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BrainStorm Cell Therapeutics Announces Peer Reviewed Publication of Results from the NurOwn Phase 2 Progressive MS Trial in Multiple Sclerosis Journal…

NEW YORK, Sept. 15, 2022 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, today announced the peer reviewed publication of data from the Phase 2 trial of NurOwn in progressive multiple sclerosis (MS) in Multiple Sclerosis Journal. The publication, entitled "Evaluation of neurotrophic factor secreting mesenchymal stem cells in progressive multiple sclerosis", can be found here.

Results from the Phase 2, single-arm, open-label study demonstrated NurOwn's safety and provided preliminary evidence of its efficacy in people with progressive MS. Additionally, biomarker analyses confirmed NurOwn's proposed mechanism of action by showing consistent treatment effects in neuroinflammation and neuroprotection pathways.

Twenty participants were enrolled into the Phase 2 trial, with seventeen receiving all three scheduled NurOwn treatments. The mean age of study participants was 47 years with a mean expanded disability status scale (EDSS) score of 5.4 at screening. Results from the trial were compared to 48 matched control patients who were selected from the from the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry (Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases) at the beginning of the trial.

Treatment with NurOwn resulted in large, clinically meaningful improvements in some patients, as defined by response criteria, across all endpoints measured. These endpoints included timed 25-foot walk speed (T25FW), 9-hole peg test (9HPT), multiple sclerosis walking scale (MSWS), symbol digit modality test (SDMT), and low contrast letter acuity (LCLA). These observed improvements diverged from what was seen in matched patients with progressive MS from the CLIMB registry. Key data from the trial, as well as relevant comparisons to the matched CLIMB registry patients, are shown below.

Across all participants, improvements in function as measured by LCLA, SDMT and MS Functional Composite (MSFC) were observed. Mean improvements from baseline of 3.3 points in the LCLA binocular (2.5% contrast), 3.8 points on the SDMT, and 0.18 points in MSFC were observed in treated trial participants. The corresponding changes in matched CLIMB registry patients estimated at 28 weeks showed declines in function on the LCLA and MSFC. The average change in function decline as measured by T25FW, 9HPT, and EDSS across all treated trial participants demonstrated stabilization of functional decline, with similar or slightly worse findings observed in the matched CLIMB registry patients for the same endpoints.

There were no adverse events related to worsening of MS disease and no clinically significant changes in safety lab results/vital signs, confirming NurOwn's favorable safety profile. Two patients developed symptoms of low back and leg pain, consistent with arachnoiditis, occurring in one of three treatments in both participants.

Treatment also consistently resulted in increases in cerebrospinal fluid neuroprotective factors (VEGFA, HGF, NCAM-1, Follistatin, LIF and FetuinA) and reductions in inflammatory biomarkers (MCP-1, sCD27, SDF-1, and Osteopontin), confirming NurOwn's proposed mechanism of action in progressive MS.

"We were pleased that the study's initial results showed efficacy in patients with progressive MS," said Jeffrey Cohen, M.D., Hazel Prior Hostetler Endowed Chair Professor, Cleveland Clinic Lerner College of Medicine, Director, Experimental Therapeutics, Mellen Center for MS Treatment and Research, and the paper's lead author. "There are both promising biological and preliminary clinical signals of a treatment effect that will require confirmation in a randomized trial."

"There is a high unmet need for better treatments for progressive forms of MS and we congratulate the Brainstorm Cell Therapeutics team for the successful completion and publication of this important study. We look forward to future studies that will help to fully understand the potential of NurOwn and other cell-based therapies for this hard-to-treat form of disease" said Bruce Bebo, EVP Research National MS Society.

Chaim Lebovits, Chief Executive Officer, BrainStorm Cell Therapeutics stated, "Having these data peer reviewed and published in the prestigious Multiple Sclerosis Journal is an important step in the evaluation of NurOwn in progressive MS. We appreciate the expertise and commitment of the study investigators and contributions of study participants to advance our understanding of NurOwn's cellular technology platform. Thanks to their efforts and those of the BrainStorm team, we believe we are closer to providing a meaningful treatment option for those with progressive MS".

