LA BioMed: One Magazine Spring 2014

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Spring 2014 Donor Magazine One Translating Science Transforming Lives > One Transformational Gift One Researcher One + One Patient One Donor > > > The Power of One

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LA BioMed is a non-profit scientific research organization dedicated to saving lives not only in the greater Los Angeles metro area but throughout the world. The organization is located on the Harbor-UCLA Medical Center Campus and affiliated with the David Geffen School of Medicine at UCLA.

Transcript of LA BioMed: One Magazine Spring 2014

Page 1: LA BioMed: One Magazine Spring 2014

Spring 2014Donor Magazine One

Translating Science Transforming Lives>

One Transformational Gift One Researcher One + One Patient One Donor> > >The Power of One

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Dear Friends,

Have you given thought to what your legacy might be?

We sincerely hope that our lives can make a difference. In the case of the Kehrl family (see page 4), their legacy will continue to have an impact on LA BioMed far into our future.

Howard Kehrl had a personal mantra: “Make your moves count.” It was something he lived by each day and taught his children as well.

Sadly, Howard passed away last October, but not before creating both a financial and emotional legacy for all who knew him.

We are immensely grateful that Howard chose to remember LA BioMed in his estate plan. His gift will sustain our mission and inspire others to step forward. It also endorses

our institutional goal of saving lives through medical innovations.

As you read through this issue of One Magazine, you will find countless ways that our donors, friends and researchers are having an impact. It is humbling for LA BioMed to be the recipient of such wonderful gifts and amazing talent.

Working together, like Howard, we will be able to ‘make our moves count’ in medical research. We can thereby assure that LA BioMed touches and improves many lives now and in the future. What a magnificent legacy that would be.

With best regards,

David Meyer, PhDPresident & CEO

Inspiring Curiosity Launching LeadersPinpointing Causes Preventing DiseaseFostering Innovation Inventing the FutureProving Ideas Bringing HopeTranslating Science Transforming Lives GalaLegends EventFriends of LA BioMed Launch Luau eventFostering Innovation Inventing the FutureLooking Closer Challenging ConventionBuilding Collaboration Finding Answers

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>Translating Science Transforming Lives

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New Board Member, Grants Support LA BioMed’s Mission

LA BioMed is successful because of support from many different important partners. A strong board, committed corporate donors, dedicated investigators and passionate staff have made our organization a powerhouse of medical breakthroughs for over six decades.

I’m pleased to announce that Sarah Jensen recently joined the LA BioMed Board of Directors. She brings a wealth of community expertise. The President and founder of Jensen & Partners, she created a new model of architecture, namely offering in-house design and construction within one firm. Her expertise includes healthcare strategy, land use planning and funding. We believe Ms. Jensen will be a tremendous asset as we execute LA BioMed’s Five-Year Strategic Plan for growth.

We also depend upon corporate and philanthropic partners to help us investigate critical health challenges. Recently, LA BioMed was awarded a $900,000 grant from the Conrad N. Hilton Foundation to study the possible role of Vitamin A in slowing or halting the progression of Relapsing-Remitting Multiple Sclerosis (MS). This will be the first largescale study of its kind in the U.S.

Offering young people opportunities to become involved in scientific research is critical for us at LA BioMed. We hope this inspires them to consider science as a life-long career. That’s why we were excited to receive a $67,000 grant from the Harbor Community Benefits Foundation for our summer fellowship program. The grant helps to underwrite 20 high school students from Wilmington and San Pedro who plan to investigate the effects of port-related respiratory issues in the Harbor community.

Thanks to all who offer their expertise and support to LA BioMed. We continue to advance our mission because of this unreserved generosity.

Sincerely,

Steven A. NissenChair, Board of Directors

Translating Science Transforming Lives>

LA BioMed BOARD OF DIRECTORS

Steven A. Nissen, Esq. (Chair)Ronald S. Swerdloff, MD (Vice Chair)Eric S. Daar, MD (Secretary)James M. Brown (Treasurer)

Webb W. Castor, PhDDorothy Avila CourtneyNoah A. Craft, MD, PhD, DTM&HPatricia I. Dickson, MDScott G. Filler, MDDelvecchio S. Finley, MPP FACHEAllan M. FrewRichard J. Glassock, MD, MACPSarah Jensen, AIA, LEED, APAdam J. Jonas, MDRichard K. LearnedIra M. Lesser, MDMelanie LundquistDavid I. Meyer, PhDAlex PopaMitchel Sayare, PhDSussan Sharifian, Esq.H. Michael Smith, AIA LEED APAndrew J. SobelBruce Stabile, MDWilliam W. Stringer, MDTimothy Van Natta, MD, FACSSamuel Eric Wilson, MD

>Inspiring Curiosity Launching Leaders

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Battling thyroid cancer since 2007, she had a number of treatments that were not effective. “A cancer diagnosis is like having a gun held to your head. Until you get good news, you can only focus on survival, “ she says.

“A cancer diagnosis is like having a gun held to your head. Until you get good news, you can only focus on survival.” Monica Berndt

Typically, thyroid cancer has a good outcome for many. However, Monica had a persistent type that did not respond well to treatment.

Her first surgery and radiation treatments appeared to go well until six months later. Another surgery followed. Determined to triumph, she fought the cancer with all the weapons at her disposal including conventional and alternative medicine.

For 18 months, the outcome looked good until she experienced increased hoarseness and then had trouble breathing. Tests revealed the cancer had come back. Or, as she says, “It may have never left.” This time the recommended surgery was drastic – a laryngectomy. “This meant the permanent loss of my voice,” she says. It was a defeat of epic proportions for a woman who had loved to sing.

