Google eases ban on ads for stem cell therapies

What started out as an effort by Google to crack down on predatory stem cell clinics advertising bogus therapies seems to be getting diluted. Now the concern is whether that will make it easier for these clinics to lure unsuspecting patients to pay good money for bad treatments?

A little background might help here. For years Google placed no restrictions on ads by clinics that claimed their stem cell “therapies” could cure or treat all manner of ailments. Then in September of 2019 Google changed its policy and announced it was going to restrict advertisements for stem cell clinics offering unproven, cellular and gene therapies.

This new policy was welcomed by people like Dr. Paul Knoepfler, a stem cell scientist at UC Davis and longtime critic of these clinics. In his blog, The Niche, he said it was great news:

“Google Ads for stem cell clinics have definitely driven hundreds if not thousands of customers to unproven stem cell clinics. It’s very likely that many of the patients who have ended up in the hospital due to bad outcomes from clinic injections first went to those firms because of Google ads. These ads and certain particularly risky clinics also are a real threat to the legitimate stem cell and gene therapy fields.”

Now the search-engine giant seems to be adjusting that policy. Google says that starting July 11 it will permit ads for stem cell therapies approved by the US Food and Drug Administration (FDA). That’s fine. Anything that has gone through the FDA’s rigorous approval process deserves to be allowed to advertise.

The real concern lies with another adjustment to the policy where Google says it will allow companies to post ads as long as they are “exclusively educational or informational in nature, regardless of regulatory approval status.” The problem is, Google doesn’t define what constitutes “educational or informational”. That leaves the door open for these clinics to say pretty much anything they want and claim it meets the new guidelines.

To highlight that point Gizmodo did a quick search on Google using the phrase “stem cells for neuropathy” and quickly came up with a series of ads that are offering “therapies” clearly not approved by the FDA. One ad claimed it was “FDA registered”, a meaningless phrase but one clearly designed to add an air of authenticity to whatever remedy they were peddling.

The intent behind Google’s change of policy is clearly good, to allow companies offering FDA-approved therapies to advertise. However, the outcome may not be quite so worthy, and might once again put patients at risk of being tricked into trying “therapies” that will almost certainly not do them any good, and might even put them in harm’s way.

Making the list of people to follow

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If you are walking down the street on a dark night, being followed is not necessarily something you want. But if you are online, having someone follow you is almost always a positive thing. And when that person is Dr. Paul Knoepfler it’s most definitely a plus.

Paul is a stem cell scientist at UC Davis (full disclosure, we have funded some of his work). He’s also one of the longest-running and most active bloggers about regenerative medicine and an ever-present presence on Twitter. His blog is always a great read and, for those of us without a science background, easy to follow and understand.

Dr. Paul Knoepfler, UC Davis: Photo courtesy UC Davis

That’s why it’s quite an honor that Paul has listed the California Institute for Regenerative Medicine’s as one of the 50 Influencers on stem cells to follow on Twitter.

Paul says this does not necessarily mean the most influential in the field of research because many researchers – such as Nobel Prize winner Dr. Shinya Yamanaka – don’t use Twitter. He says in making the list he looked for a few key elements.

“I particularly appreciate those accounts that include a mix of info, news, and opinion with original content or opinions of their own too.

“I emphasized inclusion of those accounts who regularly tweet. Also, I aimed for a good mixture of accounts across the globe, not just in the U.S. I also included stem cell policy researchers and bioethicists.”

“I picked this list of 50… for 2022 based simply on my impressions of their influence or because they do interesting tweets and/or have a fresh perspective on things, not strictly based on metrics.”

Whatever the reason, we’re delighted, and honored to be on Paul’s list.

And if you would like to see why we made the ’50 to Follow list’, then follow us on Twitter

UC Davis Health researchers aim to use CAR T cells for HIV cure

Dr. Abedi (right) in the lab at UC Davis Health. He and his team of researchers have launched a study looking to identify a potential cure for HIV. Photo Courtesy of UC Davis Health.

Worldwide, almost 38 million people are living with HIV—the virus that can lead to AIDS— and it’s estimated that 75% of them receive antiviral treatment to keep the virus in check. In California, 150,000 people live with HIV and 68% of these individuals are virally suppressed due to treatment.  

