Archive for the ‘Bone Marrow Stem Cells’ Category

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

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

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

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

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

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

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

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

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

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

Angie Grice at Clemson University(Photo: Angie Grice)

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Gavin McNaney, who passed away two years ago.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

OBJECTIVES:

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

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

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

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

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

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

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

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

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

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

There are four primary regenerative medicine options:

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Medically reviewed by Richard M. Stone, MD

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

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

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

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

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

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

People with leukemia may experience:

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

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

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

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

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

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

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

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

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

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

Treatments for leukemia can include:

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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Stem cells' role in medicine and research - The Medium

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

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

Image source: Getty Images.

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

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

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

Vertex's Chief Scientific Officer David Altshuler said,

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

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

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

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

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

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

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

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

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

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2 Small-Cap Biotechs That Soared Last Week - Motley Fool

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

RACHAEL LONG

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

RACHAEL LONG

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

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

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

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

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

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

RACHAEL LONG

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

RACHAEL LONG

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

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

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

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

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

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

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

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

RACHAEL LONG

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

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

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

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

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

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

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

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

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

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

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

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

RACHAEL LONG

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Total Posts: 94

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

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

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

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

Description:

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

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

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

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

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

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

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

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

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

Region Segmentation:

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

About NurOwn

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

About BrainStorm Cell Therapeutics Inc.

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

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

Safe-Harbor Statements

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

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

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

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

By Oliver and Elizabeth Hedgepeth

There are special suppliers of life in our great country, from North Carolina to Virginia to Alaska. They are those hospitals that collect the basic raw material for giving life. They work with a network of donor service organizations across the United States. In Virginia, it is Donate Life Virginia. In North Carolina, it is Carolina Donor Services. In Alaska, it is Life Alaska Donor Services.

The raw material that comprises those supply items are you, me, anyone from 3 months old to 75 years old, so far in our experience. Yes, a 3-month-old can die of many causes some accidents, others an incurable disease. But, that 3 month-old can give life and sight and other helpful body parts to others, as can that 75-year-old. The final person to receive such a gift is you, your wife, child, husband, mother, father, a teacher, a prisoner in jail anyone and everyone.

There are more than 50 different parts of a persons body that can be donated to help others live a better life. Those supply items are organs, corneas, tissues, hands and face, blood stem cells, cord blood, bone marrow, blood and platelets. The number of people given this gift of life exceeded 113,000 in 2019.

Real-life experience: We recently attended a Donor Family Tribute in Greenville, N.C. The sponsor of this event was Carolina Donor Services. The building was huge and looked like a country club. We were not sure if we were at the right place, and we even questioned why we should spend our Sunday afternoon there.

This nice-looking building clearly was a place to hold a special event. When we reached the register desk, we discovered our name was not on the list. We debated for three months after the invitation arrived whether we wanted to be around a group of people who lost their loved ones.

There was a meeting and dining area, much as you would expect at a professional conference. There was nice, light music playing in the background, the walls were black and there were quilts hanging all over the front of the room. The quilts had small 12-inch squares on them. It was obvious that the quilt was a remembrance of the ones who had died.

We sat at a table that had many place settings and chairs. We sat quietly for about 30 minutes, as around 200 people entered the room and took their seats. When the room filled, the talking was in whispers, as if we were in church waiting for a service to begin. We thought about quietly getting up and leaving. We did not fit in here.

The 200 people were a mix of races, ages and abilities. A spokesperson on stage invited all the guests to join the buffet line. We all did, and the group ate for about 30 minutes, again like a church social. Then it began.

The speaker asked if anyone would like to tell about a loved one who donated to help others live. Slowly, people many of whom had never spoken in front of a group walked to the microphone. One woman, smiling and happy with tears of joy running down her face, spoke about finding her 15-year-old son in his room at home, hanged. She described how it took three days for him to die of his suicide.

Then, she happily said his hand was being used by another young boy who had lost his in an accident and how her sons eyes would make another person see for the first time in years.

Another person shared the story of how a 3-month-olds death from an incurable disease helped other life-threatened babies live. The sharing of stories went on for about three hours.

When we gathered to leave, we and those 200 people were all the same. We were friends, like long-lost relatives. There was no age or race or illness separating us. We all treated each other as the same.

People are waiting: When someone you love dies, grief memoirs seem the same. Being around those who also have lost someone and are grieving seems to be a logical connection. The topic of conversation is similar and shared. But the loss is still there for the person so loved. Something changed with this donor tribute.

The 200 or so people with their common loss encountered a gain. Many of them know the person who has received a new hand, or can see, or can talk for the first time in years. Knowing that their loved one is still alive in a small part of someone else, maybe even the heart itself, gives comfort to us who have been left with such grief in the past.

The donor process of giving was not around when our parents died. If it had been, our visits to the gravesites would hold a little more light of happiness, knowing someone was walking around on a farm or in an office with our loved ones heart or arteries or hands.

Donate Life Virginia is a small part of life-giving across all of America. Please, donate in your state when your time comes. We are.

Oliver Hedgepeth is professor of logistics for the American Military University. Elizabeth Hedgepeth is former managing editor of the Petersburg Progress-Index. Contact them at: blh4835@gmail.com

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Oliver and Elizabeth Hedgepeth column: Human donations are a gift of life - Richmond.com

Just a few months after Health Canada began cracking down on private clinics offering unapproved stem cell therapies, at least one U.S. clinic has moved in to fill the vacuum with direct marketing to Canadian consumers.

The clinic from Burlington, Vermont, even offers shuttle buses to transport people from Ottawa to the clinic four hours away for treatment it suggests will end joint pain, among other things. Lunch and dinner are free, but each injection costs $6,880. Two for $10,880.

The treatments, using umbilical cord-derived mesenchymal stem cells, are not approved in either Canada or the United States. Health Canada warns that Canadians who travel abroad for stem cell treatments may put themselves at risk.

While stem cells, which were discovered at the University of Toronto in 1961 by James Till and Ernest McCulloch, promise to revolutionize many treatments and could offer breakthroughs for diseases, almost all are still considered experimental and have yet to be proven safe or effective. Clinical trials on numerous potential stem cell therapies are under way, including in Ottawa.

While research progresses, private stem cell clinics have popped up around the world making promises for treatments not yet proven safe or effective.

A 2018 study by Leigh Turner of the University of Minnesota Center for Bioethics found 43 clinics offering stem cell treatments in Canada and 750 in the U.S. Earlier this year, Health Canada sent Canadian clinics, including some in Ottawa, cease-and-desist letters.

Clinics in Vermont, near the Canadian border, appear to have ramped up marketing to Canadians since then. One clinic has been holding back-to-back seminars. Another says it stopped marketing in Canada after receiving a warning from Health Canada.

