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© 2011, 2012 Health Futures Digest
| If we can’t yet stave off cancer completely, we can at least kill it. Another cancer-killer has emerged from the Petri dish and is likely headed for clinical trials, and yet another promising anti-cancer drug is in the works.
Breakthroughs in Alzheimer’s are coming almost as fast as breakthroughs in cancer. One potential cure could be as close as four years away. And since we can now reverse memory loss in Alzheimer mice, it will not be a miracle when we can reverse it in people. The University of Alabama at Birmingham has seen the future of medicine in all of this, and the future is Neuroscience. UAB has built a major new neuroscience center and is mounting an assault on Parkinson’s, in the belief that a cure can be available within five years. Other therapeutics-related news:
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| Cancer “Executioner”
After a search through more than 20,000 structurally different synthetic compounds, University of Illinois researchers found one – called PAC-1 – that caused cancer cells to self-destruct but left healthy cells alone. The lead researcher said it could lead to a host of organic compounds with the ability to directly activate executioner enzymes, and because their effectiveness could be predicted in advance [presumably, by testing on tissues removed from the patient], the compund and dose could be tailored to the individual patient. Biotech heavyweight Genentech has signed a collaboration deal worth up to US$625 million with Inotek Pharmaceuticals, which is testing compounds that might prevent tumors from developing resistance to anticancer drugs, reports Bernadette Tansey in the San Francisco Chronicle. The compounds are designed to block the action of an enzyme known as PARP that helps cancer cells repair themselves from chemotherapy damage. The company has begun testing in glioma — a type of brain tumor — and melanoma, and is studying the compounds as a remedy for the tissue damage that can result from acute attacks of cardiovascular disease. Although PARP is normally present at low levels to repair DNA damage in otherwise-healthy cells, the enzyme is overproduced as a reaction to injuries, exposure to toxins, or the oxygen deprivation caused by clots that block circulation. The surge in PARP levels is dangerous because the enzymes use up a key molecule needed to supply cells with energy. Without that molecule, cells die. Non-surgical Cervical Cancer Treatment from HIV The recently approved cervical cancer vaccine is only good for women not already infected with the human papilloma virus (HPV) that causes it. Early lab tests in the UK have shown that HIV drug lopinavir also attacks HPV, and could be eliminate the need for surgery to remove early cervical cancers. Human clinical trials may be under way in a few years, since the drug has already been tested and approved for treating HIV. It may be administered as a cream or a pessary. It is possible that other anti-HIV drugs could be used to treat hepatitis B. There is no cure for scleroderma (“hard skin”), a chronic, often progressive rheumatic disease in which the immune system overproduces collagen, which can stiffen and thicken the skin, typically on the hands, arms, legs and face. The symptoms and their severity can vary greatly among patients, and the illness takes two main forms: Systemic, which can ravage not only the skin but internal organs including the heart, lungs and kidneys and can be life-threatening; and Localized, which can result in patches of thickened or discolored skin but does not affect internal organs, does not lead to systemic scleroderma, and is not fatal. But, writes Francine Parnes in the New York Times, there is a growing number of treatment options; but again, many of the estimated 100,000 Americans with the systemic form of the disease can’t afford the treatments and insurance will often not cover them. In part that is because the latest treatments, such as stem cell transplants which have shown great promise, are considered experimental and can easily run into five figures each time a patient receives one. The cost of intravenous immune globulin treatments, which infuse blood and new antibodies, is costing one patient US$37,000 for each of 13 prescribed infusions. The treatment is saving the patient’s life, but as of the date of Parnes’ article, he had only managed to raise $87,000 from friends and relatives. Chickens turned slightly fluorescent by the insertion of DNA from jellyfish could become “inexpensive laboratories for the production of cancer-fighting human antibodies,” reports Charlie Emrich in the Sacramento Bee. It has previously been shown that chickens can be genetically modified to produce eggs containing human antibodies, which can effectively target diseases including cancer. Now, researchers have taken primordial germ cells (PGCs) — found only in embryos — from an egg, grown and genetically modified them in the lab to produce human antibodies and added jellyfish DNA, and then injected them into another egg, where the PGCs developed into the reproductive cells of the eventual hatched, fluorsecent, chick. The fluorescence simply confirms that the genetic modifications worked, so producers can be confident that the protein harvested from the eggs of the modified chick is the desired human antibody. The result is a cheaper and easier way to produce human antibodies to fight killer diseases. Pharma Holy Grail Within Grasp A for-profit spinoff from University of Washington research is in the animal studies stage of developing drugs that could protect