Neurological Disorders

Coding Dimension ID: 
303
Coding Dimension path name: 
Neurological Disorders
Grant Type: 
Progression Award - Discovery Stage Research Projects
Grant Number: 
DISC2P-12212
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$180 000
Disease Focus: 
Neurological Disorders
Spinal Cord Injury
Human Stem Cell Use: 
Embryonic Stem Cell
Public Abstract: 
Statement of Benefit to California: 
Grant Type: 
Progression Award - Discovery Stage Research Projects
Grant Number: 
DISC2P-12150
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$202 667
Disease Focus: 
Neurological Disorders
Traumatic Brain Injury
Human Stem Cell Use: 
Embryonic Stem Cell
Public Abstract: 
Statement of Benefit to California: 
Grant Type: 
Progression Award - Discovery Stage Research Projects
Grant Number: 
DISC2P-11700
Investigator: 
Institution: 
Type: 
PI
ICOC Funds Committed: 
$202 500
Disease Focus: 
Epilepsy
Neurological Disorders
Human Stem Cell Use: 
Embryonic Stem Cell
Public Abstract: 
Statement of Benefit to California: 
Grant Type: 
Progression Award - Discovery Stage Research Projects
Grant Number: 
DISC2P-11595
Investigator: 
Institution: 
Type: 
PI
ICOC Funds Committed: 
$201 150
Disease Focus: 
Neurological Disorders
Parkinson's Disease
Human Stem Cell Use: 
iPS Cell
Public Abstract: 
Statement of Benefit to California: 
Grant Type: 
Quest - Discovery Stage Research Projects
Grant Number: 
DISC2-12666
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$1 129 512
Disease Focus: 
Amyotrophic Lateral Sclerosis
Neurological Disorders
Human Stem Cell Use: 
Adult Stem Cell
Public Abstract: 

Research Objective

A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect of the stem cells

Impact

Amyotrophic lateral sclerosis (ALS) and the way to deliver and enhance stem cell-based treatment of ALS

Major Proposed Activities

  • Complete the additional in vitro studies and initiate the in vivo studies in SOD1 mouse model
  • Determine whether the combined effect of hNSCs intraparenchymally augmented/guided by SDV1a has a synergistic effect on improving disease onset/progression & symptom-free survival in the SOD1 mouse
  • Establish the preliminary toxicity and pharmacokinetics profiles of SDV1a in mouse model
  • Elucidation of structure and other characteristics; development and validation of analytical procedures
  • Process development and characterization in lab scale, stability study
Statement of Benefit to California: 

This new therapeutic will address a significant unmet medical need in the treatment of amyotrophic lateral sclerosis (ALS) and have important benefits to the patients with ALS and impact on the healthcare and bio industry in California.

Grant Type: 
Quest - Discovery Stage Research Projects
Grant Number: 
DISC2-12577
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$1 364 903
Disease Focus: 
Angelman Syndrome
Autism
Neurological Disorders
Human Stem Cell Use: 
iPS Cell
Public Abstract: 

Research Objective

AAV9-Cas13 gene therapy for Angelman syndrome using a first-in-kind mechanism of action that will safely and permanently restore expression of endogenous UBE3A that is deficient in CNS neurons.

Impact

Angelman syndrome is a rare (1 in 15,000 births) neurogenetic disorder caused by loss of UBE3A in the brain, causing severe developmental delay, ataxia and epilepsy. There are no treatments or cures.

Major Proposed Activities

  • Determine the optimal Cas13 guide-RNA for a rodent model.
  • Determine the optimal Cas13 guide-RNA for a humans.
  • Show that the gene therapy improves gene expression in rodent models.
  • Show that the gene therapy improves gene expression in human cells.
  • Show that the gene therapy improves symptoms in rodent models.
  • Show that the gene therapy can be safe and permanent.
Statement of Benefit to California: 

In addition to directly benefiting the ~2,500 children and families living with Angelman syndrome in California, this gene therapy with a first-in-kind mechanism of action could bring new treatments and new opportunities to our state. California has long been a hub of innovation, and creates an environment in which new technologies can be born, fostered, and attract others who want to create a better future for our residents. This activity will help train some of them, and inspire many others.

Grant Type: 
Quest - Discovery Stage Research Projects
Grant Number: 
DISC2-12400
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$1 064 724
Disease Focus: 
Brain Cancer
Cancer
Neurological Disorders
Solid Tumors
Human Stem Cell Use: 
Embryonic Stem Cell
Public Abstract: 

Research Objective

These preclinical studies will discover the efficacy of stem cell-derived, nanoscale, extracellular vesicles (candidate) to treat adverse effects of cancer therapy on brain function and cognition.

