Xconomy Detroit/Ann Arbor

The University of Michigans Life Sciences Institute (LSI) is hosting its 16th annual Saltiel Life Sciences Symposium later this month, and for the first time, rather than focusing on a narrow segment of the industry, the lineup of speakers will discuss the technologies behind some of the sectors most exciting recent innovations.

Alan [Saltiel] started the tradition of a cutting-edge symposium in different areas of life sciences, says Roger Cone, who leads the LSI. Weve never focused on the technology itself, but were doing it this year because its the universitys 200th anniversary and because over the past decade, weve seen an explosion of new technologies completely changing how we do research in life sciences.

The symposiums organizers deliberately chose speakers who are pioneers focused on areas of research that dont yet have a huge presence at U-Mor anywhere else outside of the researchers lab, in some cases. The topics they will cover at the symposium include gene editing, single-cell biology, optogenetics, and cryo-electron microscopy. (More on the speakers in a minute.)

Cone feels single-cell technologies are among the most promising new innovations in biotech, especially when researchers can look at why a healthy cell becomes malignant. Thats an area where U-M has already made some inroads, he adds. Last month, U-M professor Arul Chinnaiyan published the results of a study in Nature that examined the genetic and molecular landscape of advanced cancer.

What kills people is metastatic disease, Cone explains. Its extremely important to know the changes that allow cells to become metastatic. Both in cancer and neuroscience, gene expression in single cells is revolutionary biology, and the university will absolutely play a role in developing that technology.

In fact, Cone feels this area is so important that next years life sciences symposium will be solely dedicated to single-cell innovations.

Just this week, the university announced that a multidisciplinary faculty committee has been tasked with identifying and pursuing emerging research opportunities. As part of U-M president Mark Schlissels Biosciences Initiative, a 16-member committee will have $150 million over five years to invest in new faculty hires, equipment, and other tools meant to facilitate progress and spark collaboration.

The Saltiel Life Sciences Symposium will be held on Sept. 15 at Forum Hall in Palmer Commons, on U-Ms campus. The event is free and open to the public. See below for details about the featured speakers and what theyve been working on.

George Church, a professor at Harvard and MIT and co-founder of numerous startups, wants to reanimate the woolly mammoth, edit pig genes so their organs can be transplanted safely into peopleoh, and reverse aging, according to a profile in STAT News.

Karl Deisseroth, a Stanford psychiatrist, will discuss his optogenetics technique, which the New Yorker said has given researchers unprecedented access to the workings of the brain, allowing them to observe the neural circuitry of lab animals as well as control behavior through cell manipulation.

Phillip Keller is a Howard Hughes Medical Institute researcher whose team developed a microscope that can quickly produce 3D images of whole organisms.

University of Texas Southwest Medical Center professor Daniela Nicastro uses cryo-electron microscopy, an advanced imaging technique where human samples are rapidly frozen to preserve their structure, then an electron microscope is used to produce images that can be transformed into 3D models. This helps researchers pinpoint diseases that affect tiny structures such as cilia, the infinitesimal hairs in the human body.

David Walt, a scientist at Harvard and Howard Hughes Medical Institute, has had a long career as a chemist, entrepreneur, and engineer, founding successful startups focused on genetic screening and ultra-sensitive protein analysis.

Sarah Schmid Stevenson is the editor of Xconomy Detroit/Ann Arbor. You can reach her at 313-570-9823 or sschmid@xconomy.com.

Excerpt from:
UM Life Sci Institute Looks Ahead with $150M Initiative, Symposium - Xconomy

Stem cells have the unique property of developing into any cell of the body under the right conditions. For this reason, there is a growing interest in using them to treat disorders such as hemophilia, diabetes and even neurodegenerative disorders such asParkinsons.

Apart from their therapeutic potential, researchers have shown that stem cells can be coaxed to spontaneously develop intominiature organ like structures called "organoids".Organoids recapitulate the intricate physical and biological features of organs and hence are important new tools in understanding human tissue development as well as for finding new drugs to treat disorders.

