Monthly Archives: August 2021

John Davy of Hancock marks two birthdays. The first isNov. 16, the day he was born in 1941. The second is Jan. 6,the day in 2014 when he received a lifesaving stem-cell transplantthanks to a complete stranger.

Now, John and his wife Sandhy Kale have become advocates for Be the Match, the national stem cell registry that found John his rare genetic match.

How often in the world do you get to save someones life? Davy asked. You fantasize about it sometimes. Heres an opportunity for anyone between 18 and 44 to do just that.

Davy said he began feeling abnormally tired sometime in 2013. One day, he walked to the mailbox, only a few hundred feet away from his front door, and had to stop several times on his way back to his house.

I said, Thats not me. Theres something off here, Davy said.

Davy went in to the hospital for some testing, and after a few false starts looking at his heart and running stress tests, doctors performed a Complete Blood Count, or CBC.

My blood count was so low, it wouldnt support life, he said.

Thats when Davy received his diagnosis. Myelodysplastic syndrome, or MDS, a form of blood cancer.

My first thought was, OK, what are we going to do about this? Davy said. Thats when the doctor told me there was no cure.

MDS cannot be cured through usual chemotherapy or radiation treatments. However, it can be treated with bone marrow or, as with Davy, the transplant of stem cells.

After receiving a second opinion, and speaking with a doctor experienced with stem cell transplants, Davy went on the national stem cell registry, known as Be the Match.

He was told he might have to wait upwards of a year before finding his match. But Davy got lucky in only three months, a viable donor joined the registry.

Davy knows little about the man who saved his life. He was 30 years old at the time, and a member of the United States military. Be the Match allows donors and patients to connect, if both sides are interested, but while John said he would love to shake the mans hand, his donor has wished to remain anonymous.

If I could speak to him, I would thank him profusely. For someone to be that generous, to donate to someone that hes never met, is astounding, Davy said.

Joining the Be the Match registry is as simple as swabbing a cheek.

Your genetic profile goes into the system, and, if donors are found to be a match to any patients waiting for transplants, only then are they called to go through the donation process.

There are two ways to donate stem cells. In either case, the donor will first undergo two injections to increase the production of their stem cells. In the first type of donation procedure, liquid bone marrow is extracted using a needle while the donor is under anesthesia. But the much more common way to donate used about 80 percent of the time is through a blood donation.

Similar to the process for donating plasma, the donor has blood drawn, it is cycled by a machine to remove only the stem cells, and the remaining blood is returned to the donor.

The recipient of the stem cells has to undergo a process to suppress their immune system, and the donated stem cells are given to the patient.

Because the immune system has to be repressed to accept the new cells, there is danger in the procedure, and even those who successfully accept the new stem cells can experience side effects of graft-verses-host reactions.

There is no guarantee, Kale said. This is a chance. You can take it if you want. Even if it buys you four or five years, you might get to see your kids graduating, your grandkids grow up. It was worth it to us.

And for Davy, they said, there was no other option. He accepted the risk, and said hes one of the lucky ones he had one minor reaction resulting in a rash across his chest, but overall, since his transplant, he has been able to resume a normal life. Today, seven years later, he is on no medications, and has no restrictions for how he can live his life.

It is that new lease on life that Davy said convinced himself and Sandhy that they had to become involved with Be the Match on a level besides being a recipient of their services. The two are now advocates for the system, traveling to drives to tell their story, and Davy acts as a support person for patients who may be recipients of transplants, telling them about what to expect in the process.

Its crucial, Davy said, to get as many people on the registry as possible. Because matches work on how genetically compatible two people are, people of similar ethnic backgrounds are more likely to match, and your ethnicity greatly impacts the likelihood of finding a good match.

White patients are the most likely to find a match within the system, at a rate of 79 percent. Native Americans have a 60 percent chance, Hispanic people a 48 percent, Asian 47 percent, and Black people only 29 percent.

