Therapeutic Research

Clinical trials are an important part of memory and cognitive disorders research. The information that is gathered from clinical trials is used in determining whether certain treatments are effective and safe for patients. Researchers depend upon the willingness of people with Alzheimer’s disease and other memory disorders to aid in the advancement of these treatments.


Modification of Adult Cells for Therapeutic Applications in Alzheimer’s Disease
David Borchelt, PhD

Alzheimer’s disease is thought to result from the deposition of a chemical called amyloid in the brain, which results in the death of nerve cells and the resulting dementia. Dr. Borchelt’s expertise in producing and analyzing mice that produce excessive amounts of amyloid, reproducing aspects of Alzheimer’s disease provides an opportunity to test novel therapies quickly and effectively, and thereby hasten the identification of new effective therapies. The University of Florida has been a leader in applications of stem cell therapy and in developing the technology to use recombinant viruses to deliver therapeutic agents. The emphasis in stem cell research at UF has been on using stems found in the adult nervous system.

Three phases are planned in developing these technologies as therapeutics for humans. First, the laboratory will use mouse models to test a number of parameters including, genetic modification of the stem cells to produce potentially therapeutic molecules, routes of delivery, and pre-clinical assessments of efficacy. The second phase involves isolation of human neural stem cells, genetic modification of these cells, and pre-clinical assessments of safety in animals. The third phase, will be to establish the safety and potential efficacy of modified human stem cells as therapeutics in humans. We are fortunate that colleagues here at the MBI are in engaged in similar types of studies to develop stem cell therapies for Parkinson’s disease, and thus information gained from their effort and visa versa will likely speed up development for both diseases. Similar types of approaches hold promise for a number of other neurodegenerative diseases, including Progressive Supranuclear palsy, Multisystem Atrophy, Fronto-Temporal Dementia, Diffuse Lewy body disease, Huntington’s disease and amyotrophic lateral sclerosis.


Leukoariosis in Alzheimer’s Disease
KM Heilman, MD

Patients with Alzheimer’s disease and patients with vascular dementia (i.e. from small strokes) frequently have brain lesions on magnetic resonance imaging (MRI) scans, which involve the white matter that is composed of nerve fibers connecting different parts of the brain. These white matter lesions are called leukoaraiosis (LA). The cause of LA is not known with certainty and may be due to a variety of pathological processes with different risk factors that interact to produce different clinical manifestations, patterns of cognitive disturbance, and potential hazards. It is unclear whether the processes leading to LA are the same in patients with and without Alzheimer’s disease and whether more than one process leads to changes in each individual patient. The first goal of this project seeks to determine whether patients diagnosed with Alzheimer’s disease demonstrate different patterns of cognitive and behavioral dysfunction, cognitive-motor deficits, and personality changes when LA is detected. The second goal of this project is to examine postmortem brain to determine the exact pathology of LA imaged on MRI. The third goal of this project is to develop therapies for patients with LA and Alzheimer’s disease. Two compounds which we planned to test are ergoloid mesthylate (hydergine) and mucomyst (N-acetylcysteine) in order to determine if cognition and memory are improved by these treatments.


Treating Patients with Alzheimer’s Disease with Significant Subcortical White Matter Lesions with Donepezil
KM Heilman, MD

This study is designed to examine 1) the role of white matter abnormalities on the clinical/behavioral manifestation of Alzheimer’s Disease and 2) the relationship between white matter abnormalities and responsiveness to a commonly used medication for memory problems (donepezil).


Errorless Learning Treatment of Anomia in Alzheimer’s Disease
KM Heilman, MD

Many people with dementia are able to learn skills fairly well, even when they have trouble learning specific facts. People with dementia often remember their own mistakes (which are their own actions) better than they remember the corrections to their mistakes (which is usually information they hear from someone else). Errorless learning is a way to get someone to learn something by saying or doing it, rather than by telling or showing. The person is not given the opportunity to make a mistake, so there are no mistakes to be remembered. Our preliminary studies suggest that errorless learning can help Alzheimer patients. This study will determine if errorless learning can reduce word finding problems (anomia) in patients with Alzheimer’s disease.


Note that this list includes those projects linked to the Alzheimer Research Center, but does not include all dementia related research at the University of Florida. If you are interested in participating in some memory disorder or Alzheimer’s disease research opportunities, please check this web site for upcoming clinical trials.