Big Idea - Finding a cure at all costs!

[powerpadman]

Here's a wild and crazy question: How can we, human beings, make and test a drug with ZERO interference from anyone? Rare diseases have so much stacked against them because they just don’t have the ability to make shareholders and companies money.

Here is a macro policy “big idea”: the FDA classifies a drug candidate as "preventative," "slows progression," or "curative." For curative medicines and therapies, those get a 7-day turnaround response from the FDA and the government pays out $5 billion to companies (tax-free) that actually cure diseases. We could also designate ten university hot spots that have the world's greatest expertise in those rare diseases. Those programs would get funded $100s of millions annually to work in three teams (8 hours x 3 each day). They would work around the clock to find cures.

We give away billions if not trillions to other governments to fight wars, but can’t fund rare diseases and find cures. What's it going to take?

 

[Nikki J]

Dr Appel told Congress a number of years ago ALS is not incurable it is underfunded. This quote is often repeated because there is some truth in it. More research will bring better understanding and will eventually yield better results. ALS research has come a long way even in the last ten years. It doesn’t exist in a vacuum though. Pure genetic research has contributed as have findings in other diseases. crispr is something that may help many of us. The biggest issue is delivery. That isn’t unique to als. The answer may come from MD or another neurological condition. Anyway funding is limited. We were lucky to get what we did in thecACT for ALS but imo it could have been spent differently

 

[CC46]

I was only thinking the exact same thing this morning. I live in Australia though, but same thing happens here! It frustrates me

 

[JCKJLUP]

Does anyone know anyone that has been treated at Dr. Goodenowe Research Center in Canada?

 

[Nikki J]

Apply these rules to them https://www.tandfonline.com/doi/pdf/10.1080/21678421.2020.1765518?needAccess=true

 

[lgelb]

Re Dr. Goodenowe:

His web site, book, social feeds, and podcast appearances funnel people into an intensive treatment regimen with zero justification. I hear it's thousands of dollars per month and he claims 100% success.

Unfortunately, if we could just feed people things that labs say they are low on, and cure them, we would have cured everything around by now. We're very good at testing blood, urine, and CSF these days.

For example, when you have a bad infection, your white cell and/or neutrophil counts increase. Does that mean that infusing those will cure your infection? Hardly. They are signs, not causes.

Also, when you take supplements, etc. your body has to work to metabolize them, when it's already working overtime in ALS. Supplements can also cancel each other out or increase each other's effects.

It gets even more dealer's choice when you mix in OTC and prescription drugs as most PALS do. In short, there is real risk of accelerating progression and tanking your health, as well as depleting your bank account, with regimens like these.

Re the moonshot approach:

Though FDA now allows more applications to be submitted in installments, it still all has to be considered as a whole -- which takes more than a week, a committee has to meet, etc.

You might say, why? But, unfortunately, even under the current system, rushing to judgment has pulled a rug out from PALS more than once in the last couple of years.

So it may be that we don't need more political pressure to rush, per se, but as Nikki points out, rather the resources and scientific progress to do it right under a rational disease strategy.

Maybe a shade off topic, but STAT just did a pretty good roundup of the much ballyhooed Right to Try regulation's results so far.

I've seen all kinds of grand ALS plans over the years, but the NINDS report last year and National Academy report this year are both more laundry lists than plans. Still, if you have a favorite part of either report that you'd like to see funded, by all means, write your legislators.

 

[Cbreezy]

I would like to know as well? Did you find anything out?