Ralph Kern, M.D., MHSc, President and Chief Medical Officer of BrainStorm Cell Therapeutics and co-author of the paper commented, "This publication provides preliminary evidence of NurOwn's potential to modify functional outcomes in progressive MS, which we believe warrants further study. In addition, consistent changes in cerebrospinal fluid neuroinflammation and neuroprotection biomarkers reveal how NurOwn may impact disease mechanisms in progressive MS and are complementary to biomarker results observed in our Phase 3 ALS trial. These observations provide further support for NurOwn as a platform technology with potential broad applications and will bolster BrainStorm's efforts to bring much needed solutions to patients with progressive MS, ALS, and other neurodegenerative diseases."

Study Design

The Phase 2 study (BCT-101) was designed to evaluate the safety, efficacy, and biomarker effects of three intrathecal administrations of NurOwn (MSC-NTF cells), given at two-month intervals, to adults with progressive MS. The trial was conducted at four MS centers of excellence: Cleveland Clinic Mellen Center for MS, Icahn School of Medicine at Mount Sinai, Keck School of Medicine of the University of Southern California, and Stanford University School of Medicine. Twenty participants ages 18-65 with progressive MS were enrolled and 17 received all three treatments and were followed for up to 28 weeks. Participants had baseline EDSS scores of between 3.0 and 6.5, were able to walk 25 feet in 60 seconds or less and had not experienced an MS relapse in the 6 months prior to study enrollment.

The primary efficacy outcome was pre-specified improvement (25%) in T25FW or 9-HPT. Additional efficacy endpoints included pre-specified improvements in EDSS, SDMT, LCLA, and MSWS-12. The efficacy outcomes were compared to a pre-specified matched group of progressive MS patients from CLIMB registry (n=48) (Tanuja Chitnis, M.D. Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases). The study was sponsored by Brainstorm Cell Therapeutics with additional financial support for biomarker analyses received from the National Multiple Sclerosis Society, Fast-Forward Commercial Research Funding Program. For more information on the trial, visit https://clinicaltrials.gov/ct2/show/NCT03799718.

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 MS and was supported by a grant from the National MS Society (NMSS).

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could 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, prospects for future regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our products and 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 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; the impacts of the COVID-19 pandemic on our clinical trials, supply chain, and operations; 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.

CONTACTS

Investor Relations: John Mullaly LifeSci Advisors, LLC Phone: +1 617-429-3548 jmullaly@lifesciadvisors.com

Media: Lisa Guiterman lisa.guiterman@gmail.com

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SOURCE BrainStorm Cell Therapeutics Inc.

Company Codes: NASDAQ-SMALL:BCLI

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BrainStorm Cell Therapeutics Announces Peer Reviewed Publication of Results from the NurOwn Phase 2 Progressive MS Trial in Multiple Sclerosis Journal...

Health care workers appeal dismissal of lawsuit over Maine’s vaccine mandate – Kennebec Journal and Morning Sentinel

Attorneys for a group of former Maine health care workers who sued the state over its vaccine requirements last summer are asking a panel of judges in Boston to revive their case.

Last month, U.S. District Judge Jon Levy dismissed the groups lawsuit, which argued they have a religious right to refuse the vaccine overtheir belief that fetal stem cells from abortions were used to develop it. They also argued that the state mandate was discriminatory by allowing for medical exemptions, but not religious ones.

Levy ultimately disagreed.

Exempting individuals whose health will be threatened if they receive a COVID-19 vaccine is an essential, constituent part of a reasoned public health response to the COVID-19 pandemic. It does not express or suggest a discriminatory bias against religion, Levy wrote in his order on Aug. 18.

Attorneys have a month to file a brief to the 1st Circuit Court of Appeals in Boston, outlining their reasons for an appeal.

The plaintiffs worked for MaineHealth, Genesis Healthcare, Northern Light Eastern Maine Medical Center and MaineGeneral Health. All are named as defendants in the complaint, along with Gov. Janet Mills, Maine CDC Director Nirav Shah and Commissioner Jeanne Lambrew of the Maine Department of Health and Human Services.

Nine plaintiffs originally sued in August 2021, all anonymously.

The Portland Press Herald, Kennebec Journal, Morning Sentinel and Sun Journal filed a motion last November challenging the groups right to anonymity. The newspapers argued that the plaintiffs alleged fear of harm no longer outweighs the publics interest in open legal proceedings.

Both Levy and the 1st Circuit Court of Appeals agreed, ordering the group to file a new complaint that included their names in July.

