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Are Stem Cell Companies Abusing ClinicalTrials.gov? – PLoS Blogs (blog)

By adminAugust 10, 2017Stem Cell Medicine

Im often asked about the safety of treatments that purport to inject stem cells into painful body parts. The reputation of stem cells seems to exceed the reach, with companies touting treatments that arent FDA approved or even being tested.

Back in March, an alarming article in the New England Journal of Medicinedescribed three women blinded by stem cell treatments two of the patients reported seeing a reference on the company website to registration at the National Institutes of Healths well-respected ClinicalTrials.gov, and assuming it applied to their treatment. It didnt.

In what is perhaps a modern version of hawking snake oil, companies can indeed register certain clinical trials without breaking any rules but desperate patients might not know that.

There is no doubt that some patients have misinterpreted a studys listing on ClinicalTrials.gov as a stamp of legitimacy, federal review, and compliance. In this way, treatments with no safety or efficacy data, no prior clinical study, and no ongoing clinical trials under FDA review, appear to have federal approval. Such a misunderstanding can lead to disastrous outcomes for patients, said Thomas Albini, MD, of the Bascom Palmer Eye Institute of the University of Miami, who treated the blinded women.

When I wrote about the disaster here at DNA Scienceand atMedscape Medical News, my Medscape editor asked me to take a closer look at criteria for listing investigations at ClinicalTrials.gov. It proved an interesting exercise, but I declined to write an article, fearing lawsuits if I named companies.

ClinicalTrials.gov is where research groups, in academia and pharma/biotech, describe protocols to evaluate the safety and efficacy of new drugs, biologics, and devices, which FDA regulates, typically in randomized, controlled trials. But for an observational study that just follows what happens after a treatment, no such thumbs-up is required; no investigational new drug (IND) designation or investigational device exemption (IDE) need be filed. And that creates a loophole that companies are happily jumping through and luring patients in pain, who may know little about clinical trial design, and perhaps trust too much the companies and the doctors offering these services.

Its easy to see how people are fooled. One company claims that By providing access to registered clinical studies through the NIH, we are providing patients with the ability to choose a stem cell treatment center with the highest standard of care. If the treatment is experimental, how can there even be a standard of care?

MOST STUDIES LEGIT

I love ClinicalTrials.gov its packed with information about all manner of conditions, with contacts and references. I started my investigation by searching for studies that sounded bogus.

I began with a treatment that epitomizes pseudoscience: magnets. But I was fooled. Other than legit uses in medical devices, my magnet search called up as an acronym of sorts for theMothers and Girls Dancing Together Trial, a well-designed study on preventing childhood obesity, with a decent sample size and controls.

I also thought the randomised crossover trial of the acute effects of a deep-fried Mars bar or porridge on the cerebral vasculature was fake, but it turned out to be a medical students project, well done, and published in the Scottish Medical Journal.

But trial NCT02833532, sponsored by a Korean pharmaceutical company, was likely a joke, with the stated purpose of temporary penile enhancement and one of the investigators first name being Dong. Participants must answer the question How do you rate your penile size? Very small/small/normal/big/very big to enroll. Those accepted get to try something made of hyaluronic acid, which is found, coincidentally, in cocks combs.

Searching ClinicalTrials.gov for stem cells returns more than 4,000 entries, so I gave up. Fortunately, Leigh Turner, PhD, associate professor at the Center for Bioethics at the University of Minnesota, wasnt afraid of lawyers and took a more measured, scholarly approach. He recently published the intriguing findings in Regenerative Medicine, where you can find nice tables naming the stem cell companies that use and possibly abuse ClinicalTrials.gov.

AN ACADEMIC INVESTIGATION

Dr. Turner searched ClinicalTrials.gov for stem cells along with patient-sponsored, patient-funded, and self-funded because expecting patients to pay is a red flag. Only a very few real clinical trials charge patients, and those that do must have FDA approval to do so.

He found 7 such pay-as-you-go clinical trials, each enrolling more than 100 people, at the government website, and another 11 in a database of companies that provide direct-to-consumer stem-cell-based treatments. The DTC label indicates that the treatments arent part of a real experimental protocol. One of themhad signed up more than 3,000 gullible people.

The companies that charge patients yet proclaim a ClinicalTrials.gov listing are having their proverbial cake and eating it too borrowing the governmental veneer of a sanctioned clinical trial, while collecting fees. And many health care consumers arent even aware theyre being bamboozled.

Another red flag in a stem cell pitch is an everything-but-the-kitchen-sink list of targets. Stem Cell Network, for example, claims to be able to treat, using stem cells grown from a patients fat, some 28 conditions, including the vague knee problems, and also muscular dystrophy, ankle problems, neuropathy, asthma, and alopecia areata. Also be wary of stem cells derived from one body part like butt fat being injected into another body part such as eyeballs.

Wed like people to protect themselves by going to a reliable website, like ClinicalTrials.gov, to distinguish legitimate from bogus claims of stem cell clinics. But the findings of this paper challenge that advice because this valuable resource, which is designed to promote transparency and to help people find clinical trials, lists unlicensed and unproven stem cell interventions that companies turn into personal marketing platforms. So if you have ALS, MS, Parkinsons disease, a ClinicalTrials.gov listing looks like any other study on the NIH website. Many people think a listing is credible, Dr. Turner told me.

There is an urgent need for careful screening of clinical studies before they are registered with ClinicalTrials.gov, Dr. Turners paper concludes. But in the current climate of a nuclear threat, a health care system in disarray, and possible cuts to the CDC, FDA, and NIH, ramping up scrutiny at ClinicalTrials.gov is unlikely to have priority, if the President even has a clue what it is.

