Bone marrow transplants are used in serious blood disorders, especially cancers, when the needed doses of chemotherapy or radiation would be so high it would damage or destroy the stem cells in the marrow.

WINTER HAVEN For 14 years, Dr. Christopher Miller has been treating patients at Bond Clinic where he specializes in endocrinology, diabetes and metabolism. Many local people have met him at Bonds diabetes clinic or in nearby Eloise where he volunteers at Angel Cares free clinic.

Organizers of a Be The Match drive are hoping that those who have benefited from his care, including families and friends of patients, will turn out to honor him Saturday by volunteering to be a bone marrow donor.

He received a shocking, out-of-the-blue diagnosis and is in need of a bone marrow match, said Ashley Scanlan, marketing director for Bond Clinic.

Bone marrow is the soft tissue inside bones where blood cells are produced. Transplants are used in serious blood disorders, especially cancers, when the needed doses of chemotherapy or radiation would be so high it would damage or destroy the stem cells in the marrow.

Be The Match, a national nonprofit organization that is part of the National Marrow Donor Program, is the largest registry matching donors with those in need of a marrow transplant, said Marc Silver, community engagement representative for Be The Match. It also provides support for patients and donors, information for health care professionals and conducts research.

Nearly 70 percent of people needing a marrow transplant do not have a match within their families so the registry was set up to provide a resource for matches.

The event is from 8to 11 a.m. Saturday at the Bond Clinic Main Campus, 500 E. Central Ave., Winter Haven.

Registering to be a donor is a simple process, filling out some paperwork and taking a mouth swab, Scanlan said.

Volunteers should be between 18 and 44 years old, generally in good health and be willing to donate to any patient in the future, Scanlan said.

People are asking why the cutoff is 44, but they have found that age group has the best success in transplants, Scanlan said.

People of other ages are invited to come Saturday and write a note toMiller or make a financial donation, which would go either to the American Cancer Society or to the local Angel Care clinic, she said.

Bobbie Skukowski, an advanced registered nurse practitioner who leads Bonds diabetes clinic, said, Dr. Miller is an excellent physician and an excellent teacher. He was a fellow at Emory University and has taught us all so much; he has brought up the level of diabetes care at Bond Clinic and in the Winter Haven area in general.

"He is very good with his patients and right-on in his care, she said.

If a person is later selected as a potential match, there is no cost to the donor, Scanlan said. And the potential donor can later decide to withdraw from the registry.

The paperwork will ask several questions, including whether the potential donor is willing to donate to any patient in need, willing to donate to a stranger, and willing to donate 20 to 30 hours if found to be a perfect match.

If the potential donor meets the criteria, a mouth swab is taken and later analyzed for a match.

While years ago, being a bone marrow donor was a complicated procedure, now it typically is simple, handled much like a blood donation, Scanlan said.

Over 80 percent of the donations are non-invasive, said Be The Matchs spokesman Silver.

Be The Match literature explains that the donor is given injections of a drug, filgrastim, for five days leading up to the donation to increase the number of stem cells in the blood.

Then, on the day of the donation, the donor goes through a procedure similar to donating blood platelets at a blood center. Blood is taken out of one arm, passed through a machine that collects the blood-forming stem cells, and then the red and white blood cells are returned to the donors other arm through a needle. Typically it takes eight hours.

Donors often have a headache or muscle aches for a few days 22 percent recover within two days, 53 percent within a week, 93 percent within a month, 99 percent within three months and a very few people can take as long as a year to recover, according to Be The Match.

Less than 20 percent of the time, we do a hip aspiration, which is a more complicated procedure and involves having anesthesia in an operating room, Silver said.

Be The Match literature explains that, in those cases, needles are used to withdraw liquid marrow from both sides of the back of the pelvic bone. Typically, the donor stays at the hospital from early morning to late afternoon, or occasionally overnight for observation.

Be The Match helped match 6,200 patients for marrow and cord blood transplants last year and added 472,000 new potential donors to the registry, according to the organization.

Marilyn Meyer can be reached at marilyn.meyer@theledger.com or 863-802-7558. Follow her on Twitter @marilyn_ledger.

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Bone marrow drive to help and honor Bond Clinic physician - The Ledger

Bone Marrow Donors Needed

Paula (Fitzgerald) Silvia of Middletown has been diagnosed with myeloid dysplasia syndrome (MDS), a form of blood cancer where the bone marrow cells do not mature into healthy blood cells. Paula received this devastating news at the end of June. Nothing seemed particularly out of sorts; she was travelling with her family, working, going to the beach and doing all her volunteer activities. Only indications were catching more colds and a little tired but Paulas life was always busy and she does so much for everyone, she should be tired.

Paula has started her first round of chemotherapy. Every four weeks, she has five consecutive days of chemotherapy infusion. However, it is only a temporary treatment. A bone marrow transplant is needed to cure the disease.

A bone marrow registration drive is being held on Sunday, September 17 from 4:00pm to 7:00pm at Fenner Hall, 15 Fenner Ave, Newport, for this purpose. It is being organized by her family and friends and is in conjunction with Dana-Farber Cancer Institute and http://www.BeTheMatch.org, a national bone marrow donor registry.

The first source for bone marrow matches is a sibling or child, if they fall in the age range, but Paulas family members were not a match. More than 35,000 people in the United States are diagnosed each year with leukemia, anemias, myelodysplastic disorders and other life-threatening diseases requiring treatment with a blood stem cell or bone marrow transplant. About 70 percent of bone marrow transplant recipients must rely on an unrelated donor. Finding a compatible donor is a challenge. The opportunity to register and/or donate will help many patients in need..

Donors must be 18 to 44, and be willing to donate to any patient in need.To join the registry, potential donors must complete paperwork at the drive and have a cheek swab taken. If unable to attend, donors are asked to go towww.bethematch.orgto sign up, or visit any RI Blood Center.

Paula (Fitzgerald) grew up in Newport in the Fifth Ward, attending Newport schools, graduating from Rogers in 1968. Her father, Jim Fitzgerald, was the Dean of Boys at Thompson and football coach and her mother Meg also worked in the school system. She has an older sister, Maureen, and younger siblings, Nancy and Bill. Paula is an outstanding athlete, tennis and golf being her games of recent years. After graduating from college, Paula continued working at Salas until it closed and now works for private catering companies. TR McGrath and Kitchen Companion.

Paula is married to Manny P Silvia, a retired lieutenant in Middletown police department and retired supervisor in DCYF Protective Services. They have two children, Corrine and Greg.

Paula does an amazing amount of volunteering although never wanting any recognition for her efforts. She volunteers for many organizations such as the MLK Community Center, Relay for Life, the Ladies Ancient Order of Hibernians, Mosaic Club, AARP school programs, and Vasco deGama Society. Shes a communicant of St. Augustins Church.

The news of her diagnosis is a shock, but Paula continues with her active, involved life, giving it her best. She wants to send the message that everyone should be proactive about their health and always follow up on lab work. Paula is now awaiting a bone marrow transplant!

Any questions, please contact Nancy Fitzgerald, nancyfitz53@gmail.com, 401-855-1985. To learn more please contact Dana-Farbers Bone Marrow Donor Program at866-875-3324, email nmdpdonor@dfci.harvard.edu or visit online http://www.bethematch.org

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Bone Marrow Drive To Benefit Paula Fitzgerald Silvia On Sunday, September 17th - Newport Buzz

Advancellssays its stem cell-based therapy completely reversed multiple sclerosis (MS) in an Indian pilot trial with only one MS patient.

The patient, Rahul Gupta, was diagnosed with MS seven years ago and has since suffered multiple relapses. His disease was progressing fast and he was quickly losing his ability to walk. Gupta, who lives in New Zealand, approached Advancells a company based in the Indian state of Uttar Pradesh that specializes in the use of stem cells for therapeutic purposes.

