Cellular and Gene Therapies in Multiple Myeloma: Standard of Care and Future Directions
- 1. Division of Hematology, The Ohio State University Comprehensive Cancer Center, USA
Abstract
Background: Multiple myeloma (MM) remains incurable despite >10 new drugs since 2015. Engineered cellular therapies may eradicate resistant plasma-cell clones.
Objective: To summarize current evidence on cellular and gene therapies in MM and identify major knowledge gaps and future directions.
Methods: PubMed, CENTRAL, ClinicalTrials.gov, and ASH/ASCO/ESMO abstracts from 2020–2025 were searched for preclinical and phase I–III studies of cellular or gene-engineered therapies for MM, including CAR-T, CAR-NK, γδ T-cell, and TCR-engineered products. Twelve studies met eligibility criteria, including 11 clinical studies and 1 preclinical study.
Results: FDA-approved CAR-T products ide-cel and cilta-cel achieve >70 % ORR. Memory-enriched, Early-phase studies of memory-enriched, fully human, and T stem cell memory–enriched constructs have reported ORRs ranging from 48% to 100%, although cross-trial comparisons are limited and longer follow-up is needed to assess persistence and safety. Allogeneic BCMA CAR-T, iPSC- BCMA CAR-NK and anti-CD38 γδ-CAR-T exhibit low acute toxicity yet limited in-vivo durability. Non-BCMA or dual-antigen CARs deliver high ORR, while TCR-T is active in HLA-A*02:01-positive disease.
Conclusions: BCMA-directed CAR-T therapy is an established treatment option in RRMM. Broader integration of cellular therapies will require randomized comparative trials, biomarker-driven patient selection, long-term safety registries for gene-edited products, faster manufacturing, and improved cost-effectiveness
Keywords
• Multiple myeloma
• CART product
• Gene editing
• Gene engineered T cells
citation
: Umyarova E (2026) Cellular and Gene Therapies in Multiple Myeloma: Standard of Care and Future Directions. Ann Clin Pathol 13(1): 1182.
INTRODUCTION
Multiple myeloma (MM) accounts for about 1% of all cancers and it is the second most common hematological malignancy after lymphoma. MM develops when post-germinal-center B-cells acquire certain somatic chromosomal mutations, such as t(11;14), t (4;14) or del17p, and 1q gain, leading to clonal plasma-cell proliferation within the bone-marrow. Malignant plasma cells oversecrete monoclonal immunoglobulin and free light chains that cause end-organ damage – hypercalcemia, renal failure, anemia and bony lesions. Commonly presenting symptoms include bone/back pain, fatigue, recurrent infections and pathological fractures collectively called CRAB criteria. Diagnosis of MM requires Diagnosis of MM requires clonal bone marrow plasma cells ≥10% or biopsy-proven plasmacytoma, plus one or more myeloma-defining events. These include CRAB features attributable to the plasma-cell disorder or specific biomarkers of malignancy, such as clonal marrow plasma cells ≥60%, involved/uninvolved serum free light-chain ratio ≥100, or more than one focal lesion on MRI., more than 60 %
of clonal plasma cells, serum FLC ratio more than 100, or more than 1 focal bony lesions on MRI. The later criteria were added by International Myeloma Working Group in 2014 [1].
MM is the disease of the elderly with the median age at diagnosis of 69 years old with more than 95% of the cases occurring in patients above 50 years old. Men and African Americans have higher incidence than women and Whites [2]. According to the American Cancer Society, an estimated 36,110 new cases of multiple myeloma are diagnosed in the United States each year. Patients with high-risk cytogenetics (del17p, t(4;14), 1q gain) or plasma-cell leukemia have more aggressive disease biologically and clinically with the short duration of response, PFS and OS [3].
Virtually all symptomatic MM is preceded by an asymptomatic precursor state, such as MGUS and smoldering multiple myeloma. of uncertain clinical significance (MGUS) and smoldering MM and first-degree relatives of the patients with known MM diagnosis carry a 2- to 4-fold increased risk compared to general population [4]. At current state of science effective chemoprevention is not defined, although there are populations studies, one of most famous is called “I StopMM”, that are testing for MGUS screening and early interventions [5].
