Category Archives: Cell Medicine

NEW YORK - BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, will present findings from a multicenter, open label clinical trial of NurOwn in progressive multiple sclerosis. The study, 'Phase 2 Safety and Efficacy Study of Intrathecal MSC-NTF cells in Progressive Multiple Sclerosis,' will be delivered in an oral presentation today at the fully digital 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).

The Phase 2 clinical trial was designed to evaluate intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive MS. The study achieved the primary endpoint of safety and tolerability. It demonstrated a reduction of neuroinflammatory biomarkers and an increase in neuroprotective biomarkers in the cerebrospinal fluid (CSF) and consistent improvement across MS functional outcome measures, including measures of walking, upper extremity function, vision and cognition.

'We were pleased that this study demonstrated safety, preliminary evidence of efficacy and relevant biomarker outcomes in patients with progressive multiple sclerosis, in an area of high unmet need,' said Jeffrey Cohen, M.D., Director of Experimental Therapeutics at the Cleveland Clinic Mellen Center for MS and principal investigator for the trial. 'These results should be confirmed in a randomized placebo-controlled trial.'

The study was sponsored by Brainstorm Cell Therapeutics with additional financial support for biomarker analyses from the National Multiple Sclerosis Society Fast-Forward Program. It was conducted at four U.S. MS centers of excellence: Cleveland Clinic Mellen Center for MS, Cleveland; Icahn School of Medicine at Mount Sinai, New York; Keck School of Medicine of the University of Southern California, Los Angeles; and Stanford University School of Medicine, Palo Alto, Calif.

'We very much appreciate the tremendous collaboration among many premier organizations, for their generous sharing of expertise, support and data, which enabled the important balance between scientific rigor and ethical treatment of progressive MS participants in the trial,' said Ralph Kern, M.D., MHSc., President and Chief Medical Officer, Brainstorm Cell Therapeutics. 'We are holding discussions with key MS experts, and seeking guidance from the FDA to determine next steps for the development of NurOwn in progressive MS.'

'The National MS Society is pleased to support the biomarker portion of this study through our commercial funding program Fast Forward,' said Mark Allegretta, Ph.D., Vice President, Research. 'We're encouraged to see evidence that the biomarker analysis showed proof of concept for detecting neuroprotection and reduced inflammation.'

About the trial

The Phase 2 open-label study evaluated the safety and efficacy of intrathecal administration of autologous MSC-NTF cells in patients with primary or secondary progressive MS. The primary study endpoint was safety and tolerability. Secondary efficacy endpoints included: timed 25-foot walk (T25FW); 9-Hole Peg Test (9-HPT); Low Contrast Letter Acuity (LCLA); Symbol Digit Modalities Test (SDMT); 12 item MS Walking Scale (MSWS-12); as well as cerebrospinal fluid (CSF) and blood biomarkers. Clinical efficacy outcomes were compared with matched (n=48) participants in the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry, Tanuja Chitnis, MD Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases, and 255 patient randomized double blind placebo controlled NN-102 SPRINT-MS Study, courtesy NIH/NINDS, PI: Robert J. Fox, MD, MS, FAAN, Cleveland Clinic, CTR: NCT01982942. Baseline characteristics from these two cohorts were similar allowing for comparison of efficacy results, comparisons with SPRINT-MS were with the placebo arm of this study.

Mean age of participants was 47 years, 56% were female, and mean baseline EDSS score was 5.4. 18 participants were treated, 16 (80%) received all 3 treatments and completed the entire study; 2 study discontinuations were due to procedure-related adverse events. No deaths or treatment-related adverse events due to worsening of MS were observed.

In responder analyses, 14% and 13% of MSC-NTF treated participants showed at least a 25% improvement in T25FW and 9-HPT (combined hands) respectively, compared to 5% and 0% in matched CLIMB patients and 9% and 3% in SPRINT. Twenty-seven percent (27%) showed at least an 8-letter improvement in LCLA (binocular, 2.5% threshold) and 67% showed at least a 3-point improvement in SDMT, compared to 6% and 18% in CLIMB and 13% and 35% in SPRINT, respectively.

Mean improvements of +0.10 ft/sec in T25FW and -0.23 sec in 9-HPT (combined hands), were observed in MSC-NTF treated participants, compared to a mean worsening of -0.07 ft/sec and +0.49 sec in CLIMB and -0.06 ft/sec and +0.28 sec in SPRINT, respectively. MSC-NTF treated participants showed a mean improvement of +3.3 letters in LCLA (binocular, 2.5% threshold) and 3.8 points in SDMT, compared to a mean worsening of -1.07 letters in LCLA (binocular, 2.5% threshold) and mean improvement of +0.10 in SDMT, in CLIMB and -0.6 and -0.1 in SPRINT. In addition the MSFC-4 Composite Z-score of T25W, 9-HPT, SDMT and LCLA showed a 0.18 point improvement in MSC-NTF treated participants, while CLIMB and SPRINT showed decreases of -0.02 and -0.05.

Furthermore, 38% of treated patients showed at least a 10-point improvement in the MSWS-12 a patient reported outcome that evaluates the impact of MS on walking function, whereas this outcome was not evaluated in CLIMB or SPRINT.

CSF biomarkers obtained at 3 consecutive time points, showed increases in neuroprotective molecules (VEGF, HGF, NCAM-1, Follistatin, Fetuin-A) and decreases in neuroinflammatory biomarkers (MCP-1, SDF-1, sCD27 and Osteopontin).

About NurOwn

The NurOwn technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to 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.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website at http://www.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwn manufacturing and clinical development plans, constitute 'forward-looking statements' and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as 'may,' 'should,' 'would,' 'could,' 'will,' 'expect,' 'likely,' 'believe,' 'plan,' 'estimate,' 'predict,' 'potential,' and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwn treatment candidate, the initiation, completion, and success of BrainStorm's 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 BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's 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 at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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Phone: +1 (646) 791-9729

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BrainStorm Cell Therapeutics : Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European...

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/PRNewswire/ --

NRx Pharmaceuticals (Nasdaq: NRXP),

today announced the publication of peer-reviewed results from a prospective, open-label, administratively controlled trial of aviptadil for the treatment of respiratory failure in patients with Critical COVID-19. The study reported 60-day survival in 81% of those treated with aviptadil, compared to 21% survival among those who received standard of care treatment at the Houston Methodist Hospital (P

Journal of Infectious Diseases and Treatment.

"We view this study as supportive evidence that aviptadil protects the lung against the lethal effects of the SARS-CoV-2 virus," said Jihad Georges Youssef, M.D., Lead Author of the study.