There have been cases of harm as a result of treatments, including two women who had permanent damage to their sight after stem cells were injected into their eyes at a Florida clinic. Other patients have been infected with unsterilized equipment and others have developed tumours at the site of stem cell injections.

A common harm, critics say, is exploitation.

Dr. Michael Rudnicki is director of the regenerative medicine program and Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute, says of stem cell therapy claims: If it sounds too good to be true, it probably is too good to be true.jpg

Health officials say the clinics are misusing the promise of stem cell therapy to exploit vulnerable patients.

These patients are in pain and they are suffering and they are looking for help and they are being exploited, said Dr. Michael Rudnicki, director of the regenerative medicine program and Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute.

If it sounds too good to be true, it probably is too good to be true.

At a recent seminar at a west-end Ottawa hotel meeting room, Roseanna Ammendolea of the Vermont Center for Regenerative Medicine told a packed room that her clinic and others like it had successfully treated people for pain related to arthritis, neuropathy and other ailments that affected joints using mesenchymal stem cells from umbilical cords. The stem cells, she claimed, are both effective and safe, saying there had been no issues with cell rejection.

We will not give injections if we feel that this injection will not be beneficial to our patients. This is why we are so successful.

Participants, including some who walked with canes and others who talked about being in pain and having mobility issues, were shown videos of people described as Canadian clients who claimed the treatments worked. One man said it was probably the best money I have spent in my life as far as my health. Another said she would do it again in a heartbeat and was able to do things she hadnt been able to do earlier.

They were also shown a slide showing long wait times for hip and knee replacements in Ontario, We are not a priority, she said. Where does that leave us? Participants werent told exactly how the stem cells were supposed to work, but claimed they had successfully improved pain and mobility issues in clients.

What the seminar goers werent told is that, even in the U.S., the treatment is not covered by health insurance because it remains unproven.

The U.S. Federal Drug Administration has issued a warning to consumers not to use cell therapies that are unapproved or unproven.

Stem cells have been called everything from cure-alls to miracle treatments. But dont believe the hype. Some unscrupulous providers offer stem cell products that are both unapproved and unproven. So beware of potentially dangerous procedures and confirm whats really being offered before you consider anytreatment, the FDA said in a statement.

The only stem-cell-based products that are FDA-approved for use in the United States are blood-forming stem cells derived from cord blood for limited use in patients with disorders affecting the body system that is involved in the production of blood. Bone marrow is also used for these treatments, but is generally not regulated by the FDA for that use.

Health Canada has granted market authorization for a stem cell therapy to treat graft-versus-host disease and two cell-based gene therapies to treat certain cancers. Most cell therapies are still experimental.

I totally understand the skepticism of it, Doug Argento, who works at the Vermont Center for Regenerative Medicine, said in a telephone interview, but the fact is that things that are approved now and medically paid for were seen as renegade 20 or 30 years ago.

The treatment employs technology developed by Neil Riordan, founder, chairman and chief science officer of the Stem Cell Institute in Panama, using human umbilical cord tissue-derived mesenchymal stem cells. There are 41 such clinics across the U.S. Riordan also played a role in the development of a nutritional product called Stem-Kine, which producers claim without scientific backing increases the number of stem cells circulating in a persons body.

The stem cells injected in the clinic, Argento said, are from umbilical cord tissue as a result of caesarean births to reduce risk of infection.

Rudnicki, of The Ottawa Hospital Research Institute, says there is no evidence that these sorts of cells are regenerative at all. It would not pass muster in Canada.

The public has to understand that there are people out to remove them from their money.

Rudnicki says he regularly receives inquiries from people desperate to get stem cell treatments. He says he tries to connect them with clinical trials that they might be able to participate in.

Rudnicki noted there were multiple clinical trials in Canada, including treatments of autoimmune diseases, trials involving treatment for Type 1 diabetes and others.

But the use of these inappropriate cell types for treating arthritis and joints and so on is certainly not approved by Health Canada and would not be allowed in Canada under the regulations.

There is some evidence that injections of some stem cell products might have a temporary positive impact on inflammation, he said, but it will not be regenerative and will not restore function to joints. They are being sold a bill of goods.

Leigh Turner of the University of Minnesota Center for Bioethics, meanwhile, says the explosion in clinics offering unproven stem cell therapies in the U.S. is a marketplace that traffics in misrepresentation. It is easy to see how people are taken advantage of and scammed.

It is also difficult to find out about physical harms being done to patients.

There are no safety studies. We dont have good data. But we do know there have been some serious harms.

Stem cell therapies have the potential to become standard treatment in some areas, but they are not there yet, Turner said.

Businesses are tapping into genuine human suffering, desperation and also hope.

Turner also noted there was an excellent chance that the vials of liquid being injected into patients did not actually contain stem cells.

Dr. Jonathan Fenton of another stem cell clinic in Burlington, the Vermont Regenerative Medicine, said he had complained about the new clinic, the Vermont Center for Regenerative Medicine, which has a similar name and employs hard-sell tactics, he said.

His clinic takes bone marrow from patients hips and injects it. The procedure is done the same day. He says he regularly sees Canadian patients for bone marrow aspiration therapy and platelet-rich plasma treatments, using their own blood. The treatments, he says, speed healing and are allowed in the U.S. The use of bone marrow aspiration is neither proven nor allowed in Canada.

Fenton, who is secretary-treasurer of the American Academy of Orthopedic Medicine, acknowledged many people offering stem cell treatments are not doing it to the highest ethical standards.

He has filed complaints with state officials over clinics selling unsafe or fraudulent treatments. I have asked the state and federal judiciary to close down this clinic for committing fraud.

He said his platelet and bone marrow treatments were covered by a major Vermont health insurer because they saw the cost of benefits were going down and patients were requiring fewer surgeries.

He said he was told by Health Canada that he could not market in Canada. Representatives of the Vermont Center for Regenerative Medicine, meanwhile, said they had discussions with Health Canada about what they could and could not say when marketing in Canada before holding seminars in Ottawa and Halifax.

We have looked at the information provided and have not identified any immediate non-compliance with advertising regulations pertaining to Canadian health products, a Health Canada spokesperson said, adding that the agency was continuing to assess.

Back at a west-end Ottawa hotel, some participants in the seminar, including a retired pharmacist, said they were considering getting the treatments. But its expensive.

Another participant said he was skeptical. They seemed very sketchy when I went online.

epayne@postmedia.com

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U.S. stem cell clinic offering unapproved therapies brings direct-to-consumer marketing to Ottawa - Ottawa Citizen

On the morning of April 25, 2018, in Fort Wayne, Indiana, Omarion Jordan came into the world ten-fingers-and-toes perfect. His mother, Kristin Simpson, brought her dark-haired newborn home to a mostly empty apartment in Kendallville, about 30 miles to the north. Shed just moved in and hadnt had time to decorate. Her son, however, had everything he needed: a nursery full of toys, a crib, a bassinet and a blue octopus blanket.

