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Heartbreaking story of leukaemia ‘guinea pig’ children confined to isolation rooms – Mirror Online

By Stem Cell Doctors

Every day ended the same way. Alan Corley kissed his father good night through a pane of glass, then retreated to his hospital bed clinging to his Mr Happy toy for comfort.

Diagnosed with leukaemia when he was five years old, Alan was confined to a cell like isolation room at Westminster Childrens Hospital as the slightest cold or infection could prove fatal.

Dad Andy even had to put on a gown, face mask, and rubber gloves before he could enter. Alans one other friend was the little boy in the next isolation room. That boy was Anthony Nolan.

Despite a global hunt for a bone marrow donor to cure Anthonys rare genetic condition, Wiskott-Aldrich syndrome, he died on October 21, 1979. He was seven-years-old.

His mum Shirleys tireless efforts to save him led to the creation of the bone marrow donor register and the Anthony Nolan charity.

It has since found matching donors for more than 16,000 people in need of a stem cell transplant, many of them kids with leukaemia like Alan.

Neither Alan or Anthony were expected to survive, yet Alan was lucky.

After being a guinea pig for high doses of chemo and radiotherapy he recovered. Alan, 46, from Barrow in Suffolk, says: I recently realised what a key figure Anthony was in my life.

I spent six months in that hospital and much of that time, he was the only person I could see. We would talk and play card games like Twist through the glass. When I was well enough to move to my local hospital, Anthony was alive.

I went back to Westminster a few months later for bone marrow tests. I went to see Anthony, but he wasnt there. He had passed away.

That happened all the time. My dad would see children running around the ward one day, the next he would see a four-foot body bag in the hall.

Alan was diagnosed with leukaemia after struggling to shake off flu in September 1978 and being sent for blood tests. It was 18 months before he was well enough to return to school.

He was admitted to the hospital in St Albans, where his family then lived and given a lifesaving blood transfusion before being transferred to Westminster.

Doctors feared the worst when chemotherapy and radiation treatment failed.

Alan says: One morning my dad came down and saw I was out of isolation. The doctors said, We dont think he will be here by the end of the week, so we have allowed him out to play with the rest of the children. That evening all my family came, preparing to say goodbye.

However, the doctors decided to give me a second round of treatment, much more aggressive than the first. I was given horrific volumes of chemotherapy to see if my body could take it. We were guinea pigs on that ward, because we had no other hope.

Thankfully it worked, so Alan did not need a transplant. A few months after Alan was diagnosed, his younger sister Angela was born and found to be a match. Had his treatment been unsuccessful she would have been his bone marrow donor.

By the time Alan arrived on the ward, Anthony had been in and out of Westminster Childrens Hospital for six years.

His mum had brought him to England from Australia in the hope of finding a bone marrow donor to cure his otherwise fatal blood disease.

At the time there was no donor register and Britain was in the midst of a recession. NHS cutbacks meant the search soon ground to a halt, leaving Shirley to find funds and volunteers to collect and test samples to see if potential donors were a match.

Shirleys pioneering work, charted in her book A Kiss Through Glass, led to the creation of the first bone marrow donor register in 1974.

Alan says: My dad and Shirley spent a lot of time with each other. Theyd go for a drink occasionally trying to get a bit of normality and hit the refresh button ready to be back next day.

But there were some good times. My dad made pancakes for Shrove Tuesday. Shirley asked if he would make some for Anthony, then the nurses asked him to make some more.

So my dad used the tiny stove in my room to make pancakes for the entire ward, including the nurses.

Comic actor Roy Kinnear, best known for Willy Wonka and the Chocolate Factory and the hit show That Was The Week That Was, regularly visited the ward where his daughter Karina was being treated for cerebral palsy and delighted the children with his jokes.

Forty years later Alan still has his treasured Mr Happy toy which kept him company in his isolation room.

He also has two miracle daughters, Francesca, 20, and Philippa, 17, despite being told the radiation he was blasted with would leave him infertile. Both are ardent Anthony Nolan supporters.

Andy suffers with severe back pain as chemicals injected into his spine caused several vertebrae to crumble. Hes also battled high blood pressure .

Despite this, he hopes to climb to Everest Base Camp in April next year with his brother-in-law and father-in-law and has been training with gruelling treks.

Alan, a manager at Royal Mail, says: Everything we went through has helped so many children. Im lucky to be here and I just want to make the most of my life. That is why I hope to climb Everest to raise money and awareness for Anthony Nolan.

Many know of the charity and its vital work, but dont understand where it came from. They dont remember the little boy who went through so much.

Anthonys life may have been short, but had incredible impact. Thousands have been given a second chance of life. What a legacy.

