BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND – GlobeNewswire

NEW YORK, Nov. 26, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company is proud to be a gold sponsor of the 30th International Symposium on ALS/MND.

The symposium will take place December 4 6, 2019, at the Perth Convention and Exhibition Centre in Perth, Australia. The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields.

Ralph Kern MD MHSc, BrainStorms Chief Operating and Chief Medical Officer, will deliver a podium presentation: Modulation of innate immunity by MSC-NTF (NurOwn) cells correlates with ALS clinical outcomes, on December 4, from 11:50 12:10 pm AWST during the opening day Clinical Trials Session. In addition to the podium presentation, the Company will also present Poster 153: MSC-NTF Differentiation Increases the Neurotrophic Effects of MSC Cells: Live Imaging Analysis, that directly demonstrates the neuroprotective effects of NurOwn in a neuronal cell culture model.

Our fully-enrolled phase 3 clinical trial is one of the most advanced clinical programs in ALS, stated Chaim Lebovits, President and CEO of BrainStorm. He added, The International Symposium on ALS/MND is an important venue to update the community on our clinical and scientific efforts towards the advancement of therapies that may address the unmet needs of those living with ALS. BrainStorm Cell Therapeutics is proud to serve as a sponsor of this important annual symposium which underscores our commitment to the international community of ALS and MND patients, their families and their caregivers.

Ralph Kern, MD, stated, It is a privilege to present our innovative biomarker and preclinical research at the International Symposium on ALS/MND. He added, Every year, symposium participants gather together and discuss the opportunities and the challenges that we will face during the upcoming year. Research and medical breakthroughs for the ALS and MND community continue to make significant progress and we look forward to sharing our insights and engaging with colleagues from around the globe. The International Symposium on ALS/MND reminds us how far we have come in investigational therapies and how much more progress is still needed to bring patients a better and more promising future.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic 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. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

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 NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a 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 the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at http://www.brainstorm-cell.com.

The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields. The Symposium is planned as two parallel meetings, one on biomedical research and the other on advances in the care and management of people affected by ALS/MND. Joint sessions consider issues of mutual concern, challenging current views and practices.

Safe-Harbor Statements

Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual 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, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://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.

BRAINSTORM CONTACTS:Investors:Uri Yablonka, Chief Business OfficerBrainStorm Cell Therapeutics IncPhone: : +1-201-488-0460Email: uri@brainstorm-cell.com

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

Continue reading here:
BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND - GlobeNewswire

Bedfordshire Police officer reunited with stem cell recipient – Cranfield and Marston Vale Chronicle

A Bedfordshire Police officer has been reunited with a woman whose life he saved after donating stem cells four years ago. Beatrice, a mother of two, from Palm Springs, California, was diagnosed with Myelofibrosis at the age of 23.

The condition is a rare type of blood cancer which leads to leukaemia.

She has been living with the condition for the majority of her adult life, managing the symptoms with medication and waiting for a donor match, as this is the only known treatment for this condition.

Her ethnic background Beatrice is half European and half Vietnamese meant that the chance of finding a match was extremely unlikely. In 2008, after years of searching, she gave up on ever finding a donor to focus her time on taking care of her young children. This was two months before PC Andrew Harris joined the register.

Over time her health began to deteriorate and she was in desperate need of a stem cell donation to save her life.

So in 2015, when she was about to enter into palliative care, her health care provider ran one last check on the donor register and she received the most welcome news. There was a perfect genetic match on a donor list a police officer, PC Harris, from the United Kingdom.

At the same time the news came through, Beatrices condition had deteriorated and she was very poorly. Thankfully, her health improved enough to get through the transplant procedure. The transplant was successful and after the two year waiting period, Beatrice and Andrew got in touch through Skype.

They have stayed in touch over time and met in person in London a month ago.

Read more:
Bedfordshire Police officer reunited with stem cell recipient - Cranfield and Marston Vale Chronicle

Keeping cells alive | Interviews – The Naked Scientists

One of the big challenges with making and sustaining organs in the lab is plumbing- getting nutrients in, and waste out. This is the job of the vascular system, that carries blood around the body and is vital to keeping tissues alive.Mark Skylarr-Scott and his colleagues at the Wyss Institute for Biologically Inspired Engineering at Harvard University have recently come up with a new technique for 3D printing large, vascularized human organ building blocks. The technique lays down tracks for blood vessels between clumps of cells to help keep tissues nourished. Using this method, theyve managed to keep clumps of heart cells alive for weeks, which would previously have been almost impossible. Mark took Katie Haylor through the process...

Mark- Step one would be prepare approximately one cup of stem cells. In a lab that involves using something called a bio-reactor to generate vast numbers of stem cells in three dimensions. We then need to instruct those cells to become heart cells. So we put the correct ingredients into this bio-reactor so that they are able to develop into a heart cell.

Mark- So stem cells, and then we provide chemicals that make the stem cells think that they're supposed to develop into beating heart cells. You know, this is a fairly standard process up to this point. And now if we have hundreds of millions of heart cells, a heart isn't a hundred million cells floating around in a bio-reactor. It's actually a solid organ that beats and you know, pumps out blood. We now need to think of a way to compile these together into a tissue.

Mark- So then step two, so we now have hundreds of millions of cells. At the moment, little pieces of tissue. So we actually, they're not single cells, they are in little clumps, about half a millimeter across. If we now take these little clumps and we put them in a centrifuge, we are able to sort of push them all together. We spin them down, they form, a little pellet of cells. Cool it on ice, so it's now at zero degrees.

Mark- We then come with a three D printer and inject gelatin - solid at room temperature and liquid when it's 37 degrees - in three dimensions into this group of cellular aggregates. Now, if this material were liquid, the gelatin would just sink and I wouldn't be able to create a 3D structure. If the material, if my cells were too solid, I would essentially be carving it like a turkey as I come in with a nozzle and 3D printer and inject gelatin in 3D. And this would also break the tissue. But because these cells are halfway between a liquid and a solid, I'm actually able to come in and lay material, this gelatin material in three dimensions and it will hold in place so that when I now raise the temperature, my cells all stick together. So now my tissue is become solid-like, and the gelatin that I printed melts and becomes liquid-like. If I now flush that gelatin out, I'm left with space. I'm left with channels that I can now connect a pump to those channels and actually keep the tissue alive, keep it perfused and viable.

Katie- So it's a bit like a Goldilocks porridge situation. And how does this compare to how a full scale heart would be vascularized?

Mark- This is very different in terms of the process of how we develop, but this is obviously because the goal of creating an organ for transplantation, you can't wait, you know, 20 years for an adult heart to develop, we need to be able to manufacture it quickly. So the process is very different.

Mark- In terms of the architecture, we similarly have blood vessels in our body and in our organ that start very large. The aorta comes from the heart and then it splits and it splits and you're down to what's called arterioles, little arteries. And then the arterioles become capillaries. And then the capillaries rejoin to form veins and then larger veins. Um, so this hierarchical arrangement of blood vessels we're able to reproduce, with a 3D printer - not necessarily at the resolution of capillaries, but certainly in terms of having these branched hierarchical networks.

Mark- This is actually important for transplantation. If a surgeon wants to be able to connect tissue to the patient, they don't want to have a hundred different tubes that they need to suture to connect to be able to feed that tissue. They want a single inlet that will then split, you know, and feed the full volume of the tissue and then a single outlet that they can plumb into.

