Bio Blast Pharma reports promising data on muscle wasting drug

Reuters

Bio Blast Pharma Ltd said its lead experimental drug showed signs of being effective in treating patients with a rare muscle wasting disorder, besides also hitting the main goals of safety and tolerability.

The company said the experimental drug, Cabaletta, hit multiple efficacy endpoints during a mid-stage trial on patients suffering from oculopharyngeal muscular dystrophy, according to interim results from study.

Oculopharyngeal muscular dystrophy is a rare disease where patients develop swallowing difficulties, which could lead to death in severe cases.

Bio Blast said the drug improved patients’ muscle function while also reducing difficulty in swallowing.

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Bio Blast is also testing the drug as a treatment for spinocerebellar ataxia type 3, another genetic condition that affects the nerves in the brain.

Up to Monday’s close of $5.04, the Israeli company’s U.S.-listed shares have lost a little over half their value since they went public in July last year.

(Reporting by Vidya L Nathan in Bengaluru; Editing by Savio D’Souza)
Read more at Reuters

http://www.reuters.com/article/2015/10/27/us-bio-blast-pharma-study-idUSKCN0SL14O20151027#MrVYQdKUvWOoXyXL.99

Stem cell trial seeks longer lives for victims of deadly ALS

 

Researchers at Emory University in the United States are hoping to extend the lives of patients diagnosed with the deadly neuro-degenerative disease, Amyotrophic lateral sclerosis (ALS). ALS kills by destroying a patient’s nervous system but in clinical trials, the scientists say injections of neural stem cells show promise in slowing the disease’s progress. Ben Gruber reports.
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Experiment seeks blood test for breast cancer

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News

Experiment seeks blood test for breast cancer

Tue, Mar 09 14:08 PM EST

http://us.mobile.reuters.com/mobile/m/AnyArticle/p.rdt?URL=http://www.reuters.com/article/idUSTRE6283XR20100309

By Maggie Fox, Health and Science Editor

WASHINGTON (Reuters) – An experimental approach that looks for the DNA leaking out from dead and dying cells may provide a route to a blood test for breast cancer, U.S. researchers reported on Tuesday.

An initial study showed the test detected 70 percent of breast cancer cases, and correctly cleared 100 percent of women who did not have breast cancer, the team at Chronix Biomedical, a privately-owned company in San Jose, California, said.

The experimental test is not ready to develop into a product but provides a basis for further research, they wrote in the journal Molecular Cancer Research.

“It is based on finding the unique DNA fingerprints from dead and dying cells,” Chronix CEO Howard Urnovitz said in a telephone interview.

Technological advances in DNA sequencing made the test possible, Urnovitz said. His team sequenced the entire genomes of 26 breast cancer patients and of 67 apparently healthy women.

They were looking for extra DNA in the blood of the breast cancer patients that would come from cells dying because of the tumors.

“If a breast cell is injured, it will overexpress the genes that make it a breast cell,” Urnovitz said. In theory, if a patient has excess DNA from breast cells that are dying, there is something going on that is killing breast cells.

The search is not easy. “The entire genome can be found in the blood,” Urnovitz said. And billions of cells die every day in the human body.

But eventually the Chronix team found what they believe is tell-tale DNA from dying breast cells.

“This study supports the potential of an entirely new approach to identifying cancer at its earliest stages when therapies may be most effective,” Dr. William Mitchell of Vanderbilt University School of Medicine in Tennessee, who worked on the study, said in a statement.

SCREENING AND MONITORING

“Laboratory tests using this approach may have the potential both to screen large populations for cancer before symptoms appear and to monitor patients for the recurrence of cancer once treated,” Mitchell added.

Much more testing needs to be done, Urnovitz said. But so far the test seems to specifically home in on breast cells. Unpublished data shows, for instance, that the DNA signature is not found in men with prostate cancer.

The cost of genetic sequencing will have to come down more before the test would be practical, Urnovitz added.

His team used Roche AG’s 454 sequencer at a cost of thousands of dollars per person, but companies are working to speed up sequencing and get the costs down.

The tests might be used to screen women for breast cancer and to tailor treatments, Urnovitz said.

“Imagine we can come in and say ‘you have damage to the protein kinase gene that would preclude you from these 10 cancer drugs, but here are 20 others that should work’,” he said.

“You would be selecting drug treatment based on each person’s lesions. This would be a really good example of personalized medicine.”

Urnovitz also hopes such a test could monitor patients who have completed treatment for cancer. Instead of coming to a cancer center to undergo a PET scan to check for tumors that may have returned, patients could get a blood test at their convenience and have it sent in for analysis.

“You could have one blood test for everything that is going on,” he said.

(Editing by Paul Simao)

Stem cells delay paralyzing disease

Stem cells delay paralyzing disease
Mon Oct 16, 2006 3:08 PM ET

By Maggie Fox, Health and Science Editor

WASHINGTON (Reuters) – Human fetal stem cells can graft onto the spines of rats and delay some of the paralyzing symptoms of motor neuron disease, commonly known as Lou Gehrig’s disease, U.S. researchers reported on Monday.

The new cells were resistant to the disease, also known as amyotrophic lateral sclerosis or ALS, the researchers said.

A company associated with the researchers is incubating batches of the human cells, taken from an aborted fetus, and hopes to market them as a treatment for several sorts of paralyzing conditions.

