Actinium Presents Positive Findings from Mid-Point Analysis of Pivotal SIERRA Trial of Iomab-B at 2020 Transplantation & Cellular Therapy Meetings of…

By daniellenierenberg

NEW YORK, Feb. 21, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") announced today findings from the SIERRA trial that were presented at the 2020 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (TCT) in Orlando, FL. Dr. Boglarka Gyurkocza, the principal investigator from Memorial Sloan Kettering Cancer Center, revealed that there were key differences in side effects reported in patients treated in the Iomab-B and control arms of the study with rates of febrile neutropenia, sepsis and mucositis being markedly lower in the Iomab-B arm.

The oral presentation also featured updated results from the fifty percent enrollment mid-point analysis including BMT access rates, engraftment and 100-day non-relapse transplant related mortality (TRM). After accounting for these factors, the results showed that, on an intent-to-treat or ITT basis, 78 percent of patients in the Iomab-B arm are potentially evaluable for the primary endpoint compared to 13 percent in the control arm. In addition, an important and recent protocol amendment was highlighted; the approximately 30 percent of patients who are expected to fail induction therapy with venetoclax plus hypomethylating agents1 are now eligible for enrollment in the SIERRA trial. The change is expected to increase the addressable patient population of the study given that this combination is now recommended as part of the NCCN or National Comprehensive Cancer Network guidelines, is widely used and expected to become the treatment of choice.

Key Interim Results:

The data tables that follow summarize key findings that were presented as part of the TCT proceedings. As highlighted in the table below, patients receiving Iomab-B showed lower rates of key adverse events relevant in the BMT setting in compared to patients randomized to receive physician's choice of salvage chemotherapy on the control arm. For example, patients receiving Iomab-B had a much lower incidence of sepsis of 3 percent compared to the 42 percent incidence in the control arm.

Adverse Event* N (%)

Randomized to Iomab-B andreceived BMT (N=31)

Randomized to Control Armand received BMT (N=7)

Febrile Neutropenia

8 (25.8)

3 (42.8)

Sepsis/Septic Shock

1 (3.2)

3 (42.8)

Stomatitis (mucositis)

3 (9.7)

2 (28.6)

Pneumonia/Lung Infection

4 (12.9)

1 (14.3)

Hypertension

61 (19.4)

1 (14.3)

Decreased Appetite

5 (16.1)

0 (0.0)

Device related infection

4 (12.9)

1 (14.3)

Hypophosphatemia

2 (6.5)

1 (14.3)

* All adverse events reported irrespective of attribution to protocol-directed procedures

1) 5 patients had hypertension considered unrelated to Iomab-B and 1 patient had hypertension possibly related to Iomab-B

"This detailed safety data from SIERRA is highly encouraging, particularly in this patient population, as neutropenia and sepsis are hallmark toxicities associated with chemotherapy-based conditioning regimens that next to relapse are leading causes of morbidity and mortality post-transplant," said Dr. Vijay Reddy, Vice President, Clinical Development and Head of BMT at Actinium. "Chemotherapy-based conditioning damages normal organs, such as the gastrointestinal tract, which allows gut bacteria to cause serious infections. The data showing less mucositis, febrile neutropenia, and sepsis are consistent with the targeted nature of Iomab-B, since a lack of damage to the gastrointestinal track would lead to a reduction in these adverse events. BMT is the only curable treatment option for older patients with active, relapsed or refractory AML. Yet these patients face restricted access and suboptimal outcomes due to reliance on chemotherapy-based conditioning regimens and perceptions in the hematologist community around safety and eligibility for BMT. With the SIERRA trial, our goals are to eliminate these barriers leading to more patients receiving BMT and with better patient outcomes. With this additional safety data in hand, we have even greater confidence in our ability to change the perceptions around BMT and are excited to update the transplant and hematology communities on Iomab-B's potential to positively impact patients through improved access to BMT and better outcomes."

The presentation highlighted that 100 percent (31/31) of patients receiving a therapeutic dose of Iomab-B achieved successful BMT engraftment with only a 6 percent (2/31) TRM rate compared to the control arm where 18 percent (7/38) achieved engraftment with a 29 percent (2/7) TRM rate. At the 100-day post BMT time point, on an ITT basis, there were 29 patients from the Iomab-B study arm potentially evaluable for the primary endpoint of durable Complete Remission (dCR) at 180 days compared to 5 patients in the control arm. By this measure, 78 percent of patients in the Iomab-B arm are potentially eligible for the dCR primary endpoint compared to 13 percent of patients in the control arm. The mid-point analysis and data presented at TCT can be viewed here.

Detailed engraftment data is presented in the table below:

BMT Feasibility andOutcome Data

Randomized toStudy Arm (N=37)

Randomized to Control Arm (N=38)

ReceivedTherapeutic Doseof Iomab-B andreceived BMT(N=31)1

Achieve CR andreceived standardBMT (N=7)

Did not Achieve CR(N=31/38)2

Crossed over from toIomab-B and receivedBMT (N=20)

BMT Engraftment Rate (%, N)

100% (31/31)

18% (7/38)

100% (20/20)

Median Bone Marrow Blasts% at randomization

(%, range)

29% (5-88)

26% (5-97)

At crossover: 31%

(6-87)

At randomization: 35%(5-75)

Median Days to BMT postrandomization (days, range)

30 (23-50)

67 (51-86)

64 (44-161)

Median Days to AbsoluteNeutrophil Count Engraftment(days, range)

15 (9-22)3

18 (13-82)4

14 (10-37)5

Median Day to PlateletEngraftment (days, range)

20 (4-39)3

22 (9-35)4

19 (13-38)5

100-day non-relapsetransplant related mortality(%, N)

6% (2/31)

29% (2/7)

10% (2/20)6

1) No therapeutic dose (6) due to: declining Karnofsky Performance Scale (PFS) (3), Infusion reaction (1), unfavorablebiodistribution (1), post-randomization eligibility (1)

2) Ineligible for crossover (9) due to: hospice care/progression (4), declined/ineligible for BMT (2), died pre-crossover (3),eligible for crossover (2) did not receive Iomab-B due to declining status

3) Absolute Neutrophil Count engraftment data not available (1), platelet engraftment data not available (4)

4) ANC and platelet engraftment data not available (1), engraftment failure (1)

5) ANC engraftment data not available (1) out of 20, platelet engraftment data not available (3)

6) 1 patient at 161 days had delayed transplant due to infection and respiratory failure, received Iomab-B and BMT when stable

Dr. Mark Berger, Actinium's Chief Medical Officer, stated, "As we approach critical enrollment milestones in the SIERRA trial, our focus turns to bringing Iomab-B to as many patients as possible that can benefit from this product candidate and as expeditiously as possible. Consequently, we made an important amendment to the SIERRA protocol to expand the potential patient pool by including in the eligibility criteria patients who fail induction therapy with venetoclax plus hypomethylating agents. As targeted agents such as venetoclax and others have gained approval, the acute myeloid leukemia treatment landscape has evolved with a significant percentage of patients being treated with these agents in frontline and relapsed settings. In fact, as of mid-2019, venetoclax plus hypomethylating agents have been included as part of the AML National Comprehensive Cancer Network guidelines and medical practice is embracing these regimens widely. However, these regimens are not curative, nor do they eliminate the need for BMT. Indeed, approximately thirty percent of patients fail to achieve a remission after two cycles of induction therapy with venetoclax and most patients ultimately relapse with a median duration of response of less than one year. This amendment has already had a positive impact on the trial that we expect to continue through the remaining portion of enrollment. Most importantly, if Iomab-B gains approval, this amendment will support its use for the significant and growing number of patients receiving and failing venetoclax as induction therapy instead of traditional 7+3 induction chemotherapy. We look forward to continuing to provide key updates as SIERRA reaches key milestones and completes enrollment in 2020."

