Brain cancer refers to the abnormal growth of cells within the brain that form a tumor. There are many types of brain cancer, including primary brain tumors, which originate in the brain, and secondary brain tumors, which spread to the brain from other parts of the body. Some common types of primary brain tumors include gliomas, meningiomas, and pituitary tumors.

The symptoms of brain cancer can vary depending on the location and size of the tumor, but may include headaches, seizures, vision or hearing problems, difficulty with balance or coordination, and changes in mood or personality. Treatment for brain cancer may include surgery, radiation therapy, and chemotherapy, or a combination of these approaches. The specific treatment plan will depend on the type and stage of the cancer, as well as the patient’s overall health and preferences.

Glioblastoma multiforme, also known as grade IV astrocytoma, is a malignant grade IV brain tumor that usually originates in the brain’s glial cells, which support and protect nerve cells. It is the most common type of primary brain tumor and accounts for about 15% of all brain tumors. It is one of the most lethal forms of brain cancer and is treatment-resistant in nature, wherein the patient has an extremely short survival period post-diagnosis.

Here are some statistics related to glioblastoma:

1. Incidence: Glioblastoma accounts for about 15% of all primary brain tumors and approximately 50% of all malignant brain tumors. It is more common in older adults, with the highest incidence occurring in those aged 75-84.

2. Survival Rates: The prognosis for glioblastoma is generally poor, with a median survival time of 15-16 months after diagnosis. Only about 25% of patients survive longer than one year, and less than 5% survive longer than five years.

3. Treatment: Standard treatment for glioblastoma typically includes surgery to remove as much of the tumor as possible, followed by radiation therapy and chemotherapy. However, even with treatment, recurrence is common, and the cancer often becomes resistant to therapy.

4. Treatment market size: the global GBM treatment market size reached US$ 2.0 billion in 2022. Looking forward, experts expect the market to reach US$ 3.1 billion by 2028, exhibiting a growth rate of 7.6% during 2023-2028.

Treatment for glioblastoma typically involves a combination of surgery, radiation therapy, and chemotherapy. Due to the tumor’s aggressive nature and tendency to spread quickly, treatment can be challenging, and outcomes are often difficult to predict. Often the tumor cannot be completely removed with surgery and therefore treatment may be lifelong. Research is constantly ongoing to develop new treatment methods to combat this challenging form of brain cancer.

The blood vessels that vascularize the central nervous system (CNS) possess unique properties, termed the blood–brain barrier (BBB), which allow these vessels to tightly regulate the movement of ions, molecules, and cells between the blood and the brain.

The BBB restricts the entry of most molecules, including many drugs, into the brain. This presents a major challenge for drug development, as drugs must be able to penetrate the BBB to be effective in treating CNS diseases. (To overcome this challenge, drug developers have developed a variety of strategies.)

Overall, developing drugs that can effectively cross the BBB remains a major challenge in drug development for CNS diseases.

Chemotherapy is a type of cancer treatment that uses drugs to kill cancer cells. The drugs work by targeting fast-growing cells, which includes cancer cells. Chemotherapy drugs can be given intravenously (IV), orally, or injected directly into the tumor.

Chemotherapy may be used alone or in combination with other treatments, such as surgery or radiation therapy. It may also be used before or after surgery to shrink the tumor or prevent it from coming back.

The side effects of chemotherapy can vary depending on the type of drug used and the dosage, but may include fatigue, nausea, vomiting, hair loss, mouth sores, and an increased risk of infection. These side effects can often be managed with medication or other supportive measures.

While chemotherapy can be an effective treatment for many types of cancer, it may not be effective for all patients, and it can have some significant side effects. The specific treatment plan will depend on the type and stage of the cancer, as well as the patient’s overall health and preferences.

Chemotherapy drugs can be administered in a variety of ways, including through injection into a vein, as a pill or capsule, or through an infusion pump that delivers the medication continuously over a set period of time. Once in the bloodstream, the drugs travel throughout the body, targeting cancer cells wherever they may be.

Systemic chemotherapy is a type of cancer treatment that uses drugs to destroy cancer cells throughout the body. Unlike localized treatments such as surgery or radiation therapy, which target a specific area of the body, systemic chemotherapy is designed to reach cancer cells that have spread beyond the original site of the tumor.

