StemCell Therapy

Policymakers around the world are determining how to apply existing regulations to 3D organ printing.

When Selena Gomez suffered from Lupus, her best friend Francia Raisa donated her kidney to her, saving Gomezs life. For decades, people just like Gomez have escaped death due to heroic organ donations, either from living or deceased donors.

Today, 3D printingalso referred to as 3DPmay revolutionize the practice of organ donation. Although 3D printing is currently used to make jewelry, food, and art, it may soon be used for medical solutions such as organ donations and bionic limbs.

As a result, policymakers around the world seek increased regulation of 3DP organs. Yet 3D bioprinting does not clearly fit into existing regulatory frameworks.

Since bioprinting generally falls within the regulatory domain of regenerative medicine, medical devices, and biologic drugs, regulators face the challenge of applying existing rules to this uncertain field. As of now, it is unclear whether policymakers can effectively regulate bioprinting under existing regulations, or if a new, specific regulatory process will be necessary.

In determining how to regulate 3D organ printing, policymakers must juggle many possible concerns. Since the technology is still developing, a lot of uncertainty remains about what the actual risks and ethical concerns are. For example, one ethical concern is that 3DP organs may be available to wealthy people only, while less affluent individuals will be blocked out from using these organs.

Another concern is safety. Since 3DP may require stem-cell technology, and the patients own cells may be used for replication, it is difficult to assess the safety risks. Stem-cell therapy cannot be tested on a large sample of healthy people, which limits effective clinical analysis. Also, 3DP biotechnology may open up new uses beyond 3DP organs, such as enhancement of human capacities for military use. Developers could use the technology to make military officers or even terrorists less vulnerable to injury in battle, but this would open up a whole new challenge for law enforcement and national security.

The U.S. Food and Drug Administration (FDA) focuses on the regulation of 3D printed organs. FDA so far has only released guidance on 3DP, and the recommendations do not cover bioprinting.

A significant concern in the United States is that 3D printed organs do not fit into any clear category of law. First, they are not organs because they are not born alive at any stage of development. Second, they are not drugs because drugs are used orally rather than through an invasive surgery, and drugs are primarily meant to relieve illness while donated organs may completely cure an illness.

Some policymakers in the United States propose regulating 3DP organs as a biological product, defined as a virus, therapeutic serum, toxinor analogous productapplicable to the prevention, treatment, or cure of a disease or condition of human beings.

Research company Biogelx suggests that biological products may be a promising category for printed organs. Within biological products, a 3DP organ is comparable to proteins because to print the organ, clinicians replicate healthy human cells, which include such proteins, says Biogelx. Although existing regulatory frameworks often compare 3DP organs to medical devices, Biogelx asserts that these organs should not be regulated as medical devices. Medical devices are not made of biological material and are often metal or plastic devices that help an individuals standard of life, but 3DP organs are different since they cause a chemical reaction in the body and have the purpose of wholly replacing an existing organ, says Biogelx.

International policymakers are also struggling to find a sufficient regulatory framework. In Canada, Health Canada released draft guidance last year to develop regulations for medical device manufacturers working towards bioprinting. Health Canada has several concerns about bioprinting, and it suggests that manufacturers looking for bioprinting licenses should be required to submit information regarding the use of additives in materials, the verification of the software for the bioprinting design, the method of sterilizing the machines, and the process of safe removal and reuse of bioprinting materials and residues.

Finally, Europes 3DP health technology is regulated by the European Medical Devices Directive, the Active Implantable Medical Devices Directive, and the Invitro Diagnostic Medical Devices Directive. The Medical Devices Directive categorizes bioprinting devices into several risk classes. Across the different classes, devices ranked as higher risk are subjected to third party assessment and more stringent requirements for clinical data. The highest risk class, implantable devices such as 3D organs, requires an independent design dossier review. A design dossier assesses risk, evaluates clinical data, and demonstrates the technologys compliance with regulations and requirements.

Although 3D printing of organs is right around the corner, policymakers around the world lack the information necessary to make regulatory decisions in this space. Different countries have different approaches, but many of the leading nations in 3DP share similar concerns. With more information, regulators will have to decide if existing regulatory frameworks can adequately address the safety concerns of 3D printed organs.

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The Uncertainty of Regulating 3D Organ Printing - The Regulatory Review

Following in the very successful footsteps of its sister companies GE Additive, GE Aerospace, and GE Oil and Gas, now GE Healthcare is starting to seriously look at 3D printing. After signing with Formlabs to streamline 3D printing of anatomical models, the giant is now partnering with Advanced Solutions Life Sciences (ASLS) toadvance the field of 3D biofabrication.

And if GE Healthcare is moving fast, bioprinting is not going any slower. It may be the positive momentum from the latest Termis (the leading regenerative medicine event) in Orlando, or maybe 3dpbm is just in the right place at the right time, but 3D Bioprinting Solutions has also been making some giant leaps recently. Now Advanced Solutions Life Sciences, the company founded by Michal Golway, one of the first pioneers in bioprinting, is going to benefit from GEs powerful distribution and R&D means.

As per the agreement, GE Healthcare will distribute the worlds first integrated 3D bioprinter + confocal scanner (BioAssemblyBot + GE IN Cell Analyzer 6500HS) as part of a strategic R&D and distribution partnership that sets out to personalize tissue regeneration. The integration of IN Cell Analyzer and BioAssemblyBot systems technologies will embed cellular-level assessments into the 3D bioprinting workflow used to create human tissue models.

Bioprinted tissues are small in size and die quickly, due to an inability to engineer small blood vessels the bodys supply network. ASLS patented Angiomics technology enables bioprinted microvessels to self-assemble into functional capillary beds, which deliver nutrients, oxygen, and hormones to the 3D tissue model and remove waste. This partnership would allow life scientists and tissue engineers to quickly design, build and image living, vascularized 3D tissues in a single, agile process.

Printing multi-material 3D objects inside of microwell plates allows scientists to efficiently move away from traditional 2D monocultures on plastic, to 3D discovery and cytotoxicity models that more accurately reflect native biology and disease, said Emmanuel Abate, General Manager of Genomics & Cellular Research, GE Healthcare Life Sciences. By combining this flexibility and precision of the BioAssemblyBot with the image quality and speed of the IN Cell Analyzer 6500 HS confocal screening platform, the prospect of automating high content screening in 3D models can become a reality.

