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Two Day FDA’s Regulation of Regenerative Medicine Seminar … – Business Wire (press release)

Posted: May 9, 2017 at 4:42 am

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of the "FDA's Regulation of Regenerative Medicine including Stem Cell Treatments, Tissue Engineering & Gene Therapies: 2-Day In-person Seminar" conference to their offering.

Stem cells harness the power to differentiate into numerous cells upon stimulation. This has led to their wide exploration across all of medicine, including high risk diseases. Of course, significant scientific breakthroughs in the use of stem cells to prevent, diagnose, and treat numerous diseases has caused numerous start-up companies to form. Despite, such promise, the FDA has yet to approve stem cell therapies for a wide range of diseases, except cord blood-derived hematopoietic progenitor cells for certain indications.

This tutorial will provide an historical context for the use of stem cells in medicine, where the field has been and where it is going. It will also provide the few examples of FDA approved use of stem cells in medicine and what is needed for the field to progress. For example, in 2006, the U.S. FDA implemented regulations governing the use of human cells, tissues, and cellular and tissue-based products in humans including bone, ligament, skin, dura mater, stem cells, cartilage cells, and various other cellular and tissue-based products. Currently, there is an ongoing debate in industry on how such therapies should be regulated, in particular by the FDA or under the practice of medicine, under federal law or state law, and as drugs or simply biologics.

Learning Objectives:

Fundamentals of stem cells

- What is all the excitement about

- How to control stem cell differentiation

- Sources of stem cells

- Incorporating stem cells into biomaterials

- Avoiding immune system clearance of stem cells

FDA regulatory approvals for the use of stem cells in medicine

- Currently approved use of stem cells in medicine

- FDA guidance documents for stem cell technologies

- Global approval of stem cell technologies

- How the FDA regulates regenerative treatments and therapies

- The use of human cells, tissues, and cellular and tissue-based product criteria and Minimal Manipulation Standard

- The drug and biological approval process

- Regenerative products as medical devices

- How to design appropriate clinical trials

- Applicable good manufacturing and good laboratory practices

- Product labeling, marketing and advertising

- FDA and other federal agency enforcement action

Future thoughts on approaches for regulatory approval of stem cell technologies

- Remaining hurdles

- Outlook for new technologies

For more information about this conference visit http://www.researchandmarkets.com/research/ljt255/fdas_regulation

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Reprogrammed Cells Improve Heart Function After a Heart Attack – Medical News Bulletin

Posted: May 9, 2017 at 4:42 am

In a recent study published in Circulation, researchers managed to convert de-differentiated human fibroblasts, support cells key in healing, to red blood cells or cells that line the interiors of blood vessels via manipulation of the SOX17 transcription factor. The de-differentiated fibroblasts can improve heart function after a heart attack and provide patient-specific cells that do not suffer from premature aging.

Fibroblasts, a support cell key in healing, are commonly used for de-differentiation. De-differentiated cells are similar to stem cells; depending on the environment they are in and what genes are active, they become different cells. De-differentiated cells have many medical uses; as many as the number of cells that they can become. In a recent study published in Circulation, researchers from the University of Illinois College of Medicine and the Indiana University School of Medicine managed to convert de-differentiated human fibroblasts to endothelial cells and erythroblasts. Endothelial cells are the cells that make up the lining of the interior of blood vessels. Erythroblasts are commonly known as red blood cells.

The researchers first de-differentiated the fibroblasts by overexpression of the proteins that transcribe DNA into RNA, specifically, pluripotency transcription factors. The de-differentiated cells were then placed into different media to transform them into endothelial cells and erythroblasts. The researchers observed that a specific transcriptional regulator, SOX17, and its respective activity corresponded to which cell the de-differentiated fibroblasts became. The researchers also experimented on rats that had suffered heart attacks. They implanted the de-differentiated fibroblasts into the rats and found that they improved heart function after a heart attack. Finally, the researchers observed that the cells generated from the de-differentiated fibroblast had increased production of telomerase. Telomerase is a transcription factor that produces telomeres, which are at the ends of chromosomes. The length of telomeres corresponds to the age of a cell, with longer being younger.

