Friday, February 29, 2008
AM - Gene breakthrough for motor neurone disease [This is the print version of story http://www.abc.net.au/am/content/2008/s2175916.htm]
AM - Friday, 29 February , 2008 08:00:00
Reporter: Sophie Scott
PETER CAVE: Australian scientists have made an important discovery about what causes the devastating illness, motor neurone disease. They have found it's an abnormal gene which kills the nerves from the brain to all the muscles in the body.National medical reporter, Sophie Scott, is speaking to Professor Garth Nicholson from the ANZAC Research Institute.
GARTH NICHOLSON: Some years ago, it was found that a particular protein was present in large amounts in the spinal cords of people dying from motor neurone disease and associated dementia, which is often occurring with motor neurone disease. This protein is called TDP43. So, we looked at TDP43 in families with motor neurone disease to see if it could cause motor neurone disease running in families, because we didn't know whether the protein found in the spinal cord was trying to help the body recover from the disease or was actually poisoning the body. And so this was the smoke, it had something to do with motor neurone disease, and we've actually found a fire, because we have proven in the families that have motor neurone disease with a mutation in this gene, this is causative, it causes motor neurone disease.
SOPHIE SCOTT: So, how important is that, given that we haven't really known what causes this disease up until now?
GARTH NICHOLSON: This … many people have thought for years that motor neurone disease might be some poison of some sort in the environment. But here we've been able to show that it seems to be a poison in the body itself, something's going wrong so the mechanism actually becomes dangerous and leads to the death of the neurones.
SOPHIE SCOTT: What are the implications of that for things like prevention or treatment?GARTH NICHOLSON: Yes, if we're right and this is indeed the protein that's causing damage in the majority of patients with motor neurone diseases, so opens up the chance of trying to reduce this protein or get rid of it, or prevent it. And then you'd have either a prevention or a cure for the disease.
SOPHIE SCOTT: At the moment, what's the situation with either treating patients with motor neurone or offering them some hope?
GARTH NICHOLSON: Motor neurone disease is probably many diseases, and some varieties are extremely lethal, killing people in three months, and some varieties go for years and years, you know, it doesn't really kill people. So, there's a big variation in motor neurone disease and this may reflect all different varieties of motor neurone disease with different mechanisms. But if there's an underlying mechanism that's common to all, then you've got a chance of a treatment. And because this protein is generally … is found in all motor neurone disease, this does offer some hope that we might find a general treatment for all.
PETER CAVE: Professor Garth Nicholson, ending that report from our national medical reporter, Sophie Scott.
Aussie researchers discover cause of motor neurone disease
By medical reporter Sophie Scott
Posted Fri Feb 29, 2008 7:00am AEDT Updated Fri Feb 29, 2008 9:19am AEDT
Audio: Scientists find motor neurone disease gene (AM)
Australian scientists have discovered the cause of motor neurone disease.
Scientists at Sydney's ANZAC Research Institute have been studying patients with the disease for the last 10 years.
They have found that patients with the debilitating illness have an abnormal protein that causes the fatal illness.
Lead scientist Professor Garth Nicholson says the findings open the way for new treatments.
"The proof of it is that we found patients who have this abnormality with the disease in families and people who didn't get the disease didn't have this abnormality," he said.
Professor Nicholson says the gene causes a poison that destroys motor neurones.
"Many people have thought for many years that motor neurone disease might be a poison of some sort, in the environment," he said.
"But here we have been able to show that it seems to be a poison in the body itself - something is going wrong and leads to the death of the neurones."
The findings have been published in the journal Science.
EDITOR'S NOTE: This article originally incorrectly stated the cause of motor neurone disease was a gene. It is in fact a protein
Motor neuron disease breakthrough
By Roger Highfield, Science Editor
Last Updated: 7:01pm GMT 28/02/2008
A breakthrough in understanding motor neuron disease, the incurable degenerative disorder, has been made by scientists.
The world's best known scientist, Prof Stephen Hawking, is a sufferer of the devastating disorder. Now an international effort led by King's College London reports in the journal Science that it has identified a rare mutation associated with some cases that they believe reveals the underlying mechanism that causes the disease for the first time, a key step towards developing effective treatments.
Deposits of an abnormal protein now appear central to the processes that trigger the dying neurons characteristic of the disease, also called amyotrophic lateral sclerosis (ALS), linking it to other ones marked by abnormal proteins, notably Parkinson's, variant CJD and Alzheimer's disease.
To make the find, Prof Chris Shaw, Jemeen Sreedharan and colleagues in the United Kingdom and Australia analysed a large British family with the inherited form of the disease. They found mutations in a gene called TDP-43 that can directly cause the disease.
Clumps of the TDP-43 protein accumulate within dying motor neurons. This discovery indicates that TDP-43 accumulation is not just a by product of nerve degeneration - as some had thought - but may be a critical event that is central to the disease process.
At the moment, doctors do not know the cause of over 95 per cent of cases of the disease, though they do know that genetic factors play an important role. Because drugs based on the only other gene linked to the disease, called SOD, had failed to have any beneficial effect in patients scientists had been searching for a new clue to the cause.
TDP-43 is depositied in 95 per cent of patients and although TDP-43 gene mutations are rare they do give us a potentially powerful biological tool. "If we can use the mutant human gene to generate cellular and animal models of MND we have a real chance of understanding how motor neurons degenerate. This could dramatically accelerate the drug screening process", says Prof Shaw.
