“These clinics have the potential to revolutionize how we deliver stem cell therapies to patients. Stem cell therapies are a completely new way of treating diseases and disorders so we need a completely new way of delivering those in a safe and effective manner. These clinics will help us do just that and the clinical trials carried out in this network will fulfill the agency’s promise of bringing new therapies to patients who need them.”
“Another boondoggle for some medical schools but made to order for private operators like for-profit cancer, dialysis, and laser eye specialty clinics that do one procedure. I can see each of the medical schools gifted with one as they each were gifted with about 25 million dollars for stem cell institute buildings.”
“The NIH at various times has tried to organize clinical trials groups with infrastructure, like quick reaction forces, ready to gear up for a new trial at the drop of a hat. They mainly did nothing but suck money, kept staff employed, because there are generally few drugs ready for early human trials and each treatment that is brought along requires a unique contract, ethics reviews, and different facilities, equipment and staff than planned for. The latest incarnation are CTSAs or CTSIs, clinical and translational science centers funded by the federal NIH that most if not all California medical schools already have.”
“It was felt that more discussion was needed before moving to a vote so another meeting is going to be scheduled.”
“...will decide whether to approve a grantee’s request to use a stem cell line created with paid-for eggs on the basis of whether doing so 'will advance CIRM’s mission.' This criterion is much too vague, and doesn’t include consideration of the health or welfare of the women who undergo egg retrieval. Protecting the well-being of women providing eggs is not even mentioned (though perhaps it could be considered as an element of the fifth of five 'factors to be considered by the ICOC(the agency board),' 'whether the donation…was consistent with `best practices’ at the time of donation').”The standards group also heard from a UCLA researcher who argued on behalf of the change. Kathrin Plath said she and her colleagues wanted to use a paid-for stem cell line from the Oregon experiment that cloned human stem cells.
“Clinics to conduct trials of stem cell therapies have different needs than clinics designed to deliver conventional therapies, DeWitt and Millan said. They need special facilities for handling the cells safely, as well as imaging equipment to track the cells once they're delivered into a patient’s body. Some of this infrastructure already exists, but other parts of it still need to be perfected. Establishing clinics to house multiple trials might create the critical mass needed to get the infrastructure in place, they said....
"Additionally, they said, CIRM hopes that such collaboration would encourage stem cell companies to share information -- speeding their own work and also helping out policymakers and insurers who are trying to figure out how they'll pay for stem cell therapies in the future.”
“The Alpha clinics are aimed at creation of a sturdy foundation for the stem cell industry in California, capitalizing on the burgeoning, international lure of stem cell treatments.”
“In my lab, we are ... interested in understanding what happens to the somatically silenced X chromosome when differentiated cells are reprogrammed by SCNT. The key question is: are these SCNT-ESCs more similar to iPSCs or fertilization-derived ESCs with respect to the epigenetic state of the X chromosome. Furthermore, it has been shown in mouse reprogramming that the active X chromosome becomes deregulated during SCNT-based reprogramming, and we would like to address this problem in the human system as well.
“We believe that the comparison of the epigenetic states between fertilization-derived ESCs, SCNT-ESCs and human iPSCs is important for a better characterization of these cells and understanding of their epigenetic nature.”
“His condition is currently listed as critical. Duane is in the great hands of the university’s doctors and nurses and we are very happy with the outstanding care he is receiving. We do ask that our privacy be respected as Duane heals. We will be in touch to share more.”
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| Duane Roth Connect Photo |
“Our thoughts, prayers and best wishes for a full recovery are with Duane and his family.”
“Workshop participants...emphasized the need for engaged experts who can consider and implement strategies for reimbursement (i.e.generating profits) for stem cell therapies, and to gather evidence for their value and effectiveness that will be important for coverage by payers. Having clear pathways in place will help in building financially sustainable clinics as stem cell therapies gain approval and are implemented as standard medical practice. The Alpha Coordinating Center (funded at $15 million) would work with Accountable Care Organizations (funding gouprs that tie performance to payment) and multi-stakeholder collaborations to generate evidence for informing coverage and payment policies.”
“Incentivizing company sponsors of clinical trials to participate in data sharing will be challenging, and it will be important to protect their interests as appropriate, by respecting confidentiality of proprietary information, while at the same time requiring an appropriate degree of data sharing to advance the mission of the Alpha Stem Cell Clinics Network(five clinics funded at $11 million each). It is interesting to note that at this time, the landscape of disclosing clinical trial results is in flux, and there is increasing international pressure for companies to disclose information on safety and efficacy to the research and medical communities.”
