Archive for the ‘Bone Marrow Stem Cells’ Category

MAGICAL Nature is Rebecca Bennett's first exhibition in four years following her stem cell transplant.

Running throughout December at Rydal Hall Old School Room Teashop at Ambleside, the exhibition features various media from acrylics and watercolours to photography.

Rebecca loves capturing the magic of colour and pattern within nature in her artwork. She grew up surrounded by the beautiful fells and lakes of Cumbria. Coming from an artistic family, painting and drawing from an early age was natural.

From photographs of lakes seen through the prism of a crystal ball to an enigmatic acrylic painting of a grey heron, Magical Nature includes striking images of the wildlife and countryside of Rebeccas native Lake District.

With a BA(hons) degree in Contemporary Applied Arts from Cumbria Institute of the Arts, Rebecca furthered her art practice and skills to create porcelain ceramic pieces and textile mixed media artworks.

Following university Rebecca completed a variety of successful art projects and workshops alongside her exhibitions. These included art workshops with young people at the Coniston Water Festival and Blencathra Field Studies Centre.

In 2015 Rebecca had a stem cell transplant at London's Kings College Hospital. An unrelated donor provided the stem cells to treat failing bone marrow caused by rare Gata2 deficiency and Myelodysplastic Syndrome. Painting and photography have, she says, been a great therapy throughout her transplant journey.

"When you feel up to it having something to immerse yourself in such as art and photography can be a fantastic way to help you forget about your pain and problems. For those moments, you are focused on what you are creating and can escape for a little while."

Alongside exhibiting her work for the first time since being diagnosed with her illnesses, Rebecca hopes to raise awareness of MDS and blood disorders. Donations to the charity MDS UK patient support will be made from sales of Rebeccas greeting cards and prints during the exhibition.

Originally posted here:
Artist's first exhibition following stem cell transplant - The Westmorland Gazette

Recently, a new study that appears in the journal Nature, focuses on stem cell therapy and shows unexpected ways in which it may be helpful in recovering the health of the heart. Stem cell therapy has become popular in the past few years due to its benefits for a big number of health conditions.

Currently, there is major ongoing research on stem cells since they are responsible for the regeneration of new cells and may play a fundamental role in understanding the development of a variety of different diseases as well as their potential treatments.

Some of the recent discoveries of medical science include using stem cells as regenerative medicine as they can be turned into particular types of cells that may be able to replace tissues damaged as a result of health issues and thereby control the disease.

Read also:New Study Reveals Hydromethylthionine Slows Cognitive Decline and Brain Atrophy

The therapy can be specifically useful for people with conditions such as type 1 diabetes, spinal cord injuries, Alzheimers disease, Parkinsons disease, stroke, cancer, burns, amyotrophic lateral sclerosis, heart disease, and osteoarthritis.

At the moment, the most successful procedure that involves stem cell therapy is performing a bone marrow transplant. This surgical operation replaces the cells which have been damaged during chemotherapy by programmed stem cells. People are usually able to maintain and live a normal life after recovery from the surgery.

Furthermore, stem cell usage in clinical trials designed for testing the effectiveness, safety, and potential negative impact of new drugs. To do so, the stem cells can be programmed into becoming the type of cells that the drug aims to target.

The new study, which was led by Jeffery Molkentin who is a professor of the Howard Hughes Medical Institute (HHMI) and the director of Molecular Cardiovascular Microbiology a Cincinnati Childrens Hospital Medical Center, takes data from a study from the same journal, Nature, from the years 2014 which was conducted by the same medical team.

In the new paper, the team with Molkentin as the principal investigator found some unexpected results. There were two types of stem cells in the clinical trial cardiac progenitor cells and bone marrow mononuclear cells.

The main objective of the new trial was to re-evaluate the results of the 2014 study, which showed that injecting c-kit positive heart stem in the heart does not help in the regeneration of cardiomyocytes, to see how the cell therapy can be made to be effective.

It was instead discovered that injecting an inert chemical called zymosan, which is designed particularly for inducing an innate immune response, or dead stem cells can also be beneficial for the recovery of heart as they may speed up the healing procedure.

Injecting either dead stem cells or zymosan led to a reduction in the development of cellular matrix connective tissue in the areas which had been damaged in the heart. In addition, the mechanical properties of the targeted scar also improved.

Another important finding was that chemical substances such as zymosan are required to be injected directly into the heart for optimum results. In previous clinical trials, direct injections were avoided for safety reasons.

Molkentin and the team state that follow-up studies and trials on this new discovery are imminent as they may be important for developing therapies in the future.

Read more here:
Stem Cell Therapy May Improve Heart Health In New Ways - TheHealthMania

This Mesenchymal Stem Cells research study consists of the historical data from and forecasts till 2026. The report is created keeping in mind to make it a valuable source of information for market specialists in readily accessible documents. The documents are curated with clearly presented graphs and figures.

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The global mesenchymal stem cells market size to reach USD 2,518.5 Million by 2026, growing at a CAGR of 7.0% during forecast period, according to a new research report published by Alexa Reports Research. The report Mesenchymal Stem Cells Market, [By Source (Bone Marrow, Umbilical Cord Blood, Peripheral Blood, Lung Tissue, Synovial Tissues, Amniotic Fluids, Adipose Tissues); By Application (Injuries, Drug Discovery, Cardiovascular Infraction, Others); By Region]: Market Size & Forecast, 2018 2026 provides an extensive analysis of present market dynamics and predicted future trends. The market was valued at USD 1,335.1 million in 2017. In 2017, the drug discovery application dominated the market, in terms of revenue. North America region is observed to be the leading contributor in the global market revenue in 2017.

are adult stem cells, which are traditionally found in the bone marrow. However, they can also be parted from other available tissues including peripheral blood, cord blood, fallopian tube. These stem cells mainly function for the replacement of damaged cell and tissues. The potential of these cell is to heal the damaged tissue with no pain to the individual. Scientists are majorly focusing on developing new and innovative treatment options for the various chronic diseases like cancer. Additionally, the local governments have also taken various steps for promoting the use of these stem cells.

The significant aspects that are increasing the development in market for mesenchymal stem cells consist of enhancing need for these stem cells as an efficient therapy option for knee replacement. Raising senior populace throughout the world, as well as increasing frequency of numerous persistent conditions consisting of cancer cells, autoimmune illness, bone and cartilage diseases are elements anticipated to enhance the market development throughout the forecast period. The mesenchymal stem cells market is obtaining favorable assistance by the reliable federal government policies, as well as funding for R&D activities which is anticipated to influence the market growth over coming years. According to the reports released by world health organization (WHO), by 2050 individuals aged over 60 will certainly make up greater than 20% of the globes population. Of that 20%, a traditional quote of 15% is estimated to have symptomatic OA, as well as one-third of these individuals are expected to be influenced by extreme specials needs. Taking into consideration all these aspects, the market for mesenchymal stem cells will certainly witness a substantial development in the future.

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Increasing demand for better healthcare facilities, rising geriatric population across the globe, and continuous research and development activities in this area by the key players is expected to have a positive impact on the growth of Mesenchymal Stem Cells market. North America generated the highest revenue in 2017, and is expected to be the leading region globally during the forecast period. The Asia Pacific market is also expected to witness significant market growth in coming years. Developing healthcare infrastructure among countries such as China, India in this region is observed to be the major factor promoting the growth of this market during the forecast period.

The major key players operating in the industry are Cell Applications, Inc., Cyagen Biosciences Inc. Axol Bioscience Ltd., Cytori Therapeutics Inc., Stem cell technologies Inc., Celprogen, Inc. BrainStorm Cell Therapeutics, Stemedica Cell Technologies, Inc. These companies launch new products and undertake strategic collaboration and partnerships with other companies in this market to expand presence and to meet the increasing needs and requirements of consumers.

Alexa Reports Research has segmented the global mesenchymal stem cells market on the basis of source type, application and region:

Mesenchymal Stem Cells Source Type Outlook (Revenue, USD Million, 2015 2026)

Bone MarrowUmbilical Cord BloodPeripheral BloodLung TissueSynovial TissuesAmniotic FluidsAdipose Tissues

Mesenchymal Stem Cells Application Outlook (Revenue, USD Million, 2015 2026)

InjuriesDrug DiscoveryCardiovascular InfractionOthers

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Systematic analysis and evaluation of Mesenchymal Stem Cells Market with share, growth rate, Forecasts to 2026 - WindStreetz

Emi receives kisses from her adoptive parents, Jason and Katie Ballard, at the NIH Clinical Center as she gets ready to receive a lifesaving hematopoietic stem cell transplant, the only way to cure her fatal immune deficiency, using cells donated by her birth mom.

Emi smiles at The Children's Inn at NIH prior to undergoing a hematopoietic stem cell transplant, the only potential cure for her rare and deadly immune deficiency.

Bethesda, Maryland, Nov. 26, 2019 (GLOBE NEWSWIRE) -- One Texas family has lots to be thankful for this Thanksgiving. Their daughter, now 13, is doing well after undergoing a bone marrow transplantthe only chance for a cure for her rare and deadly disease. But Emis story is not only a story about the triumph of medical research that is making her cure possibleits also a story about extraordinary parental love and sacrifices by her birth mom and her adoptive family that are giving this very ill girl the best chance at life. Emi's birth mom donated her stem cells to make the lifesaving transplant possible.

We are most thankful for an answer to years of prayers, Emis adoptive mom says. Emi got a new start at life, a rebirth day. Every holiday this year will be like the first. Were so grateful to the doctors, nurses and The Childrens Inn.

Emi and her family will be celebrating Thanksgiving at The Childrens Inn at NIH, a nonprofit hospitality house that provides free lodging and a wide variety of support services to families of children with rare and serious diseases whose best chance for a treatment is a clinical research study at the National Institutes of Health. Emi and her mom have spent several months at The Childrens Inn so far and bonded with other families. On Thanksgiving Day, families staying at The Childrens Inn who cannot go home for the holiday will be served a traditional Thanksgiving meal prepared by a group of dedicated volunteers.

It took two moms who love this little nugget to fight for her right to life, Emis adoptive mom says. We finally are getting to see that beautiful part of the story that we always knew was there.

Read Emis full story.

See photos of Emi and her family.

About The Childrens Inn at NIH:

The Childrens Inn at NIH provides free lodging and a wide range of supportive services to more than 1,500 children and their families every year whose best chance for a treatment is a clinical trial at the National Institutes of Health. Opened in 1990 and located across from the NIH Clinical Center, the worlds largest hospital dedicated entirely to medical research, The Childrens Inn has welcomed children from all 50 states and 94 countries. Children staying at The Childrens Inn are making important contributions to rare disease and cancer research, including the successful treatment of childhood leukemia, as well as treatments for HIV/AIDS, childhood asthma, bone and growth diseases, childhood onset schizophrenia and other mental health issues, neurofibromatosis type 1 and a wide variety of genetic and rare diseases. For more information, visit http://www.childrensinn.org. To support The Childrens Inn, make a donation at http://www.childrensinn.org/donate.

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The Best Thanksgiving - GlobeNewswire

It may finally be a happy Christmas for a brave toddler recovering from a rare combination of cancers after pioneering stem cell treatment.

Amelia Topa, who celebrated her second birthday yesterday, is looking forward to spending the festive period with her family at home in Turriff.

Her parents Kerri Paton, 23, and Igor Topa, 24, were told that raised purple spots across Amelias body could be a sign of something seriously wrong when she was born at Dr Grays Hospital in Elgin.