Still, she had the surgery and with typical matter-of-fact courage, learned to adjust to a new normal. Then the unthinkable happened again. She felt a pea-sized lump on her neck. PET and CT scans revealed the cancer had come back in two spots. They were in difficult to reach locations including one at the base of the tongue.

“When my surgeon explained to me that he could address the situation by removing part of my tongue, I drew the line,” she says. She knew this would seriously affect her quality of life.

She was torn about treatment. “I love life and am not beyond work and career. I have a loving family and there are children in my circle I’d like to see grow up.”

However, Monica was not ready to “seclude herself in a corner and wait for the grim reaper,” she notes with characteristic dry humor. She drew the line at this surgery and began intensively researching her options. When her oncologist mentioned a clinical drug trial for thyroid cancer patients at LA BioMed, she felt a renewed sense of hope.

She contacted Dr. Andrew Giranoukakis, the LA BioMed researcher conducting the clinical trials, and was accepted into the study for Lenvatinib, a thyrosine kinase inhibitor drug. Though cautiously excited, she remained pragmatic. “There were no guarantees,” she says. She began treatment with ‘Dr. G’ as she calls him in November of 2011.

The results have exceeded her wildest hopes. Within two months, the nodules were noticeable smaller. A follow-up CT scan showed they shrank 20-30 percent. Today, 28 months later, the treatment brought about a reduction of over 90 percent in the tumors. She can no longer feel them. She credits Dr. G and his staff at LA BioMed for their friendship and camaraderie as well as for the care of patients enrolled in the innovative drug regimen. “This drug and other new advancements in cancer treatment have changed my outlook,” she says.

“For patients like us, with very few options, these opportunities represent hope and life.” Monica Berndt

When Monica reflects on the past seven years, one thing stands out. “I don’t have the words to express the positive impact that clinical trials have. They are keeping people alive. I thank everyone involved with this from the bottom of my heart.” She observes that cancer must be fought by any means and clinical trials may be the last opportunity for many.

Monica believes the importance of clinical trials cannot be overstated. “For patients like us, with very few options, these opportunities represent hope and life. We know that through these trials, doors of hope and life will open for many others who will benefit from the results.”

Monica Berndt is a woman who cherishes her future, thanks to LA BioMed, its dedicated researchers and caring staff.

>Pinpointing Causes Preventing Disease

In 2011, Monica Berndt’s cancer had returned and was growing larger. In her words, “I was down to very few options” when she enrolled in a clinical drug trial through Harbor-UCLA Medical Center.

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Mary Kehrl and her daughter Kathy Blenko.

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“Make your moves count.”That was Howard Kehrl’s lifelong philosophy. And the Kehrl family, taking that to heart, made a move that will positively impact LA BioMed for generations.

Howard’s LA BioMed LegacyHoward passed away on October 1, 2013. During his life, he made an impact both personally and professionally. The effect of his estate bequest on LA BioMed’s researchers, clinicians and patients will resonate far into the future.

Early events shaped Howard’s life philosophy and philanthropy. He and his sister were abandoned by their father, leaving his mother to raise them. Money was in short supply but there was always love in the home. Wanting to help, Howard began working at age six during the Great Depression to support the family.He worked many jobs during his growing up years but managed time for his passion for cars – and a little drag racing. These experiences became the foundation of Howard’s life – love of family and hard work.

Love of family and hard work were the foundation of Howard Kehrl’s life.

Early Years and EducationHoward’s love of cars and mechanical things would lead him to an illustrious career as an engineer at General Motors where he eventually retired as Vice Chairman. Though he worked tirelessly to support his sister and mother, Howard was also an excellent student and earned a scholarship to Wayne State University. It was here he met the love of his life, Mary Katherine Maloney. The pair were wed for 67 years and cherished four children together.

Howard left Wayne State to enlist in the Navy during WWII. After the war ended, he resumed his education and earned a bachelor’s degree and a master’s degree.

Later in his career, he received an additional master’s degree in Industrial Management.

Mary also valued education. She holds a bachelor’s degree in social work from Wayne State University and completed her thesis before the couple’s second son was born, earning a master’s degree. Each of their four children also holds advanced degrees.

In fact, the couple so strongly believed in the power of philanthropy, they endowed a full scholarship for undergraduate science and engineering students to ‘give back’ to Wayne State University.

Career SuccessHoward began working at General Motors in the research laboratories in 1948. He worked in the Cadillac, Oldsmobile and Chevrolet divisions, eventually being named as Chief Engineer and General Manager of Oldsmobile. Rising quickly, he assumed the position of Executive Vice President in charge of General Motors technical and planning staffs and was responsible for the company’s international operations. His final position before retiring from General Motors was Vice Chairman.

Mary was an integral part of Howard’s career success. A counterpoint to his hard-driving professionalism, she was adept at the softer side of human nature. Mary and Howard often talked about work situations

“Make Your Moves Count”

Howard Kehrl’s Planned Gift Legacy

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and she enjoyed elegantly entertaining his General Motors colleagues. When Howard’s job required frequent overseas travel, Mary accompanied him.

Family is a PriorityFor all his career success, Howard and Mary were both deeply committed to their family. One of his sons described Howard as a “serious man with a big, warm heart.” In contrast, Mary was gregarious and gifted in human relations, as her daughter Kathy observes. Together, the pair had an epic love affair that lasted until the day Howard died.

As Howard’s work responsibilities became more pressing, he worked hard to carve out family time. He had grown up without a father and had no role model. Still, he was determined to be present and involved in his four children’s lives.

The family credits Howard with sharing many critical values – the importance of hard work, perseverance, loyalty and the worth of education.

