CHeT News

The University of Rochester Medicine’s Clinical Materials Services Unit (CMSU) announces its partnership with the Sean M. Healey & AMG Center for ALS at Mass General Brigham, serving as the Central Pharmacy Depot in the Healey ALS MyMatch program. CMSU has worked with the Healey & AMG Center on the HEALEY ALS Platform Trial since 2020, and after several years of successful collaboration, CMSU will also work on the Healey & AMG Center’s latest initiative, Healey ALS MyMatch. CMSU will receive active drugs and placebos (IP), processing and blinding each, and send them to investigative sites for dispensation to participants who match with specific drugs based on established biomarkers.

The Healey ALS MyMatch program aims to accelerate the development of new therapies for ALS through a series of early-phase (Phase 1b/2a) clinical trials. These trials assess a range of investigational products, including experimental and repurposed medications, cell-based therapies, gene-targeted therapies, devices, and more. Participants are matched to specific trials based on their genetic and biofluid biomarkers, placing participants at the center of the research process. By prioritizing high-quality biomarker data in early-phase clinical trials, ALS MyMatch paves the way for faster advancements in treatment.

The HEALEY ALS Platform Trial is a Phase 2/3 clinical trial that tests multiple study drugs using a shared platform and a common master protocol. The Platform Trial was developed to accelerate discovery through an established national network of ALS sites, shared resources, shorter startup timelines, and faster enrollment. Since the launch of the Platform Trial in 2020, 8 therapies have been evaluated, over 1,600 people living with ALS have participated, and 2 therapies have moved forward to Phase 3. The Platform Trial currently runs under the Next Generation Design, a refined master protocol that incorporates learnings from the first several regimens and the field. 

Approximately 80 sites across the United States are working together to enroll about 160-300 participants per regimen. The most recent regimen, Regimen I, enrolled 250 participants in under 5 months, greatly accelerating time to results. During the trial, participants have a 75% chance of receiving an active study drug being tested, and a 25% chance of receiving placebo during the 36-week randomized control trial (RCT). The HEALEY ALS Platform Trial will remain active until safe and effective treatments are found for all people living with ALS.

"CMSU is proud to have been selected as the Central Pharmacy Depot for the Healey ALS MyMatch Program and the HEALEY ALS Platform Trial," said Christopher Mark, CMSU’s Senior Director. "We are truly honored to serve as a trusted partner with the Healey & AMG Center for ALS team in advancing ALS research through this innovative platform. This partnership reflects our shared commitment to delivering the highest standards of quality, reliability, and patient-centered care."

 "We are grateful to collaborate with CMSU on our ALS clinical trial programs," says Merit Cudkowicz, MD, MSc, Director of the Healey & AMG Center, Executive Director of the Mass General Brigham Neuroscience Institute, and the Carol and James Herscot Endowed Neuroscience Institute Chair – Executive Director. "Our work together is critical to operating clinical trials, and we have accomplished so much together since partnering in 2020 on the Platform Trial. We look forward to continuing efforts with CSMU on the Platform Trial and ALS MyMatch to find effective treatments for people living with ALS."

The Sean M. Healey & AMG Center for ALS at Mass General Brigham discovers life-saving therapies for approximately 500,000 people worldwide who are affected by amyotrophic lateral sclerosis (ALS). For more information about the Healey & AMG Center for ALS, visit the center’s website and contact HealeyAMGCenterforALS@mgh.harvard.edu.

The Clinical Materials Services Unit (CMSU) at the University of Rochester Medicine provides end-to-end pharmaceutical packaging, labeling, and distribution services to advance clinical research. For more information about the CMSU, visit the unit’s website and contact Christopher Mark, Senior Director, CMSU.

The University of Rochester Medicine’s Clinical Materials Services Unit (CMSU) announces the unit’s partnership with the Keck School of Medicine of the University of Southern California (USC) for two National Institutes of Health (NIH)-funded platform clinical trials: the Alzheimer’s Tau Platform (ATP) Clinical Trial (NIH Grant #: R01AG078457) and the Progressive Supranuclear Palsy Clinical Trial Platform (PTP) (NIH Grant #: R01AG085029). The Keck School of Medicine will act as the Clinical Coordinating Center for both trials, while CMSU will serve as the Central Pharmacy Depot, receiving active drugs and placebos (IP), processing and blinding each, and sending them to investigative sites for dispensation to participants.