Amelia was soon after diagnosed with a mix of two types of leukemia acute lymphoblastic leukaemia and acute myeloid leukaemia and spent Christmas in hospital.

Miss Paton said: Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of.

Doctors gave Amelia a bone marrow transplant using stem cells donated by a managed between 16 and 30.

The treatment worked and, by the following autumn, she was home and awaiting the arrival of her baby brother.

But tragedy struck when Amelias grandmother, Angela McNabb, died suddenly from heart failure aged 48 the day before Amelias birthday.

Miss Paton said: My mum was my best friend, she was everything to me.

Mum absolutely loved Amelia and was so close to her.

My major source of support was gone and I hadnt even had the chance to say goodbye. I couldnt believe it. It was so unfair. Last Christmas was heartbreaking.

Things went from bad to worse for the family in February, when tests showed that Amelias cancer had returned.

After intense chemotherapy she was given a second transplant using stem cells from umbilical cord blood flown specially from America at the end of June.

That procedure was a success and the toddler has entered remission.

Having spent Christmas in 2017 in hospital, and in mourning last year, Amelias parents are now looking forward to a happy festive season.

Miss Paton said: Amelia soared through the transplant and shes doing really well now,I couldnt be prouder.

I hope Amelias story will help other families going through cancer there is a light at the end of the tunnel.

Amelia has now been selected to receive the first Cancer Research UK children and young people star award in recognition of the courage she showed since being diagnosed.

The award, supported by TK Maxx, is open to all people under 18s who currently have cancer or who have been treated in the last five years with every child being awarded a trophy, TK Maxx gift card, t-shirt and certificate signed by the likes of Nanny McPhee star Dame Emma Thompson.

Spokeswoman for the charity, Lisa Adams, said: We know that a cancer diagnosis is devastating at any age, but that it can be particularly difficult for a child or young person and their families.

Thats why were calling on families across Scotland to nominate inspirational youngsters for an award so that we can recognise their incredible courage.

Nominations can be made online at cruk.org/childrenandyoungpeople

Visit link:
North-east toddler overcomes cancer after pioneering stem cell treatment - Press and Journal

A TODDLER born with two types of leukaemia will spend Christmas at home after recovering from a second stem cell transplant.

Amelia Topa, who turned two on Wednesday, was donated cells harvested from a newborn babys umbilical cord blood flown in from the US.

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The brave youngster is now in remission and is preparing to enjoy the festive season with relieved parents Kerri Paton, 23, and Igor Topa, 24, in Turriff, Aberdeenshire.

Recalling Junes life-saving op, mum Kerri said: Amelia soared through the transplant and shes doing really well. I couldnt be prouder.

Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of.

Worried medics alerted Amelias parents to raised purple spots on her body shortly after she was born at Dr Grays Hospital, Elgin.

She was diagnosed with acute lymphoblastic leukaemia and acute myeloid leukaemia and spent Christmas 2017 in hospital.

The tot was given a bone marrow transplant six months later using stem cells donated by a man aged between 16 and 30.

The treatment worked but tests showed Amelias cancer had returned in February.

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She endured gruelling chemo before her second transplant in the summer.

Kerri added: I hope Amelias story will help other families going through cancer. There is a light at the end of the tunnel.

Amelia has now been chosen to receive Cancer Research UKs first Children & Young People Star Award.

The prize, backed by TK Maxx and stars including Dame Emma Thompson, is open to under-18s battling cancer or who have been treated in the last five years.

The charitys Lisa Adams said: Were calling on families to nominate inspirational youngsters so we can recognise their courage.

VISIT cruk.org/childrenand youngpeople to nominate.

scottish-sun@the-sun.co.uk

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Originally posted here:
Brave Scots tot born with two types of leukaemia to spend Christmas at home after second stem cell transplant - The Scottish Sun

Regenerative Medicine and Stem Cell technologies... All Hype? Or The Future of Anti-Aging?

We have entered into the rapidly evolving age of Regenerative Medicine, using breakthroughs in cell to cell communication to reset your body's natural ability to heal and repair itself. Advances in Stem Cell technology race forward at an ever increasing rate as people just like you are demanding non-surgical alternatives to the multiple degenerative conditions of aging and inflammation.

If you are asking What is a Stem Cell?

Think of them as simply the master cells of rebuilding the body. They are the building blocks of our genetic code and cellular programming that coordinate healing through bio-signaling to restore our innate capacity of repair & regeneration.

Regenerative Medicine is the vanguard of 21st century health According to Journal: American College of Cardiology

Regenerative Aesthetics is a new field of regenerative medicine that aims to restore and renew the body at the cellular level, dramatically reversing the sands of time and maintaining an aesthetically desirable youthful appearance.

What we really now know is that men and women all over the world want their hair back, they want their sexual organs to work and they want to look and feel their best into their later years.

This brings us to the 3 Regenerative Therapies you should know about.

The first and most widely known of these regenerative therapies is Platelet Rich Plasma or PRP which utilizes your own Blood Plasma and Platelets in a concentrated form to activate the healing cascade. Recruiting your own innate Stem Cells for accelerated wound healing and tissue regeneration.

Known as Liquid Gold, your platelets and plasma have been shown to rejuvenate the

Unfortunately, we are finding clinically that not all PRP is created equally. In fact, some people have a very low concentration of these regenerative growth factors or a high amount of inflammatory cytokines resulting in inconsistencies from person to person, session to session. As PRP continues to get more popular in the mainstream, we find it important to share some of the newer, more optimal Regenerative Technologies that have been emerging.

Which leads us to... The second regenerative therapy you should know about.

Stem Cell Growth factors & Cytokines. Sourced from Bone Marrow Mesenchymal Stem Cells (MSC's) however, they do not contain any actual stem cells or DNA. Growth factors are naturally occurring proteins in your body that regulate cellular growth, healing, proliferation and differentiation under controlled conditions and play a role in cellular communication. They are master bio-signals acting as command and control over your body's natural healing processes and modulation of inflammation.

It has been shown that cells in aging skin generate less growth factors than cells in youthful skin. For example, by the time you are 50, on average 4% of these regenerative bone marrow MSCs are in circulation compared to what you had when you were in your teens. Hence, we age because we damage faster than we repair in our later years.

By simply adding concentrated MSC Growth factors & Cytokines to our Regenerative Therapies we can consistently improve hair loss, skin rejuvenation, collagen growth, sexual organ function, and more. We know for a fact that daily use of skin care products containing stable growth factors and cytokines help reduce the appearance of fine lines & wrinkles and improve skin tone & texture.

Lastly, and most importantly is the latest frontier and the 3rd regenerative therapy you should know about.

The Future of Regenerative Medicine... Known as Stem Cell Exosomes. Science is showing us that the optimal way to provide true stem cell therapy is to directly provide the cell bio-signals in high concentrations. After all, the signaling is what we really require to regenerate a normal healthy physiology.

Exosomes are regarded as the purest form of cellular therapy available today, providing a safe and anti-inflammatory environment for healing and repairing.

The AABB recently reported that up to one in three people in the U.S. could benefit from regenerative medicine.

At Rejuvenate 528 Regenerative Aesthetics Medical Spa, we can include exosomes for enhanced wellness to the majority of our Regenerative Aesthetic Services. This can benefit your overall health and vitality as this is reversing challenges of Inflammation!

Beauty radiates and vibrates at different frequencies in everyone I see! We love to uplift and Rejuvenate both the inner vitality and the outer Radiance of all of our patients and clients. They come for the personal attention and integrative approach using regenerative medicine modalities with ancient technologies. PA Sheri Suiter

Tap into your own healing potential with these types of bio-hacking technologies to enhance your regenerative potential and get the results you truly desire. Live longer, stronger and younger.

Book a consultation for the following Regenerative procedures:

*Medical Microneedling*Vampire Facial*Liquid Facelift*Breastlift*O-Shot*P-Shot*Penile Enhancement*Hair Restoration*Stretch mark & scar repair*Hand Rejuvenation*Joint Inflammation*Overall Vitality & Wellness

Sheri Suiter CLT, MS, PA-C, Founder of Rejuvenate 528 Regenerative Aesthetics Medical Spa in Sarasota, FLRejuvenate528.com

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Discover The Latest Frontier in Anti-aging Medicine and 3 Regenerative Therapies You Should Know... - YourObserver.com

Hematopoietic stem cells (HSCs) can be used to better understand and treat blood-based diseases. Stem cell research in the model organism zebrafish is well-studied in the developmental stage, but is limited in the adult stage because HSCs are difficult to purify in this species. Researchers at Kanazawa University and their collaborators have developed a new purification scheme that allows HSCs to be purified from adult zebrafish kidneys, potentially opening new possibilities for stem cell research.

Kanazawa, Japan Hematopoietic stem cells (HSCs) are multipotent cells that can develop into every type of blood cell in the body. They can also be used in medical research to understand and treat blood-based diseases. Zebrafish (Danio rerio) are used to study HSCs, particularly in the field of developmental biology, but the research in the adult animal is often limited because stem cells are difficult to purify in this species. Researchers at Kanazawa University and their collaborators now describe a purification scheme that allows these elusive zebrafish HSCs to be collected.

Zebrafish are a great system to study how hematopoietic cells function in normal development and their role in disease, says lead researcher Isao Kobayashi. Much of their biology mirrors what we see in humans, and with zebrafish theres the added benefit of having quite a few experimental tools at our fingertips, including live cell imaging and comparative analysis among vertebrates. Unfortunately, its proven challenging to effectively isolate HSCs from this species, and this has been a major impediment to the field.

HSCs are highly abundant in the kidneys of adult zebrafish (unlike in humans, where HSCs are found in bone marrow). The challenge is separating them from other cells found in kidneys. Cell separation usually involves a purification technique called flow cytometry, where cells are sent in single file through a tube and hit with a laser beam. The machine (a flow cytometer) then sorts the cells based on how they reflect or scatter light.

In the study, published in Scientific Reports, the researchers created a strain of zebrafish that makes two light-emitting proteins, one green (Green Fluorescent Protein, GFP) and one red (mCherry), that can be sensed and sorted by a flow cytometer. Each fluorescent protein in this zebrafish strain was regulated by the genes related with blood cells, but the cells having both fluorescent proteins were limited in HSCs. By color coding the cells with two distinct blood cell markers, the team was able to purify cells that show hallmark signs stemness like the ability to self-propagate and differentiate into other types of blood cells.

So, what might the successful isolation of HSCs in zebrafish mean for the field of stem cell research?

When HSCs were finally purified in mice, the research community learned an enormous amount about how and where stem cells self-renew and differentiate to form blood cells, says co-author Mao Kondo. Were very hopeful that this might spur a similar proliferation of research in zebrafish. In addition to some experimental advantages in zebrafish, we found that zebrafish HSCs share many key genes in common with HSCs in mammals. This suggests that mechanistic discoveries in zebrafish could have direct implications for understanding blood diseases in humans and for developing new medical treatments.

Figure.

Hematopoietic stem cells can be isolated as gata2a:GFP+ runx1:mCherry+ (gata2a+ runx1+) cells in the zebrafish kidney by flow cytometry (left panels). Transplantation assays confirmed the hematopoietic potential of gata2a+ runx1+cells (right panels).

Article

Enrichment of hematopoietic stem/progenitor cells in the zebrafish kidney

Journal: Scientific Reports

Authors: Isao Kobayashi, Mao Kondo, Shiori Yamamori, Jingjing Kobayashi-Sun, Makoto Taniguchi, Kaori Kanemaru, Fumihiko Katakura & David Traver

DOI: 10.1038/s41598-019-50672-5

Funder

This work was supported in part by Grant-in-Aid for Young Scientists (B) from the Japan Society for the Promotion of Science (17K15393).

