CARLSBAD, Calif.--(BUSINESS WIRE)--Accelerated Biosciences, a regenerative medicine innovator, announced today new data that further demonstrates statistically significant cytolysis with induced pluripotent stem cell (iPSC)-derived natural killer (NK) cells programmed from its ethically sourced human trophoblast stem cells (hTSCs). Pluristyx, a Seattle-based firm supporting drug development, regenerative medicine, and cell and gene therapies, further confirmed Accelerated Biosciences hTSC line offers before-unrealized opportunities in cell-specific therapeutics. Along with this recent data on successful iPSC differentiation, Accelerated Biosciences has already demonstrated efficient differentiation of its pluripotent stem cells with remarkable doubling times and growth characteristics to programmed NK, cartilage, bone, fat, neuron, pancreas, liver, and secretome cells.

This new data validates our findings, explains Yuta Lee, President and Founder of Accelerated Biosciences. We know the properties of our trophoblast stem cells have been long-sought by the medical science community because of the potential to speed and amplify the development of life-saving therapeutics; theyre immune privileged, chromosomally stable (not tumorigenic), pathogen free, pluripotent, easy to scale and manufacturer, and of special interest, they are ethically sourced from the chorionic villi (pre-placental tissue) of non-viable and often life-threatening tubal ectopic pregnancies. Mr. Lees father, Professor Jau-Nan Lee, MB, MD, PhD, an obstetrics and gynecologic physician and researcher in Taiwan, first isolated hTSC in 2003. Mr. Lee created Accelerated Biosciences to elevate the visibility of this pluripotent human trophoblast stem cell platform to those engaged in developing allogeneic cell therapeutics and has been instrumental in the filing and prosecution of intellectual property to protect the companys hTSC platform to date holding 34 patents.

Benjamin Fryer, PhD, Co-founder and CEO of Pluristyx, worked closely with Accelerated Biosciences to prepare much of its key hTSC data. Dr. Fryer, a trophoblast expert who was previously a research scientist at Janssen Research & Development of Johnson & Johnson, now serves on Accelerated Biosciences Scientific Advisory Board. Initially I was skeptical these cells were what they said they were. If we hadnt grown and characterized them in our lab, I might have remained skeptical. These are indeed trophoblast stem cells, explained Dr. Fryer. The potential of these cells is enormous. One of the industrys largest challenges is that its almost impossible to scale primary cells. These cells are scalable. With these cells you can make the amount required for millions of patients and theyre sourced compliant to regulatory requirements. Weve made IPS cells (induced pluripotent stem cells) and NK (natural killer) cells from them, which is the next wave of cells for cell therapies. For therapeutic developers, because these cells are not sourced from a person or viable embryo, these cells deliver the trifecta of legal, ethical, and IP advantages.

As the biotechnology industry works toward developing therapies that target only diseased cells without harming healthy cells and tissues, cell-based therapies draw increasing interest, explains industry expert, Martina Molsbergen, CEO of C14 Consulting, who has partnered with Accelerated Biosciences in a business development role. With all the promise that cell therapies hold, the biotechnology industry also remains concerned that the therapeutics are derived in a socially and ethically responsible manner. Accelerated Biosciences has discovered and is now offering what scientists see as the holy grail of stem cell sources.

Prominent biosciences experts have been drawn to Accelerated Biosciences cell breakthrough. Protein chemist and molecular biologist Igor Fisch, PhD, former President and CEO of Selexis, Geneva, Switzerland, recognizes the impact that Accelerated Biosciences hTSCs will have on human health: Not only are these cells politically correct, but they can also differentiate. Because they are sourced from pre-placenta material, theyre immune privileged, which means that are not seen as foreign by the human body. With these cells, we can create a cell bank a single source for a wide range of patients.

Peter Hudson, FTSE, BSc Hons, PhD, Chief Scientist and a senior advisor to Avipep P/L in Melbourne, Australia, and an adjunct professor at the University of Queensland, led a large oncology consortium to complete the first Phase 1 clinical trial of a novel engineered antibody targeting prostate and ovarian cancer. Hudsons interest in Accelerated Biosciences hTSCs has evolved into a role on its Scientific Advisory Board. Trophoblast stem cells are likely to be the next wave of cancer-targeting therapeutics, explains Dr. Hudson. The ability to ethically source trophoblast stem cells and program them to target only diseased, cancerous cells is very powerful technology.

Why are scientists so interested in stem cell-based therapies?

The human body constantly produces specialized cells from its own stem cells (undifferentiated cells) to renew and repair itself. Current therapies harness this power in autologous cell therapies in which the patients own cells are removed, differentiated into disease-fighting cells, and reinserted.

