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Today's Top Stories
1.
Alzheimer's, diabetes may be connected by a single gene
2.
Human Microbiome Project maps healthy body microbes
3.
Pluristem's placental stem cells spur bone marrow growth
4.
After death, some stem cells remain alive for days
5.
Genetic markers uncovered for rare children's brain tumor
Editor's Corner:
In life sciences R&D, China is reaching for the stars
Also Noted: Spotlight On...
Stem cell pioneer Shinya Yamanaka splits $1.5M Millennium Technology Prize
Fruit fly gene will aid quest for new cancer drugs; Early-stage eye precursor grown in the lab;
and much more...
News From the Fierce Network:
1.
With targeted cancer drugs, cost vs. benefits gets more complicated
2.
Should FDA's standards enforcement be blamed for drug shortages?
3.
Stanford spinoff debuts cloud-based R&D software
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In life sciences R&D, China is reaching for the stars
 At the long-awaited kickoff of the 2012 BIO International Convention in Boston on June 18, many of the initial sessions weren't exactly crowded. The same can't be said, however, for the all-day session on China.
At the morning part of the session, panelists represented different facets of China's increasingly sophisticated life sciences drug discovery and development operations. The crowd very quickly became standing-room only. Clearly, China is where it's at these days, even more so than before.
What was interesting to me was that a nation that can be very modest about expressing its ambitions actually laid them out pretty clearly. You've probably heard about China's goal to build a space station by 2020? Well, the world's most populous nation has the same scope of ambition for its drug discovery and development work, explained Ying Luo, a panelist and chairman of the Beijing Continent Pharmaceutical Co. Fair enough, and the various facts you could glean from their presentations brought the point home.
Among the highlights:
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Life science researchers at Chinese universities are increasingly forming collaborations with multinational corporations and Chinese biotechs. The Beijing Cancer Hospital, for example, is working with WuXi AppTech (a massive Chinese CRO) to develop new cancer cell lines for research, noted Edward Hu, WuXi's CFO/COO.
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We've all heard about major life sciences research coming from places in the U.S. such as Harvard University, the Sloan-Kettering Cancer Center and the Scripps Research Institute. We'll see tomorrow's big research generated from places in China like the China Academy of Science, Peking University and Tsinghua University. Top research talent with major government financial support is based at those and many other academic institutions in China, said Zegon Zhang, a deputy director of the Beijing Pharma and Biotech Center, a nonprofit government agency. (We told you about China's research surge back in April.)
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Domestic Chinese biotechs are aggressively investing in R&D and do that, in part, by leveraging government support, Hu said.
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Drug discovery work in China costs as little as one-third to one-half the cost in the U.S., according to Ying Luo of Beijing Continent Pharmaceutical. Pre-IND work, for example, can cost as little as $600,000 to $1 million, he said.
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China is developing a small army of experienced scientists. WuXi, for example, employs 6,000 people globally, with more than 5,000 of its scientific cadre based in China. And the panel explained that the country continues to train scientists at its universities, or welcome back residents after they earn a Ph.D. abroad.
-- Mark Hollmer (Twitter | email)
Read more about: life sciences, BIO
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Today's Top News
1.
Alzheimer's, diabetes may be connected by a single gene
A single gene connected to Alzheimer's disease may also link to diabetes, researchers at The City College of New York believe. Though an early finding, the researchers believe it could offer scientists new drug targets and other treatment options they haven't had before for both diseases.
Their study considered how the human "amyloid precursor protein" (APP) gene is a factor in both conditions. And they started based on some circumstantial evidence. The APP gene, which processes amyloid precursor protein, appears in Alzheimer's disease patients where the condition runs in families. Similarly, they also know that people with diabetes appear to have a greater risk of also developing Alzheimer's.
Studies of the APL-1 gene in the Caenorhabditis elegans (C.elegans) worm, which is similar to the APP gene in people, helped them make the connection between the two. What they found: A mutated APL-1 gene slowed the worms' development, and scientists determined that it specifically blocked the insulin pathway, leading to their hypothesis that the human variation of the gene affects both Alzheimer's and diabetes. Interestingly, mutating the insulin pathway even more helped reverse the mutation of the APL-1 (APP equivalent) gene mutation.
This is an early finding, of course, showing only one possible link to both diseases. And there are plenty of variables. For example, the team must still determine how the amyloid precursor protein intersects with signal neurons and other cells along the insulin pathway. Further testing over many years will be necessary to make sure the discovery bears out in people. But it is a promising first step. Further details are published in the journal Genetics.
