As of this moment, there are over two million podcasts and over 48 million episodes to listen to on your favorite listening device. If you’re a true crime enthusiast like me, you’ve surely heard of Casefile or one of the other 94 podcasts on the topic. But what if you’re looking for something a little less ghastly and a little more uplifting?
The Stem Cell Podcast is an informative and entertaining resource for scientists and science enthusiasts (or really, anyone) interested in learning about the latest developments in stem cell research.
On their latest episode, dynamic co-hosts and research scientists Dr. Daylon James and Dr. Arun Sharma sit down with our President & CEO, Dr. Maria Millan, to discuss the impact of California’s culture of innovation on CIRM, the challenge of balancing hope vs. hype in the context of stem cell research/therapies, and the evolution of the agency over the past 15 years.
Listen on as Dr. Millan highlights some of CIRM’s greatest victories and shares our mission for the future.
About 10% of Americans suffer from knee osteoarthritis, a painful condition that can really impair mobility and quality of life. It’s often caused by an injury to cartilage, say when you were playing sports in high school or college, and over time it continues to degenerate and ultimately results in the loss of both cartilage and bone in the joint.
Current treatments involve either medication to control the pain or surgery. Medication works up to a point, but as the condition worsens it loses effectiveness. Knee replacement surgery can be effective, but is a serious, complicated procedure with a long recovery time. That’s why the governing Board of the California Institute for Regenerative Medicine (CIRM) voted to invest almost $6 million in an innovative stem cell therapy approach to helping restore articular cartilage in the knee.
Dr. Frank Petrigliano, Chief of the Epstein Family Center for Sports Medicine at Keck Medicine of the University of Southern California (USC), is using pluripotent stem cells to create chondrocytes (the cells responsible for cartilage formation) and then seeding those onto a scaffold. The scaffold is then surgically implanted at the site of damage in the knee. Based on scientific data, the seeded scaffold has the potential to regenerate the damaged cartilage, thus decreasing the likelihood of progression to knee osteoarthritis. In contrast to current methods, this new treatment could be an off-the-shelf approach that would be less costly, easier to administer, and might also reduce the likelihood of progression to osteoarthritis.
This is a late-stage pre-clinical program. The goals are to manufacture clinical grade product, carry out extensive studies to demonstrate safety of the approach, and then file an IND application with the FDA, requesting permission to test the product in a clinical trial in people.
“Damage to the cartilage in our knees can have a big impact on quality of life,” says Dr. Maria T. Millan, MD, President and CEO of CIRM. “It doesn’t just cause pain, it also creates problems carrying out simple, everyday activities such as walking, climbing stairs, bending, squatting and kneeling. Developing a way to repair or replace the damaged cartilage to prevent progression to knee osteoarthritis could make a major difference in the lives of millions of Americans. This program is a continuation of earlier stage work funded by CIRM at the Basic Biology and Translational stages, illustrating how CIRM supports scientific programs from early stages toward the clinic.”
Cancers of the blood, bone marrow and lymph nodes (also called hematologic malignancies) are the most common form of cancer in children and young adults. Current treatments can be effective but can also pose life-threatening health risks to the child. Now researchers at Stanford have developed a new approach and the Board of the California Institute for Regenerative Medicine (CIRM) voted to support that approach in a clinical trial.
The Board approved investing $11,996,634 in the study, which is the Stem Cell Agency’s 76th clinical trial.
The current standard of care for cancers such as acute leukemias and lymphomas is chemotherapy and a bone marrow (also called HSCT) transplant. However, without a perfectly matched donor the risk of the patient’s body rejecting the transplant is higher. Patients may also be at greater risk of graft vs host disease (GVHD), where the donor cells attack the patient’s body. In severe cases GVHD can be life-threatening.
Dr. Maria Grazia Roncarolo and her team at Stanford will test an immunotherapy cell approach using a therapy that is enriched with specialized immune cells called type 1 regulatory T (Tr1) cells. These cells will be infused into the patient following the bone marrow transplant. Both the Tr1 cells and the bone marrow will come from the same donor. The hope is this will help provide the patient’s immune system with these regulatory cells to combat life-threatening graft versus host disease and increase the success of treatment and bone marrow (HSCT) transplant.
