Cellular therapies in Japan cellular therapy in Japan.pdfvariety of medical conditions utilizing...
Transcript of Cellular therapies in Japan cellular therapy in Japan.pdfvariety of medical conditions utilizing...
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Cellular therapies in Japan
Yuji Heike, MD, Ph. D.
National Cancer Center
Akihiro Shimosaka, Ph. D.
Senior Research Adviser
Institute of Medical Science, Tokyo University
Director, Division of Research and Development
Research Foundation for Community Medicine
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Development and Regulatory Steps for regenerative therapy
in Japan1. Basic and developmental research by Academy/Company
2. Pre-clinical research needed for clinical study
3. Pre-meeting with Pharmaceutical and Medical Devices
Agent(PMDA) for IND application
4. Confirmation by PMDA for next step
5. Filing for clinical study
Pre-Meeting with PMDA for final study if needed and license
application
6. Approval
Price listing for insurance reimbursement
Some cases, without reimbursement price listing, marketing start
for limited market
7. Marketing
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Approved/ on going clinical
application
1. Regenerative therapy
2. DC therapy
3. Activated T-cell therapy
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Approved product
Autologus cultured skin for severe burn
Japan Tissue Engineering Co., Ltd. (J-Tec)
obtained approval and reimbursed by
insurance.
306,000 J Yen/Seat (Seat:8×10cm)
Price listed in January, 2009
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Process
Patients
Harvest skin
Normal skin cell isolation of cell
transplant
form seat culture
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Japanese Insurance SystemTherapy must be 100% insurance coverage
Or 100 % private coverage� no mixed treatment
Not allowed to combine insurance and private
coverage. Even only one drug is not reimbursed,
patient must cover all the expense for the whole
treatment.
� To overcome this problems, certain hospital can
apply for advanced therapy which is not be
covered by insurance but allowed to combine
private and insurance covered therapy
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Therapies approved as advanced
therapyHospital basis approval
1. MNC injection for neovascularization for
atherosclerosis/ limb ischemia
2. Treatment of systemic sclerosis with purified
autologus CD34+ stem cell
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On going study
1. Cardio vascular regenerative therapy
both company and investigator sponsored
2. Studies on other auto immune disease
3. Study on cultured cornea, not in Japan, in
France sponsored by Japanese company
4. Regenerative therapy for cartilage/bone
5. Regenerative therapy for teeth
6. others
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DC StudyMajority are Investigator IND study
Who should take responsibility when more than
two not approved elements are involved.
Like such case
PB apheresis->CD14+ separation (device)-> DC
maturation (medium, growth factor)-> autologus
tumor derived antigen (processing)->
electroporation(device) => Need paradigm change
Regulate as not a drug, not a device
but combined therapy like surgery
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Neovascularization Study
Animal study and pioneer study were reported
by Japanese researchers but only approved as
advanced therapy which is not covered by
insurance yet.
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Kamihata H, Matsubara H, Nishiue T et al.,,
Implantation of Bone Marrow Mononuclear
Cells Into Ischemic Myocardium Enhances
Collateral Perfusion and Regional Function via
Side Supply of Angioblasts,Angiogenic
Ligands, and Cytokines. Circulation
2001;104:1046-1052.)
Model: Swine
LAD occlusion
Interval 60 min
BM MNcells vs. endothelial cells
Global LV function
Angiogenesis
Myocardial Perfusion
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Hamano K, Li TS, Kobayashi T, Hirata K,
Yano M, Kohno M, Matsuzaki M.
Therapeutic Angiogenesis Induced by Local
Autologous Bone Marrow Cell Implantation
Ann Thorac Surg 2002; 73:1210-5
Model: Dog
LAD occlusion
Interval 30 d
BM MNcells
border zone
Regional LV function
Angiogenesis
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Tomita S, Mickle DA, Weisel RD, Jia ZQ,
Tumiati LC, Allidina Y, Liu P, Li RK. Improved
heart function with myogenesis and
angiogenesis after autologous porcine bone
marrow stromal cell transplantation. J Thorac
Cardiovasc Surg 2002; 123:1132-40
Model: Pig, LAD occlusion
interval 2 wks
5-azacytidine-treated stromal cells
Regional perfusion
RegionalWall motion
RegionalWall thickness
Global LV function (MIBI)
A. Stroke volume
B. Ejection fraction
A.
B.
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before 8 weeks after
before 24 weeks after before 2 weeks after
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Cardiac study
There are few groups who are studying
neovascularization using bone marrow
derived purified CD34+ cells using Isolex 300
and CliniMACS.
