2018 – ALS Finding a Cure®

ALS Organizations Collaborate to Advance New Therapeutic into Trials

Anne D Pham — Wed, 28 Nov 2018 14:58:23 +0000

ALS Organizations Collaborate to Advance New Therapeutic into Trials

First dose of potential treatment AT-1501 given to humans by Anelixis Therapeutics to determine safety and tolerability

Boston, MA (November 28, 2018) — Several ALS organizations announced on Wednesday that the first participant was successfully given a dose of AT-1501 in the first in-human trial of the potential treatment for ALS. AT-1501 was developed by the ALS Therapy Development Institute (ALS TDI), with support from The ALS Association, ALS ONE, and ALS Finding a Cure®, and is being brought into trials by Anelixis Therapeutics, Inc., a for-profit subsidiary of ALS TDI.

The phase 1 safety trial of AT-1501 is enrolling healthy volunteers and people living with ALS, following the acceptance of an Investigational New Drug (IND) application by the Food & Drug Administration (FDA). A total of 8 people with ALS will receive AT-1501 in this study, with the aim of determining safety and tolerability of the experimental antibody. The trial will also determine the pharmacokinetics of AT-1501, i.e. how the drug moves within the body.

AT-1501 is thought to block a specific immune system target expressed on lymphocytes (white blood cells), called CD40LG, which is known to drive neurodegeneration. Published research by ALS TDI has implicated a disease mechanism in the disease progression of patients with ALS, which may be blocked by the immune modulatory effects of anti-CD40 ligand monoclonal antibody therapy.

“We are excited to work closely with Anelixis to accelerate bringing AT-1501 to people with ALS”, said Dr. Merit Cudkowicz, Director of the Healey Center for ALS at Mass General Hospital, and Chief Medical Officer for ALS Finding a Cure®. “The ALS ONE-supported trial design team members at the Healey Center are already working together with Anelixis to design the next trial to assess longer term safety and efficacy. It is critical to move faster from exciting lab-based scientific discovery to clinical trials in our patients.”

On Wednesday, The ALS Association announced it had awarded an additional $1 million to Anelixis to help fund the trial. “We are proud to partner with Anelixis and other ALS organizations to help advance this promising compound into clinical trials,” said Calaneet Balas, President and CEO of The ALS Association. “Through this new funding, we are continuing our support of novel, early stage potential treatments into human clinical trials.”

“The support from other ALS organizations for this trial has been crucial to its successful launch,” said Steve Perrin, Ph.D. President & CEO of Anelixis Therapeutics. Perrin also serves as the President & CEO of the ALS Therapy Development Institute. “Today’s dosing of the first person with ALS is a significant milestone in determining the potential utility of this approach in battling back this horrific disease.”

If safety is achieved, a next step forward could be a phase IIA safety study to further assess the safety, tolerability and pharmacokinetic profile of AT-1501, along with exploratory biomarker studies for example. However, the company has made no announcements regarding next steps, rather, staying focused on completing enrollment and conducting analysis on data collected through this initial phase 1 study.

About The ALS Association
The ALS Association is the only national nonprofit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The ALS Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.alsa.org.

About ALS Finding a Cure®
ALS Finding a Cure® is dedicated to being a game changer in discovering a cure for this fatal disease. Founded in 2014 by noted philanthropist and Conair Founder and Chairman, Leandro P. Rizzuto, as a tribute to Christie Rizzuto, his daughter-in-law who was diagnosed with ALS in 2009 at the age of 41, and under the leadership of Denis Rizzuto, Peter N. Foss, and a team of renowned ALS researchers, ALS Finding a Cure® is focused on identifying the gaps in the scientific understanding of ALS that are preventing the development of a cure. The organization, a program of The Leandro P. Rizzuto Foundation, collaborates with a wide range of companies, ALS organizations, other disease research organizations, and ALS patients and families to ensure research efforts are non-duplicative, synergistic, and focused on the ultimate goal: finding a cure. For more information about ALS Finding a Cure®, visit https://www.alsfindingacure.org.

About ALS ONE
ALS ONE is a partnership of the top ALS experts from leading Massachusetts institutions, including ALS Therapy Development Institute, UMass Medical School, Massachusetts General Hospital and Compassionate Care ALS. These venerable institutions are combining forces with the goal of finding a treatment or cure for ALS, while improving care now. The approach of ALS ONE is to leverage the expertise from each of the research institutions through the sharing of ideas and responsibilities to advance each of the strongest therapeutic opportunities identified. Through the rapid and efficient exchange of information, this partnership will help eliminate unnecessary duplication and expedite the path to find ALS treatments. As the scientific and clinical efforts to advance new treatments take time and significant funding, ALS ONE is also working to improve care for persons living with ALS by creating more access to clinical trials through education and transportation, as well as providing technology, equipment, and support services. ALS ONE is a non-profit 501c3. For more information, visit www.ALSONE.org.

