Gate2Brain and its “molecular tractors” technology: new way to optimize the transport of drugs to the brain

Article by Andrea Rivero García

Meritxell Teixidó, CEO and co-founder of Gate2Brain, explains in an interview with Gaceta Médica the keys to this product and its designation as an orphan drug by the EMA

The European Medicines Agency (EMA) has granted orphan drug designation to G2B-002 technology, recognizing it as a potential treatment for a rare and serious disease. But what exactly is G2B-002 and what objectives does it pursue? Meritxell Teixidó, CEO and co-founder of Gate2Brain, explains the keys to this product in an interview with Gaceta Médica.

Gate2Brain is a biotechnology company founded in July 2020, born from the collaboration between three prominent institutions: the University of Barcelona, ​​the IRB of Barcelona and the Sant Joan de Déu Hospital, “a benchmark in pediatric oncology in southern Europe. “, points out the expert. The company emerged with the aim of advancing the development of innovative technologies to improve the transport of drugs to the brain, which could revolutionize the treatment of various neurological diseases.

“I like to explain it in a simple way: imagine tractors that go to the brain, and like trailers, they transport drugs that, on their own, would not know how to get there,” explains Teixidó. These “molecular tractors” were developed during Teixidó’s professional career at the Barcelona IRB, where he spent 15 years.

“During that period, I co-supervised 10 doctoral theses and it was there that we discovered that peptides, which are very small proteins, could act as ‘molecular tractors’ to improve the transport of drugs to the brain,” says the expert. At first, when finding these “tractors” everyone would think of diseases such as Parkinson’s or Alzheimer’s, which makes sense since, as Teixidó assures, “one in four of us will need some treatment for the brain at some point in our lives.”  However, the final decision was to focus on childhood brain tumors.

Pediatric brain tumors

Pediatric brain tumors are particularly complex to treat due to several interrelated factors. Firstly, its location in the brain, a delicate organ essential for all vital functions, makes any surgical intervention or treatment difficult without putting other areas at risk. Furthermore, these tumors usually grow quickly and aggressively, invading healthy tissues and making their complete removal difficult.

Children’s sensitivity to conventional treatments, such as chemotherapy and radiotherapy, and their potential long-term side effects on their development, add another layer of complexity. Added to this is the fact that a child’s brain is constantly developing, which influences the response to treatment and the appearance of side effects.

All of this makes it essential to develop more effective treatments for these tumors and thus improve the survival and quality of life of children and their families.

“We try to do our best, but the families who today receive a diagnosis of the disease we are working on deserve all our respect and support,” emphasizes Teixidó. “Currently, we are two years away from starting clinical trials, we are all working so that one day we will achieve significant advances, but we must be realistic: the cure is not yet at hand.

G2B-002 as a potential treatment

The orphan drug designation that G2B-002 has received is granted to drugs that target conditions that affect fewer than 5 in 10,000 people in the European Union, and that offer significant advantages over limited or non-existent alternatives.

“G2B-002 has the potential to be applied not only to childhood tumors, but also to adults in the future,” says Teixidó. “Having obtained the orphan drug designation does not mean that it is already available on the market, but rather that this designation will greatly facilitate our progress in the coming years until we reach patients,” she highlights. During this time, researchers will be able to work closely with the Medicines Agency, which will make the entire process more agile, given that this is a rare disease that currently has no cure.

“G2B-002 has the potential to be applied not only to childhood tumors, but also to adults in the future”

“Once we reach the market, this designation offers us advantages, such as the extension of the exclusivity time, something similar to what happens with patents,” explains the expert, who adds that “this, in turn, encourages more research and advances in the treatment of rare diseases.”

The principle behind “tractors” is that, by improving the efficiency of drug transport to the brain, more of the drug reaches its target directly. “If you administer 10 units, only one reaches the brain, if you want 5 to arrive you would have to administer 50 units,” explains Teixidó, who clarifies that “we cannot simply increase the amount, since that could generate side effects.” The goal is to optimize transportation to minimize these side effects, thus improving the effectiveness of the treatment.

Overcome the blood-brain barrier

Gate2Brain’s patented technology makes it easier for drugs to cross the blood-brain barrier with high precision, increasing both the effectiveness and safety of brain tumor treatment.

The blood-brain barrier is a structure that protects the brain, and although its role is crucial, it also represents a challenge when treating brain diseases. This protection makes it difficult for medications to enter, making their administration more complex. “Our technology is not based on opening the barrier, as other technologies do, since that would imply losing that natural protection, which is not our objective,” says the specialist. “What we discovered is that the peptide we developed can cross the barrier without compromising its protective function,” he emphasizes.

“We discovered that the peptide we developed can cross the barrier without compromising its protective function”

These peptides manage to cross the barrier using the natural “doors” that the barrier has to allow the passage of essential nutrients to the central nervous system, called transport mechanisms. “Our peptide finds these doors and crosses them without altering the normal functioning of nutrient transport,” explains Teixidó.

Why have they focused on childhood tumors? “Children represent 25% of our current population and 100% of our future,” says the expert, who adds that “it is known that children’s blood-brain barrier is more sealed, probably because they are a developing brain, which which makes it make sense to validate our technology in a pediatric indication initially.” Then, moving from the pediatric to the adult population is a logical step. If the technology is effective, non-toxic and works well in children, it is reasonable to assume that it will also be successful in adults,” says the specialist.

Current stage of G2B-002

Before reaching clinical trials there is a crucial stage: the preclinical phase. This phase is divided into two parts: the non-regulatory preclinical and the regulatory preclinical, which is the closest to clinical trials. “Currently, we are in the regulatory preclinical phase, which means that we have already gone one step further and have stopped working with rodent models,” explains Teixidó. “This puts us about two or three years away from being able to begin the clinical trial in humans,” says the expert. Before administering the first treatment to a patient, the clinical batch and everything necessary must be prepared to ensure that the process is safe and effective.

Read the article: La Gaceta Médica