Still, within his first couple of months, he was plagued by three different infections that required intravenous treatments. Doctors thought he had eczema and cradle cap. They said he was allergic to his mothers milk and told her to stop breastfeeding. Then, not long after he received a round of standard infant vaccinations, his scalp was bleeding and covered with green goop, recalled the first-time mother, who was then in her late teens. She took him to the hospital emergency room, where, again, caregivers seemed puzzled by the babys bizarre symptoms, which didnt make any sense until physicians, finally, ordered the right blood test.

What they learned was that Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the bubble boy disease. Caused by a mutated gene on the X chromosome, and almost always limited to males, a baby born with X-linked SCID, or SCID-X1, lacks a working immune system (hence the unusual reaction to vaccination). The bubble boy name is a reference to David Vetter, a Texas child born with SCID-X1 in 1971, who lived in a plastic bubble and ventured out in a NASA-designed suit. He died at 12, but his highly publicized life inspired a 1976 TV movie starring John Travolta.

Today, technological advances in hospitals provide a kind of bubble, protecting SCID-X1 patients with controlled circulation of filtered air. Such safeguards are necessary because a patient exposed to even the most innocuous germs can acquire infections that turn deadly. As soon as Omarion tested positive for the disorder, an ambulance carried him to Cincinnati Childrens Hospital in nearby Ohio and placed him in isolation, where he remained for the next few months. I had no idea what would happen to him, his mother recalled.

Approximately one in 40,000 to 100,000 infants is born with SCID, according to the Centers for Disease Control and Prevention. Only about 20 to 50 new cases of the SCID-X1 mutationwhich accounts for about half of all SCID casesappear in the United States each year. For years, the best treatments for SCID-X1 have been bone marrow or blood stem cell transplantations from a matched sibling donor. But fewer than 20 percent of patients have had this option. And Omarion, an only child, was not among them.

As it happened, medical scientists at St. Jude Childrens Research Hospital in Memphis, Tennessee, were then developing a bold new procedure. The strategy: introduce a normal copy of the faulty gene, designated IL2RG, into a patients own stem cells, which then go on to produce the immune system components needed to fight infection. Simpson enrolled Omarion in the clinical study and Cincinnati Childrens Hospital arranged a private jet to transport her and her son to the research hospital, where they stayed for five months.

St. Jude wasnt the first to try gene therapy for SCID-X1. Nearly 20 years ago, researchers in France reported successfully reconditioning immune systems in SCID-X1 patients using a particular virus to deliver the correct gene to cells. But when a quarter of the patients in that study developed leukemia, because the modified virus also disrupted the functioning of normal genes, the study was halted and scientists interested in gene therapy for the disorder hit the brakes.

At St. Jude, experts led by the late Brian Sorrentino, a hematologist and gene therapy researcher, set out to engineer a virus delivery vehicle that wouldnt have side effects. They started with a modified HIV vector emptied of the virus and its original contents, and filled it with a normal copy of the IL2RG gene. They engineered this vector to include insulators to prevent the vector from disturbing other genes once it integrated into the human genome. The goal was to insert the gene into stem cells that had come from the patients own bone marrow, and those cells would then go on to produce working immune system cells. It was crucial for the viral vector to not deliver the gene to other kinds of cellsand thats what the researchers observed. After gene therapy, for example, brain cells do not have a correct copy of the gene, explained Stephen Gottschalk, who chairs St. Judes Department of Bone Marrow Transplantation and Cellular Therapy.

In the experimental treatment, infants received their re-engineered stem cells just 12 days after some of their bone marrow was obtained. They went through a two-day, low-dose course of chemotherapy, which made room for the engineered cells to grow. Within four months, some of the babies were able to fight infections on their own. All eight of the initial research subjects left the hospital with a healthy immune system. The remarkably positive results made news headlines after being published this past April in the New England Journal of Medicine. Experimental gene therapy frees bubble boy babies from life of isolation, the journal Nature trumpeted.

So far, the children who participated in that study are thriving, and so are several other babies who received the treatmentincluding Omarion. As a physician and a mom, I couldnt ask for anything better, said Ewelina Mamcarz, lead author of the journal article and first-time mother to a toddler nearly the same age as Omarion. The children in the study are now playing outside and attending day care, reaching milestones just like my daughter, Mamcarz says. Theyre no different. Mamcarz, who is from Poland, came to the United States to train as a pediatric hematologist-oncologist and joined St. Jude six years ago.

Other medical centers are pursuing the treatment. The University of California, San Francisco Benioff Childrens Hospital is currently treating infant patients, and Seattle Childrens Hospital is poised to do the same. Moreover, the National Institutes of Health has seen success in applying the gene therapy to older patients, ages 3 to 37. Those participants had previously received bone marrow transplants from partially matched donors, but theyd been living with complications.

In the highly technical world of medicine today, it takes teamwork to achieve a breakthrough, and as many as 150 peoplephysicians, nurses, regulators, researchers, transplant coordinators and othersplayed a role in this one.

Sorrentino died in November 2018, but hed lived long enough to celebrate the trial results. In the early 90s, we thought gene therapy would revolutionize medicine, but it was kind of too early, said Gottschalk, who began his career in Germany. Now, nearly 30 years later, we understand the technology better, and its really starting to have a great impact. We can now develop very precise medicine, with very limited side effects. Gottschalk, who arrived at St. Jude a month before Sorrentinos diagnosis, now oversees the hospitals SCID-X1 research. Its very, very gratifying to be involved, he said.

For now the SCID-X1 gene therapy remains experimental. But with additional trials and continued monitoring of patients, St. Jude hopes that the therapy will earn Food and Drug Administration approval as a treatment within five years.

Simpson, for her part, is already convinced that the therapy can work wonders: Her son doesnt live in a bubble or, for that matter, in a hospital. He can play barefoot in the dirt with other kids, whatever he wants, because his immune system is normal like any other kid, she said. I wish there were better words than thank you.

Continue reading here:
These Scientists May Have Found a Cure for 'Bubble Boy' Disease - Smithsonian

In my view, few investment sectors are as frustrating as the pharmaceutical industry. One moment, you could be riding high on bullish momentum. The next, you could be staring at unfathomable losses. For stakeholders of Constellation Pharmaceuticals (NASDAQ:CNST), though, theyre enjoying the positive end of this dynamic. Year to date, CNST stock is up a blistering 846%.

Source: Shutterstock

Most of these bonkers gains came within the last two months. Since the beginning of October, Constellation Pharmaceuticals stock has jumped nearly 400%. And in this month alone, CNST is up over 68%. Seemingly, this company has no downside, inspiring others to jump aboard this extreme momentum name. Should you follow suit?

