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Former basketball star Greg Friel in fight of his life – Fosters – Foster’s Daily Democrat

Posted: August 15, 2017 at 5:46 am

Mike Whaley mwhaley@fosters.com @mwhaley25

Greg Friel can deal with the pain, as bad as it can be. Its the uncertainty about his future well-being that makes life difficult.

A former basketball star at Oyster River High School and Dartmouth College, Greg, 38, was diagnosed with relapsing remitting multiple sclerosis in 2010.

This unpredictable, often disabling disease of the central nervous system has weakened his body, particularly his legs below the knees, to the point where he often has to get around with a cane, and sometimes a walker.

Im in constant pain, he said. Its terrible. But Ive got a beautiful wife. Ive got a business where Im my own boss. Its a challenge. If you know the Friels, were up for a challenge. But its scary.

Fortunately, for Greg, there might be a light at the end of the tunnel. Next month, he will fly to Russia with his Uncle Bob for hematopoietic stem cell transplantation (HSCT). After being on the waiting list for three-plus years, Greg was recently granted admission to the treatment program.

The catch is that the cost for travel and treatment will be around $100,000. The treatment is not covered by insurance, so Gregs wife, Anastagia, has set up a GoFundMe page to raise funds to pay for the trip and treatment (https://www.gofundme.com/gregfrielhsct). As of Saturday morning, nearly $53,000 has been raised in three weeks.

Greg flies to Moscow on Sept. 24 for the month-long treatment, which begins on Sept. 26 at Piragov Hospital.

MS is so unpredictable, Anastagia said. Its hard to see my husband, a pillar of strength in the family, going through a weak time.

Hes such a positive person, she added. Hes charismatic. Theres such a light around him. Its hard to deny his charisma, and hes charming.

The treatment includes chemotherapy, where Greg said they shock your system down to zero. Good stem cells are injected, so basically its a stem cell rebirth.

It wasnt easy for Greg to reach out for help. An independent financial advisor in Boston, he was used to being the one helping out. It was in his nature to give.

My wife, my mom and my siblings told me I needed to open myself up, said Greg, who stands 6-foot-5. It was difficult. But if you get it early, you get proactive and you get things done, you can be in fine shape.

Thats the high-road approach Greg is taking.

Thats the athletic mentality, he said. Youre competitive because you care.

nnn

Greg comes from primo competitive stock. His dad, the late Gerry Friel, was the head mens basketball coach at the University of New Hampshire for 20 seasons (1969-89).

Greg is the middle child of five siblings. All of them played basketball at Oyster River and all five scored over 1,000 career points (Keith tallied over 2,000 points). They also all played Division I college basketball: Jennifer (UNH), Keith (Notre Dame, Virginia), Greg (Dartmouth), Jeremy (UNH) and Jilliane (UNH).

Greg and Keith starred at Oyster River in the mid-1990s, leading the Bobcats to back-to-back Class I championships in 1995 and 96.

Keith speaks fondly of those years playing with his brother. Greg was always two steps ahead and always competing. He did the little things, all the extra stuff that others didnt want to do. He loved to win, even if that meant diving for a ball or taking charges.

Greg was Wojo before Wojo, Keith said, referencing the feisty former Duke University guard of the late 1990s, Steve Wojciechowski. He was always firing up the other team, getting in their heads.

Plus he had that deadly 3-point stroke.

Greg graduated in 1997, attended New Hampton School for a post-graduate year where he counted future NBA star Darius Songaila (whos still a close friend) and Pistol Pete Maravichs son, Jason, among his teammates. Greg started for New Hampton and led them to the New England Class A prep championship game where they lost to Maine Central Institute.

Originally committed to the University of North Carolina at Wilmington, Greg decided to accept an offer from Dartmouth, coached by Somersworth native Dave Faucher, a former assistant at UNH under Gregs dad.

Two of Fauchers sons played at Lebanon High School, whom Oyster River beat in the 1995 Class I championship, 55-52. Greg set a tournament championship record with five 3-pointers in that game, a record he still shares 22 years later with two other players.

UNC-Wilmington would not let him out of his commitment, so he had to sit out a year before joining the Dartmouth team in the fall of 1999.

Greg played four years for the Big Green, serving as a captain as a senior. He graduated with a degree in economics and a minor in sociology.

As a player he got the chance to compete against both his brothers, although Jeremy laughs, I was on the bench while he was playing. I didnt get in.