In this podcast, we discuss the biology of organoids, the hope and hype in medical research as well as potential ethical issuessurrounding their use.

This podcast is written and produced by IndSciComm, a collective of Indian scientists working on increasing public science awareness.

Shruti Muralidhar is a postdoc at the Picower Institute for Learning and Memory at MIT studying how memory is encoded in the brain.

Navneet Vasistha is a postdoctoral researcher at the University of Copenhagen trying to understand the cellular basis of mental health disorders.

Abhishek Chari is a science writer at the Picower Institute for Learning and Memory at MIT with an interest in microbiology and evolution.

(Scroll down past the references to read a transcript of the podcast.)

References and further reading

Skin transplants:

Serial Cultivation of Strains of Human Epidermal Keratinocytes

Grafting of Burns with Cultured Epithelium Prepared from Autologous Epidermal Cells

Cerebral organoids:

Cerebral organoids model human brain development and microcephaly

Pituitary organoids and functional restoration:

Self-formation of functional adenohypophysis in three-dimensional culture

Proto-tooth organoids:

The development of a bioengineered organ germ method

Fully functional bioengineered tooth replacement as an organ replacement therapy

Energy and entropy in living systems:

Energy and entropy flows in living systems

The Science of Self-Organization and Adaptivity

The Ilya Prigogine Nobel Prize

Self-organization in different scientific fields:

The science of self-organization and adaptivity

Self-organization in economics:

From simplistic to complex systems in economics

Self-organization in social sciences:

Self-organization and social science

Protein folding as self-organization:

Self-organization in protein folding and the hydrophobic interaction

Self-organization of cytoskeleton:

Directed cytoskeleton self-organization

Biofilm self-organization:

Self-Organization, Layered Structure, and Aggregation Enhance Persistence of a Synthetic Biofilm Consortium

Antibiotic resistance in biofilms:

Mechanisms of antibiotic resistance in bacterial biofilms

Biofilm formation evades immune system:

Biofilm Formation Avoids Complement Immunity and Phagocytosis of Streptococcus pneumoniae

Biofilms on teeth:

Oral Biofilm Architecture on Natural Teeth

Biofilms in catheter-associated UTIs:

Role of biofilm in catheter-associated urinary tract infection

Model system limitations / Self-organization in embryos and ethical issues:

Self-Organization of Stem Cell Colonies and of Early Mammalian Embryos

Comparisons between tissue cultures and embryo development:

In vitro organogenesis in three dimensions: self-organising stem cells

Categories of self-organization:

Cytosystems dynamics in self-organization of tissue architecture

Importance of apoptosis in embryo development:

Cell death in development: shaping the embryo

Optic cup organoid:

Self-formation of optic cups and storable stratified neural retina from human ESCs

Intestinal organoids:

Establishment of Human Colon Culture System

Intestinal OrganoidsCurrent and Future Applications

14-day rule:

Embryology policy: Revisit the 14-day rule

Early embryos in a dish (commentary):

What if stem cells turn into embryos in a dish?

Early embryos in a dish (research articles):

A method to recapitulate early embryonic spatial patterning in human embryonic stem cells

Ethical issues in human organoid and gastruloid research

The Ethics of Organoids: Scientists Weigh in on New Mini-Organs

Organoids are more like fetal or neonatal organs, not adult organs:

Human cerebral organoids recapitulate gene expression programs of fetal neocortex development

hPSC-derived lung and intestinal organoids as models of human fetal tissue

Organoids and the Zika virus:

The High Schooler Behind the Mini-Brain Generator

Cold Spring Harbor grant for 3D cancer organoids:

CSHL to lead international team developing next-generation organoid cancer research models

Conclusion:

Cutting-edge stem cell therapy proves safe, but will it ever be effective?

Transcript of the podcast

Navneet: A long time ago, in a galaxy far, far... Wait, is that how were starting this podcast? No, actually in 1975, at the Massachusetts Institute of Technology, scientists James Rheinwald and Howard Green developed a method by which they could indefinitely grow human skin in the lab!

