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May 31, 2004
Memorial Day
Today is Memorial Day in the United States. Officially this day is to remember America's war dead, however it has become a day when families remember their lost loved ones.
Today is also the last day of Huntington's Disease Awareness Month. Unfortunately, many in our community are unable to discuss with friends and co-workers their struggles and losses in life due to HD. So many people struggle silently as they wrestle with this disease.
For those of you grieving today... may you find comfort and peace.
For those of you who can...do what you can to educate others about Huntington's Disease. The more people who are aware of the disease and the needs in the community, the more support we will receive.
To everybody...please contact your congressman and let him/her know that the House needs to pass the Genetic Anti-Discrimination bill so that this disease no longer has to remain hidden.
Best wishes and hope to all of you on this Memorial Day.
Posted by Dave at 08:54 AM | Comments (0) | TrackBack
May 30, 2004
Reading Genes For Disease
NPR's All Things Considered just finished their four-part series on genetic testing. You can hear these shows by going to these links to NPR's website.
Part 1 Reading Genes for Disease - Tests Reveal Answers that Can Offer Relief, or Despair
Part 2 Reading Genes for Disease - One Couple's Decision Against Testing for Cystic Fibrosis
Part 3 - Huntington's Researcher's Work Led to Genetic Test -- and Family Dilemma (Dr. Nancy Wexler
Part 4 Dilemmas - Ability to Cure Disease Lags Behind Ability to Predict It
Posted by Dave at 10:26 PM | Comments (0) | TrackBack
May 29, 2004
Oh Canada
Unlike in the United States, health care has been a major topic in Canada's political campaigns.
The Globe and Mail has a commentary piece with the wonderful title "Health Care Experts? Ask The Sick". It's definitely worth a read. Here's an excerpt:
"The Canadian health-care system is not great. It's not even very good. Mostly, it is mediocre, but the saving grace for many people, Liberals included, is that it is usually equally mediocre for almost everyone."
This was in response to:
"As Mr. Martin cried aloud the other day, incomprehensibly if passionately, "I love the United States, but I love far greater that we are different! I love that we are independent! I love that we are Canada -- and we're going to stay that way!" Yes, indeed: We are great; we are not Americans; we have the best health-care system in the world, and no one, least of all some big-headed Conservative boy with the initials S.H. is going to take that from us, hear?"
The writer then goes on to discuss how she has seen the quality of health care decline under Canada's system.
Posted by Dave at 11:59 AM | Comments (0) | TrackBack
May 28, 2004
Stem Cell Research
I've got a pet peeve. (One of many I assure you.)
I've seen a lot of articles in the press lately on "stem cell research". However, virtually every one of these articles were referring to the controversial "embryonic stem cell research" that uses human embryos for material. Stem cells are pulled from many difference sources including umbilical cords, bone marrow, and fat. They are not just pulled from embryos.
What irritates me is that writers, deliberately or accidentally, are confusing the issue by not distinguishing between embryonic stem cell research and other stem cell research. There is a lot of exciting stem cell research that doesn't involve embryos that isn't getting a lot of press.
Michael Fumento has a wonderful article on current stem cell research and the surrounding politics. It's a must read for anybody interested in stem cell research.
For the record...I'm not convinced the stem cell research will ever offer much benefit to Huntington's Disease. It's an invasive procedure that involves physically implanting cells into the brain (with the inherit risks). It's a bit like adding air to a tire with a hole in it. Early research isn't showing any advantage (so far) over non-invasive treatments. However when a cure or effective treatment is found, it might very well help repair the brain. Using the same analogy, that would be like adding air to a tire after the hole has been fixed.
Here the final paragraph from the article:
"Moreover, to the extent that breakthroughs with ASCs (adult stem cells) are confused with ESC (embryonic stem cell) technology, it harms public support for ASC research. ESC propagandists are hoping for a seesaw effect; that by exaggerating ESC research and denigrating ASC research they'll push up their side of the board. But, to the extent they succeed, they're only delaying the stream of miracles coming from adult stem cells."
