StemCell Therapy

UTRECHT, NetherlandsEls van der Heijden, who has cystic fibrosis, was finding it ever harder to breathe as her lungs are filled with thick, sticky mucus. Despite taking more than a dozen pills and inhalers a day, the 53-year-old had to stop working and scale back doing the thing she loved best, horseback riding.

Doctors saw no sense in trying an expensive new drug because it hasnt been proven to work in people with the rare type of cystic fibrosis that van der Heijden had.

Instead, they scraped a few cells from van der Heijden and used them to grow a mini version of her large intestine in a petri dish. When van der Heijdens mini gut responded to treatment, doctors knew it would help her, too.

I really felt, physically, like a different person, van der Heijden said after taking a drugand getting back in the saddle.

This experiment to help people with rare forms of cystic fibrosis in the Netherlands aims to grow mini intestines for every Dutch patient with the disease to figure out, in part, what treatment might work for them.

Its an early application of a technique now being worked on in labs all over the world, as researchers learn to grow organs outside of the body for treatmentand maybe someday for transplants.

So far, doctors have grown mini gutsjust the size of a pencil pointfor 450 of the Netherlandss roughly 1,500 cystic-fibrosis patients.

The mini guts are small, but they are complete, said Dr. Hans Clevers of the Hubrecht Institute, who pioneered the technique.

Except for muscles and blood vessels, the tiny organs have everything you would expect to see in a real gut, only on a really small scale.

These so-called organoids mimic features of full-size organs, but dont function the same way. Although many of the tiny replicas are closer to undeveloped organs found in an embryo than adult ones, they are helping scientists unravel how organs mature and providing clues on how certain diseases might be treated.

In Australia mini kidneys are being grown that could be used to test drugs. Researchers in the US are experimenting with tiny bits of livers that might be used to boost failing organs.

At Cambridge University in England, scientists have created hundreds of mini brains to study how neurons form and better understand disorders like autism. During the height of the Zika epidemic last year, mini brains were used to show the virus causes malformed brains in babies.

In the Netherlands the mini guts are used as a stand-in for cystic-fibrosis patients to see if those with rare mutations might benefit from a number of pricey drugs, including Orkambi.

Made by Vertex Pharmaceuticals, Orkambi costs about 100,000 per patient every year in some parts of Europe, and its more than double that in the US, which approved the drug in 2015.

Despite being initially rejected by the Dutch government for being too expensive, negotiations with Vertex were reopened in July.

Making a single mini gut and testing whether the patient would benefit from certain drugs costs a couple of thousand euros. The program is paid for by groups, including health-insurance companies, patient foundations and the government.

The idea is to find a possible treatment for patients, and avoid putting them on expensive drugs that wouldnt work for them. About 50 to 60 patients across the Netherlands have been treated after drugs were tested on organoids using their cells, said Dr. Kors van der Ent, a cystic fibrosis specialist at the Wilhelmina Childrens Hospital, who leads the research.

Clevers made a discovery about a decade ago that got researchers on their way. They found pockets of stem cells, which can turn into many types of other cells, in the gut. They then homed in a growing environment in the lab that spurred these cells to reproduce rapidly and develop.

To our surprise, the stem cells started building a mini version of the gut, Clevers recalled.

Cystic fibrosis is caused by mutations in a single gene that produces a protein called CFTR, responsible for balancing the salt content of cells lining the lungs and other organs.

To see if certain drugs might help cystic-fibrosis patients, the medicines are given to their custom-made organoids in the lab. If the mini organs puff up, its a sign the cells are now correctly balancing salt and water. That means the drugs are working, and could help the patient from whom the mini gut was made.

Researchers are also using the mini guts to try another approach they hope will someday work in peopleusing a gene-editing technique to repair the faulty cystic-fibrosis gene in the organoid cells.

Other experiments are under way in the Netherlands and the US to test whether organoids might help pinpoint treatments for cancers involving lungs, ovaries and pancreas.

While the idea sounds promising, some scientists said there are obstacles to using mini organs to study cancer.

Growing a mini-cancer tumor, for example, would be far more challenging because scientists have found it difficult to make tumors in the lab that behave like in real life, said Mathew Garnett of the Wellcome Trust Sanger Institute, who has studied cancer in mini organs but is not connected to Cleverss research.

Also, growing the cells and testing them must happen faster for cancer patients who might not have much time to live, he added.

Meanwhile, Clevers wants to one day make organs that are not so mini.

My dream would be to be able to custom-make organs, he said, imagining a future where doctors might have a freezer full of livers to choose from when sick patients arrive. Others said while such a vision is theoretically possible, huge hurdles remain.

There are still enormous challenges in tissue engineering with regards to the size of the structure were able to grow, said Jim Wells, a pediatrics professor at the Cincinnati Childrens Hospital Medical Center.

He added the mini organs are far smaller than what would be needed to transplant into people and its unclear if scientists can make a working, life-sized organ in the lab.

There are other limitations to growing miniature organs in a dish, Madeline Lancaster at Cambridge University said.

We can study physical changes and try to generate drugs that could prevent detrimental effects of disease, but we cant look at the complex interplay between organs and the body, she added.

For patients like van der Heijden, who was diagnosed with cystic fibrosis as a toddler, the research has helped her regain her strength. Vertex agreed to supply her with the drug.

It was like somebody opened the curtains and said, Sunshine, here I am, please come out and play. she said. Its strange to think this is all linked to some of my cells in a lab.

See original here:
Lab-made 'mini organs' helping doctors treat cystic fibrosis - Business Mirror

Along with all the other consequences of poorly managed diabetes, patients who dont or cant control their sugars are more likely to lose their teeth because of severe periodontitis, an irreversible gum disease that attacks the tissues and bone around the teeth.

The connection has long been known. But exactly why it happens hasnt been so well-understood.

Now, a new study by University of Pennsylvania researchers that was supported by the National Institute of Dental and Craniofacial Research shows that unmanaged diabetes changes bacteria in the mouth so that microbes are more capable of causing disease. That means more inflammation of the gums and bone loss.

The findings on the oral microbiome, researchers hope, will help lead to better dental treatments for people with diabetes and anyone with periodontitis.

