This document discusses potential stem cell treatments for three diseases: multiple sclerosis, sickle cell disease, and Parkinson's disease. It provides background on the diseases and how stem cells may help treat them. For multiple sclerosis, stem cells from bone marrow or fat tissue could be harvested, activated with light, and reintroduced intravenously to help repair myelin sheaths. For sickle cell disease, hematopoietic stem cell transplantation or gene therapy using lentiviral vectors could help produce healthy red blood cells. For Parkinson's disease, embryonic or induced pluripotent stem cells could potentially be differentiated into dopamine neurons for transplantation to replace those lost in the disease.

1. Major diseases and stem cells
Presented to
Dr. SYED WAQAR SHAH
Presented by
Zeeshan khan
Hammad Ali
Muzaffar Latif

2. CONTENTS
• What are stem cells?
• Diseases can be treated with stem cell therapy
• Multiple Sclerosis
• Sickle cell disease
• Parkinson’s disease

3. What are stem cells?

4. Diseases treated with stem cells
There are about 65 diseases which can be cured by stem cells
Multiple sclerosis
Parkinson’s disease
Sickle cell disease
Leukemia
Alzheimer’s
Rheumatoid Arthritis
Diabetes Type 2

6. Multiple sclerosis
• In united states there are 400,000 living with
MS and 200 more are diagnosed every week.
• In Pakistan this disease affects about 400
people per year.
• World wide the disease effects 2.5 million
people.

7. Multiple Sclerosis
• The French neurologist Jean-Martin Charcot (1825–1893)
was the first person to recognize multiple
sclerosis as a distinct disease in 1868.
• Multiple sclerosis (MS) is a potentially
disabling disease of the brain and
spinal cord (central nervous system).
• In MS, the immune system attacks the protective sheath (myelin)
that covers nerve fibers and causes communication problems
between your brain and the rest of your body.

9. Neurons in the brain protected by blood brain barrier
Only certain molecules can pass through the cells
They are activated
by myelin
More receptors are
expressed
Myelin specific T-cells
releases
the cytokines which dilate the
blood vessels
Macrophages will engulf and destroy the oligodendrocyte

10. Causes
• Genetic factors :
Female
Gene encoding for HLA-DR2(used to identify and bind foreign)
• Environmental factors:
Infections
Vitamin D deficiency

11. Signs and symptoms
• Blurred vision
• Depression or unstable mood
• Muscle spasm
• Loss of sensitivity
• Problem of speech
• Problem in swallowing.

12. Stem cell treatment for Multiple
Sclerosis
Stem cell procedure includes four basic steps
1. Harvesting
2. Separation
3. Activation
4. Treatment

13. Procedure of treatment
1. Harvesting:
Stem cells are extracted from adipose tissue
or bone marrow.
2. Separation:
Stem cells are separated from other cells
3. Activation:
Stem cells are photo activated.
(red 650nm, yellow 600nm & green 550nm)
4. Treatment:
Activated stem cells are returned via
standard IV drip.

15. Sickle cell diseases: Anemia
• Group of inherited red blood cell disorders. People with SCD
have abnormal hemoglobin, called hemoglobin S or sickle
hemoglobin, in their red blood cells.
• Results from the substitution of glutamine to valine at the sixth
position in the β-globin chain and is a result of a single
nucleotide mutation.
• When a person has two hemoglobin S genes, Hemoglobin SS,
the disease is called sickle cell anemia .

16. Sickle Cell Anemia
• Anemia can cause shortness of breath, fatigue, and delayed
growth and development in children. The rapid breakdown of
red blood cells may also cause yellowing of the eyes and skin,
which are signs of jaundice.
• At the present time, hematopoietic stem cell transplantation is
the only cure for SCD

17. Hematopoietic stem cell transplant
(HSCT)
• To prepare for a hematopoietic stem cell transplant (HSCT),
strong medicines, called chemotherapy, are used to weaken or
destroy the patient’s own bone marrow, stem cells, and
infection fighting system.
• The patient’s bone marrow then is replaced with blood-
forming stem cells from a donor who does not have sickle cell
disease.
• The new bone marrow then produces red blood cells that are
healthy since they do not contain a lot of hemoglobin S.

