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Upstate Opens Cord Blood Bank, Only the Second Public Cord Blood Bank in New York and One of Only 32 in the US – Newswise (press release)

Newswise Upstate Medical University opens the Upstate Cord Blood Bank, only the second public cord blood bank in New York and one of only 32 in the US.

The $15 million, 20,000 square foot facility features a state of the art processing laboratory and cryogenic storage containers that can store nearly 14,500 units of cord blood. The building is located on Upstates Community Campus, 4910 Broad Road in Syracuse, home to Upstates obstetric services. The cord blood bank opened Feb. 9.

The bank will collect, test, process, store and distribute umbilical cord blood donated by families throughout central and northern New York to be used by those in need of life-saving medical treatments and for medical research.

The bank is currently accepting cord blood donations from families who give birth at Upstates Community Campus. Cord blood donations will be accepted from families who give birth at Syracuse's Crouse Hospital and St. Josephs Hospital Health Center as early as summer 2017. Agreements with other area hospitals will be forthcoming.

Umbilical cord blood is blood that remains in the placenta and umbilical cord after childbirth. Cord blood that is not donated is discarded as medical waste. It is a rich source of hematopoietic stem cells that have the potential of being used in the treatment of dozens of diseases, like blood cancers and bone marrow diseases such as sickle cell anemia.

State Sen. John DeFrancisco played a key role in securing the $15 million funding for the building, enabling Syracuse to have one of only two public cord blood banks in New York and one of only 32 in the United States.

The Upstate Cord Blood Bank responds directly to the mission of our academic medical center, said Upstate President Danielle Laraque-Arena, MD, FAAP. It is a community resource that will improve the health of individuals here, throughout our region and beyond. Whether the cord blood is used for transplantation or research, we are providing hope of better health and new treatments.

Laraque-Arena said she was grateful for the support of New York State Sen. John DeFrancisco. I applaud Senator DeFranciscos efforts to move this project forward and to enable Upstate to be at the forefront of this impactful initiative, Laraque-Arena said.

The Upstate Cord Blood Bank is a project near and dear to my heart, said DeFrancisco. Its a project that has been a long time coming, and I am absolutely ecstatic that the public bank is now open and receiving donations of umbilical cord blood. I look forward to witnessing the many cures that will result from having the use of cord blood available right here in Upstate.

Upstates Robert Corona, DO, MBA, professor and chair of the Department of Pathology, said the Upstate Cord Blood Bank would put to good use what is often referred to as medical waste.The blood from the umbilical cord and placenta, cord blood, contains hematopoietic stem cells that have potential to treat many diseases including cancer, genetic disorders and blood disorders, he said. What was once medical waste becomes a source of life saving cells and a significant contribution to the field of regenerative medicine. Stem cells show great potential in treating all sorts of neurologic disorders including metabolic disorders, spastic cerebral palsy and autism. We are truly fortunate to have a new Cord Blood Bank in our community as a cutting-edge patient care and research resource.

Nicholas Greco, PhD, executive director and tissue bank director of the Upstate Cord Blood Bank, said the use of cord blood in treatment for various diseases has expanded. Historically, cord blood from public banks in transplantation has focused on the safety and use in regenerating dysfunctional or damaged bone marrow. But, within the last decade, family banks have focused on using cord blood- and cord tissue-derived stem cells to replace or regenerate human cells, tissue or organs, to restore or establish normal function (regenerative medicine). These emerging uses, extend patient options for treatment and cures.

SUNY Chancellor Nancy Zimpher applauded the opening of the Upstate Cord Blood Bank. New Yorks ongoing investment in medical research and education provides far reaching benefits for our students and faculty as well as communities not only in New York state but around the globe, Zimpher said. Congratulations to President Laraque-Arena and the entire SUNY Upstate community on the opening of this new facility, which is certain to advance research in a vital field of study, enhance patient care, and provide new educational opportunities for students.

Designation as a public cord blood bank The designation of Upstate Cord Blood Bank as a public blood bank is important in that there is no cost to donate and donated cord blood is available to anyone who needs it. Once donated, the cord blood will be stored in the bank and made available to transplant centers in the United States and throughout the world for patients needing life-saving transplants. The cord blood units will be listed initially on the Bone Marrow Donors Worldwide registry and on the Be The Match registry maintained by the National Marrow Donor Program, which maintains a large listing of cord blood units available for transplant. Those units that are not suitable for transplantation will be made available to researchers, both at Upstate Medical University and around the country.