Greg has been a financial advisor for 14 years, the last nine as his own man, running his own business.

He met his future wife while judging a beauty pageant in Las Vegas in 2009. A successful model, Anastagia has been Miss Teen Florida USA, Miss Florida USA and Miss Bahama Universe. She has also appeared in Sports Illustrateds swimsuit issue.

She actually didnt want to be at that particular pageant in 2009, but went at her moms behest and finished second.

They communicated on Facebook and it blossomed from there. Greg and Anastagia were married in May of 2014.

nnn

In 2010, when Greg was diagnosed with MS, it started with headaches.

I was feeling weak, he said. I was working 80-90 hours a week, so I was thinking it could have been that. I just kept getting weaker and weaker, my legs mostly. I was feeling lethargic. I didnt have any energy.

His legs hurt. He described it as a charley horse: You cant move, he said. Youre stuck in mud. It felt like youre cutting (to the basket) and someone knees your leg muscle. Thats what it felt like all the time.

At its worse, Greg said he couldnt move, couldnt walk, couldnt get out of bed.

Greg took a very aggressive drug called Tysabri for 4 years that cost $22,000 a month for an infusion and was partially covered by insurance. But it wasnt a cure and he didnt know the long-term effects. That was a concern since he and Anastagia want to have children.

Now theres hope with the Russian treatment.

Gregs cousin, Brendan Friel, who suffered from the same MS, went to Russia for treatment and is back in good health.

He just had his three-year stem cell anniversary, Greg said. And now hes working full-time.

Anastagia said they did their research. (The treatment) is invasive, but its got a good track record, she said.

What Greg finds irritating is that the procedure is covered by insurance for cancer, ALS and rheumatoid arthritis, but not MS.

Its crazy, he said.

As expected, its been difficult for his family.

Its heart-wrenching, said Joan, his mom. Its been more debilitating in the past year than ever. Hes going downhill since Thanksgiving of last year. Its one little setback after another this time. He has to do something; otherwise it will get so much worse.

Joan said shes there as a mom. Thats my job and I embrace it for all my children. Im a sounding board.

She added, I dont need to be intrusive. Im there if they need me and want me and, sometimes, if they dont.

Greg maintains an upbeat attitude.

Im good, he said. I try to stay positive. Ive always been a pretty positive person mentally.

nnn

The Friels have been touched by the outpouring of donations in such a short time, some from old adversaries or teammates Greg hasnt seen in 20 years.

Its not like one person wrote a check for $20,000, Greg said. Over 300 people have donated. Its very humbling. Its nice to see the support.

Later this month there will be a fundraiser at The Warehouse Bar & Grille, 40 Broad Street, in Boston to raise funds for Gregs treatment. It is being put together by a couple of old basketball opponents, Cliff Dever (Timberlane) and Marshall Chrane (Bishop Brady).

Chrane was on the Brady team that Gregs OR team beat in the 1996 Class I final, 58-49. Chrane returned the favor in 1997, hitting two foul shots with no time on the clock to beat Oyster River by a point in the tournament quarterfinals, Gregs last high school game.

The fundraiser will take place from 1 to 4 p.m. on Aug. 27.

There will be raffles, which include, among others, items donated by former pro and college football coach, Chip Kelly; UNH hockey coach Dick Umile and UMass-Amherst athletic director Ryan Bamford, Gregs old AAU teammate.

The journey to get Greg his treatment is certainly gaining momentum.

Were determined to get this right, he said.

Anastagia agrees.

I dont want anyone to be fighting alone, she said. Were committed to fighting this thing together.

Which is what good teammates do.

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First human embryo genetically modified in the US – Dailyuw

Posted: August 15, 2017 at 5:45 am

Researchers from Portland, Ore. genetically modified human embryos for the first time on American soil, but this is not a new feat. The process has already been done in China. To date, no genetically modified embryo has been inserted into a womb.

The lead researcher, Shoukhrat Mitalipov of Oregon Health and Science University, has a history of embryo work and demonstrated this round that its possible to safely remove inherited diseases by changing defective genes. This is called germline engineering. However, none of the embryos were allowed to last longer than a few days and the results are still pending publication.

Germline engineering typically uses CRISPR-Cas9, technology which precisely alters DNA. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.

