When Anna Kuehl began losing her vision, she feared losing the ability to read and go on long walks in nature—two of her favorite pastimes. Anna had been diagnosed with age-related macular degeneration, the leading cause of vision loss in the US. She lost the central vision in her left eye, which meant she could no longer make out people’s faces clearly, drive a car, or read the time on her watch.
The treatment sprang out of research done by Dr. Mark Humayun and his team at USC. In collaboration with Regenerative Patch Technologies they developed a stem cell-derived implant using cells from a healthy donor. The implant was then placed under the retina in the back of the eye. The hope was those stem cells would then repair and replace damaged cells and restore some vision.
In the past, using donor cells meant that patients often had to be given long-term immunosuppression to stop their body’s immune system attacking and destroying the patch. But in this trial, the patients were given just two months of immunosuppression, shortly before and after the implant procedure.
In a news story on the USC website, Dr. Humayun said this was an important advantage. “There’s been some debate on whether stem cells derived from a different, unrelated person would survive in the retina without long-term immunosuppression. For instance, if you were to receive a kidney transplant, long-term immunosuppression would be required to prevent organ rejection. This study indicates the cells on the retinal implant can survive for up to two years without long-term immunosuppression.”
Cells show staying power
When one of the patients in the clinical trial died from unrelated causes two years after getting the implant, the research team were able to show that even with only limited immunosuppression, there was no evidence that the patient’s body was rejecting the donor cells.
“These findings show the implant can improve visual function in some patients who were legally-blind before treatment and that the cells on the implant survive and remain functional for at least two years despite not being matched with those of the patient,” Humayun said.
For Anna Kuehl, the results have been remarkable. She was able to read an additional 17 letters on a standard eye chart. Even more importantly, she is able to read again, and able to walk and enjoy nature again.
Dr. Humayun says the study—published in the journal Stem Cell Reports—may have implications for treating other vision-destroying diseases. “This study addresses the debate over the viability of using mismatched stem cells — this shows that a mismatched stem cell derived implant can be safe and viable over multiple years.”
Researchers at the Keck School of Medicine of USC have used a stem cell-based bio-implant to repair cartilage and delay joint degeneration in a large animal model. This paves the way to potentially treat humans with cartilage injuries and osteoarthritis, which occurs when the protective cartilage at the ends of the bones wears down over time. The disorder affects millions worldwide.
The researchers are using this technology to manufacture the first 64 implants to be tested on humans with support from a $6 million grant from the California Institute for Regenerative Medicine (CIRM).
Researchers Dr. Denis Evseenko, and Dr. Frank Petrigliano led the development of the therapeutic bio-implant, called Plurocart. It’s composed of a scaffold membrane seeded with stem cell-derived chondrocytes, the cells responsible for producing and maintaining healthy articular cartilage tissue.
In the study, the researchers implanted the Plurocart membrane into a pig model of osteoarthritis, resulting in the long-term repair of articular cartilage defects. Evseenko said the findings are significant because the implant fully integrated in the damaged articular cartilage tissue and survived for up to six months. “Previous studies have not been able to show survival of an implant for such a long time,” Evseenko added.
The researchers also found that the cartilage tissue generated was strong enough to withstand compression and elastic enough to accommodate movement without breaking.
Osteoarthritis, an often-painful disorder, can affect any joint, but most commonly affects those in our knees, hips, hands and spine. The USC researchers hope their implant will help prevent the development of arthritis and alleviate the need for invasive joint replacement surgeries.
“Many of the current options for cartilage injury are expensive, involve complex logistical planning, and often result in incomplete regeneration,” said Petrigliano. “Plurocart represents a practical, inexpensive, one-stage therapy that may be more effective in restoring damaged cartilage and improve the outcome of such procedures.”
Read the full study here and learn more about the CIRM grant here.
