SBP scientist honored by the American Society for Bone and Mineral Research
AuthorMonica May
Date
September 28, 2018
José Luis Millán, PhD, professor in the Human Genetics Program at Sanford Burnham Prebys Medical Discovery Institute (SBP), has received the 2018 American Society for Bone and Mineral Research (ASBMR) Lawrence G. Raisz Award for his outstanding achievements in pre-clinical and translational research.
Millán has dedicated his career to understanding the mechanism of initiation of skeletal and dental mineralization. His pioneering research has led to the first-ever FDA-approved drug for a rare soft bone disease, hypophosphatasia (HPP); and a second drug candidate developed through a research collaboration with Daiichi Sankyo Company, Limited (Daiichi Sankyo) that entered a Phase 1, first-in-human clinical trial in 2017.
ASBMR’s award is named in honor of Lawrence G. Raisz, MD, a prominent scientist, mentor, teacher and clinician in the field of bone and mineral metabolism. Raisz was a founding member of ASBMR and the first editor-in-chief of the Journal of Bone and Mineral Research.
“Lawrence G. Raisz deeply influenced the skeletal mineralization field, so it is a true honor to receive an award in his memory,” says Millán. “In accepting this award, I want to thank the many individuals who enabled our achievements—from the National Institutes of Health (NIH), which has generously funded our research since the 1980s, to the collaborators and lab members who were instrumental in our scientific advances. I also want to thank SBP for providing state-of-the-art technologies that were invaluable to our research.”
Millán was presented the award onstage today at the ASBMR 2018 Annual Meeting in Montreal.
Interested in keeping up with SBP’s latest discoveries, upcoming events and more? Subscribe to our monthly newsletter, Discoveries.
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17th Annual Postdoctoral Research Symposium highlights
AuthorSusan Gammon
Date
September 27, 2018
The next generation of scientific leaders gathered to attend the 17th Annual Postdoctoral Research Symposium—an event that highlights the talent of SBP’s young scientists.
“Every year, the Annual Postdoctoral Symposium provides an opportunity for postdoctoral and graduate student scientists to showcase their most recent research,” says Stephen Sakuma, SBP-SN co-chair. “We are fortunate that SBP supports this event. It is great to learn more about the research conducted by my colleagues and opens up collaborative opportunities within the Institute.”
Unlike most symposiums that are specific to a field or topic of science, short talks and posters from more than 50 presenters represent the variety of science that takes place at our Institute—everything from heart disease to hair growth.
“One of the things I love about research at SBP is the range of scientific areas we study,” says Katja Birker, a graduate student who received an honorable mention for her podium talk. “I’m inspired by the work of my colleagues, and I hope my research similarly sparks ideas for others to think about.”
Prizes for best podium talks and posters were awarded. Congratulations to Michael Stec, PhD, for best oral presentation; and Joana Borlido, PhD, for best poster presentation. Stec studies skeletal muscle regeneration in the lab of Alessandra Sacco, PhD; and Borlido studies the molecular basis of leukemia in the lab of Maximiliano D’Angelo, PhD
Keith Yamamoto, PhD, professor, vice chancellor for Science Policy & Strategy, and director of Precision Medicine at UCSF, gave a keynote presentation reflecting on the importance of having a good mentor as an early-career scientist. Yamamoto has been a longtime mentor to Malene Hansen, PhD, associate dean of Student Affairs at SBP’s graduate school, and faculty adviser of Postdoctoral Training—who herself is guiding the keen minds of our Institute’s next-generation scientists.
“Getting an invitation to speak from students is much more significant than getting an invitation from faculty,” said Yamamoto. “I’m going to try to talk about my role as a mentor. I have benefited from that—being mentored is a lifetime experience and a lifelong need.
“Part of being a mentor is providing information of what you can do with a PhD and beyond,” Yamamoto continued. “It’s a community-wide and science-wide challenge for mentors.”
Many congratulations to all who participated in the 2018 symposium!
