Stem Cell Clinic

A real life Superman celebrates 5 years of survival from one of the deadliest cancers – Newswise

Newswise CLEVELAND Three college graduations. Three family weddings. The births of two grandchildren.

Andy Superman Simon has cherished each of these milestones since he was diagnosed five years ago with a glioblastoma multiforme (GBM grade 4), one of the deadliest and most challenging cancers to treat. GBM patients typically survive an average of 12-15 months. Only 6.8 percent of GBM patients survive five years, according to the National Brain Tumor Society.

But the Superman of University Hospitals Seidman Cancer Center who memorably donned a full costume for his final treatment in September 2016 and is free of cancer today with no recurrence is anything but typical.

I feel incredible, says Simon, now 56. I flew through treatment with ease, because I had the best team and the best surgeon. The way I see it, I had cancer, I dont have it.

The crushing headache, similar to a migraine yet inexplicably and mysteriously different, struck early one morning in November 2015. Pulling out of his driveway to head to the ER, Simon was equidistant from two different hospital systems. He and his wife believe that fateful turn to come to UH Ahuja Medical Center, and then UH Seidman Cancer Center, has made all the difference.

If we hadnt gone to UH, I honestly believe in my heart that Andy wouldnt be here today, said Amy, Simons wife.

Neurosurgeon Andrew Sloan, MD, Director of UHs Brain Tumor & Neuro-Oncology Center and the UH Seidman Center for Translational Neuro-Oncology, diagnosed the large mass in Simons brain as a GBM. He performed a craniotomy on Simon using 5-Aminolevulinic Acid (5-ALA), an experimental agent that improves the surgeons ability to identify the tumor. Dr. Sloans own surgical trial assessing this agent was one of only a handful of studies in the United States at the time, though it is now approved for use throughout the US by the FDA. Simon took the 5-ALA prior to surgery, which causes the cancer cells to glow hot pink, for more complete removal of these aggressive, invasive tumors.

Radiation and chemotherapy are the standard of care following a craniotomy for GBM.

Simon also took advantage of a novel phase I clinical trial that involved genetically engineering his own blood cells to express a mutant protein that made them more resistant to chemotherapy enabling him to safely withstand steadily higher doses of toxic chemotherapy through six rounds. While this phase I trial was designed only to show safety and feasibility, the median survival of the participants was 3.3-fold higher than anticipated based on case-matched historical controls with GBM undergoing standard treatment.

A new clinical trial, funded by a $2.3 million grant from the National Cancer Institute and based on the gene therapy Simon participated in, will open at UH Seidman Cancer Center in the next few months.

Andy has been a champion, Dr. Sloan says of the poster-boy for this trial, noting that five-year GBM survivors commonly experience recurrence. Hes a real fighter.

This treatment is really a game-changer. This could be the new standard of care. Its really exciting and very promising.

For the last several years, Simon has celebrated with a big party complete with a photo display of his milestones. He was planning a blowout celebration this year until the pandemic struck.

There is hope, says Simon. I have too many things to fight for, and to live for. Ive gotten too far. Im going to be a statistic for the other side. Every day is a milestone really.

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About University Hospitals / Cleveland, Ohio

Founded in 1866, University Hospitals serves the needs of patients through an integrated network of 19 hospitals, more than 50 health centers and outpatient facilities, and 200 physician offices in 16 counties throughout northern Ohio.The systems flagship academic medical center, University Hospitals Cleveland Medical Center, located in Clevelands University Circle, is affiliated with Case Western Reserve University School of Medicine. The main campus also includes University Hospitals Rainbow Babies & Children's Hospital, ranked among the top childrens hospitals in the nation; University Hospitals MacDonald Women's Hospital, Ohio's only hospital for women; University Hospitals Harrington Heart & Vascular Institute, a high-volume national referral center for complex cardiovascular procedures; and University Hospitals Seidman Cancer Center, part of the NCI-designated Case Comprehensive Cancer Center. UH is home to some of the most prestigious clinical and research programs in the nation, including cancer, pediatrics, women's health, orthopedics, radiology, neuroscience, cardiology and cardiovascular surgery, digestive health, transplantation and urology. UH Cleveland Medical Center is perennially among the highest performers in national ranking surveys, including Americas Best Hospitals from U.S. News & World Report. UH is also home to Harrington Discovery Institute at University Hospitals part of The Harrington Project for Discovery & Development. UH isone of the largest employers in Northeast Ohio with 28,000 physicians and employees.Advancing the Science of Health and the Art of Compassion is UHs vision for benefitting its patients into the future and To Heal. To Teach. To Discover.is the organizations unwavering mission. Follow UH on Facebook @UniversityHospitalsand Twitter @UHhospitals. For more information, visitUHhospitals.org.

About University Hospitals Seidman Cancer Center

UH Seidman Cancer Center is the only freestanding cancer hospital in Northeast Ohio, where all clinicians and staff are dedicated to the prevention, diagnosis and treatment of cancer while researching new and innovative treatment options through clinical trials. Nationally ranked cancer care is also available to patients through the 11-country region at 18 community-based locations. Our UH Seidman specialists make up 14 cancer-specific teams focused on determining integrated care plans tailored to patients needs. UH Seidman Cancer Center is part of the National Cancer Institute (NCI)-designated Case Comprehensive Cancer Center at Case Western Reserve University, one of 50 comprehensive cancer centers in the country. Patients have access to advanced treatment options, ranging from a pioneering stem cell transplant program founded more than 40 years ago and a wide range of immunotherapy to the first and only proton therapy center in northern Ohio for adults and children. Go to UHhospitals.org/Seidman for more information.