Sources: 1) DiNardo et al. Venetoclax combined with decitabine or azacitidine in treatment-nave, elderly patients with acute myeloid leukemia. Blood 2019 133(1): 7-17 https://doi.org/10.1182/blood-2018-08-868752

About the SIERRA TrialThe SIERRA trial (Study ofIomab-B inElderlyRelapse/RefractoryAcute Myeloid Leukemia) is the only randomized Phase 3 trial that offers BMT (Bone Marrow Transplant) as an option for older patients with active, relapsed or refractory AML or acute myeloid leukemia. BMT is the only potentially curative treatment option for older patients with active relapsed or refractory AML and there is no standard of care for this indication other than salvage therapies. The SIERRA trial is a 150-patient, multicenter randomized trial that studying Iomab-B compared to physician's choice of salvage chemotherapy. The primary endpoint of the SIERRA trial is durable Complete Remission of 180 days and the secondary endpoint is 1-year overall survival. Iomab-B is an ARC or Antibody Radiation-Conjugate comprised of the anti-CD45 antibody apamistamab and the radioisotope I-131 (Iodine-131). The 20 active SIERRA trial sites in the U.S. and Canada represent many of the leading bone marrow transplant centers by volume. For more information, visit http://www.sierratrial.com.

About Transplantation & Cellular Therapy Meetings (TCT)

TCT, formerly known as the BMT Tandem Meetings, are the combined annual meetings of the American Society for Blood and Marrow Transplantation (ASBMT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).Each year the conference brings together several thousand investigators, clinicians, researchers, nurses and other allied health professionals from over 500 transplant centers from over 50 countries around a full scientific program focused on bone marrow transplant and cellular therapies.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3Study ofIomab-B inElderlyRelapsed orRefractoryAcute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans Vitzthum LifeSci Advisors, LLCHans@LifeSciAdvisors.com (617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

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SOURCE Actinium Pharmaceuticals, Inc.

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February 21st, 2020

Stem cells and the heartthe road ahead – Science Magazine

By daniellenierenberg

Heart disease is the primary cause of death worldwide, principally because the heart has minimal ability to regenerate muscle tissue. Myocardial infarction (heart attack) caused by coronary artery disease leads to heart muscle loss and replacement with scar tissue, and the heart's pumping ability is permanently reduced. Breakthroughs in stem cell biology in the 1990s and 2000s led to the hypothesis that heart muscle cells (cardiomyocytes) could be regenerated by transplanting stem cells or their derivatives. It has been 18 years since the first clinical trials of stem cell therapy for heart repair were initiated (1), mostly using adult cells. Although cell therapy is feasible and largely safe, randomized, controlled trials in patients show little consistent benefit from any of the treatments with adult-derived cells (2). In the meantime, pluripotent stem cells have produced bona fide heart muscle regeneration in animal studies and are emerging as leading candidates for human heart regeneration.

In retrospect, the lack of efficacy in these adult cell trials might have been predicted. The most common cell type delivered has been bone marrow mononuclear cells, but other transplanted cell types include bone marrow mesenchymal stromal cells and skeletal muscle myoblasts, and a few studies have used putative progenitors isolated from the adult heart itself. Although each of these adult cell types was originally postulated to differentiate directly into cardiomyocytes, none of them actually do. Indeed, with the exception of skeletal muscle myoblasts, none of these cell types survive more than a few days in the injured heart (see the figure). Unfortunately, the studies using bone marrow and adult resident cardiac progenitor cells were based on a large body of fraudulent work (3), which has led to the retraction of >30 publications. This has left clinical investigators wondering whether their trials should continue, given the lack of scientific foundation and the low but measurable risk of bleeding, stroke, and infection.

Additionally, investigators have struggled to explain the beneficial effects of adult cell therapy in preclinical animal models. Because none of these injected cell types survive and engraft in meaningful numbers or directly generate new myocardium, the mechanism has always been somewhat mysterious. Most research has focused on paracrine-mediated activation of endogenous repair mechanisms or preventing additional death of cardiomyocytes. Multiple protein factors, exosomes (small extracellular vesicles), and microRNAs have been proposed as the paracrine effectors, and an acute immunomodulatory effect has recently been suggested to underlie the benefits of adult cell therapy (4). Regardless, if cell engraftment or survival is not required, the durability of the therapy and need for actual cells versus their paracrine effectors is unclear.

Of particular importance to clinical translation is whether cell therapy is additive to optimal medical therapy. This remains unclear because almost all preclinical studies do not use standard medical treatment for myocardial infarction. Given the uncertainties about efficacy and concerns over the veracity of much of the underlying data, whether agencies should continue funding clinical trials using adult cells to treat heart disease should be assessed. Perhaps it is time for proponents of adult cardiac cell therapy to reconsider the approach.

Pluripotent stem cells (PSCs) include embryonic stem cells (ESCs) and their reprogrammed cousins, induced pluripotent stem cells (iPSCs). In contrast to adult cells, PSCs can divide indefinitely and differentiate into virtually every cell type in the human body, including cardiomyocytes. These remarkable attributes also make ESCs and iPSCs more challenging to control. Through painstaking development, cell expansion and differentiation protocols have advanced such that batches of 1 billion to 10 billion pharmaceutical-grade cardiomyocytes, at >90% purity, can be generated.

Preclinical studies indicate that PSC-cardiomyocytes can remuscularize infarcted regions of the heart (see the figure). The new myocardium persists for at least 3 months (the longest time studied), and physiological studies indicate that it beats in synchrony with host myocardium. The new myocardium results in substantial improvement in cardiac function in multiple animal models, including nonhuman primates (5). Although the mechanism of action is still under study, there is evidence that these cells directly support the heart's pumping function, in addition to providing paracrine factors. These findings are in line with the original hope for stem cell therapyto regenerate lost tissue and restore organ function. Additional effects, such as mechanically buttressing the injured heart wall, may also contribute.

Breakthroughs in cancer immunotherapy have led to the adoption of cell therapies using patient-derived (autologous) T cells that are genetically modified to express chimeric antigen receptors (CARs) that recognize cancer cell antigens. CAR T cells are the first U.S. Food and Drug Administration (FDA)approved, gene-modified cellular pharmaceutical (6). The clinical and commercial success of autologous CAR T cell transplant to treat B cell malignancies has opened doors for other complex cell therapies, including PSC derivatives. There is now a regulatory path to the clinic, private-sector funding is attracted to this field, and clinical investigators in other areas are encouraged to embrace this technology. Indeed, the first transplants of human ESC-derived cardiac progenitors, surgically delivered as a patch onto the heart's surface, have been carried out (7). In the coming years, multiple attempts to use PSC-derived cardiomyocytes to repair the human heart are likely.

What might the first human trials look like? These studies will probably employ an allogeneic (non-self), off-the-shelf, cryopreserved cell product. Although the discovery of iPSCs raised hopes for widespread use of autologous stem cell therapies, the current technology and regulatory requirements likely make this approach too costly for something as common as heart disease, although this could change as technology and regulations evolve. Given that it would take at least 6 months to generate a therapeutic dose of iPSC-derived cardiomyocytes, such cells could only be applied to patients whose infarcts are in the chronic phase where scarring (fibrosis) and ventricular remodeling are complete. Preclinical data indicate that chronic infarcts benefit less from cardiomyocyte transplantation than do those with active wound-healing processes.

Adult cells from bone marrow or the adult heart secrete beneficial paracrine factors but do not engraft in the infarcted heart. Pluripotent stem cells give rise to cardiomyocytes that engraft long term in animal models, beat in synchrony with the heart, and secrete beneficial paracrine factors. Long-term cardiomyocyte engraftment partially regenerates injured heart, which is hypothesized to bring clinical benefits.

The need for allogeneic cells raises the question of how to prevent immune rejection, both from innate immune responses in the acute phase of transplantation or from adaptive immune responses that develop more slowly through the detection of non-self antigens presented by major histocompatibility complexes (MHCs). A current strategy is the collection of iPSCs from patients who have homozygous MHC loci, which results in exponentially more MHC matches with the general population. However, studies in macaque monkeys suggest that MHC matching will be insufficient. In a macaque model of brain injury, immunosuppression was required to prevent rejection of MHC-matched iPSC-derived neurons (8). Similarly, MHC matching reduced the immunogenicity of iPSC-derived cardiomyocytes transplanted subcutaneously or into the hearts of rhesus macaques, but immunosuppressive drugs were still required to prevent rejection (9).