All this research shows the power of de-differentiated cells. De-differentiated fibroblasts can make patient-specific cells. These cells may avoid premature aging because of the increased production of telomerase: Helpful for enabling older patients to have personalized cells. Though only shown in mice, the de-differentiated fibroblasts did improve cardiac function after their heart attacks and may benefit humans as well. Pinpointing SOX17 as the switch for making endothelial cells or erythroblasts will optimize their generation, which will prove valuable for tissue regeneration and disease modeling.

Written By:Brian Jones

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Aspirin, other antiplatelet drugs boost T-cell therapy in mice with … – FierceBiotech

Posted: May 9, 2017 at 4:41 am

Engineered T-cell therapies, a promising way to prime a patients immune system against cancer, dont work for everyone. Scientists from the Medical University of South Carolina (MUSC)found one possible explanation that can be targeted: blood platelets.

The immune system sees tumors, or uncontrolled cell growth, as part of the self, which allows cancer to evade immune attack. In adoptive T-cell therapy, a patients own T cells are isolated from his or her blood, and then primed to recognize tumor cells.

Previous research has shown that some cancers curb T-cell activity and that platelets, a component of blood responsible for clotting, might make cancer worse. The MUSC team, led by senior author Zihai Li, M.D., Ph.D.,found that platelets help cancer to hide by secreting a molecule, TGF-beta, that suppresses T cells.

RELATED: New culture method boosts T cells' ability to recognize multiple cancers

While most TGF-beta is inactive, the researchers found that GARP, a molecular hook on the surface of platelets, traps and activates TGF-beta. As a result, platelets become the main source of TGF-beta used by tumor cells to tamp down on T-cell function, according to a statement.

The team ran a battery of preclinical trials, finding that a T-cell therapy more effectively awoke the immune system when given alongside common antiplatelet drugs, such as aspirin.

First, they noticed that melanoma tumors grew more slowly in mice with defective platelets than in mice with normal platelets. Adoptive T-cell therapy was more effective in genetically modified mice without GARP. And mice with normal platelets and melanoma that were given T-cell therapy along with aspirin and clopidogrelsurvived longer and relapsed less, according to the statement. But antiplatelet drugs alone did not fight the cancer.

"We can test simple, over-the-counter antiplatelet agents to really improve immunity and make a difference in how to treat people with cancer, Li said. The study was published in Nature Immunology.

This study could lay the foundation for further work testing antiplatelet approaches in melanoma and other cancers. Melanoma is not currently treated with adoptive T-cell therapy, but with checkpoint inhibitors, such as Bristol-Myers Opdivo and Yervoy.

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Aspirin, other antiplatelet drugs boost T-cell therapy in mice with ... - FierceBiotech

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Former Tranmere footballer shares powerful picture as he begins fighting cancer with stem cell therapy – Liverpool Echo

Posted: May 9, 2017 at 4:41 am

Former Tranmere Rovers player Joe Thompson has shared an inspiring photo as he begins stem cell therapy to battle his cancer.

The 28-year-old is battling the disease for the second time, after first discovering tumours in his neck in 2013.

The footballer played 32 games for Rovers in 2012 and 2013, before being diagnosed with nodular sclerosing Hodgkins disease.

He fought through six months of chemotherapy before returning to football, joining Bury and then starting at Rochdale last summer.

Now Joe has shared a brave photo from hospital, as he undergoes stem cell therapy.

Posting the picture to Twitter he said: Little Update, First day of the Stem Cell Harvest Process! Docs are very surprised with how good my blood counts are! Hearts super fit.

When he first announced his cancer had resturned in March Joe said his wife and daughter were his daily motivation.

He said: I will fight this life hurdle with the same belief, courage and desire as my previous battle.

The support shown to me since I discovered my illness has returned has been immense.

I would like to thank my wife, Chantelle, who was my rock in my first encounter and will once again be by my side with the same encouragement, discipline and strength.