organs when blood flow is restored following heart attacks, strokes, or other conditions, writes John Cook in the Seattle Post-Intelligencer. If it succeeds – and several heavyweight venture capitalists are bettign that it will — it would be “a significant advance in patient care,” acording to no less an authority than systems biology’s leading guru Leroy Hood. The proteomics underlying the approach are apparently a closely guarded secret. One of the investors in the startup told Cook that the technology came “totally out of left field, and that is why we love it.” He added: “This has been a holy grail in the pharmaceutical industry for a decade or 20 years. People have been trying to figure out how to shut the inflammatory and immunological response down for a long time. Just about every pharmaceutical company in the world has been working on this or is working on this. This is not easy.” The UW will receive an equity stake in the company and receive royalties if a drug is ever brought to market. Human clinical trials could begin in two years. Inhaled nitric oxide has been found to reduce the incidence of the chronic lung disease bronchopulmonary dysplasia in some premature babies, which can stunt growth and leave children prone to colds and other infections, reports Regina McEnery in the Cleveland Plain Dealer, citing an article in July 27 issue of the New England Journal of Medicine. Nitric oxide is present naturally in cells that line blood and lymph vessels and the heart. When released from the cells, the arteries relax, easing blood pressure and increasing oxygenated blood flow to the arteries. Premature babies are thought to be deficient in nitric oxide, so they are fed the gas artificially through an endotracheal tube. A physician involved in one study cautioned that the therapy might not be beneficial for profoundly ill infants in respiratory failure, but said it was “the treatment for the vast majority of premature babies who do make it.” Another study found that nitric oxide reduced the risk of brain injury but had little impact on chronic lung disease when the infants weighed less than 2.2 pounds at birth. Memory Restored in Alzheimer Mice Alzheimer’s patients slowly lose not just their memories, but also their ability to memorize. That inability was reversed in transgenic mice suffering from human Alzheimer’s, by injecting them with a substance that boosted levels of the enzyme Uch-L1. The researchers then gave the mice a very mild shock through the floor of a cage. Mice with normal memories stay still when they are placed in the cage for a second time, to avoid the shock, but mice with Alzheimer’s do not because they cannot remember their first visit. The treated mice stayed still. The enzyme appeared to repair the synapse function damaged by the disease. The study showed that even in the presence of amyloid beta plaque, damage to memory capability can be reversed. A researcher said: “While this discovery is very promising, its proven effectiveness is limited to animal models and it will take some time before it could lead to therapies in humans.” An official of the Alzheimer’s Research Trust aslo cautioned that while “This is a very encouraging piece of research . . . . at this stage a cure is still a long way off.” Towards a Cure for Alzheimer’s Australian researchers have developed a once-a-day pill containing a drug known as PBT2 (related to Clioquinol, an ointment used to treat skin infections such as athlete’s foot) that could effectively cure Alzheimer’s disease. It worked in mice, whose amyloid protein levels fell by 60 percent within 24 hours of a single dose, and memory performance improved within five days. Preliminary tests in humans have already shown that the drug does not cause any major side-effects. Further human clinical trials were due to start in August, and a major international trial is planned for 2007. If all of these succeed, the drug could be on the market in four years. This was just one of many encouraging discoveries presented at the International Convention for Alzheimer’s Disease in July. Towards a Cure for Parkinson’s The University of Alabama at Birmingham (UAB)’s Center for Neurodegeneration and Experimental Therapeutics, established to speed the translation of basic scientific discoveries into treatments, is mounting an “assault” on Parkinson’s, reports Dave Parks in the Birmingham News. Two recent discoveries helped prompt the assault: First, the discovery of the genes that predispose some people to Parkinson’s, which has allowed scientists to design mice with Parkinson’s and test treatments on them; and second, the discovery of what appears to be a root cause of Parkinson’s – the overproduction of alpha-synuclein, a protein which, in overabundance, causes clumps of misshapen proteins to gather in dopamine-producing cells, which either become sick or die. Among other things, UAB will participate in the study of a gene therapy that introduces a growth factor for dopamine called neuturin. The therapy has passed some safety trials with humans, and it slowed progression of the disease and restored some function in monkeys and mice. The UAB Center’s lead researchers believe there will be a treatment available to slow or reverse Parkinson’s in as little five years, writes Parks, though several different treatments will have to be developed since, like cancer, there are many forms of the disease. The ambition of the Center’s larger research initiative — against all neurodegenerative and psychiatric diseases, from Alzheimer’s to schizophrenia – can be seen its recruitment of top researchers from top schools including the Emory University