Posted by Dave at 11:08 PM | Comments (0) | TrackBack
May 27, 2004
Cause Of Aging
From Better Humans an article on the mitochondria, the portion of cells that Huntington's Disease most affects.
"Mitochondria convert energy from food to ATP, which cells use for power.
They have their own DNA, called mitochondrial DNA, and researchers have hypothesized that mutations in this contribute to aging by interfering with how cells generate energy.
Previous research has shown that defects in mitochondrial DNA accumulate with age, but it wasn't clear whether such defects were a cause or a symptom of aging.
"What we have now is this clear-cut cause-and-effect relationship," Larsson said in an interview. "It will provide a completely new angle to treat aging-related problems."
Posted by Dave at 11:07 PM | Comments (0) | TrackBack
May 26, 2004
CAG Counts - Why Is 40 A Problem?
There are a number of diseases that are a result of too many repeats in a DNA sequence. For Huntington's Disease it tends to be 40+ CAG repeats in one specific spot.
Recently, it's been theorized that this is due to the replication process becoming 'stuck', kind of like a skip on a record album. (If you're under 35, ask your parents what that means.) This causes the number of repeats to expand over time.
Researchers studying Friedreich's Ataxia, another disease involving 40+ repeats in a DNA sequence, found that the longer strands of repeats caused the DNA to become three-stranded instead of the 'normal' double-helix formation. Somehow this causes the replication process to get stuck in a loop.
It wouldn't be a bit surprising if this HD gene also resulted of the three-stranded formation. You can read an article on this here (from the Chicago Sun-Times.)
Posted by Dave at 05:46 AM | Comments (0) | TrackBack
May 25, 2004
How Common Is HD Around The World?
The Stanford Hopes website has a synopsis of the current information on Huntington's Disease around the world.
Take these numbers with a 'grain of salt'. The research has been less than thorough in many parts of the world. Click here.
Posted by Dave at 08:40 PM | Comments (0) | TrackBack
May 24, 2004
WaPo And HD Violence
The Washington Post makes two references recently to HD patients assaulting others. I hate to see articles like these as it can stigmatize those with Huntington's. However, it is not uncommon for patients who are not properly medicated to become irritable and angry. Here's the two pieces (login required):
Here and Here.
Posted by Dave at 11:05 PM | Comments (1) | TrackBack
May 23, 2004
A Wonderful Article On Dr. Goodman
There's a great article on Dr. Goodman in the Houston Chronicle.
Dr. Goodman is a founder and driving force behind HDDW, an organization devoted to finding better treatments to those with Huntington's Disease. What she is doing is somewhat controversial and different. I believe it has a chance to save peoples lives and is a nice compliment to what other organizations are doing.
This is a wonderfully written article and I recommend reading it.
Posted by Dave at 07:01 PM | Comments (0) | TrackBack
May 22, 2004
One More - Rapamycin
This time from the Better Human's website:
"(Rapamycin) was isolated in 1975 from a strain of bacteria found in soil on Easter Island, and is now marketed as "Rapamune" by Madison, New Jersey-based Wyeth, having received US Food and Drug Administration approval in 1999.
Rubinsztein and colleagues are interested in studying the effects of rapamycin for inducing autophagy—the feeding of the body upon itself—to clear mutant huntingtin from the body.
Rapamycin has been found to cause in cells a physiological response akin to nutrient starvation, and to induce autophagy even in cells growing in a nutrient-rich culture..."
"They found that rapamycin protected against neurodegeneration in a fly model of the disease and improved performance on four different behavioral tasks in a mouse model of the disease.
The researchers are now building on and refining their studies before considering human trials."
Posted by Dave at 03:37 PM | Comments (0) | TrackBack
May 21, 2004
Generations Of Hope Announcement
This comes from Traci:
Generation’s of Hope is building a unique experience designed specifically for children and families affected and “at risk” with Huntington's disease, ataxia, Parkinson’s disease and other Hereditary Genetic Disease. It is a 6-day, 5-night getaway at the Generation’s of Hope Camp for children and Families of Genetic Disease.
