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NIH to fund unproven ALS drugs under patient-backed law

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WASHINGTON (AP) — When patients with a deadly diagnosis and few treatment options have tried to get unapproved, experimental drugs, they have long faced a dilemma: Who will pay?

Responsibility for funding so-called compassionate use has always fallen to drugmakers, though many are unwilling or unable to make their drugs available for free to dying patients.

After years of lobbying Congress, patients with the debilitating illness known as Lou Gehrig’s disease have found an unprecedented solution: make the federal government pay.

Under a recent law, the National Institutes of Health will begin spending about $25 million to enroll patients in compassionate use — also called open access — programs of unapproved drugs. The first step, announced Friday, will give patients access to a sugar-based injection called trehalose, that is thought to help nerve cells clear toxic proteins.

Only patients who can’t get into conventional drug trials are eligible for the program. And their progress must be tracked to gather data about the treatment and their underlying disease, amyotrophic lateral sclerosis, or ALS.

The initiative blurs the line between treatment and research, and it puts the NIH in the position of paying for unapproved drugs in studies that may yield limited data. While it offers a critical new option for ALS patients, it also raises the possibility that limited federal dollars could eventually be tapped for more unproven treatments in other diseases.

“We don’t typically expect the government to pay for things until we know they work,” said Holly Fernandez Lynch, a University of Pennsylvania bioethicist. “But the system we have in this country relies on drug companies to develop our drugs, and private companies are not in the business of providing their products for free.”

Fernandez Lynch and many other experts support the new approach as an innovative solution to the challenges facing ALS patients, who typically survive three to five years after initial symptoms. The disease destroys nerve cells needed to walk, talk and — eventually — breathe.

Up to 90% of ALS patients are ineligible for traditional clinical trials, according to researchers, typically because their disease has progressed too far to show major treatment benefits. Even eligible patients must compete for access. One recent analysis counted 2,000 trial openings in the U.S. for 25,000 people living with ALS.

Patients aren’t the only beneficiaries of the NIH program. The government funding essentially replaces costs previously borne by drugmakers.

One long-time patient advocate sees a troubling precedent in that financial shift.

“My sense is that it’s the companies’ responsibility, not the taxpayers’, to pick up the cost for expanded access programs,” said Gregg Gonsalves, a Yale University researcher who has informally advised ALS patients on expanded access. “But the companies have stonewalled patients for years, so as a last resort they went to Congress.”

During the 1980s and 1990s, Gonsalves and other HIV activists were instrumental in pushing drugmakers to provide early access to experimental medications.

ALS patients say most companies in their field are tiny startups that can’t afford such costs. Drugmakers have other reasons to deny access, including concerns that unexpected safety problems could hurt their approval chances.

The NIH spends the vast majority of its $45 billion budget on early-stage research focused on identifying the root causes, treatments and potential cures for diseases.

Tracking drug safety is one key aspect of the new program, along with various biological measures of ALS. But the initiative is unlikely to detect whether the drugs are actually working, because patients won’t be compared to a placebo group, the gold-standard approach to medical research.

“Unless the drug is a miracle drug, it’s unlikely you would see efficacy in this type of research,” said Dr. Walter Koroshetz, of the National Institute of Neurological Disorders and Stroke.

The initiative is part of broader legislation pushed through Congress last year by patient advocates, including I AM ALS, a nonprofit co-founded by two former Obama White House staffers.

“I’m five years in so I can’t qualify for any clinical trials,” said Brian Wallach, who launched I AM ALS with his wife after being diagnosed in 2017. “I hope to be eligible for the expanded access pathway.”

He describes NIH’s new program as a “pilot” that will be reviewed by federal inspectors, as required by the new law.

Wallach spent several years working on the legislation with congressional staffers. It passed the House last year by a 423-3 vote, a rare display of bipartisanship that also underscored the group’s political clout.

The far-reaching bill requires the Food and Drug Administration to develop a plan to accelerate drug development and form new partnerships to study neurodegenerative diseases.

The legislation grew out of patients’ deep frustration with access to experimental therapies, including a stem cell treatment from the tiny drugmaker Brainstorm Cell Therapeutics.

