Lupus consortium partners with FDA on cell therapies

Lupus ABC to work with CBER on CAR-T cell project

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) is joining the Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to focus on CAR T-cell treatments and other engineered cell therapies, a promising new therapeutic field now being explored for lupus.

“We are delighted to have the Center for Biologics Evaluation and Research join Lupus ABC at this important juncture in the development of engineered cell therapies for lupus to help us accelerate their clinical evaluation,” Teodora Staeva, PhD, vice president and chief scientific officer of the Lupus Research Alliance (LRA), said in an alliance press release.

Lupus ABC is a coalition of more than 40 groups, including pharmaceutical companies and advocacy organizations, dedicated to advancing care for lupus. The consortium was founded last year by the LRA in collaboration with the FDA’s Center for Drug Evaluation and Research.

The consortium “is the first lupus-specific public-private partnership (PPP) established in response to the urgent need for safer and more effective lupus treatment options,” Hoang Nguyen, PhD, director of scientific partnerships at the LRA, wrote in a statement to Lupus News Today.

“Lupus ABC brings people with lupus together with the [FDA] and other key stakeholders, including members from academia, industry, professional societies, governmental agencies, and advocacy groups to advance the development of new treatments for lupus in a pre-competitive setting,” Nguyen said.

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Lupus is an autoimmune disease in which the immune system erroneously starts attacking healthy parts of the body. As part of this attack, B-cells, a type of immune cell, make proteins called antibodies that target and attack the body’s own healthy tissues and organs. Lupus can cause a wide range of manifestations and symptoms, and its causes are not fully understood.

Lupus ABC has launched three projects aiming to create better outcome measures for use in lupus clinical trials. It’s focused on standardized measures to assess treatment response, the severity of cutaneous lupus (a type of lupus that affects the skin), and patient-reported outcomes. Lupus patients have been central to discussions creating these projects, the coalition said.

A fourth project is focused on CAR T-cells, a promising type of experimental lupus therapy.

T-cells are immune cells that are able to recognize and kill other cells that have gone rogue, such as cells that have been infected with a virus or become cancerous. CAR T-cell therapies work by collecting a patient’s T-cells and engineering them in a laboratory in a way to ensure they will attack and destroy a specific type of cell.

This therapeutic strategy was originally developed to treat some types of blood cancers, but researchers are now exploring this technology in autoimmune diseases like lupus. The basic idea is to use CAR T-cells to eliminate disease-driving immune cells, such as B-cells. Several experimental CAR T-cells are currently in clinical development for lupus, with some showing promising results in early trials.

“The promising outcomes of CAR-T cell therapy in a small number of lupus patients have sparked significant interest in its use for lupus treatment,” Nguyen said.

CAR T-cells are a type of biologic therapy, so CBER is expected to be a key contributor to Lupus ABC’s latest project.

The CAR T-cell Working Group was formed “to address outstanding questions on the use of CAR-T cell therapy in lupus with the goal to advance CAR-T cell therapy safely for lupus patients,” Nguyen said. Its near-term goal is “to produce a position paper on the key considerations to advance CAR-T cell therapy for lupus,” such as “patients’ eligibility for treatment, overall trial design, dose selection, outcome measure, safety monitoring.”

CBER, for its part, “shares the excitement and enthusiasm around the transformative potential of emerging technologies such as CAR-T therapies to address an unmet medical need for patients with this serious, potentially life-threatening condition,” said Peter Marks, MD, PhD, director of CBER. “We look forward to collaborating with Lupus ABC to shepherd the product development plans in a safe manner.”