Stem Cell Clinic

Coronavirus: inside the UAE stem cell centre working to treat Covid-19 – The National

When Dr Yendry Ventura began work to set up the Abu Dhabi Stem Cell Centre in late 2018, there was, he says, nothing else "related to stem cell therapy in the emirate.

Fast forward to today and the situation has changed dramatically. After opening in December last year, the centre has already received international press coverage over to its research into a treatment for Covid-19.

Their groundbreaking work has involved taking stem cells from a patients blood and returning them, via a nebuliser, as a fine mist to the lungs.

There they help regenerate lung cells and improve the body's immune response by preventing an overreaction to the infection that can damage healthy cells.

What characterises the method, says Dr Ventura, is that very little manipulation of the cells is needed for the treatment to be effective.

The future for the stem cells lies in regenerative medicine, in which you can treat almost all the degenerative conditions.

Dr Yendry Ventura

We separate a specific layer of cells from the blood, Dr Ventura told The National. Were the first one to use these cells with this route with this method.

We believe this way the cells can be aimed much better to the affected organs - the upper and lower respiratory tract.

In April, the centres efforts to develop a Covid-19 treatment led to the recovery of all 73 patients the treatment was initially trialled on. A quarter had been in intensive care.

The results appeared so promising that this month the centre secured intellectual property rights to the technique, allowing the treatment to be widely licensed, including to facilities abroad.

The ongoing work exemplifies how the centres specialists have been able to apply their expertise to help in a time of crisis, Dr Ventura said.

But the new research is a departure from the facilitys usual purpose, which involves developing cutting-edge stem cell treatments for conditions such as cancer and heart disease.

Stem cells were first extracted from humans and grown in laboratories less than a quarter of a century ago.

The human body is mostly made of specialised cell types, such as heart muscle cells, kidney cells or nerve cells, all of which have a particular form related to their function.

Stem cells, however, have not yet undergone the process of developing into a specialised cell type, and are able to be manipulated to perform a specific function.

In adults, stem cells are found in tissues including fat and bone marrow, and these can be turned into cell types.

One technique that the Abu Dhabi Stem Cell Centre plans to implement is haematopoietic stem cell transplantation, which involves stem cells being removed from an individual who is due to have cancer treatment.

The cells are then processed in a laboratory and injected into the patient after they have undergone chemotherapy or radiotherapy.

In this way, they can replace stem cells destroyed by the treatment, allowing a patient to tolerate a higher dose of therapy.

Dr Ventura says that similar treatments were applicable to most cancers of the blood as well as cancers that produce solid tumours.

There are many of these therapies still in research stage, but if you conquer this research, you can have a programme in which you can ... treat many kinds of cancers at the same time in one centre, he said.

The reality is that cell therapy is curing cancer We need to improve this therapy and make it available for many other people.

The future for the stem cells lies in regenerative medicine, in which you can treat almost all the degenerative conditions.

You can create in the future, if you have the right technologies, even artificial organs.

Set up with private sector funding in collaboration with the UAE authorities, the Abu Dhabi Stem Cell Centre works closely with experts at Sheikh Khalifa Medical City.

But the institution is keen to forge further partnerships with both public and private sector medical institutions.

Currently, it operates seven days a week and has more than 100 staff, including nurses, technicians and doctors who specialise in immunology, haematology, pathology, orthopaedics, urology and radiology.

In another initiative, the facility has recently begun running Minimal Residual Disease tests, which look at how many malignant cells remain in a patients blood or bone marrow.

These tests are useful for people with a variety of blood cancers, including lymphoma, leukaemia and myeloma. But they require fresh samples from the patient, so the lack of UAE testing facilities has, until now, required patients to travel abroad.

We try to implement the tests here in the Abu Dhabi Stem Cell Centre so that the patient does not need to travel anymore, said Dr Ventura.

Updated: June 16, 2020 02:01 PM

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Safe to Skip Radiotherapy With Negative PET in Hodgkin Lymphoma – Medscape

The majority of patients with early stage unfavorable Hodgkin lymphoma respond well enough to a current standard regimen of 4 cycles of chemotherapy, and can skip the additional radiotherapy that is normally included in the combined modality treatment, say experts reporting the final results from an international phase 3 randomized trial dubbed HD17.

