Here are all the science projects that SpaceX will deliver to the ISS – Digital Trends
By daniellenierenberg
In a couple of weeks, SpaceX will be launching a Dragon cargo spacecraft bound for the International Space Station (ISS), carrying not only supplies for the astronauts but also a range of scientific equipment and research technology. The cargo includes tools for researching everything from growing human heart cells to making more comfortable sneakers.
One of the largest additions to the ISS will be the Bartolomeo facility, a European Space Agency project to provide more room for scientific experiments by attaching to the outside of the space station. Potential uses for the extended space include Earth observation, robotics, material science, and astrophysics, according to NASA.
Other projects include one by Adidas to test out its molding process in which thousands of pellets are blown together until they fuse, creating a midsole for shoes to make them more cushioned for high-performance athletes. Theres also a study into how water droplets form in low gravity which could help reduce the amount of water used by showers here on Earth, assisting the important project of water conservation. And theres a project to test improvements in 3D printing which could be used to print spare parts and repair tools for future space voyages.
Finally, there are also two biomedical experiments being taken to the ISS. One will look at how microgravity affects biotechnology like the Organ Chip which simulates the responses of human tissue on a small chip. And the other will investigate whether it is possible to grow human heart cells from stem cells in microgravity. The researchers believe the development of these heart cells could eventually be used to treat cardiac problems here on Earth, especially among children as their cardiac issues are particularly hard to treat.
The mission is scheduled to launch at 10:45 p.m. PT on Sunday, March 1, from Space Launch Complex 40 at Cape Canaveral Air Force Station in Florida. This will be the 20th mission as part of NASAs Commercial Resupply Services contract, in which private companies like SpaceX and Boeing take over some duties for delivering supplies to the ISS.
In the future, SpaceX will be taking a larger part in ISS operations as well. It will be delivering astronauts to and from the space station as part of NASAs Commercial Crew program, using its Crew Dragon capsule. The first manned Crew Dragon mission is targeted for May 7.
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Here are all the science projects that SpaceX will deliver to the ISS - Digital Trends
Searching for the ‘big break’ that could turn stem cells into a weapon against dementia – Genetic Literacy Project
By daniellenierenberg
Recent developments in the field of stem cell research are paving a path towards a radical shift in the way we diagnose and treat dementia. Stem cells have excited scientists for years and research groups across the globe are using them to advance modern medicine. Using stem cells to aid the fight against dementia is perhaps one of the most critical applications of the technology. Dementia is the leading cause of death in the UK, sixth in US and fifth globally, with an estimated 50 million people currently affected.
The term dementia does not relate to a single disease, but more an array of symptoms that can arise from multiple conditions. The most common is Alzheimers disease (AD) which accounts for up to 80% of all cases. Dementia itself is caused by the death of cells that make up the complex circuitry of our brains and an eventual loss of large portions of the brain. Patients suffering with dementia often exhibit the same general symptoms such as confusion, memory loss and an inability to perform day to day functions. It is a debilitating condition that often strikes the most vulnerable members of society and, consequently, many research groups around the globe work to try to understand dementia-causing diseases to provide better diagnostic and treatment platforms.
In 2007, a research group at Kyoto University in Japan published a study with the potential to change the face of research into dementia along with many other fields. Professor Shinya Yamanaka and his research team developed a method whereby stem cells (cells that can be transformed/differentiated into cells from any tissue) could be generated from a sample of skin. The study, which resulted in a 2012 Nobel Prize for Prof. Yamanaka, demonstrated that skin cells could be isolated from a patient and genetically reprogrammed into induced pluripotent stem cells (iPSCs). In short, this technology made it possible to generate and study brain cells from a patient with dementia without having to remove any of their brain. All they would need to do is provide scientists with a sample of skin.
Since this development, research groups around the globe have started using iPSCs from many patients with dementia in order to understand the biological mechanisms that underlie disease. Dr Eric Hill runs a research group at Aston University in the UK that specializes in iPSCs for dementia research and he had the following to say about the technology:
Its really exciting because it allows us to study cells with genetic mutations that are patient specific. We can get a much better picture of what is actually happening in the brains of these patients. We can now generate all the different cell types found in the human brain and understand how they function together and map the changes that result in disease.
The latter was perhaps most powerfully demonstrated in a study published by a team at the University of North Carolina, led by Professor Hansang Cho. The team was able to generate three key cell subtypes that play important roles in brain function; study the impact of mutations associated with Alzheimers disease; and even replicate some of the core malfunctions found to trigger disease in the brains of patients.
Studies like this are of significance because a large part of the focus in dementia research is on trying to understand how such changes in function arise. When a patient is diagnosed with a disease such as Alzheimers it is often too late for effective treatment. Scientists, instead, seek to elucidate those early changes in brain cell function in order to diagnose patients earlier to give more time for treatment. It is very much a case of prevention being better than a cure. Dr Hill provided an encouraging statement regarding this:
When we generate brain cells from iPSCs the cells we get are developmentally very young. What is interesting is the fact we still see differences between cells from dementia patients versus healthy patients suggesting we could find markers to help us detect and prevent disease some years before it develops.
Despite such promise, however, iPSCs have yet to provide the field of dementia research with that big break. Multiple treatments have progressed into clinical trials since the technology first emerged but no therapies have been approved. Drugs that show promise in the lab fail to deliver on their potential in patient clinical trials, sending researchers back to square one.
We should not be disheartened by this, however, and should instead view it as space into which the technology of using iPSCs to study dementia can grow. A lot of drugs fail in clinical trials because the platforms used to run initial tests dont provide scientists with a wide enough perspective of how those drugs will influence human cells. Additionally, many preclinical studies use animals with dementia-causing disease artificially induced into them. Studies like this often fail to translate into humans because the initial data is not from a human perspective. This is where researchers like Dr. Hill think iPSCs can provide us with an advantage:
iPSCs could provide us with much better platforms for screening drugs to treat and prevent these diseases. They can really add to what we already have, and while we might not be able to grow a full human brain, we can generate the cells that provide the building blocks for one. They give us the chance to screen new therapies more efficiently, better test their effectiveness and reduce the amount of animal use in dementia research.
Dr Hill is not alone in seeing the promise of using iPSCs to find better treatments for preventing the progression of dementia. Multiple research groups around the world have shown the potential of iPSC-derived brain cells for studying the effectiveness of new therapies.