After my last relapse, I became determined to look for alternative treatments for multiple sclerosis,Gupta said in a press release. I started looking on the net and found that stem-cell therapy [offers] hope for people suffering with MS [and] that it is safe and would not harm me in any way. I was determined to undergo stem-cell treatment, as my illness was progressing very quickly.

Gupta enrolled inAdvancells adult stem-cell therapy program as the trials single patient. In the procedure carried outin June at a New Delhi clinic doctors isolated stem cells from his bone marrow and re-infused them back into the patientat specific points. Apart from this procedure, Gupta underwent only physiotherapy and a dietary routine.

Straight after the treatment I saw major improvements, he said. I could walk a lot better, could climb stairs which I was unable to do after 2012 and even go on the treadmill.

Dr. Lipi Singh, head of technology at Advancells, said the company is frequently approached by MS patients from around the world who want to participate in its program.

Patient selection is a key criterion for us and Rahul suited the criteria perfectly, Singh said. He is young and still at a moderate level of the disease and in a very positive frame of mind. Patients at this stage are best suited for this kind of treatment and thus we decided to accept him as a pilot case.

Singh now expects to review Guptas response sometime this fall.

It will take approximately three months for us to review changes in the magnetic resonance imaging of the patient, but the drastic changes in symptoms clearly are an indication of the fact that the treatment is working and could become a hope for millions of patients across the world who are suffering from this disease. Singh said.

He added: This is a good start to a lengthy research phase, but it seems that we are on the right track and hopefully we will be able to make a significant contribution in eradicating not only MS but a host of untreatable diseases existing today.

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India's Advancells Reports Successful Reversal of MS in Single Patient Using Stem Cell Therapy - Multiple Sclerosis News Today

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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Techshot system headed to space | News | newsandtribune.com - Evening News and Tribune

SOUTH SAN FRANCISCO, CA--(Marketwired - August 08, 2017) - VistaGen Therapeutics Inc. (NASDAQ: VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, announced today that the Company has received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for U.S. Patent Application No. 14/359,517 regarding proprietary methods for producing hematopoietic precursor stem cells, which are stem cells that give rise to all of the blood cells and most of the bone marrow cells in the body, with potential to impact both direct and supportive therapy for autoimmune disorders and cancer.

The breakthrough technology covered by the allowed U.S. patent was discovered and developed by distinguished stem cell researcher, Dr. Gordon Keller, Director of the UHN's McEwen Centre for Regenerative Medicine in Toronto, one of the world's leading centers for stem cell and regenerative medicine research and part of the University Health Network (UHN), Canada's largest research hospital. Dr. Keller is a co-founder of VistaGen and a member of the Company's Scientific Advisory Board. VistaGen holds an exclusive worldwide license from UHN to the stem cell technology covered by the allowed U.S. patent.

"We are pleased to report that the USPTO has allowed another important U.S. patent relating to our stem cell technology platform, stated Shawn Singh, Chief Executive Officer of VistaGen. "Because the technology under this allowed patent involves the stem cells from which all blood cells are derived, it has the potential to reach the lives of millions battling a broad range of life-threatening medical conditions, including cancer, with CAR-T cell applications and foundational technology we believe ultimately will provide approaches for producing bone marrow stem cells for bone marrow transfusions. As we continue to expand the patent portfolio of VistaStem Therapeutics, our stem cell technology-focused subsidiary, we enhance our potential opportunities for additional regenerative medicine transactions similar to our December 2016 sublicense of cardiac stem cell technology to BlueRock Therapeutics, while focusing VistaStem's internal efforts on using stem cell technology for cost-efficient small molecule drug rescue to expand our drug development pipeline."

About VistaGenVistaGen Therapeutics, Inc. (NASDAQ: VTGN), is a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders. VistaGen's lead CNS product candidate, AV-101, is in Phase 2 development, initially as a new generation oral antidepressant drug candidate for major depressive disorder (MDD). AV-101's mechanism of action is fundamentally different from all FDA-approved antidepressants and atypical antipsychotics used adjunctively to treat MDD, with potential to drive a paradigm shift towards a new generation of safer and faster-acting antidepressants. AV-101 is currently being evaluated by the U.S. National Institute of Mental Health (NIMH) in a small Phase 2 monotherapy study in MDD being fully funded by the NIMH and conducted by Dr. Carlos Zarate Jr., Chief, Section on the Neurobiology and Treatment of Mood Disorders and Chief of Experimental Therapeutics and Pathophysiology Branch at the NIMH. VistaGen is preparing to launch a 180-patient Phase 2 study of AV-101 as an adjunctive treatment for MDD patients with an inadequate response to standard, FDA-approved antidepressants. Dr. Maurizio Fava of Harvard University will be the Principal Investigator of the Company's Phase 2 adjunctive treatment study. AV-101 may also have the potential to treat multiple CNS disorders and neurodegenerative diseases in addition to MDD, including neuropathic pain, epilepsy, Huntington's disease, and levodopa-induced dyskinesia associated with Parkinson's disease and other disorders where modulation of the NMDA receptors, activation of AMPA pathways and/or key active metabolites of AV-101 may achieve therapeutic benefit.

About VistaStemVistaStem Therapeutics is VistaGen's wholly-owned subsidiary focused on applying human pluripotent stem cell (hPSC) technology, internally and with third-party collaborators, to discover, rescue, develop and commercialize (i) proprietary new chemical entities (NCEs), including small molecule NCEs with regenerative potential, for CNS and other diseases and (ii) cellular therapies involving stem cell-derived blood, cartilage, heart and liver cells. VistaStem's internal drug rescue programs are designed to utilize CardioSafe 3D, its customized cardiac bioassay system, to develop small molecule NCEs for VistaGen's pipeline. To advance potential regenerative medicine (RM) applications of its cardiac stem cell technology, in December 2016, VistaStem exclusively sublicensed to BlueRock Therapeutics LP, a next generation regenerative medicine company established in 2016 by Bayer AG and Versant Ventures, rights to certain proprietary technologies relating to the production of cardiac cells for the treatment of heart disease. In a manner similar to its exclusive sublicense agreement with BlueRock Therapeutics, VistaStem may pursue additional collaborations and potential RM applications of its stem cell technology platform, including using blood, cartilage, and/or liver cells derived from hPSCs, for (i) cell-based therapy, (ii) cell repair therapy, and/or (iii) tissue engineering.

For more information, please visit http://www.vistagen.com and connect with VistaGen on Twitter, LinkedIn and Facebook.

Forward-Looking StatementsThe statements in this press release that are not historical facts may constitute forward-looking statements that are based on current expectations and are subject to risks and uncertainties that could cause actual future results to differ materially from those expressed or implied by such statements. Those risks and uncertainties include, but are not limited to, risks related to the successful launch, continuation and results of the NIMH's Phase 2 (monotherapy) and/or the Company's planned Phase 2 (adjunctive therapy) clinical studies of AV-101 in MDD, and other CNS diseases and disorders, including neuropathic pain and L-DOPA-induced dyskinesia associated with Parkinson's disease, the potential for the Company's stem cell technology to produce NCEs, cellular therapies, regenerative medicine or bone marrow stem cells to treat any medical condition, including autoimmune disorders and cancer, protection of its intellectual property, and the availability of substantial additional capital to support its operations, including the AV-101 clinical development activities described above. These and other risks and uncertainties are identified and described in more detail in VistaGen's filings with the Securities and Exchange Commission (SEC). These filings are available on the SEC's website at http://www.sec.gov. VistaGen undertakes no obligation to publicly update or revise any forward-looking statements.

