Pennsylvania: In the grand scheme of things, it can seem like progress in science
and medicine moves pretty slow. For example, we still aren’t sure what
causes premature birth, or why racial disparities exist in certain
conditions. And, for all intents and purposes, Alzheimer’s is still a
big black box. But in reality, when you put science and medicine under
the microscope, you find that a single year can bring big, meaningful
gains and exciting advances. So, as we welcome a brand new year, let’s pause to reflect on some of
Penn Medicine’s biggest newsmakers from the last 12 months.1. Public health vaccines
According to the Institute for Health Metrics and Evaluation at the University of Washington, more than 41 million people around the world have died in the past decade from AIDS, malaria, tuberculosis, Ebola and other tropical diseases. While we have learned a great deal about prevention against and treatment for these epidemics, vaccines and cures remain elusive.
Still, immense progress in the fight against these devastating diseases was made this year.
At Penn Medicine, infectious disease researcher Pablo Tebas, MD, is leading a trial of an experimental DNA vaccine for the Zika virus, which burst onto the world stage as a crisis in 2015, when officials in Brazil reported the presence of neurological disorders in newborns associated with history of infection. Unlike conventional vaccines, which can take years to develop and test, DNA vaccines can be developed in mere weeks, allowing researchers to begin testing in humans in a matter of months. It’s also suspected that DNA vaccines may provide longer-lasting protection from diseases.
This year, Tebas also led groundbreaking research which provided new insights into how immunotherapy might be used to develop a functional cure for HIV, eliminating the need for people living with the virus to take a daily regimen of medications. In the study, researchers examined chronically HIV-infected patients and found that injections of one broadly neutralizing HIV antibody (bNAb), known as VRC01, were safe, generated high levels of the antibody, and modestly delayed the time of HIV viral rebound compared to historical controls. Unfortunately, in the majority of participants the treatment was not effective for more than eight weeks. Still, by demonstrating that HIV-specific antibodies can suppress or even kill HIV-infected cells, this method is a first step toward the ultimate goal of durable suppression of HIV.
At Penn Medicine, researchers are using virtual reality to treat amputees with phantom limb pain, and exploring ways to use the technology to breathe new life into standard CPR training methods.
Phantom limb pain occurs when the brain sends motor commands to the absent limb expecting a response. Up to 90 percent of people who have lost a limb experience this pain. But now, Penn Medicine researchers are teaming up with the Moss Rehabilitation Research Institute to see if providing virtual legs for these patients can trick the brain into no longer sending pain signals.
H. Branch Coslett, MD, a professor of Neurology, told 6ABC about the project earlier this month. Though it’s still experimental, Coslett said the initial results are very promising. "I think it's endlessly exciting. I think it's something that really can help people,” he said.
According to the American Heart Association, when performed in the first few minutes following a cardiac arrest, CPR can double or triple a person’s chance of survival. And yet, unless you’re a doctor or were a lifeguard in high school, the odds that you know how to properly perform CPR are pretty slim. Using a new VR system, the Penn team is observing what people do if someone collapses right in front of them, with the hope that these observations will one day turn into data that will guide training techniques.
The team demonstrated their system at the Franklin Institute’s Virtual Reality Exhibit earlier this year. In an article about the exhibit from PhillyMag.com, George Lin, who works on the project under the guidance of Marion Leary, MPH, MSN, RN, director of Innovation Research at Penn’s Center for Resuscitation Science, said the team is hoping to “see if we can improve emergency preparedness training. With this tech, we’re hoping to get more people doing CPR and ultimately a higher survival rate for those who might experience cardiac arrest.”
Likes, comments, shares, and retweets have become very powerful forces for individuals and businesses alike. But the power of social media doesn’t stop at food and vacation photos or even reaching new customers; while many consider social media to be one of life’s greatest distractions – causing us to bump into one another as we walk with our heads down, or weave through traffic on the Schuylkill, while posting our whereabouts – research published in 2016 shows that sites like Facebook, Twitter and Yelp can be a valuable resource for health care providers in several ways.
Results of a study published in JAMA Cardiology earlier this year suggest that posts to Facebook and Twitter could yield important information about patient health. In the study, Penn researchers sifted through ten billion tweets and found more than 500,000 that related to cardiovascular disease, most of which discussed risk factors or treatments, or where aimed at raising awareness about the disease.
In an article from Reuters Health, senior author Raina Merchant, MD, an assistant professor of Emergency Medicine and director of Penn’s Social Media and Innovation Lab, said she hopes other researchers will come up with questions and projects that use Twitter. "We have billions of tweets about how patients and health providers are thinking about health and heart disease," she said.
Merchant also co-authored a piece recently published by JAMA in which she and colleagues called on health providers to pay attention to reviews posted on online platforms like Yelp, where users are reading and writing reviews of businesses, including hospitals. In the article, the authors say there’s a growing body of research supporting the value of these unstructured reviews in supplementing ratings from formal sources such as the Hospital Consumer Assessment of Heathcare Providers and Systems (HCAHPS). In fact, another study from the group published earlier this year showed that while HCAHPS measure 11 different areas – such as discharge information and overall hospital environment – there are as many as 12 additional areas accounted for in Yelp reviews that are not addressed in formal surveys, including amenities, and family member services and care.
“That’s a really important population that we don’t otherwise hear from,” Merchant said in an article from STAT News.
4. Organ and Tissue Transplants
Advancements in organ transplantation are saving more lives than ever before. Kidney transplants are practically routine, and in 2016, Penn Medicine transplant surgeons not only performed their 1000th lung transplant, but also performed a record 100 lung transplants in a single year. But the fact remains, there just aren’t enough organs to go around, and many people end up waiting on transplant lists for several years. But now, thanks to multiple breakthroughs over the past year, some patients have new hope.
