Stem Cell Agency Invests in New Immunotherapy Approach to HIV, Plus Promising Projects Targeting Blindness and Leukemia

HIV AIDS

While we have made great progress in developing therapies that control the AIDS virus, HIV/AIDS remains a chronic condition and HIV medicines themselves can give rise to a new set of medical issues. That’s why the Board of the California Institute for Regenerative Medicine (CIRM) has awarded $3.8 million to a team from City of Hope to develop an HIV immunotherapy.

The City of Hope team, led by Xiuli Wang, is developing a chimeric antigen receptor T cell or CAR-T that will enable them to target and kill HIV Infection. These CAR-T cells are designed to respond to a vaccine to expand on demand to battle residual HIV as required.

Jeff Sheehy

CIRM Board member Jeff Sheehy

Jeff Sheehy, a CIRM Board member and patient advocate for HIV/AIDS, says there is a real need for a new approach.

“With 37 million people worldwide living with HIV, including one million Americans, a single treatment that cures is desperately needed.  An exciting feature of this approach is the way it is combined with the cytomegalovirus (CMV) vaccine. Making CAR T therapies safer and more efficient would not only help produce a new HIV treatment but would help with CAR T cancer therapies and could facilitate CAR T therapies for other diseases.”

This is a late stage pre-clinical program with a goal of developing the cell therapy and getting the data needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial.

The Board also approved three projects under its Translation Research Program, this is promising research that is building on basic scientific studies to hopefully create new therapies.

  • $5.068 million to University of California at Los Angeles’ Steven Schwartz to use a patient’s own adult cells to develop a treatment for diseases of the retina that can lead to blindness
  • $4.17 million to Karin Gaensler at the University of California at San Francisco to use a leukemia patient’s own cells to develop a vaccine that will stimulate their immune system to attack and destroy leukemia stem cells
  • Almost $4.24 million to Stanford’s Ted Leng to develop an off-the-shelf treatment for age-related macular degeneration (AMD), the leading cause of vision loss in the elderly.

The Board also approved funding for seven projects in the Discovery Quest Program. The Quest program promotes the discovery of promising new stem cell-based technologies that will be ready to move to the next level, the translational category, within two years, with an ultimate goal of improving patient care.

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

Finally, the Board approved the Agency’s 2019 research budget. Given CIRM’s new partnership with the National Heart, Lung, Blood Institute (NHLBI) to accelerate promising therapies that could help people with Sickle Cell Disease (SCD) the Agency is proposing to set aside $30 million in funding for this program.

barbara_lee_official_photo

Congresswoman Barbara Lee (D-CA 13th District)

“I am deeply grateful for organizations like CIRM and NHLBI that do vital work every day to help people struggling with Sickle Cell Disease,” said Congresswoman Barbara Lee (D-CA 13th District). “As a member of the House Appropriations Subcommittee on Labor, Health and Human Services, and Education, I know well the importance of this work. This innovative partnership between CIRM and NHLBI is an encouraging sign of progress, and I applaud both organizations for their tireless work to cure Sickle Cell Disease.”

Under the agreement CIRM and the NHLBI will coordinate efforts to identify and co-fund promising therapies targeting SCD.  Programs that are ready to start an IND-enabling or clinical trial project for sickle cell can apply to CIRM for funding from both agencies. CIRM will share application information with the NHLBI and CIRM’s Grants Working Group (GWG) – an independent panel of experts which reviews the scientific merits of applications – will review the applications and make recommendations. The NHLBI will then quickly decide if it wants to partner with CIRM on co-funding the project and if the CIRM governing Board approves the project for funding, the two organizations will agree on a cost-sharing partnership for the clinical trial. CIRM will then set the milestones and manage the single CIRM award and all monitoring of the project.

“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 treatments for patients suffering with Sickle Cell Disease” says Maria T. Millan, MD, President & CEO of CIRM. “This allows us to multiply the impact each dollar has to find relief for children and adults who battle with this life-threatening, disabling condition that results in a dramatically shortened lifespan.  We are pleased to be able to leverage CIRM’s acceleration model, expertise and infrastructure to partner with the NHLBI to find a cure for this condition that afflicts 100,000 Americans and millions around the globe.”

The budget for 2019 is:

Program type 2019
CLIN1 & 2

CLIN1& 2 Sickle Cell Disease

$93 million

$30 million

TRANSLATIONAL $20 million
DISCOVER $0
EDUCATION $600K

 

 

8 thoughts on “Stem Cell Agency Invests in New Immunotherapy Approach to HIV, Plus Promising Projects Targeting Blindness and Leukemia

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

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