Using big data approaches to develop cell therapies – Drug Target Review

An area where stem cell biology and medicine are combining effectively is the establishment of new cell therapies. However, current therapies are limited to a narrow set of cell types that can be isolated or created and expanded in vitro. Dr Owen Rackham discusses how utilising computational approaches will further enhance applications of stem-cell-derived therapies in the future.

For decades (or perhaps centuries) the approach in cell biology has remained relatively unchanged. We isolate cells and with our confined knowledge of their endogenous conditions, begin to experiment until we can sustain them in vitro. Once established, we can conduct further investigation to assess a cells response to different conditions, changes over time or response to manipulation. This is especially true of stem cell biology, established from tireless efforts to incrementally improve culture conditions or differentiation protocols based on fragmented knowledge of developmental processes. Despite this, the promise of stem-cell therapies is already being realised in the clinic, but the breadth of cell types being used is still relatively narrow. Recent technological advances in the field have been focused on the safe and scalable manufacture of therapies. While these are revolutionary breakthroughs, the applications are largely limited to T cells, haematopoietic- and pluripotent-stem cells (HSCs and PSCs), a small fraction in the grand heterogeneity of cell types. Consequently, the lack of cell source diversity prevents cell therapy from fulfilling its clinical potential, pointing to the need for new means to isolate or generate source cells.

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Miracle cures and fast-track approval threatens medical advances, say scientists – The University of Manchester

Prof. Volker ter Meulen, Co-chair of the EASAC-FEAM Working Group and Past President of EASAC, said: Stem cell and gene-based therapies hold great medical promises. But we are alarmed over a trend to lower requirements of evidence. Also, we see an increasing problem of commercial clinics offering unregulated products and services

The Academies say the idea of regenerative medicine is to tackle diseases which up to now are incurable. According to the report, cosmetic applications, for example, are inappropriate for the time being.

So far, regenerative medicine has proven itself only in few specific clinical indications, for example for skin disorders. Yet, we see an increasing number of unregulated clinics promising a wide range of benefits on the basis of poorly characterised medicinal products with little evidence of effectiveness. They usually advertise their services via the internet with the primary intention of financial profit, explains EASAC Biosciences Programme Director Dr. Robin Fears.

The scientists urge the EU to resist the pressure and put patients first. When countries lower regulatory standards in their eagerness to support national economic interests, it is even more important for the EU as a major global player to defend the principles of international cooperation in health regulation, says Prof. George Griffin, Co-chair of the Working Group and President of FEAM.

We all want cures to be available in the shortest time frame possible. But our analysis and recommendations aim at ensuring that regulatory procedures are robust, transparent and evidence-based, concludes Cossu. Scientific research and proof are more important than ever. The EU and national regulators should be wary of not undermining public trust in science.

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Miracle cures and fast-track approval threatens medical advances, say scientists - The University of Manchester

MSC Therapy for Acute Respiratory Distress Syndrome; It’s Time to Accelerate Clinical Trials for COVID-19 Patients in Need – PRNewswire

WASHINGTON, June 3, 2020 /PRNewswire/ --A new systematic review and meta-analysis of clinical studies using mesenchymal stromal cells (MSCs) led by a team at the Mayo Clinic, and including researchers from Emory, Duke, Case-Western, and the University of Miami, shows a trend toward improved outcomes and reduced mortality for patients with acute respiratory distress syndrome (ARDS), a major complication for patients with COVID-19. This studyand several othersalso have shown that MSCs are safe for patients.

Based on these findings, the authors call for the rapid commencement of large-scale, confirmatory clinical trials to build on the existing evidence base, which shows a trend toward improved pulmonary function and reduced severe lung inflammation for patients with ARDS, paving the way toward another treatment option for seriously ill patients with COVID-19.

To date, nearly two million Americans have tested positive for COVID-19 and more than 100,000 Americans have died. In its most severe form, COVID-19 leads to ARDSa life-threatening lung injury that allows fluid to leak into the lungs and makes it difficult for patients to breathe. More than 40 percent of individuals hospitalized for severe and critical COVID-19 develop ARDS, and 22 percent to 62 percent of those who are diagnosed and become critically ill, die from the disease. There is no effective treatment for ARDS today; MSCs potentially offer a unique therapeutic option to help patients in need.

