Abstract: Human Mesenchymal Stromal Cells Administered After Radiotherapy and Surgery in a Soft Tissue Sarcoma Mouse Xenograft Model Do Not Promote Local Recurrence or Metastasis
2018 ISCT Annual Report
Catherine Bollard, MBChB, MD
Outgoing ISCT President
(May 2016 – May 2018)
Moving ISCT Into a New Era
In August 2017, the FDA historically approved CAR T-cell therapies and for the first time ever, North Americans had access to these revolutionary treatments. That put ISCT on a real trajectory of dramatically increasing both partnerships and enthusiasm within the cell and gene therapy industry. Serving on the FDA ODAC and as the ISCT president at the time, it felt like the beginning of something of highly exciting – the beginning of a new era. It really was an energizing time for ISCT and the cell and gene therapy field as a whole.
“During my tenure as president, I learned that ISCT’s strength is rooted in the diversity of its passionate members and staff.”
As an internationally focused society, we draw on the expertise and enthusiasm of those working in cell and gene therapy across the globe. Therefore, it is critical for the field, as a whole, that we are an international society. The cell and gene therapy Industry is growing globally and as the leading cell therapy society in the field, we must evolve with that growth. From around the world, ISCT brings together minds from academia, industry, regulatory bodies and many other groups in a meaningful way, allowing the industry to “connect and collaborate.”
It’s critically important that ISCT serves different regions across the globe and continues to serve more countries and regions developing cell and gene therapies. Our slogan, “Connect. Communicate. Translate.”, is appropriate because that is exactly what ISCT does, connecting and engaging a diverse global network of cell and gene therapy professionals so they can produce new, safe therapies for you and me.
2018 was also the year my presidency at ISCT sadly ended in May. My memorable time as president was highly enriching, both professionally and personally. I have since passed the torch onto Dr. John Rasko, who I am sure will serve the society very well during this time of growth in the field. As the Immediate Past President, I am still very much involved with the Society and I am extremely confident that ISCT will continue to flourish during this extraordinarily exciting trajectory for the cell and gene therapy field.
John EJ Rasko, AO, MBBS, PhD,
FRCPA, FRACP, FAHMS
ISCT President (2018-2020)
Celebrating a Golden Age of Cell and Gene Therapy
“In 2018, there was such pinch-yourself excitement in the industry. It was excitement stemming from the fact that after decades of hard work, cell and gene therapies have become a clinical reality.”
I am truly delighted to welcome you to ISCT’s very first annual report. This marks an important milestone in our Society’s history, which mirrors the maturation of our field of cell and gene therapy. Just as a cornerstone is often ceremoniously placed near the base of a building to record historical information, so our annual report will serve as an important foundational document for future reference. The report also provides further confirmation of ISCT’s commitment during my term to promote internal and external communication for the benefit of members and professional colleagues.
I will forever remember 2018 as a significant consolidation year with an increasing number of proven cell and gene-based medicines being approved in diverse markets worldwide. New innovative technologies, increasing investment, maturing regulatory approaches and ground-breaking research are cause for reflection and celebration. The initial glow of the realization that cell and gene therapy had arrived, was reinforced by the heart-warming reality of implementing these life-saving strategies.
In 2018, there was such pinch-yourself excitement in the industry. It was excitement stemming from the fact that after decades of hard work, cell and gene therapies have become a clinical reality. Cell and gene-based therapies to treat cancer, particularly for leukemia and lymphoma, and a few select genetic diseases are now available to patients in many regions.
As you read further in this report, you will learn more about the impact ISCT has made on the cell, gene and regenerative medicine fields. You will learn about all of the passionate people within the global community of ISCT, making a significant difference in the field, potentially helping millions of people around the world.
ISCT represents an international covenant dedicated to helping the public access the safest cell and gene therapies for themselves and their families. We all share common values. All human beings have a fundamental right to access excellent health care. ISCT’s mission, vision and values focus on ultimately improving human health and reduce suffering. It is not an easy mission, but is worth the hard work.
No matter where in the world they may be located, every ISCT member sings from the same songbook when it comes our common commitment of improving lives around the world. Because of that, I think ISCT is a shining light as we pursue our goal to alleviate suffering in those individuals with unmet medical needs.
As we move forward, growing and evolving, there is much more opportunity for ISCT to connect, communicate and translate through the talented membership of our Society. 2018 was a big year and I look forward to what the future holds for cell and gene therapy. Earlier, I likened our annual report to a building cornerstone. Often these grand blocks contain a ‘time capsule’ so that historians might learn more about the epoch in which they were set. Let us ensure that our field continues to flourish with safe and effective medicines based on cell and gene technologies. I hope that future generations might confirm the sense that we are indeed living at the beginning of a Golden Age of cell and gene therapy.
Queenie Jang, BSc (Pharmacy), MBA,
Chief Executive Officer
We’re in the Midst of An Evolution
Helping facilitate that huge impact is Dr. Catherine Bollard who passionately served as our president until June 2018. She has passed the torch to the esteemed Dr. John Rasko from the University of Sydney, our first president from Down Under.
Before this evolution ever began, our founders started building the basis for this innovative field in 1992 as the only international society to bring together and to connect the entire translational value chain, helping build the thriving CGT field we know today.
Originally a society for hematotherapy and graft engineering, ISCT has since evolved, expanding its scope – and logo – to cell- and gene-based therapies, providing a wealth of education, tools, and resources to this growing field.
For 26 years, ISCT has orchestrated the work of thousands of clinicians, regulators, technologists, industry partners and academia to advance cell, gene and regenerative medicine for the benefit of patients worldwide. This includes the exciting approvals of CAR T-cell therapies which have made a huge impact – life-changing results – for patients around the globe. As more innovative cell and gene therapies come to market, ISCT recognizes the need to train the next generation of cell and gene therapy professionals to ensure the sustainability of the field.
