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August 31, 2004
Republican Convention - Day 1
Well..Day 1 of the convention and there was basically no discussion on health care or Medical Research.
There were a few 1-minute speeches that made references but that was about it. The rest of the week appears to be more of the same, unfortunately.
Posted by Dave at 11:01 PM | Comments (0) | TrackBack
August 30, 2004
Sarah Winckless Tells Her Story
I wish I had seen this sooner...Sarah Winckless, a Bronze medalist in the just concluded Olymnpics and HD positive, spoke to the London Daily Mail and HDSA has it posted on their website.
This is some of what she had to say:
It is not so much passing on the gene that worries me, because I belive that in 20 years time science will have found a treatment. But I am worried about being unable to care for my children. Neither John, nor Imy can remember a time when Mum was well - and the later I leave having children, the greater the risk that my children's experience will be the same.
With Huntigton's, there's no guarantee about age of onset. I know I'm fine now because I'm performing at this level- the very nature of Hutington's is that it affects coordination, so I know it hasn't started.
I can't fight it, so there's no point in fearing it. If it happens, it happens. I'm going to live my life and get on with it. The more positive I can be the better. That's what Mum has shown.
Some people don't last 10 years, and while every person is different, I really believe part of Mum's resiliece to the disease is her positive attitude and the fact that she keeps going.
For now, the only thing that matters is devoting my energies to fulfilling my potential on the water in Athens.
Mum, although confined to a wheelchair, is coming too. She's incredibly excited, even though she finds it hard to express herself. And if I do win a medal, she'll be the first person I give it to. She's done her bit. Now I've got to do mine."
I wish I had been there to see it ... what a remarkable family! Read the rest of what Sarah had to say.
Posted by Dave at 09:05 PM | Comments (0) | TrackBack
August 29, 2004
Eight Simple Rules
No TV show has done more for Huntington's Disease than the comedy "Eight Simple Rules".
The cast members are continuing to show their support for our community. This time it is for the fifth annual Millard Days Walk, Run & Bike for Mind and Body in Omaha, Nebraska.
Attending will be Billy Aaron Brown and Amy Davidson, co-stars on the hit ABC comedy "8 Simple Rules." Brown plays the boyfriend, "Kyle," of Davidson's character, "Kerry."
Brown, a Clarinda native, is a national spokesman for the Huntington's Disease Society of America. In addition to co-starring in "8 Simple Rules," he has also appeared in the feature film "Jeepers Creepers 2," the Disney Channel movie "Going to the Mat" and the Olsen twins movies "Holiday in the Sun" and "Getting There."
Davidson grew up in Phoenix and was introduced into an entertainment career through dancing. Since relocating to the Los Angeles area, she landed a recurring role on the ABC Family series "So Little Time," guest-starred on "Judging Amy" and appeared in the Lifetime movie "The Truth about Jane."
These two young actors will be in Omaha to help raise awareness and funds for Huntington's Disease...
Posted by Dave at 11:54 PM | Comments (0) | TrackBack
August 28, 2004
Better Late Than Never - Kerry's Speech
Due to an incredibily crazy schedule this month, I'm only now getting to writing about John Kerry's acceptance speech at the Democratic Convention.
Earlier this year I decided to cover the Presidential election from the standpoint of Huntington's Disease. At this point in the campaign I'm wondering if I didn't make a mistake in doing so. The country is strongly divided between the two candidates and health care & medical research have not received much attention in the national debate. Nevertheless, I'm prone to 'tilting at windmills' so I'm continuing this exercise until the general election.
So far in the campaign, of the two candidates, Kerry has been the only one bringing up the subject of health care. He's done this about once a month and virtually every time campaign or world circumstances have overwhelmed his message. His acceptance speech for the Democratic nomination is his best chance to put forward what is important to him and this is what he said about the two areas that would be of most importantce to the Huntington's Disease Community - health care and medical research:
"You don't value families by denying real prescription drug coverage to seniors, so big drug companies can get another windfall."
This is a knock on the current prescription drug benefits in Medicare. It's a convoluted mess, but an improvement from what we had before. Kerry seems to be arguing for more drug coverage which would be good for those in our community that qualify for Medicare. The comment about "big drug companies" and "windfall" is worrisome from a research funding standpoint as price controls tend to kill research. But not much can be read into that phrase without details.
"We believe in the family value expressed in one of the oldest Commandments: "Honor thy father and thy mother." As President, I will not privatize Social Security. I will not cut benefits. And together, we will make sure that senior citizens never have to cut their pills in half because they can't afford lifesaving medicine."
Privitization is HD neutral. The benefits/risks are dependent on the details. There is no current plan so there is no way to analyze positives or negatives. The promise to "not cut benefits" is a good one, though I've not heard of any plans from either party that would including cutting. The mention, again, that seniors would have a strong prescription benefit would aid the senior members of our community.
And that is the choice in this election.
You don't value families if you force them to take up a collection to buy body armor for a son or daughter in the service, if you deny veterans health care, or if you tell middle class families to wait for a tax cut, so that the wealthiest among us can get even more.
This sound good for our community in that he supports improving veterans health care. In this the two candidates are in agreement. Veteran health care spending has greatly increased under Bush. To know this would continue under Kerry is good.
What does it mean when Mary Ann Knowles, a woman with breast cancer I met in New Hampshire, had to keep working day after day right through her chemotherapy, no matter how sick she felt, because she was terrified of losing her family's health insurance?
As it turns out, this paragraph was a mistake for the Kerry campaign. I don't agree with the Union-Leader in that this was a lie, but it turns out this reference was VERY incorrect. Candidates rely on their campaigns to verify these stories for speeches, in this case they didn't do a good job. However, this error by Kerry's campaign did not have much affect on his national campaign. Kerry does show a sensitivity to health care coverage with this antedote.
What does it mean when 25 percent of the children in Harlem have asthma because of air pollution?
I'm including this because it relates to health. There wasn't any proposals or health care initiatives tied to this statement.
And we value health care that's affordable and accessible for all Americans.
Affordable is good. How he plans to acheive it is another matter. Hopefully not at the expense of research.
Since 2000, four million people have lost their health insurance. Millions more are struggling to afford it.
You know what's happening. Your premiums, your co-payments, your deductibles have all gone through the roof.
All true.
Our health care plan for a stronger America cracks down on the waste, greed, and abuse in our health care system and will save families up to $1,000 a year on their premiums. You'll get to pick your own doctor and patients and doctors, not insurance company bureaucrats, will make medical decisions. Under our plan, Medicare will negotiate lower drug prices for seniors.
I have not gone over his plan in detail, so I can't comment. I'm not sure how he would reduce "waste", "greed", and "abuse". As long as the cure isn't worse than the disease, this isn't bad. But this statement raises many questions (for another time).
And all Americans will be able to buy less expensive prescription drugs from countries like Canada.
This one is hurtful for our community. There is ample evidence that this would hurt funding for medical research - something our community desparately needs. For those diseases with effective treatments this is helpful.
