Edition of 06 October 2017

Palliative Care Needs of Individuals with Rare Advanced Diseases and Their Family Caregivers - R21 (NIH, USA)

This funding opportunity announcement (FOA) seeks to expand knowledge and increase the evidence base for palliative care (PC) in advanced rare diseases, including rare cancers, and to improve physical and psychosocial well-being and quality of life among seriously ill individuals and their family caregivers.

For further details

DEADLINE: October 16, 2017

Call ‘Preclinical Research in rare diseases: translational steps in large animals’ Development of experimental models and evaluation of therapeutic strategies

In the field of rare diseases, understanding the physiopathological mechanisms and moreover, the evaluation of innovative therapeutic strategies, rely for a large part on animal models, particularly rodents. The translational research represents a link between exploratory steps of proofs of principle to clinical application in human. In some cases, the use of larger animal models is necessary to confirm the relevance of a concept or to demonstrate the feasibility, the efficiency and the safety of a therapeutic approach in conditions that mimic as closely as possible the human situation. The call for proposals launched by the French Foundation for rare diseases aims to directly support intermediary key steps towards clinical development for patients.

To complete and submit an application form, please access the portal “Applicant portal”.

For further details

DEADLINE for pre-proposals: October 24, 2017 (5:00 pm) 

Clinical Observational (CO) Studies in Musculoskeletal, Rheumatic, and Skin Diseases (R01) (NIAMS, USA)

This Funding Opportunity Announcement (FOA) is to encourage Research Project Grant (R01) applications to pursue clinical observational (CO) studies to obtain data necessary for designing clinical trials for musculoskeletal, rheumatic, or skin diseases or conditions. Research data from observational cohort studies can enhance clinical trial design by providing essential information about disease symptoms, stages and timing of disease progression, comorbid conditions, availability of potential clinical trial participants, and outcomes that are important to patients. CO studies also can facilitate efforts to develop and/or validate objective biomarkers or subjective outcome measures for use in a future trial or trials. Applicants to this FOA are encouraged to propose studies that address significant obstacles or questions in the design of a clinical trial, such as determining the appropriate primary or secondary outcome measures or identifying the stages of disease during which patients are most likely to respond to an intervention. Only observational studies will be supported by this FOA.

For further details

DEADLINE: November 1, 2017

Palliative Care Needs of Individuals with Rare Advanced Diseases and Their Family Caregivers - R01 (NIH, USA)

This funding opportunity announcement (FOA) seeks to expand knowledge and increase the evidence base for palliative care (PC) in advanced rare diseases, including rare cancers, and to improve physical and psychosocial well-being and quality of life among seriously ill individuals and their family caregivers.

Research projects submitted to this FOA should focus on palliative and end-of-life care for individuals with advanced, rare diseases and their family caregivers. Studies may consider changing needs that occur across time points and disease progression, as well as ways in which EOLPC and planning can adapt to meet these changing needs. Childhood cancers are not of interest for the purposes of this FOA.

Research projects of interest include but are not limited to those that:

• Determine the unique EOLPC needs and preferences (including decision-making) of individuals with advanced, rare diseases and their family caregivers.
• Contribute seminal data on how needs and preferences of individuals with rare diseases and their family caregivers change across stages of an illness, to provide evidence for future intervention-based studies.
• Design, test, evaluate, and/or compare EOLPC interventions or models of care to support individuals living with advanced, rare diseases.
• Design, test, and evaluate, and/or compare EOLPC interventions or models of care to support family caregivers of those living with advanced, rare diseases.
• To establish key indicators of an appropriate measurements for quality outcomes in individuals with advanced, rare diseases and their family caregivers.
• Identify transition points at the advanced stages of illness which result in a change in palliative or hospice care needs and develop evidence-based interventions/models of care support during these critical transition points.
• Foster new research to determine the readiness of individuals with advanced, rare diseases and their families to engage in integrated palliative care.
• Develop and test innovative technologies for use in palliative and/or hospice care models for individuals with rare diseases.

For further details

DEADLINE: November 5, 2017

Neuronal Ceroid Lipofuscinosis (NCL) Foundation

The NCL Foundation aims to help find a cure for the most prevalent form of childhood dementia caused by mutations in the CLN3 gene. Together with the existing NCL research community, our goal is to support and move forward promising research and therapeutic avenues to help JNCL patients.

CLN3 disease (Batten disease) belongs to a group of rare lysosomal storage diseases referred to as Neuronal Ceroid Lipofuscinosis (NCL). NCLs are the most common dementia-causing neurodegenerative diseases in children. All NCL forms - including CLN3 disease - lead inevitably to early death. Clinical manifestations of CLN3 disease typically start with visual impairments around school age (5-6 years), which rapidly progress to blindness. Somewhat later patients start suffering from seizures, cognitive deficits, motor deficits, loss of speech, and loss of communication skills. In their teens CLN3 patients become completely helpless, wheelchair bound, and eventually bedridden. Patients succumb to disease in the second or third decade of life.

This year the NCL Foundation announces a call for its NCL Research Award (50,000 EUR) to support an innovative pilot project at the Postdoctoral fellowship level. We highly encourage junior scientists, clinical researchers and medical fellows worldwide to submit projects that hold promise to help find and push forward therapies for CLN3 disease. We also highly encourage applicants that work in disease areas outside NCL, provided the proposed research is relevant to elucidate the role of CLN3 or find a cure for this disease.

You can find the application forms on our homepage

Please, note that it is a 2-stage process. First of all, we need to receive your Letter of Intent before we accept your full application.

DEADLINE: November 30, 2017

We are looking forward to receiving your application via email  

NeuroNEXT Clinical Trials -U01 (NINDS, USA)

This FOA encourages applications for exploratory clinical trials of investigational agents (drugs, biologics, surgical therapies or devices) that may contribute to the justification for and provide the data required for designing a future trial, for biomarker validation studies, or for proof of mechanism clinical studies. Diseases chosen for study should be based on the NINDS’ strategic plan and clinical research interests (www.ninds.nih.gov/funding/areas/index.htm). Successful applicants will be given access to the NeuroNEXT infrastructure. Following peer review, NINDS will prioritize and order trials that are given access to the NeuroNEXT infrastructure. The NeuroNEXT Clinical Coordinating Center (CCC) will work with the successful applicant to efficiently implement the proposed study. The NeuroNEXT Data Coordinating Center (DCC) will provide statistical and data management support. The NeuroNEXT clinical sites will provide recruitment/retention support as well as on-site implementation of the clinical protocol.

Applicants do not need to be part of the existing NeuroNEXT infrastructure.

For further details

DEADLINES: December 6, 2017