ISCT Special Report: Advancing Cell Therapy Manufacturing at the 2015 Annual Meeting

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ISCTSpecRPT-Cover_1The International Society for Cellular Therapy (ISCT) will host its 21st Annual Meeting at Caesars Palace Hotel and Convention Center, Las Vegas, NV, 27–30 May 2015. More than 1,200 industry and regulatory professionals, clinicians, scientists, and laboratory professionals are expected to attend. The program covers six plenary sessions, six workshops, three technical sessions, and more than 20 total track sessions covering such topics as advances in cell therapy research, commercialization strategies, quality and operations, and regulatory issues.

BPI spoke with plenary speakers and chairs of the “Strategies for Commercialization” track about how ISCT and cell therapy professionals are advancing the field. Here, they introduce the “hot topics” within commercialization and issues driving the cell therapy industry into its “coming of age” from R&D and early phase development, through clinical development, and ultimately to market launch.

Presidential Plenary

Massimo Dominici is President of ISCT and assistant professor of medical oncology at the University of Modena and Reggio Emilia, Italy. He will chair the Presidential Plenary titled “Dissecting MSC Heterogeneity from Unexpected Ontogeny to Specialized Functions” on Thursday, 28 May 2015, 9:15–10:45 am.

Would you provide some information about the work that ISCT does for the commercialization of cell therapy? “ISCT was founded in 1992 as a global organization that aims to facilitate the translation of basic research into cell-based products in different medical areas such as cardiovascular, neurological diseases, and cancer. One of the very first examples of stem cells was provided in 1909 when a scientist named Alexander Maximov was hypothesizing the existence of stem-cell progenitor cells in adults observing lymphocytes (immune cells). After more than 100 years, the field generated several solid findings, and we now have the possibility to isolate stem cells and lymphocytes for targeted therapeutic purposes. During these developments, we also recognized that these elements can be extensively amplified outside of our body. Starting with one or very few single cells, we can obtain up to billions of cells for multiple biomedical applications. Even if this may be now considered “normal,” I am always surprised that we have been able to reach this level of technology, at relatively low costs, worldwide.

“For over 20 years, ISCT has seen a progressive understanding in the way scientists can isolate and manipulate cells for preclinical studies and as therapeutic tools. These were important milestones; however, during these improvements several fundamental questions were raised: From which tissue can we isolate the best-performing cells for therapeutic purposes? How can we manage to efficiently isolate them? How can we safely amplify them for therapeutic purposes? In which way can we distinguish cells that are and are not wanted in a precise clinical application? How can these cells last outside a body, and how can we safely deliver them to patients after that ex-vivo life? What are their real mechanisms of action?

“The answers are (in part) provided thanks to the mix of innovative basic science, mainly developed by universities, and the relevant technological improvements driven by industry. Thus, ISCT combines the value of academic research with new technologies to facilitate answers and therefore facilitate development.

“ISCT has been following the activities in cellular therapies closely and I presume we are now in a revolutionary phase at which we can actually have cells in a clinic. We are also seeing the first cell-based products approved in some geographical areas. That is very impressive, considering that those products must be framed properly into different regulatory regions such as the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Pharmaceutical and Medical Devices Agency (PMDA) in Japan. I think ISCT should constantly try to bridge academic research and industrial development within recognized regulatory frameworks. ISCT will also do its best to collaborate with other sister organizations in the field to reach a harmonized view on the final goal: the delivery of safe and effective cellular therapies to improve patients’ lives.”

Would you briefly overview the committee structure of ISCT and describe each committee’s focus? “We have seven scientific committees. Some focused on cell types and technologies, such as hematopoietic and cord blood stem cells, mesenchymal stem cells, and immune cells and gene therapy. Others are “tissue/disease focused” committees in neurology, cardiovascular, and pulmonary areas.

“ISCT also has a Laboratory Practices Committee that discusses laboratory and technical aspects related to cell processing. There we have members addressing, for example, the manner for characterizing cells and in general good practices and methods aimed to standardized assays in cell therapy development.

“We also have a Commercialization Committee made of a team of experts coming from universities and industry. They share ideas and strategies on how to transfer robust scientific findings of cell therapeutics into commercialization dealings with issues such as the reimbursement of cell-based products and other extraordinary projects. ISCT is a unique society with members from different geographic locales, globally. Each region has unique regulatory requirements. One of our goals is to try to enforce activity related to regulatory affairs. We have established committees that deal with regional needs of Europe, North America, Australia, and New Zealand. We are establishing new ones in South America, and we aim to have one in Asia.

“Finally, since communication is a very relevant aspect in cell therapy, we have a communication committee that bridges the gap between the islands of information in the Society creating a streamlined process for disseminating Society activities by several communication portals.”

