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Distinguished physician-scientist takes the helm of first Frost Institute – University of Miami
By daniellenierenberg
Trained as a chemist, biophysicist, internist, and cardiologist, Mark Yeager is eager to propel the Frost Institute for Chemistry and Molecular Science into a leading research center.
Even in his youth Mark Yeager could picture the door to his future. Scuffed, chipped, and almost black from layers of varnish, the old, wooden door had a frosted window with five words stenciled in glossy black: Laboratory of Dr. Mark Yeager.
Yet Yeager, the inaugural executive director of the University of Miamis Frost Institute for Chemistry and Molecular Science (FICMS), is quite happy that his new lab in the 94,000-square-foot building slated to open late next year wont even have a door. The $60 million facilitys open floor plan was designed to encourage the free flow of people and ideasand help transform the University into one of the worlds premier research centers for improving the health of humans and that of our planet.
That is the vision, but its not a fantastical vision, said Yeager, a distinguished biophysicist and cardiologist whose top priority is attracting a diverse and elite group of scientists as the institutes first faculty. It is achievable, and it will happen because the University has not wavered in its commitment to elevate STEM (science, technology, engineering, and mathematics) to advance scientific discovery. Theres something going on here thats organic and alive and excitingand Im thrilled to be part of it.
Yeager, whose own groundbreaking research focuses on the molecular causes of heart disease and viral infections, trained as a chemist at Carnegie-Mellon University, as a physician and biophysicist at Yale University and as an internist and cardiologist at Stanford University. He spent two decades at Scripps Research in California, where he established his first independent laboratory, served as the director of research in cardiology, and helped launch the Skaggs Clinical Scholars Program in Translational Research. He has also served as a consultant and scientific and clinical advisor to several biotech companies.
Now he is transitioning to the University from the University of Virginia School of Medicine (UVA), where he chaired the Department of Molecular Biophysics and Biochemistry for nearly a dozen years and helped establish the Sheridan G. Snyder Translational Research Building. At UVA, he also established one of the nations five regional centers for cryo-electron microscopy (cryoEM)the technique he advanced for flash-freezing, imaging, and studying proteins and other macromolecules in their near-natural state.
It is exciting to see the progress being made on the evolution of our Frost Institutes, starting with Data Science and Computing and now the emergence of Chemistry and Molecular Science. We are fortunate to have Mark overseeing our Frost Institute for Chemistry and Molecular Science and working across the entire institutionhis interdisciplinary knowledge and perspective on chemistry are essential for our success, said Jeffrey Duerk, executive vice president for academic affairs and provost. Mark brings a wealth of knowledge and experience to the University of Miami and we are looking forward to his impactful leadership continuing as we move forward.
Yeager said he knew he was making the right career move on his first visit to the University last November. Although the COVID-19 pandemic had curtailed in-person learning and suspended new construction, he heard the unmistakable sound of heavy equipment as he walked past the royal palms and fountain at the end of Memorial Drive, where the five-story FICMS now stands.
I could see an excavation area and heard a cacophony of construction noise where I had a hunch the institute should be, he recalled. That told me that the University was all in. They had made this commitment to fortify STEM and to do transformational science and nothing was going to stop them. In spite of the pandemic, it was all systems go.
The Universitys longtime benefactors, Phillip and Patricia Frost, enabled that commitment in 2017, when they announced their landmark $100 million gift to establish the Frost Institutes for Science and Engineering, now a key initiative of the Roadmap to Our New Centurythe strategic plan guiding the University toward its centennial mark. The umbrella organization for a group of multidisciplinary research centers patterned after the National Institutes of Health and its network of affiliated institutes, the Frost Institutes were envisioned to translate interdisciplinary research into solutions for real-world problems.
Though Yeager officially started his new role on June 1, he has been heavily involved in planning the FICMS' interior for months. He recently placed a $20 million order to equip the facility with five different electron microscopy instruments that chemists, molecular scientists, and engineers will use to explore the molecular structure of exquisitely beam-sensitive soft materials like proteins, hard materials such as metal alloys, as well as nanomaterials comprised of soft and hard components. Along with the buildings state-of-the-art technology and the Universitys research infrastructure, hes confident its location in the heart of the Coral Gables campus will help him recruit a diverse and elite group of scientists who are exploring challenging avenues of impactful researchsomething he has been driven to do almost his entire life.
An occasional songwriter, guitar player, and jogger who in his younger days ran 18 marathons, Yeager was always fascinated by scientific discoveries that illuminated unknown and unseen worlds. A child of the Sputnik era who began entering science fairs in junior high, he began forging his own career as a physician-scientist while in high school in Colorado Springs, Colorado, where his father, an agricultural economist, settled his family after a number of job-related moves.
Inspired by an experiment in Scientific American magazine, he convinced physicians in the therapeutic radiology department at Penrose Hospital to irradiate his fruit flies so he could compare the effects of administering different doses of radiation on their eye pigments. Delivered in Styrofoam cups, his experiments on what is now called dose fractionationand used to reduce tissue damage during cancer treatmentswon him first place in the U.S. Department of Agricultures 1967 International Science Fair and a research stint in an insect toxicology lab in Berkeley, California.
The following summer, when Yeager returned to Penrose Hospital to work as an orderly, he realized that he loved patient care as much as laboratory research and began plotting how he could pursue both careers.
I just got incredible satisfaction from helping patients get out of bed and into a wheelchair, transfer to a gurney, learn to use crutches, recalled Yeager, who joins the University as one of its 100 Talents for 100 Years, a Roadmap initiative to add 100 new endowed chairs to the faculty by the Universitys 2025 centennial. But I also loved chemistry. I loved physics. I loved too many things.
After earning his undergraduate degree in chemistry from Carnegie-Mellon, he was accepted to the Medical Scientist Training Program at Yale University, where, along with his medical degree, he earned his masters degree and doctorate in molecular biophysics and biochemistry. There, he encountered the first of many trailblazing scientists, including two future Nobel laureates, who would influence his lifes work. His Ph.D. advisor, Lubert Stryer, was particularly influential. Stryer authored a premier textbook of biochemistry, pioneered fluorescence-based techniques to explore the motions of biological macromolecules, and made fundamental discoveries on the molecular basis of vision. Yeagers graduate work on rhodopsin, a photoreceptor membrane protein, triggered his fascination with elucidating the molecular bases for such diseases as sudden cardiac death, heart attacks, HIV-1, and other viral infections.
Yeager completed his medical residency and specialized fellowship training in cardiovascular medicine at Stanford University Medical Center, where he managed the pre- and post-operative care of heart transplant patients and wrote 13 chapters in the book Handbook of Difficult Diagnoses.
He also continued exploring cellular biology in the laboratory of Nigel Unwin, who had collaborated with future Nobel laureate Richard Henderson to pioneer the use of cryoEM to determine the molecular structure of membrane proteinsand inspired Yeagers groundbreaking research on gap junction channels. The electrical conduits that connect every cell in the body to its neighbor, gap junction channels play a critical role in maintaining the normal heartbeat.
That research, which Yeager continued at Scripps and at UVA, explained how gap junction channels behave in their normal state, and during an injured state, such as a heart attack. His quest to answer another question particularly relevant todayhow viruses enter host cells, replicate, and assemble infectious particlesis exemplified by his breakthrough research on the assembly, structure, and maturation of HIV-1, the virus that causes AIDS.
Today, those insights, which Yeager humbly calls a few bricks in the edifice of science, hold important clues for developing new, more effective therapies to prevent HIV-1 infection, repair injured tissue, and treat cancer and cardiovascular diseasethe kind of impactful research that the FICMS was designed to advance with collaborative partners across the University, and beyond.
As a pioneer in the field of cryo-transmission electron microscopy, a forefront technology in materials and biological research, Marks expertise and knowledge will position the University as aleader in these cutting-edge fields, said Leonidas Bachas, dean of the College of Arts and Sciences who served as the initial interim director of the FICMS. We look forward to having him lead the Frost Institute for Chemistry and Molecular Science as we continue to advance the sciences, innovate, and expand research collaborations with our faculty and industry partners.
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Distinguished physician-scientist takes the helm of first Frost Institute - University of Miami
The Covid booster shot is not for everyone. It’s only meant for severely immunocompromised people – ETHealthworld.com
By daniellenierenberg
The increasing prevalence of new coronavirus variants is raising questions about how well protected those who've already had their COVID-19 shots are against evolving forms of the SARS-CoV-2 virus. Here, microbiology and infectious disease specialist William Petri of the University of Virginia answers some common questions about COVID-19 booster shots.
1. What is a booster shot?Boosters are an extra dose of a vaccine given to maintain vaccine-induced protection against a disease. They are commonly used to bolster many vaccines because immunity can wear off over time. For example, the flu vaccine needs a booster every year, and the diphtheria and tetanus vaccine every 10 years.
Boosters are often identical to the original vaccine. In some cases, however, the booster shot has been modified to enhance protection against new viral variants. The seasonal flu vaccine, most notably, requires an annual booster because the flu virus changes so rapidly.
3. Why aren't booster shots recommended for everyone yet?While vaccine-induced immunity may not last forever, it is not clear when a booster will be needed.
Encouragingly, all of the currently authorized COVID-19 vaccines induce a robust immune memory against the coronavirus. The vaccine teaches your immune system's memory B cells to produce antibodies when you're exposed to the virus. Researchers have detected high levels of memory B cells in the lymph nodes of people who received the Pfizer vaccine for at least 12 weeks after they got the shot.
Studies also suggest that authorized COVID-19 vaccines are continuing to offer protection even against emerging strains of the coronavirus. Among one study's participants, the Johnson & Johnson vaccine had 73% and 82% efficacy 14 days and 28 days post shot, respectively, at warding off severe disease from the beta variant. Another study found the Pfizer vaccine to be 88% effective against the delta variant.
4. How will I know if I need a booster?You may need to wait for an outbreak in people who have been vaccinated. Researchers are still figuring out the best way to measure the strength of someone's vaccine-induced immunity. The COVID-19 vaccines have been so effective that there are not many failures to test.
The best candidate to measure are certain antibodies the vaccine induces the immune system to make. They recognize the spike protein that allows the coronavirus to enter and infect cells. Evidence supporting the importance of anti-spike antibodies includes a study showing that the somewhat more effective mRNA vaccines like Pfizer and Moderna generate higher antibody levels in the blood than the adenovirus vector vaccines like Johnson & Johnson and AstraZeneca. In a preliminary study that has not yet been peer-reviewed, anti-spike antibody levels were lower in people who caught COVID-19 after they were vaccinated with the Oxford-AstraZeneca vaccine.
Medical workers would love to be able to give patients a blood test that would tell them how well protected they are or aren't against COVID-19. That would be a clear indication as to whether a booster shot is needed.
But until researchers know for sure how to measure vaccine-induced immunity, the next indication that boosters may be needed are breakthrough infections in older adults who have already been vaccinated. People over the age of 80 make lower levels of antibodies after vaccination, so their immunity may wane sooner than that of the general population. The elderly would also most likely be the most susceptible to new viral variants that evade the protection current vaccines provide.
5. Who does the FDA and CDC recommend get a third shot?An extra shot may be necessary for certain immunocompromised people. In one study, 39 of 40 kidney transplant recipients and a third of dialysis patients failed to make antibodies after vaccination. Another study identified 20 patients with rheumatic or musculoskeletal diseases on medications that suppress the immune system who also did not have detectable antibodies. Both of these studies were done after patients received the full vaccine dose.
Currently, the CDC recommends that the following people consider getting a third dose:
Those who are immunocompromised may wonder if the vaccine they received is successfully generating immunity in their body. A preliminary study that has not yet been peer-reviewed did find that a test that specifically targets the anti-spike antibodies the vaccines trigger may be helpful in determining whether the vaccine worked. But for now, the FDA does not recommend antibody tests to assess immunity.
6. Does my third dose need to match my first two?Likely not. Recent research has shown that mRNA vaccines, like Pfizer and Moderna, can be mixed with adenovirus-based vaccines like AstraZeneca with comparable results.
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The Covid booster shot is not for everyone. It's only meant for severely immunocompromised people - ETHealthworld.com
First-of-its-Kind Bio-Artificial Pancreas on Track for Type-I Diabetes Cure – Global Trade Magazine
By daniellenierenberg
Imagine a world where those living with Type 1 Diabetes, a chronic illness affecting more than 60 million adults globally, no longer had to deal with regular blood glucose monitoring, daily insulin injections or life-threatening nighttime hypoglycemic events, but instead could eat, exercise and sleep worry-free. Thats the kind of future an up-and-coming breakthrough technology is on track to creating.