The story of people struggling for health care is the story of so many Americans. But you know what, it's not the story of senators and members of Congress. Because we give ourselves great health care and you get the bill. Well, I'm here to say, your family's health care is just as important as any politician's in Washington, D.C.
To put it mildly, there's a logical problem with this statement unless he is arguing to take away the benefits of those in Congress.
And when I'm President, America will stop being the only advanced nation in the world which fails to understand that health care is not a privilege for the wealthy, the connected, and the elected it is a right for all Americans.
So every person would have health insurance? The problem is in the details but this, of course, sounds good.
What if we find a breakthrough to cure Parkinson's, diabetes, Alzheimer's and AIDs? What if we have a president who believes in science, so we can unleash the wonders of discovery like stem cell research to treat illness and save millions of lives?
This statement "believes in science" falls in the "cheap shot" category. Research spending, even stem cell research has greatly increased under Bush. However, it is apparent that Kerry would more broadly fund fetal stem cell research than Bush.
So that's it. Kerry makes several references to issues that are important to our community. Based on what the pundits are speculating we probably won't see as much of this out of Bush.
To sum up Kerry's comments and stands:
Health care coverage - Good. Want's everybody to be covered.
Health care costs - Discusses lowering, chance that it could hurt research
Medical research - Minimal mention, seems to be secondary to cost containment. I wish there had been discussion on expanding funding of NIH and helping private research.
Posted by Dave at 06:57 PM | Comments (0) | TrackBack
August 27, 2004
Killers Not Found Yet
If the lack of news is any guide, the killers of Jason Schwindler still have not been found. For those who didn't catch the earlier reports, Jason was shot and killed in an ambush while working for an armored car company. Jason was, by all accounts a loving husband to his wife who has Huntington's Disease and to his 13-month-old child.
The reward is at $125,000.
Posted by Dave at 11:12 PM | Comments (0) | TrackBack
August 25, 2004
More On Miraxion
HD Lighthouse has also posted the information from yesterday concerning Miraxion. It's the same press release but go there and read Dr. Marsha Miller's intoduction. Her last comment is one of the best:
The possibility that Miraxion will help people with higher CAG counts is not ruled out yet by any means. I know of nothing that occurs in this disease at 45 or more repeats that doesn't occur at less than 45 repeats so it may well be a problem of timing. In other words, it may well be that for those with the higher numbers of repeats, the therapeutic potential comes at an earlier stage of the disease or in delaying onset.
Posted by Dave at 08:12 PM | Comments (0) | TrackBack
August 24, 2004
Great News! 23% Improvement
As you may have noticed I've been a bit critical of Amarin in the past, and for good reason. However, the new management seems to be making up for the problems of the past.
In what appears to be in preparation for the new round of Phase III trials for Miraxion (LAX-101), Amarin broke down the numbers from the last Phase III trials. Here's what they found (vastly over-simplified version):
If you were a member of their last study and you took your meds the way you were supposed to, on average you would have improved on your test scores (TMS-4) over the course of a year by nearly 23%! If you took the placebo your scores would have declined almost 6%! These results are statistically significant.
Miraxion (LAX-101) is not available from your pharmacist but you can still take the equivalent. Miraxion is purified EPA, one of the anti-oxidants in fish oil. If you take enough fish oil so that you are getting at least 2 grams (2000mg) of EPA then you are taking the equivalent of the dosage being used in these studies.
This may help you if your CAG is over 45, but if your count is under 45 this could keep you healthy for much longer. Perhaps long enough to enjoy a potential cure to HD such as RNAi therapy which appears to be a few years away.
Read your labels, consult with your doctor, take your medications/fish oil faithfully and you might just protect yourself. If you haven't yet, start today. You have nothing to lose and perhaps the rest of your life to gain!
Here's Amarin's press release:
Analysis Identifies Characteristics of Huntington's Disease Patients Responsive to Miraxion(TM)
LONDON, August 24 -- Amarin Corporation plc (NASDAQSC: AMRN) today announced the results of a gene variant data analysis from the initial Phase III clinical trial for Miraxion(TM) (formerly referred to as LAX-101) in Huntington's disease. The analysis identifies a group of patients with a specific gene variant that experienced a significant response to Miraxion(TM). The initial twelve month, phase III multi-center, double blind, randomized placebo controlled study of Miraxion(TM) was conducted in 2002 by Laxdale Limited ("Laxdale") with 135 enrolled patients with Huntington's disease at six centers in the United States, Canada, the United Kingdom and Australia. The primary endpoint in the initial trial was the change over a one-year period in the Total Motor Score-4 (TMS-4) subscale of the Unified Huntington's Disease Rating Scale (UHDRS), the standard rating scale for trials in this disease.
The additional data analysis identifies a group of participants in the initial study for whom Miraxion(TM) showed a significant clinical benefit. Huntington's disease is believed to be caused by a genetic mutation of the cytosine, adenosine and guanine (CAG) polymorphic trinucleotide repeat. It is believed that there is a direct link between CAG repeat length and age of onset, disease progression and clinical symptoms of Huntington's disease. CAG repeat length can be measured via a genetic blood test.
Efficacy of Miraxion(TM)
The possibility that treatment efficacy of Miraxion(TM) could be related to the number of CAG repeats was proposed as part of the pre-specified analysis and exploratory analysis was conducted after completion of the trial to examine this influence. A strong correlation between the CAG repeat number and the change in TMS-4 score was revealed in those patients taking Miraxion(TM). In order to explore this effect further, patients were split into two groups based around the median number of CAG repeats, which was identified as 45. Those patients that took Miraxion(TM) and had a CAG repeat length of less than 45 comprised the responsive group. This effect was consistent across all centers. In total, 67 of the 135 patients in the initial phase III study had this specific gene variant. It is estimated that patients with a CAG repeat length of less than 45 represent over 65% of all Huntington's disease patients.
The group of patients with a CAG repeat length of less than 45 in the intent to treat group receiving Miraxion(TM) showed a statistically significant improvement over those patients receiving placebo (p=0.029, n=67). In the group of patients with a CAG repeat length of less than 45 who observed the clinical trial protocol (per protocol patients) receiving Miraxion(TM) showed a 22.7% improvement in TMS-4 score versus patients receving placebo who showed a 5.7% deterioration at the end of the twelve month study (p=0.006, n=44). This improvement was observed over a 6-month period and maintained for a further 6 months. In the 12-month open label continuation phase of the trial, this improvement continued to be maintained. At the end of the open-label phase all patients who had taken part in the trial were offered compassionate supply. More than 3 years after the commencement of this trial, 101 of the 135 patients enrolled in the trial continue to be supplied Miraxion(TM).
