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Stem Cells – Joint Preservation Institute of Iowa

Posted: September 3, 2017 at 3:41 pm

The Joint Preservation Institute of Iowa is now offering stem cell therapy in Des Moines.

Dr. Goding is one of the firstorthopaedic surgeons in the nation to offer this treatment. He uses autologous stem cell therapy and the regenerative power of mesenchymal stem cells to give patients another treatment option for dealing with orthopaedic conditions, such as osteoarthritis and other degenerative joint conditions.

You may benefit from orthopaedic stem cell therapy if you have:

Mesenchymal stem cells (MSCs) are adult stem cells found in bone marrow. The Joint Preservation Institute of Iowa performs autologous stem cell therapy, which means that the stem cells used in your treatment are taken from your own body, not from a donor. Using your own stem cells for the procedure helps reduce your risk of infection and eliminate the possibility of immune rejection.

In an autologous stem cell procedure, Dr. Goding will draw a sample of bone marrow from the iliac crest of your hip. The sample is filtered and concentrated in a sterile environment, then injected into the area of your body needing help to heal. This procedure is done on an outpatient basis while under sedation and leaves no scarring.

The idea behind orthopaedic stem cell therapy is that the injection of these concentrated regenerative cells at an area of your body experiencing degeneration will kick-start your bodys ability to heal itself. These injections can be given as an independent treatment or in conjunction with a surgical procedure.

Most cells in the human body have an assigned purpose. They are liver cells, fat cells, bone cells and so on. These cells can replicate more of their own kind, but they cannot create another type of cell.

Stem cells are the primitive cells from which all other cells developed. They are undifferentiated cells with the ability not only to self-replicate, but also to become different types of human cells. There are several types of stem cells, but the kind used in orthopaedic stem cell therapy are called mesenchymal stem cells (MSCs).

Bone Marrow Concentrate

Dr. Goding uses the BioCUE system from Zimmer Biomet. This system produces 77.5% recovery of nucleated cells.

Higher available cell numbers have correlated with improved results.

Autologous Protein Solution (APS)

APS is the newest treatment in the field of injectable orthobiologics. This procedure is essentially a further processing of platelet rich protein (PRP).

The tremendous advantage of APS is that it is derived from the blood instead of bone marrow. It is less painful, has less potential for injury, is less costly and it provides similar results.

After APS is derived from the blood, it is processed into PRP and then processed into APC. Using APC prevents macrophage cells from destroying healthy cartilage cells and has a profound anti-inflammatory effect in the joint. It also promotes growth and health of existing cartilage cells.

A recent study has shown up to 70 % pain reduction at 6 months in patients undergoing this procedure.

Dr. Goding uses the nSTRIDE system for APS treatments. An MSC has strong potential for tissue repair because it can:

In medical research, tissues such as muscles, cartilage, tendons and ligaments have shown some capacity for self repair. As a result, tissue engineering and the use of MSCs and/or bio-active molecules, such as growth factors, are being tested and studied to determine the role they can play in tissue regeneration and repair.

Articular Cartilage Damage to the articular cartilage following an injury has poor potential for repair and can lead to arthritic changes many years later. Recent studies have shown favorable outcomes and better knee scores at two-year follow ups for bone marrow derived MSCs that those of current techniques of microfracture and autologous chondrocyte implantation.

Bone Trauma and some pathological conditions can lead to extensive bone loss, which requires transplantation of bone and other bone substitutes to restore structural integrity. A large number of studies have shown great potential for mesenchymal cells to repair critically sized bone defects, producing better bone growth and more robust bone formation than controlled groups.

Tendons and Ligaments Injuries to tendons and ligaments heal by forming new tissues of inferior quality. Autografts, allografts and resorbable materials have been used to repair defects in tendons and ligaments, but these carry risks, such as donor site morbidity, scar formation and tissue rejection. A number of studies on the use of MSCs to improve the repair of tendons and tendon defects have been carried out with favorable results when measured in histology and tissue strength. The use of mesenchymal cells with tissue allografts enhances the graft and improves the biomechanical properties compared to control studies.

Meniscus Most tears of the meniscus occur in avascular zones with little or no potential for repair. Standard biological healing processes produce limited results and meniscectomy (removal of all or part of the torn meniscus) has been shown to have a strong association with subsequent development of osteoarthritis. Recently, studies have shown that self-paced therapy, including MSCs, demonstrates biological healing and adherence of meniscal tears in avascular zones.

