CAR-T Cell Therapy’s Next Advances Have a Cincinnati Connection

Key Points:

• Cincinnati Children’s took part in the clinical trial behind Kymriah, which the FDA approved in August 2017 as the first CAR-T cell therapy.
• Cincinnati Children’s $60 million Applied Gene and Cell Therapy Center in Sharonville expects to begin onsite manufacturing by early 2027.
• Tempest Therapeutics chose the Sharonville center in March 2026 to prepare its dual-target CAR-T, TPST-2003, for a planned U.S. myeloma study.
• In June 2025, the FDA cut the recommended stay near a CAR-T treatment center from four weeks to two for six approved products.

Article Summary:

CAR-T cell therapy reprograms a patient’s own T cells to attack cancer, and every FDA-approved CAR-T product still treats a blood cancer. Researchers are now testing dual-target, off-the-shelf, in-body and autoimmune versions. Cincinnati Children’s, which helped test the first approved CAR-T, is leading a 2026 pediatric trial and preparing a dual-target therapy in Sharonville.

Cincinnati Children’s helped test Kymriah, the first CAR-T therapy the FDA approved.

Nine years later, a Sharonville center is helping prepare the next generation of engineered immune cells.

You’re likely to see more news soon about CAR-T cell therapy, which turns a patient’s own immune cells against cancer. It falls under the category of “personalized medicine,” and in 2017 it became the first FDA-approved treatment that:

• reprograms a patient’s own immune T cells,
• teaches them a new skill (finding specific cancer markers),
• and allows them to multiply into a living drug inside the body.

Existing therapies work differently, and each one has drawbacks. Chemotherapy kills fast-growing cells, cancerous and healthy alike, and that’s where many of its side effects come from. Radiation damages the DNA of cancer cells, but it can also harm healthy tissue in its path. Targeted drugs block pathways that cancer cells need to grow.

If you prefer analogies:

• Chemotherapy is like treating an entire house because mold may be present in more than one room.
• Radiation is like focusing treatment on one room.
• Targeted therapy is like using a special key that fits only the lock on the room with the mold.

CAR-T is fundamentally different: it creates a new immune army that didn’t exist before. When the FDA approved Kymriah, the first CAR-T product, in August 2017, then-Commissioner Scott Gottlieb called it “a new frontier in medical innovation.”

The process goes like this. Doctors collect T cells, white blood cells that normally help protect the body, and genetically modify them so they can recognize and attack cancer cells. The “CAR” stands for chimeric antigen receptor, the engineered protein that lets a T cell spot a marker on the surface of a cancer cell. The modified cells then go back into the patient, where they can multiply and hunt for cancer.

CAR-T cell therapy has already changed treatment for some blood cancers, including certain leukemias, lymphomas and multiple myeloma. Now scientists are working on new ways to make CAR-T cells more powerful, more controllable, easier to manufacture and useful against more types of cancer.

Where Cincinnati Fits in CAR-T Cell Therapy

Cincinnati has been part of this story from the start, through one of its nationally ranked hospitals. Cincinnati Children’s says it took part in the initial clinical trial behind Kymriah’s approval, which makes it one of the most experienced pediatric CAR-T centers in the country. In the trial the FDA cited for approval, 63 children and young adults with relapsed or treatment-resistant leukemia, 83% reached remission within three months, according to the FDA.

Adults have a local option, too. UC Health offers adult CAR-T treatment through its cancer center, and Gilead has certified it as a designated CAR-T center. UC Health says patients stay in the hospital for monitoring and often go home within two weeks.

The newest piece sits in Sharonville. In November 2025, Cincinnati Children’s announced its $60 million Applied Gene and Cell Therapy Center at 10995 Canal Road, with 12 clean rooms. The hospital expects onsite manufacturing to begin by early 2027, after a yearlong certification process. Meanwhile, two 2026 deals already point it toward the CAR-T advances described below.

1. CAR-T Cells Can Target More Than One Cancer Signal

Scientists designed early CAR-T treatments to recognize a single marker on cancer cells. One problem is that cancer cells can sometimes escape by losing or reducing that marker. Researchers call this antigen escape.

So scientists are developing CAR-T cells that can recognize two or more cancer markers at the same time. The goal is to make it harder for cancer cells to hide.