What’s new at ISCT? “Many things. In 2014, we launched our Advanced Practice Professionals committee. The idea is to try to be on the edge of cell-based therapy. The team aims to promote the knowledge and the professional practice of those professionals (e.g., nurse practitioners, physician assistants, and pharmacists) who are interrelated to cell therapies and engaged in the delivery of cell therapy to patients.

“Then we have an Early Stage Professionals committee, which aims to reach out to university PhDs and post-doctorates who are devoting their time to cell therapy research. This is very important for the future of the Society. We will embrace them, share our concepts, and to listen to their new ideas.

“ISCT also has created a Presidential Task Force to address the issues on ‘unproven’ cellular therapy — meaning cells that are transplanted into patients without clues of scientific evidence. In the past, that was called ‘medical tourism.” Now people are finding those treatments in their own countries, indicating the level of the problem I was, myself, involved in this type of issue in Italy with the Stamina Foundation. Since then, I got very much involved and decided to spend some time to explore whether we can group together in ISCT expertise and more in providing clearer information for patients. Here, we aim to generate the awareness that stemness does not always lead to a curative potential. In other words, stem cells do not cure everything without preclinical evidence supporting it. The activity of this Task Force will be presented in Las Vegas, during this ISCT Annual Meeting on May 29th, and all are invited to attend.”

Why should BPI readers attend the ISCT meeting? “The people participating in ISCT meetings are those who are generally talking the same language, maybe from different perspectives, but the same language. They have the same needs. They certainly have encountered the same problems in their laboratories, their facilities, and in their manufacturing processes. They may have the same questions and during the ISCT meeting they will find answers and new ideas to be developed.

“We address continuous development in the field, and you will find speakers discussing the design and validation of current good manufacturing practice (CGMP) facilities, dealing with ancillary materials, cold supply chains, and much more. The meeting also includes basic to translational aspects on mesenchymal stem cells, hematopoietic cells, immunotherapy, and others. I encourage people working in these fields to participate and get an updated view of what is 2015 state-of-the-art in cell therapy.”

Is there anything else that you think BioProcess International readers would be interested in? If you are not familiar with the Society, I encourage you to visit our website and participate through our annual meeting. You will get a taste of the different interests that ISCT is pursuing and what are the unique benefits of membership. I’m sure that if BPI readers are involved in cell therapy, they can certainly find topics of interest that will provide answers to their questions.

Commercialization Track

Aby J. Mathew is senior vice president and chief technology officer at BioLife Solutions. He is chair of the Strategies for Commercialization track and will chair the plenary “Game Changers: Policy and Practice Changes That Have Enabled Commercialization of Cell Therapy Products” on Friday, 29 May 2015, 2:00–3:30 pm.

What are the main topics for the commercialization track? “This year’s session reflects somewhat of a transition period in cell therapies. We have recently seen some promising clinical developments with cellular immunotherapies and a related uptick in investment activity around companies in that field. We have also seen a pioneer in our industry declare bankruptcy even after achieving approval of a cell-based therapy and the Japanese regulatory environment allow for conditional approval to decrease the timeline for a potential therapy to reach patients.

“We have three speakers who are going to provide their perspectives on how regenerative medicine is evolving. Speakers will discuss ways to pay for therapies, financial markets, investors, and current Japanese commercial and regulatory perspectives. We have heard that Japan is implementing conditional accelerated approval of cell therapies. So speakers will provide their insight into some new and current issues that are going on in regenerative medicine and what is driving the industry forward.”

What are the challenges that scientists and companies are facing in commercialization? “The focus of the commercialization track extends to challenges beyond clinical aspects. We address questions such as whether these therapies will be manufactured consistently and within an affordable cost of goods so they will be sustainable. Do we have the tools and methods to characterize the therapies as we want (or should)? Can they be stored and shipped most effectively to reach a global patient population? Will governments and insurance companies pay for these therapies? Is it possible to make the approval pathway more efficient without increasing risk to patient populations? How does the investment and finance community really feel about these therapies (and what worries them about this field)? What don’t we know that we wish we knew?

“ISCT brings together the stakeholders who are key to the long-term success of this field. If clinicians interacted with only clinicians, then cell therapies might remain hospital-based therapies, and manufacturing efficiencies would not be optimized or consistent from site to site. If industry interacted with only industry, then there might be a disconnect regarding what works best for the clinicians and patients. ISCT brings different team players together to share ideas, debate the path forward, and execute a game plan. This is the best way to reduce risks and maximize the probability for successfully delivering treatments to the patients.”

