April 2024 • PharmaTimes Magazine • 6
// TREATMENTS //
Researchers from the Massachusetts Institute of Technology (MIT) have shown that a non-invasive treatment called gamma treatment could protect cancer patients from ‘chemo brain’, memory impairment and other cognitive effects of chemotherapy.
Chemo brain is a term used to describe thinking and memory problems that a patient may experience before, during or after cancer treatment.
Originally developed to treat Alzheimer’s disease (AD), the treatment works to stimulate gamma-frequency brain waves, involving exposure to light and sound with a frequency of 40 hertz.
In the new study, MIT researchers used mouse models, which evaluated a chemotherapy drug known as cisplatin, often used to treat testicular and ovarian cancers, for five days and then took it off for five days and repeated it.
One group received chemotherapy only, while another group was also given 40-hertz light and sound therapy every day.
After three weeks, mice that received cisplatin showed the expected effects of chemotherapy, such as brain volume shrinkage, DNA damage, demyelination and inflammation, in addition to reduced populations of oligodendrocytes, the brain cells that produce myelin.
In mice that received gamma therapy along with cisplatin, significant reductions in symptoms were shown and had beneficial effects on behaviour, performing better on tests designed to measure memory and executive function.
Researchers then analysed the gene expression changes that occurred in mice that received the gamma treatment using single-cell RNA sequencing.
It found that the inflammation-linked genes and genes that trigger cell death were suppressed, particularly in oligodendrocytes, while beneficial effects could still be seen in mice that received the gamma treatment along with cisplatin for up to four months later.
Additionally, the gamma treatment improved signs of chemo brain in mice that received a different type of chemotherapy drug, methotrexate, which is used to treat cancers including lung and breast.
The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Marinus Pharmaceutical’s Ztalmy (ganaxolone) to treat cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD).
The approval marks Ztalmy as the first anti-seizure medication to be used in the UK to treat the rare epileptic seizure disorder.
Affecting around one in 42,000 people in the UK, CDD is a rare genetic disorder that occurs from mutations in the CDLK5 gene, which is crucial for normal brain development, causing seizures as early as the first week of life.
Ztalmy, taken orally through a dosing syringe, is a steroid that attaches to specific receptors in the brain and aims to stop epileptic seizures from occurring.
The approval was supported by evidence from the phase 3 Marigold randomised, double-blind, placebo-controlled clinical trial, which involved 101 patients – 50 who received Ztalmy for 17 weeks and 51 in a placebo group – with CDD aged two to 19 years.
After 13 weeks, a reduction in seizure frequency of almost 30% was observed in patients receiving the steroid, while those receiving the placebo had a reduction of almost 6.5%.
Patients treated with Ztalmy for at least 12 months experienced a reduction of nearly 50% in major motor seizure frequency.
Julian Beach, interim executive director, healthcare quality and access, MHRA, said: “We’re assured that the appropriate regulatory standards for the approval of this medicine have been met.
“Keeping patients safe and enabling their access to high-quality, safe and effective medical products are key priorities for us.”
The MHRA will continue to keep the safety and effectiveness of Ztalmy under close review.
Marinus’ treatment has already received approvals from the US Food and Drug Administration and the European Commission for patients living with CDD.
The company also provides a patient support programme in the US, Ztalmy One, to facilitate access to treatment for patients and provide ongoing prescription drug support and education throughout the treatment journey.