Numerous immune gene editing approaches have been proposed to circumvent rejection, including preventing MHC class I and II molecule expression, overexpressing immunomodulatory cell-surface factors, such CD47 and human leukocyte antigen E (HLA-E) and HLA-G (two human MHC molecules that promote maternal-fetal immune tolerance), or engineering cells to produce immunosuppressants such as programmed cell death ligand 1 (PDL1) and cytotoxic T lymphocyteassociated antigen 4 (CTLA4) (10). These approaches singly or in combination seem to reduce adaptive immune responses in vitro and in mouse models. Overexpressing HLA-G or CD47 also blunts the innate natural killer cellmediated response that results from deleting MHC class I genes (11). However, these manipulations are not without theoretical risks. It could be difficult to clear viral infections from an immunostealthy patch of tissue, and possible tumors resulting from engraftment of PSCs might be difficult to clear immunologically.

Ventricular arrhythmias have emerged as the major toxicity of cardiomyocyte cell therapy. Initial studies in small animals showed no arrhythmic complications (probably because their heart rates are too fast), but in large animals with human-like heart rates, arrhythmias were consistently observed (5, 12). Stereotypically, these arrhythmias arise a few days after transplantation, peak within a few weeks, and subside after 4 to 6 weeks. The arrhythmias were well tolerated in macaques (5) but were lethal in a subset of pigs (12). Electrophysiological studies indicate that these arrhythmias originate in graft regions from a source that behaves like an ectopic pacemaker. Understanding the mechanism of these arrhythmias and developing solutions are major areas of research. There is particular interest in the hypothesis that the immaturity of PSC-cardiomyocytes contributes to these arrhythmias, and that their maturation in situ caused arrhythmias to subside.

A successful therapy for heart regeneration also requires understanding the host side of the equation. PSC-derived cardiomyocytes engraft despite transplantation into injured myocardium that is ischemic with poor blood flow. Although vessels eventually grow in from the host tissue, normal perfusion is not restored. Achieving a robust arterial input will be key to restoring function, which may require cotransplanting other cell populations or tissue engineering approaches (13, 14). Most PSC-mediated cardiac cell therapy studies have been performed in the subacute window, equivalent to 2 to 4 weeks after myocardial infarction in humans. At this point, there has been insufficient time for a substantial fibrotic response. Fibrosis has multiple deleterious features, including mechanically stiffening the tissue and creating zones of electrical insulation that can cause arrhythmias. Extending this therapy to other clinical situations, such as chronic heart failure, will require additional approaches that address the preexisting fibrosis. Cell therapy may again provide an answer because CAR T cells targeted to cardiac fibroblasts reduced fibrosis (15).

Developing a human cardiomyocyte therapy for heart regeneration will push the limits of cell manufacturing. Each patient will likely require a dose of 1 billion to 10 billion cells. Given the widespread nature of ischemic heart disease, 105 to 106 patients a year are likely to need treatment, which translates to 1014 to 1016 cardiomyocytes per year. Growing cells at this scale will require introduction of next generation bioreactors, development of lower-cost media, construction of large-scale cryopreservation and banking systems, and establishment of a robust supply chain compatible with clinical-grade manufacturing practices.

Beyond PSC-cardiomyocytes, other promising approaches include reactivating cardiomyocyte division and reprogramming fibroblasts to form new cardiomyocytes. However, these approaches are at an earlier stage of development, and currently, PSC-derived cardiomyocyte therapy is the only approach that results in large and lasting new muscle grafts. The hurdles to this treatment are known, and likely addressable, thus multiple clinical trials are anticipated.

Acknowledgments: C.E.M. and W.R.M. are scientific founders of and equity holders in Sana Biotechnology. C.E.M. is an employee of Sana Biotechnology. W.R.M. is a consultant for Sana Biotechnology. C.E.M. and W.R.M. hold issued and pending patents in the field of stem cell and regenerative biology.

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February 21st, 2020

Data On Enlivex’s Allocetra-OTS Immunotherapy for Peritoneal Solid Tumors and for Prevention of GvHD Selected for Presentation at the Transplantation…

By daniellenierenberg

Nes-Ziona, Israel, Feb. 20, 2020 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV), a clinical-stage immunotherapy company, today announced that the company wasselected, for a scientific presentation of two posters: (i) Allocetra-OTS: Early Apoptotic Cells for Immune Homeostasis in Human Stem Cell Transplantation (HSCT) and for the Prevention of Graft Versus Host Disease (GvHD), and (ii) Apoptotic Cells Reprogram Resident Macrophages to Support Chimeric Antigen Receptor (CAR) T Cell Therapy Against Peritoneal Solid Tumor, at the Transplantation & Cellular Therapy Meetings Conference of the ASTCT and CIBMTR (TCT), held on February 19-23, 2020, in Orlando, Florida.

Allocetra-OTS: Early Apoptotic Cells for Immune Homeostasis in Human Stem Cell Transplantation (HSCT) and for the Prevention of Graft Versus Host Disease (GvHD)

Results from preclinical and clinical studiesy suggested that a single infusion of donor early apoptotic cells (Allocetra) as prophylaxis for GvHD in myeloablative HSCT is safe and potentially effective and led to 0% (0/6) of acute high grade II-IV GvHD in the two higher dosages compared to 52% in matched historical control. Enlivex is planning to initiate a Phase 2/3 multi-center, open-label, 2-arm study (ENX-CL-01-002), in Israel and Germany, that will evaluate the efficacy and safety of Allocetra-OTS (140x106cells/kg) with or without anti-thymocyte globulin (ATG) for the prevention of GvHD in subjects undergoing HLA-matched HSCT from an unrelated donor.

Apoptotic Cells Reprogram Resident Macrophages to Support Chimeric Antigen Receptor (CAR) T Cell Therapy Against Peritoneal Solid Tumor

Preclinical studies showed significantly increased duration of survival and overall survival for study subjects who were treated with the combination therapy, as compared to stand-alone solid tumor CAR-T therapy. The results of these preclinical studies showed that the mechanism of action significantly increased the anti-tumor macrophage population surrounding the human solid tumor microenvironment in the subjects who were treated with the combination therapy.

ALLOCETRATMby Enlivex was designed toprovide a novel immunotherapy mechanism of actionthat targets life-threatening clinical indications that are defined as unmet medical needs, includingprevention or treatment of complications associated with bone marrow transplantations (BMT) and/or hematopoietic stem cell transplantations (HSCT); organ dysfunction and acute multiple organ failure associated with sepsis; and enablement of an effective treatment of solid tumors via immune checkpoint rebalancing.

ABOUT ENLIVEXEnlivex is a clinical stage immunotherapy company, developing an allogeneic drug pipeline for immune system rebalancing. Immune system rebalancing is critical for the treatment of life-threatening immune and inflammatory conditions which involve an out of control immune system (e.g. Cytokine Release Syndrome) and for which there are no approved treatments (unmet medical needs), as well as solid tumors immune-checkpoint rebalancing. For more information, visit http://www.enlivex.com.

ABOUT EUROPEAN MOLECULAR BIOLOGY ORGANIZATIONThe TCT | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (TCT Meetings) are the combined annual meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).

Safe Harbor Statement: This press release contains forward-looking statements, which may be identified by words such as expects, plans, projects, will, may, anticipates, believes, should, would, intends, estimates, suggests, has the potential to and other words of similar meaning, including statements regarding expected cash balances, market opportunitiesfor the results of current clinical studies and preclinical experiments, the effectiveness of, and market opportunitiesfor, ALLOCETRATMprograms, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that forward-looking statements involve risks and uncertainties that may affect Enlivexs business and prospects, including the risks that Enlivex may not succeed in generating any revenues or developing any commercial products; that the products in development may fail, may not achieve the expected results or effectiveness and/or may not generate data that would support the approval or marketing of these products for the indications being studied or for other indications; that ongoing studies may not continue to show substantial or any activity; and other risks and uncertainties that may cause results to differ materially from those set forth in the forward-looking statements. The results of clinical trials in humans may produce results that differ significantly from the results of clinical and other trials in animals. The results of early-stage trials may differ significantly from the results of more developed, later-stage trials. The development of any products using the ALLOCETRATMproduct line could also be affected by a number of other factors, including unexpected safety, efficacy or manufacturing issues, additional time requirements for data analyses and decision making, the impact of pharmaceutical industry regulation, the impact of competitive products and pricing and the impact of patents and other proprietary rights held by competitors and other third parties. In addition to the risk factors described above, investors should consider the economic, competitive, governmental, technological and other factors discussed in Enlivexs filings with the Securities and Exchange Commission, including under the heading Risk Factors contained in Enlivexs most recently filed Annual Report on Form 20-F. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward-looking statements, except as required under applicable law.

