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Having a chronic, manageable disease
is not that great, if you’re the one
with the disease.
Worldwide, less than half of the very
sickest of the 34 million people with
HIV/AIDS can access treatment.
Global Funding Pulling
Back?
Sequestration Budget
Cuts
Uncertain Future for the
AIDS Drug Assistance
Program
We need a cure.
What we expected when we started ...What we expected when we started ...
Abundant Resources!
Impossible
Research
Obstacles!
What We FoundWhat We Found
Few
Resources
Great Research !!!
Proof of concept:
The Berlin Patient was cured in
2007.
• His therapy cost $200,000, or about 1/3
of the lifetime cost of a treatment for a
person with AIDS in the United States.
• His cure is leading to safer cure
research alternatives, using a patient’s
own immune cells.
• Too dangerous: The chance of dying
from this treatment is 30%.
The ObstaclesThe Obstacles
The National Institutes of Health spent $56.4 million on AIDS cure research
and $557 million on AIDS vaccine research in 2012.
Source: NIH (for cure statistic) and IAVI (for vaccine statistic)
is that a big enough slice for you?
We learned that the
NIH is spending only
3% of its AIDS
research budget to
find a cure. We need
more than that.
We’re going to need
a bigger pie.
WE DEMANDED
MORE.
We sent 500 letters to Frank Collins, head of
the National Institutes of Health
We got big stories and op-eds in the media
We held frank meetings with top NIH officials.
The NIH found $27 million in new money for
cure research.
We aren’t finished.
Cool Developments in
The Search for a Cure for
AIDS
Cure strategies being investigated (no
guarantees):
•Flush out latent virus in viral reservoirs and kill it
•Replace your immune system with somebody else’s
•Make AIDS-proof cells that take over your body
•Treat hard and very early, stay on treatment for a few
years, then maybe you can go off drugs but stay
undetectable (but maybe not)
The Visconti Cohort
Visconti Cohort: Treated early,
now control AIDS
14 patients (out of a larger group of 75)
who started potent combination ART
within 10 weeks (median 40 days) after
HIV infection—during acute infection.
They stayed on treatment for at least 1
year (median 3 years). Then they left
treatment.
VISCONTI patients appear to have
controlled HIV, with viral load (less than
50 copies/mL), for a median of 76
months, or more than 6 years, after
treatment interruption. Without drugs.
Lesson of the VISCONTI
Cohort
It may be possible to push 15% of newly infected
people into remission (HIV-infected but don’t
need AIDS drugs to control the disease—the
body’s immune system controls it) just by
treating people early enough after they are
infected.
This might be because their bodies haven’t had
time to develop a viral reservoir. (Researchers
are still figuring this out.)
German man
(not the Berlin Patient, another guy)
CURED?
• A German man, infected in 1999, started
HIV treatment within three months of
diagnosis, when his viral load was over one
million. He quit AIDS meds in 2004, and after a
very brief viral rebound has remained
undetectable ever since.
• Reported at IAS2013 in Kuala Lumpur
MISSISSIPPI BABY—BORN LUCKY!
•Baby was born with HIV--There were five
positive tests in the baby’s first month of
life — 4 for viral RNA and 1for DNA.
•She received a potent 3 drug combo
within 30 hours of birth
•Very unusual because it was 3 strong
drugs AND very soon after birth
More about the Mississippi Baby
• The baby stopped going to the doctor,
and stopped getting AIDS drugs, at 18
months.
• 5 MONTHS LATER, she returned to the
doctor. The doctor expected a sick kid.
• Some viral genetic material was found
but—SURPRISE!
• NO REPLICABLE VIRUS
Mississippi Baby, Continued
• The baby doesn’t need AIDS drugs now.
• She is healthy and lives a normal toddler
lifestyle. She is 3 years old.
• Doctors: Hannah Gay, MD in Mississippi
• Deborah Persaud, MD at Johns Hopkins
tracked and reported the case.
A cure may require more than
one type of therapy—perhaps
a drug to activate the latent
virus in viral reservoirs, and
another drug to kill that virus,
and a third therapy to boost the
immune system.
