Miracle Workers


By Alice Short '77, Photos by Alyson Aliano

Published Jul 1, 2019 8:00 AM

Paramedics. Nurse practitioners. Improved survival rates for premature babies. And, most recently, the first new treatment for sickle cell disease in two decades. All of these medical advances, against significant odds, were pioneered by UCLA researchers at the Lundquist Institute for Biomedical Innovation in Torrance.

The gleaming new facility on the Lundquist campus.

Located Just 23 miles south of UCLA on the 405 freeway is a place where amazing medical developments have been quietly taking place for 67 years.

The Lundquist Institute for Biomedical Innovation, located next to Harbor-UCLA Medical Center in Torrance, houses researchers who seem to inhabit a great deal of space on the scientific cutting edge, racking up a remarkable series of advances since the nonprofit organization issued its first grant in 1952.

The Lundquist Institute’s list of breakthroughs includes the invention of the modern cholesterol test, a synthetic lung surfactant for treating premature babies and lifesaving enzyme replacement therapy. The first training program for women’s health nurse practitioners in the country had its start at the institute, and Lundquist researcher John Michael Criley was a driving force behind the use of — and the development of programs to train — paramedics for emergency medicine. The term “paramedic” was actually coined at Lundquist.

This impressive list of advancements might conjure notions of a futuristic campus dotted with sleek modern buildings. But for years, many of the administrators and researchers have toiled in aging structures, some of which look as though they belong on the set of a World War II movie. They are, in fact, evidence of a time when the site was home to a real-life Army hospital, constructed in 1943.

Much of that changed this spring, when the Lundquist Institute opened a gleaming, 78,000-square-foot building on its 11.5-acre campus for researchers, scientists, students and biomedical start-ups. Instead of having to put up with leaky roofs and peeling paint, employees can now take advantage of wet labs, a “freezer farm” for biological samples, an incubator to house and nurture start-ups, and a dry lab — powerful tools in the ongoing battle against infection, orphan diseases such as sickle cell anemia, chronic lung disease and a host of other maladies that plague humanity.

An aerial view of the old barracks, where staff would conduct their research.

The staff includes more than 100 researchers (M.D.s, Ph.D.s and M.D./Ph.D.s), all of whom hold professorial rank at UCLA and work in such fields as rare genetic diseases, endocrinology, cardiology and pulmonology. The institute administers 400-plus clinical trials and more than 200 industry-sponsored research projects.

“We have a large number of training programs for residents and fellows in just about every discipline,” says David Meyer ’68, Ph.D. ’72, UCLA professor emeritus and now president and CEO of the Lundquist Institute, which is academically affiliated with the David Geffen School of Medicine at UCLA and works in partnership with Harbor-UCLA Medical Center. “What physicians see in the clinic is what we study,” he adds.

But Meyer is quick to emphasize that the institute does not play a “support” role. “We are no longer just a playground for physicians working at the hospital,” he says. “We are a full-fledged institute, a not-for-profit with our own cadre of full-time researchers, as well as those county-employed physicians who come to Lundquist as part-time employees to do their research.”

Standing Up to Sickle Cell

Yutaka Niihara, clinical professor of medicine at the David Geffen School of Medicine at UCLA, says he encountered many sickle cell patients when he came to Harbor-UCLA for his fellowship in hematology/oncology. “That’s where I came to realize that it is a devastating disease,” he says — one that transforms round, free-flowing red blood cells into rigid, sticky, sickle-shaped cells that can clog blood vessels and block blood and oxygen flow.

Patients, mostly people of African descent, often experience terrible pain and illness, and treatment options have been less than optimal. Niihara started to pay special attention to sickle cell patients and they, in turn, started to seek him out, inspiring his 20-plus years in pursuit of a better treatment. The work done by Niihara and his team eventually resulted in the first new sickle cell treatment in two decades, a drug called Endari. The treatment has been called pioneering.

Approved by the FDA in 2017, Endari eases pain and blockage of small blood vessels and decreases tissue damage. It comes in powder form and can be mixed with fluid — water, juice, milk or soft drinks — and is consumed twice a day. The patients, Niihara says, “are so happy about it because they don’t have a crisis nearly as often, and when they do, the crisis is so much milder.”

Juanita Gougis was diagnosed with sickle cell anemia at the age of 2. “Growing up, I just kind of thought it was something everyone went through,” she says. It wasn’t until she was 9 or 10 that she started to notice that she was different from everyone else. She wanted to play sports, but she couldn’t keep up physically. “For most of my childhood,” she says, “I remember being in the hospital more than anything else.”

Gougis, now 30, says she heard about Endari through her mother, who met Niihara at a meeting of the Sickle Cell Disease Foundation of California. She started to participate in clinical trials of the drug as a high school student. It was, she says, “liberating.”

“My older siblings were lifeguards and grew up swimming. I wanted to follow in their footsteps, but when I was younger, I couldn’t really do that too much,” Gougis says. Cold and stress can be a trigger for sickle cell patients, but once Gougis started taking the drug, things improved. “In high school,” she continues, “I was attending practice more often because I wasn’t getting sick, and that allowed me to go to the state championships in swimming. When I told my doctors I was swimming, they were in shock.”

Professor of Medicine Yutaka Niihara catches up with his former sickle cell patient Juanita Gougis.

Today, Gougis works as an aquatics coordinator at a local YMCA. “For me, being around the water, there is a lot of joy,” she says. “It’s all good memories.”

