Advances in medical research are continuously contributing to the development of more effective treatments. In this piece, we will delve into the latest treatment options and explore ongoing IBD trials that offer hope for better management and potential cures.

Different IBD Treatment OptionsMedication Approaches

Medications are a cornerstone in the management of Inflammatory Bowel Disease (IBD). The primary aim is to reduce inflammation and achieve remission. Common categories include aminosalicylates, corticosteroids, immunomodulators, and biologics. Aminosalicylates, such as mesalamine, are often used for mild to moderate symptoms. Corticosteroids are effective for short-term flare-ups but are not recommended for long-term use due to side effects. Immunomodulators like azathioprine help reduce the immune system’s activity, offering long-term control.

Biologics, such as infliximab and adalimumab, target specific components of the immune system and have shown significant promise in treating moderate to severe cases. Janssen IBD research has been pivotal in developing new biologics, improving treatment outcomes. Understanding these medication options can help individuals with IBD work with their healthcare providers to find the most effective treatment plan.

Dietary Management

Dietary management plays a crucial role in controlling IBD symptoms and improving overall health. While there is no one-size-fits-all diet for IBD, certain dietary adjustments can help manage flare-ups and maintain remission. Many individuals find relief by following a low-residue or low-fiber diet during acute phases, which helps reduce bowel movements and ease symptoms. Incorporating anti-inflammatory foods such as fatty fish, turmeric, and green leafy vegetables can also be beneficial.

Elimination diets, where specific foods are removed and then gradually reintroduced, can help identify potential triggers. Probiotics and prebiotics are gaining attention for their role in promoting gut health, though more research is needed. Consultation with a registered dietitian can provide personalized guidance. As ongoing IBD clinical trials continue to explore the impact of diet on IBD, future dietary recommendations may become more tailored and effective.

Surgical Interventions

For some individuals with Inflammatory Bowel Disease (IBD), surgical intervention becomes necessary when other treatments fail to provide relief. Surgery can offer significant benefits, particularly for those with complications such as strictures, fistulas, or severe inflammation that doesn’t respond to medication. Common surgical procedures for IBD include resection, where the diseased portion of the intestine is removed, and colectomy, which involves removing part or all of the colon.

In cases of ulcerative colitis, proctocolectomy with ileal pouch-anal anastomosis (IPAA) is often performed to create a new pathway for waste elimination. While surgery can dramatically improve quality of life, it is not a cure and may require ongoing medical management. Advances in laparoscopic and robotic surgeries have made these procedures less invasive, reducing recovery time and complications.

The Role of Clinical TrialsImportance of IBD Trials

Clinical trials are essential for advancing the treatment of Inflammatory Bowel Disease (IBD). They provide valuable insights into the effectiveness and safety of new therapies, paving the way for innovative treatments and potential cures. Participating in an IBD trial offers patients access to cutting-edge treatments that are not yet widely available. This can be particularly beneficial for those who have not responded well to existing therapies.

Janssen IBD clinical trials, for instance, have contributed significantly to the development of new medications and treatment protocols. These trials not only help in understanding the disease better but also in identifying the most effective strategies for managing it. By participating in these studies, patients play a crucial role in the fight against IBD, helping to improve future treatment options for everyone. Therefore, the importance of IBD trials cannot be overstated, as they are fundamental to the ongoing quest for better management and eventual cures.

How to Participate in Trials

Participating in IBD clinical trials can be a rewarding experience, offering access to new treatments and contributing to medical research. The first step is to consult with your healthcare provider to determine if a clinical trial is a suitable option for your condition. They can provide information on ongoing trials and help you understand the potential risks and benefits.

You can also search for trials through databases such as ClinicalTrials.gov, where you can find detailed information about the study’s purpose, eligibility criteria, and location. Once you identify a trial, you’ll undergo a screening process to ensure you meet the eligibility requirements. If accepted, you will be closely monitored throughout the study, with regular check-ins and assessments. Your participation can make a significant impact, helping to advance IBD research and improve future treatments.

Final Thoughts

Living with Inflammatory Bowel Disease (IBD) is undoubtedly challenging, but the variety of treatment options available offers hope and relief. Medications, ranging from aminosalicylates to biologics, provide tailored approaches to managing inflammation and achieving remission. Dietary management and surgical interventions are crucial components of a comprehensive treatment plan, with ongoing research continuously refining these strategies.

Clinical trials, such as those conducted by Janssen, are at the forefront of developing new therapies and improving existing ones. Participation in these trials not only gives patients access to groundbreaking treatments but also contributes to the broader understanding and management of IBD.

