Clinical stem cell initiatives the emerging economies
Dominique McMahon Visiting Researcher: Brocher Foundation Postdoctoral fellow: Dalla Lana School of Public Health University of Toronto, Canada
It’s nothing a few stem cells and another 75 years worth of research can’t fix…
Why RM in emerging economies? •
RM is relevant to health needs of developing countries
[Image removed to reduce file size]
•
China, India and Brazil have RM capacity Commitment to stem cell research
•
Very little known about RM sectors in these countries
Regenerative medicine:
University Health Network and University of Toronto
Publications on stem cells 2500
Germany
2000
China
United Kingdom
1500
France
1000
Canada South Korea
Australia
500
0
2000
2001
2002
2003
2004
2005
2006
2007
2008
2009
India Brazil
2010
Stem cell publications by country for 2000-2009 searching (“stem cell” OR "stem cells”) in the Web of Knowledge ISI Sci-Expanded Database. China, South Korea, Australia and India, have permissive policies, and China, India and Brazil are emerging economies. (Similar to that published in McMahon et al (2010) Regen Med. 5(1) 35-44.)
Global stem cell policy o Divides world into “restrictive”, “permissive” and “flexible” policy o Does not describe level of explicit regulation or enforcement o Only for hESC research
Map: hZp://mbbnet.umn.edu/scmap.html
China: research regulations 2000
2002
Draft ethical guidelines from Beijing (Sept ’01) and Shanghai (Oct ’01)
2004
“Guidelines for research on hESC” MOST/MOH (Jan ’04) Four No’s (Nov ’02)
• • •
2006
2008
2010
ISSCR Position on Unproven commercial stem cell interventions (Dec ’08) Category 3 medical technology (May ‘09)
Research guidelines suggested by ethics groups in Shanghai and Beijing Government’s liberal guidelines for hESC research encourages innovation Stem cell guidelines brief and lack enforcement
“When we draft our guidelines, we always need to think about our culture as well. For Chinese people, we have not so strong religious ideas about the [embryo]… This is not a person we don'ʹt think so…so we accept.”
China: Translation and unproven therapies •
Unclear: SFDA or MOH?
•
Clinics offer unproven therapy patients
•
Over 200 hospitals offer stem cell therapies for almost any indication to patients from around the world
•
[Image removed to reduce file size]
No clinical evidence show that these therapies work
•
Now a “Category 3” medical technology under MOH “It’s a very bad, very bad image for Chinese in this field. So I think the government really should have tight regulations regarding clinical applications.”
Brazil: • Catholic church vocally opposed to hESC research • Biosecurity law legalizing hESC challenged in a supreme court case [Image removed to reduce file size]
• Supreme Court opened a public debate: multiple stakeholder types consulted • Stem cells discussed widely by media • National survey found 75% of Brazilians support embryonic stem cell research
Regulations in India: seeking clinical clarity 2000
2002
Ethical guidelines for Biomedical Research on Human Subjects
• • •
2004
2006
Bill introduced: “Biomedical research on Human Subjects (Promotion and Regulation) AIMS states they began stem cell clinical trial
2008
2010
“Guidelines for Stem cell research and therapy” published
Still waiting for biomedical research on human subjects bill to pass
Have guidelines for both research and clinical contexts Enforcement will depend on passing the bill on clinical regulations Clarity on clinical context necessary to move research forward
“We want to promote stem cell technology, but not in this scandalous way” -‐‑Vasantha Muthuswami (Indian Council of Medical Research)
What do these stories tell us? •
Much more open discussion on stem cell governance in Brazil than China o Moved slower in Brazil BUT – China has had to back track.
•
Both countries struggled with different stake-holder perspectives
•
The regulations of both countries were influenced by the culture and socio-political situation
•
Regulations need implementation and enforcement
•
Regulation and governance needed at each levels of the pipeline
•
Interaction of national and international regulations and guidelines
Research focus now: • Emerging issue: clinical regulation o Stem cells in particular o Brazil and China have very different approaches
• Two main approaches to clinical stem cell use: 1.
Clinical trials: usually after obtaining regulatory approval, main goal is to show safety or efficacy, contribute to science
2.
Unregulated clinical stem cell use: typically not “research”, goal is individual patient outcomes
• Research topic: Clinical stem cell use in China and Brazil, both regulated and unregulated o Social, regulatory, and ethical aspects
Data sources •
Brazil: o 1 week fieldwork in Nov. 2011 o 8 interviews with clinicians and basic researchers o Sites: Rio de Janeiro and Porto Alegre
China
•
China : o 2 weeks fieldwork in Sept 2011 o 14 interviews with clinicians, therapy providers, researchers, and ethicists o Sites: Beijing, Nanjing, Guanzhou
•
Other sources of data: o Clinical trial registry analysis o Scientometrics o Regulations, policies, laws, government reports o Secondary analysis of RM interviews: interviews with over 150 researchers, clinicians, firms, government agencies and regulatory experts.
