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Seeing through a forest of SCN2A gene variation – SFARI News

By daniellenierenberg

On August 23, 2019, the FamilieSCN2A Foundation held their biennial SCN2A Professional and Family meeting, in Seattle, Washington. The gathering brought together 37 families of individuals with mutations in the SCN2A gene, 60 investigators, eight clinicians and five industry groups that conduct research and/or clinical work on conditions related to this genetic change. A number of SFARI scientists and staff also attended the event.

The SCN2A family meeting was one of many events that family organizations of rare, neurodevelopmental disorders organized last summer. These meetings help families connect with others similarly affected as well as professionals working to better understand these conditions and develop new therapeutics. SFARI often attends and facilitates research opportunities carried on at these events.

SCN2A is a high-confidence autism risk gene, which encodes a subunit of a sodium channel in the brain called Nav1.2. When the channel malfunctions, conditions like epilepsy and autism follow. As part of its mission to understand the genetics and neurobiological underpinnings of autism, SFARI has awarded about $3 million for research on SCN2A, and some of this research was presented at the meeting. SFARI also supports a genetics first initiative called Simons Searchlight (formerly known as Simons VIP), which enrolls people with a genetic diagnosis showing rare genetic changes associated with autism and related neurodevelopmental conditions, such as SCN2A.

Many stories that may reflect the different ways SCN2A can be disabled were told at the meeting. One child had his first seizure when he was days old, and now spends many of his days irritable and immobilized by dystonia. Another developed normally until his first seizure as a toddler, which seemed to wipe out all of his skills; his milestones are now hard won in the face of continuing seizures and an autism diagnosis. Another had a sudden regression at 1 year of age, and after a misdiagnosis and seizure medication, she goes to a school for children with autism. Still another suffered from relentless seizures, which robbed her of speech; she died last year at the age of 12.

So far, about 300 different variants of the SCN2A gene have beendocumented, and the functional consequences of many are unclear. Some researchers have developed high-throughput experiments to systematically test each of thesevariants, and to screen compounds that could normalize their function2. Another approach may use genetherapy to boostexpression of the remaining good copy of SCN2A. Either way, finding appropriate in vitro testing grounds for these SCN2A variants is essential and may help personalize treatment approaches or identify more homogeneous patient groups for drug trials.

The meeting also underscored the power of family gatherings to push the science ahead. Investigators could see multiple examples of a rare genetic condition and engage new participants in research studies such as The Investigation of Genetic Exome Research (TIGER), a project of the University of Washington that compares phenotypes of single-gene conditions. In turn, families had the opportunity to express their concerns to scientists and infuse the research proceedings with urgency.

My biggest takeaway from this years conference was the mutual inspiration between the scientists and the families, says Leah Schust, meeting organizer and executive director of the FamilieSCN2A Foundation. Her son has a mutation in SCN2A.

Meeting the researchers working on a cure for our kids motivates us to fight on, Schust says. Then the scientists all say that meeting the families inspires them to go back to their labs and work even harder.

Family focus. The family meeting helped researchers reconsider what would be meaningful clinical endpoints for potential treatments. Schust says that most researchers and industry groups had thought seizure control was the most important issue. After listening to us, they realized that quality of life, movement disorders and autonomic dysfunction are higher on our list of where we would like to see improvement, she says.

When SCN2A mutations were first linked to autism, the gene stood out because it encodes a relatively well-understood protein, unlike many of the other identified genes. Nav1.2 is a voltage-gated channel found exclusively on excitatory neurons in the brain, where it controls the flow of sodium ions into the neuron, and thus its propensity for firing action potential. Experiments have revealed detailed pictures of Nav1.2s structure3, and known drugs alter its function4.

SCN2A also stands out because of its high recurrence rate in autism: unlike other autism genes, SCN2A is mutated with somewhat regular frequency5 (Figure 1).

Just as understanding why a car wont start is critical to fixing it, researchers need to understand how these SCN2A mutations alter the Nav1.2 channel. A current model1 posits that some mutations are gain-of-function, rendering the channel too active and the brain hyperexcitable, leading to infantile epilepsy; conversely, loss-of-function mutations reduce excitability and seem associated with autism and/or intellectual disability, as well as childhood-onset (as opposed to neonatal) seizures.

Yet the functional consequences of most SCN2A mutations remain unknown, and some may not fall neatly into a loss-of-function or gain-of-function category. A way of making sense of these mutations may come from looking at the working parts of Nav1.2, said Arthur Campbell of the Broad Institute of MIT and Harvard. For example, missense SCN2A variants linked to epilepsy seem to hit the channel randomly. But when marking their location on a crystal structure model of the channel, the missense variants cluster in several places: on the voltage sensor, on the linker helix responsible for conveying voltage sensor movement to the channel pore, on an area thought to interact with the beta-subunits involved in chaperoning the channel to the right place, and on the inactivation gate, which closes the pore off from sodium ion flow. He suggested that this knowledge, combined with the structural similarities between all sodium channels, may help drug development for SCN2A-related conditions.

High-throughput systems that can assay hundreds of cells at a time are helping researchers systematically explore SCN2A mutation, explained SFARI Investigator Al George of Northwestern University. While conventional electrophysiology would require weeks of work to characterize a single SCN2A variant, Georges group uses an automated patch-clamp system that can characterize multiple variants transfected into non-neuronal cell lines in a day. Using this system, two variants associated with neonatal seizures both exhibited an exceptional willingness to activate and a slowness to inactivate, which are properties consistent with a gain-of-function interpretation.

The high-throughput set up also promises to expedite the hunt for drugs to normalize SCN2A function: George described a 384-well plate design that allows measurement of the effects of two different drugs, at four different concentrations, on the SCN2A variant and control channels simultaneously. A known drug (carbamazepine) and an experimental drug (PRX-330) shifted channel inactivation to more hyperpolarized voltages, which could help quiet channels with gain-of-function mutations.

To narrow in on potentially therapeutic compounds, Jeff Cottrell and colleagues at the Broad Institute of MIT and Harvard have come up with a two-stage screen to find small molecule activators or inhibitors of Nav1.2 channels. First, compounds are initially tested on non-neural cells transfected with Nav1.2 sodium channels and potassium channels, which enables them to spike. The cells in 384-well plates are stimulated in parallel, and voltage-sensitive dyes give a readout of spiking activity; remarkably, Cottrells system allows data collection from up to 96 wells simultaneously. Any compounds that modulate spiking would then be subjected to the second stage, in a high-throughput electrophysiology assay similar to that described by George. Compounds with helpful mechanisms would then be tested for selectivity for Nav1.2 versus other sodium channels. A selective compound would then be tested in neurons, first in vitro then in vivo. This step-wise process has identified an activating compound that makes Nav1.2 more likely to open at rest and has potent effects on action potentials in brain slices and on electroencephalogram (EEG) traces from mice engineered to carry a disabled copy of SCN2A; however, Cottrell said this particular compound is not a therapeutic candidate in part because it broadens the action potential in a way that could promote seizures. A full screen is underway, and so far has identified 378 modulators from a library of 77,000 compounds.

Beyond academia, J.P. Johnson Jr. of Xenon in Burnaby, British Columbia, discussed the companys work to create sodium channel inhibitors for treating epilepsy. To obtain selective compounds, the group targets the voltage-sensing domain because its structure is the most diverse region of sodium channels. Xenon uses a trial-and-error method to optimize sodium channel inhibitor potency and selectivity. The methodical process has yielded some interesting compounds, including both selective Nav1.6 inhibitors and dual Nav1.6 and Nav1.2 inhibitors. Both quashed spiking in mouse excitatory pyramidal neurons, which contain only Nav1.2 and Nav1.6, but they did not alter spiking in Nav1.1-containing inhibitory neurons. A Nav1.6 selective inhibitor, XEN901, is currently undergoing safety trials in humans.

Kathrin Meyer of Nationwide Childrens Hospital in Columbus, Ohio, addressed the possibility of using gene therapy to normalize malfunctioning Nav1.2 channels. Meyer has been involved in several gene-therapy trials for neuromuscular disorders, including a successful one for infant-onset spinal muscular atrophy type6. Gene therapy for brain diseases was spurred by the discovery of adeno-associated virus 9 (AAV9), which can cross the bloodbrain barrier to deliver genetic material to the central nervous system. AAV9 is small, cannot replicate, does not integrate into host DNA and seems not to cause disease in humans. In considering gene therapy for SCN2A-related conditions, Meyer emphasized an approach that adds back a working copy of the gene, thus sidestepping the need for gene editing to make mutation-specific corrections. Such a treatment would only apply to those with loss-of-function mutations.

The large size of the SCN2A gene precludes its delivery by AAV9, however. As a workaround, Meyer suggested that SCN2As mRNA transcript could be targeted in an attempt to replace only the affected area of the mRNA. So far, such strategies have not been very efficient, but there are new ideas that might address some of the difficulties. Because access to tissue samples of patients with neurological disorders is limited, the development and testing of new therapies is complicated. Meyer suggested developing gene therapies in vitro using neurons reprogrammed from skin cells of patients. This might help identify which patients would react best to a certain treatment. There is likely not a one-fit-for-all situation, she said.

SFARI deputy scientific director John Spiro underscored the need for in vitro systems, citing the organizations initiative to bank blood cells to systematically generate induced pluripotent stem cells from individuals with autism. Simons Searchlight is also a resource of many different biospecimens for researchers. So far, 186 families with SCN2A-related changes have registered, and 83 of these have completed consent, lab reports and medical histories with a large number of blood samples as well. (On the sidelines of the meeting, 18 parents, 11 of their children with SCN2A mutations, and three unaffected siblings donated blood toward this initiative.) Spiro also stressed a need to come up with more quantitative methods of phenotyping, such as wearable electronics that can monitor sleep and circadian rhythms. Data that can be collected longitudinally and at home might provide sensitive outcome measures for clinical trials.

A new role for Nav1.2 has been revealed in recent work described by SFARI Investigator Kevin Bender of the University of California, San Francisco: the channels mediate back-propagating action potentials, which travel into the dendritic trees of neurons. Mice engineered to lack one copy of SCN2A a situation that mimics people with truncating SCN2A mutations that render the resulting Nav1.2 channels useless had cortical neurons with slower action potentials, reduced dendritic excitability and immature synapses based on their shape and function7. This role for Nav1.2 was particularly important later in development: when conditional knockout mice lost an SCN2A copy later in life, their cortical neurons exhibited immature synapses, though their density remained normal. Preliminary experiments suggest that adding back a working copy of SCN2A later in life through transgenic methods or by upregulating transcription of the remaining good copy of SCN2A via CRISPR techniques can restore action potential velocity and synaptic maturity.

Bender stressed how interacting with the SCN2A family group helped focus his research on important aspects of their childrens conditions. For example, parents have noted sensory hypersensitivity in their children, leading Bender to collaborate with colleague Evan Feinberg to use an eye-tracking assay in mice to measure their visual responses. He noted that SCN2A haploinsufficient mice were more sensitive to certain visual stimuli than control mice; if the assay is robust, it could help bridge the gap between SCN2A-related phenotypes in humans and behaviors measured in mice.

As meeting attendees sorted through the new findings, therapeutic questions lingered. An important issue for any therapy, whether drug or gene, will be how early in development one will have to intervene to help someone with an SCN2A mutation. Bender noted that synaptic properties could be rescued in his mice when they were 30 days old equivalent to a 10-year-old human but these and other experiments will have to probe the time periods during which therapies will be maximally effective. To find good measures of efficacy also means understanding the full complement of conditions that beset people with SCN2A mutations. For example, though seizures afflict many, Keith Coffman of Childrens Mercy Hospital in Kansas City, Missouri, suggested that, in some cases, these represent a movement disorder rather than epilepsy. Basic descriptive knowledge like this is imperative for guiding future treatment approaches.

Another smaller SCN2A meeting is planned for this year from July 30 to August 2, in Columbus, Ohio. This will be more family focused, says Schust, and there will be opportunities to participate in research.

There is clearly a lot more work to do before all the terrific basic research that was discussed at this meeting produces meaningful results for families, but it is extremely gratifying to see how much progress has been made on so many fronts and how many new good ideas are emerging, Spiro says. And its terrific to witness firsthand the positive cycle of how families drive researchers and vice versa.

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Cadets Research Bioprinting to Improve Soldier Care – MilitarySpot

By daniellenierenberg

FEBRUARY 19, 2020 When Lt. Col. Jason Barnhill traveled to Africa last summer, he took with him not only the normal gear of an Army officer, but also a 3D printer.

Barnhill, who is the life science program director at the U.S. Military Academy, traveled to Africa to study how 3D printers could be used for field medical care. Barnhills printer was not set up to print objects made out of plastics as the printers are frequently known for. Instead, his printer makes bioprinted items that could one day be used to save Soldiers injured in combat.

The 3D bioprinting research has not reached the point where a printed organ or meniscus can be implanted into the body, but Barnhill and a team of cadets are working to advance the research in the field.