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2018 Fishman Award Ceremony honors postdoctoral scholars
AuthorSusan Gammon
Date
September 26, 2018
More than 100 supporters of SBP came out to celebrate this year’s Fishman Award recipients: Usue Etxaniz Irigoien, PhD, Koen Galenkamp, PhD, Laura Martin-Sancho, PhD, and Ee Phie Tan, PhD The annual awards ceremony, held September 20 at the Sanford Consortium, recognizes postdocs who have made extraordinary efforts and encourages their passion for careers in science.
The evening began with a warm welcome from founder Reena Horowitz, who described how she began the Fishman Fund with the late Mary Bradley (her dear friend) to honor Dr. William and Lillian Fishman. The Fishmans were the founders of SBP and firmly believed in helping young postdoctoral scientists become great principal investigators—and these awards help boost young researchers to meaningful careers in science.
Jeanne Jones, the Fishman Fund’s co-founder designee, shared that since the fund’s inception, 64 recipients have been awarded. The career development awards—bestowed annually to three postdocs—provide a generous $10,000 stipend to allow them to attend workshops and travel to national and international conferences to learn the latest developments in their research fields. Additionally, a Fishman Fund Fellowship Award, which provides salary support for two years in addition to benefits and a career-development stipend, was awarded this year for the second time in SBP history.
The keynote speaker was Aman Mann, PhD, who won the Fishman Award in 2011. Today, Mann is a research assistant professor at SBP and an entrepreneur and founder of AivoCode, a neuroscience company that licenses technology originally developed at SBP. Mann reflected on how the award gave him added confidence and resources to pursue his career goals—and he encouraged this year’s recipients to similarly follow their dreams.
If you are interested in donating to the Fishman Fund, click here.
The 2018 Fishman Fund Fellowship winner, Usue Etxaniz Irigoien, PhD, was presented with her award by Reena Horowitz. Irigoien, originally from the Basque region of Spain, is the first in her family to pursue a graduate degree in science. She is currently a postdoctoral researcher in the laboratory of Lorenzo Puri, PhD, and is studying the communication between nerves and muscles. Irigoien’s research is providing insights that may lead to improved therapies for neuromuscular diseases such as ALS. In the future, she hopes to become a principal investigator of her own lab.
The Fishman Fund Awardees:
Koen Galenkamp, PhD, was presented with his award by Andrew Viterbi, a distinguished scientist and former SBP trustee. Galenkamp works in the laboratory of Cosimo Commisso, PhD, and specializes in seeking ways to starve pancreatic tumors of the food they need to survive and grow. Originally from Amsterdam, Galenkamp thanked his colleagues and wife—who is also a postdoc in San Diego—and shared his plans to use the funds to attend a comprehensive pancreatic cancer conference at Cold Spring Harbor Laboratories.
Laura Martin-Sancho, PhD, a postdoc in the lab of Sumit Chanda, PhD, received her Fishman Fund Award from Armi Williams, a Fishman Fund Board member and longtime SBP supporter. Martin-Sancho is working to develop antiviral medicines that will combat infectious diseases such as influenza, dengue, and West Nile and Zika viruses. Martin-Sancho’s career goal is to be an independent investigator in an academic organization. She will use her stipend to attend a Keystone RNA virus conference in Dublin, Ireland.
Ee Phie Tan, PhD, originally from Malaysia, joined SBP in 2016 to work in the lab of Malene Hansen, PhD Tan is studying autophagy—a cell process that tidies up cell debris and recycles parts to maintain health. Defects in autophagy are linked to many aging-related diseases such as Alzheimer’s and Parkinson’s disease, and even cancer. Tan received her award from Reena Horowitz and will use the funds to attend a Gordon Research Conference in Texas and a Keystone Symposium in Santa Fe, New Mexico.
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5 things to know about acute myeloid leukemia (AML)
AuthorMonica May
Date
September 26, 2018
It’s no surprise that our blood is important. The cargo it transports—nutrients, infection-fighting cells, clotting factors, waste and more—keeps our body healthy and running smoothly. So when blood cells don’t form properly, serious cancers can occur.