This is the first report of scientists being able to grow an organ in the lab. Their litmus test came five years later, when they were asked to treat two patients admitted to the Brigham Hospital with significant burns. Not only were Green and his colleagues able to graft skin sheets grown from the patients own cells, but in six months time, these grafts could no longer be distinguished from the surrounding unburnt skin.

Attempts at growing other organs have not met with a similar degree of success, for a variety of reasons.

However, with recent advances in stem-cell biology, researchers have found that by growing stem cells in just the right way, they can produce tiny blobs of tissue that look and function like organs.

Depending on what molecular cues are added, scientists have been able to grow what can lazily be called mini-brains, mini-pancreas, mini-retinal tissues, etc. The collective term given to these lab-grown tissues is organoids. My name is Navneet.

Abhishek: Im Abhishek.

Shruti: This is Shruti.

All: And we are IndSciComm. In this podcast, were going to be talking about what these organoids are and what they are not, how close are they are to actual organs, what their future potential is and a whole host of other interesting things.

***

Navneet: So, lets begin with the basics. What are organoids and why are they interesting?

a. An organoid is a three-dimensional mass of cells that superficially resembles an organ or a gland. Researchers have generated several kinds of organoids using what they know about the development of different organs. Some examples are cerebral or brain organoids, intestinal organoids, pituitary organoids and so on.

b. Essentially, what makes them interesting is that cells grown in a dish with the right nutrient and cell growth factors can form something like mini-organs.

c. Some organoids have been transplanted into mice to restore functions or structures that they are lacking. For example, transplanted pituitary organoids have helped to restore the function of dysfunctional pituitary glands in mice. In fact, scientists have even transplanted a proto tooth organoid into the mouth of an adult mouse and watched it develop into a fully grown tooth!

***

Abhishek: So, cells can form structures of higher complexity like organoids. In essence, simple things (cells) come together to form more complex things (organs). This phenomenon is called self-organization. But how is this possible? Doesnt the second law of thermodynamics say that entropy has to increase over time?

How can order be created out of chaos, if entropy can only be increased? Entropy, by the way, is just the technical term for randomness. The solution is to rearrange the system using energy. Any decrease in entropy in one part of the system can be compensated by a proportionally larger increase in entropy in another part of the system.

As a simple analogy, consider the problem of cleaning your room. One way is to throw everything thats lying around into a cupboard. The room definitely looks more ordered but that doesnt detract from the mess inside the cupboard. Therefore, you havent reduced the net entropy of the systemyou have merely re-distributed it.

This isnt just some quirky, obscure thermodynamics loophole. A Nobel Prize in Chemistry was given for understanding how order can be generated from disorder, to Ilya Prigogine in 1977.

So, there is a theoretical basis to explain the origin of complexity in our universe. Self-organization as a phenomenon has been studied in physics, chemistry, biology and many other disciplines, including economics and sociology.

Now, getting back to the point. The three of us, we are all biologists by training. And just to remind our listeners, we still want to talk about organoids. So, lets work our way up to organoids by showing you how self-organization is necessaryright from the level of molecules to the level of the organism.

At the simplest level, we have molecules that can self-organize into more complex configurations. This happens with proteins, that are formed as a long, disorganized chain of amino acids. But, they fold themselves into complex nanomachines. Some of these can juggle atoms between other molecules, acting as catalysts for important biological reactions.

Next, molecules can self-organize into mega-structures that form important components in cells. Polymerization of small molecules helps to form the protein-based skeleton inside cells and the protein coats of some viruses.

Moving up from molecules, even apparently simple organisms like bacteria can self-organize themselves into marvels of biological architecture called biofilms. In this combined state, bacteria in biofilms can resist antibiotics, fight off the immune system and demonstrate feats of resilience that single cells are incapable of. You can blame biofilms for everything from the gunk on your teeth after a good nights sleep, to entrenched catheter infections and many other things in between.

All organisms are dependant, to varying degrees, on self-organization to make them what they are. Every multicellular organism, all the way from slime molds to plants and animals, starts off life as a single cell that has to replicate itself to make an embryo.

