Sponsored by
We have covered Onc201 extensively in past News Blasts. The Musella Foundation has been fighting for years to get patient access to this drug, as we know it can help patients with diffuse midline glioma (DMG) with the H3K27 mutation. We have funded $875,000 in grants to help develop this treatment (we were not mentioned in the article because we did not fund this specific project). Now that results showing Onc201 nearly doubles median survival for DMG-H3K27 patients have been published in a peer-reviewed journal, there will be a wave of news coverage for this drug. But the important part of this story is that patients STILL cannot easily access this drug. This is not right, and it is a clear example of why we need the Promising Pathway Act. If you haven't already, please click here to send a letter to your Congress reps in support of the Act.
A recent study published in Immunity on August 11, 2023 found that an immunotherapy drug called anti-CTLA-4 prolonged survival in a mouse model of mesenchymal-like glioblastoma. Anti-CTLA-4 was one of the first drugs designed to stimulate the immune system to fight cancer, but it was quickly followed by another, anti-PD-1, that was less toxic and has become more widely used in clinical trials. Upon investigating why the anti-CTLA-4 therapy was more effective in this mouse study, researchers found that the drug prompted CD4+ T cells to secrete interferon gamma which activates microglia to 'eat' more tumor cells. Although promising, this is still early-stage research.
This is a new form of targeted radiation. We did a webinar about it: https://virtualtrials.org/video2022.cfm?video=202205. They reported on the early results of their phase 1 trial, which was a dose escalation trial. They reported an amazing median survival of 10 months. They did not have an external control group but in general, people with leptomeningeal mets only live 2 weeks to 4 months. As the find the correct dosages it should get better. And as I always say - combinations are the way to cure these things. In the past, the main problem was that nothing really had time to work. Now, if you could buy 10 months - that gives you a lot of time for other treatments to work. Very exciting.
Disclaimer: Plus Therapeutics is a sponsor of our organization.
As I mentioned many times recently, Sonodynamic therapy is one of my favorite experimental treatments. It is too early to tell how effective this will be, but the concept is elegant and preclinical work showed very good results. From the patient's point of view, it is relatively easy as it is non-invasive - no cutting through the skull! A dye is injected into the arm veins, which is taken up by tumor cells and not normal brain. Focused ultrasound is applied (similar to the way a sonogram is done on a fetus) which kills cells that have taken up the dye. Net effect is killing cancer cells while leaving normal cells alone.
There are two competing systems that are in clinical trials now. Nobody knows which is best. The one mentioned in the article is from the company SonALAsense, Inc. This trial is for progressive or recurrent Glioblastoma and requires that only one tumor be present. They also have a trial of the same system for newly diagnosed Glioblastoma - but only in Italy right now (check clinicaltrials.gov for updates). The other is from Alpheus Medical, Inc. and they can accept multifocal tumors.
GammaTile is an FDA approved treatment for newly diagnosed and recurrent primary and metastatic brain tumors. It is implanted at the time of surgery and slowly releases radiation to the tumor bed. It may be able to replace standard radiation. I love that they are collected the real world data in a registry so they learn from every patient. All brain tumor patients show be tracked in a registry so we can learn from each of them!
This is very early - just starting human trials. However it is also a shot at a home run therapy not only for brain tumors but most solid tumors. It is the holy grail of cancer treatments - oral, doesn't hurt normal cells, may work on all cancers by targeting a pathway that is central to all cancers. Will be keeping an eye on this. The trial is open in California now and will open in Arizona soon. https://classic.clinicaltrials.gov/ct2/show/NCT05227326
We have run out of funding and the copay program will remain closed to new and renewal application until we get enough donations to the program to reopen. Of course, we will continue to pay claims for those that have an active grant!
Follow the link in the article to see a short video explaining this new technology that maps out the important areas of the brain so the surgeon knows how much he can take out without doing too much harm. The article also talks about a new advanced radiosurgery system that they will be aquiring soon!
