The stem cell wizards of ASUs Brafman Lab. Left to right: Sreedevi Raman, Josh Cutts, Nick Brookhouser and Christopher Potts. Photo by Marco-Alexis Chaira/ASU Download Full Image

If you guessed through the use of pluripotent stem cells so named because they have the ability to turn into other types of cells then youre right on the money.

ASUs Brafman Labis on the cutting edge of this branch of research, recently earning a $1.5 million grant from the National Institutes of Health to study the mechanisms of early human neurodevelopment, and $225,000 from the Arizona Biomedical Research Commission to study the effects of aging and other risk factors for Alzheimers Disease.

Biomedical engineering Assistant Professor David Brafman, who heads the laboratory, credits his graduate students as crucial in securing these grants

The graduate students hard work, creativity and dedication were critically important for generating the data to convince the reviewers that our approach was feasible and worth funding, Brafman says of his students. Too often the success of a lab is attributed to the [principal investigator] when it is the postdocs, grad students and research technicians who are down in the trenches doing the work.

Graduate students from the Brafman Lab reviewing data. Photo by Marco-Alexis Chaira/ASU

The students working in the Brafman Lab often labor late into the night and sometimes on the weekend. They possess a special mix of a passion for their work and the knowledge that achieving potentially life-altering outcomes dont come with a simple nine-to-five job.

The laboratory they work in combines developmental biology, genetic engineering and bioinformatics to investigate the various factors that can govern a stem cells fate. If they can figure out the mechanisms behind the stem cells multipotential futures, they could use that information to design targeted therapies for ailments like idiopathic pulmonary fibrosis, heart failure and Alzheimers Disease.

Take Josh Cutts, who is pursuing his doctoral degree in biomedical engineering. He knows a thing or three about working in the Brafman Lab. Hes addicted to the thrill of discovery, regardless of any challenges or obstacles that may come his way.

Were working on things that havent been done before so its challenging sometimes frustrating to complete certain experiments or understand the results, he said.

The shapeshifting nature of the stem cells can make working with them seem like biological wizardry. In the lab the research team has made stem cells into brain cells, heart cells, lung cells and more.

Now we are working with cutting-edge brain organoids known colloquially as mini brains, which sounds a little eerie, to address many different research questions, said Cutts, who earned his bachelors and masters degrees in biomedical engineering at California Polytechnic State University, San Luis Obispo. Its miraculous to work with these every day.

Cutts work generated the preliminary data that helped the lab secure the NIH grant. After finishing his graduate work, this pluripotent scholar plans to earn a post doctorate degree to expand his knowledge and expertise. Long term, he hopes to contribute to translating stem cell technology to patients, in academia or industry.

Researcher Nick Brookhouser is working toward his doctorate in clinical translational sciences at the University of Arizonas College of Medicine in Phoenix. His research in the Brafman Lab is focused on Alzheimers Disease and investigating the contribution of the Apolipoprotein E gene, or APOE, towards the diseases progression.

He has successfully generated a set of stem cell lines from Alzheimers patients as well as other stem cell lines that serve as the control group in his research. He is currently working with gene editing techniques to investigate APOEs relationship to Alzheimers.

Brookhousers work is also supported by an Arizona Biomedical Research Commission grant. He developed patient-specific pluripotent stem cell lines and brain cell lines, and with those lines he created a 3-D neural culture system that models a brain for study. He has also been involved in testing and optimizing gene editing technologies.

In the future, he hopes to transition to more clinical-based research in the biotechnology industry. Long term he hopes to contribute to the development of cell-based therapies and work in clinical trials.

Doctoral student Sreedevi Raman has also been working on research related to Alzheimers Disease. Instead of experimenting with stem cells at their genesis, Raman is trying to make them old. She is intentionally accelerating the aging process of cells in a dish so that they may be used to model various age-related disorders.

Her work with induced pluripotent stem cells specifically has helped the Brafman Lab attain the ABRC grant. Raman can take adult stem cells and program them back into state where their fate is not yet assigned.

Christopher Potts, a research specialist with a professional science masters degree from ASU, works with gene editing. His contribution to the team is comparable to using copy and paste for genes, but a bit more complicated. Hes using technologies like CRISPR (Clustered regularly interspaced short palindromic repeats) to edit stem cell genomes.