"The analysis shows a positive trend in outcomes when treating ARDS patients with MSC therapy and represents the potential to save thousands of patients with COVID-19 induced ARDS," said Wenchun Qu, MD, PhD of the Mayo Clinic and first author of the paper. "The potential benefitcombined with the demonstrated safety of these therapiessupports the need for rapid commencement of more clinical trials."

"Acute respiratory distress syndrome is a rapidly progressive disease that can occur in critically ill patients," said Anthony Atala, MD, Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. "Having additional potential therapies, such as MSCs, could be highly beneficial to patients with COVID-19."

To date, the FDA has approved more than a dozen investigational new drug applications for the use of MSCs for COVID-19-related conditions. The National Institutes of Health (NIH) has also supported the use of MSCs and other regenerative cell therapies to help patients with other conditions. The bipartisan 21st Century Cures Act provided $30 million in funding to the NIH over three years for clinical research for such therapies. However, these limited investments expire in fiscal year 2020.

The Alliance for Cell Therapy Now and the Regenerative Medicine Foundation support the recommendation of the authors, who urge funding for larger studies that build on the results to date. Collaboration and funding are also needed to collect and analyze the evidence from multiple ongoing and new studies, to better evaluate outcomes and potential benefits of MSC therapy for COVID-19 patients in need. A portion of the more than $10 billion in funding directed by Congress to the Biomedical Advanced Research and Development Authority (BARDA) and the NIH for COVID-19 should be used to support these goals.

About the Alliance for Cell Therapy Now

Alliance for Cell Therapy Now (ACT Now) is an independent, non-profit organization devoted to advancing the availability of and access to safe and effective cell therapies for patients in need. ACT Now convenes experts and stakeholders to develop and advance sound policies that will improve the development, manufacturing, delivery, and improvement of regenerative cell therapies. See http://allianceforcelltherapynow.org/

About the Regenerative Medicine Foundation

The non-profit Regenerative Medicine Foundation (RMF) fosters strategic collaborations to accelerate the development of regenerative medicine to improve health and deliver cures. RMF pursues its mission by producing its flagship World Stem Cell Summit, honoring leaders through the Stem Cell and Regenerative Medicine Action Awards, and promoting educational initiatives. STEM CELLS Translational Medicine is RMF's official journal partner. See https://www.regmedfoundation.org/

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Groundbreaking Gene Therapies for Hereditary Diseases / Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Krner…

The current coronavirus pandemic clearly illustrates how dangerous viral infections can become for us. Independent of the present situation, there are people whose bodies are defenseless against infections because their immune systems are unable to combat them - they suffer from immunodeficiency diseases such as ADA-SCID (adenosine deaminase severe combined immunodeficiency) or Wiskott-Aldrich syndrome. Prof. Dr. Alessandro Aiuti, a physician and research scientist based in Milan who works at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) and at the Vita Salute San Raffaele University, is now being honored with the Else Krner Fresenius Prize for Medical Research 2020 for his groundbreaking successes in the development of gene therapies. The award is coupled to 2.5 million euros in prize money.

In the case of the rare immune disorder ADA-SCID, which exclusively afflicts young children and occurs about 15 times a year in Europe, a defective ADA gene within the genome disrupts lymphocyte development, leaving the young patient's body defenseless against infections. "Without effective therapy, the children rarely survive for more than 2 years because any infection can become perilous for them," Aiuti explains. Standard for this therapy is a bone marrow transplantation from a fully matched sibling. However, a suitable donor is available only for a minority of patients. "Meanwhile children with such a condition benefit from the advances we have made in the field of gene therapy. So far we have treated 36 children from 19 countries using the therapy we developed. In more than 80 percent of the cases, the treatment has had such an impact that no enzyme replacement therapy or transplantation is needed. This achievement has been made possible by the extraordinary effort and dedication of SR-Tiget researchers and clinical team throughout 25 years," Aiuti adds. All of the patients are still alive.

