Stem Cell Therapy Overview
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Questions and Answers

What impact did the addition of a costimulatory domain to CARs have on T cells?

  • It made T cells less targeted towards cancer cells
  • It decreased the effectiveness of CAR-T therapy
  • It limited T cell survival
  • It improved T cell activation and cytotoxicity (correct)

How are CAR-T cells prepared for infusion into a patient?

  • They are genetically engineered and multiplied in a lab (correct)
  • They are extracted from the patient’s tumors
  • They are derived from blood donations
  • They are created from stem cells

What type of cancers has CAR-T cell therapy shown significant potential in treating?

  • Hematological malignancies (correct)
  • Solid tumors
  • Neurodegenerative diseases
  • Benign tumors

What is the main function of CARs on the surface of T cells?

<p>To recognize and bind to cancer cell antigens (A)</p> Signup and view all the answers

What monitoring methods are used to evaluate the effectiveness of CAR-T cell therapy?

<p>CT scans, blood tests, and bone marrow biopsies (C)</p> Signup and view all the answers

What concerns remain regarding the long-term performance of CAR-T cells?

<p>Behavior of CAR-T cells over extended periods (B)</p> Signup and view all the answers

As of now, how many commercial CAR-T products are approved?

<p>6 (B)</p> Signup and view all the answers

In which types of patients was a landmark observation made regarding CD19-redirected CAR-T cells?

<p>Patients with chronic lymphocytic leukemia (D)</p> Signup and view all the answers

What are the two defining properties of stem cells?

<p>Self-renewal and differentiation (A)</p> Signup and view all the answers

What role do telomeres play in stem cells?

<p>They maintain the self-renewal ability. (C)</p> Signup and view all the answers

Which cell types contribute to muscle growth and regeneration besides satellite cells?

<p>Pericytes and immune cells (A)</p> Signup and view all the answers

What happens to adult stem cells in response to physiological turnover or tissue damage?

<p>They become activated and begin to divide. (A)</p> Signup and view all the answers

How do progenitor cells differ from stem cells?

<p>Progenitor cells are committed to differentiate into specific target cells. (B)</p> Signup and view all the answers

What is the primary function of telomerase in stem cells?

<p>To prolong the lifespan of cell divisions. (A)</p> Signup and view all the answers

Where have tissue-specific stem cells been identified?

<p>In various organs including the brain and heart (D)</p> Signup and view all the answers

What mechanism do stem cells use to maintain a pool of themselves after activation?

<p>They utilize symmetric and asymmetric cell division. (C)</p> Signup and view all the answers

What defines totipotent stem cells?

<p>They can differentiate into all cell types, including extraembryonic tissues. (B)</p> Signup and view all the answers

Which type of stem cells are primarily focused on for therapeutic applications?

<p>Pluripotent stem cells (D)</p> Signup and view all the answers

What happens to pluripotent stem cells after a certain period of time?

<p>They become multipotent. (C)</p> Signup and view all the answers

What is a characteristic of multipotent stem cells?

<p>They give rise to a limited range of cells within a specific tissue type. (D)</p> Signup and view all the answers

What is a significant advantage of engineering CAR-T cells with a control mechanism?

<p>It provides a method to deactivate T cells when necessary. (D)</p> Signup and view all the answers

Which tactic is used by some clinics to falsely convey innovation in stem cell treatments?

<p>Filing patent applications. (B)</p> Signup and view all the answers

How can stem cells be reverted to a pluripotent state?

<p>Using chemical compounds or transcription factors. (C)</p> Signup and view all the answers

What role do multipotent stem cells play in the body?

<p>They reside in tissues for repair and replacement. (D)</p> Signup and view all the answers

How do clinics use listings on government websites like clinicaltrials.gov advantageously?

<p>To suggest their treatment has completed extensive clinical testing. (B)</p> Signup and view all the answers

What ethical concern arises from companies publishing in predatory journals?

<p>It can create a false sense of scientific credibility. (B)</p> Signup and view all the answers

Which statement accurately describes differentiated cells?

