NIH Funds First Step Towards Advancing ATTR Amyloidosis Drug Development

The NIH has awarded the Amyloidosis Research Consortium $40,000 to support the organization’s Amyloidosis Forum meeting on “Advancing Drug Development in ATTR Amyloidosis in an Evolving Treatment Landscape.”

Formed in 2019, The Amyloidosis Forum (https://amyloidosisforum.org) has been developed through a public-private partnership between the Amyloidosis Research Consortium (ARC) and the US Food and Drug Administration (FDA). The overarching goal of the forum is to bring together the entire amyloidosis community to identify and bridge the scientific gaps that are acting as barriers to drug discovery and development for the treatment of amyloidosis.

The next Amyloidosis Forum meeting will set the stage by sharing the advancements in ATTR amyloidosis, evaluating the impact and unmet needs that currently exist, and identifying key initiatives with the aim of leveraging cross-stakeholder resources to collaboratively act on field-shaping priorities. The meeting will combine global clinician, health authority, industry, payor, and patient perspectives to shape the future of drug ATTR amyloidosis drug development. The NIH has awarded the Amyloidosis Research Consortium $40,000 to support the organization’s Amyloidosis Forum meeting on “Advancing Drug Development in ATTR amyloidosis in an Evolving Treatment Landscape” in June. The meeting will optimize innovation and accelerate advancement of drug development and new therapies for ATTR amyloidosis.

“ARC in partnership with FDA provides a unique environment for national and international patients, academic, industry, patient advocacy, and regulatory groups to tackle the challenges faced by amyloidosis patients.”
— NIH Review Board

This is the first ATTR amyloidosis meeting of its kind bringing together scientists, stakeholders, patients, and governmental staff together for a one-day conference to understand the impact of novel treatments, unmet patient needs, define approaches to designing clinical trials, and outline regulatory approval and reimbursement challenges. In their award letter, the NIH highlighted the unique impact of this meeting writing, “ARC in partnership with FDA provides a unique environment for national and international patients, academic, industry, patient advocacy, and regulatory groups to tackle the challenges faced by amyloidosis patients.”

A Model for Rare Disease Research & Development

The NIH R13 grant, part of the organization’s Support for Scientific Conferences program, will support this meeting of the Amyloidosis Forum at a critical time for the ATTR amyloidosis community. The first generation of drug approvals in ATTR amyloidosis has reshaped the research landscape creating the need to reconsider how to effectively design trials against a backdrop of treatments that slow progression of the disease. This Forum meeting will set the stage by sharing the advancements, evaluating the impact and unmet needs that currently exist, and identifying key initiatives with the aim of leveraging cross-stakeholder resources to collaboratively act on field-shaping priorities.

“The Amyloidosis Research Consortium’s work bringing together patients, clinicians, researchers, government agencies, and drug companies is vital to extending the lives of those living with this rare disease.”
— Congressman Jake Auchincloss (D, MA-04)

A 2022 study showed that the conservatively estimated impact of rare diseases in the U.S. in 2019 was nearly $1 trillion. 1-in-10 people live with a rare disease — that’s 33,000,000 Americans. The Amyloidosis Forum exemplifies some of the work needed to make the treatments they need available. “The Amyloidosis Research Consortium’s work bringing together patients, clinicians, researchers, government agencies, and drug companies is vital to extending the lives of those living with this rare disease,” said Congressman Jake Auchincloss, representative for Massachusetts’s 4th congressional district where ARC is located. “I’m honored to represent this innovative and impactful model for accelerating rare disease research.”

Pushing Research Forward

The June 21 meeting will be a hybrid conference, held in-person at the Food and Drug Administration’s (FDA) White Oak Campus and virtually via Zoom. Isabelle Lousada, ARC Founder and CEO, has deep experience on this conference topic, having written congressional briefings and interacted with government regulators and stakeholders in the past while also publishing papers on patient experiences and clinically relevant endpoints. The conference is innovative in both the topic and the fact that it is open to the public, with the expectation of 120-150 people in-person and 300-400 joining virtually.

“The research, working groups, and collaboration built by the Amyloidosis Forum project have made a significant impact on the amyloidosis community since it began, and it is encouraging to see NIH acknowledge and fund the Forum as we make even greater strides forward.”
— Isabelle Lousada, ARC Founder & CEO

When asked about the importance of the Amyloidosis Forum, Isabelle Lousada said, “The research, working groups, and collaboration built by the Amyloidosis Forum project have made a significant impact on the amyloidosis community since it began, and it is encouraging to see NIH acknowledge and fund the Forum as we make even greater strides forward.”

The FDA campus is an excellent venue, and the conference planning appropriately accounts for outreach to underrepresented minorities (URM) for engagement as well as the involvement of women in the conference planning and speakers. The meeting will also focus on the Val122lle mutation in the transthyretin gene, which is the most common mutation in the United States and has significant implications for better understanding how ATTR amyloidosis impacts the African American community. The potential impact on a rare and under diagnosed disease is significant. Overall, the important topic, well-planned agenda, and innovative topic generated high enthusiasm from the NIH reviewers.

To learn more about the Amyloidosis Forum and the upcoming June meeting, visit: https://amyloidosisforum.org/attr-drug-development/