Currently, biopharmaceutical companies test their drugs in 2D models and animal models. Precise 3D models provide a more physiologically relevant environment for drug testing because they mimic human reactions. The power of both of these platforms brings a new level of efficiency, speed and quality with assay designs and 3D biofabrication, said Michael Golway, President & CEO of ASLS.

Traditional 3D bioprinters are not designed for quality or interoperability with the high-throughput screening methods that pharmaceutical developers use to identify drug candidates. This alliance will result in a new product to address this challenge: an integration of GE Healthcare Life Sciences IN Cell Analyzer confocal imaging platform with IN Carta cell analysis software, and ASLS BioAssemblyBot 3D bioprinter with TSIM design software.

For pharmaceutical companies, where the average time to develop a new drug candidate may take over seven years, moving from traditional stage-gate testing processes to a lean, agile workcell for 3D tissue fabrication and assessments will shorten development timelines. The integration between IN Cell Analyzer and BioAssemblyBot enables the automated inclusion of cellular imaging information into the tissue modeling process so that new therapies can be scaled more quickly and effectively.

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GE Healthcare pairs up with Advanced Solutions on regenerative tissue manufacturing - 3DPMN

Gene therapy, once dismissed as too dangerous, has made a comeback, with two products approved in the U.S. since December 2017 and hundreds more in the pipeline. STATs latest report takes a deep dive into a crucial component of these new treatments: the viral vectors used to deliver gene therapies to cells and organs.

As dozens of new gene therapies near the market, we spoke with academic experts, pioneers in the field, and executives with 18 companies, large and small, to identify the most important challenges surrounding the engineering of better vectors, their safety, effectiveness, efficiency, production, and cost and how key players are thinking about overcoming those hurdles.

These engineered viruses are difficult to manufacture, particularly at the massive scale needed for some indications. Scientists are working hard to bring down the cost and speed up the process of making viral vectors, so that all the patients that could benefit from gene therapy will have access to it.

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Beyond the introduction, this report has four major components:

The basics of viral vectors and the history of their development;

Major challenges in the development, manufacturing, and testing of viral vectors, and possible solutions;

A close look at the status of gene therapies in 10 disease categories that are advancing through preclinical studies or are being tested in early-stage clinical trials;

And perspective on the U.S. Food and Drug Administrations approach to regulating viral vectors.

The report The STAT guide to viral vectors, the linchpin of gene therapy is intended for anyone with a strong interest in gene therapy, including biotech executives, investors, scientists, lawyers, policymakers, and patients and families interested in learning more. Our aim is to make the problems, stakes, and possibilities clear to everyone.

To buy the full report, please click here.

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New STAT report explores viral vectors, the linchpin of gene therapy - STAT - STAT

Earlier this year, the US Food and Drug Administration approved the most expensive drug ever to hit the market, a gene therapy for spinal muscular atrophy. SMA is a neuromuscular disorder that, in severe cases, can lead to infant death. The genetic correction is currently used to treat affected newborns, but as symptoms for some types of SMA may appear before birth, an earlier treatment would be potentially more effective.

In a study published December 4 in Molecular Therapy, researchers were able to fix a mutation in the survival motor neuron 1 (SMN1) genewhich causes SMA in humansin mice modelling the disease, while they were still inside their mothers uterus. The treated mice lived longer and had fewer symptoms than untreated animals.

Tippi MacKenzie, a fetal and pediatric surgeon at the University of California, San Francisco, who did not participate in this study, says it is an important paper because it is the first time fetal gene therapy has succeeded in SMA mice. Before you even think about doing something in patients, you have to first do it in the disease model of the mouse . . . so this group has supplied a very important piece to the literature, she adds.

SMN1encodes an essential protein for the maintenance of motor neurons, which are nerve cells in the brain and spinal cord responsible for controlling muscle movement. The result in children with mutations in the gene is the loss of motor neurons, leading to muscle weakness and associated complications. SMA affects one out of every 6,000 to 10,000 babies.

Correcting the SMN1 sequence is a potentially efficient treatment for those born with SMA. Zolgensma, the recently approved medication for this disorder, consists of an intravenous administration of an adeno-associated virus that ferries a functional copy of the SMN1 gene to the brain.

To see if the same fix could be accomplished before birth, the research team tested two different injection methods: one into the placenta (intraplacental or IP) and the other into one of the brain lateral ventricles (intracerebroventricular or ICV). The latter proved to be more effective. By injecting the viral vector into the fetuss brain, the virus will go directly into the cerebrospinal fluid, and it will transduce motor neurons in the spinal cord with a very high efficiency, compared to the IP [injection], says Afrooz Rashnonejad. who participated in this study while working at Ege University in Izmir, Turkey, but has recently moved to Nationwide Childrens Hospital in Columbus, Ohio.

Rashnonejad and her colleagues then monitored the injected mice that were carried to term. Those treated with the vector carrying a functional copy of SMN1 lived a median lifespan of 63 or 105 days (depending on the type of cassette carrying the gene), much longer than untreated SMA mice, which did not survive more than 14 days, but still less than wildtype pups, which had a median lifespan of 405 days. The treated mice were also heavier than untreated mice, but smaller than healthy mice.

The investigators also observed differences at the cellular and molecular levels. SMN protein levels were completely recovered in the brain and spinal cord, and the number of motor neurons was higher in treated animals.

I was just very impressed by what theyve done, says Simon Waddington, a gene therapy researcher at University College London who did not participate in this work, but was one of the reviewers of the paper. He adds that he and other colleagues had previously tried fetal gene therapy on SMA mice, but had failed as it is a technically difficult experiment. So it was really nice to see this group actually did a really good job.

This is the first time viral vectors have been used to successfully boost gene expression in SMA mice before birth. Interventions to edit the genome in utero have been previously used in mice that model other severe genetic diseases. Last year, for instance, Waddington and colleagues used fetal gene therapy to treat mice affected by Gaucher disease, a neurodegenerative disorder that can be fatal for newborns. Other successful attempts include intrauterine gene editing for mice affected by -thalassemia, an inherited blood disorder, and mice suffering a monogenic lung disease that normally results in newborn death.

MacKenzie says that, in a recent national meeting on in utero gene therapy, it was discussed how to move forward with a clinical application to the FDA. We are definitively moving towards that direction, but we dont have a particular application yet, because its still not clear which disease should be the first.

SMA makes a lot of sense because its so severe, MacKenzie adds. But at the same time, the results that are coming out at conferences, she observes, suggest that newborn babies receiving Zolgensma are doing pretty well, better than anybody could have imagined. So its not clear that you have to go before birth. A good candidate, she explains, would be a very rare type of SMA, where the baby dies before birth.