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Heartbreaking story of leukaemia 'guinea pig' children confined to isolation rooms - Mirror Online

In 2030, Our Protein Will Come From a Laband We’ll All Be Better Off For It – Singularity Hub

By Adult Stem Cells

Could a hamburger grown in a lab from Kobe beef stem cells be cheaper, better tasting, and healthier for you? Can you imagine a future where millions of square miles of pastoral land are reclaimed by nature, creating new forests and revitalizing the Earths vital carbon sinks?

Last week, we discussed the hyper-efficient food production systems of 2030. This week, we continue that discussion, but from a different perspectivebecause by the end of the next decade, we will witness the end of industrial animal agriculture as we know it.

Through the convergence of biotechnology and AgTech, we will witness the birth of the most ethical, nutritious, and environmentally sustainable food system ever devised by mankind.

Lets dive in.

Meat production is problematic, to say the least. A quarter of the planets available landmass is currently used to keep 20 billion chickens, 1.5 billion cattle, and 1 billion sheep alivethat is, until we can kill them and eat them. The suffering quotient is through the roof. As is the waste.

Worse is the water involved. Meat production accounts for 70 percent of global water use. Compared to 1,500 liters required to produce a kilogram of wheat, it takes 15,000 liters to produce a kilogram of beef, meaning theres enough water in an adult steer to float a US Navy destroyer.

Meat is also responsible for14.5 percentof all greenhouse gases and a considerable portion of our deforestation problem. In fact, we are in the midst of one of the largest mass extinctions in history, and loss of land for agricultural production is currently the largest driver of that extinction.

Enter cultured meat: meat that is grown from a few cells into a full-blown steak.

Take a few stem cells from a live animal, typically via a biopsy so the animal isnt harmed. Feed these cells a nutrient-rich solution. Power the whole process in bioreactors. Give the industry a few years to mature and the technology a few years to shed costs and, finally, we can produce an infinite number of steaks to feed an increasingly carnivorous population.

There are still numerous hurdles to overcome in the process, but we are fast approaching the point at which converging exponential technologies will enable this transformation of todays food system.

Environmental issues aside, cultured meat has the potential to become far more cost-effective than conventional meat. It will soon compete with the latter on almost every market-oriented criteria in existence.

For starters, cultured meat production is mostly an automated process without much need for land or labor. Plus, it takes a few years to grow a cow in the wild, but only afewweeksto grow a cows worth of steak in the lab.

And its more than just steak. The meats in development range from pork sausage and chicken nuggets to foie gras and filet mignonit all depends on which stems cells you start out with.

In late 2018, for example, Just Foods announced a partnership with Japanese Wagyu beef producer Toriyama to develop cultured meat from the cells of what has long been the rarest and most expensive steaks on Earth.

And whats true for meat is also true for milk.

Perfect Day Foods, a Berkeley, California-based company started by two founders with a passion for cheese, has figured out how to make the animal-derived product without any involvement from cows. Combining gene sequencing with 3D printing and fermentation science, theyve created a line of animal-free dairy products.

So what does this all mean? A fundamental reconfiguration of the way we source, consume, and pay for foodnot to mention the environmental costs that are often written off as externalities.

Such a transformation will revamp our world in ways we have only begun to imagine.

The decimation of resources alone is considerable. Cultured meat uses 99 percent less land, 82-96 percent less water, and produces 78-96 percent less greenhouses gases. Energy use drops somewhere between 7 and 45 percent depending on the meat involved (traditional chicken ranching is much more energy-intensive than traditional beef ranching).

And by liberating a quarter of our landmass, we can also reforest, providing sufficient habitat required to halt the biodiversity crisis and revitalize tremendous natural carbon sinks needed to slow global warming.

While thats a haze of numbers to consider, what they add up to is astounding: An ethical and environmental solution to world hunger.

Its also a muchhealthiersolution. As we will soon be growing steak from stem cells, we can do the impossible: Make fast food hamburgers that are actually good for you. Well be newly able to increase helpful proteins, reduce saturated fat, even add vitamins.

Another huge win: cultured meat requiresno antibiotics. Given the danger of diseases like mad cow disease, next-gen meat consumption will be far safer for humans, reducingif not eliminatingthe food industrys industrial hygiene challenges.

In fact, as a high percentage of emerging diseases come from livestock, by turning to cultured meat, were both lowering the global disease burden and decreasing our risk of pandemic.

Over the last two weeks, we have explored how converging exponentials will revolutionize one of humanitys most basic needs. By the end of the next decade, anyone anywhere will have on-demand, ultra-cheap access to lab-grown meatfar more nutritious than livestock-derived products, with a near-zero carbon footprint and safety guarantees.

Dont want to leave home? The rise of vertical farming, autonomous drone networks, and last-mile delivery advancements will make food deliverable to your doorstep, sourced from a low-land-use food production center. Local foods will be truly local. Either that, or download physiology-optimized recipes to your in-home food 3D printer.

While traditional agriculture has experienced shifts and industrialization, growing food has roughly been the same since10,000 BC.

