ALS

Partnerships between patient groups, industry, government and academia are accelerating the research and development of new treatments for rare diseases, many of which have no effective therapies. As a result of the Food and Drug Administration’s (FDA) Orphan Drug Designation, eligible companies receive incentives for product development such as tax credits for qualified clinical testing and new drugs are reaching patients sooner. Since 1983, more than 400 drugs and biologic products for rare diseases have been developed, far outpacing the number of products that were available some 40 years ago. Amyotrophic lateral sclerosis (ALS) is among the nearly 7,000 rare diseases where very few...
This article appeared online on The Huffington Post blog. Twelve years ago on Friday the 13th, Lorri Carey’s life changed forever. Her neurologist asked during one of her appointments “what do you know about ALS (amyotrophic lateral sclerosis)?” Carey was a healthy and active 37-year-old with a rewarding sales career, supportive husband and two sons when her health had taken a mysterious turn. Carey knew her symptoms matched ALS, but the only thing she remembered about ALS from her research was that it was fatal. And in fact, she was told she only had two to five years to live. “The toughest part was telling my two sons who were eleven and thirteen that I was going to die. When ALS hits a...
Increasing funding for federal health research agencies to respond to health threats, harnessing the power of precision medicine and other cutting-edge research and keeping the needs of the patient front and center, were some of the key points shared by Dr. Robert Califf, Commissioner of the Food and Drug Administration (FDA) and Senator Amy Klobuchar (D-MN) during Research!America’s 27th Annual Meeting of Members held at the Willard Intercontinental Hotel in Washington, D.C., on March 16. “I remain excited about and dedicated to direct involvement with consumers and patients,” said Dr. Califf. He emphasized that the FDA’s mission is not only to protect safety and promote innovation but to...
Dear Research Advocate: Three outstanding speakers addressed Research!America’s annual meeting of members yesterday. Newly confirmed FDA Commissioner Dr. Robert Califf kicked off the meeting, outlining his priorities for the agency. Having known Dr. Califf for some time, I wasn’t at all surprised to hear how determined he is to ensuring FDA engages the patient perspective as it works to improve policies and processes. For example, he underscored how important it is to calibrate risk/benefit calculations to take into account the seriousness of an illness and the availability of treatment options...in other words, how important it is to think like a patient, not for a patient. Bravo! Senator...
Dear Research Advocate: I’m pleased to announce that Senator Amy Klobuchar (D-MN) will be giving remarks at our Annual Meeting on March 16 at noon at the Willard Intercontinental Hotel. We will also be hearing from newly confirmed FDA commissioner Dr. Robert Califf and ALS patient advocate Lorri Carey. I hope you’ll join us at the annual meeting as well as at the Annual Advocacy Awards Dinner that evening. See our ad in Roll Call . Senators Patty Murray (D-WA) and Elizabeth Warren (D-MA) were joined by the eight other democratic members of the Senate HELP Committee today as they introduced the National Biomedical Research Act . This bill would provide a new mandatory funding stream for NIH...
Dear Research Advocate: You have by now heard about the ALS ’€œice bucket’€ challenge (show support for ALS research by dumping a bucket of ice water over your head and/or writing a check for $100 to the ALS Association, then challenge three others to do the same.) Whether viewed as a welcome late-summer distraction from imponderables like conflict in the Middle East, on-going clashes in Ferguson, Mo., or the mounting death toll from Ebola, or, rather, as the emergence of a new kind of advocacy similar to what produced the walks, runs and bike-rides for research that are ubiquitous today, the ’€œice bucket challenge’€ is worthy of attention. I think that public attention to the ’€œice...
Tell the House to Reject the House Majority’€™s Budget Plan In response to President Obama’€™s budget proposal, House Budget Committee Chairman Paul Ryan (R-WI-01) released a budget plan titled, ’€œThe Path to Prosperity.’€ In this 10-year budget, Rep. Ryan proposes drastic cuts to the funding used to support medical progress among other national priorities. If this budget became law, it is a near certainty that our nation would lose its global lead in science and innovation, undermining jobs, sabotaging any progress toward economic stability, and stalling research that is addressing deadly and disabling health threats. Research reduced cancer deaths among children by 2.1% per year from...
Dear Research Advocate, The first presidential debate gave us little to go on regarding research for health. Americans are dying to know more ’€“ many, quite literally dying ’€“ about what either presidential candidate would do to speed up medical progress in the face of Alzheimer’€™s disease, Parkinson’€™s disease, ALS and the host of other disabling and deadly health threats that breed suffering, compromise independence and drive spiraling health care costs. Add to that the pivotal role medical innovation plays in our economy, and Americans absolutely deserve to know whether candidates will champion or shortchange it. All of us must say to candidates: Tell us what you will do, share your...

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Luck shouldn't play a role in why I'm alive.
Laurie MacCaskill, a seven-year pancreatic cancer survivor