I love this company's plan: they look at failed trials that had some patients do well, and figure out why they failed and which patients benefit. Then they run the trial only on those patients predicted to have a good outcome. One of their first projects is to take Toca511 and TocaFC (now called DB107) and start a trial only for people with the correct biomarker. They did not yet annoucne the biomarker - when they do we can spread word of it and notify people in our database who have that marker. The original early trials of Toca511 looked bery good. I know of a few patients in the trial that did pretty well. However, their large phase 3 randomized trial failed. At that time they did not know which biomarkers predicted success but now they do. It did not help the average patient, but it did help a long tail of people who did have this biomarker!
This is from our friends at Pinpoint Patient Recruiting. We mentioned this recently but they need a few more participants!
They do surveys to learn about the experiences of people dealing with a Glioblastoma. They have a few surveys now. You can do as many as you have time for. They pay you $30 to $105 (depending on the length of the survey) to take the survey, and will also make a donation to the Musella Foundation for every survey you fill out! So please do them!
SurVaxM is an experimental vaccine for the treatment of brain tumors. Early results from a small trial were recently reported Of 63 patients with newly diagnosed Glioblastoma, the median overall survival was 25.9 months from the first dose of the vaccine, which was probably 3-4 months from diagnosis. Our guest speaker is Michael J. Ciesielski, PhD, CEO of Mimivax. This should be of interest to all people dealing with brain tumors.
Nice article about one of my favorite neuro-oncologists! He makes great points about disparities not only among minorities but among the elderly and the need to get community doctors involved since 70% of cancer patients are treated in the community setting!
Another successful early trial of a new treatment! They report very early data on five recurrent Glioma patients who have failed all standard treatments. They had one complete response, and two had stable disease. Unfortunately, it appears that this trial is only available in China. Hopefully once it is successful it will be brought to the USA!
This is early results on a small number of patients but the results are very good. They started with newly diagnosed patients with unmethylated MGMT who did not have a complete resection. They had only a biopsy or a debulking. This is the worst subset of Glioblastoma. They report of 6 patients, 5 out of the 6 are alive at the 15 month point, and 1 had a complete response, 2 had a 99% response, and 2 had a partial response that was durable! It is very rare to see complete response or 99% response in MGMT unmethylated patients. The trial is ongoing and worth considering for Glioblastoma patients with MGMT unmethylated.
The tumor type is Papillary craniopharyngiomas. Although this is a small study of 16 patients, the amazing thing is that no patient’s tumor progressed while on vemurafenib/cobimetinib, and none have died.
The treatment is a combination of a BRAF/MEK inhibitor (vemurafenib/cobimetinib).
If you have any tumor (not just brain - but pancreatic, ovarian, colon, melanoma, etc) with alterations in BRAF / MEK, it may be worth it to show your doctor this article. Both drugs are FDA approved so this should be easy to get - although expensive at about $13,000 per month. If you can not get these drugs, xCures (our partner) is running an expanded access program for another MAPK inhibitor for tumors with any of these alterations: KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations.
This shows the importance of getting genomic testing for your tumor. It has actionable results in a minority of cases but when it find the right markers, it opens the door to using targeted drugs that may help a lot.
SurVaxM is an experimental vaccine for the treatment of brain tumors. Early results from a small trial were recently reported Of 63 patients with newly diagnosed Glioblastoma, the median overall survival was 25.9 months from the first dose of the vaccine, which was probably 3-4 months from diagnosis. Our guest speaker is Michael J. Ciesielski, PhD, CEO of Mimivax.
This shows the need for better treatments. The biggest criticism I get about the Promising Pathway Act that I have been supporting is "Patients do not want treatments that are not fully tested." Read the statistics from this article and consider if you were in such a situation, would you be open to trying a treatment that showed good results in phase 2 trials, or would you rather wait 5-10 year for them to complete the phase 3 trials?
This article talks about using 5ALA to find tumor cells outside of the area that shows up as tumor on MRI. They conclude that it would be useful to analyze these cells for targeted therapies but missed the obvious use: sonodynamic therapy. If 5ALA can find the tumor cells outside of the main tumor which doesn't show up on MRI, then using focused ultrasound to kill the cells that take up 5ALA may be effective in preventing recurrence.
