The California Institute for Regenerative Medicine (CIRM) Board has approved funding for a range of stem cell and gene therapy programs.
The awards support projects targeting HIV, blindness, leukemia, hearing loss, autism, and sickle cell disease. Together, they reflect CIRM’s commitment to advancing promising discoveries toward new treatments for patients.
Advancing a Potential Cure for HIV
Despite major advances in HIV treatment, HIV/AIDS remains a lifelong condition for millions of people worldwide.
Current medications can control the virus, but they do not eliminate it. Long-term treatment can also create additional health challenges.
To address that need, the CIRM Board awarded $3.8 million to a team at City of Hope led by Xiuli Wang.
The team is developing an immunotherapy based on chimeric antigen receptor T cells, or CAR-T cells. Researchers have designed these engineered immune cells to recognize and destroy HIV-infected cells.
The therapy includes another innovative feature. Scientists designed the CAR-T cells to respond to a cytomegalovirus (CMV) vaccine. The vaccine can trigger the CAR-T cells to expand when needed and attack remaining HIV reservoirs.
Jeff Sheehy, a CIRM Board member and HIV/AIDS patient advocate, said the approach could have broad implications.
“With 37 million people worldwide living with HIV, including one million Americans, a single curative treatment is desperately needed,” said Sheehy.
He added that improving CAR-T technology could benefit HIV treatment, cancer therapies, and other diseases.
The project has reached the late preclinical stage. Researchers will use the funding to generate the data needed to seek FDA approval for a clinical trial.
Supporting the Next Generation of Therapies
The Board also approved three awards through CIRM’s Translation Research Program.
These projects build on promising scientific discoveries and move them closer to clinical application.
The approved awards include:
- $5.07 million to UCLA, led by Steven Schwartz, to develop a treatment for retinal diseases that can cause blindness using a patient’s own cells.
- $4.17 million to UCSF, led by Karin Gaensler, to create a vaccine that helps the immune system target leukemia stem cells using a patient’s own cells.
- Nearly $4.24 million to Stanford, led by Ted Leng, to develop an off-the-shelf treatment for age-related macular degeneration, a leading cause of vision loss among older adults.
Investing in Early-Stage Discovery
The Board also funded seven projects through the Discovery Quest Program.
The program helps researchers develop innovative stem cell technologies and prepare them for later-stage translational research.
The newly funded projects include:
| Application | Title | Institution | CIRM Committed Funding |
| DISC2-10979 | Universal Pluripotent Liver Failure Therapy (UPLiFT)
| Children’s Hospital of Los Angeles | $1,297,512
|
| DISC2-11105 | Pluripotent stem cell-derived bladder epithelial progenitors for definitive cell replacement therapy of bladder cancer
| Stanford | $1,415,016 |
| DISC2-10973 | Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases
| U.C. San Diego | $1,160,648 |
| DISC2-11070 | Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs
| Scripps | $1,827,576 |
| DISC2-11183 | A screen for drugs to protect against chemotherapy-induced hearing loss, using sensory hair cells derived by direct lineage reprogramming from hiPSCs
| University of Southern California | $833,971 |
| DISC2-11199 | Modulation of the Wnt pathway to restore inner ear function
| Stanford | $1,394,870 |
| DISC2-11109 | Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome
| Stanford | $1,415,016 |
These projects aim to generate the data needed to advance future therapies toward clinical development.
A New Partnership for Sickle Cell Disease
The Board also approved CIRM’s 2019 research budget, which includes a proposed $30 million investment in sickle cell disease programs.
The funding supports a new partnership between CIRM and the National Heart, Lung, and Blood Institute (NHLBI).
The collaboration will identify and co-fund promising cell and gene therapies for sickle cell disease. Researchers developing programs ready for IND-enabling studies or clinical trials can apply through a coordinated review process.
Under the partnership, CIRM and NHLBI will evaluate applications together and share funding responsibilities for selected projects.
Congresswoman Barbara Lee praised the collaboration and its potential impact on patients living with sickle cell disease.
Accelerating Progress Through Collaboration
CIRM President and CEO Dr. Maria T. Millan called the partnership a unique opportunity to advance potentially curative treatments.
“This is an extraordinary opportunity to create a first-of-its-kind partnership with the NHLBI to accelerate the development of curative cell and gene therapies for patients with sickle cell disease,” Dr. Millan said.
She noted that the partnership allows both organizations to maximize resources and accelerate progress for patients.
Sickle cell disease affects about 100,000 Americans and millions of people worldwide. The condition causes severe pain, organ damage, and a significantly shortened lifespan.
By combining expertise, funding, and infrastructure, CIRM and NHLBI hope to speed the development of therapies that could transform care for people living with this life-threatening disease.
Under the agreement CIRM and the NHLBI will coordinate efforts to identify and co-fund
The budget for 2019
| Program type | 2019 |
| CLIN1 & 2
CLIN1& 2 Sickle Cell Disease | $93 million
$30 million |
| TRANSLATIONAL | $20 million |
| DISCOVER | $0 |
| EDUCATION | $600K |
Greetings !
