Our Research

Research Home
All of our projects depend on the creation and advancement of direct infusion of drugs and therapies into the brain.
Brain Cancer
We have developed a technique in which nanoparticles, including viral vectors and liposomes can be infused directly into brain tumors to give enhanced drug efficacy.
Parkinson's Disease
For many years, and continuing still, we have been working on development of direct drug delivery into the brain including cell transplantation, gene transfer and growth factor infusions for Parkinson's disease.
Niemann-Pick
Through gene therapy, we are working to eliminate the gene responsible for Niemann-Pick (acid sphingomyelinase).
Dyskinesia
By studying the effects of L-Dopa on the brain, we are developing gene therapy for L-Dopa-induced dyskinesia.

Glossary

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AADC

This acronym stands for aromatic L-amino acid decarboxylase, the enzyme responsible for converting L-Dopa into Dopamine. AADC is found in peripheral tissues (liver, kidney, adipose, heart, adrenal gland and keratinocytes) and also a number of brain regions. It is most abundant in substantia nigra, the neurons that degenerate in Parkinson's disease. This loss of AADC activity through depletion of host cells is believed to play a major role In progressive loss of response to the drug, L-Dopa, as the disease advances. This phenomenon forms the basis of an enzyme replacement strategy where the the AADC gene is introduced into the striatum to restore L-Dopa responsiveness.

AAV

Adeno-associated virus (AAV) is a small virus which infects humans and some other primate species. AAV is not currently known to cause disease and consequently the virus causes a very mild immune response. AAV can infect both dividing and non-dividing cells and may incorporate its genome into that of the host cell. These features make AAV a very attractive candidate for creating viral vectors for gene therapy. (from Wikipedia at http://en.wikipedia.org/wiki/Adeno-Associated_Virus)
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Cannula

A cannula (from Latin "little reed"; plural cannulae) is a flexible tube which when inserted into the body is used either to withdraw fluid or insert medication. (from Wikipedia at http://en.wikipedia.org/wiki/cannula)

cDNA

In genetics, complementary DNA (cDNA) is DNA synthesized from a mature mRNA template in a reaction catalysed by the enzyme reverse transcriptase. (from Wikipedia at http://en.wikipedia.org/wiki/CDNA)

Convection-Enhanced Delivery (CED)

Convective delivery allows for the safe, targeted, homogeneous delivery of agents into small and large tissue volumes (multiple orders of magnitude larger than diffusion-driven processes for large molecules) in a manner that bypasses the blood-nervous system barrier. (from AANS.org at http://www.aans.org/library/Article.aspx?ShowMenu=false&ShowPrint=false&ArticleId=26334)
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Dopamine

Dopamine is a hormone and neurotransmitter occurring in a wide variety of animals, including both vertebrates and invertebrates. (from Wikipedia at http://en.wikipedia.org/wiki/Dopamine)

Dyskinesia

Dystonia (or dyskinesia) is a neurological movement disorder in which sustained muscle contractions cause twisting and repetitive movements or abnormal postures. (from Wikipedia at http://en.wikipedia.org/wiki/Dyskinesia)
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F

FMT-PET

Positron emission tomography (PET) is a nuclear medicine medical imaging technique which produces a three-dimensional image or map of functional processes in the body. (from Wikipedia at http://en.wikipedia.org/wiki/Positron_emission_tomography) FMT stands for Fluoro-metatyrosine, a substrate for AADC. The enzyme converts FMT mainly to 6-fluoro-hydroxyphenylacetic acid, and this metabolite remains trapped within the tissue. The approximate level of AADC activity can be gauged by the FMT-PET signal recorded by a PET scanner.
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Gadolinium

An MRI contrast agent, used in the Bankiewicz lab to dye certain drugs as they are delivered into the brain.

