Basel, Switzerland - The Sean M. Healey & AMG Center is thrilled to award the third annual Drs. Ayeez and Shelena Lalji & Family ALS Endowed Award for Innovative Healing to the team responsible for the development of a therapy to target FUS in people living with ALS demonstrating slowing and even reversing the disease course of FUS ALS in some people.

The global team includes:

  1. Neil Shneider, MD, PhD, the Claire Tow Associate Professor of Motor Neuron Disorders at Columbia University and Director of the Eleanor and Lou Gehrig ALS Center, USA
  2. Robert Brown, MD, DPhil, Professor and the Donna M. and Robert J. Manning Chair in Neurosciences, at UMass Memorial Health, USA
  3. Sandrine Da Cruz, PhD, Professor, Department of Neurosciences , at KU Leuven, Belgium, Group Leader at VIB Center for Brain and Disease Research
  4. Paymaan Jafar-Nejad, MD, Executive Director, at Ionis Pharmaceutical, USA
  5. Frank Rigo, PhD, SVP Functional Genomics & Core Research at Ionis Pharmaceuticals, USA

“We would like to thank the Lalji family award and the Healey & AMG Center for recognizing our efforts in discovering, developing and bringing the FUS ASO therapy to the clinic for patients with FUS ALS” the team said in a statement. “We are truly honored to receive this award and deeply thankful to the ALS patients and their families.”

Mutations in the FUS gene, cause an aggressive form of ALS. Preclinical studies using anti-sense oligonucleotide (ASO) demonstrated efficacy and suppression of FUS expression, paving the way for the clinical studies carried out initially by Dr. Shneider to demonstrate the safety of the ASO and, importantly, stabilizing the disease course over weeks in some people. These initial results led to initiation of a global phase 3 trial. Looking forward, these findings will have a broad and major impact on the development of gene modulation therapies for other genetic forms of ALS and other neurodegenerative disorders.

“I am grateful to the Lalji Family and to the Healey & AMG Center for this recognition of our efforts to develop a treatment for this rare and particularly aggressive form of ALS” said Dr. Shneider. “I am proud of the work that we have done at Columbia to move this FUS antisense drug from the laboratory into the clinic, and grateful for the partnership with Ionis Pharmaceuticals that made this possible. I congratulate my co-awardees, and thank them for their critical contributions to this work. We are all so grateful to the courageous people with FUS-ALS and families who have participated in the study of this ASO and helped to advance research for all patients with ALS.”

The award was presented to the team during the 34th International Symposium on ALS/MND in Basal, Switzerland by Merit Cudkowicz, MD, MSc Director of the Healey & AMG Center at Mass General and Dr. Shelena Lalji.

“I would like to thank the Lalji family award and the Healey & AMG Center for this award that I am truly honored to receive together with my colleagues," said Dr. Da Cruz. "A very special thanks to ALS patients and their families.”

The review committee consisting of clinicians, scientists, and individuals living with ALS, unanimously selected the deserving team as recipients of the 2023 award based on their excellence in repair, regeneration, and technology to restore function and improve quality of life for people affected by ALS.

“Drs. Shneider, Brown, Da Cruz, Jafar-Nejad, and Rigo have exemplified outstanding abilities to transform ALS treatment and care” said Cudkowicz, who is also the Chief of Neurology at MGH. “Their ground-breaking discoveries for the treatment of people with FUS ALS opens the door for a new generation of ALS research; I am certain they will continue to make strides in the fight against ALS and bring us closer to a cure.”

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.