Scientists divide blood cancers into three broad categories—leukemia, lymphoma and myeloma—based on the cell type affected. Leukemias disrupt white blood cell production; lymphomas affect the lymphatic system, which removes extra fluid from the body; and myelomas affect plasma cells, which produce intruder-fighting antibodies. There are many subsets within each category.
In honor of Blood Cancer Awareness Month, we spoke with Sanford Burnham Prebys Medical Discovery Institute scientist Ani Deshpande, PhD, to learn more about the blood cancer he studies: acute myeloid leukemia (AML). Of the 60,000 American children and adults diagnosed with leukemias each year, nearly 30 percent will have AML.
Most patients receive the same treatment used nearly five decades ago. Drug developers have created medicines for AML patients who have certain changes in their DNA, called mutations. But the majority of AML patients receive the treatments used in the ’70s: chemotherapy, radiation and possibly a bone marrow transplant. This isn’t last-decade science; it’s last-century science.
It’s deadly. The five-year survival rate for adults with AML—the number of people who are alive five years after diagnosis—is only 24 percent, according to the American Cancer Society. New medicines and treatment approaches are urgently needed.
Sequencing is making strides. Now, scientists can sequence patients’ genomes to learn the underlying mutation driving their cancer. This technology has advanced our understanding to the point that about 60 to 70 percent of the time, their doctor knows the mutation involved. Our new problem is that we don’t have effective medicines that target most of these mutations.
Speaking of sequencing. Because of DNA sequencing, we also know that a large fraction of the mutations in AML are epigenetic changes—alterations that affect which genes turn on but don’t change the DNA itself.
To better understand how epigenetic changes work, imagine a cookbook. If recipes are DNA, then epigenetic changes are bookmarks. These bookmarks signal whether the recipe should be made or not, without altering the underlying text of the recipe.
Our laboratory is studying the epigenetic changes that drive AML. Our hope is that once we identify these changes, we can create drugs that restore the epigenome to its normal state.
There is hope. After nearly 50 years of little progress, four new drugs have been approved for AML over the last 18 months. And there are currently more than 330 clinical trials enrolling patients in the U.S., so more treatments may soon follow.
Interested in keeping up with Sanford Burnham Prebys’ latest discoveries, upcoming events and more? Subscribe to our monthly newsletter, Discoveries.
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SBP scientist recognized by Biocom for contributions to San Diego life science industry
AuthorMonica May
Date
September 25, 2018
Tao Long, PhD, assistant professor in the Bioinformatics and Structural Biology Program at Sanford Burnham Prebys Medical Discovery Institute (SBP), has been named a 2018 Life Science Catalyst by Biocom, the association representing the California life science industry.
This annual award celebrates up-and-coming individuals in the life science industry—including scientists, entrepreneurs, investors, corporate leaders and business advisers—who are making a lasting and positive mark on Southern California’s life science industry prior to their 40th birthdays. Read the full list of winners.
Scientists know that individuals’ genetic makeups and gut microbiomes play important roles in human health. But sorting through genomic and clinical data is technologically complex. Long and her team are developing bioinformatics and machine-learning algorithms to help uncover links between our genes as well as the genes of the trillions of microbes that live in our gut and disease. This work could help us learn more about health conditions ranging from cancer to Alzheimer’s disease.
“Marked by their contributions to research, discovery and entrepreneurship in San Diego and Los Angeles, this year’s Life Science Catalyst Award recipients showcase the up-and-coming leaders who are transforming the life science industry,” says Joe Panetta, president and CEO of Biocom. “Biocom is honored to recognize these people who are utilizing their skill sets to positively impact human health across the globe.”
The 17 award winners will be featured in the fall edition of Biocom’s LifeLines magazine and honored during the December Biocom Board of Directors meeting.
