This competitive grant program seeks to support research conducted by Junior Investigators that advances medical knowledge in the understanding, diagnosis and treatment of ATTR Amyloidosis.

Pfizer is interested in supporting research proposals that advance our understanding of the epidemiology, basic science and early diagnosis and treatment of ATTR amyloidosis through research focused in the following areas:

Epidemiologic evaluations for ATTR amyloidosis

• Global or regionally-focused evaluations
• Gender analysis – prevalence, natural history, etc.

Approaches for the early identification and follow up of ATTR amyloidosis patients

• Multidisciplinary approaches to diagnosis and care
• Multimodality imaging approaches to diagnosis including Artificial Intelligence (AI) and Machine Learning
• New diagnostic algorithms
• Non-invasive diagnostic techniques (e.g. biomarkers, use of AI)
• Risk factors for disease penetrance
• Definition and measurement disease progression and natural history studies
• New staging systems
• New outcomes measures (Quality of life; patient reported outcomes, Imaging)
• Management of cardiac complications

RW efficacy and safety of tafamidis in the clinical setting for the management of ATTR amyloidosis

• Effect of Tafamidis on conduction disorders (i.e. arrhythmias, atrial fibrillation)
• Effect of Tafamidis on ECHO, MRI or PET measures and hemodynamics
• Effect of Tafamidis on amyloid burden
• Effect of Tafamidis on quality of life
• Changes in serum TTR, biomarker analysis
• Evaluating existing databases for clinical outcomes
• Use of tafamidis post-organ transplant

Evaluation of patients with ATTR-CM presenting with a mixed phenotype (e.g. cardiomyopathy and polyneuropathy)

• RW treatment of mixed phenotype patients with Tafamidis 61mg including examination of both cardiac and neurological outcomes
• Longitudinal evaluations of neurologic manifestations in patients with ATTR-CM including wild type
• Case Control and Cross-Sectional Studies
• Evaluating Existing Databases for clinical outcomes

Pathophysiology:

• Mechanistic studies to advance the basic science of amyloid formation and deposition including studies of the:
• Functional role of TTR
• Long term impact of TTR knockdown
• Amyloidogenesis
• Tissue tropism of amyloid deposits
• Proteolytic cleavage