Dr. Joel Kopple on Nutritional Therapy for Chronic Kidney Disease
A Full Plate: Pursuing A PhD and Raising a Family at the Same Time
The New Farima Czyzyk Center for Cardiac Research and Wellness
FALL 2023
For over 70 years, The Lundquist Institute has been saving lives.
Here are some of the innovations developed here at the Institute:
• The successful fertilization through artificial implantation of the ovum, which would lead to the first ovum transfer birth 20 years later.
• The first paramedic model and first nurse practitioner training program
• The test for high cholesterol that is still in use today.
• The first Thyroid Deficiency Test for newborns
• Tens of millions of children born prematurely are alive today due to the development of Synthetic Lung Surfactants that allow newborns with underdeveloped lungs to breathe.
• The manufacture of low-cost eye drops to treat eye infections in newborns, especially effective in developing countries, saving the eyesight of hundreds of thousands.
• The creation of Enzyme Replacement Therapy to treat Hurler-Scheie Syndrome
• The invention and production of infant heart monitors to prevent SIDS, and test for congenital heart disorders in newborns
• The first treatment for sickle cell disease ever approved for children
And so much more…from groundbreaking work in developing the first male contraceptive to developing therapies to prevent and control fungal infections and diseases, our trailblazing research continues.
A Public Ation of t he lundquist i nstitute
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2 PRESIDENT’S MESSAGE Post-pandemic Productivity
4 MEET THE BOARD
i ntroducing b oard Member f red c hristie, a longtime prominent figure in the energy industry.
6 CELEBRATING THE CAREER OF CARDIOLOGIST DR. DAVID S. CANNOM
Recognized for his groundbreaking discoveries and unwavering commitment to addressing urgent medical challenges.
10 THE LEGENDS OF LUNDQUIST
d r. e mil Kakkis’ astonishing career serves as a testament to the transformative power of perseverance, innovation, and compassion.
12 DONOR NEWS
We recognize the generous gifts from our benevolent patrons.
16 A FULL PLATE: PURSUING A PHD AND RAISING A FAMILY AT THE SAME TIME
7 VIEWPOINT
d r. s cott f iller on t he nevitable f ungal Pandemic.
b alancing the rigors of research with the responsibilities of parenthood may not be easy, but it can be a journey filled with immense joy, and personal growth.
18 SCIENCE NEWS
We present some of the latest news highlights from our investigators.
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P L ease s hare Your Though T s Wi T h u s
We want to hear from you. Send your thoughts, comments, and feedback to: editorreach@lundquist.org
Fo LLo W u s on socia L M edia @thelundquistinstitute @the_lundquist thelundquistinstitute
The Lundquist Institute for Biomedical Innovation
8 MEET OUR SCIENTISTS
d elphine lee, M d, Ph d, an immunologist and dermatologist who combines her expertise in bioinformatics and computational biology to tackle complex questions related to autoimmune diseases, autoinflammation, and cancer.
22 FUTURE TENSE, PREVENTING AND TREATING CHRONIC KIDNEY DISEASE d r. Joel d. Kopple explores the n utritional t herapy for the Prevention and treatment of c hronic Kidney d isease.
24 SECOND LUNDQUIST LITTLE EINSTEINS CLASS GRADUATES
t he second l ittle e insteins class of 38 elementary school students who are taught basic science topics by lundquist Ph d students graduated in June 2023.
“ We are bringing promising students at all levels into experiences in biomedical research starting with Little Einsteins, our program for STEM education for elementary school students, through our High School Summer Fellows and college undergrad internship programs and culminating with our PhD Program in Translational Research. ”
At many levels, the Pandemic slowed TLI’s research down, but not by much. Most affected were our clinical trials, where recruiting subjects became difficult, if not impossible. The lab-based research moved forward, first on a “shift” basis where not all staff were back on campus at once, and not too soon thereafter, back up to speed.
During that time, our researchers had more time to submit grant applications, and with marked success. Funding for individual investigators, largely from government agencies, is in the millions.
I am especially pleased that this level of funding is not focused only on our most senior and accomplished researchers, but millions of dollars are now supporting some of our early-career investigators, recruited in 2020, during the height of the pandemic.
I am particularly pleased with our “Educational Pipeline” some of which is featured in this issue of REACH. We are bringing promising students at all levels into experiences in biomedical research starting with Little Einsteins, our program for STEM education for elementary school students, through our High School Summer Fellows and college undergrad internship programs and culminating with our PhD Program in Translational Research. We are now looking into the potential for bringing our Little Einsteins program into local schools, as well as implementing an educational program at the middle school level, completing a continuum from grammar school through graduate education. The outcomes of each are clear: We have motivated students at all levels for careers in research. The next generation!
The diversity of research interests at TLI is our strength, and from my perspective, the most exciting aspect of my role. As you will see, from Scott Filler’s “Viewpoint,” we are on top of research that could well prevent an upcoming pandemic: Drug-resistant fungal infections.
Joel Kopple’s “Future Tense” demonstrates the importance of nutrition in prevention and treatment of kidney disease.
“Meet our Scientists” introduces you to Delphine Lee, MD, PhD, a multi-talented physician scientist with myriad roles in the clinic and the lab. Not to forget our illustrious past, there is an article on our Legend, Emil Kakkis, MD, PhD, who led the quest for “enzyme replacement therapy” that has saved the lives of thousands of kids who were destined to succumb from inherited diseases by ages 10-12 years. We also feature a piece on another Legend, David Cannom, MD, who was recently honored at a major retirement event sponsored by TLI. There is a lot more to touch on, but suffice it to say, we are publishing important research in top journals, obtaining financial support from agencies as well as donors to move our discoveries forward to therapies, devices, and patient care, all in an atmosphere of collegiality, collaboration, and exchange of ideas. Enjoy this issue of REACH.