Rick Stewart, Chief Executive Officer of Amarin commented, "This data analysis, showing significant clinical benefit to specific patients taking Miraxion(TM), will be an important component in the design of the planned phase III clinical trials. It will allow us to more accurately target patients with this specific gene variant, particularly relating to age of onset of the disease. Improvement and stability are particularly beneficial if initiated at an early stage in this devastating disease. This could significantly extend a patient's professional activities, maintain a good quality of life, and potentially defer the onset of the later stages of the disease."
Planned Phase III Trials
Amarin intends to commence two phase III clinical trials totaling over 400 Huntington's disease patients in early 2005, subject to consummation of Amarin's proposed acquisition of Laxdale and the completion of an equity fund raising.
Miraxion(TM) has been granted Fast Track designation for Huntington's disease by the United States Food and Drug Administration and received Orphan Drug designation both in the U.S. and in Europe. Miraxion(TM) is also in clinical development for depression.
Huntington's Disease
Huntington's disease is a genetic neurodegenerative disease characterized by movement disorder, dementia and psychiatric disturbance. It has been diagnosed in approximately 30,000 patients in the U.S. with a similar number in Europe. Additionally, over 200,000 persons in the U.S. alone are genetically "at risk" to developing the disease. Onset of symptoms is typically between 30-50 years of age with a typical life expectancy from diagnosis of 10-25 years. Patients with late stage disease require continuous nursing care, often in nursing homes, with an estimated annual cost to the U.S. economy of up to $2.5 billion. Presently, there is no effective treatment or cure for HD.
About Amarin Corporation
Amarin Corporation is a neuroscience company focused on the development and commercialisation of novel drugs for the treatment of neurological disorders affecting the central nervous system.
For press releases and other corporate information, visit our website at http://www.amarincorp.com.
Statements in this press release that are not historical facts are forward-looking statements that involve risks and uncertainties which may cause the Company's actual results in future periods to be materially different from any performance suggested herein. Such risks and uncertainties include, without limitation, the uncertainty of entering into and consummating a definitive agreement on terms acceptable to the parties, the inherent uncertainty of pharmaceutical research, product development and commercialization, the impact of competitive products and patents, as well as other risks and uncertainties detailed from time to time in periodic reports. For more information, please refer to Amarin Corporation's Annual Report for 2003 on Form 20-F and its Form 6-Ks as filed with the U.S. Securities and Exchange Commission. The company assumes no obligation to update information on its expectations.
Posted by Dave at 11:38 PM | Comments (0) | TrackBack
August 23, 2004
Flies, Worms, Mice and now...Sheep?
New Zealand researchers have won approval to create the world's first flock of sheep that will be genetically modified to get Huntington's disease.
... The research is financed by $500,000 over eight years from New Zealand Freemasons and just over $500,000 over three years from the US-based Hereditary Disease Foundation. ...
"If anyone has any doubt about whether we should use animal models, just talk to a family. We received a brain ... from a family in Nelson whose loved one died at 44. We had another brain from that same family 11 years ago." ...
Freemasons spokesman Terry Meekan said the Masons approached Auckland University after one of their members spoke about the effect of the disease on his family.
Posted by Dave at 08:57 PM | Comments (0) | TrackBack
August 22, 2004
Olympic Medal! - pHD Stikes Bronze!
Yes folks! Life isn't over if you have the gene for Huntington's!
Sarah Winckless and her partner, Elise Laverick, earned a Bronze Medal in the 2004 Olympic Games! From the London Times:
"The day’s other medal went to Sarah Winckless and Elise Laverick in the double sculls, a success that was all the better for being so unexpected. Fourth at halfway, Winckless and Laverick overtook the Bulgarian crew for their well-deserved reward. By nightfall, the British squad lay proudly on top of the medals table for Olympic rowing.
Posted by Dave at 10:18 PM | Comments (0) | TrackBack
As if...
One of the true pleasures in life is reading some of the personal stories posted on HDAC.org.
We'll they've got a bunch of new updates (again!) Be sure to take the time to read them. I want to thank those who take the time to write these very personal stories. They do make a difference...Thank you!
Be sure to read "To Live 'As If' - My Story" which is a first in what looks to be a very promising series!
Also, in an all too familiar story, but one that ends with triumph... "The Test" by Suzanne Kengla. The editor's note on Suzanne reads: Suzanne is an amazing HD warrior. She provides tireless love and support for her siblings with HD and she is a top fundraiser at the annual Arlo Guthrie's Historic Garbage Trail Walk to Massacree.
We are a community of survivors. One of the many reasons our community is special and valuable.
Posted by Dave at 09:41 PM | Comments (0) | TrackBack
August 21, 2004
Republican Convention Lineup
I just saw the speaker lineup for the Republican Convention. It doesn't look like there'll be much, if any, focus on medical research & health care. That would be sad if that were the case.
Oh...I know I haven't written on Kerry's acceptance speech yet (as I had promised) and that it's long overdue. It will be coming up shortly.
Posted by Dave at 11:27 PM | Comments (0) | TrackBack
August 20, 2004
$125,000
That's the award being offered for the robbers that killed Jason Schwindler, a loving man with a pHD wife and 13-month-old son.
He's worth more than that but, still, an impressive award. Let's hope somebody gets to spend that money - soon.
Posted by Dave at 12:05 AM | Comments (0) | TrackBack
Bronze, Silver?
The Guardian comments on Sarah Winckless's Olympic quest:
Elise Laverick and Sarah Winckless have shown commendable coolness in the women's double this season, and it may be between them and Bulgaria for the bronze. The two Britons have improved with each race and, if Winckless times her finishing burst to perfection, even the silver may be there for the taking.
Posted by Dave at 12:03 AM | Comments (0) | TrackBack
August 19, 2004
How To Kill Another Generation
"(D)o we buy the drug companies' arguments"?
That's part of a comment posted by John in response what I wrote on drug reimportation. His thoughtful & considerate post deserves a response. Here it is:
How about the arguments of economists, investors, or researchers? They'll tell tell you the same thing. If an argument is valid then "yes" I would 'buy' the argument. To do otherwise would be foolish.
The question he posed "...do we buy the drug companies' arguments about price controls limiting research, or do we worry about being able to afford the new medications if they ever discover them?" is based on a faulty premise.
Simply put, if research is limited due to price controls then the new medications are not available at ANY PRICE.
The next sentence is a beaut! ... "A balance is necessary, and the reality in politics is that American voters are only going to stand for footing the bill for the rest of the world's research for so long."
How on God's green earth would artificially forcing lower prices create "balance"? And to whom? What is the price in human life are we will have to pay to achieve this "balance"?
When governments artificially force a lower price of a good, the supply drops. If an investment provides a lower return without lowering the risk, the money will flow elsewhere. This means people in the Huntington's Disease community will continue to see our loved ones die as research is postponed due to lack of funding. That's Econ 101.
The next argument made (very politely) is especially absurd.(Sorry. I'm sure he's a very nice & caring person, but it is an absurd argument.) The US is doing the lions share of the medical research in the world because, ironically, we DON'T put the artifical price limits on the fruits of their labors. If you don't believe me, ask the German (and other) researchers who now work in the United States.
