Stem Cell Clinic

Cambridge Enterprise and Qkine to drive stem cell and regenerative medicine research – Business Weekly

By Stem Cell Medicine

Cambridge Enterprise, the commercialisation arm of the University of Cambridge, and specialist growth factor manufacturer Qkine Ltd have signed a key licensing deal for Activin A production technology.

Qkine is a recent spin-out from the university. The newly-licensed methodology, which was developed by one of the companys founders Dr Marko Hyvnen, will be used to manufacture proteins utilised for control of stem cell growth and differentiation.

Growth factors are proteins that transmit signals from one cell to another in higher organisms, orchestrating organisation of the developing embryo and regulating biological functions and repair processes in adults. Activin A, and others from its family of proteins, are essential ingredients used by stem cell scientists to mimic the environment in the human body. They allow carefully synchronised messages to be sent to stem cells, effectively telling them to turn into the desired cell type.

With exponential growth in the study of stem cells for disease modelling, drug screening, precision medicine and development of new therapeutics the need for high quality reagents for fine control of stem cell cultures is ever increasing.

Growing demand for Activin A and related growth factors and an opportunity to use protein engineering techniques to optimise these growth factors motivated Hyvnen and co-founder Dr Catherine Onley, a translational scientist, to start Qkine. Its mission is to produce high quality bioactive proteins for stem cell researchers and the regenerative medicine industry.

Hyvnen said: I have been providing growth factors to the Cambridge stem cell community for almost a decade.

Demand is growing from labs outside Cambridge and forming Qkine will allow us to focus on producing the highest quality cytokines for these scientists and establish a unique UK-based supplier of one of the enabling technologies for regenerative medicine, one of the priority areas for British manufacturing recently identified by the Government.

Qkine co-founders Dr Marko Hyvnen (picture courtesy - Chris Green) and Dr Catherine Onley (picture courtesy Suzi Ovens)

Qkine was awarded a Cambridge Enterprise Pathfinder investment in December 2016 to facilitate the founding of the company. Qkine started operation as an embedded company at the Department of Biochemistry in April 2017.

Dr Iain Thomas, head of Life Sciences at Cambridge Enterprise, said: Qkine is a great example of how opportunities are incubated in the University until the commercial time is right.

We are delighted that Qkine is taking this technology into the stem cell and regenerative medicine markets both of which are important and rapidly growing.

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Cambridge Enterprise and Qkine to drive stem cell and regenerative medicine research - Business Weekly

Doubts raised about CRISPR gene-editing study in human embryos – Nature.com

By Embryonic Stem Cells

Doubts have surfaced about a landmark paper claiming that human embryos were cleared of a deadly mutation using genome editing. In an article1 posted to the bioRxiv preprint server on 28 August, a team of prominent stem-cell scientists and geneticists question whether the mutation was actually fixed.

The 2 August Nature paper2, led by reproductive biologist Shoukhrat Mitalipov at the Oregon Health and Science University in Portland, described experiments in dozens of embryos to correct a mutation that causes a heart condition called hypertrophic cardiomyopathy.

In contrast to previous human-embryo editing studies, Mitalipovs team reported a high success rate at correcting a disease-causing mutation in a gene. The team claimed that the CRISPRCas9 genome editing tool was able to replace a mutant version of the MYBPC3 gene carried by sperm with a normal copy from the egg cell, yielding an embryo with two normal copies. Mitalipovs team also introduced a healthy version of the gene along with the CRISPR machinery, but they found that the corrected embryos had shunned it for the maternal version.

But there is reason to doubt whether this really occurred, reports a team led by Dieter Egli, a stem-cell scientist at Columbia University in New York City, and Maria Jasin, a developmental biologist at Memorial Sloan Kettering Cancer Center in New York City. George Church, a geneticist at Harvard Medical School in Boston, Massachusetts, is another co-author.

In their bioRxiv paper, Egli and Jasin and their co-authors say that there is no plausible biological mechanism to explain how a genetic mutation in sperm could be corrected based on the eggs version of the gene. More likely, they say, Mitalipovs team failed to actually fix the mutation and were misled into thinking they had by using an inadequate genetics assay. Egli and Jasin declined to comment because they say they have submitted their article to Nature.