“Every year around 500 children receive stem cell transplants in California, and while many children do well, too many experiences a rejection of the transplant or a relapse of the cancer,” says Dr. Maria T. Millan, President and CEO of CIRM. “Finding an improved therapy for these children means a shorter stay in the hospital, less risk of the need for a second transplant, and a greater quality of life for the child and the whole family.”
The CIRM Board has previously approved funding for 12 other clinical trials targeting cancers of the blood. You can read about them here.
Heart disease and stroke are two of the leading causes of death and disability and for people who have experienced either their treatment options are very limited. Current therapies focus on dealing with the immediate impact of the attack, but there is nothing to deal with the longer-term impact. The CIRM Board hopes to change that by funding promising work for both conditions.
Dr. Gary Steinberg and his team at Stanford were awarded almost $12 million to conduct a clinical trial to test a therapy for motor disabilities caused by chronic ischemic stroke. While “clot busting” therapies can treat strokes in their acute phase, immediately after they occur, these treatments can only be given within a few hours of the initial injury. There are no approved therapies to treat chronic stroke, the disabilities that remain in the months and years after the initial brain attack.
Dr. Steinberg will use embryonic stem cells that have been turned into neural stem cells (NSCs), a kind of stem cell that can form different cell types found in the brain. In a surgical procedure, the team will inject the NSCs directly into the brains of chronic stroke patients. While the ultimate goal of the therapy is to restore loss of movement in patients, this is just the first step in clinical trials for the therapy. This first-in-human trial will evaluate the therapy for safety and feasibility and look for signs that it is helping patients.
Another Stanford researcher, Dr. Crystal Mackall, was also awarded almost $12 million to conduct a clinical trial to test a treatment for children and young adults with glioma, a devastating, aggressive brain tumor that occurs primarily in children and young adults and originates in the brain. Such tumors are uniformly fatal and are the leading cause of childhood brain tumor-related death. Radiation therapy is a current treatment option, but it only extends survival by a few months.
Dr. Crystal Mackall and her team will modify a patient’s own T cells, an immune system cell that can destroy foreign or abnormal cells. The T cells will be modified with a protein called chimeric antigen receptor (CAR), which will give the newly created CAR-T cells the ability to identify and destroy the brain tumor cells. The CAR-T cells will be re-introduced back into patients and the therapy will be evaluated for safety and efficacy.
Stanford made it three in a row with the award of almost $7 million to Dr. Joe Wu to test a therapy for left-sided heart failure resulting from a heart attack. The major issue with this disease is that after a large number of heart muscle cells are killed or damaged by a heart attack, the adult heart has little ability to repair or replace these cells. Thus, rather than being able to replenish its supply of muscle cells, the heart forms a scar that can ultimately cause it to fail.
Dr. Wu will use human embryonic stem cells (hESCs) to generate cardiomyocytes (CM), a type of cell that makes up the heart muscle. The newly created hESC-CMs will then be administered to patients at the site of the heart muscle damage in a first-in-human trial. This initial trial will evaluate the safety and feasibility of the therapy, and the effect upon heart function will also be examined. The ultimate aim of this approach is to improve heart function for patients suffering from heart failure.
“We are pleased to add these clinical trials to CIRM’s portfolio,” says Maria T. Millan, M.D., President and CEO of CIRM. “Because of the reauthorization of CIRM under Proposition 14, we have now directly funded 75 clinical trials. The three grants approved bring forward regenerative medicine clinical trials for brain tumors, stroke, and heart failure, debilitating and fatal conditions where there are currently no definitive therapies or cures.”
Anyone who knows anything about CIRM knows about Bob Klein. He’s the main author and driving force behind both Proposition 71 and Proposition 14, the voter-approved ballot initiatives that first created and then refunded CIRM. It’s safe to say that without Bob there’d be no CIRM.
Recently we had the great good fortune to sit down with Bob to chat about the challenges of getting a proposition on the ballot in a time of pandemic and electoral pandemonium, what he thinks CIRM’s biggest achievements are (so far) and what his future plans are.