It is an extraordinary opportunity for people with Huntington's disease, and other Hereditary Genetic Disease to commune with nature, make new friends and share experiences with others who know what they face. It is a time for learning from professionals about what is on the horizon for a cure and the newest treatment options, playing team sports, singing campfire songs, horseback riding, Animal friends, and water rafting when available, in the beautiful Wallowa Mountains.
You can make this a reality for these families by donating to the camp fund capital campaign by August 19th 2004 to help us raise the $50,000.00 needed to purchase this camp in Wallowa County for a population who desperately needs these programs and many more.
Please send donations to:
Generations of Hope.
P.O. Box 832
Joseph, Oregon 97846
Or call us at 541-426-5966
Thank you for your support and we can do this together.
http://generationsofhope.tripod.com
Posted by Dave at 04:10 PM | Comments (0) | TrackBack
May 20, 2004
Justice Served
She got 5 years in the state pen, plus 10 more years of probation.
Queen Elnora Graham was sentenced this week for stealing over $47,000 from an elderly couple; the wife has Huntington's Disease. Juror's, or the judge, didn't buy her story that the a purchase of leather mini-skirt was for a 63-year-old HD patient in a wheelchair. What a shocker.
Here's what the judge had to say:
"Frankly, I have to tell you I'm amazed you can stand here and proclaim your innocence in the face of all the evidence I've heard," Blackwell told Graham before he imposed sentence. "All I've heard from you is: 'Everyone else is wrong and I'm right.'" ...
"The defendant was spending them into oblivion," Brock said. "The defendant has taken some of the most needy and defenseless people in our society, and she has exploited them." ...
"I'm sorry for your family, but you brought this on yourself," the judge said.
Posted by Dave at 08:07 AM | Comments (0) | TrackBack
May 19, 2004
BBC Article On Rapamycin
Not much new here but here's an excerpt from the article:
The research by the Department of Medical Genetics at Cambridge University, funded by the Medical Research Council and the Wellcome Trust, found that rapamycin can reduce the levels of a toxic protein causing Huntington's.
It does this by speeding up the break-down of the protein in cells.
Dr David Rubinsztein, who led the research, said: "This is an exciting development which could be tremendously important for people suffering from Huntington's Disease.
"Rapamycin is designed for long-term use, which is obviously crucial for someone who has this disorder.
"It is not without side effects, but you could argue that you'd be balancing side effect risk with the potential benefits."
He said more research would be need to be carried out before the drug could be used to treat Huntington's in humans, but studies would be done "as fast as possible".
Posted by Dave at 06:33 AM | Comments (0) | TrackBack
May 18, 2004
Papamycin Study Abstract
Here's the abstract for the Rapamycin study. The interesting thing to note is that they found "improved performance" not just a slowdown in the progression. I haven't seen the whole study yet, but this is certainly interesting.
Inhibition of mTOR induces autophagy and reduces toxicity of polyglutamine expansions in fly and mouse models of Huntington disease
Huntington disease is one of nine inherited neurodegenerative disorders caused by a polyglutamine tract expansion. Expanded polyglutamine proteins accumulate abnormally in intracellular aggregates. Here we show that mammalian target of rapamycin (mTOR) is sequestered in polyglutamine aggregates in cell models, transgenic mice and human brains. Sequestration of mTOR impairs its kinase activity and induces autophagy, a key clearance pathway for mutant huntingtin fragments. This protects against polyglutamine toxicity, as the specific mTOR inhibitor rapamycin attenuates huntingtin accumulation and cell death in cell models of Huntington disease, and inhibition of autophagy has the converse effects. Furthermore, rapamycin protects against neurodegeneration in a fly model of Huntington disease, and the rapamycin analog CCI-779 improved performance on four different behavioral tasks and decreased aggregate formation in a mouse model of Huntington disease. Our data provide proof-of-principle for the potential of inducing autophagy to treat Huntington disease.
Posted by Dave at 11:23 PM | Comments (0) | TrackBack
May 17, 2004
Rapamycin
One more article on Rapamycin, a drug used in organ transplants that researchers are are investigating for use in Huntinton's Disease. An excerpt:
"Tests in cell, fly and mouse models of Huntington’s disease carried out by Dr Rubinsztein’s laboratory at the Department of Medical Genetics have shown that a known drug called rapamycin can reduce the levels of the toxic protein causing Huntington’s. The treatment delayed the onset and progression of disease.