Its not possible to slash, burn, defund, and deregulate at every turn and think that federal agencies are going to improve how they function. But no administration is forever, no budget is forever, deregulatory moments dont last forever, and perhaps problems that are ignored or neglected now will be addressed in the future, with collateral damage along the way while nothing is done, warns Dr. Turner, who lives in Canada. I wonder if he has a spare room.

Those seeking stem cell treatments should check out the International Society for Stem Cell Research (ISSCR) Patient Handbook on Stem Cell Therapiesand stemcells.nih.gov. Alas, much of the media is still somewhat unfamiliar with the biology of stem cells, that they are not cells that can turn into any cell typebut that they self-renew and jettison a new stem cell at every division. Thats what makes them stem cells, not the ability to spawn specialized cells.

So I tell people who ask me if they should have stem cells shot into their aching knees or backs to do so only if they wouldnt object to an abnormal growth cancer forming there.

When it comes to stem cell therapies, its caveat emptor buyer beware!

New Mayo Clinic facility aims to increase lungs available for transplant – First Coast News

By adminAugust 10, 2017Stem Cell Clinic

In the coming months, construction will begin on a new facility at Mayo Clinic's Jacksonville campus.

Juliette Dryer, WTLV 7:05 PM. EDT August 09, 2017

Larry Rawdon received two lung transplants from Mayo Clinic in Jacksonville (PHOTO: First Coast News)

JACKSONVILLE, Fla. - In the coming months, construction will begin on a new facility at Mayo Clinics Jacksonville campus.

Mayo Clinic announced a partnership with Maryland-based United Therapeutics Corporation to build and operate a lung restoration center in 2015.

Theres only one other center right now in the country thats actually doing what we do, Windell Smith, Mayo Clinic operations administrator, said. And thats in Silver Springs, Maryland.

The lung restoration center will house a process called ex vivo lung perfusion, or EVLP. Dr. David Erasmus, medical director of the lung transplant program at Mayo Clinic, said the lungs are taken from a donor to a restoration center and hooked up to an artificial circulatory system.

And then they can be evaluated to see if they may be viable or made viable for transplant, Dr. Erasmus said.

Erasmus said the benefits of EVLP are two-fold. Right now, borderline lungs are usually discarded because doctors dont want to take the risk on a lung that may not be viable. While lungs are undergoing EVLP, doctors can monitor how they would respond inside the body and make a more informed decision whether to proceed with transplant or discard the lungs.

We can measure the pressure in the lungs, we can measure the pressure in the circulation of the lungs, we can measure blood gas tests on those lungs and we can take X-rays, Erasmus said. We can also do some of the procedures that we would do in a living person. We can do bronchoscopies where we look down into the airways of those lungs.

The lungs can also be treated to improve their condition for transplant.

One can introduce antibiotics or steroids or other types of therapies such as potentially stem cell therapy, Erasmus said. All sorts of interventions might be tried on these lungs.

Since Mayo Clinics program has not yet started, Erasmus said its difficult to predict the impact it will have on the number of lung transplants. However, he said a similar program yielded a 25 percent increase.

We would get more patients transplanted, we may be able to get them transplanted quicker, Erasmus said. The more people we transplant, the more likely we are to transplant patients that are a little lower down on the list.

Jacksonville resident Larry Rawdon received two lung transplants at Mayo. The first, a single lung transplant in 2005; the second, a double lung transplant in 2008 when his body rejected the first lung.

I still remember being able to take the first full deep breath Id been able to take in the previous three years, Rawdon said of his first transplant.

Rawdon, who started showing symptoms of idiopathic pulmonary fibrosis in 2002, was on the verge of death when he received his second transplant.

You know unless somethings done, youre not going to survive whats happening to you, he said.

Rawdon, a former Broadway cellist, took up the harmonica to strengthen his lungs and diaphragm post-surgery. He now teaches classes to other people in Mayo Clinics lung transplant program.

I used to enjoy more things, but now I enjoy things more, he said.

Rawdon is planning a Christmas trip to New York City to see his two daughters and recently celebrated his 48th wedding anniversary with his wife, Katie.

Mayo Clinic staff tell First Coast News a date has not yet been set for construction to begin, but its expected to start in the fall. Construction is expected to take two years.

2017 WTLV-TV

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Boldly Go! My Journey in Stem Cell Therapy – Health Link …

By adminAugust 10, 2017Stem Cell Medical Center

Carolyn Hastings

Patient Update

Carolyn Hastings sent us this fascinating followup piece written 9 months after the stem cell procedure she had performed on her knee by Dr. Paul Handleman here at Health Link Medical Center.

By Carolyn Hastings

I am living proof! I admit to being a little bit of a risk-taker, but it has always served me well. I had Radial Keratotomy performed when I knew no one who had. It has been so long ago that unless youre my age you probably havent even heard of it. RK was the precursor to Laser Eye Surgery. I talked a friend into having it done the same day, so I wouldnt be alone. What can I say, a few minutes in surgery and I was 20/20! So, here I am again, living proof!

Its funny how fearful we can be of the unknown, yet all around us limits are being stretched. I live with my phone in one hand and my iPad in the other. I read on Facebook about drones and rifles that shoot around corners. My real camera sits on the shelf gathering dust. Dont get me wrong, I dont jump out of perfectly good airplanes or ski off mountains that I cant get to without a helicopter but that may be because Im a little old for that kind of stuff!