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VistaGen Receives Notice of Allowance from US Patent and Trademark Office for US Patent regarding Breakthrough ... - Marketwired (press release)

Technologies that reprogram one type of cell to perform the role of another hold a huge amount of potential when it comes to medicine, possibly changing the way we treat everything from Parkinson's to pancreatic cancer to brain tumors. One broader outcome of all of this could be a game-changing ability to repair and restore damaged tissue and organs. Scientists are now reporting a promising advance in the area, in the form of patch that they say can use an electric pulse to turn skin cells into the building blocks of any organ.

The new technology has been dubbed tissue nanotransfection and was developed by scientists at The Ohio State University's Wexner Medical Center. According to the researchers, it uses the skin as a kind of regenerative cellular factory, where it produces any cell type that can then be used to repair injured or aging tissues, organs and blood vessels. It consists of a nanotechnology-based chip that is applied to the skin, which is then struck with a short electric pulse to deliver genetic instructions into the cells of the tissue.

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"These are genes that induce tissue plasticity allowing the flexibility to direct the fate," Chandan Sen, first author of the paper, explains to New Atlas. "Thus, for example, skin cells can be directed to form blood vessels, or neural cells, or some other cell of interest."

We have seen a number of promising approaches to reprogramming cells for various regenerative health purposes. In 2012, a Japanese researcher won a Nobel Prize for his discovery that skin cells from mice could be harvested and converted into stem cells in the lab, work that has inspired a number of exciting breakthroughs since.

But according to Sen, one of the main advantages his tissue nanotransfection technology holds over other approaches is the fact that the cell conversion takes place in the body. This avoids the thorny issue of immune response, in which the host cells react to the newcomers and possibly attack them, something that can cause a raft of complications.

"Ours is reprogramming of not just cells but tissue within the live body under immune surveillance," he tells us. "Our strategy must co-operate with physiological factors to achieve the end goal."

That end goal is still a while away, but his team is making promising progress all the same. It put the technology to the test on animals, and in one experiment involving mice with badly injured legs lacking blood flow, it was able to convert skin cells into vascular cells. Within about a week, the legs featured active blood vessels. By the second week they were saved.

In a separate experiment, the team was also able to use the technology to reprogram skin cells into nerve cells, which were then injected into brain-injured mice to assist with stroke recovery.

"This is difficult to imagine, but it is achievable, successfully working about 98 percent of the time," said Sen. "With this technology, we can convert skin cells into elements of any organ with just one touch. This process only takes less than a second and is non-invasive, and then you're off. The chip does not stay with you, and the reprogramming of the cell starts. Our technology keeps the cells in the body under immune surveillance, so immune suppression is not necessary."

The team hopes to move onto clinical trials some time next year, but Sen tells us they must first test the technology on larger animals and design the device to work on humans.

You can hear from Sen in the video below, while the research was published in the journal Nature Nanotechnology.

Source: Ohio State University

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Chip reprograms skin cells with a short electric pulse - New Atlas - New Atlas

A University of Chicago-based research team has overcome challenges that have limited gene therapy and demonstrated how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat many human diseases.

In a study inthe journal Cell Stem Cell, the researchers provide proof-of-concept. They describe gene-therapy administered through skin transplants to treat two related and extremely common human ailments: Type 2 diabetes and obesity.

We resolved some technical hurdles and designed a mouse-to-mouse skin transplantation model in animals with intact immune systems, said study author Xiaoyang Wu, assistant professor in the Ben May Department for Cancer Research at the University of Chicago. We think this platform has the potential to lead to safe and durable gene therapy in mice and, we hope, in humans, using selected and modified cells from skin.

Beginning in the 1970s, physicians learned how to harvest skin stem cells from a patient with extensive burn wounds, grow them in the laboratory, then apply the lab-grown tissue to close and protect a patients wounds. This approach is now standard. However, the application of skin transplants is better developed in humans than in mice.

The mouse system is less mature, Wu said. It took us a few years to optimize our 3-D skin organoid culture system.

This study is the first to show that an engineered skin graft can survive long term in wild-type mice with intact immune systems. We have a better than 80 percent success rate with skin transplantation, Wu said. This is exciting for us.

The researchers focused on diabetes because it is a common non-skin disease that can be treated by the strategic delivery of specific proteins.

They inserted the gene for glucagon-like peptide 1 (GLP1), a hormone that stimulates the pancreas to secrete insulin. This extra insulin removes excessive glucose from the bloodstream, preventing the complications of diabetes. GLP1 can also delay gastric emptying and reduce appetite.

Using CRISPR, a tool for precise genetic engineering, they modified the GLP1 gene. They inserted one mutation, designed to extend the hormones half-life in the blood stream, and fused the modified gene to an antibody fragment so that it would circulate in the blood stream longer. They also attached an inducible promoter, which enabled them to turn on the gene to make more GLP1, as needed, by exposing it to the antibiotic doxycycline. Then they inserted the gene into skin cells and grew those cells in culture.

When these cultured cells were exposed to an air/liquid interface in the laboratory, they stratified, generating what the authors referred to as a multi-layered, skin-like organoid. Next, they grafted this lab-grown gene-altered skin onto mice with intact immune systems. There was no significant rejection of the transplanted skin grafts.

When the mice ate food containing minute amounts of doxycycline, they released dose-dependent levels of GLP1 into the blood. This promptly increased blood-insulin levels and reduced blood-glucose levels.

When the researchers fed normal or gene-altered mice a high-fat diet, both groups rapidly gained weight. They became obese. When normal and gene-altered mice got the high-fat diet along with varying levels of doxycycline, to induce GLP1 release, the normal mice grew fat and mice expressing GLP1 showed less weight gain.

Expression of GLP1 also lowered glucose levels and reduced insulin resistance.

Together, our data strongly suggest that cutaneous gene therapy with inducible expression of GLP1 can be used for the treatment and prevention of diet-induced obesity and pathologies, the authors wrote.

When they transplanted gene-altered human cells to mice with a limited immune system, they saw the same effect. These results, the authors wrote, suggest that cutaneous gene therapy for GLP1 secretion could be practical and clinically relevant.

This approach, combining precise genome editing in vitro with effective application of engineered cells in vivo, could provide significant benefits for the treatment of many human diseases, the authors note.

We think this can provide a long-term safe option for the treatment of many diseases, Wu said. It could be used to deliver therapeutic proteins, replacing missing proteins for people with a genetic defect, such as hemophilia. Or it could function as a metabolic sink, removing various toxins.

Skin progenitor cells have several unique advantages that are a perfect fit for gene therapy. Human skin is the largest and most accessible organ in the body. It is easy to monitor. Transplanted skin can be quickly removed if necessary. Skins cells rapidly proliferate in culture and can be easily transplanted. The procedure is safe, minimally invasive and inexpensive.

There is also a need. More than 100 million U.S. adults have either diabetes (30.3 million) or prediabetes (84.1 million), according the Centers for Disease Control and Prevention. More than two out of three adults are overweight. More than one out of three are considered obese.

Additional authors of the study were Japing Yue, Queen Gou, and Cynthia Li from the University of Chicago and Barton Wicksteed from the University of Illinois at Chicago. The National Institutes of Health, the American Cancer Society and the V Foundation funded the study.

Article originally appeared on Science Life.

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Gene therapy via skin could treat diseases such as obesity - UChicago News

News and research before you hear about it on CNBC and others. Claim your 2-week free trial to StreetInsider Premium here.

VistaGen Therapeutics Inc. (NASDAQ: VTGN), a clinical-stage biopharmaceutical company focused on developing new generation medicines for depression and other central nervous system (CNS) disorders, announced today that the Company has received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for U.S. Patent Application No. 14/359,517 regarding proprietary methods for producing hematopoietic precursor stem cells, which are stem cells that give rise to all of the blood cells and most of the bone marrow cells in the body, with potential to impact both direct and supportive therapy for autoimmune disorders and cancer.