Link:
Researchers Find a Way to Collect Elusive Blood Stem Cells from Zebrafish - Mirage News

The parents of a baby girl were left heartbroken after she was diagnosed with two types of blood cancer.

Amelia Topa, who turns two today, is in remission from leukaemia after having a stem cell transplant using a newborn babys umbilical cord blood which was specially flown in from America.

Now doing well, Amelia has received a Cancer Research UK for Children & Young People Star Award, supported by TK Maxx, in recognition of the remarkable courage she demonstrated since being diagnosed with cancer.

Amelias parents, Kerri Paton, 23, and Igor Topa, 24, of Turriff, Aberdeenshire are hugely proud of their little girl.

Kerri said: Anyone who meets Amelia would agree that shes a star.

READ MORE - Scottish actor Gray O'Brien reveals he has been treated for stage four cancer

Being told your child has cancer is the worst sentence any parent could ever hear. I felt mad at first that someone so tiny should have to go through this horrible disease. But Amelia has been a little fighter from the day she was born. I have felt amazed by her strength and lucky to have good support from friends, family and hospital staff.

We will forever be grateful to a family in America well probably never get a chance to meet. The stem cells from America looked just like a small bag of blood but they had the power to make Amelia well again.

Around 140 children are diagnosed with cancer in Scotland every year.

Mum Kerri recalls vividly the moment their lives were turned upside down when only hours after Amelia was born on November 27 2017 medics at Dr Grays Hospital in Elgin explained that raised purple spots across Amelias body could be a sign of something seriously wrong.

Following tests, on December 14 2017 Amelia was diagnosed with leukaemia. Unusually, doctors diagnosed Amelia a mix of two types, acute lymphoblastic leukaemia and acute myeloid leukaemia. The family were transferred to the Queen Elizabeth University Hospital in Glasgow the following day.

READ MORE - Gary Rhodes: Celebrity chef dies suddenly aged 59

Kerri said: It didnt hit me properly until I walked out of the room and then I started crying, a lot.

Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of. We were looked after by the hugely experienced Professor Brenda Gibson. It helped to know we had the best oncology doctor on our side.

Amelia spent her first Christmas in hospital as the first of four rounds of chemotherapy treatment started. By spring, the family were advised that Amelias best chance of survival was a bone marrow transplant using stem cells. A match was found and the transplant went ahead on June 28 2018. The family were told was that the stem cells had been donated from a man aged between 16 and 30.

Amelia recovered well and tests showed that the transplant had worked. By autumn last year Amelia was well enough to go home and the family slowly settled back in to life in Aberdeenshire. And after a difficult year, it was a boost when Kerri discovered she was pregnant again.

Oscar was the first baby to be born in the new maternity unit in Aberdeen when he arrived on October 30 2018. Now Amelia was big sister to Oscar, Kerri dared to hope they could settle in to an ordinary family life.

But tragedy struck again. Kerris mum, Angela McNabb who had stood by the family every step of the way suddenly died from heart failure aged 48- just the day before Amelias birthday.

Kerri said: My mum was my best friend, she was everything to me.

Mum absolutely loved Amelia and was so close to her. My major source of support was gone and I hadnt even had the chance to say goodbye. I couldnt believe it. It was so unfair. Last Christmas was heartbreaking.

And it was a hammerblow on February 11 this year when tests showed that Amelias cancer had come back. Doctors were uncertain at first whether anything else could be done but they suggested a second stem cell transplant, this time using stem cells from umbilical cord blood.

Amelia had intense chemotherapy in an isolation room before she was ready for the transplant at the Queen Elizabeth University Hospital in Glasgow. The transplant went ahead on June 28 this year- exactly a year after the first transplant.

Kerri said: They had to fly the umbilical cord blood over from America.

Doctors explained to us that this was the best option to keep the leukaemia away. Amelia soared through the transplant and shes doing really well now. Were finally looking forward to a happy Christmas as a family and I couldnt be prouder. I hope Amelias story will help other families going through cancer. There is a light at the end of the tunnel.

The Cancer Research UK for Children & Young People Star Awards, supported by TK Maxx, are open to all under-18s who currently have cancer or have been treated for the disease in the last five years. There is no judging panel because every child diagnosed with cancer deserves special recognition. Everyone nominated receives a trophy, 50 TK Maxx gift card, t-shirt and a certificate signed by a host of famous faces, including Nanny McPhee and Last Christmas star Dame Emma Thompson, This Mornings Dr Ranj and childrens favourite entertainer Mister Maker. Their siblings also receive a certificate.

Now they are encouraging families across Scotland to nominate their stars for the honour in the run up to Christmas.

Lisa Adams, spokeswoman for Cancer Research UK for Children & Young People in Scotland, said: Our Star Awards, supported by TK Maxx, shine an important light on children and young people with cancer.

We know that a cancer diagnosis is devastating at any age, but that it can be particularly difficult for a child or young person and their families. Thats why were calling on families across Scotland to nominate inspirational youngsters for an award so that we can recognise their incredible courage.

The Cancer Research UK for Children & Young People Star Awards are supported by TK Maxx, the biggest corporate supporter of the charitys research in to childrens and young peoples cancers. Since the partnership began, the retailer has raised over 34 million for research in to these cancers to help more children and young people survive cancer.

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Scottish baby spent first Christmas in hospital after she was diagnosed with two types of cancer - Scotland on Sunday

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

GREENVILLE, S.C.Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.

But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.

In the beginning, it was very scary, Hailie Hyman told The Greenville News.

I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.

Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.

Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.

Alyssa Carson is 18 and has a pilot's license: She wants to be in the crew that colonizes Mars

The Hymans journey began last February atMacis 1-year-old well-child checkup.

We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.

The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.

Later, in the pediatric intensive care unit, she suffered internal bleeding, too.

It was really hard, she said. There were many nights that I would just pray and pray and pray.

Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.

The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.

Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.

Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

In children, its rarer still. Most people arediagnosed in their 70s.

We were told that just four out of 1 million children get it every year, Hailie Hyman said.

That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.

Shes the only one Ive seen in my career, she said.

Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.

When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.

The transplantis extremely risky.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.

Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.

They have to go to hell and back, she said. But its the only option for long-term survival.

Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.

Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.

Maci, who was sedated through much of it, was put on full life support, she said.

That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.

Somehow, Maci made it.

There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.

Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.

Nichole Bryant, M.D.(Photo: Provided)

It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.

Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.

She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.

A dying man wanted one last beer with his sons: The moment resonated with thousands

Maci was admitted to MUSC on June 2 and released on Oct. 14.

The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.

Treylins employer held his welding job open for him. And other friends and family members did what they could to help.

We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.

We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.

Maci's doing well, but recovery from a transplant can take months to years, Bryant said.

Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.

She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.

Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.

Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.

She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.

Want to know more about becoming a marrow donor? Go to bethematch.org.

Follow Liv Osby on Twitter:@livgnews

104-year-old woman bags first buck: 'Never underestimate the power of our senior citizens'

Indiana hospital sued: More than 1,000 patients potentially exposed to HIV or hepatitis

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South Carolina toddler survives rare cancer and the risky procedure used to treat it - USA TODAY

Appiah rang Leukaemia Cares helpline from the point of diagnosis until well after the end of her daughters life. Sometimes Id call them as a means of support, she says, when things got really rough, when her medications were really powerful, and the chemo made her so unwell. She rang when she had panic attacks; an NHS psychologist had told her that these were likely, and that she should breathe into a brown paper bag, but Appiah found speaking to a person more soothing.

With a laugh, Appiah notes that shed ring the helpline at other times, too: Sometimes Id be out with Imogin, and shed be in the pram, being naughty, and all of my patience was going down the drain, and Id phone Leukaemia Cares nurses, and say: Look, Im feeling so depressed, my daughters shouting, I dont know what to do!

But I might also say: Nurse, Im actually feeling good today.

Appiah says the support of an independent person was invaluable: When your child is so ill, you need to speak to someone who doesnt know your name you need an outsider you can unload to. I didnt want anyone thinking: Here Sheila comes again!

You become self-conscious about your situation and dont want to be a burden on your friends and family. With the helpline, you wont be judged: they just listen. You get it out of your system and then go do the shopping at Sainsburys.

When Imogin was well, shed go to school. But she also spent weeks at a time in isolation in St Georges Hospital, with her mother by her side. Once, she had a bad reaction to a medication and went into cardiac arrest. She was crying and saying, Please, please! and they were giving her all sorts of medicine. The doctors were battling to keep her stable and I dived into the bed with her and told her: Youre going to be OK. I lay down with her and I started singing with her. And then, once she stabilised, she said: Now can I watch High School Musical?"

Appiah shakes her head, laughing: Thats what she was like: I was on thedoor of death, but I have something else planned. I want to watch my video and none of you are going to stop me!

Charities sent the pair to Disneyland Paris twice. The first time was fantastic, says Appiah, the second time Imogin was in and out of consciousness. But they said we should go, to make memories, Appiah explains. Imogin got to be a celebrity for a day and went to Hamleys in a limousine to get anything she wanted.

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'My daughter's death took me to the darkest place, but I've learned it's possible to come back' - Telegraph.co.uk

Angie Grice is a such huge Clemson football fan that in 20 years shes rarely missed a home game or any of the Tigers-Gamecocks match-ups.

At her tailgate parties, the plates, the tablecloth and even a rug are orange.

Angie Grice gets a visit from the Clemson Tigers mascot during her three months in the hospital.(Photo: Bon Secours St. Francis Health System)

But for this years annual Thanksgiving weekend face-off between Clemson and USC, shell be watching from home.

Diagnosed with aplastic anemia in May, the Simpsonville woman spent three months in the hospital and is still too sick to cheer her beloved Tigersfromthe stadium. Instead, she hopes to have a few friends over to catch the gameon TV.

Ive liked Clemson forever," she told The Greenville News.

"Im missing the game this yearand Im sad about that, she said.But its OK. At least Im able to watch it.

Grice, 52, first realized something was wrong in August 2018 when she suddenly had trouble crossing the parking lot from her car to her job as a physical therapy assistant.

I was very short of breath, she recalls. It would take me a long time to do anything. I just couldnt breathe.

She saw her family doctor, who sent her to Bon Secours St. Francis Health System when her blood work wassuspicious.

Angie Grice at Clemson University(Photo: Angie Grice)

An initial bone marrow biopsy was negative.But a second revealedaplastic anemia,which prevents the bone marrow frommakingenough new blood cells for the body to function normally, according to the National Institutes of Health.

The condition is so rare it strikes only 600 to 900 Americansa year, according to the The Aplastic Anemia and MDS International Foundation.

Symptoms include fatigue, weakness, dizziness, shortness of breath, infections, and easy bruising or bleeding,the NIHreports.The cause can bethe bodys own immune system attackingthe bone marrow, heredity, some drugs, and certain toxins likepesticides and benzene.

When St. Francis hematologist Dr. Fahd Quddus first saw her, Grices platelet level was 8,000 compared to a normal of 150,000.

Whenever you drop below 20,000, youre at risk of significant, life-threatening bleeding, he said. She also had significant anemia. And her white cells were also very low.

She was started on immunosuppressive medication and other drugs in combination with blood transfusions. But sadly, he said, she suffered multiple infections, fevers and a mild stroke, requiring her to stay in the hospital.

Dr. Fahd Quddus(Photo: Bon Secours St. Francis Health System)

For a few weeks, it was touch and go, Quddus said. She was very sick.