In the last 12 months we have observed a wave of new studies using iPSCs to try to develop better treatments for diseases like Alzheimers, Parkinsons, Huntingtons disease and ALS. From studies in the University of California identifying cholesterol metabolism as a potential target to treating Alzheimers to studies in Luxembourg helping us find better treatments for Parkinsons, it is easy to see why the global effort to get that big break from iPSCs continues to gain interest. We might still be waiting for that next Noble Prize-winning discovery that will improve the lives of millions of patients but the collective effort of iPSC research groups across the world brings us a step closer with every study they publish. Dementia may, one day, be a thing of the past and iPSC research will likely be a significant part in getting us there.
Sam Moxon has a PhD in regenerative medicine and is currently involved in dementia research. He is a freelance writer with an interest in the development of new technologies to diagnose and treat degenerative diseases. Follow him on Twitter @DrSamMoxon
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Searching for the 'big break' that could turn stem cells into a weapon against dementia - Genetic Literacy Project
Breakthrough, Non-Invasive Treatment Called Axol Therapy For Erectile Dysfunction And Enhanced Sexual Performance Now Available At Colorado Urology -…
By daniellenierenberg
- Axol Softwave Therapy is a new treatment for erectile dysfunction (ED) and for men who want enhanced sexual performance
- The in-office treatment is non-invasive, safe, and effective with virtually no side effects
- Axol Therapy uses low-intensity sound waves
- Axol Therapy is an alternative to ED medications, surgical implants, penile pumps, and injections
DENVER, Feb. 24, 2020 /PRNewswire/ -- The men's sexual health specialists at Colorado Urology now offer an exciting new treatment option for men living with erectile dysfunction (ED) called Axol Softwave Therapy. This safe and non-invasive treatment option is helping many men with ED achieve spontaneous and natural erections without the help of medications. The therapy can also be used to enhance a man's sexual performance.
Colorado Urology (PRNewsfoto/Colorado Urology)
About 5 in 10 men experience erectile dysfunction (ED) at some point in their lives. First-line therapies often include oral medication to help men achieve an erection. Now, Axol Therapy is providing a safe and effective alternative.
This non-invasive procedure uses gentle full-spectrum, low-intensity sound waves that stimulate revascularization, a process in which new blood vessels form. Axol Therapy promotes improved blood flow to the penis, reduces inflammation, and stimulates the migration of the body's stem cells for long-term healing. The new treatment is helping men to achieve natural erections without ED medications, pumps, injections, or penile implants.
Learn about Axol Softwave Therapy at Colorado Urology: https://www.coloradouro.com/specialties/axol-softwave-therapy/.
Axol Therapy How it Works
Axol Therapy is a modern approach to healing the body by using four types of energy: Heat, Electrohydraulic, Acoustic, and Light (HEAL). Unfocused acoustic waves are delivered to the shaft of the penis using a treatment wand that features a patented unfocused electrohydraulic acoustic wave.
The pulsed acoustic waves are delivered through the skin into the tissue to open and repair aging blood vessels, stimulate new blood vessel growth, restore blood flow, and improve erectile quality. Axol Therapy typically takes only 20 minutes, once a week, for a total of six sessions in the physician's office.
How Well Does Axol Therapy Work?
For men who are the right candidates, Axol Therapy is a safe and effective option without the side effects often experienced with oral medications. Most patients can get the quality, rigid erections they once had with Axol Therapy's gentle acoustic pulse treatment within just six office visits. Incremental improvement in erectile function may be seen after just a few sessions.
Restoring Vitality and Quality of Life
There are a number of significant benefits to Axol Therapy. For men who are candidates for this treatment option, a future without erectile dysfunction is perhaps the biggest one. The restoration of a man's vitality and spontaneous active sex life are also major benefits of this exciting new treatment.
Learn more about Axol Softwave Therapy, the benefits, and how to schedule a consultation. Visit https://www.coloradouro.com/specialties/axol-softwave-therapy/or call 888-401-7149.
About Colorado Urology
Colorado Urology, an affiliate of United Urology Group, is Eastern Colorado's premier urology practice, which was formed when Advanced Urology, Alpine Urology, and Foothills Urology became one urology group in April 2019. The group provides a broad array of urologic services, and its integrated approach to urologic care provides patients with access to experienced specialists, a comprehensive support team of healthcare professionals, innovative diagnostic tools, and highly advanced treatments and therapies. Colorado Urology operates 12 medical offices throughout the Denver metro and Boulder area, has 18 urologists, 9 advanced practice providers, and more than 130 employees.
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About United Urology Group
United Urology Group is a national management services organization whose member groups of urology practices include: Arizona Urology Specialists with locations across the greater Phoenix area; Chesapeake Urology, with offices located throughout Maryland and Delaware; Tennessee Urology, based in Knoxville, TN; and Colorado Urology, located in the greater Denver, Boulder and Front Range areas. United Urology Group members' collective staff today number more than 1,400 employees, including 150 physicians. United Urology's vision is to support the creation of a national network of urology affiliates, which will enable urologists to better meet the needs of their patients and provide the highest level of urological care.
Media Contact:
Patricia Schnably, Senior Vice President, Marketing & Communications United Urology Group25 Crossroads Drive, Suite 306, Owings Mills, MD 21117443-738-8107 pschnably@uniteduro.com
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Breakthrough, Non-Invasive Treatment Called Axol Therapy For Erectile Dysfunction And Enhanced Sexual Performance Now Available At Colorado Urology -...
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.
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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Stem Cell Therapy Market Scope and Opportunities Analysis 2017 2025 - Instant Tech News
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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Scientists May Have Found A Way To Boost The Body's Ability To Heal Itself - IFLScience
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.
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.
See more here:
New sickle cell disease treatments are helping people live longer and giving them a higher quality of life - PhillyVoice.com
CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells – Pharmacy Times
By daniellenierenberg
CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells
T cells and B cells are 2 primary cell types in our adaptive immune system, the source of immunological memory protecting us from subsequent pathogen exposure. B cells secrete pathogen-specific antibodies, which neutralize pathogens directly, or tag them for attack by other immune cells. T cells destroy pathogenic cells directly as well as secrete cytokines to attract additional immune cells.
Chimeric antigen receptor (CAR) T cells are patient derived T cells genetically manipulated to express an artificial transmembrane receptor. The artificial receptor is engineered from modular parts to bind to a surface protein (also called an antigen) on malignant cells and activate the T cell via engineered T cell signaling switches on the CAR.