Isolex was approved for autologus CD34+
separation but not indicated
CliniMACS is not approved yet but
investigators can use under doctor decision
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Possible benefit of cardiac
regenerative application
Dilated cardiac dysfunction
Patient need organ transplant for cure
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14 yr. old patient with dilated,
non/ischemic cardiomyopathy
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LVAD implantation
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14 yr. old patient with DCM
Berlin Heart EXCOR LVAD
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Implantation of hematopoetic stem cells – 72 sites
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04/14/05 Resuscitation, Centrifugal pump in another Hospital
04/18 Transport to DHZB
04/27 Implant BerlinHeart EXCOR LVAD and autologous
bone marrow stem cell transplanation
June Repeat pump rate reduction trials show
July improvement of cardiac function
08/16 Pump stop under Dobutamine shows normal function
08/29 Explant LVAD
09/19 Discharge home
Stable normal cardiac dimensions and function since
14 yr. old patient with DCMBerlin Heart EXCOR LVAD
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Post-LVAD
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after
LVAD-Explant
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Radial Strain (%)
at LVAD Implant after LVAD Explant
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HSCT = haematopoeitic stem cell transplant
Autoimmune disease HSCT
EBMT Information from Dr. Tyndal
Reports 420
– allogeneic 11
– autologous 409
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Autologous: patients
20 (1–81)• Follow-up, median months (range)
6 (1–28)• Interval diagnosis-ASCT, median months
(range)
35 (2–69)• Age, median years (range)
271 (67%)• Sex, females (%)
94 / 21• Team / country
394• Patients transplanted
405*»Patients mobilised
*Reports = 409; double-transplant = 4ASCT = autologous stem cell transplant
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Multiple sclerosis 122
Myasthenia 1
Neuropathy 1
ALS 2
Systemic sclerosis 63
Systemic lupus erythematosus 51
Rheumatoid arthritis 69
Psoriatic arthritis 2
Juvenile chron. Arthritis 43
Ankylosing spondilitis 2
Sjoegren syndrome 1
Vasculitis 1
Dermatomyositis 7
MCTD 4
Cryoglobulinemia 3
Behcet 3
Wegener’s 3
Polychondritis 1
ITP 10
AIHA 3
Pure red cell aplasia 4
Evans 1
TTP 2
Bowel disease 2
Other 3
Disease
ALS = amyotrophic lateral sclerosis; AIHA = autoimmune haemolytic anaemia; MCTD = mixed connective tissue disease; ITP = idiopathic thrombocytopenic purpura; TTP = thrombotic thrombocytopenic purpura
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Graft type
Auto PBAuto BM
Nu
mb
er
of
pati
en
ts
400
300
200
100
0
Stem cell source
Auto = autologous; BM = bone marrow; SCT = stem cell transplant
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Cy+ G-CSF
+ other
G-CSF
300
200
100
0
Priming
Cy = cyclophosphamide; G-CSF = granulocyte colony-stimulating factor; GM-CSF = granulocyte macrophage colony-stimulating factor
Nu
mb
er
of
pati
en
ts
Cy+ G-CSF
Cy+ GM-CSF
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Chemo-therapy
TdepCD34+
+ TdepCD34+None
200
100
0
Purging
Tdep = T-cell depletion; CD34+ = CD34+ selection
Nu
mb
er
of
pati
en
ts
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OtherCy
+ Oht
Bu +
Cy/Oth±±±± ATG
Cy
+Rad±±±± Oth
Cy
+ ATG±±±± Oth
CyBEAM
±±±± ATG
120
100
80
60
40
20
0
Conditioning
BEAM = carmustine, etoposide, ara-C and melphalan; ATG = antithymocyte globulin; Rad = radiotherapy; Bu = busulphan; Oth = other
Nu
mb
er
of
pati
en
ts
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Outcome
• Patients not transplanted
– dead:
(toxicity / progressive disease / other)
– alive:
(response / flare / withdrawal /
transplant pending)
• Patients transplanted
– alive
– death by progressive disease
– death by infectious complications / toxicity
• Transplant-related mortality at 1 year (range)
9
5 (3/1/1)
4 (1/1/1/1)
348
310
13
25
7% (4–10 %)
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Response in SSc – 63 patients
• Skin score > 25% improved 69%
• Lung function trend to stabilisation
• TRM - first 45 patients 17%
– first 65 patients 12.5%
– with new exclusion criteria 7.5%
• Pulmonary hypertension > 50 mmHg outcome poor
• Cardiac toxicity and cyclophosphamide?
Binks M, et al. Ann Rheum Dis. 2001;60:548–549TRM = treatment related mortality
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Response in MS – 85 patients
• Primary progressive MS 22%
• Secondary progressive MS 55%
• Three-year progression free survival
(non primary progressive MS) 78%
NB: IFN beta = 60%
• Improved by 1 EDSS = 11 patients (6 worse later)
• Deaths:
– TRM = 5 patients , progressive disease = 2 patients
• MRI
– inactive stayed inactive
– active – 85% became inactive
Fassas, et al. J Neurol. 2002 ( in press).
MRI = magnetic resonance imaging; EDSS = expanded disability scale score; MS = multiple sclerosis
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Japanese Advanced Therapy
approval
Cy + G-CSF mobilized PB cells
CliniMACS CD34+ selection
For SS at Kyushu University
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Activated T-Cell Therapy
Autologus activated T-Cell Therapy
Doctor’s own protocol, cell culture is made by
hospital or contract cell processing center.
Not approved.