For these successes and his other work in the field of gene therapy, Alessandro Aiuti has now been honored with the Else Krner Fresenius Prize for Medical Research 2020 awarded by the Else Krner-Fresenius-Stiftung (EKFS) foundation. At 2.5 million euros, this award is one of the highest endowed prizes for medical research in the world. "Still young by comparison, this year the prize is being awarded for the third time. It honors research scientists for pioneering contributions in the areas of biomedical science. A major percentage of the prize money flows into the prizewinner's research and is supposed to contribute toward achieving further groundbreaking findings and medical breakthroughs in the future as well," emphasizes Prof. Dr. Michael Madeja, scientific director and member of the management board at EKFS.

The decision regarding the prize recipient was made by a ten-member international jury composed of renowned research scientists in the fields of genome editing and gene therapy along with delegates from the Scientific Commission at EKFS. Prof. Dr. Hildegard Bning, chairwoman of the jury and president of the European Society for Gene and Cell Therapy (ESGCT), substantiates the jury's decision: "Alessandro Aiuti is a truly outstanding physician and scientist. His work has decisively contributed to the development and successful treatment of rare, genetically caused disorders such as SCID. Thanks not least of all to the contributions he has made, even patients with other inheritable illnesses can presumably be treated successfully in the future."

After successful clinical trials, the gene therapy developed for ADA-SCID patients was approved as a pharmaceutical remedy in Europe. It is considered to be one of the key findings in the development of gene therapies worldwide. With this treatment certain blood stem cells (CD34+) are taken from the patient, then the cell DNA is modified. The cells are treated outside the body using a viral vector to accomplish this. The correct version of the gene for the ADA enzyme is introduced into the genome of the cells that were collected. The genetically modified cells are returned to the patient's bloodstream via intravenous infusion. A portion of the modified cells subsequently establish themselves in bone marrow again. The patient now has blood stem cells that function properly and produce lymphocytes to defend against infections - presumably on a life-long basis.

Alessandro Aiuti wants to utilize the prize money from EKFS to set the success story forth, to optimize the therapies further and map out the healing mechanisms involved in a better fashion. The scientist sees another major challenge in conveying the acquired knowledge beyond the successful gene therapies from Milan to as many other genetic disorders as possible. Alongside the therapy for ADA-SCID, the San Raffaele Telethon Institute for Gene Therapy has also developed gene therapies for four more hereditary diseases, among them the Wiskott-Aldrich syndrome and metachromatic leukodystrophy (MLD). To this day a total of more than 100 patients from 35 different countries have been treated.

Biography of Alessandro Aiuti

Alessandro Aiuti was born in Rome in 1966 and studied medicine there at Sapienza University. Following a stay at Harvard Medical School in Boston, Massachusetts in the USA, he received his doctorate in Human Biology in 1996 from Sapienza University. Since 1997 he has been active at the San Raffaele Scientific Institute in Milan, where he meanwhile also teaches as a professor at the Vita Salute San Raffaele University. He is furthermore Deputy Director of Clinical Research at the San Raffaele Telethon Institute for Gene Therapy and Head of the Pediatric Immunohematology Unit at San Raffaele Hospital.

Aiuti is the author of numerous and highly acclaimed publications. Over the course of his career he has received a number of prizes from national and international institutions. Aiuti is a member of the board of the European Society of Gene and Cell Therapy, and a member of the EMA Committee for Advanced Therapies since 2019.

The Else Krner Fresenius Prize for Medical Research

The international Else Krner Fresenius Prize for Medical Research came into existence in 2013 on the occasion of the 25th anniversary of Else Krner's death and is awarded in alternating fields of biomedical science. Endowed with 2.5 million euros, the prize is one of the most highly endowed medical research awards in the world. It honors and supports research scientists who have made significant scientific contributions in their fields and whose work can be expected to yield groundbreaking findings and medical breakthroughs in the future as well.

The Else Krner-Fresenius-Stiftung (EKFS) foundation - Advancing research. Helping people.

