Archive for the ‘Bone Marrow Stem Cells’ Category

Whoever saves a single life is considered by the Talmud to have saved the whole world.

In August 2013, Jeffrey Altadonna, who was on a Birthright trip, was tested at a bone marrow testing drive at the Jerusalem Gate Hotel.

It was perfectly ordinary summer day when the 29-year-old accountant from Sherman Oaks, California received the fateful phone call.

A 77-year-old woman from Los Angeles was the perfect stranger that he was deemed to save.

Diane Gebel, a widow from Cyprus, California was diagnosed with Acute Myeloid Leukemia (AML). Her husband had passed away right before she was diagnosed with cancer.

For an entire year, the donor and recipient need to remain anonymous to each other, but last week, the time came for the two to finally meet.

The two were honored at the Los Angeles One Huge Night Gala event hosted by Gift of Life.

In a statement, Birthright Israel explained that the gala also celebrated the successful 15-year partnership of Birthright Israel and Gift of Life.

This partnership has so far resulted in 83,000 Birthright donors joining the registry, with 1,900 matches made between patients and Birthright donors, and 241 life-saving transplants to date.

Prior to the meeting, Altadonna recalled his Birthright trip explaining that it was really great to see that part of the world, to go to where its our given right to visit.

It had a profound effect on my friendships and cultural Jewish identity to see that Jewish people are one people, and we have each others backs, he said. It left me with the feeling that I had backing in anything that I wanted to do in my life. It really felt like a family.

He recalled that after being swabbed at the drive, he didnt really think too much of it because everyone did it.

I got the donation call 15 months ago, he explained, adding that he immediately decided to donate. I find it very bizarre, that everyone is telling me Its such a great thing that you are doing. For me, it wasnt an option to say yes or no, its just, Okay, lets do this, Im a match. Im surprised that more people dont donate.

After doing preliminary tests, he took the plunge and donated.

I had to do it early in the morning, it lasted 6-8 hours a marathon blood donation and it was finished,Altadonna continued. It didnt seem all that hard to me.

He made it clear that this opportunity to help only came about because of the Birthright Israel and Gift of Life collaboration.

It wasnt a mission of mine. I wouldnt have gone out of my way to get swabbed, so it only happened as a result of their collaboration, he said.

In an emotional meeting, the two finally met. Of the meeting, Gebel stressed that she is here because of my selfless and generous donor.

For me, it was easy, I just had an infusion, but for him it was hard, she said. It takes a very special person to do that, to actually give the gift of life.

Gebel said she had been waiting to meet him.

I didnt know he was such a good looking guy, Gebel joked. Im here because of him. I was not ready to die. I had too much to live for.

She stressed that she has changed because of my new life.

I take risks, I live my life fully, she explained. My motto has always been that I want to go through life with a Cosmopolitan in one hand and a travel book in the other. Because of my donor, I can do that, so I thank him from the bottom of my heart.

Altadonna called on others to also take the plunge by getting tested and donating.

I stand here for my recipients valor, for her victory. I ask you to do the same: sit and swab today, so someone can swim and live tomorrow, he said.

Birthrights International CEO Gidi Mark said he was proud of our participants who register as donors and the powerful impact of such a simple choice when they get the call that they are the perfect match for a perfect stranger.

It is a great honor that Birthright Israel is able to assist in this mission, he added.

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Perfect match: How Birthright alumni saved the lives of 100s of strangers - The Jerusalem Post

Proud auntie Paola Mayfield says her niece, who has Down syndrome, is in remission from the leukemia that she battled for two years.

Last year I went to Colombia when I was pregnant, I needed to see my sister and my niece, Mayfield wrote alongside a photo of niece standing on a hospital bed with her arm attached to some medical devices. My niece has Down Syndrome and had been battling leukemia for about 2 years. But TODAY we received the greatest news! She is finally cancer free!

Paola said that the journey has been extremely difficult. With tears in my eyes I feel full of joy and happiness because I know how hard it has been the past few years for my sister.

And she wanted others who are struggling and fighting for their lives to know that there is always hope: For those who battle everyday to have another day of life, stay strong and dont lose faith. Thank you [hearts].

Leukemia is the most common type of childhood cancer. The most frequent type of childhood leukemia is acute lymphoblastic leukemia (ALL). Three out every four cases of childhood leukemia are diagnosed as acute, meaning that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months.

Acute lymphoblastic leukemia is a rare cancer thatoccurs when the bone marrow makes too much of a type of white blood cell calledlymphocytes, according to the National Cancer Institute. Signs of childhood ALL include fever and bruising. The disease can be detected using tests that examine the blood and bone marrow. Over time, there has been a lot of improvement in treatments for childhood leukemia.

There are several different approaches to treating the disease, and the treatment plan will depend on the type of ALL. Chemotherapy, radiation, chemotherapy with a stem cell transplant, and targeted therapy are all considered standard treatment, according to the American Cancer Society.

The next most common type of childhood leukemia is called acute myeloid leukemia, which occurs when the bone marrow makes a large number of abnormal blood cells called myeloblasts. As these cells build up, they prevent the growth ofhealthy white blood cells, red blood cells, and platelets.

Learn more about SurvivorNet's rigorous medical review process.

Proud auntie Paola Mayfield says her niece, who has Down syndrome, is in remission from the leukemia that she battled for two years.

Last year I went to Colombia when I was pregnant, I needed to see my sister and my niece, Mayfield wrote alongside a photo of niece standing on a hospital bed with her arm attached to some medical devices. My niece has Down Syndrome and had been battling leukemia for about 2 years. But TODAY we received the greatest news! She is finally cancer free!

Paola said that the journey has been extremely difficult. With tears in my eyes I feel full of joy and happiness because I know how hard it has been the past few years for my sister.

And she wanted others who are struggling and fighting for their lives to know that there is always hope: For those who battle everyday to have another day of life, stay strong and dont lose faith. Thank you [hearts].

Leukemia is the most common type of childhood cancer. The most frequent type of childhood leukemia is acute lymphoblastic leukemia (ALL). Three out every four cases of childhood leukemia are diagnosed as acute, meaning that the leukemia can progress quickly, and if not treated, would probably be fatal within a few months.

Acute lymphoblastic leukemia is a rare cancer thatoccurs when the bone marrow makes too much of a type of white blood cell calledlymphocytes, according to the National Cancer Institute. Signs of childhood ALL include fever and bruising. The disease can be detected using tests that examine the blood and bone marrow. Over time, there has been a lot of improvement in treatments for childhood leukemia.

There are several different approaches to treating the disease, and the treatment plan will depend on the type of ALL. Chemotherapy, radiation, chemotherapy with a stem cell transplant, and targeted therapy are all considered standard treatment, according to the American Cancer Society.

The next most common type of childhood leukemia is called acute myeloid leukemia, which occurs when the bone marrow makes a large number of abnormal blood cells called myeloblasts. As these cells build up, they prevent the growth ofhealthy white blood cells, red blood cells, and platelets.

Learn more about SurvivorNet's rigorous medical review process.

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Tears And Joy This Toddler with Down Syndrome Who Was Battling Leukemia Is Finally Cancer Free - SurvivorNet

According to results of a study published in Blood, children and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) who had minimal residual disease (MRD) prior to treatment and received high-dose preconditioning chemotherapy were most likely to respond to a second-generation CD-19 chimeric antigen receptor-T cell (CAR-T) therapy.1

Although it has been estimated that 90% or more of pediatric patients witha diagnosis of ALL will respond to multi-agent chemotherapy, the prognosis forthose with relapsed/refractory disease remains poor. One CD19-directed CAR-Ttherapy, tisagenlecleucel, is approved by the US Food and Drug Administration inpatients up to age 25 years with B-cell precursor ALL who either have refractorydisease or have experienced a second or later relapse.2

This open label, nonrandomized, phase 1 study (Clinical Trial Identifier: NCT01860937), evaluated the toxicity, feasibility, and response of 19-28z CAR-T therapy, a second-generation CD19-directed CAR-T therapy involving T cells expressing a chimeric receptor composed of an anti-CD19 antibody binding site and intracellular domains from the T-cell coactivating receptors, CD28 and the CD3-zeta chain3 in children and young adults up to 25 years of age with very high-risk ALL.1 Inclusion criteria included at least 2 relapses, early bone marrow relapse following complete response (CR), intermediate/late CR with poor response to re-induction therapy, or those with refractory disease, or ineligibility for allogeneic hematopoietic stem cell transplantation (allo-HSCT) or additional chemotherapy.1

The age range of the 25 patients treated with 19-28z CAR-T therapy onstudy was 1 to 22.5 years, with a median age of 13.5 years. Preconditioningchemotherapy involved high-dose cyclophosphamide (15 patients) and low-dosecyclophosphamide (8 patients), with 3 patients in each subgroup also receivingfludarabine.1

Regarding the feasibility of this approach, the prespecified CAR-Tcell dose was achieved for all patients for whom the 19-28z CAR-T therapyprocedure was undertaken.1

With respect to treatment toxicity,approximately one-third of patients experienced a grade 3/4 adverse event,including cytokine release syndrome (CRS) and neurotoxicity in 16% and 28% ofpatients, respectively. With the exception of 1 patient with grade 4 CRS andneurotoxicity who died following refractory Stenotrophomonas septic shock,these adverse events were reversible.1

Of the 24 patients includedin the response analysis, 75% achieved either a CR or a CR with incompletecount recovery (CRi). In the subsets of patients receiving preconditioningchemotherapy with either high- or low-dose cyclophosphamide, the CR/CRi rateswere 94% and 38%, respectively. Furthermore, treatment response was influencedby disease burden as evidenced by the considerably higher CR/CRi rate inpatients with baseline minimal residual disease (ie, less than 5% bone marrowblasts; 93%) compared with morphological evidence of disease at baseline (5% orhigher bone marrow blasts; 50%).1

The CR/CRi rate for thesubset of patients with pretreatment MRD treated with high-dose cytarabine was100%.1

Consolidation allo-HSCT was performed in 83% (15) of the patientsresponding to CAR-T therapy, with a median time from CAR-T infusion toallo-HSCT of 57 days. At a median follow-up of 28.6 months for respondingpatients, over half of these patients (8) were alive and had no evidence ofdisease.1

In their concluding remarks, the study authorscommented that thisanalysis has allowed us to determine the toxicity profile, confirm feasibility,evaluate response of this approach, and provide a direct comparison of the sameCD19-specific CAR T cell product that was previously published[3] inadult patients for the same indication.

The authors went on to highlight the findingof a reversible toxicity profile in the patients within their study as well asthe impact of preconditioning chemotherapy dose intensity and minimal pretreatmentdisease burden on response.

They further noted that within this cohort,the long-term persistence of response is encouraging, and in our primarilytransplant-naive patient population, the ability to proceed to allo-HSCT hasdemonstrated a favorable overall survival, manageable toxicity, and limitedincidence of relapse.

References

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19-28z CAR-T Therapy in Children and Young Adults With Relapsed/Refractory ALL: Promising Early Results - Cancer Therapy Advisor

Hematopoietic stem cells (HSCs) are responsible for the constant replenishment of all blood cells throughout life. One of the major challenges in regenerative medicine is to produce tailor-made HSCs to replace the defective ones in patients suffering from blood related diseases. This would circumvent the shortage of donor HSCs available for the clinic. To achieve the controlled production of bona fide HSCs in vitro, in a dish, a better understanding is required of where, when and how HSCs are physiologically produced in vivo, in the living body. Researchers from the groups of Catherine Robin(Hubrecht Institute) and Thierry Jaffredo (UPMC, LBD IBPS, Paris) have found a previously unappreciated hematopoietic wave taking place in the bone marrow of late fetuses and young adults and producing HSCs from resident hemogenic endothelial cells of somite origin. This transient hematopoietic wave fills the gap between the completion of embryonic blood production and the beginning of adult bone marrow hematopoietic production in both chicken and mice.

