Author Archives: admin


Stem Cell Clinic > Articles by: admin

New stem cell identified by Sanford Burnham Prebys researchers offers hope to people with rare liver disease – Newswise

By Adult Stem Cells

Newswise LA JOLLA, CALIF. October 11 Researchers from Sanford Burnham Prebys have discovered a new source of stem cells just outside the liver that could help treat people living with Alagille syndrome, a rare, incurable genetic disorder in which the bile ducts of the liver are absent, leading to severe liver damage and death. The findings, published recently in the journal Hepatology,have extensive biomedical implications for Alagille syndrome and for liver disease in general, including cancer.

Weve been aware of the regenerative power of the liver for a long time, possibly even going back to the ancient Greek myth of Prometheus, says lead authorDuc Dong, Ph.D., an associate professor in theHuman Genetics Programat Sanford Burnham Prebys. But the existence and nature of liver stem cells remains an intensely debated topic.

The new study suggests that the reason these cells have been so hard to find may be that researchers have been looking in the wrong place.

The stem cells that we found are actually outside the liver, not within it, which may have made their discovery difficult, adds Dong. We think these outside the box liver stem cells act more like reserves, only traveling into the liver when all other options are exhausted. It only requires a few of these cells to enter the liver and multiply to repopulate all of the cells lost to the disease.

Over 4 thousand babies each year are born with Alagille syndrome, which is caused by a mutation that prevents duct cells from forming in the liver. And while the syndrome can occasionally resolve naturally, and there are treatments available to manage the symptoms, the disease is incurable, carrying a 75% mortality rate by late adolescence for those without a liver transplant.

"We have known and supported Dr. Dong for years and we feel the work he and his team have done on this disease to date is extraordinary," says Cher Bork, Executive Director of the Alagille Syndrome Alliance. "Hope can be difficult to come by for families dealing with any incurable disease, and discoveries like this help give that hope back to families living with this life-dominating condition."

Using zebrafish, which have many of the same genes and cellular pathways as humans, Dongs research team were able to create a model of Alagille syndrome by selectively deactivating genes associated with Alagille syndrome. These genes encode for chemical messengers from the Notch pathway, a signaling system found in most animals that is involved in embryonic development and adult cell maintenance.

Our work suggests that there is potential for liver regeneration in Alagille patients, but because this signaling pathway is mutated, the regenerative cells fail to fully mature into functioning liver duct cells, says Dong.

In further animal studies, the team showed that by genetically restoring this signaling pathway, the regenerative cells could remobilize to form liver ducts, restoring the function of the liver and improving survival. The researchers are now leveraging their discovery to develop new therapies for Alagille syndrome.

Weve shown not just that regeneration is possible in models of Alagille syndrome, but, importantly, how it can be enhanced, says Dong. These missing duct cells can regenerate if Jagged/Notch is restored, and our lab has developed the first drug that can boost this pathway.

While the new drug requires further studies to advance into clinical trials, the team has already found that it could enhance regeneration and survival in animal models and can trigger the Notch pathway in cells from Alagille patients. These results will be published in separate studies.

Were hopeful that this drug will restore the regenerative potential of the liver in Alagille patients, to be more like the liver of Prometheus, adds Dong.

###

About Sanford Burnham Prebys Medical Research Institute

Sanford Burnham Prebys is a preeminent, independent biomedical research institute dedicated to understanding human biology and disease and advancing scientific discoveries to profoundly impact human health. For more than 40 years, our research has produced breakthroughs in cancer, neuroscience, immunology and childrens diseases, and is anchored by our NCI-designated Cancer Center and advanced drug discovery capabilities. For more information, visit us atSBPdiscovery.orgor on Facebookfacebook.com/SBPdiscoveryand on Twitter@SBPdiscovery.

Originally posted here:
New stem cell identified by Sanford Burnham Prebys researchers offers hope to people with rare liver disease - Newswise

College Student and Retired Teacher to Thank Stem Cell Donors They’ve Never Met for Saving Their Lives During City of Hope’s 45th Bone Marrow…

By Adult Stem Cells

DUARTE, Calif.--(BUSINESS WIRE)--As a 16-year-old high school sophomore, Julian Castaeda was focused on running track specifically, trying to run a mile in under five minutes. He was also planning to attend two camps that summer that would help him prepare for the rigors of college.

Despite being diagnosed with precursor B cell acute lymphoblastic leukemia at age 10 and receiving chemotherapy on and off for three and a half years, Castaeda had been in remission for two years. He had moved on from that difficult experience.