The family was close, though as teens Howard’s children were not always appreciative of their father’s focus on chores and tasks. However, looking back, each credits him with sharing many critical values – the importance of hard work, perseverance, loyalty and the worth of education.

So that Howard could set aside work, the family had a cottage on Walloon Lake in Michigan that they regularly enjoyed. So fiercely did

he guard his family time that he refused to have a telephone installed, making a clear delineation between work and family.

Life is an AdventureWith Mary’s support, Howard filled his children’s lives with carefully planned and executed adventures, regularly documenting fishing, boating, water skiing, snow skiing and cross country trips with his camera. To teach his children to ski, he used the principles of physics. Howard wanted the children to have fun but also learn something, particularly about how things worked. As son David laughingly points out, “He spoke repeatedly of scientific principles, in hopes they would eventually take root in our young brains.”

Later YearsAfter retiring, Mary and Howard purchased a cabin in Colorado. Shortly after that, they began planning and built their dream house on the side of a mountain with breathtaking views of the Rocky Mountains. The couple’s greatest pleasure was to entertain their children and their growing families.

Eventually, the couple re-located to California to be closer to their daughter Kathy and her family. Each day, Howard and Mary started the morning by having coffee together. As often as she could, Kathy joined them.

When Howard was diagnosed with colon cancer, Kathy and her mom became a team to take care of him. Even as he weakened, he continued to cherish his family.

Giving BackAs was his nature, Howard was a planner though often, Mary took the lead in the family’s philanthropy. Over the years, they were proud of the scholarship they endowed at Wayne State University and treasured the frequent thank you notes from the student recipients. They also gave to organizations to alleviate homelessness, supported international medical causes and were patrons of a local theater company because Mary valued arts in the community.

Planned Gift to LA BioMedIt was Mary who first became interested in LA BioMed. A family friend and neighbor, Paula Moore, had a long association with the organization and knew the Kehrls were interested in innovations and medical problem solving.

As the Kehrls learned more about LA BioMed, they felt a kinship with the organization’s mission. They liked that LA BioMed was located in their community and both found the medical research aspect to be intriguing. Two of their sons are physicians involved in medical research and a grandson recently earned a PhD in pharmacology.

By supporting LA BioMed, they felt they could make a difference in a broad number of important medical fields, and nurture their family focus on education, problem solving and medicine. They wanted to make a larger impact to have a positive long-term effect, so they decided to make a planned gift through their estate.

After Howard’s death, LA BioMed was notified about the gift details. The organization was deeply grateful. “Because of people like the Kehrl family, LA BioMed continues to do ground breaking research to address critical health problems not only in our community but around the world,” says Rodney Franks, LA BioMed’s Vice President of Development.

“It’s been therapeutic to know that in our grief, my dad lives on through helping others...” Kathy Blenko, describing the family’s support for Howard’s planned gift.

One man. One family. And one way to assure a valuable life philosophy becomes a legacy for all those who will be touched by this gift.

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LA BioMed has established a Legacy Circle endowment to secure the future. The Legacy Circle was created to encourage bequests from individuals who profoundly believe that biomedical research will provide solutions to our most pressing health challenges.

By planning ahead to provide income for the organization, you create a significant impact for generations to come. You can leave a specific amount of money, assets such as stocks, bonds, works of art, real estate or simply a remainder percentage of your estate.

To join the Society, you simply include LA BioMed in your will or estate plan. In this way, you showcase your dedication to the future, assuring the organization’s financial stability continues far beyond our lifetimes. If you would prefer to make a monetary gift now, we can place it in this endowment so it begins to work for LA BioMed immediately.

Your gift honors the substantial scientific contributions made by our researchers and investigators. It also promotes excellence in research, clinical care and medical education so these innovations can continue.

A bequest assures that LA BioMed continues to transform lives. Your legacy will inspire others while making an impact now and for countless new generations.

The LA BioMed Development Department is a valuable resource to anyone that is considering

a planned gift to LA BioMed. Our department would be happy to provide the steps and turn-key templates you need to effectively, efficiently and confidently put together your planned gift. You will be pleasantly surprised at how easy it is.

>Proving Ideas Bringing Hope

LA BioMed Legacy CircleAs a friend of LA BioMed, you can make a significant difference when you include a planned gift in your estate plan.

Rodney D. FranksVice President of Development

Create a legacy for yourself or your company. To learn more, contact Rodney Franks, Vice President, LA BioMed at 310.222.8284 or [email protected]

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The institute also honored one of the region’s leading businessmen and philanthropists, Ralph Scriba, with its Award for Community Service, and one of its most distinguished researchers, Kouichi R. “Corky” Tanaka, MD, with its Award for Leadership Excellence.

“We are privileged to have the opportunity to honor such distinguished leaders in medicine, research and philanthropy at our gala,” said Rodney D. Franks, LA BioMed vice president for development. “It is an honor to recognize Dr. Soon-Shiong for devoting his vision, his voice and his resources to improving our nation’s healthcare system. Mr. Scriba is an accomplished businessman who deserves every accolade for giving back so much to the community, and Dr. Tanaka is renowned for nearly a half a century of leadership in medicine, research and education.”

“Each of our honorees are true visionaries whose myriad contributions are helping to move healthcare forward and ensure that it serves those most in need,” said David I. Meyer, PhD, LA BioMed president and CEO. “They are inspirations to all of us who work in the field of research and strive every day to improve the lives of patients across the globe. It is fitting that the “Translating Science and Transforming Lives” gala would honor three leaders who have transformed so many lives.”