Yesterday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $8.39 million to the University of California, San Francisco (UCSF) to fund a clinical trial for sickle cell disease (SCD). An additional $51.08 million was awarded to fifteen community colleges and universities across California to fund undergraduate and master’s level programs that will help train the next generation of stem cell researchers.
SCD is an inherited blood disorder caused by a single gene mutation that changes a single base in the B globin gene leading to the production of defective hemoglobin that polymerizes and damages red blood cells thus the “sickle” shaped red blood cells. The damaged cells cause blood vessels to occlude/close up and that can lead to multiple organ damage as well as reduced quality of life and life expectancy.
Mark Walters, M.D., and his team at UCSF Benioff Children’s Hospital Oakland will be conducting a clinical trial that uses CRISPR-Cas9 gene editing technology to correct the genetic mutation in the blood stem cells of patients with severe SCD. The corrected blood stem cells will then be reintroduced back into patients with the goal of correcting the defective hemoglobin and thus producing functional, normal shaped red blood cells.
This clinical trial will be eligible for co-funding under the landmark agreement between CIRM and the National Heart, Lung, and Blood Institute (NHLBI) of the NIH. The CIRM-NHLBI agreement is intended to co-fund cell and gene therapy programs under the NHLBI’s “Cure Sickle Cell” initiative. The goal is to markedly accelerate the development of cell and gene therapies for SCD. CIRM has previously funded the preclinical development of this therapy through a Translational award as well as its IND-enabling studies through a Late Stage Preclinical award in partnership with NHLBI.
The CIRM Bridges to Stem Cell Research and Therapy program provides undergraduate and master’s students with the opportunity to take stem cell related courses and receive hands on experience and training in a stem cell research related laboratory at a university or biotechnology company. Fifteen institutions received a total of $51.08 million to carry out these programs to train the next generation of scientists.
The awards are summarized in the table below.
Bridges to Stem Cell Research and Therapy at Pasadena City College
Pasadena City College
CIRM Bridges to Stem Cell Research and Therapy Training Grant
CSU San Marcos
Bridges to Stem Cell Research Internship Program
San Diego State University
CIRM Bridges 3.0
CIRM Regenerative Medicine and Stem Cell Research Biotechnology Training Program
CSU Long Beach
Stem Cell Internships in Laboratory-based Learning (SCILL) continue to expand the scientific workforce for stem cells research and therapies.
San Jose State University
Strengthening the Pipeline of Master’s-level Scientific and Laboratory Personnel in Stem Cell Research
CIRM Bridges Science Master’s Program
San Francisco State University
CIRM Graduate Student Training in Stem Cell Sciences in the Stem Cell Technology and Lab Management Emphasis of the MS Biotechnology Program
CSU Channel Islands
CSUN CIRM Bridges 3.0 Stem Cell Research & Therapy Training Program
Stem Cell Scholars: a workforce development pipeline, educating, training and engaging students from basic research to clinical translation.
CSU San Bernardino
Training Master’s Students to Advance the Regenerative Medicine Field
Cal Poly San Luis Obispo
Building Career Pathways into Stem Cell Research and Therapy Development
City College of San Francisco
Bridges to Stem Cell Research and Therapy: A Talent Development Program for Training Diverse Undergraduates for Careers in Regenerative Medicine
CIRM Bridges to Stem Cell Research and Therapy
Berkeley City College
“We are pleased to fund a promising trial for sickle cell disease that uses the Nobel Prize winning gene editing technology CRISPR-Cas9,” says Maria T. Millan, M.D., President and CEO of CIRM. “This clinical trial is a testament to how the CIRM model supports promising early-stage research, accelerates it through translational development, and advances it into the clinics. As the field advances, we must also meet the demand for promising young scientists. The CIRM Bridges programs across the state of California will provide students with the tools and resources to begin their careers in regenerative medicine.”
When the voters of California approved Proposition 14 last November (thanks folks) they gave us $5.5 billion to continue the work we started way back in 2014. It’s a great honor, and a great responsibility.
It’s also a great opportunity to look at what we do and how we do it and try to come up with even better ways of funding groundbreaking research and helping create a new generation of researchers.
In addition to improving on what we already do, Prop 14 introduced some new elements, some new goals for us to add to the mix, and we are in the process of fleshing out how we can best do that.