***

Shruti: The early embryo is a mass of stem cells without the defining features of a multicellular organismlike a head, tail, limbs and so on. Provided they get the right cues or signals, these stem cells are capable of forming a complete organism. Researchers study these cues and other steps in embryo development using animal models like mice.

View post:
What are organoids?: A Mint on Sunday audio story - Livemint

Scientists at the University of Glasgow have discovered a process to trigger the death of cancer cells that could be more effective than current methods.

The new method of killing cancer cells called Caspase Independent Cell Death (CICD), and published today in Nature Cell Biology, led to the complete eradication of tumours in experimental models.

Currently most anti-cancer therapies (chemotherapy, radiation and immunotherapy) work by killing cancer cells through a process called apoptosis, which activates proteins called caspases, leading to cell death.

But in apoptosis, therapies often fail to kill all cancer cells, leading to disease recurrence, and can also have unwanted side effects that may even promote cancer.

The scientists wanted to develop a way to improve therapy that induces cancer cell killing while also mitigating unwanted toxicity.

Dr Stephen Tait, Cancer Research UK Beatson Institute, Institute of Cancer Sciences, said: "Our research found that triggering Caspase-Independent Cell Death (CICD), but not apoptosis, often led to complete tumour regression.

"Especially under conditions of partial therapeutic response, as our experiments mimic, our data suggests that triggering tumour-specific CICD, rather than apoptosis, may be a more effective way to treat cancer."

Unlike apoptosis, which is a silent form of cell death, when cancer cells die through CICD, they alert the immune system through the release of inflammatory proteins.

The immune system can then attack the remaining tumour cells that evaded initial therapy-induced death.

The researchers used lab-grown colorectal cancer cells to show the advantage of killing cancer cells via CICD, however, these benefits may be applicable to a wide-range of cancer types.

He added: "In essence, this mechanism has the potential to dramatically improve the effectiveness of anticancer therapy and reduce unwanted toxicity. Taking into consideration our findings, we propose that engaging CICD as a means of anti-cancer therapy warrants further investigation."

Dr Justine Alford, Cancer Research UK's senior science information officer, said: "Although many cancer treatments work by triggering apoptosis, that method sometimes fails to finish the job and instead may lead to the tumour becoming harder to treat.

"This new research suggests there could be a better way to kill cancer cells which, as an added bonus, also activates the immune system. Now scientists need to investigate this idea further and, if further studies confirm it is effective, develop ways to trigger this particular route of cell death in humans."

The paper, 'Mitochondrial permeabilization engages NF-kB-dependent anti-tumour activity under caspase deficiency,' is published in Nature Cell Biology.

Explore further: Protein that promotes 'cell-suicide' could revolutionise eye cancer treatment

More information: Evangelos Giampazolias et al. Mitochondrial permeabilization engages NF-B-dependent anti-tumour activity under caspase deficiency, Nature Cell Biology (2017). DOI: 10.1038/ncb3596

Read more:
Scientists discover potential new improved way to kill cancer cells - Medical Xpress

Dr. Nathan Reyna

Dr. Nathan Reyna, Ouachita Baptist Universitys associate professor of biology, has been selected to participate in the National Science Foundations (NSF) mentor program, CURE Net.

Dr. Reyna will have the opportunity to serve as a mentor to other science professors in the country and teach them how to run course-embedded undergraduate research experiences (CUREs) in their own institutions. This program mirrors several programs Ouachitas J.D. Patterson School of Natural Sciences already sponsors, which is among the reasons Dr. Reyna was invited to participate.

Currently, Ouachita has five CURE programs embedded into its curriculum, coordinated primarily by Dr. Reyna and Dr. Ruth Plymale, associate professor of biology. One of these CUREs focuses on synthetic biology, where students select three different genes from different organisms and put them together. Reyna and Plymale received funding through the NSF-funded EPSCoR program to create the Arkansas CURE (AR:CURE) project to helpf faculty at other institutions develop similar programs. Over the summer, 18 undergraduate faculty from 10 states participated in a three-day workshop focused on this CURE. Dr. Reyna taught these professors how to execute the experiment in a classroom setting. This workshop was so successful, Reyna noted, that funding was secured to conduct the workshop for three more years.

