(Press-News.org) $75 million NIH grant could lead to the first effective drugs for a condition with few treatment options
A clinical trial that will test three drugs concurrently, and could include more, represents new hope for patients with progressive supranuclear palsy (PSP), an incurable neurodegenerative disorder that usually kills within seven years after symptoms start.
Researchers hope the trial, which will be led by UC San Francisco and conducted at up to 50 sites nationwide, will lead to the development of new therapies. There are currently no drugs to stall the disease’s deadly progression.
The trial is made possible by a five-year grant of up to $75.4 million from the National Institute on Aging (NIA), which is part of the National Institutes of Health (NIH). It is among the largest grants that UCSF has received in recent years for a neurodegenerative disorder.
“The hope is that the study will transform the type of care that patients with PSP receive,” said Julio Rojas, MD, PhD, of the UCSF Department of Neurology, Memory and Aging Center and the Weill Institute for Neurosciences, and a principal investigator of the trial. “If none of the first three drugs are effective, we will continue trying with other drugs. Even if we slow the progression of the disease by 20%, 30%, that’s a meaningful impact on a condition with relentless progression and no cure.”
PSP is believed to be triggered by a buildup of tau protein that causes brain cells to weaken and die. It is frequently mistaken for Parkinson’s disease. Both conditions are movement disorders, but PSP is far less common, affecting approximately 30,000 Americans, most of whom are in their fifties to seventies.
Richardson’s syndrome is the most common form of PSP. In addition to cognitive difficulties, symptoms include slowness, stiffness, falling backward and difficulty with eye movement, especially looking down.
An innovative study design
The three drugs will be tested using a platform clinical trial model similar to one used for a study of amyotrophic lateral sclerosis (ALS), a neurodegenerative disorder that is much better known and better funded than PSP, although they both affect similar numbers of people. The trial is designed to reduce the time it takes to find effective therapies, decrease the number of participants on placebo and discontinue therapies as soon as they have demonstrated they are ineffective.
“Unlike typical clinical trials, platform trials can remain open with multiple new therapies tested in successive cycles if the first ones don’t work,” said Adam Boxer, MD, PhD, endowed professor in memory and aging at the UCSF Department of Neurology, as well as a principal investigator in the trial. “This means there are more opportunities to identify effective treatments in a faster timeframe, with lower cost and less burden to participants. Patients on the PSP trial will have a 75% chance of treatment with an active drug and after one year, all participants will have the opportunity to receive a drug.”
In addition, data, imaging and biospecimens from the trial will be shared with PSP researchers worldwide, Boxer noted.
Rojas and Boxer, together with principal investigators Irene Litvan, MD, from UC San Diego and Anne-Marie Wills, MD, from Massachusetts General Hospital, along with other experts and patient representatives will help select the first three drugs to be tested in the PSP trial platform.
Enrollment of diverse populations a high priority
The study will focus on enrolling participants who are underrepresented in clinical trials.
“We will build on experience from other community engaged research programs and develop relationships with Spanish-speaking communities,” Rojas said. “These participants would be served by Spanish-speaking clinicians, including a neuropsychologist conducting assessments in Spanish. Similar efforts will be made in the African American and other medically underserved communities.”
Practical and financial barriers may be eliminated by covering transportation and hotel costs, he added.
The nonprofit organization CurePSP will be collaborating with UCSF and other trial sites to recruit participants. According to Kristophe Diaz, PhD, the organization’s executive director and chief science officer, the trial “marks a pivotal step forward” in bringing renewed hope to patients and families.
“Families ask us how they can fight back, but often the most we can offer is guidance on building a support network and assembling a care team,” he said. “This platform is a beacon of hope, bringing us closer to effective treatments and ultimately a cure.”
Enrollment is expected to start in fall 2025. It will be open to patients with Richardson’s syndrome, which affects about 70% of patients with PSP. These patients must have had progressive symptoms for fewer than five years and be accompanied by a care partner.
Funding: This research is supported by the NIH’s National Institute on Aging (R01AG085029). This content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH’s NIA.
About UCSF: The University of California, San Francisco (UCSF) is exclusively focused on the health sciences and is dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. UCSF Health, which serves as UCSF's primary academic medical center, includes top-ranked specialty hospitals and other clinical programs, and has affiliations throughout the Bay Area. UCSF School of Medicine also has a regional campus in Fresno. Learn more at ucsf.edu, or see our Fact Sheet.
###
Follow UCSF
ucsf.edu | Facebook.com/ucsf | YouTube.com/ucsf
END
New hope for progressive supranuclear palsy with innovative trial
2024-09-03
ELSE PRESS RELEASES FROM THIS DATE:
Mass General Brigham Gene and Cell Therapy Institute launches RNA Therapeutics Core
2024-09-03
The Mass General Brigham Gene and Cell Therapy Institute (GCTI) today announced it has launched the RNA Therapeutics Core, a first-of-its-kind, state-of-the-art facility and resource to advance the use of RNA technologies within and beyond the Mass General Brigham research ecosystem. This new Core is dedicated to accelerating the exploration of novel therapeutic targets to effectively translate RNA-based medicines into clinical practice by leveraging advanced RNA vectors and delivery systems.