Penn Medicine

Dana Graves,D.D.S.

Studies done on the microbiome had been quite limited, says Dana Graves, a professor of periodontics at Penn Dental Medicine and senior author of the study.

Rather than only looking at the bacteria present, for the first time we examined the destructiveness of the bacteria by transferring them from a diabetic mouse to a normal germfree mouse, he said. We found that the bacteria from a diabetic animal created more inflammation and more bone loss than bacteria from normal mice.

About 47 percent of Americans ages 30 and olderand more than 70 percent of adults 65 and older have some form of periodontal disease, according to the U.S. Centers for Disease Control and Prevention. The most common is gingivitis, which causes bleeding of the gums and inflammation, but not irreversible loss of bone around the teeth. With proper dental hygiene, gingivitis is reversible.

Periodontitis an irreversible condition is much less common, affecting 15 percent to 30 percent of dental patients with gum disease and causing damage to soft tissues and destroying the bone that supports the teeth. People with poorly managed diabetes are about three times more likely to have this form of the disease.

In poorly controlled diabetes, there is greater inflammation of the gums, says Graves. You have a cycle where diabetes causes inflammation that changes the bacteria in the mouth, which causes more inflammation, which makes people with diabetes have a higher risk of periodontal disease.

In the study, Penn researchers compared the oral microbiome of healthy mice withanimals with diabetes. They found that the two groups had similar profiles before the diabetic mice developed hyperglycemia or high blood sugar levels. With high blood sugars, the oral microbiome of the diabetic mice shifted, forming a less diverse and more disease-causing community of bacteria.

The diabetic mice developed periodontitis, a loss of bone supporting the teeth, when the blood sugar levels rose, says Graves.

The next step was to uncover whether the microbial changes were responsible for the disease. Researchers tested this by transferring microbes from a diabetic mouse to a nondiabetic mouse. They found that the transferred bacteria of the diabetic animal created more inflammation and more bone loss in the normal mouse.

If you transfer normal bacteria from one mouse to another, it will create some bone loss, says Graves. But in this case, the normal mice who received microbes from the diabetic mice showed significantly more bone loss [42 percent] than mice who had received a microbial transfer from normal mice.

The researchers also found that certain inflammatory markers particularly IL-17, a signaling molecule important in immune response and inflammation were increased in diabetic animals. Higher levels of IL-17 in humans are associated with periodontal disease.

We thought if we blocked IL-17, it might prevent changes in the bacteria, says Graves. After injecting an antibody to IL-17 directly into the gums of the mice, we examined the bacteria and found that its composition had changed less and that it made the bacteria less pathogenic, proof that the greater inflammation associated with diabetes led to a change of the bacteria.

While injecting an antibody into the gums is not an option for humans, it suggests that if you can control inflammation, you could reduce the susceptibility of diabetics to periodontal disease. We need a way to target it a little more carefully, says Graves.

This article, said Renate Lux, professor of periodontics at the UCLA School of Dentistry, provides new and important understanding of the interplay between the microbiome and the host in patients with diabetes. Lux was not associated with the study.

In the future, Graves group hopes to explore whether changes observed in mice also occur in humans.

One takeaway from the study is the importance of rigorous oral hygiene for people with diabetes.

Its very important that patients with diabetes and periodontal disease keep both their physicians and periodontists informed of changes in either condition, says Terrence J. Griffin, president of the American Academy of Periodontology. People with diabetes should consider a regular oral hygiene routine, which includes brushing twice a day, flossing regularly, and checking in with a periodontist at least once a year or if they notice changes in their gums or teeth.

Periodontal disease is largely preventable, said Graves. If a person maintains good oral hygiene and good glycemic control with a hemoglobin A1c level of under 7 they will have less likelihood of developing periodontitis.

mice30@comcast.net

Published: September 1, 2017 3:01 AM EDT

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What diabetic mice can teach us about keeping teeth healthy - Philly.com

Every 21 seconds another person in the United States is diagnosed with diabetes, according to the ADA. That's 4,110 people in America diagnosed with the disease every 24 hours. Type 2 diabetes accounts for 90 to 95 percent of all those cases. The risk for developing the disease also increases drastically in people age 45 and older, and after age 65 it increases exponentially.

There has also been a troubling rise in the number of adolescents developing both prediabetes and diabetes. Weight has a lot to do with it. Among adolescents, ages 12 to 19, about 1 in 5 are considered to have obesity, and about 1 in 11 (9.1 percent) are considered to have extreme obesity, according to the National Institute of Diabetes and Digestive and Kidney Diseases.

"Nutrition for adolescents is the cornerstone of treatment," Cefalu said. "People need access to adequate nutrition, and you have to get that information in their hands."

Testing for prediabetes in children and adolescents should be considered for those who are overweight or obese, and who have two or more additional risk factors for diabetes, including having a family history of type 2 diabetes, or who are African American, Native American, Latino, or Asian Pacific Islanders. Nearly half of Asian and Hispanic Americans with diabetes are undiagnosed.

Health-care specialists say getting people to change their behavior isn't easy. "Telling people to lose weight does not give them enough information. It is not a message that helps and supports them," Albright said.

To teach people how to change and maintain a new set of lifestyle habits, the CDC is also promoting its National Diabetes Prevention Program, which was initiated in 2010.

Just by participating and staying in the program, prediabetics can lower their risk of developing type 2 diabetes by as much as 58 percent over three years, and by 71 percent for people over age 60. "We want to help them determine what is realistic and doable so they can make real life changes that are sustainable," Albright said.

A DDP program can cost as much as $500 a year. (The CDC-recognized organizations delivering these programs determine the cost, which can vary depending on factors such as the size and experience of the organization offering it.) Diabetes prevention programs are growing in number and participation, but are still underutilized, according to the ADA.

See the article here:
Type 2 diabetes may hit 84 million Americans, and they don't know it - CNBC

LISTEN: You haven't heard Darlington Nagbe like this before. In a relaxed interview from the USMNT hotel, the Timbers man explains why the middle third is his favorite, why his family comes first and what representing his country means to him. Taylor Twellman joins for the complete US-Costa Rica preview. Subscribe now and "Like" our Facebook page so you never miss a show! Download this episode!