Little Einsteins Program students shown winding DNA out of strawberries. When you crush strawberries, the cells inside them get broken open, and this is where the DNA is located. Shampoo contains soap that breaks down the cell membranes and releases the DNA. Salt is added to make the DNA molecules stick together and separate from the proteins that are also released from the cells.
David I. Meyer, PhD President and CEO
Tall, thin, and distinguished looking with a shock of white hair, H. Frederick Christie, who goes by Fred, has an air of gravitas balanced by an easy smile and demeanor. Today he is a retired executive and longtime prominent figure in the energy industry. Above all, he is someone who has committed his life to serving the greater good. As a member of the Lundquist Institute’s Board of Directors, he has extended his wide-ranging interests into the world of biomedicine.
From 1983-1987 Fred served as President and Director of Southern California Edison, (SCE) a subsidiary of the Mission Group. His association with Southern California Edison began in 1957 after completing his undergraduate studies in finance at the University of Southern California. He later obtained a master’s degree in management in 1962.
Born in Alhambra, California, Christie attended local schools before
joining the U.S. Army to have his tuition covered. His father’s affiliation with Southern California Edison influenced Christie’s decision to join the company where he spent his entire professional career. His tenure at SCE as an executive was marked by challenges, including dealing with regulatory issues and the complexities surrounding nuclear fuel. As Chief Financial Officer and later as President he dealt with numerous public hearings where sometimes different views on matters clashed and he even received death threats.
His service, spanning a professional career of over four decades, included serving on twelve New York Stock Exchange Company Board of Directors and he was also affiliated with six board clusters of mutual funds. While serving on those boards, he held leadership positions, including the role of chair on three of the boards, and as lead director on two of the boards.
In addition to his accomplishments in the corporate and financial worlds, Christie has made significant contributions to the community. He is currently Chairman Emeritus of the
Los Angeles Natural History Museum and has sat on the boards of the Music Center, the L.A. Chamber of Commerce, Occidental College, Chadwick School, United Way of Los Angeles, and the Chair of the Multiple Sclerosis Society of Los Angeles. Christie’s expertise and experience have also led him to share his knowledge as a guest lecturer at UCLA, Stanford University, New York University, and the University of Maryland.
A major pursuit of Fred’s, currently, is serving as a Lundquist Institute Board member. His wife, Sue, introduced him to the Institute, and he became intrigued by its mission and work. That interest eventually evolved into his joining the Board. For him, this was a chance to become involved in and support the cutting-edge research that is the hallmark of the Institute’s impact. He has brought his storehouse of knowledge regarding corporate governance to the Institute. He understands the fine balance between fulfilling regulatory responsibilities and allowing the organization’s experts to lead in their respective fields.
Fred says, “I have to say that the Institute plays a vital role in today’s complex world. As a nonprofit organization, it relies on successful research funding from various sources as well as individual contributions. I find great excitement in observing the Institute’s progress and witnessing
“I find great excitement in observing the Institute’s progress and witnessing the development of new therapies and products that contribute to healing and advancing medical practices worldwide.”
the development of new therapies and products that contribute to healing and advancing medical practices worldwide.”
Beyond his board duties, he enjoys biking, taking long walks with his dog, and traveling the world with his wife. They have explored countries all over the globe, amassing experiences and broadening their horizons. He also cherishes his time with his family and recently celebrated his first great-grandchild’s first birthday. He says it was quite fun. ●
“ The celebration of Dr. Cannom's retirement was a testament to his achievements and dedication to advancing cardiovascular medicine. ”
The Institute recently mounted a special event to celebrate the exceptional career of renowned cardiologist and Lundquist Legend, Dr. David S. Cannom. Recognized for his groundbreaking discoveries and unwavering commitment to addressing urgent medical challenges, Dr. Cannom’s retirement was honored by hundreds of physicians and supporters at the Torrance Marriott on April 8th. With over 56 years of experience in in cardiology, Dr. Cannom’s expertise spans diverse aspects of cardiac function, including but not limited to atrial fibrillation, familial dysautonomia, orthostatic hypotension, cardiac ablation, pacemaker implantation, and cardiac electrophysiology.
Reflecting on the touching tribute to his long career, Dr. Cannom expressed his gratitude, stating, “I’ve been swept off my feet by the wonderful tributes given to me tonight. I didn’t think these types of events could be this moving. I’m so happy I am seen in this positive light, and this evening will stay with me always. My thanks to all.”
Dr. Cannom has co-authored 150 peer-reviewed articles, solidifying his reputation as a remarkable and influential researcher. His proficiency in treating ventricular arrhythmias has garnered attention worldwide. Moreover, he had held positions such as past president of the Los Angeles Affiliate of the American Heart Association, past governor of the American College of Cardiology in California, and immediate past president of NASPE (North American Society for Pacing & Electrophysiology).