Plaintiffs named in the dismissal document are Alicia Lowe, formerly an employee of MaineHealth; Debra Chalmers and Garth Berenyi, formerly of Genesis Health; Jennifer Barbalias, Natalie Salavarria and Adam Jones, formerly of Northern Light Eastern Maine Medical Center; and Nicole Giroux, formerly of MaineGeneral Health.

They are represented by Maine attorney Steve Whiting, and lawyers from Liberty Counsel, a conservative, religious law firm based in Florida that has participated in several lawsuits against Maine and other states over COVID-19 vaccine mandates and restrictions. Theyve also opposed safe and legal access to abortions and same-sex marriage, leading the Southern Poverty Law Center to identify the firm as a hate group.

Federal judges at every level the U.S. District Court,the 1st U.S. Circuit Court of Appeals in Boston andthe U.S. Supreme Court refused to block Maines COVID-19 vaccine mandate from taking effect while the courts considered the merits of the lawsuit.

The mandate took effect in October, and major health care providers reported that most workers decided to get their shots.

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Health care workers appeal dismissal of lawsuit over Maine's vaccine mandate - Kennebec Journal and Morning Sentinel

I took an international trip with my frozen eggs to learn about the fertility industry – MIT Technology Review

Depending on whats being carried and how much they can pay, the patient or patients involved will choose either a hand-carry service, a commercial carrier such as DHL or FedEx, or something in between, such as the combination of commercial flights and local couriers that Loewen relied on during the pandemic. The cost of transporting my eggs with FlyVet Europa was 1,300 euros, or about $1,400 at the time. That includes the price of two one-way tickets for Paolo and the egg suitcase, and a few incidental expenses. (When I told Monaco how many eggs were traveling, he quipped, Uno squadro di calcio!a soccer team.)

CryoStork, the division of Cryoport devoted to the fertility sector, offers all three tiers of servicecommercial carriers for something that can be easily replaced (sperm, in other words), a middle-tier service using local couriers and air freight, and a door-to-door hand-carry servicefor prices ranging from a few hundred dollars to as much as $7,000 or $8,000 for an international hand-carry trip.

Ultimately, the pandemic boosted business for Loewen. Today, he and a team of eight colleagues, half employees and half working on a per-shipment basis, handle around 30 to 40 IVF-related shipments each month. Similarly, when the war in Ukraine began, Loewen and other colleagues received frantic requests from clients desperate to move their biomaterials out of the capital, Kiev, where most of the countrys IVF clinics and surrogacy agencies are based, and business shifted to nearby Georgia. But by September, Loewen was planning to once again deliver biomaterials to Ukraine. People want to have babiesconflict or not, he says.

What does it take to be a tissue courier, and how does one get into the field? Everyone I spoke to said that to succeed, you must love traveling, have a calm personality (in case, as happened to Loewen, youre ever surrounded by a knot of armed Belarusian soldiers at the airport and accused of trafficking human organs), and be adept at problem-solving.

Loewen looks for people with experience in the travel sector, who can navigate new cities and wont be rattled by a flight cancellation or a grumpy customs official. Mark Sawicki of Cryoport has several former pilots now working as couriers; their security clearances enable them to move through airports more easily than civilians.

Nicole Dorman, 43, has always loved children; she jokes that her current job as a courier is babysitting. She has three kids, aged 14 to 22, and has been a teachers aide and a school crossing guard, following four years in the US Army. When shes home for a week or two at a time with her kids in between gigs, she also makes deliveries for DoorDash in Clarksville, Tennessee.

WENN RIGHTS LTD / ALAMY STOCK PHOTO

Dorman had begun by transporting stem cells for a Frankfurt-based courier service. When she was looking for work in November of 2020, she emailed a half-dozen IVF courier companies and heard back from Loewen within 15 minutes. She has been working for him ever since, and also does US shipments for the Ukrainian company ARK Cryo, as well as EmbryoPort, a UK-based firm.

Dorman is on the road roughly 70% of each month; when we spoke in mid-May, she was preparing for a weeklong trip beginning with a pickup in Indianapolis, a drop-off in Bratislava, a train ride from there to Prague for another pickup, and then a flight to Greece. Like all couriers whove been working for any length of time, she has frequent flier status. In the 18 months since she started, she has transported more than 90 shipments. Now I can pretty much do it in my sleep, she says.

