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Adult Leukemia: What You Need to Know – Dana-Farber Cancer Institute

Posted: November 26, 2019 at 6:44 pm

Medically reviewed by Richard M. Stone, MD

More than 60,000 new cases ofadult leukemiaare diagnosed in the U.S. each year. Although it is one of the more common childhood cancers,leukemia occurs more often in older adults.

Leukemia is a cancer of the bodys blood-forming tissues that results in large numbers of abnormal or immature white blood cells. The main types of leukemia are:

AML causes the bone marrow to produce immature white blood cells (called myeloblasts). As a result, patients may have a very high or lowwhite blood cellcount, and lowred blood cellsandplatelets.

CLL is the second most common type of leukemia in adults. It is a type of cancer in which the bone marrow makes too many maturelymphocytes(a type of white blood cell).

ALL is a type of leukemia in which the bone marrow makes too many immaturelymphocytes. Similar to AML, the white blood cells can be high or low and oftentimes the platelets and red blood cells are low. This form of leukemia is more common in children than adults.

CML is usually a slowly progressing disease in which too many mature white blood cells are made in the bone marrow.

People with leukemia may experience:

Because these symptoms can be caused by a variety of other conditions, its important to check with your doctor if they arise.

While studies have shown men to be more atrisk than women, some other risk factors include:

While test procedures vary based on the type of leukemia, the two most common procedures are thecomplete blood count(CBC) test and the bone marrow aspiration biopsy.

CBC is a procedure used to check the redblood cell and platelet counts as well as the number and type of white bloodcells (the red cells carry oxygen, the white cells fight and prevent infection,and platelets control bleeding). A bone marrow aspiration biopsy involvesremoving a sample of bone marrow, including a small piece of bone by insertinga needle into the hipbone. The sample is then examined for abnormal cells.

Treatment for leukemia varies depending on the type and specific diagnosis.

The treatment for acute leukemias may be lengthy up to two years in ALL and is usually done in phases. The first phase, known as remission induction therapy, involves administering several chemotherapy drugs over a several-week period. The goal is to destroy as many cancer cells as possible to achieve a remission (in which cancer cells are undetectable, but small amounts are still present).

The second phase, known aspost-remission or consolidation therapy, seeks to kill leukemia cells thatremain after remission induction therapy. This phase may involve chemotherapyand/or a stem cell transplant.

Additional treatments may also be necessary. ALL patients, for example, may receive special treatment to prevent the disease from recurring in the spinal cord or brain.

The treatment for CML has been revolutionized by the advent of the oral medication imatinib and the second- and third-generation drugs known as tyrosine kinase inhibitors (TKIs). These are oral medications that work to inhibit the function of theBCR-ABLprotein. Many patients take these medications for the rest of their lives. In rare instances, a patient may require a stem cell transplant.

Some patients with CLL are recommended formonitoring and observation. Others,usually those with symptoms or low red cell or platelet counts, requiretreatment. Such treatment may involve intravenous chemotherapy, but often withoral therapy with pills that inhibit the function of a key protein, Brutonstyrosine kinase.

Treatments for leukemia can include:

Drugs that harness the immune system in fighting leukemia have shown considerable promise. Some monoclonal antibodies synthetic versions of immune system proteins are already in use to treat certain forms of leukemia and others are being studies in clinical trials.

Another form of immunotherapy, immune checkpoint inhibitors, which release a pent-up immune system attack on tumor cells, is being tested in several forms of leukemia. Cancer vaccines, which boost the immune systems ability to fight cancer, are being studied for use in leukemia.

CAR T-cell therapy, which uses modified immune system T cells to better target and kill tumor cells, has achieved impressive results in trials involving children and adults up to age 25 with relapsed ALL.

Research into new treatments for adult leukemia is moving along several tracks in addition to immunotherapy.

By tracking the specific abnormal genes within leukemia cells, physicians are increasingly able to tailor treatment to the unique characteristics of the disease in each patient. Targeted drugs such as imatinib and dasatinib, for example, are now used in treating patients with ALL whose leukemia cells have an abnormality known as the Philadelphia chromosome. Targeted agents including IDH or FLT3 inhibitors, which zero in on proteins made from mutated genes, have been approved to treat some patients with AML, while other such inhibitors are being tested in clinical trials.