My age brought me to why Im writing this story. A few months ago I had Stem Cell Therapy to rebuild the cartilage in my knee, but let me back up a little. Five years ago I had a Total Knee Replacement done on my right knee. I was scheduled to have my left knee replaced within a few months as well. I know that everyone has a little different outcome to Total Knee Replacements but mine was tough, tough enough that I immediately cancelled my second surgery and started looking for an alternative. Thats probably where this story should have begun. I looked and I looked, but to no avail. The Orthopedic world was working on it but to me it felt like more of the same. I talked to everyone that would talk to me. Almost everyone I spoke with was desperately trying to affirm their decision for having the surgery, but if you caught them in an honest moment, many had issues. As I look back, I truly dont remember any of the doctors explaining the literal brutality of the surgery. If they did, it would probably dramatically diminish the number of surgeries they perform. They dont talk much about the fact that it is virtually impossible to kneel on a metal knee. I never realized how much I would miss that simple act.

About four years into my quest for an alternative, a friend of mine mentioned someone she knew who had gone the stem cell route. Afterwards, I couldnt even remember who told me but Google Search became my best friend! My research kept me awake until the wee hours of the morning many nights. I was learning about the amazing world of stem cells.

There were articles from around the world and right in my own backyard. There were high profile people who had seen amazing results. Governor Rick Perry had a stem cell procedure done on his back while campaigning for President. Professional athletes were back on the field in record time after procedures on knees, backs and shoulders. Stem cell therapy is one of the best kept secrets in the medical arena.

You may wonder why you havent heard more about it. Number one is that at this point stem cell procedures are considered investigational and experimental by most insurance companies. Right now you may be thinkingwhoa! Dont stop reading, not just this article but if you or someone you love needs this, read everything you can get your hands on.

Because the procedure uses our own stem cells the FDA does not regulate them and most insurance companies dont cover the procedure. Keep reading! In most cases this is not a deal breaker. Later in this article I am going to go through the old Ben Franklin decision making formula of Pros and Cons with you.

Let me tell you how my procedure worked. I located a clinic in San Rafael that appeared to be what I was looking for. I researched Health Link Medical Centers/Regenexx and found out everything I possibly could about them. We are so fortunate in the Bay Area to have a facility nearby. This group has been doing this type of procedure for several years and have more experience than any other clinic I researched. I called and made an appointment with Dr. Paul Handleman, D.O. Yes, he is an Orthopedic Doctor who specializes in Regenerative Interventional Orthopedics. They asked if I had a MRI and if so, to bring it with me. My personal physician had ordered one for me so I was on my way. Dr. Handleman put my MRI up on a large screen and while we looked at it together, he told me about the procedure and answered my questions. He then did an ultrasound on my knee pointing out issues that couldnt be seen on my MRI. After I left, I had a few more questions that he willingly answered over the phone. I had made up my mind. Since I was a candidate, it was the answer to my search.

Let me share a few of the things I learned about Stem Cell Therapy. Number one, from what Ive read it is a bit of a political hot potato. In my opinion, the medical lobbyists have done a great job keeping it at bay. The average Total Knee Replacement will have a cost of $75,000-$100,000. This includes everything from hospital stays to operating rooms, to the various medical professionals involved to after-care and rehabilitation. Then you have drugs, crutches, walkers (oh my!) Let me just say, it is BIG Business. My Stem Cell Therapy was a total of four visits to the doctor. The first visit was my consultation. The actual procedure is a three visit process over a period of approximately a week.

The first of the three visits was very quick. The doctor answered my last minute questions and then did something I thought rather unusual. He anesthetized my knee and put several shots of what amounted to sugar water in the places I needed to grow back the cartilage and repair ligaments. The purpose of this is to literally make these areas angry so when the stem cells are injected they know where to go.

The second of the visits was two days later. I was nervous. I had heard stories from people who had donated stem cells and Im not sure if mine was different or the people that had shared had embellished but it was enough to create the nervousness. They first drew blood. They used the platelets from the blood for what is called Plasma Rich Platelets or PRP. The PRPs by themselves were some of the stem cells used for procedures. The Doctor then numbed my lower back to extract the Stem Cells from my Iliac Crest. This has proven to be the absolute best Stem Cells in our bodies.

This process actually turned out to be pretty funny. I credit a little white pill given to be by the doctor and a system that is pretty clean (other than my borderline sugar addiction) for the humor in that day. It seems that I was feeling no pain when the doctor did the extraction. My son, who was sitting in the waiting room, could hear me singing my heart out all during the procedure. He told me later when I asked, that probably everyone in the eight story building could have heard me singing and laughing.

After a nice lunch with my son, I returned to the actual stem cell injections into my knee. While we were having lunch the medical team was concentrating those cells in a lab. Regenexx SD is the process. I left that day on crutches with a couple of pain pills, just in case. There was some pain involved with the procedure but I had been getting Cortisone shots every three months directly into my knee. I was hoping to never have to get another one! I was instructed to not do a lot of walking for a couple of days. I had scheduled my procedure over a long weekend. Dr. Handleman had given me his cell phone number and told me to not hesitate to call.

I went back for my third and last appointment a few days later. The doctor drew some more blood and gave me booster shots in my knee of the stem cells. I was done. I went back to work. My knee has gradually (its growing) gotten better and better. They had told me that the growth is at least a year -long process. It has now been 9 months.

