Stem Cell Clinic

Therapeutic Exosomes Go From Vein to Brain – Technology Networks

By adminDecember 13, 2019Stem Cell Medical Center

A team of researchers from the University of Georgia's Regenerative Bioscience Center has found that neural exosomes--"cargo" molecules within the nervous system that carry messages to the brain--can minimize or even avert progression of traumatic brain injury when used as part of a new cell-to-cell messaging technology.

The finding could result in the first delivery platform and regenerative treatment for TBI.

The research was reported on Nov. 27 in the Journal of Neurotrauma and outlines significant steps in providing data for industry development and commercial manufacturing of a regenerative TBI-IV therapy.

The new restorative technology contains bio-manufactured exosomes that can be stored on the shelf and given as an injection into a vein. Once injected, the exosomes become message mediators to reset, regenerate and coordinate communication with both neighboring and distant cells. As a result, this novel treatment showed improved functional recovery in rats after TBI.

"The technology takes full benefit of the desirable properties of a neural stem cell therapy without introducing cells into patients," said Steven Stice, Georgia Research Alliance Eminent Scholar and D.W. Brooks Distinguished Professor in the College of Agricultural and Environmental Sciences. "We are working toward a therapeutic that has a multifunctional promise to repair brain injury and be producible in a cost-effective, off-the-shelf drug format."

Traumatic brain injuries can be difficult to detect because each TBI patient presents a unique set of circumstances determined by injury timeline and severity, as well as individual characteristics such as age, gender and occupation. With this potential new technique, RBC researchers hope to boost the brain's natural ability to recover and provide physicians with a treatment that can be administered immediately in cases of severe TBI.

"Mechanistically, TBI is a physician's nightmare," said Lohitash Karumbaiah, associate professor of regenerative medicine in UGA's College of Agricultural and Environmental Sciences and one of the publication's lead authors. "Because there are so many things going on in the brain, you can't really exactly pinpoint what is going wrong, and without therapies to immediately improve recovery, the situation becomes extremely complex."

For those affected by TBI, treatment could be administered at the time of injury with IV fluids. Using multiple low-dose, intravenous injections, the novel treatment is designed to speed up regeneration of neurons and supporting cells following injury.

"Administrating exosomes into a patient's IV drip would always be preferable to invasive brain surgery," Karumbaiah said. "What we can do is give physicians a fighting chance to regulate the inflammatory response of TBI, rather than trying to treat it after it occurs."

Another application the team has considered is the use of exosome technology as a preventive management program for all levels of TBI, including mild and moderate concussions that could minimize or prevent future damage.

Licensed to and under development by Aruna Bio, one of UGA's first Innovation Gateway startups, the exosome technology has already captured the interest and financial support of the Georgia Research Alliance's Venture Fund, one of the largest venture capital funds in the state. The GRA Venture Fund, along with other investors, contributed to the $13 million in common stock financing recently announced by Aruna Bio.

"Drug development for acute TBI has suffered so many clinical failures and will need to take an imminent paradigm shift toward a more targeted and personalized treatment," said Stice. "We still have a lot to learn, but our success could create the tipping point."

Reference: Sun, M. K., Passaro, A. P., Latchoumane, C.-F., Spellicy, S. E., Bowler, M., Goeden, M., Karumbaiah, L. (2019). Extracellular vesicles mediate neuroprotection and functional recovery after traumatic brain injury. Journal of Neurotrauma. https://doi.org/10.1089/neu.2019.6443

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Blinatumomab May Become New Standard of Care for Post-Reinduction Therapy in Young Patients With B-ALL – Cancer Therapy Advisor

By adminDecember 13, 2019Stem Cell Medical Center

According to an analysis of interim results from a randomized study of young patients with high- or intermediate risk B-cell precursor acute lymphoblastic leukemia (B-ALL) at first relapse, the overall efficacy and safety of post-reinduction therapy with the bispecific T-cell engager (BiTE) blinatumomab outperformed conventional chemotherapy. The findings from this study were presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition held in Orlando, Florida.

Disease relapse, particularly early relapse, following initialtreatment of children, adolescents, and young adults with B-ALL is a marker ofpoor prognosis. While allogeneic hematopoietic stem cell transplantation (HSCT) is typicallyconsidered the treatment of choice for these patients, barriers to itsimplementation can include concerns related to adverse events associated withreinduction and subsequent consolidation chemotherapy, and the presence ofminimal residual disease (MRD) following administration of second-remission reinductiontherapy. Those patients who experienceearly bone marrow relapse, and those with MRD greater than 0.1% in the settingof a prolonged CR, at the end of reinduction therapy are considered to have high-riskand intermediate-risk disease, respectively.

Blinatumomab is an artificial, bispecific monoclonal antibody-basedconstruct created from the fusion of single-chain variable fragments from 2different antibodies. In the case of blinatumomab, targets include the CD3receptor on T cells and CD19 on B cells, resulting in the formation of a link betweenthese 2 cell types.