What makes the human trophoblast stem cell so important to medical science?

The human trophoblast stem cell (hTSC) comes from placental tissue and has special properties that make it extremely desirable to therapeutic developers. The hTSC is such an early stem cell that it has much more capacity for growth than a stem cell taken from an adult, for example. This means that one cell can become millions. The hTSC also carries with it the same immune-privilege that a growing embryo has inside its mother: its not seen as foreign although its genetically different than its mother. Unlike other foreign materials, the hTSC is not rejected by the human body, which means that it can be used with many different patients (allogeneic cell therapy). With these benefits, the scientific community holds a high regard for hTSCs, but it also faces socio-ethical concerns about how those stem cells are typically sourced.

Accelerated Biosciences sidesteps hTSC sourcing concerns in a profoundly elegant way. Dr. Jau-Nan Lee, an OB-GYN in Taiwan, found inspiration in what was considered medical waste. When surgical intervention was necessary to remove an ectopic pregnancy that would otherwise risk the womans life, the non-viable embryo and pre-placental tissue lodged in the fallopian tube was removed, sent to pathology, and discarded. Gaining permission from institutional colleagues and sampling the pre-placental tissue, Dr. Lee isolated hTSC that offered all the benefits of hTSC pluripotency, immune privilege, and scalability without pathogens and without ethical compromises.

About Accelerated Biosciences

Founded in 2013, Accelerated Biosciences is a private company focused on regenerative medicine and built around the hTSC discoveries of obstetrics and gynecology physician and researcher, Professor Jau-Nan Lee, MB, MD, PhD. Accelerated Biosciences holds a large and robust patent portfolio and an encumbrance-free intellectual property (IP) estate. Accelerated Biosciences mission is to leverage its renewable, immune-privileged human cell source to fuel breakthrough cell therapies that effectively target the most challenging diseases of the human body. For more information about Accelerated Biosciences, visit acceleratedbio.com or email mmolsbergen@c14consultinggroup.com.

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Accelerated Biosciences' Immune-Privileged Human Trophoblast Stem Cells (hTSCs) Offer Breakthrough Opportunities in Cancer-Targeting Therapeutics and...

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Small dreams have no power to move hearts, and in a new six-year strategic plan, St. Jude Childrens Research Hospital is thinking very big.

What would it take to drastically increase cure rates for childhood cancer worldwide?

St. Judes answer: $11.5 billion and an additional 1,400 jobs.

To get a rough sense of scale, work it out with a pencil:

Spread over six yearsat $1.916 billion each yearits just under a third of the NCIs annual spend, fourfold this years projected revenues of the American Cancer Society, and more than seventyfold the budget of the World Health Organizations International Agency for Research on Cancer.

Its a broad and ambitious plan that will allow the institution to grow at an almost 8% compound annual growth rate, James Downing, president and CEO of St. Jude, said to The Cancer Letter.

At a global level, we also want to see identifiable increases in cure rates. We are watching those very carefully. Our goal is to move toward cure rates of 60% for diseases like acute lymphoblastic leukemia, Hodgkins lymphoma and Wilms tumor, Downing said. As we look at a global population, survival rates hover around 20%, and wed like to see those go up year by year.

A lot of our efforts are based on implementation science, looking at what works and what doesnt work. Workforce, drug distribution and true advancements in cure rates are what were seeking over the next six years.

The plan, rolled out on April 27, calls for an acceleration of research and treatment globallynot just for pediatric cancer, but also other illnesses, including blood disorders, neurological diseases, and infectious diseases.

Not surprisingly, this amount represents the largest investment the Memphis, Tenn. hospital has made in its nearly 60-year history. The previous strategic plan, the largest expansion in the institutions history, resulted in $7 billion in investments (The Cancer Letter, May 19, 2017).

The multi-phase expansion plan is funded almost entirely by steadily increasing donor contributions generated by ALSAC, the fundraising and awareness organization for St. Jude.

It is an ambitious plan. But were going to have lots of new personnel, new investments, new technology and new partnerships. We have formal partnerships with many U.N. associate agencies and organizations around the world.

Within the past six years, St. Jude has advanced fundamental, clinical, and translational research, Downing said.

Two years ago, we began strategically looking at the most pressing issues in the field of pediatric cancer, Downing said. As we developed the strategic plan over those two years, there were many ideas we critically assessed, and we often said, Its not really best for St. Jude to pursue that.

In the end, we aligned on goals that collectively bring the prospect of remarkable benefits to the field of childhood cancer, and to children with cancer everywhere.

On campus, St. Jude accepted nearly 20% more new cancer patients; increased faculty by 30% and staff by 23%; and embarked on several large-scale construction projects.

