Cell Remedy Varieties, Makes use of, and Future Impacts

This article is sponsored by BPS Bioscience. Learn more about the T cell immunotherapy landscape.

Blood transfusion marked the earliest form of cell therapy, which progressed significantly after Edward Donnall Thomas pioneered allogeneic hematopoietic stem cell transplantation (HSCT) to treat blood disorders in 1957.^1,2 Since then, researchers have rapidly advanced the cell therapy field, developing modern treatments such as chimeric antigen receptor T cell (CAR-T) therapy that offer promising outcomes for patients with previously untreatable conditions.³ This article explores the various cell types used for cellular therapeutics, regulatory considerations for scientists developing cell therapies, and clinical applications.

What Is Cell Therapy?

Cell therapy is an advanced biomedical approach that utilizes living cells as therapeutic agents to treat disease or repair tissue.⁴ Scientists isolate cells from a patient or a healthy donor and then manipulate and culture these cells under controlled laboratory conditions to endow them with specific functional properties. Clinicians may reintroduce therapeutically engineered cells into a patient using techniques ranging from minimally invasive injections to complex surgical implantations.

Autologous Versus Allogeneic Cell Therapy

Scientists categorize cell therapies into two main types: autologous and allogeneic. Each approach offers distinct benefits and challenges, shaping treatment options for various conditions.

Autologous cell therapy

Autologous cell therapy involves collecting a person’s own cells or tissues, processing them outside of the body, and then reintroducing them into the same individual.⁵ Scientists can use this strategy to develop highly personalized treatments tailored to the individual’s unique needs. In autologous CAR-T immunotherapy, researchers or medical professionals collect T cells from a patient and genetically engineer the cells to express CARs that recognize tumor antigens.³ Safe and effective CAR T cells can be reinfused into the patient, mounting an immune response that destroys cancer cells expressing a specific target antigen.

By using the patient’s own T cells, clinicians reduce the risk of immune reactions against donor cells.⁶ Another advantage of autologous cell therapy is that these cells may persist in the patient’s body for months or even years, potentially providing long-term therapeutic effects.

Despite its promise, autologous cell therapy faces significant challenges due to sample scarcity, which limits its use for diseases that affect large populations. Additionally, logistical complexities during cell collection, reinfusion, and transportation to and from manufacturing centers increase the risk of cellular damage, loss, and cross-contamination.⁶

Allogeneic cell therapy

Allogeneic cell therapy uses cells from a healthy donor rather than the recipient.⁶ Scientists may generate allogeneic therapeutics from peripheral blood mononuclear cells (PBMCs), induced pluripotent stem cells (iPSCs), or umbilical cord blood, with varying levels of ease and clinical applications.⁷ They use genetic engineering and specific culture methods for each cell type to achieve optimal function. In addition to conventional allogeneic CAR-T cell therapy, scientists are actively evaluating the effectiveness of CAR-natural killer (NK) cells, CAR-gamma delta (γδ) T cells, and macrophages in clinical trials.

Allogeneic cell therapy offers several advantages over autologous approaches, including better suitability for off-the-shelf production. Researchers may be able to genetically engineer batches of healthy donor cells for a desired therapeutic effect, store them in cell banks, and ship the products as needed.⁸ Off-the-shelf therapies help patients receive treatment more quickly, which is especially valuable for those with aggressive or rapidly progressing diseases. In addition, allogeneic cell therapy development is more amenable to automated and scaled up production, ultimately reducing costs and making these therapies more accessible.⁹

Despite these advantages, allogeneic cell therapy faces two major challenges; namely, graft-versus-host disease (GvHD), in which donor immune cells attack the recipient’s tissues, and host-mediated allorejection, where the recipient’s immune system rejects donor cells.⁸

Cell Therapy Types and Applications

Scientists select specific cell types for therapeutic development based on their unique functions. Additionally, researchers classify cell therapies by the type of cell involved: stem cell-based, non-stem cell-based, or multicellular.⁴ Key cell types for cell therapy production and their applications include the following.