Grice'sblood counts eventually rebounded and though shes now out of the hospital, shestill needsregulartransfusions.

She's wellenough to begin a new treatment, he said, butnot yet strong enoughfor a stem celltransplant.

Theresstill a long road to recovery, Quddussaid. But she always looks at it half full. And thats a good thing because people who stay positive can do better.

No longer able to work because of the weakness and danger of infection, Gricesays shes doing OK thanks tofamily and friends.

Angie Grice at a Clemson game(Photo: Angie Grice)

My mom and dad and sister help, she says. And I am truly blessed with a lot of friends who help.

In years past, Grice and her friends arrived at the stadiumseveral hours before kick-off, spending 10 to 12 hours thereon game days.

Inside their orange tent, they set up a coupleTVs to watch other games before and after the Clemson game. There was always plenty ofgood food,smack talk and Tigersmerchandise.

Were a little over the top, she says. But its fun.

During her grueling three-monthhospital stay, it was a visit from the Clemson Tigers mascot that lifted her spirits.

One of Angie Grice's many Clemson decorations(Photo: Angie Grice)

While watching from home wont be as exciting, Grice says shes going to make the best of it. And when asked whos going to win this years game, sheexclaims, Clemson, of course!

If you ask Carolina, they will say they are, she adds with a chuckle. But theyre delusional. Were going to win this year.

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Despite illness, this Clemson fan will be tuned in for the Tigers-Gamecocks game - Greenville News

Gavin McNaney, who passed away two years ago.

Translink are hosting a blood stem cell registration event and coffee morning this Saturday as part of the Somebodys Stranger campaign, in memory of Armagh man Gavin McNaney, who passed away two years ago.

It will take place from 10am until 2pm and it is a painless process which could potentially save a life.

Former St Catherines College teacher Gavin was just 37 years of age when he passed on November 18, 2017.

He had been diagnosed with Acute Lymphoblastic Leukaemia whilst teaching in Dubai.

Gavin spent months in hospital undergoing treatment and had a bone marrow transplant in London.

But after contracting a common cold and an infection to his lungs, his life was sadly cut short and he passed away peacefully with mum and dad, Nuala and Pat, by his side.

Friend Karl McQuaid has been raising funds and awareness after the passing of his life-long friend, whom he had first met when they both attended St Patricks Grammar School in Armagh.

He has been running registration events as part of his Somebodys Stranger campaign for nine months in Gavins memory and is keen to advise people just how easy it is to register .

He who would like to thank Leanne Armstrong and her colleagues at Translink for inviting them to come along told Armagh I : Joining the stem cell register is quick, easy and pain-free.

Potential donors have a swab taken of the inside of their cheeks with the whole process taking just a few minutes. They will then be added to DKMSs worldwide database and could be contacted at any time should they be a genetic match for a blood cancer sufferer anywhere in the world.

Those lucky enough to be a match would then be asked to donate their stem cells in a pain-free procedure similar to giving blood. This could save the life of the cancer sufferer.

Donations are at your own discretion at the event with all proceeds going to Leukaemia & Lymphoma NI Northern Irelands only charity dedicated to fighting blood cancers.

Those willing to join the register should be in general good health and aged between 18 and 55.

All are urged to come along on Saturday morning, when the city will be full of revellers for the annual Georgian Day event. Please take time to come along to the bus station and help make a huge difference.

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Translink in Armagh holding stem cell registration event in memory of Gavin McNaney - Armagh i

(MENAFN - The Conversation) The global AIDS response has made significant progress in reducing HIV infections and AIDS-related deaths. New HIV infections dropped by16%from 1.9 million 2010 to 1.6 million in 2017. And the number of AIDS-related deaths decreased from 1.4 million to 940 000 in the same period.

But HIV/AIDS has not been brought under control and new infections continue to drive the epidemic. AIDS remains a leadingcause of deathin Africa.

Even if new infections are prevented,36.9 millionpeople with HIV around the world must take antiretroviral treatment to live a healthy life. While treatment is now as simple as taking a single pill a day, there are still many challenges to daily adherence, including ongoing stigma.

An ultimate solution would be a workable cure. At the recent Conference on Retroviruses and Opportunistic Infections researchersconfirmedthe second ever case of HIV remission or 'cure'. Known as the 'London patient', the person went into remission after a stem cell transplant as part of his treatment for cancer. He emerged from the procedure free of both his life-threatening Lymphoma and need for anti-HIV therapy.

The'Berlin patient' , Timothy Brown, made global headlines in 2008 when scientists announced that he had been cured of HIV. It's been 12 years since Brown was cured, after undergoing chemotherapy, total body irradiation and two stem cell transplants. Brown has been off treatment since the transplant and, after multiple tissue sampling procedures, has no remaining evidence of HIV reservoirs. The London patient is now the longest adult HIV remission after stem cell transplantation since the 'Berlin patient'.

This development is a triumph for medical science as well as for the London patient. But, as exciting as it is, stem cell transplant is a gruelling and dangerous procedure and isn't the magic bullet that will end HIV/AIDS. This is because it's unfortunately not a scalable, feasible cure for the 39 million people currently living with HIV.

The 'London patient' was HIV positive, but it was his Hodgkin's lymphoma that led to the need for a stem cell transplant.

The HI virus must link to a human host T cell in the blood or lymph nodes to replicate and infect the body. The virus attaches itself to a set of special links on the human T cell. If one of those links isn't available due to genetic mutations, the virus may find it harder to get an infection foothold.

One such genetic mutation occurs in a link called the 'CCR5 receptor'. Some people have this mutation naturally. The 'London patient', while on antiretroviral therapy and virally suppressed, had a bone marrow transplant as part of his lymphoma treatment. The bone marrow donor had the genetic mutation and passed it on to the 'London patient' through the procedure, making it more difficult for HIV to replicate.

The 'London patient' stopped taking antiretroviral therapy 16 months after the transplant. And 18 months later the virus remains undetectable. Usually, when a person with HIV stops treatment, the virus rebounds within the first month.

The achievement of remission in a second patient has provided further critical information to inform our understanding of how HIV infection occurs and the interaction between human cells and the virus.

As important as this work is, there's no scalable cure yet and it's also vital that researchers and countries keep putting effort into prevention. Important work continues to be done in this area.

As HIV cure research goes on, so does research into HIV prevention tools, such asPre-exposure prophylaxis(a daily pill that protects you from HIV infection) and the development of apreventative vaccine .

Two late stage vaccinetrialsare underway in sub-Saharan Africa. Results will be available in 2022. A preventative vaccine would also greatly enhance efforts to being the HIV epidemic under control.

A working cure, together with a preventative vaccine would be the ingredients for HIV eradication. Until then we need to get effective, accessible treatment for all who need it, while deploying the many prevention tools at our disposal.

MENAFN2811201901990000ID1099339725

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Why ending HIV still rests on a working cure -- as well as prevention - MENAFN.COM

No longer just for professional athletes, these are the stem cell and regenerative medicine options DCs need to know about

The health care landscape continues to evolve at a dizzying pace. Insurance deductibles are increasing, and this has placed a financial burden on patients who are required to self-pay for necessary and yet uncovered services.

The opioid crisis has left physicians with limited clinical options to treat chronic pain and dysfunction. At the same time, pressure has been placed on health care providers to provide affordable alternatives to invasive procedures that provide limited clinical options with high failure rates. This confluence of supply and demand has resulted in the growth of emerging therapies in the field of stem cell and regenerative medicine. These therapies are bringing hope to patients and new opportunities to health care providers who deliver them.

Regenerative medicine is the process of replacing or regenerating cells and tissues to restore normal function. Initially popularized by professional athletes, these therapies have become mainstream. More than 27 million Americans suffer from osteoarthritis today, and in 2030 25% of U.S. adults will be diagnosed with osteoarthritis. The global regenerative medicine market is predicted to reach more than $100 billion by 2022.

These moderately-invasive regenerative procedures are eclipsing traditional highly-invasive procedures, such as hip and knee implantation, which will have a global market of $35 million over the same period.

There are four primary regenerative medicine options:

Irritant therapies include prolotherapy, ozone and prolozone. Theyincludeadding multipleirritatingsubstances along with numbing agents into degenerated or injured joints, and areas of pain.

These therapies cause inflammation to kick-start regeneration by stimulating the body to send in macrophages, which are cells that ingest and destroy theirritantsolution and trigger the healing response. Irritant therapies are an excellenttreatmentfor all forms of musculoskeletal and joint pain includingchronic neck and back pain, and rotator cuff injuries.

The effect of irritant therapies is analogous to jump-starting the battery in a tractor to get the engine to turn over.

Protease inhibition therapy eliminates the factors causing cartilage degradation, tissue breakdown, inflammation and pain. It cleans and protects joints. It is most commonly used for patients with osteoarthritis (OA) and degenerative disc disease (DDD).

It includes therapies such as alpha-2-macroglobulin (A2M) and interleukin-1 receptor antagonist protein (IRAP). A2M and IRAP are proteins found naturally in our blood. They act as protease inhibitors by binding to and inactivating damaging proteases in the body. Proteases are catabolic enzymes that break larger molecules into smaller units. Proteases trapped in the joints catabolize cartilage and break it down, causing arthritis. A2M is a large protein made in the liver. It blocks activity for all known molecules that cause cartilage breakdown. It works like a Venus flytrap by having a bait-and-trap mechanism on two sides.

Once the proteases are bound on both sides, the molecule initiates a suicide cascade and dies, allowing it to be flushed out of the area by the body.

The binding effect of protease inhibition therapy is analogous to de-weeding a garden and tilling the soil before planting.

A fibronectin-aggrecan complex test (FACT) may be used to determine the presence of FAC, which is a biomarker or indicator of cartilage breakdown caused by proteases. FAC is a unique molecular complex that is specific for painful inflammation of the spine and cartilage.

A small sample of fluid is taken from the joint or disc and sent to a lab for testing. The test looks for the presence of FAC in the fluid sample and determines where you are: FAC+ or FAC-. FAC+ patients are identified as ideal candidates for A2M injections and have a 90% rate of responding to the A2M therapy.

Stem cell therapy is focused on concentrating the workhorses of regeneration and restoration of tissues: stem cells. This results in greater cell signaling and cell recruitment than other regenerative therapies. Stem cells are known as mesenchymal signaling cells. They are considered pluripotent, which means they are undifferentiated and can replicate into various cell and tissue types.

Stem cells are found in bone marrow, the soft spongy tissue found at the center of large bones. Introducing stem cells into an injured area initiates the healing response, repairing damaged tissue by growing new, healthy tissue. The most common stem cell therapies include bone marrow aspirate concentrate (BMA), nanofat and stromal vascular fraction.

Injecting stem cells into an injured area is analogous to planting seeds in a garden.

Growth factor therapies are focused on cell signaling and cell recruitment. Blood is made up of white blood cells, red blood cells, and platelets that are suspended in plasma. Platelets are most widely known for their ability to clot blood. Platelets are also highly rich in growth factors that are proteins that stimulate healing. When an injury occurs, platelets become activated, migrate to the site of injury and release growth factors.

Growth factor therapies are the most popular provider choice for the low-cost regeneration of tissues and include platelet-rich plasma (PRP) and platelet-rich fibrin matrix (PRFM). The therapy includes drawing the patients blood followed by centrifugation to concentrate the platelets and exclude other unwanted blood products.

