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Healthcare Terms Are Out of Touch, Says Mom Advocate – Medscape

By Adult Stem Cells

This transcript has been edited for clarity.

Eric J. Topol, MD: Hello. I'm Eric Topol, editor-in-chief for Medscape. I'm thrilled to speak with Hala Durrah today, who I got to know about through a remarkable essay she wrote for Health Affairs back in March. Hala, welcome to our program.

Hala H. Durrah, MTA: Thank you for having me.

Topol: It's a real pleasure and this an important and critical topic. Your essay was "My Child Is Sick; Don't Call Her a 'Consumer.'"[1] There is so much to learn here for Medscape folks. Can you tell us a little bit about your background and your family?

Durrah: Sure. I am a patient/family engagement consultant by profession, which basically means that I work with healthcare organizations and systems around the concept of patient/family engagement, spanning anywhere from patient-centered measurements to quality improvement, and so on. I came to the work, though, not as part of a plan, but by fate and destiny. My firstborn, whose name is Ayah, was born with a very rare liver disease which we knew would require liver transplantation. She is 16 years old now, but as you can imagine, we've been on quite a journey in the healthcare system. In addition, I have four other children and my husband is an adult medical hospitalist at a major academic center on the East Coast, so healthcare is a big piece of our lives.

I realized a few years into the journey with my daughter that I wanted to become more engaged in improving healthcare and partnering with our care teamthe physicians, nurses, pharmacists, and the larger group of specialists that she was seeing on a regular basis. I had the opportunity to volunteer at a local hospital that was looking for more patient/family involvement in some of their quality improvement work, and I decided that this was definitely something I wanted to pursue further, so I did. But my daughter's healthcare journey continues and I continue to see the good, the not so good, and everything in between.

In addition, I think I have a unique perspective because my husband is a physician and works in the hospital setting. I understand and can empathize with the real challenges facing physicians that are constantly placing pressure on them and demanding their time. I am very cognizant of that, so in my work I really try to balance the voice of the patient or caregiver, and the voice of the physician or nurse, because I can definitely see all of the different perspectives. At the end of the day, I think we all really want these meaningful relationships where we communicate and empathize with one another, and at the same time, we all want the best care for each other. That is my goal and the work I've been doing for the past several years.

Topol: That is terrific. You have a panoramic view of the healthcare landscapethat's for sure.

Topol: You wrote about your daughter; it is a miracle story in so many respects. Her name, Ayah, means "sign of God"is that right?

Durrah: Yes. We named her that before I knew she was going to be born with this illness, and it's pretty remarkable that she fit that name and the meaning of her name since she was born. Her first liver transplant, unfortunately, failed within the first 24 hours. As you all know, a liver does not wait, so we were preparing to say goodbye to her when she was about 5 years old.

Subsequently, by miracle, she got another liver 2 days later. It was from the same hospital where she was at, which was even more amazing because her surgeons were prepared to travel anywhere to get another liver. She was placed number one on the nation live listing after that first transplant failed, and it's remarkable that she got another one.

Topol: After she had these two liver transplants at age 5, she got Burkitt lymphoma.

Durrah: Unfortunately, because of her therapies, and also being Epstein-Barr viruspositive, she developed stage III Burkitt lymphoma 2 years after the liver transplant, which was quite devastating, to say the least.

Topol: It required another type of transplant, a bone marrow transplant.

Durrah: Yes. Unfortunately, she had several months of a pretty intense chemotherapy regimen that ultimately failed, and we had to move to an autologous bone marrow transplant. She was not able to have donor cells because she was already an organ transplant recipient. She sat one day in the hospital with a catheter, and they took her stem cells and then gave them back to her about a month later after some more intense chemotherapy.

In all our experiences, I don't believe that physicians and nurses ever defined my daughter as a consumer.

Obviously, she has been through quite a bit. A lot goes along with being a patient of liver disease or a patient who survived cancer with all of the chemotherapy, so while her health is stable, things are constantly moving in the background that we're watching. I tell people that I sometimes look at it like a radar screen. You kind of see the little red dots bleeping and some of them get brighter and some of them get dimmer. A lot of people are monitoring those lights, but I'm the chief monitor of all of those lights and the coordinator of making sure we stay on top of them.

Topol: You are quite an advocate and it is just an amazing story. I want to get into this message that you have about using the term "consumer," because I have hated that term for years. Why do we use this term when we're talking about patients in the health world? You wrote, "I share our story because I am becoming increasingly troubled by a trend in healthcaretoward thinking of patients as 'consumers' but not actually engaging communities in healthcare improvement and innovation." Tell us a bit about this objection of this term, because I think if anybody has a right to object to the use of that term, it would be you.

Durrah: The term definitely does not resonate with me, and I share your strong feelings against the utilization of this term. Overall, I think it continues to underline and push the business imperative of healthcare versus the humanity imperative in which healthcare was initially built upon. I reject in some ways that this notion of consumerism will improve healthcare because healthcare did not start as a business imperative, nor was the framework built to support such a term.

In all of our experiences, I don't believe that physicians and nurses ever defined my daughter as a consumer. I believe [the term] distances us from one another and does not amplify or support that relationship-based care that we all seek to have and to improve. I also don't think "consumer" empowers us, although I understand why some believe it may, because the framework of healthcare is not set up in such a way where "consumer" denotes choice. A lot of healthcare is dictated to the patient or to the caregivers by their insurance companies or their lack thereof, or the communities in which they live, or by lots of different other parties that are involved in it. I'm not quite sure where the choice comes in, especially because no one chooses to be sick. A consumer has choices, but we don't have that choice.