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I took an international trip with my frozen eggs to learn about the fertility industry - MIT Technology Review

External Beam Radiation Therapy Market: Growing Awareness about Early Detection and Diagnosis of various Cancer Types to Drive the Market – BioSpace

Wilmington, Delaware, United States, Transparency Market Research Inc. The accelerating rate of cancer cases worldwide is considered the chief factor augmenting the growth of the global external beam radiation therapy market. The rapid advancements in medical technology and the growing awareness about early detection and diagnosis of various cancer types will also boost this markets growth in the coming years.

External beam radiotherapy or EBRT is considered very common among radiotherapy types. Rather than brachytherapy (fixed source radiotherapy) and unlocked source radiotherapy, in which the radiation source is inside the body, external beam radiotherapy coordinates the radiation at the tumor from outside the body.

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Malignancy or in simpler terms, cancer is a genuine medical issue influencing a huge populace across the globe. Analysis at a beginning phase and therapy for malignant growth, as it influences the insusceptible framework, is a test for medical services suppliers. External radiation is normally done during outpatient visits to an emergency clinic or therapy focus. The vast majority get external radiation therapy over numerous weeks.

In any case, a few groups may have to go to the treatment community two times every day for a less number of weeks. Radiation innovation permits cautious conveyance of external beam radiation therapy. These machines center the radiation around the specific area on the body with the goal that ordinary tissues are influenced as little as could really be expected.

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Global External Beam Radiation Therapy Market: Overview

External radiation (or external beam radiation) is the most common type of radiation therapy used for cancer treatment. A machine is used to aim high-energy rays (or beams) from outside the body into the tumor.

Radiation technology allows very careful delivery of external beam radiation therapy. These machines focus the radiation on the exact location on the body so that normal tissues are affected as little as possible.

External radiation is usually done during outpatient visits to a hospital or treatment center. Most people undergo external radiation therapy over many weeks. Usually, patients visit the treatment center every weekday for a certain number of weeks. However, some people may need to go to the treatment center twice a day for a fewer number of weeks.

Key Driver of Global External Beam Radiation Therapy Market

The global external beam radiation therapy market is growing at a rapid pace owing to increase in incidence of cancer

Cancer is a serious health problem affecting a large population across the globe. Diagnosis at an early stage and treatment for cancer, as it affects the immune system, is a challenge for health care providers. According to the WHO, 18 million new cancer cases were reported in 2018 across the world, and cancer causes around 8.2 million deaths annually. Moreover, the number of cancer cases is likely to rise by 70% across the globe, with 60% of cases in Africa, Asia, and Latin America.

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Key Restraint of Global External Beam Radiation Therapy Market

According to WHO report, an estimated two-thirds of the planet does not have access to basic radiology services. Kenya has only 200 radiologists for 43 million people. People in Nepal travel more than two days to find a facility with an X-ray and spend a months income for that. Increase in gap between radiologists and people restrains the global radiation therapy management market.

North America to Account for Major Share of Global External Beam Radiation Therapy Market

In terms of region, the global external beam radiation therapy market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa

North America is projected to dominate the global market during the forecast period. This is attributed to an increase in number of patients. Moreover, well-established health care infrastructure, presence of key players, and favorable reimbursement policies are anticipated to drive the market in the region.

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Key Players Operating in Global External Beam Radiation Therapy Market

Major players operating in the global external beam radiation therapy market include:

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External Beam Radiation Therapy Market: Growing Awareness about Early Detection and Diagnosis of various Cancer Types to Drive the Market - BioSpace

Author Correction: A microRNA program regulates the balance between cardiomyocyte hyperplasia and hypertrophy and stimulates cardiac regeneration -…

These authors contributed equally: Andrea Raso, Ellen Dirkx.

Department of Molecular Genetics, Faculty of Science and Engineering, Faculty of Health, Medicine and Life Sciences, Maastricht University, Maastricht, The Netherlands

Andrea Raso,Ellen Dirkx,Vasco Sampaio-Pinto,Hamid el Azzouzi,Lara Ottaviani,Serv Olieslagers,Paula A. da Costa Martins&Leon J. De Windt

i3S - Instituto de Investigao e Inovao em Sade, INEB - Instituto Nacional de Engenharia Biomdica, ICBAS - Instituto de Cincias Biomdicas de Abel Salazar, University of Porto, Porto, Portugal

Vasco Sampaio-Pinto&Diana S. Nascimento

Department of Molecular Genetics, Erasmus University MC, Rotterdam, The Netherlands