After the company’s 200-patient study failed to show positive results in 2020, Brainstorm allowed a handful of patients to continue receiving injections under expanded access. But company executives said a larger program was infeasible, given that Brainstorm has no revenue.

“We used millions of dollars for our small expanded access program,” said Mary Kay Turner, a company executive. “So we did the maximum we could, but it was just a tiny sliver.”

Brainstorm plans to submit its drug for FDA approval, despite a rare public statement from the agency last year that company data “do not support the proposed clinical benefit.” That followed thousands of calls and messages to the agency from ALS patients.

While many treatments may prove ineffective, Dr. Richard Bedlack of Duke University says getting patients into expanded access programs is still preferable to the current situation, in which they often seek out untested remedies on their own.

“Historically their only option was to go on the internet and try to buy these supplements or alternative therapies,” said Bedlack, who consults for several drugmakers.

It remains to be seen how many patients the NIH will enroll under its $25 million grant. The original law called for $100 million in funding over four years. House lawmakers have budgeted $80 million in spending bills for the next fiscal year, though those have not yet passed the Senate.

For now, NIH’s Koroshetz notes that the expanded access studies will be more expensive than other NIH trials because the government is bearing the cost of making and distributing the drugs.

He conceded: “It’s a little different from our usual grants, where we don’t pay the companies at all for the drug.”

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China is raising its retirement age, now among the youngest in the world’s major economies

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Starting next year, China will raise its retirement age for workers, which is now among the youngest in the world’s major economies, in an effort to address its shrinking population and aging work force.

The Standing Committee of the National People’s Congress, the country’s legislature, passed the new policy Friday after a sudden announcement earlier in the week that it was reviewing the measure, state broadcaster CCTV announced.

The policy change will be carried out over 15 years, with the retirement age for men raised to 63 years, and for women to 55 or 58 years depending on their jobs. The current retirement age is 60 for men and 50 for women in blue-collar jobs and 55 for women doing white-collar work.

“We have more people coming into the retirement age, and so the pension fund is (facing) high pressure. That’s why I think it’s now time to act seriously,” said Xiujian Peng, a senior research fellow at Victoria University in Australia who studies China’s population and its ties to the economy.

The previous retirement ages were set in the 1950’s, when life expectancy was only around 40 years, Peng said.

The policy will be implemented starting in January, according to the announcement from China’s legislature. The change will take effect progressively based on people’s birthdates.

For example, a man born in January 1971 could retire at the age of 61 years and 7 months in August 2032, according to a chart released along with the policy. A man born in May 1971 could retire at the age of 61 years and 8 months in January 2033.

Demographic pressures made the move long overdue, experts say. By the end of 2023, China counted nearly 300 million people over the age of 60. By 2035, that figure is projected to be 400 million, larger than the population of the U.S. The Chinese Academy of Social Sciences had previously projected that the public pension fund will run out of money by that year.

Pressure on social benefits such as pensions and social security is hardly a China-specific problem. The U.S. also faces the issue as analysis shows that currently, the Social Security fund won’t be able to pay out full benefits to people by 2033.

“This is happening everywhere,” said Yanzhong Huang, senior fellow for global health at the Council on Foreign Relations. “But in China with its large elderly population, the challenge is much larger.”

That is on top of fewer births, as younger people opt out of having children, citing high costs. In 2022, China’s National Bureau of Statistics reported that for the first time the country had 850,000 fewer people at the end of the year than the previous year , a turning point from population growth to decline. In 2023, the population shrank further, by 2 million people.

What that means is that the burden of funding elderly people’s pensions will be divided among a smaller group of younger workers, as pension payments are largely funded by deductions from people who are currently working.

Researchers measure that pressure by looking at a number called the dependency ratio, which counts the number of people over the age of 65 compared to the number of workers under 65. That number was 21.8% in 2022, according to government statistics, meaning that roughly five workers would support one retiree. The percentage is expected to rise, meaning fewer workers will be shouldering the burden of one retiree.

The necessary course correction will cause short-term pain, experts say, coming at a time of already high youth unemployment and a soft economy.

A 52-year-old Beijing resident, who gave his family name as Lu and will now retire at age 61 instead of 60, was positive about the change. “I view this as a good thing, because our society’s getting older, and in developed countries, the retirement age is higher,” he said.

