Will Dialysis Become a Thing of the Past? – Jewish Link of New Jersey

By Ken Stephens | February 06, 2020

A groundbreaking study has shown that it is possible to rejuvenate damaged kidneys and improve their function, a procedure that could reverse chronic kidney disease, offsetting the need for dialysis. This is the first breakthrough in decades to combat this disease, often precipitated by hypertension and diabetes, and which affects a whopping 10% of the population worldwide.

The study was conducted by Professor Benjamin Dekel, head of Pediatric Nephrology and the Pediatric Stem Cell Research Institute in the Edmond and Lily Safra Childrens hospital at Sheba Medical Center, and published this week in the prestigious Cell Reports medical journal.

In past studies, researchers discovered that the adult kidney constantly renews itself over time through the activity of colonies of cells that replace lost and degenerated cells in the kidney. Prof. Dekel and his team have now developed an innovative technology that involves the extraction of such healthy kidney cells from diseased kidneys. These cells are then expanded into large numbers within a laboratory environment. By generation of three-dimensional cultures called kidney spheres, the cells show improved function to generate new kidney tissue and replace lost cells. The new cells are then reintroduced into the kidney where they rebuild it, positively influencing neighboring cells and improving its function (see diagram).

One of the most significant aspects of the discovery is that the newly developed technology uses the patients own cells, thereby circumventing the need for immunosuppression as well as problems associated with immune rejection.

Thus far, the method has been tested on mice, where the cells have shown their ability to generate new renal structures, associated with an ability to be retained for a long time once administered into the host kidney. The treated mice displayed improved renal function.

By focusing on improving and stabilizing renal function, this treatment has the potential to help millions of patients with chronic kidney disease and who have yet to require dialysis treatment.

These astounding results will be studied in clinical trials on patients with renal failure by the KidneyCure Bio firm, which commercialized this technology.

Prof. Benjamin Dekel, who led the project said, The breakthrough in this technology, which was developed at the Sheba Medical Center, is not only in the ability to maintain the kidney-renewing cells outside the body, but also in the ability to multiply them and generate large numbers of cells and make them function properly using the 3-D cultures. This is important news for patients with chronic kidney disease, who hopefully will benefit from these discoveries in the coming years. The ability to generate new kidney tissue (to replace the damaged tissue) could help millions of patients worldwide who suffer from kidney disease.

The trailblazing research was carried out by senior researchers Dr. Orit Harari-Steinberg, Dr. Dorit Omer, and Ms. Yehudit Gnatek from the Pediatric Stem Cell Research Institute, under the leadership of Prof. Dekel.

Collaborators include: Dr. Zohar Dotan, Head of Uro-Oncology Service from the urology department at Sheba Medical Center; Dr. Tomer Kalisky and co-workers from Bar Ilan University; and Prof. Yaron Fuchs and co-workers from The Technion.

By Ken Stephens

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Will Dialysis Become a Thing of the Past? - Jewish Link of New Jersey

"Mini Brains" Are Not like the Real Thing – Scientific American

The idea of scientists trying to grow brain tissue in a dish conjures up all sorts of scary mental pictures (cue the horror-movie music). But the reality of the research is quite far from that sci-fi visionand always will be, say researchers in the field. In fact, a leader in this area of research, Arnold Kriegstein of the University of California, San Francisco, says the reality does not measure up to what some scientists make it out to be.

In a paper published on January 29 in Nature, Kriegstein and his colleagues identified which genes were active in 235,000 cells extracted from 37 different organoids and compared them with 189,000 cells from normally developing brains. The organoidsat times called mini brains, to the chagrin of some scientistsare not a fully accurate representation of normal developmental processes, according to the study.

Brain organoids are made from stem cells that are transformed from one cell type to the another until they end up as neurons or other mature cells. But according to the Nature paper, they do not always fully complete this developmental process. Instead the organoids tend to end up with cells that have not fully transformed into new cell typesand they do not re-create the normal brains organizational structure. Psychiatric and neurodevelopmental conditionsincluding schizophrenia and autism, respectivelyand neurodegenerative diseases such as Alzheimers are generally specific to particular cell types and circuits.

Many of the organoid cells showed signs of metabolic stress, the study demonstrated. When the team transplanted organoid cells into mice, their identity became crisper, and they acted more like normal cells, Kriegstein says. This result suggests that the culture conditions under which such cells are grown does not match those of a normally developing brain, he adds. Cellular stress is reversible, Kriegstein says. If we can reverse it, were likely to see the identity of cells improve significantly at the same time.

Brain organoids are getting better at recapitulating the activities of small clusters of neurons, says Kriegstein, who is a professor of neurology and director of the Eli & Edythe Broad Center for Regeneration Medicine and Stem Cell Research at U.C.S.F. Scientists often make organoids from the cells of people with different medical conditions to better understand those conditions. But some scientists may have gone too far in making claims about insights they have derived from patient-specific brain organoids. Id be cautious about that, Kriegstein says. Some of those changes might reflect the abnormal gene expression of the cells and not actually reflect a true disease feature. So thats a problem for scientists to address.

A small ball of cells grown in a dish may be able to re-create some aspects of parts of the brain, but it is not intended to represent the entire brain and its complexity, several researchers have asserted. These organoids are no more sentient than brain tissue removed from a patient during an operation, one scientist has said.

Of course, models are never perfect. Although animal models have led to fundamental insights into brain development, researchers have sought out organoids, or organs-in-a-dish, precisely because of the limitations of extrapolating biological insights from another species to humans. Alzheimers has been cured hundreds of times in mice but never in us, for instance.