Currently, blinatumomab is approved by the US Food and DrugAdministration (FDA) for the treatment of adult and pediatric patients with B-ALL in first or second CR,with minimal residual disease (MRD) greater than or equal to 0.1%, as well asfor patients with relapsed/refractory B-ALL.2

In this phase 3 Childrens Oncology Group study (AALL1331; ClinicalTrials.gov Identifier: NCT02101853), patients with B-ALL in first relapse between the ages of 1 and 30 years with bone marrow blasts less than 25% and/or failure to clear extramedullary disease following reinduction chemotherapy (UKALLR3 regimen3) were randomly assigned in a 1:1 ratio following risk assessment to receive either 2 blocks of intensive consolidation chemotherapy according to the UKALLR3 regimen3 or two 4-week cycles of otumumab separated by a 1-week break. Allogeneic HSCT was scheduled following these treatments.

The primary end point of the trial was intent-to-treat disease-freesurvival (DFS), with secondary study end points including MRDresponse, overall survival (OS), and ability to proceed to HSCT.

A planned interimanalysis of 208 patients, performed followingthe occurrence of approximately 60% of expected events,included only those with high- (67%) or intermediate-risk (33%) disease. Patientages ranged from 1 to 27 years, with a median age of 9 years.

At a medianfollow-up of 1.4 years, some of the key efficacy findings from this analysis includedrates of 2-year DFS in the intention-to-treat (ITT) population of 41.0% forpatients receiving chemotherapy and 59.3% for those treated with blinatumomab (P =.050). Rates of 2-year OS forpatients in these 2 study arms were 79.4% (blinatumomab) and 59.2% (chemotherapy),(P =.005).

The percentages ofthose achieving undetectable MRD after reinduction chemotherapy were only 22%and 18% in the chemotherapy and blinatumomab arms, respectively. Followingblock 2 of chemotherapy (ie, first cycle of consolidation chemotherapy) orcycle 1 of blinatumomab, rates of undetectable MRD increased to 29% in thechemotherapy arm and 76% in the blinatumomab arm (P <.0001).

Regarding resultsrelated to MRD response, all of the benefit of blinatumomab with respect to MRDclearance appeared to occur in the first cycle, commented PatrickA. Brown of the Sidney Kimmel ComprehensiveCancer Center, Johns Hopkins University, Baltimore, Maryland, who was thepresenting study author.

Furthermore, 45%of patients in the chemotherapy arm compared with 73% of those in the blinatumomabarm were able to proceed to HSCT (P<.0001).

Regarding patientsafety, 4 and 0 patients receiving blinatumomab or chemotherapy, respectively,experienced a postinduction, induction-related toxic death.

In addition, thefrequencies of specific adverse events were considerably higher in thechemotherapy vs the blinatumomab arm. For example, rates of grade 3 or higher febrileneutropenia were 44% and 46% for patients receiving the 2nd and 3rd blocks ofthe UKALLR3 regimen, respectively, but only 4% and 0% of patients receivingcycle 1 and cycle 2 of blinatumomab, respectively (P <.001). Similar differences between the 2 study arms wereobserved with respect to the rates of infections and sepsis.

For patientsreceiving blinatumomab, low-grade cytokine release syndrome (CRS), occurring in22% of patients, was generally limited to the to the first cycle. Seizuresoccurred in 4% and 0% of patients during cycles 1 and 2, respectively, and the incidenceof mostly low-grade encephalopathy was 14% in cycle 1 and 11% in cycle 2.

Accordingto the results of this scheduled interim analysis, the prespecified monitoring thresholdto the primary end point of DFS was not crossed. However, based on the overallresults of the study, the data monitoring committee recommended permanentclosure of study randomization for patients with high- or intermediate-riskdisease, with those in these risk groups immediately crossed over to theblinatumomab arm.

We believe that blinatumomab constitutes anew standard of care in this setting, concluded Dr Brown.

Disclosure:Some of the authors disclosed financial relationships with the pharmaceuticalindustry. For a full list of disclosures, please refer to the originalabstract.

Read more of Cancer Therapy Advisors coverage of ASHs annual meeting by visiting the conference page.

References

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Blinatumomab May Become New Standard of Care for Post-Reinduction Therapy in Young Patients With B-ALL - Cancer Therapy Advisor

Deacon Butch King learns to accept the ‘gift’ of cancer – Arkansas Catholic

By adminDecember 13, 2019Stem Cell Medical Center

By Aprille HansonAssociate Editor

Aprille Hanson

Deacon Butch King and his wife Debbie (left) stand with their daughter Paula Draeger (center) in front of the Seed of Hope garden at UAMS Winthrop P. Rockefeller Cancer Institute in Little Rock Dec. 4. Last month, King was able to place a seed of hope token into the garden, signifying he is cancer-free, thanks to a stem cell donation from his daughter.

Aprille Hanson

Deacon Butch King was given a gift in 2017. He was diagnosed with a rare disease MDS/MPN, myelodysplastic/myeloproliferative neoplasm-unclassifiable to be exact.