The Else Krner-Fresenius-Stiftung, a non-profit foundation, is dedicated to the funding of medical research and supports medical/humanitarian projects. The foundation was established in 1983 by entrepreneur Else Krner and appointed as her sole heir. EKFS receives virtually all of its income in dividends from the Fresenius healthcare group, in which the foundation is the majority shareholder. To date, the foundation has funded around 2,000 projects. With a current annual funding volume around 60 million euros the EKFS is one of the largest foundations for medicine in Germany. More information:www.ekfs.de.

The San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute has given a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than EUR 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit:www.telethon.it

Issued by news aktuell/ots on behalf of Else Krner-Fresenius-Stiftung

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Huge Growth Expected in Global Single-Cell Analysis Market in Future – Cole of Duty

The single-cell analysis market generated revenue of $1,688.9 million in 2015, and it is projected to grow at an 18.1% CAGR during the forecast period. The market is expected to be driven by the growing geriatric population, rising chronic disease prevalence, and increasing focus on personalized medicine. The study of individual cells isolated from multi-cellular organisms is referred to as single-cell analysis, which aims to study the heterogenous cell population. These studies are conducted in stem cell, neurological, cancer, and immunological research to help diagnose and treat various diseases.

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The single-cell analysis market is experiencing growth due to the rising healthcare expenditure across the globe. With the increasing prevalence of various diseases, governments of many countries are focusing more on improving the healthcare infrastructure. This fact has been agreed on by the World Health Organization, which reported that the total healthcare expenditure has witnessed an increase worldwide, thereby resulting in improvements in healthcare facilities. This is enabling people from different strata of the society in accessing healthcare facilities and tests involving single-cell analysis.

The geography segment of the single-cell analysis market is classified into Europe, Latin America, Asia, North America, and Rest of the World, among which the largest share in 2015 was accumulated by the North American region. Technical advancements in single-cell analysis products and growing geriatric population were instrumental in the North American markets growth. However, the fastest growth during the forecast period is expected to be witnessed by the Asian market, due to its large pool of patients and increasing awareness about new diagnostic techniques, such as single-cell analysis.

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GLOBAL SINGLE-CELL ANALYSIS MARKET

GLOBAL SINGLE-CELL ANALYSIS MARKET, BY TYPE OF PRODUCT

GLOBAL SINGLE-CELL ANALYSIS MARKET, BY TYPE OF CELL

GLOBAL SINGLE-CELL ANALYSIS MARKET, BY TYPE OF TECHNIQUE

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Huge Growth Expected in Global Single-Cell Analysis Market in Future - Cole of Duty

Follica Announces Positive Feedback From End of Phase 2 Meeting With FDA for Its Lead Program to Treat Male Androgenetic Alopecia – BioSpace

BOSTON--(BUSINESS WIRE)-- Follica, Inc. (Follica), a biotechnology company developing a regenerative platform designed to treat androgenetic alopecia, epithelial aging and other related conditions, today announced positive feedback from a meeting with the U.S. Food and Drug Administration (FDA) as the company prepares to advance its lead program into Phase 3 development following a successful safety and efficacy optimization study for the treatment of hair loss in male androgenetic alopecia announced in December 2019.

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Follicas approach, which is designed to stimulate the growth of new follicles and new hair, is being developed as a potential new option for the millions of people seeking treatments to grow new hair. (Graphic: Business Wire)

Follica plans to launch its Phase 3 program this year. Overall, approximately 280 patients will be enrolled, with efficacy assessed against two co-primary endpoints: visible (non-vellus) hair count and patient-reported outcomes on a pre-established scale. The randomized, controlled, double-blinded studies will be conducted in multiple centers across the U.S. A maximal use study to further understand the pharmacokinetics of the treatment will be conducted in parallel. The trial design is consistent with feedback from the FDA during the End of Phase 2 meeting.