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A real life Superman celebrates 5 years of survival from one of the deadliest cancers - Newswise

Global Cell Harvesting Market to Reach US$381,4 Million by the Year 2027 – Salamanca Press

NEW YORK, Nov. 25, 2020 /PRNewswire/ --Amid the COVID-19 crisis, the global market for Cell Harvesting estimated at US$233.2 Million in the year 2020, is projected to reach a revised size of US$381.4 Million by 2027, growing at a CAGR of 7.3% over the period 2020-2027.Manual, one of the segments analyzed in the report, is projected to grow at a 7.9% CAGR to reach US$284.4 Million by the end of the analysis period. After an early analysis of the business implications of the pandemic and its induced economic crisis, growth in the Automated segment is readjusted to a revised 5.6% CAGR for the next 7-year period. This segment currently accounts for a 28.3% share of the global Cell Harvesting market.

Read the full report: https://www.reportlinker.com/p05798117/?utm_source=PRN

The U.S. Accounts for Over 30.9% of Global Market Size in 2020, While China is Forecast to Grow at a 10.4% CAGR for the Period of 2020-2027

The Cell Harvesting market in the U.S. is estimated at US$72 Million in the year 2020. The country currently accounts for a 30.86% share in the global market. China, the world second largest economy, is forecast to reach an estimated market size of US$34.9 Million in the year 2027 trailing a CAGR of 10.4% through 2027. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 6.1% and 7% respectively over the 2020-2027 period. Within Europe, Germany is forecast to grow at approximately 6.6% CAGR while Rest of European market (as defined in the study) will reach US$34.9 Million by the year 2027.We bring years of research experience to this 5th edition of our report. The 226-page report presents concise insights into how the pandemic has impacted production and the buy side for 2020 and 2021. A short-term phased recovery by key geography is also addressed.

Competitors identified in this market include, among others,

Read the full report: https://www.reportlinker.com/p05798117/?utm_source=PRN

I. INTRODUCTION, METHODOLOGY & REPORT SCOPE I-1

II. EXECUTIVE SUMMARY II-1

1. MARKET OVERVIEW II-1 Cell Harvesting - A Prelude II-1 Impact of Covid-19 and a Looming Global Recession II-1 With Stem Cells Holding Potential to Emerge as Savior for Healthcare System Struggling with COVID-19 Crisis, Demand for Cell Harvesting to Grow II-1 Select Clinical Trials in Progress for MSCs in the Treatment of COVID-19 II-2 Lack of Antiviral Therapy Brings Spotlight on MSCs as Potential Option to Treat Severe Cases of COVID-19 II-3 Stem Cells Garner Significant Attention amid COVID-19 Crisis II-3 Growing R&D Investments & Rising Incidence of Chronic Diseases to Drive the Global Cell Harvesting Market over the Long-term II-3 US Dominates the Global Market, Asia-Pacific to Experience Lucrative Growth Rate II-4 Biopharmaceutical & Biotechnology Firms to Remain Key End-User II-4 Remarkable Progress in Stem Cell Research Unleashes Unlimited Avenues for Regenerative Medicine and Drug Development II-4 Drug Development II-5 Therapeutic Potential II-5

2. FOCUS ON SELECT PLAYERS II-6 Recent Market Activity II-7 Innovations and Advancements II-7

3. MARKET TRENDS & DRIVERS II-8 Development of Regenerative Medicine Accelerates Demand for Cell Harvesting II-8 The Use of Mesenchymal Stem Cells in Regenerative Medicine to Drive the Cell Harvesting Market II-8 Rise in Volume of Orthopedic Procedures Boosts Prospects for Stem Cell, Driving the Cell Harvesting II-9 Exhibit 1: Global Orthopedic Surgical Procedure Volume (2010- 2020) (in Million) II-11 Increasing Demand for Stem Cell Based Bone Grafts: Promising Growth Ahead for Cell Harvesting II-11 Spectacular Advances in Stem Cell R&D Open New Horizons for Regenerative Medicine II-12 Exhibit 2: Global Regenerative Medicines Market by Category (2019): Percentage Breakdown for Biomaterials, Stem Cell Therapies and Tissue Engineering II-13 Stem Cell Transplants Drive the Demand for Cell Harvesting II-13 Rise in Number of Hematopoietic Stem Cell Transplantation Procedures Propels Market Expansion II-15 Growing Incidence of Chronic Diseases to Boost the Demand for Cell Harvesting II-16 Exhibit 3: Global Cancer Incidence: Number of New Cancer Cases in Million for the Years 2018, 2020, 2025, 2030, 2035 and 2040 II-17 Exhibit 4: Global Number of New Cancer Cases and Cancer-related Deaths by Cancer Site for 2018 II-18 Exhibit 5: Number of New Cancer Cases and Deaths (in Million) by Region for 2018 II-19 Exhibit 6: Fatalities by Heart Conditions: Estimated Percentage Breakdown for Cardiovascular Disease, Ischemic Heart Disease, Stroke, and Others II-19 Exhibit 7: Rising Diabetes Prevalence Presents Opportunity for Cell Harvesting: Number of Adults (20-79) with Diabetes (in Millions) by Region for 2017 and 2045 II-20 Ageing Demographics to Drive Demand for Stem Cell Banking II-20 Global Aging Population Statistics - Opportunity Indicators II-21 Exhibit 8: Expanding Elderly Population Worldwide: Breakdown of Number of People Aged 65+ Years in Million by Geographic Region for the Years 2019 and 2030 II-21 Exhibit 9: Life Expectancy for Select Countries in Number of Years: 2019 II-22 High Cell Density as Major Bottleneck Leads to Innovative Cell Harvesting Methods II-22 Advanced Harvesting Systems to Overcome Centrifugation Issues II-23 Sophisticated Filters for Filtration Challenges II-23 Innovations in Closed Systems Boost Efficiency & Productivity of Cell Harvesting II-23 Enhanced Harvesting and Separation of Micro-Carrier Beads II-24

