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Addressing Unmet Clinical Needs in Future Medicine

 

I need to answer all the following sections  A , B ,C  mention everything in  under the A , B ,C section Which means this (i) , (ii) ,(iii) ,(iv) , (v) which means each section have points that i need to include in my essay

Future medicene
Here is what is required :
(A) Outline TWO unmet clinical needs that could be addressed by your technology.
Each of the following points should be covered and explained clearly, concisely and accurately to give a balanced and informative overview of the clinical need:
(i) Inclusion of two distinct appropriate examples of unmet clinical need. These could be diseases or another need that the technology could be applied to, eg better diagnostics. Dependent on the technology, a specific disease (eg cystic fibrosis or a broad disease class (eg solid tumors) could be chosen.
(ii) Explanation of disease aetiology, including appropriate molecular detail eg gene names.
(iii) Explanation of disease progression and consequences, with statistics where relevant.
(iv) Discussion of epidemiology in UK and internationally, with indication of disease burden and scale of clinical need, and statistics where relevant.
(v) Explanation of current therapeutic approaches and discussion of their limitations. Depending on the disease, these could vary from no current curative treatment to a range of good therapies that could still be improved due to unpleasant side effects or poor compliance.
(B) Describe how distinct technologies within your chosen technology area could be used to address these needs.
Clear explanation of how approaches from within the technology area could be applied to each unmet clinical need. For a 2:1 or above, students should cover distinct approaches for each clinical need (eg siRNA and CRISPR, or microneedles and 3D printing). The following points should be addressed:
Explanation of how the technology works. This should include a clear and accurate summary of how this specific technological approach would be used to treat this specific clinical need, with appropriate mechanistic detail, but
(i) should be concise and focused on the topic being addressed.
(ii) Explain any variables in how this approach could be applied to this clinical need, for example delivery method or specific design choices (eg ASO modification), and which of these variables is most suitable/promising, with rationale for this choice. If several approaches are promising you can summarise this and explain why.
(iii) Give an accurate overview of clinical research and development to date.
Depending on the level of research this does not need to cover everything that fias been done, but should summarise the main approaches taken, the stage of development (eg pre-clinical, or specific clinical trial stage) and give an accurate overview of how successful this approach has been in research to date, with statistics if appropriate.
(C)Describe the barriers to clinical adoption of your chosen technology for each of these needs.
Balanced and critical overview of the likelihood of each of the chosen technological approaches becoming a useful approach to each clinical need in future. Exactly what is covered here will be very individual to the topic, and students should use their judgment to discuss the barriers that are relevant to the specific topics they have covered. As a general guideline, this answer should include:
(i) Consideration of any relevant barriers to your approach being used in clinical practice. These may include the practicalities of development and production, delivery, efficacy, cost, ethics, as possible examples, but the relevant barriers will be very specific to an individual approach. Discussion of generic barriers that are not relevant to this particular application will be marked down.
(ii) Discussion of any alternative approaches to the clinical keed that are also undergoing research or development and how these may influence the adoption of your chosen technological approach, where relevant.

 

Sample Answer

 

Addressing Unmet Clinical Needs in Future Medicine

(A) Unmet Clinical Needs

(i) Examples of Unmet Clinical Needs

1. Alzheimer’s Disease:

– Aetiology: Alzheimer’s disease is characterized by the accumulation of amyloid-beta plaques and tau tangles in the brain, affecting neuronal function and leading to cognitive decline.
– Progression and Consequences: Alzheimer’s is a progressive neurodegenerative disease, with symptoms worsening over time, ultimately resulting in memory loss, impaired reasoning, and challenges in daily functioning.
– Epidemiology: Alzheimer’s disease affects millions worldwide, with a significant burden on healthcare systems and caregivers due to the increasing aging population.
– Therapeutic Approaches: Current treatments for Alzheimer’s focus on symptom management and have limited efficacy in slowing disease progression.