19. What are the possible risks of
transplant?
• Infections – Chemotherapy lowers the white blood cells,
which normally fight and prevent infections
• Graft-versus-host disease (GVHD) – This reaction occurs
when the immune cells of the donor (graft cells) sense that
cells of the patient (host cells) are different and attack them.
• Veno-occlusive disease (VOD)- Blood vessels that lead into
and pass through the liver may suffer damage after a bone
marrow transplant. This may be caused by the chemotherapy
and might lead to swelling and severe liver damage.

20. What are the possible risks of
transplant?
• Nutrition problems – The stomach and intestines are
sensitive to chemotherapy. Nausea, vomiting, mouth sores,
diarrhea, and loss of appetite may occur.
• Low blood counts
• Social and emotional concerns
• Infertility-Most patients who receive a transplant will not be
able to have their own children in the future. This is one (1)
possible side effect of drugs used while preparing for the
transplant.

21. Gene therapy using HSC
• By correcting the gene in patient’s own HSC, no immune
reactions will occur after transplant and there will be better
outcomes with fewer complications.
• For gene therapy of sickle cell diseases, the goal is to add a
normal beta globin gene to the bone marrow HSC to allow
normal cells to be made.

23. Procedure
• Functioning copy of hemoglobin is packaged into Lentiviral
vector. The vector contains small number of parts from HIV
and is very effective at moving gene into cells.
• Patient’s blood stem cells are collected & using lentiviral
vector functioning hemoglobin gene is inserted into DNA of
collected blood cells.
• Inside body chemotherapy is done to remove existing stem
cells.
• Modified stem cells are returned to body and will produce
normal RBCs.

25. What is Parkinson’s disease
• Parkinson's disease is a progressive disorder of the
nervous system that affects movement
and cordination of the body.
• A disorder of the brain that leads to shaking (tremors) and
difficulty in walking movement, and body posture.

26. Discovery
• Parkinson's disease, which was first described in "An Essay on
the Shaking Palsy" in 1817 by a London physician James
Parkinson, has probably existed for thousands of years.

27. What happens in PD?
• Nerve cells use a brain chemical called DOPAMINE to help
control muscle movement.
• Parkinson’s disease occurs when the nerve cells in the brain
that make dopamine are slowly destroyed.
• Without dopamine, the nerve cells in that part of the brain
cannot properly send messages.
• This leads to the loss of muscle function.
• The damage gets worse with time.

29. Signs and symptoms
• Tremor
• Slowed movement
• Rigid muscles
• Impaired posture and balance
• Loss of automatic movements
• Speech changes
• Difficulty swallowing

30. Causes
The cause of Parkinson's disease is unknown, but several factors
appear to play a role
• Genes
genetic mutations
• Environmental triggers
Toxins or environmental factors
• The presence of Lewy bodies
Clumps of specific substances within brain cells are microscopic
markers of Parkinson's disease. these Lewy bodies hold an
important clue to the cause of Parkinson's disease.

31. Stem cell therapy

32. Embryonic Stem cells (ESC)
• Embryonic Stem cells (ESC) are derived from the inner cell
mass of pre-implantation embryos and are a source of
pluripotent cells.
• Once established, the pluripotent ES cells can be maintained
under defined culture conditions.
• ESCs differentiate into neurons more easily than other types of
cells.
• They potentially give rise to an infinite number of
dopaminergic neurons that may be subsequently transplanted
in PD patients.

33. Induced pluripotent stem cells
(iPSCs)
• Induced pluripotent stem cells, are adult cells ES cells
transformed from fibroblasts
• They are genetically reprogrammed to an embryonic stem
cell–like state by being forced to express genes important
for maintaining the properties of ESCs.
• These can be generated in large numbers and then
differentiated into dopamine neurons for transplantation.
• The main advantage of iPSCs over ESCs is that they are
less prone to rejection by the immune system of patient,
because the transplanted cells come from patients
themselves.