Drug Innovation 9: Treating Lympho Leukemia and HIV/AIDS

I like this story from the NYT although this was published half year ago. A dedicated scientist at Washington University studying human gnome and lympoblastic leukemia has developed the same cancer that he is personally studying. and subsequently weakened him, awaiting death. His colleagues in the research team set aside many things they were doing and focused only on one thing -- find out what is that disease, how it developed, and how it can be killed. Below is portion of that news report.

http://www.nytimes.com/2012/07/08/health/in-gene-sequencing-treatment-for-leukemia-glimpses-of-the-future.html?_r=0

...Dr. Ley’s team tried a type of analysis that they had never done before. They fully sequenced the genes of both his cancer cells and healthy cells for comparison, and at the same time analyzed his RNA, a close chemical cousin to DNA, for clues to what his genes were doing.

The researchers on the project put other work aside for weeks, running one of the university’s 26 sequencing machines and supercomputer around the clock. And they found a culprit — a normal gene that was in overdrive, churning out huge amounts of a protein that appeared to be spurring the cancer’s growth.

Even better, there was a promising new drug that might shut down the malfunctioning gene — a drug that had been tested and approved only for advanced kidney cancer. Dr. Wartman became the first person ever to take it for leukemia.

And now, against all odds, his cancer is in remission and has been since last fall.

While no one can say that Dr. Wartman is cured, after facing certain death last fall, he is alive and doing well. Dr. Wartman is a pioneer in a new approach to stopping cancer. What is important, medical researchers say, is the genes that drive a cancer, not the tissue or organ — liver or brain, bone marrow, blood or colon — where the cancer originates.

One woman’s breast cancer may have different genetic drivers from another woman’s and, in fact, may have more in common with prostate cancer in a man or another patient’s lung cancer. 
Under this new approach, researchers expect that treatment will be tailored to an individual tumor’s mutations, with drugs, eventually, that hit several key aberrant genes at once. The cocktails of medicines would be analogous to H.I.V. treatment, which uses several different drugs at once to strike the virus in a number of critical areas....
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A combination of genetics science and pharmaceutical science is key to treating certain diseases, especially new or emerging ones. Which brings us to the importance of more medicine innovation and how it can be encouraged, or at least how now to impede it via more politics and more government intervention, like dishonoring intellectual property rights (IPR) like drug patents.

Below is another story on medicine innovation to treat HIV/AIDS. The good news is that more and more medicines and vaccines from more drug manufacturers are coming on stream. Since they are different from each other, both off patent and still patented, patients and health professionals can expect competition among them, both in efficacy and prices. Generic producers can help tighten the competition once newly invented drugs and vaccines have become off patent. The important thing is that someone or some firms must invent those new drugs first, assume all the risks and high cost of R&D. Once they are proven to be useful and safe, generic producers can come in later to produce new brands of the same useful molecules against HIV/AIDS.

More science, more innovation, and less politics. Public health and life expectancy are expected to be improving through time.
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http://www.bworldonline.com/content.php?section=14&title=R&D-to-combat-HIV/AIDS&id=62556

R&D to combat HIV/AIDS

Posted on December 06, 2012 06:44:33 PM

Medicine Cabinet -- Reiner Gloor

“GETTING TO ZERO” new HIV infections and zero deaths from AIDS-related illnesses may sound difficult to achieve but significant progress in the prevention and treatment of HIV/AIDS is providing hope for patients and their families.

In time for World AIDS Day on Dec. 1, biopharmaceutical companies announced that around 73 medicines and vaccines are in the pipeline for the treatment and prevention of HIV.

The Pharmaceutical Research Manufacturers and Associations (PhRMA) reported that these are centered on improving treatment regimens, more effective therapies and promising new preventative vaccines.

Between 2011-2015, the World AIDS Days will have the theme of “Getting to zero: zero new HIV infections. Zero discrimination. Zero AIDS related deaths.” This highlights the need for greater access to treatment for all.

Nearly 40 medicines have been approved to treat HIV/AIDS in the past 30 years. There have also been advances in the diagnosis of the disease, allowing for earlier treatment and care.

The World Health Organization (WHO) attributed many of the gains to life-saving antiretrovirals that reduce the amount of virus in the blood so that patients has increased chances of staying healthy and have less risk of passing the virus on to others.