It is a little strange how you qualify the recovery. I havent had a cortisone shot in a year. When I had them before I would be great for 2-3 weeks and by the time I could get another one, I would hardly be walking. I used to schedule my injections around events or travel where I knew I would need to walk a lot. Now it just gets easier and easier. I love going to church but the congregation stands while they sing. This is sometimes 30 minutes. I just couldnt do that before. Now, I stand for the whole time and dont even think about it. I also belong to a group who collects donations for our troops every month. I can now stand for the whole 2 hour shift. Everywhere I turn there is living proof that my body is healing itself.

Lets go back to Ben Franklin. On the Pro Side I hadnon-surgical, little or no recovery time, less pain, no prolonged time away from work or commitments resulting in less loss of income, very little help needed, no aftereffects, can have it done more than once to get a better result over time, and traditional surgery is still an option if it doesnt work.

The Con Side is shorter. Will it work? Private Pay. Fear of the unknown. Process takes a year. Even after I decided, I thought long and hard about the private pay aspect. I began actually running the numbers. I called my insurance company to see what my total co-pays would be for the traditional surgery. Thats when I found out what the Total Knee Replacement cost was. With Obamacare now in place the numbers seemed to get higher and higher and no one seemed to be able to give me a clear picture. The Health Link Medical Center group gave me clear numbers. It would cost me approximately $6,200. The co-pays on the traditional procedure werent that far off and with the Stem Cell procedure I was back to work much faster not interrupting my income flow.

If you are going to opt for the Stem Cell Procedure, I would highly recommend getting on a high grade nutritional supplement as soon as possible. I have been taking great supplements for several years and rarely get sick. Little did I know that my supplements would warrant me the nickname at the clinic of Stem Cell Queen? It seems that the extra nutrition plus good hydration before the procedure provided over a billion stem cells in the extraction!

Even though I did my research and due diligence, I have intentionally not gone into the more scientific aspect of Stem Cell procedures. I am not a scientist, nor a doctor, just a very grateful patient. Between the Health Link and Regenexx websites and others (UC Davis has an amazing program) you can learn as much or as little as you like. Now, BOLDLY GO!

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Wild new microchip tech could grow brain cells on your skin – CNET

By adminAugust 10, 2017Stem Cell Medical Center

Researchers demonstrate a process known as tissue nanotransfection (TNT). When it comes to healing, this TNT is the bomb.

It's usually bad news to have something growing on your skin, but new technology uses that all important layer as a sort of garden to "grow" whatever types of cells your body might need to treat an injury or disease, be it in a limb or even the brain.

Researchers atthe Ohio State University Wexner Medical Centerhave developed a nanochip that uses a small electrical current to deliver new DNA or RNA into living skin cells, "reprogramming" them and giving them a new function.

"It takes just a fraction of a second. You simply touch the chip to the wounded area, then remove it,"Chandan Sen, director of the Center for Regenerative Medicine and Cell-Based Therapies at Ohio State, said in a statement. "At that point, the cell reprogramming begins."

In a study published in the journal Nature Nanotechnology, Sen's team used a technology called Tissue Nanotransfection (TNT) to create new blood vessels in pigs and mice with badly injured limbs that lacked blood flow.

They zapped the animals' skin with the device, and within about a week, active blood vessels appeared, essentially saving the creatures' legs. The tech was also used to create nerve cells from skin that were then harvested and injected into mice with brain injuries to help them recover.

"By using our novel nanochip technology, injured or compromised organs can be replaced," Sen said. "We have shown that skin is a fertile land where we can grow the elements of any organ that is declining."

While it sounds futuristic, reprogramming skin cells is not a new idea. The ability to change skin into pluripotent stem cells, sometimes called "master" cells, earned a few scientists a Nobel Prize half a decade ago. But the new nanochip approach improves upon that discovery by skipping the conversion from skin to stem cell and instead converting a skin cell into whatever type of cell is desired in a single step.

"Our technology keeps the cells in the body under immune surveillance, so immune suppression is not necessary," Sen says.

By now I think we've all learned that beauty is only skin deep, but it might take a while to learn that the same could go for cures, at least if the system works just as well on people.

Next up, the scientists hope to find out by continuing to test their technology in human trials. The aim is that it could eventually be used to treat all sorts of organ and tissue failure, including diseases like Parkinson's and Alzheimers.

Crowd Control: A crowdsourced science fiction novel written by CNET readers.

Solving for XX:The tech industry seeks to overcome outdated ideas about "women in tech."

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Techshot system headed to space – Evening News and Tribune

By adminAugust 10, 2017Stem Cell Medical Center

GREENVILLE Onboard the next SpaceX cargo spacecraft launching to the International Space Station (ISS) from Pad 39A at the Kennedy Space Center will be a commercial research system owned and operated by Techshot Inc. The equipment will conduct regenerative medicine experiments onboard the station before returning to Earth in the same capsule for a splashdown off the coast of Southern California approximately 30 days later.

Techshots ADvanced Space Experiment Processor (ADSEP) is a device approximately the size of a microwave oven that contains three separate modules, each of which simultaneously can process experiments in three separate on-orbit replaceable automated mini-laboratory cassettes. Two of the three cassettes on the mission will conduct research for a team led by Robert Schwartz, Ph.D., from the University of Houston.

Funded by the Center for the Advancement of Science in Space (CASIS), the study will evaluate a new approach to growing human tissue for transplant. The microgravity environment onboard the ISS could improve cell growth and development and 3D tissue formation, enabling discoveries that will advance translational disease treatments. Previous studies on Earth by Schwartz and his collaborators at the Texas Heart Institute and the Baylor College of Medicine have found that low gravity environments help specially programmed stem cells move toward becoming new heart muscle cells, which may be used to repair damaged hearts on Earth.