Plenary Speakers

Presidential Plenary
Massimo Dominici, MD (University Hospital of Modena, Italy)
Ivan Martin (University Hospital, Basel, Switzerland)
Simon Mendez-Ferrer (Spanish National Center for Cardiovascular Research, Spain)
Kaj Fried (Karolinska Institute, Sweden)

Cellular Immunotherapy
Malcolm Brenner, MD, PhD (Baylor College of Medicine, USA)
Richard Childs, MD (National Heart, Lung and Blood Institute, USA)
Richard Vile, PhD (Mayo Clinic, USA)
Advanced-Phase Clinical Trial Updates
Robert Deans, PhD (Athersys, Inc. USA)
Jeffrey Cohen, MD (Cleveland Clinic, USA)
John Rasko, MBBS PhD FRCPA FRACP (Royal Prince Alfred Hospital, Australia)

Commercialization
Aby J. Mathew, PhD (BioLife Solutions Inc., USA)
Nick Crabb (National Institute for Health and Care Excellence, NICE, UK)
Reni Benjamin (H.C. Wainwright and Co., USA)
Yuzo Toda (Forum for Innovative Regenerative Medicine (FIRM), Fujifilm Corporation, Japan)

Tissue Engineering
Allan Dietz, PhD (Mayo Clinic, USA)
Jeff Ross, PhD (Miromatrix Medical Inc., USA)
Scott Hollister, PhD (University of Michigan, USA)
Utkan Demirci, PhD (Stanford University, USA)
Not-for-Profit Organizations and Contributions to Advancing Cell Therapies
Randy Mills, PhD (California Institute for Regenerative Medicine, USA)
Michael May, PhD (Center for Commercialization of Regenerative Medicine, Canada)
Keith Thompson (Cell Therapy Catapult, London, UK)

View the full ISCT 2015 Meeting Program

What are some interests from a commercialization angle? “As chair of the commercialization track program, I head up a team that focuses on topics of interest within the ‘commercialization umbrella.’ That includes manufacturing and process development, business and financial considerations, assay development, and so on. We address the underlying question of how to translate individual therapies into the broad application of medicine while being consistent and fiscally sustainable.

“We came upon some of these topics while brainstorming with key opinion leaders within our Strategies for Commercialization Committee team, the wider Commercialization Committee of ISCT, and the Organizing Committee. We were trying to address a critical juncture of regenerative medicine by considering key topics that might enable long‑term success — from a manufacturing and commercialization perspective — for moving those therapies forward.

“There are certainly policy and legal issues, such as Japan’s regulatory developments or the United Kingdom’s handling of reimbursement. There are also aspects that we haven’t yet fine-tuned, such as defining potency assays to characterize our products. Even though we are seeing clinical benefits, there is still room for improvement in terms of characterization of potency. And certainly another big topic is reimbursement and how it affects long-term success or not.

“Everyone is looking ahead to how cell therapies and regenerative medicines will get approved. That’s driving financial investment. Last year, we saw great investment into companies that focused on chimeric antigen receptor (CAR) T cells and immunotherapies. That’s a hot topic right now. We are trying to learn from what has been happening in the past one or two years to help catapult us to the next level of commercialization.”

Can you talk a little bit about your company, BioLife Solutions, and how the work your company does has helped with the efforts of ISCT? “BioLife Solutions and ISCT are great partners in the support of regenerative medicine. ISCT brings together the stakeholders involved with developing, manufacturing, and delivering regenerative medicines while also supporting development of best practices throughout those processes. Our mission at BioLife is to further biopreservation best practices. This really has been embraced and implemented by a growing number of cell therapy developers, now exceeding 175 clinical regenerative medicine applications throughout the world. Furthermore, within regenerative medicine there has been recognition and growing interest in the topic of biologistics — biopreservation and supply chain logistics to optimize therapeutic efficacy and cost of goods while minimizing risks throughout the clinical product lifecycle or this concept of vein-to-vein. That involves consideration of the stresses involved, especially when transporting either a source material or the finished product once it leaves a manufacturing site to a clinical site. Both ISCT and BioLife Solutions are supporting further discussions on this topic. ISCT is a great forum for bringing together knowledgeable experts and those who can implement evolving best practices.

Is there anything else that you’d like to share with our readers about the meeting? “ISCT has grown hand-in-hand with regenerative medicines. The Annual Meeting is always a great learning experience. I strongly encourage anyone interested in cell therapies and regenerative medicine to come out and join us in Las Vegas. I’m sure that you’ll enjoy what you learn. I’m sure you’ll meet a lot of great new folks and network with key opinion leaders in the field of regenerative medicine. And probably have a good time — it is Las Vegas.”

Commercialization Committee Chair

Miguel Forte is Chief Operating Officer at TxCell (Valbonne–Sophia Antipolis, France) and chairs ISCT’s Commercialization Committee.

Can you talk about ISCT’s involvement in the commercialization of cell therapy? “ISCT includes academic and industry members who intend to bring cell therapy products to patients. The Society addresses elements such as product development, clinical evidence, and the generation of information for regulatory discussions. Other topics include market access, pricing reimbursement, and the adoption of new products by physicians and patients.

