Perhaps you’ve seen the story of the 44-year-old American man reportedly “cured” of HIV in Germany–it’s been making the rounds over the past week. What’s actually happening here?

The Procedure

This is a story that dates back a few years; in fact, 80beats blogged about this case years ago when it first made the news. Back in 2007, the man—Timothy Ray Brown—was an HIV-positive patient suffering from acute myeloid leukemia. When standard chemotherapy couldn’t help him, his docs in Germany turned to a bone marrow transplant, with one twist.

Brown’s oncologist decided to look for a bone marrow donor who had a had a special genetic mutation that made the stem cells in it naturally resistant to HIV infection. His physician, Dr. Gero Huetter, was able to find this rare match and Brown got the bone marrow transplant.  He needed a second stem cell transplant because the cancer came back. Today, he appears to be cancer free and doctors can’t find traces of the virus that causes AIDS either. [CNN]

Brown’s treatment made a splash in the news in 2008, when the doctors first reported on it. It has resurfaced this month because the researchers published a new study in the journal Blood updating his condition.

The researchers confirmed that Brown seems to have maintained his resistance to HIV for three years, confounding their expectation that he would become reinfected. They concluded that a “cure of HIV has been achieved in this patient.” [New Scientist]

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In an attempt to find a cure for type 1 diabetes, researchers are looking to an unlikely source: testicles. In a new study, researchers extracted stem cells drawn from human testes and reprogrammed them to produce insulin. When implanted into diabetic mice, the altered cells brought down the mice’s blood glucose levels, temporarily curing their diabetes.

In type 1 diabetes, the body’s immune system mistakenly destroys the pancreatic beta and alpha cells, which regulate blood glucose levels. Without the insulin created by their beta cells, diabetics experience high glucose levels that cause serious health problems.

Lead researcher G. Ian Gallicano and his colleagues took human spermatogonial stem cells–precursor cells that give rise to sperm–and reverted them to an embryonic state. Then the researchers coaxed them to develop into insulin-producing cells that resemble beta cells. Finally, they injected these pseudo-beta cells into the pancreases of mice with type 1 diabetes.

In a presentation on Sunday at the American Society for Cell Biology annual meeting, Gallicano said the graft was able to produce enough insulin to “cure” the mice of their diabetes for a week, though insulin levels were not high enough to treat humans this way.

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This week in bizarre new forms of mammal reproduction: mice who have genetic material from two fathers but nary a mother, the next step in a progression of scientific efforts to get more creative with sex and reproduction.

“It has been a weird project, but we wanted to see if it could be done” in mice, says Richard Behringer, lead author of the study and a developmental geneticist at M.D. Anderson in Houston. [Wall Street Journal]

Weird, and also complex: The process requires several generations and some creative genetic trickery. To make it happen, Behringer’s team started with a single male mouse. Let’s call him Fred. Scientists took cells from Fred and transformed them into a line of induced pluripotent stem cells, which can grow into any kind of cell in the body. Normally, of course, a male’s sex chromosomes are X and Y. But when the researchers created these stem cells, some of them—about 1 percent—lost the Y chromosome through ordinary mistakes that happen in cell division.

Thus, the scientists had a batch of Fred-derived stem cells that had no Y, and thus were labeled XO cells. The next step was to take ordinary mice blastocysts—early stage embryos that had been conceived in the traditional fashion—and inject the XO cells into them. When this XO-injected embryo was implanted into a normal female mouse, she gave birth to offspring called chimera—what we call animals with two or more genetically distinct populations of cells. In this case the mouse possessed, in addition to the normal cells from its mother and father, some XO cells derived from Fred.

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Around two million people die each year from TB, and the bacterial infection is startlingly widespread—the World Health Organization says about one in three people around the world carry Mycobacterium tuberculosis (and humans may have been carrying it around for at least 9,000 years). Thankfully, TB is latent in the vast majority of these cases. But tuberculosis’ pervasiveness presents the question of just how the bacteria evades our immune system to set up shop on a long-term basis.

According to a study led by Gobardhan Das in the Proceedings of the National Academy of Sciences, stem cells might be the answer. Particularly, mesenchymal stem cells (MSC).

TB recruits mesenchymal stem cells to the lungs, where they help suppress the immune system that fights disease… The stem cells produce nitric oxide, a chemical that reduces the type of white blood cells called T-cells, the researchers wrote. [Bloomberg]

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In recent weeks we’ve covered new experimental treatments that involve injecting stem cells into patients to treat conditions like stroke and spinal injury. But in a new study, British researchers have pinpointed the possibility—in rats at least—of stimulating the body’s own stem cells to repair the chronic damage brought on by multiple sclerosis.