“This is an exciting development which could be tremendously important for people suffering from Huntington’s Disease,” said Dr Rubinsztein. “Rapamycin is designed for long-term use, which is obviously crucial for someone who has this disorder.”
Posted by Dave at 07:12 AM | Comments (2) | TrackBack
May 16, 2004
Drug May Control HD
Cutting to the chase, here's a quote from the article:
"This is an exciting development which could be tremendously important for people suffering from Huntington's disease," he said.
"Rapamycin is designed for long-term use which is obviously crucial for someone who has this disorder."
The drug reduces levels of the toxic protein which causes Huntington's disease, the researchers report in Nature Genetics."
Here's the article. I'll post more information as it becomes available.
Posted by Dave at 09:25 PM | Comments (0) | TrackBack
May 15, 2004
Nobel Laureate Battles Discrimination
While we wait for the House to pass the Genetic Anti-discrimination bill, a British Nobel Laureate makes his case for banning genetic discrimination.
Sir John Sulston is an expert on genetics and is on Britain's Human Genetics Commission. Read the article but here is one great point:
"There are genetic tests for about 200 disorders and six out of 10 Britons could, before the age of 60, develop a condition determined in part by their DNA. "
"But the number of tests available is due to rocket. It is estimated that about 10,000 disorders could be the result of mutations in single genes, and tests for many of these are in development."
It's time for an anti-discrimination bill. Call your representative and ask that they support this bill.
Posted by Dave at 03:26 PM | Comments (0) | TrackBack
May 14, 2004
HD Article
Savannah Morning News has a great article on Huntington's Disease.
Read the article, but here is a few excerpts:
"A lot of our members have guilt that they may have passed it on their children. Many people can become suicidal," said Cindy Arnsdorff, co-facilitator of The Lowcountry's Huntington's Disease Support and Education Group.
"The reason you don't hear about Huntington's is because it is so draining on families providing the care. We often don't have the time left over to educate people about it."
Today, Cindy, 50, takes care of her 58-year-old husband and best friend who can barely walk and talk. "You don't get much back from a Huntington's patient when they are at the stage my husband is at," Cindy said. "But when you've had 20 good years you make that sacrifice."
Posted by Dave at 02:31 PM | Comments (0) | TrackBack
May 13, 2004
It's A Bird - Part III
There's another review of "It's A Bird" in Salon.com:
"But if you still believe at this late date that comics are strictly for kids, take a look at DC's adult readers' line, Vertigo. Hitchcock would be proud of that title, as the protagonists of Vertigo's newly released or upcoming "Lovecraft," "Y: The Last Man" and "It's a Bird" are harried males at the mercy of oppressive -- sometimes feminizing -- forces arrayed against them..."
"Steven Seagle and Teddy Kristiansen's "It's a Bird" seizes upon the Superman mythos as its point of departure, but it is actually about Seagle's struggle to write a Superman comic as his family succumbs to Huntington's disease. "The two subjects collided in a unique amalgam of family history and Superman deconstruction," Seagle explained to me in an interview. "I realized I could tell one story using the other as an emotional punching bag, and that seemed like something I had never seen done before."
"There have been other metafictional moments in comics -- Grant Morrison's appearance as himself in his own superhero comic 'Animal Man' comes to mind -- but 'It's a Bird' is a bigger departure for comics than just metafiction," he added. "This is a book about the real-world Superman, the one who exists only as a comic book character. It's about the absurdity of trying to chronicle a man of infinite powers while living in a world populated with people whose 'powers' -- to speak, to walk, to feel -- are waning."
Posted by Dave at 05:45 AM | Comments (0) | TrackBack
May 12, 2004
Amarin Gives 1Q Earnings
Amarin, the company with a Phase III Huntington's Disease drug, has announced their 1Q earnings.
