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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Homelessness Committee and Advocates Urge City to Stop Confiscating Unhoused People’s Belongings

Encampment sweeps are not a new method of action to evict people from living and sleeping on the streets in San Francisco. However, recent reports indicate that city staff are not following proper policy, exacerbating the problems for unhoused people. Homeless advocates and allies held a press conference on Thursday at City Hall, condemning staff workers for destroying people’s property during encampment evictions and asking officials to ensure that important documents and medication are not being stripped from these individuals.

Published

1 week ago

October 2, 2024

Oakland Post

"By destroying the very items that could help people regain stability, the city is not just punishing people for being poor, but actively making it harder for them to escape homelessness," Jennifer Friedenbach, executive director of the Coalition on Homelessness, said.

By Magaly Muñoz

Homeless advocates and allies held a press conference on Thursday at City Hall, condemning staff workers for destroying people’s property during encampment evictions and asking officials to ensure that important documents and medication are not being stripped from these individuals.

“By destroying the very items that could help people regain stability, the city is not just punishing people for being poor, but actively making it harder for them to escape homelessness,” Jennifer Friedenbach, executive director of the Coalition on Homelessness, said.

Friedenbach criticized the city for not fixing their housing problem or finding new ways to shelter people, instead they are further adding to the harm of the “humanitarian crisis that San Francisco is facing.”

The press conference was held before the monthly Homelessness Oversight Commission (HOC) meeting, where commissioners discussed a draft resolution to submit to city staff highlighting the importance of not separating people from their items as this might cause further distress.

The resolution lists ssential items that workers should be cautious of not destroying or throwing away including medical documents and medication, work permits, identification, and survival gear, such as blankets or tents.

City policy instructs workers to “bag and tag” items left behind after an encampment sweep. These items are labeled by Public Works and kept at their operations yard for 90 days before being discarded.

But according to several reports and videos of the sweeps, the city has not always followed this policy and has on numerous occasions thrown away people’s medications or tents, leaving individuals without their essentials.

During the meeting, commissioners suggested adding school records and family related support items, such as diapers, to the resolution because of the increasing number of families living on the streets.

Virginia Taylor, senior policy advisor for Safe & Sound, said 531 families are waiting for housing in San Francisco. Many of these families are living out of their cars or in RVs, yet the city has limited safe parking spots where people can situate themselves.

Along with not throwing out people’s belongings, advocates are also continuing to ask the city to stop the encampment sweeps because all they are accomplishing is moving unhoused folks block to block without solving the root problem of lack of consistent housing.

“We need urgent action, more family shelter beds, a stop to vehicle sweeps, expanded safe parking programs and housing solutions that keep our multi-generational families together. Our children’s futures depend on it. Let’s build a San Francisco where no family falls through the crack and every child has the opportunity to thrive,” Taylor said.

Speakers referenced the RV sweep conducted in early August on Zoo Road, where dozens of people, many of them non-English speaking immigrants, were asked to leave the parking lot or else their vehicles would be towed and they would be cited.

While people were offered shelter beds or housing vouchers, some worried about where they would stay while the city processed their applications. This drew criticism of San Francisco’s method of not always having immediate options for people yet continuing to sweep unhoused folks with nowhere to go.

Commissioners of HOC agreed that the city is not trying to exacerbate the issue and the resolution is one of many steps to ensure that there are no setbacks in the progress to ending homelessness in San Francisco.

The HOC will approve the resolution at a later meeting once amendments and changes are made.

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Former Mayor Willie L. Brown Endorses Dana Lang for BART Board District 7

Former San Francisco Mayor Willie L. Brown has announced his endorsement support for Dana Lang for BART Board District 7 Seat, which includes voters from both sides of the Bay, and in San Francisco includes Bay View Hunters Point and Treasure Island. Brown acknowledged that Lang has been a behind-the-scenes force in transportation funding for many years and can help BART manage its financial challenges.

Published

1 week ago

October 2, 2024

Oakland Post

Photo courtesy of Dana Lang.

By Oakland Post Staff

Brown acknowledged that Lang has been a behind-the-scenes force in transportation funding for many years and can help BART manage its financial challenges.

