Aa
Has anyone heard about this new drug?
Has anyone heard of this?
New kind of gene "silencing" drug works in monkeys
Last Updated: 2008-03-26 16:50:49 -0400 (Reuters Health)
By Ben Hirschler
LONDON (Reuters) - A new class of drug that fine tunes the action of
genes has been shown to cut cholesterol in monkeys and may fight a
range of ills, including hepatitis C and perhaps cancer, scientists
said on Wednesday.
The compound, from Danish biotech firm Santaris Pharma, works by
blocking or "silencing" microRNAs -- tiny strands of RNA (ribonucleic
acid) that help turn genes into proteins.
The ground-breaking study is the first demonstration of microRNA
silencing in primates and an early endorsement of the technique.
Phase I safety trials are now planned in humans.
Unlike other drugs in the hotly pursued RNA interference field, the
new designer molecule, known as locked nucleic acid (LNA), can be
given as a simple injection rather than having to be delivered direct
to affected tissue.
"We think LNA is a one-stop shop for silencing," Santaris Chief
Executive Keith McCullagh told reporters.
Scientists from Santaris and the University of Copenhagen lowered
total cholesterol in African green monkeys by up to 30 percent,
without ill effects, by targeting a microRNA linked to genes in the
liver that are involved in cholesterol metabolism.
The results were published in the journal Nature, along with other
test-tube research showing that LNA effectively blocks the production
of hepatitis C virus in human liver cells.
Santaris intends to test its first LNA compounds in humans by the
middle of this year but it will take at least five years before any
medicine is ready for submission for approval.
While the cholesterol effect is interesting, McCullagh said the most
promising opportunity actually lay in pursuing LNA as a treatment for
hepatitis C, a poorly treated viral disease that can cause serious
liver damage.
Further ahead, LNA could also have a role to play in other infectious
diseases, as well as cancer and autoimmune disorders, since many
disease-associated genes are regulated by microRNAs.
"There are great prospects for future drug development both for liver
diseases and other disease types, and Europe has the potential to
match the USA in this area," said Mike Gait at the MRC Laboratory of
Molecular Biology in Cambridge.
God Bless to One and all from Rob in ireland
New kind of gene "silencing" drug works in monkeys
Last Updated: 2008-03-26 16:50:49 -0400 (Reuters Health)
By Ben Hirschler
LONDON (Reuters) - A new class of drug that fine tunes the action of
genes has been shown to cut cholesterol in monkeys and may fight a
range of ills, including hepatitis C and perhaps cancer, scientists
said on Wednesday.
The compound, from Danish biotech firm Santaris Pharma, works by
blocking or "silencing" microRNAs -- tiny strands of RNA (ribonucleic
acid) that help turn genes into proteins.
The ground-breaking study is the first demonstration of microRNA
silencing in primates and an early endorsement of the technique.
Phase I safety trials are now planned in humans.
Unlike other drugs in the hotly pursued RNA interference field, the
new designer molecule, known as locked nucleic acid (LNA), can be
given as a simple injection rather than having to be delivered direct
to affected tissue.
"We think LNA is a one-stop shop for silencing," Santaris Chief
Executive Keith McCullagh told reporters.
Scientists from Santaris and the University of Copenhagen lowered
total cholesterol in African green monkeys by up to 30 percent,
without ill effects, by targeting a microRNA linked to genes in the
liver that are involved in cholesterol metabolism.
The results were published in the journal Nature, along with other
test-tube research showing that LNA effectively blocks the production
of hepatitis C virus in human liver cells.
Santaris intends to test its first LNA compounds in humans by the
middle of this year but it will take at least five years before any
medicine is ready for submission for approval.
While the cholesterol effect is interesting, McCullagh said the most
promising opportunity actually lay in pursuing LNA as a treatment for
hepatitis C, a poorly treated viral disease that can cause serious
liver damage.
Further ahead, LNA could also have a role to play in other infectious
diseases, as well as cancer and autoimmune disorders, since many
disease-associated genes are regulated by microRNAs.