Unlike other speculative gambles, a fundamental case exists for the massive skyrocketing of CNST stock. Among the underlying companys therapies is an experimental drug called CPI-0610, a treatment for myelofibrosis. According to pharma giant Celgenes (NASDAQ:CELG) website, myelofibrosis is a rare blood cancer. Only 5,000 people in the U.S. are diagnosed with the illness each year.

Further, myelofibrosis starts in the stem cells of the bone marrow, leading to the production of faulty blood cells. Prior efforts in treating this illness have not produced substantive results. However, Constellations CPI-0610 has performed exceptionally well in a phase II study; hence, the massive surge in Constellation Pharmaceuticals stock.

In fact, all four patients that participated in the study responded positively to the drug. Because of the positive data that came from the clinical trial, Constellation will expand the study to include more patients. This, of course, suggests supreme confidence in the CPI-0610 therapy, and that could ultimately represent a paradigm-shifting breakthrough.

Still, I think you should consider the long road ahead before jumping aboard CNST stock.

By their very nature, rare diseases are difficult to address. And among this class of debilitating conditions, myelofibrosis is particularly nasty. According to Dr. Ruben Mesa, myelofibrosis is a variable disease. This means that medical doctors must apply custom-tailored treatments for different patients.

Thus, while Constellation may have won the initial round in its Phase II study, the real work is coming ahead. With many more test subjects, the chances that CPI-0610 could be considered ineffective or even adverse jump significantly.

In other words, the enthusiasm were seeing now with CNST stock could quickly go the other way.

Theres also the little matter of the economics and politics of addressing myelofibrosis. As you might imagine, combating rare diseases without financial incentives wouldnt make much economic sense. But the Orphan Drug Act, passed in 1983, encouraged pharmaceuticals to address rare diseases through various incentive programs.

Unfortunately, like anything involving government action, good intentions gave way to hellish results. Pharmaceuticals gamed the system the Orphan Drug Act created, pocketing massive profits for rare-disease therapies. Since the patients had no recourse in this monopolized environment, they (and their insurance companies) foot the bill.

Underlining the current bullish thesis for CNST stock is the idea that Constellation will become the only viable myelofibrosis player. Celgene is trying but is coming up short. Essentially, Constellation can charge what they want for their drug if theyre successful.

But even if they are successful and thats a huge if the political environment for price-gouging pharmaceuticals is extremely unfavorable.

No matter how great a scientific achievement Constellation has made, diving into Constellation Pharmaceuticals stock seems risky. With shares gaining 400% in the past month and a half, most of the good news is surely baked in.

Of course, we could hear even better results once the company expands its myelofibrosis study. But that too is a risky perspective.

For those who are not familiar with the pharmaceutical industry, I highly recommend reading Dr. Mario Beauregards book Brain Wars. Among the many topics that Dr. Beauregard covers, a central motif is the mysteries of the mind. Compelling evidence indicates that our mental state can generate healing.

But a flipside to this concept is that an alarming number of pharmaceuticals fail the placebo test; that is, many if not most drugs are no more effective than patients belief in their efficacy.

Soon, well see how good CPI-0610 really is. For those that cant handle extreme price swings, you should stay away from Constellation Pharmaceuticals stock.

As of this writing, Josh Enomoto did not hold a position in any of the aforementioned securities.

Originally posted here:
CNST Stock Is Particularly Risky After Its Recent Run - Investorplace.com

Autologous stem cell and non-stem cell based therapiesinvolve an individuals cell to be cultured and then re-introduced to the donors body. These therapies do not use foreign organism cells and are therefore free from HLA incompatibility, disease transmission, and immune reactions.Increasing demand for the new therapies in the field of regenerative medicine is directly facilitating the growth of autologous stem cell and non-stem cell based therapies market. Furthermore, since the risk to transplantation surgeries is significantly reduced in these therapies, they are increasingly being preferred for treatment of bone marrow diseases, aplastic anemia, multiple myeloma, non-Hodgkins lymphoma, Hodgkins lymphoma, Parkinsons disease, thalassemia, and diabetes.

Moreover, rising incidents of cancer, diabetes and cardiovascular diseases along with growing geriatric population is another factor attributed for its high growth. However, side-effects of autologous stem cell and non-stem cell based therapies such as nausea, infection, hair loss, vomiting, diarrhea, etc. are expected to affect the market to an extent. High cost is another factor that can act as challenge to autologous stem cell and non-stem cell based therapies market. In spite of this, less risk post transplantation surgeries and favorable tax reimbursement policies are anticipated to reduce the impact of these limitation during the forecast period.Autologous stem cell and non-stem cell based therapies market can be segmented on the basis of application, end-user, and region.

In terms of application, the autologous stem cell and non-stem cell based therapies market can be segmented into blood pressure (BP) monitoring devices, intracranial pressure (ICP) monitoring devices, and pulmonary pressure monitoring devices.

In terms of end-user, the market can be segmented into ambulatory surgical center and hospitals. By region, the market can be segmented into North America, Europe, Asia Pacific, Middle East and Africa and South America. Amongst all, Asia Pacific is anticipated to be the most attractive market owing to favorable reimbursement policies in the region.The players operating in autologous stem cell and non-stem cell based therapies market are limited. They are consistently involved in research and development activities for product development to keep up with the growing competition, thereby aiding the growth of autologous stem cell and non-stem cell based therapies market across the world.

The major players operating in autologous stem cell and non-stem cell based therapies market are Regennex, Antria(Cro), Bioheart, Orgenesis Inc., Virxys corporation , Dendreon Corporation, Tigenix, Georgia Health Sciences University, Neostem Inc, Genesis Biopharma, Brainstorm Cell Therapeutics, Tengion Inc., Fibrocell Science Inc., Opexa Therapeutics Inc, Regeneus Ltd, and Cytori Inc., among others.

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Growth of Autologous Stem Cell And Non Stem Cell Based Therapies Market projected to amplify during 2026 - Crypto News Byte

Bone marrow is soft, spongy tissue within some bones, including those in the hips and thighs. People with certain blood-related conditions benefit from a transplant that replaces damaged cells with healthy cells, possibly from a donor.

Bone marrow transplants can be lifesaving for people with conditions such as lymphoma or leukemia, or when intensive cancer treatment has damaged blood cells.

This type of transplant can be an intensive procedure, and recovery can take a long time.

Here, we provide an overview of bone marrow transplants, including their uses, risks, and recovery.

Bone marrow contains stem cells. In healthy people, stem cells in bone marrow help create:

If a medical condition such as one that damages the blood or immune system prevents the body from creating healthy blood cells, a person may need a bone marrow transplant.

A person with any of the following conditions may be a candidate for a bone marrow transplant:

There are three types of bone marrow transplant, based on where the healthy bone marrow cells come from.

In many cases, the donor is a close family member, such as a sibling or parent. The medical name for this is an allogenic transplant.