Twenty-six firsties are doing bioprinting research across seven different projects as their capstone this year. Two teams are working on biobandages for burn and field care. Two teams are working on how to bioengineer blood vessels to enable other bioprinted items that require a blood source, such as organs, to be viable. One team is working on printing a viable meniscus and the final team is working on printing a liver.

The basic process of printing biomaterial is the same as what is used to print a plastic figurine. A model of what will be printed is created on the computer, it is digitally sliced into layers and then the printer builds it layer by layer. The difference is the ink that is used.

Instead of heating plastic, 3D bioprinting uses a bioink that includes collagen, a major part of human tissue, and cells, typically stem cells.

A lot of this has to do with the bioink that we want to use, exactly what material were using as our printer ink, if you will, Class of 2020 Cadet Allen Gong, a life science major working on the meniscus project, said. Once we have that 3D model where we want it, then its just a matter of being able to stack the ink on top of each other properly.

Cadets are researching how to use that ink to create a meniscus to be implanted into a Soldiers injured knee or print a liver that could be used to test medicine and maybe one day eliminate the shortage of transplantable organs.

The research at West Point is funded by the Uniformed Services University of Health Science and is focused on increasing Soldier survivability in the field and treating wounded warriors.

Right now, cadets on each of the teams are in the beginning stages of their research before starting the actual printing process. The first stage includes reading the research already available in their area of focus and learning how to use the printers. After spring break, they will have their first chance to start printing with cells.

For the biobandage, meniscus and liver teams, the goal is to print a tangible product by the end of the semester, though neither the meniscus or liver will be something that could be implanted and used.

There are definitely some leaps before we can get to that point, Class of 2020 Cadet Thatcher Shepard, a life science major working on the meniscus project, said of actually implanting what they print. (We have to) make sure the body doesnt reject the new bioprinted meniscus and also the emplacement. There can be difficulties with that. Right now, were trying to just make a viable meniscus. Then, well look into further research to be able to work on methods of actually placing it into the body.

The blood vessel teams are further away from printing something concrete because the field has so many unanswered questions. Their initial step will be looking at what has already been done in the field and what questions still need to be answered. They will then decide on the scope and direction of their projects. Their research will be key to allowing other areas of the field to move forward, though. Organs such as livers and pancreases have been printed, so far, they can only be produced at the micro level because they have no blood flow.

Its kind of like putting the cart before the horse, Class of 2020 Cadet Michael Deegan, a life science major working on one of the blood vessel projects, said. Youve printed it, great, but whats the point of printing it if its not going to survive inside your body? Being able to work on that fundamental step thats actually going to make these organs viable is what drew me and my teammates to be able to do this.

While the blood vessel, liver and meniscus projects have the potential to impact long-term care, the work being done by the biobandage teams will potentially have direct uses in the field during combat. The goal is to be able to take cells from an injured Soldier, specifically one who suffers burns, and print a bandage with built in biomaterial on it to jumpstart the healing process.

Medics would potentially be deployed with a 3D printer in their Humvee to enable bandages to be printed on site to meet the needs of the specific Soldier and his or her exact wound. The projects are building on existing research on printing sterile bandages and then adding a bioengineering element. The bandages would be printed with specialized skin and stem cells necessary to the healing process, jumpstarting healing faster.

Were researching how the body actually heals from burns, Class of 2020 Cadet Channah Mills, a life science major working on one of the biobandage projects, said. So, what are some things we can do to speed along that process? Introducing a bandage could kickstart that healing process. The faster you start healing, the less scarring and the more likely youre going to recover.

The meniscus team is starting with MRI images of knees and working to build a 3D model of a meniscus, which they will eventually be able to print. Unlike a liver, the meniscus doesnt need a blood flow. It does still have a complex cellular structure, though, and a large part of the teams research will be figuring out how and when to implant those cells into what theyre printing.

Of the 26 cadets working on bioprinting projects, 17 will be attending medical school following graduation from West Point. The research they are doing gives them hands-on experience in a cutting-edge area of the medical field. It also enabled them to play a role in improving the care for Soldiers in the future, which will be their jobs as Army doctors.

Being on the forefront of it and just seeing the potential in bioengineering, its pretty astounding, Gong said. But it has also been sobering just to see how much more complicated it is to 3D print biomaterials than plastic.

The bioprinting projects will be presented during the academys annual Projects Day April 30.

By Brandon OConnor

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Chimerix to Report Fourth Quarter and Year End 2019 Financial Results and Provide an Operational Update on February 25, 2020 – Yahoo Finance

By daniellenierenberg

DURHAM, N.C., Feb. 18, 2020 (GLOBE NEWSWIRE) -- Chimerix(CMRX), a biopharmaceutical company focused on accelerating the development of medicines to treat cancer and other serious diseases, today announced that it will host a live conference call and audio webcast on Tuesday, February 25, 2020 at 8:30 a.m. ET to report financial results for the fourth quarter and full-year ended December 31, 2019, and to provide a business overview.

To access the live conference call, please dial (877) 354-4056 (domestic) or (678) 809-1043 (international) at least five minutes prior to the start time, and refer to conference ID 1397800. A live audio webcast of the call will also be available on the Investors section of the Company's website, http://www.chimerix.com. An archived webcast will be available on the Chimerix website approximately two hours after the event.

AboutChimerix

Chimerix is a development-stage biopharmaceutical company dedicated to accelerating the advancement of innovative medicines that make a meaningful impact in the lives of patients living with cancer and other serious diseases. The two clinical-stage development programs are dociparstat sodium (DSTAT) and brincidofovir (BCV).

Dociparstat sodium is a potential first-in-class glycosaminoglycan compound derived from porcine heparin that has low anticoagulant activity but retains the ability to inhibit activities of several key proteins implicated in the retention and viability of AML blasts and leukemic stem cells in the bone marrow during chemotherapy (e.g., CXCL12, selectins, HMGB1, elastase). Mobilization of AML blasts and leukemic stem cells from the bone marrow has been associated with enhanced chemosensitivity and may be a primary mechanism accounting for the observed increases in EFS and OS in Phase 2 with DSTAT versus placebo. Randomized Phase 2 data suggest that DSTAT may also accelerate platelet recovery post-chemotherapy via inhibition of platelet factor 4, a negative regulator of platelet production that impairs platelet recovery following chemotherapy. BCV is an antiviral drug candidate in development as a medical countermeasure for smallpox. For further information, please visit the Chimerix website, http://www.chimerix.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include those relating to, among other things, Chimerixs ability to develop BCV as a medical countermeasure for smallpox; Chimerixs ability to submit and/or obtain regulatory approvals for BCV; and Chimerixs ability to enter into a procurement contract for BCV as a medical countermeasure. Among the factors and risks that could cause actual results to differ materially from those indicated in the forward-looking statements are risks that BCV may not obtain regulatory approval from theFDAor such approval may be delayed or conditioned; risks that development activities related to BCV may not be completed on time or at all; Chimerixs reliance on a sole source third-party manufacturer for drug supply; risks that ongoing or future trials may not be successful or replicate previous trial results, or may not be predictive of real-world results or of results in subsequent trials; risks and uncertainties relating to competitive products and technological changes that may limit demand for our drugs; risks that our drugs may be precluded from commercialization by the proprietary rights of third parties; risks related to procurement of brincidofovir for the treatment of smallpox and additional risks set forth in the Company's filings with theSecurities and Exchange Commission. These forward-looking statements represent the Company's judgment as of the date of this release. The Company disclaims, however, any intent or obligation to update these forward-looking statements.

CONTACT:Investor Relations: Michelle LaSpaluto919 972-7115ir@chimerix.com

Will OConnorStern Investor Relationswill@sternir.com 212-362-1200

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BrainStorm Announces Operational Highlights and Financial Results for the Year Ended December 31, 2019 Conference Call and Webcast @ 8:00 am Eastern…

By daniellenierenberg

NEW YORK, Feb. 18, 2020 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, today announces financial results for fiscal year ended December 31, 2019.

2019 was a tremendous year for BrainStorm, with significant progress and achievements across all clinical and operational fronts, stated Chaim Lebovits, President and Chief Executive Officer of BrainStorm. Most importantly, we fully enrolled our pivotal, double blind, placebo-controlled Phase 3 trial of NurOwn for the treatment of ALS. We announced the trial conducted at six major U.S. medical centers of excellence for ALS, was fully enrolled on October 11, 2019, and on October 28, 2019 the Data and Safety Monitoring Board (DSMB), completed the second planned interim safety analysis for the first 106 patients who received repeat dosing of NurOwn in the Phase 3 trial. The DSMB concluded the trial should continue as planned without any clinical protocol changes. He added, In addition, one of the most prestigious peer-reviewed journals, Neurology, published NurOwn Phase 2 Randomized Clinical Trial in ALS: Safety, Clinical and BioMarker Results, bringing news of our investigational therapy to the global scientific community. And, just last week, we were happy to announce that the Company recently held a high level meeting with the U.S. Food and Drug Administration (FDA) to discuss potential NurOwn regulatory pathways for approval in ALS.

Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer of BrainStorm added, 2019 was also a very significant year for those who suffer from progressive Multiple Sclerosis (MS). In February 2019, we announced Cleveland Clinic would serve as our first contracted site for a Phase 2 open-label, multicenter study of repeated intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive MS (NCT03799718). We enrolled our first patient in March. We contracted with The Stanford University School of Medicine, The Keck School of Medicine of the University of Southern California, and the Mount Sinai Medical Center to further enroll patients. Dr. Kern added, The importance of our research in progressive MS was acknowledged by a $495,000 grant award from the National Multiple Sclerosis Society through its Fast Forward Program, and mid-December, the Data Safety Monitoring Board completed the first, pre-specified interim analysis, of safety outcomes for 9 participants and after careful review of all available clinical trial data, the DSMB unanimously concluded that the study should continue as planned without any protocol modification. As of December 31, 2019 we have enrolled 10 patients in the study (50% enrollment completed).

Fourth Quarter Corporate Highlights:

Financial Results for the Year Ended December 31, 2019 and Recent Updates

For further details on BrainStorms financials, including financial results for the year ended December 31, 2019, refer to the Form 10-K filed with the SEC today.

Conference Call on Tuesday, February 18th @ 8:00 am Eastern Time

The investment community may participate in the conference call by dialing the following numbers:

Those interested in listening to the conference call live via the internet may do so by visiting the Investors & Media page of BrainStorms website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

A webcast replay of the conference call will be available for 30 days on the Investors & Media page of BrainStorms website:

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwnCellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement as well as through its own patents, patent applications and proprietary know-how. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(U.S.FDA) and theEuropean Medicines Agency(EMA) in ALS. Brainstorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S.FDAapproval of autologous MSC-NTF cells in ALS. Brainstorm received U.S.FDAclearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) inDecember 2018and has been enrolling clinical trial participants sinceMarch 2019. For more information, visit the company'swebsite.

Safe-Harbor StatementStatements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: 862-397-8160pshah@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

BRAINSTORM CELL THERAPEUTICS INC.

CONSOLIDATED BALANCE SHEETSU.S. dollars in thousands(Except share data)

BRAINSTORM CELL THERAPEUTICS INC.

CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSSU.S. dollars in thousands(Except share data)

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Family honors the memory of loved one by collecting teddy bears for families dealing with cancer – KTIV

By daniellenierenberg

SIOUX CITY (KTIV) - For five years now, the Ruehle family has held a teddy bear drive in memory of Mike Ruehle, who passed from cancer in 2014.

The teddy bears are given to adults and children with a cancer connection, in Mike's memory.

Family and friends knew Mike as "Bear."

The family asks people to consider donating a teddy bear in memory of a loved one, or in honor of healthy family members.

If you'd like to donate, every bear must be new with the tag still attached. The donations will stay in Siouxland.

There are five different locations where you can drop off the bears:

Ruehle's family says the teddy bear drive has donated more than 3,000 stuffed animals to several Siouxland agencies.

But who is the man who inspired the teddy bear drive?

"Very thoughtful person, very giving, he always took the time to listen to other people, just a really compassionate, kindhearted person," said Kerry Ruehle, Mike's Widow.

Mike Ruehle, or as his family called him, Bear, passed away six years ago, after a 12 year battle with cancer.

The family said he was very active in the community and was often coaching numerous sports teams.

They said he had a big heart, and always did what he could to make others feel better, even while he himself had cancer.

"If there were any patients his doctor had, who were having a difficult time with the news or with the adjustment. He would reach out to my dad, and see if my dad would sit down with them and talk about what was going on with them," said Rhett, Mike's son.

Part of the reason the teddy bear drive was started was because of Bear's compassion towards children who were also dealing with cancer.

"He would see a young child going through similar things that he was going through. He would always go out of his way to talk to that little kid and brighten their day a little bit," said Rhett.

Mike had had three different kinds of cancers, and due to the chemotherapy, eventually was diagnosed with MDS, which is a bone marrow failure disorder.

