Category Archives: Stem Cell Treatments

Alex Carico, an 8-year-old boy from Lorain County, wants to be a dentist when he grows upand thanks to Special Wish Cleveland and Crest, he already has some pretty special hands-on experience in the field.

On New Years Eve 2019, Alex was diagnosed with neuroblastoma, a rare form of cancer.

It was discovered after what appeared to be an innocuous accident at school; Alex hit his stomach on a lunch table and fell down. The pain, however, never went away. Alexs parents took him to the hospital two weeks after the accident and doctors later performed an MRI scan of Alexs abdomen and found a massive tumor.

Doctors said the tumor wedged itself between some of his vital organs. His chance of survival was 28%.

But Alex is a fighter and his parents, Michael and Bill, picked up and moved to North Ridgeville from Las Vegas in order to get treatment at University Hospitals.

I couldnt even walk that much, I couldnt even leave my room, Alex said.

Although it was tough, Alex went through months of rigorous chemotherapy and stem cell treatment and now is cancer free, defying the odds.

Now that hes in remissionon his fourth clear scanAlex can focus on his future, which he hopes involves him being a dentist.

I want to make everyone have a beautiful smile, Alex said. Smiles bring hope to the world."

A Special Wish Foundation Cleveland made Alexs wish come true by teaming up with a local dentist and letting Alex be a dentist for a day.

That wasnt the end of Alexs journey to provide people with beautiful smiles, however.

After becoming a dentist for a day, Alex was given another opportunity by Crest to don his dental lab coat and treat a patientbut this time Alex got a very special surprise.

The patient Alex was working on was none other than Santa Claus.

Alex said all those cookies Santa eats on Christmas Eve night may have started to take a toll on his teeth.

He had a lot of cavities, Alex said.

But Santa shouldnt worry because Dr. Alex had some sound advice for him.

Brush twice daily.

And for those looking to make Santas nice list while also helping Santa keep his jolly smile bright, Alex had a pretty thoughtful tip.

Maybe what everyone should do is put toothpaste and a toothbrush right by the cookies and the milk so that he can brush afterwards when he eats it, Alex said.

Alex continues to be an inspiration to those around him and wants those who are going through their own fight to stay strong and believe in themselves.

"Be confident in yourself so you can do all the treatments and then after that you can go home," Alex said. "And get to see all your family members again."

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'Smiles bring hope to the world': 8-year-old cancer survivor in Lorain County gets to be Santa's dentist - News 5 Cleveland

Animal stem cell therapy is a usage of animals stem cell to treat a disease or disorder. The ability of stem cell is to divide and differentiate into a cell with specialized function useful for repairing body tissues damaged by injury or disease. The animal stem cell therapy process involve three steps which include collection of stem cell sample from animals and preparing the sample to concentrate the stem cells. Finally, the therapy includes transferring the stem cells into the injured site for treatment. Animal stem cell therapy increases the expectancy of life in animals with no side effects. It is available for the treatment of arthritis, degenerative joint disorders, tendon, and ligaments injuries in animals. Stem cell therapy is most often used to treat dogs, cats, and horses. But recent developments made it possible to use animal stem cell therapy in tiger, pig, etc. Present animal stem cell therapy is studied in treatments of the inflammatory bowel, kidney, liver, heart and immune-mediated diseases respectively.

Animal Stem Cell Therapy Market: Drivers and Restraints

Increasing prevalence of disease in animals with growing population and to increase the animals quality of life, the companies focus shifting towards animal stem cell therapies. Along with increasing government funding for the protection of animals and fast approvals of FDA contributing towards the rapid growth of the animal stem cell therapy. The research in animal stem cells offers great promise for understanding underlying mechanisms of animal development; it gives great opportunities to treat a broad range of diseases and conditions in animals. Animal stem cell therapy is increasingly recognized as critical translational models of human disease for treatment. All these factors act as drivers for the robust growth of the animal stem cell therapy market.

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Animal Stem Cell Therapy Market: Segmentation

Segmentation based on Applications

Segmentation based on End-user

Animal Stem Cell Therapy Market: Market Overview

Studies in the animal stem cell therapy continue at a breathtaking pace due to increasing demand and treatment cost covered in reimbursements. And animal stem cell therapy is more effective than traditional treatment available in the market which is boosting the companies to increase the spending in the R&D for innovative methods. Because of the novelty and complexity of animal stem cell therapy, FDA encourages individuals, universities and drug companies for further innovations. The future expected with double CAGR during the forecasted period.

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Animal Stem Cell Therapy Market: Region-wise Overview

Regarding geographies, North America is dominating the global animal stem cell therapy market due to the increased incidence rate and awareness about the therapy. U.S represents the largest market share in the North America due to the increasing demand for the therapy. Europe and Asia-Pacific are showing a significant growth rate during the forecasted period due to the growing adoption of the animal stem cell therapy. The animal stem cell therapy market in underdeveloped countries is slow when compared to the developed countries.

Animal Stem Cell Therapy Market: Key Participants

The key participants in the animal stem cell therapy market are Magellan Stem Cells, ANIMAL CELL THERAPIES, Abbott Animal Hospital, VETSTEM BIOPHARMA, Veterinary Hospital and Clinic Frisco, CO, etc. The companies are entering into the collaboration and partnership to keep up the pace of the innovations.

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The Animal Stem Cell Therapy market to fill the growth bill in the next decade - The Think Curiouser

A team of UB researchers has published a paper in Nature Communications that is helping to define the best time to give a specific treatment to infants born with Krabbe disease (KD). This treatment has been found to prolong life for these infants for as long as a few years.

The paper was published online in Nature Communication Oct. 23.