The Upstate Cord Blood Bank will in the near future open a family cord blood bank that will collect, test, process, store, and distribute a babys umbilical cord blood only for use by families who have a need for future use. An initial fee and annual fee will be charged for collection, processing and storage of umbilical cord blood in the family bank.

The Upstate Cord Blood Bank will operate under strict guidelines and protocols, established by state and federal health organizations, including the state Health Department, the Food and Drug Administration (FDA), AABB and the Foundation for the Accreditation of Cellular Therapy (FACT).

As is currently under way at Crouse Hospital and St. Josephs Hospital Health Center, Upstate will work with regional hospitals to develop guidelines and agreements to enable mothers who deliver in these facilities the ability to donate their cord blood for free.

Upstate officials hope the cord blood bank will ultimately receive donations from 10,000 births a year, which would represent 50 percent of the approximately 20,000 births in Central New York annually.

How is cord blood donated Once a mother has delivered her baby and after the umbilical cord is clamped and cut as is done with all deliveries, a medical provider will insert a needle into the umbilical vein that is still attached to the placenta. The process, which takes less than 10 minutes, yields about 3 to 5 ounces of cord blood, which is then sent to the Upstate Cord Blood Bank for testing, processing and storage. There is no pain for the mother or baby nor is their safety compromised during the delivery.

Treatments with cord blood Stem cell transplants from umbilical cord blood, researchers say, may be more suitable for transplants than the more common stem cells taken from bone marrow as treatment for various cancers. Umbilical cord blood has an underdeveloped immune cell system providing less of a chance that the transplanted cells will attack the recipients immune system. Hematopoietic stem cells are capable of forming all different types of blood forming cells in the human body. They are used to treat some cancers, metabolic disorders and immunodeficiency diseases, and bone marrow disorders, such as sickle cell anemia. Cord blood is rich in these hematopoietic stem cells.

Research with cord blood Umbilical cord blood stem cells will be valuable for medical research, in studies seeking to advance new treatments for cancer and using cord blood to treat and cure diseases that are not cancers, that is, in regenerative medicine applications. These latter applications may regenerate new tissues such as heart, muscle, skin, and neuronal tissues. Some Upstate researchers have expressed an interest in working with stem cells from umbilical cord blood. An available supply of cord blood would enhance and expedite research studies on finding new treatments for various diseases.

Upstate Cord Blood Bank credits: Architect: Francis Cauffman, New York, N.Y. Engineer: Buro Happold Engineers, New York, N.Y., Project Management: Pike Construction Company, Rochester, N.Y.; General Contractor: Murnane Building Contractors, East Syracuse, N.Y.

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Upstate Opens Cord Blood Bank, Only the Second Public Cord Blood Bank in New York and One of Only 32 in the US - Newswise (press release)

Mayo researcher Abba Zubair is sending stem cells for study on the … – Florida Times-Union

As a boy growing up in Nigeria, Abba Zubair dreamed of becoming an astronaut.

But as he prepared to apply to college, an adviser told him to find a different path.

He said it may be a long time before Nigeria sends rockets and astronauts into space, so I should consider something more practical, Zubair saud.

He decided to become a physician, and is currently the medical and scientific director of the Cell Therapy Laboratory at the Mayo Clinic in Jacksonville. And while hell almost certainly never get to make a journey outside the Earths atmosphere himself, if the weather stays good Saturday hell be sending a payload into space.

A SpaceX Falcon 9 rocket is scheduled to launch at 10:01 a.m. Saturday from the Kennedy Space Center on a cargo delivery mission to the International Space Station. Among the cargo it will be carrying are several samples of donated adult stem cells from Zubairs research lab.

Zubair believes adult stem cells, extracted from bone marrow, are the future of regenerative medicine. Currently at the Mayo Clinic in Jacksonville they are being used in clinical trials to treat knee injuries and transplanted lungs.