In MS patients, the immune system mistakenly attacks what’s called the myelin sheath, the protective layer around the axons of nerve cells.

The loss of myelin in MS sufferers leads to damage to the nerve fibres in the brain that send messages to other parts of the body, leading to symptoms ranging from mild numbness to crippling paralysis. [AFP]

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Embryonic stem cell treatments are finally breaking out of the lab and arriving in the clinic. In October, the first federally approved trial of a treatment derived from these controversial cells got underway in patients with spinal cord injuries. Now, the FDA has approved a second trial, this one to test a treatment for a rare disease that causes serious vision loss or blindness.

The company behind the trial, Advanced Cell Technology, will test the safety and efficacy of the treatment on 12 patients.

The trial will examine the safety of a therapy for Stargardt’s Macular Degeneration, an inherited juvenile eye disease affecting an estimated 1 in 10,000 young people in the US. As the disease progresses, a layer of the retina called the retinal pigment epithelium (RPE) degenerates, causing vision loss. It’s hoped the new therapy would also work for other types of macular degeneration, a widespread cause of blindness, particularly in the elderly. [Nature blog]

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The United States is still bogged down in uncertainty over which stem cell science the government can and can’t fund, but that doesn’t mean the march of research has ground to a halt. This week brought news of two new human stem cell treatments that are going forward.

In Britain, a former truck driver in his 60s who suffered a stroke has now become the first person to receive an experimental stem cell treatment for the condition. Doctors injected two million fetal stem cells developed by British company ReNeuron into his brain with the hope of stimulating the growth of brain cells and blood vessels.

The patient received a very low dose of stem cells in an initial trial to assess the safety of the procedure. Over the next year, up to 12 more patients will be given progressively higher doses – again primarily to assess safety – but doctors will be looking closely to see if the stem cells have begun to repair their brains and if their condition has improved. [BBC News]

If those treatments go well, those higher doses could go as high as 10 to 20 million cells. ReNeuron scientist John Sinden explains that the fetal cells were taken from a 12-week-old fetus, and were already destined to become brain cells. This treatment is thought to have fewer uncertainties than those that use embryonic stem cells, which can grow into any type of cell and which can sometimes cause tumors.

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Injecting stem cells into injured mouse muscle not only helped the muscle heal, but gave the mice enhanced muscle mass for years to come.

The study, published in Science Translational Medicine, used skeletal muscle stem cells from young donor mice and injected them into injured muscles of mature mice. Researchers figured that the stem cells would be able to create new muscle cells in the recipient mouse, but the question was: could these new cells be incorporated into the existing muscle on an adult mouse?

After injuring the recipient mouse’s muscle and injecting the cells, the researchers noticed that the injury healed quickly and the mice had larger muscles (about twice the volume, and a 50 percent increase in mass) than before the injury, which they expected. But were surprised to see that the muscle enhancement was sustained throughout the recipient mouse’s lifetime, up to two years.

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It may not be as miraculous as turning water into wine, or as wealth-generating as turning dirt into gold, but we still think this is a very cool trick: Researchers have transformed mature skin cells directly into mature blood cells. Crucially, this was done without reverting the cells to a flexible, “pluripotent” stage in which the cells can grow into any form.

The technique, described in Nature, could lead to lab-grown blood cells for transfusions and transplants for people with bone marrow diseases. Researchers think this new process may be safer than previous methods.

By skipping the pluripotent step, the researchers believe they have skirted the risk that the replacement cells might form dangerous tumors. [Los Angeles Times]

The research team, lead by Mickie Bhatia, coaxed the skin cells into becoming blood cells via a harmless virus that carried a gene called OCT4 into the cells–this reprogrammed the cells, turning their developmental clock back part of the way. Then the cells were incubated in a mixture of cell-stimulating proteins, called cytokines, which directed them on their new paths as either red blood cells, white blood cells, or platelets. The ability to grow a specific kind of blood cell has exciting clinical possibilities.

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Yes, it’s a milestone. The first federally approved trial of an embryonic stem cell therapy has begun, and the first patient with a spinal cord injury has been injected with a treatment made from embryonic stem cells. But if all goes well with the trial, conducted by the biotech company Geron, there won’t be any dramatic results–the trial is simply intended to test the treatment’s safety, and patients will receive very low doses of the stem cell concoction.

The patient, whose name was not disclosed, is enrolled at the Shepherd Center, a rehabilitation center in Atlanta; the company has said it plans to enroll 8 to 10 patients in the study at sites around the country. Even if all goes well in the early-stage study, the treatment faces many years of testing for effectiveness before it could be approved as a therapy for spinal injuries. [New York Times]

For the trial, which gained final approval in August, Geron is working with a technique pioneered by the neurobiologist Hans Keirstead, who licensed the technology to Geron.

