Stem cell-based therapy is a rapidly advancing frontier in biotechnology and regenerative medicine, holding the promise to repair, replace, or restore damaged tissues and organs. For PharmD students, understanding the fundamentals of stem cell biology—from pluripotent embryonic cells to adult somatic cells and induced pluripotent stem cells (iPSCs)—is crucial. This knowledge provides the basis for comprehending novel treatments for diseases like cancer, diabetes, and neurodegenerative disorders.
1. Which two fundamental properties define a stem cell?
- Rapid division and apoptosis
- Self-renewal and differentiation
- Motility and phagocytosis
- Antigen presentation and cytokine release
Answer: Self-renewal and differentiation
2. Which term describes the ability of a stem cell to differentiate into all cell types of the three primary germ layers (ectoderm, endoderm, and mesoderm)?
- Totipotency
- Pluripotency
- Multipotency
- Unipotency
Answer: Pluripotency
3. Embryonic stem cells (ESCs) are typically derived from which part of the blastocyst?
- The trophoblast
- The zona pellucida
- The inner cell mass
- The placenta
Answer: The inner cell mass
4. Induced pluripotent stem cells (iPSCs) are created by:
- Isolating them directly from an embryo
- Fusing a somatic cell with an embryonic stem cell
- Reprogramming a differentiated adult cell back to an embryonic-like state
- Stimulating a multipotent stem cell with growth factors
Answer: Reprogramming a differentiated adult cell back to an embryonic-like state
5. Hematopoietic stem cell transplantation (HSCT) is a well-established therapy primarily used to treat:
- Type 1 diabetes
- Parkinson’s disease
- Cancers of the blood and bone marrow
- Macular degeneration
Answer: Cancers of the blood and bone marrow
6. A primary advantage of using a patient’s own iPSCs for therapy is that they:
- Are much easier to grow than ESCs
- Avoid the risk of immune rejection
- Do not have the ability to differentiate
- Are multipotent, not pluripotent
Answer: Avoid the risk of immune rejection
7. Adult stem cells, such as mesenchymal stem cells (MSCs), are typically described as:
- Totipotent
- Pluripotent
- Multipotent
- Unipotent
Answer: Multipotent
8. A major safety concern associated with the therapeutic use of pluripotent stem cells (ESCs and iPSCs) is the risk of:
- Teratoma formation
- Viral infection
- Immediate immune rejection
- Rapid degradation upon injection
Answer: Teratoma formation
9. In an “allogeneic” stem cell transplant, the donor cells come from:
- The patient themselves
- A genetically identical twin
- A different, immunologically-matched individual
- A different species
Answer: A different, immunologically-matched individual
10. What is the primary role of adult stem cells in the body?
- To form the embryo during development
- To provide tissue maintenance and repair
- To fight off infections
- To transport oxygen in the blood
Answer: To provide tissue maintenance and repair
11. The process of guiding a stem cell to become a specific cell type, like a neuron or a cardiomyocyte, is called:
- Self-renewal
- Apoptosis
- Directed differentiation
- Transformation
Answer: Directed differentiation
12. Mesenchymal stem cells (MSCs) are adult stem cells that have the ability to differentiate into:
- All cell types of the body
- Only other MSCs
- Bone, cartilage, muscle, and fat cells
- Neurons and glial cells only
Answer: Bone, cartilage, muscle, and fat cells
13. A key ethical concern historically associated with embryonic stem cell research is:
- The high cost of the research
- The destruction of a human embryo to derive the cells
- The risk of animal pathogen transmission
- The instability of the resulting cell lines
Answer: The destruction of a human embryo to derive the cells
14. The “Yamanaka factors” are a set of transcription factors used for what purpose?
- To induce differentiation in stem cells
- To create iPSCs by reprogramming somatic cells
- To purify stem cells from a mixed population
- To kill cancerous stem cells
Answer: To create iPSCs by reprogramming somatic cells
15. In the context of CAR-T cell therapy, T-cells are genetically modified ex vivo and then re-infused. This is a form of:
- Regenerative medicine
- Stem cell therapy
- Adoptive cell transfer therapy
- In vivo gene editing
Answer: Adoptive cell transfer therapy
16. A key challenge in using stem cells to treat neurodegenerative diseases like Parkinson’s is:
- Ensuring the cells differentiate into the correct type of neuron and integrate into the existing neural circuits
- Parkinson’s disease is not caused by cell death
- The brain is highly resistant to any type of transplant
- Neurons cannot be grown in a lab
Answer: Ensuring the cells differentiate into the correct type of neuron and integrate into the existing neural circuits
