The Pitt Hopkins Research Foundation is committed to directly funding the most promising research available in the world to help find a treatment and ultimately a cure for Pitt Hopkins syndrome.
We do more than just hold fundraisers and write checks. We are defining the landscape of Pitt Hopkins research. We set the research strategy by proactively identifying and monitoring promising therapeutic areas, seeking out scientific and industry partnerships and working closely with them to advance programs through the drug development pipeline. We believe Pitt Hopkins can be cured, but time matters. A rigorous, coordinated, and agile research agenda will get us there as quickly as possible.
Our Progress: Since our beginning in 2012, we have funded over $4,500,000 in research grants and awards. In just 12 years, scientists discovered not one, but two therapeutics that have reversed the symptoms of PTHS in the mice. We are working hard to bring more drugs and more therapeutics to trial SOON. Since our efforts have shown dramatic symptom reversal in mice, we have attracted attention from university and pharmaceutical scientists, the National Institute of Mental Health (NIMH), and the California Institute for Regenerative Medicine (CIRM). The NIMH and the CIRM have supported our seed grants with $7,619,000 in additional research dollars.
We are the only organization in the world devoted to funding research for PTHS.
Our Future:
Our research focus areas for 2024 include:
- continue to transition from basic research to transformational, applied discoveries
- fund additional clinicians and clinical researchers
- fund the study of clinical phenotyping, outcome measures and potential biomarkers
- fund small molecule drug discovery program targeting the TCF4 pathway
- fund and broaden our gene therapy programs
- fund mRNA research
- fund artificial intelligence platforms to search for possible existing therapeutics
- collaborate with private pharmaceutical companies to assist in clinical trials
- fund additional future clinical trials – our first clinical trial began in 2020
Research into these key programs require funding. WE NEED YOUR HELP! Your tax-deductible gift will give so much more than money – it gives all of our families HOPE! Our ultimate goal is to close our doors for good… by finding a cure!
This research has far reaching effects into the science of memory and learning. Because we already KNOW the gene that causes PTHS, TCF4, we are in an important place to fund meaningful research, research that could possibly give insight to other learning, memory and motor disorders with no known cause, like Autism, Alzheimers, Epilepsy and Parkinsons.
OVER 94% OF ALL FUNDS DONATED GO TO RESEARCH
Our Current Projects
From Lab to Clinic, We are Transforming Lives!
Pioneering Progress: Driving Clinical Trials with Unyielding Passion!
At the PHRF, we don’t just fund research, we fund strategic, cultivated projects that lead to viable and timely treatments in the clinic. Our commitment to launching clinical trials has never wavered, and in 2021, just 8 years after we started the foundation we launched our first-ever clinical trial: Microbiota Material Transfer Therapy. Read more about it below:
Groundbreaking Clinical Trial Advances: FDA Review Underway for Revolutionary Treatment
Microbiota Material Transfer Trial – PI James Adams and Rosy Khajelmik;Arizona State University
In 2021 The Pitt Hopkins Research Foundation launched its first ever clinical trial: Microbiiota Transfer Therapy. The trial finished in 2022 with great success in reducing the severe and debilitating gut issues our children suffer with daily. As of April 2023, this treament is being reviewed by the FDA for possible approval. The fact that we have a succesful treatment to bring to the FDA for approval is unprecedented for such a young and small foundation
In Q4 2023 we hope to launch a second MMT trial, this time using a powder formulation that we hope will make the treatment more accessible for those who cannot swallow pills. Please sign up here ( CREATE LINK TO PARENT EMAIL) to receive the most up-to-date news regarding this impending trial.
Neuren NNZ-2591
Neuren Pharmaceuticals is the innovative company behind Daybue, the first treatment ever approved for Rett Syndrome. When they needed another population to trial their next compound, NNZ-2591 they called on The Pitt Hopkins Research Foundation because of our strong ties to our committed community.
Their drug NNZ-2591 has shown strong efficacy in a mouse model of Pitt Hopkins syndrome and been granted Orphan Drug Designation by both the US FDA and the European Drug Regulator, the EMA. Pre-clinical studies with NNZ-2591 have shown that it appears to normalize the underlying biology of the brain, and suggest that NNZ-2591 may have a favorable effect on the broad features of Pitt Hopkins syndrome, rather than being restricted to treating an isolated symptom or symptoms.
The purpose of the study is to look at the safety, tolerability, and efficacy of NNZ-2591 in the treatment of children with Pitt Hopkins syndrome.
UP NEXT…
Gene Therapy Breakthrough: Unlocking the Future of Healing
Our next focus, in conjunction with Mahzi Therapeutics, is bringing our first-ever gene therapy to trial. The therapy concept was proven in the lab of Dr. Allyson Muotri at UCSD. It was then successfully trialed in newborn mice in the Lab of Dr. Patricia Cogram at the University of Chile. Currently, it is being tested for toxicity in primates in efficacy in older mice.
“We (scientists) don’t know of another group that has accomplished as much as you have in 4-5 years. you’ve accomplished more per patient than any parent organization many of us have dealt with”
Apply for Grants
Our goal is to fund research directly targeted toward finding therapeutic treatments for Pitt Hopkins Syndrome.
Proposals should be no more than five pages and targeted towards 1 year of funding with a maximum award amount of 75K.
Proposals are accepted on a rolling basis. For questions and specific proposal guidelines please email audrey.davidow@gmail.com
All grant recipients, as part of the award contract, will be asked to hold quarterly conference calls with each other in order to promote and encourage real time communication and expedite the discovery process.
The PHRF does not pay any indirect costs associated with grant funding.
OVER 94% OF ALL FUNDS DONATED GO TO RESEARCH
Our board and officers are parents and professionals who work diligently to ensure over 94% of all funds donated go toward research and our mission of finding a cure.
Latest News
PHRF Summer 2024 NewsletterSeptember 1, 2024 - 5:49 pm
PHRF February 2024 NewsletterFebruary 5, 2024 - 12:13 am
Pitt Hopkins September 2023 NewsletterSeptember 1, 2023 - 3:39 pm
Niagara Detention Centre Inaugural PHRF Hockey TournamentJanuary 19, 2023 - 7:06 pm