Millions of soccer fans across the country tuned in as Jordan Morris dramatically scored the tournament-winning goal of the Gold Cup last month. And doubtless, USMNT fans will turn their attention back to him when the US host Costa Rica in tomorrows World Cup qualifier at Red Bull Arena (6:30 pm, ESPN, Univision, UDN).

But none of them were watching just nine days before that Gold Cup win when Morris, unassumingly and without fanfare, did another cool thing while wearing a US shirt.

Following the end of a USMNT practice at the University of Pennsylvanias Rhodes Field, Morris met Liam Fuller, the teenage son of Penns soccer coach, Rudy Fuller. Like Morris, Liam is a Type 1 diabetic, and reached out to Morris for guidance and advice as to how to play soccer while managing the disease.

Morris met young Liam Fuller this summer at USMNT training.

As he has done with other kids, the 22-year-old Seattle Sounders and US national team star struck up a relationship with Liam via email before they met in person for the first time, drawing huge smiles from an awe-inspired 14-year-old.

"I want to give back, Morris told MLSsoccer.com by phone shortly before rejoining the US national team ahead of Fridays World Cup qualifier. Ive been very blessed with being able to play.

For me, when I was growing up, I looked at guys who had diabetes that were playing professional sports, like Jay Cutler and Adam Morrison, and I know I would have loved to have been able to speak with them. But I definitely looked at them as an inspiration. so whenever someone reaches out to me I try to get back to them and I hopefully can be that inspiration to them with whatever their dream is whether its playing professional sports or anything else.

Morris has made it a point to mentor as many diabetic kids as he can, meeting a new one on the field after every Seattle Sounders home game. Hes been to hospitals to host talks on diabetes, and is in the process of starting his own foundation. Meanwhile, hes expanded his reach beyond Seattle by meeting kids at USMNT camps, too.

Earlier in July, he met a nine-year-old diabuddy named Aiden, giving him gifts, introducing him to Landon Donovan and Stuart Holden, and bonding over the kind of fruit snacks they need to eat before games in a video released by US Soccer. Not long after, when the cameras werent rolling, he had a similar conversation with Liam Fuller as his father proudly looked on.

Jordans been instrumental in helping my son along, so it was really nice to connect those two, said Rudy Fuller, the longtime Penn head coach and fixture in the Philadelphia soccer community. It was the first time they met in person, and Jordan was phenomenal. Hes an unbelievable guy, really generous with his time. My son walked away on cloud nine.

While the best part for Morris is seeing the kids faces light up, he also notices how much relief his talks give to parents like Rudy Fuller, who are naturally worried about how the disease may affect their son.

Morris does his best to ease those concerns, pointing to what hes been able to accomplish as proof that diabetes hasnt held him back. But he does it an honest way, opening up about some of his own struggles, listening candidly to those facing younger kids, and offering encouraging tips about utilizing some of the new technologies that have been developed. (He wears a glucose monitor on his hip that sends a blood-sugar update to his phone every few minutes).

Especially when youre younger for me too its hard to deal with because people really think its going to get in the way, said Morris, who was diagnosed with Type 1 diabetes at the age of nine. But I just try to get that out of their heads. Ive gotten some responses from parents that have been very thankful.

I cant say enough about Jordan and his generosity and the type of person he is, said the elder Fuller. Hes obviously got a really bright future. To do what hes done on the field and to have the future he has and to still be as grounded as he is, its very impressive.

It certainly seems quite clear that Morris has a bright future ahead of him on the soccer field, where in the last two years alone hes won an NCAA title, an MLS Cup and the Gold Cup.

And now, as he sets his sights on even bigger things like getting to a World Cup, hes ready to step up his off-the-field mission too.

Ive been given this platform of being able to play soccer and I want to use it to do some good, Morris said. So trying to reach as many younger diabetics as I can is important to me.

Hopefully theres a cure for diabetes at some point. Until then, I definitely want to keep speaking out and trying to be an inspiration to as many as possible."

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Jordan Morris expands his Type 1 diabetes mentorship at USMNT camps - MLSsoccer.com

About 350 supporters of the William Sansum Diabetes Center turned out on Saturday, August 26 for the 15th annual Taste of the Vine fundraiser held on the idyllic Summerland bluff grounds of QAD Inc. The center netted about $125,000 from the event for its research, care, and educationprograms.

Guests mingled on the expansive deck and grounds at QAD, enjoying the panoramic ocean views while sampling offerings from fine food and beverage purveyors. Cutlers Artisan Spirits, Santa Barbaras only distillery, was among the 37 food and beverage purveyors serving up tastes, with proprietor Ian Cutler personally serving his delectablespirits.

In her role as Mistress of Ceremonies, Executive Director Ellen Goodstein exuded the passion she brings on a daily basis to this critically important organization. She introduced Charles Mattocks, executive director of Reversed, a reality show about diabetes that premiered this summer on the Discovery Life Channel. Mattocks shared his enthusiasm for raising funds for diabetes, a disease that doesnt get the attention itdeserves.

Board President Dr. Alex DePaoli shared some of the centers exciting research projects, including developing an artificial pancreas. After 10 years of effort, the first device went on the market earlier this year. This hybrid artificial pancreas measures the blood sugar continuously and automatically adjusts basal insulin levels. Work continues on developing more advanced features. DePaoli also referenced the centers work with diabetes and pregnancy, which has lead to significant improvements in the health of both mothers andinfants.

Lastly, he shared his excitement for the Mil Familias Project, which aims to reduce diabetes among the Latino population. For the next 10 years, 1,000 Latino families in Santa Barbara County will be followed to help determine why the incidence of diabetes is higher among this ethnic group.The project is run by a consortium led by the center that includes UC Santa Barbara, the Santa Barbara Neighborhood Clinics, Westmont College and Lilly Diabetes. With $1.25 million in seed funding, the project just began last month. In a recent study of 400 Latino residents in the County, 7% of participants not knowing they had the disease tested positive and a whopping 42% were pre-diabetic, which means they have a one in three chance of developing diabetes in the next three to fiveyears.

Teen star Jackson Gillies, a Type 1 diabetic himself, wowed the crowd with a couple of his songs, and in between, shared his gratitude to the William Sansum Diabetes Center for all that itdoes.