New tests make it possible to detect ever smaller amounts of leukemia that remain after treatment. Investigators are exploring how these minute levels may influence a patients prognosis and how they might impact treatment.

Researchers are testing whether treatment periods for certain drugs can be safely reduced in some patients. For instance, studies are under way to determine if drugs such as imatinib, which are currently taken for life, can be safely stopped in some patients with CML. Researchers hope to test whether treating patients with CLL with the drug ibrutinib plus other medicine for a fixed amount of time is safe and effective.

Patients may consider treatment through a clinical trial.Dana-Farber currently has more than 30 clinical trials for adult leukemia. A national list of clinical trials is available atclinicaltrials.gov.

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Adult Leukemia: What You Need to Know - Dana-Farber Cancer Institute

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Star Trek’s William Shatner Receives Stem Cell Treatment to Restore his Youth – Magazine of Santa Clarita

Posted: November 26, 2019 at 6:44 pm

The 88-year-old actor known for his role as Captian James T. Kirk on the popular cinema and television series Star Trek, William Shatner recently Tweeted, Today I received restorative stem cells and told his followers Is it possible to turn back the clock? I will let you know.Mr. Shatner also tweeted the Stem Cells are manufactured by Invitrx.Center for Regenerative Medicine & Stem Cell Therapy at Valencia Medical Center is a pioneer in stem cell regenerative medicine in Santa Clarita Valley has been producing PRP and stem cell treatments for cosmetic treatments for cosmetic rejuvenation, hair restoration and chronic knee pain due to arthritis knee meniscus injury, cartilage, ligaments (ACL, MCL), osteoarthritis treatment. Invitrx, a California native is a global research-based company in regenerative medicine and is a major source of stem cell products for Valencia Medical Center.Non-surgical regenerative cell-based treatment uses the bodys natural healing ability to repair damaged bones, muscles, cartilage, tendons and ligaments. Knee injuries are painful and often patients are unable to walk. Our treatment protocol always uses products following FDA guidelines. Injections done with ultrasound guided needle recognition capability to ensure safety as well target the area needing treatment. Plasma; Alpha-2-Macroglobulim (A2M) is the new biologic treatment for your arthritic knee (osteoarthritis)When your hips hurt, or your knee is stiff, or your back is throbbing, that means your joint is bone on bone and there is no lubrication to ease movement.Regenerative medicine giving new hope to patients suffering from painful joint injuries such as knee, shoulder and hip with a chance to live a pain free life.Regenerative cell-based ultrasound guided injection now available to treat pain associated with joint injury. There are indications that it reduces the pain and swelling of the joints and helps lubricating and improve movements.Commonly Treated Conditions: Osteoarthritis of the Hips, Knee, and Shoulders Rotator Cuff tears of the Shoulder Meniscus, ACL and PCL tears of the kneeOur stem cell treatment using your own stem cells and with using imaging guidance ensures precise injection of stem cell, it is a highly-specialized practice.Besides treating above injuries we have advance stem cell micro-needling treatment for the following: Cell-based PRP Hair Restoration combining micro-needling with growth factors and hair follicles voluma vitamins plus BLotinyl T1, Biotin, Anti-aging and Kopexil. Non-toxin facial renewal Anti-Aging APGF Advanced Peptide Micro-needling PRP, Dual Anti-Aging Ampoules for deep hydration, more collagen to reduce wrinkles and firm skin.Dr. Ibrahim is the staff physician at Valencia Medical Center specializing in regenerative medicine, pain management, and rejuvenation. Call for a consultation at 661-222-9117.

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BioLineRx to Host Investor and Analyst Breakfast Meeting on December 5, 2019 in New York – P&T Community

Posted: November 26, 2019 at 6:44 pm

TEL AVIV, Israel, Nov. 26, 2019 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ:BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, announced today that it will host an investor breakfast meeting on Thursday, December 5, 2019 at the Convene Conference Center near Grand Central Terminal in New York, N.Y.

