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‘A blow to [STAT’s] credibility’: MD listed as author of op-ed praising drug reps didn’t write it. Ghostwriting/PR … – HealthNewsReview.org

Posted: September 8, 2017 at 1:49 am

The physician whom STAT lists as author of a now-retracted op-ed praising the vital role of drug company sales representativesdidnt actually write the piece.

Robert Yapundich, MD revealed this key detail in a telephone interview with me yesterday. He stated unequivocally that he did not come up with the idea for the article or compose the first draft of the piece. He said he provided edits to material that originated elsewhere.

Who wrote the article, then?

Here is Yapundichs account of how the events surrounding the op-ed unfolded:

Thats when the problems began in earnest.

As we reported earlier this week, Yapundich received more than $300,000 from the drug industry between 2013 and 2016, according to the federal Open Payments database. And yet disclosure of that conflict of interest was initially missing from Yapundichs op-ed; it was added only after an outcry in the comments section of the STAT piece and on Twitter.

Robert Yapundich, MD

Yapundich says he understands the importance of such financial disclosures and that the omission was unintentional. It resulted from miscommunication with AfPA and uncertainty regarding the disclosure requirements.

In regards to COI, Im not sure what is needed, he recalled writing to his AfPA liaison. Do you need the company names? Which years? What type of COI?

His contact reportedly wrote back: Hold on financial info. Hopefully only needed for AfPA and not for you individually.

Thats the last Yapundich heard about the disclosure issue until STAT called him to clarify, he says.

STAT acknowledged the omission by revising the author bio that originally appeared at the end of the article. Heres how it reads now:

Robert Yapundich, M.D., is a neurologist practicing in Hickory, N.C., and a member of the Alliance for Patient Access. The alliance, which receives funding from pharmaceutical companies, supports regulations that expand manufacturers ability to discuss off-label uses, particularly those that are accepted in compendia and practice guidelines or reimbursed by the government and insurers. According to OpenPaymentsData, Dr. Yapundich was paid $332,294 by industry between 2013 and 2016.

Editors note: This article was updated to include Dr. Yapundichs ties with industry, which were not disclosed to STAT.

So STAT finally clarified more than four days after first publishing the piece that Yapundich has a significant conflict of interest regarding this topic. But in clarifying the extent of the authors financial relationship with the drug industry, STAT raised yet more troubling questions about the articles provenance and authenticity.

Pat Skerrett, who edits the First Opinion section where the op-ed appeared in STAT, offered a slightly different explanation than Yapundich for the missing drug industry disclosures. Writing in an email he said:

I just now got off the phone with the [Yapundich]. He confirmed the industry payments, and said he had mentioned them to the PR company he was working with but that they were not passed along to STAT. [emphasis added]

This was the first indication Id received of any PR company involvement with the article.

The PR firm, which Skerrett identified as Keybridge Communications, touts itself as a DC-based public relations firm that specializes in op-eds, issues advocacy, writing, media placement, and web development. Heres their promotional pitch:

We brand thought leaders. Our goal is to get your message in front of your target audience, whether its influencers and consumers or lawmakers and voters. Opinion media drives the public debate and enables our clients to expand their footprint, sway attitudes, and achieve their strategic goals.

Exactly whose message was Keybridge trying to advance with placement of the STAT op-ed?

Bill Snyder, an associate at the firm, whose name was given to me by STATs Skerrett, declined to answer specific questions about who was paying the bills for the op-ed and what role his firm may have played in the editorial development of the piece. He cited client non-disclosure agreements.

His explanation of the financial disclosure confusion appears to cast blame on STAT for not being thorough enough. But it may also reflect failure to respect accepted standards for acknowledging conflict of interest.

As you know, Snyder wrote, Dr. Yapundich has many relationships with the pharmaceutical industry. This is no secret; the relationships are publicly detailed here. We didnt send this along as we were under the impression that the editor was asking about the Alliance for Patient Access, and Dr. Yapundich wrote the piece in his capacity as a member of AfPA.

Without seeing the entire exchange, its hard to know whether Keybridges impression here is justified. No matter what capacity Yapundich was writing in, his personal financial relationships are obviously relevant. They should have been included if there was any doubt at all as to what STAT was asking for.

Keybridge is well known for ghostwriting on political and health care issues. It has acknowledged a previous client relationship with the Pharmaceutical Research Manufacturers Association of America (PhRMA), among other pharmaceutical interests. It seems apparent that they also have some involvement with AfPA, the pharma-backed advocacy group.

Susan Hepworth

For his part, Yapundich suggested that the initial draft originated somewhere within AfPA. But given the financial backing this group receives from the drug industry, this may be a distinction without much difference. Moreover, Yapundich named Susan Hepworth as the Alliance employee who sent him the draft. She is also listed as a media relations expert for a different DC public relations firm, Woodberry & Associates. According to her bio, She focuses on driving and echoing strategic messages for its health care and education clients through the development and execution of an earned media strategy that includes both traditional and digital media, as well as surrogates.

It seems that wherever the op-ed originated, it received careful attention from PR messaging pros before it ever reached Yapundichs desk.

The practice of op-ed ghostwriting is common among politicians and celebritiesand is accepted by many editorial page editors.

But the STAT situation is different and poses significant ethical concerns, says Charles Seife, a professor of journalism at New York University.

