Bringing the science of cell therapy into autoimmune diseases

April 29, 2024     

Many are familiar with CAR T cell therapy as an important treatment for patients living with certain blood cancers, and may know Bristol Myers Squibb as a driver of innovation and progress in the space.

Now, we’re drawing on our deep expertise across cell therapy and immunology to lead the charge to bring the potential of CAR T cell therapy beyond cancer and into autoimmune disease, including severe lupus and multiple sclerosis – and it’s an area I’m profoundly excited about.

The nuances & burdens of autoimmune disease

For people with certain autoimmune diseases, the immune system that normally defends their body against disease and infection instead attacks healthy cells and tissues. Depending on which disease or manifestation, this can affect various parts of the body, and lead to serious organ damage that can become life-threatening.

A key focus of our autoimmune research is severe, treatment-resistant lupus. This research is close to my heart, as most patients living with lupus are women diagnosed between the ages of 15 and 44, with full lives ahead of them. Moreover, the disease is three times more likely to occur among people of African American or Latin descent. These young patients are actively focused on progressing their education, building careers, starting families and fulfilling dreams. They do this while living with the significant physical and emotional burden of chronic symptoms such as extreme fatigue and pain. Symptoms that require frequent, and often life-long treatment.

Current treatment approaches for lupus focus on reducing these debilitating symptoms and controlling disease flares. While many patients find relief from these important treatments, in many cases, options like steroids and immunosuppressant therapies can lead to additional side effects like diabetes, weight gain, heart disease, infertility and risk of infection. Moreover, the effectiveness of these therapies decreases over time and the disease may continue to progress and become resistant to treatment.

At the most severe end of the spectrum, these patients must fear ever more serious symptoms: pregnancy complications, organ failure, and in some cases, death. For patients who have been living with lupus or other autoimmune diseases for years, and for whom quality of life and prognosis is poor, there is a clear and urgent need for innovative, one-time treatment options that directly address the underlying condition of a patient’s disease, not just the symptoms.

Exploring the potential of “resetting” the immune system
  • CAR T in autoimmune: Antibody-producing B cells are believed to play a major role in the development of lupus and other autoimmune diseases. CD19 is a biomarker for these B cells, with elevated CD19 present in patients with these conditions. With demonstrated success and a well-understood safety profile within other B cell mediated diseases, CD-19 targeted CAR T cell therapy is being evaluated as an approach to address unmet need across the autoimmune space.  Read more here about the CAR T process and potential. 

  • What do you mean by reset? An emerging theory is that CD-19 CAR T cell therapy may be able to “reset” the immune system analogous to how we reboot a computer or phone to restart it when it is malfunctioning.

  • What we’ve learned so far:  A proof-of-concept trial led by prominent researcher Prof. Dr. Georg Schett evaluated how well CAR T cell therapies worked in patients with severe lupus, including whether patients experienced durable remissions and were able to stop their other medications. Read more about the promising early results in the New England Journal of Medicine.

  • What’s next: While the preliminary data are encouraging, we’re just beginning to uncover new ground in this exciting field, starting by testing an optimized CD19 NEX-T CAR T cell therapy in lupus, and quickly expanding our research into other autoimmune diseases such as idiopathic inflammatory myopathy, systemic sclerosis and multiple sclerosis. This research will, of course, also help us understand the associated safety of such treatments in these new diseases. Learn more about our cell therapy clinical trial in severe lupus here and the variety of pathways and mechanisms we are exploring in lupus here.
Pioneering a new frontier

We are proud to be among the pioneers evaluating the use of cell therapy to treat severe autoimmune disease. We are spearheading the field, armed with the best-in-class CAR construct we established in lymphoma, focusing on reducing manufacturing complexity and improving turnaround time with our next generation of therapies.

Delivering the next wave of advances for patients with autoimmune disease is not a journey we take alone, and we are collaborating with, and learning every day from, the patients who live with these diseases and their caregivers, healthcare professionals, industry partners, academic institutions and advocacy organizations. 

We are quickly progressing enrollment across our clinical trials and look forward to sharing results at future medical meetings to determine actionable ways to elevate patient care to new standards. Together, we can progress this science and bring the transformational potential of cell therapy to patients with lupus and other autoimmune diseases.

Author: Ashley Koegel

Executive Director and Early Clinical Development lead, Bristol Myers Squibb

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