"Most patients with this disease will not need radiotherapy any longer," concluded first author Peter Borchmann, MD, assistant medical director in the Department of Hematology/Oncology at the University Hospital of Cologne, Germany.

Borchmann was speaking online as part of the virtual edition of the European Hematology Association (EHA) 25th Annual Congress 2020.

"Importantly, the mortality of patients with early stage unfavorable Hodgkin lymphoma in the HD17 study did not differ from the normal healthy German population, and this is the first time we have had this finding in one of our studies," he emphasized.

Borchmann added that positron emission tomography (PET) imaging is key in deciding which patients can skip radiation.

"We conclude from the HD17 trial that the combined modality concept can and should be replaced by a PET-guided omission of radiotherapy for patients with newly diagnosed early stage unfavorable Hodgkin lymphoma," he said.

"The vast majority of early stage unfavorable Hodgkin lymphoma patients can be treated with the brief and highly effective 2+2 chemotherapy alone," he added.

Therefore, he continued, "PET-guided 2+2 chemotherapy is the new standard of care for the German Hodgkin study group," which conducted the trial.

The use of both chemotherapy and radiation has long been a standard approach to treatment, and this combined modality treatment is highly effective, Borchmann explained. But it can cause long-term damage, and the known longer-term negative effects of radiotherapy, such as cardiovascular disease and second malignancies, are a particular concern because patients with early stage Hodgkin lymphoma are relatively young, with a median age of around 30 years at disease onset.

An expert approached for comment said that the momentum to skip radiotherapy when possible is an ongoing issue, and importantly, this study adds to those efforts.

"The treatment of Hodgkin lymphoma has moved for many years now to less radiation therapy, and this trend will continue with the results of this study," commented John G. Gribben, MD, director of the Stem Cell Transplantation Program and medical director of the North East London Cancer Research Network Centre at Barts Cancer Center of Excellence and The London School of Medicine, UK.

"We have moved to lower doses and involved fields with the intent of decreasing toxicity, and particularly long-termtoxicity from radiotherapy," he told Medscape Medical News.

For the multicenter, phase 3 HD17 trial, Borchmann and colleagues turned to PET to identify patients who had and had not responded well to chemotherapy (PET-negative and PET-positive) and to determine if those who had responded well could safely avoid radiotherapy without compromising efficacy.

We wanted to determine if we could reduce the treatment intensity by omission of radiotherapy in patients who respond very well to the systemic treatment, so who have a complete metabolic remission after the chemotherapy, Borchmann said.

The 2+2 treatment approach includes 2 cycles of eBEACOPP (bleomycin, etoposide, doxorubicin, cyclophosphamide, vincristine, procarbazine, and prednisone) and 2 subsequent cycles of ABVD (doxorubicin, bleomycin, vinblastine, and dacarbazine).

The trial enrolled 1100 patients with newly diagnosed Hodgkin lymphoma between January 2012 and March 2017. Of these, 979 patients had confirmed PET results, with 651 (66.5%) found to be PET-negative, defined as having a Deauville score (DS) of less than 3 (DS3); 238 (24.3%) were DS3 and 90 (9.2%) were DS4.

The study met its primary endpoint of noninferiority in progression-free survival (PFS) at 5 years, with a PFS of 95.1% in the PET-guided group (n = 447) compared with 97.3% in the standard combined-modality treatment group (n = 428), over a median observation time of 45 months, for a difference of 2.2% (P = .12).

"We found that the survival levels were very high and we can safely conclude the noninferiority of the PET-guided approach in PET-negative patients," Borchmann said.

A further analysis showed that the 597 PET-negative patients who did not receive radiotherapy due to their PET status had 5-year PFS that was noninferior to the combined modality group (95.9% vs 97.7%, respectively; P = .20).

And among 646 patients who received the 2+2 regimen plus radiotherapy, of thoseconfirmed as PET-positive (n = 328), the estimated 5-year PFS was significantly lower (94.2%) compared with those determined to be PET-negative (n = 318; 97.6%; hazard ratio, 3.03).