Patients enrolled in this study were at the highest possible risk for death based on serious comorbidities that rendered them ineligible for participation in the phase 2b/3 pivotal study of aviptadil for the treatment of COVID-19 with respiratory failure. In addition, patients in the study failed to respond to all treatments approved for COVID-19 during the first surge of the pandemic in the summer of 2020.

In addition to the substantial differences seen for both survival and recovery, the study demonstrated statistically significant advantages in the aviptadil-treated group on two important intermediate endpoints: Respiratory Distress Ratio and Cytokine IL-6. Statistically significant differences on these endpoints have previously been noted in reports from the phase 2b/3 randomized controlled trial of aviptadil vs. placebo conducted at 10 sites across the United States. Aviptadil-treated participants in this open-label study demonstrated a rapid (48-96 hour) 2-fold improvement in the Respiratory Distress Ratio (RDR- a measure of the lung's ability to transmit oxygen to the blood), whereas no short-term improvement was seen in patients who were treated with standard of care (P

"Although randomized, placebo-controlled trials are the gold standard for medical evidence, the findings in this open-label study are remarkably similar to those seen at tertiary care medical centers in the phase 2b/3 trial of aviptadil vs. placebo. Therefore, we view this study as supportive evidence that aviptadil protects the lung against the lethal effects of the SARS-CoV-2 virus," said Jihad Georges Youssef, M.D., Medical Director of Advanced Lung Diseases Program at Houston Methodist J. C. Walter Jr. Transplant Center and lead author on the study. "The patients enrolled in this trial had no therapeutic alternatives and 80% of those treated with standard of care died within 60 days of hospital admission. The corresponding improvement in RDR and cytokine levels add biologic plausibility to the findings. We believe these add important perspective to the potential for aviptadil to help some of the sickest patients with Critical COVID-19 recover and return home to their families."

The primary endpoint was survival as measured by Kaplan Meier life table, with Recovery from Respiratory Failure, World Health Organization 10-point ordinal scale, and PaO2: FiO2 ratio while on a ventilator as secondary endpoints. As required in the CONSORT description, no additional resources were added or removed from the usual care setting other than treatment or non-treatment with aviptadil.

The study team enrolled Standard of Care patients between May 23 and August 15, 2020, in intensive care units (ICU) of the Houston Methodist Hospital System, in Houston, Texas. All patients enrolled in the trial had Critical COVID-19 with respiratory failure. All patients in the study were treated by the same ICU team (regardless of admitting team) and received maximally available therapy, which included steroids, anti-coagulants, remdesivir, and, in some cases, convalescent plasma, with the test group receiving aviptadil.

No unexpected drug-related Serious Adverse Events (SAEs) were recorded. Hypotension was seen in two patients that were successfully managed, and treatment with aviptadil was continued. Diarrhea was observed in 4 aviptadil-treated patients, compared to 3 control patients (19% vs. 10%; p=0.2). These adverse events are congruent with those seen in the Phase 2b/3 randomized clinical trial of aviptadil in Critical COVID-19 patients.

About ZYESAMI (aviptadil) in COVID-19Aviptadil is a synthetic form of Vasoactive Intestinal Polypeptide (VIP), first discovered by the late Prof. Sami Said in 1970, and ZYESAMI is named in his honor. Although primarily concentrated in the lung, it was first purified from the intestinal tract. VIP binds specifically to the alveolar type II cell (ATII) in the air sac (alveolus) of the lung, where it has been shown to have potent anti-inflammatory/anti-cytokine activity in animal models of respiratory distress, acute lung injury, and inflammation. Most importantly, VIP stimulates ATII cells to make the surfactant that must coat the lining of the lungs in order for them to exchange oxygen with the blood.Loss of surfactant causes respiratory failure and alveolar collapse, which are hallmarks of COVID-19.

COVID-19-related respiratory failure is caused by selective infection of the ATII cell by the SARS-CoV-2 virus. The ATII cells are vulnerable because of their (ACE2) surface receptors, which serve as the route of entry for the virus. Coronavirus infection of the ATII cell shuts down surfactant production, triggers the formation of inflammatory cytokines, and causes cell death (cytopathy). VIP is shown to upregulate surfactant production, block Coronavirus replication in the ATII cell, block cytokine synthesis, and prevent viral-induced cell death (cytopathy). Other than ZYESAMI, no currently proposed treatments for COVID-19 specifically target this mechanism of action.

About NRx PharmaceuticalsNRx Pharmaceuticals (NRx) draws upon more than 300 years of collective, scientific, and drug-development experience to bring improved health to patients. Its investigational product, ZYESAMI(aviptadil) for patients with COVID-19, has been granted Fast Track designation by the US Food and Drug Administration (FDA) and is currently undergoing phase 3 trials funded by the US National Institutes of Health, the Biomedical Advanced Research and Development Authority part of the US Department of Health and Human Services, and the Medical Countermeasures program, part of the US Department of Defense. The FDA has additionally granted Breakthrough Therapy Designation, a Special Protocol Agreement, and a Biomarker Letter of Support to NRx for NRX-101, an investigational medicine to treat suicidal bipolar depression. NRX-101 is currently in Phase 3 trials, with readouts expected in 2022. InJuly 2021, the Government ofIsraelawarded NRx the exclusive worldwide right to develop and market the BriLifeCOVID vaccine developed by the Israel Institute for Biological Research.

NRx is led by executives who have held senior roles at Allergan, J&J, Lilly, Novartis, Pfizer, and the US FDA. NRx is chaired byProf Jonathan Javitt, MD, MPH, who has held leadership roles in six biotechnology startup companies with public exits and been appointed to advisory roles in four US Presidential Administrations. The NRx board includes Dr.Sherry Glied, former US Assistant Secretary for Health (ASPE),Daniel E. Troy, JD, former Chief Counsel of the US FDA,Chaim Hurvitz, former director of Teva and President of the Teva International Group, and GeneralH.R. McMaster, Ph.D. (US Army, Ret.) the 26th United States National Security Advisor.

Cautionary Note Regarding Forward-Looking StatementsThis announcement of NRx Pharmaceuticals, Inc. includes "forward-looking statements" within the meaning of the "safe harbor" provisions of the US Private Securities Litigation Reform Act of 1995, which may include, but are not limited to, statements regarding our financial outlook, product development, business prospects, and market and industry trends and conditions, as well as the company's strategies, plans,objectives,and goals. These forward-looking statements are based on current beliefs, expectations, estimates,forecasts,and projections of, as well as assumptions made by, and information currently available to, the company's management.

The company assumes no obligation to revise any forward-looking statement, whetheras a result ofnew information, future events, or otherwise.Accordingly, you should not place reliance on any forward-looking statement, and all forward-looking statements are herein qualified by reference to the cautionary statements set forth above.

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Journal of Infectious Diseases and Treatment Publishes Positive Trial Data of Aviptadil in High Comorbidity Patients Suffering from Critical COVID-19...