Another type of growth factor therapy is amniotic fluid growth factor (GF) injection therapy. Amniotic fluid surrounds the fetus during pregnancy and provides protection and nourishment. Human amniotic fluid is sourced from consenting mothers during full-term C-sections. It contains over 200 growth factors, cytokines and proteins. The therapeutic use of amniotic fluid is regulated by the FDA. It must be tested for disease and may not include any viable cells. Amniotic fluid GF therapy has both anti-inflammatory and anti-microbial properties and includes naturally-occurring hyaluronic acid for lubrication. It is most commonly used to promote the repair and reconstruction of soft tissues including cartilage and tendons.

Exosomes are being heralded as the next frontier of growth factor therapies. While they are not cells, exosomes play a vital role in the communication and rejuvenation of all the cells in the body. Exosomes are extracellular vesicles, or small bubbles, released from cells, especially from stem cells. These culture-expanded cell secretions are derived from human placental tissue. They allow for cell-to-cell communication, transporting molecules that are important regulators of intracellular information. Exosomes act as a food source for stem cells and prolong their activity. Exosomes are anti-inflammatory and include more than 300 growth factors, cytokines and proteins.

Patients with Lyme disease, burns, chronic inflammation, autoimmune disease and other chronic degenerative diseases may benefit from including exosomes in their treatment regimen. The application of growth factor therapies is analogous to applying fertilizer to a garden to help the crop grow and flourish.

Moving stem cell and regenerative medicine forward in the treatment algorithm may eliminate the need for other ineffective or potentially harmful therapies. These therapies provide new hope for patients whose only alternatives have been long-term medication, steroid injections, and costly and time-consuming surgery and rehab.

Stem cell and regenerative medicine therapies may only be provided by licensed medical professionals following all appropriate rules and regulations. An understanding of these emerging therapies and the benefits they may provide is essential as the collaboration between doctors of medicine and chiropractic increases and we join forces to combat chronic pain, dysfunction and disease.

MARK SANNA, DC, ACRB LEVEL II, FICC, is a member of the Chiropractic Summit and a board member of the Foundation for Chiropractic Progress. He is the president and CEO of BreakthroughCoaching, and can be reached at mybreakthrough.com or800-723-8423.

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A primer: stem cell and regenerative medicine as 'the' emerging therapy - Chiropractic Economics

OBJECTIVES:

Stem cell therapy is a promising approach in the treatment of acutemyocardial infarction(AMI). Mesenchymal stem cells (MSC) from bone marrow (BM-MSC) and adipose tissue (AT-MSC) are attractive and feasible for preclinical and clinical trials. In this study, we compared the therapeutic potential of BM-MSC and AT-MSC in repairing the hearts of rats with isoproterenol (ISO)-induced AMI.

Forty-two female rats were assigned into two groups; the optimization and the experimental group. The optimization groups were further subdivided into control group and the AMI induced group (using ISO). The experimental group was subdivided into AMI+cell-free media injected in the tail vein, AMI+BM-MSC, and AMI+AT-MSC groups treated with the intravenous injection of their respective cell types. Twenty-eight days after induction, electrocardiogram (ECG) was performed, and heart tissue samples were collected for histological assessment and cells tracing.

MSC therapy repaired cardiac functions shown by the restoration of ST segment, QT and QRS intervals in the ECG when compared to the AMI group. Infarct area was significantly decreased, and cardiac tissue regeneration signs were shown on histopathological examination.

Both MSC sources proved to be equally efficient in the assessed parameters.

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Comparative Study of the Therapeutic Potential of Mesenchymal Stem Cells Derived from Adipose Tissue and Bone Marrow on Acute Myocardial Infarction...

Medically reviewed by Richard M. Stone, MD

More than 60,000 new cases ofadult leukemiaare diagnosed in the U.S. each year. Although it is one of the more common childhood cancers,leukemia occurs more often in older adults.

Leukemia is a cancer of the bodys blood-forming tissues that results in large numbers of abnormal or immature white blood cells. The main types of leukemia are:

AML causes the bone marrow to produce immature white blood cells (called myeloblasts). As a result, patients may have a very high or lowwhite blood cellcount, and lowred blood cellsandplatelets.

CLL is the second most common type of leukemia in adults. It is a type of cancer in which the bone marrow makes too many maturelymphocytes(a type of white blood cell).

ALL is a type of leukemia in which the bone marrow makes too many immaturelymphocytes. Similar to AML, the white blood cells can be high or low and oftentimes the platelets and red blood cells are low. This form of leukemia is more common in children than adults.

CML is usually a slowly progressing disease in which too many mature white blood cells are made in the bone marrow.

People with leukemia may experience:

Because these symptoms can be caused by a variety of other conditions, its important to check with your doctor if they arise.

While studies have shown men to be more atrisk than women, some other risk factors include:

While test procedures vary based on the type of leukemia, the two most common procedures are thecomplete blood count(CBC) test and the bone marrow aspiration biopsy.

CBC is a procedure used to check the redblood cell and platelet counts as well as the number and type of white bloodcells (the red cells carry oxygen, the white cells fight and prevent infection,and platelets control bleeding). A bone marrow aspiration biopsy involvesremoving a sample of bone marrow, including a small piece of bone by insertinga needle into the hipbone. The sample is then examined for abnormal cells.

Treatment for leukemia varies depending on the type and specific diagnosis.

The treatment for acute leukemias may be lengthy up to two years in ALL and is usually done in phases. The first phase, known as remission induction therapy, involves administering several chemotherapy drugs over a several-week period. The goal is to destroy as many cancer cells as possible to achieve a remission (in which cancer cells are undetectable, but small amounts are still present).

The second phase, known aspost-remission or consolidation therapy, seeks to kill leukemia cells thatremain after remission induction therapy. This phase may involve chemotherapyand/or a stem cell transplant.

Additional treatments may also be necessary. ALL patients, for example, may receive special treatment to prevent the disease from recurring in the spinal cord or brain.

The treatment for CML has been revolutionized by the advent of the oral medication imatinib and the second- and third-generation drugs known as tyrosine kinase inhibitors (TKIs). These are oral medications that work to inhibit the function of theBCR-ABLprotein. Many patients take these medications for the rest of their lives. In rare instances, a patient may require a stem cell transplant.

Some patients with CLL are recommended formonitoring and observation. Others,usually those with symptoms or low red cell or platelet counts, requiretreatment. Such treatment may involve intravenous chemotherapy, but often withoral therapy with pills that inhibit the function of a key protein, Brutonstyrosine kinase.

Treatments for leukemia can include:

Drugs that harness the immune system in fighting leukemia have shown considerable promise. Some monoclonal antibodies synthetic versions of immune system proteins are already in use to treat certain forms of leukemia and others are being studies in clinical trials.

Another form of immunotherapy, immune checkpoint inhibitors, which release a pent-up immune system attack on tumor cells, is being tested in several forms of leukemia. Cancer vaccines, which boost the immune systems ability to fight cancer, are being studied for use in leukemia.

CAR T-cell therapy, which uses modified immune system T cells to better target and kill tumor cells, has achieved impressive results in trials involving children and adults up to age 25 with relapsed ALL.

Research into new treatments for adult leukemia is moving along several tracks in addition to immunotherapy.

By tracking the specific abnormal genes within leukemia cells, physicians are increasingly able to tailor treatment to the unique characteristics of the disease in each patient. Targeted drugs such as imatinib and dasatinib, for example, are now used in treating patients with ALL whose leukemia cells have an abnormality known as the Philadelphia chromosome. Targeted agents including IDH or FLT3 inhibitors, which zero in on proteins made from mutated genes, have been approved to treat some patients with AML, while other such inhibitors are being tested in clinical trials.

New tests make it possible to detect ever smaller amounts of leukemia that remain after treatment. Investigators are exploring how these minute levels may influence a patients prognosis and how they might impact treatment.

Researchers are testing whether treatment periods for certain drugs can be safely reduced in some patients. For instance, studies are under way to determine if drugs such as imatinib, which are currently taken for life, can be safely stopped in some patients with CML. Researchers hope to test whether treating patients with CLL with the drug ibrutinib plus other medicine for a fixed amount of time is safe and effective.

Patients may consider treatment through a clinical trial.Dana-Farber currently has more than 30 clinical trials for adult leukemia. A national list of clinical trials is available atclinicaltrials.gov.

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Adult Leukemia: What You Need to Know - Dana-Farber Cancer Institute

What are stem cells and what role can they play in medicine andresearch? Stem cell research offers exciting possibilities in terms ofregenerative medicine. However, there are ethical controversies and challengesimpeding the fields advancement. In this article, The Medium presents a briefoverview of the unique abilities, applications, and challenges of stem cells.

According tothe National Institute of Health, stem cells are able to develop into manydifferent cell types in the body during early life and growth. When stem cellsdivide, the new cell can become another stem cell or it can become aspecialized cell such as a muscle cell or a brain cell. Stem cells provide newcells for the body as it grows and replaces damaged or lost specialized cells.The two unique properties of stem cells are that the stem cells can dividemultiple times to produce new cells, and as they divide, the stem cells cangenerate other types of cells found in the body.

In organs suchas the gut and the bone marrow (the soft tissue inside most bones), stem cellsroutinely divide to replace damaged tissue. However, in other organs such asthe heart, stem cells require certain physiological conditions to facilitate celldivision.

Stem cells canbe divided into two categories: embryonic stem cells and adult stem cells.Embryonic stem cells are derived from a blastocystan early stage of embryodevelopment. The blastocyst contains the trophectoderm, which will eventuallyform the placenta, and the inner cell mass, which will develop into the embryo,and later into the organism. Stem cells taken from the inner cell mass arepluripotentthey can develop into any cell type in the body. The embryonic stemcells used in research are sourced from unused embryos that were a result of anin vitro fertilization procedure and were donated for scientific research.

Adult stemcells also have the ability to divide into more than one cell type; however,they are often restricted to certain types of cells. For example, an adult stemcell found in the liver will only divide into more liver cells. In 2006, ShinyaYamanaka, a Japanese stem cell researcher, discovered how to program inducedpluripotent stem cells (iPSCs). iPSCs are adult cells which have beengenetically reprogrammed into a pluripotent embryonic stem cell-like state.Yamanaka won the Nobel Prize for Physiology or Medicine alongside Englishdevelopmental biologist Sir John Gurdon in 2012 for this important discovery.

There arenumerous ways in which stem cells can be used. Firstly, human embryonic stemcells can provide information as to how cells divide into tissues and organs.Abnormal cell division can cause cancer and birth defects, and therefore, amore comprehensive understanding of the processes underlying cell division maysuggest new therapy strategies. Another beneficial avenue involves drug testingas new medications could be tested on cells developed from stem cells in thelab. However, a challenge for researchers is to create an environment identicalto the conditions found in the human body.

Finally, stemcells present exciting possibilities in cell-based therapies and regenerativemedicine. Instead of relying on a limited supply of donated organs and tissuesto replace damaged and destroyed ones, stem cells could be directed to developinto the desired cell type and treat diseases such as heart disease, diabetes,and spinal cord injuries. For example, healthy heart muscle cells could begenerated from stem cells in a laboratory and transplanted into an individualwith heart disease. However, there is still research and testing which needs tobe conducted before researchers can confirm how to effectively and safely usestem cells to treat serious disease.