Initial Visit An initial consultation with Dr. Goding will be required to find out if you are a candidate for stem cell therapy. In some cases, an MRI may be recommended to confirm your diagnosis and rule out any underlying conditions that could cause complications. If you are determined to be a candidate for stem cell therapy, your procedure will be scheduled for another day. This initial consultation is usually covered by your insurance plan.

Preparation For two weeks prior to your procedure, do not take aspirin or anti-inflammatory medications (NSAIDS).

Procedure Stand-alone stem cell therapy is done as an outpatient procedure, so most patients will leave the clinic and resume low impact activities the same day. Some patients have reported mild pain for 48-72 hours after their procedure, but this can often be attributed to the absence of their routine anti-inflammatory medications. After this time period, most patients will experience a gradual decrease in pain and some may begin to notice increased function.

Post-procedure To give your procedure the best chance to provide lasting results, our physicians recommend the following post-procedure restrictions:

Please note: If you are having a stem cell therapy procedure in conjunction with another surgical procedure, your recommendations may change. Consult with your physician on the guidelines and restrictions for your specific case.

No. Because mesenchymal stem cell (MSC) injections are considered investigational for orthopaedic applications, most insurance companies will not cover the cost. Please contact our office to discuss cash payment options.

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FDA Cracks Down On Stem-Cell Clinics Selling Unapproved Treatments – Iowa Public Radio

Posted: September 3, 2017 at 3:41 pm

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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US record holder for most time in space Peggy Whitson returns to Earth after 288-day mission – Pulse Headlines

Posted: September 3, 2017 at 3:41 pm

NASA astronaut Peggy Whitson returned Earth on Saturday, marking the end of her record-breaking time in space. Whitson spent several months working on the International Space Station, the $100-billion orbiting laboratory located about 250 miles above Earth.

The astronaut made the trip back home with NASA astronaut Jack Fischer and Roscosmos cosmonaut Fyodor Yurchikhin. The trio safely landed the Soyuz MS-04 capsule on Earth at 9:21 EDT Saturday southeast of Dzhezkazgan in Kazakhstan.

Whitson and Fischer contributed to hundreds of experiments in biology, physical science, biotechnology, and Earth science during their stay at the orbiting outpost. Whitson, 57, now holds the U.S. record for most time in space, as she totals 665 days in orbit.

This marked Whitsons third mission to the ISS. Her 288-day mission to the space outpost which spanned 122.2 million miles and 4,623 orbits of our planetbegan in November last year. She performed four spacewalks, bringing her overall total to ten. The biochemist has repeatedly shared her enthusiasm and love for her job.

I feel great, said Whitson during an in-flight interview on Monday, according to Reuters. I love working up here. Its one of the most gratifying jobs Ive ever had.

Whitson spent most of her time in the mission working on numerous scientific experiments. Fischer and Whitson supported research in a study on how astronauts eyes can change due to prolonged exposure to a microgravity environment. The duo also conducted a new lung tissue study that analyzes how stem cells work in the microgravity environment of the space station, which could help for future research on stem cells.

NASA says Fischer and Whitson also worked on a study on antibody investigation, to increase the effectiveness of chemotherapy drugs for cancer treatment, as well as a study of plant physiology and growth in space using an advanced plant habitat.

The astronauts, who also welcomed cargo spacecraft delivering tons of supplies, food, and research experiments, helped to attach the Cosmic Ray Energetics and Mass Investigation (ISS CREAM) on the exterior of the ISS in August, which is currently observing cosmic rays from across the galaxy.

Whitson also set a record for the most time spent spacewalking by a woman. She was due to return to Earth three months ago with the two crewmates who traveled with her in November. However, she stayed longer to fill a vacancy after Russia announced it would reduce its station staff from three to two cosmonauts.

During another in-flight interview, Whitson said she looked forward to reuniting with her loved ones.

Im looking forward to seeing friends and family, she said, according to Reuters. But the thing Ive been thinking about the most, kind of been fantasizing about a little bit, are foods that I want to make, vegetables that I want to saut, things that Ive missed up here.

Back in April, she broke the 534-day U.S. record for cumulative time in space. She is now part of a space hall of fame that includes seven Russian cosmonauts in the first spots. The world record-holder for cumulative time in space is Russian cosmonaut Gennady Padalka, who spent a total 878 days in orbit.

Whitson grew up in Iowa and says she was drawn to the National Aeronautics and Space Administration after the U.S. Apollo program that landed men on the moon. When women were able to join the space agency, she also applied. Whitson became an astronaut in 1996 and was the first woman to command the ISS and the first woman and first non-pilot to serve as chief of the NASA Astronauts Corps.

