On Aug. 4, Hutchinson Regional Medical Center will open the Hutchinson Hematology and Oncology clinic. Dr. Sawsan Amireh will join the Hutch Regional team that day and see her first patients at her clinic which will be located in Suite 4 of Doctors Park at 2100 N. Waldron St., a building located to the southwest of the main hospital campus on the east side of Waldron Street.

Hematology and Oncology are two branches of medicine that continue to be a concern of members of our community and throughout the State of Kansas.

Oncology is the branch of medical science dealing with tumors, including the diagnosis and treatment of cancer. More than 1.8 million Americans, including 16,000 Kansans, are diagnosed with cancer each year. For more than 600,000 of our nations population each year, cancer is a terminal disease. The good news is that 17 million Americans are living out their lives as cancer survivors.

Cancer is best defined as the uncontrolled growth and spread of abnormal cells and the cause is often attributed to everything from heredity to an unhealthy lifestyle including tobacco use and excess weight. Treatments are medical, surgical, Chemotherapy and Radiation.

Hematology is the branch of medicine dealing with the cause, treatment and prevention of disease related to the blood. Specific issues dealt with include treatment of bleeding disorders, Lymphoma and Leukemia and blood transfusions, along with bone marrow and stem cell transplants.

Dr. Amireh, who has received training on the latest treatment protocols, arrives at Hutch Regional with impeccable credentials and is Board Certified in Internal Medicine. She received her doctorate degree in 2011 from the University of Jordan School of Medicine in Amman, Jordan. Since 2017, Dr. Amireh has participated in a Hematology and Oncology Fellowship and also served as chief fellow with the State University of New York Downstate Health Science Center in Brooklyn, New York. Also, Dr. Amireh served as Associate Chief Medical Resident and Internal Medical Resident at Saint Michaels Medical Center in New Jersey.

In 2010, Dr. Amireh completed special training at Vanderbilt University Medical Center in Nashville, Tennessee on Hepatology and Pediatric Endocrinology. She has won numerous awards and honors including the Clinical Documentation Excellence Award from St. Michaels Medical Center, the Academic Excellence Award from the University of Jordan School of Medicine, and the English Talented Writing Award from the University of Jordan.

Dr. Amireh is a member of the American Society of Clinical Oncology, American Society of Hematology, American College of Physicians, and the Jordan Medical Association.

Dr. Amireh said Oncology has entered a new era with the onset of COVID-19. "Cancer patients are now more vulnerable and will need to be more diligent to avoid infection which proves that life is more fragile than it once was," Dr. Amireh said. "I am looking forward to starting a hospital-based practice. We have a young family and southcentral Kansas is a good place to raise a two-year old. New York is too busy and not really family friendly."

"I want my practice to be inclusive and will welcome everyone who needs treatment and care," Dr. Amireh continued.

Appointments with Dr. Amireh will be accepted starting in Mid-July. Additional information including her clinics contact information will be published at that time.

With the opening of Hutchinson Hematology and Oncology, south central and western Kansas will have a new choice for quality cancer care. Dr. Amireh will provide a caring, personal approach to cancer care and her clinic will work in tandem with the Chalmers Cancer Center and the other Hutchinson Regional Healthcare System entities as well as other physician specialties in Hutchinson and surrounding communities to provide an integrated approach to care in Hutchinson.

Dr. Amireh will join her spouse, Dr. Mohammed "Mo" Al-Halawani at Hutch Regional. In July 2019, Dr. Al-Halawani joined Dr. Tariq Musa in opening Hutchinson Pulmonology Associates at Hutchinson Regional Medical Center. Dr. Al-Halawani and Dr. Musa hit the ground running and are serving patients in Hutchinson as well as an outreach clinic in McPherson, with plans to support pulmonary needs locally and in the surrounding areas.

We are excited that these high caliber healthcare professionals have chosen to move across America to provide their expertise to residents on the plains of Kansas.

Ken Johnson is the President and CEO of Hutchinson Regional Medical Center.

Continue reading here:
HRMC to open hematology and oncology clinic - The Ottawa Herald

Bone Marrow Stem Cells Comments Off on HRMC to open hematology and oncology clinic – The Ottawa Herald | June 29th, 2020

By: Lifestyle Desk | New Delhi | Updated: June 27, 2020 12:09:31 pm Heres everything to know about the blood disorder. (Source: Getty Images/Thinkstock)

Sickle cell disease (SCD) is an inherited group of blood disorder which is genetic in nature. It is usually transferred from parents to the child during birth i.e. both the parents can be carries of SCD. Healthy RBCs are round in shape, which moves through small blood vessels and carries oxygen to all parts of the body. In someone who has SCD, the RBC becomes sticky and hard and start to look like C- shaped similar to that of a farm tool sickle. The sickle cells die early, which causes a constant shortage of RBC, which leads to low oxygen carriers in the body, mentioned Dr Rahul Bhargava, director and head, haematology and bone marrow transplant, Fortis Memorial Research Institute, Gurugram. Also, when sickle cells travels they tend to get stuck in small blood vessels and restrict the blood flow. This can cause pain in the joints, chest and other serious problems such as stroke, acute chest syndrome and infections.

Anaemia

SCD can lead to shortage of RBC which makes the patient anaemic. It can lead to less oxygen supply in the body causing fatigue.

Episodes of pain. SCD patients suffer from episodes of periodic pain. Sickle-shaped blood cells can block the flow of blood which in tiny vessels of abdomen, chest and joints, thus leading to pain.

Feet and hands swelling

Sickle-shaped RBC can block then flow of blood which can cause swelling in the hands and feet.

Frequent infections

Sickle cells can damage the spleen and make the person vulnerable to diseases and infections. Vaccination is usually prescribed in childhood to keep infections at bay.

Delayed growth or puberty

RBC is the carrier of oxygen and other nutrients. With low RBC, there is no enough oxygen in the body which can slow the growth in infants and children which leads to delayed puberty in children.

Vision problems

Vision is affected when the flow of blood is affected.

SCD can lead to a lot of complication, including Stroke, acute chest syndrome, pulmonary hypertension, organ damage, blindness, leg ulcers and more such complications.

SCD can be diagnosed with a simple blood test. Parents are advised to get the screening done before conceiving as it is transmitted from parents. For newborns, the test is done in the hospitals as a routine screening. There is no single treatment for SCD patients and it varies from patient to patient on the basis the symptoms. Before starting any medication, consult a haematologist who specialises in blood disorders.

The only cure for SCD is getting a bone marrow transplant (BMT), which is a procedure to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Stem cells are easily taken from the donor and planted in the recipient. After the BMT procedure, the patient can lead a healthy and normal life.

The Indian Express is now on Telegram. Click here to join our channel (@indianexpress) and stay updated with the latest headlines

For all the latest Lifestyle News, download Indian Express App.

IE Online Media Services Pvt Ltd

Read more:
Sickle Cell Disease: All you need to know - The Indian Express

Bone Marrow Stem Cells Comments Off on Sickle Cell Disease: All you need to know – The Indian Express | June 29th, 2020

A six-year-old who won the nations hearts in his battle with leukaemia has been given a heroes welcome after flying home following months of life-saving cancer treatment in Singapore.

Brave Oscar Saxelby-Lee from St Johns, Worcester, was diagnosed with an aggressive blood cancer in December 2018 after his parents noticed unusual bruising on his legs.

Doctors gave him months to find a stem cell match to save his life which led to parents Olivia Saxelby, 25, and Jamie Lee, 28, launching a UK-wide appeal for donors.

Last March a record-breaking 4,855 volunteers queued in the freezing rain after Oscars primary school in opened as a testing centre.

In total more than 10,000 people were tested across the UK until a match was finally found.

Read more: Mum-to-be plagued by headaches diagnosed with incurable brain tumour

But just months later, the family faced further anguish after discovering Oscars cancer had returned.

After a crowdfunding campaign raised 600,000, Oscar was flown to Singapore for pioneering treatment not available on the NHS.

Oscar became only the second child in the world to undergo CAR-T therapy before having a second bone marrow transplant using stem cells from dad Jamie.

Oscar Saxelby-Lee, with his mum and dad during his time having treatment in Singapore. (SWNS)

Just three months after the successful operation, Oscar and his parents were given the all clear to return to the UK and hours after touching down he was driven see his classmates, who cheered as he drove past the school gates.

In heartwarming pictures Oscar grins from ear to ear as his proud dad sits next to him and his mum breaks down in tears of joy.

Pupils gave Oscar a huge bunch of colourful balloons which the youngster struggled to fit inside the car.

Read more:Woman diagnosed with cancer 48 hours before giving birth

Jen Kelly, from the Grace Kelly Trust which helped raise 173,000 to fund his life-saving treatment, said: The Grave Kelly Trust is delighted to see Oscar returning home to the UK with his mum and dad.

Out smiles have been growing ever wider as we watched Oscars miraculous journey.

We are so proud to have been able to play a key part in Oscars bid for life-saving treatment in Singapore and it is wonderful to see Oscar doing so well following his ground-breaking treatment.

This news is amazing, not just for Oscar, but potentially for other children in a similar position in the future.

Oscar still has a way to go and will need to be shielded for a good while longer once he gets home, but he remains cancer free and smiling.

Read more:Parents spot cancer in babys eye after photo taken

Oscar leaves Hospital in Singapore. (SWNS)

Story continues

Throughout his treatment, Oscar has kept in touch with his classmates and teachers from his hospital bed via an innovative robot - nicknamed Ozzybot.

His headteacher Kate Wilcock, who organised the huge donor registration event and Ozzybot lessons, said: There have been plenty of ups and downs to get here but this is the absolute icing on the cake that hes come home, cancer free and its worked.

Our future plans now will be to support Oscar and his family and even start some learning at home.

Then we will be working really closely with Olivia, Jamie and Oscar for some transition into school.

That will be the best day when he walks back through the door at Pitmaston Primary.

Oscar made a surprise visit to his classmates on his return from Singapore. (SWNS)

When he left hospital for the final time in Singapore Ozzy, as he is affectionately nicknamed, was given a guard of honour by medical staff.

A video posted on the Hand in Hand for Oscar Facebook page, shows the youngster beaming as he is driven away and shouts: Bye hospital, thank you!

Read more: Six-year-old runs marathon for the NHS dressed as his favourite super-heroes

His thrilled mum Olivia wrote: Cant quite believe it, crying bucket loads! The day weve dreamt of has now become a reality!

Were actually bringing him home!!!

You are our HERO Ozzy Bear, you truly truly are the most amazing miracle.

Enjoy every last minute of this journey darling, because YOU have owned [sic] it!

Additional reporting SWNS.

View original post here:
Six-year-old given heroes welcome by his classmates after months of life-saving cancer treatment in Singapore - Yahoo Singapore News

Bone Marrow Stem Cells Comments Off on Six-year-old given heroes welcome by his classmates after months of life-saving cancer treatment in Singapore – Yahoo Singapore News | June 29th, 2020

There are so many skincare brands on the market today, their number may flood your social media feed that it may be difficult to choose the right one for you this summertime. Sensitive skin, aging, tired skin, fair, dark, or a combination, its particularly rare for a single product or brand that can nourish and protect you during this season. However, by using a variety of brands that suit you, you may achieve that glowing healthy skin youve always wanted.

Here is a list of 8 products and options that can help you obtain the perfect skin this summer:

This brand is inspired and designed by leading cosmeceutical scientists for skincare, anti-aging, removal of blemish, and other skin issues. Their products are clinically tested, which makes SkinPro represent the industrys advanced skincare technology. Its perfect for enjoying the sun during the day and experience the rejuvenating effects with strengthened efficacy while being sensitive to the skin. The formulations are made by proprietary technology to give you a special combination of active ingredients that will remedy skin problems and provide lasting results.

Pair your tan with skincare that keeps your skins aging process to a minimum while enjoying the sun. You can use the Nimni Day Cream to protect and smoothen your skin with special collagen boosting complex infused with environmental shields. Summer-soft touchable skin is truly achievable while preventing possible daytime sun damage. When the sun sets and before going to bed to close another day, use the Nimni Night Cream and take some time to yourself.