Hamid el Azzouzi

The Abdus Salam International Centre for Theoretical Physics, Trieste, Italy

Ryan J. Cubero

IST Austria, Klosterneuburg, Austria

Ryan J. Cubero

Stem Cell Institute and Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA

Daniel W. Sorensen&Jop H. van Berlo

Department of Pathology, University Medical Center Utrecht, Utrecht, The Netherlands

Manon M. Huibers&Roel de Weger

Department of Cardiothoracic Surgery, Radboud University Medical Center, Nijmegen, The Netherlands

Sailay Siddiqi&Serena Zacchigna

International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy

Silvia Moimas,Consuelo Torrini,Lorena Zentillin,Luca Braga&Mauro Giacca

Department of Physiology and Cardiothoracic Surgery, Faculty of Medicine, University of Porto, Porto, Portugal

Paula A. da Costa Martins

School of Cardiovascular Medicine and Sciences, Kings College London, London, UK

Mauro Giacca

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Author Correction: A microRNA program regulates the balance between cardiomyocyte hyperplasia and hypertrophy and stimulates cardiac regeneration -...

10-year multiple myeloma survivor: Why I joined a clinical trial at MD Anderson – MD Anderson Cancer Center

When I was diagnosed with multiple myeloma at age 63, it actually didnt bother me. I felt like whatever this was, God had a plan charted for my life before I was even born. Apparently, cancer was meant to be a part of it. So, there was no sense in getting angry or upset about it or throwing myself a big pity party. This was just my lot in life.

I chose MD Anderson for my multiple myeloma treatment because thats what my doctor recommended. But Id already been coming here regularly for prostate cancer screenings. So, I trusted its doctors as medical professionals and experts in cancer treatment. As it turns out, that trust was well-placed. Because Im still here, 10 years later. And, my cancer is still in remission.

Surprise multiple myeloma diagnosis leads to first round of treatment

By the time I was diagnosed, my disease was not considered curable. Multiple myeloma has only three stages, and I was in the final one. Id experienced no symptoms, save a little back pain. But an X-ray showed cancerous lesions all over my spine and rib cage. Two of my vertebrae had cracked because the cancer was growing through the bone.

Myeloma specialist Dr. Michael Wang recommended a stem cell transplant under Dr. Betul Oran. But first, Id need two weeks of radiation therapy under radiation oncologist Dr. Chelsea Pinnix, to treat the lesions on my spine, and several rounds of a chemotherapy drug called bortezomib to prepare my body for the stem cell transplant.

Multiple myeloma relapse leads to clinical trial

I had a stem cell transplant using my own cells on July 9, 2013. Recovery was rough. I ended up back in the hospital the next two Christmases in a row for various issues. But after that, I finally started getting better.

Unfortunately, my cancer relapsed just a few years later. But Dr. Wang said I was a good candidate for a clinical trial involving a new targeted therapy drug now called daratumumab. If I joined it, Id get an infusion of that drug once a month, then take an oral chemotherapy drug called pomalidomide once every other day for 21 days out of each month.

I thought about all the people who have cancer, and how some of them can deal with it pretty well and others cant. I felt like me having this disease might be an opportunity for doctors to study it more closely and possibly find a cure. So, if I could help out with that process, I wanted to do it.

Life after a stem cell transplant and a targeted therapy clinical trial

I joined the clinical trial in September 2015. It went really well, and the experimental treatment I received put me back in remission within a year.

I still have a little weakness in my arms and legs. And Im taking a different course of drugs today due to another relapse. I may have to keep taking it for the rest of my life. But thats OK.

I dont get around as easily as Id like to anymore, but Im alive and able to do most of the things I want to do. When I was diagnosed in 2012, the life expectancy for someone with multiple myeloma was only five years. And here I am, still around after 10 years.

I havent completely recovered because multiple myeloma isnt my only issue. I also have diabetes, heart problems, high blood pressure and sometimes deep vein thrombosis, or dangerous blood clots. But I have a team of specialists at MD Anderson who help me manage all of those conditions. I see endocrinologist Dr. Sonali Thosani, cardiologists Dr. Kaveh Karimzad and Dr. Cezar Iliescu and hematolologist Dr. Cristiam Rojas Hernandez.

I also see multiple myeloma specialist Dr. Elisabet Manasanch now instead of Dr. Wang, because he shifted his practice to focus on a very specific subtype of cancer thats different from mine. I really hated to lose him, because Dr. Wang was great. But all of the doctors and staff Ive met at MD Anderson have been wonderful.And I am grateful for the care of both Dr. Wang and Dr. Manasanch.