<p>They perform specific functions in the body. (B)</p> Signup and view all the answers

Why are multipotent stem cells considered difficult to isolate?

<p>They are rare and usually grown in specific environments. (C)</p> Signup and view all the answers

What is a potential risk as described in a patient example related to stem cell treatments?

<p>Spending large sums on procedures with serious risks. (B)</p> Signup and view all the answers

What issue is raised by the tactics used for promoting unproven stem cell treatments?

<p>They may mislead consumers into trusting unproven therapies. (D)</p> Signup and view all the answers

Why might consumers be misled by superficial markers of scientific legitimacy?

<p>They often lack the expertise to evaluate scientific claims. (C)</p> Signup and view all the answers

What underlying need drives the market for scientific legitimacy in medical treatments?

<p>The need for clinical and scientific validation. (D)</p> Signup and view all the answers

What is primarily stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF)?

<p>Production of white blood cells (A)</p> Signup and view all the answers

Which condition is characterized by hyperinflammation and an overwhelming immune response after CAR-T cell therapy?

<p>Secondary hemophagocytic lymphohistiocytosis (D)</p> Signup and view all the answers

Which of the following symptoms might indicate neurotoxicity as a side effect of CAR-T cell therapy?

<p>Headache (C)</p> Signup and view all the answers

What key role does interleukin 6 play in the context of CAR-T cell therapy?

<p>Regulates immune responses and drives inflammation (D)</p> Signup and view all the answers

What is typically observed in cerebrospinal fluid (CSF) during acute neurotoxicity after CAR-T cell therapy?

<p>Elevated protein levels (C)</p> Signup and view all the answers

Which symptom is NOT typically associated with CAR-T cell-related neurotoxicity?

<p>Coughing (D)</p> Signup and view all the answers

What is the role of perforin and granzymes in cancer treatment?

<p>Directly kill cancer cells (A)</p> Signup and view all the answers

Management of secondary hemophagocytic lymphohistiocytosis often involves which type of treatment?

<p>Corticosteroids (C)</p> Signup and view all the answers

What is the primary therapeutic purpose of tocilizumab in the context of CAR-T cell therapy?

<p>To block IL-6 signaling (C)</p> Signup and view all the answers

What types of malignancies can develop as long-term adverse effects of CAR-T cell therapy?

<p>T-cell lymphoma and peripheral T-cell lymphoma (C)</p> Signup and view all the answers

Which generation of CAR T cells includes a cytokine receptor to boost T cell survival?

<p>5th generation (C)</p> Signup and view all the answers

What is a key feature of 2nd generation CAR T cells compared to the 1st generation?

<p>They include an additional costimulatory domain such as CD28 (D)</p> Signup and view all the answers

What is the significance of the incidence of second malignancies after CAR-T cell therapy?

<p>It highlights the risk of T-cell cancers post-therapy (C)</p> Signup and view all the answers

Which component is found in the 3rd generation CAR T cells to enhance T cell activation?

<p>Two costimulatory domains like CD28 and 4-1BB (B)</p> Signup and view all the answers

What is one engineering strategy to improve CAR-T cell therapies mentioned?

<p>Enhancing their ability to differentiate between cancerous and healthy cells (D)</p> Signup and view all the answers

What is a potential adverse effect mentioned after the administration of CAR-T cell therapy?

<p>Development of certain types of T-cell cancers (B)</p> Signup and view all the answers

Flashcards

Tissue-specific stem cells

Stem cells found in various tissues, crucial for tissue regeneration and repair.

Self-renewal

The ability of stem cells to divide and create more stem cells.

Differentiation

The process where stem cells generate specialized cells (e.g., skin, muscle).

Quiescent stem cells

Inactive stem cells, waiting for activation by signals.

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Telomerase

Enzyme that maintains telomeres, enabling continued cell division in stem cells.

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Telomeres

Protective caps at the ends of chromosomes.

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Progenitor cells

Cells more specific than stem cells; committed to becoming specific cell types.

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Symmetric cell division

Cell division that results in two identical stem cells.