This upcoming July is healthy vision month, a time to remember the importance of making vision and eye health a priority. It’s also a time to think about the approximately 12 million people, 40 and over in the United States, that have a vision impairment. Vision can be something that many of us take for granted, but losing even a portion of it can have a profound impact on our everyday life. It can impact your ability to do everyday things, from basic hygiene routines and driving to hobbies such as reading, writing, or watching a film.
It is because of this that CIRM has made vision related problems a priority, providing over $69 million in funding for six clinical trials related to vision loss. There is reason to be hopeful as these trials have demonstrated promising results. One of these trials, conducted by Regenerative Patch Technologies LLC (RPT), announced today results from its CIRM funded clinical trial ($16.3 million) for advanced, dry age-related macular degeneration (AMD).
AMD is a progressive disease resulting in death of the retinal pigment epithelium (RPE), an area of the eye that plays a key role in maintaining vision. Damage to the RPE causes distortion to central vision and eventually leads to legal blindness. Thanks to CIRM funding, RPT and scientists at the University of Southern California (USC) and UC Santa Barbara (UCSB) are growing specialized RPE cells from human embryonic stem cells (hESCs), placing them on a single layer scaffold, and implanting the combination device in the back of the eye to try to reverse the blindness caused by AMD.
One of the trial participants is Anna Kuehl, a USC alumna and avid nature lover. She was diagnosed with AMD in her mid 30s and gradually began losing the central vision in her left eye. Although her peripheral vision remained intact, she could no longer make out people’s faces clearly, drive a car, or read the time on her watch. This also meant she would have much more difficulty going on the nature hikes she enjoys so much. After receiving treatment, she noticed improvements in her vision.
Anna was not alone in these improvements post treatment. The implant, known as CPCB-RPE1, was delivered to the worst eye of 15 patients with AMD. All treated eyes were legally-blind having a best corrected visual acuity (BCVA) of 20/200 or worse (20/20 indicates perfect vision).
Patients in the clinical trial were assessed for visual function and the results were as follows:
At an average of 34 months post-implantation (range 12-48 months), 27% (4/15) showed a greater than 5 letter improvement in BCVA and 33% (5/15) remained stable with a BCVA within 5 letters of baseline value. The improvements ranged from 7-15 letters or 1-3 lines on an eye chart.
In contrast, BCVA in the fellow, untreated eye declined by more than 5 letters (range 8-21 letters or 1-4 lines on an eye chart) in 80% (12/15) of subjects. There was no improvement in BCVA in the untreated eye of any subject.
The implant was delivered safely and remained stably in place throughout the trial.
Refinements to the implantation procedure during the trial further improved its efficiency and safety profile.
In a news release from RPT, Mark Humayun, M.D., Ph.D., founder and co-owner of RPT, Director of the USC Ginsburg Institute for Biomedical Therapeutics and Co-Director of the USC Roski Eye Institute, Keck Medicine of USC, had this to say about the trial results.
“The improvements in best corrected visual acuity observed in some eyes receiving the implant are very promising, especially considering the very late stage of their disease. Improvements in visual acuity are exceedingly rare in geographic atrophy as demonstrated by the large decline in vision in many of the untreated eyes which also had disease. There are currently no approved therapies for this level of advanced dry age-related macular degeneration”.
The full presentation can be found on RPT’s website linked here.
Watch the video below to learn more about Anna’s story.
Age-related macular degeneration (AMD) is the leading cause of vision loss and blindness in the elderly in the U.S. It’s estimated that some 11 million Americans could have some form of the disease, a number that is growing every year. So if you are going to develop a treatment for this condition, you need to make sure it can reach a lot of people easily. And that’s exactly what some CIRM-supported researchers are doing.
Let’s back up a little first. AMD is a degenerative condition where the macular, the small central portion of your retina, is slowly worn away. That’s crucial because the retina is the light-sensing nerve tissue at the back of your eye. At first you notice that your vision is getting blurry and it’s hard to read fine print or drive a car. As it progresses you develop dark, blurry areas in the center of your vision.
