4 - Advanced Therapy Medicinal Products (ATMPs) Flashcards
ATMPs
- Biological medicines for human use that are based on genes, cells or both
- Some contain GMOs
What is considered a biological therapeutic good
- Cell-based products (including genetically modified and non-minimally manipulated cells)
- Immunotherapy products containing human cells
- Combination products (e.g. cell therapy and medical device)
- Products that comprise or contain live animal cells, tissues or organs (e.g. pancreatic islet cells isolated from pigs)
- Autologous human cells and tissue products (including stem cells)
Definition of biologicals
A thing made from, or that contains, human cells or human tissues, and that is used to treat or prevent disease
Biological medicines
- Recombinant products
- Plasma derived products
- Vaccines (that do not contain viable human cells)
- Gene-therapy vectors alone
ATMPs vs tissue/cell transplantation
- Distinction depending on how the tissue or cells are processed and used clinically
- ATMPs have been substantially manipulated or not intended for the same essential function in the recipient and the donor.
- In contrast, tissue or cellular preparations that are not subjected to substantial manipulation and are used for the same essential function are not ATMPs.
Autologous stem cell transplantation process
- Stem cell mobilisation
- Collection of stem cells
- Processing
- Conditioning and treatment
- Reinfusion back into patient
Three examples of blood cancers and type of transplantation used
- Multiple Myeloma (autologous stem cell transplantation)
- Hodgkin and Non-Hodgkin Lymphoma (autologous stem cell transplantation)
- Acute Myeloid Leukemia (allogeneic stem cell transplant)
Serum eye drops for dry eye syndrome
- Due to SLE, age, injury etc
- Resembles tears
- Composed of water, salts, proteins, lipids, growth factor
Example of autologous gene modified cell therapies
Car T cell therapy for cancer patients
CAR T cell therapy process
- Leukapheresis
- T cell activation/transduced with viral vector (chimeric antigen receptor)
- Modified T cell expansion
- Chemotherapy
- Modified T cell infusion
Chimeric antigen receptor (CAR)
Specificity of antibody combined with function of T cell
Prevalence of melanoma
- 4th most commonly diagnosed cancer in Australia
- 11th most common cause of cancer deaths
Why are solid cancers harder to treat than liquid (blood) cancers
As cancer cells are not easily approachable by T cells which normally eliminate them
Tumour infiltrating lymphocytes (TILs) for melanoma
- Tumour excision
- TIL isolation and initial expansion
- Screen TILs for autologous tumour activity
- Rapid expansion of selected TILs (and stimulated with IL-2)
- Reinfusion after host preconditioning
TILS in melanoma
Recognise melanoma tumour but because of microenviormmet of tumour TILs are arrested and thus become inactive