Despite more than ten FDA approvals since 2015, MM remains incurable. Deep and durable remissions are rare, cumulative toxicities and the financial burden increase with each subsequent line, and researchers are in need to develop new targets to control MM and search for treatment options to overcome the drug resistance
BACKGROUND
MM arises from clonally expanded plasma cells, that synthesize large amount if immunoglobulin, depend on proteasome-mediated protein disposal and remodel the bone marrow microenvironment to suppress immunity. Therefore, current standard of care front-line therapies rely on blocking proteosomal degradation, direct cytotoxicity and antibody-dependent cellular cytotoxicity in order to successfully lower the clonal cell population. Front line regimens include triplet or quadruplet chemotherapy combinations - proteasome inhibitors (PI), eg. bortezomib, immunomodulatory drugs (IMiDS), eg. Lenalidomide, with or without anti-CD38 mAB, eg. Daratumumab, plus corticosteroids with subsequent early autologous stem-cell transplant autologous hematopoietic cell transplantation and maintenance lenalidomide. autologous hematopoietic cell transplantation is the reinfusion of the autologous stem cells that is intended to prolong PFS, and is an effective option especially for HRMM patients, although it would be a poor option for older and frail patients.
Relapsed/refractory (RRMM) disease develops over time in most of the patients with various times for disease progression mainly depending on the genetic risk for the disease. RRMM is managed with consecutive use of next-generation PIs, IMiDs, anti-CD38, BCMA, anti-SLAMF7 antibodies, exportin-1 inhibitor selinexor, bispecific T-cell engagers, and alkylators [15], each line leading to lower depth and duration of response with increased cumulative toxicity. The main mechanisms of action for those medications include inhibition of proteasome units, cereblon modulation and recruitment of BCMA antigen, important for survival of plasma cells.
Although these regimens have extended median overall survival (mOS) beyond six years, every patient eventually becomes “class–refractory,” at which point overall survival (OS) drops below one year. Outcomes worsen substantially once patients develop triple-class-refractory disease, defined as refractoriness to a proteasome inhibitor, an IMiD, and an anti-CD38 monoclonal antibody. Reported median overall survival is approximately 8–9 months, and may fall to 5–6 months in penta-refractory disease. [6]. Together, these statistics highlight the pressing need for more durable, less toxic therapeutics in RRMM. An ideal treatment strategy would eradicate resistant plasma-cell clones while restoring durable immune surveillance. highly resistant plasma-cell clones and re-establish long-term immune control.
Cell and gene therapies, especially CAR-T cells, are showing promise in fulfilling the need for effective treatment in RRMM. There are 2 BCMA-directed autologous CAR-T products — idecabtagene vicleucel (Abecma) and ciltacabtagene autoleucel (Carvykti), that had been approved by FDA in 2021 and 2022, respectively, with one-time infusions achieving more than 70–95 % overall response rate (ORR) in heavily pre-treated RRMM with durable duration of response (DoR) up to 35 months [7,8].
Utilizing the same or similar concept, several next-generation cell therapy treatment modalities were constructed to attempt to prove safety and efficacy. These treatment modalities are currently in the various stages of the clinical trials and not yet FDA approved for commercial use (Table 1).