There are two kinds of AMD, a wet form and a dry form. The dry form is the most common, affecting 90% of patients. There is no cure and no effective treatment. But researchers at the University of Southern California (USC), the University of California Santa Barbara (UCSB) and a company called Regenerative Patch Technologies are developing a method that is looking promising.
They are using stem cells to grow retinal pigment epithelium (RPE) cells, the kind attacked by the disease, and putting them on a tiny synthetic scaffold which is then placed at the back of the eye. The hope is these RPE cells will help slow down the progression of the disease or even restore vision.
Early results from a CIRM-funded clinical trial are encouraging. Of the five patients enrolled in the Phase 1/2a trial, four maintained their vision in the treated eye, two showed improvement in the stability of their vision, and one patient had a 17-letter improvement in their vision on a reading chart. In addition, there were no serious side effects or unanticipated problems.
So now the team are taking this approach one step further. In a study published in Scientific Reports, they say they have developed a way to cryopreserve or freeze this cell and scaffold structure.
In a news release, Dr. Dennis Clegg of UCSB, says the frozen implants are comparable to the non-frozen ones and this technique will extend shelf life and enable on-demand distribution to distant clinical sites, increasing the number of patients able to benefit from such treatments.
“It’s a major advance in the development of cell therapies using a sheet of cells, or a monolayer of cells, because you can freeze them as the final product and ship them all over the world.”
You can’t fix a global problem at the local level. That’s the gist of a new perspective piece in the journal Stem Cell Reports that calls for a global approach to rogue stem cell clinics that offer bogus therapies.
The authors of the article are calling on the World Health Organization (WHO) to set up an advisory committee to draw up rules and regulations to help guide countries trying to shut these clinics down.
In a news release, senior author Mohamed Abou-el-Enein, the executive director of the joint University of Southern California/Children’s Hospital of Los Angeles Cell Therapy Program, says these clinics are trying to cash in on the promise of regenerative medicine.
“Starting in the early 2000s… unregulated stem cell clinics offering untested and poorly characterized treatments with insufficient information on their safety and efficacy began emerging all over the world, taking advantage of the media hype around stem cells and patients’ hope and desperation.”
The authors include Lawrence Goldstein, PhD, a CIRM Board member and a Science Policy Fellows for the International Society for Stem Cell Research (ISSCR).
Zubin Master, an associate professor of biomedical ethics at the Mayo Clinic, says the clinics prey on vulnerable people who have serious medical conditions and who have often tried conventional medical approaches without success.
“We should aim to develop pathways to provide patients with evidenced-based experimental regenerative intervention as possible options where there is oversight, especially in circumstances where there is no suitable alternative left.”
The report says: “The unproven SCI (stem cell intervention) industry threatens the advancement of regenerative medicine. Reports of adverse events from unproven SCIs has the potential to affect funding and clinical trial recruitment, as well as increasing burdens among regulatory agencies to oversee the industry.
Permitting unregulated SCIs to flourish demonstrates a lack of concern over patient welfare and undermines the need for scientific evidence for medicinal product R&D. While some regulatory agencies have limited oversight or enforcement powers, or choose not to use them, unproven SCI clinics still serve to undermine authority given to regulatory agencies and may reduce public trust impacting the development of safe and effective therapies. Addressing the continued proliferation of clinics offering unproven SCIs is a problem worth addressing now.”
The authors say the WHO is uniquely positioned to help create a framework for the field that can help address these issues. They recommend setting up an advisory committee to develop global standards for regulations governing these clinics that could be applied in all countries. They also say we need more educational materials to let physicians as well as patients understand the health risks posed by bogus clinics.
This article comes out in the same week that reports by the Pew Charitable Trust and the FDA also called for greater regulation of these predatory clinics (we blogged about that here). Clearly there is growing recognition both in the US and worldwide that these clinics pose a threat not just to the health and safety of patients, but also to the reputation of the field of regenerative medicine as a whole.