SAN DIEGO & ISTANBUL--(BUSINESS WIRE)--Immunicom, Inc., a clinical stage biotech pioneering non-pharmaceutical immunotherapies, progresses with its cancer clinical trial to evaluate the clinical effectiveness of Immunopheresis in partnership with Acibadem University conducted in Acibadem Healthcare Group hospitals. This multi-arm study has been recruiting patients since October 2020 and evaluates Immunicoms oncology platform for non-small cell lung cancer (NSCLC) in combination with Roches pharmaceutical checkpoint inhibitor, Tecentriq (atezolizumab). This is Immunicoms third clinical trial assessing Immunopheresis for solid cancers.

The Immunopheresis therapy employs Immunicoms lead product, the proprietary LW-02 subtractive column, to remove targeted immune-suppressive cytokines that block the natural immune response to attack malignant tumors. Immunopheresis is intended to treat cancer while avoiding the typical side-effects, toxicity, and negative impacts on quality of life that are frequently associated with other cancer treatments.

Immunicoms LW-02 column has received U.S. FDA Breakthrough Device designation for stage IV metastatic cancer and has received European regulatory clearance (CE Mark certification) for use in adults with advanced, refractory, triple negative breast cancer (TNBC).

Lung cancer is one of the most common cancers in the world and the most prevalent cancer in Turkey. Currently available therapies for lung cancer are commonly associated with serious side-effects and often patients tumors become resistant to even the newer immunotherapies. Moreover, the presently available immunotherapies are prohibitively expensive which precludes this option for many patients, said Ahmet ahin, MD., Prof., Chairman of the Medical Executive Board of Acibadem Healthcare Group. We are highly optimistic that Immunicoms Immunopheresis therapy will provide physicians with a novel breakthrough cancer immunotherapy that should be markedly better tolerated, improve patients quality of life, and its cost-effectiveness will make it widely available to many patients who might otherwise not be able to benefit from an immunotherapy.

The multicenter, multi-arm clinical study led by Gkhan Demir, MD, Prof., Acibadem University School of Medicine, Department of Internal Diseases Medical Oncology who serves as the Principal Investigator, and conducted by Mustafa Bozkurt, MD tests the clinical effectiveness of the LW-02 Immunopheresis column to treat stage IIIB/IV non-small cell lung cancer patients whose disease has progressed after at least one first-line chemotherapy round of treatment. The clinical study is divided into three treatment arms, each with the LW-02 column; the first in combination with Tecentriq (atezolizumab), the second in combination with Taxol (paclitaxel), and the third with Immunopheresis as a monotherapy for patients who have disease progression after a second-line of chemotherapy treatment.

With this clinical trial, Immunicom and Acibadem are centered on demonstrating the effectiveness of Immunicoms Immunopheresis therapy for treating patients facing a highly aggressive form of non-small cell lung cancer. This important study assesses Immunopheresis in combination with both Roches Tecentriq, one of the worlds leading PD-L1 checkpoint inhibitor immunotherapy drugs, and with paclitaxel, a very commonly used chemotherapy agent, said Amir Jafri, Founder and CEO of Immunicom, Inc.

We have developed Immunopheresis intending that it could be used with many different oncology regimens and the growing array of promising immunotherapy drugs, while keeping in the forefront our mission of preserving patients quality of life to ensure that they can fight cancer in a compassionate and dignified way. We are confident that our breakthrough Immunopheresis technology will provide patients the benefits associated with upregulation of their immune system to attack resistant cancers. Immunicoms motto of Healing Reimagined, reflects our mission to improve quality-of-life for all patients in a meaningful way and expedite their path to a healthy recovery, continued Mr. Jafri.

For an overview of how this breakthrough technology works, visit https://www.immunicom.com/how-it-works. Immunopheresis is an investigational therapy that has not yet been approved for use by the FDA.

About Acibadem University

Acibadem University was founded in 2007 dedicated to the field of health sciences.By using dynamic and contemporary educational programs, a strong academic teaching team trains healthcare students to be future healthcare professionals who continually research innovations in all fields of medical science. With the mission of being a strongly research oriented university, the research laboratories are fitted out with the latest state of the art equipment, designed to complement the life sciences and biotechnology fields.The Clinical Simulation and Advanced Endoscopic - Robotic Surgery Training Center - CASE is one of the most comprehensive medical training centers in the world with its accommodation of multiple departments and advanced technological infrastructure. CASE received the Center of Excellence certificate awarded by the CAE Academy to only two medical training centers worldwide. The Center is accredited by the Network of Accredited Clinical Skills Centers of Europe (NASCE) and the Society for Simulation in Healthcare (SSH), providing pre- and post-graduate training.Acibadem University offers its students the opportunity to study abroad at esteemed partner universities under the Erasmus Programme having bilateral agreements with Europes most notable universities. With 100,000 square meters of in-closed area centrally located on the Asian side of Istanbul, the Kerem Aydnlar Campus is replete with high-technology equipment and offers students a privileged university life.

About Acibadem Healthcare Group

Acibadem Healthcare Group, a world brand in health sector, offers services in 21 hospitals and 13 medical centers in 4 countries, namely, Macedonia, Bulgaria and The Netherlands, besides Turkey. In addition to hospitals and medical centers, Acibadem represents Turkey as a trustworthy health brand in all over the world, with its 36 representative offices called Health Points, which are located in 22 countries and 36 cities. Acibadem brand is associated with highest standards of care and technology in the region. Today the group has reached a total capacity of 3,164 beds and 141 operating theaters with 22 thousand employees, including 4,000 physicians and 4,200 nurses working under its roof. Acibadems rapid growth led to the signing of a partnership with IHH Healthcare Berhad, Asias largest healthcare chain, in 2012 which enabled us to be a part of worlds second largest healthcare company globally by its market capitalization.

About Immunicom

Immunicom, Inc. creates novel immunotherapies designed to treat a variety of diseases using its breakthrough Immunopheresis technology platform. The privately held medical technology company develops innovative, non-pharmaceutical approaches for treating cancer, autoimmune disease, and inflammatory and renal diseases. Immunicoms revolutionary blood-filtering Immunopheresis technology has the potential to effectively treat a wide variety of cancer types, including those that have not responded to other treatment strategies, with possibly fewer side effects. Immunicoms lead product, the LW-02 column, has received U.S. FDA Breakthrough Device designation for stage IV metastatic cancer and European regulatory clearance (CE Mark Certification) for use in adults with advanced, refractory, triple negative breast cancer (TNBC). Immunopheresis is currently being evaluated in several global oncology trials for multiple cancers. Immunicom is headquartered in San Diego, California with operations in Houston, Texas, Philadelphia, Pennsylvania and Krakow, Poland.