Current FDA-approved CAR-T cell therapies express CARs recognizing CD19, which is expressed on the surface of almost all B cells, making these therapies specific for B-cell malignancies. Following binding with a CD19-expressing cell, the CAR T cell is activated to proliferate, eliminate the CD19-expressing cell, and persist within the patient.
Tisagenlecleucel (Kymriah, Novartis) is approved to treat pediatric and young adult patients (up to age 25) with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (R/R ALL, ELIANA trial) and adult patients with R/R diffuse large B-cell lymphoma (R/R DLBCL, JULIET trial) after 2 or more lines of systemic therapy. Axicabtagene ciloleucel (Yescarta, Kite Pharma) is approved for adult patients with R/R large B-cell lymphoma (R/R DLBCL, ZUMA-1 trial, NCT02348216) after 2 or more lines of systemic therapy. There are approximately 700 new cases of pediatric and young adult R/R ALL annually in the United States. New cases of R/R DLBCL are approximately 7000 annually in the United States.
CARs can be engineered to recognize virtually any cell surface antigen and can be expressed in a variety of immune cells, suggesting that product development will result in many modular CAR units with vast application versatility. Many different antigen and cell type combinations are already currently in development to address several cancers, such as CAR T cells for pancreatic cancer (NCT03323944).
The first step in creating these personalized genemodified cell therapies is collecting patient lymphocytes via leukapheresis at a clinic or infusion center. Lymphocytes include T cells, B cells, and natural killer cells. The leukapheresis process lasts up to 4 hours and must be coordinated with the patients continuing care regimen to ensure sufficient T cells. The lymphocytes are cryopreserved and immediately shipped to a centralized manufacturing facility.
T cells are separated from the other cells in the leukapheresis product and genetically manipulated, typically using a lentiviral gene delivery method to carry DNA encoding the CAR protein, resulting in CAR T cells. The CAR T cells are cultured to a patient-specific appropriate dose. As this process is finishing, the manufacturer coordinates with the patients health care team to ensure the patient and team are prepared to infuse the CAR T cells. The manufacturing process from the apheresis process to the clinical CAR-T cell product varies widely from patient to patient, from 14 days up to a few months. The limiting step is typically reaching the appropriate CAR-T cell dose.
About a week before the scheduled CAR-T cell infusion, the patient receives multiple days of low-dose conditioning chemotherapy. This step serves to deplete lymphocytes before administration of the CAR T cells, improving the efficacy and persistence of therapy. The CAR T cells are then administered intravenously, and the patient is monitored for adverse events (AEs). The most common AEs with both currently approved products include cytokine release syndrome (CRS), neurological toxicity (NT), hypersensitivity reactions, serious infection, prolonged cytopenias, and hypogammaglobulinemia.3,4 Both products caution that therapy could cause hepatitis B viral reactivation.
The most severe reactions are CRS and NT, both of which can be life threatening. CRS, a common immune reaction following infusion of monoclonal antibodies and CAR T cells, is characterized by fever, nausea, chills, hypotension, tachycardia, asthenia, headache, rash, and dyspnea.5 Mild cases are easily managed, whereas severe cases require more aggressive and invasive therapy, such as mechanical ventilation and intravenous administration of tocilizumab. NT associated with CAR-T cell therapy is characterized by encephalopathy, headache, aphasia, delirium, insomnia, anxiety, tremor, dizziness, seizures, and peripheral neuropathy.
During the clinical trials of Kymriah and Yescarta, CRS and NT occurred in most patients with more than 10% experiencing severe CRS and more than 20% with severe NT.3,4 Initially, the 2 AEs appeared to be independent, but data are beginning to emerge suggesting a correlation. CRS might be a predictor of neurological events; however, neurological events do not predict CRS.6
Real-world evidence from patients treated with Kymriah, presented at the 2019 Society of Hematologic Oncology annual meeting, reported that slightly more than half experienced either of these conditions and less than 20% had severe cases.7 Two-year followup data regarding Yescarta reported severe CRS cases in 11% of patients and severe NT cases in 32%.8 Due to the high rate of occurrence and severity of CRS and NT, both Yescarta and Kymriah have restricted availability through Risk Evaluation and Mitigation Strategies.3,4 Both medications must be administered at a Risk Evaluation and Mitigation Strategiescertified health care facility with health care providers trained in the management and treatment of CRS and 2 doses of tocilizumab available for each patient before CART cell infusion.
Regardless of the potential for severe adverse events (AEs), the benefit of both Yescarta and Kymriah outweigh the risks, as they are highly effective singleadministration therapies. Despite the aggressive nature of the cancers treated with CAR T-cell therapy, meaningful clinical benefit can be achieved within 1 month. A summary of clinical trial primary response rates as well as 2-year data and published real-world data can be found in Table.
Despite differences between the 2 clinically available products, their safety and efficacy profiles in patients with R/R DLBCL are comparable. Differences between the therapies range from the molecular units comprising the CARs, manufacturing differences, lymphodepletion regimen, number of CAR T cells and volume infused, and whether the infusion and short-term patient monitoring occurs in an inpatient or outpatient setting.
Patient-specific genetically modified cell therapies can present many manufacturing challenges. One stark difference between the available therapies, relevant to the patient and clinician experience, has been the manufacturing time and failure rate. During ZUMA-1, of the 101 patients treated with Yescarta, the median time from leukapheresis to product delivery was 17 days (range, 14-51 days), and a 1% manufacture failure rate was reported.4 The ELIANA trial of Kymriah in patients with R/R ALL reported a 9% manufacture failure rate, whereas the JULIET R/R DLBCL trial reported a failure rate of 6.9%, and of the 106 patients receiving Kymriah, the median manufacture time was 113 days (range, 47-196 days).3
Commercial manufacture of Kymriah for DLBCL has struggled to meet specifications.11 While addressing the production issue, Novartis has initiated a safety study evaluating out-of-specification product (NCT04094311) and a managed-access program (NCT03601442).
