Author: Esteban Cortez

CIRM commits $60 million for Parkinson’s research, joins #Take6ForPD awareness campaign 

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April is Parkinson’s Awareness Month and the California Institute for Regenerative Medicine (CIRM) is joining the campaign to #Take6ForPD—take 6 minutes to raise Parkinson’s awareness and highlight our commitment to funding research for the disease. 

What is Parkinson’s Disease? 

Parkinson’s disease (PD) is a neurodegenerative disease that affects nearly 1 million people in the United States and more than 10 million people around the world. In California, more than 106,000 people live with PD. 

Symptoms of PD include tremors, slow movement, muscle rigidity, balance issues and lack of facial expressions. Parkinson’s disease occurs when the neurons or nerve cells in the portion of the brain that controls movement die off. These neurons send signals by releasing a chemical called dopamine and are referred to as dopaminergic neurons.  

Parkinson’s is the second-most common neurodegenerative disease after Alzheimer’s disease. No cure exists for the disease. 

CIRM’s Commitment to Funding Parkinson’s Research

CIRM remains committed to funding regenerative medicine research—including stem cell and gene therapy research—for PD treatments to improve quality of life for patients.  

To date, CIRM has invested more than $60 million in helping research for PD progress from a basic or Discovery level through clinical trials. 

This year, CIRM awarded $4 million to a late-stage preclinical project by Ryne Bio aiming to improve treatment for idiopathic PD. In the proposed stem cell therapy, investigators at Ryne Bio are aiming to deliver dopamine-producing cells to replace the lost neurons to the brain of PD patients to restore or improve motor function. 

“Receiving a Parkinson’s diagnosis is a difficult and life-changing event,” says Ryne Bio CEO Nick Manusos. “This Parkinson’s Awareness Month, we honor patients and their loved ones by reflecting on the clinical progress made so far, and looking forward to a brighter, healthier future.” 

Other regenerative medicine approaches CIRM has funded to target PD involve understanding the disease and looking for new drugs to treat it.  

CIRM grantees have taken skin cells from people with Parkinson’s disease, reprogrammed them back to an embryonic-like state, turning them into the kind of stem cell that can be transformed into any other cell in the body, then coaxing those cells to become dopaminergic neurons that are lost to the disease.  

Those cells showed signs of the disease in the lab dish and were distinctly different from the same cells created from people without PD.  

Another approach involves using CRISPR gene editing technology to reduce the levels of a toxic protein called alpha synuclein, which builds up in the dopaminergic brain cells affected by PD. 

Overall, CIRM has awarded more than 30 grants aiming to better understand the disease and find regenerative medicine-based treatments.  

“CIRM’s commitment to funding research for Parkinson’s is unwavering,” says CIRM President and CEO Maria Millan. “Our goal is to fund the most promising regenerative medicine research to support finding the best treatments to fight this devastating disease that affects millions.” 

CIRM’s Task Force on Neuroscience & Medicine 

As part of its Strategic Plan, CIRM has also launched a Task Force on Neuroscience and Medicine to set a plan for the $1.5 billion allocated for the support of research and the development of treatments for diseases and conditions of the brain and central nervous system. These include Parkinson’s, Alzheimer’s, stroke, dementia, epilepsy and other diseases and conditions. 

The Task Force will host public meetings to gather community input to identify potentially high-impact opportunities in basic neuroscience, neurodegenerative disease, neuropsychiatric disease, neurodevelopment, and normal brain aging.   

The goal of the task force is to provide final recommendations to CIRM and its governing board within six months of inception. 

To see a listing of upcoming and past Task Force meetings, visit our website. 

CIRM welcomes new Chair, bids farewell to Art Torres and Jonathan Thomas

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Dr. Vito Imbasciani was sworn in as the new Chair of the Board of the California Institute for Regenerative Medicine (CIRM) at its March meeting.