Beta-O2 Technologies, a privately held biomedical company headquartered in Israel with research and industry affiliates across the U.S., is working to deliver a first-of-its-kind bio-artificial pancreas as a safe, effective and long-term cure for the disease. With preliminary animal trials showing promising results for its second generation breakthrough device, called Bio-artificial Pancreas (Air), the company is planning to begin human clinical trials within the year.
We have strong pre-clinical evidence to prove the safe operation of our device on animals, said Beta-O2 CEO Amir Lichter, noting that the second generation Air is performing well in ongoing animal studies. Its an enormous achievement that is paving the road for human trials.
Measuring approximately 2.5 by 2.5 inches, Air is made of titanium. It has two components: a macrocapsule that contains pancreatic cells and an oxygen tank equipped with an external port, so patients can easily refresh oxygen levels weekly. Once implanted under a patients skin, it becomes a natural source of insulin, sensing blood glucose levels and delivering insulin as required.
While there are a couple of other artificial pancreatic solutions being explored by different industry players, Beta-O2s disruptive technology is the only bio-artificial pancreas to incorporate an active oxygen supply, necessary to keep the pancreas cells in the implanted device functional and viable over the long term. Other solutions are demonstrating limited success because they rely on a patients bloodstream to deliver enough oxygen to keep the transplanted cells viable, which is problematic, Lichter explained.
Pancreas cells (islets) are extremely delicate, he said. We solve the problem by proactively supplying oxygen through an external source, providing a superior solution.
Lichter said the beauty of the Beta-O2 solution which holds 10 global patents for its exclusive immune protection capabilities and oxygen supply mechanisms is that its very generic, meaning it can contain cells from a human donor, cells from the pancreas of a pig, or cells derived in a lab from stem cells. Other advantages are that Beta-O2s bio-artificial pancreas does not require a patient to take intensive immunosuppression therapies after implant due to its protective encapsulation capabilities, and the device can quickly be retrieved from a patient if necessary due to malfunction or other health concerns, he explained.
Beta-O2 is currently collaborating with several U.S.-based pharmaceutical companies and academics, including researchers from Harvard University, MIT, University of Virginia and Cornell University, to further enhance the Air oxygen supply and its ability to measure glucose levels and secrete insulin once implanted. The company is also in negotiations to solidify its collaboration with several stem cell providers as it looks to secure an additional $15 million in investment funds to support its aggressive go-to-market strategy.
The active oxygen supply used by Beta-O2 is currently the best and most advanced technique for maintaining viability and function of large numbers of pancreaticislets (or stem cell-derived islets) in an encapsulation transplantation device, said Clark K. Colton ofthe Department of Chemical Engineering at MIT andBeta-O2 Scientific Advisory Board member.
Calling the Beta-O2 device a next-gen treatment option, Dr. Jos Oberholzer, Professor of Surgery, Biomedical Engineering and Experimental Pathology at the University of Virginia and Beta-O2 Scientific Advisory Board member, explained that after years of insulin injections and closed-loop insulin pumps and glucose sensors, patients will finally have access to a biological device solution to treat the most brittle forms of diabetes. The Beta-O2 device is the only implant that has shown reproducible results in humans with diabetes, with measurable insulin production originating from human islet cells within the device without the need for recipients to take any immunosuppressive drugs.
An earlier safety trial involving four patients in Sweden, supported by New York-based JDRF (Juvenile Diabetes Research Foundation), successfully demonstrated that Beta-O2s device is fully safe for use. No side effects were observed in patients who carried the device for up to 10 months, and the cells remained viable and functional.
Now, current animal trials underway at Beta-O2 are focused on extending the life of functional cells even further, with promising early results showing that rats implanted with Air are maintaining normal glucose levels.
With tangible evidence that we can maintain the viability and functionality of our cells for a long duration in rats, which have an immune system very similar to humans, we are looking forward to moving ahead with our second round of human clinical trials, Lichter said, noting that the company aims to be first to show that implanted biological pancreatic cells can successfully achieve normal blood sugar levels in diabetic patients without the need for immunosuppression therapy.
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About Beta-O2 Technologies Ltd. (www.beta-o2.com)
Beta-O2 Technologies Ltd. is a biomedical company developing a proprietary implantable bioreactor, the Air, for the treatment of Type 1 Diabetes. Air is designed to address the main problems of the otherwise successful procedures in which islets of Langerhans (i.e. pancreatic endocrine cells) are transplanted in diabetic patients, such as the need for life-long immunosuppressive pharmacological treatment and limited functionality of the transplanted islets over time due to an insufficient oxygen supply. Beta-O2 investors include SCP Vitalife Partners, Sherpa Ventures, Aurum Ventures, Pitango Venture Capital, Saints Capital, Japanese and Chinese private investors.
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First-of-its-Kind Bio-Artificial Pancreas on Track for Type-I Diabetes Cure - Global Trade Magazine
Q&A: Cancer Death Rates Are Falling Nationally. Here’s What’s Happening at UVA – University of Virginia
By daniellenierenberg
This week, the American Cancer Society released some very welcome news: the cancer death rate in the U.S. dropped by 2.2% from 2016 to 2017, the largest single-year drop ever recorded.
The drop, which the report attributes to plummeting smoking rates as well as new screening and treatment methods, continues a decades-long trend, as cancer death rates have fallen by nearly 30% since 1991 about 2.9 million fewer deaths.
Dr. Thomas Loughran, director of the University of Virginia Cancer Center, said UVA is in step with this national trend.
The UVA Cancer Center is one of 71 National Cancer Institute-designated treatment centers nationwide and ranked among the nations top 50 cancer centers over each of the past four years (No. 26 last year). The center serves approximately 4 million people in Virginia and West Virginia.
We spoke with Loughran about what he is seeing at UVA and beyond, new treatments and research helping to eradicate cancer, and where he sees cancer treatment in five years.
Q. Why have cancer death rates dropped so significantly?
A. As reports of this latest drop have said, a large part of the decline can be attributed to declining rates of lung cancer. The importance of preventing cancer particularly behavioral interventions like stopping smoking has become more prominent, and there have been remarkable declines in smoking across the United States.
This is a very important focus for us at UVA. We serve a large geographical area 90 contiguous counties in Virginia and West Virginia, including rural Appalachia. Southwest Virginia in Appalachia still has high smoking rates, and as a result, high rates of lung cancer. Education, screening and tobacco cessation programs are critically important, especially in those areas.
Q. What advances in treatment have contributed to falling cancer death rates, nationally and at UVA?
A. Screening technology, especially for the more common cancers like lung, colorectal, prostate and breast cancer, has improved. The latest report probably doesnt fully reflect recent implementation of lung cancer screening using a low-dose CT scan, recommended for high risk individuals and especially those with a history of heavy smoking. That has only been around a few years, and its impact will likely show up in future reports.
The second big factor is the development of immunotherapy [cancer treatments that utilize and help the patients immune system]. UVA has invested quite a lot of institutional resources in becoming a state-of-the-art immunotherapy center, and I am proud to say we are a leader in the field.
We have created a Cancer Therapeutics Program to support the development of new therapies. Dr. Craig Slingluff, who leads that program, is a surgical oncologist internationally famous for immunotherapy treatments for melanoma. To strengthen this program, we have recruited a cadre of leading physician scientists from across the country. Dr. Karen Ballen came here to lead our stem cell and bone marrow transplant program. Dr. Lawrence Lum, the scientific director of the transplant program, has developed a novel therapy using antibodies that bind to both T-cells [patient cells that can kill cancer cells] and tumor cells, forming a bridge between the two that helps the T-cells kill the cancer cells. Dr. Trey Lee is a leader in CAR-T cell therapy.
I could keep going; there are so many great people working on this. We also have a new Good Manufacturing Practice lab, supported by a grant from the commonwealth, that will help us grow and modify T-cells as needed and give them to patients under sterile conditions. That just opened and we are very excited about that program.
Q. What other areas of research have shown great promise?
A. Some of our work in nanotechnology is really unique and exciting. [Biomedical engineering professor] Mark Kester directs UVAs nanoSTAR Institute, which is working on delivering cancer therapies by nanotechnology basically, engineering at a very small scale. For example, nanoliposomes a sort of delivery system for cancer therapy are actually smaller than individual cells and can therefore penetrate cancer cells and release treatment from inside those cells.
We are very excited about early phase trials testing this technology on solid tumors, and we also hope to use it to treat patients with acute leukemia over the next few years.
Q. Looking ahead, where do you see the next big gains coming from?
A. Immunotherapy has revolutionized cancer treatment, but why some patients respond well and some dont remains puzzling. I hope that we can begin to discover why some patients are reacting to these newer treatments differently than others. Once we figure out why some patients respond to immunotherapy, we can begin to make improvements that could benefit a larger percentage of patients with these deadly cancers.
CAR T Cell therapy one method of immunotherapy is very effective against leukemia, lymphoma and cancers of the blood, but not yet against solid tumors. Over the next five years, I hope we can determine how to deliver these T-cells to solid tumors such as those found in lung, colorectal and other common cancers again to make this advance more widely applicable to a larger number of patients.
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Q&A: Cancer Death Rates Are Falling Nationally. Here's What's Happening at UVA - University of Virginia
A Discussion With Jennifer Delgado on Life After Cancer and Weathering the Storm – Thrive Global
By daniellenierenberg
JenniferDelgado grew up in St. Louis, Missouri. She attended Webster University, whereshe received her Bachelor of Arts in Media Communications. She then went to MississippiState University, where she received a Bachelor of Science in Geosciences witha concentration in Broadcast Meteorology.
In 2006,Jennifer Delgado worked as a morning and noon meteorologist for WTVR-TV inRichmond, Virginia. Then in 2008, she began working at CNN International inAtlanta, Georgia, as their primary meteorologist, as well as a fill-inmeteorologist on all CNN networks. In 2010, she won a Peabody Award for CNNscoverage on the Deepwater Horizon oil spill in the Gulf of Mexico.
In 2013,Delgado was hired as a co-host of AMHQ (Americas Morning Headquarters) at TheWeather Channel. She anchored continuous coverage of breaking news and weatherevents, including live interviews with state and local officials, experts andresidents. She was also their fill-in co-host of Wake-Up with Al.
JenniferDelgado began freelancing as a meteorologist/anchor for WXIA-TV in 2017. Shepresented weathercasts every six minutes during a two-hour morning newscast andproduced weathercasts for radio, web, and the 24-hour weather channel.
Two yearsago, Jennifer Delgado was diagnosed with blood cancer. She underwent treatmentand received a bone marrow/stem cell transplant. Since the transplant, she hasbeen receiving treatment at the Emory Winship Cancer Institute and advocatingfor cancer awareness and more bone marrow donors.
No one is ever prepared tohear the words, you have cancer. It literally blew up my world. I had to stopworking because beating cancer became my full-time job. I knew something waswrong for months based on my symptoms. I was tiredall the time, my bones were aching, had migraines, vertigo andconfusion. Dealing with any illness is stressful, especially if you arent ableto work. Some people say cancer changed their life for the better; however, Idont want to credit cancer for anything positive. It was a wake-up call. Lifeis short, and you have to enjoy every moment.
I immediately went into adeep depression. I hid and only shared the news with my close friends andfamily. I was trying to hide the awful chemo port in my chest and made excuses for my appearanceand fatigue. It was very stressful. I think anyone dealing with a seriousmedical condition should reach out to people going through the same battle. I got some amazing tips from fellow blood cancersurvivors on Instagram and Facebook support groups. I have formed many closebonds and when I am feeling down they completely understand. Cancer patients caneasily go through their savings in a short amount of time. I was lucky to haveamazing health insurance but not everyone is that fortunate. There is a lot of grant money out there forpeople struggling financially. The Leukemia & Lymphoma Society is anamazing organization and helps patients with everything from financial help,information on clinical trials etc.
If you are strong enough, Isay its important to be your own health advocate. You know your body best. Ialso suggest if you have one, reaching out to a friend or family member whoworks in medicine (nurse, PA, doctor) to be your medical advocate. The advocatecan come to your appointments or even join a conference call during yourappointments when you need help understanding your treatment options. I waslucky to have both my mom and one of my best friends to help me interpreteverything. Never be afraid to ask your doctor questions, and dont forgetabout the physicians assistant, who often has more availability.
