Stem Cell Therapies for Childhood Apraxia of Speech

Stem Cell Therapies for Childhood Apraxia of Speech

A note from the Apraxia Kids Professional Advisory Council:

Stem Cell Therapies for Childhood Apraxia of Speech. The potential for stem cell therapies is exciting, but few stem cell therapies have proven safe and effective at this time. There are also many stem cell clinics that market unproven stem cell therapies to families without supporting evidence. Read this important article for tips on how to tell the difference between doctors conducting actual registered clinical trials from clinics selling unproven interventions.

 

Many parents today have the cord blood of their newborns collected at birth and stored for the possibility of later use. Why do some parents do this? The cord blood has been found to be rich in precursor cells known as stem cells. Stem cells are “multipotent”, which means these cells retain the ability to develop into the specialized cell types found in an adult body: blood, skin, muscle, nervous system cells, bone, etc. The hope is that these pluripotent cells may be used to generate healthy tissue which would either replace or help repair tissue damaged by injury, disease, or aging.

The potential for stem cell therapies is exciting. Stem cells therapy is promising. The potential for treatment is evident in diseases such as childhood leukemia, where stems cells have contributed to saving thousands of lives. Stem cell have been used for the development of tissue grafts to treat injuries to skin, bone, and corneal injuries and this has shown real promise. Currently, there are many other conditions for which stem cell therapies are being studied using clinical trials—an important type of research for examining intervention effectiveness.

However, there has been a proliferation of stem cell clinics which market unproven stem cell therapies directly to consumers without supporting evidence from controlled, randomized, independent clinical trials. “Although promising in theory, so far very few stem cell therapies have proven to be safe and effective.” (1). The list of disease for which stem cell treatments have a proven efficacy remains small. The stem cell industry suffers from the lack of weak laws and enforcement and regulatory oversight.  Therefore, businesses marketing unproven stem cell therapies have been the focus of criticism and discussion.

It can be difficult for patients and families to tell the difference between doctors conducting actual registered clinical trials from clinics selling unproven interventions. An experimental therapy offered for sale is different from a clinical trial. Trustworthy clinical trials have the following characteristics:

  • Physicians and other medical therapists often refer suitable candidates for a clinical trial. Clinical trials have oversight of an independent medical ethics committee to protect the rights of the participants.
  • Clinical trials are based on existing research data that indicates that the treatment being proposed is likely to be safe and the therapy has a theoretical and/or research-basis that suggest it could be effective.
  • Clinical trials are designed in such a way to answer specific questions about the proposed therapy.
  • Participants of a clinical trial are not responsible for the costs of the therapy (although they may be responsible for the costs of travel or accommodations).
  • A clinical trial is subject to conformity to regulatory requirements and will be listed in an easily recognized clinical registry such as https://clinicaltrials.gov.

In contrast, clinics which offer unproven stem cell interventions market directly to the public through the Internet and social media. Clinics in the business of selling unproven stem cell therapies will often use patient testimonies to support their claims. The problem with patient testimonies is that they can be misleading. Improvement, perceived or actual, can occur for a variety of reasons unrelated to the proposed therapy. Patients may improve because of a natural variation in the condition, ancillary treatments, lifestyle and dietary changes, or even a strong belief that the therapy will help. Since there is no carefully designed clinical trial to adjust for such improvements, it is impossible to state whether such a treatment is in fact beneficial.

Unproven therapies come with numerous risks. Out-of-pocket expenses can be considerable, yet private and government health insurances are unlikely to cover experimental therapies. There can be travel which can take time away from other family members. Even the use of one’s own stem cells for transplant, also known as “autologous transplant”, comes with additional risks.  Although an immune reaction is unlikely in autologous stem cell transplants, there is the risk that the cells will end up in places in the body they do not belong. Manipulation of the cells during collection and storage can potentially alter the normal function of the cells and introduce contamination by viruses and bacteria. The use of stored cord blood can make it unavailable for future uses. Also, the use of stem cell therapy can make one ineligible for future clinical trials

Clinical trials are designed to determine effectiveness of treatment and minimize harm. Unfortunately, there are no current clinical trial designed to determine the efficacy or safety of use of stem cell therapies specifically in children with Childhood Apraxia of Speech. As parents of children with CAS, we understand the desire “to do everything possible” to help our children. Stem cell research is making great progress in understanding how stem cells work and their potential use for future medical therapy, including possibly CAS. However, we encourage parents to thoroughly investigate any proposed stem cell interventions, ask probing questions, and to review the information with their child’s pediatrician or other health care expert before embarking on unproven therapies.