Soon to undergo one of the most monumental technological revolutions in history, our food system is about to be leagues more efficient, ethical, and sustainable than ever beforenot to mention far healthier.

In just a few years, humans will become the first animal that derives our protein from other animals, yet doesnt harm anyone in the process. Meat milesthat is, the transportation costs involved in moving meat aroundwill nearly disappear. Slaughterhouses will become a ghost story we tell our grandchildren.

And a planet that is already significantly strained under the weight of seven billion will have a fighting chance as we grow to ~10 billion by 2050.

Image Credit: Photo byCarolien van OijenonUnsplash

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In 2030, Our Protein Will Come From a Laband We'll All Be Better Off For It - Singularity Hub

Skill Checkup: Knee Injection – Medscape

By Platelet Rich Plasma Injections

Knee pain and stiffness can be debilitating and difficult to treat. Lifestyle-limiting knee conditions may negatively affect body image and emotional well-being. Weight management, exercise/strengthening programs, physical therapy, physical modalities, orthotics, medications, intra-articular knee injections, and surgery are some of the approaches used to treat knee pain.

The most common type of intra-articular knee injection is with corticosteroids, but other agents used include infliximab, hyaluronic acid, botulinum neurotoxin, and platelet-rich plasma (PRP).

Steroid injections have been shown to relieve pain and inflammation in individuals with osteoarthritis (including osteoarthritis complicated by Baker cysts), juvenile idiopathic arthritis, psoriatic arthritis, acute monoarticular gout, pseudogout, and rheumatoid arthritic knees.

Intra-articular infliximab can be used to treat refractory knee monoarthritis/synovitis in patients with rheumatoid arthritis, Behet disease, and spondyloarthropathy (eg, ankylosing spondylitis) that is resistant to systemic treatment.

Intra-articular knee injections of hyaluronic acid have been shown to provide functional and perceived benefits in knee osteoarthritis. Such injections have also been shown to be helpful in patients with knees that are both rheumatoid arthritic and osteoarthritic.

Intra-articular injection of botulinum neurotoxin A into the knee joint may provide therapeutic pain relief in patients with advanced knee osteoarthritis.

Intra-articular knee injections of homologous PRP have been shown to improve function and quality of life in patients with degenerative lesions of the knee cartilage and osteoarthritis at 6 months post-injection.

Careful initial palpation and marking of the injection site may reduce the need to repalpate an already prepared site. During the initial marking of the intra-articular injection target site, the knee should be flexed 90 to expose the joint space for the anteromedial or anterolateral approach and almost fully or fully extended for the superolateral or superomedial approach. The selected skin site for injection can be marked. Sterile gloves may be used.

Using sterile techniques, skin over the target area may be prepared with iodine disinfectant x 3, allowed to air-dry, and then wiped with alcohol prior to needle placement; alternatively, chlorhexidine may be used for skin preparation in place of iodine plus alcohol.

Any number of the relatively insoluble injectable corticosteroids, including triamcinolone acetonide 10-40 mg, triamcinolone hexacetonide 10-40 mg, or prednisolone acetate 10-25 mg; or slightly soluble corticosteroids, such as methylprednisolone acetate 40-80 mg or triamcinolone diacetate 20-40 mg, may be used.

A 10- to 15-s stream of ethyl chloride topical anesthetic spray can be steadily directed at the skin area over the target injection site prior to needle advancement. Lidocaine 1%-2% can be injected over the target site via a 25-gauge 1.5-in needle after negative aspiration for further numbing effect prior to the steroid injection, or it can be injected directly into the knee joint as a mixture with corticosteroid.

For the anterolateral or anteromedial approach, the patient can be in the sitting or supine position, with the knee flexed to 90 to allow easy access to the joint capsule. Knee radiography would show whether medial or lateral joint-space narrowing predominates.

For the superolateral or superomedial approach, the knee is almost fully or is fully extended to allow gentle rocking of the patella. The needle is directed under the proximal patella near and parallel to the undersurface of the quadriceps tendon insertion on the patella.

The best approach to a knee injection is the path of least obstruction and maximal access to the synovial cavity, which could be superolateral, superomedial, or anteromedial/anterolateral.

Superolateral approach

For the superolateral approach, the patient lies supine with the knee almost fully or fully extended, with a thin pad support underneath the knee to facilitate relaxation. The clinician's thumb is used to gently rock and then stabilize the patella while the needle is inserted underneath the superolateral surface of the patella, aimed toward the center of the patella, and then directed slightly posteriorly and inferomedially into the knee joint.

Superomedial approach

For the superomedial approach, the patient lies supine with the knee almost fully or fully extended, with a thin pad support underneath the knee to facilitate relaxation. The clinician's thumb is used to gently rock and then stabilize the patella while the needle is inserted underneath the superomedial surface of patella, aimed toward the center of the patella, and then directed slightly posteriorly and inferolaterally into the knee joint.