The third cassette contains an experiment conducted by and for Techshot itself. The company is developing a 3D bioprinter for the ISS known as the Techshot BioFabrication Facility (BFF), which it expects to launch to the station near the end of 2018. Critical to the success of the printer will be the ability to provide nutrients and mechanical stress for organs and tissues it manufactures in space strengthening them and keeping them viable for transplantation back on Earth.

Approximately 36 hours prior to launch, Techshot scientists in a laboratory at the Kennedy Space Center will 3D print a one centimeter thick construct consisting of stem cells and heart muscle cells. Theyll then place it inside the prototype BFF cell culturing subsystem, which for this mission is temporarily housed inside an ADSEP cassette. The printer used in the lab will be the same modified nScrypt unit that was the first to 3D print cardiac constructs with adult human stem cells in microgravity aboard an aircraft in parabolic flight. Video captured inside the cassette during the month-long experiment, and the tissue itself which is expected to have developed its own micro blood vessels will be evaluated for effectiveness after return from space.

Techshots space bioprinting program leverages its terrestrially based technologies for cell isolation and vascular graft development, and its decades long experience culturing cells in space, said Techshot Chief Scientist Eugene Boland, Ph.D., in a news release. Being able to test our novel approach for culturing 3D printed cells more than a year before we fly the whole BFF is invaluable. The data from this mission will get us one step closer toward our goal of helping eliminate organ shortages.

Founded in 1988, Techshot Inc., develops technologies used in the aerospace, defense and medical industries. Through its Space Act Agreement with NASA, and its role as an official CASIS Implementation Partner, the company provides equipment and services that help federal, institutional and industrial customers live and work in space. http://www.Techshot.space

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Temple native’s sickle cell case to be featured on Discovery program – Temple Daily Telegram

By adminAugust 10, 2017Stem Cell Medical Center

Deidra Flowers-Williams ended up in Building 10 of the National Institutes for Health in 2015 because she was out of options.

Flowers-Williams had sickle cell anemia and the toll it had taken on her body over the years had left her in constant pain and too weak to walk from her bed to the bathroom.

After a stem cell transplant at the NIH hospital on Nov. 19, 2015, Flowers is free of the disease and has not had a sickle cell crisis since the procedure.

Her story, along with three other NIH patients, is the subject of the three-part documentary, First in Human, airing tonight on the Discovery Channel. Flowers-Williams story will be featured in the third segment on Aug. 24.

Narrated by Jim Parsons of the Big Bang Theory, the series follows the diverse patients as they participate in a First in Human trial the initial time when a new therapy is tested in humans.

Deciding to pursue a spot in a medical trial can be daunting. There are lots of hoops to jump through to get accepted and there is always the chance that nothing will change or it might kill you, Flowers-Williams said.

She was willing to take the risk.

Tanisha Flowers, her sister, provided the stem cells. The sisters were a 100 percent match.

Sickle cell anemia is a disease in which the body produces abnormally shaped red blood cells. The cells are shaped like a crescent or sickle. They don't last as long as normal, round red blood cells. This leads to anemia. The sickle cells also get stuck in blood vessels, blocking blood flow. This can cause pain and organ damage.

Flowers-Williams was No. 43 in the NIH clinical trial on using stem cells to cure sickle cell in adults. The first transplant took place about 13 years ago and that patient is alive and free of sickle cells.

Flowers-Williams returns to the NIH every six months for a checkup.

On my last visit, everything was good, she said.

Shes had to get all-new immunizations since her slate was swept clean when her sisters stem cells took over.

The therapy couldnt fix what was damaged before the stem cell transplant.

You know I lived with sickle for 39 to 40 years and Im so grateful to be here today, Flowers-Williams said.

She said her success is hope for individuals with sickle cell anemia.

There is medical work being done that can ease our suffering, she said.

Flowers-Williams said she was excited about the series because it highlights the NIH and its work.

Those scientists and researchers are working every day to cure diseases and sickle cell is only one of many, she said. I believe the stories of the four patients will touch people and bring focus to the institution.

The research center has already come up with another treatment for sickle cell patients who dont have a stem cell match, Flowers-Williams said.

Prior to my treatments I had no prior knowledge of what they did, Flowers-Williams said. This has opened up a whole new world for me. Im so happy the National Institutes of Health is getting this exposure.

Flowers-Williams has a job, an impossibility for years. Her teenage children have a new mother.

They have only known me as being sick, she said. Were making new memories.

Ethel Flowers, a nursing instructor at Temple College, her daughter and family were in Los Angeles recently to promote First in Human.

Dee Dee was on a panel with the other patients discussing their experiences, Flowers said. They asked everybody on the panel questions, but I only focused on her.

Flowers said the family has viewed bits and pieces of the series, but havent seen it in its entirety.

The producers were supposed to send me the program on a DVD, because I told them I was old school, Flowers said. As of Wednesday the DVD hadnt arrived.

Her daughter is doing great, according to Flowers.

She has normal people problems now, like arthritis, she said.

Over a period of a year, film crews from the Discovery Channel were embedded within the hospital and followed four patients who volunteered to participate in experimental treatments in the hopes they will help them, or others in the future. The series also follows the doctors and nurses who carry out the research while caring for the patients.

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Fighting Pediatric Sickle Cell Anemia – Touro College News

By adminAugust 10, 2017Uncategorized

This is the question TouroCOM student Stephanie Chung examined this summer at the pediatric hematology oncology department at Akron Childrens Hospital in Ohio. Chung, who has done research at Cornell University in the past, said that the general usage rate for the drug is between 10-25 percent (the number at Akron is significantly higher, between 90-100 percent). There are a variety of reasons for the underutilization, Chung explained, from lack of communication between primary care providers and oncologists to the intense clinical schedule that hydroxyurea requires.

