As of June 1, Public Law No: 115-176 makes it legal to treat Huntington's victims with the gene silencing therapy, IONIS-HTTRx. The Huntington's Disease Society of America appears uninterested*.

So I've been searching in vain for any indication that some organization may be attempting to negotiate the terms with Roche Pharmaceuticals, the owner of the IONIS-HTTRx patent, to supply neurologists under the new law.

Said terms would obviously have to involve capitalization of additional production as well as a legal firewall beyond that already provided by PL115-176, and any other measures needed to ensure the efficacy trial goes forward unimpeded if not expedited.

Estimates for HD victims under treatment in the US vary from 30,000 to 500,000 (yes the stats are that bad). There are 600 slots in the upcoming efficacy trials for IONIS-HTTRx.

One thing that occurs to me is that States led the push for PL115-176 and, although this is mitigated by their Federal reimbursements, States suffer a substantial drain on Medicaid resources from HD. Moreover, the risk that IONIS-HTTRx will not be effective is very low compared to most drugs that enter efficacy trials, due to the nature of the disease and the treatment. On the other hand, government does have an incentive to accelerate the deaths of HD patients, so if the treatment is seen as possibly increasing the lifetime burden of those patients, risk-averse bureaucrats may stonewall to avoid the issue and maintain plausible deniability. The incentives for the Federal government aren't much better.

Any suggestions on how one might proceed to set up such an organization?


*Indeed, Nature magazine, on the very day "Right To Try" became law, carried an article about Huntington's Disease gene silencing, which made no mention of the law, with the nearly decade-old headline: "How the gene behind Huntington’s disease could be neutralized: Antisense oligonucleotides are providing researchers and patients with fresh hope of targeting the condition’s genetic cause". "Fresh hope?" More like stale hope, now become virtual certainty with the measured reduction of the mutant protein in the successful safety trial. Weird.



Edited 1 time(s). Last edit at 06/04/2018 05:22PM by jabowery.

I haven't been on this site for awhile. I agree with you that this is very frustrating! This shows so much promise. I understand, though, how the companies have to be careful with their trials. Also the USA is very litigious so it's probably one reason things move so slowly. If I knew this was available to anyone, I would fly overseas to get it. I am eagerly awaiting it's release, but fear it might come too late.

The sole reason RG6042 (IONIS-HTTRx) is not being administered to HD victims in the advanced stage, when degeneration is not only severe but accelerating, is that Roche (Genentech) has a responsibility to protect its stockholders from unethical litigation by the lawyers of patients who, despite having signed liability waivers protecting all parties involved in saving their lives, may nevertheless find spurious grounds to go after those who try to save them -- particularly those with deep pockets. All claims of "safety concerns" in such cases are rendered moot not merely by the just-completed safety trial that was successful, but by the medical ethics of terminal life care.

Since none of the organizations that have a responsibility to take this issue by the horns and wrestle it to the ground are doing so, and since people are going to be killed by this pathological situation, I fully expect there to arise treatment centers in jurisdictions that are outside the patent regime protecting RG6042, and in which there is insufficient legal protection of patients -- protections that might otherwise render doctors averse to accepting a liability waver signed by dying patients. Although this kind of treatment is needlessly endangering patients in accelerating loss of function, it is more compassionate and ethical than is the more "civil" environment of the developed world where, due to public relations issues, the real, underlying cause of HD patients being killed, is explained away as "concern for patient safety".



Edited 1 time(s). Last edit at 07/23/2018 11:01AM by jabowery.

I am impressed, two sentences 242 words.

Excuse me, I am not a victim, I may have HD, but it does not have me! I find your post total rubbish, it has to go through clinical trials first! And then, after successful clinical trials, then this new amendment can be used while waiting fda approval



Edited 1 time(s). Last edit at 08/05/2018 02:58AM by Barb.