Category Archives: Indiana Stem Cells

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Regenerative Cell Therapy in Fort Wayne, Indiana

View print quality imageIn a 2015 file photo, psychology professor Stephen Boehm observes a student researcher in his lab within the Science and Engineering Laboratory Building.Liz Kaye, IU Communications

Four years after its opening, it still has that "new building" smell.

The Science and Engineering Laboratory Building, known to many as SELB, opened in the fall of 2013 to much fanfare from two of IUPUI's essential programs in a STEM world.

The schools of Science and Engineering and Technology at IUPUI had a brand-new, state-of-the-art facility to conduct research at their disposal. On the second floor alone, engineers and scientists from multiple disciplines coexist and collaborate, as their labs are nestled next to one other.

The investigators had a say in the facility's design. They customized layouts and equipment. They were able to move out of buildings and labs that were starting to show their age. They also brought the bands back together.

Description of the following video:

Video transcript

IUUI Researchers Find Collaborations, Breakthroughs in SELB video on https://youtu.be/1T4GondidpQ

[Video: Exterior and interior shots of SELB]

[Words appear: IUPUI presents]

[Words appear: Christopher Lapish, Psychology, Associate Professor]

Christopher speaks: The front of the building is really beautiful, and it has a wide, huge, open atrium with a veranda kinda over it. And the atrium's really nice. We've had talks in there before, lunches quite often. It provides a space for students to engage in collaborative learning or studying in between classes.

And so those spaces are really needed here on campus, and it meets those needs. When I got here, I found that they involved the scientists and the people that would use the building in the design of the building, which was phenomenal. And so the lab that I'm in right now, many of the features that it has, I got to design, and it's really been helpful in our ability to perform science.

This is a really nice soldering iron. Here's some impedance detectors that we use here, pretty standard lab microscope here. We went from a very small, antiquated, old, dusty laboratory, into a state-of-the-art, world-class lab now. I'm really, really excited about that.

[Words appear: Stephen Boehm, Psychology, professor]

Stephen speaks: It was really great when this building opened, first and foremost, because it brought us all back together.

So all the addiction neuroscience faculty are now in the same space. This entire lab that we're sitting in right now is the main wet lab for the entire addiction neuroscience department. So all the faculty have space in this room or in one of the adjacent small rooms that branch off from this room.

And this is where all our molecular and bench-type assays are done in support of all the behavioral work that occurs up on the third floor of this building. We wanted space that could accommodate the work that the current faculty were doing, addiction neuroscience faculty. But we wanted, also, the space to support some of the basic needs of future hires that we though we might go for.

[Video: The Indiana University trident appears]

[Words appear: IUPUI]

[Words appear: Fulfilling the Promise]

[End of transcript]

"All of the addiction neuroscience faculty are now in the same space," said Stephen Boehm, professor of psychology and director for the undergraduate neuroscience program, adding how his departmental colleagues were spread across campus before SELB was built. "This entire lab is the main wet lab for the entire program. This is where all of our molecular and bench-type assays occur in support of all the behavioral work that happens on the third floor of this building."

Boehm shares his lab with associate professor Chris Lapish. They are using rats and mice to study the neurological effects of addiction.

Lapish revealed smaller labs and offices that adjoin the main lab: a maze room that tests the rats' cognizance and addiction levels and a computer room where thousands of data points are crunched from each experiment.

The electrodes that attach to the rats' brains are constructed in the main lab, which is also where the analysis of cellulose membranes takes place.

"You put it in milk," Lapish said. "All the proteins in the milk attach to the membrane. It prepares the blots for them to use to assess protein concentration of different proteins throughout the brain."

On the third floor, the rats and mice are cared for next to a skybridge that connects SELB to the Science Building. The bridge provides a majestic view of downtown Indianapolis. On the south side of the building, Military Park is visible and even audible.

"You can actually hear the music from the Lawn at White River if they're having a concert there," Lapish said.

When Lapish interviewed with IUPUI in 2010, he was lured by the promise of a new building. IUPUI delivered -- and then some.

"They said, 'We're going to build this new building, and if you come, you're going to have a new laboratory,'" Lapish recalled. "I got to design many features of the lab I'm in right now, and it's really been helpful in our ability to perform science.

"We went from a very small, old, dusty laboratory to a state of the art, world-class lab."

On the first floor, multiple open spaces enclosed by glass walls and doors are simply labeled "collaboration." A recent pass saw small groups of students huddled around laptops working on projects. Whiteboards are available for brainstorming and scrawling breakthroughs. Around the corner is a large atrium to promote further interaction among engineering, technology and science minds.

"We've had talks in there, and lunches quite often," Lapish said. "It's a space for students to engage in collaborative learning or studying between classes. Those spaces are really needed here on campus, and it meets those needs."

The east side of the building's exterior pops with colorful windows. The west side helps corral students to the common outdoor areas dubbed the "Science Lawn." Essentially, it's a nice fit.

Jason Meyer, associate professor of biology, has a lab around the corner of Lapish and Boehm's. His work examines human induced pluripotent stem cells for studies of nervous system development and disease. The cells are genetically modified to become any type of cell in the body, and they can serve as a novel platform for studies of neural development, disease progression, drug screening and cellular repopulation.

"What we're interested in is the ability to differentiate these cells into the different kinds of neurons of the retina, the light-sensing part of the eye that allows us to see," Meyer explained. "We're interested in the possible regeneration of the visual system -- can we figure out ways to encourage these cells to regrow and perhaps sometime in the future to develop new therapeutic options for blinding disorders."