LA County Supervisor Mark Ridley-Thomas and Dr. Patrick Soon-Shiong

2013 Translating Science > Transforming Lives Gala

LA BioMed honored Dr. Patrick Soon-Shiong, one of the preeminent scientific and medical minds in the world, with the Award for Advancing Innovations at its “Translating Science and Transforming Lives” gala on Thursday, December 5 at the Beverly Wilshire Hotel in Beverly Hills.

NBCLA Anchor/ Reporter, Whit Johnson,

the evening’s Emcee, LA BioMed Board

Chairman, Steven Nissen, Honoree Kouichi Tanaka,

MD, and LA BioMed President and CEO,

David Meyer, PhD

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Michele Chan and Dr. Patrick Soon-Shiong Lynn Alvarez and Steve Nissen

Board Chair, Steve Nissen and Board Member, Dorothy Courtney Honoree Kouichi Tanaka, MD and Grace Tanaka

Mitchel Sayare, PhD, Christina Sayare, and David Meyer, PhD Honoree Ralph Scriba

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LA BioMed Investigators: Anita Nelson, MD and Adam Jonas, MD

2014 Legends Honorees: Joel Kopple, MD and Robert Hockberger, MD

LA BioMed Honors LegendsLA BioMed Celebrated the Accomplishments of Two “Legends” at the May 1 Event to Honoring Drs. Joel D. Kopple and Robert Hockberger Continuing a tradition of recognizing its most outstanding physician- researchers, LA BioMed celebrated the many accomplishments of Drs. Joel D. Kopple and Robert S. Hockberger on May 1 at Legends 2014 at the Torrance Marriott South Bay.

“We are so pleased to recognize both of these distinguished physician-researchers as LA BioMed Legends because they have done so much to advance the pace of discovery and the practice of medicine,” said David I. Meyer, PhD, LA BioMed president and CEO. “Dr. Kopple has received numerous awards and is known throughout the medical community for his groundbreaking research into kidney disease, his patient care and the training he’s provided to medical students throughout his long career. Dr. Hockberger is a legend in the field of emergency medicine for the many contributions he’s made to improving emergency care and to training the next generation of emergency room physicians.”

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2004 Legend, John Michael Criley, MD, Jasminka Criley, MD and Stuart Criley

Teens for LA BioMed members

Jacob Rajfer, MD, Donald Inadomi, MD, Madelynn Kopple, Joel Kopple, MD, and Jay Vadgama, MD

Representatives from Event Sponsors Konica Minolta and Novitex

David Meyer, PhD, Harlyne Norris, Steve Nissen and Webb Castor, PhD David Meyer, PhD and Richard Lundquist

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Jamie McKinnell, MD and Rodney Franks Nani Edgar and six-time Grammy Winner Daniel Ho

Friends of LA BioMed Launch Luau event

The Friends of LA BioMed hosted a Luau themed launch event with six-time Grammy Award Winner, Daniel Ho on Sunday, May 18.

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Diana Honeycutt, Sharon Guthrie, Rodney Franks, Sue Christie and Donna Lee

Liz and Hal SnyderJim and Nancy Welsh, Trudy Park, Mary Jane Shoenheider

Sue and Fred ChristieScott Donnelly and Wade Nishimoto, MD

Rodney Franks, Board Members Joan Jones and Rick Learned

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Based on a survey of parents of children with congenital heart disease, physicians delivering the diagnosis need to do a better job of showing compassion, ensuring parents understand all their options and providing easily understandable information, according to a new study published in the February edition of the journal, Pediatric Cardiology.

Researchers at LA BioMed worked with the California Heart Connection, a nonprofit organization of parents of children with congenital heart disease, to conduct an online survey of 841 parents of children who were either diagnosed before birth or shortly afterward with congenital heart disease. The researchers said physicians remain the primary source of information for parents at diagnosis. So, they analyzed the survey’s findings to offer suggestions for how physicians and other health care providers can most effectively tell parents their child has a serious and potentially life-threatening medical condition.

“Parents confronted with a diagnosis of congenital heart disease face very difficult choices – from selecting a treatment to determining whether to continue a pregnancy, if they receive the diagnosis prenatally,” said Ruey-Kang Chang, MD, an LA BioMed researcher and the corresponding author of the study. “All of these decisions depend on the information they are given at the time of diagnosis and how well they can understand and retain the information at such a difficult time.”

Upon learning of their children’s diagnosis, most of the parents surveyed said they received no information on support groups, Internet resources or success rates at other hospitals. Two-thirds of the parents were told their children’s heart conditions were “rare.” More than half of them believed that meant fewer than one in a million infants had the condition or there were few people alive with the defect. In reality, congenital heart disease occurs in about eight in 1,000 births in the United States, with approximately one-third of those cases requiring invasive treatment during the first year of life.

“Parents confronted with a diagnosis of congenital heart disease face very difficult choices – from selecting a treatment to determining whether to continue a pregnancy, if they receive the diagnosis prenatally” Ruey-Kang Chang, MD

Of those surveyed, 13% reported feeling pressured to terminate the pregnancy by their pediatric cardiologist, and 15% felt their physician showed a lack of compassion. More than a fourth of the respondents said they sought a second opinion, and 71% of them chose the second physician for long-term follow-up care. The two most commonly cited stress factors were “uncertainty” about their child’s future (87%) and “not knowing about survivors with this defect” (52%).

“The information parents receive at diagnosis and the manner in which it is conveyed often determines their assessment of their children’s chances for survival and may shape the parents’ decisions,” said Dr. Chang. “To ensure the parents have the information they need to make these critical decisions about their children’s health, physicians should provide context for the parents, including information on success rates at different hospitals, Internet resources and referrals to support networks. Physicians should also be aware of how their demeanor and the words they use will be interpreted by the parents.”