Because of all these changes we decided it would be a good idea to hold a “Town Hall” meeting and let everyone know what these changes are and how they may impact applications for funding.
The Town Hall, on Tuesday June 29, was a great success with almost 200 participants. But we know that not everyone who wanted to attend could, so here’s the video of the event, and below that are the questions that were posed by people during the meeting, and the answers to those questions.
Having seen the video we would be eternally grateful if you could respond to a short online survey, to help us get a better idea of your research and education needs and to be better able to serve you and identify potential areas of opportunity for CIRM. Here’s a link to that survey: https://www.surveymonkey.com/r/VQMYPDL
We know that there may be issues or questions that are not answered here, so feel free to send those to us at email@example.com and we will make sure you get an answer.
Are there any DISC funding opportunities specific to early-stage investigators?
DISC funding opportunities are open to all investigators. There aren’t any that are specific to junior investigators.
Are DISC funding opportunities available for early-mid career researchers based out of USA such as Australia?
Sorry, you have to be in California for us to fund your work.
Does tumor immunology/ cancer immunotherapy fall within the scope of the CIRM discovery grants?
CIRM funding supports non-profit academic grantees as well as companies of all sizes.
I am studying stem cells using mouse. Is my research eligible for the CIRM grants?
Yes it is.
Your programs more specifically into stem cell research would be willing to take patients that are not from California?
Yes, we have treated patients who are not in California. Some have come to California for treatment and others have been treated in other states in the US by companies that are based here in California.
Can you elaborate how the preview of the proposals works? Who reviews them and what are the criteria for full review?
The same GWG panel both previews and conducts the full review. The panel first looks through all the applications to identify what each reviewer believes represents the most likely to be impactful and meet the goals of the CIRM Discovery program. Those that are selected by any reviewer moves forward to the next full review step.
If you meet your milestones-How likely is it that a DISC recipient gets a TRAN award?
The milestones are geared toward preparation of the TRAN stage. However, this is a different application and review that is not guaranteed to result in funding.
Regarding Manufacturing Public Private partnerships – What specific activities is CIRM thinking about enabling these partnerships? For example, are out of state for profit commercial entities able to conduct manufacturing at CA based manufacturing centers even though the clinical program may be primarily based out of CA? If so, what percent of the total program budget must be expended in CA? How will CIRM enable GMP manufacturing centers interact with commercial entities?
We are in the early stages of developing this concept with continued input from various stakeholders. The preliminary vision is to build a network of academic GMP manufacturing centers and industry partners to support the manufacturing needs of CIRM-funded projects in California.
We are in the process of widely distributing a summary of the manufacturing workshop. Here’s a link to it:
If a center is interested in being a sharing lab or competency hub with CIRM, how would they go about it?
CIRM will be soliciting applications for Shared Labs/Competency hubs in potential future RFAs. The survey asks several questions asking for feedback on these concepts so it would really help us if you could complete the survey.
Would preclinical development of stem cell secretome-derived protein therapies for rare neuromuscular diseases and ultimately, age-related muscle wasting be eligible for CIRM TRAN1 funding? The goal is to complete IND-enabling studies for a protein-based therapy that enhances tissue regeneration to treat a rare degenerative disease. the screening to identify the stem-cell secreted proteins to develop as therapeutics is done by in vitro screening with aged/diseased primary human progenitor cells to identify candidates that enhance their differentiation . In vivo the protein therapeutic signals to several cell types , including precursor cells to improve tissue homeostasis.
I would suggest reaching out to our Translation team to discuss the details as it will depend on several factors. You can email the team at firstname.lastname@example.org
When Proposition 14 was approved by voters in November we were given a chance to carry on the work we have been doing for more than 16 years. What we hadn’t anticipated was that we would also get a chance to do that with some of the team that helped us make CIRM what it is, but who had since moved on to other jobs.
We are delighted to say that as we build up our team again we are welcoming back a couple of dear friends, and welcoming in some new ones too. They’re a talented bunch and, if they don’t mind me saying so, a darned good looking group too.
