Hello and welcome back to the Cripple Command Center podcast. My name is Eric Valor and I will be your host for this episode. It’s my podcast so I get to hog up all of the miniscule glory from an obscure blog about a very niche subject. I promise I won’t let it go to the wig. On tap for this episode are: An update on the latest Neuraltus NP001 confirmatory Phase 2 trial, the new Brainstorm Phase 3, and a note about two decades’ worth of data on Oregon’s Death With Dignity Act.
But first, I would like to urge my readers and viewers to go to my friend Patrick O’Brien’s GoFundMe page and donate to his new independent film project. Patrick is a PALS like me, totally quadriplegic and on mechanical ventilation to live. Nevertheless, he continues on with his passion for independent filmmaking. His first project after he was diagnosed and became quadriplegic and vented, “Transfatty Lives”, won the TriBeCa Film Festival Audience Award and is available on Netflix and i-Tunes, among others. It’s amazing and I urge you all to watch it.
Please go, click, and donate. Patrick deserves the support to keep his work going.
Another urgent matter is regarding the ALS community in Puerto Rico. As you know, a severe hurricane devastated the island, effectively removing the communications infrastructure. This is especially dangerous for PALS by making it even more difficult for them to obtain the assistance they need. You have no doubt already heard of some patients dying in care facilities because the generators which powered ventilators ran out of fuel.
There are over 200 PALS in Puerto Rico who need assistance. Team Gleason, of which I am a board member, has committed at least $10,000.00 to this specific relief effort. I urge everybody watching, hearing, or reading this to donate. Your contributions will help us reach as many PALS as possible with supplies and equipment. With assistance from groups like Baton Rouge Emergency Aid Coalition, Cajun Airlift and networks of physicians in Puerto Rico and on the mainland, we will coordinate getting relief directly to those with ALS. Please consider donating to help PALS with little resources and little help on the horizon.
OK, to business. Neuraltus began their second Phase 2 trial of NP001 in February on 2017. The trial was to have 120 participants in 18 sites across North America and the 120th patient will have the final dose in December! Results are expected mid-2018. The previous trial “failed” but a post-hoc analysis found a responder group based on a set of inflammatory markers found in the blood and restricted this trial to patients fitting the responder profile. The results this time are expected to be much better.
Next item is the new Brainstorm Phase 3. This trial is the follow-up to the impressive Phase 2B which concluded in July of 2016. In the July 18, 2016 Update from Hope Now For ALS, HNFA issued its review of the Brainstorm data showed remarkable results in a group of patients classified as “Responders” (ALSFRS-R slope improved 100% after treatment compared to a 3-month lead-in observed slope of decline). “Responders” were almost half of the treated group. Their ALSFRS-R scores were significantly better than the placebo group and the surveilled biomarker candidates showed significant improvement. The Phase 3 is a multiple-dose trial where the Phase 2 was a single-dose. The results are expected to be even better, though ALS has shown itself to be quite elusive to hit when previous “slam dunk” treatments were thrown at it. Stay tuned here and to the HNFA website for news as soon as it’s released.
Now for a subject that is intensely personal and not a recommend topic for dinner conversation . It’s definitely a dating taboo. This subject is the Oregon Death With Dignity Act (DWDA) which was passed in 1997. The law, which went into effect in 1998, has strict restrictions on terminally-ill patients who would then request a prescription to end his/her own life on their own terms rather than the much more unpleasant terms dictated by terminal illnesses.
According to the Medscape article which prompted this segment of the podcast:
“To obtain a DWDA prescription, patients must be adults of sound mind, have Oregon residency status, and have a terminal illness diagnosis. In addition, two physicians must confirm the patient’s diagnosis and prognosis, the patient must be offered hospice care, and the patient must make one witnessed written request and two oral requests at least 15 days apart.”
It’s a very common-sense approach to ensure only those truly with a terminal illness have access to this program and that people requesting it are not in a state of undue stress or confusion. According to the 20 years of data compiled, the program is a total success. This is where we get into danger of ruining Thanksgiving dinner – especially when Drunk Uncle gets his inevitable too many beers and bourbons before the turkey is even out of the oven and resting.
The issue is being discussed in every individual state and on the federal level. There are many points of resistance, including religious groups and people who have had the cultural stigma of suicide ingrained in their lives. But is it really suicide if one ONLY has a medically-verified shortened life of suffering and loss of personal dignity? Isn’t it better to go out while you still have your human dignity, where family and friends can remember you whole and relatively hearty? These are questions which only the individual patients can answer for themselves.