I am changing the DNA of stem cells. Thats pretty cool, right? Potts said. I think one of the coolest things about our lab is how each student has their own project and functions basically independently, but we all help each other and are able to do much more than we could on our own.

Hes enjoying his research, but also looks forward to teaching a new generation of students in the future. He has a masters degree in science education and taught high school for four years before joining the lab.

Potts has aspirations of starting a new line of scientists through a, career in outreach or other high-level science education.

The cells he works on use signaling pathways to regulate what they will become like his multiple career options. Right now, I am just hoping for some signals to help me differentiate, he said.

Just as Brafman relies on the hard work of his students, the entire lab team relies on one another to succeed.

Our lab is pretty close-knit. We like to hang out together to socialize and I think that support system makes our lab more effective, Cutts said. If any of us are having a hard time with experiments or anything at all, you can rely on your lab members and especially [Brafman] to help you work it out.

Like Cutts, Brookhouser values the highly collaborative environment in the lab that has fostered strong professional relationships as well as lasting friendships.

Just as patient somatic cells can be reprogrammed to a pluripotent state, I feel that the skills and mentorship I have gained in this lab have allowed me to reach a pluripotent state and primed me to differentiate down many different career paths in the future, Brookhouser said.

Raman credits her positive collaborative learning experiences in the lab with helping her to make advances in research as well as open career possibilities for her future. Since she just started her doctoral work, shes got a lot of research ahead of her. Luckily, she found a good place to start.

Originally posted here:
ASU grad students' lab skills help earn funding for cutting-edge biomedical research - Arizona State University

THURSDAY, Aug. 3, 2017 (HealthDay News) -- Scientists have created genetically altered skin cells that may control type 2 diabetes in lab mice. And they believe the general concept could someday be used to treat various diseases.

Using a combination of stem cells and "gene editing," the researchers created patches of skin cells that were able to release a hormone called GLP1 in a controlled manner.

The hormone, which is normally produced in the digestive tract, spurs the production of insulin -- the body's key regulator of blood sugar levels.

The scientists found that transplanting the engineered skin patches onto diabetic lab mice helped regulate their blood sugar levels over four months.

Xiaoyang Wu, a stem cell biologist at the University of Chicago, led the "proof of concept" study. He said it raises the possibility that "therapeutic skin grafts" could be used to treat a range of diseases -- from hemophilia to drug dependence.

Wu's team focused on type 2 diabetes in these initial experiments because it's a common condition.

However, a researcher not involved in the study doubted the usefulness of the approach for diabetes specifically.

People with type 2 diabetes already manage the disease with diet, exercise and medications -- including ones that target GLP1, said Juan Dominguez-Bendala.

Using high-tech gene therapy to get the same result seems unlikely, said Dominguez-Bendala, an associate professor at the University of Miami's Diabetes Research Institute.

"I don't see something like this coming to the clinic for diabetes," he said.

But Dominguez-Bendala also pointed to what's "cool" about the experiments.

Wu's team used a recently developed technology called CRISPR (pronounced "crisper") to create the skin patches. The technique, heralded as a major breakthrough in genetic engineering, allows scientists to make precision "edits" in DNA -- such as clipping a particular defect or inserting a gene at a specific location.

Before CRISPR, scientists could not control where an inserted gene would be integrated into the genome. It might end up in a "bad" location, Dominguez-Bendala explained, where it could, for example, "awaken" a tumor-promoting gene.

Wu and colleauges used CRISPR to make specific edits in GLP1, including one that allowed the gene to be turned "on" or "off" as needed, by using the antibiotic doxycycline.

The modified gene was inserted into mouse stem cells, which were then cultured into skin grafts in the lab. Finally, those grafts were transplanted onto lab mice.

The researchers found that when the mice were fed food with tiny amounts of doxycycline, the transplanted skin released GLP1 into the bloodstream. In turn, the animals' insulin levels rose and their blood sugar dipped.