Li Bin, 35, who works in the event planning industry, said she was a bit sad.

“It’s three years less of play time. I had originally planned to travel around after retirement,” she said. But she said it was better than expected because the retirement age was only raised three years for women in white-collar jobs.

Some of the comments on social media when the policy review was announced earlier in the week reflected anxiety.

But of the 13,000 comments on the Xinhua news post announcing the news, only a few dozen were visible, suggesting that many others had been censored.

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Russia warns NATO of ‘direct war’ over Ukraine

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Moscow’s envoy to the UN has reiterated where the Kremlin’s red line is

Granting Kiev permission to use Western-supplied long-range weapons would constitute direct involvement in the Ukraine conflict by NATO, Russia’s envoy to the UN, Vassily Nebenzia, has said.

Moscow will treat any such attack as coming from the US and its allies directly, Russian President Vladimir Putin said on Thursday, explaining that long-range weapons rely on Western intelligence and targeting solutions, neither of which Ukraine is capable of.

NATO countries would “start an open war” with Russia if they allow Ukraine to use long-range weapons, Nebenzia told the UN Security Council on Friday.

“If such a decision is made, that means NATO countries are starting an open war against Russia,” Moscow’s envoy said. “In that case, we will obviously be forced to make certain decisions, with all the attendant consequences for Western aggressors.”

Putin issues new warning to NATO

“Our Western colleagues will not be able to dodge responsibility and blame Kiev for everything,” Nebenzia added. “Only NATO troops can program the flight solutions for those missile systems. Ukraine doesn’t have that capability. This is not about allowing Kiev to strike Russia with long-range weapons, but about the West making the targeting decisions.”

Russia considers it irrelevant that Ukrainian nationalists would technically be the ones pulling the trigger, Nebenzia explained. “NATO would become directly involved in military action against a nuclear power. I don’t think I have to explain what consequences that would have,” he said.

The US and its allies placed some restrictions on the use of their weapons, so they could claim not to be directly involved in the conflict with Russia, while arming Ukraine to the tune of $200 billion.

Multiple Western outlets have reported that the limitations might be lifted this week, as US Secretary of State Antony Blinken and British Foreign Secretary David Lammy visited Kiev. Russia has repeatedly warned the West against such a course of action.

 

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China makes its move in Africa. Should the West be worried?

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Beijing maintains a conservative economic agenda in its relations with the continent, while finding it increasingly difficult to avoid a political confrontation with the West

The ninth forum on China-Africa Cooperation (FOCAC) and the FOCAC summit held in Beijing on September 4-6 marked a significant phase in Africa’s relations with its global partners in the post-Covid era. China is the last major partner to hold a summit with African nations following the end of the pandemic; Africa summits were held by the EU and the US in 2022, and by Russia in 2023. The pandemic, coupled with rising global tensions, macroeconomic shifts, and a series of crises, underlined Africa’s growing role in the global economy and politics – something that China, which has undergone major changes (both internal and external) as a result of the pandemic, is well aware of.

It is clear that the relationship between China and Africa is entering a new phase. China is no longer just a preferential economic partner for Africa, as it had been in the first two decades of the 21st century. It has become a key political and military ally for many African countries. This is evident from China’s increasing role in training African civil servants and sharing expertise with them, as well as from several initiatives announced at the summit, including military-technical cooperation: officer training programs, mine clearing efforts, and over $100 million which China will provide to support the armed forces of African nations.

In the political arena, however, Beijing is proceeding very cautiously and the above-mentioned initiatives should be seen as the first tentative attempts rather than a systematic strategy.

While China strives to avoid political confrontation with the West in Africa and even closely cooperates with it on certain issues, it is becoming increasingly difficult to do so. Washington is determined to pursue a policy of confrontation with Beijing in Africa – this is evident both from US rhetoric and its strategic documents.

Dirty tactics: How the US tries to break China’s soft power in Africa

A “divorce” between China and the West is almost inevitable. This means that Chinese companies may lose contracts with Western corporations and won’t have access to transportation and logistics infrastructure. Consequently, China will need to develop its own comprehensive approach to Africa, either independently or in collaboration with other global power centers.