To fight this virus, UC Davis Health researchers—with funding from a CIRM grant—have launched a study looking to identify a potential cure for HIV. Using immunotherapy, researchers will 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. 

Targeting HIV with CAR T cells

“For this study we will educate the cells by inserting a gene to target cells that have been infected by the HIV virus,” explained Mehrdad Abedi, professor of internal medicine, hematology and oncology and the principal investigator of the study. “The idea is these modified cells will attach to the HIV-infected cells and destroy the cells that are infected while also stopping the infected cells’ ability to replicate.” 

Modified T-cells, known as CAR T cells, are an FDA-approved treatment for different forms of cancer including acute lymphoblastic leukemia, non-Hodgkin lymphoma, and multiple myeloma. With cancer, the immune system often fails to deploy T-cells right away or at all. When it does, the attack is ineffective. CAR T-cell immunotherapy changes these collected T-cells to produce chimeric antigen receptors (or CARs) that adhere to tumors to destroy them. 

Study seeking HIV patients

For the study, UC Davis Health researchers are working to identify and recruit HIV-positive patients between the ages of 18 and 65 who have had an undetectable HIV viral load for the 12 months and have been on continuous antiretroviral therapy for at least 12 months.  

Patients also need to be willing to pause their antiretroviral therapy as part of the study. 

“While it is exciting, the study will require a lot of dedication from the patient because of the time commitment involved and the necessary steps required,” said Paolo Troia-Cancio, a clinical professor of medicine with the infectious disease division with over 20 years of experience treating HIV and co-investigator on the CAR T cell study.   

The search for an HIV cure 

Three patients have been cured of HIV using bone marrow transplants, including a woman in New York who received a cord blood stem cell transplant. She received a bone marrow transplant using umbilical cord blood donor cells that bore a mutation that makes them resistant to HIV infection to treat her leukemia. 

There have also been two previous cases involving an HIV cure following allogeneic bone marrow transplants. Both patients had leukemia and received bone marrow transplants from donors who carried the same mutation that blocks HIV infection.  

“While these stories provide inspiration and hope to finding a cure for HIV, a bone marrow transplant is not a realistic option for most patients,” said Abedi. “Such transplants are highly invasive and risky, so they are generally offered only to people with cancer who have exhausted all other options.” 

Abedi and his fellow researchers see this study as a potential road map to finding a cure for HIV.  

The California Institute for Regenerative Medicine (CIRM) has funded earlier work by Dr. Abedi and his team in trying to develop a therapy to help people with HIV who also have lymphoma.  

To read the source article about this CIRM-funded study, click here

HOPE for patients with a muscle destroying disease

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Caleb Sizemore, photo by Todd Dubnicoff

Caleb Sizemore says growing up with Duchenne’s Muscular Dystrophy (DMD) was tough. The disease is a rare genetic disorder that slowly destroys a person’s muscles, impairing their ability to walk or breathe. Eventually it attacks the heart leading to premature death.

Caleb says the disease meant “I was limited in what I could do, where I couldn’t play sports and where I was teased and bullied sometimes for being different.”

In the past people with DMD – almost all of whom are boys – lost the ability to walk by the age of 12, and many died in their 20’s. But a new treatment – originally funded by CIRM – is showing promise in helping reverse some of the damage caused by the disease.

Dr. Craig McDonald working with a person who has DMD: Photo courtesy UC Davis

Results from a clinical trial – published in the journal Lancet – showed that the therapy helped halt the decline in muscle strength in the arms and hands, and in MRI’s appeared to improve heart function.

In a news release, Dr. Craig McDonald, a UC Davis professor and the lead author of the study, said: “The trial produced statistically significant and unprecedented stabilization of both skeletal muscle deterioration affecting the arms and heart deterioration of structure and function in non-ambulatory DMD patients.”

The therapy, called CAP-1002, uses cells derived from the human heart that have previously demonstrated the ability to reduce muscle inflammation and enhance cell regeneration. The clinical trial, called HOPE-2 (Halt cardiomyopathy progression in Duchenne).

Dr. McDonald says with current treatments only having a limited impact on the disease, CAP-1002 may have a big impact on the people affected by DMD and their families.

“The trial showed consistent benefits of this cell-based therapy. It suggests that this infusion may be an important treatment option for the boys and young men who have this debilitating disorder.”