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Immunicom Continues Successful Immunopheresis Clinical Trial in Advanced, Non-Small Cell Lung Cancer Treatment with Acibadem University - Business...

Too many exhausted T cells left in the wake of aggressive chemotherapy regimens for patients with advanced chronic lymphocytic leukemia (CLL) make it more challenging for chimeric antigen receptor (CAR) T cell therapy to do its job. Now, a new study from researchers in the Perelman School of Medicine at the University of Pennsylvania shows how to overcome this type of resistance and reinvigorate these T cells with an experimental small molecule inhibitor.

Reporting online in the Journal of Clinical Investigation, the team shows how the drug, known as JQ1, improved CAR T cell function by inhibiting what is known as the bromodomain and extra terminal (BET) proteins. BET, the researchers showed, can disrupt CAR expression and key acetylated histone functions in T cells in CLL.

The findings demonstrate, for the first time, this mechanism of resistance and present a much-needed target for CLL when treating patients with cellular therapies like CAR. Only a small subset of patients with advanced CLL respond to CAR T cell therapycompared to 80 percent of acute lymphocytic leukemia patients with advanced disease.

Why CAR T cells fail to fully attack cancer cells in so many CLL patients is an important question that needs to be answered in order to expand the use of these immunotherapies in CLL and other cancers, said senior author Joseph A. Fraietta, PhD, an assistant professor of Microbiology at Penn, and member of the Center for Cellular Immunotherapies. Treating these war weary T cells during the CAR T cell engineering process has the potential to boost responses, weve shown here. Its setting the stage for a very promising set of next steps that rationalize further studies, including clinical trials, to prove this approach is safe and feasible.

Using the small molecule inhibitor and the T cells and CD19 CAR T cells from multiple previously treated patients, the researchers demonstrated that the BET protein plays a role in downregulating CAR expression, and that, if blocked, can diminish CAR cell T cell exhaustion and increase the production of CAR T cells from CLL patients with poor lymphocytes.

Treatment with JQ1 also increased levels of various immunoregulatory cytokines and chemokines previously reported to be produced by CAR T cells in CLL during successful therapy. The array of native immune and CAR cells mirrored those found more typically in patients who do respond.

Given this observed reinvigoration of dysfunctional CLL patient CAR T cells by BET inhibition, the authors suggest that incorporating JQ1 into cellular engineering and expansion processes could lead to a generation of less defective and more potent final CAR T cells for patients.

To what extent the above pathways contribute to the effects of JQ1 on CAR T cells is a focus of ongoing investigations for the research group.

This work shows us that T cells can be taught new tricks, said Bruce Levine, PhD, the Barbara and Edward Netter Professor in Cancer Gene Therapy in Penns Perelman School of Medicine, and co-author on the study. That is to say that the methods of manufacturing can be adapted to improve CAR T cell function, so that what would have been exhausted or dysfunctional cells can now be reinvigorated, and potentially lead to better clinical responses in more patients than before.

Reference:Kong W, Dimitri A, Wang W, et al. BET bromodomain protein inhibition reverses chimeric antigen receptor extinction and reinvigorates exhausted T cells in chronic lymphocytic leukemia. J Clin Invest. 2021;131(16). doi:10.1172/JCI145459

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Investigational Inhibitor Improves Responses to Cellular Therapies in Advanced Leukemias - Technology Networks

The addition of zanubrutinib to the guidelines was based on the findings from the ASPEN studypublished in Blood. The study compared the safety and efficacy of zanubrutinib, a novel highly selective Bruton tyrosine kinase (BTK) inhibitor, with ibrutinib, a first-generation BTK inhibitor.2

In ASPEN, 28% of patients on zanubrutinib and 19% on ibrutinib achieved a very good partialresponse (VGPR); no patient achieved a complete response (CR). The study did not meet the primary end point, which was proportion of patients achieving CR or VGPR, but the investigators noted that there was a trend toward better disease control for zanubrutinib vs ibrutinib.

Major response rate was similar (77% for zanubrutinib vs 78% for ibrutinib), as was progression-freesurvival (85% for zanubrutinib vs 84% for ibrutinib). Patients on zanubrutinib had fewer instancesof atrial fibrillation, contusion, diarrhea, peripheral edema, hemorrhage, muscle spasms, andpneumonia, as well as adverse events that led to treatment discontinuation.

This study established that zanubrutinib is highly effective in the treatment of WM; zanubrutinibis associated with important safety advantages, especially with respect to cardiovascular toxicity,the authors wrote.

The data from ASPEN was used in a supplemental new drug application (sNDA) with the FDA forzanubrutinib in the treatment of WM, which was submitted in February 2021. Health Canada approved zanubrutinib in WM in March based on the findings of ASPEN.

We are pleased that the FDA has accepted the sNDA for Brukinsa in WM, a rare disease with significant morbidity, Jane Huang, MD, chief medical officer, Hematology, BeiGene, said in a statement. BTK inhibitors have transformed the treatment of WM in recent years, but discrepancies in response exist in patients with different subtypes, and toxicity can remain an issue.3

References1. NCCN. Clinical Practice Guidelines in Oncology. Version 1.2022. Waldenstrm macroglobulinemia/lymphoplasmacytic lymphoma. https://www.nccn.org/guidelines/guidelines-detail?category=1&id=14752. 2. Tam CS, OPat S, DSa S, et al. A randomized phase 3 trial of zanubrutinib vs ibrutinib in symptomatic Waldenstrm macroglobulinemia: the ASPEN study.Blood. 2020;136:2038-2050. doi:10.1182/blood.20200068443. BeiGene announces US FDA acceptance of supplemental New Drug Application for Brukinsa (zanubrutinib) in Waldenstrms Macroglobulinemia. News release. BeiGene. Published February 17,2021. Accessed July 20, 2021. https://ir.beigene.com/news-releases/news-release-details/beigene-announces-us-fda-acceptancesupplemental-new-drug

With more treatment options needed in glioblastoma multiforme (GBM) to successfully attack disease, attention has been paid to chimeric antigen receptor (CAR) T-cell therapy, which hasshown great promise in hematologic malignancies and is being studied in other settings, includingcentral nervous system solid tumors, like GBM. In a recent paper, investigators provide a look atclinical trials currently assessing the potential of treatment for the disease.

Although theres hope that CAR T-cell therapy can revolutionize the way GBM is treated, there isstill more research to be done, investigators write inCancer Medicine, noting that research on CART-cell therapy in this setting is still in early stages.1

Despite the promising results of CAR T technology in hematological malignancies, CAR T cellsfor GBM are still in their earlier stage, wrote the investigators. Several GBM-associated antigenshave served as the targets of ongoing clinical trials (EGFRvIII, NKG2D, B7-H3, CD147, IL13Ralpha2,and HER2). Most of the current clinical trials are still in phase 1, testing the safety and efficacy ofmostly second-generation CAR T cells constructed with CD28 and 4-1BB costimulatory intracellulardomains.