The critique levelled by Egli et al. offers no new results but instead relies on alternative explanations of our results based on pure speculation, Mitalipov said in a statement.

But other scientists contacted by Nature's news team shared the Egli team's concerns. (Natures news team is editorially independent of its journal team.) Reproductive biologist Anthony Perry at the University of Bath, UK, says that after fertilization, the genomes of the egg and sperm reside at opposite ends of the egg cell, and each is enshrouded in a membrane for several hours. This fact, Perry says, would make it difficult for CRISPR-Cas9 to fix the sperms mutation based on the eggs version of the gene, using a process called homologous recombination. Its very difficult to conceive how recombination can occur between parental genomes across these huge cellular distances, he says.

Egli and Jasin raise that issue in their paper. They suggest that Mitalipovs team was misled into believing that they had corrected the mutation by relying on a genetic assay that was unable to detect a far likelier outcome of the genome-editing experiment: that CRISPR had instead introduced a large deletion in the paternal gene that was not picked up by their genetic assay. The Cas9 enzyme breaks DNA strands, and cells can attempt to repair the damage by haphazardly stitching the genome together, often resulting in missing or extra DNA letters.

That explanation makes sense, says Gatan Burgio, a geneticist at the Australian National University in Canberra. In my view Egli et al. convincingly provided a series of compelling arguments explaining that the correction of the deleterious mutation by self repair is unlikely to have occurred.

Another possibility Eglis team raise is that the embryos were produced without a genetic contribution from sperm, a process known as parthenogenesis. Mitalipovs team showed that the paternal genome was present in only 2 out of the 6 embryonic stem cell lines they made from gene-edited embryos.

Robin Lovell-Badge, a developmental biologist at the Francis Crick Institute in London, says that it is possible that there is a novel or unsuspected biological mechanism at work in the very early human embryo that could explain how Mitalipovs team corrected the embryos genomes in the manner claimed. He would first like to hear from Mitalipov before passing judgement. It simply says that we need to know more, not that the work is unimportant, Lovell-Badge says of Egli and Jasins paper.

In the statement, Mitalipovs said his team stands by their results. We will respond to their critiques point by point in the form of a formal peer-reviewed response in a matter of weeks.

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Doubts raised about CRISPR gene-editing study in human embryos - Nature.com

Study shows human stem cells restore mobility in Parkinson’s monkeys – Borneo Bulletin Online

By Embryonic Stem Cells

| Marlowe Hood |

PARIS (AFP) Lab monkeys with Parkinsons symptoms regained significant mobility after neurons made from human stem cells were inserted into their brains, researchers reported Wednesday in a study hailed as groundbreaking.

The promising results were presented as the last step before human clinical trials, perhaps as early as next year, the studys senior author, Jun Takahashi, a professor at Kyoto University, told AFP.

Parkinsons is a degenerative disease that erodes motor functions. Typical symptoms include shaking, rigidity and difficulty walking. In advanced stages, depression, anxiety and dementia are also common.

Worldwide, about 10 million people are afflicted with the disease, according to the Parkinsons Disease Foundation.

Earlier experiments had shown improvements in patients treated with stem cells taken from human foetal tissue and likewise coaxed into the dopamine-producing brain cells that are attacked by Parkinsons.

Dopamine is a naturally occurring chemical that plays several key roles in the brain and body.

But the use of foetal tissue is fraught with practical and ethical problems.

So Takahashi and his colleagues, in a medical first, substituted so-called induced pluripotent stem cells (iPSCs), which can be easily made from human skin or blood. Within a year, some monkeys who had could barely stand up gradually recovered mobility.

They became more active, moving more rapidly and more smoothly, Takahashi said by email. Animals that had taken to just sitting start walking around in the cage.

These findings are strong evidence that human iPSC-derived dopaminergic neurons can be clinically applicable to treat Parkinsons patients, he said.

Experts not involved in the research described the results as encouraging.

The treatment, if proven viable, has the potential to reverse Parkinsons by replacing the dopamine cells that have been lost a groundbreaking feat, said David Dexter, deputy research director at Parkinsons UK.

Not only did the new cells survive but they also integrated with the existing neuronal network, he said.