Prof Shaw said the find opens "fantastic opportunities" and is now going to create human stem cells containing the mutation with Sir Ian Wilmut of Edinburgh University, which will be an invaluable tool to simulate, understand and test treatments for the devastating disease.
The disease attacks the upper and lower "motor neurons", causing weakness and wasting of muscles, increasing loss of mobility in the limbs and difficulties with speech, swallowing and breathing. It can affect any adult at any age but most people diagnosed with the disease are over the age of 40, with the highest incidence occurring between the ages of 50 and 70.
The incidence or number of people who will develop the disease each year is about two people in every 100,000. The prevalence or number of people living with MND at any one time is approximately seven in every 100,000.
Brian Dickie, director of research development at the MND Association, which partly funded the study, said: "The discovery of a new cause of the disease is of international importance, allowing researchers around the world to rapidly generate more pieces of the complex puzzle that is MND.
"This new information will be a spring board to greater understanding of the processes that cause motor nerves to die - and it is through such understanding that we will develop the treatment strategies to defeat this devastating disease."
Information appearing on telegraph.co.uk is the copyright of Telegraph Media Group Limited and must not be reproduced in any medium without licence. For the full copyright statement see Copyright
Wednesday, February 27, 2008
Tuesday, February 26, 2008
Italian ALS study
(48 patients, after 15 months)
32 TAKING RILUZOLE: 30 percent mortality, disease progressed 50 percent.
16 TAKING RILUZOLE & LITHIUM: 2 deaths-one by heart attack and one had advanced bulbar at the start of the study, 14 others w/ no measurable progression.
Source: Francesco Fornai, University of Pisa
THEY ARE DOING A STUDY WITH A 100 PALS. AND IN THE USA THEY ARE STARTING A CLINICAL TRIAL AT JHU. I'M ON LITHIUM SINCE THURSDAY WITH NO SIDE AFFECTS. IT WILL TAKE ONE MONTH TO FULLY INFILTRATE MY BLOOD.
EXCITED DOESN'T EXPLAIN MY EMOTIONS RIGHT NOW. IT'S MORE GRAND THAN EXCITED-IT IS HOPE! SOMETHING WE PALS LIVE FOR..............
Monday, February 25, 2008
Founded in 2004 by three MIT engineers whose collective experience spans from running the world's only non-profit biotechnology laboratory to large-scale online commerce applications, PatientsLikeMe is a privately funded company dedicated to making a difference in the lives of patients diagnosed with life-changing diseases. Our personal experiences with ALS (Lou Gehrig's disease) inspired us to create a community of patients, doctors, and organizations that inspires, informs, and empowers individuals. We're committed to providing patients with access to the tools, information, and experiences that they need to take control of their disease.
In 1998, a young carpenter named Stephen Heywood was diagnosed with ALS. The Heywood family began taking charge of Stephen's care, searching the world over for ideas that would extend his life and improve the way he lived. This set in motion a series of events that have led to PatientsLikeMe, a new system of medicine by patients for patients. We're here to give patients the power to control their disease and to share what they learn with others. We're here to help you.
Our goal is to enable people to share information that can improve the lives of patients diagnosed with life-changing diseases. To make this happen, we've created a platform for collecting and sharing real world, outcome-based patient data (patientslikeme.com) and are establishing data-sharing partnerships with doctors, pharmaceutical and medical device companies, research organizations, and non-profits. Contact us if you're interested in working together to achieve our goals.
Saturday, February 23, 2008
FROM THE DIRECTOR
Exciting New Human ALS Trial: Lithium and Riluzole
Recently, a study was published in the Proceedings of the National Academy of Science, by an Italian group of scientists and clinicians examining the ability of lithium to increase the neuroprotective actions of riluzole in ALS mice and patients.
This study was highly significant in that it showed, for the first time, a marked potentiation of the actions of riluzole in ALS patients. It showed that riluzole and lithium - combined - were far more effective than riluzole alone at increasing survival and slowing disease in ALS patients.
Although riluzole has been shown in multiple, large, independent human trials to be effective in ALS, its actions to slow disease are quite small. Lithium, a salt, has many, many actions in the nervous system but is best known for its ability to treat a very different brain disease: manic depression.
The combination of both lithium and riluzole in ALS patients appeared to keep patient alive longer and slow loss of breathing capacity and other clinical measures of disease progression.
To those of us who work very hard to translate science into effective therapeutics and to those who diagnose and manage ALS patients, this new study is extremely exciting.
It shows that drug combinations, when studied carefully, might actually very significantly slow down ALS and extend the lives of patients.
The actual study by the Italians was quite small. And the history of most modern medical research has taught us that small studies with patients need to be replicated with larger studies. All too often results of small studies are not reproduced when better and larger studies are carried out.
For this reason Packard Center clinicians and scientists are in the process of working with major ALS non-profits, the government, and European colleagues to move as quickly as possible to replicate this very small study with a larger one.
Details of the study will be reported as soon as we know more.
Does that mean patients should wait until a larger study of lithium and riluzole is complete before they consider taking the drug? Lithium is a drug very widely used for psychiatric disorders like manic depression. Its use must be monitored by physicians with appropriate physical exams and blood tests.
