CAR T Therapy: A Novel Treatment For Myositis Conditions

🧬 The Science Behind CAR T Cell Therapy

What is CAR T?
CAR T stands for Chimeric Antigen Receptor T-cell therapy. It’s an advanced form of immunotherapy that uses a patient’s own immune cells (T cells) to recognize and fight disease in a very targeted way.

How it works – step by step

Collecting T cells
A patient’s T cells are collected from their blood using a process called apheresis. This is done through a special line in the arm, often a PICC line, over several hours.
Engineering the T cells
In the lab, scientists reprogram the T cells by inserting a new gene that gives them a special “sensor” on their surface — the chimeric antigen receptor (CAR). This receptor acts like a GPS, guiding T cells to recognize and attack cells causing disease.
Expanding the army
Once engineered, the T cells are multiplied in the laboratory until they number in the billions. This ensures there are enough of them to have a strong therapeutic effect when given back to the patient.
Pre-conditioning chemotherapy
Before infusion, patients receive a short course of chemotherapy (usually 3 days). This prepares the body by creating “space” for the CAR T cells to expand once they are reintroduced and eliminating most of your existing B cells.
Infusion back into the patient
The engineered T cells are infused back into the patient, much like a blood transfusion. From here, they circulate, find their autoimmune B-cell targets (identified by a receptor marker such as CD19), and go to work.

What makes CAR T unique?

Unlike standard medications that need to be taken continuously, CAR T cells are living drugs — they can persist in the body, multiply, and continue to fight disease.
CAR T therapy is often personalized: each treatment is made from the patient’s own cells. New treatments may use allogenic CAR T cells, meaning they are T cells from healthy donors who are not recognized as foreing by the body
It has shown the ability to push diseases like cancers and some autoimmune conditions into remission, sometimes without the need for long-term immune suppression

👣 CAR T Therapy: The Patient Journey

Preparing for Collection
Before CAR T starts, your care team will help you plan. This may include adjusting medications and preparing for a hospital stay. You’ll also need to consider practical matters, such as where you and your caregiver will stay near the hospital. You need to have a caregiver, such as a family member or a friend, with you throughout this journey.

2. T Cell Collection (Apheresis)
Your T cells are collected through a special line placed in your arm (often a PICC line). Blood is drawn, a machine separates out your T cells, and the rest of your blood is returned to you. This process usually takes a few hours.

3. Pre-conditioning (Chemotherapy)
A short course of chemotherapy (about 3 days) is given just before your CAR T infusion. This “clears space” in your immune system so the new CAR T cells can grow. You may feel tired, nauseated, or lose your appetite during this stage.

4. The Infusion
Your engineered T cells, now multiplied into the billions, are infused back into your body in a process much like a blood transfusion. This part itself is usually not painful or difficult.

5. The Hospital Stay
Most patients stay in the hospital for 2–4 weeks for close monitoring, could be even more for some depening on symptoms. The care team checks your blood counts and watches for side effects such as fever, low blood pressure, or confusion. If needed, treatment for side effects is started right away.

6. Staying Nearby After Discharge
Even after you leave the hospital, you’ll need to remain close by (often 2–4 weeks) so doctors can see you quickly if side effects develop. Planning for housing, transportation, and caregiver support is very important here.

7. Recovery and Follow-up
Recovery looks different for every patient. Some feel stronger in weeks, others need months. Fatigue is common. You’ll have long-term follow-up visits (often for 2 years or more) to check your health and how well the CAR T cells are working.

Side Effects associated with CAR T Therapy

Cytokine Release Syndrome

CAR T cells release powerful immune signals (cytokines) when they activate. Sometimes, this immune response becomes very strong and causes:

Fever, chills
Rapid heartbeat
Low blood pressure
Trouble breathing

Most CRS is mild to moderate, but severe cases may need treatment in intensive care. Doctors monitor patients daily and have medicines ready to manage CRS if it develops.

Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS)

CAR T therapy can temporarily affect the brain and nervous system. Symptoms can include:

Confusion or difficulty concentrating
Trouble finding words or speaking
Handwriting changes
In more severe cases: seizures or drowsiness

These symptoms are usually reversible with treatment, but require careful observation. Your care team will check your thinking and speech every day during your hospital stay.

Low Blood Count (Cytopenia)

The chemotherapy given before CAR T lowers blood counts, and recovery takes time. This can lead to:

Fatigue (from low red blood cells)
Easy bruising or bleeding (from low platelets)
Higher risk of infections (from low white blood cells)

Patients may need blood transfusions, antibiotics, or other supportive care until their blood counts recover. Preventive medications are often continued for months after discharge.

Local Immune Effector Cell-Associated Toxicity

Syndrome (LICATS):

Rare, mild inflammation in

previously a ected areas, usually resolving within

about 11 days without long-term e ects.

Preconditoning Chemotherapy

Before CAR T cells are infused, patients receive a short course of chemotherapy called pre-conditioning or lymphodepleting chemotherapy. This step usually lasts 3 days and uses two medications:

Fludarabine – lowers the number of existing T cells and other immune cells, creating “space” for the engineered CAR T cells to grow and expand.
Cyclophosphamide – works alongside fludarabine to suppress the immune system and improve the persistence of CAR T cells once they are infused.