Rosa Canet-Aviles, PhD., has been named as the new Vice President Scientific Programs. Rosa is a familiar face at the agency, serving as a Science Officer with CIRM from 2008 to 2014. During that time she helped oversee the development of our Translational program, managed a broad portfolio of projects and organized workshops on Parkinson’s and autism.
After leaving CIRM she joined the Foundation for the National Institutes of Health (FINH) where she served as the Director of Neuroscience Research Partnerships. In that role she led the successful development and management of 5 new large partnerships including the Biomarkers Consortium Neuroscience Steering Committee, the Accelerating Medicines Partnership (AMP) for Alzheimer’s disease 1.0 and 2.0, AMP Parkinson’s disease and AMP Schizophrenia.
Rosa has more than 15 years of experience working in industry, academia and government and her experience in developing and managing neuroscience programs will be invaluable as CIRM looks to invest some $1.5 billion in neuroscience under Proposition 14.
“I am very excited to be back,” says Rosa. ”It is a dream come true being able to translate all the skills, learning and networks gathered over the past 7 years towards the development and implementation of CIRM’s new phase and accelerate stem cell therapies for patients in need.”
“We are thrilled to announce the timely return of Rosa to CIRM as we build our new strategic plan under Prop 14,” says Dr. Maria T. Millan, CIRM’s President & CEO. “Rosa has demonstrated time and again the unique ability to bring together often seemingly disparate stakeholders to successfully drive toward a common goal of advancing the science on behalf of patients with diseases of the brain and neuropsychiatric disorders. At CIRM, she assembled key international leaders who went on to form an international Parkinson’s Disease consortium. At the Foundation for NIH (FNIH), she directed the development of five prominent public-private partnerships. A neuroscientist by training, she is held in high regard and has been called a “quick study” in her ability to lead in new areas such as in genomics and data science, key components of her role at FNIH and at Eisai’s Center for Genetics Guided Dementia Discovery.“
In addition, CIRM is pleased to announce the following new team members:
Uta Grieshammer, PhD. is also returning to CIRM as the Senior Science Officer for our Discovery program. Uta was at CIRM from 2007 to 2015 and led the programs that created both our Genomics Initiative and our iPSC bank. She also organized several scientific conferences and workshops involving hundreds of CIRM-funded researchers.
After leaving CIRM she became the Scientific Director of the California Initiative to Advance Precision Medicine at the University of California San Francisco where she created and managed the application and peer review process. Most recently she was the Program Officer at the University of California Office of the President’s (UCOP) Tobacco Related Disease Research Program where she focused on the neuroscience of nicotine addiction. She also helped develop a scholarship program to attract students from diverse backgrounds to pursue a career in science.
Michael Bunch joins CIRM as a Business Service Officer. Michael is a decorated veteran who has been working as the Chief Business Officer at the Veterans Home in Yountville, California. In that role he implemented new contract and reviewing processes and oversaw the income and insurance tracking for some 1,000 residents. With his extensive background in acquisition management, contingency contracting, and his deep knowledge of state regulations and guidelines Michael was able to increase funding, streamline processes and assist Veterans and their families to obtain the benefits and services that they qualified for.
Michael spent 25 years in the US Army including serving as part of the NATO peacekeeping force in Kosovo. During that deployment he was awarded the Joint Service Commendation Medal (JSCM) for managing the fuel needs of 4 Multinational Task Forces and 33 Nations, an essential element in helping the mission succeed.
A Senior Drill Sergeant, Infantry Instructor and Financial and Resource Manager Michael has been awarded the Army Commendation Medal with 4 Oak Leaves, Army Achievement Medal with 4 Oak Leaves, Global War on Terrorism Service Medal, KOSOVO Campaign Medal, Military Outstanding Volunteer Service Medal, NATO Medal, Expert Infantryman Badge, Honorary Kentucky Colonel and Honorary Kentucky Admiral.
Nellie Almazan joins CIRM as a Grants Management Specialist. Nellie comes to us from the California Department of Transportation (Caltrans) where she has worked for 16 years, most recently as the Associate Transportation Planner with the Low Carbon Transit Operations program. Nellie managed more than 150 projects, reviewing grants to help reduce greenhouse gas emissions in the state and overseeing programs that had an emphasis on serving Disadvantaged Communities.