Anterolateral and anteromedial approaches

For the anterolateral and anteromedial approaches, the patient can sit or lie supine with the knee flexed 90 to afford better exposure of the intra-articular surface and thus facilitate ease of needle entry into the joint space.

The sterile needle is inserted either lateral to the patellar tendon (for the anterolateral approach) or medial to the tendon (for the anteromedial approach), approximately 1 cm above the tibial plateau, and directed 15-45 from the anterior knee surface vertical midline toward the intra-articular joint space.

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Skill Checkup: Knee Injection - Medscape

Medicare Coverage of CAR-T Cell Therapy Raises New Questions – The Heartland Institute

By Uncategorized

Still to be determined is how hospitals and other health care facilities will be reimbursed for the therapy and whether patients will have access to the therapy under health care proposals such as Medicare for All or a so-called public option.

One of the most promising cancer treatments to come along in years, CAR-T cell therapy uses the bodys own immune system to attack and kill cancer cells. The treatment involves bioengineering T cells, a white blood cell that fights foreign substances in the body, and equipping them with new Chimeric Antigen Receptors that target cancer cells.

CAR-T cell therapy has been approved by the U.S. Food and Drug Administration (FDA) for children with leukemia and adults with advanced lymphoma. The therapy is typically used alongside other, more traditional treatments such as surgery, chemotherapy, and radiation. Its use in combatting other forms of cancer is pending with the FDA, an agency known for its slow approval process.

Hefty Price Tag

There are currently two approved CAR-T treatments: Novartis Kymriah (tisagenlecleucel) and Gileads Yescarta (axicabtagene).

Like most newly introduced cutting-edge treatments, the two products come with a hefty price tag. A course of treatment of Kymriah costs $475,000 for pediatric and young adult patients with leukemia, and both are priced at $373,000 to treat lymphoma in adults, according tobiopharma.com. Under Centers for Medicare & Medicaid Services (CMS) regulations published in August, Medicare will reimburse hospitals for 65 percent of the treatments cost, or about $242,000, through Part B.

Although hospitals will likely welcome Medicares financial commitment, there still remains a sizable gap. Further complicating reimbursement is the lack of a separate Medicare billing code for CAR-T treatment, which will be handled via codes for bone marrow and stem cell transplants until a CAR-T billing code is developed, which could take up to three years.

CMS worked closely with the FDA and the National Cancer Institute in developing the new regulations, a time-consuming process rooted in the complexities of developing a reimbursement scheme and overseeing an innovative and evolving therapy.

At a July 31 Heritage Foundation panel discussion on Medicare for All, CMS Administrator Seema Verma discussed the challenges government programs face in approving innovative treatments.

Much of the problem is when Congress says you can cover durable medical treatment, supplies, and drugs, said Verma. Sounded great when they wrote that law 30 to 40 years ago but doesnt make sense in todays environment. All of these new treatments are coming out, and they dont fit nicely into the way the law has been constructed, and it creates problems for the agency.

Bonner R. Cohen,Ph.D.,(bcohen@nationalcenter.org)is a senior fellow at the National Center for Public Policy Research and a senior policy analyst with the Committee for a Constructive Tomorrow (CFACT).

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Medicare Coverage of CAR-T Cell Therapy Raises New Questions - The Heartland Institute

DiscGenics Announces US Clinical Study of Cell Therapy for Disc Degeneration Clears Final Planned Safety Review – PRNewswire

By Uncategorized

The DSMC completed a third and final planned mid-trial safety review following treatment of the first six subjects in the high dose study cohort, each of whom was treated based on random assignment into one of three arms: high dose IDCT study treatment, vehicle or placebo.

The DSMC reported there were no safety issues and recommended that the study proceed with completion of patient enrollment with no changes to the protocol.

This news closely follows a recent company announcement that the second planned safety review of the first 30-subject low dose study cohort revealed no safety issues.

"We are delighted to commence the final enrollment stage in our U.S. study of IDCT for DDD and are thrilled that the first 36 patients have now been safely treated," said Flagg Flanagan, Chief Executive Officer and Chairman of the Board of Directors for DiscGenics. "This is a blinded, first-in-human study where neither the treating clinicians nor the patients know what treatment is being administered. As a result, performance of periodic safety checks by an unblinded and independent body was essential to ensuring the ongoing safety of IDCT in a clinical setting. The members of the DSMC played a critical role in this process and we would like to sincerely thank them for their time and thoughtful review of the blinded safety data."

Patient enrollment in this U.S. study will now continue through completion of 60 total subjects.

