US and Gates Foundation plan $200m for sickle cell, HIV cures

WASHINGTON — The US government and the Bill and Melinda Gates Foundation pledged Wednesday to jointly invest $200 million over the next 4 years to achieve affordable gene therapy-based cures for sickle cell disease (SCD) and HIV.

The administration of President Donald Trump announced earlier this year its intention to end the HIV epidemic over the next decade and has also identified SCD, which disproportionately affects people of African descent, as a condition requiring greater attention.

Gene therapy is a relatively new area of medicine designed to replace faulty genes in the body that are responsible for a disorder, and has been responsible for new treatments for blindness and certain types of leukemia.

But the treatments are complex and costly, ruling them out as an option for most of the world.

Francis Collins, director of the National Institutes of Health, said the collaboration would focus therefore on "access, scalability and affordability" to make sure the eventual treatments are available globally.

The NIH and Gates Foundation aim to achieve clinical trials in the United States and countries in sub-Saharan Africa within the next seven to 10 years.

Sickle cell disease is a group of inherited red blood cell disorders characterized by the presence of an abnormal protein in the red blood cells, causing the feet and hands to swell, fatigue, jaundice, and episodic or chronic pain.

Over time the disease can harm a patients' vital organs, bones, joints and skin and it is currently only curable via a blood and bone marrow transplant, available to only a tiny fraction of people who have the disease.

When it comes to HIV, antiretroviral therapy (ART) are now able to reduce patients' viral load to the point that they are undetectable and cannot be further transmitted. 

But "a major goal is to find a cure, whereby lifelong ART would not be required," said the NIH's Anthony Fauci.

Though SCD is a genetically inherited disease, and HIV is acquired from infection, gene-based treatments are said to hold promise for both, and "many of the technical challenges for gene-based cures are expected to be common to both diseases." 

The goal for SCD is to achieve a gene-based intervention that either corrects the gene mutation responsible or promotes fetal hemoglobin gene expression to achieve normal hemoglobin function.

For HIV, the proposed cure would involve targeting the reservoir of proviral DNA that lurks inside a small number of cells even after many years of ART.

The NIH said that approximately 95 percent of the 38 million people living with HIV globally are in the developing world, with 67 percent in sub-Saharan Africa, half of whom are living untreated. Around 1.1 million Americans are affected

SCD affects approximately 100,000 Americans, according to official figures. Fifteen million babies will be born with SCD globally over the next 30 years, with about 75 percent of those births occurring in sub-Saharan Africans, said the NIH.

source: news.abs-cbn.com

Ebola vaccine promising in first human trials: NIH

WASHINGTON - Researchers say they are one step closer to developing an Ebola vaccine, with a Phase 1 trial showing promising results, but it will be months at the earliest before it can be used in the field.

The news comes amid the worst ever outbreak of the hemorrhagic fever, which has killed 5,500 people so far, mostly in West Africa.

Pharmaceutical companies and health agencies scramble to fast-track experimental drugs and vaccines that could help.

In the first phase of testing, all 20 healthy adults injected with a higher or lower dose of the vaccine developed antibodies needed to fight Ebola, said the National Institutes of Health (NIH), which conducted the study.

Results were published Wednesday in the New England Journal of Medicine.

"The unprecedented scale of the current Ebola outbreak in West Africa has intensified efforts to develop safe and effective vaccines," said Anthony Fauci, head of the National Institute of Allergy and Infectious Diseases, which is developing the vaccine alongside GlaxoSmithKline.

The vaccines under development "may play a role in bringing this epidemic to an end and undoubtedly will be critically important in preventing future large outbreaks," he noted.

"Based on these positive results from the first human trial of this candidate vaccine, we are continuing our accelerated plan for larger trials to determine if the vaccine is efficacious in preventing Ebola infection," he added.

But the NIAID/GSK vaccine is still a long way from being ready for use in the field.

The NIAID is "in active discussions with Liberian officials and other partners about next-stage vaccine testing in West Africa" for efficacy and safety, the NIH said, but no announcement on larger-scale trials was expected before early next year.

There is no licensed treatment or vaccine against the Ebola virus, which is transmitted through bodily fluids and has been fatal in an estimated 70 percent of cases in the current outbreak.

Antibodies within four weeks

The volunteers were injected starting in September, and each showed a positive result for Ebola antibodies in blood tests within four weeks.

The 10 volunteers in the higher-dose group developed higher antibody levels, the NIH said.

In addition, two of the lower-dose group and seven of the higher-dose group developed a kind of immune cell called CD8 T cells, which are an important part of the body's response against disease.

"We know from previous studies in non-human primates that CD8 T cells played a crucial role in protecting animals" who got the vaccine and then were exposed to Ebola, said researcher Julie Ledgerwood, the trial’s principal investigator.

None of the volunteers experienced serious side effects within the study period, though two had a brief, mild fever within the 24 hours after the injection.

The vaccine uses a modified chimpanzee cold virus to deliver segments of genetic material from the Ebola virus.

The genetic material cannot spread in the body like the virus does, but can still prompt the antibody response.

The version tested at NIH contains material from two species of Ebola -- the Zaire species, responsible for the outbreak in West Africa, and another called Sudan Ebola.

"This work is encouraging and another significant contribution to efforts to tackle the Ebola crisis," said Dr Jeremy Farrar, Director of the Wellcome Trust.

The White House also congratulated the vaccine researchers.

"We congratulate Drs Francis Collins and Tony Fauci and their teams at the National Institutes of Health on the first published results from Phase 1 clinical trials of a promising Ebola vaccine candidate," a White House statement said, adding that President Barack Obama would visit the NIH next week.

A second version of the vaccine, aimed at blocking just Zaire Ebola, also began human testing in October, at the University of Maryland.

Another experimental vaccine that has shown promising results in primates is the Canadian VSV-EBOV, licensed by US firm NewLink Genetics. It is also in early stages of human testing.

source: www.abs-cbnnews.com