But in March 2017, Castaeda and his mother, Erica Palacios, again received devastating news the leukemia had returned. Castaeda received chemotherapy for a few months, but the cancer kept proliferating. Castaeda would need a hematopoietic stem cell transplant (more commonly referred to as a bone marrow transplant, or BMT) this time to put his cancer back into remission.

It was heartbreaking. I knew at that point that all my plans for sophomore year would be gone, Castaeda recalled.

But Castaeda was determined to get his life back. This was possible thanks to Johannes Eppler, 27, of Breisach, Germany, who joined the bone marrow registry via DKMS, an international nonprofit that is dedicated to the fight against blood cancers and blood disorders, including the recruitment of bone marrow donors. Castaeda received a bone marrow transplant on Aug. 2, 2017, putting the cancer into remission.

He has a big heart, Palacios said about Eppler. Hes an angel. He saved my son. I am thankful that people are willing to [donate].

Castaeda, who grew up in Bakersfield, California, and was treated by City of Hopes Joseph Rosenthal, M.D., M.H.C.M., the Barron Hilton Chair in Pediatrics, is now 20 years old and a junior at California State University Northridge. He also founded Bags of Love Foundation, a nonprofit that has delivered more than 200 care packages to young cancer patients in treatment and has provided $11,000 in scholarships to survivors.

On Friday, Oct. 15, Castaeda will meet his donor for the first time virtually during City of Hopes BMT Reunion. City of Hope, a pioneer and leader in BMT, has hosted a Celebration of Life for bone marrow, stem cell and cord blood transplant recipients, their families and donors for more than 40 years. The celebration honors children and adult cancer survivors, including those who have received autologous transplants, which use a patients own stem cells, and those who received an allogeneic procedure, which require a bone marrow or stem cell donation from a related or unrelated donor.

What began with a birthday cake and a single candle representing a patients first year free from cancer has grown into an annual extravaganza that draws thousands of cancer survivors, donors and families from around the world, as well as the doctors, nurses and staff who help them through the lifesaving therapy.

Each year, patient-donor meetings are the events emotional highlight. Many recipients, though overwhelmed with curiosity and the need to express their gratitude, can only dream of meeting the stranger who saved their lives. City of Hope is making that dream come true for Castaeda, as well as Dona Garrish, a Fullerton, California resident and retired school teacher. Her donor was Michael Fischer, 35, of Wlkau, Germany.

Garrish, 75, received her transplant on March 22, 2017, after it was delayed several times due to infections and other complications that prevented her from going through with the treatment. Garrish, who was diagnosed with acute myeloid leukemia, felt a strong connection to Fischer from the first time a City of Hope employee told her a German male, whom she had never met, was a perfect match for her. She refers to him as her gift from God and her angel on Earth.

He unknowingly encouraged me to fight harder and not to become discouraged, as someday I wanted to meet him and thank him, she added. Garrish recalled watching two patients meeting their donors at the 2017 BMT Reunion. The reunions were held in front of City of Hope Helford Clinical Research Hospital, where Garrish was recovering from her transplant.

While tethered to her IV pole, Garrish looked down from the hospitals sixth floor and said, Thats what I want to do.

City of Hope nurses, doctors and staff were constantly there supporting me every step of the way, even when I couldnt take a single step, said Garrish, who was treated by City of Hopes Liana Nikolaenko, M.D. The timing was urgent, my battle was rough and long, but I live, breathe and enjoy life today because of City of Hope.

Other event highlights include videos of grateful patients wearing the signature BMT buttons that display the number of years since their transplants, comedy by City of Hope BMT patient Sean Kent and a dance/song performed by BMT nurses, known as the Marrowettes. There will be special guest appearances by a Los Angeles Dodger and Katharina Harf, executive chairwoman of DKMS U.S., to congratulate patients, their donors and the BMT program.

During our annual BMT reunion, we express our most heartfelt thanks to the many selfless individuals who each year donate their bone marrow or stem cells to save a persons life, said Stephen J. Forman, M.D., director of City of Hopes Hematologic Malignancies Research Institute and former chair of its Department of Hematology & Hematopoietic Cell Transplantation. Whether the donor is a patients family member or a person she or he has never met, we are all extremely grateful that these donors took the time to donate and gave someone a second chance at life.

About City of Hopes BMT program

City of Hopes BMT program has performed more than 17,000 transplants, making it one of the largest and most successful programs in the nation. The institution has the largest BMT program in California, performing over 700 transplants annually, and is among the top three hospitals in the nation in terms of total transplants performed.