Mark- I'd say the advantage of our method here is these organoids that are being developed, they really leverage biology's natural ability to make complex structures on their own. The instructions for generating the sort of patterns that you see in organs, of course, it's in our DNA. Organoid protocols, they take advantage of that to form these tissues that can exhibit amazingly complicated architectures that self assemble. They develop on their own for free essentially. So because at the smallest scale we have all of these structures already in place in the organoids, if we can now compile hundreds of thousands of these organoids together into a larger tissue that we can keep alive, we hope that through 3D printing, we get the large scale structures and vasculature necessary to keep it alive, but through developmental biology and the fact that the stem cell derived organoids that have all the microstructure already present, that we get the small scale structure in place as well.

Originally posted here:
Keeping cells alive | Interviews - The Naked Scientists

Dana-Farber joins with leading Boston teaching hospitals and universities – Mirage News

Some patients who have not responded to traditional medicines are now experiencing remarkable recoveries thanks to next-generation immunotherapies. These therapies equip a patients own immune cells to recognize, target, and destroy cancer cells. To do this, the patients cells are collected, modified, and re-introduced into their body a complex procedure currently available to only a small number of people. With major innovations underway, this fast-moving area of science is set to expand the pool of patients who will respond to immunotherapies and other emerging medicines. But there is a bottleneck in the discovery pipeline. Manufacturing backlogs are slowing the production of cells that are essential to research, holding up the availability of new treatments headed for the clinic.

To address these challenges, a group of Massachusetts academic, healthcare, biotech, and biopharma industry leaders have come together to establish a new center.

The new center for advanced biological innovation and manufacturing will explore and cultivate innovations in cell and gene therapy, advance biologic innovation and manufacturing, and accelerate developments in immunotherapy, cell therapies, gene editing, and other technologies that carry the promise of lasting impact on human health globally and boosting the local economy. By fostering collaboration and innovation, it holds the promise of speeding innovation and broadening the universe of patients that can be served by these emerging therapies.

Leaders from Dana-Farber, Harvard University, Massachusetts Institute of Technology (MIT), Fujifilm Diosynth Biotechnologies (FDB), GE Healthcare Life Sciences, Alexandria Real Estate Equities, Inc., will comprise the Board of Directors, while other contributing members include Beth Israel Deaconess Medical Center, Boston Childrens Hospital, Brigham and Womens Hospital, Massachusetts General Hospital, MilliporeSigma, and the Commonwealth of Massachusetts.

The $50 million center will be an independent non-profit organization located in the greater Boston area and will be named, along with incorporation, in the new year. The expectation is that this will be an independent, separate nonprofit corporation.

Scientific breakthroughs in cellular, immune and gene therapies from just the past few years are now saving lives and represent a truly revolutionary time in medicine, said Laurie H. Glimcher, MD, president and CEO of Dana-Farber Cancer Institute. By bringing together the talent that exists only in the Massachusetts life sciences ecosystem and fostering collaboration, this new manufacturing center will help to extend the benefit of these technologies to more patients and accelerate discoveries to effectively treat more diseases.

Home to a dense concentration of world-leading universities, hospitals, large pharmaceutical companies and small biotech firms, Massachusetts is at the forefront of biomedicine. These organizations are redefining traditional ideas about biomedicine and rapidly advancing discoveries from lab to clinic.

The overarching mission of the newly established consortium is to catalyze the development of transformative therapies by shortening the path between research and clinical application. The consortium will harness world-leading expertise to propel forward fast-emerging and promising science, the cost and risks of which are daunting for any single institution to tackle alone. By housing institutions with strengths in each link in the chain of innovation within one facility, the partners believe new innovations in both science and manufacturing will speed the introduction of new medicines to patients.

The ability of scientists to modify cells for therapeutic application, and to alter disease-causing genes, has ushered in a new era in biomedicine. Some of these potential therapies are entering clinical trials, others will soon be in the clinic, and still more are in early stages of investigation. There is strong motivation and acute need to translate these emergent approaches to clinical use. More than 60,000 patients globally are currently participating in clinical trials for new cell and gene therapies, including gene editing.

Currently, major obstacles and bottlenecks to getting new treatments into the clinic include production specifically, the pressure placed on highly skilled contract manufacturers to deliver customized cells and viral vectors of high quality and regulatory compliance to labs throughout the region. Because of the backlog, scientists may need to wait as long as 18 months for essential products they need to carry out research.

The center will offer three critical services to the Massachusetts life science ecosystem.

It will provide preferred access to a new manufacturing facility at favorable pricing, reducing the wait and cost for researchers at universities, hospitals and start-ups. The facility offers pharma-grade good manufacturing practices (GMP) manufacturing capacity in approximately eight cleanrooms for the production of cell and viral vector products and other related raw materials that may be used for phase 1 or phase 2 clinical trials.

The facility will have a shared innovation space where scientists from universities, hospitals, and industry can work side-by-side with dedicated, experienced, professional staff. This will be a unique opportunity to refine new methods rapidly, readying them for first-in-patient clinical trials. With access to manufacturing within the same space, the center will cultivate a community of experts across sectors who share a goal of serving patients, and who are dedicated to innovating collectively in both manufacturing processes and drug development.

The center will provide a platform for workforce development and training in a rapidly growing field, where there is a critical need for people with specialized skills.

The modular design of the new facility will make it easier for users to adapt quickly to changes in technology. Such flexibility will remove barriers to accessing promising innovations that emerge from improved methods involving gene manipulation, gene editing, oligonucleotides, peptides, and new methods and discoveries as they arise.

While there are many commercial contract manufacturing organizations, shared lab spaces, and even small manufacturing spaces at universities and hospitals in the U.S., this is a first-of-its-kind facility in three respects. First, for its intention to produce both cell and viral vector products within a single physical space. Second, for its unique partnerships between industry, academia, and leading area hospitals. Finally, for its partners aspirations to provide services to researchers and start-ups that will advance this new area of medicine through collaboration.

This powerful collaboration embodies the deep and broad world-class expertise in multiple disciplines that exists across this region, said Harvard President Larry Bacow. We are privileged to be part of this collaborative initiative. It will advance scientific discovery, reaffirm the regions global leadership in the life sciences, and bring forward life-saving and life-changing therapies that will make a difference for people around the world.

The broad question that we were trying to address was, How can we best position our region to be preeminent in the life sciences in the decades to come?' said Alan M. Garber, Harvards Provost, who helped conceive of the project more than two years ago and has shepherded it since then. We have a vibrant life sciences community, with some of the worlds greatest hospitals, universities, and life sciences companies of all kinds. We also have a strong financial sector that helps to spawn and support new companies. So the elements for rapid progress in the life sciences particularly in the application of the life sciences to human health are all here. But with such a rapid pace of innovation, its easy to fall behind. We wanted to make sure that would not happen here.

MIT researchers are developing innovative approaches to cell and gene therapy, designing new concepts for such biopharmaceutical medicines as well as new processes to manufacture these products and qualify them for clinical use, said MIT Provost Martin A. Schmidt. A shared facility to de-risk this innovation, including production, will facilitate even stronger collaborations among local universities, hospitals, and companies and ultimately, such a facility can help speed impact and access for patients. MIT appreciates Harvards lead in convening exploration of this opportunity for the Commonwealth.

Richard McCullough, Harvards vice provost for research and professor of materials science and engineering, who helped lead the project, said, the power of facilitys partners will accelerate therapeutic discoveries and have the ability to advance biologics from the lab to the bedside.

Its an exciting time for the life sciences industry with cell and gene therapies in position to revolutionize the global healthcare system. While these therapies are promising, challenges in manufacturing, access and cost must be addressed so they can reach their full potential. Initiatives such as the center are important because they bring together key life sciences stakeholders together to share their capabilities, knowledge and expertise to collaborate and accelerate innovation, said Emmanuel Ligner, CEO and President of GE Healthcare Life Sciences.