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Parents Receiving Heart Disease Diagnosis for Infants Need Better Information. LA BioMed Survey Finds More Compassion and Resources Required to Help Guide Treatment Choices

Ruey-Kang Chang, MD

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>Looking Closer Challenging Convention

Children Exposed to Methamphetamine before Birth Have Increased Cognitive Problems – Long-term Study Highlights the Need for Early Intervention

In the only long-term, National Institutes of Health-funded study of prenatal methamphetamine exposure and child outcome, researchers found youngsters exposed to the potent illegal drug before birth had increased cognitive problems at age 7.5 years, highlighting the need for early intervention to improve academic outcomes and reduce the potential for negative behaviors.

The researchers studied 151 children exposed to methamphetamine before birth and 147 who were not exposed to the drug. They found the children with prenatal methamphetamine exposure were 2.8 times more likely

to have cognitive problem scores than children who were not exposed to the drug in a test often used for measuring cognitive skills, the Connors’ Parents Rating Scale.

“These problems include learning slower than their classmates, having difficulty organizing their work and completing tasks and struggling to stay focused on their work,” said Lynne M. Smith, MD, a lead researcher at LA BioMed and corresponding author of the study. “All of these difficulties can lead to educational deficits for these children and potentially negative behavior as they find they cannot keep up with their classmates.”

Methamphetamine use among women of reproductive age is a continuing concern, with 5% of pregnant women aged 15-44 reporting current illicit drug use. Methamphetamine usage during pregnancy can cause a restriction of nutrients and oxygen to the developing fetus, as well as potential long-term problems because the drug can cross the placenta and enter the fetus’s bloodstream.

Previous research in Sweden found evidence of lower IQ scores, decreased school performance and aggressive behavior among children with prenatal methamphetamine exposure. The study tracked the children through age 15, but it didn’t compare them to children who had no prenatal methamphetamine exposure.

Researchers at LA BioMed and in Iowa, Oklahoma and Hawaii – all places where methamphetamine usage is prevalent – have been tracking children who were not exposed to the drug and children with prenatal methamphetamine exposure since 2002, as part of the Infant Development, Environment and Lifestyle (IDEAL) Study. This study, which is the only prospective, longitudinal National Institutes of Health study of prenatal methamphetamine exposure and child outcome, was conducted under the auspices of Principal Investigator Barry M. Lester, PhD, at Women & Infants Hospital of Rhode Island.

“By identifying deficits early in the child’s life, we can intervene sooner and help them overcome these deficits to help them have greater success in school and in life” Lynne M. Smith, MD

“Through the IDEAL Study, we are able to track these children and better understand the long-term effects of prenatal methamphetamine exposure.”

Lynne M. Smith, MD

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Matthew Budoff, MD

Calcium Score Predicts Future Heart Disease Among Adults with Little or No Risk Factors “LA BioMed Research studied nearly 5,600 Patients over 20 years”With growing evidence that a measurement of the buildup of calcium in coronary arteries can predict heart disease risk, LA BioMed researchers found that the process of “calcium scoring” was also accurate in predicting the chances of dying of heart disease among adults with little or no known risk of heart disease.

Previous studies had found that calcium scores were effective in predicting heart disease among adults with known heart disease risk factors, such as hypertension, diabetes, dyslipidemia, current smoking or a family history of heart disease. The study conducted by LA BioMed researchers examined 5,593 adults with no known heart disease risk or with minimal risk of heart disease, who had undergone coronary artery calcium screening by non-contrast cardiac computed tomography from 1991-2011.

Normally, the coronary arteries don’t contain calcium. A buildup of calcium can narrow the arteries to the heart and lead to a heart attack. The screening process results in a calcium score indicating the amount of calcium in the plaque lining the walls of the coronary arteries.

Among the adults in the study, even those with low coronary artery calcium scores of 1-99 were 50% more likely to die of heart

disease than adults with a calcium score of zero. Adults with moderate scores of 100-399 were 80% more likely to die from heart disease than those with a score of zero, and those with scores of 400 or more were three times more likely to die from heart disease, when compared to adults with no calcified plaque buildup, or a score of zero.

“This long-term study builds on previous research conducted at LA BioMed and other institutions that have proven the effectiveness of coronary artery calcium screening in predicting heart disease risks,” said Matthew J. Budoff, MD, one of the LA BioMed researchers who conducted the study. “Normally, calcium scoring is only recommended for patients with known heart disease risks. These findings suggest that calcium scoring can be an effective tool for assessing heart disease risks in adults with no known risk factors so that they can make the lifestyle and other changes that can help them avoid heart disease in the future.”

Normally, the coronary arteries don’t contain calcium. A build-up of calcium can narrow the arteries to the heart and lead to a heart attack.

>Looking Closer Challenging Convention

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>Looking Closer Challenging Convention

New LA BioMed team takes on glaucoma in African-Americans A team of genomics researchers recently embarked on the largest-ever study of the genetics responsible for glaucoma among African-Americans.

The team hopes its research will pave the way for medical care so precise that doctors will, one day, be able to know a person’s disease risks and successful treatment options even before an office visit. Such personalized medicine is the goal of genomics — the study of someone’s entire genetic history encoded on 23 pairs of chromosomes encased inside the nucleus of human cells.

The team at the Institute for Translational Genomics and Population Sciences has a specialized focus. Its researchers want to understand how predominant diseases among blacks, Asians and Latinos occur. Genetic testing of minority groups is still in its infancy, said the team’s director, Dr. Jerome Rotter.

Glaucoma, most devastating among African-Americans, is believed to be caused by pressure on the optic nerve that can result in decreased vision and, potentially, blindness. But once researchers identify the genetic abnormalities that cause glaucoma, Rotter said, the existing understanding of how the disease progresses could change dramatically.