4. GLOBAL MARKET PERSPECTIVE II-25 Table 1: World Current & Future Analysis for Cell Harvesting by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-25

Table 2: World Historic Review for Cell Harvesting by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-26

Table 3: World 15-Year Perspective for Cell Harvesting by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets for Years 2012, 2020 & 2027 II-27

Table 4: World Current & Future Analysis for Manual by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-28

Table 5: World Historic Review for Manual by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-29

Table 6: World 15-Year Perspective for Manual by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-30

Table 7: World Current & Future Analysis for Automated by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-31

Table 8: World Historic Review for Automated by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-32

Table 9: World 15-Year Perspective for Automated by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-33

Table 10: World Current & Future Analysis for Peripheral Blood by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-34

Table 11: World Historic Review for Peripheral Blood by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-35

Table 12: World 15-Year Perspective for Peripheral Blood by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-36

Table 13: World Current & Future Analysis for Bone Marrow by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-37

Table 14: World Historic Review for Bone Marrow by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-38

Table 15: World 15-Year Perspective for Bone Marrow by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-39

Table 16: World Current & Future Analysis for Umbilical Cord by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-40

Table 17: World Historic Review for Umbilical Cord by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-41

Table 18: World 15-Year Perspective for Umbilical Cord by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-42

Table 19: World Current & Future Analysis for Adipose Tissue by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-43

Table 20: World Historic Review for Adipose Tissue by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-44

Table 21: World 15-Year Perspective for Adipose Tissue by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-45

Table 22: World Current & Future Analysis for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-46

Table 23: World Historic Review for Other Applications by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-47

Table 24: World 15-Year Perspective for Other Applications by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-48

Table 25: World Current & Future Analysis for Biotech & Biopharma Companies by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-49

Table 26: World Historic Review for Biotech & Biopharma Companies by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-50

Table 27: World 15-Year Perspective for Biotech & Biopharma Companies by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-51

Table 28: World Current & Future Analysis for Research Institutes by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-52

Table 29: World Historic Review for Research Institutes by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-53

Table 30: World 15-Year Perspective for Research Institutes by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-54

Table 31: World Current & Future Analysis for Other End-Uses by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 II-55

Table 32: World Historic Review for Other End-Uses by Geographic Region - USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 II-56

Table 33: World 15-Year Perspective for Other End-Uses by Geographic Region - Percentage Breakdown of Value Sales for USA, Canada, Japan, China, Europe, Asia-Pacific and Rest of World for Years 2012, 2020 & 2027 II-57

III. MARKET ANALYSIS III-1

GEOGRAPHIC MARKET ANALYSIS III-1

UNITED STATES III-1 Increasing Research on Stem Cells for Treating COVID-19 to drive the Cell Harvesting Market III-1 Rising Investments in Stem Cell-based Research Favors Cell Harvesting Market III-1 Exhibit 10: Stem Cell Research Funding in the US (in US$ Million) for the Years 2011 through 2017 III-2 A Strong Regenerative Medicine Market Drives Cell Harvesting Demand III-2 Arthritis III-3 Exhibit 11: Percentage of Population Diagnosed with Arthritis by Age Group III-3 Rapidly Ageing Population: A Major Driving Demand for Cell Harvesting Market III-4 Exhibit 12: North American Elderly Population by Age Group (1975-2050) III-4 Increasing Incidence of Chronic Diseases Drives Focus onto Cell Harvesting III-5 Exhibit 13: CVD in the US: Cardiovascular Disease* Prevalence in Adults by Gender & Age Group III-5 Rising Cancer Cases Spur Growth in Cell Harvesting Market III-5 Exhibit 14: Estimated Number of New Cancer Cases and Deaths in the US (2019) III-6 Exhibit 15: Estimated New Cases of Blood Cancers in the US (2020) - Lymphoma, Leukemia, Myeloma III-7 Exhibit 16: Estimated New Cases of Leukemia in the US: 2020 III-7 Market Analytics III-8 Table 34: USA Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-8

Table 35: USA Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-9

Table 36: USA 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-10

Table 37: USA Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-11

Table 38: USA Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-12

Table 39: USA 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-13

Table 40: USA Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-14

Table 41: USA Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-15

Table 42: USA 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-16

CANADA III-17 Market Overview III-17 Exhibit 17: Number of New Cancer Cases in Canada: 2019 III-17 Market Analytics III-18 Table 43: Canada Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-18