While systemic chemotherapy can be effective in destroying cancer cells, it can also cause side effects such as nausea, hair loss, fatigue, and an increased risk of infection. In addition to the side effects, chemotherapy can also cause more serious complications, such as heart damage, kidney damage, and lung damage.

Alkylating agents work by interfering with the DNA of cancer cells, preventing them from dividing and growing. These drugs are used to treat a variety of cancers, including leukemia, lymphoma, and brain tumors.

Temozolomide is a chemotherapy drug (alkylating cytostatics) specifically used to treat glioblastoma. It works by damaging the DNA of cancer cells, leading to their death. Temozolomide is typically given orally in a pill form and may be used alone or in combination with radiation therapy.

The side effects of chemotherapy drugs, including alkylating agents and temozolomide, can vary depending on the specific drug used and the dosage. Some common side effects may include nausea, vomiting, hair loss, an increased risk of infection, and fatigue. These side effects can often be managed with medication or other supportive measures. The specific treatment plan will depend on the type and stage of cancer, as well as the patient’s overall health and preferences.

The complications and side effects of systemic chemotherapy can be minimized through intracranial delivery of cytostats. This approach is used for the drug SI-053 by the company Double Bond Pharmaceutical (the drug which starts clinical trials 2023).  This is why SI-053 is unique and can become a game changer. SI-053 uses pharmacological activity of temozolomide.

Temozolomide is a chemotherapy drug commonly used to treat glioblastoma. The effectiveness of temozolomide can be influenced by the level of expression of a protein called O6-methylguanine-DNA methyltransferase (MGMT) in the cancer cells.

MGMT is a protein that repairs DNA damage caused by chemotherapy drugs such as temozolomide. When MGMT levels are high, it can prevent temozolomide from effectively damaging cancer cells, leading to treatment resistance and poorer outcomes.

In patients with low levels of MGMT expression, temozolomide can effectively damage cancer cells and improve treatment outcomes. Therefore, testing for MGMT expression levels can help determine the most appropriate treatment plan for glioblastoma patients.

When temozolomide is used to treat glioblastoma, it is often given in combination with radiation therapy. The treatment plan typically involves six weeks of radiation therapy followed by six to twelve cycles of temozolomide. The specific treatment plan will depend on the patient’s individual circumstances and the stage of their cancer.

Overall, the treatment with temozolomide and MGMT expression level is an important consideration in the management of glioblastoma.

The cost of drug development can vary significantly depending on the type of drug, the complexity of the development process, and the regulatory requirements in different countries. According to estimates by the Tufts Center for the Study of Drug Development, the average cost of developing a new drug from discovery to approval is approximately $2.6 billion.

The cost of drug development includes various expenses, such as research and development, clinical trials, regulatory approval, marketing, and manufacturing. These costs can be influenced by various factors, including the complexity of the disease being treated, the size of the patient population, and the level of innovation in the drug.

The high cost of drug development is often cited as a barrier to the availability of affordable medicines. To address this issue, some countries have implemented policies to encourage drug development and improve access to affordable medications. These policies may include funding for research and development, streamlined regulatory processes, and pricing controls for drugs.

In recent years, there has also been an increasing interest in exploring alternative models for drug development, such as open-source drug development and collaboration between academic institutions and industry partners. These models aim to reduce the cost of drug development while ensuring the availability of affordable and effective treatments for patients.

Clinical trials are research studies that involve people and aim to test new treatments, drugs, or medical procedures to determine their safety and effectiveness. Clinical trials are essential for advancing medical knowledge and improving patient care.

In a clinical trial, participants are usually divided into groups and given different treatments, including a placebo or standard treatment, to compare their effects. The trial may also collect data on safety, side effects, and quality of life.

Participation in clinical trials is voluntary and requires informed consent. Participants may benefit from receiving new treatments or early access to new drugs or medical procedures. However, there may also be potential risks associated with participating in a clinical trial, including side effects, discomfort, and the possibility that the new treatment may not work.

