血液惡性腫瘤CAR-T療法 - 早期階段展望:KOL (關鍵意見領袖) 見解

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

出版商 FirstWord 商品編碼 520363
出版日期 內容資訊 英文
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血液惡性腫瘤CAR-T療法 - 早期階段展望:KOL (關鍵意見領袖) 見解 CAR-T Therapy in Haematological Malignancy: Early Stage Outlook: KOL Insight [2018]
出版日期: 2018年04月01日內容資訊: 英文

本報告關注早期階段的血液惡性腫瘤CAR-T療法,彙整8位美國關鍵意見領袖 (KOL) 各種見解。


  • Pipeline藥物
    • 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


  • 雖然在抗CD19 CAR-T獲得十分良好的反應,但長期耐久性有多重要?
    • 在具有高度未滿足需求的復發/難治性病患中,展現前所未有的療效
    • KOL對CAR-T療法長期耐久性和持續性見解為何?
  • CAR-T療法優點最多的疾病及其理由?
    • 抗CD19 CAR-T細胞:已在急性淋巴性白血病 (ALL)、慢性淋巴白血病 (CLL)、非何傑金氏淋巴瘤 (NHL) 上進行研究
    • 各疾病的CAR-T主要市場機會為何?
    • KOL最感興趣的市場機會?
  • 在安全面需優先考慮什麼?
    • 細胞激素釋放症候群 (CRS)和神經毒性
    • KOL對CAR-T療法安全性的擔憂為何?
    • 副作用管理展望?
  • 自體CAR-T細胞療法主要理論性課題?
    • 如何克服?
    • 自體CAR-T細胞療法理論性阻礙和KOL對克服方法的看法
  • CAR-T細胞療法藥價、核付、市場進入
    • KOL看法?
    • CAR-T療法主要市場阻礙為何?保險者在資金面是否已準備好?
  • KOL對次世代CAR-T細胞方法的關注程度?
    • 同種「現成」CAR-T細胞和具安全開關的CAR-T細胞:所有Pipeline早期階段
    • KOL認為最具潛力的方法及其理由?
  • CAR-T細胞療法和代表性移植術比較
    • 造血幹細胞移植 (HSCT):血液腫瘍治療基礎
    • KOL所見和HSCT的類似點?
    • 專家是否認為CAR-T細胞療法可取代移植?
  • 哪些企業最接近成功商業化?其理由為何?
    • Novartis・Kite Pharma・Juno Therapeutics間競爭日益激烈
    • 被定位為推動CAR-T細胞療法前進的企業?
  • 各企業如何進行自家公司的差異化?
    • 為了在競爭劇烈環境中將其推向市場,CAR-T療法製造商需要做什麼?
  • CAR-T細胞療法10-15年內對血液腫瘍治療演算法的影響為何?
    • CAR-T細胞療法可否滿足未滿足需求,確保未來治療環境的利基地位?



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