The breakthrough technology covered by the allowed U.S. patent was discovered and developed by distinguished stem cell researcher, Dr. Gordon Keller, Director of the UHN's McEwen Centre for Regenerative Medicine in Toronto, one of the world's leading centers for stem cell and regenerative medicine research and part of the University Health Network (UHN), Canada's largest research hospital. Dr. Keller is a co-founder of VistaGen and a member of the Company's Scientific Advisory Board. VistaGen holds an exclusive worldwide license from UHN to the stem cell technology covered by the allowed U.S. patent.

"We are pleased to report that the USPTO has allowed another important U.S. patent relating to our stem cell technology platform, stated Shawn Singh, Chief Executive Officer of VistaGen. "Because the technology under this allowed patent involves the stem cells from which all blood cells are derived, it has the potential to reach the lives of millions battling a broad range of life-threatening medical conditions, including cancer, with CAR-T cell applications and foundational technology we believe ultimately will provide approaches for producing bone marrow stem cells for bone marrow transfusions. As we continue to expand the patent portfolio of VistaStem Therapeutics, our stem cell technology-focused subsidiary, we enhance our potential opportunities for additional regenerative medicine transactions similar to our December 2016 sublicense of cardiac stem cell technology to BlueRock Therapeutics, while focusing VistaStem's internal efforts on using stem cell technology for cost-efficient small molecule drug rescue to expand our drug development pipeline."

See the rest here:
VistaGen Therapeutics (VTGN) Receives Notice of Allowance For Methods for Producing Blood Cells, Platelets and ... - StreetInsider.com

August 8, 2017 by Samuel Hammond

The Wall Street Journal editorial board reported yesterday that the Health Resources and Service Administration (HRSA) regulation which sought to ban compensation for blood-forming stem cell donors has been defeated. This represents a small but significant victory for advocates of compensating organ donors a practice that remains outlawed by the National Organ Transplant Act (NOTA).

The crux of HRSAs rulemaking was a move to redefine blood-forming stem cells drawn from the bloodstream as an organ, no different from the bone marrow found within the bone, and thus under NOTAs purview. Our friends at the Institute for Justice (IJ) rightly argued for years that such a move was nonsensical and illegal. Blood and plasma are explicitly exempt from NOTAs ban on donor compensation, and as such donations of some subpart of the blood, including stem cells, should also be exempt.

The battle to kill the then-pending regulation heated up late last year, as HRSA neared its deadline to finalize the rule. The Niskanen Center formally joined IJs efforts in November, when we released a report called Bone Marrow Mismatch: How compensating bone marrow donors can end the transplant shortage and save lives. The report highlighted the enormous gap between bone marrow demand and supply under the current regime of voluntary donation, and argued against the applicability of the core ethical concerns advanced by HRSA. Our research and Hill event on the issue culminated in a listening session with HRSA officials, in which we argued that the social cost of enacting the rule was well in excess of $100 million, and thus worthy of delay for a deeper cost-benefit appraisal.

Its unclear what happened next. HRSAs hard December 18 deadline came and went, with a final rule that appeared to have been written but not formally submitted to the Federal Register. Perhaps it was the incoming administration, or the threat of litigation should the rule go through, or our research which provided a clear rationale for postponement. Regardless, the rule entered a strange purgatory, which is where it stayed until HHS formally withdrew the rule last week.

The Niskanen Center has received communications from a federal employee who believes our research was to some degree responsible for the rules ultimate repeal. That said, my research was simply part of a multi-pronged and multi-year effort to oppose the rule, led early on byIJ, the entrepreneur Doug Grant, the economist Mario Macis, and Peter Jaworski, the business ethicist and creator of DonationEthics.com.

The view of the Niskanen Center is that economic rights include the right to receive compensation for organ donations. NOTA therefore deserves a much deeper legal challenge. But in the meantime, lets celebrate the defeat of this regulation as a clear example of what it means to make small steps toward a better world.

Link:
Compensating Bone Marrow Donors Will Close the Supply Gap and Save Lives. - Niskanen Center (press release) (blog)

Updated: August 9, 2017 12:44 pm

Every evening when Aadya watches children in the neighborhood play, my heart breaks. My daughter too was once an energetic presence rushing about. I know that Aadya longs to join them.

It started after Aadyas second birthday. She got high fever and rashes all over. The local doctor called it skin allergy and prescribed medicines. The fever persisted, and we sought another medical opinion.

The diagnosis was devastating. B Cell Acute Lymphoblastic Leukaemia a cancer that affects the immune system. Our only hope now lies in the contributions of caring strangers through ketto.org.

B Cell Acute Lymphoblastic Leukaemia. Big sounding medical terms that we knew nothing about, but by the look on the doctors face, clearly it was serious.

Acute lymphoblastic leukemia affects, breaks down the bodys ability to fight diseases. The cancer starts in the bone marrow, where new blood cells grow. These cells grow very fast and the bone marrows capacity to make normal cells is reduced.

The doctor said Aadya needed treatment immediately, or else the cancer would spread. From March to December 2016, she was under the care of Dr. Shweta Bansal at the Sir H.N.Reliance Foundation Hospital and Research Centre in Mumbai. Ten months is a long time for a grown person. To watch our only child suffer through so many blood tests and chemotherapy treatments was very painful and difficult.

After ten months, the treatment ended and we were full of hope that Aadya would begin to recover. Then just four months later, in April 2017, we got the terrible news that the leukemia had relapsed. Since then, Aadya has been visiting the hospital for chemotherapy and tests, every 15-30 days.

Today Aadya is three years old and it hurts us to see her childhood being taken away. She barely eats, feels tired and weak all the time, and gets bruised easily. Even the slightest exposure to infection can be dangerous so we mostly keep her at home. She has missed many days of school and plays indoors. Any exposure to dust is dangerous so we have to make sure that her clothes, food, and toys are kept dust-free at all times.

Aadyas hope is a Bone Marrow Transplant, which costs a staggering Rs 25 lakh. We have started a fundraising page with ketto.org, counting on peoples sense of humanity to help us with this life-saving surgery.

So far we have spent Rs 15 lakh on Aadyas chemotherapy treatments, medications, and hospital visits. I am a housewife and my husband earns Rs 25,000 working as a back office employee. We are completely dependent on his salary and had to raise the money for the treatment by taking loans, borrowing from family members and friends and through insurance. All that we have managed to raise until now has been used up in the treatment.

A Bone Marrow Transplant surgery will replace Aadyas damaged bone marrow with healthy bone marrow stem cells, enabling her to lead a normal, healthy life. Her father is a matching and willing donor but we need to put together Rs 25 lakh in the next one month. We have no means of raising that kind of money.

For Aadya to survive, that operation has to be done in one months time. For over a year now, Aadya has been fighting a tough, long battle. Now there is hope that this operation will finally end her nightmare and lead to that one final miracle when we can take our baby home.

We have started a fundraising page with Ketto.org in Aadyas name, in the hope that people will come forward and help us raise the funds for this surgery.

Please help us pay for her BMT by logging on to Ketto.org.

Help us to bring Aadya home.

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Help my only child survive! - The Indian Express

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."

Excerpt from:
Wild new microchip tech could grow brain cells on your skin - CNET

The chip has not been tested in humans, but it has been used to heal wounds in mice. Wexner Medical Center/The Ohio State University hide caption

The chip has not been tested in humans, but it has been used to heal wounds in mice.

Scientists have created an electronic wafer that reprogrammed damaged skin cells on a mouse's leg to grow new blood vessels and help a wound heal.