Imbasciani (pictured above at left) was elected to the six-year term at CIRM’s January Board meeting. He will replace outgoing chair Jonathan Thomas, who has served in the position since 2011. 

“My experience has positioned me to champion the aims of CIRM, advocate for it cogently, and represent it responsibly before the public and their state and federal elected representatives,” Imbasciani said. “I look forward to the challenge of advancing the groundbreaking work of this Agency, at the same time nourishing the hopes for medical advances held by the citizens of our great State.”  

Imbasciani has served as the Secretary of the California Department of Veterans Affairs (CalVet) since 2015. As Secretary, he created several new programs within the department, including forging eight independent California veteran homes into a unified system, establishing programs for veterans in state prisons, and supporting the 58 county veteran service offices. 

Current CIRM Vice Chair Maria Bonneville, Fmr. Vice Chair Sen. Art Torres (ret). and Fmr. Chair Jonathan Thomas

CIRM also bid farewell to Sen. Art Torres (ret) and Jonathan Thomas (also known as JT) for their service on our Board.

JT was elected as Chair of the Board in 2011 after he was nominated by then-Governor Jerry Brown, Treasurer Bill Lockyer, and Lieutenant Governor Gavin Newsom.

He served as chair of the Governing Board for more than 12 years. In that time, he led CIRM in granting $2.5 billion in grants to support groundbreaking research and discovery to advance stem cell research and accelerate stem cell treatments to patients with unmet medical needs.

He led the agency as it expanded its work with industry, revamped its award processes, prepared for the expiration of bond funding, supported the drafting of Proposition 14, and planned for the next phase of CIRM’s programs after the voters approved $5.5 billion in additional funding.

Senator Torres was nominated in 2009 as CIRM Vice Chair by then-Lieutenant Governor Gavin Newsom and Treasurer Bill Lockyer, and shortly after was elected by the CIRM Governing Board.

Senator Torres served on the Grants and Standards working groups, and served as the inaugural Chair of the Accessibility and Affordability Working Group. He served on numerous subcommittees, led CIRM’s government relations efforts, and played a pivotal role in launching CIRM’s SPARK high school internship program.

CIRM Vice Chair Maria Bonneville, Fmr. Chair Jonathan Thomas, Chair Vito Imbasciani, and President & CEO Maria Millan

CIRM expresses its deepest gratitude to Senator Torres and JT for their service on its Governing Board and for their dedication to the advancement of stem cell research and our mission to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse California and world.

Investing in stem cell and gene therapy treatments for HIV

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A recent article in Nature shared the news about a 53-year-old man in Germany who was declared free of HIV after receiving virus-resistant cells. 

The man—referred to as the “Düsseldorf patient”—was diagnosed with acute myeloid leukemia and underwent a stem cell transplant in 2013 that replaced his bone marrow cells with HIV-resistant stem cells from a donor.  

In the five years following the procedure, virologist Björn-Erik Jensen and his team at Düsseldorf University Hospital in Germany continued to monitor the patient. They continued to find immune cells that reacted to HIV in his body, which suggested that his body was not clear of the virus. 

In 2018, the patient stopped taking antiretroviral therapy (ART), a treatment for HIV that reduces a person’s viral load to an undetectable level. His body has remained HIV-free since then, indicating that the stem cell transplant worked.  

Similar stem cell treatments have been used to treat others living with HIV, including a patient in 2007 and another patient in 2019. 

The article in Nature states that the procedure is unlikely to be used widely in its current form due to the associated risks, including the chance that an individual will reject a donor’s marrow.  

Scientists continue to test stem cells as a treatment for HIV, including methods in which cells are taken from a person’s own body and genetically modified, eliminating the need for donor cells. 

CIRM’s Commitment to Investing in Treatments for HIV

The news of the Düsseldorf patient shows the importance of continued stem cell and gene therapy research to find treatments for HIV.  