Transplants are more likely to be effective if the donated stem cells have a similar genetic makeup to the person's own stem cells.

If a close family member is not available, the doctor will search a registry of donors to find the closest match. While an exact match is best, advances in transplant procedures are making it possible to use donors who are not an exact match.

In a procedure called an autologous transplant, the doctor will take healthy blood stem cells from the person being treated and replace these cells later, after removing any damaged cells in the sample.

In an umbilical cord transplant, also called a cord transplant, doctors use immature stem cells from the umbilical cord following a baby's birth. Unlike cells from an adult donor, the cells from an umbilical cord do not need to be as close a genetic match.

Before a bone marrow transplant, the doctor will run tests to determine the best type of procedure. They will then locate an appropriate donor, if necessary.

If they can use the person's own cells, they will collect the cells in advance and store them safely in a freezer until the transplant.

The person will then undergo other treatment, which may involve chemotherapy, radiation, or a combination of the two.

These procedures typically destroy bone marrow cells as well as cancer cells. Chemotherapy and radiation also suppress the immune system, helping to prevent it from rejecting a bone marrow transplant.

While preparing for the transplant, the person may need to stay in the hospital for 12 weeks. During this time, a healthcare professional will insert a small tube into one of the person's larger veins.

Through the tube, the person will receive medication that destroys any abnormal stem cells and weakens the immune system to prevent it from rejecting the healthy transplanted cells.

Before entering the hospital, it is a good idea to arrange:

A bone marrow transplant is not surgery. It is similar to a blood transfusion.

If a donor is involved, they will provide the stem cells well in advance of the procedure. If the transplant involves the person's own cells, the healthcare facility will keep the cells in storage.

The transplant typically takes place in several sessions over several days. Staggering the introduction of cells in this way gives them the best chance of integrating with the body.

The healthcare team may also use the tube to introduce liquids such as blood, nutrients, and medications to help fight infection or encourage the growth of bone marrow. The combination depends on the body's response to treatment.

The procedure will temporarily compromise the person's immune system, making them very susceptible to infection. Most hospitals have a dedicated, isolated space for people undergoing bone marrow transplants to help reduce their risk of infection.

After the last session, the doctor will continue to check the blood each day to determine how well the transplant has worked. They will test whether new cells are beginning to grow in bone marrow.

If a person's white blood cell count starts to rise, it indicates that the body is starting to create its own blood, indicating that the transplant has been successful.

The amount of time that it takes for the body to recover depends on:

Many other factors can affect recovery, including:

Some people are able to leave the hospital soon after the transplant, while others need to stay for several weeks or months.

The medical team will continue to monitor the person's recovery for up to 1 year. Some people find that effects of the transplant remain for life.

A bone marrow transplant is a major medical procedure. There is a high risk of complications during and after it.

The likelihood of developing complications depends on various factors, including:

Below are some of the more common complications that people who receive bone marrow transplants experience:

Some people die as a result of complications from bone marrow transplants.

A person who receives a bone marrow transplant may also experience reactions that can follow any medical procedure, including:

The body's response to a bone marrow transplant varies greatly from person to person. Factors such as age, overall health, and the reason for the transplant can all affect a person's long term outlook.

If a person receives a bone marrow transplant to treat cancer, their outlook depends, in part, on how far the cancer has spread. Cancer that has spread far from its origin, for example, responds less well to treatment.

According to the National Marrow Donor Program, the 1-year survival rate among people who have received transplants from unrelated donors increased from 42% to 60% over about the past 5 years.

A bone marrow transplant is a major medical procedure that requires preparation. This involves determining the best type of transplant, finding a donor, if necessary, and preparing for a lengthy hospital stay.

The time that it takes for the body to recover from a transplant varies, depending on factors such as a person's age and overall health and the reason for the transplant.

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Bone marrow transplant: What it is, uses, risks, and recovery - Medical News Today

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A 25-year-old Redding, Connecticut woman meets the Arizona man who was battling deadly Acute Lymphoblastic Leukemia (ALL) until she saved his life by donating her bone marrow.

Jennie Bunce joined Gift of Life Marrow Registry through a sorority swab drive at North Carolinas High Point University in 2016. "I can remember being like 13 or 14 years old during some school bucket list project. On there was save a life and I got to cross it off so thats pretty cool."

Her life-saving match-- 33-year-old father of six from Mesa, Mark Roser. Roser learned he had ALL after breaking a hip and feeling increasingly weak in 2018.

He needed a bone marrow transplant to survive. He says, "When they discovered it, 94% of my blood cells basically contaminated, so I was really at the final deadline."

Gift of Life Marrow Registry matched the Jennie to Mark with months.

The pair met for the first time at Boca Oyster Bar in Bridgeport in October. Mark says, " I feel great. Im much more positive between work and family. My priorities have completely changed. Time with the kids, time with my wife, just being there for them instead of working so much... I treasure every moment with them now."

According to the gift of Life marrow registry website: "Blood cancer is an umbrella term for cancers that affect the blood, bone marrow and lymphatic system. In most blood cancers, normal blood cell development is interrupted by uncontrolled growth of abnormal blood cells. The abnormal blood cells can prevent blood from fighting off infection or preventing uncontrolled bleeding.

Unfortunately, blood cancer can strike any one of us at any time. Approximately every three minutes, a child or adult in the United States is diagnosed with a type of blood cancer. Thats 360 people a day, 130,000 people a year.

There are three main types of blood cancers: Leukemia, cancer that is found in your blood and bone marrow; Lymphoma, blood cancer that affects the lymphatic system; and Myeloma, blood cancer that specifically targets your plasma cells.

For many, there is hope of a cure through a bone marrow or peripheral blood stem cell transplant. Today, transplantation, of healthy stem cells donated by related and unrelated volunteers, offers hope to many patients suffering from these sometimes deadly diseases.

Advances in transplantation have made this procedure a reality for thousands who are alive today because a stranger gave them the Gift of Life!."

check out: https://www.giftoflife.org to learn more and even register for a swab kit and become a donor yourself.

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Redding woman donates bone marrow, saves life of a father - FOX61 Hartford

November 15 2019, 12:01am,The Times

Al Murray

Its unfashionable to say so these days but we live in an age of miracles. Or at least potential miracles. We all have the opportunity to save someones life and you dont have rush into a burning building or a fast flowing river to do it.

My nephew Finley is six. He is, I think, like a lot of six-year-old boys, he likes trains and tanks and planes, hes into playing football and swimming, and hes almost completely beguiled by video games. So far so normal. He is, however, unlucky enough to be one of the 12 children each year who suffer from juvenile myelomonocytic leukaemia (JMML).

JMML is a rare blood cancer and as difficult to defeat as it is rare. Its sort of

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We live in an age of miracles. And my nephew needs one - The Times

Illustration by Adam De Souza

First Person is a daily personal piece submitted by readers. Have a story to tell? See our guidelines at tgam.ca/essayguide.

