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Regenexx Announces Successful Merger with Harbor View Medical – OrthoSpineNews

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BROOMFIELD, Colo.(BUSINESS WIRE)Regenexx, a Colorado-based stem-cell-treatment network and pioneer in the invention of interventional orthopedics, today announced that it successfully merged withHarbor View Medical, a leader in orthopedic stem cell therapy and part of the Regenexx network, which became effective in May 2017. As part of the transition, Jason Hellickson has assumed the CEO role. This merger positions Regenexx to further expand its National Network to better serve patients and our corporate partners.

Regenexx Corporate will be headquartered in Des Moines, IA, while Regenexxs Affiliate Program and Research and Development will be lead out of the companys Broomfield, CO location. Dr. Christopher Centeno, founder of orthopedic stem cell treatments and leader of interventional orthopedics in the United States and pioneer of the Regenexx patented procedures, will continue his role as Chief Medical Officer and remain in clinic operations in Broomfield, and continues the advancements of regenerative medicine through the largest research and data collection effort in orthopedic regenerative medicine.

As the most advanced non-surgical orthopedic care available in the United States, Im excited to continue our mission to producing the best possible patient outcomes through interventional orthopedics, said Jason Hellickson, CEO, Regenexx. In addition to individual personalized care, we will continue to provide both employers and their employees with cost savings results and successful interventions to orthopedic surgery.

Since joining the Regenexx Network in late 2014, Hellickson has reengineered clinic operations which increased capacity by more than 300 percent while offering a streamlined approach beneficial to both patients and clinic staff. He is the innovator and leader of theRegenexx Corporate Programthat enables large employers access to the Regenexx procedures. Since adding Regenexx procedures to their self-funded health and workers compensation plans, corporate partners have saved as much as 83 percent in their orthopedic surgical expenses, totaling in the many millions of dollars. In his new role, Hellickson will continue to architect the Regenexx national clinical operations to create more streamlined approaches to patient care and expand Regenexx clinics nationwide.

We look forward to continuing the build-out of Regenexx clinics, streamlining affiliate networks of more than 50 clinics nationwide, and adding additional clinics in major metropolitan areas including Chicago, Atlanta, Dallas, Philadelphia, and Charlotte under Jasons helm, said Christopher Centeno, MD. Were excited about the experience and enthusiasm that Jason brings to Regenexx.

Regenexx is the world leader in interventional orthopedics using orthobiologics and has been issued many patents for its evidence-based stem cell and blood platelet treatments used for back pain, joint pain, arthritis and acute orthopedic injuries. The benefits of interventional orthopedics are so revolutionary that seventy percent of orthopedic issues currently treated with surgery could instead be handled using regenerative methods. Mesenchymal stem cells are multipotent, adult stem cells that are therapeutic agents in the repair and regeneration of muscle, tissue, cartilage and bone. Regenexx procedures use a patients own bone marrow-derived stem cells, or blood platelets, through a blood draw, to customize needle-based, precisely-guided procedures to treat common orthopedic conditions. Its procedures have been proven to have the same or better outcomes compared to their surgical alternative.

For more information on the Regenexx Corporate Program call: 888-547-6667. For general information on Regenexx, please visitwww.Regenexxcorporate.com. For a map of current Regenexx clinics and providers clickhere.

About Regenexx and the Regenexx Physician Network The Regenexx Procedures are the nations most advanced non-surgical stem cell and blood platelet treatments for common joint injuries and degenerative joint conditions, such as osteoarthritis and avascular necrosis. These stem cell procedures utilize a patients own stem cells or blood platelets to help heal damaged tissues, tendons, ligaments, cartilage, spinal disc, or bone.

For more information on Regenexx, please visit:http://www.regenexx.com

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Regenexx Announces Successful Merger with Harbor View Medical - OrthoSpineNews

High-tech solutions top the list in the fight against eye disease – Engadget

Cataracts are the single leading cause of blindness worldwide, afflicting roughly 42 percent of the global population, including more than 22 million Americans. The disease, which causes cloudy patches to form on the eye's normally clear lens, can require surgery if left untreated. That's why Google's DeepMind AI division has teamed with the UK's National Health Service (NHS) and Moorfields Eye Hospital to train a neural network that will help doctors diagnose early stage cataracts.