One of those dual-target therapies now has a Cincinnati connection. TPST-2003, from California-based Tempest Therapeutics, recognizes two markers, CD19 and BCMA, and targets multiple myeloma. In March, Tempest picked the Sharonville center as its manufacturing partner ahead of a planned U.S. registrational study. By April, the center had received the lentiviral vector, the gene-delivery ingredient needed to make the cells, according to Tempest.

The early numbers look strong, with an important caveat. In May, Tempest reported that all 15 efficacy-evaluable patients who had never received CAR-T reached a complete response. However, those results come from 44 patients across three small studies in China, including a trial its partner, Novatim Immune Therapeutics, sponsors. They’re company-reported, not results from a peer-reviewed U.S. trial.

2. Better Tools for Fighting Solid Tumors

CAR-T cell therapy has been most successful against blood cancers. In fact, every CAR-T product on the FDA’s approved list treats a blood cancer. Solid tumors such as breast, lung, pancreatic and colon cancers are much harder to treat with CAR-T cells.

Solid tumors can create an environment that keeps immune cells from entering and working properly. Cancer cells may also carry different markers from one another. That makes it difficult to find a target that appears on every cancer cell but not on healthy tissue.

3. Off-the-Shelf CAR-T: Ready When Patients Need It

Traditional CAR-T cell therapy is custom-made for each patient. Doctors remove T cells from a patient, send them to a specialized facility, genetically modify them, grow millions of copies and then return them to the patient. This process can take weeks, an important problem when someone has a rapidly growing cancer.

Off-the-shelf CAR-T starts instead with T cells from healthy donors. In theory, a manufacturer could make doses in advance, store them and ship one the moment a patient needs it. Researchers genetically modify those donor cells to reduce the risk that they will attack the patient’s tissues or that the patient’s immune system will reject them.

Early clinical studies have shown that some of these treatments can produce meaningful responses. Still, no donor-derived CAR-T product has FDA approval yet. If researchers can show that they are safe, effective and reliable, off-the-shelf CAR-T cells could eventually cut manufacturing time and cost and reach more patients.

4. Making CAR-T Cells Inside the Body

One of the most futuristic ideas is to create CAR-T cells inside the patient. Today, CAR-T treatment generally requires doctors to remove T cells and modify them in a laboratory. With an in-body approach, scientists are developing delivery systems that could carry CAR instructions directly to a patient’s T cells. The patient’s own cells could then become CAR-T cells without ever leaving the body.

Researchers are investigating technologies including specially designed viruses and tiny particles called nanoparticles to deliver the genetic instructions.

The first human results show both the promise and the risk. In March 2026, researchers in China reported in Nature Medicine on five myeloma patients who received ESO-T01, an in-body CAR-T from AstraZeneca-owned EsoBiotec, Fierce Biotech reported. Three reached stringent complete remission by day 60. Every patient also had a grade 3 or higher side effect, and one died 19 days after treatment from spinal cord compression caused by a myeloma tumor.

So this approach remains in the early stages of development. But if scientists can make it safe, precise and controllable, it could eventually simplify CAR-T treatment and bring it to many more patients and hospitals.

5. Making CAR-T Cell Therapy Safer and More Controllable

CAR-T cell therapy can produce remarkable results, but it can also cause serious side effects. When CAR-T cells become highly active, they can trigger cytokine release syndrome, which can cause fever, low blood pressure and other problems. Some patients also experience neurological side effects. Scientists are developing ways to give doctors greater control over CAR-T cells and to protect patients.

The FDA’s own view has shifted as doctors gained experience. In June 2025, the agency dropped its special safety program, known as a REMS, for six approved CAR-T products. It also cut the no-driving period after treatment from eight weeks to two. The minimum time patients should stay near the treatment center fell from four weeks to two, according to OncLive.

That change lands differently in Cincinnati. Cincinnati Children’s says its patients travel from all over the country, and it points families to the Ronald McDonald House a few steps away. By The Cincinnati Exchange’s reading of the new labels, an out-of-town family now faces half the minimum stay near the hospital that the old labels called for. For parents juggling jobs and other kids, two weeks versus four is a real difference.

