Stem cell technologiesCurrent state

Future promiseWhere UCI fits in to the Post Prop.

71 world of biotechnology development in California

Proposition 71: Purpose and Intent

• Create California Institute for Regenerative Medicine.

Oversight by Independent Citizen’s Oversight Committee (ICOC).

• Support the development of technologies involving pluripotent stem cell and progenitor cell.

Prohibit human reproductive cloning

Proposition 71: Funds

• Funding details

Authorizes an average of $295 million per year in bonds over a 10-year period, $3 billion total

In any year, no more than $350 million in bonds to be issued If less than $350 million is issued in any year,

the remaining permitted amount may be carried over to one or more subsequent years

Proposition 71: Research facilities

Research facilities that can be used for research on “non-approved lines” are a priority.

• Accordingly, up to 10% of the funds will be used to build scientific and medical research facilities of nonprofit entities.

• These are to be constructed in the first five years, with priority given to facilities that can be brought on line within the first 2 years.

•Funding through Prop. 71 for development of facilities requires a 20% match.

Proposition 71: Oversight

The ICOC must meet in an open meeting format.Closed sessions may be conducted when discussing:

• Matters involving information relating to patients or medical subjects. Matters involving confidential intellectual property or work product

• Matters involving prepublication, confidential scientific research or data

• Matters concerning the appointment, employment, performance, compensation, or dismissal of institute officers and employees

Proposition 71: Scientific and Medical Working Groups

• Scientific and Medical Research Funding Working Group.

• Scientific and Medical Accountability Standards Working Group.

• Scientific and Medical Research Facilities Working Group.

Blastocyst -from In Vitro Fertilization Clinic

Inner Cell Mass(Stem Cells)

“Blueprint” cells

A primer on Human Embryonic Stem Cells

A Blastocyst is a hollow ball of cells with a small clump of stem cells inside

R RC

“Blueprint” cells

Human Embryonic Stem Cells

Pipette

Stem Cells

To remove the stem cells, the Blastocyst is opened and the stem cells removed with a pipette

Blastocyst -from In Vitro Fertilization Clinic

Stem Cells “Blueprint” cells

A Blastocyst is a hollow ball of cells with a

small clump of stem cells inside

Pipette

Pipette

Stem Cells

Petri Dish

Human Embryonic Stem Cells

To remove the stem cells, the Blastocyst is broken open and the stem cells removed with

a pipette(an ultra thin glass tube)

The stem cells are placed in a

dish and are fed and cared for

(each blastocyst = 1 stem cell line)

Blastocyst -from In Vitro Fertilization Clinic

Stem Cells “Blueprint” cells

A Blastocyst is a hollow ball of cells with a

small clump of stem cells inside

Stem Cells

“Blueprint” cells

NeuronMuscle

cell

Pancreatic Islet

Petri DishStem Cells

Different chemicals / molecules are added to the stem cells to make them become specific types of cells.

Growth factors Chemical cues

Donor Egg Skin Cell

Needle

Nucleus(DNA)

Nucleus(DNA)

Needle

Chemicals and Growth Factors

Dividing cellsNeuron

Muscle cell

Pancreatic Islet

Stem Cells

Somatic Cell Nuclear Transfer or Therapeutic Cloning

Blastocyst Stem Cells

The vision for UCI’s Stem Cell Center: Stem cell therapies for neurological disorders

• Brain and spinal cord injury.

• Stroke.

• Neurodegenerative diseases Parkinson’s Disease Huntington’s Disease Alzheimer’s Disease Multiple Sclerosis Lou Gerhig’s Disease (ALS)

Reeve-Irvine Research Center

Neurological disorders involve the loss of particular cell types in the nervous system

• Brain and spinal cord injury and stroke (loss of nerve cells and myelin-forming oligodendrocytes).

• Neurodegenerative diseases Parkinson’s Disease (loss of dopamine-containing nerve cells

in the brainstem). Huntington’s Disease (loss of nerve cells in the striatum). Alzheimer’s Disease (loss of nerve cells in the cerebral cortex). Multiple Sclerosis (loss of myelin-forming oligodendrocytes). Lou Gerhig’s Disease-ALS (loss of motor neurons from the

spinal cord).

• The vision: To use embryonic stem cells to restore the cells that are lost as a result of injury or neurodegenerative diseases.

Reeve-Irvine Research Center

Blastocyst -

Stem Cells

Pipette

Stem Cells“Blueprint” cells

Stem Cells

Petri Dish

“Blueprint” cells

Goal #1: to make stem cells into nerve cells

The stem cells are treated with factors to cause them to differentiate

into particular cell types Stem cells differentiated into neurons

Reeve-Irvine Research Center

Goal #2: To discover how to make stem cells integrate into neural circuits.

Reeve-Irvine Research Center

Nerve cell (neuron) Oligodendrocyte

More research is needed to find the ways to actually use stem cells for therapeutic applications.

At the RIRC, a therapy is being developed to use stem cells to replace myelin-forming cells

Myelin-forming cells (oligodendrocytes) die as a result of spinal cord injury, resulting in the loss of myelin (insulation) from nerve fibers.

An important potential therapeutic strategy: Replace myelin-forming cells using stem cells that differentiate into oligodendrocytes.

Reeve-Irvine Research Center

Then to Oligodendrocyte

Precursors

Stem cells are first differentiated to the

neural lineage.

RIRC scientists have succeeded in developing ways to produce oligodendrocytes from human ES cells and have shown that they can restore myelin after spinal cord injury in experimental animals.

Keirstead LabReeve-Irvine Research Center

But do these cells have the potential to form tumors over

longer periods of time?

1. They can generate large quantities of tissue rapidly

2. They can become any cell in the bodyEmbryonic stem

cells

Brain Heart

CartilageBone marrowFat

Embryonic Stem Cells

Human Embryonic Stem Cells

Developing facilities for stem cell research

It is likely that research involving non-approved stem cell lines will be prohibited in facilities that were built with NIH support, or that contain NIH-supported core equipment.

• In recognition of this, up to 10% of Prop. 71 funds will be used to build scientific and medical research facilities.

•Funding through Prop. 71 for development of facilities requires a 20% match.

Reeve-Irvine Research Center

Proposition 71: Funds

• Indirect costs limited to 25 % of a research award

Excluding amounts included in a facilities award

Indirect cost limitation may be increased by amount the grantee provides in matching funds

Must be in excess of 20% of the grant amount