We teach them how to assess student learning, how to do all the different things over the course of the semester with groups that are never in the same spot at any given time, Dr. Reyna explained. Thats what we do in the workshop, and thats what we do here at Ouachita. Several years ago, the biology department changed how we taught science. It is amazing that these ideas have now expanded to us helping other schools and universities.

Ouachita recently received funding to start another mentorship program with high school students and faculty. This program will take place next semester and will allow high school students and faculty to learn Ouachitas Bioinformatics CURE. This CURE allows students to use computers to analyze the genetic sequence of a bacteriophage. Ouachita students will now have the opportunity to teach and mentor people from surrounding high schools about this CURE. After the workshop, the high school faculty will have access to the program so they can share it with their students.

Were going to call it a Genome Hackathon, Dr. Reyna said. Were going to hack a virus during the course of a day. Were trying to get our students to serve as mentors, and were getting role models into the community. Were trying to increase interest in science, technology, engineering and math in the area.

In addition to these two CUREs, Ouachita has three others, focusing on a phage lab, genetics and cell biology. Ouachita has five CUREs in total in its curriculum, whereas many schools have only one, according to Reyna. The hands-on learning opportunities are an important part of the high-impact educational process for Ouachita science students.

Students will use this in the future, especially learning how to read a protocol, Dr. Reyna said. A lot of times it wont be the same protocol, but it will be the same thing when someone says, Here, read this, Ill help you for a day, but then youre on your own to redo it and think about how to fix it. Those skills are used forever. We are teaching students to learn and think like a scientist from day one.

Dr. Reyna will be able to use similar methods in place now in Ouachitas Patterson School for the NSFs CURE Net program. For more information on the CURE Net program or Ouachitas CUREs, contact Dr. Nathan Reyna at reynan@obu.edu or (870) 245-5240.

By Katie Smith

September 1, 2017

View post:
Ouachita professor Nathan Reyna invited to National Science Foundation-funded mentor program - Ouachita Baptist University News

Researchers at the University of California San Diego and at the San Diego Veterans Administration Medical Center have shown that human neural stem cells (NSCs) grafted onto the spinal cord injuries of mice produced a functional recovery after one year. The team has shown that the NSCs continue to grow slowly and steadily even18 months after implantation.

The study is published in the Journal of Clinical Investigation and set out to answer how long it would take for the cells to mature inside the rodents. Mice and humans have a very different pace when it comes to cell biology.

"The NSCs retained an intrinsic human rate of maturation despite being placed in a traumatic rodent environment," lead author Professor Paul Lu said in a statement. "That's a finding of great importance in planning for human clinical trials."

The researchers were worried that the animal model would not reflect the how this approach might in the future work in humans. For example, pregnancies last 21 days in mice and 280 days in humans. And the weight of a toddlers brain is comparable with that of a 20-day-old mouse.

"Most NSC grafting studies have been short-term, measuring survival times in weeks to a few months," added co-author Professor Mark Tuszynski. "That's not enough time to fully measure the growth and maturation rate of human NSCs or what changes might occur farther out from the original grafting. These are important considerations, not just for the basic science of stem cell biology, but for the practical design of translational human trials using NSCs for spinal cord injuries."

The researchers report that the cells maintained their natural maturation pace even though they were in a foreign environment. Thats why it took several months for the lesions to begin healing. The scientists noted that improvement in the mice mobility only happened after more mature nerve cells formed. As the grafts aged, they displayed the expected pruning and cell redistribution activities that help the development of fewer but more mature cells.

"The bottom line is that clinical outcome measures for future trials need to be focused on long time points after grafting," said Tuszynski. "We need to take into account the prolonged developmental biology of neural stem cells. Success, it would seem, will take time."