“I believe that the global spread of unproven stem cell therapies reflects critical gaps in the international system for responding to health crises, which could put the life of thousands of patients in danger,” Abou-el-Enein says. “Urgent measures are needed to enhance the global regulatory capacity to detect and respond to this eminent crisis rapidly.”
Too many acronyms? Not to worry. It is all perfectly clear in the news release we just sent out about this.
A new collaboration between the California Institute for Regenerative Medicine (CIRM) and the Chan Zuckerberg Initiative (CZI) will advance scientific efforts to respond to the COVID-19 pandemic by collaborating on disseminating single-cell research that scientists can use to better understand the SARS-CoV-2 virus and help develop treatments and cures.
CIRM and CZI have signed a Memorandum of Understanding (MOU) that will combine CIRM’s infrastructure and data collection and analysis tools with CZI’s technology expertise. It will enable CIRM researchers studying COVID-19 to easily share their data with the broader research community via CZI’s cellxgene tool, which allows scientists to explore and visualize measurements of how the virus impacts cell function at a single-cell level. CZI recently launched a new version of cellxgene and is supporting the single-cell biology community by sharing COVID-19 data, compiled by the global Human Cell Atlas effort and other related efforts, in an interactive and scalable way.
“We are pleased to be able to enter into this partnership with CZI,” said Dr. Maria T. Millan, CIRM’s President & CEO. “This MOU will allow us to leverage our respective investments in genomics science in the fight against COVID-19. CIRM has a long-standing commitment to generation and sharing of sequencing and genomic data from a wide variety of projects. That’s why we created the CIRM genomics award and invested in the Stem Cell Hub at the University of California, Santa Cruz, which will process the large complex datasets in this collaboration.”
“Quickly sharing scientific data about COVID-19 is vital for researchers to build on each other’s work and accelerate progress towards understanding and treating a complex disease,” said CZI Single-Cell Biology Program Officer Jonah Cool. “We’re excited to partner with CIRM to help more researchers efficiently share and analyze single-cell data through CZI’s cellxgene platform.”
In March 2020, the CIRM Board approved $5 million in emergency funding to target COVID-19. To date, CIRM has funded 17 projects, some of which are studying how the SARS-CoV-2 virus impacts cell function at the single-cell level.
Three of CIRM’s early-stage COVID-19 research projects will plan to participate in this collaborative partnership by sharing data and analysis on cellxgene.
Dr. Evan Snyder and his team at Sanford Burnham Prebys Medical Discovery Institute are using induced pluripotent stem cells (iPSCs), a type of stem cell that can be created by reprogramming skin or blood cells, to create lung organoids. These lung organoids will then be infected with the novel coronavirus in order to test two drug candidates for treating the virus.
Dr. Brigitte Gomperts at UCLA is studying a lung organoid model made from human stem cells in order to identify drugs that can reduce the number of infected cells and prevent damage in the lungs of patients with COVID-19.
Dr. Justin Ichida at the University of Southern California is trying to determine if a drug called a kinase inhibitor can protect stem cells in the lungs and other organs, which the novel coronavirus selectively infects and kills.
“Cumulative data into how SARS-CoV-2 affects people is so powerful to fight the COVID-19 pandemic,” said Stephen Lin, PhD, the Senior CIRM Science Officer who helped develop the MOU. “We are grateful that the researchers are committed to sharing their genomic data with other researchers to help advance the field and improve our understanding of the virus.”
CZI also supports five distinct projects studying how COVID-19 progresses in patients at the level of individual cells and tissues. This work will generate some of the first single-cell biology datasets from donors infected by SARS-CoV-2 and provide critical insights into how the virus infects humans, which cell types are involved, and how the disease progresses. All data generated by these grants will quickly be made available to the scientific community via open access datasets and portals, including CZI’s cellxgene tool.
Today the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $750,000 to Dr. Xiaokui Zhang at Celularity to conduct a clinical trial for the treatment of COVID-19. This brings the total number of CIRM clinical trials to 64, including three targeting the coronavirus.