Table 44: Canada Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-19

Table 45: Canada 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-20

Table 46: Canada Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-21

Table 47: Canada Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-22

Table 48: Canada 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-23

Table 49: Canada Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-24

Table 50: Canada Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-25

Table 51: Canada 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-26

JAPAN III-27 Increasing Demand for Regenerative Medicine in Geriatric Healthcare and Cancer Care to Drive Demand for Cell Harvesting III-27 Exhibit 18: Japanese Population by Age Group (2015 & 2040): Percentage Share Breakdown of Population for 0-14, 15-64 and 65 & Above Age Groups III-27 Exhibit 19: Cancer Related Incidence and Deaths by Site in Japan: 2018 III-28 Market Analytics III-29 Table 52: Japan Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-29

Table 53: Japan Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-30

Table 54: Japan 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-31

Table 55: Japan Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-32

Table 56: Japan Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-33

Table 57: Japan 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-34

Table 58: Japan Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-35

Table 59: Japan Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-36

Table 60: Japan 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-37

CHINA III-38 Rising Incidence of Cancer Drives Cell Harvesting Market III-38 Exhibit 20: Number of New Cancer Cases Diagnosed (in Thousands) in China: 2018 III-38 Market Analytics III-39 Table 61: China Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-39

Table 62: China Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-40

Table 63: China 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-41

Table 64: China Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-42

Table 65: China Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-43

Table 66: China 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-44

Table 67: China Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-45

Table 68: China Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-46

Table 69: China 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-47

EUROPE III-48 Cancer in Europe: Key Statistics III-48 Exhibit 21: Cancer Incidence in Europe: Number of New Cancer Cases (in Thousands) by Site for 2018 III-48 Ageing Population to Drive Demand for Cell Harvesting Market III-49 Exhibit 22: European Population by Age Group (2016, 2030 & 2050): Percentage Share Breakdown by Age Group for 0-14, 15- 64, and 65 & Above III-49 Market Analytics III-50 Table 70: Europe Current & Future Analysis for Cell Harvesting by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2020 through 2027 III-50

Table 71: Europe Historic Review for Cell Harvesting by Geographic Region - France, Germany, Italy, UK and Rest of Europe Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-51

Table 72: Europe 15-Year Perspective for Cell Harvesting by Geographic Region - Percentage Breakdown of Value Sales for France, Germany, Italy, UK and Rest of Europe Markets for Years 2012, 2020 & 2027 III-52

Table 73: Europe Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-53

Table 74: Europe Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-54

Table 75: Europe 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-55

Table 76: Europe Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-56

Table 77: Europe Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-57

Table 78: Europe 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-58

Table 79: Europe Current & Future Analysis for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-59

Table 80: Europe Historic Review for Cell Harvesting by End-Use - Biotech & Biopharma Companies, Research Institutes and Other End-Uses Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-60

Table 81: Europe 15-Year Perspective for Cell Harvesting by End-Use - Percentage Breakdown of Value Sales for Biotech & Biopharma Companies, Research Institutes and Other End-Uses for the Years 2012, 2020 & 2027 III-61

FRANCE III-62 Table 82: France Current & Future Analysis for Cell Harvesting by Type - Manual and Automated - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-62

Table 83: France Historic Review for Cell Harvesting by Type - Manual and Automated Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-63

Table 84: France 15-Year Perspective for Cell Harvesting by Type - Percentage Breakdown of Value Sales for Manual and Automated for the Years 2012, 2020 & 2027 III-64

Table 85: France Current & Future Analysis for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications - Independent Analysis of Annual Sales in US$ Thousand for the Years 2020 through 2027 III-65

Table 86: France Historic Review for Cell Harvesting by Application - Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications Markets - Independent Analysis of Annual Sales in US$ Thousand for Years 2012 through 2019 III-66

Table 87: France 15-Year Perspective for Cell Harvesting by Application - Percentage Breakdown of Value Sales for Peripheral Blood, Bone Marrow, Umbilical Cord, Adipose Tissue and Other Applications for the Years 2012, 2020 & 2027 III-67

Excerpt from:
Global Cell Harvesting Market to Reach US$381,4 Million by the Year 2027 - Salamanca Press

Tanya Siddiqi, MD, Discusses the Promise of Reduced Toxicity With Liso-Cel – AJMC.com Managed Markets Network

In addition, liso-cels distinct manufacturing process creates a defined composition of CD8+ and CD4+ T-cells, which may reduce product variability; however, the manufacturer states, the clinical significance of defined composition is unknown.

For insights on what the arrival of liso-cel could mean in the treatment landscape, The American Journal of Managed Care (AJMC) turned to Tanya Siddiqi, MD, director of the Chronic Lymphocytic Leukemia Program at Toni Stephenson Lymphoma Center and associate clinical professor, Department of Hematology & Hematopoietic Cell Transplantation at City of Hope, Duarte, California.

Siddiqi was an investigator for ZUMA-1, which led to the approval of axicabtagene ciloleucel(axi-cel), sold as Yescarta, and the TRANSCEND NHL trial for liso-cel.She has addressed major scientific meetings on the challenge of managing the toxicities associated with CAR T-cell therapyand discussed how liso-cel represents a step forward over its predecessors.

This interview, conducted before the BMS announcement, has been edited for clarity and length.

AJMC: We're anticipating an FDA decision on liso-cel before the end of the year. Can you discuss the need of the patients who would take this new CAR T-cell therapy?