It does not make sense to use that term. I understand where people come from wanting to empower individuals by changing the terminology [to a less] "passive" role of the patient, but I'm not quite sure that changing the terminology we use makes us equals, gives us any more choices, or improves the care we will receive.

Topol: Words are important. This term is hackneyed, it's pervasive, and I think you made the most eloquent case in the history of the medical literature because your intersection with the healthcare world is extensive.

Durrah: Oh my gosh. I'm humbled by that. I'm not sure if I deserve all that, but I appreciate it.

Topol: You have a master's degree from George Washington and you worked in tourism, and you made some interesting parallels between a consumer of tourism versus a patient. Can you talk to us about that?

Durrah: Sure. My master's is in tourism and administration, with a focus in meeting and event management, and here I am in healthcare advocacy now. But I found that it's been very useful because you do see in healthcare this shift of trying to create a tourism-type experience for healthcare. And generally speaking, tourism is a choice; we all choose to travel or to use a certain hotel or rental car company because we've had experiences. Most of the time, you come back from a vacation with great memories, and when you share those memories with others, they may want to have the same experiences as you.

But I don't believe that we can really equate healthcare to a vacation. I'm not quite sure who would utilize that same experience as a vacation. Things that healthcare has that no other industry has are the trauma, pain, and anguish that go into being either a patient or the caregiver of someone. I think physicians and nurses have a lot of emotions in the healthcare experience that you could never find in tourism. And tourism tries to have a constant feedback loop with their customers or consumers; healthcare does not. Yes, we tout these wonderful surveys that are supposed to increase patient satisfaction, but were any of them co-designed with the communities the health systems serve? The answer is no. Do they allow you to really respond in such a way that will provide meaningful data using stories of your experiences in the system? No. So again, trying to equate the healthcare experience with the lessons learned from tourism is a mismatch.

Topol: Right. Even that term "medical tourism" is about a different concept.

There are many parts of your daughter's story that were really touching and deep, but one of the things that stood out was how her liver doctor would hold your hand, and how that was not about consumerism but, as you say, a choice driven by humanity and compassion. This, I think, speaks to the stark dramatic differences between the terms.

Topol: Another term I'm interested in asking you about is "providers." It's another term I don't care for. Should that term be used?

Durrah: It's interesting, because I believe that in the article I do reference "care providers" at one point. Initially when I started in this work, I always [used the specific terms of] "physicians," "nurses," "pharmacists," and so on. All of a sudden that term "provider" got thrown into the mix, and I adapted it because it seemed that everyone was using it to cover all the members of the care team that might interact or touch a patient. I'm guilty of using that term and I got a lot of response after my article came out, particularly from physicians, about how the term "provider" was not liked. I believe the term came about with this whole business imperative/push in healthcare. Insurance companies use that term and healthcare systems have now adopted that term as well, and it probably is in play because of this whole consumerism push as well.

I did prefer saying "physicians" or "nurses" or "pharmacists" and that we're all part of the "care team." I prefer that terminology, just as I prefer to be called a "partner" in my care versus a "consumer" or a "customer." I think that the term does not fit the role that physicians and nurses play. They are not just a provider. I look at them as part of my team and I'm on their team, so we're all working together. I think "provider" also sounds a little bit like a hierarchy. It does not suggest that notion of that partnership and just reinforces a distance. Consumerism is kind of defined by this transactional relationship, so you would use that term to reinforce that. I see how it has developed and how it's being pushed, and I think it's all related to the business of medicine versus the actual human experience and the humanity of medicine.

We so largely lost the 'care' in 'healthcare.

Topol: These terms got adopted when healthcare was transformed to a business, and now we're relooking at this.

Your work and eloquence and what you continue to do as an advocate in patient care will help us get back on track. It's just one of many things we need to do.

Topol: You also brought up the term "lean principles." It would be interesting for you to just touch on this as well.

Durrah: I've noticed in my work in healthcare thus far that health systems are adopting a lot of frameworks. One of these is becoming "lean" or utilizing "lean principles," which are driven by efficiency and cost savings.

Typically, those who advocate for lean principles within health systems say, "This is going to benefit everyone: physicians, nurses, patients and families, and the communities we serve." But if you really dig down deep, I don't think it has trickled down at all to any cost savings for patients or families. I don't think it's improved quality for communities which the healthcare system serves. And I certainly don't see physicians and nurses not being more burned out or more extended. They get affected by lean principles, where staffing is cut in order for that cost savings to occur. They are being told to do 150 things versus 100 things a day.

When I first learned about "lean" and read about it, I thought it sounded great. But then when I got deeper into this work, I thought, "Wait a minutewho is this benefiting other than the bottom line of healthcare systems?" It's really interesting how healthcare tends to cherry-pick principles or terminology from other industries with the goal of trying to improve healthcare, when it's really not doing that. It's simply putting a Band-Aid on one problem and creating a new one.

Topol: We so largely lost the "care" in "healthcare." You referred to the "care team." You undoubtedly experienced that with your daughter, and we want to bring it back. We should be more precise about language and avoid business terms, and talk about clinicians as doctors, nurses, pharmacists, physical therapists, or whoever. We should be more specific than using this "provider" term which obviously could be a relative or a friend.

Topol: What response did you receive from the essay you wrote in Health Affairs? It clearly resonated, and when I posted it on Twitter, there was quite a bit of response.