One day, creator Chandan Sen hopes, it could be used to be used to treat wounds on humans. But that day is a long way off as are many other regeneration technologies in the works. Like Sen, some scientists have begun trying to directly reprogram one cell type into another for healing, while others are attempting to build organs or tissues from stem cells and organ-shaped scaffolding.

But other scientists have greeted Sen's mouse experiment, published in Nature Nanotechnology on Monday, with extreme skepticism. "My impression is that there's a lot of hyperbole here," says Sean Morrison, a stem cell researcher at the University of Texas Southwestern Medical Center. "The idea you can [reprogram] a limited number of cells in the skin and improve blood flow to an entire limb I think it's a pretty fantastic claim. I find it hard to believe."

When the device is placed on live skin and activated, it sends a small electrical pulse onto the skin cells' membrane, which opens a tiny window on the cell surface. "It's about 2 percent of the cell membrane," says Sen, who is a researcher in regenerative medicine at Ohio State University. Then, using a microscopic chute, the chip shoots new genetic code through that window and into the cell where it can begin reprogramming the cell for a new fate.

Sen says the whole process takes less than 0.1 seconds and can reprogram the cells resting underneath the device, which is about the size of a big toenail. The best part is that it's able to successfully deliver its genetic payload almost 100 percent of the time, he says. "No other gene delivery technique can deliver over 98 percent efficiency. That is our triumph."

Chandan Sen, a researcher at Ohio State University, holds a chip his lab created that has reprogrammed cells in mice. Wexner Medical Center/The Ohio State University hide caption

Chandan Sen, a researcher at Ohio State University, holds a chip his lab created that has reprogrammed cells in mice.

To test the device's healing capabilities, Sen and his colleagues took a few mice with damaged leg arteries and placed the chip on the skin near the damaged artery. That reprogrammed a centimeter or two of skin to turn into blood vessel cells. Sen says the cells that received the reprogramming genes actually started replicating the reprogramming code that the researchers originally inserted in the chip, repackaging it and sending it out to other nearby cells. And that initiated the growth of a new network of blood vessels in the leg that replaced the function of the original, damaged artery, the researchers say. "Not only did we make new cells, but those cells reorganized to make functional blood vessels that plumb with the existing vasculature and carry blood," Sen says. That was enough for the leg to fully recover. Injured mice that didn't get the chip never healed.

When the researchers used the chip on healthy legs, no new blood vessels formed. Sen says because injured mouse legs were was able to incorporate the chip's reprogramming code into the ongoing attempt to heal.

That idea hasn't quite been accepted by other researchers, however. "It's just a hand waving argument," Morrison says. "It could be true, but there's no evidence that reprogramming works differently in an injured tissue versus a non-injured tissue."

What's more, the role of exosomes, the vesicles that supposedly transmit the reprogramming command to other cells, has been contentious in medical science. "There are all manners of claims of these vesicles. It's not clear what these things are, and if it's a real biological process or if it's debris," Morrison says. "In my lab, we would want to do a lot more characterization of these exosomes before we make any claims like this."

Sen says that the theory that introduced reprogramming code from the chip or any other gene delivery method does need more work, but he isn't deterred by the criticism. "This clearly is a new conceptual development, and skepticism is understandable," he says. But he is steadfast in his confidence about the role of reprogrammed exosomes. When the researchers extracted the vesicles and injected them into skin cells in the lab, Sen says those cells converted into blood vessel cells in the petri dish. "I believe this is definitive evidence supporting that [these exosomes] may induce cell conversion."

Even if the device works as well as Sen and his colleagues hope it does, they only tested it on mice. Repairing deeper injuries, like vital organ damage, would also require inserting the chip into the body to reach the wound site. It has a long way to go before it can ever be considered for use on humans. Right now, scientists can only directly reprogram adult cells into a limited selection of other cell types like muscle, neurons and blood vessel cells. It'll be many years before scientists understand how to reprogram one cell type to become part of any of our other, many tissues.

Still, Morrison says the chip is an interesting bit of technology. "It's a cool idea, being able to release [genetic code] through nano channels," he says. "There may be applications where that's advantageous in some way in the future."

See the original post here:
A Chip That Reprograms Cells Helps Healing, At Least In Mice - NPR

A med-tech startup has developed a fast and easy way to treat certain burn wounds with stem cells. This technology is developed by German researcher Dr. Jrg Gerlach. He is the worlds first ever person who use a patients stem cells to directly heal the skin. The technique is meant to reduce the healing time and minimize complications, with aesthetically and functionally satisfying outcomes. There are no scars, no residual pain and its like there wasnt any burn to start with. Its not less than a miracle.

The medical technology startup has now transformed the proof-of-concept device from a complicated prototype into a user-friendly product called a SkinGun, which it hopes doctors will be able to use outside of an experimental setting. RenovaCare CEO Thomas Bold believes, the SkinGun can compete with, or even replace, todays standard of care. The sprayer allows us to have a generous distribution of cells on the wound, explained Roger Esteban-Vives, director of cell sciences at RenovaCare.

RenovaCares SkinGun sprays a liquid suspension of a patients stem cells onto a burn or wound in order to re-grow the skin without scars. Stem-cell methods helped cut this risk by quickening healing and providing a source of new skin from a very small area. Cell Mist method gets a greater yield from its harvest than mesh grafting, a more common way to treat burns. At a maximum, grafting can treat six times the size of its harvest area. Cell Mist can cover 100 times its harvest area.

When dispensing cells over a wound, its important that they make the transition without any damage. Damaged cells reduce the effectiveness of the treatment.

High cell viability also contributes to faster healing. When a wound heals naturally, cells migrate to it to build up the skin. That process can take weeks.

Stem cells have tremendous promise to help us understand and treat a range of diseases, injuries and other health-related conditions.

There is still a lot to learn about stem cells, however, and their current applications as treatments are sometimes exaggerated by the media and other parties who do not fully understand the science and current limitations

Beyond regulatory matters, there are also limitations to the technology that make it unsuitable for competing with treatments of third-degree burns, which involve damage to muscle and other tissue below the skin.

When burn victims need a skin graft they typically have to grow skin on other parts of their bodies. This is a process that can take weeks. A new technique uses stem cells derived from the umbilical cord to generate new skin much more quickly.The umbilical cord consists of a gelatinous tissue that contains uncommitted mesenchymal stemcells (MSC)

Research is underway to develop various sources for stem cells, and to apply stem-cell treatments for neurodegenertive diseasesand conditions such as diabetes, heart disease, and other conditions.

Tens of thousands of grafts are performed each year for burn victims, cosmetic surgery patients, and for people with large wounds having difficulty healing. Traditionally, this involves taking a large patch of skin (typically from the thigh) and removing the dermis and epidermis to transplant elsewhere on the body. Burns victims are making incredible recoveries thanks to a revolutionary gun that sprays stem cells on to their wounds, enabling them to rapidly grow new skin. Patients who have benefited say their new skin is virtually indistinguishable from that on the rest of the body.

Thomas Bold, chief executive of RenovaCare, the company behind SkinGun, said: The procedure is gentler and the skin that regrows looks, feels and functions like the original skin.

If you are planning to have stem cell treatments dont forget to remember these points

Stem cell researchers are making great advances in understanding normal development. They are trying to figure out what goes wrong in disease and developing and testing potential treatments to help patients. They still have much to learn. However, about how stem cells work in the body and their capacity for healing. Safe and effective treatments for most diseases, conditions and injuries are in the future.

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Dramatic Burn-Healing Through Stem Cell Treatment - Fox Weekly

Theoretically, eternal youth is now within our grasp.