Endothelial origin of hematopoietic stem cells

The constant production of short-lived blood cells, needed for proper oxygenation of tissues and protection against pathogens throughout life, relies on a small cohort of HSCs. The first HSCs derive from specialized endothelial cells, named hemogenic endothelial (HE) cells, via an endothelial to hematopoietic transition (EHT). EHT transiently occurs in the main arteries, such as the aorta, during the embryonic development of vertebrates. The pool of HSCs is then amplified before migrating to the bone marrow where HSCs will reside during adult life. Whether EHT occurs past the embryonic stage and in other organs, such as the bone marrow, was unknown until now.

Hemogenic endothelial cells in the bone marrow

To find out whether EHT occurs past the embryonic stage and in the bone marrow, the researchers used a combination of experimental embryology, genetic, transcriptomic and functional approaches on chicken and mouse models. By tracing bone marrow-forming endothelial cells through fluorescent genetic labelling and live imaging analyses, they found that the entire vascular network of the bone marrow derives from the somites. The somites are segments of the body that will progressively form important tissues of the organism as the embryo develops, including bones, muscles and skin. Unexpectedly, the researchers found that some somite-derived endothelial cells produce HSCs and multipotent progenitors in the late fetus and young adult bone marrow, through the same EHT process that was thus far only seen in the embryo. These cells are molecularly very similar to the cells undergoing EHT or recently emerged HSCs in the embryonic aorta, with a prominent Notch pathway, endothelial-specific genes and transcription factors involved in EHT. The results therefore demonstrate that HSCs are newly generated past embryonic stages, from hemogenic endothelial cells from somitic origin and via EHT, the same mechanism that occurs in the embryo.

A new wave of blood cell production

The yolk sac of the embryo produces two partially overlapping waves of hematopoiesis. The first (primitive) wave gives rise to hematopoietic cells that last only during embryonic development. The second (definitive) wave produces various progenitors that migrate to the fetal liver to produce the immediately needed blood cells. These progenitors are sufficient for the embryo to survive until birth, when the aorta-derived HSC-dependent wave will take over. The transient hematopoietic production discovered in the present study fills the gap between the end of the yolk sac hematopoiesis and the bone marrow HSC-dependent production of blood cells. Indeed, the pool of HSCs that expanded in the fetal liver starts to colonize the bone marrow only just before birth. HSCs are present in very low numbers and time is most likely required before they find their final adult-type niches and start to differentiate and proliferate into more committed progenitors and mature blood cells. The transient hematopoietic wave that the researchers describe in late fetal and young adult stages might also prepare the bone marrow niches for the HSCs coming from the fetal liver.

Stem cell therapies

Defects in HSCs lead to various blood-related disorders and cancers that are partly treated by HSC transplantations. The controlled production of bona fide HSCs from pluripotent precursors remains very difficult to achieve in vitro, in a petri dish, and therefore requires a better understanding of the HSC production as it occurs physiologically in vivo, in the living body. Identifying all steps of hematopoietic production and the molecular events controlling this process is of fundamental interest and should help to devise innovative stem cell therapies for hematopoietic disorders in the future.

Reference:Yvernogeau, L., Gautier, R., Petit, L., Khoury, H., Relaix, F., Ribes, V., Jaffredo, T. (2019). In vivo generation of haematopoietic stem/progenitor cells from bone marrow-derived haemogenic endothelium. Nature Cell Biology. https://doi.org/10.1038/s41556-019-0410-6

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Transient Wave of Hematopoietic Stem Cell Production in Late Fetuses and Young Adults - Technology Networks

Doris A. Taylors so-called replacement ghost heart suggests something otherworldly, but the eerie-looking form is far from an apparition. Its an innovative approach to organ transplantation that has inspired many in the medical community and at least one artist.

The Texas researchers process piggybacks on natures sophisticated design. Together she and a team of researchers strip cells off human and animal cadaver hearts with a soapy solution, leaving ghostly white protein shells that retain the form of the organ. They inject them with a patients blood or bone-marrow stem cells, and the ghost hearts act as scaffolding on which the newly introduced cells can slowly transform into a beating muscle.

What we said was, Wouldnt it be really cool if we could wash the sick cells out and put the healthy cells back in? said Taylor, director of Regenerative Medicine Research and director of the Center for Cell and Organ Biotechnology at the Texas Heart Institute, during a recent talk at the Radcliffe Institute for Advanced Study.

The hope is that one day these regenerated hearts will resolve the most challenging issues transplant patients currently face: the lack of a permanent artificial replacement, concerns about rejection, and the shortage of viable donor hearts.

Taylors efforts are driving what could become a revolution in organ transplants, and they have sparked the creativity of transdisciplinary artist Dario Robleto, whose latest work, on view at the Johnson-Kulukundis Family Gallery in Radcliffes Byerly Hall, recreates in images and sounds the original pulse wave of the heart first captured in visual form by scientists in the 1900s. Robleto and Taylor, longtime friends and Texas residents, explored those connections during Mondays Radcliffe discussion, which was moderated by Jennifer Roberts, Elizabeth Cary Agassiz Professor of the Humanities.

Robletos exhibit, Unknown and Solitary Seas, touches on the overlap between the medical mysteries and workings of the vascular pump, and the metaphor for the heart as the emotional center of the soul. It includes a video installation that features recreated sounds of a beating heart from the 19th century, reconstructed images of how the earliest pulse waves first appeared on the page, and a series of heart waveform sculptures in brass-plated stainless steel.

Roberts said that with his work, Robleto acknowledges the pulse waves promise, their profundity, their scientific value, but he also reclaims some of their ambiguity and asks us to wonder whether we can or should accept that these waveforms have escaped the realms of art, culture, and emotional communication.

Taylor similarly views her work as a blend of the scientific and the human. It transcends complicated, complex science, she says, in that her ghost hearts require a kind of passion, commitment, care, attention, and nurturing similar to whats required by a small child. Its really about building hearts at the emotional, mental, spiritual, and physical level that I think is going to get them to work, she said.

For Robleto, big ideas, like the creation of a new human heart, require multiple perspectives.

The artist called Taylors work one of the most fascinating and definitely one of the most emotional things Ive ever seen. As an object, he added, the ghost heart is stunningly beautiful but it also raises questions about the self, identity, emotion, the notions of form and where memory is truly held, questions he thinks artists can help address. He cited two of the nations earliest heart transplants, after which the patients wives asked their husbands, who had received donor hearts, if they still loved them.

Taylors work, Robleto said, is right at the edge of identity and materiality and so when the day comes when someone says the first ghost heart transplant I think we will have a similar moment where perhaps we will be forced to re-evaluate what we ask from our heart metaphor.

Dario Robletos Unknown and Solitary Seas is on view in Byerly Halls Johnson-Kulukundis Family Gallery through Jan. 18, 2020.

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An artist and a transplant researcher discuss the heart - Harvard Gazette

Scientists at Harvard-affiliated Massachusetts General Hospital (MGH) have identified a previously unknown biological pathway that promotes chronic inflammation and may help explain why sedentary people have an increased risk for heart disease and strokes.

In a study to be published in the November issue ofNature Medicine, MGH scientists and colleagues at several other institutions found that regular exercise blocks this pathway. This discovery could aid the development of new therapies to prevent cardiovascular disease.

Regular exercise protects the cardiovascular system by reducing risk factors such as cholesterol and blood pressure. But we believe there are certain risk factors for cardiovascular disease that are not fully understood, said Matthias Nahrendorf of the Center for Systems Biology at MGH. In particular, Nahrendorf and his team wanted to better understand the role of chronic inflammation, which contributes to the formation of artery-clogging blockages called plaques.

Nahrendorf and colleagues examined how physical activity affects the activity of bone marrow, specifically hematopoietic stem and progenitor cells (HSPCs). HSPCs can turn into any type of blood cell, including white blood cells called leukocytes, which promote inflammation. The body needs leukocytes to defend against infection and remove foreign bodies.

When these [white blood] cells become overzealous, they start inflammation in places where they shouldnt, including the walls of arteries.

Matthias Nahrendorf

But when these cells become overzealous, they start inflammation in places where they shouldnt, including the walls of arteries, said Nahrendorf.

Nahrendorf and his colleagues studied a group of laboratory mice that were housed in cages with treadmills. Some of the mice ran as much as six miles a night on the spinning wheels. Mice in a second group were housed in cages without treadmills. After six weeks, the running mice had significantly reduced HSPC activity and lower levels of inflammatory leukocytes than the mice that simply sat around their cages all day.

Nahrendorf explains that exercising caused the mice to produce less leptin, a hormone made by fat tissue that helps control appetite, but also signaled HSPCs to become more active and increase production of leukocytes. In two large studies, the team detected high levels of leptin and leukocytes in sedentary humans who have cardiovascular disease linked to chronic inflammation.

This study identifies a new molecular connection between exercise and inflammation that takes place in the bone marrow and highlights a previously unappreciated role of leptin in exercise-mediated cardiovascular protection, said Michelle Olive, program officer at the National Heart, Lung, and Blood Institute Division of Cardiovascular Sciences. This work adds a new piece to the puzzle of how sedentary lifestyles affect cardiovascular health and underscores the importance of following physical-activity guidelines.

Reassuringly, the study found that lowering leukocyte levels by exercising didnt make the running mice vulnerable to infection. This study underscores the importance of regular physical activity, but further focus on how exercise dampens inflammation could lead to novel strategies for preventing heart attacks and strokes. We hope this research will give rise to new therapeutics that approach cardiovascular disease from a completely new angle, said Nahrendorf.

The primary authors of theNature Medicinepaper are Nahrendorf, who is also a professor of radiology at Harvard Medical School; Vanessa Frodermann, a former postdoctoral fellow at MGH who is now a senior scientist at Novo Nordisk; David Rohde, a research fellow in the Department of Radiology at MGH; and Filip K. Swirski, an investigator in the Department of Radiology at MGH.

The work was funded bygrantsHL142494 andHL139598from the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

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Exercise found to block chronic inflammation in mice - Harvard Gazette

When Nicky Turkoz (aboveleft) was diagnosed with leukaemia, her only hope was an anonymous stem-cell donation. That donor was Annette Hamson (above right) and the two, once strangers, now share an unbreakable bond

I was wrestling the Christmas decorations down from the loft when I got the call. I can remember hearing the landline ring and telling my daughter, Meltem, who was steadying the ladder, to leave it. They can call back if its urgent, I said. My mobile rang and we left that too, but the landline went again. I better get it, Mum, said Meltem. Someone clearly wants to talk to you. She answered it as I was hauling the last of the decorations down. Its the surgery, she said, passing the phone to me.

That morning Id had a blood test. Id been feeling strange for months and had finally made an appointment to see the GP. I told her I was feeling lethargic and depressed, which was very unlike me, but Id put it down to ageing and a spot of empty-nest syndrome, as my youngest daughter Zeynep had just left home to work abroad. Odd things, such as a recurring gum infection, were making me wonder if something else was going on. My GP seemed unconcerned but sent me for blood tests anyway. A few hours later she was telling me to pack an overnight bag and get to hospital. Your results show youre very anaemic, she said. Theyll need to do some more tests. You might be there a while.

She never used the word cancer. Looking back, I think she knew then but was sugar- coating it for me. We left the decorations.

Three hours later, after more prodding and poking, a consultant told me I had acute myeloid leukaemia, a blood cancer. It seems unbelievable now but I can remember thinking, Well, thank goodness theyve found something wrong with me, I knew I wasnt feeling great. I had no idea, of course, what was coming down the track.

It was a Wednesday evening and I was told they would start my chemotherapy on the Friday, but Id need to stay in hospital in the meantime. So while I was being installed on a ward, my poor daughter was handed a leaflet about myeloid leukaemia and drove home to our undecorated tree.

The first round of chemo lasted 10 days, which meant I had to miss Meltems 23rd birthday on 21 December. Zeynep left Gran Canaria, where she had just started working as a holiday rep, and flew home to be with us. It has always been just the three of us, ever since they were tiny. Im a very can-do sort of person and have always just got on with life whatever it has thrown at us. I couldnt bear it when my girls suddenly had to look after me. It all just seemed so unfair. I suppose everyone feels like that though.