The neural network is being trained on a million anonymized optical coherence tomography (OCT) scans (think of a sonogram, but using light instead of sound waves) in the hopes it will eventually be able to supplement human doctors' analyses, increasing both the efficiency and accuracy of individual diagnoses.

"OCT has totally revolutionized the field of ophthalmology. It's an imaging system for translucent structures that utilizes coherent light," Dr. Julie Schallhorn, an assistant professor of ophthalmology at UC San Francisco, said. "It was first described in 1998 and it gives near-cell resolution of the cornea, retina and optic nerve.

"The optic nerve is only about 200 microns thick, but you can see every cell in it. It's given us a much-improved understanding of the pathogenesis of diseases and also their response to treatments." The new iteration of OCT also measures the phase-shift of refracted light, allowing doctors to resolve images down to the capillary level and observe the internal structures in unprecedented detail.

"We're great at correcting refractive errors in the eyes so we can give you good vision far away pretty reliably, or up close pretty reliably," Schallhorn continued. "But the act of shifting focus from distance to near requires different optical powers inside the eye. The way the eye handles this when you're young is through a process called 'accommodation.'" There's a muscle that contracts and changes the shape of the lens to help you focus on close objects. When you get older, even before you typically develop cataracts, the lens will stiffen and reduce the eye's ability to change its shape.

"The lenses that we have been putting in during cataract surgery are not able to mimic that [shapeshifting] ability, so people have to wind up wearing reading glasses," Schallhorn said. There's a lot of work in the field to find solutions for this issue and help restore the eye's accommodation.

There are two front-runners for that: Accommodating lenses, which use the same ciliary muscle to shift focus, and multifocal lenses, which work just like your parents' multifocal reading glasses except that they sit directly on the eye itself. The multifocals have been on the market for about a decade, though their design and construction has been refined over that time.

To ensure the lenses that doctors are implanting are just as accurate as the diseased ones they're removing, surgeons are beginning to use optiwave refractive analysis. Traditionally, doctors relied on measurements taken before the surgery to know how to shape the replacement lenses and combined those with nomograms to estimate how powerful the new lens should be.

The key word there is "estimate." "They especially have problems in patients who have already had refractive surgery like LASIK," Schallhorn explained. The ORA system, however, performs a wavefront measurement of the cornea after the cataract has been removed to help surgeons more accurately pick the right replacement lens for the job.

Corneal inlays are also being used. These devices resemble miniature contact lenses but sit in a pocket on the cornea that's been etched out with a LASIK laser to mimic the process of accommodation and provide a greater depth of focus. They essentially serve the same function as camera apertures. The Kamra lens from AcuFocus and the Raindrop Near Vision Inlay from Revision Optics are the only inlays approved by the FDA for use in the US.

Glaucoma afflicts more than 70 million people annually. This disease causes fluid pressure within the eye to gradually increase, eventually damaging the optic nerve that carries electrical signals from the eye to the brain. Normally, detecting the early stages of glaucoma requires a comprehensive eye exam by a trained medical professional -- folks who are often in short supply in rural and underserved communities. However, the Cambridge Consultants' Viewi headset allows anyone to diagnose the disease -- so long as they have a smartphone and 10 minutes to spare.

The Viewi works much like the Daydream View, wherein the phone provides the processing power for a VR headset shell -- except, of course, that instead of watching 360 degree YouTube videos, the screen displays the flashing light patterns used to test for glaucoma. The results are reportedly good enough to share with you eye doctor and take only about five minutes per eye. Best of all, the procedure costs only about $25, which makes it ideal for use in developing nations.

And while there is no known cure for glaucoma, a team of researchers from Stanford University may soon have one. Last July, the team managed to partially restore the vision of mice suffering from a glaucoma-like condition.

Normally, when light hits your eye, specialized cells in the retina convert that light into electrical signals. These signals are then transmitted via retinal ganglion cells, whose long appendages run along the optic nerve and spread out to various parts of the brain's visual-processing bits. But if the optic nerve or the ganglion cells have been damaged through injury or illness, they stay damaged. They won't just grow back like your olfactory sensory nerve.

