Bay Area

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.

Published

3 years ago

February 15, 2022

Oakland Post

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.”

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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Oakland Post: Week of December 18 – 24, 2024

The printed Weekly Edition of the Oakland Post: Week of December 18 – 24, 2024

Published

20 hours ago

December 20, 2024

Oakland Post

To enlarge your view of this issue, use the slider, magnifying glass icon or full page icon in the lower right corner of the browser window.

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City of Oakland Celebrates Reopening of Main Library

“Libraries are such critical facilities for all Oaklanders, whether it’s children coming to story-time, adults reading the newspapers or borrowing the latest novels, and people engaging with a range of services and programs that the library hosts,” said Council President and District 2 Councilmember Nikki Fortunato Bas. “Such library services and programs are only possible when the facility’s electricity, heating, roof, and lighting are fixed and running efficiently. I’m proud to join this re-opening of our Main Public Library.”

Published

3 days ago

December 18, 2024

Oakland Post

Left to Right- Jamie Turbak, Director of Library Services, LaTonda Simmons, Assistant City Administrator, Nikki Bas, President Oakland City Council, Shen Thao, Mayor of Oakland, Carroll Fife, Oakland City Councilmember District 3, Harold Duffey, Assistant City Administrator, Sean Maher, Projects Coordinator. Photo by Kevin Hicks.

The branch had been closed since May for critical infrastructure upgrades

Special to the Post

The City of Oakland leadership and community partners gathered to celebrate the reopening of the Main Library after completion of critical infrastructure upgrades to enhance the library’s facilities and provide a better experience for patrons.

Renovations include new roof installation, skylight repair, critical electrical system upgrades, new boiler control system installation, auditorium heating and cooling system installation, and improvements to lighting, flooring and ceilings throughout the building.

“This is truly something to celebrate, the reopening of our wonderful Main Library! I congratulate the staff and our partners for this important project to make the Main Library a more comfortable place for everyone for years to come, said Oakland Mayor Sheng Thao. “Thank you to Oakland voters and the California State Library for making these crucial improvements possible.”

“Public libraries are a wonderful resource for our residents, offering a safe space for learning and being,” said District 3 Councilmember Carroll Fife. “It is critical to improve and modernize our libraries so more members of our community can utilize and enjoy them. I’m excited that the necessary renovations to the Main Library have been completed successfully and thank everyone involved, particularly the City team, who helped secured the necessary grant funds for this work.”

“I am proud of the City staff and project partners who kept this important project on schedule and under budget,” said Assistant City Administrator G. Harold Duffey. “The library is an incredibly important resource for our community members, and this project is an investment into the library’s future.”

“December 2nd was a momentous occasion for Oakland Public Library as we proudly reopened the doors of the Main Library following extensive infrastructure repairs,” said Director of Library Services Jamie Turbak. “Closing the Main Library for six months was no easy decision, as it serves as the central hub for our library system and is truly the heart of Oakland. Yet, this renovation was essential, representing more than just physical upgrades—it reflects our ongoing commitment to creating a safe, welcoming space for everyone.”

The City Administrator Jestin Johnson also attended the press conference and signalled his support for the completion of the record-setting completion of the renovations. Gay Plair Cobb, a newly appointed Library Commissioner said the Library represents the soul and brains of our community.

The Oakland Public Library secured funding for these crititcal repairs through a variety of sources. The California State Library’s Building Forward Library Facilities Improvement Program awarded the Main Branch $4.2 million. To comply with the grant terms, the City of Oakland provided matching funds through Measures KK, as approved by the Oakland City Council in October 2023.

The Main Library will host an Open House to celebrate the reopening on February 22, 2025, 10 a.m. – 5:00 p.m.