Dr. Cannom’s wife, Phyllis Monroe, MD, has been a staunch supporter of The Lundquist Institute, having served a number of years on its Board of Directors. The celebration of Dr. Cannoms’ retirement was a testament to his achievements and dedication to advancing cardiovascular medicine. As he embarks on the next chapter of his life, the impact of his contributions will continue to resonate in the field of cardiology. His legacy reaffirms the importance of innovative research and compassionate patient care in shaping the future of healthcare. ●
A video of the evening’s festivities can be seen here: https://vimeo.com/816360651
WHEn PEOPLE THInK OF EPIDEMIcS, they usually think of bacteria, like that which causes plague, or viruses, such as smallpox and SARSCoV-2. While bacteria and viruses have killed millions of people over the years, a new class of microbial pathogens is on the rise. Driven by climate change, fungi are emerging as important causes of disease in humans and other animals. One reason that only a relatively small number of fungi infect humans is that most fungi prefer cool temperatures and are unable to grow at 37°C—the temperature of the human body. As the world gradually heats up due to climate change, fungi are adapting, growing in warmer temperatures, and developing the capacity to infect humans. An example is the fungus Candida auris which was only first discovered in 2009 in Japan, but emerged as a cause of disease in humans in three different continents almost simultaneously. Unlike its closely related species, C. albicans, C. auris can grow at 37°C. It is resistant to common disinfectants and can persist on both patients and inanimate objects for weeks, facilitating the spread of the fungus between patients, especially those living in close quarters such as in nursing homes. Even worse, the fungus is usually resistant to many antifungal drugs and some strains are resistant to all these drugs. Outbreaks of C. auris infections are steadily increasing in the USA, especially in California, New York, and Texas. Many fungi only cause severe infections in patients who have weakened immune systems due to conditions such as HIV/AIDS, cancer chemotherapy, organ transplantation or steroids. However, some fungi such as Coccidioides, which causes valley fever, and Histoplasma, which causes histoplasmosis can infect otherwise healthy individuals. Both fungi can cause pneumonia, and, in some patients, they can spread throughout the body, causing a life-threatening disseminated infection.
“ The Institute for Infection and Immunity at The Lundquist Institute has one of the largest groups of fungal investigators in the country. They are working on new approaches to prevent and treat fungal infections.”
Due to climate change, the range of these fungi is steadily increasing. Valley fever used to be found mainly in Arizona and California, but due to the warming climate, it is now found throughout the western US. While histoplasmosis was previously limited to the Ohio and Mississippi River valleys, patients with this infection have been reported in virtually all states. Patients with these infections, especially valley fever, are often treated at Harbor-UCLA Medical Center.
Due to the limited number of antifungal drugs and no antifungal vaccines, fungal infections are difficult to prevent, and patients die from these infections, even with currently available treatment. But there is hope. The Institute for Infection and Immunity at The Lundquist Institute has one of the largest groups of fungal investigators in the country. They are working on new approaches to prevent and treat fungal infections. One promising project is the development of a vaccine to prevent C. auris infections. The Lundquist Institute is also raising funds to construct a building with a high-containment BSL-3 facility, which is necessary for studying the fungi that cause valley fever and histoplasmosis. Through these efforts, the Lundquist Institute is at the forefront of preventing the next fungal epidemic, which by many has been characterized as the next pandemic. ●
Honoree Dr. David Cannom pictured with Dr. David Meyer, Lundquist President & CEO at the retirement dinner.
Pioneering translational Research at the intersection of immunology and dermatology
In the realm of scientific research, it is common to find specialists focused on a defined area of a specific field. There are, however, some exceptional scientists who thrive on the diversity and variety of their work. One such scientist is Delphine Lee, MD, PhD, an immunologist and dermatologist who combines her expertise in bioinformatics and computational biology to tackle complex questions related to autoimmune diseases, autoinflammation, and cancer. Dr. Lee’s interdisciplinary approach, fueled by her training and diverse skill set, allows her to leverage big data and clinical patient information to gain novel insights and develop innovative hypotheses.
As a young child, she harbored a deep desire to become a physician, although the origins of this aspiration remain mysterious to her. Her journey into the world of medical research began at the University of California, San Diego (UCSD), where she pursued a combined MD-PhD program. However, it was during that time in medical school that the spark of scientific curiosity ignited within her. While attending monotonous lectures and memorizing facts, Dr. Lee yearned for a more stimulating and intellectually challenging path.
Her interest in research was piqued when she observed the devastating impact of pediatric AIDS. Intrigued by the scope of the problem and driven to find a solution, she delved into the world of wet bench research and published papers that explored the complexities of HIV. This experience not only allowed her to learn the intricacies of scientific thinking, but it also opened her eyes to the power of combining different scientific disciplines. Inspired by the discovery of a mechanism of cellular suicide called apoptosis, she embarked on a journey to understand and explore this phenomenon. This quest led her to pursue a PhD, as she began to realize that her ambitions extended beyond clinical medicine into the area of deep scientific research. “Learning about apoptosis was a turning point for me,” says Dr. Lee. “It opened up a whole new world for me and set me off in a new direction.”
Through her career, Dr. Lee has been fortunate to have mentors who shaped her scientific endeavors. Dr. Maripat Corr, a clinical fellow in rheumatology and the director of the Corr Lab at UCSD, taught her the importance of rigor in science, critical thinking skills, and the power of practicality. These qualities have not only contributed to her success in research but have also aided her leadership skills and strategic decision-making.
One influential mentor was Dr. Dennis Carson, her PhD advisor at UCSD. Dr. Carson’s achievements in developing a drug to cure hairy T-cell leukemia motivated Dr. Lee to strive for similar breakthroughs in disease treatment and became an instrumental force in shaping her career.
Dr. Robert Modlin, a distinguished professor at the David Geffen School of Medicine at UCLA, played a pivotal role during Dr. Lee’s postdoctoral reseach in terms of her professional growth and development. It was under his guidance that she was introduced to the field of bioinformatics and the immense potential of harnessing big data to extract valuable information from limited tissue samples. Her training enabled Dr. Lee to begin integrating molecular and biochemical data with clinical features of actual patients and their disease outcomes that have yielded novel insights in a wide range of diseases including leprosy, breast cancer, colon cancer, melanoma, and her current focus on psoriasis and inflammatory skin disease.
Dr. Lee’s career took a significant turn when she encountered Dr. Donald Morton, a brilliant surgical oncologist. Recognizing that she possessed the capacity for tackling complex scientific inquiry, he encouraged her to explore the world of melanoma research. She was intrigued by the
parallel between vitiligo, a condition where the immune system attacks melanocytes, and melanoma, a cancer arising from melanocytes. She eagerly accepted the challenge. Her transition to melanoma research allowed her to examine both sides of the coin, studying the mechanisms of immune protection in skin diseases and the development of skin cancer.