Over the years, City of Hope has also helped pioneer several BMT innovations. In addition to being one of the first institutions to perform BMTs in older adults, it was one of the first programs to show that BMTs could be safely performed for patients with HIV. City of Hope has had growing success with nonrelated matched donors and, most recently, half matched family donors.

City of Hopes BMT program is the only one in the nation that has had one-year survival above the expected rate for 15 consecutive years, based on analysis by the Center for International Blood and Marrow Transplant Research.

City of Hope was also one of the first programs to develop a treatment for prevention of cytomegalovirus (CMV), a common and potentially deadly infection after transplant, which has nearly eliminated the threat of CMV for BMT patients. The institution successfully conducted clinical trials of a CMV vaccine developed at City of Hope. As a pioneer in the development of CAR T cells to treat cancer, City of Hope is also testing how this form of cancer immunotherapy can help patients have a more successful transplant.

In addition, Be The Match at City of Hope last year added more than 13,000 new volunteers willing to save a life when they match a patient who needs a bone marrow transplant. In total, nearly 300,000 potential donors have signed up via City of Hope, motivated by a patient at the cancer center. Be The Match encourages healthy individuals between the ages of 18 and 40 to take the first step of registering by texting COHSAVES to 61474. To learn more about the donation process, visit Be The Match at City of Hopes website.

The public can register to view the event here.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin, monoclonal antibodies and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope is ranked among the nations Best Hospitals in cancer by U.S. News & World Report. Its main campus is located near Los Angeles, with additional locations throughout Southern California and in Arizona. Translational Genomics Research Institute (TGen) became a part of City of Hope in 2016. AccessHope, a subsidiary launched in 2019, serves employers and their health care partners by providing access to NCI-designated cancer center expertise. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

Read the original here:
College Student and Retired Teacher to Thank Stem Cell Donors They've Never Met for Saving Their Lives During City of Hope's 45th Bone Marrow...

Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in…

By Adult Stem Cells

NEW YORK, Oct. 14, 2021 /PRNewswire/ --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 digital37thCongress 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:

"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 studyevaluated 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 NurOwntechnology 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 NurOwntechnology 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 atwww.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwnmanufacturing 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 NurOwntreatment 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 NurOwntreatment 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 NurOwntreatment 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 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:

Investor Relations: Eric Goldstein LifeSci Advisors, LLC Phone: +1 (646) 791-9729 egoldstein@lifesciadvisors.com

Media:Mariesa Kemble kemblem@mac.com

View original content:

SOURCE Brainstorm Cell Therapeutics Inc

See the article here:
Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in...

CellOrigin secured a new round of investment for developing its globally proprietary iPSC-CAR-Macrophage technology platform – WWNY

By Induced Pluripotent Stem Cells

Published: Oct. 15, 2021 at 8:50 AM EDT|Updated: 17 hours ago

HANGZHOU, China, Oct. 15, 2021 /PRNewswire/ -- On Oct. 11th, 2021, CellOrigin Inc. released data about its second generation of iPSC-CAR-Macrophage which has a genetically integrated secondary signal to confer controlled CAR-iMac polarization, in the 5th International Conference of IGC China, 2021, Beijing.

Recently, CellOrigin Biotech, a company committed to iPSC-derived innate immune cell therapeutics, has announced a new round of investment by Kunlun Capital. The investment will be used for the CMC development for its current pipeline of iPSC-derived innate immune cells such as iPSC-CAR-Macrophage and rationlly designed iPSC-NK cells. Before, CellOrigin have also acquired investment from Shulan Health and Nest. Bio Ventures.

CellOrigin Biotech has a long term focus on iPSC-derived innate immune cells and its applications in new cancer immune cells. Dr. Jin Zhang, the scientific co-founder of CellOrigin used to be trained as a research fellow at the Boston Children's Hospital and Harvard Medical School. Now, his team worked closely with clinicians at the First Affiliated Hospital of Zhejiang University, and for the first time his team reported the induced pluripotent stem cell or iPSC-derived CAR-macrophages (CAR-iMac), and its applications in cancer immunotherapies.

CellOrigin Biotech holds the domestic and global patents for iPSC-derived CAR-Macrophage, and the engineering for polarization. With this proprietary platform, they are collaborating with research groups in genome engineering and synthetic biology worldwide to fully unleash the potential of iPSC-derived immune cells, which are highly editable, expandable and clonal. Eventually, they would like to achieve a goal of bring more effective, universal and safe immune cell products to cancer patients, especially for those with solid tumors. The investigator initiated trials has been initiated at the First Hospital of Zhejiang University. The core proprietary technology platform and the core patents including the engineered macrophages from pluripotent stem cells has been authorized and is in the process of entering different countries worldwide.