That said, the current models are already very useful in addressing some fundamental questions in human brain development, says Hongjun Song, a professor of neuroscience at the Perelman School of Medicine at the University of Pennsylvania, who was not involved in the new research. Using brain organoids, he adds, the Zika virus was recently shown to attack neural stem cells, causing a response that could explain why some babies exposed to Zika in utero develop unusually small brains.

Michael Nestor, a stem cell expert, who did not participate in the new study, says his own organoids are very helpful for identifying unusual activity in brain cells grown from people with autism. And he notes that they will eventually be useful for screening potential drugs.

Even though the models will always be a simplification, the organoid work remains crucial, says PaolaArlotta, chair of the department of stem cell and regenerative biology at Harvard University, who was also not involved in the Nature study. Neuropsychiatric pathologies and neurodevelopmental conditions are generally the result of a large number of genetic changes, which are too complex to be modeled in rodents, she says.

Sergiu Pasca, another leader in the field, says that the cellular stress encountered by Kriegstein and his team might actually be useful in some conditions, helping to create in a dish the kinds of conditions that lead to diseases of neurodegeneration, for instance. What I considerthe most exciting feature remains our ability to derive neural cells and glial cells in vitro, understanding their intrinsic program of maturation in a dish, says Pasca, an assistant professor at Stanford University, who was not part of the new paper.

The ability to improve cell quality when exposed to the environment of the mouse brain suggests that it may be possible to overcome some of the current limitations, Arlotta says. There is not yet a single protocol for making brain organoids in a lab, which may be for the best at this early stage of the field. Eventually, she says, scientists will optimize and standardize the conditions in which these cells are grown.

Arlotta, who is also the Golub Family Professor of Stem Cell and Regenerative Biology at Harvard, published a study last year in Nature showing that she and her colleagues canover a six-month periodmake organoids capable of reliablyincluding a diversity of cell types that are appropriate for the human cerebral cortex. She says it is crucial for organoid work to be done within an ethical framework. Arlotta is part of a federally funded team of bioethicists and scientists working together to ensure that such studies proceed ethically. The scientists educate the bioethicists on the state of the research, she says, and the ethicists inform the scientists about the implications of their work.

Nestor feels so strongly about the importance of linking science, policy and public awareness around stem cell research that he has put his own laboratory at the Hussman Institute for Autism on hold to accept a year-long science-and-technology-policyfellowship with the American Association for the Advancement of Science. He says he took the post to make sure the public and policy makers understand what they need to know about organoids and other cutting-edge science and to learn how to communicate about science with them.

One thing all of the scientists interviewed for this article agree on is that these brain organoids are not actual mini brains, and no one is trying to build a brain in a dish. Even as researchers learn to make more cell types and grow them in more realistic conditions, they will never be able to replicate the brains structure and complexity, Kriegstein says. The exquisite organization of a normal brain is critical to its function, he adds. Brains are still the most complicated structure that nature has ever created.

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"Mini Brains" Are Not like the Real Thing - Scientific American

Consumer Claims Over Peter Thomas Roth’s Alleged "False Advertising" of Skincare Products to Go to Jury – The Fashion Law

A handful of the buzzy products of Peter Thomas Roth are at the center of a strongly-worded lawsuit, one that accuses the New York-based skincare company which boasts about its richly nourishing and technologically advanced offerings of peddling pseudoscience and falsifying the effectiveness of its hyaluronic acid-soaked skin creams and rose stem cell-formulated face masks in an attempt to stand out in the fiercely competitive $135 billion-plus skincare market and cater to the rising demand for anti-aging products among consumers.

According to the complaint that Peter Thomas Roth, LLC (Roth) customers Kari Miller and Samantha Paulson filed in a California state court in December 2018, Roth is running afoul of the law by making false claims about the capabilities of [its] products, at least some of which are among its best-selling products on Sephoras website. The plaintiffs assert that even in an industry known for hype, Roths outrageous marketing practices stand out among those of their competitors, as Roths claims about their [products] are not just hype; rather, they are demonstrably false.

Specifically, Miller and Paulson state that two of Roths product lines,the influencer-endorsed Rose Stem Cell line and the Water Drench line, are at the center of their suit, as both lines have allegedly been marketed and sold in conjunction with false and deceptive representations [about their] active ingredients rose stem cells and hyaluronic acid, respectively that have enabled Roth to profit enormously while its customers are left with overpriced, ineffective skin care products.

For instance the plaintiffs assert that in connection with its Water Drench line of products Roth represents that the active ingredient, hyaluronic acid, will draw moisture from the atmosphere into the users skin, and will hold 1,000 times its weight in water for up to 72 hours. This is impossible, they claim, as hyaluronic acid is incapable of absorbing anywhere near 1,000 times its weight in water, even when it is in its anhydrous (i.e., waterless; completely dry) form.

The judge notes that Roth softened the claim with the words up to in connection with the absorption power of thehyaluronic acid, but he also claims that subtle qualifications do not overcome the thrust of the ad, which is thatthe ad was one thousand times its weight in water.

As for Roths line of Rose Stem Cell products, which the brand claims are are capable of repairing, regenerating, and rejuvenating human skin andstimulating cellular turnoveras a result of the inclusion of rose stem cells, the plaintiffs argue that there is absolutely no evidence thatrosestem cells can provide such benefits. They allege thatRothis clearly attempting to capitalize on the recent media attention that has been given to medical research of human stem cells, with the goal of confusing consumers and causing them to erroneously believe that they will receive significant health benefits by using the Rose Stem Cell Products.

Such pseudo-science has enabled Roth to sell over-priced products to a growing market for skin care products, whileenjoying an unlawful advantage over [its] competitors, the plaintiffs assert in the suit, which has since been transferred from California state court to federal court.