Gene Therapy

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one. (from Wikipeda at http://en.wikipedia.org/wiki/Gene_therapy)

Glia-Derived Growth Factor (GDNF)

Glial cell line-Derived Neurotrophic Factor (GDNF) is a small protein that potently promotes the survival of many types of neurons. (from Wikipedia at http://en.wikipedia.org/wiki/GDNF)

Glioblastoma Multiforme

Glioblastoma multiforme (GBM), also known as grade 4 astrocytoma, is the most common and aggressive type of primary brain tumor, accounting for 52% of all primary brain tumor cases and 20% of all intracranial tumors. (from Wikipedia at http://en.wikipedia.org/wiki/Glioblastoma_multiforme)
H

Huntington's Disease

Huntington's disease (HD), also known as Huntington disease and previously as Huntington's chorea and chorea maior, is a rare inherited neurological disorder affecting up to almost 8 people per 100,000. (from Wikipedia at http://en.wikipedia.org/wiki/Huntington%27s_Disease)
I

Interleukin-10

nterleukin-10 (IL-10 or IL10), also known as human cytokine synthesis inhibitory factor (CSIF), is an anti-inflammatory cytokine. (from Wikipedia at http://en.wikipedia.org/wiki/Interleukin_10)
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L

L-Dopa (Sinemet)

Levodopa is used as a prodrug to increase dopamine levels for the treatment of Parkinson's disease, since it is able to cross the blood-brain barrier whereas dopamine itself cannot. Once levodopa has entered the central nervous system (CNS), it is metabolized to dopamine by aromatic L-amino acid decarboxylase. (from Wikipedia at http://en.wikipedia.org/wiki/L-Dopa)

Liposomal Gadoteridol

The drug, Gadoteridol, is a chelated form of the ionic form of the element, Gadolinium. This material is strongly visible in a Magnetic Resonance Imager (MRI). By incorporating Gadoteridol into small lipid globules called liposomes, we have created a reagent that can be used to track the movement of other nanoparticles when they are co-infused into brain tissue.

Liposomes

A liposome is a spherical vesicle composed of a bilayer membrane. (from Wikipedia at http://en.wikipedia.org/wiki/Liposome)
M

MPTP

MPTP (1-methyl 4-phenyl 1,2,3,6-tetrahydropyridine) is a chemical that is related to the opioid analgesic drugs... MPTP causes Parkinsonian side-effects, hence some users of MPPP develop these symptoms. (from Wikipedia at http://en.wikipedia.org/wiki/MPTP)

MRI

Magnetic resonance imaging (MRI), formerly referred to as magnetic resonance tomography (MRT) and, in scientific circles, nuclear magnetic resonance imaging (NMRI) or NMR zeugmatography imaging, is a non-invasive method used to render images of the inside of an object. (from Wikipedia at http://en.wikipedia.org/wiki/MRI))
N

Neurodegeneration

Neurodegeneration is progressive loss of structure or function of neurons, including death of neurons. (from Wikipedia at http://en.wikipedia.org/wiki/neurodegeneration)

Niemann-Pick Disease

Niemann-Pick disease is an inherited condition involving lipid metabolism (the breakdown and use of fats and cholesterol in the body) in which harmful amounts of lipids accumulate in the spleen, liver, lungs, bone marrow, and brain. (from Wikipedia at http://en.wikipedia.org/wiki/Niemann-Pick_disease)

Nigral (substantia nigra)

The substantia nigra (Latin for "black substance", Soemering) or locus niger is a heterogeneous portion of the midbrain, separating the pes (foot) from the tegmentum (covering), and a major element of the basal ganglia system. (from Wikipedia at http://en.wikipedia.org/wiki/Nigral)
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Parkinson's Disease

Parkinson's disease (also known as Parkinson disease or PD) is a degenerative disorder of the central nervous system that often impairs the sufferer's motor skills and speech. (from Wikipedia at http://en.wikipedia.org/wiki/Parkinson%27s_disease)

Putamen

The putamen is a structure in the middle of the brain, which, together with the caudate nucleus forms the dorsal striatum. (from Wikipedia at http://en.wikipedia.org/wiki/Putamen)
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S

Sphinogmyelinase (hASM)

Acid Sphingomyelinase is an enzyme that resides in an organelle called the lysosome. It is responsible for breakdown of sphingomyelin, a constituent of many cell membranes. Absence of the enzyme causes a rare genetic disease called Niemann-Pick disease.

Striatum

The striatum is a subcortical part of the telencephalon. It is the major input station of the basal ganglia system. Anatomically, the striatum is the caudate and the putamen. (from Wikipedia at http://en.wikipedia.org/wiki/Striatum)
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V

Viral Vector

Viral vectors are a tool commonly used by molecular biologists to deliver genetic material into cells. (from Wikipedia at http://en.wikipedia.org/wiki/Viral_vector)
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