HIV can be suppressed by combination antiretroviral therapy (ART) consisting of three or more antiretroviral (ARV) drugs. While ART does not cure HIV infection, it controls viral replication and allows an individual’s immune system to strengthen and regain the capacity to fight off infections (www.who.int).

Even with the progress, there remain great opportunities to intensify research for better treatment and prevention of the disease. Until now, there is no cure for HIV/AIDS.

The WHO said that HIV has claimed more than 25 million lives in the past three decades. Globally, about 34 million people are living with HIV in 2011.

In the Philippines, there were 295 new HIV positive individuals confirmed by the STD/AIDS Cooperative Central Laboratory in October this year, or 48% higher compared to the same period last year. In January to October this year, there were 892 more new HIV cases, 48% more compared to the same period in 2011. The significant increase was observed starting July 2011 onwards, mostly breaching 200 or even 300 new cases per month.

The Department of Health-National Epidemiology Center (DoH-NEC) reported 2,761 HIV cases, of which 148 were reported AIDS cases, from January to October this year. Around 748 young people (15 to 24), and four children below 15 were part of this total national figure.

It added that from 1984 to 2012, there were 11,125 HIV cases in the Philippines with 1,130 AIDS cases. More than 350 people in the country have died from AIDS since 1984.

In support of the Millennium Development Goal 6 to stop or reverse the incidence of HIV/AIDS by 2015, the government through PhilHealth, has rolled out an outpatient package to increase patient access to treatment and education. First implemented in 2010, the Out-Patient HIV/AIDS Treatment (OHAT) Package aims to provide patients access to ART and the benefit was later expanded to include treatment for tuberculosis.

In the battle against HIV/AIDS, PhRMA reported that medicines in development include a gene therapy that uses genetic material to remove disease-causing aspects of the virus; a transdermal vaccine that helps suppress virus replication and destroys HIV-infected cells; and a first-in-class medicine intended to prevent the HIV virus from breaking through the cell membrane.

Many of the medicines are in Phase I and II of the long, expensive and complex drug discovery process. Phase I of the clinical trial involves a small group of people, usually between 20 and 80 healthy adult volunteers, to evaluate a drug’s initial safety and tolerability profile, determine a safe dosage range, and identify potential side effects. Phase II is the stage in which the drug is given to volunteer patients, usually between 100 and 300, to see if it is effective, identify an optimal dose, and to further evaluate its short-term safety.

When a candidate drug reaches Phase III, it is given to a larger, more diverse patient population, often between 1,000 and 3,000 patients or more, to generate statistically significant evidence to confirm its safety and effectiveness. They are the longest studies, and usually take place in multiple sites around the world (For the full report, go to www.phrma.org).

Biopharmaceutical companies have expressed commitment to continue research to prevent or treat HIV/AIDS. Policies and collaborations that encourage innovation and healthcare access are critical to this mission

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See also: 

Drug Innovation 5: The US PCAST Report on Propelling Drug Discovery, October 20, 2012

Drug Innovation 6: Dealing with Drug-Resistant TB, November 30, 2012
Drug Innovation 7: IFPMA, Superbugs and Tropical Diseases, December 04, 2012
Drug Innovation 8: Treating Men's Cancer, December 20, 2012

Drug Innovation 7: IFPMA, Superbugs and Tropical Diseases

People's lifestyle and communities evolve and continuously change. Their expectations, patience or impatience for more modern lifestyle, including more modern treatment in case they get sick, also evolve and changes. That is how modern medical science, pharmaceutical R&D, diagnostic tests and other aspects of the healthcare industry evolve.

I am reposting below three nice articles by the Executive Director of the Pharmaceutical and Healthcare Association of the Philippines (PHAP), Mr. Reiner Gloor. These are about the role of drug innovation and how to better encourage it, not demonize it. These appeared in his weekly column in BusinessWorld.

Protecting intellectual property rights (IPR) like drug patents is an important policy measure by governments to encourage innovation. Policy reversals like promoting compulsory licensing (CL) and other variants of IPR confiscation can only discourage innovation. After spending 10 to 14 years in various clinical trials and drugs R&D, and some $1 B or more per candidate drug molecule, it is not wise for governments to simply coerce the innovator companies to give away those efforts to other companies which did not spend equal amount of time and money to develop more revolutionary and more disease-killer medicines.