The investor meeting schedule is as follows:

7:45 am EST Registration and breakfast8:10-9:30 am EST Formal presentations

Location:

Convene Conference Center237 Park Avenue (on 46th St. between Park and Lex)New York, N.Y.(888) 730-7307

The event will feature a presentation by Talia Golan, MD, from The Chaim Sheba Medical Center,who will discuss the current treatment landscape as well as the unmet medical need for treating patients with metastatic pancreatic adenocarcinoma (PDAC). Dr. Golan will be available to answer questions at the conclusion of the event.

BioLineRx's management team will also provide an update on the COMBAT/KEYNOTE-202 triple combination study in metastatic PDAC under its collaboration with Merck. In addition, the management team will provide a comprehensive overview of the Company's other developments for BL-8040, as well as its second lead asset, AGI-134. BL-8040 is a novel short synthetic peptide that functions as a high-affinity antagonist for CXCR4, which BioLineRx is developing for the treatment of solid tumors, acute myeloid leukemia (AML) and stem-cell mobilization for bone-marrow transplantation. AGI-134, a synthetic Gal novel immunotherapy, is currently in Phase 1/2a development for solid tumors.

Talia Golan, MD is a clinician-scientist currently conducting translational laboratory research while also serving as medical director ofthe Phase I Unit and the Pancreatic Cancer (PC) Center at Sheba Medical Center in Tel Aviv, one of the leading medical centers in the world. Her clinical interest is in patients with pancreatic cancer. Dr. Golan's career goals include expertise in clinical medicine, translational laboratory research, and drug development. Dr Golan is PI on multiple early-phase trials on immune modulators. Her clinical and research expertise focuses on the understanding and treatment of hereditary pancreatic cancer patients. Dr. Golan is co-global PI of the first biomarker-selected (BRCA) Phase III clinical trial in PC, the POLO study.Dr. Golan's translational research lab, established in 2011, is an integral part of theSheba Pancreatic Cancer Program (SPCC). The research in the lab focuses on improving the standard of care options for pancreatic cancer patients by both finding targeted treatments tailored for each patient based on his own genetic background and developing state of the art early detection methodology. Disease heterogeneity and late detection are still the main challenges of pancreatic cancer treatment.

This event is intended for institutional investors, sell-side analysts, investment bankers, and business development professionals only. Please RSVP in advance if you plan to attend, as space is limited.For those who are unable to attend in person, a live webcast and replay of the event will be accessible here.If you would like to ask a question during the live Q&A portion of the event, please submit your request via email.

About BioLineRx

BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a clinical-stage biopharmaceutical company focused on oncology. The Company's business model is to in-license novel compounds, develop them through clinical stages, and then partner with pharmaceutical companies for further clinical development and/or commercialization.

The Company'slead program, BL-8040, is a cancer therapy platform currently being evaluated in a Phase 2a study for the treatment of pancreatic cancer in combination with KEYTRUDA and chemotherapy under a collaboration agreement with MSD. BL-8040 is also being evaluated in a Phase 2b study in consolidation AML and a Phase 3 study in stem cell mobilization for autologous bone-marrow transplantation. In addition, the Company has an ongoing collaboration agreement with Genentech, a member of the Roche Group, evaluating BL-8040 in combination with Genentech's atezolizumab in two Phase 1b/2 solid tumor studies.

BioLineRx is developing a second oncology program, AGI-134, an immunotherapy treatment for multiple solid tumors that is currently being undergoing in a Phase 1/2a study.

For additional information on BioLineRx, please visit the Company's website at http://www.biolinerx.com, where you can review the Company's SEC filings, press releases, announcements and events. BioLineRx industry updates are also regularly updated on Facebook,Twitter, and LinkedIn.

Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "may," "expects," "anticipates," "believes," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Some of these risks are: changes in relationships with collaborators; the impact of competitive products and technological changes; risks relating to the development of new products; and the ability to implement technological improvements. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 28, 2019. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.