The engineered skin also seemed to protect the mice from the ravages of a high-fat diet. When the mice were fed a fat-laden diet, along with doxycycline, they gained less weight versus normal mice given the same diet. They also showed less resistance to the effects of insulin, and lower blood sugar levels.

According to Wu, the study lays the groundwork for more research into using skin cells as a way to deliver "therapeutic proteins."

For instance, he said, skin cells could be engineered to provide an essential protein that is missing because of a genetic defect. As an example, he cited hemophilia -- a genetic disorder in which people lack a protein that allows the blood to clot properly.

Skin cells could be an ideal way to deliver such therapies, Wu said.

For one, the safety of skin grafts in humans is well-established, he pointed out. Since the 1970s, doctors have known how to harvest skin stem cells from burn victims, then use those cells to create lab-grown skin tissue.

Because the skin is generated from a patient's own stem cells, that minimizes the issue of an immune system attack on the tissue.

Dominguez-Bendala agreed that using skin cells has advantages. For one, he noted, the skin graft can be easily removed if something goes awry.

But a lot of work remains before therapeutic skin grafts could become a reality for any human disease. And research in animals doesn't always pan out in humans.

A next step, Wu said, is to see whether the skin grafts maintain their effects in lab mice over a longer period. The researchers will also monitor the animals for any immune system reactions against the GLP1 protein itself.

The findings were published online Aug. 3 in Cell Stem Cell.

The U.S. National Institutes of Health has a primer on gene therapy.

Read the rest here:
Engineered Skin Cells Control Type 2 Diabetes in Mice: Study ... - Arizona Daily Star

Most nights, Marcia Dunlap attaches seven or eight leeches around her husband John's eyes as part of an effort to restore some of his vision. Tom Bailey/The Commercial Appeal

With the help of his wife Marcia, John Dunlap receives his nightly leech treatment at his home in East Memphis. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

At home most evenings, Memphis, Tennessee, attorney John Dunlap, 80, unbuttons and removes his white dress shirt and counting his steps and remembering which way to turn carefullywalks with a tall white canefrom the living room to the dining table, where his wife Marcia has a plastic container of leeches.

Twenty-six months ago,the couple's schizophrenic sonAndrewattacked them in theirhome. The injuries blinded Dunlap. He's in total darkness.

After drapinga large, peach-colored towel around John's neck, Marcia reaches into the water for the skinniest leeches. Those are the hungriest and most likely to latchonto John's face.

One at a time, she gently presses four leeches to the skin around John's left eye and three around the right. She waits patiently wait for eachto bite and stay connected to John's skin.

"You can feel a bite,'' he says. "A little, stinging bite... And then after awhile you don't feel anything.''

The Dunlaps have carried out this unusualroutine60 or so times since December. It's a type of therapy prescribed by a Los Angeles doctor who offers experimental stem cell therapy designed to regenerate tissue.

"In the beginning he made it very clear he's not anophthalmologist and not an eye surgeon but he had had some success with stem cells in treating blindness. It's experimental,'' Dunlap said.

Autoplay

Show Thumbnails

Show Captions

The doctor prescribed the leech therapy as a preliminary step because, Dunlap said, the leech enzymesenhance the blood supply to the eye and nourishthe eye tissue.

The left eye had atrophied, or withered. The idea wasto restore health to the eyebefore the stem cell treatment. There is no right eye, but the hope is that the leech enzymes will help revive that optic nerve in case a transplant is ever possible.

Since the leech therapy,the pressure in the right eye has improved significantly, Dunlapsaid, referring to follow-upexams. The retina, which had folded into an ice-cream cone shape after the trauma, has begun returning to its normal shape, he said.

Even though he still cannot see out of the left eye and the optic nerve remains severed from the retina, Dunlap said, "I now have a live eye.''

The Dunlaps decline to identify the California doctor, describing him as a"humble'' person whodoes not seek the publicity.

With the help of his wife Marcia, John Dunlap receives his nightly leech treatment at his home in East Memphis. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

Andrew, the Dunlaps' mentally ill son, is charged with attempted murder and domestic assault, and remains in jail awaiting trial. Thecouplehave told authorities that they mainly want Andrew to receive mental health treatment.

The Dunlapshave experienced tragedy long before the 2015 assault.