She is currently enrolled at Sacramento City College where her focus is on Sociology and Deaf Culture.
Alexandra Caraballo joins CIRM as a Grants Management Specialist. Alex has more than 15 years of grant administration experience with a focus on incorporating equity, diversity, and inclusion into grantmaking practices and decision-making. She comes to CIRM from the Kaiser Foundation Health Plan where she was the National Manager of Philanthropy. There she was responsible for the administration of approximately 200 grants in the national community health portfolio. Before Kaiser she was the Program Assistant and Associate Program Officer at the East Bay Community Foundation, where she partnered with donors and community-based organizations to advance racial equity and transform political, social and economic outcomes for East Bay Communities.
Alex currently serves on the Board of Directors for the Lindsay Wildlife Experience and was a former Advisory Board member for Oakland Head Start.
Following the passage of Proposition 14 CIRM has hired five new employees to help increase the team’s ability to respond to new challenges and responsibilities.
Prop 14, which was approved by voters in November 2020, gives CIRM $5.5 billion in new funding. Those funds mean CIRM can once again fund research from Discovery, through Translational and Clinical, as well as create new education and training programs. Prop 14 also adds new areas of focus for the Stem Cell Agency including creating an Accessibility and Affordability Working Group, expanding the Alpha Stem Cell Clinic network and creating new Centers of Excellence in underserved parts of California. To meet those new responsibilities the Agency has hired a highly talented group of individuals. Those include:
Kevin Marks is CIRM’s new General Counsel. Kevin studied Russian at college and originally wanted to be a diplomat, but when that didn’t work out he turned to the law. He became a highly accomplished, skilled advisor with global and domestic expertise and a history of delivering innovative legal and business results. He has served as Vice President and Head of Legal and Compliance at Roche Molecular Solutions, VP and General Counsel at Roche Molecular Diagnostics and VP and General Counsel at Roche Palo Alto, LLC.
“We are so delighted to have Kevin Marks join CIRM as a member of our executive Leadership Team,” says Maria T. Millan, MD, CIRM’s President and CEO. “He brings unique qualifications and critical skills during the formative phase and launch of our new strategic plan for California’s $5.5B investment in stem cell, genomics and regenerative medicine research and therapy development. As general counsel, he will oversee the legal department, human resources, grants management and operations at the Agency. Kevin has an established track record with global and domestic expertise and a history of delivering innovative legal and business solutions.”
“He is revered by his colleagues as an exceptional leader in his profession and in the community. Kevin is known for developing people as well as programs, and for promoting racial, ethnic and gender diversity.”
“I am incredibly honored to be joining CIRM at this stage of its journey,” says Marks. “I see the opportunity to contribute to positive patient outcomes–especially those patients with unmet medical needs–by working towards accelerating stem cell research in California as a member of the CIRM team as rewarding and perfectly aligned with my professional and personal goals.”
Pouneh Simpson as Director of Finance. Pouneh comes to CIRM from the Governor’s Office of Emergency Services in California, where she served as the Recovery Financial Administration Chief. At OES she worked with local, state, and federal government stakeholders on disaster recovery planning, exercises, and grant administration, specifically, overseeing the grant processing of all disaster recovery activity.
Prior to that Pouneh worked as the Chief Financial Officer of the Veterans Homes, where she managed finances at eight Veterans Homes with over 2,800 positions and $365 million in General Fund support. She also led the writing of legislation, regulations, policies and procedures for Cal Vet, overhauling the business and financial portions of eight Veterans Homes.
Mitra Hooshmand, PhD. as Senior Science Officer for Special Projects and Initiatives. Mitra joins CIRM after more than five years of leadership experience at Americans for Cures, a stem cell advocacy group. During this time, she mobilized hundreds of stakeholders, from scientists to national and local patient advocacy organizations, to generate support for CIRM’s mission.
Mitra has a PhD. in Anatomy and Biology from the University of California at Irvine. She also worked as a Project Scientist at the Sue and Bill Gross Stem Cell Research Center at UC Irvine, where she conducted and published academic and industry-partnered research in studies investigating the therapeutic benefit of human neural stem cell transplantation in preclinical models of spinal cord injury and aging.