All patients interested in taking lithium plus riluzole should discuss this with their local physicians and/or neurologists.
The community of scientists and patients have all too often been disappointed with outcomes of ALS clinical trials, especially after we all get excited by experimental mouse data. But human trials provide the only data that really count.
We deeply hope that this novel combination of riluzole and lithium will turn out to be the “real thing” and we are collectively working hard to make that happen.
Jeffrey D. Rothstein MD, PhD
Professor of Neurology and Neuroscience Director,
Robert Packard Center for ALS Research
By Madeline ChambersThu Feb 14, 11:36 AM ET
German lawmakers are considering changes to laws on stem cell research as pressure grows for an easing of restrictions that local scientists complain prevent them from keeping up with global advances.
The Bundestag lower house of parliament devoted nearly four hours on Thursday to the divisive issue -- a sensitive subject in Germany due to Nazi genetic experiments linked to creating a "master race." A vote is set to take place in mid-March.
In 2002, parliament banned the production of embryonic cells from pre-existing stem cell lines. To ensure foreign laboratories did not produce cell lines for the German market, it also barred German scientists from working on any lines created after January 1, 2002.
German laws are tighter than in some other European countries, including Britain and Sweden, and researchers have expressed frustration that they cannot take part in international projects using lines created after 2002.
"You have to take account of the fact that researchers need qualitatively better cells," Technology Minister Annette Schavan told German television shortly before the debate, adding she wanted to ensure German scientists could keep up globally.
A range of options are on the table -- from tightening the rules and banning all imports of stem cells to lifting the ban altogether. Leaving the law as it is also possible.
A proposal to introduce a more recent cut-off date, probably May 2007, for the import of stem cell lines has won most support from lawmakers so far.
Many legislators have yet to decide and the main parties are not promoting specific positions on the issue.
Thursday's parliamentary debate reflected the deep divisions in Germany which has roughly equal numbers of Catholics, who oppose stem cell research, and Protestants.
"There is no argument for killing human life for science," said Hubert Hueppe, a member of Chancellor Angela Merkel's Christian Democrats (CDU) who wants to tighten the rules.
Some opponents of change argued the medicinal value of stem cell research had to be proven.
"The desire to heal serious diseases through embryonic stem cell research is only a wish," said Greens lawmaker Priska Hinz.
Scientists say stem cells offer the potential to treat conditions such as diabetes and Parkinson's' disease and to regenerate damaged organs.
However, scientific advances are also likely to affect the ethical debate in future. Some researchers are using cloning technology to try to make stem cells and late last year scientists said they had found a way to make stem cells from ordinary skin cells.
(Editing by Matthew Jones)
Friday, February 22, 2008
Take the time to watch, and listen and learn...
SOME THINGS I TOOK FROM THIS:
Brick walls are there for a reason---they are there to show us how bad we really want things
Don’t complain: just work harder
Tuesday, February 19, 2008
I'M DOING WELL. I WALKED OUT OF GAYLORD THURSDAY WITH A WALKER BUT HAVE NOT USED IT SINCE. IT'S LIKE GOING TO THE DENTIST-YOU NEVER WANT TO GO BUT YOU KNOW YOU HAVE TO. THAT IS WHERE I'M AT RIGHT NOW. I KNOW I HAVE TO BUT I DON'T WANT TO GO THERE. IT'S AN EGO THING-I'M SORRY BUT IT IS. I HAVE AN ARGUMENT W/ MY EGO EVERY MORNING AND IT GOES SOMETHING LIKE THIS:
"YOU KNOW IT'S THE SAFE WAY TO WALK"
"SHUT UP, YOUR GIVING IN"
"NO, IT'S WHAT'S BEST FOR YOU"
"YOU'RE WEAK-I'M SHUTTING DOWN YOUR ABILITY TO REASON W/ ME"
"I CAN'T HEAR YOU"
AND THEN THE CONVERSATION FALLS ON DEAF EARS. SO THAT'S WHERE I'M AT. I HAVE NOT BROKEN DOWN ABOUT MY DISEASE BUT 2 TIMES. AND WHEN I DECIDED TO START A BLOG-I WANTED TO PUT IT ALL OUT THERE. I THINK I'VE CONVEYED A MESSAGE THAT IT'S OKAY TO PUT YOURSELF OUT THERE AND SHARE YOUR LIFE. WELL, I BROKE DOWN YESTERDAY. PEOPLE THAT WERE W/ ME SAID TO LET IT ALL OUT. SO I DID-AND IT FELT GOOD. A GOOD CRY IS GREAT.
AND SO THE JOURNEY GOES FORWARD. I START THE LITHIUM TOMORROW............................!!!!!!!!!!!
THANK YOU AGAIN FOR ALL THE SUPPORT-KEEP IT COMING.
Saturday, February 16, 2008
MY FRIEND MIKEY FROM CHINA SENT ME THIS. HALEY NEEDS OURS PRAYERS-THANK YOU
I don't send out a MASS E-Mail like this very often. But I know this one is real because I saw the eyes of the person telling me about it. When I was in my Stastics class this morning, I was told by one of the other students that he had to go back to the hospital. When I inquired about it, he told me all about Haley. I could see the fear in his eyes, and he was fighting to stay tough. I promised him that I would pray for her, and tell as many people that I could to do the same. I can't pretend to know why something like this happens, and it sure is hard to see God in it. BUT, that is one of the simple beauties of God's great plan. We aren't suppose to understand it, or even agree with it. It would probably be our plan then, and we all know that would be totally messed up. So please, take a minute to read over this WEB-PAGE started for Haley, and just pray for her. Then, send this out to as many people as you can. I know the healing power of PRAYER through God, I have seen his touch affect my life in so many ways. This is so important to me.