Table 1: Several next-generation cell therapy treatment modalities were constructed to attempt to prove safety and efficacy
|
Allogeneic off-the-shelf CAR-T, eg. Allo-715 |
designed to shorten vein-to-vein time and improve access for patients who have no time to wait for 4-6 weeks for autologous CART processing [9] |
|
Dual-target CARTs, for example BCMA/CD19 CART cell products using fasTCAR platforms, |
allow to recruit additional cell surface targets for high-risk MM (HRMM) and shorten the processing times [10] |
|
A non-BCMA CART constructs for example GPRC5D – CART cells |
Are utilizing additional antigens on plasma cell surface rather than already saturated BCMA space [11] |
|
Engineered natural-killer (CAR-NK) and γδ-T cells |
They have less systemic toxicity while providing antimyeloma effects [12] |
|
CRISPR and other gene-editing strategies |
yet to gain more attention in MM but available data in gene editing is promising, allowing T cells to facilitate expansion and persistence and, eventually, potency [13]. |
|
TCR-engineered T cells |
This treatment modality intends to attack MM without utilizing the surface antigens and might be a promising strategy for highly RRMM that is lacking surface antigens [14]. |
METHODS/LITERATURE SEARCH
The literature search was performed on May 15, 2025, across three electronic databases PubMed, Cochrane CENTRAL, and ClinicalTrials.gov as well as three conference annals - American Society of Hematology, American Society of Clinical Oncology and European Society of Medical Oncology. Each source was investigated from January 1, 2015, to May 15, 2025. Original articles or conference papers with completed pre-clinical translational or phase I–III clinical data were included into the search that included search terms “engineered cellular or gene-edited product directed against multiple myeloma”. Terms autologous or allogeneic CAR-T, CAR-NK, TCR-T, CRISPR-modified cells were included. Studies with at least one efficacy endpoint- ORR, PFS, MRD were included in search. Ongoing trials without posted results were excluded, as well as bispecific antibodies or cytokine therapies without a gene-modified cell product. Only primary study literature was included in this report with no addition of reviews, editorials or non-myeloma malignancies. Data extraction included study aim, study design, product characteristics, population, and key outcomes.
784 records were evaluated and after removal of 148 duplicates, 636 titles were identified and 538 were excluded, leaving 98 full-text articles. 86 did not meet search criteria (filtered through lack of results, insufficient data, disease type, type of therapy), leaving researcher with 12 studies -11 clinical and 1 preclinical studies for the detailed analysis (Figure 1) (Table 2 and Table 3).
Figure 1 Figure presents a flowchart.
Our search was confined to English written articles only, some studies lack a fully published manuscripts and only included conference abstracts, studies with negative results might be underrepresented. Our cut-off date is May 15, 2025, so studies listed after this date were not included in this manuscript.
Table 2: Integrated review chart table
|
Citation |
Study aim |
Study design |
Study population |
Key findings |
|
Munshi NC et al., 2021 KarMMa [16] |
Evaluate efficacy and safety of autologous BCMA-CAR-T in triple-class–refractory MM |
Phase 2, open-label, single-arm, multicenter clinical trial |
128 adult patients with median 6 lines of prior therapy, refractory to PI; IMiDs and anti- CD38-therapy |
The key findings: ORR 73 %; ≥CR 33 %; median PFS 8.8 mo; Gr ≥3 CRS 5 %, ICANS 3 % |
|
Berdeja J G et al., 2021 CARTITUDE-1 [17] |
Determine safety and activity of dual-epitope BCMA- CAR-T |
Phase 1/2, single-arm, multicenter clinical trial |
97 patients with RRMM and 6 median prior lines of therapy |
Key findings: ORR 97 %; sCR 67 %; Gr ≥3 CRS 4 %, ICANS 9 % |
|
Alsina et al., 2020 CRB-402 [8] |
Test memory-enriched BCMA-CAR-T for clinical outcomes like DoR |
Phase 1 dose escalation/ expansion |
68 patients with RRMM with median 6 prior lines of therapy; 57% triple refractory |
Key findings: ORR 48 %; ≥CR 18%; mDOR 11.9 mo |
|
Mailankody S et al., 2020 EVOLVE [19] |
Assess fully human scFv BCMA-CAR-T for rsafety and efficacy |
Phase 1, open label |
51 patients with RRMM > 3 lines of therapy |
Key findings: ORR 91 %; ≥CR 39 %; mPFS 9.3 mo; Gr ≥3 CRS 2 % NE ≥ 3: 4% |
|
Wang D et al., 2021FUMANBA-1 [20] |