Cincinnati Children’s is also testing a gentler design. In April, the hospital agreed to lead the global pediatric trial of Atla-cel, a CD19 CAR-T from New Zealand’s BioOra. The trial covers children and teens with relapsed B-cell acute lymphoblastic leukemia. An adult study presented in 2024 showed low rates of serious cytokine release syndrome and markedly reduced neurotoxicity, according to BioOra. Dr. Stella Davies, the trial’s principal investigator, said that if the profile holds in children, “it could mean bringing life-changing CAR-T therapy to more kids.”

The goal, then, is not simply to make CAR-T cells stronger. It’s to make them powerful when needed and controllable when necessary.

6. CAR-T Cell Therapy Beyond Cancer

Perhaps one of the most surprising developments is the use of CAR-T cell therapy to treat autoimmune diseases. In diseases such as lupus, the immune system mistakenly attacks the body’s own tissues. One possible strategy uses CAR-T cells to eliminate certain disease-causing B cells, which are part of the immune system.

Small clinical studies have produced striking results. In 2024, doctors in Erlangen, Germany, reported in the New England Journal of Medicine on 15 patients with lupus, myositis or systemic sclerosis. All eight lupus patients reached remission, and all 15 stopped their immunosuppressive drugs, over a median follow-up of 15 months. In some cases, the treatment appears to have substantially “reset” their immune systems.

Lupus has been one of the most closely studied diseases, but researchers are also investigating CAR-T approaches for other autoimmune conditions.

These results are exciting, but the studies remain small, and no CAR-T has FDA approval for an autoimmune disease yet. The first could come from Kyverna Therapeutics. The company plans to complete its application for stiff person syndrome, a rare neurological autoimmune disorder, in the fourth quarter of 2026.

What Comes Next for CAR-T in Cincinnati

CAR-T is becoming smarter, more flexible and potentially easier to deliver. The biggest challenge now is turning these promising ideas into treatments that prove safe, effective, affordable and available to large numbers of patients.

If researchers succeed, CAR-T could become much more than a treatment for certain blood cancers. It could become a broader platform for precisely programming the immune system to fight cancer, and perhaps even to correct diseases caused by a misdirected immune system.

Some of those tests will run through Cincinnati. Tempest plans to start its U.S. registrational study of TPST-2003 in the fourth quarter of this year. BioOra’s pediatric trial will enroll at sites in the U.S. and New Zealand, with Cincinnati Children’s in charge. And by early 2027, the hospital expects its Sharonville clean rooms to start onsite manufacturing.

FAQs

What is CAR-T cell therapy?

CAR-T cell therapy is a cancer treatment that collects a patient’s own T cells, genetically adds a chimeric antigen receptor that recognizes a marker on cancer cells, and infuses the cells back into the patient. There, the cells multiply and attack the cancer. The FDA approved the first CAR-T product, Kymriah, in August 2017.

Where can patients get CAR-T cell therapy in Cincinnati?

Cincinnati Children’s offers Kymriah for eligible children and young adults and says it took part in the trial behind that drug’s FDA approval. For adults, UC Health provides CAR-T treatment through its cancer center, which Gilead has certified as a designated CAR-T center. Eligibility depends on the cancer type and a patient’s treatment history.

How long do CAR-T patients have to stay near the hospital?

Under labels the FDA updated in June 2025, patients who receive one of six approved CAR-T products should stay near the treatment center for at least two weeks, down from four. The no-driving period after treatment fell from eight weeks to two. Individual care teams may still set their own requirements.

Is CAR-T approved for lupus or other autoimmune diseases?

No CAR-T product has FDA approval for an autoimmune disease as of September 2026. A 2024 New England Journal of Medicine report on 15 patients in Germany showed remissions in lupus and related conditions. Kyverna Therapeutics plans to complete an FDA application for stiff person syndrome in the fourth quarter of 2026.

What is Cincinnati Children's doing with new CAR-T therapies?

Cincinnati Children’s agreed in April 2026 to lead a global pediatric trial of BioOra’s Atla-cel, a CD19 CAR-T designed to cause fewer neurological side effects. Its Sharonville Applied Gene and Cell Therapy Center is also Tempest Therapeutics’ manufacturing partner for TPST-2003, a dual-target CAR-T for multiple myeloma.