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VOLUME 27 | ISSUE 5 | OCTOBER 2020

IN THIS ISSUE

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Bruce Levine, PhD

From the President's Desk

A World Tour from Home

ISCT President Bruce Levine discusses the challenges and hidden opportunities around staying connected to the global community of ISCT in recent months, highlighting key techniques he has used to stay in touch. 

Commissioner for Cytotherapy

An Interview with Patrick Hanley

In this exclusive interview with Dr. Patrick Hanley, the newly appointed Commissioning Editor for Cytotherapy, official journal of the ISCT, learn about his role, his vision, and what we can expect during his tenure.

Interview by C. Russell Y. Cruz
Children’s National Medical Center
Washington DC

Ashley Krull, PhD
Cellular Therapy Fellow
Department of Laboratory Medicine & Pathology
Mayo Clinic
Rochester, MN, USA
Twitter: @AshleyAKrull
LinkedIn: https://www.linkedin.com/in/ashley-krull/

From the Editors

The World According to Cellular Therapy Fellows

In this month’s editorial, contributing editor Ashley Krull provides an insightful dive into the experiences and challenges that she and three other fellows have encountered throughout their time as Cellular Therapy Fellows

UPDATES AND REPORTS
ADVERTISEMENTS AND ANNOUNCEMENTS
COMMUNITY SPOTLIGHT

Featured Member

Miguel Forte, MD, PhD

Take a few minutes to learn about Dr Miguel Forte, ISCT’s inaugural Chief Commercialization Officer, and share his passion for the great opportunities in the Cell & Gene Therapy field.

Dr. Forte’s career is a journey across the translational spectrum from clinical work on infectious diseases to academia, then to the regulatory world of the EMA and finally the commercial challenges of a Chief Executive Officer in the Cell and Gene Therapy sector.

Miguel Forte, MD, PhD
Chief Executive Officer
Bone Therapeutics S.A.
Brussels, Belgium

Vicki Antonenas BSc, MSc in Med
Principal Scientist, Scientific Laboratory Director, BMT
Sydney Cellular Therapies Laboratory, Westmead Hospital
Sydney, Australia

Leadership Spotlight

Vicki Antonenas, BSc, MSc in Med

Vicki Antonenas credits the trajectory of her career  in Australian cell therapy to the opportunities afforded her by a supportive mentor, dedicated and hard working colleagues, and her participation in ISCT global and regional activities, personal relationships with her work colleagues, and those she has met through her ISCT roles have become a major factor in her productive and satisfying career.  

Novel Treatments for COVID-19: Regulatory Challenges and Collaborative Solutions

An Early Stage Professionals Perspective

Kevin Bosse, PhD, RAC

Regulatory submissions to the US FDA present unique challenges, even more so in the face of a global pandemic. In this article, we discuss the FDA’s response to COVID-19 Investigational New Drug applications, and present our experience filing an IND to treat COVID-19 infections with a cellular therapy product. 

Kevin Bosse, PhD, RAC
Director, Drug and Device Development
Nationwide Children’s Hospital
ISCT ESP Education Committee Co-Chair
Columbus, Ohio

Janet Macpherson, PhD
Product Specialist
Cytiva
Sydney Australia

The Committees of ISCT

Aims and Scopes

ISCT has formed a variety of committees aimed to address scientific, regulatory and industry focused issues and challenges in the field. Committees are truly international in composition and scope, and serve as an invaluable networking tool.

Over the coming months we will be bringing to you some highlights of the projects and deliverables from ISCT committees. In this issue we start by sharing details of the Scientific Committees, specifically the Aims and Scope of each.

Telegraft Editorial Board

SENIOR EDITOR
Nancy H. Collins, PhD
ASSOCIATE EDITOR
Russell Cruz, MD
JUNIOR ASSOCIATE EDITORS
Satyam Arora, MD
Rounak Dubey, MBBS, MD

CONTRIBUTING EDITORS 
Shyam Bhakta, MD, MBA, FACC, FAHA, FSCAI
Ashley Krull, BSc, PhD
Anne Lamontagne, MSc
Janet Macpherson, PhD 
Lynn O’Donnell, PhD 
Michele Sugrue, MT 
Wouter Van’t Hof, PhD
Joaquim Vives, PhD

Bruce Levine, PhD

From the President's Desk

A World Tour from Home

ISCT President Bruce Levine discusses the challenges and hidden opportunities around staying connected to the global community of ISCT in recent months, highlighting key techniques he has used to stay in touch. 

