血液恶性肿瘤CAR-T疗法 - 早期阶段展望:KOL (关键意见领袖) 见解

CAR-T Therapy in Haematological Malignancy: Early Stage Outlook: KOL Insight [2018]

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出版日期 内容资讯 英文
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血液恶性肿瘤CAR-T疗法 - 早期阶段展望:KOL (关键意见领袖) 见解 CAR-T Therapy in Haematological Malignancy: Early Stage Outlook: KOL Insight [2018]
出版日期: 2018年04月01日内容资讯: 英文

本报告关注早期阶段的血液恶性肿瘤CAR-T疗法,汇整8位美国关键意见领袖 (KOL) 各种见解。


  • Pipeline药物
    • tisagenlecleucel-T (CTL019:Novartis)
    • axicabtagene ciloleucel (KTE-019:Kite Pharma)
    • JCAR014 (Juno Therapeutics)
    • JCAR017 (Juno Therapeutics)
    • JCAR018 (Juno Therapeutics)
    • UCARTs (Cellectis/Pfizer/Cellectis)、其他


  • 虽然在抗CD19 CAR-T获得十分良好的反应,但长期耐久性有多重要?
    • 在具有高度未满足需求的复发/难治性病患中,展现前所未有的疗效
    • KOL对CAR-T疗法长期耐久性和持续性见解为何?
  • CAR-T疗法优点最多的疾病及其理由?
    • 抗CD19 CAR-T细胞:已在急性淋巴性白血病 (ALL)、慢性淋巴白血病 (CLL)、非何杰金氏淋巴瘤 (NHL) 上进行研究
    • 各疾病的CAR-T主要市场机会为何?
    • KOL最感兴趣的市场机会?
  • 在安全面需优先考虑什么?
    • 细胞激素释放症候群 (CRS)和神经毒性
    • KOL对CAR-T疗法安全性的担忧为何?
    • 副作用管理展望?
  • 自体CAR-T细胞疗法主要理论性课题?
    • 如何克服?
    • 自体CAR-T细胞疗法理论性阻碍和KOL对克服方法的看法
  • CAR-T细胞疗法药价、核付、市场进入
    • KOL看法?
    • CAR-T疗法主要市场阻碍为何?保险者在资金面是否已准备好?
  • KOL对次世代CAR-T细胞方法的关注程度?
    • 同种「现成」CAR-T细胞和具安全开关的CAR-T细胞:所有Pipeline早期阶段
    • KOL认为最具潜力的方法及其理由?
  • CAR-T细胞疗法和代表性移植术比较
    • 造血干细胞移植 (HSCT):血液肿疡治疗基础
    • KOL所见和HSCT的类似点?
    • 专家是否认为CAR-T细胞疗法可取代移植?
  • 哪些企业最接近成功商业化?其理由为何?
    • Novartis・Kite Pharma・Juno Therapeutics间竞争日益激烈
    • 被定位为推动CAR-T细胞疗法前进的企业?
  • 各企业如何进行自家公司的差异化?
    • 为了在竞争剧烈环境中将其推向市场,CAR-T疗法制造商需要做什么?
  • CAR-T细胞疗法10-15年内对血液肿疡治疗演算法的影响为何?
    • CAR-T细胞疗法可否满足未满足需求,确保未来治疗环境的利基地位?



How are CAR-T cell therapies revolutionising treatment of haematological cancer?

Last year witnessed the FDA approval of the first two anti-CD19 CAR-T cell therapies in two types of blood cancer. Find out how these novel treatments are impacting treatment choices so far and how their introduction is progressing in the US. Eight of the world's leading key opinion leaders (KOLs) from the US and Europe provide their views on the opportunities and challenges that face these exciting treatments across multiple haematological cancer settings and discuss the potential logistical, pricing, reimbursement and market access (PR&MA) hurdles, 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 Therapies in Haematological Malignancy. Experts also provide their candid views on next-generation CAR-T approaches and their potential adoption in other blood cancers.