The main target for Meyer's current research is the retinal ganglion cell and its long, branchlike shape. These cells connect our eyes to our brain.

Meyer's research has had a comfortable home in SELB, whether he is studying cells in a dish from glaucoma patients or developing new drugs that could rescue stop the progression of visual degeneration to restore some degree of vision. Meyer and his students are able to grow fragile lines of stem cells.

"It takes quite a long time to grow these cells," Meyer said. "To have a dedicated space like this allows us to keep them isolated to keep them away from any kind of contaminants that might damage them or affect their growth.

Collaboration with Meyer's psychology colleagues is much more of a possibility in SELB than in previous locations, he said.

"To have those kinds of connections with neighboring labs and other researchers in those labs really helps facilitate discussions and helps synergize research," he said.

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IUPUI researchers finding collaboration, breakthroughs in lab facility - IU Newsroom

Scott McIntyre calls himself a walking miracle, and he wants to tell the world about it.

I was given three to six months to survive and Im 16 months in remission, said the 53-year-old South Bend man. I would love to get the story out and let people have hope. Dont give up. You never know.

On Friday, a University of Chicago Medicine marketing team shot video and still images of Scott at Shamrock Truck Sales, the semi-truck sales and service business he co-owns near LaPaz. His face will adorn billboards, digital and print ads in Chicagoland and northwest Indiana as soon as the U.S. Food and Drug Administration approves what UCM is calling a revolutionary breakthrough in cancer treatment.

If that FDA approval comes and UCM is preparing for it to come very soon UCM will have one of the only facilities in the Midwest certified to administer chimeric antigen receptor T-cell infusion, or CAR T-cell, a newer form of immunotherapy.

Video: CAR T treatment gives hope in cancer fight

In CAR T-cell therapy, a type of white blood cell called T-cells are extracted from the patients blood and modified in the lab to recognize specific cancer cells. These supercharged T-cells are then infused back into the patient, where they search out and destroy cancer cells.

The therapy, often described as a living drug because it is customized with each patients T-cells, will be marketed as Kymriah by Swiss pharmaceutical maker Novartis.

Scott was excited to hear news Wednesday that the FDA approved the same treatment for a form of childhood leukemia, meaning, he hopes, that it won't be long before it's approved for his form of cancer, diffuse large B-cell lymphoma. The FDA called the approval "historic" because it marks the first cell-based gene therapy approved in the United States.

Scott is one of 130 patients nationally in the clinical trial for his form of lymphoma, and he was the first to receive the treatment at UCM. That happened in May 2016, when he had exhausted all other options.

Scott has been feeling good for just less than a year. Chemotherapy has taken his hair three times but he has a full head of it once again. He can play an entire round of golf with his son. An avid Notre Dame football fan and season ticket holder, he had to miss each game in 2015, but plans to attend every game this season.

In May 2013, Scott noticed a painful growth in his groin area. His family doctor, Dr. Joseph Caruso, said he had developed a swollen lymph node, which could have resulted from his body trying to fight off an infection. Caruso asked him if he had recently had an infection, and Scott recounted recently stepping on a rusty nail while the roof on his home was being replaced. Caruso prescribed an antibiotic and the swelling seemed to go away.

But four months later, while in the shower, Scott noticed another lump under his arm. He went back to Caruso, who referred him to South Bend-based Beacon Health System oncologist Dr. Thomas Reid. After some scans, Reid diagnosed Stage 3 lymphoma.

Reid administered the standard treatment, four cycles of a chemotherapy regimen known as R-CHOP, an effective but highly toxic blend of drugs causing severe side effects. The fourth cycle had to be delayed because he developed appendicitis, and it was tougher than the first three.

After all of that, the cancer started growing again just two months later.

Reid referred him to Dr. Sonali Smith, professor of medicine and director of UCMs lymphoma program. Smith and her team knew the CAR T-cell therapy was being investigated in a few select centers. Their short-term goal was to keep him alive until they could be cleared to administer the clinical trial.

In February 2015, Scott received a stem-cell transplant, which went smoothly. But three months later, the cancer again started growing. Participation in two more clinical trials and some precisely targeted radiation therapy bought a little more time, but by late 2015, his lymphoma was gaining on him.

Then, in early February 2016, the UCM team received the go-ahead for the CAR T-cell treatment and began harvesting his T cells, a process that resembles dialysis. Scott said another patient had been slated to receive the treatment first, but that patient died.

It was during an appointment in May 2016, just a week before the treatment, that Scott first grasped how close he was to dying. Smith told him the treatment could cause severe side effects, including death. Five people in the trial had died.

I said, I understand. What other options do I have? Scott recalled. She says, Oh youve already surpassed all expectations. I said, What do you mean by that? And thats when she said, after the stem cell, if it comes back, life expectancy is six months. It was a rough day. On the way home I was pretty shaken up.

A little after 9:30 a.m. on May 18, 2016, Scott, sporting a Notre Dame baseball cap, was prepared for the treatment. Carefully observing was Dr. Michael Bishop, professor of medicine and director of the Hematopoietic Cellular Therapy Program at UCM, and about a dozen members of his team. A technician brought in his modified T-cells, thawed them out and infused them into Scott intravenously.