Caudalie is a French skincare brand, based on all-natural grape extracts from the Bordeaux, Champagne, and Burgundy regions. Red wine drinkers already know this to be true: grapes are high in antioxidants and resveratrol compounds and polyphenols which give the whole line its unique and successful philosophy of anti-aging.

The whole line of SK-II revolves on a special ingredient called Pitera. History has it that workers who were subjected to a fermentation substance at a Japanese factory have remarkably ageless and spotless palms. In a slew of anti-aging creams, serums, masks, and most famously a skin-brightening facial treatment essence the secret sauce was packaged up and shipped.

It is one of those skincare brands that you can apply by yourself or as an aftercare treatment based on your preferences and goals. CosmetiCare has everything you need to fix anti-aging, acne, or pigmentation so you can get a sun-radiant, healthy, vibrant complexion. Using their Amplify-Hyaluronic Acid Enhancing Serum to lighten your dry skin with moisture when strengthening the skins lipid barrier. It is especially good for your summer skin, which requires the extra boost from day to day activities to keep your skin moisturized.

Think of skincare product creativity! If youre hunting for skincare brands with coveted distinctions to be the best-selling brands, then youll meet Factor Five Skin here. With this best-selling serum, take your regimen to a whole new dimension that dares to deviate from conventional anti-aging products. Their Regenerative Serum works on wrinkles, sun spots, uneven texture, and more. Combining human stem cell growth factors with copper peptides will make your skin look renewed and restored in as little as 4 weeks!

Your time in the sun is not supposed to reveal the years of skin life. This youth-infusing bundle of skin-loving ingredients is nicely packaged in the Mature Skin Anti-Aging Kit to help you reverse or prevent premature aging. Turn back the time or stand still with this scientifically crafted and evidence-based ingredient combination. Prepare to erase the fine lines, wrinkles, and discolorations of the skin, and shrink large pores with this impressive collection of anti-aging skincare:

This summer, when you apply the Mature Skin Anti-Aging Kit to your regimen, your sun-kissed lips, eyes, and dcolletage region will be completely plumped up and beautifully radiant. Cap off the week with the Hydroxy Overnight Mask, an exfoliating and hydrating leave-on-night mask composed of salicylic acids.

If you tend to have skin that is prone to acne, oily, sun-damaged, or sensitive, then you will want to use this product a few times a week. The Aloe Veras soothing compounds help soothe drained, old skin cells when youre asleep! You can use it as a mask or even best as a spot-treatment for rare breakouts, and youd wake to clean and healthy skin.

Who wouldnt love skincare brands that make an utter joy in washing your face? Only the word Kale Cleanser makes you want to make every morning and evening a spa day in your house! Its a concentrated combination of superfoods and antioxidants that hydrate your skin while reducing oxidative damage to the blue light. Its so sweet, you should probably use it twice a day and wash the dirt and extra grime of the day away. Follow it up next with the Moisturizer Adaptogens Superfruits.

This yummy moisturizer feeds some of the most powerful anti-aging ingredients to amplify the natural development of collagen in your skin while helping to prevent free-radical damage. Then what better way to soothe your exposed skin during the summer than with the Adaptogens Superfruits Mask. The sun may be bright, but if it is not revitalized, it can quickly dull your skin. Its creamy, smooth, and not only smells great, but after just one try, it leaves your skin smooth and rejuvenated. Piper Berry is one of the skincare brands that we love for quick fixing and refreshing the skin anytime, anywhere.

With these products, youll be summer-ready to enjoy activities that you can only do when the suns out while protecting and nourishing your skin. If you have sensitive skin make sure to apply only a small amount and gradually increase when you dont show any signs of negative reaction. Investing in the protection and rejuvenation of your skin will surely hold off father time from getting his hands on your glowing face.

The rest is here:
Skincare Brands To Try Out This Summer - GirlTalkHQ

Skin Stem Cells Comments Off on Skincare Brands To Try Out This Summer – GirlTalkHQ | June 28th, 2020

Just like your skin, your hair also needs pampering to look good. For lustrous and long tresses, you need to provide your scalp and hair follicles with the essential nutrients including vitamins, minerals, and proteins. Though there are various supplements available in the market that can stimulate your hair growth, if you wish to go natural, there is a plethora of food items that can give you the same benefit. Read further to know about foods that can promote hair growth. Also Read - Want to Promote Hair Growth And Relieve Insomnia? Opt For Spikenard Essential Oil

Being rich in procyanidin B-2, apples can stimulate epithelial cells and promote hair growth. Also, the antioxidants present in this fruit can fight against the free radicals and reduce hair damage. Even if you wish to improve your hair thickness, you can have apples as they are packed with protein. Also Read - Potential stem cell therapy may help promote hair growth

Strawberries contain vitamin C and other strong antioxidants, that can improve the health of your hair. If anecdotal evidences are to be believed, silica present in strawberries can stop hair loss and prevent the onset of baldness. Eating strawberries every day can treat dandruff and also make your hair appear shiny. Also Read - Top 5 essential oils for hair growth and how to use them

Bananas can help in hair growth by preventing dandruff and improving the quality of your scalp. Its rich nutritional content can also unclog the scalp pores. If you want your hair to impart shine, have a banana every day. It can also prevent breakage and split-ends.

See more here:
Best Hair Growth Tips: 5 Food Items to Include in Your Daily Diet For Long And Strong Tresses - India.com

Skin Stem Cells Comments Off on Best Hair Growth Tips: 5 Food Items to Include in Your Daily Diet For Long And Strong Tresses – India.com | June 28th, 2020

Unfortunate news for those hoping the coronavirus pandemic was fading away: models suggest that an apocalyptic resurgence could be coming in the near future.

Multiple U.S. states, including Florida, Texas, and California, are currently experiencing record daily numbers of new COVID-19 cases, CNN reports. And they all still seem to be on the upswing.

Dr. Peter Hotez, dean of Baylor College of Medicines National School of Tropical Medicine, told CNN that Houston is on track to be the most coronavirus-ravaged city in the U.S. but that other Texas cities arent far behind.

The big metro areas seem to be rising very quickly and some of the models are on the verge of being apocalyptic, Hotez told CNN.

The three states hitting record numbers right now are also the most populous in the country. Combined, their new surges put more than 27 percent of the U.S. population at risk, CNN reports.

Hotez, whos also working on an experimental COVID-19 vaccine, warns that Houston in particular may quadruple its coronavirus case load over the next two weeks, which would put the same devastating strain on its healthcare system that places like New York City experienced earlier in the year.

That is really worrisome and as those numbers rise, were seeing commensurate increases in the number of hospitalizations and ICU admissions, Hotex told CNN. You get to the point where you overwhelm ICUs and thats when the mortality goes up.

Visit link:
Professor: Looming COVID Surge on "Verge of Being Apocalyptic" - Futurism

Skin Stem Cells Comments Off on Professor: Looming COVID Surge on "Verge of Being Apocalyptic" – Futurism | June 28th, 2020

This report will definitely help you make well informed decisions related to the stem cell market. The stem cell therapy market includes large number of players that are involved in development of stem cell therapies of the treatment of various diseases. Mesoblast Ltd. (Australia), Aastrom Biosciences, Inc. (U.S.), Celgene Corporation (U.S.), and StemCells, Inc. (U.S.) are the key players involved in the development of stem cell therapies across the globe.

The global stem cells market is expected to grow at an incredible CAGR of 25.5% from 2018to 2024and reach a market value of US$ 586 billion by 2025. The emergence of Induced Pluripotent Stem (iPS) cells as an alternative to ESCs (embryonic stem cells), growth of developing markets, and evolution of new stem cell therapies represent promising growth opportunities for leading players in this sector.

You Can Browse Full Report @: https://www.marketresearchengine.com/reportdetails/global-stem-cells-market-analysis-report

Due to the increased funding from Government and Private sector and rising global awareness about stem cell therapies and research are the main factors which are driving this market. A surge in therapeutic research activities funded by governments across the world has immensely propelled the global stem cells market. However, the high cost of stem cell treatment and stringent government regulations against the harvesting of stem cells are expected to restrain the growth of the global stem cells market.

The stem cell therapy market includes large number of players that are involved in development of stem cell therapies of the treatment of various diseases. Mesoblast Ltd. (Australia), Aastrom Biosciences, Inc. (U.S.), Celgene Corporation (U.S.), and StemCells, Inc. (U.S.) are the key players involved in the development of stem cell therapies across the globe.

This market research report categorizes the stem cell therapy market into the following segments and sub-segments:

The Global Stem Cell Market this market is segmented on the basis of Mode of Therapy, Therapeutic Applications and Geography.

By Mode of Therapy this market is segmented on the basis of Allogeneic Stem Cell Therapy Market and Autologous Stem Cell Therapy Market. Allogeneic Stem Cell Therapy Market this market is segmented on the basis of CVS Diseases, CNS Diseases, GIT diseases, Eye Diseases, Musculoskeletal Disorders, Metabolic Diseases, Immune System Diseases, Wounds and Injuries and Others. Autologous Stem Cell Therapy Market this market is segmented on the basis of GIT Diseases, Musculoskeletal Disorders, CVS Diseases, CNS Diseases, Wounds and Injuries and Others. By Therapeutic Applications this market is segmented on the basis of Musculoskeletal Disorders, Metabolic Diseases, Immune System Diseases, GIT Diseases, Eye Diseases, CVS Diseases, CNS Diseases, Wounds and Injuries and Others.

By Regional Analysis this market is segmented on the basis of North America, Europe, Asia-Pacific and Rest of the World.

Request Sample Report @: https://www.marketresearchengine.com/reportdetails/global-stem-cells-market-analysis-report

Table of Contents

1 INTRODUCTION

2 Research Methodology

2.1 Research Data2.1.1 Secondary Data2.1.1.1 Key Data From Secondary Sources2.1.2 Primary Data2.1.2.1 Key Data From Primary Sources2.1.2.2 Breakdown of Primaries2.2 Market Size Estimation2.2.1 Bottom-Up Approach2.2.2 Top-Down Approach2.3 Market Breakdown and Data Triangulation2.4 Research Assumptions

3 Executive Summary

4 Premium Insights

5 Market Overview

6 Industry Insights

7 Global Stem Cell Therapy Market, By Type

8 Global Stem Cell Therapy Market, By Therapeutic Application

9 Global Stem Cell Therapy Market, By Cell Source

10 Stem Cell Therapy Market, By Region

11 Competitive Landscape

12 Company Profiles

12.1 Introduction

12.1.1 Geographic Benchmarking

12.2 Osiris Therapeutics, Inc.

12.3 Medipost Co., Ltd.

12.4 Anterogen Co., Ltd.

12.5 Pharmicell Co., Ltd.

12.6 Holostem Terapie Avanzate Srl

12.7 JCR Pharmaceuticals Co., Ltd.

12.8 Nuvasive, Inc.

12.9 RTI Surgical, Inc.

12.10 Allosource

Other Related Market Research Reports:

Specialty Enzymes Market Size Worth US$ 5.5 Billion By 2022

Single Use Bioprocessing Market Size is Projected to be Around US$ 5.50 Billion By 2022

Single-cell Analysis Market is Expected to Grow US$ 3 Billion By 2022

Media Contact

Company Name: Market Research Engine

Contact Person: John Bay

Email: [emailprotected]

Phone: +1-855-984-1862

Country: United States

Website: https://www.marketresearchengine.com/

Excerpt from:
Global Stem Cells Market 2019 | How The Industry Will Witness Substantial Growth In The Upcoming Years | Exclusive Report By MRE - Cole of Duty

IPS Cell Therapy Comments Off on Global Stem Cells Market 2019 | How The Industry Will Witness Substantial Growth In The Upcoming Years | Exclusive Report By MRE – Cole of Duty | June 26th, 2020

A six-year-old who won the nations hearts in his battle with leukaemia has been given a heroes welcome after flying home following months of life-saving cancer treatment in Singapore.