Neurons made from foetal tissue grafted into brains have been known to survive for more than a decade, and the researchers said they expected those derived from iPSCs to last just as long.

Tilo Kunath, Parkinsons Senior Research Fellow at the University of Edinburgh, said the outcome was extremely promising, and highlighted the advantage of avoiding stem cells extracted from human foetal tissue.

It means that this therapy can be used in any country worldwide, including Ireland and most of South America, where medical use of human embryonic stem cells is banned.

The results, reported in the journal Nature, were not the same for the dozen monkeys in the experiment, each of which received donor neurons from a different person.

Some were made with cells from healthy donors, while others were made from Parkinsons disease patients, said lead author Tetsuhiro Kikuchi, also from Kyoto University.

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Study shows human stem cells restore mobility in Parkinson's monkeys - Borneo Bulletin Online

FDA Cracks Down On Stem-Cell Clinics Selling Unapproved Treatments – NPR

By Stem Cell Treatment

Adult stem cells can be extracted from human fat. Patrick T. Fallon /The Washington Post/Getty Images hide caption

Adult stem cells can be extracted from human fat.

The Food and Drug Administration is cracking down on "unscrupulous" clinics selling unproven and potentially dangerous treatments involving stem cells.

Hundreds of clinics around the country have started selling stem cell therapies that supposedly use stem cells but have not been approved as safe and effective by the FDA, according to the agency.

"There are a small number of unscrupulous actors who have seized on the clinical promise of regenerative medicine, while exploiting the uncertainty, in order to make deceptive, and sometimes corrupt assurances to patients based on unproven and, in some cases, dangerously dubious products," FDA Commissioner Scott Gottlieb said in a statement Monday.

The FDA has taken action against clinics in California and Florida.

The agency sent a warning letter to the US Stem Cell Clinic of Sunrise, Fla., and its chief scientific officer, Kristin Comella, for "marketing stem cell products without FDA approval and significant deviations from current good manufacturing practice requirements."

The clinic is one of many around the country that claim to use stem cells derived from a person's own fat to treat a variety of conditions, including Parkinson's disease, amyotrophic lateral sclerosis (ALS), and lung and heart diseases, the FDA says.

The Florida clinic had been previously linked to several cases of blindness caused by attempts to use fat stem cells to treat macular degeneration.

The FDA also said it has taken "decisive action" to "prevent the use of a potentially dangerous and unproven treatment" offered by StemImmune Inc. of San Diego, Calif., and administered to patients at California Stem Cell Treatment Centers in Rancho Mirage and Beverly Hills, Calif.

As part of that action, the U.S. Marshals Service seized five vials of live vaccinia virus vaccine that is supposed to be reserved for people at high risk for smallpox but was being used as part of a stem-cell treatment for cancer, according to the FDA. "The unproven and potentially dangerous treatment was being injected intravenously and directly into patients' tumors," according to an FDA statement.

Smallpox essentially has been eradicated from the planet, but samples are kept in reserve in the U.S. and Russia, and vaccines are kept on hand as a result.

But Elliot Lander, medical director of the California Stem Cell Treatment Centers, denounced the FDA's actions in an interview with Shots.

"I think it's egregious," Lander says. "I think they made a mistake. I'm really baffled by this."

While his clinics do charge some patients for treatments that use stem cells derived from fat, Lander says, none of the cancer patients were charged and the treatments were administered as part of a carefully designed research study.

"Nobody was charged a single penny," Lander says. "We're just trying to move the field forward."

In a written statement, U.S. Stem Cell also defended its activities.

"The safety and health of our patients are our number one priority and the strict standards that we have in place follow the laws of the Food and Drug Administration," according to the statement.

"We have helped thousands of patients harness their own healing potential," the statement says. "It would be a mistake to limit these therapies from patients who need them when we are adhering to top industry standards."

But stem-cell researchers praised the FDA's actions.

"This is spectacular," says George Daley, dean of the Harvard Medical School and a leading stem-cell researcher. "This is the right thing to do."

Daley praised the FDA's promise to provide clear guidance soon for vetting legitimate stem-cell therapies while cracking down on "snake-oil salesmen" marketing unproven treatments.