Based on the links provided in the writer's comments, he believes the best response is to also have the rest of America pay for the drugs in Illinois and New Hampshire? This is bringing "balance" to us paying for the bulk of the worlds medical research? It's not.
I have a question for those with this argument... wouldn't the whole WORLD prosper if activists instead pushed other countries to lift their restrictions on their own pharmaceutial & genetic research companies? If they asked their countries to increase spending on medical research?
If this was done then all the countries would bear their fair share of the research needs of the world. The results? Research would increase, lives would be saved, and the costs would be distributed more fairly. But that isn't what is happening, Instead we are being asked to kill our research industry, just as it has been killed in other countries that took this course.
Don't expect me to gladly watch the miniscule research funds we have dry up for our 'orphan' disease. I'm tired of seeing our loved ones die! A cure for Huntington's Disease is now within sight but selfishness ("I want my state to pay less") and economic ignorance could ripe that away from us and our community can then watch another generation die.
One more thing...there is an economic cost paid when price controls are instituted. Usually, this cost is higher to the country as a whole than what is saved (that's a whole 'nother book).
Here's some more food for thought...excerpts from a speech given at the Keck Graduate Institute of Applied Life Sciences on this very subject:
(...The result: The country that is now responsible for the vast majority of new drug launches each year will go the way of, say, Germany, which, with few exceptions, is no longer a serious player in developing new drugs.
Research by Carmelo Giaccotto and colleagues, published by the AEI-Brookings Joint Center for Regulatory Studies, estimated that, if price controls had been in effect in the United States between 1981 and 2002, there would be between 330 and 365 fewer new medicines on the market today.
Research by John Vernon concluded that price controls applied in the U.S. today would lead to a cutback in new medicines by two-thirds within 50 years.
The economic development argument is powerful. The Bain study shows that, while Germany saves $19 billion in drug costs through price controls, it loses $22 billion due to reduced R&D spending, bad health outcomes, loss of high value-added jobs (and the tax revenue associated with them), and so on. Free riding isn't free.
How to get drug prices even lower? Simple. Increase supply. If Europe ended price controls, more companies would enter the market, pushing down prices. If the FDA streamlined its standards, costs of bringing new drugs to market would fall, increasing supply. If property rights were protected, the cost of capital for investment in new drugs would fall, increasing supply. That is the answer.
Posted by Dave at 11:47 PM | Comments (0) | TrackBack
August 18, 2004
Winckless - Olympic Finals!
Sarah Winkless and her crewmate Elise Laverick have made it to the Olympic finals of the 'double sculls' rowing event. The finals will be held on Sunday and they have a very good chance at getting a medal.
Sarah is a great example to the world of what a person with the Huntington's gene can accomplish. (Now if we can only ban discrimination of those who've tested positive.)
Here's what one article had to say on their performance:
However, the British women's team now have three finalists with good medal chances, thanks to brilliant repêchages by Sarah Winckless and Elise Laverick in the double sculls and Katherine Grainger and Cath Bishop in the pairs.
Winckless and Laverick were a couple of seconds behind the Romanians Camelia Mihalcea and Simona Strimbeschi with three-quarters of the way gone, and then swung into a stroke that took them to victory by three seconds.
Ain't it great!
Posted by Dave at 06:58 PM | Comments (0) | TrackBack
This Isn't Good
"(Kevin) Benham is accused of kidnapping the 27-year-old woman as she took a jog on a dirt road near Fort Tuthill. A man had forced her into the trunk of his red car.
She managed to escape by kicking out a rear seat and jumping out a rear door as the man who kidnapped her drove south on Highway 89A. She was injured in the escape and was treated at the hospital."
As if the story is bad enough, here's the kicker:
The 27-year-old, Kevin Benham has tested positive for the HD gene. Since he confessed to his parents and a case worker, his lawyer is suggesting he'll use the diagnosis in his defense.
The last thing the Huntington's Disease community needs a case like this to stigmatize the entire community.
Fortunately, this isn't national news and the defense will probably seek a plea bargain. Let's hope this is the last we hear of this.
Posted by Dave at 06:34 PM | Comments (2) | TrackBack
August 17, 2004
More On Winckless
A little more Sarah Winkless, the HD gene positive British sculler who is participating in the Olympics. From The Independent:
But the British women, both veterans of the Sydney team, are strong contenders for the other medals.
Winckless is a Cambridge graduate who combines consultancy in marketing with rowing, while Laverick is a master of the double bass as well as the double scull, having studied at the Guildhall School of Music and Drama.
The versatility of the crews was shown at the Munich round of the World Cup when Winckless substituted for Houghton in the quad as well as doing her own event, and won silver medals in both.
The coaches have honed good technique with strong motivation. "We always knew it was in all our interests to bring our single sculling technique as close to the crew boats as we could," Winckless says. "We had three camps in Seville without single boats, so we all rowed with everyone, and that's something that's come very much from the top and has been very much pushed."
She'll be back at it tomorrow and I'll post the results.
Posted by Dave at 09:53 PM | Comments (0) | TrackBack
August 16, 2004
Olympian pHD Update
I'm looking for more news, but so far it appears that Sarah Winckless is having a good Olympics.
Sarah is a rower for England who has tested positive for the Huntington's gene. She is competing in the double sculls event. Here's a bit on her performance on the 14th:
However, arguably the most impressive British performance arrived in the women’s double sculls where Sarah Winckless and Elise Laverick threatened to upset New Zealander twins Georgina Evers-Swindell and Caroline Evers-Swindell, but ultimately settled for safe passage into the next stage.
Posted by Dave at 11:20 PM | Comments (0) | TrackBack
August 15, 2004
Gene Therapy Stops Cell Death
This is interesting. Scientists have demonstrated that they can prevent the type of cell death that is caused by Huntington's Disease (and others) with a gene therapy technique.
This research could impact many different diseases. Is this a gene therapy technique that could work for Huntington's Disease? Possibly, but this technique is more broad than one that specifically targets HD. It is likely to have a higher chance of having unacceptable side-effects. The recent successes in gene therapy specifically for Huntington's Disease look more promising. Still, this is important research that gives more information on how cells are induced to die.
The abstract:
DNA Cell Biol. 2004 Aug;23(8):496-501.
Gene Transfer of Baculoviral p35 by Adenoviral Vector Protects Human Cerebral Neurons from Apoptosis.
Miagkov A, Turchan J, Nath A, Drachman DB.
Department of Neurology, Johns Hopkins School of Medicine, Baltimore, Maryland.