At the California Institute for Regenerative Medicine (CIRM), we have invested more than $85 million in the search for stem cell and gene therapy treatments for HIV/AIDS, ranging from basic Discovery research to clinical trials.  

Recent CIRM investments include a study at UC Davis health, in which researchers take a patient’s own white blood cells, called T-cells, and modify them so that they can identify and target HIV cells to control the virus without medication. 

CIRM also funded a clinical trial at UCSF to develop a functional cure for HIV/AIDS. In the trial, the team takes a patient’s blood and extracts T cells, a type of immune cell. The T cells are then genetically modified to express two different chimeric antigen receptors (CAR), which enable the newly-created duoCAR-T cells to recognize and destroy HIV infected cells. The modified T cells are then reintroduced back into the patient. 

The goal of this one-time therapy is to act as a long-term control of HIV with patients no longer needing to take ART, in effect a form of HIV cure.  This approach would also address the needs of those who are not able to respond to current approaches, which is estimated to be 50% of those affected by HIV globally. 

Last year, researchers in the UCSF trial shared that they had dosed the first patient in the trial testing their anti-HIV duoCAR-T cell therapy. You can read the announcement here.  

There are approximately 38 million people worldwide living with HIV/AIDS. And each year there are an estimated 1.5 million new cases. The vast majority of those living with HIV do not have access to the life-saving antiretroviral medications that can keep the virus under control. People who do have access to the medications face long-term complications from them including heart disease, bone, liver and kidney problems, and changes in metabolism. 

To learn more about CIRM’s commitment and investments in finding treatments for HIV, visit our website. 

CIRM board member Ysabel Duron appointed to National Cancer Advisory Board by President Biden

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Ysabel Duron is an award-winning journalist, patient advocate, cancer survivor and board member of the California Institute for Regenerative Medicine (CIRM). 

Her list of achievements continues to grow, as President Biden has appointed Duron to National Cancer Advisory Board (NCAB), which plays an important role in setting the course for the national cancer research program. 

The National Cancer Advisory Board will complement the Cancer Moonshot, which President Biden reignited a year ago to invest in research and development that will help advance breakthroughs to prevent, detect and treat diseases like cancer. 

“As a Latina, and a long-time patient and community advocate, it humbles me to join this roster of stellar new appointees,” Duron said. “I look forward to the challenge of amplifying the voices of racial and ethnic communities and other vulnerable populations.” 

Duron came into the cancer space after her own bout with Hodgkins Lymphoma in 1999. She covered her own cancer battle using her reporting skills to raise awareness about the disease.  

Over time, she turned a spotlight on the many disparities—lack of access, income inequality, language barriers, among other social determinants on health—that has exacerbated the disproportionate burden of cancer in Latino communities. 

In 2017, Ms. Duron founded The Latino Cancer Institute (TLCI), a nationwide network dedicated to developing and sharing best practice programs to enhance the work of Latino community service agencies, to provide collaboration with the global cancer research community, and drive policy to solve the issues and burden of Latinx/Hispanic cancer. 

In addition to her new appointment to the NCAB and role as Board member at CIRM, Duron also serves on the Institutional Review Board for the NIH/All of Us Research program. She also recently joined the newly launched American Cancer Society National Breast Cancer Roundtable. 

Read the official White House press release here.

California agency invests $4 million in stem cell treatment for Parkinson’s Disease

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The California Institute for Regenerative Medicine (CIRM) is investing $4 million in a late-stage preclinical project by Ryne Bio aiming to improve treatment for Idiopathic Parkinson’s disease (PD).

PD is characterized by a loss of dopamine producing neurons that result in motor symptoms, such as dyskinesias (involuntary, erratic, writhing movements of the face, arms, legs or trunk) and non-motor effects such as dementia, depression and sleep disorders.

PD is the second-most common neurodegenerative disease after Alzheimer’s disease affecting approximately 1 million people in the U.S. In California, it is estimated that 116,900 people live with PD, representing the highest number of people with the disease in the country.