We are very proud to be part of this unparalleled consortium to create an innovative and collaborative center involving advanced technologies as well as next-generation manufacturing. The highly respected partner institutions have the scientific talent and the engineering capabilities to deliver truly novel therapies to patients suffering today from serious and life-threatening diseases and also to design the next-generation processes that will accelerate the translation of tomorrows cost-effective, lifesaving medicines from bench to bedside, said Joel S. Marcus, executive chairman and founder, Alexandria Real Estate Equities, Inc. and Alexandria Venture Investments.

We are excited to be a founding member of this consortia. Partnering to get medicines to patients is what we are all about. The opportunity to do this in collaboration with everyone that has come together to make this a reality is something that really meets our core purpose to deliver tomorrows medicines as a partner for life, said Martin Meeson, President & COO, FUJFILM Diosynth Biotechnologies USA.

Massachusetts new center for advanced biological innovation and manufacturing will focus first on emergent areas such as cell therapies and gene therapies, and other advanced therapy medicinal products. Cell therapies that help a patients own immune system target cancer cells have been remarkably successful. One example is CAR-T cell therapy, in which a patients own T cells are modified to identify and attack cancer cells in the blood more easily. But immunotherapy is not restricted to treating cancers. Scientists are finding new ways to harness the immune system to treat a broadspectrum of diseases, including type 1 diabetes and many others. Cell therapies more broadly harnessing unique properties of adult stem cells, for example are under wide consideration for regenerative medicine, including joint tissue repair and neurodegeneration.

Gene therapies offer new hope to patients, often children, who suffer from debilitating inherited diseases. They involve introducing, removing, or changing a targeted gene within a patients cells. The goal is to make the patients cells produce disease-fighting proteins, or to stop them from producing disease-causing versions of a protein. Gene-editing research is progressing very rapidly, but there is a marked shortage of capability for manufacturing the gene delivery vectors.

Hospitals need to be able to create customized therapeutics for their patients, but most do not have manufacturing facilities on-site. Beyond the constraint of limited facilities to produce potential new treatments, much technological innovation is required to produce these medicines more efficiently in terms of time, labor, and cost and in accordance with regulatory guidance. The new center would be equipped to handle some of this work for technology innovation and early stage clinical trial-scale production, which would directly help bring promising solutions to patients sooner.

We need more manufacturing capability in order to translate our work, especially in the stem cell field, said Leonard Zon, MD, director of the Stem Cell Research Program at Boston Childrens Hospital. For academic investigators who want to see their basic science advance into the clinic space, its important to have a manufacturing facility collaborate on protocols. Researchers can then exchange information directly with the facility, optimizing protocols and working smarter.

This collaboration represents an exciting opportunity to harness the collective efforts of leading academic, industrial and clinical institutions to further explore exciting new technologies and therapies that are inspiring scientists and offering new hope to our patients, says Peter L. Slavin, MD, MGH president. New scientific fields like regenerative medicine, gene editing and immunotherapy are unlocking clues to understanding disease which can lead to better treatments and ultimately, richer, more healthy lives for our patients and their families.

Our mission at Beth Israel Deaconess Medical Center is to provide extraordinary care supported by world-class research and education, said Peter J. Healy, president of Beth Israel Deaconess Medical Center. We are happy to be a founding member of this innovative consortium, which will allow us to work collaboratively across the diverse health care ecosystem. Together, we will propel the fields of cell therapy, gene therapy and gene editing forward with the shared goal of transforming how we care for patients right here in Boston and around the world.

Boston is an epicenter of biomedical research and innovation, said Brigham Health president Elizabeth G. Nabel, MD. In furthering the Brighams commitment to advancing development and delivery of cell and gene therapies, this unique collaboration is an opportunity to accelerate the pace and broaden the manufacturing capacity for therapies that have the potential to significantly improve patient outcomes.

Never before have we had so many breakthroughs available in the clinic. However, it can take up to 30 days, needle to needle, to deliver a CAR-T therapy to a patient, and that does not take into account any of the bottlenecks in the supply chain that could occur along the way. It is our collective responsibility to eliminate any barriers to making these life-saving medicines accessible to patients everywhere, said Udit Batra, CEO, MilliporeSigma.

The Commonwealths life sciences ecosystem is thriving because of the strength of the academic, research and industry partners that call Massachusetts home, and their commitment to collaboration, said Secretary of Housing and Economic Development Mike Kennealy. Combining a manufacturing facility, co-working labs, and workforce development and training in this first-in-the-nation center will boost the regional economy, create jobs and accelerate the delivery of next-generation therapies.

Follow this link:
Dana-Farber joins with leading Boston teaching hospitals and universities - Mirage News

Applying AI and CRISPR to stem cells to improve regenerative medicine – FierceBiotech

Human-induced pluripotent stem cells (hiPSCs) generated from a persons own adult cells can grow into complex organs that help scientists test drugs or even transplant into patients. However, directing stem cells into forming desired, functional organs in the lab remains challenging.

Now, in a study published in the journal Cell Systems, researchers from Gladstone Institutes in collaboration with Boston University (BU) described using machine learning to better understand how to use CRISPR-Cas9 gene-editing tools to control iPSC organization.

By coaxing these stem cells into forming specific arrangements, the researchers believe they could create functional organs for research or therapeutic purposes.

Like this story? Subscribe to FierceBiotech!

Biopharma is a fast-growing world where big ideas come along every day. Our subscribers rely on FierceBiotech as their must-read source for the latest news, analysis and data in the world of biotech and pharma R&D. Sign up today to get biotech news and updates delivered to your inbox and read on the go.

While researchers have managed to develop iPSCs into many different cell types but not necessarily functional 3D organs, mainly because they have struggled to manipulate the spatial patterns of stem cells, which define the tissues they eventually grow into. Some have resorted to 3D printing, but it isnt always successful, as cells often migrate away from their printed locations.

Despite the importance of organization for functioning tissues, we as scientists have had difficulty creating tissues in a dish with stem cells, Ashley Libby, a co-first author of the new study, said in a statement. Instead of an organized tissue, we often get a disorganized mix of different cell types.

The researchers previously showed that knocking down two genes, ROCK1 and CDH1, affected the layout of iPSCs in lab dishes. The proteins they encode help regulate interactions between cells, making them ideal candidates to alter the cellular organization of an iPSC group.

But there are so many variables to considerincluding the timing and level of each gene knockdown, the duration and the proportion of cells to work onthat make testing all the combinations by human almost impossible. So, they turned to machine learning for help.

RELATED:Growing transplantable arteries from stem cells

They used a CRISPR-Cas9 gene-editing system that could be triggered by adding the antibiotic doxycycline. To help link changes to specific arrangements of the iPSCs, the cells were also engineered to fluoresce in different colors when they lost ROCK1 or CDH1.

Researchers at Gladstone tested different doses and timing of gene blockade. How changes in cell subpopulations affected the observed pattern was captured, and the BU computational scientists fed the results to a machine learning algorithm, which was hence trained to classify patterns according to their similarity and infer ways of how ROCK1 and CDH1 affect iPSC organization.

Our machine-learning model allows us to predict new ways that stem cells can organize themselves, and produces instructions for how to recreate these predictions in the lab, the studys co-first author Demarcus Briers said in a statement.

The model simulated specific experimental conditionssuch as when, where and how to add drugs to the iPSCsthat could yield unique patterns in silico. Then, the team put those suggested conditions to test.

It was successful. The researchers were able to generate concentric circles to two layers of stem cell populations in a bulls-eye pattern, they reported.

We've shown how we can leverage the intrinsic ability of stem cells to organize, Todd McDevitt, the studys senior author, said in a statement. This gives us a new way of engineering tissues, rather than a printing approach where you try to physically force cells into a specific configuration.