Dear Colleagues and Friends, 

Like many of you, I am spending a lot less time (zero) traveling these days.  Most days I am in my home office, and about one day per week I am in my office at the University of Pennsylvania. Either way, I am still visiting South and Central America, Asia, Australia and New Zealand, and North America, with Europe coming soon.  Let me explain. 

In a pandemic world with travel severely restricted or impossible and in person meetings postponed or converted to virtual, how should we as a rapidly growing Society stay in touch? I set a goal of attending each ISCT Regional Executive Committee meeting to make introductions and to open lines of communication from the regions to our ISCT leadership.  So, this was my virtual listening tour. My first stop was Asia, where our Asia Regional VP Oscar Lee led the meeting in which we heard about planned webinars, the ISCT-KSBMT (Korean Society of Blood and Marrow Transplantation) joint session at the ICBMT 2020 virtual meeting (the International Congress of Blood and Marrow Transplantation), two ISCT-TSSCR joint sessions at the upcoming meeting of the Taiwan Society for Stem Cell Research (TSSCR) October 30-31, and the membership increasing by almost 80% over last year. Next, I traveled to meet with the crew in Australia and New Zealand.  It was a chance to say hello to old friends and meet new faces.  Rajiv Khanna as Regional VP led the meeting from Brisbane during which it continues to be apparent that ANZ activities encompass the breadth of the scientific, regulatory and quality, and commercialization pillars of the Society.  On a per capita basis, the ANZ membership is extraordinary, and growing! The ANZ group is working hard to adapt the ISCT ANZ Regional Meeting to continue to provide the flagship event within the region that members look forward to – more details to come soon. After some time at home, I next traveled to South and Central America.   Patricia Rocco, our dynamic and enthusiastic Regional VP led the meeting.  We heard updates that included the ABTCel-Gen Meeting/ SCA Regional Forum now planned for 2021 in Curitiba, Brazil, opportunities to increase collaboration within the region, and membership updates.  SCA membership has more than doubled since 2019, and Ecuador you are amazing- now at 52 members!  As a former North American VP, I was curious to see what they were up to, did they have the right stuff?  The answer is yes, definitely!  Mitch Cairo led our discussion which started with translating COVID-19 impact (lemons) into lemonade.  In other words, what are the new opportunities?  How best to overcome supply chain and staffing hurdles and get research back up to speed?  How can we help each other, isn’t that the essence of ISCT?  My next virtual trip is to Europe and I’m looking forward to completing my first world tour as your ISCT President.

Each of these Regional meetings included discussions around common themes of communication and engagement.  We have had a huge increase in membership in the past year, we are now 2,400 strong.  For all members, have you considered contributing in a way that you haven’t before? If you are interested in joining an ISCT committee, please contact the committee chair directly for information.  For long time members, you have a responsibility to reach out to new members at your institutions and in your region.  For new members, let us know why you joined, what you enjoy, and what you would like to see ISCT provide and accomplish.  What virtual platforms do you use, what content would you like to see?  If I may speak for ISCT global and regional leadership, we look forward to meeting new members and catching up with longtime colleagues.  Reach out, and let’s connect.

Now, I’d like to talk about connecting and communicating externally and using tools that you may not have used before, or have only used sparingly so far.  When I was younger (not a minute ago, I mean decades ago) scientists were not generally very proficient or interested in communicating to the public or to the media.  It was considered ill form, self-aggrandizing, celebrity envy.  The mindset was that serious scientists wouldn’t pander to the public or media.  Astronomer and author Carl Sagan, who wrote and popularized the Cosmos series had a different view.  He felt that scientists had an obligation to communicate to the public.  After all, through one mode or another, whether government funding or philanthropy or investments, the public funds scientific research and has a right to know and understand how those funds are used.  Further, I believe that if we are passionate about science, discovery, and technology to improve human health and treat disease, we should communicate that excitement and sense of wonder and commitment to the public.

With the development of new technologies including cell and gene therapies and editing the genetic code there is a wider gap now than there ever has been in the ability of scientists to explain these technologies to the lay public.  Suspicion and the breakdown of trust is accelerated by irresponsible use, misinformation, and misrepresentation of so-called “stem cell” therapies marketed direct to consumers.

Ironically, COVID-19 has provided an opportunity.  The global pandemic had been accompanied by a significant increase in appreciation for science and trust in scientists (1).  This is an opportunity to communicate science, to recruit students to the sciences, and to educate to dispel misinformation, thereby encouraging broad support and understanding for our mission.  What can you do?  Reach out to an ISCT colleague.  Brainstorm a project, an educational activity, a contribution to Cytotherapy or Telegraft.  Explore ISCT committees and resources.  Dive deep in to Telegraft, Cytotherapy, and the ISCT web pages. Reach out to your institutional community.  How many professionals might benefit from ISCT membership? 