About the Oakland Public Library

The Oakland Public Library is a part of the City of Oakland in California and has been in existence since 1878. Locations include 16 neighborhood branches, a Main Library, a Second Start Adult Literacy Program, the Oakland Tool Lending Library, and the African American Museum and Library at Oakland (AAMLO). The Oakland Public Library empowers all people to explore, connect, and grow. Oaklandlibrary.org

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A Student-Run Group Provides Critical Support Services to Underserved Residents

Those visiting The Suitcase Clinic can get legal advice, sign up for food assistance, receive housing resources, get medical help, or enjoy a hot, fresh meal. They can also get haircuts and foot washes from the student volunteers. Nilo Golchini, executive director of the clinic, said one of the goals for most of the students working there is helping bridge the gap of trust that exists between many unhoused people and the healthcare and social welfare systems.

Published

3 days ago

December 18, 2024

Oakland Post

UC Berkeley Law Students help a clinic visitor with legal advice at their Tuesday night services. The Clinic offers a variety of resources, including medical, to those in the community who have little access to these services. Photo by Magaly Muñoz

Part One

By Magaly Muñoz

Every Tuesday evening, the dining hall of First Presbyterian Church fills up with dozens of people eating, laughing and moving from table to table, receiving much-needed services from UC Berkeley students – just a few blocks away from the university’s campus.

Individuals seeking support services can be found in this multi-stationed room on the south end of the church talking to law students, student case managers, or receiving medical attention in a corner by healthcare professionals.

This weekly event is hosted by Cal students through a volunteer-run program called The Suitcase Clinic.

The clinic, founded in 1989, was intended to offer free resources to underserved communities in Berkeley and surrounding cities. The majority of the clinic’s clientele are unhoused or low-income people looking for extra support.

Those visiting the clinic can get legal advice, sign up for food assistance, receive housing resources, get medical help, or enjoy a hot, fresh meal. They can also get haircuts and foot washes from the student volunteers.

Nilo Golchini, executive director of the clinic, said one of the goals for most of the students working there is helping bridge the gap of trust that exists between many unhoused people and the healthcare and social welfare systems.

During their tenure in the program, many of the students say they become strong advocates for homelessness rights.

Visitors of the Suitcase Clinic can receive haircuts and foot washing by student volunteers every Tuesday evening. Photo by Magaly Muñoz.

“We’re also standing in solidarity with them. So, it’s not saying, ‘I’m going to help you, but I’m also going to stand with you,’” Golchini said.

Student volunteers get extensive training prior to working directly with clients. Those interested have to take a semester-long class to become versed in areas such as outreach, intersectionality, how to interact with unhoused people, how to sign people up for social services. and more.

Volunteers then get to pick from three different clinics: General, Women’s, or Youth and LGBTQ+.

The General Clinic is the most popular among visiting residents, while Women’s and Youth/LQBTQ+ have more specialized services for attendees.

The Women’s Clinic has many of the similar services to General, but also includes nail painting, childcare, and massages.

The Youth and LGBTQ+ Clinic offers a safe space for young people navigating living on the streets, with services that include housing referrals, wellness and recreation classes and employment resources.

Golchini explained that it’s important for them to keep these clinics separate because the different demographics experience poverty and homelessness differently than those who visit the General Clinic.

Suitcase Clinic student workers posing for a photo with a frequent clinic attendee. The Clinic is open to Berkeley unhoused and low-income residents who need medical or legal service, or a hot meal. Photo by Magaly Muñoz.

“We’re able to provide spaces where people can come in and feel safe and not feel like they’re constantly worried that something’s going to happen to them,” she said.

An outreach team also visits encampments every other Saturday in the Berkeley area to provide hygiene kits and encourage people to visit the in-person clinic, if possible.

However, Golchini said engagement has been low for some time now due to a recent decision by the U.S. Supreme Court that allows cities to ban and cite people for sleeping on the streets.

She said a lot of their clientele got displaced to other cities over time, making it difficult to stay in contact with the services the Clinic was providing for them.

But that hasn’t slowed down the students at the Clinic, if anything, it has pushed them to do more for the community they serve.