Not to mention the love shown to me by both my wife and beautiful daughter Thailula-Lily who are both my motivations daily.

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Diabetes Cured without Side Effects – Anti Aging News

Posted: May 9, 2017 at 4:41 am

Posted on May 8, 2017, 6 a.m. in Diabetes Biotechnology

Scientists cure diabetes in mice for one year, without side effects, by boosting pancreatic cells that secrete insulin.

Researchers at the University of Texas Health Science Center at San Antonio have found a way to cure diabetes in mice. This finding is particularly important because the cure does not spur any side effects. The medical team's new technique boosts cells within the pancreas that emit insulin.

The Importance of the Findings

If the cure works forhumans without side effects, it will be the first-ever cure for Type 1 diabetes that does not produce any related problems. Furthermore, the research team's novel approach might help Type 2 diabetics cease insulin shots. The researchers aim to perform human clinical trials within the next three years. However, in order to do so, they must first test the strategy on large animals. If these studies go well, testing will move on to human beings.

It is interesting to note that the studies conducted on large animals are estimated to cost upwards of $5 million. Large animals are the next logical stepping stone as their physiology is fairly similar to that of human beings. In particular, large animals and humans share a similar endocrine system. The cure's co-inventor, Bruno Doiron, Ph.D. indicates these studies will likely occur before he submits an application to the United States Food and Drug Administration for Investigational New Drug approval.

The Cure's Potential Commercialization

The research group secured a United States patent back in January. UT Health San Antonio is generating a company to hasten efforts to commercialize the cure. It is expected that the cure will work for human beings as it did for mice. According to Dr. Doiron, his team's strategy worked perfectly in mice. He states the mice were cured for a full year without side effects. This success is acclaimed as a monumental breakthrough as no one has even come close to an entire year without side effects.

About the Therapy

The pancreas has numerous cell types beyond beta cells. The research team's approach is to modify these cells so they generate insulin. However, the goal is for insulin to be secreted only in response to sugar. In essence, the aim is to have non-beta cells function similar to beta cells. Beta cells are the only cells that create insulin. Insulin is important as it decreases blood sugar levels. The immune system of a Type 1 diabetes patient destroys beta cells. As a result, the patient lacks insulin. Patients plagued by Type 2 diabetes have failed beta cells and a resulting drop in insulin. Type 2 diabetes also causesinefficient use of insulin to boot.

The therapy involves a technique known as gene transfer. A virus serves as a carrier that sends selected genes to the pancreas. These genes spur digestive enzymes and other types of cells to produce insulin. Gene transfer with a viral vector has been approved numerous times by the United States Food and Drug Administration with the hope that it would treat an array of different diseases. Indeed, this technique has proven successful for treating certain childhood diseases.

The cell populations aside from beta cells co-exist with the human body's natural immune defenses. Type 1 diabetes patients who have lived with these cells for decades will enjoy the newly secreted insulin without any sort of harmful immune system response.

Precise Sugar Control

The therapy controls blood sugar in an incredibly precise manner. This appears to be a significant improvement over conventional insulin therapy as well as diabetes medications that send blood sugar too low. The research team's gene transfer allows for the modified cells to match the characteristics of beta cells so low blood sugar does notresult.

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Charity Golf Tournament raises just over $10000, achieving it’s goal – WHAG

Posted: May 9, 2017 at 4:41 am

HAGERSTOWN, Md. - Living with type one diabetes is not easy, especially if you're just four-years-old.

"I don't want diabetes," Jacob Fredlock, a four-year-old living with diabetes, said.

However, Jacob is not alone in his fight.

When he received his diagnosis a year ago, his mom, Ashley Fredlock, hit the ground running.

She joined the Juvenile Diabetes Research Foundation as a volunteer and made it her goal to raise funds and awareness.

That's why golfers turned out in huge numbers on Monday, to Beaver Creek Country Club, to make sure research to stop type one diabetes is on par.