Thats why Sandhy and I try to get as many people involved as we can, Davy said. The more people in the registry, the better chance you have.

Be the Match currently has a donor drive scheduled for Aug. 14 from 10 a.m. to 2 p.m. at E. Paul Community Center at 61 South Street in Troy. To join the registry you must be between the ages of 18 and 44 and be in good general health, and committed to donating to anyone in need. If you cannot attend the physical drive, a free cheek swab kit will be mailed to you. If you are interested in a kit, text TroyFD to 61474.

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Hancock man advocates for national bone marrow and stem cell registry that saved his life - Monadnock Ledger Transcript

Inflamed mouse stem cells located in the basal layer (red) of the epidermis and FOS (green), a near-universal stress response factor essential to inflammatory memory. Credit: Christopher Cowley

When a tissue experiences inflammation, its cells remember. Pinning proteins to its genetic material at the height of inflammation, the cells bookmark where they left off in their last tussle. Next exposure, inflammatory memory kicks in. The cells draw from prior experience to respond more efficiently, even to threats that they have not encountered before. Skin heals a wound faster if it was previously exposed to an irritant, such as a toxin or pathogen; immune cells can attack new viruses after a vaccine has taught them to recognize just one virus.

Now,a new studyinCell Stem Celldescribes the mechanism behind inflammatory memory,also commonly referred to as trained immunity,and suggests that the phenomenon may be universal across diverse cell types.

This is happening in natural killer cells, T cells, dendritic cells from human skin, and epidermal stem cells in mice, says Samantha B. Larsen, a former graduate student in the laboratory ofElaine Fuchsat The Rockefeller University. The similarities in mechanism are striking, and may explain the remitting and relapsing nature of chronic inflammatory disorders in humans.

When thinking about our immune system, we default to specific immunitythat cadre of T cells and B cells trained, by experience or vaccination, to remember the specific contours of the last pathogen that broke into our bodies. But theres a less specific strategy available to many cells, known as trained immunity. The impact is shorter-lived, but broader in scope. Trained immunity allows cells to respond to entirely new threats by drawing on general memories of inflammation.

Scientists have long suspected that even cells that are not traditionally involved in the immune response have the rudimentary ability to remember prior insults and learn from experience. The Fuchs lab drove this point home in a 2017 study published inNatureby demonstrating that mouse skin that had recovered from irritationhealed 2.5 times faster than normal skin when exposed to irritation at a later date.

One explanation, the Fuchs team proposed, could be epigenetic changes to the skin cell genome itself. During inflammation, regions of DNA that are usually tightly coiled around histone proteins unravel to transcribe a genetic response to the attack. Even after the dust settles, a handful of these memory domains remain openand changed. Some of their associated histones have been modified since the assault, and proteins known as transcription factors have latched onto the exposed DNA. A once nave cell is now raring for its next fight.

But the molecular mechanism that explained this process, and how the cell could use it to respond to types of inflammation and injury that it had never seen before, remained a mystery.

So the Fuchs lab once again exposed mice skin to irritants, and watched as stem cells in the skin changed. We focused on the regions in the genome that become accessible during inflammation, and remain accessible afterwards, says Christopher Cowley, a graduate student in the Fuchs lab. We call these regions memory domains, and our goal was to explore the factors that open them up, keep them open and reactivate them a second time.

They observed about 50,000 regions within the DNA of thestem cellsthat had unraveled to respond to the threat, but a few months later only about 1,000 remained open and accessible, distinguishing themselves as memory domains. Interestingly, many of these memory domains were the same regions that had unraveled mostprodigiouslyin the early days of skin inflammation.

The scientists dug deeper and discovered a two-step mechanism at the heart of trained immunity. The process revolves around transcription factors, proteins which govern the expression of genes, and hinges on the twin transcription factors known as JUN and FOS.