Ethically ghostwriting becomes a problem when you have a big concern like a pharmaceutical company thats trying to put words in the mouth of the little guy, he told me. Why is it necessary to omit the name of the person who actually wrote the piece, unless youre trying to obscure the hidden hand of someone behind the scenes whos more powerful?

Charles Seife

Seife drew a parallel to Wyeths use of ghostwriters to promote the hormone replacement therapy Prempro in the medical literature. He said the company penned journal articles and recruited physicians to put their names on them. The reason you do that is to hide the influence of the drug company and to increase the impact more than you wouldve had otherwise, he said.

Its shocking if STAT is allowing this to go on, he added. Its a real blow to their credibility.

Writing in the comments section of the STAT piece, Seife had previously drawn attention to what he described as an implausible story about Yapundichs interaction with a drug company sales rep. Yapundich is listed as a promotional speaker for Acadia Pharmaceuticals drug Nuplazid in the Open Payments database. However, the STAT piece suggests that he had learned of this particular medication from a company-sponsored lunch program. That knowledge led to a patient encounter that resulted in a direct clinical benefit, according to the op-ed.

Thanks to what I had learned at the lunch program, I informed the family about this new medication and wrote a prescription for it. The drug eased the patients symptoms enough that family has been able to continue caring for him at home.

Ive heard shifting explanations as to how this particular anecdote actually went down, and I second Seifes call for an investigation to reconcile the discrepancies.

According to an email from STATs Skerrett, [Yapundich] said he absolutely did not fabricate any stories, as Charles Seife alleged. He said he heard about the Parkinsons drug from an industry rep, and spoke on its behalf later.

But in my telephone conversation with Yapundich, he told me that he was already being paid to speak on behalf of the drug when the sales rep interaction occurred. In fact, Yapundich said, his ghost-written article didnt correctly convey the details of what took place in the anecdote.

It didnt come out the way I intended it to, he said, speaking of the op-ed that carried his name. The article made it seem like Id never seen the drug before and that was not what I intended.

He told me he was well aware of the drug at the time of the encounter with the sales rep, and that the rep had said something interesting about the drug new medication data, Yapundich called it that set off a light bulb in his mind and subsequently led to the positive patient encounter.

I hope there arent other parts of the article that escaped my editorial oversight or review, Yapundich said. The next time I do one of these op-eds, I should be the one doing the drafting and they should be the ones doing the editing and reviewing.

Its worth repeating that STAT has a well-earned reputation for editorial excellence and hard-nosed reporting. A single lapse wont tarnish it.

Yet the issues raised by this situation cut to the heart of STATs credibility as a news source. And theyre not the only issues that were concerned about.

Here are my takeaways and suggestions for how STAT could proceed to repair the damage:

Lastly, I would encourage STAT to work more proactively to ensure the quality and authenticity of opinion page submissions. While authors of op-eds may get more latitude than they would in a straight news piece, the boundaries shouldnt be so wide as to include ghost-written puff pieces from PR companies.

When I raised pointed questions about my concerns with STAT editors, they declined to offer a detailed response or even make an attempt to investigate further and get back to me. They told me I was welcome to do that [myself].

It is the job of STAT to correct this. Not HealthNewsReview.org or any other external entity. STAT has a big, well-funded operation some of it supported by drug companies. Its a matter of ethical decision-making. What kind of operation do they want to be?

With the cost of medications approaching stratospheric levels, criticisms of the drug industry have been

[NOTE: This post has been updated. Scroll to the bottom for details.] I woke up

Maybe it's just seeing this - again - on the day after the President signed

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Feared Zika virus kills brain cancer stem cells, new research shows – Kansas City Star

Posted: September 8, 2017 at 1:46 am

A virus feared for its effect on the brains of the unborn might hold a key to treating a deadly form of adult brain cancer.

The Washington University School of Medicine in St. Louis on Tuesday said the Zika virus has shown it may be useful in attacking glioblastoma cancer cells.

The university collaborated with the University of San Diego School of Medicine in the research that shows the Zika virus kills brain cancer stem cells.

Zika attacks the brains of developing fetuses, leading to small and misshapen heads. The research indicates that ability can also be directed against cancer cells.

We showed that Zika virus can kill the kind of glioblastoma cells that tend to be resistant to current treatments and lead to death, Michael S. Diamond at Washington University said in a release announcing the findings, which were published Tuesday in The Journal of Experimental Medicine.

Glioblastoma is the most common form of brain cancer, afflicting about 12,000 people a year, according to the university. U.S. Sen. John McCain of Arizona is one of them.

Glioblastomsa stem cells are often resistant to standard treatment. The study injected glioblastoma tumors with two strains of the Zika virus, and both were effective in killing the stem cells, stopping them from producing more cancer cells. The study suggests that using the virus in conjunction with chemotherapy and radiation has a complementary effect. Chemo and radiation alone leaves stem cells to produce more tumors.

We see Zika one day being used in combination with current therapies to eradicate the whole tumor, Milan D. Chheda of Washington University said in the announcement.

The studys experiments were conducted on mice with tumors. The ones injected with Zika lived significantly longer than mice injected with a placebo.

The BBC and futurism.com also reported on the medical findings.

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Shawnee Woman Was Among First In The World To Undergo New FDA-Approved Cancer Therapy – KCUR

Posted: September 8, 2017 at 1:46 am

Emily Dumler, a 36-year-old mother of three, is petite, energetic and appears to be the very picture of health. To look at her, youd never know that four years ago she was at deaths door.