This trial will use blood stem cells obtained from the placenta to generate natural killer (NK) cells, a type of white blood cell that is a vital part of the immune system, and administer them to patients with COVID-19. NK cells play an important role in defense against cancer and in fighting off viral infections. The goal is to administer these cells to locate the active sites of COVID-19 infection and destroy the virus-infected cells. These NK cells have been used in two other clinical trials for acute myeloid leukemia and multiple myeloma.
The Board also approved two additional awards for Discovery Stage Research (DISC2), which promote promising new technologies that could be translated to enable broad use and improve patient care.
One award for $100,000 was given to Dr. Albert Wong at Stanford. Dr. Wong has recently received an award from CIRM to develop a vaccine that produces a CD8+ T cell response to boost the body’s immune response to remove COVID-19 infected cells. The current award will enable him to expand on the initial approach to increase its potential to impact the Latinx and African American populations, two ethnicities that are disproportionately impacted by the virus in California.
The other award was for $249,996 and was given to Dr. Preet Chaudhary at the University of Southern California. Dr. Chaudary will use induced pluripotent stem cells (iPSCs) to generate natural killer cells (NK). These NK cells will express a chimeric antigen receptor (CAR), a synthetic receptor that will directly target the immune cells to kill cells infected with the virus. The ultimate goal is for these iPSC-NK-CAR cells to be used as a treatment for COVID-19.
“These programs address the role of the body’s immune T and NK cells in combatting viral infection and CIRM is fortunate enough to be able to assist these investigators in applying experience and knowledge gained elsewhere to find targeted treatments for COVID-19” says Dr. Maria T. Millan, the President & CEO of CIRM. “This type of critical thinking reflects the resourcefulness of researchers when evaluating their scientific tool kits. Projects like these align with CIRM’s track record of supporting research at different stages and for different diseases than the original target.”
The CIRM Board voted to endorse a new initiative to refund the agency and provide it with $5.5 billion to continue its work. The ‘California Stem Cell Research, Treatments and Cures Initiative of 2020 will appear on the November ballot.
The Board also approved a resolution honoring Ken Burtis, PhD., for his long service on the Board. Dr. Burtis was honored for his almost four decades of service at UC Davis as a student, professor and administrator and for his 11 years on the CIRM Board as both a member and alternate member. In the resolution marking his retirement the Board praised him, saying “his experience, commitment, knowledge, and leadership, contributed greatly to the momentum of discovery and the future therapies which will be the ultimate outcome of the dedicated work of the researchers receiving CIRM funding.”
Jonathan Thomas, the Chair of the Board, said “Ken has been invaluable and I’ve always found him to have tremendous insight. He has served as a great source of advice and inspiration to me and to the ICOC in dealing with all the topics we have had to face.”
Lauren Miller Rogen thanked Dr. Burtis, saying “I sat next to you at my first meeting and was feeling so extraordinarily overwhelmed and you went out of your way to explain all these big science words to me. You were always a source of help and support, and you explained things to me in a way that I always appreciated with my normal brain.”
Dr. Burtis said it has been a real honor and privilege to be on the Board. “I’ve been amazed and astounded at the passion and dedication that the Board and CIRM staff have brought to this work. Every meeting over the years there has been a moment of drama and then resolution and this Board always manages to reach agreement and serve the people of California.”
Various types of cancer can become particularly aggressive and difficult to treat once they spread from their initial point of origin to other parts of the body. This unfortunate phenomenon, known as metastasis, can make treatment very challenging, decreasing the chance of survival for the patient.
In order to better understand this process, a CIRM supported study at USC looked at breast cancer cells circulating in the blood that eventually invade the brain. The findings, which appear in Cancer Discovery, shed light on how tumor cells in the blood are able to target a particular organ, which may enable the development of treatments than can prevent metastasis from occurring.