A cut-off of DS4 for positivity was associated with a stronger effect, with a lower estimated 5-year PFS of 81.6% vs 98.8% for DS3 patients and 97.6% for DS < 3 (P < .0001).

"Only DS4 has a prognostic impact, but not DS3," Borchmann said. "DS4 positivity indicates a relevant risk for treatment failure, however, there are few patients in this risk group (9.2% in this trial)."

The 5-year overall survival rates in an intent-to-treat analysis were 98.8% in the standard combined modality group and 98.4% in the PET-guided group.

With a median observation time of 47 months, there have been 10 fatal events in the trial out of 1100 patients, including two Hodgkin lymphoma-related events and one treatment-related death.

"Overall, Hodgkin lymphoma or treatment-related mortality rates were extremely low," Borchmann said.

The study was funded by Deutsche Krebshilfe. Borchmann and Gribben have reported no relevant financial relationships.

EHA 2020 Congress. Presented June 12, 2020. Abstract S101.

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Tech Start-up Launches Contest, Enabling Scientists to Promote Their Research After the Cancelation of Key Medical Research Meetings this Year -…

TORONTO, June 15, 2020 /PRNewswire/ --TodayBioRender, creator of the first web-basedgraphical tool for scientists to create publication and presentation-ready figures, announced its inauguralGraphical Abstract Contest, to give scientists a way to promote their research and win prize rewards. As key medical research meetings throughout the world have been canceled, postponed or moved to a virtual environment, BioRender is donating its $50K marketing budget to the science community in the form of cash prizes for a first-of-its-kind, worldwide, scientific art contest.

"This contest comes at a time when all conferences and meetings have been cancelled or postponed, so having the opportunity to be involved with these graphical abstracts has been a refreshing way to explore a wide variety of topics, including the pathology and immunology of SARS-CoV-2 infection," said Jarrod Dudakov, Assistant Professor of Immunology at Fred Hutch Cancer Center. "Graphical abstracts are an increasingly vital aspect of science communication and this competition, and BioRender more generally, contribute significantly toward raising the bar in the research community's progress in visual literacy."

At a time when scientists are racing for answers while also trying to help calm fears around the world, being able to network, share research, and present information in a unified, digestible manner is critical. Social distancing has made this a largely remote effort and presents a unique challenge for the science community to share research. To that end, BioRender created a virtual community to enable the continued sharing of research.

"At BioRender, we have always recognized the need for scientific knowledge to be universally understood and communicated through visuals that are clear, standardized, and accessible," said Shiz Aoki, co-founder and CEO of BioRender. "Our mission is to empower the world to communicate science faster through visuals. Graphical abstracts are quickly becoming the ideal way to lower the friction in communicating complex scientific concepts."

There are more than 5,000 entrants in the contest, which include original research from Stanford Medicine, John Hopkins Medicine, Harvard Medical School and Yale School of Medicine. Today BioRender selected People's Choice Award winners for each category of the contest:

Submitted research will be peer-reviewed and each submission will be eligible for cash prizes of up to $6,000. Submissions to theGraphical Abstract Contest contribute to a public gallery that the scientific community can virtually tour to learn about the latest in research while networking and voting for their favorites to win People's Choice Awards. Lead scientific researchers and renowned experts from around the world will come together to judge submissions within their specialization for visual appeal, clarity, accuracy, complexity, and ease of understanding with the best winning Expert's Choice Awards.

BioRender will announce the Expert's Choice Award finalists on June 17 and all winners will be announced on June 29.

About BioRender

At BioRender, we envision a world where science is universally communicated and understood through visuals. We believe science communication should always be fast, repeatable, and standardized. To help make that vision a reality, we are creating the standard visual language of biology and the suite of tools to communicate it.

Led by CEO and Co-founder Shiz Aoki, the Lead Medical Illustrator for National Geographic for a decade, our team is composed of environmentally and socially conscious engineers, science illustrators, designers, and entrepreneurs who are backed by an incredible group of investors, including Y Combinator.