So please at the very least take a minute to appreciate your loved ones. Life is so fragile, and short. PRAY FOR HALEY! ASK GOD TO STRENGTHEN HER FAMILY!--
God bless, Mikey
Friday, February 15, 2008
Thursday, February 14, 2008
THIS IS THE CLINICAL STUDY DONE IN ITALY W/ LITHIUM:
Lithium delays progression of amyotrophic lateral sclerosis
Francesco Fornai*†‡, Patrizia Longone§, Luisa Cafaro†, Olga Kastsiuchenka*, Michela Ferrucci*, Maria Laura Manca¶,Gloria Lazzeri*, Alida Spalloni§, Natascia Bellio, Paola Lenzi*, Nicola Modugno†, Gabriele Siciliano¶, Ciro Isidoro,Luigi Murri¶, Stefano Ruggieri†, and Antonio Paparelli*
*Department of Human Morphology and Applied Biology, and ¶Department of Neuroscience, Clinical Neurology, University of Pisa 56100 Pisa, Italy;
†Istituto Neurologico Mediterraneo, Istituto Di Ricovero e Cura a Carattere Scientifico Neuromed, 86077 Pozzilli (IS), Italy; §Molecular Neurobiology Unit,
Santa Lucia Foundation, 00179 Rome, Italy; and Department of Medical Sciences, University of Novara, 28100 Novara, Italy
Edited by Thomas C. Su¨ dhof, University of Texas Southwestern Medical Center, Dallas, TX, and approved December 21, 2007 (received for review
August 24, 2007)
ALS is a devastating neurodegenerative disorder with no effective
treatment. In the present study, we found that daily doses of
lithium, leading to plasma levels ranging from 0.4 to 0.8 mEq/liter,
delay disease progression in human patients affected by ALS. None
of the patients treated with lithium died during the 15 months of
the follow-up, and disease progression was markedly attenuated
when compared with age-, disease duration-, and sex-matched
control patients treated with riluzole for the same amount of time.
In a parallel study on a genetic ALS animal model, the G93A mouse,
we found a marked neuroprotection by lithium, which delayed
disease onset and duration and augmented the life span. These
effects were concomitant with activation of autophagy and an
increase in the number of the mitochondria in motor neurons and
suppressed reactive astrogliosis. Again, lithium reduced the slow
necrosis characterized by mitochondrial vacuolization and increased
the number of neurons counted in lamina VII that were
severely affected in saline-treated G93A mice. After lithium administration
in G93A mice, the number of these neurons was
higher even when compared with saline-treated WT. All these
mechanisms may contribute to the effects of lithium, and these
results offer a promising perspective for the treatment of human
patients affected by ALS.
autophagy clinical study G93A mice morphological analysis
ALS is a devastating neurodegenerative disorder with no
effective treatment that usually leads to death within 3–5
years from diagnosis (11 months for the bulbar form) (1). ALS
occurrence is primarily (90%) sporadic, while only 10% is
familial (fALS). Approximately 20% of fALS are due to mutations
of the gene coding for the enzyme copper–zinc superoxidedysmutase
(SOD1) (2). Transgenic mice over expressing the
human mutant SOD1 develop a pathology that is very similar to
that seen in ALS patients [see supporting information (SI) Text
for a comparison]. Studies in animal models or in vitro led to the
identification of a variety of alterations in ALS motor neurons
(MN) (1, 3, 4); however, other cells in the spinal cord besidesMN
are affected (5–8). For instance, a class of interneurons die
either before or concomitantly with MN, as found in mice (9, 10)
and postulated in humans for Renshaw-like cells (11). Again,
glial cells participate in the deleterious interplay leading to MN
After the generation of the SOD1 ALS mouse models,
attempts have been made to find effective treatments. However,
so far, none of these trials has led to effective clinical outcomes.
Lithium is a compound used as a mood stabilizer, which is
neuroprotective in a variety of disease models (12, 13), such as
brain ischemia (14) and kainate toxicity (15). The ability of
lithium to promote autophagy, through the inhibition of the
inositol-monophosphatase 1 (16–18), together with the protective
effects of autophagy in neurodegeneration (19–22),
prompted us to test the neuroprotective effects of lithium in the
G93A ALS mouse model. Based on the promising data, we
obtained in mice we quickly moved into a clinical trial, which is
now at the end of its second year.
Effects of Lithium on Disease Duration and Survival in G93A Mice.
G93A male mice were treated daily with lithium carbonate (1
mEq/kg, i.p.), starting at 75 days of age. Lithium treatment
prolonged the mean survival time from 110.8 5.0 days (n20)
to 148 4.3 (n 20, 36% of the life span of these mice; Fig.