In the U.S. alone, 47 million men are affected by progressive hair loss caused by androgenetic alopecia, a condition that is largely unresolved today, leaving many dissatisfied with the current available treatments and looking for a new alternative. Our recent safety and optimization study points to a new level of effect, enabled by our proprietary approach, which stimulates the growth of new follicles and new hair, said Jason Bhardwaj, chief executive officer of Follica. Were grateful to the FDA for their guidance as we prepare for our pivotal program, and we look forward to advancing the development of our treatment regimen, which has demonstrated strong potential to address the current need for those who seek treatment for androgenetic alopecia.

Follicas approach is based on generating an embryonic window in adult scalp cells via a series of short office-based treatments with its proprietary Hair Follicle Neogenesis (HFN) device. The scalp treatments, which last just a few minutes, stimulate stem cells and enable the growth of new hair follicles. A topical drug is then applied to enhance efficacy by growing and thickening new hair follicles and hair on the scalp.

Follica reported topline results from its safety and optimization study in December 2019. That trial was designed to select the optimal treatment regimen using Follicas proprietary HFN device in combination with a topical drug and successfully met its primary endpoint. The selected treatment regimen demonstrated a statistically significant 44% improvement of visible (non-vellus) hair count after three months of treatment compared to baseline (p < 0.001, n = 19). Across all three treatment arms, the overall improvement of visible (non-vellus) hair count after three months of treatment was 29% compared to baseline (p < 0.001, n = 48), reflecting a clinical benefit across the entire trial population and a substantially improved outcome with the optimal treatment regimen. Additionally, a prespecified analysis comparing the 44% change in visible (non-vellus) hair count to a 12% historical benchmark set by approved pharmaceutical products established statistical significance (p = 0.005).

In addition to the safety and optimization study, Follica has validated its approach in prior clinical studies using prototype HFN devices with different treatment parameters and therapeutic compounds. Follicas translational work builds on research by George Cotsarelis, M.D., who isolated and characterized the expression pattern of stem cells from a critical region of the follicle. An expert in epithelial stem cell biology, Dr. Cotsarelis is chair of the department of dermatology at the University of Pennsylvania and a co-founder of Follica.

About Androgenetic Alopecia Androgenetic alopecia represents the most common form of hair loss in men and women, with an estimated 90 million people who are eligible for treatment in the United States alone. Only two drugs, both of which have demonstrated a 12% increase of non-vellus hair count over baseline for their primary endpoints, are currently approved for the treatment of androgenetic alopecia1. The most effective current approach for the treatment of hair loss is hair transplant surgery, comprising a range of invasive, expensive procedures for a subset of patients who have enough donor hair to be eligible. As a result, there remains a significant need for safe, effective, non-surgical treatments to grow new hair.

About Follica Follica is a biotechnology company developing a regenerative platform designed to treat androgenetic alopecia, epithelial aging and other related conditions. Founded by PureTech (LSE:PRTC), a co-inventor of the current platform, and a group of world-renowned experts in hair follicle biology and regenerative medicine, Follicas experimental treatment platform has been shown to stimulate the development of new hair follicles and hair in three previously conducted clinical studies. The companys proprietary treatment is designed to induce an embryonic window via a device with optimized parameters to initiate hair follicle neogenesis, the formation of new hair follicles from epithelial (skin) stem cells. This process is enhanced through the application of a topical compound. Follica completed a safety and efficacy optimization study in 2019, and its Phase 3 program in male androgenetic alopecia is expected to begin in 2020. Follicas technology is based on work originating from the University of Pennsylvania that has been further developed by Follicas internal program. Follicas extensive IP portfolio includes IP exclusively licensed from the University of Pennsylvania as well as Follica-owned IP.

1 Olsen EA et al, J Am Acad Dermatol. 2002 Sep;47(3):377-85Olsen EA et al, J Am Acad Dermatol. 2007 Nov;57(5):767-74. Epub 2007 Aug 29Price VH et al, J Am Acad Dermatol. 2002 Apr;46(4):517-23Kaufman et al, J Am Acad Dermatol. 1998 Oct; 39(4):578-589

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Follica Announces Positive Feedback From End of Phase 2 Meeting With FDA for Its Lead Program to Treat Male Androgenetic Alopecia - BioSpace