The good news is that they are still in business. The bad news is that they don't have any income at the moment. At least this will motivate them to conduct and complete the (very) delayed Phase III trial of LAX-101, a fish-oil derivative. Not much else of interest in their press release but you can read it here.
Posted by Dave at 07:39 PM | Comments (0) | TrackBack
May 11, 2004
Kerry Health Care Speech
Medpundit has critiques on a speech Kerry gave to gratuating nurses. Definitely some interesting comments.
This was the first time I had heard of "Association Health Plans". Here's an excerpt:
"He could make health insurance premiums as affordable and competitive for small business owners as they are for large corporations and unions without spending one tax dollar by endorsing Association Health Plans. But Senator Kerry doesn't endorse them. Instead, he's their chief opponent in the Senate."
Posted by Dave at 09:39 PM | Comments (0) | TrackBack
May 10, 2004
Kerry Raises Health Care Issue
For now, the topic of Health Care is now on the table.
John Kerry's theme this week is the rising costs of medical coverage. His plan:
Raise taxes on families making over $200,000/yr and having the government pay for all catastrophic care.
In this political season, the health care debate is almost solely based on lowering the cost of health care. The various candidates have offered plans that would have the unwanted effect of reducing the amount of money spent on medical research and on care for non-curable diseases. Not, what the Huntington's Disease community would want. Kerry's proposal this week, whether it is a viable plan or not, doesn't appear to have a negative effect on the HD community.
Click here to see the rebuttal from the George Bush campaign.
Posted by Dave at 06:40 PM | Comments (0) | TrackBack
May 09, 2004
Happy Mother's Day!
Though it's not a happy day for everyone in the HD community...
For those who are new to HD, holidays such as Mother's Day can be stressful to many in the community. There are mothers who feel guilty for (possibly) passing on the gene to their children. There are children who have lost their mothers and there are children who do not have fond memories of their mothers.
To the Mothers I say...
It's time you forgive yourselves. While it is true that your children will have some unique challenges in their lives, they also have the joys that come from the life you gave them.
Life is a mixture of good & bad, happiness and sorrow, and smiles & tears. This is true whether Huntington's is a part of a person's life or not. I have many friends in the Huntington's community who are very special and wonderful people. They have amazing amounts of faith, love, caring, and understanding. It's been a real honor to have them in my life. Many in our community with the gene have gone on to do great things - as folk singers, firemen, writers, and college professors (to name a few).
One more thing I need to add here...at the rate research is going on Huntington's Disease, and unlike generations past, your asymptomatic children may very well have to plan for their retirements. Things are changing!
To the children I'd like to say...Do not hang on, for too long, to the wishes and dreams of what "might have been". Look to today and tomorrow for what you can make of it. You can't control the cards you were dealt in life, but you can control how you play those cards. Make the most of what you have, it's more than you realize.
Posted by Dave at 08:18 AM | Comments (0) | TrackBack
May 08, 2004
HD Movie Upsetting Audiences
Though not for the reason you'd think...
50/50, the documentary being made on Huntington's Disease, is actually a mixture of fantasy and reality with real people and actors playing parts. Movie-goers who saw the latest version of this film (it's a work in progress) were upset about being misled by the producers of the film.
This controversy is good thing for Huntington's Disease. It raises the awareness of the movie to those in the entertainment industry. As a result many will will learn of Huntington's Disease and, perhaps, then use it in their tv shows and/or movies. A little wishful thinking, I know. But, ever little bit of exposure to our world helps. So far, it has generated articles such as this one in the Baltimore Sun. Here's an excerpt from the article:
"To Bogosian, Huntington's provided the opportunity to show how advances in genetic knowledge and testing pose moral choices unimagined a few years ago: What can or should be learned about one's genetic make-up and what is to be done with that knowledge?
The movie that was shown in North Carolina (the version in Baltimore will be a slightly different one), opens with what appears to be grainy police footage about a shocking crime: A mother in Georgia has shot to death her two sons, apparently to spare them the ravages of the illness, which had already killed her husband. So begins an introduction to Huntington's and a series of people with the disease, members of their families and medical professionals who treat and counsel them. Some of the film is in the form of interviews, but the cameras also follow them in the course of activities and meetings, some quite intimate.