“When I met with Dana Lang I asked many questions, then I asked others about her contributions. Getting to know her I realized that she truly understood transportation. At a time when BART is facing a “fiscal cliff” and an upcoming deficit of nearly $360 million per year, Dana is more than ready for this job, she is ready to meet the moment!”

Over the past 24 years Lang has been a funding and grants specialist with several municipal transportation agencies, including the Metropolitan Transportation Commission, San Francisco Muni, San Francisco Police Department and San Francisco International Airport (SFO).

Lang says, “I’ve faced a number of fiscal crises in my career — such as securing $52 million in new transit security funding for SFMTA (Muni) during the 2008 Great Recession, when others thought it was not possible. I have always managed to identify new funding and ways to make transit more secure. Facing a crisis is the best time to act, through advocacy and policy setting. We’ve got to keep BART running and make it safer and more vibrant in order to meet the needs of our riders, our work force, and our community.”

Lang grew up in the low-income minority community of East Palo Alto, CA, and knew that locating grants and resources could positively impact an entire city and its surrounding region — helping to create and retain agency jobs, getting transit riders to their workplaces, and encouraging small business development near transit hubs.

With that in mind, she pursued a bachelor’s degree in economics from Wellesley College, then an MBA from UC Berkeley’s Haas School of Business. She started her municipal career as a policy advisor to Mayor Elihu Harris and helped secure grants for the City of Oakland before moving to the Metropolitan Transportation Commission to serve as a transportation grants specialist.

During her 24-year career she has helped secure hundreds of millions of dollars for Bay Area transit agencies and municipalities. In addition to BART’s financial health, Lang’s priorities for BART also include safety, cleanliness, station vitality — and bringing riders back to BART. She has served on the BART Police Civilian Review Board since 2022.

Lang is also endorsed by BART Board Director Robert Raburn, former BART Board Director Carole Ward Allen, the Rev. Amos Brown, pastor of San Francisco’s Third Baptist Church, Alameda County supervisors Keith Carson and Nate Miley, former Oakland Mayor Elihu Harris, District 4 Oakland City Councilmember Janani Ramachandran, Oakland Chinatown leader Carl Chan, and many others.

Lang is seeking the BART Board District 7 Seat, which includes San Francisco’s Bay View Hunters Point and Treasure Island, a large portion of Oakland, the cities of Alameda, Emeryville, Piedmont, and a small portion of Berkeley.

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Art

Phenomenal Woman’ Maya Angelou Monument Unveiled at San Francisco Main Library

In a joyful community celebration attended by over 200 people, including Mayor London Breed, the highly anticipated ‘Portrait of a Phenomenal Woman’ monument to Dr. Maya Angelou was unveiled at the San Francisco Main Library on Sept. 19. Oakland-based artist Lava Thomas created the 9-foot bronze and stone monument in the form of a book featuring a portrait and quotes from the celebrated author, poet, civil rights activist and former San Francisco resident.

Published

1 week ago

October 2, 2024

Oakland Post

Attending the unveiling of the monument to the late Dr. Maya Angelou were, from left, Dr. Gina M. Fromer, CEO Glide Foundation; San Francisco Mayor London Breed, Rosa Johnson, (Angelou’s niece); Ralph Remington, San Francisco director of Cultural Affairs; Lava Thomas, artist and creator; Denise Bradley-Tydus, former interim director of Cultural Affairs; San Francisco Poet Laureate Genny Lim, and San Francisco City Librarian Michael Lambert. Photo by Linda Parker Pennington.

By Linda Parker Pennington

Oakland-based artist Lava Thomas created the 9-foot bronze and stone monument in the form of a book featuring a portrait and quotes from the celebrated author, poet, civil rights activist and former San Francisco resident.

The work was commissioned by the San Francisco Arts Commission in response to legislation passed in 2018 by San Francisco’s Board of Supervisors, requiring at least 30% female representation in the public realm.

Attending the unveiling were Angelou’s grandson, Elliott Jones, social advocate, philanthropist, and board member of the Dr. Maya Angelou Foundation; and Rosa Johnson, Angelou’s niece and family archivist, who spoke about the historic unveiling of this first public monument portraying a Black woman in San Francisco’s history.