ENLIVEX CONTACT: Shachar Shlosberger, CFO Enlivex Therapeutics, Ltd.shachar@enlivex-pharm.com

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February 21st, 2020

CytoDyn Interview to Air on Bloomberg International on the RedChip Money Report – Yahoo Finance

By daniellenierenberg

VANCOUVER, Washington, Feb. 20, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (CYDY) (CytoDyn or the Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today an interview with president and chief executive officer Nader Pourhassan, Ph.D., will air on The RedChip Money Report television program. The interview will air Sunday, February 23, at 3 p.m. local time on Bloomberg International, available in 100+ million homes across Europe.

In the exclusive interview, Dr. Pourhassan discusses the Companys pipeline of innovative treatments for multiple indications, as well as the anticipated upcoming commercial launch of its HIV combination therapy.

To view the interview segment, please visit: https://youtu.be/wQUEoQlq59Y

The RedChip Money Report" delivers insightful commentary on small-cap investing, interviews with Wall Street analysts, financial book reviews, as well as featured interviews with executives of public companies.

About Leronlimab (PRO 140)The U.S. Food and Drug Administration (FDA) have granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases including NASH. Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays an important role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is therefore conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. Additional research is being conducted with leronlimab in the setting of cancer and NASH with plans to conduct additionalclinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation and may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to further support the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD and that blocking this receptor from recognizing certain immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and in immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in the first quarter of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients and plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

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Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSMedia:Grace FotiadesLifeSci Communicationsgfotiades@lifescicomms.com(646) 876-5026

Investors: Dave Gentry, CEORedChip CompaniesOffice: 1.800.RED.CHIP (733.2447)Cell: 407.491.4498dave@redchip.com

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February 21st, 2020

Biotech: Its not a get-rich-quick scheme judging by these HY reports – Stockhead

By daniellenierenberg

Were deep into half-year reporting season and a swarm of medical companies opened their books to investors overnight.

Being biotechs, some dont make money and many of those never will either, if they cant get the drug or device theyre developing to work.

Others are well on the revenue pathway.

This company is developing treatments that spur the bodys immune system to fight cancer. It technically doesnt make money per se, booking a $6m loss, but does earn money from other companies it has licensed drugs to.

In the half year, Immutep earned a milestone payment of $7.4m from GlaxoSmithKline, which dosed its first patient in a phase II clinical trial evaluating a treatment derived from an Immutep antibody in ulcerative colitis.

Immutep has licensed drugs to Novartis, CYTLIMIC, and Chinese company EOC Pharma.

Pharmaceuticals seller Mayne Pharma reported lower numbers across all metrics. Revenue was down 17 per cent, EBITDA dropped by 47 per cent, its loss widened to $17.5m, and even underlying EBITDA and operating cashflow fell too.

CEO Scott Richards said as previously foreshadowed at the AGM the company had faced aggressive competition on its key generic products in the US. Mayne Pharma cut costs by $10m and dumped some generic products.

Richards is hopeful a new oral contraceptive the company has acquired will help it bounce back.

IVF provider Monash also hasnt had a great half, with all key numbers down: revenue dipped to $77m, profit dropped 15 per cent to just under $10m, all forms of EBITDA and EBIT (there are a few ways to spin those numbers) are also lower .

IVF is a hyper competitive market in Australia. Undercut by cheap operators, who were allowed into the market a few years ago, and with strong rivals in the biggest regions of NSW, Queensland and Victoria, there arent many ways to claw back market share or grow without going overseas.

Tasmania and South Australia performed well for Monash in the half, and more women are wanting expensive genetic screening. But even the companys foray into Malaysia delivered bad news as the number of stimulated cycles women undertook fell.

Cynata is trying to cure disease with stem cells. Japanese company Sumitomo tried to buy it for $2-a-share in the half, but they couldnt agree on terms and the talks fizzled.

The company made money in the quarter because FUJIFILM Corporation paid $US3m to exercise a long-awaited licence option for a treatment for graft-versus-host disease (GvHD), a rare condition when donor bone marrow or stem cells attack their new host. However, Cynata made a $2.5m loss and has $5.9m in cash at the end of calendar 2019.

Cynata has three phase two clinical trials expected to start in 2020 for osteoarthritis, critical limb ischemia and GvHD. Its also looking at sepsis, coronary artery disease, and organ transplant rejection.

Another stem cell biotech, Exopharm listed in the prior corresponding half, so its figures are not as simple to compare.

Revenue rose 6,239 per cent to $39,494, although this is entirely from interest on money in the bank, and its loss widened to $3.7m.

The company mainly spent its money on R&D and employees.

The Alzheimers cure researcher has had to dig deep into its data following a spectacular failure of a phase two trial in May.

In the last half, Actinogen found its lead drug Xanamem produced a statistically significant clinical effect on improving cognition in healthy elderly patients at 20mg daily (rather than the lower dose in the phase two trial).

Without a clinical trial underway the loss fell from $7m to $4m, while R&D costs halved.

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February 21st, 2020

BrainStorm Announces Operational Highlights and Financial Results for the Year Ended December 31, 2019 – Yahoo Finance

By daniellenierenberg

Conference Call and Webcast @ 8:00 a.m. Eastern Time Today

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

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

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

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Fourth Quarter Corporate Highlights:

Received notice of US Patent Allowance for NurOwn Cellular Therapeutic Technology Platform

Grant of New Japanese Patent for NurOwn

Presentations at the 30th International Symposium on ALS/MND

Phase 2 Biomarker Data Presentation at NEALS 18th Annual Meeting

Chaim Lebovits as Keynote Speaker at Cell Series UK 2019

Presentation at 7th Annual International Stem Cell Meeting

Presentation at 35th ECTRIMS Congress

Presentation at Neuromuscular Drug Development Summit

Presentation at Dawson James Securities 5th Annual Small Cap Growth Conference

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

Cash, cash equivalents, and short-term bank deposits were approximately $0.6 million as of December 31, 2019.

As of February 14, 2020, we raised gross proceeds of approximately $18.6 million utilizing the ATM facility.

Cash, cash equivalents, and short-term bank deposits as of today were approximately $11.1 million.

In addition, available funding from non-dilutive CIRM and IIA grants amounts to approximately $3.4 million.

Research and development expenses, net, for the year ended December 31, 2019 were $17.2 million, compared to $8.3 million, net for year ended December 31, 2018.

Excluding participation from IIA and CIRM under the grants and proceeds received under the Hospital Exemption regulatory pathway, research and development expenses increased by $8.4 million from $16.3 million for the year ended December 31, 2018 to $24.7 million for the year ended December 31, 2019.

General and administrative expenses for the year ended December 31, 2019 and 2018 were $5.79 million and $5.77 million respectively.

Net loss for the year ended December 31, 2019 was $23.2 million, or ($1.06) per share, as compared to a net loss of $13.9 million or ($0.70) per share for the year ended December 31, 2018.

As of February 14, 2019, the Company had 26,230,839 shares and 4,474,868 warrants issued and outstanding.

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

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

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

Conference ID:

13698896

Toll Free:

1-877-423-9813

Toll/International:

1-201-689-8573

Audio Webcast:

Link to Webcast

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

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

Toll Free:

1-844-512-2921

Toll/International:

1-412-317-6671

Replay Pin Number:

13698896

Replay Start:

Tuesday February 18, 2020, 11:00 AM ET

Replay Expiry:

Tuesday March 3, 2020, 11:59 PM ET

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

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

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

CONTACTS

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

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

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

BRAINSTORM CELL THERAPEUTICS INC.

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

December 31,

2019

2018

U.S. $ in thousands

ASSETS

Current Assets:

Cash and cash equivalents

536

942

Short-term deposit (Note 9)

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February 20th, 2020

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

By daniellenierenberg

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

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

AboutChimerix

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

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

Forward Looking Statements

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

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

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

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

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February 18th, 2020

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

By daniellenierenberg

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

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

Family and friends knew Mike as "Bear."

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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February 18th, 2020

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

By daniellenierenberg

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

Fourth Quarter Corporate Highlights:

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

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

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

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

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

CONTACTS

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

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

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

BRAINSTORM CELL THERAPEUTICS INC.