Recently heralded in Nature as 'a staple of biomedical research drawings', our first product is a web-based graphical tool that enables scientists to create publication and presentation-ready figures up to 50 times faster than traditional tools. With a comprehensive library of 20,000+ icons and intuitive drag-and-drop functionality, this tool enables scientists across 30+ fields of life science to create professional illustrations that more effectively communicates their research.

Media Contact: Interdependence PR for BioRender Samantha Harber; [emailprotected] (406) 272-2627

SOURCE BioRender

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Plinabulin Combo May Be Superior Therapy for Prevention of Chemotherapy-Induced Neutropenia – Targeted Oncology

Treatment with plinabulin in combination with pegfilgrastim (Neulasta) achieved the primary end point and several key secondary end points in the phase 3 PROTECTIVE (Study 106) clinical trial, which evaluated the combination compared with pegfilgrastim alone for superiority in chemotherapy-induced neutropenia prevention, according to the interim results announced by BeyondSpring, developer of the drug.

These results demonstrate significant enhancement to granulocyte colony-stimulating factors (G-CSFs) with the combination compared with pegfilgrastim alone for the prevention of grade 4 or severe neutropenia, supporting the potential for superiority with the combination compared with pegfilgrastim alone.

These interim results from the PROTECTIVE-2 Phase 3 study, which compares the Plinabulin-Neulasta combination to Neulasta alone, have the potential to be clinically meaningful for cancer patients receiving chemotherapy, stated Douglas W. Blayney, MD, professor of Medicine at Stanford Medical School and global principal investigator of Plinabulins chemotherapy-induced neutropenia studies. Since most infections, hospitalizations, and other complications of chemotherapy-induced neutropenia occur in the first week after chemotherapy, it is particularly gratifying to see the combinations clinical benefit demonstrated.

The primary end point was the rare of severe neutropenia prevention in cycle 1 (P <.01). The key secondary end points included duration of severe neutropenia in cycle 1 (P <.05) and duration of severe neutropenia in the first 8 days of cycle 1 (P <.05). The achievement of these end points demonstrates the agents ability to provide early protection against severe neutropenia induced by chemotherapy.

The interim analysis of the double-blind, active-controlled, global PROTECTIVE-2 study was pre-specified for the first 120 patients accrued. The study also has procedures in place for the prevention of potential bias after the planned interim analysis, and BeyondSpring opted to be informed by independent statisticians on whether the pre-specified P values were met rather than the exact P values.

The study was designed to assess both the safety and efficacy of plinabulin in patients with breast cancer treated with docetaxel, doxorubicin, and cyclophosphamide (TAC) in a 21-day cycle. Plinabulin was administered in a 40 mg dose on day 1 with a 6 mg dose of pegfilgrastim on day 2, while pegfilgrastim was administered at the same 6 mg dose on day 2 in the control arm. TAC is considered a high-risk chemotherapy regimen associated with neutropenia.

PROTECTIVE-2 is a superiority study for chemotherapy-induced neutropenia efficacy compared with pegfilgrastim alone and is currently enrolling patients. Grade 4 neutropenia rate for TAC and pegfilgrastim is observed in 83% to 93% of patients, which represents a severe unmet medical need.

The study drug and G-CSF have complementary mechanisms of action for preventing chemotherapy-induced neutropenia. Plinabulin is a first-in-class differentiated immune and stem cell modulator, which is currently in late-stage clinical development as a potential treatment for increasing overall survival in patients with cancer in addition to its ability to alleviate chemotherapy-induced neutropenia.

Plinabulin is also under evaluation in a phase 3 study as a direct anticancer drug as treatment of patients with nonsmall cell lung cancer and 2 phase 3 studies for the prevention of chemotherapy-induced neutropenia. The data for this agent in preventing chemotherapy-induced neutropenia highlights its ability to boost the number of hematopoietic stem or progenitor cells (HSPcs), as well as lineage-/cKIT+/Sca1+ cells in mice. Plinabulins effects on HSPcs may explain its ability to treat chemotherapy-induced neutropenia as well as its ability to reduce chemotherapy-induced thrombocytopenia and increase circulating CD34-positive cells.

