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Be a Bone Marrow Hero – University of Utah Health Care

By daniellenierenberg

Feb 25, 2020 12:00 AM

Every ten minutes, someone passes away from a blood disorder. Thats 148 people a day. There is a way to prevent many of these deathsa bone marrow transplant. DNA matching has the power to help thousands of people waiting for a life-saving bone marrow donation, but this special donor list depends entirely upon the willingness of individuals to sign up. Could your unique DNA hold the match that helps one person live to see tomorrow? Heres how you can find out.

Be The Match is a global hub for bone marrow donor registry working with hundreds of partners to support the transplant community. Signing up is easy online. You provide registration information, receive a kit in the mail, use the DNA swab as directed, and send it back for DNA typing. Your potentially life-saving information is secure and becomes available to specialized doctors around the world.

Even if you arent a match right away, the fact that every three minutes a person is diagnosed with a blood disorder means you could be called at any time to be a hero in someones time of need. Paloma Cariello, MD, MPH, says, Its absolutely a life-saving procedure. Its a new life that people getwe call it a new birthday, and at many hospitals they give it as a new birthday date in their chart. We sing Happy Birthday. Its a big event.

To find a close enough match to help fortify a patients immune system, doctors have to be precise. They first reach out to family, but even then, only 30% of patients find a good match. The odds of finding a match in an unrelated donor can be as low as 18%, especially with minorities.

The need for more individuals of every background cannot be overstated, says University of Utah Health Hematologist Sagar Patel, MD. He emphasizes the need for ethnic minorities to register. Every ethnicity is represented in the pool of patients, so the donor pool likewise needs to be diversified to improve the availability of similar DNA typing.

If a doctor finds you to be a suitable match, they select the ideal method for their patient and prepare you for donation. There are two donation methods: peripheral blood stem cell (PBSC) donation and bone marrow donation. Because every donor is carefully screened and prepared, and because a small amount of fluid is ultimately needed, neither procedure method impacts the performance of your own immune system, says Cariello.

With PBSC retrieval, you receive a stimulant for five days to increase the presence of blood-forming cells in your blood stream. Then a refined process of extraction occurs: Your blood is drawn, a machine collects just the cells the patient needs, and your remaining fluids are safely returned to you. This process can usually be done in one eight-hour session. Most donors report a full recovery within a week to 10 days, but you will be followed-up with until your full recovery.

If the doctor determines that the patient needs bone marrow, your procedure is a bit different. Marrow needs to be drawn from your pelvic bones. It happens in a hospital and under anesthesia, and you will feel no pain as the donation is collected. You can go back to routine activity the same day, and your system fully replenishes within four to six weeks.

Even with thousands of people in need, only about one in 430 donors in the Be The Match system are called in as a match. And the simple processes and expert professional care you receive minimize potential risk. A common side effect is bruising at the procedure sites, and some donors occasionally experience mild pain, fatigue, or dizziness. Reactions related to the use of anesthesia might also occur.

With such little risk, it shouldnt be a question as to whether you sign up, but when. And today is a perfect day. The low odds of finding a cure that these patients face are as extreme as the high rewards that await themand youwhen you make the choice to become a donor. Visit BeTheMatch.org to learn more and to become the one who initiates the miraculous call: We found a match.

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YOUR HEALTH: Knee relief can be found in the womb – WQAD.com

By daniellenierenberg

CHICAGO About 54 million Americans suffer from the aches and pains of arthritis.

Treatments range from pain medications to injections to surgery.

None of it seemed to work for 77-year-old Marty Ciesielczyk.

And it jeopardized something he loved: jogging.

"For me, it's just enjoyable, and if you're not a runner, then you would have no idea what I'm talking about."

But Marty's active lifestyle was in jeopardy when knee pain took over.

"When you got to lay on the floor to get dressed, it's tough."

It happens when there's a loss of cartilage in the joint.

"It's like a tire, and as you slowly lose rubber on the tire, it wears away," explained Dr. Adam Yanke, a surgeon with Midwest Orthopaedics at Rush University.

"You might need to have the tire replaced at some point."

Marty's arthritis was too advanced for a scope procedure but not bad enough for a joint replacement.

So he enrolled in a study testing whether amniotic fluid, which surrounds a growing baby in the uterus, could help his pain.

"Amniotic products come from patients that are having healthy, elective C-sections, and they choose to donate these products at the time of the delivery," said Dr. Yanke.

It's thought to increase tissue healing and lower inflammation.

Doctor-diagnosed arthritisis more common in womenthan in men. Arthritis and other joint disorders are among the five most costly conditions among adults 18 and older.

Your bone marrow makes mesenchymal stem cells, or MSCs. They are known to grow into new tissues, including cartilage.

By gathering these cells and injecting them into the knee joint, the hope is that they will give growth to new cartilage and reduce inflammation.

Marty received a placebo during the study, but then chose to have the amniotic fluid when the study ended.

"I mean I didn't care if it was Pixie dust, as long as my knee was going to feel better."

He went from not being able to get dressed to jogging about a week after having the injection.

"This morning, I ran three, three miles, and I had no problem at all."

Amniotic fluid is also being used to treat ulcers in the eye.

Rush University will be enrolling patients for a larger follow-up study on amniotic fluid for joint pain in the future.

Clinical trialsare still going on and most studies are still early.

A review published in 2016 in BMC Musculoskeletal Disorders concluded that MSC-based therapies offer an "exciting possibility" for treatment, but further studies need to be done on how they can best be used and how well they work.

They are also known to be very expensive.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens atjim.mertens@wqad.comor Marjorie Bekaert Thomas atmthomas@ivanhoe.com.

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD – Yahoo Finance

By daniellenierenberg

NEW YORK, Feb. 24, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that aggregated results from 309 children treated with Ryoncil (remestemcel-L) were presented atthe American Society for Transplantation Cellular Therapy and the Center for International Blood & Bone Marrow Transplant Research (TCT) meeting in Orlando, Florida on February 22. The data showed that treatment with RYONCIL across three separate trials resulted inconsistent treatment responses and survival outcomesinchildren with steroid-refractory acute graft versus host disease (SR-aGVHD).

Key findings and conclusions were:

Mesoblast Chief Medical Officer Dr Fred Grossman said: These aggregated data from three studies demonstrate consistent efficacy and safety of RYONCIL in children suffering from steroid refractory acute graft versus host disease. If approved, RYONCIL has the potential to be an effective and safe therapy to improve survival outcomes in the most vulnerable population of children with severe forms of this disease who can have mortality rates as high as 90 percent.

In January, Mesoblast filed a Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) for RYONCIL for the treatment of children with steroid-refractory aGVHD. The Company has requested Priority Review of the BLA by the FDA under the product candidates existing Fast Track designation. If approved, RYONCIL is expected to be launched in the US in 2020.

About Acute GVHDAcute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing.1 In patients with the most severe form of acute GVHD (Grade C/D or III/IV) mortality is as high as 90% despite optimal institutional standard of care.2,3. There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Ryoncil Mesoblasts lead product candidate, RYONCIL, is an investigational therapy comprising culture- expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in SR- aGVHD by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

References1. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey.2. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology.3. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation.

About MesoblastMesoblast Limited (Nasdaq: MESO; ASX: MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platforms to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblasts proprietary manufacturing process yields industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has filed a Biologics License Application to the United States Food and Drug Administration (FDA) to seek approval of its product candidate Ryoncil (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GvHD). Remestemcel-L is also being developed for other rare diseases. Mesoblast is completing Phase 3 trials for its rexlemestrocel product candidates for advanced heart failure and chronic low back pain. If approved, RYONCIL is expected to be launched in the United States in 2020 for pediatric steroid-refractory acute GVHD. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Story continues

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Mesoblasts Forward-Looking StatementsThis announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about the timing, progress and results of Mesoblasts preclinical and clinical studies; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

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Stem Cell Therapy Market Scope and Opportunities Analysis 2017 2025 – Instant Tech News

By daniellenierenberg

Stem Cell Therapy Market: Snapshot

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

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Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

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Cumberland County family turns to non-FDA approved stem cell treatment to help two-year-old son with cerebral palsy – FOX43.com

By daniellenierenberg

Lance was diagnosed with cerebral palsy a year ago. His family hopes non-FDA approved stem cell treatment for the disease can help him walk and talk.

CAMP HILL, Pa. A family in Cumberland County has turned to stem cells to treat their two-year-old son diagnosed with cerebral palsy. The only problem: stem cell treatment for the disease hasn't been approved by the FDA.

The day he was born, when he wheeled him down the hall and he was only a pound, and I started to cry and said, will he live? And he said, of course Hes only small," said Danielle Maxwell, Lance's mom.

The words, "he's only small," are what Lance's mom and father Rob have lived by since the day he was born. The preemie, born three months early, has been through several surgeries and complications along the way. But, Lance has always been a fighter.

Lance fought so hard just to survive the beginning of life, and come home with us," said Danielle. "And he is just so happy and loving and amazing.