The team now hope to be able to apply to the Food and Drug Administration for permission to start a bigger clinical trial involving more patients.

Caleb Sizemore took part in an earlier clinical trial involving this approach. He says MRI’s showed that the therapy appeared to reduce scarring on his heart and gave him greater energy.

In 2017 Caleb talked to the CIRM governing Board about DMD and his part in the clinical trial. You can see that video here.

CIRM Board gives thumbs up to training and treatment programs

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CIRM Bridges student discusses her poster presentation

At CIRM, the bread and butter of what we do is funding research and hopefully advancing therapies to patients. But the jam, that’s our education programs. Helping train the next generation of stem cell and gene therapy scientists is really inspiring. Watching these young students – and some are just high school juniors – come in and grasp the science and quickly become fluent in talking about it and creating their own experiments shows the future is in good hands.

Right now we fund several programs, such as our SPARK and Bridges internships, but they can’t cover everything, so last week the CIRM Board approved a new training program called COMPASS (Creating Opportunities through Mentorship and Partnership Across Stem Cell Science). The program will fill a critical need for skilled research practitioners who understand and contribute at all levels in the translation of science to medicine, from bench to bedside.

The objective of the COMPASS Training Program is to prepare a diverse group of undergraduate students for careers in regenerative medicine through the creation of novel recruitment and support mechanisms that identify and foster untapped talent within populations that are historically under-represented in the biomedical sciences. It will combine hands-on research with mentorship experiences to enhance transition of students to successful careers. A parallel objective is to foster greater awareness and appreciation of diversity, equity and inclusion in trainees, mentors, and other program participants

The CIRM Board approved investing $58.22 million for up to 20 applications for a five-year duration.

“This new program highlights our growing commitment to creating a diverse workforce, one that taps into communities that have been historically under-represented in the biomedical sciences,” says Dr. Maria T. Millan, President and CEO of CIRM. “The COVID19 pandemic made it clear that the benefits of scientific discovery are not always accessible to communities that most need them. CIRM is committed to tackling these challenges by creating a diverse and dedicated workforce that can meet the technical demands of taking novel treatment ideas and making them a reality.”

The Board also approved a new $80 million concept plan to expand the CIRM Alpha Stem Cell Clinic Network. The Network clinics are all in top California medical centers that have the experience and the expertise to deliver high-quality FDA-authorized stem cell clinical trials to patients.

There are currently five Alpha Clinics – UC San Diego; UCLA/UC Irvine; City of Hope; UCSF; UC Davis – and since 2015 they have hosted more than 105 clinical trials, enrolled more than 750 patients in these trials, and generated more than $95 million in industry contracts. 

Each award will provide up to $8 million in funding over a five-year period. The clinics will have to include:

  • A demonstrated ability to offer stem cell and gene therapies to patients as part of a clinical trial.
  • Programs to help support the career development of doctors, nurses, researchers or other medical professionals essential for regenerative medicine clinical trials.
  • A commitment to data sharing and meeting CIRM’s requirements addressing issues of diversity, equity and inclusion and meeting the needs of California’s diverse patient population.

How these scholars are growing the regenerative medicine field in California

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CIRM Scholar Alessandra Rodriguez y Baena

Through our new Strategic Plan, the California Institute for Regenerative Medicine (CIRM) will build inclusive participation opportunities for all stakeholders, from the students to the workforce to the patients.  

That said, it’s important to recognize the important work CIRM has already done to train the next generation of scientists and grow the field of regenerative medicine. Alessandra’s story illustrates just one of the many ways we have done that in the past, and we intend to do even more in the future. 

Gaining Exposure to Innovative Research

CIRM Scholar Alessandra Rodriguez y Baena was a Master’s student at Cal Poly, San Luis Obispo. With the support of CIRM’s Bridges Program, she became a CIRM intern in the Willert Lab at UC San Diego.  

As a student researcher, CIRM provided her with supportive mentors (both at Cal Poly and UCSD), hands-on training in the field of regenerative medicine, and exposure to innovative ideas and research. The program also provided Alessandra with a stipend to help cover expenses. This was particularly helpful for students from low-income backgrounds who otherwise might not be able to afford to go to college. 

“I always recommend my undergraduate students who are interested in research to apply to the Bridges programs because, to me, it was a defining experience that led me to pursue my passion for stem cell research as well as teaching,” Alessandra says. 

