Robert Packard Center for ALS Research at Johns Hopkins

06/10/2026

Failure in science isn't the end — it's data. In a new Nature spotlight, Packard Center Founder and Executive Director, Dr. Jeffrey Rothstein reflects on the phase I failure of BIIB078, a promising ALS gene therapy that performed flawlessly in the lab but produced the opposite result in patients. His takeaway: even a trial that doesn't work teaches us something vital — and for Rothstein, that lesson is already driving the next experiment.

Read the full article here:

In clinical trials, there are many times (and ways) a therapeutic can come up short.

06/10/2026

We are pleased to participate in the annual Wyss Institute at Harvard University Retreat today at the David Rubenstein Treehouse. The Wyss Institute is pioneering breakthrough technologies inspired by nature to transform healthcare, sustainability, and robotics. By bringing together scientists, engineers, and entrepreneurs, the Institute accelerates the translation of innovative discoveries from the laboratory into real-world solutions with global impact.

The Packard Center is currently supporting the work of Dr. David Walt at the Wyss Institute through a project titled “The Development of an Ultrasensitive Platform to Profile Cryptic Exons in Biofluids.” This research aims to advance highly sensitive molecular profiling technologies with potential applications in disease detection, diagnosis, and monitoring.

06/05/2026

The Allen Institute announced the launch of a new initiative with $400 million in funding and the ultimate goal of developing gene therapies for neurodegenerative diseases including Alzheimer’s, Parkinson’s, Huntington’s, ALS, and Lewy body dementia.

The Brain HealthAccelerator initiative is a massive global effort aimed at mapping the precise brain cells and circuits vulnerable to neurodegenerative diseases, with ALS as a primary focus from day one.

The strategy is human-first, built for advanced AI, and focused on the shared biological mechanisms that link diseases like ALS, Alzheimer's, and Parkinson's. By understanding the common threads, we can discover smarter, targeted therapies and accelerate discoveries.

Read more:

The Allen Institute is launching a $400 million initiative, the Brain Health Accelerator, to develop gene therapies for neurodegenerative diseases including Alzheimer's, Parkinson's, Huntington's, and ALS. It marks the first time the Seattle research organization, founded by Paul Allen in 2003, has....

06/05/2026

We are excited to be a part of MDA's Wings Over Wall Streetght in New York City. The Packard Center is a long-term partner with MDA and proceeds from tonight’s event benefit the Packard Center’s research efforts. Congratulations to all the award recipients.

06/04/2026

Applications are now open for the in-person ALS Clinical Research Learning Institute® (CRLI), taking place October 26–27, 2026, in Clearwater Beach, Florida.

This two-day program provides education and training for individuals impacted by ALS who are interested in clinical research and therapy development. Graduates will be recognized as NEALS ALS Research Ambassadors and may have opportunities to help shape the future of ALS research.

People living with , caregivers, family members, and genetic carriers are encouraged to apply. Selected participants living with ALS may bring one caregiver or family member, with travel and a two-night hotel stay covered by NEALS (Network of Excellence for ALS - NEALS).

Application Deadline: July 6, 2026
For more information visit: https://ow.ly/g93c50Z7ge4

05/08/2026

05/07/2026

We’re proud to be funding the hashtag – a global challenge accelerating the use of AI in the fight against amyotrophic lateral sclerosis (ALS).

Today, the Prize awarded £2 million to 20 of the world's most promising multidisciplinary teams! Each Discovery Award winner receives £100,000 to identify and validate new drug targets using AI.

The winning teams include over 70 organizations from 12 countries. They will now gain access to the largest and most comprehensive ALS patient dataset of its kind, including the genomic sequences of 9,000 ALS patients.

Congratulations to all the successful teams! Learn more about the winners here: https://bit.ly/4tCWpcH

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The Longitude Prize on ALS is a global challenge to incentivise the use of AI-based approaches to transform drug discovery for ALS.

05/07/2026

An article in the May 6 edition of The New York Times highlights the effectiveness of the new drug Tofersen to treat ALS. The development of tofersen stemmed largely from the early fundamental research by Packard Center investigators and members of the Center’s scientific advisory board Don Cleveland, PhD and Timothy Miller, MD, PhD who made the critical discoveries and model systems necessary to develop tofersen as the candidate therapy.

https://packardcenter.org/in-a-milestone-for-a-l-s-a-treatment-helps-some-patients-improve/

05/02/2026

One down and many more to go! We will be following along on this epic journey and your can too! You can track the ride or make a gift to support those with ALS.

https://www.coast2coast4als.org/the-ride

01/01/2026

Happy New Year from the staff and researchers at the Robert Packard Center.