A Haven for Start-Ups

It’s not easy to launch a new drug, and in the case of Endari, Niihara says, big pharmaceutical companies weren’t lining up to invest. Lundquist encouraged Niihara to form a company, Emmaus Life Sciences, Inc., one of 13 start-ups shepherded by the research institute.

Meyer is not shy about promoting the Lundquist start-up process. “We are agile, nimble and entrepreneurial,” he says. A small review committee at Lundquist looks at all new technologies, and if the committee decides a piece of intellectual property is valuable, “we can have a provisional patent filed within 24 hours,” Meyer explains. “If we feel there is a real cause, we move ahead as quickly as possible by either licensing this to an outside company or by starting our own start-up. We’ve been able to do start-up companies within one week.”

One of those start-ups is QT Medical, Inc., which produces a small ECG (electrocardiogram) device and cloud-based platform designed for home use. Ruey-Kang Chang M.P.H. ’93, a professor-in-residence of pediatrics at the Geffen School of Medicine and founder and CEO of QT Medical, says the concept for such a device emerged 15 years ago.

“I was seeing babies with heart disease, and it’s very hard for babies to have an ECG,” says Chang, a pediatric cardiologist. “Normally, we used a system designed for adults to make it work for babies.”

The QT Medical device, which includes an easy-to-use strip of electrodes and a data recorder that can fit in a pocket, was designed for screening newborns for long QT syndrome, a rare genetic disease that is a cause of sudden infant death syndrome (SIDS).

Ruey-Kang Chang holds a small heart monitor that he invented for babies.

At the moment, the device has been cleared for adult use with a prescription, and QT Medical is working to secure clearance to market the device for pediatric use. “This ECG platform for home or layperson use can be useful in places such as commercial flights, prison or health screening of individuals at risk for cardiac events, such as competitive athletes and firefighters,” Chang explains. “It can also be useful for patients with chronic heart disease or for patients following coronary bypass surgery.”

The list of Lundquist’s “greatest hits” is lengthy. Paul Fu invented a groundbreaking blood test for serum cholesterol level in the 1970s. The following decade, Alan Jobe developed synthetic lung surfactants for premature infants. In the ’90s, Iraj Khalkhali pioneered the use of noninvasive techniques for breast imaging, and Sherwin Isenberg ’69, M.D. ’73 created low-cost eye drops to prevent blindness in children in the developing world. More recently, Richard Casaburi pioneered exercise-based rehab strategies for COPD patients that eliminate, to a great extent, the need for inhalers, steroids and other drug-based therapies.

Some Lundquist discoveries are connected to rare diseases. Among them is a drug called Aldurazyme, an enzyme-replacement therapy for treating mucopolysaccharidosis, a rare, life-threatening disorder. Patients lack an enzyme that breaks down what Meyer calls “cellular garbage,” and they typically experience a deterioration of organ function, physical abilities and cognitive issues, starting at a very early age. Left untreated, children might live to be 10 or 12 — a prognosis delivered to Mark and Jeanne Dant when their son, Ryan, was 3 years old.

Dant and his fiancée, Silvia Ramirez.

But the Dants were tenacious. They started a foundation that helped fund research being conducted by Emil Kakkis Ph.D. ’88, M.D. ’89 at Lundquist, and Ryan Dant was part of the first clinical trial in 1998. “I was 10 weeks away from turning 10,” Dant recalls. “I’m now the person who has been on Aldurazyme the longest in the world.”

He receives weekly doses of the medicine, administered intravenously, but because the IV dose does not cross the blood-brain barrier, he also gets a quarterly intrathecal injection (into the spinal canal), which does. Dant graduated from the University of Louisville in 2017 with a degree in sports administration and completed a summer internship as an equipment manager with the San Francisco 49ers.

At 31, Dant continues to deal with the repercussions of the disease, but he is working as a transporter at a local hospital and recently became engaged to his longtime girlfriend, Silvia Ramirez. Is he optimistic? “I can’t just live in fear,” he says. “I have to appreciate what each day brings.”

Full Speed Ahead

David Meyer, president and CEO of Lundquist, relaxes in the institute’s bright new lobby.

There will be more breakthroughs. For the past two years, scientists at Lundquist have been working on a vaccine to fight Candida auris, a hospital-acquired fungal infection that’s resistant to antibiotics. And last November, the Lundquist Institute joined forces with the University of Washington School of Medicine, the Population Council and the National Institute of Child Health and Human Development to announce the first clinical trial of a male contraceptive gel.

“Lundquist is redefining itself,” Meyer says proudly. “The research institute will grow and become an essential element of the first bioscience hub in the County of Los Angeles.”

There are also high hopes for the new building, which houses a lecture hall and public spaces that will lend themselves to large gatherings and fundraisers. The institute got a huge boost in February when longtime donors Richard and Melanie Lundquist pledged $70 million in honor of their 70th birthdays. In April, the facility, formerly called the Los Angeles Biomedical Research Institute, was named after the Lundquists.

In addition, the Los Angeles County supervisors have approved the development of a 15-acre bioscience tech park on the Lundquist campus. That inspires Meyer to imagine a scenario in which a physician at the hospital sees a problem, which leads to research at Lundquist and, later, a patent filing. The next step could involve a start-up company that spends time in the Lundquist incubator and then moves into the tech park to produce a device or a treatment that is clinically tested in the hospital and approved by the FDA.

“That,” Meyer says,“is going to be monumental.”



Most Tweeted

    Range: Nov 16, 2020 – Present

      Range: Oct 17, 2020 – Present

        Range: Sep 02, 2020 – Present