The hybrid disease results when bone marrow overproduces unhealthy blood cells, according to University of Arkansas for Medical Sciences in Little Rock.

The diagnosis sent the family on a harrowing journey for the next two and a half years: four changes of insurance coverage and medical facilities, 19 rounds of chemotherapy, 430 lab results, 14 bone marrow biopsies, 11.25 gallons of donated blood and the disease progressing to Acute Myeloid Leukemia.

Looking at a deadly disease as a gift takes a radical faith in God, one that King and his wife Debbie have carried with grace to his cancer-free diagnosis Nov. 4.

It was given to us as a gift. And how do we manage gifts? We care for them, we nurture them, we polish them, show them off with pride and we give thanks to God. Those are his words, our words together. We had a gift and we had to manage it, we didnt get a choice, his wife said.

King was ordained a deacon in 2012, serving at Immaculate Conception Church in North Little Rock. The couple has four children, 12 grandchildren and six great-grandchildren, with another on the way in March. After 23 years of serving in the U.S. Air Force working in secure communications, he spent 22 years with the U.S. Postal Service.

In October 2016, he had a metal stent placed in his heart and could not have any surgeries for the following six months. In November of that year, he twisted his knee at work. When he was finally ready to have knee surgery in May, his lab work was irregular. In June they learned he had developed a rare blood disorder, MDS, which later in the year progressed to MPN. It required a stem cell transplant, with only a 30 percent chance of surviving a transplant.

I was kind of stunned at first, King said. As a deacon, he had been used to visiting the sick in nursing homes and hospitals.

This is one of the stories you can say, I know how you feel because Ive been there or were praying for you and really mean it, he said.

With every roadblock of insurance not covering the procedure or a hospital turning the transplant down because he was high risk, faith prevailed.

In December 2017, their youngest daughter Paula Draeger, 38, was a perfect match for a stem cell transplant, an extremely rare result.

OK, we can do this; were going to heal him. Weve got the perfect match. If this doesnt work, nothing will. So that was just kind of the reaction, lets do it, the married mother of two said.

Debbie King said, Shes a Spina bifida baby. We were told that she would be a vegetable when she had her spinal surgery. So shes a miracle to be here; long before this ever came God had a plan.

Once Medicare kicked in, insurance would cover a transplant if a clinical trial was available. It led the family to 13 visits to University of Oklahoma Stephenson Cancer Center in Oklahoma City, though they refused the transplant.

Debbie King said they specifically chose Oklahoma City because the family had been, and still are, praying daily for Blessed Stanley Rothers intercession.

The martyr, who grew up on a farm in Okarche, Okla., was declared blessed on Sept. 23, 2017, in Oklahoma City. He was killed in 1981 while serving his people in Guatemala.

He needed a miracle. And we said God provides miracles, Debbie King said.

Before we started any treatment we would place the entire illness and what would be happening at Blessed Stanley Rothers gravesite in Oklahoma City, visiting 11 times, she said.

Whats the miracle? The miracle is the faith. And thats what Butch has said, she said.

On March 13, the Kings were told they wouldnt be continuing the trial in Oklahoma.

We were ready to just be on maintenance and enjoy the days we had, she said. On March 14, our 44th wedding anniversary we were celebrating what we thought could be our last one.

But Dr. Appalanaidu Sasapu, hematologist oncologist with the UAMS Stem Cell Transplantation and Cellular Therapy Program, never gave up on them. Because Kings disease had progressed to leukemia in April, the stem cell procedure could now be done at UAMS and covered by insurance.

Draeger said the stem cell donations, done over a weekend via a port, were simple, with no side effects aside from building her energy up in the following week.

For what youre able to give somebody, what you have to endure pales in comparison to what hes been through and what you can give him, she said.

King no longer has the blood disease and is cancer free, though he will continue at least a years worth of chemotherapy treatments.

Since his diagnosis, they attend the smaller St. Patrick Church in North Little Rock for Mass, but he cannot yet return to ministry.

We do our prayer time in the mornings and evenings, we count our blessings every night before we go to bed and we just know, what was our blessing today? Did we see somebody that we havent seen before that God put in our path? Is it a new doctor who is going to take this on? King said.

But through this whole process weve been truly blessed, had no regrets. If I had to do it over, if thats the path of my life that God wants me to take, then Ill do it.

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Deacon Butch King learns to accept the 'gift' of cancer - Arkansas Catholic

14 Advantages and Disadvantages of Embryonic Stem Cell …

By adminDecember 13, 2019Embryonic Stem Cells

Embryonic stem cells are derived from embryos that develop from eggs that were created through the in vitro fertilization process. These eggs are then donated for research purposes with the informed consent of their donors. Researchers do not derive embryonic stem cells from eggs that are fertilized in a womans body.

Women do not have abortions to harvest their embryonic stem cells, nor do any providers sell fetal tissue for these cell lines to develop.