The stimulus-specific STAT3 transcription factor responds first, deployed to coordinate a genetic response to a particular genre of inflammation. This protein hands the baton to JUN-FOS, which perches on the unspooled genetic material to join the melee. The specific transcription factor that sounded the original alarm will eventually return home; FOS will float away as the tumult quiets down. But JUN stands sentinel, guarding the open memory domain with a ragtag band of other transcription factors, waiting for its next battle.

When irritation strikes again, JUN is ready. It rapidly recruits FOS back to the memory domain, and the duo charges into the fray. This time, no specific transcription factor is necessary to respond to a particular type of inflammation and get the ball rolling. The system unilaterally activates in response to virtually any stressalacrity that may not always benefit the rest of the body.

Trained immunity may sound like a boon to human health. Veteran immune cells seem to produce broader immune responses; experienced skin cells should heal faster when wounded.

But the same mechanism that keeps cells on high alert may instill a sort of molecular paranoia in chronic inflammation disorders. When the Fuchs lab examined data collected from patients who suffer from systemic sclerosis, for instance, they found evidence that JUN may be sitting right on the memory domains of affected cells, itching to incite an argument in response to even the slightest disagreement.

These arguments need not always be disagreeable, as animals benefit by healing their wounds quickly and plants exposed to one pathogen are often protected against others, says Fuchs. That said, chronic inflammatory disorders may owe their painful existence to the ability of their cells to remember, and to FOS and JUN, which respond universally to stress.

The scientists hope that shedding light on one possible cause of chronic inflammatory disease may help researchers develop treatments for these conditions. The factors and pathways that we identify here could be targeted, both in the initial disease stages and, later, during the relapsing stages of disease, says Cowley. Larsen adds: Perhaps these transcription factors could be used as a general target to inhibit the recall of the memories that cause chronic inflammation.

Reference: Establishment, maintenance, and recall of inflammatory memory by Samantha B. Larsen, Christopher J. Cowley, Sairaj M. Sajjath, Douglas Barrows, Yihao Yang, Thomas S. Carroll and Elaine Fuchs, 27 July 2021, Cell Stem Cell.DOI: 10.1016/j.stem.2021.07.001

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How Cells Use Memories of Past Inflammation To Respond to New Threats - SciTechDaily

Glioblastoma multiforme (GBM) is a lethal brain tumor characterized by developmental hierarchical phenotypic heterogeneity, therapy resistance and recurrent growth. Neural stem cells (NSCs) from human central nervous system (CNS), and glioblastoma stem cells from patient-derived GBM (pdGSC) samples and cultured in both 2D well-plate and 3D monoclonal neurosphere culture system (pdMNCS). The pdMNCS model shows promise to establish a relevant 3D-tumor environment that maintains GBM cells in the stem cell phase within suspended neurospheres. Utilizing the pdMNCS, we examined GBM cell-lines for a wide spectrum of developmental cancer stem cell markers, including the early blastocyst inner-cell mass (ICM)-specific Nanog, Oct3/4,B, and CD133. We observed that MNCS epigenotype is recapitulated using gliomasphere-derived cells. CD133, the marker of GSC is robustly expressed in 3D-gliomaspheres and localized within the plasma membrane compartment. Conversely, gliomasphere cultures grown in conventional 2D culture quickly lost CD133 expression, indicating its variable expression is dependent on cell-culture conditions. Critically, this experiment demonstrates incomplete differentiation of cytoskeleton microtubules and intermediate filaments (IFs) of patient derived cells, similar to commercially available GBM cell lines. Subsequently, in order to determine whether Oct3/4 it was necessary for CD133 expression and cancer stemness, we transfected 2D and 3D culture with siRNA against Oct3/4 and found a significant reduction in gliomasphere formation. These results suggest that expression of Oct3/4,Aand CD133 suppress differentiation of GSCs.

