I've stayed silent...very silent, since the press release on March 1, 2018, from Ionis Pharmaceuticals, Inc.:
The Huntington's community waited patiently for this update since the previous press release on December 11, 2017: Ionis Pharmaceuticals Licenses IONIS-HTT Rx to Partner Following Successful Phase 1/2a Study in Patients with Huntington's Disease
Since March 1, 2018, WeHaveAFace has received many emails and Facebook messages regarding this news/update. Inasmuch, we [as patients and caregivers] have our own questions. On December 13, 2017, I went LIVE on WeHaveAVoice Radio because the BUZZ was utterly overwhelming across all social media platforms. Not only was it my opinion, but it was my responsibility to ask our community to be extremely cautious about this news.
Since then, we waited again for additional information regarding the Ionis/Roche press release. To be quite frank, I sincerley hoped that it would broaden the conversation and bring forward many answers. The March 1st press release offered promise. Sadly, only a portion of the questions and concerns we received were going to be answered.
Actual questions from Huntington's patients and caregivers:
- Did any of the patients who participated in this trial show signs that the disease was slowing?
- What was the CAG of the patients who participated?
- Did they only accept patients with early onset of the HD?
- Did is slow or stop chorea? Depression? Anxiety?
- Will this be available for Juvenile Huntington's disease?
So what do we know?
The initial goal of this study was to examine safety and tolerability of the drug. It was then discovered that the IONIS-HTTRx (RG6042), could lower the mutant huntingtin protein. "Top-line IONIS-HTTRx (RG6042) Phase 1/2 Study Results: Conference Call and Webcast"(Slide 55). Good news, right?
As per EHDN (European Huntington's Disease Network):
"...we must be cautious, and these results certainly do not yet represent evidence of an effective treatment. We think that huntingtin protein levels are reduced in the cerebrospinal fluid because the drug gets to and acts in the brain. But we don’t know how much of the drug reaches and works in the parts of the brain which are most affected in HD, and we don’t know whether the amount of huntingtin protein lowering is sufficient to offer clinical benefit and lead to a real change in HD patients’ lives. We also don’t yet know whether the dosing regimen used so far is the right one, or whether the treatment has been given at the optimal stage of disease. Answering these questions will require further clinical trials that follow more people over a longer time period. These next trials will probably not start until late this year and will take several years to complete."
We also know specifics regarding the participant's CAG. However, after reviewing the "Top-line IONIS-HTTRx (RG6042) Phase 1/2 Study Results: Conference Call and Webcast" slides, many community members were perplexed to see "Early Stage HD" in relation to a CAG upwards of 55 - (slide 48).
More questions funneled through:
"Does this mean that a participant with a CAG of 55 took part in this study? How often do you see or hear of someone with a CAG of 55 in early stage?" - Michelle, Cleveland Ohio.
"How are they defining early stage HD? Were none of the patients showing signs?" - Theresa, Orlando, Florida
"Did any of the patients in the study have chorea? If not, did any of the patients have depression, anxiety, or suicidal thoughts?" - Craig, Atlanta, Georgia.
WeHaveAFace wishes we could obtain the answers to these questions, however until then, we must continue to remain hopeful and cautious.
UPDATE: March 10, 2018:
Following the release of this article, Dr. Ed Wild tweeted the follow: "The data will be presented at the American Academy of Neurology annual meeting in Los Angeles on April 21-27." - ALZForum Networking for a Cure
Hopefully the AAN will address these questions and concerns.
In the article: "How close are we to a cure for Huntington’s?" it underscores the caution that we as patients and caregivers need to accept and truly understand. "And although the excitement about the result was fully justified, Sarah Tabrizi sounds a note of caution: it will take time to move things forward. “We expect that planning for this study will take about a year, and then it will take several years to enrol and complete. In these studies, if the drug provides clinical benefit in people with Huntington’s disease while maintaining a favourable safety profile, Roche will apply for marketing approval.” It is this, ultimately, that will decide whether a safe and effective treatment has finally arrived."
The article also brought forward criticism from Dr. Ed Wild (University College London, and co-founder of HDBuzz. “...there’s a problem of hype in HD press releases”. For the leading therapy, the Ionis drug, this has meant underlining the necessary timescale for the crucial phase III trial: three to four years is likely, plus the time it would take to license it if successful, so perhaps five to six years in all."
As per a tweet by Dr. Ed Wild, "I wasn’t referring to Ionis when I mentioned the problem of hype - I think their press releases have been very reasonable and have (unusually) been accompanied by thoughtful statements directly to the HD community."
I completely understand and accept Dr. Wild's two-cents. As a patient, former caregiver, future caregiver (unfortunately), and founder of WeHaveAFace.org, I believe that we deserve much more than, "the hype." Personally, I knew that the community was not going to get the answers to their questions because I knew the nature of the study. However, many patients and caregivers still wish to have direct and outlined answers to their questions. They are not asking Roche [in this case] to "dumb-it-down," but to take time to address their concerns. It is a fact that these questions and concerns are clearly known, let alone elementary. Moreover, when publishing press releases and webinars without addressing these important questions and concerns, inadvertently a company is forgetting the community they claim to serve.
I believe there is hope...I know one day there will be a viable treatment or therapy/cure for Huntington's disease. The aim is to fill trials and build momentum and to get the entire Huntington's community involved, right? If so, a great start would be to join us down here in the trenches.
- James Valvano