“Understanding the true cause of the disease is the goal,” Rotter said. “The power of what we do is to get the real cause. Genetics hits you in the face with the cause.”

Every human has 3 billion DNA base pairs and, of those, there are about 10 million instances where one person differs from another. Rotter’s team will analyze 2,500 glaucoma patients in San Diego, the Los Angeles Harbor Area and at two other sites in other states. They will detail 2.5 million genetic markers within each patient over a five-year period. The data hopefully will result in identification of the genes that cause glaucoma, Rotter said.

Pinpointing the problematic DNA is only the beginning, however.

All disease is caused by a combination of genetics and a person’s interaction with his or her environment, Rotter

said. Without knowing a person’s genotype, doctors are dependent only on family medical histories and his or her physical appearance.

“The mutation in the gene can tell you someone’s pre- disposition, but to determine how drugs will react with that person is more complicated,” Rotter said. “We will be able to evaluate at-risk patients earlier, but new therapies will have to be developed.”

The goal of personalized medicine is often described as “providing the right patient with the right drug and the right dose at the right time.”

Such ideas were the stuff of science-fiction stories until the Human Genome Project outlined the complete genetic blueprint of one human being in 2003. Subsequent genome studies such as the International HapMap (haplotype map) project, and technological innovation from companies like Illumina, have resulted in making genotyping much more affordable. This affordability is what made it possible to launch the glaucoma study, Rotter said.

“The goal of our therapy is to know how to treat a patient before they even walk in the door.”

“Ten years ago, I studied one gene at a time and it cost me $2 or so per gene,” Rotter said, adding that the team is looking at 2.5 million genes per person in this study. “Rather than $5 million per person, I can do it now for $250 per person because of new technology and knowledge.”

As it is, medical treatment is often predicated on trial-and-error processes in which doctors test drugs on patients to see which works the best. Rotter calls this system a “tragedy.”

“This exposes them to side effects or drugs with no therapeutic effects and we need to do better than that,” he said.

Jerome Rotter, MD

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Ashraf S. Ibrahim, MD

New Investigational Drug Holds Promise For Combatting Deadly Mucormycosis Infections – LA BioMed Team says New Treatments “Urgently Needed”

With very few treatment options available to fight deadly mucormycosis infections, a new LA BioMed study holds hope for adding to the arsenal of therapies physicians have to combat an increasingly common infection afflicting people with weakened immune systems.

An LA BioMed research team reported in an online, ahead-of-print study in the Antimicrobial Agents and Chemotherapy journal that the investigational drug isavuconazole was as effective in reducing mucormycosis infections in disease models as the most widely used treatment currently on the market, high-dose liposomal amphotericin

B. Isavuconazole is an investigational once-daily intravenous and oral broad-spectrum antifungal for the potential treatment of severe invasive and life-threatening fungal infections. It is currently in phase 3 of clinical development.

“Expanding the options for fighting these deadly infections is especially important for patients who can’t tolerate current treatments or whose infections may not respond to the antifungals on the market today,” said Ashraf S. Ibrahim, PhD, an LA BioMed lead researcher and the corresponding author for the study. “With such a limited number of treatments available, developing new therapies is critical to increasing the number of people who survive these extremely lethal infections.”

Patients with weakened immune systems, hyper-glycemia, acidosis (diabetic ketoacidosis or other forms of acidosis), malnutrition, or trauma patients are at increased risk of mucormycosis infection. Despite the current treatments and surgeries

available to treat mucormycosis, more than half of patients with the infections die.

“Clearly, new strategies for preventing and treating mucormycosis are urgently needed,” the researchers concluded.

They examined the most common form of this deadly infection, mucormycosis due to Rhizopus delemar, and concluded that their results support the further development of isavuconazole.

Clearly, new strategies for preventing and treating mucormycosis are urgently needed.

>Looking Closer Challenging Convention

19Translating Science Transforming Lives> LA BioMed One

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20 LA BioMed One One Researcher

After conducting a systematic literature review focusing on the management of women experiencing at least three UTIs per year, a team led by Loren G. Miller, MD, MPH developed a model of recurrent UTI for each management strategy for which at least two adequate trials had been published.

As the researchers described in Clinical Infectious Diseases (58[2]:147-160), they simulated a cohort that experienced three UTIs per year and a secondary cohort that experienced eight UTIs per year to learn more about treatment efficacy, patient and payer cost, and health-related quality of life.

Five strategies for the prevention and management of recurrent UTIs were analyzed: daily antibiotic (nitrofurantoin) prophylaxis, daily estrogen prophylaxis, daily cranberry prophylaxis, acupuncture prophylaxis, and symptomatic self-treatment. The nitrofurantoin option proved to be the most effective as well as the most expensive to the payer in the model of three UTIs per year: Antibiotic therapy reduced the UTI rate to 0.4 per year, at an annual cost to the payer of $821.

The acupuncture recurrence rate was 0.7 per year. The use of cranberry pills brought the recurrence rate to 1.1 per year, as did estrogen therapy. Symptomatic self-treatment did not

reduce recurrence rates but was associated with the highest quality of life of all management strategies (all regimens improved this measure).

Whereas insurance providers saved money on all five approaches, all but self-diagnosis and treatment increased out-of-pocket costs for patients, from a mean of $140 per year for antibiotics to a mean of $946 per year for acupuncture, the latter of which was often paid wholly or mostly by the patient. (In contrast, insurance often covered all or part of the costs of antibiotic therapy.)