At its early stages, PD can be treated with medication such as Levodopa to treat symptoms but these become less effective as the disease progresses.

The proposed stem cell therapy in this project offers the potential to restore dopamine neurons, which play a role in many important body functions, including movement and memory.

Investigators at Ryne Bio are aiming to deliver dopamine producing cells to replace the lost neurons to the brain of Parkinson’s disease patients to restore/improve motor function.

The current grant is being funded to conduct Investigational New Drug (IND) enabling, nonclinical safety studies per the US Food and Drug Administration (FDA) Guidance. The IND is the authorization needed to begin a clinical trial in Parkinson’s patients.

CIRM has a vested interest in seeing this therapy succeed. To date, CIRM has invested more than $59 million in helping research for Parkinson’s disease progress from a basic or Discovery level through clinical trials.

Funding development of a vaccine for acute myelogenous leukemia (AML)

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Dr. Karin Gaensler. Photo credit: Steve Babuljak/UCSF

Adult acute myelogenous leukemia—also known as acute myeloid leukemia (AML)—is a blood cancer in which the bone marrow makes a large number of abnormal blood cells. 

About 20,000 new cases of AML are diagnosed each year in the US with a 5-year survival rate of around 29%. In 2022, there were nearly 12,000 deaths from AML. Many AML patients—a majority of which are over 60 years old—relapse after treatment. Blood stem cell transplant can be curative, but many older patients do not qualify, showing that there is a significant unmet medical need in treating AML. 

That’s why the California Institute for Regenerative Medicine (CIRM) awarded $6,000,000 to Dr. Karin Gaensler at the University of California, San Francisco (UCSF) to support development of a safe and effective vaccine for the blood cancer AML to improve relapse-free survival. 

To develop the cancer vaccine, Dr. Gaensler and her team will engineer the patient’s blood stem cells to maximize stimulation of leukemia-specific killing activity and reintroduce engineered cells back to the patient to target and kill residual leukemia stem cells.  

This approach holds the potential for long-term effectiveness as it targets both AML blasts and leukemic stem cells that are often the source of relapse.  

This award is a continuation of a previous CIRM grant that will support the manufacture of the vaccine and the completion of late-stage testing and preparation needed to apply to the US Food and Drug Administration (FDA) for permission to begin a clinical trial. 

The Most Read Stem Cellar Blog Posts of 2022

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This year was a momentous one for the California Institute for Regenerative Medicine (CIRM). You can read some of our achievements in our 2021-2022 annual report.  

As always, we shared our most exciting updates and newsworthy stories—topics ranging from stem cell research to diversity in science—right here on The Stem Cellar. More than 100,000 blog visitors followed along throughout the year!  

In case you missed them, here’s a recap of our most popular blogs of 2022. We look forward to covering even more topics in 2023 and send a sincere thank you to our wonderful Stem Cellar readers for tuning in!   

In Memory of Kevin McCormack 

We cannot close out the year without honoring our dear friend and colleague Kevin McCormack, who passed away suddenly in December. Kevin was CIRM’s Director of Patient Advocacy and loved writing for The Stem Cellar. He did a wonderful job in translating complex science for the general public and was a great mentor to the CIRM team. Many of his closest friends and colleagues wrote memories about him, and we compiled them in this blog post honoring his life and dedication to CIRM and patients everywhere.  

How stem cells helped Veronica fight retinitis pigmentosa and regain her vision 

We shared the story of Veronica McDougall, who thought everyone saw the world the way she did: blurry, slightly out-of-focus and with tunnel vision. When she was 24, she went to see a specialist who told her she had retinitis pigmentosa, a rare degenerative condition that would eventually leave her legally blind. Click through to read about her experience participating in a CIRM-funded clinical trial with a company called jCyte. 

