Stem Cell Clinic

Cell therapy firm in flurry of activity as hope nears for bone marrow patients – The Times of Israel

By Stem Cell Treatment

The excitement at Jerusalem-based Gamida Cell, a maker of cell and immune therapy technologies, is palpable.

The biotechnology company has started enrolling patients for a last-stage clinical trial for a drug it believes will help increase the success of bone marrow transplants in blood cancer patients, and help them better withstand the ordeal of the lifesaving procedure.

The patients are being enrolled in the US, Spain, The Netherlands and Singapore. Should the results of the trial, as hoped, be positive, that would lead to the launch of a commercially available product in 2020, Gamida Cells CEO Yael Margolin said in an interview with The Times of Israel.

We are at an exciting transition point, and moving from being a research and development firm, based in Israel, to an international commercial firm, said Margolin who has headed the company for the past 12 years in her sun-drenched office at the biotech firms headquarters in Jerusalem. We need to prepare to commercialize the product. We are now looking at various sites in Israel for a new manufacturing facility and looking to employ some 100 people. These workers will be added to the 40 already employed in Jerusalem.

Gamida Cells CEO, Dr. Yael Margolin (Courtesy)

Preliminary clinical data has already revealed that the risk of their leading product for blood cancers, NiCord, not meeting its targets in the Phase 3 trial, is low, added Margolin.

The drug has already received a breakthrough therapy designation by the US Food and Drug Administration (FDA). The designation is given to a drug that is meant to treat a serious or life-threatening condition, and where preliminary clinical evidence indicates that it may demonstrate a substantial improvement on at least one clinically significant target (endpoint) over other available therapies. The designation also entitles the company to get more and closer FDA guidance to help bring the treatment faster to patients.

The combination of the low clinical risk based on the previous trial results and the lower regulatory risk, because the drug is being developed in close collaboration with the FDA, has spurred the company into a flurry of activity that is aimed at scaling up its production facilities to get ready for the day NiCord hits the markets.

The company said last month it raised $40 million from investors including Novartis, which is already a major shareholder in the firm. The funds will support the ongoing Phase 3 stage for NiCord. The company also announced, on July 20, that it received a $3.5-million grant from the Israeli government that will support the further development of NiCord and other drugs that the company is developing, including therapies for sickle cell disease and for blood and solid cancers. Gamida has also appointed a new chief medical officer, Ronit Simantov, who will be based in the US.

The first market for our drug will be the US, Margolin said.

The Gamida Cell lab in Jerusalem where umbilical cord blood is stored in tanks, July 16, 2017. (Shoshanna Solomon/Times of Israel)

NiCord, which would be the first drug developed by Gamida to hit the market if the trial goes well is believed to increase the chances of a successful bone marrow transplantation process for patients who do not have a rapidly available, fully matched, bone marrow donor.

Today some high-risk blood cancers cannot be cured unless patients undergo a bone marrow graft. For that purpose, a perfect 100-percent match needs to be found, a process that in the US takes an average of three to four months, if the patient is lucky. Sometimes, no match is found.

There are 70,000 patients a year globally with blood cancers who need a bone marrow transplant, Margolin said. It is a rare condition. But for that transplant, you need a donor with full tissue matching. As many as 50% dont get to the transplant phase, because they havent found a matching donor in time.

Umbilical cord blood collected from newborn babies contains stem cells, which can be used to treat diseases. Today cord-blood banks around the world store the cord blood. It great advantage is that because it is so young, there is no need for a full tissue matching.

The big advantage with umbilical blood is that you dont need full tissue matching; a partial match is enough, Margolin continued. Most patients generally find at least one unit of cord blood that partially matches them.

Stem cells in a bag in Gamida Cells Jerusalem lab, July 16, 2017 (Shoshanna Solomon/Times of Israel)

The problem is that the quantity of cells in each unit is not huge, and it is the number of stem cells in the cord blood that is critical to the success of transplantation.

Our idea is to leverage the advantages of the cord blood and overcome the limitations of the cell number by applying our own platform technology, called NAM Technology, added Margolin. This technology allows us to take one unit of umbilical cord blood and expand the number of stem cells within it and enhance their performance.

Gamida Cell selects the stem cells from the unit and puts them in a culture together with a molecule called Nicotinamide (NAM) a form of Vitamin B3 and adds other ingredients. This culture, to which the firm holds intellectual property rights, increases the number of stem cells, and enhances their functionality, Margolin said.

The cells are then harvested from the culture, frozen in a small blood-bag in a final formulation that is ready for infusion, and then shipped to hospitals via couriers. Doctors thaw the product by the bedside of the patients and infuse the fluid into them.

From start to finish, our process takes three weeks, Margolin said. The average search for a bone marrow match takes three to four months.

The clinical trial underway is enrolling patients aged 16 years and older.

An earlier trial of the drug showed that patients transplanted with NiCord showed a more rapid engraftment the amount of time needed for the development of a minimal amount of white blood cells, or neutrophils, in the blood. That minimum amount indicates the patient is now less vulnerable to infections and bleeding following the transplant, and is an indication of success.

In the pilot phase clinical trials, the median time to neutrophil engraftment with NiCord was 11 days, compared to three to four weeks in patients who received standard umbilical cord blood. The results in a study conducted at Duke University also showed a lower rate of infection 22% vs 54%; and a lower duration of hospitalization compared to standard umbilical cord engraftment, Margolin said.