Vanessa Singh, as Human Resources Manager. Vanessa has 15 years of experience working for the state of California, working at the Departments of General Services, Insurance and Human Services. In those roles she gained experience in performing, processing, developing, implementing, and advising on many personnel aspects such as compensation, benefits, classifying positions, recruitment and hiring, salary structure (exempt and civil service), organization structure, and retirement.
When COVID struck Vanessa stepped up to help. She worked as a Case Investigator for San Bernardino Local Health Jurisdiction, Department of Public Health, doing contact tracing. She talked to people diagnosed with coronavirus and collected information on people they may have had close contact with who may have been exposed to the virus.
Claudette Mandac as Project Manager Review. Claudette has more than seven years’ experience with UCSF’s Human Research Protection Program. In that role she prepared protocols for scientific, regulatory and ethical review, pre-screening submissions to ensure they were complete and consistent, and then routing them to the appropriate reviewers for administrative, expedited or Committee review. She also managed an Institutional Review Board Committee, preparing and distributing protocols for review by designated scientific and nonscientific reviewers, coordinating meetings, recruiting and training members, and maintaining records of conflicts of interest. At UCSF she annually helped process up to 3,000 IRB modifications, continuing reviews, and post-approval safety reports for domestic and international socio-behavioral or biomedical research.
Claudette has two degrees from U.C. Berkeley; one in Arts and History and another in Science, Conservation and Resource Studies.
A CIRM-funded clinical trial to help kidney transplant patients avoid the need for anti-rejection or immunosuppressive medications has completed enrollment and transplantation of all patients.
Medeor Therapeutics’ MDR-101 Phase 3 multi-center clinical trial involved 30 patients; 20 of them were treated with MDR-101, and 10 control subjects were given standard care. CIRM awarded Medeor, based in South San Francisco, $18.8 million for this research in January 2018.
More than 650,000 Americans suffer from end-stage kidney disease – a life-threatening condition caused by the loss of kidney function. For these people the best treatment option is a kidney transplant from a genetically matched, living donor. Even matched patients, however, face a lifetime on immunosuppressive drugs to prevent their immune system from rejecting the transplanted organ. These drugs can be effective at preventing rejection, but they come at a cost. Because they are toxic these medications increase a transplant patient’s life-time risk of cancer, diabetes, heart disease and infections.
Medeor Therapeutics developed its MDR-101 therapy to reprogram the patient’s immune system to accept a transplanted kidney without the need for long term use of immunosuppression drugs.
The company takes peripheral blood stem cells from the organ donor and infuses them into the patient receiving the donor’s kidney. This creates a condition called “mixed chimerism” where immune cells from the donor help the patient’s immune system adapt to and tolerate the donor’s kidney.
After a standard kidney transplant, the patient is given a combination of three anti-rejection medications which they typically have to remain on for the rest of their lives. However, the Medeor patients, by day 40 post-transplant, are only taking one medication and the hope is that immunosuppression is discontinued at the end of one year.
“Chronic kidney disease and kidney failure are a growing problem in the US, that’s why it’s so important that we find new ways to reduce the burden on patients and increase the odds of a successful transplant with long term benefit,” says Maria T. Millan, M.D., President and CEO of CIRM. “Medeor’s approach may not only reduce the likelihood of a patient’s body rejecting the transplanted organ, but it can also improve the quality of life for these people and reduce overall health care costs by eliminating the need to stay on these immunosuppressive medications for life.”
In an earlier Phase 2 trial, a majority of patients achieved mixed chimerism. Approximately 74 percent of those patients have been off all immunosuppressive drugs for more than two years, including some who continue to be off immunosuppressive medications 15 years after their surgery.
“Today’s news is a tremendous milestone not only for Medeor but for the entire transplant community. This is the first randomized, multi-center pivotal study designed specifically to stop the use of all immunosuppressive anti-rejection drugs post-transplant. This therapy can be a true game changer in our efforts to transform transplant outcomes and help patients live healthier lives,” said Dan Brennan, MD, Chief Medical Officer at Medeor Therapeutics.
If the results from this pivotal clinical trial show that MDR-101 is both safe and effective, Medeor may apply to the Food and Drug Administration (FDA) for approval to market their approach to other patients in the U.S.