Parents might also be leery of the drug since it is also used in cancer treatment. As a volunteer EMT in her hometown of Lodi, NJ, Chung said she was used to working with the elderly, but working with parents and children is a new experience for her. When you treat adults its a one-on-one relationship, said Chung. As a pediatrician, you have to work with both the parent and the patient. She plans to publish a paper about the underutilization of hydroxyurea and an additional paper about the social media discussions occurring around the drug.

Chung also believes that a new approach is needed for the drug.

Hydroxyurea decreases the rate of organ damage, said Chung. The way you have to approach the use of the drug is that its a preventative measure instead of using it for a pain crisis.

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Immunotherapy Shown Safe in Type 1 Diabetes Clinical Trial – Sioux City Journal

By adminAugust 10, 2017Uncategorized

WEDNESDAY, Aug. 9, 2017 (HealthDay News) -- A small clinical trial showed an immune system therapy was safe for people with type 1 diabetes, British researchers report.

The immunotherapy also showed signs of helping to keep insulin production steady in people newly diagnosed with the disease, the study authors said. However, because this was a placebo-controlled safety trial, there weren't enough people included to know for sure how well the treatment works.

The therapy is similar to an allergy shot in the way it works, the researchers explained.

"Type 1 diabetes comes about when the immune system inadvertently and irreparably damages beta cells that make insulin," said one of the study's authors, Dr. Mark Peakman. He's a professor of clinical immunology at King's College London in England.

Insulin is a naturally occurring hormone that helps usher the sugar from foods into the body's cells to be used as energy. If the immune system continues to attack the beta cells, which are found in the pancreas, a person with type 1 diabetes will no longer make enough insulin to meet the body's needs. It's at this point that they must take insulin injections or use an insulin pump to replace the lost insulin.

Peakman and his colleagues are trying to stop the attacks on the beta cells.

"We have learned that immune attacks like this can be suppressed by immune cells called T-regs (regulatory T cells)," Peakman said.

When people develop type 1 diabetes, it's likely that they don't have enough of the right type of T-regs or those T-regs aren't working very well. So, the investigators developed a type of treatment called peptide immunotherapy using disease-related autoantigens.

Autoantigens are the substances that cause an autoimmune attack, but it's not clear which ones are responsible for a person's diabetes, according to Simi Ahmed, a senior scientist at JDRF (formerly the Juvenile Diabetes Research Foundation) in New York City.

Ahmed said the immunotherapy re-educates the immune system, and teaches the cells that they shouldn't attack the beta cells.

Peakman pointed out that the researchers "used peptide immunotherapy as a way to try and induce more of these cells and/or make them work better. Our results show encouraging signs that this can be achieved. Next steps will be bigger trials to test whether the therapy can halt beta cell damage."

And, he added, "Scientists think this works by enhancing natural immune networks that control inflammation."

The study included 27 people with type 1 diabetes who had been diagnosed with the disease within 100 days. The study volunteers were randomly selected to be in one of three groups: a placebo group; a group given immunotherapy once every four weeks; and a group that received the immunotherapy injection once every two weeks.

The study team measured levels of a substance called C-peptide, which is created when insulin is produced. Stable or increasing levels of C-peptide indicate that insulin is being made. Declining levels indicate that less insulin is being made.

The volunteers given immunotherapy once every four weeks saw no decline in C-peptide, the findings showed. The group given the treatment once every two weeks saw a decrease in C-peptide at the 12-month mark. Those given a placebo had decreased C-peptide levels at 3, 6, 9 and 12 months.

Peakman said there weren't enough people in the study to know why there were slight differences in the treatment groups. That's for a larger study to figure out, he suggested.

But the researchers found that the treatment was quite safe, and there were no side effects "of note," Peakman added.

The study also looked at insulin use. Over 12 months, insulin use went up by 50 percent in the placebo group. But there was no increase in the treated groups.

And even though all the study volunteers still needed insulin, both diabetes experts said that the need for less insulin would likely improve quality of life and lessen complications.

It's not yet clear from this trial how often the treatment would need to be given to keep the immune attack at bay.

It's also not clear if it could benefit people who've had type 1 diabetes for a while. Peakman said it probably wouldn't help them because most of their beta cells would be gone. But Ahmed noted that if someone had detectable C-peptide levels, it's possible that this treatment or future ones that may contain more than one autoantigen might have an effect.

In December 2016, a study published in Diabetologia found that about one-third of people with type 1 diabetes for at least 10 years still had detectable C-peptide levels. And two-thirds of them showed signs of an ongoing immune system attack.

Findings from the new study were published Aug. 9 in Science Translational Medicine.

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Immunotherapy Shown Safe in Type 1 Diabetes Clinical Trial - Sioux City Journal

Stem cells may treat lung fibrosis diseases – Futurity: Research News

By adminAugust 10, 2017Uncategorized

Researchers have taken a step towards a possible treatment for several often-fatal lung conditions that affect millions of Americanssuch as idiopathic pulmonary fibrosis (IPF), chronic obstructive pulmonary disease (COPD), and cystic fibrosisby harvesting and using lung stem cells.

In research appearing in the journal Respiratory Research, the scientists demonstrated that they could harvest lung stem cells from people using a relatively non-invasive, doctors-office technique. They were then able to multiply the harvested lung cells in the lab to yield enough cells sufficient for human therapy.