To support the CMC of its pipeline products, on Oct 1st, CellOriginhas announced the launch of its 3000 square feet GMP facility at Hangzhou, China.

About Kunlun Capital

Founded in 2015, Kunlun capital is committed to long-term value investment, establishing long-term partnership with entrepreneurs, and focusing on investing in enterprises with high technical barriers, excellent founding team and explosive growth potential. In recent years, Kunlun capital has successively invested in KEYA Medical, EdiGene, Cytek (NASDAQ:CTKB), Hui-Gene Therapeutics, OBiO, Okeanos, Ucell Biotech, CellOrigin, Soonsolid, Inke (HK:03700), Dada (NASDAQ:DADA), Dreame, Bamboocloud, Pony.ai, PingCAP, Leyan Technologies.

View original content:

SOURCE Cell Origin

The above press release was provided courtesy of PRNewswire. The views, opinions and statements in the press release are not endorsed by Gray Media Group nor do they necessarily state or reflect those of Gray Media Group, Inc.

Go here to see the original:
CellOrigin secured a new round of investment for developing its globally proprietary iPSC-CAR-Macrophage technology platform - WWNY

ASU professor researches origins of Alzheimers to find a cure – Eight, Arizona PBS

By Induced Pluripotent Stem Cells

Tuesday, Oct. 12, 2021

ASU Associate Professor Dr. David Brafman is using personalized medicine to study the origins of Alzheimers. Dr. Brafman is tracing the origins of Alzheimers as a way to reprogram stem cells and potentially find a cure for the disease. We recently spoke to Dr. Brafman about his research.

What we use to model this disease is a special type of Stem cell called induced pluripotent stem cells and these cells are derived from patients and reprogram cells to essentially take on the characteristics of early development, Brafman said.

Brafman says this type of research can help his lab find the origins of the disease. He also says this research can help identify why some people are more predisposed to develop this disease, as well as commonalities between those who develop it.

A difficult aspect of treating Alzheimers is that by the time many develop noticeable symptoms, treatment becomes very difficult.

What were trying to identify is genetic diagnostic markers that might have profiles that predispose them towards Alzheimers disease so we could maybe introduce therapeutic interventions earlier, Brafman said

Another therapeutic strategy thought about is potentially swapping the harmful genes that lead to Alzheimers for less harmful ones. Brafman said his lab is looking at the potential for genome editing and genetic risk factors that may lead to the disease.

Brafman said this research could be used to trace the origins of other diseases down the road as well, and not just Alzheimers.

Continue reading here:
ASU professor researches origins of Alzheimers to find a cure - Eight, Arizona PBS

Personalized Cell Therapy Market Size Worth US$ 53.8 Billion With a CAGR of 23.5% By 2028 Otterbein 360 – Otterbein 360

By Induced Pluripotent Stem Cells

Coherent Market Insights released a new market study on 2021-2028Personalized Cell Therapy Market with 100+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. At present, the market is developing its presence. The Research report presents a complete assessment of the Market and contains a future trend, current growth factors, attentive opinions, facts, and industry validated market data. Report offering you more creative solutions that combine our deep geographic experience, intimate sector knowledge and clear insights into how to create value in your business. The research study provides estimates for 2021-2028 Personalized Cell Therapy Market Forecast till 2028*.

Request Here For Market Latest Insights(PDF): https://www.coherentmarketinsights.com/insight/request-pdf/204

Manufacturers are focusing on strategies such as expansion of their manufacturing facilities to produce large volume of cell therapy candidates. This will aid manufacturers in penetrating the untapped markets in emerging economies and increase the number of personalized cell therapy manufacturing procedures, worldwide. For instance, in April 2019, Kite, a subsidiary of Gilead Company, announced that it will build a new production facility in Frederick County, Maryland, U.S. for the production of innovative personalized cell therapies useful for cancer treatment.