About a year ago, Lance was diagnosed with cerebral palsy. Doctors told his family, he will never walk, talk or take care of himself.

We just dont believe that," said Danielle. "We dont.

Lance receives a lot of different therapies but, his parents did not want to just stop there.

We both overwhelmingly feel, he never gave up, he never gave up on us, he never gave up on himself," said Rob. "So, we owe it to him to give him the opportunity. Its really that simple, he deserves the opportunity."

Danielle began researching stem cell therapies, even speaking to doctors in countries overseas where treatment with stem cells is more readily accessible than in the U.S. The FDA has approved stem cell treatments for some conditions but not cerebral palsy. However, trials to determine the effectiveness of stem cell treatment for the disease are underway.

What weve seen is a small but real appearing improvement in motor function," said Doctor Charles Cox with University of Texas Health in Houston, began a trial in 2013 on the safety and effectiveness of banked cord blood or bone marrow stem cells in children with cerebral palsy, and is now just wrapping up the results from the trial.

The overall results of this study depend if youre a glass half full or half empty kind of person," said Dr. Cox. "It is not a compelling miraculous result. Its not, Oh my God, this child was treated and look at this profound benefit.'"

Because stem cell treatment for cerebral palsy is still in trial phases, it's not approved treatment by the FDA. However, the Maxwells did find a doctor in Harrisburg willing to transfer stem cells from a full-term baby's umbilical cord to Lance. But, since it isn't FDA approved, we were not allowed to be there to show Lance receiving the stem cells. The Maxwells are hopeful following this procedure Lance may someday walk and more importantly be able to communicate with them.

He wants to be involved," said Rob. "You can tell hes trying to communicate he just cant get over that hump. We believe stem cells could be that bridge to help him move a little faster.

Danielle says, it will take about six months to see if the stem cells will have any definitive benefits for Lance. But, already says she's seeing progress. She says Lance is not able to stand on his own.

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Scientists May Have Found A Way To Boost The Body’s Ability To Heal Itself – IFLScience

By daniellenierenberg

Its not just Wolverine that has the ability to rebuild and restore wounded tissue. In fact, we all have a quite remarkable capacity to heal when we suffer an injury, thanks to our ability to produce new stem cells. Obviously, there is a limit to how much damage our bodies can repair, although researchers may have just discovered a way to enhance our powers of restoration by increasing the rate at which these stem cells are generated.

A new study in the journal Regenerative Medicine describes how scientists were able to stimulate the self-repair response of rats in order to rebuild broken spines. Healing similar injuries in humans is currently not possible, and the study authors are hopeful that their technique could one day help people recover from a range of previously untreatable injuries.

Rats in the study were given a cocktail of two drugs, one of which is normally administered during bone marrow transplants while the other is used for bladder control. This caused the rats bone marrow to produce an elevated number of mesenchymal stem cells, which are stem cells that can develop into bone tissue.

As a consequence, enhanced calcium binding was seen at the site of the rats spinal injuries, speeding up the formation of new bone and healing the wounds.

The figure on the right shows the level of healing with no treatment, while the figure on the left shows the effect of the two drugs in combination. The red coloring indicates calcium incorporating into the bone, which is associated with enhanced healing. Image: Imperial College London

We know that when bones break they will heal, and this requires the activation of stem cells in the bone, explained study co-author Sara Rankin in a statement. However, when the damage is severe, there are limits to what the body can do of its own accord.

We hope that by using these existing medications to mobilize stem cells, as we were able to do in rats in our new study, we could potentially call up extra numbers of these stem cells, in order to boost our bodies own ability to mend itself and accelerate the repair process.

Because the drugs involved are already widely used, the researchers are hopeful that human trials can proceed without the need for extensive safety testing. If these trials produce the same results as those seen in rats, then this treatment could help to not only repair spinal injuries, but to speed up the rate at which broken bones heal and even mend damaged tissues in other organs.

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD – BioSpace

By daniellenierenberg

NEW YORK, Feb. 24, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that aggregated results from 309 children treated with Ryoncil (remestemcel-L) were presented atthe American Society for Transplantation Cellular Therapy and the Center for International Blood & Bone Marrow Transplant Research (TCT) meeting in Orlando, Florida on February 22. The data showed that treatment with RYONCIL across three separate trials resulted inconsistent treatment responses and survival outcomesinchildren with steroid-refractory acute graft versus host disease (SR-aGVHD).

Key findings and conclusions were:

Mesoblast Chief Medical Officer Dr Fred Grossman said: These aggregated data from three studies demonstrate consistent efficacy and safety of RYONCIL in children suffering from steroid refractory acute graft versus host disease. If approved, RYONCIL has the potential to be an effective and safe therapy to improve survival outcomes in the most vulnerable population of children with severe forms of this disease who can have mortality rates as high as 90 percent.

In January, Mesoblast filed a Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) for RYONCIL for the treatment of children with steroid-refractory aGVHD. The Company has requested Priority Review of the BLA by the FDA under the product candidates existing Fast Track designation. If approved, RYONCIL is expected to be launched in the US in 2020.

About Acute GVHDAcute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing.1 In patients with the most severe form of acute GVHD (Grade C/D or III/IV) mortality is as high as 90% despite optimal institutional standard of care.2,3. There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Ryoncil Mesoblasts lead product candidate, RYONCIL, is an investigational therapy comprising culture- expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in SR- aGVHD by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

References1. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey.2. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology.3. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation.

About MesoblastMesoblast Limited (Nasdaq: MESO; ASX: MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platforms to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblasts proprietary manufacturing process yields industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has filed a Biologics License Application to the United States Food and Drug Administration (FDA) to seek approval of its product candidate Ryoncil (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GvHD). Remestemcel-L is also being developed for other rare diseases. Mesoblast is completing Phase 3 trials for its rexlemestrocel product candidates for advanced heart failure and chronic low back pain. If approved, RYONCIL is expected to be launched in the United States in 2020 for pediatric steroid-refractory acute GVHD. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Mesoblasts Forward-Looking StatementsThis announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about the timing, progress and results of Mesoblasts preclinical and clinical studies; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD - BioSpace

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New sickle cell disease treatments are helping people live longer and giving them a higher quality of life – PhillyVoice.com

By daniellenierenberg

Treatment for sickle cell disease has come a long way since the 1970s when the life expectancy of people living with it was less than 20 years.

People with sickle cell disease are not only living longer life expectancy is now 42 to 47 years of age but are enjoying a better quality of life, too.

"In the Philadelphia area, there has been great pediatric care for sickle cells disease and because of that people who have it are living very well," said Dr. Farzana Sayani, a hematologist at Penn Medicine.

Sayani is the director of a comprehensive sickle cell program focusing on adults living with the disease. Penn also has an active transition program for youth transitioning from a pediatric institution to adult care.

Sickle cell disease is an inherited red blood cell disorder that affects about 100,000 Americans.It is most often found in people of African or Hispanic descent.About 1 in 365 African-American babies are born with sickle cell disease, according to Sayani.

People who have the disease inherit an abnormal type of hemoglobin in their red blood cells, called Hemoglobin S, from both their mother and father.When only one parent has the hemoglobin S gene, a child will have the sickle cell trait, but usually does not develop the disease. But they may pass it on to their children.

Hemoglobin is the protein in the blood responsible for carrying oxygen to the rest of the body. Hemoglobin S causes red blood cells to become stiff and sickle-shaped. Instead of being round in shape, they look like crescent moons.

Sickle cells are sticky and can bind together, blocking the flow of blood and preventing oxygen from getting where it needs to go in the body. This causes sudden attacks of pain referred to as a pain crisis.

There are severaldifferent types of sickle cell disease.Hemoglobin SS, also known as sickle cell anemia, is the most common and most severe type of sickle cell disease.

Anemia occurs when red blood cells die at a rate faster than the body can replace them. Normal red blood cells generally live for 90 to 120 days. Sickled cells only live for 10 to 20 days. This shorter life-to-death cycle is harder for the body to sustain.

Another form,Hemoglobin SC, is not as severe as sickle cell anemia, but it can still cause significant complications, Sayani said.Other forms include Hemoglobin S0 thalassemia, Hemoglobin S+ thalassemia, Hemoglobin SD and Hemoglobin SE.

Sickle cell disease screening is a mandatory part of newborn screenings in Pennsylvania.

If the screening is positive, the family is informed and plugged into the health care system in order to receive the proper care.

If the disease is not diagnosed at birth, a blood test can confirm it at any age in which symptoms start to surface.

The severity of sickle cell disease can vary.

Each individual is affected differently, making it difficult to predict who will get what complications, Sayani said. That is why a comprehensive sickle cell program is so important.

Early signs include a yellowish tint to the skin or jaundice, fatigue and a painful swelling of the hands and feet.

"Young children with sickle cell disease may be tired, not eat very well and have delayed growth," Sayani said. "They may also develop anemia, be at greater risk of infection and start to experience pain crises."