Take a tour of the report now

The table of contents >

The key business questions answered >

The key KOL quotes >

See the therapies covered >

Find out who the 3 EU & 5 US KOLs are >

Review an extract from the report - 1 drug profile >

Top takeaways

  • Kymriah and Yescarta are now FDA-approved but how are they being prescribed in the post-approval setting? Experts reveal how the roll out of CAR-T therapies in the US is progressing so far and what factors impact their prescribing decisions.
  • Which anti-CD19 CAR-T therapy is likely to emerge on top in terms of efficacy and safety? Find out how KOLs compare and contrast the six-month efficacy and safety data reported for Kymriah, Yescarta and JCAR017 in the DLBCL setting.
  • Will CAR-T therapies reach the European market in 2018? European KOLs weigh up the prospects of CAR-T therapy launching in Europe. What are the particular challenges facing CAR-T in the European market?
  • Pricing, reimbursement and market access of CAR-T cell therapy; what are KOLs' opinions? What market barriers are CAR-T therapies facing in the US and Europe and are payers prepared to fund them?
  • Which other disease areas hold the most promise for CAR-T therapy? What are the key opportunities for CAR-T in other disease areas (multiple myeloma, lymphoma and chronic lymphocytic leukaemia [CLL]) and which ones are KOLs most excited about?
  • Which next-generation CAR-T cell approaches fuel KOL excitement? Allogeneic 'off-the-shelf' approaches, dual-targeting CAR-T and combination strategies are in the early-stage pipeline. Which approach do KOLs think offers the most promise and why?
  • Can CAR-T cell therapies compete with gold-standard transplantation? Do experts envisage CAR-T cell therapies eventually sidelining transplantation and what do they expect from key ongoing studies such as Kite/Gilead's ZUMA-7?
  • What are the main clinical and commercial differentiators for CAR-T therapy going forward? Find out if KOLs rank efficacy, safety, ease of use or cost as most important. What do CAR-T manufacturers need to do in order to compete effectively in this landscape?
  • How will CAR-T cell therapies impact future treatment algorithms of haematological cancers in 10 to 15 years? Can CAR-T therapies secure a niche in front-line, consolidation or first-relapse treatment settings?


"There's absolutely no reason that CAR-T should not be at least second-line for DLBCL; patients who relapse with large cell lymphoma are incurable and this is probably the easier and safer therapy." US Key Opinion Leader

"There is no doubt about the fact that EMA will approve [CAR-T]. Judging from the data plus the experience, I think there is no doubt that both products will be approved." EU Key Opinion Leader

"[CAR-T] will be used anywhere between 10 and 30 percent of patients in general with these diseases. I would not be surprised if it's 50 percent or more." EU Key Opinion Leader

Sample of therapies covered

Marketed Therapies

  • Kymriah (tisagenlecleucel-T/CTL019; Novartis)
  • Yescarta (axicabtagene ciloleucel/KTE-019; Kite Pharma/Gilead)

Key Pipeline Therapies

  • JCAR017 (lisocabtagene maraleucel; Juno Therapeutics/Celgene)
  • JCAR014 (Juno Therapeutics/Celgene)
  • JCAR018 (Juno Therapeutics/Celgene)
  • UCARTs (Cellectis/Servier/Pfizer/Allogene)
  • bb2121 (bluebird bio/Celgene)
  • bb21217 (bluebird bio/Celgene)
  • CTL119 (Novartis)
  • AUTO2 (Autolus)
  • AUTO3 (Autolus)
  • CYAD-01 (Celyad)
  • Sleeping Beauty CAR-T cell programme (Ziopharm)
  • Plus other next-generation CAR-T therapy approaches

KOLs interviewed

KOLs from North America

  • Dr. Terry J. Fry MD, Director of Cancer Immunotherapy and co-Director of Human Immunology and Immunotherapy Initiative at the University of Colorado School of Medicine, Denver, Colorado, US
  • Dr. Partow Kebriaei MD, Professor, Department of Stem Cell Transplantation, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas
  • Dr. Frederick Locke MD, Medical Oncologist and Translational Researcher in the Department of Blood and Marrow Transplant and Cellular Immunotherapy at the Moffitt Cancer Center, Tampa, Florida, US
  • Dr. Craig S. Sauter MD, Hematologic Oncologist at the Memorial Sloan Kettering Cancer Center (MSKCC), New York, US
  • One Anonymous US KOL (MD, PhD) leading expert in CAR-T cell research and transplantation

KOLs from Europe

  • Dr. Reuben Benjamin MBBS MRCP FRCPath PhD, Consultant Haematologist and CAR-T cell specialist at King's Hospital London, London, UK
  • Professor Dr. Ulrich Jager MD PhD, Professor of Hematology, Head of the Clinical Department of Hematology and Hemostasis, Medical University of Vienna, Austria
  • Professor Gilles Salles MD PhD, Chair of the Lymphoma Hub, Head of the Haematology Department of the Lyon Sud University, Lyon, France

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Table of Contents

1. Executive Summary

2. Research Objectives

3. Research Focus

  • 3.1 What are CAR-T cell therapies?
  • 3.2 Key Indications
  • 3.3 Report Focus

4. Clinical Opportunities and Challenges for CAR-T Therapies

  • 4.1 Efficacy of Autologous anti-CD19 CAR-T therapies
  • 4.2 Safety and Toxicity Management of Autologous anti-CD19 CAR-T Therapies

5. Commercial Opportunities and Challenges for CAR-T Therapies

  • 5.1 Logistics of CAR-T cell therapies
  • 5.2 CAR-T Therapies and Transplantation
  • 5.3 Pricing, Reimbursement and Market Access of CAR-T cell therapies

6. Next-Generation CAR-T Programmes

  • 6.1 Disease Insights: Key Insights for CAR-T Therapies in Other Haematological Malignancies
  • 6.2 CAR-T therapies in multiple myeloma
  • 6.3 CAR-T therapies in other lymphomas, CLL and AML

7. Long-Term Outlook for CAR-T Cell Therapies

  • 7.1 Key insights summary

8. Conclusion

9. Appendix