Siddiqi: So, for CAR T-cell therapy targeting CD19-positive B-cell lymphomasspecifically aggressive B-cell lymphomaswe already have a couple of FDA-approved options. The question is: what is liso-cel? How is it different? Why would people pick this over other things? In the trials that we've conducted, we found that liso-cel seems to have lesser toxicity in terms of the specific CAR T-cell side effects of cytokine release syndrome or hyper-inflammation, as well as neurotoxicity. We've just seen fewer severe adverse events so much so that at some [cancer] centers across the country, weve been able to give liso-cel CAR T-cells to patients in the clinic or outpatient setting rather than having to admit them to the hospital , depending on the patient's situation.

Those are the strengths of liso-celless toxicity and thus, a better chance of giving it in the outpatient setting with hospital admission available to anyone who develops a fever or other side effects. This means fewer days of inpatient hospitalization for these patients, so it may be less costly overall. I dont think the efficacy is necessarily differentmeaning that it seems to work as well as the other FDA-approved products already commercially available. But for the reasons that I've listed, I think it might be a very good option for older patients, maybe patients who are bit more frail, or younger patients who just don't want to be admitted to the hospitalthey just want to try to do it in the outpatient setting.

AJMC:You touched on this already, but can you discuss how Iiso-cel differs from earlier CAR T-cell therapiesboth in the way it's manufactured and how it works, and what that reduced variability means for patients?

Siddiqi: Liso-celis manufactured in a way that it gives very precise, equal numbers of CAR cells that are labeled CD4 and CD8, in a 1:1 ratio. All of us have T cells to fight infections with, and these T cells are what we take from patients. Then, we modify them in the lab by genetic engineering in order to produce CAR T-cells so that now instead of looking for infections, these CAR T cells are going to look for B-cell lymphoma cells and fight lymphoma.

The other products are given back to patients as a bag of CAR T cells mixed with potentially varying ratios of different types of T cellsCD4+, CD8+, etc. With liso-cel the manufacturing process actually separates out the CD4+ and CD8+ types of T cells first, and then manufactures CAR-T cells out of them separately. So, when we give the cells back to patients, we give it in a 1:1 ratio of CD4+ and CD8+ cells. We know exactly how many CD4+ and how many CD8+ T-cells these patients receive. And the thought is, the researchers and the drug manufacturer feel that this helps to have an expectation of what expansion you will have of these cells in the body.

Therefore, we potentially have an idea of what type of side effects or how severe the side effects might be. It may limit some of those side effects, or at least make them a little bit more predictable or controlled.

AJMC:Thats a great way to shift to your own work on length of stay due to CRS. What do we know about the key variables in determining whether a patient will experience a side effect that requires an extended stay in the hospital, and can more be done to avoid lengthy hospital stays?

Siddiqi: That's a very important question. Because lengthy hospital stays, especially in the [intensive care unit], really adds to the bill and the financial burden of these treatments. We know that people who have a big burden of disease going into CAR T-cell therapy, meaning they have a lot of lymphoma in their bodies, they tend to be at higher risk for more side effects like cytokine release syndrome and neurotoxicity. Probably because there's so much inflammation thats generated while these CAR T-cells are trying to fight the lymphoma. What we know is that people who come to us for CAR T-cells with lesser disease might have fewer side effects potentially and a better overall outcome.

So, we always try to advise our referring physicians, and educate patients, at conferences, to try to send these patients to us before they are at the end of the linebefore theyve tried and failed everything, and now theres just rampant disease. [At that point,] you're dealing with a situation where the patient is going to have more side effects and will not be able to tolerate the CAR T cells as well. Instead, if they fail two lines of therapy and the disease is still small in volume, but it's starting to progress, we can treat them more effectively with CAR T cells and with fewer side effects potentially.

AJMC:That brings up the next topicthere have been discussions that CAR T-cell therapy should be given earlier during treatment. As you said, if its not given as the last resort, patients might respond better. Where do you see those patterns heading in the future? And would that be truer for some patients than others?

Siddiqi: With aggressive diffuse large B-cell lymphoma, there's about a 60% to 70% chance of curing that in the frontline setting. With the line of chemo-immunotherapy, you can cure 60% to 70% of patients so that it never comes back. But the rest of themwhen it just relentlessly keeps coming back and it's hard to cureonce those patients relapse they tend to keep relapsing. So, our mainstay in the relapse setting is to give them salvage chemo-immunotherapy, collect stem cells, and take them to autologous stem cell transplantation if they've achieved a remission with the salvage chemotherapy. If they haven't achieved remission with that salvage chemotherapy, then they should go on to CAR T cells directly instead of waiting and trying more and more chemotherapies. After failing second line therapy, the FDA approval allows us to try CAR T cells. There are studies that are now ongoing that are comparing CAR T cells to autologous stem cell transplantation after failing first line therapy. So, once patients relapse the first time, these studies are comparing giving them salvage chemotherapy and transplant, versus taking them straight to CAR T cells. Once we have that data, we'll know better whether we can do CAR T cells even earlier in the lines of therapy.

AJMC:Weve been hearing for some time more about allogeneic or off-the-shelf therapies. What progress has been made on in that technology?

Siddiqi: I'm not too involved with these trials myself, but I know we have trials at City of Hope that are ongoing with off-the-shelf therapy. What I can tell you is that it's very attractive in that you don't have to collect T cells from patients, keeping their lymphoma under control while these T cells then go to the lab and CAR T cells are manufactured in 2-4 weeks depending on which product it is, and then they come back and get infused. With off-the-shelf products, you can just grab it and go as soon as you know the patient needs it.