Durrah: I got an incredible response from the article, and I appreciate you tweeting and retweeting it because a lot of people saw that and reached out to me and started retweeting as well. A number of people sent me personal emails and messages, and I really was humbled by the response. I was quite nervous to write the article I did because in my work, there is kind of a fine line that you can only push the boundary so far, and then if you push them too far you get a lot of pushback. I sometimes feel like I'm this one little patient advocate voice among all these other voices. The article really resonated with physicians in particular, and they really understood the story which I shared about my daughter's liver doctor.

When we found out that she was diagnosed with cancer, she asked to sit in the meeting with the oncologists. She didn't have to do that, but she wanted to. I said, "Of course you can join us." She sat next to me and held my hand the entire meeting. Every time they said something distressing, she would just squeeze my hand, but she didn't utter a word. I think that encapsulates the relationship that patients and caregivers have with their physician or nurse. It's such a special bond, and the term "consumer" could never define that particular bond; she would never look at this as a transactional relationship. Would "consumer" teach her to do that? No. As you said before, it is the humanity and compassion of medicine, and that is why most everyone who comes into the profession really wants to help humanity and give that empathy and compassion.

We have a lot to learn, and it's going to take many voices to speak up and push back against consumerism because some pretty large organizations and groups are trying to push consumerism and continue this business imperative of healthcare that is motivated by bottom lines. I think we're going to get there. Maybe we're already there. We're at a tipping point. There is so much dissatisfaction, and so many parts of the system are broken. To say that consumerism will give you more choices, shorter wait times, and maybe even price transparency, even though you still have to deal with your insurance provider or lack thereof, makes me giggle because I don't think that is going to be the solution.

The only solution I see is that we have to partner with one another and co-create solutions to improve healthcare as teamsequal voices at the tableand begin this pushback. If we don't, I fear what is ahead because I've already had a glimpse of it. I'm nervous because as my daughter slowly moves into adulthood, and she is almost there, these were the things I hoped we would have conquered before she got to that point. A lot of this is pushing us away from one another and distancing ourselves from one another, and that is not going to change healthcare.

Topol: You said it so well. I've been eager to meet you since I read your work months ago, and I wanted the whole Medscape audience to get to know you and your story. Most of them are not on Twitter and most don't read Health Affairs. Your story is so darn important, and you convey it in a powerful way.

I'm so glad to know that your daughter is okay, having gone through 16 years of rough times, especially in her earlier years. I just want you to carry on. You are an important voice. You may qualify yourself as only one, but it's a powerful one, and very few people have had an experience like yours. You're in a rarified group and can transmit the emotion and the sense of caring. Let's get these words right, and let's zap "consumer" from this story of future healthcare.

Thanks so much for joining us today on Medscape, and we will look forward to following you and learning more from you in the future.

Durrah: Thank you. I look forward to partnering with your audience on improving healthcare.

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Healthcare Terms Are Out of Touch, Says Mom Advocate - Medscape

ISCT forms cell and gene therapy sector-wide coalition to combat the rise of unproven commercial cell banking services – PharmiWeb.com

By Induced Pluripotent Stem Cells

Vancouver, Canada, October 21, 2019 ISCT, the International Society for Cell and Gene Therapy, the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector, today announces it has formed a global consortium of a wide range of leading professional and education societies to combat the rise in the number of unproven commercial cell banking services. Full details of the statement can be foundhere.

The consortium partners include the International Society for Stem Cell Research (ISSCR), Society for Immunotherapy of Cancer (SITC), American Society for Transplantation and Cellular Therapy (ASTCT),American Society of Gene & Cell Therapy (ASGCT), European Society for Blood and Marrow Transplantation (EBMT), Foundation for the Accreditation of Cellular Therapy (FACT), Joint Accreditation Committee ISCT-EBMT (JACIE) and the Forum for Innovative Regenerative Medicine (FIRM).

The consortium has been formed following ISCT issuingpatient advice and concern on unproven T-cell preservation services on August 7, 2019. These services include the banking of T-cells, dental cells and cells for the derivation of induced pluripotent stem cells for potential therapeutic uses.

The joint statement from ISCT and the consortium partners includes an agreement on a number of key points. Commercial cell banking services are not supported by current scientific evidence, as opposed to the range of cell therapies such as CAR-T therapies, that follow established approval processes. Additionally, cell banking services cannot claim to know that the cells they preserve today could ever be appropriate for clinical use, could be used by manufacturers, or meet the requirements of many national and international regulatory agencies. As a result, there is no clear pathway to legitimate clinical use. All parties agree offering these services commercially to patients is thus premature, misleading, and drives false hope.

In addition, the ISCT joint statement makes clear that patients, being misled by these services, are thus prevented from giving a full and valid informed consent. Cell banking companies mislead patients in a number of ways, including using tokens of scientific legitimacy that suggest a stronger scientific basis than currently exists. These tokens include endorsements from individuals or scientific advisory boards that might not fully endorse the specific products, links to scientific articles, and references to ongoing clinical trials.

ISCTs raison detre is to lead the industry in supporting scientifically validated cell and gene therapies. As a result, ISCT will continue to welcome all innovations, including cell banking approaches, that increase the number of patients who can benefit from these therapies, said Bruce Levine,President-Elect, ISCT and one of the inventors of CAR-T therapies.However, ISCT also leads industry action on unproven cell therapies and services in the cell and gene sector. This is why ISCT has forged a consortium throughout the industry against the marketing of speculative cell banking services that do not have appropriate pre-clinical, and clinical evidence and a plausible pathway to the clinical use of banked cells. We collectively believe these banks have the potential to be detrimental to the future development of cell and gene therapies.