I believe if my lifes journey were finished, then I would be finished, too. So, I must not have done everything Im supposed to do just yet. I am grateful to both God and MD Anderson for giving me more time to meet my goals, because I know with their help, I will.

Request an appointment at MD Anderson online or by calling 1-877-632-6789.

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10-year multiple myeloma survivor: Why I joined a clinical trial at MD Anderson - MD Anderson Cancer Center

Researchers make progress toward a stem cellbased therapy for blindness – Ophthalmology Times

What if, in people with blinding retinal disorders, one could simply introduce into the retina healthy photoreceptor cells derived in a dish from stem cells, and restore sight?

According to a news release form the University of Pennsylvania, it is a straightforward strategy to curing blindness, yet the approach has been met with a number of scientific roadblocks, including introduced cells dying rapidly or failing to integrate with the retina.

A new study, published in Stem Cell Reports, overcomes these challenges and marks significant progress toward a cell-based therapy. The work, led by a team at the University of Pennsylvania School of Veterinary Medicine, in collaboration with researchers at the University of Wisconsin-Madison, Childrens Hospital of Philadelphia, and the National Institutes of Healths National Eye Institute (NEI), introduced precursors of human photoreceptor cells into the retinas of dogs. A cocktail of immunosuppressive drugs enabled the cells to survive in the recipients retinas for months, where they began forming connections with existing retinal cells.

In this study, we wanted to know if we could, one, improve the surgical delivery of these cells to the subretinal space; two, image the cells in vivo; three, improve their survival; and four, see them migrate to the layer of the retina where they should be and start integrating, said William Beltran, a professor of ophthalmology at Penn Vet and senior author on the study. The answer to all those questions was yes.

Beltran and Gustavo Aguirre at Penn Vet have long been interested in addressing retinal blinding disorders and they have had great successes to date at producing corrective gene therapies for conditions with known causative genes. But for many cases of inherited retinal degeneration, a gene has not been identified. In other patients, the disease has progressed so far that no photoreceptor cells remain intact enough for gene therapy. In either scenario, a regenerative medicine approach, in which photoreceptors could be regrown outright, would be extremely valuable.

To develop a cell therapy, Beltrans team joined with groups led by John Wolfe of CHOP and Penn Vet; David Gamm at the University of Wisconsin-Madison; and Kapil Bharti at the NEI, in a consortium supported by the NEIs Audacious Goals Initiative for Regenerative Medicine. The partnership combined Beltrans teams expertise in canine models of retinal degeneration and vast experience in cell-based therapy approaches from the Wolfe, Gamm, and Bharti labs.

According to the news release, photoreceptor cells, which are made up of rods and cones, constitute a layer of the outer retina critical to initiating the process of vision, whereby the energy of light transforms into an electrical signal. To function properly, they must form a connection, or synapse, with cells of the inner retina to pass along the visual information. Thus, the goal of this cell therapy is to recreate this layer and enable it to integrate with the retinas other cell types in order to relay signals from one layer to the next.

In the current work, the team used stem cellderived precursors of human photoreceptor cells developed in the Gamm lab to serve as the basis of the cell therapy. In collaboration with the Bharti lab, they developed a new surgical approach to inject the cells, which were labeled with fluorescent markers, into the retinas of seven dogs with normal vision and three with a form of inherited retinal degeneration, then used a variety of non-invasive imaging techniques to track the cells over time.

The use of a large animal model that undergoes a naturally occurring form of retinal degeneration and has a human-size eye was instrumental to optimize a safe and efficient surgical procedure to deliver doses of cells that could be used in patients, Gamm said in the news release.

The researchers observed that cell uptake was significantly better in the animals with retinal degeneration compared to those with normal retinas.

What we showed was that, if you inject the cells into a normal retina that has its own photoreceptor cells, the retina is pretty much intact and serves as a physical barrier, so the introduced cells dont connect with the second-order neurons in the retina, the bipolar cells, Beltran noted in the news release. But in three dogs that were at an advanced stage of retinal degeneration, the retinal barrier was more permeable. In that environment, cells had a better ability to start moving into the correct layer of the retina.