Here are some of the
different approaches that
are being investigated:
HDAC Inhibitors to activate latent virus.
Vorinostat
Activates LATENT HIV in “viral reservoirs”. Already
approved by the FDA for a type of skin cancer. But new
evidence shows that reservoirs include long-lasting CD4
memory cells, complicating this strategy.
Vorinostat. Yep, that's
it.
Studied by David Margolis, MD at UNC Chapel Hill and
Sharon Lewin, MD Monash University, Melbourne, Australia
HDAC Inhibitors: Panobinostat
Similar to Vorinostat but activates more virus
—Being developed by Novartis to treat acute
myeloid leukemia and multiple myeloma.
CYCLICAL DOSING PATTERN
(take it one week but not the next):
20 mgs 3 times per week—every other week
•Increased HIV RNA plasma detected after
each dose; effect didn’t decrease after
multiple doses
•Virus in viral reservoirs was flushed out
of hiding.
PD-1 Inhibitors Also
activate the HIV Virus In Viral
Reservoirs so It can be killed.
Immune Based Strategies:
People with HIV/AIDS Need to
Beef up their Immune System
—Defense as well as Offense.
Studies using the drug Alpha Interferon
indicate that it may be able to strengthen
patients’ immune response so that they
can be cured more easily. (This
research is happening at the Wistar
Institute in West Philly. If they get
funding.)
Boston
(Harvard, specifically)
Tim Henrich, MD, Harvard University
Two Boston AIDS patients
(one was born with HIV)
Like the Berlin patient, these patients had
HIV and cancer and needed a dangerous bone
marrow transplant to fight the cancer.
Unlike the Berlin patient, their doctor had
them stay on AIDS drugs during the bone
marrow transplant—and they were given an
easier conditioning regimen to prep for the
transplant (easier on their bodies).
Unlike the Berlin Patient, their donor wasn’t
born immune to HIV.
Boston Patients, The Results
• After their bone marrow transplant—the Boston
patients kept taking AIDS drugs, but had no
detectable AIDS virus in their bodies or their
blood.
• Recently, researchers asked them to stop taking
their AIDS drugs—AIDS did not return!
• Time will tell if they are permanently cured; they
are considered functionally cured or in
“remission.”
• This Oakland, California company is infusing
HIV patients with their own T-cells, treated to
remove CCr5 receptors— so the t-cells can’t
get infected with HIV. They are only testing it in
tiny numbers of patients, so far.
• This is the “zinc finger technology” you may
have heard about.
• Sustained increase in CD4 cells
• Lower viral load
Sangamo!
This zinc finger technology creates AIDS-proof
cells that, after they are infused into a person
living with AIDS, spread throughout the patient’s
bodies, making them (so far) temporarily
healthier.
One challenge: Making enough stuff to cure
someone: Transforming enough regular CD4
cells or stem cells into these AIDS-proof cells to
cure a whole person (and then several million
people).
Sangamo is testing their treatment on
people born immune to AIDS (one
dominant unimmune trait, one
recessive immune trait)
Post infusion with the altered cells, a 16-week
ART treatment interruption can lead to viral
load reduction.
o Two out of four subjects showed
reduction in viral load during treatment
interruption
o One subject achieved temporary undetectable
viral load during treatment interruption after
treatment.
Someone to Watch: Irvin Chen
Los Angeles researcher Irvin Chen is infusing
patients with STEM CELLS (not T-cells) that use
zinc finger technology to make them AIDS-
proof. Stem cells can develop (differentiate) into
a whole array of different kinds of cells
Clinical trials are starting and the first patient was
infused a few weeks ago.
Uses CMV to cure 36 monkeys.
People are next.
Louis Picker, MD, Oregon Health and Science University
Monkeys cured
About half the monkeys Dr. Picker tries his CMV
vaccine on are cured—the other half are not.
We don’t know why.
Human trials will happen in about 2 years—people
are much more varied than lab monkeys, so we
will have to see what happens.