Human embryonic stem cells come from a transference of cells from a preimplantation-stage embryo in a laboratory culture dish. It is mixed with culture medium, allowing the cells to divide, and then spread over the surface of this dish. These cells can then develop into all three derivatives of the primary germ layers, making it possible for them to eventually turn into one of the over 200 different cell types that are found in the human body.

It is not possible to save the embryo when these cells are harvested from it, which ends the potential future viability of human life. That is where a majority of the embryonic stem cell research pros and cons focus on when discussing this subject. How you personally define human life will usually dictate which side of the debate you support.

1. The embryonic stem cells are harvested 5-7 days after conception.Adult stem cells do not provide the same benefits as embryonic ones from a therapeutic standpoint. They fall short in their viability to treat genetic diseases. Thats because the same disease found in the adult body can be present in their stem cells. When the harvesting takes place, it occurs during the first week after conception. At this stage, the embryo has not yet developed to a stage where a personal identity can be assigned to it, nor can it live outside of a specialized environment. It is essentially a mass of cells.

2. Researchers use the embryonic stem cells from discarded embryos.The IVF process which creates embryos for transplantation in the first place are often discarded without a second though. Reporting by The Telegraph in 2012 found that over 1.7 million human embryos were discarded during or after the conception process. Between 1991-2012, there were 3.5 million human embryos created, but only roughly 235,000 successful implantation procedures. About 840, 000 were put into cold storage, while just 2,000 were stored for donation.

Approximately 6,000 embryos during this two-decade period were set aside for medical research. Compared to the 1.4 million that were implanted as a way to start a pregnancy, where 1 in 6 failed, the issue of morality is more complex than the black-and-white world that some people create.

3. Embryonic stem cells can be harvested ethically from almost any perspective.New technologies make it possible for doctors to harvest remaining embryonic stem cells from the umbilical cord after a child is born. Even if parents decide to store cord blood instead of make a donation, the product can still contain a line of embryonic stem cells that could be useful for research purposes. Since the umbilical cord doesnt stay attached to the child, nor does the cord blood get reabsorbed into the mother or the baby, the only way to unethically take this resource is to do it without asking.

4. Pain is not felt during the embryonic stem cell procedure.When researchers destroy an embryo as they harvest the line of stem cells that develops, there is no pain experienced by this cell group. Researchers believe that a fetus doesnt have the concept of pain developed until around the 20th week of gestation. Most of the embryos that are used for this process have been frozen anyway, kept in storage because there is no intention to use the cells to hopefully create a pregnancy one day anyway.

Fetal tissues wouldnt supply the embryonic stem cells anyway because at that stage of development, they have already turned into what they are going to be. That makes it virtually impossible to study their qualities at the level where they would be medically beneficial.

5. No embryonic stem cells are taken without consent.IVF doctors dont take fertilized eggs away from women or couples with an evil laugh, thinking about all the dastardly ways they can manipulate embryonic stem cells for personal gain. People dont steal frozen embryos, encourage abortions, or harvest the tissues from a growing fetus to serve a medical or political agenda. Every embryonic stem cell line comes from the consent of its donor. No research on those cells will take place unless there is explicit consent offered by those involved.

6. We do not know the full potential of this new field of medical science.We are still in the early stages of research to determine the full potential value of embryonic stem cells as a treatment option for some individuals. As the University of Michigan notes, it may lead to more effective treatments for serious human ailments. The future discoveries in this field could alleviate the suffering for millions of people around the world. Spinal cord injuries, Parkinsons disease, Alzheimers disease, and juvenile diabetes are just a few of the conditions which could be improved if medical studies are given time and funding to reach a conclusion.

7. Treatments using embryonic stem cells have already produced results.Early embryonic stem cell treatments through the use of cord blood therapies have already produce positive outcomes for roughly 10,000 people. These treatments offer new ways to find a cure for 70+ different diseases with this option. Kids that have an immunodeficiency disorder and receive this form of treatment see a treatment success rate of 90% today and that figure continues to grow.

1. It destroys the future potential of human life.Whether you feel that life begins at conception, at some stage in the womb, or after birth, everyone can agree on the idea that an embryo represent the future potential of life. We can get lost in the semantics of how life begins to support how we feel, but the bottom line here is that the termination of an embryo stops the future potential for that group of cells. Using it for research purposes, even with the consent of the mother or couple involved, means youre trading future human potentiality for current potentiality. Is that really a justifiable action?

2. The number of successful treatment outcomes is relatively minor.There are significant barriers in place when looking at the potential of an embryonic stem cell treatment. There are unstable gene expressions which occur when this method is used, along with the formation of tumors, and some people even see a failure in the cells ability to activate to a specific purpose. Until these challenges are addressed in clinical settings, the full potential of this treatment can never be realized. Does it make sense to continue harvesting cells from embryos if the failure rate remains high?

3. People can still reject embryonic stem cell treatments.The human body naturally rejects the items that are not part of its regular genetic makeup through its immune system response. That is why the people who go through an organ transplant procedure receive anti-rejection medication that slows or stops this response. Even if the embryonic stems cells go through their regular activation method, there is still the potential of rejection present.