“We collect knowledge and consider how it affects activities performed in both academia and industry in terms of taking products from preclinical research to the market. In addition, we consciously acknowledge that it is important to understand the end users, prescribing physicians, and patients who will need to use those products. We also need to ensure the right evidence, education, and use of those products for the right patients. That all leads to the fact that many cell therapies are personalized medicines tailored to a given patient either because the therapy is autologous or because it treats a specific disease. Having that end use and that end adoption in mind is a particular aim of the society.

“Within ISCT is a Commercialization Committee that takes those discussions into consideration to look at all the steps, challenges, and opportunities to move a product (or a candidate product) from preclinical research all the way to patients. Like the Society at large, that committee (which I chair) consists of academics — people in academia who already are developing cell therapy products — as well as representatives from small, medium, and large industry organizations that have products or a committed interest in cell therapies.

“The main committee meeting combines all members, but then within the group there is a series of subcommittees that focus on particular aspects. One of those is the Product and Process Development Subcommittee, which is particularly focused on developing controls, robustness of manufacturing, comparability of manufacturing, and all the elements that go into auxiliary products and elements.

“Another subgroup takes into consideration all the aspects that go into the cost of goods that relate to manufacturing or process development as well as business models for new products, which we will support and ensure that there is value for all key stakeholders. It is essential that these products are adopted and adequately reimbursed and paid in the market. So there is a subcommittee that talks about market access, in particular the elements of reimbursement.”

Strategies for Commercialization Track Speakers

Ancillary Materials
Claudia Zylberberg, PhD (Akron Biotech, USA)
Lynn Csontos (STEMCELL Technologies Inc., Canada)
Jennifer Solomon, PhD (STEMCELL Technologies Inc.)
Joanne Kurtzberg, MD (Duke University Medical Center, USA)
Jiwen Zhang (GE Healthcare)
Brian Newsom (Thermo Fisher Scientific, Inc., USA)
Potency and Safety Assay Development
Christopher Bravery (Consultant in Advanced Biologicals Ltd., UK)
Claudia Zylberberg, PhD (Akron Biotech, USA)
Robert Perry (Athersys, Inc., USA)
Jon Rowley, PhD (RoosterBio Inc., USA)

Reimbursement
Richard Maziarz MD (Oregon Health and Science University, USA)
Stephanie Farnia, MPH (National Marrow Donor Program, USA)
Panos Kefalas (Cell Therapy Catapult, UK)
Dawn Driscoll, PhD (DCi Biotech, USA)
Optimizing Costs of Cold Supply Chain
William Milligan (Steminent Biotherapeutics Inc., Canada)
Kevin O’Donnell (BioLife Solutions, Inc., USA)
Brian Murphy (Celgene, USA)
Michael Trocchia (Novartis Pharmaceuticals Corp., USA)

Financing and Investment
Reni Benjamin, PhD (H.C. Wainwright & Co., USA)
Joshua Schimmer, PhD (Piper Jaffray, USA)
Jason Kolbert (Maxim Group, USA)
Appropriate Valuation of Transformative Therapies under Development
Michael May, PhD (Centre for Commercialization of Regenerative Medicine, Canada)
Phil Vanek (GE Healthcare, USA)
Geoff MacKay (Proteus Ventures, USA)
Stem Cell Bioprocessing/Manufacturing Technologies
Knut Niss, PhD (Novartis Pharmaceuticals Corp., USA)
Steve Oh, PhD (Bioprocessing Technology Institute, Singapore)
Anthony Davies (Dark Horse Consulting, USA)
Sunil Mehta (kSep Systems, USA)

Would you talk about the committee’s current work? “One element is to make sure that we have the right way to manufacture cell therapies and regenerative medicine. That is particularly relevant for readers of BioProcess International who are working on bioprocessing these products. Another focus of the committee is the regulatory environment and changes in it. The FDA, the EMA, and the PMDA in Japan all have issued guidances on cell therapy products. Some countries are quite aggressive in supporting activities in this field, with an understanding that this field has an opportunity to address some unmet medical needs. Because cellular therapy is a new field, it present new challenges and new requirements of knowledge and expertise. Particularly in Japan, there has been a lot of fostering of cell therapy products recently, with some changes in regulation. In Europe, for quite a while the Committee for Advanced Therapies (CAT) has issued guidance in assessing products in this space and is very committed to partner with industry and contribute to discussions. These professionals will be at our annual meeting.

“The committee interacts with its regulatory agencies and issues opinions on requests for comment on published guidance. Within the society are regulatory affairs groups. Within ISCT are groups that focus on regulatory activities for North America, Europe, and Australia/New Zealand. And they closely interact with the Commercialization Committee for addressing regulatory hurdles and opportunities on the path of taking a product to market.