Waddington says that researchers might have to wait for neonatal gene therapy to become standard for certain diseases before using fetal gene therapy in humans. Once we actually understand how efficient this is, and if we come to the point where we discover that the earlier that you go the more effective it is . . . in a human setting, then we may be able to do fetal gene therapy. I think that we are looking at more than five years away before thats even likely to happen, he hypothesizes.

A. Rashnonejad et al., Fetal gene therapy using a single injection of recombinant AAV9 rescued SMA phenotype in mice,Molecular Therapy, 27:212333, 2019.

Alejandra Manjarrez is a freelance science journalist. Email her atalejandra.manjarrezc@gmail.com.

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Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy - The Scientist

Law360 (December 11, 2019, 1:28 PM EST) -- The gene therapy industry is in an exciting phase of growth, undergoing significant mergers and acquisitions activity, product sales and new marketing authorizations that are being issued with increasing regularity globally.

Recent reports have estimated that the market is likely to be almost four times its current value by 2025[1], with up to 20 new product approvals expected every year[2].

This rapid growth brings inevitable challenges. Significant issues relating to regulatory standards in manufacturing plants, establishing acceptable reimbursement policies and antitrust investigations are among a few.

The intellectual property landscape has been lower profile, with the exception of the ongoing CRISPR...

In the legal profession, information is the key to success. You have to know whats happening with clients, competitors, practice areas, and industries. Law360 provides the intelligence you need to remain an expert and beat the competition.

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The Rise Of Patent Wars In Europe's Gene Therapy Space - Law360

An effective andinnovative way to treat people with sickle cell anemia using gene therapy may soon be available thanks to efforts by several pharmaceutical companies, a Bloomberg report says.

Sickle cell anemia, a genetic defect that causes red blood cells to form in theshape ofa sickle, hinders the bodys ability to adequately distribute oxygen. This is due to atypical hemoglobin molecules, which is the protein in blood that transports oxygen. Sickle cell disease can be extremely painful, causing blood cells to get trapped in blood vessels and lead to heart failure, debilitating fatigue, strokes and blood clots.About 100,000 people suffer from sickle cell anemia in the U.S,with African Americansbeing disproportionately affected by this condition.

New developments with gene therapy, however, could work to have a positive impact on these symptoms. One of the innovative manufacturers, Bluebird Bio, stole the show at the annual conference of the American Society of Hematology in Florida. Its product, LentiGlobin, debuted positive results; in 17 patients treated with LentiGlobin,more than 40 percent of the hemoglobin in patients' red blood cells appearedin a healthier form thanks to gene therapy, per the article.

Bluebird isnt the only biotechnology making strides in gene therapies. Another potential treatment being researched is based on the technology called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a gene-editing tool that is being used for a wide range of biomedical applications.

Documented in an NPR report, sickle cell patient Victoria Gray recently became the first person in the U.S. to have billions of her own cells genetically edited with CRISPR and reintroduced into her body. These cells will hopefully produce fetal hemoglobin to compensate for the faulty hemoglobin in Grays red blood cells. The trial is being expanded to include more patients and is being conducted by Vertex Pharmaceuticals and CRISPR Therapeutics of the Boston area.

Current treatments for sickle cell include blood and bone marrow transfusions and medication. Studies on gene therapy treatments have been encouraging so far, but there is more testing to be done before either CRISPR or LentiGlobin hits the market.

Link:
Gene therapy could be a revolutionary new treatment for sickle cell disease - The Hill

Fox News senior judicial analyst Judge Andrew Napolitano on the potential fallout from reports DNA-testing company Family TreeDNA will share data with the FBI in an effort to solve crimes.

Libella Gene Therapeutics is charging volunteers $1 million to undergo clinical trials of a treatment it is working on that is designed to prevent, delay or even reverse aging.

However, participants will be required to go to a small clinic in Cartagena, Colombia, to participate, which the Kansas-based company said was the easiest site among eight different countries it looked into, calling it the path of least resistance.

In a press release, a company executive said traditional clinical trials in the U.S. take years and millions or even billions of dollars.

The treatment would be delivered intravascularly and participants will be monitored over the course of a year, according to the company's website. Gene therapy treatments are intended to be one-off treatments, attacking the problem at its source.

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The technology focuses on lengthening telomeres, which are structures found at the end of chromosomes. Their main function is to protect DNA during cell division.

Every time a cell divides, a part of the telomere is lost until it becomes too short and the cell dies. Some believe that as cells age, so does the body.

Telomerase is an enzyme that lengthens telomeres and thus prevents the cell from dying.

Libellas technology rebuilds the ends of telomeres, andthereby affects the aging process.

I know what were trying to do sounds like science fiction, but I believe its a science reality, Jeff Mathis, CEO of Libella Gene Therapeutics, said in an interview with OneZero.

The treatment may potentially treat other diseases, like cancer and Alzheimers.

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Not everyone, however, agrees that lengthening telomeres will have any effect on the aging process. For example, researchers at the University of Utah were unable to conclude whether shorter telomeres were simply a sign of aging or actually a contributor to the process.

Dr. Andrew Stern, who is one of the founders of Libella Gene Therapeutics, was also one of the principal discoverers of portions of human telomerase.

In order to be eligible for the trial, individuals must be 45 years or older. So far the company has recruited two people, according to the OneZero interview published on Medium.

The study will look into the change in the length of telomeres, and into the incidence of serious adverse events.

The FDA declined to comment specifically on Libella Gene Therapeutics and its decision to hold its trial outside of the U.S. It does, however, accept foreign clinical data and results so long as certain conditions are met.

A spokesperson for Libella Gene Therapeutics did not return FOX Business request for comment.

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Would you pay $1M to partake in an anti-aging gene therapy trial? - Fox Business

One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune responses.

A team from Johns Hopkins Medicine is proposing an alternative method for transporting large therapies into cellsincluding genes and even the gene-editing system CRISPR. Its a nano-container made of a polymer that biodegrades once its inside the cell, unleashing the therapy. The researchers described the invention in the journal Science Advances.

The team, led by biomedical engineer Jordan Green, Ph.D., was inspired by viruses, which have many properties that make them ideal transport vehicles. They have both negative and positive charges, for example, which allows them to get close to cells. So Green and his colleagues developed a polymer containing four molecules with both positive and negative charges. They used it to make a container that interacts with the cell membrane and is eventually engulfed by it.

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RELATED: Could a grape-based compound improve gene therapy efficiency?