“This study provides comparisons of prevention approaches so women can knowledgeably discuss with their providers the most appropriate way for them to manage recurrent urinary tract infections,” Dr. Miller affirmed. “Because patient preferences are very diverse, we laid out the benefits and costs of each approach to help the patient and provider choose an approach that best suits the patient’s lifestyle and preferences.”

Loren G. Miller, MD, MPH

Antibiotics Best, But Not Only Way to Treat Recurrent UTIsDaily antibiotic use came out on top when compared with several other strategies for managing recurrent urinary tract infections (UTIs), but daily cranberry pills, daily estrogen therapy, and acupuncture also had good showings of cost-effectiveness.

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21LA BioMed OneTranslating Science Transforming Lives>

>Looking Closer Challenging Convention

LA BioMed Researchers Report on Promising New Therapy For Devastating Genetic Disorder. Investigational Drug Tested for Treatment of Sanfilippo B Syndrome

Patricia Dickson, MD

A promising new therapy has – for the first time – reduced damage to the brain that can be caused by Sanfilippo B (MPS IIIB), a rare and devastating genetic disease, LA BioMed presented at the Lysosomal Disease Network’s 10th Annual WORLD Symposium™.

Sanfilippo B syndrome is a lysosomal storage disease belonging to the group of mucopolysaccharidoses (MPS) that is characterized by severe and rapid intellectual deterioration. LA BioMed pioneered the research that led to the first enzyme replacement therapy for MPS I, Aldurazyme©, which has saved hundreds of lives in the nearly 11 years since it was approved by the Food and Drug Administration.

LA BioMed’s MPS Research Laboratory reported the results of its latest study for treating Sanfilippo B syndrome in a disease model that employed recombinant human “NAGLU-IGF2” (a novel fusion protein of alpha-N-acetyglucosaminidase, or NAGLU, coupled to a peptide derived from the insulin-like growth factor, IGF2). The fusion protein was produced by BioMarin Pharmaceutical Inc.

LA BioMed researcher Shih-Hsin Kan reported that the laboratory’s tests found NAGLU-IGF2 is taken up by neurons in the disease model, which then reduces brain damage by reversing heparan sulfate storage – one of the causes of damage to the brain in patients with Sanfilippo B syndrome. The researchers concluded that NAGLU-IGF2 can be the basis for enzyme replacement therapy for Sanfilippo B syndrome.

“This is an exciting new development that needs to be examined further to determine its effectiveness in patients with Sanfilippo B syndrome who currently have no therapies available to reduce the damage to their brains,” said Patricia Dickson, MD, director of the MPS Research Laboratory at LA BioMed. “We look forward to continuing our partnership with BioMarin in developing therapies for MPS.

BioMarin announced that it has developed BMN 250, a manufactured form of NAGLU-IGF2, for the treatment of Sanfilippo B syndrome and expects to initiate clinical studies with BMN 250 in mid-2015.

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22 LA BioMed One One Researcher

>Building Collaboration Finding Answers

LA BioMed Research In The News

Richard J. Glassock, MD

The Medal of Excellence is the organization’s “most prestigious award, honoring a renal physician for their extraordinary work in advancing patient care, ensuring the quality of patient care, being a strong patient advocate and being a mentor to fellow colleagues to ensure the continuation of these important attributes.”

Dr. Glassock is an internationally recognized expert in the field of glomerular diseases and clinical nephrology. He has published more than 450 original papers, books, book chapters, editorials and reviews. He has lectured in more than 95 countries, has been a visiting professor at more than 125 academic institutions, and has trained more than 50 nephrologists.

“Richard’s entire career as a physician and teacher has been dedicated to advancing healthcare. At LA BioMed, we feel very fortunate to benefit from his dedication and his knowledge in helping to guide our research endeavors,” said David I. Meyer, PhD, LA BioMed president and CEO.

LA BioMed board member Dr. Richard Glassock honoredRichard J. Glassock, MD, received the 2014 Medal of Excellence from the American Association of Kidney Patients at a March conference and will be honored throughout the year at a series of events.

Charles Grob, MD

Seeking new therapies for the treatment of social anxiety in autistic adults, LA BioMed researchers are initiating a novel study into the safety and effectiveness of MDMA-assisted therapy.

The study is the latest in an expanding program of research into the therapeutic use of MDMA funded by the nonprofit Multidisciplinary Association for Psychedelic Studies (MAPS). The research seeks to examine effective treatments for adults on the autism spectrum, who often face social adaptability challenges and greater anxiety, depression and victimization than typically developing adults.

Conventional prescription medication that may help other adults is often ineffective in autistic adults. Moreover, difficulties in establishing a rapport with a therapist can interfere with conventional psychotherapy.“We know we need new supportive treatments, and we have anecdotal evidence that autistic adults who had experimented with MDMA experienced a reduction in anxiety and an increased confidence in their abilities to interact socially,” said Charles Grob, MD, LA BioMed’s lead researcher for the study.

LA BioMed Launches Study of MDMA-Assisted Therapy For Social Anxiety in Autistic Adults

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23LA BioMed OneTranslating Science Transforming Lives>

International study finds lower-dose IUDs are safe and effective

In a finding that could expand the use of one of the most effective forms of birth control, two intrauterine contraceptive systems that had lower doses of the contraceptive hormone, levonorgestrel, were found to be safe and effective in preventing pregnancies, according to an international study that included researchers at LA BioMed.

“By demonstrating the safety and efficacy of low-dose intra-uterine contraceptive devices, this study will help expand the contraceptive options available to women who have not had children before,” said Anita Nelson, MD, an LA BioMed lead researcher and corresponding author of the study. “Additionally, a lower dose system may be preferable to women who are seeking to reduce their exposure to synthetic hormones.”