However, the Stanford team found that subjecting mice to a few weeks of high-contrast visual stimulation after giving them drugs to reactivate the mTOR pathway, which has been shown to instigate new growth in ganglion cells, resulted in "substantial numbers" of new axons. The results are promising, though the team will need to further boost the rate and scope of axon growth before the technique can be applied to humans.

Researchers from Japan have recently taken this idea of cajoling the retina into healing itself and applied it to age-related macular degeneration cases. AMD primarily affects people aged 60 and over (hence the name). It slowly kills cells in the macula, the part of the eye that processes sharp detail, and causes the central focal point of their field of vision to deteriorate, leaving only the peripheral.

The research team from Kyoto University and the RIKEN Center for Developmental Biology first took a skin sample from a human donor, then converted it into induced pluripotent stem (IPS) cells. These IPS cells are effectively blank slates and can be coerced into redeveloping into any kind of cell you need. By injecting these cells into the back of the patient's eye, they should regrow into retinal cells.

In March of this year, the team implanted a batch of these cells into a Japanese sexagenarian who suffers from AMD in the hope that the stem cells would take hold and halt, if not begin to reverse, the damage to his macula. The team has not yet been able to measure the efficacy of this treatment but, should it work out, the researchers will look into creating a stem-cell bank where patients could immediately obtain IPS cells for their treatment rather than wait months for donor samples to be converted.

And while there isn't a reliable treatment for dry-AMD, wherein fatty protein deposits damage the Bruchs membrane, a potent solution for wet-AMD, which involves blood leaking into the eyeball, has been discovered in a most unlikely place: cancer medication. "Genentech started developing a new drug when an ophthalmologist in Florida just decided to inject the commercially available drug into patients eyes," Schallhorn explained.

"Generally this is not a great idea because sometimes things will go terribly wrong," she continued, "but this worked super-well. It basically stops and reverses the growth of these blood vessels." The only problem is that the drugs don't last, requiring patients to receive injections into their eyeballs every four to eight weeks. Genentech and other pharma companies are working to reformulate the drug -- or at least develop a mechanical "reservoir" -- so it has to be injected only once or twice a year.

Stem-cell treatments like those used in the Kyoto University trial have already proved potentially effective against a wide range of genomic diseases, so why shouldn't it work on the rare genetic condition known as choroideremia? This disease is caused by a single faulty gene and primarily affects young men. Similar to AMD, choroideremia causes light-sensitive cells at the back of the eye to slowly wither and die, resulting in partial to complete blindness.

In April of 2016, a team of researchers from Oxford University performed an experimental surgery on a 24-year-old man suffering from the disease. They first injected a small amount of liquid into the back of the eye to lift a section of the retina away from the interior cellular wall. The team then injected functional copies of the gene into that same cavity, replacing the faulty copies and not only halting the process of cellular death but actually restoring a bit of the patient's vision.

Gene therapy may be "surely the most efficient way of treating a disease," lead author of the study, Oxford professor Robert MacLaren, told BBC News, but its widespread use is still a number of years away. Until then, good old-fashioned gadgetry will have to suffice. Take the Argus II, for example.

The Argus II bionic eye from Second Sight has been in circulation since 2013, when the FDA approved its use in treating retinitis pigmentosa. It has since gotten the go-ahead for use with AMD in 2015. The system leverages a wireless implant which sits on the retina and receives image data from an external camera that's mounted on a pair of glasses. The implant converts that data into an electrical signal which stimulates the remaining retinal cells to generate a visual image.

The Argus isn't the only implantable eyepiece. French startup Pixium Vision developed a similar system, the IRIS II, back in 2015 and implanted it in a person last November after receiving clearance from the European Union. The company is already in talks with the FDA to bring its IRIS II successor, a miniaturized wireless subretinal photovoltaic implant called PRIMA, to US clinical trials by the end of this year.

Ultimately, the goal is to be able to replace a damaged or diseased eye entirely, if necessary, using a robotic prosthetic. However, there are still a number of technological hurdles that must be overcome before that happens, as Schallhorn explained.

"The big thing that's holding us back from a fully functional artificial eye is that we need to find a way to interface with the optic nerve and the brain in a way that we transmit signals," she said. "That's the same problem we're facing with prosthetic limbs right now. But there are a lot of smart people in the field working on that, and I'm sure they'll come up with something soon."