In a couple of recent developments in the case, Judge William Alsup of the U.S. District Court for the Northern District of California denied the plaintiffs bid for class action approval, a move that would enable other individuals who have purchased the allegedly misrepresented products to join in their suit and any ultimate settlement sum. According to Judge Alsups January 22 decision, The plaintiffs can obtain their requested liability determination [for their false advertising claims] and statewide injunction against Roths challenged ads without certifying a class.

Meanwhile, in a separate January 22 order, the judge decided on Roths motion for summary judgment, refusing (for the most part) to issue a final decision resolving the plaintiffs claims ahead of trial because there are still issues of fact to be determined, namely whether Roths marketing claims are deceptive.

According to Judge Alsup, it is unclear how a reasonable consumer might view the marketing claims that Roth uses in connection with its Rose Stem Cell Mask namely, the labels, rose stem cells, cutting edge bio-technology, bio-repair, regenerates, and rejuvenates. While some reasonable consumers might interpret this [language] as mere puffery, and thus, not objective, actionable statements, others could sensibly conclude that rose stem cells actually repair human skin, which the plaintiffs argue is untrue, thereby, making the marketing claims deceptive.

In terms of Roths Water Drench line of products, the judge states that the plaintiffs contend the reasonable consumer would believe that hyaluronic acidactually canattract and retain one thousand times its weight in water, and in fact, a jury could find that, based on the ad, reasonable consumers would expect that hyaluronic acid absorbs and retains about one thousand times its weight in water.As such, these issues must go before a jury, which, Judge Alsup says will look forward to an in-court demonstration in which a certain amount of hyaluronic acid is placed in a beaker, one thousand times that weight in water is placed in another beaker, and the contents are combined, all watching to see if all the water will be absorbed.

*The case is Kari Miller, et al., v. Peter Thomas Roth, LLC, et al.,3:19-cv-00698 (N.D.Cal.)

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Consumer Claims Over Peter Thomas Roth's Alleged "False Advertising" of Skincare Products to Go to Jury - The Fashion Law

NIH Grants $4.8 Million to Gladstone Institutes to Study ApoE4 in Alzheimer’s – PharmaLive

The best-known genetic marker for Alzheimers disease is ApoE4, a form of apolipoprotein E, which is a protein involved in repairing neurons injured by aging, stroke or other reasons. ApoE4 is found in about a quarter of all people, but in about two-thirds of Alzheimers patients.

The National Institutes of Health (NIH)granted$4.8 million to Robert Mahley, President Emeritus of the Gladstone Instituteslocated in the UCSF Medical Center at Mission Bay, San Francisco. The focus of the research will be to learn more about ApoE4 and how and why it affects Alzheimers disease.

ApoE4 dramatically rewires cellular pathways in neurons and impairs their function, Mahley said. Our goal is to understand how this rewiring occurs and identify potential new treatment strategies to negate the detrimental effects.

Everybody has the ApoE gene, which has three different types, called isoforms: ApoE2, ApoE3 and ApoE4. Researchers at Gladstone have argued for some time that ApoE4 acts as more than a risk factor, directly contributes to brain cell damage. For example, another researcher at Gladstone, Yadong Huang, found that ApoE4 is easily broken into fragments inside neurons, and the fragments impair the function of the neurons.

There are a number of detrimental effects, Mahley said of that research in September 2019. And right now, we have no therapy that targets ApoE4.

The difference between ApoE3 and ApoE4 is a single point mutation, but that change means ApoE4 has a different shape from ApoE3. This makes it more susceptible to the fragmentation.

Our work suggests that these ApoE4 fragments are toxic to neurons and cause sweeping changes to the collection of proteins expressed within a neuron, Mahley said in the most recent announcement. We suspect that their toxicity may underlie much of the neurodegeneration seen in Alzheimers disease.

Mahley plans to use the funds to work with Senior Investigator Nevan Krogan and Gladstone Mass Spectrometry Facility Director Danielle Swaney. They will leverage affinity purification mass spectrometry (AP-MS) to first identify which proteins in a single cell directly interact with ApoE4 fragments.

Swaney said, AP-MS is an important first step because it will allow us to define physical interactions between proteins that may underlie the functional deficits observed in neurons that express ApoE4.

They will be the AP-MS work on neuronal cells derived from mice, which are similar to human neurons. They will also utilize advanced protein analysis to learn more about the abnormal regulation in neurons with ApoE4 being expressed. This will be conducted in neurons from human induced pluripotent stem (hiPS) cells. The procedure for that was developed by Shinya Yamanaka, a 2012 Nobel prize winner and Gladstone senior investigator.

We are quite excited to be involved in this project, Krogan said. My lab has successfully applied AP-MS and other cutting-edge proteomic and genetic techniques to many different diseases, and we now hope to enable a much deeper understanding of ApoE4.

The hope is that by combining that work, they will compile a list of proteins involved in pathways specifically changed in ApoE4 neurons compared to ApoE3 neurons. They will then pick lead candidates for more research in neurons grown from hiPS cells, which will be guided by Yadong Huang.

The plan after that is to use CRISPR gene editing to see if they can reverse the effects of ApoE4 by activating or inhibiting the genes that control their chosen protein candidates.

By the end of the project, we hope to narrow down our list to just a few target genes or proteins that protect or restore neuronal health when we activate or inhibit them in live mice with the ApoE4 gene, Swaney said. They could then be explored as potential targets for Alzheimers treatment in humans.

