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The Latest: Will be moved as others are uploaded |
RNAi
Suppresses Polyglutamine-Induced Neurodegeration In A Model of Spinocerebellar Ataxia - Nature Medicine Published Online July 4, 2004: Haibin Xia, Qinwen
Mao, Steven L. Eliason, Scott Q. Harper, Ines H. Martins, Harry T. Orr, Henry L. Paulson, Linda Yang, Robert M. Kotin &
Beverly L. Davidson
Nature Medicine article on the RNAi success on SCA1 at the University of Iowa presented at the American Society of Gene Therapy. According to their press
release they were nominated the top abstract at the conference. Note that this study a ‘two-fer’. It is not only
a statement of success on RNAi…it is a statement on their success using a viral vector to deliver the therapy
Related news releases:
University of Iowa researchers have shown for the first time that gene therapy delivered to the brains of living mice
can prevent the physical symptoms and neurological damage caused by an inherited neurodegenerative disease that is similar
to Huntington's disease (HD).
The
study appeared in the August issue of Nature Medicine and in the journal's advanced online publication Sunday.
If the approach can be used in humans, it could provide treatment for a group of incurable, progressive
neurological diseases called polyglutamine-repeat diseases, which include Huntington's disease.
US scientists
have prevented mice from developing a hereditary brain disease by injecting their brains with fragments of genetic material
designed to switch off unhealthy genes. The research paves the way for similar therapies in humans, including treating Alzheimer's
and Huntington's diseases. Five-week-old mice with spinocerebellar ataxia - an untreatable, hereditary neurodegenerative condition
similar to Huntington's - were treated using a gene therapy specifically targeted to eradicate the diseased genes. |
Rapamycin Study In Huntington's
Disease
Input
from HD Researcher:
This study adds to our growing list of compounds that have been shown to have a good effect
against HD in animal models.What I think is equally as exciting about the news of a good effect of rapamycin is the way in
which the drug works - by unblocking autophagy,which is blocked by TOR. Autophagy is the garbage collection system in the
cell, and lots of research has gone on in the last year studying this mechanism as it relates to HD. The support that this
paper lends to the notion that autophagic stimulation could be beneficial to HD means that research in this area will become
more focused and hopefully accelerated.
5 page PDF Document
Huntington's disease may be more straightforward
to fight than doctors have feared, paradoxically because the genetic brain disorder is more complicated
than anyone knew, U.S. researchers said on Thursday. Their research in fruit flies shows that nerve cells modify the mutated
protein responsible for Huntington's disease, and this basic cell process could in theory be altered with a drug. The researchers believe their finding, published in this week's issue of the journal Science, opens a new approach
to treating the fatal and incurable disease. |
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New Model for Prediciting
Age of Onset in HD Mar 2004 PDF 11 pages
A new
model for prediction of age of onset and penetrance for Huntongtin's diseased based on CAG length.
Anne B. Young "As many steps in the process of functional decline and cell death represent
potential drug targets, we should eventually find a cure."
The current study suggests that quantitative assessment of basal
ganglia may provide a means to trace early signs of decline in individuals with the Huntington's gene mutation prior to clinical
onset.
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Scientific
Brochure
October 11, 2002 Word 51 pages
Update on HSG Clinical Trials
Recent Results, Current
Studies, Future Plans
The implicit aim of current research in HD is to find a cure for this terrible disorder. Although
this goal has eluded us so far, ideas about which strategies to pursue are not lacking. Treatments can be classified into
primary (treating asymptomatic gene carriers in order to postpone or prevent symptomatic conversion) and secondary (treating
affected individuals, to postpone or prevent progression) neuroprotective interventions. In addition, symptomatic treatments
intend to alleviate the burden of the disease, while restorative treatments aim at reversing the decline that has already
taken place.
December 2002 Word 8 pages
Cattaneo, Elena; Rigamonti, Dorotea; Zuccato, Chiara: Armed with a better understanding
of the complexities of Huntington's disease, we can now turn to devising better treatments for the disease. The drugs that
are currently available alleviate just some of the symptoms of the disorder and can have serious side effects.Indeed, the
drugs often ameliorate one symptom only to make another worse. Although doctors commonly prescribe sedatives to people with
Huntington's to control their involuntary movements, these drugs also lower levels in the brain of the neurotransmitter dopamine,
worsening the person's depressive symptoms. |
Impact
On Couple Relationships of Predictive Testing
To Huntington Disease: A Longitudinal Study -2003 Fiona Richards, Department of Clinical Genetics and Katrina Williams,
Department of Clinical Epideiology,The Children's Hospital at Westmead, Sydney, Australia PDF 9 pages
This longitudinal study compared the level of marital adjustment of 23 couples in which the
at-risk partner is undergoing predictive testing for HD [the testing group] with that of 20 couples in which the at-risk partner
is not under-going testing [the non-testing group]. |
Couples' Experiences of Predictive Testing and Living With the Risk of Reality of Huntington Disease: A Qualitative
Study - 2003 Fiona Richards, Department of Clinical Genetics, The Children's Hospital at Westmead, Sydney, Australia
PDF 13 pages
The findings of this study highlight both the individuality and the complexity of
psycho-logical effects on the intimate relationships of couples who live with the risk or reality of HD, and provide
important insights for professionals offering support to these couples. |
Potential Treatments/Medications |
Science article on the Huntington's Disease Drug Works [HDDW] program! |
Paxil Delays Onset
Apr. 2004
5 page
PDF Document
Paroxetin Retards Disease Onset and Progression Huntingtin Mutant Mice
We report that administration of paroxetine, a widely prescribed antidepressant drug
that acts by inhibiting reuptake of the neurotransmitter serotonin, suppresses the neurodegenerative process and increases
the survival of huntingtin mutant mice, an animal model of Huntington's Disease [HD]. |
A Study WORD 18 pages
Evidence showed an inverse correlation between the onset of cognitive impairment and the number
of trinucleotide repeats in the HD gene. Additionally, this study showed that although a statistically significant correlation
exists between the number of repeats and the progressive dementia, the relationship is tenuous.