Daesung Shin, assistant professor in the Department of Biotechnical and Clinical Laboratory Sciences and the Neuroscience Program, both in the Jacobs School of Medicine and Biomedical Sciences at UB, is the lead investigator. He also conducts research at UBs Hunter James Kelly Research Institute.

KD is an inherited disorder that destroys myelin, the protective coating of nerve cells in the brain and throughout the nervous system. In most cases, signs and symptoms of Krabbe disease develop in babies before 6 months of age, and the disease usually results in death by age 2. When it develops in older children and adults, the course of the disease can vary greatly.

The progressive neurologic disorder is caused by a deficiency of galactosylceramidase (GALC). GALC is an enzyme that breaks down galactosylceramide, an important component of myelin, which ensures the rapid transmission of nerve impulses.

Although there is no cure for KD, hematopoietic stem cell therapy (HSCT), a therapy that makes blood cells, reduces neurologic deterioration and improves developmental advances. These benefits are dependent on the severity of the disease at the time the stem cells are transplanted, and are only beneficial if delivered at a clinically defined pre-symptomatic time point before symptoms appear.

Even though it is widely accepted that early treatment is essential for the most positive outcome, the precise therapeutic window for treatment and what happens during this early time have never been elucidated, Shin says.

To address that issue, his team used mutations to create a novel mouse model of KD.

We engineered an inducible knockout mouse for the GALC gene deletion in specific cells at specific times, which provided us with the opportunity to directly ask when and where GALC enzyme is required for brain development, Shin explains.

We were particularly interested in the role of early developmental GALC function, he says. Our study not only revealed a key developmental process that requires GALC in the perinatal period, but also demonstrated that temporal GALC expression is likely a major contributor to brainstem development.

The researchers found that by increasing GALC levels at or before this newly defined perinatal period they could improve the effectiveness of therapeutic interventions for KD.

For the first time, our work showed the mechanistic evidence to explain why treatment must occur so early, with the defined critical postnatal period at days 4-6 in mice, and demonstrated that temporal GALC expression during this time is a major contributor to brainstem development, Shin says. Augmenting GALC levels at or prior to this newly defined period would likely improve the efficacy of therapeutic interventions for Krabbe patients.

While the time scale between mice and humans is considerably different, the sequence of key events in brain maturation between the two is consistent, he notes. It was estimated that the mouse nervous system at postnatal days 4-6 corresponds to a gestational age of 32 weeks in humans. Therefore, we anticipate that if our result is correct, then in utero treatments at, or prior to, 32 weeks should have better outcomes than conventional postnatal treatment for Krabbe babies.

Shin says his team will further identify which cell type needs to be targeted with therapy.

This work will directly impact the design of novel treatment options for KD patients, he says, noting that KD studies are at the basis of research on other, more common neurodegenerative diseases, such as multiple sclerosis and Parkinsons disease. Therefore, the teams work will have implications beyond KD.

Co-authors on the research were Nadav I. Weinstock, MD-PhD student, and Conlan Kreher, former masters student, both of the HJKRI and the Department of Biochemistry in the Jacobs School; Jacob Favret, research technician in the Department of Biotechnical and Clinical Laboratory Sciences; Lawrence Wrabetz and M. Laura Feltri, both co-directors of the HJKRI and members of the departments of Biochemistry and Neurology, as well as the Neuroscience Program.

Duc Nguyen and Ernesto R. Bongarzone of the Department of Anatomy and Cell Biology in the College of Medicine at the University of Illinois at Chicago also participated in the research.

The project was initiated with the support from Empire State development fund for HJKRI, and further developed and finalized by the R01, R56 and R03 grants from National Institutes for Health-National Institute for Neurological Disorders and Stroke awarded to Shin.

See the article here:
UB researcher narrows time window for administering specific treatment to infants with Krabbe disease - UB Now: News and views for UB faculty and...

The decision to offer the therapy puts Cambridge University Hospitals in the premier league of world class cancer centres.

It also supports progress of the new Cambridge Cancer Research Hospital on the Addenbrookes site, which the Government confirmed in October is included in the second wave of the Hospital Infrastructure Programme (HIP2).

To begin with, the pioneering treatment will be offered to patients with aggressive B-cell lymphomas and acute lymphoblastic leukaemia (ALL) who have relapsed or not responded well to chemotherapy or stem cell treatment.

It is also likely to be offered to cancer patients aged over 70 who are considered to be too high risk to have stem cell transplants.

CAR-T cell therapy can have extremely positive results, but because it can cause unpleasant side effects, it tends to be used when there are few other treatment options available.

CAR-T cell therapy works by re-engineering the patients own immune cells.

Our immune cells, which are called T-cells, circulate around the bloodstream seeking out and destroying any alien intruders, such as infections.

But because cancer cells evolve from our own cells, sometimes our T-cells do not identify them as intruders, and leave them alone.

The new CAR-T cell therapy works by harvesting a patients own T-cells. These are sent to a specialist lab, where they are reprogrammed to express a molecule on their surface, called a Chimeric Antigen Receptor, or CAR, that targets them to the cancer. The reprogrammed cells are grown in a huge numbers over a few weeks and then infused back into the patients body, where they seek out and destroy the cancer. Its a bit like giving immune cells a sat nav.

Until now patients in the east of England needing CAR-T cell therapy have had to make frequent trips to London in preparation for the treatment, which can take another month to administer.

One such patient was Steve Johnson from Bourne who underwent CAR-T cell therapy for relapsed leukaemia at the University College Hospital in London as part of a clinical trial.

Steve explained: "Having the treatment is not pleasant I had a number of fevers and temperature spikes for two weeks after the CAR-T cells were put back in, but I have absolutely no doubt this treatment saved my life and without it I would not be here today."

Steve added: I was lucky for me the trial came at the right time. Having the option to explore and provide revolutionary treatments at places like Addenbrookes and the soon to be built Cambridge Cancer Research Hospital, is vital if we are going to rewrite the story of this devastating illness.