Stem cell clinical trials • [Unpublished graph removed]
Stem cell sources Interplay between qualitative and quantitative data: Is stem cell sources affected by socio-cultural values? Regulation? Capacity? Ethics? Market forces? Others?
[Unpublished graphs removed]
Stem cell source Brazil’s primarily mononuclear versus India and China’s broader use of stem cell types • Genetic diversity of local populations • Technical capacity • Regulation (formal, informal, enforcement) • Public versus commercial interest o Cost and access – mononuclear better for local needs o Firms: provide technical expertise, extraction and proliferation devices and materials. • China’s 1 child policy: investing in cord blood
In the news… Adult stem cell treatment brings major league pitcher back to the mound by Thaddeus Baklinski Tue Jun 07 2:46 PM EST
China BOCA RATON, Florida, June 7, 2011 (LifeSiteNews.com) - Bartolo Colon’s career seemed to be going down the drains, after being one of major league baseball’s top starting pitchers. In 2005 Colon won the American League Cy Young award, but a series of shoulder and elbow injuries sidelined him until, in 2010, he didn’t play in the majors at all. By 2009, Colon’s astonishing 97 mph fastball had slowed down considerably and every pitch he threw resulted in agonizing pain: so he went home to the Dominican
Republic, defeated. But in March of 2010, a doctor in the Dominican Republic, Leonel Liriano, contacted Dr. Joseph R. Purita, an orthopedic surgeon who runs a regenerative medicine clinic in Boca Raton. Dr. Liriano asked Purita if he would help in treating Colon. Purita has used adult stem cell therapy to help numerous athletes with sports injuries, including athletes with the Baltimore Ravens, the Miami Dolphins, the Chicago White Sox and the Texas Rangers. The treatments involve adult stem cells and platelet-rich plasma therapy, or P.R.P., as an alternative to surgery or in combination with it. In April 2010 Dr. Purita agreed to Dr. Liriano’s request and flew to the Dominican Republic to interview Colon. hZp://www.lifesitenews.com/news/adult-‐‑ stem-‐‑ cell-‐‑treatment-‐‑ brings-‐‑ major-‐‑league-‐‑ pitcher-‐‑ back-‐‑to-‐‑ the-‐‑mound/
In the news…
China
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NATURE BIOTECHNOLOGY | NEWS
South Korea's stem cell approval
China
Heiko Yang Nature Biotechnology 29, 857 (2011) doi:10.1038/nbt1011-857b Published online 13 October 2011 On July 1, the Korea Food and Drug Administration approved a stem cell treatment for acute myocardial infarction developed by FCB-Pharmicell of Seongnam. Locals view the regulatory go-ahead as a world first and also a vote of confidence for the nation's scientific expertise following the
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Adult stem cell treatment bringsof major back to the S. Korea approves sales newleague stempitcher cell drug mound Foreign 2012-01-19 16:14 by Thaddeus Baklinski
SEOUL, January 19, 2012 (AFP) - South Korea's government drug agency cleared the way Thursday for
Tue Jun 07 2:46 PM of EST commercial sales what it called the world's first approved medicine using stem cells collected from other
China
people.
BOCA RATON, Florida, June 7, 2011 (LifeSiteNews.com) - Bartolo Colon’s career seemed to be going down the drains, after being one of major league baseball’s top Cartistem, developed by Seoul-based Medipost, willColon help regenerate knee cartilage stem cells starting pitchers. In 2005 won the American League Cyusing Young award, butdeveloped a from newborns' umbilical cord blood, the Korea Food and Drug Administration said.in 2010, he didn’t play in the series of shoulder and elbow injuries sidelined him until, majors at all.
"Cartistem is... the world's first approved allogeneic (taken fromfastball different of considerably the same species) stem By 2009, Colon’s astonishing 97 mph hadindividuals slowed down and cell drug, that can offer newevery opportunity treatment degenerative arthritis," the administration pitch he for threw resultedofin patients agonizingwith pain: so he went home to the Dominican said in a statement. Republic, defeated. But in March of 2010, a doctor in the Dominican Republic, Leonel Liriano, contacted Dr. Joseph R. Purita, an Medipost said 27 billion wona ($23.8 million) from private andDr. government funds hadif he been invested orthopedic surgeon who runs regenerative medicine clinic ininvestors Boca Raton. Liriano asked Purita would help to develop Cartistem in treating Colon. since 2001. The drug can be injected into a patient's knees via surgery. Purita has used adult stem cell therapy to help numerous athletes with sports injuries, including athletes with the Clinical trials havethe been under way in the the Chicago United States since last year, theRangers. statement said. Baltimore Ravens, Miami Dolphins, White Sox and the Texas The treatments involve adult stem cells and platelet-rich plasma therapy, or P.R.P., as an alternative to surgery or Two of the world's top 10 drugmakers are in talks to seek a worldwide licence to make the drug, a Medipost in combination with it.
