The Cure for AIDS?

Story first appeared in ABC News.
The fact that a middle-aged  man is a reasonably healthy 46-year-old is no small thing. Only a few years ago, he had AIDS.

He is currently the only person in the world to be cured of AIDS, the result of a transplant of blood stem cells he received to treat leukemia.

Luck was on his side, the blood stem cells he received came from a donor with a special genetic mutation that made him resistant to HIV. The genetic mutation occurs in less than 1  percent of Caucasians, and far less frequently in people of other races. Before he cgot his transplant in 2007, doctors tested nearly 70 donors for this genetic mutation before they found one who was a match.

But doctors hope that a similar solution could help other people with HIV: umbilical cord blood transplants.

A medical director of StemCyte, an umbilical cord blood bank, said although the patient was cured by his transplant, the process was complicated because the blood stem cells came from an adult donor. When you do that you have to have a very close match between donor and recipient. With umbilical cord blood, you don’t need such a close match. It’s far easier to find donor matches.

But it’s still not that easy, the blood bank has tested 17,000 samples of cord blood so far, and found just 102 that have the genetic HIV-resistant mutation. The team performed the first cord blood transplant on an HIV-infected patient a few weeks ago, and they have another transplant planned for a similar patient in Madrid, Spain,  later this year. It will still be months before researchers can tell if the transplants have any effect on the patients’ HIV.

The doctor also noted that transplants aren’t performed solely to treat AIDS. Patients who get them have an additional condition that requires a blood stem cell transplant. Curing their AIDS would be an incredible bonus.

The patient had his transplant in February 2007. Today, his body shows no signs of the virus. He feels guilty being the only person to have been cured of the virus when millions still live with it. But he hopes his story will inspire others that a cure is possible.

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Move Over Stem Cells, Here Come MicroRNA

Story first appeared on Fox News.

When a person suffers a heart attack, scar tissue forms over the damaged areas of the heart, reducing the organ’s function.  However, in a recent study, scientists successfully turned this scar tissue into working heart muscle without the use of stem cells, say Livonia Cardiologists.

Duke University researchers used molecules called microRNAs to convert scar tissue (called fibroblasts) into heart muscle cells in a living mouse, improving the heart’s ability to pump blood.  According to the scientists, this process is much simpler than stem cell transplants and has none of the ethical concerns, making it a potential turning point in the science of tissue regeneration.

Right now, there’s no good evidence stem cells can do the job.

Scientists believe embryonic stem cells are the best to use for tissue regeneration because they are pluripotent—meaning they can become any type of cell in the body.  However, there have not been enough experiments done to prove how functional the stem cells are in regenerating tissues and whether or not they may form deadly tumors. Additionally, there are ethical concerns about using cells derived from a human embryo.

Meanwhile, adult stem cells avoid the controversy surrounding embryonic stem cells but have a limited capacity to form other types of cells.  The results of using these adult stem cells for tissue regeneration are not as satisfying as one would like.

Rather than stem cells, the new method uses microRNA molecules—which typically control gene activity—and delivers them into the scar tissue that develops after a heart attack.  The microRNAs are able to reprogram, or trick, the scar tissue into becoming heart muscle again instead.  Testing is still in its early stages, but so far, the method appears to be relatively easy, and the data looks very promising, according to the researchers.

It’s a much more simplified, feasible way of causing regeneration; very easy to use as therapy. With stem cells, you have to take them from the embryo or tissue in the body, grow them in culture, and re-inject them—and then there can be technical and biological problems. With microRNA, after a heart attack you can simply convert some of the fibroblasts and tell them to become the right cell type and regenerate.

According to Hamtramck Heart Doctors, this new method also has the potential to treat stroke, spinal cord injuries, chronic conditions such as heart disease—and even the normal damage that can come with aging.  It can feasibly be used for any type of organ in the body, though the process of converting the cells may be different for each organ.

The method must still be tested in then larger animals, and if successful there, it can move onto human clinical trials.  But one could think about all these things of possibilities.  Specialists with Garden City Stroke Care centers are excited about these possibilities. Could you use it to treat the disease of aging and losing brain cells?  Can you convert other cells in the brain to working brain cells?


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Walker wants UW to Focus on Adult Stem Cell Research

Milwaukee Journal-Sentinel

Milwaukee County Executive Scott Walker said Tuesday if elected governor he would shift funding from embryonic stem cell research to adult stem cell research at the University of Wisconsin-Madison.

"I'm going to put the money behind adult stem cells - not embryonic," the Republican told reporters. "The money the state's involved (with), the money that comes from the federal government, should be focused on the greatest potential for success, and that's adult stem cell research."

David Gamm, a stem cell researcher at UW-Madison, called Walker's position "ridiculous" and "not practical" because it would mean researchers would lose millions in federal grants.

"This is basically ignorance," Gamm said. "I would pick up my funding and go somewhere else. I can't give up (nearly) $2 million in grants to stay in Wisconsin."