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High-tech solutions top the list in the fight against eye disease - Engadget

SBP Scientist Receives Prestigious WM Keck Foundation Grant – Newswise (press release)

Newswise La Jolla, Calif., July 10, 2017 Sanford Burnham Prebys Medical Discovery Institute (SBP) is pleased to announce that the W.M. Keck Foundation has awarded a $1M grant to Duc Dong, Ph.D., to advance research to generate replacement organs and cellssuch as insulin producing cells for diabetes or dopamine-producing neurons for Parkinsons diseasedirectly in the body and without the use of stem cells.

The grant will allow Dong to extend his studies reprogramming dispensable cells such as skin, vasculature and fat cells while they remain in the body, in vivo, without the use of cultures or induced pluripotent stem cell techniques, which could be a safer and more effective method to treat degenerative diseases and injuries.

Dong, an assistant professor in the Human Genetics Program, uses zebrafishtiny striped fish commonly used for genetic studies-as a vertebrate model to reprogram cells into unrelated types, completely within the body of these living animals. Dongs research team already has proof-of-concept data demonstrating in vivo conversion of muscle and skin cells into beta-cell precursors (the cells that mature to produce insulin)an accomplishment assumed to be impossible by most scientists.

Im honored to have received this grant from the Foundation, says Dong. The award will support my vision to ultimately make in vivo cell lineage reprogramming a practical therapeutic strategy to replace or even enhance lost, damaged or aging tissues. Our next steps are to develop technologies to convert any cells in the body, at any age, into any cell type of interest, and to uncover the molecular mechanisms driving that process.

Based in Los Angeles, the W. M. Keck Foundation was established in 1954 by the late W. M. Keck, founder of the Superior Oil Company. The Foundations grant making is focused primarily on pioneering efforts in the areas of medical research, science and engineering and undergraduate education. The Foundation also maintains a Southern California Grant Program thatprovides support for the Los Angeles community, with a special emphasis on children and youth. For more information, please visit http://www.wmkeck.org.

About SBPSanford Burnham Prebys Medical Discovery Institute (SBP) is an independent nonprofit medical research organization that conducts world-class, collaborative, biological research and translates its discoveries for the benefit of patients. SBP focuses its research on cancer, immunity, neurodegeneration, metabolic disorders and rare childrens diseases. The Institute invests in talent, technology and partnerships to accelerate the translation of laboratory discoveries that will have the greatest impact on patients. Recognized for its world-class NCI-designated Cancer Center and the Conrad Prebys Center for Chemical Genomics, SBP employs about 1,100 scientists and staff in San Diego (La Jolla), Calif., and Orlando (Lake Nona), Fla. For more information, visit us at SBPdiscovery.org or on Facebook at facebook.com/SBPdiscovery and on Twitter @SBPdiscovery.

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SBP Scientist Receives Prestigious WM Keck Foundation Grant - Newswise (press release)

Research on Stem Cell Therapy | Liveyon Regenerative Medicine

Liveyon LLC is the exclusive worldwide distributor of a regenerative medicine product that is derived from umbilical cord. This product contains cells, stem cells and growth factors which may serve as a therapy for various degenerative diseases/disorders.

Stem cells and cell based therapies have shown tremendous promise; yet controlled studies are still needed in order to confirm its efficacy. Professional judgment and expertise is needed in using these therapies for any therapeutic use, and we urge anyone embarking on the use of stem cell therapies or any regenerative medicine product to consult the national health data bases to evaluate current information from clinical trials. The FDA websites on human tissue should also be consulted to get its current evaluation of any regenerative therapy.

Stem cells, like other medical products that are intended to treat, cure or prevent disease, generally require FDA approval before they can be marketed. FDA has not approved any stem cell-based or regenerative medicine products for use, other than cord blood-derived hematopoietic progenitor cells (blood forming stem cells) for certain indications.

http://www.fda.gov/AboutFDA/Transparency/ Basics/ucm194655.htm

844-548-3966 support@liveyon.com

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Squeezing innovation out of the NASA Twins study: Pipetting and cell isolation in space – Phys.Org

July 11, 2017 NASA immunologist Hawley Kunz performs a microgravity evaluation of the pipetting steps necessary for terrestrial purification of cells. Credit: NASA

Just like early explorers, NASA Twins Study investigators are venturing into new territory. Conducting human omics research on twin astronauts as part of the One Year Mission that took place aboard the International Space Station is one such venture. As technology evolves so does the research. NASA is evaluating more efficient and innovative research techniques to prepare for the journey to Mars.