A few days after my stem cell transplant this year, a young cleaner entered my hospital room to disinfect and swab. Broad faced and friendly, she saw me lying in bed reading a book.

Do you like reading, she asked? Well, I have the book for you. It is called Fifty Shades of Grey. Its porno!

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That last part was whispered behind a cupped hand, as she grinned and then giggled. For good measure, she also recommended the teen vampire series Twilight.

Once shed left I laughed out loud in a way I hadnt done for days, weeks in fact. When you have cancer, these moments are golden.

Over the last year I have spent months in hospitals, being infused with chemotherapy that laid me low and then undergoing a risky transplant of stem cells from a heroic unknown donor. During this long period of remission and recovery, I have valued every opportunity to smile, to breathe and to feel hope. Much of this sense of being fully alive has come from the kindness of others.

The transplant had made me feel very sick and there was a point at which I was terrified of dying. I asked the hospital staff for a spiritual adviser and the next day a Buddhist monk came to visit me. I didnt expect this, but his calm face and compassionate manner brought me peace. He read me poems for meditation, encouraged deep breathing, and assured me that all emotions in illness are human expressions of identity and not to be judged or feared. His gentleness was echoed two days later, when a nurse with the loveliest face I had ever seen knelt down next to my bed, held my hand, and reassured me I would be okay.

Day by day, my son, his girlfriend, and my husband encouraged and supported me, too, even when I could barely hold up my head or speak without tears. My 21-year-old son sat with me through many painful procedures, setting his phone to play Bachs Brandenburg Concertos, squeezing my hand, looking into my face, loving me and giving me strength I didnt think I had.

I was diagnosed with acute myeloid leukemia in February 2019; before that fateful month I was a modern German historian teaching university students on the Weimar Republic, Nazism and the Holocaust. There were days when I had wept and raged with my students over the historical accounts of Nazi inhumanity, barbarity and chilling callousness inflicted upon innocent civilians, especially the Jews. I have often questioned whether human nature is fundamentally selfish, violent and nasty. Right now, in this world of hateful populism and climate devastation, I ask these questions even more. But since I became sick, the kindness, indeed the goodness, of other people has been a constant companion to me. I have been overwhelmed by the extraordinary outpouring of support and concern from so many. Compassion, care, affection, hope all have been expressed to me by family, friends, students and colleagues. Blood drives were organized in my name, and students asked me if they could be tested as a possible bone-marrow donor. My sister (who hates medical procedures) underwent several tests to see if she could be a sibling transplant. One colleague even offered me the umbilical blood he had saved from his three children. (Ultimately the hospital found a donor from an international registry.)

Friends and family kept in touch or visited despite the long drives to the two hospitals where I received treatment. Two of my girlfriends texted me every day, sending love, inspiration and photos of flowers. From other well-wishers I received quilts and artwork and shawls, books and lotion and lip balm. I read notes and e-mails that told me I was not alone, that love surrounded me and would lift me up. Prayers were said for me in Protestant, Catholic, Unitarian, Muslim and Jewish places of worship. Students sent me good luck charms, including a chemo bear (it worked! I went into remission). Money was donated in a go-fund-me campaign to help with the costs of travel and accommodation to cancer centres. Strangers (friends of friends) offered their homes at the times when we couldnt find accommodation. Delicious meals were dropped off at my home or brought to the hospitals: lentil soups, macaroni and cheese, banana bread and smoothies, all preventing me from having to imbibe those horrible meal-replacement drinks or the cafeteria food. Cancer patients came to see me and shared their experiences and wisdom. A quietly stoic man in his 40s with Stage 4 colorectal cancer expressed hope in the advances in cancer treatment; another inspirational friend with breast cancer revealed she had undergone over 100 chemo treatments and still managed to propel her bike in the annual Ride to Conquer Cancer. Other leukemia patients in my wards became friends and sources of enormous support. My sister-in-law, a liver transplant survivor, understood my physical and emotional pain and talked me through several hard times. On the stranger than fiction level, old boyfriends and ex-friends reappeared, expressing their love and sending me cards or messages that brought tears to my eyes. At the same time high-school and university pals from my ancient past got in touch and told me to hang in there!

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I got through the worst days because of the superb doctors and nurses, the donor who gave her or his stem cells, and our excellent health-care system. But I also made it this far because I did not feel alone. I was constantly reminded that I am loved and that I have so much to live for. In the arduous world of my cancer treatment, the face of compassion has appeared so many times and in such beautiful ways that I now place much more faith in the goodness of human nature because I have seen that many of us will care for each other, especially in hard times.

I may not decide to read Fifty Shades of Grey, but I love that this young woman wanted to suggest something to make me forget the cancer and feel better. And, really, because of her and the support that surrounded me, I did.

Carolyn Kay lives in Peterborough, Ont.

Excerpt from:
Im grateful for the kindness of strangers in my cancer recovery - The Globe and Mail

Transplant strategies for AML

Today, the only curative approach to treat patients with AML is the administration of high-dose chemotherapy followed by allogeneic HSCT (allo-SCT). Although autologous HSCT (auto-SCT) may still be an option for certain patients with favorable or intermediate risk AML, its use has been debated due to the fact that AML is a blood and bone marrow malignancy, thus transplantation with the patients own cells runs the risk of giving back some of the patients leukemia cells.1 In contrast, during the process of allo-SCT, cells from a donor are infused. This provides an additional benefit, called the graft-versus-leukemia or tumor (GvL or GvT) effect, whereby the donor immune cells have the potential of recognising and eradicating remaining leukemia cells in the host, thus reducing the risk of relapse.2

Various donors can be used for an allo-SCT, while the best stem cell source remains to be from a human leukocyte antigen (HLA)-matched sibling donor (MSD). However, in approximately 70% of the cases such donors are unavailable, leaving the next best option of an HLA-matched unrelated donor (MUD).3 For patients where MSD or MUD are unavailable, a partially HLA-matched related donor can be used. This is referred to as haploidentical transplant (HD) and is usually a 50% HLA-match. In the past, HD has been associated with a slow immune reconstitution and high mortality from infections. Today, the use of post-transplant management treatments, like post-transplant cyclophosphamide (PTCy), reduces mortality and has made HD a viable option for patients with AML. This was further discussed by Arnon Nagler in his interview4 with the AML Global Portal (AGP) during the 2019 European Society for Blood and Marrow Transplantation (EBMT) meeting (video below). Nevertheless, HD still leads to inferior outcomes when compared to MSD in patients with AML (read AGP article here). Other donor types include cells from umbilical cord (read AGP article here) or from HLA-mismatched unrelated donors (MMUD).2 The impact of donor type on the outcomes of allo-SCT has recently been explored in an article by the AGP here. The authors of the study concluded that the traditional hierarchy of donors (MSD, MUD, and then others) remains true in patients with AML and should be used as a treatment algorithm.