Browse 23 Market Data Tables and 19 Figures spread through 157 Pages and in-depth TOC on Personalized Cell Therapy Market, by Cell Type (Lymphocytes, Mesenchymal Stem Cell, Hematopoietic Stem Cell, and Others), by Therapeutic Area (Cardiovascular Diseases, Neurological Disorders, Inflammatory Diseases, Diabetes, and Cancer), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa), Global Forecast to 2027

The rising regulatory approval of investigational personalized cell therapy candidates is expected to significantly drive growth of the personalized cell therapy market over the forecast period. For instance, in July 2019, Personalized Stem Cells Inc. received approval from the U.S. FDA for its investigational new drug application that involves usage of adipose-derived stem cells for the treatment of osteoarthritis. Personalized Stem Cells Inc. initiated the first clinical trial for this new therapy candidate in August 2019. The company used stem cells for the treatment of knee osteoarthritis in this clinical trial. The adoption of inorganic strategies such as partnerships and joint ventures by major players is increasing, which is expected to fuel growth of the personalized cell therapy market. For instance, in December 2016, Bayer AG and Versant Ventures launched its joint venture BlueRock Therapeutics, a next-generation regenerative medicine company, for the development of induced pluripotent stem cell (iPSC) therapies to cure various diseases. Moreover, an investment of US$ 225 million was made by Bayer and Versant to strengthen BlueRock Therapeutics manufacturing platform and product pipeline.

The major focus of startup biotech companies are on the development of personalized cell therapies, which is fueling growth of the global personalized cell therapy market. For instance, in October 2019, ElevateBio, a U.S.-based cell and gene therapy developer, initiated a new startup, HighPassBio, for developing novel T cell immunotherapies. In 2019, Phase 1 clinical trial was initiated on an engineered T cell receptor (TCR) T cell therapy for HA-1 expressing tumors targeting relapse of leukemia following hematopoietic stem cell transplant (HSCT).

Key Takeaways of the Global Personalized Cell Therapy Market:

The global personalized cell therapy market is expected to exhibit a CAGR of 23.5% during the forecast period (2019 2027). This is attributed to rising number of personalized cell therapy candidates in investigational phase.

The rising number of acquisitions by major players in the market who are involved in the development of personalized cell therapies is expected to fuel growth of the personalized cell therapy market. For instance, in July 2018, Novartis acquired CellforCure from LFB group, a company that specializes in innovative personalized cell therapy. Through this acquisition, Novartis acquired the cell and gene manufacturing facility of CellforCure for contract manufacturing of Novartis leading CAR-T cell therapy Kymriah (tisagenlecleucel) located in Les Ulis, France.

Personalized Cell Therapy Market Competitive Landscape

Key players functioning in the global prosthetic liners market consists of Cytori Therapeutics Inc., Bellicum Pharmaceuticals, Inc., Saneron CCEL Therapeutics, Inc., MolMed S.p.A., Vericel Corporation, Novartis AG, Gilead Sciences, Inc., Celgene Corporation, Bluebird Bio, Inc. and Aurora Biopharma Inc.

Purchase this Report at Discount Of US $2000 Flat OFF https://www.coherentmarketinsights.com/promo/buynow/204

Reasons to Purchase this Report

Current and future of global Personalized Cell Therapy Market outlook in the developed and emerging markets The segment that is expected to dominate the market as well as the segment which holds highest CAGR in the forecast period. Regions/countries that are expected to witness the fastest growth rates during the forecast period The latest developments, market shares, and strategies that are employed by the major market players

Major Point Answered in Personalized Cell Therapy Market Research Study are:

Request Here Sample Report @ https://www.coherentmarketinsights.com/insight/request-sample/204

Contact Us

Mr. Shah Coherent Market Insights 1001 4th Ave, #3200 Seattle, WA 98154, U.S. Email: sales@coherentmarketinsights.com United States of America: +1-206-701-6702 United Kingdom: +44-020-8133-4027 Japan: +050-5539-1737 India: +91-848-285-0837

See the original post here:
Personalized Cell Therapy Market Size Worth US$ 53.8 Billion With a CAGR of 23.5% By 2028 Otterbein 360 - Otterbein 360

Citius Pharmaceuticals to Host Investor Webcast to Discuss the Acquisition of Late Phase 3 Cancer Immunotherapy I/ONTAK (E7777) – Yahoo Finance

By Induced Pluripotent Stem Cells

Investor Webcast will be held on October 20, 2021 at 8:30 a.m. ET

CRANFORD, N.J., Oct. 13, 2021 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR) today announced that it will host a webcast on October 20, 2021 at 8:30 a.m. ET to discuss the Company's previously announced acquisition of I/ONTAK (E7777), an improved formulation of immunotoxin ONTAK, which was previously approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with persistent or recurrent cutaneous T-cell lymphoma (CTCL).

