Healey Center adds Jefferson Weinberg ALS Center as clinical enrolling site for Expanded Access Protocol (EAP) companion program to the HEALEY ALS Platform Trial

 

With philanthropic support from Kulicke & Soffa through the TechVSALS campaign, Massachusetts General Hospital and Jefferson Hospital announced a multi-year donation to establish and support the Multicenter Expanded Access Protocol (EAP) Companion Program to the HEALEY ALS Platform trial led by the team from Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. This program will be adding the Jefferson Hospital Weinberg ALS Center as a participating center 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.

The primary objective of this multicenter intermediate EAP led by Merit Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS and chief of the Department of Neurology at MGH, is to provide access to investigational products for participants with ALS who are ineligible for the HEALEY Platform Trial. An IP offered through EA is experimental, so its effectiveness as a treatment for ALS is not yet known. To read more about EA, visit the FDA's website.

“Our ultimate goal is to transform the care for ALS patients into an individualized, likely multi-prong approach able to prevent disease progression sufficiently to allow for a dignified life with limited disability. This program is a significant step forward to achieve our goal,”   said Dr Hristelina Ilieva, Clinical Director of the Weinberg ALS Center.

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

"We are grateful for the opportunity to expand access to new medications in development for ALS patients. At the Weinberg ALS Center, we provide comprehensive care that puts research at the core of what we do. Our mission will be strengthened by this donation, which will allow us to work with and hopefully help more patients in the Philadelphia region," said Dr. Piera Pasinelli, PhD, Director of the Weinberg ALS Center at the Vickie and Jack Farber Institute for Neuroscience.

“This collaboration with Jefferson Hospital will open doors for patients, research, and innovation in ALS care” said Merit Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS and chief of the Department of Neurology at MGH, “We look forward to furthering the success of the EAP program alongside the Jefferson Weinberg Center.”

 For more information on the Expanded Access program at the Healey & AMG Center, Click here Expanding Access to Therapies (massgeneral.org)

 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 three FDA therapies approved specifically for treating ALS symptoms—riluzole, nuedexta and edaravone. But there is no cure.

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.

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, patients and families working together to accelerate the pace of ALS therapy discovery and development.

Under the leadership of Merit Cudkowicz, MD, chief of the Department Neurology, 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 the Northeast ALS Consortium

The Northeast ALS Consortium (NEALS) is an international, independent, non-profit group of 134 research sites around the world that collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. The mission of NEALS is to translate scientific advances into new treatments for people with ALS and motor neuron disease as rapidly as possible. The NEALS member sites are committed to the principles of open scientific communication, peer review and democratic governance of the Consortium’s organization and activities. Governed by an Executive Committee, the Consortium’s research activities are advised by an experienced Scientific Advisory Board. The NEALS Science Advisory Board also regularly guides academic and industry partners on ALS targets, trial design and conduct.

NEALS is dedicated to educating people with ALS about clinical research and therapy development to invite their participation, empowering them to be advocates for ALS clinical research, along with encouraging them to influence and improve the ALS research process.

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.