Key TakeawaysCAR-T therapy has revolutionized care for certain blood cancers, providing treatment options beyond chemotherapy.Evolving research shows CAR-T may also successfully treat other blood cancers and solid tumors.Future uses of CAR-T might include noncancerous conditions, too.

Key Takeaways

CAR-T therapy has revolutionized care for certain blood cancers, providing treatment options beyond chemotherapy.Evolving research shows CAR-T may also successfully treat other blood cancers and solid tumors.Future uses of CAR-T might include noncancerous conditions, too.

CAR-T therapy is one of the newest and buzziest treatments for leukemia and lymphoma,hailed a potential cancer “cure” earlier this yearfor its long-term ability to keep patients cancer-free.

The FDA approved the first CAR-T therapy in 2017,and early outcomes show that it has the potential to be effective and well-tolerated for many individuals who do not respond to existing traditional treatments.

The question is: Whatelsecan CAR-T do? Ongoing research shows the technology has the potential to expand to other conditions—like tumorous cancers and noncancerous diseases.

An Overview of CAR-T Therapy

What Is CAR-T?

For patients with certain types of blood cancers that do not respond to chemotherapy or recur after treatment, chimeric antigen receptor T-cell therapy (CAR-T) offers another possible treatment course.

“The immune system is great at patrolling cancers,”Lee Greenberger, PhD, Chief Scientific Officer for theLeukemia and Lymphoma Society(LLS), told Verywell. “As you get older, unfortunately, the immune system becomes less effective. We are replacing immunotherapy for what nature does.”

T cells, a type of white blood cell, are one of the body’s primary defenses against infection and cancer. CAR-T therapy transforms a person’s native T cells into cancer fighters. It relies on four steps:

What Is Leukapheresis?

Because the genetically altered T cells can live in the body and remain active for years,CAR-T is a one-and-done approach to fighting cancer. Patients do not need to repeat treatments as they do with chemotherapy.

CAR-T Is Currently Approved to Treat Leukemia and Lymphoma

As of March 2022,six CAR-T therapies have FDA approvalto treat cancers that recur after treatment or do not respond to other therapies:

How Lymphoma Is Treated

Promising Research for Other Blood Cancers

Current CAR-T therapies program T cells to attach to one of two markers found on many blood cancer cells: CD-19 or BCMA.However, other markers also play a role in many blood cancers.

Researchers at Fred Hutchinson Cancer Center have been studying an experimental CAR-T therapy calledMB-106that targets a blood cancer marker called CD20. In addition to treating patients with B-cell non-Hodgkin lymphoma like some of the FDA-approved CAR-T drugs, MB-106 may also be used to treat chronic lymphocytic leukemia (CLL).

“Each CAR-T construct has a target on a lymphoma cell that it goes for,” Mazyar Shadman, MD, MPh, associate professor of the clinical research division at Fred Hutch Cancer Center, told Verywell. “We’re aiming for a different target than other CAR therapies, which gives us another way of targeting and attacking lymphoma cells.”

How CAR T-Cell Therapy ‘Cured’ 2 Cancer Patients

Targeting alternative markers may also be useful when existing CAR-T treatments fail. Shadman noted only 30% to 40% of patients are cured from current CAR-T treatments.

“[MB-106] covers a space that is considered to be an unmet need in lymphoma treatment, including patients who have already received CD-19-directed CAR T,” he said.

Shadman said his research team is particularly hopeful that MB-106 will prove effective at treatingWaldenstrom macroglobulinemia(WM), a rare subtype of non-Hodgkin lymphoma.

Next Stop: Treating Other Cancers

LLS’s Greenberger said it’s possible CAR-T can be used to treat cancer beyond leukemia and lymphoma, which are blood cancers.

“I suspect that what we learn about blood cancers will also apply to solid tumors,” he said. For example, a recent study suggests that some breast cancers might respond to CAR-T therapy.

While all currently FDA-approved CAR therapies modify the T cells (and are thus known as CAR-T), some CAR therapies in development will employ other cells within the immune system. This includes natural killer (NK) cells and macrophages, expanding the potential reach of the treatment.

CAR-T May Have Uses Beyond Cancer, Too

CAR-T therapies may also be an option for some noncancerous illnesses in the future. Right now, researchers are exploring its use across a variety of conditions:

Will CAR-T Become a First-line Treatment Option?

CAR-T therapy is still in its infancy, and much remains unknown. CAR-T could become the first treatment of choice for some patients in the future, but for the time being, many traditional treatment options remain the gold standard.

“We know the safety profiles of the FDA-approved CAR-T therapies, but nobody should be giving CAR-T for conditions it’s not approved to treat,” Greenberger said. “It will take more time to get current CAR-T therapies approved as first-line treatment. Trials must prove first that these treatments are both efficacious and safe. They are also expensive, so you have to weigh their benefits against the cost of traditional treatment.”

High-quality research takes time, and many CAR-T researchers often work with a limited subject pool.

“Some of the conditions we are studying are so rare it takes a while to get the data we need,” Greenberger said. “But I anticipate that in the next 5 or 10 years, you will see CAR-T move from the third-line course of action to first or second-line treatment.”

In the meantime, researchers are excited to see the potential CAR-T therapies hold for patients.

