Unproven Cell Therapies: What Have We Done? Can We Do More?

Transcription

1 Unproven Cell Therapies: What Have We Done? Can We Do More? Kurt C Gunter, MD President, ISCT Chief Medical Officer, Cell Medica

2 The Good: First Cord Blood Transplant Patient in North Carolina, USA Cord blood processed in Indiana, USA Patient, doctors, and cord blood traveled to France for the therapy Medical tourism that changed medical history!

3 The Bad Fetal neural stem cell transplantation Moscow 13 yo boy in Israel with ataxia telangiectasia Received repeated intracerebellar and intrathecal injections of human neural stem cells 4 yrs after cell injections, he presented with a multifocal glioneuronal brain tumor containing cells from at least two donors

4 The Ugly XCell-Center in Dusseldorf, Germany Patients paid up to $32,000 for stem cell injections into the back and brain Cerebral palsy, multiple sclerosis, autism, Parkinson's, Alzheimer's, heart disease, diabetes and spinal cord injuries Aug 2010: 18-month-old boy from Romania was injected in the brain with stem cells followed by bleeding and death May 2010, 10 yo boy from Azerbaijan had the same procedure and almost died

5 Time for Action: What Are the Options? Unethical medial tourism for terminal diseases has a long and inglorious tradition Significant risks to» Patients» The field of cellular therapy Mainstream medicine has not responded to protect patients What can the cell therapy community do? Balance needs to» Mitigate patient risks» Promote scientific development» Compassionate and early access to promising therapy

6 Spectrum of Cell Therapy Medical Tourism Clinical Trials Pilot Studies Phase I, II, III Innovative medical care Non-approved, possibly unproven therapy provided by legitimate (regulated; with oversight) caregivers with appropriate informed consent Unethical Unproven, unregulated medical procedures marketed as beneficial therapy without informed consent

7 Patients Rights Right to information» Accurate representation regarding safety and efficacy record of treatment» Possible side effects Informed consent» Clinical trials» Unproven therapies Right to seek treatments

8 We Are Trying!

9 Vision A broad based coalition of all stakeholders focused on patient advocacy and protection in the area of cell therapy medical tourism Speaking with a unified voice and leveraging our Scientific expertise Reputations Diverse audiences www. loyaltyhammer.com

10 Patient Advisory for Stem Cell Therapy and Medical Tourism: Key Elements Self education Informed consent process Question and answer process Clinical trial participation Treatment procedures and follow up Reporting of treatment results and outcomes Disclosure of all potential expenses

11 Patient Advisory for Stem Cell Therapy and Medical Tourism: Participating Organizations

12 ISCT Presidential Task Force: Unproven Cell Therapies Chair: Massimo Dominici, ISCT President Elect Key Goals: Review field and compile a list of major scientific/ethical priorities. Publish follow up Position Statement in Cytotherapy. Generate and maintain database of cell types and indications with clinical evidence of safety and efficacy. Recommend and effectively communicate action steps that ISCT and other cell therapy stakeholder organizations can take to protect patients. Collaborate with other cell therapy groups and regulatory agencies to achieve these goals.

13 Google Search: Stem Cell Therapy (6 Sep 13)

14 What Can Cell Therapy Stakeholders Do? Options for Discussion Raise awareness» Public service announcements/messages» Web site banners/announcements» Patient education initiatives Incentives for publication Consumer tools» Cell therapy guide for patients and caregivers» ISSCR: Partnership with patient advocacy groups» Trusted source of non-biased information Aggressive regulatory compliance inspections and enforcement

15 What Can Cell Therapy Stakeholders Do? Options for Discussion New regulations» Advertising and promotion» Patient treatment reporting/registration» Adverse event reporting» Import/export of patient treatment materials Global regulatory harmonization» Adverse event reporting requirements» Standardized informed consent» Investigator conflict of interest/financial disclosure requirements» Voluntary standards for cell therapy/stem cell clinics

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