The Hopkins researchers performed four experiments to prove the nanocontainers would travel into cells and deliver complex therapies once inside. First, they packaged a small protein into the polymer material and mixed it with mouse kidney cells in a lab dish. Using fluorescent tags, they confirmed that the protein made it into the cells. Then they repeated the experiment with a much larger medicinehuman immunoglobulinand observed that 90% of the kidney cells received the treatment.

From there, they made the payload even bulkier, packaging the nanocontainers with the gene-editing system CRISPR. With the help of fluorescent signals, they were able to confirm that CRISPR went to work once inside the cells, disabling a gene 77% of the time.

"That's pretty effective considering, with other gene-editing systems, you might get the correct gene-cutting result less than 10 percent of the time," said graduate student Yuan Rui in a statement.

Finally, the Hopkins researchers injected CRISPR components into mouse models of brain cancer using the polymer nanocontainers. Again they saw evidence that successful gene editing had occurred.

Developing improved methods for gene therapy is a priority in the field. In October, for example, scientists at Scripps Research described a way to use a small molecule called caraphenol A to lower levels of interferon-induced transmembrane (IFITM) proteins, which could, in turn, allow viral vectors to pass more easily into cells. And earlier this year, an Italian team described a method for including the protein CD47 in lentiviral vectors to improve the transferring of therapeutic genes into liver cells.

The next step for Hopkins researchers Rui and Green is to improve the stability of the nanocontainers so they can be injected into the bloodstream. They hope to be able to target them to cells that have certain genetic markers, they reported.

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Hopkins team invents non-viral system for getting gene therapy into cells - FierceBiotech

Pfizer moved into gene therapies earlier than some of its peers, partnering with Spark Therapeutics in 2014 and paying close to $200m (180m) upfront to acquire Bamboo Therapeutics two years later. The Bamboo takeover gave Pfizer ownership of a manufacturing facility in North Carolina, US.

Earlier this year, Pfizer doubled down on in-house production of gene therapies, committing $500m to expand its footprint in North Carolina.

Talking at a recent investor conference, Mikael Dolsten, chief scientific officer at Pfizer, said the spending commitment is, in part, a reflection of a belief that keeping production in-house will deliver better results than relying on third parties.

Dolsten said, When we compare that with what we get from other companies, we think we can really improve the yield, the purity and the characterization of the product.

Across the industry, poor yields have exacerbated capacity constraints created by the rapid expansion of the gene therapy pipeline, turning quality manufacturing capacity into a sought after resource.

A desire to possess in-house manufacturing capacity was a factor in many of the recent acquisitions of gene therapy companies, such as Astellas $3bn takeover of Audentes Therapeutics.

Gene therapy startups, such as Audentes and Bamboo, bypassed the limitations of contract capacity by establishing internal capabilities. Those capabilities enabled the companies to advance their gene therapies and, ultimately, to attract takeover offers, but their creation required the sort of upfront investments in infrastructure that many venture-backed startups typically try to avoid.

Through its $500m gene therapy investment, Pfizer thinks it can provide an alternative for startups that are struggling to access high-quality contract capacity but are unable or unwilling to build their own facilities.

Dolsten said, We think it's a competitive advantage, not just for our product, but for companies that want to partner with Pfizer that may allow them to have an easier and more high-end dialogue with regulators across the globe about this new field and a new type of product.

If Dolsten is right, the North Carolina manufacturing capacity could give Pfizer an edge when it tries to partner with gene therapy startups that have other options open to them, such as alliances with rival drugmakers and contract manufacturing organizations.

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Pfizer to bring gene therapy production in-house - BioPharma-Reporter.com

Dr. James Wilson is a pioneer in gene therapy. That does not mean he is necessarily impressed with the current state of affairs.

In five years, when we look back on the way were executing on gene therapy now, were going to realize that things are going to be very different, Wilson said at the STAT Summit in Cambridge, Mass., recently. The way in which were going to treat Duchenne muscular dystrophy, potentially cure it, is not the way in which its being evaluated in the clinic now.

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Dr. James Wilson, a scientific pioneer, on the future of gene therapy - STAT - STAT

Abstract / Synopsis:

Two anti-VEGF gene therapies are being investigated in clinical trials of patients with exudative age-related macular degeneration. Initial efficacy and safety results are encouraging.

Anti-VEGF gene therapy for exudative age-related macular degeneration (AMD) has transformative potential for reducing treatment burden and improving patient outcomes, according to Szilrd Kiss, MD.

Two investigational anti-VEGF gene therapies are currently being investigated in clinical trialsRGX-314 (Regenxbio) and ADVM-022 (Adverum). Dr. Kiss described the two technologies and reviewed some preliminary clinical trial results that support their promise for providing sustained benefit with a single injection.

Considering the treatment burden of anti-VEGF therapy for other ocular diseases, we can imagine that exudative AMD is just the first indication that will be targeted for anti-VEGF gene therapy, said Dr. Kiss, chief, Retina Service, associate professor of ophthalmology, and associate dean at Weill Cornell Medical College, New York, NY.

RGX-314 delivers a gene for an anti-VEGF fab protein that is similar to ranibizumab. It uses adeno-associated virus-8 (AAV8) as a vector and is administered in the operating room as a subretinal injection.

AAV is the most common viral vector carrier used for gene therapy. Different AAV serotypes have different tissue selectivity, Dr. Kiss explained. AAV8 is a wild type AAV that has the propensity for greater transfection of retinal cells compared with AAV2 following subretinal gene therapy delivery.

RELATED:AAO 2019: Encouraging results revealed from early trial of subretinal gene therapy for wet AMD

Disclosures:

Szilrd Kiss, MDe: [emailprotected]This article was adapted from Dr. Kiss presentation at the 2019 meeting of the American Academy of Ophthalmology. Dr. Kiss is a consultant to RegenxBio and Spark Therapeutics and is a consultant and equity owner in Adverum.

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Research targets gene therapy for exudative AMD patients - Modern Retina

Its said that nothing is certain except death and taxes. But doubt has been cast over the former since the 1970s, when scientists picked at the seams of one of the fundamental mysteries of biology: the molecular reasons we get old and die.

The loose thread they pulled had to do with telomeresmolecular timepieces on the ends of chromosomes that shorten each time a cell divides, in effect giving it a fixed life span. Some tissues (such as the gut lining) renew almost constantly, and it was found that these have high levels of an enzyme called telomerase, which works to rebuild and extend the telomeres so cells can keep dividing.