Stem-cell research is "a major revolution in medicine. It's bound to ultimately deliver cures," Daley says. "But it's so early in the field," he adds. "Unfortunately, there are unscrupulous practitioners and clinics that are marketing therapies to patients, often at great expense, that haven't been proven to work and may be unsafe."

Others agreed.

"I see this is a major, positive step by the FDA," says Paul Knoepfler, a professor of cell biology at the University of of California, Davis, who has documented the proliferation of stem-cell clinics.

"I'm hoping that this signals a historic shift by the FDA to tackle the big problem of stem-cell clinics selling unapproved and sometimes dangerous stem cell "treatments" that may not be real treatments," Knoepfler says.

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FDA Cracks Down On Stem-Cell Clinics Selling Unapproved Treatments - NPR

Novartis AG’s CAR-T cell therapy for leukemia approved by FDA in ‘historic action’; price to be based on outcomes – MarketWatch

By Uncategorized

Novartis AG's NVS, -0.57% CAR-T cell therapy was approved by the Food and Drug Administration on Wednesday, making it the first gene therapy to be available in the U.S. Novartis' Kymriah was approved for young people up to age 25 with a form of acute lymphoblastic leukemia. CAR-T, or chimeric antigen receptor T-cell therapy, uses a patient's immune T-cells and re-engineers them to better fight cancer. As such, each dose of Kymriah is customized to the individual patient's T-cells through genetic modification. Novartis said on Wednesday that it will work with the Centers for Medicare and Medicaid Services so medicine prices can be "based on the clinical outcomes achieved, which would eliminate inefficiencies from the health care system." For Kymriah, Novartis is also working with CMS "to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month." Novartis did not give any specifics as to what the price range might be. Even before Novartis' Kymriah was approved, there was concern about pricing of the new therapy, given that new therapies are typically priced based on level of innovation and cancer is a particularly expensive area. On Wednesday, the FDA also expanded approval of Roche's ROG, +0.29% Genentech's rheumatoid arthritis drug Actemra to treat CAR-T cell-induced cytokine release syndrome, which consists of high fever and flu-like symptoms and can be life-threatening; nearly 70% of patients had CRS completely resolved in two weeks using one or two doses of Actemra, the FDA said. Kite Pharma, which is also working in the CAR-T space, has also been racing to gain FDA approval; the biotech's $11 billion acquisition by Gilead Sciences Inc. GILD, +0.05% was reported earlier this week. Gilead shares surged 5.5% in extremely heavy midday trade. Novartis shares declined 1% in heavy midday trade. Novartis shares have risen 2.8% over the last three months, compared with a 1.6% rise in the S&P 500 SPX, +0.20%

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Novartis AG's CAR-T cell therapy for leukemia approved by FDA in 'historic action'; price to be based on outcomes - MarketWatch

Modern Health & Wellness of Lima institutes regenerative stem cell … – Lima Ohio

By Uncategorized

LIMA A chiropractic center that specializes in treating chronic pain is now offering regenerative stem cell therapy, a new healing procedure that is the first of its kind in the area.

Modern Health & Wellness, located at 2425 Allentown Road, has partnered with Ohio Stem Cell and the Stem Cell Institute of America to bring this procedure to the region. According to Ohio Stem Cells doctors, patients can experience a significant decrease in pain and an improvement in range of motion within weeks of one treatment.

The research behind this technology is showing amazing results, said Modern Health & Wellness owner Dr. Patrick Gorman. In time, its our hope that this truly amazing therapy will eliminate the need for drugs and surgery.

Ohio Stem Cell doctors will be on site to administer the regenerative stem cell therapy, which has been approved by the Food and Drug Administration. Gorman said painless stem cell injections will help with arthritic and/or degenerative conditions, especially those found in the knees, hips, shoulders, neck and lower back.

These treatments can repair tissue in the body that has been damaged from age, disease or degeneration. It is accomplished by pinpointing the impaired areas, removing the swelling with anti-inflammatory properties and healing them by regenerating new cells and tissue.

This type of therapy is particularly effective in treating conditions such as degenerative arthritis, degenerative cartilage and ligaments, bone spurs, degenerative joint disease, bursitis and tendinitis.

