Friends and Colleagues -
2020 has been one of the worst years ever. I’ve found it helpful to get through it by searching for ways to help folks or causes through charitable giving.
One such opportunity is to help save my friend Brian Wallach by donating to a cutting edge treatment to defeat his ALS and further demonstrate that ASOs can treat neurological diseases like ALS, Alzheimer’s and Parkinsons.
Brian and I met our freshman year at Yale. He was a dynamo then and has since built an awesome career working for Obama and as an Assistant U.S. Attorney in Chicago
3 years ago, Brian was diagnosed with ALS and was told he had 6-12 months to live; devastating news for anyone but especially a husband and father to two girls under the age of 3. Rather than capitulating, he decided to fight - for his own life and all ALS patients. He founded IAMALS and helped secure over $55m in federal funding for ALS research. He has been battling for 3 years with a variety of treatments, but his disease is progressing and he is running out of time.
Last October, Brian and his wife raised $200k to test dozens of formulations of ASOs on Brian’s actual cells. Shockingly, they identified an ASO that Brian’s cells are responding positively to. Now, they need $1.7m to prove the treatment is safe.
Claim your tax deduction and ask your employer to match. The nonprofit that is hosting the One to All project is the Hopewell Fund. Hopewell’s EIN number is 47-3681860 and address is: 1201 Connecticut Ave NW, Suite 300, Washington DC 20036. If your employer has Hopewell Fund as a listed partner, make sure to specify this is for Project: One to All. Some of the larger companies partner with Benevity, a matching platform. We have a Hopewell Fund One To All page set up on Benevity here
You can find more details about Brian, the treatment, etc. in his wife Sandra’s email below…
Family and friends,
We hope this email finds you all managing to put one foot in front of the other. And sometimes to be ok standing still. We are working hard over here to enjoy daily joy which takes hustle. But doesn’t everything?
We do have some very good news about our hustle with ALS that has come out of these long quiet months of quarantine. And with Brian turning 40 today, our biggest birthday wish is to have your help with this effort.
When we (this is us for those who don’t know) were first diagnosed with ALS 3.5 years ago, we asked what came of the millions invested in ALS. It turns out, a lot of errors. But also some critical success. One of the key discoveries of the major investments of the last 10 years is the power of the use of ASOs (antisense oligonucleotides) for patients with genetic mutations of SOD1 and C9. We were told while those forms of ALS were some of the fastest, most aggressive kinds, they were also likely the first kinds to be cured due to the advances in research that had unearthed this.
Our friend in this fight, Chris Snow has the SOD1 version that moves so fast he lost multiple family members in under a year after diagnosis. As a result of Chris’ treatment with this ASO in a clinical trial, you can see here that Chris is doing pushups and kicking a field goal with barely any physical decline since he was diagnosed 18 months ago. This is the clinical paper that explains how powerful the ASO approach is and the NEJM on how positive the results are from the Phase 2 trial. Chris and other SOD1 patients may be among the first survivors of ALS due to this incredible work with ASOs.
We want to be in the second wave. Over the last three years, we’ve been through a lot of different treatments in an effort to find a way to halt Brian’s progression. As you all know, those treatments had varying levels of success. Rather than continue experimenting, we stepped back and pulled together the top scientists in the field to identify a targeted treatment that could work on Brian and have been working with them for the last year developing this treatment. Specifically, in 2019, two breakthrough research papers showed that “sporadic” ALS patients--those who ALS is not caused by a single gene mutation like SOD1--experience a phenomenon where a protein, TDP-43, moves outside the cell nucleus and when it does, it stops working the way it should. The mislocalization and malfunctioning of TDP-43 causes a cascading loss of function of other critical proteins and RNAs that cause motor neurons to die. These papers and post mortem studies suggested that this occurs in the vast majority of ALS patients and critically identified a target to attack and try to stop or reverse neuronal death. Based on our research with the leading scientists, we believe this is what is happening in Brian today. Moreover, subsequent papers have suggested that this phenomenon occurs in and this same target is relevant to Frontotemporal Dementia, Parkinson’s and beyond.
If we waited for the standard clinical trial process to play out, it could take 5-7 years to develop, screen and test a treatment for Brian based on this discovery and we don’t have that kind of time left. So last October we raised nearly $200,000 and funded a couple of the top labs in the country to test dozens of formulations of ASOs that could get this protein to work again and significantly slow or stop Brian’s ALS, much like the SOD1 ASO is doing for Chris Snow. This partnership has resulted in a battery of tests being run on Brian’s actual cells which have then been discussed on weekly team calls and has enabled us to achieve dramatic results in an expedited timeline. Specifically, this work truncated what would have been a four year process into less than 8 months. Most importantly through it, we’ve identified an ASO that is actually regrowing Brian’s motor neurons in the lab work - something we never thought we’d see.
Now this is where we need your help. We want to be cutting edge but we also want to be cautious and thoughtful. So far, we have tested the treatment in mice and it appears to be not only effective but also safe. However, in order for FDA to approve an N of 1 therapy it has to be tested more extensively at a top CRO - a private company that does rigorous drug safety testing. We have been working with that testing company and they have shared with us what the safety studies will entail and that it will cost $1.7m to do this.
I AM ALS’ patient led legislative advocacy has delivered on $55 million in new federal funding for ALS research. This type of funding helps support dozens of critical research projects, including the breakthrough research papers that identified the work on which this treatment is based. The additional federal funds were critical to get us to this point; however they cannot be directed to a treatment for an individual person.
Right now we need our family and friends - our incredible village - who keeps supporting us every step of the way as we make unlikely things likely. We need your help to raise this $1.7m to fund these safety studies. Today, we are launching One to All - www.onetoall.org (password: brian) - to raise this funding. Over one hundred friends and family members have signed on to be team captains so if you’re able to donate, please join one of their teams! We’ve partnered with the Hopewell Fund so that every dollar donated is tax deductible, and we can accept a wide variety of contributions including but not limited to stocks, donor advised funds, and foundation gifts.
We want you to reach out to your own loved ones for their help. However, we would like to keep this effort off of social media for a few reasons, most notably to continue working easily with the FDA and to avoid distracting from the work of I AM ALS, as this is a separate endeavor.
Thank you for saying yes. Everything starts with yes. We find our way up these mountains because you do say yes. Every time. You hold us up and help us solve the next challenge. Let’s give Brian the most important gift he can have on his 40th birthday - this opportunity to beat ALS.
Sandra & Brian