The investigators write that, to date, evidence suggests combined therapy is more effective thanmonotherapy in GBM, which not only gives insight into how to enhance the efficacy of CAR T-celltherapy but also to potentially curtail adverse effects. The investigators outlined various ongoingtrials of CAR T-cell therapy that combine the therapy with other agents in patients with GBM.

With 70% of patients with GBM expressing B7-H3, which is not expressed on normal tissues, 1 ongoing trial is currently assessing the safety and tolerability of B7-H3 CAR T-cell therapy withtemozolomide, with B7-H3 CAR T being injected between temozolomide cycles. Data from the study are expected in 2022.2

EGFRvIII CAR T-cell therapy is being studied in several trials, with an ongoing phase 1 studyassessing the therapy in combination with temozolomide and another assessing EGFRvIIICAR T following radiosurgery in patients with recurrent GBM.

CAR T-cell therapy in combination with immune checkpoint inhibitors is also being assessed for both recurrent and resistant disease, say the investigators. A phase 1 trial is currently looking at the safety and efficacy of IL13Ralpha2-CRT T cells used alone or in combination with nivolumab and ipilimumab.

Although still in the preclinical stage, Fc-CRs T cells may prove to be a leader in the treatment ofGBM and other solid tumors, say the investigators. As research on CAR T-cell therapy in GBMprogresses, the investigators are also calling attention to the challenges facing the therapy inthe disease, including the highly unstable tumor microenvironment and the variable genetic natureof the disease.

The mechanism by which the apparent tumor responses or growth delay in CAR-T cell-treatedGBM are multifactorial. This cannot be attributed to the therapy but could instead result from differences in the natural history of disease between patients, commented the investigators. Although there are no direct ways to demonstrate the actual killing of tumor cells by CAR T in situ, previous clinical and preclinical data suggest that CAR-T-EGFRvIII cells induce their action by antigen-directed cytolysis after crossing the bloodbrain barrier.

References

1. Marei HE, Althani A, Afifi N, et al. Current progress in chimeric antigen receptor T cell therapy for glioblastoma multiforme.Cancer Med. Published online June 19, 2021. doi:10.1002/cam4.4064

2. Pilot Study of B7-H3 CAR-T in Treating Patients With Recurrent and Refractory Glioblastoma. Updated May 12, 2020. Accessed July 15, 2021. https://clinicaltrials.gov/ct2/show/NCT04385173.

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August 2021: Targeted Therapy & Immuno-Oncology - AJMC.com Managed Markets Network

WASHINGTON, Aug. 16, 2021 /PRNewswire/ --The ASH Research Collaborative (ASH RC) has announced the first 10 clinical research consortia to join the ASH RC Sickle Cell Disease Clinical Trials Network. The sites will be able to enroll children and adults living with sickle cell disease (SCD) within their patient populations in clinical trials as part of an unprecedented national effort to streamline operations and facilitate data sharing to expedite the development of new treatments for this disease.

SCD is a chronic, progressive, life-threatening, inherited blood disorder that affects more than 100,000 Americans and an estimated 100 million persons worldwide. Clinical trials hold incredible promise for the development of much-needed new treatments, and possibly even a cure. While there are currently only four U.S. Food and Drug Administration (FDA)-approved drugs to treat the disease, there is a robust SCD drug development pipeline that will drive demand for clinical trials to a new level, providing a prime opportunity to advance treatment and care of those affected by SCD.

The SCD Clinical Trials Network is poised to accelerate progress by bringing together a community of research-ready clinical sites and experienced investigators, connecting industry sponsors to sites and the community, leveraging real world data to support clinical trials through the ASH RC Data Hub, and facilitating efficient, coordinated clinical trial startup. Engagement with the community has been foundational to the development of the SCD Clinical Trials Network. The ASH RC provides support for SCD Community Advisory Boards at each consortium to ensure that Network efforts are informed by the needs and desires of those living with and caring for those with SCD at both local and national levels.

"We're entering a new era for how clinical trials are designed and conducted, and the ASH Research Collaborative SCD Clinical Trials Network is committed to ensuring that the voice of the SCD community is heard at every stage of the process, while ensuring a coordinated, efficient approach. We hope to accelerate our ability to bring new treatments to individuals who are living with this difficult disease," said Martin S. Tallman, MD, of Memorial Sloan Kettering Cancer Center, who serves as president of the ASH Research Collaborative and the American Society of Hematology.

Each consortium in the Network consists of a clinical trials unit (CTU) that serves as the lead clinical trial site, or hub, providing administrative expertise and infrastructure to support various clinical research sites (CRS), or spokes, within each consortium (see full list below). A CRS can be a hospital, outpatient clinic, community health center, private practice, or local health department clinic. Approximately 24,000 children and adults living with sickle cell disease are currently represented by the Network.

"The ASH RC Sickle Cell Disease Clinical Trials Network is uniquely situated to accelerate clinical research and the development of novel therapeutics to help those living with sickle cell disease," said Charles S. Abrams, MD, Chair of the ASH RC SCD Clinical Trials Network Oversight Committee. "This an unprecedented opportunity to make a profound difference in the lives of people with sickle cell disease. We are committed to partnering with patients, families, and the broader sickle cell disease community."

Inclusiveness, commitment to rigorous standards, and improving the access of the SCD community to clinical trials are guidingprinciples of the SCD Clinical Trials Network. In addition to the ten consortia that have already been activated, additional network sites are in the onboarding process. Additions to the SCD Network will continue to be announced on a rolling basis.

The ASH RC Sickle Cell Disease Clinical Trials Network Sites:

Central Carolinas Clinical Trials CollaborationClinical Trials Unit: Duke University Medical Center (Adult & Pediatrics)Clinical Research Sites: UNC Medical Center (Adult & Pediatrics; Wake Forest Baptist Health (Adult & Pediatrics)

Chicagoland Regional Research in Sickle Cell Partnership (CRiSP) Clinical Trials Unit: Ann & Robert H. Lurie Children's Hospital of Chicago Clinical Research Sites:Indiana Hemostasis and Thrombosis Center; Northwestern Memorial Hospital; OSF Healthcare Children's Hospital of Illinois; University of Illinois at Chicago; University of Chicago (University Hospital; Comer Children's Hospital; LaRabida Children's Hospital)

DMV Sickle Cell Disease Consortium (DMVSCDC) Clinical Trials Unit: Children's National Health SystemClinical Research Sites:Howard University; Kaiser Permanente Mid-Atlantic Permanente Medical Group (MAPMG) and Mid Atlantic Permanente Research Institute (MAPRI); Medstar Washington Hospital Center; Pediatric Specialists of Virginia; Virginia Commonwealth University/Children's Hospital of Richmond