Brave Oscar Saxelby-Lee from St Johns, Worcester, was diagnosed with an aggressive blood cancer in December 2018 after his parents noticed unusual bruising on his legs.

Doctors gave him months to find a stem cell match to save his life which led to parents Olivia Saxelby, 25, and Jamie Lee, 28, launching a UK-wide appeal for donors.

Last March a record-breaking 4,855 volunteers queued in the freezing rain after Oscars primary school in opened as a testing centre.

In total more than 10,000 people were tested across the UK until a match was finally found.

Read more: Mum-to-be plagued by headaches diagnosed with incurable brain tumour

But just months later, the family faced further anguish after discovering Oscars cancer had returned.

After a crowdfunding campaign raised 600,000, Oscar was flown to Singapore for pioneering treatment not available on the NHS.

Oscar became only the second child in the world to undergo CAR-T therapy before having a second bone marrow transplant using stem cells from dad Jamie.

Oscar Saxelby-Lee, with his mum and dad during his time having treatment in Singapore. (SWNS)

Just three months after the successful operation, Oscar and his parents were given the all clear to return to the UK and hours after touching down he was driven see his classmates, who cheered as he drove past the school gates.

In heartwarming pictures Oscar grins from ear to ear as his proud dad sits next to him and his mum breaks down in tears of joy.

Pupils gave Oscar a huge bunch of colourful balloons which the youngster struggled to fit inside the car.

Read more:Woman diagnosed with cancer 48 hours before giving birth

Jen Kelly, from the Grace Kelly Trust which helped raise 173,000 to fund his life-saving treatment, said: The Grave Kelly Trust is delighted to see Oscar returning home to the UK with his mum and dad.

Out smiles have been growing ever wider as we watched Oscars miraculous journey.

We are so proud to have been able to play a key part in Oscars bid for life-saving treatment in Singapore and it is wonderful to see Oscar doing so well following his ground-breaking treatment.

This news is amazing, not just for Oscar, but potentially for other children in a similar position in the future.

Oscar still has a way to go and will need to be shielded for a good while longer once he gets home, but he remains cancer free and smiling.

Read more:Parents spot cancer in babys eye after photo taken

Oscar leaves Hospital in Singapore. (SWNS)

Story continues

Throughout his treatment, Oscar has kept in touch with his classmates and teachers from his hospital bed via an innovative robot - nicknamed Ozzybot.

His headteacher Kate Wilcock, who organised the huge donor registration event and Ozzybot lessons, said: There have been plenty of ups and downs to get here but this is the absolute icing on the cake that hes come home, cancer free and its worked.

Our future plans now will be to support Oscar and his family and even start some learning at home.

Then we will be working really closely with Olivia, Jamie and Oscar for some transition into school.

That will be the best day when he walks back through the door at Pitmaston Primary.

Oscar made a surprise visit to his classmates on his return from Singapore. (SWNS)

When he left hospital for the final time in Singapore Ozzy, as he is affectionately nicknamed, was given a guard of honour by medical staff.

A video posted on the Hand in Hand for Oscar Facebook page, shows the youngster beaming as he is driven away and shouts: Bye hospital, thank you!

Read more: Six-year-old runs marathon for the NHS dressed as his favourite super-heroes

His thrilled mum Olivia wrote: Cant quite believe it, crying bucket loads! The day weve dreamt of has now become a reality!

Were actually bringing him home!!!

You are our HERO Ozzy Bear, you truly truly are the most amazing miracle.

Enjoy every last minute of this journey darling, because YOU have owned [sic] it!

Additional reporting SWNS.

Read this article:
Six-year-old given heroes welcome by his classmates after months of life-saving cancer treatment in Singapore - Yahoo Canada Sports

Bone Marrow Stem Cells Comments Off on Six-year-old given heroes welcome by his classmates after months of life-saving cancer treatment in Singapore – Yahoo Canada Sports | June 26th, 2020

Dana-Farber Patient Recovering Well After Cancer and the Coronavirus

Pam Dobay is a warrior. In the last three years, the 67-year-old has dealt with a cancer diagnosis and stem cell transplant before recently contracting the coronavirus.

None of it was easy, but today, Dobay is recovering at home. She says she cannot begin to express the gratitude she feels towards everyone who has cared for her, including her Dana-Farber care team and her family.

When this is all over, I want to show everyone at Dana-Farber what they did, and thank them for everything, says Dobay.

A Blood Cancer Diagnosis

In February 2018, Dobay was diagnosed with myelofibrosis, a blood disorder in which the bone marrow is unable to produce healthy red blood cells. Dobays primary care physician first worried something wasnt right after her test results from routine blood work came back abnormal. Myelofibrosis is a precursor condition for leukemia, meaning it puts those who are diagnosed at a much higher chance of developing the disease.

Dobay, who lives in Holbrook, MA, was placed under the care ofCorey Cutler, MD, MPH, medical director of theAdult Stem Cell Transplantation Programat Dana-Farber/Brigham and Womens Cancer Center. Initially, she was given blood transfusions to help her body compensate for the bone marrows inability to produce red blood cells. This treatment is not designed to be a permanent fix, despite being highly effective for a short period of time: Eventually, Dobay would need a bone marrow transplant.

In September 2018, just six months after her diagnosis, Dobay underwent areduced-intensity transplant(sometimes referred to as a mini-transplant). Mini-transplant patients receive lower doses of chemotherapy than are used in a full-intensity transplant, and in general, receive no radiation therapy. The reduced-intensity procedure was developed for older patients and others who often cant tolerate the harsh side effects of full-intensity treatments.

The procedure still proved to be difficult for Dobay, who ended up in the intensive care unit (ICU) due to complications. This was a possibility her care team had prepared for, and slowly, her condition improved. While she still has some symptoms of chronic graft-versus-host disease (GVHD), she and her family including Robert Dobay, her husband of 45 years hoped this would be her toughest test.

This article was originally published on June 18, 2020, by Dana-Farber Cancer Institute. It is republished with permission.

View original post here:
Recovering from Cancer, a Stem Cell Transplant and Coronavirus - Cancer Health Treatment News

Bone Marrow Stem Cells Comments Off on Recovering from Cancer, a Stem Cell Transplant and Coronavirus – Cancer Health Treatment News | June 26th, 2020

The Cell Therapy Manufacturing Market Research Report 2020 published by Prophecy Market Insights is an all-inclusive business research study on the current state of the industry which analyzes innovative strategies for business growth and describes significant factors such as top developers/manufacturers, production value, key regions, and growth rate. Impact of Covid-19 pandemic on the market will be completely analyzed in this report and it will also quantify the impact of this pandemic on the market.

The research study encompasses an evaluation of the market, including growth rate, current scenario, and volume inflation prospects, based on DROT and Porters Five Forces analyses. The market study pitches light on the various factors that are projected to impact the overall market dynamics of the Cell Therapy Manufacturing market over the forecast period (2019-2029).

Regional Overview:

The survey report includes a vast investigation of the geographical scene of the Cell Therapy Manufacturing market, which is manifestly arranged into the localities. The report provides an analysis of regional market players operating in the specific market and outcomes related to the target market for more than 20 countries.

Australia, New Zealand, Rest of Asia-Pacific

The facts and data are represented in the Cell Therapy Manufacturing report using graphs, pie charts, tables, figures and graphical representations helping analyze worldwide key trends & statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the market.

Get Sample Copy of This Report @ https://www.prophecymarketinsights.com/market_insight/Insight/request-sample/21

The research report also focuses on global major leading industry players of Cell Therapy Manufacturing market report providing information such as company profiles, product picture and specification, R&D developments, distribution & production capacity, distribution channels, price, cost, revenue and contact information. The research report examines, legal policies, and competitive analysis between the leading and emerging and upcoming market trends.

Cell Therapy ManufacturingMarket Key Companies:

harmicell, Merck Group, Dickinson and Company, Thermo Fisher, Lonza Group, Miltenyi Biotec GmBH, Takara Bio Group, STEMCELL Technologies, Cellular Dynamics International, Becton, Osiris Therapeutics, Bio-Rad Laboratories, Inc., Anterogen, MEDIPOST, Holostem Terapie Avanazate, Pluristem Therapeutics, Brammer Bio, CELLforCURE, Gene Therapy Catapult EUFETS, MaSTherCell, PharmaCell, Cognate BioServices and WuXi AppTec.

The predictions mentioned in the Cell Therapy Manufacturing market report have been derived using proven research techniques, assumptions and methodologies. This market report states the overview, historical data along with size, share, growth, demand, and revenue of the global industry.

Segmentation Overview:

The report provides an in-depth analysis of the Cell Therapy Manufacturing market segments and highlights the latest trending segment and major innovations in the market. In addition to this, it states the impact of these segments on the growth of the market. Apart from key players analysis provoking business-related decisions that are usually backed by prevalent market conditions, we also do substantial analysis of market based on COVID-19 impact, detailed analysis on economic, health and financial structure.

Request Discount @ https://www.prophecymarketinsights.com/market_insight/Insight/request-discount/21

Key Questions Answered in Report:

Stakeholders Benefit:

Get In-depth TOC @ https://www.prophecymarketinsights.com/market_insight/Global-Cell-Therapy-Manufacturing-Market-21

About us:

Prophecy Market Insights is specialized market research, analytics, marketing/business strategy, and solutions that offers strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high-value opportunities in the target business area. We also help our clients to address business challenges and provide the best possible solutions to overcome them and transform their business.

Contact Us:

Mr Alex (Sales Manager)

Prophecy Market Insights

Phone: +1 860 531 2701

Email: [emailprotected]

VISIT MY BLOG:- https://medium.com/@varshashirode0000/global-blockchain-in-genomics-market-trends-analysis-and-forecast-till-2029-3f92b8bdc622

Read the original post:
Cell Therapy Manufacturing Market: Opportunities Forecast and Value Chain 2020-2030 - 3rd Watch News

Bone Marrow Stem Cells Comments Off on Cell Therapy Manufacturing Market: Opportunities Forecast and Value Chain 2020-2030 – 3rd Watch News | June 26th, 2020

CRANFORD, N.J., June 26, 2020 /PRNewswire/ --Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, announced today that the Company has received a written response from the U.S. Food and Drug Administration (FDA) in regards to its pre-investigational new drug (PIND) application for its induced mesenchymal stem cells (iMSCs) to treat and reduce the severity of acute respiratory distress syndrome (ARDS) in patients with COVID-19.

The FDA acknowledged that the Company could apply for fast track designation and also provided Citius with the chemistry, manufacturing, and control (CMC) requirements for the proposed trials. The Company plans to initiate actions on the FDA's recommendations and follow up with the FDA with an Investigational New Drug (IND) application under the Coronavirus Treatment Acceleration Program (CTAP).

Myron Holubiak, Chief Executive Officer of Citius, commented, "We appreciate the FDA's thoughtful guidance on our unique, allogenic mesenchymal stem cells derived from induced pluripotent stem cells (iPSCs). We understand that iPSC-derived stem cells are not the same as adult-donor derived cells and, therefore, would require different proof of concept studies. Since we believe in the advantages of iPSC MSCs over donor-derived cells, we intend to develop assays recommended by the FDA and demonstrate the safety of these MSCs in our preclinical studies. We are committed to the successful completion of the required clinical trials to provide an effective and safe therapy for ARDS due to COVID-19."

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visitwww.citiuspharma.com.

About Citius iMSCCitius's mesenchymal stem cell therapy product is derived from a human induced pluripotent stem cell (iPSC) line generated using a proprietary mRNA-based (non-viral) reprogramming process. The iMSCs produced from this clonal technique are differentiated from adult donor-derived MSCs (bone marrow, placenta, umbilical cord, adipose tissue, or dental pulp) by providing genetic homogeneity. In in-vitro studies, iMSCs exhibit superior potency and high cell viability. The iMSCs secrete immunomodulatory proteins that may reduce or prevent pulmonary symptoms associated with acute respiratory distress syndrome (ARDS) in patients with COVID-19. The Citius iMSC is an allogeneic (unrelated donor) mesenchymal stem-cell product manufactured by expanding material from a master cell bank.