"There are great prospects for future drug development both for liver
diseases and other disease types, and Europe has the potential to
match the USA in this area," said Mike Gait at the MRC Laboratory of
Molecular Biology in Cambridge.
God Bless to One and all from Rob in ireland
http://www.newscientist.com/channel/health/dn13539-hepatitis-c-is-first-target-for-new-therapy.html?feedId=online-news_rss20
Here too, I was just looking at it yesterday.
5 years they reckon till treatment. But what of this
http://www.newscientist.com/article.ns?id=mg19025534.600
"One way this can be done is by making viruses produce longer RNA pieces that fold in two like a hairpin. These get snipped up into siRNAs by enzymes already inside the cell. It is this approach that has proved fatal. Mark Kay's team at Stanford University in California has been studying mice treated with viruses engineered to produce a range of RNA hairpins. Many of the viruses caused severe liver damage, killing some mice (Nature, vol 441, p 537).
Kay believes the RNA hairpins disrupted essential gene regulatory mechanisms, which snip up naturally occurring "microRNA" to produce an RNAi-like effect. The hairpins seemed to monopolise the cellular machinery needed to process microRNA.
The good news is that not all hairpins have this effect - 13 of the 49 Kay tested were safe. Even so, the results will make regulators cautious about approving clinical trials based on viruses that produce hairpin RNA. And Kay's team has found no way of predicting which hairpins are dangerous. "People are just going to have to do toxicity studies," he says.
Kay's findings also do not apply if siRNAs are used directly, stresses John Maraganore, president of Alnylam Pharmaceuticals in Cambridge, Massachusetts. Alnylam is developing the respiratory syncytial virus therapy and other RNAi treatments, and its studies suggest that siRNAs are safe. Other researchers have raised concerns, though, after finding that adding siRNAs to cell cultures can silence genes other than the intended target.
We're more chimp than mouse...but still.
Here too, I was just looking at it yesterday.
5 years they reckon till treatment. But what of this
http://www.newscientist.com/article.ns?id=mg19025534.600
"One way this can be done is by making viruses produce longer RNA pieces that fold in two like a hairpin. These get snipped up into siRNAs by enzymes already inside the cell. It is this approach that has proved fatal. Mark Kay's team at Stanford University in California has been studying mice treated with viruses engineered to produce a range of RNA hairpins. Many of the viruses caused severe liver damage, killing some mice (Nature, vol 441, p 537).
Kay believes the RNA hairpins disrupted essential gene regulatory mechanisms, which snip up naturally occurring "microRNA" to produce an RNAi-like effect. The hairpins seemed to monopolise the cellular machinery needed to process microRNA.
The good news is that not all hairpins have this effect - 13 of the 49 Kay tested were safe. Even so, the results will make regulators cautious about approving clinical trials based on viruses that produce hairpin RNA. And Kay's team has found no way of predicting which hairpins are dangerous. "People are just going to have to do toxicity studies," he says.
Kay's findings also do not apply if siRNAs are used directly, stresses John Maraganore, president of Alnylam Pharmaceuticals in Cambridge, Massachusetts. Alnylam is developing the respiratory syncytial virus therapy and other RNAi treatments, and its studies suggest that siRNAs are safe. Other researchers have raised concerns, though, after finding that adding siRNAs to cell cultures can silence genes other than the intended target.
We're more chimp than mouse...but still.
http://www.bioportfolio.com/biotech_news/Santaris_Pharma_16.htm
"Dr Keith McCullagh, President and CEO of Santaris Pharma commented: “In collaboration with Professor Peter Sarnow and Sylvia Schutz from Stanford University, we also show in the study published today in Nature that LNA-mediated silencing of microRNA-122 leads to efficient inhibition of Hepatitis C virus production in human liver cells, confirming previous work by the Stanford group on the importance of microRNA-122 for Hepatitis C replication. Now that we have confirmed that an LNA-antimiR oligonucleotide can effectively antagonize microRNA-122 in primates at low doses, we are encouraged to investigate the potential of LNA-mediated therapy in the treatment of HCV infection.”
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