Now that you are armed with information and ready to communicate, consider that email is so 20th Century.  Explore a platform that you may not have used before.  Join LinkedIn and connect with your ISCT colleagues, and with me!  Did you know there is an ISCT LinkedIn group page?  Find it here.  When you join the group, you will see colleagues who are also in the group.  And, by the way, there are more than 5,300 members of the group and 2,400 ISCT members, so that group is ripe for you to convince your colleagues who may not yet be ISCT members to join ISCT.  Think of Twitter like a supermarket where your buy and sell currency limit is 280 characters.  You can buy junk food or you can buy nutritious foods.  There is a wealth of information to view, from scientific journals, medical and biotechnology news, meeting reports, and science writing.  I think of Twitter as a very useful tool to have others do your collating and for disseminating newly published work, webinar announcements, summaries of talks that you give or that you find interesting, and much more (2).  Follow ISCT @ISCTGlobal and you can find me @BLLPHD.  If I can convince some of you who have so far been resistant to join, even if it is just to read content, then I’ll consider that a step forward in increasing our communications and accessibility. Remember our ISCT middle name is Communicate.   

So, let’s end with the middle. An item I’ve been saving on my Penn desk, comes from the middle of a fortune cookie. The aphorism reads, “Don’t pursue happiness, create it.”  That got me to thinking, if chaos is the order of the day, then put order to the chaos. Chaos and uncertainty are the order of the day in the wider world.  In cell and gene therapy development and translation, uncertainty is a way of life.  Let’s turn the lemons of chaos and uncertainty into the lemonade of new opportunities for communication, engagement, and growth for ISCT and cell and gene therapy.

References

  1. 2020 State of Science Index, 3M. Accessed 7 October, 2020 at https://www.3m.com/3M/en_US/state-of-science-index-survey/
  2. Soragni A, Maitra A. Of scientists and tweets. Nat Rev Cancer. 2019 Sep;19(9):479-480

Ashley Krull, PhD
Cellular Therapy Fellow
Department of Laboratory Medicine & Pathology
Mayo Clinic
Rochester, MN, USA
Twitter: @AshleyAKrull
LinkedIn: https://www.linkedin.com/in/ashley-krull/

From the Editors

The World According to Cellular Therapy Fellows

In this month’s editorial, contributing editor Ashley Krull provides an insightful dive into the experiences and challenges that she and three other fellows have encountered throughout their time as Cellular Therapy Fellows

World, meet Cellular Therapy Fellows. Cellular Therapy Fellows, meet the World. Turns out, we have a lot in common and a lot we can accomplish together. If you have never knowingly met a cellular therapy fellow before, don’t worry. We historically haven’t been the best self-promoters. We barely knew our own programs existed before joining them. But now that we’re here and growing in numbers, we thought it would be a good time to introduce ourselves and share our unique outlook on the future of the field of cell and gene therapy.

We have come a long way from the original residency and fellowship programs. For one, we no longer have to live at the hospital or lab – we will get kicked out at some point. However, our caseloads are larger and the scientific problems more complex than ever before. Not only do we absorb and apply current evidence-based practices for patient care, we also learn to interpret and implement regulations governing nearly every aspect of the programs we aspire to lead. From day one, we practice balancing academic pressures and professional service opportunities while demonstrating core competencies that are still in the process of being formalized (partly in response to our own first-hand feedback). In essence, completing a cellular therapy fellowship is like competing in the all-around gymnastics final at the Olympics – you are going to flex a lot of different muscles and demonstrate a multitude of skills before it’s over. 

One way in which cellular therapy fellowships have been ahead of the curve is in embracing learners from diverse backgrounds. On one hand, frankly, there are not enough people well-versed in the broad field of cell therapy to create a pool of “experienced” candidates in the traditional sense – candidates who have spent appreciable time on cell therapy-related scholarly pursuits or high-level research projects. On the other hand, this paucity of experience has led to the inclusion of diverse learners whose top qualifications are intelligence, leadership potential, and a penchant for innovation. If you are interested in cell therapy, then the field of cell therapy is interested in you. There is too much work to do to be turning away gifted, interested individuals who demonstrate a desire to advance the field and treat patients.