Contact:

Tim McCarthyLifeSci Advisors, LLC+1-212-915-2564tim@lifesciadvisors.com

or

Tsipi HaitovskyPublic Relations+972-52-598-9892 tsipihai5@gmail.com

View original content:http://www.prnewswire.com/news-releases/biolinerx-to-host-investor-and-analyst-breakfast-meeting-on-december-5-2019-in-new-york-300965306.html

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BioLineRx to Host Investor and Analyst Breakfast Meeting on December 5, 2019 in New York - P&T Community

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Vor Biopharma and MaxCyte Announce Clinical and Commercial License Agreement for Engineered Hematopoietic Stem Cells (eHSCs) to Treat Cancer -…

Posted: November 26, 2019 at 6:44 pm

CAMBRIDGE, Mass. & GAITHERSBURG, Md.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and MaxCyte, Inc., a global cell-based therapies and life sciences company, today announced a clinical and commercial license agreement under which Vor will use MaxCytes Flow Electroporation technology to produce eHSCs and initiate Investigational New Drug (IND)-enabling studies to accelerate its progress towards the clinic.

Under the terms of the agreement, Vor obtains non-exclusive clinical and commercial use rights to MaxCytes Flow Electroporation technology and ExPERT platform to develop up to five engineered cell therapies, including VOR33, Vors lead eHSC candidate, which is in development for acute myeloid leukemia (AML). In return, MaxCyte will receive undisclosed development and approval milestones and sales-based payments in addition to other licensing fees.

Vor will use MaxCytes cell engineering platform to deliver its gene editing machinery into hematopoietic stem cells to remove biologically redundant cell surface proteins that are also expressed on blood cancer cells. Once the eHSCs are transplanted into a cancer patient, these cells are effectively hidden from complementary targeted therapies that target the relevant protein, while diseased cells are left vulnerable to attack. Vors approach thereby could unleash the potential of targeted therapies by broadening the therapeutic window and improving the utility of complementary targeted therapies.

MaxCyte is a leader in GMP electroporation technology, and we are thrilled that this agreement provides us with long-term access to a platform technology applicable to a pipeline of eHSC programs used to treat AML and other blood cancers, said Sadik Kassim, Ph.D., Chief Technology Officer of Vor. As we build on promising in vivo data from our lead candidate VOR33, we can now expand our manufacturing capabilities to support later-stage studies, regulatory filings and commercialization of VOR33.

MaxCytes ExPERT instrument family represents the next generation of leading, clinically validated, electroporation technology for complex and scalable cellular engineering. By delivering high transfection efficiency with enhanced functionality, the ExPERT platform delivers the high-end performance essential to enable the next wave of biological and cellular therapeutics.

We look forward to expanding our relationship with Vor Biopharma as the company pioneers a potential future standard of care in hematopoietic stem cell transplants for cancer patients in need, said Doug Doerfler, President & CEO of MaxCyte. This agreement represents another key business milestone for MaxCyte, emphasizing the value of our technology platform applied to next-generation engineered cell therapies that may make a true difference in patient outcomes.

About VOR33Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, CD33 becomes a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. In so doing, Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from AML.

About Vor BiopharmaVor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vors lead program has been published in Proceedings of the National Academy of Sciences.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.

About MaxCyteMaxCyte is a clinical-stage global cell-based therapies and life sciences company applying its proprietary cell engineering platform to deliver the advances of cell-based medicine to patients with high unmet medical needs. MaxCyte is developing novel CARMA therapies for its own pipeline, with its first drug candidate in a Phase I clinical trial. CARMA is MaxCytes mRNA-based proprietary therapeutic platform for autologous cell therapy for the treatment of solid cancers. In addition, through its life sciences business, MaxCyte leverages its Flow Electroporation Technology to enable its biopharmaceutical partners to advance the development of innovative medicines, particularly in cell therapy. MaxCyte has placed its flow electroporation instruments worldwide, including with all of the top ten global biopharmaceutical companies. The Company now has more than 80 partnered programme licenses in cell therapy with more than 45 licensed for clinical use. With its robust delivery technology platform, MaxCyte helps its partners to unlock the full potential of their products. For more information, visit http://www.maxcyte.com.