The team noticed that none of the implanted NSCs migrated from the graft but some supportive astrocytes cells did, which could be a potential safety concern. No tumors or anomalous formation were created by these cells and modified grafting should fix the problem. A better understanding of this approach, so that the results can be carefully assessed, is required before we can even think to try it on humans.

Read the original:
Human Stem Cells Repair Spinal Cord Injuries In Mice At Human Biological Rate - IFLScience

Iowa State researchers were awarded a four year, $2.98 million grant Tuesday from the National Institutes of Health.

The research will work todevelop innovative technology to search the genome of zebrafish for genes leading to advances in human health.

By identifying specific genes related to disease and switching them off and on, the researchers hope their findings could lead to new treatments for health issues such as cancer, vascular disease and neurological disorders.

We need to determine if a gene is curative, said Jeff Essner, professor ofgenetics, development and cell biologyand research team member. Were hoping to develop a toolbox that will allow us to identify genes in zebrafish, and ultimately in humans, that can be targeted with therapy to cure various ailments.

Zebrafish are small, freshwater fish.Zebrafish are ideal for this kind of genetics work because their embryos are fertilized outside the body of the mother.

The embryos are also transparent, making them easy for scientists to collect and target with the gene-editing technology.

Zebrafish share many genes with humans which lead to disease, said Maura McGrail,professor ofgenetics, development and cell biologyand another research team member.

The researchers can activate fluorescent genes in the zebrafish to cause certain tissues to glow.

Essner said this effect allows the researchers a direct way to confirm the gene-editing technology is working as they intend.

The team will also includeDrena Dobbs, a university professor of genetics, development and cell biology. They will also work withKarl Clark and Stephen Ekker at the Mayo Clinic in Rochester, Minnesota, who are conducting similar gene editing research in cultured human cells.

See the original post here:
ISU researchers receive $2.98 million grant - Iowa State Daily

In a step that heralds a new era in cancer treatment, the U.S. Food and Drug Administration said Wednesday it has approved a form of gene therapy that is highly effective at fighting an aggressive form of leukemia in young patients with no other options.

The treatment, to be marketed under the name Kymriah, is neither a pill nor an injection, but a personalized medicine service that functions as a living drug. Patients would have their bodys own disease-fighting T cells fortified and multiplied in a lab, then get the cells back to help them fight their cancer.

In clinical trials of 88 patients with a relapsing or treatment-resistant form of acute lymphoblastic leukemia, 73 went into remission after receiving the experimental treatment.

FDA Commissioner Scott Gottlieb, himself a survivor of blood cancer, predicted that this new approach to cancer treatment will change the face of modern medicine.

Cancer researchers and physicians outside the agency shared Gottliebs enthusiasm.

Dr. Crystal L. Mackall, associate director of Stanford Universitys Cancer Institute, called Kymriah a transformative therapy. It represents an entirely new class of cancer therapies that holds promise for all cancer patients.

Acute lymphoblastic leukemiais the most common form of pediatric cancer, affecting some 3,000 children and young adults yearly in the United States. Though it is considered highly curable in most patients, about 600 each year either do not respond to chemotherapy or see their leukemia return after an initial round of successful treatment.

Those patients dont make it none of them do, said Dr. Stephan A. Grupp, director of the cancer immunotherapy program at Childrens Hospital of Philadelphia, who administered the first course of Kymriah five years ago when it was an experimental treatment called CTL019.

That initial patient, 7-year-old Emily Whitehead of Philipsburg, Pa., saw her leukemia remit completely within three weeks of getting the treatment. Now 12, she was among those calling on the FDA to approve Kymriah for other patients like her.

Certainly for blood cancers, this is a game-changer, Grupp said. Adapting this therapy for patients with solid tumors, he said, will be the work of the next five years.

The new approach was designed to fight some of the most stubborn cancers by giving the bodys immune system a very specific assist.

It starts by harvesting a cancer patients T cells, the warriors of the immune system. The cells are delivered to a specialized lab where scientists alter their DNA, essentially reprogramming them to target cancer cells. These reengineered cells are called chimeric antigen receptor T cells, or CAR-T cells.