Yurchikhin, who came back with the American astronauts, also broke his record, as he has spent a total of 673 days in space, putting him seventh (one spot higher than Whitson) on the all-time space endurance list.

The Russian cosmonaut will head to Russias cosmonaut center in Moscow, and Fischer and Whitson will go to NASAs Johnson Space Center in Houston.Because hurricane Harvey caused severe damages all across Houston, NASA was forced to close the center temporarily and to come up with a contingency plan for getting Fischer and Whitson home.

NASA teamed up with the European Space Agency and sent the astronauts to the ESA center in Cologne, Germany. There, the NASA Gulfstream jet is scheduled to pick them up and carry them back to Houston on Sunday night. Whitson recently said NASA has been working non-stop after Harvey.

Our home is fine, but so many friends and co-workers have been impacted [by the storm], Whitson said in an interview with NASA, according to Space.com. For example, in order to keep mission control running, the team (three shifts of a skeleton support crew) were sleeping on cots in the backup mission control rooms. Their sacrifices for the station and keeping things running up here are amazing.

Source: Reuters

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Split-brain fruit fly research gives insight into autism – Nevada Today

Posted: September 3, 2017 at 3:40 pm

A better understanding of the cause of autism may come from an unlikely source,eurological studies of the fruit fly. Neuroscientists working in the biology department at the University of Nevada, Reno have identified a new genetic mechanism they believe is responsible for disruption of the brain pathways connecting the left and right hemispheres of the brain; which has separately been linked to autism.

This is an exciting find, Thomas Kidd, associate professor in the University's biology department, said. In the one striking mutant, called commissureless or comm, there are almost no connections between the two sides of the fruit flys nervous system.

The fruit fly nervous system research was conducted in Kidds lab over several years. Fruit flies have brains and nerve cords that form using molecules surprisingly similar to those in human brains and spinal cords. The study, published in the scientific journal PLOS Genetics, shows that the human gene, called PRRG4, functions the same way as the fruit fly Comm at the molecular level, regulating which signals neurons can respond to in their environment.

The Comm gene was thought to be unique to insects but our work shows that it is not, Elizabeth Justice, lead author of the PLOS Genetics article and a former postdoctoral neuroscience researcher in Kidds lab, said.

Comm is required for nerve fiber guidance and synapse formation in the fly, so PRRG4 could contribute to the autistic symptoms of WAGR by disturbing either of these processes in the developing human brain.

PRRG4 appears very likely to control how nerve fibers link the two sides of the nervous system in humans, and this is being actively tested, Sarah Barnum, a former undergraduate researcher in the Kidd lab who worked on the project, said.

MIssing GenesThe fruit fly has no left-right connections when two copies of the gene are missing. In humans there is a condition called WAGR syndrome in which a group of genes are missing on one chromosome. When the gene Kidds team is interested in, the PRRG4 gene, is missing, autistic symptoms are observed.

The function of the gene was obscure but we now show that it can regulate whether key proteins make it to the cell surface when neuronal wiring is navigating, Kidd said. This would tie it to our colleague Jeff Hutslers work that indicates autistic changes start in utero.

Jeffrey Hutsler, in the department of Psychology, and the Cognitive and BrainSciences Program and also in the Universitys neuroscience program, is an expert on autism and split-brain patients.

Bridges in the brainSplit brain patients have the connections between the left and right brain hemispheres severed, usually to relieve epilepsy symptoms. The disrupted structure is called the corpus callosum, a bridge consisting of millions of nerve fibers that allows constant exchange of information between the two sides of the brain. The corpus callosum forms during pregnancy and subtle disruptions to the structure are associated with developing autism.

Hutsler, who was not involved in the study, is also very excited by the work.

We know that brain wiring is altered in autism spectrum disorders and our own work has found similarities in the way visual information is integrated between the two brain hemispheres of split-brain patients and autistic individuals, Hutsler said. It is therefore very plausible that PRRG4 will be found to play a part in the altered formation of the corpus callosum in individuals with autism.

The journal which published the study, PLOS Genetics, commissioned a perspective on the article because of its significance.

Understanding the genetic mechanisms underlying the assembly of brain circuits is likely to be essential to the design of new diagnostic tools and therapeutic strategies for Autistic Spectrum Disorders, Jimena Berni wrote in the perspective, explaining that the study links the genetic alterations and neural circuitry development revealing a novel role for the PRRG4 gene as a regulator.