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Many stem cell clinics promise unapproved treatments: How to stay safe – NBC News

Posted: November 25, 2019 at 8:45 pm

Google has cracked down on pervasive, predatory ads promoting sketchy medical treatments a move that could affect many of the hundreds of stem cell treatments advertised online.

At the end of October, the internet giant stopped accepting ads for unproven or experimental medical techniques, according to a statement from the company. Google is taking this step after seeing a rise in bad actors offering deceptive and untested treatments, the company said.

Stem cells came up this week during the senate confirmation hearing for the next head of the Food and Drug Administration on Wednesday.

"FDA has taken enforcement action, which it should, against stem cell clinics and manufacturers that might be misleading people," Sen. Lamar Alexander (R-Tenn.) said. "On the other hand, we have diabetes advocates who say regenerative medicine may restore pancreas or put out of business a heart transplant surgeon by restoring a heart. Will you commit to taking seriously the promise of regenerative medicine and stem cell medicines?"

Dr. Stephen Hahn, President Trump's nominee to lead the agency, replied that he would "certainly commit to that."

The number of stem cell clinics in the U.S. doubled every year from 2009 to 2014, according to a report in the journal Regenerative Medicine.

Major renowned medical centers such as the Mayo Clinic are researching stem cell therapies for conditions including arthritis and heart problems.

But as stem cell clinics not affiliated with medical centers proliferate, so have serious concerns. In 2017, the New England Journal of Medicine reported that three women were virtually blinded from unproven "stem cell" treatments that actually involved having fat injected into their eyes.

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That has brought increased scrutiny from the FDA, which in June won a court ruling allowing the agency to regulate treatments at the Florida clinic where the three women were harmed.

Stem cell products have only been approved for two uses by the agency: for blood cancers and certain immune disorders. That hasnt stopped some providers from claiming their stem cell treatments can "cure" a wide range of conditions, from macular degeneration to multiple sclerosis unapproved treatments that are not only not effective, but could pose serious health risks.

Stem cells are very young cells that can develop into specialized cells, such as blood cells, bone cells and brain cells. According to the FDA, stem cells have the potential to replace, repair, restore and regenerate other cells, and could possibly be used to treat a number of medical conditions.

The only stem cell-based products currently approved by the FDA consist of blood stem cells derived from umbilical cord blood. These products are usually used in patients with blood cancers, such as leukemia and immune disorders. For patients with blood cancers, for example, stem cells are used after chemotherapy to replenish the blood cells that were destroyed during the treatment.

However, other uses are being studied. There are currently more than 650 active clinical trials for stem cell procedures, looking at a myriad of conditions including kidney failure and Crohn's disease.

But the purpose of a clinical trial is to determine if a treatment works or if it's safe.

People should understand that they're trying something that could potentially have benefit, Dr. Peter Marks, FDA director of biologics, said. But we don't know that benefit is really there, which means that they should understand the risk.

We do our best to make sure people are safe when they're getting investigational products, Marks said. When a product is in a clinical trial, the FDA can monitor it to make sure its safe, he added.

For patients considering stem cell treatments, the FDA recommends asking a health care provider if the FDA has reviewed the treatment.

This also applies to stem cell treatments that use a person's own cells. You may be told that because these are your cells, the FDA does not need to review or approve the treatment. That is not true, according to the FDA website.

The FDA has given that road map on how to do it responsibly, said Dr. Shane Shapiro, medical director at the Mayo Clinics Regenerative Medicine Therapeutics Program in Jacksonville, Florida.

Shapiro also recommends going to a provider that has an expertise in the disease or injury that they are offering stem cell products to treat.

I think it is important to point out when we see a patient at Mayo, they are still seeing someone with the expertise of a physician that is a specialist in the disease they need help with, he said.

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Patrick Martin is an associate producer in the NBC News Health & Medical Unit.