Smoking marijuana could be bad for your heart, but there is an unusual remedy 

Millions of Americans use marijuana for medical reasons, such as reducing anxiety or helping ease the side effects of cancer therapy. Millions more turn to it for recreational reasons, saying it helps them relax. Now a new study says those who smoke marijuana regularly might be putting themselves at increased risk of heart disease and heart attack. Check out this blog to learn how a team at Stanford Medicine used the iPSC method to create human endothelial cells and, in the lab, found that THC appeared to promote inflammation in the cells. 

A pioneering couple uproot their lives to help their baby 

This year, we shared some encouraging news about a CIRM-funded stem cell clinical trial for spina bifida at UC Davis Health. Spina bifida is a birth defect that occurs when the spine and spinal cord don’t form properly and can result in life-long walking and mobility problems for the child, even paralysis. This blog told the story of parents Michelle Johnson and Jeff Maginnis, who learned 20 weeks into the pregnancy that the fetus had spina bifida. Read the whole story to learn about their experience and the status of their baby Tobi.  

And that wraps up The Stem Cellar’s top blog posts of 2022! If you’re looking for more ways to get the latest updates from The Stem Cellar and CIRM, follow us on social media on Facebook, Twitter, LinkedIn, and Instagram.

Finding a treatment for Tay-Sachs disease

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The California Institute for Regenerative Medicine (CIRM) has awarded $4,048,253 to Dr. Joseph Anderson and his team at UC Davis to develop a blood stem cell gene therapy for the treatment of Tay-Sachs disease.  

Tay-Sachs disease is a rare genetic disorder where a deficiency in the Hex A gene results in excessive accumulation of certain fats in the brain and nerve cells and causes progressive dysfunction.  

There are several forms of Tay-Sachs disease, including an infant, juvenile, and adult forms. Over a hundred mutations in the disease-causing Hex A gene have been identified that result in enzyme disfunction. There are currently no effective therapies or cures for Tay-Sachs. 

Illustration by Kateryna Kon

The UC Davis team will genetically modify the patient’s own blood stem cells to restore the Hex A enzyme that is missing in the disease.  

The goal is to complete safety studies and to apply to the US Food and Drug Administration for an Investigational New Drug (IND), the authorization needed to begin a clinical trial in people.  

“The successful development of this therapy will not only help patients with Tay-Sachs but will demonstrate the use case of this therapeutic approach for other monogenic neurodegenerative diseases,” the UC Davis team said. 

This work is a continuation of a CIRM grant that the team received. 

CIRM funds clinical trial to make cancer therapy safer, less toxic

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Blood stem cell transplantation following high dose chemotherapy is standard of care and potentially curative for aggressive forms of lymphoma. However, this treatment regimen is limited by severe toxicity and life-threatening complications due to delayed recovery of the blood system and vascular related damage of multiple organs.

Today the governing Board of the California Institute for Regenerative Medicine (CIRM) funded a Phase 3 clinical trial to support development of a safer, more tolerable alternative.

This brings the number of clinical trials funded by CIRM to 86.

The Board awarded $15,000,000 to Dr. Paul Finnegan and Angiocrine Bioscience to test AB-205, human endothelial cells engineered to express a pro-survival factor.

Prior data suggest that, in the setting of chemotherapy and stem cell transplantation, AB-205 cell therapy can accelerate the recovery of the blood system and protects from toxicity by enhancing the recovery from vascular damage. AB-205 is being studied in a Phase 3 trial in adults with lymphoma undergoing high-dose chemotherapy and autologous blood stem cell transplant.

“If successful, this approach can overcome hurdles to the success of chemotherapy and blood stem cell transplantation for the treatment of advanced blood cancer,” says Dr. Maria T. Millan, President and CEO of CIRM. “This Phase 3 trial is the culmination of preclinical research and the initial clinical trial previously funded by CIRM.”