Could be used as both a vaccine AND A CURE
What do we want?What do we want?
A Scalable CureA Scalable Cure
$ 240 Million in new money For the CURE
More funding streams (private foundations;
philanthropists)
Support for new ideas – More People like Gero
Huetter
Better collaboration among researchers
(although this is improving)
Innovation in the way research is managed
A big, ugly difficult, expensive cure. Leading to a
refined, cheap, scalable cure. And that’s okay.
TIME FOR A CURE—FOR THIS
GENERATION!
Kate@AIDSPolicyProject.org
JoseMarcos9@yahoo.com
215-939-7852
Sign up: www.aidspolicyproject.org

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AIDS Cure Day Webinar on AIDS Research and Activism

  • 1.
  • 2. Having a chronic, manageable disease is not that great, if you’re the one with the disease.
  • 3. Worldwide, less than half of the very sickest of the 34 million people with HIV/AIDS can access treatment. Global Funding Pulling Back? Sequestration Budget Cuts Uncertain Future for the AIDS Drug Assistance Program
  • 4. We need a cure.
  • 5. What we expected when we started ...What we expected when we started ... Abundant Resources! Impossible Research Obstacles!
  • 6. What We FoundWhat We Found Few Resources Great Research !!!
  • 7.
  • 8. Proof of concept: The Berlin Patient was cured in 2007. • His therapy cost $200,000, or about 1/3 of the lifetime cost of a treatment for a person with AIDS in the United States. • His cure is leading to safer cure research alternatives, using a patient’s own immune cells. • Too dangerous: The chance of dying from this treatment is 30%.
  • 10. The National Institutes of Health spent $56.4 million on AIDS cure research and $557 million on AIDS vaccine research in 2012. Source: NIH (for cure statistic) and IAVI (for vaccine statistic)
  • 11.
  • 12. is that a big enough slice for you? We learned that the NIH is spending only 3% of its AIDS research budget to find a cure. We need more than that. We’re going to need a bigger pie.
  • 13. WE DEMANDED MORE. We sent 500 letters to Frank Collins, head of the National Institutes of Health We got big stories and op-eds in the media We held frank meetings with top NIH officials. The NIH found $27 million in new money for cure research. We aren’t finished.
  • 14. Cool Developments in The Search for a Cure for AIDS Cure strategies being investigated (no guarantees): •Flush out latent virus in viral reservoirs and kill it •Replace your immune system with somebody else’s •Make AIDS-proof cells that take over your body •Treat hard and very early, stay on treatment for a few years, then maybe you can go off drugs but stay undetectable (but maybe not)
  • 16. Visconti Cohort: Treated early, now control AIDS 14 patients (out of a larger group of 75) who started potent combination ART within 10 weeks (median 40 days) after HIV infection—during acute infection. They stayed on treatment for at least 1 year (median 3 years). Then they left treatment. VISCONTI patients appear to have controlled HIV, with viral load (less than 50 copies/mL), for a median of 76 months, or more than 6 years, after treatment interruption. Without drugs.
  • 17. Lesson of the VISCONTI Cohort It may be possible to push 15% of newly infected people into remission (HIV-infected but don’t need AIDS drugs to control the disease—the body’s immune system controls it) just by treating people early enough after they are infected. This might be because their bodies haven’t had time to develop a viral reservoir. (Researchers are still figuring this out.)
  • 18. German man (not the Berlin Patient, another guy) CURED? • A German man, infected in 1999, started HIV treatment within three months of diagnosis, when his viral load was over one million. He quit AIDS meds in 2004, and after a very brief viral rebound has remained undetectable ever since. • Reported at IAS2013 in Kuala Lumpur
  • 19.