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Gliomagenesis is orchestrated by the Oct3/4 regulatory network. - Physician's Weekly

Dublin, Aug. 03, 2021 (GLOBE NEWSWIRE) -- The "Hematopoietic Stem Cell Transplantation (HSCT) Market - Size, Share, Outlook, and Opportunity Analysis, 2019 - 2027" report has been added to ResearchAndMarkets.com's offering.

Hematopoietic stem cell transplantation is a procedure in which multipotent hematopoietic stem cells sourced from peripheral blood cells, bone marrow, or umbilical cord blood are transplanted into the patient. Hematopoietic stem cell transplantation is commonly used in the treatment of lymphoma (Hodgkin, Non-Hodgkin), leukemia, multiple myeloma, thalassemia, sickle cell anemia, and osteoporosis. It includes two transplantation sources; 1) autologous, that uses stem cells from the patient's own body, 2) and allogeneic that sources stem cells from a donor's body. According to World Health Organization (WHO), over 50,000 hematopoietic stem cell transplantation procedures are carried out globally, every year and this number is expected to increase over the years.

Market Dynamics

The global hematopoietic stem cell transplantation market is expected to witness significant growth during the forecast period owing to the increasing prevalence of leukemia and lymphoma. According to Center for Disease Control and Prevention (CDC), in the U.S., around 45,360 people were diagnosed with leukemia in 2013, leading to 23,549 fatalities (13,625 men and 9,924 women). According to the same source the condition is more prevalent among men than women. Leukemia accounts for around 3% of all new cancer cases.

Key features of the study:

This report provides in-depth analysis of the global hematopoietic stem cell transplantation market, market size (US$ Mn), and compound annual growth rate (CAGR %) for the forecast period 2020-2027, considering 2019 as the base year

It elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for this market

This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by leading players

It profiles key players in the global hematopoietic stem cell transplantation market based on the following parameters - company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments, and strategies

Key players covered as a part of this study are Pluristem Therapeutics Inc., CellGenix GmbH, Regen Biopharma Inc., Lonza Group, Kiadis Pharma, Taiga Biotechnologies, Inc., Takeda Pharmaceutical Company Limited, Escape Therapeutics, Inc., Bluebird Bio, Talaris Therapeutics, Inc., Marker Therapeutics Inc., and Stempeutics Research Pvt Ltd.

Insights from this report would allow marketers and management authorities of companies to make informed decision with respect to future product launches, government initiatives, technological upgradation, market expansion, and marketing tactics

The global hematopoietic stem cell transplantation market report caters to various stakeholders in this industry, including investors, product manufacturers, distributors, and suppliers in the hematopoietic stem cell transplantation market, research and consulting firms, new entrants, and financial analysts.

Key Topics Covered:

1. Research Objectives and Assumptions

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2. Market Overview

3. Market Dynamics, Regulations, and Trends Analysis

Market Dynamics

Drivers

Restraints

Market Opportunities

Impact Analysis

Key Developments

Pipeline Analysis

PEST Analysis

Reimbursement Scenario

Regulatory Scenario

Epidemiology

Government Initiatives

Treatment Algorithm

4. Impact Analysis of COVID-19

5. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Transplant Type, 2016 - 2027, (US$ Million)

Introduction

Market Share Analysis, 2020 and 2027 (%)

Y-o-Y Growth Analysis, 2017 - 2027

Segment Trends

Autologous

Introduction

Market Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Allogeneic

Introduction

Market Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

6. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Indication, 2016 - 2027, (US$ Million)

Introduction

Market Share Analysis, 2020 and 2027 (%)

Y-o-Y Growth Analysis, 2017 - 2027

Segment Trends

Acute Myeloid Leukemia (AML)

Introduction

Market Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Acute Lymphoblastic Leukemia (ALL)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Hodgkin lymphoma (HL)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Non-Hodgkin Lymphoma (NHL)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Multiple Myeloma (MM)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Other Non-malignant Disorders

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

7. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Application, 2016 - 2027, (US$ Million)