Dr. Lee’s daily routine is multifaceted and combines several critical aspects of her work. In addition to teaching clinical medicine, she engages in both lecture-style teaching and bedside teaching, providing hands-on guidance to medical students and residents. Her research activities involve analyzing results, planning experiments, and strategizing the analysis of large datasets. Dr. Lee also participates in meetings aimed enhancing clinical care in dermatology for underserved populations. Through her contributions, she aims to bring us closer to understanding disease mechanisms and finding effective solutions and treatments. ●
Dr. Emil Kakkis is a one-of-a-kind researcher and medical scientist. He has dedicated his entire career to developing treatments for rare and ultrarare diseases. His groundbreaking work in enzyme replacement therapy (ERT) has saved many lives, offering hope to patients with rare diseases that were once considered incurable. Driven by a passion to make a difference, Kakkis’ journey began in the humble surroundings of a World War II-era Army bungalow at The Lundquist Institute, where he conducted pioneering research that would revolutionize the field of ERT.
During the early 1990s, Kakkis embarked on a fellowship in medical genetics. It was during this time that he crossed paths with Dr. Elizabeth Neufeld, a renowned geneticist who had cloned the gene responsible for the enzyme alpha-L-iduronidase, a crucial enzyme defective in Mucopolysaccharidosis (MPS) Type I. This breakthrough opened up new possibilities for large-scale production of the enzyme and the potential for treating patients through ERT. Little did Kakkis know then that this seemingly short-term research project would shape his entire medical
research career.
The primary challenge Kakkis and his colleagues faced was producing sufficient alpha-L-iduronidase for effective treatment. While Kakkis possessed the necessary knowledge in molecular biology, he had never been involved in scaled-up manufacturing. Nevertheless, armed with his expertise and a big dose of determination, he embarked on the journey of creating an expression vector—a specialized DNA piece containing the alpha-L-iduronidase gene—and introducing it into a cultured cells capable of producing the required enzyme. After an extensive period of experimentation and refinement, Kakkis and his team successfully treated the first animal with MPS I in the fall of 1992.
Their work continued in the cramped quarters of bungalows C3 and E4 at the Institute. Despite facing
financial constraints, Kakkis’ dedication attracted the support of the Dant family, which spearheaded fundraising efforts to ensure the project’s continuation. Their son, Ryan, had been diagnosed with MPS I. However, the path to treating human patients proved to be a daunting task, necessitating strict quality control systems and substantial funding. A turning point came with the establishment of BioMarin, a new company that recognized the potential of Kakkis' research, and provided a substantial grant to further the work. With this funding in hand, Kakkis and his team transformed bungalow E4 into a state-of-the-art Good Manufacturing Practices (GMP) lab, allowing the team to manufacture the enzyme and conduct clinical trials. In a remarkable nine-month period, Kakkis and his team completed the required construction, developed a manufacturing process, obtained regulatory approval and produced the first lot of the enzyme for treatment. The culmination of their efforts came just two days before the first patient received the therapy. The fact that their operation encompassed both manufacturing and clinical trials within the confines of the Lundquist Institute exemplified their groundbreaking vision and the Institute’s overall longtime commitment to finding innovative solutions.
While ERT is not a cure for MPS I, it has undoubtedly been lifechanging for many patients. Ryan Dant is a case in point. When first introduced to Kakkis as a child, Ryan’s life expectancy was a few years. He might survive to 10 or 12 years of age. With the ERT treatment, he is now a 34-year-old adult with a college degree and married, living a full life.
“We now have the science and the ability to change the future for people with devastating genetic diseases,” says Kakkis. “When you can make that much of a difference in someone’s life, you find a way to keep moving toward that
“ While ERT is not a cure for MPS I, it has undoubtedly been life-changing for many patients. Ryan Dant is a case in point. When first introduced to Kakkis as a child, Ryan’s life expectancy was a few years. He might survive to 10 or 12 years of age. With the ERT treatment, he is now a 34-year-old adult with a college degree and married, living a full life.”
goal. When we pitched the unorthodox plan to transform our bungalow lab space into a manufacturing suite to develop a first-ever treatment for MPS I, the Institute supported us in the spirit of innovation and discovery. That support made all the difference for Ryan and the many other children who have received this life-changing treatment that was first developed, manufactured and tested at The Lundquist Institute.”
The treatment developed by Kakkis and his team requires weekly infusions, providing the body with the necessary enzyme levels it lacks. While other potential treatments, such as gene therapies, are being explored, Kakkis emphasizes the importance of ERT in providing consistent relief. Throughout his career, he has led the development of eleven FDA-approved therapies, transforming the lives of thousands of people living with rare disease. He founded the EveryLife Foundation for Rare Diseases to empower the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures. And he is the founder, president and CEO of Ultragenyx, a biopharmaceutical company dedicated to developing novel treatments for rare and ultrarare genetic diseases. His unwavering dedication has contributed to a growing recognition of rare diseases and the possibilities of treatment, which has, in turn, attracted vital new talent to the field.
Dr. Emil Kakkis’ astonishing journey from the old Army bungalows at The Lundquist Institute to the forefront of rare disease research serves as a testament to the transformative power of perseverance, innovation, and compassion. This is why Emil Kakkis is a Lundquist Legend. ●
Million
the new farima Czyzyk Center for Cardiac research and wellness opened in April 2023 and will provide lifesaving, low-cost heart scanning services for underserved patients in the lundquist institute community service areas of south los Angeles.
