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I have been interested in this drug for a long time. I met the inventors of this drug and gave them a research grant back in 2013. I am very excited that the company opened an expanded access program for pediatric patients with Diffuse Midline Glioma and DIPGs. There is a huge unmet need for these tumor types, and we badly need more treatment options.
As I mentioned many times before, I helped start xCures and am a paid consultant to xCures. They are revolutionizing how expanded access works. In the past, most doctors did not like to participate in expanded access programs because of the extra time needed for paperwork and to work out logistics. xCures takes over most of the paperwork and logistics now, and helps the doctors with the little paperwork that they have to do.
In the past, expanded access programs did not help speed up drug approvals. Now, xCures monitors the patients in expanded access programs as a virtual trial, gathering real world evidence of how the drug works. Best part is programs like this accept most patients with the specified tumor types so we really get to know how the drug performs. This is in contrast to clinical trials that pick the patients that would make the drug look best. This real world evidence can (and has) been used to get FDA designations and as part of new drug applications to support the evidence from clinical trials.
And best of all, xCures makes it much easier for the patient. First, our patient navigation program (Musella Foundation, Cancer Commons and xCures) can help find the best treatment options for each patient - including expanded access programs, then we do a lot of the work needed to actually enroll and get the drug.
xCures ran the first expanded access program for Onc-201, which was a big success, and is currently running another expanded access program - Ulixertinib, which is for any type of cancer (including braintumors) that has mutations in the MAPK pathway (KRAS, BRAF, NRAS, HRAS, MEK and ERK mutations). They are also running the Patient-Centric Platform Trial for Precision Oncology which is the basis of our patient navigation program!
OKN-007 is an experimental treatment that reduces the expression of genes important for tumor growth and also reduces the amount of blood vessels that can grow into the tumor. So it acts directly to kill the tumor cells like a chemotherapy and indirectly by reducing the nutrients getting to the tumor like bevacizumab. There is a clinical trial open of OKN-007 for Newly Diagnosed Glioblastoma in Adults.
A new expanded access program for OKN-007 just opened for pediatric patients (1 to 18 years old) who have diffuse midline glioma. Unlike most clinical trials, the expanded access program accepts patients with a Karnofsky score of 50 or more (most trials do not allow scores under 70). Patient have to complete radiation before starting this expanded access program, but can, but do not have to, show progression - so it can be done right after radiation or any time thereafter! The drug is supplied at no cost to the patient. For details and to get started with the expanded access program, go to https://patient.xcures.com/signup?program=okn-eap&source=olp
This program is being run by xCures, in conjunction with Oblato, Inc. Having xCures run the expanded access program means it is much easier for the patient and doctor than most expanded access programs - xCures does most of the work. (Disclaimer: I am a paid consultant to xCures!).