Stem cell injections and therapy can help people that have bone-on-bone arthritis in their knee, and it can actually regenerate the tissue like the cartilage and the meniscus to help heal that area and allow people to go back to activities and function like they did before, Gorman said.

For those who may be concerned that stem cell therapy is against their religious beliefs, Gorman said it is illegal in the United States to obtain stem cells via an abortion.

The clinics that are harvesting these stem cells actually have to demonstrate and prove beyond a shadow of a doubt that these have been harvested via a successful C-section, he said. No babies are being aborted to obtain these cells.

Gorman added that anyone who is thinking of undergoing regenerative stem cell therapy should set up a consultation at the wellness center, or attend monthly lectures on this topic. The lectures are provided two to three times a month at the Area Agency on Aging 3, which is located in the same building as the wellness center.

We have a lot of people attend those lectures, and its mainly there to help educate people and explain to them what stem cell has done in the past, and what it can do for you, he said.

The next lecture is scheduled for 10 a.m. Sept. 9 at AAA3. Another lecture will be held at 10 a.m. Sept. 23.

Dr. Patrick Gorman, owner of Modern Health & Wellness in Lima, speaks to attendees at a ribbon-cutting ceremony celebrating the centers newly implemented regenerative stem cell therapy on Thursday.

http://limaohio.com/wp-content/uploads/2017/08/web1_stem-cell.jpgDr. Patrick Gorman, owner of Modern Health & Wellness in Lima, speaks to attendees at a ribbon-cutting ceremony celebrating the centers newly implemented regenerative stem cell therapy on Thursday. John Bush | The Lima News

Reach John Bush at 567-242-0456 or on Twitter @Bush_Lima.

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Modern Health & Wellness of Lima institutes regenerative stem cell ... - Lima Ohio

Cell Therapy Can Be Fast and Easy: Just Add mRNA Nanocarriers … – Genetic Engineering & Biotechnology News

By Uncategorized

Essentially, nanoparticles carried a gene-editing tool to T cells of the immune system that snipped out their natural T-cell receptors, and then was paired with genes encoding a chimeric antigen receptor, or CAR, a synthetic molecule designed to attack cancer.

Next, nanoparticles were targeted to blood stem cells and equipped with mRNA that enabled the stem cells to multiply and replace blood cancer cells with healthy cells when used in bone marrow transplants.

Finally, nanoparticles were targeted to CAR T cells and equipped with Foxo1 mRNA, which signals the anticancer T cells to develop into a type of "memory" cell that is more aggressive and destroys tumor cells more effectively and maintains antitumor activity longer.

"Our goal is to streamline the manufacture of cell-based therapies," said lead author Matthias Stephan, M.D., Ph.D., a faculty member in the Fred Hutch Clinical Research Division and an expert in developing biomaterials. "In this study, we created a product where you just add it to cultured cells and that's itno additional manufacturing steps."

Dr. Stephan and his colleagues developed a nanoparticle delivery system to extend the therapeutic potential of mRNA, which delivers molecular instructions from DNA to cells in the body, directing them to make proteins to prevent or fight disease.

The researchers' approach was designed to zero in on specific cell typesT cells of the immune system and blood stem cellsand deliver mRNA directly to the cells, triggering short-term gene expression. It's called "hit-and-run" genetic programming because the transient effect of mRNA does not change the DNA, but it is enough to make a permanent impact on the cells' therapeutic potential.

Other attempts to engineer mRNA into disease-fighting cells have been tricky. The large messenger molecule degrades quickly before it can have an effect, and the body's immune system recognizes it as foreignnot coming from DNA in the nucleus of the celland destroys it.

Stephan and his Fred Hutch collaborators devised a workaround to those hurdles.

"We developed a nanocarrier that binds and condenses synthetic mRNA and protects it from degradation," Dr. Stephan explained. The researchers surrounded the nanoparticle with a negatively charged envelope with a targeting ligand attached to the surface so that the particle selectively homes in and binds to a particular cell type.

The cells swallow up the tiny carrier, which can be loaded with different types of man-made mRNA. "If you know from the scientific literature that a signaling pathway works in synergy, you could co-deliver mRNA in a single nanoparticle," Dr. Stephan elaborated. "Every cell that takes up the nanoparticle can express both."