Ten minutes later, the treatment was finished. Afterward, he and his wife Cindy spent 28 days in the hospital and then were required to live in an apartment within 10 minutes of the university hospital. They were allowed to move back home to South Bend in July, about two months after the treatment.

Its incredible, Cindy said of Scotts recovery thus far. We did not realize what we were getting into, all of the risks, until days before. She (Dr. Smith) may have mentioned it but it didnt sink in. We both realized that win, lose or draw, theyre going to learn so much, just from how he responds to it.

Cindy praised how well Drs. Reid and Smith worked together between South Bend and Chicago, and how they told them just enough to be informed without telling them so much that they panicked.

She said, theres this trial, Cindy said. This is for you. You were designed for this trial and it was designed for you. We just have to keep you going until we can give it to you.

The treatment was on a Wednesday. By Friday night, his first fever came and it wasnt a surprise. Once they enter the body, each T cell multiplies rapidly, producing thousands of offspring. Then they launch a vigorous assault. All of that warfare occurring inside the body can cause severe flu-like symptoms: fever, swelling, low blood pressure.

On Sunday his fever spiked to 104 degrees. They packed him in ice around his neck and under his arms, and managed to break the fever without sending him into intensive care.

He also experienced some neurological effects, including tremors, cognitive delays and blurred vision.

Now, more than a year later, Smith still wants to see Scott every three months, and he remains very susceptible to infections because his immunity will always be compromised not from the CAR T-cell but from all of the chemotherapy. He still has some swelling because the scar tissue from three surgeries restricts the flow of lymphotic fluids.

I feel it all the time and I have very limited range of movements but it doesnt stop me, he said.

Unless the lawn needs to be mowed, then it really bothers him, she said. Some things will never change.

She said she never imagined she had married a pioneer.

I knew I had married somebody very unique, very special, but definitely not a pioneer, she said. He was the last person you ever thought would be sick. Doesnt drink. Doesnt smoke. Never had ventured on the wild side. This wasnt supposed to happen.

So far the FDA has only approved T-cell treatments for blood cancers, such as lymphoma and leukemia, but not solid tumor cancers, such as breast and colon cancer, which kill many more people. But Bishop of UCM said that day is coming. He expects those clinical trials to begin within a year or two, and receive FDA approval within about five years.

Its very exciting, Bishop said. The technology is a little more complicated but it has the potential to treat a broad spectrum of cancers. Ive been doing this for 25 years and this is one of the most significant advances Ive seen in my career.

Meanwhile, Scott will keep telling his story of hope to everyone he can, including himself. Bishop said Scott's cancer has a 10- to 20-percent chance to recur.

Youre still thinking that the other shoe can drop, Scott said. The mantra I use when negative thoughts enter my head is, Alright Scott, are you giving up? No. Are you quitting? No. Then shut up. I dont know if that will ever go away.

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South Bend man a 'walking miracle' after cancer treatment ... - South Bend Tribune

01 Sep 2017

A paper in the September 1 Nature claims a cadre of rogue microglia are all it takes to orchestrate neurodegeneration. Researchers led by Frederic Geissmann and Omar Abdel-Wahab of Memorial Sloan Kettering Cancer Center in New York, and Marco Prinz of the University of Freiburg in Germany, induced a somatic mutation in about 10 percent of microglia that switched on ERK kinase signaling. The mice later developed a severe neurodegenerative disease that paralyzed them. The researchers determined that damaging inflammation caused by the mutated microglia was likely to blame. The findings raise the possibility that similar somatic mutations in people are responsible for a rare neurodegenerative disease that occurs inchildren.

This is a great paper for many reasons, commented Bart De Strooper of the Dementia Research Institute in the U.K. I am particularly excited about the concept of acquired genetic mosaicism as a cause of neurodegenerative disorder. The paper also shows that microglia mutations can be directly causative inneurodegeneration.

Most famous for their role in causing cancer, somatic mutations can spontaneously arise in any cell, sometimes giving it a proliferative edge. Mutations in the RAS-MEK-ERK signaling pathway, for example, can cause diseases called histiocytoses if they arise in the myeloid cell lineage, which gives rise to blood and immune cells, including macrophages and microglia. Histiocytoses manifest in different ways, including leukemias, other tumors, and malfunctions in multiple organs. Mysteriously, a small fraction of carriers also get a neurodegenerative disease that manifests between childhood and middle age, with symptoms such as cerebellar ataxia and tremor (Lachenal et al., 2006; Wnorowski et al., 2008). The reason for the neurodegeneration has been amystery.

Geissmann and colleagues speculated it could be caused by microglia descended from erythro-myeloid progenitor cells (EMPs) harboring the same RAS-MEK-ERK somatic mutations. EMPs arise in the embryonic yolk sac early in development, and give rise to microglia in the brain and macrophages in other tissues (Perdiguero et al., 2014; Feb 2015 conference news).In contrast, circulating monocytes are continually replenished by hemotopoietic stem cells (HSCs) in the bonemarrow.

Doomed During Development? Histiocytoses arise from somatic mutations in hematopoietic stem cells (HSCs, left) or in erythro-myeloid progenitor (EMP) cells (right), which give rise to macrophages and microglia. The mutant microglia may cause inflammation, leading to neurodegeneration. [Courtesy of Tarnawsky and Yoder, Nature, News & Views,2017.]