Acute pain crises, also known as vaso-occlusive crises, can lead to long stays in the hospital to manage the crippling pain. Children with sickle cell disease also tend to experience delayed growth and puberty.

As a person with sickle cell disease grows older, the sickled red blood cells start to affect various organs, bones and joints.

This can lead to acute chest syndrome, which occurs when damaged lung tissues makes it difficult to breathe. Brain complications, including stroke, are possible.People with sickle cell disease are also prone to heart damage, eye problems, and infections like chlamydia, salmonella and staphylococcus. Chronic and acute pain is common.

There are different types of medicine that can help manage sickle cell disease.

Last year, an oral medicine was approved that makes sickle cells less likely to sickle. So was an intravenous medicine that has been shown to reduce pain crises and hospitalizations by 50%. Some people living with sickle cell disease also may need regular blood transfusions.

Hydroxyurea has also been used successfully for many years to reduce pain crises and the need for blood transfusions and hospitalizations.

Currently, blood and bone marrow transplant is the only way to cure the disease. But it is not an option for everyone because of the difficulty of finding a well-matched stem cell donor.

A related donor is best but only about a third of sickle cell patients have a donor that is related and fully-matched, Sayani said.

While these transplants have a 85% or more success rate, they also are associated with significant risks, including organ dysfunction, infection and graft vs. host disease which can be quite debilitating.

Transplants completed in children have the best results, Sayani said. But because of the risks involved, doctors only suggest it for patients with severe forms of the disease.

Early clinical trials with gene therapy are also showing promise, she added.

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Experimental study speeds up bone healing with 2 common medications – New Atlas

By daniellenierenberg

A new proof-of-concept study has found a combination of two drugs, already approved by the FDA for other uses, may boost the release of stem cells from bone marrow and accelerate the healing of broken bones. Only demonstrated in animals at this stage, the researchers suggest clinical trials could progress rapidly considering the drugs have already been demonstrated as safe in humans.

"The body repairs itself all the time, says corresponding author on the study Sara Rankin. We know that when bones break they will heal, and this requires the activation of stem cells in the bone. However, when the damage is severe, there are limits to what the body can do of its own accord.

A great deal of current research is focusing on mesenchymal stem cell (MSC) therapies. MSCs are a type of adult stem cell that can grow into a variety of different cell types including muscle, fat or bone. Many current MSC treatments in development involve extracting a small number from a patient, growing them in laboratory conditions, then injecting them back into the patient.

The new research set out to investigate whether any currently approved drugs can function to mobilize the bodys natural ability in releasing MSCs, with a view on speeding up healing of bone fractures. A study published in the journal npj Regenerative Medicine, describes the testing of two already approved drugs in a rodent spinal injury model.

The two drugs tested were an immunostimulant called Plerixafor, used to stimulate the release of stem cells from bone marrow in cancer patients, and a beta-3 adrenergic agonist developed to help bladder control.

The results suggest the duo of drugs mobilize MSCs into the bloodstream and speed up the process of bone formation and healing by enhancing the binding of calcium to the injury site. Tariq Fellous, first author on the new study, suggests the next step is to investigate whether this drug combination enhances blood MSC levels in human subjects.

We first need to see if these medications release the stem cells in healthy volunteers, before we can then test them in patients with fractures, says Fellous. We have the drugs and know they are safe to use in humans - we just need the funding for the human trials.

The researchers say prior studies have identified circulating MSCs increase in volume following injuries such as burns, bone fractures, and even heart attack. The hypothesis is that the release of MSCs is a physiological process aiding general regeneration following injury, and if circulating numbers of MSCs could be pharmacologically enhanced then a variety of types of tissue regeneration could be accelerated.

It is important to note the current study only examined increases in circulating MSCs and the rate of spine injury healing compared to no drug treatment. The current research offers no indication whether the drug duo influences nerve healing or restores movement.

So, more work is certainly necessary to understand how clinically useful these results actually are. However, as the studys co-first author Andia Redpath notes, this re-purposing of existing medicines to boost stem cell activity is an easier, cheaper, and more efficient way to enhance healing compared to other, more complex and time-consuming, stem cell treatments in development.

Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the bodys own stem cells, using existing drugs, says Redpath. We already know the treatments in our study are safe, its now just a matter of exploring further if they help our bodies heal.

The new study was published in the journal npj Regenerative Medicine.

Source: Imperial College London

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New hope for the healing of spinal column injuries with generally available drugs – The Washington Newsday

By daniellenierenberg

A combination of two commonly available drugs may help the body heal spinal fractures.

Scientists have discovered a combination of two commonly available drugs that could help the body heal spinal fractures.

The early-stage research in rats, by a group of scientists led by Imperial College London, revealed two existing medications can boost the bodys own repair machinery, by triggering the release of stem cells from the bone marrow.

The scientists published their research in the journalnpj Regenerative Medicine.

The team says the two drugs (currently used for bone marrow transplants and bladder control) could be used for different types of bone fractures, including to the spine, hip, and leg, to aid healing after surgery or fractures.

When a person has a disease or an injury, the bone marrow (the spongy tissue within bone) mobilizes different types of stem cells to help repair and regenerate tissue.

This image shows repair of the spine three weeks after surgery. The image on the left is with no drug treatment, with the image on the right (b3+AMD3100) showing the effect of the two drug treatments. The red colour indicates calcium incorporating into the bone, which is associated with enhanced healing. Credit: Imperial College London/Beaumont Health

The new research, involving scientists from Beaumont Health in the U.S, suggests it may be possible to boost the bodys ability to repair itself and speed repair, by using new drug combinations to put the bone marrow into a state of red alert and send specific kinds of stem cells into action.

In the new study, funded by Wellcome, the researchers used drugs to trigger the bone marrow of healthy rats to release mesenchymal stem cells, a type of adult stem cell that can turn into bone, and help repair bone fractures.

Professor Sara Rankin, corresponding author of the study from the National Heart and Lung Institute at Imperial College London, said: The body repairs itself all the time. We know that when bones break they will heal, and this requires the activation of stem cells in the bone. However, when the damage is severe, there are limits to what the body can do of its own accord. We hope that by using these existing medications to mobilize stem cells, as we were able to do in rats in our new study, we could potentially call up extra numbers of these stem cells, in order to boost our bodies own ability to mend itself and accelerate the repair process. Further down the line, our work could lead to new treatments to repair all types of bone fracture.

The two treatments used in the research were a CXCR4 antagonist, used for bone marrow transplants, and a beta-3 adrenergic agonist, that is used for bladder control.

The rats were given a single treatment with the two drugs, which triggered enhanced binding of calcium to the site of bone injury, speeding bone formation and healing.

The researchers stress they did not analyze restoration of movement in the bone, or repair to additional tissue such as nerves.

One of the drugs used in the study was found to trigger fat cells in the bone marrow to release endocannabinoids, which suggests they may have a role in mobilizing the stem cells and thereby promoting healing. However, the researchers add that phytocannabinoids (such as cannabis) would not have the same effect, as they act on the brain rather than the bone marrow.

The researchers say the drug combinations now need to be tested in humans.

Dr. Tariq Fellous, first author of the research from Imperials National Heart and Lung Institute (NHLI) said: We first need to see if these medications release the stem cells in healthy volunteers, before we can then test them in patients with fractures. We have the drugs and know they are safe to use in humans we just need the funding for the human trials.

Dr. Andia Redpath, co-first author from the NHLI, added that repurposing existing medications that help the body heal itself so called Regenerative Pharmacology could have great potential as an efficient and cost-effective approach for a range of diseases. Rather than devising new stem cell treatments from scratch that involve lengthy and expensive trials, our approach harnesses the power of the bodys own stem cells, using existing drugs. We already know the treatments in our study are safe, its now just a matter of exploring further if they help our bodies heal.

This work was funded by Wellcome. Further support was provided by the National Heart and Lung Institute Foundation and the Lumbar Spine Research Society.

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Leukemia: Cancer Of The Blood – Modern Ghana

By daniellenierenberg

DEFINITIONLeukemia is a Cancer of blood forming tissues including bone marrow and the lymphatic system.This type of cancer hinders the body's ability to fight infection, leukemia involves the white blood cells.

CAUSES OF LEUKEMIALeukemia can develop due to a problem with blood cell production.

Several factors have been identified which increase the risk of having the cancer:

A family history of leukemiaSmokingGenetic disorder such as Down syndromeExposure to chemicals such as benzeneExposure to high levels of radiationTYPES OF LEUKEMIALeukemia can be Acute or Chronic. In Acute leukemia, Cancer cell multiply quickly while in Chronic leukemia, the disease progresses slowly and early symptoms may be very mild.

Leukemia can also be classified according to types of cells which are myelogenous leukemia and lymphotic leukemia involving myeloid cells and lymphocytes respectively.