As explainedby the University of Rochesters medical centre, there are several challengesassociated with stem cells. Researchers first need to learn about how embryonicstem cells develop so that they can control the type of cells generated fromstem cells. Scientists also need to determine how to ensure that the cellsdeveloped from stem cells in the lab are not rejected by the human body. Adultpluripotent stem cells are found in small amounts in the human body and arehard to grow in the lab. There are also numerous ethical issues surrounding theuse of embryonic stem cells as some individuals believe that using cells froman unused blastocyst and consequently, rendering it incapable to develop intoan organism, is similar to destroying an unborn child. Others argue that theblastocyst is not a child yet as it needs to be imbedded into the mothersuterus wall before it has the chance to develop into a fetus. Supporters ofembryonic stem cell research also say that many surplus blastocysts aredestroyed in fertility clinics and can be better used to research medicaltreatments which could save peoples lives.

Students canlearn more about stem cells in BIO380H5: Human Development. Furthermore, Dr.Ted Erlicks lab at UTM is researching how complex neural circuits developfrom an initial population of stem cells. Stem cell research offers promisingavenues of treating diseases and understanding how humans develop. However,there is still a substantial amount of research which needs to be conducted andethical concerns which need to be appropriately addressed and resolved.

More:
Stem cells' role in medicine and research - The Medium

The stocks of Magenta Therapeutics (NASDAQ:MGTA) and Molecular Templates (NASDAQ:MTEM) bolted skywards last week, to the tune of 39% and 28% respectively.

Cutting-edge gene-editing therapies, chimeric antigen receptor T-cell (CAR-T) treatments, and stem cell transplants all require priming or conditioning regimens. Doctors today utilize older chemotherapy drugs or radiation, which often lead to infection or hospitalization. Magenta Therapeutics and Molecular Templates are among the companies seeking to develop less toxic, non-chemotherapy options for patients.

Image source: Getty Images.

On Nov. 18, Molecular Templates and Vertex Pharmaceuticals (NASDAQ:VRTX) forged a discovery and development collaboration to create novel targeted conditioning regimens applicable to gene-editing, CAR-T, and stem cell transplants. Vertex shelled out $38 million of up-front cash and an equity investment in Molecular Templates. The stock barely flinched, losing $0.03 from the prior day's closing price.

The next day, Nov. 19, Vertex and its collaborator CRISPR Therapeutics announced positive safety and efficacy data for the gene-editing therapy CTX001 in its first two patients. One patient had severe sickle cell disease; the other had beta thalassemia. These interventions edit a patient's genome, potentially allowing for a one-time curative treatment. Both patients received the chemotherapy busulfan prior to CTX001.

Revisiting the prior day's collaboration announcement, biotech investors focused on comments made by Vertex about how Molecular Templates could benefit the CTX001 program.

Vertex's Chief Scientific Officer David Altshuler said,

"We believe that gene editing holds significant promise in the treatment of severe hemoglobinopathies such as sickle cell disease and beta thalassemia, and Molecular Templates' unique technology platform could play an important role in creating a targeted conditioning regimen that could replace chemotherapy currently required in conditioning regimens and thus enhance the overall future treatment experience for patients."

Investors jumped on the message from Vertex, one of the biotech industry's stalwarts: Non-chemotherapy conditioning approaches are the future for gene and cell therapies.

In response, the stocks of other companies focused on achieving that goal (like Magenta) shot up. In fact, Magenta's nearly 40% gain in share price came during a week when it didn't release any news.

Magenta plans to present data on Dec. 6 at the American Society of Hematology's Annual Meeting for its lead program CD117-ADC. Targeting a protein on hematopoietic stem cells called CD117, the treatment eliminated mutated cells without the need for chemotherapy or radiation. Magenta believes CD117-ADC can potentially be used for genetic diseases like sickle cell disease, prior to either gene therapy or hematopoietic stem cell transplantation (HSCT).

Magenta and Molecular Templates are not the only players in the field. Forty Seven and bluebird bio paired up earlier this month to develop antibody-based conditioning regimens for HSCT. According to the World Health Organization, 50,000 HSCT procedures are performed annually worldwide.

Furthermore, recently approved CAR-T for cancer, such as Kymriah from Novartis or Yescarta from Gilead Sciences, require three days of cyclophosphamide and fludarabine. Developers of these and next-generation CAR-T treatments also seek to eliminate chemotherapy or radiation.

Patients greatly need less toxic methods to prepare them for gene- and cell-based therapies, or stem cell and bone marrow transplants. Many patients, particularly the elderly, are deemed ineligible for these interventions because the toxicity could be too severe. Any success could have broad implications for the treatment of cancers and genetic diseases.

While a variety of successful approaches may ultimately emerge, Magenta has taken an early lead with CD117-ADC. Molecular Templates, with Vertex as a seasoned partner by its side, may soon leap onto the scene with a targeted approach derived from its "engineered toxin bodies" platform.

The investor takeaway is clear: New treatment modalities will be dependent on non-chemotherapy conditioning. Investors in biotech companies that can figure out that piece of the puzzle should be richly rewarded.

See more here:
2 Small-Cap Biotechs That Soared Last Week - Motley Fool

Allee McKee exchanges a glance with her 11-year-old twin brother Matthew as he receives a blood transfusion Oct. 29 at St. Louis Children's Hospital.

RACHAEL LONG

In late October, laughter permeated The Olson Family Garden at St. Louis Children's Hospital as Matthew McKee got the chance to do something abnormal: run and play outside.

The 11-year-old Trinity Lutheran student was diagnosed in August with aplastic anemia, a rare condition in which damage to stem cells hinders the bone marrow's production of red blood cells, white blood cells and platelets.

According to the Aplastic Anemia and MDS International Foundation, between 600 and 900 people in the United States learn they have aplastic anemia each year. Anyone can be diagnosed with the disease, but according to the foundation, aplastic anemia is most commonly diagnosed in children, young adults and older adults.

Before his diagnosis, Matthew was experiencing life the way you'd expect a young person his age would -- by spending time with his friends, attending school, tagging along on float trips and annoying his twin sister, Allee.

Just before the first week of school, strange things started happening to Matthew.

Roughly two weeks before he was hospitalized, Allee and Matthew had been wrestling when -- as part of what could only have been an epic battle between siblings -- Allee bit her brother. Their father, Jason McKee, recalled seeing a "horrific" bite mark near his son's shoulder.

"I was so angry with Allee," Jason remembered. "I said, 'Why would you bite him that hard?' And she said, 'Dad, I didn't bite him that hard.'"

On Aug. 3, Matthew returned from a float trip covered in "significant" bruising, and as his mother, Wendy McKee, recalls, "more bruising than what it should be for a normal 11-year-old boy."

Three days later, Matthew had a nosebleed that lasted for three hours. Not normal; we'll take him to see the doctor tomorrow, his mother thought.

But when tomorrow came, Matthew awoke with something his parents described to look like a "nasty rash" called petechiae, a condition that causes pinpoint, round spots to appear on the skin as a result of bleeding.

That day, the McKees took Matthew to Saint Francis Medical Center in Cape Girardeau. A few blood tests confirmed some bad news: Matthew would have to be taken to St. Louis, immediately.

Transported north by way of ambulance, Wendy and Matthew left to find answers -- they have not returned home since.

On Dec. 25, 2007, Allee was born 2 minutes before Matthew -- an important time difference, depending on who you ask.

The siblings have what their mother calls a "love-hate" relationship. It's a phase -- she hopes.

But when Matthew got sick, Allee didn't hesitate for a moment. Her parents recall one of the first things Allee said: "What can I do?"

Allee McKee maintains her balance while running atop a ledge Oct. 29 in The Olson Family Garden at St. Louis Children's Hospital.

RACHAEL LONG

"We were blessed with twins 11 years ago for a reason," Wendy said with a smile.

While half of her family has been living temporarily in St. Louis, Allee has had to go on with life in Cape Girardeau as though things are normal. But when a sibling is suddenly diagnosed with a life-threatening illness, "normal" doesn't exist.

"Oh, it's really made an impact [on Allee]," Jason said. "You know, an 11-year-old girl, it's hard for her to express her emotions. But inside, you know there's just an ocean of emotion ... about this. ... We think of Matthew, but it's so much her story, too."

Though no one can take the place of her twin, Jason said it helps Allee to have extended family and friends around.

If everything else about Allee's life has changed, her relationship with Matthew is ever the annoying, hilarious, infuriating, loving sibling relationship it always has been.

Allee McKee erupts in laughter after grossing out her 11-year-old twin brother Matthew during a break in a day of medical appointments Oct. 29 in The Olson Family Garden at St. Louis Children's Hospital.

RACHAEL LONG

Just before she was anesthetized for the transplant, Jason said Allee was beginning to feel anxious about the imminent procedure. Not for a moment forgetting the many ways to leverage something over her younger twin, Allee said, "He's gonna owe me big."

More than a month later, sitting beside Matthew while he received a blood transfusion, Allee's message remained the same. Asked how she feels about the chance to donate blood marrow to her brother, Allee, with a mischievous grin, said, "It's good because I can bring it up and he owes me."

Before they knew what was making Matthew sick, his parents said all signs pointed to leukemia.

"He had zero platelets," Wendy said of the initial blood tests run at Saint Francis.

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In fact, doctors had to rule everything else out before they could officially diagnose Matthew with aplastic anemia. Once diagnosed, the discussion surrounding odds for locating a donor tissue match was no walk in the park.

When Matthew's doctors laid out his treatment options, Jason remembered them saying, "First and foremost, we see he has a sibling; we would like to test her to see if a bone marrow transplant is even a possibility."

A successful bone-marrow transplant can cure a case of aplastic anemia in a young person, where other treatment options may be more complicated and less effective.

Allee had a 1-in-4 chance of being the right genetic match to donate and save her brother's life. Other treatment options presented to the family, as Jason remembers them, included a "drug-induced protocol that had a lesser success rate but [one that] still would have given him a chance," and placement on a national donor list, an option with a higher risk of rejection.

"As a realist, when you hear a 25% chance, I'm already thinking of Step 2, thinking about the [other options], just [crossing] my fingers and praying that Allee is a match," Jason said.

Matthew's donor needed to be a human leukocyte antigen protein match, not a blood-type match. One of Matthew's doctors in the hematology and oncology clinic at St. Louis Children's Hospital explained the science behind a human leukocyte antigen protein match.

"You get half of those proteins from your mom and half of those proteins from your dad," said Dr. Shalini Shenoy, a pediatric oncologist and the director of the pediatric stem-cell transplant program at St. Louis Children's Hospital. "Fifty percent of the time, you're going to be half-matched, so you'll get the right set from mom and maybe the wrong set from dad. ... Twenty-five percent of the time, you share no antigens, no proteins at all because you got the wrong set."

But the other 25% of the time, as was the case for Allee and Matthew, the donor and recipient will be a full match.

Shenoy explained Allee could not have been a better match for her brother, even if she had been born his identical twin.

The fact Allee and Matthew are non-identical twins, Shenoy said, means there was no guarantee they would be a match. But, hypothetically, if Matthew had an identical twin, Shenoy said there would have been "some concern" about that kind of match.

Cape Girardeau twins Matthew and Allee McKee wrestle in The Olson Family Garden during a day of medical appointments Oct. 29 at St. Louis Children's Hospital.

RACHAEL LONG

"Something happened to [Matthew's] bone marrow. His immune system just worked against his bone marrow and knocked it off. Would that have [been the case for an] identical twin? It would have been hard to say. Even if the twin was normal at the time of the transplant, would that bone marrow have held? Or would it have done the same thing again?

"Luckily they were matched, and so that made her the best donor for him," Shenoy said.

Before Matthew could receive his sister's donation, his medical team had to eliminate what was left of his immune system by way of chemotherapy. It was a 21-day process involving an isolation room and constant fear of infection.