The initial concerns were because the cells are not from the patient themselvesthe cells are from donors. Across the board there might be concerns of rejection and what's called graft-versus-host disease and things like that. So far, I don't think in the trial they've come up with such side effects to any significant extent. What I don't know is whether they've come up with a good result yet. Is it looking like the benefits of taking off-the-shelf CAR T cells are as good as autologous CAR T cells, meaning patients own CAR T cells? I think that remains to be seen. If they are, then it's much easier to use off-the-shelf CAR T cells. Maybe at the American Society of Hematology annual meeting in December we will see more data.

AJMC: How is COVID-19 affecting the clinical trial process for CAR T cell therapy?

Siddiqi: When the pandemic kind of started surging early in the year, and when we went into lockdown mode from March onward, we and other centers across the country took a lot of steps to slow down our clinical trial enrollment. Our staff started staggering who would come into work which day of the week and who could work from home. For those in the clinical trials office, there was a lot of need for safety and logistical reasons for us to slow down enrollment onto clinical trials. And there were other questions, such as, who would take care of patients at home once we discharged them after they received CAR T cells? What if their caregivers were exposed and got sick? Logistically, it was difficult to safely do many trials, especially CAR T cell trials and transplants earlier in the year.

Since the end of summer, we ramped up again, and we're now doing as many transplants and CAR T cells as we were probably doing last year. So, we're pretty much all the way up again, but I don't know how this winter will go because COVID is surging again.

As far as just CAR T cells themselves, we had to also think about travel for the cells because Juno Therapeutics is in Seattle, and Kite Pharma is here in Los Angeles, but Novartis is elsewhere. Just the movement of these cells was a concern because of travel restrictions during COVID-19. But as far as I know, the companies did not lose that commitmentthey told us, well get the cells to you, we will find a way to do it. I don't think any patients went without cells who should have received cells.

AJMC: What advice do you have for community oncologists interested in CAR T cell therapy for their patients?

Siddiqi: Theres good news for community physicians. We may soon have a therapeutic option of liso-cel CAR T cell therapy which seems to have lesser side effects. So, this might make things cheaper due to less need for hospitalization potentially without compromising the chance of cure. We want these patients to try CAR T cell therapy sooner rather than later in their relapses. You can always try multiple cycles of chemotherapy at some other time if you relapse again, but if you can be cured with CAR T cells such that you never need treatment again, why not try that first? For the patients who respond well to CAR T cells, the treatment works extremely well. And that's the Holy Grail to find the cure for all patients.

Maybe only half the patients will currently have a very good and durable responsebut those patients may never relapse again. So why not try it sooner rather than later? And of course, we're always looking for trial patients, because now we need to improve these results even further. So, community oncologists should also refer for trials, because I think that its very important to have trials with different combinationsCAR T cells plus another immunotherapy agentto see if we can improve upon the response rates even more.

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Tanya Siddiqi, MD, Discusses the Promise of Reduced Toxicity With Liso-Cel - AJMC.com Managed Markets Network

Tetracycline-based Antibiotics Show Promise for Combating Zika Infections – Global Biodefense

A widely used antibiotic methacycline was shown effective at preventing brain infections and reducing neurological problems associated with Zika virus in mice models.

National Institutes of Health (NIH) researchers used advanced drug screening techniques to test out more than 10,000 compounds in search of Zika virus therapeutics. The scientists discovered that the widely used antibiotic methacycline was effective at preventing brain infections and reducing neurological problems associated with the virus in mice. Additionally, they found that drugs originally designed to combat Alzheimers disease and inflammation may also help fight infections.

In this study, the researchers looked for drugs that prevent the virus from reproducing by blocking the activity of a protein called NS2B-NS3 Zika virus protease. The Zika virus is a protein capsule that carries long strings of RNA-encoded instructions for manufacturing more viral proteins. During an infection, the virus injects the RNA into a cell, resulting in the production of these proteins, which are strung together, side-by-side, like the parts in a plastic model airplane kit. The NS2B-NS3 protease then snaps off each protein, all of which are critical for assembling new viral particles.

The study was a collaboration between scientists on Dr. Naths team and researchers in laboratories led by Anton Simeonov, Ph.D., scientific director at the NIHs National Center for Advancing Translational Sciences (NCATS) and Radhakrishnan Padmanabhan, Ph.D., Professor of Microbiology & Immunology, Georgetown University Medical Center, Washington, D.C.

The Zika virus is primarily spread by the Aedes aegypti mosquito. In 2015 and 2016, at least 60 countries reported infections. Some of these countries also reported a high incidence of infected mothers giving birth to babies born with abnormally small heads resulting from a developmental brain disorder called fetal microcephaly. In some adults, infections were the cause of several neurological disorders including Guillain-Barr syndrome, encephalitis, and myelitis. Although many scientists have tried, they have yet to discover an effective treatment or vaccination against the virus.

Proteases act like scissors. Blocking protease activity is an effective strategy for counteracting many viruses, said Rachel Abrams, Ph.D., an organic chemist in Dr. Naths lab and the study leader. We wanted to look as far and wide as possible for drugs that could prevent the protease from snipping the Zika virus polyprotein into its active pieces.