That was enough to win Elizabeth Blackburn, Carol Greider, and Jack Szostak a Nobel Prize in 2009. The obvious question, then, was whether telomerase could protect any cell from agingand maybe extend the life of entire organisms, too.

While telomere-extending treatments in mice have yielded intriguing results, nobody has demonstrated that tweaking the molecular clocks has benefits for humans. That isnt stopping one US startup from advertising a telomere-boosting genetic therapyat a price.

Libella Gene Therapeutics, based in Manhattan, Kansas, claims it is now offering a gene therapy to repair telomeres at a clinic in Colombia for $1 million a dose. The company announced on November 21 that it was recruiting patients into what it termed a pay-to-play clinical trial.

Buyer beware, though: this trial is for an unproven, untested treatment that might even be harmful to your health.

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The company proposes to inject patients with viruses carrying the genetic instructions cells need to manufacture telomerase reverse transcriptase, a molecule involved in extending the length of telomeres.

The dangers are enormous, says Jerry Shay, a world expert on aging and cancer at the University of Texas Southwestern Medical Center. Theres a risk of activating a pre-cancerous cell thats got all the alterations except telomerase, especially in people 65 and over.

For years now, people involved in the company have made shifting claims about the study, raising uncertainty about who is involved, when it might start, and even where it would occur. Trial listings posted in October to clinicaltrials.gov currently show plans for three linked experiments, each with five patients, targeting critical limb ischemia, Alzheimers, and aging, respectively.

Jeff Mathis, president of Libella, told MIT Technology Review that two patients have already paid the enormous fee to take part in the study: a 90-year-old-woman and a 79-year-old man, both US citizens. He said they could receive the gene therapy by the second week of January 2020.

The decision to charge patients a fortune to participate in the study of an experimental treatment is a red flag, say ethics experts. Whats the moral justification for charging individuals with Alzheimers? asks Leigh Turner, at the University of Minnesotas Center for Bioethics. Why charge those bearing all the risk?

The telomere study is occurring outside the US because it has not been approved by the Food and Drug Administration. Details posted to clincaltrials.gov indicate that the injections would be carried out at the IPS Arcasalud SAS medical clinic in Zipaquir, Colombia, 40 kilometers (25 miles) north of Bogot.

It takes a lot longer, is a lot more expensive, to get anything done in the US in a timely fashion, Mathis says of Libellas choice to go offshore.

To some promoters of telomerase gene therapy, urgency is justified. Heres the ethical dilemma: Do you run fast and run the risk of low credibility, or move slowly and have more credibility and global acceptancebut meanwhile people have died? says Mike Fossel, the president of Telocyte, a company planning to run a study of telomerase gene therapy for Alzhheimer's in the US if it can win FDA signoff.

Our reporting revealed a number of unanswered questions about the trial. According to the listings, the principal investigatorwhich is to say the doctor in charge--is Jorge Ulloa, a vascular surgeon rather than an expert in gene transfer. I dont see someone with relevant scientific expertise, says Turner.

Furthermore, Bill Andrews, who is listed as Libellas chief scientific officer, says he does not know who Ulloa is, even though on Libellas website, the mens photos appear together on the list of team members. He said he believed that different doctors were leading the trial.

Turner also expressed concerns about the proposed 10-day observation period described in the posting for the overseas study: If someone pays, shows up, has treatment, and doesnt stick around very long, how are follow-up questions taking place? Where are they taking place?

Companies seeking to try the telomere approach often point to the work of Maria Blasco, a Spanish scientist who reported that telomere-lengthening gene therapy benefited mice and did not cause cancer. Blasco, director of the Spanish National Centre for Cancer Research, says she believes many more studies should be done before trying such a gene experiment on a person.

This isnt the first time Libella has announced that its trial would begin imminently. It claimed in late 2017 that human trials of the telomerase therapy would begin in the next few weeks. In 2016, Andrews (then partnered with biotech startup BioViva) claimed that construction of an age reversal clinic on the island nation of Fiji would be complete before the end of the year. Neither came to pass.

Similar questions surround Libellas most recent claims that it has two paying clients. Pedro Fabian Davalos Berdugo, manager of Arcasalud, said three patients were awaiting treatment in December. But Bioaccess, a Colombian contract research organization facilitating the Libella trial, said that no patients had yet been enrolled.

Also unclear is where Libella is obtaining the viruses needed for the treatment. Virovek, a California biotech company identified by several sources as Libellas manufacturer, did not answer questions about whether any treatment had been produced.

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Buyer beware of this $1 million gene therapy for aging - MIT Technology Review

Dec. 11, 2019 14:00 UTC

BERKELEY, Calif.--(BUSINESS WIRE)-- GenEdit Inc., a developer of a novel polymer nanoparticle technology platform for non-viral- and non-lipid-based delivery of gene therapies, today announced that it has entered into a worldwide, exclusive license and collaboration agreement with Editas Medicine, Inc., a leading genome editing company. GenEdit has developed a comprehensive delivery system for CRISPR-based therapeutics, including gene knockout and gene repair therapies, to enable safer delivery options with improved efficiency.

"This license and collaboration agreement further validates the strength of our intellectual property portfolio and the potential of GenEdits technology," said Kunwoo Lee, Ph.D., co-founder and chief executive officer of GenEdit. "We are pleased to establish our relationship with Editas Medicine as they leverage our technology to develop potential genomic medicines."

Under the terms of the agreement, GenEdit has granted Editas Medicine an exclusive worldwide license, with rights to sublicense, to GenEdits Cpf1-based technologies. In return for these rights, GenEdit will receive undisclosed upfront and development milestone payments, including royalties on net sales of products incorporating the licensed intellectual property. In addition, GenEdit and Editas Medicine will collaborate on evaluating delivery of Cpf1-based technologies with GenEdits nanoparticle platform. Editas Medicine will provide research funding and have an option to continue development after the initial collaboration period.

GenEdits nanoparticle platform consists of a proprietary non-viral, non-lipid library of polymers that efficiently encapsulate and deliver cargo [RNA, DNA, protein and/or ribonucleic acid-protein complexes (RNP)] to specific tissues. The company screens the library to identify initial hits and then uses computational analysis and medicinal chemistry for iterative lead optimization. The company has used this platform to identify multiple candidate polymers for efficient and specific delivery of gene editing to a range of tissues.

"Compared to viral vectors and lipid-based nanoparticles, our approach has the potential for better targeting, more cargo, and lower manufacturing cost," said Timothy Fong, Ph.D., chief scientific officer of GenEdit. "In particular, our approach has the potential to enable in vivo gene editing of multiple tissues with CRISPR and expand the potential of gene therapies to treat more diverse sets of diseases."