- here's the release
Related Articles:
Lilly takes aim at blockbuster Lantus with promising PhII diabetes drug data
GE Healthcare joins Australian government on Alzheimer's Dx study
Potential Alzheimer's treatment disrupts amyloid protein formation
Read more about: Diabetes, amyloid precursos protein
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2.
Human Microbiome Project maps healthy body microbes
The Human Microbiome Project, a 5-year, $173 million enterprise, has produced a major breakthrough: A detailed genetic map of microbes that live in a healthy human body or on it. An army of 200 researchers at 80 different institutions took part in the National Institutes of Health-funded endeavor, The Wall Street Journal reports.
Details are published in Nature and also the Public Library of Science.
While there is more work to be done, the scientists believe that their effort could help advance research in general but in particular the diagnosis of disease and the quest to find new ways to treat it. (Not coincidentally, microbiome startups are beginning to grow in number.) As the story notes, scientists have long known that the body carries "trillions of microorganisms" but didn't always know what they were, where they were located or how they could vary between individuals and body locations. Knowing that information should help researchers who are trying to figure out how to treat diseases caused by genetics, combined with a change in the bacteria the body carries, according to the article. For example, doctors could possibly fight obesity knowing better which gut microbes regulate fat digestion.
Researchers drew samples from the mouth, nose, skin, intestine, vagina and other parts of the body from 242 healthy volunteers in the U.S., according to the article. Once obtained, they used advanced versions of the DNA-sequencing machines put to work in the Human Genome Project to map DNA. What they found: A human microbiome contains more than 8 million genes. Also, a body carries more than 10,000 different microbe species. Future work will involve analyzing microbiomes from children and senior citizens, people with disease and individuals from Africa and South America.
- here's the Wall Street Journal article (sub. req)
- check out the Nature paper
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Read more about: Human Microbiome Project, microbes
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3.
Pluristem's placental stem cells spur bone marrow growth
Pluristem Therapeutics' ($PSTI) special stem cells drawn from discarded placentas have shown major promise in a preclinical study. As the company announced today, they spurred hematopoietic stem cells to grow bone marrow in mice after their injection into muscle.
Being that this is the big week for the 2012 Bio International Convention in Boston, the Israeli company saved the announcement for the industry megagathering. FierceBiotechResearch spoke with Pluristem Chairman and CEO Zami Aberman and William Prather, the company's senior vice president of corporate development, about the big news. They emphasized that they see the performance of their Placental eXpanded cells, or PLX, as a major advance in spurring the body's stem cells into regenerative work, in part because injecting the treatment into the muscle appeared to generate a stronger systemic response than giving the treatment intravenously, which is a more common route for stem cell treatment tests.
And such a treatment, if it ultimately gains regulatory approval, would be a better way to deliver such a treatment because muscle injections are easier to track and measure, Aberman told me.
"When injected into muscles you know where [the cells] are," Aberman said during an interview across from Israel's booth in the hangar-like exhibit hall. He noted that intravenous injections can't be tracked well through the body to determine their effectiveness or how long they last.
The study involved irradiating mice to kill their bone marrow development and then injecting the PLX treatment intramuscularly. Pluristem found that the method of treatment stimulated the hematopoietic stem cells in bone marrow to produce red cells, white cells and platelets. The treatment boosted mouse survival time and increased how many bone marrow cells survived.The hope is to use the treatment eventually to treat blood disorders as well as primary and secondary bone marrow failure that can take place after radiation sickness or chemotherapy complications.
Just last month, Pluristem announced that it had used the treatment in a 7-year-old girl who was suffering from a bone marrow disorder, and that the treatment helped boost both red and white blood cell levels. The stem cells have also been tested in two Phase I/II safety/dosing clinical trials in the U.S. and Germany, for patients with critical limb ischemia, showing enough promise to warrant further trials.
- read the release
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Stem cell therapy rescues child with marrow disease
FDA gives orphan OK to Pluristem's delivery platform for Buerger's disease
Read more about: Stem Cells
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4.
After death, some stem cells remain alive for days
By becoming dormant, skeletal muscle stem cells can survive in a human body after a person dies, for a good 17 days after the fact. During that time, it turns out, they're still viable enough to be revived and then subdivide into workable cells, Fabrice Chretien of the Pasteur Institute in Paris and his colleagues have discovered. Their finding, published in the journal Nature Communications, could someday allow for patients to donate their stem cells, post-mortem, for use as a possible tissue rebuilding treatment.
