Scleroderma added to NIH project targeting autoimmune diseases

Partnership program aims to understand diseases, find treatments

Written by Andrea Lobo, PhD |

People's hands meet in a circle in this hands-in illustration.

Scleroderma is now part of a federal program that aims to help understand underlying mechanisms and develop new treatments and diagnostic targets for autoimmune conditions.

The Foundation for the National Institutes of Health (FNIH) has expanded its Accelerating Medicines Partnership Autoimmune and Immune-Mediated Diseases (AMP AIM) program, a research collaboration that brings together the NIH,  private companies, foundations, and patient groups.

The National Scleroderma Foundation and the Scleroderma Research Foundation are joining the program as patient advocacy partners, “ensuring that perspectives of individuals with lived experience remain central to the research process and that scientific priorities align with patient needs,” according to a FNIH press release.

Program partners are contributing more than $9 million to support the expansion, adding to the $62 million originally invested.

“The expanded initiative reflects a growing commitment across public, private, academic, and patient communities to accelerate progress in autoimmune disease research, which together affect more than 15 million Americans,” said Meghan Pennini, PhD, director of translational science, inflammation and immunity at the FNIH.

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“By using advanced tools to study cells and molecules, this program will help us better understand both the shared and unique biological processes that drive different autoimmune diseases,” Pennini said.

Scleroderma is characterized by thick, hardened skin due to excessive buildup of scar tissue, which may also accumulate and damage internal organs. A combination of genetic and environmental factors is believed to play a role in disease development, but its underlying mechanisms are not completely understood.

Although treatments can help manage scleroderma symptoms, they may come with significant side effects and do not cure the condition.

Scleroderma joins four other autoimmune conditions in the AMP AIM program: rheumatoid arthritis, lupus, Sjögren’s disease, and psoriatic diseases (autoimmune diseases that may affect the skin and joints). Having multiple conditions as part of the program will enable comparisons across diseases to uncover the cells and biological pathways driving autoimmune conditions and, according to the FNIH, help reveal new targets for diagnosis and treatment.

According to the National Scleroderma Foundation, which led the inclusion of the disease in the expansion and serves on the AMP AIM steering committee, the collaboration will allow researchers to analyze individual cells from patient samples to understand how the immune system interacts with scar-forming processes that drive the disease.

Investigators expect the initiative to provide new insights into how scleroderma develops and progresses, why symptoms vary so widely among patients, and which therapies may be most effective.

Boston University Medical Center, the Hospital for Special Surgery, University of Michigan, University of Pittsburgh Medical Center, University of Texas Health Sciences Center, and Yale University are participating in the project. All are National Scleroderma Foundation Designated Scleroderma Research and Treatment Centers.

“This project brings together a network of researchers to tackle this extremely complex disease,” Mary J. Wheatley, National Scleroderma Foundation CEO, said in a foundation press release. “We can leverage technologies through this project that we could never achieve through single investigator research initiatives.”

Carol Feghali-Bostwick, PhD, chair of the scleroderma foundation, said participation in the project “can be a key step towards gaining novel insights into disease processes and developing personalized medicine approaches for people living with scleroderma and other autoimmune disease.”

The AMP AIM program evolved from an earlier initiative, AMP rheumatoid arthritis/systemic lupus erythematosus, which analyzed cell subsets and single cells to understand disease mechanisms.

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