The center (formerly known as the Sansum Diabetes Research Institute), is not part of Sansum Clinic, but was founded by Dr. William Sansum in 1944. It is a leading research institution thatalso provides direct clinical care to patients, including free services to thousands of Santa Barbara County residents. It also offers free education programs to the 50,000 county residents impacted by thedisease.

For more info about the center, go to sansum.org.

If viewing this from a mobile device, click on Desktop site for more photos. Send event invites to Gail atsociety@independent.com.

By GailArnold

Boardmember Wayne Hewitt, Boardmember Joan Arnold, Director of Research and Innovation Dr. David Kerr, and Boardmember GeorgeEmerson.

Treasurer Anthony Castillo, Boardmember and Immediate Past President Sandra Svoboda, and.Boardmember and lead event sponsor CurtCruthirds.

Read more here:
Sansum Diabetes Center Holds Taste of the Vine - Santa Barbara Independent

Welcome to this week's Chutes and Ladders, our roundup of hirings, firings and retirings throughout the industry. Please send the good wordor the badfrom your shop to Eric Sagonowsky (email) or Angus Liu (email)and we will feature it here at the end of each week.

SanofiPeter Guenter leaves as global diabetes and cardiovascular head.

Guenterspent 22 years with Sanofi and was most recently the French drugmakers EVP and global diabetes and cardiovascular chief, but he has left that post to join Spanish pharma Almirall as its CEO, taking over from Eduardo Sanchiz, who has been Almiralls CEO since July 2011. Sanofis diabetes business is not without trouble; its lead blockbuster Lantus is facing biosimilar competition, and a much-anticipated cholesterol drug, Praluent, hasnt been doing so well as expected and is facing patent challenge by Amgen. CEO Olivier Brandicourt, who lifted Guenter to his current position during a management rejig, will look after that section of business until Guenters replacement is found. After a 2014 revamp that included a $2.2 billion respiratory divesture to AstraZeneca, Almirall now focuses on dermatology. FiercePharma

PhRMARichard Moscicki, M.D., joins PhRMA as CMO and EVP.

Its almost a full life science journey for Richard Moscicki, M.D. With a medical degree from Northwestern University and after completing a fellowship at Massachusetts General Hospital in immunology and immunopathology, he remained on staff at MGH and on the faculty of Harvard Medical School from 1979 until 2013. He joined Genzyme in 1992 and spent nearly two decades with the company, most recently holding the position of SVP, head of clinical development and CMO, responsible for worldwide global regulatory and pharmacovigilance matters, as well as all clinical and medical affairs for the company. He then joined the FDAs Center for Drug Evaluation and Researchas deputy center director for science operations. And starting from this October, he will be joining nongovernment industry organization PhRMA as CMO and EVP. Release

Vir BiotechnolgyAlpna Seth, Ph.D., became COO.

In the most recent episode of executive exodus at Biogen, the biotechs SVP and global head of the biosimilars unit, Alpna Seth, Ph.D., quietly left the company in July and joined her former boss, Biogens former CEO George Scangos, at his San Francisco-based startup Vir Biotechnolgoy. Seth was with Biogen since 1998 and helped launch Biogens India office. Scangos landed the top job at Vir In January, shortly after he was replaced by Michel Vounatsos at Biogen. With lead investors including ARCH Venture Partners and the Bill & Melinda Gates Foundation, Vir is focusing on infectious diseases for which solutions are nonexistent or inadequate. Boston Business Journal story

> Christian Meyer, M.D., Ph.D.,uniQure's four-year CMO until June, will joinTherachon as its CMO, helping to lead its R&D efforts in rare diseases. FierceBiotech

>North Carolina-based CDMO Avista Pharma appointed industry veteran Eric Evans as CFO. Release

>Deirdre BeVard, whohas held leadership roles in clinical affairs and development operations for the past 25 years,joined Elligo Health Research, a clinical research infrastructure provider,as COO. Release

>Health intelligence provider Outcome Health hired Nandini Ramani, formerly VP of engineering at Twitter, as its chief engineering officer tostrengthen the companys technology platform. Release

>Heart medtech company Abiomed announced that its CFO Michael Tomsicek has left the company to pursue other interests, and thatitsformer CFO, Robert Bowen, would step inuntil a permanent replacement is found. Release

> Bone Therapeutics namedJean-Luc Vandebroek as CFO; he will replace Wim Goemaere, whois leaving the company to take up a senior position within a not-for-profit organization.

Original post:
Chutes & LaddersSanofi's diabetes and CV head Guenter jumps ship to Almirall - FierceBiotech

AUSTIN (KXAN) Sending a child off to pre-kindergarten for the first time can be scary as a parent.

For Ted Hennessy and his wife, the risk is even higher.

Their 4-year-old daughter Esme has type 1 diabetes, which means she is insulin dependent to keep blood sugar at a healthy level. She was diagnosed with the disease at the age of two and a half after nearly going into a coma.

Thursday, Esme spread out her medical supplies on the floor in her living room to walk us through how she checks her blood sugar three times a day.

The pinkies are the best ones, Esme said as she pricked her finger and then placed a spot of blood on a glucose test strip.

One four nine, she said reading the number on the screen and holding up the handheld device to show her dad.

Esmestarted pre-k at Becker Elementary School a couple of weeks ago.The plan was for Hennessy to spend the first few days on campus making sure everyone was on board with his daughters care. Nearly two weeks in, and he is still there.He has been posting upin the school library every single day because he says he is not yet comfortable putting Esmes health in someone elses hands.

We dont have any consistency right now with her care, said Hennessy.

On Monday, Aug. 21, the first day of school, he briefly saw a nurse, but it was her last day with Austin ISD. Hennessy says the next nurse came toward the end of the week. Most of the time hes seen a variety ofstudent health assistants.

Theres been frustrating days with [SHAs] because they dont know what to do or not comfortable going through the process with us, said Hennessy.

After taking their concerns to the superintendents office, a nurse has been on campus over the last couple of days. Hennessy has also been told a full time student health assistant is starting soon.

Monday night, a handful of parents voiced their concerns to the school board. Several accused the school district of violating state law by not telling them about school nurse changes.

