Sean M. Healey & AMG Center for ALS at Mass General awards second annual Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing

The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital awarded the second annual Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing to two outstanding teams this year:

 

1. Dr. Clive N. Svendsen, Executive Director, Board of Governors Regenerative Medicine Institute Cedars-Sinai, Regenerative Medicine Institute, and Professor of Biomedical Sciences at Cedars-Sinai Medical Center in Los Angeles, CA, for his team’s work on transplanting progenitor cells producing the growth factor GDNF into the spinal cord of patients with ALS, Dr. Jeffrey Rothstein, Director of the Robert Packard Center for ALS Research and Professor of Neurology at Johns Hopkins, and the Answer ALS Team. Answer ALS is a global project dedicated to developing and implementing a unified strategy to stop ALS. The team aims to achieve change through unifying the global community toward agreed upon goals in research, science, technology and education.
2. Dr. Clotilde Lagier-Tourenne of MGH and Harvard Medical School, Dr. Don Cleveland of the University of California San Diego (UCSD), Dr. Jone Lopez-Erauskin of UCSD, Dr. Zevik Melamed of UCSD, and Dr. Kevin Eggan of BioMarin for their work on identifying Stathmin-2 as a therapeutic target for ALS. The team identified that the mRNA encoding stathmin-2 is the most affected mRNA following reduction of TDP-43 function. Their findings suggest that stathmin-2 has high therapeutic relevance for the majority of ALS including sporadic and familial ALS cases and offers the possibility of a novel clinically feasible therapy.

The award was virtually presented to the team during the virtual 33rd International Symposium on ALS/MND by Merit Cudkowicz, MD, MSc Director of the Healey & AMG Center at Mass General and Drs. Ayeez and Shelena Lalji.

The review committee consisting of clinicians and scientists selected both recipients of the 2022 award based on their excellence in transformative scientific discoveries focused on the repair and regeneration of neurological function in ALS.

“Both teams have exemplified outstanding abilities to transform ALS treatment and care” said Cudkowicz, who is also the Chief of Neurology at MGH. "With the innovation and research coming from these teams, I am certain they will continue to make strides in the fight against ALS and make an enormous impact on the ALS community." 

“We would like to thank the Lalji family award for recognizing the team who developed and delivered a new type of combined stem cell and gene therapy approach to the clinic for patients with ALS” said Dr. Svendsen”.  “We are also very happy to have helped generate stem cells from ALS patients as part of Answer ALS”

“The entire Answer ALS team is deeply grateful for the recognition of the Lalji family award.  We are especially thankful for the wonderful participation of the ALS patient community in helping to build this new foundation of research tools and data.  We are especially hopeful that this massive set of freely and openly accessible patient data and the living cell lines from our patients will one day aid in finding a truly effective therapy for sporadic and familial ALS,” said Dr. Rothstein of the Answer ALS team.

“This project started by converting skin cells (fibroblasts) of ALS patients into neurons to model “disease in a dish” - and now we have nearly completed that circle, moving back to patients with a therapeutic agent,” said Dr. Melamed of the Stathmin 2 team. “This research was possible thanks to the generosity of ALS patients willing to participate in research, and the fantastic research teams and leadership, carrying the main goal to uncover critical mechanisms leading to motor neuron loss in ALS, all while staying mindful that we need to convert those findings into cures. The short time between the initial discovery and the demonstration of therapeutic efficacy in vivo demonstrates that sincere intention. This is such a great honor to receiving this award!”

“Receiving this recognition is a real honor, and we want to thank the Lalji family and the Healey & AMG Center for acknowledging our efforts,” added Dr. Lopez-Erauskin of the Stahmin 2 team.

The Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing is an annual, global prize with the goal of identifying therapies and modalities to regain lost function in people living with ALS. To learn more about this $40,000 USD prize and how to nominate an individual or team for the annual award, click here.