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'I've become best friends with the woman who saved my life' - Telegraph.co.uk

Arizona has been called "the wild, wild west" of regenerative medicine.

The Valley is one of the most popular places in the country for stem cell clinics. The new and controversial therapy is being marketed and practiced all over Phoenix and Scottsdale.

The less invasive procedure promises to heal pain, nearly anywhere in their body. It is advertised as effective, safe, and ethical, but outside experts and industry insiders say consumers need to do their research to avoid being exploited, and potentially spending thousands in cash on a worthless injection.

"IT HAS GREAT POTENTIAL"

The world of regenerative medicine is still being explored and developed.

"It actually gives you really good results," explained Dr. Matthew Hernandez, a naturopathic physician with Ethos.

"There's a lot of hope and promise, generally around the prospects for stem cells," said ASU Professor Emma Frow.

"Were still in the developmental stage. Stem cell therapy has been around for less than ten years. Thats new in medicine," said Dr. Steven Sorr, a naturopathic physician who runs Source of Health in Scottsdale.

"It encourages your own body to heal itself," said Janet McConnell, a 63-year-old bodybuilder who "had cartilage damage several years ago."

Instead of a surgery that would have derailed her competition training for months, she opted for injections.

"Three years ago, instead of the surgery, I had a PRP treatment," said McConnell. "It was very effective."

Years later, she returned to Dr. Hernandez for another round.

For most, Stem Cell and Platelet Rich Plasma (PRP) therapy is a mystery. "It's kind of controversial and experimental," said Matthew Riddle, Director of Sales for Celling Biosciences.

The treatments concentrate platelets or stem cells, usually from the patient's own blood. Experts say it is important to always ask the doctor or provider where the "growth factors" are coming from, because in order to ensure they are alive they should be coming from the patient's own blood, fat, or bone marrow. Otherwise, patients can receive "dead" stem cells, which are not nearly as effective.

"We are very adamant to use the patient's own cells," said Riddle, who uses a centrifuge to separate out the blood, saline and growth factors that will be re-injected. "When we inject that into an area, we are telling your body to go heal that spot," said Dr. Hernandez.

"Stem cell treatment is really about trying to take the stem cells out of your body and...inject them back into another part of your body, in order to try and heal whatever part of the body is suffering," said Professor Frow.

"IT'S THE NEW WAVE"

According to researchers, Scottsdale and Phoenix are two of the seven "hot spot" cities in the country.

Arizona State University professors Emma Frow and Dave Brafman spent years studying the industry , and mapping out dozens of clinics in the Valley. They believe there are many more, as some intentionally practice under the radar. "I don't believe right now that there is enough evidence to suggest that they work," said Professor Frow.

"They are unregulated, unproven and for-profit," added Professor Brafman.

The profits are plentiful. "There's cash involved, so this isn't covered by insurance," said Dr. Hernandez.

"PREYING ON PEOPLE'S PAIN"

The thousands in cash is one of many reasons the burgeoning industry is ripe for exploitation.

"The other piece too, it is it is new and upcoming," said Dr. Hernandez.

Many potential patients do not know the first thing about the procedure they are being sold, and doctors say many fall for sales tactics that are practiced at traveling seminars.

"They are preying on people's pain," said Dr. Sorr. "I think its really unethical and it upsets me."

Dr. Sorr believes the seminars are "a scam" that specifically targets an elderly clientele.

"They wine you and dine you. They go through a little dinner presentation and it is not the doctor, it's a marketing agency," he said.

The doctor told ABC15 he has had clients who have been duped, even after he told them they were not ideal candidates for stem cell or PRP therapy.

"It really broke my heart that he spent thousands upon thousands of dollars for something that was worthless.

"I don't agree with how they are done," said Dr. Hernandez. "They inject people and they get money. That's not practicing medicine, that is selling."

Both naturopathic physicians told ABC15 that some patients do not need the treatment, or will get subpar results from the injections. They say it is well known in the industry that some practices will continue to sell in order to reap the thousands in cash.

"ALL OF IT FALLS ON THE PATIENT"

Right now, there is little regulation or oversight of the industry in Arizona.

"Really all of it the falls on the patient, with very little recourse if things go wrong," said Dr. Emma Frow.

During the course of our investigation, ABC15 discovered the Arizona Medical Board and County Health Department do not take complaints or oversee the people performing injections. The federal government has also been slow to implement widespread regulation.

"The FDA has their hands tied," said Dr. Sorr. "There are too many people out there that are doing this that havent had the proper training, they dont have the right experience, the right tools and all that."

There are some larger regulations in Arizona, governing who can handle a needle and perform injections.

Unlike other industries though, including massage therapy, there is no board that checks on licensing or investigates complaints involving botched procedures or alleged fraud.

"The state medical boards, need to become a little bit more involved in sort of identifying, or responding to claims," said Professor Brafman.

"I don't think it would hurt to have it, for sure. At the end of the day it's about protecting the public," said Dr. Hernandez.

For thousands of Arizonans, like Janet McConnell, regenerative medicine has helped heal chronic pain. Before spending thousands thousands though, do your research. "Always get a second opinion," said Dr. Sorr.

"I think this is really a case of buyer beware, or consumer beware," said Professor Frow.

If you are planning on undergoing a stem cell or PRP treatment, click here for questions experts say you should always ask ahead of time.

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Arizona the "wild west" of stem cell therapy; experts say promising therapy ripe for exploitation - ABC15 Arizona

A potentially life shattering diagnosis turned into motivation to help others. A Peoria woman is hosting a Be the Match event at the Greater Peoria YMCA on Wednesday.

While many might say Why me? when diagnosed with a rare bone marrow cancer, Marsha Krone continued to fight on, choosing to make the most of the life she has been given.

A mother of three, grandmother of two, lover of sewing, and teacher for over 30 years, Marsha Krone had a lot to smile about. Until an existing blood disorder threatened to steal her joy.

I went to the Mayo Clinic and they did a bone marrow biopsy and they told me that yes, you have progressed to Myelofibrosis. And so what that means is my bone marrow is essentially turning hard. said Marsha Krone, diagnosed in 2016

Krone needs a stem cell transplant. Her sisters were not a match.Despite millions of donors being listed on the registry she is still waiting for the perfect stranger. However of the ten markers needed to match, two are extremely rare, making it difficult, but not impossible to find one.

Her hope comes from faith.

I cant write my story because he writes my story and I just can choose how Im going to live it. So I choose joy. said Krone

As an ambassador for Be the Match she has put on numerous events, including the one coming up at the Greater Peoria YMCA where shes been a member for many years.

We talked about how could we help in this way and she said well lets hold a match event and try to get as many people engaged as we possibly can. Shes not doing this for herself, shes doing this for everyone out there that needs to find a match said President and CEO of the Greater Peoria YMCA, Andy Thornton

The process is simple. You swab your cheek for DNA, provide your contact information, and join the registry where you could match anyone in the world for stem cells and bone marrow.

If someone else was matched from a drive we promoted, that would be really exciting for me to know that I was the go between to helping someones life be saved. said Krone.

The event takes place Wednesday 11/6 from 3 7 P.M. at The Greater Peoria YMCA

Link:
Using her diagnosis to help others, Peoria woman to host 'Be the Match' event - week.com

CAMBRIDGE, Mass. & OSAKA, Japan--(BUSINESS WIRE)--Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) ("Takeda") today announced the randomized, Phase 3 TOURMALINE-MM4 study met its primary endpoint of progression free survival (PFS). The trial evaluated the effect of single-agent oral NINLARO (ixazomib) as a first line maintenance therapy versus placebo in adult patients diagnosed with multiple myeloma not treated with stem cell transplantation. TOURMALINE-MM4 is the first industry sponsored Phase 3 trial to explore the concept of switch maintenance, the use of medicines not included in initial induction therapy, in this setting. NINLARO is currently not approved for this specific use.

We are very encouraged by the results of the TOURMALINE-MM4 trial and continue our forward momentum in developing maintenance options for multiple myeloma patients. Importantly, this is the third positive Phase 3 readout from the TOURMALINE clinical trial program, said Phil Rowlands, Ph.D., Head, Oncology Therapeutic Area Unit, Takeda. We remain committed to bringing this convenient and well-tolerated treatment option to patients.

The safety profile of NINLARO in the maintenance setting was consistent with previously reported results of single-agent NINLARO use, and there were no new safety signals identified in TOURMALINE-MM4.

Full data results will be submitted for presentation at an upcoming medical meeting.

About the TOURMALINE-MM4 Trial

TOURMALINE-MM4 is a randomized, placebo-controlled, double-blind Phase 3 study of 706 patients, designed to determine the effect of single-agent oral NINLAROTM (ixazomib) maintenance therapy on progression-free survival (PFS), compared to placebo, in adult patients newly diagnosed with multiple myeloma not treated with stem cell transplantation, who have completed 6-12 months of initial therapy and achieved a partial response or better. For additional information, please visit https://clinicaltrials.gov/ct2/show/NCT02312258.

About Multiple Myeloma

Multiple myeloma is a life-threatening rare blood cancer that arises from the plasma cells, a type of white blood cell that is made in the bone marrow. These plasma cells become abnormal, multiply and release a type of antibody known as a paraprotein, which causes symptoms of the disease, including bone pain, frequent or recurring infections and fatigue, a symptom of anemia. These malignant plasma cells have the potential to affect many bones in the body and can cause a number of serious health problems affecting the bones, immune system, kidneys and red blood cell count. The typical multiple myeloma disease course includes periods of symptomatic myeloma followed by periods of remission. Nearly 230,000 people around the world live with multiple myeloma, with approximately 114,000 new cases diagnosed globally each year.

About NINLAROTM (ixazomib) capsules

NINLARO (ixazomib) is an oral proteasome inhibitor which is being studied across the continuum of multiple myeloma treatment settings. NINLARO was first approved by the U.S. Food and Drug Administration (FDA) in November 2015 and is indicated in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy. NINLARO is currently approved in more than 60 countries, including the United States, Japan and in the European Union, with more than 10 regulatory filings currently under review. It was the first oral proteasome inhibitor to enter Phase 3 clinical trials and to receive approval.

The comprehensive ixazomib clinical development program, TOURMALINE, includes several ongoing pivotal trials, which together are investigating major multiple myeloma patient populations:

In addition to the TOURMALINE program, ixazomib is being evaluated in multiple therapeutic combinations for various patient populations in investigator initiated studies globally.

NINLAROTM (ixazomib) capsules: Global Important Safety Information

SPECIAL WARNINGS AND PRECAUTIONSThrombocytopenia has been reported with NINLARO (28% vs. 14% in the NINLARO and placebo regimens, respectively) with platelet nadirs typically occurring between Days 14-21 of each 28-day cycle and recovery to baseline by the start of the next cycle. It did not result in an increase in hemorrhagic events or platelet transfusions. Monitor platelet counts at least monthly during treatment with NINLARO and consider more frequent monitoring during the first three cycles. Manage with dose modifications and platelet transfusions as per standard medical guidelines.

Gastrointestinal toxicities have been reported in the NINLARO and placebo regimens respectively, such as diarrhea (42% vs. 36%), constipation (34% vs. 25%), nausea (26% vs. 21%), and vomiting (22% vs. 11%), occasionally requiring use of antiemetic and anti-diarrheal medications, and supportive care.

Peripheral neuropathy was reported with NINLARO (28% vs. 21% in the NINLARO and placebo regimens, respectively). The most commonly reported reaction was peripheral sensory neuropathy (19% and 14% in the NINLARO and placebo regimens, respectively). Peripheral motor neuropathy was not commonly reported in either regimen (< 1%). Monitor patients for symptoms of peripheral neuropathy and adjust dosing as needed.