Alessandra is now a fourth-year PhD student in the Forsberg Lab in the department of Molecular, Cell & Developmental Biology at UC Santa Cruz where she is studying the epigenetic regulation of aging in bone marrow stem cells.  

In addition to Alessandra, CIRM has provided opportunities in science to nearly 3,000 students across California. These include high schoolers in our SPARK Program, as well as undergrads and graduate students in our Bridges Program and pre and post-doctoral students in our Research Training program. Many of these are from diverse backgrounds.  

A Game Changer

Sneha Santosh, another CIRM Scholar, first heard about CIRM’s Bridges to Stem Cell Therapy and Research internship when she was graduating from the UC Davis. She was pursuing a degree in microbial biotechnology and thinking about getting a master’s degree in biotechnology. She said the opportunity to be part of a program that is training the next generation of scientists was a game changer for her.  

Through the Bridges Program, she learned about stem cells’ power to treat a disease’s root cause rather than just the symptoms. She saw how these transformative therapies changed people’s lives. 

Today, she is a cell culture associate with Novo Nordisk, a leading global healthcare company in Fremont, California 

CIRM’s New Strategic Plan

Alessandra and Sneha’s stories capture CIRM’s commitment to building education and training programs, and providing opportunities to build a diverse, highly skilled regenerative medicine workforce. We’ll be covering this ambitious yet achievable goal in our upcoming blog posts.  

To learn more about CIRM’s work and plans build the regenerative medicine field, check out our new 5-year strategic plan on our website.  

Overcoming obstacles and advancing treatments to patients

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UC Davis GMP Manufacturing facility: Photo courtesy UC Davis

When you are trying to do something that has never been done before, there are bound to be challenges to meet and obstacles to overcome. At the California Institute for Regenerative Medicine (CIRM) we are used to coming up with great ideas and hearing people ask “Well, how are you going to do that?”

Our new 5-year Strategic Plan is how. It’s the roadmap that will help guide us as we work to overcome critical bottlenecks in bringing regenerative medicine therapies to people in need.

Providing more than money

People often think of CIRM as a funding agency, providing the money needed to do research. That’s true, but it’s only part of the story. With every project we fund, we also offer a lot of support. That’s particularly true at the clinical stage, where therapies are being tested in people. Projects we fund in clinical trials don’t just get money, they also have access to:

  • Alpha Stem Cells Clinic Network – This is a group of specialized medical centers that have the experience and expertise to deliver new stem cell and gene therapies.
  • The CIRM Cell and Gene Therapy Center – This helps with developing projects, overcoming manufacturing problems, and offers guidance on working with the US Food and Drug Administration (FDA) to get permission to run clinical trials.
  • CIRM Clinical Advisory Panels (CAPs) – These are teams put together to help advise researchers on a clinical trial and to overcome problems. A crucial element of a CAP is a patient advocate who can help design a trial around the needs of the patients, to help with patient recruitment and retention.

Partnering with key stakeholders

Now, we want to build on this funding model to create new ways to support researchers in bringing their work to patients. This includes earlier engagement with regulators like the FDA to ensure that projects match their requirements. It includes meetings with insurers and other healthcare stakeholders, to make sure that if a treatment is approved, that people can get access to it and afford it.

In the past, some in the regenerative medicine field thought of the FDA as an obstacle to approval of their work. But as David Martin, a CIRM Board member and industry veteran says, the FDA is really a key ally.

“Turning a promising drug candidate into an approved therapy requires overcoming many bottlenecks… CIRM’s most effective and committed partner in accelerating this is the FDA.”

Removing barriers to manufacturing

Another key area highlighted in our Strategic Plan is overcoming manufacturing obstacles. Because these therapies are “living medicines” they are complex and costly to produce. There is often a shortage of skilled technicians to do the jobs that are needed, and the existing facilities may not be able to meet the demand for mass production once the FDA gives permission to start a clinical trial. 

To address all these issues CIRM wants to create a California Manufacturing Network that combines academic innovation and industry expertise to address critical manufacturing bottlenecks. It will also coordinate training programs to help build a diverse and expertly trained manufacturing workforce.