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NIH Grants $4.8 Million to Gladstone Institutes to Study ApoE4 in Alzheimer's - PharmaLive

Judith C. Brings WELCOME TO THE CANCER CAFE to The Marsh Berkeley – Broadway World

A PT Clinical Specialist in Chronic Pain, Judith C. never imagined being on the opposite end of treatment -until she was diagnosed with Multiple Myeloma, an incurable blood cancer with a limited life expectancy. This March, Judith's journey from diagnosis to stem cell transplant unfolds in her hilarious and heartbreaking solo show Welcome to The Cancer Caf at The Marsh Berkeley. Enlightening, elevating, and deeply personal, Judith brings audiences on her unexpected journey from provider to patient, sharing the profound lessons this role reversal offers. Using actual conversations with providers, Judith portrays her interactions with the medical establishment, bringing keen insight and offering a better understanding of the journey and choices faced by people of similar circumstances. Proceeds from each show will be donated to a local cancer organization chosen by Judith.

Talkback engagements after every performance will also be offered. Welcome to The Cancer Caf will be presented March 8 -22, 2020 with performances at 2:00pm Sundays at The Marsh Berkeley, 2120 Allston Way, Berkeley. For tickets ($20-$35 sliding scale; $55 and $100 reserved) or more information, the public may visit http://www.themarsh.org or call The Marsh Box office at 415-282-3055 (open Monday through Friday, 1:00pm-4:00pm).

Motivated by portrayals of cancer heroes, cancer miracles, and people that "fight" for their lives, Judith C. was determined to shed light on different responses to cancer than the typical warrior model. Welcome to The Cancer Caf has been performed at the Stanford Oncology Grand Rounds (2017), Bone Marrow Transplant Life Survivorship Symposium in Denver, CO (2018) and Orlando, FL (2019), Kehilla Community Synagogue (2018), and for various cancer support groups throughout Northern CA. Originally presented at The Marsh Berkley as part of its Marsh Rising series in November 2019, Welcome to The Cancer Caf returns in March 2020 for a full run.

Proceeds from each performance will be donated to a local cancer organization chosen by Judith. Talkback engagements following all performances of Welcome to The Cancer Caf will also be offered:

Sunday, March 8: Proceeds will go towards Camp Kesem (Berkeley), one of many chapters of a wider organization that supports children through and beyond a parent's cancer. Founded at Stanford University in 2000, Camp Kesem has since expanded to 116 chapters in 42 states around the country, offering free summer camps for children who have been impacted by this life-changing event. Camp Kesem Outreach Coordinator Autumn Frlekin and Development Coordinator Shaked Salem will discuss how the camp brings together a community of children with similar experiences in a safe, welcoming environment to have fun and rediscover their childhood.

Sunday, March 15: Charlotte Maxwell Complementary Clinic for Low-Income Women with Cancer in Oakland will be receiving proceeds from the March 15th performance. For 25 years, this free public health clinic and 501(c)(3) non-profit organization has provided integrative cancer care and supportive services that compliment mainstream cancer treatments. These services include acupuncture, herbs, homeopathy, bodywork, guided imagery, movement, and nutritional therapies. Dr. Loveleena Virk and Dr. Divya Chandrasekar, Palliative Care MDs at Kaiser Oakland, will discuss approaches that improve the quality of life of patients and their families facing the problem associated with life-threatening illness.

Sunday, March 22: Proceeds from the final performance will go towards the Women's Cancer Resource Center (WCRC) in Berkeley. This non-profit organization creates opportunities for women with cancer to improve their quality of life through education, supportive services, and practical assistance. Dolores Moorehead, WCRC Lead Client Navigator and Multicultural Client Support Clinician, will be accompanied by a client to share how this organization helps people with cancer navigate through the overwhelming health care system, allowing them to get the appropriate care and treatment they need.

Described by the San Francisco press as "the monologue maestro" and "the dean of solo performance," David Ford has been collaborating on new and unusual theater for decades and has been associated with The Marsh for most of that time. After seeing Welcome to the Cancer Caf, Ford said, "I've worked for 30 years with people who are writing and performing their own stories. In that time, I've heard a lot of people give witness to the trials of having a life-threatening disease. Judith C. goes further than anyone I've ever encountered. She gets the awful contrasts of fear and courage, agency and impotence, armor and vulnerability."

Judith C. (Actor/Writer) is a PT Clinical Specialist in Chronic Pain/Feldenkrais practitioner, with decades of clinical and teaching experience. At age 58, she was diagnosed with Multiple Myeloma, an incurable blood cancer with no clear, accepted treatment path. Following her diagnosis, Judith began writing and performing about her experience as a way to cope with her life as a Myeloma patient being the "new normal" - eventually leading to the birth of her first solo work, Welcome to The Cancer Caf.

Rebecca Fisher (Director) is the creator of The Marsh's Writing Cabaret and the co-producer of the long-running Marsh series Tell it on Tuesday. Her solo performance work has been called "smart, challenging, and unmistakably affecting" by the San Francisco Chronicle and has won a San Francisco Best of Fringe award. She has directed solo shows in national fringe festivals, as well as Mark Kenward's Nantucket and Diane Barnes' My Stroke of Luck, both presented at The Marsh. Fisher was a teaching artist with The Lincoln Center affiliated arts education program in Bay Area schools and has directed theater camps with the Berkeley Playhouse/Julia Morgan Center for the Arts and The Marsh.

The Marsh is known as "a breeding ground for new performance." It was launched in 1989 by Founder and Artistic Director Stephanie Weisman, and now annually hosts more than 600 performances of 175 shows across the company's two venues in San Francisco and Berkeley. A leading outlet for solo performers, The Marsh's specialty has been hailed by the San Francisco Chronicle as "solo performances that celebrate the power of storytelling at its simplest and purest." The East Bay Times named The Marsh one of Bay Area's best intimate theaters, calling it "one of the most thriving solo theaters in the nation. The live theatrical energy is simply irresistible."