About the IDCT TrialThe IDCT trial is a prospective, randomized, double-blinded, vehicle- and placebo-controlled, multicenter clinical study to evaluate the safety and preliminary efficacy of IDCT in subjects with single-level, symptomatic lumbar intervertebral disc degeneration. The trial is underway in 14 centers across 12 states in the U.S. and will enroll 60 subjects. Those subjects who meet all eligibility criteria are being randomized to one of four treatment cohorts: low dose IDCT (n=20), high dose IDCT (n=20), vehicle (n=10) and placebo (n=10). Each subject receives a single intradiscal injection of his or her assigned treatment into the target symptomatic lumbar intervertebral disc. Following treatment, subjects will be observed and evaluated for a period of one year, with a one-year extension period. Primary outcome measures include safety and reduction in pain. Secondary outcome measures include reduction in disability and radiographic improvement.

Through this study, IDCT is being evaluated under an investigational new drug (IND) allowanceby the U.S. Food and Drug Administration (FDA) and will be regulated as a drug-biologic through a therapeutics biologics license application (BLA). Importantly, DiscGenics announced in August 2019that the FDA granted Fast Track designation for IDCT as a potential treatment option for chronic low back pain. For more information on the U.S. study, please visit: https://clinicaltrials.gov/ct2/show/NCT03347708.

IDCT is also being evaluated in a multicenter safety study in Japan, which is supported by a Clinical Trial Notification (CTN) approved by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). For more information on the Japanese study, please visit: https://clinicaltrials.gov/ct2/show/NCT03955315.

About DiscGenicsDiscGenics is a privately held, clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. As the only company in the world to develop an allogeneic cell therapy derived from intervertebral disc cells to treat diseases of the disc, DiscGenics believes it has a unique opportunity to harness the restorative potential of the human body to heal millions of patients suffering from the debilitating effects of back pain. DiscGenics' first product candidate, IDCT, is a homologous, allogeneic, injectable cell therapy that utilizes biomedically engineered progenitor cells derived from intervertebral disc tissue, known as Discogenic Cells, to offer a non-surgical, potentially regenerative solution for the treatment of patients with mild to moderate degenerative disc disease. For more information, visit discgenics.com.

Media ContactLindsey Saxonlindsey@discgenics.com

SOURCE DiscGenics, Inc.

http://www.discgenics.com

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DiscGenics Announces US Clinical Study of Cell Therapy for Disc Degeneration Clears Final Planned Safety Review - PRNewswire

Pioneers of cellular reprogramming and cell therapies join bit bio – PRNewswire

By Uncategorized

CAMBRIDGE, United Kingdom, Oct. 28, 2019 /PRNewswire/ -- bit bio(formerly Elpis Biomed) today announced its founding team, a group of pioneering scientists and business executives at the forefront of stem cell biology, cellular reprogramming and cell therapies. Biotech entrepreneur and scientist Paul Morrill will join as Chief Business Officer; and biotech veteran Florian Schuster will join as CFO/COO.

The company also announced today two additions to its scientific advisory board: Marius Wernig, a pioneer in cell reprogramming and Ramy Ibrahim, a leading innovator in the field of immuno-oncology. They join Chief Scientific Advisor Roger Pedersen, a central figure in the field of human stem cell biology.

Recent successes in cell therapy have sparked new hopes for the treatment of cancer. But the limited availability of human cells make them expensive and restrict their application. Drug development is also affected by the lack of human cells: differences between the animal models currently used and human biology are the major reasons why drugs fail in clinical trials.

Founded by stem cell biologist and neurosurgeon Mark Kotter, bit bio is commercializing Opti-OX, a proprietary technology platform for the efficient and consistent reprogramming of human cells for use in research, drug discovery, and cell therapy. With the expansion of its team, bit bio is entering its next phase of development to transform drug discovery and enable a new generation of cell therapies.

"The next generation of medicine hinges on widespread access to human cells," said Kotter. "That is the challenge we have set out to solve at bit bio, and I am incredibly proud to announce our founding team, which features innovators who have not only helped invent our field but who continue to push the envelope."

"At bit bio, we all share the same core ethos: democratizing access to human cells for the benefit of patients," added Chief Business Officer Paul Morrill. "I'm excited to join this world-class team as we work toward this ambitious goal."

Paul Morrill(PhD) joins the company as Chief Business Officer. Paul is an entrepreneur and scientist with over thirty years of experience in the biotech and pharma industries. Most recently, he was the commercial founder and President of Horizon Discovery Group, and founder of CellRx Limited, a growth factors company serving the biopharmaceutical, stem cell and research sectors. Paul holds a PhD in Biotechnology from the University of Cambridge.

Florian Schusterjoins as CFO / COO. Florian is an active entrepreneur and investor. Previously, he was CFO & Head of Strategic Partnerships for Tessa Therapeutics, a clinical-stage cell therapy company. Florian is a former investment banker, a graduate of the Stanford School of Engineering, and an alumnus of Harvard Business School.

