In this recent edition of our “Ask the Doc” series, Cure JM’s Shannon Malloy and Cabaletta Bio’s Dr. Tania Gonzalez Rivera discuss the science behind CAR-T therapy, the growing interest in CAR-T therapy as a potential treatment for autoimmune diseases, and Cabaletta Bio’s RESET ™ clinical trials with CABA-201 (investigational CD19-CAR T cell therapy).
Exciting news from the European Congress of Rheumatology (EULAR) 2024 last month in Vienna, Austria! Cure JM partner Cabaletta Bio, presented encouraging results from their CAR-T cell therapy, CABA-201, in treating myositis.
Positive Initial Data
Initial data from the first two patients dosed with CABA-201 showed positive signs in terms of safety, clinical response, and biological markers. Cabaletta believes this data validates the potential of CABA-201 to “reset the immune system” in patients with myositis and other autoimmune diseases.
Both patients tolerated the treatment well with no serious side effects reported and the therapy led to clinically meaningful improvements in the patients’ conditions.
“By demonstrating a potentially well-tolerated safety profile along with initial clinical and translational data …we believe CABA-201 may be uniquely positioned to fulfill unmet patient needs across a broad range of autoimmune diseases,” said David J. Chang, MD, chief medical officer at Cabaletta, in a statement.
“Immune System Reset” May Lead to Long-Term Remission
CABA-201 is a one-time cell therapy designed to “reset” the immune system, potentially leading to long-term remission without the need for constant medication. This approach offers a promising alternative for patients with myositis and other autoimmune diseases.
While the initial data is encouraging, further testing is necessary.
Cabaletta’s Commitment to Autoimmune Disease Research:
Cabaletta is actively investigating CABA-201 in five different autoimmune diseases, including myositis. Learn more about the myositis trial at https://www.curejm.org/research-volunteers-and-assistance-needed/
What is CAR-T?
As you know, the immune system is made up of cells that behave like “soldiers,” protecting the body from invaders like viruses. These soldiers are made up of B cells and T cells that work together to protect your body. Normally, the T cells tell the B cells about the enemy invaders, and the B cells defeat the invaders.
However, with myositis, the B cells are like misbehaving soldiers.
Instead of protecting the body against invaders, these misbehaving soldiers are attacking healthy skin and muscles.
Traditional myositis medications try to calm down the immune system so these misbehaving soldiers can cause less damage to healthy skin and muscle.
CAR-T is a new way of treating autoimmune conditions like myositis. Instead of trying to calm down the immune system, CAR-T “resets” the immune system. It is like a “living drug” to treat the myositis.
With CAR-T, doctors remove the cells of the immune system, modify the cells so they are behaving correctly, and put them back into the patient. It is our hope that when the “corrected” immune system is put back into the patient, the immune system will now be healthy.
Much testing remains to be done, but the early results are promising.
How CAR-T Works in Myositis:
- Extracting T Cells: Doctors take some of your immune system’s soldier cells (T cells) out of your body. This process will seem similar to an infusion.
- Giving The T Cells Special Instructions: In a lab, scientists add special instructions to your T cells, like giving them a superhero suit. This new suit is called a CAR (Chimeric Antigen Receptor). This new suit tells the T cells to recognize and attack the specific cells that are causing the problem in your body.
- Sending Them Back: The improved T cells are then put back into your body through an infusion.
- Targeted Attack: These super T cells can now directly target the misbehaving B cells causing your disease, leaving the healthy parts of your body alone. In this way, the CAR T is like a living drug that can prevent the body from attacking itself.
- Long-Term Protection: CAR-T therapy might help some people stay healthy for a long time. This is why we are testing this treatment and look forward to more results from current and future trials in myositis.
Important to Remember:
While we always want to keep you informed of the latest developments, please keep in mind that CAR-T therapy is still new for myositis and being studied carefully. Doctors will continue to monitor patients closely after receiving CAR-T therapy.
As always, please discuss all treatment options with your healthcare team. Your doctor knows you best!
If you would like to learn more about the CAR-T trial, currently accepting JM patients age 18-65, please visit https://www.curejm.org/research-volunteers-and-assistance-needed/
Cabaletta Bio’s David Chang, M.D., M.P.H, FACR, Chief Medical Officer, and Arun Das, M.D. Chief Business Officer, speaking with Cure JM’s Young Adult Advocates Council about their CAR-T trial. Photo taken at Cure JM’s National Family Conference, June 30, 2023 in Gaithersburg, MD.
(Read more about this new research at https://www.forbes.com/sites/juergeneckhardt/2024/05/30/car-t-cell-therapy-could-be-a-gamechanger-for-autoimmune-diseases/)