By staying informed about the latest treatment options and actively engaging with healthcare providers, individuals with IBD can take proactive steps towards managing their condition effectively. The future holds promising advancements in IBD treatment, driven by medical research and the dedication of those participating in clinical trials. Together, these efforts bring us closer to a world where IBD can be managed more effectively and, ultimately, cured.

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Regulatory Guidelines in the US

In the US, the Department of Health and Human Services (DHHS) is responsible for regulating clinical trials, with the support of two other organizations, namely Office for Human Research Protections (OHRP) and the United States Food and Drug Administration (USFDA). It is important to highlight that all investigators are required to comply with the regulations established by the aforementioned authorities, while conducting gene therapy focused clinical trials. According to the OHRP, clinical research involving human subjects should be necessarily reviewed and approved by the Institutional Review Board’s (IRB). Another DHHS agency, the National Institutes of Health (NIH), is responsible for conducting federally funded clinical trials, in compliance to a series of guidelines specified in the Code of Federal Regulations (CFR).

Further, based on the risks associated with the complex nature of gene therapy products and the required expertise, the Office of Cellular, Tissue, and Gene Therapies (OCTGT) at Center for Biologics Evaluation and Research (CBER) regulates gene therapy products (including medical device-based gene therapy products) and reviews applications related to the use of gene therapies under / intended for clinical investigation, or licensure. It is worth mentioning that the biologics which are being regulated under Section 351 of the Public Health Services Act are termed as 351 Products. Accordingly, such biologics (including gene therapy products) are bound to receive pre-market approval and fulfil the good manufacturing practices (GMP). Moreover, for gene therapy products intended for clinical investigation in human subjects, the concerned developers are required to file an Investigational New Drug (IND) application in the US.

Regulations Governing Gene Therapies in the US

The following section provides key takeaways related to the regulations governing the development and approval of gene therapies in the US:

• The manufacturers are responsible for analyzing all components of their gene therapy products in order to determine the application area. Additionally, the guidelines include preclinical, manufacturing, licensing and post-approval requirements, which are applicable to the product, or to specific manufacturing components; for instance, there are set of regulations governing the use of cell lines and adherence to current Good Tissue Practices (cGTPs).
• To determine which component of the gene therapy product is responsible for the therapeutic effect, the manufacturer may submit a Request for Designation (RFD) to the Office of Combination Products (OCP). This agency uses a codified decision-making algorithm to determine the primary mode of action of such therapies.
• The NIH guidelines for Research Involving Recombinant Synthetic Nucleic Acid Molecules contain information on responsibilities and procedures for conducting clinical investigations and an index of points, which needs to be considered for designing clinical protocols. Section IV-B-2-a-(1) of the guidelines calls for the use of an Institutional Biosafety Committee (IBC) to oversee the trial. The IBC is required to ensure patient and environmental safety, and evaluate the risk and safety associated with the use of the investigational agent and the overall conduct of the clinical trial.
• Only the USFDA has the authority to approve or deny a protocol for a trial in an IND application. In addition, it is the only organization that can make any legally binding decisions related to product identification, application or review determination of report submitted in product applications, including clinical investigations and supplemental materials.
• The investigator should anticipate and document all the risks associated with the delivery mechanism used for the gene therapy, based on the nature of the delivery vehicle (for instance, AAVs are known to induce inflammatory responses and lentiviral vectors have the potential to cause mutagenesis).
• All the gene therapy products should undergo bio-distribution testing to determine the expression of the vector and the impact of its translation products in both on and off-target cells and tissues, as well as the dosing regimen required to achieve the desired level of expression.
• According to the USFDA, manufacturers of investigational gene therapy products are required to submit a Quality Control (QC) Plan, along with the Chemical, Manufacturing and Control (CMC) portion of the IND, specifically designed for the oversight of production methods, in addition to standard quality control processes associated with GMP.
• As some of the gene therapy products may modify the genome of a host or alter the pre-therapy expression of a target through genetic intervention / manipulation, the potential for long-term risks must be carefully assessed. Patients receiving certain types of therapies should be monitored closely for delayed adverse effects, which are not evident during or soon after the clinical testing.
• Gene therapies are considered high-risk therapies and therefore, only serious clinical conditions should be considered for treatment using such ATMPs.
• In 2009, President Barack Obama signed the executive order 13505 (Removing Barriers to Responsible Scientific Research Involving Human Stem Cells), which allowed researchers to receive NIH funding and pursue research on prohibited cell lines.
• In 2016, the NIH made some changes in the aforementioned policy, which currently approves research involving the implantation of human pluripotent stem cells in animal embryos.