Ramy Ibrahim(MD) joins as a member of the scientific advisory board. Ramy is a leading immuno-oncology clinician who has helped to develop some of the breakthrough therapies in this field. Ramy is currently the Chief Medical Officer at the Parker Institute of Cancer Immunotherapy. He has served as the Vice President of Clinical Development for Immuno-oncology at AstraZeneca, and as a member of the Bristol-Myers Squibb Immuno-oncology program.

Marius Wernig(MD PhD) joins as scientific advisory board member. Marius is a pioneer in cellular reprogramming. Marius' seminal 2010 paper in Nature demonstrating direct conversion of fibroblasts into neurons has sparked a widespread interest in cell reprogramming. His lab uses cellular reprogramming to understand how neurons are induced, and how they mature and maintain their identity. Marius is professor and co-director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine.

This team joins Mark Kotter and Roger Pedersen:

Mark Kotter(MD PhD, Founder/CEO)is a stem cell biologist and neurosurgeon at the University of Cambridge. By combining synthetic and stem cell biology, his team has developed a benchmark technology for the efficient and consistent production of human cells for use in research, drug development, and cell therapy. He is the founder of bit bio and co-founder of the cultured meat startup Meatable.

Roger Pedersen(PhD, Chief Scientific Advisor)is a pioneer and thought leader in the field of human stem cell biology. His lab was the first to isolate pluripotent epiblast stem cells from the epiblast layer of the developing mammalian embryo. In addition, he developed one of the first cellular reprogramming protocols. Roger was co-founder of the Cambridge Stem Cell Institute. His lab is currently focused on the differentiation of pluripotent stem cells, with potential applications for drug discovery, toxicity testing and cell therapies.

About bit biobit bio is the cell coding company. Based in Cambridge, UK,bit bio's team includes world leaders in stem cell biology, cellular reprogramming and cell therapy who are harnessing the power of synthetic biology to tackle the problem of inconsistency in the production of human cells. bit bio is developing Opti-OX, a proprietary technology platform capable of producing any human cell for research, drug discovery and cell therapy. For more information, visit bit.bio.

Press contact: Ben Kellogg, ben@bwkny.com +1 (917) 816-0831

SOURCE bit bio

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Thought Leadership & Innovation Foundation to Expand Its Regenerative Medicine Program Through New Collaboration with RenovaCare – Business Wire

By Uncategorized

MCLEAN, Va.--(BUSINESS WIRE)--The Thought Leadership & Innovation Foundation (TLI) announces today plans to build on its existing Regenerative Medicine Program through a research collaboration with cellular therapy industry leader RenovaCare. As part of TLIs efforts to conduct vital research in regenerative medicine and chronic disease, this initiative aims to innovate methods for reducing complications from burn and diabetic wounds across large populations.

Our research base, collaborative institutions and long history of innovation align with RenovaCares commitment to breakthrough biomedical technologies, says Bill Oldham, founder and chairman of the Board, TLI. The patented RenovaCare SkinGun technology and its ability to ultra-gently spray stem cells could present a special opportunity for investigations and applications in a wide range of regenerative therapies. Working together, our overall goal is to improve the quality, efficiency and effectiveness of patient care by not only developing new treatment methods, but also by making thoughtful and systematic changes to healthcare and health systems.

TLIs Regenerative Medicine program seeks to adapt new strategies based upon sound scientific evidence, utilizing its infrastructure to support the continuation of scientific and medical work, as well as the development of grant-funded research and other initiatives.

Dr. Robin A. Robinson, who is a TLI Fellow, Vice President of Scientific Affairs, RenovaCare, and named one of the top 100 innovators in medicine by Medicine Maker in 2018, states, This exciting collaboration between RenovaCare and TLIs Regenerative Medicine Program is the first step toward the development of meaningful and quality therapeutic treatments that will benefit patients around the world.

About TLI Foundation:

TLI Foundation is a nonprofit foundation focused on driving innovative thinking and action on global issues relating to health, education and economic empowerment. The organization is committed to fostering transformative change and improving the health and well-being outcomes of communities around the world. Visit https://www.thoughtfoundation.org/

About RenovaCare:

RenovaCare, Inc. is a biotechnology company focused on developing first-of-their-kind autologous (self-donated) stem cell therapies for the regeneration of human organs. Initial products under development target the bodys largest organ, the skin. Investigative clinical use of their flagship technology has shown to be promising new alternatives for patients suffering from burns, and chronic and acute wounds. https://www.renovacareinc.com.

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Thought Leadership & Innovation Foundation to Expand Its Regenerative Medicine Program Through New Collaboration with RenovaCare - Business Wire

News – Michigan’s CON Board Sidelines Promising Cancer Treatment – The Heartland Institute

By Uncategorized

The commission adopted the rule at the behest of several of Michigans established cancer treatment providers and on the advice of a panel of clinical experts it appointed to evaluate the need for the regulation.