The first patients to be approved for CAR-T cell treatment at Addenbrookes will start having their cells harvested from this week.

Ben Uttenthal, consultant haematologist specialising in CAR-T cell therapy at Addenbrookes, said: This is an exciting new way of treating patients that attacks cancer in a different way from previously available medicines.

"Being able to offer CAR-T cell therapy in our armoury against cancer puts us in the premier league of cancer centres.

It is also a testament to the expertise available across many different specialties in Addenbrookes. Through offering treatments like this, now and in the new Cambridge Cancer Research Hospital, we will be able to benefit cancer patients locally, regionally and nationally.

Initially, the hospital expects to be able to offer CAR-T cell therapy to around 40 patients a year. In the future, treatment is likely to be expanded to include patients with other cancers.

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Addenbrooke's becomes first regional centre to offer CAR-T cell treatment for cancer patients - Cambridge Network

By Alya Zayed

Pictured above: Addenbrookes patient Steve Johnson who received the new treatment. Photo credit: Addenbrooke's Hospital.

A revolutionary cancer treatment that could work on patients not responding to chemotherapy will be offered by Addenbrookes Hospital.

The hospital will be the first in East Anglia to offer CAR-T cell treatment, which puts it in the premier league of cancer centres, said one doctor from Cambridge University Hospitals NHS Foundation Trust, which runs the hospital.

The pioneering treatment will be offered to patients with some cancers who have relapsed or not responded well to chemotherapy or stem cell treatment - namely, aggressive B-cell lymphomas and acute lymphoblastic leukaemia (ALL).

It is also likely to be offered to cancer patients aged over 70 who are considered to be too high risk to have stem cell transplants.

The new therapy can have extremely positive results, but is used as a last resort because of its unpleasant side effects, such as high fevers and low blood pressure.

CAR-T cell therapy works by re-engineering the patients own immune cells.

Immune cells, which are called T-cells, circulate around the bloodstream seeking out and destroying any intruders, such as infections.

But because cancer cells evolve from our own cells, sometimes T-cells do not identify them as intruders, and leave them alone.

The new CAR-T cell therapy works by harvesting a patients own T-cells. These are sent to a specialist lab, where they are reprogrammed to express a molecule on their surface, called a Chimeric Antigen Receptor, or CAR, that targets them to the cancer.

The reprogrammed cells are grown in huge numbers over a few weeks and then infused back into the patients body, where they seek out and destroy the cancer.

It can be a bit like giving immune cells a Sat-Nav, explained the hospital.

The first patients to be approved for CAR-T cell treatment at Addenbrookes will start having their cells harvested from this week.

Initially, the hospital expects to be able to offer CAR-T cell therapy to around 40 patients a year.

In the future, treatment is likely to be expanded to include patients with other cancers.

Ben Uttenthal, consultant haematologist specialising in CAR-T cell therapy at Addenbrookes, said: This is an exciting new way of treating patients that attacks cancer in a different way from previously available medicines.

Being able to offer CAR-T cell therapy in our armoury against cancer puts us in the premier league of cancer centres.

It is also a testament to the expertise available across many different specialties in Addenbrookes.

Through offering treatments like this, now and in the new Cambridge Cancer Research Hospital, we will be able to benefit cancer patients locally, regionally and nationally.

Until now, patients in East Anglia have had to travel to London for the treatment.

Addenbrookes patient Steve Johnson, from Bourn, south Cambridgeshire, is one of many who went to University College Hospital, London, to take part in a clinical trial.

Steve, whose leukemia had relapsed, said: Having the treatment is not pleasant I had a number of fevers and temperature spikes for two weeks after the CAR-T cells were put back in, but I have absolutely no doubt this treatment saved my life and without it I would not be here today.

I was lucky for me the trial came at the right time. Having the option to explore and provide revolutionary treatments at places like Addenbrookes and the soon to be built Cambridge Cancer Research Hospital, is vital if we are going to rewrite the story of this devastating illness.

The new Cambridge Cancer Research Hospital on the Addenbrookes site was confirmed by the government in October as one of the new hospitals to be built.

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"Revolutionary" cancer treatment to be offered at Addenbrooke's Hospital Cambridge - In Your Area

First look at preclinical data in frontotemporal dementia with progranulin mutations (GRN-FTD) and new amyotrophic lateral sclerosis (ALS) program

NOD2 mutation revealed as Crohns disease (CD) genetic target, associated with 7-10% of all CD cases in the U.S. and Europe

Deep dive on transduction enhancers and stable cell line technology innovations that support manufacturing for larger indications

BOSTON and LONDON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today previewed details on its investigational hematopoietic stem cell (HSC) gene therapy research programs in GRN-FTD and NOD2-CD in advance of an upcoming virtual R&D investor event. The company also disclosed a new research program in ALS. A live webcast of the presentation will be available in the Investors & Media section of the companys website at http://www.orchard-tx.com starting Friday, November 13, 2020 at 9:00 a.m. ET.

We are excited to draw back the curtain at tomorrows event on our work in larger indications that form an important part of Orchards evolution as a company, including a new program in ALS, in addition to our work in genetic subsets of FTD and Crohns disease, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. These research programs have been established using a scientific approach that has resulted in more than 160 patients being treated across multiple rare diseases and a recent positive CHMP opinion in the EU for Libmeldy. We believe that HSC gene therapy has the power to transform lives, and we are excited about the possibilities for Orchard and patients with its expanded application.