The new and improved cells are copied millions of times before theyre sent back to the patient. Once infused into the bloodstream, the CAR-T cells are much better equipped to hunt down and kill cancer cells, wherever they may hide.

Novartis, the company that developed Kymriah, intends to have 32 certified treatment centers up and running by the end of 2018. Patients up to the age of 25 would go to one of these centers to have their T cells harvested and later reintroduced in their modified form.

The cells themselves will be genetically engineered at a Novartis manufacturing facility in Morris Plains, N.J.

Kymriah is the first CAR-T treatment to come before the FDA, but it wont be the last. No fewer than 76 CAR-T treatments are currently under review at the FDA, and Gottlieb predicted that other approvals would follow.

Therapies that would operate in similar ways engineering the immune systems T cells to fight disease more effectively are under investigation for a host of other conditions, including HIV/AIDS, genetic and autoimmune disorders and other forms of cancer.

Todays FDA ruling is a milestone, said Dr. David Maloney, medical director of cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle. This is just the first of what will soon be many new immunotherapy-based treatments for a variety of cancers.

Novartis, the Swiss pharmaceutical company that is gearing up to provide Kymriah to as many as 600 patients a year, said it would charge $475,000 for the treatment.

Novartis representatives said they calculated a cost-effective price for the therapy that fell between $600,000 and $750,000. But the company chose instead to charge a price that it said would cover costs, and to introduce a novel approach to billing. Chief Executive Joseph Jimenez said the company will not charge hospitals for the therapy if the patient does not fully respond in a given period of time.

The company also said it will launch a patient assistance program for those who are uninsured or underinsured, and provide some travel assistance for patients and caregivers seeking the treatment.

Gottlieb touted Kymriahs approval as a turning point for the FDA as well. Novartis application for Kymriah came just seven months ago. The agency tagged the application with two designations that ensured its speedy review.

First proposed in 1972, the idea of correcting or enhancing genes to treat disease has a history buoyed by promise but also buffeted by failures. With recent advances in genomic medicine, cell biology and genetic engineering, efforts to locate and edit the genes and cells that play a key role in disease have injected new hope for such treatments.

Gene and cell therapies that target the immune system for enhancement have been particularly promising. They do, however, come with risks specifically, that the activation of immune cells will run amok, sparking reactions ranging from rash and itching to fever and flu-like symptoms that can lead to death.

In approving Kymriah, the FDA warned that it has the potential to cause severe side effects, including cytokine release syndrome, an overreaction to the activation and proliferation of immune cells that causes high fever and flu-like symptoms, and neurological events. Both can be life-threatening. Kymriah can also cause serious infections, low blood pressure, acute kidney injury, fever and low oxygen levels.

The FDA called for continuing safety studies of the new therapy.

melissa.healy@latimes.com

@LATMelissaHealy

MORE IN SCIENCE

FDA cracks down on clinics selling unproven stem cell 'therapies'

Catastrophic storms, once rare, are almost routine. Is climate change to blame?

NASAs Cassini spacecraft nears a fiery, brutal end, when it will plunge into Saturn

Read more from the original source:
'Transformative' cancer treatment: FDA approves gene therapy that functions as a 'living drug' - Los Angeles Times

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.

Continue reading here:
FDA cracks down on stem-cell clinics selling unapproved treatments - 89.3 KPCC

A newly discovered process to trigger the death of cancer cells could be more effective than current methods such as chemotherapy, scientists have said.

The new method of killing cancer cells, called Caspase Independent Cell Death (CICD), led to the complete eradication of tumours in experimental models.

Most current anti-cancer therapies (chemotherapy, radiation and immunotherapy) work by killing cancer cells through a process called apoptosis, which activates proteins called caspases, leading to cell death.

However in apoptosis, therapies often fail to kill all cancer cells, leading to disease recurrence, and can also have unwanted side effects that may even promote cancer.

Cancer Research's new ad is a live colonoscopy

Scientists from the University of Glasgow wanted to develop a way to improve therapy that induces cancer cell killing while also mitigating unwanted toxicity.