To find out if somatic mutations in EMPs could beget microglia that trigger neurodegeneration, first author Elvira Mass and colleagues induced a somatic mutation that causes histiocytoses into mice. They chose the V600E variant of the BRAF gene, a substitution that switches on ERK signaling. The researchers generated transgenic mice carrying an inducible copy of the mutated BRAF gene, which could only be switched on via tamoxifen-induced Cre recombination in EMPs. This also turned on yellow fluorescent protein so the researchers could identify the cells. At embryonic day 8.5, they injected pregnant mice with a teeny dose of the drug to ensure that only a fraction of the embryos EMPs would express the mutation. About 10 percent of tissue resident macrophages, including microglia, in the resulting offspring expressed V600E BRAF at one month ofage.

The mutant microglia took up their positions in the brain, but were different from their normal counterparts from the get-go. Those carrying the V600E BRAF expressed elevated markers of proliferation, ERK signaling, and inflammation. In one-month-old mice, these feisty microglia had yet to cause trouble, but by four months of age, the researchers noticed neurological symptoms in the mice, including loss of hind limb reflexes and shortened stride. At seven months, 90 percent of the animals were affected and by nine months 60 percent of the mice had full hind limb paralysis. These symptoms, similar to cerebellar ataxia, are common in people with cerebral histiocytoses. Feeding the mice a BRAF inhibitor starting at one month of age drastically delayed onset and slowedprogression.

Compared to wild-type mice (left), animals with induced BRAF mutations in their EMPs had an expansion of mutant microglia expressing YFP in their spinal cord (middle). Microglia also expressed the activation marker CD68 (top) and phosphorylated ERK (bottom). [Courtesy of Mass et al., Nature2017.]

The researchers next searched for pathological changes that could have triggered the disorder. In month-old mice, the researchers found signs of elevated microglial and astrocyte activation, but not neuronal death. Oddly, by immunohistochemistry using the 22C11 antibody, the researchers noticed deposits of amyloid precursor protein (APP) in the inflamed areas, a phenomenon that Geissmann attributed to release of the membrane protein from newly damaged axons. In six-month-old animals, large clusters of activated, phagocytic microglia carrying the BRAF mutation crowded in the thalamus, brain stem, cerebellum, and spinal cord. These same regions were rife with synaptic and neuronal loss, demyelination, and astrogliosis. The mutant microglia had a small proliferative advantage compared with their wild-type counterparts, but Geissmann attributed the bulk of the neuronal damage to the activation of the cells, rather than their expansion. Treatment with a BRAF inhibitor mitigated theseresponses.

Gene expression analysis of mutant microglia taken from paralyzed mice revealed the differential expression of around 8,000 genes, 80 percent of which were upregulated compared to microglia from control mice. These genes included a bevy of pro-inflammatory mediators, including cytokines, phagocytosis boosters, matrix proteins, and growthfactors.

For some reason, the thalamus, brain stem, cerebellum, and spinal cord were uniquely vulnerable to the presence of the V600E BRAF mutant cells. Tissue macrophages carrying the mutation also expanded in the liver, spleen, kidney, and lung, even more so than in the brain, but did not cause damage in those regions. Geissmann speculated that differences in the tissue microenvironment could play a role in this selective vulnerability. For example, normal liver macrophages are in a near constant state of activation, Geissmann said, so the organ is equipped to deal with them. Perhaps the posterior part of the brain is unaccustomed to constant microglial activation, he said. Indeed, chronic microglial activation occurs during AD as well, and appears to ultimately inflict damage, rather than helpfulresponses.

Finally, the researchers investigated whether patients with histiocytoses also had abnormal microglia. They analyzed postmortem brain tissue from three patients with Erdheim-Chester disease (ECD), and conducted gene expression analysis on brain biopsies from one person with Langerhans cell histiocytosis (LCH), and another with juvenile xanthogranuloma (JXG). All of these patients had neurodegenerative disease associated with their histiocytoses, which were all caused by BRAF V600E mutations. In the ECD samples, the researchers spotted abundant activated microglia gathered at sites of neuronal loss, astrogliosis, and demyelination. Compared with data from five control samples, gene expression analysis on the JXG and LCH samples revealed an upregulation of genes in the MAPK pathway, including multiple pro-inflammatorycytokines.

The findings support the idea that activated microglia wreak havoc in the brain and cause neurodegeneration in people withhistiocytoses.

For a somatic mutation to have an effect, affected cells must propagate sufficiently. EMPs proliferate during early development, making it a prime time for mutant clones to multiply, Geissmann said. Perhaps the number of mutant clones born during the EMP stage would suffice to harm neurons, he said. However, if microglia are also bestowed with a proliferative edge, this would likely exacerbate the damage, he added. Either way, Geissmann proposed that inhibitors of ERK signaling might thwart neurodegeneration when mutant microglia areinvolved.

In an accompanying editorial, Stefan Tarnawsky and Mervin Yoder at Indiana University in Indianapolis noted opportunities for better diagnosis in this scenario. When somatic mutations occur in EMPs during early development, macrophages in many regions of the body will likely carry the mutations, not just microglia in the brain. This suggests that it might be possible to collect macrophage samples from more easily accessible, non-CNS tissues to look for biomarkers when diagnosing microglia-related disease, theywrote.