“We are in the midst of a CGT revolution and immense growth for both ISCT and the cell, gene and regenerative medicine field at large.”
From our hematotherapy origins to our expanded cell and gene therapy scope, ISCT has a proven track record of evolving to meet the needs of an ever-changing industry. Join us, as we continue to connect, communicate and translate in establishing the CGT field as the next mainstream therapeutic area.
Our Three Pillars
Dr. Daniel Weiss, MD, PhD
Chief Scientific Officer, ISCT
Academia: Where Ideas Are Born
“By working with researchers and the scientific community, we can safely bring these cell and gene therapies to market. Science underscores everything. It’s the foundation on which all of these therapies are based on – without the science, you have nothing.”
Academic and scientific research are the infrastructure of cell and gene therapies. Without visionary scientists and researchers dreaming up the next cell and gene therapy, these medicines would not exist.
“ISCT is one of the few organizations in the world that is supporting and facilitating the cell and gene therapy field,” Dr. Weiss says. “It is extremely gratifying to be involved with this pioneering work, possibly helping many people who are ill.”
He says that through collaborative scientific and academic research, ISCT members and the global cell and gene therapy field are able to communicate in one, universal language.
“Science is a global language,” he explains. “No matter what country you’re from, ISCT enables members from across the globe to collaborate on the academic and scientific aspects of cell and gene therapy. This is where the ideas for therapies are born.”
The University of Vermont professor says cell and gene therapy is a “very exciting field.”
“It’s an exciting, fast-moving, cutting-edge field and couldn’t see myself doing anything else,” he says with a smile. “I love it because it encompasses a number of different disciplines like cell therapy, gene therapy and cancer research.”
In response to a controversial article printed in Nature International Journal of Science, ISCT members published a response, applauding the article’s effort to discuss the vexing issue of stem cell tourism. The response made it clear that ISCT, a leader in the cell and gene therapy field, is against unethical and illegal selling of unproven stem-cell therapies of all ilk and mesenchymal stromal cells (MSCs) in particular. The ISCT authors also proposed that MSC terminology promotes deceit by medical tourism groups by specifically appropriating the key word “stem” as a means to confuse the public, calling back to ISCT’s landmark position statement on MSC nomenclature .
ISCT is represented on the International Organization for Standardization (ISO) TC 276 Biotechnology technical committee by ISCT MSC Committee member Dr. Sowmya Viswanathan.
ISO TC 276 Scope
Standardization in the field of biotechnology processes includes the following topics:
Dr. Viswanathan and ISO/TC 276 Biotechnology works closely with related ISCT committees to identify standardization needs and gaps, and collaborate with other organizations to avoid duplications and overlapping standardization activities. For more information, visit the ISO/TC 276 page here.
In 2018, ISCT scientific committees published four papers outlining innovative ideas and work taking place in cell and gene therapy.
Chair: Jacques Galipeau, MD, USA
- Challenges for mesenchymal stromal cell therapies
- Response to Nature Commentary “Clear up this stem-cell mess”
Orthopedic & Musculoskeletal Therapies Committee
Co-Chairs: George Muschler, MD, USA and Christian Jorgensen, MD, PhD, France
- Proceedings of the signature series symposium “cellular therapies for orthopaedics and musculoskeletal disease proven and unproven therapies—promise, facts and fantasy
Co-Chairs: Rachele Ciccocioppo, MD, Italy and Giuseppe Orlando, MD, PhD, USA
|The ISCT Scientific Signature Series offers the unique opportunity to gather key opinion leaders in the chosen field to discuss, present and develop position statements to move the field forward. These events are aimed at driving thought leadership by providing content and building collaboration around concept papers, consensus statements, clinical networks, regulatory proposals, and recommendations for investment in clinical and basic research in cell and gene therapy medicine. The last series was in held in Florence as part of the regional meeting for Europe.|
Karen Nichols, Esq.
Chief Regulatory Officer, ISCT
Regulatory Oversight: An Essential Step for Safe Therapies
“It’s important to focus on this area because the regulatory process is what helps get safe and approved drugs into the market. Just like the academic and scientific sides, regulations are an essential step of approving safe therapies.”
“It began before the commercialization of CAR T therapies and was validated by their approval,” she explains. “These therapies are on the market and now helping many people who are stricken by disease from around the world.”
To help more people and to advance the clinical translation of cell and gene therapies, ISCT has a strong regulatory focus, driven by its Legal and Regulatory Affairs (LRA) Committees in North America, Europe, and Australia and New Zealand. These committees address challenges through liaison meetings with regulatory agencies, provide expert recommendations on official guidance documents, and deliver timely information and education to stakeholders. As a global strategy, the Global Regulatory Task Force aims to provide strategic oversight and support for the regional LRAs and the flagship Global Regulatory Perspective Workshop at the annual meeting, which is a unique forum that brings together regulators from around the globe.
“It’s important to focus on this area because the regulatory process is what helps get safe and approved drugs into the market,” she says. “Just like the academic and scientific sides, regulations are an essential step of approving safe therapies”.
But with this progress, there are significant challenges, particularly around training enough professionals to keep the industry’s momentum going. With more therapies being developed and regulated, there is an increased need for passionate people to work in the industry.
“I think there are hiring challenges depending on the geography we’re working in,” she says. “ISCT’s role is to, in my case, engage regulatory professionals from other societies. We need to collaborate with other groups to address the regulatory aspects of cell and gene therapy.”
“We need to be training and attracting younger generations, without a doubt,” she continues. “Not only will there be jobs for younger generations, but we need their skills and fresh insight to keep the industry going. I think a solution might be in the ESP program. A lot of heart and soul went into the program and folks are very passionate about preparing young generations to enter this workforce.”