Interested in keeping up with SBP’s latest discoveries, upcoming events and more? Subscribe to our monthly newsletter, Discoveries.
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3 things to know about celiac disease
AuthorMonica May
Date
September 13, 2018
Gluten-free products seem to be popping up everywhere—from grocery store shelves to restaurant menus. Going gluten-free may seem like a fad—but for more than 3 million Americans with celiac disease these products are their only treatment option.
This autoimmune disease is caused when the immune system—for unknown reasons—starts to identify gluten protein not as food but an intruder. The resulting immune response damages the walls of the small intestine. This causes symptoms such as gastrointestinal (GI) upset, fatigue and weight loss.
However, more than half of adults with celiac disease have non-digestive symptoms, including depression, osteoporosis and arthritis. A whopping 80 percent of Americans with celiac disease are not diagnosed.
In honor of celiac awareness day, we spoke with Scott Peterson, PhD, professor at Sanford Burnham Prebys Medical Discovery Institute (SBP), who studies links between the microbiome and chronic inflammation. He’s also familiar with the struggles people with the condition face each day: His wife was diagnosed with celiac disease about ten years ago.
Here are three things he told us we should know about celiac disease:
It could unlock secrets to other autoimmune disorders. Unlike many autoimmune disorders, scientists know the cause of celiac disease—gluten protein. This provides a unique opportunity to understand what triggers celiac disease, which could help us better understand more autoimmune disorders.
Signs point to the microbiome. Nearly 70 percent of our immune cells are in our gut. Our immune system is constantly surveilling our gut microbiome—it’s like the best friend you don’t quite trust. And indeed, scientists are finding the gut microbiome of people with celiac differs from people without the disease, and changes after they start a gluten-free diet. This could explain the brain-related symptoms: Nerve cells line the GI tract, connecting our gut and brain.
Truly gluten-free diets are hard to achieve. Removing gluten sounds simple—but this protein shows up in surprising places. Play-dough, lip balm and even medicine can contain gluten. For this reason, nearly 30 percent of people with celiac disease who go gluten-free still experience symptoms. In addition to improving their quality of life, a drug could help them avoid long-term effects of untreated celiac disease, including cancer and bone loss.
Interested in keeping up with SBP’s latest discoveries, upcoming events and more? Subscribe to our monthly newsletter, Discoveries.
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SBP secures grant to prepare early career scientists for jobs in industry
AuthorMonica May
Date
August 22, 2018
A five-year training grant from the National Cancer Institute (NCI) has been awarded to Garth Powis, D. Phil., professor and director of the Sanford Burnham Prebys Medical Discovery Institute (SBP) NCI-designated Cancer Center. The funding will establish a training program to help graduate students and postdoctoral scholars at SBP explore potential careers in cancer research in the biotech industry.
In 1973, more than half of biologists with PhD’s obtained tenure-track positions within six years of graduation. In 2006, this number shrank to 15 percent. The majority of researchers now work in government; at pharmaceutical or biotechnology companies; for independent research institutes; or in research-related positions, such as patent law, science writing and more.
“Opportunities for scientists today are incredibly diverse, creating a need for training programs that help early-career researchers better understand their career possibilities,” says Powis. “We are grateful for the support from the NCI, a leader in creating environments to help innovators learn the business side of science. This program will help streamline the career path for young scientists interested in advancing cancer research in an industry setting, helping them attain successful, fulfilling roles at an earlier point in their career.”
The program will have the capacity for one graduate student and three postdoctoral scientists at SBP. Participants would apply for the program and be selected based on pre-specified criteria. Applications are currently being accepted.
Powis adds, “This training program is also valuable for scientists who wish to stay in basic research, as their discoveries will eventually transition to biotech and pharmaceutical scientists on their journey to patients. Firsthand understanding of the complexities of drug development increases the chances an idea can become a medicine, so this is an invaluable learning opportunity for SBP’s graduate students and postdoctoral scholars.”
Read more about the National Cancer Institute (NCI) Ruth L. Kirschstein National Research Service Award (NRSA) Institutional Research Training Grant (T32).