1a; P 0.001) and, most importantly, increased disease duration
(from a mean of 9 days to 38 days, 300%; Fig. 1b; P 0.05)
compared with the G93A mice treated with saline. Even when
lithium treatment was started at the onset of motor symptoms,
the increase in disease duration was still comparable (data not
shown). More specifically, lithium delayed the onset of paralysis
and limb adduction (Fig. 1c) and significantly improved additional
tests we report in SI Fig. 6, such as rotarod, grip strength,
and stride length.
Effects of Lithium Treatment on Motor Neuron Survival (Lamina IX of
Lumbar and Cervical Spinal Cord and Brainstem Motor Nuclei). These
effects were accompanied by a reduced loss of lumbar MN at 90
days of age (SI Fig. 7). However, at the end of disease (which
occurred later following lithium), the number of alpha-MN
within lumbar lamina IX of the G93A mice treated with lithium
was comparable to that found in the saline-treated mice that had
died previously (SI Fig. 8). However, even at this stage, we
detected a disease modifying effect of lithium. This consisted of
(i) preservation of the size of MN (SI Fig. 8 d and e); (ii)
preservation of MN number and size in those areas [i.e., cervical
spinal cord (SI Fig. 9) or the nucleus ambiguous (SI Fig. 10)],
which degenerate later compared with lumbar lamina IX (23,
24); (iii) decreased astrocytosis (SI Fig. 11); and (iv) decreased
alpha-synuclein, ubiquitin, and SOD1 aggregation (see SI Fig. 6
and Discussion in SI Text).
Effects of Lithium Treatment on the Renshaw-Like Cell Area (Lamina
VII). Lamina VII contains a larger number of interneurons,
defined as Renshaw cells, which form a collateral circuit that
Author contributions: F.F., P. Longone, C.I., L.M., S.R., and A.P. designed research; O.K.,
M.F., M.L.M., G.L., A.S., N.B., P. Lenzi, N.M., and G.S. performed research; L.C., M.F., M.L.M.,
G.L., P. Lenzi, G.S., C.I., L.M., S.R., and A.P. analyzed data; and F.F. and P. Longone wrote the
The authors declare no conflict of interest.
This article is a PNAS Direct Submission.
Freely available online through the PNAS open access option.
‡To whom correspondence should be addressed. E-mail: firstname.lastname@example.org.
This article contains supporting information online at www.pnas.org/cgi/content/full/
© 2008 by The National Academy of Sciences of the USA
2052–2057 PNAS February 12, 2008 vol. 105 no. 6 www.pnas.orgcgidoi10.1073pnas.0708022105
Wednesday, February 13, 2008
Stem cell scientists explore options
By Yevgeniy Sverdlik, CORRESPONDENT
Article Created: 02/13/2008 02:41:12 AM PST
SAN RAFAEL — Stem cell research offers the promise of finding cures for some of the most feared and intractable diseases known to man.
A panel of biologists made that case over the weekend to an audience of Bay Area university students, other scientists and members of the community.
Stanford University's Renee A. Reijo Pera, director of the Center for Human Embryonic Stem Cell Research and Education, emphasized the importance of learning more about embryonic development at its earliest stages to realize the therapeutic potential of stem cells.
"It's important that we begin to really understand human development and how cell fate is determined," Pera said. "It's the essence of human embryonic stem cell biology."
Although scientists have learned how to create human embryonic stem cell lines, Pera said, they have yet to learn how to direct those stem cells to morph into specific types of cells that are needed to cure some of the diseases the technology promises to cure.
"Can we direct the fate of the cells?" Pera said. "In most cases, the answer is 'No, we can't do it exactly right.' But we actually are getting a lot closer."
The keynote speech was delivered by Gilberto R. Sambrano, head of the Training Grant Program at the California Institute for Regenerative Medicine. The institute was created in 2004 as a result of state Proposition 71, which charged it with distributing $3 billion of state money for stem cell research.
One of the institute's goals is to develop tangible proof of the principle that transplanted cells derived from stem cells can be used to restore function for at least one disease, Sambrano said.
To address the uncertainty of this relatively new area in science and the amount of money being spent on it, Sambrano pointed to the optimistic mind-set scientists have about it.
"Like any research conducted, it is research and it is a question," Sambrano said. "Do we want to explore it or not, and if we don't explore it, what do we miss out? I guess, until we get there, we don't know. But I think that the potential for it as an enabling technology has excited scientists like nothing has in recent times."
Mary Khan of San Anselmo came to the conference, which was held at Dominican University of California in San Rafael, to learn how far stem cell research has come. Khan's husband suffers from kidney failure.
"My husband is a dialysis patient," Khan said, "and I am trying to research what I think is the only way someone can benefit and ever possibly get help for kidneys, which is to basically to re-grow and re-generate your kidney."
THERE IS ALWAYS HOPE
Tuesday, February 12, 2008
Geron CEO plans embryonic stem cell tests in humans this spring if the biotech meets the FDA's 'high bar.'
By Aaron Smith
February 12 2008: 9:07 AM EST
NEW YORK -- The first experiments using human embryonic stem cells in human subjects could begin within a few months, the chief executive of biotech Geron said Monday.
At the annual BIO CEO conference in New York, Dr. Thomas Okarma said Geron plans to start embryonic stem-cell studies in humans with spinal cord injuries toward the end of the second quarter. Okarma said the tests would involve up to 40 human patients, while all prior tests involved rats.
