Sean M. Healey & AMG Center for ALS and Jefferson Hospital Weinberg ALS Center Announce First Participants Dosed in new Expanded Access Program

Philadelphia, PA – The Weinberg ALS Center at Jefferson Hospital in Philadelphia, PA has successfully dosed the first participants in its newly established Expanded Access program. With philanthropic support from Kulicke & Soffa, this EAP program under the direction of Hristelina Ilieva, MD, PhD, Clinical Director of the Weinberg ALS Center, and Piera Pasinelli, PhD, Department of Neuroscience & Jefferson Weinberg ALS Center Director, will help to provide patients in the Philadelphia area with the access to ALS investigational treatment. 

Expanded Access (EA), also referred to as Compassionate Use, is a pathway for patients with a serious and life-threatening disease to access an investigational product (IP) that is not yet approved by the FDA. To read more about EA, visit the FDA's website.

Support from Kulicke & Soffa comes from the TechVsALS campaign founded by Shawn Sarbacker, a tech-industry veteran and person with ALS (PALS). TechVsALS aims to connect with companies, foundations, organizations and individuals to join in sponsoring Expanded Access Protocols nationwide under the direction of Sarbacker and MGH.

“Bringing hope to the ALS community and pushing for treatment options beyond the conventional time frame of the clinical trials is extremely important to our patients, caregivers and members of the Jefferson ALS center team. It is a new chapter for our Center and we are thankful for the opportunity” said Dr. Ilieva.

“We are thrilled that the Weinberg ALS Center has officially begun dosing for this EA program” said Sabrina Paganoni, MD, PhD, physician scientist at the Healey & AMG Center, Co-Director of the Neurological Clinical Research Institute at MGH and a Principal Investigator of NIH-funded expanded access protocols. “We are hopeful that this innovative research approach will lead to more opportunities for people with ALS who are not eligible for traditional clinical trials and will contribute to our growing knowledge about several investigational drugs.”

Jefferson Weinberg ALS Center opened its doors in 2016 and has since become a leading site in ALS treatment and research.

Background on ALS
Amyotrophic lateral sclerosis, ALS, is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in progressive muscle weakness and atrophy. There are currently few FDA therapies approved for treating ALS—riluzole, edaravone (IV and oral formulation), sodium phenylbutyrate/taurursodiol , and tofersen. Dextromethorphan/quinidine is also used for the symptomatic treatment of pseudobulbar affect (PBA) in people with ALS.

About the Sean M. Healey & AMG Center for ALS at Mass General
At the Sean M. Healey & AMG Center for ALS at Mass General, we are on a quest to discover life-saving therapies for all individuals affected by ALS. Launched in November 2018, the Healey Center leverages a global network of scientists, physicians, nurses, caregivers, people with ALS and families working together to accelerate the pace of ALS therapy discovery and development.

Under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, we are reimagining how to develop and test the most effective therapies to treat the disease, identify cures and, ultimately, prevent it.

The key to our success is our tightly integrated research and clinical efforts, encouraging opportunities to bring the challenges our patients face every day into our laboratories, focusing investigations on finding solutions that will make a meaningful difference to our patients without delay. Our collaborative efforts are designing more efficient and effective clinical trials while broadening access to these trials for people with ALS.

About Jefferson Weinberg ALS Center

The Jefferson Weinberg ALS Center (JWALSC) is a patient-centric clinical & research integrated program of the Vickie & Jack Farber Institute for Neuroscience. As an ALSA Certified Treatment Center of Excellence, it combines a multi-disciplinary ALS clinic, 4 basic and translational research laboratories and a clinical research program all working together in a seamless bench-to-bedside approach.

JWALSC was launched six years ago with the goal to build of an all-in-one clinical-research enterprise solely focused on the care and treatment of ALS. Thus, JWALSC is structured around an academic model in which research and clinical programs are developed based on disease, rather than academic specialties, creating a collaborative environment in which members from multiple disciplines/departments work together and include the patients in everything they do.

By involving the community in every step, whether in the clinic or in the research labs, our mission is to help patients live a purposeful life with, and despite, ALS. Life comes with personalized symptomatic treatment in clinic. Purpose comes with involvement and education in research.

The Vickie and Jack Farber Institute for Neuroscience is home to preeminent centers for the diagnosis and treatment of stroke and cerebrovascular diseases, Alzheimer's disease, sleep, brain tumors, epilepsy, Parkinson's disease, multiple sclerosis, headache, ALS, traumatic brain injury, and spine and spinal cord injuries. We treat more than 120,000 outpatients a year and perform more than 6,000 neurosurgical/interventional procedures annually. Advanced treatments include: fractionated stereotactic radiosurgery, mechanical and pharmaceutical thrombectomy for stroke, minimally invasive cranial base surgery and endoscopic neurosurgery, endovascular treatments for aneurysms and AVMs, complex reconstructive spine and spinal cord injury surgery, and functional neurosurgery including deep brain stimulation and laser ablation for epilepsy and brain tumors.

About TechVsALS
Founded by 30-year tech industry veteran and person with ALS (pALS) Shawn Sarbacker, the TechVsALS fundraising team is reaching out to the innovative and forward-looking "Tech" community. In collaboration with the Mass General Healey ALS Center, we are engaging companies, foundations, organizations, and individuals who might join us in sponsoring ALS Expanded Access Protocol (EAP) programs nationwide to extend, and perhaps save, the lives of those suffering from ALS while accelerating novel treatments and cures.