Myron Holubiak, Chief Executive Officer of Citius Pharmaceuticals, will be joined by Leonard Mazur, Executive Chairman and Dr. Myron Czuczman, Chief Medical Officer to discuss the Company's strategy and the I/ONTAK opportunity. Jaime Bartushak, Chief Financial Officer of Citius Pharmaceuticals will join management for a question-and-answer session following the presentations.

Pre-registration for the webcast is required. Questions related to the I/ONTAK acquisition may be submitted in advance or during the live call via the webcast portal.

Date

Thursday, October 20, 2021

Time

8:30 a.m. ET

Registration link

To join the webcast, please register prior to the event date using this link

US (Toll free)

877-407-6176

US (Toll) / International

201-689-8451

Webcast (live and archive)

Available at http://www.citiuspharma.com in the "Events" section

Q&A

Questions may be submitted in advance to ir@citiuspharma.com

About I/ONTAK (E7777)I/ONTAK (E7777) is a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments. The agent specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis. I/ONTAK, a purified version of denileukin diftitox, is a reformulation of previously FDA-approved oncology treatment ONTAK. ONTAK was marketed in the U.S. from 2008 to 2014, when it was voluntarily withdrawn from the market to enable manufacturing improvements. These improvements resulted in I/ONTAK, which maintains the same amino acid sequence but features improved purity and bioactivity. I/ONTAK has received regulatory approval in Japan for the treatment of cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL). In 2011 and 2013, the FDA granted orphan drug designation (ODD) to I/ONTAK for the treatment of PTCL and CTCL, respectively, making it eligible for seven years of market exclusivity post-approval.

Story continues

A global, multicenter, open-label, single-arm Pivotal study of I/ONTAK in participants with recurrent or persistent CTCL (NCT01871727) is underway. The last patient has been recruited; top line results are anticipated in the first half of 2022. A BLA for I/ONTAK is expected to be filed with the FDA by the end of 2022.

About Citius Pharmaceuticals, Inc.Citius is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, with a focus on oncology, anti-infectives in adjunct cancer care, unique prescription products, and stem cell therapies. The Company has two late-stage product candidates, Mino-Lok, an antibiotic lock solution for the treatment of patients with catheter-related bloodstream infections (CRBSIs), which is currently enrolling patients in a Phase 3 Pivotal superiority trial, and I/ONTAK (E7777), a novel IL-2R immunotherapy for an initial indication in cutaneous T-cell lymphoma (CTCL), which has completed enrollment in its Pivotal Phase 3 trial. Mino-Lok was granted Fast Track designation by the U.S. Food and Drug Administration (FDA). I/ONTAK has received orphan drug designation by the FDA for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). Through its subsidiary, NoveCite, Inc., Citius is developing a novel proprietary mesenchymal stem cell treatment derived from induced pluripotent stem cells (iPSCs) for acute respiratory conditions, with a near-term focus on acute respiratory distress syndrome (ARDS) associated with COVID-19. For more information, please visit http://www.citiuspharma.com.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "plan," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: our ability to successfully integrate I/ONTAK (E7777) into our operations; the risks of relying on a third party to complete the development of I/ONTAK (E7777); risks relating to the results of research and development activities, including those from existing and new pipeline assets, including I/ONTAK (E7777); our ability to successfully undertake and complete clinical trials and the results from those trials for our product candidates; uncertainties relating to preclinical and clinical testing; our need for substantial additional funds; the early stage of products under development; our dependence on third-party suppliers; the estimated markets for our product candidates and the acceptance thereof by any market; the ability of our product candidates to impact the quality of life of our target patient populations; our ability to commercialize our products if approved by the FDA; market and other conditions; risks related to our growth strategy, including our ability to successfully integrate and develop I/ONTAK (E7777); patent and intellectual property matters; our ability to attract, integrate, and retain key personnel; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis, including I/ONTAK (E7777); our ability to procure cGMP commercial-scale supply; government regulation; competition; as well as other risks described in our SEC filings. These risks have been and may be further impacted by Covid-19. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission ("SEC") filings which are available on the SEC's website at http://www.sec.gov, including in our Annual Report on Form 10-K for the year ended September 30, 2020, filed with the SEC on December 16, 2020 and updated by our subsequent filings with the SEC. These forward-looking statements speak only as of the date hereof, and we expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Investor Relations for Citius Pharmaceuticals:Ilanit AllenVice President, Investor Relations & Corporate Communications T: 908-967-6677 x113 E: ir@citiuspharma.com

Citius Pharmaceuticals, a late-stage biopharmaceutical company (PRNewsfoto/Citius Pharmaceuticals, Inc.)