About the ALS Therapy Development Institute
The ALS Therapy Development Institute (ALS TDI) and its scientists actively discover and develop treatments for ALS. It is the world’s first and largest nonprofit biotech focused 100 percent on ALS research. Led by people with ALS and drug development experts, ALS TDI understands the urgent need to slow and stop this disease. Based in Cambridge, MA, ALS TDI has over 30 full-time, industry trained, drug development experts on staff. ALS TDI is internationally recognized as a leader in optimizing preclinical models of neurodegeneration for clinical translation, and partners with pharmaceutical companies and biotechs around the world. Rated a CharityNavigator.com four-star charity, ALS TDI spends 87% of each dollar raised on research to find an effective treatment and cure. Visit www.als.net.

About Anelixis Therapeutics
ALS TDI created Anelixis Therapeutics in 2012 as a for-profit clinical development subsidiary to develop AT-1501 as a potential treatment for ALS. Anelixis has been successful at raising capital to complete large scale manufacturing, IND-enabling safety and toxicity studies, and regulatory filing for AT-1501. Anelixis was granted Orphan Drug Designation for AT-1501 from the FDA in April 2018 and submitted a successful Investigational New Drug (IND) application to the Food & Drug Administration (FDA); leading to this phase 1 trial being launched. A more comprehensive timeline tracking the development of AT-1501 can be viewed online here.

About ALS
Every 90 minutes, someone is diagnosed with Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease or Motor Neuron Disease (MND). It is a progressive, neurodegenerative disease that causes muscle weakness, difficulty breathing and swallowing, and paralysis while leaving the senses intact. ALS attacks certain cells in the brain and spinal cord needed to keep muscles moving. Most people survive two to five years after their diagnosis, with an estimated 30,000 people in the US and 450,000 worldwide living with the disease. Currently, there is no effective treatment nor cure.

Source: http://web.alsa.org/site/PageNavigator/pr_112818.html

The ALS Association and Prize4Life announce the ALS Assistive Technology Challenge

Anne D Pham — Mon, 18 Jun 2018 15:00:08 +0000

The ALS Association and Prize4Life announce the ALS Assistive Technology Challenge

After the tremendous success of the ALS Ice Bucket Challenge, ALS organizations are in a position to foster the creation of much needed solutions for people with ALS. One such solution is assistive technology for communication, which for people with ALS who struggle with communication or are completely unable to communicate, has the potential to be not just assistive but transformative.

In April 2015, The ALS Association and Prize4Life organized a workshop for representatives of people with ALS and experts in the field of ALS technology to discuss how to foster the development of technologies for people with ALS. This workshop set the stage for the establishment of the ALS Assistive Technology Challenge.

The ALS Assistive Technology Challenge aims to address the most immediate need of ALS patients and accelerate the development of tools that enable communication. These tools should be:

Effective and innovative beyond currently available
Easy to use and adaptive to the diverse and changing needs of individuals with ALS, including those with no limb movement or speech capabilities. Solutions should as much as possible accommodate all disease stages and be continuously usable as the disease progresses. Solutions will be tested on patients in all stages. Software solutions should be adaptable to diverse platforms.
Cost effective with regards to development and affordable for ALS patients (either directly through price or through a reasonable path to becoming integrated into standard care and reimbursement by international insurance organizations).
Accessible to patients as much as possible (i.e., can be used in a wide range of places and situations, portable and easy to manipulate).
Scalable within a reasonable timeline (including regulatory requirements from FDA, FCC and UL).

Preference will be given to solutions that are developed with interfaces in multiple languages.

To best foster the development of such technology, the ALS Assistive Technology Challenge Program is built as a two stage grant:

Phase I: The ALS Association and Prize4Life will solicit grant proposals for the development of communication solutions for ALS according to the guidelines above. Each grant will be for the amount of $50,000. Proposals will be considered by the challenge

committee, composed of technology leaders and patient representatives. Up to four proposals will be selected to receive funding for development of technologies for 9-10 months.

Phase II- At the end of Phase I, prototypes developed by the teams will be comparatively assessed by an expert panel of:

Technology experts to assess the technology
A group of Testers (patients and caregivers) for testing final solutions
Clinicians and communication experts to assess adaptability
Industry and insurance representative to assess market integration

At the end of the Phase II final assessment event, the best performing team as judged by the challenge committee will receive the second phase of funding ($400,000) to further the development of their technological solutions. Prototypes that haven’t received funding in Phase I would also be allowed to participate at this stage.

In that stage we will also look at several parameters regarding patient satisfaction, such as ease of use, user experience and design, effect on battery life, and reliability of use. Testing in patients is highly valuable and encouraged. The organizers will try to assist interested teams in connecting to relevant patients for testing.