Until now, a Core of this kind has not existed within an academic setting. With this launch, the RNA Therapeutics Core enables ...
Dangerous airborne fungus boosted by California droughts
2024-09-03
Valley fever is an emerging fungal disease in the western United States that most often causes flu-like symptoms, but can also cause dangerous or even deadly complications. By analyzing data on reported cases of Valley fever in California, which have increased dramatically over the last two decades, researchers from University of California San Diego and University of California, Berkeley, have identified seasonal patterns that could help individuals and public health officials better prepare for future surges in Valley fever cases. The findings also have important implications for how the changing climate can exacerbate the threat of infectious diseases. The findings are published in The ...
$1.8 million NIH grant to FAU engineering fuels quest to decode human evolution
2024-09-03
Natural selection is an important evolutionary force that enables humans to adapt to new environments and fight disease-causing pathogens. However, the unique footprints of natural selection in our genome can be buried beneath those left by other evolutionary forces. Thus, by leveraging information about multiple evolutionary forces, researchers can identify signatures of natural selection in the human genome, and ultimately determine its role in human adaptation and disease.
Low-cost DNA sequencing has ...
Communication helps parent relationships with new college students but has limits
2024-09-03
PULLMAN, Wash. -- When young adults first go off to college, more communication with parents generally leads to better relationships, but parents should avoid always initiating it, according to a study led by Washington State University researchers.
In a paper published in the journal Emerging Adulthood, WSU Assistant Professor Jennifer Duckworth and co-authors found that phone, text, video or in-person communication made first-year students feel better about the relationship with their parents. Students also felt better about the relationship when parents offered support or advice, and when they discussed important topics, such as studying and friendships. However, researchers found ...
Natural selection may create inter-species exploitation
2024-09-03
A modeling study suggests that one-sided interspecies cooperation can spontaneously emerge and persist over time, despite only one species benefitting. Evolutionary game theory, and the prisoner’s dilemma in particular, are often used to model the evolution of cooperation within a single species. In the prisoner’s dilemma, both parties benefit by cooperating, but the greatest benefit is earned by a defector who plays with a cooperator. The temptation to cheat tends to push players towards defection, ...
Targeted cancer therapies: Getting radioactive atoms to accumulate in tumors
2024-09-03
Prostate cancer is the second most common cancer among men worldwide, following lung cancer. In the United States alone, nearly 300,000 new cases are diagnosed annually. While reducing testosterone and other male hormones can be an effective treatment for prostate cancer, this approach becomes ineffective once the disease progresses to metastatic castration-resistant prostate cancer (mCRPC). At this stage, the cancer advances quickly and becomes resistant to conventional hormonal therapies and chemotherapy.
A clever strategy for fighting mCRPC is to exploit the ...
Gigantic asteroid impact shifted the axis of Solar System's biggest moon
2024-09-03
Around 4 billion years ago, an asteroid hit the Jupiter moon Ganymede. Now, a Kobe University researcher realized that the Solar System's biggest moon's axis has shifted as a result of the impact, which confirmed that the asteroid was around 20 times larger than the one that ended the age of the dinosaurs on Earth, and caused one of the biggest impacts with clear traces in the Solar System.
Ganymede is the largest moon in the Solar System, bigger even than the planet Mercury, and is also interesting for the liquid water oceans beneath its icy surface. Like the Earth’s moon, it is tidally locked, meaning that it always shows the ...
Finger wrap uses sweat to provide health monitoring at your fingertips—literally
2024-09-03
A sweat-powered wearable has the potential to make continuous, personalized health monitoring as effortless as wearing a Band-Aid. Engineers at the University of California San Diego have developed an electronic finger wrap that monitors vital chemical levels—such as glucose, vitamins, and even drugs—present in the same fingertip sweat from which it derives its energy.
The advance was published Sept. 3 in Nature Electronics by the research group of Joseph Wang, a professor in the Aiiso Yufeng Li Family Department of Chemical and Nano Engineering ...
Large sharks may be hunting each other – and scientists know because of a swallowed tracking tag
2024-09-03
Who killed the pregnant porbeagle?
In a marine science version of the game Cluedo, researchers from the US have now accused a larger shark, with its deciduous triangular teeth, in the open sea southwest of Bermuda. This scientific whodunnit is published in Frontiers in Marine Science.
“This is the first documented predation event of a porbeagle shark anywhere in the world,” said lead author Dr Brooke Anderson, a former graduate student at Arizona State University.
“In one event, the population not only lost a reproductive female that could contribute to population growth, but it also lost all her developing ...
Can’t stop belching? Dietary habits or disease could be the reason
2024-09-03
Belching is a common bodily function, but when it escalates to a level that interferes with daily life, it is defined as belching disorders. International surveys have reported that approximately 1% of adults have belching disorders, but the percentage in Japan and the factors involved often elude medical professionals.
To examine the relationship between the rate of belching disorders, comorbidities, and lifestyles in Japan, a research team led by Professor Yasuhiro Fujiwara of Osaka Metropolitan University’s Graduate School of Medicine ...