School of Medicine, Baylor College of Medicine, the University of Michigan, and Washington University School of Medicine. The Center will continue to expand, having secured significant grant funding, because “There is tremendous opportunity in neuroscience right now. We have just gone through an explosion of understanding the brain,” its leader told Parks. Wet Macular Degeneration Reversed In July, the US Food and Drug Administration approved Genentech’s “Lucentis,” the first drug that improves vision in elderly people with the early stage “wet” form of age-related macular degeneration (AMD). The drug costs US$1,950 per dose and typically requires five to seven treatments. Medicare is “likely” to pay for it, says medical writer Patricia Anstett in the Detroit Free Press. Genentech provides drugs free to uninsured people with incomes of less than $75,000 a year. Ophthalmologists have hailed the drug as a breakthrough. “A metro Detroit woman whose vision improved significantly after three treatments called it a miracle,” says Anstett. People who got the drug in the early stages of the disease had the best vision improvements. The drug does not benefit patients who have progressed to stages of the disease where scars form inside the eye. Treatments for the dry form of AMD are limited, Artificial Blood: Drive Continues As of July, the US Food and Drug Administration was reconsidering its rejction of a US Navy trial of Biopure Corporation’s blood substitute, “Hemopure.” More than 900 badly hemorrhaging civilian accident victims around the country would be involved, write Thomas Burton in the Wall Street Journal, with some receiving the blood substitute and others saline solution en route to hospitals. Non-consent studies are “rare but legal” where there is no practical way to obtain consent, such as when patients are in shock or unconscious, and where there is a reasonable likelihood that individual patients would benefit from the proposed treatment. The Navy wants a blood substitute because donor blood lasts only about 42 days before its oxygen-carrying capacity is diminished, while artificial blood could be stored aboard ships for an estimated three years. Northfield Laboratories is nearing completion of a non-consent, 720 trauma patient study of its own “PolyHeme” blood substitute. In a previous trial of surgery patients, 10 of 81 people who received PolyHeme suffered heart attacks, versus zero of 71 receiving donor blood. Past studies have raised questions about Hemopure as well. A clinical study comparing Hemopure with donor blood in consenting orthopedic-surgery patients concluded that Hemopure was no’t only not working well but also was “harmful.” The Navy has maintained that a study of trauma patients would be significantly different from the trial with surgery patients, and argues that the oxygen-carrying capacity of Biopure’s product could lower the death rate in trauma patients by 25 percent versus saline. Jesse Sullivan, 59, is the first amputee to be fitted with a thought-controlled artificial arm. He thinks, “Close hand,” and his prosthetic hand closes. He is testing the arm as part of a joint US National Institutes of Health and Defense Advanced Research Projects Agency initiative to refine artificial limbs controlled by BMIs (brain-machine interfaces). The arm was described by its developer at the Center for Artificial Limbs at the Rehabilitation Institute of Chicago as “not as smooth as a normal arm but . . . much smoother than a normal prosthesis.” British researchers have demonstrated how nerve cells can grow along bundles of a genetically modified silk worm fiber called “Spidrex” (for its similarity to spider silk) which appears to encourage cell re-growth when used to connect severed nerves, and might even repair damaged spinal cords. In both in-vitro and in-vivo animal tests, the silk acted as a scaffold on which nerve cells grew. In the animal studies, Spidrex supported nerve growth both in the spinal cord and in peripheral nerves. Spidrex can be assembled into complex tubes designed to fit the nerves and the length of the gap that needed bridging. The researchers anticipate the silk may first be used to treat patients with peripheral nerve damage — someone who has received a bad cut to the hand, for example. The repair of damaged spinal cords, being much more complex, will come later. Early clinical trials of a technology based on the model of deer antlers which enables artificial limbs to be attached to a human skeleton without risk of infection as the prosthesis breaches the skin have been “very promising.” University College London scientists developed the “Intraosseous Transcutaneous Amputation Prosthesis” (ITAP) method, in which a titanium rod is passed through the skin and attached directly into the bone. An artificial limb can then be attached to the rod. Infection that could be caused by bacteria passing from the external limb through the rod to the bone is avoided because the skin tissue meshes around the rod to form both a seal and an anchor point strengthening the tissue/rod joint, just like deers’ antlers. Early clinical trials on a small group of patients who had lost fingers or thumbs were very encouraging, according to a BBC News report. Future trials will involve upper and lower limb replacements. The technology could be widely used for thumb and forefingers in a few years, and upper and lower limb replacements using this method could be in place in five years. The chairman of the Limbless Association welcomed the advance but “on the downside, I would still be worried about infection.” |
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