I have argued it over and over in this blog and other papers, that the main function of government is to promulgate the rule of law, protect private property rights, and the citizens' liberty, freedom of expression and freedom from aggression by bullies. Protecting IPR is consistent with this government role.

The three papers are entitled "Impact of innovation", "Protecting innovation", and "Encouraging innovation". Enjoy reading.

(1) Impact of innovation

Posted on 05:03 PM, November 08, 2012

Medicine Cabinet -- Reiner W. Gloor

http://www.bworldonline.com/weekender/content.php?id=61108

THERE was a time when infectious diseases such as pneumonia, typhoid fever and tuberculosis ravaged people around the world with no medicines available to treat or prevent them.

Alexander Fleming’s discovery of penicillin and subsequent research yielded new antibiotics that continue to protect the people from simple to complex infections. Since the 1920s, scientists also had their eyes on prevention, management and even cure for non-communicable diseases. Now, thousands of medicines have been produced to treat or prevent diseases, thanks to pharmaceutical innovation.

During the biennial conference of the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) in Geneva last week, the global research-based medicines industry called for a shared commitment to promote global health through the promotion of further innovation.

These medical innovations, along with other health interventions, have, after all, helped in beating polio on a global scale, and prevented measles, mumps, rubella and other ailments.

IFPMA President Dr. John Lechleiter said that in the past decade alone, vaccines for measles, polio, and diphtheria-tentanus-pertusis have saved the lives of an estimated 21.2 million children under the age of five each year.

He added that between 2000 and 2006, immunization campaigns reduced the number of deaths caused by measles by 68% worldwide and 91% in Africa. Furthermore, immunizations provided to nearly 300 million children in 72 developing countries have saved over five million lives. Reports also showed that global infant mortality fell from 77 deaths per 1,000 births to 62 or a reduction of 20% between 2000 and 2009.

Innovations in medicine have also helped increase cancer survival rates, and dramatically reduce deaths due to HIV/AIDS.

Biopharmaceutical research is likewise being focused on non-communicable diseases such as cardiovascular diseases, diabetes, chronic respiratory diseases, and cancers that have grown to become the world’s biggest killers.

The biopharmaceutical sector has similarly invested heavily on understanding so-called rare diseases or those which affect fewer than 200,000 people. A deeper look at an individual’s genetic make-up is putting personalized medicine in the forefront of today’s innovation so that detection, treatment and prevention of the disease will be more tailored to the needs of each patient (For more information on personalized medicine, go to www.innovation.org.)

People are also living longer due to medical innovations. In illustrating this, a child born in 1955 had an average life expectancy at birth of only 48 years. In 2000, a child could expect to live 66 years. Life expectancy will increase to 73 years in 2025, it is said. Dr. Lechleiter commented that developing countries are seeing the most rapid gains.

Professor Frank Lichtenberg of Columbia University disclosed “new medicines accounted for 40% of the increase in life expectancy during the 1980s and 1990s in 52 developed and developing countries.”

Apart from the impact to life and health, biopharmaceutical innovations also resulted in economic gains by way of years of productive work, economic value added, consumer spending, and taxes paid.

No less than the World Health Organization (WHO) said that a nation needs a healthy population to achieve economic development. In fact, the 2001 report by the WHO Commission on Macroeconomics and Health stated that “health is a creator and pre-requisite of development.” It emphasized that increasing the coverage of health services and a small number of critical interventions to include the world’s poor could “save millions of lives, reduce poverty, spur economic development, and promote global security.”

The IFPMA Assembly also pointed out that there is strong evidence that innovative medicines are the most cost-effective part of health care. In another study, Mr. Lichtenberg found that for every $1 spent on new medicines for cardiovascular diseases in Organization for Economic Cooperation and Development (OECD) countries, close to $4 were saved in hospitalization and other healthcare costs.

More than economic gains are the social impact of medical innovations on patients who may be our children, siblings, parents and friends.

These days, a diagnosis of a disease does not necessarily mean an end to ties with families and friends.

These gains and the processes that these innovations go through must not be taken for granted. As Mr. Lechleiter said, we must build upon and not rest upon the contributions of the past. For despite our tremendous progress, much more remains to be done.