spokesman told AFP, adding that final trials involving a large number of people would likely begin in the US in hZp://news.yahoo.com/korea-‐‑ pproves-‐‑ sales-‐‑request stem-‐‑cell-‐‑ In April 2010 Dr. Purita agreedato Dr. Liriano’s anddrug-‐‑ flew 1to70020636.html the Dominican Republic to interview Colon. 2015.
In the news…
China Stops Unapproved Stem Cell Treatments | Fox News
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Adult stem cell treatment brings major league pitcher back to the mound
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by Thaddeus Baklinski
China Stops Unapproved Stem Cell Treatments Tue Jun 07 2:46 PM EST Published January 10, 2012 | Reuters
China
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BOCA RATON, Florida, June 7, 2011 (LifeSiteNews.com) - Bartolo Colon’s career China has ordered a halt to all unapproved stem treatments anddrains, clinicalafter trials,being state media on Tuesday, as Beijing seemed to be cell going down the one ofreported major league baseball’s top starting pitchers. In now 2005 won the seeks to rein in the largely untested stem cell therapies onColon offer across theAmerican country. League Cy Young award, but a series of shoulder and elbow injuries sidelined him until, in 2010, he didn’t play in the majors new at all. The Ministry of Health will stop accepting applications for stem cell programs, a ban that will last until July and comes as China By 2009, Colon’s astonishing 97 mph fastball had slowed down considerably and begins a one-year program to regulate the sector better, Xinhua cited a ministry spokesman as saying. every pitch he threw resulted in agonizing pain: so he went home to the Dominican Republic, defeated. A growing number of hospitals and clinics in large cities in China have been offering stem cell therapies for treatment of diseases But in from March of 2010, a doctor intothe Dominican Republic, Leonel Liriano, contacted Joseph R. Purita, an and which ranging cancer and Alzheimer's spinal cord injuries, treatments that are backed by littleDr. or no scientific evidence orthopedic surgeon who runs a regenerative medicine clinic in Boca Raton. Dr. Liriano asked Purita if he would help are considered at best experimental. in treating Colon. Puritaofhas used adult stem cell therapy help patients numerous with sports including--athletes the Some these involve large general hospitalstowhere payathletes thousands -- or even injuries, tens of thousands of dollarswith for treatments Baltimore Ravens, the Miami Dolphins, the Chicago White Sox and the Texas Rangers. that are advertised online, which attract both Chinese patients and those from overseas, sparking what experts say is a dubious Theoftreatments involve adult stem cells and platelet-rich plasma therapy, or P.R.P., as an alternative to surgery or type medical tourism. in combination with it. In April 2010 Dr. Purita agreed to Dr. Liriano’s request and to flew to theearlier, Dominican Republic to interview According to patients, doctors and relatives of patients spoke Reuters patients have come away withColon. little or no hZp://www.reuters.com/article/2012/01/10/us-‐‑ cwho hina-‐‑ health-‐‑ stem-‐‑ cell-‐‑ idUSTRE8090GA20120110
Thinking about commercialization… • What evidence base is needed to support the use of new treatments? • # clinical trials, design of clinical trials, quality, effect? • Should stem cells be treated as… • A drug? Other biologics (blood)? Surgery? Devices? • (How do these shape access? Affordability?) • How does the the approach to “commercializing” stem cells shape • Translational model? • Type of cells used? • Who regulates? • How accessible? University Health Network and University of Toronto
Thinking about unregulated treatments… • When should patients pay for unproven (stem cell) treatments? • As part of a clinical trial? • As part of companionate/emergency care? • As patients seeking treatment abroad? • What responsibilities do doctors have to patients that return? • To continue conventional treatment? • To continue the unconventional treatments? • How should patients be informed about these procedures • Currently direct to consumer advertising, ISSCR handbook…
University Health Network and University of Toronto
Questions? Comments?
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Thank you! Special thanks:
Funding:
Brocher Foundation All our key informants Colleagues at the University of Toronto Canadian Institutes of Health Research Rmethnet Network funding Master’s Training Awards Banting and Best Doctoral awards Ontario Graduate Scholarships