Austin ISD sent KXAN the following statement:

Austin ISD remains cognizant of the Texas Education Code when implementing any programming or service within the district. The addition of telemedicine increases the variety of how health services are provided and as such, does not fundamentally change the services provided in AISD. Nursing services are still available at all campuses, whether students will be seen by registered nurses or student health assistants supervised by nursing staff.

Link:
Dad posting up at daughter's school to oversee diabetes care - KXAN.com

Egg donors and sperm donors are the angels sent from above. Theyre the answer to prayers for a baby for many people trying to grow their family. Singles, gay couples, and straight couples suffering from infertility alike all turn to the generosity of gamete donors. But when it comes to whether a donor should enjoy anonymity or not, the debate continues to rage on.

International Bewilderment. South Africa is the latest country to take the issue seriously. The South African Law Reform Commission released a discussion paper open for public comment through the end of this month. It concerns the question of whether the law should entitle donor-conceived children the right to know their biological roots.

Right to Know Ones Biological Identity. If South Africa opted to take this path, it would not be the first country to ban anonymity for sperm and egg donors. Australia, Britain, and Sweden all give donor-conceived children the legal right to know the identity of their genetic parents. The South African discussion paper argues that children unable to know their genetic identity may suffer from genealogical bewilderment. (The paper doesnt mention Jon Snow by name, but I assume that is who the researchers have in mind.) The argument continues that some children need to know their genetic identity for normal psychological development. (Hmm, isnt Bran the creepy one these days?)

Pro for Anonymity. Its easy to say that a donor-conceived person should be able to know their identity. Who would object to that? But its not so simple. Fertility doctors object, for instance. And people trying to conceive using a donor. And, of course, donors themselves. The spokesperson for South Africas Infertility Awareness Association, Meggan Zunkel, reported that most donors do not want to be contacted by their donor-conceived offspring, and that many people considering turning to an egg or sperm donor would not do so without the promise of donor anonymity.

Dr. Paul Le Roux, the spokesman for the African Society for Reproductive Medicine and Gynecological Endoscopy, elaborated that non-disclosure can be essential for some parents as they need to keep the gamete donation private for specific reasons. This can be important in cultures where gamete donation is less accepted, or where gamete donation is not accepted for religious reasons for example the Muslim Community in South Africa. Le Roux further noted that: Studies on children who have not been informed of their biological parents show that their development has not been harmed and that they are also doing psychologically well.

Is This Debate Moot? Many argue that there is no true choice to remain anonymous. Anonymity, in other words, is dead.Wendy Kramer, the founder of the Donor Sibling Registry (www.donorsiblingregistry.com) a website that connects donor-conceived children with their half-siblings points to a quote from Dr. JLH Ever, Editor-in-Chief of Human Reproduction: All parties concerned must be aware that, in 2016, donor anonymity has ceased to exist.

Kramer explains, if you are planning on being an anonymous donor, it is important to understand that because of commercial DNA testing and Internet search engines, the likelihood of your remaining anonymous in the future is not likely. Kramer feels strongly that egg and sperm banks need to stop promoting the false idea of anonymity to their donors. She notes that just this morning there was a post to the Donor Sibling Registry Facebook page, Exciting news. We are only a few weeks into the DNA journey but I think I have identified my childs donor. Kramer notes that this type of message is very common. Anonymous donors can be found.

Its just that easy. Its time to put the debate to bed and focus on educating donors of the reality of anonymity. Now if only Jon Snow had access to a 23andMe kit. He could have saved us 7 seasons of wondering.

Ellen Trachman is the Managing Attorney of Trachman Law Center, LLC, a Denver-based law firm specializing in assisted reproductive technology law, adoption, and estate planning, and Co-Director of Colorado Surrogacy, LLC, a surrogacy matching and support agency. You can reach her at babies@abovethelaw.com.

Originally posted here:
Do You (And Jon Snow) Suffer From Genealogical Bewilderment? - Above the Law

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SANTA MONICA, Calif., Aug. 31, 2017 /PRNewswire/ --Pot Valet is a leading provider of premium-grade cannabis in California. With its medical marijuana delivery service growing to every city in the state, and soon the whole country, the company offers patients safe, legal, and discreet access to their medicine, eliminating their need to visit a cannabis dispensary.According to Pot Valet, intellectual property rights for cannabis strains are a growing, yet dangerous trend.

Companies are creating their own marijuana strains and protecting them with trademark laws. Profits are taking priority over the rights of patients to access genetically pure medication. A trademark is a visual cue, such as a symbol, phrase, or word that businesses use to identify and distinguish their products from those of their competitors. While this may be a branding advantage, it has consequences.

The purpose of patenting strains and trademarking them is to create an association in consumer's minds between brands and products. It gives the owner exclusionary rights to it, which means that he or she can prevent others from using it, or have confusingly similar marks of their own. Establishing whether a mark infringes on another, hinges on the likelihood of confusing consumers over the product's source.

A trademark environment in the cannabis industry can endanger the genetic purity of the marijuana plant. Companies are spending vast sums of money creating strains nobody will ever hear about in an already strain-flooded market. If the goal of strain breeding is to trademark varieties for future profits, there is a real risk of monopolies forming that can undermine the quality of genetically pure strains.

Creating strains willy-nilly will create chaos in the marijuana industry. Not only will finding the correct strains become an overwhelming task for medical patients, but many 'breeders' will also not comply with proper breeding and registration practices. Creating quality strains requires a database of information about parents, chemical levels, and other crucial characteristics of every strain created.

Overwhelming the market with unregistered strains will diminish the value of this knowledge. Unknown hybrid varieties can confuse entire lineages, reduce their quality, and lead to cross-pollination issues. Pot Valet stands by original household-name cannabis strains. Lab-tested and verified by SC Labs for medical quality, all of its products have a platinum-grade, genetically pure guarantee.

Pot Valet is an online cannabis dispensary based in Santa Monica serving patients across California. Its large selection of products consistently meet regulatory requirements for the highest quality medical marijuana and the company only delivers to valid medical patients.