Even if an embryonic clone of an individual could be created to product exact cells which mimic the bodys genetic makeup, there would still be a risk of rejection because of the genetic duplication process.

4. It can be argued that embryos do meet the definition of life from a scientific view.There are currently three specific guidelines in the framework of the definition of life as we think of it when encountered on our planet or perhaps elsewhere in our solar system or galaxy one day.

There must be a capacity for growth that produces functional activity. It must offer some type of reproduction capability during one stage of its existence. There must be a change which occurs over the lifetime of the cells in question that happens before death.

5. People fund research activities with their taxes.Federal law in the United States prohibits taxpayer funding to be used for abortion services unless specific exceptions apply. What many people do not realize is that over $500 million in research funding has been given to this medical field since 1996 because even though portions of this field were outlawed, all existing stem cell lines currently being worked on at the time were grandfathered into the legislative process.

The Supreme Court affirmed the federal stem cell research could continue in 2013 despite a long-running appeal that such an action is a violation of the Dickey-Wicker Act that prohibits the destruction of an embryo.

6. It is a time-consuming process to create viable embryonic stem cells.For the stem cells to become a viable research tool, they must undergo several months of development in strict laboratory conditions before they are valuable in any way. Then there is the cost involved with the process as well. The 2017 estimated federal funding for all categories of stem cell research was $1.58 billion. Embryonic stem cell research received $347 million, while umbilical cord blood or placenta-based stem cells received $34 million.

7. Many of the stem cell lines under research are two decades old.Most of the approved embryonic stem cell research lines that are worked on in the United States were created on or before August 2001. Those lines were found to be contaminated with animal proteins, which seems to have prevented any of them from being created as a model to treat human disease. Only 16 out of the 70 lines approved by the federal government remain because a majority of them were inadequately characterized. These cells also came from an Israeli clinic, which means they do not incorporate the levels of racial and ethnic diversity that genuine research requires.

The pros and cons of embryonic stem cells look at the potential of what this new field of medical research could provide compared to the harm it may cause. There are deeper issues here that go beyond we can or we shouldnt. Since a majority of embryos are thrown away, shouldnt there be outrage over that fact from the pro-life movement? And since 1 in 6 attempted implants fails, is there not more to consider here than the intentional actions of research? By taking a look at all sides of this issue, the debate tends to become a complex set of moral judgments made on the individual level instead of a generic right or wrong answer to determine.

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14 Advantages and Disadvantages of Embryonic Stem Cell ...

How Will Animals Get Benefitted by Stem Cell Therapy? – Medical Tech Outlook

By adminDecember 13, 2019Embryonic Stem Cells

ESPCs derived from pig provide important implications for developmental biology, organ transplantation, regenerative medicine, disease modeling, and screening for drugs.

FREMONT, CA: Stem cell therapy, usually applied to humans, is now extended to animals too. It is a regenerative treatment applied to cats, dogs, pigs, and other animals. It includes removing cells from bone marrow, blood or fat, umbilical cords, and the cell can grow into any kind of cell and can repair damaged tissues. The regenerative therapy has been successful in animals. It can be used mainly for the treatment of spinal cord and bone injuries along with the problems with tendons, ligaments, and joints. One of the breakthroughs is the embryonic stem cell lines obtained from the pig.

Scientists have derived Expanded Potential Stem Cells (EPSCs) from pig embryos for the first time. They offer the groundbreaking potential to study embryonic development and produce translational research in genomics and regenerative medicine. Embryonic stem cells (ESC) are derived from the inner cells of early embryos called blastocysts. They are pluripotent cells as they can develop into various cell types of the body in the culture dish. The newly derived porcine EPSCs isolated from pig embryos are the first well-characterized cell lines worldwide. Their pluripotent ability provides important implications for developmental biology, organ transplantation, regenerative medicine, disease modeling, and screening for drugs.

The stem cells can renew themselves, showing that they can be kept in culture indefinitely while showing the typical morphology and gene expression patterns of embryonic stem cells. Because somatic cells have a limited lifespan, they cannot be used for such applications, and therefore the new stem cells are better suited for the lengthy selection process. These porcine stem cell lines can easily be edited with new genome editing techniques like CRISPR/Cas, and are currently the simplest, most versatile and precise method of genetic manipulation.

The EPSCs have a greater capacity to develop into numerous cell types of the organism as well as into extraembryonic tissue, the trophoblasts, rending them very unique and, thus, their name. This capacity is valuable for the future promising organoid technology where organ-like small cell aggregations are grown in 3D aggregates and used for research into early embryo development, various disease models, and testing of new drugs in Petri dishes. Also, they offer a unique possibility to investigate functions or diseases of the placenta in vitro.