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Tendon stem cell discovery could lead to improvements in injury recovery – Drug Target Review

Posted: November 25, 2019 at 8:45 pm

Researchers have discovered the existence of tendon stem cells, which could lead to improvements in treating tendon injuries, avoiding surgery.

New research has revealed the existence of tendon stem cells which could potentially be harnessed to improve tendon recovery after an injury and perhaps even avoid surgery.

The research was led by Chen-Ming Fan at the Carnegie Institution of Science, US.

Once tendons are injured, they rarely fully recover, which can result in limited mobility and require long-term pain management or even surgery. This is due to fibrous scars, which disrupt the tissue structure of the tendon.

This image shows the Patellar tendon 30 days after an injury. The red marks newly discovered tendon stem cells that have self-renewed and are layered over green marked, original tendon cells. During regeneration, some tendon stem cells differentiate to make newly regenerated tendon cells a process during which they transition into a yellow-orange colour. The blue indicates cellular nuclei (credit: Tyler Harvey).

Fan, along with Carnegies Tyler Harvey and Sara Flamenco, revealed all of the cell types present in the Patellar tendon, found below the kneecap, including previously undefined tendon stem cells.

Because tendon injuries rarely heal completely, it was thought that tendon stem cells might not exist, said lead author Harvey. Many searched for them to no avail, but our work defined them for the first time.

The teams research showed that both fibrous scar tissue cells and tendon stem cells originate in the same space the protective cells that surround a tendon. Moreover, these tendon stem cells are part of a competitive system with precursors of fibrous scars, which explains why tendon healing is such a challenge.

It was thought that tendon stem cells might not exist but our work defined them for the first time

The team demonstrated that both tendon stem cells and scar tissue precursor cells are stimulated into action by a protein called platelet-derived growth factor-A. When tendon stem cells are altered so that they do not respond to this growth factor, then only scar tissue and no new tendon cells form after an injury.

Tendon stem cells exist, but they must outcompete the scar tissue precursors in order to prevent the formation of difficult, fibrous scars, Fan explained. Finding a therapeutic way to block the scar-forming cells and enhance the tendon stem cells could be a game-changer when it comes to treating tendon injuries.

The research was published in Nature Cell Biology.

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

Posted: November 25, 2019 at 8:45 pm

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.

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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.

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Tokyo’s Heartseed reaps $26M to test its stem cell injections for heart failure – FierceBiotech

Posted: November 25, 2019 at 8:45 pm

Tokyo-based Heartseed has raised 2.8 billion yen, or about $26 million U.S., to help develop its stem cell-based treatment for heart failure.

The company has its eyes on two clinical trials, set to start in the next year: an investigator-initiated study through its research partner Keio University in dilated cardiomyopathy, followed by a phase 1/2 trial in late 2020 for heart failure with reduced left ventricular ejection fraction.

Heartseeds treatment differs in approach from other, similar therapies using induced pluripotent stem cells (iPSCs), where sheets of cells are grafted onto the surface of the heart to improve vascularization and blood flow.

How ICON, Lotus, and Bioforum are Improving Study Efficiency with a Modern EDC

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Instead, the company forms spherical clusters of heart cells from purified, allogeneic iPSCs, which are injected directly into the heart muscle wall. As they grow and electrically sync with other cells within the myocardium, Heartseed expects to see improvements in the strength of contractions.

The companys series B round included new investors SBI Investment, JMDC, Gene Techno Science, Nissay Capital and SMBC Capital as well as Astellas Venture Management, returning from Heartseeds series A raise. The latest proceeds bring its total funding to 3.8 billion yen, or about $35 million.

RELATED: Growing transplantable arteries from stem cells

Heartseed was founded in 2015 through the research of its CEO, Keiichi Fukuda, a professor in the department of cardiology at Keio University, and his teams work in regenerative medicine and cell production, purification and delivery.

We are grateful for the support of our investors, which I believe is a reflection of their expectation and confidence that our lead pipeline HS-001 can be a curative therapy for severe HF, with the mechanism that transplanted ventricular-specific highly-purified cardiomyocytes engraft to patients heart and retain for a long-term, Fukuda said in a statement.