I was going back and forthto the doctor for nearly a year, and they keep dismissing my symptoms. At onepoint, one doctor told me to take probiotics. I finally decided it was time toget a second opinion when I was having trouble walking. Luckily, I found Dr.Drew Freilich, whom I credit with saving my life. He recognized that mysymptoms were severe and insisted that I needed an MRI. Thats how theydiscovered I had a blood cancer that was attacking my bones. I could havebecome disabled if I had waited longer to get help. If you know something iswrong, you have to be persistent about getting answers.
I know it sounds clich, butmy friends, family, and neighbors. They all took excellent care of me. Theydrove me to the hospital for chemotherapy or bone marrow biopsies. My friends were great and woulddrop by to bring me food or help clean up myhouse.
I know it may sound sillybut my dogs really helped keep my spirits up. Quite often, it was just me and the dogs and duringisolation. I truly believe that pets are healing, and studies show that havingone improves your mental health. There were several weeks when I had to be awayfrom my dogs because my immune system was too weak. I was lucky enough to havegreat friends watch my fur babies. I even tried to convince my friends to driveby Emory Hospital so that I could see them.
I would say you have to bepositive. It seems like its a long way away, and you wonder at times whetheror not everything you did is going to pay off when you finally get toremission. So, I think you have to be positive because you get very paranoid. Ibelieve positive thinking can be healing and improve your health. Keeping inmind that everyones journey is different, I think its also important to see apsychologist or therapist. Sometimes its easier to share your real concernswith a stranger. We always try and put on a brave face for family and friends.
Aftereverything, I felt like I had to give back to the cancer community and EmoryWinship Cancer Center. I got my dogs certified to be Happy Tails therapydogs, and now we visit patients battling cancer while they are getting chemo.Its amazing and emotional all at the same time. Many times, patients will say,your puppy made my day.
Iam also trying to raise awareness for the need of more bone marrow donors.Right now, the majority of donors come from Europe. It would be awesome if morepeople would register to be a bone marrow donor. Its a simple swab test. Ithink its a small price to pay, considering more than 170,000 people arediagnosed with blood cancer every year. Check out Be The Match or The Leukemia& Lymphoma Society.
I am not going to sugarcoatit, staying motivated is extremely challenging and a daily battle. I thinkevery cancer survivor questions, why did this happen to me? Is it gone? How longwill I stay in remission? It can be quite depressing, but you have to live forthe day and stick to a routine. I try to remind myself that there is a reasonwhy I am still alive, and I want to give back to others who are struggling.
Everything. I had months ofchemo to get my cancer level down enough to collect my stem cells for thetransplant. I wondered constantly, will I be in remission? And then once Iwas in remission, how long will I stay in remission before I relapse? Whenyoure dealing with blood cancers, most have no cure. So, theres always thatchance of relapse, and youre always worrying about it.
I did six rounds of chemobefore I was even ready to get a transplant. The stem cell transplant wassomething I was dreading because of the high dose of chemotherapy and losing myhair. That can be a very difficult experience, especially for women. After thosesix rounds, they collected my stem cells, which is not a fun process. Then theyprepped me, and I had the transplant.
After, I was in isolation atthe hospital for three weeks. Then I went home, and I was still under isolationfor another 100+ days. I felt like I was ready to lose my mind. During thistime, your white blood cells are regenerating, which means you dont have animmune system, and you suffer from extreme fatigue and pain. Walking up a shortflight of stairs would wipe me out. I couldnt eat salads, fruits, basicallyanything raw. When I left the house, Id have to wear a mask to protect myimmune system. I really hated that because everyone would stare and pretty muchknew I had cancer.
However, to put a positivespin on it, because of my time in isolation at home, I really felt my creativejuices start to flow. I began brainstorming and thinking of a lot of differentthings because life is short, and the cancer was my wake-up call.
So, my best advice duringthat period is to make a reading list and binge-watch shows on Netflix. I readthe Game of Thrones series. Iliterally had a calendar counting down to 100 days. Thats also the time whenyour hair finally starts to grow back!
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A Discussion With Jennifer Delgado on Life After Cancer and Weathering the Storm - Thrive Global
World Cord Blood Day 2019 to Welcome Leading Transplant Doctors and Pioneering Cellular Therapy Researchers – Yahoo Finance
By Dr. Matthew Watson
Registration is now open for World Cord Blood Day 2019 (November 15th) including the official online conference (free event) which will feature numerous cord blood transplant doctors and cellular therapy researchers such as Dr. Joanne Kurtzberg, Dr. Karen Ballen, Dr. Elizabeth Shpall, Dr. Wise Young and Dr. Filippo Milano. In addition, free educational events will be held around the globe.
TUCSON, Ariz., Oct. 9, 2019 /PRNewswire/ -- World Cord Blood Day (November 15th, 2019) is a free event and open to the public. In addition to events worldwide, World Cord Blood Day will feature a free online conference. Renowned researchers and leading transplant doctors will give introductory presentations for the public as well as academic lectures specifically designed for healthcare professionals.
Attendees will learn about the 40,000+ cord blood transplants performed since 1988 to treat over 80 life-threatening diseases including sickle cell anemia, thalassemia, lymphoma and leukemia. In addition, attendees will learn about exciting advances in the emerging field of regenerative medicine to potentially treat autism, cerebral palsy, spinal cord injury and more.
As the host and organizer of World Cord Blood Day 2019, Save the Cord Foundation is proud to announce the following speakers for this year's program (in order of appearance): Dr. Joanne Kurtzberg (Duke Department of Pediatrics, Duke Center for Autism and Brain Development), Dr. Karen Ballen (University of Virginia), Dave Murphy and Monroe Burgess (Quick Specialized Healthcare Logistics), Dr. Wise Young (Rutgers University), Dr. Elizabeth Shpall (MD Anderson Cancer Center), Dr. Filippo Milano (Fred Hutchinson Cancer Research Center). In addition, attendees will hear from Dr. Alexes Harris who beat cancer thanks to a cord blood transplant from a donor and young Luke Fryer who was treated for cerebral palsy with his own cord blood in a clinical trial.
The morning session will focus on the success of cord blood transplants over the past 30 years and how transplant doctors use cord blood stem cells today, namely, to fight blood cancer. The afternoon session will look at new directions in cord blood research. Attendees will receive updates on several ground-breaking clinical trials using cord blood in regenerative medicine, cellular therapy and more. To view the full agenda, please visit: https://www.worldcordbloodday.org/online-medical-conference-agenda.html
Organized and hosted by Save the Cord Foundation (501c3 non-profit), this year's event is officially sponsored by Quick Specialized Healthcare Logistics. Inspiring Partners include the Cord Blood Association (CBA), Be the Match (NMDP), World Marrow Donor Association (WMDA-Netcord), AABB Center for Cellular Therapy and Foundation for the Accreditation of Cellular Therapy (FACT).
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Visit http://www.WorldCordBloodDay.org to learn how you can participate and/or host an event. Join us on social media using the hashtags: #WCBD19 and #WorldCordBloodDay.
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Tests show no signs of cancer for Danville 2-year-old – GoDanRiver.com
By JoanneRUSSELL25
Two-year-old Nathan DeAndrea who underwent two stem cell transplants to treat neuroblastoma is free of cancer, according to his mother.
Testing last week that included a CT scan and a full-body scan showed no evidence of cancer, Shannon DeAndrea said during an interview at her home Monday morning.
No more cancer! said Nathans sister, 4-year-old Kailynn.
However, the DeAndreas are awaiting the results of a bone marrow biopsy performed on Nathan last week, Shannon said. Everyone is optimistic.
The doctor said he has never seen a bone marrow biopsy come back positive when everything else is clear, she said.
Results are expected this week, Shannon said.
Nathan was diagnosed with stage 4 neuroblastoma on Aug. 23, 2016. He had a tumor in his abdomen that spread to his bone marrow. He had spots on his skull, ribs and spine. He has had several rounds of chemotherapy, radiation and two stem cell transplants.
Neuroblastomas are cancers that begin in early nerve cells of the sympathetic nervous system, according to the American Cancer Society.
The scans results brought relief to Shannon and her family.
Its like I could breathe, she said.
As Kailynn put it, We said, hooray!
The next phase of treatment will include strengthening Nathans immune system. He will be in the hospital one week a month for six months, Shannon said.
Its to keep it [the cancer] from coming back, she said.
His immune system is still compromised. The genetic makeup of Nathans tumor put him at a higher risk of relapse, Shannon said.
Nathans first transplant included four or five days of chemo. The new stem cells following the chemo that killed off his old stem cells from the transplant were like a rescue, she said.
Its wiping you out and then giving you your cells back to restart your immune system, DeAndrea said.
A second round of heavy chemo was to try to kill what was left of the cancer and replenish cells, she said.
Nathans stem cell transplants were from his own cells, Shannon said.
Two types of stem cell transplants include autologous, which uses stem cells from the patients own body, and allogeneic using stem cells from another person.
The procedure is used for conditions including multiple myeloma, lymphoma, sickle cell anemia and leukemia, and other blood and immune disorders.
Stem cell transplants began in the late 50s/early 60s with the first successful procedure done in an identical twin. However, stem cell transplants were limited until medicines that prevent rejections became available.
The number of procedures increased in the 1980s.
Betsie Letterle, community engagement representative with BeTheMatch in Burlington, North Carolina, said there are more than 14 million bone marrow/stem cell donors in the BeTheMatch registry.
Bone marrow transplants traditionally involved taking the marrow from the back of the donors hip. But since then, weve progressed tremendously, Letterle said.
The newest way is to take stem cells from a vein in the donors arm, Letterle said. The donor receives an injection of medication to help their body manufacture a large amount of stem cells, she said.
Those are taken from the vein, similar to a plasma donation. Letterle said.
Anyone aged 18-44 can join the registry, but commitment is paramount among donors, she said.
Commitment is important because patients depend on us, Letterle said. We dont want anyone whos not really sure they could donate if called.
Only about one in 540 registered donors end up donating, she said. Everyone is an active donor until they turn 61, Letterle said.
Younger donors are healthier and make the most stem cells, she said.
We want to give the patient the optimum opportunity to get the best stem cells they can, she said.
If a donor comes up as a match, they will be asked for about 20-30 hours of their time over several weeks, Letterle said.
We work around the donors schedule, she said.
They get blood work done, and a physical to make sure theyre healthy enough to donate, Letterle said.
The donor never pays for anything, she said.
The doctor determines whether the procedure would be a stem cell or a traditional bone marrow transplant. That depends on the patients or recipients age and condition, Letterle said.
About 80 percent of registered donors are Caucasian, and BeTheMatch is looking for more minority donors, Letterle said. Many minority patients have trouble finding a match, she said.
The recipients blood type becomes whatever blood type the donor has, Letterle said.
Dr. William Clark with the Massey Cancer Center at Virginia Commonwealth University will speak about bone marrow and stem cell transplants from 11:30 a.m. to 1 p.m. July 11 at Ballou Recreation Center. A bone marrow/stem cell donor drive will also be held that day.
For more information on stem cell/bone marrow transplants, call Betsie Letterle at BeTheMatch at (877) 601-1926, ext. 7721.
JohnCrane reports for the Danville Register & Bee. Contact him atjcrane@registerbee.comor(434) 791-7987.
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Tests show no signs of cancer for Danville 2-year-old - GoDanRiver.com
After two stem cell transplants and several rounds of chemo, ‘now he’s just like a normal 2-year-old’ – GoDanRiver.com
By daniellenierenberg
When Shannon DeAndrea saw a knot on her 18-month-old sons head last July, she thought he had just fallen.
But more popped up and wouldnt go away. He also began feeling sick.
I finally decided he needed to see a pediatrician, said DeAndrea, who lives in Blairs.
She was told he had ear infections and her son, Nathan, was put on rounds of antibiotics. The knots were normal, she was told.
Another medical provider said he looked anemic. Blood work revealed his hemoglobin was dangerously low.
We ended up in the ER, DeAndrea said. They couldnt figure out why he was anemic.
Shannon and Nathan were sent to Roanoke, where he was diagnosed with a stage 4 neuroblastoma on Aug. 23. He had a tumor in his abdomen that spread to his bone marrow. He had spots on his skull, ribs and spine.
Neuroblastomas are cancers that begin in early nerve cells of the sympathetic nervous system, according to the American Cancer Society.
Since his diagnosis, her son now 2 has had several rounds of chemotherapy and two stem cell transplants and is doing well.
Now hes just like a normal 2-year-old, DeAndrea said. Hes running around with his sister. Hes eating well.