Impact

Stem cell-derived extracellular vesicles will address the confounders of stem cells (tumors, immunorejection, immunosuppression) & mitigate debilitating side-effects of cancer therapy on the brain.

Major Proposed Activities

  • Demonstrate the effectiveness of IV injections of stem cell-derived, nanoscale, extracellular-vesicles (EVs) to improve cognition in the mouse model of radiation- and chemo-therapy for brain cancers.
  • Determine the ability of EV treatment to protect against adverse effects of cancer therapy including neuro-inflammation, synaptic and micro-vascular damage in the brain.
  • Establish the neurocognitive benefits of injecting stem cell-derived-EV in brain cancer-bearing mice receiving combined radiation- and chemo-therapy (temozolomide, TMZ).
  • Elucidate the impact of stem cell-derived-EV injections on neuropathological hallmarks of radiation- and chemo-therapy (TMZ) in the cancer-bearing mice brains.
  • Determine the safety and rule out the toxicity of stem cell-derived EV treatment in brain and peripheral organs in the mice receiving radiation- and chemo-therapy (TMZ) for brain cancer.
  • Confirm miRNA-124-based mechanism (commonly found within the EV cargo) of stem cell-derived EV-mediated neuroprotection in the mice undergoing radiation- and chemo-therapy for brain cancers.
Statement of Benefit to California: 

In California, nearly 187,000 patients diagnosed with cancer will be alive in 5 years & more than 1.88 million have a history of cancer. Importantly, adult & childhood cancer survivors suffer from severe & persistent cognitive deficits that adversely affect their quality of life (QOL). A stem cell-based therapeutic could reduce inflammation & restore the cognitive function that may significantly improve patient’s QOL, reduce financial hardship on patients, caregivers & the state of California.

Grant Type: 
Quest - Discovery Stage Research Projects
Grant Number: 
DISC2-12169
Investigator: 
ICOC Funds Committed: 
$250 000
Disease Focus: 
Neurological Disorders
Stroke
Human Stem Cell Use: 
iPS Cell
Public Abstract: 

Research Objective

This grant proposes development of a stem cell based therapy that is derived from human induced pluripotent stem cells. These cells are in the form of a brain support cell, an astrocyte.

Impact

The cell candidate will treat vascular dementia, the second leading cause of dementia, and stroke by overcoming a bottleneck in the ability to make large quantities of the cells for clinical use.

Major Proposed Activities

  • In vivo tumorigenic studies.
  • Development and optimization of potency assays
  • Qualification and stability of cell delivery system.
Statement of Benefit to California: 

This research will develop a therapy for a disease with no treatment, vascular dementia, that is common and devastating in its consequences. The intellectual property for this therapy is held by a State of California public university (UCLA) and commercialization will directly benefit the State of California.

Grant Type: 
Quest - Discovery Stage Research Projects
Grant Number: 
DISC2-12164
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$250 000
Disease Focus: 
Neurological Disorders
Traumatic Brain Injury
Human Stem Cell Use: 
iPS Cell
Public Abstract: 

Research Objective

A cell therapy product comprised of inhibitory neurons that can migrate, integrate and restore neurologic function after traumatic brain injury.

Impact

Traumatic brain injury

Major Proposed Activities

  • Examine the most effective dose and safety profile of human iPSC-derived MGE cells grafted into rodent hippocampus.
  • Determine whether human iPSC-derived MGE cells mature into appropriate cortical interneurons in the traumatically injured brain
  • Evaluate the effect of human GABA neurons on synaptic activity in the injured brain
  • Evaluate the therapeutic potential of human-derived interneurons
Statement of Benefit to California: 

Nearly 6 million Americans - including 700,000 Californians - live with permanent physical or mental health problems resulting from a traumatic brain injury, but there are no treatments. We propose studies to create a cell therapy product that is capable of restoring neurologic function to these patients.

Grant Type: 
Quest - Discovery Stage Research Projects
Grant Number: 
DISC2-12158
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$249 997
Disease Focus: 
Amyotrophic Lateral Sclerosis
Neurological Disorders
Human Stem Cell Use: 
iPS Cell
Public Abstract: 

Research Objective

We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS).

Impact

ALS is fatal and incurable, and if successful, we will develop a treatment that slows or stops ALS progression across a broad range of patients.

Major Proposed Activities

  • Selection of the lead drug by testing several candidates for efficacy and safety on ALS patient-derived nerve cells.
  • Confirmation that the lead drug is effective and stable in mice.
  • Confirmation that the lead drug is safe in mice.
Statement of Benefit to California: 

ALS is a fatal, incurable disease and California has one of the highest number of ALS patients of any state. By testing our drug on stem cell-derived nerve cells from Californian ALS patients, we will increase the chances that it will be effective on the types of ALS patients found in California. If successful, our drug will substantially slow or stop ALS disease progression.

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