About ISCT

Established in 1992, ISCT, the International Society for Cell and Gene Therapy is a global society of clinicians, regulators, researchers, technologists and industry partners with a shared vision to translate cellular therapy into safe and effective therapies to improve patients lives worldwide.

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three key areas of translation: Academia, Regulatory and Commercialization. Through strong relationships with global regulatory agencies, academic institutions and industry partners, ISCT drives the advancement of research into standard of care.

Comprised of over 1,500 cell therapy experts across five geographic regions and representation from over 50 countries, ISCT members are part of a global community of peers, thought leaders and organizations invested in cell therapy translation. For more information about the society, key initiatives and upcoming meetings, please visit:

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ISCT forms cell and gene therapy sector-wide coalition to combat the rise of unproven commercial cell banking services - PharmiWeb.com

Artificial embryo without sperm or egg forms live fetus – ZME Science

By Stem Cell Medical Center

For the very first time, scientists have made artificial embryos from scratch, without sperm or egg, and implanted them into female mice. The embryos developed into live fetuses, but these exhibited major malformations.

The team at the University of Texas Southwestern Medical Center used extended pluripotent stem cells, which are cells that have the potential, like an embryo, to develop into any type of tissue in the body. These master cells are able to form all three major types of cell groups (ectoderm, endoderm, and mesoderm). Unlike simple pluripotent stem cells, the extended variety can develop into tissues that support the embryo, such as the placenta.Without this type of stem cells, embryos cannot develop and grow properly.

The researchers coaxed stem cells to form into all the cells required for the development of an embryo by bathing them into a solution made of nutrients, growth stimulants, and signaling molecules. The cells assembled into embryo-like structures, including placental tissue.

Next, the artificial embryos were implanted into the uteruses of female mice. Only 7% of the implants were successful but those embryos that did work actually started developing early fetal structures. There were major malformations, however, as the tissue structure and organization did not closely resemble that of a normal embryo.

Previously, other research groups had managed to grow artificial embryos but this was the first time that they were successfully implanted and developed placental cells.

In the future, the University of Texas researchers plan on refining their method in order to grow fetuses that are indistinguishable from normal ones. The goal is to replace real embryos and make artificial ones at scale. These embryo models could then be grown in dishes to study early mammalian development and accelerate drug development.

Some of the cells that the researchers used to grow into embryos originally came from the ear of a mouse. Theoretically, the same should be possible for human embryos, but why would we? Besides testing drugs, artificial embryos could be grown from the skin cells of an infertile person. Then, in the lab, these embryos could be studied in order to identify potential genetic defects that might cause infertility.

Even if such stem cell-derived embryos do not completely mimic normal embryo growth, there is still a lot we can learn about mammalian development. But, as is always the case with research that breaks the frontiers of what was once thought possible, our policies havent yet kept up with advances. There are serious ethical considerations to possibly making a person from a synthetic embryo. Although such a prospect is still science fiction, rapid developments such as the present study suggest that it is not impossible and we better prepare.

The findings were reported in the journal Cell.

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Artificial embryo without sperm or egg forms live fetus - ZME Science

CRISPR Therapeutics and KSQ Therapeutics Announce License Agreement to Advance Companies’ Respective Cell Therapy Programs in Oncology – Business Wire

By Uncategorized

ZUG, Switzerland & CAMBRIDGE, Mass.--(BUSINESS WIRE)--CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and KSQ Therapeutics, a biotechnology company using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development, today announced a license agreement whereby CRISPR Therapeutics will gain access to KSQ intellectual property (IP) for editing certain novel gene targets in its allogeneic oncology cell therapy programs, and KSQ will gain access to CRISPR Therapeutics IP for editing novel gene targets identified by KSQ as part of its current and future eTILTM (engineered tumor infiltrating lymphocyte) cell programs. The financial terms of the agreement are not being disclosed.

We are thrilled to gain access to CRISPR Therapeutics foundational IP estate through this agreement, said David Meeker, M.D., Chief Executive Officer at KSQ Therapeutics. Our eTILTM programs involve editing gene targets in human TILs that were discovered at KSQ by applying our proprietary CRISPRomics approach to immune cells in multiple in vivo models. This agreement clears an important path for us to be able to bring these programs through development and commercialization, leveraging CRISPR Therapeutics proprietary editing technology.

The gene targets within the scope of the license agreement were identified using KSQs proprietary CRISPRomics drug discovery engine, which allows genome-scale, in vivo validated, unbiased drug discovery. These specific targets were uncovered in screens to identify genetic edits that could enhance the functionality and quality of adoptive cell therapies in oncology.

KSQ has built an industry-leading platform to screen for novel gene targets using its technology, and has identified a group of targets that could help unlock the full potential of adoptive cell therapy in oncology, said Samarth Kulkarni, Ph.D., Chief Executive Officer at CRISPR Therapeutics. As a result of this license agreement, CRISPR Therapeutics will have the opportunity to bring these novel targets into our leading allogeneic CAR-T development platform to further strengthen our future programs in this important therapeutic area.