To be included in the study, patients should be candidates for TAC in either the adjuvant or neoadjuvant setting, have early-stage breast cancer, and have received no prior chemotherapy. Patients must also have an ECOG performance status of 0 or 1 and a life expectancy of at least 3 months. Patients cannot be included in the study if they have a history of myelogenous leukemia, myelodysplastic syndrome, or sickle cell disease; use of CYP3A4, CYP2D6 or P-glycoprotein inhibitors and inducers within 14 days prior to receiving the study drug; or have received an investigational agent or tumor vaccine within 2 weeks, any concurrent anticancer therapies, or a bone marrow or stem cell transplantation.

These results could help to confirm the patient benefit of Plinabulins different mechanism of action from the G-CSF-based agents, such as Neulasta, Blayney stated. Plinabulin appears to have chemotherapy-induced neutropenia protection in Week 1, and G-CSFs have protection in Week 2 of chemotherapy cycles. The combination should logically provide significantly better protection than Neulasta alone as shown in the interim readout. We are well on our way to confirming that the combination offers protection throughout the chemotherapy cycle, which is an unmet medical need.

Reference

BeyondSpring Announces Positive Topline Interim Results from PROTECTIVE-2 (Study 106) Phase 3 Trial Evaluating Superiority of Plinabulin in Combination with Neulasta for Chemotherapy-Induced Neutropenia Prevention. NewsRelease. BeyondSpring. June 15, 2020. Accessed June 15, 2020. https://bit.ly/2YDJbiQ

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Plinabulin Combo May Be Superior Therapy for Prevention of Chemotherapy-Induced Neutropenia - Targeted Oncology

Children’s Hospital Los Angeles Ranked No. 1 Children’s Hospital in the Western U.S., No. 5 Nationally for Second Straight Year – Newswise

Newswise LOS ANGELES (June 16, 2020) Children's Hospital Los Angeles (CHLA) ranks again among the nations premier destinations for pediatric care, according to the U.S. News & World Report Best Children's Hospitals annual list released today.

CHLA not only retained its national No. 5 ranking in U.S. News Honor Roll of Best Childrens Hospitalswhich recognizes institutions with the most outstanding pediatric clinical careit continued its four-year streak of being the highest-scoring childrens hospital in the entire Western United States.

To make U.S. News & World Reports prestigious Best Childrens Hospitals Honor Roll, one must demonstrate the strongest achievements in clinical excellence, with a matchless team of expert, compassionate specialists committed to research and education as well as protocols that drive safety and quality and consistently lead to the best health outcomes for patients, says CHLA President and Chief Executive Officer Paul S. Viviano. "This honor affirms the work of every CHLA team member and our belief that when parents choose Childrens Hospital Los Angeles, they are choosing the best care for kids."

Every year, U.S. News scores nearly every major hospital and health system in the country and ranks them according to performance benchmarks, peer review, certifications, and other data provided by the hospital and third-party measurements of excellence. Children's hospitals are ranked separately from other facilities due to the specialized expertise, equipment and facilities required to care for infants, children and youth.

This year, U.S. News surveyed 118 pediatric medical centers, including hospitals that are freestanding or part of a larger institution. CHLA improved its ranking over last year in seven of the 10 pediatric specialty categories the survey considers, including a number two ranking for Neonatal Care. In all, the hospital earned top-10 recognition in seven of those categories:

CHLA has an organization-wide commitment to providing our patients the care they need no matter their circumstances, says CHLA Chief Medical Officer James Stein, M.D., MSc. "Our clinical teams often treat the most acute cases that are outside the scope or expertise of other childrens hospitals in California, and being named a Top-5 childrens hospital in the U.S. is a testament to the clinicians and staff who work every day to make sure each child receives the best care and experience possible.

Founded in 1901, Children's Hospital Los Angeles is a pediatric academic medical center built around its mission of creating hope and building healthier futures for children. Renowned for its world-class clinical care, leading-edge research and one of the largest and most successful pediatric training programs in the countryall while being the pediatric safety net hospital for the entire regionCHLA now sees more than 600,000 patient visits annually between its main hospital and five neighborhood care clinics.

