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alone5; however, clinical studies face challenges such
as regulatory hurdles, slow trial recruitment and lack
of validated biomarkers6. Indeed, the Food and Drug
Administration (FDA) has not approved a new drug for
the treatment of Alzheimer’s disease since 20035.
Regenerative properties of stem cells
Stem cells can be derived from multiple sources,
including embryonic cells, umbilical cord blood and
various adult tissues (adipose tissue, skin and bone
marrow). They have the potential to regenerate
damaged tissue, reduce inflammation and promote
healing. Stem cell therapies are prepared by
extracting stem cells from a donor or the patient’s
own tissue. Cells are sometimes genetically modified
and then incubated to increase their number before
administering them to the patient.
Researchers discovered the presence of endogenous
neural stem cells, consequently disproving the long-
held theory that the adult brain was not capable of
regenerating new neurons7
. These endogenous neural
stem cells are capable of migrating and integrating
into injured regions of the brain8. One theoretical
approach to treat neurodegeneration in dementia is
to augment the function of endogenous stem cells,
thereby promoting protection of remaining tissue and
generation of new neurons. Longstanding research
assessing the potential for newly generated neurons
to integrate efficiently into brain tissue and to effect
neurological improvements is ongoing2,8
There are multiple active clinical studies investigating
the potential to use stem cells to treat Alzheimer’s
disease, the majority of which use mesenchymal stem
cells (MSCs)9. MSCs can be obtained from various
sources, such as umbilical cord blood, adipose tissue
and bone marrow. There are multiple advantages to
using MSCs: they can be administered intravenously,
exhibit blood-brain barrier penetration, have low
tumorigenicity and do not elicit a strong immune
Transplantation of MSCs into rodents can reportedly
inhibit Aβ- and τ-related cell death, reduce Aβ plaque
formation, and stimulate neurogenesis, thereby
successfully treating Alzheimer’s disease in pre-
clinical studies2. While the neuroprotective effects and
reduced cognitive decline reported in animal studies
are yet to be demonstrated in humans, the successful
demonstration of safety and efficacy in animal models
has supported the approval of several human trials
using stem cells2.
Multiple Phase I and II clinical studies to investigate
the potential of MSCs from various sources are
underway in Korea, China and the United States2,11
open-label Phase I clinical trial has been completed
to evaluate the safety and tolerability of intracranially
injected MSCs derived from umbilical cord blood.
While no patients showed serious adverse effects,
the procedure did not slow cognitive decline over 24
months of follow up11,12
Future investigations may involve a multimodal
approach to treatment using a combination of
pharmacological therapies, augmentation of
endogenous stem cells and administration of
exogenous stem cells to achieve regeneration of
neurons, and reverse the symptoms of dementia. The
outlook is optimistic; stem cell therapies do have the
potential to be used in the treatment of Alzheimer’s
disease and other forms of dementia.
1. Huges J. ‘This is one of the biggest global health crises of the 21st Century’ [Internet].
World Economic Forum. 2017. Available from: https://www.weforum.org/agenda/2017/09/
2. Duncan T, Valenzuela M. ‘Alzheimer’s disease, dementia, and stem cell therapy’. Stem
Cell Res Ther. 2017;8(1):111.
3. Dementia Statistics [Internet]. Dementia Australia. 2018. Available from: https://www.
4. What is Dementia? [Internet]. Dementia Australia. 2018. Available from: https://www.
5. Cummings J, Lee G, Ritter A, Zhong K. ‘Alzheimer’s disease drug development pipeline:
2018’. Alzheimer’s Dement Transl Res Clin Interv. 201AD;4(May):1–18.
6. Alzheimer’s Drug Discovery Foundation. ‘Closing in on a cure: 2017 Alzheimer’s clinical
trials report’. Alzheimer’s Drug Discov Found. 2017.
7. TAKAGI Y. ‘History of Neural Stem Cell Research and Its Clinical Application’. Neurol Med
Chir (Tokyo). 2016;56(3):110–24.
8. Tang J. ‘How close is the stem cell cure to the Alzheimer’s disease future and beyond?’
Neural Regen Res. 2012.
9. Clinical trials for Alzheimer’s disease using stem cells [Internet]. 2018.
Available from: https://www.clinicaltrials.gov/ct2/results?cond=Alzheimer%27
10. Kwak K, Lee S, Yang J, Park Y. Review Article ‘Current Perspectives regarding Stem Cell-
Based Therapy for Alzheimer’s Disease’. 2018.
11. Kang JM, Yeon BK, Cho S-J, Suh Y-H. ‘Stem Cell Therapy for Alzheimer’s Disease: A
Review of Recent Clinical Trials’. J Alzheimer’s Dis. 2016;54(3):879–89.
12. Shukla AA, Hubbard B, Tressel T, Guhan S, Low D.’ Downstream processing of
monoclonal antibodies - application of platform approaches’. J Chromatogr B Analyt
Technol Biomed Life Sci. 2007 Mar 15;848(1):28–39.
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