Innovative thinking could improve the way biological samples are processed and transported from space back to research labs on Earth for future studies. This thinking was prompted by researchers in NASA's Human Research Program (HRP) and Twins Study investigators at Johns Hopkins Medicine.

Freshly isolated samples yield better results than cells isolated from frozen samples returned to Earth from the orbiting laboratory. Pipetting fresh samples at ambient temperature and performing cell isolation on the space station also eliminates the need for expeditious transportation logistics, and allows for more frequent sampling. Once cells are isolated, the samples can be viably frozen and return on any transfer vehicle at any time for further analysis.

On an aircraft that is used as a parabolic flight analog to create short periods of simulated microgravity, Twins Study Investigators Dr. Andrew Feinberg and Lindsay Rizzardi of Johns Hopkins Medicine tested a theory that liquids could be transferred safely in microgravity using a pipettor, which is a slender, graduated measurement tube. Previously researchers thought transferring biological fluids in space could pose risks to precisely controlling the sample.

"This analog demonstrated that pipetting of open fluids is relatively simple and easily controlled and that all fluid transfer steps associated with centrifugation can be replicated in microgravity," Feinberg said. "When dealing with genetic material, research requires precise transfer of liquids among different types of tubes in order to purify DNA, RNA or protein from biological samples to perform molecular analyses."

Coinciding with the fluid transfer research was cell isolation research being conducted by NASA immunologists Brian Crucian, Clarence Sams, Hawley Kunz and NASA astronaut and molecular biologist Kate Rubins. NASA researchers tested terrestrial protocols for cell purification in microgravity using the parabolic flight analog. They found that cell isolation and purification could both be performed in microgravity. Rubins also confirmed some of these findings in space. They published their research with Feinberg and Rizzardi in the July 2016 issue of NPJ Microgravity.

Crucian said, "Laboratory procedures for isolating and purifying cells typically require sensitive gradient centrifugation, careful extraction of isolated cells, and general open pipetting of liquids for washing and transferring the isolated cells."

Being able to transfer fluids and isolate cells in space is significant for a variety of reasons. Mars is a challenging distance from Earth if diagnostics are ever needed of a crewmember. Enabling astronauts to conduct more human research independently could help diagnose an illness more quickly, possibly saving a life in a medical emergency.

As NASA prepares for its journey to Mars, the way researchers handle and processes biological samples in space could change. The protocols validated by the Johns Hopkins and NASA investigators demonstrate that standard cell isolation protocols may indeed be performed in space, something which may enable certain types of genetic, or 'omics', research onboard the space station. Molecular biology technologies such as hand-held sequencers continue to evolve pushing the boundaries of scientific research. HRP will continue to adapt its methodologies to support novel research that protects and ensures the safety of future crews on long-duration missions while opening the door for innovative opportunities.

Explore further: Study examines effects of spaceflight on immune system

More information: Lindsay F Rizzardi et al, Evaluation of techniques for performing cellular isolation and preservation during microgravity conditions, npj Microgravity (2016). DOI: 10.1038/npjmgrav.2016.25

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Method determines cell age more accurately, could help elderly patients – Medical Xpress

July 11, 2017

Sure, you know how old you are, but what about your cells? Are they the same age? Are they older, younger? Why does it matter?

Led by scientists at the Johns Hopkins University, a team of researchers is reporting progress in developing a method to accurately determine the functional age of cells, a step that could eventually help clinicians evaluate and recommend ways to delay some health effects of aging and potentially improve other treatments, including skin graft matching and predicting prospects for wound healing.

In the current issue of Nature Biomedical Engineering, lead author Jude M. Phillip, who conducted this research while completing his doctorate in chemical and biomolecular engineering at Johns Hopkins, reports success in creating a system that considers a wide array of cellular and molecular factors in one comprehensive aging study.