The new strategic plan focuses on five areas: fundamental science, childhood cancer, pediatric catastrophic diseases, global impact, and workforce and workplace culture.

Were coming out of a six-year strategic plan in which we increased our number of cancer patients by 20%, with 30% new faculty, 23% more staff, many large-scale construction projects, said Charles Roberts, executive vice president of St. Jude and director of the hospitals Comprehensive Cancer Center. And were now going into a new strategic plan that is 60% larger than our prior plan.

Under the plan, St. Jude will hire nearly 70 new faculty members, plus supporting laboratory staff, to work in basic, translational, and clinical research across 22 departments.

These investigators will have the freedom to pursue the type of conceptually driven research that leads to tomorrows clinical advances.

As we launch a strategic plan, weve identified the most exciting opportunities and challenges at that point in time, Roberts said to The Cancer Letter. However, we fully realize that we dont know whats coming next. New discoveries will be made, and new opportunities will emerge. Via the blue-sky process, weve set aside substantial funds every year to invest in the pursuit of emerging opportunities suggested by faculty and staff.

Part of what brought me here from Boston was the last strategic plan, and its so exciting to be a part of this. But just looking at the numbers, 1,400 new positions, average salary of $90,000. Six hundred and forty of those positions are in research, 266 are in clinical, 100 are in global pediatric medicine, and 394 in support.

Those are the kinds of numbers that you need to make these things real, and I think its exciting for St. Jude and for the field of cancer research, as we bring in all of these new folks.

During the next six years, St. Jude will invest more than $250 million to expand technology and resources available to scientists and clinicians in their search to understand why pediatric catastrophic diseases arise, spread and resist treatments. These investments will include:

St. Jude will invest $3.7 billion during the next six years to expand cancer-focused research and related clinical care. These efforts will center on raising survival rates for the highest-risk cancers and for children with relapsed diseases, while simultaneously improving quality of life for pediatric cancer survivors. The investments will include:

In the U.S., more than 80% of children diagnosed with cancer will be cured. In contrast, 80% of children with cancer live in limited-resource countries, where a mere 20% survive their disease. To address this, St. Jude will more than triple its investment in its international efforts coordinated through St. Jude Global and the St. Jude Global Alliance during the next six years.

This represents an investment of more than $470 million. Global initiatives include:

Under the plan, St. Jude will expand research and treatment programs to advance cures for childhood catastrophic diseases. The $1.1 billion, six-year investment includes work in nonmalignant hematological diseases, such as sickle cell disease; a new laboratory-based research program in infectious diseases that affect children worldwide; and a new research and clinical program to better understand and treat pediatric neurological diseases.

The plan outlines several strategies to encourage teamwork, and internal and external collaboration. These will include:

It is estimated that 87% of funds to sustain and grow St. Jude over the next six years will come from public donations.

Patients at St. Jude do not receive a bill for treatment, travel, housing or fooda model established by ALSAC and St. Jude founder Danny Thomas, who believed in equal access to medical care and driving research advances.

There are an incredible number of donors across the United States who support St. Jude, Downing said. This carries a great responsibility for us to seek the maximum possible impact to improve outcomes for childhood cancer.

Downing and Roberts spoke with Matthew Ong, associate editor of The Cancer Letter.

Matthew Ong: Congratulations on the official launch of St. Judes second six-year strategic plan. Could you briefly walk us through whats in it?

James Downing: It is an exciting time for St. Jude Childrens Research Hospital. Were finishing our prior six-year strategic plan, which started in Fiscal Year 2016. That $7 billion investment in the organization spanned fundamental science, clinical and translational research, clinical operations, and our global efforts. During the course of the plan, we increased faculty by 30% and staff by 23% and accelerated progress against pediatric catastrophic diseases.

About two years ago, we started discussing the next strategic plan. We looked critically at what we had accomplished under the previous plan, the expertise we had assembled, and the major problems in the field of pediatric catastrophic diseases, including cancer, infectious diseases, nonmalignant hematologic diseases and pediatric neurologic diseases. During that period, we involved more than 200 individuals across the institution to develop the new strategic plan.

This plan, at its core, focuses on accelerating progress against pediatric catastrophic diseases on a global scale. It outlines a $11.5 billion investment during the next six years, which includes the addition of 1,400 jobs and $1.9 billion in new capital investments, construction and renovations. Its a broad and ambitious plan that will allow the institution to grow at an almost 8% compound annual growth rate.

The plan has 11 goals, divided among five major areas: fundamental science, pediatric cancer, other childhood catastrophic diseases, global impact, and a focus on people and place. In each of these areas, were recruiting new individuals, investing in new technology, and expanding collaborations across campus, across the United States, and globally.