Doctors determined that the best treatment would be a stem cell transplant. The transplant surgery went well, but months later his body began rejecting his brother's stem cells, and within five weeks he had passed away.

"It was a shock because he had been ahead of schedule and everything else. And he had come through things beautifully, so it was quite a shock. But it was god's plan I guess," said Kerry.

But Mike's family wanted a way to keep Mike's memory alive, and that's what also helped start the teddy bear drive.

"My oldest granddaughter is five, she never met her grandpa. But in some ways, she feels as though she did, because of talking about him and she helps me with the bear drive," said Kerry

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Bone Marrow Transplantation Market: Lower risk of disease recurrence and significant rise in the number of healthy donors drive the market growth -…

By daniellenierenberg

The latest market report published by Credence Research, Inc.GlobalBone Marrow Transplantation Market Growth, Future Prospects, Competitive Analysis, 2018 2026,

Access Free Sample Copy of Research Report @ https://www.credenceresearch.com/sample-request/59660

Market Insights:

Blood cancer is characterized by overproduction of an irregular type of blood cells, resulting in the overproduction of normal cells in the bone marrow preventing normal cells from performing important functions. According to the study references issued by Bristol-Myers Squibb Company by 2040 nearly 1,100,000 people will die from blood cancer, which will account for 7 percent cancer deaths.

Allogeneic bone marrow transplant are reigning the market. The inherent features associated with allogeneic procedures are the graft is free from contaminated tumor cells. Immune graft versus malignancy effect is produced by the immunocompetent cells derived from healthy donor. Low risk of disease recurrence and significant rise in the number of healthy donors drive the allogeneic bone marrow transplantation market growth. Autologous procedure will grow at a rapid pace in the near future due to rise in number of stem cell banks, which store healthy cells from patients and after conditioning treatment are introduced in them to produce healthy blood cells.

Lymphoma is dominating the indication segment for bone marrow transplantation market. There are 2 types Hodgkin lymphoma and Non-Hodgkin lymphoma. The primary factors responsible for its supremacy are significant rise in the patients newly diagnosed with Hodgkin lymphoma and excellent survival rate in patients post bone marrow transplantation procedure. Leukemia is the abnormal high production of white blood cells by the bone marrow. Technological advancement in the bone marrow transplantation technique will reduce the disease recurrence in leukemia patients receiving stem cell therapy.

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North America is presently the leading regional segment for bone marrow transplantation market with a market share of 38%. The major factors responsible for its significant growth are rising prevalence of chronic lymphocytic leukemia and presence of sophisticated healthcare infrastructure. Europe with a market share of 32% is at second position owing to significant rise in the number of myeloma patients and domicile of major players such as PromoCell GmbH, Merck Millipore Corporation and Lonza Group Ltd. Asia Pacific with a market share of 20% will grow at a faster pace in the near future on account of technological advancement in bone marrow transplantation technique and proactive government policies to curb the mortality rate associated with blood cancer.

Biotechnology firms actively engaged in bone marrow transplantation procedures are AllCells LLC., Conversant Bio., Cellular Dynamics International, Gamida Cell Ltd., Hemacare Corporation, Lonza Group Ltd., Merck Millipore Corporation, Mesoblast Ltd., PromoCell GmbH and STEMCELL Technologies.

Key Market Movements:

Browse the full reportBone Marrow Transplantation Market athttps://www.credenceresearch.com/report/bone-marrow-transplantation-market

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Credence Researchis aworldwide market research and counseling firm that serves drivingorganizations, governments, non-legislative associations, and not-for-benefits.We offer our customers some assistance with making enduring enhancements totheir execution and understand their most imperative objectives. Over almost acentury, weve manufactured a firm extraordinarily prepared to this task.

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Age Stop Switzerland Creates an Exceptional Range of Skincare Products That Can Repair and Rejuvenate Aging Skin – MENAFN.COM

By daniellenierenberg

(MENAFN - GetNews) Age Stop Switzerland luxury line of ground-breaking anti-aging solutions provides women the opportunity to regain their youthful appearance again.

Age Stop Switzerland is a premier skincare solutions company, globally reputed for their exceptional anti-aging products that are backed by scientific results. The Swiss-based beauty products developer and manufacturer have incorporated a powerful combination of innovative ingredients known to stimulate and prolong the skin's youthful appearance.

'At Age Stop Switzerland, our core philosophy is to provide our customers exceptionally advanced skincare formulations that can repair and reverse the visible signs of aging on their facial skin, says the spokesperson for Age Stop Switzerland. 'We are proud to offer you our bouquet of luxury anti-aging treatments that can make skin appear visibly renewed, softer and younger. We strive to take age-defying skin care to a new level with our exceptionally effective formulations.

The Age Stop Switzerland product line contains a high concentration of anti-aging elements in well-researched and tested combinations. Some of the innovative ingredients used in these products include bio-mimetic peptides, Swiss Snow Algae, Alpine Flower Stem Cells, hyaluronic acid, oxygen fusion, vitamins, and bio-available precious stone extracts.

The refreshingly unique and exceptionally effective snow algae powder is sourced from algae found on the snowfields of high mountains. The valuable molecules found in the algae helps protect and maintain the skin's youthful appearance.

Hyaluronic Acid is another powerful anti-aging ingredient used in many Age Stop Switzerland skincare products. Hyaluronic acid has the ability to keep skin hydrated for an extended period of time because of its unique ability to hold 1,000 times its weight in moisture.

Edelweiss plant stem cells extracted from its apical meristem is used as one of the key ingredients in their premium range of products. It works by stimulating the skin stem cells and has an inherent protection mechanism to shield human skin against harsh climatic conditions.

Age Stop Switzerland offers a complete range of anti-aging daily creams and lotions, powered by a dynamic and deeply researched mix of potent ingredients that have been proved by science to deliver real and visible results within just days of use.

The luxury anti-aging line strictly avoids the use of parabens, mineral oil, sulfates, and even any kind of synthetic color and fragrance. The products are never tested on animals.

The scientifically advanced products have been carefully formulated to trigger positive cellular changes and visible creates a rejuvenated and youthful appearance. The skin's natural beauty is enhanced by gradual reduction and elimination of wrinkles, fine lines, dark spots, and other blemishes.

For more information, visit http://www.Age-Stop.EU

About Age Stop Switzerland:

Age Stop Switzerland is a premium Swiss skin care brand committed to advancing the science of staying young. The company offers a wide range of luxury anti-aging skincare products that redefines skin protection. The products from Age Stop Switzerland are created to deliver the latest in globally proven, award-winning anti-aging science based on the revolutionary discovery of ingredients such as Swiss Snow Algae Powder and the pioneering Plant Stem Cell research.

Media Contact Company Name: Age Stop Switzerland Contact Person: Customer Support Email: Send Email Phone: +447432691133 City: Kettering State: Northamptonshire Country: United Kingdom Website: http://age-stop.eu

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SMAC mimetics and RIPK inhibitors as therapeutics for chronic inflammatory diseases – Science

By daniellenierenberg

New therapeutic approaches for chronic inflammatory diseases such as inflammatory bowel disease, rheumatoid arthritis, and psoriasis are needed because current treatments are often suboptimal in terms of both efficacy and the risks of serious adverse events. Inhibitor of apoptosis proteins (IAPs) are E3 ubiquitin ligases that inhibit cell death pathways and are themselves inhibited by second mitochondria-derived activator of caspases (SMAC). SMAC mimetics (SMs), small-molecule antagonists of IAPs, are being evaluated as cancer therapies in clinical trials. IAPs are also crucial regulators of inflammatory pathways because they influence both the activation of inflammatory genes and the induction of cell death through the receptor-interacting serine-threonine protein kinases (RIPKs), nuclear factor B (NF-B)inducing kinase, and mitogen-activated protein kinases (MAPKs). Furthermore, there is an increasing interest in specifically targeting the substrates of IAP-mediated ubiquitylation, especially RIPK1, RIPK2, and RIPK3, as druggable nodes in inflammation control. Several studies have revealed an anti-inflammatory potential of RIPK inhibitors that either block inflammatory signaling or block the form of inflammatory cell death known as necroptosis. Expanding research on innate immune signaling through pattern recognition receptors that stimulate proinflammatory NF-B and MAPK signaling may further contribute to uncovering the complex molecular roles used by IAPs and downstream RIPKs in inflammatory signaling. This may benefit and guide the development of SMs or selective RIPK inhibitors as anti-inflammatory therapeutics for various chronic inflammatory conditions.

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Getting Gray Hair Early Actually Can Mean You Are Stressed The F*ck Out – BroBible

By daniellenierenberg

Going gray is a natural part of getting older. It typically starts with a small streak of white in the hair or beard, a sign that a man has reached a certain level of maturity and is now on that steady, inevitable decline to the grave. Hell, it is even conceivable (and highly likely) that some of you might even pluck one of those pale bastards out of your pube patch one night while trying to determine if the source of a vicious case of crotch itch is the crabs or just dry skin. Listen, all were saying is dont be surprised if, within the next few years, you find yourself staring down at your junk, thinking about how that lustrous man bush of yours is starting to resemble Colonel Sanders with a skinless chicken leg dangling out of his mouth. It happens to the best of us.

But hey, getting old isnt always indicative of a silver coiffure. I knew a guy back in high school who had more gray hair on his head than my 73-year-old father does now. So, it seems that age alone isnt always the culprit. In fact, it has been long since believed that stress also plays a critical role in making some men look distinguished beyond their years. Well, come to find out, the concept of stressful events turning us into gray beasts before we are officially deemed DILFs is probably real. Only science says it really comes down to how our fight or flight response is triggered throughout the years that determine when our manes will be deprived of color.

Researchers at Harvards Stem Cell Institute believe they have found a direct correlation between stress and going gray. They recently published a study in the journal Nature, which shows that three kinds of extreme stress mild, short-term pain, psychological stress, and restricted movement has a way of bringing around the gray quicker than what would happen under natural circumstances. At least that is the conclusion reached by lead researcher Dr. Ya-Chieh Hsu and team after putting a legion of mice through the wringer and watching for their response.

The graying process happens as pigment cells called melanocytes start to fade from our hair follicles. Its just that over time, these cells become less prevalent and we begin brandishing that salt and pepper look popularized by legendary screen stars like Brad Pitt and George Clooney.

Eventually, however, all of those cells fade into extinction and our hair just goes completely white, we start receiving AARP benefits and eating apple sauce with every meal. Yet, researchers say that stress can cause these cells to fade out quicker than theyre supposed to long before we start collecting social security and yelling at the neighborhood kids to get off our lawn.

Without getting overly scientific about it, researchers found that high stress seems to produce elevated levels of a chemical in the brain known as noradrenaline. Its one of the kick-ass substances manufactured by the adrenal gland when a persons fight or flight response starts firing on all cylinders. Thats the real culprit to early aging, researchers concluded. They say that once mice were injected with this chemical, they began losing melanocytes and going gray. And the transformation didnt take long either. It turns out that stress can zap our hair color in a matter of days.

When we started to study this, I expected that stress was bad for the body but the detrimental impact of stress that we discovered was beyond what I imagined, Hsu said in a statement. After just a few days, all of the melanocyte stem cells were lost. Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.

While it might be challenging to manage stress in a way that keeps us looking young forever, there is a silver-lining here, boys. Women, presumably the root of all of that premature grayness in the first place, are especially hot for this look. Seriously, a recent survey from Match.com finds that 72 percent of the women on the dating scene find men with gray hair more attractive than those with darker dos. Other studies on the subject have turned out similar results. The only caveat is that women are really only turned on by the gray as long as it doesnt make the man look old. This has something to do with them wanting to feel like a guy can provide some safety and security without having to worry about changing his diapers down the road.

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34 Years with a New Heart and Counting | 90.1 FM WABE – WABE 90.1 FM

By daniellenierenberg

Whenever Harry Wuest has a doctors appointment in northern Atlantas hospital cluster dubbed Pill Hill, he makes sure to stop by the office of Dr. Douglas Doug Murphy for a quick chat.

And Murphy, unless hes tied up in the operating room, always takes a few minutes to say hello to his former patient. Remember when . . . ? is how the conversation typically starts, and its always tinged with laughter, often joyful, sometimes bittersweet.

Its a reunion of two men who shaped a piece of Georgias medical history.

Almost 35 years ago, Murphy opened the chest of Wuest and sewed in a new heart, giving him a second shot at life. Wuest was the third heart transplant patient at Emory University Hospital.

Tall, lanky, with short curly hair and a quiet demeanor, Wuest is the longest-surviving heart transplant recipient in Georgia and one of the longest-surviving in the world. The 75-year-old accountant still plays golf twice a week and only recently went from working full-time to part-time.

My heart is doing just fine, he says.

Murphy is now the chief of cardiothoracic surgery at Emory Saint Josephs Hospital and still in the operating room almost every day. He has moved on to become the worlds leading expert in robotically assisted heart surgery.