About GenEdit

GenEdit was founded to transform the delivery of gene and gene editing therapies. We have synthesized the NanoGalaxy library of polymers that can encapsulate RNA, DNA, protein and/or RNP. Through advanced screening methods, computational analysis and iterative medicinal chemistry, we have demonstrated efficient delivery of gene editing cargo to specific tissues. We seek development partnerships for specific tissues and/or gene targets while advancing our internal pipeline of gene editing therapies.

For more information, please visit http://www.genedit.com.

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GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery - BioSpace

An American biotech company has launched clinical trials in Colombia to test a new therapy designed to reverse the aging process, and in turn, treat age-related diseases, according to news reports.

But to steal a sip from this purported fountain of youth, participants in the trial must first fork over $1 million a fee that seems even more astronomical when you consider that most clinical trials are either free or provide participants with financial compensation, according to a report by OneZero, a Medium publication about tech and science.

The pricey trial is being run by Libella Gene Therapeutics, a Kansas-based company whose website proclaims that "the future is here." The company announced its intention to test its anti-aging remedies in Cartagena, Colombia, in 2018, and began recruiting for the trials in October of this year. Using a single-gene therapy, Libella aims to "prevent, delay, or even reverse" the general effects of aging, as well as treat diseases that emerge in old age, such as Alzheimer's, according to ClinicalTrials.gov.

In fact, in its own press release, the company boasted, without evidence, that its gene therapy "may be the world's first cure for Alzheimer's disease." The bold claim raises an obvious question: Will the treatment actually work?

Short answer: No one really knows, but the fact that Libella shipped its operation beyond the reach of the U.S. Food and Drug Administration (FDA) doesn't inspire confidence, experts told OneZero.

Related: 5 Reasons Not to Fear Getting Older

Unlike anti-aging face creams that soften the superficial signs of aging, the Libella therapy aims to reverse aging from the ground up, so to speak, starting at the level of our genes. Specifically, the gene therapy is intended to lengthen patients' telomeres structures that cap the tips of chromosomes and prevent the genetic material inside from fraying. Telomeres grow shorter each time a cell divides, and when the structures reach a critical length, cells either stop dividing or perish, according to Stanford Medicine.

The theory goes, if you rebuild the body's shortened telomeres, the process of aging might be thrown in reverse. This is not a new idea. Several studies in mice suggest that using gene therapy to lengthen telomeres can reverse certain signs of aging in the animals. A 2015 study from Stanford prompted similar effects in isolated human cells; the treatment lengthened cells' telomeres by fiddling with a close cousin of DNA, called RNA, which helps cells build proteins.

The Libella therapy aims to help cells rebuild telomeres by activating a gene in their DNA that would normally be switched "off." The gene, called TERT, contains instructions to build a protein called "telomerase," an enzyme that adds molecules to the end of telomeres and prevents the structures from shortening during cell replication, according to a 2010 report in the journal Biochemistry.

Libella's lead scientific officer, molecular biologist William Andrews, originally helped identify the human telomerase enzyme at the biotech firm Geron. Later, he licensed a gene therapy based on the finding to Libella, according to OneZero. "I can't say [telomere shortening is] the only cause of aging, but it plays a role in humans," Andrews told the publication.

Related: 8 Tips for Healthy Aging

Andrews' therapies will soon be put to the test in Colombia, where one 79-year-old will receive the anti-aging treatment in next month, according to OneZero. The anti-aging trial will include four more participants over age 45 and focus on verifying that the treatment is "safe and tolerable," meaning it does not harm patients or cause unacceptable side effects.

Two more trials will use the same therapy but aim to "prevent, delay, or even reverse the development" of Alzheimer's disease and critical limb ischemia, an age-related condition in which a person's arteries become severely obstructed. Participants in these trials must already be diagnosed with the disorders.

After treatment, participants in all three trials will remain in the clinic for 10 days for further monitoring, and then return at regular intervals for checkups over the following year.

Libella's gene therapy involves a one-time injection delivered through an IV; the Alzheimer's therapy uses the same formula but doctors inject the product into the patient's spinal fluid. Within the product, a modified virus carries the TERT gene into cells and injects the genetic material into their DNA. The modified viruses cannot transmit diseases to people, but in high enough doses, the germs could provoke a harmful immune response in the patient, according to a 2018 animal study. Libella representatives declined to say how high a dose their clinical trial participants will receive.

"All I can say is, it's a lot," Andrews told OneZero.

Potential side effects aside, the fact that the Libella treatment will be administered beyond the purview of the FDA is telling, according to one expert. Leigh Turner, a bioethicist at the University of Minnesota, told OneZero that "even though the company is based in the United States, they've managed to find a way to evade U.S. federal law by going to a jurisdiction where it's easier to engage in this activity."

The $1 million entry fee is also alarming, Turner said, given that most clinical trials don't charge patients anything to enter. Andrews told OneZero that the fee is justified because it costs the company hundreds of thousands of dollars to make enough product to treat just one person.

The appearance of the trials on ClinicalTrials.gov, an official registry maintained by the National Institutes of Health, does not boost their credibility, she added. The automated database can be easily manipulated and "can basically be used as a marketing platform," she said.

Other stakeholders in the telomere-lengthening business are concerned, too. Michael Fossel, founder and president of the biotech startup Telocyte, told OneZero that his company's own therapy is similar to the Libella treatment the difference is that Telocyte is seeking approval through the FDA. "We're afraid that something will go wrong [with the Libella trials], whether it's from a safety or efficacy standpoint," he said.

Related: Extending Life: 7 Ways to Live Past 100

But even in a best case scenario, wherein no patients come to harm, the Libella therapy still might not deliver any notable health benefits. Some research suggests that no link exists between telomere length and aging.

For instance, a study published this year examined more than 261,000 people between age 60 and 70, and found no correlation between participants' telomere lengths and their age-related health outcomes, including their overall cognitive function, muscular integrity and the age of their parents. Long telomeres were associated with a lowered risk of coronary heart disease as compared with short telomeres, but longer telomere length was also linked to a heightened risk of cancer.

"Telomere lengthening may offer little gain in laterlife health status" and lead to an increased risk of cancer, the authors noted.

It remains to be seen whether Libella has truly tapped the fountain of youth, but given the dubious nature of their clinical trials, potential participants may want to exercise caution before relocating to Colombia and shelling out $1 million for a chance to live longer.

Originally published on Live Science.