The approach involves mixing the freeze-dried nanoparticles with water and a sample of cells. Within four hours, cells start showing signs that the editing has taken effect. Boosters can be given if needed. Made from a dissolving biomaterial, the nanoparticles are removed from the body like other cell waste.

"Just add water to our freeze-dried product," Dr. Stephan emphasized. Since it's built on existing technologies and doesn't require knowledge of nanotechnology, he intends for it to be an off-the-shelf way for cell-therapy engineers to develop new approaches to treating a variety of diseases.

The approach could replace labor-intensive electroporation, a multistep cell-manufacturing technique that requires specialized equipment and clean rooms. All the handling ends up destroying many of the cells, which limits the amount that can be used in treatments for patients.

Gentler to cells, the nanoparticle system developed by the Fred Hutch team showed that up to 60 times more cells survive the process compared with electroporation. This is a critical feature for ensuring enough cells are viable when transferred to patients.

"You can imagine taking the nanoparticles and injecting them into a patient; then you don't have to culture cells at all anymore," he asserted.

Dr. Stephan has tested the technology in cultured cells in the lab, but it's not yet available as a treatment. He is looking for commercial partners to move the technology toward additional applications and into clinical trials where it could be developed into a therapy.

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Cell Therapy Can Be Fast and Easy: Just Add mRNA Nanocarriers ... - Genetic Engineering & Biotechnology News

Gilead Makes Long-Awaited Splash With $12B Bet on Kite, Cell Therapy – Xconomy

By Uncategorized

Xconomy San Francisco

Investors have been waiting for years for Gilead Sciences to make another big splash. This morning, it finally did, agreeing to buy Kite Pharma for close to $12 billion in a significant bet on the success of an emerging, cutting edge type of cancer immunotherapy known as CAR-T.

Gilead (NASDAQ: GILD), of Foster City, CA, is paying $180 per share in cash for Santa Monica, CA-based Kite (NASDAQ: KITE), a 29 percent premium to the companys $139.10 per share closing price on Friday and a deal that values the company at $11.9 billion. Kite shares promptly climbed 16 percent, to $162 apiece, early Monday. The agreement was first reported by the Wall Street Journal. It is expected to close during the fourth quarter. Kites lead product, to treat desperate cases of non-Hodgkin lymphoma, is expected to get FDA approval by the end of 2017.

For Gilead, the acquisition shows a renewed effort by the big drugmaker to make a dent in the oncology field. Gilead is known for its HIV drugs and more recently hepatitis C treatments, thanks to an $11 billion buyout of Pharmasset in 2011 that gave it the mega-blockbuster drugs sofosbuvir (Sovaldi) and sofosbuvir-ledipasvir (Harvoni). But over the years, Gilead has also steadily made a series of business development moves in oncology, among them an acquisition of Calistoga Pharmaceutials that gave the company its first, and to this point, only marketed cancer drug, idelalisib (Zydelig). In 2016, the drug generated $168 million in sales.

Gilead has been hoping for much more than that in cancer, and the pressure for the company to do something significant to generate excitement about its future has been building over the past few years as competing hepatitis C treatments have arrived and eroded its market share. Shares of Gilead are down almost 40 percent from their all-time highs in the summer of 2015 as calls for Gilead to use its pile of cashit had $36.6 billion on hand at the end of Juneon a transformative deal have intensified.

Will the Kite buyout be the jumpstart Gilead has been searching for? The deal is a gamble on a field of high promise, but substantial risk: a type of cancer immunotherapy treatment known as CAR-T, in which a patients immune cells are removed, modified, and re-infused into the body to find and kill cancer. The approach has shown promise in certain forms of blood cancer, in some cases wiping out leukemias or lymphomas in patients at deaths door, and is close to its first FDA approval. Novartis (NYSE: NVS) could bring the first CAR-T product, CTL019, to market later this year, and Kites axicabtagene ciloleucel (or axi-cel for short) could follow close behind. Kite has also filed for approval of axi-cel in Europe; a decision is expected next year.

The acquisition of Kite establishes Gilead as a leader in cellular therapy and provides a foundation from which to drive continued innovation for people with advanced cancers, said Gilead president and CEO, John Milligan, in a statement.