At its roots, CRISPR is comprised of a small piece of RNA and a protein called Cas9. The RNA is preprogrammed to match a specific genetic code to then subsequently alter a specific strand of DNA once injected. The RNA guides the injection, and Cas9 tags along because, as an enzyme, it is able to break the DNA at an exact spot.

The challenge is that DNA tends to repair itself pretty fast. To avoid this, some CRISPR injections carry another strand of DNA the cell can use to fix the break thats created, therefore allowing genetic alterations.

The implications are very large, Dr. Charles Murry, Director of the UW Medicines Institute for Stem Cell and Regenerative Medicine, said. It gives us the ability to permanently eradicate a genetic disease from a familys pedigree. And as a physician, thats something thats extremely exciting to me.

Genetic modifications have been around for decades, and CRISPR has applied since early 2013. The possibilities for CRISPR were first realized through a natural bacterial process that defends against invasive viruses also known as this all started with yogurt, surprise.

However, the real breakthrough happened in 2015 with Junjiu Huangs first human embryo edits in China. Scientists are also looking at this system to eliminate pests and the diseases they carry.

Theres another side to it of course, Murry contended. When humans begin to rewrite our own genetic code, and there are all kinds of chances to not only make corrections as we edit but to make new mistakes as we edit we may inadvertently create problems in the attempt to solve others.

UW Health Sciences and Medicine public information editor Leila Gray said UW Medicine researchers are using CRISPR on specific somatic cells, which are the ones that make up your body. These cells were collected from patients with their approval. One team, for example, is trying to edit cells with kidney disease, studying certain conditions in petri dishes. But no UW researcher is reporting work to remove genetic diseases from human embryos.

Currently, the National Institutes of Health wont federally fund this research. However, the National Academy of Sciences and the National Academy of Medicine are recommending cautious reconsideration.

Murry predicts that before any of this would apply to a human being, a large animal would have to successfully carry to term a genetically modified embryo. Scientists would also likely have to monitor the newborns life afterward.

There are ethical conundrums with this new technology. Its so concerning that upon its first big embryonic debut, there was a three-day summit in December 2015 for hundreds of local and global scientists, policymakers, and the US presidential science adviser.

Some worry genetic engineering could lead to a dark future where humans are pre-edited for appearance, physical strength, or intelligence.

George Church, a Harvard Medical School geneticist, first told the Washington Post two years ago that there were nearly 2,000 genetic therapy trials already underway that didnt use CRISPR. The difference between those and the few that have is cost.

Its about 1,000 times cheaper for an ordinary academic to do, Church is quoted in the article. It could be a game-changer.

Reach reporter Kelsey Hamlin at news@dailyuw.com. Twitter: @ItsKelseyHamlin

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Stem cell therapy may help knees – News – Citizens’ Voice – Citizens Voice

Posted: August 15, 2017 at 5:44 am

Q: I read that you can use your own stem cells to rejuvenate worn-out knees. Does this really work?

A: Worn out is a good way to term what happens to the knee joint with prolonged use. Lets look at how this happens, starting with cartilage.

The lower portion of the knee joint (at the tibia) contains shock absorbers called menisci made of cartilage. You have one on the inner portion and another on the outer portion of each knee. The upper portion of the knee joint (at the femur) is lined with cartilage as well. All of this cartilage helps protect the bones at the joint but it doesnt heal or regenerate well due to limited blood supply. When severe, worn cartilage leads to arthritis of the knee. In knee X-rays of people over age 60, 37 percent have shown evidence of arthritis of the knees.

The intriguing thing about stem cells is that they have the ability to become any type of cell that the body needs. The cells used for stem cell injections in the knees are called mesenchymal stem cells, and they can differentiate into bone, fat or cartilage cells. These stem cells can come from the fat cells of your body, from your bone marrow or from the inner lining of your knee joint; theyre then replicated in the laboratory and injected into the knee joint.

Heres what the research shows so far.

In a 2013 study, 32 patients with meniscal tears of the knee were injected with a combination of stem cells, platelet-rich plasma and hyaluronic acid. The study reported improved symptoms and even MRI evidence of meniscal cartilage regeneration.

In a 2014 study, 55 patients who had surgery for meniscal tears of the knees were separated into three groups, with two of the groups receiving stem cell injections. Researchers found that, after six weeks, pain had decreased substantially in the two groups that received stem cell injections and that the decrease was even greater at one and two years after the injection.