In addition to the human resource issues, Karen says it is also a challenge working with the different regulatory frameworks for cell and gene therapies around the world.
“There are different frameworks we have to face in each country,” she says. “There’s also the varying maturity of those frameworks in different countries – some have very young, undefined regulatory structures, whereas others are more mature and more defined. This increases or decreases the level of uncertainty in getting approvals for these products.”
As ISCT’s Chief Regulatory Officer, Karen says she enjoys working with other sectors in the CGT industry – all for a common goal.
Since 2004, ISCT has been the host organization leading over 20 invited stakeholder organizations in an annual face-to-face meeting with the FDA Cell Therapy Liaison Meeting (CTLM). These closed meetings enable the cell and gene therapy community, and industry to inform the FDA of specific concerns, challenges and recent developments to advance the regulatory field.
The 2018 CTLM was held on January 30th in Bethesda, USA.
Since 2015, ISCT participates in bilateral meetings with the Health Canada BGTD and national stakeholder organizations in Canada in an effort to improve interactions between health care and the cell and gene therapy community as well as to improve the navigation of regulations and discuss challenges in the field.
The 2018 meetings were held on April 24th and December 4th in Ottawa, Canada.
The ISCT Global Regulatory Perspectives (GRP) represents the diverse activities and partnerships between international regulatory bodies, industry, clinicians and academia. The ISCT GRP steering group is comprised of representatives from the Society’s Legal and Regulatory Affairs Committees from Asia, Australia, Europe, North America, and South and Central America. Once a year, this joint working group holds the GRP Workshop, an annual one-day workshop that is run in tandem with the Society’s Annual Meeting. This program brings together regulators from North America, Europe, Asia, South and Central America, and Australia/New Zealand.
The Quality and Operations Track is a series of sessions held at the ISCT Annual Scientific Meeting that are dedicated to educating technical and quality professionals in the cell and gene therapy field. At the 2018 ISCT Annual Scientific Meeting in Montreal, attendees of the track were educated on a number of timely topics including advancing standards for regenerative medicines as well as day-to-day operations in a stem cell lab.
Dr. Miguel Forte, MD, PhD
Chief Commercialization Officer, ISCT
This Will Change Health Care
“The biggest revolution in commercialization is the wealth of opportunities for long-term benefit through cell and gene therapies. This is a major revolution because we go from managing attempts of chronic disease to treatments that have a major long-term impact on the patient – this will change health care. The fact we are able to modify the gene and to harness the power of the cell, enables us to provide long-term benefits with the possibility of real cures.”
“What I enjoy most is helping develop and materialize ideas into life-saving realities for these patients,” says ISCT’s Chief Commercialization Officer. “Doing that, with ISCT people who have the same passion and vision, we’re helping to bring value to patients – it’s a great thing. You have the added bonus of having fantastic discussions, seeing fantastic science and leading a passionate group of people who are all aligned on bringing value to patients.”
For some, the commercialization of cell and gene therapies could be the most exciting stage, finally bringing these revolutionary medicines into the market for all stakeholders to benefit. As a translational society, ISCT helps to take ideas through development towards launching the product into the market for patients and medical practitioners. The objective is to allow patients daily use of these approved therapies, helping millions of suffering people in need – it is the commercialization that enables that.
“The biggest revolution in commercialization is the wealth of opportunities for long-term benefit through cell and gene therapies,” Dr. Forte explains. “This is a major revolution because we go from managing attempts of chronic disease to treatments that have a major long-term impact on the patient – this will change health care. The fact we are able to modify the gene and to harness the power of the cell, enables us to provide long-term benefits with the possibility of real cures.”
He says that his experiences as ISCT’s Chief Commercialization Officer have contributed to his passion and knowledge for the field.
“It enables me to feel that I have an impact,” he says, smiling. “It enables me to connect with others who have the same passion. It enables me to see across the board in different areas – different companies and different academic institutions – as well as the advancement, the innovation and the value that’s being brought to patients all over the world. This is an opportunity to do this with likeminded people who share the same passion. It’s not only a personal pleasure but a professional one as well.”
The second ISCT Commercialization Committee Signature Series was held this past January at the Phacilitate meeting. This special event focused on product characterization and comparability, essential topics to adequately harness and control any product development, but of particular complexity in the context of live replicating cell products. Attendees of the invite-only event include members of industry, academia and other leaders from the cell and gene therapy field, all with the objective of tackling and discussing high-profile issues in the field.
|A popular issue around cell and gene therapies is the commercialization of these products and increasing access of these innovative medicines for the public. At the ISCT 2018 Annual Scientific Meeting in Montreal, Quebec, the Society held a Strategies for Commercialization Track. The seven informative sessions explored timely topics such as investor interest in cell and gene therapies, how to design a clinical trial and the tools available to accelerate cell and gene therapy development.|
In October 2017, ISCT formed a strategic partnership with Japan’s Forum for Innovative Regenerative Medicine (FIRM) to create the ISCT Japan Webinar Series. In 2018, the second webinar, ISCT Webinar 2018: Partnering in Japan: The Athersys • Healios Story, was held, educating attendees on the actual partnering experience, how to partner, the value of partnering, and key points of education from the Athersys-Healios Japan partnership. The comprehensive webinar provided insights from Athersys and Healios K.K. as well as provide the valuable manufacturing perspective for Japan partnerships from Nikon CeLL Innovation.
ISCT is a truly global organization with five distinct regions: North America, Europe, Asia, Australia and New Zealand, and South and Central America.