Now the company is enrolling patients for its larger, Phase 3 multi-national, randomized controlled registration study. And in February it said it had already transplanted its first patient, as part of the trial.

We hope to publish positive topline data from the Phase3 study in the first half of 2019 and launch the product on the market in 2020, she said.

Metal barrel with a frozen bag of umbilical cord stem cells ready for delivery from Gamida Cells Jerusalem lab, July 16, 2017. (Shoshanna Solomon/Times of Israel)

A metal barrel within which was a frozen bag of umbilical cord stem cells was waiting to be picked by a courier in the lobby of the Gamida Cell offices, ready to be thawed and injected into a patient somewhere around the world.

We have a sophisticated infrastructure that coordinates everything between the cord bank blood and our manufacturing site and the hospital where the patient is to be treated, Margolin continued. This infrastructure is 100% robust, but we plan to scale this up toward commercialization.

The $40 million in funds the company raised last month is expected to last until late 2019. After that, she added, all options are on the table: an IPO, or teaming up with a strategic partner, are both possibilities for the future.

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Cell therapy firm in flurry of activity as hope nears for bone marrow patients - The Times of Israel

US Scientists First Ever To Genetically Modify Human Embryos – Chronicle Day

By Embryonic Stem Cells

The United States seems to be in the running when it comes to the experiments with human DNA. However, the subject s still very much covered with a veil of secrets and questions we have no answers to.

It looks like that the MIT Technology Review published a news report last week concerning the experiments in which the aim was to create genetically modified human embryos. This research was led by Shoukhrat Mitalipov, director of the Oregon Health & Science Universitys Center for Embryonic Cell and Gene Therapy, but neither he or his unversity wanted to make any statements concerning the research.

The university press office did, however, release a statement which said: Results of the peer-reviewed study are expected to be published soon in a scientific journal. No further information will be provided before then. It is pretty clear that there will be no way to get the extra details on the research before the study is published in the journal as to avoid any speculation and possible issues.

Mitalipov has also refused to comment any of the questions or statements released, including the report that was published. He has also not confirmed not negated that he has seen the report.

In 2013 Mitalipov and his research team had their first breakthrough when they cloned human stem cells and reprogrammed them to go back to their embryonic state. In 2007, they have announced that they made a clone of monkey embryo and managed to extract the cells from it.

Although it is not certain, and at this time cannot be confirmed, it is suspected that the genes targeted in this particular research are the ones associated with inherited diseases. Arthur Caplan, a professor and founder of the division of bioethics at New York University Langone Medical Center, who was not involved in the research commented: Im not surprised that they were looking at genetic diseases to try and see if they could target them, because thats exactly where I think the future inevitably leads.

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US Scientists First Ever To Genetically Modify Human Embryos - Chronicle Day

Cell Design Labs Appoints Roger Sidhu, MD as Chief Medical Officer – GlobeNewswire (press release)

By Uncategorized

August 01, 2017 08:00 ET | Source: Cell Design Labs

EMERYVILLE, Calif., Aug. 01, 2017 (GLOBE NEWSWIRE) -- Cell Design Labs, Inc. today announced the appointment of Roger Sidhu, M.D., to the newly created position of Chief Medical Officer. Dr. Sidhu will be responsible for the design and development of Cell Design Labs clinical programs as well as providing insight into therapeutic applications for its proprietary CAR-T and adoptive T cell therapies (TCR-T) for the treatment of cancer and other complex diseases. In this new role, Dr. Sidhu will report to Brian Atwood, Chief Executive Officer of Cell Design Labs.

Roger brings extensive experience as he was a leader in the development of Amgens deep hematology/oncology portfolio, said Brian Atwood, Co-Founder, President and Chief Executive Officer of Cell Design Labs. His broad clinical knowledge and established track record will be instrumental in guiding the clinical development of our innovative cell therapies, particularly as we look toward the application of our proprietary synNotch and THROTTLE Switch technology platforms in liquid and solid tumors.

The science and vision of Cell Design Labs management team is quite compelling, said Dr. Sidhu. The opportunity to create next-generation customized cell therapies represents a new era in medicine as this powerful technology platform may play a major role in developing more effective and safer therapeutics for patients with cancer, autoimmune and infectious diseases.

Dr. Sidhu comes to Cell Design Labs from Amgen, where he most recently held the position of Global Product General Manager. In that role, he was responsible for strategy, development and commercialization of Amgens portfolio of early-stage immuno-oncology molecules with a focus on solid tumors. He directed programs across multiple platform technologies including CAR-T cell therapies, antibodies, vaccines and bispecific T-cell engagers (BiTEs). Prior to serving as Global Product General Manager, he held escalating positions within Amgens hematology/oncology department where he provided therapeutic area leadership for a portfolio of hematology/oncology therapeutics including Vectibix, Imlygic, Blincyto, Kyprolis, rilotumumab, trebaninib, Neupogen/Neulasta, Aranesp, Nplate and XGEVA. He has extensive experience leading global product specific partnerships and collaborations. Importantly, he led successful regulatory filings for oncology therapeutics resulting in marketing approval from both the U.S. Food and Drug Administration and the European Medicines Agency. He received his B.S. in Biochemistry from the University of Alberta in Alberta, Canada, his M.D. from Queens University in Ontario, Canada and completed a medical oncology fellowship at the British Columbia Cancer Agency in British Columbia, Canada. He is an author on numerous publications and presentations.