***

Harry Wuest is originally from Long Island, N.Y. After a stint in the U.S. Air Force, he moved to Florida to work and go to school. He wanted to become a physical education teacher. Then, in 1973, he fell ill. It started with some pain on his left side. He didnt think much of it, but when he got increasingly winded and fatigued, he went to see a doctor.

Several months and numerous specialists later, he received the diagnosis: Cardiomyopathy, a disease of the heart muscle that can make the heart become enlarged, thick and rigid, preventing it from pumping enough blood through the body.

They didnt know how I got it, says Wuest, sitting back in a brown leather armchair in the dark, wood-paneled living room of his Stone Mountain home. Maybe it was a virus. And back then, there wasnt much they could do to treat it, except bed rest.

For the next 12 years, Wuest lived life as best as he could. He got a degree in accounting from the University of Central Florida and worked for a real estate developer. There were good days, but there were more bad days. He was often too weak to do anything, and his heart was getting bigger and bigger.

***

The first successful human-to-human heart transplant was performed in Cape Town, South Africa, in 1967 a medical breakthrough that catapulted the surgeon, Dr. Christiaan Barnard, onto the cover of Life magazine and to overnight celebrity status.

This highly publicized event was followed by a brief surge in the procedure around the world, but overall, heart transplants had a rocky start. Most patients died shortly after the surgery, mainly due to organ rejection. Back then, immunosuppressive drugs, which can counteract rejection, were still in their infancy. Many hospitals stopped doing heart transplants in the 1970s.

That changed with the discovery of a highly effective immunosuppressive agent. Cyclosporine got FDA approval in 1983 and altered the world of organ transplants.

It was shortly thereafter when Emory University Hospital decided to launch a heart transplant program, but none of the senior surgeons wanted to do it. Even with the new drug, it was a risky surgery, and mortality was still high.

Its an all-or-nothing operation, Murphy says, as he sits down in his small office overlooking the greyish hospital compound. Hes wearing light blue scrubs from an early morning surgery. At 70, he still has boyish looks, with a lean build and an air of laid-back confidence. If you have a number of bad outcomes initially, it can be detrimental to your career as a surgeon, he says.

But Murphy didnt really have a choice. He remembers that during a meeting of Emorys cardiac surgeons in 1984, he was paged to check on a patient. When he returned, the physicians congratulated him on being appointed the head of the new heart transplant program. He was the youngest in the group and had been recruited from Harvards Massachusetts General Hospital just three years before.

Yeah, thats how I became Emorys first transplant surgeon, says Murphy.

He flew to California to shadow his colleagues at Stanford University Hospital, where most heart transplants were performed at the time. Back home at Emory, he put together a team and rigorously rehearsed the operation. The first transplant patient arrived in April 1985. The surgery was successful, as was the second operation less than a month later.

Around the same time, Harry Wuest wound up in a hospital in Orlando. He needed a transplant, but none of the medical centers in Florida offered the procedure. One of his doctors recommended Emory, and Wuest agreed. I knew I was dying. I could feel it. He was flown to Atlanta by air ambulance and spent several weeks in Emorys cardiac care unit until the evening of May 23, when Murphy walked into his room and said, Weve got a heart.

***

The heart, as the patient later learned, came from a 19-year-old sophomore at Georgia Tech who had been killed in a car crash.

Organ transplants are a meticulously choreographed endeavor, where timing, coordination and logistics are key. While Murphy and his eight-member team were preparing for the surgery, Wuest was getting ready to say farewell to his family his wife and three teenage sons and to thank the staff in the cardiac ward.

I was afraid, he recalls, especially of the anesthesia. It scared the heck out of me. He pauses during the reminiscence, choking briefly. I didnt know if I was going to wake up again.

The surgery took six hours. Transplants usually happen at night because the procurement team, the surgeons who retrieve different organs from the donor, only start working when regularly scheduled patients are out of the operating room.

Despite the cultural mystique surrounding the heart as the seat of life, Murphy says that during a transplant surgery, its not like the big spirit comes down to the operating room. Its very technical. As the team follows a precise routine, emotions are kept outside the door. We dont have time for that. Emotions come later.

After waking up from the anesthesia, Wuests first coherent memory was of Murphy entering the room and saying to a nurse, Lets turn on the TV, so Harry can watch some sports.

Wuest spent the next nine days in the ICU and three more weeks in the hospital ward. In the beginning, he could barely stand up or walk, because he had been bedridden weeks before the surgery and had lost a lot of muscle. But his strength came back quickly. I could finally breathe again, he says. Before the surgery, he felt like he was sucking in air through a tiny straw. I cannot tell you what an amazing feeling that was to suddenly breathe so easily.

Joane Goodroe was the head nurse at Emorys cardiovascular post-op floor back then. When she first met Wuest before the surgery, she recalls him lying in bed and being very, very sick. When she and the other nurses finally saw him stand up and move around, he was a whole different person.

In the early days of Emorys heart transplant program, physicians, nurses and patients were a particularly close-knit group, remembers Goodroe, whos been a nurse for 42 years and now runs a health care consulting firm. There were a lot of firsts for all of us, and we all learned from each other, she said.

Wuest developed friendships with four other early transplant patients at Emory, and he has outlived them all.

When he left the hospital, equipped with a new heart and a fresh hunger for life, Wuest made some radical changes. He decided not to return to Florida but stay in Atlanta. Thats where he felt he got the best care, and where he had found a personal support network. And he got a divorce. Four months after the operation, he went back to working full-time: first in temporary jobs and eventually for a property management company.

After having been sick for 12 years, I was just so excited to be able to work for eight hours a day, he recalls. That was a big, big deal for me.

At 50, he went back to school to get his CPA license. He also found new love.

Martha was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.

Having worked in the transplant office, I saw the good and the bad, Martha Wuest says. A petite woman with short, perfectly groomed silver hair, she sits up very straight on the couch, her small hands folded in her lap.Not every transplant patient did as well as Harry. And I had a lot of fear in the beginning. Now he may well outlive her, she says with a smile and a wink.

Wuests surgeon, meanwhile, went on to fight his own battles. Two and a half years into the program, Murphy was still the only transplant surgeon at Emory and on call to operate whenever a heart became available. Frustrated and exhausted, he quit his position at Emory and signed up with Saint Josephs (which at the time was not part of the Emory system) and started a heart transplant program there.

At St. Joes, Murphy continued transplanting hearts until 2005. In total, he did more than 200 such surgeries.

Being a heart transplant surgeon is a grueling profession, he says, and very much a younger surgeons subspecialty.

He then shifted his focus and became a pioneer in robotically assisted heart surgery.He has done more than 3,000 operations with the robot, mostly mitral valve repairs and replacements more than any other cardiac surgeon in the world.

***

Since Murphy sewed a new heart into Wuest, 35 years ago, there has been major progress in the field of heart transplants,but it has been uneven.

Medications to suppress the immune system have improved, says Dr. Jeffrey Miller, a transplant surgeon and heart failure specialist at Emory. As a result, we are seeing fewer cases of rejections of the donor heart.

Also, there are new methods of preserving and transporting donor hearts.

Yet patients requiring late-stage heart failure therapy, including transplantation, still exceed the number of donor hearts available. In 2019, 3,551 hearts were transplanted in the United States, according to the national Organ Procurement and Transplantation Network. But 700,000 people suffer from advanced heart failure, says the American Heart Association.

New technologies and continued research are providing hope to many of these patients. There has been significant progress in the development of partial artificial hearts, known as Left Ventricular Assist Devices, or LVADs, says Miller.

These are implantable mechanical pumps that assist the failing heart. Patients are back out in society living normal lives while theyre waiting for their donor hearts, he explains.

LVADs are used not only as bridge devices but as destination therapy as well, maintaining certain patients for the remainder of their lives.

Also, total artificial hearts have come a long way since the first artificial pump was implanted in a patient in 1969.

Long-term research continues into xenotransplantation, which involves transplanting animal cells, tissues and organs into human recipients.

Regenerative stem cell therapy is an experimental concept where stem cell injections stimulate the heart to replace the rigid scar tissue with tissue that resumes contraction, allowing for the damaged heart to heal itself after a heart attack or other cardiac disease.

Certain stem cell therapies have shown toreverse the damage to the heart by 30 to 50 percent, says Dr. Joshua Hare, a heart transplant surgeon and the director of the Interdisciplinary Stem Cell Institute at the University of Miamis Miller School of Medicine.

All of these ideas have potential, says Miller. But they have a lot of work before were ready to use them as alternatives to heart transplantation. I dont think were talking about the next few years.

Besides Emory, other health care systems in Georgia that currently have a heart transplant program are Piedmont Healthcare, Childrens Healthcare of Atlanta and Augusta University Health.

Organ rejection remains a major issue, and long-term survival rates have not improved dramatically over the past 35 years. The 10-year survival is currently around 55 percent of patients, which makes long-term-survivors like Harry Wuest rare in the world of heart transplants.

The United Network of Organ Sharing, or UNOS, which allocates donor hearts in the United States, doesnt have comprehensive data prior to 1987. An informal survey of the 20 highest-volume hospitals for heart transplants in the 1980s found only a scattering of long-term survivors.

***

Being one of the longest-living heart transplant recipients is something that Wuest sees as a responsibility to other transplant patients, but also to the donors family, which hes never met. If you as a transplant recipient reject that heart, thats like a second loss for that family.

Part of this responsibility is living a full and active life. Both he and Martha have three children from their previous marriages, and combined they have 15 grandchildren. Most of their families live in Florida, so they travel back and forth frequently. Wuest still works as a CPA during tax season, and he does advocacy for the Georgia Transplant Foundation. In addition to golf, he enjoys lifting weights and riding his bike.

Hes had some health scares over the years. In 2013, he was diagnosed with stage 1 kidney cancer, which is in remission. Also, he crossed paths with his former surgeon, and not just socially. In 2014, Murphy replaced a damaged tricuspid valve in Wuests new heart. That operation went well, too.

Murphy says there are several reasons why Wuest has survived so long. Obviously, his new heart was a very good match. But a patient can have the best heart and the best care and the best medicines and still die a few months or years after the transplantation, the surgeon says. Attitude plays a key role.

Wuest was psychologically stable and never suffered from depression or anxiety, Murphy says. Hes a numbers guy. He knew the transplant was his only chance, and he was set to pursue it.

Wuest attributes his longevity to a good strong heart from his donor; good genetics; great doctors and nurses; and a life that he loves. Im just happy to be here, he says.

Quoting his former surgeon and friend, he adds: Doug always said, Having a transplant is like running a marathon. And Im in for the long haul.

Katja Ridderbusch is an Atlanta-based journalist who reports for news organizations in the U.S. and her native Germany. Her stories have appeared in Kaiser Health News, U.S. News & World Report and several NPR affiliates.

This is a slightly modified version of the article 34 Years with a New Heart, published by Georgia Health News on February 18, 2020.

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Could this patch help mend a broken heart? – Medical Design & Outsourcing

By daniellenierenberg

(Image from Trinity College Dublin)

Researchers in Ireland have developed a prototype patch that they say does the same job as crucial aspects of heart tissue.

The patch was designed to withstand the mechanical demands of heart tissue and mimic the electrical signaling properties that allow the heart to pump blood throughout the body. The researchers believe it brings medtech one step closer to a functional design that could mend a broken heart.

Cardiac patches lined with heart cells can be applied surgically to restore heart tissue in patients who have had damaged tissue removed after a heart attack and to repair congenital heart defects in infants and children. Ultimately, though, the goal is to create cell-free patches that can restore the synchronous beating of the heart cells, without impairing the heart muscle movement. The bioengineers report their work in the journal Advanced Functional Materials.

Researchers are continuously looking to develop new treatments which can include stem cell treatments, biomaterial gel injections and assistive devices, said senior author Michael Monaghan, an assistant professor at Trinity College Dublin, in a news release. Ours is one of few studies that looks at a traditional material, and through effective design allows us to mimic the direction-dependent mechanical movement of the heart, which can be sustained repeatably. This was achieved through a novel method called melt electrowriting and through close collaboration with the suppliers located nationally we were able to customize the process to fit our design needs.

This work was performed in the Trinity Centre for Biomedical Engineering, based in the Trinity Biomedical Sciences Institute in collaboration with Spraybase, a subsidiary of Avectas Ltd.

The mechanical demands of heart muscle cannot be met using polyester-based thermoplastic polymers, which are predominantly the approved options for biomedical applications, according to the researchers. However, the functionality of thermoplastic polymers could be leveraged by its structural geometry. They made a patch that could control the expansion of a material in multiple directions and tune this using an engineering design approach.

The patches were manufactured via melt electrowriting, a core technology of Spraybase, which the company says is reproducible, accurate and scalable. The patches were also coated with the polymer polypyrrole to provide electrical conductivity while maintaining cell compatibility. The patch withstood repeated stretching, which is a dominant concern for cardiac biomaterials, and showed good elasticity, to accurately mimic that key property of heart muscle.