Because the transplanted human cells could be interpreted by the dogs immune system as foreign entities, the researchers did what would be done in other tissue transplant procedures: They gave the dogs immunosuppressive drugs. The trio of medications had been tested previously by Oliver Garden, a veterinary immunologist with Penn Vet at the time of the study, who is now dean of Louisiana State University School of Veterinary Medicine.

Indeed, while the injected cell populations declined substantially in dogs that did not receive the immune-suppressing drugs, the cell numbers dipped but then sustained in the dogs that received the cocktail.

The university noted that further characterization of the introduced cells revealed evidence of potential synapses.

We saw that yes, some are appearing to shake hands with those second-order neurons, Beltran added in the release. There appeared to be contact.

The next stage for this project will be to continue optimizing the therapy, and then test whether there is a functional responsein other words, improved visionin its recipients.

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Researchers make progress toward a stem cellbased therapy for blindness - Ophthalmology Times

New milestone organoid synthesis will boost disease and drug development research – RegMedNet

The concept of synthesizing small-scale human organs in lab dishes has matured from pure science fiction to legitimate bioscientific reality in recent years. However, the usefulness of organoids as a research tool for studying the digestive system quickly ran into a roadblock, due to the fact that these in-demand tissues remain difficult to create.

Organoids are stem cell-derived three-dimensional tissue cultures that are designed to exhibit detailed characteristics of organs or act as model organs to produce a specific cell type in laboratory conditions. However, when growing organoids, the yield from each batch of starting material can vary massively and can even fail to produce any viable organoids at all. This of course results in severe delays in their production and utilization in pre-clinical experiments that test the efficacy and safety of drugs.

In a recently published paper from Stem Cell Reports, researchers from Cincinnati childrens (OH, USA) have developed a new practice that overcomes the organoid production hurdle. This novel procedure is already being utilized within the medical facility to boost organoid studies. However, because the materials utilized can be frozen and thawed while still producing high-quality organoids, this discovery allows for the shipment of starter materials to other labs anywhere in the world, foreseeably leading to a dramatic increase in the utilization of human gastrointestinal organoids in medical research.

This method can make organoids a more accessible tool, explains the first author Amy Pitstick, manager of the Pluripotent Stem Cell Facility at Cincinnati Childrens. We show that the aggregation approach consistently produces high yields and we have proven that precursor cells can be thawed from cryogenic storage to produce organoids of the small intestine.

Using this approach will make it possible for many research labs to use organoids in their experiments without the time and expense of learning how to grow induced pluripotent stem cells (iPSCs), states corresponding author Chris Mayhew, director of the Pluripotent Stem Cell Facility. The ability to freeze the precursor cells also will allow labs to easily make organoids without having to start each new experiment with complicated and highly variable iPSC differentiation.

Generally, organoid creation begins with the collection of skin or blood cells, which are then transformed in the lab to become induced pluripotent stem cells. To create intestinal organoids, highly skilled lab professionals produce a flat layer of organ precursor cells known as the mid-hindgut endoderm.

Under the correct conditions, early-stage organoids, termed spheroids, autonomously develop into a three-dimensional ball of cells. These are then collected and placed into a growth medium, which supplies the required signals for the cells to develop into the specialized cell types of a human organ.

However, the quantity of spheroids produced in this manner has been unpredictable. The Cincinnati Childrens researchers discovered that they could harvest the unused precursor cell layer and employ a centrifuge to transport cells into hundreds of tiny wells housed on small plastic plates. This causes the creation of 3D cell aggregates, which may then be collected and utilized to produce organoids.

The experiment described in the research paper demonstrates that the spheroids created in this manner had no discernible differences from those that formed naturally. The scientists then stored samples of the progenitor cells in freezers. These cells generated viable spheroids after being frozen and aggregated.

The paper goes on to verify that these spheroids can be consistently grown into mature organoids, which can simulate organ function. In the case of this research, the mature organoids went on to mimic the function of the small intestine, large intestine and the antrum, the portion of the stomach that links to the intestine.

Although this development is a welcome and promising advance in organoid fabrication, years of research will be required to create organoids large enough and complex enough to be utilized as replacement tissue in transplant surgery. However, having access to a large number of readily manufactured organoids offers up numerous possibilities for medical study.

More labs will be able to create patient-specific organoids in order to evaluate drugcombination therapiesfor precision treatment of complex or rare disease states that necessitate personalized care. Scientists also conducting basic research to understand more about the genetic factors and molecular pathways at play in digestive tract diseases will be able to incorporate organoids in their experiments by procuring frozen spheroid precursors.