Known causes of CAR-T cell therapy failure are T cell exhaustion and antigen escape. T cell exhaustion is characterized by a loss of responsive T cells due to changes in gene expression and can be prevented by immune checkpoint inhibitors PD-1, PD-L1, or CTLA- 4. Antigen escape describes a condition in which some cancer cells do not express the CAR-targeted antigen; therefore, they escape immune activation and survive within the patient. Engineering a secondary CAR to a different antigen, such as CD22 in the case of ALL, increases the likelihood of targeting all malignant cells. Solutions to both of these inhibitory mechanisms are currently under clinical trial investigation.12,13
The therapeutic success of CAR T cells ensures gene-modified immune cell therapy will be refined, optimized, and broadly applied until limits are reached. Many clinical groups are investigating biomarkers associated with severe AEs to provide an additional layer of precision care to the CAR-T cell therapy model.6,14 In addition, clinical trials are underway evaluating combination therapies to enhance the efficacy and improve the safety of CART cell therapy. Early-stage research is evaluating the possibility of off-the-shelf CAR-T cell therapy, not a patient-unique manufactured product, to reduce the time to treatment and achieve manufacturing efficiencies and consistencies.15,16 Gene-modified cell therapy, such as CAR T-cell therapy, is revolutionizing oncology, and this living drug model is breathing life into the hopes of patients with cancer and caregivers.
Excerpt from:
CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells - Pharmacy Times
Stress and Gray Hair – ThirdAge
By daniellenierenberg
Medical ResearchThe belief that acute stress can turn hair gray is a popular one, but until now it hasnt been scientifically proven.
But findings that appeared in the publication Nature indicate that the belief may be more than a myth. The study, which used mice as models, was funded in part by the National Institutes of Healths National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other NIH components.
Hair color is determined by cells called melanocytes, which produce the pigment melanin. New melanocytes are made from melanocyte stem cells that live within the hair follicle at the base of the hair strand. As we age, these stem cells gradually disappear. The hair that regrows from hair follicles that have lost melanocyte stem cells has less pigment and appears gray.
A research team, led by Dr. Ya-Chieh Hsu of Harvard University, used mice to examine stress and hair graying. The mice were exposed to three types of stress involving mild, short-term pain, psychological stress, and restricted movement. All caused noticeable loss of melanocyte stem cells and hair graying.
Having established a link between stress and graying, the scientists then explored several potential causes, including the role of the stress hormone corticosterone, but altering its levels didnt affect stress-related graying.
The researchers eventually turned to the neurotransmitter noradrenaline, which, along with corticosterone, was elevated in the stressed mice. They found that noradrenaline, also known as norepinephrine, was key to stress-induced hair graying. By injecting noradrenaline under the skin of unstressed mice, the researchers were able to cause melanocyte stem cell loss and hair graying.
Noradrenaline is produced mostly by the adrenal glands. However, mice without adrenal glands still showed stress-related graying. Noradrenaline is also the main neurotransmitter of the sympathetic nervous system, which is responsible for the fight-or-flight reaction in response to stress.
Ultimately, the team discovered that signaling from the sympathetic nervous system plays a critical role in stress-induced graying. Sympathetic nerves extend into each hair follicle and release noradrenaline in response to stress. Normally, the melanocyte stem cells in the follicle are dormant until a new hair is grown. Noradrenaline causes the stem cells to activate.
Using fluorescent labelling, the researchers observed the stem cells change to melanocytes and migrate away from their reserve in the hair follicle. With no remaining stem cells, no new pigment cells can be made, and any new hair becomes gray, then white.
When we started to study this, I expected that stress was bad for the body but the detrimental impact of stress that we discovered was beyond what I imagined, Hsu says. After just a few days, all of the melanocyte stem cells were lost. Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.
The authors highlight the need to further study the interactions between the nervous system and stem cells in different tissues and organs. A news release from the NIH said that the knowledge gained in this work will be useful in future investigations into the impact of stress on the body and the development of new interventions.
Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD – Benzinga
By daniellenierenberg
Patient Analyses and Safety Data Continue to Underscore Positive Impact of KD025 in cGVHD
Pre-NDA Meeting with FDA Planned for March 2020; Topline Results of Primary Analysis to be Announced in Q2 2020
NEW YORK, NY / ACCESSWIRE / February 23, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced expanded results from the previously reported interim analysis of ROCKstar (KD025-213), its ongoing pivotal trial of KD025 in chronic graft-versus-host disease (cGVHD). The data were presented today in the oral latebreaker session at the 2020 Transplantation & Cellular Therapy (TCT) Meetings.
As announced in November 2019, KD025 met the primary endpoint of Overall Response Rate (ORR) at the study's planned interim analysis, two months after completion of enrollment. KD025 showed statistically significant and clinically meaningful ORRs of 64% with KD025 200 mg once daily (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (95% CI: 54%, 78%; p<0.0001). In the expanded KD025-213 dataset presented today, ORRs were consistent with the previously reported interim analysis across key subgroups, including in patients with four or more organs affected by cGVHD (n=69; 64%), patients who had prior treatment with ibrutinib (n=45; 62%) and patients who had prior treatment with ruxolitinib (n=37; 62%). Three patients achieved a Complete Response. Responses were observed in all affected organ systems, including in organs with fibrotic disease. KD025 has been well tolerated: adverse events were consistent overall with those expected to be observed in cGVHD patients receiving corticosteroids, and no apparent increased risk of infection was observed. Additional secondary endpoints, including duration of response, corticosteroid dose reductions, Failure-Free Survival, Overall Survival and Lee Symptom Scale reductions continue to mature and will be available later in 2020.
"KD025 has been well tolerated and has already demonstrated high response rates in patients with severe and complex cGVHD after a median of five months of follow-up," said Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine, Harvard Medical School; Medical Director, Adult Stem Cell Transplantation Program, Dana-Farber Cancer Institute and a KD025-213 study investigator and Steering Committee member.
"We are extremely pleased with the interim outcomes of this pivotal trial of KD025 in cGVHD, which track closely our findings from our earlier Phase 2 study. KD025 achieved robust response rates across all subgroups of this difficult-to-treat patient population, who had a median of four prior lines of therapy, and 73% of whom had no response to their last line of treatment," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We plan to meet with the FDA for a pre-NDA meeting in March 2020 and to announce topline results from the primary analysis of this trial in Q2 2020."
At the TCT Meetings, Kadmon also presented long-term follow-up data from KD025-208, its ongoing Phase 2 study of KD025 in cGVHD (Abstract #15205). These data were recently presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition in December 2019.
About the ROCKstar (KD025-213) Trial
KD025-213 is an ongoing open-label trial of KD025 in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive KD025 200 mg once daily or KD025 200 mg twice daily, enrolling 66 patients per arm. Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%.