Determine activity of fully human BCMA-CAR-T (CT103A) and determine its safety and efficacy |
Phase 1, single arm |
18 patients with RRMM including patients previously exposed to murine BCMA-directed CAR constructs. |
Key findings: ORR 100 %; sCR 72.2 %; 12-mo PFS 58.3 %; |
|
Mailankody S et al., 2023 UNIVERSAL [9] |
First-in-human off-the-shelf. allogeneic BCMA-CAR-T allo 715 evaluated for safety and tolerability |
Phase 1, dose-escalation |
43 patients with RRMM |
Key findings: ORR 55.8 % mDoR 8.3 mo; Gr ≥3 CRS 2.3 %, no Gr ≥3 ICANS |
|
Mailankody et al., 2022 MCARH109 [11] [23] |
Target BCMA-independent antigen to prevent escape - GPRC5D CAR-T |
Phase 1, first-in-human |
17 patients with RRMM, heavily pre-treated, including relapsed post BCMA CART |
Key findings: ORR 71 %; |
|
Qiang W et al., 2024 GC012F [10] |
Test dual-antigen CAR-T targeting BCMA/CD19 for deeper MRD negativity |
Phase 1 single arm, open label, single center |
22 patients with NDMM |
Key findings: ORR 100 %; robust CART cell expansion with peak copy numbers at 10 days (9-14) |
|
Gregory et al., Tscm CART [21] |
Evaluate high purity engineered BCMA-CAR-T with T stem cell memory phenotype |
Phase 1 single arm, open label |
4 patients with RRMM more than 3 lines of therapy |
Key findings: Favorable safety profile with no increase in CRS biomarkers. |
|
Dhakal et all FR576 [12] |
First of off-the-shelf iPSC-derived BCMA CAR-NK assessment of safety and tolerability |
Phase 1, dose-escalation |
9 patients with RRMM |
Key findings: no dose limiting toxicities, no CRS, ICANS or GVHD |
|
Rapoport AP et al., 2015 NY-ESO-1 TCR-T post-ASCT [14] |
Explore engineered TCR-T targeting intracellular antigen to investigate safety and activity |
Phase I/II |
20 antigen-positive MM pts post-ASCT |
Key findings near CR/sCR 70 %; mPFS 19 mo; mOS 32.1 mo; no CRS/ICANS |
|
Hattori Y., 2025 Anti-CD38 γδ-CAR-T [13] |
Develop γδ-T CAR-T for off-the-shelf usevin tandem with allogeneic transplantation |
Preclinical |
in-vivo immunodeficient mice models; ex-vivo - patient samples |
Potential antitumor effects in vivo, suppressing tumor growth. |
Table 3: Study Evaluation
|
Citations |
Strengths |
Limitations |
Future work |
|
Munshi NC et al., 2021 KarMMa [7] Methodological journal (IF > 100) |
The strengths of KarMMa study include multicenter, international design, prospective study, correlative work on biomarkers and dose exploration; no control arm leading to selection and confounding bias, open label |
Single arm study, small lowest dose cohort, short follow up on overall survival, limited racial and ethnic diversity, no quality- of-life measures and no reports on manufacturing. Powered to ORR, but not OS, underpowered subgroup and dose level cohorts |
Studies on increased persistence mitigation of toxicity long term safety |
|
Berdeja J G et al., 2021 CARTITUDE-1 [17] (Journal with IF~ 28) |
Multicenter clinical trials, Investigates the treatment for highly refractory population, constructs included dual-epitope BCMA, they have deep and durable efficacy endpoints Single arm, sponsor open leading to potential reporting bias |
Single arm, open design, relatively small sample size, underrepresented and short median follow up, no quality of life assessment and limited details on manufacturing, not powered to PFS. |
Evaluate for the earlier line, control of late neurotoxicity studies on combination and maintenance therapy post CART |
|
Alsina et al., 2020 CRB-402 [8] (IF >20) |
First in human, multicenter, dose escalation trial, tests hypothesis to increase persistence, good signal on safety and efficacy in heavily treated population. No comparator and short follow up |
Single arm study with relatively small sample short follow ups, complex manufacturing. US centers, might be hard to replicate |
Phase II studies, investigation in earlier line and in earlier line and in combination, attempt to simplify the manufacturing. |
|
Mailankody S et al., 2020 EVOLVE [19] (IF 45) |
This study tests fully human BCMA construct in heavily pretreated cohort with significant clinical efficacy and relatively stable safety. Since center investigator led study |
Small sample size, short follow up time to assess the efficacy, concern for late infections. Exploratory study with CI |
Randomized larger sample trials, combinations and maintenance trials. |
|
Wang D et al., 2021FUMANBA-1 [20] (IF 7) |
This study is testing fully human BCMA construct with lower immunogenicity. and higher persistence. Very good efficacy parameters and CART persistence. Single site |