About Acute Respiratory Distress Syndrome (ARDS)ARDS is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

About Coronavirus Treatment Acceleration Program (CTAP)In response to the pandemic, the FDA has created an emergency program called the Coronavirus Treatment Acceleration Program (CTAP) to accelerate the development of treatments for COVID-19. By redeploying staff, the FDA is responding to COVID-19-related requests and reviewing protocols within 24 hours of receipt. The FDA said CTAP "uses every available method to move new treatments to patients as quickly as possible, while at the same time finding out whether they are helpful or harmful." In practice, that means developers of potential treatments for COVID-19 would benefit from an unusually faster track at the FDA to shorten wait times at multiple steps of the process.

Safe Harbor

This press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risk of successfully negotiating a license agreement for a potential ARDS therapy with Novellus, Inc. within the option period; the ability to access the FDA's CTAP program for our planned ARDS therapy; risks associated with developing our product candidates, including any licensed from Novellus, Inc., including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; our need for substantial additional funds; risks associated with conducting our Phase 3 trial for Mino-Lok, including completing patient enrollment, opening study sites and achieving the required number of catheter failure events; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks related to our growth strategy; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; the early stage of products under development; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to attract, integrate, and retain key personnel; 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.

Contact:

Andrew Scott Vice President, Corporate Development (O) 908-967-6677 x105[emailprotected]

SOURCE Citius Pharmaceuticals, Inc.

Home

Originally posted here:
Citius Receives FDA Response on Pre-Investigational New Drug (PIND) Application for its Induced Mesenchymal Stem Cells (iMSCs) to Treat Acute...

Skin Stem Cells Comments Off on Citius Receives FDA Response on Pre-Investigational New Drug (PIND) Application for its Induced Mesenchymal Stem Cells (iMSCs) to Treat Acute… | June 26th, 2020

Editors note: Exposure to UV-C light poses known threats to human health. While the research discussed in this story examined ways that UV-C might be broadly deployed to stem the spread of COVID-19, it is presented only as information about new research. Readers are strongly cautionednot to experiment on their own with UV-C as an antimicrobial measure.

COVID-19 has upended society and brought chaos to generous swaths of the global economy, as governments have locked down to prevent the spread of the virus. The pandemic has also boosted interest in a variety of technologies that might help in the coronavirus fightincluding the use of ultraviolet light to sterilize scarce personal protective equipment (PPE) such as face masks, for reuse.

In recent papers, two research teams have now proposed approaches to take the use of UV-C much farther. The two groups lay out different schemes for using this high-energy light to scrub the pandemic virus from the air in occupied indoor spaceswhile still keeping the occupants safe from the usual harmful long-term effects of exposure to UV radiation. The teams argue that the approaches they suggest, if widely adopted, could sharply reduce viral transmission in public indoor spaces, and thereby help the world resume something closer to normal economic activity.

The antimicrobial effects of UV-C lightwhich occupies the wavelength band from 200 to 280 nmhave long been known, and 254-nm mercury-vapor germicidal lamps are routinely used to disinfect empty hospital rooms, clean up tainted water, and otherwise cleanse surfaces and areas where bacteria and viruses may lurk. The problem is that the same light that hammers pathogenic airborne bacteria also wreaks havoc on human cells, with long-term impacts such as skin cancer and eye damage.

As a result, much of the recent interest in leveraging UV-C light against the COVID-19 virus, SARS-CoV-2, has centered around possible uses in disinfecting PPE for doctors, nurses and first responders. (One such effort, focusing on a low-cost system that can be used to disinfect scarce N95 face masks for reuse in resource-limited areas, is being pursued by an interdisciplinary team including OSA Fellow and past president Thomas Baer.)

The teams behind the two recent papers favor substantially broadening the use of UV-C against SARS-CoV-2. They suggest that, with the right precautions and setups, the light might be used as a prophylactic in occupied indoor spaces such as workplaces, schools, hospitals and other public areas, silently scrubbing the air of the coronavirus even as the occupants of the buildings go about their daily lives.

One of the studies was led by OSA Fellow Javier Garca de Abajo of the Institute of Photonic SciencesICFO, Spain, and tapped a multinational, interdisciplinary team of researchers in virology, aerosols, immunology and other areas (ACS Nano, doi: 10.1021/acsnano.0c04596). In the study, the researchers looked at how the judicious, strategic installation of UV-C sources might attack the most common routes of indoor viral transmission.

The team began by inventorying the indoor places and systems most likely to spread the virus. These, the researchers argue, include interior ventilation systems in a wide range of settings, which can spread airborne viruses; infrastructure items touched by many persons, such as elevator buttons, stair rails and public-transit handles; and common public facilities with repeated, periodic high use, such as public toilets, storerooms and other areas.

The ICFO-led team argues that antimicrobial UV-C light sources could be placed in a variety of locations associated with viral spread, such as ventilation systems and other areas. The lamps could then be operated without a direct optical pathto humans, or while the roomsare not in use, to help reduce virus propagation without endangering human health. [Image: Sketches by Nacho Gaubert] [Enlarge image]

The team then analyzed how a range of UV-C sources, including LEDs and conventional mercury-vapor lamps, could be safely deployed to attack coronavirus in these individual spread scenarios. For example, the team argues that lamps could be deployed inside ventilation systems, with no direct optical path to occupants in the building, and run continuously, to disinfect ambient air. In other settings such as public restrooms, high-intensity UV-C light could be applied during the periods in which the rooms are unoccupied. And frequently touched surfaces, such as elevator buttons, might be subjected to continuous, weak UV-C illumination for ongoing disinfectionas human interactions with these surfaces tend to be brief, and thus would involve only a very low dose of radiation.

The team acknowledges that putting such an approach into effect would require a massive new deployment of UV lighting, with a correspondingly huge required uptick in its production. Yet the cost, they argue, may not be excessive, given the scale of the problem humanity now confrontsnot just in human mortality, but in the economic devastation that antiviral lockdowns have created.

The team estimates, in fact, that disinfection with fluorescence lamps could be implemented at a cost of a few dollars per person with minimum changes in infrastructure. Thus, they argue, a global capital investment of a few billion [U.S.] dollars could protect on the order of 109 indoor workers worldwide. The authors add, however, that current manufacturers of UV-C sources may have difficulty coping with the expected rise in demand originated by the SARS-CoV-2, pandemic, as the global market for UV-C light barely reaches one billion dollars a year at present.

Another team, based at Columbia Universitys Irving Medical Center, USA, took a different view of how UV-C might be used to fight coronavirus in occupied indoor settings (Sci. Reports, doi: 10.1038/s41598-020-67211-2). The team focused in particular on the specific wavelengths of UV-C light that might be used against the virusstressing that not all UV-C light is necessarily the same in terms of its hazard to human health.

Conventional germicidal lamps operate at a wavelength of 254 nm, as that is one of the sweet spots for UV-C absorption by DNA or RNA molecules; the light thus scrambles and inactivates the pathogens genetic machinery. It can also penetrate into the skin and eyes, causing similar genetic and other damage to living human cells.

The Columbia team points out, however, that light in the far UV-C, between 207 and 222 nm, is also effective at killing microorganismsyet studies to date suggest that these wavelengths do not cause the human health issues associated with the 254-nm radiation of germicidal lamps. The reason, according to the team, is that light in the far UV-C penetrates less than a few micrometers into biological materials. Thats too short a distance to pierce the nonliving protective layers of the skin and eye into the living cells beneath. But its more than sufficient to bore into tiny bacteria and viruses.

These considerations, the Columbia team argues, suggest that far-UV-C light should have about the same anti-microbial properties as conventional germicidal UV light, but without producing the corresponding health effects. Thus, they conclude, continuous, low-dose application of light in the wavelength area of 222 nm might be used in occupied public areas to stem the spread of SARS-CoV-2, without posing other hazards to the occupants themselves.

To test the idea out, the team used a misting device to aerosolize two common coronaviruses that were structurally similar to SARS-CoV-2, and then flowed the aerosols through the air in front of a 222-nm UV-C lamp. The researchers found that, at very low doses, the far UV-C light killed more than 99.9% of the viral load. The team is now at work on tests using the actual SARS-CoV-2 pathogenand says that preliminary data suggest that the far-UV-C light effectively kills that virus, too.

Putting these results together with the safety data, the team leader, David J. Brenner, asserted in a press release accompanying the work that far-UV-C at very low doses could be used in combination with other measures, like wearing face masks and washing hands, to limit the transmission of SARS-CoV-2 and other viruses.

See the rest here:
COVID-19: Putting UV-C to Work - Optics & Photonics News

Skin Stem Cells Comments Off on COVID-19: Putting UV-C to Work – Optics & Photonics News | June 26th, 2020

Regenerative Medicine Market: Snapshot

Regenerative medicine is a part of translational research in the fields of molecular biology and tissue engineering. This type of medicine involves replacing and regenerating human cells, organs, and tissues with the help of specific processes. Doing this may involve a partial or complete reengineering of human cells so that they start to function normally.

Get Sample Copy of the Report @https://www.tmrresearch.com/sample/sample?flag=B&rep_id=1889

Regenerative medicine also involves the attempts to grow tissues and organs in a laboratory environment, wherein they can be put in a body that cannot heal a particular part. Such implants are mainly preferred to be derived from the patients own tissues and cells, particularly stem cells. Looking at the promising nature of stem cells to heal and regenerative various parts of the body, this field is certainly expected to see a bright future. Doing this can help avoid opting for organ donation, thus saving costs. Some healthcare centers might showcase a shortage of organ donations, and this is where tissues regenerated using patients own cells are highly helpful.

There are several source materials from which regeneration can be facilitated. Extracellular matrix materials are commonly used source substances all over the globe. They are mainly used for reconstructive surgery, chronic wound healing, and orthopedic surgeries. In recent times, these materials have also been used in heart surgeries, specifically aimed at repairing damaged portions.

Cells derived from the umbilical cord also have the potential to be used as source material for bringing about regeneration in a patient. A vast research has also been conducted in this context. Treatment of diabetes, organ failure, and other chronic diseases is highly possible by using cord blood cells. Apart from these cells, Whartons jelly and cord lining have also been shortlisted as possible sources for mesenchymal stem cells. Extensive research has conducted to study how these cells can be used to treat lung diseases, lung injury, leukemia, liver diseases, diabetes, and immunity-based disorders, among others.

Global Regenerative Medicine Market: Overview

The global market for regenerative medicine market is expected to grow at a significant pace throughout the forecast period. The rising preference of patients for personalized medicines and the advancements in technology are estimated to accelerate the growth of the global regenerative medicine market in the next few years. As a result, this market is likely to witness a healthy growth and attract a large number of players in the next few years. The development of novel regenerative medicine is estimated to benefit the key players and supplement the markets growth in the near future.

Global Regenerative Medicine Market: Key Trends

The rising prevalence of chronic diseases and the rising focus on cell therapy products are the key factors that are estimated to fuel the growth of the global regenerative medicine market in the next few years. In addition, the increasing funding by government bodies and development of new and innovative products are anticipated to supplement the growth of the overall market in the next few years.

On the flip side, the ethical challenges in the stem cell research are likely to restrict the growth of the global regenerative medicine market throughout the forecast period. In addition, the stringent regulatory rules and regulations are predicted to impact the approvals of new products, thus hampering the growth of the overall market in the near future.

Global Regenerative Medicine Market: Market Potential

The growing demand for organ transplantation across the globe is anticipated to boost the demand for regenerative medicines in the next few years. In addition, the rapid growth in the geriatric population and the significant rise in the global healthcare expenditure is predicted to encourage the growth of the market. The presence of a strong pipeline is likely to contribute towards the markets growth in the near future.

Global Regenerative Medicine Market: Regional Outlook

In the past few years, North America led the global regenerative medicine market and is likely to remain in the topmost position throughout the forecast period. This region is expected to account for a massive share of the global market, owing to the rising prevalence of cancer, cardiac diseases, and autoimmunity. In addition, the rising demand for regenerative medicines from the U.S. and the rising government funding are some of the other key aspects that are likely to fuel the growth of the North America market in the near future.