About Cell Design Labs, Inc. Cell Design Labs is a biotherapeutics company pioneering breakthrough science to develop disruptive cell-based therapies. Based on innovative research from Dr. Wendell Lims lab at UC San Francisco, Cell Design Labs leverages the power of the bodys immune system to develop smart, living therapies with the capability to treat our most challenging diseases with unprecedented power, precision, safety and durability. Using its proprietary technology platform for custom cell engineering, Cell Design Labs will develop its own portfolio of anticancer therapies as well as create partnerships with leading oncology companies. Initially focused on cancer, including both hematologic and solid tumors, this broad technology may also have applications in other complex diseases such as autoimmune and degenerative disorders. To learn more about Cell Design Labs, please visit our web site at: http://www.celldesignlabs.com.

synNotch and THROTTLE Switch are trademarks of Cell Design Labs, Inc.

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Cell Design Labs Appoints Roger Sidhu, MD as Chief Medical Officer - GlobeNewswire (press release)

New cancer therapy in clinical trial at Nebraska Medical Center has … – Omaha World-Herald

By Uncategorized

By the time scans showed that his B-cell non-Hodgkin lymphoma was coming back last fall, James Olson had run through many of his treatment options.

In early January, his doctor recommended that the Kansas City, Missouri, man travel to the Nebraska Medical Center in Omaha to see whether he was a candidate for a new type of therapy still in clinical trials.

In May, Olson, 69, received an infusion of his own immune cells, which had been removed from his body and modified to recognize and attack the cancer. Known as CAR-T or chimeric antigen receptor T-cell therapy, it represents a new way of targeting some cancers beyond that of traditional treatments.

About 10 weeks later, his scans are good and hes got the stamina to mow his lawn and do some house painting. Hes enjoying the little pleasures of life, even seasonal chores like installing a window air conditioning unit at his home.

His doctors at the medical center, meanwhile, hope theyll soon be able to offer CAR-T to more patients.

The trial that Olson participated in still is underway, and the medical center continues to accept new patients. Another version of the treatment has shown so much promise that its been fast-tracked by the Food and Drug Administration and could receive conditional approval late this fall. The medical center is set to participate in an expanded trial for that treatment, possibly as soon as this month.

Recently, an expert panel unanimously endorsed a version of CAR-T for children and young adults with recurring acute lymphoblastic leukemia. If the FDA clears the drug, medical center doctors hope to offer the treatment to young adults up to age 25. Theyd also like to see new trials with that drug or others for older adults with recurring ALL, as the leukemia is known.

These people need something else, and this may be it, said Dr. Matthew Lunning, a hematologist and oncologist with Nebraska Medicine.

The federal panels endorsement came at a time when a number of companies are racing to develop therapies based on the approach, which scientists first began to explore decades ago. If approved by the FDA, the Novartis product endorsed by the panel would become the first gene therapy approved in the United States. In addition to targeting relapsed lymphoma and leukemia, researchers also are beginning to study the therapy for solid tumors, the kind that start in organs or tissue. Leukemia and lymphoma are considered blood cancers.

Dr. Julie Vose, chief of hematology and oncology at Nebraska Medicine, said the therapy offers many bright spots in oncology.

We just have to learn how to use it in each specific disease and in each specific patient, she said, noting that the therapy is not a one-size-fits-all treatment as existing methods have been. We have to personalize that treatment.

But even with FDA approval, she said, the treatments potential side effects, which include fever and flu-like symptoms ranging from mild to extremely severe, mean it can only be done at specialized centers like the medical center. Indeed, one earlier trial was closed because some patients suffered serious complications.

Vose said trials in non-Hodgkin lymphoma all have produced good results, including the trial currently underway at the medical center and eight other sites nationwide.

Nationally, patients involved in the trial have had a complete remission rate of 60 percent after 30 days and 40 percent after 60 days. Officially, the condition is known as refractory diffuse large B-cell non-Hodgkin lymphoma, an aggressive disease that is among about 80 subtypes of non-Hodgkin lymphoma.

These were patients who had failed every other possible treatment, Vose said. And more than half had good response to the treatment.

Treatment for newly diagnosed non-Hodgkin lymphoma and acute lymphoblastic leukemia usually begins with chemotherapy. About 30 percent of non-Hodgkin lymphoma patients and roughly 40 percent of acute lymphoblastic leukemia patients relapse after chemo. The majority of those go on to have a blood or bone marrow transplant, from which about half will relapse.

Patients who relapse after the transplant, or those who arent candidates for a transplant, could be potential candidates for CAR-T therapy.

Unlike traditional gene therapy, however, CAR-T doesnt involve replacing disease-causing genes with healthy ones. Instead, technology is used to reprogram T cells, immune cells that normally help the body fight infection and cancer. In lymphoma patients, however, Vose said, T cells go haywire and dont properly fight cancer.

During treatment, the patients T cells are collected during an outpatient procedure and sent to a lab in California for processing. The patient then receives several days of intense chemotherapy. The modified T cells are returned to Omaha and put back into the patient, who is monitored at the hospital for seven to 10 days. The entire process takes about three weeks.