The Lundquist Institute received a $1.5 million gift from Farima and Joseph Czyzyk to make its life-saving imaging technology, used to find signs of heart disease, osteoporosis, and other diseases in their early stages, more accessible to the general public. In recognition of this generous gift, the Institute’s Diagnostic and Wellness Center was renamed the Farima Czyzyk Center for Cardiac Research and Wellness.
The Czyzyks’ gift is being used primarily to support the work of Dr. Matthew Budoff, whose research is devoted to advancing procedures that can help doctors in the early identification of patients who are at high-risk for cardiac events and the progression of atherosclerosis.
Dr. Budoff’s research at The Lundquist Institute also focuses on determining the effect of different therapies on atherosclerosis and determining if heart disease can be reversed. His team has also shown utility of supplementary measurements on cardiac CT can be evaluated for thoracic aortic calcification, aortic valve calcification, mitral annular calcification, pericardial fat, liver fat, myocardial scar, and bone mineral density.
“As a patient, I have benefited from this screening technology at Harbor-UCLA,” said Farima Czyzyk, adding, “and I am very impressed with Dr.
Budoff’s work and mission.”
Czyzyk also said that those who lack the means too often forego critical preventative care that could ultimately save or extend their lives.
Czyzyk, who was a registered and public health nurse, began her career on the Harbor-UCLA campus as a Nurse Epidemiologist working in the Division of Infectious Diseases at what was then Harbor General Hospital. After several years she left the hospital for higher education to further her career in healthcare and hospital administration.
“I’ve been in healthcare long enough to know that preventative care needs improvement,” she said, adding, “So many lives can be saved if more people can use this technology. Dr. Budoff is a gem. If I can help anyone advance their work, I want it to be him.”
“The endorsement, by the Czyzyks of the work at the Lundquist Institute, and especially Dr. Budoff’s innovative research, is a validation of the impact that The Lundquist Institute is having in developing cutting-edge programs that improve people’s lives, daily,” according to Dr. David Meyer, PhD, President & CEO of The Lundquist Institute. “We appreciate this vote of confidence through the establishment of The Farima Czyzyk Center for Cardiac Research and Wellness.” ●
The Joe & Sandy Samberg Foundation has made a gift of $300,000 to The Lundquist Institute (TLI) to study the use of psilocybin in alleviating demoralization and the psychological distress associated with life-threatening illness in palliative care. The gift will support the work of Charles Grob, MD, an investigator at TLI and professor at the David Geffen School of Medicine at UCLA and Anthony P. Bossis, PhD, an investigator at TLI. The gift will facilitate an FDA-approved multi-site clinical trial.
Dr. Grob’s research has included the first FDA-approved Phase 1 study of the physiological and psychological effects of MDMA (3,4-methlyenedioxymethamphetamine); a multi-national, collaborative study of the Amazonian plant hallucinogen decoction, ayahuasca, in Brazil; and a pilot investigation of the safety and efficacy of psilocybin in the treatment of anxiety in adult patients with advanced-stage cancer. Dr. Bossis was director of palliative care research and co-principal investigator on a 2016
“ The trial found that a single psilocybin session led to a rapid decrease in depression, anxiety, hopelessness, and demoralization, as well as improvements in spiritual well-being and quality of life.”
New York University clinical trial and publication that demonstrated the significant potential of psilocybin in reducing emotional distress in persons with cancer. Specifically, the trial found that a single psilocybin session led to a rapid decrease in depression, anxiety, hopelessness, and demoralization, as well as improvements in spiritual well-being and quality of life.
“We are excited by the unique and comprehensive design of this trial, including the apprenticeship model
for the facilitators and the use of a novel control condition,” said Sandy Samberg, President of the Samberg Foundation. “With the renowned leadership team, we’re confident this study will be well executed and that the findings will be applicable for real-world settings.”
“I am delighted to have received this grant from the Samberg Foundation to further the study of psilocybin in patients suffering from psychological distress associated with life-threatening and terminal illness,” said Dr. Grob. “Clearly, more work is needed in this area. Our earlier research with psilocybin suggests that improved mood and lessening of death anxiety and demoralization can occur, even in the context of extreme and dire health circumstances. I look forward to continuing our work in this area as it is vital to continue to explore ways to minimize the anxiety, pain, and psychospiritual distress even in the face of impending death.” ●
Dr. Ronald Fraback, an accomplished rheumatologist and former resident at Harbor-UCLA, has dedicated his life to improving the well-being of his patients and building a healthier world. His time as a resident at Harbor-UCLA Medical Center made a lasting impression on him and inspired his longstanding support of The Lundquist Institute.
Throughout his career, Dr. Fraback has made significant contributions to medicine in patient care, teaching, and medical staff leadership. His work has positively impacted the lives of countless patients with rheumatic disease. Although now residing in the Pacific Northwest, he laid the foundation for his achievements in Southern California.
As a medical resident at Harbor-UCLA in the 1970s, he trained under esteemed Lundquist Legend Kouchi Tanaka, MD, as well as with James Louie, MD, chief of rheumatology during Dr. Fraback’s time at Harbor. Dr. Fraback attributes his success as a doctor to the guidance and mentorship he received from Drs. Tanaka and Louie, and these formative experiences inspired him to make his first gift shortly after completing the residency program.
For more than four decades, Dr. Fraback has been a dedicated
donor, annually contributing to The Lundquist Institute’s life-saving research and innovative initiatives. Through his recent planned gift, he’s pleased that his support will continue to benefit generations who will pass through the Institute’s doors. By pledging a percentage of his estate, Dr. Fraback demonstrates his unwavering commitment to building a healthier world, not only for his patients but for the broader community.
Reflecting on his support of the Institute through the years, Dr. Fraback says, “My time on campus was truly transformative. Drs. Tanaka and Louie played instrumental roles in shaping my career and nurturing my passion for making a difference through medicine. hope that my philanthropy will enable the same crucial mentoring relationships that I benefitted from.”