What about somatic mutations that might arise later in life, when tissue resident macrophages or microglia are already nestled into their permanent residences? Though recent studies reported that microglia are relatively long-lived cells, they proliferate in response to threats (Aug 2017 news),perhaps setting the stage for expansion of mutant cells, Geissmann speculated. That said, beyond people with histiocytoses, the contribution of somatic mutations in microglia to neurodegenerative disease is unclear. De Strooper and others have reported that genetic mosaicism in neurons could cause neurodegeneration (Jul 2015 news). A major impediment to studying this phenomenon is that somatic mutations that arise in the brain go undetected in standard genomic sequencing.JessicaShugart

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Somatic SNAFUCan a Few Mutant Microglia Cause Neurodegenerative Disease? - Alzforum

The Food and Drug Administration is cracking down on "unscrupulous" clinics selling unproven and potentially dangerous treatments involving stem cells.

Hundreds of clinics around the country have started selling stem cell therapies that supposedly use stem cells but have not been approved as safe and effective by the FDA, according to the agency.

"There are a small number of unscrupulous actors who have seized on the clinical promise of regenerative medicine, while exploiting the uncertainty, in order to make deceptive, and sometimes corrupt assurances to patients based on unproven and, in some cases, dangerously dubious products," FDA Commissioner Scott Gottlieb said in a statement Monday.

The FDA has taken action against clinics in California and Florida.

The agency sent a warning letter to the US Stem Cell Clinic of Sunrise, Fla., and its chief scientific officer, Kristin Comella, for "marketing stem cell products without FDA approval and significant deviations from current good manufacturing practice requirements."

The clinic is one of many around the country that claim to use stem cells derived from a person's own fat to treat a variety of conditions, including Parkinson's disease, amyotrophic lateral sclerosis (ALS), and lung and heart diseases, the FDA says.

The Florida clinic had been previously linked to several cases of blindness caused by attempts to use fat stem cells to treat macular degeneration.

The FDA also said it has taken "decisive action" to "prevent the use of a potentially dangerous and unproven treatment" offered by StemImmune Inc. of San Diego, Calif., and administered to patients at California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, Calif.

As part of that action, the U.S. Marshals Service seized five vials of live vaccinia virus vaccine that is supposed to be reserved for people at high risk for smallpox but was being used as part of a stem-cell treatment for cancer, according to the FDA. "The unproven and potentially dangerous treatment was being injected intravenously and directly into patients' tumors," according to an FDA statement.

Smallpox essentially has been eradicated from the planet, but samples are kept in reserve in the U.S. and Russia, and vaccines are kept on hand as a result.

But Elliot Lander, medical director of the California Stem Cell Treatment Centers, denounced the FDA's actions in an interview with Shots.

"I think it's egregious," Lander says. "I think they made a mistake. I'm really baffled by this."

While his clinics do charge some patients for treatments that use stem cells derived from fat, Lander says, none of the cancer patients were charged and the treatments were administered as part of a carefully designed research study.

"Nobody was charged a single penny," Lander says. "We're just trying to move the field forward."

In a written statement, U.S. Stem Cell also defended its activities.

"The safety and health of our patients are our number one priority and the strict standards that we have in place follow the laws of the Food and Drug Administration," according to the statement.

"We have helped thousands of patients harness their own healing potential," the statement says. "It would be a mistake to limit these therapies from patients who need them when we are adhering to top industry standards."

But stem-cell researchers praised the FDA's actions.

"This is spectacular," says George Daley, dean of the Harvard Medical School and a leading stem-cell researcher. "This is the right thing to do."

Daley praised the FDA's promise to provide clear guidance soon for vetting legitimate stem-cell therapies while cracking down on "snake-oil salesmen" marketing unproven treatments.

Stem-cell research is "a major revolution in medicine. It's bound to ultimately deliver cures," Daley says. "But it's so early in the field," he adds. "Unfortunately, there are unscrupulous practitioners and clinics that are marketing therapies to patients, often at great expense, that haven't been proven to work and may be unsafe."

Others agreed.

"I see this is a major, positive step by the FDA," says Paul Knoepfler, a professor of cell biology at the University of of California, Davis, who has documented the proliferation of stem-cell clinics.

"I'm hoping that this signals a historic shift by the FDA to tackle the big problem of stem-cell clinics selling unapproved and sometimes dangerous stem cell "treatments" that may not be real treatments," Knoepfler says.

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FDA Cracks Down On Stem-Cell Clinics Selling Unapproved Treatments - Northeast Indiana Public Radio

Alumnus tells first-year students they can change the world during address at convocation

With a processional led by a contingent of bagpipers, Misericordia University welcomed 436 first-year students, the third largest class in school history, during the annual convocation ceremony on Thursday, Aug. 24 in the Wells Fargo Amphitheater. Misericordia University received 2,397 applications for the first-year class, which hails from 13 states, including Connecticut, Delaware, Florida, Illinois, Indiana, Maryland, Massachusetts, New Jersey, New York, Ohio, Pennsylvania, Virginia and Washington.

The university also welcomed 75 transfer students. With the new class of students, the university expects to have between 2,750-2,775 undergraduate and graduate students in full- and part-time academic programs for the fall semester. Misericordia has 960 students scheduled to live in residence halls and townhomes on campus.

The convocation program included a welcome by President Thomas J. Botzman, Ph.D., and an address by alumnus Tariq Adwan '05, Ph.D., chief scientific officer of Alpha Genomix of Lawrenceville, Georgia, a member of the Class of 2005. During the ceremony, the Misericordia University Alumni Association presented Dr. Adwan with the Young Alumnus Award. The award is bestowed, from time to time, to a traditional undergraduate alumna or alumnus who has graduated within the past ten years and who has demonstrated outstanding professional achievements and/or community or civic service.