Membership: 51% outside of the United States from 60+ countries
With education as the core of ISCT’s value proposition to our members and the cell and gene therapy community, ISCT delivers highly curated scientific programming around the globe through its annual and regional meetings. The rotation of these meetings throughout the world provides its members with leading-edge education and networking opportunities to advance the field and drive the clinical translation of cell and gene therapies to patients.
ISCT works globally with organizations that share a common vision to drive the translation of cell and gene therapies for the benefit of patients worldwide.
- Council for Advanced Regenerative Medicine (CARM)
- Forum for Innovative Regenerative Medicine (FIRM) – 2018 Partner Organization Report
- Japanese Society for Regenerative Medicine (JSRM)
- Strategic Center for Regenerative Medicine (SCRM)
Australia and New Zealand
- Biotherapeutics Association of Australasia (BAA) – 2018 Partner Organization Report
- European Infrastructure for Translational Medicine (EATRIS)
- Joint Accreditation Committee – ISCT & EBMT (JACIE) – 2018 Partner Organization Report
- Alliance for Harmonization for Cellular Therapy Accreditation (AHCTA) – 2018 Partner Organization Report
- International Council for Commonality in Blood Banking Automation (ICCBBA)
- International Organization for Standardization (ISO) – 2018 Partner Organization Report
- Worldwide Network for Blood & Marrow Transplantation (WBMT) – 2018 Partner Organization Report
- AABB – 2018 Circular of Information CT Task Force Partner Organization Report
- Alliance for Regenerative Medicine (ARM) – 2018 Partner Organization Report
- American Society for Transplantation and Cellular Therapy (ASTCT) – 2018 Partner Organization Report
- Foundation for the Accreditation of Cellular Therapy (FACT) – 2018 Partner Organization Report
- National Academies of Sciences Engineering Medicine (NASEM) – 2018 Partner Organization Report
- United States Pharmacopeial Convention (USP) – 2018 Partner Organization Report
A Legacy of Engagement
Since 1992, ISCT has had connected more than 17,000 delegates through our Annual Meetings and communicated with more than 30,000 cell and gene therapy professionals at cutting-edge meetings, events, webinars and seminars to translate the advancement of research into clinical adoption and standard of care over the past 26 years.
Every year, the ISCT Annual Meeting delivers a highly curated scientific program showcasing the latest clinical results and technologies expanding global access to new and emerging cell and gene therapies.
About ISCT 2018 Montreal
ISCT 2018 Montreal Top 10 Countries
- United States: 759
- Canada: 333
- United Kingdom: 64
- South Korea: 62
- Germany: 55
- Australia: 40
- Japan: 38
- France: 35
- Taiwan: 22
- Singapore: 21
ISCT provided global expertise at over 10 joint sessions with leading organizations including the Japan Society for Hematology (JSH), Asian Cellular Therapy Organization (ACTO), Associação Brasileira de Terapia Celular (ABTCel), Society for Immunotherapy of Cancer (SITC), the British Society for Gene and Cell Therapy (BSGCT) and Phacilitate Trade Mission (Japan and Korea), Business of Regenerative Medicine in Philadelphia, Stanford CGMP Symposium.
Massimo Dominici, MD
ISCT Presidential Task Force Chair
Looking Out For You and Your Family
The cell and gene therapy field is experiencing a historical boom right now, growing faster than ever before. With this growth has come an increasing number of clinics and physicians around the world promoting and administering cellular therapies without the proper regulatory approval and/or scientific validation, putting patients at risk.
“With the increase in approvals of cell and gene therapies around the world and the relevant expectations of the public, we felt the need to consolidate the action of the PTF to anticipate possible issues of a booming industry and to protect patients. There are many good products available but there are some that are sold without any scientific evidence.“
To address this issue, the Society created the ISCT Presidential Task Force (PTF) on the Use of Unproven and/or Unethical Cell and Gene Therapies. This diverse group is made up of influential academics, clinicians, regulatory experts and patient advocates to address the complicated, sensitive and contentious issues around medical tourism and unethical stem cell use.
“With the increase in approvals of cell and gene therapies around the world and the relevant expectations of the public, we felt the need to consolidate the action of the PTF to anticipate possible issues of a booming industry and to protect patients,” explains Dr. Massimo Dominici, Chair of the PTF. “There are many good products available but there are some that are sold without any scientific evidence.”
He pauses for a second.
“This surplus in the availability of cell and gene therapy is a double-edged sword – one side is good because some of these therapies can help people and on the other side, there are those delivering these therapies without any scientific evidence,” he continues. “We need to stand up and develop programs like the Presidential Task Force which looks out for patients searching for therapies to treat their illnesses.”
To increase the safety of cell-based therapies for patients, the PTF collaborates with other cell and gene therapy groups, all working towards a common, noble goal: to increase the safety of cell-based therapies for patients by providing sufficient information, allowing them to make a conscious decision regarding the limitations and the high risks related to unproven cell and gene therapy strategies.
Dr. Dominici says that the strength of the PTF lies in its collaborative, passionate members, coming from different fields of cellular therapies, working together across the globe to determine the safety of the growing number of call therapies available to the public.
“What makes ISCT and our task force special is that we have a large contingent of colleagues focused also on the ethical implications of these therapies around the world,” he says. “We recognize this is an important issue and we’re doing something about it within this task force, also taking into account how much different cultural perspectives around the world may influence the way in which cell therapies are proposed and delivered to patients.”
Have you ever searched for a treatment but was not sure if it was safe and effective? Were you uncertain if the therapy had gone through the proper testing and regulatory approvals? With the explosive growth in the cell and gene therapy field comes a lot of opportunity for new therapies, but it also comes with much uncertainty for patients. To combat this, the ISCT Presidential Task Force (PTF) on the Use of Unproven and/or Unethical Cell and Gene Therapies has developed the Patient Reference Document, a comprehensive, in-depth guide for professionals and patient advocacy groups to navigate the cell and gene therapy field where there is growing uncertainty around which cell therapies have been proven with scientific and clinical evidence. The PTF also developed and published the ISCT CGT Market Authorization Report, a centralized resource and snapshot for all who are involved in the CGT industry including the scientific community, healthcare stakeholders and patient associations.