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Aspen Neuroscience Launches With $6.5 Million Seed Funding to Advance First-of-its-Kind Personalized Cell Therapy for Parkinson’s Disease – P&T…

By adminDecember 13, 2019Embryonic Stem Cells

SAN DIEGO, Dec. 12, 2019 /PRNewswire/ -- Aspen Neuroscience, Inc. today announced its launch following a $6.5 million seed round led by Domain Associates and Axon Ventures and including Alexandria Venture Investments,Arch Venture Partners,OrbiMedand Section 32 to develop the first autologous cell therapies for Parkinson's disease. Aspen's proprietary approach was developed by the company's co-founders, Jeanne F. Loring, Ph.D., Professor Emeritus and founding director of the Center for Regenerative Medicine at The Scripps Research Institute, and Andres Bratt-Leal, Ph.D., a former post-doctoral researcher in Dr. Loring's lab. The company was initially supported by Summit for Stem Cell, a founding partner and non-profit organization which provides a variety of services for people with Parkinson's disease. Aspen is led by industry veteran Howard J. Federoff, M.D., Ph.D., as Chief Executive Officer.

Parkinson's disease is characterized by the loss of specific brain cells that make the chemical dopamine. Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems. Aspen is focusing on human pluripotent stem cells, cultured cells that can become any cell type in the human body. The company's research is specific to induced pluripotent stem cells (iPSCs), which it develops by taking a skin biopsy from a person with Parkinson's disease and turning the tissue into pluripotent stem cells using genetic engineering. Aspen then differentiates the pluripotent stem cells into dopamine-releasing neurons that can be transplanted into that same person (autologous), thereby restoring the types of neurons lost in Parkinson's disease.

As an autologous cell therapy for Parkinson's disease, Aspen's treatment would eliminate the need for immunosuppression because the neurons are transplanted back into the same patient from which they were generated. The use of immunosuppression is necessary with currently available cell therapies for Parkinson's disease and when transplanting cells from one patient to another (allogeneic) to prevent rejection but can pre-dispose the patient to life-threatening complications including infection and add cost to the patient and health system. Aspen is the only company in the world offering an autologous neuron replacement therapy for Parkinson's disease.

Aspen encompasses a powerful executive leadership team including Dr. Federoff who, in addition to his leadership roles at the UC Irvine Health System, was the Executive Vice President for Health Sciences and the Executive Dean of Medicine at Georgetown University. Dr. Federoff also has significant biotech industry experience including co-founding MedGenesis Therapeutix and Brain Neurotherapy Bio, as well as leading the U.S. Parkinson's Disease Gene Therapy Study Group. The company is also proud to announce the addition of several experienced and well-known members to its leadership team including Edward Wirth, M.D., Ph.D., as Chief Medical Officer.

Dr. Wirth currently serves as the Chief Medical Ofcer for Lineage Cell Therapeutics where he oversees clinical development of its two therapeutic programs for spinal cord injuries and lung cancer. He received his M.D. and Ph.D. from the University of Florida in 1994 and remained to conduct postdoctoral research including leading the University of Florida team that performed the rst human embryonic spinal cord transplant in the U.S. Dr. Wirth went on to serve as the Medical Director for Regenerative Medicine at Geron Corporation where the world's rst clinical trial of human embryonic stem cell (hESC)-derived product occurred which demonstrated initial clinical safety.

Drs. Federoff and Wirth are joined by Dr. Loring, as Chief Scientific Officer; Jay Sial, as Chief Financial Officer; Andres Bratt-Leal, Ph.D., as Vice President of Research and Development; Thorsten Gorba, Ph.D., as Senior Director of Manufacturing and Naveen M. Krishnan, M.D., M.Phil., as Senior Director of Corporate Development.

"Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinson's disease," said Dr. Federoff. "We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist."

Aspen's lead product (ANPD001) is currently undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic Parkinson's disease. Aspen is also developing a gene-edited autologous neuron therapy (ANPD002) that is in the research stage and targeted toward familial forms of Parkinson's disease beginning with the most common genetic variant in the gene encoding glucocerebrosidase (GBA). Aspen leverages proprietary machine-learning tools and artificial intelligence to ensure quality control during manufacturing and to deliver a safe and reproducible product for each cell line.

"Aspen's financial backing, combined with its experienced and proven leadership team, positions it well for future success," said Kim P. Kamdar, Ph.D., Partner at Domain Associates, one of Aspen's seed investors. "Domain prides itself on investing in companies that can translate scientific research into innovative medicines and therapies that make a difference in people's lives. We clearly see Aspen as fitting into that category, as it is the only company using a patient's own cells for replacement therapy in Parkinson's disease."

About Aspen Neuroscience

Aspen Neuroscience Inc. is a development stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinson's disease. Aspen's therapies are based upon the scientific work of world-renowned stem cell scientist, Dr. Jeanne Loring, who has developed a novel method for autologous neuron replacement. For more information and important updates, please visithttp://www.aspenneuroscience.com.