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UPDATE: In a terse statement (pdf) released today—Thursday, Sept. 9—the U.S. Court of Appeals has issued a stay on the ruling of District Court judge Royce Lamberth against federal funding for embryonic stem cell research. Earlier in the week, Lamberth refused to grant a stay on his own ruling; see below. This doesn’t mean the appeals court is siding with the Justice Department against Lamberth’s ruling; it simply means the three judges want adequate time to consider the ruling and its repercussions. And because the stay lasts until September 20 at least, the National Institutes of Health can re-up the $54 million in projects currently due for annual renewal.

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The ruling stands, for now.

The Obama Administration asked U.S. District Court judge Royce Lamberth to grant a stay of his injunction against federal funding for embryonic stem cell research, insisting that it could trigger job losses and research setbacks. But yesterday the judge issued an order in which he refused to lift the ban, and dismissed the Justice Department’s arguments that the ruling would cause chaos by immediately shutting down all research.

Lamberth indicated that his injunction was less restrictive than had been interpreted by the Obama administration. “Defendants are incorrect about much of their ‘parade of horribles’ that will supposedly result from this Court’s preliminary injunction,” Lamberth wrote. The ruling did not necessarily apply to research that had been funded under guidelines issued during the Bush administration or that had previously been “awarded and funded,” Lamberth wrote. [Washington Post]

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The U.S. Department of Justice has now officially asked Royce Lamberth, the District Court judge who ruled that the Obama administration’s expansion of embryonic stem cell research violated federal law, to suspend the injunction he issued last week that prevents any more funds from going to stem cell projects. The DOJ is also taking the case to the Court of Appeals.

In a 23-page legal filing, Justice Department lawyers said the stay was needed to avoid terminating research projects midstream and negating years of scientific progress toward finding new treatments for devastating illnesses. The department said the ruling would cause irrevocable harm to “millions of extremely sick or injured people” who could benefit from stem-cell research, as well to scientists and taxpayers “who have already spent hundreds of millions of dollars on such research through public funding of projects which will now be forced to shut down and, in many cases, scrapped altogether.” [Wall Street Journal]

Most ongoing projects had been allowed to continue for now, but only if they used National Institutes of Health money to research at their home universities. However, NIH head Francis Collins notes (pdf) the $54 million in projects due for renewal at the end of this month—without a change in the ruling, NIH is forbidden to renew them. Additionally, projects underway on the NIH campus itself have been ordered shut down.

DISCOVER will keep you posted on further updates.

Related Content:
80beats: Stem Cell Decision Fallout: What’s Next, and Who Were the Plaintiffs?
80beats: Judge: Obama’s Expansion of Stem Cell Research Violates Federal Law
80beats: The Trouble With Lab-Created Stem Cells—and Why They Won’t Displace Embryonic Ones
80beats: FDA Green-Lights First Trials Using Embryonic Stem Cells (Again)

Image: iStockphoto

Stem cell work will go on. But the shape of its long-term future is a mystery.

A court ruling yesterday that said the federal government can’t fund embryonic stem cell research even if no money goes to destroying embryos has thrown the field into confusion. Today, though, NIH head Francis Collins says that while the government can’t fund new projects (at least until the legal dispute is resolved), researchers in the middle of federally funded projects can continue.

Yes, you saw a similar headline in 80beats in January of 2009, but this time we mean it. We think.

Back in 2009 the FDA approved an application from Geron Corporation to begin the first human safety trials of a therapy derived from embryonic stem cells, a move that was heralded as a strong vote of confidence in this controversial but exciting area of medicine. But before the treatment of patients with spinal cord injuries could begin, the FDA reversed course and put a hold on the trial, noting that Geron had discovered cysts in some rats injected with the cells.

Since then, Geron has been scrambling to prove that its treatment is safe via new animal studies, and has agreed to change some procedures to minimize the likelihood of cyst formation. Now that the FDA has signed off, Geron expects to begin the small safety trial (involving only 7 to 10 patients) before the year’s end.

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Step 1: Take a rat lung. Step 2: Strip away all of its living cells, leaving only a fibrous “scaffold” of connective tissue. Step 3: Bathe the scaffold in lung cells taken from newborn rats, and put the whole thing in a bioreactor to let the cells multiply and spread. Step 4: A few days later, when the reconstructed lung is again filled with blood vessels and alveoli, transplant the organ into a living rat. Step 5: Watch in awe as the lung begins to function.

That’s the short version of the experiment Yale University researchers just published in Science. The study was a result of a change in direction for lead researcher Laura Niklason:

Niklason spent several years trying to create a synthetic lung scaffold, but in the end concluded it was too difficult. “I decided I couldn’t do it, and probably nobody else could either,” she said. [National Geographic]

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