Endometriosis, an often excruciating condition in which cells from the lining of the uterus grow outside of the womb, is common: one in 10 women of reproductive age have it. Unfortunately, it’s also hard to diagnose; doctors can only confirm its presence through invasive surgery. 

But Feinstein Institutes researchers are working to change that through ROSE (Research OutSmarts Endometriosis), an ongoing multi-center research study that’s unraveling the mysteries of endometriosis by analyzing menstrual blood. 

Six years ago, researchers Christine Metz, PhD, and Peter Gregersen, MD, had a novel idea. During menstruation, endometrial cells are one of many types shed by the uterus.  Could looking at women’s menstrual blood, or effluent, be a way to identify women with the condition — and learn more about it, too?

Lupus, an autoimmune disease that affects several hundred thousand Americans and can cause chronic pain and organ failure, has no cure. But over the last few decades, new treatments known as biologics have helped overcome the excessive activity of the immune system in lupus patients, allowing them to live healthier, more comfortable lives. At Northwell’s Division of Rheumatology and the Feinstein Institutes for Medical Research, scientists have had a hand in developing some of those game-changing treatments. Now, they’re fine-tuning a new one called litifilimab that researchers say has major potential. 

Litifilimab is a monoclonal antibody — a drug that targets a specific cell type that plays an important role in a disease. In a paper published last fall in the New England Journal of Medicine (NEJM), Feinstein Institutes researchers shared the findings of a randomized, double-blind, placebo-controlled study led by Northwell division of rheumatology chief Richard Furie, MD, sponsored by the drug’s maker Biogen, Inc. The study evaluated the drug’s safety and efficacy in people with systemic lupus erythematosus (SLE).

In the 132-person, Phase II trial, participants were given 450 mg of litifilimab or a placebo in an injection under the skin every four weeks; over 20 weeks, the participants got six injections and an additional dose in week two. Dr. Furie and colleagues tracked the patients for six months and found that participants who received the drug experienced greater improvement in arthritis symptoms compared to the group getting placebo injections.

A related study that looked at litifilimab’s effects on people with lupus of the skin (known as cutaneous lupus erythematosus, CLE) was published in NEJM last July. Patients with CLE who received litifilimab also saw symptom improvement. 

As in all autoimmune disorders, in lupus, the body’s immune system runs on overdrive, turning the body against itself. Different cells and inflammatory pathways contribute to the disease process, including interferons. Interferons are crucial to the body’s immune response against infection — but they’re also pro-inflammatory, and the source of trouble for patients with lupus, an inflammatory disease. Litifilimab works by interrupting interferon production, dampening the inflammatory response that drives lupus symptoms such as joint pain, rashes and more. 

“Patients live with SLE their entire lives and experience chronic inflammation, pain and potentially organ failure,” says Dr. Furie, a professor in the Institute of Molecular Medicine at the Feinstein Institutes. “The results from this trial show that litifilimab blocks the body’s production of harmful inflammatory molecules in people with SLE, easing their joint pain and improving overall disease activity.”  

In a larger, global Phase 3 trial of the drug that is currently underway, researchers will see if they can replicate those results.

As with other biologics, litifilimab has the potential to enhance the quality of patients’ lives. “We’ve come a long way with lupus,” Dr. Furie says. “For patients, the future is definitely brighter.” 

Chemotherapy is a vital tool in the fight against cancer, but it’s hard on patients — and some cancers are resistant to the treatment. So what if a physician could predict whether a patient’s cancer will respond before chemotherapy is even administered?

That’s the premise of an innovative approach to treating pancreatic cancer that’s being tested and refined by researchers at the Feinstein Institutes and Cold Spring Harbor Laboratory.

There are few effective treatments for pancreatic cancer, a disease that is often found late, after it has spread; only 7% of patients survive five years past their diagnosis.

Most patients with this cancer don’t have time to waste trying different therapies — which is where the new approach comes into play.