I made my decision because I knew that I could suck the marrow from he bones of technology and life a full life full of triumph and tragedy, love and loss, creation and oblivion. I have done all that rather more intensely in my shortly over a decade of living with ALS than the previous nearly 4 decades of healthy living. But I feel that every citizen of the United States should have the right to make this decision for themselves. Adults of sound mind should be allowed to be the masters of their own lives. We do not have the luxury of any opinion about when, where, how, and by whom we are brought into this world. It’s the ultimate indignity to be denied the ability to choose our own terms of exit after tragic illness sets the date. However, this is an individual’s choice. I invite discussion on the Comments Section following each text transcript on my website.
I think 2018 is going to be a landmark year in the otherwise long and somber history of ALS. The previous clinical trials of Brainstorm’s Nurown and Neuraltus’ NP001 have had results that were much more remarkable than any in ALS history and, although history has been brutal with Phase 3 failures, there’s no reason to believe that these Phase 3s will be any different from their previous results. I believe this even more since sub-groups of PALS have been identified as very likely to respond to treatment, something not done in past clinical trials for other ALS treatments. We now have two “Real Deal” prospective treatment options going for registration (known to people not in the clinical trial business as approval) which should soon provide effective treatment for many PALS, finally extending lives in meaningful quantities. And for the unlucky among us, the Death With Dignity issue has become a national debate. With excellent data such as those coming from Oregon, hopefully it will become a nationally-recognized right for those of us unfortunate to contract a fatal disease to control our own destinies.
But like everything, PALS will need to go out and get these things for themselves. If Neuraltus and Brainstorm do decide to apply for registration, they will need our help by showing our support for approval and demanding FDA follow through. Likewise, we must also forcefully demand that lawmakers recognize our natural-born rights to control our own lives. Again, like how every gain PALS have made came from one or a few of us starting a small movement, with more joining in and staying active until the goal was achieved, we together can always achieve our goals. Not bad for a bunch of people confined to wheelchairs and beds who cannot physically speak. With a little clever use of technology we are still very potent advocates for ourselves.
A parting point is a political one. It has to do with the new Republican tax bill recently introduced in Congress. One particularly problematic issue with the bill, among multiple others, is the elimination of the Orphan Drug Tax Credit (ODTC). According to NORD, the National Organization for Rare Disorders:
“The ODTC allows drug manufacturers to claim a tax credit of 50 percent of the costs of clinical research and testing of orphan drugs. The ODTC is part of a package of provisions enacted in 1983 in the Orphan Drug Act (ODA) that provide incentives for drug companies to develop products for rare diseases. This legislation has been extremely successful, resulting in over 600 therapies for rare diseases coming to market in the last 35 years.
Without the Orphan Drug Tax Credit, approximately 33 percent fewer orphan therapies would have been developed, and 33 percent fewer orphan therapies will be developed going forward if the tax credit is repealed. This means that at least 10 fewer therapies would come to market for rare diseases each year, only exacerbating the stark need for rare disease treatments even further.”
This is a serious issue for developing treatments not only ALS but all other rare diseases. This is simply unacceptable for not just the patient community but all of America. Giving companies incentives to develop treatments for rare diseases creates jobs and innovation. And, when effective treatments are found, they create economic boosts from the sustained new jobs associated with the new treatment’s production and distribution. But primarily the patients could remain healthy and continue fully participating in the local and national economies. The ODTC is not a “loophole” and eliminating it is penny-wise but pound foolish, paying for tax cuts by eliminating excellent investments in the future of America.
Please help stop this by telling Congress that this is unacceptable. The ODTC is one of the only tax credits that actually saves lives. Tell your Senators and Representative to oppose repealing the Orphan Drug Tax Credit, and stand up for the 95 percent of rare disease patients still searching for a treatment.
I would be remiss if I did not remind my viewers, readers, and listeners that open enrollment for health insurance plans administered under the Affordable Care Act (ACA, also colloquially known as Obamacare) runs from November 1st to December 15th. Certain states are open longer – check your state exchange website for the dates you need to know. This is half the length of open enrollment in previous years because the Trump Administration cut it from 3 months to 6 weeks (a month and a half). Trump also decimated the advertising budget, so people needing coverage under the ACA might not be made sufficiently aware of the new shorter schedule. So, remember this year to get your enrollment completed before December 15th. Don’t wait until the last minute – take some time to take care of yourself today. December 15th is the deadline so make sure your enrollment is complete with plenty of time left. Log on to either your state exchange or the national website and make sure you’re covered by the plan of your choice before December 15th.
That’s this episode of the C-to-the-3 podcast on ericvalor.org. I have been and ever will be Eric N. Valor. Until next time, keep breathing easy.