The panel also recommended requiring providers to be accredited by the Foundation for the Accreditation of Cellular Therapy (FACT), a national nonprofit organization that inspects cellular therapy facilities.

Keeping Out Competition

CON laws, which in some states are called Certificate of Public Need laws, are designed to keep health care providers from engaging in unnecessary capital outlays that would ultimately be passed on to patients in the form of higher costs. In practice, this means hospitals and other health care providers must get the approval of a state agency before offering new services, expanding their operations, or implementing new medical technologies. Dominant providers frequently use CON laws to limit competition from smaller hospitals.

CAR-T therapy is different from most health care services, states Anna Parsons, a policy coordinator with the American Legislative Exchange Council, because it does not require a capital investment (see commentary).

An FDA-certified hospital should be capable of offering these treatments, since all the high-tech bioengineering is done at other locations, Parsons told Reason.com.

Michigan As Outlier

Michigan state Sen. Curt VanderWall (R-Ludington), who chairs the Senate Health Policy and Human Services Committee, says he opposes CON regulation of CAR-T.

It is concerning to me that the CON commission expanded a requirement into a new clinical service area that goes well beyond the federal requirement, VanderWall toldHealth Care News.

The Centers for Medicare and Medicaid Services makes no mention of FACT accreditation in its August 7 press release stating Medicare will cover the proceedurein healthcare facilities enrolled in the FDA risk evaluation and mitigation strategies for FDA-approved indications.

The new CON requirement to obtain third-party accreditation will be a barrier to access, create an unnecessary financial burden for providers, and limit the sites of care from offering cellular therapies to patients, said VanderWall.

VanderWall says patients should be allowed to choose their own CAR-T provider.

Patient choice and access are priorities for me, said VanderWall. Patients will be able to work with their doctors to find the best treatment site based on safety and access.

Michigan should not be an outlier, VanderWall said. No other state has a CON standard for immune effector cell therapy (IECT), and safe treatment will be offered according to the established federal guidelines in the other 49 states.

A Joint Legislative Committee and the governor were allowed 45 days from the September 19 decision to review the new language to regulate IECT, of which CAR-T is a form, and the FACT accreditation requirement.

AnneMarie Schieber(amschieber@heartland.org)is managing editor ofHealth Care News.

Official Connections:

Michigan State Senator Curt VanderWall, (R Ludington):https://www.senatorcurtvanderwall.com

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News - Michigan's CON Board Sidelines Promising Cancer Treatment - The Heartland Institute

Gene Therapy/Editing Series 1: A Brief Introduction To Gene Therapy – Seeking Alpha

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The recent approval of various gene therapies, for example, Luxturna and Zolgesma and high premium acquisitions of gene therapy companies have shifted the investor focus to this rapidly growing biotechnology field. In this series of review articles, I will review the gene therapy and gene editing field, starting first with the basics, including a brief overview of the history of the field and then moving on to some technical aspects, for example, the manufacturing, different methods of delivery, and then moving on to discussing the competitive landscape covering one genetic disease in each article.

Let's first define what is a gene? A gene is a sequence of nucleotides in DNA that encodes the synthesis of a gene product, which is usually a protein.

(F8 gene, mutations in the gene cause Hemophilia A)

Usually, the code (in the form of a specific arrangement of nucleotides or base pairs contained in the gene is used to form mRNA (called transcription) which acts as a messenger to take the code to the target organ of the body. The information stored in the mRNA is then used to encode and synthesize the target protein (called translation) which then performs its intended function in the body.

(Steps involved in synthesizing a protein from the code in a gene)

An estimated number of protein-coding genes in the human body is approximately 20,000 to 25,000, which has been revised down from the initial prediction of 100,000 genes. Each gene contains a number of base pairs, the number of which is estimated to range from about 50 million to 300 million in the human body. In general, a gene therapy can be broadly defined as delivering in a new gene into the cells to compensate for a defective gene. In gene therapy, a newly delivered gene can perform different functions; for example, it can either replace the defective gene or it can silence an abnormal gene.

(An example of a gene therapy using an adenoviral vector to deliver the normal gene)