About KSQ Therapeutics

KSQ Therapeutics is using CRISPR technology to enable the companys powerful drug discovery engine to achieve higher probabilities of success in drug development. The company is advancing a pipeline of tumor- and immune-focused drug candidates for the treatment of cancer, across multiple drug modalities including targeted therapies, adoptive cell therapies and immuno-therapies. KSQs proprietary CRISPRomics drug discovery engine enables genome-scale, in vivo validated, unbiased drug discovery across broad therapeutic areas. KSQ was founded by thought leaders in the field of functional genomics and pioneers of CRISPR screening technologies, and the company is located in Cambridge, Massachusetts. For more information, please visit the companys website at http://www.ksqtx.com.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement

This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the intellectual property coverage and positions of CRISPR Therapeutics, its licensors and third parties and (ii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the outcomes for each CRISPR Therapeutics planned clinical trials and studies may not be favorable; that one or more of CRISPR Therapeutics internal or external product candidate programs will not proceed as planned for technical, scientific or commercial reasons; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties inherent in the initiation and completion of preclinical studies for CRISPR Therapeutics product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

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CRISPR Therapeutics and KSQ Therapeutics Announce License Agreement to Advance Companies' Respective Cell Therapy Programs in Oncology - Business Wire

Justin Thomas, Danny Lee tied for 3rd-round lead at CJ Cup – Sports and Weather Right Now

By Stem Cell Doctors

JEJU ISLAND, South Korea Justin Thomas was looking at a three-stroke lead after three rounds at the CJ Cup with one hole to play Saturday, which would have set him up nicely for his second win in three years at South Koreas only PGA Tour event.

But the 18th hole saw a big swing in the scoring at the Nine Bridges course.

South Korean-born New Zealander Danny Lee holed a long eagle putt to finish with a 68. Thomas, who won the inaugural CJ Cup in 2017, bogeyed the 18th for a 70 and he and Lee were tied with a three-round total of 15-under 201 after three rounds.

Australian Cameron Smith was in third place, three strokes behind, after a 68. Jordan Spieth shot 70 and was in a three-way tie for fourth, four behind.

I played well, I just didnt hit a lot of good putts that burned the edges and didnt quite go in, Thomas said. If a couple of those putts fall, I dont make a bad bogey on 18 and the other two bogeys I made, I hit good putts, they just caught the edge and lipped out.

Obviously Dannys playing well, a lot of guys are playing well, so I need to go out and play well tomorrow. I just need to keep doing what Ive done the last couple days and just get a little bit more out of it.

Lee said he was pleased to play well before his family and fans.

Ive never played exceptionally well in Korea, so I also felt I could play better in front of my Korean fans. On top of that my parents and my wife are Korean and my granddad lives here so that would be great. I am glad I had a strong showing this week and hopefully I can say the same tomorrow.

The biggest movers Saturday were last years runner-up Gary Woodland and first-year professional Colin Morikawa of the United States. Their 65s moved them into a tie for seventh place which included Ian Poulter (66) and Graeme McDowell (68).

Defending CJ Cup champion and top-ranked Brooks Koepka withdrew from the tournament before the third round after aggravating a left knee injury a day earlier when he slipped on wet concrete. J.B. Holmes also withdrew after the first nine holes of his third round with an unknown ailment.

Koepka, playing his second event since stem cell treatment on the knee Aug. 25, shot a 3-over 75 on Friday at Nine Bridges after opening with a 69. He was 13 strokes behind Thomas in the limited-field event with no cut.

After consulting with my doctor, I have decided to withdraw from the CJ Cup and head home for further tests, Koepka said in a statement. I appreciate everyones concerns and support. Ill keep you posted as I learn more.

Koepka returned to Florida to meet with his doctors, leaving his status for the HSBC Champions in two weeks in China uncertain. He missed the cut two weeks ago in his season debut at Las Vegas.

The CJ Cup is the first of three PGA Tour events in Asia, continuing next week at the Zozo Championship in Japan which will feature Tiger Woods and ending with the World Golf Championship event in Shanghai.

Clarkston native Joel Dahmen shot a 2-under 70 and entered the final round at 1-under 215.

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Justin Thomas, Danny Lee tied for 3rd-round lead at CJ Cup - Sports and Weather Right Now

Crispr’s next frontier is in-human treatment, says co-inventor – The Business Times

By Stem Cell Treatment

Mon, Oct 21, 2019 - 5:50 AM

New York

AS investors await results from the first US clinical trials of the gene-editing system known as Crispr, scientists are focused on finding ways to administer it directly into humans, according to the technology's co-inventor, Jennifer Doudna.

Right now, in studies using Crispr that have treated patients, researchers have had to extract their cells to be able to make edits to faulty DNA before infusing them back into the body for treatment.

Being able to do precise edits directly inside humans, animals or plants could open the door to new applications, Ms Doudna said.

"With advances and delivery techniques, it may be possible to do that kind of very highly efficient targeted genome editing in the patient, without having to remove cells, but actually to just do a treatment in the patient where the delivery vehicle takes the editing molecule to the right cells," she said in an interview before the Welch Foundation Conference on chemical research this week.

"Sounds fantastical today, but I think that's coming."

In essence, Crispr is a gene-editing system that can splice away parts of human DNA that make people susceptible to disease or defects. While it can be used in plants and animals, scientists are working on therapeutic applications that can offer a one-time cure for certain diseases.

Crispr Therapeutics AG was the first company to start a human trial back in February, and is due to report initial results by year-end.

Editas Medicine Inc is leading efforts in "in-vivo", or inside the body, testing and initiated a clinical study in July. Intellia Therapeutics Inc is expected to follow with its own study next year.

A safe delivery of Crispr directly into humans would shorten manufacturing times and offer new opportunities for the companies.

The biggest challenge is to find a way to deliver gene-editing molecules into specific cell types safely and efficiently, Ms Doudna said.

"That's kind of the next frontier," she added. "If we figure that out, it really does open the way to many, many more kinds of applications in genome editing than are possible today."