Her symptoms struck out of the blue in the middle of the day. It was lunchtime and her kids were with her. Her husband, a medical device salesman, was at work. Dumler called him and told him she needed to go to urgent care right away.

My blood wasnt clotting properly and so I went into urgent care and then ended up in the hospital for 43 days, Dumler says.

Initially, she was diagnosed with ITP, a disorder that leads to excessive bleeding caused by low levels of platelets. It turned out she had something else: non-Hodgkins lymphoma, a blood cancer.

Dumler wound up undergoing six rounds of outpatient chemotherapy, one round every three weeks. The initial results looked good. The cancer appeared to be gone. But it wasnt. It came roaring back.

In January 2015, she underwent a stem cell transplant using her own stem cells at M.D. Anderson Cancer Center in Houston.

They just basically wreck your body so much that you almost are basically dying, and then they give you your stem cells back just to keep you alive, Dumler says of the stem cell therapy.

She lost 20 pounds in a matter of weeks. And then came the bad news: the transplant failed. At that point, she was pretty much out of options. Her only hope was getting into a clinical trial at M.D. Anderson for an experimental treatment. It had just begun and luckily she qualified.

I was the third patient in the world to be in this trial, which is called CAR T-cell therapy, Dumler says.

Reengineering the body's own cells

CAR: Its a mouthful, but it stands for chimeric antigen receptors. The therapy involves removing a patients T cells, a type of white blood cell, and genetically engineering them to recognize and attack the patients tumors. The T cells are then put back into the patients body.

Dr. Joseph McGuirk is director of blood cancers and stem cell transplants at the University of Kansas Cancer Center. McGuirk oversaw a CAR-T clinical trial at KU that showed promising results.

When patients develop cancer, the T-cells have failed to do their job, because part of their job is surveillance for misbehaving cells or abnormally shaped cells recognize that and attack and destroy them, McGuirk explains.

Unlike traditional intensive chemotherapies or radiation, CAR-T re-arms patients immune systems, allowing them to do the work. It is, says McGuirk, a revolution in cancer medicine thats ongoing right now.

Just last week, the U.S. Food and Drug Administration for the first time approved a CAR-T therapy, in that case to treat kids and young adults with leukemia.

KUs clinical trial involved patients like Dumler with an extremely aggressive form of non-Hodgkins lymphoma. One of those patients, a 47-year-old Australian who traveled to KU to undergo CAR-T therapy in late 2015, died last year. But a significant percentage of patients nearly half achieved complete remissions.

It is a keyhole view into this extraordinary future thats coming down the line in cancer medicine, McGuirk says.

Costly treatment

The KU clinical trial is no longer accepting patients. But now that the FDA has approved CAR-T therapy, KU is expected to be one of the few facilities to offer it.

While CAR-T therapy has delivered impressive results and may eventually be approved for solid tumors, its extremely expensive.

When we talk about the cost of treatment, there's going to be the cost of the drug itself, there's going to be the cost, from the hospital side, of preparing the patient and then giving the treatment, and then there's going to be the cost of actually administering the treatment, says Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society.

In fact, Novartis, the drugmaker behind the FDA-approved treatment, has priced it at $475,000. And some patients, after showing promising results, have died from the side effects of CAR-T therapy.

Because as the CAR-T cells begin to get into the body, as they begin to attack the cancer, then there's the release of various proteins and other substances into the bloodstream that can actually cause, sometimes, fatal events, Lichtenfeld explains.

Emily Dumler got her CAR-T treatment on July 13, 2015, just over two years ago. And she did have side effects. One of them was neurotoxicity: basically her brain fogged up.

And it's just a little unnerving to be outside of what you can, I don't know, cognitively understand, so I did experience not knowing anything, Dumler says.

But the side effects eventually dissipated. And now, Dumler says, she feels great.

I actually just had my two-year scans in Houston a couple of weeks ago and Im still in remission today, Dumler says.

Before her brush with death, Dumler had been a stay-at-home mom. Now shes director of religious education at the Catholic church she attends.

She says she might have wound up taking the job anyway. But having gone through what shes gone through, she now says she felt called to do it.

Dan Margolies is KCURs health editor. You can reach him on Twitter @DanMargolies.

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Army medic donates bone marrow to stranger in need – Hawaii Army Weekly

Posted: September 8, 2017 at 1:45 am

WASHINGTON, D.C. Sgt. 1st Class Benjamin Heimstead, 25th Sustainment Brigade surgeon cell noncommissioned officer in charge, poses for a photo during his donation of bone marrow August 15 at the Georgetown University Medical Center. Only one in 430 people who have registered in the National Marrow Donor Program will go on to donate. (Photo by Capt. Aaron Moshier)

Staff Sgt. Heather Denby 25th Sustainment Brigade 25th Infantry Division

SCHOFIELD BARRACKS Sgt. 1st Class Benjamin Heimstead had only been in Hawaii for a few weeks before he received an email that would change the odds of survival for one special person.

My lab results showed that I was an excellent match, said Heimstead, the 25th Sustainment Brigade surgeon cell noncommissioned officer in charge. I had done a lot of research on bone marrow transplants, and it turns out that only 38 percent of those in need are able to find someone suitable to donate, so I knew this was something very special.