Patient Reference Document
This global guide addresses the issue of unproven cell and gene therapies, which are currently being advertised, sold and administered to patients, although they fail to achieve recognized medical standards of proof for safety or efficacy. These unproven treatments are often expensive and offered outside the coverage of routine clinical care. They are not part of a conventional clinical trial and lack regulatory oversight. With this document, the ISCT Presidential Task Force hopes to educate patients and health care professionals, providing resources to determine whether a therapy has been deemed safe and effective by the appropriate governing bodies.
CGT Market Authorization Report
Recently published in Cytotherapy, the official ISCT peer-reviewed journal, the ISCT CGT Market Authorization Report is a regularly updated document that will be republished yearly and available on the ISCT website. This report is the result of an increase in the number of CGT market authorizations, as well as an increase in unproven approaches where cells are delivered as treatments without rigorous scientific and regulatory assessment, and authorization. Like the Patient Reference Document, but more of a snapshot, this reliable, up-to-date resource will help practitioners and patients to better understand what is on the market for cell and gene therapies. This report ultimately allows practitioners and patients to make informed decisions, avoiding exposure to unproven and unlicensed cell interventions not approved by a regulatory or medical agency.
The Worldwide Network for Blood & Marrow Transplantation (WBMT), in partnership with the ISCT Presidential Task Force on the Use of Unproven/Unethical Cell & Gene Therapies, is preparing a position paper on unproven cell-based therapies: A Summary of the Current Global Status and Recommendations to the World Health Organization (WHO).
An international and multidisciplinary group of experts representing international societies involved in stem cell transplantation, cell therapy and blood transfusion was convened under the auspices of the WBMT. WBMT is a non-governmental organization affiliated with the WHO.
ISCT is a member of the National Academies of Sciences Engineering Medicine’s Forum on Regenerative Medicine. The forum brings together academia, industry, government, patient and provider organizations, regulatory bodies, foundations, societies, associations, and other groups, to discuss the challenges and opportunities of regenerative medicine, potentially improving the health of millions of people worldwide through the development of effective new therapies. The ISCT representatives on this reputable forum are Dr. Daniel Weiss and Karen Nichols.
Helping Dot the i’s in the Cell and Gene Therapy Field
Bill Milligan, Co-Chair, ISCT Business Models and Investment Sub-Committee
Patrick Rivers, Co-Chair, ISCT Business Models and Investment Sub-Committee
There is no doubt that the cell and gene therapy world is experiencing unprecedented growth at the moment. While it is an exciting time for cell and gene therapies (CGT), it is also a new, evolving field with a significant need for knowledgeable guidance and education. To support this need for providing more knowledge and education, as it relates to financing CGT research and development, ISCT created the investigators to investors (“i to i”) Program.
“The program will help enable investigators to better understand how to present a compelling case to investors to secure support for their technology or product candidate development,”
explains Bill Milligan, Co-Chair of ISCT’s Business Models and Investment Sub-Committee. “The investigator needs to really understand and articulate how their CGT candidate works, how to make it, and what benefit it is going to bring the patient. On the other side, we can educate investors about the important considerations for investing in this new CGT industry. It’s a win-win situation.”
Patrick Rivers is a Co-Chair of the sub-committee and he is also a Managing Principal at Aquilo Capital Management. He emphasizes that as an investor, the i to i Program is a valuable resource that is needed in the industry right now.
“Cell and gene therapy is a complex and highly technical space within drug development,” Patrick explains. “One of the goals of the i to i Program is to provide investors with a forum to learn more about the challenges and opportunities in this rapidly growing landscape within human therapeutics. Ultimately, we want to make it easier for investors to execute scientific diligence that gets them comfortable with investing more capital in cell and gene therapy companies.”
Unlike conventional medicines, cell and gene therapy development has many more complex factors to consider, including intricate manufacturing dynamics which result in higher costs, and advanced cold-chain infrastructures for storage and distribution.
“The whole world is starting to see how much benefit there is in these emerging cell and gene therapies, but also how complicated it is,” said Bill, who is also Senior VP of Business Development at Steminent BioTherapeutics Inc..
To determine further what financial backers are looking for from investigators, ISCT partnered with Bloomberg Intelligence to develop and distribute a survey last fall to over 2,000 investors. The information gathered from the survey is being used to create educational initiatives to help empower investigators in the field.
“With the i to i Program, we want to take the elements we learned from the survey and turn that into educational, insightful information for investigators,” Bill commented.
Patrick says the information from the survey is valuable to investors as well.
“By first exploring the current attitudes and concerns of investors regarding cell and gene therapy with this survey, we’ve identified several areas where we can build content using the thought leadership within ISCT, directing that content back to the investment community,” Patrick commented.
“Investors are always looking for new avenues to conduct diligence that gives them an informational advantage relative to other market participants – the i to i Program can help them achieve this for CGT.”
Training the Next Generation
With the dramatic growth in the cell and gene therapy field, there is an urgent need to train the next generation of professionals. ISCT is playing a key role in developing the careers of Early Stage Professionals (ESP) as they enter the CGT field in record numbers.
“The biggest problem facing the growth around the world is a shortage of qualified people – it is a global, industry-wide issue.”
– Bruce Levine, PhD, President-Elect, ISCT
Over the past 10 years, the cell and gene, and regenerative therapy field has been experiencing unprecedented growth around the world. The US Food and Drug Administration predicts that, incredibly, the approval of cell and gene therapies will increase up to 900 percent in the next 10 years.