Apoptosis plays an important role in neuronal cell death in both chronic and acute human neurological diseases, including ALS, Huntington's disease, cerebral ischemia, and HIV encephalopathy. We evaluated the ability of an extremely powerful antiapoptotic agent, baculoviral p35, to prevent apoptosis and cell death of human cerebral neurons that undergo severe neurotoxic changes in a culture system when treated with agents that are implicated in human neurological disorders, that is, tumor necrosis factor (TNFalpha) and the HIV proteins Tat and gp120. P35 is a potent broad-spectrum antiapoptotic protein derived from baculovirus, that inhibits nearly all caspases, and has other antiapoptotic actions as well. Adenoviral vectors expressing p35 (Ad. p35) or a control gene (lacZ) efficiently transduced human neurons. Treatment of control cultures with the toxic agents TNFalpha, TNFalpha plus Actinomycin D, or Tat and gp120, induced neurotoxicity and death of neurons. Transduction of neurons with Ad. p35 blocked apoptosis, and eliminated cell death due to TNFalpha, or Tat and gp120. Viral vector transfer of the p35 gene efficiently protects human neurons from TNFalpha, or Tat and gp120-induced apoptosis and cell death. These results suggest that p35 transduction of neurons by viral vectors could be therapeutically useful in the treatment of human neurodegenerative diseases.
PMID: 15307952
Posted by Dave at 11:20 PM | Comments (0) | TrackBack
August 14, 2004
Reasons For Hope
Dr. Marsha Miller has a wonderful article on the HDAC.org website.
The article is called "Reasons For Hope - 2004" and she's right...there is hope.
Read the whole article here. Here's an excerpt:
Last time I pointed to RNA interference therapy as the most promising potential cure for Huntington’s Disease. The idea here is to bypass the attempt to alter the gene itself and instead block the instructions to make the HD protein itself, letting the normal huntingtin’s gene take over.
This line of research has made significant progress since I last wrote on “Hope”. Researchers can now ‘cure’ HD in a test tube and cure a similar triplet repeat neurodegenerative disorder in mice using this technique. Dr. Beverly Davidson, the lead researcher in RNAi, estimates that there will be clinical trials for people within five years.
Philip Sharp, a Nobel laureate at the Massachusetts Institute of Technology, was quoted in the British newspaper, the Independent as saying, “The broader science of RNAi is spectacular. It’s just absolutely spectacular. This is not hype. The biggest science prizes in the world will fall to RNAi.”
In the meantime, there has been an interesting convergence between HD and cancer research. There is a lot of money for cancer research and these drugs are already being tested with cancer patients, so more will be known about these potential treatments and they would be ready for approval a lot sooner if found to be effective.
HDAC inhibitors are being tested for use with cancer patients. They have also been found to arrest neurodegeneration in fruitflies and delay onset, lengthen survival times, reduce oxidative stress in the neurons, and improve both body weight and motor performance in an HD mouse model compared to untreated mice.
Geldanamycin, an anti-tumor agent, has been found to activate a heat shock response and inhibit huntingtin aggregation in a cell culture model of Huntington's disease and to rescue fruitflies from neurodegeneration.
Rapamycin, in Phase II clinical trials for cancer patients, has been found to lengthen survival times and reduce aggregate formation in an HD cell model, arrest neurodegeneration in a fruitfly model, and improved the behavioral performance of a model of HD mice compared to untreated mice.
Posted by Dave at 07:05 PM | Comments (0) | TrackBack
August 13, 2004
Kerry & Health Care Spending
One item I haven't been able to find, until now, is how much Kerry is intending to spend on health care. He's made several proposals, but the press has been short in providing details.
It appears his plans would increase spending on health care by $900 billion over the next 10 years. One concern about the source of this information...it comes from the American Enterprise Institute which is a right-leaning think-tank. To put it mildly, they aren't the most supportive of Kerry. Actually, they are downright hostile so the numbers may be inflated. They do provide sources for their numbers, but I haven't verified them. So use your own judgment on the accuracy. I didn't see anything on medical research (other than AIDS), which is something I'd like to see the candidates address.
Here's their breakdown on Kerry's health care plans (Section B: Community Living would appear to have a very positive effect on the HD community.)
II.8 Health Care
A. AIDS Research: “As president, John Kerry will increase AIDS research funding at the National Institutes of Health, the Center for Disease Control and other agencies.”
Source: “The Kerry-Edwards Plan to Respond to the AIDS Crisis,” www.johnkerry.com
10-Year Cost: $1.768 billion
Source: The NTUF estimates that this proposal would cost $160 million in its first year based on the difference between the $240 million increase requested by the AIDS community for NIH in FY2005 and the Bush administration’s proposed FY2005 funding increase of $80 million (AIDS Action, 2/11/04). Starting with this amount and adjusting it for inflation over ten years gives a total cost of $1.768 billion.
B. Community Living: Enact MiCASSA and the Money Follows the Person Act to promote the provision of community-based health support services.
Source: “Freedom, Independence, and Choices for Americans with Disabilities.”
10-Year Cost: $4.151 billion
Source: In the 108th Congress, Kerry co-sponsored MiCASSA (S. 971) and the Money Follows the Person Act (S. 1394). MiCASSA authorized $50 million a year for three years to provide grants to help states improve community-based services, and the Money Follows the Person Act appropriated $350 million a year for 5 years to fund state demonstration projects. Assuming that these programs are extended for ten years with adjustments for inflation, the total cost of enacting these two bills is $4.151 billion. (Note: This is an addition to the NTUF study.)
C. Drug Treatment: Increase funding for “treatment on demand” programs.
Source: National Taxpayers Union Foundation (NTUF Policy Paper 153, 7/12/04, www.ntu.org)
10-Year Cost: $1.292 billion
Source: The NTUF bases its estimate on a similar bill in the 107th Congress (S. 160) that authorized $125 million a year for five years to be appropriated for alternative treatment programs. Assuming full funding and extending the program for ten years by adjusting the fifth-year cost estimate at the rate of inflation, the 10-year cost of Kerry’s proposal is $1.292 billion.
D. Health Care Plan: Kerry’s comprehensive health care plan will extend coverage to 95 percent of Americans, including providing health care coverage for every child in America. This plan includes tax credits for small businesses, laid-off workers, and early retirees to purchase health care coverage.
Source: “Health Care,” www.johnkerry.com
10-Year Cost: $849.5 billion
Source: Kerry estimates that his entire health care plan will cost $653 billion over ten years, including $177 billion for the tax cuts (“John Kerry’s Framework to Cut the Deficit in Half and Invest in Affordable Health Care and Better Schools,” 4/7/04 Kerry press release, www.johnkerry.com). Removing questionable cost savings from disease management and information technology included in Thorpe’s estimates of the Kerry plan puts the cost of the plan at $849.5 billion over ten years (“Federal Costs and Savings Associated with Senator Kerry’s Health Care Plan,” Kenneth E. Thorpe, 8/2/2004, available here).