These results show that the biophysical qualities of cells, such as cell movements and structural features, make better measures of functional age than other factors, including cell secretions and cell energy.

The multidisciplinary team of engineers and clinicians examined dermal cells from just underneath the surface of the skin taken from both males and females between the ages of 2 to 96.

The researchers from Johns Hopkins, Yale University and the National Cancer Institute of the National Institutes of Health hoped to devise a system that through computational analysis could take the measure of various factors of cellular and molecular functions. From that information, they hoped to determine the biological age of individuals more accurately using their cells, in contrast to previous studies, which makes use of gross physiology, or examining cellular mechanisms such as DNA methylation.

"We combined some classic biomolecular hallmarks of aging, and sought to further elucidate the role of biophysical properties of aging cells, all in one study," said Phillip, now a post-doctoral fellow at Weill Cornell Medicine.

Researchers trying to understand aging have up to now focused on factors such as tissue and organ function and on molecular-level studies of genetics and of epigenetics, meaning heritable traits that are not traced to DNA. The level in between - cells - has received relatively little attention, the researchers wrote.

This research was meant to correct for that omission by considering the biophysical attributes of cells, including such factors as the cells' ability to move, maintain flexibility and structure. This focus emerges from the understanding that changes associated with aging at the physiological level - such as diminished lung capacity, grip strength and mean pressure in the arteries - "tend to be secondary to changes in the cells themselves, thus advocating the value of cell-based technologies to assess biological age," the research team wrote.

For example, older cells are more rigid and do not move as well as younger cells, which, among other consequences, most likely contributes to the slower wound healing commonly seen in older people, said Denis Wirtz, the senior author, Johns Hopkins' vice provost for research and the Theophilus H. Smoot Professor of Chemical and Biomolecular Engineering at the Whiting School of Engineering. Wirtz and Phillip conducted their research in the Johns Hopkins Institute for NanoBioTechnology.

From the analysis, they were able to stratify individuals' samples into three groups: those whose cells roughly reflected their chronological age, those whose cells were functionally older, and those whose cells were functionally younger. The results also showed that the so-called biophysical factors of cells determined a more accurate measure of age than biomolecular factors such as cell secretions, cell energy and the organization of DNA.

Phillip explained that this better accuracy from the biophysical factors most likely results from the orchestration of many biomolecular factors. He compared it to the more complete picture you get looking at a forest from a distance without binoculars.

"With binoculars you can see details about the individual trees, the color and shapes of the leaves, the roughness of the bark, the type of tree, but without the binoculars you can now see the density of the trees, and whether there is a barren plot, or a group or dying trees," Phillip said. "This is something you may miss with the binoculars, unless you are looking at the correct spot."

The more accurate system could eventually enable clinicians to see aging in cells before a patient experiences age-related health decline. This in turn could allow doctors to recommend treatments or changes in life habits, such as exercise or diet changes, Wirtz said. Phillip said the work could potentially help clinicians produce more successful skin grafts by matching cell characteristics of the donor and the graft site. Other potential applications range from toxicology screening for cosmetics and topical therapeutics to predicting progression of some age-related diseases.

The researchers acknowledge that the system needs further testing with a larger cell sample, but the results are robust and encouraging. Conducted along with clinicians such as Jeremy Walston, the Raymond and Anna Lublin Professor of Geriatric Medicine, and co-director of the Biology of Healthy Aging program at the Johns Hopkins School of Medicine, this work promises to allow clinicians to measure a person's health in the present and the future.

"It opens the door to finally be able to track how a person is doing at the cellular level," Wirtz said. "This platform is also more than just a cellular age predictor, it has the ability to do so much more in terms of assessing an individual's cellular health," Phillip said.

Explore further: New cellular target may put the brakes on cancer's ability to spread

More information: Jude M. Phillip et al, Biophysical and biomolecular determination of cellular age in humans, Nature Biomedical Engineering (2017). DOI: 10.1038/s41551-017-0093

Most cells can divide only a limited number of times and eventually undergo permanent cell cycle arrest, a state known as cellular senescence. Cellular senescence is mediated by activation of specific cellular signaling pathways ...

