Month: February 2023

Join the movement to fight rare diseases

/ Katie Sharify / 2 Comments

Tomorrow, February 28th, is Rare Disease Day. It’s a day to remind ourselves of the millions of people, and their families, struggling with these diseases. These conditions are also called orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments.

Here at the California Institute for Regenerative Medicine (CIRM), we understand the importance of funding research that impacts not just the most common diseases. In fact, 50% of all the projects we fund target a rare disease or condition such as: Retinitis pigmentosa, Sickle cell disease, Huntington’s disease, and Duchenne Muscular Dystrophy.

Over the years, CIRM has invested millions of dollars in helping children born with severe combined immunodeficiency (SCID), including $12 million to test a newly designed therapy in a clinical trial at UC San Francisco.

Children born with SCID have no functioning immune system so even a simple infection can prove life-threatening or fatal. We recently shared an update from one of the young patients in the trial.

Additionally, last December, the CIRM governing Board 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. 

The irony of rare diseases is that a lot of people have them. The total number of Americans living with a rare disease is estimated at between 25-30 million. Two-thirds of these patients are children.

Right now, individual disease programs tend to try individual approaches to developing a treatment, which is time consuming and expensive. That’s why this past summer, CIRM signed a Memorandum of Understanding (MOU) with the Foundation for the National Institutes of Health (FNIH) to join the Bespoke Gene Therapy Consortium (BGTC).

BGTC is a public-private partnership, managed by FNIH, that brings together the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private sector organizations to streamline the development and delivery of gene therapies for rare diseases.

“At CIRM we have funded several projects using gene therapy to help treat, and even cure, people with rare diseases such as severe combined immunodeficiency,” says Dr. Maria T. Millan, the President and CEO of CIRM. “But even an agency with our resources can only do so much. This agreement with the Bespoke Gene Therapy Consortium will enable us to be part of a bigger partnership, one that can advance the field, overcome obstacles and lead to breakthroughs for many rare diseases.”

CIRM is proud to fund and spread awareness of rare diseases and invites you to watch this video about how they affect families around the world.

Investing in stem cell and gene therapy treatments for HIV

/ Esteban Cortez / 1 Comment

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

/ Esteban Cortez / Leave a comment

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 initiative, 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

/ Esteban Cortez / 3 Comments

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.