Furthermore, Asia Pacific is expected to register a substantial growth rate in the next few years. The high growth of this region can be attributed to the availability of funding for research and the development of research centers. In addition, the increasing contribution from India, China, and Japan is likely to supplement the growth of the market in the near future.

Request TOC of the Report @https://www.tmrresearch.com/sample/sample?flag=T&rep_id=1889

Global Regenerative Medicine Market: Competitive Analysis

The global market for regenerative medicines is extremely fragmented and competitive in nature, thanks to the presence of a large number of players operating in it. In order to gain a competitive edge in the global market, the key players in the market are focusing on technological developments and research and development activities. In addition, the rising number of mergers and acquisitions and collaborations is likely to benefit the prominent players in the market and encourage the overall growth in the next few years.

Some of the key players operating in the regenerative medicine market across the globe areVericel Corporation, Japan Tissue Engineering Co., Ltd., Stryker Corporation, Acelity L.P. Inc. (KCI Licensing), Organogenesis Inc., Medtronic PLC, Cook Biotech Incorporated, Osiris Therapeutics, Inc., Integra Lifesciences Corporation, and Nuvasive, Inc.A large number of players are anticipated to enter the global market throughout the forecast period.

See the rest here:
Regenerative Medicine Market Analysis Growth Demand, Key Players, Share Size, and Forecast To 2025 - 3rd Watch News

Cardiac Stem Cells Comments Off on Regenerative Medicine Market Analysis Growth Demand, Key Players, Share Size, and Forecast To 2025 – 3rd Watch News | June 26th, 2020

Eustasis Psychiatric & Addiction Health

417-322-6622| 3600 S. National Ave., Springfield

Eustasis Psychiatric & Addiction Health is pleased to announce construction of their brand new site in the heart of Medical Mile, opening in July! There is expanded access to medication management, psychotherapy, testing and advanced treatment options.

Dr. Alok Jain and his wife Breanna Jain started the clinic in 2018 with the mission of providing the highest quality psychiatric care to all patients. Since then they have put together an amazing team of board-certified providers and support staff who have tirelessly served the Springfield community.

We wanted to build something really vital for our patients. A place that everyone could come, regardless of age or diagnosis, says Dr. Jain.

Dr. Alok Jain has been honored as a 417 Top Doctor every year since 2007 and is 2020's top psychiatrist. He is a board-certified psychiatrist, member of the American Psychiatric Association and has an extensive background in consultation-liaison psychiatry and psychopharmacology.

Eustasiss immediate-access site has provided patients with an unprecedented way to receive psychiatric care without delay. Their walk-in and be seen model is changing the face of psychiatry. The new location has eight providers and room for growth! They are providing the most state of the art modalities, including ADHD testing and esketamine.

It is super exciting, says Breanna Jain, CEO, PMHNP-BC. We have patients who come in all hours of the day, pediatric or adult, all payor sources. They will ask, You mean I can really be seen right now? We can proudly tell them of course! This is just the way we believe mental health should be done.

Both Dr. Jain and Breanna know that people need options in this community. Patients struggles are numerous, ranging from addiction, bipolar, ADHD, trauma, anxiety and more. The Jains like to think of Eustasis as a one-stop-shop.

There shouldnt be high levels of bureaucracy. This is what overwhelms patients. Barriers have no place when it comes to mental health, the Jains explain.

Eustasis is committed to helping patients find the optimal balance of emotions during these difficult times. They have expanded their hours and have both in person and telemedicine options available. They are always accepting new patients!

See original here:
Medical Professionals in the Ozarks - 417mag

Cardiac Stem Cells Comments Off on Medical Professionals in the Ozarks – 417mag | June 26th, 2020

One day before Sila Jio Boonklomjit was set to donate desperately needed stem cells to save his sisters life, doctors made an alarming discovery: Hed contracted COVID-19.

The 5-year-old COVID-19 patient is now being credited with saving his big sisters life by going through with an experimental procedure to cure her of a genetic blood disorder without passing along the coronavirus.

Saying it was the first known case of such a procedure, Ramathibodi Hospital claimed victory yesterday after successfully transplanting bone marrow from Jio to his sister, Jintanakan Jean Boonklomjit, who was born with thalassemia and was in a severe condition.

Its as if my daughter is reborn and gets a new life, said the childrens father, Suchai Boonklomjit.

Thalassemia is a hereditary disorder that limits the bloods ability to carry oxygen and affects an estimated 1% of all Thais. Rather than being treated by ongoing blood transfusions, recent breakthroughs have shown it can be cured through gene therapy.

The procedure began in April but wasnt completed until yesterday by Suradej Hongeng of the hospitals pediatrics department.

Posted by onTuesday, June 23, 2020

According to Suradej, it was a long and uncertain road to this happy outcome. It had been difficult to find a donor compatible with Jean, leading them to settle on Jio as her best hope. After they were confirmed to be a genetic match in 2018, they prepared for the transplantation procedure.

Other difficulties followed, in part due to the young age of both patients. Moreover, Jeans immune system was compromised by chemotherapy while Jio had to be placed in quarantine on the eve of the procedure. Doctors believe he was likely infected by his mother, Sasiwimol Boonklomjit.

The case is believed to be the first successful stem cell transplant from a donor with active COVID-19. Both Jio and his mother have since recovered from the virus.

Read more of Coconuts Bangkoks content here.

This article, 5-year-old Thai boy with COVID-19 saves sisters life with his stem cells, originally appeared on Coconuts, Asia's leading alternative media company. Want more Coconuts? Sign up for our newsletters!

Read more:
5-year-old Thai boy with COVID-19 saves sisters life with his stem cells - Yahoo Singapore News

Bone Marrow Stem Cells Comments Off on 5-year-old Thai boy with COVID-19 saves sisters life with his stem cells – Yahoo Singapore News | June 25th, 2020

Hematopoietic Stem Cell Transplantation (HSCT) Market report provides (6 Year Forecast 2020-2026) including detailed Coronavirus (COVID-19) impact analysis on Market Size, Regional and Country-Level Market Size, Segmentation Market Growth, Market Share, Competitive Landscape, Sales Analysis and Value Chain Optimization. This Hematopoietic Stem Cell Transplantation (HSCT) market competitive landscape offers details by topmost key manufactures (Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc) including Company Overview, Company Total Revenue (Financials), Market Potential, Presence, Hematopoietic Stem Cell Transplantation (HSCT) industry Sales and Revenue Generated, Market Share, Price, Production Sites and Facilities, SWOT Analysis, Product Launch. For the period 2014-2020, this study provides the Hematopoietic Stem Cell Transplantation (HSCT) sales, revenue and market share for each player covered in this report.

Key Target Audience of Hematopoietic Stem Cell Transplantation (HSCT) Market: Manufacturers of Hematopoietic Stem Cell Transplantation (HSCT), Raw material suppliers, Market research and consulting firms, Government bodies such as regulating authorities and policy makers, Organizations, forums and alliances related to Hematopoietic Stem Cell Transplantation (HSCT) market.

Get Free Sample PDF (including COVID-19 Impact Analysis, full TOC, Tables and Figures)of Hematopoietic Stem Cell Transplantation (HSCT)[emailprotected]https://www.researchmoz.us/enquiry.php?type=S&repid=2276986

Synopsis of Hematopoietic Stem Cell Transplantation (HSCT) Market:In 2019, the market size of Hematopoietic Stem Cell Transplantation (HSCT) is million US$ and it will reach million US$ in 2025, growing at a CAGR of from 2019; while in China, the market size is valued at xx million US$ and will increase to xx million US$ in 2025, with a CAGR of xx% during forecast period.

In this report, 2018 has been considered as the base year and 2019 to 2025 as the forecast period to estimate the market size for Hematopoietic Stem Cell Transplantation (HSCT).

Based onProduct Type, Hematopoietic Stem Cell Transplantation (HSCT) market report displays the manufacture, profits, value, and market segment and growth rate of each type, covers:

Allogeneic Autologous

Based onend users/applications, Hematopoietic Stem Cell Transplantation (HSCT) market report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate for each application, this can be divided into:

Peripheral Blood Stem Cells Transplant (PBSCT) Bone Marrow Transplant (BMT) Cord Blood Transplant (CBT)

Hematopoietic Stem Cell Transplantation (HSCT) Market: Regional analysis includes:

Do You Have Any Query Or Specific Requirement? Ask to Our Industry[emailprotected]https://www.researchmoz.us/enquiry.php?type=E&repid=2276986

The Hematopoietic Stem Cell Transplantation (HSCT) Market Report Can Answer The Following Questions:

What are the Upstream Raw Materials And Manufacturing Equipment of Hematopoietic Stem Cell Transplantation (HSCT)? What is the manufacturing process of Hematopoietic Stem Cell Transplantation (HSCT)?

Who are the key manufacturers of Hematopoietic Stem Cell Transplantation (HSCT) market? How are their operating situation (Capacity, Production, Price, Cost, Gross and Revenue)?

Economic impact on Hematopoietic Stem Cell Transplantation (HSCT) industry and development trend of Hematopoietic Stem Cell Transplantation (HSCT) industry.

What is the (North America, South America, Europe, Africa, Middle East, Asia, China, Japan) Production, Production Value, Consumption, Consumption Value, Import And Export of Hematopoietic Stem Cell Transplantation (HSCT)?

What will the Hematopoietic Stem Cell Transplantation (HSCT) Market Size and The Growth Rate be in 2026?

What are the key market trends impacting the growth of the Hematopoietic Stem Cell Transplantation (HSCT) market?

What are the Hematopoietic Stem Cell Transplantation (HSCT) Market Challenges to market growth?

What are the types and applications of Hematopoietic Stem Cell Transplantation (HSCT)? What is the market share of each type and application?

What are the key factors driving the Hematopoietic Stem Cell Transplantation (HSCT) market?

What are the Hematopoietic Stem Cell Transplantation (HSCT) market opportunities and threats faced by the vendors in the Hematopoietic Stem Cell Transplantation (HSCT) market?

Contact:

ResearchMozMr. Rohit Bhisey,Tel: +1-518-621-2074USA-Canada Toll Free: 866-997-4948Email:[emailprotected]

Browse More Reports Visit @https://www.mytradeinsight.blogspot.com/

Read the original post:
Hematopoietic Stem Cell Transplantation (HSCT) Market Expand Their Businesses With New Investments In 2020 And Coming Future - Bulletin Line

Bone Marrow Stem Cells Comments Off on Hematopoietic Stem Cell Transplantation (HSCT) Market Expand Their Businesses With New Investments In 2020 And Coming Future – Bulletin Line | June 25th, 2020

Summary

A review of 423 patients treated at MSK finds that most people with cancer dont fare any worse if they get COVID-19 than other people who are hospitalized for that infection.

In the early days of the COVID-19 pandemic, many doctors worried that people undergoing treatment for cancer would do particularly poorly if they became infected with the virus that causes the disease. Thats because treatments for cancer, especially chemotherapy, can lower a persons immune defenses and put them at higher risk for all kinds of infections.

But according to a new study from Memorial Sloan Kettering published June 24 in Nature Medicine, most people in active cancer treatment dont fare any worse if they get COVID-19 than other people who are hospitalized with the infection. Further research is needed to look at the effects of certain drugs mainly immunotherapies called checkpoint inhibitors, which did seem to make COVID-19 worse. But the researchers say their findings suggest that no one should delay cancer treatment because of concerns about the virus.

If youre an oncologist and youre trying to figure out whether to give patients chemotherapy, or if youre a patient who needs treatment, these findings should be very reassuring, says infectious disease specialist Ying Taur, one of the studys two senior authors.

Infectious disease expert Ying Taur has cared for many MSK patients who were hospitalized with COVID-19.

The study looked at 423 MSK patients diagnosed with COVID-19 between March 10 and April 7, 2020. Overall, 40% were hospitalized for COVID-19, and 20% developed severe respiratory illness. About 9% had to be placed on a mechanical ventilator, and 12% died. The investigators found that patients taking immunotherapy drugs called immune checkpoint inhibitors were more likely to develop severe disease and require hospitalization. But other cancer treatments, including chemotherapy and surgery, did not contribute to worse outcomes.