RELATED:Nose drop with adult stem cells restores sense of smell in mice

Stem cells are a key venue for regenerative research, either for studying disease and potential treatment or for transplant. Last year, scientists from the University of Edinburgh used 3D scaffolds made of polycaprolactone to carry embryonic stem cells and iPSCs, and successfully generated functional liver tissues that help diseased mice break down the amino acid tyrosine. A research team at the Morgridge Institute for Research recently used a drug called RepSox to help iPSCs form better smooth muscle cells as building blocks for functional arteries.

For the Gladstone-BU team, the researchers are planning to expand the model to include other genes to get an even wider pool of possible cell configurations. On top of that, rather than just making flat patterns as in this study, their goal is to design 3D shapes or organs.

We're now on the path to truly engineering multicellular organization, which is the precursor to engineering organs, said McDevitt. When we can create human organs in the lab, we can use them to study aspects of biology and disease that we wouldn't otherwise be able to.

See the article here:
Applying AI and CRISPR to stem cells to improve regenerative medicine - FierceBiotech

Decoding the building blocks of life: bit bio races toward a sustainable source of human cells – Proactive Investors UK

The ability to turn human cells into anything we want sounds like the stuff of science fiction. But one Cambridge biotech says it's cracked the code

A sustainable source of human stem cells is one of the holy grails of modern medicine.

With applications as broad as re-growing failed organs, fighting cancer, and stopping animal testing, stem cell therapy is predicted to be worth US$35bn by 2023.

Now, Cambridge startup bit bio, has a new approach to re-coding skin cells from adult humans, and rewinding the clock to give them the power of stem cells, and then turn them into whatever we want them to be all without the controversial involvement of human embryos.

This, says neurosurgeon and founder Dr Mark Kotter, will democratise stem cells, so that anyone can use them, at any time.

The private sector is already placing big bets on the technology, with start-ups in the space raising as much as US$16mln in recent funding rounds.

Kotter says that our inability to produce enough human stem cells to match our need puts troubling limits on research and drug development.

In drug discovery, the biggest bottleneck is the mismatch between animal models and animal cell lines used for drug discovery, and then human setting used in the clinical trial, he explains.

Around 3% of new drugs make it all the way through trials and to market, he says, and the biggest reasons treatments tend to fail in clinical study is that they are either toxic to humans, or they dont work.

The only solution is to bring the human element back to the early stages, says Kotter.

If new therapies were tested on human tissue first, it would reduce or even bypass the need to test on animals, as well as speeding up development.

Kotter founded bit bio, formerly known as Elpis BioMed, in 2016, in addition to startup Meatable, which produces meat by growing cultures in the lab, rather than rearing animals for the table.

The time is now for bit bio, because what it is doing has only been possible since a Nobel Prize-winning discovery twelve years ago, which turned the world of stem cell research upside down.

Kyoto University researcher Shinya Yamanaka proved that it was possible to take a mature human skin cell and reprogram it to be like the stem cell of an embryo.

Until this revelation, stem cell research had been dogged by controversy and expense, as scientists had to use human embryos and umbilical cords as a source of stem cells, and then simulate complex conditions inside the womb in order to make them develop into the cells they desired.

One big problem in early cell reprogramming was that stem cells are incredibly alert to invading DNA and silences any foreign material it detects.

This meant that past attempts run a different program inside a cell often failed, because the cell destroyed it.

What happened next was a moment of "serendipity" in the lab, says Kotter.

Through trial and error, bit bio found they could use certain safe harbours where information is protected within cells, to stop theinterference.

By taking the genetic switch for gene silencing and placing it inside a safe harbour, and then separately running the new cell program inside another safe harbour, scientists found they could override gene silencing in order to change the cell type.

This approach is what Kotter says makes bit bio unique.

The lab can produce up to a kilogram of human cells now, and its tech platform OptiOx has also proved that it can generate two human cell types with 100% accuracy.

Kotter says that now the range of cells able to be produced matters more than the quantity.

The company is now focused on discovering what separates one type of cell from another, which Kotter says will allow the firm to decode the building blocks of life.

To this end, bit bio is using machine learning to analyse the differences between every type of human cell, from bone marrow cells to liver cells, and create a reference map for all the different types.

Once the research is complete, the company hopes it willbe able to generate any type of human cell, at scale, and with ultimate precision.

Preparations are underway for a Series A funding round, and Kotter says that he is determined not to sell the business, having already rejected offers from would-be buyers.

Bit bio though is in an area hot with competition, which moves quickly.

A US$16mln Series A mega funding round was recently announced in October by another Cambridge start-up, Mogrify, which is hoping to master direct cell reprogramming and turn blood cells straight into brain cells, or any other type.

Mogrify uses big data to identify the small molecules needed to convert, maintain and culture a target cell type.

While both companies were finalists in the 2018 Cambridge Startup of the Year award, bit bio was the one to scoop the prize.

One aspect that separates the two companies is that Mogrify uses its technology to turn cells directly into other cell types, rather than using it to rewindto the stem cell phase, which is when cells can reproduce very quickly,

Kotter says that this stem cell phase focusis whatallows bit bio to havea stable supply of human cells.

If bit bio completes a similar, or even bigger, fundraise, it could advance the fledgling firm from seed to stem, in its attempt to stabilise a production line for essential cell technology.

See more here:
Decoding the building blocks of life: bit bio races toward a sustainable source of human cells - Proactive Investors UK

1st SCD Trial Patient Shows CTX001 Gene Editing to be Safe, Effective – Sickle Cell Anemia News

CTX001 safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crisesin the first severesickle cell disease(SCD) patient receiving the therapy, according to preliminary data from a Phase 1/2 clinical trial.

CTX001 is a CRISPR-based gene editing therapy developed byCRISPR TherapeuticsandVertex Pharmaceuticals as a potential treatment for hemoglobin-associated diseases, includingSCD and beta-thalassemia.

It uses the CRISPR-Cas9 gene editing system to genetically modify a patients hematopoietic (bone marrow) stem cellsto produce high levels of fetal hemoglobin in red blood cells, which are then delivered back to the patient as part of a stem cell transplant.

The CRISPR-Cas9 system, which is similar to the editing system used by bacteria as a defense mechanism, allows researchers to edit parts of the genome by adding, removing, or changing specific sections of DNA.

Fetal hemoglobin, the main form of oxygen-carrying hemoglobin in the human fetus and newborn, largely disappears between six months to one year after birth, being replaced by its adult form.

Since the adult form is the one containing the defective component of hemoglobin in people with SCD and beta-thalassemia, an artificial increase of fetal hemoglobin has the potential to compensatefor the defective hemoglobin produced by these patients and reduce or prevent theirsymptoms.

The open-label, multi-center Phase 1/2 CLIMB-SCD-121 study (NCT03745287) is currently evaluating the safety and effectiveness of a single administration of CTX001 in people ages 18 to 35 with severe SCD.

The trial, which is expected to enroll up to 45 people, is stillrecruiting at 12 clinical sites in the United States, Canada, and Europe. Participants will be followed for approximately two years after treatment, and have the opportunity to enter a long-term follow-up study.

Before receiving CTX001, participants will undergo myeloablativechemotherapy, a strategy that kills cells in the bone marrow, thereby lowering the number of blood-forming cells. This way, the stem cell transplant will have more chances to rebuild a healthy bone marrow.

Researchers will first determine when the transplanted modified cells begin to produce mature blood cells in the patients, a process known as engraftment. After confirmation of engraftment, safety and effectiveness will be assessed as part of the trials primary and secondary goals.

One primary goal is to assess the proportion of people with an increase of at least 20% in the production of fetal hemoglobin, starting six months after CTX001 treatment. This increase must be sustained for more than three months at the time of analysis.