Olson, who drove to Omaha last week for a checkup, said he didnt really have any side effects from the procedure. I wondered if theyd given me a placebo, said Olson, who spent his working life in real estate and was diagnosed in 2010. He was well aware of the possible risks after reading and signing a 33-page consent form.

The next question, for patients and researchers alike, is how long the treatment will hold.

We dont know how long because this is such a new treatment, Vose said, but many of the patients (who) have been treated over the past year are doing well.

That question, like many in cancer treatment, comes with its own risks and benefits to balance. If half of the lymphoma is gone and it lasts years, Lunning said, thats better than a remission that lasts a month.

This report includes material from the Washington Post and Associated Press.

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New cancer therapy in clinical trial at Nebraska Medical Center has ... - Omaha World-Herald

ViaCyte Announces First Patients Implanted with PEC-Direct Islet Cell Replacement Therapy in International Clinical … – PR Newswire (press release)

By Uncategorized

The first cohort of type 1 diabetes patients is receiving multiple small-format cell-filled devices called sentinels in order to evaluate safety and implant viability. These sentinel units will be removed at specific time points and examined histologically to provide early insight into the progression of engraftment and maturation into pancreatic islet cells including insulin-producing beta cells. A second cohort of up to 40 patients is expected to begin enrolling later this year to evaluate both safety and efficacy. The primary efficacy measurement in the trial will be the clinically relevant production of insulin, as measured by the insulin biomarker C-peptide, in a patient population that has little to no ability to produce endogenous insulin at the time of enrollment. Other important endpoints will be evaluated including injectable insulin usage and the incidence of hypoglycemic events. ViaCyte's goal is to demonstrate early evidence of efficacy in the first half of 2018 and definitive efficacy 6 to 12 months later.

"Islet transplants have been used to successfully treat patients with unstable, high-risk type 1 diabetes, but the procedure has limitations, including a very limited supply of donor organs and challenges in obtaining reliable and consistent islet preparations," said trial investigator James Shapiro, MD, PhD, FRCSC, Director of the Clinical Islet Transplant Program, University of Alberta. "An effective stem cell-derived islet replacement therapy would solve these issues and has the potential to help a greater number of people."

"Patients with high-risk type 1 diabetes complications, such as hypoglycemia unawareness, are at constant risk of life-threatening low blood glucose," said Jeremy Pettus, MD, investigator in the clinical trial and Assistant Professor of Medicine at UC San Diego. "The PEC-Direct islet cell replacement therapy is designed to help patients with the most urgent medical need."

The PEC-Direct product candidate is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or severe hypoglycemic episodes. It is estimated that about 140,000 people in Canada and the US have such high-risk type 1 diabetes. In addition to providing an unlimited supply of cells for implantation, the PEC-Direct approach has other potential advantages relative to cadaver islet transplants such as delivering a more consistent product preparation under quality-controlled cGMP conditions, and a more straightforward and safe mode of delivery.

The clinical trial is being supported in part by JDRF, the leading global organization funding type 1 diabetes research. "JDRF remains dedicated to accelerating the delivery of beta cell replacement therapies to the T1D community, and we commend ViaCyte in its announcement of the first patients to be implanted with the PEC-Direct islet cell replacement therapy," said Derek Rapp, JDRF President and Chief Executive Officer. "JDRF is excited to support this clinical development given its potential to help those people with type 1 diabetes that need it the most those at high risk of life-threatening acute complications. JDRF and ViaCyte share a continuing commitment to realizing the potential of beta cell replacement strategies to deliver insulin independence without immune suppression for people living with type 1 diabetes, and ultimately, at JDRF we hope this will move us forward in fulfilling our vision of a world without type 1 diabetes."

"There are limited treatment options for patients with high-risk type 1 diabetes to manage life-threatening hypoglycemic episodes," said Paul Laikind, PhD, President and Chief Executive Officer of ViaCyte. "We believe that the PEC-Direct product candidate has the potential to transform the lives of these patients and we are excited to move closer to that goal with the initiation of clinical evaluation announced today. This also represents a step towards a broader application of the technology. We remain fully committed to developing a functional cure for all patients with insulin-requiring diabetes. To that end, we are hard at work on next-generation approaches as well, and expect the work with PEC-Direct to further advance our knowledge and drive progress."

In addition to JDRF, the California Institute for Regenerative Medicine (CIRM)'s Alpha Clinic, the Sanford Stem Cell Clinical Center, the JDRF Canadian Clinical Trials Network (CCTN), the Stem Cell Network, and Alberta Innovates Health Solutions (AIHS) are all providing support for the trial.

About the PEC-Direct Product Candidate The PEC-Direct product candidate delivers stem cell-derived pancreatic progenitor cells, called PEC-01 cells, in a device designed to allow direct vascularization of the cells in the device. After implantation, these cells are expected to become mature human islet tissue including well-regulated beta cells producing insulin on demand. The direct vascularization of the implanted cells is expected to allow for robust and consistent engraftment but will necessitate the use of maintenance immune suppression therapy.

About ViaCyte ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte's product candidates are based on the derivation of pancreatic progenitor cells from stem cells, which are then implanted in a durable and retrievable cell delivery device. Once implanted and matured, these cells are designed to secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two products in clinical development. The PEC-Direct product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is headquartered in San Diego, California. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. For more information on ViaCyte, please visit http://www.viacyte.com and connect with ViaCyte on Twitter and Facebook.

