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Three Ideas Presentation
Emily Kazarinoffon 17 May 2013
Transcript of Three Ideas Presentation
Team SuperFly Emily, Christina, Linda, Mounica,
Annie, Kerry, & Michael Brain Tissue Targeting Diabeat-it Importance! Several diseases deal with demyelination: Multiple Sclerosis, Devic's Disease, Guillain-Barré Syndrome, Krabbe's Disease, etc.
No cure- treatment only slows progression
Symptoms are severe: Impaired muscle coordination, loss of sensation, speech, sight, & hearing issues, memory loss, other neurological issues, tremors, fatigue, paralysis 2.1 million people worldwide with Multiple Sclerosis (200 new cases every week in US alone)
Method of treatment applicable to many diseases
Greatly improve quality of life; works to reverse demyelination instead of slowing disease progression Impact There is no reliable remyelination process
Researchers have had varying levels of success:
-using chemokines to stimulate differentiation of endogenous oligodendrocyte progenitor cells
-stimulating neural stem cells to produce oligodendrocytes
-targeting hyaluronidase, an enzyme whose byproduct could prevent natural remyelination processes
-implanting myelin producing cells into brain Competition Blocking the expression of the gene TRIP-Br2 - modulates fat storage by regulating energy expenditure and lipolysis
Increased lipolysis and energy expenditure - reduces obesity
Glucokinase - enzyme "glucose sensor"
Protects against obesity and insulin resistance Very prevalent disease
High mortality rates (due to other complications like heart disease and kidney failure)
Lots of background information on mechanism of disease is available but still no cure Importance With gene therapy, no more need for constant supply of medication or insulin injections - greater quality of life Impact Medications commonly prescribed (e.g. Amaryl, Glucophage, Diabeta, etc.) Competition Symptoms include high blood sugar/glucose levels, low insulin production, increased insulin resistance
Can lead to other conditions like heart disease, kidney failure, etc.
Type 2 caused by lifestyle/dietary issues
Current medication/insulin injections help, but cannot cure
How gene therapies work Cause of type 1 (speculated that it is an autoimmune disease)
If gene therapy can actually cure diabetes in humans
If a lower calorie diet (600 cal/day diet) can "cure" diabetes and if so, temporary or permanent?
Best approach to curing diabetes Knowns vs. Unknowns Facilitating drugs to brain tissue is extremely difficult because of its surrounding protection and its location in the body
We want to develop a method to make it easier for pharmaceuticals that can potentially treat brain related disorders to reach their target tissue.
We also want to localize the drugs to the brain so they don't induce harmful side effects Importance: In the world...
*4 million are affected by Parkinson's
*18 million people affected by Alzheimer's, estimated 34 million by 2025
*about 50 million people affected by Epilepsy Impact (Who is affected?) Challenges Blood Brain Barrier (BBB): a membrane that separates the brain extracellular fluid from capillaries.
Blood Cerebrospinal Fluid Barrier (BCF): a barrier that connects the epithelial cells on the surface of the choroid plexus. Barriers to the Brain Well Developed:
*polyamidoamine (PAMAM) starburst dendrimers
*xrays & ultrasound initiation
*build off of the advantages of existing methods
*add a CD4 protein (or something similar) to get past the immune system
*need to circumvent the Blood-Brain Barrier (analyze how) What do we already know? - How will the biological system be removed from the brain?
- Will the system or the by products of the system have any effect on body/brain functions?
- Will the immune system reject the biological system?
- How long and in what quantities should the drugs be delivered to the brain tissue? Unknowns... Gene Therapy Remyelination Quick Overview of Diabetes around 250 million people worldwide
380 million by 2025 http://www.ninds.nih.gov/disorders/parkinsons_disease/parkinsons_disease_backgrounder.htm
http://www.cdc.gov/features/worldalzheimersday/index.html Daily insulin injections Diet restrictions Other gene therapy methods We Know-
How myelin can be destroyed
We Don't Know-
-Which method is better? Stimulating present cells, or implementing new cells?
-How would new cells target/recognize cells in need?
-What effects would these methods have?
-Stimulation of the wrong cells? Overproduction? How would additional cells affect the body? Will disease target treatment? Knowns and Unknowns plant virus encapsulation *Barriers to Central Nervous System: blood-brain barrier & blood-cerebrospinal fluid barrier (BBB & BCB)
*Capillaries of the vertebrate brain and spinal cord lack the small pores that allow rapid movement of solutes from circulation into other organs 1 in 8 of those 65+ suffer from Alzheimer's