Target Audience

The target audience for this grant includes teams currently in the assistive technology field as well as communities that possess the necessary skill set but are not currently working with people with ALS. The rapid growth in technological solutions for communication (e.g., touchscreens and hand-free devices) demonstrates that there is a vast technological potential in both industry and academia that can be tapped to better the lives of people with ALS.

Timeline:

October 8, 2015: Solicitation phase begins (submission through The ALS Association’s submission mechanism).

November 9, 2015: Deadline for abstract proposals for Phase I.

December 14, 2015: The Challenge Committee will issue requests for full proposals to the groups with the most promising proposals.

January 15, 2016: Deadline for full proposals for Phase I (submission through ALSA’s submission mechanism).

March 1, 2016: Announcement of Phase I Winners

March – October 2016– Phase I duration. On October 31, 2016 participants will have to produce 3 functional prototypes.

November 2016: The ALS Assistive Technology Final Event: Prototypes will be tested by the testers and assessed by the judging panel. At the end of the event, Phase II winners will be declared.

Bio-IT World Announces Finalists For 2018 Best Practices Awards

Anne D Pham — Fri, 20 Apr 2018 19:33:13 +0000

April 20, 2018 | The finalists for the 2018 Bio-IT World Best Practices competition were announced today. This year, 21 projects from 30 different organizations were chosen for the final round of competition. Winners will be announced live at the Bio-IT World Conference & Expo on Thursday, May 17, during the plenary keynote session.

Since 2003, the Best Practices Awards have highlighted outstanding examples of how technology innovations and strategic initiatives can be powerful forces for change in the life sciences, from basic biomedical research to drug development and beyond.

This year’s competition showcased an exciting class of entrepreneurs and innovators, from the clinic, lab, and board room, who have developed new approaches to some of the key pain points in research, discovery, and clinical development.

“Every year we set out to recognize the work being done in the field,” said Bio-IT World’s Editorial Director, Allison Proffitt. “We in the Bio-IT World community take pride in promoting collaborative excellence, and the finalists showcased here demonstrate that dedication as well.”

2018 Bio-IT World Best Practices Awards Finalists

Alexion Pharmaceuticals nominated by EPAM Systems

SmartPanel: A Rare Genetic Disease Diagnosis Algorithm Competition Platform

Amgen nominated by Thermo Fisher Scientific

Integrated Biologics LIMS Enabling High Throughput Drug Discovery

AstraZeneca nominated by Genedata

Deep Learning for Automated Phenotypic Image Analysis

AstraZeneca nominated by SolveBio

Vertically Integrated Translational Oncology: How AstraZeneca leverages expert in-house knowledge and micro-apps for actionable NGS results.

Baylor College of Medicine nominated by CLIRINX – Clinical Research IT

Electronic Data Collection and Artificial Intelligence in Epilepsy Research

The Human Genome Sequencing Center at Baylor College of Medicine nominated by DNAnexus

More Variants for Less Compute: Unified Structural Variant Discovery Scaled to 22,000 Whole Genomes

Celgene

Celgene Laboratory Instrument Mobile Alert

Children’s Hospital of Philadelpha nominated by Edico

Children’s Hospital of Philadelphia, Edico Genome and AWS Achieve Fastest-Ever Analysis Of 1,000 Genomes

DrugDev

The DrugDev Spark Site Engagement Platform

Glaxo-Smith Kline nominated by Science Exchange

Procurement as Innovative Driver for Glaxo-Smith Kline Consumer Health

Hitachi nominated by EPAM Systems

Fast Distributed Optical Maps Assembler for Large Genomes

ICON

FIRECREST Pre-Screen

The Jackson Laboratory for Genomic Medicine

The Jackson Laboratory Clinical Knowledgebase (JAX-CKB)

Center for Innovation and BioInformatics, Neurological Clinical Research Institute, Massachusetts General Hospital

NeuroBANK Patient-Centric Platform for Clinical Research

Natera

Signatera (RUO) ctDNA platform for oncology research

Pfizer / SciBite

SciBite-Pfizer ClassifR: An Artificial Intelligence-driven tool for enabling pharmaceutical acquisitions and collaborations

Pistoia Alliance

User Experience for Life Sciences toolkit (UXLS)

Roche Pharmaceutical Research and Early Development nominated by Linguamatics

Artemis – a text mining tool for Chemists

Saama Technologies

Life Science Analytics Cloud

Synchrogenix, a Certara company

Nothing But Net – Synchrogenix’s GlobalSubmit RIM Products Succeed in Reducing Sponsor’s Regulatory Risk

Takeda nominated by Deloitte

Project Platypus

Source: https://www.bio-itworld.com/news/2018/04/20/bio-it-world-announces-finalists-for-2018-best-practices-awards