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

BRAINSTORM CELL THERAPEUTICS INC.

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

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

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February 18th, 2020

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

By daniellenierenberg

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

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

Market Insights:

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

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

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

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

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

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

Key Market Movements:

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

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

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February 18th, 2020

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

By daniellenierenberg

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

First, the two communicated communicated through the registry.

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

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

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

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

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

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

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

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

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

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

See the article here:
News Royal Oak native meets woman she saved with vital stem cell donation Jenn Schanz 11 - WXYZ

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February 17th, 2020

FHSU partners with Be the Match for bone marrow registry event – hays Post

By daniellenierenberg

Brynn Niblock, FHSU junior in pre-med from Hoxie, swabs her cheek as part of the sign up for the Be the Match bone marrow registry Feb. 6 at Gross Memorial Coliseum.

By CRISTINA JANNEYHays Post

Usually Tiger basketball games are a time to have fun cheer the home team, eat some popcorn but students and community members at Feb. 6 game took a few minutes to stop and potentially save a life.

FHSU student health sponsored a Be the Match bone marrow registry drive.

Potential donors ages 18 to 44 answered a list of qualifying health questions on their smartphones and then swabbed their cheeks to be matched with a potential cancer sufferer in need of bone marrow transplants.

Kathy Pyke of Hays knows too the well the importance of the registry. Pyke was at Gross Memorial Coliseum the night of the drive as a volunteer handing out information to potential donors.

Her husband, Tom, was diagnosed with leukemia on March 1, 2014. Family members were tested, and they were not matches. Doctors were also unable to find a bone marrow match on the national registry. There were 6.2 million people in the registry at the time.

In lieu of a bone marrow transplant, Pyke was given donated umbilical cord blood.

Initially the treatment improved Pyke's condition. However, he ultimately died as a result of the disease on Feb. 12, 2015 at the age of 62.

Kathy said the family was rocked by Tom's illness. He was playing golf and went fishing the week before he was diagnosed with cancer.

Kathy said she wishes she could be on the registry to help another family, but her age prevents her from doing so.

"Not only for my husband," she said of the importance of the registry. "I did pray there had been a match. We stayed at the Hope Lodge that was run by the American Cancer Society in Kansas City. There were 45 apartments there and everyone there has someone who has cancer plus a caregiver in it. You just see so many lives being touched. ...

"If this is something that can help somebody, it is just an easy thing to do."

Kathy said she had a good friend who had a family member sign up for the registry, and he was able to donate to someone who had cancer in England.

Pyke said she would also like to see more hospitals participate in the cord blood bank, which is what helped her husband. At the time of Tom's illness, HaysMed was not participating in the umbilical cord blood bank.

Michelle Toogood, BSN, RN, supervisor of Hays Meds Women's andInfant Care Center/NICU, said parents wishing to participate in cord blood donation should initiate the process prior to delivery. HaysMed staff will then aid in the collection of the specimen.

"I just can't express how much people need to do this," Pyke said of signing up for the registry. "It is just so easy to swab test and they could potentially save more than one person's life. It is so easy to do and so important."

If you are identified as a match to someone suffering from cancer, you would be contacted through the registry and asked if you are willing to donate,Amanda McCord, RN at the FHSU student health center.

"Finding the perfect match is essential for people who are fighting this type of cancer," McCord said. "The closer the match the better their chances of remission and beating whatever cancer they are fighting."

There are over 70 diseases that can be treated by bone marrow transplants, according to Be the Match.

Physicians will usually look for matches among relatives first, but only 70 percent of the time are matches made from family members, McCord said.

Statistics also indicate minority patients are less likely to find matches than Caucasian patients. Be the Match is trying to boost minority participation as there are fewer minority participants in the registry at this time, McCord said.

Donating bone marrow is a little bit different for every donor, McCord said.

Most give through a Peripheral Blood Stem Cell (PBSC) donation. A machine draws blood from one arm, extracts the cells it needs, and returns the remaining blood through your other arm, according to the Be the Match website.

Others give through a marrow donation. Liquid marrow is withdrawn from the back of your pelvic bone with a needle. In this case, youll receive anesthesia and feel no pain during the procedure, the Be the Match website said.

According to Be the Match,PBSC donors may experience headaches or body aches several days before collection, but these disappear shortly after donation. Most donors feel completely recovered within a few weeks.

If you missed the Be the Match event at FHSU last week, you can contact Be the Match though its website, and the organization will send you the cheek swab kit to sign up for the registry.

The Be the Match website also has information on the donation process and a link to make monetary donations to the Be the Match program.

Continue reading here:
FHSU partners with Be the Match for bone marrow registry event - hays Post

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February 17th, 2020

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

By daniellenierenberg

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

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

So, his family started making calls.

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

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

Sevack has gained an intense appreciation for donors.

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

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

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

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

A closer look at plans for the new $920M University of Michigan hospital

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

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

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

The search

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

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

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

Mothers breast cancer pushes Ypsilanti woman to get preventative mastectomy

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

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

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

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

The transplant

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

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

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

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

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

The value

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

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

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

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

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

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

Where to donate blood in Washtenaw County during shortage

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

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

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

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February 17th, 2020

50 children diagnosed with cancer in Estonia every year – ERR News

By daniellenierenberg

The youngest children diagnosed with cancer are just one day old. The most frequent type of cancer diagnosed is leukemia, ETV's current affairs show "Aktuaalne kaamera" reported.

"Leukemia is a disease of hematopoietic stem cells; hematopoietic stem cells are the cells which derive from all the cells that circulate in our blood - red blood cells, thrombocytes, white blood cells. Every one of them has a function and if they're not present, they're not able to fill the function," Ain Kaare, Head of the Department of Hematology and Bone Marrow Transplantation in University of Tartu said.

Kaare said that what helps children to cope with cancers is hope and optimism, and they are able to enjoy it, when they are feeling good.

These courageous children make an effort over years in order to get better. Kaare noted that there are children in the clinic who have undergone hematopoietic stem cell transplantation three times in a row, which often adults don't survive.

"In my work office, I have a picture on a wall by a boy who was five at the time, where he has written that "doctor Kaare is my friend". In the picture, there was I with a bag in my hand and in that bag there were supposed to be the hematopoietic stem cells that we transplanted to him," Kaare said.

Kaili Lellep, president of the Estonian Association of Parents of Children with Cancer (Vhihaigete laste vanemate liit) said that a child with a cancer needs a supportive parent around all the time to comfort them and explain everything if necessary, and to get their thoughts away from their illness. Children with cancer often stay in hospital for months.

On Saturday, golden ribbons and the golden light on Tallinn's TV tower and at Tallinn's Children Hospital (Tallinna lastehaigla) sent a message to hospital wards and homes alike, that seriously ill children are not alone and are being thought of, hoping that tomorrow they will be better. The golden ribbon is well-known all over the world.

"This ribbon symbolises support for children with cancer and support for the suffering of children with cancer," Lellep said.

Download the ERR News app for Android and iOS now and never miss an update!

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50 children diagnosed with cancer in Estonia every year - ERR News

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February 17th, 2020

Cord Stem Cell banking Market 2020-2026 Booming With Healthy CAGR || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd,…

By daniellenierenberg

Global Cord stem cell banking market is estimated to reach USD 13.8 billion by 2026 registering a healthy CAGR of 22.4%. The increasing number of parents storing their childs cord blood, acceptance of stem cell therapeutics, high applicability of stem cells are key driver to the market.

Request for FREE sample copy or PDF Herehttps://www.databridgemarketresearch.com/request-a-sample?dbmr=global-cord-stem-cell-banking-market&raksh

Few of the major market competitors currently working in the globalcord stem cell banking marketareCBR Systems, Inc., Cordlife, Cells4Life Group LLP, Cryo-Cell International, Inc., Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord, SMART CELLS PLUS., Cryoviva India, Global Cord Blood Corporation, National Cord Blood Program, Vita 34, ReeLabs Pvt. Ltd., Regrow Biosciences Pvt. Ltd. , ACROBiosystems., Americord Registry LLC., New York Blood Center, Maze Cord Blood, GoodCell., AABB, Stem Cell Cryobank, New England Cryogenic Center, Inc. among others

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

Market Definition: Global Cord Stem Cell Banking Market

Cord stem cells banking is nothing but the storing of the cord blood cell contained in the umbilical cord and placenta of a newborn child. This cord blood contains the stem cells which can be used in future to treat disease such as leukemia, thalassemia, autoimmune diseases, and inherited metabolic disorders, and few others.