Organoids are miniature tumors grown in a lab from cell samples drawn from patients’ bodies. David Tuveson, MD, PhD, director of the Cold Spring Harbor Laboratory Cancer Center and the chief scientist for the Lustgarten Foundation, first described their potential for acting as a kind of test kitchen for treatments in 2015. 

Fighting Covid with famotidine

Early in the pandemic, before the advent of Covid-19 vaccines and other key therapeutics, researchers all over the world raced to develop treatments against the novel coronavirus. At the Feinstein Institutes and Cold Spring Harbor Laboratory, scientists turned their sights toward famotidine for Covid, the active ingredient in a common over-the-counter heartburn medication with anti-inflammatory potential. Inflammation fueled Covid symptoms. Might a high dose of famotidine, they wondered, quiet them down?

In a fully remote, randomized outpatient clinical trial of 55 unvaccinated people with mild to moderate Covid, researchers led by Tobias Janowitz, MD, PhD, assistant Professor, Cold Spring Harbor Laboratory and adjunct professor at the Feinstein Institutes, sought to find out. Results of the study were encouraging. Patients who took oral doses of 80mg of famotidine, commonly known by its brand name, Pepcid, three times a day for 14 days experienced more rapid resolution of their symptoms overall. They also experienced greater improvement of 14 out of 16 symptoms, including ease of breathing, chest congestion, cough and abdominal pain.

The study was also boundary-pushing in another way. Making clinical trials more diverse and accessible is a goal that many research institutions share — including Northwell. One of the goals of designing a fully remote famotidine trial was to make it more accessible to underserved populations with less flexible schedules, more limited access to transportation and other hurdles that can impede their participation. Nearly two-thirds of enrollees in the trial were Black, mixed-race or Hispanic.

Participants were mailed home a cellular-activated Apple iPad, Bluetooth-enabled scale and other technology, and were called daily by a research coordinator; several other aspects of study design also aimed at making it easier for participants to take part.

Ultimately the study provided two important pieces of info: High-dose famotidine may be able to speed recovery from mild to moderate Covid-19 — and fully remote trials may be a promising way to promote research accessibility.

“We showed we could take the effort and cost of participating in a trial down by bringing it to patients’ homes,” Dr. Janowitz says. “This study will contribute to shifting the paradigm of how we deliver clinical trials.”

Diabetes is a chronic disease that affects an astounding 422 million people worldwide. To date, blood sugar tests, insulin injections and drugs like metformin that address the symptoms of the disease have been management mainstays. Now, researchers are working on a brand-new treatment approach that uses ultrasound — and could transform the way diabetes is addressed.

For the past six years, GE Research has been honing a novel, non-invasive ultrasound technique to stimulate specific neural pathways within organs associated with disease — including the liver, which plays a critical role in regulating blood sugar.

In 2022, a GE Research-led team that included scientists from the Feinstein Institutes showed that focused ultrasound therapy may be able to prevent or reverse the onset of diabetes.

In the study (supported in part by the federal government’s Defense Advanced Research Projects Agency, DARPA), researchers from GE, the Feinstein Institutes, UCLA Samueli School of Engineering, Yale School of Medicine and Albany Medical College used three different preclinical model systems to study the effects of ultrasound stimulation on blood glucose levels, nerve activity and other key changes at the cellular level.

The results showed that stimulating the livers of hyperglycemic animals with ultrasound stabilized the animals’ blood sugars levels.

“What’s encouraging is that this is noninvasive,” says Sangeeta S. Chavan, PhD, a Feinstein Institutes bioelectronic medicine researcher and one of the study’s senior authors. (Dr. Chavan’s colleague Stavros Zanos, MD, PhD, was also a senior author.) “There’s no need to implant an electrode or device, or give anesthesia.”

“The long lasting treatments for diabetes are still lacking. This research opens up new avenues of using ultrasound stimulation and bioelectronic medicine,” Dr. Chavan adds.

“There’s a possibility that we can develop this as a therapy for diabetes and other metabolic diseases that affect millions worldwide. This exciting research is a major step forward.”