"[There were] so many things that could be just devastating, that could make him gravely ill," Jason said. "Those 21 days, they lasted forever."

The treatment Matthew went through didn't just cost him his immune system, it also cost him his hair.

"He's written in school papers that his best attribute is his hair," Jason said. "You tell a kid he is going to lose his hair, and he fought that until the bitter end."

"He spends more time in the bathroom than myself and his sister, doing his hair," Wendy said.

Cape Girardeau twins Allee and Matthew McKee sit near their mother, Wendy McKee, as they laugh at a joke made by their father, Jason McKee (not pictured) on Oct. 29 in the Olson Family Garden at St. Louis Children's Hospital.

RACHAEL LONG

After being told he would lose most or all of his hair, Matthew stubbornly -- and with no small amount of pride -- held on to about 25% to 30% of his hair, Jason said.

"He's pretty proud of that," Jason said, laughing.

Despite prolonged isolation, chemotherapy, a bone-marrow transplant, being away from home and missing school, Matthew never lost his good spirits.

"He's had a smile on his face every day," Wendy said. "He is a very good-spirited boy; he kind of goes with the flow, and he may not like what he's doing, but by God, he's got a very positive attitude when he does it."

Matthew must remain in St. Louis for 100 days after his transplant, which took place Sept. 19. After that time is up, barring any complications, Matthew will finally return home, though he will be restricted to settings with a small number of people and limited visits from friends.

"He gets to go home but stay at home, more or less," Jason said. "We're going to have to be super, duper diligent in screening anybody that comes in to make sure they don't have any symptoms of any kind of illness."

Because his immune system had to be completely erased in order to receive a transplant, Matthew will also need to be revaccinated before he can return to life as he knew it.

"He has the immune system of a newborn," Jason said.

Some of those vaccinations he will be able to receive a year after his transplant; but for others, the waiting period is longer.

"We're looking at two years out before he can actually live life like a normal teenage boy," Wendy said.

Matthew's parents are optimistic he could return to Trinity Lutheran for the next school year.

Matthew McKee sits on top of the world during a day of medical appointments Oct. 29 at St. Louis Children's Hospital.

RACHAEL LONG

Though Wendy and Matthew have not returned to Cape Girardeau since August, life back home hasn't fallen apart -- not by any means.

"We have a wonderful family at home that is supporting us," Wendy said, noting family members have brought her winter clothes during visits, as the temperature was upwards of 90 degrees when she left town.

The family is living temporarily in a furnished Ronald McDonald apartment, keeping them close by the hospital and allowing Matthew distance from outside germs. Allee is mostly in Cape Girardeau, but she often makes trips to see her family.

Everywhere the McKees go, a community waits to support them.

"You don't realize how supportive people can be until you're put in a situation where you're in need of help," Wendy said.

A family member set up a GoFundMe fundraiser -- which may be found at gofundme.com/f/team-mckee-matthewallee-bone-marrow-transplant -- for the McKee family to help with medical bills, everyday expenses and other costs they have incurred over the last three months.

"It's so hard to take a gift from somebody," Jason said. "But so many people have come to me and said, 'This is all we can do for you, and we've got to do something.'"

But that's not the only way the community has stepped forward to help the McKees. Trinity Lutheran School in Cape Girardeau has hosted fundraisers and a blood drive in Matthew's honor.

The school even took the time to recognize Allee during one of her volleyball games.

"They had her stand up and said some words, and they gave her a standing ovation," Jason said. "It was just very special for her."

The school even sold T-shirts with the words "Team McKee" as a fundraiser for the family.

"The community has just been wonderful ... Cape Girardeau, his school, family and friends -- they've all just been amazing," Jason said.

There is no easy way to navigate life after sickness touches a family, especially for parents of a sick child. But the McKees continue to give thanks in spite of their situation.

"I am most thankful the Lord is giving us a road that can be traveled," Jason said. "Because some patients here don't ... as bad as the road is gonna be, at least there is a road."

Read more:
Thankful People -- 'He's gonna owe me big': Matthew McKee receives bone-marrow donation from twin sister Allee - Southeast Missourian

CTX001 safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crisesin the first severesickle cell disease(SCD) patient receiving the therapy, according to preliminary data from a Phase 1/2 clinical trial.

CTX001 is a CRISPR-based gene editing therapy developed byCRISPR TherapeuticsandVertex Pharmaceuticals as a potential treatment for hemoglobin-associated diseases, includingSCD and beta-thalassemia.

It uses the CRISPR-Cas9 gene editing system to genetically modify a patients hematopoietic (bone marrow) stem cellsto produce high levels of fetal hemoglobin in red blood cells, which are then delivered back to the patient as part of a stem cell transplant.

The CRISPR-Cas9 system, which is similar to the editing system used by bacteria as a defense mechanism, allows researchers to edit parts of the genome by adding, removing, or changing specific sections of DNA.

Fetal hemoglobin, the main form of oxygen-carrying hemoglobin in the human fetus and newborn, largely disappears between six months to one year after birth, being replaced by its adult form.

Since the adult form is the one containing the defective component of hemoglobin in people with SCD and beta-thalassemia, an artificial increase of fetal hemoglobin has the potential to compensatefor the defective hemoglobin produced by these patients and reduce or prevent theirsymptoms.

The open-label, multi-center Phase 1/2 CLIMB-SCD-121 study (NCT03745287) is currently evaluating the safety and effectiveness of a single administration of CTX001 in people ages 18 to 35 with severe SCD.

The trial, which is expected to enroll up to 45 people, is stillrecruiting at 12 clinical sites in the United States, Canada, and Europe. Participants will be followed for approximately two years after treatment, and have the opportunity to enter a long-term follow-up study.

Before receiving CTX001, participants will undergo myeloablativechemotherapy, a strategy that kills cells in the bone marrow, thereby lowering the number of blood-forming cells. This way, the stem cell transplant will have more chances to rebuild a healthy bone marrow.

Researchers will first determine when the transplanted modified cells begin to produce mature blood cells in the patients, a process known as engraftment. After confirmation of engraftment, safety and effectiveness will be assessed as part of the trials primary and secondary goals.

One primary goal is to assess the proportion of people with an increase of at least 20% in the production of fetal hemoglobin, starting six months after CTX001 treatment. This increase must be sustained for more than three months at the time of analysis.

Among secondary goals is determining whether CTX001 reduces the annualized rate of vaso-occlusive crises.

In February, CRISPR Therapeutics and Vertex announced the enrollment of the first patient in the CLIMB-SCD-121 study, who was recruited in the U.S. and received CTX001 in mid-2019.

Now, the companies have shared the preliminary four-month data of this patient, a 33-year-old woman who had experienced seven vaso-occlusive crises per year the annualized rate of the two years before her enrollment in the trial.

Results showed that she had a confirmed engraftment 30 days after receiving CTX001 treatment. Four months after treatment, no vaso-occlusive crises were reported and she had stopped blood transfusion treatments.

After four months, her total hemoglobin levels were 11.3 g/dL, fetal hemoglobin levels had increased from 9.1% to 46.6%, and the percentage of fetal hemoglobin-producing red blood cells had increased from 33.9% to 94.7%.

CTX001s early safety profile was consistent with that previously reported for myeloablative chemotherapy followed by stem cell transplant. The woman experienced three serious adverse events, all of them resolved and considered to be unrelated to treatment.

Positive preliminary data were also announced for the first patient with beta-thalassemia receiving CTX001 in the Phase 1/2 CLIMB-Thal-111 study (NCT03655678).

We are very encouraged by these preliminary data [which] support our belief in the potential of our therapies to have meaningful benefit for patients following a one-time intervention, Samarth Kulkarni, PhD, CRISPR Therapeutics CEO, said in a press release.

A webcast and presentation about these preliminary results are available on the companys website.

The data are remarkable and demonstrate that CTX001 has the potential to be a curative CRISPR/Cas9-based gene-editing therapy for people with sickle cell disease and beta thalassemia, said Jeffrey Leiden, MD, PhD, Vertexs chairman, president, and CEO.

Leiden added that the trial is still in its early phase and that he looks forward to its final results.

Early this year, CTX001 receivedfast track statusfor the treatment of sickle cell disease by theU.S. Food and Drug Administration, which is expected to accelerate CTX001s development and regulatory approval process.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

Total Posts: 94

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

Link:
1st SCD Trial Patient Shows CTX001 Gene Editing to be Safe, Effective - Sickle Cell Anemia News

Stem Cell Banking Market Report 2018-2026includes a comprehensive analysis of the present Market. The report starts with the basic Stem Cell Banking industry overview and then goes into each and every detail.

Stem Cell Banking Market Report contains in depth information major manufacturers, opportunities, challenges, and industry trends and their impact on the market forecast. Stem Cell Banking also provides data about the company and its operations. This report also provides information on the Pricing Strategy, Brand Strategy, Target Client, Distributors/Traders List offered by the company.

Description:

High potential of cord blood and tissues for the treatment of patients with autoimmune diseases is expected to propel the market growth. Moreover, currently available immunosuppressive agents such as steroids, induce long term side effects despite temporary improvements. According to the Health Research Funding, 2015, around 28% of cord blood transplants have been used to treat genetic diseases, with the most common genetic disease treated being severe combined immune deficiency, followed by aplastic anemia. According to the National Cord Blood Program, 2015, cord blood from unrelated donors has been used as an alternative to bone marrow or mobilized stem cells, as a source of hematopoietic stem cells, with over 35,000 stem cell transplants successfully performed worldwide.

Stem Cell Banking Market competition by top manufacturers/players, with Stem Cell Banking sales volume, Price (USD/Unit), Revenue (Million USD) and Market Share for each manufacturer/player; the top players including: Allergan, Plc., Galderma S.A., Integra LifeSciences Corporation, Merz Pharma GmbH & Co. KGaA., Sanofi S.A., SciVision Biotech Inc., Sinclair Pharma Plc., Suneva Medical, Valeant Pharmaceuticals International, Inc., and Anika Therapeutics, Inc.

Get Free Sample Copy Of This Report @ https://www.coherentmarketinsights.com/insight/request-sample/1354

Important Features that are under offer & key highlights of the report:

What all regional segmentation covered? Can the specific country of interest be added?Currently, the research report gives special attention and focus on the following regions:North America (U.S., Canada, Mexico), Europe (Germany, U.K., France, Italy, Russia, Spain etc), South America (Brazil, Argentina etc) & Middle East & Africa (Saudi Arabia, South Africa etc)** One country of specific interest can be included at no added cost. For inclusion of more regional segment quote may vary.

What all companies are currently profiled in the report?The report Contain the Major Key Players currently profiled in this market.** List of companies mentioned may vary in the final report subject to Name Change / Merger etc.

Can we add or profiled new company as per our need?Yes, we can add or profile new company as per client need in the report. Final confirmation to be provided by the research team depending upon the difficulty of the survey.** Data availability will be confirmed by research in case of a privately held company. Up to 3 players can be added at no added cost.

Can the inclusion of additional Segmentation / Market breakdown is possible?Yes, the inclusion of additional segmentation / Market breakdown is possible to subject to data availability and difficulty of the survey. However, a detailed requirement needs to be shared with our research before giving final confirmation to the client.** Depending upon the requirement the deliverable time and quote will vary.