Segmentation: Global Cord Stem Cell Banking Market

Cord Stem Cell banking Market : By Storage Type

Cord Stem Cell banking Market : By Product Type

Cord Stem Cell banking Market : By Service Type

Cord Stem Cell banking Market : By Indication

Cord Stem Cell banking Market : By Source

Cord Stem Cell banking Market : By Geography

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Key Developments in the Cord Stem Cell banking Market:

Cord Stem Cell banking Market : Drivers

Cord Stem Cell banking Market : Restraint

Competitive Analysis: Global Cord Stem Cell Banking Market

Global cord stem cell banking market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions and others to increase their footprints in this market. The report includes market shares of cord stem cell banking market for Global, Europe, North America, Asia Pacific, South America and Middle East & Africa.

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

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Cord Stem Cell banking Market 2020-2026 Booming With Healthy CAGR || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd,...

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February 17th, 2020

What are stem cells and bone marrow? – Macmillan Cancer …

By daniellenierenberg

There are two different types of stem cell transplants:

To understand these treatments, it helps to know about bone marrow and stem cells.

Bone marrow is part of our immune system which protects us from infection and disease. It is found inside our bones, mainly in the hip bone and the breast bone. The bone marrow is where stem cells are made.

Stem cells are blood cells at the earliest stage of development. All our blood cells develop from stem cells in the bone marrow. Stem cells stay inside the bone marrow and when they are fully developed they go into the bloodstream.

Blood cells do not live long. The bone marrow normally makes millions of new blood cells every day to replace blood cells as they are needed.

There are three main types of blood cells:

There are two main types of white blood cell. These are called neutrophils and lymphocytes. Neutrophils are the most common. You will hear your doctor or nurse talk about your neutrophil count during your treatment.

Original post:
What are stem cells and bone marrow? - Macmillan Cancer ...

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February 16th, 2020

Bride saves babys life by donating her bone marrow then meets her for the first time as a flower girl at he – The Sun

By daniellenierenberg

WHEN student Hayden Ryals saw a call for blood stem cell donors, she didn't think twice about signing up.

But she was shocked when she received a call just a year later, asking for her help to save a one-year-old girl's life.

Hayden, now 27, from Alabama, had the operation and instantly felt bonded to little Skye.

And two years later, the pair met for the first time, when Skye was invited to be a flower girl at Hayden's wedding.

Speaking exclusively to Fabulous Digital, Hayden tells her story...

Since I was about 16, I have always given blood. It seemed like such a small thing to do to help people. Donating only takes 10 minutes.

Then I went to University in Auburn, Alabama. I was walking to a lecture one day and saw a stall from Be the Match. I wandered over to see what they were about.

The lady at the stall told me they were looking for bone marrow donors. I just needed a swab in my cheek, so they would have my tissue, and then I would be put on the register. You only get called if you're a match.

I happily said yes, it only took two minutes, and didnt think much more about it.

Almost a year to the day later, in April 2016, I got a phone call. It was from Be the Match.

When I spoke to her mum Talia, it was emotional. 'Youve saved my daughters life,' she said through tears

"You have been matched with a one-year-old little girl," I was told.

At first I thought it was a mistake, surely I wouldnt be able to do something so big? But it was true, she needed me to save her life.

I had been feeling so down in life, wondering what I was supposed to be doing, I felt a surge of joy at being able to help someone.

I found out the little girl, Skye Savren-McCormick, wasdiagnosed withjuvenile myelomonocytic leukemia, a rare and aggressive form of blood cancer. She lived in California, miles away from me.

I immediately knew I wanted to help her.

I sent her parents a letter, through a co-ordinator, telling them I wouldnt back out.

They already felt like family to me. It was strange how connected I felt to a little girl I had never met.

In July 2016, we did the transfer. I went to a hospital three hours away in Birmingham Alabama and was put under general anaesthetic for an hour.

They put a hollow needle into my hip bone to extract my bone marrow. It really wasnt bad, and I didnt feel much pain afterwards.

I was told Skye responded well, but had to wait a year to contact Skye's parents.

When I eventually spoke to her mum Talia, it was emotional. "Youve saved my daughters life," she said through tears.

Around this time, I started dating my childhood friend Adrian Ryals, now 34. He was in the US Air Force, and was posted to Korea for a year in 2017.

Just before he left, Adrian popped the question. Delighted, we set a wedding date for June 2018.

In March 2018, I sent Skye a birthday present and asked if she would consider being my flower girl.

Becoming a blood stem cell donor

Every 20 minutes, someone is diagnosed with blood cancer in the UK.

For many, blood stem cell donation is their only chance of survival.

It's difficult to find a match, because of the millions of different stem cell combinations, so the DKMS are constantly on the look out for donors.

To become a blood stem cell donor, you just need to request a kit and do a five-minute cheek swab at home.

You can check your eligibility, and request a kit, here.

The odds are you may never be called upon.

Most blood stem cells are collected through a needle in the arm, just like giving blood.

Bone marrow collection involves stem cells being collected from the back of the hip under general anaesthetic. It takes one to two hours and most donors return to normal activities within a week. This method is only used in 10% of cases.

I knew it was a big ask, she lived a six-hour flight away, but she was so special to me.

If there was a chance she could come, I wanted her at my wedding.

Skye still lived in hospital at the time and had never flown. But in May 2018, the doctor gave her the green light to be able to come to the wedding.

Talia, Skye and her dad Todd flew down the Thursday before the wedding.

As we arrived at the church where we were having the rehearsal, they were already there waiting.

I was breathless and speechless to finally meet this little human, and know I was fortunate enough to have been able to help her.

I just ran up to her, got on my knees and gave her a big hug. She wasnt shy with me, as we had spoken on the phone a few times before.

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Skye didnt really understand what I had done for her, but she knew I was somebody special.

I was so honoured to have Skye be my flower girl, it made the wedding even more beautiful. I feel so lucky to have her in my life.

Earlier this week, we reported on a mother-of-the-bride who demanded her ginger bridesmaid dyed her hair for the wedding - because it "clashed" with her hair.

Read the rest here:
Bride saves babys life by donating her bone marrow then meets her for the first time as a flower girl at he - The Sun

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February 15th, 2020

8-year-old bone marrow recipient and donor celebrate in emotional reunion – WFAA.com

By daniellenierenberg

DALLAS It is more blessed to give than to receive. That's what a Bible verse says. And watching the smiles of a now healthy 8-year-old boy, that's what a recent bone marrow donor will gladly tell you too.

"I felt like the earth underneath my legs was pulled out and like I didn't know what to do," said Anitha Nagilla of Frisco of the 2017 chronic myeloid leukemia diagnosis for her then 6-year-old son Akshaj.

After chemotherapy and other treatments, he would need a bone marrow transplant to regenerate his immune system. But no one in his family, including his older sister, was a close enough match. Also, the likelihood of someone with Asian Indian heritage having a matched, available donor is only 41%.

The Nagilla family moved to north Texas from southern India 13 years ago.

WFAA

Every three minutes, someone is diagnosed with a blood cancer, like leukemia or lymphoma. For many patients, finding a bone marrow donor who is a match is their only chance for a cure. Finding a match can happen anywhere in the world at any time.

In nearby Colleyville, another immigrant, unknowingly, was about to help save the Akshaj's life.

WFAA

"Typical immigration story. Parents moved for a better life for their children and for themselves," said Dr. Prasanthi Ganesa, an oncologist who works at the Center for Cancer and Blood disorders in Fort Worth.

Dr. Ganesa moved to the U.S. as a 10-year-old child, also from southern India. She now works with adult cancer patients.

But as a medical student at Texas A&M, she and a group of friends decided to join the nationalBone Marrow Registry. The donation of blood, stem cells, and bone marrow can help people recover from a variety of cancer-related illnesses.

"At that time it was pretty simple," Dr. Ganesa said of joining the registry approximately 17 years ago. "You fill out some paperwork. It's a (saliva) swab and they have my information. So I've been on it, really not thinking about it, until I got the call. It was a surprise. It was a delightful surprise."