The Heartland/Southwest SCD Consortium Clinical Trials Unit: Washington University School of Medicine Clinical Research Sites:Children's Mercy of Kansas City; University of Arkansas for Medical Sciences; University of Iowa Medical Center; University of Missouri Health Center; University of Oklahoma Health Sciences Center

New York Sickle Cell CTN Consortium Clinical Trials Unit: Montefiore Medical Center Clinical Research Sites:Brookdale Hospital Medical Center; Columbia University Medical Center; Cohen Children's Medical Center; Cornell Medical Center; Interfaith Medical Center; Methodist Hospital; Mount Sinai Hospital

North Texas Clinical Trials Consortium Clinical Trials Unit: UT Southwestern Medical Center Clinical Research Sites:Children's Medical Center Dallas; Clements University Hospital; Cook Children's Health Care System; Medical City Children's Hospital, Dallas; Parkland Health and Hospital System

SCD Clinical and Research Consortium of Southeastern WisconsinClinical Trials Unit:Medical College of WisconsinClinical Research Sites: Children's Hospital of Wisconsin; Froedtert Hospital

Sickle Mid Atlantic Research Team (SMART) Clinical Trials Unit: The Johns Hopkins University School of MedicineClinical Research Sites:Charleston Area Medical Center (CAMC); Christiana Care Health Services; Herman & Walter Samuelson Children's Hospital at Sinai Hospital; Inova Health System (Fairfax); Johns Hopkins All Children's Hospital- FL; Nemours / Alfred I DuPont Hospital for Children; Peninsula Regional Medical Center; University of Maryland Medical Center

South Carolina Sickle Cell Disease Network Clinical Trials Unit: Prisma Health Children's Hospital - UpstateClinical Research Sites:Medical University of South Carolina Lifespan Comprehensive SCD Center; Prisma Health Children's Hospital - Midlands

Western States Sickle Cell Disease Clinical Trials Network (WeSt SCD CTN) Clinical Trials Unit: University of California San Francisco Benioff Children's Hospital of Oakland Clinical Research Sites:UCSF Helen Diller Medical Center at Parnassus; UCSF Zuckerberg San Francisco General Hospital; University of California Davis Medical Center

More Information:

About the ASH Research CollaborativeThe ASH Research Collaborative (ASH RC) is a non-profit organization established by the American Society of Hematology (ASH)to improve the lives of people affected by blood diseases by fostering collaborative partnerships to accelerate progress in hematology. The foundation of the ASH RC is its Data Hub and Clinical Trials Network. TheData Hub, a technology platform that facilitates the exchange of information by aggregating and sharing research-grade data on hematologic diseases. The Sickle Cell Disease Clinical Trials Network (CTN) optimizesthe conduct of clinical trials research in sickle cell disease (SCD) and leverages the Data Hub to collect key information and identify gaps to advance SCD research and treatment.

SOURCE American Society of Hematology

http://www.hematology.org

Link:
ASH Research Collaborative Announces First Ten Clinical Trial Consortia to Join the Sickle Cell Disease Clinical Trials Network - PRNewswire

reproduction

Living for many years with quality of life, with health, without disease, is the dream of any human being. As far as science and technological progress depend, this is the near future. The trend is global and Brazil can be proud to be at the forefront of this movement.

CLICK HERE AND GET NEWS IN OUR WHATSAPP AND TELEGRAM GROUPS

In this scientific field, the highlight is the research on the use of mesenchymal stem cells, which function in the regeneration of many tissues of the human body, and the release of chemicals that facilitate the regeneration process and that modulate the immune system. These cells are multipotent, as they have the potential to form different tissues.

Mesenchymal stem cells, especially those taken from baby teeth, have a high quality because they are so small. They have the ability to help regenerate different types of tissues and organs, such as: skin, pancreas, cartilage, nervous tissue, fatty tissue, bone, heart tissue, liver, teeth, eyes, and muscles. In addition, the improvement in the quality of life or even the treatment of chronic diseases or diseases that are now considered incurable such as Parkinsons disease, the consequences of stroke and even spinal cord injuries are getting closer.

With this important discovery, people will be able to live longer and better, using their own materials to regenerate any type of tissue, eliminating the risk of rejection. Currently, for example, we are having tremendous success in the experimental treatment of children with a cleft lip, which is conventionally corrected with highly invasive surgery, which occurs around 8 years of age and consists in removing part of the hip bone to bridge the gap, an extremely painful procedure with slow recovery. On average, up to three surgeries are needed. With stem cell therapy, bone tissue is formed, and within six months, the cleft lip and palate are completely closed. This is just an example of what is happening and what lies ahead. More effective, less painful treatments and better results are becoming a reality, says scientist Jose Ricardo Muniz Ferreira, who has studied and improved the technology of extracting cells from baby teeth, storing them and implanting them. A true treasure: young, highly versatile stem cells, he asserts.

Ferreira is the president of R-Crio a Brazilian cell treatment center with stakeholders from various fields such as: dentistry, biology, biomedicine, medicine and engineering which, despite the unstable economic scenario that our country is going through, decided to invest heavily because they know the benefits that can That this area brings to all residents. According to him, Brazil is far ahead in this issue compared to other countries. In our travels to universities around the world, such as England and India, where I have spoken recently, I have spoken a lot with many colleagues and have been able to see Brazil at the forefront in this sector, he celebrates. He also says that patent production by Brazilian researchers is increasing in this sector, making our country a reference in this matter. We are no longer the stigmatized country of selling goods and we are increasingly moving towards the production of intellectual property, the generation of business and jobs and the promotion of the economy and health, he explains.

According to the scientist, public health in our country tends to gain a lot from regenerative medicine. In addition to the population health gain, the government will save significantly by using it.

Recently, the National Health Surveillance Agency ANVISA, through Resolution 214 and Public Consultation 416, is working in favor of regulating good research practices in this area, strengthening work with stem cells.

It is up to the press and the community to spread this good news! It has been and will remain increasingly constant and important in peoples lives, increasing expectations and quality of life, concludes scientist Jos Ricardo Muniz Ferreira.CLICK HERE AND GET NEWS IN OUR WHATSAPP AND TELEGRAM GROUPS

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Medical developments in the near future - BOB fm

From left Evanthia Roussos Torres, Elizabeth Jaffee, Roisin Connolly and Vered StearnsImage Courtesy of Johns Hopkins Kimmel Cancer Center

Mega Doctor NEWS

byJohns Hopkins Medicine

Newswise Combining a histone deacetylase inhibitor drug with immunotherapy agents is safe, and may benefit some patients with advanced cancers that have not responded to traditional therapy, according to results of a phase 1 clinical trial led by researchers at theJohns Hopkins Kimmel Cancer Center,Bloomberg~Kimmel Institute for Cancer Immunotherapyand several other centers including University of Pittsburgh Cancer Institute, Yale Cancer Center and City of Hope in Los Angeles, which participated in study enrollment, and the University of Southern California and University College Cork in Ireland, which collaborated on analysis of the data.