Ill start with fundamental science. In our last strategic plan, we invested heavily in increasing basic science programs on campus by expanding faculty numbers, technology and institutional data infrastructurein the belief that expanding fundamental science leads to new knowledge that helps advance cures. This is investigator-initiated science, often not related to diseases, but rather to the fundamental questions of biology.

In this new plan, were again investing heavily in expanding fundamental science at St. Jude. Weve committed more than $1 billion to fundamental science. This includes increasing laboratory-based faculty by more than 33% during the next six years, and more than $250 million dedicated to investments in technology.

Our goal is to make sure every dollar is spent wisely and effectively in pursuit of our missionto advance cures and means of prevention for pediatric catastrophic diseases through research and treatment.

The $250 million will fund multiple shared resources, department-based technology centers and new centers of excellence. Some of the faculty are being recruited to the centers of excellence, including those in data-driven discovery, innate immunity and inflammation, leukemia and advanced microscopy. These individuals will also have homes in academic departments.

On the technology front, were investing in shared resources. Well bring online some new ones, as well as some (Center for Modeling Pediatric Diseases and the Center for High-Content Screening) created at the end of the last strategic plan. The newest is focused on spatial transcriptomics. It will allow scientists across campus to look at the expression of genes in tissue context and at the single-cell level.

A new effort in structural biology is to create a $20 million Cryo-Electron Tomography Center. This is the next level of cryo-electron microscopy, which allows the identification of the structure of molecules or molecular machines within the context of cells. Its a developing technology that will feed other investments weve made in structural biology, such as the installation of one of the largest magnets in the world in our NMR facility, our Cryo-Electron Microscopy Center and single-cell analysis capabilities. The plan brings those tools to bear on defining normal biology and disease states.

Another effort is a Center of Excellence in Advanced Microscopy. Over the last six years, weve become one of the leading centers in the application of advanced microscopy to fundamental biology. This has been led by investigators in our Developmental Neurobiology, Cell and Molecular Biology, and Immunology departments.

Were positioned to build the next generation of microscopes to explore biology in ways never dreamed. With new faculty recruitments and collaborations with commercial companies and other institutions, we seek to develop the next generation of microscopes, and apply that to normal biology and to pediatric catastrophic diseases.

Another area were investing in heavily is data science. Over the last six years, and even before that, we expanded data sciences across campus. This initially started with the Pediatric Cancer Genome Project in 2010. Since then, we recruited many data scientists, and coalesced them into our Computational Biology, Biostatistics, and Epidemiology and Cancer Control departments, and into shared resources that provide bioinformatics support.

But over the last several years, weve seen the explosion of data, from structural biology to microscopy.

As we look to the future and the capabilities weve amassed, were poised to significantly increase our investment in data science and become a leading institution in the application of data science to biologic discovery. This is a $40 million investment with 30 full-time employees.

We have a task force led by faculty members to develop the roadmap for how were going to move forward. As data is accumulated and we look across those disparate data types, we can gain knowledge through true data scienceexploring that data and advancing our understanding of biology.

The last area in fundamental research is our graduate school. During the last strategic plan, we developed the St. Jude Graduate School of Biomedical Sciences, which offers a PhD and two masters programs.

Were going to expand that over the coming six years by increasing the number of students in the Biomedical Sciences PhD, the Master of Science in Global Child Health and the Master of Science in Clinical Investigations programs. We will also create a new masters program in data science. That will bring a new population of students to campus, which will further enhance our scientific enterprise.

Pediatric cancer is our next area of focus. This has always been our institutions major focus. This area encompasses $3.7 billion of the operating dollars we will spend over the next six years. Although weve invested heavily in this effort in the past, were going to expand it significantly.

Were going to focus on pediatric cancers where the least progress has been madecancers that are incurable and relapsed diseaseand gain insights into how we can change the outlook for those cancers.

The first area of investment is new faculty10 laboratory-based individuals who will expand our research efforts in understanding the biology of cancer. Some of those faculty will go into the Center of Excellence in Leukemia, but others will focus on solid tumors, brain tumors, or on biologic aspects that cut across cancer types.

Our second area for expansion will focus on assessing new therapeutic approaches for the highest-risk cancers. We need to access and evaluate more new therapies in a rigorous manner to identify those which should be moved forward into frontline clinical trials. Pediatric cancer encompasses many different types of cancer.

To run clinical trials, you need a sufficient number of patients to be able to answer questions in a reasonable time frame. We need a way to identify the best new agents to move into clinical trials.

