Baltimore - Johns Hopkins University

Valina Dawson, Ph.D.
(RG) Targeted gene therapy in motor neuron disease
$ 66,817.00	7/1/99 - 6/30/00	Year 3

Summary: Adenovirus carrying neuroprotective genes will be given to transgenic familial amyotrophic lateral sclerosis (FALS) mice to see if this treatment can delay FALS disease and death.

Daniel Drachman, M.D.
(RG) Specific immunotherapy of myasthenia gravis (MG):  
Targeting by "safe" stimulation
$ 75,000.00	7/1/99 - 6/30/00	Year 3

Summary: This study will use new strategies to treat MG by "targeting" and inactivating or killing, only those cells of the immune system that are specifically involved in the disease-producing process. These novel methods may be applicable to other autoimmune neuromuscular diseases as well.

(RG) Transgenic myasthenic mice: Production and characterization
$ 54,000.00	7/1/99 - 6/30/00	Year 1

Summary: Ideally, treatment of MG should specifically eliminate only those cells of the immune system that are specifically involved in the disease. Researchers will develop a new transgenic mouse model, in which the great majority of T lymphocytes are specific for the disease process. This model will tremendously enhance development of specific treatments for MG and other neuromuscular autoimmune diseases.

(EMG) Restricted gift for research
$ 17,708.00	4/1/99 - 3/30/00	Year 1

Raquelli Ganel, Ph.D.
(DG) Mechanism for aberrant glutamate transport 
in amyotrophic lateral sclerosis (ALS)
$ 35,000.00	1/1/00 - 12/31/00	Year 1
$ 35,000.00	1/1/01 - 12/31/01	Year 2
$ 35,000.00	1/1/02 - 12/31/02	Year 3

Summary: Glutamate transporters prevent excitotoxicity, a possible cause of ALS. The proposed studies will further our understanding on the underlying cellular mechanisms responsible for loss of glutamate transporter in ALS, which may also provide a new therapeutic approach.

Diane Griffin, M.D., Ph.D.
(RG) Genetic determinants of susceptibility 
to fatal motor neuron disease
$ 50,000.00	1/1/00 - 12/31/00	Year 1
$ 50,000.00	1/1/01 - 12/31/01	Year 2

Summary: The genes that lead to susceptibility to fatal paralytic diseases like ALS will be identified using a mouse model of virus-induced motor neuron disease.

J. Marie Hardwick, Ph.D.
(RG) Role of SMN:Bcl-2 interactions in neuron survival
$ 41,952.00	7/1/99 - 6/30/00	Year 2
$ 43,177.00	7/1/00 - 6/30/01	Year 3

Summary: This project is a study of the neuron survival mechanism mediated by the pro-survival protein Bcl-2 and the SMN protein mutated in spinal muscular atrophy (SMA).

Douglas A. Kerr, M.D., Ph.D.
(DG) Investigation of the role of survival of motor 
neuron protein in neuronal apoptosis
$ 35,000.00	7/1/99 - 6/30/00	Year 1
$ 35,000.00	7/1/00 - 6/30/01	Year 2

Summary: This study will investigate mechanisms underlying the ability of SMN to modulate neuronal death by evaluating the effect of distinct SMN protein derivatives expressed in central nervous system neurons utilizing a novel viral delivery system and a transgenic animal approach.

Ralph W. Kuncl, M.D., Ph.D.
(RG) Potent neuroprotection of motor neurons by PEDF
$ 60,000.00	7/1/99 - 6/30/00	Year 2
$ 60,000.00	7/1/00 - 6/30/01	Year 3

Summary: A new growth factor from the retina, PEDF, has properties predicting its importance for survival and outgrowth of motor nerve cells. This study will elucidate its basic biology, efficacy, and mechanisms as a basis for its development as a potential neuroprotective treatment for ALS and SMA.

Jeffrey Rothstein, M.D., Ph.D.
(RG) Neuroprotective properties of TGFß family 
neurotrophic factors
$ 75,879.00	1/1/00 - 12/31/00	Year 3

Summary: Researchers will use a unique culture system to explore a new family of trophic factors, and important, new small chemicals that mimic the actions of trophic factors to determine if they will be useful ultimately in amyotrophic lateral sclerosis (ALS) patients.

(RG) Copper chaperone (CCS) and protein aggregation 
in amyotrophic lateral sclerosis (ALS)
$ 104,265.00	1/1/00 - 12/31/00	Year 2
$ 107,441.00	1/1/01 - 12/31/01	Year 3

Summary: These experiments will explore a new idea that the familial form of ALS (FALS) could be due to aggregation of the mutant protein with other critical cellular proteins.

(RG) Clinical trial of creatine in amyotrophic lateral sclerosis (ALS)
$ 225,276.00	9/1/99 - 8/31/00	Year 1
$ 19,893.00	9/1/00 - 2/28/01	Year 2

Summary: A multi-center clinical trial of the nutritional supplement creatine to determine benefit in ALS will be conducted.

Baltimore - University of Maryland

Robert J. Bloch, Ph.D.
(RG) Macromolecules of the sarcolemma
$ 58,310.00	7/1/99 - 6/30/00	Year 2
$ 61,226.00	7/1/00 - 6/30/01	Year 3

Summary: A novel way to document and quantify the changes in the muscle membrane that occur in muscular dystrophy has recently been discovered. Researchers propose to use the new method to learn how these changes are related to the severity of muscle disease. This approach should lead to a better understanding of the mechanisms leading to muscular degeneration and the designing of better methods for assessing the efficacy of genetic therapies.

Robert J. Bloch, Ph.D.
(RG) Intermediate filaments that organize the sarcolemma
$ 35,000.00	1/1/00 - 12/31/00	Year 1

Summary: A network of proteins including dystrophin and filaments linking the dystrophin network to nearby structures helps maintain membrane stability. How this is accomplished and then disrupted in disease will be determined.

Bethesda - National Institutes of Health

Kenneth Fischbeck, M.D.
(RG) Connexin and X-linked Charcot-Marie-Tooth (CMTX) disease
$ 65,531.00	1/1/99 - 12/31/00	Extension

Summary: Patients with CMTX have mutations in connexin 32. Researchers will study how these mutations cause the manifestations of the disease.

(RG) Androgen effects on motor neurons
$ 71,051.00	7/1/99 - 6/30/00	Year 2
$ 73,850.00	7/1/00 - 6/30/01	Year 3

Summary: Researchers will investigate how androgens work in motor nerve cells as a step toward identifying new factors that may promote motor neuron survival.

Return to MDA Research Grants

| What's New | Diseases | Research | Clinics & Services | Community Programs | Publications | En Español | Telethon | Ways to Help | Video | Search | Site Map | Help Now | Home |