While the ORR endpoint was met at the interim analysis, which was conducted as scheduled two months after completion of enrollment, topline data from the primary analysis of the KD025-213 study, six months after completion of enrollment, will be reported in Q2 2020. Full data from the primary analysis will be submitted for presentation at an upcoming scientific meeting.
About KD025
KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates immune response as well as fibrotic pathways. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209). KD025 was granted Breakthrough Therapy Designation and Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy.
About cGVHD
cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD, and approximately 5,000 new patients are diagnosed with cGVHD per year.
About Kadmon
Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Our clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.
Forward Looking Statements
This press release contains forward-looking statements. Such statements may be preceded by the words "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "targets," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) our reliance on the success of our product candidates; (iv) the timing or likelihood of regulatory filings and approvals; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe, China, Japan and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel (xx) the potential benefits from any of our product candidates being granted orphan drug or breakthrough designation; (xxi) the future trading price of the shares of our common stock and impact of securities analysts' reports on these prices; and/or (xxii) other risks and uncertainties. More detailed information about Kadmon and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the U.S. Securities and Exchange Commission (the "SEC"), including the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2018 and subsequent Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC's website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.
Contact Information
Ellen Cavaleri, Investor Relations646.490.2989ellen.cavaleri@kadmon.com
SOURCE: Kadmon Holdings, Inc.
View source version on accesswire.com: https://www.accesswire.com/577466/Kadmon-Announces-Expanded-Results-of-Interim-Analysis-of-Pivotal-Trial-of-KD025-in-cGVHD
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Kadmon Announces Expanded Results of Interim Analysis of Pivotal Trial of KD025 in cGVHD - Benzinga
Stress could be a major cause of grey hair – BOL News
By daniellenierenberg
Stress may play a key role in just how quickly hair goes from colored to ashen.
Scientists have long understood some link is possible between stress and grey hair.
But this new research more deeply probes the exact mechanisms at play.
The researchers initial tests looked closely at cortisol, the stress hormone that surges in the body when a person experiences a fight or flight response.
Its an important bodily function, but the long-term presence of heightened cortisol links to a host of negative health outcomes.
But the culprit ended up being a different part of the bodys fight or flight response the sympathetic nervous system.
These nerves are all over the body, including making inroads to each hair follicle, the researchers reported.
Chemicals released during the stress response causes pigment producing stem cells to activate prematurely, depleting the hairs reserves of color.
The detrimental impact of stress that we discovered was beyond what I imagined, a lead study author said.
After just a few days, all of the pigment-regenerating stem cells were lost.
Once theyre gone, you cant regenerate pigments anymore. The damage is permanent.
But stress isnt the only reason that most people get grey hair.
In most cases, its simple genetics.
Gray hair caused by loss of melanocytes (pigment cells) in the hair follicle.
This happens as we age and, unfortunately, there is no treatment that can restore these cells and the pigment they produce, melanin, a dermatologist told.
Genetic factors determine when you go grey.
There is nothing that can be done medically to prevent this from happening when it is genetically predetermined to happen.
That doesnt mean environmental factors such as stress dont play a role.
Smoking, for instance, is a known risk factor for premature graying.
So kick the habit if you want to keep that color a little longer.
Other contributing factors to premature graying include deficiencies in protein, vitamin B-12, copper, and iron as well as aging due in part to an accumulation of oxidative stress.
That stress prompted by an imbalance between free radicals and antioxidants in your body that can damage tissue, proteins, and DNA.
And some degree of oxidative stress is a natural part of life.
Changes you can pursue to delay premature grays include eating a diet high in omega-3 fatty acids such as walnuts and fatty fish.
It doesnt spend too much time in the skin-damaging and hair-damaging ultraviolet light of the sun, and taking vitamin B-12 and vitamin B-6 supplements.
That said, if you are going gray prematurely, it wouldnt hurt to go have a checkup just in case natural genetic factors arent the sole culprit.
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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Experimental study speeds up bone healing with 2 common medications - New Atlas
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.
Here is the original post:
New hope for the healing of spinal column injuries with generally available drugs - The Washington Newsday
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.
Link:
Community give child a hero's homecoming - Thehour.com
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.
Originally posted here:
Leukemia: Cancer Of The Blood - Modern Ghana
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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Adoption of Hematology Analyzer Reagent Market to Soar Across Top Countries in the Globe Forecast to 2015 to 2021 - Lake Shore Gazette
34 years with a new heart and counting – MDJOnline.com
By daniellenierenberg
Whenever Harry Wuest has a doctors appointment in northern Atlantas hospital cluster dubbed Pill Hill, he makes sure to stop by the office of Dr. Douglas Doug Murphy for a quick chat.
And Murphy, unless hes tied up in the operating room, always takes a few minutes to say hello to his former patient. Remember when ... ? is how the conversation typically starts, and its always tinged with laughter, often joyful, sometimes bittersweet.
Its a reunion of two men who shaped a piece of Georgias medical history.
Almost 35 years ago, Murphy opened the chest of Wuest and sewed in a new heart, giving him a second shot at life. Wuest was the third heart transplant patient at Emory University Hospital.
Tall, lanky, with short curly hair and a quiet demeanor, Wuest is the longest-surviving heart transplant recipient in Georgia and one of the longest-surviving in the world. The 75-year-old accountant still plays golf twice a week and only recently went from working full-time to part-time. My heart is doing just fine, he says.
Murphy is now the chief of cardiothoracic surgery at Emory Saint Josephs Hospital and still in the operating room almost every day. He has moved on to become the worlds leading expert in robotically assisted heart surgery.
Harry Wuest is originally from Long Island, New York. After a stint in the Air Force, he moved to Florida to work and go to school. He wanted to become a physical education teacher. Then, in 1973, he fell ill. It started with some pain on his left side. He didnt think much of it, but when he got increasingly winded and fatigued, he went to see a doctor.
Several months and numerous specialists later, he received the diagnosis: Cardiomyopathy, a disease of the heart muscle that can make the heart become enlarged, thick and rigid, preventing it from pumping enough blood through the body.
They didnt know how I got it, says Wuest, sitting back in a brown leather armchair in the dark, wood-paneled living room of his Stone Mountain home. Maybe it was a virus. And back then, there wasnt much they could do to treat it, except bed rest.
For the next 12 years, Wuest lived life as best as he could. He got a degree in accounting from the University of Central Florida and worked for a real estate developer. There were good days, but there were more bad days. He was often too weak to do anything, and his heart was getting bigger and bigger.