Dr. Min Yu and her lab at USC were able to isolate breast cancer cells from the blood of breast cancer patients whose cancer had already metastasized. The team then expanded the number of cancer cells through a process known as cell culture. These expanded human tumor cells were then injected into the bloodstream of animal models. It was found that these cells migrated to the brain as was predicted.
Upon further analysis, Dr. Yu and her lab discovered a protein on the surface of the tumor cells in the bloodstream that enable them to breach the blood brain barrier, a protective layer around the brain that blocks the passage of certain substances, and enter the brain. Additionally, Dr. Yu and her team discovered another protein inside the tumor cells that shield them from the brain’s immune response, enabling these cells to grow inside the brain.
In a news release in Science Magazine, Dr. Yu talks about how these findings could be used to improve treatment and prevention options for those with aggressive cancers:
“We can imagine someday using the information carried by circulating tumor cells to improve the detection, monitoring and treatment of the spreading cancers. A future therapeutic goal is to develop drugs that get rid of circulating tumor cells or target those molecular signatures to prevent the spread of cancer.”
CIRM has also funded a separate clinical trial related to the treatment of breast cancer related brain metastases.
A new independent report says developing stem cell treatments and cures for some of the most common and deadly diseases could produce multi-billion dollar benefits for California in reduced healthcare costs and improved quality and quantity of life.
Predicting the future is always complicated and uncertain and many groups are looking at the best models to determine the value and economic impact of cell and gene therapy as the first products are just entering the market. This study provides some insights into the potential financial benefits of developing effective stem cell treatments for some of the most intractable diseases affecting California today.
The impact could affect millions of people. In
2018 for Californians over the age of 50:
Nearly half were
predicted to develop diabetes in their lifetime
More than one third
will experience a stroke
Between 5 and 8 percent will develop either breast, colorectal,
lung, or prostate cancer
The report says that a therapy that decreased
the incidence of diabetes by 50 percent in Californians over the age of 51
would translate into a gain for the state of $322 billion in social value
between now and 2050. Even just reducing diabetes 10% would lead to a gain of
$60 billion in social value over the same period.
For stroke a 50 percent reduction would generate an estimated $229 billion in social value. A 10 percent reduction would generate $47 billion
For breast cancer a 50 percent reduction would generate $56 billion in social value; for colorectal cancer it would be $72 billion; for lung cancer $151 billion; and prostate cancer $53 billion.
The impact of a cure for any one of those
diseases would be enormous. For example, a 51-year-old woman cured of lung
cancer could expect to gain a lifetime social value of almost half a million
dollars ($467,275). That’s a measure of years of healthy life gained, of years
spent enjoying time with family and friends and not wasting away or lying in a
The researchers say: “Though advances in
scientific research defy easy predictions, investing in biomedical research is
important if we want to reduce the burden of common and costly diseases for
individuals, their families, and society. These findings show the value and
impact breakthrough treatments could have for California.”
“Put in this context, the CIRM investment
would be worthwhile if it increased our chances of success even modestly.
Against the billions of dollars in disease burden facing California, the
relatively small initial investment is already paying dividends as researchers
work to bring new therapies to patients.”
The researchers determined the “social value”
using a measure called a quality adjusted life-year (QALY). This is a way of
estimating the cost effectiveness and consequences of treating or not treating
a disease. For example, one QALY is equivalent to one year of perfect health for
an individual. In this study the value of that year was estimated at $150,000.
If someone is sick with, say, diabetes, their health would be estimated to be
0.5 QALY or $75,000. So, the better health a person enjoys and the longer they
enjoy it the higher QALY score they accumulate. In the case of a disease
affecting millions of people in that state or country that can obviously lead
to very large QALY scores representing potentially billions of dollars.
An independent Economic Impact Report says the California Institute for Regenerative Medicine (CIRM) has had a major impact on California’s economy, creating tens of thousands of new jobs, generating hundreds of millions of dollars in new taxes, and producing billions of dollars in additional revenue for the state.
