Mouse Study Demonstrates Promise of New Therapy for Rare Genetic Conditions

If you ask a scientist how old you are, you may be surprised to get a different answer depending on who you’re talking to. That’s because age can be measured both ‘chronologically’ — in terms of time — and at a cellular level. Indeed, certain genetic mutations cause cells to age faster, leading to a host of health problems. Fortunately, a recent IRP study performed in mice suggests that boosting levels of a specific molecule could help alleviate some of those patients’ symptoms.

IRP Research Examines Under-Appreciated Factors in Alzheimer’s Disease

This June, the observance of Alzheimer’s and Brain Awareness month reminds us just how devastating the impact of Alzheimer’s and other dementias is across the world. About 55 million people are currently living with Alzheimer’s disease or related dementias such as frontotemporal dementia and Lewy body dementia. This includes the estimated 6.7 million people in the U.S. over age 65 with Alzheimer’s disease, a population whose cognitive decline imposes huge financial costs on the American economy and often unrecognized burdens on the unpaid family members who provide care for those patients.

While the disease has been a target of intensive investigation ever since Alois Alzheimer first discovered clumps of amyloid beta protein and tangles of tau in the brain of a deceased patient in 1906, scientists have made only modest progress in treating it. Efforts to design drugs targeting the abnormal globs of amyloid and tau thought to cause Alzheimer’s have met with only limited success. That’s why IRP investigator Keenan Walker, Ph.D., is taking a different approach: exploring the link between dementia risk and the immune system.

Highlighting Drugs and Vaccines Stemming from NIH Discoveries

On May 3, more than six decades of IRP research culminated in the U.S. Food and Drug Administration (FDA) approving the world’s first vaccine for respiratory syncytial virus (RSV), a disease that puts tens of thousands of Americans in the hospital each year and kills thousands. While the new vaccine, called Arexy, has been getting all the headlines recently, it is only the latest example of a slew of FDA-approved medications and vaccines that might never have existed without the tireless efforts of scientists at NIH.

Indeed, a recently published study led by Mark Rohrbaugh, Ph.D., J.D., a special advisor in the NIH Office of Science Policy, found that inventions developed at NIH have contributed to more FDA-approved products than those created at any other nonprofit research institution in the world over the past five decades. NIH tops the study’s list with 27 FDA-approved products, six more than the study attributed to the combined efforts of all the schools in the University of California system.

Budding Scientists Assemble for First Time Since 2019

In many ways, working at NIH feels like being at a world-renowned university, complete with a variety of classes, intellectually stimulating lectures, social events, and opportunities for professional development. It’s no wonder, then, that NIH has long been a destination for young people who have just departed from their alma maters with their newly earned undergraduate degrees.

These new graduates come to the IRP to hone their scientific skills in NIH’s Postbac IRTA program, conducting research in IRP labs for one or two years under the expert guidance of the IRP’s seasoned investigators. The program also provides the opportunity once per year for participants to present the fruits of their efforts to all their IRP colleagues at Postbac Poster Day, and this year’s event on April 19 and 20 was the first to include an in-person component since 2019. Read on to learn about a few of the nearly 1,000 postbacs who showed off their research at this year’s event, which spanned fields from neuroscience and cancer to genetics and virtual reality.

IRP Research Aims to Explain the Perils of Peanuts and Other Foods

If you are a parent of school-age children, you’ve probably received a list of prohibited lunch foods and bans on birthday cupcakes. Going out to eat or cooking for guests can present a similar minefield of ingredients that many people must avoid. If it seems like food allergies are on the rise, it’s not your imagination. The U.S. Centers for Disease Control and Prevention (CDC) reports that the prevalence of food allergies has increased by 50 percent since the 1990s, making it a serious public health concern.

This May, Food Allergy Awareness Week reminds parents, kids, teachers, food service workers — really all of us — that we must remain vigilant to the risks of reactions to certain foods. These allergies affect nearly 32 million Americans, including 1 in 13 kids. If you think about the average classroom, that could be two or three children with severe allergies in one room. For many, even a tiny amount of an allergen can trigger a serious, even life-threatening response by the body’s immune system. To address this growing concern, IRP senior investigator Pamela A. Guerrerio, M.D., Ph.D., and her colleagues in the Food Allergy Research Section at the National Institute of Allergy and Infectious Diseases (NIAID) are working to unravel how genetics, immune system development, and environmental factors interact to cause food allergies in children.

Three Young Women Honored at Annual Event

There’s no doubt that science flourishes when it welcomes individuals with different backgrounds and perspectives. In pursuit of that goal, NIH has put considerable effort into closing the gender gap in the biomedical sciences, a field in which men significantly outnumber women, especially as the heads of labs and in leadership positions. Those efforts have so far yielded promising progress, with the proportion of women in IRP senior investigator positions increasing from 22 percent at the end of fiscal year 2016 to 27 percent at the end of fiscal year 2022. Over that same time period, women went from comprising 38 percent of IRP tenure-track investigators to 44 percent.