The goal of the Alzheimer’s Tau Platform (ATP) is to conduct a randomized, placebo-controlled, Phase 2 platform trial in preclinical-prodromal Alzheimer’s disease that aims to simultaneously test at least two different tau-directed therapies, alone or in combination with an anti-amyloid therapy, to determine safety, tolerability, and biological-based proof of concept based on the tau PET tracer 18F MK6240 and other tau biomarkers. Individuals with elevated amyloid and either normal or mild changes in cognition are eligible to participate.

The Progressive Supranuclear Palsy Clinical Trial Platform (PTP) is a two-year trial (12-month Double-Blind (DB) phase followed by a 12-month open-label extension (OLE)) that aims to test at least three different tau-related or neuroprotective therapies to determine safety, tolerability, and clinical proof of concept for these therapies. Individuals diagnosed with mild-moderate progressive supranuclear palsy, with symptoms presenting for less than 5 years, are eligible to participate.

“CMSU is proud to partner with the Keck School of Medicine of USC on these upcoming platforms to help support drug development for these patients,” said Christopher Mark, Senior Director, CMSU. “CMSU plays an important role to ensure the safe handling and tracking of drug products to support these clinical trials. We are looking forward to contributing to the success of these platforms and hope they yield positive results for the future of patient care.”

“The more that we share our ideas and work together, the faster we are going to make progress toward our common goal: eradicating Alzheimer’s Disease and related neurodegenerative dementias,” said Paul Aisen, M.D., founding director of the USC Epstein Family Alzheimer’s Therapeutic Research Institute.

Both trials have been developed by leading researchers and leverage the experience of the Alzheimer’s Clinical Trials Consortium (ACTC) in trial design and conduct and will be managed by USC’s Epstein Family Alzheimer’s Therapeutic Research Institute (ATRI). The ATP trial will be conducted under the direction of principal investigators Dr. Adam Boxer, MD, PhD, University of California San Francisco, and Dr. Keith Johnson, MD, Harvard Medical School. The PTP trial will be conducted under the direction of principal investigators Dr. Adam Boxer, MD, PhD, Dr. Irene Litvan, MD, University of California San Diego, Dr. Anne-Marie Wills, MD, MPH, Massachusetts General Hospital, and Dr. Julio Rojas Martinez, MD, PhD, University of California San Francisco.

• For more information about the ATP trial, visit the trial’s website and contact ADtau-study@atrihub.io.
• For more information about the PTP trial, visit the trial’s website and contact psp-study@atrihub.io.
• For more information about the Clinical Materials Services Unit, visit the unit’s website and contact Christopher Mark, Senior Director, CMSU (Christopher_Mark@urmc.rochester.edu).
• For more information about the Keck School of Medicine of the University of Southern California, visit the department’s website and contact ATRI-info@atri.usc.edu.

Disclaimer: This research was supported by the National Institutes of Health’s National Institute on Aging, grant(s): R01AG078457 and R01AG085029. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. 

PhotoPharmics, a medical device company developing Celeste^®, an investigational photo-neuromodulation device for Parkinson’s disease, today announced Last Patient Last Visit in its Pivotal, Phase 3 Light for PD clinical trial. The University of Rochester Medicine’s Clinical Trials Coordination Center (CTCC) conducted all participant visits and trial coordination. The completion of all participant activity is a critical step in the Celeste^® program.

The Light for PD trial was a fully remote, randomized, double-blind, sham-controlled trial evaluating Celeste in individuals living with Parkinson’s disease. The trial enrolled 351 participants across the United States. With all participant activity now complete, the company will proceed with database lock and final data analysis.

PhotoPharmics will present topline results from the Light for PD trial to healthcare professionals at the World Parkinson Congress (WPC), taking place May 25–27, 2026, in Phoenix. The presentation is scheduled for May 26 at 7:45 AM in Room 224. The company will also exhibit at Booth #520 throughout the congress.

“This milestone belongs first to the patients — people living with Parkinson’s disease who contributed their time to a trial aimed at advancing new approaches to care,” said Kent Savage, Chief Executive Officer and Co-Founder of PhotoPharmics. “We are grateful to the care partners, investigators, and coordinators who made this program possible and look forward to sharing the trial findings with the clinical community.”