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Judith C. Brings WELCOME TO THE CANCER CAFE to The Marsh Berkeley - Broadway World

The Center for Breakthrough Medicines is Building the World’s Largest Cell and Gene Therapy Contract Development and Manufacturing Organization (CDMO)…

KING OF PRUSSIA, Pa., Jan. 22, 2020 /PRNewswire/--The Discovery Labs and Deerfield Management Company have formed The Center for Breakthrough Medicines, a Contract Development and Manufacturing Organization (CDMO) and specialty investment company, to alleviate the critical lack of capacity that is preventing patients from accessing critically needed cell and gene therapies. The CDMO is occupying over 40 percent of The Discovery Labs' 1.6 million square foot biotech, healthcare and life sciences campus in King of Prussia, PA.

The CDMO provides preclinical through commercial manufacturing of cell and gene therapies and component raw materials. It offers process development, plasmid DNA,viral vectors, cell banking, cell processing, and support testing capabilities all under one roof. The immense $1.1 billion facility will provide instant capacity as the largest known single source for accelerating the delivery and affordability of lifesaving and life-changing therapies from the bench to the patient's bedside.

The Company has initiated a substantial hiring effort targeting the best and brightest of the life sciences community including, experts in CGMP manufacturing. The Company expects to hire over 2,000 team members within the next 30 months.

The CDMO has retained Nucleus Careers, a cloud-based specialty life sciences human capital recruiting and retention management expert, to buildout the entire team. Nucleus has proprietary recruiting and retention software designed for large scale human capital buildouts of high growth companies.

In addition to developing the world's largest single-point cell and gene therapy manufacturing facility, The Discovery Labs is establishing THE COLONY which will provide custom built discovery labs, breakthrough funding, sponsored research agreements, housing and relocation for the world's leading iconic experts in cell and gene therapy.

THE COLONY will seek to work hand in hand with scientists from both academic and pharmaceutical institutions to unlock and expedite groundbreaking therapies.

Marco A. Chacn, Ph.D., Founder of Paragon Bioservices and Chairman of The Discovery Labs states, "musicians, artists, members of religious communities and great thinkers throughout time have formed colonies where freedom of thought and expression combined with unlimited dreams and potential have resulted in the world's greatest accomplishments." Dr. Chacn went on to say, "the goal of THE COLONY is to unshackle the potential of the world's greatest scientific minds."

The ability for the industry's greatest scientists to cohabitate, collaborate, cooperate, and communicate via technology and in person will create an exponential therapeutic "X FACTOR." THE COLONY seeks to unlock institutional barriers prohibiting the world's greatest scientists from moving at a pace necessary in today's ever-changing therapeutic revolution. THE COLONY will partner with the institutions where the scientists currently work by providing equity, license fees, and revenue sharing.

"The Center for Breakthrough Medicines will be serving companies from the earliest stages through commercialization. Its exceptional scale and offering will quickly relieve the production bottleneck for advanced therapies by reducing the time, complexity, and cost of commercializing vitally needed gene and cell therapies," noted Audrey Greenberg, Board Member and Executive Managing Director for The Discovery Labs.

The addition of this end-to-end manufacturing capability is expected to significantly enhance the offerings of The Discovery Labs in an area that has become one of the largest life sciences hubs in the world. Renovations are underway to construct a total of 86 plasmid, viral vector production, universal cell processing, CGMP testing, process development and cell banking suites. The viral vector and cell processing suites will be fully compliant with both U.S. Food and Drug Administration and European Medicines Agency standards. All suites will offer the flexibility to meet client-specific workflows and will be able to adapt quickly to meet demand. The Company is in the process of reserving capacity now for late 2020.

"Today brilliant scientists are advancing an unprecedented number of gene and cell therapy drug candidates. The real tragedy, however, is a scarcity of manufacturing know-how, which is complex and expensive," said Alex Karnal, Partner and Managing Director of Deerfield Management and a Board Member of the Discovery Labs. "With its visionary business model, it is hoped that The Center for Breakthrough Medicines will help realize the promise of cell and gene therapies in time to treat the many patients who need them."

The Discovery Labs provides a central campus where the world's greatest scientists can collaborate on new therapeutic discoveries to eradicate diseases affecting small and large segments of the global population. The Center for Breakthrough Medicines will work with these leaders, life sciences companies, large pharmaceutical companies, and academic and government institutions.

This new manufacturing capability is a transformational addition to The Discovery Labs market offering and dovetails with The Discovery Labs biotech incubator, Unite IQ. Unite IQ offers immediate space to emerging life sciences companies and scientists giving them the ability to grow from startup to enterprise company on one campus. The incubator and accelerator space at Unite IQ provides a comprehensive home for startups with every resource needed to initiate business operations. Unite IQ tenants are expected to utilize the discovery, development, testing, and manufacturing capabilities of the Center for Breakthrough Medicines with seamless forward integration of processes and analytics, and seamless tech transfer from research lab to large scale production

The Emerging Field of Cell and Gene Therapy in Pennsylvania

The demand for clinical and commercial manufacturing capacity is acute and expected to remain that way. The current shortfall in manufacturing for cell and gene therapies is severely underserved with few approved products. There are currently approximately 1,100 advanced therapies in the pipeline pending FDA approval. This will greatly increase highly skilled manufacturing demand. Dr. Peter Marks, Director of the FDA Center for Biologics Evaluation and Research, states, "what keeps me up at night is will we be able to manufacture these on a scale that will allow us to bring the benefit of these therapies to patients?"He further added that "if we can help see cost of goods and ability to manufacture reproducibly improve, I think that'll be a big thing."All of this adds up to a supply constrained market that The Center for Breakthrough Medicines aims to help address.