To find candidates, Dr. Abrams worked with scientists on Dr. Simeonovs and Dr. Padmanabhans teams to create assays, or tests, for assessing the ability of drugs to block NS2B-NS3 Zika virus protease activity in plates containing hundreds of tiny test tubes. Each assay was tailored to a different screening, or sifting, technique. They then used these assays to simultaneously try out thousands of candidates stored in three separate libraries.

One preliminary screen of 2,000 compounds suggested that commonly used, tetracycline-based antibiotic drugs, like methacycline, may be effective at blocking the protease.

Meanwhile, a large-scale screen of more than 10,000 compounds helped identify an investigational anti-inflammatory medicine, called MK-591, and a failed anti-Alzheimers disease drug, called JNJ-404 as potential candidates. A virtual screen of over 130,000 compounds was also used to help spot candidates. For this, the researchers fed the other screening results into a computer and then used artificial intelligence-based programs to learn what makes a compound good at blocking NS2B-NS3 Zika virus protease activity.

These results show that taking advantage of the latest technological advances can help researchers find treatments that can be repurposed to fight other diseases, said Dr. Simeonov.

The Zika virus is known to preferentially infect stem cells in the brain. Scientists suspect this is the reason why infections cause more harm to newborn babies than to adults. Experiments on neural stem cells grown in petri dishes indicated that all three drugs identified in this study may counteract these problems. Treating the cells with methacycline, MK-591, or JNJ-404 reduced Zika virus infections.

Because tetracyclines are U.S. Food and Drug Administration-approved drugs that are known to cross the placenta of pregnant women, the researchers focused on methacycline and found that it may reduce some neurodevelopmental problems caused by the Zika virus. For instance, Zika-infected newborn mice that were treated with methacycline had better balance and could turn over more easily than ones that were given a placebo. Brain examinations suggested this was because the antibiotic reduced infections and neural damage. Nevertheless, the antibiotics did not completely counteract harm caused by the Zika virus. The weight of mice infected with the virus was lower than control mice regardless of whether the mice were treated with methacycline.

These results suggest that tetracycline-based antibiotics may at least be effective at preventing the neurological problems associated with Zika virus infections, said Dr. Abrams. Given that they are widely used, we hope that we can rapidly test their potential in clinical trials.

Therapeutic Candidates for the Zika Virus Identified by a High Throughput Screen for Zika Protease Inhibitors.PNAS, November 23, 2020

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Study Suggests AYAs Treated for AML Have High Risk of Developing Long-Term Complications – Cancer Network

Study results published in the International Journal of Epidemiology suggested that adolescent and young adult (AYA) patients treated for acute myeloid leukemia (AML) have a high risk of developing long-term health complications.1

The most common complications, or late effects as they are referred to in the study, observed among AYA survivors in this study were cardiovascular, endocrine, and respiratory diseases. Moreover, these complications were found to be more prevalent among non-white AYA patients and those living in more impoverished communities.

Our study shed light on the high burden of late effects among young survivors of AML, lead author Renata Abraho, MD, MSc, PhD, a postdoctoral fellow at theUC Davis Comprehensive Cancer Centerand the Center for Healthcare Policy and Research, said in a press release.2

To estimate the cumulative incidence and investigate the main predictors of late effects among this patient population, researchers identified 1168 eligible AYAs with AML who survived at least 2years after diagnosis from 1996 to 2012 in the California Cancer Registry. Importantly, late effects were reported from State hospital discharge data, and patients were followed through 2014.

Ultimately, the most common late effects reported at 10years after diagnosis were endocrine (26.1%), cardiovascular (18.6%), and respiratory (6.6%), followed by neurologic (4.9%), liver/pancreatic (4.3%), renal (3.1%), avascular necrosis (2.7%), and second primary malignancies (2.4%). Moreover, of the total study cohort, 547 (46.8%) received a hematopoietic stem cell transplant (HSCT).

Following multivariable adjustments, the investigators found that AYAs who underwent HSCT or had non-favorable risk AML experienced an approximately 2-fold or higher increased likelihood of all late effects. Additionally, AYAs of Hispanic, Black, or Asian/Pacific Islander race or ethnicity and those who resided in lower socio-economic neighborhoods were shown to be at higher risk of numerous late effects when compared with non-Hispanic White patients.

This higher risk may relate to the financial hardship that patients with cancer often experience, senior author Theresa Keegan, PhD, MS, associate professor at the UC Davis Comprehensive Cancer Center, said in a press release. As a result of cancer, AYA survivors and their families may miss work, experience income loss, and incur substantial out-of-pocket expenses.

According to the researchers, many factors may have led to the observed disparities in disease burden. These include differences in therapeutic management, patients response to treatment, AML with high-risk mutations, coexisting diseases, and socioeconomic factors.

Further, compared to younger or older cancer survivors, the investigators indicated AYA patients suffer a higher financial burden. They may go without treatment and long-term follow-up visits that could mitigate the impact of late effects. Moreover, their risk of late effects may be exacerbated by unhealthy lifestyle habits such as smoking, excessive alcohol consumption, lack of exercise, non-protected sun exposure, and poor diet.

Our findings underscore the need for long-term surveillance for the prevention, early detection and treatment of late effects, and can inform the development of AYA-focused consensus-based guidelines that will ultimately improve the quality of life and survival of these young vulnerable patients, the study authors wrote.