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ViaCyte Announces First Patients Implanted with PEC-Direct Islet Cell Replacement Therapy in International Clinical ... - PR Newswire (press release)

3D printed brain-like tissue made from stem cells offers hope to address neurological disorders – Genetic Literacy Project

By Embryonic Stem Cells

Scientists in Australia have used a 3D printer to create nerve cells found in the brain using a special bio-ink made from stem cells.

The research takes us a step closer to making replacement brain tissue derived from a patients own skin or blood cells to help treat conditions such as brain injury, Parkinsons disease, epilepsy and schizophrenia.

The bio-ink is made of human induced pluripotent stem cells (iPSC), which have the same power as embryonic stem cells to turn into any cell in the body, and possibly form replacement body tissues and even whole organs.

3D printing with bio-ink (ABC News)

[Jeremy Crookfrom the University of Wollongong stated]many neuropsychiatric disorders result from an imbalance of key chemicals called neurotransmittersFor example, he said, defective serotonin and GABA-producing nerve cells are implicated in schizophrenia and epilepsy[Thus] the team used 3D printing to make neurones involved in producing GABA and serotonin.

Apart from providing customized transplants, 3D printed tissue could be useful for medical research.

For example, tissue from a patient with epilepsy or schizophrenia could be created, specifically to study their particular version of the condition.

You can compare how neuronal networks form differently compared to healthy patient, said Dr Crook.

[Read the full study here]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Scientists create 3D-printed brain-like tissue from stem cells

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3D printed brain-like tissue made from stem cells offers hope to address neurological disorders - Genetic Literacy Project

Controversial milestone: Scientists genetically modify human embryos for first time, reports say – The San Diego Union-Tribune

By Embryonic Stem Cells

A team of researchers that includes a scientist from the Salk Institute in La Jolla has created the first genetically modified human embryos, the MIT Technology Review reported this week.

If the achievement is true the scientists in question have neither confirmed nor disputed the account it could mark a milestone in preventing transmission of genetic diseases instead of just treating them.

It would also rev up debate about the safety and ethics of genetically changing human beings, including what laws exist to safeguard patients and what constitutes a medically legitimate genetic modification.

The technology could be used to alter people for nonmedical purposes such as making them taller, giving them a specific eye shape or switching out their black hair for a shade of blonde decisions that could be seen as fundamentally upending the definition of human nature.

The Technology Review story said the scientists harnessed the gene-editing method called CRISPR, a milestone in its own right, to modify one-celled embryos and allow them to develop for a few days. Other news organizations have published their own articles about this purported accomplishment, including the well-respected biomedical website Stat.

Prominent biologist Shoukhrat Mitalipov of Oregon Health & Science University was the lead researcher on the study, according to the Technology Review and Stat stories. Both reports said he declined to comment.

Results of the peer-reviewed study are expected to be published soon in a scientific journal, Oregon Health & Science spokesman Erik Robinson said Thursday. He declined to specify what the study discovered.

The Technology Review story also said Jun Wu of the Salk Institute for Biological Studies took part in the research. On Thursday, the institute declined to discuss the study.

Mitalipov gained fame in 2013 for spearheading development of the first human embryonic stem cells genetically matched to specific living individuals. The method he and some colleagues employed, called somatic cell nuclear transfer, was originally used two decades ago to create Dolly the cloned sheep.

Those researchers had taken a nucleus from a donor cell in a sheep and transferred it into a sheep egg cell that had had its own nucleus removed. The combination cell acted like a normal fertilized egg, producing Dolly. That sheep had the DNA of the donor cell, so it was a nearly exact clone of the sheep where the donor cell was taken from.

Growing a creature in this way is called reproductive cloning, and the U.S. government bans such procedures on people. Mitalipov and colleagues performed what is called therapeutic cloning: They used the process to cultivate human embryonic stem cells, which are likewise genetically matched to the donor nucleus.

In theory, these stem cells could be grown into replacement tissues to repair disease or injury in the person with the matching DNA. Genetically matching the stem cells to a particular patient lowers the risk that tissue transplants would be rejected by the persons immune system.

Wu and other Salk researchers in the lab of Juan Carlos Izpisa Belmonte have collaborated with Mitalipov to explore somatic cell nuclear transfer as a therapy for mitochondrial diseases. Mitochondria are organelles that make most of the energy cells use and perform other vital functions. They carry their own DNA.

The scientists generated human stem cells in the lab, repaired mitochondrial defects and found that they were able to restore certain desired functions in cells.

They took human skin cells and inserted their nuclei into human egg cells with healthy mitochondria that had their own nuclei removed. Those manipulated egg cells were then grown until they produced embryonic stem cells, free of the defective mitochondria.

The United Kingdom has approved a method that resembles reproductive cloning to prevent inheritance of mitochondrial diseases. This process involves replacing the nucleus of an egg cell from a donor with healthy mitochondria with that from the egg cell of the mother-to-be with diseased mitochondria.

Whether the reports this week about genetically modified human embryos are true, the capability of genetically engineering human embryos is fast approaching, said a bioethicist and a stem cell researcher who have examined the issue.

But having the capability doesnt mean it should be done, said Michael Kalichman, co-founding director of the the Center for Ethics in Science and Technology at UC San Diego.