Essentially, our material addresses a lot of requirements, Monaghan said. The bulk material is currently approved for medical device use, the design accommodates the movement of the pumping heart, and has been functionalized to accommodate signaling between isolated contractile tissues. This study currently reports the development of our method and design, but we are now looking forward to furthering the next generation of designs and materials with the eventual aim of applying this patch as a therapy for a heart attack.

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bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress – Yahoo Finance

By daniellenierenberg

- First conditional approval of ZYNTEGLOTM (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent -thalassemia who do not have 0/0 genotype in Europe achieved in 2019; Germany launch underway

- Announced positive top-line data from pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma

- Presented clinical data across studies of LentiGlobin gene therapy for -thalassemia (betibeglogene autotemcel) and LentiGlobin gene therapy for sickle cell disease (SCD) and bb21217 in multiple myeloma at American Society of Hematology (ASH) Annual Meeting

- Ended quarter with $1.24 billion in cash, cash equivalents and marketable securities

bluebird bio, Inc. (NASDAQ: BLUE) today reported financial results and business highlights for the fourth quarter and full year ended December 31, 2019.

"2019 was truly a transformative year for bluebird, with our first commercial product now launched in Europe and exciting progress across our first four clinical programs and pipeline," said Nick Leschly, chief bluebird. "Notably, our data in SCD continues to build, and at the ASH annual meeting in December we presented data that showed a 99% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome (ACS) in HGB-206 Group C patients with history of VOCs and ACS who had at least six months follow-up. In -thalassemia, the consistency with which patients who do not have a 0/0 genotype in our Northstar-2 (HGB-207) study are achieving transfusion independence is very encouraging and were starting to see indications that we may be able to see similar outcomes with many patients with 0/0 genotypes as well in our Northstar-3 (HGB-212 study). These data put us in a strong position as we progress our European launch, currently underway in Germany. At the end of 2019, we also announced positive top-line data from the pivotal KarMMa study of ide-cel. We and our partners at BMS look forward to submitting these data to the FDA in the first half of this year. Amidst all of our progress in 2019, our birds demonstrated time and again their dedication to patients and ability to meet and learn from the many challenges we have faced along the way. I look forward to facing the challenges of 2020 with this amazing flock."

Story continues

Recent Highlights:

TRANSFUSION-DEPENDENT -THALASSEMIA

LAUNCH IN GERMANY In January 2020, bluebird bio announced the launch of ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available in Germany. The company signed its first agreements with statutory health insurances utilizing bluebirds innovative value-based payment model and providing coverage for ZYNTEGLO for up to 50% of patients in Germany, and the first qualified treatment center was established at University Hospital of Heidelberg to provide ZYNTEGLO to patients. The company anticipates treating the first commercial patient in the first half of 2020.

UPDATED LENTIGLOBIN FOR -THALASSEMIA DATA At the American Society of Hematology (ASH) meeting in December 2019, bluebird bio presented new data from its studies of LentiGlobin gene therapy for -thalassemia (betibeglogene autotemcel) in patients with TDT: long-term data from the completed Phase 1/2 Northstar study (HGB-204), updated data from the Phase 3 Northstar-2 study (HGB-207) in patients with non-0/0 genotypes, and updated data from the Phase 3 Northstar-3 study (HGB-212) in patients with 0/0 genotypes or an IVS-I-110 mutation.

BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION bluebird bio has initiated its rolling BLA submission of LentiGlobin for -thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for -thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.

SICKLE CELL DISEASE (SCD)

HGB-211 bluebird bio is announcing today plans to launch HGB-211, the companys second Phase 3 study of LentiGlobin for sickle cell disease (SCD). This study is expected to enroll approximately 18 patients ages 2-14 years with SCD and elevated stroke risk, stroke being one of the most severe complications during childhood and adolescence. The primary endpoint of the study will be transcranial doppler response without transfusion. HGB-211 is in addition to the companys previously announced Phase 3 study (HGB-210) and is intended to support potential approval of LentiGlobin for SCD in pediatric patients at elevated stroke risk. HGB-211 is expected to begin enrolling patients in 2020.

UPDATED LENTIGLOBIN FOR SCD DATA At the ASH meeting in December 2019, bluebird bio presented new data from patients in Groups A, B and C in the Phase 1/2 HGB-206 study in patients with SCD. Group C patients are being treated under a study protocol utilizing hematopoietic stem cell (HSC) mobilization and apheresis with plerixafor, and a refined manufacturing process to increase vector copy number and engraftment potential of gene-modified HSCs. The company also disclosed that the target enrollment in HGB-206 has been achieved.

MULTIPLE MYELOMA

KARMMA TOPLINE In December 2019, Bristol-Myers Squibb and bluebird bio announced positive top-line results from the pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma. The study met its primary endpoint and key secondary endpoint, demonstrating deep and durable responses in a heavily pre-treated multiple myeloma patient population. Safety results are consistent with the data presented in CRB-401 study.

BB21217 DATA At the ASH meeting in December 2019, bluebird bio and Bristol-Myers Squibb presented updated data from ongoing CRB-402 Phase 1 study of BCMA-targeted CAR T cell therapy bb21217 in relapsed and refractory multiple myeloma. The dose escalation part of CRB-402 is complete, and the dose expansion part of the study is ongoing.

COMPANY

FORTY SEVEN COLLABORATION In November 2019, bluebird bio and Forty Seven announced that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Sevens novel antibody-based conditioning regimen, FSI-174 (anti-cKIT antibody) plus magrolimab (anti-CD47 antibody), with bluebirds ex vivo lentiviral vector hematopoietic stem cell (LVV HSC) gene therapy platform. Under the terms of the agreement, bluebird bio will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its innovative antibody-based conditioning regimen for the collaboration.

Upcoming Anticipated Milestones:

Regulatory

Submission of a BLA to the U.S. FDA for ide-cel in patients with relapsed and refractory multiple myeloma in the first half of 2020, in partnership with Bristol-Myers Squibb.

Submission of a BLA to the U.S. FDA and a Marketing Authorization Application to the European Medicines Agency for Lenti-D in patients with cerebral adrenoleukodystrophy by the end of 2020.

Clinical

Submission for presentation of ide-cel clinical data from the KarMMa study in the first half of 2020, in partnership with Bristol-Myers Squibb.

Submission for presentation of ide-cel clinical data from the CRB-401 study in 2020, in partnership with Bristol-Myers Squibb.

Initiation of the Phase 3 HGB-210 study of LentiGlobin for SCD in patients with a history of vaso-occlusive crises in the first half of 2020.

Initiation of the Phase 3 HGB-211 study of LentiGlobin for SCD in patients at risk of stroke in 2020.

Updated data presentation from ALD-102 in patients with CALD by the end of 2020.

Updated data presentation from the Northstar-2 (HGB-207) clinical study in patients with transfusion-dependent -thalassemia (TDT) and non-0/0 genotypes by the end of 2020.

Updated data presentation from the Northstar-3 (HGB-212) clinical study in patients with TDT and a 0/0 genotype or an IVS-I-110 mutation by the end of 2020.

Updated data presentation from HGB-206 clinical study in patients with SCD by the end of 2020.

Commercial and Foundation Building

ZYNTEGLO first commercial patients treated in the first half of 2020.

ZYNTEGLO access and reimbursement in additional EU countries established by the end of 2020.

Fourth Quarter and Full Year 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2019 and December 31, 2018 were $1.24 billion and $1.89 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating and commercial-readiness activities.

Revenues: Collaboration and license and royalty revenues were $10.0 million for the three months ended December 31, 2019 compared to $19.2 million for the three months ended December 31, 2018. Collaboration and license and royalty revenues were $44.7 million for the year ended December 31, 2019 compared to $54.6 million for the year ended December 31, 2018. The decrease in both periods was primarily attributable to a decrease in collaboration revenue under our arrangement with Bristol-Myers Squibb, partially offset by an increase in license and royalty revenue.

R&D Expenses: Research and development expenses were $161.8 million for the three months ended December 31, 2019 compared to $119.7 million for the three months ended December 31, 2018. Research and development expenses were $582.4 million for the year ended December 31, 2019 compared to $448.6 million for the year ended December 31, 2018. The increase in both periods was primarily driven by costs incurred to advance and expand the companys pipeline.

SG&A Expenses: Selling, general and administrative expenses were $76.2 million for the three months ended December 31, 2019 compared to $53.5 million for the three months ended December 31, 2018. Selling, general and administrative expenses were $271.4 million for the year ended December 31, 2019 compared to $174.1 million for the year ended December 31, 2018. The increase in both periods was largely attributable to costs incurred to support the companys ongoing operations and growth of its pipeline as well as commercial-readiness activities.

Net Loss: Net loss was $223.3 million for the three months ended December 31, 2019 compared to $149.0 million for the three months ended December 31, 2018. Net loss was $789.6 million for the year ended December 31, 2019 compared to $555.6 million for the year ended December 31, 2018.

LentiGlobin for -thalassemia Safety

Non-serious adverse events (AEs) observed during the HGB-204, HGB-207 and HGB-212 clinical studies that were attributed to LentiGlobin for -thalassemia were hot flush, dyspnoea, abdominal pain, pain in extremities, thrombocytopenia, leukopenia, neutropenia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

With more than five years of follow-up to date, there have been no new unexpected safety events, no deaths, no graft failure and no cases of vector-mediated replication competent lentivirus or clonal dominance. In addition, there have been no new reports of veno-occlusive liver disease (VOD) as of the data cutoff presented at ASH.

About LentiGlobin for -Thalassemia (betibeglogene autotemcel)

The European Commission granted conditional marketing authorization for LentiGlobin for -thalassemia, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin for -thalassemia adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

bluebird bio has initiated its rolling BLA submission of LentiGlobin for -thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for -thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for -thalassemia. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys financial condition, results of operations, as well as statements regarding the plans for regulatory submissions and commercialization for ZYNTEGLO and the companys product candidates, including anticipated regulatory milestones, the execution of the companys commercial launch plans, planned clinical studies, as well as the companys intentions regarding the timing for providing further updates on the development and commercialization of ZYNTEGLO and the companys product candidates. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or future clinical trials; the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; the risk that we will encounter challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product, in the adoption of value-based payment models, or in obtaining sufficient coverage or reimbursement for our products; the risk that our collaborations, including the collaborations with Bristol-Myers Squibb and Forty Seven, will not continue or will not be successful; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

bluebird bio, Inc.Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except per share data)(unaudited)

For the three months endedDecember 31,

For the year endedDecember 31,

2019

2018

2019

2018

Revenue:

Collaboration revenue

$ 7,159

$ 18,382

$ 36,469

$ 52,353

License and royalty revenue

2,838

861

8,205

2,226

Total revenues

9,997

19,243

44,674

54,579

Operating expenses:

Research and development

161,821

119,722

582,413

448,589

Selling, general and administrative

76,202

53,508

271,362

174,129

Cost of license and royalty revenue

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bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress - Yahoo Finance

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Autologous Stem Cell And Non-Stem Cell Based Therapies Market 2020-2025 Booming || Leadinf Players Fibrocell, Genesis Biopharma, Georgia Health…

By daniellenierenberg

TheGlobalAutologous Stem Cell and Non-Stem Cell Based Therapies Marketis expected to reach USD113.04 billion by 2025, from USD 87.59 billion in 2017 growing at a CAGR of 3.7% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2015 & 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

For In depth Information Get Sample Copy of this Report @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market&raksh

Some of the major players operating in the global autologous stem cell and non-stem cell based therapies market areAntria (Cro), Bioheart, Brainstorm Cell Therapeutics, Cytori, Dendreon Corporation, Fibrocell, Genesis Biopharma, Georgia Health Sciences University, Neostem, Opexa Therapeutics, Orgenesis, Regenexx, Regeneus, Tengion, Tigenix, Virxsys and many more.

The data and information included in this Global Autologous Stem Cell And Non-Stem Cell Based Therapies business report helps businesses take sound decisions and plan about the advertising and sales promotion strategy more successfully. This Autologous Stem Cell And Non-Stem Cell Based Therapies market research report is generated by taking into account a range of objectives of market research that are vital for the clients success. This report also includes strategic profiling of key players in the market, systematic analysis of their core competencies, and draws a competitive landscape for the Healthcare industry. The Global Autologous Stem Cell And Non-Stem Cell Based Therapies business report includes market shares for global, Europe, North America, Asia Pacific and South America.

Market Definition:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

In autologous stem-cell transplantation persons own undifferentiated cells or stem cells are collected and transplanted back to the person after intensive therapy. These therapies are performed by means of hematopoietic stem cells, in some of the cases cardiac cells are used to fix the damages caused due to heart attacks. The autologous stem cell and non-stem cell based therapies are used in the treatment of various diseases such as neurodegenerative diseases, cardiovascular diseases, cancer and autoimmune diseases, infectious disease.