Shana is a professional model, dancer and gymnast whose mother is in an advanced stage of the disease. Andrea and Will, a young married couple, are awaiting Andrea's test results. And then there's singer Arlo Guthrie, whose legendary father Woody died from Huntington's. Some of the characters are real and some are not, and there is nothing in the film - at least the North Carolina version - that signals which is which."
Posted by Dave at 10:32 PM | Comments (0) | TrackBack
May 07, 2004
Another Take On Reimportation
Mort Kondracke has a take on the drug reimportation issue. Key phrase:
"(reimportation is) tantamount to importing foreign price controls, which retard drug research and development of new treatments for disease."
As with the article from yesterday, I can't agree with everything it says. But...it does give one something to think about.
Posted by Dave at 07:51 PM | Comments (2) | TrackBack
May 06, 2004
Can't Lower Drug Prices?
Derek Lowe's "In The Pipeline" blog points to this article in Business Week.
Turns out that forcing drug prices down in one area can raise prices in another area. I can't say I agree with everything in the article, but it is a good read for anybody wanting to better understand the difficulties in lowering the costs of drugs. Click here for the article.
Posted by Dave at 08:03 PM | Comments (0) | TrackBack
May 05, 2004
NaPro Changes Name
NaPro BioTherapeutics, a young pharmaceutical company developing a promising drug for Huntington's Disease, has changed it's name to Tapestry Pharmaceuticals.
The good news from the following press release is that their HD drug could be one of four drugs that Tapestry will select for Phase I human trials over the next 18 months. Here's the full press release:
Nasdaq Ticker Symbol Changes to TPPH
BOULDER, Colo., May 4 - NaPro BioTherapeutics, Inc., (Nasdaq: TPPH) announced today that it has changed its name to Tapestry Pharmaceuticals, Inc. The company's stock symbol on the Nasdaq SmallCap Market has changed from "NPRO" to "TPPH" effective immediately. The company's new website is http://www.tapestrypharma.com.
"Our new name, Tapestry, was selected because it suggests the complex intricate interdisciplinary nature of pharmaceutical drug development. The name also reinforces our development and product strategy: to build a diverse portfolio of next generation, first in class, proprietary therapeutic products for the treatment of cancer and hereditary disease," commented Leonard P. Shaykin, Chairman and Chief Executive Officer.
Tapestry currently has six programs in pre-clinical development: four in oncology and two in hereditary disease. The company expects to advance two compounds into the clinic in 2004 and two more compounds into the clinic in 2005.
In December 2003 the company sold its worldwide generic paclitaxel business for $71.7 million minus balance sheet adjustments. The net proceeds from that sale, approximately $50 million, are now funding the development of the company's proprietary therapeutic programs. The six programs that Tapestry currently has in pre-clinical development are:
NBT-287 A proprietary third generation taxane designed to overcome multidrug (MDR-1) and mutant tubulin resistance in taxane refractory solid tumors
NBT-273 A proprietary Quassinoid analogue which appears to selectively downregulate C-myc overexpressing tumors
BBN Taxane A proprietary Bombesin/paclitaxel conjugate which selectively targets and internalizes to Bombesin overexpressing tumors
HN-1 Taxane A proprietary synthetic peptide/paclitaxel conjugate that selectively targets and internalizes specifically to squamous cell carcinomas of the head and neck
HD-1 A proprietary oligonucleotide which appears to break up the protein aggregates characteristic of Huntington's Disease
SCD-1 A proprietary "gene editing" oligonucleotide which is part of an ex vivo cell therapy treatment for sickle cell disease
"We are blessed with a rich and diverse portfolio of proprietary compounds to choose from as we transition, over the remainder of this year, the focus of our development activity into human clinical trials. Animal studies are currently underway in all but the Sickle Cell program. We will be selecting, over the next six months, two of our most advanced compounds to begin Phase I human trials. We are excited and optimistic about the future of our progress and the future of Tapestry Pharmaceuticals, Inc.," commented Mr. Shaykin.