Among secondary goals is determining whether CTX001 reduces the annualized rate of vaso-occlusive crises.

In February, CRISPR Therapeutics and Vertex announced the enrollment of the first patient in the CLIMB-SCD-121 study, who was recruited in the U.S. and received CTX001 in mid-2019.

Now, the companies have shared the preliminary four-month data of this patient, a 33-year-old woman who had experienced seven vaso-occlusive crises per year the annualized rate of the two years before her enrollment in the trial.

Results showed that she had a confirmed engraftment 30 days after receiving CTX001 treatment. Four months after treatment, no vaso-occlusive crises were reported and she had stopped blood transfusion treatments.

After four months, her total hemoglobin levels were 11.3 g/dL, fetal hemoglobin levels had increased from 9.1% to 46.6%, and the percentage of fetal hemoglobin-producing red blood cells had increased from 33.9% to 94.7%.

CTX001s early safety profile was consistent with that previously reported for myeloablative chemotherapy followed by stem cell transplant. The woman experienced three serious adverse events, all of them resolved and considered to be unrelated to treatment.

Positive preliminary data were also announced for the first patient with beta-thalassemia receiving CTX001 in the Phase 1/2 CLIMB-Thal-111 study (NCT03655678).

We are very encouraged by these preliminary data [which] support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention, Samarth Kulkarni, PhD, CRISPR Therapeutics CEO, said in a press release.

A webcast and presentation about these preliminary results are available on the companys website.

The data are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia, said Jeffrey Leiden, MD, PhD, Vertexs chairman, president, and CEO.

Leiden added that the trial is still in its early phase and that he looks forward to its final results.

Early this year, CTX001 receivedfast track statusfor the treatment of sickle cell disease by theU.S. Food and Drug Administration, which is expected to accelerate CTX001s development and regulatory approval process.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

Continue reading here:
1st SCD Trial Patient Shows CTX001 Gene Editing to be Safe, Effective - Sickle Cell Anemia News

CStone announces first patient dosed in the Phase I bridging registrational study of ivosidenib – BioSpace

SUZHOU, China, Nov. 19, 2019 /PRNewswire/ -- CStone Pharmaceuticals ("CStone" or the "Company", HKEX: 2616) today announced that the first patient has been dosed in the Phase I bridging registrational study of ivosidenib (TIBSOVO) in China. This stand-alone trial is designed to validate the efficacy, safety, and pharmacokinetics of ivosidenib in patients with IDH1 mutant relapsed or refractory acute myeloid leukemia (R/R AML).

Developed by CStone's partner, Agios Pharmaceuticals (NASDAQ: AGIO), ivosidenib was approved by the U.S. FDA in July 2018 for the treatment of adult patients with R/R AML with a susceptible IDH1 mutation as detected by an FDA-approved test. In May 2019, CStone submitted a new drug application (NDA) for ivosidenib in Taiwan for the treatment of adult patients with IDH1 mutant R/R AML.

Current standard of care treatment for newly diagnosed AML patients mainly includes intensive induction chemotherapy (IC), followed by consolidation therapy such as allogeneic hematopoietic stem cell transplantation (Allo-HSCT) in order to attain durable remission. Approximately 35% to 40% of those treated patients achieve complete remission, while only about 25% achieve 3 years or longer survival. The majority of AML patients develop acquired resistance to treatment or eventually relapse, leading to R/R AML, which has a very poor prognosis in the absence of standard of care treatment options globally. With the emergence of DNA sequencing technology, the detection of genetic mutations has presented new opportunities and challenges in AML treatment. IDH1 mutations are associated with around 6% to 10% of all AML cases.

Dr. Frank Jiang, Chairman and CEO of CStone, commented: "AML is the most common acute leukemia affecting adults with over 30,000 new cases estimated in China every year. AML is characterized by its rapid progression with a five-year survival rate below 20%. We are faced with the urgent need for clinical development, particularly for IDH1 mutant R/R AML patients, due to the lack of any effective treatment in China. We will rigorously press ahead with the clinical development of ivosidenib to achieve its regulatory approval in China which will allow more AML patients in Greater China to benefit from this precision therapy."

CStone's Chief Medical Officer, Dr. Jason Yang, noted: "Ivosidenib is a potent and highly selective IDH1 inhibitor, and the only targeted therapy currently approved by the U.S. FDA for IDH1 mutant AML. It is very encouraging that we have already initiated two registrational studies of ivosidenib in China, including the global Phase III AGILE study of ivosidenib in combination with azacitidine in adult patients with newly diagnosed IDH1 mutant AML who are not eligible for intensive chemotherapy."

About TIBSOVO (ivosidenib)

TIBSOVO is indicated for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in:

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 25% (7/28) of patients with newly diagnosed AML and 19% (34/179) of patients with relapsed or refractory AML treated with TIBSOVO experienced differentiation syndrome. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal if not treated. Symptoms of differentiation syndrome in patients treated with TIBSOVO included noninfectious leukocytosis, peripheral edema, pyrexia, dyspnea, pleural effusion, hypotension, hypoxia, pulmonary edema, pneumonitis, pericardial effusion, rash, fluid overload, tumor lysis syndrome, and creatinine increased. Of the 7 patients with newly diagnosed AML who experienced differentiation syndrome, 6 (86%) patients recovered. Of the 34 patients with relapsed or refractory AML who experienced differentiation syndrome, 27 (79%) patients recovered after treatment or after dose interruption of TIBSOVO. Differentiation syndrome occurred as early as 1 day and up to 3 months after TIBSOVO initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, initiate dexamethasone 10 mg IV every 12 hours (or an equivalent dose of an alternative oral or IV corticosteroid) and hemodynamic monitoring until improvement. If concomitant noninfectious leukocytosis is observed, initiate treatment with hydroxyurea or leukapheresis, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms and administer corticosteroids for a minimum of 3 days. Symptoms of differentiation syndrome may recur with premature discontinuation of corticosteroid and/or hydroxyurea treatment. If severe signs and/or symptoms persist for more than 48 hours after initiation of corticosteroids, interrupt TIBSOVO until signs and symptoms are no longer severe.

QTc Interval Prolongation: Patients treated with TIBSOVO can develop QT (QTc) prolongation and ventricular arrhythmias. One patient developed ventricular fibrillation attributed to TIBSOVO. Concomitant use of TIBSOVO with drugs known to prolong the QTc interval (e.g., anti-arrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) and CYP3A4 inhibitors may increase the risk of QTc interval prolongation. Conduct monitoring of electrocardiograms (ECGs) and electrolytes. In patients with congenital long QTc syndrome, congestive heart failure, or electrolyte abnormalities, or in those who are taking medications known to prolong the QTc interval, more frequent monitoring may be necessary.

Interrupt TIBSOVO if QTc increases to greater than 480 msec and less than 500 msec. Interrupt and reduce TIBSOVO if QTc increases to greater than 500 msec. Permanently discontinue TIBSOVO in patients who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia.

Guillain-Barr Syndrome: Guillain-Barr syndrome occurred in <1% (2/258) of patients treated with TIBSOVO in the clinical study. Monitor patients taking TIBSOVO for onset of new signs or symptoms of motor and/or sensory neuropathy such as unilateral or bilateral weakness, sensory alterations, paresthesias, or difficulty breathing. Permanently discontinue TIBSOVO in patients who are diagnosed with Guillain-Barr syndrome.

ADVERSE REACTIONS

DRUG INTERACTIONS

Strong or Moderate CYP3A4 Inhibitors: Reduce TIBSOVO dose with strong CYP3A4 inhibitors. Monitor patients for increased risk of QTc interval prolongation.

Strong CYP3A4 Inducers: Avoid concomitant use with TIBSOVO.