"We actually didn't have a lot of exposure until type one diabetes chose us through his (Jacob's) diagnosis," Ashley, who is a Co-Chair of the tournament, explained. "So, we went through a little bit of a whirlwind as far as where to go and where to turn to."

That is, until JDRF stepped in and provided Jacob's family with the resources to cope and with fundraising efforts.

Over 100 people, between volunteers and golfers, came out to the Charity Golf Tournament to show their support.

Even those in attendance, whose lives haven't been directly touched, were moved by the cause.

"I got two small boys, so they're my world," Jared Bellman, a golf tournament attendee, said. "They're everything to me, so I can't imagine me personally having to deal with some of those challenges. My heart goes out to those that have to deal with this everyday."

"You want to do everything. You want to fix it. As a parent, you can't fix it when your kid is sick," Aileen Grabill, Co-Chair of the tournament, said. "So, you do the next best thing; you work hard and you raise funds."

The Charity Golf Tournament raised just over $10,000, achieving the goal for the day.

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Judge Blames Repeated Tardiness On Diabetes, Thyroid Condition – NewsChannel5.com

Posted: May 9, 2017 at 4:41 am

NASHVILLE, Tenn. - If you're called to court, the judges expect you to show up on time.

But a NewsChannel 5 hidden-camera investigation discovered that, for one Davidson County judge, the same rules don't apply.

NewsChannel 5 Investigates began watching after hearing complaints from people who appear in General Sessions Judge Rachel Bell's courtroom.

But we discovered was there's a big difference between what happened when she knew our cameras were watching and what happened when she didn't know we were there.

***

Inside the Davidson County courthouse, the 8:30 a.m. session inside Courtroom 1A is called the one-stop docket for a reason.

It's a chance for people who've been hit with minor citations to get booked and have their cases heard in one stop so they can get on with their lives.

On one day that we filed an official request to have cameras in the courtroom, Bell took the bench at 8:42.

Thirty minutes later, she was quickly disposing of cases -- one after another.

NewsChannel 5 Investigates noted to Bell, "The day you knew our cameras were going to be in the courtroom, you were on time."

"I promise you," she replied, "it had nothing to do with your cameras. I just had a really, really good health day -- honestly."

But two days earlier, we were watching the 8:30 docket with hidden cameras as people like Rufus Beasley came looking for justice.

"I went in the courtroom and sat and waited, waited, waited. There was no judge," Beasley said.

In fact, Beasley got a $329 speeding ticket, trying to make sure he wasn't late.

That same day, we were watching, and Judge Bell did not even leave her house until 9:48.

She got to the courthouse just after 10.

That day, the judge finally made it to her 8:30 docket at 11:20.

She says she had a 9 a.m. criminal docket that she had to handle first.

"I'm going to need ADA accommodations for the rest of my life until there is a cure -- honestly," she explained.

Bell said she has diabetes and a thyroid condition that makes it difficult to control her blood sugar first thing in the morning.

As a result, she said she has a right under the Americans with Disabilities Act to modify her schedule.

We asked, "Which means you don't have to show up to work on time?"

"As I indicated to you," she answered, "there are courtroom rules and regulations. And when you figure out what is best for your ADA accommodations, I'm in compliance with my ADA accommodations."

"But that means you don't have to show up to work on time?"

"My ADA accommodations allow me to run my courtroom within the local rules."

But we watched Bell's courtroom for three weeks and discovered that she almost never left her house until at least an hour after court was supposed to start.

On one day, when she had a 9 a.m. criminal docket, she finally left her house at 10:35.

Fifteen minutes later, she pulled into the garage -- finally taking the bench at 10:56 where plenty of people were waiting.

NewsChannel 5 Investigates asked,"Will you concede that sometimes people are waiting in the courtroom for your to show up?"

"I will not concede that people are waiting for me," Bell replied.

The judge said that, even when she's still at home, that's time when prosecutors can work out potential settlements with defendants -- so that more cases are ready to go when she takes the bench.

We noted, "If people show up at 8:30 or 9 o'clock and they're ready to go, they still have to wait on you."