E. Immigrant Health Care: Restore health care benefits to legal immigrants.
Source: National Taxpayers Union Foundation (NTUF Policy Paper 153, 7/12/04, www.ntu.org)
10-Year Cost: $11.606 billion
Source: The NTUF estimates the cost of this proposal from the projected savings from the Personal Responsibility and Work Opportunity Act of 1996, which restricted Medicaid benefits for legal immigrants (NTUF Policy Paper 153, 7/12/04, www.ntu.org). In 1996, the Congressional Budget Office estimated that this law would save $5.290 billion over 6 years. Extending the law to ten years by growing the sixth-year estimate at the rate of inflation gives a ten-year savings estimate of $11.606; undoing the law would thus cost an estimated $11.606 billion over ten years (www.cbo.gov).
F. Minority AIDS Initiative: “John Kerry supports the highest level of funding for the Minority HIV/AIDS Initiative (MHAI) which assists in community efforts to improve the public health infrastructure to better respond to the epidemic.”
Source: “The Kerry-Edwards Plan to Respond to the AIDS Crisis,” www.johnkerry.com
10-Year Cost: $2.221 billion
Source: The NTUF estimates that this proposal would cost $201 million in its first year based on the difference between the $204 million increase requested by the AIDS community for MHAI in FY2005 and the Bush administration’s proposed FY2005 funding increase of $3 million (AIDS Action, 2/11/04). Starting with this amount and adjusting it for inflation over ten years gives a total cost of $2.221 billion.
G. Native American Health Services: “The Indian Health Service is severely under funded, and Senator Kerry supports meaningful increases for this vital means of providing health care to Native communities.”
Source: “Ensuring Tribal Sovereignty and Working to Improve the Lives of Native Americans,” www.johnkerry.com
10-Year Cost: $37.571 billion
Source: The NTUF uses Senator Tom Daschle’s estimate that $5.5 billion is necessary for full funding in FY2005, which is $3.4 billion greater than the Bush administration’s proposed FY2005 funding (daschle.senate.gov/sd_nativeAm_budget.html). Starting with this $3.4 billion increase and adjusting it for inflation over ten years, the total cost of Kerry’s spending increase is $37.571 billion.
H. Ryan White Act: “As president, John Kerry will increase funding for the Ryan White CARE Act.”
Source: “The Kerry-Edwards Plan to Respond to the AIDS Crisis,” www.johnkerry.com
10-Year Cost: $3.138 billion
Source: The NTUF estimates that this proposal would cost $284 million in its first year based on the difference between the $319 million increase requested by the AIDS community for the Ryan White CARE Act in FY2005 and the Bush administration’s proposed FY2005 funding increase of $35 million (AIDS Action, 2/11/04). Starting with this amount and adjusting it for inflation over ten years gives a total cost of $3.138 billion.
I. Substance Abuse Treatment: Enact the Child Protection/Alcohol and Drug Partnership Act.
Source: National Taxpayers Union Foundation (NTUF Policy Paper 153, 7/12/04, www.ntu.org)
10-Year Cost: $4.970 billion
Source: This bill from the 107th Congress (H.R. 1909) authorizes appropriations of $200 million, $275 million, $375 million, $475 million, and $575 million in its first five years, respectively. Assuming full funding of the bill and a 2.2% inflation rate after the fifth year, the 10-year cost is $4.970 billion.
Posted by Dave at 08:42 AM | Comments (0) | TrackBack
August 12, 2004
One Paper Gets It Right
I thought I'd written my last word the subject but, once again, I was wrong.
As those who've read this blog for awhile know, I've railed against the media outlets that don't get the facts correct when it comes to stem-cell research. I'm tired of talking about it. The same reporters that were science-illiterate yesterday will be that way tomorrow (especially if the facts get in the way of a good story). It doesn't make sense to keep talking about. Well...I was surprised to see this today:
One paper seems to have actually gotten the facts straight (though it is a little too kind to a candidate's wife.) The paper that got it right?...
The Wall Street Journal.
Go figure. Don't they just cover the stock market? Anyway, they at least understand the difference between "stem cell" and "embryonic stem cell". They also understand that the argument is over funding, not legal restictions. The article makes one good contribution to the discussion and it is this chart:
|
What Funding Ban? Amount spent by the National Institutes of Health on stem cell research, in millions.
|
Spending on stem cell research has grown 57% in the last two years. Embryonic stem cell research has grown 341% in the last two years.
Some "ban". We should all be so lucky.
You know what the sad thing is? The biggest bang for the buck is in RNAi (siRNA). That's a technology that is further along, has far more potential, and will eventually save millions of lives.
We need a candidate that will promise (and deliver) major funding increases in this (RNAi) technology. I'd vote for him/her in a heartbeat (if not sooner).
P.S. A fraction of this money would cure Huntington's Disease.
Posted by Dave at 07:08 PM | Comments (2) | TrackBack
Starfish Gene
Check out this article from the New Zealand Herald. Scientists are using a gene from the starfish in an attempt to regenerate cells.
The lead of the story points to the goal of regenerating limbs, but look at this little piece at the end of the article:
Auckland University Professor Garth Cooper said New Zealand researchers hoped to use similar techniques to regenerate brain cells in patients with degenerative conditions such as Huntington's disease.
It never ceases to amaze the growth in medical research.
Posted by Dave at 06:36 PM | Comments (0) | TrackBack
August 11, 2004
Another Biker For HD
18-year-old Tom Andrews is bicycling 1000 miles over a two week period as a fundraiser. Part of what he raises will go the Huntington's Disease Association (England). You can read all about it here.
You have to admire these riders who take two and three week rides to raise money and awareness for Huntington's Disease. Marie, Boyd, and Tom...thanks!
Posted by Dave at 10:06 PM | Comments (0) | TrackBack
August 10, 2004
Health Care Debate Sidelined Again
This will get buried in the news, and it happens to every candidate at one time or another, but...this doesn't help the health care debate.
Posted by Dave at 09:39 PM | Comments (2) | TrackBack
Shooting Update
Police are still looking for the three men that shot Jason Schwindler (See here.) As you may recall, Jason's wife has Huntington's Disease. Their 13-month-old son is at risk for this disease.
Best wishes to the entire family.
Posted by Dave at 07:56 PM | Comments (1) | TrackBack
Biker For Huntington's
Boyd Runnings has drawn some media coverage on his 3 1/2 week, 500-mile ride for Huntington's Disease. (More here.) He appeared on the local news at this CBS affiliate in Grand Island, Nebraska. Here's a bit from the online article:
He says he felt a guilt that he was lucky enough not to get the disease, through that feeling he decided to do something for those who did have Huntington's. "I decided to get awareness out there; there are a lot of people without support groups."
And with no cure and not treatment a support group is all many people have, something Runnings wants to change.
"If we can get rid of Alzheimer’s or Huntington's or Parkinson’s or Lou Gehrig's if we could knock one down we could knock all of them down because they're so similar."
He's right, Huntington's Disease research is benefiting the research of these other diseases.
Posted by Dave at 07:44 AM | Comments (0) | TrackBack
August 09, 2004
3rd Anniversary
I know, I know...you're tired of hearing about stem cell research.
Today is the 3rd anniversary of President Bush's announcement that the federal government will only fund research of embryonic stem cells from existing cell lines.
