The Pediatric AIDS Coalition Cause Education Committee

1. Modern Drug Treatments for
HIV: Potential Cures
Saskia Leonard and AnnaTong

2. Why look for a cure?
Timothy Ray Brown, also known as the "Berlin patient," was the first person to be cured
of HIV infection, more than a decade ago.
- HIV is not a death sentence anymore with daily intake of medication
- But only 21 million of 37 million people have access to drug therapy
- Lower-income countries have lower access to consistent/wide spread treatment
- Drug resistant virus

3. Current Case
- Now researchers are reporting a second patient has lived 18 months after
stopping HIV treatment without sign of the virus following a stem-cell
transplant.
- Referred to as “London Patient”
- Was suffering from Hodgkins Lymphoma and was infected with HIV
- Received bone marrow cells as part of his cancer treatment in 2016 from a
donor who had a malfunctioning CCR5 gene, which creates a protein crucial
for HIV to invade white blood cells
- Key is to shut down the CCR5 gene

4. Why is this exciting?
- Big news for the research community looking at how
gene therapy can disable the CCR5 gene by using
other technologies including the gene editing technology CRISPR
- Proof that HIV is curable
- “The hope is that this will eventually lead to a safe, cost-effective and easy
strategy to achieve these results using gene technology or antibody
techniques.”
- Informs broader community- in the news!

5. Why is it problematic?
- Not readily available to do a stem-cell transplant
- Can have severe side effects
- Stem cell transplants are risky
- Donors must be a genetic match to recipients and very few
people naturally carry two copies of the disabled CCR5 gene

6. Genome Editing - A Controversial Case
● Chinese scientist who made the world’s first genome edited twin babies: He Jianuki
○ Impregnated a woman with babies who lacked the gene utilized by HIV to infect cells
○ The editing was successful and only the target gene was edited
● Used CRISPR-Cas9 genome-editing tool to disable CCR5 - protein used by HIV to enter T-cells
● Father HIV-positive

7. Ethical Implications of Gene Editing
● There are other safe and effective ways to use genetic editing for HIV prevention
○ No unmet medical need that gene editing fulfills
● “Directly jumping into human experiments can only be described as crazy”
● Marginalizes already marginalized groups - very expensive
● Where to draw the line in terms of gene editing
● Incredibly risky- may target other genes inadvertently and may affect other normal functioning
● Some strains of HIV do not even use this protein to enter the cells, but use another called
CXCR4

8. Other advancements in treatment
- Conference on Retroviruses and Opportunistic Infections making progress on treatment especially in Africa
- Monthly injection of HIV drugs suppress the virus the same amount as daily pills
- Good because many people forget to take their pills daily.
- Doctors working in impoverished countries are eager for injections or implants that will release small daily
doses of antiretroviral drugs because the devices can be used in secrecy.
- Providing injections may be harder than handing out pills, but the option may attract patients with H.I.V. who
would otherwise stay away because of
- Fear and stigma
- Contraceptive injections are favorable and more effective because many women have to hide birth
control from their partners who might get angry that they don’t want more kids
- Gay or bisexual men may want to be discreet, because often hiding from spouses or families that they
have sex with men
- Truveda , PrEP pill, taken every day are almost 100% protected against getting HIV from unprotected sex or
drug injection
- $20,000/year and hard to get it to uninsured in the US
- Discovy is an alternative to Truveda that is cheaper but more likely to raise cholesterol, which is problematic