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Sickle Cell Clinical Trial Aims to Cure Disease by Correcting Patient’s Mutated Gene

Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently, and progresses to a chronic vascular disease that affects approximately 100,000 Americans and millions worldwide, with a disproportionate effect on the Black community.

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Brooklynn Haynes is participating in a research trial that aims to cure sickle cell anemia, a disease that affects 100,000 people in the U.S. Photo courtesy of UCSF.
Brooklynn Haynes is participating in a research trial that aims to cure sickle cell anemia, a disease that affects 100,000 people in the U.S. Photo courtesy of UCSF.

Benioff Children’s Oakland Receives $17M in Grants to Launch 4-Year Study Using CRISPR to Eradicate Sickle Cell Mutation

By Lorna Fernandes

UCSF Benioff Children’s Hospital Oakland has received the largest research trial grant in its history to launch an innovative clinical trial that aims to cure sickle cell disease.

The study, which plans to enroll its first patient by the middle of 2022, will use CRISPR-Cas9 gene-editing technology on patients’ own blood stem cells to correct the mutated gene that causes the debilitating disorder.

Benioff Oakland researchers have received $17 million to support the 4-year trial, in two grants of $8.4 million from the California Institute for Regenerative Medicine (CIRM) and $8.6 million from the NIH Cure Sickle Cell Initiative, supported by the National Heart, Lung, and Blood Institute (NHLBI).

This trial is the first to apply CRISPR-Cas9 technology in humans using a non-viral therapy to correct the mutation that causes sickle cell disease and is part of a UC research consortium led by UCSF with UCLA and UC Berkeley.

Researchers are recruiting patients for treatment in Oakland and Los Angeles, beginning with up to six adults with sickle cell disease. If found to be safe and effective, it will expand to enroll three adolescents aged 12 to 17 years old. Seven patients are expected to be treated in Oakland and two at UCLA.

It is the first-time clinical researchers have attempted to correct a harmful beta-globin gene mutation in a patient’s own cells with non-virally delivered CRISPR gene-correction tools, according to Mark Walters, MD, a professor of pediatrics at UCSF and principal investigator of the clinical trial and gene editing project.

“This therapy has the potential to transform sickle cell disease care by producing an accessible, curative treatment that is safer than the current therapy of stem cell transplant from a healthy bone marrow donor,” Walters said. “If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease.”

Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently, and progresses to a chronic vascular disease that affects approximately 100,000 Americans and millions worldwide, with a disproportionate effect on the Black community.

This clinical study continues BCH Oakland’s commitment to treating and finding a cure for sickle cell disease. The Comprehensive Sickle Cell Disease Center was created at the hospital in 1973 to provide expert diagnosis, treatment and long-term health management for children and adults with sickle cell disease.

Now known as the BCH Oakland Sickle Cell Center of Excellence, it is recognized as having one of the largest and most comprehensive sickle cell programs in the Western United States.

This trial will combine CRISPR technology developed at Innovative Genomics Institute (IGI) – a joint UC Berkeley-UCSF initiative founded by Nobel Laureate Jennifer Doudna – with UCLA’s expertise in genetic analysis and cell manufacturing, as well as its clinical excellence in the field, and nearly 50 years of expertise at Benioff Children’s Oakland in sickle cell care, including cord blood and marrow transplantation, and gene therapy.

“The use of CRISPR gene editing to fix the mutation causing sickle cell disease in each patient’s own blood-forming stem cells required the development of new methods to produce more than 100 million cells per patient that are gene-corrected, healthy and pure,” said Donald Kohn, MD, medical director of the UCLA Human Gene and Cell Therapy Facility. “Previous research studies were limited to producing ½ to 2 million genetically corrected cells, so this represents a significant increase in scale.”

The disease is caused by a single-letter change, or mutation, in human DNA. This trial will use CRISPR genome editing to correct this mutation directly. The project team from UCSF, the IGI, and UCLA have developed CRISPR_SCD001, a patient-specific blood stem cell therapy that has been modified by a CRISPR-Cas9 nuclease to stimulate repair of the sickle mutation.

In the current trial, the patient’s blood stem cells will be extracted and sent to UCLA’s gene manufacturing lab to be processed using electrical pulses that create temporary pores in their membranes. These pores allow the CRISPR-Cas9 platform to enter the cells and travel to the nucleus, where it corrects the sickle cell mutation before the cells are reintroduced to the patient.

“The goal is to develop a cure that is not just safe and effective, but one that is affordable by to those who need it most,” Doudna said. “There have been a lot of promising developments in recent years with CRISPR-based therapies for sickle cell and other genetic diseases, but it’s essential that we have studies like this that can help us create truly accessible cures.”

