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Novel Cell Therapy Overview

Novel cell therapy is one of the fastest-growing areas in biomedical research. The goal is to treat patients with blood diseases using cells that have been altered genetically. This technology uses a gene editing tool known as CRISPR to replace missing or mutated genes. The FDA has approved two different platforms based on CRISPR technology.

AMDCs are Used in a Novel Cell Therapy

A new cell therapy study at UC Davis Health is aiming to reverse dysphagia by injecting autologous muscle-derived progenitor cells (AMDCs) into the tongue. These cells can fuse with existing muscle fibers and increase tongue strength. AMDCs are a hybrid type of cells that are derived from patient muscle biopsies.

Several cell-based therapies are currently being developed. The development and regulatory pathways for these therapies differ from those for traditional medicines. However, many of the principles of good clinical development apply. To get approval, a new cell therapy must demonstrate an attractive safety and efficacy profile. The clinical trials also have to be well designed. In the case of MSCs, large pivotal trials are required to demonstrate safety and efficacy. This is the case for the Teva Phase 3 study of mesenchymal precursor cells.

Regulatory requirements for cell-based therapies are evolving to meet the specific characteristics of these therapies. The EU has issued a specialized committee, the Committee for Advanced Therapies (CAMTC), to help ensure that these treatments meet the necessary requirements. The committee has also issued a number of guidelines and reflection papers.

Genetically Modified T Cells

Researchers in the field of immunotherapy are developing genetically modified T cells, or CAR T cells, that are capable of targeting specific antigens in cancer cells. These engineered cells have been shown to induce a powerful anti-tumor attack. These engineered cells carry the TCR-T receptor, which enables them to target antigens on cancer cells and produce cytotoxic granules. This process may lead to the death of cancer cells.

CAR T cells are made of a hybrid protein that contains an antigen-binding domain from an antibody and a T-cell activation domain from a T-cell receptor (TCR). These cells can recognize and kill a specific antigen on cancer cells. These cells are still in their early stages of development, and clinical trials are needed to see if they can be effective.

Adoptive Cell Therapy

Adoptive cell therapy involves isolating the patient’s own immune cells and reinfusing them into the body. Most strategies use T cells from peripheral blood while others use natural killer cells. Cell therapies using T cells include tumor-infiltrating lymphocytes and T cell receptor T cells (CAR T). These cells may work as living drugs by targeting tumor-associated antigens.

Adoptive cell therapy has demonstrated transformative potential for hematologic malignancies. However, its effectiveness is limited in the majority of solid tumor indications. This is due to poor antigenic quantity, T-cell dysfunction, and tumor immune-evasion mechanisms. However, adoptive cellular therapy has the potential to overcome these hurdles by overcoming the immunosuppressive tumor microenvironment. Moreover, recent breakthroughs in gene editing technologies have opened a new frontier in the field of adoptive cell therapy.

CD19-CAR-T Cell Platforms have received FDA Approval

CAR-T cell platforms targeting CD19 have recently received FDA approval for use in the treatment of multiple solid tumours. However, while these therapies can successfully target B-cell malignancies, they may also have several side effects, ranging from on-target to off-target toxicities. Furthermore, a few lingering questions about the efficacy of CD19-CAR-T cell therapy remain unanswered.

One CD19-CAR-T cell product approved by the FDA is liso-cel. This drug incorporates the 4-1BB costimulatory domain into its formulation, which can be delivered into defined CD4-CD8 T cells. This product has been used in the treatment of patients with R/R LBCL, DLBCL, and DLBCL NOS. It has been administered to a total of 344 patients.

Roth’s Targeted Pooled Knock-in Screens

Targeted pooled knock-in screens for novel cancer cell therapies are now available to researchers. These approaches combine single-cell transcriptome analysis with knockin-screening to identify cells’ abundance and states ex vivo. These approaches accelerate the discovery of knockin programs and enable the parallel re-writing of endogenous genetic sequences.

Roth’s team used this strategy to expand their knockin library. They identified novel therapeutic knockin constructs that altered T cell fitness, immunosuppressive cytokines, and apoptotic receptors. These new constructs showed strong tumor killing capacities in vitro.

Targeted pooled knock-in screens are becoming a powerful functional genomics tool to discover novel drug targets. To perform a pooled knock-in screen, a cell population containing diverse gene knockouts is generated by infecting Cas9-expressing cells with lentiviral particles. The pool of knockout cells is then exposed to selected perturbations. The phenotypic effects of these gene constructs are monitored by comparing the mutant cells to a reference control cell population.

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