Huntington's Disease Press Releases

Scientists have developed the first genetically altered monkey model that replicates some symptoms observed in patients with Huntington's disease. This advance, reported in Nature, could lead to major breakthroughs in the effort to develop new treatments for a range of neurological diseases.

Results of a placebo-controlled, double-blind phase II study of the antioxidant idebenone in children with Friedreich's ataxia (FA) suggest that the treatment may lead to improvements in neurological function. It is the first placebo-controlled study to suggest that the neurological deterioration associated with this disease can be slowed or reversed.

Lithium carbonate, a compound commonly used to treat depression, might also provide symptomatic relief for a group of inherited movement disorders that includes the fatal disease spinocerebellar ataxia type 1 (SCA1).

Added to its devastating neurological symptoms, Huntington's disease (HD) carries with it a lesser-known horror. The genetic mutation that causes the disease can grow larger, causing its symptoms – involuntary movements, dementia, and dramatic personality changes – to grow worse across generations and even during a single lifetime. New research sheds light on how the mutation grows and offers hope for locking it down.

The National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), has named Walter J. Koroshetz, M.D., as its Deputy Director. Effective January 2, 2007, he will work with the NINDS Director in program planning and budgeting, as well as oversee Institute scientific and administrative functions.

For the first time, researchers have linked mutations in a gene that regulates how potassium enters cells to a neurodegenerative disease and to another disorder that causes mental retardation and coordination problems. The findings may lead to new ways of treating a broad range of disorders, including Alzheimer's and Parkinson's diseases. The study was funded in part by the National Institutes of Health's National Institute of Neurological Disorders and Stroke (NINDS).

For the first time, researchers have identified how the gene for a hereditary neurodegenerative disease called spinocerebellar ataxia type 1 (SCA1) disables an important group of neurons in the brain. The findings improve understanding of how SCA1 and related diseases develop and may lead to new ways of treating them.

U.S. Department of Health and Human Services Secretary Michael O. Leavitt announces three new appointments and one reappointment to the National Advisory Neurological Disorders and Stroke Council, the major advisory panel of the National Institute of Neurological Disorders and Stroke (NINDS). The NINDS, a component of the National Institutes of Health (NIH), is the nation’s primary supporter of basic, translational, and clinical research on the brain and nervous system. NINDS Director Story Landis, Ph.D., will introduce the new members, who will serve through July 2009, at the Council’s September 15, 2005 meeting.

Silencing the activity of a mutant gene prevents disease symptoms in a mouse model for Huntington's disease (HD), a new study shows. The study is the first to directly target the underlying problem that causes HD, and it may lead to a new way of treating this disorder.

A new study shows that a common antibiotic used to treat bacterial infections increases survival rates and delays nerve damage in a mouse model for amyotrophic lateral sclerosis (ALS). The antibiotic works by activating or "turning on" the gene encoding the glutamate transporter in neurons. This finding may lead to new drug treatments for ALS and other neurodegenerative diseases.

Using a specially designed robotic microscope to study cultured cells, researchers have found evidence that abnormal protein clumps called inclusion bodies in neurons from people with Huntington's disease (HD) prevent cell death. The finding helps to resolve a longstanding debate about the role of these inclusion bodies in HD and other disorders and may help investigators find effective treatments for these diseases.
Fact Sheet

A new study reveals for the first time how gene mutations lead to the inherited form of amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease. The study suggests that the two most prominent theories of how familial ALS (FALS) and other related diseases develop are both right in part.
Fact Sheet

A new study shows that combining the supplement creatine and the antibiotic minocycline significantly slows disease progression and prolongs survival in a mouse model of amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease.
Fact Sheet

Results of the first placebo-controlled, multicenter clinical trial of the compound coenzyme Q10 suggest that it can slow disease progression in patients with early-stage Parkinson's disease (PD). While the results must be confirmed in a larger study, they provide hope that this compound may ultimately provide a new way of treating PD.
Fact Sheet

A new study shows that most patients with movement disorders caused by damage to the cerebellum also have psychiatric symptoms. The study suggests that patients with cerebellar diseases may benefit from screening and treatment of psychiatric symptoms.

The antibiotic minocycline delays onset and slows progression of symptoms in a mouse model for amyotrophic lateral sclerosis (ALS), a new study shows. The study also revealed that the drug may work by blocking release of a molecule that triggers cell death. The findings may lead to new ways of treating ALS or other neurodegenerative disorders.
Fact Sheet

A drug called cystamine alleviates tremors and prolongs life in mice with the gene mutation for Huntington's disease (HD), a new study shows. The drug appears to work by increasing the activity of proteins that protect nerve cells, or neurons, from degeneration. The study suggests that a similar treatment may one day be useful in humans with HD and related disorders.
Fact Sheet

A large-scale clinical trial that tested the ability of the investigational drugs remacemide and Coenzyme Q10 to slow the progression of Huntington's disease showed that neither drug resulted in any significant improvement for the patients. Although after one year of treatment, the disease seemed to progress more slowly in patients treated with Coenzyme Q10, the investigators say that overall the results are inconclusive as to whether there is real benefit from this drug.
Fact Sheet

Scientists have identified the gene altered in one of the most common hereditary ataxias, spinocerebellar ataxia 2 (SCA2). The discovery allows improved genetic testing and provides new clues about how genetic mutations cause several neurological disorders, including Huntington's disease. The findings are reported by three different groups in the November issue of Nature Genetics.

Scientists have identified a new type of trinucleotide repeat mutation that leads to Friedreich's ataxia (FA), a rare childhood neurodegenerative disease. The discovery allows accurate screening for carriers of the disease and may lead to the first effective treatments.

For the first time, scientists have linked a critical cellular enzyme to the gene defect found in Huntington's and several other hereditary neurological diseases. The finding provides important clues about how these diseases may develop and suggests that a single therapy eventually may be developed to treat them.

Scientists have discovered that another nervous system degenerative disorder, spinocerebellar ataxia type 1 (SCA1), has the same type of gene mutation occurring in Huntington's and Kennedy's diseases. In the disease, a normal three-base sequence in the genetic code — cytosine, adenine and guanine, or CAG — is abnormally repeated, according to Drs. Huda Y. Zoghbi, who led one team at the Baylor College of Medicine in Houston, Texas, and Harry T. Orr, who headed the other team at the University of Minnesota in Minneapolis. The same CAG repeat was reported earlier this year in Huntington's disease and in 1991 in the very rare Kennedy's disease, also called X-linked spinobulbar muscular atrophy.

Scientists have identified the genetic mutation that causes Huntington's disease (HD), a fatal, neurodegenerative disorder characterized by progressive physical and mental deterioration. The discovery, to be reported in the March 26 issue of Cell,* is the culmination of a 10-year-long collaboration between investigators in six laboratory groups around the world with major support from the National Institute of Neurological Disorders and Stroke (NINDS).

Using a novel strategy, scientists from the National Institute of Neurological Disorders and Stroke have isolated segments of DNA sequence that uniquely identify more than 2,300 brain genes. The recent data, combined with data from 347 segments sequenced earlier by NINDS scientists, doubles the total number of human genes identified by sequencing, scientists report in the February 13 issue of Nature.

Using a novel strategy, scientists at the National Institute of Neurological Disorders and Stroke (NINDS) have isolated key identifying regions of more than 400 genes that work inside the human brain. The scientists say their work should help identify genetic defects that cause brain disease and speed progress of genetics research.