 

REFERENCES

Stem Cell Therapies for Childhood Apraxia of Speech

A note from the Apraxia Kids Professional Advisory Council:

Stem Cell Therapies for Childhood Apraxia of Speech. The potential for stem cell therapies is exciting, but few stem cell therapies have proven safe and effective at this time. There are also many stem cell clinics that market unproven stem cell therapies to families without supporting evidence. Read this important article for tips on how to tell the difference between doctors conducting actual registered clinical trials from clinics selling unproven interventions.

 

Many parents today have the cord blood of their newborns collected at birth and stored for the possibility of later use. Why do some parents do this? The cord blood has been found to be rich in precursor cells known as stem cells. Stem cells are “multipotent”, which means these cells retain the ability to develop into the specialized cell types found in an adult body: blood, skin, muscle, nervous system cells, bone, etc. The hope is that these pluripotent cells may be used to generate healthy tissue which would either replace or help repair tissue damaged by injury, disease, or aging.

The potential for stem cell therapies is exciting. Stem cells therapy is promising. The potential for treatment is evident in diseases such as childhood leukemia, where stems cells have contributed to saving thousands of lives. Stem cell have been used for the development of tissue grafts to treat injuries to skin, bone, and corneal injuries and this has shown real promise. Currently, there are many other conditions for which stem cell therapies are being studied using clinical trials—an important type of research for examining intervention effectiveness.

However, there has been a proliferation of stem cell clinics which market unproven stem cell therapies directly to consumers without supporting evidence from controlled, randomized, independent clinical trials. “Although promising in theory, so far very few stem cell therapies have proven to be safe and effective.” (1). The list of disease for which stem cell treatments have a proven efficacy remains small. The stem cell industry suffers from the lack of weak laws and enforcement and regulatory oversight.  Therefore, businesses marketing unproven stem cell therapies have been the focus of criticism and discussion.

It can be difficult for patients and families to tell the difference between doctors conducting actual registered clinical trials from clinics selling unproven interventions. An experimental therapy offered for sale is different from a clinical trial. Trustworthy clinical trials have the following characteristics:

  • Physicians and other medical therapists often refer suitable candidates for a clinical trial. Clinical trials have oversight of an independent medical ethics committee to protect the rights of the participants.
  • Clinical trials are based on existing research data that indicates that the treatment being proposed is likely to be safe and the therapy has a theoretical and/or research-basis that suggest it could be effective.
  • Clinical trials are designed in such a way to answer specific questions about the proposed therapy.
  • Participants of a clinical trial are not responsible for the costs of the therapy (although they may be responsible for the costs of travel or accommodations).
  • A clinical trial is subject to conformity to regulatory requirements and will be listed in an easily recognized clinical registry such as https://clinicaltrials.gov.

In contrast, clinics which offer unproven stem cell interventions market directly to the public through the Internet and social media. Clinics in the business of selling unproven stem cell therapies will often use patient testimonies to support their claims. The problem with patient testimonies is that they can be misleading. Improvement, perceived or actual, can occur for a variety of reasons unrelated to the proposed therapy. Patients may improve because of a natural variation in the condition, ancillary treatments, lifestyle and dietary changes, or even a strong belief that the therapy will help. Since there is no carefully designed clinical trial to adjust for such improvements, it is impossible to state whether such a treatment is in fact beneficial.

Unproven therapies come with numerous risks. Out-of-pocket expenses can be considerable, yet private and government health insurances are unlikely to cover experimental therapies. There can be travel which can take time away from other family members. Even the use of one’s own stem cells for transplant, also known as “autologous transplant”, comes with additional risks.  Although an immune reaction is unlikely in autologous stem cell transplants, there is the risk that the cells will end up in places in the body they do not belong. Manipulation of the cells during collection and storage can potentially alter the normal function of the cells and introduce contamination by viruses and bacteria. The use of stored cord blood can make it unavailable for future uses. Also, the use of stem cell therapy can make one ineligible for future clinical trials

Clinical trials are designed to determine effectiveness of treatment and minimize harm. Unfortunately, there are no current clinical trial designed to determine the efficacy or safety of use of stem cell therapies specifically in children with Childhood Apraxia of Speech. As parents of children with CAS, we understand the desire “to do everything possible” to help our children. Stem cell research is making great progress in understanding how stem cells work and their potential use for future medical therapy, including possibly CAS. However, we encourage parents to thoroughly investigate any proposed stem cell interventions, ask probing questions, and to review the information with their child’s pediatrician or other health care expert before embarking on unproven therapies.

 

REFERENCES



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