Introduction

Market Share Analysis, 2020 and 2027 (%)

Y-o-Y Growth Analysis, 2017 - 2027

Segment Trends

Bone Marrow Transplant (BMT)

Introduction

Market Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Peripheral Blood Stem Cells Transplant (PBSCT)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

Cord Blood Transplant (CBT)

Introduction

Size and Forecast, and Y-o-Y Growth, 2016 - 2027, (US$ Million)

8. Global Hematopoietic Stem Cell Transplantation (HSCT) Market, By Region, 2016 - 2027, (US$ Million)

Introduction

Market Share Analysis, By Region, 2020 and 2027 (%)

Y-o-Y Growth Analysis, By Region, 2017 - 2027

Regional Trends

North America

Market Size and Forecast, By Transplant Type, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Indication, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Application, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Country, 2016 - 2027, (US$ Million)

U.S.

Canada

Europe

Market Size and Forecast, By Transplant Type, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Indication, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Application, 2016 - 2027, (US$ Million)

Market Size and Forecast, By Country, 2016 - 2027, (US$ Million)

U.K.

Germany

Italy

France

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Insights on the Hematopoietic Stem Cell Transplantation Global Market to 2027 - Key Drivers, Restraints and Opportunities - Yahoo Finance

Whethers its the birth of a new space tourism industry or simply the ultimate game of one-upmanship it seems like a new billionaire blasts off every week.

So its worth keeping in mind that there are actually important things to study in space. In particular, theres vast potential for the kind of space-based manufacturing being studied by the University of Pittsburghs McGowan Institute for Regenerative Medicine, and the International Space Station National Laboratory, currently orbiting the Earth with scientists aboard.

The International Space Station Research and Development Conference 2021, which is taking place this week, includes a fireside chat with McGowan Institute Director William Wagner about the opportunities for biomanufacturing in space, particularly in the fields of tissue engineering and regenerative medicine. A preprint (not yet peer-reviewed) of their research was posted online this week.

So what is regenerative medicine, and why does it work well in space?

A definition that I like is that regenerative medicine involves harnessing the bodys ability to heal itself, says Wagner. That can include the use of stem cells or biomaterials and devices.

International Space Station. Photo courtesy of NASA.

Pittsburghs expertise in regenerative medicine can be traced back to Thomas Starzl.

Dr. Starzl made Pittsburgh ground zero for organ transplantation and revolutionized care for patients in end-stage organ failure, says Wagner. This built a group of experts in Pittsburgh interested in how to manage patients with failing organs. Transplant is an attractive option, but given that there are limited donors and there are delays in availability what about other approaches? This led to the development of the McGowan Institute, which initially focused on artificial organs (such as hearts), but broadened its mission to embrace regenerative medicine approaches.

The advantage of studying this in space is because of how microgravity in low Earth orbit favorably affects biology, including a higher reproduction rate of stem cells. The conference illuminated several opportunities for space-based biomanufacturing research and development.

Photo courtesy of the International Space Station Research and Development Conference.

Its a new space race, one that has vast implications for the future of medicine.

I personally believe that the future will find engineers and life scientists harnessing microgravity phenomena to develop medical products, says Wagner.

The McGowan Institute team in Pittsburgh has been working for two years to develop a roadmap for biomanufacturing in low Earth orbit.

The number of launches into orbit has increased dramatically over the past several years, costs have plummeted and private enterprise is making its own investments This capacity and cost reduction further opens up the ability for life science advancements, says Wagner.

International Space StationISSMcGowan Institute for Regenerative Medicinespaceuniversity of pittsburgh

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Pitt researchers partner with International Space Station on biomanufacturing - NEXTpittsburgh

GoodCell's FSA/HSA eligible product helps consumers improve healthcare outcomes & reduce medical costs

WALTHAM, Mass., Aug. 3, 2021 /PRNewswire/ -- GoodCell ("LifeVault Bio"), an FSA/HSA eligible member-based program offering genetic testing, health screenings and personal biobanking, added Navia Benefit Solutions, a national leader in consumer health insurance and benefits administration, to its growing affiliate network.