Our investment in preclinical testing will help us set up that infrastructure. Much of it was established in the last strategic plan, but it must be expanded so that we have the best pediatric cancer models and can assess single and combination therapies to see which are worth moving forward into clinical trials.

On the clinical trial front, we want to expand our infrastructure to run those clinical trialsnot only on our campus, but in collaborations across the United States and around the globe. To make progress in some of these high-risk pediatric cancers, we need many patients. For many of the high-risk cancers, there are not a sufficient number of patients in the United States to conduct the trials. We, therefore, need to set up global collaborative networks that can address these high-risk cancers.

So, were investing in our ability to access drugs through commercial sources, to rigorously assess these in preclinical models and to establish the global infrastructure to run these clinical trials with an associated translational science infrastructure second to none.

Our third emphasis under the cancer focus is cancer immunotherapy. We began our work in cancer immunotherapy decades ago. We developed the chimeric antigen receptor, or CAR, against CD-19. That is the basis for the FDA-approved therapy that is being used right now on a variety of different fronts. Over the last several years, weve also invested heavily in expanding our cancer immunotherapy efforts, primarily focused on CAR-modified T cells.

As part of this new strategic plan, we are creating a new program, the Translational Immunology and Immunotherapy Initiative. It will facilitate cross-departmental efforts focused on cancer immunotherapy and will explore the fundamental biology of chimeric antigen receptor approaches to cancer immunotherapy.

What makes an ideal antigen that can be attacked by a chimeric antigen receptor? How does one manipulate CAR T cells so that they undergo exhaustion and stop killing the tumor? How do we change that? And what are the features of the microenvironment that decrease the killing potency of CAR T cells? These will require significant investments, including additional faculty.

Another emphasis will be looking at long-term toxicities of the therapies we use to treat children with cancer. As we cure more and more pediatric cancers, we must continually look at the toxicities from therapy and figure out how to reduce those without sacrificing the ability to be cured. Part of that is precision medicine, and so we are continuing to invest in our genomic and pharmacogenomic efforts and our proton therapy center.

Part of reducing toxicities comes from learning from long-term survivors. So, we will continue to invest in St. Jude LIFE, our long-term, follow-up study. We will expand that to some of the newer pediatric cancer therapies, including immunotherapy and targeted agents. We will assess long-term complications from these therapeutic approaches and try to define which patients will be susceptible to these toxicities.

MO: I have to mention the obvious: $11.5 billion is quite the budget. Your new strategic plan is work that, one could argue, might be on par or exceeds the coordination and budget required to realize multiple projects, say, at the World Health Organization or even at some U.S. federal agencies. What did it take for you and your team at St. Jude to get to this point?

JD: There are an incredible number of donors across the United States who support St. Jude. Our goal is to make sure every dollar is spent wisely and effectively in pursuit of our missionto advance cures and means of prevention for pediatric catastrophic diseases through research and treatment. This carries a great responsibility for us to seek the maximum possible impact to improve outcomes for childhood cancer.

We have the ideal team at St. Jude to execute this. Our leadership meets multiple times each week. Two years ago, we began strategically looking at the most pressing issues in the field of pediatric cancer. We discussed which areas represented the greatest opportunities for St. Jude to contribute. We talked to many experts inside and outside of the institutionaround the globeand made hard decisions as we went forward.

Strategic planning is deeply engrained at St. Jude as a rigorous process that is part of our scientific culture. We knew it was going to take two years to develop this plan. We dont hire consultants; we do it all ourselves. Faculty across the institution participated in the development of priorities and goals for this strategic plan via structured meetings.

As we developed the strategic plan over those two years, there were many ideas we critically assessed, and we often said, Its not really best for St. Jude to pursue that. In the end, we aligned on goals that collectively bring the prospect of remarkable benefits to the field of childhood cancer, and to children with cancer everywhere.

Every child who comes on this campus is part of our mission. We provide them with the best care possible. We do that in the context of research studies, so that were learning from every single patient. That means were not only helping children today; were also advancing cures for children tomorrow.

Weve rolled out the new strategic plan across campus during the last month, and the excitement is palpable. Our commitment continues long after the strategic plans launch.

We have routine strategic planning retreats, where we assess the goals for the year, evaluate progress against the prior years goals, perform talent assessments and proactively seek out emerging opportunities. Every employee on campus will develop yearly goals that cascade down from the goals of this plan.

As we develop this roadmap, we know there are going to be new ideas. Charlie can tell you about a process incorporated into the strategic plan that allows us to not only move forward on this roadmap, but also add initiatives as new ideas emerge.

Charles Roberts: Its a process we began with the last strategic plan, called our blue-sky process. As we launch a strategic plan, weve identified the most exciting opportunities and challenges at that point in time.