Dr. William Clark is associate professor of medicine and attending physician at Virginia Commonwealth University Massey Cancer Center Stem Cell Transplantation Program. Clark said the procedure is used for conditions including multiple myeloma, lymphoma, sickle cell anemia and leukemia.
Stem cell transplants are used to replace bone marrow that has been destroyed by cancer or destroyed by the chemo and/or radiation used to treat the cancer, according to the American Cancer Society.
High doses of chemo (sometimes along with radiation), work better than standard doses to kill cancer cells. However, high doses can also kill the stem cells and cause the bone marrow to stop making blood cells, which are needed for life. The transplanted stem cells replace the bodys stem cells after the bone marrow and its stem cells have been destroyed by treatment, according to the American Cancer Society.
Two types of stem cell transplants include autologous, which uses stem cells from the patients own body, and allogeneic using stem cells from another person, Clark said.
For leukemia patients, most of the time, we give them stem cells from someone else, Clark said. Chemotherapy helps lower the leukemia disease burden, but the new immune system provided by the new stem cells can fight against the cancer cells and get rid of them, he said.
Virginia Commonwealth Universitys cancer center performs an average of about 160-195 stem cell transplants per year, Clark said. Slightly more than half are autologous procedures, and the rest are allogeneic, he said.
Whitt Clement, former delegate who represented the Danville area in the General Assembly, underwent a stem cell transplant for acute myeloid leukemia in September 2015.
The most important aspect for patients is being self-aware and their own best advocates, Clement said.
My experience was that the patient has to ask a lot of questions throughout the process, he said.
He suspected something was wrong when he noticed his platelet count declining over seven years. He went to a hematologist and had a bone marrow biopsy that revealed his condition.
If I had not taken the initiative myself and gone to see a hematologist, matters would have progressed to the point where I would have been symptomatic, Clement said.
Finding the perfect match in a donor is also important, Clement said. Fortunately, he had a sibling who met all the criteria and donated stem cells.
A person can get great matches from unrelated donors, but its preferable for a donor to be a sibling, said Clement, partner at Hunton & Williams law firm in Richmond.
Your body has an easier time tolerating the new stem cells, he said.
Clement served in the Virginia House of Delegates from 1988-2002, and as Virginias secretary of transportation from 2002-2005 under Gov. Mark Warner.
For someone with multiple myeloma, the transplant does not cure the disease but delays the time it returns by up to seven and a half years, Clark said.
Lymphoma, leukemia and sickle cell anemia can be cured with the procedure, Clark said. Lymphoma can be cured in about 50 to 80 percent of cases, depending on the lymphoma, Clark said.
The first 30 days after the transplant are the most critical, Clement said. During that time, different organs can have varying reactions to the new cells. It can affect the kidneys, liver, gastrointestinal tract, skin, and cause other side effects.
The idea is that the closer the match, the less likely youll have those adverse reactions, he said.
The process includes being put on an immunosuppressant to prevent the immune system from attacking the new cells, Clement said.
He credits the quality of his recovery to asking lots of questions and being his own advocate tape recording conversations with medical providers, coming in with written questions.
Ive been able to recover better because of that, he said.
Its a long journey and so a person confronted with the transplant situation has got to prepare himself for a long journey that requires a lot of questions along the way, Clement said.
There are about 20 million potential stem cell/bone marrow donors in the BeTheMatch Registry in the United States, Clark said.
Stem cell transplants began in the late 50s/early 60s with the first successful procedure done in an identical twin, Clark said. However, stem cell transplants were limited until medicines that prevent rejections became available.
The number of procedures increased in the 1980s, Clark said.
Danville resident Susan Mathena, cancer patient navigator at Danville Regional Medical Center, became a donor about 20 years ago because she wanted to help people. Mathena has also been an organ donor since she got her drivers license.
I see patients all the time that need stem cell transplants, Mathena said. We always need a source of bone marrow donation.
Though she will age out of the stem cell donor list soon, she could still be contacted if she is the only match for someone in need, she said.
Clark will speak next month on stem cell/bone marrow transplants at Ballou Recreation Center at an event held by the Cancer Research and Resource Center of Southern Virginia in Danville.
Thousands of patients with blood cancers like leukemia or other diseases like sickle cell anemia need a bone marrow/stem cell transplant to survive, including some of our own community members, said Kate Stokely Powell, coordinator at the center.
Clarks presentation offers an opportunity in Southside for people battling illness, medical students and professionals and the public to learn from an expert in the field of stem cell transplants, Powell said.
Doctors, hospitals and families affected by a blood cancer disease have done a great job of building a massive database of blood types for potential donor matches, Clement said.
For DeAndrea and her son, Nathan, the first transplant included four or five days of chemo. The new stem cells following the chemo that killed off his old stem cells from the transplant were like a rescue, she said.
Its wiping you out and then giving you your cells back to restart your immune system, DeAndrea said.
A second round of heavy chemo was to try to kill what was left of the cancer and replenish cells, she said.
It was rough, it was a nightmare, DeAndrea said. It was by far the worst phase of his treatment, but I believe, in the long run, its worth it.
She said the procedures should increase Nathans chances for survival and prevent a relapse.
Nathan just finished radiation Tuesday and will go in for a biopsy of his bone marrow this week, DeAndrea said.
Well find out next week where we stand as far as the cancer goes, she said.
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After two stem cell transplants and several rounds of chemo, 'now he's just like a normal 2-year-old' - GoDanRiver.com
Robot hearts: medicine’s new frontier – The Guardian
By raymumme
On a cold, bright January morning I walked south across Westminster Bridge to St Thomas Hospital, an institution with a proud tradition of innovation: I was there to observe a procedure generally regarded as the greatest advance in cardiac surgery since the turn of the millennium and one that can be performed without a surgeon.
The patient was a man in his 80s with aortic stenosis, a narrowed valve which was restricting outflow from the left ventricle into the aorta. His heart struggled to pump sufficient blood through the reduced aperture, and the muscle of the affected ventricle had thickened as the organ tried to compensate. If left unchecked, this would eventually lead to heart failure. For a healthier patient the solution would be simple: an operation to remove the diseased valve and replace it with a prosthesis. But the mans age and a long list of other medical conditions made open-heart surgery out of the question. Happily, for the last few years, another option has been available for such high-risk patients: transcatheter aortic valve implantation, known as TAVI for short.
This is a non-invasive procedure, and takes place not in an operating theatre but in the catheterisation laboratory, known as the cath lab. When I got there, wearing a heavy lead gown to protect me from X-rays, the patient was already lying on the table. He would remain awake throughout the procedure, receiving only a sedative and a powerful analgesic. I was shown the valve to be implanted, three leaflets fashioned from bovine pericardium (a tough membrane from around the heart of a cow), fixed inside a collapsible metal stent. After being soaked in saline it was crimped on to a balloon catheter and squeezed, from the size and shape of a lipstick, into a long, thin object like a pencil.
The consultant cardiologist, Bernard Prendergast, had already threaded a guidewire through an incision in the patients groin, entering the femoral artery and then the aorta, until the tip of the wire had arrived at the diseased aortic valve. The catheter, with its precious cargo, was then placed over the guidewire and pushed gently up the aorta. When it reached the upper part of the vessel we could track its progress on one of the large X-ray screens above the table. We watched intently as the metal stent described a slow curve around the aortic arch before coming to rest just above the heart.
There was a pause as the team checked everything was ready, while on the screen the silhouette of the furled valve oscillated gently as it was buffeted by pulses of high-pressure arterial blood. When Prendergast was satisfied that the catheter was precisely aligned with the aortic valve, he pressed a button to inflate the tiny balloon. As it expanded it forced the metal stent outwards and back to its normal diameter, and on the X-ray monitor it suddenly snapped into position, firmly anchored at the top of the ventricle. For a second or two the patient became agitated as the balloon obstructed the aorta and stopped the flow of blood to his brain; but as soon as it was deflated he became calm again.
Prendergast and his colleagues peered at the monitors to check the positioning of the device. In a conventional operation the diseased valve would be excised before the prosthesis was sewn in; during a TAVI procedure the old valve is left untouched and the new one simply placed inside it. This makes correct placement vital, since unless the device fits snugly there may be a leak around its edge. The X-ray picture showed that the new valve was securely anchored and moving in unison with the heart. Satisfied that everything had gone according to plan, Prendergast removed the catheter and announced the good news in a voice that was probably audible on the other side of the river. Just minutes after being given a new heart valve, the patient raised an arm from under the drapes and shook the cardiologists hand warmly. The entire procedure had taken less than an hour.
According to many experts, this is what the future will look like. Though available for little more than a decade, TAVI is already having a dramatic impact on surgical practice: in Germany the majority of aortic valve replacements, more than 10,000 a year, are now performed using the catheter rather than the scalpel.
In the UK, the figure is much lower, since the procedure is still significantly more expensive than surgery this is largely down to the cost of the valve itself, which can be as much as 20,000 for a single device. But as the manufacturers recoup their initial outlay on research and development, it is likely to become more affordable and its advantages are numerous. Early results suggest that it is every bit as effective as open-heart surgery, without many of surgerys undesirable aspects: the large chest incision, the heart-lung machine, the long period of post-operative recovery.
The essential idea of TAVI was first suggested more than half a century ago. In 1965, Hywel Davies, a cardiologist at Guys Hospital in London, was mulling over the problem of aortic regurgitation, in which blood flows backwards from the aorta into the heart. He was looking for a short-term therapy for patients too sick for immediate surgery something that would allow them to recover for a few days or weeks, until they were strong enough to undergo an operation. He hit upon the idea of a temporary device that could be inserted through a blood vessel, and designed a simple artificial valve resembling a conical parachute. Because it was made from fabric, it could be collapsed and mounted on to a catheter. It was inserted with the top of the parachute uppermost, so that any backwards flow would be caught by its inside surface like air hitting the underside of a real parachute canopy. As the fabric filled with blood it would balloon outwards, sealing the vessel and stopping most of the anomalous blood flow.
This was a truly imaginative suggestion, made at a time when catheter therapies had barely been conceived of, let alone tested. But, in tests on dogs, Davies found that his prototype tended to provoke blood clots and he was never able to use it on a patient.
Another two decades passed before anybody considered anything similar. That moment came in 1988, when a trainee cardiologist from Denmark, Henning Rud Andersen, was at a conference in Arizona, attending a lecture about coronary artery stenting. It was the first he had heard of the technique, which at the time had been used in only a few dozen patients, and as he sat in the auditorium he had a thought, which at first he dismissed as ridiculous: why not make a bigger stent, put a valve in the middle of it, and implant it into the heart via a catheter? On reflection, he realised that this was not such an absurd idea, and when he returned home to Denmark he visited a local butcher to buy a supply of pig hearts. Working in a pokey room in the basement of his hospital with basic tools obtained from a local DIY warehouse, Andersen constructed his first experimental prototypes. He began by cutting out the aortic valves from the pig hearts, mounted each inside a home-made metal lattice then compressed the whole contraption around a balloon.
Within a few months Andersen was ready to test the device in animals, and on 1 May 1989 he implanted the first in a pig. It thrived with its prosthesis, and Andersen assumed that his colleagues would be excited by his works obvious clinical potential. But nobody was prepared to take the concept seriously folding up a valve and then unfurling it inside the heart seemed wilfully eccentric and it took him several years to find a journal willing to publish his research.
When his paper was finally published in 1992, none of the major biotechnology firms showed any interest in developing the device. Andersens crazy idea worked, but still it sank without trace.
Andersen sold his patent and moved on to other things. But at the turn of the century there was a sudden explosion of interest in the idea of valve implantation via catheter. In 2000, a heart specialist in London, Philipp Bonhoeffer, replaced the diseased pulmonary valve of a 12-year-old boy, using a valve taken from a cows jugular vein, which had been mounted in a stent and put in position using a balloon catheter.
In France, another cardiologist was already working on doing the same for the aortic valve. Alain Cribier had been developing novel catheter therapies for years; it was his company that bought Andersens patent in 1995, and Cribier had persisted with the idea even after one potential investor told him that TAVI was the most stupid project ever heard of.
Eventually, Cribier managed to raise the necessary funds for development and long-term testing, and by 2000 had a working prototype. Rather than use an entire valve cut from a dead heart, as Andersen had, Cribier built one from bovine pericardium, mounted in a collapsible stainless-steel stent. Prototypes were implanted in sheep to test their durability: after two-and-a-half years, during which they opened and closed more than 100m times, the valves still worked perfectly.
Cribier was ready to test the device in humans, but his first patient could not be eligible for conventional surgical valve replacement, which is safe and highly effective: to test an unproven new procedure on such a patient would be to expose them to unnecessary risk.
