Project partner InNerMeD InNerMeD Members: Brains for Brain FoundationThe BRAINS FOR BRAIN Foundation (B4B) is an International Task Force composed of outstanding scientists and ‘clinicians’ who are leaders in the neurological field. Together with Biotech companies they aim to facilitate joint efforts to improve the understanding of rare genetic diseases affecting and seriously damaging the brain, in particular in children.

BRAINS FOR BRAIN Foundation aims to develop new and innovative therapeutic strategies to overcome the Blood Brain Barrier, a net made up of capillaries which shields and defends the central nervous system from circulating potential neurotoxic substances. It has a very important filtering and protective function, but unfortunately it also prevents access to the brain by most candidate therapeutic drugs under development for CNS diseases.

BRAINS FOR BRAIN’s discoveries and implementations of knowledge are fundamental not only for children life but also for treating adult’s neurological chronic pathologies as Parkinson’s and Alzheimer’s diseases.

The Foundation is promoting and coordinating research projects focused on the identification of biomarkers for the understanding of pathophysiological processes of the inherited neurodegenerative disorders, in particular Lysosomal Storage Diseases (LSDs). This is a group of more than 50 rare genetic metabolic diseases, characterised by irreversible neurodegenerative effects on the brain and Central Nervous System.

At present B4B involves 60 top ranking Universities from 12 European Countries and America, Australia, and Brazil. The Foundation, in particular, works in close collaboration with international Family Associations and Research Organisations such as the European Brain Council, ORPHANET and the European Study Group for Lysosomal Diseases. B4B has been granted by the European Science Foundation for an Exploratory Workshop on Neurodegenerative Paediatrics Disorders (see www.esf.org) and has organised the workshop on Rare Neurological Diseases of Childhood “We treat the child to treat the Adult” which was held in December 2010 at the European Parliament, Bruxelles. B4B organised the roundtable “Paving the way for a competitive and dynamic EU knowledge economy:  the way forward in rare diseases”  that was held inside the EU Parliament in Brussels, on November 26th, 2013. The Meeting was focused at addressing the research gaps in research in the field of rare paediatric diseases, which also limits the brain drain created through the lack of career opportunities in this field in the EU. To this aim B4B has created a network of Universities and Scientific Societies to start a Doctorate Program aimed at furthering the knowledge on neurometabolic diseases amongst young physicians and scientists in order to maintain excellence in Europe. B4B does also organize Annual International B4B scientific workshops since 2007.

B4B is the organization leading the InNerMeD project and thanks to its meaningful expertise in the management of projects and its long-standing experience and solid clinical competence in the field of child neurological disorders assures the partners coherence and ability to share decisions and put together relevant knowledge and research competences.

In particular, because of its solid experience in developing and fostering networking and collaboration of synergy among research centres and experts and its capacity in strengthening the tie between basic and clinical research in the neurological field, B4B is leader of WP1 and WP2 with the following roles and responsibilities:

WP1 – Coordination of the project

B4B provides the scientific leadership of the overall project assuring the good scientific, administrative, technical coordination and management of the project, closely monitoring integration activities and joint research in order to ensure that the network is operating effectively and to confirm that milestones and deliverables are being achieved (or recommend corrective action if necessary).

WP2 – Dissemination of the project

B4B is responsible for the exploitation and dissemination of new scientific knowledge according to the detailed dissemination plan developed at the beginning of the project which is finalised to maximize the impact of the project and to increase its awareness among the different stakeholders. In particular, B4B is in charge of spreading awareness about objectives and potentiality of the project and disseminating deriving achievements and results within a large scientific and lay community including patient and Health Authorities. Several dissemination strategies will be undertaken to ensure the appropriate propagation of the main topics and in particular: the keywords to be mentioned in the activities; the iNMDs specific characteristics (genetic, disabling, sharing among them diagnostic tools and biomarkers, therapeutic orphan, etc); the low awareness on the current situation of the major stakeholders including health authorities; the need to identify new markers and to develop multinational/multidisciplinary research; the need to inform and empower patients and their families; the development of collaborations among all involved stakeholders, including Financing Bodies.