CIRM will work with academic institutions that already have their own manufacturing facilities (such as UC Davis) to help develop improved ways of producing therapies in sufficient quantities for research and clinical trials. The Manufacturing Network will also involve industry partners who can develop facilities capable of the large-scale production of therapies that will be needed when products are approved by the FDA for wider use.

CIRM, in collaboration with this network, will also help develop education and hands-on training programs for cell and gene therapy manufacturing at California community colleges and universities. By providing internships and certification programs we will help create a talented, diverse workforce that is equipped to meet the growing demands of the industry.

You can read more about these goals in our 2022-27 Strategic Plan.

Creating a diverse group of future scientists

Students in CIRM’s Bridges program showing posters of their work

If you have read the headlines lately, you’ll know that the COVID-19 pandemic is having a huge impact on the shipping industry. Container vessels are forced to sit out at anchor for a week or more because there just aren’t enough dock workers to unload the boats. It’s a simple rule of economics, you can have all the demand you want but if you don’t have the people to help deliver on the supply side, you are in trouble.

The same is true in regenerative medicine. The field is expanding rapidly and that’s creating a rising demand for skilled workers to help keep up. That doesn’t just mean scientists, but also technicians and other skilled individuals who can ensure that our ability to manufacture and deliver these new therapies is not slowed down.

That’s one of the reasons why CIRM has been a big supporter of training programs ever since we were created by the voters of California when they approved Proposition 71. And now we are kick-starting those programs again to ensure the field has all the talented workers it needs.

Last week the CIRM Board approved 18 programs, investing more than $86 million, as part of the Agency’s Research Training Grants program. The goal of the program is to create a diverse group of scientists with the knowledge and skill to lead effective stem cell research programs.

The awards provide up to $5 million per institution, for a maximum of 20 institutions, over five years, to support the training of predoctoral graduate students, postdoctoral trainees, and/or clinical trainees.

This is a revival of an earlier Research Training program that ran from 2006-2016 and trained 940 “CIRM Scholars” including:

• 321 PhD students
• 453 Postdocs
• 166 MDs

These grants went to academic institutions from UC Davis in Sacramento to UC San Diego down south and everywhere in-between. A 2013 survey of the students found that most went on to careers in the industry.

  • 56% continued to further training
  • 14% advanced to an academic research faculty position
  • 10.5% advanced to a biotech/industry position
  • 12% advanced to a non-research position such as teaching, medical practice, or foundation/government work

The Research Training Grants go to:

AWARDINSTITUTIONTITLEAMOUNT
EDUC4-12751Cedars-SinaiCIRM Training Program in Translational Regenerative Medicine    $4,999,333
EDUC4-12752UC RiversideTRANSCEND – Training Program to Advance Interdisciplinary Stem Cell Research, Education, and Workforce Diversity    $4,993,115
EDUC4-12753UC Los AngelesUCLA Training Program in Stem Cell Biology    $5 million
EDUC4-12756University of Southern CaliforniaTraining Program Bridging Stem Cell Research with Clinical Applications in Regenerative Medicine    $5 million
EDUC4-12759UC Santa CruzCIRM Training Program in Systems Biology of Stem Cells    $4,913,271
EDUC4-12766Gladstone Inst.CIRM Regenerative Medicine Research Training Program    $5 million
EDUC4-12772City of HopeResearch Training Program in Stem Cell Biology and Regenerative Medicine    $4,860,989
EDUC4-12782StanfordCIRM Scholar Training Program    $4,974,073
EDUC4-12790UC BerkeleyTraining the Next Generation of Biologists and Engineers for Regenerative Medicine    $4,954,238
EDUC4-12792UC DavisCIRM Cell and Gene Therapy Training Program 2.0    $4,966,300
EDUC4-12802Children’s Hospital of Los AngelesCIRM Training Program for Stem Cell and Regenerative Medicine Research    $4,999,500
EDUC4-12804UC San DiegoInterdisciplinary Stem Cell Training Grant at UCSD III    $4,992,446
EDUC4-12811ScrippsTraining Scholars in Regenerative Medicine and Stem Cell Research    $4,931,353
EDUC4-12812UC San FranciscoScholars Research Training Program in Regenerative Medicine, Gene Therapy, and Stem Cell Research    $5 million
EDUC4-12813Sanford BurnhamA Multidisciplinary Stem Cell Training Program at Sanford Burnham Prebys Institute, A Critical Component of the La Jolla Mesa Educational Network    $4,915,671  
EDUC4-12821UC Santa BarbaraCIRM Training Program in Stem Cell Biology and Engineering    $1,924,497
EDUC4-12822UC IrvineCIRM Scholars Comprehensive Research Training Program  $5 million
EDUC4-12837Lundquist Institute for Biomedical InnovationStem Cell Training Program at the Lundquist Institute    $4,999,999