The report, done by Dan Wei and Adam Rose at the Price School of Public Policy at the University of Southern California, looked at the impacts of CIRM funding on both the state and national economy from the start of the Stem Cell Agency in 2004 to the end of 2018.
The total impacts on the California economy are estimated
billion of additional gross output (sales revenue)
million of additional state/local tax revenues
million of additional federal tax revenues
additional full-time equivalent (FTE) jobs, half of which offer salaries
considerably higher than the state average
Millan, M.D., CIRM’s President and CEO, says the report reflects the Agency’s
role in building an ecosystem to accelerate the translation of important stem
cell science to solutions for patients with unmet medical needs. “CIRM’s
mission on behalf of patients has been the priority from day one, but this
report shows that CIRM funding brings additional benefits to the state. This report
reflects how CIRM is promoting economic growth in California by attracting
scientific talent and additional capital, and by creating an environment that
supports the development of businesses and commercial enterprises in the state”
In addition to the benefits to California, the impacts
outside of California on the US economy are estimated to be:
billion of additional gross output (sales revenue)
million of additional state (non-Californian) & local tax revenue
million of additional federal tax revenues
additional full-time equivalent (FTE) jobs
researchers summarize their findings, saying: “In terms of economic impacts, the
state’s investment in CIRM has paid handsome dividends in terms of output, employment,
and tax revenues for California.”
The estimates in the report are based on the economic stimulus
created by CIRM funding and by the co-funding that researchers and companies
were required to provide for clinical and late-stage preclinical projects. The
estimates also include:
Investments in CIRM-supported projects from private funders such
as equity investments, public offerings and mergers and acquisitions,
Follow-on funding from the National Institutes of Health and other
organizations due to data generated in CIRM-funded projects
Funding generated by clinical trials held at CIRM’s Alpha Stem
Cell Clinics network
researchers state “Nearly half of these impacts emanate from the $2.67 billion
CIRM grants themselves.”
economic impact of California’s investment in stem and regenerative cell
research is reflective of significant progress in this field that was just
being born at the time of CIRM’s creation,” says Dr. Millan. “We fund the most
promising projects based on rigorous science from basic research into clinical
trials. We partnered with researchers and companies to increase the likelihood
of success and created specialized infrastructure such as the Alpha Clinics
Network to support the highest quality of clinical care and research standards
for these novel approaches. The
ecosystem created by CIRM has attracted scientists, companies and capital from outside
the state to California. By supporting promising science projects early on,
long before most investors were ready to come aboard, we enabled our scientists
to make progress that positioned them to attract significant commercial
investments into their programs and into California.”
think one of the greatest strengths of CIRM has been their focus on development
of new stem cell therapies that can become real medicines,” says UCLA and
Orchard Therapeutics’ Don Kohn, M.D. “This has meant guiding academic
investigators to do the things that may be second nature in
industry/pharmaceutical companies but are not standard for basic or clinical
research. The support from CIRM to perform the studies and regulatory
activities needed to navigate therapies through the FDA and to form alliances
with biotech and pharma companies has allowed the stem cell gene therapy we
developed to treat SCID babies to be advanced and licensed to Orchard
Therapeutics who can make it available to patients across the country.”
support has been instrumental to our early successes and our ability to rapidly
progress Forty Seven’s CD47 antibody targeting approach with magrolimab,” says
Mark Chao, M.D., Ph.D., Founder and Vice President of Clinical Development at
Forty Seven Inc. “ CIRM was an early collaborator in our clinical
programs, and will continue to be a valued partner as we move forward with our
MDS/AML clinical trials.”
researchers say the money generated by partnerships and investments, what is
called “deal-flow funding”, is still growing and that the economic benefits
created by them are likely to continue for some time: “Deal-flow funding
usually involves several waves or rounds of capital infusion over many years,
and thus is it expected that CIRM’s past and current funding will attract
increasing amounts of industry investment and lead to additional spending
injections into the California economy in the years to come.”
They conclude their report by saying: “CIRM has led to
California stem cell research and development activities becoming a leader
among the states.”