(Press-News.org) Vanderbilt University Medical Center received a $13 million Department of Defense grant to lead a multisite clinical trial that will evaluate repurposed FDA-approved drugs as treatment options for patients with Rett syndrome.
Affecting 1 in 10,000 females at birth, and males even more rarely, Rett syndrome is a rare genetic neurodevelopmental disorder that affects brain development.
“It robs affected individuals of the ability to use their hands or speak and causes problems with mobility, as well as a number of other issues,” said Jeffrey Neul, Annette Schaffer Eskind Professor, director of the Vanderbilt Kennedy Center and the grant’s principal investigator. “People with Rett syndrome require lifelong care for activities of daily living. Although symptom management, therapies and a newly FDA-approved drug can help, there remains a great need to find new therapies to improve the lives of people with Rett syndrome.”
The study is an umbrella trial that will compare three FDA-approved drugs, ketamine, vorinostat and donepezil, to a common placebo. Participants will be randomized equally to one of the four treatment arms for a 16-week treatment to assess safety, tolerability, efficacy and biomarker response.
“The study will evaluate FDA-approved drugs that have preclinical evidence supporting potential clinical benefit,” Neul said. “If any of these drugs show benefit in this trial, further clinical evaluation and ultimately use treating people with Rett syndrome could progress rapidly. Additionally, successful establishment of this clinical trial platform will allow accelerated evaluation of other potential therapies in Rett syndrome.”
Participants will be recruited from four trial sites, including VUMC, Children’s Hospital of Philadelphia, Boston Children’s Hospital and Children’s Hospital of Colorado, all International Rett Syndrome Foundation (IRSF)-designated Rett Syndrome Centers of Excellence. Coordination of the trial will be led by VUMC, which has led a number of industry-sponsored and investigator-initiated clinical trials for Rett syndrome, including the recent successful phase 3 trial that led to the first FDA-approved drug for this condition.
“Vanderbilt has a long history of providing best-in-class care to Rett syndrome patients as one of only 18 Rett clinics across the country that IRSF designated as a Center of Excellence,” said Melissa Kennedy, chief executive officer of IRSF. “Dr. Neul and the Vanderbilt team have proven themselves as research leaders as well, conducting clinical research and coordinating trials that have not only expanded our understanding of the fundamental science behind Rett syndrome but also played a pivotal role in delivering the first-ever FDA-approved treatment.”
According to Kennedy, more approved treatments for individuals with Rett means they can “do more and suffer less” as soon as possible.
“Rett is such a complex disorder that no one treatment is going to be right for every individual. We need multiple options to have an impact on every individual with Rett syndrome. By testing compounds that are already FDA-approved and show promise for application in Rett patients, this study can help get treatments into the hands of families much sooner than a compound just entering the pipeline,” she said.
According to Neul, the drugs being tested in this study may uncover benefits for other neurological and mental health disorders. This includes building the safety profile of ketamine in the pediatric population, which is being studied separately for the treatment of depression, post-traumatic stress disorder and other conditions in adults.
END
Multisite clinical trial will compare three FDA-approved drugs for Rett syndrome treatment
2024-01-16
ELSE PRESS RELEASES FROM THIS DATE:
St. Jude Home Care, LLC is first US pediatric home health agency to earn new category of industry certification
2024-01-16
St. Jude Children’s Research Hospital announces today that St. Jude Home Care LLC, a home health agency for the hospital’s patients, earned dual certifications in both pediatrics and home health from Community Health Accreditation Partners (CHAP), an independent, non-profit, accrediting body for home and community-based healthcare organizations. St. Jude Home Care LLC is the nation’s first agency to achieve that distinction. CHAP is the only organization in the U.S. that grants a discrete pediatric certification ...
Study pinpoints breast cancer ‘cells-of-origin’ in high-risk women
2024-01-16
Australian scientists have pinpointed likely ‘cells-of-origin’, the source cells that can grow into breast cancer, in women carrying a faulty BRCA2 gene who are at high risk of developing the disease.
The WEHI-led study also showed these cells have potential to be targeted with an existing cancer drug to delay tumour growth, in findings that may lead to future preventive treatments for the disease.
At a glance
Women with faulty BRCA2 genes are at a substantially higher risk of developing breast ...
Supports help keep Aussie firefighters safe
2024-01-16
House fires, road crashes and emergency rescues – they’re all part of the job for Aussie firefighters. And in such physically demanding roles, maintaining a high level of fitness and movement quality is essential.