Sensitive CYP3A4 Substrates: Avoid concomitant use with TIBSOVO.

QTc Prolonging Drugs: Avoid concomitant use with TIBSOVO. If co-administration is unavoidable, monitor patients for increased risk of QTc interval prolongation.

LACTATION

Because many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed children, advise women not to breastfeed during treatment with TIBSOVO and for at least 1 month after the last dose.

Please see full Prescribing Information, including Boxed WARNING.

About CStone

CStone Pharmaceuticals (HKEX:2616) is a biopharmaceutical company focused on developing and commercializing innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a world-class management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 15 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, five late-stage candidates are at or near pivotal trials. With an experienced team, a rich pipeline, a robust clinical development-driven business model and substantial funding, CStone's vision is to become globally recognized as a leading Chinese biopharmaceutical company by bringing innovative oncology therapies to cancer patients worldwide.

For more information about CStone Pharmaceuticals, please visit: http://www.cstonepharma.com.

Forward-looking Statement

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

View original content:http://www.prnewswire.com/news-releases/cstone-announces-first-patient-dosed-in-the-phase-i-bridging-registrational-study-of-ivosidenib-300961590.html

SOURCE CStone Pharmaceuticals

Company Codes: HongKong:2616, HongKong:02616

Read more from the original source:
CStone announces first patient dosed in the Phase I bridging registrational study of ivosidenib - BioSpace

Novocure Announces 43 Presentations on Tumor Treating Fields at 24th Annual Meeting of the Society for Neuro-Oncology – Arizona Daily Star

Presentations on Tumor Treating Fields cover a broad and growing range of topics, with nearly 80 percent of presentations prepared by external authors

ST. HELIER, Jersey--(BUSINESS WIRE)--#btsm--Novocure (NASDAQ: NVCR) today announced 43 presentations on Tumor Treating Fields, including three oral presentations, will be featured at the 24th Annual Meeting of the Society for Neuro-Oncology (SNO) on Nov. 20 through Nov. 24 in Phoenix. Presentations on Tumor Treating Fields cover a broad and growing range of topics. External authors prepared 34 of the 43 presentations.

The oral presentations on Tumor Treating Fields include an EF-14 post hoc subgroup analysis on tumor growth rates, and the pilot study results of Tumor Treating Fields combined with radiotherapy and temozolomide for the treatment of newly diagnosed glioblastoma.

Highlights among poster presentations include the combinations of Tumor Treating Fields with other therapies such as radiation and immunotherapies, simulations, health economics and outcomes research, patient advocacy, and research on the mechanism of action.

Year after year, it is amazing to see the continued focus on Tumor Treating Fields at the SNO Annual Meeting, said Novocure CEO Asaf Danziger. From our first presentation at SNO in 2008 to today, more than 250 abstracts on Tumor Treating Fields have been included at one of the most important conferences in neuro-oncology worldwide. I am proud of our team for their relentless focus on innovative research and for their consistent drive in raising awareness of our therapy among the scientific community. We look forward to another productive year at SNO.

(Abstract #: ACTR-46) Tumor Treating Fields combined with radiotherapy and temozolomide for the treatment of newly diagnosed glioblastoma: Final results from a pilot study. R. Grossman. 2:45 to 2:50 p.m. MST Nov. 22.

(Abstract #: RTHP-28) TTFields treatment affects tumor growth rates: A post-hoc analysis of the pivotal phase 3 EF-14 trial. Z. Bomzon. 4:05 to 4:10 p.m. MST Nov. 22.

(Abstract #: QOLP-24) Patients/parents experiences of receiving Optune delivered tumor treatment fields: A Pediatric Brain Tumor Consortium Study: PBTC-048. J. Lai. 7:50 to 7:54 p.m. MST Nov. 22.

(Abstract #: RDNA-10) TTFields treatment planning for targeting multiple lesions spread throughout the brain. Z. Bomzon. 7:30 to 9:30 p.m. MST Nov. 22. (Radiation Biology and DNA Repair/Basic Science)

(Abstract #: NIMG-20) Evaluation of head segmentation quality for treatment planning of tumor treating fields in brain tumors. Z. Bomzon. 7:30 to 9:30 p.m. MST Nov. 22. (Neuro-Imaging/Clinical Research)

(Abstract #: HOUT-24) Challenges and successes in the global reimbursement of a breakthrough medical technology for treatment of glioblastoma multiforme. C. Proescholdt. 7:30 to 9:30 p.m. MST Nov. 22. (Health Outcome Measures/Clinical Research)

(Abstract #: EXTH-02) The blood brain barrier (BBB) permeability is altered by Tumor Treating Fields (TTFields) in vivo. E. Schulz. 7:30 to 9:30 p.m. MST Nov. 22. (Experimental Therapeutics/Basic Science)

(Abstract #: IMMU-06) TTFields induces immunogenic cell death and STING pathway activation through cytoplasmic double-stranded DNA in glioblastoma cells. D. Chen. 7:30 to 9:30 p.m. MST Nov. 22. (Immunology/Basic Science)

(Abstract #: DRES-06) Prostaglandin E Receptor 3 mediates resistance to Tumor Treating Fields in glioblastoma cells. D. Chen. 7:30 to 9:30 p.m. MST Nov. 22. (Drug Resistance/Basic Science)

(Abstract #: EXTH-34) In vitro tumor treating fields (TTFields) applied prior to radiation enhances the response to radiation in patient-derived glioblastoma cell lines. S. Mittal. 7:30 to 9:30 p.m. MST Nov. 22. (Experimental Therapeutics/Basic Science)

(Abstract #: CSIG-20) Effect of tumor-treating felds (TTFields) on EGFR phosphorylation in GBM cell lines. M. Reinert. 7:30 to 9:30 p.m. MST Nov. 22. (Cell Signaling and Signaling Pathways/Basic Science)

(Abstract #: CBMT-14) The dielectric properties of brain tumor tissue. M. Proescholdt. 7:30 to 9:30 p.m. MST Nov. 22. (Cell Biology and Metabolism/Basic Science)

(Abstract #: CSIG-26) Is intrinsic apoptosis the signaling pathway activated by tumor-treating fields for glioblastoma. K. Carlson. 7:30 to 9:30 p.m. MST Nov. 22. (Cell Signaling and Signaling Pathways/Basic Science)

(Abstract #: ATIM-08) Trial in Progress: CA209-9Y8 phase 2 trial of tumor treating fields (TTFs), nivolumab plus/minus ipilimumab for bevacizumab-nave, recurrent glioblastoma. Y. Odia. 7:30 to 9:30 p.m. MST Nov. 22. (Adult Clinical Trials Immunologic/Clinical Research)

(Abstract #: ACTR-60) A phase 2, historically controlled study testing the efficacy of TTFields with adjuvant temozolomide in high-risk WHO grade II and III astrocytomas (FORWARD). A. Allen. 7:30 to 9:30 p.m. MST Nov. 22. (Adult Clinical Trials - Non-Immunologic/Clinical Research)

(Abstract #: TMIC-54) Comparison of cellular features at autopsy in glioblastoma patients with standard treatment of care and tumor treatment fields. A. Lowman. 7:30 to 9:30 p.m. MST Nov. 22. (Tumor Microenvironment/Basic Science)

(Abstract #: ACTR-26) Safety and efficacy of bevacizumab plus Tumor Treating Fields (TTFields) in patients with recurrent glioblastoma (GBM): data from a phase II clinical trial. J. Fallah. 7:30 to 9:30 p.m. MST Nov. 22. (Adult Clinical Trials Non-immunologic/Clinical Research)