2. Pancreatic Cancer:

– Aetiology: Pancreatic cancer often arises due to genetic mutations, such as KRAS, p53, and BRCA2 alterations, leading to uncontrolled cell growth in the pancreas.
– Progression and Consequences: Pancreatic cancer is aggressive, with a low survival rate as it is often diagnosed at advanced stages when treatment options are limited.
– Epidemiology: Pancreatic cancer has a poor prognosis, with high mortality rates globally and limited effective treatment options available.
– Therapeutic Approaches: Current treatments for pancreatic cancer include surgery, chemotherapy, and radiation therapy, but outcomes remain suboptimal due to late-stage diagnosis and tumor aggressiveness.

(B) Technological Approaches to Address Unmet Needs

Alzheimer’s Disease:

1. Gene Therapy Targeting Amyloid-beta Production:

– Explanation: Utilizing gene therapy to target and reduce amyloid-beta production in the brain, potentially slowing disease progression.
– Variables: Delivery method optimization is crucial for effective gene therapy penetration into the brain. Non-viral vectors may offer safer delivery compared to viral vectors.
– Clinical Research: Pre-clinical studies have shown promising results in reducing amyloid-beta levels in animal models, with ongoing research focusing on safety and efficacy.

2. Nanotechnology for Drug Delivery:

– Explanation: Utilizing nanocarriers to deliver therapeutic agents across the blood-brain barrier for targeted treatment of Alzheimer’s disease.
– Variables: Surface modification of nanoparticles for enhanced brain penetration and controlled drug release may enhance therapeutic efficacy.
– Clinical Research: Nanotechnology-based drug delivery systems are in pre-clinical stages, showing potential for improving drug bioavailability and targeting brain pathology.

Pancreatic Cancer:

1. CRISPR-Cas9 Gene Editing for Targeted Mutation Correction:

– Explanation: CRISPR-Cas9 technology can target specific genetic mutations in pancreatic cancer cells, potentially inhibiting tumor growth.
– Variables: Optimizing CRISPR delivery to pancreatic tumors and minimizing off-target effects are crucial for clinical translation.
– Clinical Research: CRISPR-based therapies are in early stages of development, showing promise in pre-clinical models for correcting cancer-associated mutations.

2. Immunotherapy Using CAR-T Cells:

– Explanation: Chimeric antigen receptor (CAR) T-cell therapy can harness the immune system to target pancreatic cancer cells specifically.
– Variables: Improving CAR-T cell persistence and tumor infiltration for enhanced anti-tumor efficacy is a key focus for clinical translation.
– Clinical Research: CAR-T cell therapies are under investigation in clinical trials for pancreatic cancer, showing potential for inducing durable responses in some patients.

(C) Barriers to Clinical Adoption

Alzheimer’s Disease:

1. Barriers:

– Practicalities of gene therapy delivery to the brain pose challenges.
– High cost of gene therapy development and production may limit accessibility.
– Ethical considerations regarding genetic manipulation in neurodegenerative diseases.

2. Alternatives:

– Immunotherapy targeting tau protein aggregation in Alzheimer’s research.
– Stem cell-based therapies for neuronal regeneration in Alzheimer’s treatment.

Pancreatic Cancer:

1. Barriers:

– Efficacy of CRISPR-Cas9 gene editing in complex pancreatic tumor microenvironment.
– Cost and scalability of CAR-T cell manufacturing for widespread clinical use.
– Ethical concerns regarding genetic modifications in cancer treatment.

2. Alternatives:

– Targeted chemotherapy advancements for pancreatic cancer.
– Combination therapies integrating immunotherapy with standard treatments for improved outcomes.

In conclusion, addressing unmet clinical needs in future medicine requires innovative technological solutions tailored to specific diseases like Alzheimer’s and pancreatic cancer. Overcoming barriers to clinical adoption and exploring alternative approaches will pave the way for transformative advancements in treating these challenging medical conditions.

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