During the study, 33 patients with advanced solid tumors received the histone deacetylase inhibitor drug entinostat for two weeks. Then, some patients received the immunotherapy agent nivolumab, a checkpoint inhibitor, in combination with the entinostat, while others received both nivolumab and a second checkpoint inhibitor agent, ipilimumab, after the initial dosing with entinostat.

Checkpoint inhibitors, such as nivolumab and ipilimumab, release important brakes that permit the immune system to fight the cancer cells. The epigenetic inhibitor drug entinostat has been shown in preclinical models and laboratory studies (which led to this current clinical trial) to drive changes in the chemical environment of cells to make them more amenable to immunotherapy, saysVered Stearns, M.D., director of the Womens Malignancies Disease Group at the Johns Hopkins Kimmel Cancer Center.

The authors found that the objective response rate, or percentage of patients whose cancer responded to therapy, was 16%. One patient with triple-negative breast cancer had a complete response, meaning a total shrinkage of the cancer. Because the cancer types included in the study are not felt to have high response rates to immune checkpoint therapy, these preliminary results are very promising. Treatment-related adverse events were mostly low grade, and included fatigue, nausea, anemia and diarrhea.

These results were published online in the June 16 issue ofClinical Cancer Research.

Study co-author Evanthia T. Roussos Torres, M.D., Ph.D., an adjunct professor at Johns Hopkins and assistant professor at the University of Southern Californias Keck School of Medicine and oncologist and researcher with USCs Norris Comprehensive Cancer Center in Los Angeles, says the entinostat functioned as an immune system-priming agent prior to using immunotherapy in solid tumors that traditionally are nonresponsive to checkpoint inhibition therapy.

One of the major findings of our study is that this is a safe and tolerable combination of therapies, she says. There arent very many trials investigating dual immune checkpoint inhibition in combination with other novel therapeutics. We determined a safe, tolerable dose with this combination that had very few adverse effects. That is significant.

The researchers collected blood and tumor samples from the 33 patients before and after the two weeks of entinostat to assess the impact of the entinostat on the immune system, and also after the addition of the immune checkpoint agents to evaluate the effect of each of the treatments on the immune environment. The study team found a significant increase in the ratio of CD8/FoxP3 gene expression in tumors following checkpoint inhibitor treatment but not after entinostat treatment alone. This suggests that the combination therapy helped increase both cytotoxic immune system T cells immune cells that directly kill cancer cell as well as decrease immune system T regulatory cells immune cells that modulate the immune response which equaled a stronger immune response, Roussos Torres says.

The median age of participants was 60, most (91%) were female, 30% had breast cancer (the main tumor type studied) and the median number of prior regimens tried for their disease was 3.5. The median progression-free survival, or time until disease progression or death, was 6.1 months, and median overall survival was 10.6 months. Stable disease, considered the best response, was observed in 44% of participants. Clinical benefit rate, or the percentage of patients who achieved stable disease, or partial or complete response, was 60%.

The most robust increases in CD8/FoxP3 ratio were observed in two patients with HR-positive breast cancer who experienced a partial response and stable disease. Three additional patients who experienced stable disease had adenocarcinoma, a cancer of glandular tissue.

The complete response in a patient with breast cancer is a promise that there may be a role for this combination of therapies in breast cancers, says senior study author Roisin M. Connolly, M.B.B.Ch., M.D., an adjunct professor at Johns Hopkins and chair in cancer research at University College Cork and Cork University Hospital in Ireland. Immunotherapy hasnt been as big a blockbuster to date in advanced breast cancer as in other cancers, such as lung cancers or melanoma. This study paves the way for novel combination therapies that have the potential to improve response rates to immune checkpoint inhibitors in traditionally less-responsive tumor types.

This drug combination is being investigated further in a group of 24 patients with HER2-negative breast cancer as part of the same NCI-sponsored clinical trial. We are excited to have completed accrual to these ongoing studies in advanced HER2-negative breast cancer, and expect to report on these findings later this year, says Stearns.

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Inhibitor Drug Entinostat Primes the Body to Better Respond to Anti-Cancer Treatment with Immunotherapy - Mega Doctor News

CARLSBAD, Calif.--(BUSINESS WIRE)--Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today provided an update on the clinical advancement of OPC1, its investigational allogeneic oligodendrocyte progenitor cell (OPC) transplant therapy for the treatment of spinal cord injury (SCI). Following feedback received from an interaction held with the U.S. Food and Drug Administration (FDA) last week under the FDAs Regenerative Medicine Advanced Therapy (RMAT) program, Lineage intends to submit an amendment to its Investigational New Drug application (IND) for OPC1 to support a Phase 1 clinical study to evaluate the safety and performance of Neurgain Technologies Inc.s Parenchymal Spinal Delivery System (Neurgain PSD system) to deliver OPC1 cells to the spinal cord. In February, the Company entered into an exclusive option and license agreement with Neurgain to evaluate its novel PSD system in both preclinical and clinical settings. The IND amendment is expected to be submitted to the FDA in the fourth quarter of 2021. The data from the Phase 1 clinical study is intended to validate the Neurgain PSD system for use in a late-stage clinical study, expected to begin in 2022 following the completion of the Phase 1 study.

It is a privilege to report that our novel OPC1 program will be returning to clinical testing earlier than anticipated. There currently are few opportunities for SCI patients to participate in clinical trials, so we are excited to re-engage with these patients and their advocacy community as part of our efforts to improve outcomes for individuals with this debilitating condition, for which there are no FDA-approved treatments, stated Brian M. Culley, Lineages CEO. In the past 18 months, we have significantly increased the purity and production scale of the OPC1 cells utilized in a prior clinical study. This improved production process has been transferred to our in-house Current Good Manufacturing Practice (cGMP) suite and will support production of clinical study material for later-stage clinical work. In parallel, we are finalizing plans to test the safety of the Neurgain PSD system to deliver OPC1 in SCI patients. We believe this device can improve the ease and precision of delivering our cells to the spinal parenchyma. As an added benefit, based on feedback from the FDA, in addition to patients with subacute SCI, we anticipate that patients with chronic SCI also will be eligible for enrollment in this study. Gaining additional OPC1 safety and device performance data across a broader range of patients and injury types will be more informative to the program and support further product and device development. Our recent accomplishments in areas of production and delivery contributed real-world feasibility to the promising clinical results previously reported with this program, in which OPC1 demonstrated improvements to quality of life and motor function for certain SCI patients. Importantly, we are working to be in a position to initiate a late-stage clinical study in SCI next year.