Kalichman said society isnt ready for genetically modifying humans, and that its time for the public to start paying attention to what has been considered a futuristic scientific issue.

The strongest argument for genetic modification is to stop diseases, he said. The strongest argument against the technology is that it might cause unanticipated problems.

Paul Knoepfler, a stem cell researcher at UC Davis, said no matter how much effort is spent to ensure patient safety, there are no guarantees.

The bottom line is that well never really know until someone tries it, Knoepfler said. Potential harm might not emerge until adulthood or even until the genetically altered people have their own children, he added.

The other big thing is, I am not really convinced we can draw a clear line between doing this for only medical purposes versus (cosmetic) traits, he said.

Finally, its not clear why genetically editing human embryos would even be needed to prevent transmission of a genetic disease, Knoepfler said.

We already have an existing technology which is basically embryo screening, he said. Multiple embryos can be generated through in vitro fertilization to find one that doesnt have the disease.

That would be much safer than actually doing an edit, he said.

Stem cells could treat mitochondrial disease

Oregon scientists make embryos with 2 women, 1 man

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Controversial milestone: Scientists genetically modify human embryos for first time, reports say - The San Diego Union-Tribune

Scientists create 3D-printed brain-like tissue from stem cells – ABC Online

By Embryonic Stem Cells

Scientists in Australia have used a 3D printer to create nerve cells found in the brain using a special bio-ink made from stem cells.

The research takes us a step closer to making replacement brain tissue derived from a patient's own skin or blood cells to help treat conditions such as brain injury, Parkinson's disease, epilepsy and schizophrenia.

The bio-ink is made of human induced pluripotent stem cells (iPSC), which have the same power as embryonic stem cells to turn into any cell in the body, and possibly form replacement body tissues and even whole organs.

Jeremy Crook, who led the research, said the ability to customise brain tissue from a person's own body tissue was better for transplantation.

"That circumvents issues of immune rejection, which is common in organ transplantation," said Dr Crook, from the University of Wollongong and ARC Centre of Excellence for Electromaterials Science.

"It's personalised medicine."

Dr Crook said many neuropsychiatric disorders result from an imbalance of key chemicals called neurotransmitters, which are produced by specific nerve cells in the brain.

For example, he said, defective serotonin and GABA-producing nerve cells are implicated in schizophrenia and epilepsy while defective dopamine-producing cells are implicated in Parkinson's disease.

The team used 3D printing to make neurones involved in producing GABA and serotonin, as well as support cells called neuroglia, they reported in the journal Advanced Healthcare Material.

In the future, they plan to print neurones that produce dopamine.

"We might want to make a tissue that specifically generates that neurotransmitter for grafting into the brain of a Parkinson's patient," said Dr Crook.

"That's absolutely achievable."

To make the neurones, Dr Crook and colleagues used their bio-ink to print layers of a hatched pattern to create a 5 millimetre-sized cube.

They then "crosslinked" the cube into a firm jelly-like substance.

Growth factors and nutrients were then fed into the holes of this spongey "scaffold", encouraging the stem cells to grow and turn into neurons and support cells, linking up to form tissue.

Waste was also removed via the holes in the scaffold.

Dr Crook said once scaled up, blood vessels would be needed, but small transplants could be theoretically possible using the tissue developed so far.

Tissue engineer Makoto Nakamura from Toyama University in Japan said the study was "very impressive".

"This article indicates the good feasibility of 3D bioprinting with human iPS cells to engineer neural tissues," said Professor Nakamura, who recently wrote an overview on the use of 3D bioprinting in the journal Tissue Engineering.

But he said there were also risks with the technology.

A close up of the 'scaffold' made of 3D-printed induced pluripotent stem cells (iPSCs)

(Supplied: Gu et al/Advanced Healthcare Materials)

A close up of the 'scaffold' made of 3D-printed induced pluripotent stem cells (iPSCs)

Supplied: Gu et al/Advanced Healthcare Materials

One of the challenges of using iPSCs is that, like embryonic stem cells, they have the potential to develop into teratomas disturbing looking tumours that contain more than one type of tissue type (think toenails growing in brain tissue, or teeth growing in ovary tissue).

According to Professor Nakamura, it would be important to ensure all the stem cells had turned into nerve cells in the final transplanted material.

"Undesired tissue may grow if even only one immature [stem] cell contaminates [the tissue to be transplanted]," he said.

Dr Crook said the team was currently carrying out animal experiments to test if teratomas developed from the 3D printed nerve cells.

While this is a first step towards 3D printing of whole organs, Dr Crook said a whole functioning brain would be a much more complex task.

"That's a whole different scale. The tissue we print is uniform, and not made up of different regions like a brain," said Dr Crook.

Still, it is a goal the researchers are heading towards.

"We would like to get as close as possible to replicating the function of the brain on the bench," said research team member Professor Gordon Wallace.

Apart from providing customised transplants, 3D printed tissue could be useful for medical research.

For example, tissue from a patient with epilepsy or schizophrenia could be created, specifically to study their particular version of the condition.

"You can compare how neuronal networks form differently compared to healthy patient," said Dr Crook.

And the tissue could also be used to screen for effective drugs or electrical stimulation treatments.