Ray Dorsey, MD, lead investigator and a Professor of Neurology at University of Rochester Medicine,  said, “The Light for PD trial was designed to rigorously evaluate a hypothesis that has been building in the scientific literature for years — that circadian biology and Parkinson’s disease are meaningfully connected, and that a precisely controlled light intervention could have an impact across both motor and non-motor domains. Completing trial visits in a fully remote, 351-person pivotal trial is itself a scientific achievement. We look forward to examining the results of the trial.”

Parkinson’s disease remains a complex, progressive condition with substantial unmet needs — particularly for the persistent motor and non-motor signs and symptoms not fully addressed by existing therapies. Celeste^® is an investigational device designed to deliver precisely tuned light to the eyes to engage melanopsin-containing intrinsically photosensitive retinal ganglion cells (ipRGCs) and associated neural pathways believed to play a role in circadian dysregulation, sleep, and other dysfunction.

The Pivotal, Phase 3 trial adds to prior clinical experience, which informed trial design and supported further evaluation in a larger, controlled setting. The trial was designed to evaluate Celeste^® across a broad population of individuals living with Parkinson’s disease in a real-world, home-use setting — without clinic visits, travel, or modifications to existing medical care.

“The conclusion of participant follow-up represents the culmination of years of scientific work and a major step to completion of an innovative and patient-oriented trial design,” said Karl Kieburtz, MD, MPH, Chief Medical Officer of PhotoPharmics. “We built this trial to be rigorous — remote assessments, double-blinded, and designed to evaluate outcomes across both motor and non-motor measures. We now have the opportunity to analyze a dataset that this trial was designed to generate.”

“This trial reflects a decade of work to better understand how precisely timed, specialized wavelengths of light can engage neural systems relevant to Parkinson’s disease,” said Dan Adams, Chief Science Officer and Co-Founder of PhotoPharmics. “With completion of the trial, we now have a comprehensive dataset to further evaluate photo-neuromodulation as a novel, non-invasive approach under investigation in Parkinson’s disease.”

Celeste^® has received Breakthrough Device designation from the U.S. Food and Drug Administration, a U.S.-specific program with no equivalent in other jurisdictions. Following data readout, PhotoPharmics plans to advance regulatory activities, including a De Novo submission to the FDA.

BROOKLYN, NY – April 9, 2026 – In observance of World Parkinson’s Day, Hispanic Information and Telecommunication Network, Inc. (HITN), the leading Spanish-language public media network in the United States will broadcast The Long Road to Hope: Ending Parkinson’s Disease, a documentary produced by the University of Rochester Center for Health and Technology (CHeT), on Saturday, April 11, at 5:00 PM EDT. The program can also be viewed on demand on HITNGo.

Based on the Parkinson’s Foundation’s estimate of more than 1.1 million people in the United States living with Parkinson’s disease and 2023 U.S. Census data showing around 62 million Hispanics nationwide, an estimated 200,000 Hispanics in the U.S. are currently living with Parkinson’s disease. This number underscores the importance of continuing to grow Spanish-language resources, culturally responsive outreach, and Hispanic representation in Parkinson’s research.

“Teaming up with the University of Rochester Center for Health and Technology to air The Long Road to Hope is exactly the kind of work HITN exists to do. We want to ensure that Hispanics living with Parkinson’s disease in this country have information in their language, with relevant stories and knowledge that reflects our community and that hope — and prevention — are real possibilities. This broadcast is our commitment to being a trusted space with valuable information,” said Erika Vogt-Lowell, Vice President of Content, HITN.

The University of Rochester Center for Health and Technology produced The Long Road to Hope, based on the book Ending Parkinson's Disease, co-written by Dr. Ray Dorsey, a neurologist and leading advocate for raising awareness and advancing the global effort to prevent and ultimately end Parkinson's disease.

"We hope the stories of those in The Long Road to Hope light a way to a world where Parkinson's is increasingly rare. For almost all, Parkinson's is preventable. Two centuries of Parkinson's is enough. Let's be the generation that brings about the fall of this terrible disease," said Dr. Ray Dorsey, coauthor of Ending Parkinson's Disease (endingpd.org) and The Parkinson's Plan (pdplan.org).

What makes a clinical trial truly effective?

Chad Heatwole, MD, MS-CI, director of the Center for Health + Technology at the University of Rochester Medical Center, shares how patient-centered outcome measures are transforming research—moving beyond clinician judgment to what actually impacts daily life. He also discusses how collaboration across teams drives innovation in rare disease and neurological research.