CHLA physicians, nurses and clinical staff provide compassionate and lifesaving pediatric care for patients ranging from infants to young adults, hailing from all 50 states and more than 75 countries. Clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC. Many of the hospital's achievements in care are made possible through a cohesive relationship between clinical experts at the bedside and the basic, translational, and clinical research conducted in The Saban Research Institute of CHLA.

In the past year, CHLA has had several notable achievements, including:

U.S. News and World Report works with research firm RTI International to develop its annual Best Children's Hospitals list, a collaboration between hospitals and the magazine to benchmark the performance of childrens hospitals for the benefit of parents and their children. The survey evaluates hundreds of data points, including patient survival and surgical complication rates; staffing, technology and special services; infection prevention and delivery of care; reputation among peer physicians nationwide (i.e. Where would the best pediatric specialists send their kids?); how involved parents are in their childrens care; and many other measurements of excellence.

U.S. News Media Group, the parent of U.S. News & World Report, announced the 2020-21 hospital rankings online at 12:01 a.m. EST on Tuesday, June 16. For additional information, please visit the Best Childrens Hospitals Honor Roll and specialty rankings page at usnews.com/childrenshospitals.

About Children's Hospital Los Angeles Founded in 1901,Children's Hospital Los Angelesis ranked the topchildrens hospital in California andfifth in the nation for clinical excellence with its selection to the prestigious U.S. News & World Report Honor Roll of childrens hospitals. Clinical care is led by physicians who are faculty members of the Keck School of Medicine of USC through an affiliation dating from 1932.The hospitalalso leads thelargest pediatric residency training program at a freestanding childrens hospital of its kind in the western United States.TheSaban Research Instituteof Childrens Hospital Los Angeles encompasses basic, translational and clinical research conducted at CHLA. The hospitals Global Health Program facilitates services for international patients from more than 75 countries. To learn more, follow us on Facebook, Instagram, LinkedIn and Twitter, and visit our blog for families (CHLA.org/blog) andour research blog (ResearCHLABlog.org).

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Children's Hospital Los Angeles Ranked No. 1 Children's Hospital in the Western U.S., No. 5 Nationally for Second Straight Year - Newswise

Targeted policy support for emerging biomedical innovations – Open Access Government

Like many states, the UK government has committed to supporting disruptive innovations.1 These are considered to hold greater potential for economic growth and development than incremental advances in established technologies. Within this broad strategy the bioeconomy, the area of industrial activity based on commercialising life sciences research is given a particular importance. The bioeconomy includes sectors like biofuels, agricultural biotechnology, and medical biotechnology.2 In the latter case, advances in medical biotechnologies hold promise for treating, and even curing, serious and chronic diseases as well as driving growth and prosperity. Regenerative medicine (RM), the biotechnology-based use of cells, tissues, and genes as medicinal products, is certainly disruptive in that they differ in important ways from traditional pharmaceuticals and medical devices.3

The UK has taken a number of policy measures to support the development of the RM industry. The Regenerative Medicine Platform funding schemes promote and co-ordinate academic translational research. The Catapult centres, including the Cell and Gene Therapy Catapult, the Medicines Discovery Catapult and the High Value Manufacturing Catapult, provide advice, facilities and infrastructure to support businesses, especially Small and Medium-sized Enterprises (SMEs); with potential to contribute to the RM value chain. The Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Office offers a RM advice service to help academic and commercial developers navigate the complex regulatory framework for biological therapies, while the recent Accelerated Access Review proposed a raft of measures to speed up the regulatory timeline for transformative new therapies more generally.4

However, it does not necessarily follow that all parts of the biomedical sector will be equally disrupted by any given RM technology, nor that all RM technologies will be disruptive in exactly the same way.5 The ESRC-funded Biomodifying Technologies project6 analysed three case studies of biotechnologies with disruptive potential: gene-editing which allows faster, more accurate genetic modification, induced pluripotent stem cell (iPSC) technology that allows an ordinary skin or blood cell to be turned into a stem cell capable of producing any tissue type in the human body, and 3D bioprinting which can produce three-dimensional structures made from living tissues.