The Commercialization Committee makes sure to partner with other organizations that have an interest in cell therapies and regenerative medicines — for example, the Alliance for Regenerative Medicine (ARM) and other scientific societies — to ensure that we join efforts, align ideas, and share experiences to foster the field. One particular aspect that is very dear to ISCT in general and for the Commercialization Committee is interaction with patient associations. We want to educate the public about what cell therapies are. That’s the perspective of ISCT members in general.

“The Commercialization Committee has a particular focus on ensuring that we understand patient expectations and patient needs. We aim to understand how patients can offer input on the development of a product because, in the end, that product will address their medical needs. So that end-user perspective always should affect what we do. We always should be aware of that perspective when we are developing products. As the Commercialization Committee, we are very keen on making sure that we factor that element into our progress and discussions.”

I understand you have a background involving T cells in the commercial setting. Can you share information about your company that relates to the work of ISCT? “At TxCell, we are developing an antigen-specific autologous T regulatory-cell therapy for autoimmune diseases. Currently in clinical studies, are treatments for Crohn’s disease and noninfectious uveitis. We take blood from patients, adjudicate the cells, freeze them, and then readminister them. Then through multiple mechanisms of action, those cells target the site of inflammation and deliver in presence of the antigen and an antiinflammatory stimulus. This is an opportunity because both of those diseases currently are served by some treatments, but a large proportion of the patients eventually become refractory to those treatments and thus have an unmet medical need.

“I believe there are three critical success factors that should be present in any company in this field: first is a significant focus on process development and manufacturing, making it robust, transferrable, and with adequate cost of goods. The second is to make sure that you have a clear vision about your product and how it will be used in the market so that you can address market needs. That means understanding what patients and physicians want, what the competitive space is, and what your target product profile looks like. Having a vision of your product and how it’s going to be used is essential for the success. Finally, make sure that you have relentless project management, which ensures that you always keep your focus and always work toward your target product profile. These are the challenges that we all are facing. Being together at the ISCT meeting allows us to exchange experiences, learn about new opportunities, and participate in networking and business developments that benefit all of us.”

Why would BioProcess International readers be interested in attending the ISCT meeting? “The meeting is very useful for anyone interested in any aspect of cell therapy. The current biggest challenge in cell therapy is to make sure that we can consistently develop and manufacture the products while holding discussions with suppliers and end users. Meeting current challenges to advance manufacturing is a key element of the discussion.

“There will be opportunities to assist and participate in sessions that relate to manufacturing and process development topics. These are taken over on generation of clinical evidence, the regulatory impact, and the broad regulatory context, as well as aspects of market access. So in terms of scientific content, participants will have an opportunity to cover all aspects, particularly process development and manufacturing within the cell therapy space. Discussions are guided with strong academic participation and strong industry and multiple-level participation. So the meeting provides a broad range of views.

“In addition, a number of suppliers and support organizations will be present for an exhibition. Attendees have the opportunity to see multiple booths where business is being done to promote process development and product manufacturing, as well as the other services. We already have a well-furnished exhibition area that I expect to be very fruitful and offer many opportunities for interaction.

“Of additional interest is the opportunity for attendees to view posters and attend original presentations of ongoing work. Those provide opportunities to see some emerging efforts from industry and definitely from academia across the world.

“With all of that are multiple opportunities for interaction, networking, and advancing business development. You will see professionals from all over the world coming for the broad presentations, the exhibitions, the oral presentations, and abstracts and posters. It’s a huge opportunity for networking and business development.

“The ISCT meeting is an opportunity to pick and choose updates on specific areas of scientific and industrial interest. And it is an opportunity to interact with leaders in the field and network in business development.”

Process and Product Development Committee Roundtable

Ohad Karnieli, PhD, MBA is vice president of technology and manufacturing at Pluristem Therapeutics, Inc. (Haifa, Israel). He is chair of the Process and Product Development (PPD) track subcommittee and will lead panel discussions with experts from the industry, including developers and users, on Thursday, 28 May 2015, 7:30–9:00 am.

What are the main topics in this session? “The topics were chosen by the PPD Subcommittee in a pool out of nine lead topics that were suggested by the group. The three main topics are serum-free media for cell therapy (main components, challenges, advantages and tips for cell developers), bioreactors (introduction to bioreactor development and moving from static cultures to bioreactors), and particulates in bioprocessing bags and packaging.

“The panel includes committee members and experts from GIBCO (Thermo Fisher), GE Healthcare, Biological Industries, Irvine Scientific, STEMCELL Technologies, and Charter Medical. We will also have representatives from Wake Forest Institute for Regenerative Medicine, Catapult, Pluristem, Pfizer, Novartis, Mesoblast, and BTI. Regenerative medicine regulatory scientist and consultant Christopher Bravery will be a regulatory representative.”