(Abstract #: RBTT-02) Radiosurgery followed by Tumor Treating Fields for brain metastases (1-10) from NSCLC in the phase 3 METIS trial. V. Gondi. 7:30 to 9:30 p.m. MST Nov. 22. (Randomized Brain Tumor Trials in Development/Clinical Research)

(Abstract #: INNV-16) Complete response of thalamic IDH wildtype glioblastoma after proton therapy followed by chemotherapy together with Tumor Treating Fields. M. Stein. 7:30 to 9:30 p.m. MST Nov. 22. (Innovations in Patient Care/Clinical Research)

(Abstract #: INNV-20) A systematic review of tumor treating fields therapy for primary for recurrent and glioblastoma. P. Shah. 7:30 to 9:30 p.m. MST Nov. 22. (Innovations in Patient Care/Clinical Research)

(Abstract #: STEM-16) Dual Inhibition of Protein Arginine Methyltransferase 5 and Protein Phosphatase 2a Enhances the Anti-tumor Efficacy in Primary Glioblastoma Neurospheres. H. Sur. 7:30 to 9:30 p.m. MST Nov. 22. (Stem Cells/Basic Science)

(Abstract #: CBMT-13) 3DEP system to test the electrical properties of different cell lines as predictive markers of optimal tumor treating fields (TTFields) frequency and sensitivity. M. Giladi. 5 to 7 p.m. MST Nov. 23. (Cell Biology and Metabolism/Basic Science)

(Abstract #: EXTH-37) A novel transducer array layout for delivering Tumor Treating Fields to the spine. Z. Bomzon. 5 to 7 p.m. MST Nov. 23. (Experimental Therapeutics/Basic Science)

(Abstract #: NIMG-41) Rapid and accurate creation of patient-specific computational models for GBM patients receiving Optune therapy with conventional imaging (T1w/PD). Z. Bomzon. 5 to 7 p.m. MST Nov. 23. (Neuro-Imaging/Clinical Research)

(Abstract #: HOUT-17) Utilities of rare cancers like malignant pleural mesothelioma and glioblastoma multiforme - do they compare? C. Proescholdt. 5 to 7 p.m. MST Nov. 23. (Health Outcome Measures/Clinical Research)

(Abstract #: INNV-17) Innovative educational approaches to enhance patient and caregiver understanding of Optune for glioblastoma. M. Shackelford. 5 to 7 p.m. MST Nov. 23. (Innovations in Patient Care/Clinical Research)

(Abstract #: EXTH-05) Therapeutic implications of TTFields induced DNA damage and replication stress in novel combinations for cancer treatment. N. Karanam. 5 to 7 p.m. MST Nov. 23. (Experimental Therapeutics/Basic Science)

(Abstract #: EXTH-31) Combination of tumor treating fields (TTFields) and paclitaxel produces additive reductions in proliferation and clonogenicity in patient-derived metastatic non-small cell lung cancer (NSCLC) cells. S. Michelhaugh. 5 to 7 p.m. MST Nov. 23 (Experimental Therapeutics/Basic Science)

(Abstract #: EXTH-53) Tumor Treating Fields leads to changes in membrane permeability and increased penetration by anti-glioma drugs. E. Chang. 5 to 7 p.m. MST Nov. 23. (Experimental Therapeutics/Basic Science)

(Abstract #: RDNA-01) Tubulin and microtubules as molecular targets for TTField therapy. J. Tuszynski. 5 to 7 p.m. MST Nov. 23. (Radiation Biology and DNA Repair/Basic Science)

(Abstract #: SURG-01) OptimalTTF-1: Final results of a phase 1 study: First glioblastoma recurrence examining targeted skull remodeling surgery to enhance Tumor Treating Fields strength. A. Korshoej. 5 to 7 p.m. MST Nov. 23. (Surgical Therapy/Clinical Research)

(Abstract #: ATIM-39) Phase 2 open-labeled study of adjuvant temozolomide plus Tumor Treating Fields plus Pembrolizumab in patients with newly diagnosed glioblastoma (2-THE-TOP). D. Tran. 5 to 7 p.m. MST Nov. 23. (Adult Clinical Trials Immunologic/Clinical Research)

(Abstract #: ACTR-49) Initial experience with scalp preservation and radiation plus concurrent alternating electric tumor-treating fields (SPARE) for glioblastoma patients. A. Song. 5 to 7 p.m. MST Nov. 23. (Adult Clinical Trials - Non-Immunologic/Clinical Research)

(Abstract #: RTHP-25) TTFields dose distribution alters tumor growth patterns: An imaging-based analysis of the randomized phase 3 EF-14 trial. M. Ballo. 5 to 7 p.m. MST Nov. 23. (Radiation Therapy/Clinical Research)

(Abstract #: ACTR-19) Report on the combination of Axitinib and Tumor Treating Fields (TTFields) in three patients with recurrent glioblastoma. E. Schulz. 5 to 7 p.m. MST Nov. 23. (Adult Clinical Trials - Non-Immunologic/Clinical Research)

(Abstract #: PATH-47) TTF may apply selective pressure to glioblastoma clones with aneuploidy: a case report. M. Ruff. 5 to 7 p.m. MST Nov. 23. (Molecular Pathology and Classification Adult and Pediatric/Clinical Research)

(Abstract #: RARE-39) Combination of Tumor Treating Fields (TTFields) with lomustine (CCNU) and temozolomide (TMZ) in newly diagnosed glioblastoma (GBM) patients - a bi-centric analysis. L. Lazaridis. 5 to 7 p.m. MST Nov. 23. (Rare Tumors/Clinical Research)

(Abstract #: ACTR-31) The use of TTFields for newly diagnosed GBM patients in Germany in routine clinical care (TIGER: TTFields in Germany in routine clinical care). O. Bahr. 5 to 7 p.m. MST Nov. 23. (Adult Clinical Trials Non-Immunologic/Clinical Research)

(Abstract #: INNV-09) Clinical efficacy of tumor treating fields for newly diagnosed glioblastoma. Y. Liu. 5 to 7 p.m. MST Nov. 23. (Innovations in Patient Care/Clinical Research)

(Abstract #: EXTH-61) Celecoxib Improves Outcome of Patients Treated with Tumor Treating Fields. K. Swanson. 5 to 7 p.m. MST Nov. 23. (Experimental Therapeutics/Basic Science)

(Abstract #: INNV-23) Glioblastoma and Facebook: An Analysis Of Perceived Etiologies and Treatments. N. Reddy. 5 to 7 p.m. MST Nov. 23. (Innovations in Patient Care/Clinical Research)

(Abstract #: INNV-12) Outcomes in a Real-world Practice For Patients With Primary Glioblastoma: Impact of a Specialized Neuro-oncology Cancer Care Program. N. Banerji. 5 to 7 p.m. MST Nov. 23. (Innovations in Patient Care/Clinical Research)

(Abstract #: RBTT-11): NRG Oncology NRG-BN006: A Phase II/III Randomized, Open-label Study of Toca 511 and Toca FC With Standard of Care Compared to Standard of Care in Patients With Newly Diagnosed Glioblastoma. M. Ahluwalia. 5 to 7 p.m. MST Nov. 23. (Randomized Brain Tumor Trials Development/Clinical Research)

Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer through the development and commercialization of its innovative therapy, Tumor Treating Fields. Tumor Treating Fields is a cancer therapy that uses electric fields tuned to specific frequencies to disrupt solid tumor cancer cell division. Novocures commercialized products are approved for the treatment of adult patients with glioblastoma and malignant pleural mesothelioma. Novocure has ongoing or completed clinical trials investigating Tumor Treating Fields in brain metastases, non-small cell lung cancer, pancreatic cancer, ovarian cancer and liver cancer.