Peripheral edema was reported with NINLARO (25% vs. 18% in the NINLARO and placebo regimens, respectively). Evaluate patients for underlying causes and provide supportive care, as necessary. Adjust the dose of dexamethasone per its prescribing information or the dose of NINLARO for severe symptoms.

Cutaneous reactions occurred in 19% of patients in the NINLARO regimen compared to 11% of patients in the placebo regimen. The most common type of rash reported in both regimens was maculo-papular and macular rash. Manage rash with supportive care, dose modification or discontinuation.

Hepatotoxicity, drug-induced liver injury, hepatocellular injury, hepatic steatosis, and hepatitis cholestatic have been uncommonly reported with NINLARO. Monitor hepatic enzymes regularly and adjust dose for Grade 3 or 4 symptoms.

Pregnancy- NINLARO can cause fetal harm. Advise male and female patients of reproductive potential to use contraceptive measures during treatment and for an additional 90 days after the final dose of NINLARO. Women of childbearing potential should avoid becoming pregnant while taking NINLARO due to potential hazard to the fetus. Women using hormonal contraceptives should use an additional barrier method of contraception.

Lactation- It is not known whether NINLARO or its metabolites are excreted in human milk. There could be potential adverse events in nursing infants and therefore breastfeeding should be discontinued.

SPECIAL PATIENT POPULATIONSHepatic Impairment: Reduce the NINLARO starting dose to 3 mg in patients with moderate or severe hepatic impairment.

Renal Impairment: Reduce the NINLARO starting dose to 3 mg in patients with severe renal impairment or end-stage renal disease (ESRD) requiring dialysis. NINLARO is not dialyzable and, therefore, can be administered without regard to the timing of dialysis.

DRUG INTERACTIONSCo-administration of strong CYP3A inducers with NINLARO is not recommended.

ADVERSE REACTIONSThe most frequently reported adverse reactions ( 20%) in the NINLARO regimen, and greater than in the placebo regimen, were diarrhea (42% vs. 36%), constipation (34% vs. 25%), thrombocytopenia (28% vs. 14%), peripheral neuropathy (28% vs. 21%), nausea (26% vs. 21%), peripheral edema (25% vs. 18%), vomiting (22% vs. 11%), and back pain (21% vs. 16%). Serious adverse reactions reported in 2% of patients included thrombocytopenia (2%) and diarrhea (2%). For each adverse reaction, one or more of the three drugs was discontinued in 1% of patients in the NINLARO regimen.

For European Union Summary of Product Characteristics: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/003844/WC500217620.pdf For US Prescribing Information: https://www.ninlarohcp.com/pdf/prescribing-information.pdf For Canada Product Monograph: http://www.takedacanada.com/ninlaropm

About Takeda Pharmaceutical Company LimitedTakeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Rare Diseases and Neuroscience. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. For more information, visit https://www.takeda.com.

Important NoticeFor the purposes of this notice, press release means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (Takeda) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, Takeda is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words we, us and our are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking StatementsThis press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takedas future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as targets, plans, believes, hopes, continues, expects, aims, intends, ensures, will, may, should, would, could anticipates, estimates, projects or similar expressions or the negative thereof. Forward-looking statements in this document are based on Takedas estimates and assumptions only as of the date hereof. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: the economic circumstances surrounding Takedas global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the timing and impact of post-merger integration efforts with acquired companies; and the ability to divest assets that are not core to Takedas operations and the timing of any such divestment(s), any of which may cause Takedas actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takedas results, performance, achievements, or financial position, see Item 3. Key InformationD. Risk Factors in Takedas most recent Annual Report on Form 20-F and Takedas other reports filed with the U.S. Securities and Exchange Commission, available on Takedas website at: https://www.takeda.com/investors/reports/sec-filings/ or at http://www.sec.gov. Future results, performance, achievements or financial position of Takeda could differ materially from those expressed in or implied by the forward-looking statements. Persons receiving this press release should not rely unduly on any forward-looking statements. Takeda undertakes no obligation to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results of Takeda in this press release may not be indicative of, and are not an estimate, forecast or projection of Takedas future results.

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Phase 3 Trial of NINLAROTM (ixazomib) as First Line Maintenance Therapy Met Primary Endpoint in Multiple Myeloma Patients not treated with Stem Cell...

NEW YORK, Nov. 05, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Company will hold a conference call to update shareholders on financial results for the third quarter ended September 30, 2019, and provide a corporate update, at 8:00 a.m., Eastern Standard Time, on Thursday, November 14, 2019.

BrainStorms President & CEO, Chaim Lebovits, will present a corporate update, after which, participant questions will be answered. Joining Mr. Lebovits to answer investment community questions will be Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer, and Preetam Shah, PhD, Chief Financial Officer.

Participants are encouraged to submit their questions prior to the call by sending them to: q@brainstorm-cell.com; Questions should be submitted by 5:00 p.m., Eastern Standard Time, Tuesday, November 12.

The investment community may participate in the conference call by dialing the following numbers:

Those interested in listening to the conference call live via the internet may do so by visiting the Investors & Media page of BrainStorms website at http://www.ir.brainstorm-cell.com and clicking on the conference call link.

A webcast replay of the conference call will be available for 30 days on the Investors & Media page of BrainStorms website:

About NurOwnNurOwn (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 recently 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 U.S. sites 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. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual 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, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://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.

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BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Comprehensive Corporate Update - Yahoo Finance

The Phase 3 clinical trial testing BrainStorm Cell Therapeutics cell therapy candidateNurOwn inamyotrophic lateral sclerosis (ALS) patients is continuing as planned after a second safety assessment by the trials independent Data Safety Monitoring Board (DSMB) found no reasons to stop, the company announced.

The DSMBs recommendation comes after a pre-specified interim analysis of the first 106 ALS patients treated repeatedly with NurOwn in this randomized, placebo-controlled clinical trial.

After reviewing all of the safety data as of September 30th, the DSMB has recommended the study continue without any changes in the protocol. We did not identify any significant safety concerns, Carlayne Jackson, MD, a professor of Neurology and Otolaryngology UT Health San Antonioand the DSMB chairperson, said in a press release.

DSMBs consist of research experts who monitor the progress of a clinical trial and review safety and efficacy data while the study is ongoing. This panel can recommend that a trial be stopped early because of safety concerns or evidence a therapy is not working as intended, or if the trials main goals have already been reached.

NurOwn consists of mesenchymal stem cells (MSCs; stems cells able to generate various cell types) collected from a patients bone marrow. These MSCs are expanded and matured into a specific cell type called MSC-NTF by growing them under conditions that induce them to secrete high levels of neurotrophic factors (NTFs) that support the growth, survival, and maturation of nerve cells.

MSC-NTF cells also deliver immune system regulating cytokines, small proteins important in cell signaling or messaging, to sites of damage, BrainStorm reports. It is thought this will help to slow or stabilize disease progression.

The double-blind Phase 3 trial (NCT03280056),fully enrolledat its six U.S. sites, is investigating use of NurOwn in 200 ALS patients whose symptoms became evident within two years of the studys start. Patients are randomized 1:1 to either NurOwn or placebo, given via intrathecal (spinal canal) injection every two months.

The studysprimary measures of safety and efficacy are being determined using the ALS functional rating scale score (ALSFRS-R; a score of abilities like swallowing, speech, handwriting, walking, etc.) in patients after 28 weeks of treatment compared to placebo.

A secondary goal is assessing how biomarkers, such as cell-secreted neurothrophic factors, inflammatory agents, andcytokines, change in the blood and cerebrospinal fluid (the liquid surrounding the brain and spinal cord) after treatment with NurOwn.

BrainStorm is expecting to have topline trial data by the end of 2020, which will potentially support the submission of a Biologics License Application (BLA) to theU.S. Food and Drug Administration requesting approval.

We are very pleased with the DSMB recommendation that the Phase 3 clinical trial continue without any protocol modification. This represents an important clinical trial advancement for BrainStorm and for the development of NurOwn as an innovative cellular therapy approach for ALS patients, added Ralph Kern MD, BrainStorms chief operating officer and chief medical officer.

This clinical trial is being funded by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989), and other types of investment.

NurOwn was given orphan drug status by both theFDA and the European Medicines Agency (EMA) as apotential ALS treatment.

Iqra holds a MSc in Cellular and Molecular Medicine from the University of Ottawa in Ottawa, Canada. She also holds a BSc in Life Sciences from Queens University in Kingston, Canada. Currently, she is completing a PhD in Laboratory Medicine and Pathobiology from the University of Toronto in Toronto, Canada. Her research has ranged from across various disease areas including Alzheimers disease, myelodysplastic syndrome, bleeding disorders and rare pediatric brain tumors.

Total Posts: 5

Ins Martins holds a BSc in Cell and Molecular Biology from Universidade Nova de Lisboa and is currently finishing her PhD in Biomedical Sciences at Universidade de Lisboa. Her work has been focused on blood vessels and their role in both hematopoiesis and cancer development.

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Phase 3 Trial of ALS Cell Therapy, NurOwn, Gets Thumbs Up in Safety Review by Monitoring Board - ALS News Today

Mr. McManushas over 20 years of leadership experience in the biopharmaceutical industry that includes leading the transformation of several biotech companies through strategic partnerships and execution of value-based strategies.Prior to joining PTx, John was the CEO of Aeolus Pharmaceuticals, where he managed a $100+ million BARDA contract for the advanced development of a radiation medical countermeasure.Before joining Aeolus, John served in strategic and financial roles at Spectrum Pharmaceuticals where he focused on oncology and NeoTherapeutics where he focused on Alzheimer's, Parkinson's, ALS and spinal cord injury.John holds a B.S. in International Finance and Business Economics from the University of Southern California.

"We are very pleased to welcome John to our team to lead our business development activities," said Bob Mulroy, PTx's Chief Executive Officer. "His breadth of experience as a biotech executive and business leader will be tremendous assets for our team as we continue to build and grow the opportunity for Leukine to help patients in need. We also will benefit from John's extensive experience working with the U.S. government to advance our ability to serve as a partner for a wide range of important government programs."

"I am excited to join the PTx team in strengthening and expanding the Leukine franchise and identifying additional products that would benefit from the Company's development and clinical expertise," said Mr. McManus. "Leukine is a critical drug in the treatment of adults and children facing life-threatening diseases and a key medical countermeasure for improving survival after lethal levels of radiation exposure.I am especially excited about working to support the development of Leukine in new indications like melanoma and diseases of the central nervous system like Alzheimer's and Parkinson's and expanding its use as a medical countermeasure through development partnerships with third parties including the U.S. government.I see tremendous potential for Leukine to help patients across a number of difficult-to-treat diseases."

PTx acquired the global rights to develop, manufacture, and commercialize Leukine in 2018.

Leukine is a multi-lineage immune-stimulant that has been demonstrated to promote growth and activation of monocytes, macrophages, neutrophils and dendritic cells.It is the only FDA-approved recombinant human granulocyte-macrophage colony stimulating factor (GM-CSF). It is currently indicated for the treatment of AML in older adults to reduce the incidence of severe and life-threatening infections resulting in death; use in the treatment of allogeneic bone marrow transplants to reduce the incidence of bacteremia and other culture positive infections and shorten the median duration of hospitalization; to prolong the survival of patients who are experiencing bone marrow transplant failure or delay; and to increase survival in adult and pediatric patients from birth to 17 years of age acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [H-ARS]).

About Leukine(sargramostim)

Leukine is a yeast-derived recombinant humanized granulocyte-macrophage colony stimulating factor (rhuGM-CSF) and the only FDA approved GM-CSF.GM-CSF is an important leukocyte growth factor known to play a key role in hematopoiesis, effecting the growth and maturation of multiple cell lineages as well as the functional activities of these cells in antigen presentation and cell mediated immunity3.