Guidance Document Framework for Gene Therapies

In 2018, the USFDA announced a new framework for the development, review and approval of gene therapies, which was complementary to the policy framework designed for regenerative medicines. Additionally, the USFDA released six scientific guidance documents to help the new gene therapy products in meeting the gold standards set for safety and effectiveness. The three drafted guidance documents have been mentioned below:

• Human gene therapy for hemophilia: The guidance document assists stakeholders who are involved in developing human gene therapy products for the treatment of hemophilia. Further, it provides recommendations on the clinical trial design and preclinical considerations for the development of new products. In addition, it helps in addressing the variations that may arise in factor VIII and factor IX activity assays. However, the draft does not include recommendations for products targeting hemophilia C.
• Human gene therapy for retinal disorders: The guidance document assists stakeholders who are engaged in the development of human gene therapy products for retinal disorders affecting both adult and pediatric population. Further, it provides recommendations on the development of product, preclinical testing and clinical trial design.
• Human gene therapy for rare diseases: The guidance document assists stakeholders who are engaged in the development of human gene therapy products for the treatment of rare diseases in both adult and pediatric population. Further, it provides recommendations regarding manufacturing, preclinical and issues related to clinical trial design. In addition, the draft helps in designing clinical trials for those products, where there is a limitation in the study population size, feasibility and other safety issues.

Furthermore, the USFDA has updated three existing guidance documents related to gene therapy manufacturing:

• CMC Information for Human Gene Therapy IND Applications: The draft provides recommendations to industry players regarding the CMC information necessary to assure product identity, quality, purity, strength, potency and safety of the investigational product.
• Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up: The draft provides recommendations to industry players on RCR testing during gene therapy products manufacturing and during follow-up monitoring of patients. In addition, it provides general guidelines, such as the amount of material to be tested and information on the testing methods.
• Long Term Follow-up after Administration of Human Gene Therapy Products: This draft helps in designing long term follow-up observational studies with specific considerations for different gene therapy products. Exposure of investigational gene therapy product for longer duration may lead to delayed adverse outcomes. Hence, long term follow-up observations are important to monitor the safety of gene therapy product. In addition, the document provides recommendations on patient monitoring for licensed gene therapy products.

It is worth mentioning that, in January 2020, the USFDA finalized the above-mentioned draft guidance documents focused on the development of gene therapies. This draft supersedes the CMC guidance that was released in April 2008. In addition, a new draft guidance was released, along with these documents, which focused on the USFDA process of interpreting similarity of two gene therapy products targeting the same indication, under the orphan drug regulations.

In January 2021, the USFDA released a new draft guidance document focused on gene therapies targeting neurodegenerative diseases. The drafted document has been highlighted below:

• Human gene therapy for neurodegenerative diseases: The draft assists stakeholders who are engaged in the development of human gene therapy products for the treatment of neurodegenerative diseases affecting pediatric and adult population. Further, it provides recommendations on the development of product, preclinical testing and clinical trial design. In addition, the draft helps in designing marketing approval pathways for new investigational gene therapy products.

During the same time frame, another guidance document was released by the USFDA related to the manufacturing of both licensed and investigational gene therapy products. The document featured the following considerations:

• Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency: The guidance document provides risk-based recommendations in order to minimize the transmission of infection during the development of therapy products. Further, it specifically highlights the manufacturing process of cell and gene therapy products and the consideration of source material (cells or tissues) for any contamination with coronavirus.

In September 2021, the USFDA issued a draft guidance providing an elaborative structure to study multiple versions of a cell and gene therapy product for a particular indication in a single early-phase trial. Brief description of the draft guidance document has been mentioned below:

• Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial: The draft assists the stakeholders to conduct umbrella trials for a single indication, wherein each candidate will have a unique IND application. Further, the early-phase clinical study will aid in selecting a suitable therapy candidate for later phases of development.

During the same month, the USFDA issued a final guidance to obtain an orphan drug designation and exclusivity to bring all the gene therapy products targeting rare diseases under the same umbrella. Brief description of the guidance document has been mentioned below:

• Interpreting Similarity of Gene Therapy Products Under the Orphan Drug Regulations: The guidance assists both industrial and academic stakeholders seeking orphan drug designation for gene therapies targeting rare diseases. It specifies the list of factors intended to be used by the USFDA in determining the orphan drug status for such products.

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