Crispr and Intellia Therapeutics have licensed their technology from the University of California at Berkeley, Ms Doudna's academic home, while Editas is using inventions from the Broad Institute in Massachusetts.

The two institutions are fighting over who was first to invent breakthrough gene-editing technology. Ms Doudna is a co-founder of Editas and other Crispr startups and is a scientific board member at Intellia.

The gene-editing field, which only recently entered human testing and has been plagued by research raising safety concerns, recently got some encouraging news.

Chinese researchers safely treated a man with leukemia and HIV using gene-edited stem cells, according to a report in the New England Journal of Medicine. While the attempt to cure his HIV failed, his cancer is in remission 19 months after the treatment, and the modified cells integrated into his body.

The case, which is the first detailed report in a major academic journal of how doctors are using Crispr in living patients, is an "important milestone" and suggests that gene editing will be "a safe technology and that the challenge now is to have it be really effective in different disease settings", Ms Doudna noted. BLOOMBERG

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Crispr's next frontier is in-human treatment, says co-inventor - The Business Times

Damien Cook confident that Sam Burgess will continue playing despite shoulder injury – Sporting News AU

By Stem Cell Treatment

South Sydney and Australian hooker Damien Cook is confident that club teammate Sam Burgess will continue playing on despite a lingering shoulder injury.

Rumours of Burgess retirement continue to swirl after it was revealed thathe wasconsidering his playing future due to arthritis in his shoulder.

It was reported last week that the English international was set to travel to the United States to undergo stem cell treatment in the hopes to treat the issue.

This ledThe Australian'sBrent Read to speculate about his future, tellingSporting News Australiathat he would lucky to see out his current contract.

"The shoulder is a massive issue," Read said.

"He's got three years left, I'd be stunned if he saw out the three years.

"He may play one more and then be gone,

"I think that's how bad it is."

However, teammate Damien Cook was not concerned about his potential retirement, revealing that he had no doubts about his playing future.

"Sam is someone who brought himself back early a few times because we needed him back on the field to help get us to the finals," Cook told the media.

"Now he just needs a good rest and to look after his shoulder.

"We want to make sure first and foremost his health is there.

"You don't want any permanent damage to any player after their career.

"Sam has given his whole body and everything to the game.

"I've got no doubts he will be playing with us next year."

Cook is currently in Kangaroos camp preparing for their clash against New Zealand on Friday in Wollongong, with the hooker eager to avenge the loss from 12 months ago.

"It was my first game in the jersey and first loss Australia had in a while," Cook said.

"It wasn't the best memory but getting that chance was special. We didn't play our best footy but still managed to score some points.

"This time we don't want to wait for the Tonga game to get it right."

Originally posted here:
Damien Cook confident that Sam Burgess will continue playing despite shoulder injury - Sporting News AU

Teen Cancer Survivor uses Haunted House to Help Others Fight – WFMYNews2.com

By Stem Cell Treatment

RALEIGH, N.C. WNCN - A 14-year-old cancer survivor is giving back to other children with cancer, and hes using a haunted house to do it.

According to WNCN CBS 17, a spooky scene on S. Mere Court in Raleigh has a haunted house filled with werewolves, spiderwebs, and just about everything else you find creepy, but the boy behind it is all smiles.

I always wanted to do a haunted house but mom and dad would always say no, Edward Thompson told WNCN. They hate me scaring them.

The Thompsons have been through something so much scarier than this, though. Hodgkins lymphoma. Edward was diagnosed with it, on the last day of 6th grade. He needed chemotherapy, radiation, and a stem cell transplant to fight cancer.

One day I was very depressed since I was going through all this. I think I had just relapsed, Edward told WNCN. Mom said, You know what. This year were going to do a haunted house.'

He got so excited and he would sit there during chemo and plan out the haunted house, added his mom.

Im reminded of what I went through and how I got through it, said Edward, who even included the mask he used for radiation treatment among the Halloween decorations.

The house will be open the nights of October 26, 27, 30, and 31st.

Its free to tour, but the family told WNCN they are accepting donations for Give Kids the World a resort for children who are part of the Make-A-Wish program. Edward went after finishing treatment.

We werent the oddballs anymore. We werent the outcasts; we werent the kids with cancer, he remembered. Im giving back to them because I know what theyve been through.

After dealing with very real fear for a very long time, Edward still loves to scare people. I love to scare. I love to get scared. Scaring is just a part of me, he said.

The haunted house is located at:9401 S. Mere Ct. Raleigh, NC 27615 It is open October 26, 27, 30, and 31st from 6:30 p.m. until 8 p.m.

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Teen Cancer Survivor uses Haunted House to Help Others Fight - WFMYNews2.com

Five ways fasting can help in the fight against cancer – The Standard

By Stem Cell Treatment

ALSO READ: What you need to know about prostate cancer

There have been many questions on the role fasting plays in the fight against cancer and during cancer treatment.

Different studies suggest that fasting can help fight cancer by lowering the resistance of insulin and also helps in fighting the side effects of chemotherapy.

Scientists also believe that fasting can make cancer cells more responsive to chemo and at the same time protect blood and other cells from the harsh side effects of chemotherapy treatment.

Below are some of the positive effects fasting has on cancer and chemotherapy treatment.

1. It improves insulin sensitivity

Insulin is a hormone that allows cells to extract glucose from the body and use it as energy.

When there is food available in the body, the cells become less sensitive to insulin and create insulin resistance, meaning the cells will no longer respond to insulin signals which will lead to high levels of glucose in the blood and higher storage of fat.