In a 2017 study in the British Journal of Sports Medicine, researchers analyzed six studies that used stem cells for osteoarthritis of the knees. In five of the studies, stem cells were given after surgery to the knee; in the other study, stem cells from a donor were administered without surgery. All the studies showed reduced pain and improved knee function. Further, in three of the four trials, MRIs corroborated the cartilage improvements.

There may be benefit to stem cell injections for cartilage loss of the knees, but more data are needed. Id also like to see more data on this type of therapy as a preventive measure for younger patients before their knees are worn out.

ASK THE DOCTORS is written by Robert Ashley, M.D., Eve Glazier, M.D., and Elizabeth Ko, M.D. Send questions to askthedoctors@

mednet.ucla.edu, or write: Ask the Doctors, c/o Media Relations, UCLA Health, 924 Westwood Blvd., Suite 350, Los Angeles, CA, 90095.

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Medical Tourism Corporation Announces Stem Cell Therapy in Mexico With GIOSTAR – NBC Right Now

Posted: August 15, 2017 at 5:44 am

The two entities join hands for all-inclusive stem cell treatment in Los Algodones, Tijuana, and Guadalajara. Package includes personalized therapy protocol, travel assistance, and more. GIOSTAR is a global stem cell leader with more than 4,000 people treated.

DALLAS-August 14, 2017- (Newswire.com)

The major medical tourism facilitator has added another much-needed treatment to its comprehensive list of medical services, stem cell treatment in Mexico, as a way of helping people with degenerative, immunological, and blood-based diseases receive alternative healthcare and lead a happy, healthy life.

About GIOSTAR Mexico

Founded by a leading stem cell scientist, Dr. Anand Srivastava who is credited with setting up stem cell research programs in Sal Research Institute, UCSD, UCI and Sanford Burnham Institute, GIOSTAR is a visionary organization in the field. With its headquarters in San Diego and multiple top-notch facilities, offices and hospitals in various locations including India, Mexico, Brazil and Colombia, the venture is expanding its reach and making treatment for several devastating immunological and blood- related diseases accessible for all.

GIOSTAR Mexico offers stem cell therapy in Tijuana, Guadalajara, and Los Algodones. Current treatments include Diabetes Type I and Type II, Lupus, Multiple Sclerosis, Crohn's disease, and Spinal Cord Injuries (SCI), among others. The therapies for Alzheimer's, Autism, Anti-Aging Treatments, Parkinson's disease, Heart and Retinal Degeneration, and many more diseases are being developed by the dedicated and skilled members of the institute.

Some prominent features of GIOSTAR are:

About Medical Tourism Corporation (MTC)

The Texas-based Medical Tourism Corporation is a Better Business Bureau (BBB) accredited health tourism organizer. The corporation aims to connect medical tourists from all over the world to excellent healthcare services without the underlying stress. The most daunting part of the medical tourism process is the lack of information about quality treatments in Mexico, India, and other health tourism hubs. MTC emerges as a helping hand, and assists its customers in planning every little detail of their medical trip and even offers generous post-op services and follow-up care for a safe recovery.

With the introduction of alternative therapies for taxing and draining conditions, such as stem cells for Parkinson's in Mexico, the organization is expanding its arsenal of services and treatments offered.

Medical Tourism Corporation has recently partnered with GIOSTAR with the goal of making quality healthcare come true. The two entities have collaborated to offer personalized protocols for various conditions and injuries, including stem cell therapy for spinal injuries in Mexico.

Aiming to provide exemplary stem cell treatment in Mexico, MTC provides a host of services and features that set the venture apart:

What to Expect?

The National Institutes of Health (NIH) estimates up to 23.5 million Americans suffer from autoimmune diseases. All that is standing between these patients and life-altering medical treatments is a border away.

This timely collaboration between GIOSTAR and MTC is an important step forward in bridging the gap between demand and supply of effective alternative treatments for people suffering from the aforementioned ailments when conventional therapies fail to treat. More information on stem cell therapy in Mexico is available on the official website of Medical Tourism Corporation.

Related LinksStem Cell Treatment for Diabetes Type 1 and 2 in MexicoStem Cell Treatment for Arthritis in Mexico

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CAR T-Cell Therapy Shown to Eliminate Tumors in Some Chronic Lymphocytic Leukemia Patients During Trial – Lymphoma News Today

Posted: August 15, 2017 at 5:44 am

CAR T-cell immunotherapy was seen to eradicate or shrink tumors in 70% of patients with chronic lymphocytic leukemia (CLL) who had exhausted other treatment options, according to a clinical trial report.

