The Bertarelli Program in Translational Neuroscience and Neuroengineering, a collaborative program between Harvard Medical School and Switzerland’s École Polytechnique Fédérale de Lausanne (EPFL), said today they have awarded a total $3.6 million in grants for five research projects.
Three of the five projects will pursue new methods to diagnose and treat hearing loss. A fourth will focus on the dynamics of brain networks in children with autism, and the fifth, on cell transplantation strategies with the potential to reverse some forms of blindness.
Harvard Medical School and EPFL said the research projects were selected for their scientific quality, the novelty of the approach proposed and the potential for genuine clinical impact. Funding conditions stipulate that each project be an equal collaboration between Harvard and at EPFL.
Details of the winning research programs:
- Developing new methods for diagnostics of hearing loss—Researchers will collaborate to develop new imaging methods for the human inner ear, in a continuation of a previously funded program. While previous studies showed they can image the inner ear in a minimally invasive fashion, researchers will now extend advanced endoscopic two-photon technology to allow subcellular imaging, use the fluorescence of two natural metabolic products to assess the health of the inner ear, and extend initial results to enable imaging of the whole hearing organ.
- New generation of auditory brainstem implants—Continuing a Bertarelli-funded program, researchers will extend their study to long-term experiments in mice to test the safety, durability, and effectiveness of experimental auditory brainstem implants, using high-density, flexible electrodes. They will also extend the flexible electrodes to human tissue. In 2011, the team optimized design and fabrication of the implants, and carried out short-term experiments in mice.
- Gene therapy to treat deafness—Also continuing a 2011 Bertarelli-funded program, researchers will explore new vectors to carry genes into the mechanosensory cells of the inner ear, with the goal of broadening the range of treatable genetic deafness. A longstanding problem for gene therapy for hearing loss, however, is that very few viral vectors will enter these sensory cells. Researchers will also use genome editing technologies to repair specific mutations that cannot be corrected by simple gene replacement.
- Brain networks in children with autism—Researchers will first develop methods to detect and correct for head motion in children and other difficult patients. They will then use Functional magnetic resonance imaging (fMRI) scans of autistic children to test abnormal connectivity between brain regions, which is hypothesized as a cause of autism. Finally they will identify aspects of brain connectivity that correlate with specific types of autism, and ask whether connectivity can be improved with current autism treatments. The experiments will address general problems of fMRI in moving patients, with specific studies of autistic patients and the role of connectivity in this disorder.
- Tissue engineering the macula—Researchers will work to develop cell lines transplantable into the retina to reverse some forms of blindness, as well as to discover drugs that could prevent or reverse retinal degeneration. First, researchers will coax progenitor cells to become cone photoreceptors, responsible for color vision and high-acuity vision. Investigators will also engineer scaffolds capable of supporting the growth and differentiation of these photoreceptors. Finally, the researchers will use such scaffolds as a platform to test potential compounds that can reverse retinal degenerative disorders.
The Bertarelli program is designed to combine basic neuroscience with the technology and problem-solving focus of engineering, with the intent of accelerating the delivery of new treatments for clinical testing. The program began in 2010 and awarded its first grants the following year.
The program is funded by the Bertarelli Foundation, founded in 1998 in memory of Fabio Bertarelli, the father of Ernesto Bertarelli, a graduate of Harvard Business School and member of the Harvard Medical School Board of Fellows, and his sister Dona. The family built Serono over three generations until Merck KGaA acquired the pharmaceutical company in 2007 for $13.3 billion.
The Foundation has focused its activities on two main areas, life sciences and marine conservation. Ernesto and Dona are the Foundation’s co-presidents, while their mother, Maria Iris, and Ernesto’s wife, Kirsty, are also board members.