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FDA Approves First Sickle Cell Drug in 20 Years – Scientific American

The FDA approved a new medicine Friday to reduce the complications associated with sickle cell disease, a rare blood disorder.

The drug, Endari, is made by privately held Emmaus Medical and is the first new treatment for sickle cell disease to secure FDA approval in almost 20 years. However, the active ingredient in EndariL-glutamineis an old chemical that can be purchased over the counter, which could complicate Emmauss ability to obtain insurance coverage.

In Emmauss pivotal clinical trial, treatment with Endari over 48 weeks reduced the frequency and length of hospital visits for sickle cell pain crises compared to placebo. Commonly reported side effects of the drug included constipation, nausea, and headache.

Sickle cell disease is a rare, inherited disorder characterized by abnormally sickle-shaped red blood cells. These malformed red blood cells clog blood vessels and cut off oxygen to the bodys tissues, leading to episodes of severe pain and organ damage.

Approximately 100,000 people in the U.S. have sickle cell disease, according to the National Institutes of Health.

While Endari focuses on reducing the complications of sickle cell disease, Bluebird Bio is developing a gene therapy to treat the diseases underlying cause and potentially cure patients. Research using the CRISPR gene-editing technology aims to accomplish the same goal.

Other companies working on new, novel drugs targeting sickle cell include Global Blood Therapeutics and Novartis.

Republished with permission fromSTAT. This articleoriginally appearedon July 7, 2017

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FDA Approves First Sickle Cell Drug in 20 Years - Scientific American

Neural stem cells steered by electric fields in rat brain – Medical Xpress

July 11, 2017 Transplants of neural stem cells might be used to treat brain injuries, but how to get them to the right location? UC Davis researcher Min Zhao and Junfeng Feng, a neurosurgeon at Ren Ji Hospital, Shanghai, showed that they can steer transplanted stem cells (green, in inset on right) to one part of a rat's brain using electrical fields. Credit: Junfeng Feng

Electric fields can be used to guide neural stem cells transplanted into the brain towards a specific location. The research, published July 11 in the journal Stem Cell Reports, opens possibilities for effectively guiding stem cells to repair brain damage.

Professor Min Zhao at the University of California, Davis School of Medicine's Institute for Regenerative Cures studies how electric fields can guide wound healing. Damaged tissues generate weak electric fields, and Zhao's research has shown how these electric fields can attract cells into wounds to heal them.

"One unmet need in regenerative medicine is how to effectively and safely mobilize and guide stem cells to migrate to lesion sites for repair," Zhao said. "Inefficient migration of those cells to lesions is a significant roadblock to developing effective clinical applications."

Dr. Junfeng Feng, a neurosurgeon at Ren Ji Hospital, Shanghai Jiao Tong University and Shanghai Institute of Head Trauma, visited Zhao's lab to study how electric fields might guide stem cells implanted in the brain.

Natural neural stem cellscells that can develop into other brain tissuesare found deep in the brain, in the subventricular zone and hippocampus. To repair damage to the outer layers of the brain (the cortex), they have to migrate some distance, especially in the large human brain. Transplanted stem cells might also have to migrate some way to find an area of damage.

Stem Cells Move "Upstream"

Feng and Zhao developed a model of stem cell transplants in rats. They placed human neural stem cells in the rostral migration stream - a pathway in the rat brain that carries cells towards the olfactory bulb, which governs the animal's sense of smell. Cells move along this pathway partly carried by the flow of cerebrospinal fluid and partly guided by chemical signals.

By applying an electric field within the rat's brain, they found that they could get the transplanted stem cells to swim "upstream" against the fluid flow and natural cues and head for other locations within the brain.

The transplanted stem cells were still in their new locations weeks or months after treatment.

"Electrical mobilization and guidance of stem cells in the brain therefore provides a potential approach to facilitate stem cell therapies for brain diseases, stroke and injuries," Zhao said.

Explore further: Distant brain regions selectively recruit stem cells

More information: Jun-Feng Feng et al, Electrical Guidance of Human Stem Cells in the Rat Brain, Stem Cell Reports (2017). DOI: 10.1016/j.stemcr.2017.05.035

Journal reference: Stem Cell Reports

Provided by: UC Davis

Stem cells from bone marrow or fat improve recovery after stroke in rats, finds a study published in BioMed Central's open access journal Stem Cell Research & Therapy. Treatment with stem cells improved the amount of brain ...