Heimstead participated in a bone marrow sample drive at Fort Bliss, Texas, in 2010 while he was stationed there. He went on to Fort Benning, Georgia, where he served as a drill sergeant for a couple years, and later as a senior operations NCO.

I really hadnt given the sample donation much thought after I did it, he said.

Seven years had passed before the match was found.

There are several steps toward ensuring a bone marrow match is made, according to the National Marrow Donor Program website. The steps are spread over several weeks and ensure an optimal match is made through medical evaluation.

Only 1 in 430 people who have registered in the NMDP will go on to donate; as of August, Heimstead is now one of them.

I thought I was ready for what was to come, he said. But nothing can prepare you for what the medicine is going to do to your body. The side effects were a lot of pain, insomnia and you cant take anything that is nonsteroidal or anti-inflammatory, because it will affect your platelets. But you know what? Id do it again in a heartbeat.

Heimsteads supervisor, Maj. Paula E. Young, 25th Sust. Bde. surgeon, said she will never forget the sacrifice her Soldier made.

I remember the disgruntled look on his face every time he would go to sit down or stand up from his desk, she said. The medicine that the donors are given build up their bone density, especially in your hips, and it can be quite painful.

Heimsteads final step in his bone marrow donation was coordinated through the C.W. Bill Young Department of Defense Marrow Donor Recruitment and Research Program, which flew him to Washington, D.C., where his stem cells were collected and prepared for transplant.

As a medic, Ive gotten used to being in the field, treating a patient and loading them up for transport, Heimstead said. Very rarely do you find out whether that patient survived, what happened or if you did the right thing on the spot, but this time I knew I had done the right thing.

This was one of those times where I could do what I had to do and actually have a really good chance of saving someones life, he continued.Heimstead will have to wait up to a year to find out if the transplant was successful through the MDRR program.

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Data Strategy: The Connective Tissue Required to Bring Cell and Gene Therapies to Market – Drug Discovery & Development

Posted: September 8, 2017 at 1:45 am

The stakes are high for cell and gene therapy manufacturers. The nature of these treatments, and their use of living human cells or genetic material, creates new intricacies and dependencies that disrupt the traditional commercialization process. The new commercialization process for these therapies is interconnected and personalized, requiring a higher degree of integration between specialized logistics, patient programs, and health outcomes evidence making data a strong connective thread that helps weave together the value story for these products.

Stakeholders, ranging from regulatory bodies to payers to prescribers and even to patients, will review the data generated during a cell or gene therapy clinical trial with more scrutiny than ever before. And, each stakeholder will have its own set of priorities and metrics by which it will judge the therapy. While this dynamic is not entirely new, the challenges for a manufacturer to provide this data and the consequences that may arise without it are. The decisions a manufacturer makes in clinical trial design and reporting will have greater implications for the commercial supply chain compared to traditional products.

To design a clinical trial that will deliver all of the data complexities ultimately required of a cell or gene therapy, a manufacturer must start with the end in mind - when the therapy is approved, covered, and available to patients. Trial designers must think about each stakeholder along the supply chain individually and collectively to understand the qualitative and quantitative measures each will need to appreciate the value of the therapy.

Ideally, the manufacturer should create a multi-disciplinary advisory group, made up of providers, payers, distributors and patients, which can offer guidance and perspective throughout the therapys life cycle. Their perspectives can be informed by early discussions with regulators agencies as well. For example, I have been on advisory boards where the preferred trial design for regulatory bodies differed substantially from the design preferred by healthcare decision makers. There will be times when stakeholder needs converge and diverge, so understanding their needs as early as possible in the development process will aid in the development of an effective data strategy to address the evidentiary gaps.

Two to three years pre-launch, manufacturers should go directly to relevant regulatory agencies to gain insight into their requirements and review the current marketplace to understand the data needed to support their therapy. Certainly, regulators are going to be interested in cure rate, failure rate, adverse events, and other anticipated data; however, they are also going to be particularly interested in the comparator that is selected for the study. More than 30 percent of cell and gene therapies target rare diseases associated with small and often widespread patient populations. Meaning, researchers will need to recruit patients from diverse geographic regions, and in each region, there could be a different established standard of care. Therefore, manufacturers will need to carefully consider and understand the treatment landscape in the geographies from which they pool patients to fill their trials.

Evaluating a commercialization partner

Fortunately, there is significant information available to help manufacturers educate themselves on these market nuances. Evaluating commercialization partners with expertise that ranges from a global to hyper-local understanding of care and requirements can help this process. Together, manufacturers and their partners can review the data, looking first for overlaps (e.g., evaluating which countries have the same standards of care and provide a comparator that is relevant to multiple regulatory bodies).

If, however, there is not significant overlap, a partner can help a manufacturer identify which country may be the best in which to launch its innovative product, providing it with an opportunity to obtain additional evidence for future markets. In addition, a partner can help identify what data is available from other clinical trials to possibly serve as an indirect comparator, allowing the manufacturer to benefit from not only its own data but also the research that has been done previously or is being done simultaneously.

As noted earlier, regulatory bodies arent the only stakeholders that need to be considered during the design of a cell or gene therapy clinical trial. Payers play a critical role in creating access to a manufacturers product. They, too, must be considered early in development at least prior to Phase III. Not only would it be devastating to patients eagerly awaiting the promise of a cell or gene therapy but also its extremely costly for a manufacturer to successfully navigate regulation, receive approval, and then not receive coverage for the therapy.