SYMPTOMS OF LEUKEMIAExcessive sweating most especially in the nightFatigue and weaknessUnintentional weight lossFever or chillsFrequent infectionsBleeding easily and bruising easilyEnlargement of the liver or spleenLEUKEMIA IN PREGNANCYLeukemia affects approximately 1 in 10000 pregnancies.Women with leukemia have non-specific symptoms and some of them could also be attributed to pregnancy.

The damage in the fetus is correlated with the time of exposition and the fetus is most vulnerable during organnogenesis phase, Although chemotherapy has effect in the fetus, there are reports of cases with successful pregnancy.

It is necessary to study the relation among chemotherapy, the leukemia and the fetus in long term studies where fetus has been exposed to chemotherapy agents and is reported with normal characteristics at birth.

ENZYMES DEFICIENT IN LEUKEMIABlast cells from 100 cases of Acute leukemia were evaluated for the presence of methylthioadenosine phosphorylase (MTAase), an enzyme important in polyamine metabolism.

Ten cases (10%) had undetectable levels of MTAase activity. A relatively high frequency (38%) of MTAase deficiency was seen in all of T-cell origin.

MTAase deficiency occurs in a wide variety of acute leukemia, that the lack of enzyme activity is specific in malignant cells. The absence of MTAase in some leukemia may be therapeutically exploitable.

TREATMENT OF LEUKEMIALeukemia is usually treated by a hematologist-oncologist. These are doctors who specialize in blood disorders and cancer.

The treatment depends on the type and stage of the cancer, the treatment includes the following:

Chemotherapy uses drugs to kill leukemia cells.Radiation therapy uses high energy radiation to damage leukemia cells and inhibit their growth.

Stem cell transplantation replaces diseased bone marrow with healthy bone marrow.

Biological or immune therapy uses treatments that help your immune system recognize and attack cancer cells.

Targeted therapy uses medication advantages of vulnerabilities in cancer cells.

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Community give child a hero’s homecoming – Thehour.com

By daniellenierenberg

FLORENCE, Ala. (AP) Cole Kelley grinned widely and waved Saturday (Feb. 15) as his head kept swiveling from one direction to the other to take in the amazing view surrounding him.

Hundreds of family members, friends, Mars Hill School classmates and other members of the community lined both sides of the school's parking lot to welcome the 8-year-old home.

The Mars Hill student and his parents, John and Caroline Kelley, had been away from home for 15 months while he has battled a rare disease.

That included a 460-day stay at the National Institutes of Health facility in Bethesda, Maryland, which is one of the few facilities with experience treating the disease dada2, which is a deficiency of the adenosine deaminase 2 (ADA2) enzyme.

According to dada2.org, the disease causes "recurrent strokes, severe systemic inflammation, immune deficiency, and damage to many of the body's tissues and organs."

The community organized the homecoming parade, which included an escort from Florence's police and fire departments, as well as people dressed in superhero outfits.

Along with Cole's classmates, Mars Hill High School cheerleaders joined the crowd, as did the school's football players, who showed up in their jerseys. The long reception line spanned the length of the parking lot coming off Cox Creek Parkway.

The procession made two laps through the parking lot amid loud cheers and flowing tears from the congregation before the family, clearly emotional from the outpouring, drove home.

"It's been amazing," Cole's cousin, Sara Beth Searcy, said while wearing a "Best Day Ever" shirt she bought during a Disney World trip Cole and the family took before his long hospital stay.

"This whole community has supported us from the very beginning," she said. "We could not have gotten through this without this entire community wrapping their arms around us."

Cole classmate Rogan Willingham held a sign that read "God answered our prayers."

"He's prayed, I don't know how many times a day, for Cole," his mother, Ginger Willingham, said.

"I'm glad he's back," Rogan said, adding he looks forward to being able to "play with him and all kinds of stuff."

"I was praying for him a lot," he said.

There were numerous other signs with messages, such as "Sweet Home, Alabama," "Super Cole" and "Welcome home, Buddy."

Emily Stutts, a friend of the family who helped organized the welcome, and fellow member of Jackson Heights Church of Christ, said Cole has been sick since he was 2 months old.

He had a bone transplant at the National Institutes.

"He lived off of having blood transfusions because his body did not make red blood cells," Stutts said. "After a while that became risky because his body can't filter out all the iron. So a bone marrow transplant was the only answer."

He was enrolled in the "Be the Match" bone marrow registry and was a match with a young man from Australia, who had volunteered to join the registry.

"He was able to give the bone marrow and they flew it over to Maryland and did the transplant," Stutts said. "It didn't take initially, and Cole has had three stem-cell transplants, all from the one donation from the Australian man.

"The fact that he's coming home is a miracle, because he had some very, very scary times that they didn't think he would survive. He had zero immune until Christmas day. That day his immune response occurred."

She said John and Caroline often talk about the amazing support they have been receiving back home. A community spaghetti supper, T-shirt sales and a Christmas fundraising event are among methods supporters have used to provide financial assistance for the parents, who have not been able to work during their son's hospitalization.

"The people at the hospital there can't believe the support that they've been given from this community," Stutts said. "They told them they see people from all over the world, but they've never seen the outpouring of love like they saw for the Kelleys.

"They deserve it. They're such good people."

Florence company High Cotton Homes provided assistance in a major way, Stutts said.

"They had to have all new heating and air units and vents put in and a water purification system, all new flooring and all new duct work because of his immune system," she said. "High Cotton took that on, got donations and completely remodeled his room and made it so Cole would have his own bathroom to help with germ prevention."

Cole still is susceptible to sickness and will continue to have medical appointments, Stutts said.

"His liver was damaged from the transplant so he's going to go to Vanderbilt for them to check his liver," she said. "There's still things that need to be addressed, but as far as his bone marrow, that is doing OK."

Stutts said Cole's disease has not kept him down.

"He is full of life," she said. "He's a happy kid. Even though he's been sick, if you had seen him, you would have never know it. He's made a huge impact on the people at the National Institutes for Health because no child's been there that long. That became his family. They had a going-away party at one of the doctor's homes."

The emotions of Saturday's homecoming were obvious among the crowd, many of whom hugged one another and cried.

"The one thing I kept hearing from everybody is it was more emotional than they through it would be," said Ronnie Pannell, family minister at Jackson Heights. It's been a big show of love. Mars Hill loves Cole Kelley, there's no doubt about that.

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Adoption of Hematology Analyzer Reagent Market to Soar Across Top Countries in the Globe Forecast to 2015 to 2021 – Lake Shore Gazette

By daniellenierenberg

Hematology is the branch of medicine which deals with the study, diagnosis, and treatment of blood-related disorders. It diagnoses issues related to white blood cells, red blood cells, platelets, bone marrow, and lymph nodes. Hematology also deals with the liquid portion of blood known as plasma. Some blood-associated diseases are anemia, leukemia, myelofibrosis, blood transfusion, malignant lymphomas, and bone marrow stem cell transplantation. Hematology analyzers, coagulation analyzers, flow cytometers, and slide strainers are some of the hematologic instruments used to analyze blood and blood-related disorders. Hematology analyzers are highly specialized instruments used to count the number of white blood cells, red blood cells, and platelets.

Hematology analyzers are made up of multiple analytical modules with unique sample preparation processes. They assist in the diagnosis of various diseases, such as infections, anemia, viruses, diabetes, genetic problems, and cancer. It also regulates plasma drug level for both therapeutic and illicit drugs. Fully automated hematology analyzers and semi-automated analyzers are the two types of hematology analyzer available based on the type of automation. Fatigue, pale skin, and shortness of breath with exertion are some of the common symptoms of blood disorders. Clinical testing laboratories, hospitals, and research institutions are some of the users of hematology analyzers and reagents.

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Currently, North America dominates the global hematology analyzer & reagent market, followed by Europe. This is due to the regions high disposable income and increasing adoption of automated hematology instruments by diagnostics laboratories in these regions. Asia is expected to be the fastest growing market in the next five years. This is due to the developing healthcare infrastructure and increasing funding towards the development of hematology products in this region. Moreover, rising awareness about better healthcare and a large patient population base are also driving the growth of the hematology analyzer & reagent market in Asia.

Increasing adoption of automated hematology instruments, rising technological advancements, and integration of basic flow-cytometry techniques in modern hematology analyzers are some of the key factors driving the growth of the global hematology analyzer & reagent market. In addition, rising demand for high-throughput hematology analyzers and development of high-sensitivity point-of-care (POC) hematology testing are also fuelling the growth of the global hematology analyzer & reagent market. However, the high cost of hematology analyzers and intense competition among existing players are restraining the growth of the global hematology analyzers & reagent market. In addition, stringent and time-consuming regulatory policies for hematology instruments also impede the market growth.

Usage of microfluidics technology in hematology analyzers and introduction of digital imaging system in hematology laboratories could open up opportunities for new players in the global hematology analyzer & reagent market. In addition, increasing focus toward emerging markets, such as India and China, could also open up opportunities for new players in the global hematology analyzer & reagent market. Moreover, safety and quality of hematology analyzer could be a challenge for the growth of the global hematology analyzer & reagent market.