Cision

View original content to download multimedia:https://www.prnewswire.com/news-releases/citius-pharmaceuticals-to-host-investor-webcast-to-discuss-the-acquisition-of-late-phase-3-cancer-immunotherapy-iontak-e7777-301399505.html

SOURCE Citius Pharmaceuticals, Inc.

Read the original:
Citius Pharmaceuticals to Host Investor Webcast to Discuss the Acquisition of Late Phase 3 Cancer Immunotherapy I/ONTAK (E7777) - Yahoo Finance

Wolter Earns Young Investigator Award | Newsroom – UNC Health and UNC School of Medicine

By Induced Pluripotent Stem Cells

The Brain & Behavior Research Foundation named Justin Wolter, PhD, postdoc in the Neuroscience Research Center, as a recipient of the Young Investigator Award.

Justin Wolter, PhD, a postdoctoral researcher in the labs of Jason Stein, PhD, and Mark Zylka, PhD, at the UNC Neuroscience Research Center, the UNC Department of Genetics, and the UNC Department of Cell Biology and Physiology, was named a recipient of the 2021 Young Instigator Award by the Brain & Behavior Research Foundation (BBRF). The award is for $70,000 over two years.

In his research at the UNC School of Medicine, Wolter aims to understand the molecular and cellular mechanisms of neurodevelopmental diseases. With the BBRF award, he will establish a resource to systematically identify genetic interactions between high-risk autism genes and common genetic variation. This project will build upon work in which Wolter established a cell culture-based approach to conduct genome wide association studies in primary human neural progenitor cells.

Wolter will establish a pilot library of genetically diverse induced pluripotent stem cell (iPSC) lines to explore how common and rare genetic variation interact to influence risk and resilience in a genetically defined subtype of autism.

In 2020, Wolter was first author of a Nature paper from the Zylka lab showing how to use the gene-editing technology CRISPR-Cas9 as part of a potential gene therapy approach to treating Angelman syndrome, an autism spectrum disorder.

Initiated in 1987, the BBRF Young Investigator Grant program provides support for the most promising young scientists conducting neurobiological and psychiatric research. This program facilitates innovative research through support of early-career basic, translational and clinical investigators.

This year, the Foundations Scientific Council, led by Herbert Pardes, MD, and comprised of 176 world-renowned scientists with expertise in every area of brain research, reviewed more than 780 applications and selected the 150 meritorious research projects. Many of the Young Investigator grantees are pursuing basic research projects. Others are specifically focusing on new ideas for therapies, diagnostic tools, and technologies. These research projects will provide future insights and advances that will help move the fields of psychiatry and neuroscience forward.

See original here:
Wolter Earns Young Investigator Award | Newsroom - UNC Health and UNC School of Medicine

Stem cells and their role in lung transplant rejection – Michigan Medicine

By Stem Cell Medicine

A lung transplant can mean the difference between life and death for people with diseases such as pulmonary fibrosis, chronic obstructive pulmonary disease (COPD) and even severe COVID-19. Yet, recipients of donor lungs must take daily medications to stave off damage caused by their own immune system, which attacks the organs it recognizes as foreigna process known as rejection.

A new University of Michigan Health study, published in the Journal of Clinical Investigation, has identified cells that appear to play a pivotal role in creating the scarring, or fibrosis, characteristic of chronic rejection following a lung transplant.

Almost 15 years ago, Vibha Lama, MBBS, M.S., a professor in the Division of Pulmonary Disease and Critical Care Medicine, and her lab described the presence of stem-cell-like cells, called mesenchymal stromal cells, in lung sample fluid from lung transplant recipients.

We found that even ten years post-transplant, these cells belonged to the donor, not the recipient, she explained. At that time, we had no clue where in the lung they were coming from or what role they played.

To figure this out, her lab generated a mouse model to recreate what happens within a lung transplant recipient. With the model, they followed a transcription factor known as FOXF1 as a sort of trail of breadcrumbs back to the cells original location.

MORE FROM THE LAB: Subscribe to our weekly newsletter

They discovered that these cells formed a reservoir of stem cells within the bronchovascular bundle deep inside the lung. These bundles contain a bronchus (airway), arteries, connective tissue and other structures and is the part of the lung which connects it to the outside environment.

In this study, explained Lama, who is senior author on the paper, they show that these specific stem cells are interacting with neighboring epithelial cells within that airway niche.