  • 20. MISSISSIPPI BABY—BORN LUCKY! •Baby was born with HIV--There were five positive tests in the baby’s first month of life — 4 for viral RNA and 1for DNA. •She received a potent 3 drug combo within 30 hours of birth •Very unusual because it was 3 strong drugs AND very soon after birth
  • 21. More about the Mississippi Baby • The baby stopped going to the doctor, and stopped getting AIDS drugs, at 18 months. • 5 MONTHS LATER, she returned to the doctor. The doctor expected a sick kid. • Some viral genetic material was found but—SURPRISE! • NO REPLICABLE VIRUS
  • 22. Mississippi Baby, Continued • The baby doesn’t need AIDS drugs now. • She is healthy and lives a normal toddler lifestyle. She is 3 years old. • Doctors: Hannah Gay, MD in Mississippi • Deborah Persaud, MD at Johns Hopkins tracked and reported the case.
  • 23. A cure may require more than one type of therapy—perhaps a drug to activate the latent virus in viral reservoirs, and another drug to kill that virus, and a third therapy to boost the immune system.
  • 24. Here are some of the different approaches that are being investigated:
  • 25. HDAC Inhibitors to activate latent virus. Vorinostat Activates LATENT HIV in “viral reservoirs”. Already approved by the FDA for a type of skin cancer. But new evidence shows that reservoirs include long-lasting CD4 memory cells, complicating this strategy. Vorinostat. Yep, that's it. Studied by David Margolis, MD at UNC Chapel Hill and Sharon Lewin, MD Monash University, Melbourne, Australia
  • 26. HDAC Inhibitors: Panobinostat Similar to Vorinostat but activates more virus —Being developed by Novartis to treat acute myeloid leukemia and multiple myeloma. CYCLICAL DOSING PATTERN (take it one week but not the next): 20 mgs 3 times per week—every other week •Increased HIV RNA plasma detected after each dose; effect didn’t decrease after multiple doses •Virus in viral reservoirs was flushed out of hiding.
  • 27. PD-1 Inhibitors Also activate the HIV Virus In Viral Reservoirs so It can be killed.
  • 28. Immune Based Strategies: People with HIV/AIDS Need to Beef up their Immune System —Defense as well as Offense. Studies using the drug Alpha Interferon indicate that it may be able to strengthen patients’ immune response so that they can be cured more easily. (This research is happening at the Wistar Institute in West Philly. If they get funding.)
  • 30. Tim Henrich, MD, Harvard University
  • 31. Two Boston AIDS patients (one was born with HIV) Like the Berlin patient, these patients had HIV and cancer and needed a dangerous bone marrow transplant to fight the cancer. Unlike the Berlin patient, their doctor had them stay on AIDS drugs during the bone marrow transplant—and they were given an easier conditioning regimen to prep for the transplant (easier on their bodies). Unlike the Berlin Patient, their donor wasn’t born immune to HIV.
  • 32. Boston Patients, The Results • After their bone marrow transplant—the Boston patients kept taking AIDS drugs, but had no detectable AIDS virus in their bodies or their blood. • Recently, researchers asked them to stop taking their AIDS drugs—AIDS did not return! • Time will tell if they are permanently cured; they are considered functionally cured or in “remission.”
  • 33. • This Oakland, California company is infusing HIV patients with their own T-cells, treated to remove CCr5 receptors— so the t-cells can’t get infected with HIV. They are only testing it in tiny numbers of patients, so far. • This is the “zinc finger technology” you may have heard about. • Sustained increase in CD4 cells • Lower viral load
  • 34. Sangamo! This zinc finger technology creates AIDS-proof cells that, after they are infused into a person living with AIDS, spread throughout the patient’s bodies, making them (so far) temporarily healthier. One challenge: Making enough stuff to cure someone: Transforming enough regular CD4 cells or stem cells into these AIDS-proof cells to cure a whole person (and then several million people).
  • 35. Sangamo is testing their treatment on people born immune to AIDS (one dominant unimmune trait, one recessive immune trait) Post infusion with the altered cells, a 16-week ART treatment interruption can lead to viral load reduction. o Two out of four subjects showed reduction in viral load during treatment interruption o One subject achieved temporary undetectable viral load during treatment interruption after treatment.