In his current effort to generate transplantable intestinal tissues, Michael Helmrath, Director of Clinical Translation for the Center for Stem Cell & Organoid Medicine (CuSTOM) at Cincinnati Childrens, has already begun employing materials made from this new method.

This is a great step forward for the field on many fronts, Helmrath says. To be able to reduce the complexity of the process and provide higher yields is beneficial to our work. And to be able to translate the methods to other labs will help move regenerative medicine forward.

Source: https://linkinghub.elsevier.com/retrieve/pii/S2213671122003599

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New milestone organoid synthesis will boost disease and drug development research - RegMedNet

Research discovery may help diagnose and treat cancer and brain disorders – EurekAlert

Researchers at Queens University Belfast have revealed how the pathway of an identified protein could lead to early diagnosis and targeted treatment for several cancers and brain disorders.

The team of researchers discovered how the journey or molecular pathway of an identified protein is both essential for brain development and howan alteration to its pathway could result in the spread of cancer.

The study, published today inNature Cell Biology, has revealed the molecular mechanisms of a timely and spatially controlled movement of cells that is essential for the migration of newborn neurons during brain development and can also cause the spread of cancer, or cancer metastasis throughout the body.

It is expected this discovery will have a huge impact on the fundamental understanding of cancer metastasis and brain development and could lead to earlier diagnosis and better treatments, the research authors said.

During brain development, neural stem cells give birth to neurons, which then migrate to specific locations within the brain where they form connections and mature in function. A defect in this process is known to cause several neurodevelopmental disorders. A better understanding of these events is key to decoding fundamental mechanisms of brain development and revealing novel diagnostics and therapeutic avenues for such disorders.

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, or nearly one in six deaths. The majority of tumours are solid, except for a few cancer types of blood origin. Often by the time solid tumours are detected, some cells from the primary tumor have begun to spread to other parts of the body by a process called metastasis, giving rise to secondary tumors whose cells are often resistant to chemotherapy. While surgical removal, chemotherapy and other types of anti-tumour therapy can target the primary tumour, metastasis makes the outcome unpredictable and can lead to more aggressive relapse. It is crucial to understand the features of cancer in order to tackle it.

Epithelial to Mesenchymal Transition (EMT) is a particular molecular pathway that enables cell migration and is vital for early development processes including brain development as well as for wound healing later in life but is also used by cancer cells for metastasis. The research team identified a particular protein, ZNF827, which they identified as a critical regulator of EMT. The study shows how the journey or molecular pathway of the protein is both employed for migration of newborn neurons to proper places during brain development and also exploited by tumour cells to gain migration potential and thereby cause metastasize to different organs.

Lead Author, Dr Vijay Tiwari from the Wellcome-Wolfson Institute for Experimental Medicine at Queens University, said: Our study not only sheds light on the development of one of the most important organs in our body the brain but it also shows how the same protein that is key for brain development can also be the cause or target for the spread of cancer in the body, a real Jekyll and Hyde protein.

The process for migrating newborn neurons to proper places during brain development is the same process exploited by tumour cells to gain migration potential, causing the movement of cancer throughout the body, or cancer metastasis.

By identifying key regulators of these pathways, we open new opportunities for a therapeutic intervention against cancer and a better understanding of neurodevelopmental disorders involving defects in brain development.

The international team includes researchers from Queens University Belfast, Salk Institute for Biological Studies, Altos Labs, University of Montpellier, Karolinska Institutet, University Medical Center of the Johannes Gutenberg University Mainz and Translational Oncology at the University Medical Center of the Johannes Gutenberg University Mainz gGmbH (TRON gGmbH).

This study was supported by the Deutsche Forschungsgemeinschaft, Wilhelm Sander Stiftung and Innovation to Commercialisation of University Research programme.

Nature Cell Biology

Experimental study

Animals

'A complex epigenome-splicing crosstalk governs epithelial-to-mesenchymal transition in metastasis and brain development'

9-Aug-2022

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Research discovery may help diagnose and treat cancer and brain disorders - EurekAlert

Stem Cell Alopecia Treatment Market Size, Scope, Revenue, Opportunities and Growth by 2028 Shanghaiist – Shanghaiist

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Stem Cell Alopecia Treatment Market Size, Scope, Revenue, Opportunities and Growth by 2028 Shanghaiist - Shanghaiist