Emorys first transplant surgeon
The first successful human-to-human heart transplant was performed in Cape Town, South Africa, in 1967 a medical breakthrough that catapulted the surgeon, Dr. Christiaan Barnard, onto the cover of Life magazine and to overnight celebrity status.
This highly publicized event was followed by a brief surge in the procedure around the world, but overall, heart transplants had a rocky start. Most patients died shortly after the surgery, mainly due to organ rejection. Back then, immunosuppressive drugs, which can counteract rejection, were still in their infancy. Many hospitals stopped doing heart transplants in the 1970s.
That changed with the discovery of a highly effective immunosuppressive agent. Cyclosporine got FDA approval in 1983 and altered the world of organ transplants.
It was shortly thereafter when Emory University Hospital decided to launch a heart transplant program, but none of the senior surgeons wanted to do it. Even with the new drug, it was a risky surgery, and mortality was still high.
Its an all-or-nothing operation, Murphy says, as he sits down in his small office overlooking the grayish hospital compound. Hes wearing light blue scrubs from an early morning surgery. At 70, he still has boyish looks, with a lean build and an air of laid-back confidence. If you have a number of bad outcomes initially, it can be detrimental to your career as a surgeon, he says.
But Murphy didnt really have a choice. He remembers that during a meeting of Emorys cardiac surgeons in 1984, he was paged to check on a patient. When he returned, the physicians congratulated him on being appointed the head of the new heart transplant program. He was the youngest in the group and had been recruited from Harvards Massachusetts General Hospital just three years before.
Yeah, thats how I became Emorys first transplant surgeon, says Murphy.
He flew to California to shadow his colleagues at Stanford University Hospital, where most heart transplants were performed at the time. Back home at Emory, he put together a team and rigorously rehearsed the operation. The first transplant patient arrived in April 1985. The surgery was successful, as was the second operation less than a month later.
Around the same time, Harry Wuest wound up in a hospital in Orlando. He needed a transplant, but none of the medical centers in Florida offered the procedure. One of his doctors recommended Emory, and Wuest agreed. I knew I was dying. I could feel it. He was flown to Atlanta by air ambulance and spent several weeks in Emorys cardiac care unit until the evening of May 23, when Murphy walked into his room and said, Weve got a heart.
I could finally breathe again
The heart, as the patient later learned, came from a 19-year-old sophomore at Georgia Tech who had been killed in a car crash.
Organ transplants are a meticulously choreographed endeavor, where timing, coordination and logistics are key. While Murphy and his eight-member team were preparing for the surgery, Wuest was getting ready to say farewell to his family his wife and three teenage sons, and to thank the staff in the cardiac ward.
I was afraid, he recalls, especially of the anesthesia. It scared the heck out of me. He pauses during the reminiscence, choking briefly. I didnt know if I was going to wake up again.
The surgery took six hours. Transplants usually happen at night because the procurement team, the surgeons who retrieve different organs from the donor, only start working when regularly scheduled patients are out of the operating room.
Despite the cultural mystique surrounding the heart as the seat of life, Murphy says that during a transplant surgery, its not like the big spirit comes down to the operating room. Its very technical. As the team follows a precise routine, emotions are kept outside the door. We dont have time for that. Emotions come later.
Waking up from the anesthesia, Wuests first coherent memory was of Murphy entering the room and saying to a nurse, Lets turn on the TV, so Harry can watch some sports.
Wuest spent the next nine days in the ICU, and three more weeks in the hospital ward. In the beginning, he could barely stand up or walk, because he had been bedridden weeks before the surgery and had lost a lot of muscle. But his strength came back quickly. I could finally breathe again, he says. Before the surgery, he felt like he was sucking in air through a tiny straw. I cannot tell you what an amazing feeling that was to suddenly breathe so easily.
Joane Goodroe was the head nurse at Emorys cardiovascular post-op floor back then. When she first met Wuest before the surgery, she recalls him lying in bed and being very, very sick. When she and the other nurses finally saw him stand up and move around, he was a whole different person.
In the early days of Emorys heart transplant program, physicians, nurses and patients were a particularly close-knit group, remembers Goodroe, whos been a nurse for 42 years and now runs a health care consulting firm. There were a lot of firsts for all of us, and we all learned from each other, she said.
Wuest developed friendships with four other early transplant patients at Emory, and he has outlived them all.
When he left the hospital, equipped with a new heart and a fresh hunger for life, Wuest made some radical changes. He decided not to return to Florida but stay in Atlanta. Thats where he felt he got the best care, and where he had found a personal support network. And he got a divorce. Four months after the operation, he went back to working full-time: first in temporary jobs and eventually for a property management company.
After having been sick for 12 years, I was just so excited to be able to work for eight hours a day, he recalls. That was a big, big deal for me.
At 50, he went back to school to get his CPA license. He also found new love.
Martha was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.
Harry Wuest and his wife, Martha. She was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.
Having worked in the transplant office, I saw the good and the bad, Martha Wuest says. A petite woman with short, perfectly groomed silver hair, she sits up very straight on the couch, her small hands folded in her lap. Not every transplant patient did as well as Harry. And I had a lot of fear in the beginning. Now he may well outlive her, she says with a smile and a wink.
Wuests surgeon, meanwhile, went on to fight his own battles. Two and a half years into the program, Murphy was still the only transplant surgeon at Emory and on call to operate whenever a heart became available. Frustrated and exhausted, he quit his position at Emory and signed up with Saint Josephs (which at the time was not part of the Emory system) and started a heart transplant program there.
At St. Josephs, Murphy continued transplanting hearts until 2005. In total, he did more than 200 such surgeries.
Being a heart transplant surgeon is a grueling profession, he says, and very much a younger surgeons subspecialty.
He then shifted his focus and became a pioneer in robotically assisted heart surgery. He has done more than 3,000 operations with the robot, mostly mitral valve repairs and replacements more than any other cardiac surgeon in the world.
Heart transplants "remain the gold standard"
Since Murphy sewed a new heart into Wuest 35 years ago, there has been major progress in the field of heart transplants, but it has been uneven.
There is improved medication to prevent rejection of the donor heart, as well as new methods of preserving and transporting donor hearts.