Antibiotic Resistance Is Now Rife across the Entire Globe

Dangerous antibiotic-resistant bacteria and other pathogens have now emerged in every part of the world and threaten to roll back a century of medical advances. That’s the message from the World Health Organization in its first global report on this growing problem.

Antibiotic resistance is putting patients in peril in both developing and developed countries, as bacteria responsible for an array of dangerous infections evolve resistance to the drugs that once vanquished them.

“It is terrifying in scope. This is a massive public health problem that is just starting to bubble to the surface,” says Brad Spellberg, LA BioMed researcher.

Breakthrough Shows Unique Garlic May Reverse Heart Disease

Although previous studies have focused on garlic’s effect on cholesterol, this research done by Dr. Matthew Budoff, LA BioMed researcher, is the first to look at its direct effect on the heart’s arteries.

Budoff told NBC4’s Dr. Bruce Hensel that the reserchers wanted “to see if there is actually benefit to the arteries themselves; if there’s heart disease benefit by taking aged garlic extract.”

“The results showed that aged garlic extract actually causes some regression, a little bit of less plaque,” Budhoff said.

Matthew Budoff, MD

In the News

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>Building Collaboration Finding Answers

LA BioMed Research In The News

24 LA BioMed One One Researcher

Forty-two of LA BioMed’s physician-researchers will be ranked among America’s Top Doctors when it issues its 2014 publication later this year or they were listed among the Southern California Super Doctors for 2014 in the January edition of Los Angeles Magazine.

Among the LA BioMed physician-researchers named to the two lists are specialists in cardiology, endocrinology, neurology, internal medicine, nephrology, oncology, obstetrics/gynecology, ophthalmology, surgery, pediatrics, psychiatry, pulmonary medicine, immunology and many other specialties.

LA BioMed Physician-Researchers Recognized As the Nation’s and the Region’s Best Doctors. Honorees Demonstrate Dedication to Advancing Innovation and Improving Medical Care.Forty-two of LA BioMed’s physician-researchers were ranked among America’s Top Doctors or they were listed among the Southern California Super Doctors for 2014 in the January edition of Los Angeles Magazine.

Following is a list of the physician-researchers who were honored. (A * means the physician ranks among Southern California Super Doctors list published in 2014. A + means the physician will be ranked among America’s Top Doctors when the 13th edition is issued later this year.)

Sharon G. Adler, MD *Thomas L. Anderson, Jr., MD *Arnold S. Bayer, MD +Carol D. Berkowitz, MD+Matthew J. Budoff, MD *+Richard Casaburi, PhD, MD *Rowan T. Chlebowski, MD +Noah Craft, MD, PhD*Eric S. Daar, MD *+Christian M. de Virgilio, MD *Carlos E. Donayre, MD*John E. Edwards, Jr., MD *+Viktor E. Eysselein, MD* William J. French, MD *+Charles S. Grob, MD*

Kenneth R. Huff, MD*Eli Ipp, MD *Sherwin J. Isenberg, MD *+Adam J. Jonas, MD* +Margaret A. Keller, MD *Iraj E. Khalkhali, MD*Siri Linda Kjos, MD*Stanley R. Klein, MD*Wai-Nang Paul Lee, MD *Ira M. Lesser, MD *Gregory R. Mason, MDChrisanna M. Mink, MDRichard Mink, MD *Anita L. Nelson, MD *Ronald J. Oudiz, MD, FACC

Brant A. Putnam, MD*Jacob Rajfer, MD*Michael G. Ross, MD, MPH *Jerome I. Rotter, MD*Bruce E. Stabile, MD*Darryl Y. Sue, MD*Ronald S. Swerdloff, MD *+Timothy L. Van Natta, MD*Christina Wang, MD *Rodney A. White, MD* +Sylvia Yeh, MD *Kenneth M. Zangwill, MD*

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LA BioMed to Study Vitamin A’s Potential Benefits for Multiple Sclerosis Patients – Conrad N. Hilton Foundation Awards $900,000 Grant for ResearchThe Conrad N. Hilton Foundation recently awarded LA BioMed a $900,000 grant to study the role of serum Vitamin A in halting – or at least slowing – disease progression in people with Relapsing-Remitting MS. About 80% of MS patients have Relapsing-Remitting MS, in which new symptoms flare up and then go into remission.

This grant will jump start this area of research in the United States and will be the first large-scale study of whether higher levels of Vitamin A in the bloodstream may lead to improved outcomes for people who have Relapsing-Remitting MS. If the study finds MS patients with higher levels of Vitamin A have reduced progression of the disease, we could then examine using Vitamin A as an additional treatment.

LA BioMed is a Recipient of $67,000 from Harbor Community Benefit FoundationThe Port of Los Angeles approved $550,000 for nine “Healthy Harbor Grants” which are administered by Harbor Community Benefit Foundation. LA BioMed is a first-time recipient of one of these grants for $67,000 to expand the established 8-week Summer Fellowship Program. Twenty high school students from Wilmington and San Pedro will investigate such critical topics as the effects of port-related impacts on respiratory health in the Harbor community, and will each receive a stipend for their work.

The “Healthy Harbor Grants” were funded from the Port Community Mitigation Trust Fund, administered through the Port of Los Angeles’ partnership with San Pedro based Harbor Community Benefit Foundation (HCBF), an independent non-profit organization created to mitigate the environmental impacts of port operations.

These grants provide an opportunity for health interests, care providers, schools and other community organizations to apply for grants for projects and programs that improve the overall quality of life for harbor community residents of all ages. HCBF’s “Healthy Harbor Grants” connect San Pedro and Wilmington residents to a wide array of health and wellness resources. Local programs foster new partnerships and collaboration between the community and the Port, promoting economic growth and improving community health.

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