CAR-T Therapy in Haematological Malignancy: Early Stage Outlook: KOL Insight [2017]

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Publication Date:
June 2017
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Will CAR-T cell therapies shape the treatment of blood cancers in the next decade?

Anti-CD19 CAR-T cell therapies have demonstrated unprecedented response rates across patient subgroups of acute lymphocytic leukaemia (ALL) and non-Hodgkin lymphoma (NHL) with high unmet needs. How will the impressive efficacy and safety of these therapies pan out in the long-term?  Key opinion leaders (KOLs) explore the clinical challenges and opportunities that face these exciting treatments across multiple haematological cancer settings. Experts discuss the logistical, pricing, reimbursement and market access (PR&MA) hurdles facing these novel therapies and how they can be overcome. Learn how KOLs see the future CAR-T therapy market evolving, and how they expect developers to successfully differentiate their CAR-T therapies in KOL Insight: CAR-T cell Therapy in Haematological Malignancy. Eight US KOLs also provide their candid views on the potential for next-generation approaches, allogeneic CAR-T therapies and other novel strategies

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Top Takeaways

  • Impressive responses achieved with anti-CD19 CAR-T but how important is long-term durability? Efficacy is unprecedented in relapsed/refractory patients with high unmet needs but how do KOLs view the long-term durability and persistence of CAR-T therapies?
  • Which diseases show most benefit to CAR-T treatment and why? Anti-CD19 CAR-T cells have been investigated in acute lymphocytic leukaemia (ALL), chronic lymphocytic leukaemia (CLL), non-Hodgkin lymphoma (NHL). What are the key opportunities facing CAR-T in each disease and which ones are KOLs most excited about?
  • What needs to be prioritised in terms of safety? Cytokine release syndrome (CRS) and neurotoxicity are associated with CAR-T treatment. How concerned are KOLs about safety of these treatments and how will side effects be managed going forward?
  • What are the main logistical challenges facing autologous CAR-T cell therapies and how can they be overcome? Find out what logistical hurdles face autologous CAR-T cell therapies and what KOLs think can be done to overcome them.
  • Pricing, reimbursement and market access of CAR-T cell therapy; what are KOL’s opinions? What key market barriers will CAR-T therapies face and will payers be prepared to fund them?
  • Do next-generation CAR-T cell approaches ignite KOL excitement? Allogeneic ‘off-the-shelf’ CAR-T cells and CAR-T cells incorporating safety switches are all in the early-stage pipeline. Which approach do KOLs think offers most promise and why?
  • How do CAR-T cell therapies compare to gold-standard transplantation? Haematopoietic stem cell transplants (HSCT) are a cornerstone of treatment in haematological cancer. What parallels can KOLs draw from HSCT and do experts envisage CAR-T cell therapies eventually replacing transplantation?
  • Which key players are best positioned for commercial success and why? Competition is mounting between Novartis, Kite Pharma and Juno Therapeutics but which key player is best positioned to carry CAR-T cell therapies forward according to the KOLs?
  • How can companies differentiate themselves to market CAR-T therapies? What do CAR-T manufacturers need to do in order to bring these therapies to market in a competitive landscape?
  • How will CAR-T cell therapies impact the future treatment algorithms of haematological cancers in 10 to 15 years? Can CAR-T therapies fulfil unmet needs and secure a niche in future treatment landscapes?


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“The response is impressive enough that I see [CAR-T therapies] being around in 10 to 15 years. Exactly where to use them and when to use them is still unknown. I see them being used for lymphoma, ALL, and in multiple myeloma, we'll have to see.”
US Key Opinion Leader

“The responses for ALL are unprecedented. Somewhere between 80 to 90 percent of patients achieve a complete remission, and that's across different CAR programmes, and so it really does tell you it's a feature of the technology and not some secret at one company or one institution over another.”
US Key Opinion Leader

Sample of therapies covered

Pipeline Therapies

  • tisagenlecleucel-T (CTL019; Novartis)
  • axicabtagene ciloleucel (KTE-019; Kite Pharma)
  • JCAR014 (Juno Therapeutics)
  • JCAR017 (Juno Therapeutics)
  • JCAR018 (Juno Therapeutics)
  • UCARTs (Cellectis/Pfizer/Servier)
  • Plus 4 more - Download the complete list here >

Sample of KOLs interviewed

KOLs from North America

  • Professor Michael R. Bishop, MD. Professor of Medicine and Director of Hematopoietic Stem Cell Transplantation Program at the University of Chicago.
  • Dr. Marco L. Davila, MD, PhD. Medical oncologist in the Department of Blood and Marrow Transplantation at the Moffitt Cancer Center, Florida.
  • Dr. Noelle Frey, MD, MSCE. Assistant Professor of Medicine at the Hospital of the University of Pennsylvania.
  • Plus 5 more - Download the complete list here >

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