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SBP scientist awarded BrightFocus Foundation grant to advance Alzheimer’s research
AuthorMonica May
Date
August 21, 2018
The BrightFocus Foundation, a nonprofit working to end diseases of mind and sight, has awarded a two-year grant to Yingjun Zhao, PhD, research assistant professor at Sanford Burnham Prebys Medical Discovery Institute (SBP). This funding will advance Zhao’s study of a protein associated with memory loss in Alzheimer’s disease, called appoptosin.
Alzheimer’s disease is a growing epidemic. In the U.S. alone, more than 5 million people over age 65 are living with the disease. In thirty years, as the population ages, scientists expect this number will nearly triple to 14 million Americans—unless we find a prevention or treatment.
The protein Zhao studies, appoptosin, regulates cell death. His previous work showed the protein exists at higher levels in people with Alzheimer’s disease. Removing the protein slowed memory loss in mice—indicating it has therapeutic potential. Now, this grant will allow Zhao to better determine the link between appoptosin and memory loss in Alzheimer’s disease. The outcome of the research could yield new therapeutic targets for Alzheimer’s—valuable insights for scientists on the hunt for new treatments.
“Most studies focus on memory formation, but people with Alzheimer’s
Zhao and new arrival, Harry.
have trouble both forming and keeping memories. Our work focuses on forgetting,” says Zhao. “We hope new leads for drug development will arise from this research, which will offer hope for people with Alzheimer’s and their caregivers. Thank you to the BrightFocus Foundation for supporting this important research.”
Zhao has another milestone to celebrate: He recently welcomed his second child. As a father, this work takes on special meaning for Zhao. “I hope one day not only my children—but everyone’s children—can live a life free from Alzheimer’s disease,” he says.
Interested in keeping up with SBP’s latest discoveries, upcoming events and more? Subscribe to our monthly newsletter, Discoveries.
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SBP’s Alexey Terskikh advances hair growth research
AuthorMonica May
Date
August 16, 2018
Three years ago, Alexey Terskikh, PhD, associate professor in Sanford Burnham Prebys Medical Discovery Institute’s (SBP’s) Development, Aging and Regeneration Program, published a groundbreaking study showing that stem cells could be used to grow hair.
This discovery could help more than 80 million men, women and children in the United States experiencing hair loss. Across cultures, personal identity is connected with hair. As a result, hair loss often affects emotional well-being and self-esteem. There is clear interest in the technology: Our 2015 story on this finding remains our blog’s most-read article.
Since then, Terskikh and his team have been working hard to advance this technology. We caught up with Terskikh to learn about his progress—and how far away the research remains from human studies.
Alexey Terskikh, PhD
Could you fill us in on your work since 2015?
For the past three years, my team and I have been working to overcome several obstacles to the technology’s real-world use. We’ve made progress on multiple fronts, summarized below:
Generating unlimited cells
Instead of embryonic stem cells, which are difficult to obtain, our method now uses induced pluripotent stem cells (iPSC), which are derived from a simple blood draw or skin sample. iPSCs allow us to create an unlimited supply of cells to grow hair. Not having enough hair is one reason current transplants don’t work, so this is a critical advance.
Creating a natural look
Hair actually grows in a specific direction, so it’s important to control the orientation of hair growth to achieve a natural look. Your hair stylist is familiar with this!
We’ve found a solution—3D biodegradable scaffolds—and partnered with leading scientists in the field to advance our project. The scaffold allows us to control the number of cells transplanted, their direction and where they are placed.
Helping the transplant “take”
The scaffold has a second job of helping seed hair follicles. Skin is a good barrier—that’s its job—so we needed something to help the transplant “take.” The scaffold provides the “soil” from which the hair can grow.
Hair-inducing human cells (red)
generated from iPSC present within hair
follicles grown in mouse skin.
I understand you have formed a company based on this research. Can you tell us more?