Walker, who opposes embryonic stem cell research because it destroys embryos, repeatedly refused Tuesday to say whether he supported an outright ban on embryonic stem cell research.

Walker has been endorsed by Pro-Life Wisconsin, however, and that group endorses only candidates who support a ban on embryonic stem cell research.

The university conducts research on both embryonic and adult stem cells and is known internationally as the place where embryonic stem cells were grown for the first time.

About $5 million a year in federal money goes toward embryonic stem cell research at UW.

Adult stem cells are cells that can turn into different cell types but have more limited potential than embryonic stem cells.

Embryonic stem cells have shown promise for curing diseases because they can become all cell types. Reprogrammed adult stem cells also have shown potential as a possible alternative to embryonic stem cells, but there have been some indications that they may not be equivalent. For example, the reprogrammed cells appear to retain a memory of their original cell type.

Walker's Democratic opponent, Milwaukee Mayor Tom Barrett, said in a statement Tuesday that Walker's "ideological views threaten scientific research in our state that can save and improve lives, and they jeopardize significant business opportunities and thousands of jobs in Wisconsin's growing tech economy."

New Method makes Adult Cells Act like Embryonic Cells

USA Today

Stem cell researchers on Thursday reported a new method for reprogramming adult cells into ones that act like more versatile embryonic stem cells, an advance that could open a new avenue for lab-grown transplant tissues.

Stem cells are the building blocks from which replacement cells, everything from blood to bone to brain, grow in early development and throughout life. Researchers consider embryonic stem cells the most useful type for generating replacement tissues, but obtaining them requires the controversial destruction of embryos. Then, in 2006, a Kyoto University team first reported adult skin cells can be "induced" into resembling embryonic stem cells.

That breakthrough relied on viruses infecting cells with four cancer-related genes to spur the transformation. But the new study pulls off the trick with ribonucleic acid (RNA) molecules, normally the workhorses that translate DNA-encoded genes into proteins and perform other housekeeping roles inside cells.

In the journal Cell Stem Cell, a team led by Derrick Rossi of Children's Hospital Boston unveils a cancer-mutation-free method, 40 to 100 times more productive than normal, to create induced stem cells. Induced stem cells are similar to embryonic stem cells in their potential to transform into every type of tissue in the body — but lack the controversy.

"This is very exciting," Rossi says. "We have a new experimental process that can efficiently give us patient cells in clinically useful types."

"RNA, if it works, sounds like a very realistic, promising alternative," says stem cell biologist Gary Stein of the University of Massachusetts Medical School in Worcester, who was not part of the study. Worries about virus contamination and cancer have dogged induced stem cells since their discovery, he notes, along with concerns about their low efficiency rate, with perhaps one cell in 1,000 transforming through the use of viruses.

Treating adult human cells with RNA engineered to overcome immune system defenses reliably creates induced stem cells, the study says. Moreover, the same RNA molecule cued to fire up different genes over three days' time can then be used to turn those induced stem cells into specific types of tissues, such as muscle.

For that reason, "this paper is a major advance in the field of regenerative medicine," says stem cell researcher Douglas Melton of Harvard, who was not part of the study team. Since the isolation of human embryonic stem cells in 1998, researchers have sought ways to turn early stem cells into heart, spine and other tissues needed by transplant patients. The RNA advance means labs can experiment on deriving these tissues in test tubes, he says, speeding progress in the field.

U.S. Can Continue Stem-Cell Funding, Court Says

Bloomberg / BusinessWeek

The U.S. government can continue funding embryonic stem-cell research while it seeks to reverse a lower-court order that barred support to scientists who use the cells, an appeals court said.

The ruling by U.S. Court of Appeals in Washington today puts on hold an order cutting off funding by U.S. District Judge Royce Lamberth, which the government argued would cause irreparable harm to researchers, taxpayers, and scientific progress while the case is appealed.

The government’s lawyers “have satisfied the standards required for a stay pending appeal,” the judges wrote in a one- page order that said the appeal will be expedited and the parties would later be informed the briefing and oral arguments schedule.

Lifting the ban allows the government to temporarily continue funneling tens of millions of dollars to scientists seeking cures for diseases such as Parkinson’s, spinal cord injuries, and genetic conditions. Embryonic stem cells can grow into any kind of tissue and may have the potential to accelerate a range of research.

The decision, coming after oral arguments yesterday, was made by Judith Rogers, appointed by former President Bill Clinton, and Thomas Griffith and Brett Kavanaugh, both appointed by former President George W. Bush.

Geren Corp., which was cleared in July to proceed with the first human test on an embryonic stem-cell therapy aimed at patients with a spinal cord injury, rose 5 percent in after hours trading.

Representaives from the U.S. Justice Department and Thomas Hungar, an attorney for the opponents of the funding, didn’t immediately return calls seeking comment.

Halt in Funding

Lamberth on Aug. 23 issued an order temporarily stopping the Health and Human Services Department and the National Institutes of Health from funding or conducting the studies. On Sept. 7, Lamberth denied a U.S. request to reconsider his ruling. The court of appeals then allowed funding to resume pending today’s decision.