These are not the only awards we make to support training the next generation of scientists. We also have our SPARK and Bridges to Stem Cell Research programs. The SPARK awards are for high school students, and the Bridges program for graduate or Master’s level students.

UCSF Nursing Professor Joins CIRM Board

Elena Flowers, PhD, RN, newest member of the CIRM Board: Photo courtesy UCSF

Elena Flowers, PhD, RN, an associate professor of physiological nursing at the University of California, San Francisco (UCSF) is joining the Board of the California Institute for Regenerative Medicine (CIRM), the state’s Stem Cell Agency.

Dr. Flowers was appointed to the Board by State Controller Betty T. Yee who said: “Ms. Flowers’ experience and express commitment to equitable health outcomes for California’s diverse communities will bring a valued perspective to the work ahead.”

Dr. Flowers is a member of the UCSF Institute for Human Genetics and the International Society of Nurses in Genetics. As a researcher her work focuses on genomics involving precision medicine and risk factors for cardiovascular health and type 2 diabetes. She is also a teacher and has lectured internationally on issues such as topics from racial disparities in Type 2 Diabetes to the implications of genomic technologies for the nursing workforce.

CIRM Board Chair, Jonathan Thomas, PhD, JD, welcomed the appointment: “Dr. Flowers brings a wealth of experience and expertise to our Board and, as a nurse, she will bring a different perspective to the work we do and help us in trying to better address the needs of underserved communities.” 

“I am honored to have the opportunity to serve the citizens of California in this capacity,” says Dr. Flowers. “CIRM has ambitious goals, seeking to improve upon common limitations of public research agencies by its commitment to delivering meaningful findings and ultimately treatments for patients as rapidly as possible. I’m particularly committed to improving inclusion and access to these treatments across the entire diverse California population.”

Dr. Flowers got her undergraduate degree at UC Davis and then served as a research assistant at Zuckerberg San Francisco General Hospital. She then went on to get her MS and Doctor of Philosophy degrees at the UCSF School of Nursing.

In her spare time she has no spare time because she is the mother of two young daughters.

Partners in health

From left to right: Heather Dahlenburg, Jan Nolta, Jeannine Logan White, Sheng Yang
From left to right: Heather Dahlenburg, staff research associate; Jan Nolta, director of the Stem Cell Program; Jeannine Logan White, advanced cell therapy project manager; Sheng Yang, graduate student, Bridges Program, Humboldt State University, October 18, 2019. (AJ Cheline/UC Davis)

At CIRM we are modest enough to know that we can’t do everything by ourselves. To succeed we need partners. And in UC Davis we have a terrific partner. The work they do in advancing stem cell research is exciting and really promising. But it’s not just the science that makes them so special. It’s also their compassion and commitment to caring for patients.

What follows is an excerpt from an article by Lisa Howard on the work they do at UC Davis. When you read it you’ll see why we are honored to be a part of this research.

Gene therapy research at UC Davis

UC Davis’ commitment to stem cell and gene therapy research dates back more than a decade.

In 2010, with major support from the California Institute for Regenerative Medicine (CIRM), UC Davis launched the UC Davis Institute for Regenerative Cures, which includes research facilities as well as a Good Manufacturing Practice (GMP) facility.

In 2016, led by Fred Meyers, a professor in the School of Medicine, UC Davis launched the Center for Precision Medicine and Data Sciences, bringing together innovations such as genomics and biomedical data sciences to create individualized treatments for patients.

Last year, the university launched the Gene Therapy Center, part of the IMPACT Center program.

Led by Jan Nolta, a professor of cell biology and human anatomy and the director of the UC Davis Institute for Regenerative Cures, the new center leverages UC Davis’ network of expert researchers, facilities and equipment to establish a center of excellence aimed at developing lifelong cures for diseases.