• Hematopoietic stem cells (HSCs): Researchers use HSCs from bone marrow or umbilical cord blood to regenerate blood and immune cells, especially for treating hematological cancers such as leukemia and lymphoma.¹⁰
• iPSCs: Scientists reprogram adult cells into iPSCs, which can then develop into nearly any cell type. They use these cells to create patient-specific therapies and disease models.¹¹
• Embryonic stem cells (ESCs): Researchers derive ESCs from early-stage embryos and use them for their potential to become any cell type, exploring treatments for degenerative diseases.¹²
• CAR T cells: Scientists engineer T cells to express chimeric antigen receptors and use these CAR T cells to target and destroy specific cancer cells.¹³
• Mesenchymal stem cells (MSCs): Scientists primarily derive MSCs from adult bone marrow and adipose tissue. Clinical trials have demonstrated that these cells effectively treat severe degenerative and inflammatory diseases.¹⁴

Stem cell-based cell therapies

Pluripotent stem cells (PSCs) such as iPSCs, ESCs, and epiblast stem cells can be differentiated into any cell type. In contrast, the therapeutic potential of adult stem cells, including HSCs and mesenchymal stem cells (MSCs), lies in their natural ability to differentiate into specialized cell types that replenish damaged or aged cells, particularly in regenerative medicine research.

Non-stem cell-based cell therapies

Non-stem cell-based therapies rely on specialized somatic cells, including fibroblasts, chondrocytes, keratinocytes, hepatocytes, pancreatic islet cells, and immune cells to treat, prevent, or diagnose disease.⁴ Researchers isolate these cells from specific tissues using enzymatic digestion or blood processing, and often further manipulate or treat them before introducing the cells into patients. Clinicians use somatic cell-based therapies to replace damaged cells and correct metabolic disorders, as with hepatocyte or pancreatic islet cell transplants. Non-stem cell-based cell therapies may also be used to promote tissue repair in wounds and cartilage using scaffold-based and scaffold-free systems.

Multicellular therapies

Multicellular therapies combine at least two different types of stem or non-stem cells.⁴ Researchers generate these therapies by selectively expanding desired cell populations, often using automated cell-processing technologies rather than traditional purification methods. The variety of cell types in multicellular therapies provides a broad range of biological activities, creating complex mechanisms of action that often resemble natural tissue function. Examples of multicellular therapies include adoptive cell transfer products, scaffold-based and scaffold-free cellular therapies, stromal vascular fraction, stem cell transplants, and bone marrow aspirate-derived treatments.

Regulatory Challenges for Cell Therapy

Many countries have approved cell therapies for treating various diseases. For example, the US, UK, countries in the EU, and Japan have approved types of CAR-T cell therapies for treating cancers such as relapsed or refractory multiple myeloma. Japan has also approved an epithelial cell-based treatment for limbal stem cell deficiency.¹⁵

As live drugs, it is especially crucial that all cell therapies first undergo rigorous safety and efficacy assessments before approval. Major regulatory agencies have issued specific regulations and guidelines to support the development of cell therapy products for human use.¹⁶ These guidelines ensure high standards for quality, safety, and efficacy. Regulatory bodies oversee the development, testing, and marketing of cell therapy products to ensure timely access to safe and innovative treatments.

In contrast to traditional small-molecule or biological drugs, cell therapy applications encounter more regulatory objections, which delay market approval and clinical adoption.¹⁷ These objections often stem from issues linked to preclinical factors, such as animal study design, model selection, study endpoints, and therapeutic mechanism of action. Additionally, because expanding, editing, and differentiating stem cells in vitro can leave residual undifferentiated or transformed cells, it is imperative that cell therapy developers evaluate tumorigenicity before clinical trials.¹⁸

Emerging Trends and Future Perspectives in Cell Therapy

Scientists are actively exploring new cell types to diversify the landscape of cell-based treatments.¹⁹ Continued innovation and cross-disciplinary collaboration drive cell therapies to transform medicine, opening new avenues for cures and better patient outcomes across many diseases.

Advances in cell engineering, manufacturing technologies, and cell biology fuel a highly promising outlook for cell therapies.²⁰ Researchers develop novel approaches, including tunable genetic modifications and optimized delivery systems, to improve safety and therapeutic effectiveness. Automation and decentralized manufacturing lower production costs and improve scalability, making these therapies more accessible to patients worldwide.

As regulatory frameworks mature and standardization improves, scientists can efficiently translate laboratory discoveries into clinical applications. These advances accelerate the approval and adoption of next-generation cell therapies, especially for complex or previously untreatable diseases.