Taking ACTioN
Advancing cell therapy for autoimmune diseases
Rosanna Ricafort:
CAR T cell therapy has delivered transformational benefits in certain types of blood cancers, such as non-Hodgkin lymphoma and multiple myeloma, and has really changed the way that we treat those diseases.
Daniela van Eickels:
We are now investigating if we can transfer the concept of selectively targeting cells to cells that are not uncontrollably proliferating, but that are dysfunctional, and those dysfunctional B-cells are intimately involved in the autoimmune reaction in the body.
Rosanna Ricafort:
In autoimmune diseases, there's a war inside the body where patients’ B cells really turn against and attack certain proteins in the body. And in a disease such as severe lupus, this can have many manifestations in tissue damage to multiple different organs, such as the kidneys, the skin, the joints and the heart. Patients with autoimmune diseases are treated with chronic, long-term immunosuppressive therapy to keep their disease under control. Applying the theory that CAR T cells can be programed to detect and kill disease cells, we can deliver CAR T cells that are against those autoreactive B cells that are implicated in the pathogenesis of certain autoimmune diseases.
Jonathan Leith:
Cell therapies are being investigated in autoimmune diseases because currently the available therapies do not address the underlying pathology of the disease. And so we encounter patients who have relapsed and refractory disease who are in desperate need of therapies that can give them long-term remissions and the potential to not have to take therapies lifelong, which is currently the case.
Rosanna Ricafort:
ACTioN, which stands for Autoimmunity Cell Therapy Network, is a global collaborative effort to bring together scientists from hematology, rheumatology, immunology and neurology to focus on accelerating cell therapies for patients with autoimmune disease.
Jonathan Leith:
ACTioN is a network of our best and brightest clinicians and scientists with external collaborators, clinicians and scientists, to address some of the key underlying challenges we see with bringing not only these new cellular therapies to patients with autoimmune disease, but also addressing some of the key challenges that we see in treating these patients.
Daniela van Eickels:
So we are bringing together different disciplines, for instance, rheumatologists, neurologists, hematologists, and they will share from their perspective and their knowledge base. And so, people will learn and will be able to further develop the concepts of cellular therapy in autoimmunity.
Daniela van Eickels:
For me, ACTioN is an incredible opportunity to bring people together very early on and to use our learnings from hematology of what makes cell therapy successful.
Jonathan Leith:
ACTioN is about working together. And so what it means to me is I'm just really excited that we can make a difference. That we can take on this challenge and we can work together with clinicians, scientists and patients to advance cellular therapies in autoimmunity as quickly as we can.
Rosanna Ricafort:
It's the way in which we work together as a global community, bringing together global scientists, researchers, patient advocates to really think outside the box. And this deep conviction that if we follow the science and where it is going, we can fundamentally change the way that we treat autoimmune diseases and improve the lives for these patients.
CAR T cell therapy is a proven, transformational treatment approved to treat many forms of blood cancer, and initial research demonstrates the potential for cell therapy to be used to treat severe forms of certain B cell-mediated autoimmune diseases.
As the only company with two approved CAR T cell therapies for two distinct targets in hematology, a pioneer in harnessing the immune system to fight cancer and a leader in treating autoimmune diseases, Bristol Myers Squibb has assumed an important leadership role within the industry. Alongside a group of experts, researchers and patient advocates, BMS is taking ACTioN to expand the science of CAR T into autoimmune disease.
What is ACTioN?
Cross-disciplinary collaboration – and collaboration with patients – plays a crucial role in advancing the science that could make CAR T cell therapy an option for treating autoimmune diseases and accelerating these advances to patients.
ACTioN stands for the Autoimmunity Cell Therapy Network, and the name helps underscore our drive and passion to move this work forward with speed and focus. A first-of-its-kind network, BMS created ACTioN to accelerate interdisciplinary collaboration by bringing together leading global scientific experts, clinicians and patient advocates.
We are working to unlock the full potential of cell therapy and deliver potentially transformative treatments to patients living with certain autoimmune diseases and are currently exploring cell therapy in patients who have been living with their diseases for years, have stopped responding to therapy, and have a poor quality of life and prognosis.
Accelerating medical advancement, together
With the goal of expanding the potential of cell therapy to people living with autoimmune diseases, ACTioN is organized around three interconnected pillars:
- Scientific leadership aims to expand scientific advancements in cell therapy to autoimmune patients faster through evidence generation. This important work is driven by leading scientists and clinicians working in cell therapy and/or autoimmunity.
- Clinical excellence focuses on facilitating knowledge exchange across the multidisciplinary care teams who collaborate across various specialties, such as rheumatology, nephrology, neurology and hematology, to treat patients with autoimmune diseases.
- Ecosystem expansion focuses on expanding referral networks for clinical trials and developing a strong foundation to support treatment ramp-up of cell therapy in autoimmune disease. Importantly, it ensures we hear directly from patients about their daily challenges, integrate their collective voice into the development of these therapies, and develop strategies to reduce barriers to access to clinical trials.
By bringing these stakeholders together, encouraging open and productive dialogue and developing strategies to address challenges in near-real time, we are driving ACTioN and progress to one day unlock the full potential of cell therapy for patients living with autoimmune diseases.
ACTioN leadership
Jonathan Leith, PhD
Vice President Early Development Program Lead, Hematology, Oncology and Cell Therapy (HOCT)
Chair
Daniela van Eickels, MD, MPH
Vice President, Head of Medical Affairs, Cell Therapy Organization
Co-Chair
Rosanna Ricafort, MD
Vice President, Senior Global Program Lead for Hematology and Cell Therapy
Co-Chair
ACTioN is about the way we work together as a global community, bringing together scientists, researchers and patient advocates to think outside the box, with the deep conviction that if we follow the science and where it is going, we can fundamentally change the way that we treat autoimmune diseases and improve the lives of these patients. 
Urgent need for innovative therapies to treat autoimmune diseases
People living with autoimmune diseases face a range of progressive, often severe symptoms that profoundly impact their quality of life and can lead to life-threatening organ damage or death.
Together with ACTioN members, BMS is investigating cell therapy for the treatment of autoimmune diseases such as systemic lupus erythematosus, multiple sclerosis, idiopathic inflammatory myopathies and systemic sclerosis:
- People with systemic lupus erythematosus (SLE) - including lupus nephritis – live with a range of debilitating symptoms that escalate significantly over time, can lead to life-threatening organ damage and significantly impact quality of life. Most patients living with lupus are women, and Black or African American, Hispanic, Asian, and American Indian and Alaska Native (AI/AN) populations are affected more than White populations.
- Multiple sclerosis (MS) attacks the central nervous system and greatly impacts people’s ability to complete daily tasks, with more severe symptoms including permanent neurodegeneration and cognitive decline. MS is considered an “immune-mediated disease,” in which the body’s immune system affects the brain and spinal cord and it can develop at any age, but most commonly appears between the ages of 20 and 40.
- Idiopathic inflammatory myopathies (IIM) are a group of conditions characterized by the inflammation of muscles and other body systems, which can cause symptoms like weakness and difficulty swallowing or breathing.
- Systemic sclerosis (SSc) is a group of rare diseases that involve the hardening and tightening of the skin, and in severe cases can involve the lungs, heart and other organs.