This chemotherapy is not designed to fight the disease directly. Instead, it prepares the immune system so that the infused CAR T cells can take hold and multiply effectively.

🌡️ Common Challenges During and After Pre-conditioning

Patients often tolerate the chemotherapy reasonably well, but it can cause mild to moderate side effects, including:

Fatigue or feeling “worn out”
Nausea or vomiting
Loss of appetite
Low blood counts (which can increase the risk of infection or anemia)

For most patients, these side effects appear within days of treatment. If they become severe, the CAR T infusion may be delayed for a few days until the body recovers.

🏥 Post-infusion Considerations

After the CAR T infusion, patients remain closely monitored because chemotherapy’s effects overlap with early CAR T side effects:

Low blood counts may persist for weeks, meaning increased risk of infections and bleeding.
Fatigue and appetite loss can continue, sometimes blending into the recovery period.
Slow immune recovery: Because chemotherapy reduces immune cells, it can take months for the immune system to rebuild. During this time, vaccines may be less effective, and protective measures against infections are especially important.

This is why most patients remain in the hospital for 2–3 weeks after infusion and must stay nearby for several weeks more, so the care team can respond quickly if complications arise.

Long-Term Antibotic Treatment

🔹 Why Antibiotics Are Used After CAR Theapy?

Low blood counts: The chemotherapy given before CAR T (fludarabine + cyclophosphamide) lowers white blood cells. Recovery can take weeks, leaving patients at higher risk for infections.
Delayed immune recovery: CAR T cells themselves also weaken the immune system, and full immune reconstitution can take months.
Risk of opportunistic infections: Patients are vulnerable to pneumonia, shingles, and fungal infections. Prophylactic antibiotics help reduce this risk.

🔹 What Patients May Receive

Protocols vary by hospital, but common prophylaxis includes:

Antibiotics (e.g., trimethoprim-sulfamethoxazole or alternatives) to prevent Pneumocystis jirovecii pneumonia.
Antivirals (e.g., acyclovir) to prevent herpes zoster (shingles) or herpes simplex virus reactivation.
Antifungals (e.g., fluconazole) in some cases, especially if neutropenia (low white blood cells) is prolonged.

🔹 Duration

Many centers continue antibiotics/antivirals for at least 6 months, and sometimes up to 12 months or longer, depending on blood counts and immune recovery.
Patients are monitored through regular bloodwork, and prophylaxis may be adjusted based on how quickly immune function returns.

🔹 What Patients Should Know

Preventive medications are a standard part of post-CAR T care, not a sign of complications.
Even when feeling well, patients should take these meds as prescribed, since infections can develop suddenly.
Vaccinations may not work well for months after CAR T, which makes these medications even more important.
Patients may need to stay away from their pets, even those used for support, during these treatments, as they may be more susceptible to animal infections.

FAQ-Brochure.pdf

FAQ Patient Brochure: For Patients and Myositis Centers

Please download the item below to read more about the CAR T. therapy and share it with your family members

This brochure was developed through a patient-centered process. Three people living with myositis who underwent CAR T cell therapy (two with dermatomyositis and one with antisynthetase syndrome) shared their experiences. Their insights shaped the content, ensuring that the questions and answers reflect the real concerns, challenges, and hopes of patients going through this journey.

To make sure the information is accurate and aligned with current research, the brochure was also reviewed by the Myositis Clinical Trial Consortium (MCTC), a leading group of specialists and researchers driving myositis clinical trials worldwide.

This combination of lived experience and expert review makes the brochure both authentic and reliable—a guide designed by patients, for patients, with the support of the medical community.

Simply Put Information on CAR T Therapy .pdf

Background on CAR T for Myositis

Background: At the 2024 Global Conference on Myositis (GCOM) last year, we learned all about the new treatment called CAR-T therapy that has been working wonders for some cancers and now holds exciting possibiites for several types of myositis, excluding IBM.

Intent: The Research Team is working to produce "Simply Put" materials to help YOU better understand the potential for this emerging therapy. We have partnered with the largest on-line Multiple Myeloma Facebook Support Group - The Multiple Myeloma Warrior Community to better under the patient perspective and adapt this information for our myositis community. We will update our education materials to include FAQs and suggested guidance for pharma during pre clinical trial design and development to ensure the patient perspective is represented.

Summary:

CAR-T Cell Therapy is notable for its potential to achieve remission in some patients who have not responded to other treatments, making it a breakthrough in personalized autoimmune tratment
It can cause significant side effects, including cytokine release syndrome, which requires careful management by medical professionals
Understanding potency of CAR-T Cell Therapy in controlling autoimmunity over the long term could have a disruptive effect in the myositis field
Clinical trials involving CAR-T cells in autoimmune malignancies are already underway including Lupus, Scleroderma, and many Myositis type (except IBM)
Accessibility of CAR-T Cell Therapy - Information on insurance, cost, and other health equity issues that are current barrier to treatment will impact its availability long-term.
Visit the Cabaletta website at: Phase 1/2 Trial in Myositis

CAR T therapy: the patiet journey

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