Navia plan participants now have access to GoodCell product and services which will help them to better understand their current health, track the progression & regression of major chronic diseases, understand future genetic predispositions, and bank their stem cells for potential future use.

"Our members are engaged in the decisions for their health and their families, and they want access to genetic information that is actionable, not just for their current lifestyle, but for the medical treatments that will be available as they age and even for their future generations," said Hilarie Aitken, Chief Executive Officer, Navia Benefit Solutions. "We chose to partner with GoodCell to give our members access to the most comprehensive genetic testing on the market."

"Innovative health administrators like Navia Benefit Solutions are realizing the value of empowering individuals with the information and tools to make decisions for the health of themselves and their families." said Trevor Perry, Founder and Chief Executive Officer, GoodCell. "With nearly 800 stem cell therapies in clinical trials and an anticipated 10-20 new FDA approved therapies a year by 2025*, GoodCell is the only solution available to consumers to bank their own stem cells for their future health."

To learn more about GoodCell and how to become a member, visit http://www.GoodCell.com. To inquire about our affiliate partnership program, visit http://www.goodcell.com/affiliates.

About GoodCell

GoodCell empowers individuals to live longer and healthier lives with a medically actionable service that identifies, tracks and helps mitigate health risks. Driven by mounting evidence in support of cellular therapy, the company offers the only holistic solution that taps genetic and biological information as well as banked stem cells for potential use in the future. Committed to shaping the future of personalized health as today's science becomes tomorrow's medicine, GoodCell is led by a founding team of science and technology innovators with a diverse set of medical research. Learn more at: http://www.goodcell.com.

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About Navia Benefits Solutions

Navia is a national, consumer-directed benefits provider serving 7,000+ employers across all 50 states. The company provides comprehensive health and compliance solutions to employers and consumers, and offers industry-leading customer service, communications, and technology. Founded in 1989, Navia began as Flex-Plan Services, and over the years has grown into one of the nation's premier benefit providers. Navia offers FSA, HSA, HRA, Commuter, Wellness, and COBRA administration. http://www.naviabenefits.com

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GoodCell Adds Navia Benefit Solutions to its Affiliate Partnership Network Expanding Targeted Reach for its Direct-to-Consumer Healthcare Services -...

Marcela Iglesias, 43, who lives in Los Angeles, US, has spent 60,000 on cosmetic procedures to look like the popular toy doll but claims she has never had plastic surgery

A mum who has shelled out thousands so she can look like Barbie has revealed she wants to make an army of clones.

Marcela Iglesias, 43, who lives in Los Angeles, US, has spent 60,000 on cosmetic procedures to look like the popular toy doll but claims she has never had plastic surgery.

The mum-of-one has recently learned of human cloning and claims that not only are scientists currently working on this but by cloning herself, she could potentially help save lives by donating organs.

Marcela stumbled across the topic through Professor Nakauchi from the University of Tokyo, who has been conducting research into growing organs based on a patients stem cells.

She believes that cloning is already happening but that the general public just doesn't know about it yet.

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And the model is determined to have her very own army of Barbies, so that she can donate her organs to those who need them.

"I want to be cloned and then see if we can use those organs for donation or for myself In the future," Marcela said.

"An army of clones to use for a good cause would be great. There are far more patients requiring organ transplants than organ donations.

"By donating my stem cells and some of my eggs, they can be used to create human organs in order to help people that need the transplant."

Marcela has a personal connection to the topic, having lost her father, who needed a kidney transplant.

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Since his death, she has had 30 million stem cells transplanted into her bloodstream from an umbilical cord.

She said: "If I had a kidney for my father, he would be alive today and the fact that I have 30 million more stem cells that were injected into my body means that I can help with that research.