The surprise was that she was a match for a young boy, the same age as her own youngest child. After an outpatient procedure where marrow was extracted from a location on each hip, she went home, then back to work, and waited to hear what happened to whoever it was that received her donation.

Dr. Prasanthi Ganesa

Friday morning at Children's Medical Center in Dallas, she met him face to face.

In a meeting room all decorated for Valentine's Day, they celebrated what is also known as "National Donor Day."

"Hi buddy, how are you?" Dr. Ganesa said as she reached out to hug 8-year-old Akshaj Nagilla. "Oh my goodness," she said as they embraced.

"Definitely is on the list of one of my happiest proudest moments in my life," Dr. Ganesa said. "And Akshaj I have you to thank for that. You know what this means right? We're gonna have to be friends forever," she said as the audience of doctors, nurses, and family and friends laughed and applauded.

Being on the marrow registry is easy. All it takes is a swab of saliva. And minority donors are needed the most. Dr. Ganesa was one of two marrow donors for Akshaj. She supplied the first donation.

Akshaj initially recovered but relapsed in the fall of 2018, when he received a second transplant from a different donor, also of Asian Indian heritage. But his family credits the first donation with starting his long road to recovery.

"She being close here in Dallas is undoubtedly remarkable, in that it happened to our family," Anitha Nagilla said.

"She saved my son's life directly. But she saved some other lives as well in the family," Nagilla said of the impact on her entire extended family. "She is a lifesaver for others as well."

Friday morning at their first meeting, the Nagilla family presented Dr. Ganesa with a gold bracelet and a card with a personal message from Akshaj inside. "I'm doing good and wish you well always", he wrote.

"How wonderful is this," Dr. Ganesa said as they embraced again.

The usually shy 8-year-old also mustered up the courage to climb up to a podium and address the entire crowd, but mostly his message was for Dr. Ganesa.

"I am very thankful because I am still alive," he said.

"I just encourage everybody of Indian origin, of southeast Asian origin, any minority group to get yourself on the registry because it could save a life," Dr. Ganesa said.

"It feels really good to be able to say I gave a part of myself and I saved this person's life. I think it's the ultimate meaning of being a human being. We are here to love all serve all. And what an opportunity that I have had. So I am so grateful that I've had that opportunity," Dr. Ganesa said.

She says she feels like she received more than she gave. As she, her own two sons and the Nagilla family gathered for a group hug, her own extended family maybe just got a lot bigger too.

See more here:
8-year-old bone marrow recipient and donor celebrate in emotional reunion - WFAA.com

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February 15th, 2020

Carl June on CRISPR, CART and how the Vietnam War dropped him into medicine – Endpoints News

By daniellenierenberg

In August of 2011, Carl June and his team published a landmark paper showing their CART treatment had cleared a patient of cancer. A year-to-the-month later, Jennifer Doudna made an even bigger splash when she published the first major CRISPR paper, setting off a decade of intense research and sometimes even more intense public debate over the ethics of what the gene-editing tool could do.

Last week, June, whose CART work was eventually developed by Novartis into Kymriah, published in Sciencethe first US paper showing how the two could be brought together. It was not only one of the first time scientists have combined the groundbreaking tools, but the first peer-reviewed American paper showing how CRISPR could be used in patients.

June used CRISPR to edit the cells of three patients with advanced blood cancer, deleting the traditional T cell receptor and then erasing the PD1 gene, a move designed to unleash the immune cells. The therapy didnt cure the patients, but the cells remained in the body for a median of 9 months, a major hurdle for the therapy.

Endpoints caught up with June about the long road both he and the field took to get here, if the treatment will ever scale up, and where CRISPR and other advancements can lead it.

The interview has been condensed and edited.

Youve spoken in the past about howyou started working in this field in the mid-90s after your wife passed away from cancer. What were some of those early efforts? How did you start?

Well, I graduated from high school and had a low draft number [for the Vietnam War] and was going to go to study engineering at Stanford, but I was drafted and went into the Naval Academy in 1971, and I did that so I wouldnt have to go to the rice fields.

The war ended in 73, 74, so when I graduated in 1975, I was allowed to go to medical school, and then I had a long term commitment to the Navy because they paid for the Acadamy and Medical school. And I was interested in research and at the time, what the Navy cared about was a small scale nuclear disaster like in a submarine, and like what happened at Chernobyl and Fukushima. So they sent me to the Fred Hutchinson Cancer Center where I got trained in cancer, as a medical oncologist. I was going to open a bone marrow transplant center in Bethesda because the Navy wanted one in the event of a nuclear catastrophe.

And then in 1989, the Berlin Wall came down and there was no more Cold War. I had gone back to the Navy in 86 for the transplant center, which never happened, so then I had to work in the lab full time. But in the Navy, all the research has to be about combat and casualty. They care about HIV, so my first papers were on malaria and infectious disease. And the first CAR-T trials were on HIV in the mid-90s.

In 96, my wife got diagnosed with ovarian cancer and she was in remission for 3-4 years. I moved to the University of Pennsylvania in 1999 and started working on cancer because I wasnt allowed to do that with the Navy. My wife was obviously a lot of motivation to do that. She passed away in 2001. Then I started working with David Porter on adoptive transfer T cells.

I got my first grant to do CAR-T cells on HIV in 2004, and I learned a whole lot. I was lucky to have worked on HIV because we did the first trials using lentiviruses, which is an engineered HIV virus.

I was trained in oncology, and then because of the Navy forced to work on HIV. It was actually a blessing in disguise.

So if you hadnt been drafted, you wouldve become an engineer?

Yes. Thats what I was fully intending. My dad was a chemical engineer, my brother is an engineer. Thats what I thought I was going to do. No one in my family was ever a physician. Its one of those many quirks of fate.

Back then, we didnt have these aptitude tests. It was just haphazard. I applied to three schools Berkeley, Stanford and Caltech and I got into all three. It was just luck, fate.

And it turned out when I went to the Naval Academy, they had added a pre-med thing onto the curriculum the year before, so thats what I did when I started, I did chemistry.

I wouldve [otherwise] been in nuclear submarines. The most interesting thing in the Navy then was the nuclear sub technology.

You talked about doing the first CAR-T trials on HIV patients because thats where the funding was. Was it always in your head that this was eventually going to be something for cancer?

So I got out of the Navy in 99 and moved to Penn. I started in 98 working on treating leukemia, and then once I got to Penn, I continued working one day a week on HIV.

Its kind of a Back-to-the-Future thing because now cancer has paved out a path to show that CART cells can work and put down the manufacturing and its going to be a lot cheaper making it for HIV. I still think thats going to happen.

Jim Riley, who used to be a postdoc in my lab, has some spectacular results in monkeys with HIV models. They have a large NIH and NIAID research program.

So were going to see more and more of that. The CAR technology is going to move outside of cancer, and into autoimmune and chronic infections.

I want to jump over to cytotoxic release syndrome (CRS)because a big part of the CRISPR study was that it didnt provoke this potentially deadly adverse effect. When did you first become aware that CRS was going to be a problem?

I mean we saw it in the very first patient we treated but in all honesty, we missed it. Im an MD, but I dont see the patient and David Porter tookcare of the first three patients and our first pediatric patient,Emily Whitehead.

In our first patients, 2 out of 3, had complete remission and there were fevers and it was CRS but we thought it was just an infection, and we treated with antibiotics for 3 weeks and[eventually] it went away. And sort of miraculously he was in remission and is still in remission, 9 years later.

And then when we treated Emily. She was at a 106-degree fever over three days, and there was no infection.

Ive told this story before. My daughter has rheumatoid arthritis, and I had been president of the Clinical Immunologists Society from 2009 to 2010, and the first good drug for juvenile rheumatoid arthritisthat came out. I was invited to give the Japanese scientist Tadamitsu Kishimoto the presidential award for inventing the drug.

Then in 2012, Emily Whitehead was literally dying from CRS, she had multiple organ failures. And her labs came back and IL-6 levels were 1000x normal. It turns out the drug I was looking at for my daughter, it blocks IL-6 levels. I called the physician and I said, listen theres something actionable here, since its in your formulary to give it to her off-label.

And she gave her the appropriate dose for rheumatoid arthritis. It was miraculous. She woke up very rapidly.