ALSO READ: HPV vaccine: Why your daughter must get the shot, your son too

The body tries to conserve as much energy as possible when it doesn't have enough food and this task is only accomplished by making the cell membranes more sensitive to insulin.

During fasting, cells metabolize insulin more effectively and remove glucose from the blood.

When there is better insulin sensitivity in the body, it makes the conditions harder for any cancer cells to grow and develop.

2. Reverses effects of chronic illnesses

Type 2 diabetes and obesity have been said to be some of the risk factors of cancer and they are both linked to people being at a higher risk of getting multiple types of cancers and having low survival rates.

A case study carried out in 2017 that looked at the effects of intermittent fasting on type 2 diabetes showed that after a period of four months, the patient who participated had a 17.8 percent in weight reduction and had reduced their waist sizes as well.

The study showed that after two months of constant short term fasting, the type 2 diabetes patients no longer needed insulin treatment after only two months.

ALSO READ: Six ways you can reduce risk of cancer

3. It promotes autophagy

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Autophagy is a cellular process where parts of cells break down for reuse later. This process is critical for maintaining the proper functioning of cells and it helps defend cells in the body. Autophagy also plays a major role in the prevention and treatment of cancer.

Different studies and tests carried out in mice showed that the lack of autophagy leads to one having lower levels of important tumour suppressing genes.

Even though having low autophagy can enable a tumour formation, this is not exactly responsible for the growth and spreading of a malignant tumour.

4. Improves response to chemo during treatment

Scientists believe that fasting tends to improve one's response to chemotherapy because it promotes cellular regeneration, reduces the impact of chemo side effects and it protects the blood against harmful effects of chemo.

A study carried out in 2018 that used a 60 hour fasting period for testing, and started 36 hour before the start of chemo sessions discovered that fasting can help improve the quality of life of cancer patients undergoing chemo treatment for ovarian cancer and breast cancer.

The result of the research showed that the patients who were fasting during chemotherapy had reported a higher tolerance to chemo and had fewer side effects to the treatment and had high energy levels.

5. Boosts the immune system to help it fight cancer

A study conducted in 2014 that focused on whether fasting produces any cancer fighting effects in mice stem cells as stem cells are important because of their regenerative ability revealed that fasting for two to four days could protect stem cells against negative effect of chemotherapy on the immune system.

The study also revealed that fasting activated stem cells in the immune system in order to renew them and enable them to repair themselves.

According to the study, fasting does not only reduce the damage to the cells but it also replenishes white blood cells and replenishes the damaged ones.

When white blood cells levels drops due to chemotherapy, it affects the immune system since white blood cells are meant to fight infections and destroy any cells that cause diseases, which leaves one exposed to infections since the body has a harder time fighting any infection.

When fasting, the number of white blood cells tend to reduce but the number goes up once the fasting period is over and the body gets the right amount of food.

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Five ways fasting can help in the fight against cancer - The Standard

Stem cell therapy is for animals too – SciTech Europa

By Somatic Stem Cells

Stem cell therapy for animals has seen breakthroughs

Stem cell therapy is increasingly becoming a more mainstream form of medicine. Usually applied to humans, the use of this regenerative treatment is now also being extended to animals including cats and dogs. Regenerative medicine, particularly stem cell treatment has seen many advancements in recent years with some groundbreaking studies coming to light.

Taking the cells from bone marrow, umbilical cords, blood or fat, stem cells can grow to become any kind of cell and the treatment has seen many successes in animals. The regenerative therapy has been useful particularly for treatment of spinal cord and bone injuries as well as problems with tendons, ligaments and joints.

Expanded Potential Stem Cells (EPSCs) have been obtained from pig embryos for the first time. The cells offer groundbreaking potential for studying embryonic development and producing transnational research in genomics and regenerative medicine, biotechnology and agriculture.

The cells have been efficiently derived from pig preimplantation embryos and a new culture medium developed in Hong Kong and Cambridge enabled researchers from the FLI to establish permanent embryonic stem cell lines. The cells have been discovered in a collaboration between research groups from the Institute of Farm Animal Genetics at the Friedrich-Loeffler-Institut (FLI) in Mariensee, Germany, the Wellcome Trust Sanger Institute in Cambridge, UK and the University of Hong Kong, Li Ka Shing Faculty of Medicine, School of Biomedical Sciences.

Embryonic stem cells (ESC) are derived from the inner cells of very early embryos, the so-called blastocysts. Embryonic stem cells are all-rounders and can develop into various cell types of the body in the culture dish. This characteristic is called pluripotency. Previous attempts to establish pluripotent embryonic stem cell lines from farm animals such as pigs or cattle have resulted in cell lines that have not really fulfilled all properties of pluripotency and were therefore called ES-like.

Dr Monika Nowak-Imialek of the FLI said: Our porcine EPSCs isolated from pig embryos are the first well-characterized cell lines worldwide. EPSCs great potential to develop into any type of cell provides important implications for developmental biology, regenerative medicine, organ transplantation, disease modelling and screening for drugs.

The stem cells can renew themselves meaning they can be kept in culture indefinitely, and also show the typical morphology and gene expression patterns of embryonic stem cells. Somatic cells have a limited lifespan, so these new stem cells are much better suited for long selection processes. It has been shown that these porcine stem cell lines can easily be modified with new genome editing techniques such as CRISPR/Cas, which is particularly interesting for the generation of porcine disease models.