Important Safety Information for LEUKINE (sargramostim)

Contraindications

Warnings and Precautions

Adverse Reactions

Adverse events occurring in >10% of patients receiving LEUKINE in controlled clinical trials and reported in a higher frequency than placebo are:

Please see full Prescribing Information for LEUKINE at http://www.leukine.com

Indications and Usage

LEUKINE (sargramostim) is a leukocyte growth factor indicated for the following uses:

About Partner Therapeutics, Inc.:

PTx is an integrated commercial-stage biotech company focused on the development and commercialization of therapeutics that improve health outcomes in the treatment of cancer. PTx's development focus spans the entire range of cancer therapy from primary treatments to supportive care. The company believes in delivering great products with the purpose of creating the best possible outcomes for patients and their families.

SOURCE Partner Therapeutics, Inc.

https://www.partnertx.com/

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Partner Therapeutics (PTx) Announces the Appointment of John McManus as Chief Business Officer - PRNewswire

MONTREAL, Nov. 06, 2019 (GLOBE NEWSWIRE) -- ExCellThera Inc., an advanced biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, announced today the publication of full data from the first clinical trial using ECT-001 (single UM171-expanded cord blood) in patients with haematological malignancies. The data were published in the peer-reviewed medical journal, The Lancet Haematology.

The clinical trial findings indicate that ECT-001 cell therapy is feasible, safe (as suggested by the low transplant-related mortality, low incidence of severe acute graft-vs-host disease (GVHD), and absence of moderate to severe chronic GVHD) and allows for the use of small cords without compromising engraftment. In addition, ECT-001 has shown potential to overcome the disadvantages of unexpanded cord blood transplants while maintaining their benefits of low risk of chronic GVHD and relapse. The Lancet Haematology paper provides the first detailed analysis of the study results presented at the 60th American Society of Hematology Annual Meeting (ASH 2018) in December 2018 and supports the recent advancement of the ECT-001 clinical program.

Were pleased that these important results from the first clinical trial using ECT-001 in haematological malignancies are now fully available to the broader bone marrow transplant community, said Dr. Guy Sauvageau, CEO and founder of ExCellThera, and co-senior author of the paper. These results indicate that ECT-001 transplants combine the advantages of conventional grafts using bone marrow (low treatment-related mortality), peripheral blood (fast engraftment) and cord blood (greater accessibility, low relapse and chronic GvHD) in a single, low cost, easy to produce 7-day culture product, which could lead to a paradigm shift in bone marrow transplantation.

The FDA granted ECT-001 Orphan Drug Designation for the prevention of graft-versus-host disease in 2018 and Regenerative Medicine Advanced Therapy Designation in the treatment of hematologic malignancies in 2019. ECT-001 is currently being used for the treatment of blood disorders in other ongoing and approved clinical trials in the United States and Canada. ExCellThera also plans to initiate a European clinical trial as well as a pivotal, multi-centre clinical trial in the coming months.

About ExCellThera Inc.

ExCellThera is an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for use in novel one-time curative therapies for patients with hematologic malignancies, autoimmune and other diseases. ExCellTheras lead solution combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials. excellthera.com

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ExCellThera announces publication in The Lancet Haematology highlighting excellent clinical results of ECT-001 in patients with haematological...

Antineoplastic is a class of medicine to restrain or kill tumor cells in the body. It plays an important role in comprehensive cancer treatment, particularly for disseminated tumor and leukemia that cannot be treated with surgery. Most current antineoplastics work by inhibiting DNA or RNA synthesis, or even damaging the structure of DNA. Antineoplastics can be divided into CCNSA (Cell cycle nonspecific drug) and CCSA (Cell Cycle Specific agents) according to their different effect on tumor cells in different stages of generation cycles. Both of them have a killing effect on bone marrow hematopoietic stem cells and other cells with a short growth cycle, as well as an inhibiting effect on the bodys immune response.

Factors such as growing incidence of cancer across the globe and demand for cost-effective treatment options are believed to play crucial role for the growth of global anti-neoplastic agents market. Rapid innovation in the field of personalized medicine presents a huge opportunity to manufacturers of anti-neoplastic agents. However, heterogeneous nature of cancer and high development cost of neoplastic agents are the factors limiting the growth of global anti-neoplastic agents market.

Access Report Details at: https://www.themarketreports.com/report/global-anti-neoplastic-agents-market-research-report

The global Anti-Neoplastic Agents market is valued at xx million US$ in 2018 is expected to reach xx million US$ by the end of 2025, growing at a CAGR of xx% during 2019-2025.

This report focuses on Anti-Neoplastic Agents volume and value at global level, regional level and company level. From a global perspective, this report represents overall Anti-Neoplastic Agents market size by analyzing historical data and future prospect. Regionally, this report focuses on several key regions: North America, Europe, China and Japan.

Key companies profiled in Anti-Neoplastic Agents Market report are Hoffmann-La Roche, Amgen, Bristol-Myers Squibb Company, Baxter, Boehringer Ingelheim, Bayer, Teva Pharmaceutical Industries, Johnson & Johnson, Merck, Pfize, Accord Healthcare, Lundbeck, Abbvie and more in term of company basic information, Product Introduction, Application, Specification, Production, Revenue, Price and Gross Margin (2014-2019), etc.

Purchase this Premium Report at: https://www.themarketreports.com/report/buy-now/1416837

Table of Content

1 Anti-Neoplastic Agents Market Overview

2 Global Anti-Neoplastic Agents Market Competition by Manufacturers

3 Global Anti-Neoplastic Agents Production Market Share by Regions

4 Global Anti-Neoplastic Agents Consumption by Regions

5 Global Anti-Neoplastic Agents Production, Revenue, Price Trend by Type

6 Global Anti-Neoplastic Agents Market Analysis by Applications

7 Company Profiles and Key Figures in Anti-Neoplastic Agents Business

8 Anti-Neoplastic Agents Manufacturing Cost Analysis

9 Marketing Channel, Distributors and Customers

10 Market Dynamics

11 Global Anti-Neoplastic Agents Market Forecast

12 Research Findings and Conclusion

13 Methodology and Data Source

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Global Anti-Neoplastic Agents Market: Development History, Current Analysis and Estimated Forecast to 2025 - Market Research Reporting

Many for-profit stem cell clinics advertise therapies that are not backed by science and may actually cause harm.

For-profit stem cell clinics have popped up around California in recent years, advertising that they can treat everything from arthritis to Alzheimers, without FDA approval.

They claim that injections of stem cells (naturally occurring blank slate cells that can grow into any type of cell) can help alleviate pain or illness by replacing or regenerating diseased tissue claims that are not supported by existing research. The procedures can cost thousands of dollars out-of-pocket, and regulators have warned that patients have developed tumors, suffered infections and even lost eyesight after unapproved procedures.

No one knows how many clinics there are, but California reportedly has more than any other state. We asked Arnold Kriegstein, MD, PhD, director of the UC San Francisco Developmental & Stem Cell Biology Program, about whats real and whats not in stem cell medicine.

How do these clinics operate?

There has been an explosion of so-called clinics offering stem cell treatments for a wide range of ailments, none of which have been shown to be effective. They are largely unregulated. Many clinics claim that they can treat untreatable illnesses like Alzheimer's disease, autism, muscular dystrophy, or stroke. The list is quite extensive.

The majority are using fat tissue for their stem cells, obtained through liposuction. These are usually autologous cells, which means that they are taking the patient's own tissue and extracting cells to re-administer to the same patient, usually through an intravenous route. In addition to fat cells, some clinics administer bone marrow stem cells or umbilical cord or placental stem cells, which come from unrelated donors.

The clinics often advertise through testimonials from patients who've received their therapies. Many of the conditions that the testimonials address are the kinds that normally improve or fluctuate over time, such as joint pain, low back pain, arthritis, or multiple sclerosis.

The problem is that patients will receive a treatment, and then, within a month or two, they'll notice that the aches and pains in the joints are improving, and they will attribute the improvement to the stem cell therapy, when in fact it would've happened regardless.

What is the risk of trying an unproven stem cell treatment?

Reports of physical harm have included infections and the development of tumors. When using cells that are not the patients own, umbilical cord cells for example, immune responses can occur often triggering inflammatory conditions.

In cases where stem cells have been delivered into the eye, blindness has been reported, and when they have been delivered to the central nervous system through lumbar puncture (spinal tap), adverse outcomes including serious infections of the central nervous system and tumors have occurred.

Then there's the emotional cost associated with raising false hope, and the financial loss that comes from exorbitant fees charged for ineffective, potentially harmful therapies.

Why arent there more legitimate stem cell therapies available?

Stem cells have been in the news so much over the last decade or so that I think it has created the impression that therapies are already on the market. The reality is that it is very early days for the science. The most interesting, most promising animal studies are only now beginning to be translated into clinical trials, and the process for approval of therapies takes many years and very few are likely to succeed.

Unfortunately, the public needs to be patient, but the good news is that potential treatments are progressing along the pipeline.

What are some examples of proven stem cell therapies?

For the last 50 years or so, there have been countless patients successfully treated with hematopoietic stem cells, commonly known as bone marrow transplants. This remains the prototype for how a stem cell therapy can work. Other successful examples include corneal stem cell grafts for certain eye conditions, and skin grafts for burn victims.

There are efforts to see if stem cells could successfully treat diseases like Parkinson's and diabetes, particularly type 1 diabetes. There are clinical trials testing whether stem cell therapy might work against macular degeneration, a blinding disease that is very common as people age. There are also early stage clinical trials for nervous system disorders including stroke, spinal cord injury, and ALS (Lou Gehrigs disease).

All of these examples are still at a very early stage, where the primary goal is to make sure that the approaches are safe. To determine if they are effective will require large, well-controlled, relatively long-term clinical trials.

What will it take to advance stem cell therapy into more real treatments?

This is where basic research comes in. The field is evolving quickly, there's much to be done, and there's still a huge amount of promise in stem cell therapies down the road. But it's going to take a lot of very careful and very laborious research before we get there.

Original post:
Stem Cell Therapy: What's Real and What's Not at California's For-Profit Clinics - UCSF News Services

Three researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the states stem cell agency.

The recipients are Dr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; and Dr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.

Dengs four-year, $10.3 million award will fund a clinical trial for a blinding eye condition called limbal stem cell deficiency. Limbal stem cells are specialized stem cells in eye tissue that help maintain the health of the cornea. Because of genetic defects or injuries caused by infections, burns, surgeries or other factors, some people do not have enough limbal stem cells, which results in pain, corneal scarring and blindness.

The approach she is testing involves extracting a small number of limbal stem cells from a persons eye, multiplying them in a lab, and then transplanting them back into the eye, where they could regenerate the cornea and restore vision. The research will be conducted in collaboration with theUCLAUCI Alpha Stem Cell Clinic, a partnership between UCLA and UC Irvine.

The grants awarded to Chen and Kuo are for projects that are heading toward the FDAs investigational new drug application process, which is required by the agency before a phase 1 clinical trial the stage of testing that focuses on a treatments safety.

Chens two-year, $3.2 million award will fund efforts to create a more effectiveCAR T cell therapyfor multiple myeloma, a blood cancer that affects white blood cells. The research will evaluate a specialized form of CAR T therapy that simultaneously targets two markers, BCMA and CS1, commonly found on multiple myeloma cells. CAR T therapies that target BCMA alone have been effective in clinical trials, but the presence of BCMA on multiple myeloma cells is not uniform.

Previous research has shown that the marker CS1 is present in around 90% of multiple myeloma cells. A CAR T therapy that targets both markers could potentially help more patients and reduce the likelihood of a cancer relapse.

Kuos 2 1/2-year, $4.9 million award, will support the development of a stem cell gene therapy for a deadly immunodeficiency called X-linked hyper IgM syndrome, or XHIM.

The syndrome, which is caused by a mutation in the CD40LG gene, results in invasive infections of the liver, gastrointestinal tract and lungs. Currently, the only potential cure is a bone marrow transplant from a matched donor, which carries life-threatening risks and is often less effective for XHIM patients than patients with other forms of immune deficiency. Even with current treatments, only 30% of people with the syndrome live to age 30.