The big message now is clear: People should stay vigilant but not stop or postpone checkpoint immunotherapy or any other cancer treatment.

Factors that did make COVID-19 worse were the same as those seen in studies of people who didnt have cancer. We found that being older, as well as preexisting conditions like heart disease and diabetes, are all drivers of severe COVID-19 illness, says MSK Chief Medical Epidemiologist Mini Kamboj, the studys other senior author. This wasnt surprisingbecause these connections are well established.

Although the study wasnt large enough to make determinations about every treatment and every cancer type, patterns did emerge. Dr. Taur says there was initially great concern about people receiving high doses of chemotherapy for leukemia, especially those who had recently undergone bone marrow or stem cell transplants. Thats because transplants require a persons entire immune system to be wiped out with chemotherapy before they receive new blood cells, leaving them susceptible to all kinds of infections.

Surprisingly, though, Dr. Taur cared for recent transplant recipients who were infected with COVID-19 but didnt have any symptoms. If you think about it more, it makes sense, he says. Most of the complications seen in people with COVID-19 seem to be caused by the bodys immune response to the virus.

On the other hand, immunotherapy drugs called checkpoint inhibitors work by freeing up the immune system to attack cancer. Patients receiving these agents may develop a more robust reaction to the virus that causes COVID-19. This may explain why this study observed higher rates of complications in people with COVID-19 infection who were treated with checkpoint inhibitors.

Even with immune checkpoint inhibitors, though, these findings should not affect whether patients get treated. Everyone who needs these drugs should still receive them, Dr. Kamboj says. Its just important for doctors to be extra vigilant about testing and monitoring for the virus and for people with cancer to take extra precautions to avoid infection.

A study published in May 2020 by MSK immunotherapy expert Matthew Hellmann focused exclusively on people with lung cancer who got COVID-19. The researchers didnt find the same risks from immune checkpoint drugs as this Nature Medicine study. But that study included data on far fewer patients treated at MSK, which could explain the difference.

Dr. Kamboj notes that one aspect of this research that sets it apart from other studies is that it included at least 30 days of follow-up after a COVID-19 diagnosis. Also, it reported severe respiratory illness as a main outcome rather than death.

Having that follow-up time is something that a lot of other studies have not included because everyone is in a rush to get their data out. In addition, reporting death rates can overestimate infection-related mortality, especially in the early phase of an epidemic, Dr. Kamboj says. Also, the clinical spectrum and course of this disease is still not fully understood, especially in people with cancer. We wanted to give patients enough time to recover and make sure they didnt need to be readmitted to the hospital.

Even with immune checkpoint inhibitors, though, these findings should not affect whether patients get treated. Everyone who needs these drugs should still receive them.

She adds that another strength of the study is that patient outcomes were not affected by constraints caused by a lack of space or supplies even though MSK is in the heart of the COVID-19 epicenter in New York City, where other hospitals faced overcrowding and other issues. This gave researchers a true picture of how cancer patients fare with COVID-19. We saw a surge during the peak of the epidemic in New York, but everyone got the care they needed, Dr. Kamboj explains. We had enough ventilators for everyone who needed them. We never had to make decisions about who to admit to intensive care because of a lack of critical equipment.

Drs. Taur and Kamboj agree that this is just one of many studies that will need to be done on the connections between cancer and COVID-19. We still need to find out more. We need to look at the connections between COVID-19 and particular types of cancer as well as outcomes related to specific chemotherapy drugs, Dr. Taur concludes. But the big message now is clear: People should stay vigilant but not stop or postpone checkpoint immunotherapy or any other cancer treatment.

Link:
Should You Delay Cancer Treatment Because of COVID-19? Study Says Most Treatments Dont Worsen Coronavirus Infection - On Cancer - Memorial Sloan...

Bone Marrow Stem Cells Comments Off on Should You Delay Cancer Treatment Because of COVID-19? Study Says Most Treatments Dont Worsen Coronavirus Infection – On Cancer – Memorial Sloan… | June 25th, 2020

Bone marrowaspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

Get Access to sample pages @ https://www.trendsmarketresearch.com/report/sample/3184

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.Europe and North America spearheaded the market as of 2016, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

In 2016, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy.

Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others

Covid 19 Impact[emailprotected] https://www.trendsmarketresearch.com/report/covid-19-analysis/3184

See the rest here:
Bone Marrow Processing Systems Market Business Analysis, New Innovation | Share, Revenue, And Sales Till 2025 - Cole of Duty

Bone Marrow Stem Cells Comments Off on Bone Marrow Processing Systems Market Business Analysis, New Innovation | Share, Revenue, And Sales Till 2025 – Cole of Duty | June 25th, 2020

Disruption of the TP53 gene and mutation of the KMT2D gene are predictive of poor outcomes in patients with mantle cell lymphoma (MCL) who are receiving high-dose therapy, according to a new study.

The findings, which build upon growing knowledge of the genetics of MCL, can be used to better classify patients into risk categories, the investigators said. The study was published in the journal Haematologica.1

Corresponding author Simone Ferrero, MD, of the University of Torino, in Italy, noted that the current treatment paradigm for patients with MCLcytarabine-based chemotherapy followed by autologous stem cell transplantation (ASCT)has led to dramatic advancements in the outcomes of patients who are young and healthy enough for the therapy. However, Ferrero said as many as one-quarter of those patients will experience early treatment failure.

So far, the best tool clinicians have had to identify patients at high risk of relapse has been the MCL international prognostic index (MIPI), and the Ki-67 proliferation index. The combination of the 2 is known as the MIPI-c score. Although the tool is of value, Ferrero and colleagues asserted that the resulting scores lack the precision necessary to develop tailored schedules specifically for high-risk patients.

In an effort to better elucidate the factors associated with a high risk of failure, the investigators used samples from the phase III FIL-MCL0208 trial, which is a prospective, open-label, multicenter study designed to evaluate lenalidomide (Revlimid) as a maintenance therapy versus observation in patients in MCL remission following high-dose chemotherapy including rituximab (Rituxan) followed by ASCT (NCT02354313).

Ferrero and colleagues performed targeted resequencing and DNA profiling on purified tumor samples of the patients in the study. Out of 300 patients enrolled in the study, samples from 186 patients were able to be evaluated for genetic mutations and abnormalities in copy numbers.

The analysis confirmed earlier reports2,3 that TP53 disruption is a significant prognostic factor. After 4 years, patients with mutations or deletions of TP53 had lower progression-free survival (PFS) and overall survival (OS) rates compared with patients without the disruptions.

However, the authors broke new ground by identifying KMT2D as another important genetic factor. In wild-type cases, those with KMT2D mutations had PFS rates of just 33.2%, versus 63.7% in those without the mutation after 4 years (P <.001). Overall survival was similarly affected; the 4-year OS rate among patients with KMT2D mutations was 62.3% versus 86.8% among those without the mutation (P = .002).

In the FIL-MCL0208 trial, KMT2D mutations emerged as a novel biomarker heralding chemo-immunotherapy failure, with a predictive value similar to that of TP53 aberrations, Ferrero and colleagues wrote.

The authors then used their findings to create a new scoring system to identify patients at the highest risk.

The independent adverse prognostic value of TP53 and KMT2D aberrations prompted us to integrate the molecular results into the MIPI-c, aiming at further improving its ability to discriminate high-risk patients, the authors said.

The model begins with MIPI-c score; those with low or intermediate risk scores under MIPI-c were given 0 points in the new model, and those placed in the high-risk category by the MIPI-c model were given one point. In addition, patients with TP53 disruptions were given 2 additional points, as were those with the KMT2D mutation. In this new scoring system, which the investigators dubbed MIPI-g, patients with a score of 0 were deemed low risk, patients with scores of 1 to 2 were deemed intermediate risk, and patients with scores of 3 or higher were categorized as high risk.

When investigators performed PFS and OS calculations based on their risk categories, they found PFS rates varied dramatically among the groups, from 72.0% in the low-risk group to 11.5% in the high-risk group after 4 years (P <.0001). Four-year OS rates similarly dropped from 94.5% in the low-risk group to 44.9% (P <.0001). Among patients in the intermediate group, the 4-year PFS rate was 42.2% and the OS rate was 65.8%.

In the Nordic validation series, patients with KMT2D mutations showed similar worse outcomes compared with wild-type patients (median OS, 8.4 vs 12.7 years). Among patients with TP53 mutations, the median OS was 2.0 years compared with 12.7 years for patients with wild-type TP53. The validation series also showed similar 4-year OS rates by risk groups: 91.3% for low-risk patients, 72.2% for intermediate risk, and 15.4% for high risk.

Among the studys limitations, the authors noted that their analysis was performed only on CD19-positive bone marrow cells. The investigators also said they do not yet have sufficient randomization data to know whether and to what extent lenalidomide maintenance affected the patients with these mutations within the broader FIL-MCL0208 trial. However, they said it is unlikely that full data will be able to offer clear takeaways, since only 27 patients with the TP53/KMT2D mutations were finally randomized in the study, due to a high rate of progressive disease among these patients. Of those 27, only 9 were started on lenalidomide maintenance.

In their conclusion, Ferrero and colleagues said that the ability to distinguish the highest-risk patients could be used by clinicians to identify high-risk patients for novel therapeutic approaches.

As in other lymphoid disorders, novel non-chemotherapeutic strategies specifically designed for [high-risk] patients need to be investigated in MCL, the authors said. Besides the approved drugs lenalidomide and ibrutinib [Imbruvica], new molecules such as the BCL-2 inhibitor venetoclax [Venclexta] might be very promising for these chemorefractory patients, especially for TP53 disrupted cases.

References:

1. Ferrero S, Rossi D, Rinaldi A, et al. KMT2D mutations and TP53 disruptions are poor prognostic biomarkers in mantle cell lymphoma receiving high-dose therapy: a FIL study. Haematologica. 2020;105(6):1604-1612. doi:10.3324/haematol.2018.214056

2. Nordstrm L, Sernbo S, Eden P, et al. SOX11 and TP53 add prognostic information to MIPI in a homogeneously treated cohort of mantle cell lymphoma--a Nordic Lymphoma Group study. Br J Haematol. 2014;166(1):98-108. doi:10.1111/bjh.12854

3. Halldrsdttir AM, Lundin A, Murray F, et al. Impact of TP53 mutation and 17p deletion in mantle cell lymphoma. Leukemia. 2011;25(12):1904-1908. doi:10.1038/leu.2011.162

See the rest here:
TP53, KMT2D Abnormalities Linked With Poor Prognosis in MCL - Targeted Oncology

Bone Marrow Stem Cells Comments Off on TP53, KMT2D Abnormalities Linked With Poor Prognosis in MCL – Targeted Oncology | June 25th, 2020

On February 67, 2020, the Simons Foundation Autism Research Initiative (SFARI) convened a two-day workshop to explore the possibility of gene therapies for autism spectrum disorder (ASD), a neurodevelopmental condition associated with changes in over 100 genes. Inspired by the recent, stunning successes of gene therapy for the fatal neuromuscular disorder spinal muscular atrophy (SMA)1, and by the accumulation of genes confidently associated with ASD2, SFARI welcomed a diverse collection of researchers to begin to think about whether a similar approach could be taken for ASD. Because gene therapy attempts to fix what is broken at the level of a causative gene, it would offer a more direct and imminent strategy than mitigation of the many and as yet mostly unclear downstream effects of a damaged gene.

The workshop was organized in 20 talks and several discussion panels, which tackled many outstanding issues, including how to choose candidate target genes and predict outcomes; how to optimize vectors for gene delivery; how to decide when to intervene; which animal models to develop; how to find appropriate endpoints for clinical trials and understand the available regulatory pathways. SFARI also raised the question of how its funding might best propel gene therapy efforts amid the emerging, complex ecosystem of academic laboratories, biotech companies, and pharmaceutical industries.

Even the opportunity to have this discussion is very rewarding, said SFARI Investigator Matthew State of the University of California, San Francisco (UCSF), one of the investigators who directed teams of geneticists to analyze the Simons Simplex Collection (SSC).

