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Applied regenerative medicine assignment
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Applied regenerative medicine assignment for final year biomedical science undergraduate degree. First class assignment example on stem cell transplants.
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Assessment 1 Case Report.19002410Introduction
Treatment options for patients diagnosed with acute lymphoblastic leukemia (ALL) rely on their
efficacy and risk according to the age of the patient, stage of disease and side effects that may impact
on quality of life. As the patient is CD19 positive, it is of great importance that targeted
immunotherapies are specific to reduce the likelihood of relapse and increase the chances of success
and complete remission- considering the key causes of relapse following treatment which include the
loss of antigenic expression of CD19, high tumour burden and the role of t-regulatory cells that could
impact patient outcomes.
Advantages and limitations of CAR t-cell therapy
For patients up to the age of 25, several therapies using CAR t-cells have been approved and regulated
by NICE and the NHS in instances of relapse following unsuccessful hematopoietic stem cell therapy.
Although patients who exhibit CD19 positivity are eligible for CAR t-cell therapy, there is a lower
chance of success due to a higher loss of CD19 expression seen in 10-20% of patients with a
diagnosis of acute lymphoblastic leukemia compared to other types of cancer.
If CAR t-cell therapy is still deemed as suitable, clinicians should consider the patient’s
karnofsky score as requiring additional support for the patient to manage living with the challenging
side effects after treatment, including adequate support from family and social care services to ensure
that there is no subsequent loss of independence or daily function.
Although the patient’s ALL does not involve nerve function, side effects of CAR t-cell
therapy could trigger neurotoxicity that did not previously exist in the patient prior to treatment. The
cumulative effects of cytokine release syndrome (CRS) and neurotoxicity can be fatal, however the
rate of complete remission in patients after unsuccessful HSCT can extend to 90%, however relapse
remains a risk, relative to around 34% of patients. On the other hand, allogeneic donor derived CAR t-
cells can provide a promising alternative, although yield and quality of lymphocytes can be unreliable
through this method of treatment (Hua et al., 2020).
Monoclonal antibodies in treatment and maintenance therapy
If the patient reaches a stage of remission, blinatumomab may be used as maintenance therapy-
extending the duration of time before relapse. Specifically, as this patient has experienced relapse
from allogeneic stem cell transplantation, blinatumomab is shown to increase the length of survival
for patients who had received blinatumomab after allogeneic stem cell transplantation, increasing
from 4.3 to 10.6 months (Tambaro et al., 2020).
Table 1 Summary of suitable autologous and allogeneic treatments that would be suitable considering the patient's history,
relative to regulatory framework from professional bodies, side effects and success rates.
Treatment NICE criteria Side effects Success rate
CAR t-cell therapy For treating Neurotoxicity Complete remission=
relapsed ALL Cytokine up to 90%
In patients up release
to the age of syndrome
25 (CRS)
Blinatumomab- Cytokine
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