OTL-204 for GRN-FTD and new ALS research program

The GRN-FTD and ALS programs are based on the same HSC gene therapy approach that has been clinically validated with Libmeldy (OTL-200), Orchards program for metachromatic leukodystrophy, and is under clinical evaluation in the OTL-203 and OTL-201 programs for mucopolysaccharidosis type I and mucopolysaccharidosis type IIIA, respectively. Development work in GRN-FTD and ALS will be undertaken as part of a collaboration with Boston Childrens Hospital (BCH), the University of Padua (UNIPD) and Prof. Alessandra Biffi, chair of the Pediatric Hematology, Oncology and Stem Cell Transplant Division at UNIPD and co-director of the Gene Therapy Program at BCH.

Prof. Biffi commented, The ability of HSC gene therapy to restore healthy microglia function supports the use of this technology for the development of treatments for a variety of diseases with central nervous system involvement. In GRN-FTD, initial in vitro data shows progranulin expression and secretion in culture and uptake indicative of cross-correction. My previous work at BCH researching ALS supports the novel approach of treating this severe neurodegenerative condition by targeting the NOX2 pathway.

OTL-104 for NOD2-CD

Orchards preclinical program in CD targets mutations in the nucleotide-binding oligomerization domain-containing protein 2 (NOD2) gene, which plays a role in immune cell response to bacterial peptides in the gastrointestinal (GI) tract. The companys proposed approach leverages this link, using gene modified HSC-derived cells (monocytes) to replace GI resident macrophages, thus potentially correcting the inflammation and colitis associated with NOD2-CD.

Manufacturing Innovations to Support Work in Larger IndicationsTransduction enhancers (TEs) and stable cell line technology (SCLT)

Orchard has completed a thorough TE screening process and identified and validated several novel TE compounds, which in combination, facilitate lentiviral vector entry into HSCs and have shown a greater than 50% reduction in vector requirements. The enhancers mode of action is expected to be effective in each of Orchards HSC gene therapy programs. An evaluation of enhancer-treated HSC engraftment potential in mice is currently underway.

The company has worked extensively with SCLT, including the technology licensed from GSK for certain programs, to both develop processes to efficiently create SCLs for new vectors and scale up the production of SCLs to clinical grade. Results have delivered consistent levels of high-titer lentiviral production comparable to those seen using conventional methods. Selection of single high-titer clones for new vectors using this method has been achieved within three months. Work at Orchard is ongoing to develop upstream and downstream processes to further improve productivity and scalability.

We have a clear roadmap for Orchards future that prioritizes strategic growth and draws on the many synergies across our scientific, manufacturing and emerging commercial platforms, said Frank Thomas, president and chief operating officer. Over the next 12 months we have an array of exciting commercial, regulatory and clinical milestones that will continue to showcase the breadth and depth of our advanced HSC gene therapy portfolio.

Webcast Information

A live webcast of the presentation New Horizons in Gene Therapy will be available under Events in the Investors & Media section of the companys website at http://www.orchard-tx.com. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchards Research Collaborations

In connection with its previously disclosed collaboration with Prof. Alessandra Biffi, Orchard has signed agreements with Boston Childrens Hospital and the University of Padua to develop and exclusively license new ex vivo HSC gene therapy programs, patents and technologies for the treatment of neurodegenerative disorders. As part of the collaboration, Orchard has initiated sponsored research agreements and obtained exclusive options to license multiple new preclinical programs, including frontotemporal dementia with progranulin mutations (GRN-FTD), amyotrophic lateral sclerosis (ALS) and other rare and less rare indications.Orchard continues to support Professor Biffis labs in the development of new proprietary technology focused on enhancing the application of gene-modified HSC therapy for CNS disorders.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements.Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including with respect to its manufacturing strategy, expected future milestones, and its plans and expectations for the development of its product candidates, including the product candidates referred to in this release, and the therapeutic and commercial potential of its product candidates.These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials of Orchards product candidates will not be repeated or continue in ongoing or future studies or trials involving its product candidates; the risk that the market opportunity for its product candidates may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on preclinical and clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading Risk Factors in Orchards quarterly report on Form 10-Q for the quarter ended September 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1Knopman DS, Roberts RO. J Mol Neurosci. 2011, Onyike CU, Diehl-Schmid J. Int Rev Psychiatry. 2013 and Riedl L, et al Neuropsychiatr Dis Treat. 20142 Centers for Disease Control and Prevention; European Crohns and Colitis Organisation (ECCO); Ashton, James J et al.Clin Transl Gastroenterol. 2020 Feb

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Orchard Therapeutics Unveils Details on New HSC Gene Therapy Research Programs as Part of R&D Investor Event Tomorrow at 9:00 a.m. ET - GlobeNewswire

Multiple myeloma is a plasma cell malignancy that accounts for approximately 1% of new cancer diagnoses per year. The development of a combination approach of mainstay therapies including immunomodulatory drugs (IMiDs), proteasome inhibitors (PIs), and antiCD-38 antibodies has greatly improved up-front remission rates and response duration and prolonged overall survival in the past decade. However, for patients who have been heavily exposed to or are triple or penta-refractory to these classes of therapy, survival is under 1 year.1 Therefore, the emergence of cellular-based therapies represents a major opportunity to improve outcomes in the heavily pretreated and refractory myeloma population.

Chimeric antigen receptor (CAR) T-cell therapies approved by the FDA for several relapsed and refractory B-cell lymphomas are presently under investigation in a variety of treatment line settings for multiple myeloma. As more patients in the community become eligible for CAR T-cell therapies, community oncologists will need to be increasingly familiar about the various products, including their immediate and longer-term risks. In addition, it is key to understand the optimal time for referring these patients to an academic institution, as well as how to manage the requisite post CAR T-cell therapy in the community setting.

