Mitochondrial Replacement Techniques (MRT)

Should the U.S. approve

Mitochondrial Replacement

Techniques/Therapies (MRT)

for human trials?

• Who has heard of MRT before?

• Does anyone remember what a mitochondria does?

Background

'Regular' DNA

v.

Mitochondrial DNA

We have 2 types of DNA in our cells?!?

... yes!

"Diseases"

Solution & Conclusion

(can occur a any age!)

Problem

• Developmental Delays
• Weakness and Fatigue
• Blindness and Deafness
• Dementia
• Strokes and Seizures
• Major Organ Failure
• Death

12,000 women in the U.S. have damaged mitochondrial DNA that prevents them from having their own genetically related child

(about 800 births a year)

+

1 in 5,000 children worldwide are born with mitochondrial diseases

+

150 children die every year in the U.K. from these diseases

• engage and educate the public
• conduct more animal trials (monitor generational effects)
• allow FDA to approve applications to begin human clinical trials (limited number of institutions; start with males*)
• more oversight than IVF + require results to be published
• limit MRT with federal regulations with principled guidelines interpreted by professional societies

Goal

• help women not suffer from multiple miscarriages
• allow them to have their own genetically related children
• eliminate these diseases from affecting more of the population & future generations

How to "MRT"

Current State of MRT

in the World

(Simplified terms!)

"Mitochondrial Replacement"

=

Replacing Bad Mitochondria

Short Video Showing the Two Techniques

• Step 1: take the woman's damaged mitochondria out of an egg
• Step 2: put a donor's healthy mitochondria into the egg
• Step 3: the end!
• healthy baby!
• no miscarriage!

MRT in the U.K.

Today in the U.S.

(scientific terms)

Mitochondrial Replacement Technique"s"

#1 Maternal Spindal Transfer

#2 Pronuclear Transfer

• 1st country in the World to legalize (02/2016)
• own regulatory body = HFEA
• HFEA grant's government licenses

• "limbo" (animal trials only)
• no authoritative body to govern human embryology and fertilization (so FDA has the power)
• application process
• FDA's February 2016 "rejection email" (Federal Budget excuse)
• Pressure & Prediction

Controversy

&

Ethical Dilemmas

Questions

#6

is it worth the risk?

#1

Child will have DNA from 3 people

• benefit a small % of the population today
• v. future good / harm
• interfering with nature, "playing God"? diseases are part of human fatality
• striving for human perfection
• doesn't affect the germline, just a transfer (no nuclear DNA involved)
• unknown side effects

• public perception / values
• how to educate the public

#5

#7

Should the Donor have any rights?

"Genetic Alternation" ?

• slippery slope
• 1st: mtDNA diseases ->
• 2nd: anxiety? ->
• 3rd: down syndrome? ->
• 4th: designer babies?
• disruption of social order
• wealthy may use it for aesthetics instead of medical purposes

• genetics of mitochondria give donor parental rights over the child (social and legal rights)
• less that 1% of total DNA = little effect on personality, physical traits
• it is like donating an organ

#2

100% success rate has not been guaranteed

• positive results on rats and monkeys for 15 years
• we might not be able to produce a healthy human
• do the benefits outweigh the risks?
• side effects will not be seen for generations

#4

Effects on the Human "Germline"

(will be passed down through generations)

• good: eliminate damaged mitochondria and diseases from future generations (benefits sustained)
• bad: if something went wrong between mtDNA and nuclear DNA = mistake will be passed down through generations (no reversal*)

#3

MRT could affect the personal identity of the child?

• confused/complicate how the child perceives itself (3 parents?)
• does mitochondrial DNA have an effect on identity though?
• 35 genes = <1% of genetic material
• few know mitochondria have genes or are in our cells = unlikely to effect social perception
• is any amount is significant?

• Who thinks the U.S. should allow human trials for MRT?
• Does anyone think the U.K. should have waited?
• Any other ethical issues you can think of?
• Would you do it / want your spouse to?

Thank you!