Part of what makes the IRP a welcoming place for female scientists is the NIH Women Scientists Advisors (WSA), a group of IRP scientists elected to represent the interests of women in the IRP. Once per year, the WSA selects a few female postdoctoral fellows or graduate students conducting research at NIH as WSA Scholars. At a symposium on April 13 honoring the achievements of this year’s Scholars, the awardees presented their efforts to learn more about a devastating childhood neurological condition, decrease health disparities in breast cancer, and use stem cells to investigate the roots of a nerve-destroying disorder. Read on to learn more about this year’s WSA Scholars and the important work they’re doing in their IRP labs.

Three Young Women Honored at Annual Event

There’s no doubt that science flourishes when it welcomes individuals with different backgrounds and perspectives. In pursuit of that goal, NIH has put considerable effort into closing the gender gap in the biomedical sciences, a field in which men significantly outnumber women, especially as the heads of labs and in leadership positions. Those efforts have so far yielded promising progress, with the proportion of women in IRP senior investigator positions increasing from 22 percent at the end of fiscal year 2016 to 27 percent at the end of fiscal year 2022. Over that same time period, women went from comprising 38 percent of IRP tenure-track investigators to 44 percent.

Part of what makes the IRP a welcoming place for female scientists is the NIH Women Scientists Advisors (WSA), a group of IRP scientists elected to represent the interests of women in the IRP. Once per year, the WSA selects a few female postdoctoral fellows or graduate students conducting research at NIH as WSA Scholars. At a symposium on April 13 honoring the achievements of this year’s Scholars, the awardees presented their efforts to learn more about a devastating childhood neurological condition, decrease health disparities in breast cancer, and use stem cells to investigate the roots of a nerve-destroying disorder. Read on to learn more about this year’s WSA Scholars and the important work they’re doing in their IRP labs.

The IRP community mourns the recent passing of our colleague W. Michael Kuehl, M.D., former senior investigator in the Genetics Branch at the National Cancer Institute (NCI), after a long struggle with renal cancer.

In his more than 30 years at NCI, Michael was devoted to understanding the cellular and molecular biology of multiple myeloma (MM) and pre-malignant MGUS (monoclonal gammopathy of uncertain significance), a precursor of the disease. He identified multiple mechanisms of molecular pathogenesis in MM and he linked pre-malignant MGUS to MM, which is relevant for the early diagnosis and treatment of MM.

IRP Research Breathes New Life into Therapies for Treatment-Resistant Asthma

It may start with a wheeze, a cough, or a feeling of tightness in the chest, but the result is the same. Acute asthma attacks make sufferers feel like they’re breathing through a straw while underwater. And even between attacks, having asthma can sometimes feel like your lungs are bound in tight bandages that make it difficult to take a deep breath.

World Asthma Day, observed this year on May 2, raises awareness of asthma, a common inflammatory disease that causes difficulty breathing in more than 260 million people worldwide, including 25 million in the U.S., roughly 8 percent of the country’s population. While many people can control their symptoms by taking medications and limiting certain activities, the condition still causes significant illness and even death.

The IRP community is saddened by the recent passing of our esteemed colleague, Herb Geller, Ph.D., who died April 16, 2023, at the NIH Clinical Center from complications of advanced prostate cancer. He is survived by his wife of 55 years, Nancy Geller, Ph.D., who is the Director of the Office of Biostatistics Research at the National Heart, Lung, and Blood Institute (NHLBI).

Dr. Geller joined the National Institutes of Health in 2001, where he became the Chief of the Developmental Neurobiology Laboratory in the Cell and Developmental Biology Center at NHLBI. During his long and successful career in neurobiology, Dr. Geller made a significant impact on the field of neuroscience. His laboratory focuses on understanding the role of the extracellular matrix — the intricate network of molecules that surround cells which control migration, pathfinding, and growth of neurons during brain development.

Understanding How Parasite Feeds May Lead to New Treatments

World Malaria Day, commemorated annually on April 25, highlights the need to end an infectious disease that sickens nearly 250 million people around the world each year, killing more than 600,000 in regions where it is common. Children are particularly susceptible to its deadly effects.

Unfortunately, there is still no highly effective vaccine against malaria, so management is mostly limited to preventive measures like bed nets and medications that treat the infection, which must be taken over an extended period to effectively treat the disease and stop it from spreading. However, the speed with which the malaria parasite, Plasmodium falciparum, adapts to antimalarial drugs has created a critical need for novel treatments — a need that IRP researchers led by Joshua Zimmerberg, M.D., Ph.D., are taking a unique approach to filling.

Interrupting Sedentary Time Could Help Stave Off Health Problems

Many people don’t get much exercise these days, and kids are no exception. Whether at school, doing homework, or entertaining themselves online, children and teens spend hours on end sitting around. That lack of physical activity raises their risk for metabolic conditions like obesity and type 2 diabetes, but according to a recent IRP study, breaking up those long, sedentary periods with just a few minutes of exercise could yield noticeable benefits for their health.

IRP Researchers Pursue New Approaches to Treating Alcohol Use Disorder

While most adults in the U.S. consume alcohol in moderation, for nearly 30 million of them, going even one day without alcohol feels nearly impossible. For these Americans, alcohol use disorder (AUD) is a serious condition that harms their health, relationships, and career. Unfortunately, only a small percentage of people with AUD receive treatment, and even then, for many patients, the chances of a relapse are high.