Yet patients requiring late-stage heart failure therapy, including transplantation, still exceed the number of donor hearts available. In 2019, 3,551 hearts were transplanted in the United States, according to the national Organ Procurement and Transplantation Network. But 700,000 people suffer from advanced heart failure, says the American Heart Association.
New technologies and continued research are providing hope to many of these patients. There has been significant progress in the development of partial artificial hearts, known as Left Ventricular Assist Devices, or LVADs. They can be used as bridge devices, to keep patients alive until donor hearts are available, or as destination therapy, maintaining patients for the remainder of their lives.
Also, total artificial hearts have come a long way since the first artificial pump was implanted in a patient in 1969. The technology is promising, says Dr. Mani Daneshmand, the director of Emorys Heart & Lung Transplantation Program. But its not perfect.
Long-term research continues into xenotransplantation, which involves transplanting animal cells, tissues and organs into human recipients.
Regenerative stem cell therapy is an experimental concept where stem cell injections stimulate the heart to replace the rigid scar tissue with tissue that resumes contraction, allowing for the damaged heart to heal itself after a heart attack or other cardiac disease. Certain stem cell therapies have shown to reverse the damage to the heart by 30 to 50 percent, says Dr. Joshua Hare, a heart transplant surgeon and the director of the Interdisciplinary Stem Cell Institute at the University of Miamis Miller School of Medicine.
All of these ideas have potential, says Daneshmand. But none of them are ready to replace a human donor heart. A heart transplant remains the gold standard, because you cant accommodate the same success with a machine right now, he says.
Efforts around expanding the donor pool are really the best way to address this problem, while we wait for technology to catch up, he adds.
Besides Emory, other health care systems in Georgia that currently have a heart transplant program are Piedmont Healthcare, Childrens Healthcare of Atlanta and Augusta University Health.
Organ rejection remains a major issue, and long-term survival rates have not improved dramatically over the past 35 years. The 10-year survival is currently around 55 percent of patients, which makes long-term survivors like Harry Wuest rare in the world of heart transplants.
The United Network of Organ Sharing, or UNOS, which allocates donor hearts in the United States, doesnt have comprehensive data prior to 1987. An informal survey of the 20 highest-volume hospitals for heart transplants in the 1980s found only a scattering of long-term survivors.
In for the long haul
Being one of the longest-living heart transplant recipients is something that Wuest sees as a responsibility to other transplant patients, but also to the donors family, which hes never met. If you as a transplant recipient reject that heart, thats like a second loss for that family.
Part of this responsibility is living a full and active life. Both he and Martha have three children from their previous marriages and combined they have 15 grandchildren. Most of their families live in Florida, so they travel back and forth frequently. Wuest still works as a CPA during tax season, and he does advocacy for the Georgia Transplant Foundation. In addition to golf, he enjoys lifting weights and riding his bike.
Hes had some health scares over the years. In 2013, he was diagnosed with stage 1 kidney cancer, which is in remission. Also, he crossed paths with his former surgeon, and not just socially. In 2014, Murphy replaced a damaged tricuspid valve in Wuests new heart. That operation went well, too.
Murphy says there are several reasons why Wuest has survived so long. Obviously, his new heart was a very good match. But a patient can have the best heart and the best care and the best medicines and still die a few months or years after the transplantation, the surgeon says. Attitude plays a key role.
Wuest was psychologically stable and never suffered from depression or anxiety, Murphy says. Hes a numbers guy. He knew the transplant was his only chance, and he was set to pursue it.
Wuest attributes his longevity to a good strong heart from his donor; good genetics; great doctors and nurses; and a life that he loves. Im just happy to be here, he says.
Quoting his former surgeon and friend, he adds: Doug always said, Having a transplant is like running a marathon. And Im in for the long haul.
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34 years with a new heart and counting - MDJOnline.com
NASA to send equipment to International Space Station to research Improving Shoes, Showers, 3D Printing – Clarksville Online
By daniellenierenberg
Houston, TX A variety of science investigations, along with supplies and equipment, launch to the International Space Station on the 20th SpaceX commercial resupply services mission.
The Dragon cargo spacecraft is scheduled to leave Earth March 2nd from Space Launch Complex 40 at Cape Canaveral Air Force Station in Florida. Its cargo includes research on particle foam manufacturing, water droplet formation, the human intestine and other cutting-edge investigations.
Airbus workers unpack the Bartolomeo platform at NASAs Kennedy Space Center in Florida in preparation for its launch to the International Space Station. The platform, manufactured by Airbus Defence and Space, hosts multiple external payloads in low-Earth orbit. (NASA)
The space station, now in its 20th year of continuous human presence, provides opportunities for research by government agencies, private industry, and academic and research institutions.
Such research supports Artemis, NASAs missions to the Moon and Mars, and leads to new technologies, medical treatments and products that improve life on Earth.
Particle foam molding is a manufacturing process that blows thousands of pellets into a mold where they fuse together. The shoe company Adidas uses this process to make performance midsoles, the layer between the sole of a shoe and the insole under your foot, for its products.
The BOOST Orbital Operations on Spheroid Tesellation (Adidas BOOST) investigation looks at how multiple types of pellets behave in this molding process. Using one type of pellet creates a foam with the same properties throughout the sole component. Using multiple pellet types can allow engineers to change mechanical properties and optimize shoe performance and comfort. Removing gravity from the process enables a closer look at pellet motion and location during the process.
Results of this investigation could demonstrate the benefits of microgravity research for manufacturing methods, contributing to increased commercial use of the space station. New processes for particle foam molding could benefit a variety of other industries, including packaging and cushioning materials.
The Bartolomeo facility, created by ESA (European Space Agency) and Airbus, attaches to the exterior of the European Columbus Module. Designed to provide new scientific opportunities on the outside of the space station for commercial and institutional users, the facility offers unobstructed views both toward Earth and into space.
Airbus is collaborating with the United Nations Office of Outer Space Affairs to offer UN Member States the opportunity to fly a payload on Bartolomeo. Developing countries are particularly encouraged to participate, and the mission is devoted to addressing the UNs Sustainable Development Goals. Bartolomeo is named for the younger brother of Christopher Columbus.
Droplet Formation Studies in Microgravity (Droplet Formation Study) evaluates water droplet formation and water flow of Delta Faucets H2Okinetic showerhead technology. Reduced flow rates in shower devices conserve water, but also can reduce their effectiveness.
That can cause people to take longer showers, undermining the goal of using less water. Gravitys full effects on the formation of water droplets are unknown, and research in microgravity could help improve the technology, creating better performance and improved user experience while conserving water and energy.