Yes, we have formed a company this year and assembled a great team with the expertise needed to move the technology forward. These experts include hair transplantation specialists, experienced entrepreneurs and experts in manufacturing cells at large scale (not a trivial endeavor).
While hair loss affects people’s self-esteem and self-image, it isn’t life threatening, so it’s not a top priority for many funding agencies. Forming a company gives us a vehicle for raising capital to advance this technology.
Do you know how the stem cell–generated hair will look? Can you control hair color?
We hope that stem cell–generated hair will look exactly as the original hairs that have been lost. Of course, it will take some time to grow a “perfect” hair, but we believe this should be possible in the long run.
Has anything surprised you during this process?
I expected to hear from young and older men, but I was surprised by the number of women who reached out to express interest in our research. I received about an equal number of emails from women. Pregnancy, menopause and ovarian conditions may all cause hair loss for women.
Most heartbreaking were emails from parents of children with alopecia, a condition where a child cannot grow hair. As you can imagine, hair loss at such a young age can affect relationship formation and self-image. All these emails continue to motivate me to keep advancing this research as quickly as possible.
What work needs to be done before you can test this on humans? How far away are we from this product being used on humans?
The good news is that we’ve resolved the biological mystery of hair growth using stem cells. Now, it is mostly an engineering exercise: how to get robust and properly oriented hair growth.
Before we can discuss human studies with the U.S. Food and Drug Administration (FDA), we need to complete safety and tumorigenicity tests in mice. We are performing these tests very soon.
Provided we have the proper funding, we expect it will take two years before we can start discussions with the FDA.
Assuming all goes as planned and the FDA approves a first-in-human study, will everyone be eligible for the trial?
At that point we will work very closely with clinical experts in the field to determine which individuals are most likely to benefit from this research and should be involved in the trial.
How did you first get started on this research?
That’s actually a funny story. My father—who is a scientist—wanted to stay more in touch, so we decided to do a joint project. I was researching stem cells, and he was researching skin follicles, so we ended up here! If you look at the paper, you’ll see two authors who have the same last name—him and me.
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Surprising science: Not all our cells have the same DNA
AuthorMonica May
Date
August 15, 2018
We learned in biology class that every cell in the body has the same DNA. Whether a heart cell, skin cell or muscle cell—they all read from the same genetic blueprint.
Now, scientists are learning there is more to the story. New research into brain cells is revealing that their DNA blueprint is radically different than expected. Compared to DNA in other cells, neurons have more, less and rearranged DNA. And these changes accumulate over time.
Jerold Chun, MD, PhD, professor and senior vice president of Neuroscience Drug Discovery at Sanford Burnham Prebys Medical Discovery Institute (SBP), is pioneering research into this phenomenon, called genomic mosaicism. Due to his expertise, he was recently selected by a leading scientific journal to write an overview of the latest research on the topic.
We caught up with Chun and asked, “What are three things we should know about genomic mosaicism in the brain?” Below are his answers.
It could explain many medical mysteries.Why do brain disorders arise sporadically? Why don’t babies get Alzheimer’s? What causes autism? Brain disorders are one of the greatest mysteries and medical challenges of our time. Genomic mosaicism provides a potential explanation.
Brain cells aren’t the only ones with jumbled DNA. Our immune system was an early example of genomic mosaicism occurring in normal cells. To protect us, our immune system rearranges DNA to create cells that can recognize and remove unwanted intruders. Genomic mosaicism may not be as unusual as we had thought.
We don’t yet know how this genomic mosaicism occurs. We know DNA in brain cells varies wildly. But we don’t know how our neurons create these differences. Understanding the mechanism is a critical next step for the field. If we understand how brain-cell DNA changes, we can better understand when the process goes awry and if that causes disease.
Unlocking the secrets of genomic mosaicism in the brain has the power to change textbooks and, more important, people’s lives. The underlying cause of many brain disorders—Alzheimer’s, autism, schizophrenia and more—may lie in the surprising scrambling of our brains’ DNA.
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