The EPSCs have a high capacity to develop not only into numerous cell types of the organism, but also into extraembryonic tissue, the trophoblasts, making them very unique and lending them their name. This capacity could prove valuable for the future promising organoid technology, where organ-like small cell aggregations are grown in 3D aggregates that can be used for research into early embryo development, various disease models and testing of new drugs in petri dishes. In addition, the authors were able to show that trophoblast stem cells can be generated from their porcine stem cells, offering a unique possibility to investigate functions or diseases of the placenta in vitro.

A major hurdle to using neural stem cells derived from genetically different donors to replace damaged or destroyed tissues, such as in a spinal cord injury, has been the persistent rejection of the introduced material (cells), necessitating the use of complex drugs and techniques to suppress the hosts immune response.

Earlier this year, an international team led by scientists at University of California San Diego School of Medicine successfully grafted induced pluripotent stem cell (iPSC)-derived neural precursor cells back into the spinal cords of genetically identical adult pigs with no immunosuppression efforts. The grafted cells survived long-term, displayed differentiated functionality and caused no tumours.

The researchers also demonstrated that the same cells showed similar long-term survival in adult pigs with different genetic backgrounds after only short course use of immunosuppressive treatment once injected into injured spinal cord.

Senior author of the paper Martin Marsala, MD, professor in the Department of Anesthesiology at UC San Diego School of Medicine said: The promise of iPSCs is huge, but so too have been the challenges. In this study, weve demonstrated an alternate approach.

We took skin cells from an adult pig, an animal species with strong similarities to humans in spinal cord and central nervous system anatomy and function, reprogrammed them back to stem cells, then induced them to become neural precursor cells (NPCs), destined to become nerve cells. Because they are syngeneic genetically identical with the cell-graft recipient pig they are immunologically compatible. They grow and differentiate with no immunosuppression required.

Co-author Samuel Pfaff, PhD, professor and Howard Hughes Medical Institute Investigator at Salk Institute for Biological Studies, said: Using RNA sequencing and innovative bioinformatic methods to deconvolute the RNAs species-of-origin, the research team demonstrated that pig iPSC-derived neural precursors safely acquire the genetic characteristics of mature CNS tissue even after transplantation into rat brains.

NPCs were grafted into the spinal cords of syngeneic non-injured pigs with no immunosuppression finding that the cells survived and differentiated into neurons and supporting glial cells at all observed time points. The grafted neurons were detected functioning seven months after transplantation.

Then researchers grafted NPCs into genetically dissimilar pigs with chronic spinal cord injuries, followed by a transient four-week regimen of immunosuppression drugs again finding long-term cell survival and maturation.

Marsala continued: Our current experiments are focusing on generation and testing of clinical grade human iPSCs, which is the ultimate source of cells to be used in future clinical trials for treatment of spinal cord and central nervous system injuries in a syngeneic or allogeneic setting.

Because long-term post-grafting periods between one and two years are required to achieve a full grafted cells-induced treatment effect, the elimination of immunosuppressive treatment will substantially increase our chances in achieving more robust functional improvement in spinal trauma patients receiving iPSC-derived NPCs.

In our current clinical cell-replacement trials, immunosuppression is required to achieve the survival of allogeneic cell grafts. The elimination of immunosuppression requirement by using syngeneic cell grafts would represent a major step forward said co-author Joseph Ciacci, MD, a neurosurgeon at UC San Diego Health and professor of surgery at UC San Diego School of Medicine.

Other recent advancements include the advancement toward having a long-lasting repair caulk for blood vessels. A new method has been for generating endothelial cells, which make up the lining of blood vessels, from human induced pluripotent stem cells. When endothelial cells are surrounded by a supportive gel and implanted into mice with damaged blood vessels, they become part of the animals blood vessels, surviving for more than 10 months.

The research was carried out by stem cell researchers at Emory University School of Medicine and could form the basis of a treatment for peripheral artery disease, derived from a patients own cells.

Young-sup Yoon, MD, PhD, who led the team, said: We tried several different gels before finding the best one. This is the part that is my dream come true: the endothelial cells are really contributing to endogenous vessels.

When cells are implanted on their own, many of them die quickly, and the main therapeutic benefits are from growth factors they secrete. When these endothelial cells are delivered in a gel, they are protected. It takes several weeks for most of them to migrate to vessels and incorporate into them.

Other groups had done this type of thing before, but the main point is that all of the culture components we used would be compatible with clinical applications.

This research is particularly successful as previous attempts to achieve the same effect elsewhere had implanted cells lasting only a few days to weeks, using mostly adult stem cells, such as mesenchymal stem cells or endothelial progenitor cells. The scientists also designed a gel to mimic the supportive effects of the extracellular matrix. When encapsulated by the gel, cells could survive oxidative stress inflicted by hydrogen peroxide that killed unprotected cells. The gel is biodegradable, disappearing over the course of several weeks.

The scientists tested the effects of the encapsulated cells by injecting them into mice with hindlimb ischemia (restricted blood flow in the leg), a model of peripheral artery disease.

After 4 weeks, the density of blood vessels was highest in mice implanted with gel-encapsulated endothelial cells. The mice were nude, meaning genetically immunodeficient, facilitating acceptance of human cells.

The scientists found that implanted cells produce pro-angiogenic and vasculogenic growth factors. In addition, protection by the gel augmented and prolonged the cells ability to contribute directly to blood vessels. To visualise the implanted cells, they were labelled beforehand with a red dye, while functioning blood vessels were labelled by infusing a green dye into living animals. Implanted cells incorporated into vessels, with the highest degree of incorporation occurring at 10 months.

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Stem cell therapy is for animals too - SciTech Europa

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