Doctors are close to discovering a real life fountain of youth that could theoretically enable patients to live forever. Advances in stem cell treatments and now, tissue nanotransfection (TNT), which is a new technique, can theoretically provide patients with the benefits of youth for life.

The fountain of youth is within the grasp of science, but so far, only for mice. Human trials come next year.

LOS ANGELES, CA (California Network) -- The quest for eternal life is ancient. It is mentioned in the first and oldest story we have, the Epic of Gilgamesh. In that ancient Sumerian tale, only Utnapishtim, a man who built and ark and survived a great flood in a story that is almost identical to the story of Noah's ark, knows the secret to eternal life, which ultimately proves elusive. In the centuries that followed, people have tried every remedy imaginable to prolong life. They searched for the fabled fountain of youth, and according to some legends, bathed in the blood of virgins and children.

Today, we know none of these endeavors would work because ageing is carried on in the genes. The only way to reverse ageing is to manipulate the genes. And this is precisely what doctors are looking to do in order to produce new cells, and even whole organs.

Researchers now know the primary difference between a young person and an old person is the number of stem cells in their body. Young people have many times more stem cells. This is the basic, underlying reason why young people are so youthful. A young body can repair itself more rapidly and thoroughly than an older one because of the number of stem cells. But if stem cells could be injected into an older body, in quantities similar to those enjoyed by a young person, what would happen then?

Nobody knows for certain because the experiment hasn't been conducted, but the hypothesis is that the older person would become more youthful, healthier, and longer lived.

As stem cells enter the medical mainstream, and may become a standard part of medical treatment in the near future, there is another development that could make stem cells irrelevant. Nanotransfection, abbreviated as TNT, is a new method whereby skin cells can be turned into any other cell in the body using a special microchip and electricity.

The device, called a nanochip, is loaded with genetic material essential to turning cells into other kinds of cells. The electrical current enables the device to inject the genetic material into the skin where it ends up inside the cells. These cells can then travel though the body and take on the properties of healthy cells around damaged tissue, facilitating repair. On other words, a damaged liver or heart can be repaired with this tiny device. The advantage of this method is that stem cells are not required. Your skin cells simply become whether other kind of cells they are told to become by the injected genetic material.

A study affirming the effectiveness of this approach was published in the journal, Nature Nanotechnology. It has been tested on mice and was successful in restoring function to non-functioning limbs. It will be tested on humans within the next year.

Scientists have known they can reprogram cells into other kinds of cells for a long time now, but only recently have they developed the method to do so cheaply and efficiently. The actual procedure requires a chip that is as small as a penny, and takes only a second to work.

If the procedure works on humans, then doctors may have a cheap and efficient way to repair and even replace organs. The discovery is so dramatic is it difficult to believe. More testing is required, but it shows just how far we have come in our ability to edit genes and reprogram cells to grow specific forms of tissue within the body.

In a generation or less, it is reasonable that we will have unlocked the secret to reversing ageing. Of course, this discovery opens a whole host of ethical and philosophical questions, but that's for the ethicists and politicians to work out. For now, science is about to take the first sip from the fountain of youth, and we await the result.

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Pope Francis Prayer Intentions for JULY 2017Lapsed Christians. That our brothers and sisters who have strayed from the faith, through our prayer and witness to the Gospel, may rediscover the merciful closeness of the Lord and the beauty of the Christian life.

Excerpt from:
Stem cells: science prepares to take the first sip from the real fountain of youth - Catholic Online

The PASE, or post-implantation amniotic sac embryoid, is a structure grown from human pluripotent stem cells that mimics many of the properties of the amniotic sac that forms soon after an embryo implants in the uterus wall. The structures could be used to study infertility. Credit: University of Michigan

The first few weeks after sperm meets egg still hold many mysteries. Among them: what causes the process to fail, leading to many cases of infertility.

Despite the importance of this critical stage, scientists haven't had a good way to explore what can go wrong, or even what must go right, after the newly formed ball of cells implants in the wall of the human uterus.

But a new achievement using human stem cells may help change that. Tiny lab-grown structures could give researchers a chance to see what they couldn't before, while avoiding ethical issues associated with studying actual embryos.

A team from the University of Michigan reports in Nature Communications that they have coaxed pluripotent human stem cells to grow on a specially engineered surface into structures that resemble an early aspect of human development called the amniotic sac.

The cells spontaneously developed some of the same structural and molecular features seen in a natural amniotic sac, which is an asymmetric, hollow ball-like structure containing cells that will give rise to a part of the placenta as well as the embryo itself. But the structures grown at U-M lack other key components of the early embryo, so they can't develop into a fetus.

It's the first time a team has grown such a structure starting with stem cells, rather than coaxing a donated embryo to grow, as a few other teams have done.

"As many as half of all pregnancies end in the first two weeks after fertilization, often before the woman is even aware she is pregnant. For some couples, there is a chronic inability to get past these critical early developmental steps, but we have not previously had a model that would allow us to explore the reasons why," says co-senior author Deborah Gumucio, Ph.D. "We hope this work will make it possible for many scientists to dig deeper into the pathways involved in normal and abnormal development, so we can understand some of the most fascinating biology on earth." Gumucio is the Engel Collegiate Professor of Cell & Developmental Biology at Michigan Medicine, U-M's academic medical center.

A steady PASE

The researchers have dubbed the new structure a post-implantation amniotic sac embryoid, or PASE. They describe how a PASE develops as a hollow spherical structure with two distinct halves that remain stable even as cells divide.

One half is made of cells that will become amniotic ectoderm, the other half consists of pluripotent epiblast cells that in nature make up the embryonic disc. The hollow center resembles the amniotic cavity - which in normal development eventually gives rise to the fluid-filled sac that protects and cushions the fetus during development.

Gumucio likens a PASE to a mismatched plastic Easter egg or a blue-and-red Pokmon ball - with two clearly divided halves of two kinds of cells that maintain a stable form around a hollow center.

The team also reports details about the genes that became activated during the development of a PASE, and the signals that the cells in a PASE send to one another and to neighboring tissues. They show that a stable two-halved PASE structure relies on a signaling pathway called BMP-SMAD that's known to be critical to embryo development.

Gumucio notes that the PASE structures even exhibit the earliest signs of initiating a "primitive streak", although it did not fully develop. In a human embryo, the streak would start a process called gastrulation. That's the division of new cells into three cell layersendoderm, mesoderm and ectodermthat are essential to give rise to all organs and tissues in the body.

Collaboration provides the spark

The new study follows directly from previous collaborative work between Gumucio's lab and that of the other senior author, U-M mechanical engineering associate professor Jianping Fu, Ph.D.

In the previous work, reported in Nature Materials, the team succeeded in getting balls of stem cells to implant in a special surface engineered in Fu's lab to resemble a simplified uterine wall. They showed that once the cells attached themselves to this substrate, they began to differentiate into hollow cysts composed entirely of amnion - a tough extraembryonic tissue that holds the amniotic fluid.

But further analysis of these cysts by co-first authors of the new paper Yue Shao, Ph.D., a graduate student in Fu's lab, and Ken Taniguchi, a postdoctoral fellow in Gumucio's lab, revealed that a small subset of these cysts were stably asymmetric and looked exactly like early human or monkey amniotic sacs.

The team found that such structures could also grow from induced pluripotent stem cells (iPSCs)cells derived from human skin and grown in the lab under conditions that give them the ability to become any type of cell, similar to how embryonic stem cells behave. This opens the door for future work using skin cells donated by couples experiencing chronic infertility, which could be grown into iPSCs and tested for their ability to form proper amniotic sacs using the methods devised by the team.

Important notes and next steps

Besides working with genetic and infertility specialists to delve deeper into PASE biology as it relates to human infertility, the team is hoping to explore additional characteristics of amnion tissue.