The University of Nevada, Reno study will inspire members of several diverse fields and drive research forward in several ways, including:

1. Axon (nerve fibers) guidance a range of physical interactors have been identified for PRRG proteins and these provide promising new avenues for investigation in all axon guidance systems.

2. Vertebrate brain development and human genetics the PRRG genes are expressed during brain development and in adults, but detailed surveys of expression patterns are lacking. Examination of key midline crossing structures such as the floor plate of the spinal cord and the corpus callosum is an obvious next step, but many other brain structures should be examined.

3. Yeast protein trafficking The findings offer the intriguing possibility that yeast genetics can be used to identify the mechanisms by which Rcr/Comm/PRRG proteins regulate protein trafficking to the cell surface.

The PLOS Genetics article is available on the Public Library of Science website.

The research was funded by the National Institute of Neurological Disorders and Stroke. The Kidd lab was part of a $10 millionCenter for Biomedical Research Excellence Project in Cell Biology of Signalingat the University, which is funded by the National Institute of Health's Institute of General Medical Sciences. Kidd is also a fellow in the Universitys Research and Innovation Office.

Both Jeff Hutsler Kidd are part of the Universitys Integrative Neuroscience program. In 2010, Hutsler received the Slifka-Ritvo Award for Innovation in Autism Research from International Society for Autism Research.

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Genes causing intellectual disabilities identified – The Indian Express

Posted: September 3, 2017 at 3:40 pm

By: IANS | London | Updated: September 3, 2017 7:01 pm The researchers expect this mechanism to help and play a role in a much larger proportion for patients with intellectual disabilities. (Source: Thinkstock Images)

Researchers have discovered 15 genes that play a role in the development of intellectual disabilities. Intellectual disabilities are often caused by a mutation that damages a gene responsible for protein production in cells thus preventing the associated protein from functioning properly.

In a number of disease-related genes, it is shown that a de novo mutation does not eliminate the gene, but probably alters its function. To find out how often this mechanism is involved, researchers combined the gene mutations in Dutch patients with a large international database comprising de novo mutations in patients.

A de novo mutation is a genetic alteration that is present for the first time in one family member as a result of a variant (or mutation) in a germ cell (egg or sperm) of one of the parents, or a variant that arises in the fertilised egg itself during early embryogenesis.

The results of the study, published in the American Journal of Human Genetics, showed 15 genes in which mutations cluster closely together, 12 of which were associated with developmental disorders. With our method, we were able to detect genes in which mutations not so much eliminate as affect the gene in another way, said Christian Gilissen, geneticist from Radboud University in Netherlands.

We also found three new genes that are likely to play a role in the development of intellectual disabilities as well, Gilissen added. The de novo mutations that were found only change a very small part of a protein. The function of the protein remains largely, but not entirely the same.

The mutations are more likely to affect superficial parts of the proteins. These disturb interactions with other proteins and cause problems, Gilissen said. The three newly-discovered genes playing a role in the development of intellectual disabilities provide new diagnostic possibilities for patients. It is important that we have discovered a mechanism that has not yet been a focus of study. We expect this mechanism to play a role in a much larger proportion of patients with intellectual disabilities.

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Has the Era of Gene Therapy Finally Arrived? – Scientific American … – Scientific American (blog)

Posted: September 2, 2017 at 5:47 am

In 1990, geneticist William French Anderson injectedcells with altered genes into a four-year-old girl with severe immunodeficiency disorder. This was the first sanctioned test of gene therapy, in which genetic material is used to treat or prevent disease.

If were lucky, Anderson told The Chicago Tribune, with this little girl weve opened the door for genetic engineering to attack major killers and cripplers, particularly AIDS, cancer and heart disease.

Gene therapy has never fulfilled these grand hopes. In the decades since Andersons experiment, thousands of clinical trials of gene therapies have been carried out. But the first gene therapy was only approved for sale in the U.S. this week. The Food and Drug Administration announced its approval of Kymriah, a gene therapy produced by Novartis for a form of childhood leukemia. A few gene therapies have previously become available in China and Europe.

An FDA press release emphasizes the historic nature of the approval. Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, FDA Commissioner Scott Gottlieb says.

As I have noted in previousposts (see Further Reading), the hype provoked by genetic research has always outrun the reality. Gene-therapy proponents have long predicted that it will eliminate diseases such as cystic fibrosis and early-onset breast cancer, which are traceable to a defective gene, as well as disorders with more complex genetic causes.Enthusiasts also envisioned genetically engineered "designer babies" who would grow up to be smarter than Nobel laureates and more athletic than Olympians.