Among those who had no sign of cancer left in the bone marrow four weeks after treatment, all had survived with no signs of cancer after six months.

Researchers at the Fred Hutchinson Cancer Research Center also suggested, based on the studies, that analyzing bone marrow using a new genetic technique is a better method than the more commonly used cell counts when attempting to determine a prognosis for the disease.

The report,Durable Molecular Remissions in Chronic Lymphocytic Leukemia Treated With CD19-Specific Chimeric Antigen ReceptorModified T Cells After Failure of Ibrutinib,described the outcomes of 24 CLL patients whose cancer had progressed after treatment with Imbruvica (ibrutinib), which was approved for the treatment of CLL in 2014. The research was published in theJournal of Clinical Oncology.

It was not known whether CAR T-cells could be used to treat these high-risk CLL patients, Cameron Turtle, an immunotherapy researcher at Fred Hutch and lead author of the study, said in apress release. Our study shows that CD19 CAR T-cells are a highly promising treatment for CLL patients who have failed ibrutinib.

CD19 is a molecule found on the surface of leukemia cells. T-cells, gathered from a patient, are engineered to specifically recognize this factor. When that happens, the body launches an aggressive immune response toward these cancer cells.

The Phase 1/2 study (NCT01865617) which is still recruiting participants included patients who had failed a median of five previous treatment rounds. The participants ages ranged from 40 to 73.

Before injecting the engineered T-cells into patients, their white blood cells were destroyed by chemotherapy.

One patient had a severe toxic response to the treatment and was not assessed for the therapys effects. Among the remaining patients, 16 of 23 70% had a response four weeks after treatment.

Not all patients had chemotherapy before CAR T-cell treatment, but among the 19 who did, four had a complete response, and 10 had a partial response.

Two additional patients responded after a second round of chemotherapy and CAR T-cell treatment.

Analyzing bone marrow using a method that can sort cancerous cells from normal cells indicated that 88% were free of disease after treatment. But repeating the analysis in 12 of these patients using a method called IGH deep sequencing showed that only 58% of them had no disease present in the bone marrow.

Those in which the genetic analysis found no traces of cancer all survived with no further traces of disease for a median of 6.6 months after treatment.

For more information about the trial, which also includes patients with relapsed or refractory non-Hodgkins lymphoma or acute lymphoblastic leukemia, see the trial registration page at thislink. The trial treats patients at theSeattle Cancer Care Allianceclinic.

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Cardiac stem cells rejuvenate rats’ aging hearts, study says – CNN – CNN

Posted: August 15, 2017 at 5:44 am

The old rats appeared newly invigorated after receiving their injections. As hoped, the cardiac stem cells improved heart function yet also provided additional benefits. The rats' fur fur, shaved for surgery, grew back more quickly than expected, and their chromosomal telomeres, which commonly shrink with age, lengthened.

The old rats receiving the cardiac stem cells also had increased stamina overall, exercising more than before the infusion.

"It's extremely exciting," said Dr. Eduardo Marbn, primary investigator on the research and director of the Cedars-Sinai Heart Institute. Witnessing "the systemic rejuvenating effects," he said, "it's kind of like an unexpected fountain of youth."

"We've been studying new forms of cell therapy for the heart for some 12 years now," Marbn said.

Some of this research has focused on cardiosphere-derived cells.

"They're progenitor cells from the heart itself," Marbn said. Progenitor cells are generated from stem cells and share some, but not all, of the same properties. For instance, they can differentiate into more than one kind of cell like stem cells, but unlike stem cells, progenitor cells cannot divide and reproduce indefinitely.

Since heart failure with preserved ejection fraction is similar to aging, Marbn decided to experiment on old rats, ones that suffered from a type of heart problem "that's very typical of what we find in older human beings: The heart's stiff, and it doesn't relax right, and it causes fluid to back up some," Marbn explained.

He and his team injected cardiosphere-derived cells from newborn rats into the hearts of 22-month-old rats -- that's elderly for a rat. Similar old rats received a placebo injection of saline solution. Then, Marbn and his team compared both groups to young rats that were 4 months old. After a month, they compared the rats again.

Even though the cells were injected into the heart, their effects were noticeable throughout the body, Marbn said

"The animals could exercise further than they could before by about 20%, and one of the most striking things, especially for me (because I'm kind of losing my hair) the animals ... regrew their fur a lot better after they'd gotten cells" compared with the placebo rats, Marbn said.