  • 36. Someone to Watch: Irvin Chen Los Angeles researcher Irvin Chen is infusing patients with STEM CELLS (not T-cells) that use zinc finger technology to make them AIDS- proof. Stem cells can develop (differentiate) into a whole array of different kinds of cells Clinical trials are starting and the first patient was infused a few weeks ago.
  • 37. Uses CMV to cure 36 monkeys. People are next. Louis Picker, MD, Oregon Health and Science University
  • 38. Monkeys cured About half the monkeys Dr. Picker tries his CMV vaccine on are cured—the other half are not. We don’t know why. Human trials will happen in about 2 years—people are much more varied than lab monkeys, so we will have to see what happens. Could be used as both a vaccine AND A CURE
  • 39. What do we want?What do we want? A Scalable CureA Scalable Cure $ 240 Million in new money For the CURE More funding streams (private foundations; philanthropists) Support for new ideas – More People like Gero Huetter Better collaboration among researchers (although this is improving) Innovation in the way research is managed A big, ugly difficult, expensive cure. Leading to a refined, cheap, scalable cure. And that’s okay.
  • 40.
  • 41.
  • 42. TIME FOR A CURE—FOR THIS GENERATION! Kate@AIDSPolicyProject.org JoseMarcos9@yahoo.com 215-939-7852 Sign up: www.aidspolicyproject.org

Hinweis der Redaktion

  1. Introduce yourself and talk about the group
  2. This is Winstone Zulu. He is the first publicy HIV+ person in the country of Zambia, a father of four, commended by Nelson Mandela for his public health work. He was a huge supporter of the AIDS Policy Project and coined the term, Time for a cure! He ran through his treatment options because of stockouts in Zambia—he became resistant to the drugs he had available to him. He died in 2011.
  3. We figured that there were billions of dollars being pumped into the search for the cure for AIDS. We thought that the scientific obstacles were too great to overcome—it’s a retrovirus—it mutates so quickly. We were wrong.
  4. There was very little funding. And really great research. Activists had almost given up on a cure. PWAs thought it was a lost cause. BUT—the research itself was quietly going incredibly well. One person had already been cured, and there were lots of other promising approaches being investigated.
  5. This is the Berlin patient Timothy Ray Brown, the first person ever cured of AIDS. During a tour we gave him last year of the AIDS research labs at the University of Pennsylvania.
  6. Dangerous because of toxic preparation (intense chemotherapy and radiation) to prepare for stem cell transplant. Also dangerous because of risk of rejection.
  7. US: $56.2 MILLION/CURE $557 MILLION/VACCINES Here’s one of the biggest obstacles to a cure. The bar on the left—really it’s a slate patio tile of funding—is the amount the NIH is spending right now on direct AIDS cure research. The middle bar, more like a pancake, is AIDS vaccine research. The NIH spends 9 times more on AIDS vaccines research than on the cure. On the right is the total amount, $24 BILLION , that the US spends on domestic and foreign AIDS programs. That money is keeping millions of people alive. But we need more money for a cure.
  8. US: $56.4 MILLION/CURE $557 MILLION/VACCINES Here’s one of the biggest obstacles to a cure. The bar on the left—really it’s a slate patio tile of funding—is the amount the NIH is spending right now on direct AIDS cure research. The middle bar, more like a pancake, is AIDS vaccine research. The NIH spends 9 times more on AIDS vaccines research than on the cure. On the right is the total amount, $24 BILLION , that the US spends on domestic and foreign AIDS programs. That money is keeping millions of people alive. But we need more money for a cure.
  9. US: $56.4 MILLION/CURE $557 MILLION/VACCINES Here’s one of the biggest obstacles to a cure. The bar on the left—really it’s a slate patio tile of funding—is the amount the NIH is spending right now on direct AIDS cure research. The middle bar, more like a pancake, is AIDS vaccine research. The NIH spends 9 times more on AIDS vaccines research than on the cure. On the right is the total amount, $25 BILLION (per Kaiser Family Foundation) that the US spends on domestic and foreign AIDS programs. That money is keeping millions of people alive. But we need more money for a cure.