Epithelial cells line and protect the airways and produce a protein known as Sonic hedgehog. Via this protein, epithelial cells signal the stem-cell-like mesenchymal cells, which make up the scaffolding of the lungs, to make FOXF1, a repressor that keeps the stem cells in check.

We are just recently understanding that there are many different kinds of mesenchymal cells in the lung, said Lama. What we describe here is not only are there many kinds of mesenchymal cells, FOXF1 is retained only in these specific stem-cell-like cells.

In the case of lung transplant rejection, Lama hypothesized that immune cells from the recipient attack the epithelial cells which disrupts the balance between them and the mesenchymal cells.

Like Podcasts? Add the Michigan Medicine News Break oniTunes, Google Podcasts or anywhere you listen to podcasts.

Because of the damage caused by rejection, the epithelial cells get damaged, Sonic hedgehog is reduced and that interrupts the signaling to the mesenchymal cells to keep quiet, she said. Because of that, these cells start dividing and they lay down more collagen, which leads to fibrotic scarring.

The work sets the stage for more research into the interaction of these cells with epithelial and other cells it their vicinity to further characterize what happens during chronic rejection and potentially how to prevent it. Furthermore, discovery of these cells is also important in understanding other airway diseases like asthma and COPD.

Paper cited: Transcription factor FOXF1 identifies compartmentally distinct mesenchymal cells with a role in lung allograft fibrogenesis, J Clin Invest. DOI: 10.1172/JCI147343

See the original post:
Stem cells and their role in lung transplant rejection - Michigan Medicine

New Stem Cell Approach Through Using Wavelength Laser Might have Discovered Why Humans Lose Hair – Tech Times

By Stem Cell Medicine

Urian B., Tech Times 05 October 2021, 12:10 am

(Photo : Image from Unsplash Website) New Stem Cell Approach Through Using Wavelength Laser Might have Discovered Why Humans Lose Hair

A new stem cell approach through the use of wavelength lasers might have discovered why humans lose their hair. Rui Yi, a professor of pathology at Northwestern University, is now setting out to answer the question.

According to the Straits Times, a generally accepted hypothesis regarding stem cells notes that they replenish tissues and organs, which include hair, but they will then eventually be exhausted and then even die in place. This particular process is seen as quite an integral part of the aging process.

Stem cells reportedly play a huge role when it comes to the growth of human and mice hair. The director of the Black Family Stem Cell Institute at the Icahn School of Medicine located at Mount Sinai, Sarah Millar, gave a statement. Luminate Medicine has been able to find a way to avoid chemotherapy hair loss.

Sarah Millar wasn't reportedly involved in Yi's paper and explained that the cells gave rise to the hair shaft as well as its sheath. After a period of time, which is short for human body hair and still much longer for hair on a person's head, the follicles then become inactive, and its lower part starts to degenerate. Sarah Millar's discovery can be found on Eurekalert.

The hair shaft then stops its growth and starts to shed, which is only to be replaced by a brand new strand of hair while the cycle repeats. While the rest of the follicles then die, a collection of stem cells still remains in the bulge and are ready to start turning into hair cells in order to grow a strand of hair.

Researchers who study aging usually take chunks of tissue from animals at different ages and examine the changes. There are, however, two drawbacks to this approach, according to Yi. There has also been a relation made betweenhair loss and teeth.

First, it was noted that the tissue was already dead. It is also not clear as to what led to the charges that are reportedly observed or what will then come after them. He then decided that the team would use a different approach.

Read Also:Best Diabetes Apps for Sugar-Conscious Peeps 2021

They reportedly watched the growth of other individual hair follicles in the ears of mice through the use of a long-wavelength laser that will be able to penetrate deep into the tissue. They then start labeling hair follicles along with green fluorescent protein, anesthetizing the animals in order for them to not move.

They then put their ear under the microscope and started to go back and forth to watch what was happening to the exact same hair follicle. The result showed that when the animals got older and grey, they started to lose their hair, their stem cells also started to escape their own small homes in the bulge.

The cells then changed their shapes from around to certain amoeba-like and squeezed out of small holes in the follicles. They then reportedly recovered their normal shapes and started darting away.

Related Article:Gaming And Mental Health: A Closer Look

This article is owned by Tech Times

Written by Urian B.

2021 TECHTIMES.com All rights reserved. Do not reproduce without permission.

Continue reading here:
New Stem Cell Approach Through Using Wavelength Laser Might have Discovered Why Humans Lose Hair - Tech Times

→←