Much like the approach to regulators, manufacturers should invite payers to join in their processes. Its important to understand how insurers will view a particular therapy is this gene therapy likely a first line treatment or a third or fourth option? The trial data may show that the therapy had even higher efficacy rates in a sub-population within the cohort. This will influence how a payer interprets the value of the therapy and how it considers different coverage models.

Payers will look not only for the data relevant to regulators but also associated costs. For example, what is the cost of an adverse event while on a new therapy? And, how does that differ from the standard of care? It is important that a manufacturers data strategy capture resource utilization while on its therapy and the comparator or accepted standard of care to answer key questions. Are there hidden costs associated with the current standard of care that are eliminated by the new therapy? Does current practice create a side effect that must be managed and, therefore, has a cost to both the patient and the payer?

Of course, not all questions will be answered immediately. Commercial and private payers are also struggling to balance the burden of paying the upfront cost of the therapy with ensuring a long-term economic benefit to offset the initial investment. Significant research being done to better understand patient movement across healthcare plans and how that could potentially impact cell and gene therapy models moving forward. In the meantime, manufacturers must work with partners to understand payer concerns and to create the most compelling value story possible.

Beyond clinical trials

Once approvals and coverage are granted, manufacturers must contend with the level of uncertainty in real-world results, which is particularly high for cell and gene therapies. This uncertainty could strongly affect stakeholder confidence in cell and gene therapy use, which in turn, affects both the products success and ability to help patients. As we have seen with many specialty products in the past, approval does not always equate to adoption. Provider and patient education will be a large and critical component of any commercialization strategy for cell or gene therapies, and that education can be bolstered significantly by data. The right mix of data can help stakeholders gain confidence and mitigate concerns regarding efficacy as well as comparative effectiveness, adverse events and the impacts to the patient.

Therefore, the need for data does not end with the conclusion of a clinical trial. Cell and gene therapies have a level of interconnectivity between manufacturing, logistics, patient support, and reimbursement rarely seen in health care. As a result, cell and gene therapies will need in some ways like many other products constant, ongoing data collection, interpretation, and distribution. Manufacturers will need to build or more likely and more effectively enlist partners who can build integrated data sets that provide not only the therapy owner, but all stakeholders needle-to-needle visibility.

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Sen. Isakson applauds FDA approval of Kymriah gene therapy – Life … – Life Science Daily

Posted: September 8, 2017 at 1:45 am

Sen. Johnny Isakson (R-GA) recently commended the Food and Drug Administrations (FDA) approval of the first cell-based gene therapy available in the United States.

Officials said Kymriah was approved for certain pediatric and young adult patients suffering from a form of acute lymphoblastic leukemia, serving as an innovative therapy that reprograms a patients own cells to attack a deadly cancer.

This type of therapy is exactly what we had in mind when I began working for the Advancing Hope Act, which was ultimately approved and extended in last years 21st Century Cures legislation, Isakson said. When I heard this wonderful news directly from the FDA, I thanked them and told them to get it on the market, because its time to start saving kids lives.

The FDA said Kymriah would be used to treat acute lymphoblastic leukemia, a cancer of the bone marrow and blood that progresses quickly and is the most common childhood cancer in the United States referencing the National Cancer Institute estimates 3,100 patients aged 20 and younger are diagnosed with acute lymphoblastic leukemia yearly.

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 said. New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.

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Families raise money for research into rare diseases – KARE

Posted: September 8, 2017 at 1:45 am

University of Minnesota groundbreaking gene therapy research

Lindsey Seavert, KARE 3:22 PM. CDT September 07, 2017

Andrea and RyanShaughnessy, from the Traverse City, Michigan area, have been at the University of Minnesota Masonic Childrens Hospital for nine months, as their son, Anderson, 2, underwent two blood stem cell transplants for Hurler Syndrome. (Photo: KARE 11)

MINNEAPOLIS - The FDA just recently approved the first gene therapy available in the United States for childhood leukemia, ushering in a new frontier in medicine to reprogram a patient's own cells to attack a deadly cancer.

The breakthrough is also bringing a long-awaited promise at the University of Minnesota for children undergoing treatment for rare, life-threatening diseases.

An estimated 20 families whose children have undergone blood stem cell transplants for rare metabolic diseases, have joined together to launch a crowdfunding campaign to help U of M doctors research safer, more effective therapies, including new gene therapy that could bring life-saving impact for their children.

Andrea and Ryan Shaughnessy, from the Traverse City, Michigan area, have been at the University of Minnesota Masonic Childrens Hospital for nine months, as their son, Anderson, 2, underwent two blood stem cell transplants for Hurler Syndrome.

The rare genetic disease, affecting 1 in every 100,000 children, occurs when the body has a defective gene and as a result, cannot make an important enzyme. Children with Hurlers Syndrome have a life expectancy of 5 to 10 years old.

Time is not on our side, the more we can do earlier on, the better off it is for his long-term survival and development, said Andrea Shaughnessy. If we could help keep anybody else from living in our shoes because it is so hard, you know it might not be able to directly impact the help Anderson needs today, but it doesnt mean that we cant help others so they can have a better outcome and life expectancy tomorrow.