With the potential to treat and even cure disabling, and deadly diseases, gene and cell therapies are ushering in a new era of medicine. These therapies may eventually be able to cure genetic conditions, such as cystic fibrosis, hemophilia A, and a range of cancers. The Philadelphia area has become the epicenter for the flourishing field of gene and cell therapy. Research from CBRE currently ranks the market among the top biotech clusters for medical research and health services. The cluster has become known worldwide as "Cellicon Valley"for its leadership in research and development of this rapidly evolving field. The Discovery Lab's suburban Philadelphia location offers a talent rich environment due to the area's preponderance of large pharmaceutical companies and the Philadelphia region's position boasting the top 10 universities and primary school systems in nation.

Over the past three years, multiple Philadelphia companies have received approvals for major breakthroughs in cell and gene therapy. In 2017, the U.S. FDA approved the first-ever CAR-T cell therapy, Novartis's Kymriah, which originated at the University of Pennsylvania. Shortly thereafter, the FDA gave landmark approval for the first-ever gene therapy to treat a genetic blindness condition to Spark Therapeutics, a start-up founded by researchers at Children's Hospital of Philadelphia. These discoveries and others in the pipeline are attracting billions of dollars of venture capital. The Greater Philadelphia Region set a recent record in venture capital financing.

The Discovery Labs Center for Breakthrough Medicines joins more than 25 healthcare, life sciences and tech-enabled companies that already call The Discovery Labs King of Prussia home.

Brian O'Neill, Founder of The Discovery Labs Center for Breakthrough Medicines, and Tony Khoury, Board Member of The Discovery Labs and Engineer at Project Pharma, will be speaking at the 2020 PhacilitateWorld Stem Cell Summit discussing The Future of Gene Therapy Manufacturing at 4 p.m. today at the Hyatt Regency in Miami, Florida.

Contact Audrey Greenberg at agreenberg@thediscoverylabs.com for more information about development services, manufacturing capacity, incubator space or leasing information at the property.

About The Discovery LabsPart of MLP Ventures, The Discovery Labs is a global provider of world-class cGMP manufacturing, turnkey laboratory solutions, critical materials and office space that support therapeutic products and services to the biotechnology and pharmaceutical industry so that groundbreaking medicines get to the patients that need them. The location in eastern King of Prussia is a prototype for a global rollout of The Discovery Labs, providing Big Pharma, emerging life sciences, consumer and technology companies flexible, end-to-end technical real estate and business infrastructure for the customer's entire lifecycle from discovery to delivery, including manufacturing capacity. It is the first fully integrated environment that merges technology and life sciences under one roof to drive innovation.

About Deerfield Management

Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

Media Contact:Tony DeFazio, DeFazio Communications(o) 484-534-3306 (c) 484-410-1354tony@defaziocommunications.com

SOURCE The Discovery Labs

The Discovery Labs

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The Center for Breakthrough Medicines is Building the World's Largest Cell and Gene Therapy Contract Development and Manufacturing Organization (CDMO)...

Toxys Strengthens Leadership Team with Appointment of CBO and COO Positions – BioSpace

LEIDEN, The Netherlands, January 21, 2020 /B3C newswire/ --Today,Toxysannounces the appointment of a Chief Business Officer (CBO) and a Chief Operating Officer (COO). Toxys BV is a biotech company based in Leiden, The Netherlands, that provides innovative, high-fidelityin vitrotoxicity screening solutions to identify carcinogenic and other hazardous properties of compounds for the pharmaceutical, chemical, cosmetics and food industries. With this strengthened leadership, Toxys will further accelerate business growth to become an industry-leader in animal-free chemical safety testing.

Paula van Rossumis promoted toCBO. Paula joined Toxys in 2016 as Director Business Development & Sales and has been instrumental for the business growth of Toxys. Paula holds a MSc degree from Leiden University in Biomedical Sciences and Science-based Business.

Remco Derris promoted toCOO. Remco joined Toxys in 2014 as lab manager and product specialist. He has been instrumental for management of all commercial toxicity screening as well operational management of the R&D laboratory. Remco holds a MSc degree from Leiden University in Biomedical Sciences.

Paula and Remco are valuable additions to the senior Toxys Management Team. Their proven experience and expertise in business development, sales, customer relations and lab management will further boost the growth of Toxyssaid Giel Hendriks, CEO of Toxys BV.

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AboutToxysToxys is a Dutch biotech companythat offers a broad spectrum of innovativein vitrotoxicology solutions. Toxys was founded in 2014 as a spin-off from the Leiden University Medical Center and has its state-of-the-art laboratory facilities located at the Leiden BioScience Park. We are experts in the field of genetic and developmental toxicology with a focus on Mode-of-Action. We are dedicated to bringing relevant information to our clients on potential human health hazards of novel and existing drugs, chemicals and other substances. Toxys is currently working with 7 of the Top10 global Pharma companies and several major chemical, cosmetics and food multinationals.

Toxys has developed the unique ToxTracker and ReproTracker assays. ToxTracker is a high-throughput stem cell-based reporter suite of assays that allows reliable identification of genotoxic carcinogens. ToxTracker provides mechanistic insight into undesired properties of chemicals. ReproTracker is currently under development and its commercial launch (planned for early 2020) will allowin vitrodevelopmental toxicity hazard identification. These assays can be particularly useful in Mode-of-Action and Adverse Outcome Pathway (AOP) approaches for chemical safety assessment.

We offer ToxTracker and ReproTracker as tailored service to our customers, often in combination with standard toxicology assays or under license and as kits for use in partners own laboratories. Toxys is highly valued for its scientific expertise, high quality results and responsiveness.