The judge cited the still-in-force 1996 Dickey-Wicker Amendment in his ruling, saying that Congress prohibited funding any research in which a human embryo was destroyed. By implication, that included all stem-cell research, Lamberth said.

In March 2009, President Barrack Obama reversed an executive order of Bush’s to allow research on cells derived from embryos that would otherwise be disposed of after in vitro fertilization procedures.

Under Bush’s order in August 2001, Dickey-Wicker was interpreted to allow research on lines of stem cells that already had been created using human embryos. Bush limited federal funding for such research to about 20 existing lines of embryonic cells and banned funding on any additional lines.

Lamberth’s order prevented the National Institutes of Health from acting on grant applications that have been reviewed, and from considering dozens of other applications that are in the review process, the U.S. wrote in its appeal. It may take as long as eight months to reinitiate the review process for grant applications, the U.S. claimed.

Judge's Ruling sets back Stem Cell Research

San Francisco Chronicle

 

Talk about judicial activism. U.S. District Judge Royce Lamberth just ruled that the federal government can't fund any stem cell research because it destroys embryos. He voided not just President Obama's stem cell policy, but former President George W. Bush's policy, too.

Lamberth relied on a reading of the Dickey-Wicker Amendment, which forbids federal funding of "research in which a human embryo or embryos are destroyed." The anti-abortion community is ecstatic, but the ruling goes far further than even Bush's policies on stem cells. Bush approved federal funding of research on stem cell lines that existed by August 2001. It's unlikely that even this policy would be allowed under Lamberth's reasoning.

The Obama administration is already planning an appeal, as it should. Congress could, and should, easily clarify things by simply passing a stem cell funding bill. That would eliminate the need to go through the courts, and the bill would be unlikely to encounter a presidential veto this time around.

In the meantime, the scientific community is frustrated and confused. The California Institute for Regenerative Medicine will be able to press on with its embryonic stem cell research, thanks to the wisdom of California's voters. But it means the institute's scientists won't be able to collaborate with those whose funding was dependent on federal grants.

Congress should move quickly to ensure that this potentially valuable research can continue.

The Bionic Eye: New Worlds Of Sight For The Blind

NPR

This retinal prosthesis has been implanted in the eyes of 32 patients. 

The device receives wireless data from the camera which it then 

translates into electronic signals that are sent to the brain, restoring sight.

Stem cells and electronics can help restore vision to people who've been blinded by retinal diseases, scientists reported in Chicago at Neuroscience 2009, the annual meeting of the Society for Neuroscience.

Diseases of the retina cause blindness by damaging the cells that line the back of the eye, where images of the world are normally transformed into nerve impulses that go to the brain.

"There's very little therapeutic treatment out there right now for people with diseased retinas," says Brian Mech, a vice president of Second Sight Medical Products in Sylmar, Calif.

But Second Sight is hoping to change that. The company has developed an experimental bionic eye that has been tried in more than 30 patients with macular degeneration or retinitis pigmentosa

Building An Artificial Retina

Each patient wears a pair of glasses that incorporates a video camera, Mech says. The video signal from the camera is sent to an implant on the eye itself, which in turns communicates with an array of electrodes attached to the patient's retina.

And those electrodes do what the old retina can't anymore: send electrical signals to the brain that allow sight.

Mech says it usually takes patients' brains a little while to make sense of the new signals.

"They learn to use the device better over time," he says. "Someone that has had the device for a year will do better than they did at three months."

The artificial eye uses just 60 electrodes to replace millions of retinal cells. Mech says that means the restored vision is rudimentary, so people can find doors and follow lines on the floor. But most can't read, and those who can are only able to make out very large letters.

At the neuroscience meeting, Second Sight presented a study showing that patients could use the artificial eye to tell which direction an object was moving.

Despite the limitations of the artificial eye, Mech says patients who have received one tend to get emotional when they realize they can see even a little bit.

"There's a lot of crying, a lot of smiling," he says. "It's a sensory input that they haven't had in a very long time, and so they're excited."

Growing New Retina Cells

A team led by Robert Aramant of the University of California, Irvine, offered a different approach to restoring sight.

Since the 1980s, Aramant has been working to fix retinas damaged by diseases including macular degeneration and retinitis pigmentosa.

And for several years now, the team has been treating patients using fetal retinal cells. Their approach is to retrieve an intact sheet of fetal retinal cells and transplant the entire sheet into a damaged eye.

The transplanted cells then mature the same way they would in a developing fetus, creating all the layers of a normal retina, Aramant says.

The team has treated just 10 patients so far, because of funding constraints. But Aramant says seven of those patients got better, including one woman whose vision went from 20-800, which is severely impaired, to 20-200, which is good enough for many daily tasks.

After treatment, Aramant says, the woman was able to play computer games, write emails, and read a large-print version of Reader's Digest.

Also at the meeting in Chicago, scientists presented studies showing ways to create new light-sensitive molecules in the eye, and to use stem cells to grow specific types of retinal cells.