Thank you to Larrie & Diane Grenz! - The ALS Association Greater ...

More documents

Recommendations

Info

Research PAGE 10 Proposed PDUFA Agreement: the Right Prescription for People with Lou Gehrig's Disease The ALS Association applauds the proposed agreement for the reauthorization of the Prescription Drug User Fee Act (PDUFA), which includes provisions that would help to encourage and expedite the approval of new treatments for rare diseases like ALS, also known as Lou Gehrig’s Disease. The proposed agreement was released by the Food and Drug Administration (FDA) on September 1. PDUFA authorizes the FDA to collect fees from companies that develop drugs and biologic products. These fees help the agency cover the costs of drug reviews and currently account for about half of the agency’s drug review costs. The law originally was established in 1992 and has been reauthorized every five years since that time. The current law expires next year unless it is reauthorized by September 30, 2012. “The reauthorization of PDUFA is critical to discovering a treatment and cure for ALS,” said Jane Gilbert, President and CEO of The ALS Association. “There is no effective treatment for ALS and it is fatal in an average of just two to five years. By providing the agency with the resources it needs to review new treatments and ensure their efficacy and safety, PDUFA helps to make sure that funding limitations do not block innovative treatments from reaching the patients who so urgently need them.” “The additional resources and the establishment of drug review timeframes are important elements of PDUFA,” said Steve Gibson, The ALS Association’s Chief Public Policy Officer. “This proposal is particularly significant because it was developed with the input of the patient community in partnership with the FDA and industry. And it is significant because it includes key provisions that promote the development of new treatments and recognize that the practice of medicine is not a one-size-fits-all proposition, especially when it comes to a disease like Lou Gehrig’s Disease.” Since July 2010, The ALS Association participated in monthly meetings with FDA officials to bring the perspective of people with ALS to the discussion on the reauthorization of PDUFA. Three key provisions advocated by The Association were included in the proposed agreement. They include: Developing Treatments for Rare Diseases: Directs FDA to issue regulatory policies and guidance that encourage the development of drugs to treat rare diseases and to include patients as active participants in this process. Assessing Risk-Benefit: Instructs the agency to develop an objective and qualitative framework for assessing risk and benefit during the drug review process and to ensure patient involvement. The provision takes into account the fact that people with ALS who have no treatment options and who will die in just two to five years have a much different view of risk and benefit than does someone with a treatable or chronic condition. Utilizing Biomarkers in Clinical Trials: The proposal encourages greater use of biomarkers and other markers, or “sign-posts,” to help determine risk and benefit when reviewing new drugs. Biomarkers have the potential to significantly accelerate access to new drugs by helping to determine much sooner whether a treatment works and in which patients. “Congress has authorized PDUFA four times beginning in 1992. But perhaps no other version of the law is as important to people with ALS as PDUFA V,” said Gibson. “Right now people with ALS have no way to defend themselves in the fight against the disease. It will take their lives. But PDUFA V can help them fight back. It promotes new treatments. It speeds access. And it makes sense. We urge Congress to swiftly adopt the PDUFA V proposal in 2012.” The proposed agreement released by the FDA is available on the FDA website here: http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf. The ALS Association is the only non-profit 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 Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure.
Research PAGE 11 Research Indicates TDP-43 and FUS/TLS Work Together Although several new genes have now been linked to ALS including the recent exciting finding of mutations in the gene Ubiquilin-2 linked to familial ALS, exactly how these mutations cause disease remains unclear and is the focus of research in labs world-wide. Two proteins with similar structure and function, TDP43 and FUS, linked to familial ALS with and without frontotemporal dementia are thought to be involved in the disease, either by causing some new toxic property or by a loss of their normal function. To identify the potential mechanism, investigators led by Brian McCabe, Ph.D., assistant professor of pathology & cell biology in the Taub Institute for Research on Alzheimer’s Disease and the Aging Brain and a member of the Center for Motor Neuron Biology and Disease at Columbia University Medical Center used the Drosophila-fly model. They demonstrated that these two proteins work in tandem to support the long-term survival of motor neurons. The findings were published in the September 1, 2011 online edition of the Journal of Clinical Investigation. The investigators demonstrate that the drosophila TDP-43 and FUS/TLS proteins bind to each other, and using the mutant proteins linked to familial-ALS, they show that these proteins work together in a common genetic pathway. They show that FUS/TLS mutant Drosophila can be fully rescued by wild type human FUS/TLS but not familial ALS mutant versions. Finally they show that over expression of FUS/TLS can effectively rescue TDP-43 mutants. Their results argue that loss of a common molecular activity that requires TDP- 43 and FUS/TLS could contribute to ALS, in contrast to current models suggesting toxic accumulation. “The two genes make proteins with similar form and function, which suggested to us that they could work together, and that disruptions of either gene would affect neuronal survival,” said Dr. McCabe, who was awarded his first grant for ALS research from The Association. “I am very grateful to The ALS Association for my first ALS grant on ALS2, which set us off on this path,” added Dr. McCabe. “Understanding how these two proteins cause disease is critical for therapeutic development. Further studies together with this new finding will provide the necessary clues to help intervene and develop appropriate treatments," commented ALS Association Chief Scientist Lucie Bruijn, Ph.D.
Page 1 and 2: Thank you to Larrie & Diane Grenz!
Page 3 and 4: Lend a Hand Page 3 VOLUNTEERS WANTE
Page 5 and 6: Care Services Page 5 This Year’s
Page 7 and 8: Understanding Medi-Care Page 7 Atte
Page 9: Advocacy and Public Policy PAGE 9 A
Page 13 and 14: A Special Thank You to our 2011 Wal
Page 15 and 16: Jim Hall Terry Hall Carol Hall Bria

Thank you to Larrie & Diane Grenz! - The ALS Association Greater ...

Create successful ePaper yourself

Delete template?

Save as template?