In early 2002, he was introduced to a 57-year-old man who was, in surgical terms, a hopeless case. He had catastrophic aortic stenosis which had so weakened his heart that with each stroke it could pump less than a quarter of the normal volume of blood; in addition, the blood vessels of his extremities were ravaged by atherosclerosis, and he had chronic pancreatitis and lung cancer. Several surgeons had declined to operate on him, and his referral to Cribiers clinic in Rouen was a final roll of the dice. An initial attempt to open the stenotic valve using a simple balloon catheter failed, and a week after this treatment Cribier recorded in his notes that his patient was near death, with his heart barely functioning. The mans family agreed that an experimental treatment was preferable to none at all, and on 16 April he became the first person to receive a new aortic valve without open-heart surgery.
Over the next couple of days the patients condition improved dramatically: he was able to get out of bed, and the signs of heart failure began to retreat. But shortly afterwards complications arose, most seriously a deterioration in the condition of the blood vessels in his right leg, which had to be amputated 10 weeks later. Infection set in, and four months after the operation, he died.
He had not lived long nobody expected him to but the episode had proved the feasibility of the approach, with clear short-term benefit to the patient. When Cribier presented a video of the operation to colleagues they sat in stupefied silence, realising that they were watching something that would change the nature of heart surgery.
When surgeons and cardiologists overcame their initial scepticism about TAVI they quickly realised that it opened up a vista of exciting new surgical possibilities. As well as replacing diseased valves it is now also possible to repair them, using clever imitations of the techniques used by surgeons. The technology is still in its infancy, but many experts believe that this will eventually become the default option for valvular disease, making surgery increasingly rare.
While TAVI is impressive, there is one even more spectacular example of the capabilities of the catheter. Paediatric cardiologists at a few specialist centres have recently started using it to break the last taboo of heart surgery operating on an unborn child. Nowhere is the progress of cardiac surgery more stunning than in the field of congenital heart disease. Malformations of the heart are the most common form of birth defect, with as many as 5% of all babies born with some sort of cardiac anomaly though most of these will cause no serious, lasting problems. The heart is especially prone to abnormal development in the womb, with a myriad of possible ways in which its structures can be distorted or transposed. Over several decades, specialists have managed to find ways of taming most; but one that remains a significant challenge to even the best surgeon is hypoplastic left heart syndrome (HLHS), in which the entire left side of the heart fails to develop properly. The ventricle and aorta are much smaller than they should be, and the mitral valve is either absent or undersized. Until the early 1980s this was a defect that killed babies within days of birth, but a sequence of complex palliative operations now makes it possible for many to live into adulthood.
Because their left ventricle is incapable of propelling oxygenated blood into the body, babies born with HLHS can only survive if there is some communication between the pulmonary and systemic circulations, allowing the right ventricle to pump blood both to the lungs and to the rest of the body. Some children with HLHS also have an atrial septal defect (ASD), a persistent hole in the tissue between the atria of the heart which improves their chances of survival by increasing the amount of oxygenated blood that reaches the sole functioning pumping chamber. When surgeons realised that this defect conferred a survival benefit in babies with HLHS, they began to create one artificially in those with an intact septum, usually a few hours after birth. But it was already too late: elevated blood pressure was causing permanent damage to the delicate vessels of the lungs while these babies still in the womb.
The logical albeit risky response was to intervene even earlier. In 2000, a team at Boston Childrens Hospital adopted a new procedure to create an ASD during the final trimester of pregnancy: they would deliberately create one heart defect in order to treat another. A needle was passed through the wall of the uterus and into the babys heart, and a balloon catheter used to create a hole between the left and right atria. This reduced the pressures in the pulmonary circulation and hence limited the damage to the lungs; but the tissues of a growing foetus have a remarkable ability to repair themselves, and the artificially created hole would often heal within a few weeks. Cardiologists needed to find a way of keeping it open until birth, when surgeons would be able to perform a more comprehensive repair.
In September 2005 a couple from Virginia, Angela and Jay VanDerwerken, visited their local hospital for a routine antenatal scan. They were devastated to learn that their unborn child had HLHS, and the prognosis was poor. The ultrasound pictures revealed an intact septum, making it likely that even before birth her lungs would be damaged beyond repair. They were told that they could either terminate the pregnancy or accept that their daughter would have to undergo open-heart surgery within hours of her birth, with only a 20% chance that she would survive.
Devastated, the VanDerwerkens returned home, where Angela researched the condition online. Although few hospitals offered any treatment for HLHS, she found several references to the Boston foetal cardiac intervention programme, the team of doctors that had pioneered the use of the balloon catheter during pregnancy.
They arranged an appointment with Wayne Tworetzky, the director of foetal cardiology at Boston Childrens Hospital, who performed a scan and confirmed that their unborn childs condition was treatable. A greying, softly spoken South African, Tworetzky explained that his team had recently developed a new procedure, but that it had never been tested on a patient. It would mean not just making a hole in the septum, but also inserting a device to prevent it from closing. The VanDerwerkens had few qualms about accepting the opportunity: the alternatives gave their daughter a negligible chance of life.
The procedure took place at Brigham and Womens Hospital in Boston on 7 November 2005, 30 weeks into the pregnancy, in a crowded operating theatre. Sixteen doctors, with a range of specialisms, took part: cardiologists, surgeons, and four anaesthetists two to look after the mother, two for her unborn child. Mother and child needed to be completely immobilised during a delicate procedure lasting several hours, so both were given a general anaesthetic. The team watched on the screen of an ultrasound scanner as a thin needle was guided through the wall of the uterus, then the foetuss chest and finally into her heart an object the size of a grape.
A guidewire was placed in the cardiac chambers, then a tiny balloon catheter was inserted and used to create an opening in the atrial septum. This had all been done before; but now the cardiologists added a refinement. The balloon was withdrawn, then returned to the heart, this time loaded with a 2.5 millimetre stent that was set in the opening between the left and right atria. There was a charged silence as the balloon was inflated to expand the stent; then, as the team saw on the monitor that blood was flowing freely through the aperture, the room erupted in cheers.
Grace VanDerwerken was born in early January after a normal labour, and shortly afterwards underwent open-heart surgery. After a fortnight she was allowed home, her healthy pink complexion proving that the interventions had succeeded in producing a functional circulation.
But just when she seemed to be out of danger, Grace died suddenly at the age of 36 days not as a consequence of the surgery, but from a rare arrhythmia, a complication of HLHS that occurs in just 5%. This was the cruellest luck, when she had seemingly overcome the grim odds against her. Her death was a tragic loss, but her parents courage had brought about a new era in foetal surgery.
Much of the most exciting contemporary research focuses on the greatest, most fundamental cardiac question of all: what can the surgeon do about the failing heart? Half a century after Christiaan Barnard performed the first human heart transplant, transplantation remains the gold standard of care for patients in irreversible heart failure once drugs have ceased to be effective. It is an excellent operation, too, with patients surviving an average of 15 years. But it will never be the panacea that many predicted, because there just arent enough donor hearts to go round.
With too few organs available, surgeons have had to think laterally. As a result, a new generation of artificial hearts is now in development. Several companies are now working on artificial hearts with tiny rotary electrical motors. In addition to being much smaller and more efficient than pneumatic pumps, these devices are far more durable, since the rotors that impel the blood are suspended magnetically and are not subject to the wear and tear caused by friction. Animal trials have shown promising results, but, as yet, none of these have been implanted in a patient.
Another type of total artificial heart, as such devices are known, has, however, recently been tested in humans. Alain Carpentier, an eminent French surgeon still active in his ninth decade, has collaborated with engineers from the French aeronautical firm Airbus to design a pulsatile, hydraulically powered device whose unique feature is the use of bioprosthetic materials both organic and synthetic matter. Unlike earlier artificial hearts, its design mimics the shape of the natural organ; the internal surfaces are lined with preserved bovine pericardial tissue, a biological surface far kinder to the red blood cells than the polymers previously used. Carpentiers artificial heart was first implanted in December 2013. Although the first four patients have since died two following component failures the results were encouraging, and a larger clinical trial is now under way.
One drawback to the artificial heart still leads many surgeons to dismiss the entire concept out of hand: the price tag. These high-precision devices cost in excess of 100,000 each, and no healthcare service in the world, publicly or privately funded, could afford to provide them to everybody in need of one. And there is one still more tantalising notion: that we will one day be able to engineer spare parts for the heart, or even an entire organ, in the laboratory.
In the 1980s, surgeons began to fabricate artificial skin for burns patients, seeding sheets of collagen or polymer with specialised cells in the hope that they would multiply and form a skin-like protective layer. But researchers had loftier ambitions, and a new field tissue engineering began to emerge.
High on the list of priorities for tissue engineers was the creation of artificial blood vessels, which would have applications across the full range of surgical specialisms. In 1999 surgeons in Tokyo performed a remarkable operation in which they gave a four-year-old girl a new artery grown from cells taken from elsewhere in her body. She had been born with a rare congenital defect which had completely obliterated the right branch of her pulmonary artery, the vessel conveying blood to the right lung. A short section of vein was excised from her leg, and cells from its inside wall were removed in the laboratory. They were then left to multiply in a bioreactor, a vessel that bathed them in a warm nutrient broth, simulating conditions inside the body.
After eight weeks, they had increased in number to more than 12m, and were used to seed the inside of a polymer tube which functioned as a scaffold for the new vessel. The tissue was allowed to continue growing for 10 days, and then the graft was transplanted. Two months later the polymer scaffold around the tissue, designed to break down inside the body, had completely dissolved, leaving only new tissue that would it was hoped grow with the patient.
At the turn of the millennium, a new world of possibility opened up when researchers gained a powerful new tool: stem cell technology. Stem cells are not specialised to one function but have the potential to develop into many different tissue types. One type of stem cell is found in growing embryos, and another in parts of the adult body, including the bone marrow (where they generate the cells of the blood and immune system) and skin. In 1998 James Thomson, a biologist at the University of Wisconsin, succeeded in isolating stem cells from human embryos and growing them in the laboratory.
But an arguably even more important breakthrough came nine years later, when Shinya Yamanaka, a researcher at Kyoto University, showed that it was possible to genetically reprogram skin cells and convert them into stem cells. The implications were enormous. In theory, it would now be possible to harvest mature, specialised cells from a patient, reprogram them as stem cells, then choose which type of tissue they would become.
Sanjay Sinha, a cardiologist at the University of Cambridge, is attempting to grow a patch of artificial myocardium (heart muscle tissue) in the laboratory for later implantation in the operating theatre. His technique starts with undifferentiated stem cells, which are then encouraged to develop into several types of specialised cell. These are then seeded on to a scaffold made from collagen, a tough protein found in connective tissue. The presence of several different cell types means that when they have had time to proliferate, the new tissue will develop its own blood supply.
Clinical trials are still some years away, but Sinha hopes that one day it will be possible to repair a damaged heart by sewing one of these patches over areas of muscle scarred by a heart attack.
Using advanced tissue-engineering techniques, researchers have already succeeded in creating replacement valves from the patients own tissue. This can be done by harvesting cells from elsewhere in the body (usually the blood vessels) and breeding them in a bioreactor, before seeding them on to a biodegradable polymer scaffold designed in the shape of a valve. Once the cells are in place they are allowed to proliferate before implantation, after which the scaffold melts away, leaving nothing but new tissue. The one major disadvantage of this approach is that each valve has to be tailor-made for a specific patient, a process that takes weeks. In the last couple of years, a group in Berlin has refined the process by tissue-engineering a valve and then stripping it of cellular material, leaving behind just the extracellular matrix the structure that holds the cells in position.
The end result is therefore not quite a valve, but a skeleton on which the body lays down new tissue. Valves manufactured in this way can be implanted, via catheter, in anybody; moreover, unlike conventional prosthetic devices, if the recipient is a child the new valve should grow with them.
If it is possible to tissue-engineer a valve, then why not an entire heart? For many researchers this has come to be the ultimate prize, and the idea is not necessarily as fanciful as it first appears.
In 2008, a team led by Doris Taylor, a scientist at the University of Minnesota, announced the creation of the worlds first bioartificial heart composed of both living and manufactured parts. They began by pumping detergents through hearts excised from rats. This removed all the cellular tissue from them, leaving a ghostly heart-shaped skeleton of extracellular matrix and connective fibre, which was used as a scaffold onto which cardiac or blood-vessel cells were seeded. The organ was then cultured in a bioreactor to encourage cell multiplication, with blood constantly perfused through the coronary arteries. After four days, it was possible to see the new tissue contracting, and after a week the heart was even capable of pumping blood though only 2% of its normal volume.