About Tapestry Pharmaceuticals
Tapestry Pharmaceuticals, Inc. is a company focused on the development of proprietary therapies for the treatment of cancer and hereditary disease.
Forward Looking Statements
The statements in this press release that are not historical facts are forward-looking statements that represent management's beliefs and assumptions as of the date of this press release, based on currently available information. Forward-looking statements can be identified by the use of words such as "believes," "intends," "estimates," "may," "will," "should," "anticipates," "expected" or comparable terminology or by discussions of strategy, and include statements regarding the Company's expectation as to the advancing of compounds into the clinic, the selection of compounds to move into human trials and related timing. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, it cannot assure that these expectations will prove to be correct. Such statements involve risks and uncertainties, including whether any such compounds show sufficient prospect in the treatment of disease, the Company's ability to prepare appropriate regulatory filings in a timely manner and development of data or new information that delays the design of protocols and the submission of filing, as well as other factors identified under the captions "Risk Factors," "Special Note Regarding Forward Looking Statements" or "Cautionary Note Regarding Forward Looking Statements" in the Company's documents filed from time to time with the SEC, including the Company's Annual Report on Form 10-K for the year ending December 31, 2003 filed with the SEC on March 11, 2004. Should one or more of these risks materialize (or the consequences of such a development worsen), or should the underlying assumptions prove incorrect, actual results could differ materially from those forecasted or expected. The Company disclaims any intention or obligation to update publicly or revise such statements whether as a result of new information, future events or otherwise.
For further information, please contact L. Robert Cohen, Vice President, Investor Relations of Tapestry Pharmaceuticals, Inc., at 212 218 8715.
Posted by Dave at 05:50 AM | Comments (0) | TrackBack
May 04, 2004
Couch Potato Marathon
Now here is fund raiser after my own heart! From the San Diego Union-Tribune:
"There's a new marathon in town that could give all the others a run for their money. Or, rather, no run. It's the Couch Potato Marathon, a no-sweat way to support Huntington's disease research. You pay NOT to run."
"It's easy, reasons Jody Goldstein at UCSD's Huntington's disease center. A $25 donation covers NOT having to pay a race registration fee. Then consider the other ways to save and donate, say organizers: $50 not spent for new running shoes; no $100 after-race massage; tell the $250 personal trainer to take a hike; or pay $500 to conceal from your loved ones how really out of shape you are. Plus, get sponsors to contribute $1 for every kilometer you don't run. Marathon organizers love the overhead – only mailing expenses to worry about."
Posted by Dave at 08:28 PM | Comments (0) | TrackBack
May 03, 2004
May Is HD Awareness Month
Just a friendly reminder...
May is Huntington's Disease Awareness month. If you are in a position to do so, talk to friends, neighbors, and co-workers about Huntington's Disease. Let them know about the importance getting better care and finding a cure to this very hidden and devastating disease.
HD needs to come out of the closet in order to improve fundraising and help make legislative changes!
Posted by Dave at 11:30 PM | Comments (0) | TrackBack
May 02, 2004
"Blood Can Make Brain"
Forbes has an excellent article on the bone marrow stem cell news. Here's a few excerpts:
"Blood can make brain," said lead researcher Dr. Christopher R. Cogle, a postdoctoral associate from the University of Florida. "The brain is not a static organ. It does have some regenerative capacity."
"The research team looked at brain tissue from three women who had received bone marrow from male relatives to help treat leukemia. They found male Y chromosomes in the brain tissue of all three women."
"If it is possible for bone marrow to turn into nervous tissue, then everybody is walking around with a potential replacement source of nervous system cells,"
"...this finding is "amazing." It is the first demonstration that confirms that blood stem cells can become neurons."
Read the whole thing here.
Posted by Dave at 08:09 AM | Comments (0) | TrackBack
May 01, 2004
HDDW May Update
We are very lucky in the Huntington's community to have the Huntington's Disease Drug Works (HDDW). If you're not familiar with HDDW do a search on that term on this website or on the HD Lighthouse.
This is the May update from Dr. LaVonne Veatch Goodman. One of the HD community's most valuable members.