These efforts have offered up multiple potentially feasible therapeutic targets. Though rare, de novo disruptive mutations in the highest confidence ASD genes often result in severe impairment characterized not only by social difficulties, but also by intellectual disability and seizures. The combination of a single gene mutation of large effect coupled with particularly severe outcomes that include ASD are likely to offer the most immediate targets for gene therapy. For now, this leaves out a large number of individuals with autism for whom genetic causes are not yet known and are likely the result of a combination of many small effect alleles across a large number of genes.

Highlights from talks and discussion panel, chaired by Rick Lifton of Rockefeller University

In the first talk of the workshop, State brought the group up to speed on ASD genomics. The most recent tally from exome-sequencing in simplex cases of ASD highlighted 102 genes in which rare mutations confer individually large risks2. In contrast, the task of identifying common variants carrying very small risks remains quite challenging, with less than a half dozen alleles so far identified with confidence3. The rare, disruptive mutations that result in loss of function of one gene copy are an attractive focus for gene therapy because of the tractability of targeting a single spot in the genome per individual and because, in the vast majority of cases, there remains a single unchanged allele. This points to ways to boost gene and/or protein expression back toward the normal state by leveraging the unaffected copy. But both the limited number of cases known so far combined with the possibility that different mutations to the same gene may have different effects complicate thinking about how to prioritize targets for gene therapy.

State made several points that were continually touched on throughout the workshop. Many ASD genes are highly expressed during midfetal development in the cortex, and additional experiments will need to determine whether and how long a window of opportunity may be present for successful gene therapy postnatally. Given the relatively small number of people with these conditions, new clinical trial designs are needed that dont rely on comparisons between large control and intervention groups (see also Bryan Kings talk below).

Beyond the gene-crippling mutations found in the exome, disruptions to transcription may also dramatically raise risk for autism and may be corrected with a type of gene therapy using ASOs. SFARI Investigator Stephan Sanders of UCSF focused on the role of splicing, the process by which an initial transcript is turned into messenger RNA by removal of introns and joining together of exons. Splicing is disrupted in at least 1.5 percent of individuals with ASD4, and possibly many more, as suggested by transcript irregularities found in postmortem autism brain5. Sanders described Illuminas Splice AI project in which machine-learning helps predict noncoding variants that can alter splicing, including those beyond typical splice sites found near a gene6. As a result of incorporating sequence information around and between splice sites, this computational tool detected more mutations with predicted splice-altering consequences in people with ASD and intellectual disability than in those without the condition.

An ASO designed to bind specific portions of RNA could conceivably correct errors in transcription. ASOs have already been approved for use in other disorders in order to skip exons, retain exons or to degrade mRNA. Unlike other forms of gene therapy, ASOs do not permanently alter the genome, making it a kind of gene therapy lite. This reversibility has both disadvantages (having to re-infuse the ASO every few months) and advantages (multiple opportunities to optimize the dose and target; serious adverse effects are not permanent).

Jonathan Weissman of UCSF discussed the available toolbox for controlling gene expression developed by many different laboratories. To turn genes on or off, he has developed a method to combine CRISPR with an enzymatically inactive (dead) Cas9, which can then be coupled with a transcriptional activator (CRISPRa) or repressor (CRISPRi)7 (Figure 2). In the case of loss-of-function mutations, Weissman outlined strategies to make the remaining good allele work harder: increase transcription via CRISPRa, decrease mRNA turnover, increase translation of a good transcript via modification of upstream open reading frames (uORFs) or increase a proteins stability, possibly through small molecules acting on the ubiquitin system8. That said, the effects on a cell may be complicated. Using Perturb-Seq screens, Weissman described genetic interaction manifolds that show nonlinear mapping between genotype and single cell transcriptional phenotypes9. Additionally, Weissman summarized recent work from his laboratory that has identified large numbers of uORFs that result in polypeptides, some of which affect cellular function.

SFARI Investigator Michael Wigler of Cold Spring Harbor Laboratories echoed the idea of a gene-therapy strategy that increases expression of the remaining good copy of a gene, especially given that in his estimate, 45 percent of simplex cases of autism carried a de novo, likely disrupting variant. He also called attention to the uterine environment, especially the challenge posed by expression of paternally derived antigens in the fetus and the impact of a potential maternal immune response, and the need to understand how it interacts with de novo genetic events.

Highlights from talks and discussion panel, chaired by Arnon Rosenthal of Alector

The discussion turned to finding ways of getting genes into the central nervous system. The AAV is the darling of gene therapy, given that it does not replicate and is not known to cause disease in humans. A version that can cross the blood-brain barrier (AAV9) was used to deliver a gene replacement to children with SMA intravenously; though this effectively delivered the genetic cargo to ailing motor neurons in the spinal cord, it does not work that well at delivering genes throughout the brain.

Ben Deverman of the Stanley Center at the Broad Institute of MIT and Harvard detailed his efforts to optimize AAV for efficient transduction of brain cells through a targeted evolution process: his team engineers millions of variants in the capsid of the virus, then screens them for entry into the nervous system and transduction of neurons and glia. This has yielded versions (called AAV-PHP.B and AAV-PHP.eB) that more efficiently enter the brain10,11. One successfully delivered the MECP2 gene to the brain of a Rett syndrome mouse model, resulting in ameliorated symptoms and an extended lifespan12. Unfortunately, these viruses dont work in human cells or in all mouse strains. A quick mouse genome-wide association study (GWAS) revealed that the Ly6a gene mediates efficient blood-brain barrier crossing of AAV-PHP.B and AAV-PHP.eB13. Now his group has identified Ly6a-independent capsids that may translate better to humans. He also noted that the PHP.B vectors have tissue specificity for brain and liver.

With an estimated 87 percent of autism-associated genes raising risk through haploinsufficiency (having only one functional gene copy out of the two), SFARI Investigator Nadav Ahituv of UCSF made the case for approaches that boost expression of the remaining good copy of a gene through endogenous mechanisms a strategy he called cis-regulation therapy. This method also provides a way to work around the small four kb payload of AAV, which strains to contain cDNA of many autism genes. A recent study by his group used CRISPRa targeted at an enhancer or promoter of SIM1 and promoter of MC4R, both obesity genes, in mice. Using one AAV vector for a dCas9 joined to a transcription activator, and another AAV vector having a guide RNA targeting either a promoter or an enhancer, and a guide RNA targeting a promoter, the researchers injected the vectors together into the hypothalamus, which resulted in increased SIM1 or MC4R transcription and reversed the obesity phenotype brought on by loss of these genes14. Targeting regulatory elements had the added benefit of tissue specificity, and there seemed to be a ceiling effect for SIM1 expression, which suggested an endogenous safeguard against overexpression at work. He is now collaborating with SFARI Investigator Kevin Bender, also at UCSF, to apply this approach to the autism gene SCN2A.

Botond Roska of the Institute of Molecular and Clinical Ophthalmology in Basel, Switzerland pointed out that getting genes to the cells where they are needed is crucial when treating eye diseases. Off-target effects there can induce degeneration of healthy cells. For this reason, Roska and his group have created AAVs that target specific cell types in the retina by developing synthetic promoters that efficiently promote expression of the viruss cargo15. The promoters they designed were educated guesses based on four approaches: likely regulatory elements close to genes expressed with cell-type specificity in the retina, conserved elements close to cell typespecific genes, binding sites for cell typespecific transcription factors and open chromatin close to cell typespecific genes. Screening a library of these in mouse, macaque and human retina revealed some with high cell-type specificity (Figure 3). Importantly, macaque data predicted success in human retina much better than did mouse data. In preliminary experiments, and more relevant to gene therapy for ASD, these cell-specific vectors also had some success in mouse cortex, for example lighting up parvalbumin neurons or an apparently new type of astrocyte.

Roska also described new methods for delivery, in which nanoparticles are coated with AAV, then drawn into the brain using magnets16. This magnetophoresis technique allows a library of experimental AAVs to be tested at the same time in one monkey. Steering nanoparticles with magnets gives more control of vector placement and gene delivery. He argued that these in the future could access even deep structures of the brain.

Highlights from talks and discussion panel, chaired by Steven Hyman of the Broad Institute at MIT and Harvard

Kathy High of Spark Therapeutics reviewed the story of gene therapy for spinal muscular atrophy (SMA) type 1. Though she was not directly involved in that research, she is well aware of the regulatory atmosphere surrounding gene therapy, given that Spark Therapeutics developed the first approved AAV-delivered gene for a form of retinal dystrophy. The SMA story is a useful case study in that an ASO-based therapy (nusinersen, marketed as Spinraza), approved in 2016, set the stage for a gene-replacement therapy, marketed as Zolgensma (onasemnogene abeparvovec). Ultimately, the amount of data supporting Zolgensmas approval was modest: a Phase one dose study of 15 infants1, and an ongoing Phase three trial of 21 infants and safety data from 44 individuals. Yet the approval was helped by the dramatic results and clear endpoints: those receiving a single intravenous infusion of an AAV9 vector containing a replacement gene all remained alive at 20 months of age, whereas only 8 percent survived to that age in the natural history data, which compiles the diseases untreated course. High mentioned that maintaining product quality for gene therapeutics may prove trickier than for typical medications.

The attractive, highly customizable nature of gene therapy might have a regulatory downside in that different vector payloads, even when designed to do the same thing, could invite separate approval processes. Though not knowing how regulatory agencies would view this, High said that their perspectives are bound to evolve as more gene therapy trials are completed.

Getting to ASD-related syndromes, Bender talked about SCN2A, which encodes the sodium channel Nav1.2. SCN2A mutations in humans can be gain of function or loss of function; gain-of-function mutations are associated with early onset epilepsy, and loss-of-function mutations with intellectual disability and ASD. In a mouse model missing one copy of SCN2A, Bender and his group have discovered a role for SCN2A in action potential generation in the first week after birth, and in synaptic function and maturation afterward through regulation of dendritic excitability18 (Figure 4). Using AAV containing CRISPRa constructs developed with the Ahituv lab, the researchers successfully increased SCN2A expression, and recovered synapse function and maturity, even when done several weeks postnatally. Getting the appropriate dosage is critical since gain-of-function mutations are linked to epilepsy. However, Bender reported even when SCN2A expression increased to double normal levels, no hints of hyperexcitability appeared. We might be able to overdrive this channel as much as we want and actually may not have risk of producing an epileptic insult, he said. Next steps are to figure out the developmental windows for intervention, evaluate changes in seizure sensitivity and extend this kind of cis-regulatory approach to other ASD genes.

Angelman syndrome is another condition that attracts interest for gene therapy, in part because neurons already harbor an appropriate replacement gene. Angelman syndrome stems from mutations to the maternally inherited UBE3A gene, which is particularly damaging to neurons because they only express the maternal allele, while the paternal allele is silenced by an antisense transcript. SFARI Investigator Mark Zylka of the University of North Carolina and colleagues showed in 2011 that this paternal allele could be unsilenced with a cancer drug in a mouse model of Angelman syndrome19. Since then, three companies have built ASOs to do the same thing, and these are going into clinical trials. To get a more permanent therapeutic, Zylka has been developing CRISPR/Cas9 systems to reactivate paternal UBE3A, and preliminary experiments show that injecting this construct into the brains of embryonic mice, and then again at birth, results in brain-wide expression of paternal UBE3A and is long-lasting (at least 17 months). Zylka is now making human versions of these constructs. He later noted rare cases of mosaicism for the Angelman syndrome mutation people with 10 percent normal cells in blood have a milder phenotype20, which suggests that even inefficient transduction of a gene vector could help.