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Increasing instances of partnership among hematology instruments and consumables manufacturers is one of the recent trends in the global hematology analyzer & reagent market. The major companies operating in this market are

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Firefighter raise thousands within hours for one of their own – WKYT

By daniellenierenberg

LEXINGTON, Ky. (WKYT) - Firefighters in Lexington spent their Saturday morning supporting their own with a pancake breakfast raising money for a young woman with a rare disease.

Chris OBryan has worked as a firefighter in Kentucky for 23 years. This past summer his daughter began experiencing stomach pain. After months of testing, doctors diagnosed her PCH, a rare disease caused by a mutation in bone marrow stem cells.

Once other Lexington Firefighters heard about OBryans daughter, they decided to help however they can.

Its seven days that this came together, said firefighter William Tabor.

Tabor initiated the breakfast without ever meeting OBryan and his family before.

Both men say that goes to show the family community within the fire department. Within just more than an hour of beginning the breakfast at 8 Saturday, they raised $5,000.

The money thats raised here is twofold, started OBryan. Some of its for my daughter and some of its for a Louisville firefighter who was tragically killed in Saint Louis.

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Transplant for Szary Syndrome is Patient’s First Step in Returning to the Dance Floor – Dana-Farber Cancer Institute

By daniellenierenberg

The first time Bill Cronin Googled his own cancer diagnosis in 2016, his heart sank. He had Szary syndrome, a rare and aggressive form of cutaneous T-cell lymphoma and staring back at him were countless articles predicting a negative prognosis.

However, after receiving a stem-cell transplant at Dana-Farber/Brigham and Womens Cancer Center, Cronin is returning to the life he enjoyed before cancer.

Im at a place I never thought Id get to, Cronin says.

In 2015, Cronin, then 60, started feeling incredibly itchy and developed an accompanying rash. He went to his dermatologist, who diagnosed him with eczema and told him to return in five months. The rash continued to grow, however, and at the five month mark, Cronins dermatologist encouraged him to undergo further testing at Dana-Farber.

A blood test revealed that Cronins T-cells a type ofwhite blood cells that make up part of the immune system had becomecancerous. In the case of Szary syndrome, lymphoma cells will circulatethrough the blood stream and deposit in different areas of the skin. This willgenerally lead to a full-body rash and intense itchiness.

Cronin would need a stem cell transplant to combat the disease, but before he could receive one, his care team had to get him into remission. Patients who do not achieve remission prior to transplant have a high chance of relapsing.

When they first told me everything, I was really scared, says Cronin. But I knew I was in one of the best places in the world to figure out and treat this rare disease.

Cronins pre-transplant care was spearheaded by oncologists David Fisher, MD, and Nicole LeBoeuf, MD, MPH, clinical director of Cutaneous Oncology at Dana-Farber, with his transplant conducted by Corey Cutler, MD, MPH, medical director of the Adult Stem Cell Transplantation Program at Dana-Farber. Initially, Cronins disease was incredibly resistant; for nearly three years, mainstay drugs including steroids, monoclonal antibodies, and enzyme blockers all failed to put his disease into remission.

Ultimately, it would take a new drug, mogamulizumab (a type of immunotherapy that directly kills T-cells involved with Sezary Syndrome) to get Cronins disease into remission.

In May 2019, Cronin was cleared to undergo an allogeneic transplant, a type of transplant that uses a donors stem cells, in this case, Cronins brother. Since his transplant Cronin has remained in remission.

We had to use all of our big guns to get him totransplant, but Im pleased with where we are now, says Cutler.

I know the situation can always change, but it was great tobe able to share some good news with my family and friends, adds Cronin.

Patients like Cronin serve as a reminder of how stem cell transplants have improved and continue to impact patient outcomes, Dana-Farber experts note. Initially offered to only an incredibly small patient population when first performed at Dana-Farber in the 1970s, research advancements have, and continue to, broaden who is eligible for a transplant. In 2019, Dana-Farber/Brigham and Womens Cancer Center (DF/BWCC) surpassed 10,000 total adult transplants.

This milestone indicates our success as a program and our volume has allowed us to do the research to help move the field forward rather impressively, says Joseph Antin, MD, chief emeritus of Adult Stem Cell Transplantation at DF/BWCC.

In 1996, Dana-Farber Cancer Institute and Brigham and Womens Hospital merged their then separate transplant centers. By pooling together physical and intellectual resources, the new combined program was able to more than double the number of transplants each hospital could perform individually.

We always felt collaboration was better than competition, explains Robert Soiffer, MD, vice chair of Medical Oncology for Hematological Malignancies and chief of the Division of Hematologic Malignancies, who oversaw the merger with Antin. Each side could learn from the other, and that helped to catapult us into the leadership position we have today.

The Stem Cell Transplantation Program is also bolstered by the Connell and OReilly Families Cell Manipulation Core Facility (CMCF), which was established in 1996. The state-of-the-art center, led by Jerome Ritz, MD, not only processes the stem cells for transplant; it also assists researchers in developing new cell-based therapies for patients.

Another key component to the programs success has been the creation of the Ted and Eileen Pasquarello Tissue Bank. The Pasquarello Tissue Bank receives, processes, banks, and distributes research samplesof blood, bone marrow, and other tissues. Through a database overseen by Vincent Ho, MD, the Institute is able to log, assess, and later review every patients disease, including all complications and mutations. This technology allows researchers to explore the genetic makeup of past donors and better understand why a transplant was or was not successful.

Were still learning from biological specimens we collected 20 years ago, and it will continue to impact care 20 years from now, Soiffer says.

Today, there is a continuous push to develop new and more precise therapies to complement and improve stem cell transplants. The hope is to bring new treatment options to patients like Cronin who are facing rare and difficult diseases.

Before his diagnosis, Bill, and Barbara Finney, his partner ofnearly 30 years, were avid English Country dancers. English Country dancingevolved from the court dances of Europe in the early 17th century, and Croninand Barbara have friends from all over the country who share their passion forit.

While Cronin isnt dancing just yet, as hes stillrecovering from his transplant, he says he couldnt have gotten through thiswithout his partner on the dance floor and in life.

Barbara has been amazing and has helped take care ofeverything I couldnt do, he adds. Ive been fortunate and privileged to notonly have her, but to have been able to come to Dana-Farber.

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From Germany with love: Alfie, four, meets his stem cell saviour – Express

By daniellenierenberg

Alfie Commons and mum Lorna, 40, met Christin Bouvier, 34, for the first time on Wednesday after spending more than two years communicating via anonymous letters due to donation laws.

Alfie, from Toton, Notts, was diagnosed with acute lymphoblastic leukaemia (ALL) at seven months after he had a cold for six weeks.

After chemotherapy failed, Alfie was put on a trial immunotherapy drug which helped him to recover to be eligible for a stem cell transplant.

Unable to find a family match, his family found Christin on a worldwide register operated by blood cancer charity DKMS. She donated her cells in a one-hour op and they were flown to the UK and slowly passed into Alfies body in August 2016.

When the teacher, from Schwerin, Germany, was finally allowed to meet the family at Chiswick Town Hall in west London, the little boy gave a gift of Lacoste Pink perfume.

Mum-of-two Lorna, who works in HR, said: The meet-up was just amazing, it was everything we could have possibly dreamed of there were lots of hugs and tears.

It didnt feel like I was meeting her for the first time because wed been chatting for so long before.

As a family, we owe so much to Christin, words of thanks will never feel enough.

Christin just cried when she heard the recipient was a baby. She said: After I donated my bone marrow and the anaesthetic wore off, I called DKMS.

They told me that Alfie was a small baby and living in the UK but couldnt tell me any more due to the laws. When I found out Alfie was responding to treatment, so many tears of joy ran down my face. I still cant describe that moment.

Its a moment that is always with me. Whenever I feel a bit down, I think back to it as it always brings me so much happiness! She added that the meeting was so amazing. I was very nervous and shaking at first and when we finally met we cried a lot and hugged.

Alfie was shy at first but after a bit of time he became more comfortable and we played with some balloons and had a slice of cake.

After the transplant, Alfie developed a deadly immune condition, but this was controlled by medication. He was given the cancer all-clear in 2017 and has started school.

Lorna added: I just want more people to sign up to become donors theres a match for everyone.

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From Germany with love: Alfie, four, meets his stem cell saviour - Express

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Single-cell Analysis Market Worth $5.6 Billion by 2025 – Exclusive Report by MarketsandMarkets – PRNewswire

By daniellenierenberg

CHICAGO, Feb. 21, 2020 /PRNewswire/ -- According to the new market research report "Single-cell Analysis Marketby Cell Type (Human, Animal, Microbial), Product (Consumables, Instruments), Technique (Flow Cytometry, NGS, PCR, Mass Spectrometry, Microscopy), Application (Research, Medical Application), End User - Global Forecasts to 2025", published by MarketsandMarkets, the Single-cell Analysis Marketis projected to reach USD 5.6 billion by 2025 from USD 2.1 billion in 2019, at a CAGR of 17.8% during the forecast period.