MAY UPDATE: A POSSIBLE FIX FOR THE FIX WE ARE IN
HDDW now has formal approval of Individual Therapeutic Trials in Huntington’s Disease from Western Institutional Review Board. This organization (WIRB) is an independent group of professionals who review protocols that involve the study of people in clinical trials. All research and pharmaceutical sponsored trials must undergo this process that includes review of the science, goals, methods, safety issues, consent process, and other necessary parts of the protocol. Though it was not required for our trials, we are glad to have learned from them. We thank them for their timely reviews.
We also thank the companies who have supported HDDW by offering their products to us for either no charge, or significantly below market value. These include Avicena (creatine), Brownwood Acres (blueberry extract), Cargill (trehalose), Omega Brite (omega fatty acids), and Tishcon (coenzyme Q).
HDDW is a team and we thank the major players who include: HD participants, family and friend helpers, private physicians, and HDDW coordinators at local and central levels. All of these players have volunteered time, effort and hope for the chance, not guarantee of some benefit to people with HD. We don’t deny the realities involved: We know there is no surety and that there are (and will be) obstacles to overcome.
And so we begin. We begin with trials that are different from the usual clinical trial in several ways: (1) There are no placebo controls: Each participant serves as their own. This is possible because each person with Huntington’s has their own rate of progression which is fairly constant (and therefore predictable). Therefore individual responses can be measured and compared to the “expected” progression. (2) We will observe and measure clinical response to combined agents, and (3) The participant will have choice among available agents.
RATIONALE FOR COMBINED AGENTS
Therapy development in Huntington’s disease doesn’t fit well into the usual situation of drug development.
In the past for almost every disease that has treatment, drugs have come down a development pipeline pretty much in single file. Pretty much one drug at a time.
The HD situation is different. We have many drugs near the end of the pipeline at the same time in need of clinical trial. And making the HD situation unique; many of these drugs are already safety tested and FDA approved. Ironically, this same good fortune makes for big problems if we follow the usual clinical trial path of testing one drug at a time.
As example, assume (for point of argument) that: (1) we have five potential drugs, (2) these drugs, as in the mouse model, are expected to have modest benefit, (3) drugs shown beneficial will likely give better results when used in combination as is the case in the mouse model. For this example, the math tells us that we need thirty-two separate clinical trials to study five drugs alone and in all possible combinations. Thirty-two. And this assumes best dosage is known in all five drugs, which unfortunately isn’t the case.
One potential solution to this problem is to stringently select the very few agents that can go to clinical trial among dozens of candidates. This is the SET-HD research approach. If we use this approach (even with stringent selection), it is likely, even if the drugs we have now are beneficial to take at least a decade before first treatments to get to people.
BACKWARDS MAY BE BEST
So HDDW is using a different approach by making the clinical trial design a better fit for HD and the present situation of many drug candidates. We’ll approach from the other side: We’ll go backwards. This approach is feasible because HD has several safe drugs already available.
It works like this: HDDW trials will measure clinical parameters of up to six combined agents. Each will be added at intervals of at least a month under the medical supervision of the private physician. HDDW will make these agents available to individuals with HD. HDDW will give participants choice over which and how many agents they want to try. Clinical measures of individual disease progression on combined agents will be obtained using web technology interactive programs.
There are at least three possible outcomes:
(1) We find no benefit. Even so we will have provided increased medical supervision of HD people at the local level. The HD community will have more information that these agents might be a waste of money. And then we’ll go back to the drawing board; just as was necessary for the research community after the CARE study. Sadder, but wiser.
(2) We find small benefit. Even if benefit is partial and even if for the minority, this would be a good thing. Then further new and refined trials would be in order.
(3) We find greater benefit. This result is possible: Not for sure, but possible. If we have this good fortune, then we can go backwards; by subtracting one agent at a time. In this manner, agents not useful can be eliminated.
And most importantly, if any of these drugs work, trials using several agents from the start will have maximum chance of benefit for the HD person in the shortest time. Backwards might be better for this generation. It might be the fix for the fix we’re in.
Posted by Dave at 06:10 PM | Comments (0) | TrackBack