Planned gifts enable donors to support The Lundquist Institute while achieving their personal financial and charitable goals. To learn more about joining Dr. Fraback in supporting The Lundquist Institute through a planned gift, we encourage you to contact us at development@lundquist.org or call us at (323) 457-1920. ●
The journey of pursuing a PhD is a demanding and challenging endeavor. It requires immense dedication, focus, and hard work. Now, imagine adding the responsibilities of raising a family to this already arduous pursuit. Balancing the rigors of research with the responsibilities of parenthood is no easy task, but it is possible. Out of a group of 20 at The Lundquist Institute, there are four exceptional PhD students who are successfully navigating the challenges of raising a family while working towards their doctoral degrees in Translational Research.
The first of these remarkable students is Ashley Barbarino, who embarked on her PhD journey in 2019. In addition to her coursework, lab work, and study time, Ashley is the proud mother of two daughters, Isabella and Evangelina, aged five and two respectively. She recognizes that her ability to pursue her PhD while raising her children is largely due to having a robust support system. Ashley relies on her parents, in-laws, and her husband, who assist her in caring for the children when she is in the lab or attending courses. Her mentor, Dr. Ashraf Ibrahim, also offers the necessary flexibility in her schedule to ensure she can be present for her girls when needed. Ashley says that without the invaluable understanding and daily support, completing the program would be significantly more challenging.
With four children, Harvey Perez is another inspirational Lundquist PhD student, who joined the program in October 2020. His four children’s ages range from six-years to onemonth old. While balancing the demands of his academic pursuits and family life, Harvey emphasizes the importance of time management and self-care. He readily acknowledges that maintaining a routine for his children helps with time management demands and fosters a sense of stability for his family. However, unforeseen circumstances like illness or disrupted sleep patterns can pose difficulties to maintaining a work life balance. Nonetheless, Harvey finds solace in that fact that his children understand that he is a student who conducts experiments.
Jacqueline (Jackie) Hernandez, a PhD student since 2019, is also the parent of two children. They are three-years and seven-months old respectively. She values the support of her husband and prioritizes tasks to effectively balance family life with her academic pursuits. Like the other PhD students-parents, the challenges of time management and multitasking are always present. Jackie appreciates the positive aspects of being a parent while working towards her doctorate. The patience she has honed through raising children has proven irreplaceable as she conducts her research endeavors. Moreover, having children has influenced her approach to teaching, especially as she has participated with her student-colleagues in designing and developing
the Lundquist Little Einsteins program, which provides science education to elementary school students.
Sunna Nabeela is another PhD graduate student who is a parent. She started the program in 2021. Along with her full-time studies, she is a mother of two children, aged six and ten. Unlike the other parents mentioned, Sunna has a unique situation being an immigrant with no relatives in the United States. Therefore, she completely depends on her husband and help from her lab mates when she is needed as a mother. Sunna receives emotional support from her overseas family, but the day-in, day-out routine struggle is hard with children and her education. The main challenge she faces is keeping their education and non-scholastic activity organized.
Sunna’s daughter and son have been a part of one of the Institute’s hallmark educational pipeline programs, Little Einsteins. Sunna’s daughter understands what it means for her mother to be a graduate student and what research means. Much of her daughter’s understanding is attributed to her participation in the Little Einsteins program, which
has influenced her hope to become a doctor or marine biologist.
“Every day I am impressed by our PhD students who are also parents,” says Jody Spillane, Senior Vice President for Public Affairs and the director of the Institute’s education programs. “Their stories and lives serve as an inspiration for others who may be contemplating or currently walking a similar path, affirming that with the right perspective and support, it is possible to strike a harmonious balance between academic and family life.”
These four PhD students exemplify the resilience and determination required to pursue a doctoral degree while raising a family. Their experiences underscore the importance of having a strong support system, practicing effective time management, and maintaining a positive outlook. Parenting while pursuing a PhD undeniably presents a full plate of responsibilities. While it necessitates sacrifices, meticulous planning, and adaptability to unforeseen circumstances, it is also a journey filled with immense joy, personal growth, and the opportunity to serve as an example for one’s children. ●
A Full Plate: Pursuing a PhD and Raising a Family at the Same Time
the research will apply state-of-the-art science to address antibiotic resistant bloodstream infections caused by staphylococcus aureus (MrsA) and Candida albicans
The Lundquist Institute (TLI) Principal Investigator, Michael Yeaman, PhD, has been awarded a grant totaling $11.5M from the National Institute of Allergy and Infectious Diseases (NIAID), Department of Health & Human Services.
Along with his role at TLI, Dr. Yeaman is Professor of Medicine at UCLA, and Chief, Division of Molecular Medicine at Harbor-UCLA Medical Center.
This new NIH U19 Center program will decode patterns of the human immune system and microbial pathogens that result in infections that are not cleared by antibiotics. In turn, defining such patterns will enable new ways to predict individuals at risk of such persistent infections, and inform best antibiotic regimens for cures. The focus of this U19 is on infections caused by the bacterium Staphylococcus, including methicillin-resistant S. aureus (MRSA) and the invasive fungus, Candida These pathogens are among highest priorities for public health as determined by the NIAID and Centers for Disease Control & Prevention (CDC).
Dr. Yeaman and his team made previous discoveries that laid essential groundwork for this project to
find new ways to prevent and treat persistent bloodstream infections. The new research will take an even more innovative approach to this goal. Traditional genetics focuses on the sequence of DNA to identify potential correlates of infection and immunity. The new U19 award goes further—by exploring patterns in how DNA is modified beyond sequence in ways that impact immune response and antibiotic efficacy against infection. This exciting frontier of biomedical research is called epigenomics.
