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PRO-ACT Wants You!
May 10, 2013
This past December, Prize4Life and the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital launched the PRO-ACT database (www.alsdatabase.org), which consists of over 8,500 ALS patient records from 17 completed Phase II and Phase III clinical trials conducted by companies including Sanofi, Novartis and Regeneron. In April, Prize4Life and NCRI were awarded the 2013 Best Practices Award in the Clinical & Health IT category from Bio-IT World for PRO-ACT. Click here to read about how merging multiple datasets from different clinical trials in PRO-ACT can be used to identify ways to "streamline clinical trials.” Also read about Prize4Life’s and NCRI’s plans to expand PRO-ACT, which includes potentially adding the data from the recent Phase III ALS clinical trials. With PRO-ACT continually expanding, there are many outstanding research questions that can now be addressed. Click here to learn about how you can use PRO-ACT to help advance ALS clinical research, and if you have an ALS clinical trial dataset that has not yet been included, PRO-ACT needs your data!


ALS-ETF Partners with Denovo Biomarkers to Identify Patients Who Respond to ALS Drugs
May 10, 2013
The ALS Emergency Treatment Fund (ALS-ETF) helps provide post-Phase II ALS therapies to ALS patients, who might not qualify for clinical trials, through the government’s expanded access programs (EAPs). ALS-ETF just announced that they are teaming up with Denovo Biomarkers to see if Denovo can use their proprietary genomic biomarker discovery technology to identify ALS patient groups that respond to particular ALS drugs. Through the partnership, ALS-ETF will provide biological samples collected from individuals participating in the EAPs to Denovo for the analysis. Being able to identify unique patient-responder groups could transform the current clinical trial process, which is one reason why Prize4Life and NCRI built the PRO-ACT database, to enable a retrospective analysis of potential responders from previous trials. Maybe the data in PRO-ACT may also be a useful resource to Denovo!


NeuroPhage Raises $6.4 Million to Support Pre-IND Development Efforts
May 6, 2013
NeuroPhage Pharmaceuticals just secured $6.4 million to support the pre-IND development of their lead drug candidate, NPT002, as well as to support the continued development of their "second-generation fusion protein” drug candidates. The funds were primarily contributed by Mérieux Développement, with additional contributions from Shire LLC and other undisclosed investors. Dr. Valérie Calenda, Partner at Mérieux Développement, said "We believe the company's transformative approach to addressing protein misfolding could make a major contribution to achieving greatly improved treatments for these disabling conditions that affect millions of people worldwide." NeuroPhage has previously reported positive preclinical results in animal models of Alzheimer’s disease and Parksinson’s disease and the mechanism of action of their drug seems to hold great promise for ALS as well.


Critique of Precision StemCell’s Stem Cell Therapy for ALS
May 6, 2013
Precision StemCell is an "outpatient imaging and image-guided treatment facility” established in 2012 by radiologist Dr. Jason Williams in Gulf Shores, Alabama. Since opening his doors, Dr. Williams has treated 24 patients with his reprogrammed stem cells, including 18 people with ALS. However, Dr. Williams’ stem cell therapy and medical approach isn’t approved by the FDA, nor has it ever been tested in any preclinical animal models, including any animal model of ALS. In February 2013, two individuals from ALS Worldwide visited Dr. Williams’ clinic and subsequently published an astonishing report providing detailed information about the unsanitary facility conditions and the highly questionable therapy and procedure, which could significantly harm patients. Click here to read this informative blog post which urges the FDA to take action against Dr. Williams.


European Medicines Agency Recommends Approval of NUEDEXTA in Europe
April 26, 2013
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended "NUEDEXTA (dextromethorphan hydrobromide and quinidine sulfate) be approved for the treatment of pseudobulbar affect (PBA), irrespective of neurologic cause.” Although this is encouraging news to the European community, the European Commission (EC) will have the final say over whether NUEDEXTA will be approved for use (although the EC usually takes the recommendation of CHMP). NUEDEXTA was approved by the U.S. Food and Drug Administration in October 2010 for PBA, which is a neurologic condition characterized by frequent outbursts of involuntary crying or laughing. PBA occurs in ALS, multiple sclerosis, traumatic brain injury as well as in Alzheimer’s disease and Parkinson’s disease.