Richard Bedlack MD, PhD, is the Stewart, Hughes, and Wendt Distinguished Professor of ALS and Director of the ALS Program at Duke University. He’s been treating people living with ALS for 25 years. He’s worked with more than 5,000 patients—less than 1,000 of whom remain living today. Dr. Bedlack believes that funding for expanded access programs (EAPs) under the ACT for ALS reauthorization is more important now than ever. He believes that people living with ALS deserve access to experimental treatments (outside of alternative and off label treatments found on the internet), and that EAPs make the most sense from both a scientific and a humanitarian perspective. 

Dr. Bedlack points out that, while there are more clinical trials offered now than ever, they are also more restrictive and often are targeting subsets of ALS patients. For this reason, many people living with ALS don’t qualify—up to 90% of people living with ALS will not qualify for a clinical trial. 

People living with ALS (pALS) understandably want access to experimental options. Clinical trials often aren’t an option. Outside of clinical trials, there are only two paths to treatment for pALS: EAPs and alternative or ‘off-label’ treatments (such as the ones you can find and buy on the internet).

Alternative and off-label treatments (AOTs) present a host of problems. Information on the internet about these treatments is often inaccurate or incomplete. Many people living with ALS have been harmed by AOTs, Dr. Bedlack says. With more and more people searching the internet for answers and alternatives (in lieu of access to other therapies), the problems with AOTs are worsening, and the stakes are higher than ever. 

“Self-experimentation with internet AOTs is fraught with danger,” Dr. Bedlack says.

Canadian Broadcasting Network reported in June on a Canadian clinic offering ”plasmalogen treatment” (cocktail containing many supplements). The clinic boasted a “100% success rate in stopping progression and in restoring function of people with ALS” but there was no evidence to back this claim up—no presentations at ALS meetings, no scientific publications. All of the claims were backed up by patient testimonials. At least one featured a patient who felt like he was responding for a short time but according to his family later progressed and died from ALS— yet the video of that patient remained on the clinic website as an example of treatment benefit. In the words of one client in touch with several others, “everybody had declined pretty significantly.” The cost per patient for this cocktail of drugs was $70,000-$90,000 USD per treatment. Ultimately, the CBC reported, the treatment failed to deliver what it promised. 

Our current administration has demonstrated that they recognize this problem with lack of access to vetted experimental treatments: Pres. Trump signed into law the “Right to Try” in 2018. This law, both at the federal and state levels, allows terminally ill patients to access experimental drugs that have not yet been fully approved by the FDA. This is intended for patients who have exhausted all other treatment options and are ineligible for clinical trials. The law bypasses the usual FDA approval process for these specific cases.

While there were good intentions behind the Right to Try law, it has not resulted in a significant number of new experimental options for people living with ALS. 

“Prior to 2021 and ACT for ALS, I had no EAP options,” says Dr. Bedlack. “I did the only thing I could do back then, which was to create ALS Untangled.” 

ALS Untangled reviews alternative and off label treatments, with the goal of helping people with ALS make more informed decisions about them. “Prior to EAPs, I saw a lot of pALS experience financial, physical, and psychological harm due to AOTs,” he says.

EAPs offer better experimental options than AOTs, Dr. Bedlack says. Products are vetted in terms of scientific promise and purity. EAPs have safety oversight and monitoring from the Food and Drug Administration (FDA), the Institutional Review Board (IRB), and other investigators.  And some data can be gathered so we can learn how these drugs perform in a real world patient population, which is quite different from a trial population.

“Since the 2021 passing of ACT for ALS, I have been able to steer at least 75 of my patients away from dangerous Internet AOTs and into safer (and more promising) EAPs,” Dr. Bedlack says. 

Dr. Bedlack points out that EAPs are also a way to empower people by allowing them to continue their treatment. Sometimes, a person living with ALS appears to be benefitting from the drug they are getting in the trial.  Unfortunately, once the trial is over, most of the time  they can no longer receive the treatment. EAPs changed that, allowing for at least some patients to continue receiving treatments after the trial period is complete

Before EAPs, “I would have to tell patients that they can’t continue to receive treatment,” he says. “It is truly heartbreaking to have to do that. EAPs now empower pALS to continue the experimental product.”

There are myriad scientific reasons to continue to fund EAPs, as well. “ALS is not the same disease in everyone,” Dr. Bedlack says. By targeting specific subsets of ALS, Dr. Bedlack says that there are some “home-run” treatments (including Tofersen, Jacifusen)—but these are only for patients with specific genetic mutations (mSOD1, mFUS).

“Since we do not know how to subtype non-genetic ALS, it makes sense to try more treatments in more patients,” he says. “Ideally this would happen in clinical trials, but the next best option is in EAPs.” 

EAPs allow drugs to be tested in more people when similar scope trials can’t happen. “Getting more vetted drugs in bodies, trying more safe treatments, and seeing what helps some patients is really the best thing we can be doing now,” he says. What’s more: “we can learn about safety and tolerability in more ‘real world’ populations than trials enroll,” he points out. 

EAPs and the data they collect can also offer insight into the efficacy of a drug. “We now have a biomarker (neurofilament light chain) which can give us a hint as to whether drugs in EAPs might be helping people with ALS,” Dr. Bedlack says. “This information will be important to clinicians who must decide which patients to treat with newly approved drugs, as well as payors who will make coverage rules.” 

While the cost of EAPs can be viewed as a negative, Dr. Bedlack believes that the high cost will not go away if EAPs go away—they will just be moved downstream. “The government will pay for the ways people suffer inevitably” he says. “If EAPs were to go away, the government will pay for off-label medications to treat pALS (some of which may be unsafe or intolerable), and for harm caused by AOTs and resulting treatment needed as a result. So you’re achieving downstream efficiency by incurring the cost of EAPs. You pay the money up front and it saves money down the line.”

“I strongly support continued and even increased funding for EAPs in ALS,” he says. “EAPs offer an experimental option in which products are vetted, there is safety and scientific oversight, and there is an opportunity to learn about safety and tolerability and get a hint of efficacy in more “real world” populations than those in trials.”

“This topic is very meaningful to me,” Dr. Bedlack says. “I try to remind my patients in every way I can that, despite this being a devastating disease, ALS can’t touch the best parts of who you are. And there is hope.”