"No, they don't," she insisted. "They do not have to wait on me."

"We saw it, judge."

"I don't believe, Mr. Williams, I don't believe that you know if a case is ready or not."

But another judge had to intervene in the case of Richard Atchley last year after Bell left him sitting in jail for almost three weeks.

In testimony, Atchley's attorney said the defense was ready to go for his first hearing at 9:15.

Bell didn't show up until 11:15, then she announced she had to leave at 12:30 to go teach a high school class.

And when attorneys tried to find another date to finish Atchley's hearing, Bell insisted she could not take the bench any day before 10:30.

Criminal Court Judge Mark Fishburn reviewed the case, writing: "It is disturbing that court did not begin until almost two hours beyond the scheduled opening of court."

He said Bell exhibited a "cavalier attitude" about making time to hear the case.

NewsChannel 5 Investigates told her, "That is a fellow judge saying that is not appropriate."

Bell answered, "Judge Fishburn was in his own courtroom. Judge Fishburn was not in my courtroom on that day."

"He heard testimony, though."

"I don't have comment about Judge Fishburn's comments or thoughts."

Rufus Beasley said,"If I don't show up, I don't get paid."

For the people who appear in her courtroom, Bell offered no apologies for taking her time getting to work.

"I have ADA accommodations. I'm in compliance. I'm also in compliance with the courtroom rules and regulations."

And that, she said, is all the public really needs to know.

We asked,"Are you healthy enough to do the job?"

"I'm more than capable," she declared.

Judge Bell -- who makes $170,000 ayear -- has faced ethics complaints after questions were raised last year about her work habits.

Those complaints were filed with Tennessee's Board of Judicial Conduct.

She said those complaints are still pending.

Past Investigations Focused On Absent, Tardy Judges

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Scientists find skin cells at the root of balding, gray hair – Medical Xpress

Posted: May 8, 2017 at 7:45 am

May 8, 2017 by Carol Marie Cropper Strand of human hair at 200x magnification. Credit: Jan Homann/Wikipedia

UT Southwestern Medical Center researchers have identified the cells that directly give rise to hair as well as the mechanism that causes hair to turn gray findings that could one day help identify possible treatments for balding and hair graying.

"Although this project was started in an effort to understand how certain kinds of tumors form, we ended up learning why hair turns gray and discovering the identity of the cell that directly gives rise to hair," said Dr. Lu Le, Associate Professor of Dermatology with the Harold C. Simmons Comprehensive Cancer Center at UT Southwestern. "With this knowledge, we hope in the future to create a topical compound or to safely deliver the necessary gene to hair follicles to correct these cosmetic problems."

The researchers found that a protein called KROX20, more commonly associated with nerve development, in this case turns on in skin cells that become the hair shaft. These hair precursor, or progenitor, cells then produce a protein called stem cell factor (SCF) that the researchers showed is essential for hair pigmentation.

When they deleted the SCF gene in the hair progenitor cells in mouse models, the animal's hair turned white. When they deleted the KROX20-producing cells, no hair grew and the mice became bald, according to the study.

The findings are published online in Genes & Development.

Dr. Le, who holds the Thomas L. Shields, M.D. Professorship in Dermatology, said he and his researchers serendipitously uncovered this explanation for balding and hair graying while studying a disorder called Neurofibromatosis Type 1, a rare genetic disease that causes tumors to grow on nerves.

Scientists already knew that stem cells contained in a bulge area of hair follicles are involved in making hair and that SCF is important for pigmented cells, said Dr. Le, a member of the Hamon Center for Regenerative Science and Medicine.

What they did not know in detail is what happens after those stem cells move down to the base, or bulb, of hair follicles and which cells in the hair follicles produce SCF or that cells involved in hair shaft creation make the KROX20 protein, he said.

If cells with functioning KROX20 and SCF are present, they move up from the bulb, interact with pigment-producing melanocyte cells, and grow into pigmented hairs.