What are you most excited about hearing and discussing at this year’s ISCT meeting? “The PPD session in its new format aims to provide a platform for people to ask experts about the issues that are of interest. The panel represents both vendors and users. Their discussion should allow real information sharing to hopefully provide meeting attendees with real practical value.”

Specific to your session topic, what challenges are scientists and companies facing? How does ISCT help? “ISCT provides a unique platform with its Commercialization Committee and the PPD Subcommittee for bias-free discussion of white papers and talks/panels on issues that are of interest to all the industry. Members represent tools and materials developers as well as cell therapy developers. That allows for real discussion and a community knowledge base to assist all stakeholders in building this new and challenging industry.”

Is there anything else about your session that you would like to share with our readers? “Come with questions for the experts!”

Ancillary Materials Session

Lynn Csontos is associate director of quality assurance and regulatory affairs at STEMCELL Technologies (Vancouver, Canada). She will co-chair this session of the commercialization track with Claudia Zylberberg, PhD, founder, CEO, and president of Akron Biotechnology in Boca Raton, Florida, on Thursday, 28 May 2105, 11:15 am – 12:30 pm.

What are the main topics in this session? “We will discuss the ever-changing global regulatory landscape for ancillary materials used in cellular therapy applications. Questions to address include the following: Who is accountable for qualification of ancillary materials? What does a risked-based qualification plan include for an  ancillary material? Who pays for quality? What is the effect of quality on the cost of goods? When is the right time to switch from research grade to more compliant materials? What are the differences between in-process ancillary materials and final-formulation raw materials? Perspectives on all of those topics will come from academic, translational, and clinical research (from biotechnology and pharmaceutical companies).”

What are you most excited about hearing and discussing at this year’s ISCT meeting? “More companies are populating the space, which confirms that the cellular therapy market is growing. As such, we continue to see more advances in the clinical pathway for these therapies. Therefore, challenges regarding cost of manufacturing, business models, and reimbursement are becoming critical pieces of the puzzle.

“Because the approach to cellular therapy is so decentralized in the market, we are also excited to hear about progress being made, as well as the struggles faced within the translational and clinical space. This allows us to understand possible trends and advances and apply lessons learned to emerging policies.”

Specific to your session topic, what challenges are scientists and companies facing? How does ISCT help with these challenges? “One challenge that scientists and companies face related to ancillary materials is a lack of standardization around definitions that dictate qualification approaches. For example, how do you classify animal-component–free (ACF) materials? The definition of ACF varies widely throughout the world. How that complex term is defined will dictate the level of qualification that will be appropriate for this type of ancillary material.

“When comparing cellular therapy manufacturing with classical bioprocessing of biologics, there are fewer process steps to ‘purify’ the product. Therefore, the quality, traceability, and risk assessment of the components become priorities. Limitations in analytical characterization of biological materials can prove to be a barrier that will require a risk-based approach to qualification plans of ancillary materials.

“ISCT acts as facilitator of novel ideas that blossom from academia and industry alike. It provides centralized information to assist the industry in fulfilling the promise of this new therapeutic market.”

Is there anything else about your session that you would like to share with our readers? “Cellular and regenerative therapies are rapidly changing fields. Attending ISCT allows companies and scientists to be a part of the ever-changing conversation that will shape future businesses and regulatory policies. Although our session is focused on ancillary materials, ISCT does an outstanding job of bringing light to the entire product life cycle, from breakthrough research and cutting-edge scale-up manufacturing, all the way to reimbursement strategies.

“If you are in the early stages of your clinical pathway or if you are well into the clinical trial process and ready to commercialize your product, then this session will enlighten you with new information and tools to address the compliance and costs associated with raw materials.”

Potency and Safety Assay Development Session

Christopher Bravery, PhD, is a regenerative‑medicine regulatory scientist and consultant on advanced biologics (London, UK). He will chair the Potency and Safety Assay Development session on Thursday, 28 May 2015, 4:00–5:30 pm.

What are the main topics in this session? This session is less about individual hot topics and more about the continued grind of developing appropriate assays that allow cell therapies to be measured for activity and/or safety. It is an ongoing evolution of knowledge that we keep building upon brick by brick as various clinical therapies progress through development. What we want to accomplish is to share knowledge and perspectives on assay development (and lessons learned) compared with last year or the year before. One difficulty in this topic is that there might be progress in the knowledge base of assay development, but the associated methods may be proprietary or not publicly disclosed.”

What are you most excited about hearing and discussing at this year’s ISCT meeting? “I am looking forward to what folks have seen as the progressions of potency or safety assay development. Because a number of cell therapies might have mechanisms of action involving secreted factors, there is an ongoing discussion about how to effectively identify those factors and assess their functional properties. Which assays are appropriate when there might be different clinical indications for a single manufactured cell therapy product? With the recent rise in advancement of autologous cellular immunotherapies, developing appropriate potency and safety assays for patient‑specific indications has its own set of challenges. The chair and speakers in this session work with a number of groups in the cell therapy field. I look forward to their sharing tidbits of the knowledge that they have picked up from their various collaborations and from their own work.”