In a second study, which appears in the journal Stem Cells Translational Medicine, the team showed that, in rodents, they could use the same type of lung cell to successfully treat a model of IPFa chronic, irreversible, and ultimately fatal disease characterized by a progressive decline in lung function.

The researchers have been in discussions with the FDA and are preparing an application for an initial clinical trial in patients with IPF.

This is the first time anyone has generated potentially therapeutic lung stem cells from minimally invasive biopsy specimens, says co-senior author of both papers Jason Lobo, an assistant professor of medicine at the University of North Carolina at Chapel Hill and medical director of lung transplant and interstitial lung disease.

We think the properties of these cells make them potentially therapeutic for a wide range of lung fibrosis diseases.

Co-senior author Ke Cheng, an associate professor in North Carolina State Universitys molecular biomedical sciences department and UNC/NCSUs joint biomedical engineering department, says, We think the properties of these cells make them potentially therapeutic for a wide range of lung fibrosis diseases.

These diseases of the lung involve the buildup of fibrous, scar-like tissue, typically due to chronic lung inflammation. As this fibrous tissue replaces working lung tissue, the lungs become less able to transfer oxygen to the blood. Patients ultimately are at risk of early death from respiratory failure.

In the case of IPF, which has been linked to smoking, most patients live for fewer than five years after diagnosis.

The two FDA-approved drug treatments for IPF reduce symptoms but do not stop the underlying disease process. The only effective treatment is a lung transplant, which carries a high mortality risk and involves the long-term use of immunosuppressive drugs.

Scientists have been studying the alternative possibility of using stem cells to treat IPF and other lung fibrosis diseases. Stem cells are immature cells that can proliferate and turn into adult cells in order to, for example, repair injuries. Some types of stem cells have anti-inflammatory and anti-fibrosis properties that make them particularly attractive as potential treatments for fibrosis diseases.

Cheng and Lobo have focused on a set of stem cells and support cells that reside in the lungs and can be reliably cultured from biopsied lung tissue. The cells are called lung spheroid cells for the distinctive sphere-like structures they form in culture.

As the scientists reported in an initial paper in 2015, lung spheroid cells show powerful regenerative properties when applied to a mouse model of lung fibrosis. In their therapeutic activity, these cells also outperform other non-lung-derived stem cells known as mesenchymal stem cells, which are also under investigation to treat fibrosis.

In the first of the two new studies, Lobo and his team showed that they could obtain lung spheroid cells from human lung disease patients with a relatively non-invasive procedure called a transbronchial biopsy.

We snip tiny, seed-sized samples of airway tissue using a bronchoscope, Lobo says. This method involves far less risk to the patient than does a standard, chest-penetrating surgical biopsy of lung tissue.

Cheng and his colleagues cultured lung spheroid cells from these tiny tissue samples until they were numerous enoughin the tens of millionsto be delivered therapeutically. When they infused the cells intravenously into mice, they found that most of the cells gathered in the animals lungs.

These cells are from the lung, and so in a sense theyre happiest, so to speak, living and working in the lung, Cheng says.

For the second study, the researchers first induced a lung fibrosis condition in rats. The condition closely resembled human IPF. Then the researchers injected the new cultured spheroid cells into one group of rats. Upon studying this group of animals and another group treated with a placebo, the researchers saw healthier overall lung cells and significantly less lung inflammation and fibrosis in the rats treated with lung spheroid cells.

Our vision is that we will eventually set up a universal cell donor bank

Also, the treatment was safe and effective whether the lung spheroid cells were derived from the recipients own lungs or from the lungs of an unrelated strain of rats, Lobo says. In other words, even if the donated stem cells were foreign, they did not provoke a harmful immune reaction in the recipient animals, as transplanted tissue normally does.

Lobo and Chen expect that when used therapeutically in humans, lung spheroid cells initially would be derived from the patient to minimize any immune-rejection risk. Ultimately, however, to obtain enough cells for widespread clinical use, doctors might harvest them from healthy volunteers, as well as from whole lungs obtained from organ donation networks. The stem cells could later be used in patients as-is or matched immunologically to recipients in much the same way transplanted organs are typically matched.

Our vision is that we will eventually set up a universal cell donor bank, Cheng says.

Cheng, Lobo, and their teams are now planning an initial study of therapeutic lung spheroid cells in a small group of IPF patients and expect to apply later this year for FDA approval of the study. In the long run, the scientists hope their lung stem cell therapy will also help patients with other lung fibrosis conditions of which there are dozens, including COPD, cystic fibrosis, and fibro-cavernous pulmonary tuberculosis.

Additional researchers contributing to this work are from UNC, NC State, the joint UNC-NC State biomedical engineering department, and two hospitals in Shijiazhuang, China. The National Institutes of Health, the UNC General Assembly Research Opportunities Initiative, and the NC State Chancellors Innovation Fund funded this research.

Source: UNC-Chapel Hill

ASU grad students’ lab skills help earn funding for cutting-edge biomedical research – Arizona State University

By adminAugust 10, 2017Induced Pluripotent Stem Cells

August 8, 2017

While clues to treating diseases that ravage the body and mind later in life sometimes appear in early stages of human development, studying a subjects entire lifespan is neither efficient nor practical.

So how can researchers study these early stages to combat diseases that manifest themselves later on? The stem cell wizards of ASUs Brafman Lab. Left to right: Sreedevi Raman, Josh Cutts, Nick Brookhouser and Christopher Potts. Photo by Marco-Alexis Chaira/ASU Download Full Image

If you guessed through the use of pluripotent stem cells so named because they have the ability to turn into other types of cells then youre right on the money.

