Clinical Trials
Numerous clinicians in North America, Europe and around the world are interested in finding new therapies for essential tremor. Each year trials are reported that were started after anecdotal favorable reports or “educated guesses.” Although much progress has been made through this empirical approach in identifying therapies, much more progress is needed. Eventually, we may anticipate essential tremor-specific therapies based on knowledge of the abnormal cell and the abnormal genes.
Relatively few centers are conducting clinical trials of therapies for essential tremor on a regular basis. One such site is that of Adrian Handforth, M.D. and Fredricka Martin, Ph.D. at the VA Greater Los Angeles Healthcare System. These investigators are utilizing a mouse model to screen for potential effectiveness. If the medication passes the mouse test, it is then assessed in a small open-treatment pilot clinical trial. The final stage will be a controlled clinical trial. These trials require a series of outpatient visits. Interested participants should be within driving distance. Subjects do not have to be a veteran, but should be at least age eighteen. Email: [email protected].
The Limiting Factor
Contrary to popular belief, the lack of ideas is not the most common factor holding up scientific progress. Instead it is the lack of funds that hamper progress. After a vigorous growth period of NIH research budgets, research funding has stabilized, so that new applicants will have a hard time obtaining funding. More than ever, essential tremor needs funding support from private donors.
Dr. Handforth’s call for funding is synonymous with TAN’s objective to stimulate an increase in both federal and private sector funding.
“Life is not merely to be alive, but to be well.”
Marcus Valerius Martialis
NINDS Clinical Trials
Pathogenesis Study of Essential Tremor
93-N-0202 is a new clinical trial for the study of the development of essential tremor. Patients will undergo nerve conduction tests, electromyograms, and blood testing. For more information, contact Dr. Kirsten Zeuner at (301) 402-3499 or Dr. Fiona Molloy at (301) 594-0937.
Octanol to Treat Essential Tremor
04-N-0147 evaluates the effectiveness of 1-octanol for treating essential tremor. This study is an active follow-up not currently recruiting new patients.
Pharmacokinetics of 1-Octanol in Adults with Essential Tremor
05-N-0092 characterizes the pharmacokinetics of 1-octanol in humans. Gas Chromatography (GC) will be used for the quantification of octanol from plasma and urine samples. Contact Patient Recruitment for more details.
Phenotype/Genotype Correlations in Movement Disorders
01-N-0206 identifies families with inherited movement disorders and evaluates disease manifestations to establish an accurate clinical diagnosis by using newest technological advances and investigate the underlying molecular mechanisms. For further information, contact Patient Recruitment.
Botulinum Toxin for the Treatment of Involuntary Movement Disorders
85-N-0195 examines the effectiveness of botulinum toxin as treatment for a variety of movement disorders. Patients will be eligible for participation if they have a movement disorder that, in the judgment of the treating physician, might be amenable to treatment with botulinum toxin. Contact Patient Recruitment for more information.
Roadmap
NIH Roadmap is regularly updated to reflect developments in three main areas: new pathways to discovery, research teams of the future, and re-engineering the clinical research enterprise.
The Brain Bank
Essential tremor is a disease that affects the lives of as many as one in twenty Americans but its cause remains a medical mystery. Brain donation (also called postmortem examination or autopsy) is the only way to solve this mystery. Unfortunately, there is a severe shortage of essential tremor brains. Medical researchers have only been able to study a few brains from people with essential tremor because, until recently, no national brain donor program has existed. Medical researchers at Columbia University have recently taken a huge step forward. In 2003, these researchers, lead by Elan Louis M.D., established for the first time a centralized brain bank to serve the essential tremor community. This will enable doctors to begin to actively study what happens in the brains of people with essential tremor. With a brain donor program, doctors will be able to study the changes in brain structure and metabolism that may explain the cause of essential tremor. Better treatments and, eventually, a cure for essential tremor, will only come about when doctors have developed a better understanding of these brain changes. Brain donation is essential to this effort. This can only be a collaborative effort involving doctors and people with essential tremor. We need your help. If you are interested in helping us to build this brain bank, please let us know by calling us at (212) 305-8513 or emailing us at [email protected] and we will send you an informational packet.
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Duke Center for Human Genetics
The Center for Human Genetics at Duke University Medical Center is actively recruiting large families for genetic studies in ET. Whenever possible, the family should be large enough to have living individuals (both with and without essential tremor) in at least three generations, many of whom are willing to participate in research. There is no cost to participate. Participation does NOT require travel to Duke University Medical Center.
Participating families will be asked to contribute the following forms of information:
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1. A telephone interview, at the familys convenience, to review the family history.
- 2. A review of the medical records of one person in the family who has been diagnosed with ET.
If the family history and availability of family members is appropriate, we will complete telephone interviews with eligible families. Eligible families will then be scheduled to meet with a member of our research team to obtain blood samples and videotaped, directed neurological examinations. The research team member will come to the familys home or any convenient location to do this.
No individual genetic results will be given to study participants. We will share our overall findings with families through periodic newsletters and scientific publications. All the information obtained from participants is kept strictly confidential.
For additional study information, contact Stefanie Knauer at (877) 244-9272 or via email, [email protected].
Utah Neurology
Dr. Kevin Flanigan is currently conducting essential tremor gene mapping studies in three very large Utah families, and a few smaller ones.
The Utah families are good candidates because of their size and detailed family records. For information regarding collaborative research studies, contact Dr. Flanigan at
(801) 587-9540.
Genizon BioSciences
Dr. Jean-Michel Vidal (M.D., Ph.D.) is recruiting from the French Canadian founder population located primarily in Quebec and in some specific surrounding locations, in order to identify genes responsible for essential tremor.
Genizon has introduced the novel approach of studying family PPC Trios (patient, spouse and biological child, or patient and his/her two biological parents). The recruitment of the patient for this study is through the neurologists who collaborate with Genizon.
French Canadians from Quebec who wish to participate can also call Dr. Vidal at
1 (888) 244-3088 or send an e-mail to [email protected].