However, we fully realize that we dont know whats coming next. New discoveries will be made, and new opportunities will emerge. Via the blue-sky process, weve set aside substantial funds every year to invest in the pursuit of emerging opportunities suggested by faculty and staff.

Ideas that have emerged from the blue-sky process have been phenomenal. Our engagement with World Health Organization (WHO)a collaboration to raise childhood cancer survival rates internationallyis one example.

The Center for Modeling Pediatric Diseases is another example. This center makes iPS cells that come from patients so that we can investigate mechanisms that underlie cancer predisposition.

In another blue-sky project, were looking at DNA methylation to characterize pediatric solid tumors with the goal of identifying new therapeutic opportunities. Some of our immunotherapy initiatives also came out of the blue-sky process. Were looking forward to growing our blue-sky endeavors as we go forward.

Were coming out of a six-year strategic plan in which we increased our number of cancer patients by 20%, with 30% new faculty, 23% more staff, many large-scale construction projects. And were now going into a new strategic plan that is 60% larger than our prior plan.

The other central part of our strategic planning process focuses on the importance of collaboration. We have systematically incorporated a focus upon collaboration into our entire strategic planning and execution process. Our strategic planning efforts began by bringing together the intellectual resources of faculty and staff at St. Jude. This yielded projects that have interactions between many investigators on campus.

We recognize, however, that were still a relatively small institution, and theres a lot of expertise outside. We asked: How can we engage top scientists to tackle problems related to cancer and other catastrophic illnesses of childhood?

In pursuit of this, during our last strategic plan, we created the St. Jude Research Collaboratives, in which we fund investigators from multiple institutions who collaborate with investigators at St. Jude.

Initially, we were planning to fund two or three Collaboratives. However, they were remarkably successful, and top scientists eagerly joined.

Consequently, weve grown the program to five St. Jude Research Collaboratives already. These teams are each funded at an average of $8 million over 5 years, so each investigator is getting R01-level funding, or a little bit better. This has been a phenomenal success.

In the new strategic plan, were going to grow the program to a steady state of 11 funded collaboratives, representing close to a $90 million investment. So far, three of the Collaboratives are directly focused on childhood cancer. A fourth is a basic science-focused project relevant to childhood cancer. Were excited about the growth of this collaboration-focused program.

Lastly, Id like to address global collaboration. If you look across the globe, in low- and middle-income countries, the cure rates for childhood cancer are less than 20%.

This is a problem we know we can solve. Weve proven in the United States we can drive the cure rate to 80%. How can we help the rest of the world?

Because of the resources brought to us by our donors, we are able to think about these things, and so were now collaborating around the globe to drive cure rates forward for childhood cancer worldwide.

JD: As an example of new ideas and how rapidly we can act on them, Id like to tell you about a new blue-sky proposal that came up at the end of the last strategic plan. This idea was precipitated at a faculty retreat. One of our senior investigators was presenting, and during a coffee break, someone said, Well, what if you did this? That emerged into a blue-sky proposal, Seeing the Invisible in Protein Kinases. This was work from Dr. Babis Kalodimos, our Structural Biology department chair. He had a Science paper that came out several months ago, where he used the high-field NMR spectroscopy to look at the structure of the ABL kinase. He was able to identify transient conformational states that help to explain how resistant mutants work.

This gave us new insights into transient states that exist in molecules that can only be seen under high-field NMR, not with other structural biology approaches.

Based on that, we started thinking, Well, what if you did this on all kinases? What if you just did it against tyrosine kinases, serine kinases, receptor tyrosine kinases? What new rules would emerge from this? What would it tell us about families of kinases? What would it tell us about mechanisms of inhibition to kinase inhibitors? What might it tell us about new approaches to developing drugs against protein tyrosine kinases?

And since kinases are a major focus for targeted therapy, there was great excitement about pursuing these studies. Dr. Kalodimos developed the proposal and brought it forward; however, it was clear that this effort would be beyond the scope of our blue-sky process.

Blue-sky initiatives are usually somewhere in the $12 million range, and this was north of $30 million. Yet, in the end after thorough internal and external reviews, the project will move forward as part of the new strategic plan..

This is an approach that will give us fundamental knowledge and can have a profound impact on our understanding of a major class of targets for next-generation therapy.

MO: If I recall accurately, St. Jude has a network of partnerships with a few dozen countries worldwide. Does this plan call for an expansion of efforts within each of those countries? And how many of them?

JD: When I took over in 2014, we had what we called the International Outreach Program, which was 24 programs in 17 countries. During the programs 25-year history, we had made great progress. We were making significant impact and changing the outlook for children with cancer in those 17 countries. But we were affecting only about 3% of children with cancer across the globe, and the International Outreach Program was not scalable.