The Shaughnessy family made the second donation to the crowdfunding campaign, called the Pediatric BMT Metabolic Program Research Fund.

I think its really inspiring they are doing this, said Dr. Weston Miller, a U of M pediatric blood and marrow physician overseeing many blood stem cell transplants. Research is expensive and really driving novel therapies and improving on existing therapies takes time and money.

Dr. Miller noted the lack of research and development for rare diseases, and said the gene therapy reduces the health risks associated with undergoing and surviving blood stem cell transplants.

Really the unifying theme of all these novel therapies is going to be make it safer and more effective, said Dr. Miller. So, what we of course wish and hope for is we can find a way to have effective therapies and look Mom and Dad in the eye, and say there is closer to 100 percent they will be walking out of here.

The families from across the country and world have a goal to raise $1 million to fund research projects that might otherwise never make it to the laboratory.

We are pretty proud of this team and we know they can do it, its amazing the tenacity they bring, said Andrea Shaughnessy.

The crowdfunding page details their plea for support.

They have helped countless families from all around the world navigate the uncertainty of a life-threatening diagnosis and make heart-wrenching decisions. They go above and beyond, whether it is Google translating an email to correspond with parents in other countries or wearing a Minions shirt. They have revolutionized the way the diseases are treated, drastically improved the quality of life for many of their patients, and given families hope.

2017 KARE-TV

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Continuing the debate about right-to-die issues in Appleton – Wichita Eagle

Posted: September 6, 2017 at 7:44 pm

Nearly one year after Jerika Bolen, the Appleton teenager with an incurable genetic disease, announced her intention to go without a life-sustaining ventilator, experts say her case has had surprisingly minimal impact on the right-to-die debate.

"I fully expected it to continue in the dialogue," said Paul J. Ford, director of the NeuroEthics Program at Cleveland Clinic, about Jerika's story.

Bolen died last September after a lifelong battle with spinal muscular atrophy type 2, which destroys nerves cells in the brain stem and spinal cord that control voluntary muscle activity. She died at Sharon S. Richardson Hospice in Sheboygan Falls, after a final summer that included a prom in her honor in July.

"When I decided, I felt extremely happy and sad at the same time," Jerika told USA TODAY NETWORK-Wisconsin in July 2016. "There were a lot of tears, but then I realized I'm going to be in a better place, and I'm not going to be in this terrible pain."

Jerika's decision drew national attention, including an overwhelming amount of support from well-wishers worldwide. But her story also drew the ire of disability rights groups who attempted to intervene in Jerika's decision to stop treatment.

"It was an exceedingly complicated case," said Arthur Caplan, head of the division of bioethics at New York University's School of Medicine. "(Jerika) was 14, so not quite old enough to be legally able to make her decisions, but old enough that many (medical experts) would say she was old enough to help determine her care."

Jerika was mostly immobile and in chronic pain from SMA. She ranked her pain as a seven on a scale of one to 10 on her best days.

Medications had damaged her body. She had more than 30 visits to operating rooms. She had her spine fused in 2013 and the heads of her femurs removed in 2015.

The day of Jerika's death, Jen Bolen, who declined to be interviewed for this story, told USA TODAY NETWORK-Wisconsin that "no one in their right mind would let someone suffer like she was."

"Suffering is a pretty strong, compelling reason to back away," Caplan said.

Not Dead Yet, a national disability rights group, was one of five disability rights groups that asked authorities to conduct an investigation into Jerika's care.

Diane Coleman, Not Dead Yet's president and CEO, said the groups questioned Jerika's decision to die, as well as the public's response.

"We were trying to be gentle and respectful, but also to say that Jerika had a lot to live for, even if she couldn't yet see that herself," Coleman said.

A letter Not Dead Yet and other disability rights groups wrote in early August 2016 raised questions about Jerika's care and said the teenager was "clearly suicidal." Disability Rights Wisconsin also wrote a letter to Outagamie County child protection authorities.

"For Jerika's case, it really pushes the boundaries between the right to refuse treatment and assisted suicide," Coleman said. "If she had continued using her (ventilator)...things would be different, and she didn't get to get there.

"Almost all of the coverage supported her death. That's what's wrong."

Ford said it's difficult from the outside to understand a person's life and level of suffering.

"(Jerika) went through a lot," Caplan said. "She knows more about that than many people weighing in on what should happen."

Caplan said Jerika's story didn't take on the dimension of Terry Schiavo, a Florida woman who remained in a "persistent" vegetative state for 15 years, or Brittany Maynard, a 29-year-old with brain cancer who relocated to Oregon so she could legally kill herself with medication.

"(Jerika) was saying, 'I've been through so much. I don't want to do this anymore,'" Caplan said. "Which is an important question, but it isn't quite analogous to what happens either when someone requests help in dying or says, 'I don't want to be maintained because I'm so old and so frail that there's no point.' She was in a different situation."

Caplan said Americans are "completely and utterly confused" about right-to-die issues, including how to deal with mental impairment in dying, whether to honor a child's request and even what constitutes death.

"Where views diverge is saying how much suffering is too much to ask someone to bear, and whose responsibility is it to partake in ending a life if it's more suffering than anyone ought to bear," Ford, the Cleveland Clinic ethicist, said.

One of those issues is physician-assisted suicide. Public opinion about the practice remains divided: a 2013 Pew Research Center survey found that 47 percent of Americans approve of laws to allow the practice for the terminally ill, while 49 percent disapprove.