Sharing the award with Dr. Yeaman will be
TLI Investigator Dr. Scott Filler, expert colleagues from the Immunogenetics Center and Department of Bioengineering at UCLA as well as Duke University Medical Center, plus TLI Co-Investigators Dr. Liana Chan and Dr. Marc Swidergall. They will apply powerful immunological and computational methods to detect hidden epigenomic patterns within vast datasets. The ultimate goal of
this effort is to accelerate discovery and development of new anti-infective agents, immunotherapies, and vaccines to improve and save lives.
“We will use state-of-the-art technologies in synergistic research to under-
stand human-pathogen interactions driving persistence,” said Dr. Yeaman. “The challenge of infections that are resistant to conventional antibiotics is growing every day—and will pose an increasing threat to public health unless we find new, improved ways to predict, prevent and cure these infections.” ●
The Lundquist Institute's and Advent Therapeutics' landmark collaboration has resulted in a significant $3 million Small Business Innovation Research (SBIR) Phase IIB grant from the National Institutes of Health (NIH). The funding will directly support the groundbreaking work of Dr. Virender Rehan, which focuses on developing a first-of-its-kind aerosolized vitamin A formulation. This innovative treatment aims to counteract Bronchopulmonary Dysplasia (BPD), a life-threatening condition affecting premature infants, with an eye towards commercialization by 2025. Each year, BPD, the leading severe complication in premature infants, impacts an estimated 20,000-30,000 newborns in the United States. This condition imposes not only emotional hardship
Jerome Rotter, MD, and Matthew Budoff, MD, two of TLI’s investigators, are co-authors of a article, “Coronary Artery Calcium Score and Polygenic Risk Score for the Prediction of Coronary Heath Disease Events,” in JAMA (the Journal of the American Medical Association). The study assessed whether a coronary artery calcium score was associated with better discrimination than a polygenic risk score for the prediction of cardiovascular disease (CVD) in middle-aged to older adults from the United States and Netherlands.
Both tools — polygenic risk and
coronary artery calcium — are being used in coronary health disease risk assessment but they have never been directly compared head-to-head. The main result of the study showed a statistically significant and clinically meaningful improvement in risk discrimination for middle-aged adults when the coronary artery calcium score was added to a traditional risk factor-oriented model.
“What we found is an excellent way to identify who is at risk for coronary heart disease in middle-age,” said Dr. Rotter, Lundquist Investigator and Director of the TLI Institute for
Translational Genomics and Population Sciences. “The leading cause of death in the U.S. is heart disease and if we can better assess risk by using CT scans, we should move in that direction given what this study suggests. Genetics is an important risk assessment tool but may have a larger clinical role earlier in life.” The study indicates that imaging with computer tomography (CT) to detect coronary artery calcium can be a reliable predictor of CHD.
“Studies showing the ability of coronary artery calcium to predict events have become more frequent,” said Dr. Budoff, Lundquist Investigator and Director of Cardiac CT, Division of Cardiology, Harbor-UCLA Medical Center. “What this paper really identifies is a strong cohort of middle aged and older patients who benefit from the CT screening for heart disease.” ●
on families but also a considerable financial burden on the healthcare system.
“With no approved drugs for BPD, these fragile, at-risk premature infants require prolonged mechanical ventilation or oxygen support, and lengthy hospital stays often resulting in recurring hospitalizations and pulmonary infections. Current treatments are inadequate,” said Dr. Rehan, Principal Investigator at The Lundquist Institute, Professor of Pediatrics at the David Geffen School of Medicine at UCLA, Chief of the Division of Neonatology, and Director of the Neonatal Care Unit, Harbor-UICLA Medical Center.
The aerosolized vitamin A therapy under development by Dr. Rehan and Advent Therapeutics represents a pivotal advancement in neonatal
care. The revolutionary therapy, upon approval, has the potential to non-invasively deliver a safe and effective dose of vitamin A directly to an infant's lungs, significantly reducing the occurrence of BPD.
Dr. Rehan emphasized the critical importance of Advent's groundbreaking inhalable vitamin A therapy, highlighting the profound impact it could have. This innovative product holds the potential to revolutionize treatment by delivering a safe and effective aerosolized vitamin A dose directly to the lungs of neonates. By specifically targeting the lungs, this novel lung-targeted inhalation therapy aims to significantly reduce the development of bronchopulamonary dysplasia (BPD), a condition commonly experienced by fragile newborns. ●
The Lundquist Institute spinoff, Vitalex Biosciences, has been awarded an SBIR Phase 2 grant from NIH’s National Institute of Allergy and Infectious Diseases (NIAID). The grant is for Vitalex’s VX-01, a monoclonal antibody (mAb) platform targeting the debilitating indication of the fungal disease, mucormycosis. This serious fungal infection often occurs in people who are immunocompromised and is spreading throughout the world. Mucormycosis is only curable when diagnosed in its early stages.
Despite debilitating surgical
disfigurement and adjunctive fungal therapy, the overall mortality rate from mucormycosis is 50% and approaches 100% in patients with disseminated disease, persistent neutropenia, or brain infection. With the global prevalence of mucormycosis on the rise including among COVID-19 patients, Vitalex’s VX-01 is a first-in-class mAb therapy targeted to prevent and control the infection and dissemination of the disease.
Partnering with BrevisRefero Corporation for program management and technical oversight, as well as JOINN
Biologics for the process/analytical development and manufacturing of VX-01, and with the support of NIAID/ NIH’s crucial SBIR Phase 2 grant award, Vitalex will be focusing on the scaling-up and manufacturing of VX-01 to allow the completion of its Investigational New Drug (IND)-enabling Good Laboratory Practices (GLP) toxicology study.
