA,
  • expertise in a drug formulation to deliver the therapeutic miRNA into humans and understanding the synthesis of complex drug formulations, plus
  • in-depth knowledge of the therapeutic indication in head and neck cancer and skillfulness in toxicity tests that are required for RNA based drug registration.

The final outcome of the project will be a safe and tested miRNA based treatment of head and neck cancers that can be taken to the next level, clinical testing.


  1. Kamangar JCO 2006
  2. www.cancer.gov



“This project has received funding from the European Union’s Seventh Framework Programme for research, technological development and demonstration under grant agreement no 606486”.

Project title: “MiRacle — Novel tumor-selective lethal miRNAs for the treatment of head and neck cancer”.

FP7-SME-2013 - Research for the benefit of specific groups