BOSTON- The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Rapa Therapeutics have announced the first participant enrolled in the Expanded Access to RAPA-501 Protocol (EAP). This EAP is sponsored by the National Institutes of Health (NIH) - Neurological Disorders and Stroke (NINDS), as part of the U.S. government’s landmark Accelerating Access to Critical Therapies for ALS Act (ACT for ALS). The first enrolled participant is scheduled to receive RAPA-501, an investigational product from Rapa Therapeutics, a clinical-stage biotech company focused on developing novel cell therapies for ALS and end-stage solid tumors.  The study is led by principal investigators James Berry, MD, MPH, Suma Babu, MPH, MBBS, and Sabrina Paganoni MD, PhD, physician investigators at the Healey & AMG Center for ALS at Massachusetts General Hospital.

“The RAPA-501 program is one of only a handful of ALS targeted cell-therapy programs, and the first ACT for ALS funded EAP of a cell therapy.  The enrollment of the first participant marks an exciting time for this program,” said Dr. Berry. “We are excited to be advancing our understanding of cell therapies in a new population of people living with ALS who would not otherwise have access.”

“ACT for ALS has made it possible to test these innovative investigational drugs and brings us closer to providing answers for people with ALS and their families,” said Dr. Babu. “This program is a testament to the power of collaboration between the scientific and patient communities, and we look forward to expanding access to experimental treatments across the country.”

“We are grateful to the participating centers of the NEALS consortium, the patient community, and the NIH whose unwavering commitment to research has made this milestone possible,” said Dr. Paganoni. “Together, we are forging new paths forward so that more people living with ALS have the opportunity to participate in the research process.” 

"We're thrilled with commencement of patient enrollment in our clinical trial and extend our gratitude to the NIH for their support in increasing access to RAPA-501's groundbreaking cell therapy. Rapa is dedicated to delivering our reprogrammed T stem cell therapy, with a goal to slow, stop or reverse this devastating orphan disease where patients have few to no alternatives," stated Brian Radecki, Rapa’s Chief Executive Officer. "The initiation of patient accrual to the trial marks a significant milestone toward our overarching objective of enhancing respiratory function and extending survival in high-risk ALS patients, who are ineligible for other experimental clinical trials," added Dr. Daniel Fowler, Rapa’s Chief Medical Officer.

RAPA-501 is an epigenetically reprogrammed autologous cell therapy aimed at reducing inflammation and slowing the progression of ALS. RAPA-501 cells are manufactured ex-vivo to attain dual TREG/Th2 anti-inflammatory activity and a T-stem phenotype that permits T cell therapy without conditioning chemotherapy. In the Phase I/II clinical trial of RAPA-501 in people with ALS, RAPA-501 cells were found to be safe (no product-related adverse events), biologically active (diverse anti-inflammatory effects) and showed trends toward slowing pulmonary function decline. The Phase 2/3 component of this continuous, adaptively designed trial is ongoing, thereby setting the stage for more advanced clinical investigations into the potential therapeutic effects of RAPA-501 in ALS and other neurodegenerative diseases.

Expanded Access Protocols (EAPs), also referred to as Compassionate Use, are pathways for people with a serious and life-threatening disease to gain access to investigational products that are part of ongoing clinical trials, but not yet approved by the FDA. The EAP program brings new options to people by coordinating access to experimental drugs and enables the collection of safety and biomarker data in a patient population not studied in clinical trials. Data obtained through EAP implementation can inform the next trial and/or support an accelerated approval for a broader group of people with ALS than those typically included in clinical trials.

This EAP will provide access to RAPA-501 for patients living with ALS (pwALS) who have reduced pulmonary function and are otherwise not eligible for ALS trials. The RAPA-501 trial will provide important safety, biomarker and efficacy data in this high-risk population, which has an estimated 50% rate of respiratory failure or death within 6-months of study entry. The RAPA-501 EAP will be conducted at up to 10 enrolling centers across the U.S., treat patients in an outpatient setting, and employ machine learning, AI-driven “virtual controls” rather than a placebo study arm (collaboration with Dr. David Ennist, CEO at Origent Data Sciences). The RAPA-501 EAP seeks to enroll a diverse population of pwALS and incorporates state-of-the-art remote monitoring during trial implementation (collaboration with Indu Navar, CEO at the citizen-driven research organization, EverythingALS).

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 committed to bringing together a global network of scientists, physicians, nurses, foundations, federal agencies, and people living with ALS, their loved ones, and caregivers to accelerate the pace of ALS therapy discovery and development.

Launched in November 2018, the Healey & AMG Center, under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, is reimagining how to develop and test the most promising therapies to treat the disease, identify cures and ultimately prevent it.

With dozens of active clinical trials and lab-based research studies in progress right now, we are ushering in a new phase of ALS treatment and care. Together, we will find the cures.

About Rapa Therapeutics

Rapa Therapeutics is a clinical-stage T cell immunotherapy company located in Rockville, MD that has developed a Next-Generation Cell Therapy Platform focusing on innovative adoptive T cell products that harness the power of the immune system to create safe, cost-effective, curative T cell therapies. Rapa inhibits the mechanistic target of rapamycin (mTOR) pathway to epigenetically reprogram autologous polyclonal RAPA-201 Th1/Tc1 cell therapy for solid tumors and hybrid RAPA-501 induced TREG/Th2 T-stem cell therapy for the treatment of autoimmune and neurodegenerative diseases (initially, ALS). Rapa’s unwavering mission is to address the high unmet public health needs in these end-stage diseases and to extend the lives of patients so that they can spend more time with their families. Rapa’s clinical trial efforts using RAPA-501 have been partially supported by an award from the ALS Association.