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The growth in this market is driven by technological advancements in single-cell analysis products, increasing government funding for cell-based research, growing biotechnology and biopharmaceutical industries, wide applications of single-cell analysis in cancer research, growing focus on personalized medicine, and the increasing incidence and prevalence of chronic and infectious diseases. However, the high cost of single-cell analysis products is expected to restrain the growth of this market to a certain extent during the forecast period.

The research applications segment accounted for the largest share of the market, by application, in 2018

Based on application, the Single-cell Analysis Market is segmented into research (cancer, immunology, neurology, stem cell, and other research applications) and medical applications (noninvasive prenatal diagnosis, in vitro fertilization, and circulating tumor cell detection). The research applications segment accounted for the largest share of the market in 2018. Increasing government initiatives in stem cell research and the wide usage of single-cell analysis in cancer research are the major factors driving the growth of the research applications segment.

Browsein-depth TOC on"Single-cell Analysis Market"

225 Tables 50 Figures252 Pages

The flow cytometry segment accounted for the largest market share in 2018

Based on technique, the market is segmented into flow cytometry, NGS, PCR, microscopy, mass spectrometry, and other techniques. The flow cytometry segment accounted for the largest market share in 2018. The large share of this segment is attributed to the wide usage of flow cytometry in detecting and measuring the physical and chemical characteristics of a population of cells or particles. However, the NGS segment is projected to register the highest growth rate during the forecast period. The high growth of the NGS segment is driven by the increasing application of single-cell analysis products in drug discovery for cancer and other chronic diseases.

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North America dominates the Single-cell Analysis Market

The global market is segmented into five major regions, namely, North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. In 2018, North America accounted for the largest share of the market. The growth in this market can be attributed to the increasing drug development activities in the pharmaceutical and biotechnology industries, rising prevalence of chronic and infectious diseases, and an increase in stem cell research activities.

Prominent players in the Single-cell Analysis Market include Becton, Dickinson and Company (US), Danaher Corporation (US), Merck Millipore (US), QIAGEN (Netherlands), Thermo Fisher Scientific (US), General Electric Company (US), 10x Genomics (US), Promega Corporation (US), Illumina (US), Bio-Rad Laboratories (US), Fluidigm Corporation (US), Agilent Technologies (US), NanoString Technologies (US), Tecan Group (Switzerland), Sartorius AG (Germany), Luminex Corporation (US), Takara Bio (Japan), Fluxion Biosciences (US), Menarini Silicon Biosystems (Italy), and LumaCyte (US).

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Cell Counting Marketby Product (Instruments (Spectrophotometer, Cell Counter, Hemocytometer, Flow Cytometer, Hematology Analyzer), Consumables (Reagent, Assay Kits, Microplate)), End User (Pharmaceutical, Hospital, Research) - Global Forecast to 2023

Cell Isolation/Cell Separation Marketby Product (Reagents, Beads, Centrifuge), Cell Type (Human, Animal), Cell Source (Bone Marrow, Adipose), Technique (Filtration), Application (Cancer), End User (Hospitals, Biotechnology) - Global Forecast to 2024

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Single-cell Analysis Market Worth $5.6 Billion by 2025 - Exclusive Report by MarketsandMarkets - PRNewswire

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Cord Stem Cell Banking Market 2020 to Witness Great Growth || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord,…

By daniellenierenberg

By keeping an eye on the market conditions and market trends, market research study is initiated depending on clients requirements to form this business document. This Cord Stem Cell Banking market report gives the details about market definition, market drivers, market restraints, market segmentation with respect to product usage and geographical conditions, key developments taking place in the market, competitor analysis, and the research methodology. One of the most noteworthy parts of this Cord Stem Cell Banking Market report is competitor analysis with which businesses can estimate or analyse the strengths and weaknesses of the competitors to gain benefits.

Global Cord stem cell banking market is estimated to reach USD 13.8 billion by 2026 registering a healthy CAGR of 22.4%. The increasing number of parents storing their childs cord blood, acceptance of stem cell therapeutics, high applicability of stem cells are key driver to the market.

Request for sample copy or PDF Here https://www.databridgemarketresearch.com/request-a-sample?dbmr=global-cord-stem-cell-banking-market&raksh

Few of the major market competitors currently working in the globalcord stem cell banking marketareCBR Systems, Inc., Cordlife, Cells4Life Group LLP, Cryo-Cell International, Inc., Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord, SMART CELLS PLUS., Cryoviva India, Global Cord Blood Corporation, National Cord Blood Program, Vita 34, ReeLabs Pvt. Ltd., Regrow Biosciences Pvt. Ltd. , ACROBiosystems., Americord Registry LLC., New York Blood Center, Maze Cord Blood, GoodCell., AABB, Stem Cell Cryobank, New England Cryogenic Center, Inc. among others

Market Definition: Global Cord Stem Cell Banking Market

Cord stem cells banking is nothing but the storing of the cord blood cell contained in the umbilical cord and placenta of a newborn child. This cord blood contains the stem cells which can be used in future to treat disease such as leukemia, thalassemia, autoimmune diseases, and inherited metabolic disorders, and few others.

Segmentation: Global Cord Stem Cell Banking Market

Cord Stem Cell banking Market : By Storage Type

Cord Stem Cell banking Market : By Product Type

Cord Stem Cell banking Market : By Service Type

Cord Stem Cell banking Market : By Indication

Cord Stem Cell banking Market : By Source

Cord Stem Cell banking Market : By Geography

Browse Detailed TOC, Tables, Figures, Charts and Companies @https://www.databridgemarketresearch.com/toc?dbmr=global-cord-stem-cell-banking-market&raksh

Key Developments in the Cord Stem Cell banking Market:

Cord Stem Cell banking Market : Drivers

Cord Stem Cell banking Market : Restraint

Competitive Analysis: Global Cord Stem Cell Banking Market

Global cord stem cell banking market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions and others to increase their footprints in this market. The report includes market shares of cord stem cell banking market for Global, Europe, North America, Asia Pacific, South America and Middle East & Africa.

Scope of the Cord Stem Cell banking Market Report :

The report shields the development activities in the Cord Stem Cell banking Market which includes the status of marketing channels available, and an analysis of the regional export and import. It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments. This will benefit the reports users, that evaluates their position in Cord Stem Cell banking market as well as create effective strategies in the near future.

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Cord Stem Cell Banking Market 2020 to Witness Great Growth || Key Players Cryo-Save AG, Lifecell, StemCyte India Therapeutics Pvt. Ltd, Viacord,...

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Actinium Presents Positive Findings from Mid-Point Analysis of Pivotal SIERRA Trial of Iomab-B at 2020 Transplantation & Cellular Therapy Meetings of…

By daniellenierenberg

NEW YORK, Feb. 21, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") announced today findings from the SIERRA trial that were presented at the 2020 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (TCT) in Orlando, FL. Dr. Boglarka Gyurkocza, the principal investigator from Memorial Sloan Kettering Cancer Center, revealed that there were key differences in side effects reported in patients treated in the Iomab-B and control arms of the study with rates of febrile neutropenia, sepsis and mucositis being markedly lower in the Iomab-B arm.

The oral presentation also featured updated results from the fifty percent enrollment mid-point analysis including BMT access rates, engraftment and 100-day non-relapse transplant related mortality (TRM). After accounting for these factors, the results showed that, on an intent-to-treat or ITT basis, 78 percent of patients in the Iomab-B arm are potentially evaluable for the primary endpoint compared to 13 percent in the control arm. In addition, an important and recent protocol amendment was highlighted; the approximately 30 percent of patients who are expected to fail induction therapy with venetoclax plus hypomethylating agents1 are now eligible for enrollment in the SIERRA trial. The change is expected to increase the addressable patient population of the study given that this combination is now recommended as part of the NCCN or National Comprehensive Cancer Network guidelines, is widely used and expected to become the treatment of choice.

Key Interim Results:

The data tables that follow summarize key findings that were presented as part of the TCT proceedings. As highlighted in the table below, patients receiving Iomab-B showed lower rates of key adverse events relevant in the BMT setting in compared to patients randomized to receive physician's choice of salvage chemotherapy on the control arm. For example, patients receiving Iomab-B had a much lower incidence of sepsis of 3 percent compared to the 42 percent incidence in the control arm.