“Alpha Stem Cell Clinics will create unique models for assessing the financial viability of stem cell therapies in a clinical setting. Cost and revenue data may serve as the basis for developing reimbursement options among the public and private sector as therapies accumulate. Identification of reimbursement options can reduce investor uncertainties and encourage further investment by pharmaceutical companies, investors, funding agencies and philanthropists.
“The elements of the Alpha Stem Cell Clinics Network, namely the clinical sites and central coordinating center, will interact with external entities to generate revenue streams that would ultimately ensure financial sustainability independent of CIRM funding (Figure 3).”
“The Coordinating and Information Management Center could develop a fee for service model for its consulting services, and operate like a CRO. Sponsors could choose whether to use the consulting services and whether to use the Alpha Stem Cell Clinics Sites. The Alpha Stem Cell Clinics Sites would generate operating revenue by running the clinical trials and could perhaps support the Center with revenues, as the volume of trials grows and the Network builds steam.
“In a second model, clinical trial sponsors would all contract with the Coordinating and Information Management Center, which would then set prices according to services needed. From this revenue, the Center would pay the Alpha Stem Cell Clinics Sites for costs associated with the clinical trials.
“By the time the CIRM seed money is depleted, the clinics should have established a strong track record and brand for excellence in conducting clinical trials in stem cell therapies, which would attract more clinical trial sponsors, including companies. To have the largest possible impact on healthcare, it will be desirable to increase industry involvement for cell therapies. However engaging corporate involvement could be challenging, given that many cell therapies are not considered patentable, and many of the ongoing trials are conducted in academic settings. It was suggested that the Alpha Stem Cell Clinics Network align as much as possible with corporate “mentality” to maximize corporate participation.
“Overall, the Alpha Stem Cell Clinics Network will help companies increase their 'bandwidth' and run stem cell therapy clinical trials more effectively and successfully. Many companies will already have sufficient in-house expertise for administration and regulatory guidance, and may not need to engage the Center’s consulting services. They would be attracted to the Alpha Stem Cell Clinics Network because of advantages for clinical trials such as benefiting from the collective databases, know-how and experience of the 'brain trust,' positive interactions and lines of communication with the FDA and regulatory experts, positive branding, quality control, accreditation for their trials, access to patient registries for outreach and patient recruitment and, through its clinical trial management resources, help with enrollment.
“Workshop participants also emphasized that attaining reimbursement for stem cell therapies will be necessary for delivering approved therapies into medical practice. It is likely that the business models for at least some of the clinics will resemble bone marrow/HSC transplant clinics, where for stem cell therapies that are proven superior to the standard of care, the clinic will directly receive reimbursement from insurers for the procedure and follow-up care.
“Reaching this point will require advance advocacy and preparation, and the engagement with the CMS at an early stage of product development. It was recommended that the Alpha Stem Cell Clinics Coordinating and Information Management Center should employ experts in reimbursement methodologies to facilitate entry of approved products into healthcare delivery. One workshop participant, Jeff Sheehy(a stem cell agency board member), suggested that early engagement with Accountable Care Organizations (ACOs) could be helpful, given that stem cell therapies offer the promise of cures, which in the long run will offer high value and effectiveness, albeit with ' high front end costs and the necessity of developing the appropriate infrastructure for delivering complex cell based procedures.' Consortiums such as the Green Park Collaborative are being established to develop methodological standards to demonstrate the effectiveness and value of new technologies. These data could be used to inform healthcare payers of the overall benefits of cell therapies and their long-term value, as compared with existing therapies."
“In 2016 and beyond, CIRM will initiate the 'Delivery' phase, in which priorities will be 'facilitating commercialization (and non-commercial adoption, where appropriate) of therapies, advancing therapies to patients, and enabling business models for stem cell-based therapies.' Connecting this network to other networks and centers with a similar mission outside of California will further accelerate the development and delivery of stem cell therapies, through the global exchange of information and expertise. International testing of experimental therapies developed in California will be essential for ensuring their sustainability, as regulatory approval and widespread delivery of these products in major markets throughout the world will ensure commercial viability and sustainability of companies and universities that produce them, which will in turn help deliver returns to investors. Ultimately, establishing a solid foundation for investor engagement will ensure the viability of stem cell therapies as they move into clinical practice.'”
“The late historian of science and technology, Ivan Illich, warned against the processes of medical industries which 'create new needs and control their satisfaction and turn human beings and their creativity into objects.'"