Milligan added that the cell therapy field has advanced very quickly, to the point where the science and technology have opened a clear path toward a potential cure for patients. And Gilead appears to be going all in, saying in its statement that it wants to build an industry-leading cell therapy franchise. But there are lingering questions about CAR-Ts overall potential. It comes with safety risks, namely figuring out how to harness the altered cells without causing the body significant harm in the process. A common reaction to treatment, for instance, is an immune system reaction called cytokine release syndrome that has proven deadly in some cases. Kite and Seattle competitor Juno Therapeutics (NASDAQ: JUNO) have also seen certain instances of brain swellingJuno had to abandon its most advanced treatment after the side effect led to multiple deaths in clinical testing.

In addition, CAR-T treatments havent yet worked in solid tumors, which include more prevalent cancers of the breast and lung. They also involve a complex manufacturing process and are likely to be very expensive; their commercial prospects are uncertain.

We are encouraged that Gilead has finally executed an acquisition and we think that the Kite deal is a major strategic positive, wrote Barclays analyst Geoff Meacham, in a research note. [T]he question will be if Kite will be big enough to move the needle and re-accelerate earnings growth to Sovaldi/Harvoni-launch levels.

Heres more on CAR-Ts promise and the questions that lie ahead for its developers.

Gilead will hold a conference call this morning to discuss the deal.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com

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Gilead Makes Long-Awaited Splash With $12B Bet on Kite, Cell Therapy - Xconomy

Sofa Vergara’s ex might finally be out of luck in his battle for custody … – Slate Magazine (blog)

By Embryonic Stem Cells

Sofa Vergaras embryos are citizens of California.

Christopher Polk/Getty Images for TNT

After years of battling ex-fiance Sofa Vergara in court for custody of a pair of frozen embryos they once made together, condiment entrepreneur Nick Loeb might finally be out of luck. Last week, a federal judge in Louisiana dismissed Loebs suit, saying that the embryos are citizens of California, where Vergara and Loeb conceived and froze them. Thus, the judge ruled, embryos have no legal standing to sue in Louisiana, the only state that gives embryos the right to sue and be sued.

Christina Cauterucci is a Slate staff writer.

Embryos are frozen when they are just clumps of a few dozen cells, equivalent to a vaginally inseminated egg that would still take another week to become embedded in the uterine wall. Louisiana law deems these cells juridical personsnot quite human beings, but deserving of legal rights. In Louisiana, embryos are not merely property of the two people who made them, so any legal disputes must meet the best interest of the in vitro fertilized ovum.

Thats the most likely reason why Loeb sued Vergara in Louisiana, despite the fact that neither party maintains a residence there.* (Loeb says he chose the state because the couple broke up there; he dropped an earlier California suit because he didnt want to name his previous girlfriends whod had abortions.) Actually, Loeb didnt exactly sue Vergarathe embryos, Emma and Isabella, did. Plaintiff EMMA is a female human being at the embryonic stage of life, five days old developmentally, the right to live suit read, claiming that Vergara had effectively abandoned and chronically neglected her children by keeping them frozen in a medical tank since 2013. Though Vergara and Loeb had signed a contract when they were together agreeing that the embryos would never be implanted anywhere without both parties consent, Loeb wanted to nullify the agreement and implant them in a surrogate.

Over the past couple of years, the Vergara-Loeb embryo battle has become a proxy fight for anti-abortion advocates who think frozen embryos should be treated like people. Anti-abortion groups have funded or filed amicus briefs in the embryo disputes of split-up couples, including the case of a Missouri woman named Jalesia McQueen. In the middle of her own court battle over two frozen embryos (Noah and Genesis) with an ex-husband who wanted to dispose of them, McQueen founded an organization called Embryo Defense to advocate for all excess embryos in legal limbo. She aggregates news on the Vergara-Loeb case and uses their photos in images made for sharing on social media. The graphics say things like Sofia says its selfish to let the embryos be born without both parents being in a loving relationship. Shouldnt both parents just love the child? and Please pray for Sofia Vergara and those she called her frozen babies, that shed open her heart so they could be a blessing in her life. One pairs a picture of Loebs face with the question what about a fathers right to choose?