Contact

Giel HendriksCEO+31 71 3322474g.hendriks@toxys.com

Media Contact

Paula van RossumCBO+31 71 3322470p.vanrossum@toxys.com

Keywords: Humans; Carcinogens; Chemical Safety; Cosmetics; Carcinogenesis; Industry; Food Industry; DNA Damage; Drug-Related Side Effects and Adverse Reactions; Mutagenicity Tests; Biotechnology

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Toxys Strengthens Leadership Team with Appointment of CBO and COO Positions - BioSpace

Share the Love on Valentine’s Day! Join the Cure 4 The Kids Foundation Blood Drive and Registry Event – Nevada Business Magazine

LAS VEGAS Regardless of your relationship status on Valentines Day, you can have an incredible impact on someones life in our communityand possibly save a life by donating blood or by becoming part of the bone marrow registry.

Cure 4 The Kids Foundation is well aware of the national shortage of blood and platelets, and how vital a reliable blood supply is to the on-going health of our community. Thats why Cure 4 The Kids Foundation is organizing the Valentines Day Blood Drive and Bone Marrow Registry Event on Friday, February 14, 2020.

The public is strongly encouraged to take part in either effort between 9:00 a.m. and 4:00 p.m. at One Breakthrough Way, Las Vegas, Nevada 89135.

Blood donors can ensure an easy and relatively quick donation process by making an appointment prior to Valentines Day by going towww.bloodhero.comand entering the code: cure4kids under sponsor. You can also schedule an appointment by calling Vitalant at 877-25-VITAL. The Vitalant bloodmobile will be parked in front of the One Breakthrough Way building.

Blood Donation Reminders:

Blood donors must be at least 16 years old (16 and 17-year olds must bring a signed permission form from a parent or guardian)Weigh at least 110 poundsBe in good general healthDrink plenty of water 24 to 48 hours prior to donationBring ID

Cure 4 The Kids Foundation is also partnering with the Be The Match Registry. The registry offers hope to those affected by cancer and other conditions by matching potential blood stem cell donors with those who are in need of a transplant.

The first step in joining the registry is to undergo a mouth swab by one of our volunteers. Those who are a possible match will be contacted at a later time. This service is free and available between 9:00 a.m. and 4:00 p.m. on February 14, 2020, in the lobby of the One Breakthrough Way building.

Bone Marrow Registry Reminders:

Donors between 18 and 44 years of age are especially neededYou cannot become a donor if you had have certain diseasesLearn more atjoin.bethematch.org

About Cure 4 The Kids Foundation

Founded in Las Vegas in 2007, Cure 4 The Kids Foundation exists to provide high-quality, research-focused medical treatment to children battling cancer and many other life-threatening conditions. Cure 4 The Kids Foundation operates the only outpatient childhood cancer treatment center in the State of Nevada and is proudly accredited by The Joint Commission. This stringent medical accreditation and the required unannounced inspections ensure that patients are getting the best care possible. From the beginning, the mission of Cure 4 The Kids Foundation has been to bring increased community access to these specialized treatments that are leading the way to improved patient outcomes. Cure 4 The Kids Foundations Charity Care Program provides high-quality treatment to those who are uninsured, underinsured and those unable to pay for treatment. No patient is ever turned away for financial reasons. We are a 501(c)(3) nonprofit organization.www.cure4thekids.org

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Share the Love on Valentine's Day! Join the Cure 4 The Kids Foundation Blood Drive and Registry Event - Nevada Business Magazine

Botox, medical drips and more offered at NVY Center – SILive.com

STATN ISLAND, N.Y. -- As a Manhattan-based attorney, Chris Cardillo has overseen his share of medical management over the years.

Paperwork involved with being a doctor is so overwhelming these days, he said, noting that many physicians turn to attorneys to help manage their practices.

So with a wide knowledge of the medical industry, he sought to open a business that would house different physicians, as well as spa services, in a medical spa setting under one roof.

The result is NVY Center, a medical management company that opened in Richmond Valley more than one year ago.

As a Manhattan-based attorney, Chris Cardillo has completed his share of medical management over the years. Paperwork involved with being a doctor is so overwhelming these days, he said, noting that many physicians turn to attorneys to help manage their practices. (Staten Island Advance/Tracey Porpora)

I became very interested in the concept because I think its the wave of the future, said Cardillo, who has several partners in the business. Almost every medical office has a medical manager. ...When you go to a hospital, like New York University Medical Center, the hospital does all the management for doctors with affiliated practices. What hospitals do for their doctors, we do on a private level.

MEDICAL, SPA SERVICES UNDER ONE ROOF

NVY Center rents a clean, white expansive space to various medical professionals, and aestheticians, who provide spa services.

We are hoping to be a national brand. What we do is solicit doctors to lease space for us and we manage them. That means when a doctor comes in here the only thing the doctor has to worry about is taking care of the patient, said Cardillo. We take care of the billing. They dont have to do any of the paperwork. Everything they need to do their job is here for them already.

Under the NVY Center roof are an array of spa services -- from stem cell therapy and IV drips to facials and aesthetic injections. (Staten Island Advance/Tracey Porpora)

MEDICAL SPA SERVICES

Under the NVY Center roof are also an array of medical spa services, from stem cell therapy and IV drips, to facials and aesthetic injections.

Included in the facilitys design are individual medical offices, rooms where different procedures, such as facials, can take place, and an IV drip chair area. And the facility always has a medical professional on the premises, said Cardillo.

We do everything from anti-hangover to anti-flu drip. ... Soon, well be able to come to your house and do an IV drip, explained Cardillo. We want this to be a one-stop-shop for modern services."