Adverse Event* N (%)

Randomized to Iomab-B andreceived BMT (N=31)

Randomized to Control Armand received BMT (N=7)

Febrile Neutropenia

8 (25.8)

3 (42.8)

Sepsis/Septic Shock

1 (3.2)

3 (42.8)

Stomatitis (mucositis)

3 (9.7)

2 (28.6)

Pneumonia/Lung Infection

4 (12.9)

1 (14.3)

Hypertension

61 (19.4)

1 (14.3)

Decreased Appetite

5 (16.1)

0 (0.0)

Device related infection

4 (12.9)

1 (14.3)

Hypophosphatemia

2 (6.5)

1 (14.3)

* All adverse events reported irrespective of attribution to protocol-directed procedures

1) 5 patients had hypertension considered unrelated to Iomab-B and 1 patient had hypertension possibly related to Iomab-B

"This detailed safety data from SIERRA is highly encouraging, particularly in this patient population, as neutropenia and sepsis are hallmark toxicities associated with chemotherapy-based conditioning regimens that next to relapse are leading causes of morbidity and mortality post-transplant," said Dr. Vijay Reddy, Vice President, Clinical Development and Head of BMT at Actinium. "Chemotherapy-based conditioning damages normal organs, such as the gastrointestinal tract, which allows gut bacteria to cause serious infections. The data showing less mucositis, febrile neutropenia, and sepsis are consistent with the targeted nature of Iomab-B, since a lack of damage to the gastrointestinal track would lead to a reduction in these adverse events. BMT is the only curable treatment option for older patients with active, relapsed or refractory AML. Yet these patients face restricted access and suboptimal outcomes due to reliance on chemotherapy-based conditioning regimens and perceptions in the hematologist community around safety and eligibility for BMT. With the SIERRA trial, our goals are to eliminate these barriers leading to more patients receiving BMT and with better patient outcomes. With this additional safety data in hand, we have even greater confidence in our ability to change the perceptions around BMT and are excited to update the transplant and hematology communities on Iomab-B's potential to positively impact patients through improved access to BMT and better outcomes."

The presentation highlighted that 100 percent (31/31) of patients receiving a therapeutic dose of Iomab-B achieved successful BMT engraftment with only a 6 percent (2/31) TRM rate compared to the control arm where 18 percent (7/38) achieved engraftment with a 29 percent (2/7) TRM rate. At the 100-day post BMT time point, on an ITT basis, there were 29 patients from the Iomab-B study arm potentially evaluable for the primary endpoint of durable Complete Remission (dCR) at 180 days compared to 5 patients in the control arm. By this measure, 78 percent of patients in the Iomab-B arm are potentially eligible for the dCR primary endpoint compared to 13 percent of patients in the control arm. The mid-point analysis and data presented at TCT can be viewed here.

Detailed engraftment data is presented in the table below:

BMT Feasibility andOutcome Data

Randomized toStudy Arm (N=37)

Randomized to Control Arm (N=38)

ReceivedTherapeutic Doseof Iomab-B andreceived BMT(N=31)1

Achieve CR andreceived standardBMT (N=7)

Did not Achieve CR(N=31/38)2

Crossed over from toIomab-B and receivedBMT (N=20)

BMT Engraftment Rate (%, N)

100% (31/31)

18% (7/38)

100% (20/20)

Median Bone Marrow Blasts% at randomization

(%, range)

29% (5-88)

26% (5-97)

At crossover: 31%

(6-87)

At randomization: 35%(5-75)

Median Days to BMT postrandomization (days, range)

30 (23-50)

67 (51-86)

64 (44-161)

Median Days to AbsoluteNeutrophil Count Engraftment(days, range)

15 (9-22)3

18 (13-82)4

14 (10-37)5

Median Day to PlateletEngraftment (days, range)

20 (4-39)3

22 (9-35)4

19 (13-38)5

100-day non-relapsetransplant related mortality(%, N)

6% (2/31)

29% (2/7)

10% (2/20)6

1) No therapeutic dose (6) due to: declining Karnofsky Performance Scale (PFS) (3), Infusion reaction (1), unfavorablebiodistribution (1), post-randomization eligibility (1)

2) Ineligible for crossover (9) due to: hospice care/progression (4), declined/ineligible for BMT (2), died pre-crossover (3),eligible for crossover (2) did not receive Iomab-B due to declining status

3) Absolute Neutrophil Count engraftment data not available (1), platelet engraftment data not available (4)

4) ANC and platelet engraftment data not available (1), engraftment failure (1)

5) ANC engraftment data not available (1) out of 20, platelet engraftment data not available (3)

6) 1 patient at 161 days had delayed transplant due to infection and respiratory failure, received Iomab-B and BMT when stable

Dr. Mark Berger, Actinium's Chief Medical Officer, stated, "As we approach critical enrollment milestones in the SIERRA trial, our focus turns to bringing Iomab-B to as many patients as possible that can benefit from this product candidate and as expeditiously as possible. Consequently, we made an important amendment to the SIERRA protocol to expand the potential patient pool by including in the eligibility criteria patients who fail induction therapy with venetoclax plus hypomethylating agents. As targeted agents such as venetoclax and others have gained approval, the acute myeloid leukemia treatment landscape has evolved with a significant percentage of patients being treated with these agents in frontline and relapsed settings. In fact, as of mid-2019, venetoclax plus hypomethylating agents have been included as part of the AML National Comprehensive Cancer Network guidelines and medical practice is embracing these regimens widely. However, these regimens are not curative, nor do they eliminate the need for BMT. Indeed, approximately thirty percent of patients fail to achieve a remission after two cycles of induction therapy with venetoclax and most patients ultimately relapse with a median duration of response of less than one year. This amendment has already had a positive impact on the trial that we expect to continue through the remaining portion of enrollment. Most importantly, if Iomab-B gains approval, this amendment will support its use for the significant and growing number of patients receiving and failing venetoclax as induction therapy instead of traditional 7+3 induction chemotherapy. We look forward to continuing to provide key updates as SIERRA reaches key milestones and completes enrollment in 2020."

Sources: 1) DiNardo et al. Venetoclax combined with decitabine or azacitidine in treatment-nave, elderly patients with acute myeloid leukemia. Blood 2019 133(1): 7-17 https://doi.org/10.1182/blood-2018-08-868752

About the SIERRA TrialThe SIERRA trial (Study ofIomab-B inElderlyRelapse/RefractoryAcute Myeloid Leukemia) is the only randomized Phase 3 trial that offers BMT (Bone Marrow Transplant) as an option for older patients with active, relapsed or refractory AML or acute myeloid leukemia. BMT is the only potentially curative treatment option for older patients with active relapsed or refractory AML and there is no standard of care for this indication other than salvage therapies. The SIERRA trial is a 150-patient, multicenter randomized trial that studying Iomab-B compared to physician's choice of salvage chemotherapy. The primary endpoint of the SIERRA trial is durable Complete Remission of 180 days and the secondary endpoint is 1-year overall survival. Iomab-B is an ARC or Antibody Radiation-Conjugate comprised of the anti-CD45 antibody apamistamab and the radioisotope I-131 (Iodine-131). The 20 active SIERRA trial sites in the U.S. and Canada represent many of the leading bone marrow transplant centers by volume. For more information, visit http://www.sierratrial.com.

About Transplantation & Cellular Therapy Meetings (TCT)

TCT, formerly known as the BMT Tandem Meetings, are the combined annual meetings of the American Society for Blood and Marrow Transplantation (ASBMT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).Each year the conference brings together several thousand investigators, clinicians, researchers, nurses and other allied health professionals from over 500 transplant centers from over 50 countries around a full scientific program focused on bone marrow transplant and cellular therapies.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3Study ofIomab-B inElderlyRelapsed orRefractoryAcute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans Vitzthum LifeSci Advisors, LLCHans@LifeSciAdvisors.com (617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

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SOURCE Actinium Pharmaceuticals, Inc.

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Actinium Presents Positive Findings from Mid-Point Analysis of Pivotal SIERRA Trial of Iomab-B at 2020 Transplantation & Cellular Therapy Meetings of...

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Stem cells and the heartthe road ahead – Science Magazine

By daniellenierenberg

Heart disease is the primary cause of death worldwide, principally because the heart has minimal ability to regenerate muscle tissue. Myocardial infarction (heart attack) caused by coronary artery disease leads to heart muscle loss and replacement with scar tissue, and the heart's pumping ability is permanently reduced. Breakthroughs in stem cell biology in the 1990s and 2000s led to the hypothesis that heart muscle cells (cardiomyocytes) could be regenerated by transplanting stem cells or their derivatives. It has been 18 years since the first clinical trials of stem cell therapy for heart repair were initiated (1), mostly using adult cells. Although cell therapy is feasible and largely safe, randomized, controlled trials in patients show little consistent benefit from any of the treatments with adult-derived cells (2). In the meantime, pluripotent stem cells have produced bona fide heart muscle regeneration in animal studies and are emerging as leading candidates for human heart regeneration.

In retrospect, the lack of efficacy in these adult cell trials might have been predicted. The most common cell type delivered has been bone marrow mononuclear cells, but other transplanted cell types include bone marrow mesenchymal stromal cells and skeletal muscle myoblasts, and a few studies have used putative progenitors isolated from the adult heart itself. Although each of these adult cell types was originally postulated to differentiate directly into cardiomyocytes, none of them actually do. Indeed, with the exception of skeletal muscle myoblasts, none of these cell types survive more than a few days in the injured heart (see the figure). Unfortunately, the studies using bone marrow and adult resident cardiac progenitor cells were based on a large body of fraudulent work (3), which has led to the retraction of >30 publications. This has left clinical investigators wondering whether their trials should continue, given the lack of scientific foundation and the low but measurable risk of bleeding, stroke, and infection.

