Everolimus (Rapalog): Teen 'cured' of brain cancer in world first as docs 'watched tumour disappear'

Lucas and his family traveled from Belgium to France so that he could become one of the first patients to join the BIOMEDE trial which tests potential new drugs for DIPG.

From the start, Lucas responded strongly to the cancer drug everolimus, which he was randomly assigned.

“Over a series of MRI scans, I watched as the tumor completely disappeared,” Grill told AFP.

But the doctor did not dare stop the treatment regimen – at least until a year and a half ago, when Lucas revealed he was no longer taking the drugs anyways.

“I don’t know of any other case like him in the world,” Grill said.

Exactly why Lucas so fully recovered, and how his case could help other children like him in the future, remains to be seen.

Seven other children in the trial survived years after being diagnosed, but only Lucas’s tumor completely vanished.

“Lucas’s tumor had an extremely rare mutation which we believe made its cells far more sensitive to the drug,” he added.


Read “The Brighter Side Of News”. It seems hardly a day goes by that they don’t report on a cancer cure. I believe that there is a lot of breakthrough drugs held up by the regulatory process at the FDA.


But this case was repurposing everolimus rather than testing a new drug.


I missed that. Thanks


Anyone know the dose? Also is the dosing from Everolimus to Rapamycin the same?

Besides the happy story, I find the fact that everolimus somehow reached the tumor in the brain, being good news. .

Here is the trial they are talking about I think:

The trial team think that giving a targeted cancer drug (a biological therapy Open a glossary item) alongside standard radiotherapy might be better. But they aren’t sure.

Some targeted cancer drugs work by blocking pathways in cancer cells that tell them to grow and divide. These are called cancer growth blockers. Certain substances (biomarkers) in the tissue of the tumour show that these pathways are active.

As part of the trial researchers take a small sample of your tumour tissue (biopsyOpen a glossary item) to look for these biomarkers. Depending on which biomarkers are present you have one of the following as well as radiotherapy:

The aim of this trial is to find how well erlotinib, everolimus and dasatinib with radiotherapy works for children and young people with diffuse intrinsic pontine glioma.

Here are the details on the study:

Dosing Used in this Study:

Drug: Everolimus

Tablets of 2.5 mg or 10mg. The prescribed dose is 5 mg/m²/day, orally, once daily. Dose will be capped at 10mg once daily.

More details on Everolimus dosing and comparison to Rapamycin: Everolimus instead of Sirolimus / Rapamycin? Anyone else trying?

Note: 10mg per day is a very high dose, a dosing that is typically only used in serous cancer situations. There is a significant level of immune suppression at that level, and in at least one clinical study it has resulted in serious life-threatening adverse effects and death due to infection.

Below are some examples in a different study - a high dose (e.g. 10mg/day) everolimus study for tuberous sclerosis complex patients and there was a death of a 27 year old woman due to e-coli sepsis.

Rapamycin is not a risk-free drug, especially as you increase doses above the regular 5 to 8mg dosing once per week level.

The most common Adverse Effects (AEs) of everolimus therapy were laboratory abnormalities (100% of patients) and infection complications (83 episodes in 15 patients). Infectious episodes of pharyngitis (67%), diarrhea (44%), stomatitis (39%), and bronchitis (39%) were the most common infections. They were mostly mild or moderate in severity (grade 1–2).

In two cases, life-threatening conditions related to mTOR inhibitor treatment were encountered. The first was classified as grade 4 pleuropneumonia and Streptococcus pneumoniae sepsis, whereas the second was classified as death related to AE (grade 5) Escherichia coli sepsis.

A 27-year-old woman with TSC was started on everolimus
treatment because of AML of the left kidney
(60 Å~ 48 Å~ 36mm in size). The other signs of TSC were
facial angiofibroma, hypomelanotic macules of the skin,
and shagreen patch. The diagnosis of TSC was made
12 years earlier when the patient underwent nephrectomy
because of a large tumor of the right kidney. The
patient received everolimus at a dose 10 mg/day and the
trough concentrations of the drug ranged from 4.08 to
5.08 ng/ml. After 3 months of everolimus therapy, a
reduction in AML was observed (40 Å~ 31 Å~ 20mm in
size). During treatment, hypercholesterolemia (309 mg/
dl) and transient leukopenia (3.2 Å~ 109/l) with neutropenia
(1.34 Å~ 109/l) was observed. She also reported
oligomenorrhea. After a gynecological consultation, a
functional ovarian cyst was identified and contraceptives
were prescribed. However, 2 weeks later, she was
admitted to the gynecological unit because of subabdominal
pain and an ovarian cyst (64 Å~ 53mm in seize)
on ultrasound examination. Torsion of the ovarian cyst
was suspected. On the day of admission, WBC was
9.2 Å~ 109/l, the absolute neutrophil count (ANC) was
6.6 Å~ 109/l, the hemoglobin level was 10.8 mg/dl, the
PLT count was − 275 Å~ 109/l, and the C-reactive protein
concentration was 8.0 mg/dl (normal < 5.0 mg/dl). The
patient was advised to continue intake of contraceptives
and everolimus. The next day, the general condition of
the patient aggravated. Her blood pressure was low (85-
/50mmHg). Her WBC and ANC decreased (WBC
−2.4 Å~ 109/l, ANC − 1.8 Å~ 109/l), whereas the hemoglobin
level (11.0 g/dl), the PLT count (185 Å~ 109/l), and coagulation
tests were normal. Computed tomography of the
abdomen and pelvis showed AML of the left kidney (size
as in the previous examination), an ovarian cyst measuring
65 Å~ 50 Å~ 40 mm, and fluid in the retroperitoneal
space with density of the blood. Further aggravation of
her general condition was observed. The patient was
transferred to the ICU and she died after 2 h with
symptoms of shock and multiorgan failure. Blood and
urine cultures collected when she was in the ICU were
positive for Escherichia coli.

Complications of mammalian target of rapamycin inhibitor anticancer treatment among patients with tuberous sclerosis complex are common and occasionally life-threatening



This certainly suggests that Everolimus gets through the BBB, at least in the doses used for the therapy.