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Stem cell research: the debate continues to rage – CosmicNovo.com (Science and Technology)

By Embryonic Stem Cells

The list of medical or scientific endeavours mired in controversy is fairly short, but stem cell research and related therapy are some of the most contentious issues in modern science. Simply, stem cell therapy involves the use of stem cells to treat or prevent a disease or condition, a form of this type of treatment involves bone marrow transplant which is a relatively common operation. Whilst this may not strike you as something worthy of debate, it is because it is the further research in stem cell therapy that has become a battleground of ideology and discussion.

There is research and case studies showing that stem cell therapy involving cells from the umbilical cord blood of infants as well embryonic stem cells from human embryos. Although the former is fairly innocuous, is the latter, which requires a human embryo that has caused controversy, as to harvest them, you must destroy the embryo.

Understandably, there is a lot of opposition to the use of human embryonic stem cells in research, often times based on a range of philosophical, moral, or religious objections, with most protesters worried of cloning embryos just to harvest these cells. Theology, philosophy and morality aside, the medical possibilities of embryonic stem cells are almost limitless.

Doctors have explained that due to the nature of these cells, they are more flexible and can be put to a far greater range of uses than other more conventional stem cells. They pertain that these cells could help treat an incredibly high amount of diseases and illnesses including but not limited to neurodegenerative diseases and conditions such as diabetes and heart disease.

Of course, this only adds to the mounting debate surrounding the use of these cells, further driving questions from a moral and philosophical viewpoint as to whether or not it is ethical to be using embryonic stem cells, despite the purported benefits. Although research continues into the use of these specific cells, and governments grapple with potential legal and medical ramifications, it is important to realize that there are several other stem cell opportunities that do not require the same controversial source.

Although there has been blowback on other forms of research in the sector namely the use of umbilical blood the use of bone marrow transplants and other such alternative continue to save lives daily. However, until society catches up with science and medicine, there will be a continued debate as to the ethics and morality of this type of research, its applications, and what it could open the door for.

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Stem cell research: the debate continues to rage - CosmicNovo.com (Science and Technology)

How to Become a Regenerative Stem Cell Doctor

By Stem Cell Doctors

Overview

A new frontier in medicine, regenerative medicine or stem cell therapy involves the study of or the application of stem cells as a form of healing. Stem cells are naturally reproduced in the body therefore they are a renewable source of medicine with enormous potential. Still a relatively new area of scientific study, it is unknown just how far and wide reaching the healing capabilities of this type of therapy will be, but the potential seems quite limitless. A medical student choosing to specialize in this field of study will likely witness and participate in many new and exciting developments as this type of medicine evolves.

What is a Regenerative Stem Cell Doctor

A regenerative stem cell doctor is a specialist who uses stem cell therapy to treat patients. As this is still a comparatively new form of medicine, this type of doctor will likely be involved in research or clinical trials, discovering new treatments and their effects as well as preventative approaches based on cellular technology to treat currently unmanageable human diseases.

Stem cells generally fall under three categories:

A stem cell doctor may use this type of medical treatment with patients who suffer from autoimmune deficiencies or genetic blood diseases. It's also typical for stem cell doctors to focus on pharmaceutical product development in areas of systemic diseases, cardiac and vascular diseases, or orthopedic diseases of the spine or bone marrow transplantation outcomes.

A regenerative stem cell doctor may also be involved in cancer research through the use of stem cell medicine as an alternative to damaging treatments such as radiation and chemotherapy. Stem cell therapy is also currently being used to treat bone, skin and corneal diseases and injuries through tissue grafting.

Educational Requirements for Becoming a Regenerative Stem Cell Doctor

A doctoral or professional degree is the educational requirement for a Regenerative or Stem Cell Doctor

Falling under the category of medical scientist, a student wishing to become involved in the exciting field of cell-based regenerative medicine will find themselves at the forefront of the ground breaking evolution of medicine. Of course, the educational path to such an esteemed profession will require lifelong dedication to learning and continued research and may even require dual graduate degrees depending upon the student's individual goals; as well as a commitment to meeting challenges and the fortitude to deal with setbacks as this type of medicine evolves.

Step-by-Step Educational Path to Becoming a Regenerative Stem Cell Doctor

Bachelor's Degree

Holding an undergraduate degree from an accredited college is necessary in order to procure a place in medical school or to be accepted into a doctoral program. For those whose sights are set on eventually becoming a medical scientist and working in the field of regenerative stem cell medicine, it is ideal to begin with a strong background in biology, chemistry and biophysics. Majoring in life science, biology, microbiology or pre-med is a good starting point as well as taking writing classes as this field of study will require reporting, journaling and possibly public speaking at some point. Future medical students will need to take The Medical College Admissions Test (MCAT).

Volunteer

Volunteering is always a good idea and will make an impressive addition to any resume or application submitted towards attaining the candidate professional goals. Students interested in stem cell therapy may find volunteer opportunities at universities or laboratory environments as research assistants.

Internship

Students will gain hands on experience through an internship by working under the supervision of experienced stem cell researchers, assisting with current investigations, writing reports and sharpening other required skills in preparation for entering medical school or a doctoral program. Several internships can be applied toward college credit hours.

Masters Degree

A Master of Science Degree or a master's degree with a focusin stem cell biology and regenerative medicine will prepare the way for entry into medical or PhD programs. During this course of study the candidate will acquire an in depth understanding of how the body can naturally repair and restore damaged cells, tissues and organs through courses in cutting-edge biomedical sciences such as molecular, cellular, and developmental biology. These master's programs will also provide hands-on laboratory experience working with stem cells.