“We were extremely surprised to see that the grafted stem cells were not negatively affected by the degenerating cells around them, as many feared introducing healthy cells into a diseased environment would only kill them,” said Dr. Vassilis Koliatsos of Johns Hopkins University, who helped lead the study.

The researchers, who published their findings in the journal Transplantation, used specially bred rats that always develop symptoms of ALS and die. Like people with the disease, they gradually become paralyzed until they suffocate when breathing muscles stop working.

There is no cure for ALS and the causes are not clear. But the Johns Hopkins team wanted to see if grafting new cells into the body might help preserve some muscle function.

They used cells taken from an 8-week-old fetus, which had been donated by the mother after an abortion. Stem cells are partially developed but can give rise to a variety of different tissues if put into the right environment.

The cells from the aborted fetus are not the same as embryonic stem cells, currently at the center of a political debate in the United States. Fetal stem cells are intermediate between embryonic stem cells and so-called adult stem cells.

 
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They are somewhat more malleable than adult stem cells but not as plastic as embryonic stem cells.

In this case, the researchers took the cells from the developing spine. These cells are already destined to become nervous system tissue and do not elicit an immune rejection by the body, said Karl Johe, chairman and chief scientific officer of Neuralstem Inc. in Rockville, Maryland.

NO CURE

The researchers only transplanted cells into the lower spinal cords of the rats, in part because the animals are so tiny and the job is tricky, said Johe. Because the upper spinal cord controls the upper half of the body including breathing, there was no chance of curing the rats.

“They do develop symptoms and also they still die, but the onset is more slowly developing and the longevity is extended,” Johe said in a telephone interview.

They injected the human fetal stem cells into adult rats not yet showing symptoms and also killed some of the stem cells and injected them into other rats to act as a control.

On average, the rats treated with live stem cells started losing weight — one of the first symptoms of disease — after 59 days and they lived for 86 days. In contrast, the rats given the sham treatment started to lose weight at 52 days and only lived for 75 days.

While all the rats grew steadily weaker, the treated rats maintained their ability to crawl up a slope for much longer than untreated rats.

After the rats died the researchers examined their spines and saw that 70 percent of the transplanted cells had developed into nerve cells.

Johe said the company was growing and nurturing the cells and hoped to create many batches of purified cells that could be used for transplants for a range of patients with spinal cord diseases or injuries.

“If we see in a year … really significant effects (in rats) then I think we could be ready for a (human) clinical trial in another year after that,” Johe said

Stem cells delay paralyzing disease

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Stem cells delay paralyzing disease
Mon Oct 16, 2006 3:08 PM ET

By Maggie Fox, Health and Science Editor

WASHINGTON (Reuters) – Human fetal stem cells can graft onto the spines of rats and delay some of the paralyzing symptoms of motor neuron disease, commonly known as Lou Gehrig’s disease, U.S. researchers reported on Monday.

The new cells were resistant to the disease, also known as amyotrophic lateral sclerosis or ALS, the researchers said.

A company associated with the researchers is incubating batches of the human cells, taken from an aborted fetus, and hopes to market them as a treatment for several sorts of paralyzing conditions.

“We were extremely surprised to see that the grafted stem cells were not negatively affected by the degenerating cells around them, as many feared introducing healthy cells into a diseased environment would only kill them,” said Dr. Vassilis Koliatsos of Johns Hopkins University, who helped lead the study.

The researchers, who published their findings in the journal Transplantation, used specially bred rats that always develop symptoms of ALS and die. Like people with the disease, they gradually become paralyzed until they suffocate when breathing muscles stop working.

There is no cure for ALS and the causes are not clear. But the Johns Hopkins team wanted to see if grafting new cells into the body might help preserve some muscle function.

They used cells taken from an 8-week-old fetus, which had been donated by the mother after an abortion. Stem cells are partially developed but can give rise to a variety of different tissues if put into the right environment.

The cells from the aborted fetus are not the same as embryonic stem cells, currently at the center of a political debate in the United States. Fetal stem cells are intermediate between embryonic stem cells and so-called adult stem cells.

 

They are somewhat more malleable than adult stem cells but not as plastic as embryonic stem cells.

In this case, the researchers took the cells from the developing spine. These cells are already destined to become nervous system tissue and do not elicit an immune rejection by the body, said Karl Johe, chairman and chief scientific officer of Neuralstem Inc. in Rockville, Maryland.

NO CURE

The researchers only transplanted cells into the lower spinal cords of the rats, in part because the animals are so tiny and the job is tricky, said Johe. Because the upper spinal cord controls the upper half of the body including breathing, there was no chance of curing the rats.

“They do develop symptoms and also they still die, but the onset is more slowly developing and the longevity is extended,” Johe said in a telephone interview.

They injected the human fetal stem cells into adult rats not yet showing symptoms and also killed some of the stem cells and injected them into other rats to act as a control.

On average, the rats treated with live stem cells started losing weight — one of the first symptoms of disease — after 59 days and they lived for 86 days. In contrast, the rats given the sham treatment started to lose weight at 52 days and only lived for 75 days.

While all the rats grew steadily weaker, the treated rats maintained their ability to crawl up a slope for much longer than untreated rats.

After the rats died the researchers examined their spines and saw that 70 percent of the transplanted cells had developed into nerve cells.

Johe said the company was growing and nurturing the cells and hoped to create many batches of purified cells that could be used for transplants for a range of patients with spinal cord diseases or injuries.

“If we see in a year … really significant effects (in rats) then I think we could be ready for a (human) clinical trial in another year after that,” Johe said.