Private cover only but popular among end
stage cancer patients
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Business Model
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ISCT Mission
ISCT is a global association driving the translation of
scientific research to deliver innovative cellular therapies
to patients
•Foster international translational research
•Inform national and global regulatory framework
development and harmonization
•Drive commercialization strategies
•Educate principal investigators, lab directors, technologists,
regulators and commercial stakeholders
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Medical Tourism
• Medical tourism: Travel across international borders to
acquire healthcare, often on a temporary basis
•Estimates vary
•1.5 million per year US
in 2008 (Toucan Capital)•750,000 Americans
traveled abroad in 2007 (Deloitte quoting
Baliga/Horowitz, 2008)
•Projected to be 1.6 million patients by 2010
(Deloitte, 2008)
Deloitte, 2008
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What’s on the Web?Stem Cell Research Forges Ahead in Latin America: Positive results have been noted in a variety of medical conditions utilizing stem cell therapies, including Parkinson’s, multiple sclerosis, diabetes, vision problems, and other neurological disease…..
StemCellsChina.Com: If you are interested in receiving stem cell therapy, please fill out this short form. A qualified stem cell doctor will be glad to contact you with helpful treatment information….
Cell Medicine arranges stem cell therapy for patients with the following conditions: Autoimmune diseases, cerebral palsy, critical limb ischemia, degenerative joint disease, diabetes, heart failure, multiple sclerosis, osteoarthritis, rheumatoid arthritis & spinal injury…Treatment is only available outside the US and Canada and is not covered by most insurance…..
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Korean Company in Japan
Therapy is regulated as drug in Korea and is
difficult for the company to run the study. In
Japan, autologus cell therapy can be regulated as
clinical treatment under doctor own decision and
can make business.
Japan is thought to be a easy country to run
clinical application without regulatory control.
Reported on news paper.
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Korean Company in Japan
• クリニックを韓国でなく日本に設立したの
は、韓国では自己幹細胞の治療を薬事法
で「医薬品」として管理して
おり、複雑な臨床試験など厳しい規制を設
けているため。日本では、自己幹細胞の治
療を「高度先進医療技術」と
定め、医師の判断の下で自由に治療が可
能だ。
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Cell Therapy Medical Tourism
• Medical tourism for terminal diseases: a
long and inglorious tradition
• Significant risks to
– Patients
– The field of Cellular Therapy
• Mainstream medicine has not responded to
protect patients
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Clinical Trials vs Experimental
Therapies/Medical Innovation
• Field will never progress without well controlled
clinical trials
• Patients not eligible for controlled clinical trials
should be able to choose unproven but
scientifically validated therapies, if truthfully and
ethically informed
• Patients’ need to understand the difference
between the two paradigms
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Bedrock of Ethical Principles
• Declaration of Helsinki
• WHO
• Belmont report
• Clinical trials directive of the European
Commission
• Professional societies
• Others
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Patients’ Rights
1. Right to Seek Treatments
2. Right to Information
– Accurate representation to regarding safety and
efficacy record of treatment
– Likely side effects
– Need for centralized repository of therapies
3. Right to Informed Consent
– Clinical trials
– Unproven therapies
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Role of Local Regulatory Authority
• ISCT acknowledges jurisdiction of local
regulatory authorities
• However the degree of regulation and safeguards
vary markedly and are often not enforced
• Key roles for independent ethics committees
• Need for international harmonization
– International Conference on Harmonization (ICH)
– International industry/professional organizations
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Responsibility of Investigators
• Regulatory compliance
• Publication/dissemination of results
• Following GCPs/clinical standards
established by local regulatory authorities
• Responsibility in advertising
• Reporting adverse events
• Patient supportive care and follow up
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What Can ISCT Do?
• Ensure
– Mitigation of patient risks
– Promotion of scientific development
– Compassionate and early access to
promising therapy
– Promotion of full financial disclosure
• Simple warnings are not enough!
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Proposal
1. Cell Therapy Consumer’s Guide
– To differentiate among
• Approved/standard therapies (eg, HSC transplant)
• Controlled clinical trials
• Valid compassionate use of unapproved therapies
• Treatments not subject to independent scientific and ethical review
– Provide an unbiased review of claims made for unapproved therapies
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Proposal
2. Leverage ISCT expertise in regulatory
affairs and international presence to work
toward global regulatory harmonization
– Working through ICH
– Partnership with other societies
– Beneficiaries
• Patients (safety & efficacy)
• Field of cell therapy (scientific advancement)
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Proposal
3. Partnership with patient advocacy groups
and professional societies
– A personalized and empathetic connection to
patients searching for support
– Access to experts, assessment of emerging
technology/treatments & professional
recommendations
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Conclusions
• Cellular therapy medical tourism is here to stay
• Legitimate cell therapy opportunities for medical tourists: appropriately regulated by local authorities
• However there are many more unethical and potentially dangerous cell therapies
• Proposals for ISCT
1. Consumers’ guide
2. International harmonization
3. Partnership with patient advocacy groups
– Will organize Asian Pacific Activity
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Thank You very much