Doctoral Degree

Having a PhD in a biological sciences field is perhaps the final stepping stone to qualify for positions at the forefront of the exciting field of cell-based regenerative medicine. One must also obtain a doctorate in medicine if their intention is to administer stem cell treatments. Some schools offer dual degree programs and the candidate may pursue both MD and PhD degrees concurrently.

At this point the candidate learning how to become a stem cell doctor will evolve from education to application through translational science as they study and develop new therapies based on stem cell behavior. Future stem cell doctors will receive specialized training in subjects such as genetics and developmental biology, participate in laboratory-based stem cell courses, take advanced courses in stem cell biology and regenerative medicine, and gain experience through clinical rotations as well as producing original research in the form of a thesis or dissertation.

It is also typical during the doctoral degree program that a future doctor will focus on specialties such as law, business and/or engineering pertaining to stem cell research or focus on areas more relevant to physicians and surgeons.

Licensing

Medical students are required to pass the United States Medical License Exams (USMLE) and complete residency training as well as acquiring the appropriate state(s) licenses in order to practice medicine.

Post Graduate Residency

After becoming a licensed doctor, in order to practice medicine it is necessary to complete a residency program in the area of their chosen specialty. Doctors who wish to work in stem cell based therapies will most likely complete a residency in internal medicine and may want to focus on developing skills in oncology or hematology. However, research involving stem cell medicine bleeds over into many specialty domains.

Fellowship

Post-doctoral education and training may entail working with researchers and staff on projects involving gene therapy, organ engineering or microbial studies. The American Academy of Anti-Aging Medicine (A4M) is at the forefront of this area of education by being the world's first school to offer a Stem Cell Fellowship Program. Requirements include 5 modules of training and experience as well as completing the A4M Board Certification. More programs for this type of fellowship are being designed and implemented as awareness rises, advances continue, and more and more doctors and students are in need of resources specific to stem cell education and application.

Continuing Education

A select few graduates, post doctoral fellows and clinical fellows may be selected to participate in curriculum intended to develop leadership skills for applicants with the potential to become future independent scientists in stem cell research. The educational requirements for a future in stem cell medicine are never ending as the potential for new therapies continue to grow and evolve and new and exciting discoveries are made.

Practicing physicians and surgeons who specialize in stem cell therapies should become certified by The American Board of Stem Cell Medicine and Surgery (ABSCMS). Visit their website for more information.

Understanding the Career Path

Work Environments

The tentacles of stem cell research are far reaching. There are worldwide opportunities for professionals in this field and as new developments occur and are found to be effective and receive government approval, the doors of opportunity will continue to open. Most regenerative stem cell doctors will work full time in office or laboratory environments working with samples, studying data and creating or studying reports.

The U.S. Department of Labor lists the top industries employing medical scientistsincluding

Many practicing physicians perform stem cell related therapies that have not yet been approved by the FDA. Below are a few scenarios in which a stem cell scientist or stem cell doctor may find employment.

Universities

Opportunities in stem cell research and development abound and someone applying for these positions will find many opportunities in universities to include laboratory research as well as academic positions. A typical day may include working on the development of new treatments, working with samples, recording detailed reports involving studies and results, as well as writing and submitting grant proposals to gain additional funding for programs. Academic positions may include classroom instruction through texts, seminars or hands on training with students or writing publications regarding new discoveries as stem cell research uncovers new and exciting possibilities.

Hospitals

Doctors or medical scientists working in hospitals or hospital laboratories may work with individuals or groups of patients, administering treatments and monitoring the results during clinical trials. Surgeons specializing in stem cell therapies may work in hospitals with patients suffering from very specific conditions such as cancer patients and those with blood diseases. They will be involved in procedures such as bone marrow transplantation or the implantation of stem cells into the body to treat blood disorders such as lymphoma, leukemia, or sickle cell anemia. They may also perform surgeries involving tissue grafting for skin or corneal diseases and injuries.

Pharmaceutical Companies

Pharmaceutical companies are becoming more and more involved with stem cells as drug therapies as new experimental avenues are opened. Stem cell specialists may work with these companies combining stem cells with other pharmaceutical compounds in experimentation processes for the development of new therapeutics.

Salary Expectations

The median annual salary a stem cell doctor (medical scientists, except epidemiologists) can expect to earn, according to the U.S. Bureau of Labor Statistics, was $76,980 as of May 2012 with the highest ranking positions being compensated at over $146,000 and the lower 10% bringing in less than $42,000 annually. O Net reports the median wage (2014) to be equivalent to $38.43 hourly or $79,930 annually. This field of employment is expected to grow by 13% between 2012 and 2022.

Growth is expected to be driven by natural causes such as population growth and the continued spreading of infectious diseases which provide an ever increasing need for development in this field of medicine which can improve overall human health and create new cures for illnesses.

While becoming a regenerative or stem cell doctor isn't specific to any one medical specialty at this point, an interest in this field leaves many options for the beginning medical student. It's due to the relatively new nature of this field of study that candidates have several career options to explore. This profession has the potential to change lives, discover cures and provide higher quality medical care for billions of people living across the globe.

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How to Become a Regenerative Stem Cell Doctor

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