While I will discuss the various steps and delivery systems in gene therapy in detail later, viruses like lentiviruses and adenovirus are most commonly used as vectors in gene therapy. It was as early as 1950s that scientists first discovered that a virus can be used to inject the DNA in the cells of the host. In 1970s, various experiments started to use viruses as delivery systems for genes in the human body. In 1971, Merril, et al conducted a scientific experiment showing that DNA could be injected into the human cells to fix a biological problem in the cells. This group of scientists extracted the cells from patients suffering from a disease called Galactosemia. It is important to note that this first gene therapy experiment involves the manipulation of genes ex vivo, that is in cells growing in a petri dish outside the body in a lab, which is easier to perform than manipulating the genes inside the human body, called in vivo approach. In 1972, a famous article in the prestigious journal Science by authors Friedman and Roblin first proposed that the gene therapy may ameliorate some human genetic diseases in the future. During the 1980s, various scientists like Martin Cline and French Anderson conducted experiments on using viruses as delivery vehicles for DNA in human or mouse cells. The first human trials of gene therapy started in the late 1980s and the results were reported in early 1990s. One of the first reported clinical studies in humans involved ex vivo modification of white blood cells taken from patients with advanced melanoma, using a retroviral vector to insert a gene coding for interleurkin-2 and injecting the genetically altered cells back into the patients. During the 1990s, French Anderson reported a successful clinical trial where a retroviral vector was used to transfer a gene encoding for adenosine deaminase, ADA in children with severe combined immunodeficiency, SCID. During the 1990s, most of the work in gene therapy continued in the therapeutic area of ADA-SCID.

Despite reasonably successful clinical results, the field of gene therapy suffered a serious setback in 1999. Jesse Gelsinger, an 18-year-old patient with a disease called ornithine transcarbamylase, OTC deficiency, which results due to a missing gene coding OTC died 4 days after receiving the gene therapy in a clinical trial conducted by the University of Pennsylvania due to massive immune response resulting in multi-organ failure. As a result, FDA put a suspension on various gene therapy clinical trials.

The field of gene therapy was then suspended for almost a decade. Glybera, a gene therapy was approved in Europe for reading a genetic disease, lipoprotein lipase deficiency in 2012. However, Glybera was a commercial failure after insurers in Europe were reluctant to pay for its expensive $1 million per patient tag. Finally, uniQure (QURE) the company that developed Glybera discontinued it.

Another commercial gene therapy failure was Strimvelis, a stem cell gene therapy to treat ADA-SCID. Despite its price being lower than Glybera ($665,000 per year), the therapy was not commercially successful in Europe and was sold by GlaxoSmithKline (GSK) to Orchard Therapeutics (ORTX) in 2018. In the US, the first approved gene therapy was Kymriah, an autologous CAR-T therapy to treat autologous lymphoblastic leukemia (ALL), which was developed by Novartis (NYSE:NVS).

After Kymriah, another autologous CAR-T therapy, Yeskarta (by Kite Pharmaceuticals) was approved by FDA to treat adult diffuse large B-cell lymphoma. Kite was later acquired by Gilead (NASDAQ:GILD). The first in vivo gene therapy approval in the US was Luxturna, an AAV gene therapy for patients with RPE 65 mutation-associated retinal dystrophy, which was developed by Spark Therapeutics which also was later acquired. Luxturna was another major milestone in the history of gene therapy as it resulted in a miraculous effect of restoring vision to children who were blind since birth. Recently, bluebird bio's (BLUE) gene therapy for transfusion-dependent beta-thalassemia was approved in Europe.

The developmental landscape of gene therapies can be summarized in some excellent figures from the journal Molecular Therapy published by the American Society of Gene and Cell Therapy (ASGCT). A group of researchers reviewed the medical literature and identified 336 gene therapies being developed for 138 different clinical indications covering 165 genetic targets excluding oncology. The researchers found that 74% of these 336 gene therapies were concentrated in five medical specialties, that is, hematology, endocrinology, neurosciences, cardiology, and ophthalmology. When classifying by different disease families, inborn errors of metabolism was the disease category with a majority of ongoing gene therapy trials.

(Landscape of gene therapy programs by organ system and disease area, source: Mol. Therapy)

When looking at specific clinical indications, Duchenne muscular dystrophy (DMD) was the clinical indication with the highest number of gene therapies being developed (15). HIV gene therapies (12 gene therapy programs) and hemophilia (11 gene therapy programs) took the second and third place respectively.

In terms of the number of gene therapy/editing programs being developed by a particular company or organization, Sangamo Therapeutics (SGMO) took the top spot (see the figure below).

(Landscape of gene therapy programs by company/organization, source: Mol. Therapy)

In conclusion, gene therapy has recovered from its earlier setbacks to emerge as one of the most innovative areas in biotechnology. In this first article of the series, I have provided a brief background about gene therapy, its history, and a broad top-down landscape. In the next article in the premium service, I will discuss various delivery systems for gene therapy.

A free two-week trial for the premium Marketplace service is open for another week only. Only 25 more spots left.

Disclosure: I am/we are long BLUE, ORTX, QURE, SGMO, AXGT, CRSP. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Gene Therapy/Editing Series 1: A Brief Introduction To Gene Therapy - Seeking Alpha

Animal Stem Cell Therapy Market Revenue, Opportunity, Segment and Key Trends 2017 2025 – Health News Office

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Animal Stem Cell Therapy Market Revenue, Opportunity, Segment and Key Trends 2017 2025 - Health News Office

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