Their son Jeff, one of four children, was a St. Jude Children's Research Hospital patient who died of cancer at age 10, in September 1974.

Dunlap recalls a return car tripfrom Knoxville, where he and Marcia had been visiting grandchildren shortly after he was released from rehab.

"As we were driving back I started thinking of all the things I won't get to do again. In my mind, I was going down the list,'' he said.

It would be a long list, including some leisure activities he loves. An avid Cubs fan, heenjoyed attending spring training games in Arizona. A passionate golfer, he enjoyedwatching how the ball flew when he struck it well.

But Dunlap stopped himself from completing the list of losses, telling himself, " 'You don't want to dwell on that'. . . It's as if the Lord sent me a message that hit me across my forehead, saying, 'John, get over it. It could be a whole lot worse.'

"Anytime I want to start thinking about the things I'm missing or not doing what I used to do, I think 'Get over it. Move on'.''

Sudden blindness is such a change in lifestyle. "I guess some people may feel the world has ended for them, but it hasn't,'' he said.

Marcia Dunlap gets special leeches for her husband John's nightly treatment from the laundry room where she keeps it out of sight. Marcia places several leeches on his face in an effort to increase pressure in his left eye. In conjunction with stem cell treatment, the Dunlaps hope that one day John may be a viable candidate for a procedure that could return some of his vision.(Photo: Jim Weber/The Commercial Appeal)

The stem cell and leech therapy is expensive and not covered by health insurance. Some have expressed their skepticism about the legitimacy of the experimental treatments.

"You have some people who are concerned for you, that your approach is not going to be effective,'' Dunlap said.

"Yet, several folks up herehave said, 'John, I'd take a shot at it. It is expensive but you're the one with the white cane and the one who is blind and has to live with it. You have everything to gain and nothing to lose.'''

While some might be concerned about the unusual treatments, many others are inspired by the Dunlaps,saidBlanche Tosh, a fellow church member and friend since high school.

"I have told them so many times, 'You just can't begin to know the lives you have affected,'' Tosh said.

"I know so many people who look at the way they are dealing with multiple things. How could anybody endure that and just go on and be pleasant and make it from day to day with the consistent attitude that the world sees.

"You are not going to find many people whoever see one of them without a smile,'' Tosh said.

She was inspired to start a gofundme account (gofundme.com/johndunlapvision) to help coverthe Dunlaps' expenses. As of midweek, $8,795 of the $100,000 goal had been raised.

Memphis lawyer John Dunlap and his wife Marcia continue to search for some medical procedure to restore at least partial vision after John was blinded a few years ago when their mentally ill son attacked him. (Photo: Jim Weber/The Commercial Appeal)

Since December, Dunlap has undergone two-and-a-half rounds of leech therapy and two series ofstem cell treatments. The couple traveled to California in June for the most recent stem cell procedures, and returned home with stem-cell eye drops and injections.

Nowthey are in the middle of the leech therapy they resumed this summer.

John has a follow-up exam next week, when he will learn if there's been continued progress from the stem cell and leech therapies.

The California doctor "indicated it would take two to three months to see if we were getting any results from stem cell therapy out there,'' Dunlap said. That time could come sometime this month or in September.

If the stem cell therapy has not worked by then, he said,"We'll just have to see what any third plan looks like, and the cost involved.''

Late in life, Dunlap has been forced to learn to type, work a computer, navigate with a cane, count the steps and memorize the turns from one spot to another, communicate with Siri, and smile as blood-sucking leeches dangle from his cheeks.

Asked about his sources of inner-strength, he responded, "I don't know I'd call it inner-strength.

"I can tell you I certainly believe in the Lord. We pray daily. I appreciate the prayers of others. I think it certainly is a faithissue.''

He also credits his late mother, Cora, a single parentwho managed a grocery. "She was a very optimistic, loving person,'' he recalled.

"And I've had Marcia's support. Marcia wasn't going to let me give up, just sit down and do nothing.''

The Dunlaps are starting to consider resuming their annual trips to Cubs spring training in Arizona. Maybe next spring.

"You may have your vision by then,'' Marcia told John.

"I might,'' he responded."We'll see.''