"I started the research of stem cell therapy because of my fathers diseases and I have learned a lot about it.

"Unfortunately, my dad passed and he didnt want to go through the treatment, so I took the advantage of all the information and I decided that it was time for me to do it.

"After all the information I gathered, I decided that mesenchymal stem cells via IV therapy was the one for me, there are many other procedures, but this was the most convenient."

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She had the procedure done in the US for around $6,000 (USD) and according to Marcela, it was successful and she believes it will boost her immune system.

The model said: "The treatment went great; its like receiving IV fluid, I was there for an hour and a half and I felt very relaxed.

"The doctor showed me the three tubes where the 30 million stem cells arrived, they were frozen.

"The treatment is not cheap but I feel that people, in general, including myself, spend a lot of money on things that have no benefit in the long term, especially when it comes to health, so I decided to dig into my savings and give my body a great gift."

The model is currently trying to get in touch with Professor Nakauchi so that she can discuss making her clones.

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Mum who spent 60,000 turning into a real life Barbie wants 'army of clones' - The Mirror

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Medexus Pharmaceuticals and medacGmbHreceiveda Complete Response Letter (CRL) from the Food and Drug Administration (FDA) for its New Drug Application (NDA) for treosulfan. The drug was being submitted for use in combination with fludarabine as a preparation for allogeneic hematopoietic stem cell transplantation (allo-HSCT). The companies had a target action date of August 11.

A preparative regimen is given to the patient before stem cell transplantation with the goal of decreasing the tumor burden. And in the case of allogeneic transplantation, it allows for engraftment of the donor cells.

The CRL indicated that it couldn't be approved in its present form. It offered specific recommendations, including additional clinical and statistical data related to the primary and secondary endpoints of the pivotal Phase III trial. The companies say they are reviewing the CRL to decide on a course of action.

Ken d'Entremont, chief executive officer of Medexus, said, "Given the recent Health Canada approval, European Medicines Agency approval in 2019, as well as supporting data from more than 100 publications, we were all surprised by the FDA's response."

"That being said, Medexus and medac look forward to continuing to work with the FDA to address their requests in a timely manner, and we remain optimistic for a future, albeit delayed, approval of treosulfan in the United States, complete with Orphan Drug Designation."

He went on to say that the current standard of care "is not suitable for numerous at-risk groups, due to the high toxicity effects, and treosulfan has demonstrated excellent survival data among those groups."

Medexus, headquartered in Toronto, Ontario, Canada, and medac, based in Wedel, Germany,inkeda licensing deal on July 12 to commercialize treosulfan in Canada. It is being marketed in Canada under the name Trecondyv. Medexus will handle sales and marketing, while medac will handle manufacturing and supply.

The therapy has been distributed in Canada under the Special Access Program and recently received approval for commercialization from Health Canada for adults with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS) who are at increased risk for standard conditioning therapies. It was also approved for children older than one-year-old with AML or MDS.

At the time, Magnus Kuster, vice president of International Sales & Regions for medac, noted, "The treosulfan-based conditioning regimen stands out for its combination of being highly effective similar to the potency of myeloablative procedures while simultaneously exhibiting significantly reduced toxicity. We at medac are very proud of our first-in-class conditioning agent as it fully meets our company's goals of improving patients' lives and supporting healthcare professionals in the best possible way."

In response to the CRL, Meduxus's general manager of U.S. Operations, Michael Adelman, said, "We are disappointed with the immediate result, but are encouraged by an incredible amount of support from key opinion leaders and the medical community for use of treosulfan in the United States. With the extensive launch preparations we have taken to date, we are well positioned to meet the expected strong demand for treosulfan."

They will work with the FDA to address the issues in the CRL while ready to trigger their U.S. marketing plan when it gets approved.

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FDA Hands Out Surprising Rejection of Meduxus and Medac's Treosulfan - BioSpace