For example, early rupture of the amnion tissue can endanger a fetus or be the cause of a miscarriage. The team also intends to study which aspects of human amnion formation also occur in development of mouse amnion. The mouse embryo model is very attractive as an in vivo model for investigating human genetic diseases.

The team's work is overseen by a panel that monitors all work done with pluripotent stem cells at U-M, and the studies are performed in accordance with laws regarding human stem cell research. The team ends experiments before the balls of cells effectively reach 14 developmental days, the cutoff used as an international limit on embryo researcheven though the work involves tissue that cannot form an embryo. Some of the stem cell lines were derived at U-M's privately funded MStem Cell Laboratory for human embryonic stem cells, and the U-M Pluripotent Stem Cell Core.

Explore further: Team uses stem cells to study earliest stages of amniotic sac formation

More information: Yue Shao et al, A pluripotent stem cell-based model for post-implantation human amniotic sac development, Nature Communications (2017). DOI: 10.1038/s41467-017-00236-w

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Amniotic sac in a dish: Stem cells form structures that may aid of ... - Phys.Org

For patients with severe and end-stage heart failure there are few treatment options left apart from transplants and stem-cell therapy. But a new Israeli study finds that stem-cell therapy may harm heart-disease patients.

The research, led by Prof. Jonathan Leor of Tel Aviv Universitys Sackler Faculty of Medicineand Sheba Medical Center and conducted by TAUs Dr. Nili Naftali-Shani, explores the current practice of using cells from the host patient to repair tissue and contends that this can prove toxic for patients.

We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury, said Leor. Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle.

Tissue or adult stem cells blank cells that can act as a repair kit for the body by replacing damaged tissue encourage the regeneration of blood vessel cells and new heart muscle tissue. Faced with a worse survival rate than many cancers, many heart-failure patients have turned to stem-cell therapy as a last resort.

But our findings suggest that stem cells, like any drug, can have adverse effects, said Leor. We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient.

The researchers, who published their study in the journal Circulation, also discovered the molecular pathway involved in the negative interaction between stem cells and the immune system as they isolated stem cells in mouse models of heart disease. Afterward, they focused on cardiac stem cells in patients with heart disease.

The results could help improve the use of autologous stem cells those drawn from the patients themselves in cardiac therapy, Leor said.

We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells, said Leor. Our findings determine the potential negative effects of inflammation on stem-cell function as theyre currently used. The use of autologous stem cells from patients with heart disease should be modified. Only stem cells from healthy donors or genetically engineered cells should be used in treating cardiac conditions.

The researchers are currently testing a gene editing technique (CRISPER) to inhibit the gene responsible for the negative inflammatory properties of the cardiac stem cells of heart disease patients. We hope our engineered stem cells will be resistant to the negative effects of the immune system, said Leor.

Excerpt from:
Stem-cell treatment may harm heart disease patients - ISRAEL21c

Earlier this year, Texas Heart Institute received Alpha Phi Foundations 2017 Heart to Heart Grant. The $100,000 grant will fund research led by Doris Taylor, Ph.D., director of the Regenerative Medicine Research and the Center for Cell and Organ Biotechnology at the Texas Heart Institute, to study cardiac repair in women at the cellular level.

Were just really passionate about these projects that have long-term clinical relevancy, as a women-driven organization and being committed to womens heart health, said Colleen Sirhal, vice chair of the Alpha Phi Foundation.

The study will explore sex differences in blood, bone marrow and stem cells of patients enrolled in cell therapy clinical trials.

While bone marrow cell therapy has been used to treat cardiovascular disease in clinical trials, very few studies have been conducted to assess the sex differences in efficacy and outcomes. By performing a proteomic analysis of the samples and evaluating the proteins that cells produce and secrete, the results could shed light on unanswered questions related to critical sex-specific differences in cardiovascular disease, potentially leading to improved cell therapies.

Its about time that were paying attention to sex differences, Taylor said. Were not just small men. The biology is different.

Heart disease remains the No. 1 cause of death in both men and women in the United States, yet theres a limited understanding in the scientific community as to why it affects men and women differently. For example, women 45 years old and younger have a higher likelihood than men of dying within a year of their initial heart attack.

In addition, women have a higher risk of developing small vessel disease, in which the walls of tiny vessels within the heart muscle become blocked rather than larger arteries, causing heart-related chest pain. Because the major coronary arteries may look normal, women with small vessel disease can have a heart attack go undiagnosed and untreated.

We know heart disease happens differently in men and women, Taylor said. More young women than men die of heart disease. Why is that? Is there something that happens early? If we only look at these women who are older, are we missing something major? By looking at healthy, normal younger women, were going to be able to do comparisons across time, comparisons by disease, and comparisons by sex. I think thats really exciting.

Historically, women and minorities have largely been underrepresented in research and clinical trials, especially pertaining to cardiovascular disease.

Dr. Taylors colleague at the Texas Heart Institute, Stephanie Coulter, M.D., a cardiologist and the director of the Center for Womens Heart and Vascular Health at Texas Heart Institute and a recipient of the 2013 Heart to Heart Grant, is actively recruiting younger women to participate in her research registry.

Since women are typically affected by heart disease a decade or more later than men, age may also have played a role in this underrepresentation, Coulter said. Our Womens Center research is focusing on women age 18 and older to address this very issue.

Coulter added that trials focusing on prevention in women, such as the Womens Health Initiative and Womens Health Study, have, in fact, had clinical impact. However, the percentage of women enrolling in clinical trials continues to be disproportionate to the prevalence of cardiovascular disease in women, but we are seeing improvements thanks to multiple initiatives in the U.S. that continue to address the issue of women in clinical trials.

Its easy for people to assume that if you study men, itll apply to women, but it seems anathema to people to assume that if you study women it might benefit men, Taylor said. At the end of the day, when it comes time to look at the data and ask, How does this treatment work in women? How does this treatment work in men?, oftentimes there arent enough women enrolled in the trials to split that out. Statistically, youd be doing yourself a disservice.

Taylor has spent nearly two decades studying key contributors to cardiac repair at the cellular level, specifically looking at proteins cells produce and secrete based on gender as a new frontier in cell therapy.

Early on in Taylors career, she studied how bone marrow cells behaved based on gender. She extracted cells from male mice and administered them to female mice and vice versa, allowing her to track the Y chromosome. The results showed that only the males treated with female cells improved. This phenomenon raised the question of whether or not the bone marrow cells were the same.

After measuring the bone marrow cells that were present in males and females, Taylor discovered that the cells were inherently different: In the male mice, there were more inflammatory cells, fewer progenitor and stem cells and a different number of immune cells than in the female mice. In addition, when the bone marrow cells were placed in a petri dish, the female cells produced more growth factors responsible for recruiting repair cells after an injury.

Taylor conducted follow-up experiments in which she gave female and male cells to both female and male mice. The results confirmed her hunch: The only cells that were reparative were the female cells.

It made me realize a critical detail for the first time:Every time we take bone marrow from a different person with the intention of delivering it back to them as a therapy, if we look at the cells present in the marrow, theyd be different, Taylor said. Which means, every time were doing an autologous cell therapytrial, in which you take bone marrow and deliver it back to an individual, you are giving each person a completely different or unique drug in that trial.

Through the Heart to Heart grant, the data from Taylors research will allow her to build upon her early research on sex differences and, hopefully, identify a way to optimize cell therapy.

Already cells are as good as some drugs. If we optimize them and choose the right cells for the right patient at the right time, maybe well hit the home run, Taylor said.

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Texas Heart Institute Awarded Grant to Study Sex Differences in Cardiac Repair - Texas Medical Center (press release)

Daiichi Sankyo Company has signed an investment contract with Cuorips Inc., an Osaka University spin-off venture to receive an option right concerning the worldwide commercialization of iPS-derived cardiomyocyte (iPS-CM) sheet developed by Cuorips.

