# Advancing Drug Development in AL Amyloidosis: Capitalizing Natural History and Assessing Endpoints

> **NIH NIH R13** · AMYLOIDOSIS RESEARCH CONSORTIUM, INC. · 2021 · $24,392

## Abstract

Project Summary
Amyloidosis Research Consortium (ARC), a 501(c)(3) nonprofit organization focused on
accelerating the development, approval, and access to new and innovative treatments for
amyloidosis. In 2019, ARC entered into a Public Private Partnership (PPP) with the US Food
and Drug Administration (FDA) to identify and bridge the scientific gaps in drug discovery and
development for the treatment of AL Amyloidosis. Amyloidosis is the term for a group of rare,
progressive and fatal misfolded protein diseases. AL amyloidosis a rare and multi-systemic
disease in which there is no approved FDA therapy. AL amyloidosis is a disease that
disproportionately afflicts older adults and has a poor prognosis when diagnosed in an
advanced stage. All the emerging therapies are aimed at treating the underlying plasma cell
dyscrasia are most advantageous when administered early in the course of the disease.
As part of the PPP, ARC and FDA are proposing to host the second in a series of cross-
stakeholder scientific meetings (with involvement from patients, academia, industry, and
regulatory agencies) entitled The Amyloidosis Forum. Each of these meetings is focused on
identifying the knowledge gaps, barriers, and strategically addressing these to advance drug
development in this challenging disease. The next public meeting will be held on 12th
November, 2020, entitled: Amyloidosis Forum Meeting 2: Advancing Drug Development in
AL Amyloidosis: Capitalizing Natural History and Assessing Endpoints:
Forum Objective 1: Natural History and Endpoint Development: The morning sessions will
provide an in depth look at the importance of natural history from academia, industry and
regulatory perspectives. There will be presentations on current sources of natural history from
academia, industry (placebo data) and other sources, as well as discussion about how to leverage
existing datasets to identify meaningful endpoints, disease specific measurements, and ultimately
accelerate research.
Forum Objective 2: Endpoint Development: Given the complex multi-systemic nature of AL
amyloidosis, endpoint choice is challenging. The second part of the forum will focus on endpoint
development; traditional designs versus novel designs. such as hierarchical or multimodal
endpoints, with case studies from industry and FDA. This meeting will seek to define innovative
approaches toward endpoint development including considerations for a multi-domain responder
index (MDRI).

## Key facts

- **NIH application ID:** 10144046
- **Project number:** 1R13AG071150-01
- **Recipient organization:** AMYLOIDOSIS RESEARCH CONSORTIUM, INC.
- **Principal Investigator:** Isabelle R. Lousada
- **Activity code:** R13 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $24,392
- **Award type:** 1
- **Project period:** 2021-01-08 → 2021-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10144046

## Citation

> US National Institutes of Health, RePORTER application 10144046, Advancing Drug Development in AL Amyloidosis: Capitalizing Natural History and Assessing Endpoints (1R13AG071150-01). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10144046. Licensed CC0.

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