Patients across California can now get their orders in less than 45 minutes. Those still using the company's Overnight Delivery Service get their medicine the next day. Pot Valet is expanding its Immediate Delivery Service throughout the United States and soon, patients everywhere will enjoy the same benefits.

To order marijuana delivery through Pot Valet, patients must upload a copy of their Medical Marijuana Recommendation and a government I.D. photograph. The company is fully compliant with state laws and regulations.

DISCLAIMER: The content of this press release is the opinion of Pot Valet, written from the company's perspective.

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Because gene therapy involves making changes to the bodys set of basic instructions, it raises many unique ethical concerns. The ethical questions surrounding gene therapy include:

How can good and bad uses of gene therapy be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

Current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed on to a persons children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the inserted gene to be passed on to future generations. This approach is known as germline gene therapy.

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they cant choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.

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What are the ethical issues surrounding gene therapy ...

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 Europe and China.

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 previously, for 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. 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 estimates the cost of its treatment at $475,000 per patient. 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, 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 aroused hopes that gene therapy might finally fulfillexpectations. Researchers recently reported that they employed CRISPR 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 producing designer babies with enhanced physical and mental traits. But as Science noted recently, CRISPR poses some of the same risks as gene therapies. CRISPR still has a long way to go before it can be used safely and effectively to repairnot just disruptgenes in people.

So to answer the question posed in the headline: No, the gene-therapy era has still not arrived.

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

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.

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

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

Campbell Soup Co. CPB, -0.22% had a rough quarter, but the company is facing a dire long-term problem.

Novartis AG NVS, -0.08% 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.

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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, +0.11% was called the weakest major U.S. retailer this week by Moodys Investors Service. But T. Rowe Price Media and Telecommunications Fund PRMTX, +0.69% 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, +0.05% right now.

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

Want more from MarketWatch? Check out our Personal Finance Daily or other newsletters, and get the latest news, personal finance and investing advice.

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

MINNEAPOLIS (WCCO) Colin Cooley of Burnsville beat lymphoma four years ago, but the lymphoma came back in a different spot two years later.

Chemo wasnt cutting it, Cooley said. It was keeping it in check, but it wasnt getting rid of it.

He decided to undergo a clinical trial at the University of Minnesota. He received a gene therapy called CAR-T and is now cancer-free.

The FDA approved CAR-T Wednesday as the first type of gene therapy in the United States.

The treatment has been called a breakthrough in the fight against cancer. It is only approved right now to treat children with acute lymphoblastic leukemia, but doctors are excited about its potential for other cancers and diseases.

Doctors at the University of Minnesotas Cancer Day at the Minnesota State Fair called the therapy a major leap.

(credit: CBS)

Were able to take a patients own cells and turn them into something that can actually attack their specific cancer, said Dr. Edward Greeno, medical director of the University of Minnesotas Masonic Cancer Clinic. Many people have referred to this as living cancer because were taking live cells and turning them into your treatment.

First, a patients blood is drawn and their T-cells, or immune cells, are separated out. Those T-cells are then sent to a laboratory to be genetically modified and reprogrammed to zero in on the cancer.

Those modified cells are then multiplied in the lab before being returned to the patient via blood. They are essentially revved-up cells that are missiles for the cancer.

In one significant study, 83 percent of the patients who received CAR-T went into remission.

This treatment is expected to be offered for lymphoma patients next year. Dr. Greeno says it could be decades, though, before its offered to patients with other types of cancer.

Right now, its expensive almost $500,000 and used mostly on patients when other methods of treatment, like chemotherapy, have failed.

Before I didnt know if Id be here in three or four or five years, I didnt know, Cooley said. Now I feel like I have a new lease, some minor issues, but a new lease on life, and thats pretty exciting.

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How Does Gene Therapy Work? - CBS Minnesota / WCCO

Nightstar Therapeutics has filed to raise up to $86 million in a Nasdaq IPO. The money will equip Nightstar to complete a phase 3 trial of its choroideremia gene therapy and advance two other eye disease candidates through early-stage clinical studies.

London-based Nightstar, also known as NightstaRx, is set to move the choroideremia asset into phase 3 in the first half of next year. The therapy, NSR-REP1, is advancing into the 140-patient trial on the strength of data on 32 subjects treated in investigator-sponsored studies. Those trials found 90% of patients either maintained or improved their visual acuity in the year after receiving the gene therapy.

Given choroideremia causes currently-untreatable progressive vision loss, Nightstar sees the data as supporting further development. The asset is moving forward with a fairly clean safety profile in the 50 people treated to date. Investigators have seen one adverse eventtransient intraocular inflammationthat may have stemmed from treatment with NSR-REP1.

Challenges await Nightstar as it scales up for the phase 3 trial and potentially commercial sales, though. The biotech acknowledges the administration of NSR-REP1 requires significant skill and training, potentially creating a bottleneck to use of the gene therapy. And as a small, unpartnered player in a new field, any number of events could knock it off course.

What Nightstar does have is a head start. Spark Therapeutics has the most advanced challenger to NSR-REP1 but its program is yet to move past phase 1/2. The field is similarly clear for Nightstars follow-up candidate NSR-RPGR, which moved into the clinic just ahead of MeiraGTxs rival X-linked retinitis pigmentosa gene therapy. AGTCs Biogen-partnered candidate is close behind.

Nightstar has reached this point using money from a succession of private rounds, starting with the 12 million Syncona invested when the biotech spun out of the University of Oxford in 2014. The biotech pulled in a further 5 million when it named former Johnson & Johnson VP David Fellows as CEO early in 2015. A $35 million series B round followed late in 2015. And Nightstar broadened its investor base and raised a further $45 million in a series C round a few months ago.

Along the way, Nightstar has built out a team in preparation for the broadening of its clinical trial program and life on public markets. Last month, Ex-Pfizer clinical lead Tuyen Ong, M.D., left PTC Therapeutics to serve as chief development officer. And in April, Nightstar hinted at its IPO plans by recruiting the man who led Intercept Pharmaceuticals repeated public raises, Senthil Sundaram.

The question now is how receptive public investors are to gene therapy biotechs. The companies in the sector to go public to date have delivered mixed returns, with the successes of bluebird bio and Spark offset by the steady decline of uniQure and implosion of Dimension Therapeutics.