This shared motivation for patient care and product innovation was evident in my communications with three other fellows from across the country with whom I was able to connect while preparing this editorial. All of us have advanced training in biomedical research and have demonstrated the ability or desire to translate therapeutic products from the lab into clinical trials. My predecessor at Mayo Clinic, Dr. Tim Wiltshire, Ph.D., had experience in a cGMP manufacturing facility and had completed a postdoctoral fellowship developing novel cancer therapeutics before becoming the cellular therapy fellow and spearheading in-house CAR T-cell manufacturing.  Dr. Rebecca Epperly, MD, at St. Jude Children’s Research Hospital is using her fellowship year to expand both her clinical skills in managing pediatric cell therapy patients and her research skills in translating cellular therapy products for early phase clinical applications. Dr. Brian Shy, MD, Ph.D., at UCSF is able to apply graduate-level experience with pluripotent stem cells and genome editing to his practice, citing the “ability to directly translate relatively recent research breakthroughs to patient therapies” as one of his favorite parts of speciality training in cell therapy.

Just from these three examples, one can see the value in the inclusion of professionals with varied research and medical backgrounds to train to be the next generation of laboratory directors. Personally, I came to the field of cellular therapy from a biochemistry and neuroscience background. I never had a desire to attend medical school, but I always wanted my research to have a direct impact on patient care. Dr. Wiltshire had the perfect statement to articulate my thoughts on this fellowship and the chance to be a cell therapy lab director: “This is a unique role for a Ph.D. trained scientist to have the opportunity to use our skills to directly affect patient outcomes…It’s quite a sobering, humbling and exciting experience to deliver a therapy to a patient that desperately needs it.”

From the other side, having training programs that can expose medical trainees to the rigors of cGMP-level manufacturing and clinical trial design will help to synergize the future of cellular therapy development. Even though I had worked in translational research for most of my training, I was astonished by the realities of taking a therapy from the lab and making it suitable for clinical use. I imagine it would be just as difficult coming from the clinical side and needing to make sense of the cGMP regulations and cell manufacturing demands before you can hope to treat a single patient. Cellular therapy fellowships can help bridge this gap in knowledge, ideally helping both sides to have more productive conversations about how to transition laboratory discoveries into the clinic.

As Dr. Epperly asserted, “Collaboration between clinicians and scientists with diverse training backgrounds is key in accelerating innovation and clinical translation of cellular therapies…Incorporating insight from scientists across these disciplines at each phase promotes a seamless transition from laboratory observation to clinical application.”

Each of us fellows hit on the realization that we are part of a (hopefully growing) group of specially-trained professionals who will be exposed to all aspects of the cell therapy development life cycle before entering independent practice. We recognize that this increased level of awareness and shared fluency can only expedite the safe translation of pure, potent products from our cell therapy labs into well-designed clinical trials.

But what will the cellular therapy labs of the future look like? What kinds of teams will we be leading to bring these advancements to our patients? Most of us train within labs specialized in traditional hematopoietic stem cell transplants. Many of these labs are now incorporating in-house manufacturing of multiple cell-based therapies, such as mesenchymal stromal cells and dendritic cells. “I think this is where the future of cell therapy will diverge from traditional BMT labs into manufacturing facilities that operate independently to support a variety of clinical trials,” forecasted Dr. Wiltshire.

With the pace of cellular therapy development, the next generation of laboratory directors must be prepared to onboard new lines of manufacturing without compromising existing product quality and productivity. This will require a unique vision of the therapeutic landscape and potential for external collaboration. At Mayo Clinic, our training includes time in a traditional BMT lab as well as in a lab that focuses solely on delivering externally manufactured products to patients, which comes with its own set of logistical and regulatory concerns. The balance is constantly maintained between partnering with external companies and driving innovation in-house to continue providing the optimal therapies to our patients. However, as with everything else in our training, the rules are constantly being written and re-written, and we must be willing to adapt.  Dr. Shy added, “It will be interesting to see which (aspects of product development) are maintained at academic centers vs. more centralized facilities.” We have all witnessed the expansion of the field to include players in academia, medicine, and industry – and this is not likely to end any time soon. This ever-expanding universe makes our connections within the field that much more valuable and necessary as our careers progress.

Surprisingly, before I reached out for this article, not one of us had interacted with cellular therapy fellows outside of our own institutions. But we soon discovered the bedrock was there for community building. For instance, Dr. Epperly and I researched in neighboring biochemistry labs at the University of Iowa without ever knowing it. Now that we have connected, it will be up to us to continue supporting one another and to maintain these connections as we all progress into leadership positions. Fortunately, as Early Stage Professionals within ISCT, we have access to resources and opportunities for staying connected and sharing insights over the years. I encourage other cellular therapy fellows to connect with us and to engage within the ISCT community. In turn, I encourage the ISCT community to engage with cellular therapy fellows.  We are specifically training to be leaders and partners within the ISCT community to advance scientific knowledge and provide life-saving cell and gene therapies for our patients.