Long-time ISCT member and President-Elect Bruce Levine believes the field is going through a very pivotal phase at the moment.
“I think that we have a huge amount of seed and young plants spread around and there is going to be a weeding-out process,” he says. “We’re all going to grow and some are going to be successful therapies and companies, but others may not. The ones given sunshine and water and sustenance will grow and flourish – the ones in the shade without water will die away. We’re in a very unique phase of innovation where there are there are many new ideas coming out from academia now being developed by industry – the way to effective therapies is through different approaches.”
Even though times are positive in the cell and gene therapy field, there are challenges, particularly with the qualified human resources to accommodate this boom in the cell and gene therapy field.
“The biggest problem facing the growth around the world is a shortage of qualified people – it is a global, industry-wide issue,” he says looking out the window. “The field is growing so fast that there just aren’t enough undergrad and grad students who have a background in cell and gene therapy. So we need to encourage that pipeline of talent while taking people from other fields and cross-training them.”
With this dramatic growth in the cell and gene therapy field, Bruce thinks that we need the qualified infrastructure in place to train future professionals in the relatively young cell and gene therapy industry.
“We need programs in place to train these people,” he says. “I’m part of a couple of programs at our institution and collaborators for education on cell manufacturing and training programs. Because it is such a young industry, we are just thinking about how expand and build on existing programs to train more cell and gene therapy professionals.”
In August 2018, the ESP Committee launched the ISCT Mentoring Program following a successful pilot program in 2017. Mentors and mentees represented all five ISCT regions. The program provides ESPs with a unique opportunity to receive professional guidance from leaders in the cell and gene therapy field. The program pairs an experienced cell and gene therapy leader with two mentees who learn and grow from the knowledge passed down by the mentor. Recruited from a range of cell and gene therapy-related areas, the experienced ISCT mentors provide a wealth of career and professional development guidance for aspiring cell and gene therapy professionals entering the field.
Australia and New Zealand
- Janet Macpherson, Product Specialist – Cell & Gene Therapy ANZ, GE Healthcare Life Sciences
- Dominic Wall, Director Pathology, Operations, Peter MaCallum Cancer Center and CSO, Cell Therapies Pty Ltd
- Tatiana Astrelina, Head of Center for Biomedical Technologies, State Research Center Burnasyan Federal Medical Biophysical Center FMBA of Russia
- Christian Chabannon, Head and QP, Cell Collection and Processing Facilities, Institut Paoli-Calmettes
- Cornelia Kasper, Professor, Department for Biotechnology, BOKU
- Ali Mohamed, VP of CME, Immatics Biotechnologies
- Anthony Ting, VP of Regenerative Medicine, Athersys, Inc
- Bruce Levine, Professor and Director of the Clinical Cell and Vaccine Production Facility, University of Pennsylvania
- Ed Horwitz, Acting Professor of Pediatrics, Director of Transplantation Biology and Therapeutics, Emory University School of Medicine
- Nadim Mahmud, Associate Professor of Medicine, University of Chicago Illinois School of Medicine
- Sandeep Soni, Associate Professor of Pediatrics, Stanford University
- Shirley Bartido, Director, Regulatory Affairs, Cellectis, Inc.
South & Central America
- Roberto Fanganiello, Consultant, RDF Consultancy, R-Crio, InnovaSpace, STAR Associates
The ISCT 2017-2019 Strategic Plan identified the importance of grooming the next generation of ISCT leaders. This led to the development of a strategic objective to have one Early Stage Professional (ESP) on every ISCT committee. This objective strengthens the future of the Society and the cell and gene therapy field by involving younger generations in ISCT’s many initiatives.
ISCT ESP Networking event at ISCT 2018 in Montreal, Canada
There is an unfulfilled need for cell therapy training covering the process of translational research to cell manufacturing and clinical trials in cellular therapy including regulatory components. As a result, ISCT and the American Society for Blood and Marrow Transplantation (ASBMT) have created the exclusive Cell Therapy Training Course to train the next generation of translational experts in cellular therapy. After a thorough and comprehensive application process, six North American scholars and six international scholars are selected to attend this leading-edge course, taught by world class faculty. The third biennial 2019 Cell Therapy Training Course will be held this fall in Philadelphia, hosted by the University of Pennsylvania.
With the tremendous growth in the cell and gene therapy field comes a greater need to attract emerging professionals to drive this thriving industry. ISCT created the Early Stage Professionals (ESPs) Committee to provide a networking and educational forum for new cell and gene therapy professionals from around the globe, working to establish the future leaders of not only ISCT, but the field at large. The ESP Committee is comprised of 20 members from 6 countries who develop programming targeted at their fellow young professionals including sessions at the ISCT Annual Meeting, mentoring programs, the Young Investigator Abstract Awards, and more.
“I knew I could make a difference if I chose this field.”
Dr. Nancy Collins’ passion for curing cancer came at a young age.
“It started when I was in eighth grade,” she recalls. “For my science project that year, I chose leukemia. I chose it because a friend of the family had died of leukemia and I was interested in why.”
Seeing that this passion was real, Nancy’s aunt arranged for her young niece to visit a local medical lab.
“I looked around where the med tech worked and I thought he had a lot of cool stuff and was doing important work,” she recalls. “He explained to me how they diagnose leukemia, what a centrifuge was, what a white blood cell was and I was hooked – it lit a spark. I knew I could make a difference if I chose this field.”
40 years later, that spark has become a bright, burning flame. Nancy got her PhD in microbiology and immunology (“I became allergic to all of my lab animals.”) and has become a leading authority in cell and gene therapies, establishing a distinguished research career – and co-founding the ISCT.