Gene editing and iPSC are advances on earlier generations of genetic engineering and stem cell technologies. They align reasonably well with the existing skill sets, goals, equipment, and techniques of researchers working in both academic and commercial settings. They are not especially disruptive at the level of basic research. Bioprinting requires skills, tools and techniques from engineering, materials chemistry, computer-aided design, biology, and medicine. This has necessitated greater disruption in the form of organisational change, to create new research groups and foster collaborative learning across disciplines.

For all three technologies, there are also well-established pathways to extract near-term value from basic research: peer-reviewed publications, patent applications, and the market for reagents, tools, and equipment. Each case demonstrates clear growth in the number of papers, patents, and reagent/equipment sales, although the rate of acceleration is greatest for CRISPR-based gene editing and slowest for bioprinting.

The pathways to realise longer-term, clinical, and economic value are less well established for RM. The healthcare sector is seen as particularly resistant to disruptive innovations, due to the lengthy regulatory process and powerful incumbent firms, which have historically been wary of investing in RM.7 The process of scaling laboratory protocols for cell or gene-based therapies into industrial procedures, taking products through clinical trials to establish safety and efficacy, and securing reimbursement, is every bit as experimental and involves as much learning by trial and error as exploratory laboratory research, but with much higher financial stakes. Interest from incumbents appears to be growing, as recent years have seen an increase in the number of cell or gene-based therapies reaching the market. However, there is no off the shelf manufacturing solution, as different RM products have different attributes: in the industry there is a popular idiom the product is the process. This means that the acceleration seen at the basic R&D stage does not unproblematically translate into speedy translation further down the pathway.

Rather, initial clinical applications of gene editing, iPSC and bioprinting are targeted at a more limited range of niche applications. The niches for each technology are shaped by a number of critical factors. Smaller tissues, such as the eye require fewer replacement cells or lower titres of gene editing vector, which are more manageable with current manufacturing capacity. The challenges of manufacturing at scale, combined with high anticipated costs, combine to make narrowly defined subsets of disease categories, with high unmet need, a preferred route for commercial development, especially where there is potential for a disruptive new product to demonstrate significant Quality of Life gains over the current standard of care.

Indications that draw on procedures, standards and requirements established for previous therapies are seen as less risky and thus promising clinical targets. Gene editing to treat thalassemia and other blood disorders builds on decades of clinical expertise with the bodys haematopoietic (blood-forming) system, gained by treating leukaemia patients. Even treatments that were not ultimately successful such as foetal stem cell transplants for Parkinsons disease (PD) can provide expertise with clinical trials and regulation to support a next-generation iPSC-based cell therapy for PD.

While the government has rightly been wary of picking winners, as particular niches for early clinical adoption of biomodifying technologies become apparent they may require specific, targeted support, to complement the broader support for the field already provided by polices described above. Innovations in related fields such as biomaterials and automation, potentially supported by the High Value Manufacturing Catapult, are likely to improve manufacturing capacity and speed over time. These innovations may be relatively incremental in the manufacturing phase but could have disruptive effects further down the value chain at the clinical delivery phase, as greater supply makes biomodifying RM therapies accessible to less tightly defined patient cohorts. The next policy challenge will be to provide targeted support for clinical delivery whilst avoiding lock-in to infrastructure or procedures that would inhibit the evolution of the field over time.

The research underpinning this piece was supported by the Economic and Social Research Council grant number ES/P002943/1 and the Leverhulme Trust grant number RPG-2017-330

References

1 Department for Business, Industry and Industrial strategy (2017) Industrial Strategy: building a Britain fit for the future. https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/730048/industrial-strategy-white-paper-web-ready-a4-version.pdf

2 Department for Business, Industry and Industrial strategy (2018) Bioeconomy strategy: 2018 to 2030. https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/761856/181205_BEIS_Growing_the_Bioeconomy__Web_SP_.pdf

3 Open Access Government (2019) The promises and challenges of biomodifying technologies for the UK https://www.openaccessgovernment.org/biomodifying-technologies/68041/

4 Accelerated Access Review (AAR). (2016). Final Report: Review of Innovative Medicines and Medical Technologies. London: The Crown.