Personally, as a University student myself in the field of Biology, i am very excited by the extensive backing being shown here towards various fields of research using stem cells. It’s certainly been a controversial topic in the past but it’s something that can help so many people in the future. It definitely hits home for me, as I’ve had family members affected by cancer. Even concerning myself, I’ve had struggles with my vision since I was young, and it’s quite promising to see large investments being made to further the research concerning these and many other widespread diseases such as Sickle Cell and AIDS/HIV. It will certainly be a brighter future when individuals and their loved ones are able to access much more effective and efficient solutions.
Stem cells would be a great breakthrough in the scientific community and I fully support its research. Despite being controversial where ethics is concerned, stem cells have the ability to positively enhance our healthcare systems by being able to treat many of the incurable diseases that continue to plague our populations. It gives both the young and the old suffering from diseases such as cancer and Alzheimer’s a chance of living a full life. These diseases not only affect the patient physically but it also affects families emotionally. The total understanding of stem cells helps to eliminate one of the negative factors facing our society.
Ms. Constantine, you clearly recognize the potential of stem cell research as have I. I would like to know your thoughts on the Stem Cell Agency’s investment in new immunotherapy approach to HIV and projects targeting blindness and leukemia and its relation to stem cell research.
From the viewpoint of a student of biochemistry, stem cell research present an exciting cache of opportunities. these embryonic cells contain the instruction set for all cell types within the human body as well as the capability to read and select any of those instruction sets and carry them out. The opportunities presented to us, should we be able to understand how this is done can mean unlocking the secrets hidden from us throughout the year. We can understand why degenerative diseases start and find a way sound them, we would be able to reprogram cells susceptible to auto immune diseases and even potentially reset the cells of organs about to fail to a healthy condition.
As members of the scientific community, it is our duty to ensure that we advance the course of human life in some small way and stem cell research presents a pathway to fast tracking humanity to an era where there is no diseases, and no loss of loved ones to medical situations.
I truly do believe that stem cells hold a great value when it comes to medical application, due to the areas in which its applied to within the medical field and the variety of diseases that can be treated using such cells. Diseases that were once deemed incurable such as diabetes, Parkinson’s, Alzheimer’s, Heart and even spinal cord injury. These cells also play a great role in the formation of new skin tissue for those patients that have been severely burnt which is known ad stem cell grafting. It seem to me that these cells hold a great power with them and could open many doors in near future for science and medicine.
I think in the heat of the moment, people arguing against stem cell research forget that there are real people with real degenerative diseases that are literally (and that is by no means an exaggeration) suffering every single day. It baffles me tht morality is still in question when there are clear definite examples of stem cell research that are not only successful but also regularly practiced. In seeing all these reputable establishments not only figuratively but also financially supporting stem cell research, it gives me hope that light will be shed in this relative ‘grey area’. And with this light, where more and more practical uses of stem cell research will be uncovered and proven crucial in medical advancements, I hope that those against, even if they won’t agree, would acknowledge that this research helps much more people- living breathing people, than it hurts the blastocysts of potential human beings. And so while it is always important to understand the science behind these things, it is also very important to relate this knowledge to humanity and those who are truly affected by the consequences of our debates. People right now in this world need our help, and it is our moral obligation to help them.
The value of stem cell research is priceless, and its use in medicine will greatly affect the world in a positive way. Stem cells are a part of every human being and it and it helps to build and maintain our complex structure. They have tremendous regenerative capabilities and is used naturally to regenerate worn-out tissue. This provides a basis to cure degenerative diseases, such as the ones that cause blindness. Conversely, stem cells can regenerate uncontrollably and lead to various diseases such as the bone disease leukaemia. This uncontrolled stem cell proliferation may have resulted from genetic defects in the cells, thus it must be removed and replaced with healthy stem cells since these bone marrow (haematopoietic) stem cells are needed to form all blood cell types. The potential stem cells possess to cure many diseases unlike any other treatment is enough reason to pursue this research at all costs.
I think it is marvellous that an agency took the responsibility to fund research for a cure these debilitating and/or life-threatening ailments: HIV/AIDS, retinal disorders and leukaemia. A new approach to curing HIV/AIDS is needed since the lives of many people globally are still adversely affected by this seemingly incurable disease. Similarly, a new approach to curing retinal disorders and leukemia, since current treatment such as chemotherapy has other adverse side-effects. In my view, a cure without side effects for these ailments is desperately needed and no expense should be spared to find a cure. Perhaps stem cells would play a key role in these researchers’ pursuit of a cure for these disorders.
Stem cells play a pivotal and valuable role within human development. For medical applications I believe stem cells have an enormous potential and value. It has produced positive results in many fields of study. For example, stem cells have the potential to form cells and tissues for medical therapies. These cells can be used as a source of replacement cells to treat and cure diseases. And can potentially reduce the morbidity and mortality for those waiting for transplants. Due to the fact that areas of research where stem cells have benefited individuals suffering from diseases (Parkinson’s disease, Type 1 diabetes, Burn victims and Cardiovascular diseases), it is evidently clear that this science needs a push forward. And I think weighing up the potential benefits of embryonic stem cell research one must accept the costs and proceed in a carefully regulated context. As its current benefits are well documented, I strongly believe its continued research will pave the way the way for new treatments or perhaps reversal of chronic diseases.