The rats that received cardiosphere-derived cells also experienced improved heart function and showed longer heart cell telomeres.

Why did it work?

The working hypothesis is that the cells secrete exosomes, tiny vesicles that "contain a lot of nucleic acids, things like RNA, that can change patterns of the way the tissue responds to injury and the way genes are expressed in the tissue," Marbn said.

It is the exosomes that act on the heart and make it better as well as mediating long-distance effects on exercise capacity and hair regrowth, he explained.

Looking to the future, Marbn said he's begun to explore delivering the cardiac stem cells intravenously in a simple infusion -- instead of injecting them directly into the heart, which would be a complex procedure for a human patient -- and seeing whether the same beneficial effects occur.

Dr. Gary Gerstenblith, a professor of medicine in the cardiology division of Johns Hopkins Medicine, said the new study is "very comprehensive."

"Striking benefits are demonstrated not only from a cardiac perspective but across multiple organ systems," said Gerstenblith, who did not contribute to the new research. "The results suggest that stem cell therapies should be studied as an additional therapeutic option in the treatment of cardiac and other diseases common in the elderly."

Todd Herron, director of the University of Michigan Frankel Cardiovascular Center's Cardiovascular Regeneration Core Laboratory, said Marbn, with his previous work with cardiac stem cells, has "led the field in this area."

"The novelty of this bit of work is, they started to look at more precise molecular mechanisms to explain the phenomenon they've seen in the past," said Herron, who played no role in the new research.

One strength of the approach here is that the researchers have taken cells "from the organ that they want to rejuvenate, so that makes it likely that the cells stay there in that tissue," Herron said.

He believes that more extensive study, beginning with larger animals and including long-term followup, is needed before this technique could be used in humans.

"We need to make sure there's no harm being done," Herron said, adding that extending the lifetime and improving quality of life amounts to "a tradeoff between the potential risk and the potential good that can be done."

Capicor hasn't announced any plans to do studies in aging, but the possibility exists.

After all, the cells have been proven "completely safe" in "over 100 human patients," so it would be possible to fast-track them into the clinic, Marbn explained: "I can't tell you that there are any plans to do that, but it could easily be done from a safety viewpoint."

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2m collaboration to focus on gene and cell therapy – Drug Target Review

Posted: August 15, 2017 at 5:44 am

A leading gene and cell therapy group has announced a collaboration focusing on gene and cell therapy manufacturing..

A leading gene and cell therapy group, has announced it has agreed to enter into a collaboration agreement with a consortium of partners, the agreement is a two-year 2 million collaboration project focused on gene and cell therapy manufacturing, co-funded by the UKs innovation agency, Innovate UK.

Cell and gene therapies offer unprecedented promise for the cure, treatment or long-term management of disease and we are delighted that this consortium has been awarded funding from Innovate UK that will help to keep Oxford BioMedica (OXB), our partners and the UK, at the forefront of innovation in industrial viral vector manufacturing., said John Dawson, Chief Executive Officer of Oxford BioMedica.

The aim of the collaboration is to explore and apply novel advanced technologies to further evolve OXBs proprietary suspension LentiVector platform to deliver even higher quality vectors for both clinical and commercial use. The project aims to deliver tangible benefits to patients by shortening the time-to-clinic and time-to-market as well as to improve the cost and access of bringing novel gene and cell therapies to patients.

Each partner in the collaboration holds proprietary technology and know-how that can be used to develop an innovative approach to viral vector manufacturing.

Collaborating on developing improved process analytic technologies with our partners will help drive productivity in viral vector manufacturing, accelerating the development of these transformative advanced therapies. We have the opportunity to both transform patients lives and grow an industry in the UK that we can be proud of,said Keith Thompson, Chief Executive Officer of Cell and Gene Therapy Catapult.

The aims of this pioneering project are closely aligned with the current government national priorities to make the UK a global hub for manufacturing advanced therapies, which will benefit economic growth and create and retain more highly skilled employment.

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A New Gene Editing Technique Could Finally Allow Us to Treat ALS – Futurism

Posted: August 15, 2017 at 5:44 am

In BriefResearchers from UC San Diego's School of Medicine have tested a modified CRISPR-Cas9 technique designed to target RNA instead of DNA. Rcas9 could potentially improve the lives of patients with ALS, Huntington's disease, or myotonic dystrophy by delaying the progression of their disorders.