Watch the Video

Read the full article in ISSUU: https://issuu.com/urmc/docs/neuroscience_volume_27_2025/6

The University of Rochester Center for Health + Technology (CHeT) stands as one of academic medicine's most successful experiments in translating science into patient benefit. This article highlights CHeT's work over nearly four decades, playing an instrumental role in bringing landmark treatments to market for Parkinson’s, Huntington’s, and neuromuscular diseases, while pioneering new ways to design, measure, and deliver clinical trials.

Read full press release: https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-fda-breakthrough-therapy-designation

WALTHAM, Mass., June 17, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1). The company also announced an updated plan for obtaining U.S. Accelerated Approval for DYNE-101 in DM1 following a Type C meeting with the FDA and analysis of new long-term functional data.

“After our Type C meeting, we were granted Breakthrough Therapy Designation for DYNE-101 in DM1. We appreciate the FDA’s active engagement and guidance as we advance this promising program through the Accelerated Approval pathway in the U.S.,” said John Cox, president and chief executive officer of Dyne. “Based on feedback from the FDA, along with our 6-month and new 12-month efficacy data, we have submitted a revised protocol for the ongoing Registrational Expansion Cohort of the ACHIEVE trial with vHOT as the primary endpoint for potential Accelerated Approval.”

Accelerated Approval Pathway for DYNE-101 in DM1

• In May 2025, Dyne participated in a Type C meeting with the Center for Drug Evaluation and Research (CDER) at the FDA and discussed the path to regulatory approval, including U.S. Accelerated Approval, for DYNE-101 in DM1.
  • Dyne and FDA agreed that the next step toward Accelerated Approval was to submit for review the revised protocol for the Registrational Expansion Cohort of the ACHIEVE trial with video hand opening time (vHOT) as the primary endpoint, to serve as an intermediate clinical endpoint.
  • In June, Dyne submitted the revised protocol to the FDA.
• Dyne has revised the ongoing Registrational Expansion Cohort in the ACHIEVE trial as follows:

  • The primary endpoint is change from baseline in middle finger myotonia as measured by vHOT at 6 months, compared to placebo.

  • Secondary endpoints include change from baseline in splicing as measured by the composite alternative splicing index (CASI-22), muscle strength as assessed by Quantitative Muscle Testing (QMT), performance on both the 10-Meter Walk/Run Test (10MWR) and 5 Times Sit to Stand Test (5xSTS), and the Myotonic Dystrophy Health Index (MDHI) patient reported outcome measure, all at 6 months compared to placebo.

  • This cohort is expected to enroll 60 participants, randomized 3:1 to receive DYNE-101 6.8 mg/kg Q8W or placebo.

  • Additional clinical trial sites are being added, including sites in the U.S., to support enrollment.

• Dyne intends to use data from the Registrational Expansion Cohort and from the already enrolled patients in the multiple ascending dose (MAD) and ongoing long-term extension portions of the ACHIEVE trial to support a potential submission for Accelerated Approval in the U.S.

Accelerated Approval Milestones for DYNE-101 in DM1

• Dyne plans to complete enrollment in the Registrational Expansion Cohort in Q4 2025.
• Data from this cohort are planned for mid-2026 to support a potential U.S. Accelerated Approval submission in late 2026.
• Dyne plans to initiate a confirmatory Phase 3 clinical trial in Q1 2026.
• Dyne is also pursuing expedited approval pathways globally for DYNE-101.

New Long-term Data from Multiple Ascending Dose (MAD) Portion of ACHIEVE Trial

• Today, Dyne reported new long-term data from adult DM1 patients enrolled in the randomized, placebo-controlled MAD portion of the DYNE-101 ACHIEVE trial, including data from the 6.8 mg/kg Q8W cohort (n=6) at up to 12 months.
• At the registrational dose of 6.8 mg/kg Q8W, DYNE-101 demonstrated robust and sustained improvement in myotonia as measured by vHOT as well as sustained improvements across multiple other endpoints.
• These data support improvement in vHOT as an early indicator of clinical benefit with DYNE-101 in DM1 and its potential as an intermediate clinical endpoint for U.S. Accelerated Approval.
  • As previously disclosed, treatment with DYNE-101 led to an improvement in vHOT of 3.3 seconds as compared to placebo at 6 months.
  • New data demonstrated that mean improvements at 6 months were sustained at 12 months for vHOT, 10MWR, 5xSTS, MDHI and QMT, which demonstrated a 10% improvement in strength at 6 months, increasing to 20% at 12 months relative to baseline.
• Dyne also reported updated safety and tolerability data1 from 56 patients enrolled through the 6.8 mg/kg Q8W cohort of the ACHIEVE trial. DYNE-101 continued to demonstrate a favorable safety profile, and no related serious treatment emergent adverse events have been identified.