So, at the beginning of the last strategic plan, we recruited Dr. Carlos Rodriguez-Galindo. He developed a vision that after assessing, we decided to move forward on. This new effort encompasses the Department of Pediatric Global Medicine, St. Jude Global and the St. Jude Global Alliance.

These are all integrated. We developed a model that we think is scalable around the world, and we think this model ultimately can affect children with cancer everywhere.

The idea is that first we must train a workforce to treat children with cancer around the globe. We cant train the workforce ourselves, but we can train the trainers, who will then train the workforce.

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St. Jude's $11.5B, six-year plan aims to improve global outcomes for children with cancer and catastrophic diseases - The Cancer Letter

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Focus on Immuno-Oncology and Next-Generation Vaccines is Supported with a Strong Balance Sheet

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Celsion Corporation Reports First Quarter 2021 Financial Results and Provides Business Update

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CRANBURY, N.J., May 14, 2021 (GLOBE NEWSWIRE) --  PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutants, today reported financial results for the quarter ended March 31, 2021 and provided corporate highlights.

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LEXINGTON, Mass., May 14, 2021 (GLOBE NEWSWIRE) -- Keros Therapeutics, Inc. (“Keros” or the “Company”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel treatments for patients suffering from hematological and musculoskeletal disorders with high unmet medical need, today announced results from a preclinical study of KER-012 on pulmonary and cardiac dysfunction in an established rodent model of pulmonary arterial hypertension (“PAH”) at the virtual American Thoracic Society International Conference held May 14 through 19, 2021. Additional data from a previously conducted nonclinical study in cynomolgus monkeys was also included in the presentation.

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Keros Therapeutics Presents Results from Preclinical Study of KER-012 in Pulmonary Arterial Hypertension at the American Thoracic Society...

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TORONTO, May 14, 2021 (GLOBE NEWSWIRE) -- Spectral Medical Inc. (“Spectral” or the “Company”) (TSX: EDT), a late stage theranostic company advancing therapeutic options for sepsis and septic shock, as well as commercializing a new proprietary platform targeting the renal replacement therapy market through its wholly-owned subsidiary Dialco Medical Inc. (Dialco), today announced its financial results for the first quarter ended March 31, 2021, and provided a corporate update.

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MIAMI, May 14, 2021 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and life-threatening conditions, today provided a business update regarding a number of ongoing clinical trials, and also reported its financial results for the first quarter ended March 31, 2021.

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Longeveron Inc. Provides Corporate Update and Reports First Quarter 2021 Financial Results

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ISELIN, N.J., May 14, 2021 (GLOBE NEWSWIRE) -- Outlook Therapeutics, Inc. (Nasdaq: OTLK), a?late clinical-stage?biopharmaceutical company?working to develop the first FDA-approved ophthalmic formulation of bevacizumab?for use in retinal?indications, today announced recent corporate highlights and financial results for its fiscal second quarter ended March 31, 2021.Outlook Therapeutics also provided a clinical development and pre-commercialization update on ONS-5010 / LYTENAVA™ (bevacizumab-vikg), its investigational ophthalmic formulation of bevacizumab for the treatment of wet age-related macular degeneration (wet AMD) and other retinal indications.“Outlook Therapeutics continues to advance ONS-5010 towards a BLA filing for wet AMD. Having reported promising results from two of the three clinical trials for our planned BLA, we are now in the home stretch and focused on the topline data readout next quarter from NORSE TWO, our pivotal Phase 3 study,” commented Lawrence A. Kenyon, President, CEO and CFO of Outlook Therapeutics. “Additionally, with the funds raised in February, we are well positioned to complete NORSE TWO and prepare the BLA. We are excited about the upcoming clinical milestones and look forward to providing future updates as we continue to advance ONS-5010.”

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Outlook Therapeutics Reports Financial Results for Second Quarter of Fiscal Year 2021 and Provides Corporate Update

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- First Patient Dosed in Phase 1 AL102 Combination Trial with Novartis’ Anti-BCMA Agent for the Treatment of Multiple Myeloma -

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Ayala Pharmaceuticals Reports First Quarter 2021 Financial Results and Provides Business Update

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- Anticipate Emergency Use Submission for SARS-CoV-2Antibody Biosensor Test in the Second Half of 2021-

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GBS Inc. Reports First Quarter 2021 Financial Results and Recent Business Highlights

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Hamilton, Bermuda, May 14, 2021 – Auris Medical Holding Ltd. (NASDAQ: EARS), a clinical-stage company dedicated to developing therapeutics that address important unmet medical needs in neurotology, rhinology and allergy and CNS disorders, today announced that it has reached the midpoint for enrollment in Part B of its Phase 2 proof-of-concept trial of AM-125 (intranasal betahistine) in the treatment of acute peripheral vertigo.