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Exclusive: Two pigs engineered to have monkey cells born in China – New Scientist News

By adminDecember 13, 2019Embryonic Stem Cells

By Michael Le Page

Tang Hai

Pig-primate chimeras have been born live for the first time but died within a week. The two piglets, created by a team in China, looked normal although a small proportion of their cells were derived from cynomolgus monkeys.

This is the first report of full-term pig-monkey chimeras, says Tang Hai at the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing.

The ultimate aim of the work is to grow human organs in animals for transplantation. But the results show there is still a long way to go to achieve this, the team says.

Hai and his colleagues genetically modified cynomolgus monkey cells growing in culture so they produced a fluorescent protein called GFP. This enabled the researchers to track the cells and their descendents. They then derived embryonic stem cells from the modified cells and injected them into pig embryos five days after fertilisation.

More than 4000 embryos were implanted in sows. Ten piglets were born as a result, of which two were chimeras. All died within a week. In the chimeric piglets, multiple tissues including in the heart, liver, spleen, lung and skin partly consisted of monkey cells, but the proportion was low: between one in 1000 and one in 10,000.

It is unclear why the piglets died, says Hai, but because the non-chimeric pigs died as well, the team suspects it is to do with the IVF process rather than the chimerism. IVF doesnt work nearly as well in pigs as it does in humans and some other animals.

The team is now trying to create healthy animals with a higher proportion of monkey cells, says Hai. If that is successful, the next step would be to try to create pigs in which one organ is composed almost entirely of primate cells.

Something like this has already been achieved in rodents. In 2010, Hiromitsu Nakauchi, now at Stanford University in California, created mice with rat pancreases by genetically modifying the mice so their own cells couldnt develop into a pancreas.

In 2017, Juan Carlos Izpisua Belmontes team at the Salk Institute in California created pig-human chimeras, but only around one in 100,000 cells were human and, for ethical reasons, the embryos were only allowed to develop for a month. The concern is that a chimeras brain could be partly human.

This is why Hai and his team used monkey rather than human cells. But while the proportion of monkey cells in their chimeras is higher than the proportion of human cells in Belmontes chimeras, it is still very low.

Given the extremely low chimeric efficiency and the deaths of all the animals, I actually see this as fairly discouraging, says stem cell biologist Paul Knoepfler at the University of California, Davis.

He isnt convinced that it will ever be possible to grow organs suitable for transplantation by creating animal-human chimeras. However, it makes sense to continue researching this approach along with others such as tissue engineering, he says.

According to a July report in the Spanish newspaper El Pas, Belmontes team has now created human-monkey chimeras, in work carried out in China. The results have not yet been published.

While interspecies chimerism doesnt occur naturally, the bodies of animals including people can consist of a mix of cells. Mothers have cells from their children growing in many of their organs, for instance, a phenomenon called microchimerism.

Journal reference: Protein & Cell, DOI: 10.1007/s13238-019-00676-8

BioRestorative Therapies Receives Patent in Israel For Its Metabolic Program – Yahoo Finance

By adminDecember 13, 2019Embryonic Stem Cells

MELVILLE, N.Y., Dec. 12, 2019 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (BioRestorative or the Company) (BRTX), a life sciences company focused on stem cell-based therapies, today announced that the Israeli Patent Office has issued BioRestorative a Notice of Allowance on its patent application for a method of generating brown fat stem cells. This is the eighth patent issued, in the United States and other countries, for the Companys brown fat technology related to BioRestoratives metabolic program (ThermoStem Program).

Once issued in Israel, the final patent will allow for a method of isolating and differentiating a non-embryonic human brown adipose-derived stem cell into functional human brown adipocytes and a method of identifying compounds that modifies metabolic activity of human brown adipocytes. The technology is applicable for potential therapeutic uses for treating a wide range of degenerative and metabolic disorders, including but not limited to diabetes, obesity, hypertension and cardiac deficiency.

We continue to drive innovative and novel technology focusing on transformative therapies for our brown fat program, said Mark Weinreb, CEO of BioRestorative Therapies. We are pleased to add to our intellectual property library this recently issued patent by the Israeli Patent Office for our metabolic program to help power disruptive ways to treat metabolic disorders.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, whether the Company will be able to consummate the private placement and the satisfaction of closing conditions related to the private placement and those set forth in the Company's Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

Aspen Neuroscience launches with $6.5M seed funding to develop personalized and autologous cell therapy for Parkinson’s disease – TechStartups.com

By adminDecember 13, 2019Embryonic Stem Cells

Parkinsons disease is characterized by the loss of specific brain cells that make the chemical dopamine. Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems. Aspen is focusing on human pluripotent stem cells, cultured cells that can become any cell type in the human body. Many health technology startups are on the raise to cure this disease. At the forefront is Aspen Neuroscience, a healthtech startup developing first-of-its-kind personalized cell therapy for Parkinsons disease.

Aspen Neuroscience is a development stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinsons disease.

Today,Aspen Neuroscience announced its official launch with $6.5 million seed financingto develop the first autologous cell therapies for Parkinsons disease.The round was led by Domain Associates and Axon Ventures and including Alexandria Venture Investments, Arch Venture Partners, OrbiMed and others.