“We are very proud to have received this significant and timely award from NIAID,” said Ashraf Ibrahim, an Investigator at The Lundquist Institute and the founder and CEO of Vitalex Biosciences. “This is a significant breakthrough for us and demonstrates the importance of developing and manufacturing an mAb therapy that can prevent this highly infectious disease now spreading throughout the world.” ●
Chronic kidney disease (CKD) is a global public health problem. It is estimated that roughly 9% of adult humans worldwide have CKD. Only a small proportion of these individuals will progress to end-stage kidney failure (ESKF) and require chronic dialysis treatment or kidney transplantation to prevent death. A large proportion of the remainder, however, may die prematurely, primarily from cardiovascular or cerebrovascular disease. There are essentially three ways in which healthy or unhealthy nutrition bears on the incidence and progression of kidney failure. 1. As a cause of illnesses that predispose to developing CKD. 2. As a means of relieving many of the nutritional and metabolic disorders that occur in CKD, especially as chronic kidney failure (CKF) develops. 3. As a treatment to slow the progression of CKD to kidney failure. Let us discuss these three aspects in turn.
1Unhealthy Nutritional Intake Predisposes to CKD. In adults, the two most common causes of ESKF are type 2 diabetes mellitus and hypertension (high blood pressure). Although there are many causes for type 2 diabetes in adults, obesity due to excessive calorie intake is one of the most powerful of these causes. Approximately 38 percent of people with type 2 diabetes will eventually develop CKD due to diabetes. Obesity, even without diabetes mellitus, is associated with an increased incidence of CKD and more rapid progression of CKD toward ESKF. There are also many causes for hypertension, and many of these causes are due to excess nutritional intake or, for some nutrients, deficient intake. The following nutritional intakes or conditions predispose to hypertension: excessive intake of calories (without diabetes), sodium or sodium chloride (salt), and fructose; deficient intake of potassium and fetal underweight or the fetal undernutrition syndrome. High potassium diets, green coffee bean extract, green or oolong or black tea may decrease blood pressure. Also, the Dietary Approaches to Stop Hypertension (DASH), Nordic and Mediterranean diets, which are all high in plant foods and low in saturated animal fat, are associated with reduction in blood pressure. Most impressively among these blood pressure lowering diets is the DASH diet and especially the DASH low sodium diet which lower or normalize blood pressure in people with mild essential hypertension.
2Mitigating Many Nutritional and Metabolic Disorders occurring in CKD. The failing kidney is not able to normally excrete many ingested nutrients or products of the metabolism of ingested nutrients. Some of these chemicals, when they accumulate in the body, may be toxic. The metabolism of proteins and amino acids (proteins are composed of amino acids) generates many chemicals that cause toxicity when they accumulate as a result of reduced excretion by the kidney. In addition, water, and many minerals, especially sodium, potassium and phosphorus may accumulate when the kidney fails. Dietary intake of all these nutrients needs to be regulated to ensure that toxic levels are not attained while at the same time preventing the development of malnutrition. However, even in people with kidney failure who are undergoing treatment with either hemodialysis or peritoneal dialysis, nutritional management is necessary to prevent intake of too much or too little of specific nutrients that can lead to uremic toxicity or malnutrition.
3Dietary Treatment to Slow the Progression of CKD to Kidney Failure. The ability of low protein diets to slow the progression of CKD is rather controversial. Although many research studies suggest that low protein, low phosphorus diets may retard progression of CKD, the largest study did not confirm this possibility. Since this study was initiated, however, new medicines and treatments of CKD have been developed, some of which mimic the physiological actions of low protein diets on the kidney, and they may supplant most or all the effectiveness of low protein, low phosphorus diets on slowing CKD progression. Currently methods for reducing the rate of progression of CKD include the use of medicines that block the actions of the hormones renin, angiotensin and aldosterone, ingestion of diets very high in plant foods that provide an alkaline load to the patient or taking alkalizing medicines, and the use of medicines that inhibit the absorption of glucose (sugar) from the proximal tubules of the nephron. Nonetheless, large numbers of clinical trials of low protein diets for CKD patients do indicate that people with CKD who are prescribed these low protein, low phosphorus diets often do have a delay in the onset of chronic dialysis treatment or kidney transplantation.
In short, there is no substitute for maintaining a proper and healthy diet when it comes not only to preventing CKD and its downstream consequences, but along the way, helping to avoid other metabolic diseases, not to mention obesity. ●
“ There are essentially three ways in which healthy or unhealthy nutrition bears on the incidence and progression of kidney failure.”
The second Little Einsteins class of 38 elementary school students who are taught basic science topics by Lundquist PhD students graduated in June 2023.
This new Institute initiative began offering elementary school students its first set of workshops on science-based curriculum in December of 2021. The Little Einsteins program is part of the Institute’s educational science pipeline which runs from the elementary school level all the way to the PhD level.
“This new endeavor brings together young grammar school students with our PhD students who will mentor them,” said Jody Spillane, Senior Vice President for Public Affairs. “The concept is designed to expose the students to science through a curriculum created by these students. Each month, the elementary school students, ages 8-10, learn about new aspects of science from those working at the cutting edge of discovery.”
The program begins with an overall introduction to the science and the basics of cell biology. In the second lesson, the focus is on human anatomy where the students learn about different
Not just women, but one man after another has come to our Institute, telling us that it was time for men to step up and take responsibility for birth control.
Here at Lundquist, our researchers led by Dr. Christina Wang and her lab have been ahead of the curve and running clinical trials with NIH sponsorship on various male birth control methods including contraceptive gels, oral pills, and long-term injectables.
So far, these trials have shown that a totally reversible inhibition of sperm production could be successful, without showing any change in mood or sexual function.
Once FDA approved and on the market, we already know that more than 40% of unplanned pregnancies would be avoided with better and more effective male contraception options.
The Lundquist Institute has been in operation for over 70 years. One of our hallmarks is knowing that our award-winning researchers work every day at the cutting edge of scientific discovery.