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Scientists discover treatment target for pulmonary fibrosis – Medical Xpress

July 11, 2017 by Anna Williams The figure demonstrates how human pathology (top) can be digitized for precision medicine (bottom). Before, we were limited to histologic analysis of lung tissues. Now we can measure the expression of every gene and every cell within that lung and actually digitize that biology, Budinger said. Credit: Northwestern University

In new research published in the Journal of Experimental Medicine, Northwestern Medicine scientists have demonstrated that a unique population of immune cells plays a key role in the development of pulmonary fibrosis. They also showed that targeting such cells could lead to new treatments for the disease.

The study, a collaboration across multiple divisions, departments and schools at Northwestern, was led by Scott Budinger, MD, chief of Pulmonary and Critical Care in the Department of Medicine, and Harris Perlman, PhD, chief of Rheumatology in the Department of Medicine.

Pulmonary fibrosisincluding idiopathic pulmonary fibrosis and scleroderma-associated pulmonary fibrosisis a fatal disease marked by scarring and hardening of lung tissue. The cause is often unknown, and there is currently no effective treatment.

Previously, it was generally accepted in the field that immune cells were unimportant to the development of pulmonary fibrosis. But data from Northwestern's extensive research program in sclerodermaan autoimmune disease closely tied to pulmonary fibrosis that results in hardening of the skinsuggested immune cells may actually play an important role.

To test that hypothesis, the team of scientists utilized next-generation sequencing technologies and novel animal models generated at Northwestern to trace immune cells throughout the progression of pulmonary fibrosis.

They also applied those tools to the analysis of tissue samples collected at Northwestern Medicine hospitals, correlating the animal model data with the patient samples. "One of the strengths of our study is that we go from bench to bedside," Perlman said.

The scientists discovered that a new sub-population of immune cells called monocyte-derived alveolar macrophages were in fact a key driver of disease development in pulmonary fibrosis. Further, the genetic deletion of this population of cells prevented fibrosis in mouse models.

"This will be transformative for the field," said first author Alexander Misharin, MD, PhD, assistant professor of Medicine in the Division of Pulmonary and Critical Care. "Pulmonary fibrosis is a complex diseaseit's not driven by a single gene or cell typebut this study now demonstrates that these immune cells play a key role. This will change the current paradigm."

The findings have important implications for the development of future therapies, especially given that targeting such cells may lead to fewer adverse effects. "These cells are attractive for therapy because they don't need to be there. They aren't necessary for normal function or developmental purposes," said Perlman, also the Mabel Greene Myers Professor of Medicine.

The new methods used in the research could also spark future research in the field.

"This is a novel application of genomic technologies to understanding pulmonary fibrosis," said Budinger, also the Ernest S. Bazley Professor of Airway Diseases and a professor of Cell and Molecular Biology. "By showing that these technologies can be directly applied to patient samples, we offer the promise of incorporating them into personalized medicine approaches. It creates a resource for the research community to develop novel therapies."

The team is now actively deploying these technologies to examine lungs from patients with pulmonary fibrosis, fibrotic skins from patients with scleroderma and joints from patients with rheumatoid arthritis to identify other common immune mechanisms in fibrosis that might be targets for new therapies.

Budinger, Perlman and Misharin are also members of the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

In a separate finding, the scientists discovered that injury to a mouse early in life permanently altered the immune cell population in the lungs, findings that could have implications for aging research. "Severe injury early in life permanently changes you, in a way that might impact your susceptibility to disease when you get older," Budinger said.

Explore further: Vaccine improves fibrosis in mouse model of idiopathic pulmonary fibrosis

More information: Alexander V. Misharin et al. Monocyte-derived alveolar macrophages drive lung fibrosis and persist in the lung over the life span, The Journal of Experimental Medicine (2017). DOI: 10.1084/jem.20162152

A Yale-led research team has identified an important enzyme that could lead to new therapies for a chronic fatal lung disease that affects hundreds of thousands in the United States each year.

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Scientists discover treatment target for pulmonary fibrosis - Medical Xpress

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