17. What does the term “autologous” mean in the context of cell therapy?
- The cells are derived from a different species
- The cells are derived from a matched donor
- The cells are derived from and then given back to the same patient
- The cells are genetically modified
Answer: The cells are derived from and then given back to the same patient
18. The use of iPSCs to create “disease-in-a-dish” models is a powerful tool for:
- Treating patients directly with the dish
- Drug discovery and studying disease pathophysiology
- Growing organs for transplantation
- Manufacturing vaccines
Answer: Drug discovery and studying disease pathophysiology
19. Hematopoietic stem cells are primarily found in what tissue in adults?
- The brain
- Adipose (fat) tissue
- The liver
- Bone marrow
Answer: Bone marrow
20. A “stem cell niche” refers to:
- A type of laboratory incubator
- The specific microenvironment in a tissue that supports and regulates stem cells
- A company that specializes in stem cell research
- The process of stem cell differentiation
Answer: The specific microenvironment in a tissue that supports and regulates stem cells
21. A totipotent stem cell is unique because it can give rise to:
- Only multiple cell types within one germ layer
- All cell types of the body, but not the placenta
- All cell types of the body, including extraembryonic tissues like the placenta
- Only a single, specific cell type
Answer: All cell types of the body, including extraembryonic tissues like the placenta
22. A major hurdle for the clinical application of stem cell therapies is:
- A complete lack of understanding of stem cell biology
- The challenges of manufacturing cells on a large scale under Good Manufacturing Practice (GMP) standards
- The refusal of all regulatory agencies to consider them
- The fact that stem cells cannot be frozen for storage
Answer: The challenges of manufacturing cells on a large scale under Good Manufacturing Practice (GMP) standards
23. The process of graft-versus-host disease (GVHD) is a major risk in which type of transplant?
- Autologous HSCT
- Allogeneic HSCT
- A blood transfusion
- An organ transplant from an identical twin
Answer: Allogeneic HSCT
24. How can gene editing technologies like CRISPR-Cas9 be combined with stem cell therapy?
- To correct a genetic defect in a patient’s stem cells ex vivo before transplantation
- To edit the DNA of the pharmacist administering the therapy
- To make the stem cells more likely to be rejected
- It is not possible to combine these two technologies
Answer: To correct a genetic defect in a patient’s stem cells ex vivo before transplantation
25. A primary application of stem cell therapy in cardiology is to:
- Replace the entire heart with a lab-grown organ
- Regenerate heart muscle (cardiomyocytes) after a myocardial infarction
- Lower cholesterol levels
- Dissolve atherosclerotic plaques
Answer: Regenerate heart muscle (cardiomyocytes) after a myocardial infarction
26. The main advantage of iPSC technology over ESC technology from a patient-specific perspective is that it avoids:
- The need for cell culture
- Ethical controversies and the risk of immune rejection
- The need for reprogramming factors
- The ability to create pluripotent cells
Answer: Ethical controversies and the risk of immune rejection
27. Before a patient receives an allogeneic hematopoietic stem cell transplant, they typically undergo myeloablative therapy (chemo/radiation) to:
- Strengthen their existing immune system
- Destroy the patient’s existing bone marrow and immune cells
- Stimulate their adult stem cells to differentiate
- Test their tolerance for the procedure
Answer: Destroy the patient’s existing bone marrow and immune cells
28. Unproven stem cell treatments offered by clinics, often called “stem cell tourism,” are a major concern because:
- They are always effective but too expensive
- They often lack scientific evidence of safety and efficacy
- They are only available in FDA-approved hospitals
- They are regulated with strict oversight
Answer: They often lack scientific evidence of safety and efficacy
29. A key step in ensuring the safety of a pluripotent stem cell therapy is to:
- Make sure the final cell product for transplantation contains undifferentiated cells
- Differentiate all pluripotent cells into the desired cell type and remove any remaining undifferentiated cells
- Inject the cells as quickly as possible without quality control
- Mix the pluripotent cells with adult stem cells
Answer: Differentiate all pluripotent cells into the desired cell type and remove any remaining undifferentiated cells