This assumes that the Food and Drug Administration gives Geron a green light to proceed with the human test. Okarma said the FDA will set a "high bar" in regulating this new type of science.
Ren Benjamin, analyst for Rodman & Renshaw, believes the regulatory process could be time-consuming, because it's unprecedented.
"This is the first time that a human embryonic stem cell application is being submitted to the FDA, so there's a good chance that some questions will arise," he said.
Geron (GERN), based in Menlo Park, Calif., is also in early-stage studies with stem cell-based drugs for diabetes and heart failure. All of these experimental treatments are years away from potentially entering the market.
Advanced Cell Technology Inc. and Novocell Inc. also use human embryonic stem cells, but Geron is the only one of the these companies that's traded on the Nasdaq. Advanced Cell plans to begin testing in humans this year, but Novocell is further behind.
Cytori Therapeutics (CYTX) and Osiris Therapeutics (OSIR) use stem cells taken from adult human tissue, which insulates from the controversy surrounding the use of human embryonic stem cells, which are obtained through in vitro fertilization.
Human-based embryonic stem cells are prized by researchers for their ability to regenerate quickly and morph into different types of cells. Supporters believe their use could someday help people with spinal injuries walk again or cure patients with degenerative diseases like Alzheimer's and Parkinson's.
But the pro-life contingent, including President Bush, opposes using human-derived stem cells, on the grounds that it creates embryos only to destroy them. Supporters of the science dispute that notion, saying that the blastocysts used as stem cell sources would be thrown out as medical waste if they weren't used in research.
In 2001, President Bush limited federal funding for human-derived stem cells to only those lines that existed at the time. In 2007, he vetoed a Congressional bill to lift those restrictions.
But in November, scientists unveiled a new type of experimental technology on Nov. 21 that sent Geron's stock into a volatile tailspin. Research teams from the University of Wisconsin in Madison and the University of Kyoto in Japan separately announced that they'd "reprogrammed" adult cells to act like embryonic stem cells.
Geron's stock has fallen more than 40 percent since then, though Okarma shrugged off the potential threat of reprogramming because it's still in the infantile stages.
"The natural human embryonic stem cell is the gold standard and we have yet to see anything else come close," Okarma said at the BIO CEO conference Monday.
But Benjamin of Rodman & Renshaw doesn't believe reprogramming poses an immediate threat to Geron.
"I think [reprogramming] is very intriguing and has a lot of potential, but it's at a nascent stage that I consider more noise than anything else," said Benjamin.
Mark Gross, a physics professor at California State University who was attending the BIO CEO conference as a potential investor, agreed that reprogramming technology is in too early of a stage to threaten Geron.
"[Reprogramming] is promising, but these things take time and we don't know which path will lead to fruition and which will lead to a dead end," he said.
Monday, February 11, 2008
Wife's death was mercy killing, husband's family says
by The Oregonian
Wednesday February 06, 2008, 10:55 AM
Friends and family of John Roberts, the Gresham man arrested in the death of his wife, Virginia, say her death Saturday was not a cold-blooded attack but an act of compassion for a woman living with ALS, or Lou Gehrig's Disease.
John Roberts' brother and daughter and a close family friend told The Oregonian that Virginia's ALS had worsened in the weeks leading to her death on Saturday. They say John Roberts hinted to them that Virginia had asked him to help take her life when she no longer wished to carry on.
Friends and family of John Roberts say Virginia Roberts was born in Guatemala. No members of her family could be immediately reached for confirmation of her reported disease.
"There is a story that makes this not just a murder," said Greg Roberts, a former Seattle police detective and John Roberts' brother. "It is my firm belief that this was a pact between the two of them, that she asked him to do this.
"And part of the reason why they chose this method rather than going down the assisted suicide route was that she was so proud that she didn't want to let herself get into the condition she would need to be in before they'd be allowed" to participate in Oregon's Death with Dignity program, which requires a person to be within six months of death.
Police arrested Roberts on an accusation of murder after receiving a 9-1-1 call at 9:44 a.m. Saturday. The caller said he shot someone.
Inside the couple's one-story home at 4160 NE El Camino Drive, police found Virginia Roberts dead from a single gunshot wound to the head. Police are not releasing the recording of the 9-1-1 call. Roberts remains in jail without bail.
Roberts' attorney, Angel Lopez, and Don Rees, a Multnomah County senior deputy district attorney prosecuting the case, declined to comment. Gresham police Capt. Tim Gerkman also declined to discuss specifics of the investigation.
"We're trying to track down all leads and possible motives, or prove or disprove different theories or reasons that this crime was committed," Gerkman said.
A grand jury is scheduled to review the case on Friday. Roberts is scheduled to appear in court Tuesday.
According to the ALS Association, amyotrophic lateral sclerosis is a progressive disease affecting nerve cells in the spinal cord and brain. Those with the disease are eventually robbed of their ability to control muscle movements, leading to paralysis. About 80 percent of those diagnosed with the disease die within five years.
The ALS association estimates that 5,600 each year are diagnosed with the disease and about 30,000 Americans may have the disease at a given time.
-- Brad Schmidt
Saturday, February 9, 2008
One is Evil. It is anger, envy, jealousy, sorrow, regret, greed, arrogance, self-pity, guilt, resentment, inferiority, lies, false pride, superiority, and ego.