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Nightstar files for $86M IPO to fund gene therapy trials - FierceBiotech

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Dr. Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at C.S. Mott Children's Hospital in Ann Arbor, works with Maryam Rasheed of Macomb Township. Maryam was part of a clinical trial using gene therapy to successfully treat her leukemia.(Photo: Sophie Masson/Michigan Medicine)

The U.S. Food and Drug Administrationapproved on Wednesdaythe first-ever gene therapytotreat children and young adults withleukemia.

Called Kymriah, but better known as CAR T-cell treatment, the therapy is being hailed by doctors as revolutionary. Itinvolves genetically modifyinga patient's own T-cells, which thencantarget and kill a form of acute lymphoblastic leukemiacells.

This new treatment has the potential to change the face of cancer therapy for years to come, not just in childhood acute lymphoblastic leukemia but in other cancers in which a patients own T-cells can be collected, genetically modified and redirected to kill a patients tumor," said Dr.Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at the University of Michigan's C.S. Mott Children's Hospital. Mottwas one of a few hospitals nationally to take part inclinical trials of the treatment.

"This allows us to turn patients own cells into a powerful weapon to fight the disease a weapon that does not rely on chemotherapy but takes a whole new approach to attacking childhood leukemia, Yanik said.

The CAR T-cell treatmentoffers new hope for children like Maryam Rasheed, 10, of Macomb Township.

Maryam was diagnosed with B-cell acute lymphoblastic leukemia at age 4, when her family was seeking refuge from religious persecution in Turkey, said Maryam's mother, Asmaa Rasheed.

Maryam Rasheed (right) with her brother, Rashid, and sister Samantha. Maryam, 10 of Macomb Township, survived acute lymphoblastic leukemia.(Photo: Rasheed family photo)

"My country is Iraq," Asmaa Rasheedsaid. "It wasnt safe. We are Christian. It was so hard over there in Baghdad. We run away to Turkey.

"We take her to hospital the first timebecause ... she stopped eating, stopped walking, stopped talking. We bring her to emergency. The doctor decided to take her bone marrow to do tests. Then the results came back, and she have leukemia."

Maryam underwent her firstchemotherapy treatment in Turkey.

"Over there, it was so hard," Rasheed said. "The doctors dont speak English over there. We know English a little bit. We speak Arabic."

Maryam Rasheed of Macomb Township undergoes treatment for acute lymphoblastic leukemia. She is now in remission.(Photo: Rasheed family photo)

Rasheed stayed with her daughter for two months in the Turkish hospital. A few months later,the Rasheed family was able to immigrate to the U.S. and settled in Michigan.

But Maryam's cancer returned. She was treated at Children's Hospital of Michigan with more chemotherapy and radiation. In 2013,her younger brother, Rashid, proved to be a match for a bone marrow transplant.

Still, the cancer wouldn't relent.

The Rasheed family learned of a clinical trial for CAR T-cell therapy under way atMott. It was the family's last chance,Rasheed said.

Maryam Rasheed, 10, of Macomb Township holds up her arms joyfully. She's surrounded by her sister Samantha (left), brother, Rashid, and baby sister Annabell.(Photo: Rasheed family photo)

"There was nothing to do," her mother said."In Detroit, there was chemo, radiation, bone marrow transplant. It returned back three times. She lose her hair three times. It was so hard for her and my family."

She remembers the date Maryam started the clinical trial at Mott: Dec. 17, 2014. Maryam spent Christmas and her seventh birthday in the hospital.

"I think we waited like 100 days,I dont remember exactly, and they did a bone marrow test, and the medicine, it work!" Rasheed said.

"It was like a dream, you know, like light coming from far away when youre in the dark. Theres nothing else we could do. But the CART-cell was like a shining light from far away."

Maryam has been in remission two years, andis starting fourth grade next week at Shawnee Elementary School in Macomb Township.

"Now, shes start her life, and doing everything a little kid is doing," said Rasheed, who says she hopes the treatment helps other children, too.

So does Yanik.

"Acute lymphoblastic leukemia is the most common form of cancer in children, accounting for approximately25% of all childhood cancers," Yanik said. "This particular therapy utilizes a childs own immune system to target their leukemia."

Theclinical trials focused on the 15% to 20% ofchildren whoseB-cell acute lymphoblastic leukemia had either relapsed or who had residual leukemia cells in their bone marrow after treatment.

"Historically, such patients would have an estimated cure rate of approximately 10%," Yanik said. "The two trials were groundbreaking. In the most recent trial, 52 of 63 patients with childhood leukemia successfully entered complete remission with this therapy."

Novartis Pharmaceuticals Corp. got the FDA approval for the gene cell therapy, whichinvolves drawing blood from childrenwith B-cell acute lymphoblastic leukemia. The T-cellsin the child's blood are thenshipped to a lab where they are genetically engineered so theywillseek outa particular protein in the leukemia cells and attack. Patients are then infused with the modified blood, and the T-cells go to work to find and kill the leukemia.

The New York Times reported Wednesday that the therapy will cost $475,000 for the initial treatment, with additional treatments administered at no cost.

Although 83% of the children in the clinical trials for CAR T-cell therapy went into remission, Yaniksaid it's too early to tell howcurative treatmentswill prove in the long run. And, its use will be limited to only a few medical centers in the U.S.

"The University of Michigan is the only site in the state and within this region that is licensed to administer these cells for childhood leukemia," he said.

Offering the treatment at a large medical center like U-Mis essential, said Dr. Rajen Mody,a pediatric oncologist at Mott, because of the severity ofpotential side effects.

"It can cause serious side effects, especially within the first 21 days," said Mody, who is Mott's director of pediatric oncology. "Patients can have high fevers, bleeding complications, trouble breathing, infections. ... Thats why a hospital like the University of Michigan is the ideal place. ... Patients who undergo this treatment are usually so sick after an infusion of the CAR-T cells, that they can't be safely treated at smaller hospitals."

Dr. Rajen Mody, a pediatric oncologist at the University of Michigan's C.S. Mott Children's Hospital.(Photo: University of Michigan)

Yanik is hopeful that successful treatment with CAR T-cell therapy in children with leukemia will open the door for similar therapies targeting other cancers.

