Mayuri Saxena first knew something was off when she began tripping at work and struggling to get out of her seat during her NYC subway commute. In just three months the 32-year-old became wheelchair-bound, but eventually she learned what was wrong: She had ALS.
Facing a two- to five-year life expectancy from Amyotrophic Lateral Sclerosis, a degenerative neuro-muscular disease that largely impacts voluntary muscles including vocal cords, Saxena and her family looked to experimental drug trials as their only chance at treating the disease. One by one she was rejected either because of her wheelchair use, or being too far along in her disease.
“That’s honestly when our hope started going down the drain,” said Mayak Saxena, Mayuri’s brother. “To know that a treatment like that exists on this planet and on the other hand to see my sister suffering and dying and in pain. It’s such a huge injustice that has to be remedied on so many fronts.”
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A new bill making its way through Congress could soon offer hope for Saxena and others with ALS who have been unable to make it into clinical trials. The bill, H.R.8662, and its Senate companion aims to give ALS patients early, expanded access to medical treatments in their stage 3 clinical phase. It would speed up access to the potentially life-saving treatments for those whose time is limited. It’s estimated that 30,000 people in the U.S. have ALS and 6,000 die of it annually. There is no current treatment for the disease.
Advocates say that despite the risks associated with using a non-finalized drug, the law would give ALS patients a shot at life where there currently isn’t one. And they are hoping to get the legislation passed before the current Congress clocks out at the end of the year, warning that if the bill is pushed off until next session, more patients in need will die.
“Everybody in the ALS community says, ‘I’m 100 percent going to die. With no options, there’s nothing you can give me that is going to make me die, when I wouldn’t have died. There is a different risk/benefit analysis for me, let me make that choice,’” said Michelle Lorenz of Everything ALS, a patient-focused nonprofit. “We’re going to lose 12,000 more people, if we don’t [pass it this year], by the time we get it reintroduced next session.”
The 2014 Ice Bucket Challenge is largely attributed to raising the national name recognition of ALS, also known as Lou Gehrig’s disease. The viral social media stunt that spurred friends to dare one another to dump buckets of ice cold water on their heads ultimately raised $115 million for one of the major ALS nonprofits.
But today most patients when diagnosed still face few treatment options besides getting their houses in order and a hospice plan underway, a reality that creates what some patients label “ALS defeatism.” And there is still much that is not known about the disease. For 10 percent of patients, including Saxena, the disease is genetic, but for others the cause is unknown. The disease is known to disproportionately impact white men over the age of 60. Some experts believe there’s a link between chemicals found on military bases and elsewhere that explains why so many veterans have been diagnosed with ALS.
Diagnosing patients with ALS is also tricky, and can take years—a major limiting factor for patients who want to get into the handful of experimental ALS drug trials. The companies developing the drugs set strict standards for the ideal test subject to measure outcomes by. Fewer than 10 percent of the ALS population qualifies for a trial.
It’s a reality that can be hard to stomach for ALS patients and families who have no other options.
“It’s infuriating, because it’s like, well, why do these trials even exist? What’s the point of compassionate use if there’s not even any compassion that they’re using,” said Mayak Saxena of his sister’s experience.
It’s led to an increased push by advocates, and a growing sense of anger amongst patients at the limitations imposed by the Federal Drug Administration on drug access.
“ALS has traditionally been a disease where, because of the time scenario and because of the age of people it normally strikes, there’s been no time for protests. There’s been no time to get angry about this disease. And that’s changing,” said Mike Henson, an ALS patient who is part of the online patient advocate group No More Excuses. “Patients have had enough of this crap.”
Henson knows how important access to trial drugs are for those living with ALS. Earlier this year he was lucky enough to partake in the stage 3 trial for the drug NurOwn, and found success. During the several months that he was on the drug, his disease didn’t get any worse.
Yet it’s taken NurOwn 10 years to develop to this point, and it’s still not available on the market. Even Henson can no longer access it now that the trial is over and he said he’s already seen his motor functions begin to drop.
When he first found out he had ALS two years ago at the age of 47, Henson said he went to a “pretty dark place.”
“You don’t live with ALS, you die with ALS,” he said.
Of the drugs, Henson said access is everything.
“We’re already written off. So truly for us, anything we try is, you know, the risk factor is zero, essentially.”
In addition to opening up access for patients logistically, the legislation also authorizes for appropriations of $100 million in funding to cover the cost of the medication for patients so there would be no out-of-pocket fees. Advocates for the federal bill are pushing hard to get it signed into law before the end of this Congress, less than two months away. Any delay means more lost lives.
For some of the members of Congress sponsoring the bill, the passage is also personal. Rep. Jeff Fortenberry (R-NE) lost his brother-in-law to ALS this past April. Sen. Lisa Murkowski (R-AL) also has a cousin-in-law who died of the disease.
So far there are 75 co-sponsors on the House bill and advocates are eagerly waiting for Chairman of the House Energy and Commerce Committee Frank Pallone (D-NJ) to bring it to the committee for a full vote.
Witnessing the slow crawl of the bi-partisan bill has been hard for some to bear, especially as they see COVID-19 drugs like remdesivir being fast-tracked and greenlit for consumer use in a groundbreaking time period.
“I think in the ALS community that’s really angered people, like, ‘Why do you get access to a drug, and I can’t?’” said Lorenz.
Advocates and legislators know that it will be an uphill battle getting the ALS bill passed during a lame duck session coming off of a tumultuous presidential election. They have less than two months to get it to President Trump’s desk.
“We have the logistical challenges of people not being in their offices, you know, but despite that, we still need to push through because we’ve worked so hard to just get where we are right now. And we’re just around six weeks or so from the New Year. So we were really down to the wire,” said Mayak Saxena.
“But, you know, we still hang our hat on hope.”
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