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New Anti-Aging Clinical Trial Begins. For $1 Million, You Can Be a Participant. - Livescience.com

A gene therapy for bladder cancer that recently received $400 million in support from the private equity company Blackstone Group helped more than half of treated patients with resistant disease achieve remission.

The therapy, called nadofaragene firadenovec, was discovered by a Finnish-based research institute and first entered clinical study in 2012. The data revealed today at the Society of Urologic Oncology meeting came from a Phase 3 trial that is part of the agent's Biologics License Application now before the FDA.

Licensed by its original owner, FKD Therapies Oy, to Switzerland-based Ferring Pharmaceuticals, nadofaragene firadenovec is now in the hands of the U.S. subsidiary FerGene. That company was created with the Blackstone investment and an additonal $170 million from Ferring. FerGene will commercialize the gene therapy in the U.S., with Ferring holding rights elsewhere.

Nadofaragene firadenovec is an an adenovirus-based gene therapy encoding production of the immunity-stimulating protein interferon alfa-2b. Viral vectors containing the gene are administered by catheter once every three months into the bladder, where they are absorbed into cells in the organ's walls and begin stimulating interferon.

Delivery through a catheter, called intravesical administration, limits systemic exposure to both the viral vectors and to inteferon, said Neal Shore, medical director for the Carolina Urologic Research Center and an investigator in the trial.The side-effects of interferon include flu-like symptoms in patients who inject it for other conditions like multiple sclerosis.

The clinical trial enrolled 157 patients with bladder cancer that has not spread to muscle walls and has stopped responding to treatment with Bacillus Calmette-Gurin vaccine.

Alternative treatments for these patients include chemotherapy or a procedure called "complete cystectomy." This surgery entails complete removal of the bladder, which in men means removal of the prostate and seminal vesicles and in women the uterus, ovaries, fallopian tube and part of the vagina.

"Radical cystectomy is one of the most invasive surgeries we do not just in urology but in all of surgery," Shore said, requiring a lengthy hospital stay and having a high rate of post-procedural complications.

Out of a group of 103 patients with superficial tumors in the bladder wall, just over half were in complete remission at three months, 41% at six months, and 24% at one year. In a group of 48 patients whose cancer had spread to the connective tissue outside the bladder, 73% had no recurrence of serious disease at three months, which fell to 44% at 12 months.

In this type of bladder cancer, the FDA has said a single-arm trial, without a placebo control, using complete remission is sufficient to be considered for approval, and the study does not need to pre-specify a rate that would define success. "The natural history of [disease]is well understood, and the complete response rate is negligible in the absence of therapy," the agency said in guidelines published in February 2018.

One chemotherapy agent, called Valstar (valrubicin), is approved for this patient group. It won FDA approval on a complete response rate of 18%.

In seeking FDA approval, nadofaragene firadenovec is in a race with Merck & Co.'s Keytruda (pembrolizumab) to achieve approval first. That immuno-oncology agent tested Keytruda in a similar population in the Keynote-057 trial, in which it achieved a 39% complete response rate.

Keytruda will be the subject of a meeting of the FDA's Oncologic Drugs Advisory Committee on Dec. 17.

Aside from the remission rates,Shore said nadofaragene firadenovec would differentiate itself from Keytruda in practice because its intravesical delivery means it could be administered by community-based urologists at outpatient clinics.

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Cancer gene therapy backed by Blackstone gets trial win - BioPharma Dive

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

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Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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A #ReUp of 2019: The year when gene therapy, DNA modifications came of age & saved lives - Economic Times

Ru-Yin Hu,13,* Xiao-Bin Tian,3,* Bo Li,3 Rui Luo,3 Bin Zhang,3 Jin-Min Zhao1

1Department of Orthopaedics, Guangxi Medical University, Nanning 530021, Peoples Republic of China; 2Department of Orthopaedics, The First Affiliated Hospital of Guangxi Medical University, Nanning 530021, Peoples Republic of China; 3Department of Orthopaedics, Guizhou Provincial Peoples Hospital, Guiyang 550002, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Jin-Min ZhaoDepartment of Orthopaedics, Guangxi Medical University, No. 22 Shuangyong Road, Nanning, Guangxi 530021, Peoples Republic of ChinaTel +86 771 13985048001Email zhao948586007@126.com

Background: Existing drugs are far from enough for investigators and patients to administrate the therapy of rheumatoid arthritis. Drug repositioning has drawn broad attention by reusing marketed drugs and clinical candidates for new uses.Purpose: This study attempted to predict candidate drugs for rheumatoid arthritis treatment by mining the similarities of pathway aberrance induced by disease and various drugs, on a personalized or customized basis.Methods: We firstly measured the individualized pathway aberrance induced by rheumatoid arthritis based on the microarray data and various drugs from CMap database, respectively. Then, the similarities of pathway aberrances between RA and various drugs were calculated using a KolmogorovSmirnov weighted enrichment score algorithm.Results: Using this method, we identified 4 crucial pathways involved in rheumatoid arthritis development and predicted 9 underlying candidate drugs for rheumatoid arthritis treatment. Some candidates with current indications to treat other diseases might be repurposed to treat rheumatoid arthritis and complement the drug group for rheumatoid arthritis.Conclusion: This study predicts candidate drugs for rheumatoid arthritis treatment through mining the similarities of pathway aberrance induced by disease and various drugs, on a personalized or customized basis. Our framework will provide novel insights in personalized drug discovery for rheumatoid arthritis and contribute to the future application of custom therapeutic decisions.

Keywords: rheumatoid arthritis, drug repositioning, individualized pathway aberrance, differential pathway

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Individualized Drug Repositioning For Rheumatoid Arthritis Using Weigh | PGPM - Dove Medical Press

Joint pain in winter can be reduced by keeping yourself covered with layers of warm clothes

Joint pain in winter: Arthritis is surely a difficult time for people suffering from arthritis. Not only does joint pain worsen during the cold winter months, patients also experience more joint stiffness and reduced range of motion. Proper health care, diet and lifestyle measures can together help arthritis patients deal with worsening symptoms this time of the year. The link between temperature drop and worsening of joint pain is still unclear and needs more research. However, a few tips and tricks can help improve quality of life and day-to-day functioning in arthritis patients in winter.

Dressing up appropriately by covering yourself top to bottomwith proper layers, can help you keep warm and reducedebilitating joint pain. Wear gloves and extra layers on your knees and legs to keep them warm and protected. Wearing multiple layers protect you from fluctuations in temperature.