In an innovative clinical trial, transplant surgeons are now able to transplant kidneys from deceased donors who had the Hepatitis C virus (HCV) into patients who do not have HCV, with the intent to treat and ultimately cure the contracted infection. Early data published in September show that after transplanting HCV-positive kidneys into patients without the virus, doctors were able to eradicate the virus by treating participants with a full regimen of Zepatier – a recently-approved oral medication prescribed to eradicate HCV. Though more research is necessary before the procedure can be considered standard of care, the Penn team says it has the potential to provide a chance at a lifesaving kidney transplant for hundreds more patients each year.
"We always dreaded hepatitis C," said Peter Reese, MD, MSCE, an assistant professor of Medicine and Epidemiology, who is helping lead the research, in an article from the Associated Press. "But now hepatitis C is just a different disease," and the article says it’s “enough to consider what he calls the tradeoff of getting a new kidney years faster but one that comes with a hopefully treatable infection.”
Kiran Shelat, who received an HCV-positive kidney as part of the study, with his wife and daughter
Organs weren’t the only advancements in transplant this year. In
recent years Penn Medicine has proven to be a leader in vascularized
composite allotransplantation surgery – a fancy way of saying
“transplantation of multiple tissues such as muscle, bone, nerve and
skin, as a functional unit,” such as hands or a face. Penn Medicine
performed its first double hand transplant in 2011, but this year
continued to make strides toward improving the quality of life for
patients who have suffered devastating injuries and illnesses. In October, Penn surgeons performed Penn’s second bilateral hand transplant, this time on a woman from Paris – the very first international patient to receive two new hands in the United States. After living as a quadruple amputee for nearly nine years, patient Laura Nataf was to receive new hands at her local hospital in Paris. Unfortunately, complications prevented the surgery from taking place, and she was referred to Penn.
"Laura represents not only the progress being made in the field of bilateral hand transplantation," said L. Scott Levin, MD, FACS, chair of Orthoaedics and director of Penn's hand transplant program, in an article from The Philadelphia Inquirer. "But, she is living proof of our ability to collaborate with medical centers around the world to improve the quality of life of international patients, as well as those in the United States."
Just two months earlier, Levin welcomed back Penn Medicine and CHOP patient Zion Harvey, who one year ago became the first pediatric patient to undergo a bilateral hand transplant. While Zion’s surgery didn’t take place in 2016, his progress this year makes him worthy of being mentioned on this list. Just one year after receiving new hands, today Zion can throw a baseball over home plate, write in a journal, dress himself, make himself lunch, and break a huddle with the Baltimore Ravens.
"Here we are a year later. We're still working very hard as a team. We're still learning. We're still engaged. We see Zion in the clinic. We ask questions. We challenge each other, as we did during preparation," Levin told NBC Nightly News.
5. CRISPR
Developed four years ago, CRISPR – which stands for Clustered Regularly Interspaced Short Palindromic Repeats – is a tool that allows scientists to alter a single piece of DNA. Though the technology is still relatively new and many are urging caution in pushing CRISPR’s capabilities, two Penn Medicine teams are involved in cutting edge research that will uncover just how influential the technology may be, and more work is in the pipeline in other areas.
A group of researchers known as the “CRISPR Pioneers,” including Carl June, MD, a professor in the department of Pathology of Laboratory Medicine and director of the Center for Cellular Immunotherapies at Penn’s Abramson Cancer Center were named a runner up for Time’s 2016 Person of the Year. June and Edward Stadtmauer, MD, chief of Hematologic Malignancies and a professor of Hematology-Oncology in the Abramson Cancer Center, are poised to lead the nation’s first trial using CRISPR to treat 18 people with end-stage melanoma, sarcoma, and multiple myeloma. The trial, which will be the most extensive manipulation of the human genome ever, will use CRISPR to genetically engineer cells aimed at fighting disease. The article from Time explains that June’s team will “extract their T cells, a kind of immune cell, and use CRISPR to alter three genes in those cells, essentially transforming them into superfighters. The patients will then be reinfused with the cancer-fighting T cells to see if they do what they’re supposed to do: seek and destroy cancerous tumors.”
The trial is getting off the ground with backing from Silicon Valley entrepreneur Sean Parker through the groundbreaking Parker Institute for Cancer Immunotherapy, which Penn formed along with five other top-flight cancer centers last April during a star-studded launch in Los Angeles.
Meanwhile, over on the basic science side, Penn researchers are using CRISPR to make strides toward treating hemophilia – a genetic disorder that prevents blood from clotting, leaving those with the condition at risk of bleeding to death from even a minor scrape – in mice.
Hemophilia is caused by a genetic mutation in which an essential clotting protein is either missing or defective. In the new study, researchers removed the clotting protein from mice – essentially giving them hemophilia – and then used CRISPR to engineer human DNA that would resemble the protein in the mouse’s liver. By delivering injections of the engineered human DNA, the clotting protein levels returned to normal in both newborn and adult mice.
“Basically we cured the mice,” said lead author Lili Wang, PhD, in an article from Gizmodo. “With a gene therapy like this, hopefully we can just people one injection and correct them forever.”
Like many of these advancements and others, CRISPR research is in its very early stages and has a long way to go before we’ll fully grasp its abilities. But, if 2016 has taught us anything, it’s that an awful lot can change in just one year. And with that, we welcome 2017 and can’t wait to see what it has in store!