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Research, drugs, treatment, new diagnosis Forum for medical issues, and for the recently diagnosed (and families)

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Old 03-08-15, 01:18 AM   #41
shiraz
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Default Re: Another Cautionary Tale About Breakthrough Cures

Melody....

There's safety in numbers...spread the word. I mean....what's that he's got in his hand? A radar, a ray gun, a melting 69er...oops, 99er - if it is the chocolate flake has gone....we should be doubly worried!!
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Old 03-08-15, 09:49 PM   #42
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Default Re: Another Cautionary Tale About Breakthrough Cures

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Melody....

There's safety in numbers...spread the word. I mean....what's that he's got in his hand? A radar, a ray gun, a melting 69er...oops, 99er - if it is the chocolate flake has gone....we should be doubly worried!!
Poor Allan you do a sterling job, I hope you have a good sense of humour. x
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Old 26-08-15, 01:06 AM   #43
Allan
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Default Another Cautionary Tale About Breakthrough Cures

.
Now that all the fun and games are over have a look through this: HD Insights: A Huntington disease research periodical

To me, it stretches from Amazing - all the way through to Abhorrent, Desirable and Objectionable - all at the same time.

...... and I'm sure that mouse's nose twitched!

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Old 19-10-15, 06:42 PM   #44
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Default Re: Another Cautionary Tale About Breakthrough Cures

.

This is what we’ve all been waiting for – apparently.

http://hdresearch.ucl.ac.uk

www.bbc.co.uk/news/health-34552041

http://medicalxpress.com/news/2015-10-patients-dosed-gene-silencing-drug.html

www.uclh.nhs.uk/News/Pages/FirstHuntington.aspx

I find it interesting that they don’t mention numbers. There were originally 36 volunteers in Canada, Germany and England [6]. I recently saw that the number is now 32 and I understand that there are now 4 in England.

So, a “global” trial with 32 volunteers.

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Old 19-10-15, 07:17 PM   #45
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Default Re: Another Cautionary Tale About Breakthrough Cures

I have about switched off totally to all of this cure business..call me a pessimist but I am not normally I just cant/daren't raise my hopes. Saw the bbc news about it and have to say it was less than convincing...maybe it's a start...xx
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Old 19-10-15, 09:34 PM   #46
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Default Re: Another Cautionary Tale About Breakthrough Cures

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This is great news and real hope for the future. No, its not a cure as such, but it has got the potential to improve symptoms and maybe even reverse them as it has in the Huntington's mice.

Well done to the hd sufferers who are taking part in this first clinical trial. You are the real heroes, its a big ask to be the first people to be injected with an experimental brain drug.....even though quite rightly there has to be rigorous safety risk assessments throughout the 10 years of development to get to this stage. Good luck guys and gals, I know all HD families thank you from the bottom of their hearts.

Check out Enroll HD for more hd research and how to get out involved .....not all the research is clinic based and your details are confidential.

M x (needing that little bit of hope)
I think we all need a bit of hope... OK so we are some way off, but at least there is something new...I do understand Banda... The media hype these things up etc. But at least something new is being tested and hats off to all those, however few, taking part.

Take care all. x

Sorry Allan, forgot to say, thanks for posting. I wasn't aware. x
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Old 19-10-15, 11:46 PM   #47
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Default Re: Another Cautionary Tale About Breakthrough Cures

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I’m not one for generally taking in outspoken or media headlines – so I have to do my own sort of research. Here’s a short history of “Gene Silencing” research. But first something I read over the weekend on the Huntington’s Disease UK facebook page [NOT the HDA]. It has a text banner on the right hand side describing HD. Here are two paragraphs:
  • There is currently no cure for HD. Only symptomatic treatment is available. Death is normally caused by secondary infection, choking or suicide. [see point 1 below]
  • Each family situation and HD sufferer are different, making the disease and symptoms even more problematic to predict and deal with. You can’t predict how the sufferer will be from week-to-week or how fast they may deteriorate.
Slightly incorrect but hard facts, nonetheless. Now, here’s the complete history of the current “Gene Silencing” research\trials: CDHI + ISIS =