The major CAR T-cell therapies in trials for multiple myeloma are directed toward B-cell maturation antigen (BCMA), a surface antigen expressed on B cells starting from their period of development in the germinal centers onward and persisting through the time of plasma cell differentiation. BCMA is minimally expressed in normal human tissues but is heavily expressed on myeloma plasma cells. This allows for safe and intentional targeting of neoplastic myeloma cells using CAR T-cell technology.

The first FDA-approved commercial CAR T-cell therapy will likely arrive in the first quarter of 2021. Patients enrolled on the initial BCMA CAR T-cell trials were required to have had 3 or more prior lines of therapy with exposure to a PI, an IMiD, and an anti-CD38 monoclonal antibody.

The earliest trial was the KarMMa-1 study (NCT03361748) of idecabtagene vicleucel (ide-cel, previously bb2121), which demonstrated an overall response rate (ORR) of 73% and a median progression-free survival (PFS) of 8.8 months in all dose levels. Additionally, a median PFS of 12.1 months was observed in the highest dose level of CAR T cells (450 106).2,3 Patients on this trial (n = 128) had a median of 6 prior lines of therapy, and 84% were triple refractory.

A concurrent trial, CARTITUDE-1 (NCT03548207), studying JNJ-4528 (ciltacabtagene autoleucel in China), also targeting BCMA, demonstrated an unprecedented 100% ORR in patients, with 86% achieving stringent complete response (sCR), the deepest response level by International Myeloma Working Group criteria.4 In the most recent evaluation of PFS, 86% of patients on CARTITUDE-1 had not progressed in disease at a median follow-up time of 9 months.5 New data for JNJ-4528 and the other CAR T-cell therapies being investigated will be made available during the 2020 American Society of Hematology Annual Meeting and Exposition in December and should further reinforce the potential for CAR T-cell therapy to deliver deep and durable responses to patients with multiple myeloma who are heavily pretreated.

What Type of Patients Might Benefit the Most From CAR T?

As in the case of BCMA, antigens for CAR T cells are chosen for their unique expression in tumor tissue and low expression in normal, healthy tissue. The complications that occur in patients receiving CAR T-cell therapy will be largely specific to the targeted antigen. Patients must undergo a multistep process to safely and effectively undergo CAR T-cell treatment and should know up front about the process involved.

The initial step involves leukapheresis, or collecting the patients T cells. Once this is completed, the patient must wait 3 to 5 weeks for typical manufacturing of their CAR T cells at a production facility. If required, patients typically can undergo bridging chemotherapy during this waiting period to maintain their disease, particularly if myeloma progression is accelerating. Once the patients CAR T cells are produced, the patient will undergo lymphodepletion (typically with fludarabine/cyclophosphamide) for 3 days before being infused with prepared CAR T cells. Patients must have sufficient health reserve to withstand the potential adverse effects (AEs) associated with CAR T-cell therapy, including cytokine release syndrome (CRS) and immune effector cellassociated neurotoxicity syndrome (ICANS). Therefore, early referral to an academic center is key to getting these patients on therapy at the optimal time.

CRS is the most common acute AE that occurs in patients treated with CAR T-cell therapy. It occurred in nearly 9 out of 10 patients in the KarMMa-1 and CARTITUDE-1 trials, though severe CRS occurred only in 5% to 7% of patients.3,5 CRS is the phenotypic presentation of a supraphysiologic production of cytokines prompted by immune cell activation, including T cells, macrophages, and monocytes (Table 16 ). Preclinical models have shown that macrophages amplify CRS severity and are the source of several of the culprit cytokines of CRS, including interleukin (IL) 6, IL-1, and IFN-. CRS onset occurs variably depending on the CAR construct used.

Table 1. ASTCT Criteria for CRS6

For example, CRS occurred at median 1 day after infusion of ide-cel, whereas CRS occurred at median 7 days after infusion of JNJ-4528. Our primary treatment for CRS is tocilizumab (Actemra), a monoclonal antibody directed against IL-6; its use does not diminish efficacy of CAR T cells. Subsequent agents such as the IL-1 receptor antagonist anakinra (Kineret), antiIL-5 monoclonal antibody siltuximab (Sylvant), and corticosteroids may be employed for severe cases of CRS.

The second notable AE associated with CAR T-cell therapy is ICANS, which is defined by the American Society for Transplantation and Cellular Therapy as a disorder characterized by a pathological process involving the central nervous system.Symptoms or signs can be progressive and may include aphasia, altered level of consciousness, impairment of cognitive skills, motor weakness, seizures, and cerebral edema.6 A primarily encephalopathic picture appears when patients develop ICANS, which typically occurrs later than CRS. The primary treatment for ICANS involves corticosteroids such as dexamethasone, initially every 6 hours with a quick taper as symptoms start resolving.

Signs and symptoms of neurotoxicity should be screened at least daily during the acute CAR T-cell treatment period and can be assessed with validated tools such as those from CAR T-cell therapy-associated TOXicity (CARTOX) Working Group or the Immune-Effector Cell-Associated Encephalopathy (ICE) tool.6 Severe neurotoxicity occurred at only a 3% rate in KarMMa-1 and CARTITUDE-1 and can be treated readily if caught early with close monitoring.

Other common AEs such as cytopenia and infection may occur during and after the period of CAR T-cell infusion and expansion. Anemia, thrombocytopenia, or neutropenia may occur for months after the initial treatment period, and clinicians should utilize supportive transfusions and/or granulocyte colonystimulating factor and thrombopoietin mimetics as indicated.

Cytomegalovirus quantitative polymerase chain reaction should be checked monthly. If the patient is persistently neutropenic, opportunistic infections should be considered. Intravenous immunoglobulin should be administered before initial lymphodepletion, but also every month for 6 months after CAR T-cell infusion and during winter months if the patient has a history of recurrent infections. In addition, patients should be vaccinated at 1-year post CAR T-cell therapy; a recommended vaccine schedule is included in Table 2.