Now its co-labeled. When the FDA approvedKymriah, it was co-labeled. It kind of saved the field.

How were you feeling during this time? Did you have any idea what was happening to her?

No, not until we got the cytokine levels, and then it was really clear. The cytokine levels go up and it exactly coincided. Then we retroactively checked out adults and they had adverse reactions and it easy to see. We hadnt been on the lookout because it wasnt in our mouse models.

And it appeared with those who got cured. Its one of the first on-target toxicities seen in cancer, a toxicity that happens when you get better. All the toxicities from chemotherapy are off-target: like leukopenia or hair loss.

I had a physician who had a fever of 106, I saw him on a fever when he was starting to get CRS. When the nurse came in and it said 106, they thought the thermometer must be broken. On Monday, I saw him, and said how are you feeling and he said fine. And I looked at the thermometer and histemperature was still 102.

People will willingly tolerate on-target toxicity thats very different from chemotherapy if they know it helps get them better. Thats a new principle in cancer therapy.

You had these early CART results almost at the same time that Doudna publishes the first CRISPR papers, then still in bacteria. When did you first start thinking about combining the two?

Yeah, it was published inSciencein 2012 and thats when Emily Whitehead got treated. Its an amazing thing.

Thats something so orthogonal. You think how in the heck can that ever benefit CART cells? but my lab had done the first edited cells in patients, published in 2012. And we used zinc-fingered nucleases, which were the predecessors to CRISPR. It knocked out one gene at a time, but we showed it was safe.

I was already into gene editing because it could make T cells resistant to HIV. So it was pretty obvious that there were candidates in T cells that you can knock out. And almost every lab started working on some with CRISPR, cause it was much easier.

We were the first to get full approval by the FDA, so we worked on it from 2012, had all the preclinical data by 2016, and then it takes a while to develop a lot of new assays for this as we were very cautious to optimize safety and it took longer than we wanted, but in the end, we learned a tremendous amount.

So what did we learn?

First of all our patients had advanced metastatic cancer and had had a lot of chemotherapy. The first patient had had 3 bone marrow transplants.

One thing is feasibility: could you really do all the complex engineering? So we found out we could. feasibility was passed.

Another was the fact that cas9 came out of bacteria, forms of strep and staph. Everyone has pre-existing immunity to Cas9 and we had experience from the first trial with Sangamo[with zinc-finger nucleases] where some patients had a very high fever. In that case, we had used adenoviruses, and it turned out our patients had very high levels of baseline immune response to adenoviruses, so we were worried that would happen with CRISPR, and it did not happen.

It did not have any toxicity. If it had, it would have really set the field back. If there was animmune response to cas9 and CRISPR, there couldve been a real barrier to the field.

And then, the cells survived in the patients. The furthest on, it was 9 months. The cells had a very high level of survival. In the previous trials, the cells survived less than 7 days. In our case, the half-life was 85 days. We dont know the mechanism yet.

And we found very big precision in the molecular scissors, and that was a good thing for the field. You could cut 3 different genes on 3 different chromosomes and have such high fidelity.

It [CRISPR] is living up to the hype. Its going to fix all these diseases.

Whats the potential in CAR-T, specifically?

Well theres many many genes that you can add. There are many genes that knocking outwill make the cells work better. We started with the cell receptor. There are many, I think, academics and biotechs doing this now and it should make the cells more potent and less toxic.

And more broadly, what else are you looking at for the future of CART? The week before your paper, there were the results from MD Anderson on natural killer cells.

Different cell types, natural killer cells, stem cells putting CAR molecules into stem cells, macrophages. One of my graduate students started a company to do CAR macrophages and macrophages actually eat tumor cells, as opposed to T cells that punch holes in them.

There will be different cell types and there will be many more ways to edit cells. The prime editing and base editing. All different new variations.

Youve talked about how people used to think the immuno-oncology, if it ever worked, would nevertheless be a boutique treatment. Despite all the advancements, Novartis and Gilead still have not met the sales they once hoped to grab from their CART treatments. Are you confident CART will ever be widely accessible?

Oh yeah, Novartis sales are going up. They had a hiccup launching.

Back in 96 or 97, when Genentech launched Herceptin, their commercial antibody, they couldnt meet the demand either and then they scaled up and learned how to do better cultures. So right now Novartis is using tech invented in my lab in the 1990s culture tech thats complex and requires a lot of labor, so the most expensive part is human labor. A lot can be made robotic. The scale problem will be much easier.

Thats an engineering problem that will become a thing of the past. The manufacturing problem will get a lot cheaper. Here in the US, we have a huge problem with how drugs are priced. We have a problem with pricing. Thats a political issue.

But in cell therapy, its just kind of the growth things you see in a new industry. Itll get worked out.

This article has been updated to reflect that Jim Riley conducted work on CAR in HIV.

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February 15th, 2020

Breakthrough in Stem Cell Research: First Image of Niche Environment | Newsroom – UC Merced University News

By daniellenierenberg

By Lorena Anderson, UC Merced

Professor Joel Spencer and his lab have made a huge breakthrough in stem cell research.

Professor Joel Spencer was a rising star in college soccer and now he is an emerging scientist in the world of biomedical engineering, capturing for the first time an image of a hematopoietic stem cell (HSC) within the bone marrow of a living organism.

Everyone knew black holes existed, but it took until last year to directly capture an image of one due to the complexity of their environment, Spencer said. Its analogous with stem cells in the bone marrow. Until now, our understanding of HSCs has been limited by the inability to directly visualize them in their native environment until now.

This work brings an advancement that will open doors to understanding how these cells work which may lead to better therapeutics for hematologic disorders including cancer.

Understanding how HSCs interact within their local environments might help researchers understand how cancers use this same environment in the bone marrow to evade treatment.

Spencer studied biological sciences at UC Irvine where he was the captain of the mens Division 1 soccer team. He initially planned to pursue a career in professional soccer until faculty mentors opened doors for research and introduced Spencer to biophotonics the science that deals with the interactions of light with biological matter.

UC faculty were a big part of my research experience; they became mentors and friends, Spencer said. My first foray into research was as a lab tech, and that is where I met people who were doing biomedical imaging, and it just caught my wonder.

An image of a stem cell in its natural niche

Spencer left his native California to earn his Ph.D. in bioengineering at Tufts University in Boston and took a postdoctoral research position in the Wellman Center for Photomedicine at Massachusetts General Hospital and Harvard Medical School. In Boston, he learned about live-animal imaging and his wonder became a passion.

Now his emphasis is on biomedical optics: building new microscopes and new imaging techniques to visualize and study biological molecules, cells and tissue in their natural niches in living, fully intact small animals.

I work at the interface of engineering and biology. My lab is seeking to answer biological questions that were impossible until the advancements in technology we have seen in the past couple decades, he said. You need to be able to peer inside an organ inside a live animal and see whats happening as it happens.

Based on work conducted at UC Merced and in Boston, he and his collaborators including his grad student Negar Tehrani visualized stem cells inside the bone marrow of live, intact mice.

He and his collaborators have a new paper published in the journal Nature detailing the work they conducted to study HSCs in their native environment in the bone marrow.

We can see how the cells behave in their native niches and how they respond to injuries or stresses which seems to be connected to the constant process of bone remodeling, Tehrani said. Researchers have been trying to answer questions that have gone unanswered for lack of technology, and they have turned to engineering to solve those puzzles.

Its important for researchers to understand the mechanics of stem cells because of the cells potential to regenerate and repair damaged tissue.

Spencer, left, and students from his lab

Spencer returned to California three years ago, joining the Department of Bioengineering in the School of Engineering at UC Merced. Hes also an affiliate of the Health Sciences Research Institute and the NSF CREST Center for Cellular and Biomolecular Machines . This is his third paper in Nature, but the first stemming from work conducted in his current lab.

He didnt come to UC Merced just because he loves biology Spencer also joined the campus because of the students.

Now Im back in the UC system Im a homegrown UC student whos now faculty, Spencer said. As a student within the system I was able to participate in myriad opportunities, including mentorships that advanced my career. Now I try to encourage graduate and undergrad students to follow their dreams. I love being able to give them opportunities its something I really want to do for the next generation.

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Breakthrough in Stem Cell Research: First Image of Niche Environment | Newsroom - UC Merced University News

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February 14th, 2020

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