Gene therapy turned out to be extremely difficult, because it can trigger unpredictable, fatal responses from the body's immune system.The National Institutes of Health warnsthat gene therapy can have very serious health risks, such as toxicity, inflammation, and cancer.

Kymriah is a case in point. The FDA press release warns that Kymriah can cause life-threatening immune reactions and neurological events, as well as serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). According to The New York Times, the FDA is requiring that hospitals and doctors be specially trained and certified to administer [Kymriah], and that they stock a certain drug needed to quell severe reactions.

Kymriah illustrates another problem with gene therapy: high cost. Novartis is charging $475,000 for Kymriah. As a recent Reuters article notes, over the past five years two gene therapies have been approved for sale in Europe, one for a rare blood disease and the other for the bubble-boy immunodeficiency disorder. The therapies cost $1 million and $700,000, respectively. So far, the companies that make the therapies have achieved a total of three sales.

As journalist Horace Freeland Judson points out in this excellent 2006 overview, The Glimmering Promise of Gene Therapy, biology and economics have conspired against gene therapy. Judsonnotes that most individual diseases caused by single-gene defectsthe kind that seem most likely to be cured by gene therapyare rare. (Sickle-cell anemia and some other hemoglobin disorders are among the few exceptions.)

Judson adds that because different diseases have different genetic mechanisms and affect different types of tissue, each presents a new set of research problems to be solved almost from scratch. As the millions burned away, it became clear that even with success, the cost per patient cured would continue to be enormous. And success had shown itself to be always glimmering and shifting just beyond reach.

The advent of CRISPR, a powerful gene-editing technique, has inspired hopes that gene therapy might finally fulfillexpectations. Researchers recently employed CRISPRin human embryos to counteract a mutation that causes heart disease. Potentially, The New York Times reported last month, the method could apply to any of more than 10,000 conditions caused by specific inherited mutations.

CRISPR has also renewed concerns about the ethics of engineering people with enhanced physical and mental traits. These concerns are grossly premature. As Science noted recently, CRISPR poses some of the same risks as other gene therapies. The methodstill has a long way to go before it can be used safely and effectively to repairnot just disruptgenes in people.And in fact questions have now been raised about the CRISPR research on embryos mentioned above.

Some day, applied genetics might live up to its hype, but that day is far from arriving.

Further Reading:

Could Olympians Be Tweaking Their Genes?

Have researchers really discovered any genes for behavior?

My Problem with Taboo Behavioral Genetics? The Research Stinks!

Hype of Feel-Good Gene Makes Me Feel Bad.

New York TimesHypes "Infidelity Gene."

Quest for Intelligence Genes Churns out More Dubious Results.

Warrior Gene Makes Me Mad.

Should Research on Race and IQ Be Banned?

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Pioneering gene therapy approved for leukaemia in the US – New Scientist

Posted: September 2, 2017 at 5:47 am

Carrying a $475,000 price-tag

AP

By Mallory Locklear

A CAR-T treatment a type of gene therapy for cancer has been approved for use in the US. Announced by the US Food and Drug Administration (FDA) on Wednesday, this is the first approval anywhere in the world for a type of CAR-T therapy, although the techniques have been used experimentally for some time.

CAR-T therapy made headlines earlier this year, when it was announced a CAR-T approach had saved the life of Layla, a young child in the UK who had leukaemia. The approach involves reprogramming a persons own immune cells to make them better at targeting cancerous ones.

The drug that has been approved by the FDA is Kymriah, a treatment for B-cell acute lymphoblastic leukaemia, the most common childhood cancer in the US.

To synthesise Kymriah, a patient first has a type of immune cell, called T-cells, removed from their body and transported to a facility in New Jersey operated by the pharmaceutical firm Novartis. Here, viruses will be used to insert a gene into these cells. The gene codes for a protein called a chimeric antigen receptor (CAR).

These cells are then reinfused back into the person. The added protein helps these modified T-cells home in on and fight leukemia cells.

In a trial, this approach achieved an 83 per cent remission rate over a period of three months in people who hadnt responded to other treatment options. The FDA has approved Kymriah for people aged 25 or under who have not responded to other treatments, or who have relapsed.

Nearly half the people in the trial experienced a side effect caused by an unwanted immune response triggered by the altered T-cells. Because of this, the FDA is requiring staff at the 32 facilities approved for this treatment to undergo specific training to recognise this response, called cytokine release syndrome.