Now, new research from health and fitness experts at the University of South Australia shows that professional firefighters have reduced movement quality as they age, which could put them at greater risk of injury.
Conducted by UniSA masters researcher, Alex Redshaw, in partnership with the South Australian Metropolitan Fire Service (MFS), the findings indicate that firefighters over the age of 50 generally have lower movement ...
Study: New deepfake detector designed to be less biased
2024-01-16
BUFFALO, N.Y. — The image spoke for itself.
University at Buffalo computer scientist and deepfake expert Siwei Lyu created a photo collage out of the hundreds of faces that his detection algorithms had incorrectly classified as fake — and the new composition clearly had a predominantly darker skin tone.
“A detection algorithm’s accuracy should be statistically independent from factors like race,” Lyu says, “but obviously many existing algorithms, including our own, inherit a bias.”
Lyu, PhD, co-director of the UB Center for Information Integrity, and his team have now developed what they believe are the ...
Researchers find that using patients’ own blood, rather than saline, helps preserve veins in coronary bypass grafts
2024-01-16
In a collaboration between the Fralin Biomedical Research Institute at VTC and Carilion Clinic, researchers learned that by preserving large superficial leg veins intended for coronary bypass grafting in a mixture of the anticoagulant heparin and blood, rather than heparin and saline, the veins were better protected from cell and tissue damage.
Their findings, published in January in the Journal of Vascular Research, could inform surgical practices and enhance the long-term success of vein grafts in coronary bypass surgeries.
“Ultimately, we're putting healthier blood vessels ...
What if cows could talk?
2024-01-16
You may not know it, but cows share information every time they burp, moo, and chew that speaks volumes about their health and welfare.
Through the work of researchers in Virginia Tech’s College of Agriculture and Life Sciences, we may soon know more about what cows are “telling” us and be able to use that information to improve their well-being.
James Chen, an animal data sciences researcher and assistant professor in the School of Animal Sciences is using a $650,000 grant from the U.S. Department ...
Allen Fawcett named Director of the Joint Global Change Research Institute
2024-01-16
By Greg Koller
COLLEGE PARK, Md.— Allen Fawcett — an energy expert and economist who has played a leading role in formulating and coordinating U.S. climate policy — is the new director of the Joint Global Change Research Institute.
Fawcett joined the Environmental Protection Agency in 2003 and, since 2012, served as the chief of EPA’s Climate Economics Branch, which advances the science of climate economics to inform policy. From 2010 to 2011, Fawcett took leave from EPA to serve as the deputy associate director for energy ...
Parents more likely to attempt suicide in first years after child’s cancer diagnosis
2024-01-16
Parents who have a child with cancer are more likely to attempt suicide during the first years after diagnosis, according to a new study conducted by Qianwei Liu of Southern Medical University, China, and colleagues, published January 16th in the open access journal PLOS Medicine.
Receiving a cancer diagnosis for a child is an incredibly stressful and distressing experience for parents. These parents, especially mothers, face an increased risk of psychiatric disorders, but little is known about the risk of suicide. In the new study, researchers looked at the number of suicide attempts ...
Energy-starved breast cancer cells consume their surroundings for fuel
2024-01-16
New study from the University of Sheffield identifies a novel mechanism employed by breast cancer cells to survive in the challenging environment within tumours
The findings provide a new insight into a previously unknown mechanism of cancer cell survival and may offer a new target for developing therapies
The research found breast cancer cells take advantage of nutrients in the extracellular matrix in times of nutrient starvation
Energy starved breast cancer cells ingest and consume their surroundings to overcome starvation, a new study has found.
The research, conducted by scientists at the University of Sheffield and published today (Tuesday 16 January ...
How did free wi-fi help unlock Hanoi wet markets’ mysteries?
2024-01-16
Researchers at the Alliance of Bioversity International and CIAT and their collaborators have been working on how to harness the power of the estimated 549 million Wifi hotspots worldwide, resulting in a project that used anonymized data gathered from free Wi-Fi to better understand the impact of COVID-19 on Hanoi’s wet markets during the first stage of the Covid-19 pandemic.
In the paper “Using free Wi-Fi to assess impact of COVID-19 pandemic on traditional wet markets in Hanoi” published in December 2023 in the scientific journal Food Security, the researchers analyzed and interpreted mobile device tracking ...