As senior editor for ISCT’s newsletter, The Telegraft, as well as a number of other roles including teaching at the University of Toledo, Nancy has also become a mentor to future generations in the cell and gene therapy field, a job she says is “fulfilling,” especially after retirement.
“We need to transfer the existing knowledge and high standards that ISCT has built and transfer them to younger, enthusiastic generations so they can continue the good work,” says the winner of last year’s ISCT Career Achievement Award. “As editor of the Telegraft newsletter, I have the opportunity to mentor others and give back what I know. When I was offered the editor position as a mentoring opportunity it hit my sweet spot because if we set up a good mentoring system, we can pass a lot of knowledge to the younger generations.”
The cell and gene therapy field has recently experienced a period of explosive growth and because of that, Nancy says that organizations like ISCT are needed more now than ever.
“We need to have strong communications between the various parts of the field,” she explains. “The clinical part, the translational part, the laboratories, the regulatory bodies – they all need to be coordinated and we need everyone talking to each other. ISCT is the central forum that connects all of these groups and people together to create new therapies for people around the world.”
For 17 successful years, Dr. John Barrett devoted his time and seasoned knowledge as the Senior Editor of Cytotherapy, the official journal of ISCT.
I had the privilege of editing Cytotherapy since September 2001 and at the end of 2018, I passed the baton of responsibility to Donald Phinney, the new Senior Editor. It has been a rich experience for me and an exciting adventure in publishing, serving the International Society for Cell and Gene Therapy. I am happy to leave the journal in a strong position, well deserving its subtitle “The Journal of Cellular Therapy.”
As to success, the journal metrics speak for themselves. First, the volume of papers published has increased three-fold. In 2000 we had six issues per annum and published 41 papers. In the last 12 months we have published 26 reviews, commentaries, and 123 regular articles and letters. Meanwhile our impact factor began below 1.0 in 2000, rose to the 3’s in 2006, reaching 3.99 this year. The contents have broadened from the solid base of mesenchymal stromal cells to cover the development of a huge spectrum of cell types used in therapy, including iPS cells, and increasingly gene-modified cells, and the new field of exosomes. Notable growing areas in the journal repertoire are immunotherapy – T cells, NK cells and dendritic cells, clinical cell therapy trials, technical and regulatory reports and numerous position papers and authoritative documents from the ISCT membership.
At the heart of the success of the journal is the editorial team, the publisher, and the Society. Sustaining a journal that meets the needs of the authors and the acclaim of the readership has needed teamwork and strong collaboration between Elsevier, the publisher, the ISCT, and critically the Senior Editor and the Associate Editors.
Elsevier has served as our publisher since 2013 and continues to be a powerful force for delivering our product, driving expansion, and supporting the goals of the journal and the ISCT. In particular I should mention our past publisher Vindra Dass and Angela Welch our current publisher who serves the journal with imagination and dedication. Elsevier is strongly committed to fostering the journal’s upward trajectory. Our publishers can call upon the worldwide presence, sophisticated information technology and not least, the international recognition of Elsevier.
ISCT has always recognized the importance of Cytotherapy for the Society and Queenie Jang, our CEO, has been a major influence in the development and expansion of the journal, strengthening its integration within ISCT. Since 2000, the journal has lived through eight ISCT presidencies and the fact that four of them have served, or continue to serve as associate editors, is an indication of the close association of the journal with the ISCT.
The team: I have had the privilege of working with a team of associate editors who have put in hours of work to process manuscripts and provide their input on the quality or otherwise of the material submitted. Metrics of paper handling provide a feedback to every editor and have tracked an improvement in the pace of manuscript turn-around. We are conscious of our role in serving would-be authors by rapid paper processing, fair, expert, and in depth reviews, and a full explanation of the reasons for declining a manuscript when we reject. The rarity of author complaints is a sign that we provide good customer relations here. I cannot finish without listing all my colleagues who have served as editors from Graham Sharp, Gunnar Kvalheim, Jed Wolchok, Edwin Horwitz, Massimo Dominici, Robert Deans, Cath Bollard, Katy Rezvani, Don Phinney, John Rasko, to Peiman Hematti. To these loyal colleagues who have kept the lifeblood of Cytotherapy flowing, day in, day out, for many years – and to Elsevier and the ISCT – a heartfelt thank you! I could not have done it without you!
Cytotherapy is fortunate to have Donald Phinney as its new Senior Editor. A strong ISCT member with a broad experience in journal publishing, Don will handle the day-to-day running of the journal with rapid and impeccably fair and well considered turnarounds for submitted papers. Beyond that, he will innovate the journal’s shape and contents. I look forward to seeing Cytotherapy continue to rise and remain a flagship not just for the ISCT but the for the entirety of this fast-moving and always exciting cellular therapy field.
One of the world’s foremost publications on drug development, The Medicine Maker, distinguished Dr. Miguel Forte, ISCT Chief Commercialization Officer as one of the top 100 inspirational industry professionals on its 2018 Power List. Dr. Forte was recognized in the Industry Influencers category – other categories included Master of the Bench, Business Captains and Champions of Change. All individuals on this notable list have, in some way, contributed to life-saving work in the global biopharmaceutical industry, improving lives around the world.