In his address, the native of Palestine talked about how Dr. Carol Rittner, RSM, a member of the Misericordia Board of Trustees, was instrumental in his decision to make the 10,000-mile trip from his home on the West Bank to the Misericordia campus in 2001. Dr. Rittner, a chaired scholar of Holocaust studies at Stockton State University, had met Dr. Adwan's father, who with a colleague of the Jewish faith, was working in support of the Middle East peace process.

"Although my interaction with Sister Carol at the time was relatively brief, she expressed enough compassion, selflessness, acceptance and respect that it did not take much thought before I decided that Misericordia was where I wanted to spend the next four years of my life," Dr. Adwan said of his decision. He was a student at Misericordia for only three weeks when terrorists attacked the United States on Sept. 11.

"Being the only Muslim student on campus at the time, I was devastated. I was afraid and I even contemplated going back to Palestine for fear of retaliation. Much to my surprise, by the evening of that day, my dorm room was filled with fellow students where we gathered in solidarity with the victims and their families as we tried to make sense of what just happened.

"Needless to say, what I experienced that night left a lasting impression on me, and the friendships I have made were nothing like I have ever experienced before or since," he stated. "I realized that day, that changing the world was possible, but that I needed people to do it with.

"So as you embark on your journey I encourage you to get to know as many people that are different from you as possible. Engage in face-to-face interaction with them and you will find that the people that are most different from you are those that will inspire you the most. You will also find that even though these people may seem different from you, they are, after all, people just like you."

He added, "Share your story; it matters. You might be a first-generation college student or a first-generation immigrant ... Whoever you are and whatever your story might be, it is a chapter in the American story that makes us who we are. When we know who we are, we realize that we are a nation of all creeds, colors, races and national origins. It is then that we become less threatened and more welcoming of the stranger. For we, once upon a time, were the strangers."

As a Misericordia sophomore, Adwan participated in designing and helping prepare an experiment that was placed on the 16-day Columbia shuttle mission. "Growth of Bacterial Biofilm on Surfaces During Spaceflight" was done under the direction of the Israeli Aerospace Medical Institute and Johnson Space Center Astrobiology Center. It combined a proposal from Adwan, submitted from his Misericordia residence hall, and another from Yuval Landau, a student at Tel Aviv University. Sadly, the shuttle broke up on re-entry and all members of the crew, including the first Israeli astronaut, were killed on Feb. 1, 2003.

Adwan holds a Bachelor of Science degree in biology and chemistry with honors from Misericordia, and a Ph.D. in cell biology from the University of Colorado, Denver, Colorado, specializing in stem cells and development. As chief scientific officer at Alpha Genomix, he oversees all scientific, technological and research operations, and helps identify new opportunities for growth with industry partners. Alpha Genomix is a personalized medicine testing and molecular diagnostics laboratory for pharmacogenetics, the field of research on how a person's genetic makeup affects that individual's response to medications and drugs. He is also an adjunct faculty member at Georgia Gwinnet College, Lawrenceville, Georgia, where he teaches biology.

The annual convocation ceremony welcomes first-year students and their families to Misericordia University, and acts as the official start to the new academic year. Orientation begins later in the afternoon and continues until the first day of class on Monday, Aug. 28. The orientation program includes the Orientation Days of Service on Aug. 26-27 in which first-year students and other members of the campus community volunteer in the region.

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News | Misericordia University: Alumnus tells first-year students they ... - Misericordia University

FREMONT, Calif., Aug. 23, 2017 (STL.NEWS) Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, today announced that Washington University School of Medicine in St. Louis, MO, has been added as a clinical site in the companys ongoing SCiStar Phase 1/2a clinical trial of AST-OPC1, a stem-cell derived investigational therapy, in patients with severe cervical spinal cord injury (SCI). There are now nine clinical sites enrolling patients in the study.

Washington University and its partner Barnes-Jewish Hospital have one of the largest clinical spine care practices in the United States. W. Zachary Ray, MD, Associate Professor of Neurological and Orthopedic Surgery at Washington University School of Medicine, will be the sites principal investigator. Patients enrolled in the trial will receive the AST-OPC1 treatment during surgery at Barnes-Jewish.

Given the studys encouraging early results, we look forward to evaluating whether AST-OPC1 can advance treatment options for patients with severe cervical spinal cord injuries. This investigational therapy is an important contribution to our comprehensive program to treat spinal cord injuries here at Washington University and Barnes-Jewish, said Dr. Ray.

Barnes-Jewish Hospitals Trauma Center was the first in Missouri to receive the American College of Surgeons (ACS) Level I verification, which is the highest national recognition possible from ACS.