Kuo will evaluate a stem cell gene therapy that corrects the genetic mutation that causes XHIM. After removing blood-forming stem cells from a person with the syndrome, the therapy would use a genetic engineering technique called CRISPR to insert a correct copy of the affected gene into the DNA of the stem cells. The corrected blood-forming stem cells would be infused back into the patient, where they could regenerate a healthy immune system.

She will collaborate with Dr. Donald Kohn, a UCLA distinguished professor of microbiology, immunology and molecular genetics who has successfully treated two other immune deficiencies bubble baby disease and X-linked chronic granulomatous disease with a similar therapy.

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Three UCLA scientists receive grants totaling more than $18 million - UCLA Newsroom

Saving the life of a fellow Canadian can be as easy as checking a box online or saying yes to being an organ donor when you renew your drivers license. But, thats just the beginning for those wanting to make a difference.

Deceased donations

In Alberta, individuals over the age of 18 can register their intent to become an organ or tissue donor when they die by using the Alberta Organ and Tissue Donation Registry. (Go to myhealth.alberta.ca online and search organ donation registry.) As well, agents and provincial registries are required to ask the donor question when clients are renewing a drivers licence or identification card.

For those who have Alberta Health Cards issued prior to 2018, the back of the card can be signed (with a witness) to declare their intention to donate.

The Alberta registry has been integrated into the provinces health care system through the use of donor co-ordinators. If a person has declared his or her intent to donate and is in a position to be considered for organ or tissue donation, a co-ordinator will discuss it with family members, who ultimately make the final decision.

Each deceased donor can provide up to eight organs (both lungs, both kidneys, liver, heart, pancreas, intestines), while donated tissues can benefit up to 75 individuals.

Living donations

The vast majority of living organ donors spares one of their two functioning kidneys to a person in need, though living liver donations also occur to a lesser extent.

In most cases, family members or acquaintances donate a living organ if theyre healthy enough to safely act as a donor. Once a viable donor is found, transplant programs in both Calgary and Edmonton perform the surgeries for kidneys, while live liver transplants are only performed in Edmonton.

Theres also been a rise in so-called altruistic donors, who are willing to share their organs with a stranger. Both the Kidney Foundation of Canada and Canadian Blood Services can advise prospective living donors on where to turn, while Alberta Health can connect donors to local living donor programs.

Canadian Blood Services also operates the Kidney Paired Donation Program, an inter-provincial initiative that maintains prospective donors in a registry if they arent a compatible match for their intended recipient. Since January 2009, some 500 living donors across Canada have entered the KPD program, including 90 anonymous donors who joined the program without a specific recipient in mind. Non-directed, anonymous donations are responsible for more than two-thirds of the transplants in the KPD program, and all patients with a match have received a transplant in less than a year.

The Living Donor Services Program Edmonton: Phone 780-407-8698; toll free 1-866-253-6833; email: livingdonors@ahs.ca.

Southern Alberta Transplant Program Calgary: Phone 403-944-4635.

More information on kidney health is available from the Kidney Foundation of Canada: http://www.kidney.ca; 780-451-6900 or 403-255-6108.

More information on liver health is available from the Canadian Liver Foundation: http://www.liver.ca; 403-276-3390 or 1-800-563-5483.

Details about Green Shirt Day and Logan Boulet are at greenshirtday.ca.

Stem cell donations

Stem cell transplants replace a patients unhealthy stem cells with a donors healthy ones, and can be used to treat cancers and other diseases. The three sources of stem cells are from bone marrow, peripheral (circulating) blood and umbilical cord blood.

Prior to any donation, the donor will undergo a comprehensive health assessment before undergoing the procedure. Peripheral blood stem cell donation only requires blood to be drawn from a needle in hospital following five days of under-the-skin injections to boost the number of blood cells in the bloodstream.

Bone marrow donations are performed under anesthesia, with hollow needles used to withdraw stem cells from bone marrow in the back of pelvic bones. The procedure lasts between 45 to 90 minutes and the marrow replenishes itself in four to six weeks.

Those who wish to become a stem cell donor can call Canadian Blood Services at 1-888-2-DONATE (1-888-236-6283) or by visiting the agencys website at blood.ca.

Read the original post:
Organ donations: What you can do to help save a life - Calgary Herald

The research provides in-depth study and analysis on Stem Cell Banking market. This report also gives complete overview of the global market, covering the different aspects such as product definitions along with leading market players. To get better perspectives of global market, relevant chart and graphs are included in the report.

Stem cell banking or preservation is a combined process of extraction, processing and storage of stem cells, so that they may be used for treatment of various medical conditions in the future, when required. Stem cells have the amazing power to get transformed into any tissue or organ in the body. In recent days, stem cells are used to treat variety of life-threatening diseases such as blood and bone marrow diseases, blood cancers, and immune disorders among others.

Stem Cell Banking market report gives the reasonable picture of the current industry situation which incorporates authentic and anticipated market estimate in terms of esteem and volume, technological advancement, macroeconomic and governing factors in the market. The report provides detailed statistics and strategies of the best key players in the industry. The report additionally gives a broad study of the distinctive market sections and areas.

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Some of the leading key players profiled in this study:Cordlife, ViaCord (A Subsidiary of PerkinElmer), Cryo-Save AG, StemCyte India Therapeutics Pvt. Ltd., Cryo-Cell International, Inc., SMART CELLS PLUS, Vita 34, LifeCell, Global Cord Blood Corporation, CBR Systems, Inc

What the report features:-

Global analysis of Stem Cell Banking market from 2017 2027 illustrating the progression of the market.

Forecast and analysis of Stem Cell Bankingmarket by Dosage, Route of Administration and Application from 2017 2027

Forecast and analysis of Stem Cell Banking market in five major regions, namely; North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and South & Central America

The market of stem cell banking is anticipated to grow with a significant rate in the coming years, owing to factors such as, development of novel technologies for stem cell preservation and processing, and storage; growing awareness on the potential of stem cells for various therapeutic conditions. Moreover, increasing investments in stem cell research is also expected to propel the growth of the stem cell banking market across the globe. On other hand rising burden of major diseases and emerging economies are expected to offer significant growth opportunities for the players operating in stem cell banking market.

The Global Stem Cell Banking Market is characterized by the presence of a large number of global, regional, and local players and is highly-competitive. These international players are increasingly focusing on expanding their geographical presence and they have huge production facilities located across the world. Several vendors are increasingly competing against each other based on factors such as innovations, price, and quality of the product. Vendors with better financial and technological resources can withstand changes in different market conditions when compared to their competitors.

The various factors supporting the markets growth and those posing threat are studied in detail in this report. Additionally, the market study segments the Global Stem Cell Banking Market based on end-users, verticals, and size. In these sections, it shields various factors impelling the markets trajectory across the segments. Furthermore, it recognizes the most lucrative of them all to help investors take the well-informed decision.

The Global Stem Cell Banking Market Analysis to 2025 is a specialized and in-depth study of the biotechnology industry with a focus on the global market trend. The stem cell banking market report aims to provide an overview of global stem cell banking market with detailed market segmentation by source, service type, application, and geography. The global stem cell banking market is expected to witness high growth during the forecast period. The stem cell banking market report provides key statistics on the market status of the leading market players and offers key trends and opportunities in the market.

Relating to the latest hierarchy in the global Stem Cell Banking market, the report summaries some of the crucial players operative in the market. Discriminating information about the significant players including their revenue, business segmentation, product portfolio, and financial overview has been integrated in the report. Latest improvements in the industry have been taken into concern while anticipating the future perspective of the market. The report also exemplifies the various marketing channels prevailing in the global market and conveys information about few of the critical distributors functioning in the market. The report assists as a helpful guide for the new as well as prevailing players in the market.

Market Segmentation:

The global stem cell banking market is segmented on the basis of source, service type, and application. The source segment includes, placental stem cells (PSCS), dental pulp-derived stem cells (DPSCS), bone marrow-derived stem cells (BMSCS), adipose tissue-derived stem cells (ADSCS), human embryo-derived stem cells (HESCS), and other stem cell sources. Based on service type the market is segmented into, sample processing, sample analysis, sample preservation and storage, sample collection and transportation. Based on application, the market is segmented as, clinical applications, research applications, and personalized banking applications.

This report includes several arrangements, definitions, the chain assembly of the industry in one piece, and the various uses for the global market. This section also integrates an all-inclusive analysis of the different enlargement plans and government strategies that influence the market, its cost assemblies and industrialized processes. The second subdivision of the report includes analytics on the Global Stem Cell Banking Market based on its size in terms of value and volume.

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The most crucial key factors in the businesses have been elaborated to get ample and accurate data of market dynamics. Rising needs and popularity of Stem Cell Banking Market sector is driving the flow of the market towards progress. In addition to this, it lists the factors which are restraining the growth of the market.

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Stem Cell Banking Market Anticipated to Grow at a Significant Pace by 2028 - Weekly Spy

How isleukaemia treated?

The treatment of leukaemia varies depending on the patient and type of leukaemia they have.

Acute leukaemia (fast developing) is usually curable with standard treatments, such as chemotherapy.

Chronic leukaemia (slow developing), is often incurablebut treatable. For CLL (a form of chronic leukaemia) some patients are not given treatmentstraight away;however if they do require treatment it will often involve chemotherapy.

The main treatments for leukaemia are:

Chemotherapy: This treatment involves theuse ofdrugs.Chemotherapy drugs either kill cancerous cells or stop them from dividing; they can also kill normal blood cells as a side effect.The type of leukaemia you have will depend on the amount and strength of chemotherapy you are offered, along with other factors such as your age and fitness.

Radiation therapy:Similar to chemotherapy, radiation therapy can be used to destroy the cancerous cells but using radiation waves rather than drugs.Again, the type of leukaemia you have will determine what treatment you're offered. External beam radiation therapy (EBRT) is often used for CLL.It is a fast, painless procedure which usually lasts just a few minutes.

Targeted therapy:Drugs are used to block the growth of cancer cells by disturbing specific molecules in the cells. Targeted therapy can also kill cancer cells by stimulating the patient's immune system to recognise the cells as a threat and consequently kill them.

Biological therapy:This treatment does not target the cancer cells directly, but instead helps to stimulate the body's immune system to act against the cancer. It is also often referred to as "immunotherapy". It is often usedfor patients with CML.

Stem cell or bone marrow transplant: Transplants for stem cells or bone marrow are commonly carried out for patients withacute leukaemia,if chemotherapy does not prove effective.By undergoing a stem cell or bone marrow transplant it can help replenish the healthy bone marrow in patients, and stimulate new growth that restores the immune system. It is usually given to younger, or more healthy patients.

Leukaemia Care, which provides support to individuals and families affected by blood cancer, is one ofthree charities supported by this years Telegraph Christmas Charity Appeal. Our two other charities are Wooden Spoon, which works with Britains rugby community to raise money for sick, disabled and disadvantaged children; and The Silver Line, a 24-hour helpline and support service for lonely elderly people. To make a donation, visit telegraph.co.uk/charity or call 0151 284 1927

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Leukaemia: what is it, how to spot the warning signs and who is at risk? - The Telegraph

Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways

Stem Cells In The Body

All humans are born and develop from a small tiny structure called an egg. The cells in the egg have a tremendous potential to develop, multiply and form different cells that are functional in the body. These cells are called mother cells or in scientific terms, they are called stem cells. And all human beings have these stem cells preserved in the body. It is these cells that help us in every day wear and tear and also for tissue repair.

The Body Can Heal Itself

Most of the cells in our body have a definite lifespan that need to be replaced by new cells. The stem cell reserves in the body make up for this and it is done without our knowledge! In fact, any cut or injury, external or internal is healed by the bodys innate mechanism. Our intelligent body recognises the signal of injury and recruits the required stem cells. These stem cells transform themselves into the cells that are required for the repair of the injury and it is always many types of cells in various permutations and combinations.

Where Stem Cells Reside

Bone marrow can be considered as the manufacturing unit of stem cells as it is continuously making blood cells and keeps our circulatory system working perfect all the time. Circulating blood is another source of stem cells, because it works as a courier, carrying cells and other essential enzymes, hormones from one organ to the other in the body. The body converts all the extra material into fat which gets accumulated around the belly. This fatty tissue works like a fixed deposit of stem cells.