The other is Good. It is joy, peace, love, hope, serenity, humility, kindness, benevolence, empathy, generosity, truth, compassion and faith."
The grandson thought about it for a minute and then asked his grandfather: "Which wolf wins?"
The old Cherokee simply replied, "The one you feed".
Friday, February 8, 2008
J.M.C. has left a new comment on your post "SOME INFO ON THAT STUDY (ITALIAN STUDY ON LITHIUM)...":
"We can only hope this does not end the way Myotrophin did. Mixing with Rilutek is interesting but not all PALS agree with that drug nor do they take it. What will be their option"?
TO ANSWER YOUR QUESTION-WHAT OTHER CHOICE DO WE HAVE?? Riluzole BRAND NAME RILUTEK? I WAS TOLD BY MY DOCTOR THAT FIRST, IT REALLY ONLY PROLONGS LIFE FOR 3 MONTHS. IF I'M AT THAT POINT IN MY DISEASE-I WOULD JUST RATHER DIE WITH DIGNITY. I DON'T WANT ANY HEROIC MEASURES TAKEN WHEN I'M AT THAT POINT-THAT IS NO WAY TO LIVE--I HAVE ALREADY SIGNED THE DNR-(DO NOT RECESITATE).
AND SECONDLY, MY DOCTOR ADVISED ME AGAINST TAKING RILUTEK W/ LITHIUM-THE SIDE EFFECTS FAR OUT WEIGH THE POSITIVES. AND I PLAN ON KICKING THIS DISEASE'S ASS ANYWAY I CAN. WE DON'T KNOW ENOUGH ABOUT LITHIUM AND ALS YET AND I'M NOT GOING TO WAIT AROUND FOR THE RESULTS OF A UNITED STATES CLINICAL TRIAL-WE DON'T HAVE THE LUXURY OF TIME-THIS IS SOMETHING POSITIVE IN AN ALS WORLD DOMINATED BY NEGATIVITY.
J.M.C., IT SCARES THE SHIT OUT OF ME TO THINK ABOUT WHAT THE LATTER PART OF THIS DISEASE IS CAPABLE OF.
SO AGAIN, I ASK YOU- WHAT OTHER OPTION DO WE HAVE??????
FINALLY SOME GOOD NEWS-EMBRACE IT.
EMAIL ME AT email@example.com
Thursday, February 7, 2008
The ALS Association Works with Investigators onFollow Up Study of Lithium for Treating ALS
[Quick Summary: An Italian study of 44 people reports that a 15-month trial found that daily doses of the drug lithium, used to treat bipolar disorder, significantly slowed the progression of ALS in patients also taking riluzole, the only FDA-approved for the treatment of Lou Gehrig’s disease.
The ALS Association is working with investigators and funding organizations to plan a follow up clinical study of lithium as a treatment for ALS.
The 15-month trial conducted on 44 people with amyotrophic lateral sclerosis, commonly known as Lou Gehrig’s disease, reported that the progressive neurodegenerative disease was slowed significantly in patients receiving daily doses of lithium along with riluzole, the only drug approved by the U.S. Food & Drug Administration for the treatment of ALS.
Francesco Fornai at the University of Pisa (Italy) with colleagues at the University of Novara (Italy) and the Santa Lucia Foundation in Rome, demonstrated that lithium was neuroprotective and increased survival in the mouse model of ALS. These promising results led to the clinical study.
IF WE (PALS-PEOPLE W/ ALS) WAIT AROUND FOR THE GOOD OLD USA TO DO A CLINICAL TRIAL-WELL YOU KNOW.......................
THE FULL REPORT CAN BE FOUND ON:
Wednesday, February 6, 2008
AS YOU MAY OR MAY NOT KNOW-I WENT TO COLUMBIA LAST THURSDAY TO MEET DR. ANDREWS. SHE WAS GREAT--- THOROUGH AND EXTREMELY CURRENT. ANDRA AND I WENT ARMED W/ QUESTIONS ABOUT A LITHIUM STUDY THAT WAS DONE IN ITALY THAT SHOWED OVER A 15 MONTH PERIOD TO BRING ALS TO A STANDSTILL. IN LOW DOSES IT SHOWED THAT THE PROGRESSION OF ALS SLOWED TO A SLOW CRAWL. GREAT NEWS. THE WHITE PAPER IS GOING TO BE PRESENTED APRIL 16TH BUT NEWS HAS SPREAD LIKE WILD FIRE. DR. ANDREWS KNEW ALL ABOUT IT AND SAID IT WAS A REPUTABLE STUDY. SHE ALSO ENCOURAGED ME TO TRY IT-SO I AM IN TWO MORE WEEKS.