Washington University School of Medicine is a great addition to our current AST-OPC1 SCiStar study. We hope Washington University School of Medicine will also participate in a future randomized controlled trial of AST-OPC1, stated Dr. Edward Wirth III, Chief Medical Officer of Asterias Biotherapeutics.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may have retained some minimal sensory function below their injury site. AST-OPC1 is being administered 21 to 42 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at nine centers in the U.S. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago, Santa Clara Valley Medical Center in San Jose jointly with Stanford University, Thomas Jefferson University Hospital in partnership with Magee Rehabilitation Hospital in Philadelphia, UC San Diego Health in San Diego, California, and Washington University School of Medicine in partnership with Barnes-Jewish Hospital in St. Louis, MO.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells originally isolated in 1998, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries in the current SCiStar study, which represents the first targeted population for registration trials. Asterias has completed enrollment in the first four cohorts of this study. Results to date have continued to support the safety of AST-OPC1. Additionally, Asterias has recently reported results suggesting reduced cavitation and improved motor function in patients administered AST-OPC1 in the SCiStar trial.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The companys proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The companys research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates) should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

source; Asterias Biotherapeutics , published on STL.NEWS by St Louis Media, LLC

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Washington University School of Medicine; Asterias Biotherapeutics ... - STL.News

FREMONT, Calif., Aug. 09, 2017 (GLOBE NEWSWIRE) -- Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, today announced enrollment and dosing of the first patient in the fifth and final cohort in the companys ongoing SCiStar Phase 1/2a clinical study of AST-OPC1 in motor complete cervical spinal cord injury (SCI). This cohort will dose between five and eight patients with AIS-B (motor complete, sensory incomplete) cervical spinal cord injuries with 20 million AST-OPC1 cells.

Enrollment in our SCiStar study has accelerated over the last several quarters as evidenced by the completion of enrollment of the third and fourth cohorts in July, and now the dosing of the first patient in the fifth and final cohort immediately after clearing the 30-day waiting period required by our study protocol, said Dr. Edward Wirth, Chief Medical Officer of Asterias. We remain on pace to complete enrollment of the entire SCiStar study by the end of 2017.

Asterias has completed enrollment and dosing in four of the five planned SCiStar study cohorts and has enrolled twenty-two patients in the SCiStar study. Twenty-seven patients have been administered AST-OPC1 after including patients from a previous Phase 1 safety trial and results-to-date continue to support the safety of AST-OPC1. In June 2017, Asterias reported 9-month data from the studys second dose cohort (AIS-A patients dosed 10 million cells). Subjects in this cohort showed meaningful improvements in arm, hand and finger function that were first observed at 3-months after administration of AST-OPC1, and were maintained and further increased at up to 9-months. The company expects multiple safety and efficacy readouts from the SCiStar study during the remainder of 2017 and 2018.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with subacute, C-4 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may have retained some minimal sensory function below their injury site. AST-OPC1 is being administered 21 to 42 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at eight centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago, Santa Clara Valley Medical Center in San Jose jointly with Stanford University, Thomas Jefferson University Hospital in partnership with Magee Rehabilitation Hospital in Philadelphia, and UC San Diego Health in San Diego, California.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells originally isolated in 1998, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries in the current SCiStar study, which represents the first targeted population for registration trials. Asterias has completed enrollment in the first four cohorts of this study. Results to date have continued to support the safety of AST-OPC1. Additionally, Asterias has recently reported results suggesting reduced cavitation and improved motor function in patients administered AST-OPC1 in the SCiStar trial.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

Link:
Asterias Enrolls First Patient in Final Cohort of SCiStar Clinical Trial - GlobeNewswire (press release)

There was no dance floor that didnt appeal to Dennis Trackwell.

At weddings, reunions and backyard barbecues, the southside Indianapolis resident was known to break out his best moves and boogie. On vacation, the family would usually have a deck party once the music fired up.

He would dance in the middle of the day, seemingly for no reason at all. He had a zest for life, said Devon Scott, his daughter.

Story continues below gallery

He loved anything, any kind of upbeat music, she said. We used to have impromptu dance parties. I remember my high school graduation open house, dancing in our living room.

Trackwell died from multiple myeloma, a blood cancer, in 2013. But his fun-loving spirit lives on through his family and friends. Dancing for Dennis is a yearly fundraiser founded to honor Trackwell, as well as to raise money for myeloma research and enhanced patient care at Indiana University Health.

Its basically a big, huge party, Scott said. We wanted to celebrate my dads life, while also doing something good for someone else. There are so many people who have this same disease.

Organizers have been raising money throughout the year, including a painting event at Craft and Cork art studio in Greenwood, and an 80s-themed skating party in June.

But the main event will feature a silent auction, dinner and, of course, dancing. Proceeds will benefit Miles for Myeloma, a bicycle race charity that focuses on myeloma.

Its allowed the family to take a precious memory of Dennis, and really immortalize him and keep his memory alive, along with keeping their relationship alive with his care team, said Lizzie Conkle, assistant director of development for Indiana University Melvin and Bren Simon Cancer Center

Miles for Myeloma was founded by Dr. Rafat Abonour, an Indiana University myeloma researcher and IU Health physician, to help find a cure for the disease. The two-day cycling event has raised more than $3.3 million for multiple myeloma research at the IU Simon Cancer Center.

Money being raised last year and this year will help hire a myeloma nurse navigator position, helping patients and their families handle all of the appointments, different doctors and myriad commitments necessary for treatment.

The goal of the event was to raise hope and support for myeloma patients and their families, Conkle said. Its a way for the patient to connect patient to patient, and caregiver to caregiver, to create networks of support for those diagnosed with a rare blood cancer.

Trackwell grew up in Indianapolis, served in the U.S. Air Force until 1966 and went on to work 40 years at Eli Lilly and Co.

He was diagnosed with multiple myeloma in December 2006. After a seemingly minor fall, Trackwell seemed to have a lingering injury in his abdomen that didnt feel right. Doctors told him he had a broken rib, a red flag for multiple myeloma.