Headquartered in Jersey, Novocure has U.S. operations in Portsmouth, New Hampshire, Malvern, Pennsylvania and New York City. Additionally, the company has offices in Germany, Switzerland, Japan and Israel. For additional information about the company, please visit http://www.novocure.com or follow us at http://www.twitter.com/novocure.

Optune is intended as a treatment for adult patients (22 years of age or older) with histologically-confirmed glioblastoma multiforme (GBM).

Optune with temozolomide is indicated for the treatment of adult patients with newly diagnosed, supratentorial glioblastoma following maximal debulking surgery, and completion of radiation therapy together with concomitant standard of care chemotherapy.

For the treatment of recurrent GBM, Optune is indicated following histologically- or radiologically-confirmed recurrence in the supratentorial region of the brain after receiving chemotherapy. The device is intended to be used as a monotherapy, and is intended as an alternative to standard medical therapy for GBM after surgical and radiation options have been exhausted.

The NovoTTF-100L System is indicated for the treatment of adult patients with unresectable, locally advanced or metastatic, malignant mesothelioma (MPM) to be used concurrently with pemetrexed and platinum-based chemotherapy.

Important Safety Information

Do not use Optune in patients with GBM with an implanted medical device, a skull defect (such as, missing bone with no replacement), or bullet fragments. Use of Optune together with skull defects or bullet fragments has not been tested and may possibly lead to tissue damage or render Optune ineffective. Do not use the NovoTTF-100L System in patients with MPM with implantable electronic medical devices such as pacemakers or implantable automatic defibrillators, etc.

Use of Optune for GBM or the NovoTTF-100L System for MPM together with implanted electronic devices has not been tested and may lead to malfunctioning of the implanted device.

Do not use Optune for GBM or the NovoTTF-100L System for MPM in patients known to be sensitive to conductive hydrogels. Skin contact with the gel used with Optune and the NovoTTF-100L System may commonly cause increased redness and itching, and may rarely lead to severe allergic reactions such as shock and respiratory failure.

Optune and the NovoTTF-100L System can only be prescribed by a healthcare provider that has completed the required certification training provided by Novocure.

The most common (10%) adverse events involving Optune in combination with chemotherapy in patients with GBM were thrombocytopenia, nausea, constipation, vomiting, fatigue, convulsions, and depression.

The most common (10%) adverse events related to Optune treatment alone in patients with GBM were medical device site reaction and headache. Other less common adverse reactions were malaise, muscle twitching, and falls related to carrying the device.

The most common (10%) adverse events involving the NovoTTF-100L System in combination with chemotherapy in patients with MPM were anemia, constipation, nausea, asthenia, chest pain, fatigue, device skin reaction, pruritus, and cough.

Other potential adverse effects associated with the use of the NovoTTF-100L System include: treatment related skin toxicity, allergic reaction to the plaster or to the gel, electrode overheating leading to pain and/or local skin burns, infections at sites of electrode contact with the skin, local warmth and tingling sensation beneath the electrodes, muscle twitching, medical site reaction and skin breakdown/skin ulcer.

If the patient has an underlying serious skin condition on the treated area, evaluate whether this may prevent or temporarily interfere with Optune and the NovoTTF-100L System treatment.

Do not prescribe Optune or the NovoTTF-100L System for patients that are pregnant, you think might be pregnant or are trying to get pregnant, as the safety and effectiveness of Optune and the NovoTTF-100L System in these populations have not been established.

Forward-Looking Statements

In addition to historical facts or statements of current condition, this press release may contain forward-looking statements. Forward-looking statements provide Novocures current expectations or forecasts of future events. These may include statements regarding anticipated scientific progress on its research programs, clinical trial progress, development of potential products, interpretation of clinical results, prospects for regulatory approval, manufacturing development and capabilities, market prospects for its products, coverage, collections from third-party payers and other statements regarding matters that are not historical facts. You may identify some of these forward-looking statements by the use of words in the statements such as anticipate, estimate, expect, project, intend, plan, believe or other words and terms of similar meaning. Novocures performance and financial results could differ materially from those reflected in these forward-looking statements due to general financial, economic, regulatory and political conditions as well as more specific risks and uncertainties facing Novocure such as those set forth in its Quarterly Report on Form 10-Q filed on July 25, 2019, with the U.S. Securities and Exchange Commission. Given these risks and uncertainties, any or all of these forward-looking statements may prove to be incorrect. Therefore, you should not rely on any such factors or forward-looking statements. Furthermore, Novocure does not intend to update publicly any forward-looking statement, except as required by law. Any forward-looking statements herein speak only as of the date hereof. The Private Securities Litigation Reform Act of 1995 permits this discussion.

Media and Investor Contact:

More video from this section

See original here:
Novocure Announces 43 Presentations on Tumor Treating Fields at 24th Annual Meeting of the Society for Neuro-Oncology - Arizona Daily Star

Maltese abroad: the best-selling author with roots in Gozo – Times of Malta

Marianne Curley is a Maltese-Australian author best known for her Guardians of Time Trilogy and Old Magic books. She has had to overcome tremendous difficulties that are of inspiration to many.

My Dad was born in Gozo and moved to Australia when he was only 15. He eventually settled on the Hawkesbury River in New South Wales. He returned to Gozo when the war ended and met my mother. They married within three months and moved to Australia.

Mum became homesick and they moved back. But Dad was a farmer and longed for the open spaces and green fields of Australia. In August of 1957, as a family of five, they returned to the Hawkesbury River property. Two years later I was born.

My teen daughters asked me to write a book for their age. I had attempted to write commercial romance, with no success. I was about to start another romance when my thirteen-year-old daughter complained there were not enough books for her age to read. Her sister, older by one year, agreed and that is how I started writing books for young adults.

I found I loved writing for young adults. It was challenging and exciting with more complex plot lines and themes I could explore, and young adult readers appeared to enjoy my writing style.

I find inspiration almost anywhere if Im looking for it. I listen to music, look through photographs or glance at passing scenery in a car or train. Our world is full of ideas. They surround us wherever we go.

The plot for Old Magic, my first published novel, came to me while I was having lunch in a park with friends on Dorrigo Mountain. Our children were walking across a field together when a mist rolled in from behind them. I watched as the mist caught up and continued to roll past, making the children almost invisible. It struck me how magical the scene was.

My readers are my proudest achievements, through their emails and messages, that have given many reasons to be most proud of them. My readers have let me know how my books, the characters and stories within, have affected them, inspired them, assisted them in tough times, and changed their lives.

Surviving my type of cancer was a miracle that I thank God for every day. The odds were not in my favour, I had a 30-35 per cent chance of surviving, and without a successful transplant I would have died within two months of my diagnosis. I am thankful for my sister Therese for giving me her stem cells and for the prayers and support I received from my friends and relatives.

I could not write for years. Weeks after surviving my transplant, and on the way to recovering, I sustained a fall in my hospital room, that broke my back. My bones were week from the chemotherapy, my spine crumbled, and I lost several vertebrae. My spine wasnt strong enough for any supportive treatment. The pain when I moved was unimaginable. Because of this, I was unable to write again for a few years.

Only focus on today. Whatever you are facing, life can become complex and at times overwhelming. You think you cant cope. Dont think of all you have yet to do, just think about what you have to do today. Only today. Tomorrow will come, theres no doubt about that. Think of what you must do tomorrow, when it is tomorrow.

I dream of visiting Malta. I had planned to visit Malta as a reward after my cancer battle, but by then Id had the fall which made travelling difficult. So sadly, Ive never had the chance to visit Malta, which just makes me miss it more. My hope is that one day I can overcome my physical difficulties enough for a long visit.

Are you a Maltese person living abroad? Contact malteseabroad@timesofmalta.com

Continued here:
Maltese abroad: the best-selling author with roots in Gozo - Times of Malta