B-SMART Develops RNA-based Therapy for Neurodegenerative Diseases
Neurodegenerative diseases such as Alzheimer’s affect over 7 million people in Europe – a figure which is expected to double every 20 years as the population ages and thus poses a significant healthcare burden to European society. Despite this fact, few treatments for this group of diseases are available so far. Existing drugs, however, only suppress symptoms or delay further deterioration but do not address the cause of the disease. For Alzheimer’s disease, no drug has been approved in the past five years.
Nanomedical approaches can make a difference, providing new therapeutic options by helping drugs to enter the brain. Therefore, the multinational research project B-SMART sets out to provide an RNA-based therapy perspective for neurodegenerative diseases targeting the direct cause of the disease instead of its symptoms. The project is funded with a total budget of €6 million over the next five years by the European Commission’s current Research Framework Programme Horizon 2020.
“Transferring RNA therapeutics to the brain crossing the blood-cerebrospinal fluid barrier is a unique research endeavor,” said Prof. Raymond Schiffelers from the University Medical Center Utrecht and coordinator of B-SMART. “Finding a novel gateway to transport RNA to the brain will enable the development of causal therapies not only for Alzheimer’s, but for other diseases as well.”
The consortium will work on specific nanocarriers protecting the RNA against enzymatic degradation while delivering it to the human brain. Crossing the brain-cerebrospinal fluid barrier requires specific targeting ligands, which will be based on heavy chain-only nanobodies that are smaller and more stable than conventional antibodies. The modular delivery system will be tested for safety and efficacy using in vitro and in vivo assays. To improve the nanomedicine manufacturability, B-SMART chose an operator-independent and scalable production method based on microfluidics.
In the frame of this ambitious project, Eurice is in charge of the overall project management and coordinates exploitation and communication activities. These will include strategic development, innovation monitoring and implementation of various communication actions such as the setup and maintenance of the project website and the design of dissemination materials.
B-SMART assembles a multi-national team of leading experts from 9 partnering institutions with long-standing experience in their respective fields. The B-SMART partners are involved in numerous exceptional national and international research programmes targeting close communication with the industry throughout all project stages in order to bridge the research-industry gap and enable timely exploitation of the project results.
B-SMART officially kicks off its activities with a first project meeting held in Utrecht on 1 and 2 February 2017.