“Women's research eggs (have) become the hot new bio-product, increasing the profits of the multibillion-dollar-per-year infertility industry at the expense of women's health, safety and possibly, their future fertility. Is this the 'equity' we want for ourselves, our sisters and our daughters?”
“...bore witness to the destructive power of modern institutions that 'create needs faster than they can create satisfaction, and in the process of trying to meet the needs they generate, they consume the earth.'”
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| Alpha clinic organizational diagram Graphic by CIRM |
“By providing this resource, the public and potential patients would be better educated and informed, whether or not they should opt to enroll in clinical trials or approved treatments at any of the Alpha clinics.”
“CIRM will pay for an unneeded infrastructure that will be empty space and staff sitting on their hands 99 percent of the time. Or worse yet, CIRM will pay but the space will be used for other things, other clinic procedures paid for by insurance.”
“Rao regularly asks researchers hoping to advance promising stem cell therapies whether they require additional clinical infrastructure. 'So far, what they've told us is they'll let us know if they need anything more than (the) programs that we have already established,' he says.”
“(The proprosal) will yield better clinical trial design, accelerated approval of high quality treatments, and data and know-how to inform regulatory and reimbursement decisions.”
“To accelerate therapeutic development and delivery of stem cell therapies, CIRM proposes establishing the CIRM Alpha Stem Cell Clinics Network (CASC Network). The network will be designed to support projects emanating from CIRM’s funding pipeline, as well as scientifically outstanding stem cell products being developed worldwide and brought to California. Conceptually, the CASC Network is intended to be a sustainable infrastructure designed to support academic- and industry-initiated clinical trials, and delivery of therapies proven safe and effective.”
“The major thrusts (of the overall plan) will be:
• Development of clinical capacity and associated resources designed to support the effective implementation and execution of clinical trials and delivery of registered stem cell therapies
• Compilation of data and information concerning clinical trial experience and therapy outcomes to further inform the research, regulatory, and general community about the status of investigational stem cell interventions and long-term outcomes
• Dissemination of information to the public and counseling of patients and potential trial subjects about therapeutic options and clinical trials involving stem cells in the network and elsewhere.”
“The long-term vision is for the Alpha Clinics to expand and accommodate a broad array of stem cell-based clinical trials, where the trial meets the scientific, clinical trial design and ethical standards set forth by the Alpha Clinics Network, as well as FDA approved treatments.”
The coordinating and information center would be expected to :
“Build relationships with Accountable Care Organizations, and participate in initiatives for informing coverage and payment decisions
“Design strategies to attract investors and philanthropists to CASC network
“Create business plans, and marketing and branding strategies for financial sustainability of the Alpha Clinics Sites and (the coordination/information center)”.
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| Jon Shestack(l) with J.T. Thomas, chairman of CIRM, at a 2012 board meeting California Stem Cell Report photo |
“Over eight years there were moments that were inspiring, some were contentious, and there was a bruising number of meetings but through it all, the board was involved, passionate and, will forever be for me, the gold standard when it comes to integrity.
“The same goes double for the staff – truly the most excellent, devoted, committed group of people I have ever had the pleasure of working with.”
“When I started at CIRM, my sweet son with autism was 12. Now he is 21. Over eight years our family has learned more about how many are the challenges that await him and how few the opportunities he has to look forward to. We have seen his world get smaller and smaller. While my son is special to me. He is not unique. There are thousands and thousands affected by mental illness who need a better life.
“Sometimes feel that I have failed these people, in particular those affected by autism or cerebral palsy. Though CIRM ran first-rate workshops on these disorders, we did not do all we could to follow up, put out disease-specific RFAs and get in proposals that addressed the workshop recommendations. I wish I had been more persuasive."
“In the movies, the third act is where the hero takes stock of all the previous wins and losses, all the hardships and lessons learned, and she puts all that knowledge together in new, and surprising ways until victory is within reach! As CIRM enters its third act, I hope it will do the same. I hope it will challenge itself, always put the urgency of the mission ahead of everything else and be willing to question the policies that have been so successful in the past, and consider that new ones may be needed for the future.
“And this is the future as I see it for CIRM. We will have faith, but we will continue to earn our miracles We will use our hearts and our minds to rip those miracles out of the dreamy future and make them real today. We will seek out the best scientists and encourage them to use all their wisdom, art and discernment to bring us cures. And when we have done that, we will do it again the next day. We will be optimistic, but not satisfied. We will question authority, despise complacency and above all love those among us in need of healing--this is the obligation without end, whose reward is also without end.”