References:

1. Abraho R, Huynh JC, Benjamin DJ, et al. Chronic medical conditions and late effects after acute myeloid leukaemia in adolescents and young adults: a population-based study. International Journal of Epidemiology. doi: 10.1093/ije/dyaa184

2. Young survivors of acute myeloid leukemia have long-term complications from treatment [news release]. Published November 9, 2020. Accessed November 17, 2020. https://www.newswise.com/articles/young-survivors-of-acute-myeloid-leukemia-have-long-term-complications-from-treatment?sc=sphr&xy=10019792

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Study Suggests AYAs Treated for AML Have High Risk of Developing Long-Term Complications - Cancer Network

Mustang Bio Announces Positive Opinion from the European Medicines Agency on Orphan Drug Designation for Its Lentiviral Gene Therapy for the Treatment…

November 24, 2020 08:00 ET | Source: Mustang Bio, Inc.

WORCESTER, Mass., Nov. 24, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the European Commission (EC) issued a positive opinion on its application for Orphan Drug Designation for Mustangs lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease. The Designation applies both to MB-107 for the treatment of newly diagnosed infants between two months and two years of age and to MB-207 for the treatment of patients who have been previously treated with hematopoietic stem cell transplantation (HSCT) and for whom re-treatment is indicated. The European Medicines Agency (EMA) previously granted Advanced Therapy Medicinal Product classification to MB-107 in April 2020. The U.S. Food and Drug Administration (FDA) also previously granted Rare Pediatric Disease and Orphan Drug Designations to MB-107 and MB-207, as well as Regenerative Medicine Advanced Therapy Designation to MB-107.

Orphan Drug Designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the European Medicines Agency Committee for Orphan Medicinal Products (EMA COMP). To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA Orphan Drug Designation provides companies with certain benefits and incentives, including protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and 10 years of market exclusivity.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are very pleased to receive a positive opinion from the EC on Orphan Drug Designation for our lentiviral gene therapy for XSCID. It is an important milestone for Mustang as we approach the initiation of our pivotal MB-107 and MB-207 clinical trials, which we anticipate will support regulatory filings in both the U.S. and EU. We look forward to working closely with the EMA as we continue our progress to make MB-107 and MB-207 available for patients suffering with XSCID.

MB-107 is currently being assessed in a Phase 1/2 clinical trial for XSCID in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital (St. Jude), UCSF Benioff Childrens Hospital in San Francisco and Seattle Childrens Hospital. Mustang submitted an investigational new drug application (IND) to the FDA to initiate a pivotal multi-center Phase 2 clinical trial of MB-107 in this same patient population. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multi-center trial led by St. Jude, will be compared with 25 matched historical control patients who have undergone HSCT. The primary efficacy endpoint will be event-free survival. The initiation of this trial is expected soon. Mustang is targeting topline data from this trial in the second half of 2022.

Earlier this month, Mustang signed an agreement with Minaris Regenerative Medicine GmbH (Minaris), a leading contract development and manufacturing service provider for the cell and gene therapy industry, to enable technology transfer and GMP clinical manufacturing of Mustangs MB-107 lentiviral gene therapy program for the treatment of XSCID in newly diagnosed infants in Europe. Under the terms of the agreement, Minaris will perform technology transfer of the manufacturing and analytical processes, as well as their adoption to the European regulatory environment, for the GMP-compliant manufacturing of the drug product at its site in Ottobrunn, Germany, with the goal of supplying clinical trials in Europe.

MB-207 is currently being assessed in a Phase 1/2 clinical trial at the National Institute of Allergy and Infectious Diseases for XSCID patients who have been previously treated with HSCT and for whom re-treatment is indicated. Mustang expects to file an IND with the FDA to initiate a pivotal multi-center pivotal Phase 2 clinical trial of MB-207 in this same patient population in the first quarter of 2021 and is targeting topline data from this trial in the second half of 2022.

About X-linked Severe Combined Immunodeficiency (XSCID) X-linked severe combined immunodeficiency is a rare genetic disorder that occurs in approximately 1 per 225,000 births. It is characterized by the absence or lack of function of key immune cells, resulting in a severely compromised immune system and death by one year of age if untreated. Patients with XSCID have no T-cells or natural killer cells. Although their B-cells are normal in number, they are not functional. As a result, XSCID patients are usually affected by severe bacterial, viral or fungal infections early in life and often present with interstitial lung disease, chronic diarrhea and failure to thrive.

The specific genetic disorder that causes XSCID is a mutation in the gene coding for the common gamma chain (c), a protein that is shared by the receptors for at least six interleukins. These interleukins and their receptors are critical for the development and differentiation of immune cells. The gene coding for c is known as IL-2 receptor gamma, or IL2RG. Because IL2RG is located on the X-chromosome, XSCID is inherited in an X-linked recessive pattern, resulting in almost all patients being male.

About Mustang Bio Mustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (SEC). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.

Company Contacts: Jaclyn Jaffe and William Begien Mustang Bio, Inc. (781) 652-4500 ir@mustangbio.com

Investor Relations Contact: Daniel Ferry LifeSci Advisors, LLC (617) 430-7576 daniel@lifesciadvisors.com

Media Relations Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com

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Mustang Bio Announces Positive Opinion from the European Medicines Agency on Orphan Drug Designation for Its Lentiviral Gene Therapy for the Treatment...