Key staff involved in the project

MD, PhD. Maurizio Scarpa was a postdoc fellow at the European Molecular Biology Laboratory (EMBL) in Heidelberg, Germany, and at the Howard Hughes Medical Institute, Institute for Molecular Genetics, Baylor College of Medicine in Houston, TX, USA on genetics and gene therapy. He was appointed as consultant on Molecular Biology at the Middlesex Hospital and the London Hospital in London, UK.

He currently coordinates the SHIRE CNS Working Group and is a Member of the HOS Global Executive Committe. He collaborates with Biotech Companies such as Genzyme, Actelion, Biomarin for the development and testing of orphan drugs, for optimising the efficiency and safety of recombinant drugs and for drawing guidelines for the treatment and the management of patients affected by LSDs.

Founder and President of Brains for Brain Foundation, he is also coordinator of all B4B Taskforce activities and represents B4B within the Board of the European Brain Council. He has extensive expertise as a basic scientist in genetics and biotechnology, and as a clinician in the treatment of paediatric rare disorders. He is head of the laboratory of Diagnosis and Therapy of Lysosomal Disorders at the Department of Women and Children Health at the University of Padova. In the same University, he was head of the Center for Rare disorders and Director of the PhD School in Genetics and Molecular Biochemistry  at the Department of Paediatrics and Director of the International Affairs Office.

He is also the Coordinator of the Center for Rare Diseases – IRCCS “Casa Sollievo della Sofferenza”, Scientific Institute and Hospital, San Giovanni Rotondo, Foggia in Italy.

At present, he is the Director of the new Institute for Rare Diseases at the Department of Pediatrics of the Horst Schmidt Klinik, Wiesbaden which he funded together with Christina Lampe. He is a co-founder of the Brains for Brain Clinical Research Institute.

He is member of several international groups: the European Society for Gene Therapy, Society for the study of the inborn Errors of Metabolism, European Study Group for Lysosomal Diseases and the Global Organisation for Lysosomal Diseases. In addition, he is a member of several Scientific Committees concerning paediatric neurodegenerative diseases and other related disorders. He is author and co-author of many peer-reviewed international publications.

MD. Surgeon with extensive experience on Mucopolysaccharidosis (MPS) multisystemic management.

Coauthor of guidelines regarding the diagnosis and management of MPS, she is particularly interested in the craniocervical compression and related symptoms, a major neurological complication present in particular on MPSI, IV and VI patients. Member of several boards and advisory council on MPS she has described a severity score system for the grading of spine-cervical complication in MSPIV and VI.

Christina Lampe’s interests are also devoted to correction of multiplex dysplasia in collaboration with orthopedicians for the creation of customized supports for the management of spine malformations, joint problems and hip dysplasia. She is the medical advisor of German MPS Society as well as  of several patient associations and medical associations in particular in the East European Countries.

Christina Lampe is the Vice-director of the Institute for Rare Diseases of the Horst Schmidt Klinik in Wiesbaden and responsible for adult MPS patients. She is a medical advisor of the Brains For Brain Foundation and a founding member of the Brains for Brain Clinical Research Institute.

PhD. Brains for Brain Foundation’s scientific officer and medical writer is involved in the exploitation and dissemination activities related to neurological rare diseases. She was trained at the Imperial College of London participating in a number of multi-centre research studies. She is particularly interested in rare genetic diseases and brain research, specifically in the group of neurological paediatric diseases that B4B is specialised in, but also in all those conditions characterised by CNS inflammation (e.g. Alzheimer’s, Parkinson’s, multiple sclerosis, tumours, stroke, etc.). Several of her scientific articles have been published in peer reviewed journals.

She is the project assistant manager for the InNerMeD on inherited neurometabolic diseases where she is responsible for data elaboration and communication activities related to rare diseases. Her responsibilities entail ensuring that, wherever possible and relevant, effective communication is undertaken and information is shared promptly with the academic and general public and external stakeholders, including government, industry, patients and family associations, and the media.

PhD. Molecular Biologist with experience in the research field of Lysosomal Storage Disorders (LSDs). She is involved in pathophysiological and pharmacological studies on LSDs, in particular in the bioinformatic analysis of transcriptomic and genomic data. Her research interests also include new therapeutic strategies for the neurological involvement of LSDs, such as adenoviral vectors, liposomes, nanoparticles and neural stem cells. She has published as coauthor in some peer-reviewed journals. In InNerMeD project she will be responsible for data mining and entry and for communication.

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