San Francisco, California - [August 1, 2024] - Effective treatment options to reduce mortality and prevent long-term morbidity caused by traumatic brain injury (TBI) are lacking in both military and civilian medicine. Over 30 clinical trials for TBI have failed, due in part to inadequate identification of unique patient characteristics for targeted therapy, lack of objective diagnostic and prognostic TBI biomarkers, and imprecise approaches to outcome measurement. This has prompted the call for a paradigm shift in the design of TBI Phase 2 trials. The result is a groundbreaking study just launched at UCSF to evaluate the effectiveness of experimental drug treatments in improving recovery outcomes for TBI patients. This research aims to compare the recovery progress of patients receiving the experimental treatment against a control group administered a placebo.

The aims of the study include:

• The use of FDA-approved oral drugs in an "off-label" capacity, meaning they are not currently approved specifically for TBI treatment.
• The study seeks to determine if these experimental treatments can significantly improve the recovery process for TBI patients.
• Researchers will conduct ongoing assessments to measure changes in recovery throughout the study period, potentially removing or adding drugs in the trial in an adaptive fashion. Adaptive trials are designed to enable changes to the trial after it has begun, without damaging the trial’s validity.

Key features of the protocol:

• Participant Profile: The study will enroll approximately 672 participants presenting at the emergency departments of up to 23 level 1 trauma center sites across the United States, with Glasgow Coma Scale (GCS) scores between 9 and 15 and positive head CT scans (e.g. evidence of bleeding), aiming for 504 completed follow-ups at 6 months.
• Timing: Treatment will begin within 24 hours of injury.
• Study Design: This Phase 2, multi-center, double-blind, placebo-controlled trial will employ an adaptive platform, precision medicine approach under a single multi-arm, multi-stage (MAMS) study framework. This will enable multiple treatments to be assessed simultaneously versus the more traditional “one drug-one trial” design.
• Treatment Interventions: Participants will be randomized to receive one of four treatments: Atorvastatin calcium (cholesterol-lowering drug), Minocycline hydrochloride (an antibiotic), Candesartan cilexetil (a blood pressure medication), or a matching placebo. These treatments have demonstrated benefit in animal models of TBI.
• Comprehensive Assessment includes:
  • The Glasgow Outcome Scale-Extended (GOSE-TBI) is the primary endpoint, and assesses functional impairment or recovery, from Week 2 to Month 3 postinjury; other clinical outcome assessments including a broad range of cognitive function, mental health, social participation, and symptom measurements will be collected as secondary endpoints.
  • Analysis of blood-based biomarker levels at intervals across study duration.
  • Performance of MRI at intervals across study duration.

University of California, San Francisco is the coordinating institution for the study, under the direction of corresponding principal investigator, Geoffrey T. Manley, MD PhD, Chief of Neurosurgery at Zuckerberg San Francisco General Hospital. The study will continue to roll out in the coming year to level 1 trauma centers that comprise the TRACK-TBI NETWORK. In addition to the recruitment sites, the study is also supported by research cores including the University of Pittsburgh Biospecimen Core led by Ava Puccio, PhD; UCSF Imaging Core led by Pratik Mukherjee MD PhD; Statistical Cores at the University of California, San Diego led by Sonia Jain, PhD and the University of Washington led by Nancy Temkin, PhD; and the Outcomes Core led by Joseph T. Giacino, PhD from Spaulding Rehabilitation Hospital and Mike McCrea, PhD and Lin Nelson, PhD at the Medical College of Wisconsin. The University of Rochester Center for Health + Technology (CheT) Clinical Trials Coordination Center (CTCC) led by Cynthia Casaceli MBA, and Melissa Kostrzebski MBA, MS, is performing Contract Research Organization support. This study, and future studies to be carried out under the adaptive platform trial program is funded by the U.S. Army Medical Materiel Development Activity (USAMMDA-Fort Detrick, MD) through the Medical Technology Enterprise Consortium (MTEC). Further support has been contributed by Abbott Laboratories. The trial is registered at https://clinicaltrials.gov/study/NCT05826912.

For more information, please contact: Brian Fabian, TRACK-TBI NET Program Manager, brian.fabian@ucsf.edu