But a big problem with using stem cells to treat illnesses is that it may require up to 200 million cells to treat a human being and the cells take a long time to reproduce. Based on studies using simulators on Earth, Zubair believes that the stem cells will more quickly mass produce in microgravity.

Thats the hypothesis hell be testing as the stem cells from his lab spend a month aboard the space station. Astronauts will conduct experiments measuring changes in the cells. They will then be returned on an unmanned rocket and Zubair will continue to study them in his lab.

We want to understand the process by which stem cells divide so we can grow them at a faster rate and also so we can suppress them when treating cancer, he said.

Zubair became interested in the idea of sending stem cells into space four years ago, when he learned of a request for proposals that involved medicine and outer space. Hes been trying to arrange to send stem cells into space for three years.

In May 2015, he sent stem cells to the edge of space as a hot-air balloon carried a capsule filled with cells from his lab to about 100,000 feet then dropped the capsule. The idea was to test how the cells handled re-entry into the Earths atmosphere.

It turned out well, he said. The cells were alive and functioning.

Zubair was supported in that effort as he is being supported in sending cells to the space station by the Center for Applied Spacee Technology. Its chief executive is Larry Harvey, a retired Navy pilot and former astronaut candidate who lives in Orange Park.

While stem cells have myriad potential medical applications, one that particularly interests Zubair is the use of them in treating stroke patients. Its a personal cause to Zubair, whose mother died of a stroke in 1997.

Weve shown that an infusion of stem cells at the site of stroke improves the inflammation and also secretes factors for the regeneration of neurons and blood vessels, he said.

Zubair hasnt entirely given up on his old dream of being an astronaut. Hes applied for the civilian astronaut program. But he doesnt expect that to happen.

Im not sure I made the cut, he said. I just wanted to apply.

And he realizes what a long, strange trip hes made.

I have come so far from Africa to here, he said, and now Im sending stem cells into space.

Charlie Patton: (904) 359-4413

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Mayo researcher Abba Zubair is sending stem cells for study on the ... - Florida Times-Union

THON by the numbers: An anaylsis of the work done and the funds raised – The Daily Collegian Online

With THON merely hours away, lets take a look at the massive event by the numbers. One thing is certain: the statistics are remarkable.

Two-thirds of all money raised since THONs inception in 1977 has been raised in the past 10 years.

Since 2007, THON has raised over $93 million dollars for Four Diamonds, according to the Four Diamonds annual financial reports.

Four Diamonds provides financial and medical assistance to children with pediatric cancer and their families, specifically for care and treatment not covered by insurance or other means as well as additional expenses that disrupt the welfare of the children.

According to Four Diamonds website, 100 percent of all patients bills are covered.

However, the Four Diamonds does not just cover the treatment bills for kids with pediatric cancer. Four Diamonds also enables specialty care providers to be made exclusively available to patients and their families. These specialty care providers offer psychological care, nutritional care, social work such as transportation assistance, musical therapy to help cope with hospital settings and pastoral care for spiritual and religious needs.

Based on the information aggregated from Four Diamonds annual financial and impact reports, Four Diamonds on average takes in and provides funding for treatment for about 93 new patients per year and provides funding for treatment for about 558 patients per year total.

The funding Four Diamonds has received has allowed them to fund researchers and research initiatives for fighting and treating cancer. Specifically, funds from THON have allowed for the creation of stem cell transplant clinic, a pediatric cancer experimental therapeutics research program.

Gil Pak, the Operations Director for the Hershey Childrens Hospital, said the fundraising from

THON is one of the primary contributors to research funding.

The original goal of the Millards, when they founded Four Diamonds was to assist families, but with the fundraising success of THON and Mini-THONs we can go beyond that and invest in research, Pak said. Providing assistance to families will always be the main goal, but now we can go beyond just helping families and help find a cure.

From 2007 to 2014 the funds raised by THON increased steadily. In 2007 THON raised $4,497,001, but by 2012 THON was raising eight figure sums and in 2014 THON raised a record breaking $13.3 million in funds.

However, the last couple years saw a dip in funds raised by THON.

Despite Four Diamonds receiving record of $20 million in 2015, THON actually experienced a slight drop in fundraising from the previous year.

In 2015 THON raised $12,732,897, about $60 thousand less than 2014 when THON raised a record breaking $13,336,675.