Additionally, investigators have struggled to explain the beneficial effects of adult cell therapy in preclinical animal models. Because none of these injected cell types survive and engraft in meaningful numbers or directly generate new myocardium, the mechanism has always been somewhat mysterious. Most research has focused on paracrine-mediated activation of endogenous repair mechanisms or preventing additional death of cardiomyocytes. Multiple protein factors, exosomes (small extracellular vesicles), and microRNAs have been proposed as the paracrine effectors, and an acute immunomodulatory effect has recently been suggested to underlie the benefits of adult cell therapy (4). Regardless, if cell engraftment or survival is not required, the durability of the therapy and need for actual cells versus their paracrine effectors is unclear.

Of particular importance to clinical translation is whether cell therapy is additive to optimal medical therapy. This remains unclear because almost all preclinical studies do not use standard medical treatment for myocardial infarction. Given the uncertainties about efficacy and concerns over the veracity of much of the underlying data, whether agencies should continue funding clinical trials using adult cells to treat heart disease should be assessed. Perhaps it is time for proponents of adult cardiac cell therapy to reconsider the approach.

Pluripotent stem cells (PSCs) include embryonic stem cells (ESCs) and their reprogrammed cousins, induced pluripotent stem cells (iPSCs). In contrast to adult cells, PSCs can divide indefinitely and differentiate into virtually every cell type in the human body, including cardiomyocytes. These remarkable attributes also make ESCs and iPSCs more challenging to control. Through painstaking development, cell expansion and differentiation protocols have advanced such that batches of 1 billion to 10 billion pharmaceutical-grade cardiomyocytes, at >90% purity, can be generated.

Preclinical studies indicate that PSC-cardiomyocytes can remuscularize infarcted regions of the heart (see the figure). The new myocardium persists for at least 3 months (the longest time studied), and physiological studies indicate that it beats in synchrony with host myocardium. The new myocardium results in substantial improvement in cardiac function in multiple animal models, including nonhuman primates (5). Although the mechanism of action is still under study, there is evidence that these cells directly support the heart's pumping function, in addition to providing paracrine factors. These findings are in line with the original hope for stem cell therapyto regenerate lost tissue and restore organ function. Additional effects, such as mechanically buttressing the injured heart wall, may also contribute.

Breakthroughs in cancer immunotherapy have led to the adoption of cell therapies using patient-derived (autologous) T cells that are genetically modified to express chimeric antigen receptors (CARs) that recognize cancer cell antigens. CAR T cells are the first U.S. Food and Drug Administration (FDA)approved, gene-modified cellular pharmaceutical (6). The clinical and commercial success of autologous CAR T cell transplant to treat B cell malignancies has opened doors for other complex cell therapies, including PSC derivatives. There is now a regulatory path to the clinic, private-sector funding is attracted to this field, and clinical investigators in other areas are encouraged to embrace this technology. Indeed, the first transplants of human ESC-derived cardiac progenitors, surgically delivered as a patch onto the heart's surface, have been carried out (7). In the coming years, multiple attempts to use PSC-derived cardiomyocytes to repair the human heart are likely.

What might the first human trials look like? These studies will probably employ an allogeneic (non-self), off-the-shelf, cryopreserved cell product. Although the discovery of iPSCs raised hopes for widespread use of autologous stem cell therapies, the current technology and regulatory requirements likely make this approach too costly for something as common as heart disease, although this could change as technology and regulations evolve. Given that it would take at least 6 months to generate a therapeutic dose of iPSC-derived cardiomyocytes, such cells could only be applied to patients whose infarcts are in the chronic phase where scarring (fibrosis) and ventricular remodeling are complete. Preclinical data indicate that chronic infarcts benefit less from cardiomyocyte transplantation than do those with active wound-healing processes.

Adult cells from bone marrow or the adult heart secrete beneficial paracrine factors but do not engraft in the infarcted heart. Pluripotent stem cells give rise to cardiomyocytes that engraft long term in animal models, beat in synchrony with the heart, and secrete beneficial paracrine factors. Long-term cardiomyocyte engraftment partially regenerates injured heart, which is hypothesized to bring clinical benefits.

The need for allogeneic cells raises the question of how to prevent immune rejection, both from innate immune responses in the acute phase of transplantation or from adaptive immune responses that develop more slowly through the detection of non-self antigens presented by major histocompatibility complexes (MHCs). A current strategy is the collection of iPSCs from patients who have homozygous MHC loci, which results in exponentially more MHC matches with the general population. However, studies in macaque monkeys suggest that MHC matching will be insufficient. In a macaque model of brain injury, immunosuppression was required to prevent rejection of MHC-matched iPSC-derived neurons (8). Similarly, MHC matching reduced the immunogenicity of iPSC-derived cardiomyocytes transplanted subcutaneously or into the hearts of rhesus macaques, but immunosuppressive drugs were still required to prevent rejection (9).

Numerous immune gene editing approaches have been proposed to circumvent rejection, including preventing MHC class I and II molecule expression, overexpressing immunomodulatory cell-surface factors, such CD47 and human leukocyte antigen E (HLA-E) and HLA-G (two human MHC molecules that promote maternal-fetal immune tolerance), or engineering cells to produce immunosuppressants such as programmed cell death ligand 1 (PDL1) and cytotoxic T lymphocyteassociated antigen 4 (CTLA4) (10). These approaches singly or in combination seem to reduce adaptive immune responses in vitro and in mouse models. Overexpressing HLA-G or CD47 also blunts the innate natural killer cellmediated response that results from deleting MHC class I genes (11). However, these manipulations are not without theoretical risks. It could be difficult to clear viral infections from an immunostealthy patch of tissue, and possible tumors resulting from engraftment of PSCs might be difficult to clear immunologically.

Ventricular arrhythmias have emerged as the major toxicity of cardiomyocyte cell therapy. Initial studies in small animals showed no arrhythmic complications (probably because their heart rates are too fast), but in large animals with human-like heart rates, arrhythmias were consistently observed (5, 12). Stereotypically, these arrhythmias arise a few days after transplantation, peak within a few weeks, and subside after 4 to 6 weeks. The arrhythmias were well tolerated in macaques (5) but were lethal in a subset of pigs (12). Electrophysiological studies indicate that these arrhythmias originate in graft regions from a source that behaves like an ectopic pacemaker. Understanding the mechanism of these arrhythmias and developing solutions are major areas of research. There is particular interest in the hypothesis that the immaturity of PSC-cardiomyocytes contributes to these arrhythmias, and that their maturation in situ caused arrhythmias to subside.

A successful therapy for heart regeneration also requires understanding the host side of the equation. PSC-derived cardiomyocytes engraft despite transplantation into injured myocardium that is ischemic with poor blood flow. Although vessels eventually grow in from the host tissue, normal perfusion is not restored. Achieving a robust arterial input will be key to restoring function, which may require cotransplanting other cell populations or tissue engineering approaches (13, 14). Most PSC-mediated cardiac cell therapy studies have been performed in the subacute window, equivalent to 2 to 4 weeks after myocardial infarction in humans. At this point, there has been insufficient time for a substantial fibrotic response. Fibrosis has multiple deleterious features, including mechanically stiffening the tissue and creating zones of electrical insulation that can cause arrhythmias. Extending this therapy to other clinical situations, such as chronic heart failure, will require additional approaches that address the preexisting fibrosis. Cell therapy may again provide an answer because CAR T cells targeted to cardiac fibroblasts reduced fibrosis (15).

Developing a human cardiomyocyte therapy for heart regeneration will push the limits of cell manufacturing. Each patient will likely require a dose of 1 billion to 10 billion cells. Given the widespread nature of ischemic heart disease, 105 to 106 patients a year are likely to need treatment, which translates to 1014 to 1016 cardiomyocytes per year. Growing cells at this scale will require introduction of next generation bioreactors, development of lower-cost media, construction of large-scale cryopreservation and banking systems, and establishment of a robust supply chain compatible with clinical-grade manufacturing practices.

Beyond PSC-cardiomyocytes, other promising approaches include reactivating cardiomyocyte division and reprogramming fibroblasts to form new cardiomyocytes. However, these approaches are at an earlier stage of development, and currently, PSC-derived cardiomyocyte therapy is the only approach that results in large and lasting new muscle grafts. The hurdles to this treatment are known, and likely addressable, thus multiple clinical trials are anticipated.

Acknowledgments: C.E.M. and W.R.M. are scientific founders of and equity holders in Sana Biotechnology. C.E.M. is an employee of Sana Biotechnology. W.R.M. is a consultant for Sana Biotechnology. C.E.M. and W.R.M. hold issued and pending patents in the field of stem cell and regenerative biology.

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