Five states California, Colorado, Oregon, Vermont and Washington and Washington, D.C., have legalized the practice, and Montana recognized it following a state Supreme Court ruling.

Ford said there was "a great energy among states" to continue the legislation for terminally ill adults a year ago.

"Those have sort of taken a backseat, recently," he said.

Earlier this year, Wisconsin State Rep. Sondy Pope introduced legislation, modeled closely after other physician-assisted suicide laws, that would allow terminally ill Wisconsin adults to receive medication to end their lives.

"It's about as restrictive as it could be. ... There are so many safeguards that it's almost impossible to use," Pope said.

Pope, who conceded that the legislation has no immediate chance of becoming law, said she would support legislation to allow a minor who isn't terminal to die with "very, very thoughtful safeguards that include input from loved ones."

"That's way down the road in a case-by-case individual basis ... It doesn't seem right, morally, to say, 'I'm sorry. You're not 18. You have to suffer.'"

___

Information from: Post-Crescent Media, http://www.postcrescent.com

An AP Member Exchange shared by the Post-Crescent.

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UAS Ketchikan welcomes new assistant professor hopes to implement community hikes: Pawlus teaching chemistry … – Ketchikan Daily News

Posted: September 6, 2017 at 7:44 pm

UASKetchikan welcomes new assistant professor hopes to implement community hikes: Pawlus teaching chemistry, geology, anatomy and physiology; hopes to coordinate hikes

By ALAINA BARTELDaily News Staff Writer

As a part of his community service hours, new University of Alaska Southeast Assistant Professor Matthew Pawlus, Ph.D., hopes to organize community nature hikes that are open to the public. With children of his own, the new assistant professor of science at the UAS Ketchikan campus especially enjoys working with younger groups, and plans to implement nature hikes and beach walks through tidal pools. Adults are welcome as well, as he hopes to start a trail building group.

Nothing official is planned quite yet for those hikes, as Pawlus is warming up to the campus and has just begun teaching his first semester at UAS.

Before receiving the position in Ketchikan, Pawlus had lived around the country. After being born in Ohio, his family moved to Wasilla when he was 8 years old and was there until he finished high school.

His next move was to Grand Junction, Colorado, where he attended Mesa State College a small school in the desert surrounded by a mountain biking and whitewater rafting community. While there, he met his wife, who was in school for nursing.

Pawlus relocated to Denver to attend grad school at the University of Colorado, where he received his doctorate in molecular biology. His main focus was on breast and kidney cancer, and ho w low oxygen levels, or hypoxia, drives cancer growth.

After that, I got a postdoc position in Seattle at the University of Washington, Pawlus said. I worked at an institute called ISCRM, the Institute for Stem Cell and Regenerative Medicine. Like the name suggests, its all about regenerative medicine growing back body parts, repairing tissue after its been damaged.

I did a bunch of work with stem cells there and a little bit of work with zebrafish, which are regenerative animals, he continued. Theyre pretty cool, you can cut them in half, cut off tissue, you can amputate 20 percent of their heart and theyll grow back.

Pawlus focused on modeling traumatic brain injuries in the fish, where hed poke them in the nostril with a small needle and make a hole in their brain. The wound would heal in a few weeks, and he would watch how that process evolved, and see if he could adapt it to humans.

While in Seattle, he began teaching a summer class at the University of Washington with other postdoc students about wound healing, and moved to Boulder, Colorado, where he was teaching science classes.

Pawlus then relocated to Spearfish, South Dakota, where he was teaching biology, and finally ended up in Ketchikan. He said his main goal at UAS is to engage and interest his students, especially since most of his classes are introductory courses.

Its always good when people are having fun and paying attention, thats really the goal, Pawlus said. I like to integrate a lot of activities whenever we can. If people are enjoying themselves and doing something, hopefully theyll remember what they learned.

He will be teaching general chemistry, anatomy and physiology, and an online geology class focusing on the evolution of life and fossils.

By implementing lab activities and fun chemical reactions, he hopes his students are able to take a liking to the subject.

I expect a lot out of them but I can remember whats it like to be in college. Im not going to bore them to death with lectures, he noted. I hate standing up there for long periods of time. Hopefully with these activities and interesting visuals, they can actually stay interested and maybe even be entertained a little bit.

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IUPUI researchers finding collaboration, breakthroughs in lab facility – IU Newsroom

Posted: September 6, 2017 at 7:43 pm

View print quality imageIn a 2015 file photo, psychology professor Stephen Boehm observes a student researcher in his lab within the Science and Engineering Laboratory Building.Liz Kaye, IU Communications

Four years after its opening, it still has that "new building" smell.

The Science and Engineering Laboratory Building, known to many as SELB, opened in the fall of 2013 to much fanfare from two of IUPUI's essential programs in a STEM world.

The schools of Science and Engineering and Technology at IUPUI had a brand-new, state-of-the-art facility to conduct research at their disposal. On the second floor alone, engineers and scientists from multiple disciplines coexist and collaborate, as their labs are nestled next to one other.

The investigators had a say in the facility's design. They customized layouts and equipment. They were able to move out of buildings and labs that were starting to show their age. They also brought the bands back together.