Impact
of Huntington's Disease
On The Individual and The Family
Mar 2004 Word 12 page Slide Show
By Melinda Kavanaugh, MSW
Washington University School of Medicine
HD What We See, What We Don't See, The Issues, Inheritance, Discrimination, Insurance Discrimination.
Relationships, Self Worth and Esteem, Financial Hardships, Care & Treatment
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Successful
Multimodality Treatment of Severe Behavioral Disturbancein a Patient With Advanced Huntington's Disease Dec. 2001 Word 12 pages
Treatment
of the psychiatric manifestations of Huntington's disease include pharmacological and behavioral interventions. Here we discuss
the case of a young man with advanced Huntington's disease and severe behavioral, affective, perceptual, and cognitive disturbances
who was successfully treated by using a combination of approaches within a highly structured, multidisciplinary inpatient
setting. This case discussion will also illustrate the complex psychiatric symptoms that emerge in Huntington's disease or
as a result of its treatment, the use of a differential reinforcement schedule in advanced dementia, and the important
role of a tertiary care center in the treatment of this kind of complicated illness. This case highlights many challenges
inherent in the care of chronically ill patients with degenerative disorders and ultimately illustrates the clear benefits
of applying intensive diagnostic and treatment methods, despite the impossibility of curing the underlying disorder |
Huntington's
Disease An Insurance Model 2004 PDF 28 pages. Epidemiology of HD, fitting models to [a] rate of onset, depending on
CAG repeath lenght as well as age; [b] to post-onset rates of mortality; and [c] to the distibution of CAG repeat lengths
in the population.
HD Critical Illiness
Insurance and Life Insurance 2004 PDF 24 pages. Using above, the critical illness and life insurance markets were studied.
Caculates premiums based on genetic test results that disclose the CAG repeat length, or more simply a family history of HD.
Complexities suggest that restrictions on disclosure, rather than use, of genetic information. |
A
Personal Perspective on Estate Planning For HD Families PDF 18 pages. Written
by Steven Hurst for the Huntington's Society of Canada. Covers Getting Started; Round One-Where We Need Help, Round
Two- Getting Details, How Much Life Insurance?, Guarding Against The Cost of HD, Using Trusts To Protect
Resources, The Law & Family Trusts, Early Planning-A Must For HD Trusts, |
Note: These latest article once again, will stir up the hopes
for fetal transplant in HD in a country [USA] where it would be nearly impossible for HD patients to participate in any clinical
trials for fetal transplantation. For those families seeking this procedure for someone with HD, they need to keep
in mind the restrictive guidelines for accepting patients. Those guidelines were established based on the outcome
of the earlier procedures done in the USA at both the University of South Florida, Tampa, Fl. and The Neurosciences Institute,
Good Samaritan Hospital, Los Angeles, CA. These procedures were not referenced in latest article however reference to
them can be found in the other information below. |
We use here the example of Huntington's disease to show what fetal neural transplants may uniquely
offer for that disease. Article concludes " Symptomatic relief of chorea and psychiatric symptoms in particular seems
with reach already, but should clearly be combined with a neuroprotective treatment as soon as one can be identified.
Neural replacement, through fetal neural transplantation, which it is the only option at the
present time, will be a welcome complement, providing patients with the possibility not only of stabilization but also even
of the recovery of affected functions." |
2003 Mar Word 1 page University of South Florida, Tampa, FL - Outcome of procedures with
7 HD patients. Concludes: Transplantation of human fetal striatal cells is feasible and survival of transplanted cells
was demonstrated. Patients with moderately advanced.
Dec 5 2000 PDF 6 pages University of South Florida, Tampa, FL Seven HD patients.
This study demonstrates that grafts derived from human fetal striatal tissue can survive, develop,
and are unaffected by the disease process, at least for 18 months, after transplantation into a patient with HD. |
PDF 8 pages Providing general description of HD from the Huntington's Society of Canada |
Stanford University has given permission for me to share this excellent document written for young people
living HD until they are able to upload it to their website. Please visit their website for the HD HOPES Project -> Stanford University HD HOPES Project |
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