Andrew Shechtel and the Gabriel Trust and Endurance Funding Trust: http://digitallyclaim.com/?p=1214

www.philanthropyroundtable.org/almanac/medicine_and_health/1997_a_crusade_against_huntingtons_disease

www.businessweek.com/graphics/2014_billionaire_charities

So, although a somewhat secretive guy, the CHDI donor has always been known to be Andrew Shechtel.

http://chdifoundation.org/isis-and-chdi-renew-collaboration

www.newshd.net/tag/isis-pharmaceuticals

Here are a few points that I picked out that influence my sometimes, seemingly unpopular views on Pharmaceutical companies and the research that they finance:
  1. Symptoms of HD usually appear between the ages of 30 to 50 years, and continually worsen over a 10 to 25 year period. Ultimately, the weakened individual succumbs to pneumonia, heart failure or other complications. Presently, there is no effective disease modifying treatment, and current approaches only focus on managing the severity of some disease symptoms.
  2. If the results of the Phase II trial are positive, a Phase III trial involves even greater numbers of patients, carefully examining the drug’s side effects, effectiveness, and safety. A successful Phase III trial is the kind of result that drug companies need to get a drug approved by national regulatory agencies.
  3. In summary, we’re excited that the first human trial of a drug that targets the root cause of HD will start as early as next year*. This excitement is tempered by the realization that this first trial is all about safety, involves only a very small number of HD patients, and that many details of the study are still uncertain.
  4. With a $30 million investment in Isis’s preparations for the clinical trial and the prospect of additional future payments, Roche last year* acquired the rights to the drug. The deal allowed CHDI to switch to an advisory role as intended and enabled Roche to bring to bear its expertise in developing drugs and bringing them to the market. Because of the high cost of drug development, a small biotech firm such as Isis must partner with a drug manufacturer to get its remedies to patients.
*You’ll have to read the links to find the correct dates

A Field Guide to Working with Mouse Models of Huntington’s Disease

www.enroll-hd.org/participate

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Old 20-10-15, 12:35 AM   #48
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Default Re: Another Cautionary Tale About Breakthrough Cures

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Have to say I can't influence capitalism culture with regards to medical research funding, I've tried but failed! Where would we be without CHDI? Nowhere. No research, no trials, no hope because there are not enough of us for the drug companies to make a profit out off. Trapped in a never-ending cycle of despair and hd misery for generations past and to come.

Need to be able to look my loved ones in the face and say there is some hope. For the first time since we found out about this insidious disease I can do that....the light at the end of the tunnel is not just the on-coming train. I going to keep that little bit of positivity shining as long as I can.

32 (or 34 or 36) hd sufferers, thank you, for being pioneers.

M x
We all need hope. Ditto above. But thank you Allan for all of your research.. x
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Old 20-10-15, 10:30 AM   #49
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Default Re: Another Cautionary Tale About Breakthrough Cures

There are two sides to every argument/story..... two sides to every coin!! A balanced approach in all things HD related...... but, I too have to remain positive and have hope for my family - my second daughter as yet untested, my grandchildren and many others within our family. Hope was what I gave them when we first told them of HD and hope is what I will always continue to give them. My husband was going to participate in one of the research programmes a couple of years ago - a Phase 2 trial which I believe is still ongoing (but for other medical reasons was not able to in the end). It caused him great distress that he was not able to. Thank you to all those who do participate in trials and to those involved in research - however long it takes to get there - get there in the end we must, to end untold suffering to so many families.
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Old 21-10-15, 11:33 AM   #50
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Default Re: Another Cautionary Tale About Breakthrough Cures

At the risk of sounding like a stick in the mud....I read an article yesterday just published that says signs of changes in the brain begin decades before any physical symptoms start to show and these cannot be reversed. So, once these trials are completed and drugs licensed in say another 10-20 years how is testing for those at risk going to be tackled? Given all the misdiagnosis that has gone on for years, it means babies will need testing. And not just for those at risk....so many people are unaware that hd is in their family, so many gp's are clueless about hd. Hope is indeed a wonderful thing....but false hopes of anything turning up soon is just depressing.....Sorry if this sounds negative but I guess it's just the realist in me, and the selfish realisation that this ain't gonna help our situation or so many others on here. Patience is a virtue, but time is running out for soo many.
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