Read or Share this story: https://usat.ly/2wtjL7V

See the rest here:
Will putting leeches on his face help this blind man see? - USA TODAY

HACKENSACK, N.J. and PETACH TIKVAH, Israel, Aug. 7, 2017 /PRNewswire/ --BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, announced today the appointment of Mary Kay Turner to the position of Vice President of Patient Advocacy and Government Affairs, effective August 7, 2017. Ms. Turner joins BrainStorm from Mitsubishi Tanabe Pharma America, where she was Head of Patient Advocacy and Communications supporting the commercialization of edaravone (Radicava) for amyotrophic lateral sclerosis (ALS). Her prior industry experience includes senior sales leadership roles and she was Head of State Government Affairs and Advocacy for Bristol-Myers Squibb Company.

"We are pleased to have Mary Kay join our company as we advance our ALS clinical program and prepare to initiate Phase 3 testing of NurOwn," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm Cell Therapeutics. "Having a strong patient advocacy perspective and voice in the community is integral to our mission of advancing tomorrow's medicines. Mary Kay brings an extensive background in patient advocacy, government affairs and health policy. We are fortunate to have someone with her biopharma expertise and dedication to the patient community join our team."

"Mary Kay will lead the development and implementation of external collaborations that will address and support the needs of people living with ALS", said Dr. Ralph Z. Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer of BrainStorm. "Working directly with patient advocacy groups deepens our understanding of patients' needs and aligns our efforts with the broader ALS community."

"I have been impressed by the progress BrainStorm has made with NurOwn, particularly the outstanding Phase 2 clinical data which showed meaningful improvements in disease symptoms," said Mary Kay Turner. "Joining the company at this exciting juncture provides a great opportunity to work with respected leaders in neurology and cell therapy and to contribute to the development of a new treatment that could potentially transform the ALS landscape."

Mary Kay is recognized as a leader within the biopharma industry for developing advocacy and government affairs strategies. Most recently she worked at Mitsubishi Tanabe Pharma America as Head of Patient Advocacy and Communications. Prior to this, Mary Kay spent 26 years at Bristol Myers Squibb Company where she played an essential role in establishing the company's advocacy function and held various positions of increasing responsibility in sales leadership, patient advocacy and government affairs. From 2010 to 2016, she was Head of State Government Affairs and Advocacy at Bristol-Myers. In this role she led a team of government affairs professionals that focused on significant legislative and regulatory issues that promoted and protected the discovery and development of innovative therapies. Mary Kay helped to define the company's advocacy strategy and engagement for each of its key therapeutic areas.

In 2016, Mary Kay was appointed to the Arizona Biomedical Research Commission by Arizona Governor Doug Ducey and served a one-year term.She was on the Board of Advisers for the International Cancer Advocacy Network (ICAN).Currently, Mary Kay is a member of the Board of Directors for the ALS Hope Foundation. She also served on the Board of Directors for Mental Health America for 3 years. Mary Kay has a Bachelor of Arts degree in Political Science and History from the University of Oregon.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. The Company holds the rights to develop and commercialize its NurOwn technology through an exclusive, worldwide licensing agreement with Ramot, the technology transfer company of Tel Aviv University. NurOwn has been administered to approximately 75 patients with ALS in clinical trials conducted in the United States and Israel. In a randomized, double-blind, placebo-controlled clinical trial conducted in the U. S., a clinically meaningful benefit was demonstrated by higher response to NurOwn compared with placebo. For more information, visit the company's website at http://www.brainstorm-cell.com.

Radicava is a registered trademark of Mitsubishi Tanabe Pharma America.

ContactsMedia:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188rel="nofollow">uri@brainstorm-cell.com

Investors:Michael RiceLifeSci Advisors, LLCPhone: 646-597-6979rel="nofollow">mrice@lifesciadvisors.com

View original content with multimedia:http://www.prnewswire.com/news-releases/brainstorm-supports-commitment-to-als-patient-community-with-appointment-of-a-vice-president-of-patient-advocacy-and-government-affairs-300499934.html

SOURCE BrainStorm Cell Therapeutics Inc.

Excerpt from:
BrainStorm Supports Commitment to ALS Patient Community with ... - Markets Insider