30. Which of the following is an example of a unipotent stem cell?
- An embryonic stem cell
- A hematopoietic stem cell
- A spermatogonial stem cell
- An induced pluripotent stem cell
Answer: A spermatogonial stem cell
31. The process of cells “homing” refers to their ability to:
- Remain at the site of injection
- Migrate to a specific tissue or site of injury
- Self-replicate indefinitely in a culture dish
- Differentiate into any cell type
Answer: Migrate to a specific tissue or site of injury
32. What is the main therapeutic goal of using stem cells to treat Type 1 Diabetes?
- To replace the patient’s immune cells
- To regenerate insulin-producing beta cells in the pancreas
- To provide a source of glucose for the patient
- To treat the common cardiovascular complications of diabetes
Answer: To regenerate insulin-producing beta cells in the pancreas
33. What is a key role for a pharmacist in the context of stem cell therapy?
- To perform the cell transplantation procedure
- To manage immunosuppressive drugs and other supportive care medications for transplant patients
- To derive new embryonic stem cell lines
- To develop new cell culture media
Answer: To manage immunosuppressive drugs and other supportive care medications for transplant patients
34. The “engraftment” phase after a hematopoietic stem cell transplant refers to:
- The initial infusion of the cells
- The transplanted stem cells starting to grow and make new blood cells in the bone marrow
- The patient being discharged from the hospital
- The removal of the patient’s original bone marrow
Answer: The transplanted stem cells starting to grow and make new blood cells in the bone marrow
35. A key difference between adult stem cells and embryonic stem cells is that adult stem cells are:
- Easier to isolate and grow in culture
- Naturally pluripotent
- Generally limited in their differentiation potential (multipotent)
- More likely to form teratomas
Answer: Generally limited in their differentiation potential (multipotent)
36. A potential strategy to overcome immune rejection in allogeneic cell therapy is:
- To use cells from a completely unmatched donor
- To create “universal” donor cells by genetically editing out immune markers like HLA proteins
- To avoid the use of any immunosuppressive drugs
- To transplant the cells into a different tissue than intended
Answer: To create “universal” donor cells by genetically editing out immune markers like HLA proteins
37. Cryopreservation is a critical technology for cell-based therapies that allows for:
- The long-term storage and banking of cells at very low temperatures
- The rapid differentiation of cells into any lineage
- The purification of cells from a mixed culture
- The real-time analysis of cell function
Answer: The long-term storage and banking of cells at very low temperatures
38. What is the source of stem cells in umbilical cord blood?
- They are maternal cells transferred to the infant
- They are primarily embryonic stem cells
- They are a rich source of hematopoietic and mesenchymal stem cells
- They are reprogrammed adult cells
Answer: They are a rich source of hematopoietic and mesenchymal stem cells
39. A “bioreactor” used for stem cell manufacturing is designed to:
- Provide a controlled environment for large-scale cell expansion
- Sort cells based on their surface markers
- Genetically edit the cells
- Store the cells at room temperature
Answer: Provide a controlled environment for large-scale cell expansion
40. A potential therapeutic use of mesenchymal stem cells (MSCs) is for their:
- Ability to differentiate into blood cells
- Immunomodulatory and anti-inflammatory effects
- Ability to form teratomas
- Direct antiviral properties
Answer: Immunomodulatory and anti-inflammatory effects
41. The first step in creating patient-specific iPSCs is to:
- Obtain a tissue sample, such as skin or blood cells, from the patient
- Isolate embryonic stem cells from a donor
- Perform a bone marrow transplant
- Design a gene editing vector
Answer: Obtain a tissue sample, such as skin or blood cells, from the patient
42. Which of the following is a limitation of using adult stem cells for therapy?
- They are difficult to obtain and may be present in very small quantities
- They are pluripotent
- They have no known function in the body
- They always cause immune rejection
Answer: They are difficult to obtain and may be present in very small quantities
43. The FDA regulates most human stem cell products as:
- Dietary supplements
- Medical devices
- Drugs, biologics, or medical devices, requiring clinical trials for approval
- Cosmetics
Answer: Drugs, biologics, or medical devices, requiring clinical trials for approval
44. Why is a patient’s age a consideration for autologous cell therapies?
- Younger patients do not have any stem cells
- A patient’s own adult stem cells may have accumulated mutations or lost regenerative capacity over time
- Only cells from elderly patients are effective
- Age has no impact on stem cell function
Answer: A patient’s own adult stem cells may have accumulated mutations or lost regenerative capacity over time
45. Somatic Cell Nuclear Transfer (SCNT) is a technique that was famously used to:
- Create the first monoclonal antibody
- Clone an animal, such as Dolly the sheep
- Sequence the human genome
- Discover the CRISPR-Cas9 system
Answer: Clone an animal, such as Dolly the sheep
46. A challenge in using iPSCs for disease modeling is that the reprogramming process might:
- Erase some of the epigenetic signatures relevant to the disease
- Make the cells resistant to all drugs
- Cause the cells to grow too slowly
- Prevent the cells from being studied in a petri dish
Answer: Erase some of the epigenetic signatures relevant to the disease
47. A pharmacist is asked by a patient about a local clinic offering a “stem cell cure” for arthritis. The most appropriate response is to:
- Immediately endorse the clinic and its treatment
- Advise the patient that most such treatments are unproven and to discuss it with their primary physician
- Tell the patient that all stem cell therapies are dangerous
- Offer to invest in the clinic with the patient
Answer: Advise the patient that most such treatments are unproven and to discuss it with their primary physician
48. The field of tissue engineering often combines stem cells with what other components to create functional tissues?
- Scaffolds and growth factors
- Antibiotics and antivirals
- Metals and plastics
- Vitamins and minerals
Answer: Scaffolds and growth factors
49. For a stem cell therapy to be successful, the transplanted cells must:
- Undergo immediate apoptosis
- Survive, engraft, and integrate functionally with the host tissue
- Transform into cancerous cells
- Trigger a massive immune response
Answer: Survive, engraft, and integrate functionally with the host tissue
50. The ultimate goal of regenerative medicine using stem cells is to:
- Provide only symptomatic relief for diseases
- Create a market for unproven therapies
- Restore normal function to diseased or injured tissues
- Replace the need for all small-molecule drugs
Answer: Restore normal function to diseased or injured tissues
I am a Registered Pharmacist under the Pharmacy Act, 1948, and the founder of PharmacyFreak.com. I hold a Bachelor of Pharmacy degree from Rungta College of Pharmaceutical Science and Research. With a strong academic foundation and practical knowledge, I am committed to providing accurate, easy-to-understand content to support pharmacy students and professionals. My aim is to make complex pharmaceutical concepts accessible and useful for real-world application.
Mail- Sachin@pharmacyfreak.com