According to World Health Organization (WHO), cardiovascular disease (CVD) causes more than half of all deaths across the European Region. The disease leads to death or frequently it is caused by AIDS, tuberculosis and malaria combined in Europe. With the prevalence of cancer and diabetes in all age groups globally the need of steam cell based therapies is increasing, according to article published by the US National Library of Medicine National Institutes of Health, it was reported that around 382 million people had diabetes in 2013 and the number is growing at alarming rate which has increased the need to improve treatment and therapies regarding the diseases.

Browse Detailed TOC Herehttps://www.databridgemarketresearch.com/toc/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market&raksh

Market Segmentation:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

Competitive Analysis:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

The global autologous stem cell and non-stem cell based therapies market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of autologous stem cell and non-stem cell based therapies market for global, Europe, North America, Asia Pacific and South America.

Major Autologous Stem Cell and Non-Stem Cell Based Therapies Market Drivers and Restraints:

Introduction of novel autologous stem cell based therapies in regenerative medicine

Reduction in transplant associated risks

Prevalence of cancer and diabetes in all age groups

High cost of autologous cellular therapies

Lack of skilled professionals

Customization of the Report:

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This Autologous Stem Cell And Non-Stem Cell Based Therapies Market report will enable both of the sides in market be an established firm or a relative new entrant. It helps the established firms to know about the moves which are being performed by their competitors and also helps the new entrants by educating them about the market situations and the industry trends. This Autologous Stem Cell And Non-Stem Cell Based Therapies Market report is quite fruitful in helping to understand the market definition and all the aspects of the market including the CAGR value and key profiles.

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Autologous Stem Cell And Non-Stem Cell Based Therapies Market 2020-2025 Booming || Leadinf Players Fibrocell, Genesis Biopharma, Georgia Health...

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Aspect Biosystems on the restorative capacity of bioprinting today – 3DPMN

By daniellenierenberg

The medical AM segment is multifaceted, consisting of 3D printed medical devices, anatomical models, prosthetics and more. Within the industry,bioprintinghas carved out a prominent position, gaining interest across the board for its huge potentials in drug development and screening, therapeutic treatments and regenerative medicine, to name but a few. While much of the excitement surrounding bioprinting is focused on the futurewhat it could dowe want to look at what is happening now in the field that is exciting.

As part of our Medical AM Focus, we asked bioprinting leaders from across the sector what they consider to be the most exciting application for their bioprinting technologies today. In this segment, we hear from Canadian bioprinting company Aspect Biosystems.

Based in Vancouver, Aspect Biosystems is a biotech company that specializes in the microfluidic 3D bioprinting of human tissues. The company has brought to market a sophisticated bioprinting systemthe RX1 Bioprinterwhich is today used by researchers all over the globe for medical applications, including neural and cardiac research. The company also conducts its own research in-house, developing therapeutic tissue programs for orthopedic and metabolic disorders.

According to Aspect Biosystems, one of the most exciting regenerative medicine applications it is currently exploring is the bioprinting of pancreatic tissue, which could help in the treatment of diabetes patients.

The moonshot for the bioprinting industry is to create whole replacement organs, the company tells us. While this is a great goal, it is still a long way off. At Aspect, we are focused on restoration of function versus replication of entire organ structure. Many diseases and disorders are a result of missing or lost function, in which case patients dont necessarily need a whole new organthey need an implantable tissue that replaces the lost function of the damaged organ.

This is particularly true of people living with type 1 diabetes. A patient with type 1 diabetes has a pancreas that does not perform its intended functions of secreting insulin and regulating blood sugar levels. Therefore, we are not aiming to create a replacement pancreas, but rather an implantable tissue therapeutic that would restore the the intended functions of the organ.

Microfluidic-based 3D bioprinting enables the creation of well-defined microstructures, thus allowing us to print a tissue structure with specific features such as immune-protection, vascularization, or production of hormones like insulin, says Aspect Biosystems. In the case of pancreatic tissue, microfluidics gives us the ability to print a multi-layered structure with a core of insulin-producing pancreatic islet cells (stem cell-derived beta-like cells) surrounded by an immuno-protective layer.

Our goal is to use these bioprinted encapsulated pancreatic beta cells as the foundation for developing an implantable 3D tissue therapeutic. Adding an outer layer that immune cells cant penetrate, but that allows diffusion of insulin, would ensure the success of the implanted tissue therapeutic as a long-term treatment for patients with type 1 diabetes without the need for immunosuppressant therapy.

As Aspect Biosystems moves forward in its bioprinting R&D, it is supported by a recent investment of $20 million, led by VC firm Radical Ventures. This injection of capital will help the company to accelerate its bioprinting workboth for the pancreatic tissue development and beyond.

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Improvements to the BioFabrication Facility on the ISS Thanks to Lithoz – 3DPrint.com

By daniellenierenberg

Scientific discoveries and research missions beyond Earths surface are quickly moving forward. Advancements in the fields of research, space medicine, life, and physical sciences, are taking advantage of the effects of microgravity to find solutions to some big problems here on Earth. Researchers in 3D printing and bioprinting have taken advantage of space facilities that are dedicated to conducting multiple experiments in orbit, such as investigating microgravitys effects on the growth of three-dimensional, human-like tissues, creating high-quality protein crystals that will help scientists develop more effective drugs, and even growing meat with 3D printing technology.

The BioFabrication Facility (BFF) by Techshot and nScrypt (Credit: Techshot)

On November 2, 2019, a Northrop Grumman Antares rocket successfully launched a Cygnus cargo spacecraft on a mission to the International Space Station (ISS). The payload aboard the Cygnus included supplies for the 3D BioFabrication Facility (BFF), like human cells, bioinks, as well as new 3D printed ceramic fluid manifolds that replaced the previously used that were printed out of polymers. According to Lithoz the company behind the 3D printed ceramic fluid manifolds they are enabling advancements in bioprinting at the ISS.

The additive manufactured ceramics have been in service since November 2019 and Lithoz claims they have proven to provide better biocompatibility than printed polymers, resulting in larger viable structures.

Lithoz, a company specializing in the development and production of materials and AM systems for 3D printing of bone replacements and high-performance ceramics, printed the ceramic manifolds using lithography-based ceramic manufacturing (LCM) on a high-resolution CeraFab printer in collaboration with Techshot, one of the companies behind the development of the BFF. Moreover, the ceramic fluid manifolds are used inside bioreactors to provide nutrients to living materials in space by the BFF.

Testing of the ceramic 3D printed manifolds is focusing on biocompatibility, precision, durability, and overall fluid flow properties; and the latest round of microgravity bioprinting in December yielded larger biological constructs than the first BFF attempts in July.

NASA engineer Christina Koch works with the BioFabrication Facility in orbit (Credit: NASA)

Techshot and Lithoz engineers and scientists worked together to optimize the design and the manufacturing processes required to make it. Techshot Senior Scientist Carlos Chang reported that its been an absolute pleasure working with Lithoz.

While Lithoz Vice President Shawn Allan suggested that their expertise in ceramic processing really made these parts happen. The success of ceramic additive manufacturing depends on working together with design, materials, and printing. Design for ceramic additive manufacturing principles was used along with print parameter control to achieve Techshots complex fluid-handling design with the confidence needed to use the components on ISS.

Headquartered in Vienna, Austria, and founded in 2011, Lithoz offers applications and material development to its customers in cooperation with renowned research institutes all over the world, benefiting from a variety of materials ranging from alumina, zirconia, silicon nitride, silica-based for casting-core applications through medical-grade bioceramics.

This work, in particular, highlighted an ideal use case for ceramic additive manufacturing to enable the production of a special compact device that could not be produced without additive manufacturing while enabling a level of bio-compatibility and strength not achievable with printable polymers. Lithoz reported that Techshot engineers were able to interface the larger bio-structures with the Lithoz-printed ceramic manifolds and that the next steps will focus on optimized integration of these components and longer culturing of the printed biological materials. While conditioned human tissues from this mission are expected to return to Earth in early 2020 for evaluation.

Back in July 2019, Gene Boland,chief scientist atTechshot, and Ken Church, chief executive officer atnScrypt, discussed the BFF at NASAs Kennedy Space Center in Port Canaveral, Florida, how they planned to use the BFF in orbit to print cells (extracellular matrices), grow them and have them mature enough so that when they return to Earth researchers can encounter a close to full cardiac strength. Church described how a tissue of this size has never been grown here on Earth, let alone in microgravity. The 3D BFF is the first-ever 3D printer capable of manufacturing human tissue in the microgravity condition of space. Utilizing adult human cells (such as pluripotent or stem cells), the BFF can create viable tissue in space through a technology that enables it to precisely place and build ultra-fine layers of bioink layers that may be several times smaller than the width of a human hair involving the smallest print nozzles in existence.

Flight engineer Andrew Morgan works with the BioFabrication Facility (Credit: NASA)

Experts suggest that bioprinting without gravity eliminates the risk of collapse, enabling organs to grow without the need for scaffolds, offering a great alternative to some of the biggest medical challenges, like supplying bioprinted organs, providing a solution to the shortage of organs.

With NASA becoming more committed to stimulating the economy in low-Earth orbit (LEO), as well as opening up the ISS research lab to scientific investigations and experiments, we can expect to learn more about some of the most interesting discoveries that could take place 220 miles above Earth. There are already quite a few bioprinting experiments taking place on the ISS, including Allevi and Made In Spaces existing Additive Manufacturing Facility on the ISS, the ZeroG bio-extruder which allow scientists on the Allevi platform to simultaneously run experiments both on the ground and in space to observe biological differences that occur with and without gravity, and CELLINKs collaboration with Made In Space to identify 3D bioprinting development opportunities for the ISS as well as for future off-world platforms. All of these approaches are expected to have an impact on the future of medicine on Earth.

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The Best Serums to Brighten and Refresh Dull, Tired Skin – gearpatrol.com

By daniellenierenberg

Heres the truth about serum: Its the biggest secret to having younger-looking, radiant skin.

You know all the other steps to staying healthyand showing it: wearing SPF, and applying retinol. You stay hydrated and eat healthy foods. You get lots of exercise and plenty of sleep. But, are you applying serums? They can give your skin some of the most immediate and impactful benefits.

Serum seeps into all three layers of your skin to work its magic at deeper levels than moisturizer ever could. Those moisturizing creams merely sit atop your skin. Theyre equally important, and shield skin from toxins, but they do little to reverse dark spots and smooth wrinkles. Serums, on the other hand, can correct hyperpigmentation and puffy eyes.

Serum brightens your skin complexion, and can preserve moisture levels in ways that moisturizers never could (funny, given its name). No two serums are alike in terms of ingredients and overall benefits, but almost all of them brighten your skin and smooth complexion. Here are seven of our favorite serums; apply them to clean, dried skin before moisturizers, SPFs, retinol or night creams.

If your skin suffers from redness or discoloration, then look for a corrective serum. SkinCeuticals gel serum uses thyme and cucumber extract to soothe irritation, as well as mulberry extract to even skin tone and gently hydrate the skin. If most moisturizers make your skin break out, then this is a gentle and top-notch moisturizing alternative.

Vitamin C is one of the best ingredients you can apply to skin for brighter, more even complexion. Glossiers serum is packed with it, along with magnesium, for an instant pick-me-up for tired or dull skin. Use it daily to preserve a fresh face, and to get ahead of the inevitable hangovers or early mornings.

The skin around your eyes is more sensitive than the rest of your face, and is much thinner, too hence why you quickly show signs of tiredness. (Its fluid buildup from a lack of rest, shining through your skin.) So, target the eyes with a special-blend serum that tightens and brightens at the same time. In OSEAs eye serum, hyaluronic acid preserves firmness and moisture levels, grape-seed-derived resveratrol shields this thin skin from toxins, and algae extract plumps and nourishes tired eyes (and alert eyes, if youre plenty rested already everyone benefits). It also prevents and helps reverse fine lines and other signs of aging.

Some serums are best worn during the day those that focus on hydration and pure brightening. Others, like corrective and exfoliating serums, are often best suited for bedtime so that they can sync with your bodys circadian rhythm to boost cellular turnover (and in turn guarantee that you wake up looking rested and more youthful). However, some serums cover both bases: Brickells night serum is ultra-hydrating so much so that its impact is maximized by wearing it overnight. Its loaded with plant stem cells, hyaluronic acid and vitamin C, and promises to magnify the benefits of a full nights sleep. Your friends and colleagues will notice the difference within days. (Assuming you arent pairing it with a lovely binge-drinking or chain-smoking habit.)

Theres an entire family of serums that focuses on exfoliation, by using ingredients that dissolve dead skin cells and reduce hyperpigmentation (dark spots and discoloration). Herbivores serum does so with alpha- and beta-hydroxy acids, all of which are naturally derived, despite their sciencey-sounding names. Theyre extracted from things like willow bark and sugar maple, and in turn leave skin bright, while minimizing pore size and smoothing skin texture.