The Kerry campaign was hoping to 'make some hay' on this (as with the poorly worded weekly address) but they are being sidelined (once again) by other news. It looks like the news of this week will be Kerry's apparent lie that he spent Christmas, 1968 in Cambodia. This will be the third or fourth time that health care was to be the topic-of-the-week for the campaign only to be sidelined by other events.
Maybe next month the public debate will shift to health care and medical research.
Posted by Dave at 07:41 PM | Comments (0) | TrackBack
August 08, 2004
pHD's Husband Slain
Today's Washington Post has a sad story of Chrissy Schwindler, a woman with Huntington's disease and 13-month-old baby, whose husband was killed while working for an armored car company. Her husband, Jason Schwindler, was ambushed outside of a bank and shot three times.
He sounds like he was a wonderful husband, some excerpts:
Swindler knew his wife's condition was "only going to go downhill," Randy Clemens said. (Chrissy declined an interview request.) "But he didn't leave. He stuck with her. . . . His whole world revolved around Chrissy and Nate."
When she developed the random compulsions that often accompany the disease -- in her case, a yearning for Starbucks coffee -- he would take her to get coffee before and after work. Just to make her smile, Clemens said.
Nathan would run to the door clamoring for his father when he came home, looking forward to wrestling and playing trucks. The little boy doesn't yet understand what happened, said Chrissy's mother, Brenda Clemens.
"I don't think he realizes it," she said. "We showed him a picture of his daddy, and he started smiling and laughing."
Posted by Dave at 04:44 PM | Comments (1) | TrackBack
August 07, 2004
Stem Cell Slogans
Anne Applebaum gets it right in this column from the Times-Union...
"I'd still like it better if debates about science began with facts."
Here, here! In today's Democratic response to the President's Saturday address, John Kerry embarrassed himself with this statement:
"At this very moment, some of the most pioneering cures and treatments are right at our fingertips, but because of the stem cell ban, they remain beyond our reach..."
I say 'embarrassed' because Kerry knows there is no stem cell ban, just a limit on the federal funding of embryonic stem cells. Also, while adult stem cell research is making great progress, embryonic stem cells are light years from offering "cures and treatments". This is pandering at its worst.
For a more accurate take, read this from the Applebaum column:
Stem cell research is not, in fact, either illegal or unfunded: The federal budget in 2003 included $24.8 million for human embryonic stem cell research -- up from zero in 2000.
Private funding of stem cell research, which is unlimited, runs into the tens and possibly hundreds of millions of dollars. The current, admittedly hairsplitting, policy came about because Congress in 1995 passed a ban on federal (but not private) funding for any form of research that involved the destruction of human embryos, because it is a form of research many American voters dislike and don't want to pay for. After some important (privately funded) breakthroughs, the Clinton administration began looking for legal ways to bypass the ban, but never got around to paying for any actual research.
The Bush administration thought about it, too, and came up with a solution: Federal funding could be used for research on stem cell lines already in existence. In practice, this means scientists who get their funding from the government are restricted in which materials they can use.
Although this compromise will soon become a real obstacle to research, for the moment the irritant is largely philosophical. "What hampers people is the concept that there is a lack of freedom to operate," one scientist told me.
UPDATE: Tech Central Station has posted an interesting commentary also taking Kerry to task for his comments in the Saturday address. (Such as calling the funding limitation a "far-reaching ban".) The commentator also poses the question about whether this issue may cause Bush to lose the election.
Posted by Dave at 10:16 PM | Comments (0) | TrackBack
August 06, 2004
Oops
Everybody makes mistakes, but I'm an expert at it!
I made (another) one a little while back. Michael Fumento had written an article on Ron Reagan's speech at the Democratic convention. After some internal debate I decided to write a piece on it and I had these 'nice things' to say about Michael Fumento's article: "heavy-handed in his criticism of Ron Reagan" and I said that it "isn't true" that embryonic stem cells had never been used in human testing. I even referenced a study as proof. (As I'm sure you know, anybody who references a study is always right!)
Well, Mr. Fumento read what I had to say and he had some choice words to say to me...
And they were all pleasant and polite. If you read the comments on that posting you'll see he wrote:
"Thanks for the mention, but I'm right even on that one part. The abstract you link to explicitly refers to "fetal neural tissue," which is exactly that. It is not embryonic stem cells. A fetus by definition has developed past the embryonic stage and contains no ESC cells.
Best,
Mike Fumento"
And, yes, he's right. I missed the distinction, which in an important one in this discussion. Not only is he a good writer, he's nice also. Where would the world be if everybody was like this? Probably much more pleasant with skinnier newspapers.
I guess I better buy his book.
Posted by Dave at 08:48 PM | Comments (0) | TrackBack
August 05, 2004
Health Insurance Scams
I saw this in my old home town paper...health insurance scams.
Yep, there are people out there selling "health insurance" that isn't. They collect the premiums and don't ever pay for medical care. Even in the rural and lightly populated state of Montana they have cease & decist orders against 15 different companies. They are investigating 45 more. According to the article, here's what to look for:
According to Elliott, the offers come in over faxes, by telephone or in newspaper or television advertisements.
"They make offers like, for $89.95 a month, we'll insure your whole family," she said. "People are desperate for insurance so they fall for these scams."
Companies and individuals served cease-and-desist orders included All American Health Care, Continental Health, Med One, American Med Care, Health One, Preferred Providers (NAPP), Liberty Discount Benefits, Medical Savings Program, Laren Leehin, Penny Lee, Rhonda K. Rabie, Wes Long, Steven Whatley, Anthony J. Pozzolo, Bruce Weitzberg, and their agents and representatives.
Scam artists make the policies more than tempting, according to Pat Aberle, CEO of New West Health. He said they offer to cover doctor visits, hospital stays, home care, dental, vision and more for $100 a month.
"Those offers are just too good to be true," Aberle said.
Posted by Dave at 10:09 PM | Comments (0) | TrackBack
August 04, 2004
Know A Good Lawyer?
I've spent a chunk of the today deleting spam messages that have been added to this website. They do this knowing that most website won't delete them and the extra links move them up on the search engines. And this takes time, which I am out of for today.
If there are any lawyers who would like to work on a percentage basis, I've got an idea....Since these websites are so eager to post their spa...err, advertising on my website. I'll let them do that, for a $10,000 fee per message. Of course I can't promise that the message will remain on the website for very long, but that is the chance they'd take.
Think some enterprising lawyer would like to collect on that? Could add up to millions of profits for the ambitious attorney. I bet web sites would sign up for miles around to offer this advertising, if they knew it would be collected.f
Doubt I'll find one tongiht. One other thing...
Tell your loved one's that you love them. It's one of the greatest gifts you can give.