This was a brilliant achievement, but scaling the procedure up to generate a human-sized heart is made far more difficult by the much greater number of cells required. Surgeons in Heidelberg have since applied similar techniques to generate a human-sized cardiac scaffold covered in living tissue. The original heart came from a pig, and after it had been decellularised it was populated with human vascular cells and cardiac cells harvested from a newborn rat. After 10 days the walls of the organ had become lined with new myocardium which even showed signs of electrical activity. As a proof of concept, the experiment was a success, though after three weeks of culture the organ could neither contract nor pump blood.
Growing tissues and organs in a bioreactor is a laborious business, but recent improvements in 3D printing offer the tantalising possibility of manufacturing a new heart rapidly and to order. 3D printers work by breaking down a three-dimensional object into a series of thin, two-dimensional slices, which are laid down one on top of another. The technology has already been employed to manufacture complex engineering components out of metal or plastic, but it is now being used to generate tissues in the laboratory. To make an aortic valve, researchers at Cornell University took a pigs valve and X-rayed it in a high-resolution CT scanner. This gave them a precise map of its internal structure which could be used as a template. Using the data from the scan, the printer extruded thin jets of a hydrogel, a water-absorbent polymer that mimics natural tissue, gradually building up a duplicate of the pig valve layer by layer. This scaffold could then be seeded with living cells and incubated in the normal way.
Pushing the technology further, Adam Feinberg, a materials scientist at Carnegie Mellon University in Pittsburgh, recently succeeded in fabricating the first anatomically accurate 3D-printed heart. This facsimile was made of hydrogel and contained no tissue, but it did show a remarkable fidelity to the original organ. Since then, Feinberg has used natural proteins such as fibrin and collagen to 3D-print hearts. For many researchers in this field, a fully tissue-engineered heart is the ultimate prize.
We are left with several competing visions of the future. Within a few decades it is possible that we will be breeding transgenic pigs in vast sterile farms and harvesting their hearts to implant in sick patients. Or that new organs will be 3D-printed to order in factories, before being dispatched in drones to wherever they are needed. Or maybe an unexpected breakthrough in energy technology will make it possible to develop a fully implantable, permanent mechanical heart.
Whatever the future holds, it is worth reflecting on how much has been achieved in so little time. Speaking in 1902, six years after Ludwig Rehn became the first person to perform cardiac surgery, Harry Sherman remarked that the road to the heart is only two or three centimetres in a direct line, but it has taken surgery nearly 2,400 years to travel it. Overcoming centuries of cultural and medical prejudice required a degree of courage and vision still difficult to appreciate today. Even after that first step had been taken, another 50 years elapsed before surgeons began to make any real progress. Then, in a dizzying period of three decades, they learned how to open the heart, repair and even replace it. In most fields, an era of such fundamental discoveries happens only once if at all and it is unlikely that cardiac surgeons will ever again captivate the world as Christiaan Barnard and his colleagues did in 1967. But the history of heart surgery is littered with breakthroughs nobody saw coming, and as long as there are surgeons of talent and imagination, and a determination to do better for their patients, there is every chance that they will continue to surprise us.
Main photograph: Getty Images
This is an adapted extract from The Matter of the Heart by Thomas Morris, published by the Bodley Head
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Robot hearts: medicine's new frontier - The Guardian
Kidney research leads to heart discovery – Newsplex – The Charlottesville Newsplex
By JoanneRUSSELL25
CHARLOTTESVILLE, Va. (NEWSPLEX) -- Researchers at the University of Virginia School of Medicine were looking into kidneys and learned more about the formation of the heart.
They also identified a gene that is responsible for a deadly cardiac condition.
According to a release, scientists discovered the heart's inner lining forms from the same stem cells, known as precursor cells, that turn into blood.
That means a single type of stem cell created both the blood and part of the organ that pumps it.
A particular gene, called S1P1, is necessary for the proper formation of the heart, and without it, the tissue develops a sponginess that compromises the heart's ability to contract tightly and pump blood efficiently.
That condition is called ventricular non-compaction cardiomyopathy, which often leads to early death.
"Many patients who suffer from untreatable chronic disease, including heart and kidney disease, are in waiting lists for limited organ transplantation. Therefore, there is an urgent need to understand what happens to the cells during disease and how can they be repaired," said researchers Yan Hu, PhD. "Every organ is a complex machine built by many different cell types. Knowing the origin of each cell and which genes control their normal function are the foundations for scientists to decipher the disease process and eventually to find out how to guide the cells to self-repair or even to build up a brand new organ using amended cells from the patients."
The researchers were looking into how the kidneys form when they noted a deletion of the S1P1 gene in research mice led to deadly consequences elsewhere in the bodies of the mice.
"We were studying the role of these genes in the development of the vasculature of the kidney," said Maris Luisa S. Sequeira-Lopez, MD, of UVA's Child Health Research Center. "The heart is the first organ that develops, and so when we deleted this gene in these precursor cells, we found that it resulted in abnormalities of the heart, severe edema, hemorrhage and low heart rate."
In looking closer at the heart, the researchers discovered the gene deletion caused thin heart walls and other cardiac problems in developing mice embryos.
"For a long time, scientists believed that each organ developed independently of other organs, and the heart developed from certain stem cells and blood developed from blood stem cells," said researcher Brian C. Belyea, MD, of the UVA Children's Hospital. "A number of studies done in this lab and others, including this work, shows that there's much more plasticity in these precursor cells. What we found is that cardiac precursor cells that are present in the embryonic heart do indeed give rise to components of the heart in adults but also give rise to the blood cells."
He also said the discovery may one day lead to the development of better treatments for the cardiac condition.
The findings have been published in the journal Scientific Reports.
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Kidney research leads to heart discovery - Newsplex - The Charlottesville Newsplex
Opinion/Commentary: Seniors put at risk by outdated Medicare policies – The Daily Progress
By LizaAVILA
Almost 30 years ago, the federal government helped make it easier for patients with leukemia and lymphoma to receive lifesaving stem cell transplants. Now, we need the federal governments help again to ensure that Medicare patients with these cancers and other serious blood disorders can access the care they need.
In 1987, Congress approved funding for a national database of patients willing to donate bone marrow or peripheral blood stem cells. That database is now known as the Be The Match Registry, operated by the National Marrow Donor Program/Be The Match. According to the NMDP/Be The Match, patients searching the registry have access to 27 million potential volunteer bone marrow and peripheral blood stem cell donors worldwide, along with more than 680,000 units of cord blood donated by mothers after giving birth.
Having access to such a large registry has made it easier for patients to find a match if they dont have a fully matched sibling donor, which is the case for about 70 percent of patients who receive a stem cell transplant. The registry has helped 80,000 patients receive bone marrow transplants, peripheral blood stem cell transplants, or cord blood transplants from an unrelated donor.
While the federal governments foresight and financial support have helped make adult stem cell and cord blood transplants the only cure available for these diseases possible for thousands of patients, Medicare coverage policies have not kept pace with this breakthrough treatment.
Medicare is more restrictive than private insurance companies in deciding for what indications stem cell transplants and cord blood transplants will be covered. With private insurance companies, we have the opportunity to talk with a medical director about the indication and provide literature to support the decision for a transplant. This opportunity is not available for our Medicare patients.
In most cases, Medicare doesnt decide whether to cover a stem cell or cord blood transplant until after the procedure is completed. This leaves most Medicare patients an impossible choice: Turn down their only chance at a cure or potentially face paying the significant cost of a transplant themselves. Even when Medicare does decide to reimburse for these transplants, according to the NMDP/Be The Match, it covers less than half the cost of the transplant.
Addressing this issue is especially important because seniors make up a large portion of the patients with the cancers and blood diseases that can be cured by a stem cell or cord blood transplant. For example, 24 of the 65 patients who received stem cell or cord blood transplants at University of Virginia Health System in 2016 had Medicare coverage.
So I am asking the Centers for Medicare & Medicaid Services to expand Medicare coverage for stem cell and cord blood transplants, along with paying for the search and procurement costs as they already do for solid organ transplants.
The federal government has helped save the lives of tens of thousands of patients through better access to stem cell and cord blood transplants. I hope now they will act to make sure all Medicare patients who need one of these transplants can receive it.
Tamila L. Kindwall-Keller, DO, MS is the associate clinical director of the Stem Cell Transplant Program at the University of Virginia Health System.
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Opinion/Commentary: Seniors put at risk by outdated Medicare policies - The Daily Progress
The US is wrong to ban pay for bone-marrow donors – Standard-Examiner
By raymumme
(c) 2017, Bloomberg View.
Two years ago, Doreen Flynn of Lewiston, Maine, won her case against the U.S. government, successfully arguing that bone-marrow donors should be able to receive compensation.
Flynn, a mother of three girls who are afflicted with a rare, hereditary blood disease called Fanconis anemia, has a strong interest in bone-marrow transplantation. At the time of the court ruling, her oldest daughter, Jordan, 14, had already received a transplant, and one of the younger twins, Jorja, was expected to need one in a few years.
Locating a marrow donor is often a needle-in-a-haystack affair. The odds that two random individuals will have the same tissue type are less than 1 in 10,000, and the chances are much lower for blacks. Among the precious few potential donors who are matched, nearly half dont follow through with the actual donation. Too often, patients dont survive the time it takes to hunt for another donor.
Allowing compensation for donations could enlarge the pool of potential donors and increase the likelihood that compatible donors will follow through. So the ruling by a three-judge panel of the U.S. Court of Appeals for the Ninth Circuit was promising news for the 12,000 people with cancer and blood diseases currently looking for a marrow donor. (James F. Childress, an ethicist at the University of Virginia, and I submitted an amicus brief in the case.)
Soon after the verdict, Shaka Mitchell, a lawyer in Nashville, Tennessee, and co-founder of the nonprofit MoreMarrowDonors.org, began collecting funds to underwrite $3,000 donor benefits, which were to be given as scholarships, housing allowances or gifts to charity.
Mitchell also invited a team of economists to evaluate the effects of the ruling on peoples willingness to join a registry and to donate when they are found to be a match. The researchers were to specifically assess whether cash payments would be any more or less persuasive than noncash rewards or charitable donations.
Now comes the bad news. On Oct. 2, the U.S. Department of Health and Human Services proposed a new rule that would overturn the Ninth Circuits decision. The government proposes designating a specific form of bone marrow -- circulating bone-marrow stem cells derived from blood -- as a kind of donation that, under the 1984 National Organ Transplant Act, cannot be compensated. If this rule goes into effect (the public comment period ends today), anyone who pays another person for donating these cells would be subject to as much as five years in prison and a $50,000 fine.
The problem with this rule is that donating bone marrow is not like donating an essential organ. Indeed, the Ninth Circuit based its decision on the fact that modern bone-marrow procurement, a process known as apheresis, is more akin to drawing blood. In the early 1980s, when the transplant act was written, the process was more demanding, involving anesthesia and the use of large, hollow needles to extract marrow from a donors hip. But today, more than two-thirds of marrow donations are done via apheresis. Blood is taken from a donors arm, the bone-marrow stem cells are filtered out, and the blood is then returned to the donor through a needle in the other arm.
The Ninth Circuit panel held that these filtered stem cells are merely components of blood -- no different from blood-derived plasma, platelets and clotting factors, for which donor compensation is allowed.
The strongest opposition to compensation comes from the National Marrow Donor Program, the Minneapolis-based nonprofit that maintains the nations largest donor registry. Michael Boo, the programs chief strategy officer, says of reimbursement, Is that what we want people to be motivated by?
The problem with this logic is that altruism has proven insufficient to motivate enough people to give marrow and, as a result, people die.
HHS is presumably under pressure from the National Marrow Donor Program. The department does not otherwise explain its proposed rule except to claim that compensation runs afoul of the transplant acts intent to ban commodification of human stem cells and to curb opportunities for coercion and exploitation, encourage altruistic donation and decrease the likelihood of disease transmission.
But how could such concerns plausibly apply to marrow stem cells and not to blood plasma? The process of collecting plasma is safe: No serious infection has been transmitted in plasma-derived products in nearly two decades, according to the Plasma Protein Therapeutics Association. Strenuous screening and testing in a robust regulatory environment, coupled with voluntary industry standards and sophisticated manufacturing processes, have created what has been called the safest blood product available today.