Zylka also made a case for prenatal interventions in Angelman syndrome: studies of mouse models indicate that early reinstatement of UBE3A expression in mouse embryos rescues multiple Angelman syndrome-related phenotypes, whereas later postnatal interventions rescue fewer of these21; for humans, a diagnostic, cell-based, noninvasive prenatal test will be available soon22; ultrasound-guided injections into fetal brain of nonhuman primates have been developed23; prenatal surgeries are now standard of care for spinal bifida; and intervening prenatally decreases the risk of an immunogenic response to an AAV vector or its cargo. During the discussion, it was noted that another benefit of acting early was that less AAV would be needed to transduce a much smaller brain; however, a drawback is the lack of data on Angelman syndrome development from birth to one year of age. This natural history would be necessary for understanding whether a prenatal therapy is more effective than treatment of neonates.

SFARI Investigator Guoping Feng of the Massachusetts Institute of Technology has been investigating SHANK3, a high-confidence autism risk gene linked to a severe neurodevelopmental condition called Phelan-McDermid syndrome, which is marked by intellectual disability, speech impairments, as well as ASD. SHANK3 is a scaffold protein important for organizing post-synaptic machinery in neurons. Mouse studies by Feng have shown that SHANK3 re-expression in adult mice that have developed without it can remedy some, but not all, of their phenotypes, including dendritic spine densities, neural function in the striatum and social interaction24. Furthermore, early postnatal re-expression rescued most phenotypes. This makes SHANK3 a potential candidate for gene therapy; however, it is a very large gene 5.2kb as a cDNA that is difficult to fit into a viral vector. To get around this, Fengs group has designed a smaller SHANK3 mini-gene as a substitute for the full-sized version. Preliminary experiments show that AAV delivery of the mini-gene can rescue phenotypes like anxiety, social behavior and corticostriatal synapse function in SHANK3 knockout mice. Feng also discussed his success in editing the genome in marmosets and macaques using CRISPR/Cas9 technology and showed data from a macaque model of SHANK3 dysfunction25. These models may help test gene therapy approaches and identify biomarkers of brain development closely related to the human disorder.

For people with rare conditions brought on by even rarer mutations, individualized gene therapies can provide a pathway for treatment. SFARI Investigator Timothy Yu of Boston Childrens Hospital/Harvard described his N-of-1 study in treating a girl with Batten disease, a recessive disorder in which a child progressively loses vision, speech and motor control while developing seizures. In a little over a year, an ASO that targeted her unusual splice-site mutation in the CLN7 gene was designed, developed and given intrathecally to the girl26. The lift was in negotiating with the FDA and working with private organizations, not just in the science, Yu said. After a year of treatment with the ASO (dubbed milasen after the girl, Mila), there were no serious adverse events; seizure frequency and duration had decreased (Figure 5); and possibly her decline had slowed. Though she remains blind, without intelligible speech and unable to walk on her own, she was still attentive and could respond happily to her familys voices. The highly personalized framework for this drugs approval is completely different from how medications meant for populations are approved, and it opens a regulatory can of worms, Yu said, though he added that the regulators were willing to countenance drug approval for an individuals clinical benefit.

Rett syndrome is a neurodevelopmental condition caused by mutations to the MECP2 gene that has a substantial research base in mouse models. Over 10 years ago, mouse models highlighted the possibility for therapeutics in this condition when Rett-associated phenotypes were rescued by adding back MECP2, even in adulthood27. This reversibility has spurred interest in gene therapy for Rett syndrome, but getting the MECP2 dose right is critical, said Stuart Cobb of the University of Edinburgh and Neurogene: just as too little MECP2 leads to Rett syndrome, too much also results in severe phenotypes. For this reason, it would be nice to package a replacement MECP2 gene with other regulatory elements to control its expression, but this results in constructs that do not fit into viral vectors. To make more room, Cobb and his colleagues have been able to chop away two-thirds of the MECP2, reserving two domains that interact to make a complex on DNA (Figure 6). Mice with this mini-gene are viable and have near normal phenotypes; likewise, injecting this mini-gene into MECP2-deficient mice extended their survival28. Doubling the dose, however, substantially lowered survival. Putting in safety valves to prevent overexpression is going to be quite important, he said. One idea is to add back a construct containing only the last two exons of MECP2, which is where most Rett mutations land. These would then be spliced into native transcripts (called trans-splicing), and thus their expression controlled by endogenous regulatory elements.

Underscoring the double-edged sword of MECP2 dosage, Yingyao Shao from Huda Zoghbis lab at Baylor described an MECP2 duplication syndrome (MDS) in humans, which features hypotonia, intellectual disability, epilepsy and autism. Experiments in an MDS mouse model, which carries one mouse version and one human version of MECP2, recapitulates some of the phenotypes of the human condition and can be rescued by an ASO targeting the human allele29. Shao described work to optimize the ASO for translation into humans, which involved developing a more humanized MDS model that carries two human MECP2 alleles. An acute injection of the ASO was able to knock down MECP2 expression in a dose-dependent manner in these mice, and RNA levels dropped a week after injection, with protein levels falling a week later. MECP2 target genes also normalized their expression level, and one maintained this for at least 16 weeks post-injection. The ASO also rescued behavioral phenotypes of motor coordination and fear conditioning, but not of anxiety; these corrections followed the molecular effects, and these timelines would be important to keep in mind while designing clinical trials. Shao also noted that overtreatment with the ASO resulted in Rett-associated phenotypes, but that this was reversible, which suggests that some fine-tuning of dosing in humans might be possible.

To avoid overtreatment and toxicity of any MDS-directed therapy, Mirjana Maletic-Savatic, also at Baylor, is leaving no stone unturned in a hunt for MDS biomarkers that can predict, in each individual, the safety of a particular dose and regimen. Such biomarkers would also help monitor individuals during treatment, give information about target engagement and identify candidates for a particular treatment. Anything found to be sensitive to expression levels of MECP2 could also be useful for Rett, though she noted that MECP2 levels measured in blood do not track linearly with gene copy number. Thus, because of interindividual variability, her approach is to collect a kitchen sink of data deriving composite biomarkers that accurately reflect the stage and severity of disease in a given case. She and her colleagues are collecting clinical, genetic, neurocircuitry (such as EEG and sleep waves), immunology and molecular data detected in blood, urine and CSF. These measures are also being explored in induced neurons derived from skin samples of people with MDS. She highlighted two interrelated potential biomarkers in the blood of those with this condition; both measures are downstream targets of MECP2 and are responsive to ASO treatment.

Highlights from Early detection and clinical trial issues talks and panel discussion, chaired by Paul Wang of SFARI

Coming up with objective measures of a persons status either their eligibility for a treatment, or whether the treatment has engaged with its target or even whether the treatment is effective is a real necessity in autism-related conditions, which comprise multiple interrelated behaviors. Eye-tracking methodology may provide such a marker, argued SFARI Investigator Ami Klin of Emory University. Focusing on the core social challenges of autism, Klin, Warren Jones and colleagues have been studying children as they view naturalistic social scenes to quantify their social attention patterns. This has revealed how remarkably early in development social visual learning begins and that this process is disrupted in infants later diagnosed with ASD prior to features associated with the condition appearing. By missing social cues, autism in many ways creates itself, moment by moment, Klin said. In considering gene therapy, it may be useful to know that eye looking (how much a subject looks at a persons eyes, an index of social visual engagement) in particular and social visual engagement in general are under genetic control30; that eye-tracking differences emerge as early as 26 months of age; and that homologies in social visual engagement exist between human babies and nonhuman infant primates.

In getting to a point to test gene therapies, identifying those who need them is essential. Wendy Chung of Columbia University and the Simons Foundation illustrated how diagnosis is yoked closely to therapy. To illustrate this, she described her pilot study of newborn blood spots to screen for SMA; at the start, no treatment was available, but the screen identified newborns for a clinical trial of nusinersin. Notably, the screen only cost an additional 11 cents per baby. In the three years since her pilot screen began, the FDA approved two gene therapies for SMA and the SMA screen was adopted for nationwide newborn screening. Currently she is piloting a screen for Duchenne muscular dystrophy and plans to develop a platform that will allow researchers to add other conditions. In prioritizing genetic conditions for gene therapy, she outlined some ideas for focus, such as genes resulting in phenotypes that would not be identified early without screening, those that are relatively frequent, those that are lethal or neurodegenerative, those with a treatment in clinical trials or with FDA-approved medications, and those conditions that are reversible.

In the meantime, Chung also outlined SFARIs involvement in establishing well-characterized cohorts of individuals with autism, which can help lay a groundwork for gene therapy. People with an ASD diagnosis can join SPARK (Simons Foundation Powering Autism Research for Knowledge), which collects medical, behavioral and genetic information (through analysis of DNA from saliva, at no cost to the participant). If a de novo genetic variant is found in one of ~150 genes, that person is referred to Simons Searchlight, which fosters rare conditions communities and which is also compiling natural history data on people with these mutations.

Bryan King of UCSF discussed how current trial designs for ASD were inadequate for gene therapy trials. As ASD prevalence has grown, parallel design trials with one group receiving an experimental medicine and the other a placebo are the standard, but these wont be possible for the rare conditions that are candidates for gene therapy. Also, change is hard to capture, given the malleable nature of ASD: with no intervention, diagnosis can shift between ASD and pervasive developmental disorder-not otherwise specified (PDD-NOS) in 1284 months (as defined by the DSM-IV). Current scales are subjective and may miss specific items of clinical significance. (Last year, SFARI funded four efforts to develop more sensitive outcome measures.) King outlined other pitfalls in ASD clinical trials, including significant placebo responses, inadequate sample sizes and not being specific enough when asking about adverse effects. King also mentioned improvements that may arise from just enrolling in a study, which could prompt previously housebound families to venture out with their child, which could kick off a cascade of positive effects. He reiterated how, for gene therapy, a natural history comparison group may be more appropriate, combined with solid outcome measures.

SFARI Investigator James McPartland of Yale University then underlined the need for objective biomarkers for clinical trials, for which there are currently none that are FDA qualified for ASD. As the director of the Autism Biomarkers Consortium for Clinical Trials (ABC-CT), he works with other scientists to develop reliable biomarkers that can be scaled for use in large samples across different sites. McPartland noted a biomarker studied in the ABC-CT: an event-related potential (N170) to human faces, which is on average slower in ASD than in typically developing children. He is working on ways to make it easier for people with ASD and intellectual disabilities to participate in biomarker studies and to make them more socially naturalistic. In discussion, he mentioned he thought it would be possible to look for these kinds of biomarkers in younger children.

SFARI Investigator Shafali Jeste of the University of California, Los Angeles recounted her experience in working with children with genetic syndromes associated with neurodevelopmental conditions. Though she is asked to participate in clinical trials for these conditions, she senses the field has some work to do to be ready for these trials, particularly in those with additional challenges such as epilepsy and intellectual disability. Meaningful and measurable clinical endpoints are still insufficient, and there needs to be more ways to improve accessibility of these trials for these rare conditions. This means developing new measures, such as gait-mat technology that senses walking coordination, or EEG measures in waking and sleep, which have been applied to people with chromosome 15q11.2-13.1 duplication (dup15q) syndrome, who have severe intellectual disability and motor impairments. Jeste also emphasized that increasing remote access to some measures can make a big difference for a trial; for example, a trial of a behavioral intervention for tuberous sclerosis complex that required weekly lab visits was disappointingly under-enrolled until researchers revamped it so most of the intervention could be done remotely31.

By grappling with the challenges to gene therapy for ASD, the workshop marked out a faint road map of a way forward. As the scientific questions are answered, the regulatory and clinical trial infrastructure will need to develop apace, and coordination between private, academic and advocacy sectors will be essential. But as gene therapy for diverse human conditions continues to be explored and gene discovery in ASD continues, there is reason to believe that some forms of ASD can eventually benefit from this strategy.This workshop provided a terrific discussion about the challenges in developing targeted gene interventions and their potentially transformative effects as therapies, said John Spiro, Deputy Scientific Director of SFARI. We are grateful to all theparticipants, and SFARI looks forward to translating these discussions into focused funding decisions in the near future.

Back to Top

Continued here:
SFARI | SFARI workshop explores challenges and opportunities of gene therapies for autism spectrum disorder - SFARI News

Spinal Cord Stem Cells Comments Off on SFARI | SFARI workshop explores challenges and opportunities of gene therapies for autism spectrum disorder – SFARI News | June 25th, 2020