ASUs Brafman Labis on the cutting edge of this branch of research, recently earning a $1.5 million grant from the National Institutes of Health to study the mechanisms of early human neurodevelopment, and $225,000 from the Arizona Biomedical Research Commission to study the effects of aging and other risk factors for Alzheimers Disease.

Biomedical engineering Assistant Professor David Brafman, who heads the laboratory, credits his graduate students as crucial in securing these grants

The graduate students hard work, creativity and dedication were critically important for generating the data to convince the reviewers that our approach was feasible and worth funding, Brafman says of his students. Too often the success of a lab is attributed to the [principal investigator] when it is the postdocs, grad students and research technicians who are down in the trenches doing the work.

Graduate students from the Brafman Lab reviewing data. Photo by Marco-Alexis Chaira/ASU

The students working in the Brafman Lab often labor late into the night and sometimes on the weekend. They possess a special mix of a passion for their work and the knowledge that achieving potentially life-altering outcomes dont come with a simple nine-to-five job.

The laboratory they work in combines developmental biology, genetic engineering and bioinformatics to investigate the various factors that can govern a stem cells fate. If they can figure out the mechanisms behind the stem cells multipotential futures, they could use that information to design targeted therapies for ailments like idiopathic pulmonary fibrosis, heart failure and Alzheimers Disease.

Take Josh Cutts, who is pursuing his doctoral degree in biomedical engineering. He knows a thing or three about working in the Brafman Lab. Hes addicted to the thrill of discovery, regardless of any challenges or obstacles that may come his way.

Were working on things that havent been done before so its challenging sometimes frustrating to complete certain experiments or understand the results, he said.

The shapeshifting nature of the stem cells can make working with them seem like biological wizardry. In the lab the research team has made stem cells into brain cells, heart cells, lung cells and more.

Now we are working with cutting-edge brain organoids known colloquially as mini brains, which sounds a little eerie, to address many different research questions, said Cutts, who earned his bachelors and masters degrees in biomedical engineering at California Polytechnic State University, San Luis Obispo. Its miraculous to work with these every day.

Cutts work generated the preliminary data that helped the lab secure the NIH grant. After finishing his graduate work, this pluripotent scholar plans to earn a post doctorate degree to expand his knowledge and expertise. Long term, he hopes to contribute to translating stem cell technology to patients, in academia or industry.

Researcher Nick Brookhouser is working toward his doctorate in clinical translational sciences at the University of Arizonas College of Medicine in Phoenix. His research in the Brafman Lab is focused on Alzheimers Disease and investigating the contribution of the Apolipoprotein E gene, or APOE, towards the diseases progression.

He has successfully generated a set of stem cell lines from Alzheimers patients as well as other stem cell lines that serve as the control group in his research. He is currently working with gene editing techniques to investigate APOEs relationship to Alzheimers.

Brookhousers work is also supported by an Arizona Biomedical Research Commission grant. He developed patient-specific pluripotent stem cell lines and brain cell lines, and with those lines he created a 3-D neural culture system that models a brain for study. He has also been involved in testing and optimizing gene editing technologies.

In the future, he hopes to transition to more clinical-based research in the biotechnology industry. Long term he hopes to contribute to the development of cell-based therapies and work in clinical trials.

Doctoral student Sreedevi Raman has also been working on research related to Alzheimers Disease. Instead of experimenting with stem cells at their genesis, Raman is trying to make them old. She is intentionally accelerating the aging process of cells in a dish so that they may be used to model various age-related disorders.

Her work with induced pluripotent stem cells specifically has helped the Brafman Lab attain the ABRC grant. Raman can take adult stem cells and program them back into state where their fate is not yet assigned.

Christopher Potts, a research specialist with a professional science masters degree from ASU, works with gene editing. His contribution to the team is comparable to using copy and paste for genes, but a bit more complicated. Hes using technologies like CRISPR (Clustered regularly interspaced short palindromic repeats) to edit stem cell genomes.

I am changing the DNA of stem cells. Thats pretty cool, right? Potts said. I think one of the coolest things about our lab is how each student has their own project and functions basically independently, but we all help each other and are able to do much more than we could on our own.

Hes enjoying his research, but also looks forward to teaching a new generation of students in the future. He has a masters degree in science education and taught high school for four years before joining the lab.

Potts has aspirations of starting a new line of scientists through a, career in outreach or other high-level science education.

The cells he works on use signaling pathways to regulate what they will become like his multiple career options. Right now, I am just hoping for some signals to help me differentiate, he said.

Just as Brafman relies on the hard work of his students, the entire lab team relies on one another to succeed.

Our lab is pretty close-knit. We like to hang out together to socialize and I think that support system makes our lab more effective, Cutts said. If any of us are having a hard time with experiments or anything at all, you can rely on your lab members and especially [Brafman] to help you work it out.

Like Cutts, Brookhouser values the highly collaborative environment in the lab that has fostered strong professional relationships as well as lasting friendships.

Just as patient somatic cells can be reprogrammed to a pluripotent state, I feel that the skills and mentorship I have gained in this lab have allowed me to reach a pluripotent state and primed me to differentiate down many different career paths in the future, Brookhouser said.

Raman credits her positive collaborative learning experiences in the lab with helping her to make advances in research as well as open career possibilities for her future. Since she just started her doctoral work, shes got a lot of research ahead of her. Luckily, she found a good place to start.

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ASU grad students' lab skills help earn funding for cutting-edge biomedical research - Arizona State University