Stem cells either from the donor (allogenic) or from the patient (autologous) are being used for more than 50 years and especially for treatment. Blood cancers and other blood-related diseases can be cured using a perfect matched donor stem cells obtained from bone marrow. Patients suffering from organ cancers like breast cancer etc. are given autologous stem cells as a supportive treatment along with chemotherapy and/or radiation.

Protocols for these treatments are standardised globally and considered as standard-of-care. In recent years, umbilical cord blood derived stem cells are being used as an alternative to bone marrow, especially in the paediatric age group. People fall victim to numerous degenerative diseases which occur, as the repairing stem cell system from the body fails slowly with age. Stem cells from the patients body when isolated and administered at an appropriate time and at the right place, with the right dose, is expected to help the patient in various ways. It may also replace, rejuvenate or restore the damaged tissues.

Our body carnes its own repairing kit in the form of stem cells and the body tries its level best to make use of these stem cells to ward off diseases. However, it is possible that with age, the bodys power to recruit and make use of the stem cells diminishes slowly. This is when dreadful degenerative diseases like diabetes, arthritis, Parkinsons disease and heart problems, set in. Heres what the clinical applications of regenerative medicine have found novel mechanisms of:

It is increasingly observed that this kind of autologous therapy takes care of the root cause of disease and offers benefits to patients to whom there is no further solution in other modalities of treatment.

Since each tissue and organ of our body is made up of cells that are derived from the egg cell, any disease which is due to derangement or degeneration of cells can be cured using autologous cellular therapy. And though the list can be endless, here are some examples where there have been very promising results:

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How Stem Cells Can Heal The Body - Version Weekly

This level of disease stabilization has not been observed to this date in approved or investigational ALS therapies.

- Mr. Chaim Lebovits, CEO, Brainstorm Cell Therapeutics

In May of this year, I published an article on Brainstorm Cell Therapeutics (BCLI). This small company is developing a mesenchymal stem cell product called NurOwn, which is in late phase 3 trials targeting amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease. My article was bearish, deploring not only the company's cash position but also phase 2 trial data. The article can be read here.

That article received a lot of critical comments from the ALS community. That made me realize that a fair overview of the issues could be best addressed by going through the comments, as well as my own coverage, and by asking BCLI management, specifically its CEO, Chaim Lebovits, to clarify some of these issues. So, that's what I did. I emailed a set of 11 questions to Mr. Lebovits, and he was kind enough to respond to them in great detail. The entire interview, sans any edits, is available to Total Pharma Tracker members.

Mr. Lebovits has been with BCLI for well over 12 years, joining in 2007 as president and also becoming the CEO in 2015. He has helped develop NurOwn through its preclinical stage to its current stage and is, therefore, just the right person to talk to if we want to understand NurOwn and BCLI.

I began by asking him to locate NurOwn in the ALS therapy space and where it stands with respect to competitors. What's its mechanism of action, and how does that MOA distinguish it from the competition?

Mr. Lebovits said that there are "currently 4 products active in phase 3 ALS clinical trials (Brainstorm (NurOwn, autologous MSC-NTF cells secreting neurotrophic factors), Orion (levosimendan, muscle troponin calcium sensitizer), Orphazyme (arimochlomol, heat shock protein enhancer), and Biogen (SOD1, antisense oligonucleotide)." Top-line data from these ALS phase 3 trials is expected in 2020 (Q4 2020 for Brainstorm) and Orion, 2021 (Orphazyme), and 2022 (Biogen). He discussed a number of earlier-stage compounds as well as various stem cell therapies. He said that what distinguishes NurOwn among ALS therapies is that it "confers both neuroprotection and immunomodulation by delivering neuronal survival factors and immune regulatory molecules, including microRNA directly to the CNS compartment at or near the site of disease, and therefore directly addresses two important ALS disease mechanisms."

Among stem cell therapies, Mr. Lebovits said that NurOwn distinguishes itself by being autologous and because it can produce high levels of neurotrophic factors. Moreover, unlike most stem cell competitors, it's delivered directly into the spinal fluid through bimonthly lumbar punctures, unlike others that need an invasive surgical procedure "that carries considerable morbidity."

This feature it shares with a competing product from Corestem. However, it's differentiated from Corestem because "NurOwn is more convenient than the Corestem product as a single bone marrow cell harvest due to validated cryopreservation, whereas the Corestem product requires repeat bone-marrow aspiration for each treatment."

My next question was a technical question about pharmacoresistance. I wanted to know how NurOwn is managing to cross the blood-spinal cord barrier despite the strong pharmacoresistance (body's resistance to drugs) seen in ALS, specifically for disease-modifying neurotrophic factors. What was it about NurOwn's delivery mechanism that the company thinks is overcoming this natural resistance. So I asked: "Talking about MOA, pharmacoresistance is a disease driving mechanism in ALS. Can you discuss NurOwn's delivery mechanism vis-a-vis the inability of neurotrophic factors to effectively cross the blood-brain barrier, or, specifically, the blood-spinal cord barrier (BSCB)? Please correlate that discussion regarding the observed increase in CSF NTFs post-treatment as seen in the phase 2 trial."

Mr. Lebovits explained this with great clarity - for his entire response, take a look at the complete interview. Broadly, what he said was that NurOwn, being delivered through lumbar puncture directly into the spinal fluid, has an advantage. Moreover, the cells secrete neuronal survival factors as well as molecules that regulate the immune system, so that they are able to survive and overcome the pharmacoresistance. Systemically administered NTFs are unable to do that.

As he said, "In the phase 2 trial, CSF biomarkers obtained just prior to treatment and two weeks afterward demonstrated that MSC-NTF cell-secreted neurotrophic factors were significantly increased post-treatment and correlated with the reduction in inflammatory biomarkers, consistent with the proposed mechanism of action."

My third and fourth questions related to aspects of the phase 2 study. One, comparison of safety and efficacy data with competitors, and two, the relevance of the reported caspase-3 reduction of 60% in responders versus 30% in non-responders.

Mr. Lebovits said that although the phase 2 study was not powered for efficacy, it exhibited a "level of disease stabilization (that) has not been observed to this date in approved or investigational ALS therapies." About the ongoing phase 3 study, he said the following:

Those who read my original article will recall I was particularly puzzled by the increased occurrence of serious adverse events in active-treatment groups than in placebo groups. 8/36 or 22.2% patients in the treatment arm had an SAE compared to only one out of 12 placebo patients, or 8.3%. Most SAEs were related to the progression of the underlying ALS, most commonly dysphagia. No SAEs were related to study treatment. So I asked Mr. Lebovits how this data could be interpreted in the most positive way.

According to him, this decline was not an effect of treatment itself and simply indicated the need for repeat dosing in this patient group. His exact response was as follows:

The MSC-NTF treated group had a slightly more rapid rate of decline compared to the placebo group in the three-month run-in period and most ALS disease progression in the treated group was seen toward the end of the clinical trial, long after a single transplantation. In fact, the bulbar subscale, that includes assessment of swallowing, was the subscale most improved after MSC-NTF treatment in rapid progressors, suggesting that the late decline in motor function was not an adverse effect of treatment per se. Hence the need for repeated dosing.

Last week, the DSMB recommended continuation of the phase 3 trial without any modification. This was major good news, so we asked him about this. Mr. Lebovits said that this was a second interim safety review, and there were no significant safety concerns. Therefore, the DSMB recommended no modification in protocol and no other interim analysis is planned. Phase 3 data will be available by mid-2020 according to this interviewer's reading of the press release.

Now we moved on to another critical aspect of our analysis - funds, or rather, the lack of it. Since this is an important issue, here's the exact exchange we had.

Dr. Ashok Dutta: How does the company plan to fund its operations through the next couple years until the lead development candidate is approved and commercialized? Given the weak financial position, does Brainstorm see the possibility for ATM operations, or thinks about selling rights in regions like China, Japan or Europe to increase the financial condition?

CEO Chaim Lebovits: As you are aware we do receive proceeds from the hospital exemption pathway and also receive grant funding from CIRM and IIA. These avenues have allowed to fund and continue with our trials over the years with non-dilutive financing. From a business standpoint as our ALS phase 3 trial is now fully enrolled, the management team continues to hold high level conversation with some of the leading global pharmaceutical and biotechnology companies. We are actively engaged in strategic partnering and collaboration discussions and although we cannot disclose the details of our conversations due to NDAs we signed with them... we are exploring several opportunities with key interested parties to advance the opportunities for NurOwn development and commercialization. As you have rightly pointed out, we have a $20mm ATM facility in place with Raymond James. We may activate the ATM as required and raise up to $20mm by selling our stock "at the market" only if the prices are attractive to us. So far as of end of Q3'19, we have not activated the ATM. If the need arises and the prices are attractive to us, we may employ this tool to raise capital.

This is reassuring that the company intends to focus on non-dilutive financing. The ATM facility, coupled with the grants, should ideally see them through the approval phase. We still wonder how they will manage marketing and sales. Perhaps those commercialization NDAs they have signed will help.

Next, we discussed market potential and a question about a recent patent grant. The CEO's detailed responses can be found in the complete interview material.

The strong involvement of the ALS community impressed us previously, so we now asked the CEO about the recent roundtable convention they had with ALS advocacy groups. Since this will be important for the ALS community as a whole, here's Mr. Lebovits' entire response on the question:

Finally, we asked him what we ask everyone: Give us three simple and straightforward reasons why investors would be interested. Here's what he said:

Thanks to the ALS community for inspiring us to conduct this interview, and to Mr. Chaim Lebovits, CEO of Brainstorm Cell Therapeutics, for answering our questions.

Thanks for reading. At the Total Pharma Tracker, we interview management of important small biotech doing disruptive work in healthcare. Our members are given exclusive access to these interviews, which helps them with additional primary resource in doing DD on their investments. Sometimes, extracts from these interviews may be published for everyone; but TPT members always get the exclusive view.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: General Disclaimer - This is to confirm that Avisol Capital Partners has neither requested, nor been offered, any monetary compensation for conducting this interview, by any party other than Seeking Alpha.

Also to be noted, this was an emailed questionnaire, and certain editorial material is present in this version, which may or may not reflect BCLI or its CEO's position on the issues discussed.

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Interview With Chaim Lebovits, CEO Of Brainstorm Cell Therapeutics - Seeking Alpha

MULTIPLE sclerosis campaigners have hailed a huge step forward for patients in Scotland after a stem cell therapy was recommended for use on the NHS for the first time.

Haematopoietic stem cell transplantation (HSCT) has been described as a game-changer for MS after an international clinical trial showed that it could reboot patients immune systems and halt the progress of the disease.

Some patients who had been in wheelchairs prior to treatment said their condition improved so dramatically it was like they had never been diagnosed with MS.

READ MORE: Scots MS patients 'missing out' on pioneering stem cell treatment available in England

The Scottish Health Technologies Group (SHTG) said there is now sufficient evidence for it to recommend making HSCT available on the NHS in Scotland to MS patients who have the relapsing-remitting form of the disease, and who were not responding to drug treatments.

Iain Robertson, chairman of the SHTG, said: Our committee members were able to advise that this treatment should be considered for those with this particular type of MS who have not responded to treatment with disease-modifying therapies.

We hope that our advice will be of use in helping decide the best course of treatment for these patients.

The SHTG also stressed that patients must be made aware of the demands, risks and uncertainties of the treatment, which uses chemotherapy to wipe out patients' 'faulty' immune systems before replenishing it with a transplant of stem cells harvested from their own bone marrow.

It puts patients at high risk from infections, which can be fatal, but the theory is that the treatment works by enabling patients to 'reset' their immune system to stop it attacking the central nervous system as is the case in MS.