Kymriah will cost $475,000. This sounds high, but its lower than some analysts expected, and unlike many expensive cancer drugs, it is a one-off treatment that could result in years, not months, of extended lifespan.

The FDAs decision has been hailed as the first approval for a gene therapy in the US. Some argue that this isnt a true gene therapy, as the genes introduced into the T-cells are not the treatment themselves it is the transformed T-cells that go on to fight the cancer. But the FDA defines human gene therapy as products that introduce genetic material into a persons DNA to treat a disease, so has classified Kymriah as such.

Europe has already approved two gene therapies for inherited diseases, while China approved a gene therapy for cancer treatment in 2004.

As for CAR-T therapies, other firms have similar treatments in the works, while Novartis also plans to get Kymriah approved for treating lymphoma.

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Lexington biotech plots $86M IPO as key gene therapy trial nears – Boston Business Journal

Posted: September 2, 2017 at 5:47 am


FierceBiotech
Lexington biotech plots $86M IPO as key gene therapy trial nears
Boston Business Journal
A Lexington biotech developing gene therapy treatments for rare eye diseases has announced plans to raise up to $86 million in an initial public offering. Nightstar Therapeutics, a 23-employee company with a 3,300 square foot office in Lexington and a ...
Nightstar files for $86M IPO to fund gene therapy trialsFierceBiotech

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Healthwatch: Gene Therapy – WeAreGreenBay.com

Posted: September 2, 2017 at 5:47 am

GREEN BAY, Wis. (WFRV) - (CBS News) The Food and Drug Administration approved a treatment that engineers a patient's own blood cells to fight cancer. It's the first type of gene therapy to hit the U-S market. It has already been used to treat children dying from leukemia, who are now cancer free.

The treatment reprograms a patient's own cells to destroy cancer, and then multiply to continue fighting disease for months or even years.

A new study looks at the risks associated with mononucleosis. A study in the journal Neurology finds that people who are infected with mono may have an increased risk for multiple sclerosis. White patients who had mono were twice as likely to develop MS. Black patients were four times more likely.

And American dads are getting older. A study at Stanford finds the average age of newborns' fathers has gone up by three and a half years since the 1970s. Men over the age of 40 now account for about 9-percent of U-S births. The potential downside is that older fatherhood is linked to higher rates of disorders including autism and schizophrenia.

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Weekend roundup: Campbell in the soup | New cancer gene therapy | Exposing bad investment advice – MarketWatch

Posted: September 2, 2017 at 5:47 am

MarketWatch rounded up 10 of its most interesting topics over the past week.

Campbell Soup Co. CPB, -1.73% had a rough quarter, but the company is facing a dire long-term problem.

Novartis AG NVS, -0.57% received FDA approval for the first cancer gene therapy available in the U.S. Emma Court explained how important this is for young people suffering from a type of acute lymphoblastic leukemia (ALL), and she interviewed Janney analyst Paul Knight, who made recommendations for investors on how to play a potential decade-long growth cycle for gene therapy.

Here are charts that will help you sift through a boatload of absurdity spouted every day by self-styled stock-market gurus.

Nissan Motor Co. 7201, +0.27% is about to launch a redesigned Leaf electric car. The company has a big advantage over Tesla Inc. TSLA, -0.14% because of its huge manufacturing scale, but one big question is the new Leafs battery range, as Claudia Assis reports.

The startling increase in value for bitcoin rivals that of other types of assets that have bubbled and burst. Andrew Left believes the Bitcoin Investment Trust GBTC, -20.40% is a very dangerous investment.

The damage from Hurricane Harvey to the Houston area has been devastating. The coming flurry of activity as the damage is repaired might cause a rise in U.S. GDP, but Caroline Baum calls claims of real economic benefits predictable nonsense.

Amazon.com Inc. AMZN, -0.24% was called the weakest major U.S. retailer this week by Moodys Investors Service. But T. Rowe Price Media and Telecommunications Fund PRMTX, -0.38% is a big believer. The fund, which had more than quadrupled the S&P 500s return over the past 15 years, had more than 10% of its assets in Amazons shares as of July 31.

If you are retired, you might think it will be very difficult to get a mortgage loan because of low income. But there are many financing options available for those without a steady monthly income, according to Darrow Kirkpatrick.

Jeff Reeves weighs the pros and cons of scooping up shares of Apple Inc. AAPL, +0.03% right now.

If you get excited by Labor Day sales, you might be missing out on bigger savings later.

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Weekend roundup: Campbell in the soup | New cancer gene therapy | Exposing bad investment advice - MarketWatch

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