Its hard to categorize Caudelies skin-brightening serum, since it uses the brands proprietary blend of ingredients (Viniferine, derived from grape vines) but they seem to deliver on their promise to brighten skin 62 times better than Vitamin C. All around the globe, this is what Caudelies spa customers pay top dollar for, and you can enjoy the same complexion-smoothing effects for less than a buck a day (assuming you just use a couple drops daily).

If your skin texture needs attention just as much as your skin tone, then look for a corrective and smoothing serum. This one from PCA Skin is pumped with peptides, and it waxes poetic on your skin to undo signs of aging (like the appearance of fine lines, wrinkles, dark spots and acne spots). It also helps skin retain moisture thanks to the inclusion of hyaluronic acid, rice bran extract, squalene, ensuring firmness and brightness.

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Validea’s Top Five Healthcare Stocks Based On Motley Fool – 2/16/2020 – Nasdaq

By daniellenierenberg

The following are the top rated Healthcare stocks according to Validea's Small-Cap Growth Investor model based on the published strategy of Motley Fool. This strategy looks for small cap growth stocks with solid fundamentals and strong price performance.

CORCEPT THERAPEUTICS INCORPORATED (CORT) is a small-cap growth stock in the Biotechnology & Drugs industry. The rating according to our strategy based on Motley Fool is 83% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: Corcept Therapeutics Incorporated is a pharmaceutical company engaged in the discovery, development and commercialization of drugs for the treatment of severe metabolic, psychiatric and oncologic disorders. The Company is developing mifepristone, a compound that modulates the effects of cortisol by acting as a competitive antagonist at the glucocorticoid receptor (GR). As of December 31, 2016, it had discovered three structurally distinct series of selective cortisol modulators, all of which share mifepristone's affinity for GR but, unlike mifepristone, do not bind to the progesterone receptor. It is conducting two clinical trials of its selective cortisol modulator, CORT125134. One trial is investigating CORT125134 as a treatment for patients with Cushing syndrome. The second trial is investigating the combination of CORT125134 and nab-paclitaxel (Celgene Corporation's Abraxane) to treat patients with solid-tumor cancers.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

ZYNEX INC. (ZYXI) is a small-cap growth stock in the Medical Equipment & Supplies industry. The rating according to our strategy based on Motley Fool is 83% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: Zynex, Inc. operates through the Electrotherapy and Pain Management Products segment. The Company conducts its business through its subsidiaries and the operating subsidiary is Zynex Medical, Inc. (ZMI). Its other subsidiaries include Zynex Monitoring Solutions, Inc. (ZMS) and Zynex Europe, ApS (ZEU). ZMI designs, manufactures and markets medical devices that treat chronic and acute pain, as well as activate and exercise muscles for rehabilitative purposes with electrical stimulation. ZMS is in the process of developing its blood volume monitoring product for non-invasive cardiac monitoring. ZEU intends to focus on sales and marketing its products within the international marketplace, upon receipt of necessary regulatory approvals. It markets and sells Zynex-manufactured products and distributes private labeled products. Its products include NexWave, NeuroMove, InWave, Electrodes and Batteries. ZMI devices are intended for pain management to reduce reliance on drugs and medications.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

INMODE LTD (INMD) is a small-cap growth stock in the Medical Equipment & Supplies industry. The rating according to our strategy based on Motley Fool is 79% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: Inmode Ltd is an Israel-based company. It designs, develops, manufactures and commercializes energy-based, minimally-invasive surgical aesthetic and medical treatment solutions. The Company's proprietary technologies are used by physicians to remodel subdermal adipose, or fatty, tissue in a variety of procedures including fat reduction with simultaneous skin tightening, face and body contouring and ablative skin rejuvenation treatments. Its products target a wide array of procedures including simultaneous fat killing and skin tightening, permanent hair reduction, skin appearance and texture, among others. The Company's products may be used on a variety of body parts, including the face, neck, abdomen, upper arms, thighs and intimate feminine regions. It owns six product platforms: BodyTite, Optimas, Votiva, Contoura, Triton and EmbraceRF. All are market and sell traditionally to plastic and facial surgeons, aesthetic surgeons and dermatologists, among others.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

MEDPACE HOLDINGS INC (MEDP) is a mid-cap growth stock in the Biotechnology & Drugs industry. The rating according to our strategy based on Motley Fool is 76% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: Medpace Holdings, Inc. is a clinical contract research organization. The Company provides clinical research-based drug and medical device development services. The Company partners with pharmaceutical, biotechnology, and medical device companies in the development and execution of clinical trials. The Company's drug development services focus on full service Phase I-IV clinical development services and include development plan design, coordinated central laboratory, project management, regulatory affairs, clinical monitoring, data management and analysis, pharmacovigilance new drug application submissions, and post-marketing clinical support. The Company also provides bio-analytical laboratory services, clinical human pharmacology, imaging services, and electrocardiography reading support for clinical trials. The Company's operations are principally based in North America, Europe, and Asia.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

CRISPR THERAPEUTICS AG (CRSP) is a mid-cap growth stock in the Biotechnology & Drugs industry. The rating according to our strategy based on Motley Fool is 72% based on the firms underlying fundamentals and the stocks valuation. A score of 80% or above typically indicates that the strategy has some interest in the stock and a score above 90% typically indicates strong interest.

Company Description: CRISPR Therapeutics AG is a Switzerland-based gene-editing company. It focuses on the development of transformative gene-based medicines for serious diseases. The Company develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 gene-editing platform, which allows for precise directed changes to genomic deoxyribonucleic acid (DNA). The Company has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine and rare diseases. Its lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. The Company has business operations in London and the United Kingdom, as well as research and development operations in the United States.

The following table summarizes whether the stock meets each of this strategy's tests. Not all criteria in the below table receive equal weighting or are independent, but the table provides a brief overview of the strong and weak points of the security in the context of the strategy's criteria.

For a full detailed analysis using NASDAQ's Guru Analysis tool, click here

Since its inception, Validea's strategy based on Motley Fool has returned 625.78% vs. 240.03% for the S&P 500. For more details on this strategy, click here

About Motley Fool: Brothers David and Tom Gardner often wear funny hats in public appearances, but they're hardly fools -- at least not the kind whose advice you should readily dismiss. The Gardners are the founders of the popular Motley Fool web site, which offers frank and often irreverent commentary on investing, the stock market, and personal finance. The Gardners' "Fool" really is a multi-media endeavor, offering not only its web content but also several books written by the brothers, a weekly syndicated newspaper column, and subscription newsletter services.

About Validea: Validea is an investment research service that follows the published strategies of investment legends. Validea offers both stock analysis and model portfolios based on gurus who have outperformed the market over the long-term, including Warren Buffett, Benjamin Graham, Peter Lynch and Martin Zweig. For more information about Validea, click here

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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Working In Science Was A Brutal Education. Thats Why I Left. – BuzzFeed News

By daniellenierenberg

Stephanie Singleton for BuzzFeed News

Do you miss being a scientist? some people ask.

Sometimes.

When people talk about science, they usually mean people in white lab coats doing things, like solving equations on the board or preparing solutions in beakers. What they mean is science as this crude mechanism of discovery by which humans refine over decades and centuries a small kernel of knowing. What they mean is grant dollars. What they mean is wild hair. What they mean is clean, aseptic, analytical. Brainy little robot people. White.

I try to be honest about my time in science about the feeling of satisfaction I had when I plotted all of my confocal data and there was a beautiful curve depicting the drop-off in signal as one moved further down the tissue of the gonad. I think about the calculations we did on scraps of paper to check the ratios of inheritance of the genes we introduced. I think of the little side room where we took our coffee and bagels. I think of the feeling of friendship and family that comes with being in a big lab, where everyone has a place, a role, an expertise, a skill. I remember the surprise I felt when people started to come to me because I knew something, because I could help. And how rare that was for me.

For the better part of several years, I saw my labmates every day. For hours and hours. Every holiday, every break, we stayed. We worked. We supported each other. We fought. We feuded. We gossiped. We threw parties for each other. We celebrated. We said goodbye at graduations and retirements. There were people who supported me and cherished me and looked after me. People who treated me like I mattered. A lab is a family. In a way.

Science was beautiful and it was wild and it was unknowable. Science was spending days and weeks on a single experiment with no way to know if it would work and no real way to tell if it had worked. Science was like trying to find your way to a dark forest only to realize that you had always been inside of the forest and that the forest is inside of another, greater, darker forest. Science was laughing with my labmates about television the night before, about the song of the summer, about tennis, about the unruly nature of mold growing on our plates, about cheap wings at Buffalo Wild Wings. Science was being taught to think. Taught to speak. Science was a finishing school. Science was a brutal education. Science made me ruthless. Science made me understand the vast beauty of the world.

But science was also working 15 hours a day for weeks or months. Science was working weekends and holidays. Science was being called lazy for taking a break. Science was the beat of doubting silence after I answered a question put to me. Science was being told that racism was not racism. Science was being told that I was fortunate that I had running water while growing up and that I was actually privileged because there are some places that do not. Science was being told that I was mistaken for a waiter at a party because I had worn a black sweater. Science was being told that I had to work harder despite working my hardest. Science was being told that I talked too much. Science was being told that I was too loud. Science was being told that I was behind, always behind. Science was being told that I had failed but had been gifted a pass by virtue of who you are. Science was being told that I had never once been to class despite attending every session and office hour because I was mistaken for someone else.

Science was being the only black person in the program for four years. Science was saying nothing because I was tired of being corrected about the particulars of my own experience. Science was being told that I should consider moving to the other side of town where more black people live. Science was someone suggesting that I find a church in order to find community. Science was having my hair stroked and touched. Science was being told that I was articulate. Science was watching peoples eyes widen slightly in surprise when I told them what program I was in. Science was the constant humiliation of wondering if I had justified my presence or if I had made it harder for the next black person to get admitted. Science was having to worry about that in the first place.

Science was a place I ultimately left, not so much because I wanted to, but because I had to. Science is not being able to say that because I reflexively feel the rebuttal waiting on the other end of that sentence: You could have made it work if you wanted it enough. Science is not knowing whether I wanted it enough.

Does science influence your writing?

Oh, sure. I guess.

Do you write science fiction?

No, I write domestic realism.

After the above exchange, people sometimes look at me like Im joking and at any moment will drop the faade to reveal that I do in fact write and love science fiction, after all.

But no, I do not write science fiction. I think that if people knew more scientists and spent significant time in their company, they would understand that the worst possible preparation for a career as a science fiction writer is an intensive science education. My training as a scientist makes it difficult to absent myself in the way I need to in order to write good fiction. I can never turn off the part of my brain that knows about protein folding or microscopy or tissue preparation or stem cells or physics or chemistry. Writing science fiction would be an extended exercise in pedantry.

People presume that science and writing are quite different. But they are both ways of knowing. They are ways of understanding the greater mystery of the world. They are systems of knowledge and inquiry. I do not understand something until I have written it, or more accurately put, until I have written my way through it.

Science was being the only black person in the program for four years. Science was saying nothing because I was tired of being corrected about the particulars of my own experience.

I think in many ways, the best preparation for a writer is a period of prolonged and rigorous thought about a difficult and complicated question. You learn to assemble your resources. You learn to fight with yourself. You learn to quarrel on the page with your worst ideas and with the ones you hold dearest. You treat your expectations with suspicion. You demand proof. You demand evidence. You think hard about the alternate hypothesis or other explanations, and you devise strategies to root these out. You learn to live with doubt. You try to prove yourself wrong. You look for places where you have been too soft. Too vague. You eliminate language that contains falsehoods. You eliminate language that can mislead your reader. You ask questions. You pursue answers with all the energy you can muster. You try to put language to what it is you observe. You develop a stamina for iteration. You develop a thick skin. You learn to seek criticism. You treat criticism like kindness. You churn the raw material of life into something that can be understood, and when you fail, you marvel at the mystery of things.

Do you miss science?

Yes. No. Yes. No.

Sometimes, when I dont feel well, I consider the question of how to derive an expression for the degradation of a molecular species in a particular tissue under a given set of circumstances. Old calculus. I turn to YouTube lectures from MIT about thermodynamics. I think of my first winter in Madison, Wisconsin.

The first snowfall was in October. It had been a hot, rainy summer, so much so that the weather seemed to turn all at once with very little warning. I was either in the middle or at the start of my second rotation as a biochemistry graduate student, working in a biophysical chemistry lab and spending most of my day in the windowless instrument facility in the basement. My project was to deduce the effect of protein concentration on the ability of a polymer of DNA to wind itself. I spent a lot of time pipetting various liquids into each other in little cuvettes, slotting them into a machine, and then waiting for the reading. It was the kind of work to which I felt ideally suited, and I could have gone on that way forever. I had recently moved to the Midwest from Alabama to pursue a PhD, and it seemed as likely as anything else that I would go on pipetting and measuring the effect of things like DNA polymer length and protein concentration on DNA winding. It was as removed from the circumstances of my previous life as anything else, and so I didnt have a compelling reason to doubt that this would be the shape my life held.