Outlawing compensation for stem blood cells but not mature blood cells might even violate the constitutional guarantee of equal protection of the law, according to Jeff Rowes, a lawyer at the Institute for Justice, which represented Flynn.
HHS should withdraw its proposal. Ideally, Congress should thwart future regulatory mischief by amending the National Organ Transplant Act to stipulate that marrow stem cells are not organs.
Each year, 2,000 to 3,000 Americans in need of marrow transplants die waiting for a match. Altruism is a virtue, but clearly it is not a dependable motive for marrow donation.
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Satel, a psychiatrist and a resident scholar at the American Enterprise Institute, is a co-author of Brainwashed: The Seductive Appeal of Mindless Neuroscience. To contact the editor responsible for this story: Mary Duenwald at mduenwald@bloomberg.net.
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Keywords: SATEL-OP-ED-MARROWDONORS
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The US is wrong to ban pay for bone-marrow donors - Standard-Examiner
Stem Cells Symptoms, Causes, Treatment – Why are stem …
By daniellenierenberg
Why are stem cells important?
Stem cells represent an exciting area in medicine because of their potential to regenerate and repair damaged tissue. Some current therapies, such as bone marrow transplantation, already make use of stem cells and their potential for regeneration of damaged tissues. Other therapies are under investigation that involves transplanting stem cells into a damaged body part and directing them to grow and differentiate into healthy tissue.
During the early stages of embryonic development the cells remain relatively undifferentiated (immature) and appear to possess the ability to become, or differentiate, into almost any tissue within the body. For example, cells taken from one section of an embryo that might have become part of the eye can be transferred into another section of the embryo and could develop into blood, muscle, nerve, or liver cells.
Cells in the early embryonic stage are totipotent (see above) and can differentiate to become any type of body cell. After about seven days, the zygote forms a structure known as a blastocyst, which contains a mass of cells that eventually become the fetus, as well as trophoblastic tissue that eventually becomes the placenta. If cells are taken from the blastocyst at this stage, they are known as pluripotent, meaning that they have the capacity to become many different types of human cell. Cells at this stage are often referred to as blastocyst embryonic stem cells. When any type of embryonic stem cells is grown in culture in the laboratory, they can divide and grow indefinitely. These cells are then known as embryonic stem cell lines.
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Melissa Conrad Stppler, MD, is a U.S. board-certified Anatomic Pathologist with subspecialty training in the fields of Experimental and Molecular Pathology. Dr. Stppler's educational background includes a BA with Highest Distinction from the University of Virginia and an MD from the University of North Carolina. She completed residency training in Anatomic Pathology at Georgetown University followed by subspecialty fellowship training in molecular diagnostics and experimental pathology.
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Stem Cells Symptoms, Causes, Treatment - Why are stem ...
Predicting the storm: Can computer models improve stem cell transplantation?
By LizaAVILA
PUBLIC RELEASE DATE:
4-Dec-2014
Contact: John Wallace wallacej@vcu.edu 804-628-1550 Virginia Commonwealth University @vcunews
Is the human immune system similar to the weather, a seemingly random yet dynamical system that can be modeled based on past conditions to predict future states? Scientists at VCU Massey Cancer Center's award-winning Bone Marrow Transplant (BMT) Program believe it is, and they recently published several studies that support the possibility of using next-generation DNA sequencing and mathematical modeling to not only understand the variability observed in clinical outcomes of stem cell transplantation, but also to provide a theoretical framework to make transplantation a possibility for more patients who do not have a related donor.
Despite efforts to match patients with genetically similar donors, it is still nearly impossible to predict whether a stem cell transplant recipient will develop potentially fatal graft-versus-host disease (GVHD), a condition where the donor's immune system attacks the recipient's body. Two studies recently published by the online journal Frontiers in Immunology explored data obtained from the whole exome sequencing of nine donor-recipient pairs (DRPs) and found that it could be possible to predict which patients are at greatest risk for developing GVHD and, therefore, in the future tailor immune suppression therapies to possibly improve clinical outcomes. The data provides evidence that the way a patient's immune system rebuilds itself following stem cell transplantation is representative of a dynamical system, a system in which the current state determines what future state will follow.
"The immune system seems chaotic, but that is because there are so many variables involved," says Amir Toor, M.D., member of the Developmental Therapeutics research program at Massey and associate professor in the Division of Hematology, Oncology and Palliative Care at the VCU School of Medicine. "We have found evidence of an underlying order. Using next-generation DNA sequencing technology, it may be possible to account for many of the molecular variables that eventually determine how well a donor's immune system will graft to a patient."
Toor's first study revealed a large and previously unmeasured potential for developing GVHD for which the conventional approach used for matching DRPs does not account. The conventional approach for donor-recipient compatibility determination uses human leucocyte antigen (HLA) testing. HLA refers to the genes that encode for proteins on the surface of cells that are responsible for regulating the immune system. HLA testing seeks to match DRPs who have similar HLA makeup.
Specifically, Toor and his colleagues used whole exome sequencing to examine variation in minor histocompatibility antigens (mHA) of transplant DRPs. These mHA are protein fragments presented on the HLA molecules, which are the receptors on cells' surface to which these fragments of degraded proteins from within a cell bind in order to promote an immune response. Using advanced computer-based analysis, the researchers examined potential interactions between the mHA and HLA and discovered a high level of mHA variation in HLA-matched DRPs that could potentially contribute to GVHD. These findings may help explain why many HLA-matched recipients experience GVHD, but why some HLA-mismatched recipients experience none remains a mystery. This seeming paradox is explained in a companion paper, also published in the journal Frontiers in Immunology. In this manuscript, the team suggests that by inhibiting peptide generation through immunosuppressive therapies in the earliest weeks following stem cell transplantation, antigen presentation to donor T cells could be diminished, which reduces the risk of GVHD as the recipients reconstitute their T-cell repertoire.
Following stem cell transplantation, a patient begins the process of rebuilding their T-cell repertoire. T cells are a family of immune system cells that keep the body healthy by identifying and launching attacks against pathogens such as bacteria, viruses or cancer. T cells have small receptors that recognize antigens. As they encounter foreign antigens, they create thousands of clones that can later be called upon to guard against the specific pathogen that presented the antigen. Over the course of a person's life, they will develop millions of these clonal families, which make up their T-cell repertoire and protect them against the many threats that exist in the environment.
This critical period where the patient rebuilds their immune system was the focus of the researchers' efforts. In previous research, Toor and his colleagues discovered a fractal pattern in the DNA of recipients' T-cell repertoires. Fractals are self-similar patterns that repeat themselves at every scale. Based on their data, the researchers believe that the presentation of minor histocompatability antigens following transplantation helps shape the development of T-cell clonal families. Thus, inhibiting this antigen presentation through immunosuppressive therapies in patients who have high mHA variation can potentially reduce the risk of GVHD by influencing the development of their T-cell repertoire. This is backed by data from clinical studies that show immune suppression soon after transplantation improves outcomes in unrelated DRPs.
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Predicting the storm: Can computer models improve stem cell transplantation?
Pluripotent cells created by nuclear transfer can prompt immune reaction, researchers find
By daniellenierenberg
PUBLIC RELEASE DATE:
20-Nov-2014
Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center @sumedicine
Mouse cells and tissues created through nuclear transfer can be rejected by the body because of a previously unknown immune response to the cell's mitochondria, according to a study in mice by researchers at the Stanford University School of Medicine and colleagues in Germany, England and at MIT.
The findings reveal a likely, but surmountable, hurdle if such therapies are ever used in humans, the researchers said.
Stem cell therapies hold vast potential for repairing organs and treating disease. The greatest hope rests on the potential of pluripotent stem cells, which can become nearly any kind of cell in the body. One method of creating pluripotent stem cells is called somatic cell nuclear transfer, and involves taking the nucleus of an adult cell and injecting it into an egg cell from which the nucleus has been removed.
The promise of the SCNT method is that the nucleus of a patient's skin cell, for example, could be used to create pluripotent cells that might be able to repair a part of that patient's body. "One attraction of SCNT has always been that the genetic identity of the new pluripotent cell would be the same as the patient's, since the transplanted nucleus carries the patient's DNA," said cardiothoracic surgeon Sonja Schrepfer, MD, PhD, a co-senior author of the study, which will be published online Nov. 20 in Cell Stem Cell.
"The hope has been that this would eliminate the problem of the patient's immune system attacking the pluripotent cells as foreign tissue, which is a problem with most organs and tissues when they are transplanted from one patient to another," added Schrepfer, who is a visiting scholar at Stanford's Cardiovascular Institute. She is also a Heisenberg Professor of the German Research Foundation at the University Heart Center in Hamburg, and at the German Center for Cardiovascular Research.
Possibility of rejection
A dozen years ago, when Irving Weissman, MD, professor of pathology and of developmental biology at Stanford, headed a National Academy of Sciences panel on stem cells, he raised the possibility that the immune system of a patient who received SCNT-derived cells might still react against the cells' mitochondria, which act as the energy factories for the cell and have their own DNA. This reaction could occur because cells created through SCNT contain mitochondria from the egg donor and not from the patient, and therefore could still look like foreign tissue to the recipient's immune system, said Weissman, the other co-senior author of the paper. Weissman is the Virginia and D.K. Ludwig Professor for Clinical Investigation in Cancer Research and the director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine.
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Pluripotent cells created by nuclear transfer can prompt immune reaction, researchers find
Tse Named Director of Bone Marrow Transplantation Division at University of Louisville
By NEVAGiles23
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Newswise LOUISVILLE, Ky. William Tse, M.D., associate professor of medicine and eminent scholar in hematologic malignancies research at the Mary Babb Randolph Cancer Center at West Virginia University, has been named the new director of Bone Marrow Transplantation at the University of Louisville James Graham Brown Cancer Center, a part of KentuckyOne Health. Tse will join UofL Nov. 1.
Tse will hold the Marion F. Beard Endowed Chair in Hematology Research at UofL and become a member of the cancer centers Developmental Biology Program.
Dr. Tse is emerging as one of the thought leaders in bone marrow transplantation, said Donald Miller, M.D., Ph.D., director of the JGBCC. He has trained and worked at several of the leading blood cancer programs in the nation. We look forward to his leading our program at UofL.
Tse has been at West Virginia since 2009, where he also is the co-leader the Osborn Hematologic Malignancies Program. Prior to joining West Virginia, Tse was on the faculty at the University of Colorado Denver, where he was the director of translational research program for bone marrow transplantation and hematologic malignancies. He also previously was with Case Western Reserve University and the Fred Hutchinson Cancer Research Center/University of Washington Medical Center.
Tse is active in national organizations, serving in several capacities with the American Society of Hematology, including section chair for the annual meetings Oncogene Section and bone marrow transplantation outcome section, as well as the American Society of Clinical Oncology as an annual meeting abstract reviewer and the section chair on geriatric oncology. Tse also serves leadership roles on several editorial boards including as the senior editor of the American Journal of Blood Research, stem cell biomarkers section editor for Biomarker Research, senior editor of the American Journal of Stem Cells and the academic editor of PLoS One.
A graduate of the Sun Yat-Sen University School of Medicine in Guangzhou, Guangdong, in China, he did a thoracic surgical oncology residency at Sun Yat-Sen University Cancer Center in Guangzhou before completing postdoctoral research fellowships in medical biophysics, immunology and cancer at the Princess Margaret Hospital/Ontario Cancer Institute and the Hospital for Sick Children in Ontario, Canada. He completed clinical pathology and internal medicine residencies at North Shore-Long Island Jewish Hospital before undertaking a senior medical fellowship in clinical research and medical oncology divisions at the Fred Hutchinson Cancer Research Center at the University of Washington Medical Center.
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Tse Named Director of Bone Marrow Transplantation Division at University of Louisville
UVA Expands Cancer Treatment
By daniellenierenberg
UVA joins National Marrow Donor Program giving greater access to cancer treatments by Ishaan Sachdeva | Jun 25 2014 | 06/25/14 10:11pm | Updated 14 hours ago
The Emily Couric Cancer Center of the University of Virginia Health System has expanded its access to bone marrow and hematopoietic stem cell transplant donors. Now designated as a National Marrow Donor Program (NMDP), the Health System will have access to the Be The Match Registry, the worlds largest and most diverse bone marrow registry. Implications of this change are significant for patients afflicted with blood cancers like leukemia who obtain treatment through the Health System.