The Neurgain PSD system has been designed to allow for the administration of cells to the spinal cord without stopping the patients ventilator during the procedure. Elimination of the need to stop respiration during surgery is expected to reduce the complexity, risk, and variability of administering cells to the area of injury. The Neurgain PSD system has been designed to provide delivery of cells with accurate anatomical positioning and dosing, is more compact than existing devices and is attached directly to the patient during the procedure. This innovative delivery system is expected to provide a significant improvement in usability and provide more flexibility to the surgeon when compared to the methods and tools utilized to deliver OPC1 cells in the completed Phase 1/2a SCiStar study of OPC1 for the treatment of cervical SCI. Neurgain Technologies, Inc. is a medical device company that is developing technologies developed by neurosurgeons at the University of California San Diego.

Lineage plans to evaluate the safety and performance of the Neurgain PSD system to deliver OPC1 to the spinal cord in both the preclinical and clinical setting. If results of these studies are positive, Lineage may exercise its option to enter into a pre-negotiated license and commercialization agreement with Neurgain. Pursuant to that agreement, Lineage may integrate the Neurgain PSD system into a late-stage clinical trial and, if approved, commercial use of OPC1 for the treatment of patients with spinal cord injury. There currently are no FDA approved treatments for spinal cord injury.

About Spinal Cord InjuriesA spinal cord injury occurs when the spinal cord is subjected to a severe crush or contusion and frequently results in severe functional impairment, including limb paralysis, aberrant pain signaling, and loss of bladder control and other body functions. There are approximately 18,000 new spinal cord injuries annually in the U.S. The cost of a lifetime of care for a severe spinal cord injury can be as high as $5 million.

About OPC1OPC1 is an oligodendrocyte progenitor cell (OPC) transplant therapy designed to provide clinically meaningful improvements in motor recovery in individuals with subacute spinal cord injuries. OPCs are naturally occurring precursors to the cells which provide electrical insulation for nerve axons in the form of a myelin sheath. While variability exists for the precise duration of each phase, subacute SCI generally refers to the phase that is three to six weeks post-injury and chronic SCI refers to the phase beginning after the subacute phase. The OPC1 program has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine (CIRM). OPC1 has received Regenerative Medicine Advanced Therapy (RMAT) designation for its use in subacute cervical SCI and Orphan Drug designation from the FDA.

About Lineage Cell Therapeutics, Inc.Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of subacute spinal cord injuries; and (iii) VAC2, an allogeneic dendritic cell therapy produced from Lineages VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking StatementsLineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, can, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to advancement of the clinical development of OPC1 to treat SCI, OPC1s potential to improve quality of life and/or motor function for patients with SCI, the potential benefits of using the Neurgain PSD system to deliver OPC1 for the treatment of SCI, OPC1s regulatory approval pathway, and Lineages potential exclusive license and commercialization agreement with Neurgain. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Lineage's OPC1 Cell Therapy for the Treatment of Spinal Cord Injury to Return to Clinical Testing - Business Wire

SEATTLE--(BUSINESS WIRE)--NanoString Technologies, Inc. (NASDAQ: NSTG), a leading provider of life science tools for discovery and translational research, today announced the launch of the nCounter Stem Cell Characterization Panel for the analysis and optimization of stem cell lines used in the development of potential novel therapeutics.

Recent breakthroughs in stem cell therapy, regenerative medicine, and CRISPR engineering have advanced the development of promising new treatments for debilitating diseases across a broad range of research areas, including neurological and cardiovascular disease, vision loss, and certain types of cancers. However, one of the biggest challenges with stem cell research is the high variability found within the development and manufacturing process that impacts the ability of the stem cells to differentiate and function. The new nCounter Stem Cell Characterization panel measures the eight essential components of stem cell biology and provides a novel, standardized assay for evaluating factors that influence and determine viability, functionality, and pluripotency.

"The simple, automated workflow and highly reproducible, digital results make the nCounter system an excellent fit for all types of stem cell applications," said Chad Brown, senior vice president of Sales and Marketing at NanoString. "With this panel, researchers have a powerful new tool that can quickly assess stem cell health to advance development efforts and optimize stem cell production, achieving robust results in less than 24 hours."

"The Process Development team at ARMI-BioFabUSA is very excited to use the nCounter Stem Cell Characterization panel across a number of our projects where we are developing human tissues composed of mature cells differentiated from stem cells. The Stem Cell Characterization Panel will give us greater insight into the differentiation status of our cells and the success of our current process development and manufacturing runs," said Damian Hile, senior process development scientist at Advanced Regenerative Manufacturing Institute-BioFabUSA (ARMI-BioFabUSA).

The novel 770 gene panel is available for humans and mice and was designed at NanoString with input from leading stem cell experts. To learn more about the nCounter Stem Cell Characterization Panel, visit NanoString at the virtual 2021 ISSCR Conference June 21-26. In addition, NanoString is sponsoring the Cellular Identity: Pluripotency Dynamics session, with Joseph Beechem, Ph.D., chief scientific officer at NanoString.

To learn more about the panel and how the development of the panel can expedite stem cell research, visit the Brief nCounters stem cell experience.

About ARMI-BioFabUSA

The Advanced Regenerative Manufacturing Institute (ARMI), headquartered in Manchester, NH, is an organization funded by the United States Department of Defense. ARMI's mission is to make practical the large-scale manufacturing of engineered tissues and tissue-related technologies to benefit existing industries and grow new ones. ARMI brings together a consortium of over 150 partners from across the industry, government, academia and the non-profit sector to develop next-generation manufacturing processes and technologies for cells, tissues and organs. For more information on ARMI-BioFabUSA, please visit http://www.ARMIUSA.org.

About NanoString Technologies, Inc.

NanoString Technologies is a leading provider of life science tools for discovery and translational research. The company's nCounter Analysis System is used in life sciences research and has been cited in more than 4,300 peer-reviewed publications. The nCounter Analysis System offers a cost-effective way to easily profile the expression of hundreds of genes, proteins, miRNAs, or copy number variations, simultaneously with high sensitivity and precision, facilitating a wide variety of basic research and translational medicine applications, including biomarker discovery and validation. The company's GeoMx Digital Spatial Profiler enables highly-multiplexed spatial profiling of RNA and protein targets in a variety of sample types, including FFPE tissue sections.

For more information, please visit http://www.nanostring.com.

NanoString, NanoString Technologies, the NanoString logo, GeoMx, and nCounter are trademarks or registered trademarks of NanoString Technologies, Inc. in various jurisdictions.

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NanoString Launches nCounter Stem Cell Characterization Panel to Advance the Development of Stem Cell Therapy - Business Wire