"Our research found that triggering Caspase-Independent Cell Death (CICD), but not apoptosis, often led to complete tumour regression.

"Especially under conditions of partial therapeutic response, as our experiments mimic, our data suggests that triggering tumour-specific CICD, rather than apoptosis, may be a more effective way to treat cancer," Dr Stephen Tait, Cancer Research UK Beatson Institute, from the Institute of Cancer Sciences, said.

Unlike apoptosis, which is a silent form of cell death, when cancer cells die through CICD, they alert the immune system through the release of inflammatory proteins.

The immune system can then attack the remaining tumour cells that evaded initial therapy-induced death.

In research published in Nature Cell Biology, the researchers used lab-grown colorectal cancer cells to show the advantage of killing cancer cells via CICD, however, these benefits may be applicable to a wide-range of cancer types.

Dr Tait added: "In essence, this mechanism has the potential to dramatically improve the effectiveness of anti-cancer therapy and reduce unwanted toxicity.

"Taking into consideration our findings, we propose that engaging CICD as a means of anti-cancer therapy warrants further investigation."

The paper was majority funded by Cancer Research UK.

Dr Justine Alford, Cancer Research UK's senior science information officer, said: "Although many cancer treatments work by triggering apoptosis, that method sometimes fails to finish the job and instead may lead to the tumour becoming harder to treat.

"This new research suggests there could be a better way to kill cancer cells which, as an added bonus, also activates the immune system. Now scientists need to investigate this idea further and, if further studies confirm it is effective, develop ways to trigger this particular route of cell death in humans."

Additional reporting by PA

Go here to read the rest:
New cancer treatment may be more effective than chemotherapy - The Independent

Researchers can activate fluorescent genes in zebrafish to cause certain tissues to glow, an indication their gene editing techniques are working as planned. ISU scientists hope to find genes in zebrafish that can lead to new treatments for diseases in humans. Image courtesy of Wesley Wierson. Larger image.

Ames, Iowa Iowa State University researchers have received a grant to further develop innovative technology that allows them to scour the genome of zebrafish for genes that might lead to advances in human health.

The researchers will use the latest gene editing techniques to create precise mutations in zebrafish. The project, supported by a four-year, $2.98 million grant from the National Institutes of Health, aims to identify genes connected to some of the most serious ailments humans and animals face, including cancer, vascular disease and neurological disorders.

By identifying particular genes related to disease and then switching them off and on again, the researchers hope their findings could lead to new treatments for various diseases.

We need to determine if a gene is curative, said Jeff Essner, a professor of genetics, development and cell biology and research team member. Were hoping to develop a toolbox that will allow us to identify genes in zebrafish, and ultimately in humans, that can be targeted with therapy to cure various ailments.

Many of the genes that lead to disease in humans are present in the zebrafish genome as well, said Maura McGrail, an assistant professor of genetics, development and cell biology and a member of the research team.

If we identify a gene in a zebrafish that affects disease, theres a good chance those results carry over to humans and agriculturally important animals as well, McGrail said. The genomes are about the same size and complexity. There are differences, but its a great starting point.

The Essner and McGrail laboratories boast scores of tanks that contain zebrafish, a small freshwater species that grow only a few centimeters in length. Zebrafish make good model organisms for this kind of genetics work because their embryos are fertilized outside the body of the mother and are transparent, making them easy for scientists to collect and target with the gene-editing technology.

The researchers can even activate fluorescent genes in the zebrafish to cause certain tissues to glow. Essner said doing so offers a direct way to confirm the gene-editing technology is working as intended. It also makes for a striking image.

The ISU team also includes Drena Dobbs, a University Professor of genetics, development and cell biology. The team will collaborate with Karl Clark and Stephen Ekker at the Mayo Clinic in Rochester, Minnesota, who are conducting similar gene editing research in cultured human cells.

Continued here:
New grant will help Iowa State University scientists search zebrafish genome to promote human health - Iowa State University News Service