The concept of a fathers right to procreate without input from the woman whose egg created the embryo is a favorite rallying cry of the embryo-protector set. Loeb himself made this argument in a 2015 New York Times op-ed with the magnificent headline Sofa Vergaras Ex-Fianc: Our Frozen Embryos Have a Right to Live. A woman is entitled to bring a pregnancy to term even if the man objects, Loeb wrote. Shouldnt a man who is willing to take on all parental responsibilities be similarly entitled to bring his embryos to term even if the woman objects?

The federal judges decision against Loeb is only the latest in a string of disappointing court losses for those who believe embryos should be treated like people. When Barack Obama lifted a ban on federal funding for embryonic stem cell research in 2009, he was hit with a lawsuit from one Mary Scott Doe, a frozen embryo symbolizing all existing frozen embryos. A federal appeals court ruled that Doe had no standing as an amorphous class that could not prove any actual harm. In 2015, a California Superior Court judge ruled that a contract a divorced couple signed at the medical center where they created the embryo prevented either party from taking unilateral custody of the embryos unless one of the parties died. Even McQueen, the founder of Embryo Defense, lost her suit late last year when a St. Louis court ruled that embryos are marital property of a special character, not human beings with unalienable rights.

That St. Louis case might be the most promising decision yet for those who believe that no person should be able to incubate an embryo without the consent of the other person whose genetic material it carries. The others, which rest on the tenets of contract law, legal standing, and jurisdiction, say more about how the suits were filed than what rights adults have to the embryos they create. Still, Loebs loss could set a welcome precedent in these kinds of cases: Jurisdiction-shopping embryo protectors might find Louisiana to be a less hospitable home for a lawsuit than theyd imagined.

*Correction, August 31, 2017: This piece originally referred to "the most likely reason why Loeb sued Vergara in California, despite the fact that neither party maintains a residence there." It should have said Louisiana.

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Sofa Vergara's ex might finally be out of luck in his battle for custody ... - Slate Magazine (blog)

Ethical Stem Cells Relieve Parkinson’s in Monkeys – National Review

By Adult Stem Cells

The advance of ethical stem cell research continues exponentially. Neurons made from induced pluripotent stem cellswhich were, in turn, made from skin cellshave relieved Parkinsons symptoms in monkeys. From the Nature story:

Takahashis team transformed iPS cells derived from both healthy people and those with Parkinsons into dopamine-producing neurons. They then transplanted these cells intomacaquemonkeys with a form of the disease induced by a neuron-killing toxin.

The transplanted brain cells survived for at least two years and formed connections with the monkeys brain cells, potentially explaining why the monkeys treated with cells began moving around their cages more frequently.

Crucially, Takahashis team found no sign that the transplanted cells had developed into tumours a key concern with treatments that involve pluripotent cells or that they evoked an immune response that couldnt be controlled with immune-suppressing drugs.

Human trials may begin in within a few years.

Two points about this, well three:

First, this study validates George W. Bushs prediction, when he placed mild limitations on federal embryonic stem cell funding,that scientists would be able to find ethical means of furthering regenerative medicine without using embryos.

Second, contrary to embryonic stem cells being the only hope, as so many Bush funding policy opponents claimed,embryonic stem cell research has not advanced nearly as far as adult stem cells and IPSCadvances have.

I keep bringing this up because all through the Bush terms in office, the scientists engaged in a mendacious campaign of hype and outright liesabout the potential and timing of treatments from embryonic stem cell research, as they poo-poohed the potential of alternative methods. But they were wrong and those who supported the Bush policy were right.

In other words, just because the Science Establishment says something, that doesnt make it so. Sometimes the scientists are wrong, or are conflating ideology with science, properly understood.

Third, contrary to animal rights ideologues and others, primate research is absolutely essential to furthering medical science. None of the potential we are seeing in this study could be known without testing on animals before humans.

So, lets hope that IPSCs and adult stem cells continue to advance into the clinical setting. They not only provide hope for efficacious treatmentslets not say curesbut offer a bridge across ethical divides that have roiled the field.

See original here:
Ethical Stem Cells Relieve Parkinson's in Monkeys - National Review

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