Single center study, small cohort, conducted only in China where some medications are not available. High grade CRS and prolonged cytopenias. Descriptive statistics in a single site, applicability can be questioned |
How to mitigate high grade CRS, studies directed for extramedullary disease. |
|
Mailankody S et al., 2023 UNIVERSAL [9] (IF > 100) |
First study using allogeneic BCMA CART. Can potentially address the issues with autologous collections and manufacturing time, since it is off the shelf. No GVHD and severe CRS/ICANS. Complex eligibility |
Complex manufacturing processes makes it expensive; unknown long term risks of gene editing: especially secondary malignancies, limited CART persistence, less diverse patient population. Not powered for efficacy |
Studies on secondary malignances, studies on durability of response, need direct comparison trials for autologous and allogeneic CART |
|
Mailankody et al., 2022 MCARH109 [11] (short format for high IF journal) |
Testing the different CART beyond BCMA target, shows significant efficacy in high risk relapsed refractory population, might have potential combinations with BCMA CART. No comparator |
Very small population, potential dose dependent toxicity and skin and mucosa associated side effects. Exploratory and not powered study |
Need larger sample trials, limit dose related toxicities and head-to-head comparison with BITes |
|
Qiang W et al., 2024 GC012F [10] (IF ~20) |
First construct including dual CART product targeting 2 receptors: BCMA and CD19, quick manufacturing, favorable early signals on safety with no exceptional toxicity. Single center study. |
Small study sample in single center, short follow up period, no details on manufacturing. Power not specified |
Needs a bigger study, with larger sample, longer follow up study and studies on CART persistence. |
|
Gregory et all Tscm CART [21] (brief research in high IF journal) |
First in human evaluation of BCMA- CAR-T with T stem cell memory phenotype, favorable efficacy and safety profile, special effect on extramedullary disease. Very small design concern for observation bias |
Very small sample, purely a signal seeking study that is not powered |
Larger clinical trial for better definition of side effects and safety signals |
|
Dhakal et all FR576 [12] (int IF journal) |
This study tests clonally engineered NK cells – available off the shelf product, early safety profile is very favorable, this might increase accessibility being “off the shelf” product. Sponsor open labeled. |
Expensive and time- consuming manufacturing, very small sample study, small combination sample – unknown efficacy in combination with daratumumab, no data on persistence. Descriptive statistics |
Larger studies on combinations, studies evaluating the persistence of the product |
|
Rapoport AP et al., 2015 NY-ESO-1 TCR-T post-ASCT [14] |
First TCR-T product that targets intracellular cancer antigen. Favorable efficacy data, and persistence data, no contemporary control |
Might only fit a specific cohort of patients HLA - A*02:01 which is a minority of myeloma patients, single arm, small sample. Not powered beyond PFS, HLA population restricts generalizability |
Need to consider additional targets to improve generalizability, consider combinations with currently approved backbone treatments |
|
Hattori Y., 2025 Anti-CD38 γδ-CAR-T [13] |
The study uses allogeneic T cells, allowing to manufacture “off the shelf” product and increase accessibility good preclinical efficacy signaling; Unable to detect off target cytopenia risk |
Minimal human myeloma lines, no data across multiple donors. Uncertain human translation. |
Needs studies on DLT, long term persistence, distribution; considerations for clinical trials with combinations of currently approved myeloma backbone regimens and targets/ combination of targets. |
DISCUSSION/CONCLUSION
Relapsed-refractory multiple myeloma (RRMM) still defies cure, and each subsequent line of standard therapy yields shallow and shorter-lived remissions. Engineered gene- and cell-based treatments have emerged as the modalities capable of eradicating highly drug-resistant plasma-cell clones and achieving molecular-level remissions and potentially durable responses. Studies presented in this systematic review attempt to provide an overview of the current landscape of the cellular therapy in multiple myeloma.