But without SCF, the hair in mouse models was gray, and then turned white with age, according to the study. Without KROX20-producing cells, no hair grew, the study said.

UT Southwestern researchers will now try to find out if the KROX20 in cells and the SCF gene stop working properly as people age, leading to the graying and hair thinning seen in older people as well as in male pattern baldness, Dr. Le said.

The research also could provide answers about why we age in general as hair graying and hair loss are among the first signs of aging.

Explore further: New research provides clues on why hair turns gray

More information: Chung-Ping Liao et al. Identification of hair shaft progenitors that create a niche for hair pigmentation, Genes & Development (2017). DOI: 10.1101/gad.298703.117

UT Southwestern Medical Center researchers have identified the cells that directly give rise to hair as well as the mechanism that causes hair to turn gray findings that could one day help identify possible treatments ...

Men and women differ in obvious and less obvious waysfor example, in the prevalence of certain diseases or reactions to drugs. How are these connected to one's sex? Weizmann Institute of Science researchers recently uncovered ...

Salk Institute scientists have developed a novel technology to correct disease-causing aberrations in the chemical tags on DNA that affect how genes are expressed. These types of chemical modifications, collectively referred ...

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Researchers have undertaken the world's largest genetic study of childhood overgrowth syndromes - providing new insights into their causes, and new recommendations for genetic testing.

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Scientists find skin cells at the root of balding, gray hair - Medical Xpress

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My husband’s heart failure inspired life-saving stem cell therapy – Telegraph.co.uk

Posted: May 8, 2017 at 7:44 am

Its our goal for this to be a normal NHS procedure, so everyone who has a heart problem [and could benefit from this] will be able to. There are few downsides because theres no rejection as theyre your own stem cells, and every patient who has successfully had this treatment ends up taking less medication.

Jenifer is overjoyed with the progress already made, and knows that Ian would be, too, had he lived to tell his story.

For Ian, the treatment gave him an extra three years of life, but in 2006 he died from heart failure, at the age of 70.

He would be so thrilled, says Jenifer. His concern would be were not doing it quick enough, because for him everything had to be done immediately. But to have achieved this much well, the medical world says weve done it all in a very short space of time.

The couple spent their final years together alternating between their family home in St Johns Wood, north London, and a holiday home in Miami.

They were both each others second spouses, having married in 1980 after a whirlwind romance in Cannes Jenifers first husband had died, while Ian had divorced his wife and did not have children together. But Ian had two children from his first marriage, as well as two young grandchildren who he was able to spend those extra three years with.

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My husband's heart failure inspired life-saving stem cell therapy - Telegraph.co.uk

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San Jose Girl’s Lemonade Stand Raises Funds For Type 1 Diabetes … – NBC Bay Area

Posted: May 8, 2017 at 7:44 am

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A young girl in San Jose sets up a lemonade stand to raise awareness about Type 1 diabetes and gather donations for the Juvenile Diabetes Research Foundation. (May 7, 2017)

A traditional lemonade stand helps youngsters fill their piggy banks, but one such stand in the South Bay on Sunday helped raise funds to find a cure for a medical condition that hampers children across the world.

For the second straight year, Makoda Daszko of San Jose pitched together a booth, dished out cups of the sweet drink and collected dollars upon dollars all in the name of raising money to combat Type 1 diabetes, an ailment that she herself suffers from.

"We're celebrating the chance for a cure," Tim Daszko, Makoda's father, said.

Last year, Makoda Daszko raised roughly $5,000 for the Juvenile Diabetes Research Foundation. That event wasn't publicized, but hundreds of lemonade drinkers took notice.

The goal Sunday was to surpass that number by leaps and bounds.

"Every little bit helps," Tim Daszko said. "We're just trying to focus on helping out JDRF the best that we can."

No word yet on much money the family was able to collect.

Anyone wishing to learn more about Type 1 diabetes or donating is encouraged to visit JDRF.org.

Published at 3:30 PM PDT on May 7, 2017 | Updated at 10:21 PM PDT on May 7, 2017

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