9 SourcesVerywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.Food and Drug Administration.FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome.Leukemia & Lymphoma Society.Chimeric antigen receptor (CAR) t-cell therapy.National Cancer Institute.CAR T cells: engineering immune cells to treat cancer.National Library of Medicine.Study to assess safety, tolerability and efficacy of MB-106 in patients with relapsed or refractory B-Cell NHL or CLL.Pan K, Farrukh H, Chittepu VCSR, Xu H, Pan CX, Zhu Z.CAR race to cancer immunotherapy: from CAR T, CAR NK to CAR macrophage therapy.J Exp Clin Cancer Res. 2022;41(1):119. doi:10.1186/s13046-022-02327-zShrestha B, Walton K, Reff J, et al.Human CD83-targeted chimeric antigen receptor T cells prevent and treat graft-versus-host disease.J Clin Invest. 2020;130(9):4652-4662. doi:10.1172/JCI135754Mougiakakos D, Krönke G, Völkl S, et al.CD19-targeted CAR T cells in refractory systemic lupus erythematosus.N Engl J Med. 2021;385(6):567-569. doi:10.1056/NEJMc2107725Gao TA, Chen YY.T cells to fix a broken heart.Science. 2022;375(6576):23-24. doi:10.1126/science.abn0851Seif M, Kakoschke TK, Ebel F, et al.CAR T cells targetingAspergillus fumigatusare effective at treating invasive pulmonary aspergillosis in preclinical models.Sci Transl Med. 2022;14(664):eabh1209. doi:10.1126/scitranslmed.abh1209

9 Sources

Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.Food and Drug Administration.FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome.Leukemia & Lymphoma Society.Chimeric antigen receptor (CAR) t-cell therapy.National Cancer Institute.CAR T cells: engineering immune cells to treat cancer.National Library of Medicine.Study to assess safety, tolerability and efficacy of MB-106 in patients with relapsed or refractory B-Cell NHL or CLL.Pan K, Farrukh H, Chittepu VCSR, Xu H, Pan CX, Zhu Z.CAR race to cancer immunotherapy: from CAR T, CAR NK to CAR macrophage therapy.J Exp Clin Cancer Res. 2022;41(1):119. doi:10.1186/s13046-022-02327-zShrestha B, Walton K, Reff J, et al.Human CD83-targeted chimeric antigen receptor T cells prevent and treat graft-versus-host disease.J Clin Invest. 2020;130(9):4652-4662. doi:10.1172/JCI135754Mougiakakos D, Krönke G, Völkl S, et al.CD19-targeted CAR T cells in refractory systemic lupus erythematosus.N Engl J Med. 2021;385(6):567-569. doi:10.1056/NEJMc2107725Gao TA, Chen YY.T cells to fix a broken heart.Science. 2022;375(6576):23-24. doi:10.1126/science.abn0851Seif M, Kakoschke TK, Ebel F, et al.CAR T cells targetingAspergillus fumigatusare effective at treating invasive pulmonary aspergillosis in preclinical models.Sci Transl Med. 2022;14(664):eabh1209. doi:10.1126/scitranslmed.abh1209

Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.

Food and Drug Administration.FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome.Leukemia & Lymphoma Society.Chimeric antigen receptor (CAR) t-cell therapy.National Cancer Institute.CAR T cells: engineering immune cells to treat cancer.National Library of Medicine.Study to assess safety, tolerability and efficacy of MB-106 in patients with relapsed or refractory B-Cell NHL or CLL.Pan K, Farrukh H, Chittepu VCSR, Xu H, Pan CX, Zhu Z.CAR race to cancer immunotherapy: from CAR T, CAR NK to CAR macrophage therapy.J Exp Clin Cancer Res. 2022;41(1):119. doi:10.1186/s13046-022-02327-zShrestha B, Walton K, Reff J, et al.Human CD83-targeted chimeric antigen receptor T cells prevent and treat graft-versus-host disease.J Clin Invest. 2020;130(9):4652-4662. doi:10.1172/JCI135754Mougiakakos D, Krönke G, Völkl S, et al.CD19-targeted CAR T cells in refractory systemic lupus erythematosus.N Engl J Med. 2021;385(6):567-569. doi:10.1056/NEJMc2107725Gao TA, Chen YY.T cells to fix a broken heart.Science. 2022;375(6576):23-24. doi:10.1126/science.abn0851Seif M, Kakoschke TK, Ebel F, et al.CAR T cells targetingAspergillus fumigatusare effective at treating invasive pulmonary aspergillosis in preclinical models.Sci Transl Med. 2022;14(664):eabh1209. doi:10.1126/scitranslmed.abh1209

Food and Drug Administration.FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome.

Leukemia & Lymphoma Society.Chimeric antigen receptor (CAR) t-cell therapy.

National Cancer Institute.CAR T cells: engineering immune cells to treat cancer.

National Library of Medicine.Study to assess safety, tolerability and efficacy of MB-106 in patients with relapsed or refractory B-Cell NHL or CLL.

Pan K, Farrukh H, Chittepu VCSR, Xu H, Pan CX, Zhu Z.CAR race to cancer immunotherapy: from CAR T, CAR NK to CAR macrophage therapy.J Exp Clin Cancer Res. 2022;41(1):119. doi:10.1186/s13046-022-02327-z

Shrestha B, Walton K, Reff J, et al.Human CD83-targeted chimeric antigen receptor T cells prevent and treat graft-versus-host disease.J Clin Invest. 2020;130(9):4652-4662. doi:10.1172/JCI135754

Mougiakakos D, Krönke G, Völkl S, et al.CD19-targeted CAR T cells in refractory systemic lupus erythematosus.N Engl J Med. 2021;385(6):567-569. doi:10.1056/NEJMc2107725

Gao TA, Chen YY.T cells to fix a broken heart.Science. 2022;375(6576):23-24. doi:10.1126/science.abn0851

Seif M, Kakoschke TK, Ebel F, et al.CAR T cells targetingAspergillus fumigatusare effective at treating invasive pulmonary aspergillosis in preclinical models.Sci Transl Med. 2022;14(664):eabh1209. doi:10.1126/scitranslmed.abh1209

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