But I remember sitting down at the desk in the lab and looking out the broad window. There was a large tree at the center of the courtyard that had recently turned yellow. Fall was there in name, but not in temperature. The labs were kept quite cold, and so I wore a sweater indoors and shucked it as soon as I got outside. But that day, I looked out of the window and saw snow drifting down. The flakes were thick and fluffy, and they seemed almost fake. It was the first time I had seen snow in years, and I was totally enamored by it. The other people in the lab were on edge because snow in October portended something dark and awful a hard winter, a long, brutal freeze. Where they saw inconvenient travel and slushy roads, I saw something beautiful if frivolous, a minor novelty. Winter came early that year, and it didnt end until the very beginning of the following summer. When I went to the lake on my birthday in early June, there was still ice in the water.

People presume that science and writing are quite different. But they are both ways of knowing.

When people ask me about my time in science, it is this day which presents itself to me in jewel-like clarity. It is the day something about my life altered irrevocably. Or perhaps it is that the snow has accumulated, the way all such moments do in life, the weight of meaning, of prophecy. Inevitability is an artifact of retrospection. It is because the snow represented a stark deviation from the previous course of events in my life, at the precise moment when my life was changing so wildly, that I remember it. It is not that the snow changed me, but it came at a point when I was starting not to resemble myself. I cannot use the snow to explain to people what my life was like in science. It has the whiff of superstition, folklore. It feels too much like a memory and not enough like an answer. I do not tell them about the snow or how it seemed a benediction at the outset of something I needed desperately to work.

It was only later that I realized this was wishful thinking, and that the snow was just snow.

Do you think youd ever go back to science?

That part of my life is over now.

Ive come to understand that what people want in such a situation is to have their own conceptions of the world confirmed. That is, they want me to say that when you leave science because you have written a novel and a book of stories and have decided to attend an MFA program in creative writing, you are doing something that is antithetical to science. People presume that it is akin to picking up and leaving your home in the middle of the night under great duress, never to return. What they want is the spectacle of the forgotten treasured item, the confirmation that something has been lost, perhaps forever.

I think if people knew what it was that I left, then theyd know better than to ask. It would be like asking someone if they were sad to have left their home with no prospect of returning. It would be like asking someone if they were sad to have left their faith behind. It would be like asking someone if they were sad to have given up some fundamental idea about who they are. It would be like asking someone if they were sad to have watched their life burn to the ground. It would be like asking someone if they were sad to have left their family and friends.

They would mind their own business if they knew.

But they do not know, and so they say things like Science, wow, thats so cool, like, do you miss it?

And I smile because that is what I have learned to do. Because explaining is too hard. Too messy. There is no clean or easy or simple way to make it known to others that I left because I had to, because it was necessary to leave that I do miss it, but I also dont because Im still that person but not that person, that every day I remind myself less of the person I was then. Its sad, like losing a memory of myself, and all those years are lost to me now, all the little tricks and habits of home dropping down and away, as I become this other person known for this other thing, and its too much in the moment to say that I miss it both more and less every day, that I become a person more capable of appreciating what is lost in the grand scheme of things but less a person who knows what it is Ive actually lost, and that there is some painful, brutal, awful misalignment in the scale of those two losses.

When people ask if I miss science, the only answer available to me is an incomplete solution to the problem: Yes. No. Sometimes. Its over now.

Brandon Taylor is the senior editor of Electric Literatures Recommended Reading and a staff writer at Literary Hub. His writing has earned him fellowships from Lambda Literary Foundation, Kimbilio Fiction, and the Tin House Summer Writer's Workshop. He holds graduate degrees from the University of Wisconsin-Madison and the University of Iowa, where he was an Iowa Arts Fellow at the Iowa Writers Workshop in fiction. Learn more about his first novel Real Life here.

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News Royal Oak native meets woman she saved with vital stem cell donation Jenn Schanz 11 – WXYZ

By daniellenierenberg

When you see Kelly Schneider and Alex Barr together, you'd think they go way back; they laugh at the same moments, seem to have inside jokes, and generally send off a vibe that they're old friends.

"Our families are both Middle Eastern, so we just have this connection. Weve just been gabbing and eating like we know each other," Schneider told Action News at her mother's house in Bloomfield Hills on Sunday.

It's where Barr and her mother came from the Boston area to meet Schneider and her family or the very first time in person, and to say thank you, since sharing something pretty personal back in August of 2018.

"I mean, she kind of is morphing into me now that she has my DNA. Thats how this works, right?" Schneider joked.

About a year earlier, in the summer of 2017 Barr, then in graduate school in the Boston area, learned she had Leukemia for the second time.

"It was just unreal. Like I couldnt even process it," Barr told Action News.

Barr didn't know it then, but Schneider had already signed up with Be The Match, and a national bone marrow registry, when she learned a close friend was diagnosed with cancer.

"We went and got tested and we donated blood. And unfortunately she did not survive. But after 4 or 5 months after she passed away, I got a call from Be The Match.

That call was on behalf of Barr, hoping Schneider might be willing to donate life-saving bone marrow.

Soon after, Schneider was getting treatment to donate stem cells from her bone marrow, to save Barr's life, who was still a stranger at the time.

All Schneider knew then was that her donation was going to help a 24-year-old from Michigan.

"How could you not? If someone needs it?" She said.

"When you hear bone marrow donation that sounds scary like theyre going to drill into your bone or something," Barr said, noting that it really wasn't as intense of a procedure as some people may think.

In this case, Schneider had to get a series of shots, the stem cells were collected, and then shipped to Boston for Barr, who is now in remission.

Its incredible. Like, I cant even describe. And I know that I would do the same," Barr said.

First, the two communicated communicated through the registry.

"We had been talking back and forth like online since September. I could tell that we would really hit it off," Barr told Action News.

Then, they decided to meet in person at Schneider's mother's house.

Not only do the two now share some of the same DNA, they keep finding other things they have in common.

Like a photo of Barr's cousin, which looks strikingly similar to Schneider.

I look like her! she said, pointing at the photo Barr brought with her.

For both Barr and Schneider, this full-circle experience is a reminder of how important the the Be The Match registry is, for the thousands of people waiting to find their life-saving donor, and just possibly, a life-long connection too.

Barr, who is now a healthy 26-year-old, is working in the health field. She works in the Hemostasis and Thrombosis Division at Beth Israel Deaconess Medical Center (BIDMC), hoping to help others who have been diagnosed with potentially terminal diseases.

Barr said her experience beating Leukemia inspired her to go into the medical field as a biologist to study diseases of the blood.

She is a currently also a volunteer with Be The Match, and conducts her own registry drives as living proof of how important the registry is and how bone marrow donations can save lives.

Click here to join the register or the learn more about the Be The Match.

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Mixed ethnic backgrounds make it that much harder to find a bone marrow transplant – MLive.com

By daniellenierenberg

ANN ARBOR, MI They searched the world for a match.

Bennett Sevack needed a bone marrow transplant. He was diagnosed with myelodysplastic syndrome after treatment for chronic lymphocytic leukemia. But there was no match for the Ann Arbor resident in the international registry.

So, his family started making calls.

Doctors first tried his siblings and cousins. When that didnt work, his sisters, friends and family spent six weeks calling and sharing flyers with synagogues around the world to encourage people to join the bone marrow registry, hoping someone with Sevacks Sephardic and Ashkenazi Jewish background would be willing to donate.

By fall, a partial match was found in an Italian obstetrics hospital in an umbilical cord blood sample. Soon after, another cord blood match was found in the U.S. It was enough to perform a transplant.

Sevack has gained an intense appreciation for donors.

You can save somebodys life..." he said. Its a blessing to do.

Finding an acceptable bone marrow donor is a challenge in general, but its that much harder for people of color and patients of complex ethnic backgrounds like Sevack, according to health care providers and families whove embarked on far-reaching searches for a match.

A white person has a 77% shot at finding a matching donor, according to Be The Match, a widely used donor registry. A black persons chances are 23%. For people of mixed backgrounds, the search can become more complex.

Part of the issue is the relatively narrow population of donors in the registry. Another part is the science: Certain HLA types, the protein that needs to match in bone marrow transplants, are more unique to specific backgrounds.

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Sevacks transplant was completed in October 2019 more than three years after his initial diagnosis, and four months after getting news that he would definitely need a transplant.

He spent eight weeks healing from his transplant and two weeks healing from a bout of graft-versus-host disease in the hematology oncology clinic on the seventh floor of Ann Arbors C.S. Mott Childrens Hospital.

On the same floor, a 9-year-old Ann Arbor-area patient undergoing chemotherapy for acute myeloid leukemia is in the same predicament that Sevack once faced. She may need a bone marrow transplant as well, but a donor match her ethnic background is Cantonese, Hispanic and Caucasian has yet to be found in the registry.

The search

Genetics play a major role in finding a good match, said Mark Vander Lugt, a pediatric bone marrow transplant specialist at C.S. Mott working with the 9-year-old. People get four HLA types from each parent, meaning donors need to match in at least six spots, though sometimes a stronger match is required.

You get combinations that are hard to find in the registry because of that, Vander Lugt said.

Not everyone needs a transplant once theyre diagnosed. Doctors often treat blood cancers and related illnesses with chemotherapy first killing off the bad cells and waiting to see if healthy cells regenerate. If they dont, a transplant replaces the bone marrow, where blood stem cells are made.

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The registry is so low on certain populations that some began taking matters into their own hands. Athena Asklipiadis founded Mixed Marrow, a nonprofit that conducts outreach to encourage multi-ethnic bone marrow and blood cell donations. She said the misconceptions of donation is one of the largest challenges in getting people registered. She started by reaching out to Facebook groups and college clubs on the West Coast, where she is based. Eventually, families began reaching out to her to help in the donor search.

People use this phrase, When theres a cure for cancer or If theres a cure for cancer, Asklipiadis said. But these are actual blood diseases and cancers that are curable. Theyre curable by the selflessness of a donor and thats the beautiful thing about it.

Sevacks sister Laurel Bernstein was part of the group of relatives and supporters who called synagogues in search of donors. She estimated they called hundreds of people and added more than 1,000 to the donor registry while searching for Sevacks match.

The nine of us all felt so energized by what happened, Bernstein said. "We had not finished calling the list, even though we got the match. We went through to the end of the list to help pay it forward, to help other people, to get that much more DNA into the database.

The transplant

For Sevack, it was umbilical cord blood that saved his life. DNA from the two samples created enough HLA type matches to begin the transplant.

Vander Lugt said the preferred transplant method is still donor transplants, because cord blood units are smaller and in more limited supply than donor cells.

Misconceptions of transplants are another roadblock. A bone marrow harvest in which a donor undergoes surgery to collect marrow from the hip is widely believed to be painful. But experts describe the process as a mild discomfort with a two-to-three day recovery time, instead.

And direct harvest is no longer the only method. Many hospitals also practice apheresis, which collects the peripheral blood that can generate new bone marrow through a process that looks like dialysis. Theres no surgery involved.

Choosing between the two collection processes is based on multiple factors, including the health of the patient undergoing general anesthesia and doctor and donor preference, Vander Lugt said.

The value

Fear of the unknown is what affected Sevack the most going into treatment. He said he had an anxiety breakdown the morning of his transplant, when he realized what was about to happen.

My heart was to the moon, Sevack said. I was fully dressed and every part of me was drenched. In about eight minutes I had gone through this journey of fear because the reality of what was going to come was upon me. (My wife) calmed me down, I changed my clothes and this journey began. Theres been plenty of times when Ive been scared to death.

Finding a transplant match taught his wife Phyllis Sevack the importance of facing each day separately. Fighting cancer was about healing every day making big battles smaller.

The reality of it hits all at once, Phyllis Sevack said. "I wouldnt want anybody to be afraid to go through it because it is every day take it day by day. And as soon as Bennett got over that hes not doing everything on one day, (he got better mentally).

Now at home, Bennett Sevack may take a year to fully heal. He looks forward to small improvements in his quality of life, like an expanded diet and doing his own grocery shopping at Meijer, he said.

Even the routine blood and platelet donations he received in treatment were invaluable to him. They saved his life.

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Every day, theyd give me sacks of stuff that made me sick to think about it, Sevack said. It was someones donation that was given, that was literally giving me life. People who donate would do an amazing service to someone. You may not know who that is but its somebody.

Potential donors can begin the registration process by filling out a questionnaire about their medical history at bethematch.org. A cheek swab will confirm whether you meet the medical guidelines for donation. You may remain on the registry for years before a match is found. More information is available here.

Wolverines for Life and the Michigan Medicine Bone Marrow Transplant department are hosting a registration drive from 10:30 a.m. to 5 p.m. Tuesday, Feb. 18 at the Pierpoint Commons lobby, 2101 Bonisteel Blvd., Ann Arbor.

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