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Stem Cells

Undifferentiated cells with extensive proliferation and high differentiation potential, capable of becoming various specialized cells in the body.

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Totipotent Stem Cells

Stem cells capable of developing into every cell type in the body, plus extraembryonic tissue (like the placenta).

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Pluripotent Stem Cells

Stem cells capable of generating all cell types in the body but not extraembryonic tissue.

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Multipotent Stem Cells

Stem cells that can differentiate into a limited number of cell types within a specific tissue.

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Induced Pluripotent Stem Cells (iPSCs)

Adult cells which have been reprogrammed to have a pluripotent state.

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Embryonic Stem Cells

Cells derived from the early embryo and with significant potential for differentiation.

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Extraembryonic tissue

Tissue outside the embryo. Important parts of the pregnancy.

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CAR-T cell therapy

A cancer treatment where a patient's T cells are modified to target and destroy cancer cells.

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CARs

Chimeric Antigen Receptors; genetically engineered proteins on modified T cells.

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Second-generation CARs

CARs with added costimulatory domains, like CD28 or 4-1BB, improving T cell activity.

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T cell activation

The process where T cells become active and ready to fight.

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Cancer cell destruction

The process where cancer cells are killed by T cells.

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Commercial CAR-T products

Approved treatments using CAR-T cell therapy for specific cancers

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Hematological malignancies

Cancers of the blood or blood-forming tissues.

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CD19 redirected CAR-T cells

CAR-T cells modified to target CD19, specifically found on some cancer cells.

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Tocilizumab

An IL-6 receptor antagonist used to treat CRS by blocking IL-6 signaling.

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Second malignancies (CAR-T)

Possible cancers that arise after CAR-T cell therapy.

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Long-term CAR-T side effects

Certain T-cell cancers (like lymphomas) can develop after CAR-T therapy.

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CAR T-cell generations

Different versions of CAR T cells with varying signaling domains.

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1st generation CAR T cells

Simplest CAR T cells, using only the CD3ζ signaling domain.

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3rd generation CAR T cells

CAR T cells with two costimulatory domains like CD28 and 4-1BB for enhanced activation.

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Emerging CAR-T strategies

New approaches to improve safety, specificity, and efficacy of CAR-T cell therapies.

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GM-CSF's Role

Granulocyte-macrophage colony-stimulating factor (GM-CSF) stimulates white blood cell production.

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IL-6's Dual Role

Interleukin 6 (IL-6) plays a part in immune regulation but can also lead to inflammation.

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CAR-T Neurotoxicity (ICANS)

CAR-T cells can damage the blood-brain barrier, leading to brain inflammation (ICANS).

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sHLH/MAS Symptoms & Diagnosis

sHLH/MAS is a severe immune response, often after treatments. Diagnosis needs signs like fever and low blood cell counts (cytopenia), or multi-organ dysfunction.

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sHLH/MAS Management

sHLH/MAS management involves immunosuppressive treatments like corticosteroids and others to control the overactive immune response.

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IL-6 in CRS

IL-6 is a key inflammatory cytokine causing the systemic inflammation seen with cytokine release syndrome (CRS).

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CAR-T Neurotoxicity Signs (Mild)

Mild symptoms can include dysgraphia, impaired attention, headaches, sleep disorders, anxiety, myoclonus, and motor dysfunction.

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CAR-T Neurotoxicity Signs (Severe)

Severe symptoms may include encephalopathy, aphasia, delirium, tremors, seizures, and cerebral edema.

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CAR-T cell control mechanism

An engineered system in CAR-T cells that allows for activation or deactivation of the cells through drugs or small molecules, preventing excessive activation and reducing toxicity.

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Stem cell treatment promotion

Marketing unproven stem cell treatments, often without scientific evidence of safety and effectiveness, using misleading tactics.

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Patent application (in medical context)

Filing a patent for a medical treatment gives an impression of innovation and ownership, but does not guarantee effectiveness.

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Clinical trial listing

Listing clinical trials on platforms like clinicaltrials.gov to make treatments appear part of legitimate clinical research, sometimes misleadingly.