VIDEO INTERVIEW: EBMT 2019 | Haploidentical hematopoietic transplantation: current status and future perspectives

How to choose the right patients for transplantation

Choosing the right patient to receive transplantation following chemotherapy is crucial for maximising outcomes and reducing the risk of relapse and toxicity. As mentioned by Uwe Platzbecker in his AGP interview during the 2019 EBMT meeting (video below), there are two main considerations when choosing the right candidate for allo-SCT:

According to the ELN guidelines, patients are classified as favorable-risk, intermediate-risk, or poor-risk depending on the possibility of disease relapse. Patients with favorable-risk are usually not considered for allo-SCT after achieving their first complete remission (CR1), as the risk of toxicity and serious side effects outweighs the potential benefit from allo-SCT. For these patients, auto-SCT instead of chemotherapy after CR1 could be beneficial (read AGP article here). On the contrary, allo-SCT at CR1 is a common strategy for poor-risk patients with AML. In the case of intermediate-risk patients (the majority of patients with AML), the most suitable treatment option is less clear.6 Due to the high relapse rates seen in AML, allo-SCT has also been considered as a potential treatment strategy in second remission (CR2), although outcome is inferior compared to allo-SCT performed in CR1.7 In a recent study, summarized here by the AGP, it seems that myeloablative conditioning (MAC) and reduced intensity conditioning (RIC) lead to similar outcomes after allo-SCT in CR2, however more prospective trials are needed to tailor them for maximum efficacy and minimum toxicity.7 To date, it is evident that clinical decisions to perform transplantation need to be made on an individual basis. Recently, measurable residual disease (MRD) as a marker for disease severity and relapse risk has emerged as an important factor that can guide treatment decisions in the context of HSCT and has been reviewed in depth here by the AGP.

VIDEO INTERVIEW: EBMT 2019 | Considerations for transplantation in AML

Post-transplant issues & how to tackle them

Regardless of the advances in the transplantation field, allo-SCTs are associated with two main post-transplant issues: disease relapse and graft-versus-host disease (GvHD).

There is still a considerable number of patients that relapse after HSCT. At the moment, the best strategies to decrease the risk of post-transplantation relapse include:

Such agents include the use of FMS-like tyrosine kinase-3 (FTL3) inhibitors that are shown to delay disease relapse and to potentiate the GvL effect in patients with FLT3 mutations after allo-SCT.7 Multiple pre-clinical and clinical trials are currently underway to examine the efficacy of other targeted inhibitors, like sorafenib, lestaurtinib, sunitinib, tandutinib, quizartinib, and midostaurin, amongst others.8 Another drug that has been shown to prevent disease relapse and to potentially increase the GvL effect is azacitidine. This is a hypomethylating agent that is currently used as a safe and effective prophylactic therapy in high-risk patients following allo-SCT (read AGP article here).9

Charlie Craddock provided an extensive presentation on the strategies for GvL effect optimization at the 2019 EBMT meeting (see full article on the AGP here):

VIDEO INTERVIEW: American Society of Clinical Oncology (ASCO) 2019 | Who should get azacitidine after transplant?

GvHD remains a major post-transplant challenge, occurring when transplanted donor cells start attacking host cells and tissues.10 There are two main strategies used today to prevent GvHD:

In a clinical trial, PTCy has shown superior outcomes when compared to ATG in patients undergoing HD transplant, leading to improved overall survival, leukemia-free survival, and GvHD-relapse free survival. The results of this study were discussed by Arnon Nagler in his interview with the AGP at European Hematology Association (EHA) 2019. A comprehensive review on available treatments for GvHD prophylaxis and their efficacies has been published here by our GvHD Hub.

VIDEO INTERVIEW: EHA 2019 | Should we use PTCy or ATG as GvHD prophylaxis in haploidentical stem cell transplantation?

In patients who develop severe GvHD, systemic administration of steroids remains the first choice of treatment. Treating GvHD is considered by many as a double-edged sword, since on one side it is necessary, but on the other hand it may hinder the GvL effect, thus contributing to potential disease relapse. Further research is needed to clarify the role of GvHD treatment on the GvL effect and to establish the best agent to treat GvHD without hindering the benefits of graft transplantation to the host. Some patients do not respond to post allo-SCT corticosteroids and are classified as steroid-refractory GvHD patients. These patients have a high mortality rate after allo-SCT with a 1-year survival between 30-35%. Many novel approaches are being tested for these patients with the Janus kinase 1/2 (JAK1/2) inhibitor, ruxolitinib, and the Brutons tyrosine kinase (BTK) inhibitor, ibrutinib, being recently approved by the Food and Drug Administration (FDA) for steroid-refractory GvHD.11,12

VIDEO INTERVIEW: EHA 2019 | What are the current treatment recommendations for acute GvHD and the promotion of the GvL effect?

Can the new treatments for AML reduce the need for transplantation?

With the recent therapeutic advances in the field of AML, one major question arises: Can these advances in diagnostics and new therapies replace allo-SCT? During the 1st National Cancer Research Institute (NCRI) AML academy meeting, AGP was pleased to film the headline debate on recently licensed drugs versus recent advances in transplantation, which can be accessed here. Although an unresolved issue, it is evident that some of the new treatments lack the toxicity associated with allo-SCT and have demonstrated improved survival rates. Moreover, with new diagnostic tools, the identification of the right subgroups of patients who may benefit from a transplant-free and more targeted approach will be feasible. One such novel approach to AML treatment is the use of CAR-T cells. Their use as monotherapy or in combination with allo-SCT for the treatment of relapsed or refractory AML is currently under consideration and of great interest in the field. More details on the potential of CAR-T cell therapy for AML can be found here in a recently published article by the AGP. However, it is too early to say whether these new treatment approaches can replace allo-SCT as a curative approach to treat AML. This topic was discussed by Gert Ossenkoppele in the interview with the AGP shown below.

VIDEO INTERVIEW: EHA 2019 | What is the clinical value of new drugs in AML?

Conclusions

Despite the curative potential conferred by allo-SCT in patients with AML, there is still a high risk of non-relapse mortality (mostly due to severe GvHD) in addition to the risk of relapse associated with transplantation. This warrants the need for the development of either novel management and prophylactic therapies that can improve post-transplantation outcomes or of transplantation-free approaches for the treatment of AML. With numerous clinical trials underway with novel targeted agents as monotherapy or in combination, the future of AML treatment starts to look more promising.

Read the rest here:
An introduction to the use of transplantation for the treatment of AML - AML Global Portal

SHANGHAI and SUZHOU, China, Nov. 15, 2019 /PRNewswire/ --Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell therapy company, today announced five presentations to be delivered at the upcoming American Society of Hematology (ASH) Annual Meeting in Orlando, Florida, held from December 7-10.

