The conclusion by the authors in the article is that this is a promising treatment, however they reported fairly average results:  for recurrent glioblastoma, the median OS was 7.9 months ([95% confidence interval (CI) 6.5-9.2 months] and the median PFS was 2.6 months (95% CI 2.3-2.9 months).

  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!  For those of you who need help with Temozolomide, try costplusdrugs.com  They sell temozolomide for about 10% of the cost of most pharmacies. 

Gallium maltolate is an experimental treatment for brain tumors that is showing early promise. The Musella Foundation recently gave them a grant to help start an expanded access program for the drug. Watch the webinar for details!

Lots of news coming from GBM CAR-T trials this month. This one from Mass General Cancer Center reported on the first 3 patients enrolled in the trial. All 3 experienced significant reductions in tumor size within a few days of a single infusion of the trial treatment (CAR-T cells + T-cell engaging antibody molecules). However, all 3 eventually had progression. The next step may be testing multiple infusions or testing the treatment together with chemotherapy to get more durable response. 

Our Copay program has given out grants to 108 patients this year, which puts us on pace for our best (or worst - as it is unfortunate that our program is even needed) year ever.     

Unfortunately, we are running low on funds and will have to close to new and renewal applicants soon. We do not know when or if the program will reopen as we work to obtain donations. This is a lifesaving program, as many of the recipients tell us they wouldn't take the treatments without our help. If you would like to help, make a donation and select "copay fund" for where it is to be used!

We have our 5K fundraisers coming up in May - but those funds are dedicated to brain tumor research only. We will award brain tumor research grants in June.  (Researchers can apply now - contact me for details).

Rod Norland's new book has received a warm welcome and positive reviews from the brain tumor community, with many people joining the discussion about brain cancer as a result.

 Participation is free and easy - no pre-registration required. Just go to virtualtrials.org/webinar a few minutes before the event!

Good results from a large Phase 1 trial for IL13Rα2-targeting CAR T cell therapy. This trial enrolled heavily pre-treated recurrent high-grade glioma patients. Most were GBM and ~75% were at second recurrence or more. Half of the patients (29/58) achieved stable disease for at least 2 months, and there were a few partial and complete responses. The study cohort who received the maximum dose had the best median OS at 10.2 months. 

 Zotiraciclib is a multi-kinase inhibitor that can be taken orally. A previous Phase 1 study showed zotiraciclib may be effective in people with recurrent gliomas containing mutations in the IDH1 or IDH2 genes. I like this trial because there is no placebo group; the trial team will use a matched external control group instead. 

 Rod Norland is a Pulitzer Prize-winning journalist. He has covered conflict zones in over 150 countries, and he was diagnosed with GBM in 2019. His new memoir, Waiting for the Monsoon, shares his experiences both with war and with brain cancer.

 Multiple studies on TTFields have shown a correlation between clinical outcomes and time on treatment. This new paper explores a number of factors impacting TTFields dosimetry, which also has important implications for efficacy.

 Very exciting.  Berberine is an easily available oral supplement.  In the lab, it greatly enhances the ability of 5-ALA to light up tumor cells, especially the glioma stem cells.   The article mentions how important that is when surgically removing the tumors, but another use that should be investigated is the use during sonodynamic therapy.    If this works in people (the article is from lab tests), it might be part of  a major breakthrough when used at surgery to remove more of the tumor then with sonodynamic therapy to mop up those remaining cells!

 This paper summarizes the four clinical trials and the expanded access programs for Onc-201 in recurrent diffuse midline glioma.  Onc-201 has a new brand name - it is now called dordaviprone.   When used by itself, about 40% of the patients had the tumors stable or shrink, with a median survival of about 13.7 months from the time of starting Onc-201. There was no control group, but the median survival for this tumor type is only 12 months from diagnosis. These patients all had recurrent disease and were on average 10.9 months from diagnosis when starting Onc-201. Bottom line is these results are good, possibly the best ever reported so far in this disease, but of course not good enough. We need to get this drug approved so it can be used in creative combinations to improve these numbers.

They looked at their database and found that these drugs: metformin, beta-blockers, ACE Inhibitors, and angiotensin receptor blockers as well as having Parkinson's disease, are all correlated with a longer survival period for elderly patients with Glioblastoma!  And these are significant gains!

 The technique they use - which is called fractional tumor burden (FTB) mapping, can help differentiate between treatment effects, swelling, necrosis and true progression, and can quantify the volume of the tumor better so you can more accurately tell if a treatment is working or not.
Here, they apply the technique to monitor glioblastoma patients who use Optune.  They couldn't tell in the first 2 months if Optune was working, but by month 3,4 and 5, they could predict if it was working or not, even though a standard MRI at the 3 month mark is expected to show pseudoprogression when Optune is working - which sometimes causes patients to stop the therapy prematurely even though it was helping. This could be a game-changer and should be consider for all treatments, not just Optune.

 From our good friends at the Cure Starts Now.  This is an amazing event and supports a great cause.   

 The HUGE news in this report is the FDA set a PDUFA date of April 30, 2024 for their drug tovorafenib  in relapsed or progressive pLGG.  A PDUFA date is the target date for when the FDA will make a decision on approving the drug or not.  Tovorafenib is an investigational, oral, brain-penetrant, highly selective type II RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is also  being investigated in primary brain tumors or brain metastases of solid tumors. Tovorafenib is currently under evaluation in two pivotal clinical trials for pLGG. Tovorafenib is also being evaluated as a combination therapy for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway aberrations (FIRELIGHT-1).  

 This doubled survival for recurrent glioblastoma and is easily available. Worth considering.

 We are planning to introduce version 2.0 of the Promising Pathway Act sometime next week. This open house is for patient advocates to hear about the act and to network with other groups. If you are in the area, we need people to show up to let them know we support this bill!  

 Excellent paper on sonodynamic therapy - the history, mechonism of action, current trials  and future prospects. The authors discuss all of the available devices - not just their own.    I beleive this therapy is going to be  a major breakthrough in the treatment of brain tumors. It is very elegant, non-invasive, early indications are that it is safe and well toerated, and it can be repeated as often as needed.