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Auris Medical Reaches Midpoint for Enrollment in Part B of AM-125 Phase 2 TRAVERS Trial in Acute Peripheral Vertigo

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JERSEY CITY, N.J., May 14, 2021 (GLOBE NEWSWIRE) -- SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company pioneering innovative medicines to overcome and prevent difficult-to-treat and drug-resistant infections, today reported that it has secured a $60 million term loan facility with Hercules Capital, Inc. (NYSE: HTGC) and Silicon Valley Bank (SVB). The capital strengthens SCYNEXIS’ balance sheet ahead of the anticipated commercial launch of Brexafemme, the expected trade name for ibrexafungerp, an oral antifungal product candidate for the treatment of vaginal yeast infections, which is under regulatory review by the U.S. Food and Drug Administration (FDA) with a PDUFA target action date of June 1, 2021. This non-dilutive financing further extends SCYNEXIS’ projected cash runway into 2023, based on current operating plans.

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SCYNEXIS Secures Term Loan Facility for up to $60 Million with Hercules Capital and Silicon Valley Bank

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CAMBRIDGE, Mass., May 14, 2021 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal imbalance, today announced that management will participate in the following upcoming virtual investor conferences:

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Praxis Precision Medicines to Present at Upcoming Investor Conferences

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Underwriters’ Full Exercise of Option Brings Gross Proceeds to $34.5 Million

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Recro Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares

Global News Feed Comments Off on Recro Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares | May 15th, 2021

CAMBRIDGE, Mass. and AVENTURA, Fla., May 14, 2021 (GLOBE NEWSWIRE) -- Cytovia Therapeutics, Inc., a biopharmaceutical company developing allogeneic “off-the-shelf” gene-edited Chimeric Antigen Receptor (CAR)-NK cells derived from induced pluripotent stem cells (iPSCs) and NK cell engager multifunctional antibodies, announced today that its CEO, Dr. Daniel Teper, will be presenting at the following industry conferences:

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Cytovia Therapeutics CEO Dr. Daniel Teper to Present at Two Upcoming Conferences

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Conference call scheduled for May 18, 2021 at 4:10 p.m. EDT.

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Qualigen Therapeutics, Inc. Reports First Quarter 2021 Financial Results and Provides Corporate Update

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VANCOUVER, British Columbia, May 14, 2021 (GLOBE NEWSWIRE) -- TOWER ONE WIRELESS CORP. (CSE: TO) (OTCQB: TOWTF) (Frankfurt: 1P3N) (“Tower One” or the “Company”) provides a construction update for the month of April 2021.

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Tower One Construction Update for the Month of April 2021

Global News Feed Comments Off on Tower One Construction Update for the Month of April 2021 | May 15th, 2021

TORONTO, May 14, 2021 (GLOBE NEWSWIRE) -- Aleafia Health Inc. (TSX: AH, OTCQX: ALEAF) (“Aleafia Health” or the “Company”) a global leader in cannabis wellness products and services, today announced that Benjamin Ferdinand, chief financial officer, is departing the Company on amicable terms to pursue a new opportunity in the technology sector. He will continue to serve as CFO during a transition period. The Company has initiated a search process for a new chief financial officer with the help of an executive search firm and will consider internal and external candidates.

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Aleafia Health Announces Changes to Executive Team

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SAN DIEGO, May 14, 2021 (GLOBE NEWSWIRE) -- Prometheus Biosciences, Inc. (Nasdaq: RXDX), a clinical-stage biotechnology company pioneering a precision medicine approach for the discovery, development, and commercialization of novel therapeutic and companion diagnostic products for the treatment of immune-mediated diseases, starting first with inflammatory bowel disease (IBD), today announced that Mark McKenna, President and CEO, will participate virtually in a fireside chat during the 2021 RBC Capital Markets Global Healthcare Conference on Wednesday, May 19, 2021 at 10:55 a.m. ET.

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Prometheus Biosciences to Present at 2021 RBC Capital Markets Global Healthcare Conference

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WORCESTER, Mass., May 14, 2021 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced financial results and recent corporate highlights for the first quarter ended March 31, 2021.

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Mustang Bio Reports First Quarter 2021 Financial Results and Recent Corporate Highlights

Global News Feed Comments Off on Mustang Bio Reports First Quarter 2021 Financial Results and Recent Corporate Highlights | May 15th, 2021