The most efficient and effective gene-editing tool in use today is CRISPR-Cas9. Just this year, researchers have successfully used it fora wide variety of experiments, from modifying garden vegetables to encoding a GIF in bacterial DNA. Most recently, the tool was used to remove a genetic disease from a human embryo.

Although undeniably powerful, CRISPR-Cas9 does have its limitations; it can only target DNA. To extend its capabilities to includeRNA editing, researchers from the University of California San Diego (UCSD) School of Medicinedeveloped amodification of CRISPR, and theyre calling their toolRNA-targeting Cas9 (RCas9).

In a study published in Cell, the UCSD team tested their technique by correcting the kinds of molecular mistakes that cause people to develop microsatellite repeat expansion diseases, such ashereditary amyotrophic lateral sclerosis (ALS)and Huntingtons disease.

During standard CRISPR-CAs9 gene editing, a guide RNA is instructed to deliver a Cas9 enzyme to a specific DNA molecule. The researchers from UCSD instead instructed it to target an RNA molecule.

Tests conducted in the laboratory showed that RCas9 removed 95 percent ofproblem-causing RNA for myotonic dystrophy types 1 and 2, Huntingtons disease, and one type of ALS. The technique also reversed 93 percent of the dysfunctional RNA targets in the muscle cells of patients with myotonic dystrophy type 1, resulting in healthier cells.

This is exciting because were not only targeting the root cause of diseases for which there are no current therapies to delay progression, but weve re-engineered the CRISPR-Cas9 system in a way thats feasible to deliver it to specific tissues via a viral vector, senior author Gene Yeo, a cellular and molecular medicine professor at UCSD School of Medicine, explained in a press release.

Across the globe, an estimated 450,000 patients are said to be living with ALS. Roughly 30,000 of those are from the U.S. where 5,600 people are diagnosed with the diseases every year. The exact number of Huntingtons disease cases, however, isnt quite as easy to pin down. One estimate says that around 30,000 Americans display symptoms of it, while more than 200,000 are at risk.

Regardless of the exact numbers, these two neurological diseases clearly affect a significant number of people. This prevalence and the absence of a known curemakes the UCSD teams research all the more relevant. Even more exciting is the fact that the same kinds of RNA mutations targeted by this study are known to cause more than 20 other genetic diseases.

Our ability to program the RCas9 system to target different repeats, combined with low risk of off-target effects, is its major strength, co-first author of the study Ranjan Batra said in the UCSD press release.

However, the researchers do know that what theyve accomplished is just a first step. While RCas9 works in a lab, they still have to figure out how it will fare when tested in actual patients.

The main thing we dont know yet is whether or not the viral vectors that deliver RCas9 to cells would elicit an immune response, explained Yeo. Before this could be tested in humans, we would need to test it in animal models, determine potential toxicities, and evaluate long-term exposure.

Ultimately, while RCas9 couldnt exactly deliver a cure, it could potentially extend patients healthy years. For disease like ALS and Huntingtons, thats a good place to start.

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Clinical trial uses a genetically engineered virus to fight cancer – Medical Xpress

Posted: August 15, 2017 at 5:44 am

Dr. Steven Powell. Credit: Sanford Health

Sanford Health is the first site in the United States to launch a clinical trial using a genetically-engineered virus that aims to destroy therapy-resistant tumors.

The Phase I immunotherapy trial is for those ages 18 and older with metastatic solid tumors that have not responded to standard treatments. The treatment injects an oncolytic (cancer-destroying) virusvesicular stomatitis virus (VSV)into the tumor. The virus is engineered to grow in cancer cells, destroy these tumors, and then spread to other cancer sites. During this process, it recruits the immune system to the area with the goal of triggering an immune response.

The virus, commonly known as VSV, can infect cattle, but it rarely causes serious infections in humans.

The virus is genetically altered by adding two genes. The first gene is a human interferon beta gene, which is a natural anti-viral protein. This protects the normal, healthy cells from being infected, while still allowing the virus to work against cancer cells.

The second gene makes the NIS protein found in the thyroid gland, which allows the researchers to track the virus as it spreads to tumor sites. Vyriad, a biopharmaceutical company in Rochester, Minnesota, developed this technology and is led by Stephen Russell, M.D., Ph.D., a professor of molecular medicine at the Mayo Clinic and an expert in oncolytic virus therapy.