An important sign of the growing confrontation between the US and China in Africa was the signing of a trilateral memorandum of understanding between China, Tanzania, and Zambia regarding the reconstruction of the Tanzania-Zambia Railway (TAZARA), which was originally built by China in the 1970s. If it is expanded, electrified, and modernized, TAZARA has the potential to become a viable alternative to one of the key US investment projects in the region: the Lobito Corridor, which aims to enhance logistics infrastructure for exporting minerals (copper and cobalt) from the Democratic Republic of the Congo and Zambia by modernizing the railway from the DR Congo to the Angolan port of Lobito.

In inland regions such as Eastern Congo, transportation infrastructure plays a crucial role in the process of mineral extraction. Considering the region’s shortage of rail and road networks, even a single non-electrified railway line leading to a port in the Atlantic or Indian Ocean can significantly boost the operation of the mining sector and permanently tie the extraction and processing regions to specific markets.

It appears that China’s initiative holds greater promise compared to the US one, particularly because Chinese companies control major mines both in the Democratic Republic of the Congo and Zambia. This gives them a clear advantage in working with Chinese operators and equipment, facilitating the export of minerals through East African ports. Overall, this indicates that East Africa will maintain its role as the economic leader on the continent and one of the most integrated and rapidly developing regions for imports.

A former colonial European power returns to Africa. What is it after now?

The highlight of the summit was China’s pledge to provide $50 billion to African countries over the next three years (by 2027). This figure echoes the $55 billion commitment to China made by the US (for 3 years) at the 2022 US-Africa Summit and the $170 billion that the EU promised to provide over seven years back in 2021. Consequently, leading global players allocate approximately $15-20 billion annually to Africa.

In recent years, there has been noticeable growth in such promises. Nearly every nation is eager to promise Africa something – for example, Italy has pledged $1 billion annually. However, these large packages of so-called “financial aid” often have little in common with actual assistance, since they are typically commercial loans or corporate investments. Moreover, a significant portion of these funds is spent in the donor countries (e.g. on the procurement and production of goods), which means that they contribute to the economic growth of African nations in a minimal way.

As for China, it will provide about $11 billion in genuine aid. This is a substantial amount which will be used for developing healthcare and agriculture in Africa. Another $30 billion will come in the form of loans (roughly $10 billion per year) and a further $10 billion as investments.

The overall financial framework allows us to make certain conclusions, though it’s important to note that the methodology for calculating these figures is unclear, and the line between loans, humanitarian aid, and investments remains blurred. In terms of investments (averaging around $3 billion per year), Beijing plans to maintain its previous levels of activity – in recent years, China’s foreign direct investments (FDI) have ranged from $2 billion to $5 billion annually. Financial and humanitarian aid could nearly double (from the current $1.5 billion-$2 billion per year) while lending is expected to return to pre-pandemic levels (which would still be below the peak years of 2012-2018).

Can Africa seize control of its own energy?

China’s economic plan for Africa seems to be quite conservative. It’s no surprise that debt issues took center stage during the summit. During the Covid-19 pandemic, macroeconomic stability in African countries deteriorated, which led to challenges in debt repayments and forced Africa to initiate debt restructuring processes assisted by the IMF and the G20. Starting in 2020, a combination of internal and external factors led China to significantly cut its lending to African countries – from about $10-15 billion down to $2-3 billion. This reduction in funding has triggered economic reforms in several African countries (e.g. Ghana, Kenya, and Nigeria), which have shifted toward stricter tax and monetary policies. While promises to increase lending may seem like good news for African nations, it’s likely that much of this funding will go toward interest payments on existing obligations and debt restructuring, since China wants to ensure that its loans are repaid.

Despite China’s cautious approach to Africa, its interaction with the continent will develop as a result of external and internal changes affecting both Africa and China. Africa will gradually become more industrialized and will reduce imports while the demand for investments and local production will increase. China will face demographic challenges, and its workforce will decrease. This may encourage bilateral cooperation as some production facilities may move from China to Africa. This will most likely concern East African countries such as Ethiopia and Tanzania, considering China’s current investments in their energy and transportation infrastructure. Additionally, with Africa’s population on the rise and China’s population declining, Beijing is expected to attract more African migrant workers to help address labor shortages.

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