The iPS-CM sheet is an allogeneic cell therapy product consisting of cardiomyocyte derived from human iPS cells. Its transplantation is expected to provide improvement of cardiac function and amelioration of heart failure and become a new treatment option for patients with severe heart failure, who have no remedies other than heart transplantation or artificial heart implantation.

Based on the cutting-edge cell therapy research targeting heart diseases, the team at the Department of Cardiovascular Surgery, Osaka University Graduate School of Medicine, led by Professor Yoshiki Sawa, has been working on the iPS-CM research and development by participating in the Research Center Network for Realization of Regenerative Medicine, which is run by the Japan Agency for Medical Research and Development (AMED). They are currently preparing for clinical research as well as investigator initiated clinical study.

Cuorips is an Osaka University spin-off venture founded to develop and commercialize iPS-CM sheets based on the research data and technologies developed by the university.

Daiichi Sankyo Group has been conducting research on iPS cell-derived cardiomyocyte and their production, and is currently working on the efficient production process capable for commercial supply.

Daiichi Sankyo and Cuorips are aiming to commercialize iPS-CM sheets as a pioneering treatment for severe heart failure. iPS cells are capable of almost unlimited proliferation and differentiation into any organ, and are expected to be used in the field of cell therapy. There are two types of cell therapy: autologous therapy where the patients own cells are collected, cultured and processed, and allogeneic therapy where a donors cells are collected, cultured and processed.

Read more here:
Daiichi Sankyo signs Investment contract with Cuorips to commercialize iPS-derived cardiomyocyte sheet - pharmabiz.com

A novel device that reprogrammes skin cells could represent a breakthrough in repairing injured or ageing tissue, researchers say.

The new technique, called tissue nanotransfection, is based on a tiny device that sits on the surface of the skin of a living body. An intense, focused electric field is then applied across the device, allowing it to deliver genes to the skin cells beneath it turning them into different types of cells.

That, according to the researchers, offers an exciting development when it comes to repairing damaged tissue, offering the possibility of turning a patients own tissue into a bioreactor to produce cells to either repair nearby tissues, or for use at another site.

By using our novel nanochip technology, injured or compromised organs can be replaced, said Chandan Sen, from the Ohio State University, who co-led the study. We have shown that skin is a fertile land where we can grow the elements of any organ that is declining.

The ability for scientists to reprogram cells into other cell types is not new: the discovery scooped John Gurdon and Shinya Yamanaka the Nobel Prize in 2012 and is currently under research in myriad fields, including Parkinsons disease.

You can change the fate of cells by incorporating into them some new genes, said Dr Axel Behrens, an expert in stem cell research from the Francis Crick Institute in London, who was not involved in the Ohio research. Basically you can take a skin cell and put some genes into them, and they become another cell, for example a neuron, or a vascular cell, or a stem cell.

But the new approach, says Sen, avoids an intermediary step where cells are turned into what are known as pluripotent stem cells, instead turning skin cells directly into functional cells of different types. It is a single step process in the body, he said.

Furthermore, the new approach does not rely on applying an electric field across a large area of the cell, or the use of viruses to deliver the genes. We are the first to be able to reprogramme [cells] in the body without the use of any viral vector, said Sen.

The new research, published in the journal Nature Nanotechnology, describes how the team developed both the new technique and novel genes, allowing them to reprogramme skin cells on the surface of an animal in situ.

They can put this little device on one piece of skin or onto the other piece of skin and the genes will go there, wherever they put [the device], said Behrens.

The team reveal that they used the technique on mice with legs that had had their arteries cut, preventing blood flow through the limb. The device was then put on the skin of the mice, and an electric field applied to trigger changes in the cells membrane, allowing the genes to enter the cells below. As a result, the team found that they were able to convert skin cells directly into vascular cells -with the effect extending deeper into the limb, in effect building a new network of blood vessels.

Seven days later we saw new vessels and 14 days later we saw [blood flow] through the whole leg, said Sen.

The team were also able to use the device to convert skin cells on mice, into nerve cells which were then injected into the brains of mice who had experienced a stroke, helping them to recover.

With this technology, we can convert skin cells into elements of any organ with just one touch. This process only takes less than a second and is non-invasive, and then youre off, said Sen.

The new technology, said Behrens is an interesting step, not least since it avoids all issues with rejection.

This is a clever use of an existing technique that has potential applications but massive further refinement is needed, he said, pointing out that there are standard surgical techniques to deal with blockages of blood flow in limbs.

Whats more, he said, the new technique is unlikely to be used on areas other than skin, since the need for an electric current and the device near to the tissue means using it on internal organs would require an invasive procedure.

Massive development [would be] needed for this to be used for anything else than skin, he said.

But Sen and colleagues say they are are hoping to develop the technique further, with plans to start clinical trials in humans next year.

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Nanochip could heal injuries or regrow organs with one touch, say researchers - The Guardian

The Potential of CRISPR

The potential of the gene editing toolCRISPRjust seems to keep growing and growing, and the latest experimental use of the technology is creating skin grafts that trigger the release of insulin and help manage diabetes.

Researchers have successfully tested the idea with mice that gained less weight and showed a reversed resistance to insulin because of the grafts (high insulin resistance is a common precursor to type 2 diabetes).

In fact, the team from the University of Chicago says the same approach could eventually be used to treat a variety of metabolic and genetic conditions, not just diabetes its a question of using skin cells to trigger different chemical reactions in the body.

We didnt cure diabetes, but it does provide a potential long-term and safe approach of using skin epidermal stem cells to help people with diabetes and obesity better maintain their glucose levels,says one of the researchers, Xiaoyang Wu.

If youre new to theCRISPR(Clustered Regularly Interspaced Short Palindromic Repeats) phenomenon, its a new and innovative way of editing specific genes in the body, using a biological copy and paste technique: it can doeverything fromcut out HIV virus DNA to slow thegrowth of cancer cells.

For this study, researchers used CRISPR to alter the gene responsible for encoding a hormone calledglucagon-like peptide-1(GLP-1), which triggers the release of insulin and then helps remove excess glucose from the blood.

Type 2 diabetescomes about due to a lack of insulin, also known as insulin resistance.

Using CRISPR, the GLP-1 gene could be tweaked to make its effects last longer than normal. The result was developed into skin grafts that were then applied to mice.

Around 80 percent of the grafts successfully released the edited hormone into the blood, regulating blood glucose levels over four months, as well as reversing insulin resistance and weight gain related to a high-fat diet.

Significantly, its the first time the skin graft approach has worked for mice not specially designed in the lab.

This paper is exciting for us because it is the first time we show engineered skin grafts can survive long term in wild-type mice, and we expect that in the near future this approach can be used as a safe option for the treatment of human patients,says Wu.

Human treatments will take time to develop but the good news is that scientists are today able to grow skin tissue very easily in the lab using stem cells, so that wont be an issue.

If we can make it safe, and patients are happy with the procedure, then the researchers say it could be extended to treat something likehaemophilia, where the body is unable to make blood clots properly.

Any kind of disease where the body is deficient in specific molecules could potentially be targeted by this new technique. And if it works with diabetes, it could be time to say goodbye to needles and insulin injections.

Other scientists who werent directly involved in the research, including Timothy Kieffer from the University of British Columbia in Canada, seem optimistic.

I do predict that gene and cell therapies will ultimately replace repeated injections for the treatment of chronic diseases, Kieffer told Rachel Baxter atNew Scientist.

The findings have been published inCell Stem Cell.

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CRISPR Skin Grafts Could Replace Insulin Shots For Diabetes - Futurism