Funds raised for the Four Diamonds by THON 2016, $10,262,256 was the lowest since THON 2011 when it raised, $9,266,841.

The cutbacks on canning in 2015 to 2016 (THON 2016 but the 2015-2016 Annual Financial Report), following the death of Vitalya Tally Sepot, a sophomore at Penn State, in September of 2015, may have been one of the factors that led to THON raising less money that year.

Sepots death sent shockwaves through the Penn State community and caused THON to evaluate the safety of canning trips. Several canning trips were cancelled by THON organizers due to concerns about less than ideal weather or other safety concerns.

Other factors could be at play as well. The drop in funds raised could also be that it is just difficult for those canning and participating in other fundraising events to continue raising money that will top the previous years eight figure sums. One student quoted in a previous report by The Daily Collegian said that the number of people canning in the same areas made it more competitive.

However, as reported previously by The Daily Collegian, many of those participating THON do not see topping the previous year's funds as important as how much the funds are benefiting kids with childhood cancer.

Even though funds raised by THON have decreased over the last couple years, the funds raised by Mini-THONs have steadily increased each year.

In 2007, Mini-THONs which are as events similar to THON that are held in elementary schools, middle schools, high schools and colleges, raised $624,246, but by 2016 though less than a decade later funds the funds raised were $5,497,215, about 8.8 times the funds raised by Mini-THONs in 2007.

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THON by the numbers: An anaylsis of the work done and the funds raised - The Daily Collegian Online

New Nanofiber Matrix Enhances Stem Cell Production – Drug Discovery & Development

A new nanofiber-on-microfiber matrix could lead to more and better quality stem cells for disease treatment and regenerative therapies.

The matrix, produced by researchers from Kyoto University in Japan, is made of gelatin nanofibers on a synthetic polymer microfiber mesh and may provide a better way to culture large quantities of healthy human stem cells.

Researchers have been developing 3D culturing systems to allow human pluripotent stem cells (hPSC) to grow and interact with their surroundings in all three dimensions, as they would inside the human body, rather than in two dimensions like they do in a petri dish.

Pluripotent stems cells can differentiate into any type of adult cell and have potential for tissue regeneration therapies, treating diseases and for research.

The majority of 3D culturing systems have limitations and result in low quantities and quality of cultured cells.

The research team was able to fabricate gelatin nanofibers onto a microfiber sheet made of synthetic, biodegradable polyglycolic acid. They then seeded human embryonic stem cells onto the matrix in a cell culture medium.

The matrix allowed for an easy exchange of growth factors and supplements from the culture medium to the cells.

The stem cells also adhered well to the matrix, resulting in robust cell growth. After four days of culture more than 95 percent of the cells grew and formed colonies.

The research team also scaled up the process by designing a gas-permeable cell culture bag in which multiple cell-loaded, folded fiber-to-fiber matrices were placed.

The system was designed so that minimal changes were needed to the internal environment, which reduced the amount of stress placed on the cells. This also yielded a large number of cells compared to conventional 2D and 3D culture methods.

Our method offers an efficient way to expand hPSCs of high quality within a shorter term, the research team wrote in a statement. Additionally, as nanofiber matrices are advantageous for culturing other adherent cells, including hPSC-derived differentiated cells, FF matrix might be applicable to the large-scale production of differentiated functional cells for various applications.

According to the study, clinical-grade scaffolds and high-quality hPSCs are required for cell expansion as well as easy handling and manipulation of the products.

Current hPSC culture methods do not fulfill these requirements because of a lack of proper extracellular matrices (ECM) and cell culture wares.

The layered nano-on-micro fibrous cellular matrix mimicking ECM enables easy handling and manipulation of cultured cells.

The results show that the matrix supports effective hPSC culture with maintenance of their pluripotency and normal chromosomes over two months, as well as effective scaled-up expansion with fold increases of 54.115.6 and 40.48.4 in cell number per week for H1 human embryonic stem cells and 253G1 human induced pluripotent stem cells, respectively.

The study was published in Biomaterials.