Specific to your session topic, what challenges are scientists and companies facing? How does ISCT help scientists and businesses with these challenges? “I think one challenge is that development of these assays is still evolving in conjunction with manufacturing advancements related to scale‐up, scale‐out, and automation. To assess the effectiveness of manufacturing advancements, you would perform comparability tests using methods such as potency assays. But those potency assays are still maturing. Those multiple variables create some risks, but the good thing about our cell-therapy community and ISCT is the information sharing, collaboration, and working groups pooling together knowledge and talent. That can lead to progress such as the ISCT white papers on this topic published in Cytotherapy.”

Is there anything else about your session that you would like to share with our readers? “I anticipate that some members of the session audience will quickly jump back into their own laboratories after hearing what is shared in the session and work to progress their own potency and/or safety assay development.”

Reimbursement Session

Richard Maziarz, MD, is a physician at Oregon Health and Sciences University specializing in treating bone marrow transplantation and blood cancers. His research focuses on how the immune system recovers after a bone marrow transplant and how it works to fight cancer. He will chair the Reimbursement session on Friday, 29 May 2015, 8:00-9:00 am.

What are the major topics in your session? “This session will reflect what the field considers to be the main challenges and opportunities for reimbursement and adoption of cell therapies. We conducted a survey to identify the topics on which this subcommittee of the Commercialization Committee should focus so that ISCT can deliver focused value on this topic.

“The main topic relates to geographic differences in challenges and opportunities for cell therapy reimbursement. This field is moving quickly, and new technologies (such as engineered cell therapies) are surfacing. Regulatory issues are evolving, particularly in some countries, such as Japan.

“Another important topic concerns the cost of goods and corresponding business models for traditional and innovative risk-sharing reimbursement strategies.

Finally, a very relevant topic, particularly in the United States, pertains to discussion about cell therapy as a service, procedure, or a medicinal product and the implications for access to these therapies. That will also be part of the session.”

What are you most excited about hearing and discussing at this year’s ISCT meeting? “What excites me about the discussion this year will be how emerging new approaches in cell therapy will gain adoption in the market. Some new approaches are bringing significant value to several unmet–medical-need challenges but still may face important hurdles in pricing and reimbursement, potentially hampering patient access.

“These hurdles reflect the topics I mentioned above and relate to the need to consider new models and approaches for market access and reimbursement of innovative and valuable therapies. This should raise passionate discussion.”

Specific to your session topic, what challenges are scientists and companies facing? How does ISCT help scientists and businesses with these challenges? “Scientists and clinical developers must gear their efforts to documenting the safety, tolerability, and efficacy of their cell-therapy product candidates. This should be done with a clear target product profile (TPP) that aims to maximize the value provided to patients with minimal hurdles to adoption (e.g., compatibility to current clinical practice). The important challenge they face — which is critical for success — is to ensure that all activities include that vision from very early in the development process and the corresponding generation of relevant evidence on added value to enable pricing and reimbursement discussions.

“ISCT members and speakers at the annual meeting represent a pool of extensive experience with these challenges. There already have been some successes. The meeting will facilitate learning and discussion opportunities, sometimes with specific examples, helping those who have to lead and deliver in this area to be better equipped for success. In addition to the annual meeting, the ISCT (through the Commercialization Committee and its subcommittees, in this particular case the Reimbursement Subcommittee) represent a very productive forum for networking and broadening learning for its membership.”

Is there anything else about your session that you would like to share with our readers? “The session will address a progressively more relevant topic as new product candidates approach market access challenges. I expect good attendance, and we will work toward ensuring an interactive part of the session with intense discussion between the presenting experts and the audience. It should be an educating and enjoyable experience.”

Optimizing Costs of Cold Supply Chain Session

William Milligan (Steminent Biotherapeutics Inc.) will chair the session on “Cell Therapy Biologistics: Optimizing Costs of Cold Supply Chain” on Friday, 29 May 2015, 11:15 am – 12:30 pm.

What are the main topics in your session? “To address cost-effective cold‑chain planning and execution, we wanted a speaker with immense experience in effectively solving challenges for cryopreserved products requiring ultra cold‑chain distribution. We wanted a speaker with depth in good cold-chain distribution practices and knowledge about key considerations for quality and cost management. Then to complement this strategic framework, we wanted speakers who have specific experience in managing and addressing these challenges with their own cell therapy products.

What are you most excited about hearing and discussing? “I am excited about discussing successes with innovative solutions from leading cell therapy developers about managing challenges related to advancing cell product candidates that can promise more sustainable business models. Specifically in this session, I look forward to hearing leading‑edge insights and ideas for managing the cost of cold-chain distribution.