Table 2. Recommended Vaccine Schedules After CAR T-Cell Therapy

The multiple myeloma treatment landscape is rapidly evolving, with many exciting new therapies on the forefront for relapsed and refractory multiple myeloma. CAR T-cell therapy is a 1-time treatment that allows patients to achieve deep and durable remissions without the need for the usual continuous cancer-directed treatments. The aforementioned trials and therapies have paved the way for subsequent iterations of cellular therapies. These include nonBCMA-targeted CAR T cells; next-generation CAR T cells with improved costimulatory domains for improved safety, efficacy, or faster production; and allogeneic CAR T cells derived from pluripotent stem cells available off the shelf to patients. In the coming year, as commercially available myeloma CAR T-cell therapies are approved, it will be even more important for community oncologists to better understand these therapies so they can offer them to their patients.

References

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CAR T-Cell Therapy for Multiple Myeloma: What Community Oncologists Need to Know - OncLive

Newswise Baltimore, MD, November 12, 2020 Robert C. Gallo, MD, the Homer & Martha Gudelsky Distinguished Professor in Medicine at the University of Maryland School of Medicine (UMSOM) and Co-Founder & Director of the UMSOMs Institute of Human Virology (IHV), announced today that a team of leading scientists in human immunology, virology and stem cell biology, led by Lishan Su, PhD joined IHV on October 1 with academic appointments in the UMSOM Department of Pharmacology. As part of the Maryland E-Nnovation Initiative Fund (MEIF) to recruit top research faculty and a donation to IHV from the Charles Gordon Estate, Dr. Su has been named the Charles Gordon Smith Endowed Professor for HIV Research. Dr. Su will also head IHVs Division of Virology, Pathogenesis and Cancer.

The team will include a 12-person Laboratory of Viral Pathogenesis and Immunotherapy with two faculty appointments as well as major public and private sector research funding.

Dr. Gallo made the announcement in conjunction with University of Maryland School of Medicine Dean E. Albert Reece, MD, PhD, MBA and Margaret M. McCarthy PhD, James & Carolyn Frenkil Deans Professor, Chair of the Department of Pharmacology.

Dr. Su is one of the most successful active basic researchers in America, said Dr. Gallo, who is also Co-Founder and Chairman of the International Scientific Leadership Board of the Global Virus Network. His research is groundbreaking, and we are so pleased to have him join IHV and lead our Division of Infectious Agents and Cancer, which under his sound leadership, will flourish.

Dr. McCarthy added:Dr. Sus continuing ground-breaking work in HIV and Hepatitis B will be a huge asset to the Department of Pharmacology. I look forward to working with him on advances that could open the door to new therapeutics.

Dr. Su was a faculty member in the Lineberger Comprehensive Cancer Center and Professor in the Department of Microbiology & Immunology at University of North Carolina-Chapel Hill since 1996. He received his BS degree in Microbiology from Shandong University, his PhD degree in Virology from Harvard University, and did his post-doctoral training in Stem Cell Biology & Immunology at Stanford University. He worked as a senior research scientist at SyStemix/Sandoz (Novartis), focusing on HIV-1 pathogenesis and stem cell-based gene therapy in humanized mice and in patients.

I am excited to continue and expand my research programs at the Institute of Human Virology (IHV), said Dr. Su. I have long been impressed by the Baltimore-DC area's research centers with great basic and clinical research programs. IHV, co-founded and directed by Dr. Robert Gallo, is one of the first research institutes in the U.S. to integrate basic science, population studies and clinical trials to understanding and treating human virus-induced diseases. The Department of Pharmacology, headed by Dr. Margaret McCarthy, in the University of Maryland School of Medicine, has been outstanding in developing novel therapeutics including breast cancer drugs. I look forward to working with my new colleagues at IHV and the Department of Pharmacology, and across the University of Maryland School of Medicine, to expand and translate my research programs to treating human inflammatory diseases including virus infection and cancer.

Dr. Su has extensive research experience in human immunology, virology and stem cell biology. Dr. Su made important contributions to several areas of human immunology and infectious diseases, particularly in studying human immuno-pathology of chronic virus infections. His lab at UNC-Chapel Hill published important findings in identifying novel virological and immunological mechanisms of HIV-1 pathogenesis. Furthermore, his lab established humanized mouse models with both human immune and human liver cells that support HCV or HBV infection, human immune responses and human liver fibrosis. In recent years, Dr. Sus group discovered, and focused on, the pDC-interferon axis in the immuno-pathogenesis and therapy of chronic HIV & HBV infections. The group also started investigation of the pDC-IFN axis in tumor microenvironments and in cancer immune therapy.

Im so pleased to welcome Dr. Su to our faculty. His work advances the mission of the School of Medicine, which is to provide important new knowledge in the area of immunology and chronic disease to discover new approaches for treatments, said Dean Reece, who is also University Executive Vice President for Medical Affairs and the John Z. and Akiko K. Bowers Distinguished Professor. Dr. Sus stellar research capabilities will provide vital opportunities for collaboration across our Institutes and Departments.

About the Institute of Human Virology

Formed in 1996 as a partnership between the State of Maryland, the City of Baltimore, the University System of Maryland and the University of Maryland Medical System, IHV is an institute of the University of Maryland School of Medicine and is home to some of the most globally-recognized and world-renowned experts in all of virology. The IHV combines the disciplines of basic research, epidemiology and clinical research in a concerted effort to speed the discovery of diagnostics and therapeutics for a wide variety of chronic and deadly viral and immune disorders - most notably, HIV the virus that causes AIDS. For more information,www.ihv.organd follow us on Twitter @IHVmaryland.