As part of its legacy, BCH Oakland was recognized for establishing the first not-for-profit sibling donor cord blood bank, leading to children being cured of sickle cell disease and other blood diseases.

Among its many accomplishments in treating sickle cell disease, BCH Oakland scientists studied and received FDA approval for the drug Voxelotor to improve hemoglobin levels and reduce anemia and hemolysis in sickle cell patients.

Going forward, the UCSF research team is hoping to use expanded resources under development in the UCSF Living Therapeutics Initiative as this new treatment is expanded, including the potential to manufacture the investigational cell therapy product at UCSF.

Lorna Fernandes is the senior public information representative at the UCSF Office of Communications and Public Affairs.

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San Francisco Is Investing Millions to Address Food Insecurity. Is Oakland Doing the Same?

There are over 350 grocery programs across San Francisco. Less than a handful in District 10, a neighborhood classified as a food desert, and includes Hunters Point, one of the lowest income areas in the city.

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The San Francisco District 10 Community Market is a fully government funded free grocery store for families in need of food assistance. The market is located in Hunters Point, one of the lowest income areas in the city. Photo by Magaly Muñoz.
The San Francisco District 10 Community Market is a fully government funded free grocery store for families in need of food assistance. The market is located in Hunters Point, one of the lowest income areas in the city. Photo by Magaly Muñoz.

By Magaly Muñoz

On a Thursday evening in February, Marquez Boyd walked along the aisles of San Francisco’s District 10 Community Market looking for eggs and fresh produce to take home to his children. He has been trying new recipes with ingredients he previously couldn’t afford or access.

“I learned how to cook greens since they got a lot of fresh greens here,” Boyd said. “All that stuff is better and more healthy for my kids because they’re still young.”

Meals filled with fresh produce are now possible for Boyd since the District 10 market in Hunters Point opened in 2024 when Bayview Senior Services, a non-profit running the program, received a $5 million investment from the city of San Francisco.

The market is a twist on a traditional food bank, where people can often wait in long lines for pre-bagged groceries they may not need. Here, the goal is to offer people in need a more traditional grocery store setting, with a bigger range of healthy options and less shame for needing assistance.

It’s a twist that Boyd appreciated. “This set up is way better as opposed to maybe like a food bank line,” he said. “It’s easier and faster.”

Similar models exist in Santa Barbara and Tennessee.

There are over 350 grocery programs across San Francisco. Less than a handful in District 10, a neighborhood classified as a food desert, and includes Hunters Point, one of the lowest income areas in the city.

Census Bureau data show that the median income for households in the 94124 zip code, where Hunters Point is located, is just under $83,000 annually. Black households earn about $46,000, Native Hawaiian or other Pacific Islanders earn almost $41,000, and Hispanic households make just above the median income- an average of $86,000.

Located at 5030 3rd Street, the aisles are lined with fresh produce, canned goods, bread and snacks. While refrigerators and freezers in the back of the market are filled with dairy products and meat.

The best part- everything inside is free for eligible customers.

The San Francisco District 10 Community Market is stocked with fresh produce, dairy, meat and chicken, bread, and cultural food staples. Directors of the market say they pride themselves on providing healthy options for community members. Photo by Magaly Muñoz.

The San Francisco District 10 Community Market is stocked with fresh produce, dairy, meat and chicken, bread, and cultural food staples. Directors of the market say they pride themselves on providing healthy options for community members. Photo by Magaly Muñoz.

“The interesting thing about this market is that it’s a city-funded effort to create something besides the average food line to give more dignity and choice than is normally given to low-income people,” said Cathy Davis, executive director of Bayview Senior Services.

Davis said people feel more comfortable coming into the market because they can choose the food they want and at a time that’s convenient for them.

Boyd, a single father of two kids, recently lost his job and relied on his sister’s generosity before discovering the market. He comes to market when he gets off of work in the evening.

“It’s a lot of people in these communities that don’t get a chance to eat healthy,” Boyd said. “They don’t have the money to go to grocery stores to buy expensive stuff.”

Another shopper, Rhonda Hudson, said the market helped her meet her grandson’s diet-related health problems. She used to travel outside the neighborhood for affordable groceries, but now she no longer has to.

According to the city’s Human Services Agency, there are no plans to expand the markets in San Francisco due to budget constraints.

But Davis isn’t worried about losing the market funding.

“City leaders were on board with creating it and finding the money to put it together so I would say we didn’t have to advocate because it came through the government. Now it’s our job to keep it going to prove that it’s a pilot worth maintaining,” Davis said.

District 10 Supervisor Shamann Walton, who co-sponsored the ordinance, said that projects like the market are “essential to our neighborhoods,” where access to affordable food has been a challenge.