“It is exciting to hear about experiences and different approaches that can potentially lead to solutions for some of your most daunting product development and distribution challenges.”

Specific to your session topic, what challenges are scientists and companies facing? How does ISCT help scientists and businesses with these challenges? “Ensuring that you can deliver a consistent cellular therapy product to each patient is a much greater challenge than it is with small and large molecules. Cryopreserved products require cold- or ultracold-chain distribution options, which tend to be much more expensive than traditional shipping. Substantial losses can be incurred if a product thaws prematurely, gets contaminated, cracks, or is delayed in receiving. So maintaining the quality of distribution is essential. Therefore, although the cost of cold-chain distribution will need to be managed aggressively using best practices, you must balance that with regulatory requirements compliance, speed, and convenience of effectively delivering products globally to patients in need.

“Through annual meetings, regional meetings, webinars, white papers, the BEACON suite of navigational tools, and working committees, ISCT provides the framework, venues, and mission to ensure that industry champions are working together to connect, collaborate, and advance policies and best practices that support the cell therapy R&D community to manage issues such as cold-chain distribution. That ensures that the valuable knowledge gained by members of the community can be rapidly shared and disseminated to catalyze industry momentum.”

Is there anything else about your session that you would like to share with our readers? “Optimizing the costs of cold-chain distribution for cell therapy products is one key aspect in the final steps of the “needle-to-needle” continuum of value creation that defines cellular therapy. However, planning for achieving optimal cold‑chain distribution costs and optimizing those costs at every step of a cell therapy product’s development pathway — from donor to patient — should be addressed very early in the planning of each new cell therapy target product profile. Decisions made early in development often affect your costs for delivering a product downstream. So this session will provide insight to cell therapy developers about how they can manage

 

distribution costs by considering ways to reduce distribution complexity for their products earlier in design and development.”

Financing and Investment Session

Reni Benjamin, PhD, is the managing director and equity research analyst at H.C. Wainwright & Co. (New York, NY). He will chair the “Financing and Investment” session on Friday, 29 May 2015, 4:00–5:30 pm.

What are the main topics in your session? “The biotechnology index has been outpacing the vast majority of the market for the past several years. Last year was also a banner year for the cell therapy space, with sales of approved cell therapy products combined totaling >$1 billion and with more cell therapies advancing through various stages of clinical development. Our panel will focus on what’s hot and not in cell therapy and provide insights from several Wall Street analysts as to how investors view this exciting and novel therapeutic approach. Specifically, the areas of gene therapy, CAR T cells, and regenerative medicine will be discussed, with clinical data generated to date and the potential to permanently ‘cure’ a wide variety of disease indications.”

Specific to your session topic, what challenges are scientists and companies facing? “We believe that an investor’s perspective (what drives the investment process) should benefit scientists and companies seeking funding to move their therapeutics forward in the clinic and toward marketing approval.”

Is there anything else about your session that you would like to share with our readers? “Additional points that will be discussed include Wall Street’s perspective on clinical efficacy seen to date, the best and worst business models, pricing strategies and what we may have learned from the Dendreon debacle, long- and short-term views for each area, and risks and potential pitfalls.”

Manufacturing Technologies Session

Knut Niss, PhD, is the senior technical project leader at Novartis Pharmaceuticals Corp. (Boston, MA). He will chair the last commercialization track session, “Stem Cell Bioprocessing/Manufacturing Technologies,” on Friday, 29 May 2015, 1:45–3:15 pm.

What are the major topics in your session? “Now that the cell therapy field is maturing and entering various pivotal trials, manufacturing technologies are coming to the forefront of attention. Cost concerns and unique technology requirements will play a major role in developing therapies. This session will provide an overview of current developments.”

What are you most excited about hearing and discussing at this year’s ISCT meeting? “I look forward to hearing more about the progress of therapies going into phase 3 studies as well as learning more about new emerging therapy concepts.”

Specific to your session topic, what challenges are scientists and companies facing? How does ISCT help scientists and businesses with these challenges? “To date, only a few cell‑therapy technology platforms have been developed. Most processes use platforms designed for the biologics field. With their unique requirements, cell therapies will benefit from cell‑therapy–specific technology platforms. ISCT brings together a diverse group from academia as well as therapeutic-focused and technology-focused companies. That diversity fosters discussions around current and future needs and as such is facilitating advancement of the field.”

Is there anything else about your session that you would like to share with our readers? “This session will provide different perspectives on issues of process development and manufacturing technologies for cell therapies. The speakers were selected based on their in-depth knowledge of the field as well as their outstanding experience.” •

Maribel Rios is managing editor of BioProcess International, mrios@bioprocessintl.com.

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