About the University of Maryland School of Medicine

The University of Maryland School of Medicine was chartered in 1807 and is the first public medical school in the United States and continues today as an innovative leader in accelerating innovation and discovery in medicine. The School of Medicine is the founding school of the University of Maryland and is an integral part of the 11-campus University System of Maryland. Located on the University of Marylands Baltimore campus, the School of Medicine works closely with the University of Maryland Medical Center to provide a research-intensive, academic and clinically based education. With 43 academic departments, centers and institutes and a faculty of more than 3,000 physicians and research scientists plus more than $400 million in extramural funding, the School is regarded as one of the leading biomedical research institutions in the U.S. with top-tier faculty and programs in cancer, brain science, surgery and transplantation, trauma and emergency medicine, vaccine development and human genomics, among other centers of excellence. The School is not only concerned with the health of the citizens of Maryland and the nation, but also has a global vision, with research and treatment facilities in more than 30 countries around the world. For more information, visitwww.medschool.umaryland.edu.

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Leading Human Immunology and Infectious Disease Experts to Join UM School of Medicines Institute of Human Virology - Newswise

Osteoarthritis and osteoporosis are common conditions in which millions of patients are affected worldwide. Despite the common nature of the diseases, patients are not often regularly scanned to see if they have 1 of them. Instead, they suffer pain and have an increased risk of fragility fractures and worse quality of life.

Nancy Lane, MD, of UC Davis Health is an expert in osteoarthritis and osteoporosis who spoke at the Congress of Clinical Rheumatology East 2020 meeting about the state of both diseases today. HCPLive had the opportunity to speak with Lane about the 2 spaces, popular agents for the treatments of the diseases, and why patients are being scanned regularly for the conditions.

HCPLive: Whats going on in the world of osteoarthritis right now?

Lane: It's an exciting time in the space of osteoarthritis. There's tremendous work being done in the basic science of the disease and there are some very exciting translational phase 2-type studies that are right now recruiting patients that are looking at agents that can stimulate the cartilage to grow and thicken, that can reduce the degradation and increase cartilage matrix formation. There are studies just reported where inhibitor seville 1 reduced joint replacements over 3.5 years by over 50% and a group of agents that by themselves don't reverse or slow the course of the disease, but are an actual inhibitor of pain in the joint by a novel pathway of inhibiting nerve growth factor, that compound tanezumab may be available to our patients in the early part of next year.

So, those are some examples of the excitement in terms of new therapeutics. In addition, although I will not be spending a lot of time talking about this, there finally has been a way to get stem cells to turn into cartilage and grow cartilage. It's a little bit complicated, but it requires some skeletal stem cells and then giving it a growth factor and a factor to inhibit new blood vessel formation, anti-VEGF, and cartilage has now been generated in a mouse where there's been a defect. And if we can translate any of that to the clinic, we're really going to be able to help our patients.

HCPLive: What agents are you most excited about?

Lane: Well to inhibit pain for a prolonged period of time, I'm excited about the inhibition of nerve growth factor of the tanezumab work. For the IL-1 inhibitor, the data are for canakinumab. The stem cell work is in its infancy and is being done out of Stanford.

HCPLive: Youre also an expert in osteoporosis. How do you individualize treatment for that condition?

Lane: Well, that's a great question. We look at it in terms of patients who are at high risk for fracture and we try to prevent fractures by giving them long-acting bisphosphonates or other inhibitors. Then we have patients who present to us and they've already had fragility fractures. And for those patients I will emphasize starting them on an anabolic agent, either teriparatide, abaloparatide, or romosozumab followed then by a strong and resorptive agent to maintain that bone that they have gained and maintain their improved bone strength.

HCPLive: It's interesting you bring up bisphosphonates. I was actually just looking at an abstract that was presented earlier today at the North American Young Rheumatologists Investigator Forum and the investigators noted how osteoporosis is a condition a lot of patients are not necessarily screened for. Why is that?

Lane: Well, that's a great question. There's been a sea change over the last 10 years. Ten years ago, many physicians had bone density machines in their office, and they would do the scans right there and interpret them. However, there was a change in reimbursement and it resulted in many of the bone density machines no longer being available, except in hospitals or in large clinics, which for some reason, has reduced physicians and healthcare providers interest in getting bone densities, and the result being less people are treated for their osteoporosis and then osteoporosis comes to the attention of the physician later when there's a fragility fracture. That alone is a diagnosis of osteoporosis. But actually, screening and treating people at high risk, we're lucky if we can do it, lucky if it's done at age 65.

Now, the guidelines are very clear. All women should have a bone density scan at age 65 and all men at the age of 70. Women below the age of 65 should if they've had a fragility fracture or have significant risk factors. So, all women for the most part should be screened by age 65 and all men at age 70, but that's rarely happening today. As a result, we are seeing more fragility fractures in our population.

HCPLive: How detrimental can that be?

Lane: Well, the first fracture by itself, say its a vertebral fracture, may not be detrimental. But 1 fracture significantly increase your risk of another fracture and another fracture, at which point, with the vertebral fractures, you're losing height, you're bent over, and your quality of life goes down. So, we really need to try to recognize that first fracture and get it treated. If we can't do it before the fracture, then we definitely need to do it when they first have a fragility fracture.

HCPLive: How are you able to do that without the machinery?

Lane: Well, it's difficult, but the patients who have a clinical or painful vertebral fracture, or an osteoporotic fracture fragility fracture by say, falling from a high standing height. Hopefully they will get referred to fracture liaison services or to doctors that will work them up. Otherwise, if we don't catch them at the first couple of fractures, that's a bad position to be in. So, we all have to be on the lookout for these patients. Rheumatologists are very good at identifying these patients. We see older patients with other musculoskeletal ailments and rheumatologists tend to be pretty vigilant about getting bone densities, assessing our patients, treating them, and following them.