“Investing in local community markets helps ensure that families have reliable, healthy food options close to home, addressing food insecurity and supporting the well-being of our community regardless of income,” Walton said.

Rhonda Hudson is a shopper of the District 10 Community Market in San Francisco. The fresh produce she gets at the free grocery store program helps her grandson, who has a diet-related illness, stay healthy. Photo by Magaly Muñoz.

Rhonda Hudson is a shopper of the District 10 Community Market in San Francisco. The fresh produce she gets at the free grocery store program helps her grandson, who has a diet-related illness, stay healthy. Photo by Magaly Muñoz.

Why Not Oakland?

Only slightly larger than San Francisco, Oakland has over 400 food distribution sites. Oakland provides grants to nonprofit-run organizations who run grocery programs. But in recent months, the city has begun to reduce those, forcing some organizations to regroup, and making it challenging to implement a community market similar to San Francisco’s.

The Oakland Post repeatedly reached out to city and county officials for comment on the story but did not receive a response.

At several food banks across West and East Oakland, residents shared their frustrations about long lines, wilting produce, and limited food choices.

At one food bank, located at Christian Tabernacle Church, a young mother, who requested anonymity for privacy reasons, waited in the rain for over three hours for a single bag of groceries.

“I like to get here early because I get better [quality] fruits and vegetables,” she said. She added that it’s not a lot of food that she receives for her family, but it helps close the gap when her budget is tight.

Behind her, several other women waited their turn. Neither the timing of the distribution nor the location of the food bank fit their schedules, the women said, but their choices feel limited.

Only a handful of Oakland food bank sites operate throughout the day, like the San Francisco market. Most food distribution programs are sustained by Alameda County Food Bank, not by city funding. Private grants and donations also help fund the programs.

Securing city funding is increasingly challenging. Oakland faces a $130 million budget shortfall, with a projected $280 million deficit in the next biennial cycle. Citing budget concerns, the city has reduced numerous department budgets and grants. One of those cuts included slashing the longstanding SOS Meals on Wheels grant, which helped provide food to 3,000 seniors.

Charlie Deterline, executive director of Meals on Wheels, said the termination of their $150,000 annual grant could mean that Oakland residents might see a change in the amount of meals they receive. The organization has gone 19 months without funding from that grant, Deterline said, but “continued working on good faith from the city” because they were assured they would be paid out. Now, Deterline is having doubts.

The program also received a grant of more than $125,000 from the Sugar Sweetened Beverage Tax. Yet, on June 12, the city informed grant recipients that the funding could be rescinded in order to balance the budget. That ultimately happened, said Deterline.

“Oakland is by far the most expensive city for us to operate in. It is also where the greatest need is – for us to meet that need, it will take the entire community coming together,” Deterline said.

From the sugar tax, money from that measure is also not being allocated correctly as the majority of the funding has been used to fund government services, said members of the SSB tax advisory board.

The tax generates around $7 million annually. 25% to 40% of the funding goes towards grants for community based organizations instead of the 60% allocation that the SSBT advisory board recommended the city to use for health programs. The rest of the funding goes to the city, according to Oakland’s mid-cycle budget.

Advisory board member Dwayne Aikens said he’s not sure Oakland will ever renew the grants that have been cut from this tax. “I’m looking at the conditions of the city and I’m not optimistic,” Aikens said. “If they don’t have the money now, I don’t think they’ll have the money in the future.”

Aikens said the tax was “kind of a waste.” He’s heard displeasure from the community about the lack of funding into Black and Brown neighborhoods, groups who typically live in areas of Oakland that see health and income disparities.

Meanwhile, the Community Market, which reflects the diversity of the Bayview Hunters Point community, is investing in over 800 of the city’s most vulnerable households. In-store staff and directors speak the languages common to the area and the program provides a culture-of-the week selection of foods for those interested in trying something new.

Davis said it’s up to local municipalities to ensure that residents don’t go to bed hungry, and investments need to be made in order to combat the pockets of neighborhoods who are on the brink of food insecurity.

“That’s just such a core responsibility and a core goal of everyone, to make sure that people are fed and healthy. It’s not a luxury item,” Davis said. “It’s something that needs to happen, whether we’re in a budget crisis or not.”

Reporter Magaly Muñoz produced this story as part of a series as a 2024 USC Annenberg Center for Health Journalism Data Fellow and Engagement Grantee.

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Oakland Post: Week of March 12 – 18, 2025

The printed Weekly Edition of the Oakland Post: Week of March 12 – 18, 2025

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Oakland Post: Week of March 5 – 11, 2025

The printed Weekly Edition of the Oakland Post: Week of March 5 – 11, 2025

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