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New Nanofiber Matrix Enhances Stem Cell Production - Drug Discovery & Development

How does the Catholic Church resolve new bioethical questions? – Catholic Free Press

Father Tadeusz Pacholczyk

By Father Tadeusz Pacholczyk

A number of years ago, I participated in a debate at Harvard on embryonic stem cell research which also included a Jewish rabbi, an Episcopalian clergyman, and a Muslim imam. The debate went smoothly and cordially, although I was the only voice in the group who defended the human rights of individuals who happen still to be embryos. After the debate, the Episcopalian clergyman pulled me aside and told me how he thought Catholics should consider themselves fortunate to have such an authoritative reference point in the Church and the Vatican, particularly when it comes to resolving new bioethical questions. With surprising candor, he shared how he had sat on various committees with others from his own faith tradition where they had tried to sort through the ethics of embryonic stem cells, and he lamented, we just ended up discussing feelings and opinions, without any good way to arrive at conclusions.

Many people, indeed, appreciate that the Catholic Church holds firm and well-defined positions on moral questions, even if they may remain unsure about how or why the Church actually arrives at those positions, especially when it comes to unpacking new scientific developments like embryonic stem cell research.

So how does the Church arrive at its positions on bioethics? For one thing, it takes its time, and doesnt jump to conclusions even in the face of media pressure for quick sound bites and rapid-fire news stories.

I once had a discussion with a journalist for a major newspaper about the ethics of human-animal chimeras. He mentioned that a leading researcher working on chimeras had met the pope and afterwards implied that the pope had given his blessing to the project. I reminded him that its quite common for the pope to offer general encouragement and blessings to those he meets, though that wouldnt be the same thing as sanctioning new and morally controversial techniques in the biosciences. As a rule, the Catholic Church does not address important bioethical questions that way, through chance encounters with the pope as you are strolling through the hallways of the Vatican.

Instead, the Church may reflect for months, years, or even decades, to identify important considerations and guiding principles when new moral dilemmas arise in the biosciences. Even with this slow and deliberative process, I think its fair to say that the Church generally stays ahead of the curve. By the time of the successful cloning of Dolly the sheep in 1996, for example, the Catholic Church had already been reflecting on the question of human cloning for many years, and concluded, nine years prior to Dolly, that human cloning would be morally unacceptable in an important document called Donum Vitae (On the Gift of Life).

This same document also identified key moral problems with doing human embryonic stem cell research 11 years before it was even possible to destructively obtain those cells from human embryos. When the first test tube baby was born in 1978, the serious moral concerns raised by the procedure had already been spelled out twenty-two years earlier, by Pope Pius XII, in his 1956 Allocution to the Second World Congress on Fertility and Human Sterility wherein he concluded: As regards experiments of human artificial fecundation in vitro, let it be sufficient to observe that they must be rejected as immoral and absolutely unlawful.

Whenever definitive conclusions about medical ethics are reached or otherwise clarified by the Church, they are normally promulgated through official Church documents, like papal encyclicals and addresses, or, with the approval of the pope, documents and commentaries from the Congregation for the Doctrine of the Faith (CDF the Vatican office responsible for preserving and interpreting Catholic doctrine), or other congregations, councils or dicasteries of the Church.

Even today, certain bioethical controversies remain under active discussion within the Church, such as the question of whether it would be allowable to adopt abandoned frozen embryos by implanting and gestating them in volunteer mothers. While a 2007 CDF document expressed some reservations and concerns about the proposal, debate continues inside and outside the Vatican.

New medical discoveries and technological developments challenge us to careful moral reflection and discernment. These scientific developments can either be an opportunity for genuine human advancement or can lead to activities and policies that undermine human dignity. The U.S. Bishops in a recent document summed it up this way: In consultation with medical professionals, church leaders review these developments, judge them according to the principles of right reason and the ultimate standard of revealed truth, and offer authoritative teaching and guidance about the moral and pastoral responsibilities entailed by the Christian faith. While the Church cannot furnish a ready answer to every moral dilemma, there are many questions about which she provides normative guidance and direction.

Rev. Tadeusz Pacholczyk, Ph.D. earned his doctorate in neuroscience from Yale and did post-doctoral work at Harvard. He is a priest of the diocese of Fall River, MA, and serves as the Director of Education at The National Catholic Bioethics Center in Philadelphia. See http://www.ncbcenter.org

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How does the Catholic Church resolve new bioethical questions? - Catholic Free Press

Mayo researcher Abba Zubair is sending stem cells for study on the International Space Station – St. Augustine Record

As a boy growing up in Nigeria, Abba Zubair dreamed of becoming an astronaut.