HCPLive: Is there anything else you would like to add about osteoarthritis or osteoporosis that is happening right now?

Lane: The most important thing is that physicians recognize the need for every woman over 65 and every male over 70 to have a bone density scan. If they're treated, remember after 3 years to check their bone densities to see if they've improved, or if they haven't fractured and they've been on intravenous bisphosphonates, then they should have a drug holiday. If they've been on oral for 5 years and they arent having fractures and their bone density is good, they should be on a drug holiday and remember to follow them. Remember to see them in about 2-3 years and reassess them because many patients after these holidays slip back into needing therapy.

Another very important thing to remember is osteoporosis is like any chronic disease of aging. Once you have it, you always have it and your risk of fracture may change but you need to assess for it the rest of a patient's life.

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Nancy Lane, MD: The State of Osteoarthritis and Osteoporosis - MD Magazine

TOKYO--(BUSINESS WIRE)--SanBio Co., Ltd. (headquarters: Chuo-ku, Tokyo, Representative Director and President: Keita Mori, hereafter SanBio) hereby announces that it has obtained new analytical results from the Phase 2b clinical trial (the trial) of SB623 for the treatment of chronic motor deficit resulting from ischemic stroke the SanBio Group (SanBio Co., Ltd. and its subsidiary SanBio, Inc.) conducted in the US. It also announces that based on the newly obtained results, it has updated its development plans, including in regard to late-stage clinical trials for the ischemic stroke and hemorrhagic stroke programs of SB623 in Japan.

The trial evaluated efficacy and safety of SB623 in 163 patients suffering from chronic motor dysfunction from ischemic stroke. On January 29, 2019, SanBio announced that the trial did not meet its primary endpoint, as it failed to demonstrate statistical significance in the difference in the proportion of patients whose Fugl-Meyer Motor Scale (FMMS) score improved by 10 or more points from the baseline (primary endpoint) between the treatment group that received SB623 and the control group. Since then, the SanBio Group had continued to work on additional analysis of the trial data, and results of the additional analysis are as follows.

In conducting the additional analysis, from the perspective of minimal clinically important difference (MCID, or the minimal change in scores or other metrics that could be interpreted to mean the change in a patient is clinically meaningful) and based on the results of the Phase 2 clinical trial of SB623 for the treatment of chronic motor deficit from traumatic brain injury (TBI; STEMTRA trial), the company reevaluated trial data using composite FMMS. Of the total 163 patients enrolled in the trial, the company specifically looked at 77 patients who had infarct areas smaller than a certain size (47% of all patients enrolled in this trial). The SanBio Group evaluated the proportion of patients that met one or more of the following FMMS score improvement criteria 24 weeks after treatment: 6-point improvement on FMMS score for upper extremity, 4-point improvement on FMMS score for lower extremity, and 9-point improvement on FMMS total score (all from the baseline). Of the 51 patients in the treatment group that received SB623, improvement was seen in 49%, versus in 19% of 26 patients in the control group that received sham surgery, the difference between the two groups being statistically significant (p-value of 0.02). SanBio Group thinks that even compared to the primary endpointthe proportion of patients whose FMMS score improved by 10 or more points over the baseline six months after treatmentthe endpoint using composite FMMS can adequately explain clinical significance of the treatment efficacy. Details of the additional analysis results will be announced at the financial results briefing for institutional investors and the media held on September 15, 2020. The briefing video will be made available to the public on our website on the 16th of September or thereafter.

Based on the above results, the SanBio Group has begun preparations for the next late-stage clinical trials in the ischemic stroke and hemorrhagic stroke programs of SB623. 2021. Specific designs of the clinical trials and the contents of development for those two programs will be announced promptly upon being finalized. To maximize the value of SB623 at an early stage by selecting areas to focus the Groups management resources on, the SanBio Group plans to prioritize the development of the ischemic stroke and hemorrhagic stroke programs in Japan at the same time as it prepares to file for approval of SB623 for the treatment of chronic motor deficit resulting from TBI in Japan by the end of the current fiscal year (ending January 2021). The Group, however, postponed the global Phase 3 clinical trial for the TBI program of SB623 it had planned to commence this fiscal year to the next or subsequent fiscal years.

Many patients suffering from the chronic effects of ischemic stroke are said to be regularly taking drugs to prevent recurrence. However, because there is no drug that can fundamentally cure motor dysfunction, there is high unmet need for therapeutic drugs to restore motor functions for patients in the chronic phase of stroke. The SanBio Group aims to contribute to improving the lives of these patients, as well as of their family members, suffering from motor impairment and difficulties it causes in carrying out their daily lives through SB623.

About SB623

SB623 is an allogeneic mesenchymal stem cell produced by modifying and culturing bone marrow derived from healthy donors. Implantation of SB623 cells into nerve tissues is expected to promote regeneration of damaged nerve cells. Because SB623 is made from allogeneic cells, large-scale production is possible and there is no need for complex cell processing required for treatments using autologous cells, e.g., cell preparation for each patient at medical institutions. Hence, pharmaceutical products made from allogeneic cells, such as SB623, can be provided to many patients in uniform quality.

About SanBio Co., Ltd. and SanBio, Inc.

SanBio Group is engaged in the regenerative cell medicine business, spanning research, development, manufacture, and sales of regenerative cell medicines. The Companys propriety regenerative cell medicine product, SB623, is currently being investigated for the treatment of several conditions including chronic neurological motor deficit resulting from traumatic brain injury and ischemic stroke. The Company is headquartered in Tokyo, Japan and Mountain View, California, and additional information about SanBio Group is available at https://sanbio.com.

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Additional Analytical Results of the US-Based Phase 2b Clinical Trial of Regenerative Cell Medicine SB623 for the Treatment of Chronic Motor Deficit...