Please take a listen to this important interview with Ashley Cavaliere and her family. Her sister Alex was diagnosed with ALS about 2 years ago. Also joining us is their mom Lorri Cavaliere and advocate Michelle Lorenz.
Please take a moment to listen to the passion both Ashley and her mother have for Alex. We have to fight to get Act for ALS to go through so people with ALS can get access to phase 3 drugs.
Her name may sound familiar to you, as she is the sister of Ashley Cavaliere, long time air talent part of our iHeartRadio family here in CT and beyond.
This is Alex Cavaliere - Gasser, with one of her dogs Sophie, diagnosed with ALS in her twenties. Please contact your legislatures and help us fight for Alex!
Congressional Help for People with ALS
ALS is a 100% fatal disease. Before it slowly kills you, it paralyzes your entire body. There are promising drugs in clinical trials for the first time in the 150 year history of the disease. We need your help to cosponsor and pass the ACT for ALS – S.1813 -- which empowers the FDA’s existing Expanded Access Program (“EAP”), helping 30,000 Americans fight for their lives.
Her ALS Story - click to read more about this inspiring group of women.
App where people can send letters or call their legislators: www.helpmayuri.com
Heightened Risk to our Military
One in six people with ALS is a veteran. In 2008, the VA began to recognize ALS as a service-related disease, caused by exposures to toxins during their service to our country. Overall, the prevalence of ALS among Veterans is approximately 2x the risk of civilians, but that prevalence is on the rise with Post-Gulf War Veterans. A recent study published in Military Medicine revealed a 10x risk to Pilots & Tactical Operations Officers; 6x risk for USAF; and 4x risk for Navy, Coast Guard & Army. It also appears to be striking our Veterans decades earlier than the average age of onset between 55-65. It is not uncommon for Post-Gulf War Veterans to get diagnosed with ALS in their 20s & 30s. CLICK HERE FOR MORE INFO
No causation has been clinically confirmed yet, but hypotheses about the toxic causes of ALS include: (1) VOCs & PAHs from Burn Pits; (2) PFAS from firefighting foam; (3) industrial toxins like TCE; (4) diesel & jet fuel; (5) diesel fueled fires inside tents for warmth; (6) insecticides like permethrin used on military uniforms; (7) concussions; (8) hypoxia; (8) a cyanobacteria called BMAA; and (9) viruses.
In 2007, Brig. Gen. Thomas Mikolajcik testified before Congress asserting that we had a moral obligation to our military men & women who got ALS because of their service. He encouraged Congress to unleash a “MEDICAL ARSENAL” on the enemy of ALS. Thirteen years later, the ACT for ALS would supply that “medical arsenal.”
How Heterogeneity & Rapid Lethality Complicate Trials in ALS
The free market of drug development fails people with rare, heterogenous diseases like ALS. Resultingly, people with ALS have no different outcome today than when Lou Gehrig had ALS in 1939.
As with most rare diseases, ALS trials are complicated by a small population and underfunding. Big Pharma rarely invests in rare diseases as there is little fiscal upside & a significant risk of drugs not being approved. Small pharma can’t fund or enroll large clinical trials, nor pay for Expanded Access. Further shrinking the already small trial population in a rare disease, approximately 90% of people with ALS do not qualify for clinical trials -- less than 5% participate. Thus, for people dying of ALS, the therapies available via EAPs are the only viable disease modifying health care options they have. EAPs further Democratic goals of health care for all.
EAPs also further FDA goals of improving the scientific study of rare diseases. With an average of 100 people in a Phase 3 trial arm, it makes drawing statistical conclusions about efficacy woefully problematic. This is exponentially complicated because ALS is plagued by vast heterogeneity in causation, onset, pathology, phenotype & genotype. For example, approximately 10% of ALS cases are caused by mutations in 40+ monogenic genes & 150+ polygenic genes – some of those genes have dozens of different variants. Each has a different pathophysiological impact & drug target.
The other 90% of ALS cases are sporadic, caused by undetermined numbers of under-funded, under-researched environmental exposures & toxins. The ALS community has identified ALS clusters that are “associated” with EPA Superfund sites, Toxics Release Inventory, industrial & agricultural toxins, heavy metals in water, PFAs, et al. Sometimes that prevalence has been as much as 100x above the national prevalence. Yet, few are investigating these tragic implications from environmental toxins. Because we don’t have data about causation, we don’t know, but suspect, that each lethal toxic combination may have a different pathophysiological impact and thus, a different drug target.
This heterogeneity of ALS makes one-size-fits-all drug development & FDA approval difficult. No one would expect one chemotherapy drug to show efficacy on 200+ types of cancers. And yet, that dichotomy is t