"Aseparate CAR T-cell trial targeting diffuse large-cell lymphoma was recently completed with the results in that clinical trial now under review at the FDA," he said. That trial alsoincluded adult patientsat the University of Michigan.

Mody called the gene therapy revolutionary.

"This is clearly a life-saving and potentially curative therapy," he said."Its being tested in other types of leukemia and solid tumors. Its too early to say whether its going to work as well for other cancers.... We are not there yet."

Still, he said, it's made all the difference for Maryam and her family.

"She was one of the lucky ones coming from Iraq, and with all the things she has survived. And then coming here and surviving this,... she clearly has some goodluck.

"I think she should do very well. Patients who actually survive the first six months and still have CAR T-cells detected in their systems tend todo very, very well."

Contact Kristen Jordan Shamus: 313-222-5997 or kshamus@freepress.com. Follow her on Twitter @kristenshamus.

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First gene therapy to treat cancer gets FDA approval; UM only Michigan hospital to use it - Detroit Free Press

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KANSAS CITY, Mo. -- Lucas Novick, 27, has been in a battle with leukemia since his freshman year of college.

"I was having headaches that were so bad that they were causing vomiting pretty regularly and I couldn`t see straight well enough that I felt safe driving myself to school," Novick said.

Since 2009, Novick has endured a number of treatments including chemotherapy and a bone marrow transplant. The treatments have taken a physical and mental toll on Novick's body.

"The transplant that was supposed to save my life also nearly took it from me," Novick said. "The damage chemotherapy did to my body when I was first treated in 2009 and 2010 was such that I was walking with a cane after my 21st birthday. It did so much damage to my hip joints that they were replaced in 2011."

But after Novick's leukemia returned for a second time, he went to Children's Mercy Hospital where doctors were performing an experimental treatment.

"The approval of the CTL019 product for pediatric patients with relapsed refractory acute lymphoblastic leukemia is really exciting for us," Doctor Doug Myers, of Children's Mercy Hospital, said. "We`ve spent a lot of time working on ways to get the immune system into the fight against cancer because we think it can decrease toxicity, decrease the amount of chemotherapy and radiation that we use for these cancers."

Dr. Myers said the treatment helped Novick, a musician, back onto the stage and has held his leukemia awayfor two years.

"Those are really special rewards for us in this field that have seen so many failures of this type of therapy in the past. To see this go forward, move forward, do well enough for a pharmaceutical company will pick this up and take it the rest of the way, that`s a really special time for us," Dr. Myers said.

While doctors believe it's too early to call the new treatment a cure, many agree this is the first step to a new generation of cancer treatment.

"I know at the end of the day that this is the future of medicine," Novick said.

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Man describes new FDA-approved gene therapy for leukemia that ... - fox4kc.com

Erlanger hopes to bring the treatment to the patients who need it most within the next few years . (Coutesy: WTVC)

Wednesday, the Food and Drug Administration cleared the way for a ground breaking cancer treatment in the United States.

It's a gene therapy treatment named CAR-T therapy.

Dr. Meghann McManus is a Pediatric Hematologist Oncologist at Erlanger in Chattanooga.

On Thursday, Dr. McManus described the treatment as the "first gene therapy treatment for any type of cancer in the pediatric world or adult world."

In layman's terms, Dr. McManus said the treatment allows doctors to remove a patients "T-cells, which are a type of white blood cell," send them to a lab "where their genetically modified to fight their certain type of leukemia."

"It uses the patients own immune system, their own cells from their own body, to fight their leukemia. Which is not something that we do with any other disease," Dr. McManus said.

At this time, Dr. McManus said CAR-T therapy is approved for "children that have relapsed or with refractory disease."

"We may not be able to stop cancers from happening... but if we could treat it in a way to get rid of the cancer without big side effects or tolls on patients, it would change the way we do our job," Dr. McManus said.

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Local pediatric oncologist hopeful new gene therapy will save lives - WTVC

A new study has discovered a genetic mutation on growth hormone receptors that they believe could expand our lifespan by an average of up to 10 years.

A new study led by Prof. Gil Atzmon head of the Laboratory of Genetics and Epigenetics of Aging and Longevity at the University of Haifa and his team have discovered a genetic mutation on growth hormone receptors that they believe could expand our lifespan by an average of up to 10 years. "We were aware before that variants involved with genetic paths related to the growth hormone are also associated with longevity. Now we have found a specific variant whose presence or absence is directly connected to it," Prof. Atzmon explained.

According to other research its well known that IGF-1 (insulin-like growth factor-1) contributes to longevity as well as associated with HGH and other growth hormones.

From theSciencegroup in the journalScience Advances,scientists have discovered a variation in genes that changes hormones in men that extend their life-span. Professor Nir Barzilai at Einstein, compared 3 groups of 100-year-old men from around the world with 100 American Jewish men with a control group of 70-year olds.

They discovered that men without Exon 3 on growth hormone receptor genes were more likely to live up to 10 years longer. According to Prof. Atzmon, this variant ensured longevity absolutely. "This study nicely wraps up the connection between growth hormone function and longevity. Our goal now is really to understand the mechanism of the variation we found, so that we can implement it and enable longevity while maintaining quality of life," Prof. Atzmon concluded.

Coincidently the research found that men with this genetic variation were also about 3 cm taller than their counter-parts. This is in opposition to other species in nature wherein that smaller of the strains usually live longer. This phenomenon holds true in insects, rodents and other mammals. In human males however the receptor anomaly caused less growth hormone absorption.

This research is very encouraging for World Health and A4M members. It further explains what we have hypothesized for years, that certain genetics allow for longer life. Our entire existence at A4M and World Health is devoted to Anti-Aging and how to enhance life and longevity.

Article by: Dr. Michael J. Koch, Editor withwww.WorldHealth.net and for Dr. Ronald Klatz, DO, MD President of the A4M has 28,000 Physician Members, has trained over 150,000 Physicians, health professionals and scientists in the new specialty of Anti-aging medicine. Estimates of their patients numbering in the 100s of millions World Wide that are living better stronger, healthier and longer lives.

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Growth Hormone Receptor Gene Mutation Discovered - Anti Aging News