Autologous BCMA-CAR-T studies (e.g. ide-cel, cilta-cel, bb21217, CT103A, P-BCMA-101) share several features in common [16- 20]. They include rapid cytoreduction with more than 70 % overall response even in penta-refractory disease, manageable acute toxicities when current CRS/ ICANS treatments are applied, and a clear dose–response relationship for depth and persistence. Divergence appears in the tail of the curve and likely related to the CART cell persistence – for example cilta-cel and fully-human CT103A show more than 60 % 18-month PFS, whereas ide-cel and orva-cel plateau nearer 12 months. Factors driving these gaps are yet to be learned as patient biology and correlative studies analysis vary across different trials.
“Off-the-shelf” and non-BCMA CART products share some additional heterogeneity [9-12]. Universal ALLO-715 and FT576 NK data confirm that gene-edited allogeneic products can be infused safely with single-digit severe-CRS side effect rates, yet persistence rarely exceeds 2–3 months and mDOR remains about eight months.
Next arm of non-BCMA or dual CART products - GPRC5D CAR-T (MCARH109) and dual BCMA/CD19
fasTCAR (GC012F) - demonstrate that recruiting different targets or enhancing BCMA leads to additional efficacy on RRMM, but skin and neuro-toxicities unique to GPRC5D, that happen in up to 30% pf the patients, and the uncertain incremental benefit along with the double side effect profile of dual targeting warrant additional larger studies [10, 11]. Dual CART products seek to avoid resistance with early studies reporting deeper MRD negativity but also additive cytopenia and CRS.
TCR-engineered NY-ESO-1 cells illustrate that an intracellular antigen target can achieve MRD-negative, multi-year remissions without CRS, yet generalizability is limited to HLA-A*02:01 patients, which includes minority of Western MM population [14].
Gene-edited T-cell (ALLO-715) and iPSC-derived CAR-NK (FT576) products eliminate the vein-to-vein delay and enable repeat dosing. Severe CRS or GvHD remain low, yet CAR/NK persistence rarely exceeds 2–3 months and median duration of response is approximately 8 months due to potential allo-immune rejection and lack of memory [9-12].
Additional targets under early clinical evaluation include CD38, CD138, CD229 investigate additional targets in post BCMA relapse. Clinical activity is still being investigated and the optional schedule is yet to be learned.
In addition, current research lacks knowledge on comparators and sequencing. There are no trials that would randomize against modern regimens as well as the optimal integration into sequence of therapy remains undefined in majority of the products undergoing the clinical trial. Another limitation includes long-term follow-ups. Long-term safety data assessments beyond three years are uncommon, data on T-cell exhaustion are still to be learned as well as effects on hypogammaglobulinemia, myelodysplasia and secondary malignancies mutagenesis risk, particularly for CRISPR- or iPSC-edited products. Published studies underreport access times, quality of lives and cost. Additional limitations include underrepresentation of the minority groups, despite higher incidence of MM in African American population, also lacking data on elderly patients and patients with renal failure.
Though most of the studies show positive results, those are mostly small sample phase-I/early phase-II studies that are still a subject for maturity, outside the scope of two FDA approved autologous BCMA products. Currently, there is insufficient evidence to replace autologous stem-cell transplants or FDA approved bispecific antibodies that carry no cellular vehicle and are fully manufactured off the shelf products.
Cellular therapy products certainly have great potential in addressing the unmet need in management of RRMM although further research is needed to provide evidence of efficacy and safety as well as good QoL and cost-effectiveness.
Next steps should include larger scale randomized clinical trials, comparison trials with the standard of care backbone treatments for RRMM, optimal sequencing of therapies, longer follow up periods for safety and efficacy as well as incorporation of the QoL questionnaires and geriatric fitness assessments. Additional preclinical studies on T cell exhaustion and persistence, determining mechanisms of antigen escape as well as optimization of the manufacturing and shortening the time from the bag to the vein [23]. Last but not least – creation of long- term registries to capture late toxicities and long-term survival outcomes.
The incurable nature of myeloma demands new treatment modalities and by proving that these cellular and gene constructs can deliver definitive, potentially curable and safe options would allow these treatments to be transitioned to frontline
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