The presentations centre on Gracell's breakthrough FasTCARtechnology, and other two platform technology in four product categories used in the treatment of hematological malignancies, each with well-defined objectives, including:

The four product candidates are currently being studied in ongoing phase I clinical trials conducted by Gracell, Hebei Yanda Lu Daopei Hospital, and Xinqiao Hospital of AMU, and six other hospitals nationwide in China.

"These clinical studies demonstrated Gracell's product development strategy and strong capabilities to bring multiple novel therapies through clinical investigations," said Dr. William CAO, CEO of Gracell. "These invaluable data provides guidance for and enhance our confidence in pipeline selection."

Oral presentations:

A Feasibility and Safety Study of a New CD19-Directed Fast CAR-T Therapy for Refractory and Relapsed B cell Acute Lymphoblastic LeukemiaAbstract #825Session Name: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Therapeutics StrategiesPresenter: Peihua Lu, M.D., Hebei Yanda Lu Daopei HospitalLocation: Orange County Convention Center, Tangerine 1 (WF1), Level 2Time: 5:00 pm, Monday, December 9, 2019https://ash.confex.com/ash/2019/webprogram/Paper121751.html

Anti-CD19/CD22 Dual CAR-T Therapy for Refractory and Relapsed B-Cell Acute Lymphoblastic LeukemiaAbstract #284Session Name: 612. Acute Lymphoblastic Leukemia: Clinical Studies: Novel TherapiesPresenter: Peihua Lu, M.D., Hebei Yanda Lu Daopei HospitalLocation: Orange County Convention Center, W224, Level 2Time: 4:15pm, Saturday, December 7, 2019https://ash.confex.com/ash/2019/webprogram/Paper126429.html

Poster presentations:

CD19-Directed Fast CART Therapy for Relapsed/Refractory Acute Lymphoblastic Leukemia: From Bench to BedsideAbstract #1340Session Name: 614. Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Poster IPresenter: Cheng Zhang, M.D., Xinqiao Hospital of AMULocation: Orange County Convention Center, Hall B, Level 25:30-7:30 pm, Saturday, December 7, 2019https://ash.confex.com/ash/2019/webprogram/Paper128006.html

A Bcma and CD19 Bispecific CAR-T for Relapsed and Refractory Multiple MyelomaAbstract # 3147Session Name: 653. Myeloma: Therapy, excluding Transplantation: Poster IIPresenter: Hua Zhang, PhD., Gracell Biotechnology Ltd., Shanghai, China, Shanghai, ChinaLocation: Orange County Convention Center, Hall B, Level 26:00 PM-8:00 pm, Sunday, December 8, 2019https://ash.confex.com/ash/2019/webprogram/Paper131056.html

Role of Donor-Derived CD19.CAR-T Cells in Treating Patients That Relapsed after Allogeneic Hematopoietic Stem Cell TransplantationAbstract #4561Session Name: 723. Clinical Allogeneic and Autologous Transplantation: Late Complications and Approaches to Disease Recurrence: Poster IIIPresenter: Cheng Zhang, M.D., Xinqiao Hospital of AMULocation: Orange County Convention Center, Hall B, Level 26:00-8:00 pm, Monday, December 9, 2019https://ash.confex.com/ash/2019/webprogram/Paper128262.html

About FasT CAR-19

FasT CAR-19, or GC007F, is an investigational CD19-targeted CAR-T cell therapy for adolescent and adult patients with refractory or relapsed B-ALL, as well as aggressive non-Hodgkin lymphoma. Thanks to Gracell's patented FasTCAR technology, the bioprocessing time for GC007F has been significantly reduced from two weeks to 24 hours with substantially lower cost. The improved CAR-T cell fitness resulted in superior proliferation capabilities, potency, and extensive bone marrow migration making GC007F a potential best-in-class therapy for refractory or relapsed B-ALL.

About Dual CAR-19-22

Dual CAR-19-22, or GC022, is an investigational CAR-T cell therapy redirected to target CD19 and CD22, in treating patients with CD19+, or/and CD22+ relapsed/refractory B-ALL. A low toxicity with dose-dependent high CR rate including patients who previously treated with CD19 CAR-T cells were observed.

About Dual CAR-BCMA-19

Dual CAR-BCMA-19, or GC012, is an investigational CAR-T cell therapy redirected to target BCMA and CD19, in treating patients with BCMA+, or/and CD19+ relapsed/refractory multiple myeloma. Previous research shows CD19 could express on the myeloma progenitor cells, while BCMA is a well validated target for MM.

About Donor CAR-19

Donor CAR-19, or GC007G, is an investigational CD19 targeted CAR-T cell therapy manufactured in use of donor's lymphocytes. The objective of this study is to further investigate and better understand the safety and efficacy of donor derived CAR-T cells in treatment of relapsed and refractory B-ALL patients.

About B-ALL

B-ALL is a sub-type of acute lymphoblastic leukemia, although rare, is one of the most common forms of cancer in children between the ages of two and five and adults over the age of 50[1]. In 2015, ALL affected around 876,000 people globally and resulted in 110,000 deaths worldwide[2]. It is also the most common cause of cancer and death from cancer among children. ALL is typically treated initially with chemotherapy aimed at bringing about remission. This is then followed by further chemotherapy carried out over several years.

About MM

Myeloma begins when a plasma cell becomes abnormal. The abnormal cell divides to make copies of itself. These abnormal plasma cells are called myeloma cells. In time, myeloma cells collect in the bone marrow. They may damage the solid part of the bone. When myeloma cells collect in several of your bones, the disease is called "multiple myeloma." This disease may also harm other tissues and organs, such as the kidneys. Myeloma cells make antibodies called M proteins and other proteins. These proteins can collect in the blood, urine, and organs[3].

About Gracell

Gracell Biotechnologies Co., Ltd. ("Gracell") is a clinical-stage biopharma company, committed to developing highly reliable and affordable cell gene therapies for cancer. Gracell is dedicated to resolving the remaining challenges in CAR-T, such as high production costs, lengthy manufacturing process, lack of off-the-shelf products, and inefficacy against solid tumors. Led by a group of world-class scientists, Gracell is advancing FasTCAR, TruUCAR (off-the-shelf CAR), Dual CAR and Enhanced CAR-T cell therapies for leukemia, lymphoma, myeloma, and solid tumors.

CONTACT:

Linc HE Associate Director of Business Development sunwei.he@gracellbio.com

Dr. William Cao Founder, Chairman and CEOwilliam.cao@gracellbio.com

SOURCE Gracell

http://www.gracellbio.com

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Gracell Announces Five Presentations at the Annual Meeting of American Society of Hematology (ASH) - PRNewswire