Al Musella

Pres. of the Musella Foundation For Brain Tumor Research & Information, Inc. anything I say here is my own opinion and does not represent the foundation

[Al Musella @AlMusella]

Yes. Optune got approval for recurrent gbm based mostly on quality of life with no survival advantage. They got approval for newly diagnosed based on a large overall survival advantage. However Medicare used that to save money. They will only pay when used for newly diagnosed. You have to start within 7 weeks of the end of radiation or Medicare will not pay ( there are exceptions but this is the rule).

[Al Musella @AlMusella]

There were 2 excellent presentations on DCVax at ASCO yesterday. I uploaded them to my website at https://t.co/WYnXT78R4w One is about the manufacturing of DCVAx and the other is about the trial design and results. It nicely explains the endpoints and external controls. #GBM

[Al Musella @AlMusella]

@BTNcares Happy birthday 🎉!

[Al Musella @AlMusella]

@mukundiyngr @oncodaily @Dr_RShatsky @OncoAlert Can you add brain cancer? Looks great

[Al Musella @AlMusella]

Not true. Unfortunately when the patient can’t afford the deductible they go without care when possible. No more yearly physicals or blood pressure drugs. That is why the original post said hospitals loan the money for the deductible even when they know they won’t get paid. They are forced to overcharge on the insurance to make up for that loss

[Steve Martin @RighttoTryGuy]

These are the scans of an 11 year old girl from Florida whose brain cancer has metastasized to her spine.

Her family has exhausted all traditional treatment options, and is now seeking access to personalized treatments. The same kind of personalized treatments which more than 18 laws passed in states like NC, LA, CO, and TX were designed to provide access to.

And yet instead her family is scrimping together every last penny to fly her to Germany. Why? Because providers in the US have no guarantees they can participate in a state law without risking federal enforcement by @US_FDA .

I don’t think this is what the President had in mind when he signed the Right to Try act into law in 2018. FDA can, and should, issue guidance around enforcement discretion to provide safe harbor for companies making medicines for use under a state law.

[Al Musella @AlMusella]

@mgood695 Congratulations! What will you be working on?

[Al Musella @AlMusella]

I question the gbm stats. 33% 5 year survival with no increase looks wrong. We can probably get to 33% if we combine some treatments but the typical patient doesn’t get access to these treatments resulting in probably a 7% (as reported by cbtrus). We had some gains over the last 30 years.

[Al Musella @AlMusella]

@andrewcaravello @grok @DrOzCMS @VP @WHFraudTF The FDA sits on an enormous amount of clinical trial data. Instead of requiring every new therapy to build a fresh control group from scratch, why not use a continuously updated matched external control database—especially in lethal diseases like GBM where time matters?

[Al Musella @AlMusella]

@rachelreising96 @POTUS @realDonaldTrump @RFKJr_Official Rachel, take a look at the promising pathway act. It fixes the problems with right to try. We (brain cancer organization) are working with I am als on it

[Al Musella @AlMusella]

@Neuroscope_mp Why not use it at a very low dose and try to lose 2 pounds a month maybe get rid of side effects

[Al Musella @AlMusella]

@PRVWatch He did have a lot of great ideas. This is one of them and it should be expanded to include treatments for all diseases with no effective treatment

[Al Musella @AlMusella]

@NeilFlochMD Maybe the side effects of the drugs could be controlled if they shoot for much smaller weight loss. It can’t be healthy to lose weight so fast.

[Al Musella @AlMusella]

@RNAiAnalyst And conditional approval! But add guardrails à la the promising pathway act.

[Al Musella @AlMusella]

Today is our anniversary.

The Musella Foundation received its 501(c)(3) approval on May 12, 1998, after incorporating on March 12, 1997.

I started the first online brain tumor support group in 1992, when my sister-in-law was diagnosed with glioblastoma. That was the beginning of what became our mission: helping brain tumor patients and families find information, support, and hope.

Tonight at 7 PM Eastern, we are holding a webinar on oxygen in the treatment of glioblastomas.

Join us at: https://t.co/XXMox05kIN

[Al Musella @AlMusella]

@LogicalValueMx @US_FDA @DrMakaryFDA @laurencurehd @DesertDweller93 @LuckyPenguin10 @peter_mantas Right to try was great in theory but in practice it failed for the reasons Trump mentions. The promising pathway act fixes those problems and keeps the intention right to try

[Al Musella @AlMusella]

@thomslvey I lost 2 family members to the same type of brain tumor. Glioblastoma right before that story arc. It was really emotional for me. And that song was perfect.

[Al Musella @AlMusella]

The promising pathway act may be the best solution. Conditional fda approval after a phase 1 trial. Time limited to 8 years to give time to determine if it is worthy of full approval around traditional rules. All patients have to be followed in a virtual trial with deidentified live data available to the doctors so they can make evidence based decisions. Risk to patients is the same as entering a phase 2 trial. Consent is required as is limitation of liability.
My original version required insurance to pay and no limit on charging but I see that may cause other problems so now looking at different options. I want patients to be able to get the treatments and the drug companies to make a small profit to incentivize them to use the pathway. Perhaps require Medicare to set the allowed charge and maybe offer a discount until the treatment graduates to full approval. Any other ideas on pricing?

[Al Musella @AlMusella]

@andrewcaravello @RighttoTryGuy Andrew. This Montana pathway may be perfect for dcvax.

[Al Musella @AlMusella]

@zalaly @JAMANetworkOpen You would think these drugs would reduce diabetic retinopathy by reducing weight and bringing the a1c down. No?