Previously, these kind of stem cells were thought to survive for no more than a day or two, but the research team's data on the survival skills of human skeletal stem cells in a dead body explodes that notion. The same thing goes for mice: The researchers determined that these same kind of stem cells remain alive in rodents for about 16 days, the Daily Mail, Agence France Press, and others reported. Separately, they learned that bone marrow stem cells in mice survived for about four days after death and could still rebuild tissue in the wake of a bone marrow transplant.
The key to remaining viable: The skeletal stem cells slowed their metabolism until they became dormant, allowing them to conserve vital energy even as the body in which they are housed dies around them.
All of this is promising of course, but it could be years before the viability of stem cells harvested from dead individuals can be tested in human trials. The researchers themselves offer the disclaimer, noting that much more testing will be needed. But if it works, the researchers believe the method of harvesting stem cells from patients after they die (with their consent, of course) could help address shortages of tissues and stem cells harvested through more traditional means.
- here's the journal abstract
- read the Daily Mail story
- check out the AFP piece
- read additional coverage here
Related Articles:
Special Report: Stem cell research advances in fits and starts
Nanotech morphed stem cells into bone cells on command
Scientist uses stem cells to rebuild bone
Read more about: skeletal muscle stem cells, post-mortem
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5.
Genetic markers uncovered for rare children's brain tumor
Central nervous system primitive neuroectodermal brain tumors (CNS PNET for short) are a particularly rare but nasty form of cancer that leaves patients with a poor prognosis, particularly when very young children are stricken. But scientists at The University of Nottingham in the U.K. have identified genetic markers that they think will lead to vital new diagnostics for the condition.
While that advance may be a long way into the future, the researchers believe that those eventual diagnostics could help researchers improve the kinds of drugs and therapies on hand to treat the cancer. While such advances are likely many years away, more treatment options for CNS PNET are long overdue. The researchers note that high-dose chemotherapy and cranio-spinal radiotherapy are two of the primary options at hand right now, but they often don't work and leave patients with long-term side effects.
Details are published in the latest journal Lancet Oncology.
In collaboration with the Hospital for Sick Kids in Toronto, they conducted their study using 142 samples of CNS PNET collected from 20 different institutions in 9 countries, with a focus on the tumor genetics. Their discovery: The tumors have three subtypes--each with particular genetic mutations that give patients different outcomes. And each group had a separate genetic signature based on variations in the way they expressed the LIN28 and OLIG2 genetic markers.
- read the release
- check out the journal article summary
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Glioma peptide vaccine shows early hints of efficacy
Engineered measles virus prolongs lifespan of brain cancer-stricken mice
Read more about: central nervous system primitive neuro-ectodermal brain tumors, CNS PNET, brain tumors
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Also Noted
SPOTLIGHT ON...
Stem cell pioneer Shinya Yamanaka splits $1.5M Millennium Technology Prize
Stem cell research pioneer Shinya Yamanaka is sharing this year's $1.5 million Millennium Technology Prize with Linus Torvalds, the creator of Linux. The Technology Academy Finland gave the award in Helsinki on June 13 in recognition of "life-enhancing technical innovations," Wired reports. Yamanaka is known for discovering a new way to develop pluripotent stem cells for medical research. Story
Cancer
> Scientists at the Kimmel Cancer Center at Thomas Jefferson University in Philadelphia have determined a linking between aging of normal cells and the growth of cancerous tumors and their subsequent metastasis. Release
> Scientists at Loyola University in Illinois have identified a fruit fly gene that they believe can help in the development of new cancer drugs. Release
Stem Cells
> Yoshiki Sasai of the Riken Center for Developmental Biology in Kobe, Japan, has grown the precursor of a human eye in the lab, using embryonic stem cells, Nature reports. Story
> Stem cell researchers are urging the European parliament not to cut off funding for human embryonic stem cell research in response to anti-abortion protestors who want support for the field to stop. Story
Genetics
> An international team of researchers based in the U.K. reveal they've determined a way to sequence genomes of Plasmodium falciparum, the deadliest and most common form of malaria, using patient blood samples, National Public Radio reports. Story
> The European Society of Human Genetics has condemned the use of genetic testing to establish "racial purity," based on an incident in Hungary. Release
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