Keep yourself warm and covered during the cold winter monthsPhoto Credit: iStock

Also read:Start Your Day With This Golden Drink To Prevent Bloating, Lose Weight And Reduce Joint Pain

Well, this is one important step in all seasons. Drinking sufficient water can prevent muscle cramps, keep your immunity in check and also prevent incidence of diseases. Drinking water time to time can help you be more active. Also, even mild dehydration can make you more sensitive to pain so make sure your water intake is optimum. Apart from drinking water, you can also include chicken soups, bone broth, vegetable soups, bone broth, etc in your diet to keep your hydration in check.

Being overweight or obese can make you feel lazier and less active. An effective way to deal with arthritis pain is by keeping yourself active and alsolosing weight if required. Make sure you exercise regularly. Include both cardio and weight training exercises in your routine. They will keep you warm and prevent worsening of arthritis symptoms. If going to the gym in cold weather seems too difficult a task, then exercise indoors. The idea is to not skip exercising for better management of arthritis.

Also read:Achieve Your Weight Loss Goals This Winter By Adding These Seasonal Fruits To Your Diet

There is nothing more comforting than a warm bath in winter. Warm baths can provide relief to arthritis patients, according to the Arthritis Foundation. Warm baths can relax your muscles and help you feel calm. Just don't step directly in cold after taking the bath. Your body needs some time to normalise temperature after a warm bath. Cover yourself properly before you come out of the bathroom. Similarly, you can also opt for warm compresses in to deal with worsened joint pain.

Warm baths in winter can help in reducing joint pain in arthritis patientsPhoto Credit: iStock

Low levels of Vitamin D in the body can make you more sensitive to pain, especially in winter. Vitamin D deficiency also puts you at risk of osteoporosis. It is recommended to spend some time under the sun. Anything from 15 minutes to half an hour can help your body synthesise some amount of the sunshine vitamin.Besides, include Vitamin D-rich foods like eggs, mushrooms, fatty fish, milk and milk products in your diet. You can also opt for supplements, but only under the supervision of your doctor.

Also read:Signs And Symptoms Of Vitamin D Deficiency; Best Sources Of Vitamin D Other Than Sunlight

Disclaimer: This content including advice provides generic information only. It is in no way a substitute for qualified medical opinion. Always consult a specialist or your own doctor for more information. NDTV does not claim responsibility for this information.

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Joint Pain In Winter: 5 Tips To Deal With Joint Pain And Other Arthritis Symptoms In Cold Winter Months - NDTV News

COLUMBUS (WCMH) Did you know the No. 1 cause of disability in the United States is arthritis?

More than 54 million Americans live with arthritis including 2.8 million people in Ohio.

On Saturday, at Genoa Park by COSI over 1,000 people came together to rally behind the arthritis foundation.

The annualJingle Bell Run for arthritis is bringing holiday cheer to downtown Columbus. Theirgoal was to raise $205,000 this year.

There is often a misconception that arthritis only affects older people, but thats far from the truth.

Five months ago I was diagnosed with rheumatoid arthritis, my life was miserable in the mornings, said 18-year-old, Colten Phay.

When I would wake up my whole body would be aching with pain, miserablewith pain and I couldnteven eat breakfast or put on clothes on, Colton said.

With the help of the research from the Arthritis Foundation, Colten went on a medication they helped discover and now his life is back to normal. It changed my life, expressed Colten. It helps so much. I feel fine now.

Christopher Haverlock with the Arthritis Foundation says that this is something that is a big deal.

It affects more people in the country than any other disease.

He also told us the Jingle Bell Run is more than just a way to raise money.

An event like this is great because they can be around other people who understand what theyre going through. They can celebrateliving and saying yes to doing more things, explained Haverlock.

Taking place in more than 100 cities nationwide, with Columbus being a Top Five Race, the Arthritis Foundations Jingle Bell Run benefits the more than 54 million Americans (1 in 4 adults), including 300,000 children (1 in every 250), living with arthritis every day.

From funding cutting-edge research for new treatments and ultimately a cure, to advocating for health care access, the Arthritis Foundation helps those living with arthritis score everyday victories, one step at a time.

To learn more visit JBR.org/Columbus or contact the Arthritis Foundation at 614-362-7370

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More than 1,000 participate in annual Jingle Bell Run for arthritis - NBC4 WCMH-TV

The prevalence of arthritis in children with Down syndrome may be 2 to 3 times greater than previously reported, according to study data presented at the 2019 American College of Rheumatology/Association of Rheumatology Professionals (ACR/ARP) Annual Meeting, held November 8 to 13, 2019, in Atlanta, Georgia.

Investigators screened children (aged 0-21 years) with Down syndrome at a regional screening clinic, where a detailed musculoskeletal examination was performed by a pediatric rheumatology clinical fellow. Suspected cases of arthropathy of Down syndrome (A-DS) were confirmed by a second physician at an affiliated clinic. Children with arthropathy received treatment according to existing guidelines for juvenile idiopathic arthritis (JIA). Data from a convenience sample of 21 children newly diagnosed with JIA were collected and compared with the Down syndrome cohort.

Over 18 months, 503 children with Down syndrome were screened for arthritis, among whom 18 were newly diagnosed with A-DS. The total number of A-DS cases was 33, including children with a diagnosis prior to screening. Based on these results, prevalence of A-DS was indicated to be 20 in 1000. Significant delay in A-DS diagnosis was observed.

The majority of A-DS cases presented with polyarticular rheumatoid factor negative arthritis, with small joints of the hands and wrists predominantly affected. No children with A-DS were positive for antinuclear antibodies. Erosive changes were reported on radiographs in a significantly greater number of children with A-DS (42%) than with children with JIA (14%; P <.05). In the majority of A-DS cases, erythrocyte sedimentation rate and C-reactive protein levels were not helpful in arriving at a diagnosis.

These data support the addition of a musculoskeletal examination to the health surveillance guidelines for children with Down syndrome. Investigators also proposed a new clinical term to better capture A-DS: DS-associated arthritis. Further research in a larger cohort is necessary to describe the pathogenesis of DS-associated arthritis and to identify biomarkers.

Reference

Foley C, Deely D, MacDermott EJ, Killeen O. Arthropathy of Down syndrome: an under-diagnosed inflammatory joint disease that warrants a name change. Presented at: 2019 ACR/ARP Annual Meeting; November 8-13, 2019; Atlanta, GA. Abstract 1817.

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Prevalence of Arthropathy in Children With Down Syndrome Higher Than Previously Reported - Rheumatology Advisor