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Tokyo's Heartseed reaps $26M to test its stem cell injections for heart failure - FierceBiotech

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Global Stem Cell Banking Market by Source, Application, Service Type & Region – Forecast to 2025 – ResearchAndMarkets.com – Business Wire

Posted: November 25, 2019 at 8:45 pm

DUBLIN--(BUSINESS WIRE)--The "Stem Cell Banking Market by Source, by Application, and by Service Type: Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018-2025" report has been added to ResearchAndMarkets.com's offering.

The report covers a forecast and an analysis of the stem cell banking market on a global and regional level. The study provides historical data from 2016 to 2018 along with a forecast from 2019 to 2025 based on revenue (USD Billion).

The study includes the drivers and restraints of the stem cell banking market along with their impact on the demand over the forecast period. Additionally, the report includes the study of opportunities available in the stem cell banking market on a global level.

In order to give the users of this report a comprehensive view of the stem cell banking market, we have included a competitive landscape and an analysis of Porter's Five Forces model for the market. The study encompasses a market attractiveness analysis, wherein all the segments are benchmarked based on their market size, growth rate, and general attractiveness.

The report provides company market share analysis to give a broader overview of the key players in the market. In addition, the report also covers key strategic developments of the market including acquisitions & mergers, new launch, agreements, partnerships, collaborations & joint ventures, research & development, and regional expansion of major participants involved in the market.

The study provides a decisive view of the stem cell banking market by segmenting it on the basis of source, application, service type, and region. All the segments have been analyzed based on present and future trends and the market is estimated from 2019 to 2025. The regional segment includes the current and forecast demand for North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

Some major players of the global stem cell banking market are Cord Blood Registry (CBR) Systems, Cordlife Group Limited, Cryo-Cell International, ViaCord, Cryo-Save, LifeCell International, StemCyte, Global Cord Blood Corporation, Vita34, Smart Cells International, and CryoHoldco.

Key Topics Covered

Chapter 1. Introduction

1.1. Report Description & Scope

1.2. Research Scope

1.3. Research Methodology

Chapter 2. Executive Summary

2.1. Global Stem Cell Banking Market, 2016-2025 (USD Billion)

2.2. Global Stem Cell Banking Market: Snapshot

Chapter 3. Stem Cell Banking Market - Industry Dynamics

3.1. Introduction

3.2. Market Drivers

3.3. Market Restraints

3.4. Opportunities

3.5. Porter's Five Forces Analysis

3.6. Market attractiveness analysis

Chapter 4. Global Stem Cell Banking Market - Competitive Landscape

4.1. Company Market Share Analysis

4.2. Strategic Development

Chapter 5. Global Stem Cell Banking Market - Source Analysis

5.1. Global Stem Cell Banking Market: Source Overview

5.2. PSCs

5.3. hESCs

5.4. BMSCs

5.5. ADSCs

5.6. Others

Chapter 6. Global Stem Cell Banking Market - Application Analysis

6.1. Global Stem Cell Banking Market: Application Overview

6.2. Personalized Banking

6.3. Clinical

6.4. Research

Chapter 7. Global Stem Cell Banking Market - Service Type Analysis

7.1. Global Stem Cell Banking Market: Service Type Overview

7.2. Sample Collection & Transportation

7.3. Sample Processing

7.4. Sample Analysis

7.5. Sample Preservation & Storage

Chapter 8. Global Stem Cell Banking Market - Regional Analysis

8.1. Global Stem Cell Banking Market: Regional Overview

8.2. North America

8.3. Europe

8.4. Asia-Pacific

8.5. Latin America

8.6. The Middle East & Africa

Chapter 9. Company Profiles

9.1. Cord Blood Registry (CBR) Systems

9.2. Cordlife Group Limited

9.3. Cryo-Cell International

9.4. ViaCord

9.5. Cryo-Save

9.6. LifeCell International

9.7. StemCyte

9.8. Global Cord Blood Corporation

9.9. Smart Cells International

9.10. Vita34

9.11. CryoHoldco

For more information about this report visit https://www.researchandmarkets.com/r/t0fyz4