Insight gained from this investigation also has potential applications in various uses of fluids on spacecraft, from human consumption of liquids to waste management and use of fluids for cooling and as propellants.
Human intestine cells forming microvilli inside Emulates Intestine-Chip. (Emulate)
Organ-Chips as a Platform for Studying Effects of Space on Human Enteric Physiology (Gut on Chip) examines the effect of microgravity and other space-related stress factors on biotechnology company Emulates human innervated Intestine-Chip (hiIC). This Organ-Chip device enables the study of organ physiology and diseases in a laboratory setting. It allows for automated maintenance, including imaging, sampling, and storage on orbit and data downlink for molecular analysis on Earth.
A better understanding of how microgravity and other potential space travel stressors affect intestine immune cells and susceptibility to infection could help protect astronaut health on future long-term missions. It also could help identify the mechanisms that underlie development of intestinal diseases and possible targets for therapies to treat them on Earth.
Self-assembly and self-replication of materials and devices could enable 3D printing of replacement parts and repair facilities on future long-duration space voyages. Better design and assembly of structures in microgravity also could benefit a variety of fields on Earth, from medicine to electronics.
Called self-assembled colloidal structures, these are vital to the design of advanced optical materials, but control of particle density and behavior is especially important for their use in 3D printing. Microgravity provides insight into the relationships among particle shape, crystal symmetry, density and other characteristics.
Functional structures based on colloids could lead to new devices for chemical energy, communication, and photonics.
The Multi-use Variable-g Platform (MVP) used for the MVP Cell-03 experiment, shown with the MVP door removed and two carousels inside. (Techshot Inc.)
Generation of Cardiomyocytes From Human Induced Pluripotent Stem Cell-derived Cardiac Progenitors Expanded in Microgravity (MVP Cell-03) examines whether microgravity increases the production of heart cells from human-induced pluripotent stem cells (hiPSCs).
HiPSCs are adult cells genetically reprogrammed back into an embryonic-like pluripotent state, which means they can give rise to several different types of cells. This makes them capable of providing an unlimited source of human cells for research or therapeutic purposes.
For MVP Cell-03, scientists induce the stem cells to generate heart precursor cells, then culture those cells on the space station for analysis and comparison with cultures grown on Earth.
These heart cells or cardiomyocytes (CMs) could help treat cardiac abnormalities caused by spaceflight. In addition, scientists could use them to replenish cells damaged or lost due to cardiac disease on Earth and for cell therapy, disease modeling and drug development. Human cardiac tissues damaged by disease cannot repair themselves, and loss of CMs contributes to eventual heart failure and death.
These are just a few of the hundreds of investigations currently aboard the orbiting laboratory. For daily updates, follow @ISS_Research, Space Station Research and Technology News or our Facebook. Follow the ISS National Lab for information on its sponsored investigations. For opportunities to see the space station pass over your town, check out Spot the Station.
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NASA to send equipment to International Space Station to research Improving Shoes, Showers, 3D Printing - Clarksville Online
On the other hand – J-Wire Jewish Australian News Service
By daniellenierenberg
February 23, 2020 by Michael Kuttner
Read on for article
As we hurtle towards round three in our general elections the frenetic canvassing of voters by desperate political parties is in stark contrast to the switched-off interest shown by those targeted.
Thankfully the same cannot be said for the daily announcements of further advances in good news whether it is scientific, medical or social spheres. Israeli ingenuity continues to be a light unto the nations.
QUICKER RESULTS EQUALS QUICKER DIAGNOSIS
The Israeli innovation can test 100 saliva samples in 15 minutes as opposed to one blood test that takes an hour to confirm coronavirus.
Quick diagnosis can help prevent the spread ofcoronavirus by slashing the timeit takes to decide that patients need to be quarantined and treated.
The technology is already in use for diagnosing the Zika virus and is used at Israels Tel Hashomer Hospital in Ramat Gan by the Ministry of Healths central virology laboratory.
ANOTHER ADVANCE IN CARDIAC CARE
Researchers succeeded in producing 3D engineered cardiac tissues from chamber-specific heart cells derived from human stem cells. This medical development opens the door for creating personalized medications for cardiac patients and advances in new cardiac drug developments.
This research model simulates the most common irregular heartbeat (arrhythmia), called atrial fibrillation. It opens the door for testing the success of various drugs on individual patients to prevent or stop arrhythmia.
Because they were able to separate atrial and ventricular tissue models, researchers can discover which drugs improve atrial cell function without damaging ventricular cell function.
DEFEATING CYBER HACKERS
Researchers from Ben-Gurion University (BGU) presented at the Cybertech Global Tel Aviv conference the first all-optical stealth encryption technology. The innovation uses fibre-optic light transmissions to secure cloud computing and data centre network transmission.
The technology uses standard optical equipment to send data in a manner that cannot beintercepted by hackers, unlike conventional digital methods. Another aspect of the system is that data gets destroyed if a hacker tries to decode it.
Because an eavesdropper can neither read the data nor even detect the existence of the transmitted signal, the optical stealth transmission provides thehighest level of privacy and securityfor sensitive data applications.
The patented technology has multiple applications, including high-speed communication and sensitive transmission of financial, medical or social media-related information. According to the Senior Vice President, Exact Sciences & Engineering, BGN Technologies, An eavesdropper will require years to break the encryption key.
TWO THOUSAND YEARS LATER
Long after the Romans departed archeological discoveries continue to be made. Two thousand years later the descendants of the Jews they tried to ethnically cleanse, now restored in their homeland, walk again in the very places they were once exiled from.
RECLAIMING A LOST HERITAGE
One of the miracles one witnesses by living in Israel is meeting Jews long lost to their heritage somehow finding their way back to their Faith and People.
Whether it is the Bnei Menashe from India, tribes from Uganda, individuals from Kaifeng, China or Jews from Ethiopia the common theme is of a return to Zion.
Often overlooked but now becoming a frequent occurrence is the discovery by descendants of Conversos, those driven underground or forcibly converted by the Spanish and Portuguese Inquisition five hundred years ago, of their Jewish heritage.
Watch this moving video of one of the latest such personal dramas. We truly are living in amazing times when lost Jews from the four corners of the world are returning.
Visit J-Wire's main page for all the latest breaking news, gossip and what's on in your community.
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On the other hand - J-Wire Jewish Australian News Service