about stem cell treatment Everything

Know the facts!

Everything

about stem cell treatment

without determining their safety or efficacy as treatment options has led concerns regarding the overall well-being of the patients in general.

Therefore, it is of prime importance to protect patients from any harm due to unauthorized medical practice, in the form of stem cell treatment, based on accurate analyses and providing information on major public health issues that can be directly linked to general human health.

To address this health related issue, the Korean National Evidence-based healthcare Collaborating Agency (NECA) formed an expert panel of 18 members, including experts from the Korean Society for Stem Cell Research, National Research Institute of Health, Korea Research Institute of Bioscience and Biotechnology, Korea Institute of Drug Safety & Risk Management, and related organizations, as well as representatives from the general public. The aim of the panel was to hold a NECA round table conference, where an information booklet titled “Ask the right questions! Know the facts! Everything about stem cell therapy” was introduced to provide details to patients undergoing stem cell treatment or to the general public interested in knowing the essential facts about the entire process of stem cell treatment.

Ask the right questions!

Know the facts!

Everything about stem cell therapy

Part 04 If you want a safe stem cell treatment, confirm and reconfirm!

• What information does the patient need to know when receiving a stem cell treatment?

• Does the patient need to pay for stem cell treatment(s) that has not been approved yet?

• When stem cell treatment is recommended, what should be checked first?

Part 05 Stem cell therapy (drug): adverse effects and risks

• Is the approved stem cell therapy (drug) safe and have no adverse effects?

• What are the common adverse effects of stem cell therapy (drug)?

• What are the specific risks associated with injecting stem cells harvested from one’s own body (autologous stem cells)?

• What are the specific risks associated with injecting stem cells harvested from someone else?

• How long does the safety and efficacy of the stem treatment last?

Part 06 Frequently asked questions

Q1 What are the differences among stem cell therapeutic agents, therapies, procedures, injections, injection treatment, and treatment techniques?

Q2 What are the differences in usefulness, efficacy, and validity of using stem cell therapy?

Q3 What are the differences among clinical study, clinical experiment, and clinical trial?

Q4 What methods are available for verifying which stem cell therapy or therapeutic agent has received official approval for use in Korea?

Q5 What options do I have if stem cell treatment is ineffective or cause adverse effects?

Q6 What happens to hospitals that perform unauthorized stem cell treatments?

Q7 Can I be compensated if I suffer adverse effects due to unauthorized stem cell treatment?

Q8 Can I believe someone who claims to have developed a revolutionary stem cell treatment method on their own?

Part 01 Stem cells: definition and types

• What are stem cells?

• What are the different types of stem cells?

• What are the types of stem cells that can be directly used to treat patients?

Part 02 Understanding stem cell therapy for the selection of appropriate

• What are the differences between stem cell therapeutic agent and stem cell therapy?

• What developing stages and approval processes does a stem cell therapeutic agent need to go through?

• What developing stages and approval processes does a stem cell therapy go through?

Part 03 Stem cell treatment at the pre-clinical trial stage: everything the

• What is a clinical trial?

• Why is it mandatory to fill out a clinical trial patient consent form?

• What information must a proper patient consent form contain?

• What other details should be checked besides the patient consent form?

Fertilized egg Embryo

Blastocyst

Inner cell mass

Embryonic stem cell (ESC) Induced pluripotent stem cell (iPSC)

Patient’s somatic cells Reverse differentiation/Reprogramming Pluripotent stem cell (PSC)

Stem cells inside the body tissues

Bone marrow Umbilical cord

Adult stem cells Mesenchymal stromal cells

Hematopoietic stem cells

Brain Liver Pancreas

Skin Adipose

Types of stem cells

Stem cells are undifferentiated cells that are capable of growing and differentiating into various body tissues, such as the bones, muscles, heart, brain, etc., depending on the requirement of the body. A traditional concept of stem cells categorizes these cells as: embryonic stem cells (originating from a fertilized egg after the union of a sperm and an egg) and adult stem cells (present in body tissues, such as in the bone marrow). Recently,

induced pluripotent stem cells with similar properties and pluripotency as that of the human embryonic stem cells have been generated via cell reprogramming.

Stem cells:

definition and types

Stem cell therapeutic agent Stem cell therapy

Definitions “Drug” that uses stem cells “Medical practice or treatment regime” involving minimal manipulation of patient -derived tissues that is reintroduced in the patient

Related laws Pharmacy law Medical law

Development Process Animal trials → Phase I clinical trial → Phase II clinical trial → Phase III clinical trial

Research focusing on the safety and efficacy of such therapy, involves clinical trials carried out under the supervision of physicians

Approval Process Approved as a new drug, when its safety and efficacy are confirmed based on the outcome of phase III clinical trial

Approved as a new medical technology when the safety and efficacy are confirmed through multiple medical journal reviews of manuscripts reporting the outcomes of using the particular technology

Review/Approval

Agencies Korea Ministry of Food and Drug Safety Committee of New Health Technology Assessment, Korea Ministry of Health and Welfare

Treatment Costs

• Treatment costs are not payable by the patients during the development process

• Patients are responsible to bear the cost of treatments that uses approved stem cell therapeutic agents

• Treatment costs are not billed until the procedure is recognized as a new medical technology (exception: only payable in the case of cosmetic surgeries for aesthetic purposes)

• The patient is responsible for the treatment costs only after the procedure has been approved as a new medical technology

While ‘Stem cell therapeutic agent’ refers to a “drug/agent” used, ‘stem cell therapy’ refers to the “treatment regime/procedure”. Therefore, there are differences in their respective development, review, and approval processes.