But as he prepared to apply to college, an advisor told him to find a different path.

He said it may be a long time before Nigeria sends rockets and astronauts into space, so I should consider something more practical, Zubair saud.

He decided to become a physician, and is currently the medical and scientific director of the Cell Therapy Laboratory at the Mayo Clinic in Jacksonville. And while hell almost certainly never get to make a journey outside the Earths atmosphere, if the weather stays good Saturday hell be sending a payload into space.

A SpaceX Falcon 9 rocket is scheduled to launch at 10:01 a.m. Saturday from the Kennedy Space Center on a cargo delivery mission to the International Space Station. Among the cargo it will be carrying are several samples of donated adult stem cells from Zubairs research lab.

Zubair believes adult stem cells, extracted from bone marrow, are the future of regenerative medicine. Currently at the Mayo Clinic in Jacksonville they are being used in clinical trials to treat knee injuries and transplanted lungs.

But a big problem with using stem cells to treat illnesses is that it may require up to 200 million cells to treat a human being and the cells take a long time to reproduce. Based on studies using simulators on Earth, Zubair believes that the stem cells will more quickly mass produce in microgravity.

Thats the hypothesis hell be testing as the stem cells from his lab spend a month aboard the space station. Astronauts will conduct experiments measuring changes in the cells. They will then be returned on an unmanned rocket and Zubair will continue to study them.

Zubair became interested in the idea of sending stem cells into space four years ago, when he learned of a request for proposals that involved medicine and outer space. Hes been trying to arrange to send stem cells into space for three years.

In May 2015, he sent stem cells to the edge of space as a hot-air balloon carried a capsule filled with cells from his lab to about 100,000 feet then dropped the capsule. The idea was to test how the cells handled re-entry into the Earths atmosphere.

It turned out well, he said. The cells were alive and functioning.

Zubair was supported in that effort as he is being supported in sending cells to the space station by the Center for Applied Science Technology. Its chief executive is Lee Harvey, a retired Navy pilot and former astronaut candidate who lives in Orange Park.

While stem cells have myriad potential medical applications, one that particularly interests Zubair is the use of them in treating stroke patients. Its a personal cause to Zubair, whose mother died of a stroke in 1997.

Weve shown that an infusion of stem cells at the site of stroke improves the inflammation and also secretes factors for the regeneration of neurons and blood vessels, he said.

Zubair hasnt entirely given up on his old dream of being an astronaut. Hes applied for the civilian astronaut program. But he doesnt expect that to happen.

Im not sure I made a cut, he said. I just wanted to apply.

And he realizes what a long, strange trip hes made.

I have come so far from Africa to here, he said, and now Im sending stem cells into space.

Charlie Patton: (904) 359-4413

The rest is here:
Mayo researcher Abba Zubair is sending stem cells for study on the International Space Station - St. Augustine Record

Local vet taking part in stem cell therapy study for dogs – WPTV.com

TARPON SPRINGS, Fla. - Cosby just doesnt get around like he used to.

We have six dogs and hes always the one thats the last to get up. The last to get out, said his owner Brian Cirillo.

And for Cirillo, its sad to see.

I hate it. Its always like he always on his tippy topes on his back legs. So its heartbreaking.

But a new trial study that is about to start at the St. Francis Pet Care Center in Tarpon Springs, could be just what Cosby needs.

Veterinarian Mike Amsberry is offering stem cell therapy for dogs.

They are seeing that its very, very safe. And very effective.

This study is focused specifically on four-legged friends with arthritis.

But in the past hes seen stem cell treatments work wonders for other ailments.

Its cells treating the body, rather than then some foreign substance. Some medication.

In this trial, the stem cells come from umbilical cords of donor dogs.

Not only can qualified pets get the treatment for free, but owners are paid too.

Cosby seems like the perfect candidate.

I think thats where everything seems to be going with regard to medicine. So to be on the leading edge of that to potential help him without having to put him on a bunch of medicine is definitely a plus, said Cirillo.

The hope is one day Cosby will be able to keep up with the rest.