"Oncolytic viruses are the next wave of promising cancer immunotherapy treatments," says Dr. Steven Powell, a medical oncologist with the Sanford Cancer Center in Sioux Falls, S.D., who collaborated with Vyriad on the development of this clinical trial. "We are very excited about using VSV as researchers have seen promising results using other similar viruses, such as the polio virus, in early clinical trials."

Dr. Shannon Peck, an interventional radiologist at Sanford with experience in interventional therapeutics, oversees the viral injection procedures. Enrollees in the trial are given a one-time injection and then are followed for 43 days to evaluate for safety and clinical benefit. To ensure safety during this period, other anti-cancer therapies cannot be used. However, after this 43-day period, chemotherapy, immunotherapy or targeted therapy can be restarted.

Sanford Health is the first in the nation to launch the Vyriad solid tumor oncolytic virus clinical trial. Call 1-877-SURVIVAL to learn more or to see if you qualify.

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Georgia colleges gear up for new semester – AJC.com – Atlanta Journal Constitution

Posted: August 15, 2017 at 5:44 am

More than 300,000 students return to college and university campuses this month in Georgia.

The biggest changes include a newcampus carry law that allows students with licensed weapons permits to carry firearms on portions of campuses, additional credit to studentswho take approved STEM courses to keep their HOPE scholarships and the repurposing of Turner Field into Georgia States new football stadium.

Heres a look at some changes at some of metro Atlantas largest campuses and the University of Georgia.

The renovation of Rebekah Scott Hall. The $16.5 million project will house a new welcome center, updated offices for admissions and financial aid and residential space for students who will live on the upper two floors.

Atlanta Metropolitan State College

An online Bachelor of Science degree in organizational leadership. It includes a choice of concentration in public service, healthcare administration or office administration and technology.

Its createdthe Department of Cyber-Physical Systems. It will include new bachelor of science programs in cybersecurity, robotics, and data analytics.

The first-two floors of a new $400 million hospital tower opened on July 31, bringing the total number of licensed beds at Emory University Hospital to 733. Patient floors begin opening in late August, and the hospital tower will be fully operational by the end of October.

Awidening of a portion of Clifton Road and its sidewalks, a bike lane, new landscaping and improved visibility of intersections along Clifton Road.

The campus West Village, which includes five micro-restaurants, Panera Bread and Starbucks, music classrooms, and shared meeting rooms.

Georgia Techs West Village, which will include shops, restaurants, classrooms and meeting rooms. PHOTO CONTRIBUTED

The new football stadium, which will have its first game on Aug. 31 against Tennessee State.

A new College of the Arts that offers 20 top undergraduate, graduate and non-degree programs in art, design, music, film, digital media, theater, etc.

New building for its growing Creative Media Industries Initiative.

Kennesaw State University

Students applying to KSU for fall 2018 can choose to apply through a non-binding, early action application or through a regular decision application, a process used by most competitive universities in the state. Meeting the minimum requirements will no longer guarantee a spot at the university.

New degree programs in computer engineering and cybersecurity.

The college is expanding its health science classes. For the first time, classes in human anatomy, microbiology and ethnobotany will be offered this fall to attract more students interested in pursuing careers as dentists, pharmacists, and medical doctors.

Interim president Harold Martin, a former valedictorian. The college is conducting a search process for a permanent president.

The university is breaking ground on the I.W.Ike Cousins for Science and Innovation. The center will have laboratory-classrooms, independent study labs, open study rooms and faculty offices.

The college has a new documentary filmmaking and photography majors beginning this fall. Both new majors are part of the Department of Art & Visual Culture, formerly the Department of Art & Art History.

In September, theyll open a facility to support the Center for Molecular Medicine. The state provided $17 million to support the project. The faculty for this center are working on cures and therapies for diseases such as diabetes, cancer and dementia.

Best quality of life: Emory University (No. 3), Agnes Scott College (No. 20) Great financial aid: Emory University (No. 15) Most conservative students: Berry College (No. 20) Most liberal students: Agnes Scott College (No. 12) Most LGBTQ-friendly: Agnes Scott College (No. 7) Lots of race/class interaction: Agnes Scott College (No. 20) Most beautiful campus: Berry College (No. 9) Most active student government: Agnes Scott College (No. 9) Best college dorms: Emory University (No. 8) Most religious students

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