I HAVE TO WAIT TILL MY MUSCLE TREMOR AND ANTI-SPASTIC MEDICATION IS TITRATED. MEANING I HAVE TO BUILD UP TO MY REGULAR DOSAGE-3 PILLS A DAY:
IT IS CALLED-BACLOFEN AND IT IS USED IN PATIENTS WHO HAVE MS BUT SINCE I AM STILL REALLY STRONG AND MY MUSCLES SHOW NO SIGN OF ATROPHY, SHE THOUGHT IT WAS A GOOD IDEA. THE REASON BEING-I HAVE BALANCE ISSUES. IT WOULD BE ONE THING IF I'M HAVING BALANCE ISSUES W/ LOSS OF MUSCLE BUT I'M HAVING THE ISSUES WITH MY MUSCLES INTACT. SO, I'M STRONG ENOUGH TO HOLD MYSELF UPRIGHT IF MY MUSCLES DON'T SPASM. OTHERWISE, I WALK LIKE FRANKENSTEIN!! HAHA. HOPING THE BACLOFEN WILL RELAX MY MUSCLES WHEN I WALK. RIGHT NOW IT'S LIKE WALKING WHEN YOUR MAKING A MUSCLE-TRY IT-WALK WHILE YOUR FLEXING YOUR MUSCLES IN YOUR LEG. IT'S HARD. I TAKE IT AT BEDTIME FOR THE FIRST WEEK TO GET MY BODY TO BECOME USED TO IT. AND THE 2ND WEEK-ONE IN THE MORNING AND ONE AT NIGHT AND THEN AT WEEK 3 -MORNING-NOON-AND NIGHT. AND THEN THE LITHIUM. I WILL LET YOU KNOW HOW THAT WORKS OUT.
What is baclofen?
Baclofen is a muscle relaxant and an antispastic agent. The exact way that baclofen works is unknown.
Baclofen is used to relieve the muscle spasms, pain, and muscular rigidity associated with multiple sclerosis.
Baclofen may also be used for purposes other than those listed in this medication
ENOUGH ABOUT THAT
I'M A PERFECT GINUEA PIG FOR ALMOST EVERY CLINICAL TRIAL THEY HAVE-SO THATS GREAT NEWS. WHATEVER IT TAKES!!!!!!!!
AND I'M TOTALLY HEALTHY FOR SOMEONE WHO HAS MY CHALLANGE. SO GOOD NEWS THERE.
COLUMBIA HAS THE MOST COMPREHENSIVE MULTI-DISCIPINED ALS CLINIC IN THE USA-SO GOOD NEWS THERE TOO.
MORE TO COME..............
THAN YOU AGAIN FOR ALL OF YOUR SUPPORT-YOU PEOPLE ARE TRULY AMAZING!
Sunday, February 3, 2008
Our deepest fear is that we are powerful beyond measure.
It is our Light, not our Darkness, that most frightens us.
We ask ourselves, "Who am I to be brilliant, gorgeous, talented and fabulous?"
Actually, who are you not to be?"
You are a Child of God!
Nelson Mandela, 1994 Inaugural Speech
Saturday, February 2, 2008
AND AN ANSWER TO THE QUOTE I HAVE HIGHLIGHTED-I'M NOT DYING A NATURAL DEATH, POPE BENEDICT!! IF YOU WERE IN MY SHOES-YOU WOULD DO ANYTHING TO LIVE. SHATTERING HUMAN DIGNITY WOULD BE GIVING UP. ALTHOUGH I RESPECT ANYONE'S OPINION-I DO NOT HAVE TO AGREE-I'M GOING TO THE POPE'S MASS IN APRIL-TO BE CONTINUED..........
Pope says some science shatters human dignity
By Philip PullellaThu Jan 31, 9:57 AM ET
Pope Benedict said on Thursday that embryonic stem cell research, artificial insemination and the prospect of human cloning had "shattered" human dignity.
In an address to members of the Vatican department on doctrinal matters, Benedict said the Church had a duty to defend the "great values at stake" in the field of bioethics.
The speech was the latest in a series in which the conservative Pope has told his listeners that scientific progress should not be accepted uncritically.
Benedict, who headed the same department for years before his election in 2005, said the Church was not against scientific progress but wanted it based on "ethical-moral principles."
He said this included total respect for the human being as a person "from conception until natural death," and respect for the natural transmission of life through sexual intercourse.
Practices like freezing embryos, suppression of embryos in multiple pregnancies, embryonic stem cell research, the prospect of human cloning and artificial insemination outside the body had "shattered the barriers meant to protect human dignity," he said.
"When human beings in the weakest and most defenseless state of their existence are selected, abandoned, killed or used as pure 'biological material,' how can one deny that they are being treated not as 'someone' but as 'something,"' he said.
Such practices "questioned the very concept of the dignity of man," he said in the speech to the department known as the Congregation for the Doctrine of the Faith.
Widespread interest in medicine by the general public, who get most of their information from the media, had made it even more imperative for the Church to take a stand, he said.
Embryonic stem cell research involves the destruction of embryos. Scientists hope to use stem cells to transform medicine, providing regenerative treatments for injuries and seeking new insights into diseases like cancer and AIDS.
Last year scientists reported they had tricked ordinary skin cells into behaving like embryonic stem cells.
The Pope said the Church "appreciates and encourages" research on stem cells that come from other parts of the body and do not involve embryos or their destruction.
He rejected accusations from critics who say the Church is an obstacle to science and human progress, saying growing concern about cloning and other practices showed it was right to raise the alarm.
It was the Pope's latest foray into scientific issues. On Monday he warned against the "seductive" powers of science, saying it was important that science did not become the sole criteria for goodness.
U.S. Cardinal William Levada, Benedict's successor as head of the doctrinal department, said it was mulling the possibility of preparing a new Vatican document on bioethical issues.
(Editing by Michael Winfrey)
Today, I will remember to face my climb with optimism. Even if my progress sometimes seems slow to me, it's still a long way from where I once was.