  10. The NIH spends about 3% of their overall AIDS research budget on direct cure research. The AIDS Policy Project was the only group to ever ask how much the NIH was spending to CURE AIDS.
  11. Another problem is red tape at the NIH and FDA—there is less than there used to be, and a lot of good will in trying to solve it, but it still slows down the research. This is ¼ of Dave Margolis, a major AIDS researcher at UNC Chapel Hill. This is is his Vorinostat study. And a letter delaying it from the FDA . It took three years of pushing to untangle it from red tape—three critical years for people who have run out of treatment options. We made his study a centerpiece of our grassroots campaign for more AIDS funding at the NIH. Now it’s one of three new projects in the Marin Delaney Collaboratory, a new research program named after Marty Delaney of Project Inform.
  12. EACH ONE TEACH ONE
  13. It either worked or it didn’t—and the french researchers are trying to figure out why this worked for some people and not for others. Theory is that if you can treat people very early, they can’t develop a viral reservoir.
  14. The MISSISSIPPI Baby.
  15. Her doctor decided to take a chance and treat her more aggressively than normal. This treatment may become the standard of care for babies born to HIV-positive mothers who did not receive AIDS drugs to prevent mother-to-child-transmission of AIDS.
  16. THIS MAY EVENTUALLY BE A NEW STANDARD OF CARE – new research is being done.
  17. The goal of AIDS cure research is to eradicate AIDS from the whole body, including viral reservoirs. Viral reservoirs are the last, stubborn pockets of HIV left in the body after HIV has been wiped out in the bloodstream. There are viral reservoirs in the gut, in the brain, and in other parts of the body. The virus in those pockets is not activated and almost impossible to detect. The challenge is to activate this virus so it can be detected and killed. Vorinostat activates the virus so it can be detected, but it doesn’t kill it. That may take a second drug. Research is continuing on this drug.
  18. Dr. Montaner at the Wistar Institute has been experimenting with Alpha-Interferon. He documented the first immune therapy that can control HIV in absence of ART and can reduce viral reservoirs.  He completed a proof-of-concept study demonstrating Alpha-Interferon’s ability to activate anti-HIV responses when started during antiretroviral therapy. After people were taken off their AIDS drugs (but kept on alpha-Interferon, , viral suppression occurred in 45% of people tested-----and those people’s HIV reservoir levels were reduced.  It remains to be determined whether alpha-interferon could be an immune-boost approach to "treat" HIV reservoirs and accelerate a cure. Dr. Montaner has been waiting for THREE YEARS for funding to follow up research to this important study. WE NEED MORE FUNDING FOR THIS RESEARCH.
  19. HIV in your blood is what “viral load” is.
  20. Last day of a conference so long we called it FOREVERCON. We have a long interview with him from last year on our blog.
  21. See AIDS Policy Project’s blog for an interview with Tim Hetherwick, the Harvard researcher who is tracking these patients. Last day of a conference so long we called it FOREVERCON.
  22. HIV in your blood is what “viral load” is. No viral rebound. One patient stopped taking antiretroviral drugs seven weeks ago. For the other, it has been 15 weeks. No virus or antibodies to the virus have been found in their blood or other tissues since.
  23. First doctors sort out some healthy T-cells and send them to the company. It removes the CCr5 receptors, then sends the cells back to the patient’s researcher, who infuses them into the patient. This is a SAFER WAY TO REPLICATE THE BERLIN PATIENT TREATMENT. You are using your own cells instead of an outside donor, so there is no risk of rejection and no radiation or chemotherapy to kill off your immune system, as there is before a stem cell transplant.
  24. This is Louis Picker, MD, an AIDS vaccine and cure researcher based at Oregon Health and Science University. He has used CMV virus as a vector for SIV (the monkey form of AIDS) and CURED 36 macaques (a type of monkey). Next up: He will make a vector with CMV and HIV and try to cure people.
  25. And an X Prize! READ OUR REPORT: CURING AIDS FASTER
  26. Our pal Timothy.