And lead the way to help thousands of other dogs.

For more information on the trial study go to petstemcells.org.

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Local vet taking part in stem cell therapy study for dogs - WPTV.com

Mesoblast’s cell therapy candidate MPC-300-IV shows positive effect in mid-stage study in treatment-resistant … – Seeking Alpha

A Phase 2 clinical trial assessing Mesoblast's (NASDAQ:MESO) allogeneic cell therapy candidate MPC-300-IV in patients with rheumatoid arthritis (RA) who have not responded to anti-Tumor Necrosis Factor (TNF) therapy [e.g., AbbVie's Humira (adalimumab)] showed a durable improvement in symptoms, physical function and disease activity with no safety signals observed.

The study enrolled 48 patients with active RA who were on a stable regimen of methotrexate and had an inadequate response to at least one anti-TNF agent. 63% (n=30/48) has received 1 - 2 biologic agents. Participants were randomized to receive a single infusion of 1MMesenchymal Precursor Cells(MPCs)/kg (n=16), 2M MPCs/kg (n=16) or placebo (n=16). The primary study period was 12 weeks and the total study duration was 52 weeks.

Both doses of MPC-300-IV outperformed placebo at both week 12 and week 39 as measured by ACR20/50/70 (ACR20 = 20% improvement in RA symptoms). The 2M-cell cohort showed the greatest response. At week 12, for example, 27% of the 2M-cell group achieved ACR70 compared to 0% for placebo. The proportion was 36% in the subgroup (n=11) who had received 1 - 2 biologics. The treatment effect was maintained through week 39.

The clinical development of MPC-300-IV is ongoing.

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Mesoblast's cell therapy candidate MPC-300-IV shows positive effect in mid-stage study in treatment-resistant ... - Seeking Alpha

Capricor Drops Cenderitide to Focus on Cell and Exosome Therapies – Genetic Engineering & Biotechnology News

Capricor Therapeutics is dropping further development of its Phase II-stage heart failure drug Cenderitide and is terminating its license agreement with the Mayo Clinic for the natriuretic peptide receptor agonist program. "Our decision to return these rights is a strategic move as we prioritize our efforts to advance our core cell and exosome-based therapeutic development programs," said Linda Marbn, Ph.D., Capricor president and CEO.

Capricor said it expects to reach key clinical milestones with both the cell and exome therapy programs during 2017. Topline data from the Phase I/II HOPE clinical study with lead cell therapy candidate CAP-1002 (allogeneic cardiosphere-derived cells) is expected during Q2 2017. The trial is evaluating CAP-1002 in patients with Duchenne muscular dystrophy (DMD)-associated heart disease. The firm said it is separately planning a clinical trial, anticipated to start during 2017, to investigate whether CAP-1002 therapy can boost skeletal muscle function in men and boys with DMD.

We are also committing increased attention to our exosomes program, and we expect to file an IND application for CAP-2003 (cardiosphere-derived cell exosomes) in the second half of this year," added Dr. Marbn. Capricor is developing CAP-2003 for the potential treatment of ophthalmic disorders, including ophthalmic graft-versus-host disease (oGvHD).

CAP-1002 is an allogeneic cardiac cell therapy product manufactured from donor heart tissue. The treatment has been granted orphan drug designation by the FDA for the treatment of DMD. Capricor and partner Janssen Biotech are developing a commercial manufacturing process for CAP-1002 as part of their potentially $337.5 million collaboration, established in January 2014, to develop the cell therapy program for cardiovascular applications. In May of last year, Capricor received a $3.8 million grant from the California Institute for Regenerative Medicine to support the Phase I/II HOPE study.

CAP-2003 exosomes are obtained from CAP-1002 cardiosphere-derived cells (CDCs). The exosome technology is being developed under a 2014 license agreement with the Cedars-Sinai Medical Center. In October 2016, Capricor was granted up to $4.2 million from the NIH to evaluate CAP-2003 exosome therapy for treating hypoplastic left heart syndrome. The previous month the firm received a $2.4 million grant from the U.S. Department of Defense to establish a commercial manufacturing platform for CAP-2003.

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Capricor Drops Cenderitide to Focus on Cell and Exosome Therapies - Genetic Engineering & Biotechnology News