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Stem Cell Banking Market Dynamics in the world mainly, the worldwide 2018-2026 Stem Cell Banking Market is analyzed across major global regions. CMI also provides customized specific regional and country-level reports for the following areas:

Region Segmentation:

North America (USA, Canada and Mexico)Europe (Germany, France, UK, Russia and Italy)Asia-Pacific (China, Japan, Korea, India and Southeast Asia)South America (Brazil, Argentina, Columbia etc.)Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Further in the report, the Stem Cell Banking market is examined for Sales, Revenue, Price and Gross Margin. These points are analyzed for companies, types, and regions. In continuation with this data, the sale price is for various types, applications and region is also included. The Stem Cell Banking industry consumption for major regions is given. Additionally, type wise and application wise figures are also provided in this report.

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In this study, the years considered to estimate the market size of 2018-2026 Stem Cell Banking Market are as follows:History Year: 2015-2017Base Year: 2017Estimated Year: 2018Forecast Year 2018 to 2026

Read more here:
Stem Cell Banking Market to Expand Steadily in the Coming Years till 2018-2026 - Crypto Journal

Drugs commonly used to treat arthritis may help to prevent breast cancer spreading to the bone, where it is incurable, new research suggests.

In a major new study published in Nature Communications, scientists propose that NHS arthritis drugs anakinra, canakinumab and sulfasalazine could in future be repurposed to help treat breast cancer, following the discovery of the role of bone marrow in the spread of the disease to the bone.

The study, largely funded by Breast Cancer Now, found that bone marrow releases a protein called interleukin 1-beta (IL-1) which encourages breast cancer cells to form secondary tumours once they reach the bone.

Crucially, the scientists at The University of Manchester and The University of Sheffield established that the process started by this molecule can be blocked by drugs already used in treating arthritis, with anakinra found to be able to prevent breast cancer forming secondary tumours in the bone in a study in mice.

While further research is needed to understand how these drugs may interact with the immune system or work together with other cancer therapies, it is hoped the findings could be quickly advanced into trials in women with breast cancer to try to prevent the disease spreading to the bone.

Research and care charity Breast Cancer Now said the findings offered another promising step in repurposing existing drugs to try to prevent the spread of breast cancer, following the recent addition of osteoporosis drugs bisphosphonates to NHS breast cancer treatment for certain patients.

Breast cancer is the UKs most common cancer, with around 55,000 women and 370 men being diagnosed each year and around 11,500 women still losing their lives each year in the UK.

Almost all of these deaths are attributable to secondary breast cancer, where breast cancer has spread to form tumours in other parts of the body. While secondary breast cancer (also known as metastatic breast cancer) can be controlled for some time, it currently cannot be cured.

One of the most common parts of the body for breast cancer to spread to is the bone, which can cause debilitating symptoms such as joint pain or fractures that often require surgery.

Special types of cells, called breast cancer stem cells, are thought to be responsible for the disease spreading around the body with previous research suggesting that healthy cells in different parts of the body can release certain molecules that help cancer stem cells settle and grow in new locations.

In a new study, research teams led by Dr Rachel Eyre and Professor Rob Clarke at The University of Manchester and Dr Penelope Ottewell from the Department of Oncology and Metabolism at The University of Sheffield investigated the growth of breast cancer cells in the lab and in mice to establish what helps the disease settle and grow in this location. They discovered the importance of certain factors released by the bone, and these findings were supported using data from patients with secondary breast cancer1.

The researchers first grew human breast cancer cells using liquid that human bone marrow had previously been grown in. They found that these cancer cells grew into tumours more easily than breast cancer cells that werent exposed to bone marrow liquid, suggesting bone marrow releases a molecule that helps cancer growth.

By tracking which signalling pathways2 became active in breast cancer cells after they had been exposed to bone marrow, the researchers discovered that the molecule IL-1 (which is released by bone marrow) was responsible for helping breast cancer stem cells grow into tumours.

They found that IL-1 activates a signalling pathway called NFKB/CREB-Wnt, which promotes the formation of secondary tumours a discovery that identifies multiple new targets (IL-1 receptor, NFKB, Wnt) for drugs to try to prevent the growth of breast cancer tumours in the bone.

Drugs that can inhibit the action of IL-1 already exist and are used in treating other conditions on the NHS. The researchers tested whether blocking the effect of IL-1 with clinically available arthritis drugs such as anakinra, as well as another drug, currently in trials for treating cancer, called vantictumab, could prevent the formation and growth of secondary breast cancer in the bone in mice.

They found that blocking the role of IL-1 using these drugs significantly reduced the ability of breast cancer cells to form secondary tumours in the bone in mice. For example, following treatment with anakinra, only 14% of mice developed secondary tumours in the bone, compared to 42% of controls.

Research is ongoing to understand how blocking the action of IL-1 to stop breast cancer spreading may affect the immune system, and whether drugs such as anakinra, canakinumab and sulfalazine could work with existing therapies including bisphosphonates to prevent the spread of the disease to the bone. With these drugs being well-tolerated and already in use in treating arthritis, the authors hope the findings could be quickly progressed into clinical trials for breast cancer in the future.

The researchers are also now working to understand whether the same signalling pathway (NFKB/CREB-Wnt) may be important in the spread of breast cancer to other parts of the body such as the liver or lungs.

The study was largely funded by Breast Cancer Now, with additional support from Weston Park Cancer Charity and the Medical Research Council.

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Arthritis drugs could be repurposed to help prevent breast cancer spreading to the bone, study suggests - The University of Manchester

CAMBRIDGE, Mass. & GAITHERSBURG, Md.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, and MaxCyte, Inc., a global cell-based therapies and life sciences company, today announced a clinical and commercial license agreement under which Vor will use MaxCytes Flow Electroporation technology to produce eHSCs and initiate Investigational New Drug (IND)-enabling studies to accelerate its progress towards the clinic.

Under the terms of the agreement, Vor obtains non-exclusive clinical and commercial use rights to MaxCytes Flow Electroporation technology and ExPERT platform to develop up to five engineered cell therapies, including VOR33, Vors lead eHSC candidate, which is in development for acute myeloid leukemia (AML). In return, MaxCyte will receive undisclosed development and approval milestones and sales-based payments in addition to other licensing fees.

Vor will use MaxCytes cell engineering platform to deliver its gene editing machinery into hematopoietic stem cells to remove biologically redundant cell surface proteins that are also expressed on blood cancer cells. Once the eHSCs are transplanted into a cancer patient, these cells are effectively hidden from complementary targeted therapies that target the relevant protein, while diseased cells are left vulnerable to attack. Vors approach thereby could unleash the potential of targeted therapies by broadening the therapeutic window and improving the utility of complementary targeted therapies.

MaxCyte is a leader in GMP electroporation technology, and we are thrilled that this agreement provides us with long-term access to a platform technology applicable to a pipeline of eHSC programs used to treat AML and other blood cancers, said Sadik Kassim, Ph.D., Chief Technology Officer of Vor. As we build on promising in vivo data from our lead candidate VOR33, we can now expand our manufacturing capabilities to support later-stage studies, regulatory filings and commercialization of VOR33.

MaxCytes ExPERT instrument family represents the next generation of leading, clinically validated, electroporation technology for complex and scalable cellular engineering. By delivering high transfection efficiency with enhanced functionality, the ExPERT platform delivers the high-end performance essential to enable the next wave of biological and cellular therapeutics.

We look forward to expanding our relationship with Vor Biopharma as the company pioneers a potential future standard of care in hematopoietic stem cell transplants for cancer patients in need, said Doug Doerfler, President & CEO of MaxCyte. This agreement represents another key business milestone for MaxCyte, emphasizing the value of our technology platform applied to next-generation engineered cell therapies that may make a true difference in patient outcomes.

About VOR33Vors lead product candidate, VOR33, consists of engineered hematopoietic stem cells (eHSCs) that lack the protein CD33. Once these cells are transplanted into a cancer patient, CD33 becomes a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. In so doing, Vor aims to improve the therapeutic window and effectiveness of CD33-targeted therapies, thereby potentially broadening the clinical benefit to patients suffering from AML.

About Vor BiopharmaVor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. By removing biologically redundant proteins from eHSCs, these cells become inherently invulnerable to complementary targeted therapies while tumor cells are left susceptible, thereby unleashing the potential of targeted therapies to benefit cancer patients in need.

Vors platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vors lead program has been published in Proceedings of the National Academy of Sciences.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.

About MaxCyteMaxCyte is a clinical-stage global cell-based therapies and life sciences company applying its proprietary cell engineering platform to deliver the advances of cell-based medicine to patients with high unmet medical needs. MaxCyte is developing novel CARMA therapies for its own pipeline, with its first drug candidate in a Phase I clinical trial. CARMA is MaxCytes mRNA-based proprietary therapeutic platform for autologous cell therapy for the treatment of solid cancers. In addition, through its life sciences business, MaxCyte leverages its Flow Electroporation Technology to enable its biopharmaceutical partners to advance the development of innovative medicines, particularly in cell therapy. MaxCyte has placed its flow electroporation instruments worldwide, including with all of the top ten global biopharmaceutical companies. The Company now has more than 80 partnered programme licenses in cell therapy with more than 45 licensed for clinical use. With its robust delivery technology platform, MaxCyte helps its partners to unlock the full potential of their products. For more information, visit http://www.maxcyte.com.

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Vor Biopharma and MaxCyte Announce Clinical and Commercial License Agreement for Engineered Hematopoietic Stem Cells (eHSCs) to Treat Cancer -...

NEW YORK, Nov. 26, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company is proud to be a gold sponsor of the 30th International Symposium on ALS/MND.

The symposium will take place December 4 6, 2019, at the Perth Convention and Exhibition Centre in Perth, Australia. The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields.

Ralph Kern MD MHSc, BrainStorms Chief Operating and Chief Medical Officer, will deliver a podium presentation: Modulation of innate immunity by MSC-NTF (NurOwn) cells correlates with ALS clinical outcomes, on December 4, from 11:50 12:10 pm AWST during the opening day Clinical Trials Session. In addition to the podium presentation, the Company will also present Poster 153: MSC-NTF Differentiation Increases the Neurotrophic Effects of MSC Cells: Live Imaging Analysis, that directly demonstrates the neuroprotective effects of NurOwn in a neuronal cell culture model.

Our fully-enrolled phase 3 clinical trial is one of the most advanced clinical programs in ALS, stated Chaim Lebovits, President and CEO of BrainStorm. He added, The International Symposium on ALS/MND is an important venue to update the community on our clinical and scientific efforts towards the advancement of therapies that may address the unmet needs of those living with ALS. BrainStorm Cell Therapeutics is proud to serve as a sponsor of this important annual symposium which underscores our commitment to the international community of ALS and MND patients, their families and their caregivers.

Ralph Kern, MD, stated, It is a privilege to present our innovative biomarker and preclinical research at the International Symposium on ALS/MND. He added, Every year, symposium participants gather together and discuss the opportunities and the challenges that we will face during the upcoming year. Research and medical breakthroughs for the ALS and MND community continue to make significant progress and we look forward to sharing our insights and engaging with colleagues from around the globe. The International Symposium on ALS/MND reminds us how far we have come in investigational therapies and how much more progress is still needed to bring patients a better and more promising future.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at http://www.brainstorm-cell.com.

The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields. The Symposium is planned as two parallel meetings, one on biomedical research and the other on advances in the care and management of people affected by ALS/MND. Joint sessions consider issues of mutual concern, challenging current views and practices.

Safe-Harbor Statements

Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect," "likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

BRAINSTORM CONTACTS:Investors:Uri Yablonka, Chief Business OfficerBrainStorm Cell Therapeutics IncPhone: : +1-201-488-0460Email: uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839Email:sean.leous@icrinc.com

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BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND - GlobeNewswire