5 Joyce Tait & David Wield (2019) Policy support for disruptive innovation in the life sciences, Technology Analysis & Strategic Management, DOI: 10.1080/09537325.2019.1631449

6 Open Access Government (2019) The promises and challenges of biomodifying technologies for the UK https://www.openaccessgovernment.org/biomodifying-technologies/68041/

7 Joyce Tait & David Wield (2019) Policy support for disruptive innovation in the life sciences, Technology Analysis & Strategic Management, DOI: 10.1080/09537325.2019.1631449

Please note: This is a commercial profile

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Targeted policy support for emerging biomedical innovations - Open Access Government

Alzheimer’s: Could Fat Obtained From Liposuction Treat the Disease? – Gilmore Health News

The start-up Cell-Easy and the University Hospital of Toulouse have joined forces to carry out a trial to see if stem cells obtained from liposuction could help treat Alzheimers.

Liposuction Image Courtesy of Michael S. Schwartz, M.D

Alzheimers disease is the leading cause of dementia in the world. It accounts for 60 to 70% of cases. By 2015, 9.9 million people will have been diagnosed, a new case every three seconds according to the WHO. The disease most often manifests itself through memory disorders, and then other brain functions are affected. Gradually, daily tasks become more and more difficult and it becomes almost impossible for patients to adapt to new situations. The disease is currently incurable and, given the number of people affected, researchers are eagerly trying to at least slow down the progression of the disease. The treatment of stem cells to replace damaged cells with healthy ones is one of the therapeutic approaches that is increasingly being used. In France, the university hospital of Toulouse (CHU de Toulouse) declared on June 14 to 20 minutes a French journal that they will soon launch a study based on stem cells obtained by plastic surgeons during liposuction.

Read Also: Exifone Reverses DNA Damage in Mice Suffering From Dementia and Alzheimers in MIT Study

The start-up company Cell-Easy, based in Toulouse and dedicated to the large-scale production of stem cells, is the only structure of its kind in France. It recently obtained an authorization to carry out clinical trials with human stem cells obtained from fat tissue, which is available in almost unlimited quantities in the human body.

The first challenge for Cell-Easy was to reduce the cost of fat stem cell production by a factor of 10 in order to make this therapy available to as many people as possible. We have succeeded in this. Today we can open our pharmaceutical facility. Actually we already started equipping the laboratory. Production will start in about six months. The production capacity of the plant will reach 100,000 doses per year, explains Pierre Monsan, director of Cell-Easy and founder of the French biotechnology association in La Tribune de Toulouse.

We take this waste and turn it into medicine. Although stem cells often come from umbilical cords or painful lumbar punctures, our technology allows us to multiply them, he explained to 20 minutes a French journal.

Read Also: Alzheimers Breakthrough: An Antibody That Can Recognize the Disease Much Earlier Is Now Available

For this reason, the start-up has just signed a cooperation agreement with the University Hospital of Toulouse for a clinical study on Alzheimers disease, which is scheduled to start in early 2021. The researchers will accompany nine patients aged between 50 and 85 years who have just been diagnosed with the disease in the university hospitals of Toulouse and Montpellier.

Although the disease is characterized by chronic inflammation of the brain, which causes protein deposits, the scientists will test the anti-inflammatory effect of stem cells and their ability to slow down the progression of the disease, he continues. Therefore, the participants will be injected with stem cells via the bloodstream. Depending on the initial results, the cohort may then grow to about 50 people.

Read Also: Speaking Multiple Languages Can Delay the Onset of Alzheimers

Medical research into the treatment of Alzheimers disease is making great progress. Recently, scientists have discovered a promising clue to establish a biomarker for the disease in the retina. Indeed, the top layer of neurons in the retina of a mouse model of Alzheimers disease showed a change in its structural texture. Combined with data on changes in the thickness of this layer, the new measurement could be a more readily available biomarker for Alzheimers disease. Early detection of the disease could significantly improve the quality of life of millions of people worldwide.

https://www.20minutes.fr/sante/2799371-20200614-toulouse-quand-liposuccions-debouchent-espoir-traitement-maladie-alzheimer

https://toulouse.latribune.fr/innovation/2020-06-11/cell-easy-devient-le-premier-etablissement-a-produire-des-cellules-souches-en-france-850013.html

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