As the search for a reliable and effective treatment continues, IRP senior investigator Lorenzo Leggio, M.D., Ph.D., is exploring the biological processes that underlie alcohol cravings to unlock new approaches to therapy. April is Alcohol Awareness Month, so we took the opportunity to speak with him about recent discoveries made by his IRP team and its collaborators.

IRP Study Points to Strategies to Stop Disease From Spreading

Ancient Greek myth describes how the hero Hercules battled the many-headed hydra, which regrew two heads every time Hercules cut one off. This frustrating fight against a seemingly invulnerable opponent would be an apt metaphor for treating cancer, in which tumor cells sometimes die in a particular way that actually helps their brethren multiply and spread to other parts of the body. In a study of that phenomenon using a mouse model of breast cancer, IRP researchers discovered that it occurs because that form of cell death suppresses the immune system’s response to the cancer, a finding that points to several potential ways to improve cancer therapy.

NIH Scientist Proves What Happens in the Mouth Doesn’t Stay in the Mouth

For many people, good oral health means a pretty smile, not necessarily a healthy body. However, poor dental health can lead to serious illness and disability. IRP senior investigator Jennifer Webster-Cyriaque, D.D.S., Ph.D., has spent her career illuminating the connection between our mouths and our overall health. Dr. Webster-Cyriaque, who recently joined NIH as Deputy Director of the National Institute of Dental and Craniofacial Research (NIDCR) and Chief of the Viral Oral Infections in Immunosuppression and Cancer Laboratory at the National Institute of Allergy and Infectious Diseases (NIAID), was elected to the National Academy of Medicine in October for her seminal contributions to our understanding of viruses that infect the mouth.

NIH Archives Document the Tremendous Importance of Female Scientists

Women’s History Month is celebrated every March, and women scientists have undoubtedly made invaluable contributions to IRP research over the course of NIH’s history. Despite this, women still remain under-represented in biomedical science at NIH and elsewhere today, prompting the IRP to make supporting the careers of female researchers an important priority. While NIH works to rectify the gender imbalance in scientific research, it’s important to take time to celebrate the many women who, even when confronted with significant historical obstacles, have made a name for themselves in the lab and on the pages of scientific journals. Join me in taking a look through the archives of the Office of NIH History & Stetten Museum to learn about some of the many women scientists who have been at the forefront of science and administration at NIH.

Study Suggests New Treatment Approach for Deadly Lung Infection

Oxygen is, quite literally, the air we breathe (or, more accurately, 21 percent of it). However, just as oxygen in the air can turn a handy garden tool into a useless hunk of rust, certain unstable, oxygen-containing molecules in our bodies can wreak havoc on our cells. According to new IRP research, revving up cellular systems that prevent this kind of damage could significantly improve outcomes for people with tuberculosis.

IRP Researchers Are Peering Into the Brain to Learn Why Opioid Drugs Are So Hard to Quit

The ancient Egyptians, despite their significant anatomical knowledge, thought the heart was the seat of intelligence. Over the millennia, that view changed as philosophers and scientists alike came to appreciate the extraordinary role of the brain. It is partly thanks to them that we celebrate Brain Awareness Week every March. In honor of this observance, we took the opportunity to talk with IRP senior investigator Yihong Yang, Ph.D., and postdoctoral fellow Ida Fredriksson, Ph.D., Pharm.D., about their investigation into how cravings for opioids build during a period of prolonged abstinence, often leading to relapse.

Event Includes In-Person Presentations for First Time Since 2020

Three years after COVID-19 dramatically changed the way scientists and many others work, much of life in the NIH IRP has begun to resemble the way things were in February of 2020. This includes the return of in-person scientific poster sessions like the one that took place on February 16 as part of the 19th annual NIH Graduate Student Research Symposium. Nearly 130 graduate students conducting their Ph.D. research in IRP labs as part of NIH’s Graduate Partnership Program presented their progress at that poster session and its virtual counterpart held February 15.

The two poster sessions made it clear that IRP graduate students are essential contributors to the life-changing discoveries made at NIH, from using geckos to learn about human eye diseases to investigating how the immune system combats infectious invaders to exploring ways to improve cancer treatment. Keep reading to learn about some of the bright scientists-in-training who showed off their work during the two-day event.

IRP Research Hastens Development of First Treatment for Genetic Muscle Condition

An old medical adage warns doctors that when they hear hoofbeats, they should first think of horses, not zebras. After all, when someone comes into the hospital with a cough, the most likely explanation is something mundane like the flu. However, some patients truly are medical zebras, affected by a disease that afflicts very few others.

IRP senior investigator Marjan Huizing, Ph.D., has learned quite a bit about those zebras since arriving at the National Human Genome Research Institute (NHGRI) as a postdoctoral fellow in 1998. To help commemorate Rare Disease Day today, Dr. Huizing spoke with the “I Am Intramural” blog about her research on an array of ailments linked to a small sugar molecule called sialic acid, some of which are extremely rare.