Understanding stem cell therapy for

the selection of appropriate stem cell

therapeutic agent

Is the concerned treatment still at the clinical trial stage?

3

What is the cost of the treatment?

4

Have you been recommended

stem cell treatment earlier?

Consider asking your physician these following questions:

It is not at the clinical trial stage.

(You can find the corresponding information from KMFDS’ website (http://ezdrug.mfds.go.kr)

regarding approved new drugs)

The patient must pay for the treatment.

Private health insurance may cover the treatment expenses.

It is not at the clinical trial stage.

(You can find the corresponding information from the website of Committee of New Health Technology Assessment (http://nhta.neca.re.kr), the nHTA Report section, regarding approved

new health technologies)

The patient must pay for the treatment.

Private health insurance may cover the treatment expenses.

It is at the clinical trial stage Research funding should cover the treatment cost (patient does not pay for the treatment).

The treatment is not covered by health insurance.

Treatment cost cannot be billed to the patient Although it can be claimed as being in

clinical trial stage, it is still an unauthorized treatment since it did not go through the official

approval process.

It is at the clinical trial stage. Research funding should cover the treatment cost (patient does not pay for the treatment).

The treatment is not covered by health insurance.

Is it a stem cell therapeutic agent or stem cell therapy?

1

Is the concerned treatment at the developmental stage or has it already been

approved for use?

2

If it is not relevant to the above,

this is an unauthorized agent or therapy! It has not undergone any approval procedure, so its safety and efficacy have not been verified.

Has the procedure been developed and received necessary approval as

a new health technology?

Has the agent developed and received necessary approval from the Korea Ministry of Food and Drug Safety (KMFDS)?

Is it a stem cell therapeutic agent? Is it at the developing stage?

Is it a stem cell therapy? Is it at the developing stage?

Performing stem cell therapy that has not been approved in Korea to treat patients, as a part of clinical trials, is regarded as

“unauthorized medical practice,” and considered illegal. The patient is not required to pay for any cost associated with the “clinical trials” without the final approval or as a part of an “unauthorized medical practice.”

When a patient is recommended by his/her physician to undergo stem cell treatment, the following details should be checked.

No

No

No

Yes Yes

Yes

Yes

Yes Yes

Yes

Stem cell therapy (drug):

adverse effects and risks

In general, stem cell therapy (drug) is susceptible to various risks associated with changes in biological properties of the cells and incorporation of foreign substances that are obtained from in vitro processing. Even approved stem cell therapy (drug) may have adverse effects, and its safety may vary in patients depending on the method of administration and dose, as well as the patient’s condition. Therefore, it is necessary to consult a physician in charge regarding the treatment effects (benefits) versus the risks associated with the treatment process.

A clinical trial involves research that directly use a therapeutic agent, therapy, or therapeutic device (under development) to treat patients in order to verify the safety and efficacy based on scientific means. Therefore, it is important to

understand that a new therapeutic agent or therapy at the clinical trial stage should not be considered a proven treatment method.

Patient consent form for a clinical trial must contain the following information:

Stem cell treatment at the pre-clinical trial stage:

everything that the patient needs to know

Patient consent form for participation in a clinical trial must contain the following information to ensure that the patient does not have inflated expectations about the new stem cell therapeutic agent that is being used as a part of the clinical trial.

• The fact that a clinical trial stage is experimental in nature; the fact that the therapy (drug) to be administered during the trial has aspects that have not been proven yet;

and also the fact that the trial is attempting to find out

• Inherent risks associated with the therapy (drug) and any risks or discomfort that may arise from participation in the clinical trial

• Explanation regarding potential patient benefits from participating in the clinical trial;

explanation even in the absence of such benefits

• Explanation regarding other medical treatment(s) available ( if the patient is unwilling to participate in the clinical trial), the potential risks, and benefits associated with the alternate treatment method(s)

Clinical trials must include explanations regarding the steps involved in investigating the safety and efficacy of the new stem cell therapeutic agent.

• What is the therapeutic agent (drug) that is being studied?

• How long does the trial last?

• How many patients are expected to participate in the trial?

• If the trial is designed as a randomized controlled study, what is the probability of receiving different treatment (placebo or alternative treatment) besides the intervention drug?

• Procedures involved and conditions of the patients must comply with the trial, such as blood collection, before, during, and after the trial.

The following must also be included to protect patient rights of those participating in the clinical trials.

• Who is conducting the trial?

• In case of clinical trial-related injury, what are the compensation and treatments that the patient is entitled to?

• Detailed contact information of person(s) and responsible agencies related to patient rights

• Responsibilities of the patient as a participant in the clinical trial; information regarding individuals who may access patient’s trial/medical data; information related to patient rights for the protection of confidentiality

• The fact that personally identifiable information of the patient will be kept confidential even when the outcomes from the clinical trial are published

• The fact that the patient has the right to receive any new information that may influence his or her willingness to remain as a participant in the clinical trial

• Situations that the patient’s participation in the trial may be terminated

• The fact that the patient has the right to unconditionally withdraw from participating in the trial

• Expected costs associated with the participation of the patient in the clinical trial

• If the patient is entitled to monetary compensation for participating in the clinical trial,

what is the expected amount; the fact that such amount may be adjusted according to

the level and duration of participation in the clinical trial

Ask the right questions!

Know the facts!

Everything about stem cell treatment