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Global Stem Cell Banking Market by Source, Application, Service Type & Region - Forecast to 2025 - ResearchAndMarkets.com - Business Wire

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ALS Stem Cell Therapy Developer Seeks Amendment to its AstroRx Trial – ALS News Today

Posted: November 25, 2019 at 8:45 pm

Kadimastem plans to submit an amendment to the protocol of its ongoing Phase 1/2a trial in Israel assessing the use of AstroRx, an off-the-shelf stem cell therapy, for the treatment of amyotrophic lateral sclerosis (ALS).

The company wants to change the planned dosage in one of the studys cohorts and test repeated injections of a low dose of AstroRx, instead of the medium dose that was originally planned.

The request is based on positive interim data from the first set of five patients given a single intrathecal (into the spinal canal) injection of the lowest dose (100 x 106 cells) of AstroRx. At this dose, the therapy was found to be safe with no serious side effects or dose-limiting toxicities identified, according to the data, which was released in September.

Moreover, AstroRx significantly reduced disease progression after three months of treatment, compared to the start (baseline) of the trial. The ALS Functional Rating Scale revised (ALSFRS-R) score decreased on average by 0.87 per month during the three months before treatment; however, it started to increase again (on average by 0.26 per month) in the three months after treatment. The ALSFRS-R score is a validated assessment of disability progression, with lower scores indicating greater motor impairment.

Evidence suggests that poorly working astrocytes (cells which support and protect neurons) are involved in the progression of ALS. AstroRx is composed of healthy functional astrocytes, which have been derived from human embryonic stem cells. The treatment, injected into a patients spinal fluid, is thought to compensate for the diseased astrocytes and prevent the death of motor neurons, thereby slowing disease progression.

AstroRx was granted orphan drug status by the U.S. Food and Drug Administration in November 2018 for the treatment of ALS.

Preclinical (in the lab) studies have shown that AstroRx was safe, delayed disease onset, maintained muscle function, and increased survival in rodent models of ALS.

The ongoing open-label Phase 1/2a clinical trial (NCT03482050) is testing the safety and effectiveness of AstroRx in ALS patients. The trial is being conducted at Hadassah Ein-Kerem Medical Center in Israel where it recruited 21 patients withearly stagedisease.

The trials original protocol included four doses of AstroRx delivered into the spinal canal: a low (100 x 106 cells), medium (250 x 106 cells), or high (500 x 106 cells) dose.

The primary outcome of the trial is to assess the safety and tolerability of AstroRx. Secondary outcome measures include changes in patients ALSFRS-R scores, respiratory muscle strength, hand grip strength, limb muscle strength, and quality of life.

In cohort A, participants received a single low dose of the therapy. In cohort B, participants received a single medium dose of the therapy. Results from cohort A are expected to be reported by the end of 2019, and cohort B results are expected in 2020.

Based on the positive interim results from cohort A, Kadimastem is seeking to amend the therapy regime being assessed in cohort C and D, so that cohort C will receive two injections of the low dose (instead of the originally planned medium dose), with the injections being separated by two to three months. Results from this section of the trial are expected to be reported in the first half of 2021. Under the amendment, cohort D participants will receive the regimen originally planned for cohort C (repeated administration of the medium dose, 250 x 106). Cohort D dosing will be dependent on the results of the previous cohorts.

We are the first to treat ALS patients with astrocyte cells. Following our positive interim results, we look forward to achieving a prolonged therapeutic effect in the repeated low dose administration, bringing new hope for patients with this incurable disease Rami Epstein, CEO of Kadimastem, said in a press release.

Kadimastem expects to submit an investigational new drug application to the U.S. Food and Drug Administration by mid-2021 with the aim of testing AstroRx in a multi-center clinical trial, which will compare the current and frozen version of the therapy.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

Total Posts: 279

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.

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ALS Stem Cell Therapy Developer Seeks Amendment to its AstroRx Trial - ALS News Today

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