Clinical trials are conducted in phases, with each phase designed to answer specific research questions and test different aspects of the treatment. Phase I trials are typically the first stage and involve a small number of participants to determine safety and dosage. Phase II and III trials involve larger groups of participants to assess safety and effectiveness, respectively.

Clinical trials are an essential part of the drug development process, and they help ensure that new treatments are safe and effective before they are approved for widespread use. Patients with cancer may be eligible to participate in clinical trials as part of their treatment plan, and their healthcare provider can provide more information on the available options.

Phase 1 clinical trials are the first stage of human trials for new drugs or treatments. These trials are designed to evaluate the safety and tolerability of the drug, as well as to determine the appropriate dosage levels. Phase 1 trials usually involve a small group of healthy volunteers or patients with the target disease.

During a phase 1 clinical trial, the drug is usually administered in gradually increasing doses to a small group of participants. The participants are closely monitored for any adverse reactions or side effects. The main goal of the trial is to determine the maximum tolerated dose (MTD) of the drug, which is the highest dose that can be given without causing unacceptable side effects.

In addition to evaluating safety and tolerability, phase 1 trials may also collect data on the pharmacokinetics (how the drug is absorbed, distributed, metabolized, and excreted) and pharmacodynamics (how the drug affects the body) of the drug.

Phase 1 trials typically last several months and involve a relatively small number of participants. If the drug is found to be safe and tolerable in phase 1, it can then move on to phase 2 trials, which focus on evaluating the effectiveness of the drug in a larger group of patients.

Double Bond Pharmaceutical is a Swedish biotechnology company that specializes in the development of innovative therapies for cancer and other diseases. The company was founded in 2010 and is headquartered in Uppsala, Sweden.

Double Bond Pharmaceutical has a diverse pipeline of drug candidates, including a novel drug (current name: SI-053) for the treatment of glioblastoma, which starts phase 1 clinical trials 2023.

In addition to its drug development programs, Double Bond Pharmaceutical has also developed a drug delivery technology platform called Belogal. This platform enables the targeted delivery of drugs to cancer cells and other diseased tissues, while minimizing side effects.

The drug SI-053 is protected by orphan status and by several patents.

Orphan status for a drug refers to a special designation granted by regulatory agencies, such as the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA), to drugs intended for the treatment of rare diseases or conditions that affect a small number of patients.

The benefits of orphan status for a drug include:

1. Market exclusivity: Orphan drugs are granted market exclusivity for a certain period of time, which means that no other similar drug can be approved for the same indication during that time. This provides an opportunity for the developer of the drug to recoup their investment and make a profit.

2. Tax credits and grants: Developers of orphan drugs are eligible for tax credits and grants to help cover the cost of clinical trials and other development expenses.

3. Protocol assistance: Regulatory agencies provide protocol assistance to developers of orphan drugs to help them navigate the approval process and ensure that the trials are designed to meet regulatory requirements.

4. Faster approval: Orphan drugs are subject to a faster approval process than other drugs, which means that they can reach patients more quickly.

5. Patient advocacy: Orphan drugs often have strong patient advocacy groups, which can help to raise awareness of the condition and increase support for the drug’s development.

Overall, orphan status can provide significant benefits to the developer of a drug intended for the treatment of rare diseases or conditions, including financial incentives and regulatory assistance, which can help to bring new treatments to patients more quickly.

Protection of intellectual rights refers to the legal framework and mechanisms that exist to safeguard the exclusive rights of individuals and entities who have created or own intellectual property. Intellectual property can include inventions, trademarks, copyrights, and trade secrets.

To protect these rights, individuals and entities can take various measures such as registering their intellectual property with the relevant government agencies, enforcing their rights through litigation or negotiation, and implementing measures to prevent unauthorized use or infringement of their intellectual property.

Governments also play a key role in protecting intellectual property by enacting laws and regulations that govern the ownership, use, and protection of intellectual property, and by providing legal remedies and sanctions for violations of these rights.

Effective protection of intellectual rights is critical to fostering innovation and creativity, promoting economic growth, and incentivizing investment in research and development. It also helps to ensure that inventors and creators are able to benefit from their creations and receive fair compensation for their efforts.