Bone marrow, the soft, spongy tissue within bones like the sternum or the ilium of the pelvis, forms hematopoietic or blood-forming stem cells. These cells, unlike embryonic stem cells, differentiate only into types of blood cells- red blood cells, white blood cells or clotting platelets. Leukemia causes bone marrow to produce abnormal, leukemic white blood cells that divide uncontrollably, forming tumors that deprive cells of oxygen and reduce infection defense. One treatment method is autologous bone marrow transplant, in which patients receive stem cells from their healthy, non cancerous bone marrow.
The idea [of autologous transplants] is that you extract healthier bone marrow from the patient to have a source of stored, non-cancerous bone marrow. You can then treat the patient with higher doses of treatment than you can normally give because the most common limitation to treatment is that treatment will kill off healthy bone marrow you might have, said Thomas P. Loughran Jr., MD, the Universitys Cancer Center director.
Essentially, a patients healthy bone marrow is safeguarded outside their body while aggressive treatment is administered to kill cancerous marrow. Another form of treatment is allogeneic treatment, in which bone marrow is transplanted from a sibling or an unrelated donor.
In an allogeneic transplant, you are also transplanting in a new immune system. The new immune system comes in and recognizes the body as a foreign tissue and starts attacking that tissue. This causes a beneficial graft vs. leukemia effect where this new immune system attacks any residual leukemia, but may also cause a harmful graft versus host disease where normal tissue is also attacked, Loughran said.
The donor and recipient tissue interaction underscores the genetic component of bone marrow transplants from external donors. Despite the curative potential of a bone marrow transplant, a strong genetic match between donor and recipient is crucial to the utility of a transplant.
The ability of any donor to be successful is based on genetics. Its called HLA [human leukocyte antigen] typing. The HLA system has four genes called A, B, C and D, and it turns out that A, B and D are influential. We have half of our genes each from both parents, so we have six of these: 2 A, 2 B and 2 D. The best case is a six out of six match from a brother or sister, but the chances are only 1 in 4, said Loughran. The consequence of low genetic probabilities is a large pool of unrelated donors, like the Be The Match Registry. Through such services, patients have a greater chance of finding an unrelated donor who may provide a successful genetic match.
The coordinating center would identify the place where the donor is living and tell them they are potentially able to donate. In the past, the donor would have bone marrow directly extracted. Now it is almost always from the PBSCT [peripheral blood stem cell transplantation] procedure. The donor takes a growth factor that stimulates growth of the needed hematopoietic stem cells within their peripheral blood circulation. A catheter collects this blood and the stem cells are separated from the blood by a machine, and the blood is returned back to the donor. The collected stem cells are sent to the lab where they are purified and frozen, Loughran said.
Meanwhile, the patient in preparation for the transplant is given the highest dose of chemotherapy that can be tolerated. The donated stem cells are administered to the patient in a way similar to IV fluid.
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UVA Expands Cancer Treatment
Immune system harnessed to improve stem cell transplant outcomes
By daniellenierenberg
ScienceDaily (Oct. 1, 2012) A novel therapy in the early stages of development at Virginia Commonwealth University Massey Cancer Center shows promise in providing lasting protection against the progression of multiple myeloma following a stem cell transplant by making the cancer cells easier targets for the immune system.
Outlined in the British Journal of Hematology, the Phase II clinical trial was led by Amir Toor, M.D., hematologist-oncologist in the Bone Marrow Transplant Program and research member of the Developmental Therapeutics program at VCU Massey Cancer Center. The multi-phased therapy first treats patients with a combination of the drugs azacitidine and lenalidomide. Azacitidine forces the cancer cells to express proteins called cancer testis antigens (CTA) that immune system cells called T-cell lymphocytes recognize as foreign. The lenalidomide then boosts the production of T-cell lymphocytes. Using a process called autologous lymphocyte infusion (ALI), the T-cell lymphocytes are then extracted from the patient and given back to them after they undergo a stem cell transplant to restore the stem cells' normal function. Now able to recognize the cancer cells as foreign, the T-cell lymphocytes can potentially protect against a recurrence of multiple myeloma following the stem cell transplant.
"Every cell in the body expresses proteins on their surface that immune system cells scan like a barcode in order to determine whether the cells are normal or if they are foreign. Because multiple myeloma cells are spawned from bone marrow, immune system cells cannot distinguish them from normal healthy cells," says Toor. "Azacitidine essentially changes the barcode on the multiple myeloma cells, causing the immune system cells to attack them," says Toor.
The goal of the trial was to determine whether it was safe, and even possible, to administer the two drugs in combination with an ALI. In total, 14 patients successfully completed the investigational drug therapy. Thirteen of the participants successfully completed the investigational therapy and underwent a stem cell transplant. Four patients had a complete response, meaning no trace of multiple myeloma was detected, and five patients had a very good partial response in which the level of abnormal proteins in their blood decreased by 90 percent.
In order to determine whether the azacitidine caused an increased expression of CTA in the multiple myeloma cells, Toor collaborated with Masoud Manjili, D.V.M., Ph.D., assistant professor of microbiology and immunology at VCU Massey, to conduct laboratory analyses on bone marrow biopsies taken from trial participants before and after treatments. Each patient tested showed an over-expression of multiple CTA, indicating the treatment was successful at forcing the cancer cells to produce these "targets" for the immune system.
"We designed this therapy in a way that could be replicated, fairly inexpensively, at any facility equipped to perform a stem cell transplant," says Toor. "We plan to continue to explore the possibilities of immunotherapies in multiple myeloma patients in search for more effective therapies for this very hard-to-treat disease."
In addition to Manjili, Toor collaborated with John McCarty, M.D., director of the Bone Marrow Transplant Program at VCU Massey, and Harold Chung, M.D., William Clark, M.D., Catherine Roberts, Ph.D., and Allison Hazlett, also all from Massey's Bone Marrow Transplant Program; Kyle Payne, Maciej Kmieciak, Ph.D., from Massey and the Department of Microbiology and Immunology at VCU School of Medicine; Roy Sabo, Ph.D., from VCU Department of Biostatistics and the Developmental Therapeutics program at Massey; and David Williams, M.D., Ph.D., from the Department of Pathology at VCU School of Medicine, co-director of the Tissue and Data Acquisition and Analysis Core and research member of the Developmental Therapeutics program at Massey.
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Immune system harnessed to improve stem cell transplant outcomes
Biostem Appoints Philip A. Lowry, MD as Chairman of Its Scientific and Medical Board of Advisors
By NEVAGiles23
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Posted May 29, 2012
Philip A. Lowry
Highly Recognized Bone Marrow Stem Cell Transplant Specialist Added to Existing Member Expertise in Maternal Fetal Medicine, Cardiology, and Pathology
CLEARWATER, FL -- Biostem U.S., Corporation, (OTCQB: HAIR) (PINKSHEETS: HAIR) a stem cell regenerative medicine sciences company, announced that Philip A. Lowry, MD, has been appointed as the Chairman of its Scientific and Medical Board of Advisors (SAMBA).
According to Biostem CEO, Dwight Brunoehler, "As Chairman, Dr. Lowry will work with a team drawn from a cross-section of medical specialties. His combination of research, academic and community practice experience make him the perfect individual to coordinate and lead the outstanding group of physicians that makes up our SAMBA. As a group, The SAMBA will guide the company to maintain the highest ethical standards in every effort, while seeking and developing new cutting edge technology based on stem cell use. I am privileged to work with Dr. Lowry, once again."
Dr. Lowry stated, "Dwight is an innovative businessman with an eye on cutting-edge stem cell technology. His history in the industry speaks for itself. I like the plan at Biostem and look forward to working with everyone involved."
Dr. Philip A. Lowry received his undergraduate degree from Harvard College before going on to the Yale University School of Medicine. His completed his internal medicine residency at the University of Virginia then pursued fellowship training in hematology and oncology there as well. During fellowship training and subsequently at the University of Massachusetts, he worked in the laboratory of Dr. Peter Quesenberry working on in vitro and in vivo studies of mouse and human stem cell biology.
Dr. Lowry twice served on the faculty at the University of Massachusetts Medical Center from 1992-1996 and from 2004-2009 as an assistant and then associate clinical professor of medicine establishing the bone marrow/stem cell transplantation program there, serving as medical director of the Cryopreservation Lab supporting the transplant program, helping to develop a cord blood banking program, and teaching and coordinating the second year medical school course in hematology and oncology. Dr. Lowry additionally has ten years experience in the community practice of hematology and oncology. In 2010, Dr. Lowry became chief of hematology/oncology for the Guthrie Health System, a three-hospital tertiary care system serving northern Pennsylvania and southern New York State. He is charged with developing a cutting-edge cancer program that can project into a traditionally rural health care delivery system.
Dr. Lowry has also maintained a career-long interest in regenerative medicine springing from his research and practice experience in stem cell biology. His new role positions him to foster further development of that field. As part of a horizontally and vertically integrated multi-specialty team, he is closely allied with colleagues in cardiology, neurology/neurosurgery, and orthopedics among others with whom he hopes to stimulate the expansion of regenerative techniques.
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Biostem Appoints Philip A. Lowry, MD as Chairman of Its Scientific and Medical Board of Advisors
Biostem U.S., Corporation Appoints Philip A. Lowry, MD as Chairman of Its Scientific and Medical Board of Advisors
By raymumme
CLEARWATER, FL--(Marketwire -05/29/12)- Biostem U.S., Corporation, (HAIR.PK) (HAIR.PK) (Biostem, the Company), a fully reporting public company in the stem cell regenerative medicine sciences sector, today announced that Philip A. Lowry, MD, has been appointed as the Chairman of its Scientific and Medical Board of Advisors (SAMBA).
According to Biostem CEO, Dwight Brunoehler, "As Chairman, Dr. Lowry will work with a team drawn from a cross-section of medical specialties. His combination of research, academic and community practice experience make him the perfect individual to coordinate and lead the outstanding group of physicians that makes up our SAMBA. As a group, The SAMBA will guide the company to maintain the highest ethical standards in every effort, while seeking and developing new cutting edge technology based on stem cell use. I am privileged to work with Dr. Lowry, once again."
Dr. Lowry stated, "Dwight is an innovative businessman with an eye on cutting-edge stem cell technology. His history in the industry speaks for itself. I like the plan at Biostem and look forward to working with everyone involved."
Dr. Philip A. Lowry received his undergraduate degree from Harvard College before going on to the Yale University School of Medicine. His completed his internal medicine residency at the University of Virginia then pursued fellowship training in hematology and oncology there as well. During fellowship training and subsequently at the University of Massachusetts, he worked in the laboratory of Dr. Peter Quesenberry working on in vitro and in vivo studies of mouse and human stem cell biology.
Dr. Lowry twice served on the faculty at the University of Massachusetts Medical Center from 1992-1996 and from 2004-2009 as an assistant and then associate clinical professor of medicine establishing the bone marrow/stem cell transplantation program there, serving as medical director of the Cryopreservation Lab supporting the transplant program, helping to develop a cord blood banking program, and teaching and coordinating the second year medical school course in hematology and oncology. Dr. Lowry additionally has ten years experience in the community practice of hematology and oncology. In 2010, Dr. Lowry became chief of hematology/oncology for the Guthrie Health System, a three-hospital tertiary care system serving northern Pennsylvania and southern New York State. He is charged with developing a cutting-edge cancer program that can project into a traditionally rural health care delivery system.
Dr. Lowry has also maintained a career-long interest in regenerative medicine springing from his research and practice experience in stem cell biology. His new role positions him to foster further development of that field. As part of a horizontally and vertically integrated multi-specialty team, he is closely allied with colleagues in cardiology, neurology/neurosurgery, and orthopedics among others with whom he hopes to stimulate the expansion of regenerative techniques.
About Biostem U.S., Corporation
Biostem U.S., Corporation is a fully reporting Nevada corporation with offices in Clearwater, Florida. Biostem is a technology licensing company with proprietary technology centered on providing hair re-growth using human stem cells. The company also intends to train and license selected physicians to provide Regenerative Cellular Therapy treatments to assist the body's natural approach to healing tendons, ligaments, joints and muscle injuries by using the patient's own stem cells. Biostem U.S. is seeking to expand its operations worldwide through licensing of its proprietary technology and acquisition of existing stem cell-related facilities. The company's goal is to operate in the international biotech market, focusing on the rapidly growing regenerative medicine field, using ethically sourced adult stem cells to improve the quality and longevity of life for all mankind.
More information on Biostem U.S., Corporation can be obtained through http://www.biostemus.com, or by calling Fox Communications Group 310-974-6821.
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Biostem U.S., Corporation Appoints Philip A. Lowry, MD as Chairman of Its Scientific and Medical Board of Advisors