READ MORE: Anger of Scots MS patients travelling abroad for stem cell therapy available to some on NHS England

HSCT is not considered an effective treatment for patients with the progressive form of MS, however, as stem cells cannot regrow nerves or repair damaged myelin - the protective sheath which coats nerves.

It will also be unavailable to patients with relapsing-remitting MS who no longer show signs of inflammation on an MRI brain scan.

Scotland has one of the highest rates of MS in the world, but until now Scottish patients seeking HSCT have had to travel overseas to Mexico, Russia and Israel and bankroll their own private treatment at a cost of around 40-60,000.

It has also been available privately in London since 2017, but with a 100,000 price tag.

A small number of MS patients in England have been able to access the treatment on the NHS, however, because there are clinical trials into HSCT taking place at NHS hospitals in Sheffield and London.

Morna Simpkins, director of MS Society Scotland, said: The decision from SHTG to approve HSCT for the treatment of MS is good news and could help in the development of a clear pathway, for people who could potentially benefit, to access it.

We will push to ensure that this decision leads to real change for people with MS by continuing to engage with other groups to offer the treatments, including HSCT, which are right for them.

READ MORE: Stem cells help mother with MS make 'remarkable' recovery

The SHTG said eligible patients must have equal access to the procedures regardless of where they live, but it is unlikely all health boards will be able to provide it.

The MS Society wants a centre, or centres, of excellence set up where patients from across Scotland can be referred.

Lucy Clarke from the Scottish HSCT Network said the recommendation was "a huge step forward" for people in Scotland living with MS.

Ms Clarke underwent HSCT in Russia and credits it with substantially reversing her disability.

She added: This important decision supports HSCT as a treatment option where other treatments have failed. We will continue to push so that this treatment is available to people in Scotland who need it.

A Scottish Government spokeswoman said: We are grateful to the Scottish Health Technologies Group for this important work.

"NHS Boards are expected to consider their advice on technologies in the planning and provision of its services and clinicians are expected to follow their professional judgement, working within the management structure of their Board.

We will work closely with MS Society Scotland, other third sector bodies and the clinical community to consider what the Technologies Groups findings means for provision in Scotland, including the information that needs to be available to people about eligibility and risks.

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Stem cell therapy approved for MS patients in Scotland - HeraldScotland

In September end, a good news greeted the biomedical world when a team led by Takanori Takebe at Cincinnati Children's Hospital Medical Center succeeded at growing a connected set of three organs: the liver, pancreas and biliary ducts, in the lab, from human stem cells. The findings were published in journal Nature.

While human organoids already provide a sophisticated tool for research, the connected set of three organs, for the first time, allow scientists to study how human tissues work in concert. This was dubbed as a significant step forward.

In October, another news came. At the annual meeting of the Society for Neuroscience, researchers said that brain cell clusters prepared in the lab- a type of organoid- show abnormal behaviour as compared to the normal brain cells.

They said that the cells in these clumps had ambiguous identities and made more stress molecules than cells taken directly from human brains. However, these abnormalities were found to be alleviated a little bit when the implanted into a more hospitable environment - a mouses brain.

What are organoids?

With the available technology, scientists can grow a group of cells in laboratories into three-dimensional, miniature structures that mimic the cell arrangement of a fully-grown organ.

This is done using stem cells.

Stem cells are special human cells that have the ability to develop into many different cell types, from muscle cells to brain cells.

The embryonic stem cells that are derived from unused embryos (These are created from an in vitro fertilization procedure and used for scientific research) are pluripotent, meaning, they can turn into any type of cell.

On the other hand are adult stem cells. They are derived from fully developed tissues, like the brain, skin, and bone marrow. These cells often have capability of turning into only certain types of cells. For example, a stem cell derived from the liver will only generate more liver cells.

However, the adult stem cells can be manipualted in the laboratory to act like embryonic stem cells. These are called induced pluripotent stem cells. (The technique was developed in 2006). However, scientists are yet to find adult pluripotent stem cells that can develop every kind of cell and tissue.

When scientists create right environment in the laboratory for them, these stem cells follow their own genetic instructions to develop into tiny structures that resemble miniature organs composed of many cell types.

Using these, researchers have been able to produce organoids that resemble the brain, kidney, lung, intestine, stomach, and liver etc.

For example, in the three-organ research mentioned above, Dr Takebe started with stem cells from human skin cells and then guiding and prodding those stem cells to form two very early-stage "spheroids" of cells loosely termed the foregut and the midgut (In human embryos, these form late in the first month of gestation. Over time, they merge and morph into the organs that constitute the digestive tract).

The spheroids were first placed next to each other in a lab dish suspended in a gel used to support organoid growth, then placed on top of a thin membrane that covered a carefully mixed batch of growth medium.

From this point on, the cells knew what to do, and 70 days later, the mini organoids began processing bile acids as if they were digesting and filtering food.

Why are organoids important?

The technique to develop organoids was named by The Scientist as one of the biggest scientific advancements of 2013.

Organoids are an excellent tools to study biological processes like uptake of nutrients, drug transport, secretion of hormones and enzymes etc. This way, diseases related to malabsorption of nutrients, and metabolism-related diseases like obesity, diabetes, insulin resistance can be studied at the cellular-level.

Recently, scientists at the at Memorial Sloan Kettering created a tumor organoid to develop a more accurate rectal cancer model.

In the case of the human brain, organoids opens a window to understand some of the most complicated and hidden aspects of our own biology. They can be used to study neuropsychiatric or neurodevelopmental diseases like schizophrenia or autism spectrum disorder, which are uniquely human diseases that affect the whole human genome.

Organoids also provide a window into how cells interact with each other and their environment. They can be used to create cellular models of human disease, which can be studied in the laboratory to better understand the causes of disease and identify possible treatments. The effects of different drugs and be tested.

Scientists have even used gene editing techniques (CRISPR-Cas9) on the stem cells to to introduce targeted mutations in genes corresponding to two different kidney diseases. When these modified pluripotent cells grew into human kidney organoids, they exhibited the diseases.

Using such organoids relieves the scientific community from experimenting on human and animal subjects. Also, certain treatments that would be unethical to administer on the latter, can be tested on the organoids.

With organoids, researchers can produce a limitless supply of tissue from each patient. This will also be extremely useful for the study of rare diseases, where the number of patients on which to conduct research and test treatments is limited.

Organoids are also being used to develop personalised and precision medicine.

For example, it was found that repairing the CFTR protein could give relief to a patient suffering from non-cystic fibrosis, an inherited disease caused due to a gene mutation. Using the Intestinal organoids grown from a patients stem cells, the doctors could quantify the patients response to the CFTR modulating therapy.

Organoids can have significant therapeutic applications. For example, pluripotent stem cells derived from a diabetes patient could be transformed into insulin-producing beta-like cells.

Organoids also offer an incredible opportunity to study developmental biology. Using them, for example, we can learn more about how organs are formed in embryonic stages and associated disorders.

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Growing Human Organs In A Lab: As Scientists Develop Pathbreaking Three-Organ System, Heres All You Need To Know - Swarajya

WHEN Laura Farmer-Maia's daughter suddenly became clingy and unhappy, she initially brushed it off as nothing but "a phase".

And given little Beatriz was just three-years-old, the last thing to cross her mind was cancer.

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Yet, months after doctors repeatedly dismissed the symptoms as clinginess, the diagnosis was confirmed - Beatriz had an aggressive childhood cancer known as a neuroblastoma.

The horrifying news came after Beatriz's father Tiago Maia refused to leave the hospital after discovering the potential diagnosis himself on Google.

Shocked, Laura, 39, and Tiago, 40, are now urging all parents to be vigilant and check their kids for signs of the disease.

The mum, who works in advertising, first suspected something was wrong last July, when Beatriz's behaviour dramatically changed.

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She said: "Before she was diagnosed, Beatriz was quite naughty but when she reached two, she suddenly became clingy and picky with her food, and had a fever all the time.

"We took her to the GP who believed it was a virus and after recurrent visits they gave her some antibiotics to cover for a potential bacterial cause, which didnt have any effect.

"Beatriz started to complain that her legs hurt so we took her to A&E, where they did some more tests and still said it might be a virus.

"Its hard to get a diagnosis right when a child is too young to explain how theyre feeling, but in the back of our minds we knew it was something bad.

"We want to spread awareness of the difficulty of diagnosing cancer in young children - if your child doesnt seem right, you should push for further tests."

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Tiago pushed the GP for more tests, and blood tests showed something was wrong, so Beatriz was sent urgently to hospital.

It was there medics finally discovered a lump above her kidney and diagnosed her with neuroblastoma in September last year.

The cancer is aggressive and has a 40 per cent chance of long-term survival.

Everything moved so quickly and we all felt frightened as they carried out the tests

Tiago, originally from Portugal, added: "At the hospital, they twice said it was likely to be a virus and I refused to leave until I saw a specialist.

"I waited for three hours until a more senior doctor was free, and then Beatriz was examined by different specialists who admitted her to do all kinds of tests and observations including X-rays and ultrasounds - it was the last one that confirmed there was a lump.

"When my fears from Google turned out to be true, it was very strange because even though my life had just flipped upside down, I was almost relieved to be right - it was weird and confusing."

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Beatriz was referred straight to Great Ormond Street Hospital in London, where they carried out further tests including scans, blood tests and biopsies.

She began chemotherapy just a week after being diagnosed and underwent eight gruelling rounds of chemo over the next 18 months.

Doctors then carried out a stem cell transplant to regenerate bone marrow destroyed by high dose chemo, which meant Beatriz couldnt leave the hospital for eight weeks.

Tiago, a design director, said: "I was quite scared when Beatriz was diagnosed because my mum and dad had only recently died from cancer I thought of the worst.

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"Everything moved so quickly and we all felt frightened as they carried out the tests.

"Doctors found that the cancer had spread across her body, so she began chemotherapy just weeks after being diagnosed.

"We were told the treatment would last 18 months which was a massive shock to us.

"A week after Beatriz started chemotherapy she massively improved, but it was tricky being in hospital at first.

"Now, she still has periods of discomfort but sometimes shes happy to be in hospital because she has toys and people to come and play with her."

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Laura added: "The stem cell transplant was a hard time for us all because we had to spend a lot of time apart from our other daughter, Clara, six.

"Beatriz was diagnosed in Claras first week of school and it was difficult because that was supposed to be an exciting time for her."

After more scans and hopes of an all-clear, doctors found more metastatic growths still remaining in Beatrizs head, which meant that the cancer hadnt fully cleared up and she had relapsed.

The brave youngster is now undergoing immunotherapy and is due to start a six month medical trial on the NHS at Great Ormond Street Hospital, called the Beacon Trial.

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What is neuroblastoma?

Neuroblastoma is a type of cancer that most commonly afflicts babies and young children.

The disease develops from special nerve cells, known as neuroblasts, which get left behind from the child's development in the womb.

It mostly begins in the sufferer's adrenal glands located above the kidneys but can occur in the nerve tissue that runs along the spinal cord in the neck, chest, abdomen or pelvis.

The vicious illness can then spread to other organs like the bone, bone marrow, lymph nodes and skin.

Neuroblastoma afflicts around 100 children a year in the UK but the cause of the disease is still not known.

Its symptoms can include:

It is uncertain whether the trial will work and, even if Beatriz goes into remission, relapse rates are high but her parents are determined to do everything they can to stop the cancer from returning.

Laura and Tiago are now trying to raise 200,000 to help get their daughter into remission or to keep the cancer away if her treatment goes well.

The money is hoped to go towards further treatment, or if Beatriz gets the all-clear, a special vaccine in New York which helps keep the disease away.

Laura said: "After the stem cell transplant, the end was almost in sight but then she relapsed.

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"Were afraid that the cancer will get worse and worse and want to raise money to help get her into remission the ideal outcome is that the trial works and clears the disease.

"Luckily, compared to other two-year-olds, Beatriz has suffered less side effects with treatment and despite losing her curly hair shes powering through."

You can donate on Beatriz's JustGiving page here.

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Docs said our toddler was just clingy but we learned the truth on Google it was cancer - The Sun