ISCT Abstract Awards
To help up-and-coming scholars in cell and gene therapy field, the Society presents the ISCT Abstract Awards, which awards thousands of dollars to emerging scholars and an opportunity to showcase their research on a global platform. Please meet the 2018 winners:
ISCT 2018 Best Poster Abstract Award ($1,500)
Abstract: Human Placental-Derived Adherent Stromal Cells Co-Induced with TNF-α and IFN-γ Inhibit Triple-Negative Breast Cancer in Nude Mouse Xenograft Models
ISCT 2018 Best Oral Abstract Award ($1,500)
Abstract: Allogeneic Amniotic Epithelial Cells for Established Bronchopulmonary Dysplasia in Premature, Low Birthweight Infants: A First-in-human Safety Trial
ISCT 2018 Technologist Abstract Award ($1,000)
Abstract: Safety Considerations in the Generation of Clinical Grade Autologous IPS Cell Lines
Abstract: First Trimester Human Umbilical Cord Perivascular Cells Expanded With CGMP Compliant Human Platelet Outperform Conventional MSC Sources As Regenerative Therapy in a Rat Model
ISCT 2018 Young Investigator Abstract Award ($1,000)
Abstract: Combined Cell and Gene Therapy Towards the Treatment of Age-related Macular Degeneration and Diabetic Retinopathy
Abstract: Close Neighbors in the Niche: Paracrine Signals From Endothelial Cells Promote Alveolar Epithelial Differentiation of Induced Pluripotent Stem Cell-derived Lung Progenitors
Abstract: Safety of Stem Cell-Derived Encapsulated Liver Tissue to Treat Liver Failure: Immune-Isolation and Absence of Foreign Body Reaction or Tumor Formation Upon Transplantation
ISCT EU 2018 Awards
Abstract: Targeted Stromal Cells Expressing Trail as a Therapy for Lung Cancer
Abstract: Safety and Feasibility of a Tolerogenic Dendritic Cell-Based Vaccination in MS: A Collaborative Initiative Comparing Intranodal and Intradermal Cell Administration
Presented to Riccardo Saccardi, JACIE Medical Officer
ISCT ANZ 2018 Awards
ABTCel 2018 Awards
2018 was a big year for Cytotherapy, the official journal for ISCT. An essential resource for cell and gene therapy clinical researchers, oncologists, hematologists, physicians, and regulatory experts, the publication named Dr. Donald Phinney as the new senior editor as well as Dr. Oscar Lee, Dr. Luis Ortiz and Dr. Sowmya Viswanathan as the new associate editors. The new editorial staff brings a wealth of experience to the journal, undoubtedly reinforcing Cytotherapy as a premier source for cutting-edge findings, clinical trials of cell-based therapies, and news and opinion pieces on all aspects of the rapidly expanding field of cell-based treatments for cancer, degenerative disorders, immunotherapy and stem cell transplantation.
You can read issues of Cytotherapy here.
Available to ISCT members around the world, Telegraft is the Society’s leading global cell therapy newsletter which updates readers on new cell and gene therapy developments, regulatory updates, and regular columns on related meetings, organizations, and events, summaries of work being done in both academic and industry labs around the world. In 2018, not only was Telegraft redesigned with a fresh new look, but the bi-monthly e-newsletter named Dr. Nancy Collins as its new senior editor, a prominent researcher, mentor and leader in the cell and gene therapy field. Telegraft also recruited two Early Stage Professionals (ESPs) Dr. Alireza Abazari and Dr. Satyam Arora, as the new junior associate editors, bringing fresh perspectives to the publication.
You can read issues of Telegraft here.
ISCT membership benefits your cell and gene therapy career in a number of valuable ways with exclusive access and complimentary subscriptions to Telegraft and Cytotherapy, as well as networking opportunities to collaborate with key opinion leaders, government regulators, commercial partners and cell therapy technologists. An ISCT membership also provides:
- Reduced rates at ISCT-sponsored events, including webinars, technical workshops, research and clinical symposia, and the ISCT meetings.
- Access to members-only web resources, including the ISCT Member Networking Database and online discussion boards.
To learn more about ISCT memberships and how to join, visit our website.
ISCT has 41 stakeholder committees, working in a number of scientific, commercial and regulatory disciplines within cell and gene therapy. Joining an ISCT committee is an invaluable networking opportunity, allowing you to connect with cell and gene therapy professionals from all over the world. If you would like to bolster your career by joining an ISCT committee, visit our Committees page to see how.
Are you interested in recruiting for a cell therapy-related position in your company or institution? The ISCT Career Center is the perfect opportunity to highlight your organization, profile open positions, and recruit highly qualified cell therapy professionals from a targeted audience of cell therapy experts, including scientists, technologists, and business professionals.
ISCT has a large number of valuable and comprehensive resources for ISCT members and cell and gene therapy professionals. This toolbox of resources includes a useful glossary of cell therapy terms, the ISCT Cell Therapy Bioprocessing Tools and Reagents Database, a centralized catalogue of available products for use in cell therapy processing, manufacturing, and research, as well as valuable patient resources from the innovative ISCT Presidential Task Force (PTF) on the Use of Unproven and/or Unethical Cell and Gene Therapies website, which was launched in 2018.
Visit the Cell Therapy Community Resources page to learn more.
Cytotherapy, the official journal of ISCT (International Society for Cell and Gene Therapy), publishes novel and innovative results from high-quality scientific and clinical studies in the fields of cell and gene therapy. Reaching a global audience of cell and gene therapy professionals each month, content published in Cytotherapy is an essential and reliable resource for clinical researchers, oncologists, hematologists, physicians, and regulatory experts around the world.
Aims and Scope
Studies evaluating the potency of experimental cell and gene therapies in clinically relevant animal models of disease and describing important advances in cell/gene-based product manufacturing and validation are welcomed. Results of clinical studies evaluating the safety and efficacy of cell and gene therapies in early- and late-phase trials are also of interest. In addition to short reports and full-length articles, the journal also accepts editorials addressing emerging trends and potential controversies in the field, and review articles summarizing bodies of work that have made lasting impacts in the field.
– Short Reports
– Full-Length Articles on:
– Translational Studies
– Cell Therapy
– Gene Therapy
– Regenerative Medicine
Please visit the journal submission page for more information.