Posted by Dave at 10:24 PM | Comments (0) | TrackBack
August 03, 2004
CoQ10 Article
There's a story that's been reprinted in a few newspapers on CoQ10. Surprisingly, the article is pretty accurate (papers rarely double-check these types of stories). I will reiterate that there is no proof that it helps those who are healthy. The studies seem to show benefit primarily for diseases that involve mitachondrial damage. As always, check with your doctor.
Key points from the article:
"CoQ-10 supplements may interact with medications, however, including some anti-clotting drugs and diabetes drugs.
Also take vitamin E if you take CoQ-10. These two substances work together, at least in lab studies.
No one knows how much CoQ-10 to take. Most studies have used doses of 50 to 200 milligrams a day. But the new study on Parkinson's tested 300, 600 and 1,200 milligrams, with the largest dose having the greatest effect.
It's expensive - usually $15 to $45 a month. The larger doses some people recommend for heart patients cost $3 a day, and a 1,200-milligram dose could cost you $10 a day or more at the health-food store.
Remember this: There's no reason to take CoQ-10 if you are healthy, "just to be safe." The long-term effects are unknown, and the price is high. If you try CoQ-10 to treat a disease, get medical advice about doses and formulations - even though it's just guesswork at this point - as well as about potential drug interactions."
Posted by Dave at 11:32 PM | Comments (0) | TrackBack
August 02, 2004
The HD Nobody Sees Article
The Huntington's Disease Advocacy Center website has another great article.
This one is written by a person with Huntington's Disease (pHD) and what she is going through every day. It is a very frank and open narrative on her life. If someone in your life is coping with Huntington's Disease and you'd like to better understand what they are experiencing, I recommend you read this article.
The writer, Kelly B., sounds like a strong and wonderful person and she's done a real service for the HD community by writing this article. She finishes the article with this wonderful thought:
"I have been at peace with my Huntington’s for several years now. I just take it one day at a time. Just like anyone else dealing with anything else.
For a long while I was stuck somewhere between anger and depression, but I realized that being mad or sad about the HD only wasted both my energy and the precious time I have left. While I do not have a choice in having HD, I still have a choice in how I chose to live my life and whether I see myself as blessed or cursed. It truly is a matter of perception. You can let the things that happen in life make you bitter, or make you better. That is sometimes the only choice we always have..."
Posted by Dave at 07:18 PM | Comments (0) | TrackBack
August 01, 2004
Another Article On The Breakthrough
One of the best article,s yet, this one from The Independent, on the genetic therapy success out of the University of Iowa. Here's the highlights from the article:
In a groundbreaking study, scientists have shown for the first time that it is possible to stop a progressive brain disease in mice with a genetic technique known as RNA interference (RNAi).
Dr Davidson said that the findings, published in this month's Nature Medicine, were among the most important results of her career because they demonstrated the possibility of directly attacking the faulty gene responsible for Huntington's disease. "I'm extremely excited about the potential of RNAi and cautiously optimistic about its possible use in human medicine," Dr Davidson said.
"This is the first example of targeted gene silencing of a disease gene in the brains of live animals and it suggests that this approach may eventually be useful for human therapies," Dr Davidson said. "We have had success in tissue culture, but translating those ideas to animal models of disease has been a barrier. We seem to have broken through that barrier," she said.
Nancy Wexler of Columbia University in New York, a world authority on Huntington's disease, said RNAi offers the most promising potential treatment for the disease she has seen. "When I first heard of this work, it just took my breath away. Its everything you ever wanted to hear and more," said Professor Wexler, president of the Hereditary Disease Foundation in New York and a member of the team that originally discovered the Huntington's gene.
Phillip Sharp, a Nobel laureate from the Massachusetts Institute of Technology in Boston, said Dr Davidson's findings were "striking" because they demonstrated that RNAi may work for human patients suffering from a range of debilitating brain diseases. "It shows that, in the context of the biology, it's possible to do. This is a significant step, there's no doubt about it," Professor Sharp said.
The big question was whether this ability to silence genes could be "delivered" to all the cells of the body that needed it. Beverly Davidson's work shows that in a mammal the delivery of RNAi can work, even across the notoriously difficult biological barrier that protects the brain. She used a harmless carrier vehicle called adeno-associated virus to take the RNAi molecule into the diseased cells of the brain where the defective Huntington gene needed to be silenced. It worked.
"The broader science of RNAi is spectacular. Its just absolutely spectacular," Professor Sharp said.
"This is not hype. The biggest science prizes in the world will fall to RNAi."
Posted by Dave at 09:45 PM | Comments (0) | TrackBack
John Edward's Convention Speech
For John Edwards, he had to speak very carefully about health care as he made tens of millions of dollars suing doctors for malpractice. This has not endeared him to the medical community. Today, there are many who believe that lawsuits are driving up the cost of health care.
As a presidential candidate, Edwards did not include much on his website about health care and nothing on medical research. So what did Edwards have to say about health care? Here are the excerpts from the speech:
"(My mother's) last job was working at the post office so my parents could have health care."
This sentence makes a subtle point as the cost of health care.
"We can build one America where we no longer have two healthcare systems. One for people who get the best healthcare money can buy and then one for everybody else, rationed out by insurance companies, drug companies, and HMOs—millions of Americans who don’t have any health insurance at all."
There's a lot in these two sentences. He seems to be advocating nationalized health care which has been disasterous in other countries. Here, he's negative on drug companies and that is unfortunate for the Huntington's Disease community. Without an effective treatment or cure, we need research. If he plans to reduce the ability of pharmaceutical companies to make a profit, it will hurt investment in those companies and it will have the effect of reducing research. Most likely, 'rare' disease would be affected the most as there is less profit potential already. This position is, however, popular with voters as most are concerned with 'costs' and not medical research.
"We have a plan that will offer everyone the same health care your Senator has. We can give tax breaks to help pay for your health care. And we will sign into law a real Patients’ Bill of Rights so you can make your own health care decisions."
Try as I might, I couldn't find anything on the Senate's health care plan. It sounds good, but is it practical and affordable? I'm all for a patient having control over their medical care but, as they say, "the devil is in the details". We'll have to see what they are actually proposing to be in this "Bill of Rights".
(T)he 26 million veterans in this country won’t have to wonder if they’ll have health care next week or next year—they will have it always because they took care of us and we will take care of them.
This sentence is a bit disingenuous. There is no worry about whether veterans will or will not have health care. They'll have it. The problem is..what is the QUALITY of the health care. While the services of the VA have improved over the last 20 years, it still has areas where it is substandard. I'd like to hear more on how they would do to improve it.
So that's the health care elements of Edward's speech. It was a well-written speech that did not do him any harm. He had nothing to say about medical research and there are still a lot of questions and concerns about the health care policies. So we'll have to see if more details come out over time.
I'll be writing about Kerry's acceptance speech shortly and he had several references to health care. In four weeks, the Republicans will be having their convention and I'll write about their speeches as well. I'll be writing about both parties official platforms on health care and medical research in addition to what both candidates have on published on their websites.
Posted by Dave at 08:36 AM | Comments (0) | TrackBack