Lymphoma is the most common blood cancer and one of the most common cancers in the United States, accounting for about 4% of all cancers according to the American Cancer Society and the 6th most commonly diagnosed cancer among men and women in California.  It is estimated that there will be 89,010 new cases of lymphoma and 21,170 lymphoma related deaths in the US in 2022 alone.  In California, it is estimated that there will be over 9,250 new cases of lymphoma with over 2,100 deaths.

“Angiocrine Bioscience is honored to be awarded this grant from CIRM to support our AB-205 Phase 3 trial,” commented Angiocrine CEO Dr. Paul Finnegan. “CIRM has been an instrumental partner in our development of AB-205, a novel therapeutic that acts on the patients’ endogenous stem cell niches. The grant award will considerably aid in our effort to bring forth a solution to the unmet need of transplant-related complications.”

Three women with ties to CIRM featured in 2022 Women in Biopharma list 

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Endpoints News released its 2022 Women in Biopharma list, which recognizes 20 of the top women leading biopharma research and development (R&D).  

This year, the publication received more than 500 entries and selected 20 women “who have blazed trails and are still promising to reshape biopharma R&D for years to come.” 

There are many amazing finalists featured this year, but three in particular stood out for their ties to the California Institute for Regenerative Medicine (CIRM). Those three are:  

Jennifer Gordon, Ph.D. — Senior Vice President of Research and Development at Excision BioTherapeutics 

Dr. Jennifer Gordon and the team at Excision Bio Therapeutics have developed a therapeutic candidate called EBT-101. CIRM is funding a clinical trial to test EBT-101 in patients with HIV. 

This is the first clinical study using the CRISPR-based platform for genome editing and excision of the latent form of HIV-1, the most common form of the virus that causes AIDS in the US and Europe.  

The goal of the treatment is to eliminate or sufficiently reduce the hidden reservoirs of virus in the body to the point where the individual is effectively cured. 

Barbara Wirostko — Co-founder and CMO at Qlaris Bio 

Barbara Wirostko is the co-founder and CMO at Qlaris Bio, a clinical stage biotech company committed to developing therapies for patients suffering from serious and debilitating ophthalmic diseases.  

In addition to her work there, Barbara is a member of CIRM’s Grants Working Group (GWG), which is responsible for evaluating the scientific merit of all applications submitted to CIRM and provides funding recommendations to the CIRM board.  

In the Endpoints News profile, Wirostko shares that she was inspired by her father, also an ophthalmologist, and his desire to help people. 

“I think that was the other thing that really drew me to ophthalmology — is that you were able to work with patients, make a difference in people’s lives, also have a surgical as well as a medical aspect, practicing medicine, and then also have a family,” she said. 

Dr. Lili Yang – UCLA associate professor, Co-founder of Appia Bio and Immune Design 

We’ve written about Dr. Lili Yang’s work on the Stem Cellar blog.  

Dr. Yang at UCLA was recently awarded $1.4 million by CIRM to develop an off-the-shelf cell therapy for ovarian cancer, which causes more deaths than any other cancer of the female reproductive system. 

With support from several CIRM grants, Dr. Yang has developed a platform that can use healthy donor blood stem cells to produce clinical scalable “off-the-shelf” iNKT cells. That has led to the creation of start-up company Appia Bio, and talks with the FDA about testing a series of iNKT cell products in clinical trials. 

“I have this dream that cell therapy can become off-the-shelf, and how this would really help all cancer patients in need. The current cancer cell therapy requires treating patients one-by-one, resulting in a steep price that is hard to afford,” Dr. Yang says.  

“Not everyone lives near a hospital capable of handling such a personalized therapy or can afford such a steep price. If we can make this therapy with centralized manufacturing, pre-quality controlled and ready for wide use then we don’t need to worry about the gender or age or location of the patient.” 

CIRM congratulates all the extraordinary women featured in the Endpoints News 2022 Women in Biopharma list. To see all the finalists, read the official announcement here or visit the Endpoints News website.