He noted the IV drips often include vitamins and are used for various purposes, including by those people who desire to lose weight.

We have fast-drips that are 15 minutes long, where we can fill you with fluids if youre not feeling good, said Cardillo. Depending on what were trying to treat, we will mix vitamins and other medicines into the IV drip. But the fluid helps no matter what.

He said the company also hosts IV drip and Botox parties.

Other services include Trusculpting," a fat reduction procedure to lose weight, and laser hair removal, said Cardillo.

NVY CENTER AT A GLANCE

Address: 236 Richmond Valley Rd.

Website: http://www.nvycenter.com

Facebook: http://www.facebook.com/nvycenter/

New Businesses in Focus is a weekly column that relates the stories of new Staten Island businesses owners.

If you have a new business on Staten Island, e-mail porpora@siadvance.com.

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Botox, medical drips and more offered at NVY Center - SILive.com

Walter named Edward P. Evans Endowed Professor – Washington University School of Medicine in St. Louis

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Oncologist honored for research, patient care in myelodysplastic syndromes

Medical oncologist Matthew Walter, MD, presents his research after being installed as the inaugural Edward P. Evans Endowed Professor of Myelodysplastic Syndromes at Washington University School of Medicine in St. Louis.

Matthew J. Walter, MD, has been named the inaugural Edward P. Evans Endowed Professor of Myelodysplastic Syndromes at Washington University School of Medicine in St. Louis. These syndromes make up a group of rare blood disorders that prevents the body from making sufficient healthy blood cells.

Walter, an international leader in the study and treatment of myelodysplastic syndromes (MDS), is a professor of medicine and director of the Edward P. Evans Center for Myelodysplastic Syndromes at the School of Medicine. The endowed professorship and the center are supported by funding from the Edward P. Evans Foundation, established in 1984 by Edward Parker Evans, a businessman and philanthropist whose foundation supports MDS research nationwide. He died in 2010 from a blood cancer related to MDS.

Walter treats patients with MDS and related blood disorders at Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine. His new professorship was announced by Chancellor Andrew D. Martin and David H. Perlmutter, MD, executive vice chancellor for medical affairs, the George and Carol Bauer Dean of the School of Medicine, and the Spencer T. and Ann W. Olin Distinguished Professor.

Matthew Walter has devoted his career to the study and treatment of myelodysplastic syndromes, and it is a great pleasure to be able to honor his work and commitment to his patients in this way, Martin said. There is no one better suited to lead this center. This endowed professorship will help support his work to understand the genetic and environmental underpinnings of this disease so that researchers can understand how it develops and work toward developing improved precision therapies to prevent or treat MDS.

Some 40,000 Americans are diagnosed with MDS each year. The symptoms can be vague, including shortness of breath and fatigue, which makes it difficult to diagnose. Low blood cell counts are among the first signs of the disease, and patients are diagnosed based on the appearance of abnormal blood cells. MDS can be fatal, but some patients with low-risk MDS live for many years with the disease and never know they have it. About one-third of patients with MDS develop an aggressive form of the disease that progresses to acute myeloid leukemia (AML), which is a fast-growing blood cancer. Without a successful stem cell transplant, AML is fatal.

Walter is interested in studying patients at risk of developing MDS but who dont yet have the disease. Such patients have mutations in a few important genes linked to MDS but dont yet have symptoms. This condition is called age-related clonal hematopoiesis. Even though relatively few of these patients go on to develop MDS, studying this group of patients could help doctors understand the differences between those who develop MDS and those who dont.

The work that Matthew Walter is leading at the new Edward P. Evans MDS Center, including his work with patients, is the epitome of precision medicine, Perlmutter said. This research will help illuminate what leads patients to progress to MDS or more aggressive blood disorders. His vision includes developing ways to prevent this progression, as well as novel therapies, including immunotherapy strategies. It is a great pleasure to recognize the innovative work Matthew Walter is doing to better care for these patients.

Said Victoria J. Fraser, MD, the Adolphus Busch Professor of Medicine and head of the Department of Medicine: Matthew Walter is a leader in the field in harnessing his labs research discoveries for the benefit of his patients. This professorship will help him continue his important research into the origins of this disease and potential ways to prevent and treat it.

Walter earned his bachelors degree in 1990 from American University in Washington and his medical degree in 1995 from Saint Louis University School of Medicine. He was a research scholar at the Howard Hughes Medical Institute and later completed his internship and residency at Johns Hopkins Hospital. He came to Washington University as a hematology-oncology fellow and joined the faculty in 2004.

Matthew Walter, MD, (center) shakes hands with Washington University Chancellor Andrew D. Martin as Victoria J. Fraser, MD, head of the Department of Medicine, applauds Walters installation as the inaugural Edward P. Evans Endowed Professor of Myelodysplastic Syndromes.

He has been honored with numerous awards, including the Leukemia and Lymphoma Society Scholar Award, the MDS Foundation Young Investigator Award, and the American Society of Hematology Scholar Award. He is an elected member of the American Society for Clinical Investigation, and he serves on the National MDS Steering Committee of the National Institutes of Health (NIH), and on the American Society of Hematology Task Force on Precision Medicine. He is the 2019 chair of the American Society of Hematology Scientific Committee on Myeloid Neoplasia.

Edward Parker Evans ran Spring Hill Farm, the largest thoroughbred breeding farm in Virginia. Since his death, the Edward P. Evans Foundation has supported cutting-edge translational research on MDS at leading institutions across the country, including Washington University, which is noted for its innovative research and leadership in developing new therapies for patients with cancerous disorders of the blood.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Walter named Edward P. Evans Endowed Professor - Washington University School of Medicine in St. Louis