Today there is no regenerative therapy available for treatment of complete spinal cord injuries (SCI). BioArctic Neuroscience develops a novel treatment of complete SCI. The method is based on the regeneration of functional nerves in the injured spinal cord. Nerve grafts are transplanted into the injured spinal cord, by using a biodegradable device, loaded with a growth factor and nerve grafts (these three components are abbreviated SC0806). Efficacy and safety of the treatment has been demonstrated in an in vivo model of SCI. The therapy has been designated Orphan Drug status in Europe and in USA. Ethical approval to commence into clinical trial in Sweden was recently granted. The proposed project is the first clinical trial of the treatment of complete spinal cord injury. Following surgery at the Karolinska University Hospital, patients will receive rehabilitation in their home countries, with state of the art robotics equipment, at clinics in Denmark, Finland and Sweden. This clinical study will be the first in-human trial with this new treatment concept. Several interim analyses are included in the study design to secure the safety of the patients. This is a single dose study in up to 3 sequences (A, B and C) in 27 subjects with complete traumatic SCI. In the first sequence (A), 6 subjects will be randomized to a surgical procedure where SC0806 will be implanted at the spinal cord, and receive robotics assisted walking training for 18 months, and 3 subjects will be randomized to specific walking training only. For safety reasons, the first three subjects will be operated on and implanted with at least 1 month in between. If efficacy is demonstrated, control subjects from (A) and (B) and (C) will later be given the opportunity to receive treatment with SC0806.The proposed project spans a period of 42 months and will start nov 2014 with screening, inclusion and treatment of patients in Sweden.

Neurodegenerative diseases are one of the most important contributors to morbidity and mortality in the elderly. In Europe, over 14 million people are currently living with dementia, at a cost of over 400 billion EUR annually. Comorbidities with these conditions are frequent and a major obstacle to optimal diagnosis and management. Recent advances in diagnostic technologies and the advent of disease-modifying therapies (DMT) for Alzheimer’s disease (AD), the most common aetiology of dementia, heralds the beginning of precision medicine in this disease area. PROMINENT will develop a digital platform for precision medicine that will remove barriers that currently exists for leveraging these technological advancements in the routine care of patients with neurodegenerative disorders and co-morbidities. The platform gives clinicians access to prediction models leveraging multimodal diagnostic data automatically derived from multiple sources (imaging repositories, medical records, mobile devices), helping them choose optimal care pathways and improving diagnostic precision. It will provide personalized, relevant and meaningful information on diagnosis and prognosis in a format understandable by patients and care partners. Further, it will support the introduction of new health technologies such as DMT for AD, by ensuring adherence to appropriate use guidelines and facilitating the prospective collection of data on real-world usage, safety and effectiveness. The expected impact of the project is to increase diagnostic accuracy and optimized use of existing and new treatment options. It will empower patients and caregivers by engaging them in more person-centric health care decisions, leading to improved adherence and patient experience. Ultimately this is expected to lead to cost-effective care, improved health outcomes and quality of life.

Too often, physicians have to rely on a trial and error strategy until the most effective treatment for an individual patient is identified. This leads to a critical loss of time, making the healthcare system ineffective and expensive. In respect to drug development, a high failure rate is apparent at all stages of development and ways to reduce this have high priority for the pharmaceutical industry. To this end, drug development and medicinal treatment need to be more personalized and molecular imaging techniques, in particular Positron Emission Tomography (PET) imaging, can be used to achieve this by 1) evaluating the effectiveness of new treatments emerging from the pharmaceutical industry, 2) improving the ability to diagnose diseases, and 3) aid tailoring the treatment based on individual patient pathology. A limiting factor to develop new effective PET imaging probes is the insufficient number of radiopharmaceutical scientists presently available. Our consortium has assembled 6 radiopharmaceutical frontline academic and 10 non-academic partners to address this problem and train the next generation of radiopharmaceutical scientists. Our training will be focused on an unmet scientific need for Parkinson’s Disease (PD). Fibril formation of protein α-synuclein is thought to be the hallmark for PD, but presently, there are no validated PET tracers available for visualization of pathogenic aggregates of this protein. Our scientific goal is to develop such tracer. A successful tracer will have a strong impact on PD-diagnosis and facilitate the development of effective disease modifying treatments. To reach our goals, it is necessary to train our early stage researchers in a highly interdisciplinary fashion spanning from chemistry, over biology, GMP and pharmacy to nuclear medicine. Furthermore, this consortium will train entrepreneurial skills and facilitate start up new enterprises in the current favorable radioisotope market situation.

Parkinson’s disease (PD) is one of the most prevalent neurodegenerative disorders and a disease with a significant unmet medical need. World-wide, more than 4.6 million individuals over the age of 50 suffer from PD and it is estimated that the number will more than double by 2030. The economic impact of the disease is enormous and the estimated annual European cost of PD is 14 billion euros. At present, only symptomatic therapies are available for PD and there is a huge unmet need for treatments that also slow or halt disease progression. Improved patient outcomes are achieved by early diagnosis and disease modifying treatment. BioArctic Neuroscience is developing a PD biomarker assay, with the potential of becoming the first biochemical biomarker assay that can reflect the underlying pathophysiology of disease. The assay is based on CSF measurements of toxic oligomer/protofibril forms of alpha-synuclein. In this project we will: • Conduct biochemical biomarker assay development for PD • Conduct a small pilot study on clinical cohorts including patients with PD and healthy controls • Evaluate the business opportunities and further development path for PD biomarkers Today, there are no reliable biochemical biomarkers for PD and the disease is often difficult to diagnose. The differential diagnosis of PD is based on clinical features and the golden standard still remains neuropathological confirmation. Misclassification, especially in early PD, occurs frequently. High sensitivity and specificity can only be obtained at specialized centres, and after several years of follow-up. To be able to modify or halt the disease progression it is beneficial to initiate a disease modifying treatment at an earlier stage than what is possible today. The clinical validation of a sensitive and specific biomarker that also could mirror the treatment effect would make an enormous advantage and need to be developed in parallel with the development of new disease modifying therapeutics.