The cancer forms from plasma cells that multiply uncontrollably. Malignant plasma cells can form tumors on the bones or elsewhere in the body. Low blood counts, infections and weakened bones can result from the disease.

Trackwell received his diagnosis right as he was preparing to retire from Eli Lilly.

It was so bittersweet. At his retirement party, he hadnt told anyone and everyone was asking what he was going to do now, Scott said. All of us were thinking, Oh my gosh, hes going to fight cancer.

But Trackwell was defined by his positive, upbeat nature, seeing the bright side of situations even when there didnt appear to be one, Scott said.

His doctors recommended a stem-cell transplant to infuse his body with healthy red blood cells, and for three years Trackwell went into remission. But in 2009, he was diagnosed again. The cancer this time attacked his kidneys, forcing him onto dialysis for the rest of his life.

After Trackwell died on Aug. 1, 2013, family and friends grieved. As part of that grieving process, Scott had an overwhelming need do something cathartic to ease the emotional burden.

His birthday is in September, so a month after he died, I put a message up on Facebook asking people to put on their favorite song and dance like they dont have a care in the world, and if anyone asks, say youre dancing for Dennis she said. I had all of these people sending me pictures and videos of that day.

Scott had initially wanted to establish a benefit 5K to raise awareness and money for multiple myeloma, but opted instead for a dance that kids and adults could all take part in.

The event will be held at Primo Banquet Hall & Conference Center on the southside. Tickets includes dinner, beer and wine. A special kids area will be set up, with students from Roncalli High School helping to entertain the younger guests so the adults can dance and enjoy dinner themselves, Scott said.

Last year, more than 400 people attended, and $10,000 was raised for Miles for Myeloma.

It really does mean everything that theyve worked so hard on this, Conkle said. Miles for Myeloma event came about through patients, and it really is a joint effort between patients and the myeloma care team. Having the family involved in this effort speaks to the care that Dennis received and the connection that the family made with his doctor.

Organizing the food, location and army of sponsors means that the Trackwell family works year-round to prepare for the event. But though its hard work, the impact Dancing for Dennis has had makes it all worthwhile, Scott said.

My dad was always one who told us to put ourselves out there, to do things that maybe were uncomfortable, she said. I feel like this is what were supposed to do. I think hes proud that were doing this, and in the meantime, weve met so many people who have been affected by this.

If you go

Dancing for Dennis

What: A dinner, dance and silent auction in honor of Dennis Trackwell, a southside resident who died from multiple myeloma in 2013.

When: 6 p.m. to 1 a.m. Aug. 12

Where: Primo Banquet Hall & Conference Center, 2614 National Ave., Indianapolis

Cost: Adults $30 in advance, $35 at the door; children $15; kids 6 and under free

Benefits: The event raises money for Miles for Myeloma, a fundraiser of Indiana University Health that focuses on research into myeloma as well as patient care.

Information and tickets: Facebook.com/dancingfordennis or dancingfordennis.weebly.com

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Dance event aims to raise funds for cancer research - Daily Journal

FREMONT, Calif., Aug. 03, 2017 (GLOBE NEWSWIRE) -- Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, today announced that two additional clinical sites have opened to enroll subjects for the companys ongoing SCiStar Phase 1/2a clinical study of AST-OPC1 in complete cervical spinal cord injury (SCI). The additional clinical sites include: Thomas Jefferson University Hospital, in partnership with Magee Rehabilitation Hospital, in Philadelphia, PA; and UC San Diego Health in San Diego, CA. Asterias now has eight clinical sites throughout the country enrolling patients in the study.

We are excited about the clinical site openings at Thomas Jefferson University Hospital and UC San Diego Health, stated Dr. Edward Wirth III, Chief Medical Officer of Asterias Biotherapeutics. These sites provide additional geographical reach and previous experience with spinal cord injury trials to our SCiStar study. We have recently reported completion of enrollment in four out of five cohorts in our SCiStar study so we hope these institutions will also participate in a future, larger study of AST-OPC1.

Each of the two additional clinical sites is recognized in the treatment of SCI:

The two additional clinical sites join existing clinical sites for the SCiStar study at the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.

Asterias has completed enrollment and dosing in four of the five planned SCiStar study cohorts and enrolled twenty patients in the SCiStar study. Twenty-five patients have been administered AST-OPC1 after including patients from a previous Phase 1 safety trial and results-to-date continue to support the safety of AST-OPC1. In June 2017, Asterias reported 9 month data from the AIS-A 10 million cell cohort that showed improvements in arm, hand and finger function observed at 3-months and 6-months following administration of AST-OPC1 were confirmed and in some patients further increased at 9-months. The company intends to complete enrollment of the entire SCiStar study later this year, with multiple safety and efficacy readouts anticipated during the remainder of 2017 and 2018.

About the SCiStar Trial

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may have retained some minimal sensory function below their injury site. AST-OPC1 is being administered 21 to 42 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which has provided $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells originally isolated in 1998, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries in the current SCiStar study, which represents the first targeted population for registration trials. Asterias has completed enrollment in the first four cohorts of this study. Results to date have continued to support the safety of AST-OPC1. Additionally, Asterias has recently reported results suggesting reduced cavitation and improved motor function in patients administered AST-OPC1 in the SCiStar trial.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

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Asterias Biotherapeutics Opens Two Additional Clinical Sites for ... - GlobeNewswire (press release)