Aspens proprietary approach was developed by the companys co-founders, Jeanne F. Loring, Ph.D., Professor Emeritus and founding director of the Center for Regenerative Medicine at The Scripps Research Institute, and Andres Bratt-Leal, Ph.D., a former post-doctoral researcher in Dr. Lorings lab. The company was initially supported by Summit for Stem Cell, a founding partner and non-profit organization which provides a variety of services for people with Parkinsons disease. Aspen is led by industry veteran Howard J. Federoff, M.D., Ph.D., as Chief Executive Officer.

The companys research is specific to induced pluripotent stem cells (iPSCs), which it develops by taking a skin biopsy from a person with Parkinsons disease and turning the tissue into pluripotent stem cells using genetic engineering. Aspen then differentiates the pluripotent stem cells into dopamine-releasing neurons that can be transplanted into that same person (autologous), thereby restoring the types of neurons lost in Parkinsons disease.

As an autologous cell therapy for Parkinsons disease, Aspens treatment would eliminate the need for immunosuppression because the neurons are transplanted back into the same patient from which they were generated. The use of immunosuppression is necessary with currently available cell therapies for Parkinsons disease and when transplanting cells from one patient to another (allogeneic) to prevent rejection but can pre-dispose the patient to life-threatening complications including infection and add cost to the patient and health system. Aspen is the only company in the world offering an autologous neuron replacement therapy for Parkinsons disease.

Aspen encompasses a powerful executive leadership team including Dr. Federoff who, in addition to his leadership roles at the UC Irvine Health System, was the Executive Vice President for Health Sciences and the Executive Dean of Medicine at Georgetown University. Dr. Federoff also has significant biotech industry experience including co-founding MedGenesis Therapeutix and Brain Neurotherapy Bio, as well as leading the U.S. Parkinsons Disease Gene Therapy Study Group. The company is also proud to announce the addition of several experienced and well-known members to its leadership team including Edward Wirth, M.D., Ph.D., as Chief Medical Officer.

Dr. Wirth currently serves as the Chief Medical Ofcer for Lineage Cell Therapeutics where he oversees clinical development of its two therapeutic programs for spinal cord injuries and lung cancer. He received his M.D. and Ph.D. from the University of Florida in 1994 and remained to conduct postdoctoral research including leading the University of Florida team that performed the rst human embryonic spinal cord transplant in the U.S. Dr. Wirth went on to serve as the Medical Director for Regenerative Medicine at Geron Corporation where the worlds rst clinical trial of human embryonic stem cell (hESC)-derived product occurred which demonstrated initial clinical safety.

Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinsons disease, said Dr. Federoff. We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist.

Pig-Monkey Hybrid Chimeras Made By Scientists in China Dies! What is Chimera? Are There Human Chimeras? – LatestLY

By adminDecember 13, 2019Embryonic Stem Cells

Monkey Pig (Photo Credits: New Scientist)

Chinese scientists recently developed monkey-pig hybrids called chimera as a part of their research into the growth of human organs for transplantation in animals. Although they died within a week of being born, the piglets carried DNA from macaque monkeys in their heart, liver, spleen, lung and skin. The monkey-pigs were bred from more than 4,000 embryos that were implanted into a sow using IVF. Chimera is a single organism which carries cells with distinct genotypes. Animal chimeras are produced by merging multiple fertilised eggs. In this case, they contained DNA from individuals, a pig and a monkey. Human Chimeras also exist but are quite rare. They contain the cells of two or more individuals. Their bodies contain two different sets of DNA. Around 100 cases of chimerism have been recorded in the modern medical literature.First Monkey-Pig Chimeras Created by Chinese Scientists, Die Within a Week (See Picture)

While chimera is today used to refer to a hybrid variety of animals or to about an impossible thing, the word has a reference in Greek mythology. According to which, chimera is a fantastical beast made from different animals. It is named after a fire-breathing monster which had the head of a lion, the body of a goat and serpent's tail. Chimera is also the Greek term for a female goat.

To create pig-primate chimeras, Tang Hai, a researcher at the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing and his co-authors first grew cells from cynomolgus monkeys (Macaca fascicularis) in lab dishes. The altered the cell's DNA by inserting instructions to build a fluorescent protein that made the cells bright green in colour. These luminescent cells gave rise to radiant embryonic stem cells which were then injected into pig embryos. These glowing spotshelped researchers to track the monkey cells as the embryos grew into piglets in due time.

In total, 4,000 embryos received an injection of monkey cells. The pigs bore 10 piglets but only two of them grew into both pig and monkey cells. The team found monkey cells scattered throughout multiple organs, including the heart, liver, spleen, lungs and skin. It was that in each organ, between one in 1,000 and one in 10,000 cells turned out to be monkey cells which were they were 99 percent pigs.

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Pig-Monkey Hybrid Chimeras Made By Scientists in China Dies! What is Chimera? Are There Human Chimeras? - LatestLY