Description of the following video:

Video transcript

IUUI Researchers Find Collaborations, Breakthroughs in SELB video on https://youtu.be/1T4GondidpQ

[Video: Exterior and interior shots of SELB]

[Words appear: IUPUI presents]

[Words appear: Christopher Lapish, Psychology, Associate Professor]

Christopher speaks: The front of the building is really beautiful, and it has a wide, huge, open atrium with a veranda kinda over it. And the atrium's really nice. We've had talks in there before, lunches quite often. It provides a space for students to engage in collaborative learning or studying in between classes.

And so those spaces are really needed here on campus, and it meets those needs. When I got here, I found that they involved the scientists and the people that would use the building in the design of the building, which was phenomenal. And so the lab that I'm in right now, many of the features that it has, I got to design, and it's really been helpful in our ability to perform science.

This is a really nice soldering iron. Here's some impedance detectors that we use here, pretty standard lab microscope here. We went from a very small, antiquated, old, dusty laboratory, into a state-of-the-art, world-class lab now. I'm really, really excited about that.

[Words appear: Stephen Boehm, Psychology, professor]

Stephen speaks: It was really great when this building opened, first and foremost, because it brought us all back together.

So all the addiction neuroscience faculty are now in the same space. This entire lab that we're sitting in right now is the main wet lab for the entire addiction neuroscience department. So all the faculty have space in this room or in one of the adjacent small rooms that branch off from this room.

And this is where all our molecular and bench-type assays are done in support of all the behavioral work that occurs up on the third floor of this building. We wanted space that could accommodate the work that the current faculty were doing, addiction neuroscience faculty. But we wanted, also, the space to support some of the basic needs of future hires that we though we might go for.

[Video: The Indiana University trident appears]

[Words appear: IUPUI]

[Words appear: Fulfilling the Promise]

[End of transcript]

"All of the addiction neuroscience faculty are now in the same space," said Stephen Boehm, professor of psychology and director for the undergraduate neuroscience program, adding how his departmental colleagues were spread across campus before SELB was built. "This entire lab is the main wet lab for the entire program. This is where all of our molecular and bench-type assays occur in support of all the behavioral work that happens on the third floor of this building."

Boehm shares his lab with associate professor Chris Lapish. They are using rats and mice to study the neurological effects of addiction.

Lapish revealed smaller labs and offices that adjoin the main lab: a maze room that tests the rats' cognizance and addiction levels and a computer room where thousands of data points are crunched from each experiment.

The electrodes that attach to the rats' brains are constructed in the main lab, which is also where the analysis of cellulose membranes takes place.

"You put it in milk," Lapish said. "All the proteins in the milk attach to the membrane. It prepares the blots for them to use to assess protein concentration of different proteins throughout the brain."

On the third floor, the rats and mice are cared for next to a skybridge that connects SELB to the Science Building. The bridge provides a majestic view of downtown Indianapolis. On the south side of the building, Military Park is visible and even audible.

"You can actually hear the music from the Lawn at White River if they're having a concert there," Lapish said.

When Lapish interviewed with IUPUI in 2010, he was lured by the promise of a new building. IUPUI delivered -- and then some.

"They said, 'We're going to build this new building, and if you come, you're going to have a new laboratory,'" Lapish recalled. "I got to design many features of the lab I'm in right now, and it's really been helpful in our ability to perform science.

"We went from a very small, old, dusty laboratory to a state of the art, world-class lab."

On the first floor, multiple open spaces enclosed by glass walls and doors are simply labeled "collaboration." A recent pass saw small groups of students huddled around laptops working on projects. Whiteboards are available for brainstorming and scrawling breakthroughs. Around the corner is a large atrium to promote further interaction among engineering, technology and science minds.

"We've had talks in there, and lunches quite often," Lapish said. "It's a space for students to engage in collaborative learning or studying between classes. Those spaces are really needed here on campus, and it meets those needs."

The east side of the building's exterior pops with colorful windows. The west side helps corral students to the common outdoor areas dubbed the "Science Lawn." Essentially, it's a nice fit.

Jason Meyer, associate professor of biology, has a lab around the corner of Lapish and Boehm's. His work examines human induced pluripotent stem cells for studies of nervous system development and disease. The cells are genetically modified to become any type of cell in the body, and they can serve as a novel platform for studies of neural development, disease progression, drug screening and cellular repopulation.

"What we're interested in is the ability to differentiate these cells into the different kinds of neurons of the retina, the light-sensing part of the eye that allows us to see," Meyer explained. "We're interested in the possible regeneration of the visual system -- can we figure out ways to encourage these cells to regrow and perhaps sometime in the future to develop new therapeutic options for blinding disorders."

The main target for Meyer's current research is the retinal ganglion cell and its long, branchlike shape. These cells connect our eyes to our brain.

Meyer's research has had a comfortable home in SELB, whether he is studying cells in a dish from glaucoma patients or developing new drugs that could rescue stop the progression of visual degeneration to restore some degree of vision. Meyer and his students are able to grow fragile lines of stem cells.

"It takes quite a long time to grow these cells," Meyer said. "To have a dedicated space like this allows us to keep them isolated to keep them away from any kind of contaminants that might damage them or affect their growth.

Collaboration with Meyer's psychology colleagues is much more of a possibility in SELB than in previous locations, he said.

"To have those kinds of connections with neighboring labs and other researchers in those labs really helps facilitate discussions and helps synergize research," he said.

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