Nolta began her career at the University of Southern California working with Donald B. Kohn on a cure for bubble baby disease, a condition in which babies are born without an immune system. The blood stem cell gene therapy has cured more than 50 babies to date.

Work at the UC Davis Gene Therapy Center targets disorders that potentially can be treated through gene replacement, editing or augmentation.

“The sectors that make up the core of our center stretch out across campus,” said Nolta. “We work with the MIND Institute a lot. We work with the bioengineering and genetics departments, and with the Cancer Center and the Center for Precision Medicine and Data Sciences.”

A recent UC Davis stem cell study shows a potential breakthrough for healing diabetic foot ulcers with a bioengineered scaffold made up of human mesenchymal stem cells (MSCs). Another recent study revealed that blocking an enzyme linked with inflammation enables stem cells to repair damaged heart tissue. A cell gene therapy study demonstrated restored enzyme activity in Tay-Sachs disease affected cells in humanized mouse models.

Several cell and gene therapies have progressed to the point that ongoing clinical trials are being conducted at UC Davis for diseases, including sickle-cell anemia, retinopathy, muscle injury, dysphasia, advanced cancer, and Duchenne muscular dystrophy, among others.

“Some promising and exciting research right now at the Gene Therapy Center comes from work with hematopoietic stem cells and with viral vector delivery,” said Nolta.

Hematopoietic stem cells give rise to other blood cells. A multi-institutional Phase I clinical trial using hematopoietic stem cells to treat HIV-lymphoma patients is currently underway at UC Davis.

.Joseph Anderson

Joseph Anderson

“We are genetically engineering a patient’s own blood stem cells with genes that block HIV infection,” said Joseph Anderson, an associate professor in the UC Davis Department of Internal Medicine. The clinical trial is a collaboration with Mehrdad Abedi, the lead principal investigator.

“When the patients receive the modified stem cells, any new immune system cell, like T-cell or macrophage, that is derived from one of these stem cells, will contain the HIV-resistant genes and block further infection,” said Anderson.

He explained that an added benefit with the unique therapy is that it contains an additional gene that “tags” the stem cells. “We are able to purify the HIV-resistant cells prior to transplantation, thus enriching for a more protective cell population.

Kyle David Fink

Kyle David Fink

Kyle David Fink, an assistant professor of neurology at UC Davis, is affiliated with the Stem Cell Program and Institute for Regenerative Cures. His lab is focused on leveraging institutional expertise to bring curative therapies to rare, genetically linked neurological disorders.

“We are developing novel therapeutics targeted to the underlying genetic condition for diseases such as CDKL5 deficiency disorder, Angelman, Jordan and Rett syndromes, and Juvenile Huntington’s disease,” said Fink.

The lab is developing therapies to target the underlying genetic condition using DNA-binding domains to modify gene expression in therapeutically relevant ways. They are also creating novel delivery platforms to allow these therapeutics to reach their intended target: the brain.

“The hope is that these highly innovative methods will speed up the progress of bringing therapies to these rare neurodegenerative disease communities,” said Fink.Jasmine Carter, a graduate research assistant at the UC Davis Stem Cell Program.

Jasmine Carter, a graduate research assistant at the UC Davis Stem Cell Program, October 18, 2019. (AJ Cheline/UC Davis)

Developing potential lifetime cures

Among Nolta’s concerns is how expensive gene therapy treatments can be.

“Some of the therapies cost half a million dollars and that’s simply not available to everyone. If you are someone with no insurance or someone on Medicare, which reimburses about 65 percent, it’s harder for you to get these life-saving therapies,” said Nolta.

To help address that for cancer patients at UC Davis, Nolta has set up a team known as the “CAR T Team.”

Chimeric antigen receptor (CAR) T-cell therapy is a type of immunotherapy in which a patient’s own immune cells are reprogrammed to attack a specific protein found in cancer cells.

“We can develop our own homegrown CAR T-cells,” said Nolta. “We can use our own good manufacturing facility to genetically engineer treatments specifically for our UC Davis patients.”

Although safely developing stem cell treatments can be painfully slow for patients and their families hoping for cures, Nolta sees progress every day. She envisions a time when gene therapy treatments are no longer considered experimental and doctors will simply be able to prescribe them to their patients.

“And the beauty of the therapy is that it can work for the lifetime of a patient,” said Nolta.