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Predatory journals

Low-quality journals that charge authors to publish, often without proper peer review.

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Scientific legitimacy in medical treatments

The need for clinical and scientific validation of treatments, especially for emerging fields.

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Misleading market tactics

Strategies like patent applications, clinical trial listings, and publication in predatory journals used to make unproven treatments appear legitimate.

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Financial exploitation example

A patient was misled into paying for a procedure, listed as a clinical trial, without proper validation, with unknown risks, and the procedure costs considerable money.

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Study Notes

Stem Cell Therapy

  • Stem cells are undifferentiated cells with the ability to proliferate and differentiate into various specialized cells.
  • There are about 200 different types of specialized cells in the body, all originating from stem cells.
  • Once a stem cell commits to a specific differentiation pathway, it can no longer become another cell type.
  • Totipotent stem cells arise from a zygote and can differentiate into all tissue types and extraembryonic structures.
  • Pluripotent stem cells can differentiate into all three germ layers (ectoderm, endoderm, and mesoderm) but not extraembryonic tissues.
  • Multipotent stem cells differentiate into a limited range of cell types within a specific tissue.
  • Adult stem cells reside in various tissues, responsible for repair and replacement.
  • Stem cell niches are microenvironments that regulate stem cell maintenance and repair within tissues.
  • Pluripotent stem cells (PSCs) include embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs).
  • iPSCs are derived from adult cells and can be used to create a range of specialized cells.
  • SCNT (somatic cell nuclear transfer) is a method for creating cloned animals and creating stem cells.
  • Stem cell therapies are used to treat various diseases, including diabetes, blood disorders, and certain cancers.
  • Stem cells are used for drug testing and safety evaluations before clinical trials.

Pluripotent Stem Cells

  • Pluripotent stem cells (PSCs) are cells that can differentiate into all three germ layers, which give rise to all cells in the body.
  • They are distinguished by the expression of particular marker genes such as: Oct4, Sox2, Klf4, cMyc, Nanog.
  • Embryonic stem cells (ESCs) come from the inner cell mass of embryos and are self-renewing, pluripotent cells.
  • iPSCs have been generated from the conversion of adult/somatic cells into pluripotent stem cells using transcription factors.
  • PSCs are used to generate various cell types, including tissues needed for cell therapies and for various research purposes.

Stem Cell Therapy Applications

  • Hematopoietic stem cells are used to treat blood disorders and cancers.
  • Stem cells are used for diabetes treatment through beta-cell transplantation.
  • Stem cells are used for age-related macular degeneration treatment.
  • Stem cells are potentially used for other conditions, such as heart failure, Parkinson's disease, and spinal cord injury.
  • Stem cells are used in drug testing to examine drug efficacy and safety before use in patients.

Cell Therapy Side Effects

  • Autologous transplantation involves using the patient's own cells.
  • Allogeneic transplantation uses cells from another donor.
  • GVHD (graft-versus-host disease) can occur in allogeneic transplants and is the rejection of the recipient's tissues by the donor's immune system.
  • Acute GVHD occurs within the first three months post-transplant.
  • GVHD can involve several organs, and cause various effects including diarrhea, skin rashes, and liver problems.
  • Chronic GVHD can develop after acute GVHD and is a more severe form.

CAR-T Cell Therapy

  • CAR-T cell therapy involves extracting and modifying the patient's T cells and using them to fight cancer cells in a laboratory setting.
  • The modifications aim to enable the T cells to identify and bind to cancer cells for destroying them.
  • Serious side effects such as neurotoxicity, and cytokine release syndrome happen due to on-target, off-tumor effects.
  • CAR-T cell use has yielded promising results in cancer therapy but there can be serious side effects that need cautious management.

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Stem Cell Therapy Lecture 9 PDF

Description

Explore the fascinating world of stem cells and their therapeutic potential. This quiz covers various types of stem cells, their differentiation capabilities, and their roles in tissue repair and regeneration. Test your understanding of totipotent, pluripotent, and multipotent stem cells.

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